Moorestown, New Jersey, United States
biotecHOPE™
Every Victory – BiotecHOPE™
BiotecHOPE™ Every Victory
Every Victory–BiotecHOPE
May 23, 2021 2:00 PM ETABBV, AMGN...
Summary
- the virus impact on Latin America, Brazil, India, Nepal, and many other countries brings such devastating and depressing news that it is difficult to feel upbeat about any biotech victories.
- In the time of many medical advances the bitcoin “Crypto” issue is irrelevant.
- I have been adding long positions in (MRK)(GILD)(AMGN)(JNJ)(ABBV)(AZN)(LLY) since September 2020.
- upbeat biotech/medical news is still not enough to counter the ongoing politico attacks against the biotech and bio-pharma community.
- “We will lift our eyes, we won't fear the fight"
- https://seekingalpha.com/instablog/5205071-danwatson888/5594643-every-victory-biotechope
Vaccinations
Covid-19 Tracker
Johns Hopkins University & Medicine
Johns Hopkins experts in global public health, infectious disease, and emergency preparedness have been at the forefront of the international response to COVID-19.
Coronavirus Vaccine Tracker
BiotecHOPE™ New Day
BiotecHOPE™ New Day
Mar. 14, 2021 3:08 PM ET. AZN, BNTX, JNJ, LLY, MRK, MRNA, NVAX, PFE, VIR
Summary
- The vaccine and therapeutic news and updates coming on the Covid-19 disease has been nothing less than remarkable.
- For once HOPE, and specifically www.biotecHOPE.com is in the air.
- Unfortunately, the www.biohazard2020.com is not over, the virus continues to mutate and is still an issue to those who are susceptible and/or at risk.
- I am increasing my long positions in the following companies – (MRK), (ABBV), (AMGN), (JNJ), (GILD), (BMY), (LLY) and will consider adding the following (MRNA), (BNTX), (NVAX), (VIR).
- even with all the positive vaccine news, I am not feeling as upbeat as I thought I would regarding my personal life and the lives of my family.
https://seekingalpha.com/instablog/5205071-danwatson888/5566982-biotechope-new-day
Welcome to BiotecHOPE™ and thank you for visiting www.biotecHOPE.com.
BiotecHOPE™
There are many new medicines in testing, development and clinical trials that are currently not available or recently approved to the marketplace and many are having encouraging and positive results. There are also many approved meds that are in tests involved for other ailments/conditions that might work, where these new trials will determine. BiotecHOPE™ will seek to highlight these trials/efforts many of which are from smaller/newer biotech companies and/or are being developed in partnership with Universities, Hospitals, Medical Centers, larger Bio-Pharma companies.
Welcome to BiotecHOPE™ and thank you for visiting www.biotechope.com.
#DannyGokey #NewDay #MusicVideo
Danny Gokey - New Day (Official Music Video)
•Premiered Jan 29, 2021 Danny Gokey
New Day
Wake up and breathe in deeper than yesterday Take on the morning like your soul's been remade
Roll down the windows, let your cares fly away Good things are yours to claim, you don't have to wait
All across the sky New mercies rise And the future's bright This is a new day Everything bursting with hope
Coming alive This moment, moment You've got a freedom No looking back anymore Open your eyes
It's coming, coming This is a new day The old has gone away The old has gone away This is a new day
Don't let it slip away Don't let it slip away Go on and reignite impossible dreams
Become the one you never thought you could be, woah
All across the sky New mercies rise And the future's bright Oh-oh-ooh-ohh
This is a new day (come on) Everything bursting with hope (woah)
Coming alive This moment, moment You've got a freedom (you've got a freedom)
No looking back anymore (no looking back anymore)
Open your eyes It's coming, coming This is a new day The old has gone away The old has gone away
This is a new day Don't let it slip away Don't let it slip away This is a new day The old has gone away
The old has gone away This is a new day Don't let it slip away Don't let it slip away
Love hasn't give up on you Love hasn't give up on you Love is making the old things new
Today and every single day Love hasn't give up on you Love hasn't give up on you Love... (1, 2, 3)
A new day, a new day This is a new day Everything bursting with hope
Coming alive This moment, moment You've got a freedom (you've got a freedom)
No looking back anymore (no looking back anymore)
Open your eyes It's coming, coming This is a new day The old has gone away The old has gone away
This is a new day Don't let it slip away Don't let it slip away This is a new day The old has gone away
The old has gone away This is a new day (a new day, a new day)
Don't let it slip away Don't let it slip away This is a new day It's a new day It's a new day It's a new day
Yeah, yeah It's a new day It's a new day It's a new day
Songwriters: Danny Gokey, Colby Wedgeworth, Ethan Hulse
Won't Stop Now feat. Brandon Lake | Live | Elevation Worship
•Jul 20, 2020 Elevation Worship
Written by Steven Furtick, Chris Brown
biotecHOPE™ Won’t Stop Now
biotecHOPE™ Won’t Stop Now
Aug. 26, 2020 5:37 PM ET|
About: AstraZeneca PLC (AZN), BNTX, CYDY, GILD, GSK, MRK, NVAX, PFE, SNY
Summary
"The country behaved like a horror-movie character who believes the danger is over, even though the monster is still at large."
there is encouraging news coming from biotech/pharma companies with regard to vaccines and therapies to fight this virus.
"I know breakthrough is coming, By faith I see a miracle
My God made me a promise, And it won't stop now""
biotecHOPE™ July/June/May/Apr/Mar/Feb/Jan/2021
VAXNEUVANCE™ (Pneumococcal 15-valent Conjugate Vaccine)
RYLAZE™ (asparaginase erwinia chrysanthemi (recombinant)-rywn), Formerly JZP458
VAXNEUVANCE™ (Pneumococcal 15-valent Conjugate Vaccine)
Merck Announces VAXNEUVANCE™ (Pneumococcal 15-valent Conjugate Vaccine) Met Key Immunogenicity and Safety Endpoints in Phase 3 Pivotal Trial Evaluating Use in Infants, PNEU-PED (V114-029)
August 25, 2021 6:45 am ET
VAXNEUVANCE Also Met Key Safety Objectives in the Phase 3 PNEU-LINK (V114-031) Study in Infants
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced topline results from the pivotal PNEU-PED (V114-029) study evaluating the immunogenicity, safety and tolerability of VAXNEUVANCE™ (Pneumococcal 15-valent Conjugate Vaccine) in healthy infants enrolled between 42-90 days of age (n=1720). In the trial, infants were given a 4-dose regimen of either VAXNEUVANCE or the licensed 13-valent pneumococcal conjugate vaccine (PCV13) at 2, 4, 6, and 12-15 months of age.
In the PNEU-PED study, primary endpoints demonstrated:
- VAXNEUVANCE had a safety profile generally comparable to PCV13 following receipt of any vaccine dose.
- At 30 days following the third dose in the series (PD3), VAXNEUVANCE was non-inferior to PCV13 for all 13 shared serotypes based on serotype-specific response rates (proportions of individuals achieving the accepted immunoglobulin G (IgG) threshold value of 0.35mcg/mL) and for 12 of the 13 shared serotypes based on serotype-specific IgG geometric mean concentrations (GMCs).
- The lower bounds of the two-sided 95 percent confidence intervals (CI) for the serotype-specific IgG GMC ratios were greater than 0.5 for 12 shared serotypes; the lower bound was 0.48 for serotype 6A, narrowly missing non-inferiority criteria.
- At 30 days following the fourth (toddler) dose (PD4), VAXNEUVANCE was non-inferior to PCV13 for all 13 shared serotypes based on serotype-specific IgG GMCs.
Secondary endpoints demonstrated statistically superior immune responses for VAXNEUVANCE in comparison to PCV13 for shared serotype 3 and unique serotypes 22F and 33F based on prespecified criteria, as well as non-inferior immune responses to antigens contained in several routinely used pediatric vaccines when administered concomitantly with VAXNEUVANCE or PCV13.
“By nature, pneumococcal disease is constantly evolving. Strains of the disease associated with invasiveness cause significant disease burden in children, calling for innovation to help protect this vulnerable population worldwide,” said Dr. Roy Baynes, senior vice president and head of global clinical development, chief medical officer, Merck Research Laboratories. “At Merck, our goal is to design pneumococcal vaccines that target strains causing the greatest proportion of disease while maintaining a strong immune response to these serotypes. With the inclusion of serotypes 22F and 33F, VAXNEUVANCE has the potential to play an important role in the prevention of invasive pneumococcal disease in children.”
In PNEU-LINK (V114-031), a Phase 3 study to evaluate the safety and tolerability of VAXNEUVANCE in healthy infants, VAXNEUVANCE was generally well-tolerated with a safety profile generally comparable to PCV13 in healthy infants enrolled at 42-90 days of age (n=2409). Full results from the PNEU-PED and PNEU-LINK studies will be presented at an upcoming scientific congress.
There are 100 different types of pneumococcal bacteria, which can affect children differently than adults. Children under the age of 2 are particularly vulnerable to pneumococcal infection, and incidence of invasive pneumococcal disease remains highest in the first year of life. Certain pneumococcal serotypes continue to put children at risk, including serotypes 22F, 33F and 3, which represent more than a quarter of all cases of invasive pneumococcal disease in children under the age of 5.
The VAXNEUVANCE Phase 3 clinical development program is comprised of 16 trials investigating the safety, tolerability and immunogenicity of VAXNEUVANCE in a variety of populations who are at increased risk for pneumococcal disease, including 10 that investigated VAXNEUVANCE for use in children and infants.
On July 16, 2021, the U.S. Food and Drug Administration (FDA) approved VAXNEUVANCE for adults 18 years of age and older for active immunization for the prevention of invasive disease caused by the 15 S. pneumoniae serotypes contained in the vaccine. Plans are on track for submission of a supplemental regulatory licensure application to the FDA for use in children before the end of the year.
About PNEU-PED
PNEU-PED is a Phase 3, multicenter, randomized, double-blind, active-comparator-controlled study evaluating the safety, tolerability and immunogenicity of a 4-dose regimen of VAXNEUVANCE in healthy infants enrolled at 42-90 days of age (n=1720).
In the primary analysis, participants were randomized to receive VAXNEUVANCE or PCV13. Immune responses for the 13 shared serotypes contained in VAXNEUVANCE and PCV13 were measured at 30 days post-dose three (PD3) and post-dose four (PD4). Immune responses were assessed based on anti-pneumococcal polysaccharide (PnPs) serotype-specific immunoglobulin G (IgG) response rates at 30 days PD3 and geometric mean concentrations (GMCs) at 30 days PD3 and PD4.
In the secondary analysis, antibody responses to select licensed pediatric vaccines were evaluated when administered concomitantly with VAXNEUVANCE or PCV13. Additionally, immune responses for serotypes 22F and 33F, the two serotypes unique to VANXNEUVANCE, were assessed based on anti-PnPs serotype-specific IgG response rates at 30 days PD3 and PD4, and immune responses for serotype 3 were assessed based on anti-PnPs serotype-specific IgG response rates at 30 days PD3 and IgG GMCs 30 days PD3 and PD4.
Safety analyses were performed in the All-Participants-As-Treated (APaT) population, defined as all randomized participants who received at least one dose of study vaccination.
About VAXNEUVANCE (Pneumococcal 15-valent Conjugate Vaccine)
VAXNEUVANCE, Merck’s 15-valent pneumococcal conjugate vaccine, consists of purified capsular polysaccharides from S. pneumoniae serotypes 1, 3, 4, 5, 6A, 6B, 7F, 9V, 14, 18C, 19A, 19F, 22F, 23F and 33F individually conjugated to CRM197 carrier protein. VAXNEUVANCE is indicated for active immunization of adults 18 years of age and older for the prevention of invasive disease caused by the S. pneumoniae serotypes contained in the vaccine. It is currently under investigation in the pediatric population. VAXNEUVANCE previously received Breakthrough Therapy designation from the FDA for the prevention of invasive pneumococcal disease in pediatric patients 6 weeks to 18 years of age.
Merck is involved in litigation challenging the validity of several Pfizer Inc. patents that relate to pneumococcal vaccine technology in the United States and several foreign jurisdictions.
To learn more about Merck’s infectious diseases pipeline, visit www.merck.com.
For more information, visit www.merck.com
Please see Prescribing Information for VAXNEUVANCE (pneumococcal 15-valent conjugate vaccine) at https://www.merck.com/product/usa/pi_circulars/v/vaxneuvance/vaxneuvance_pi.pdf and Patient Information at https://www.merck.com/product/usa/pi_circulars/v/vaxneuvance/vaxneuvance_ppi.pdf.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210825005054/en/
Source: Merck & Co., Inc.
Merck's Vaxneuvance met key endpoints in late-stage PNEU-PED (V114-029) trial
Aug. 25, 2021 7:51 AM ET
By: Mamta Mayani, SA News Editor
- Merck (NYSE:MRK) announces topline results from the PNEU-PED (V114-029) study evaluating the immunogenicity, safety and tolerability of VAXNEUVANCE (Pneumococcal 15-valent Conjugate Vaccine) in healthy infants enrolled between 42-90 days of age (n=1720).
- https://seekingalpha.com/news/3733883-mercks-vaxneuvance-met-key-endpoints-in-late-stage-pneu-ped-v114-029-trial
- https://seekingalpha.com/symbol/MRK
- https://www.merckvaccines.com/vaxneuvance/
CFT7455 is an orally bioavailable MonoDAC™ degrader
RYLAZE™ (asparaginase erwinia chrysanthemi (recombinant)-rywn), Formerly JZP458
VAXNEUVANCE™ (Pneumococcal 15-valent Conjugate Vaccine)
C4 Therapeutics Announces FDA Orphan Drug Designation for CFT7455 for the Treatment of Multiple Myeloma
WATERTOWN, Mass., Aug. 11, 2021 (GLOBE NEWSWIRE) -- C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company pioneering a new class of small-molecule medicines that selectively destroy disease-causing proteins through degradation, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to CFT7455 for the treatment of multiple myeloma.
The FDA’s Office of Orphan Products Development grants orphan designation status to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases, or conditions that affect fewer than 200,000 people in the U.S. Orphan Drug Designation provides certain benefits, including financial incentives, to support clinical development and the potential for up to seven years of market exclusivity in the U.S. upon regulatory approval.
“We are pleased to receive FDA’s orphan drug designation for CFT7455 in multiple myeloma and believe this designation highlights the potential of CFT7455 to improve clinical outcomes for patients with multiple myeloma who face an incurable disease,” said Adam Crystal, M.D., Ph.D., chief medical officer of C4 Therapeutics. “With far too many patients relapsing on numerous lines of therapy and succumbing to multiple myeloma, we are focused on advancing our Phase 1/2 trial to bring this new treatment option to patients.”
CFT7455 is an orally bioavailable MonoDAC™ degrader targeting IKZF1/3 for the treatment of multiple myeloma and non-Hodgkin’s lymphomas, including peripheral T-cell lymphoma and mantle cell lymphoma. In June 2021, C4T initiated the Phase 1/2 clinical trial to primarily investigate safety, tolerability, and anti-tumor activity, with secondary and exploratory objectives to characterize the pharmacokinetic and pharmacodynamic profile of CFT7455. Across the Phase 1/2 trial, C4T plans to enroll approximately 160 patients.
To learn more about C4 Therapeutics, visit www.c4therapeutics.com.
https://c4therapeutics.com/pipeline
FDA grants orphan drug status to C4 Therapeutics' multiple myeloma treatment CFT7455
Aug. 11, 2021 4:06 PM ET
By: Aakash Babu, SA News Editor
- C4 Therapeutics (NASDAQ:CCCC) announces that the U.S. FDA has granted orphan drug designation (ODD) to CFT7455 for the treatment of multiple myeloma.
- CFT7455 is an orally bioavailable MonoDAC degrader targeting IKZF1/3 for the treatment of multiple myeloma and non-Hodgkin’s lymphomas, including peripheral T-cell lymphoma and mantle cell lymphoma.
- https://seekingalpha.com/news/3729087-fda-grants-orphan-drug-status-to-c4-therapeutics-multiple-myeloma-treatment-cft7455
- https://seekingalpha.com/symbol/CCCC
- https://c4therapeutics.com/
RYLAZE™ (asparaginase erwinia chrysanthemi (recombinant)-rywn), Formerly JZP458
RYLAZE™ (asparaginase erwinia chrysanthemi (recombinant)-rywn), Formerly JZP458
RYLAZE™ (asparaginase erwinia chrysanthemi (recombinant)-rywn), Formerly JZP458
LIGAND PARTNER JAZZ PHARMACEUTICALS LAUNCHES RYLAZE™ (ASPARAGINASE ERWINIA CHRYSANTHEMI (RECOMBINANT)-RYWN), FORMERLY JZP458
Download as PDFJuly 21, 2021
Ligand expects to receive $7 million in milestone payments
Rylaze for the treatment of ALL or LBL utilizes Ligand’s Pelican Expression Technology™ Platform
EMERYVILLE, Calif.--(BUSINESS WIRE)-- Ligand Pharmaceuticals Incorporated (NASDAQ: LGND) today announced Jazz Pharmaceuticals plc (NASDAQ: JAZZ) has launched Rylaze™ (asparaginase erwinia chrysanthemi (recombinant)-rywn), also known as JZP458. Rylaze, which was approved by the FDA on June 30, 2021, is a recombinant erwinia asparaginase used as a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LBL) in adult and pediatric patients 1 month or older who have developed hypersensitivity to E. coli-derived asparaginase.
Under the terms of the license agreement with Jazz Pharmaceuticals, Ligand received a $2 million payment upon FDA’s acceptance for review of the product BLA and is entitled to receive a $5 million payment upon the first commercial sale following launch. Ligand is eligible to receive up to an additional $155.5 million in milestone payments and tiered low to mid-single digit royalties based on worldwide net sales of any products resulting from this collaboration, including Rylaze.
“This partnership with Jazz Pharmaceuticals is one of the core scientific programs that catalyzed our acquisition of Pfenex last year. The Rylaze commercial launch really showcases our highly productive partnership with Jazz and the exceptional ability of our Pelican Expression Technology to enable life-saving therapeutics,” said John Higgins, CEO of Ligand. “The robust manufacturing afforded by Ligand’s Pelican Expression Technology combined with Jazz’s demonstrated success in development and commercialization has enabled the delivery of a high-quality recombinant asparaginase option for patients with hypersensitivity to E. coli-derived asparaginase with reliable supply.”
For more information, please visit www.pelicanexpression.com.
For more information, please visit www.ligand.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210721005347/en/
Source: Ligand Pharmaceuticals Incorporated
Released July 21, 2021
Jazz Pharmaceuticals launches leukemia/lymphoma treatment Rylaze
Jul. 21, 2021 9:55 AM ET
Ligand Pharmaceuticals Incorporated (LGND), JAZZ
By: Aakash Babu, SA News Editor
- Ligand Pharmaceuticals (LGND -1.2%) partner Jazz Pharmaceuticals (JAZZ +1.8%) has launched Rylaze for the treatment of acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LBL) in certain adult and pediatric patients.
- https://seekingalpha.com/news/3717418-jazz-pharmaceuticals-launches-leukemialymphoma-treatment-rylaze
- https://seekingalpha.com/symbol/JAZZ
- https://seekingalpha.com/symbol/LGND
- https://www.rylaze.com/
- https://www.jazzpharma.com/science/pipeline/
COSELA™ (TRILACICLIB)
COSELA™ (TRILACICLIB)
RYLAZE™ (asparaginase erwinia chrysanthemi (recombinant)-rywn), Formerly JZP458
G1 THERAPEUTICS RECEIVES FAST TRACK DESIGNATION FROM U.S. FOOD AND DRUG ADMINISTRATION FOR COSELA™ (TRILACICLIB) IN COMBINATION WITH CHEMOTHERAPY FOR THE TREATMENT OF LOCALLY ADVANCED OR METASTATIC TRIPLE NEGATIVE BREAST CANCER
July 19, 2021 at 9:20 AM EDTPDF Version
RESEARCH TRIANGLE PARK, N.C., July 19, 2021 (GLOBE NEWSWIRE) -- G1 Therapeutics, Inc. (Nasdaq: GTHX), a commercial-stage oncology company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to COSELA™ (trilaciclib) investigation for use in combination with chemotherapy for the treatment of locally advanced or metastatic triple negative breast cancer (TNBC). COSELA is currently being evaluated in PRESERVE 2, a pivotal Phase 3, randomized, double-blind, placebo-controlled study (NCT04799249) in patients receiving first- or second-line gemcitabine and carboplatin chemotherapy for TNBC. (press release)
Fast track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill unmet medical needs. The purpose is to get important new drugs to the patient earlier. A drug that receives Fast Track designation may be eligible for more frequent engagements with the FDA to discuss the drug’s clinical development plan, eligibility for Accelerated Approval and Priority Review, and Rolling Review in which the Company can submit completed sections of its New Drug Application (NDA) for FDA review rather than waiting until every section of the NDA is completed before the entire application can be reviewed.
For additional information, please visit www.g1therapeutics.com
G1 Therapeutics gets FDA fast track for breast cancer treatment COSELA
Jul. 19, 2021 9:45 AM ET
By: Aakash Babu, SA News Editor2 Comments
- G1 Therapeutics (GTHX -4.7%) announces that the U.S. FDA has granted Fast Track designation to COSELA (trilaciclib) for use in combination with chemotherapy for the treatment of locally advanced or metastatic triple negative breast cancer (TNBC).
- https://seekingalpha.com/news/3716317-g1-therapeutics-gets-fda-fast-track-for-breast-cancer-treatment-cosela
- https://seekingalpha.com/symbol/GTHX
- https://www.g1therapeutics.com/pipeline/trilaciclib/
- https://www.cosela.com/patient
- https://www.g1therapeutics.com/
Leronlimab
COSELA™ (TRILACICLIB)
Leronlimab
CytoDyn Announces Preliminary Results from 30 mTNBC Patients Treated with Leronlimab. Decreases in CAMLs after 4 Doses of Leronlimab were Identified in Over 70% of Patients and were Associated with a 450% Significant Increase in Overall Survival at 12-Month Analysis
Download as PDFJuly 19, 2021 6:00am EDT
CytoDyn will seek FDA guidance on proceeding with an expedited regulatory plan for approval of leronlimab with existing FDA Fast Track designation for mTNBC
VANCOUVER, Washington, July 19, 2021 (GLOBE NEWSWIRE) -- CytoDyn Inc. (OTCQB: CYDY) ("CytoDyn" or the "Company"), a late-stage biotechnology company developing leronlimab, a CCR5 antagonist with the potential for multiple therapeutic indications, announced today strong preliminary results from its Phase 1b/2 trials and compassionate use with a total of 30 metastatic triple-negative breast cancer (mTNBC) patients. Patients in Phase 1b/2 were treated with leronlimab in combination with carboplatin.
Key findings from the interim 12-month analysis include the following:
- 72% of patients had a decrease in CAMLs (cancer-associated macrophage-like cells) ~30 days after induction of leronlimab
- The decrease in CAMLs was associated with:
- A ~300% increase in mean progression-free survival (mPFS)
- A significant ~450% increase in overall survival (OS) at 12 months
- High CCR5 in tumor tissue biopsies may help to stratify patients likely to progress on leronlimab
- Decreases in CAMLs and CTCs (circulating tumor cells) appear to be related to slower progression and lower mortality
- CAMLs appear to identify populations that are responding to leronlimab
About Leronlimab
The U.S. Food and Drug Administration (FDA) granted CytoDyn Fast Track designation to explore two potential indications using leronlimab to treat Human Immunodeficiency Virus (HIV) and metastatic cancer. The first indication is combination therapy with HAART for HIV-infected patients, and the second is for metastatic triple-negative breast cancer (mTNBC). Leronlimab is an investigational humanized IgG4 mAb that binds to CCR5, a cellular receptor important in HIV infection, tumor metastases, and other diseases, including nonalcoholic steatohepatitis (NASH). Leronlimab has been studied in 16 clinical trials involving more than 1,200 people and met its primary endpoints in a pivotal Phase 3 trial (leronlimab combined with HIV standard care in patients with multi-drug resistance to current available classes of HIV drugs).
Leronlimab, among various potential applications, is a viral-entry inhibitor in HIV/AIDS. It binds to CCR5, thus protecting healthy T cells from viral infection by blocking the predominant HIV (R5) subtype from entering those cells. Leronlimab does not work on other strains of HIV (for example X4), however, R5 is the most dominant strain of HIV. Five clinical trials have demonstrated leronlimab could significantly reduce or control HIV viral load in humans. The leronlimab antibody appears to be a powerful antiviral agent with fewer side effects and less frequent dosing requirements than currently used daily drug therapies. Cancer research has shown CCR5 may play a role in tumor invasion, metastases, and tumor microenvironment control (for example, through angiogenesis). Published studies have shown that blocking CCR5 can reduce tumor metastases in laboratory and animal models of aggressive breast and prostate cancer. Leronlimab reduced human breast cancer metastasis by more than 97% in a murine xenograft model. As a result, CytoDyn is conducting two clinical trials, one, a Phase 2 in mTNBC, which was granted Fast Track designation by the FDA in 2019, and a second, a Phase 2, basket trial which encompasses 22 different solid tumor cancers.
The CCR5 receptor plays a central role in modulating immune cell trafficking to sites of inflammation. After completing two clinical trials with COVID-19 patients (a Phase 2 and a Phase 3), CytoDyn initiated a Phase 2 investigative trial for post-acute sequelae of SARS COV-2 (PASC), also known as COVID-19 Long-Haulers. This trial will evaluate the effect of leronlimab on clinical symptoms and laboratory biomarkers to further understand the pathophysiology of PASC. It is currently estimated that between 10-30% of those infected with COVID-19 develop long-term sequelae. Common symptoms include fatigue, cognitive impairment, sleep disorders, and shortness of breath. If this trial is successful, CytoDyn plans to pursue clinical trials to evaluate leronlimab’s effect on immunological dysregulation in other post-viral syndromes, including myalgic encephalomyelitis/chronic fatigue syndrome (ME/CFS).
CytoDyn is also conducting a Phase 2 clinical trial for NASH to evaluate the effect of leronlimab on liver steatosis and fibrosis. Preclinical studies revealed a significant reduction in NAFLD and a reduction in liver fibrosis using leronlimab. There are currently no FDA approved treatments for NASH, which is a leading cause of liver transplant. About 30 to 40 percent of adults in the U.S. live with NAFLD, and 3 to 12 percent of adults in the U.S. live with NASH. There have been no strong safety signals identified in patients administered leronlimab in multiple disease spectrums, including patients with HIV, COVID-19, and oncology.
More information is at www.cytodyn.com.
CytoDyn reports preliminary leronlimab results from mid-stage breast cancer study
Jul. 19, 2021 6:19 AM ET
CytoDyn Inc. (CYDY)CytoDyn Inc. (CYDY)
By: Mamta Mayani, SA News Editor12 Comments
- CytoDyn (OTCQB:CYDY) announces strong preliminary results from its Phase 1b/2 trials and compassionate use with a total of 30 metastatic triple-negative breast cancer (mTNBC) patients, treated with leronlimab in combination with carboplatin.
- https://seekingalpha.com/news/3716166-cytodyn-reports-preliminary-leronlimab-results-from-mid-stage-breast-cancer-study
- https://seekingalpha.com/symbol/CYDY
- https://www.cytodyn.com/pipeline
- https://www.cytodyn.com/
ADXS-504
COSELA™ (TRILACICLIB)
Leronlimab
Advaxis Announces Initiation of Phase 1 Clinical Trial of ADXS-504 for the Treatment of Early Prostate Cancer
Study in biochemically recurrent prostate cancer expands off-the-shelf ADXS-HOT program to second indication
July 15, 2021 08:00 ET | Source: Advaxis, Inc.
- ...
MONMOUTH JUNCTION, N.J., July 15, 2021 (GLOBE NEWSWIRE) -- Advaxis, Inc. (Nasdaq: ADXS), a clinical-stage biotechnology company focused on the development and commercialization of immunotherapy products, today announced the initiation of its Phase 1 clinical study evaluating ADXS-504 in patients with biochemically recurrent prostate cancer. The study, being conducted at Columbia University Irving Medical Center, is the first clinical evaluation of ADXS-504, Advaxis’ off-the-shelf neoantigen immunotherapy drug candidate for early prostate cancer. Mark Stein, M.D., associate professor of medicine in the Division of Hematology/Oncology at Columbia University Vagelos College of Physicians and Surgeons, is the study’s principal investigator.
“Evaluating ADXS-504 in patients with biochemically recurrent prostate cancer is the first step in testing whether Lm-based immunotherapies may have a role in the adjuvant setting in cancer patients who have undergone radical therapy and who have no detectable primary tumor or metastatic disease at enrollment,” said Dr. Andres Gutierrez, Chief Medical Officer of Advaxis. “This Phase 1 study will evaluate the safety, tolerability, immunogenicity and clinical activity of ADXS-504 in men who have undergone radical prostatectomy or radiotherapy and whose prostate specific antigen (PSA) levels in the blood are rising, which we believe are ideal patients to evaluate with this approach. Moreover, we believe our multi-neoantigen vector has the potential to drive T cell responses against both hormone-sensitive and hormone-resistant cancer cells, which could result in disease control and may delay the start of androgen blockade therapy and its associated long-term complications. This study will also examine our lowest dose of an Lm-based immunotherapy to date, which could enhance the risk/benefit ratio that we have previously observed in our ADXS-HOT program.”
The Phase 1 open-label dose escalation study will evaluate the safety and tolerability of two dose levels (1e7 and 1e8 CFU) of ADXS-504 monotherapy, administered via infusion every four weeks for six total doses in 9-18 patients with biochemically recurrent prostate cancer (i.e., those with elevation of prostate-specific antigen (PSA) in the blood after radical prostatectomy or radical radiotherapy (external beam or brachytherapy) and who are not currently receiving androgen ablation therapy). The study will also evaluate preliminary clinical and immune responses following treatment with ADXS-504 monotherapy.
ADXS-504 is a novel Lm-based immunotherapy, bioengineered to elicit T cell responses against 24 tumor antigens that include 14 peptide antigens derived from frequently occurring and commonly shared hotspot mutations in patients with prostate cancer and 10 peptide antigens derived from sequence-optimized tumor-associated antigens (TAAs) that are differentially expressed or overexpressed in prostate cancer. ADXS-504 is designed to express multiple tumor antigen targets to which patients may generate a broad set of effector T cells for tumor control. Similar to Advaxis’s other Lm-based immunotherapies, ADXS-504 is expected to induce an innate immune response followed by the adaptive response and modification of the immunosuppressive tumor microenvironment (TME) by reducing regulatory T cells (Tregs) and myeloid-derived suppressor cell (MDSC) frequencies in the TME.
Advaxis initiates early-stage ADXS-504 prostate cancer study
Jul. 15, 2021 8:21 AM ET
By: Aakash Babu, SA News Editor
- Advaxis (NASDAQ:ADXS) announces the initiation of its Phase 1 study evaluating ADXS-504 in patients with biochemically recurrent prostate cancer.
- https://seekingalpha.com/news/3715455-advaxis-initiates-early-stage-adxs-504-prostate-cancer-study
- https://seekingalpha.com/symbol/ADXS
- https://www.advaxis.com/
- https://www.advaxis.com/psa-associated-cancers
Vicineum™
Belapectin in Combination with KEYTRUDA®
Bria-IMT™ (SV-BR-1-GM)
PRESS RELEASE
Sesen Bio Announces Productive Late-Cycle Meeting with the FDA for Vicineum™
July 14, 2021 at 8:00 AM EDTPDF Version
No Advisory Committee meeting is planned at this time
No confirmatory trial required at this time
Company believes it remains on track for August 18th target PDUFA date
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jul. 14, 2021-- Sesen Bio (Nasdaq: SESN), a late-stage clinical company developing targeted fusion protein therapeutics for the treatment of patients with cancer, today announced that on July 13, 2021, the Company participated in a productive Late-Cycle Meeting with the U.S. Food and Drug Administration (FDA) regarding the Company’s Biologics License Application (BLA) for Vicineum for the treatment of BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) currently under Priority Review with a target Prescription Drug User Fee Act (PDUFA) date of August 18, 2021.
The Late-Cycle Meeting is held late in the BLA review process between members of the FDA review team and the applicant to discuss the status of the review. The purpose of the meeting is to share information, discuss any substantive review items identified to date and to discuss the objectives for the remainder of the review. The meeting does not address the final regulatory decision for the application.
“We are very pleased with the outcome of the Late-Cycle Meeting and continue to feel encouraged by the level of engagement from the FDA in our ongoing discussions regarding the BLA for Vicineum,” said Dr. Thomas Cannell, president and chief executive officer of Sesen Bio. “We understand the FDA’s position on the remaining review items and anticipate a successful resolution of these matters prior to the target PDUFA date. We remain focused on the patient and bringing a differentiated product to market that has the potential to improve patient outcomes while reducing overall healthcare costs.”
About Vicineum™
Vicineum, a locally administered fusion protein, is Sesen Bio’s lead product candidate being developed for the treatment of BCG-unresponsive non-muscle invasive bladder cancer (NMIBC). Vicineum is comprised of a recombinant fusion protein that targets epithelial cell adhesion molecule (EpCAM) antigens on the surface of tumor cells to deliver a potent protein payload, Pseudomonas Exotoxin A. Vicineum is constructed with a stable, genetically engineered peptide tether to ensure the payload remains attached to the antibody binding fragment until it is internalized by the cancer cell. This fusion protein design is believed to decrease the risk of toxicity to healthy tissues, thereby improving its safety. In prior clinical trials conducted by Sesen Bio, EpCAM has been shown to be overexpressed in NMIBC cells with minimal to no EpCAM expression observed on normal bladder cells. Sesen Bio is currently in the follow-up stage of a Phase 3 registration trial in the US for the treatment of BCG-unresponsive NMIBC. In February 2021, the FDA accepted for filing the Company’s BLA for Vicineum for the treatment of BCG-unresponsive NMIBC and granted the application Priority Review with a target PDUFA date of August 18, 2021. Additionally, Sesen Bio believes that cancer cell-killing properties of Vicineum promote an anti-tumor immune response that may potentially combine well with immuno-oncology drugs, such as checkpoint inhibitors. For this reason, the activity of Vicineum in BCG-unresponsive NMIBC is also being explored at the US National Cancer Institute in combination with AstraZeneca’s immune checkpoint inhibitor durvalumab.
For more information, please visit the Company’s website at www.sesenbio.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210714005347/en/
Source: Sesen Bio
Sesen Bio (SESN) stock perks up 4% after announcing late-cycle meeting with FDA for Vicineum
Jul. 14, 2021 8:38 AM ETSesen Bio, Inc. (SESN)By: Mamta Mayani, SA News Editor1 Comment
- Sesen Bio (NASDAQ:SESN) pops 3.7% premarket after announcing that it participated in a Late-Cycle Meeting with the FDA regarding its Biologics License Application (BLA) for Vicineum for the treatment of BCG-unresponsive non-muscle invasive bladder cancer.
- https://seekingalpha.com/news/3715074-sesen-bio-perks-up-4-after-announcing-late-cycle-meeting-with-fda-for-vicineum
- https://seekingalpha.com/symbol/SESN
- https://sesenbio.com/our-programs/#head-and-neck-cancer
Bria-IMT™ (SV-BR-1-GM)
Belapectin in Combination with KEYTRUDA®
Bria-IMT™ (SV-BR-1-GM)
BriaCell Phase I/IIa Clinical Trial Combination Study in Advanced Breast Cancer Patients Open for Enrollment
July 14, 2021
NEW YORK and VANCOUVER, British Columbia, July 14, 2021 (GLOBE NEWSWIRE) -- BriaCell Therapeutics Corp. (Nasdaq: BCTX, BCTXW) (TSX-V:BCT) ("BriaCell" or the "Company"), a clinical-stage biotechnology company specializing in targeted immunotherapies for advanced breast cancer and other cancers, today announces the open recruitment and enrollment of their collaborative clinical study with Incyte. The Phase I/IIa combination study is designed to evaluate BriaCell's lead candidate, Bria-IMT™, with Incyte's checkpoint inhibitor, retifanlimab, and IDO1 inhibitor, epacadostat, for the treatment of advanced breast cancer.
The BriaCell and Incyte clinical program is a non-exclusive clinical trial collaboration to evaluate the effects of combinations of novel clinical candidates. Under the agreement, Incyte is providing compounds from its development portfolio, including retifanlimab, an anti-PD-1 monoclonal antibody, and epacadostat, an IDO1 inhibitor, for use in combination studies with Bria-IMT™, in advanced breast cancer patients.
BriaCell and Incyte had previously treated two patients under this Phase I/IIa combination study subsequent to the corporate collaboration commencement in April 2019. Some of those patients were included in BriaCell's recent survival data announcement. With funding now secured, BriaCell has once again opened this collaborative study to enrollment.
More information is available at https://briacell.com/.
BriaCell/Incyte opens enrollment for Phase I/IIa combo study in breast cancer
Jul. 14, 2021 7:30 AM ET
By: Mamta Mayani, SA News Editor
- BriaCell Therapeutics (NASDAQ:BCTX) announces the open recruitment and enrollment of their collaborative clinical study with Incyte (NASDAQ:INCY).
- https://seekingalpha.com/news/3715023-briacellincyte-opens-enrollment-for-phase-iiia-combo-study-in-breast-cancer
- https://seekingalpha.com/symbol/INCY
- https://seekingalpha.com/symbol/BCTX
- https://briacell.com/novel-technology/briaimt/
- https://briacell.com/
Belapectin in Combination with KEYTRUDA®
Belapectin in Combination with KEYTRUDA®
Belapectin in Combination with KEYTRUDA®
Galectin Therapeutics Announces Positive Top-Line Results from a Phase 1b Clinical Trial Extension of Belapectin in Combination with KEYTRUDA® in Advanced Metastatic Melanoma and Head and Neck Cancer
- Belapectin and KEYTRUDA® combination immunotherapy in patients with treatment-refractory and progressive diseases shows a cancer control rate of 56% in melanoma and of 40% in head and neck cancer
- Melanoma patients in the study had a particularly severe prognosis, with four out of nine having choroidal primary tumors and six out of nine having liver metastasis
- No toxicities deemed related, probably related, or possibly related to Belapectin were reported
- Similar to the previously announced phase I study, the frequency and severity of toxicities observed with the combination were less than the anticipated toxicity with KEYTRUDA alone
- Portland’s Earle A. Chiles Research Institute team, a division of Providence, led by principal investigator Dr. Brendan Curti, M.D., is further encouraged by the results that strengthen the rationale to conduct a larger, randomized controlled Phase 2 study
NORCROSS, Ga., July 09, 2021 (GLOBE NEWSWIRE) -- Galectin Therapeutics Inc. (NASDAQ:GALT), the leading developer of therapeutics that target galectin proteins, and the Earle A. Chiles Research Institute, a division of the Providence Cancer Institute, today announced top-line clinical data from the extension cohort of an investigator-initiated Phase 1b clinical trial of Belapectin, a galectin-3 inhibitor, in combination with KEYTRUDA® (pembrolizumab) in patients with metastatic melanoma and head and neck cancer1. The study is conducted under the direction of Dr. Brendan D. Curti, M.D., a renowned cancer and melanoma expert2.
The extension study enrolled nine melanoma patients and five head and neck squamous cell carcinoma cancer patients. Compared to the initial phase 1b patients, reported earlier, the cohort in this extension study was heavily pretreated with systemic therapy, including chemotherapy, immunotherapy with checkpoint inhibitors and cytokines, melanoma mutation-directed therapies (BRAF inhibitors and MEK inhibitors), as well as surgery and radiation therapies (external and radio-labeled). Patients also had a high burden of metastasis, with the lungs, soft tissues, and the liver being the most frequently involved organs. Four of the nine melanoma patients had a choroidal (ocular) tumor as a primary site of their cancer and had also developed liver metastasis.
The treatment consisted of Belapectin 4 mg/Kg of lean body mass administered every three weeks by infusion, after the infusion of pembrolizumab. Pembrolizumab was administered according to its label. Patients’ response was evaluated at day 85, according to the Response Evaluation Criteria in Solid Tumors (RECIST) criteria. The median number of treatment cycles was four (range 3-15) for melanoma patients and five (range 4-8) for head and neck cancer patients.
The combination of Belapectin and pembrolizumab was well tolerated and appeared safe. The most frequent adverse event related to pembrolizumab, in six patients, was grade 1 (mild) pruritus (itching), a known and labeled side-effect of pembrolizumab. The second most frequent adverse event related to pembrolizumab was grade 2 fatigue in three patients. All other adverse events were mild (grade 1). There were no grade 3 or above adverse events. Similar to the initial phase 1 study results, the frequency and severity of toxicities related to pembrolizumab, notably immune-mediated adverse events, was less than anticipated. No adverse event was deemed related to belapectin.
Additional information about the Providence clinical trial may be found at:
www.clinicaltrials.gov/ct2/show/NCT02575404
Additional information about the NASH NAVIGATE clinical study may be found at:
The NAVIGATE Study Clinical Trial in NASH Cirrhosis (navigatenash.com)
About Belapectin (GR-MD-02)
Belapectin (GR-MD-02) is a complex carbohydrate drug that targets galectin-3, a critical protein in the pathogenesis of NASH and fibrosis. Galectin-3 plays a major role in diseases that involve scarring of organs including fibrotic disorders of the liver, lung, kidney, heart and vascular system. Belapectin binds to galectin-3 and disrupts its function. Preclinical data in animals have shown that belapectin has robust treatment effects in reversing liver fibrosis and cirrhosis.
A Phase 2 study showed belapectin may prevent the development of esophageal varices in NASH cirrhosis, and these results provide the basis for the conduct of the NAVIGATE trial. The NAVIGATE trial (NAVIGATEnash.com), entitled “A Seamless Adaptive Phase 2b/3, Double-Blind, Randomized, Placebo-controlled Multicenter, International Study Evaluating the Efficacy and Safety of Belapectin (GR-MD-02) for the Prevention of Esophageal Varices in NASH Cirrhosis” began enrolling patients in June 2020 and is posted on www.clinicaltrials.gov (NCT04365868).
Galectin-3 also has a significant role in cancer, and the Company is supporting a Phase 1 study in combined immunotherapy of belapectin and KEYTRUDA® in treatment of advanced melanoma and in head and neck cancer.
Additional information is available at www.galectintherapeutics.com.
Visit providenceoregon.org/cancer to learn more.
Galectin Therapeutics and its associated logo is a registered trademark of Galectin Therapeutics Inc. Belapectin is the USAN assigned name for Galectin Therapeutics’ galectin-3 inhibitor GR-MD-02.
Galectin Therapeutics shares soar on early-stage Belapectin cancer study data
Jul. 09, 2021 8:18 AM ETGalectin Therapeutics Inc. (GALT)By: Aakash Babu, SA News Editor12 Comments
- Galectin Therapeutics (NASDAQ:GALT) shares soar more than 60% during premarket trading after the company posted positive results from the early-stage trial of Belapectin in certain cancer patients.
- https://seekingalpha.com/news/3713939-galectin-therapeutics-shares-soar-on-early-stage-belapectin-cancer-study-data
- https://seekingalpha.com/symbol/GALT
- https://seekingalpha.com/symbol/MRK
- https://galectintherapeutics.com/
- https://galectintherapeutics.com/about-belapectin/
- https://galectintherapeutics.com/pipeline-overview/
Zilebesiran (ALN-AGT)
COVIDROPS™ (Intranasal COVI-AMG Neutralizing Antibody – STI 2099)
Belapectin in Combination with KEYTRUDA®
Alnylam Initiates KARDIA-1 Phase 2 Study of Zilebesiran (ALN-AGT) in Patients with Mild-to-Moderate Hypertension
Jun 30, 2021
– KARDIA-1 will Evaluate Efficacy and Safety of Quarterly and Biannual Regimens of Zilebesiran as Monotherapy –
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jun. 30, 2021-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced initiation of KARDIA-1, a global Phase 2 study evaluating the efficacy and safety of zilebesiran (pronounced “zile-BEE-siran” and formerly known as ALN-AGT), an investigational subcutaneous RNAi therapeutic targeting liver-expressed angiotensinogen (AGT) in development for the treatment of hypertension. KARDIA-1 will evaluate zilebesiran as monotherapy across different doses administered quarterly and biannually. The Company will host an “RNAi Roundtable” webinar today at 10:00 a.m. ET to discuss the zilebesiran program.
The primary endpoint of KARDIA-1 is the change from baseline in systolic blood pressure as measured by 24-hour ambulatory blood pressure monitoring after three months of treatment. Additional endpoints will include change from baseline in blood pressure at six months, time-averaged reduction of blood pressure as a measure of tonic control, and safety. The study initiation is based on encouraging Phase 1 data, including results presented earlier this year at the 2021 Joint Meeting of the European Society of Hypertension (ESH) and the International Society of Hypertension (ISH). KARDIA-1 has been activated at clinical sites in the U.S. and will also be conducted at sites in Europe.
About KARDIA-1 Phase 2 Study
The KARDIA-1 Phase 2 trial is a randomized, double-blind (DB), placebo-controlled, dose-ranging study to evaluate the efficacy and safety of zilebesiran as monotherapy in adults with mild-to-moderate hypertension. This global, multicenter trial will enroll approximately 375 adults with untreated hypertension or who are on stable therapy with one or more anti-hypertensive medications. Any patients taking prior anti-hypertensive medications will complete at least a four-week wash-out before randomization. Study participants will be randomized to one of five treatment arms during a 12-month DB period and DB extension period: 1) 150 mg zilebesiran subcutaneously once every six months; 2) 300 mg zilebesiran subcutaneously once every six months; 3) 300 mg zilebesiran subcutaneously once every three months; 4) 600 mg zilebesiran subcutaneously once every six months; or 5) placebo. Patients who receive placebo will be randomized to one of the four initial zilebesiran dose regimens beginning at month six. The study’s primary efficacy endpoint is the change from baseline in systolic blood pressure, assessed by 24-hour ambulatory blood pressure monitoring, after three months of treatment. In addition to the evaluation of the safety of zilebesiran, key secondary and exploratory endpoints in this study include additional measures of blood pressure reduction at six months, time-adjusted change in blood pressure, and change in daytime average and night-time average blood pressure. For more information on KARDIA-1 (NCT04936035), please visit clinicaltrials.gov, email clinicaltrials@alnylam.com or call 877-256-9526 in North America and +31 20 369 7861 in Europe.
About Zilebesiran
Formerly known as ALN-AGT, zilebesiran (pronounced “zile-BEE-siran”) is an investigational, subcutaneously administered RNAi therapeutic targeting angiotensinogen (AGT) in development for the treatment of hypertension in high unmet need populations. AGT is the most upstream precursor in the Renin-Angiotensin-Aldosterone System (RAAS), a cascade which has a demonstrated role in blood pressure regulation and its inhibition has well-established anti-hypertensive effects. Zilebesiran inhibits the synthesis of AGT in the liver, potentially leading to durable reductions in AGT protein and ultimately, in the vasoconstrictor angiotensin (Ang) II. Zilebesiran utilizes Alnylam's Enhanced Stabilization Chemistry Plus (ESC+) GalNAc-conjugate technology, which enables subcutaneous dosing with increased selectivity and a wide therapeutic index. The safety and efficacy of zilebesiran have not been established or evaluated by the FDA, EMA or any other health authority.
For more information about our people, science and pipeline, please visit www.alnylam.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210630005138/en/
Source: Alnylam Pharmaceuticals, Inc.
Alnylam launches mid-stage zilebesiran study in hypertension
Jun. 30, 2021 7:24 AM ETAlnylam Pharmaceuticals, Inc. (ALNY)By: Mamta Mayani, SA News Editor
- Alnylam Pharmaceuticals (NASDAQ:ALNY) has initiated Phase 2 KARDIA-1 study evaluating the efficacy and safety of zilebesiran for the treatment of hypertension.
- https://seekingalpha.com/news/3711257-alnylam-launches-mid-stage-zilebesiran-study-in-hypertension
- https://seekingalpha.com/symbol/ALNY
- https://www.alnylam.com/alnylam-rnai-pipeline/
- https://www.alnylam.com/
COVIDROPS™ (Intranasal COVI-AMG Neutralizing Antibody – STI 2099)
COVIDROPS™ (Intranasal COVI-AMG Neutralizing Antibody – STI 2099)
COVIDROPS™ (Intranasal COVI-AMG Neutralizing Antibody – STI 2099)
Sorrento Receives Authorization From the UK Regulatory Agency to Conduct a Phase 2 Clinical Trial for COVI-DROPS in an Outpatient Setting
June 11, 2021 at 4:11 PM EDTDownload PDF
- Large Phase 2 efficacy trial cleared to start in the United Kingdom in newly diagnosed SARS-CoV-2 infected patients.
- COVI-DROPS is administered by intranasal drops and the antibody is active against the original SARS-CoV-2 virus, as well as the UK/Alpha and India/Delta variants, currently prevalent in the UK and US.
SAN DIEGO, June 11, 2021 (GLOBE NEWSWIRE) -- Sorrento Therapeutics, Inc. (Nasdaq: SRNE, "Sorrento") today announced that the Medicines and Healthcare products Regulatory Agency (MHRA), the United Kingdom’s regulatory agency, has cleared Sorrento’s COVI-DROPS product candidate for a Phase 2 efficacy trial. The application was submitted as a rolling application and the MHRA cleared the study in less than a month from Sorrento’s first submission to the MHRA. The application was supported by the safety data from a healthy subject study completed in the US, which showed a safety profile comparable to placebo with doses up to 60 mg. In this study, there were no serious adverse effects or dose limiting toxicities and all adverse effects were mild in severity. The maximum tolerated dose was not reached.
The Phase 2 efficacy trial is a large double-blind clinical trial enrolling 350 outpatients with COVID-19 who are asymptomatic or have mild symptoms in a 2:2:1 randomization with patients receiving 10mg, 20mg or placebo (details can be found on www.ClinicalTrials.gov using the identifier NCT04900428). This trial will complement the Phase 2 trial currently being started in the US and a separate trial to be started in Mexico.
COVI-DROPS is administered as an intranasal instillation in each nares to recently infected patients and utilizes the same neutralizing antibody drug substance as COVI-AMG, the intravenous formulation. The antibody is active against the original SARS-CoV-2 virus as well as the most prevalent viral variants of concern (VoCs) currently infecting the UK and the US. These variants include the UK/Alpha and the India/Delta variants of concern.
The results of this Phase 2 trial in the UK will be combined with the results of the US and Mexico Phase 2 trials and should the results of these studies demonstrate that COVI-DROPS is both safe and effective against SARS-CoV-2, Sorrento will apply for Emergency Use Authorization in the US, India, UK, Mexico and European Union as well as other territories.
For more information visit www.sorrentotherapeutics.com.
Sorrento rises on U.K. regulatory clearance of mid-stage trial for COVID-19 therapy
Jun. 11, 2021 5:13 PM ETSorrento Therapeutics, Inc. (SRNE)By: Dulan Lokuwithana, SA News Editor3 Comments
- Sorrento Therapeutics (NASDAQ:SRNE) has risen ~3.5% in extended trading after announcing that the company received regulatory clearance in the U.K. to start a Phase 2 efficacy trial for its COVI-DROPS product candidate.
- https://seekingalpha.com/news/3705814-sorrento-rises-on-uk-regulatory-clearance-of-mid-stage-trial-for-covid-19-therapy
- https://seekingalpha.com/symbol/SRNE
COVIDROPS™ (Intranasal COVI-AMG Neutralizing Antibody – STI 2099)
In addition to the intravenous formulation of COVI-AMG™ neutralizing antibody, Sorrento is developing COVIDROPS which will be the intranasal formulation of COVI-AMG™.
https://sorrentotherapeutics.com/research/covid-19/covidrops/
betibeglogene autotemcel (beti-cel)
COVIDROPS™ (Intranasal COVI-AMG Neutralizing Antibody – STI 2099)
COVIDROPS™ (Intranasal COVI-AMG Neutralizing Antibody – STI 2099)
betibeglogene autotemcel (beti-cel) One-Time Gene Therapy for β-thalassemia Continues to Demonstrate Durable Efficacy Across Pediatric and Adult Patient Populations and All Genotypes in Data Presented at EHA2021 Virtual
With 51 patients enrolled, data from the long-term follow-up study (LTF-303) show that all patients treated with beti-cel who achieve transfusion independence (TI) remain free from transfusions, with the longest follow-up of seven years
Across Phase 3 studies, 89% (32/36) of evaluable patients across ages and genotypes achieved TI and remain transfusion free, including 91% (20/22) of evaluable pediatric patients under the age of 18
The absence of drug product-related adverse events beyond two years post-infusion supports a favorable long-term safety profile of beti-cel
Data from bluebird bio’s Phase 1/2 and Phase 3 clinical studies represent more than 220 patient-years of experience with beti-cel
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jun. 11, 2021-- bluebird bio, Inc. (Nasdaq: BLUE) today presented data from several studies of betibeglogene autotemcel (beti-cel) gene therapy (licensed as ZYNTEGLO™ in the EU and UK) in adult, adolescent and pediatric patients with transfusion-dependent β-thalassemia (TDT). These data were presented during EHA2021 Virtual, the 26th Annual Congress of the European Hematology Association, taking place June 9-17, 2021.
“Our maturing clinical data continue to deliver the results we had hoped for, with patients free from transfusions and maintaining strong hemoglobin levels over the course of years,” said Richard Colvin, M.D., Ph.D., VP, interim chief medical officer, bluebird bio. “TDT is usually diagnosed in the first two years of life, and patients with this disease will require regular blood transfusions for the rest of their lives – most as often as every few weeks. It is truly transformative for these patients and their families that they no longer need ongoing blood transfusions.”
Beti-cel is a one-time gene therapy that adds functional copies of a modified form of the β-globin gene (βA-T87Q-globin gene) into a patient’s own hematopoietic (blood) stem cells (HSCs). Once patients have the βA-T87Q-globin gene, they HSCs have the potential to produce HbAT87Q, which is gene therapy-derived adult Hb, at levels that can eliminate or significantly reduce the need for transfusions.
In studies of beti-cel, transfusion independence is defined as no longer needing red blood cell transfusions for at least 12 months while maintaining a weighted average Hb of at least 9 g/dL.
Phase 3 Northstar-2 and Northstar-3 studies
As of March 9, 2021, 41 patients were treated in the Phase 3 studies HBG-207 (Northstar-2; n=23; median follow-up 24.3 months [min-max: 13.0 - 27.5]); and HGB-212 (Northstar-3; n=18; median follow-up 23 months [min-max: 4.1 – 26.8]).
Following treatment with beti-cel, 89% (32/36) of evaluable patients across ages and genotypes in both Phase 3 studies achieved transfusion independence (TI). As of the data cut-off date, these patients continue to be free of transfusions for a median duration of 25 months (min-max: 12.5 – 38.5), with median weighted average total hemoglobin levels during TI of 11.6 g/dL (min-max: 9.3 – 13.7).
Median gene therapy-derived hemoglobin (HbAT87Q) was stable approximately six months post-infusion: 8.8 g/dL at Month 6 (n=33); 9.2 g/dL at Month 9 (n=34); 8.7 g/dL at Month 12 (n=36); 9.3 g/dL at Month 18 (n=29); and 8.9 g/dL at Month 24 (n=26).
In exploratory analyses, biomarkers of ineffective erythropoiesis trended toward normal over time in patients who achieved TI, supporting the disease-modifying potential of beti-cel in patients with TDT; additionally, biomarkers of hemolysis normalized in patients who achieved TI.
The treatment regimen, comprising mobilization/apheresis, conditioning and beti-cel infusion, has a safety profile consistent with the known effects of mobilization with G-CSF and plerixafor and myeloablation with single-agent busulfan.
Grade ≥3 veno-occlusive liver disease in three patients was attributed to busulfan conditioning and resolved with defibrotide treatment. One patient developed serious, Grade 3 congestive heart failure unrelated to drug product, which was downgraded to Grade 1 at 5 months and resolved at 12 months.
Adverse events (AEs) considered related or possibly related to the drug product included thrombocytopenia (n=3), abdominal pain (n=3), leukopenia (n=1), neutropenia (n=1), pain in extremity (n=1), tachycardia (n=1) and autoimmune disorder (n=1).
Post-infusion non-hematologic Grade ≥3 AEs in ≥3 patients in either study unrelated to beti-cel included oropharyngeal inflammation (n=29), febrile neutropenia (n=20), epistaxis (n=8), decreased appetite (n=6), pyrexia (n=5), alanine aminotransferase increase (n=5) and veno-occlusive liver disease (n=3). There were no deaths, no graft failures or graft-versus-host disease (GVHD), and no cases of replication-competent lentivirus, insertional oncogenesis, clonal predominance, or malignancy.
Phase 3 pediatric patients
As of March 9, 2021, 27 pediatric patients (<12 years: n=16; ≥12 to <18 years: n=11) were treated in the Phase 3 HGB-207 (Northstar-2) and HGB-212 (Northstar-3) studies and had a median follow-up of 25.5 months (min-max: 4.1 – 41.5 months).
Following treatment with beti-cel, 91% (20/22) of evaluable patients under the age of 18 years (ages 4 to 17), including 10 patients under age 12, achieved transfusion independence (TI). These patients continue to be free of transfusions through their last follow-up, with median weighted average Hb levels during TI of 10.0 g/dL in patients under age 12 (n=10) and 11.7 g/dL in patients age 12-18 (n=10).
The median baseline score for 18 patients who achieved TI was 79.90 (range, 47.83-97.83; n=18) on the PedsQL-4.0; healthy children reach scores of approximately 84. At 24 months, improvement in quality of life was approximately three-fold higher than the minimal clinically significant meaningful difference (MCMD, a change between 4.30 and 4.83 points) as measured by the PedsQL-4.0. Improvements were more pronounced in patients with more severe scores at baseline, showing greater than five-fold higher improvements over the MCMD.
Adverse events (AEs) in pediatric patients during the HGB-207 and HGB-212 trials that were considered related or possibly related to beti-cel were non-serious and included tachycardia (Grade 1, n=1) and abdominal pain (Grade 1, n=2) on the day of infusion, and Grade 3 thrombocytopenia in one patient post-infusion.
There were no deaths, no graft failures or GVHD, and no cases of replication-competent lentivirus or insertional oncogenesis. No clonal predominance has been observed.
Grade 4 veno-occlusive liver disease occurred in two patients (ages ≥12 to <18 years) and one Grade 2 event occurred in a patient age <12 years; all events resolved after treatment with defibrotide.
Post-infusion non-hematologic Grade ≥3 AEs in ≥2 patients <18 years of age (n=27) unrelated to beti-cel included stomatitis (n=15), febrile neutropenia (n=15), epistaxis (n=6), decreased appetite (n=5), alanine aminotransferase increase (n=3), hypoxia (n=3), pyrexia (n=3), pharyngeal inflammation (n=3), mucosal inflammation (n=3), veno-occlusive disease (n=2) and dyspepsia (n=2).
The presentations are now available on demand on the EHA2021 conference website:
About betibeglogene autotemcel (beti-cel)
Betibeglogene autotemcel (beti-cel) is a one-time gene therapy that adds functional copies of a modified form of the β-globin gene (βA-T87Q-globin gene) into a patient’s own hematopoietic (blood) stem cells (HSCs). Once a patient has the βA-T87Q-globin gene, they have the potential to produce HbAT87Q, which is gene therapy-derived adult hemoglobin (Hb), at levels that may eliminate or significantly reduce the need for transfusions. In studies of beti-cel, transfusion independence (TI) is defined as no longer needing red blood cell transfusions for at least 12 months while maintaining a weighted average Hb of at least 9 g/dL.
beti-cel is manufactured using the BB305 lentiviral vector (LVV), a third-generation, self-inactivating LVV. The promoter, a regulatory element of the LVV that controls the expression of the transgene, selected for BB305 is a cellular (non-viral) promoter that drives gene expression only in the erythroid lineage cells (red blood cells and their precursors).
The European Commission granted conditional marketing authorization (CMA) for beti-cel, marketed as ZYNTEGLO™ gene therapy, for patients 12 years and older with TDT who do not have a β0/β0 genotype, for whom hematopoietic stem cell (HSC) transplantation is appropriate, but a human leukocyte antigen (HLA)-matched related HSC donor is not available. Non-serious adverse events (AEs) observed during clinical studies that were attributed to beti-cel included abdominal pain, thrombocytopenia, leukopenia, neutropenia, hot flush, dyspnea, pain in extremity, tachycardia and non-cardiac chest pain. One serious adverse event (SAE) of thrombocytopenia was considered possibly related to beti-cel.
Additional AEs observed in clinical studies were consistent with the known side effects of HSC collection and bone marrow ablation with busulfan, including SAEs of veno-occlusive disease. For details, please see the Summary of Product Characteristics (SmPC).
On April 28, 2020, the EMA renewed the CMA for beti-cel. The CMA for beti-cel is valid in the 27 member states of the EU as well as the UK, Iceland, Liechtenstein and Norway. In November 2020, bluebird bio submitted to the EMA an application for the second renewal of the CMA. This procedure is currently on hold while the EMA's Pharmacovigilance Risk Assessment Committee (PRAC) reviews the safety of ZYNTEGLO. The CMA is valid while the renewal application review is ongoing by the regulatory agency.
The FDA granted beti-cel Orphan Drug status and Breakthrough Therapy designation for the treatment of TDT.
bluebird bio is on track to complete its rolling Biologics License Application (BLA) submission to the FDA for beti-cel in mid-2021. This submission is anticipated to include adults, adolescents and children with transfusion dependent β-thalassemia across all genotypes (including non-β0/β0 genotypes and β0/β0 genotypes). Beti-cel is not approved in the U.S.
Beti-cel continues to be evaluated in the ongoing Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies. bluebird bio is conducting a long-term safety and efficacy follow-up study, LTF-303, for people who have participated in bluebird bio-sponsored clinical studies of beti-cel.
For more information, visit bluebirdbio.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210611005081/en/
Bluebird's gene therapy for β-thalassemia shows sustained and durable efficacy across pediatric and adult patients
Jun. 11, 2021 6:40 AM ETbluebird bio, Inc. (BLUE)By: Mamta Mayani, SA News Editor5 Comments
- bluebird bio (NASDAQ:BLUE) presents data from several studies of betibeglogene autotemcel (beti-cel) gene therapy in adult, adolescent and pediatric patients with transfusion-dependent β-thalassemia (TDT).
- https://seekingalpha.com/news/3705544-bluebirds-gene-therapy-for-thalassemia-shows-sustained-and-durable-efficacy-across-pediatric-and-adult-patients
- https://seekingalpha.com/symbol/BLUE
biotecHOPE™ Apr/Mar/Feb/Jan/2021
TYVYT (Sintilimab Injection) plus Fruquintinib
TYVYT (Sintilimab Injection) plus Fruquintinib
TYVYT (Sintilimab Injection) plus Fruquintinib
Innovent and HUTCHMED Release Phase 1b preliminary Results of TYVYT (Sintilimab Injection) plus Fruquintinib as a Third Line Treatment for Advanced Colorectal Cancer at ASCO Annual Meeting 2021
NEWS PROVIDED BY
Jun 06, 2021, 20:00 ET
SAN FRANCISCO and SUZHOU, China, June 6, 2021 /PRNewswire/ -- Innovent Biologics, Inc. ("Innovent", HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of cancer, metabolic, autoimmune and other major diseases, announced with HUTCHMED (Nasdaq/AIM: HCM) that the results of the Phase 1b study in advanced colorectal cancer (CRC) patients were released today in an poster discussion at the 2021 American Association for Clinical Oncology (ASCO) Annual Meeting.
This is a Phase 1b dose escalation and dose expansion study to evaluate the tolerability, safety and preliminary efficacy of sintilimab plus fruquintinib, and to determine the recommended phase 2 dose (RP2D). 44 CRC patients that had failed at least 2 previous lines of therapy containing fluoropyrimidine, oxaliplatin or irinotecan were enrolled. They received either 5mg-intermittent or 3mg-continous dosage (n=22, each). The objective response rate (ORR) was 22.7% (10/44, 95% CI: 11.5-37.8%) with 27.3% (6/22, 95% CI: 10.7-50.2%) in the 5mg-intermittent group and 18.2% (4/22, 95% CI: 5.2-40.3%) in the 3mg-continuous group. With a median follow-up time of 11.3 (range: 9.8-11.7) months, the Kaplan-Meier estimated median progression free survival (PFS) was 5.6 (95% CI:4.3-7.5) months among all 44 patients, with 6.9 (95% CI:5.4-8.3) months for the 5mg-intermittent group and 4.2 (95% CI:2.9-9.5) months for the 3mg-continuous group. The safety for this combination therapy was controllable.
About TYVYT® (Sintilimab Injection)
TYVYT® (sintilimab injection) is an innovative PD-1 inhibitor with global quality standards jointly developed by Innovent and Lilly. Sintilimab is a type of immunoglobulin G4 monoclonal antibody, which binds to PD-1 molecules on the surface of T-cells, blocks the PD-1 / PD-Ligand 1 (PD-L1) pathway, and reactivates T-cells to kill cancer cells. Innovent is currently conducting more than 20 clinical studies of sintilimab to evaluate its safety and efficacy in a wide variety of cancer indications, including more than 10 registrational or pivotal clinical trials.
In China, TYVYT® (sintilimab Injection) has been approved for two indications, including:
- The treatment of relapsed or refractory classic Hodgkin's lymphoma after two lines or later of systemic chemotherapy
- In combination with pemetrexed and platinum chemotherapy, for the first-line treatment of nonsquamous non-small cell lung cancer
- In combination with gemcitabine and platinum chemotherapy, for the first-line treatment of squamous non-small cell lung cancer
Additionally, Innovent currently has regulatory submissions under review in China for TYVYT® (sintilimab Injection):
- In combination with BYVASDA® (bevacizumab injection) for the first-line treatment of hepatocellular carcinoma
- The second-line treatment of squamous non-small cell lung cancer
In May 2021, the U.S. FDA accepted for review the Biologics License Application (BLA) for sintilimab in combination with pemetrexed and platinum chemotherapy for the first-line treatment of nonsquamous non-small cell lung cancer.
TYVYT® (sintilimab Injection) was included in China's National Reimbursement Drug List (NRDL) in 2019 as the first PD-1 inhibitor and the only PD-1 included in the list in that year..
About ELUNATE® (fruquintinib)
Fruquintinib is a highly selective and potent oral inhibitor of VEGFRs –1, –2 and –3. VEGFR inhibitors play a pivotal role in blocking tumor angiogenesis. Fruquintinib was designed to improve kinase selectivity to minimize off-target toxicities, improve tolerability and provide more consistent target coverage. The generally good tolerability in patients to date, along with fruquintinib's low potential for drug-drug interaction based on preclinical assessment, suggests that it may also be highly suitable for combinations with other anti-cancer therapies.
Fruquintinib was approved for marketing by the NMPA in September 2018 and is marketed under the brand name ELUNATE® in China. ELUNATE® is for the treatment of patients with metastatic CRC that have been previously treated with fluoropyrimidine, oxaliplatin and irinotecan, including those who have previously received anti-VEGF therapy and/or anti-EGFR therapy (RAS wild type).
HUTCHMED retains all rights to fruquintinib outside of China. In China, HUTCHMED is partnered with Eli Lilly and Company and is responsible for development and execution of all on-the-ground medical detailing, promotion and local and regional marketing.
For more information, please visit: www.innoventbio.com.
For more information, please visit: www.hutch-med.com
Innovent Bio, Hutchmed's Tyvyt + Fruquintinib show promising action in colorectal cancer study
Jun. 07, 2021 12:08 AM ETHUTCHMED (China) Limited (HCM), IVBXFBy: Mamta Mayani, SA News Editor
- Innovent Biologics (OTCPK:IVBIY) with Hutchmed (NASDAQ:HCM) present Phase 1b study results in advanced colorectal cancer (CRC) patients at the 2021 American Association for Clinical Oncology (Ahttps://seekingalpha.com/news/3703530-innovent-bio-hutchmeds-tyvyt-fruquintinib-show-promising-action-in-colorectal-cancer-studySCO) Annual Meeting.
- https://seekingalpha.com/symbol/IVBIY
- https://seekingalpha.com/symbol/HCM
- https://www.hutch-med.com/pipeline-and-products/our-pipeline/
ZYNLONTA™ (loncastuximab tesirine-lpyl)
TYVYT (Sintilimab Injection) plus Fruquintinib
TYVYT (Sintilimab Injection) plus Fruquintinib
ADC Therapeutics Presents Updated Clinical Data at 2021 ASCO Annual Meeting
JUNE, 04, 2021
ZYNLONTA™ LOTIS-2 updated data includes median duration of response of 13.4 months in heavily pre-treated patients with relapsed or refractory DLBCL
Subgroup analyses include patients with double- / triple-hit, advanced stage or transformed DLBCL, DLBCL refractory to first-line therapy, and patients older than 65
LAUSANNE, Switzerland--(BUSINESS WIRE)--
ADC Therapeutics SA (NYSE: ADCT), a commercial-stage biotechnology company leading the development of novel antibody drug conjugates (ADCs) to treat hematological malignancies and solid tumors, today announced updated clinical data from the ZYNLONTA™ (loncastuximab tesirine-lpyl) Phase 2 LOTIS-2 trial in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) were presented at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting, which is being held virtually June 4-8, 2021.
“The maturing duration of response from the ZYNLONTA Phase 2 trial reported at ASCO reflects the strong data set that served as the basis of the accelerated FDA approval in April,” said Jay Feingold, MD, PhD, Senior Vice President and Chief Medical Officer of ADC Therapeutics. “We are especially encouraged to see this positive trend continue to strengthen in a heavily pre-treated patient population, including patients with double- / triple-hit, advanced stage or transformed DLBCL, DLBCL refractory to first-line therapy, and patients older than 65.”
In LOTIS-2, a single-arm, open-label, 145-patient Phase 2 clinical trial in patients with relapsed or refractory DLBCL who had failed ≥2 established therapies, ZYNLONTA demonstrated continued substantial antitumor activity and an acceptable safety profile. Updated results, including analysis of response in high-risk subgroups, were presented in a poster (abstract number: 7546) by Paolo F. Caimi, MD, University Hospitals Cleveland Medical Center and Case Comprehensive Cancer Center, Case Western Reserve University.
About ZYNLONTA™ (loncastuximab tesirine-lpyl)
ZYNLONTA™ is a CD19-directed antibody drug conjugate (ADC). Once bound to a CD19-expressing cell, ZYNLONTA is internalized by the cell, where enzymes release a pyrrolobenzodiazepine (PBD) payload. The potent payload binds to DNA minor groove with little distortion, remaining less visible to DNA repair mechanisms. This ultimately results in cell cycle arrest and tumor cell death. The U.S. Food and Drug Administration (FDA) has approved ZYNLONTA (loncastuximab tesirine-lpyl) for the treatment of adult patients with relapsed or refractory (r/r) large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (NOS), DLBCL arising from low-grade lymphoma and also high-grade B-cell lymphoma. This indication is approved by the FDA under accelerated approval based on overall response rate and continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
The FDA approval was based on data from LOTIS-2, a large (n=145) Phase 2 multinational, single-arm clinical trial of ZYNLONTA for the treatment of adult patients with r/r DLBCL following two or more prior lines of systemic therapy. The trial included a broad spectrum of heavily pre-treated patients (median three prior lines of therapy) with very difficult to treat disease, including patients with high-grade B-cell lymphoma. The trial also enrolled patients who did not respond to first-line therapy, patients refractory to all prior lines of therapy, and patients who had stem cell transplants and CAR-T therapy prior to their treatment with ZYNLONTA. Results from the trial demonstrated an overall response rate (ORR) of 48.3% (70/145 patients), which included a complete response (CR) rate of 24.1% (35/145 patients) and a partial response (PR) rate of 24.1% (35/145 patients). Patients had a median time to response of 1.3 months. At the most recent data cut-off for patients enrolled in the trial, the median duration of response (mDoR) was 13.4 months. In a pooled safety population the most common adverse reactions (≥20%) were thrombocytopenia, gamma-glutamyltransferase increased, neutropenia, anemia, hyperglycemia, transaminase elevation, fatigue, hypoalbuminemia, rash, edema, nausea and musculoskeletal pain. In LOTIS-2, the most common (≥10%) grade ≥3 treatment-emergent adverse events were neutropenia (26.2%), thrombocytopenia (17.9%), gamma-glutamyltransferase increased (17.2%) and anemia (10.3%).
ZYNLONTA is being evaluated in combination for earlier lines of therapy and as a monotherapy in other B-cell malignancies.
ZYNLONTA™ is a trademark of ADC Therapeutics SA.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210604005572/en/
ADC Therapeutics Zynlonta shows strong duration of response in B-cell lymphoma
Jun. 05, 2021 10:12 AM ET
By: Jonathan M Block, SA News Editor
- A phase 2 trial in relapsed or refractory diffuse large B-cell lymphoma ("DLBCL") of ADC Therapeutics' (NYSE:ADCT) Zynlonta (loncastuximab tesirine-lpyl) a median duration of response of 13.4 months.
- https://seekingalpha.com/news/3703510-adc-therapeutics-zynlonta-shows-strong-duration-of-tresponse-in-b-cell-lyhttps://seekingalpha.com/symbol/ADCTmphoma
- https://www.adctherapeutics.com/
Camidanlumab Tesirine
TYVYT (Sintilimab Injection) plus Fruquintinib
valoctocogene roxaparvovec
ADC Therapeutics Announces Online Publication of Camidanlumab Tesirine Phase 1 Results in The Lancet Haematology
MAY, 26, 2021
Camidanlumab tesirine (Cami) demonstrated anti-tumoral activity response rate in 86% of Hodgkin lymphoma patients at dose currently being evaluated in ongoing pivotal Phase 2 trial
LAUSANNE, Switzerland--(BUSINESS WIRE)-- ADC Therapeutics SA (NYSE:ADCT), a commercial-stage biotechnology company leading the development of novel antibody drug conjugates (ADCs) to treat hematological malignancies and solid tumors, today announced that results of the Phase 1 clinical trial of camidanlumab tesirine (Cami), an anti-CD25 ADC, in patients with relapsed or refractory Hodgkin and non-Hodgkin lymphomas have been published online in The Lancet Haematology.
“There is a significant unmet medical need for novel therapies that improve outcomes in patients with relapsed or refractory Hodgkin lymphoma,” said Mehdi Hamadani, MD, Professor of Internal Medicine at the Medical College of Wisconsin, Division of Hematology & Oncology and lead author of The Lancet Haematology paper. “This patient population is often heavily pretreated, as was the case in this published study in which patients experienced a median of five previous systemic therapies. The Phase 1 study demonstrates encouraging potential for Cami to provide a new treatment option for patients with relapsed or refractory Hodgkin lymphoma.”
Key results include:
- In the total patient population, the overall response rate (ORR) was 58%, with 38 (29%) of 130 patients reported to have a complete response.
- In heavily pretreated patients with Hodgkin lymphoma, across all doses, the ORR was 71%, with 32 (42%) of 77 patients reported to have a complete response.
- In Hodgkin lymphoma patients who received 45 μg/kg (the recommended Phase 2 starting dose), the ORR was 86%, with 18 (49%) of 37 patients reported to have a complete response.
- In Hodgkin lymphoma patients who received 30 μg/kg, the ORR was 55%, with seven (35%) of 20 patients reported to have a complete response.
- In patients with non-Hodgkin lymphoma, the ORR was 38%, with five (9%) of 53 patients reported to have a complete response.
- The overall median duration of response was 6.6 months for all patients with Hodgkin lymphoma and 7.2 months for Hodgkin lymphoma patients who received 45 μg/kg.
- Cami demonstrated an acceptable safety profile. The most commonly observed adverse events included elevated liver enzymes (without hepatic synthetic dysfunction), rash, fatigue, oedema or effusion, and nausea.
Based on the data from this Phase 1 clinical trial, a Phase 2 trial to further evaluate the safety and efficacy of Cami in patients with relapsed or refractory Hodgkin lymphoma is ongoing. Interim data from the pivotal Phase 2 trial presented at the 2020 American Society of Hematology meeting demonstrated encouraging antitumor activity as a single agent with an ORR of 83%, a complete response rate of 38% and no new safety signals. These data highlight the potential for Cami to address an unmet need in heavily pretreated patients (median of seven prior lines of systemic therapy).
About Camidanlumab Tesirine (Cami)
Camidanlumab tesirine (Cami, formerly ADCT-301) is an antibody drug conjugate (ADC) comprised of a monoclonal antibody that binds to CD25 (HuMax®-TAC, licensed from Genmab A/S), conjugated to the pyrrolobenzodiazepine (PBD) dimer payload, tesirine. Once bound to a CD25-expressing cell, Cami is internalized into the cell where enzymes release the PBD-based payload, killing the cell. This applies to CD25-expressing tumor cells and also to CD25-expressing Tregs. The intra-tumoral release of its PBD payload may also cause bystander killing of neighboring tumor cells, and PBDs have also been shown to induce immunogenic cell death. All of these properties of Cami may enhance immune-mediated anti-tumor activity.
Cami is being evaluated in a pivotal Phase 2 clinical trial in patients with relapsed or refractory Hodgkin lymphoma and a Phase 1b clinical trial as monotherapy and in combination with pembrolizumab in solid tumors.
ADC Therapeutics is based in Lausanne (Biopôle), Switzerland and has operations in London, the San Francisco Bay Area and New Jersey. For more information, please visit https://adctherapeutics.com/ and follow the Company on Twitter and LinkedIn.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210526005542/en/
ADC Therapeutics' camidanlumab tesirine shows anti-tumoral activity in Hodgkin lymphoma
May 26, 2021 7:46 AM ET ADC Therapeutics SA (ADCT)ADC Therapeutics SA (ADCT)
By: Mamta Mayani, SA News Editor
- Results of ADC Therapeutics' (NYSE:ADCT) Phase 1 clinical trial of camidanlumab tesirine (Cami), an anti-CD25 ADC, in patients with relapsed or refractory Hodgkin and non-Hodgkin lymphomas have been published in The Lancet Haematology.
- https://seekingalpha.com/news/3700230-adc-therapeutics-camidanlumab-tesirine-shows-anti-tumoral-activity-in-hodgkin-lymphoma
- https://seekingalpha.com/symbol/ADCT
Camidanlumab tesirine is ADCT’s second lead candidate. It has demonstrated significant clinical activity in heavily pretreated patients with Hodgkin lymphoma. Based on its mechanism targeting CD25/regulatory T cells, camidanlumab tesirine is also demonstrating potential in the treatment of solid tumors.
valoctocogene roxaparvovec
valoctocogene roxaparvovec
valoctocogene roxaparvovec
BioMarin Provides Highlights of 5 Years of Clinical Data from Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec with the Longest Duration of Clinical Experience for a Gene Therapy in Hemophilia A
All Study Participants in 6e13 vg/kg and 4e13 vg/kg Dose Cohorts Remain off Factor VIII Prophylactic Therapy95% Reductions in Mean Annualized Bleed Rate (ABR) and 96% Reduction in Mean Annualized Factor VIII Usage Through Year 5 in 6e13 vg/kg Dose Cohort92% Reductions in Mean ABR and 95% Reduction in Mean Annualized Factor VIII Usage Through Year 4 in 4e13 vg/kg Dose CohortMAA Submission to EMA on Track for June 2021Data to be Shared in Oral Presentation at Upcoming International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress (July 17-21)May 19, 2021
SAN RAFAEL, Calif., May 19, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today an update to its previously reported results from an open-label Phase 1/2 study of valoctocogene roxaparvovec, an investigational gene therapy treatment for adults with severe hemophilia A. The Company plans to share the data during an oral presentation at the upcoming International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress (July 17-21).i
Five-year and four-year post-treatment follow-up of the 6e13 vg/kg and 4e13 vg/kg cohorts, respectively, shows a sustained treatment benefit of valoctocogene roxaparvovec. All participants in both cohorts remain off prophylactic Factor VIII treatment. Mean cumulative annualized bleed rates (ABR) remain less than one in the 6e13 vg/kg cohort and substantially below pre-treatment baseline levels; the mean ABR in year five for the 6e13 vg/kg cohort was 0.7 with an ABR reduction of 95% and Factor VIII use reduction of 96% through five years, compared to pre-infusion. The mean ABR in year four for the 4e13 vg/kg cohort was 1.7 with a mean cumulative ABR reduction of 92% and Factor VIII use reduction of 95% through four years, compared to pre-infusion. Factor VIII activity levels declined commensurate with the most recent years' observations and continue to remain in a range to provide hemostatic efficacy.
Robust Clinical Program
BioMarin has multiple clinical studies underway in its comprehensive gene therapy program for the treatment of hemophilia A. In addition to the global Phase 3 study GENEr8-1 and the ongoing Phase 1/2 dose escalation study, the Company is actively enrolling participants in a Phase 3b, single arm, open-label study to evaluate the efficacy and safety of valoctocogene roxaparvovec at a dose of 6e13 vg/kg with prophylactic corticosteroids in people with severe hemophilia A. The Company is running a Phase 1/2 Study with the 6e13kg/vg dose of valoctocogene roxaparvovec in approximately 10 participants with pre-existing AAV5 antibodies, as well as another Phase 1/2 Study with the 6e13 vg/kg dose of valoctocogene roxaparvovec in people with hemophilia A with active or prior FVIII inhibitors.
For additional information, please visit www.biomarin.com
BioMarin reports long-term data from mid-stage gene therapy study in hemophilia A
May 19, 2021 8:53 AM ET
BioMarin Pharmaceutical Inc. (BMRN)
By: Mamta Mayani, SA News Editor
- BioMarin Pharmaceutical (NASDAQ:BMRN) announces an update to its Phase 1/2 study of valoctocogene roxaparvovec, an investigational gene therapy treatment for adults with severe hemophilia A, reported previously.
- https://seekingalpha.com/news/3698183-biomarin-reports-five-year-data-from-mid-stage-gene-therapy-study-in-hemophilia-a
- https://seekingalpha.com/symbol/BMRN
- https://www.biomarin.com/our-treatments/pipeline/valoctocogene-roxaparvovec-for-severe-hemophilia-a/
Sotatercept
valoctocogene roxaparvovec
Ziltivekimab
ACCELERON PRESENTS PRELIMINARY DATA FROM THE SPECTRA PHASE 2 TRIAL OF SOTATERCEPT IN PULMONARY ARTERIAL HYPERTENSION (PAH) AT THE AMERICAN THORACIC SOCIETY 2021 INTERNATIONAL CONFERENCE
MAY 19, 2021
– Treatment with sotatercept in the ongoing SPECTRA Phase 2 trial was associated with improvements in resting and exercise hemodynamics at week 24 –
– Sotatercept was generally well tolerated, consistent with the previously reported safety profile in PAH and in other diseases –
- Company-hosted investor and analyst conference call and webcast with guest PAH key opinion leader to be held today, Wednesday, May 19th at 10:30 a.m. EDT –
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Acceleron Pharma Inc. (Nasdaq: XLRN), a leading biopharmaceutical company in the discovery, development, and commercialization of TGF-beta superfamily therapeutics to treat serious and rare diseases, today presented at the American Thoracic Society 2021 International Conference (ATS 2021) preliminary interim data from the SPECTRA Phase 2 trial of sotatercept in patients with pulmonary arterial hypertension (PAH).
The findings, presented during the session “Clinical Advances in Pulmonary Hypertension: Lessons from Best Abstracts,” included outcomes obtained from the first 10 patients evaluated among a total of 21 trial participants. These preliminary data from the ongoing trial, which is designed to assess resting and exercise hemodynamics and peak oxygen uptake—as recorded by invasive cardiopulmonary exercise testing (iCPET)—show that patients treated with sotatercept experienced improvements in multiple key hemodynamic measures.
“Despite the relatively small number of patients evaluated to date, the consistency and scale of improvements seen in a range of clinically meaningful measures are very encouraging,” said Aaron Waxman, M.D., Ph.D.*, Director, Pulmonary Vascular Disease Program at Boston’s Brigham and Women’s Hospital, who presented at ATS 2021. “Our analyses are ongoing, but observing such beneficial changes among heavily pretreated patients with fairly advanced disease suggests that sotatercept may be affecting the underlying pathology of PAH.”
Sotatercept is an investigational therapy that is not approved for any use in any country.
Dr. Waxman’s detailed presentation is available on the “Publications” page under the “Science & Pipeline” section of Acceleron’s website, www.acceleronpharma.com.
*Dr. Waxman is the principal investigator of the SPECTRA trial and a paid consultant to Acceleron.
About the SPECTRA Trial
The SPECTRA Phase 2 trial is a single arm, open-label, multi-center exploratory study to determine the effects of sotatercept plus standard of care in adults with WHO functional class III PAH. The primary endpoint of the trial is the change from baseline in peak oxygen uptake (VO2 max) at 24 weeks, as recorded by invasive cardiopulmonary exercise testing (iCPET). Secondary hemodynamic endpoints as well as endpoints of exercise capacity and tolerance assessed via iCPET and right heart catheterization include change from baseline at 24 weeks in: ventilatory efficiency (VE/VCO2 slope); cardiac index (L/min/m2); mean pulmonary artery pressure (mPAP); pulmonary vascular resistance (PVR); arteriovenous oxygen content difference (Ca-vO2); ventilatory efficiency; “dead space” assessment (VE/VCO2 slope); and oxygen consumption at anaerobic threshold (VO2 at AT).
A total of 21 patients are to receive stable background combination PAH therapy plus sotatercept at a starting dose level of 0.3 mg/kg delivered subcutaneously for one cycle, escalating to 0.7 mg/kg at cycle 2 for the remainder of the treatment period. Following the 6-month open-label treatment period, participants in the trial are eligible to continue in the 18-month extension period, which includes iCPET conducted at 48 weeks.
About Sotatercept
Sotatercept is an investigational reverse-remodeling agent designed to be a selective ligand trap for members of the TGF-beta superfamily to rebalance signaling in the BMP pathway, which is a key molecular driver of PAH. In preclinical studies, sotatercept was shown to reverse the vascular remodeling that is a hallmark of PAH. The PULSAR Phase 2 trial evaluating sotatercept in combination with approved PAH-specific medicines in patients with PAH achieved its primary endpoint of improvement in pulmonary vascular resistance and its key secondary endpoint of improvement in 6-minute walk distance. Sotatercept was generally well tolerated in the trial. Adverse events observed in the study were generally consistent with previously published data on sotatercept in other diseases. Following the PULSAR results, which were published in a recent edition of the New England Journal of Medicine, sotatercept was granted Breakthrough Therapy designation from the FDA and Priority Medicines designation from the EMA in PAH. Sotatercept is also being evaluated in the SPECTRA Phase 2 exploratory trial.
The Company recently presented details of its Phase 3 development plan, including the design for the registrational STELLAR trial, which is currently enrolling patients with PAH. Acceleron is planning two additional Phase 3 studies in patients with PAH: the HYPERION trial in newly diagnosed patients and the ZENITH trial assessing intervention in patients diagnosed with World Health Organization (WHO) functional class IV disease.
Sotatercept is an investigational therapy that is not approved for any use in any country. Sotatercept is part of a licensing agreement with Bristol Myers Squibb.
For more information, please visit www.acceleronpharma.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210519005172/en/
Source: Acceleron Pharma Inc.
Acceleron's sotatercept shows potential to treat pulmonary arterial hypertension in mid-stage Pulsar trial
May 19, 2021 8:36 AM ET
By: Mamta Mayani, SA News Editor
- Acceleron Pharma (NASDAQ:XLRN) announces interim results from the open-label extension of the PULSAR Phase 2 trial of sotatercept in patients with pulmonary arterial hypertension (https://seekingalpha.com/news/3698172-accelerons-sotatercept-shows-potential-to-treat-pulmonary-arterial-hypertension-in-mid-stage-pulsar-trialPAH).
- https://seekingalpha.com/symbol/XLRN
- https://acceleronpharma.com/
- https://seekingalpha.com/symbol/BMY
Ziltivekimab
valoctocogene roxaparvovec
Ziltivekimab
Ziltivekimab reduced inflammatory biomarkers associated with atherosclerosis in people with chronic kidney disease in phase 2 trial
12:30 17 May 2021
PLAINSBORO, N.J., May 17, 2021 /PRNewswire/ -- Novo Nordisk today presented results from RESCUE, a phase 2 randomised, double-blind, placebo controlled clinical trial assessing the effect of once-monthly, investigational ziltivekimab, an interleukin-6 (IL-6) inhibitor, on biomarkers of inflammation. The trial showed a significant reduction of multiple inflammatory biomarkers associated with atherosclerosis in people with advanced chronic kidney disease (CKD) and elevated high-sensitivity C-reactive protein (hsCRP), representing high cardiovascular risk. The data were announced today at the virtual American College of Cardiology's (ACC) 70th Annual Scientific Session1 and simultaneously published in The Lancet 2.
The RESCUE trial met its primary endpoint, showing that after 12 weeks, median levels of hsCRP were significantly reduced with ziltivekimab compared with placebo (77%, 88% and 92% reduction in those receiving 7.5 mg, 15 mg and 30 mg of ziltivekimab, respectively, compared to 4% for placebo). The proportion of people achieving both a greater than 50% reduction in hsCRP and hsCRP levels of less than 2 mg/L, a secondary endpoint, was also significantly higher with ziltivekimab than placebo (66%, 80% and 93% in those receiving 7.5 mg, 15 mg and 30 mg of ziltivekimab, respectively, compared to 4% for placebo). Dose-dependent reductions were also observed for four additional inflammatory biomarkers (fibrinogen, serum amyloid A, haptoglobin and secretory phospholipase A2). Treatment emergent adverse events were considered to be mild, moderate, or severe and were similar between the placebo and ziltivekimab groups. Ziltivekimab was generally well tolerated, with no unexpected side effects2.
About the RESCUE trial 9
RESCUE is a phase 2, randomised, double-blind, placebo-controlled clinical trial assessing the effect of once-monthly subcutaneous ziltivekimab on biomarkers of inflammation in people with advanced CKD and elevated hsCRP. The study, which enrolled 264 participants, was designed to assess if ziltivekimab can safely and effectively reduce levels of inflammatory biomarkers relevant to atherosclerosis. The pre-specified primary endpoint was change in hsCRP after 12 weeks of treatment, with additional data on safety and other inflammatory biomarkers (fibrinogen, serum amyloid A, haptoglobin and secretory phospholipase A2) collected over 24 weeks of treatment.
About investigational ziltivekimab
Ziltivekimab is a fully human monoclonal antibody designed to lower systemic inflammation through inhibition of IL-6 (a pro-inflammatory cytokine with a causal role in atherosclerosis). With its extended half-life technology, ziltivekimab has been designed to enable once-monthly administration by subcutaneous (SC) injection. Ziltivekimab is being developed by Novo Nordisk following the acquisition of Corvidia Therapeutics, announced in June 2020.
For more information, visit novonordisk.us
Novo Nordisk meets primary endpoint in mid-stage trial for patients with high cardiovascular risk
May 17, 2021 1:31 PM ETNovo Nordisk A/S (NVO)By: Dulan Lokuwithana, SA News Editor1 Comment
- Novo Nordisk (NVO +1.7%) announced that its monoclonal antibody ziltivekimab met the primary endpoint in a Phase 2 trial that was designed to assess its potential to reduce the levels of inflammatory biomarkers linked to atherosclerosis.
- https://seekingalpha.com/news/3697276-novo-nordisk-meets-primary-endpoint-in-mid-stage-trial-for-patients-with-high-cardiovascular-risk
- https://seekingalpha.com/symbol/NVO
The trial is registered with ClinicalTrials.gov, NCT03926117.
https://www.novonordisk-us.com/
RGX-121
VXA-NVV-104 / Phase 1 Noro GI.1/GII.4
VXA-NVV-104 / Phase 1 Noro GI.1/GII.4
REGENXBIO PRESENTS ADDITIONAL POSITIVE INTERIM DATA FROM PHASE I/II TRIAL OF RGX-121 FOR THE TREATMENT OF MPS II (HUNTER SYNDROME) AT AMERICAN SOCIETY OF GENE AND CELL THERAPY'S 24TH ANNUAL MEETING
May 14, 2021 at 7:05 AM EDT
ROCKVILLE, Md., May 14, 2021 /PRNewswire/ --
- RGX-121, a one-time gene therapy for MPS II, continues to be well-tolerated with no drug-related serious adverse events
- Biomarkers and measures of neurodevelopmental function from patients in Cohorts 1 and 2 continue to indicate CNS activity following RGX-121 administration
- Evidence of systemic enzyme expression and biomarker activity continues to be observed
- REGENXBIO recently initiated dosing of patients in Cohort 3
REGENXBIO Inc. (Nasdaq: RGNX) today announced a safety update and additional positive interim data from the ongoing Phase I/II trial of RGX-121 for the treatment of patients up to 5 years old diagnosed with Mucopolysaccharidosis Type II (MPS II), also known as Hunter Syndrome. The latest data from this trial will be presented today at the American Society of Gene and Cell Therapy (ASGCT) 24th Annual Meeting by Dr. Roberto Giugliani, Professor, Department of Genetics, UFRGS, Medical Genetics Service, HCPA, Porto Alegre, Brazil.
"I am pleased to report additional data from the Phase I/II trial of RGX-121 in patients with MPS II. The biomarker data from these patients continues to demonstrate that the I2S enzyme is active in the CNS and importantly, continued cognitive development has been observed in the majority of patients who have been followed for more than 6 months. In addition, emerging evidence of systemic enzyme expression and activity has been shown in urine and plasma, including in patients who are naive to enzyme replacement therapy, the current standard of care," said Dr. Giugliani. "The potential to provide therapeutic benefit from a one-time gene therapy would be a meaningful advancement for the treatment of MPS II patients."
RGX-121
RGX-121 is an investigational one-time gene therapy designed to deliver the gene that encodes the iduronate-2-sulfatase (I2S) enzyme using the AAV9 vector. RGX-121 is administered directly to the central nervous system (CNS). Patients in Cohorts 1 and 2 received doses of RGX-121 at 1.3x1010 genome copies per gram (GC/g) of brain mass and 6.5x1010 GC/g of brain mass, respectively. REGENXBIO began dosing patients in Cohort 3 at an increased dose of 2.0x1011 GC/g of brain mass. As of April 25, 2021, RGX-121 is reported to be well-tolerated with no drug-related serious adverse events (SAEs) in nine patients dosed with RGX-121 in Cohorts 1-3.
Data Summary and Safety Update from Cohort 1 and 2
Time of post-administration follow-up for patients in Cohorts 1 and 2 ranges from 24 weeks to two years. Five patients have completed the 48-week immunosuppression regimen, per study protocol. Six of the patients were receiving weekly, intravenous enzyme replacement therapy (ERT) at the time of enrollment; two of these patients have since discontinued ERT.
The study findings are available at https://www.asgct.org.
About RGX-121
RGX-121 is a product candidate for the treatment of Mucopolysaccharidosis Type II (MPS II), also known as Hunter Syndrome. RGX-121 is designed to use the AAV9 vector to deliver the human iduronate-2-sulfatase gene (IDS) which encodes the iduronate-2-sulfatase (I2S) enzyme to the central nervous system (CNS). Delivery of the IDS gene within cells in the CNS could provide a permanent source of secreted I2S beyond the blood-brain barrier, allowing for long-term cross correction of cells throughout the CNS. RGX-121 has received orphan drug product, rare pediatric disease and Fast Track designations from the U.S. Food and Drug Administration.
View original content to download multimedia:http://www.prnewswire.com/news-releases/regenxbio-presents-additional-positive-interim-data-from-phase-iii-trial-of-rgx-121-for-the-treatment-of-mps-ii-hunter-syndrome-at-american-society-of-gene-and-cell-therapys-24th-annual-meeting-301291376.html
SOURCE REGENXBIO Inc.
Regenxbio reports positive interim data from mid-stage RGX-121 study in Hunter Syndrome
May 14, 2021 7:38 AM ET
By: Mamta Mayani, SA News Editor
- Regenxbio (NASDAQ:RGNX) announces a safety update and additional positive interim data from Phase I/II trial of RGX-121 for the treatment of patients up to 5 years old diagnosed with Mucopolysaccharidosis Type II (MPS II), also known as Hunter Syndrome.
- https://seekingalpha.com/news/3696522-regenxbio-reports-positive-interim-data-from-mid-stage-rgx-121-study-in-hunter-syndrome
- https://seekingalpha.com/symbol/RGNX
- https://regenxbio.com/rgx-121/
- https://regenxbio.com/
VXA-NVV-104 / Phase 1 Noro GI.1/GII.4
VXA-NVV-104 / Phase 1 Noro GI.1/GII.4
VXA-NVV-104 / Phase 1 Noro GI.1/GII.4
Vaxart Announces First Subject Enrolled in Phase 1b Norovirus Dose-Ranging Trial in Elderly Adults
May 7, 2021 at 8:00 AM EDTPDF Version
Study will evaluate safety and immunogenicity of oral norovirus vaccine in elderly population
Norovirus represents a significant unmet need in the elderly — there currently is no approved vaccine
Safety and immunogenicity data to also inform oral COVID-19 vaccine program in older population
SOUTH SAN FRANCISCO, Calif., May 07, 2021 (GLOBE NEWSWIRE) -- Vaxart, Inc. (Nasdaq: VXRT), a clinical-stage biotechnology company developing oral recombinant vaccines that are administered by tablet rather than by injection, today announced that it has enrolled the first subject in a Phase 1b placebo-controlled, dose-ranging, repeat dose trial investigating its oral norovirus vaccine candidate in elderly subjects aged 55 – 80 years. This study is designed to evaluate the safety and immunogenicity of Vaxart’s candidate, which is the only clinical stage norovirus oral tablet vaccine actively being developed.
Norovirus is the leading cause of vomiting and diarrhea from acute gastroenteritis among people of all ages. In the United States, this virus contributes to 56,000 to 71,000 hospitalizations and 570 to 800 deaths annually.
Vaxart has already performed and reported on three previous clinical trials with a norovirus vaccine candidate:
- In a Phase 1 bivalent study the vaccine showed no interference with the monovalent arms of the study performing as well as the bivalent arm.
- The study met all primary endpoints for safety and demonstrated robust immunogenicity, with 78% - 93% of subjects responding by eliciting IgA antibody secreting cells, a key marker for mucosal immunity and a potential correlate of protection for norovirus disease.
- The study met all primary endpoints for safety and demonstrated robust immunogenicity, with 78% - 93% of subjects responding by eliciting IgA antibody secreting cells, a key marker for mucosal immunity and a potential correlate of protection for norovirus disease.
- In an earlier Phase 1 monovalent study more than 80% of recipients of the high dose vaccine developed mucosally-primed norovirus specific circulating antibody secreting cells, IgA memory and effector B cells expressing the α4β7 gut homing receptor, and a greater than tenfold increase in fecal IgA antibodies.
- In the high dose group of another Phase 1 dose optimization monovalent study, 100 percent of adults responded as measured by a significant increase in IgA and IgG antibody secreting cells after 2 doses.
The dose ranging trial in the elderly is the third of four Vaxart norovirus trials that are being conducted currently or are planned for 2021. Vaxart currently is administering a second booster dose to a subset of subjects who had participated in the prior Phase 1b bivalent study and recently began a study designed to evaluate different boosting regimens.
Vaxart also expects to launch a Phase 2 norovirus human challenge study later this year.
VXA-NVV-104 Phase 1b Trial Design
The Phase 1b study is designed to enroll 48 subjects aged 55 to 80 years old. Subjects will be randomized into two cohorts stratified by age: Cohort 1 will receive either low dose vaccine candidate (1e10 I.U. n=16) or placebo (n=8); Cohort 2 will receive high dose vaccine candidate (1e11 I.U. n=16) or placebo (n=8). The study drug will be an oral tablet administered on Days 1 and 29. The endpoints are safety and immunogenicity. For more information, refer to ClinicalTrials.gov. (NCT04854746).
Ph 1b: Safety & Immunogenicity of Ad5 Based Oral Norovirus Vaccine (VXA-NVV-104)
https://www.clinicaltrials.gov/ct2/show/NCT04854746?term=NCT04854746&draw=2&rank=1
Enrollment underway in Vaxart's early-stage norovirus trial in elderly population
May 07, 2021 8:22 AM ETVaxart, Inc. (VXRT)
By: Mamta Mayani, SA News Editor6 Comments
- Vaxart (NASDAQ:VXRT) has enrolled the first subject in a Phase 1b trial, VXA-NVV-104, evaluating safety and immunogenicity of oral norovirus vaccine in elderly subjects aged 55 – 80 years. The study drug will be an oral tablet administered on Days 1 and 29.
- https://seekingalpha.com/news/3693136-enrollment-underway-in-vaxarts-early-stage-norovirustrial-in-elderly-population
- https://seekingalpha.com/symbol/VXRT
- https://vaxart.com/
COM902
VXA-NVV-104 / Phase 1 Noro GI.1/GII.4
Bardoxolone
Compugen Publishes Preclinical Data Demonstrating Therapeutic Potential of COM902 in Cancer Immunology, Immunotherapy
April 27, 2021Peer reviewed data demonstrate synergistic effect of anti-TIGIT antibody, COM902, with PVRIG and PD-1 blockade for the treatment of cancer
Phase 1 trial of COM902 monotherapy in patients with advanced malignancies is currently on track to report data in Q4 2021; Initiation of dual combination study for COM701 with COM902 expected as planned in H2 2021
HOLON, Israel, April 27, 2021 /PRNewswire/ -- Compugen Ltd. (Nasdaq: CGEN), a clinical-stage cancer immunotherapy company and a leader in predictive target discovery, today announced the publication of a peer-reviewed article titled "COM902, a Novel Therapeutic Antibody Targeting TIGIT Augments Anti-Tumor T Cell Function in Combination with PVRIG or PD-1 Pathway Blockade" in Cancer Immunology, Immunotherapy. The preclinical data discussed in the paper highlight the potential of COM902, Compugen's anti-TIGIT therapeutic antibody, to enhance anti-tumor immune responses.
Article highlights include:
- Development and characterization of COM902, a fully human anti-TIGIT antibody which binds specifically to TIGIT and disrupts the binding of TIGIT with PVR, the cognate ligand of TIGIT
- Expression of TIGIT, a checkpoint within the DNAM-1 axis discovered in 2009 by Compugen and others, is induced on lymphocytes infiltrating the tumor microenvironment, including Tregs, effector T cells, and NK cells in diverse solid tumors
- PVR is expressed in a large setof solid tumors
- COM902, designed to have reduced Fc receptor engagement to avoid potential depletion of TIGIT-expressing effector T cells, does not demonstrate T cell depletion activity in-vitro or in-vivo, making it unlikely to elicit direct depletion of TIGIT-expressing effector T cells, which are important for anti-tumor activity
- Blockade of TIGIT/PVR binding by COM902 enhances human T and NK cell function in vitro. This effect can be increased by dual blockade of COM902 with either an anti-PVRIG antibody, COM701, or an anti-PD-1 antibody
- In vivo, in murine tumor models, COM902 combined with anti-PVRIG or anti-PD-L1 antibodies enhances anti-tumor lymphocyte responses and inhibits tumor growth
COM902 is currently being studied as monotherapy in a Phase 1 trial of patients with advanced malignancies (NCT04354246). The trial, which was initiated in 2020, is on track to report initial data in Q4 2021. In addition, the initiation of the dual combination study of COM701 with COM902 is expected as planned in H2 2021.
For additional information, please visit Compugen's corporate website at www.cgen.com
View original content:
SOURCE Compugen Ltd.
Compugen's COM902 demonstrates potential benefit in cancer
Apr. 27, 2021 8:15 AM ET
By: Mamta Mayani, SA News Editor
- Compugen (NASDAQ:CGEN) announces preclinical data highlighting the potential of COM902, an anti-TIGIT therapeutic antibody, to enhance anti-tumor immune responses.
- https://seekingalpha.com/news/3686026-compugens-com902-demonstrates-potential-benefit-in-cancer
- https://seekingalpha.com/symbol/CGEN
- https://cgen.com/
Bardoxolone
DTX301 Ornithine Transcarbamylase (OTC)
Bardoxolone
Reata Announces FDA Accepted for Filing the NDA for Bardoxolone for the Treatment of Patients With Chronic Kidney Disease Caused by Alport Syndrome
April 26, 2021DOWNLOAD(OPENS IN NEW WINDOW)
Approximately 30,000-60,000 Patients in the United States Are Affected With Alport Syndrome, a Life-Threatening Disease With No Approved Therapies
Application Assigned a PDUFA Date of February 25, 2022
If Approved, Bardoxolone Would Become the First Approved Therapy for Alport Syndrome in the United States
PLANO, Texas, April 26, 2021 (GLOBE NEWSWIRE) -- Reata Pharmaceuticals, Inc. (Nasdaq: RETA) (“Reata,” the “Company,” or “we”), a clinical-stage biopharmaceutical company, today announced that the U.S. Food and Drug Administration (“FDA”) accepted for filing the New Drug Application (“NDA”) for bardoxolone methyl (“bardoxolone”) for the treatment of patients with chronic kidney disease (“CKD”) caused by Alport syndrome.
This NDA submission is based on the efficacy and safety data from the CARDINAL Phase 3 clinical trial. The FDA will review the application under a Standard Review timeline. The Prescription Drug User Fee Act (“PDUFA”) date, the FDA action date for the application, is scheduled for February 25, 2022. The FDA also advised the Company that it is currently planning to hold an Advisory Committee meeting to discuss the application.
“We are pleased with the FDA’s decision to accept for filing our NDA for bardoxolone and look forward to continuing to work with the Division during the review process,” said Warren Huff, Reata’s President and Chief Executive Officer. “Alport syndrome is one of the most rapidly progressive forms of CKD and a truly devastating disease to those patients and the families who are affected by it. If approved, bardoxolone may be the first therapy to slow the progression of kidney disease in patients with this serious and debilitating disease.”
About the CARDINAL Phase 3 Clinical Study
The CARDINAL Phase 3 study was a double-blind, placebo-controlled, randomized trial that enrolled 157 patients with CKD caused by Alport syndrome at approximately 50 study sites in the United States, Europe, Japan, and Australia. Patients were randomized 1:1 to once-daily, oral bardoxolone or placebo. The primary endpoint for Year 2 of the study was the change from baseline in eGFR after 100 weeks of treatment. The key secondary endpoint for Year 2 of the study was the change from baseline in eGFR at Week 104 (four weeks after the last dose in the second year of treatment). Results from CARDINAL demonstrated that patients treated with bardoxolone experienced a statistically significant improvement in kidney function as measured by eGFR at Week 100 and Week 104, compared to patients treated with placebo. Bardoxolone was generally reported to be well tolerated in this study, and the safety profile was similar to that observed in prior trials. The reported adverse events (“AE”) were generally mild to moderate in intensity, and the most common AEs observed more frequently in patients treated with bardoxolone compared to patients treated with placebo were muscle spasms and increases in aminotransferases.
About Bardoxolone
Bardoxolone is an investigational, once-daily, orally administered activator of Nrf2, a transcription factor that induces molecular pathways that promote the resolution of inflammation by restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling. The FDA has granted Orphan Drug designation to bardoxolone for the treatment of Alport syndrome and autosomal dominant polycystic kidney disease (“ADPKD”). The European Commission has granted Orphan Drug designation in Europe to bardoxolone for the treatment of Alport syndrome.
In addition to the CARDINAL Phase 3 study, bardoxolone is currently being studied in FALCON, a Phase 3 study for the treatment of ADPKD, MERLIN, a Phase 2 study for the treatment of patients with CKD at risk of rapid progression, and AYAME, a Phase 3 study for the treatment of diabetic kidney disease that is being conducted by our licensee, Kyowa Kirin Co., Ltd., in Japan. Bardoxolone treatment has produced positive results in Phase 2 studies in patients with CKD caused by ADPKD, IgA nephropathy, focal segmental glomerulosclerosis, and type 1 diabetes.
FDA accepts Reata's chronic kidney disease treatment bardoxolone NDA for review
Apr. 26, 2021 5:03 PM ET
Reata Pharmaceuticals, Inc. (RETA)
By: Aakash Babu, SA News Editor
- Reata Pharmaceuticals (NASDAQ:RETA) says that the FDA has accepted its NDA for bardoxolone for the treatment of patients with chronic kidney disease (“CKD”) caused by Alport syndrome for review and has set an action date of February 25, 2022.
- https://seekingalpha.com/news/3685823-fda-accepts-reatas-chronic-kidney-disease-treatment-bardoxolone-nda-for-review
- https://seekingalpha.com/symbol/RETA
- https://www.reatapharma.com/home/default.aspx
Rigosertib
DTX301 Ornithine Transcarbamylase (OTC)
DTX301 Ornithine Transcarbamylase (OTC)
Apr 22, 2021
Onconova Announces First Patient Dosed In Investigator-Initiated Phase 2 Study Of Rigosertib In Recessive Dystrophic Epidermolysis Bullosa-Associated Squamous Cell Carcinoma
NEWTOWN, Pa., April 22, 2021 (GLOBE NEWSWIRE) -- Onconova Therapeutics, Inc. (NASDAQ: ONTX), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, announced today that the first patient has been dosed in an investigator-initiated Phase 2 study to assess the efficacy and safety of rigosertib in patients with recessive dystrophic epidermolysis bullosa (RDEB)-associated locally advanced/metastatic squamous cell carcinoma (SCC). The patient was dosed at the EB House Austria, a center of expertise for epidermolysis bullosa at the University Hospital Salzburg, Austria. Additional sites are anticipated to be opened in the UK and in the US to study this rare and genomically driven devastating disease.
In this open-label investigator-initiated study, 12 patients will receive either oral or intravenous rigosertib at the clinician’s discretion given the various clinical manifestations of the disease, which may dictate the need for either oral or intravenous administration of rigosertib. These patients have skin desquamation making intravenous access difficult, or may form esophageal strictures, which make oral administration difficult. Patients will receive either oral rigosertib in four-week cycles (three weeks on, one week off) for up to 13 cycles, with 560 mg of oral rigosertib in the morning and again in the afternoon, for a total of 1,120 mg/day. Alternatively, patients will receive intravenous (IV) rigosertib as a 72-hour IV infusion on days 1, 2 and 3 of eight 2-week cycles, and on days 1, 2 and 3 of nine 4-week cycles thereafter, with each 24-hour infusion consisting of 1,800 mg of rigosertib.
Onconova’s product candidate oral rigosertib is being studied in an investigator-initiated study program, including in a dose-escalation and expansion Phase 1 investigator-initiated study targeting patients with KRAS+ lung adenocarcinoma in combination with nivolumab. In addition, Onconova continues to conduct preclinical work investigating rigosertib in COVID-19.
For more information, please visit www.onconova.com.
Onconova announces dosing of first patient in mid-stage skin cancer study
Apr. 22, 2021 8:28 AM ET
Onconova Therapeutics, Inc. (ONTX)
By: Dulan Lokuwithana, SA News Editor
- Onconova Therapeutics (NASDAQ:ONTX) says that the first patient has been dosed in an investigator-initiated Phase 2 study for its oral candidate rigosertib in advanced/metastatic squamous cell carcinoma ("SCC").
- https://seekingalpha.com/news/3684516-onconova-announces-dosing-of-first-patient-in-mid-stage-skin-cancer-study
- https://seekingalpha.com/symbol/ONTX
- https://www.onconova.com/
DTX301 Ornithine Transcarbamylase (OTC)
DTX301 Ornithine Transcarbamylase (OTC)
DTX301 Ornithine Transcarbamylase (OTC)
Apr 22, 2021
Ultragenyx Completes Successful End-of-Phase 2 Meeting with FDA and Finalizes Phase 3 Study Design for DTX301 Ornithine Transcarbamylase (OTC) Gene Therapy Program
Phase 3 Study on Track to Initiate in Second Half 2021
NOVATO, Calif., April 22, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for rare and ultra-rare diseases, today announced the successful completion of an End-of-Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration (FDA) for the DTX301 ornithine transcarbamylase (OTC) deficiency gene therapy program. The meeting focused on the discussion of the Phase 1/2 data and alignment on Phase 3 design and endpoints.
Based on the outcome of this meeting, Ultragenyx has finalized the Phase 3 study design, which will include a 64-week primary efficacy analysis period and enroll approximately 50 patients 12 years of age and older, randomized 1:1 to DTX301 (1.7 x 10^13 GC/kg dose) or placebo. The co-primary endpoints are change in 24-hour plasma ammonia levels and the percent of patients who achieve a response as measured by discontinuation or reduction in baseline disease management. Ultragenyx previously completed an initial Scientific Advice process with the European Medicines Agency (EMA) and this design incorporates the scientific advice.
The Phase 3 study is expected to begin dosing as planned in the second half of 2021. Following the initial 64-week study period, all patients who received placebo will be eligible to receive DTX301.
DTX301 Ornithine Transcarbamylase (OTC)
About DTX301
DTX301 is an investigational AAV type 8 gene therapy designed to deliver stable expression and activity of OTC following a single intravenous infusion. It has been shown in preclinical studies to normalize levels of urinary orotic acid, a marker of ammonia metabolism. DTX301 was granted Orphan Drug Designation in both the United States and Europe.
In the Phase 1/2 study of DTX301, nine patients were treated in the first three dose finding cohorts and an additional 2 patients were enrolled in a cohort where prophylactic steroids were used. Six of the patients in the dose finding cohorts responded to treatment and have demonstrated durable metabolic control and remain in excellent clinical condition with no significant adverse events, hospitalizations or other events related to OTC deficiency.
The 64-week Phase 3 study will enroll 50 patients 12 years of age and older, randomized 1:1 to DTX301 or placebo. The Phase 3 dose for DTX301 is 1.7 x 10^13 GC/kg, as determined by the droplet digital PCR (ddPCR) test method. The co-primary endpoints are change in 24-hour plasma ammonia levels and the percent of patients who achieve a complete response (complete discontinuation of baseline disease management) or a partial response (decreased baseline ammonia scavenger medication and/or liberalized protein-restricted diet by at least 50%). Secondary endpoints include change in ureagenesis which evaluates the capacity to generate urea from ammonia, change in cognitive function, and safety.
https://www.ultragenyx.com/pipeline/dtx-301-for-otc/
For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com.
Ultragenyx finalizes Phase 3 study design for DTX301 OTC gene therapy program
Apr. 22, 2021 8:42 AM ET
Ultragenyx Pharmaceutical Inc. (RARE)
By: Mamta Mayani, SA News Editor
- Ultragenyx Pharmaceutical (NASDAQ:RARE) successfully completes an End-of-Phase 2 (EOP2) meeting with the FDA for the DTX301 ornithine transcarbamylase (OTC) deficiency gene therapy program.
- https://seekingalpha.com/news/3684531-ultragenyx-finalizes-phase-3-study-design-for-dtx301-otc-gene-therapy-program
- https://seekingalpha.com/symbol/RARE
- https://www.ultragenyx.com/
- https://www.ultragenyx.com/pipeline/
biotecHOPE™ Apr/Mar/Feb/Jan/2021
Pepinemab in Combination with Avelumab
Pepinemab in Combination with Avelumab
Pepinemab in Combination with Avelumab
Vaccinex Announces Publication of Results from CLASSICAL-Lung Phase 1b/2 Clinical Trial in Non-Small Cell Lung Cancer in the Peer-Reviewed Journal Clinical Cancer ResearchCLASSICAL-Lung study evaluated pepinemab in combination with the checkpoint inhibitor BAVENCIO® for the treatment of non-small cell lung cancer
ROCHESTER, N.Y., April 21, 2021 (GLOBE NEWSWIRE) -- Vaccinex, Inc. (Nasdaq: VCNX), a clinical-stage biotechnology company pioneering a differentiated approach to treating cancer and neurodegenerative disease through the inhibition of SEMA4D, today announced the publication of results from the company’s CLASSICAL-Lung clinical trial in the journal Clinical Cancer Research, a publication of the American Association for Cancer Research (AACR). CLASSICAL-Lung was a Phase 1b/2 trial evaluating the company’s lead clinical candidate, pepinemab, in combination with the immune checkpoint inhibitor BAVENCIO® (avelumab) for the treatment of non-small cell lung cancer (NSCLC).
The paper, entitled, “A Phase 1b/2 Study of Pepinemab in Combination with Avelumab in Advanced Non-Small Cell Lung Cancer,” presents data showing that pepinemab is clinically active when combined with BAVENCIO® and was well tolerated, with no identified safety concerns. The combination appeared to halt or reverse tumor progression (partial response or stable disease) in a subset of both immunotherapy naïve patients, including patients with often difficult to treat PD-L1–negative or PD-L1–low tumors, and some patients with primary or acquired resistance to prior single-agent anti-PD-1/L1 therapy.
The publication is now available electronically at: https://clincancerres.aacrjournals.org/content/early/2021/04/06/1078-0432.CCR-20-4792
About the CLASSICAL – Lung Clinical Trial
The design of the trial consisted of a 12-subject dose escalation phase to determine the recommended Phase 2 dose of pepinemab in combination with avelumab, followed by a 50-subject dose expansion phase. The study included a total of 21 evaluable patients who were immunotherapy naïve and 32 patients who were refractory or resistant to prior treatment with immune checkpoint inhibitors (predominantly anti–PD-1). The primary objective was to assess safety and tolerability. Secondary objectives included evaluation of efficacy, immunogenicity, and PK/PD. An exploratory objective was to identify candidate biomarkers of activity.
For more information: NCT03268057
Vaccinex's pepinemab combo study shows encouraging activity in lung cancer
Apr. 21, 2021 8:30 AM ETVaccinex, Inc. (VCNX)By: Mamta Mayani, SA News Editor
- Vaccinex (NASDAQ:VCNX) announces results from the its CLASSICAL-Lung clinical trial in the journal Clinical Cancer Research.
- The Phase 1b/2 trial evaluated pepinemab, in combination with the immune checkpoint inhibitor Bavencio (avelumab) for the treatment of non-small cell lung cancer (NSCLC).
- https://seekingalpha.com/news/3683890-vaccinexs-pepinemab-combo-study-shows-encouraging-activity-in-lung-cancer
- https://seekingalpha.com/symbol/VCNX
- https://vaccinex.com/
Pepinemab
Neurology
First in class, anti-SEMA4D therapy for neuroinflammatory/neurodegenerative
disease
https://vaccinex.com/pipeline/pepinemab-neurology/
INDICATIONS
BAVENCIO is a prescription medicine used to treat:
- a type of skin cancer called Merkel cell carcinoma (MCC) in adults and children 12 years of age and older. BAVENCIO may be used when your skin cancer has spread
This indication is approved under accelerated approval based on a clinical trial that measured how many patients responded and how long they responded. Continued approval may depend on benefit demonstrated in ongoing clinical trials.
- a type of cancer in the bladder or urinary tract called urothelial carcinoma (UC) when it has spread or cannot be removed by surgery (advanced UC). BAVENCIO may be used:
- as maintenance treatment when your cancer has responded or stabilized after you have received platinum-containing chemotherapy as your first treatment
- when you have received platinum-containing chemotherapy, and it did not work or is no longer working
It is not known if BAVENCIO is safe and effective in children under the age of 12.
Please see full Prescribing Information, including Medication Guide for patients.
You may also report side effects to EMD Serono at 1-800-283-8083 ext.5563.
(XB2001)
Pepinemab in Combination with Avelumab
Pepinemab in Combination with Avelumab
FDA Gives Go-Ahead for XBiotech’s Candidate Therapy for Phase I/II Double-blind Placebo Controlled Study in Pancreatic Cancer
Apr 19, 2021Download PDF
XBiotech’s New Drug to Enter Clinical Studies in Combination Therapy for Pancreatic Cancer
AUSTIN, Texas, April 19, 2021 (GLOBE NEWSWIRE) -- XBiotech (NASDAQ: XBIT) announced today that the FDA has granted permission to commence clinical trials with its novel drug candidate for treating patients with pancreatic cancer. From 1992 to 2018 the death rate from pancreatic cancer steadily increased in the USA. It is now predicted that pancreatic cancer will claim 48,220 lives and be the 3rd leading cause of cancer death in the USA in 2021 (National Cancer Institute’s Surveillance, Epidemiology, and End Results (SEER) program).
Pancreatic cancer is typically identified at an advanced stage and treatment often includes surgery and aggressive chemotherapy. A current approved treatment involves combination chemotherapy including ONIVYDE and 5-fluorouracil, drugs that have significant toxicities and provide only modest response rates. XBiotech’s new drug candidate (XB2001) specifically targets a process potentially involved in the growth and spread of malignant tumors; and the drug also blocks inflammation associated with tissue injury, which may reduce toxicity associated with the chemotherapy and allow these drugs to be better tolerated and more effective.
The Phase I/II clinical study will evaluate XBiotech’s new drug candidate when added to the ONIVYDE/5-FU combination therapy. The clinical study is chaired by Dr. Shubham Pant, a leading researcher and oncologist at MD Anderson Cancer Center; and will involve at least 15 other top cancer centers around the United States. The Phase 1 portion of the study will examine increasing doses of XBiotech’s new drug and assess tolerability of the combination at escalating doses. Once a safe dose has been determined, the phase 2 portion will begin, enrolling 60 patients, which will be randomized to receive treatment with ONIVYDE/5-FU or ONIVYDE/5-FU combined with XB2001. Clinical endpoints in the study are safety, overall survival, objective response rate, progression free survival, time to treatment failure, clinical benefit response, number of severe adverse advents, as well as biological measures of experimental drug activity.
About True Human™ Therapeutic Antibodies
XBiotech’s True Human™ antibodies are derived without modification from individuals who possess natural immunity to certain diseases. With discovery and clinical programs across multiple disease areas, XBiotech’s True Human antibodies have the potential to harness the body’s natural immunity to fight disease with increased safety, efficacy and tolerability.
About XBiotech
For more information, visit www.xbiotech.com.
FDA gives go-ahead for XBiotech’s XB2001 combo therapy in pancreatic cancer
Apr. 19, 2021 4:27 AM ET XBiotech Inc. (XBIT)
By: Mamta Mayani, SA News Editor2 Comments
- XBiotech (NASDAQ:XBIT) announces that the FDA has granted permission to commence clinical trials with its novel drug candidate for treating patients with pancreatic cancer.
- https://seekingalpha.com/news/3682789-fda-gives-go-ahead-for-xbiotechs-xb2001-combo-therapy-in-pancreatic-cancer
- https://seekingalpha.com/symbol/XBIT
Pepinemab in Combination with Avelumab
lenzilumab™,
lenzilumab™,
Molnupiravir (EIDD-2801/MK-4482)
lenzilumab™,
Humanigen Reports Positive Data With Lenzilumab in the ZUMA-19 CAR-T Phase 1b Study in DLBCL and Plans to Initiate a Potential Registrational Study
Apr 19, 2021 DownloadPDF Format (opens in new window)
- At the recommended Phase 2 dose, lenzilumab in combination with CAR-T, demonstrated a 100% objective response rate (ORR) and no severe cytokine release syndrome or severe neurotoxicity
- Lenzilumab reduced IL-6, CRP, ferritin, MCP-1, IL-8, and IP-10 (CXCL-10) among others
- Humanigen now plans to conduct a randomized, potentially registrational, Phase 2 study with lenzilumab combined with all commercially available CD19 CAR-T therapies in DLBCL
- Humanigen has terminated the ZUMA-19 clinical collaboration agreement with Kite, a Gilead Company
BURLINGAME, Calif.--(BUSINESS WIRE)-- Humanigen, Inc. (Nasdaq: HGEN) (“Humanigen”), a clinical-stage biopharmaceutical company focused on preventing and treating an immune hyper-response called ‘cytokine storm’ with its lead drug candidate, lenzilumab™, today announced positive data from the Phase 1b portion of ZUMA-19 evaluating the efficacy and safety of lenzilumab in patients treated with CAR-T in diffuse large B-cell lymphoma (DLBCL). At the recommended Phase 2 dose of lenzilumab, the ORR was 100% and no patient experienced severe cytokine release syndrome (CRS) or severe neurotoxicity (NT).
ZUMA-19 was a clinical study designed to evaluate the efficacy and safety of lenzilumab and CAR-T (axicabtagene ciloleucel, Axi-Cel) in patients with relapsed or refractory DLBCL.
This study was a standard 3+3 design with three patients administered 600 mg lenzilumab (cohort 1) and three patients administered 1,800 mg lenzilumab (cohort 2) just prior to CAR-T. The recommended Phase 2 dose was determined to be 1,800 mg.
In the six study patients, the ORR was 83% (n=5) which included four complete responses (CR). In cohort 1, there was no severe CRS (≥ grade 3). One patient experienced grade 3 NT with a two-day duration. At the recommended Phase 2 dose (cohort 2), ORR was 100% (n=3) and the toxicity-free CR (CRS and NT < grade 2) was 66% (n = 2). There was no severe CRS or severe NT at the recommended Phase 2 dose. There were no adverse events attributed to lenzilumab across the study.
For more information, visit www.humanigen.com
Humanigen achieves positive outcome for Lenzilumab in small early-stage study
Apr. 19, 2021 9:37 AM ET
By: Dulan Lokuwithana, SA News Editor1 Comment
- Humanigen (HGEN -2.8%) announced positive data from the Phase 1b portion of the ZUMA-19 trial which involved six patients in two cohorts.
- The study was designed to evaluate the efficacy and safety of Lenzilumab with CAR-T in diffuse large B-cell lymphoma (DLBCL). The overall ORR was 83% (n=5) which included four complete responses (“CR”).
- https://seekingalpha.com/news/3682960-humanigen-achieves-positive-outcome-for-lenzilumab-in-small-early-stage-study
- https://seekingalpha.com/symbol/HGEN
CLINICAL-STAGE PIPELINE
https://www.humanigen.com/pipeline
Molnupiravir (EIDD-2801/MK-4482)
Molnupiravir (EIDD-2801/MK-4482)
Molnupiravir (EIDD-2801/MK-4482)
Merck and Ridgeback Biotherapeutics Provide Update on Progress of Clinical Development Program for Molnupiravir, an Investigational Oral Therapeutic for the Treatment of Mild-to-Moderate COVID-19
April 15, 2021 6:45 am ET
Phase 3 MOVe-OUT Study of Molnupiravir in Outpatients to Proceed, Phase 2/3 MOVe-IN Study in Hospitalized Patients Will Not Proceed
KENILWORTH, N.J., & MIAMI--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, and Ridgeback Biotherapeutics today provided an update on the clinical development program for molnupiravir (MK-4482/ EIDD-2801), an investigational orally available antiviral therapeutic. Based on a planned interim analysis of data from the Phase 2, dose-finding portion (Part 1) of two ongoing placebo-controlled Phase 2/3 trials evaluating molnupiravir administered twice a day for five days in outpatients (MOVe-OUT) and hospitalized patients (MOVe-IN) with COVID-19, and from a previously completed Phase 2a dose-ranging study in outpatients, the decision has been made to proceed with the Phase 3 portion (Part 2) of MOVe-OUT in outpatients with COVID-19, evaluating the 800 mg dose of molnupiravir twice daily. Data from MOVe-IN indicate that molnupiravir is unlikely to demonstrate a clinical benefit in hospitalized patients, who generally had a longer duration of symptoms prior to study entry; therefore, the decision has been made not to proceed to Phase 3.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210415005258/en/
About Molnupiravir Protocol MK-4482-006 (also known as EIDD-2801-2003)
Protocol 6 (MK-4482-006) is a Phase 2a, double-blind, placebo-controlled, randomized trial designed to compare the safety, tolerability, and antiviral activity of molnupiravir versus placebo as measured by viral RNA detection in symptomatic, outpatient (at baseline) adults at least 18 years old with SARS-CoV-2 infection as confirmed by viral RNA detection within seven days of symptom onset. Of 202 treated participants, molnupiravir was considered generally well tolerated and of the 4 serious adverse events reported, none were considered study drug related. Preliminary data from this study was previously presented at CROI 2021.
About Molnupiravir Nonclinical studies
Merck has conducted a comprehensive nonclinical program to characterize the safety profile of molnupiravir. This program included assays such as Big Blue and PIG-a which are designed to provide a robust measure of a drug or chemical’s ability to induce mutations in vivo. Animals were administered molnupiravir for longer and at higher doses (mg/Kg) than those employed in human studies. The totality of the data from these studies indicates that molnupiravir is not mutagenic or genotoxic in in vivo mammalian systems.
About Molnupiravir
Molnupiravir (EIDD-2801/MK-4482) is an investigational, orally administered form of a potent ribonucleoside analog that inhibits the replication of multiple RNA viruses including SARS-CoV-2, the causative agent of COVID-19. Molnupiravir has been shown to be active in several preclinical models of SARS-CoV-2, including for prophylaxis, treatment, and prevention of transmission, as well as SARS-CoV-1 and MERS. Molnupiravir was invented at Drug Innovations at Emory (DRIVE), LLC, a not-for-profit biotechnology company wholly owned by Emory University. For more information on molnupiravir clinical trials please visit https://merckcovidresearch.com/
For more information, visit www.merck.com
Merck advances molnupiravir into Phase 3 MOVe-OUT study in COVID-19
Apr. 15, 2021 7:38 AM ET
By: Mamta Mayani, SA News Editor5 Comments
- Merck (NYSE:MRK) and Ridgeback Biotherapeutics provide an update on the development of molnupiravir (MK-4482/ EIDD-2801), an orally available antiviral therapeutic.
- https://seekingalpha.com/news/3682044-merck-advances-molnupiravir-into-phase-3-move-out-study-in-covid-19
- https://seekingalpha.com/symbol/MRK
Lomecel-B
Molnupiravir (EIDD-2801/MK-4482)
Molnupiravir (EIDD-2801/MK-4482)
Longeveron Announces Positive Results of Phase I Clinical Study of Lomecel-B Cell Therapy for Alzheimer’s Disease
April 13, 2021 08:30 ET | Source: Longeveron
- ...
Study meets primary safety endpoint; positive secondary efficacy assessments support potential benefit from Lomecel-B
Decline in cognitive function slower in patients who received low-dose Lomecel-B as compared with placebo
Quality of life metrics improved with Lomecel-B compared to placebo
Complete results being prepared for publication.
On track to initiate Phase 2 study in the second half of 2021.
MIAMI, April 13, 2021 (GLOBE NEWSWIRE) -- Longeveron Inc. (NASDAQ: LGVN) announced today the final results of its Phase I clinical study evaluating the safety and efficacy of intravenous (i.v.) administration of Lomecel-B, an allogeneic bone marrow-derived medicinal signaling cell (MSC) product, in subjects with mild Alzheimer’s disease. Preliminary results were previously reported in the Company’s S-1/A Registration Statement as part of Longeveron’s successful Initial Public Offering in the first quarter of 2021. The study met its primary safety endpoint, which paves the way for future trials in subjects with Alzheimer’s disease. Importantly, several pre-specified secondary efficacy endpoints and biomarker results support potential benefit from Lomecel-B. The complete trial results are currently being prepared for publication in a peer-reviewed journal, and will be posted on the Company’s website in the future. Longeveron also indicated they are on track to commence a Phase 2 study of Lomecel-B in Alzheimer’s disease in the second half of 2021.
The phase 1 trial, funded in part by an Alzheimer’s Association Part the Cloud Challenge on Neuroinflammation grant, used a randomized, placebo-controlled double-blind design testing single i.v. infusion of Lomecel-B 20 million cells (“low-dose”; (n=15)), Lomecel-B 100 million cells (“high-dose”; n=10)), or placebo (n=8). Subjects were followed for 52 weeks post-infusion.
Key findings from new and previously disclosed data:
- Lomecel-B infusion was well-tolerated in this trial, with no treatment-related serious adverse events observed throughout the 1-year follow-up, including no indications of amyloid-related imaging abnormalities (ARIA) as assessed by magnetic resonance imaging (MRI);
- The average Mini Mental State Exam (MMSE) score, which is a measure of cognitive function, declined more slowly in the low-dose Lomecel-B group compared to the placebo group. At 13 weeks after infusion, the low-dose Lomecel-B group MMSE score was higher (better) compared to placebo (difference of 2.69 ± 1.39 points; p=0.0182; 2-sided 95% CI 0.51 – 4.97);
- At 26 weeks post-infusion, patients in the low-dose Lomecel-B arm showed a significantly higher (better) average score on the Quality of Life in Alzheimer Disease (QOL-AD) compared to placebo (difference of 3.85 ± 1.943 points; p=0.0444; 2-sided 95% CI 0.13 – 9.12);
- At 26 weeks post-infusion, the Alzheimer's Disease Cooperative Study Activities of Daily Living (ADCS-ADL), a measure of competence in basic and instrumental activities of daily living, was significantly higher (better) in the low-dose Lomecel-B group compared to placebo (difference of 6.95 ± 3.46 points; p=0.0118; 95% CI 1.99 – 13.94);
- Biomarkers:
- Subjects receiving Lomecel-B had significantly increased serum levels of several pro-vascular biomarkers (VEGF, IL4, and IL-6) relative to the placebo group post-infusion.
- There was a significant increase in D-dimer in the high-dose, but not low-dose, Lomecel-B arm versus placebo;
- Subjects receiving Lomecel-B had significantly increased serum levels of several anti-inflammatory cytokines (sIL-2Rα, IL-4, IL-10 and IL-12) relative to the placebo group post-infusion;
- There were no significant changes in the Lomecel-B arms versus the change in placebo for any of neuronal-related biomarkers examined;
- MRIs showed a significant increase in change in left hippocampus volume in the high-dose Lomecel-B arm versus the change in placebo group at Week 13 (p=0.0311). By Week 26, the difference was no longer significant. The low dose Lomecel-B arm showed no significant difference versus placebo.
Lomecel-B
Potential of Lomecel-B for Treating Alzheimer’s Disease
Medicinal Signaling Cells (MSCs), as the main ingredient of Lomecel-B, have numerous mechanisms of action that may potentially treat the complex pathology associated with Alzheimer’s disease. Beyond the hallmarks of beta amyloid deposits (plaques) and neurofibrillary tangles, Alzheimer’s disease is also characterized by inflammation in the brain (referred to as “neuroinflammation”), poor functioning of the blood vessels of the brain (“neurovasculature dysfunction”), and degeneration of brain cells (“neurodegeneration”), among other features. The properties of MSCs can potentially treat all of these aspects of Alzheimer’s disease pathology, and preclinical studies support this conclusion. Using mouse models of Alzheimer’s disease, MSCs were shown to be able to decrease inflammation in the brain, promote break-down and clearance of Aβ (the protein component of beta amyloid), decrease the protein that causes neurofibrillary tangles, promote new nerve cell formation (“neurogenesis”), and improve cognitive/behavioral performance.
Additional information about the Company is available at www.longeveron.com.
Longeveron (LGVN) shares surge 26% on positive Lomecel-B results in Alzheimer’s disease
Apr. 13, 2021 8:57 AM ETLongeveron Inc. (LGVN)By: Mamta Mayani, SA News Editor
- Longeveron (NASDAQ:LGVN) soars 26% premarket in reaction to the announcement of final results of its Phase I clinical study evaluating the safety and efficacy of intravenous administration of Lomecel-B in subjects with mild Alzheimer’s disease (AD).
- https://seekingalpha.com/news/3681311-longeveron-lgvn-shares-surge-26-on-positive-lomecel-b-results-in-alzheimers-disease
- https://seekingalpha.com/symbol/LGVN
- https://www.longeveron.com/
CLINICAL TRIALS
https://www.longeveron.com/clinical-trials
REQORSA™ immunogene therapy (quaratusugene ozeplasmid)
REQORSA™ immunogene therapy (quaratusugene ozeplasmid)
REQORSA™ immunogene therapy (quaratusugene ozeplasmid)
Genprex Collaborators Report Positive Preclinical Data for REQORSA™ Immunogene Therapy in Non-Small Cell Lung Cancer at the 2021 AACR Annual Meeting
April 12, 2021
REQORSA Enhances Efficacy of Chemo-Immune Combination Therapy in KRAS-LKB1 Mutant NSCLC in Humanized Mice
REQORSA Overcomes Resistance to Targeted Therapy Osimertinib
AUSTIN, Texas — (April 12, 2021) — Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced that its collaborators presented positive preclinical data for the combination of TUSC2 immunogene therapy (REQORSA™) in combination with chemotherapy and immunotherapies for the treatment of non-small cell lung cancer (NSCLC). Collaborators also presented positive preclinical data for the use of REQORSA in combination with targeted therapies for the treatment of NSCLC. These data were presented in two presentations at the 2021 American Association of Cancer Research (AACR) annual meeting. The TUSC2 gene is a tumor suppressor gene and is the active agent in REQORSA.
“We are pleased to have these positive data that provide further support for the therapeutic potential of REQORSA in combination with immunotherapies and targeted therapies in NSCLC presented before an audience of the world’s leading cancer researchers. These data are particularly encouraging as we look to initiate our upcoming combination Acclaim-1 and Acclaim-2 clinical trials of REQORSA in NSCLC,” said Rodney Varner, President and Chief Executive Officer of Genprex. “We know that many patients inevitably develop resistance to immune checkpoint blockade therapy or EGFR-TKI therapy. These data show that REQORSA in combination with immunotherapies and targeted therapies may provide enhanced efficacy in NSCLC that has become resistant to these regimens, offering hope to a large patient population who currently has limited treatment options.”
For more information, please visit the Company’s web site at www.genprex.com
Genprex's Reqorsa combo shows encouraging activity in lung cancer, shares up 3%
Apr. 12, 2021 8:54 AM ETGenprex, Inc. (GNPX)By: Mamta Mayani, SA News Editor
- Genprex (NASDAQ:GNPX) presents positive preclinical data for the combination of TUSC2 immunogene therapy (Reqorsa) in combination with chemotherapy and immunotherapies for the treatment of non-small cell lung cancer (NSCLC). These data were presented at the AACR Meeting 2021.
- https://seekingalpha.com/news/3680963-genprexs-shares-rise-as-reqorsa-combo-shows-encouraging-activity-in-lung-cancer
- https://seekingalpha.com/symbol/GNPX
Pamrevlumab
REQORSA™ immunogene therapy (quaratusugene ozeplasmid)
REQORSA™ immunogene therapy (quaratusugene ozeplasmid)
FibroGen Receives Fast Track Designation from the U.S. FDA for Pamrevlumab for the Treatment of Duchenne Muscular Dystrophy
SAN FRANCISCO, April 12, 2021 (GLOBE NEWSWIRE) -- FibroGen, Inc. (NASDAQ: FGEN) announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the company’s anti-CTGF antibody, pamrevlumab, for the treatment of patients with Duchenne muscular dystrophy (DMD). This designation follows review of the Phase 2 clinical data from a single-arm trial in non-ambulatory patients with DMD, and represents recognition by the FDA that pamrevlumab has the potential to address an unmet medical need for this disease. Pamrevlumab is currently being evaluated in two Phase 3 trials for the treatment of DMD.
“Fast Track designation by the FDA for pamrevlumab in DMD underscores the high unmet medical need for patients suffering from this debilitating disease and potential to advance a new treatment option,” said Mark Eisner, M.D, M.P.H, Chief Medical Officer, FibroGen. “We look forward to working closely with the FDA on the development of pamrevlumab as a potential therapy for DMD.”
About Pamrevlumab
Pamrevlumab is a first-in-class antibody developed by FibroGen that inhibits the activity of connective tissue growth factor (CTGF), an important biological mediator in fibrotic and proliferative disorders. Pamrevlumab is in Phase 3 clinical development for the treatment of locally advanced unresectable pancreatic cancer (LAPC), Duchenne muscular dystrophy (DMD), and idiopathic pulmonary fibrosis (IPF). For information about pamrevlumab studies currently recruiting patients, please visit www.clinicaltrials.gov.
For more information, please visit www.fibrogen.com.
FibroGen wins FDA’s fast track designation for experimental DMD therapy
Apr. 12, 2021 12:35 PM ETFibroGen, Inc. (FGEN)By: Dulan Lokuwithana, SA News Editor
- FibroGen (FGEN +4.5%) announced that the FDA has granted the Fast Track designation for the company’s anti-CTGF antibody, pamrevlumab, for the treatment Duchenne muscular dystrophy (“DMD”).
- https://seekingalpha.com/news/3681069-fibrogen-wins-fdas-fast-track-designation-for-experimental-dmd-therapy
- https://seekingalpha.com/symbol/FGEN
Pamrevlumab is a first-in-class antibody that inhibits the activity of connective tissue growth factor (CTGF), a common factor in chronic fibrotic and proliferative disorders, characterized by persistent and excessive fibrous tissue which can lead to organ dysfunction and failure, and in cancer, characterized by promotion of tumor growth. Pamrevlumab is in Phase 3 clinical development for the treatment of idiopathic pulmonary fibrosis (IPF) and for the treatment of locally advanced unresectable pancreatic cancer (LAPC) and Duchenne muscular dystrophy (DMD), and in Phase 2 clinical development for the treatment of COVID-19. The U.S. Food and Drug Administration has granted Orphan Drug Designation to pamrevlumab for the treatment of patients with IPF, LAPC, and DMD. Pamrevlumab has also received Fast Track designation from the U.S. Food and Drug Administration for the treatment of patients with IPF and LAPC. Across all clinical studies to date, pamrevlumab has consistently demonstrated a good safety and tolerability profile. For information about pamrevlumab studies currently recruiting patients, please visit www.clinicaltrials.gov.
https://www.fibrogen.com/pamrevlumab/
Mavrilimumab
REQORSA™ immunogene therapy (quaratusugene ozeplasmid)
Rubraca® (Rucaparib) & Xtandi® (enzalutamide)
Kiniksa Announces Positive Results for Mavrilimumab Phase 2 Trial in Non-Mechanically Ventilated Severe COVID-19 Patients
April 12, 2021
– Primary endpoint achieved: the proportion of patients alive and free of mechanical ventilation at Day 29 was 12.3 percentage points higher with mavrilimumab versus placebo (p=0.1224 met predefined statistical threshold of p<0.2) –
– 65% reduction in risk of mechanical ventilation/death with mavrilimumab versus placebo (p=0.0175) –
– 61% reduction in risk of death with mavrilimumab versus placebo (p=0.0726) –
– Clinical improvement was observed on top of steroids and/or antivirals –
– Enrollment in the Phase 3 portion of the trial ongoing –
HAMILTON, Bermuda, April 12, 2021 (GLOBE NEWSWIRE) -- Kiniksa Pharmaceuticals, Ltd. (Nasdaq: KNSA) (“Kiniksa”), a biopharmaceutical company with a portfolio of assets designed to modulate immunological pathways across a spectrum of diseases, today announced the Phase 2 portion of the Phase 2/3 trial of mavrilimumab in non-mechanically-ventilated patients (Cohort 1) with severe COVID-19 pneumonia and hyperinflammation achieved its primary efficacy endpoint of the proportion of patients alive and free of mechanical ventilation at Day 29. Mavrilimumab is an investigational fully-human monoclonal antibody that targets granulocyte macrophage colony stimulating factor receptor alpha (GM-CSFRα).
“These data suggest that mavrilimumab may be a transformational treatment option for patients with severe pneumonia due to hyper-inflammatory syndromes, including COVID-19,” said Sanj K. Patel, Chief Executive Officer and Chairman of the Board of Kiniksa. “Additionally, they reinforce our belief in the potential broad utility of mavrilimumab, which has demonstrated positive clinical data across three indications: giant cell arteritis, rheumatoid arthritis, and severe COVID-19. We are engaged with various government agencies to potentially secure resources to help bring mavrilimumab to patients as soon as possible.”
The Phase 2/3 trial is a global, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of mavrilimumab treatment in adults hospitalized with severe COVID-19 pneumonia and hyperinflammation.
Non-mechanically ventilated patients (Cohort 1) treated with mavrilimumab demonstrated a reduction in mechanical ventilation and death at Day 29 pooled across dose levels.
- The proportion of patients alive and free of mechanical ventilation at Day 29 was 12.3 percentage points higher in mavrilimumab recipients (86.7%) compared to placebo recipients (74.4%) (Primary efficacy endpoint; p=0.1224).
- Mavrilimumab recipients experienced a 65% reduction in the risk of mechanical ventilation or death through Day 29 (Hazard Ratio (HR) = 0.35; p=0.0175).
Mavrilimumab
About Mavrilimumab
Mavrilimumab is an investigational fully-human monoclonal antibody that blocks activity of GM-CSF by specifically binding to the alpha subunit of the GM-CSF receptor. Mavrilimumab was dosed in over 550 patients with rheumatoid arthritis through Phase 2b clinical studies in Europe and achieved prospectively-defined primary endpoints of efficacy and safety. Kiniksa’s lead indication for mavrilimumab is giant cell arteritis (GCA), a rare inflammatory disease of medium-to-large arteries. A Phase 2 trial in GCA achieved both the primary and secondary efficacy endpoints with statistical significance. Kiniksa is also evaluating mavrilimumab in severe COVID-19 pneumonia and hyperinflammation. The FDA granted Orphan Drug designation to mavrilimumab for the treatment of GCA in 2020.
Kiniksa posts positive results from mid-stage mavrilimumab COVID-19 trial
Apr. 12, 2021 7:44 AM ETKiniksa Pharmaceuticals, Ltd. (KNSA)By: Aakash Babu, SA News Editor
- Kiniksa Pharmaceuticals (NASDAQ:KNSA) announces the Phase 2 portion of the Phase 2/3 trial of mavrilimumab in non-mechanically-ventilated patients (Cohort 1) with severe COVID-19 pneumonia and hyperinflammation achieved its primary efficacy endpoint of the proportion of patients alive and free of mechanical ventilation at Day 29.
- https://seekingalpha.com/news/3680912-kiniksa-posts-positive-results-from-mid-stage-mavrilimumab-covid-19-trial
- https://seekingalpha.com/symbol/KNSA
- https://www.kiniksa.com/
- https://www.kiniksa.com/our-pipeline/mavrilimumab/
Rubraca® (Rucaparib) & Xtandi® (enzalutamide)
Bria-IMT™ alone and in combination with immune checkpoint inhibitors
Rubraca® (Rucaparib) & Xtandi® (enzalutamide)
Clovis Oncology Highlights Rubraca® (Rucaparib) Clinical Data At AACR Virtual Annual Meeting 2021
April 10, 2021 Download this Press ReleasePDF Format (opens in new window)
- Findings from the Phase 1b RAMP study evaluating the combination of Rubraca and Xtandi® (enzalutamide) in men with unselected mCRPC lay the groundwork for the Phase 3 CASPAR study which is expected to begin enrolling patients shortly
- Phase 1 data from the RUCA-J study of Rubraca in Japanese patients with advanced solid tumors show similar safety and pharmacokinetic profiles to those observed in Western patients
BOULDER, Colo.--(BUSINESS WIRE)-- Clovis Oncology, Inc. (NASDAQ: CLVS) announced that Phase 1 clinical data from studies exploring Rubraca in combination with Xtandi for the treatment of advanced prostate cancer (RAMP) and Rubraca monotherapy in advanced solid tumors in Japanese patients (RUCA-J) will be presented during week one of the American Association for Cancer Research Virtual Annual Meeting (AACR), taking place April 10-15, 2021.
“We remain committed to understanding how Rubraca may benefit patients with cancer, and the data presented at AACR further enhance our understanding in different patient populations and solid tumor types,” said Patrick J. Mahaffy, President and CEO of Clovis Oncology. “The Phase 1b RAMP data for the combination of Rubraca and Xtandi in unselected mCRPC patients help inform the Alliance for Clinical Oncology-sponsored CASPAR Phase 3 trial which is expected to begin enrolling patients soon, and we look forward to learning more about the combination.”
The presentations can also be viewed at https://www.clovisoncology.com/pipeline/scientificpresentations/ .
About Rubraca (rucaparib)
Rucaparib is an oral, small molecule inhibitor of PARP1, PARP2 and PARP3 being developed in multiple tumor types, including ovarian and metastatic castration-resistant prostate cancers, as monotherapy, and in combination with other anti-cancer agents. Exploratory studies in other tumor types are also underway.
Please click here for full Prescribing Information for Rubraca.
Please visit www.clovisoncology.com for more information.
About Alliance for Clinical Trials in Oncology
The Alliance for Clinical Trials in Oncology develops and conducts clinical trials with promising new cancer therapies, and utilizes the best science to develop optimal treatment and prevention strategies for cancer, as well as research methods to alleviate side effects of cancer and cancer treatments. The Alliance is part of the National Clinical Trials Network (NCTN) sponsored by the National Cancer Institute (NCI) and serves as a research base for the NCI Community Research Oncology Program (NCORP). The Alliance comprises nearly 10,000 cancer specialists at hospitals, medical centers, and community clinics across the United States and Canada. Learn more about the Alliance, visit www.AllianceforClinicalTrialsinOncology.org.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210410005008/en/
Clovis Oncology's Rubraca shows promise for prostate cancer in phase 1 trial
Apr. 11, 2021 10:00 AM ETClovis Oncology, Inc. (CLVS)By: Jonathan M Block, SA News Editor5 Comments
- Phase 1b data presented at the AACR 2021 conference on Clovis Oncology's (NASDAQ:CLVS) Rubraca (rucaparib) showed that a combination of the drug with Astellas/Pfizer's Xtandi (enzalutamide) in advanced prostate cancer patients led to a reduction in prostate-specific antigen.
- https://seekingalpha.com/news/3680850-clovis-oncology-rubraca-shows-promise-for-prostate-cancer-in-phase-1-trial
- https://seekingalpha.com/symbol/CLVS
- https://www.rubraca.com/
- https://www.xtandi.com/
Bria-IMT™ alone and in combination with immune checkpoint inhibitors
Bria-IMT™ alone and in combination with immune checkpoint inhibitors
Bria-IMT™ alone and in combination with immune checkpoint inhibitors
BriaCell Therapeutics Presents Clinical Data at the American Association for Cancer Research (AACR) Annual Meeting 2021
POSTED ON APRIL 12, 2021 BY FARRAH DEAN
Clinical and pathological data presented from the clinical trials of Bria-IMT™ alone and in combination with immune checkpoint inhibitors in advanced breast cancer indicates high responding subset with protracted progression-free survival:
• Data suggests a link with cancer grade, disease control, and progression-free-survival in patients treated with Bria-IMT™.
• Highest rate of disease control and longest progression free survival were observed in patients with Grade I/II tumors.
• Median overall survival of 12.5 months in patients with Grade I/II tumors versus 7.2-9.8 months in a recent study of third line breast cancer.
BERKELEY, Calif. and VANCOUVER, British Columbia, April 12, 2021— BriaCell Therapeutics Corp. (Nasdaq: BCTX, BCTXW) (TSX-V:BCT) (“BriaCell” or the “Company”), a clinical-stage biotechnology company specializing in targeted immunotherapies for advanced breast cancer, is pleased to announce the presentation of results from clinical studies with its lead product candidate, Bria-IMT™, summarized in a poster session held at the American Association for Cancer Research (AACR) Annual Meeting 2021, a virtual meeting, held over two weeks (Week 1: April 10-15; Week 2: May 17-21).
The findings indicate disease control in advanced breast cancer patients, including stable disease (SD), partial responses (PR) or complete responses (CR). Disease control was especially noted in patients with Grade I/II (i.e. well or moderately differentiated) tumors or those that matched Bria-IMT™ at 2 or more HLA alleles. Patients with low or undetectable levels of circulating cancer cells were more likely to benefit from therapy.
Analysis and Discussion:
• The Bria-IMT™ regimen with or without checkpoint inhibitors is able to induce an effective immune response and disease control in heavily pre-treated advanced breast cancer patients. The patients were all heavily pretreated and failed multiple prior regimens.
• Delayed Type Hypersensitivity (DTH) to Bria-IMT™ analysis identified a group with significantly higher rates of disease control and progression-free survival (8 months) in both monotherapy and combination therapy studies suggesting a robust immune response is predictive of clinical benefit in these patients.
• Highest levels of disease control and PFS was observed in patients who matched Bria-IMT™ at 2 or more HLA alleles in the monotherapy study but not in the combination therapy study supporting our strategy to develop Bria-OTS™, an off-the-shelf personalized immunotherapy for advanced breast cancer.
• Patients with Grade I/II tumors (median of 8 prior therapy regimens) were more likely to respond with disease control (67%) and longer progression free survival. The response was more pronounced in the patients in the combination therapy study suggesting additive or synergistic effects of checkpoint inhibitors when combined with the Bria-IMT™ regimen. Bria-IMT™, with a molecular signature most closely related to Grade I/II tumors, may result in disease control and clinical benefit especially in this subset of patients.
A copy of the poster is posted at the following: https://briacell.com/novel-technology/scientific-publications/.
For additional information on BriaCell, please visit: https://briacell.com/.
Bria-IMT™
LATEST DEVELOPMENTS
BriaCell is currently conducting a Phase I/IIa clinical trial of Bria-IMT™, BriaCell’s lead candidate, in a Combination Study with immune checkpoint inhibitors such as the Incyte drugs INCMGA00012, an anti-PD-1 antibody similar to pembrolizumab (KEYTRUDA®; manufactured by Merck & Co., Inc., and epacadostat, an orally bioavailable small-molecule inhibitor of indoleamine 2,3-dioxygenase 1 (IDO1). The Combination Study is listed in ClinicalTrials.gov as NCT03328026.
https://briacell.com/novel-technology/bria-imt/
BriaCell's Bria-IMT regimen shows clinical benefit in breast cancer patients, shares up 16%
Apr. 12, 2021 7:05 AM ETBriacell Therapeutics (BCTX)By: Mamta Mayani, SA News Editor
- BriaCell Therapeutics (NASDAQ:BCTX) soars 16% premarket after announcing results from clinical studies with its lead product candidate, Bria-IMT at AACR Meeting 2021.
- https://seekingalpha.com/news/3680893-briacells-bria-imt-regimen-shows-clinical-benefit-in-breast-cancer-patients-shares-up-16
- https://seekingalpha.com/symbol/BCTX
biotecHOPE™ March/February/January 2021
Annamycin
Surufatinib in Combination with Tislelizumab
Copiktra (duvelisib)
Moleculin Receives FDA Approval of Fast Track Designation for Annamycin in the Treatment of Sarcoma Lung Metastases
March 30, 2021
HOUSTON, March 30, 2021 /PRNewswire/ -- Moleculin Biotech, Inc., (Nasdaq: MBRX) (Moleculin or the Company), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting highly resistant tumors and viruses, today announced that the U.S. Food and Drug Administration ("FDA") has approved its request for Fast Track Designation for its drug, Annamycin, for the treatment of soft tissue sarcoma (STS) lung metastases.
Annamycin
"We are pleased to receive our second Fast Track Designation from the FDA for Annamycin. We now have potential pathways for accelerated approval in two indications, STS lung metastases, and the treatment of relapsed or refractory acute myeloid leukemia," commented Walter Klemp, Moleculin's Chairman and CEO. "Not only does this make us eligible for accelerated approval and priority review for our NDA submission, but it serves as an important reminder of the unmet need in STS lung metastases. We are now focused on initiating our internally funded clinical trial in the US, possibly prior to mid-year. In addition, we recently announced a $1.5 million grant awarded in Poland for an investigator initiated clinical trial there for this indication which should start later this year."
Annamycin is a "next generation" anthracycline that has recently been shown in animal models to accumulate in the lungs at up to 30-fold the level of doxorubicin. Importantly, Annamycin has also demonstrated a lack of cardiotoxicity in recently conducted human clinical trials for the treatment of acute myeloid leukemia, so we believe that the use of Annamycin may not face the same usage limitations imposed on doxorubicin.
Annamycin: The Next Generation Anthracycline
Annamycin has little to no cardiotoxicity, avoids multidrug resistance, has been shown to be more potent in AML cell lines and has shown activity in patients for whom standard of care has failed.
https://www.moleculin.com/technology/annamycin/
For more information about the Company, please visit http://www.moleculin.com.
Moleculin Biotech wins second FDA Fast Track Designation for Annamycin
Mar. 30, 2021 8:04 AM ET
Moleculin Biotech, Inc. (MBRX)
By: Dulan Lokuwithana, SA News Editor2 Comments
- Moleculin Biotech (NASDAQ:MBRX) has climbed ~18.1% in the pre-market after announcing that the FDA has approved its request for Fast Track Designation for its experimental drug, Annamycin for the treatment of soft tissue sarcoma (“STS”) lung metastases.
- https://seekingalpha.com/news/3677523-moleculin-biotech-wins-fda-fast-track-designation-for-annamycin
- https://seekingalpha.com/symbol/MBRX
Copiktra (duvelisib)
Surufatinib in Combination with Tislelizumab
Copiktra (duvelisib)
On 25 March 2021, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Copiktra, intended for the treatment of adult patients with relapsed or refractory chronic lymphocytic leukaemia (CLL) and refractory follicular lymphoma (FL). The applicant for this medicinal product is Verastem Europe GmbH.
Copiktra will be available as 15-mg and 25-mg hard capsules. The active substance of Copiktra is duvelisib, an anti-neoplastic agent (ATC code: L01EM04) which acts by inhibiting phosphatidylinositol 3-kinase p110δ (PI3K-δ) and PI3K-γ. These enzymes are involved in the proliferation and survival of malignant B-cell lines and primary CLL tumour cells, and in immunological pathways in the tumour microenvironment of malignant B cells.
The benefits of Copiktra are that it prolongs the survival time without any progression of the disease as compared to ofatumumab in patients with CLL who have received 2 or more prior lines of treatment and induces tumour responses in patients with FL who have received 2 or more prior treatments. The most common side effects are respiratory tract infections, neutropenia, anaemia, thrombocytopenia, headache, dyspnoea, cough, decreased appetite diarrhoea/colitis, nausea, vomiting, abdominal pain, constipation, rash, musculoskeletal pain, arthralgia, pyrexia, fatigue and increased transaminases.
The full indication is the treatment of adult patents with:
- Relapsed or refractory chronic lymphocytic leukaemia (CLL) after at least two prior therapies. (see SmPC section 4.4.and 5.1).
- Follicular lymphoma (FL) that is refractory to at least two prior systemic therapies (see SmPC section 4.4.and 5.1).
Copiktra should be prescribed by physicians experienced in the treatment of cancer.
First published: 26/03/2021
EMA/CHMP/84697/2021
19 Studies found for: CH5126766 OR defactinib | Recruiting, Not yet recruiting, Active, not recruiting, Completed, Enrolling by invitation Studies
50 search results for duvelisib
https://www.verastem.com/?s=duvelisib
Verastem's Copiktra gets positive opinion from CHMP
Mar. 26, 2021 7:53 AM ET
By: Jonathan M Block, SA News Editor1 Comment
- The European Medicine Agency's Committee for Medicinal Products ("CHMP") for Human Use has issued a positive opinion for Verastem's (NASDAQ:VSTM) Copiktra (duvelisib) for the treatment of relapsed/refractory chronic lymphocytic leukemia (CLL) and refractory follicular lymphoma.
- https://seekingalpha.com/news/3676706-verastem-copiktra-gets-positive-opinion-from-chmp
- https://seekingalpha.com/symbol/VSTM
Surufatinib in Combination with Tislelizumab
Surufatinib in Combination with Tislelizumab
Surufatinib in Combination with Tislelizumab
Oncology / Immunology, Press Releases, RNS Announcements | 24 Mar 2021
HUTCHMED Initiates a Phase Ib/II Trial of Surufatinib in Combination with Tislelizumab in Patients with Advanced Solid Tumors
Hong Kong, Shanghai & Florham Park, NJ — Wednesday, March 24, 2021: Hutchison China MediTech Limited (“HUTCHMED”) (Nasdaq/AIM: HCM) has initiated a Phase Ib/II study of surufatinib in combination with BeiGene’s tislelizumab in patients with advanced solid tumors in the U.S. and Europe. The first patient was dosed on March 23, 2021. This trial is to explore potential synergistic activity of the novel, oral angio-immuno kinase inhibitor surufatinib with the anti-PD-1 antibody tislelizumab in enhancing overall antitumor activity from inhibition of angiogenesis along with stimulation of an immune response.
This is an open-label study to evaluate the safety, tolerability, pharmacokinetics and efficacy of surufatinib in combination with tislelizumab in patients with advanced solid tumors. The study consists of two parts: dose finding (Part 1) and dose expansion (Part 2). Part 1 will be conducted to determine the recommended Phase II dose (“RP2D”) and/or the maximum tolerated dose (MTD) of surufatinib in combination with tislelizumab in patients with advanced or metastatic solid tumors who have progressed on, or are intolerant to, standard therapies. Part 2 will be an open-label, multi-cohort design to evaluate the anti-tumor activity of surufatinib in combination with tislelizumab in patients with specific types of advanced or metastatic solid tumors, including neuroendocrine tumors, colorectal cancer, small cell lung cancer, gastric cancer, and soft tissue sarcoma. Patients will receive the RP2D determined in Part 1 of this study. Additional details may be found at clinicaltrials.gov, using identifier NCT04579757.
About Surufatinib
Surufatinib is a novel, oral angio-immuno kinase inhibitor that selectively inhibits the tyrosine kinase activity associated with vascular endothelial growth factor receptor (VEGFR) and fibroblast growth factor receptor (FGFR), which both inhibit angiogenesis, and colony stimulating factor-1 receptor (CSF-1R), which regulates tumor-associated macrophages, promoting the body’s immune response against tumor cells. Its unique dual mechanism of action may be very suitable for possible combinations with other immunotherapies, where there may be synergistic anti-tumor effects.
HUTCHMED currently retains all rights to surufatinib worldwide.
About Surufatinib Development
NETs in the U.S. and Europe: In the U.S., surufatinib was granted Fast Track Designations for development in pNET and epNET in April 2020, and Orphan Drug Designation for pNET in November 2019. A U.S. FDA NDA rolling submission was initiated in December 2020, to be followed by a marketing authorization application (MAA) submission to the European Medicines Agency (EMA) in Europe. The basis to support these filings includes the completed SANET-ep[10] and SANET-p[11] studies, along with existing data from surufatinib in U.S. epNET and pNET patients (clinicaltrials.gov identifier: NCT02549937).
epNETs in China: On December 30, 2020, surufatinib was granted drug registration approval by the National Medical Products Administration of China (“NMPA”) for the treatment of epNET. Surufatinib is marketed in China under the brand name Sulanda®. The approval was based on results from the SANET-ep study, a Phase III trial (clinicaltrials.gov identifier: NCT02588170) in patients with advanced epNETs conducted in China. The study met the pre-defined primary endpoint of progression-free survival (“PFS”) at a preplanned interim analysis. The positive results of this trial were highlighted in an oral presentation at the 2019 ESMO Congress and published in The Lancet Oncology in September 2020.[12] Median PFS was significantly longer for patients treated with surufatinib at 9.2 months, compared to 3.8 months for patients in the placebo group (HR 0.334; 95% CI: 0.223-0.499; p<0.0001). Surufatinib had an acceptable safety profile, with the most common treatment-related adverse events of grade 3 or worse being hypertension (36% of surufatinib patients vs. 13% of placebo patients), proteinuria (19% vs. 0%) and anemia (5% vs. 3%).
pNETs in China: In 2016, we initiated the SANET-p study, which is a pivotal Phase III study in patients with low- or intermediate-grade, advanced pNET in China. It was terminated early as the pre-defined primary endpoint of PFS was met (clinicaltrials.gov identifier: NCT02589821) at a preplanned interim analysis, leading to a second NDA accepted by the NMPA in September 2020. The positive results of this study were presented at the 2020 ESMO Virtual Congress and published simultaneously in The Lancet Oncology[13], demonstrating that surufatinib reduces the risk of disease progression or death by 51% in patients, with median PFS of 10.9 months compared to 3.7 months on placebo (HR 0.491; 95% CI: 0.391-0.755; p=0.0011). The safety profile of surufatinib was manageable and consistent with observations in prior studies.
Biliary tract cancer in China: In March 2019, we initiated a Phase IIb/III study comparing surufatinib with capecitabine in patients with advanced biliary tract cancer whose disease progressed on first-line chemotherapy. The primary endpoint is overall survival (OS) (clinicaltrials.gov identifier: NCT03873532).
Immunotherapy combinations: We have entered into collaboration agreements to evaluate the safety, tolerability and efficacy of surufatinib in combination with anti-PD-1 monoclonal antibodies, including with tislelizumab (BGB-A317), Tuoyi® (toripalimab) and Tyvyt® (sintilimab), which are approved as monotherapies in China.
About Tislelizumab
Tislelizumab (BGB-A317) is a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages. In pre-clinical studies, binding to FcγR on macrophages has been shown to compromise the anti-tumor activity of PD-1 antibodies through activation of antibody-dependent macrophage-mediated killing of T effector cells. Tislelizumab is the first drug from BeiGene’s immuno-oncology biologics program and is being developed internationally as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers.
The NMPA has granted tislelizumab full approval for first-line treatment of patients with advanced squamous non-small cell lung cancer (NSCLC) in combination with chemotherapy. Tislelizumab has also received conditional approval from the NMPA for the treatment of patients with classical Hodgkin’s lymphoma (cHL) who received at least two prior therapies, and for the treatment of patients with locally advanced or metastatic urothelial carcinoma (UC) with PD-L1 high expression whose disease progressed during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. Full approval for these indications is contingent upon results from ongoing randomized, controlled confirmatory clinical trials.
In addition, three supplemental Biologics License Applications for tislelizumab have been accepted by the Center for Drug Evaluation (CDE) of the NMPA and are under review for first-line treatment of patients with advanced non-squamous NSCLC in combination with chemotherapy, for the second- or third-line treatment of patients with locally advanced or metastatic NSCLC who progressed on prior platinum-based chemotherapy, and for previously treated unresectable hepatocellular carcinoma.
Currently, 15 potentially registration-enabling clinical trials are being conducted in China and globally, including 12 Phase 3 trials and two pivotal Phase 2 trials.
In January 2021, BeiGene and Novartis entered into a collaboration and license agreement to develop, manufacture, and commercialize tislelizumab in North America, Europe, and Japan.
Tislelizumab is not approved for use outside of China.
Tislelizumab
Tislelizumab (BGB-A317) is a humanized IgG4 anti–PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages. In pre-clinical studies, binding to FcγR on macrophages has been shown to compromise the anti-tumor activity of PD-1 antibodies through activation of antibody-dependent macrophage-mediated killing of T effector cells. Tislelizumab is the first drug candidate produced from BeiGene’s immuno-oncology biologic program, and we believe it could serve as a key element of our immuno-oncology combination platform. Tislelizumab is being developed as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers.
https://www.beigene.com/science-and-product-portfolio/pipeline/tislelizumab
For more information, please visit: www.hutch-med.com.
Chi-Med, BeiGene launches mid-stage combo study in solid tumors
Mar. 24, 2021 3:17 AM ET
Hutchison China MediTech Limited (HCM)
By: Mamta Mayani, SA News Editor
- Hutchison China MediTech (NASDAQ:HCM) has initiated a Phase Ib/II study of surufatinib in combination with BeiGene’s (NASDAQ:BGNE) tislelizumab in patients with advanced solid tumors in the U.S. and Europe.
- https://seekingalpha.com/news/3675531-chi-med-beigene-launches-mid-stage-combo-study-in-solid-tumors
- https://seekingalpha.com/symbol/HCM
- https://seekingalpha.com/symbol/BGNE
Oral Insulin (ORMD-0801)
177Lu-PSMA-617, a targeted radioligand therapy
Surufatinib in Combination with Tislelizumab
Oramed Initiates Second Phase 3 Oral Insulin Study Under the FDA’s Approved Dual Concurrent Protocol
Patients screened in ORA-D-013-2 study which is recruiting 450 patients in 53 sites in the U.S., Europe and Israel
NEW YORK, March 23, 2021 /PRNewswire/ — Oramed Pharmaceuticals Inc. (Nasdaq: ORMP) (TASE: ORMP) (www.oramed.com), a clinical-stage pharmaceutical company focused on the development of oral drug delivery systems, announced today it has screened the first patients in its ORA-D-013-2 study, the second of two concurrent Phase 3 studies of its oral insulin capsule, ORMD-0801, for the treatment of type 2 diabetes (T2D).
The studies are taking place under U.S. Food and Drug Administration (FDA) approved protocols to treat T2D patients who have inadequate glycemic control over a period of 6 to 12 months. [Enrollment for the other Phase 3 study, ORA-D-013-1, is ongoing and has surpassed 25%]. The double-blinded, placebo-controlled, multi-center randomized studies will recruit a total of 1,125 patients to evaluate the efficacy and safety of ORMD-0801. Efficacy data for the studies will become available after all patients have completed the first 6-month treatment period.
About the Study
The ORA-D-013-2 study is recruiting 450 T2D patients with inadequate glycemic control who are managing their condition with either diet alone or with diet and metformin monotherapy. Patients will be recruited through 28 sites in the U.S. and 25 sites in Western Europe and Israel. The double-blind study will randomize patients 1:1 into two cohorts dosed with 8 mg of ORMD-0801 at night and placebo at night. The primary endpoint of the study is to compare the efficacy of ORMD-0801 to placebo in improving glycemic control as assessed by A1c over a 26-week treatment period, with a secondary endpoint of comparing ORMD-0801 to placebo in maintaining glycemic control over a 52-week treatment period.
About Oramed Pharmaceuticals
Oramed Pharmaceuticals is a platform technology pioneer in the field of oral delivery solutions for drugs currently delivered via injection. Established in 2006, with offices in the United States and Israel, Oramed has developed a novel Protein Oral Delivery (POD™) technology. Oramed is seeking to transform the treatment of diabetes through its proprietary lead candidate, ORMD-0801, which has the potential to be the first commercial oral insulin capsule for the treatment of diabetes. The Company has completed multiple Phase 2 clinical trials under an Investigational New Drug application with the U.S. Food and Drug Administration. In addition, Oramed is developing an oral GLP-1 (Glucagon-like peptide-1) analog capsule, ORMD-0901.
For more information, please visit www.oramed.com.
View original content:http://www.prnewswire.com/news-releases/oramed-initiates-second-phase-3-oral-insulin-study-under-the-fdas-approved-dual-concurrent-protocol-301253898.html
SOURCE Oramed Pharmaceuticals Inc.
https://www.oramed.com/pipeline/ormd-0801-type-1/
https://www.oramed.com/pipeline/ormd-0801-type-2/
Oramed initiates second phase 3 study of oral insulin candidate; shares up 14%
Mar. 23, 2021 9:39 AM ETOramed Pharmaceuticals Inc. (ORMP)By: Jonathan M Block, SA News Editor
- Oramed Pharmaceuticals (NASDAQ:ORMP) has begun the second of two phase 3 trials of its oral insulin candidate for type 2 diabetes, ORMD-0801.
https://seekingalpha.com/symbol/ORMP
177Lu-PSMA-617, a targeted radioligand therapy
177Lu-PSMA-617, a targeted radioligand therapy
177Lu-PSMA-617, a targeted radioligand therapy
Novartis announces positive result of phase III study with radioligand therapy 177Lu-PSMA-617 in patients with advanced prostate cancer
Mar 23, 2021
- Phase III VISION study with 177Lu-PSMA-617 met both primary endpoints, significantly improving overall survival (OS) and radiographic progression-free survival (rPFS) in patients with PSMA-positive metastatic castration-resistant prostate cancer1
- VISION trial findings to be presented at upcoming medical meeting, with regulatory submissions in the US and EU anticipated in 2021
- Novartis is committed to reimagining prostate cancer through targeted radioligand therapy with 177Lu-PSMA-617
- More than 15 dedicated early to late development and research programs underway to identify the next wave of radioligand therapies for cancer
Basel, March, 23, 2021 — Novartis today reported the first interpretable results of the Phase III VISION study evaluating the efficacy and safety of 177Lu-PSMA-617, a targeted radioligand therapy in patients with progressive PSMA-positive metastatic castration-resistant prostate cancer (mCRPC) compared to best standard of care alone. The trial met both primary endpoints of overall survival and radiographic progression-free survival1, helping to move closer the ambition of becoming the targeted treatment for >80% of patients with advanced prostate cancer. The safety profile was consistent with data reported in previous clinical studies1. Results from the VISION trial will be presented at an upcoming medical meeting and included in US and EU regulatory submissions.
177Lu-PSMA-617, a targeted radioligand therapy
“Patients with metastatic castration-resistant prostate cancer have a less than 1 in 6 chance of surviving 5 years2 and need new treatment options. These groundbreaking data confirm our belief in the potential of 177Lu-PSMA-617 to reimagine outcomes for these patients through phenotypic precision medicine. We intend to submit these data to regulatory authorities as soon as possible,” said John Tsai, Head of Global Drug Development and Chief Medical Officer for Novartis. “We would like to thank the patients who volunteered to participate in this study as well as the clinical teams at each of the trial sites. We would not be able to realize our commitment to reimagining medicine without the partnership of patients and their families.”
Radioligand therapy combines a targeting compound that binds to markers expressed by tumors and a radioactive isotope, causing DNA damage that inhibits tumor growth and replication. This therapeutic approach enables targeted delivery of radiation to the tumor, while limiting damage to the surrounding normal tissue. Novartis has established global expertise and specialized supply chain and manufacturing capabilities across its network of four radioligand therapy production sites, and is further increasing capacity to ensure delivery of radioligand therapies like 177Lu-PSMA-617 to patients in need.
About 177Lu-PSMA-617
177Lu-PSMA-617 is an investigational PSMA-targeted radioligand therapy for metastatic castration-resistant prostate cancer. It is a type of precision cancer treatment combining a targeting compound (ligand) with a therapeutic radioisotope (a radioactive particle)10-12. After administration into the bloodstream, 177Lu-PSMA-617 binds to prostate cancer cells that express PSMA13, a transmembrane protein, with high tumor-to-normal tissue uptake10,14,15. Once bound, emissions from the radioisotope damage tumor cells, disrupting their ability to replicate and/or triggering cell death. The radiation from the radioisotope works over very short distances to limit damage to surrounding cells14,16.
About VISION
VISION is an international, prospective, randomized, open-label, multicenter, phase III study to assess the efficacy and safety of 177Lu-PSMA-617 (7.4 GBq administered by i.v. infusion every 6 weeks for a maximum of 6 cycles) plus investigator-chosen best standard of care in the investigational arm, versus best standard of care in the control arm17. Patients with PSMA PET-scan positive mCRPC, and progression after prior taxane and androgen receptor-directed therapy (ARDT), were randomized in a 2:1 ratio in favor of the investigational arm. The alternate primary endpoints were rPFS and OS. The study enrolled 831 patients1.
Find out more at https://www.novartis.com.
Novartis' late-stage Vision study in prostate cancer meets endpoints
Mar. 23, 2021 4:07 AM ET
By: Mamta Mayani, SA News Editor
- Novartis (NYSE:NVS) reports results of the Phase III VISION study evaluating the efficacy and safety of 177Lu-PSMA-617, a targeted radioligand therapy in patients with progressive PSMA-positive metastatic castration-resistant prostate cancer (mCRPC) compared to best standard of care alone.
- https://seekingalpha.com/news/3675070-novartis-late-stage-vision-study-in-prostate-cancer-meets-endpoints
- https://seekingalpha.com/symbol/NVS
Mirikizumab
177Lu-PSMA-617, a targeted radioligand therapy
177Lu-PSMA-617, a targeted radioligand therapy
Lilly's Mirikizumab Helps Patients Achieve Clinical Remission and Improves Symptoms in Adults with Ulcerative Colitis in 12-Week Phase 3 Induction Study
March 16, 2021
Patients treated with mirikizumab met the primary endpoint of clinical remission and all key secondary endpoints compared to placebo
- LUCENT-1 is the first and only Phase 3 study of an anti-IL-23p19 monoclonal antibody to demonstrate reduced bowel urgency in moderate to severe ulcerative colitis
- Safety results in this study were consistent with that of the previous mirikizumab study in ulcerative colitis and studies with the anti-IL-23p19 antibody class
INDIANAPOLIS, March 16, 2021 /PRNewswire/ -- Eli Lilly and Company (NYSE: LLY) announced today that mirikizumab met the primary and all key secondary endpoints in LUCENT-1, a 12-week Phase 3 induction study evaluating the efficacy and safety of mirikizumab for the treatment of patients with moderate to severe ulcerative colitis (UC). LUCENT-2, a multicenter, randomized, double-blind, placebo-controlled maintenance study of mirikizumab in patients who have completed the 12-week LUCENT-1 induction study is ongoing.
About Mirikizumab
Mirikizumab is a humanized IgG4 monoclonal antibody that binds to the p19 subunit of interleukin 23. Mirikizumab is being studied for the treatment of immune diseases, including psoriasis, ulcerative colitis and Crohn's disease.
About the LUCENT Clinical Trial Program
The LUCENT Phase 3 clinical development program for mirikizumab includes LUCENT-1, LUCENT-2 and LUCENT-3. LUCENT-1 (NCT03518086) is a multicenter, randomized, double-blind, placebo-controlled, Phase 3 induction study of mirikizumab in patients with moderate to severe UC who had failed conventional and/or biologic treatments. LUCENT-2 (NCT03524092) is a multicenter, randomized, double-blind, placebo-controlled maintenance study of mirikizumab in patients who have completed the 12-week LUCENT-1 induction study. LUCENT-3 (NCT03519945) is an open label extension study for eligible patients who have participated in mirikizumab UC trials.
The program began in 2018, with full results from the induction and maintenance studies anticipated in early 2022. To learn more about Lilly, please visit us at lilly.com
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SOURCE Eli Lilly and Company
Lilly's mirikizumab meets endpoints in late-stage ulcerative colitis study
Mar. 16, 2021 8:35 AM ET
By: Mamta Mayani, SA News Editor
- Eli Lilly (NYSE:LLY) announces results from Phase 3 LUCENT-1 study evaluating mirikizumab in severe ulcerative colitis (UC).
- In LUCENT-1, mirikizumab met the primary endpoint of clinical remission at Week 12 compared to placebo (p<0.0001).
- https://seekingalpha.com/news/3673072-lillys-mirikizumab-meets-endpoints-in-ulcerative-colitis-study
- https://seekingalpha.com/symbol/LLY
novel anti-amyloid-beta (Abeta) vaccine
Study of COVID-19 Vaccine Candidate in Pediatric Population
Study of COVID-19 Vaccine Candidate in Pediatric Population
AC Immune Announces New Clinical Results in Down Syndrome and Plans for Future Development of Anti-Amyloid-Beta Vaccine
March 16, 2021
PDF Version
Topline ACI-24 Phase 1b immunogenicity and safety results reported today at a global Down syndrome symposium
Reported new data in non-human primates for optimized vaccine formulation which shows strong response against key pathological Abeta species, including oligomeric and pyroglutamate Abeta
LAUSANNE, Switzerland, March 16, 2021 (GLOBE NEWSWIRE) -- AC Immune SA (NASDAQ: ACIU), a Swiss-based, clinical-stage biopharmaceutical company with a broad pipeline focused on neurodegenerative diseases, today announced plans to advance its novel anti-amyloid-beta (Abeta) vaccine into mid-stage clinical testing to treat and prevent the progression of Down syndrome (DS)-related Alzheimer’s disease (AD). Topline results reported today by AC Immune’s Chief Scientific Officer, Dr. Marie Kosco-Vilbois, at a global DS symposium co-sponsored by AC Immune, showed that ACI-24 demonstrated encouraging immunogenicity and safety in Phase 1b clinical testing in people with DS. The Company also disclosed new non-human primate data for an optimized formulation of the vaccine, which shows broad potential for the treatment and prevention of Abeta-driven diseases based on its superior efficacy in non-human primates.
Highlights from the Phase 1b study in DS-related AD
- Vaccination of adults with DS with ACI-24 resulted in encouraging immunogenicity (generation of anti-Abeta antibodies)
- A positive pharmacodynamic response was observed, as measured by an increase in plasma Abeta
- ACI-24 was safe and well tolerated with no serious adverse events (SAEs) reported
- There was no evidence of central nervous system (CNS) inflammation, meningoencephalitis, or ARIA (amyloid-related imaging abnormalities), including ARIA-E (-edema) and ARIA-H (-hemorrhage)
- AC Immune plans to present the full Phase 1b study results at the upcoming Alzheimer’s Association International Conference (AAIC)
Due to the high vulnerability of people with DS to severe COVID-19 sequelae, initiation of the next clinical trial will be delayed to ensure the safety of study participants. In the interim, AC Immune is taking advantage of this time to accelerate development of its optimized anti-Abeta vaccine formulation, which demonstrated encouraging safety and superior immunogenicity results in mouse and non-human primate (NHP) studies. Dr. Kosco-Vilbois presented some of these key findings during her presentation today:
Key preclinical results for the optimized anti-Abeta vaccine formulation in NHPs
- The optimized vaccine formulation primes, boosts, and maintains strong anti-Abeta antibody responses in two NHP species
- The optimized vaccine formulation generates conformation-specific antibodies targeting key pathological Abeta species, including oligomeric and pyroglutamate Abeta
- The antibodies elicited by the optimized vaccine formulation in NHPs showed clear target engagement by binding to human Abeta plaques on AD patient-derived brain tissue
ACI-24 is also currently being tested in a Phase 2 clinical trial in patients with mild AD. In this study, there have been no safety concerns nor evidence for CNS inflammation or ARIA related to ACI-24 in any subject. The Phase 2 study is progressing toward an 18-month interim analysis, which is planned for Q2 2021.
AC Immune shares rise on advancement of Alzheimer’s disease related vaccine
Mar. 16, 2021 8:22 AM ET
By: Aakash Babu, SA News Editor
- AC Immune SA (NASDAQ:ACIU) announces plans to advance its novel anti-amyloid-beta (Abeta) vaccine into mid-stage clinical testing to treat and prevent the progression of Down syndrome (DS)-related Alzheimer’s disease (AD).
- https://seekingalpha.com/news/3673062-ac-immune-shares-rise-on-advancement-of-alzheimers-disease-related-vaccine
- https://www.acimmune.com/
- https://seekingalpha.com/symbol/ACIU
Study of COVID-19 Vaccine Candidate in Pediatric Population
Study of COVID-19 Vaccine Candidate in Pediatric Population
Study of COVID-19 Vaccine Candidate in Pediatric Population
Moderna Announces First Participants Dosed in Phase 2/3 Study of COVID-19 Vaccine Candidate in Pediatric Population
March 16, 2021 at 6:59 AM EDT
Phase 2/3 study expected to enroll 6,750 healthy pediatric participants less than 12 years of age
Study will enroll in the U.S. and Canada
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Mar. 16, 2021-- Moderna Inc. (Nasdaq: MRNA), a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, today announced that the first participants have been dosed in the Phase 2/3 study, called the KidCOVE study, of mRNA-1273, the Company’s vaccine candidate against COVID-19, in children ages 6 months to less than 12 years. The study is being conducted in collaboration with the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH) and the Biomedical Advanced Research and Development Authority (BARDA), part of the Office of the Assistant Secretary for Preparedness and Response at the U.S. Department of Health and Human Services.
About the Moderna COVID-19 Vaccine
The Moderna COVID-19 Vaccine is an mRNA vaccine against COVID-19 encoding for a prefusion stabilized form of the Spike (S) protein, which was co-developed by Moderna and investigators from the NIAID’s Vaccine Research Center. The first clinical batch, which was funded by the Coalition for Epidemic Preparedness Innovations, was completed on February 7, 2020 and underwent analytical testing; it was shipped to the NIH on February 24, 2020, 42 days from sequence selection. The first participant in the NIAID-led Phase 1 study of the Moderna COVID-19 Vaccine was dosed on March 16, 2020, 63 days from sequence selection to Phase 1 study dosing. On May 12, 2020, the U.S. Food and Drug Administration granted the Moderna COVID-19 Vaccine Fast Track designation. On May 29, 2020, the first participants in each age cohort: adults ages 18-55 years (n=300) and older adults ages 55 years and above (n=300) were dosed in the Phase 2 study of the vaccine. On July 8, 2020, the Phase 2 study completed enrolment. Results from the second interim analysis of the NIH-led Phase 1 study of the Moderna COVID-19 Vaccine in the 56-70 and 71+ age groups were published on September 29, 2020 in The New England Journal of Medicine. On July 28, 2020, results from a non-human primate preclinical viral challenge study evaluating the vaccine were published in The New England Journal of Medicine. On July 14, 2020, an interim analysis of the original cohorts in the NIH-led Phase 1 study of the vaccine was published in The New England Journal of Medicine. On November 30, 2020, Moderna announced the primary efficacy analysis of the Phase 3 study of the vaccine conducted on 196 cases. On November 30, 2020, the Company also announced that it filed for Emergency Use Authorization with the U.S. FDA and a Conditional Marketing Authorization (CMA) application with the European Medicines Agency. On December 3, 2020, a letter to the editor was published in The New England Journal of Medicine reporting that participants in the Phase 1 study of the Moderna COVID-19 Vaccine retained high levels of neutralizing antibodies through 119 days following first vaccination (90 days following second vaccination). On December 18, 2020, the U.S. FDA authorized the emergency use of the Moderna COVID-19 Vaccine in individuals 18 years of age or older. Moderna has also received authorization for its COVID-19 vaccine from health agencies in Canada, Israel, the European Union, the United Kingdom, Switzerland, Singapore and Qatar. Additional authorizations are currently under review in other countries and by the World Health Organization.
The Biomedical Advanced Research and Development Authority (BARDA), part of the Office of the Assistant Secretary for Preparedness and Response (ASPR) within the U.S. Department of Health and Human Services (HHS) is supporting the continued research and development of the Company’s COVID-19 vaccine development efforts with federal funding under contract no. 75A50120C00034. BARDA is reimbursing Moderna for 100 percent of the allowable costs incurred by the Company for conducting the program described in the BARDA contract. The U.S. government has agreed to purchase supply of mRNA-1273 under U.S. Department of Defense contract no. W911QY-20-C-0100.
Authorized Use
Moderna COVID-19 Vaccine is authorized for use under an Emergency Use Authorization (EUA) for active immunization to prevent coronavirus disease 2019 (COVID-19) caused by severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) in individuals 18 years of age and older. Moderna COVID-19 Vaccine is investigational and not approved by FDA.
To learn more, visit www.modernatx.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210316005514/en/
Dosing underway in Moderna's study of COVID-19 vaccine in pediatric population
Mar. 16, 2021 7:07 AM ET
By: Mamta Mayani, SA News Editor5 Comments
- The first participants have been dosed in Moderna's (NASDAQ:MRNA) Phase 2/3 KidCOVE study, of mRNA-1273, in children ages 6 months to less than 12 years against COVID-19.
- https://seekingalpha.com/news/3673007-dosing-underway-in-modernas-study-of-covid-19-vaccine-in-pediatric-population
- https://seekingalpha.com/symbol/MRNA
Belzutifan
Study of COVID-19 Vaccine Candidate in Pediatric Population
elivaldogene autotemcel (eli-cel, Lenti-D™) Gene Therapy
Merck Receives Priority Review From FDA for New Drug Application for HIF-2α Inhibitor Belzutifan (MK-6482)
March 16, 2021 6:45 am EST
Application Based on Objective Response Rate From Phase 2 Trial Evaluating Belzutifan in Patients With Von Hippel-Lindau Disease-Associated Renal Cell Carcinoma
New Filing Further Strengthens Merck’s Expanding and Diverse Oncology Portfolio
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted priority review for a New Drug Application (NDA) for the hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor belzutifan (pronounced bell-ZOO-ti-fan), a novel investigational candidate in Merck’s oncology pipeline, for the potential treatment of patients with von Hippel-Lindau (VHL) disease-associated renal cell carcinoma (RCC), not requiring immediate surgery. This NDA is based on data from the Phase 2 Study-004 trial, in which belzutifan showed a confirmed overall response rate of 36.1% (n=22/61) (95% CI: 24.2-49.4) in patients with VHL disease-associated RCC. The FDA has set a Prescription Drug User Fee Act (PDUFA), or target action, date of September 15, 2021.
Belzutifan
About the Phase 2 Study-004 Trial
This application is based on data from Study-004 (ClinicalTrials.gov, NCT03401788 ), which is a Phase 2, open-label trial evaluating belzutifan for the potential treatment of patients with VHL disease who had at least one measurable solid tumor localized to the kidney and who did not require immediate surgery. The study enrolled 61 patients who received belzutifan 120 mg orally once daily until disease progression or unacceptable toxicity. The primary endpoint was objective response rate in VHL disease-associated RCC. Secondary endpoints in RCC tumors include disease control rate, duration of response, time to response, progression-free survival, time to surgery and safety.
Additionally, this study evaluated response rates in other common VHL disease-associated tumors, including pancreatic cysts, pancreatic neuroendocrine tumors, central nervous system (CNS) hemangioblastomas, and retinal hemangioblastomas.
About Belzutifan
Belzutifan (MK-6482) is a novel, potent and selective inhibitor of HIF-2α. Proteins known as hypoxia-inducible factors, including HIF-2α, can accumulate in patients when VHL, a tumor-suppressor protein, is inactivated. If not properly regulated, the accumulation of HIF-2α can stimulate several oncogenes associated with cellular proliferation, angiogenesis and tumor growth, leading to the growth of both benign and malignant tumors. This inactivation of VHL has been observed in more than 90% of clear cell RCC tumors. Research into VHL biology that led to the discovery of HIF-2α was awarded the Nobel Prize in Physiology or Medicine in 2019. For more information, visit www.merck.com View source version on businesswire.com: https://www.businesswire.com/news/home/20210316005293/en/
FDA accepts Merck's Belzutifan application for Von Hippel-Lindau
Mar. 16, 2021 7:01 AM ET
By: Gaurav Batavia, SA News Editor
- Under priority review, the FDA accepts Merck's (NYSE:MRK) New Drug Application (NDA) for HIF-2α Inhibitor Belzutifan (MK-6482) for the potential treatment of patients with von Hippel-Lindau (VHL) disease-associated renal cell carcinoma, not requiring immediate surgery.
- https://seekingalpha.com/news/3673000-fda-accepts-merck-belzutifan-application-for-treatment-of-von-hippel-lindau
- https://seekingalpha.com/symbol/MRK
elivaldogene autotemcel (eli-cel, Lenti-D™) Gene Therapy
BioNTech Recruits Rivals to Boost Covid-19 Vaccine Production
elivaldogene autotemcel (eli-cel, Lenti-D™) Gene Therapy
bluebird bio Presents Long-Term Data for elivaldogene autotemcel (eli-cel, Lenti-D™) Gene Therapy for Cerebral Adrenoleukodystrophy (CALD)
90% of evaluable patients (27/30) alive and free of major functional disabilities (MFDs) at two years follow-up in Phase 2/3 Starbeam study (ALD-102)
Patients in long-term follow-up study (LTF-304) continue to remain alive and MFD-free through up to nearly seven years of follow-up, suggesting eli-cel stabilizes the progression of disease
No reports of graft failure, graft rejection, graft-versus-host disease, replication competent lentivirus or insertional oncogenesis in the 51 patients treated with eli-cel in clinical studies (ALD-102/LTF-304 and ALD-104)
Data presented in oral session during Presidential Symposium at the 47th Annual Meeting of the EBMT
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Mar. 15, 2021-- bluebird bio, Inc. (Nasdaq: BLUE) announced new data from the clinical development program for its investigational elivaldogene autotemcel (eli-cel, Lenti-D™) gene therapy in patients with cerebral adrenoleukodystrophy (CALD), including updated results from the pivotal Phase 2/3 Starbeam study (ALD-102) and the long-term follow-up study LTF-304, as well as safety outcomes from the Phase 3 ALD-104 study. Data were presented today in an oral presentation during the Presidential Symposium at the 47th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT 2021), taking place virtually from March 14 - 17, 2021.
elivaldogene autotemcel (eli-cel, Lenti-D™) Gene Therapy
Eli-cel is a one-time investigational gene therapy designed to add functional copies of the ABCD1 gene into a patient’s own hematopoietic (blood) stem cells (HSCs) that have been transduced ex vivo with the Lenti-D lentiviral vector (LVV). The addition of the functional ABCD1 gene allows patients to produce the adrenoleukodystrophy protein (ALDP), which is thought to activate the breakdown of VLCFAs. The goal of treatment with eli-cel is to stabilize the progression of CALD and consequently preserve as much neurological function as possible. Importantly, with eli-cel, there is no need for donor HSCs from another person.
“CALD is a terrible disease that occurs in early childhood and, if left untreated, often leads to eventual death for these boys, a difficult fact for any clinician to bear. These data from the Phase 2/3 Starbeam study show some potentially promising evidence, with up to almost seven years of follow-up and nearly all patients have a stable neurologic function score (n=31/32), indicating that minimal neurologic function was lost following eli-cel infusion. In addition there were no reports of graft failure, graft rejection, or graft-versus-host disease,” said Dr. Jörn-Sven Kühl, Department of Pediatric Oncology, Hematology and Hemostaseology, Center for Women’s and Children’s Medicine, University Hospital Leipzig. “These long-term results therefore suggest treatment with eli-cel may stabilize disease progression and consequently preserve as much neurological function as possible in boys with CALD.”
eli-cel Presentation at EBMT21
Elivaldogene autotemcel (eli-cel; Lenti-D) gene therapy for cerebral adrenoleukodystrophy: Updated results from the Phase 2/3 study and safety outcomes report from the Phase 3 study
Presenting Author: Jörn-Sven Kühl, M.D., Department of Pediatric Oncology, Hematology and Hemostaseology, Center for Women’s and Children’s Medicine, University Hospital Leipzig
Oral Session & Number: GS2-8 Presidential Symposium
Date & Time: Monday, March 15, 3:33-3:42 PM CET/10:33-10:42 AM EDT; Auditorium 1
All EBMT sessions will be available to registered attendees on-demand on the Annual Meeting website after they are aired live; content will be accessible online for two months following the close of the meeting.
About elivaldogene autotemcel (eli-cel, formerly Lenti-D™ gene therapy)
In October 2020, the European Medicines Agency (EMA) accepted bluebird bio’s marketing authorization application (MAA) for its investigational eli-cel gene therapy for the treatment of patients with cerebral adrenoleukodystrophy (CALD). The EMA accepted eli-cel gene therapy for the treatment of CALD into its Priorities Medicines scheme (PRIME) in July 2018, and previously granted Orphan Medicinal Product designation to eli-cel.
The U.S. Food and Drug Administration (FDA) granted eli-cel Orphan Drug status, Rare Pediatric Disease designation, and Breakthrough Therapy designation for the treatment of CALD. bluebird bio is currently on track to submit the Biologics License Application (BLA) in the U.S. in mid-2021.
Eli-cel is not approved for any indication in any geography.
bluebird bio is currently enrolling patients for a Phase 3 study (ALD-104) designed to assess the efficacy and safety of eli-cel after myeloablative conditioning using busulfan and fludarabine in patients with CALD. Contact clinicaltrials@bluebirdbio.com for more information and a list of study sites.
Additionally, bluebird bio is conducting a long-term safety and efficacy follow-up study (LTF-304) for patients who have been treated with eli-cel for CALD and completed two years of follow-up in bluebird bio-sponsored studies.
The Phase 2/3 Starbeam study (ALD-102) has completed enrollment. For more information about the ALD-102 study visit: www.bluebirdbio.com/our-science/clinical-trials or clinicaltrials.gov and use identifier NCT01896102.
More information about newborn screening is available at https://www.bluebirdbio.com/patients-and-advocacy/newborn-screening-toolkit-for-ALD.
For more information, visit bluebirdbio.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210315005105/en/
Bluebird bio gains 4% on promising eli-cel data in rare metabolic brain disorder
Mar. 15, 2021 6:31 AM ET
By: Mamta Mayani, SA News Editor5 Comments
- Bluebird bio (NASDAQ:BLUE) announces new data from the clinical development program for its its elivaldogene autotemcel (eli-cel, Lenti-D) gene therapy in patients with cerebral adrenoleukodystrophy (CALD), a rare, X-linked metabolic disorder.
- Starbeam Study (ALD-102)/Long-Term Follow-Up Study (LTF-304)
- https://seekingalpha.com/news/3672480-bluebird-bio-gains-4-on-promising-eli-cel-data-in-rare-metabolic-brain-disorder
- https://seekingalpha.com/symbol/BLUE
BioNTech Recruits Rivals to Boost Covid-19 Vaccine Production
BioNTech Recruits Rivals to Boost Covid-19 Vaccine Production
BioNTech Recruits Rivals to Boost Covid-19 Vaccine Production
BioNTech Recruits Rivals to Boost Covid-19 Vaccine Production
German company enlists other firms as its partner, Pfizer, struggles to meet production targets
By Bojan Pancevski
March 13, 2021 8:00 am ET
BioNTech SE, a German company that joined with Pfizer Inc. to manufacture and distribute its vaccine, has marshaled an alliance of 13 companies, including Novartis AG , Merck KGaA and Sanofi SA, in an effort to meet—and perhaps exceed—an ambitious target of making two billion doses of vaccine this year.
The European Union has been struggling with a shortage of vaccines as manufacturers, including British-Swedish pharmaceutical firm AstraZeneca PLC, have fallen behind on their delivery pledges to the bloc.
BioNTech forming an alliance to boost COVID-19 vaccine supplies: WSJ
Mar. 13, 2021 11:59 PM ET
By: Dulan Lokuwithana, SA News Editor14 Comments
- BioNTech (NASDAQ:BNTX), the partner of Pfizer (NYSE:PFE) in COVID-19 vaccine development has enlisted as many as 13 companies in forming a new manufacturing alliance aimed at accelerating the deliveries of its messenger-RNA based COVID-19 shot, the Wall Street Journal reports.
https://seekingalpha.com/symbol/BNTX
https://seekingalpha.com/symbol/PFE
Welcome to BioNTechs’s COVID-19 information portal
Why mRNA represents a disruptive new drug class
https://biontech.de/how-we-translate/mrna-therapeutics
donanemab
BioNTech Recruits Rivals to Boost Covid-19 Vaccine Production
BioNTech Recruits Rivals to Boost Covid-19 Vaccine Production
Lilly's donanemab slowed Alzheimer's disease progression in Phase 2 trial: full data presented at AD/PD™ 2021 and published in NEJM
March 13, 2021Download PDF
Findings from the primary endpoint supported by consistency of all secondary outcome measures assessing cognition and function
TRAILBLAZER-ALZ is the first study to screen and enroll patients based on their tau pathology
Biomarker results suggest potential for long-term disease modification following fixed duration treatment with slowing of tau accumulation across key brain regions
INDIANAPOLIS, March 13, 2021 /PRNewswire/ -- Phase 2 TRAILBLAZER-ALZ results presented today by Eli Lilly and Company (NYSE: LLY) at the 15th International Conference on Alzheimer's & Parkinson Diseases™ 2021 (AD/PD™ 2021) held virtually March 9-14, 2021 and published simultaneously in the New England Journal of Medicine (NEJM) expand on previously reported top-line data that found donanemab met its primary endpoint and showed significant slowing of decline on the integrated Alzheimer's Disease Rating Scale (iADRS), a composite measure of cognition and daily function, in patients with early symptomatic Alzheimer's disease compared to placebo1,2.
About TRAILBLAZER-ALZ Study
TRAILBLAZER-ALZ (NCT03367403) is a randomized, placebo-controlled, double-blind, multi-center Phase 2 study to assess the safety, tolerability and efficacy of donanemab in patients with early symptomatic Alzheimer's disease. The trial enrolled 272 patients who were selected based on cognitive assessments in conjunction with amyloid plaque imaging and tau staging by PET imaging. The study's primary endpoint is change from baseline until 76 weeks in the Integrated Alzheimer's Disease Rating Scale (iADRS), a composite tool combining the Alzheimer's Disease Assessment Scale-Cognitive subscale (ADAS-Cog13) and the Alzheimer's Disease Cooperative Study - instrumental Activities of Daily Living (ADCS-iADL) for function. Key secondary endpoints include changes between baseline and 76 weeks in the Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog13), ADCS-iADL, MMSE, and Clinical Dementia Rating Scale Sum of Boxes (CDR-SB) scores. Other secondary biomarker endpoints include changes from baseline to week 76 in brain amyloid deposition and brain tau deposition and volumetric MRI. The safety, tolerability and efficacy of donanemab are also being evaluated in the ongoing randomized, placebo-controlled, double-blind, multi-center Phase 2 study TRAILBLAZER-ALZ 2 (NCT04437511).
donanemab
Donanemab is a monoclonal antibody that was designed to bind a specific form of post-translationally modified Aß, N-terminal pyroglutamate, and thereby yield rapid and complete clearance of
"We are confident in the results of the TRAILBLAZER-ALZ study," said Daniel Skovronsky, M.D., Ph.D., Lilly's chief scientific officer and president of Lilly Research Laboratories. "This is the first late-stage study in Alzheimer's disease to meet its primary endpoint at the primary analysis. Donanemab has the potential to become a very important treatment for Alzheimer's disease. We were pleased to see not only slowing of cognitive and functional decline, but also very substantial clearance of amyloid plaques and slowing of spread of tau pathology. The constellation of clinical and biomarker results indicates the potential for long-term disease modification. We are grateful to the patients, caregivers, and investigators who participated in this landmark study."
Specifically, at 76 weeks compared to baseline, treatment with donanemab slowed decline by 32 percent compared to placebo as measured by the iADRS, which was statistically significant. As early as nine months (36 weeks) after initiation of treatment, a significant difference in decline by iADRS was observed.
In addition, 40 percent of participants treated with donanemab achieved amyloid negativity as early as six months after starting treatment and 68 percent achieved this target by 18 months. Donanemab is a monoclonal antibody that was designed to bind a specific form of post-translationally modified Aß, N-terminal pyroglutamate, and thereby yield rapid and complete clearance of amyloid plaques.
March 13, 2021
https://seekingalpha.com/symbol/LLY
biotecHOPE™ March/February/January 2021
Morphomer™ TDP-43
DARPin® candidates, EnsovibepMP0420 and MP0423,
DARPin® candidates, EnsovibepMP0420 and MP0423,
AC Immune Presents New Preclinical Data for Therapeutic and Diagnostic Candidates Addressing Novel Targets for Neurodegenerative Diseases
March 09, 2021
PDF Version
Four presentations at AD/PD™ 2021 feature latest findings from wholly owned therapeutic and diagnostic programs targeting pathological forms of alpha-synuclein and TDP-43
First biologically active small molecule inhibitors of intracellular alpha-synuclein aggregation advancing toward in vivo proof-of-concept studies
Anti-TDP-43 therapeutic antibody candidates demonstrate dual mechanism of action against pathological TDP-43 in vivo
LAUSANNE, Switzerland, March 09, 2021 (GLOBE NEWSWIRE) -- AC Immune SA (NASDAQ: ACIU), a Swiss-based, clinical-stage biopharmaceutical company with a broad pipeline focused on neurodegenerative diseases, today outlined new preclinical data that will be presented at the 15th International Conference on Alzheimer’s & Parkinson’s Diseases (AD/PD™), taking place virtually from March 9–14, 2021. Data from the Company’s wholly owned, first-in-class therapeutic and diagnostic programs targeting pathological forms of alpha-synuclein and TAR DNA-binding protein 43 (TDP-43) are described during four oral and e-poster presentations. Together the presentations further illustrate the synergy between AC Immune’s SupraAntigen™ and Morphomer™ technology platforms to deliver a precision medicine approach to treating neurodegenerative diseases (NDD).
Morphomer™ TDP-43 anti-TDP-43 antibody
Details of AC Immune’s AD/PD™ 2021 presentations
Morphomer™ TDP-43 imaging
First-in-class TDP-43 PET tracers were characterized using a newly optimized radiobinding assay, which enabled the identification of several distinct chemical series of promising Morphomers™ that bind to recombinant and brain-derived TDP-43 aggregates. Selected compounds also demonstrate direct target engagement on patient-derived brain tissue, as assessed by a positive signal in a proprietary high-resolution autoradiography assay that co-localized with pathological TDP-43. Medicinal chemistry is ongoing to further optimize the properties of hit compounds, and investigational new drug (IND)-enabling studies are expected to begin in Q3 2021.
Title: Discovery of PET tracers for TDP-43 proteinopathies
Date: Thursday, March 11, 2021 | 10:45 – 11:00 am CET
Presenter: Oral presentation by Tamara Seredenina
Anti-TDP-43 antibody
Data to be presented show that AC Immune’s lead anti-TDP-43 antibody, the first with reported in vivo activity, significantly reduced levels of pathological (phosphorylated or insoluble) forms of TDP-43 in the brain in a murine neurodegenerative disease model. New data also demonstrate the antibody’s dual mechanism of action, showing that it inhibits TDP-43 aggregation and promotes the uptake and clearance of pre-existing TDP-43 aggregates by microglia. The lead candidate is currently in IND-enabling studies and is expected to start preclinical toxicology studies by year end.
Title: TDP-43 antibody directed microglial clearance and inhibition of seeded aggregation mitigates neuropathology in models of TDP-43 proteinopathy
Date: Thursday, March 11, 2021 | 11:15 – 11:30 am CET
Presenter: Oral presentation by Tariq Afroz
Morphomer™ alpha-synuclein imaging
New preclinical data for ACI-12589, a next-generation alpha-synuclein PET tracer being developed as a first-in-class diagnostic imaging agent for Parkinson’s disease and other alpha-synucleinopathies, confirm that the Morphomer™-derived candidate has a desirable brain-PET ligand pharmacokinetic profile in non-human primates. ACI-12589 has previously shown excellent target engagement and signal specificity on tissue samples from patients with alpha-synucleinopathies, including Parkinson’s disease, multiple system atrophy (MSA) and dementia with Lewy bodies (DLB). ACI-12589 is currently being evaluated in a first-in-human study.
Title: [18F]ACI-12589, a novel alpha-synuclein radiotracer as a biomarker in patients with Parkinson’s disease and other synucleinopathies
E-poster ID: P531 / #868
Presenter: E-poster presentation by Efthymia Vokali
Morphomer™ alpha-synuclein small molecule aggregation inhibitor
The first biologically active small molecule inhibitors targeting intracellular alpha-synuclein aggregates have been identified using the Morphomer™ platform. Data to be presented for the first time show that these initial compounds, from several distinct chemical series, significantly decrease alpha-synuclein aggregate formation in cellular assays by interfering with the fibrillation process. Iterative medicinal chemistry optimization led to the identification of compounds with favorable CNS-penetrant pharmacokinetic properties, which will be progressed into in vivo proof-of-concept studies in models of alpha-synucleinopathies, expected to begin in Q3 2021.
AC Immune presents new preclinical data in neurodegenerative diseases
Mar. 09, 2021 8:04 AM ET AC Immune SA (ACIU) By: Mamta Mayani, SA News Editor
AC Immune (NASDAQ:ACIU) has outlined new preclinical data that will be presented at the 15th International Conference on Alzheimer’s & Parkinson’s Diseases, taking place from March 9–14, 2021.
DARPin® candidates, EnsovibepMP0420 and MP0423,
DARPin® candidates, EnsovibepMP0420 and MP0423,
DARPin® candidates, EnsovibepMP0420 and MP0423,
Molecular Partners and Novartis Report Positive Initial Results from Phase 1 Study of its COVID-19 Antiviral Therapy, Ensovibep, in Healthy Volunteers
- Initial findings show ensovibep to be safe and well tolerated with no significant adverse events
- Predictable exposure seen post administration, confirming the expected half-life of 2‑3 weeks
- Global phase 2/3 registrational study planned to initiate in Q2 2021
Zurich-Schlieren, Switzerland, March 09, 2021.
Molecular Partners AG (SIX: MOLN), a clinical-stage biotech company that is developing a new class of custom-built protein drugs known as DARPin® therapeutics, and its collaborator Novartis, today announced initial results from its ongoing phase 1 study of its first tri-specific COVID-19 antiviral treatment, ensovibep (MP0420), in healthy volunteers. DARPin® candidates, MP0420 and MP0423,
About Molecular Partners’ anti-COVID-19 program
Molecular Partners two antiviral DARPin® candidates, MP0420 and MP0423, are designed to target multiple different sites on the SARS-CoV-2 virus simultaneously for enhanced antiviral effects and potential use as both prophylactics and treatments. The benefits of this multi-specificity include cooperative binding, extremely high potencies and potential prevention of viral ‘escape’ via mutations. The candidates are formatted with a half-life extending DARPin® domain that binds to human serum albumin (HSA) to support long-acting activity. All DARPin® candidates are constructed to benefit from high-yield and low-cost microbial manufacturing. Molecular Partners is investigating whether the high thermal stability of DARPin® molecules can be used to overcome cold-chain requirements.
Following strong preclinical data supporting the anti-COVID-19 program candidates, in October 2020 the Company entered into a collaboration with Novartis in the form of an option and license agreement to develop, manufacture and commercialize Molecular Partners’ anti-COVID-19 DARPin® program. Per the terms of the agreement, Molecular Partners is conducting Phase 1 clinical trials for ensovibep and performing all remaining preclinical work for MP0423; Novartis will conduct Phase 2 and Phase 3 clinical trials, with Molecular Partners as sponsor of these trials. Upon option exercise, Novartis would be responsible for all further development and commercialization activities. Molecular Partners is also collaborating with AGC Biologics, Baccinex, and Ivers-Lee Clinical Supply Management (IL-CSM) to support development of its anti-COVID-19 program, and has reached an agreement with the Swiss Government regarding rights to purchase up to 3.2 million doses of MP0420, if it is approved in Switzerland.
For more information see www.molecularpartners.com
Find out more at https://www.novartis.com.
Molecular Partners and Novartis Report Positive Initial Results from Phase 1 Study of its COVID-19 Antiviral Therapy, Ensovibep, in Healthy Volunteers
Tue March 9, 2021 1:00 AM| Accesswire
- Initial findings show ensovibep to be safe and well tolerated with no significant adverse events
- Predictable exposure seen post administration, confirming the expected half-life of 2‑3 weeks
- Global phase 2/3 registrational study planned to initiate in Q2 2021
ZURICH-SCHLIEREN, SWITZERLAND / ACCESSWIRE / March 9, 2021 / Molecular Partners AG (MLLCF) (SWX: MOLN)
https://seekingalpha.com/symbol/MLLCF
Novartis, Molecular Partners report positive early-stage results of COVID-19 antiviral therapy, ensovibep
Mar. 09, 2021 1:44 AM ET Novartis AG (NVS) By: Mamta Mayani, SA News Editor
Molecular Partners (OTCPK:MLLCF) and its collaborator Novartis (NYSE:NVS) announced initial results from their ongoing phase 1 study of first tri-specific COVID-19 antiviral treatment, ensovibep (MP0420), in healthy volunteers.
https://seekingalpha.com/symbol/NVS
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Islatravir (formerly MK-8591)
DARPin® candidates, EnsovibepMP0420 and MP0423,
Islatravir (formerly MK-8591)
Merck Presents Results from Phase 1 Trial Evaluating Investigational Islatravir Subdermal Implant for the Prevention of HIV-1 Infection at CROI 2021
March 8, 2021 11:40 am EST
Company to Proceed with Phase 2 Development Program for Islatravir Subdermal Implant
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced results from a Phase 1 study evaluating the safety, tolerability and pharmacokinetics (PK) of the company’s investigational subdermal drug-eluting implant with potential for extended administration of islatravir for pre-exposure prophylaxis (PrEP) of HIV-1 infection. Islatravir is an investigational nucleoside reverse transcriptase translocation inhibitor (NRTTI) currently being evaluated across a variety of doses, formulations and frequencies for both the treatment of HIV-1 infection in combination with other antiretroviral agents and for the prevention of HIV-1 infection as a single agent. Study results, presented as a late-breaking oral presentation [Presentation 88] at the 2021 Conference on Retroviruses and Opportunistic Infections (CROI 2021), demonstrate that the implant achieved active drug concentrations above the pre-specified PK threshold at 12 weeks across the three doses of islatravir studied (48 mg, 52 mg and 56 mg), and is projected to provide drug concentrations likely above threshold for one year at the 56 mg dose. Based on these findings, Merck plans to initiate a Phase 2 trial to further explore the potential of a subdermal implant containing islatravir as a long-acting option for PrEP for up to 12 months.
Islatravir (formerly MK-8591)
About Islatravir (MK-8591)
Islatravir (formerly MK-8591) is Merck’s investigational nucleoside reverse transcriptase translocation inhibitor (NRTTI) under evaluation in clinical trials for the treatment of HIV-1 infection in combination with other antiretrovirals, including the ILLUMINATE clinical trials program for once-daily treatment. Islatravir is also being studied for pre-exposure prophylaxis (PrEP) of HIV-1 infection as a single agent across a variety of formulations, including the IMPOWER clinical trials evaluating an oral once-monthly regimen.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210308005050/en/
For more information, visit www.merck.com
https://seekingalpha.com/symbol/MRK
UX053
Teplizumab (PRV-031
Islatravir (formerly MK-8591)
Mar 8, 2021PDF Version
Ultragenyx Announces FDA Clearance of Investigational New Drug (IND) Application for UX053, an mRNA for the Treatment of Glycogen Storage Disease Type III
NOVATO, Calif., March 08, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for UX053, an investigational mRNA therapy being evaluated for the treatment of Glycogen Storage Disease Type III (GSDIII). Enrollment in a Phase 1/2 study is expected to begin in the second half of 2021.
About UX053
UX053 is an investigational mRNA-based biologic therapy encoding full-length, glycogen debranching enzyme encapsulated in a lipid nanoparticle (LNP) designed to provide the deficient protein in GSDIII. For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com.
Ultragenyx gets FDA greenlight for UX053 Glycogen Storage Disease trials
Mar. 08, 2021 8:53 AM ETUltragenyx Pharmaceutical Inc. (RARE)By: Aakash Babu, SA News Editor
- Ultragenyx Pharmaceutical (NASDAQ:RARE) announces that the U.S. FDA has given it the go-ahead to proceed with its Investigational New Drug (IND) application for UX053, an investigational mRNA therapy being evaluated for the treatment of Glycogen Storage Disease Type III (GSDIII).https://seekingalpha.com/news/3670217-ultragenyx-gets-fda-greenlight-for-ux053-glycogen-storage-disease-trialshttps://seekingalpha.com/symbol/RARE
VLA 15
Teplizumab (PRV-031
Teplizumab (PRV-031
VALNEVA AND PFIZER ANNOUNCE INITIATION OF PHASE 2 STUDY FOR LYME DISEASE VACCINE CANDIDATE
Monday, March 08, 2021 - 08:30amEST
- The Phase 2 study will include both adult and pediatric subjects with an aim to support acceleration of the vaccine candidate’s pediatric program
- VLA15 will be tested at two different schedules (Month 0-2-6 or Month 0-6) receiving the selected dose of 180µg
- VLA15 is the only Lyme disease vaccine candidate in active clinical development
Saint-Herblain (France) and New York, NY, March 8, 2021 – Valneva SE (“Valneva”), a specialty vaccine company focused on prevention of diseases with major unmet needs, and Pfizer Inc. (NYSE: PFE) today announced initiation of study VLA15-221. The VLA15-221 study builds on previous positive Phase 2 studies, incorporates new dose regimens and is anticipated to be the final Phase 2 study readout before a decision to progress into pivotal Phase 3 studies.
About VLA 15
VLA15 is the only active Lyme disease vaccine candidate in clinical development today, and covers six serotypes that are prevalent in North America and Europe. This investigational multivalent protein subunit vaccine targets the outer surface protein A (OspA) of Borrelia, an established mechanism of action for a Lyme disease vaccine. OspA is one of the most dominant surface proteins expressed by the bacteria when present in a tick. VLA15 has demonstrated strong immunogenicity and safety data in pre-clinical and clinical studies so far. The program was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) in July 20173.
Valneva and Pfizer announced a collaboration for VLA15’s development and commercialization at the end of April 2020. The two companies are working closely together on the next development steps.
About Clinical Study VLA15-221
VLA15-221 is a randomized, observer-blind, placebo-controlled Phase 2 study. It is the first clinical study with VLA15 that enrolls a pediatric population aged 5 years and older.
A total of approximately 600 participants will receive VLA15 at two different immunization schedules (Month 0-2-6 or Month 0-6, 200 volunteers each) or placebo (Month 0-2-6, 200 volunteers). Vaccinees will receive VLA15 at a dose of 180µg, which was selected based on data generated in the two previous Phase 2 studies. The main safety and immunogenicity readout (Primary Endpoint analysis) will be at Month 7, where peak antibody titers are expected. A subset of participants will receive a booster dose of VLA15 or placebo at Month 18 (Booster Phase) and will be followed up for further three years to monitor antibody persistence.
VLA15 will be tested as an alum-adjuvanted formulation and administered intramuscularly. The study will be conducted at sites which are located in areas where Lyme disease is endemic and will enroll volunteers with a cleared past infection with Borrelia burgdorferi, the bacteria that cause Lyme disease, as well as B. burgdorferi naïve volunteers. In addition, to learn more, please visit us on www.Pfizer.com https://valneva.com/
Pfizer and Valneva begin mid-stage study for Lyme disease vaccine candidate
Mar. 08, 2021 9:05 AM ETPfizer Inc. (PFE)By: Dulan Lokuwithana, SA News Editor1 Comment
- Pfizer (NYSE:PFE) and Valneva SE (OTCPK:INRLF) announced the initiation of VLA15-221, a randomized, observer-blind, placebo-controlled Phase 2 study, to evaluate their Lyme disease vaccine candidate, VLA15. Pfizer and Valneva teamed up to co-develop VLA15 in April 2020.https://seekingalpha.com/news/3670230-pfizer-and-valneva-begins-mid-stage-study-for-lyme-disease-vaccine-candidatehttps://seekingalpha.com/symbol/PFEhttps://seekingalpha.com/symbol/INRLF
Teplizumab (PRV-031
Teplizumab (PRV-031
Teplizumab (PRV-031
Provention Bio Announces Publication of Extended Follow-up Data from the Pivotal "At-Risk" TN-10 Study of Teplizumab in Science Translational Medicine-One course of teplizumab delayed insulin dependence by approximately three years and improved beta cell function in at-risk (Stage 2) type 1 diabetes patients-March 03, 2021
RED BANK, N.J., March 3, 2021 /PRNewswire/ -- Provention Bio, Inc. (Nasdaq: PRVB), a biopharmaceutical company dedicated to intercepting and preventing immune-mediated disease, today announced that extended follow-up data from the pivotal "At-Risk" TN-10 Study were published in Science Translational Medicine. Results show that a single 14-day infusion course of teplizumab (PRV-031) delayed the onset of clinical disease and insulin dependence in at-risk type 1 diabetes (T1D) patients by approximately three years (median of 32.5 months), adding one year to previously reported results. The TN-10 Study was conducted through the Type 1 Diabetes TrialNet, an international research collaboration aimed at discovering ways to delay or prevent type 1 diabetes.
Teplizumab (PRV-031
About Teplizumab (PRV-031):
Teplizumab is an investigational anti-CD3 monoclonal antibody (mAb) with a filed Biologics License Application (BLA) under Priority Review by the U.S. Food and Drug Administration (FDA) for the delay or prevention of clinical type 1 diabetes (T1D) in at-risk individuals. More than 800 patients have received teplizumab in multiple clinical studies involving more than 1,000 subjects. In previous studies of newly diagnosed patients, teplizumab consistently demonstrated the ability to preserve beta-cell function, a measure of endogenous insulin production. It correspondingly reduced the need for exogenous insulin use. Teplizumab has been granted Breakthrough Therapy Designation by the FDA and PRIME designation by the European Medicines Administration. Provention is currently also evaluating teplizumab in patients with newly diagnosed insulin-dependent T1D (the Phase 3 PROTECT study).
Teplizumab, Provention's lead drug candidate, is an anti-CD3 monoclonal antibody currently under review by the U.S. Food and Drug Administration (FDA) for the delay or prevention of clinical T1D in at-risk individuals, defined as having two or more T1D-related autoantibodies and dysglycemia (Stage 2 T1D). The lifetime risk of insulin-dependent clinical disease (Stage 3 T1D) approaches 100% in these pre-symptomatic Stage 2 patients.
"Teplizumab is the first disease-modifying investigational drug with data showing an ongoing delay to insulin-dependent T1D, now by approximately three years after a single course," said Dr. Kevan Herold, M.D., Professor of Immunology and Medicine at Yale University, lead author of the study. "These data build on existing clinical evidence demonstrating the potential for teplizumab to change the course of the disease and advance the treatment paradigm. We are continuing to observe patients in the TN-10 Study to determine whether the observed delay will extend even further over time."
Visit www.ProventionBio.com for more information
Follow-up data published for Provention Bio's type 1 diabetes candidate teplizumab
Mar. 03, 2021 5:02 PM ET
By: Jonathan M Block, SA News Editor5 Comments
- Provention Bio (NASDAQ:PRVB) says extended follow-up data from a phase 3 trial of its type 1 diabetes candidate teplizumab showed that a single 14-day infusion of the monoclonal antibody (mAb) delayed the onset of clinical disease and insulin dependence in at-risk type 1 diabetes (T1D) patients by approximately three years (median of 32.5 months)
https://seekingalpha.com/symbol/PRVB
Efgartigimod
REGN1908-1909
REGN1908-1909
argenx Announces FDA Acceptance of BLA Filing for Efgartigimod for the Treatment of Generalized Myasthenia Gravis
News March 2, 2021argenx Announces FDA Acceptance of BLA Filing for Efgartigimod for the Treatment of Generalized Myasthenia Gravis
Breda, the Netherlands
- If approved, efgartigimod will be the first-and-only approval of an FcRn antagonist
- Prescription Drug User Fee Act (PDUFA) target action date is December 17, 2021
- Pre-approval access program opened in U.S. for efgartigimod for eligible people living with gMG
Efgartigimod
About Efgartigimod
Efgartigimod is an investigational antibody fragment designed to reduce disease-causing immunoglobulin G (IgG) antibodies and block the IgG recycling process. Efgartigimod binds to the neonatal Fc receptor (FcRn), which is widely expressed throughout the body and plays a central role in rescuing IgG antibodies from degradation. Blocking FcRn reduces IgG antibody levels representing a logical potential therapeutic approach for several autoimmune diseases known to be driven by disease-causing IgG antibodies, including: myasthenia gravis (MG), a chronic disease that causes muscle weakness; pemphigus vulgaris (PV), a chronic disease characterized by severe blistering of the skin; immune thrombocytopenia (ITP), a chronic bruising and bleeding disease; and chronic inflammatory demyelinating polyneuropathy (CIDP), a neurological disease leading to impaired motor function.
For more information, visit www.argenx.com
https://www.argenx.com/pipeline
FDA accepts argenx's efgartigimod application for neuromuscular disease
Mar. 02, 2021 2:32 AM ET
By: Mamta Mayani, SA News Editor
- The FDA has accepted for review argenx's (NASDAQ:ARGX) Biologics License Application (BLA) for intravenous (IV) efgartigimod for the treatment of generalized myasthenia gravis (gMG).
https://seekingalpha.com/symbol/ARGX
REGN1908-1909
REGN1908-1909
REGN1908-1909
February 27, 2021 at 6:25 PM EST
REGENERON ANNOUNCES POSITIVE PHASE 2 DATA EVALUATING FEL D 1 ANTIBODY COCKTAIL IN CAT-ALLERGIC PATIENTS WITH MILD ASTHMA
TARRYTOWN, N.Y., Feb. 27, 2021 /PRNewswire/ --
Single administration of novel antibody cocktail controlled patients' allergic response to cat allergen, preventing early asthma reactions for the duration of the 3-month trial
Patients experienced significant improvements in lung function and cat allergen tolerance from the first assessment at week 1
Results presented at the virtual 2021 AAAAI Annual Meeting
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced detailed results from a Phase 2 proof-of-concept trial evaluating the investigational antibody cocktail REGN1908-1909 in cat-allergic patients with mild asthma. The trial met the primary endpoint of preventing early asthma reactions (EAR, defined as a ≥20% decline in forced expiratory volume over one second [FEV1]). The trial also met key secondary endpoints, including improved lung function and an increased amount of cat allergen that patients could tolerate following a single dose of treatment, from as early as the first assessment conducted at week 1. The results were shared in an oral presentation at the virtual 2021 American Academy of Allergy, Asthma and Immunology (AAAAI) Annual Meeting.
About the Phase 2 Trial
The randomized, double-blind, parallel-group, single-dose proof-of-concept trial enrolled 56 cat-allergic patients with mild asthma who were not living with a cat. The trial consisted of up to a 12-week screening period followed by 1:1 randomization on day 1 to receive 600 mg REGN1908-1909 or placebo administered subcutaneously, followed by a 12-week assessment period and a 4-week safety follow-up period. During the screening period, patients underwent a 2-hour CAC in an EEU after initially being evaluated in a placebo challenge. Patients performed spirometry every 10 minutes during the EEU CAC until they experienced an EAR, or bronchoconstriction when their FEV1 was reduced by ≥20%. Following the CAC, patients were monitored for 6 hours and those meeting eligibility requirements were then randomized to receive a single 600 mg subcutaneous dose of REGN1908-1909 or placebo, and returned to the trial site to undergo a 4-hour CAC in the EEU (plus a 6-hour observation period) at weeks 1, 4, 8 and 12 following treatment administration.
The primary endpoint was prevention of EAR, as measured by FEV1, compared to placebo 1 week after treatment, following the CAC. Key secondary endpoints included the prevention of EAR on weeks 4, 8 and 12; change in FEV1 on weeks 1, 4, 8 and 12; and change in cat allergen quantity, as experienced by patients during exposure on weeks 1, 4, 8 and 12.
REGN1908-1909
About Regeneron's VelocImmune® Technology
REGN1908-1909 was invented using Regeneron's VelocImmune technology that utilizes a proprietary genetically-engineered mouse platform endowed with a genetically-humanized immune system to produce optimized fully-human antibodies. When Regeneron's co-Founder, President and Chief Scientific Officer George D. Yancopoulos was a graduate student with his mentor Frederick W. Alt in 1985, they were the first to envision making such a genetically-humanized mouse, and Regeneron has spent decades inventing and developing VelocImmune and related VelociSuite® technologies. Dr. Yancopoulos and his team have used VelocImmune technology to create multiple antibodies including Dupixent® (dupilumab), Libtayo®, Praluent® (alirocumab), Kevzara® (sarilumab), Evkeeza™ (evinacumab-dgnb), Inmazeb™ (atoltivimab, maftivimab, and odesivimab-ebgn) and Regeneron's antibody cocktail for COVID-19, which was recently granted Emergency Use Authorization (EUA) in the U.S.
For additional information about the company, please visit www.regeneron.com
Regeneron’s antibody cocktail shows promise in cat-allergic patients with mild asthma
Feb. 27, 2021 9:04 PM ET
Regeneron Pharmaceuticals, Inc. (REGN)
By: Dulan Lokuwithana, SA News Editor4 Comments
- Regeneron Pharmaceuticals (NASDAQ:REGN) has announced positive results from a Phase 2 proof-of-concept trial evaluating its investigational antibody cocktail REGN1908-1909 in cat-allergic patients with mild asthma.
https://seekingalpha.com/symbol/REGN
Tipifarnib
REGN1908-1909
Tipifarnib
Kura Oncology Receives FDA Breakthrough Therapy Designation for Tipifarnib in Head and Neck Squamous Cell Carcinoma
February 24, 2021 at 7:00 AM EST
SAN DIEGO, Feb. 24, 2021 (GLOBE NEWSWIRE) -- Kura Oncology, Inc. (Nasdaq: KURA), a clinical-stage biopharmaceutical company committed to realizing the promise of precision medicines for the treatment of cancer, today announced that its investigational drug, tipifarnib, has been granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with recurrent or metastatic HRAS mutant head and neck squamous cell carcinoma (HNSCC) with variant allele frequency ≥ 20% after disease progression on platinum-based chemotherapy. Tipifarnib is currently being evaluated in an ongoing registration-directed clinical trial (AIM-HN) in this indication of high unmet need.
Tipifarnib
Tipifarnib’s Breakthrough Therapy Designation is based on data from RUN-HN, a Phase 2 clinical trial evaluating tipifarnib in patients with recurrent or metastatic HRAS mutant HNSCC. Data from this trial, presented at the American Society of Clinical Oncology Virtual Scientific Program in May 2020, showed an ORR of 50%, median PFS of 5.9 months and a median OS of 15.4 months among the 18 evaluable patients. HRAS represents approximately 4-8% of HNSCC patients. The HRAS biomarker can be found on most commercially available genomic panels.
About Tipifarnib
Tipifarnib, is a potent, selective and orally bioavailable inhibitor of farnesyl transferase in-licensed from Janssen. Previously, tipifarnib was studied in more than 5,000 cancer patients and showed compelling and durable anti-cancer activity in certain patient subsets; however, no molecular mechanism of action had been determined that could explain its clinical activity across a range of solid tumor and hematologic indications. Leveraging advances in next generation sequencing as well as emerging information about cancer genetics and tumor biology, the Company is seeking to identify those patients most likely to benefit from tipifarnib. In addition to Breakthrough Therapy Designation, tipifarnib has been granted Fast Track designation by the FDA for the treatment of patients with HRAS mutant HNSCC. In addition to HNSCC, tipifarnib has demonstrated encouraging clinical activity in a number of additional genetically defined tumor types. Kura has received multiple issued patents for tipifarnib, providing patent exclusivity in the U.S. and foreign countries.
For additional information about Kura, please visit the Company’s website at www.kuraoncology.com.
Tipifarnib in Patients with HRAS Mutant Head and Neck Squamous Cell Carcinoma (HNSCC)
https://kuraoncology.com/pipeline/#tipifarnib
Kura climbs on FDA breakthrough therapy status for tipifarnib in head and neck carcinoma
Feb. 24, 2021 7:27 AM ET
By: Dulan Lokuwithana, SA News Editor
- Kura Oncology (NASDAQ:KURA) has added ~14.1% in the premarket after announcing that it has received the breakthrough therapy designation from the FDA for tipifarnib in the treatment of recurrent or metastatic HRAS mutant head and neck squamous cell carcinoma (“HNSCC”).
https://seekingalpha.com/symbol/KURA
GEN-1
EPI-7386
Tipifarnib
Feb 22, 2021
Celsion Corporation Receives FDA Fast Track Designation for GEN-1 in Advanced Ovarian Cancer
Celsion’s Commitment to Promoting Immune System Solutions to Fight Life-Threatening Diseases
Granted an Accelerated Development Pathway
Designation Provides Potential for an Expedited Regulatory Review.
LAWRENCEVILLE, N.J., Feb. 22, 2021 (GLOBE NEWSWIRE) -- Celsion Corporation (NASDAQ: CLSN), a clinical stage development company focused on DNA based immunotherapy and next generation vaccines, today announced that it has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for GEN-1, its DNA-mediated interleukin-12 (IL-12) immunotherapy currently in Phase II development for the treatment of advanced ovarian cancer. GEN-1 was designed using TheraPlas, Celsion's proprietary, synthetic, non-viral nanoparticle delivery system platform.
GEN-1
About GEN-1 Immunotherapy
GEN-1, designed using Celsion's proprietary TheraPlas platform technology, is an IL-12 DNA plasmid vector encased in a nanoparticle delivery system, which enables cell transfection followed by persistent, local secretion of the IL-12 protein. IL-12 is one of the most active cytokines for the induction of potent anti-cancer immunity acting through the induction of T-lymphocyte and natural killer (NK) cell proliferation. The Company has previously reported positive safety and encouraging Phase I results with GEN-1 given as monotherapy or a combination therapy in patients with advanced peritoneally metastasized primary or recurrent ovarian cancer, and recently completed a Phase Ib dose-escalation trial (OVATION I Study) of GEN-1 in combination with carboplatin and paclitaxel in patients with newly diagnosed ovarian cancer.
GEN-1 is the subject of Celsion’s Phase II OVATION 2 Study, which combines GEN-1 with standard-of-care neoadjuvant chemotherapy (NACT) in patients newly diagnosed with Stage III/IV ovarian cancer. NACT is designed to shrink the cancer as much as possible for optimal surgical removal after three cycles of chemotherapy. Following NACT, patients undergo interval debulking surgery, followed by three adjuvant cycles of chemotherapy and up to nine additional weekly GEN-1 treatments, the goal of which is to delay progression and improve overall survival. The OVATION 2 Study is an open-label, 1-to-1 randomized trial, 80% powered to show the equivalent of a 33% improvement in progression-free survival (PFS) (HR=0.75), the primary endpoint, when comparing the treatment arm (standard of care + GEN-1) with the control arm (standard of care alone).
For more information on Celsion, visit www.celsion.com.
Celsion shares surge 51% following ovarian cancer candidate Fast Track designation
Feb. 22, 2021 8:16 AM ETCelsion Corporation (CLSN)By: Jonathan M Block, SA News Editor1 Comment
- The FDA has granted Fast Track designation to Celsion's (NASDAQ:CLSN) GEN-1, an immunotherapy in phase 2 for advanced ovarian cancer.
https://seekingalpha.com/symbol/CLSN
RGX-314
EPI-7386
EPI-7386
REGENXBIO ANNOUNCES ADDITIONAL POSITIVE INTERIM PHASE I/IIA AND LONG-TERM FOLLOW-UP DATA OF RGX-314 FOR THE TREATMENT OF WET AMD
February 16, 2021 at 7:05 AM EST
ROCKVILLE, Md., Feb. 16, 2021 /PRNewswire/ --
- RGX-314 using subretinal delivery continues to be generally well-tolerated at all dose levels
- Positive interim update from Cohorts 4 and 5 at 1.5 years after RGX-314 administration
- Durable treatment effect observed with stable visual acuity, decreased retinal thickness, and reductions in anti-VEGF injection burden
- Long-term, durable treatment effect over three years demonstrated in Cohort 3
- Mean improvement in vision and stable retinal thickness
- 50% of patients (3/6) remain anti-VEGF injection-free over three years; 67% of patients (4/6) are anti-VEGF injection-free from nine months to three years
- ATMOSPHERE™, the first of two planned pivotal trials for RGX-314, is active and enrolling
REGENXBIO Inc. (Nasdaq: RGNX) reported at the Angiogenesis, Exudation, and Degeneration 2021 conference additional positive interim data from Cohorts 4 and 5 of its RGX-314 Phase I/IIa trial for the treatment of wet age-related macular degeneration (wet AMD), and Cohort 3 of its Long-Term Follow-Up (LTFU) study. RGX-314 is a potential best-in-class, one-time gene therapy for the treatment of wet AMD.
RGX-314
About RGX-314
RGX-314 is being developed as a potential one-time treatment for wet AMD, diabetic retinopathy, and other chronic retinal conditions. RGX-314 consists of the NAV® AAV8 vector, which encodes an antibody fragment designed to inhibit vascular endothelial growth factor (VEGF). RGX-314 is believed to inhibit the VEGF pathway by which new, leaky blood vessels grow and contribute to the accumulation of fluid in the retina.
REGENXBIO is advancing two separate routes of administration of RGX-314 to the eye, through a standardized subretinal delivery procedure as well as delivery to the suprachoroidal space. REGENXBIO has licensed certain exclusive rights to the SCS Microinjector® from Clearside Biomedical, Inc. to deliver gene therapy treatments to the suprachoroidal space of the eye.
View original content to download multimedia:http://www.prnewswire.com/news-releases/regenxbio-announces-additional-positive-interim-phase-iiia-and-long-term-follow-up-data-of-rgx-314-for-the-treatment-of-wet-amd-301228344.html
https://regenxbio.com/rgx-314/
Regenxbio's RGX-314 shows potential as a one-time treatment option in wet AMD
Feb. 16, 2021 7:36 AM ET
By: Mamta Mayani, SA News Editor3 Comments
- Regenxbio (NASDAQ:RGNX) reports additional positive interim data from Cohorts 4 and 5 of its RGX-314 Phase I/IIa trial for the treatment of wet age-related macular degeneration (wet AMD), and Cohort 3 of its Long-Term Follow-Up study.
https://seekingalpha.com/symbol/RGNX
EPI-7386
EPI-7386
EPI-7386
ESSA Pharma Presents Favorable Initial Phase 1 Clinical Pharmacology Data of EPI-7386 for Advanced Forms of Prostate Cancer at the 2021 ASCO Genitourinary Cancers Symposium Vancouver, Canada and Houston, Texas, February 11, 2021 - ESSA Pharma Inc. (“ESSA”, or the “Company”) (NASDAQ: EPIX), a clinical-stage pharmaceutical company focused on developing novel therapies for the treatment of prostate cancer, today will present preclinical and clinical pharmacology data from ESSA’s Phase 1 clinical trial of EPI-7386 for the treatment of patients with metastatic castration-resistant prostate cancer (“mCRPC”) at the 2021 American Society of Clinical Oncology Genitourinary (“ASCO GU”) Cancers Symposium. EPI-7386, ESSA’s lead product candidate, is an investigational, highly-selective, oral, small molecule inhibitor of the androgen receptor’s Nterminal domain. ASCO GU is being held virtually from Thursday, February 11 to Saturday, February 13, 2021. The oral poster presentation titled, “Preclinical and clinical pharmacology of EPI-7386, an androgen receptor Nterminal domain inhibitor for castration-resistant prostate cancer,” will be presented and available for viewing starting February 11 at 8:00am ET.
The poster is available on the ASCO GU Virtual Symposium and on the “Events & Presentations” section of the Company’s website at www.essapharma.com.
EPI-7386
About EPI-7386 EPI-7386 is an investigational, highly-selective, oral, small molecule inhibitor of the N-terminal domain of the androgen receptor. EPI-7386 is currently being studied in a Phase 1 clinical trial (NCT04421222) in men with metastatic castration-resistant prostate cancer (“mCRPC”) whose tumors have progressed on current standard-ofcare therapies. The Phase I clinical trial of EPI-7386 began in calendar Q3 of 2020 following FDA allowance of the IND and Health Canada acceptance. The U.S. FDA has granted Fast Track designation to EPI-7386 for the treatment of adult male patients with mCRPC resistant to standard-of-care treatment. ESSA retains all rights to EPI-7386 worldwide.
r. For more information, please visit www.essapharma.com
Pipeline
At ESSA, we are focused on the development of small molecule drugs for the treatment of cancer, with an initial focus on advanced prostate cancer
https://www.essapharma.com/pipeline/
ESSA is up after early data for EPI-7386 in prostate cancer
Feb. 11, 2021 2:19 PM ET ESSA Pharma Inc. (EPIX)
By: Dulan Lokuwithana, SA News Editor
- ESSA Pharma (NASDAQ:EPIX) has climbed ~46.9% after the company disclosed the initial Phase 1 data for EPI-7386 for the treatment of patients with metastatic castration-resistant prostate cancer.
https://seekingalpha.com/news/3661349-essa-is-up-after-early-data-for-epi-7386-in-prostate-cancer
https://seekingalpha.com/symbol/EPIX
biotecHOPE™ February/January 2021
Elafibranor
Islatravir (formerly MK-8591)
Islatravir (formerly MK-8591)
GENFIT Announces Publication of Positive Results from the Phase 2 Clinical Trial Evaluating Elafibranor in Patients with PBC in the Journal of Hepatology
February 9, 2021PDF Version
Lille, France; Cambridge, MA; February 09, 2021 - GENFIT (Nasdaq and Euronext: GNFT), a late-stage biopharmaceutical company dedicated to improving the lives of patients with metabolic and liver diseases, today announced that the positive results from the Phase 2 clinical trial evaluating elafibranor in patients with Primary Biliary Cholangitis (PBC) with incomplete response to ursodeoxycholic acid (UDCA) have been published in the Journal of Hepatology.
GENFIT is a publicly traded company listed on the Nasdaq Global Select Market and on compartment B of Euronext’s regulated market in Paris (Nasdaq and Euronext: GNFT). www.genfit.com
Elafibranor
Dr. Jörn Schattenberg, Director Metabolic Liver Research Program, University Medical Center, Mainz, Germany added: “These promising findings along with existing safety data derived from past clinical trials suggest elafibranor is a promising development candidate as a potential novel treatment for patients with PBC. Regulatory authorities know the disease well and there remains an important unmet need to be addressed as many patients at present remain without a long-term therapeutic option.”
Based upon the Phase 2 data, elafibranor was granted Breakthrough Therapy designation by the Food and Drug Administration (FDA), as well as Orphan Drug designation by the FDA and the European Medicines Agency (EMA). In September 2020, GENFIT initiated enrollment of patients in ELATIVE™, a global pivotal Phase 3 clinical trial to evaluate the efficacy and safety of elafibranor in patients with PBC and an inadequate response to UDCA. The randomized study (2:1, elafibranor: placebo) will evaluate approximately 150 patients following 52 weeks of treatment. Topline data are expected in Q1 2023.
Dr. Kris V. Kowdley, Director, Liver Institute Northwest, Clinical Professor Elson S. Floyd College of Medicine, Washington State University added: “These encouraging Phase 2 data are particularly exciting as they highlight a favorable trend in pruritus, which is a debilitating symptom of PBC and one that significantly affects patients’ quality of life. These data suggest that elafibranor is a promising drug candidate, and I’m eager to see whether this trend becomes more significant following longer-term administration, while maintaining the favorable safety/tolerability profile we have seen in the Phase 2 trial.”
Genfit skyrockets 150% on promising elafibranor data in chronic liver disease
Feb. 10, 2021 1:27 AM ETGenfit SA (GNFT)By: Mamta Mayani, SA News Editor2 Comments
- Genefit (NASDAQ:GNFT) soars 150% after-hours on positive results from the Phase 2 trial of elafibranor in patients with Primary Biliary Cholangitis (PBC) with incomplete response to ursodeoxycholic acid (UDCA), which were published in the Journal of Hepatology.
https://seekingalpha.com/symbol/GNFT
https://www.genfit.com/pipeline/elafibranor-in-pbc/
Islatravir (formerly MK-8591)
Islatravir (formerly MK-8591)
Islatravir (formerly MK-8591)
Merck Presents Interim Findings from Phase 2a Clinical Trial Evaluating Investigational Once-Monthly Oral Islatravir for the Prevention of HIV-1 Infection at HIVR4P 2021
January 26, 2021 9:06 am EST
Clinical Trial is Part of the Company’s HIV-1 Prevention Clinical Program Studying Islatravir as a Long-Acting PrEP Agent
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced new interim data from the Phase 2a trial (NCT04003103) in adults evaluating the safety, tolerability and pharmacokinetics (PK) of the once-monthly oral islatravir tablet -- the company’s investigational oral nucleoside reverse transcriptase translocation inhibitor (NRTTI) -- for pre-exposure prophylaxis (PrEP). Interim findings demonstrate that once-monthly oral islatravir achieved the pre-specified efficacy PK threshold for PrEP at both of the two doses studied (60 mg and 120 mg). In the interim analysis using blinded data, both monthly doses of islatravir were found to have an acceptable tolerability profile. These data are shared as a late-breaking oral presentation during the virtual 2021 HIV Research for Prevention Conference (HIVR4P 2021) and featured in the official press conference of HIVR4P 2021.
Oral Islatravir
About Islatravir
Islatravir (formerly MK-8591) is Merck’s investigational nucleoside reverse transcriptase translocation inhibitor (NRTTI) under evaluation in clinical trials for the treatment of HIV-1 infection in combination with other antiretrovirals, including the ILLUMINATE clinical trials program for once-daily treatment, as well as for pre-exposure prophylaxis (PrEP) of HIV-1 infection as a single agent, across a variety of formulations.
To learn more about Merck’s infectious diseases pipeline, visit www.merck.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210126005705/en/
HTX-011 (ZYNRELEF™)*
Islatravir (formerly MK-8591)
HTX-011 (ZYNRELEF™)*
Heron Therapeutics Announces Publication of Results from EPOCH 1 Follow-On Study of HTX-011 in Patients Undergoing Bunionectomy Surgery
- 77% of Bunionectomy Patients Receiving HTX-011 Required No Opioids to Manage Their Postoperative Pain Through 72 Hours After Surgery and Continued To Be Opioid-Free Through 28 Days of Recovery -
SAN DIEGO, Jan. 21, 2021 /PRNewswire/ -- Heron Therapeutics, Inc. (Nasdaq: HRTX
HTX-011 (ZYNRELEF™)*
*ZYNRELEF is the approved trade name for HTX-011 in the European Union.
About HTX-011 for Postoperative Pain (ZYNRELEF in the European Union and European Economic Area)
HTX-011, an investigational non-opioid analgesic, is a dual-acting, fixed-dose combination of the local anesthetic bupivacaine with a low dose of the nonsteroidal anti-inflammatory drug meloxicam. It is the first and only extended-release local anesthetic to demonstrate in Phase 3 studies significantly reduced pain and opioid use through 72 hours compared to bupivacaine solution, the current standard-of-care local anesthetic for postoperative pain control. The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to HTX-011 and the New Drug Application (NDA) received Priority Review designation. A complete response letter (CRL) was received from the FDA regarding the NDA for HTX-011 in June 2020 relating to non‑clinical information. No clinical safety or efficacy issues and no chemistry, manufacturing and controls (CMC) issues were identified. Heron resubmitted an NDA to the FDA for HTX-011 in November 2020 and the FDA set a Prescription Drug User Fee Act goal date of May 12, 2021. Heron is working to respond to a list of questions received from Health Canada in July 2020. In September 2020, the European Commission (EC) granted a marketing authorization for ZYNRELEF (also known as HTX-011) for the treatment of somatic postoperative pain from small- to medium-sized surgical wounds in adults. The EC's centralized marketing authorization is valid for the 27 countries that are members of the European Union, and the other countries in the European Economic Area, including the United Kingdom.
For more information, visit www.herontx.com.
View original content:http://www.prnewswire.com/news-releases/heron-therapeutics-announces-publication-of-results-from-epoch-1-follow-on-study-of-htx-011-in-patients-undergoing-bunionectomy-surgery-301212178.html
Heron Therapeutics Announces Publication of Results from EPOCH 1 Follow-On Study of HTX-011 in Patients Undergoing Bunionectomy Surgery
Thu January 21, 2021 8:30 AM|PR Newswire| About: HRTXPR Newswire
SAN DIEGO, Jan. 21, 2021 /PRNewswire/ -- Heron Therapeutics, Inc. (HRTX),
Heron's HTX-011 long term data published in a medical journal
Jan. 21, 2021 12:19 PM ET Heron Therapeutics, Inc. (HRTX)
By: Vandana Singh, SA News Editor2 Comments
- Heron Therapeutics (HRTX +0.1%) has announced that the results from an EPOCH 1 follow-on study evaluating HTX-011 in bunionectomy (surgery that corrects a deformed area of the foot near the big toe), have been published online, by the Journal of the Ameri can Podiatric Medical Association.
https://seekingalpha.com/news/3653193-herons-htx-011-long-term-data-published-in-a-medical-journal
https://seekingalpha.com/symbol/HRTX
Dostarlimab
NERLYNX® (neratinib)
HTX-011 (ZYNRELEF™)*
16 January 2021
GSK presents positive efficacy data of dostarlimab in mismatch repair-deficient (dMMR) solid cancers at ASCO Gastrointestinal Cancers Symposium
For media and investors only
Issued: London, UK
- Data from GARNET cohort F shows a 38.7% objective response rate with dostarlimab in patients with dMMR advanced solid cancers
- Durable responses across tumour types
GlaxoSmithKline (GSK) plc announced updated data from GARNET cohort F evaluating dostarlimab in mismatch repair-deficient (dMMR) non-endometrial advanced solid cancers being presented today at the 2021 American Society of Clinical Oncology Gastrointestinal Cancers Symposium (ASCO GI). Study results (abstract #9) showed a 38.7% objective response rate (ORR) (N=106, 95% CI; 29.4–48.6) in patients with dMMR advanced solid cancers who received dostarlimab, an investigational anti-programmed death-1 (PD-1) monoclonal antibody. Additionally, after a median follow-up of 12.4 months, the median duration of response (DoR) had not yet been reached, and responses were durable across tumour types.
Dostarlimab
About Dostarlimab
Dostarlimab is a humanised PD-1 monoclonal antibody that binds with high affinity to the PD-1 receptor and blocks its interaction with the ligands PD-L1 and PD-L2.[ii] In addition to GARNET, dostarlimab is being investigated in other registrational enabling studies, including the phase 3 RUBY study for patients with recurrent or primary advanced endometrial cancer in combination with standard of care (SOC) chemotherapy[iii] and the phase 3 FIRST study of platinum-based therapy with dostarlimab and niraparib versus SOC platinum-based therapy as first-line treatment of stage III or IV non-mucinous epithelial ovarian cancer. Dostarlimab is also being evaluated in combination with other therapeutic agents for patients with advanced solid tumours or metastatic cancer.
Dostarlimab was discovered by AnaptysBio and TESARO, Inc. under a Collaboration and Exclusive License Agreement signed in March 2014. The collaboration has resulted in three monospecific antibody drugs that have progressed into the clinic. These are: dostarlimab (GSK4057190), a PD-1 antagonist; cobolimab, (GSK4069889), a TIM-3 antagonist; and GSK4074386, a LAG-3 antagonist. GSK is responsible for the ongoing research, development, commercialisation, and manufacture of each of these products under the Agreement.
A Study of Dostarlimab (TSR-042) Plus Carboplatin-paclitaxel Versus Placebo Plus Carboplatin-paclitaxel in Patients with Recurrent or Primary Advanced Endometrial Cancer (RUBY). https://clinicaltrials.gov/ct2/show/NCT03981796. Accessed December 2020.
For further information please visit www.gsk.com/about-us.
GlaxoSmithKline posts promising data from GARNET study of dostarlimab
Jan. 19, 2021 4:14 PM ET GlaxoSmithKline plc (GSK) By: Aakash Babu, SA News Editor1 Comment
https://seekingalpha.com/symbol/GSK
NUC-3373
NERLYNX® (neratinib)
NERLYNX® (neratinib)
NuCana Presents Encouraging Data at ASCO GI for NUC-3373 in Heavily Pre-Treated Patients with Metastatic Colorectal Cancer
Fri January 15, 2021 8:01 AM|GlobeNewswire|About: NCNA
Promising Efficacy Signals Including a 62% Disease Control Rate and a Partial Response in Patients who had Progressed on Prior Fluoropyrimidine Therapy
Edinburgh: January 2021
NuCana Presents Encouraging Data at ASCO GI for NUC-3373 in Heavily Pre-Treated Patients with Metastatic Colorectal Cancer
READ MORE
http://www.nucana.com/news.html
Safety Profile Continues to be Favorable
EDINBURGH, United Kingdom, Jan. 15, 2021 (GLOBE NEWSWIRE) -- NuCana plc (NCNA) today announced interim data from the ongoing NuTide:302 study at the ASCO GI Conference, being held virtually January 15-17, 2021.
NUC-3373
NuTide:302 is a three-part study investigating NUC-3373, NuCana’s targeted thymidylate synthase inhibitor, in heavily pre-treated patients with metastatic colorectal cancer. The study is evaluating NUC-3373’s optimal dose and schedule in combination with agents commonly used to treat patients with colorectal cancer and is assessing safety, pharmacokinetics and anti-cancer activity. NUC-3373 has been designed to overcome the main challenges associated with 5-FU and capecitabine, including cancer-resistance mechanisms which limit efficacy, off-target toxicity and administration burdens.
http://www.nucana.com/index.html
https://seekingalpha.com/symbol/NCNA
NuCana's NUC-3373 demonstrates encouraging data in metastatic colorectal cancer
Jan. 15, 2021 8:32 AM ET NuCana plc (NCNA)
By: Mamta Mayani, SA News Editor
- NuCana (NASDAQ:NCNA) announces interim data from the ongoing three-part study NuTide:302 investigating NUC-3373, a targeted thymidylate synthase inhibitor, in heavily pre-treated patients with metastatic colorectal cancer.
NERLYNX® (neratinib)
NERLYNX® (neratinib)
NERLYNX® (neratinib)
Puma Biotechnology Presents Interim Results from the Biliary Tract Cancers Cohort of the Phase II SUMMIT “Basket” Trial of Neratinib at ASCO GI
LOS ANGELES, Calif., Jan. 15, 2021 - Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, announced interim results from the biliary tract cancers cohort of the ongoing SUMMIT trial of neratinib at the virtual 2021 Gastrointestinal Cancers Symposium hosted by the American Society of Clinical Oncology (ASCO GI) that is currently taking place. The presentation, entitled, “Targeting HER2 (ERBB2) mutation-positive advanced biliary tract cancers with neratinib: Results from the phase II SUMMIT “basket” trial,” is being presented at a Poster Session by James J. Harding, MD, Regional Director, Early Drug Development, Memorial Sloan Kettering Cancer Center, an investigator of the trial. A copy of this poster presentation is available on the Puma website.
Further information about Puma Biotechnology may be found at www.pumabiotechnology.com.
NERLYNX® (neratinib)
NERLYNX® (neratinib) tablets, for oral use
INDICATIONS AND USAGE: NERLYNX is a kinase inhibitor indicated:
- As a single agent, for the extended adjuvant treatment of adult patients with early-stage HER2-positive breast cancer, to follow adjuvant trastuzumab-based therapy.
- In combination with capecitabine, for the treatment of adult patients with advanced or metastatic HER2-positive breast cancer, who have received two or more prior anti-HER2 based regimens in the metastatic setting.
Please see Full Prescribing Information for additional safety information.
Puma Biotechnology Presents Interim Results from the Biliary Tract Cancers Cohort of the Phase II SUMMIT “Basket” Trial of Neratinib at ASCO GI
Fri January 15, 2021 8:03 AM| Business Wire|About: PBYI
LOS ANGELES--(BUSINESS WIRE)-- Puma Biotechnology, Inc. (PBYI)
Puma Bio's neratinib shows encouraging effect in biliary tract cancers
Jan. 15, 2021 8:51 AM ET Puma Biotechnology, Inc. (PBYI) By: Mamta Mayani, SA News Editor
- Puma Biotechnology (NASDAQ:PBYI) announces interim results from the biliary tract cancers cohort of the ongoing SUMMIT trial of neratinib.
Tivozanib (FOTIVDA®) in Combination with IMFINZI® (durvalumab)
Tivozanib (FOTIVDA®) in Combination with IMFINZI® (durvalumab)
Tivozanib (FOTIVDA®) in Combination with IMFINZI® (durvalumab)
AVEO Oncology Announces Results from Phase 1b Portion of DEDUCTIVE Study of Tivozanib (FOTIVDA®) in Combination with IMFINZI® (durvalumab) in Previously Untreated Metastatic Hepatocellular Carcinoma
Jan 15 | 2021Download PDF
– Data Presented at the 2021 ASCO GI Cancers Symposium –
BOSTON--(BUSINESS WIRE)--Jan. 15, 2021-- AVEO Oncology (Nasdaq: AVEO) today announced the presentation of results from the Phase 1b portion of the Phase 1b/2 DEDUCTIVE clinical trial of tivozanib (FOTIVDA®), AVEO’s vascular endothelial growth factor receptor (VEGFR) tyrosine kinase inhibitor (TKI) drug candidate, in combination with IMFINZI® (durvalumab), AstraZeneca’s (LSE/STO/Nasdaq: AZN) human monoclonal antibody directed against programmed death-ligand 1 (PD-L1), in patients with advanced or metastatic hepatocellular carcinoma (HCC). The results are being presented today in a poster session at the 2021 American Society of Clinical Oncology Gastrointestinal (ASCO GI) Cancers Symposium being held virtually.
Tivozanib (FOTIVDA®) in Combination with IMFINZI® (durvalumab)
About Tivozanib (FOTIVDA®)
Tivozanib is an oral, once-daily, next-generation VEGFR TKI discovered by Kyowa Kirin Co. and approved as FOTIVDA® for the treatment of adult patients with advanced RCC in the European Union and other countries in the territory of the Company’s partner, EUSA Pharma (UK) Limited (EUSA territory). It is a potent, selective and long half-life inhibitor of all three VEGF receptors and is designed to optimize VEGF blockade while minimizing off-target toxicities, potentially resulting in improved efficacy and minimal dose modifications.1,2 Tivozanib is being studied in the TIVO-3 trial, which is supporting a regulatory submission of tivozanib in the U.S. seeking marketing approval as a treatment for adult patients with relapsed or refractory advanced RCC. Tivozanib has been shown to significantly reduce regulatory T-cell production in preclinical models3 and has demonstrated synergy in combination with nivolumab (anti PD-1) in a Phase 2 study in RCC.4 Tivozanib has been investigated in several tumor types, including renal cell, hepatocellular, colorectal, ovarian and breast cancers. Tivozanib is also being studied by partner Kyowa Kirin Co. in non-oncology indications.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210115005128/en/
AVEO Oncology Announces Results from Phase 1b Portion of DEDUCTIVE Study of Tivozanib (FOTIVDA®) in Combination with IMFINZI® (durvalumab) in Previously Untreated Metastatic Hepatocellular Carcinoma
Fri January 15, 2021 8:00 AM| Business Wire|
– Data Presented at the 2021 ASCO GI Cancers Symposium –
BOSTON--(BUSINESS WIRE)-- AVEO Oncology (Nasdaq: AVEO)
Aveo's tivozanib combo demonstrates 29% partial response in liver cancer study
Jan. 15, 2021 8:31 AM ET AVEO Pharmaceuticals, Inc. (AVEO) By: Vandana Singh, SA News Editor
- Aveo Oncology (NASDAQ:AVEO) has announced results from the Phase 1b portion of Phase 1b/2 DEDUCTIVE trial, evaluating tivozanib (Fotivda), in combination with AstraZeneca's Imfinzi (durvalumab), in patients with advanced or metastatic hepatocellular carcinoma. Data were presented at the 2021 American Society of Clinical Oncology Gastrointestinal Cancers Symposium.
https://seekingalpha.com/symbol/AVEO
Onvansertib in Combination With FOLFIRI and Bevacizumab
Tivozanib (FOTIVDA®) in Combination with IMFINZI® (durvalumab)
Tivozanib (FOTIVDA®) in Combination with IMFINZI® (durvalumab)
Cardiff Oncology Presents Data that Continues to Demonstrate the Clinical Benefit of Onvansertib in KRAS-Mutated mCRC and Initial Findings from its Expanded Access Program- Of the 12 Phase 1b patients evaluable for efficacy as of the ASCO-GI data cut-off date (November 1, 2020), 5 (42%) achieved a partial response (PR) and 8 (67%) have shown a durable response ranging from 6.1 to 13.7 months- Since the ASCO-GI data cut-off date, 2 additional Phase 1b patients have had their initial 8-week scan showing stable disease (SD) and both remain on treatment to-date- Time to achieving a PR ranges from 2 to 6 months in patients on treatment- The recommended Phase 2 dose (RP2D) of onvansertib has been established at 15mg/m2 and the Phase 2 segment of the ongoing trial is open to full enrollment of 26 patients across 6 sites in the U.S.- In the Expanded Access Program (EAP), 6 (66%) of the initial 9 patients treated have shown tumor shrinkage and remain on treatment to-date with durable responses lasting an average of 6 months; 5 different KRAS mutation subtypes are represented (G12A, G12C, G12V, G13D, A146T); all patients had received prior treatment with FOLFIRI- Decreases in the KRAS mutational burden in patients after the first cycle of treatment have been predictive of subsequent tumor shrinkage in both the clinical trial and EAP
SAN DIEGO, Jan. 15, 2021 /PRNewswire/ -- Cardiff Oncology, Inc. (Nasdaq: CRDF)
For more information, please visit https://www.cardiffoncology.com.
Onvansertib in Combination With FOLFIRI and Bevacizumab
https://clinicaltrials.gov/ct2/show/NCT03829410
About the Phase 1b/2 Trial of Onvansertib in KRAS-mutated mCRC
This is a multi-center, open-label Phase 1b/2 trial of onvansertib in combination with standard-of-care FOLFIRI and Avastin® (bevacizumab) to evaluate the safety and preliminary efficacy of the combination regimen in the second-line treatment of patients with KRAS-mutated mCRC. The trial, A Phase 1b/2 Study of Onvansertib (PCM-075) in Combination with FOLFIRI and Bevacizumab for Second–Line Treatment of Metastatic Colorectal Cancer in Patients with a KRAS Mutation, will enroll up to 44 patients with a KRAS mutation and histologically confirmed metastatic and unresectable disease. In addition, eligible patients must have failed treatment with, or be intolerant to, FOLFOX (fluoropyrimidine and oxaliplatin) with or without bevacizumab. The trial is being conducted at six cancer centers across the U.S.: USC Norris Comprehensive Cancer Center, The Mayo Clinic (Arizona, Rochester and Jacksonville), Kansas University Medical Center (KUMC) and CARTI Cancer Center. For more information on the trial, please visit https://clinicaltrials.gov/ct2/show/NCT03829410.
Cardiff Oncology reports additional data from onvansertib combo trial in colorectal cancer
Jan. 15, 2021 9:16 AM ETCardiff Oncology, Inc. (CRDF)By: Vandana Singh, SA News Editor5 Comments
- Cardiff Oncology (NASDAQ:CRDF) drops 9% in premarket after announcing additional data from Phase 1b/2 trial and initial findings from its Expanded Access Program (EAP) launched in June 2020, evaluating onvansertib combined with the chemo regimen FOLFIRI and Avastin (bevacizumab), for the second-line treatment of patients with KRAS mutation-positive metastatic colorectal cancer (mCRC). Data were presented at the American Society Clinical Oncology Gastrointestinal Cancers Symposium.
https://seekingalpha.com/symbol/CRDF
https://cardiffoncology.com/pipeline/
Zanidatamab
Tivozanib (FOTIVDA®) in Combination with IMFINZI® (durvalumab)
FGFR2b Overexpression and Reinforce Potential of Bemarituzumab Plus Chemotherapy
Zanidatamab Data Highlight Durable Antitumor Activity in HER2‑Expressing Biliary Tract and Gastroesophageal Cancers at ASCO Gastrointestinal Cancers Symposium
Company Release - 01/15/2021
- Durable Responses Observed in Refractory Biliary Tract Cancer (BTC) and Gastroesophageal (GEA) Cancers
- Enrollment Continues for Zanidatamab Global Pivotal Trial in BTC; Global Pivotal Trial in First-Line GEA Slated to Open in Mid-2021
VANCOUVER, British Columbia--(BUSINESS WIRE)-- Zymeworks Inc. (NYSE: ZYME), a clinical-stage biopharmaceutical company developing multifunctional biotherapeutics, today announced new and updated clinical data for the HER2‑targeted bispecific antibody zanidatamab, in both HER2-expressing biliary tract cancer (BTC) and gastroesophageal adenocarcinoma (GEA). The data are being presented today at the American Society of Clinical Oncology (ASCO) Gastrointestinal Cancers Symposium, taking place virtually January 15 – 17, 2021.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210115005163/en/
Zanidatamab
About Zanidatamab
Zanidatamab is a bispecific antibody, based on Zymeworks’ Azymetric™ platform, that can simultaneously bind two non-overlapping epitopes of HER2, known as biparatopic binding. This unique design results in multiple mechanisms of action including dual HER2 signal blockade, increased binding, and removal of HER2 protein from the cell surface, and potent effector function leading to encouraging antitumor activity in patients. Zymeworks is developing zanidatamab in multiple Phase 1, Phase 2, and pivotal clinical trials globally as a targeted treatment option for patients with solid tumors that express HER2. The FDA has granted Breakthrough Therapy designation for zanidatamab in patients with previously treated HER2 gene-amplified BTC, and two Fast Track designations to zanidatamab, one as a single agent for refractory BTC and one in combination with standard of care chemotherapy, for first-line gastroesophageal adenocarcinoma (GEA). These designations mean zanidatamab is eligible for Accelerated Approval, Priority Review and Rolling Review, as well as intensive FDA guidance on an efficient drug development program. Zanidatamab has also received Orphan Drug designations for the treatment of biliary tract, gastric and ovarian cancers, as well as Orphan Drug designation for the treatment of gastric cancer from the European Medicines Agency.
For additional information about Zymeworks, visit www.zymeworks.com
Zanidatamab Data Highlight Durable Antitumor Activity in HER2‑Expressing Biliary Tract and Gastroesophageal Cancers at ASCO Gastrointestinal Cancers Symposium
Fri January 15, 2021 8:30 AM|Business Wire|About: ZYME
- Durable Responses Observed in Refractory Biliary Tract Cancer (BTC) and Gastroesophageal (GEA) Cancers
- Enrollment Continues for Zanidatamab Global Pivotal Trial in BTC; Global Pivotal Trial in First-Line GEA Slated to Open in Mid-2021
VANCOUVER, British Columbia--(BUSINESS WIRE)-- Zymeworks Inc. (ZYME)
Zymeworks highlights positive data updates from biliary tract cancer study of zanidatamab
Jan. 15, 2021 9:18 AM ETZymeworks Inc. (ZYME)By: Aakash Babu, SA News Editor3 Comments
- Clinical-stage biopharma company Zymeworks (NYSE:ZYME) announces new positive clinical data for the HER2‑targeted bispecific antibody zanidatamab, in HER2-expressing biliary tract cancer (BTC) and gastroesophageal adenocarcinoma (GEA) at the American Society of Clinical Oncology (ASCO) Gastrointestinal Cancers Symposium.
https://seekingalpha.com/symbol/ZYME
FGFR2b Overexpression and Reinforce Potential of Bemarituzumab Plus Chemotherapy
FGFR2b Overexpression and Reinforce Potential of Bemarituzumab Plus Chemotherapy
FGFR2b Overexpression and Reinforce Potential of Bemarituzumab Plus Chemotherapy
Phase 2 FIGHT Trial Results Presented at ASCO GI Validate Importance of FGFR2b Overexpression and Reinforce Potential of Bemarituzumab Plus Chemotherapy as a Frontline Targeted Treatment for FGFR2b+ Gastric and GEJ Cancers
January 15, 2021 at 6:55 AM ESTPDF Version
- All three efficacy endpoints (PFS, OS and ORR) in the FIGHT Phase 2 trial met pre-specified statistical significance
- 2021 priorities for the bemarituzumab program include collaborating with regulatory agencies on next steps, initiating a global Phase 3 trial in gastric and GEJ cancers and evaluating bemarituzumab in other epithelial cancers that overexpress FGFR2b
- Results presented today in a late-breaking oral presentation at the 2021 American Society of Clinical Oncology (ASCO) Gastrointestinal Cancers Virtual Annual Symposium
- Five Prime to host webcast and conference call today at 1:30pm PST / 4:30pm EST
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Jan. 15, 2021-- Five Prime Therapeutics, Inc. (NASDAQ: FPRX)
FGFR2b Overexpression and Reinforce Potential of Bemarituzumab Plus Chemotherapy
About FGFR2b
The fibroblast growth factor (FGF)/fibroblast growth factor receptor (FGFR) pathway is implicated in the development and growth of cancer cells. FGFR2b is a splice form of FGFR which can be found in tumors of epithelial origin. Data from the FIGHT trial suggests that approximately 30 percent of patients with non HER2 positive gastroesophageal cancers overexpress FGFR2b.1 Five Prime and Roche Tissue Diagnostics have also found that FGFR2b is overexpressed in numerous other cancers, including squamous non-small cell lung cancer (NSCLC), triple negative breast cancer (TNBC), ovarian, pancreatic and intrahepatic cholangiocarcinoma.
About Bemarituzumab
Bemarituzumab (anti-FGFR2b) is a first-in-class targeted antibody that blocks fibroblast growth factors (FGFs) from binding and activating FGFR2b, inhibiting several downstream pro-tumor signaling pathways and potentially slowing cancer progression. Bemarituzumab is being developed in gastric and GEJ cancer as a targeted therapy for tumors that overexpress FGFR2b. The company is also evaluating the potential for bemarituzumab in other cancers that overexpress FGFR2b.
Five Prime granted an exclusive license to Zai Lab to develop and commercialize bemarituzumab in Greater China, and Zai Lab collaborated with Five Prime on the Phase 2 FIGHT trial in Greater China.
Bemarituzumab
https://www.fiveprime.com/programs/bemarituzumab/
For more information, please visit www.fiveprime.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210115005136/en/
Phase 2 FIGHT Trial Results Presented at ASCO GI Validate Importance of FGFR2b Overexpression and Reinforce Potential of Bemarituzumab Plus Chemotherapy as a Frontline Targeted Treatment for FGFR2b+ Gastric and GEJ Cancers
Fri January 15, 2021 6:55 AM|Business Wire|About: FPRX
Five Prime's bemarituzumab shows PFS benefit in mid-stage gastric cancer study
Jan. 15, 2021 7:46 AM ET Five Prime Therapeutics, Inc. (FPRX)By: Vandana Singh, SA News Editor1 Comment
- Five Prime Therapeutics (NASDAQ:FPRX) gains 25% in premarket in response to results from a Phase 2 FIGHT trial, evaluating bemarituzumab in advanced gastric or gastroesophageal junction (GEJ) cancer. Results will be presented at the 2021 ASCO Gastrointestinal Cancers Virtual Annual Symposium.
https://seekingalpha.com/symbol/FPRX
VIVITROL® (naltrexone for extended-release injectable suspension)
FGFR2b Overexpression and Reinforce Potential of Bemarituzumab Plus Chemotherapy
VIVITROL® (naltrexone for extended-release injectable suspension)
NIDA-Funded Study Evaluating Extended-Release Injectable Naltrexone Plus Bupropion for the Treatment of Methamphetamine Use Disorder Published in New England Journal of Medicine
DUBLIN, Jan. 14, 2021 /PRNewswire/ -- Results from a National Institute on Drug Abuse (NIDA)-funded study evaluating the efficacy and safety of naltrexone for extended-release injectable suspension (XR-NTX) administered once every three weeks plus oral extended-release bupropion administered daily as a combination treatment for adults with moderate or severe methamphetamine use disorder (MUD) were published today by Dr. Madhukar H. Trivedi et al. in the New England Journal of Medicine (NEJM).1 This is the second published study evaluating this combination regimen for the treatment of MUD.2
VIVITROL® (naltrexone for extended-release injectable suspension)
About VIVITROL
VIVITROL® (naltrexone for extended-release injectable suspension) is a once-monthly medication for the treatment of alcohol dependence and for the prevention of relapse to opioid dependence, following opioid detoxification. Treatment with VIVITROL should be part of a comprehensive management program that includes psychosocial support. For more information, visit www.vivitrol.com.
For more information, please visit Alkermes' website at www.alkermes.com.
Cision View original content to download multimedia:http://www.prnewswire.com/news-releases/nida-funded-study-evaluating-extended-release-injectable-naltrexone-plus-bupropion-for-the-treatment-of-methamphetamine-use-disorder-published-in-new-england-journal-of-medicine-301208061.html
NIDA-Funded Study Evaluating Extended-Release Injectable Naltrexone Plus Bupropion for the Treatment of Methamphetamine Use Disorder Published in New England Journal of Medicine
Thu January 14, 2021 7:00 AM|PR Newswire|About: ALKS
DUBLIN, Jan. 14, 2021 /PRNewswire/ -
Alkermes' naltrexone + bupropion shows promising results in methamphetamine addiction
Jan. 14, 2021 8:38 AM ET Alkermes plc (ALKS)
By: Mamta Mayani, SA News Editor
- Results evaluating the efficacy and safety of Alkermes' (NASDAQ:ALKS) Vivitrol (naltrexone for extended-release injectable suspension (XR-NTX)) plus oral extended-release bupropion combination treatment for adults with moderate or severe methamphetamine use disorder (MUD) were published in the New England Journal of Medicine.
https://seekingalpha.com/symbol/ALKS
VIVITROL® (naltrexone for extended-release injectable suspension) is a once-monthly medication for the treatment of alcohol dependence as well as for the prevention of relapse to opioid dependence, following opioid detoxification. Treatment with VIVITROL should be part of a comprehensive management program that includes psychosocial support.
For more information, visit www.vivitrol.com.
https://www.alkermes.com/products/vivitro
HPN217 FOR TREATMENT OF MULTIPLE MYELOMA
FGFR2b Overexpression and Reinforce Potential of Bemarituzumab Plus Chemotherapy
VIVITROL® (naltrexone for extended-release injectable suspension)
January 13, 2021
HARPOON THERAPEUTICS GRANTED ORPHAN DRUG DESIGNATION FROM FDA FOR HPN217 FOR TREATMENT OF MULTIPLE MYELOMA
SOUTH SAN FRANCISCO, Calif., Jan. 13, 2021 (GLOBE NEWSWIRE) -- Harpoon Therapeutics, Inc. (NASDAQ: HARP), a clinical-stage immunotherapy company developing a novel class of T cell engagers, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for HPN217 for the treatment of multiple myeloma. HPN217, a tri-specific T cell activating recombinant protein construct (TriTAC®) targets B-cell maturation antigen (BCMA), a well-validated antigen expressed on malignant multiple myeloma cells. Harpoon has four drug product candidates in clinical development for the treatment of solid and hematologic malignancies based on its proprietary TriTAC platform.
HPN217 FOR TREATMENT OF MULTIPLE MYELOMA
About the Phase 1/2 Trial for HPN217
In April 2020, Harpoon announced dosing of the first patient with HPN217 (BCMA TriTAC) in a Phase 1/2 clinical trial focused on relapsed/refractory multiple myeloma (RRMM). HPN217 is covered by a global development and option agreement with AbbVie Inc. (NYSE: ABBV). Dose escalation for HPN217 in the Phase 1/2 clinical trial is progressing rapidly. HPN217 has been well tolerated, and no DLTs had been observed as of the most recent December 1, 2020 data cutoff date. A presentation of interim data is anticipated in 2021, with initiation of a dose expansion cohort in the second half of 2021.
For additional information about the trial, please visit clinicaltrials.gov using the identifier NCT04184050.
For additional information about Harpoon Therapeutics, please visit www.harpoontx.com.
Harpoon Therapeutics Granted Orphan Drug Designation from FDA for HPN217 for Treatment of Multiple Myeloma
Wed January 13, 2021 7:30 AM|GlobeNewswire|About: ABBV, HARP
SOUTH SAN FRANCISCO, Calif., Jan. 13, 2021 (GLOBE NEWSWIRE) -- Harpoon Therapeutics, Inc. (HARP),
https://seekingalpha.com/symbol/HARP
Harpoon Therapeutics' HPN217 an orphan drug in U.S. for multiple myeloma
Jan. 13, 2021 7:59 AM ETHarpoon Therapeutics, Inc. (HARP)By: Mamta Mayani, SA News Editor2 Comments
- The FDA has granted Orphan Drug Designation to Harpoon Therapeutics' (NASDAQ:HARP) HPN217 for the treatment of multiple myeloma.
- HPN217 is covered by a global development and option agreement with AbbVie (NYSE:ABBV).
https://www.harpoontx.com/pipeline/
https://seekingalpha.com/symbol/HARP
biotecHOPE™ December 2020
TransConTM C-Type Natriuretic Peptide (CNP)
DTX401, an investigational adeno-associated virus (AAV)
DTX401, an investigational adeno-associated virus (AAV)
VISEN Pharmaceuticals Receives IND Approval to Initiate Phase 2 Clinical Trial of TransCon™ CNP in Achondroplasia (ACH) in China
2021-01-07
-TransConTM C-Type Natriuretic Peptide (CNP), an investigational once-weekly long-acting prodrug of CNP, is ready for Phase 2 Clinical Trial in China-
SHANGHAI, China, January 7, 2021 - VISEN Pharmaceuticals, a biotech company committed to the treatment of endocrine-related diseases, introducing the world’s leading treatment methods and drugs into the China market and hoping to provide more Chinese patients quick access to the world's most advanced and reliable treatment solutions, today announced that the China Center for Drug Evaluation (CDE) of National Medical Products Administration (NMPA) has approved the IND application to conduct a phase 2 clinical trial of TransConTM C-Type Natriuretic Peptide (CNP) for patients with achondroplasia (ACH). The trial is ready to be launched in China as“the ACcomplisH China trial”, in coordination with the ACcomplisH trial, an ongoing phase 2 global clinical trial of TransCon CNP being conducted globally outside of China by Ascendis Pharma.
TransConTM C-Type Natriuretic Peptide (CNP
About TransCon™ Technology
TransCon refers to “transient conjugation.” The proprietary TransCon platform is an innovative technology designed to create new therapies that potentially optimize therapeutic effect, including efficacy, safety and dosing frequency.
TransCon molecules have three components: an unmodified parent drug, an inert carrier that protects it, and a linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from clearance. When injected into the body, physiologic conditions (e.g., pH and temperature) initiate the release of the active, unmodified parent drug in a predictable manner. Because the parent drug is unmodified, its original mode of action may be maintained. TransCon technology is designed to be applied broadly to a protein, peptide or small molecule in multiple therapeutic areas, and to be used systemically or locally.
Phase 2 Clinical Trial of TransCon CNP Sets to Accelerate Research and Development
Preclinical and clinical data have shown that increased CNP can offset the effects of the FGFR3 mutation, thereby promoting bone growth5. However, the half-life of natural CNP in the human body is short, only 2-3 minutes6, requiring continuous intravenous infusion, posing significant difficulties for clinical application.
VISEN Pharmaceuticals Receives IND Approval to Initiate Phase 2 Clinical Trial of TransCon ™ CNP in Achondroplasia (ACH) in China
Thu January 7, 2021 5:45 AM|PR Newswire|About: ASNDPR Newswire
SHANGHAI, Jan. 7, 2021 /PRNewswire/ -- VISEN Pharmaceuticals
Ascendis’ Chinese partner to start Phase 2 achondroplasia trial; closes new financing round
Jan. 09, 2021 4:19 AM ET Ascendis Pharma A/S (ASND) By: Dulan Lokuwithana, SA News Editor
- VISEN Pharmaceuticals, formed as a joint venture of Ascendis Pharma (NASDAQ:ASND), has received the Chinese regulatory signoff to conduct a phase 2 clinical trial of TransCon TM C-Type Natriuretic Peptide (“CNP”) for patients with achondroplasia (“ACH”).
http://www.visenpharma.com/index.php?lang=en
https://seekingalpha.com/symbol/ASND
TRANSCON CNP
Designed for continuous CNP exposure to rebalance bone growth in skeletal disorders
https://ascendispharma.com/pipeline/endocrinology/transcon-cnp/
DTX401, an investigational adeno-associated virus (AAV)
DTX401, an investigational adeno-associated virus (AAV)
DTX401, an investigational adeno-associated virus (AAV)
Press Releases
Jan 8, 2021 PDF Version
Ultragenyx Announces Progress Across Broad Gene Therapy Portfolio and Positive Longer-Term Data from Multiple Phase 1/2 Gene Therapy Studies
Durable and Clinically Meaningful Responses Reported from Phase 1/2 Studies of DTX401 for GSDIa and DTX301 for OTC
Phase 3 Studies for DTX401 and DTX301 to Begin in 2021
IND for UX701 for Wilson Disease Submitted; Expect to Enter Clinic in First Half 2021 using AAV Drug Product Made by HeLa PCL Platform
NOVATO, Calif. , Jan. 08, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE)
DTX401, an investigational adeno-associated virus (AAV)
DTX401 (GSDIa) Program
Phase 1/2 data update: All patients (n=9) responding and demonstrating continued improvement of glucose control while reducing or eliminating cornstarch therapy
All nine patients continue to demonstrate improved glucose control while tapering or discontinuing oral glucose replacement with cornstarch and improvements in energy metabolism pathways over the long term. Patients continue to taper the amount and frequency of cornstarch dosing with progress in eliminating overnight and daytime cornstarch doses. At the primary evaluation timepoint at Week 52, the overall mean reduction in cornstarch was 77% across all three cohorts, including two patients in Cohort 3 showing a reduction of greater than 75%. Longer term follow-up for more than two years for the three patients in Cohort 1 have shown sustained and continued cornstarch reductions with a mean reduction of 91% through weeks 104 and 120. Two patients (one each from Cohort 1 and 3) are completely off cornstarch therapy at weeks 127 and 60, respectively.
For more information on Ultragenyx, please visit the company’s website at www.ultragenyx.com.
https://www.ultragenyx.com/pipeline/
https://seekingalpha.com/symbol/RARE
Ultragenyx's gene therapy shows durable effect in long-term glycogen storage study
Jan. 08, 2021 11:18 AM ET Ultragenyx Pharmaceutical Inc. (RARE) By: Vandana Singh, SA News Editor
PF-06939926
DTX401, an investigational adeno-associated virus (AAV)
AXS-05 (dextromethorphan-bupropion modulated delivery tablet)
PFIZER DOSES FIRST PARTICIPANT IN PHASE 3 STUDY FOR DUCHENNE MUSCULAR DYSTROPHY INVESTIGATIONAL GENE
About CIFFREO
CIFFREO is a Phase 3 global, multi-center, randomized, double-blind, placebo-controlled study to assess the safety and efficacy of PF-06939926 investigational gene therapy in 99 ambulatory male participants, ages 4 through 7 years, with a genetic diagnosis of DMD who are on a stable daily regimen of glucocorticoids. The participants are negative for neutralizing antibodies against AAV9, as measured by the test done for the study as part of screening.
For more information, visit: ciffreoduchennetrial.com.
About PF-06939926
PF-06939926 is an investigational recombinant adeno-associated virus serotype 9 (rAAV9) capsid carrying a shortened version of the human dystrophin gene (mini-dystrophin) under the control of a human muscle-specific promotor. The rAAV9 capsid was chosen as the delivery vector because of its potential to target muscle tissue.
to learn more, please visit us on www.pfizer.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210107005031/en/
https://seekingalpha.com/symbol/PFE
AXS-05 (dextromethorphan-bupropion modulated delivery tablet)
AXS-05 (dextromethorphan-bupropion modulated delivery tablet)
AXS-05 (dextromethorphan-bupropion modulated delivery tablet)
Axsome Therapeutics Initiates ACCORD Phase 3 Trial of AXS-05 in Alzheimer’s Disease Agitation
ACCORD is the second pivotal trial of AXS-05 in Alzheimer’s disease agitation
No treatments are currently approved for Alzheimer’s disease agitation
NEW YORK, Dec. 31, 2020 (GLOBE NEWSWIRE) -- Axsome Therapeutics, Inc. (NASDAQ: AXSM), a biopharmaceutical company developing novel therapies for the management of central nervous system (CNS) disorders, announced the initiation of the ACCORD (Assessing Clinical Outcomes in Alzheimer’s Disease Agitation) study, a Phase 3, randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of AXS-05 in the treatment of Alzheimer’s disease (AD) agitation. AXS-05 (45 mg dextromethorphan-105 mg bupropion modulated delivery tablet) is a novel, oral, investigational NMDA receptor antagonist with multimodal activity. There is currently no approved treatment for AD agitation.
AXS-05 (dextromethorphan-bupropion modulated delivery tablet)
About AXS-05
AXS-05 (dextromethorphan-bupropion modulated delivery tablet) is a novel, oral, patent-protected, investigational NMDA receptor antagonist with multimodal activity under development for the treatment of major depressive disorder and other central nervous system (CNS) disorders. AXS-05 utilizes a proprietary formulation and dose of dextromethorphan and bupropion, and Axsome’s metabolic inhibition technology, to modulate the delivery of the components. The dextromethorphan component of AXS-05 is an uncompetitive N-methyl-D-aspartate (NMDA) receptor antagonist, also known as a glutamate receptor modulator, which is a novel mechanism of action, meaning it works differently than currently approved therapies for major depressive disorder. The dextromethorphan component of AXS-05 is also a sigma-1 receptor agonist, nicotinic acetylcholine receptor antagonist, and inhibitor of the serotonin and norepinephrine transporters. The bupropion component of AXS-05 serves to increase the bioavailability of dextromethorphan, and is a norepinephrine and dopamine reuptake inhibitor, and a nicotinic acetylcholine receptor antagonist. AXS-05 is covered by more than 93 issued U.S. and international patents which provide protection out to 2040. AXS-05 has been granted U.S. Food and Drug Administration Breakthrough Therapy and Fast Track designations for Alzheimer’s disease agitation. AXS-05 is not approved by the FDA.
AXS-05
AXS-05 is a novel, oral, investigational NMDA receptor antagonist with multimodal activity under development for the treatment of CNS disorders. AXS-05 consists of a proprietary formulation and dose of bupropion and dextromethorphan, or DM, and utilizes our metabolic inhibition technology. The DM component of AXS-05 is a non-competitive N-methyl-D-aspartate, or NMDA, receptor antagonist, also known as a glutamate receptor modulator. The DM component of AXS-05 is also a sigma-1 receptor agonist, nicotinic acetylcholine receptor antagonist, and inhibitor of the serotonin and norepinephrine transporters. The bupropion component of AXS-05 serves to increase the bioavailability of dextromethorphan, and is a norepinephrine and dopamine reuptake inhibitor, and a nicotinic acetylcholine receptor antagonist. AXS-05 is an investigational drug not yet approved by the FDA.
https://axsome.com/axs-pipeline/about-axs-05/
For more information, please visit the Company’s website at axsome.com.
https://seekingalpha.com/symbol/AXSM
CABOMETYX® (cabozantinib)
AXS-05 (dextromethorphan-bupropion modulated delivery tablet)
CABOMETYX® (cabozantinib)
Exelixis Announces Cabozantinib Significantly Improved Progression-Free Survival in COSMIC-311 Phase 3 Pivotal Trial in Patients with Previously Treated Radioiodine-Refractory Differentiated Thyroid CancerPDF Version
– Cabozantinib reduced the risk of disease progression or death by 78%; hazard ratio = 0.22, (p<0.0001) compared to placebo –
– Exelixis will discuss the trial results and regulatory filing plans with the U.S. Food and Drug Administration (FDA) –
ALAMEDA, Calif.--(BUSINESS WIRE)--Dec. 21, 2020-- Exelixis, Inc. (NASDAQ: EXEL)
CABOMETYX® (cabozantinib)
About CABOMETYX® (cabozantinib)
In the U.S., CABOMETYX tablets are approved for the treatment of patients with advanced RCC and for the treatment of patients with HCC who have been previously treated with sorafenib. CABOMETYX tablets have also received regulatory approvals in the European Union and additional countries and regions worldwide. In 2016, Exelixis granted Ipsen exclusive rights for the commercialization and further clinical development of cabozantinib outside of the United States and Japan. In 2017, Exelixis granted exclusive rights to Takeda Pharmaceutical Company Limited for the commercialization and further clinical development of cabozantinib for all future indications in Japan. Exelixis holds the exclusive rights to develop and commercialize cabozantinib in the United States.
CABOMETYX is not indicated for radioiodine-refractory differentiated thyroid cancer.
INDICATIONS AND IMPORTANT SAFETY INFORMATION
What is CABOMETYX?
CABOMETYX is a prescription medicine used to treat people with:
- Advanced kidney cancer (renal cell carcinoma)
- Liver cancer (hepatocellular carcinoma) who have been previously treated with the medicine sorafenib.
It is not known if CABOMETYX is safe and effective in children.
Exelixis Announces Cabozantinib Significantly Improved Progression-Free Survival in COSMIC-311 Phase 3 Pivotal Trial in Patients with Previously Treated Radioiodine-Refractory Differentiated Thyroid Cancer
Mon December 21, 2020 7:30 AM|Business Wire|About: EXEL
– Cabozantinib reduced the risk of disease progression or death by 78%; hazard ratio = 0.22, (p<0.0001) compared to placebo –
– Exelixis (EXEL) will discuss the trial results and regulatory filing plans with the U.S. Food and Drug Administration (FDA) –
ALAMEDA, Calif.--(BUSINESS WIRE)-- Exelixis, Inc. (NASDAQ: EXEL)
https://seekingalpha.com/symbol/EXEL
View source version on businesswire.com: https://www.businesswire.com/news/home/20201221005168/en/
Exelixis' cabozantinib shows positive effect in late-stage thyroid cancer study
Dec. 21, 2020 8:56 AM ETExelixis, Inc. (EXEL)By: Vandana Singh, SA News Editor3 Comments
- Exelixis (NASDAQ:EXEL)
ALVR106
AXS-05 (dextromethorphan-bupropion modulated delivery tablet)
CABOMETYX® (cabozantinib)
AlloVir Announces FDA Clearance of Investigational New Drug Application for ALVR106, an Allogeneic, Off-the-Shelf, Multi-Virus Specific T Cell Therapy Targeting Four Devastating Respiratory Viruses
Proof-of-concept phase 1/2 trial to initiate in 2021 to treat severe respiratory viral infections in patients following hematopoietic stem cell transplantation
ALVR106 designed to target devastating diseases caused by four respiratory viruses: respiratory syncytial virus, influenza, parainfluenza virus, and human metapneumovirus
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Dec. 17, 2020-- AlloVir (Nasdaq: ALVR), a late clinical-stage cell therapy company, announced that the U.S. Food and Drug Administration (FDA) has cleared the Investigational New Drug (IND) application for ALVR106, an allogeneic, off-the-shelf virus-specific T cell therapy (VST) designed to target infections and diseases caused by respiratory syncytial virus (RSV), influenza, parainfluenza virus (PIV), and human metapneumovirus (hMPV). The IND enables AlloVir to initiate a Phase 1/2 proof-of-concept clinical study in allogeneic and autologous hematopoietic stem cell transplant (HSCT) patients with respiratory infections caused by RSV, influenza, PIV or hMPV.
ALVR106
About ALVR106
ALVR106 is an allogeneic, off-the-shelf, multi-virus specific VST investigational therapy designed to target infections and diseases caused by the respiratory syncytial virus (RSV), influenza, parainfluenza virus (PIV), and human metapneumovirus (hMPV). In vitro data demonstrate that ALVR106 has antiviral activity against each of the targeted viruses with minimal or no activity against non-virus-infected cells. This preclinical data supports the potential for antiviral benefit and safety of ALVR106 when administered to patients.
For more information visit www.allovir.com.
https://seekingalpha.com/symbol/ALVR
FDA clears AlloVir's ALVR106 application in respiratory viruses
Dec. 17, 2020 7:48 AM ETAlloVir, Inc. (ALVR)By: Mamta Mayani, SA News Editor
https://seekingalpha.com/news/3645249-fda-clears-allovirs-alvr106-application-in-respiratory-viruses
Nurtec ODT (rimegepant)
Motixafortide (BL-8040)
Motixafortide (BL-8040)
BIOHAVEN'S RIMEGEPANT FOR PREVENTIVE TREATMENT OF MIGRAINE PUBLISHED IN THE LANCET
12/16/2020- Rimegepant 75 mg, already approved for the acute treatment of migraine, demonstrated efficacy and safety for the preventive treatment of migraine in this pivotal Phase 3 trial in adults- Rimegepant resulted in a -4.3 day reduction in the mean number of migraine days per month- Approximately half of rimegepant-treated patients demonstrated a 50% or greater reduction in the number of moderate-to-severe migraine days per month- Rimegepant is the first and only CGRP targeting medication in development to show dual efficacy for both the acute and preventive treatment of migraine
NEW HAVEN, Conn., Dec. 16, 2020 /PRNewswire/ --- Biohaven Pharmaceutical Holding Company Ltd. (NYSE: BHVN) announced today that The Lancet published the company's positive results from a Phase 3 clinical trial of rimegepant for the preventive treatment of migraine in adults. These data show rimegepant was superior to placebo in the reduction of monthly migraine days. Rimegepant was approved by the U.S. Food and Drug Administration (FDA) for the acute treatment of migraine in February 2020. Rimegepant is marketed as NURTEC® ODT, and is the first and only calcitonin gene-related peptide (CGRP) receptor antagonist available in an orally disintegrating tablet (ODT) designed for rapid onset of action.
Nurtec ODT (rimegepant)
RIMEGEPANT
Biohaven's Nurtec ODT (rimegepant) Received FDA Approval in February 2020. Nurtec ODT is the first and only CGRP receptor antagonist available in an orally disintegrating tablet (ODT) for the acute treatment of migraine, and the only oral CGRP receptor antagonist with product label claims including return to normal functioning and sustained durability of efficacy up to 48 hours after a single dose. – Read the Full Press Release
If you are a patient, caregiver, or healthcare provider with a question regarding Nurtec ODT, please click here.
Visit Nurtec.com for more information.
https://www.biohavenpharma.com/science-pipeline/cgrp/rimegepant
Indication
NURTEC ODT is indicated for the acute treatment of migraine with or without aura in adults.
Please click here for full Prescribing information and Patient Information.
WHAT IS NURTEC ODT?
Nurtec ODT (rimegepant) orally disintegrating tablets is a prescription medicine for the acute treatment of migraine attacks with or without aura in adults. Nurtec ODT is not used as a preventive treatment of migraine. It is not known if Nurtec ODT is safe and effective in children.
More information about Biohaven is available at www.biohavenpharma.com.
https://seekingalpha.com/news/3644824-biohavens-rimegepant-effective-in-migraine-prevention
https://seekingalpha.com/symbol/BHVN
Motixafortide (BL-8040)
Motixafortide (BL-8040)
Motixafortide (BL-8040)
BioLineRx Announces Final Results from Phase 2a COMBAT/KEYNOTE-202 Triple Combination Study of Motixafortide in Second Line Metastatic Pancreatic Cancer (PDAC)
December 16, 2020
PDF Version- Substantial improvement observed across all study endpoints, including overall survival, progression free survival and overall response rate, in the most challenging PDAC patients -- Company plans to meet with regulatory authorities as it evaluates next development steps -- Company to host Key Opinion Leader (KOL) webinar to discuss these results today, December 16, at 8:00 am EST; registration link below -
TEL AVIV, Israel, Dec. 16, 2020 /PRNewswire/ -- BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a late clinical-stage biopharmaceutical Company focused on oncology, today announced results from the triple combination arm of the Company's COMBAT/KEYNOTE-202 clinical study evaluating motixafortide (BL-8040) in combination with KEYTRUDA® (pembrolizumab) and chemotherapy in patients with second-line stage IV pancreatic ductal adenocarcinoma (PDAC).
Motixafortide
About Motixafortide in Cancer Immunotherapy
Motixafortide is targeting CXCR4, a chemokine receptor and a well validated therapeutic target that is over-expressed in many human cancers including PDAC. CXCR4 plays a key role in tumor growth, invasion, angiogenesis, metastasis and therapeutic resistance, and CXCR4 overexpression has been shown to be correlated with poor prognosis.
Motixafortide is a short synthetic peptide used as a platform for cancer immunotherapy with unique features allowing it to function as a best-in-class antagonist of CXCR4. It shows high-affinity, long receptor occupancy and acts as an inverse agonist.
In a number of clinical and preclinical studies, motixafortide has been shown to affect multiple modes of action in 'cold' tumors, including immune cell trafficking, tumor infiltration by immune effector T cells, and reduction in immunosuppressive cells (such as MDSCs) within the tumor microenvironment, turning 'cold' tumors, such as pancreatic cancer, into "hot" (i.e., sensitizing them to immune checkpoint inhibitors and chemotherapy).
For additional information on BioLineRx, please visit the Company's website at www.biolinerx.com
BioLineRx Announces Final Results from Phase 2a COMBAT/KEYNOTE-202 Triple Combination Study of Motixafortide in Second Line Metastatic Pancreatic Cancer (PDAC)
Wed December 16, 2020 6:01 AM|PR Newswire|About: BLRX
- Company plans to meet with regulatory authorities as it evaluates next development steps -
- Company to host Key Opinion Leader (KOL) webinar to discuss these results today, December 16, at 8:00 am EST; registration link below -
PR Newswire
TEL AVIV, Israel, Dec. 16, 2020 /PRNewswire/ -- BioLineRx Ltd. (BLRX) (TASE: BLRX)
https://seekingalpha.com/symbol/BLRX
BioLineRx jumps 22% on promising triple combination data of motixafortide in pancreatic cancer
Dec. 16, 2020 6:30 AM ET
BioLineRx Ltd. (BLRX)By: Mamta Mayani, SA News Editor5 Comments
- BioLineRx (NASDAQ:BLRX) gains 22% premarket after announcing results from its COMBAT/KEYNOTE-202 clinical study evaluating motixafortide (BL-8040) in combination with Merck's (NYSE:MRK) KEYTRUDA (pembrolizumab) and chemotherapy in patients with second-line stage IV pancreatic ductal adenocarcinoma (PDAC).
CUTX-101
Motixafortide (BL-8040)
Trilaciclib in Combination with Chemotherapy
Fortress Biotech Announces Breakthrough Therapy Designation for CUTX-101, Copper Histidinate, for the Treatment of Menkes Disease
Download as PDFDecember 15, 2020
Rolling submission of New Drug Application to the FDA for CUTX-101 on track to begin in the first quarter of 2021 and to be completed by the end of the second quarter of 2021
NEW YORK, Dec. 15, 2020 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (Nasdaq: FBIO)
CUTX-101
About CUTX-101 (Copper Histidinate)
CUTX-101 is in clinical development to treat patients with Menkes disease by replenishing Copper Histidinate, restoring copper homeostasis and maintaining serum copper levels in the normal age appropriate range. CUTX-101 is a subcutaneous injectable formulation of Copper Histidinate manufactured under current good manufacturing practice (“cGMP”) and physiological pH that bypasses the defect in absorption of orally administered copper in patients with Menkes disease. In a Phase 1/2 clinical trial conducted by Stephen G. Kaler, M.D., M.P.H., at the National Institutes of Health (“NIH”), early treatment of patients with Menkes disease with CUTX-101 led to an improvement in neurodevelopmental outcomes and survival. A Phase 3 trial of CUTX-101 in patients with Menkes disease also led by Dr. Kaler has completed enrollment. In August 2020, Cyprium reported positive topline clinical efficacy results for CUTX-101, demonstrating statistically significant improvement in overall survival for Menkes disease subjects who received early treatment (ET) with CUTX-101, compared to an untreated historical control (HC) cohort, with a nearly 80% reduction in the risk of death. A Cyprium-sponsored expanded access protocol for patients with Menkes disease is ongoing at Nationwide Children’s Hospital (https://www.nationwidechildrens.org/specialties/menkes-disease-clinic) and other US medical centers.
Cyprium was founded by Fortress Biotech, Inc. (Nasdaq: FBIO) and is based in New York City. For more information, visit www.cypriumtx.com.
For more information, visit www.fortressbiotech.com.
https://www.fortressbiotech.com/programs
Fortress Biotech Announces Breakthrough Therapy Designation for CUTX-101, Copper Histidinate, for the Treatment of Menkes Disease
Tue December 15, 2020 8:00 AM|GlobeNewswire|About: FBIOGlobeNewswire
NEW YORK, Dec. 15, 2020 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (FBIO)
https://seekingalpha.com/symbol/FBIO
Trilaciclib in Combination with Chemotherapy
Trilaciclib in Combination with Chemotherapy
Trilaciclib in Combination with Chemotherapy
G1 Therapeutics Presents Final Phase 2 Clinical Data on Trilaciclib in Combination with Chemotherapy in Metastatic Triple-Negative Breast Cancer Demonstrating Significant Improvement in Overall Survival at 2020 San Antonio Breast Cancer Symposium
Wed December 9, 2020 5:00 PM|GlobeNewswire|About: GTHXGlobeNewswire
RESEARCH TRIANGLE PARK, N.C., Dec. 09, 2020 (GLOBE NEWSWIRE) -- G1 Therapeutics, Inc. (GTHX) (Nasdaq: GTHX)
Trilaciclib in Combination with Chemotherapy
Trilaciclib is a first-in-class FDA-designated "Breakthrough Therapy" designed to improve outcomes for people with cancer who are treated with chemotherapy. Positive data have been reported from four randomized trials – three in small cell lung cancer (SCLC) and one in metastatic triple-negative breast cancer (mTNBC). The FDA accepted our New Drug Application (NDA) for trilaciclib for SCLC patients being treated with chemotherapy and granted Priority Review in August 2020 with a Prescription Drug User Fee Act (PDUFA) action date of February 15, 2021.
https://www.g1therapeutics.com/pipeline/trilaciclib/
G1 Therapeutics is based in Research Triangle Park, N.C. For additional information, please visit www.g1therapeutics.com
G1 is advancing two novel therapies for people living with cancer. Trilaciclib is a first-in-class investigational therapy designed to improve outcomes for people with cancer who are treated with chemotherapy. Rintodestrant is a potential best-in-class oral selective estrogen receptor degrader, or SERD, for the treatment of ER+ breast cancer. In 2020, G1 out-licensed global development and commercialization rights to its differentiated oral CDK4/6 inhibitor, lerociclib.
https://www.g1therapeutics.com/science/pipeline/
Yselty® (linzagolix)
Trilaciclib in Combination with Chemotherapy
Yselty® (linzagolix)
ObsEva Announces Additional Phase 3 PRIMROSE 1 and 2 Study Results Confirming Sustained Efficacy and Continued Safety of linzagolix in the Treatment of Uterine Fibroids
Thu December 10, 2020 1:00 AM|GlobeNewswire|About: OBSV
- PRIMROSE 1 52-week results confirm the sustained efficacy and continued safety of linzagolix (Yselty®), with a potential best-in-class high-dose option (200 mg with add-back therapy [ABT])
- Show the unique low-dose option (100 mg without ABT) has the potential to address the needs of up to 50% of U.S. women with uterine fibroids for whom ABT may be contraindicated
- Support the potential value of a high-dose non-ABT option when rapid and substantial volume reduction are required
- PRIMROSE 2 76-week results show continued pain reduction and demonstrate evidence of bone mineral density (BMD) recovery following 52 weeks of treatment
GENEVA, Switzerland and BOSTON, MA (December 10, 2020) – ObsEva SA (OBSV)
Yselty® (linzagolix)
About Linzagolix
Yselty® (linzagolix) is a novel, once daily, oral GnRH receptor antagonist with a potentially best-in-class profile. Linzagolix is currently in late-stage clinical development for the treatment of heavy menstrual bleeding associated with uterine fibroids and pain associated with endometriosis. ObsEva licensed linzagolix from Kissei in late 2015 and retains worldwide commercial rights, excluding Asia, for the product. Linzagolix is not currently approved anywhere in the world.
For more information, please visit www.ObsEva.com.
https://seekingalpha.com/symbol/OBSV
https://www.obseva.com/linzagolix/
ObsEva Announces Additional Phase 3 PRIMROSE 1 and 2 Study Results Confirming Sustained Efficacy and Continued Safety of linzagolix in the Treatment of Uterine Fibroids
December 10, 2020
PRIMROSE 1 52-week results confirm the sustained efficacy and continued safety of linzagolix (Yselty®), with a potential best-in-class high-dose option…
Magrolimab
Trilaciclib in Combination with Chemotherapy
Yselty® (linzagolix)
December 06, 2020
Magrolimab Demonstrates Clinical Responses in Ongoing Phase 1b Trial of Previously Untreated Acute Myeloid Leukemia Patients
-- Similar Overall and Complete Response Rates Observed Across Frontline AML Patients Ineligible for Intensive Chemotherapy and in High Unmet Need Patients with TP53-Mutant AML --
FOSTER CITY, Calif.--(BUSINESS WIRE)-- Gilead Sciences, Inc. (Nasdaq: GILD) today announced updated results from the magrolimab Phase 1b trial. Magrolimab is an investigational, potential first-in-class, anti-CD47 monoclonal antibody being studied in previously untreated acute myeloid leukemia (AML) patients who are ineligible for intensive chemotherapy, including patients with TP53-mutant AML. The study continues to demonstrate high response rates with magrolimab in combination with azacitidine, with an overall response rate of 63% (n=27/43) among the total patient population and 69% (n=20/29) in TP53-mutant patients. The data were presented at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition (Abstract #330).
Magrolimab
About Magrolimab
Magrolimab is a potential, first-in-class investigational monoclonal antibody against CD47 and a macrophage checkpoint inhibitor that is designed to interfere with recognition of CD47 by the SIRPα receptor on macrophages, thus blocking the "don't eat me" signal used by cancer cells to avoid being ingested by macrophages. Magrolimab is being developed in several hematologic cancers, including myelodysplastic syndrome (MDS), as well as solid tumor malignancies.
For more information on Gilead Sciences, please visit the company’s website at www.gilead.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20201206005027/en/
https://seekingalpha.com/symbol/GILD
biotecHOPE™ November 2020
CTX001
Atuzaginstat (COR388)
Atuzaginstat (COR388)
CRISPR Therapeutics and Vertex Present New Data for Investigational CRISPR/Cas9 Gene-Editing Therapy, CTX001™ at American Society of Hematology Annual Meeting and Exposition, Together With Publication in the New England Journal of Medicine
Sat December 5, 2020 12:30 PM|GlobeNewswire|About: CRSP, VRTX
- Beta thalassemia: All seven patients were transfusion independent with 3 to 18 months of follow-up after CTX001 infusion -
- Sickle cell disease: All three patients were free of vaso-occlusive crises with 3 to 15 months of follow-up after CTX001 infusion -
- Nineteen patients have been dosed with CTX001 across both programs -
- The New England Journal of Medicine publishes CTX001 manuscript containing the first report of investigational use of CRISPR/Cas9-based gene editing to treat inherited diseases in humans -
ZUG, Switzerland and CAMBRIDGE, Mass. and BOSTON, Dec. 05, 2020 (GLOBE NEWSWIRE) -- CRISPR Therapeutics (CRSP) (Nasdaq: CRSP) and Vertex Pharmaceuticals Incorporated (VRTX) (Nasdaq: VRTX)
CTX001
About CTX001
CTX001 is an investigational, autologous, ex vivo CRISPR/Cas9 gene-edited therapy that is being evaluated for patients suffering from TDT or severe SCD, in which a patient’s hematopoietic stem cells are edited to produce high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is a form of the oxygen-carrying hemoglobin that is naturally present at birth, which then switches to the adult form of hemoglobin. The elevation of HbF by CTX001 has the potential to alleviate transfusion requirements for patients with TDT and reduce painful and debilitating sickle crises for patients with SCD.
http://www.crisprtx.com/programs/pipeline
DEC 05, 2020
For more information, please visit www.crisprtx.com.
https://seekingalpha.com/symbol/CRSP
For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com
https://seekingalpha.com/symbol/VRTX
https://www.vrtx.com/research-development/pipeline/
We are investigating the use of genetic therapies aimed at the underlying cause of SCD. The cause of SCD has been known since Linus Pauling described the "first molecular disease" in 1949, yet many people still don't have a treatment to address the underlying cause of their disease. With the discovery of tools like CRISPR gene editing, we now potentially have an opportunity to address diseases at their root cause.
We are collaborating with CRISPR Therapeutics to investigate the use of gene-editing technology, known as CRISPR-Cas9, to discover and develop a new one-time treatment for SCD. CTX001 is an investigational ex-vivo CRISPR gene-edited therapy, which aims to edit a person’s hematopoietic stem cells to produce fetal hemoglobin (HbF; hemoglobin F) in red blood cells. The aim of using the body's own machinery to switch red blood cells back to fetal hemoglobin production is a significant reduction or elimination of symptoms associated with the disease.
Atuzaginstat (COR388)
Atuzaginstat (COR388)
Atuzaginstat (COR388)
Cortexyme’s Phase 2/3 GAIN Trial of Atuzaginstat (COR388) in Patients with Alzheimer’s Disease Successfully Advances Past Interim Analysis
Fri December 4, 2020 7:00 AM|Business Wire|About: CRTX
-- GAIN Trial passes futility analysis and will continue to 1-year endpoint following the independent Data Monitoring Committee recommendation; topline results expected on time in December 2021 --
-- Final study enrollment remains at 643; no sample size adjustment --
-- Conference call and webcast today, Friday, December 4, 2020, at 8:30 a.m. EST / 5:30 a.m. PST --
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Cortexyme, Inc. (CRTX),
Atuzaginstat (COR388)
Atuzaginstat targets the toxic proteases, or gingipains, produced by P. gingivalis, which have been discovered in greater than 90% of AD patients and shown to produce Alzheimer’s pathology and neurodegeneration in infected animals. P. gingivalis is best known as a keystone bacterium in the development of periodontal disease.
https://www.cortexyme.com/pipeline/
To learn more about Cortexyme, visit www.cortexyme.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20201204005235/en/
https://seekingalpha.com/symbol/CRTX
Late-stage study of Cortexyme's Alzheimer's med atuzaginstat passes interim tollgate
Dec. 04, 2020 7:17 AM ET Cortexyme, Inc. (CRTX)By: Douglas W. House, SA News Editor
Cortexyme’s Phase 2/3 GAIN Trial of Atuzaginstat (COR388) in Patients with Alzheimer’s Disease Successfully Advances Past Interim Analysis
Amivantamab
Atuzaginstat (COR388)
Amivantamab
Janssen Submits Application to U.S. FDA Seeking Approval of Amivantamab for the Treatment of Patients with Metastatic Non-Small Cell Lung Cancer with EGFR Exon 20 Insertion Mutations
Thu December 3, 2020 7:00 AM|PR NewswirePR Newswire
RARITAN, N.J., Dec. 3, 2020 /PRNewswire/ -- The Janssen Pharmaceutical Companies of Johnson & Johnson
Amivantamab
About Amivantamab
Amivantamab is an investigational, fully-human EGFR-MET bispecific antibody with immune cell-directing activity that targets tumors with activating and resistance EGFR mutations and MET mutations and amplifications.3,4,5,6 Amivantamab is pending regulatory review as a potential treatment for patients with NSCLC with EGFR exon 20 insertion mutations whose tumors typically do not respond to current standard of care. Companion diagnostics to identify patients with EGFR exon 20 insertion mutations have been an integral part of the development program for amivantamab. The production and development of the antibody followed Janssen Biotech, Inc.'s licensing agreement with Genmab for use of its DuoBody® technology platform.
Additional information about the expanded access protocol can be found on clinicaltrials.gov (NCT04599712) and at https://www.janssen.com/compassionate-use-pre-approval-access.
Learn more at www.janssen.com.
View original content:http://www.prnewswire.com/news-releases/janssen-submits-application-to-us-fda-seeking-approval-of-amivantamab-for-the-treatment-of-patients-with-metastatic-non-small-cell-lung-cancer-with-egfr-exon-20-insertion-mutations-301185216.html
SOURCE The Janssen Pharmaceutical Companies of Johnson & Johnson
https://seekingalpha.com/symbol/JNJ
Mitapivat
Dosing underway in study of Molecular Partners and Novartis COVID-19 candidate, MP0420
Amivantamab
Agios Announces the Phase 3 ACTIVATE Trial of Mitapivat Achieved Its Primary Endpoint in Adults with Pyruvate Kinase Deficiency Who Are Not Regularly Transfused
Tue December 1, 2020 7:00 AM|GlobeNewswire|About: AGIO
– 40 Percent of Patients Treated with Mitapivat Achieved a Sustained Hemoglobin Increase of ≥1.5 g/dL Compared to 0 Placebo Patients (p<0.0001) –
– Safety Profile Consistent with Previously Reported Data –
– Topline Data from the Mitapivat Phase 3 ACTIVATE-T Trial in Regularly Transfused PK Deficiency Expected in Q1 2021 –
CAMBRIDGE, Mass., Dec. 01, 2020 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (AGIO)
Mitapivat
Mitapivat Clinical Development
ACTIVATE is one of two studies intended to support a marketing application for mitapivat in patients with PK deficiency. In addition to the ACTIVATE trial, Agios has fully enrolled the global, pivotal Phase 3 ACTIVATE-T trial in adults with PK deficiency who receive regular transfusions. The primary endpoint of this single-arm trial is the proportion of patients who achieve a reduction in transfusion burden compared to individual historical transfusion burden standardized to 24 weeks. Agios anticipates reporting topline ACTIVATE-T data in Q1 2021. Agios is also enrolling an extension study for adults with PK deficiency previously enrolled in ACTIVATE or ACTIVATE-T, which is designed to evaluate the long-term safety, tolerability and efficacy of treatment with mitapivat.
Multimedia Assets: Rare Diseases at Agios, Mitapivat Development, PK Deficiency Patient Testimonials
There are no currently approved therapies for PK deficiency. For more information, please visit www.knowpkdeficiency.com.
For more information, please visit the company's website at www.agios.com.
https://seekingalpha.com/symbol/AGIO
https://www.agios.com/pipeline/overview/
Dec 1, 2020
PR006
Dosing underway in study of Molecular Partners and Novartis COVID-19 candidate, MP0420
Dosing underway in study of Molecular Partners and Novartis COVID-19 candidate, MP0420
Prevail Therapeutics Receives European Commission Orphan Designation for PR006 for the Treatment of Frontotemporal Dementia
Mon November 30, 2020 7:00 AM | GlobeNewswire | About: PRVL
NEW YORK, Nov. 30, 2020 (GLOBE NEWSWIRE) -- Prevail Therapeutics Inc. (PRVL) (Nasdaq: PRVL)
PR006
Supporting Publications: PR006
https://www.prevailtherapeutics.com/our-science/#overview
https://www.prevailtherapeutics.com/
NOVEMBER 30, 2020
Prevail Therapeutics Receives European Commission Orphan Designation for PR006 for the Treatment of Frontotemporal Dementia
https://seekingalpha.com/symbol/PRVL
Prevail's gene therapy Orphan drug in Europe for a type of dementia
Nov. 30, 2020 10:54 AM ET Prevail Therapeutics Inc. (PRVL)By: Vandana Singh, SA News Editor
Dosing underway in study of Molecular Partners and Novartis COVID-19 candidate, MP0420
Dosing underway in study of Molecular Partners and Novartis COVID-19 candidate, MP0420
Dosing underway in study of Molecular Partners and Novartis COVID-19 candidate, MP0420
Novartis announces collaboration with Molecular Partners to develop two DARPin® therapies designed for potential use against COVID-19
Oct 28, 2020
- Novartis has been granted an option to in-license global rights of MP0420 and MP0423 - multi-targeted direct acting antiviral therapeutic candidates demonstrating potential efficacy against COVID-19
- MP0420 and MP0423 are potential medicines with a unique approach for both the prevention and treatment of COVID-19, with the possibility to manufacture at scale, easy administration and with the potential to bypass cold storage
- Switzerland based Molecular Partners, a global leader in the development of DARPin® therapeutics, will be responsible for the conduct of phase 1 & 2 trials that may lead to emergency use approval; Novartis will be responsible for further development, manufacturing, distribution and commercialization
- This collaboration strengthens Novartis ongoing commitment to research and partner with other companies to find and develop treatment options for COVID-19 and make them available around the world as fast as possible
Basel, October 28, 2020 — Novartis and Molecular Partners AG
More information about the Novartis response to COVID-19 is available at https://novartis.com/coronavirus
https://www.molecularpartners.com/
Find out more at
https://www.novartis.com.
DARPin®-based approach to address the overwhelming need for effective therapeutics against COVID-19.
https://www.molecularpartners.com/our-darpin-platform/
PIPELINE
Our DARPin® engine is an unlimited source of novel therapeutic designs. Explore our pipeline:
https://www.molecularpartners.com/pipeline/
Molecular Partners Doses First Cohort in Phase 1 Trial of COVID-19 DARPin(R) Therapeutic Candidate MP0420
Mon November 23, 2020 1:00 AM|Accesswire
ZURICH-SCHLIEREN, SWITZERLAND / ACCESSWIRE / November 23, 2020 / Molecular Partners AG (MLLCF) (SIX:MOLN),
https://seekingalpha.com/symbol/MLLCF
https://seekingalpha.com/symbol/NVS
Lenacapavir
Pegcetacoplan (APL-2)
Lenacapavir
Gilead Announces Investigational Long-Acting HIV-1 Capsid Inhibitor, Lenacapavir, Achieves Primary Endpoint in Phase 2/3 Study in Heavily Treatment-Experienced People Living With HIV
Wed November 18, 2020 8:35 AM|Business Wire|About: GILD
– CAPELLA Trial Results Support Further Study of Lenacapavir in People with Multidrug Resistant HIV-1 Infection Who are Failing Current Treatment Regimen –
FOSTER CITY, Calif.--(BUSINESS WIRE)-- Gilead Sciences, Inc. (GILD)
Lenacapavir
About Lenacapavir
Lenacapavir acts in a novel way compared with currently available antiretroviral agents by interrupting the activity of HIV capsid, a protein that surrounds and protects the virus’ genetic material and essential enzymes. In in vitro studies, lenacapavir interrupts multiple distinct stages of the viral lifecycle, potentially preventing the virus from becoming infectious and gaining access to uninfected cells.
For more information about Gilead, please visit the company’s website at www.gilead.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20201118005679/en/
https://seekingalpha.com/symbol/GILD
Gilead's lenacapavir meets primary endpoint in mid-stage HIV study
Nov. 18, 2020 9:14 AM ETGilead Sciences, Inc. (GILD)By: Vandana Singh, SA News Editor
VTX-801
Pegcetacoplan (APL-2)
Lenacapavir
Vivet Therapeutics and Pfizer Inc. Announce FDA Authorization to Proceed with GATEWAY, the Phase 1/2 Study for VTX-801, Vivet’s Investigational Gene Therapy for Wilson Disease
Wed November 18, 2020 1:00 AM|GlobeNewswire
Vivet Therapeutics and Pfizer Inc. Announce FDA Authorization to Proceed with GATEWAY, the Phase 1/2 Study for VTX-801, Vivet’s Investigational Gene Therapy for Wilson Disease
PARIS, France and NEW YORK, N.Y.—November 18, 2020— Vivet Therapeutics
VTX-801
VTX-801 is a novel, investigational rAAV-based gene therapy vector designed to deliver a miniaturized ATP7B transgene encoding, a functional protein that has been shown to restore copper homeostasis, reverse liver pathology and reduce copper accumulation in the brain of a mouse model of Wilson disease. VTX-801’s rAAV serotype was selected based on its demonstrated tropism for transducing human liver cells.
About GATEWAY - Phase 1/2 Clinical Trial of VTX-801 in Wilson disease
The GATEWAY trial is a multi-center, non-randomized, open-label, Phase 1/2 clinical trial designed to assess the safety, tolerability and pharmacological activity of a single intravenous infusion of VTX-801 in adult patients with Wilson disease, prior to and following background WD therapy withdrawal.
https://clinicaltrials.gov/ct2/show/NCT04537377?term=VIVET&draw=2&rank=1
Please visit us on www.vivet-therapeutics.com
In addition, to learn more, please visit us on www.Pfizer.com
https://www.vivet-therapeutics.com/en/pipeline
VIVET AND PFIZER INC. ANNOUNCE FDA AUTHORIZATION TO PROCEED WITH GATEWAY, THE PHASE 1/2 STUDY FOR VTX-801, VIVET’S INVESTIGATIONAL GENE THERAPY FOR WD
18TH NOVEMBER, 2020
Pegcetacoplan (APL-2)
Pegcetacoplan (APL-2)
Pegcetacoplan (APL-2)
Apellis Announces FDA Acceptance and Priority Review of the New Drug Application for Pegcetacoplan for the Treatment of PNH
Mon November 16, 2020 7:00 AM|GlobeNewswire|About: APLS
- PDUFA target action date is May 14, 2021
- FDA has stated that it is not currently planning to hold an advisory committee meeting to discuss the application
- Pegcetacoplan demonstrated superiority to eculizumab in improving hemoglobin levels in Phase 3 PEGASUS head-to-head study as well as substantial improvements in other clinical measures
- Apellis plans to open an early access program in the United States for pegcetacoplan for people living with PNH
WALTHAM, Mass., Nov. 16, 2020 (GLOBE NEWSWIRE) -- Apellis Pharmaceuticals (APLS), Inc. (Nasdaq: APLS)
More information on the EAP is available at https://apellis.com/for-patients/early-access-program/.
The PEGASUS study (APL2-302; NCT03500549) is a multi-center, randomized, active-comparator controlled Phase 3 study in 80 adults with paroxysmal nocturnal hemoglobinuria (PNH).
Pegcetacoplan (APL-2)
About Pegcetacoplan (APL-2)
Pegcetacoplan is an investigational, targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body’s immune system, which can lead to the onset and progression of many serious diseases. Pegcetacoplan is a synthetic cyclic peptide conjugated to a polyethylene glycol polymer that binds specifically to C3 and C3b. Apellis is evaluating pegcetacoplan in several clinical studies across hematology, ophthalmology, nephrology, and neurology. Pegcetacoplan was granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and the treatment of geographic atrophy, and received orphan drug designation for the treatment of C3G by the FDA and European Medicines Agency. For additional information regarding our clinical trials, visit https://apellis.com/our-science/clinical-trials.
For more information, please visit http://apellis.com.
https://seekingalpha.com/symbol/APLS
Islatravir (MK-8591)
semaglutide, a GLP-1 receptor agonist, w/ investigational FXR cilofexor/ ACC inhibitor firsocostat
Pegcetacoplan (APL-2)
Merck Advances Phase 3 Trial to Evaluate Investigational Islatravir as Once-Monthly Oral PrEP for Women at High Risk for Acquiring HIV-1
Mon November 16, 2020 6:45 AM|Business Wire|About: MRK
Collaboration with the Bill & Melinda Gates Foundation Seeks to Bring Forward a New HIV Prevention Option to Help Address the HIV Epidemic with Focus on Women in Sub-Saharan Africa
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (MRK)
Islatravir (MK-8591)
About Islatravir (MK-8591)
Islatravir (formerly MK-8591) is Merck’s investigational nucleoside reverse transcriptase translocation inhibitor (NRTTI) currently being evaluated in clinical trials for the treatment of HIV-1 infection in combination with other antiretrovirals, as well as for pre-exposure prophylaxis (PrEP) of HIV-1 infection as a single investigational agent, across a variety of formulations. In 2012, Merck licensed islatravir (4’-ethynyl-2-fluoro-2’-deoxyadenosine or EFdA) from the Yamasa Corporation based in Choshi, Japan.
For more information, visit www.merck.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20201116005095/en/
https://seekingalpha.com/symbol/MRK
RG6346 for Treatment of Chronic Hepatitis B Virus (HBV)
semaglutide, a GLP-1 receptor agonist, w/ investigational FXR cilofexor/ ACC inhibitor firsocostat
semaglutide, a GLP-1 receptor agonist, w/ investigational FXR cilofexor/ ACC inhibitor firsocostat
Dicerna Announces Positive Updated Data From Phase 1 Trial of RG6346 for Treatment of Chronic Hepatitis B Virus (HBV) Infection at AASLD’s The Liver Meeting® Digital Experience™ 2020
Mon November 16, 2020 7:31 AM|Business Wire|About: DRNA
– Data Presentations Show Treatment With Up to Four Monthly Doses of RG6346 Resulted in Substantial and Durable Reductions in HBsAg Levels Lasting Up to One Year After Last Dose –
– RG6346 Was Shown to be Safe and Well Tolerated in This Trial –
LEXINGTON, Mass.--(BUSINESS WIRE)-- Dicerna Pharmaceuticals, Inc. (DRNA) (Nasdaq: DRNA)
RG6346 for Treatment of Chronic Hepatitis B Virus (HBV)
About the RG6346 Phase 1 Proof-of-Concept Trial
The RG6346 Phase 1 proof-of-concept trial comprises three groups of adult participants: Group A, composed of 30 healthy volunteers who received single RG6346 doses up to 12.0 mg/kg (completed 2019); Group B, composed of nine participants who were newly diagnosed with chronic HBV and naive to any NUC antiviral therapy, randomized 5:31 to a single 3.0 mg/kg dose of RG6346 or placebo, respectively (completed early 2020); and Group C, composed of 18 participants who were diagnosed with chronic HBV and actively receiving NUC therapy, randomized 2:1 to four monthly doses of 1.5, 3.0 or 6.0 mg/kg of RG6346 or placebo, respectively. The last participant visit in the double-blind period up to Day 112 for Group C occurred in October 2020. Participants in Groups B and C were eligible to enter an extended follow-up observation period if they achieved an HBsAg reduction from baseline of ≥1.0 log10 IU/mL at the end of the treatment period (12 weeks/85 days for Group B; 16 weeks/112 days for Group C).
About Chronic Hepatitis B Virus (HBV) Infection
Hepatitis B virus (HBV) is the world’s most common serious liver infection and affects an estimated 292 million people worldwide.2 According to the Hepatitis B Foundation, 30 million people become newly infected with HBV each year, and it is estimated that more than 880,000 people die annually from hepatitis B and related complications such as liver cancer.3
About RG6346
RG6346 is an investigational GalXC™ RNAi therapeutic candidate in development in collaboration with Roche for the treatment of chronic hepatitis B virus (HBV) infection. Dicerna is currently conducting a Phase 1 proof-of-concept trial of RG6346 in adult patients with non-cirrhotic chronic HBV infection. Current therapies for HBV, such as nucleos(t)ide analogs, can provide long-term viral suppression if taken continuously, but they rarely lead to long-term functional cures, as measured by the clearance of HBV surface antigen (HBsAg) and sustained HBV deoxyribonucleic acid (DNA) suppression in patient plasma or blood. By contrast, RG6346 is designed to employ RNAi to knock down selectively specific genes involved in the creation of HBV messenger RNA (mRNA) and the entry of the virus into liver cells. Preclinical data have demonstrated greater than 99.9% reduction in circulating HBsAg, as observed in mouse models of HBV infection. Unlike current therapies that typically provide long-term suppression of the virus, we believe RG6346 has the potential to provide a functional cure as part of a combination regimen for patients living with chronic HBV.
For more information, please visit www.dicerna.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20201116005423/en/
https://seekingalpha.com/symbol/DRNA
Dicerna/Roche's hepatitis B candidate shows durable effect in early-stage study
Nov. 16, 2020 1:17 PM ETDicerna Pharmaceuticals, Inc. (DRNA)By: Meghavi Singh, SA News Editor
semaglutide, a GLP-1 receptor agonist, w/ investigational FXR cilofexor/ ACC inhibitor firsocostat
semaglutide, a GLP-1 receptor agonist, w/ investigational FXR cilofexor/ ACC inhibitor firsocostat
semaglutide, a GLP-1 receptor agonist, w/ investigational FXR cilofexor/ ACC inhibitor firsocostat
November 15, 2020
Gilead and Novo Nordisk Present New Data from Proof-of-Concept Trial in NASH
-- Results from First Phase 2 Trial Describing a Triple Combination Regimen Targeting NASH Presented at The Liver Meeting Digital Experience --
FOSTER CITY, Calif. & BAGSVÆRD, Denmark--(BUSINESS WIRE)-- Gilead Sciences, Inc. (Nasdaq: GILD) and Novo Nordisk A/S (NASDAQ Copenhagen: NOVO B)
Firsocostat, GS-834356 and cilofexor
For more information, visit novonordisk.com,
View source version on businesswire.com: https://www.businesswire.com/news/home/20201115005154/en/
Gilead, Novo Nordisk provide insights from mid-stage trial describing triple combo regimen in NASH
Nov. 16, 2020 3:13 AM ETGilead Sciences, Inc. (GILD)By: Mamta Mayani, SA News Editor8 Comments
- Gilead Sciences (NASDAQ:GILD) and Novo Nordisk (NYSE:NVO) presents results from a Phase 2 proof-of-concept trial.
https://seekingalpha.com/symbol/GILD
https://seekingalpha.com/symbol/NVO
biotecHOPE™ November 2020
inclisiran (KJX839)
ATA3271 against mesothelin-expressing cell lines,
inclisiran (KJX839)
New Novartis analyses for investigational inclisiran demonstrate consistently effective and sustained LDL-C reduction at month 17 regardless of age and gender
Nov 13, 2020
- Pooled data analyses from Phase III ORION-9, -10 and -11 showed that inclisiran consistently reduced low-density lipoprotein cholesterol (LDL-C) by approximately 51% in both male and female adult patients and in three age categories1,2
- Sustained LDL-C reduction with inclisiran was observed regardless of age or gender differences1,2 with two doses a year, after an initial dose and then again at three months; the overall trial dosing schedule was at months 1, 3 and then every 6 months up to month 17
- LDL-C is the most readily modifiable risk factor for atherosclerotic cardiovascular disease (ASCVD), yet despite widespread statin use 80% of high-risk patients do not reach guideline-recommended LDL-C targets3,4
- Inclisiran recently received a positive CHMP opinion and recommendation for marketing authorization in Europe and is under review by the U.S. Food and Drug Administration
Basel, November 13, 2020 — Novartis
About the Pooled Post-Hoc Analyses from Phase III ORION-9, -10 and -11 trials: Age and Gender
The pooled analyses include data from inclisiran’s ORION-9, -10 and -11 trials, which were multicenter, double-blind, randomized, placebo-controlled,18-month studies evaluating inclisiran in patients with heterozygous familial hypercholesterolemia (ORION-9), ASCVD (ORION-10) and ASCVD or ASCVD risk equivalents (ORION-11) on statin therapy who required additional LDL-C lowering. The primary endpoints for these studies were percentage change in LDL-C from baseline to 17 months and time-adjusted percentage change in LDL-C from baseline between 3 months and up to 18 months. The primary endpoints were achieved in all three studies5-7.
inclisiran (KJX839)
- Pooled data analyses from Phase III ORION-9, -10 and -11 showed that inclisiran consistently reduced low-density lipoprotein cholesterol (LDL-C) by approximately 51% in both male and female adult patients and in three age categories1,2
- Sustained LDL-C reduction with inclisiran was observed regardless of age or gender differences1,2 with two doses a year, after an initial dose and then again at three months; the overall trial dosing schedule was at months 1, 3 and then every 6 months up to month 17
- LDL-C is the most readily modifiable risk factor for atherosclerotic cardiovascular disease (ASCVD), yet despite widespread statin use 80% of high-risk patients do not reach guideline-recommended LDL-C targets3,4
- Inclisiran recently received a positive CHMP opinion and recommendation for marketing authorization in Europe and is under review by the U.S. Food and Drug Administration
Basel, November 13, 2020 — Novartis today announced results from two pooled post-hoc analyses of Phase III ORION-9, -10 and -11 trials, evaluating the impact of age and gender on the efficacy and safety of inclisiran, an investigational and potential first-in-class small interfering RNA (siRNA) for hyperlipidemia in adults with atherosclerotic cardiovascular disease (ASCVD) or heterozygous familial hypercholesterolemia (HeFH). The data showed that at month 17, inclisiran was well-tolerated and provided effective and sustained reduction in low-density lipoprotein cholesterol (LDL-C) when used in addition to other lipid lowering therapies regardless of patients’ age and gender1,2. During the trials, inclisiran was administered at months 1, 3 and then every 6 months up to month 17. Results were presented at the virtual American Heart Association Scientific Sessions 2020.
“High LDL-C and other risk factors for ASCVD, as well as the potential for treatment side effects, may increase with age and differ by gender,” said Kausik Ray, MD, ORION-11 trial principal investigator, Professor of Public Health at Imperial College London and Honorary Consultant Cardiologist at the Imperial College NHS Trust. “These data are important as they show that inclisiran, as a siRNA, has the potential to provide consistent efficacy and tolerability despite the cholesterol-lowering treatment challenges posed by age and gender with two doses a year after the initial dosing regimen on day 1 and month 3.”
In post-hoc analyses of the pooled results from the ORION Phase III trials in more than 3,600 patients, treatment with inclisiran delivered similar LDL-C reductions of approximately 51% from baseline for both women and men (50.6% vs 50.6% respectively) compared to placebo1. Results from a second pooled analysis showed that inclisiran-treated patients in three age categories all achieved similar LDL-C reductions of approximately 51% (−51.3% <65 years; −49.9% ≥65 years to <75 years; −51.0% ≥75 years)2. In both analyses, inclisiran was well-tolerated1,2.
“Whether you look at it from an age or a gender perspective, inclisiran analyses continue to show consistency with effective and sustained LDL-C reduction lasting over the dosing interval,” said David Soergel, MD, Global Head of Cardiovascular, Renal and Metabolic Drug Development, Novartis. “As we move forward in our journey to reimagine treatment for ASCVD, these data analyses reinforce the potential of inclisiran as a first-in-class siRNA treatment to transform LDL-C management with two doses a year, following the initial dose and another dose at three months, and a positive tolerability profile.”
On Friday, October 16, 2020, the Committee for Medicinal Products for Human Use of the European Medicines Agency adopted a positive opinion and recommended granting marketing authorization for inclisiran. Novartis is anticipating a final regulatory decision in Europe in December 2020 and is under review by the US Food and Drug Administration.
About the Pooled Post-Hoc Analyses from Phase III ORION-9, -10 and -11 trials: Age and Gender
The pooled analyses include data from inclisiran’s ORION-9, -10 and -11 trials, which were multicenter, double-blind, randomized, placebo-controlled,18-month studies evaluating inclisiran in patients with heterozygous familial hypercholesterolemia (ORION-9), ASCVD (ORION-10) and ASCVD or ASCVD risk equivalents (ORION-11) on statin therapy who required additional LDL-C lowering. The primary endpoints for these studies were percentage change in LDL-C from baseline to 17 months and time-adjusted percentage change in LDL-C from baseline between 3 months and up to 18 months. The primary endpoints were achieved in all three studies5-7.
The pooled analyses assessed inclisiran’s efficacy for lowering LDL-C, as well as safety and tolerability, across age ranges and by gender1,2.
Impact of age on the efficacy of inclisiran versus placebo (<65 years; ≥65 years to <75 years; ≥75 years)2:
- Percentage change between inclisiran and placebo in LDL-C at month 17 was similar across all ages: −51.3% (<65 years), −49.9% (≥65 years to <75 years), −51.0% (≥75 years)
- Time-adjusted percentage LDL-C change between inclisiran and placebo at month 18 was similar across all ages: −49.6% (<65 years), −51.5% (≥65 years to <75 years), −50.8% (≥75 years)
Impact of gender on the efficacy of inclisiran versus placebo1:
- Percentage change between inclisiran and placebo in LDL-C at month 17 was consistent across women and men at -50.6% and -50.6% respectively
- Time-adjusted LDL-C change between inclisiran and placebo at month 18 was consistent across women and men at -50.5% and -50.6%
Across both analyses, inclisiran was reported to be well-tolerated irrespective of age or gender. Injection site reactions (ISR) were more frequent in female versus male patients and in the <65 population versus elder patients, all ISRs were transient and mild or moderate in terms of severity.
About the ORION Phase III Low-density Lipoprotein Cholesterol (LDL-C)-lowering Studies
ORION-9 was a pivotal Phase III, placebo-controlled, double-blind, randomized study to evaluate the efficacy, safety and tolerability of inclisiran sodium salt 300 mg, equivalent to 284 mg of inclisiran, administered subcutaneously by a healthcare professional starting it at an initial dose5. Inclisiran was then administered again at 3 months and then every 6 months thereafter in 482 participants with clinical or genetic evidence of heterozygous familial hypercholesterolemia and elevated LDL-C, despite a maximally tolerated dose of LDL-C-lowering therapies (e.g. a statin or ezetimibe). For the primary endpoints of ORION-9, inclisiran delivered mean placebo-adjusted percentage change in LDL-C reductions of 48% (P<.0001) at 17 months and demonstrated time-adjusted percentage change in LDL-C reductions of 44% (P<.0001) from 3 through 18 months. The international study was conducted at 46 sites in eight countries5.
ORION-10 was a pivotal Phase III, placebo-controlled, double-blind, randomized study to evaluate the efficacy, safety and tolerability of inclisiran sodium salt 300 mg, equivalent to 284 mg of inclisiran, administered subcutaneously by a healthcare professional starting it at an initial dose6. Inclisiran was then administered again at 3 months and then every 6 months thereafter in 1,561 participants with atherosclerotic cardiovascular disease (ASCVD) and elevated LDL-C, despite a maximally tolerated dose of LDL-C-lowering therapies (e.g. a statin and/or ezetimibe). For the primary endpoints of ORION-10, inclisiran delivered mean placebo-adjusted percentage change in LDL-C reductions of 52% (P<.0001) at 17 months and demonstrated time-adjusted percentage change in LDL-C reductions of 54% (P<.0001) from 3 through 18 months. The study was conducted at 145 sites in the United States6,7.
ORION-11 was a pivotal Phase III, placebo-controlled, double-blind, randomized study to evaluate the efficacy, safety and tolerability of inclisiran sodium salt 300 mg, equivalent to 284 mg of inclisiran, administered subcutaneously by a healthcare professional starting it at an initial dose6. Inclisiran was then administered again at 3 months and then every 6 months thereafter in 1,617 patients with ASCVD or ASCVD-risk equivalents and elevated LDL-C despite a maximally tolerated dose of statin therapy (with or without ezetimibe). For the primary endpoints of ORION-11, inclisiran delivered placebo-adjusted change in LDL-C reductions of 50% (P<.0001) at 17 months and demonstrated time-adjusted LDL-C reductions of 49% (P<.0001) from 3 through 18 months. The international study was conducted at 70 sites in seven countries6,7.
About Atherosclerotic Cardiovascular Disease (ASCVD)
Atherosclerosis corresponds to the accumulation of lipids over time mainly low-density lipoprotein cholesterol (LDL-C) in the inner lining of the arteries. Unexpected rupture of the atherosclerotic plaque can cause an atherosclerotic cardiovascular event such as a heart attack or stroke8,9. ASCVD accounts for over 85% of all cardiovascular disease deaths10. ASCVD is the primary cause of death in the European Union and its burden in the United States is greater than that from any other chronic diseases11,12. ASCVD risk equivalent corresponds to conditions that confer a similar risk for a ASCVD event (e.g. diabetes, heterozygous familial hypercholesterolemia)6,13.
About Inclisiran
If approved, inclisiran (KJX839) would be the first and only therapy to use the small interfering RNA (siRNA mechanism) of action to lower low-density lipoprotein cholesterol (LDL-C), which could help improve outcomes for patients with atherosclerotic cardiovascular disease (ASCVD), a deadly form of cardiovascular disease5,6,14. With two doses a year and effective and sustained LDL-C reduction, inclisiran works as a complement to statins. Inclisiran works differently from other therapies by preventing the production of the target protein in the liver, increasing hepatic uptake of LDL-C and clearing it from the bloodstream14. Inclisiran is dosed initially, again at 3 months, and then once every 6 months. In three clinical trials, patients taking inclisiran maintained LDL-C reduction throughout each 6-month dosing interval5,6. Administered in-office as a subcutaneous injection, inclisiran integrates seamlessly into a patient’s healthcare routine5,6.
Find out more at https://www.novartis.com.
https://www.novartis.com/our-science/novartis-global-pipeline
https://seekingalpha.com/symbol/NVS
sotatercept
ATA3271 against mesothelin-expressing cell lines,
inclisiran (KJX839)
Acceleron Presents Preliminary Interim Data from the SPECTRA Phase 2 Trial of Sotatercept in Pulmonary Arterial Hypertension (PAH) at the 2020 American Heart Association Scientific Sessions
Fri November 13, 2020 10:01 AM|Business Wire|About: XLRN
– Treatment with sotatercept in first set of patients in the ongoing SPECTRA Phase 2 trial was associated with substantial improvements in hemodynamics, exercise tolerance and exercise capacity at week 24 –
– Sotatercept was generally well tolerated, consistent with the previously reported safety profile in PAH and in other diseases –
– Acceleron to host investor and analyst conference call and webcast with guest PAH key opinion leaders today, Friday, November 13, at 11:00 a.m. EST –
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Acceleron Pharma Inc. (XLRN)
The presentation referenced above is available on the “Publications” page under the “Science & Pipeline” section of Acceleron’s website, www.acceleronpharma.com.
sotatercept
Sotatercept is a fusion protein that acts as a ligand trap for proteins involved in fibrosis and late-stage erythropoiesis (red blood cell production). The company says it can potentially restore vascular homeostasis (stable equilibrium).
Sotatercept
Ligand trap in development to rebalance BMPR-II signaling for patients with Pulmonary Arterial Hypertension
Sotatercept is an investigational product being studied in patients with pulmonary arterial hypertension (PAH) – a rare, progressive, and life-threatening blood vessel disorder.
https://acceleronpharma.com/science-pipeline/sotatercept/
Pipeline
https://acceleronpharma.com/science-pipeline/pipeline/
Acceleron Pharma (XLRN -1.8%) announces new data from two Phase 2 clinical trials, SPECTRA and PULSAR, evaluating sotatercept in patients with pulmonary arterial hypertension (PAH).
A 284-subject Phase 3 trial, STELLAR, should launch next month. The primary endpoint will be the change from baseline to week 24 in the 6-minute walk test.
Nov 13, 2020
https://seekingalpha.com/symbol/XLRN
ATA3271 against mesothelin-expressing cell lines,
ATA3271 against mesothelin-expressing cell lines,
ATA3271 against mesothelin-expressing cell lines,
Atara Bio's cancer cell therapy shows encouraging action in preclinical study
Nov. 12, 2020 12:42 PM ET|About: Atara Biotherapeutics, Inc. (ATRA)|By: Vandana Singh, SA News Editor
PIPELINE
ATA2271/ATA3271
https://www.atarabio.com/pipeline/ata2271-ata3271/
PIPELINE
A Robust Pipeline
Multiple T-Cell Immunotherapy Product Candidates in Clinical Development
Atara Biotherapeutics’s robust, late-stage pipeline features a host of potentially transformative T-cell immunotherapies for cancer, autoimmune and viral diseases. Tab-cel® (tabelecleucel) is in Phase 3 development for patients with Epstein-Barr virus-associated post-transplant lymphoproliferative disease (EBV+ PTLD) as well as in earlier stage development for other EBV-associated diseases.
https://www.atarabio.com/pipeline/
Atara Biotherapeutics Presents New Preclinical Data on ATA3271, a Next-Generation Allogeneic Mesothelin-Targeted CAR T to Treat Solid Tumors, at the 35th Society for Immunotherapy of Cancer Annual Meeting (SITC 2020)
Download as PDFNovember 12, 2020 8:30am EST
Atara’s allogeneic CAR T therapy leverages the combination of cell intrinsic PD1DNR checkpoint inhibition and 1XX CAR signaling technologies built on the Company’s novel EBV T-cell platform
Preclinical findings demonstrate potent antitumor activity, persistence and low toxicity profile of ATA3271, supporting further clinical investigation
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Atara Biotherapeutics, Inc. (Nasdaq: ATRA)
https://seekingalpha.com/symbol/ATRA
Berotralstat
lacutamab (IPH4102)
ATA3271 against mesothelin-expressing cell lines,
BioCryst Presents Data Showing Sustained Attack Rate Reductions, Improved Patient Satisfaction and Quality of Life for HAE Patients Taking Berotralstat in APeX-2 Trial
Fri November 13, 2020 7:02 AM|GlobeNewswire|About: BCRXGlobeNewswire
RESEARCH TRIANGLE PARK, N.C., Nov. 13, 2020 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (BCRX) (Nasdaq:BCRX) today presented new clinical data that further evaluates the attack rate reductions, patient satisfaction and quality of life of hereditary angioedema (HAE) patients in the APeX-2 trial over 48 weeks. Berotralstat is an investigational treatment for the prevention of attacks in patients with HAE.
For more information, please visit the Company's website at www.BioCryst.com.
Berotralstat
https://www.biocryst.com/our-program/
Hereditary Angioedema (HAE)
BioCryst is seeking to revolutionize treatment for Hereditary Angioedema (HAE) by developing orally-administered drugs to prevent and control HAE attacks
Our lead compound, ORLADEYO™ (berotralstat), is designed to be an oral, once-daily capsule that prevents attacks by inhibiting plasma kallikrein. In December 2019, BioCryst submitted a new drug application to the U.S. Food and Drug Administration seeking approval of oral, once-daily ORLADEYO for the prevention of HAE attacks.
https://www.biocryst.com/our-program/hae-program/
https://seekingalpha.com/symbol/BCRX
ANX005
lacutamab (IPH4102)
lacutamab (IPH4102)
Annexon Expands Classical Complement Platform into Neurodegenerative Diseases of the Brain with Initiation of Huntington’s Disease Clinical Program
Thu November 12, 2020 4:05 PM|GlobeNewswire|About: ANNX
– First patient dosed in Phase 2 study of ANX005 C1q targeted mAb –
SOUTH SAN FRANCISCO, Calif., Nov. 12, 2020 (GLOBE NEWSWIRE) -- Annexon, Inc. (ANNX)
Annexon (NASDAQ:ANNX) has initiated a Phase 2 study of monoclonal antibody ANX005 in Huntington’s Disease (HD), a progressive movement disorder. Initial results from the trial expected in 2H of 2021.
More information can be found at www.annexonbio.com or www.clinicaltrials.gov, identifier NCT04514367, or the HD Coalition for Patient Engagement https://www.huntingtonsociety.ca/hdcope/.
For more information, visit www.annexonbio.com.
https://annexonbio.com/pipeline/index
Nov 12, 2020
Additional Formats
PDF Version
ANX005
ANX005 is a clinical-stage investigational monoclonal antibody intended to treat patients with antibody-mediated autoimmune and complement-mediated neurodegenerative disorders. This novel therapy is formulated for IV administration and is designed to inhibit C1q and the entire classical complement pathway.
https://annexonbio.com/pipeline/anx005
https://seekingalpha.com/symbol/ANNX
lacutamab (IPH4102)
lacutamab (IPH4102)
lacutamab (IPH4102)
Innate Pharma Receives Prime Designation From the European Medicines Agency for Lacutamab in Sézary Syndrome
Fri November 13, 2020 1:00 AM|GlobeNewswire|About: IPHAGlobeNewswire
MARSEILLE, France, Nov. 13, 2020 (GLOBE NEWSWIRE) -- Innate Pharma SA (Euronext Paris: IPH – ISIN: FR0010331421; Nasdaq: IPHA)
lacutamab (IPH4102)
About Lacutamab:
Currently in Phase 2 development, lacutamab (IPH4102) is a proprietary first-in-class anti-KIR3DL2 humanized cytotoxicity-inducing antibody, designed for treatment of CTCL, an orphan disease. This group of rare cutaneous lymphomas of T lymphocytes has a poor prognosis with few therapeutic options at advanced stage
Learn more about Innate Pharma at www.innate-pharma.com
https://seekingalpha.com/symbol/IPHA
INNATE PHARMA RECEIVES PRIME DESIGNATION FROM THE EUROPEAN MEDICINES AGENCY FOR LACUTAMAB IN SÉZARY SYNDROME
Friday, November 13, 2020 - 07:00
Priority Medicines (PRIME) designation supports the potential for lacutamab to benefit Sézary Syndrome patients in need of new treatment options
INBRX-109 in Chondrosarcoma Patients
INBRX-109 in Chondrosarcoma Patients
INBRX-109 in Chondrosarcoma Patients
Inhibrx Announces Positive Interim Results from the Phase 1 Trial of INBRX-109 in Chondrosarcoma Patients
Wed November 11, 2020 4:11 PM|PR Newswire|About: INBX
- Conference call to be held today at 2:30pm PT
PR Newswire
SAN DIEGO, Nov. 11, 2020 /PRNewswire/ -- Inhibrx, Inc. (INBX)
INBRX-109 in Chondrosarcoma Patients
About INBRX-109
INBRX-109 is a precisely engineered tetravalent single domain antibody (sdAb) based therapeutic candidate that agonizes DR5 to induce tumor selective programmed cell death. A three-part, Phase 1 clinical trial was initiated in November 2018. Part 1, dose escalation, was completed in August 2019 with enrollment of 20 patients. INBRX-109 was well-tolerated, with no significant toxicities observed at doses up to and including the maximum administered dose of 30 mg/kg. No maximum tolerated dose was reached. Part 2, single agent dose expansion, commenced in September 2019, while Part 3, chemotherapy combination cohorts, initiated this month in epithelioid subtype malignant pleural mesothelioma and pancreatic adenocarcinoma.
View original content to download multimedia:http://www.prnewswire.com/news-releases/inhibrx-announces-positive-interim-results-from-the-phase-1-trial-of-inbrx-109-in-chondrosarcoma-patients-301171332.html
https://inhibrx.com/inbrx-109/
Inhibrx Announces Positive Interim Results from the Phase 1 Trial of INBRX-109 in Chondrosarcoma Patients
- Disease control observed in 92% of patients- Conference call to be held today at 2:30pm PT
SAN DIEGO, Nov. 11, 2020 /PRNewswire/ -- Inhibrx, Inc. (Nasdaq: INBX)
https://seekingalpha.com/symbol/INBX
XELJANZ ® (tofacitinib)
INBRX-109 in Chondrosarcoma Patients
INBRX-109 in Chondrosarcoma Patients
Pfizer Announces Positive Phase 3 Study Results for XELJANZ ® (tofacitinib) in Ankylosing Spondylitis (AS)
Fri November 6, 2020 6:45 AM|Business Wire|About: PFE
Late-breaking data to be presented at ACR Convergence: the American College of Rheumatology/Association of Rheumatology Professionals’ Virtual Meeting
NEW YORK--(BUSINESS WIRE)-- Pfizer Inc. (PFE) announced today positive results from a Phase 3 investigational study evaluating the safety and efficacy of tofacitinib in adults with active ankylosing spondylitis (AS). Tofacitinib is not currently approved by the U.S. Food and Drug Administration (FDA) for the treatment of AS.
XELJANZ ® (tofacitinib) in Ankylosing Spondylitis (AS)
Pfizer (NYSE:PFE) has announced today positive results from a Phase 3 study evaluating efficacy and safety of tofacitinib in adults with active ankylosing spondylitis (AS), is a rare type of arthritis affecting the spine. Data will be presented on Monday, at the American College of Rheumatology/ Association of Rheumatology Professionals Annual Meeting.
About XELJANZ® (tofacitinib)
XELJANZ® (tofacitinib) is approved in the U.S. in four indications: adults with moderately to severely active rheumatoid arthritis (RA) after methotrexate failure, adults with active psoriatic arthritis (PsA) after disease modifying antirheumatic drug (DMARD) failure, adults with moderately to severely active ulcerative colitis (UC) after tumor necrosis factor inhibitor (TNFi) failure, and patients 2 years of age or older with active polyarticular course juvenile idiopathic arthritis (pcJIA). XELJANZ has been studied in more than 50 clinical trials worldwide and prescribed to over 208,000 adult patients (the majority of whom were RA patients) worldwide in the last eight years.7,8,9
To learn more, visit www.pfizer.com/science/immunology-inflammation.
to learn more, please visit us on www.pfizer.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20201106005130/en/
https://seekingalpha.com/symbol/PFE
PFIZER ANNOUNCES POSITIVE PHASE 3 STUDY RESULTS FOR XELJANZ ® (TOFACITINIB) IN ANKYLOSING SPONDYLITIS (AS)
Friday, November 06, 2020 - 06:45am
Late-breaking data to be presented at ACR Convergence: the American College of Rheumatology/Association of Rheumatology Professionals’ Virtual Meeting
NEW YORK--(BUSINESS WIRE)-- Pfizer Inc. (NYSE: PFE) announced today positive results from a Phase 3 investigational study evaluating the safety and efficacy of tofacitinib in adults with active ankylosing spondylitis (AS). Tofacitinib is not currently approved by the U.S. Food and Drug Administration (FDA) for the treatment of AS.
Bemarituzumab (anti-FGFR2b, FPA144)
INBRX-109 in Chondrosarcoma Patients
cancer vaccine (PCV) mRNA-4157 in combination with Merck’s Keytruda®
Five Prime Announces Bemarituzumab Plus Chemotherapy Demonstrates Significant Progression-Free and Overall Survival Benefit Compared to Placebo Plus Chemotherapy in Front-Line Advanced Gastric and Gastroesophageal Junction Cancer
Tue November 10, 2020 4:12 PM|Business Wire|About: FPRXQ3: 11-03-20 Earnings Summary
EPS of $-0.74 misses by $-0.11 Revenue of $2.05M (-31.37% Y/Y) misses by $-2.5M
- All 3 efficacy endpoints in the Global Phase 2 FIGHT trial met pre-specified statistical significance
- Median progression-free survival (PFS) improved from 7.4 months to 9.5 months. Hazard ratio (HR) 0.68 (95% CI: 0.44-1.04) p=0.073
- Median overall survival (OS) improved from 12.9 months to not reached. HR 0.58 (95% CI: 0.35-0.95) p=0.027
- Overall response rate (ORR) improved by 13.1% (p=0.106)
- Bemarituzumab is a first-in-class therapeutic antibody targeting FGFR2b+ tumors found in approximately 30 percent of HER2- gastric cancers worldwide
- Trial results validate FGFR2b as a novel target for the third most common cause of cancer mortality worldwide and highlight development opportunities for other tumors that overexpress FGFR2b
- Webcast and conference call today at 1:30pm PST / 4:30pm EST
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Five Prime Therapeutics, Inc. (FPRX) (NASDAQ: FPRX)
About the FIGHT Trial
The FGFR2b Inhibition in Gastric and Gastroesophageal Junction Cancer Treatment (FIGHT) trial (NCT03694522) was designed to evaluate the efficacy and safety of bemarituzumab in combination with modified FOLFOX6 (mFOLFOX6; leucovorin calcium, fluorouracil, and oxaliplatin) vs. mFOLFOX6 plus placebo in the front-line setting of patients with newly diagnosed FGFR2b positive, locally advanced or metastatic gastric and GEJ cancer.
FGFR2b
The fibroblast growth factor (FGF)/fibroblast growth factor receptor (FGFR) pathway is implicated in the development and growth of cancer cells. FGFR2b is a form of FGFR found in epithelial cells, such as those in the stomach and skin.
Bemarituzumab (anti-FGFR2b, FPA144) is a first-in-class targeted antibody that blocks fibroblast growth factors (FGFs) from binding and activating FGFR2b, inhibiting several downstream pathways. Blocking FGFR2b activation is thought to slow cancer progression. Bemarituzumab is being developed in gastric and GEJ cancer as a targeted therapy for tumors that overexpress FGFR2b.
The fibroblast growth factor pathway is an important one for several cancers. Bemarituzumab (also known as FPA144) is a first-in-class antibody which specifically targets the fibroblast growth factor receptor 2b, or FGFR2b. There are currently no other FGFR2b-specific antibodies in clinical development. In gastric cancer, overexpression of FGFR2b has been associated with poorer overall survival.
Bemarituzumab is designed to block tumor growth through two distinct mechanisms:
- It binds specifically to FGFR2b and prevents the binding of certain fibroblast growth factors (FGF7, 10 and 22) that promote tumor growth
- It has been engineered to recruit natural killer immune cells to kill tumor cells and enhance antibody-dependent cell-mediated cytotoxicity
Five Prime Therapeutics (NASDAQ:FPRX) announces results from a Phase 2 clinical trial, FIGHT, evaluating the combination of bemarituzumab and chemo regimen mFOLFOX6 in patients with fibroblast growth factor receptor 2b-positive (FGFR2b+), non HER2-positive (non HER2+) advanced gastric or gastroesophageal junction (GEJ) cancer in a first-line setting.
https://seekingalpha.com/symbol/FPRX
cancer vaccine (PCV) mRNA-4157 in combination with Merck’s Keytruda®
cancer vaccine (PCV) mRNA-4157 in combination with Merck’s Keytruda®
cancer vaccine (PCV) mRNA-4157 in combination with Merck’s Keytruda®
Moderna Announces Clinical Updates on Personalized Cancer Vaccine Program
Wed November 11, 2020 7:09 AM| Business Wire| About: MRNA
Q3: 11-05-20 Earnings Summary
EPS of $1.63 beats by $0.14 Revenue of $10.54B (75.46% Y/Y) beats by $199.75M
Interim data from Phase 1 dose expansion cohort of mRNA-4157 in combination with pembrolizumab shared at The Society for Immunotherapy of Cancer’s Annual Meeting 2020
Data support expansion of Head and Neck Squamous Cell Carcinoma (HNSCC) cancer patient cohort
Praveen Aanur, MBBS joins Moderna (MODNA) as Vice President, Therapeutic Area Head for Oncology Development
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Moderna, Inc. (MRNA), (Nasdaq: MRNA)
To learn more, visit www.modernatx.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20201111005423/en/
Moderna (NASDAQ:MRNA) announces promising preliminary results from a Phase 1 clinical trial evaluating mRNA personalized cancer vaccine candidate mRNA-4157, combined with Merck's (NYSE:MRK) Keytruda (pembrolizumab), in patients with unresectable solid tumors. The results were presented at The Society for Immunotherapy of Cancer's (SITC) Annual Meeting.
cancer vaccine (PCV) mRNA-4157 in combination with Merck’s Keytruda®
https://seekingalpha.com/symbol/MRNA
Moderna Announces Clinical Updates on Personalized Cancer Vaccine Program
November 11, 2020 at 7:09 AM ESTPDF Version
Interim data from Phase 1 dose expansion cohort of mRNA-4157 in combination with pembrolizumab shared at The Society for Immunotherapy of Cancer’s Annual Meeting 2020
Data support expansion of Head and Neck Squamous Cell Carcinoma (HNSCC) cancer patient cohort
Praveen Aanur, MBBS joins Moderna as Vice President, Therapeutic Area Head for Oncology Development
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Nov. 11, 2020-- Moderna, Inc., (Nasdaq: MRNA)
STI-2099 (COVI-DROPS™)
cancer vaccine (PCV) mRNA-4157 in combination with Merck’s Keytruda®
Epcoritamab is an investigational IgG1-bispecific antibody
Sorrento Announces IND Filing for COVI-DROPS, an Intranasal Formulation of a High Potency Neutralizing Antibody Against SARS-CoV-2
Wed November 11, 2020 9:00 AM|GlobeNewswire|About: SRNEQ3: 11-09-20 Earnings SummaryEPS of $-0.1869 misses by $-0.03 Revenue of $11.75M (103.41% Y/Y) beats by $0.23M
- IND filing today for STI-2099 (COVI-DROPS™) for a phase 1 safety and pharmacokinetic study in healthy volunteers and outpatients with mild COVID-19 disease with or without a simultaneous intravenous injection of COVI-AMG™.
- Initial trial is expected to be followed by a phase 2 trial in both mild and moderate COVID-19 patients, either as a stand-alone nasal application or as a combination nasal and intravenous administration.
SAN DIEGO, Nov. 11, 2020 (GLOBE NEWSWIRE) -- Sorrento Therapeutics, Inc. (SRNE)
For more information, visit www.sorrentotherapeutics.com
STI-2099 (COVI-DROPS™)
COVID-19 Programs
- COVI-TRACE™ (Diagnostic Test for the Detection of SARS-CoV-2 in Saliva)
- COVI-TRACK™ (Antibody Test for the Detection of Antibodies to SARS-CoV-2 in blood)
- COVI-GUARD™ (Neutralizing Antibody – STI 1499)
- COVI-AMG™ (Affinity Matured COVI-GUARD Neutralizing Antibody – STI 2020)
- COVI-DROPS™ (Intranasal COVI-AMG Neutralizing Antibody – STI 2099)
- COVI-SHIELD™ (Neutralizing Antibody Cocktail)
- COVIDTRAP™ (ACE2 Receptor Decoy – STI 4398)
- ABIVERTINIB (Cytokine Storm – STI 5656)
- SALICYN-30 (Anti-viral – STI 2030)
- MESENCHYMAL STEM CELL (Acute Respiratory Distress Syndrome – STI 8282)
https://sorrentotherapeutics.com/research/covid-19/
COVI-DROPS™ (Intranasal COVI-AMG Neutralizing Antibody – STI 2099)
https://sorrentotherapeutics.com/research/covid-19/covi-drops/
https://seekingalpha.com/symbol/SRNE
Epcoritamab is an investigational IgG1-bispecific antibody
cancer vaccine (PCV) mRNA-4157 in combination with Merck’s Keytruda®
Epcoritamab is an investigational IgG1-bispecific antibody
Genmab Announces Phase 3 Study Evaluating Epcoritamab in Patients with Relapsed or Refractory Diffuse Large B-cell Lymphoma
Thu November 5, 2020 8:39 AM|GlobeNewswire|About: GMAB
Company Announcement
- First Phase 3 study of epcoritamab as part of broad clinical development plan with AbbVie
- Diffuse Large B-cell Lymphoma is the most common form of non-Hodgkin’s lymphoma worldwide
Copenhagen, Denmark; November 5, 2020 – Genmab A/S (GNMSF) (Nasdaq: GMAB)
Genmab A/S (NASDAQ:GMAB) announces the initiation of a 480-subject Phase 3 clinical trial comparing bispecific antibody (simultaneously binds to CD3 and CD20) epcoritamab to chemo in patients with diffuse large B-cell lymphoma (DLBCL). The primary endpoint is overall survival.
About Epcoritamab
Epcoritamab is an investigational IgG1-bispecific antibody created using Genmab’s proprietary DuoBody technology. Genmab’s DuoBody-CD3 technology is designed to direct cytotoxic T cells selectively to tumors to elicit an immune response towards malignant cells. Epcoritamab is designed to simultaneously bind to CD3 on T cells and CD20 on B cells and induces T cell mediated killing of lymphoma B cells.6 CD20 is a clinically validated therapeutic target, and is expressed on many B-cell malignancies, including diffuse large B-cell lymphoma, follicular lymphoma, mantle cell lymphoma and chronic lymphocytic leukemia.7,8 Epcoritamab is being co-developed by Genmab and AbbVie as part of the companies’ broad oncology collaboration.
https://seekingalpha.com/symbol/GMAB
https://seekingalpha.com/symbol/ABBV
https://www.genmab.com/pipeline/#page-products
Genmab Announces Phase 3 Study Evaluating Epcoritamab in Patients with Relapsed or Refractory Diffuse Large B-cell Lymphoma
Nov 5, 2020 at 2:38 PM CET
Company Announcement
- First Phase 3 study of epcoritamab as part of broad clinical development plan with AbbVie
- Diffuse Large B-cell Lymphoma is the most common form of non-Hodgkin’s lymphoma worldwide
Copenhagen, Denmark; November 5, 2020 – Genmab A/S (Nasdaq: GMAB) announced today that it will initiate a Phase 3 study of epcoritamab in diffuse large B-cell lymphoma (DLBCL).
biotecHOPE™ November & October 2020
Anti-C5aR Antibody TJ210/MOR210
LB-001 for the treatment methylmalonic acidemia (MMA)
LB-001 for the treatment methylmalonic acidemia (MMA)
I-Mab Announces Preclinical Data on Differentiated Anti-C5aR Antibody TJ210/MOR210 at SITC 2020
Wed November 11, 2020 8:00 AM|PR Newswire|About: IMABPR Newswire
SHANGHAI and GAITHERSBURG, Md., Nov. 11, 2020 /PRNewswire/ -- I-Mab (IMAB)
Anti-C5aR Antibody TJ210/MOR210
About TJ210/MOR210
TJ210/MOR210 is a novel human antibody directed against C5aR derived from MorphoSys's HuCAL Platinum® technology. C5aR, the receptor of the complement factor C5a, is investigated as a potential new drug target in the field of immuno-oncology and autoimmune diseases. Tumors have been shown to produce high amounts of C5a, which, by recruiting and activating myeloid-derived suppressor cells (MDSCs), M2 macrophages and neutrophils, is assumed to contribute to an immune-suppressive pro-tumorigenic microenvironment. TJ210/MOR210 is intended to block the interaction between C5a and its receptor, thereby potentially neutralizing the immune suppressive function of C5a and enabling immune cells to attack the tumor.
For more information, please visit http://ir.i-mabbiopharma.com
View original content to download multimedia:http://www.prnewswire.com/news-releases/i-mab-announces-preclinical-data-on-differentiated-anti-c5ar-antibody-tj210mor210-at-sitc-2020-301170813.html
https://seekingalpha.com/symbol/IMAB
Nov 11, 2020
I-Mab Announces Preclinical Data on Differentiated Anti-C5aR Antibody TJ210/MOR210 at SITC 2020
- Oral presentation highlights the unique epitope, pharmacological profile and anti-tumor properties of TJ210/MOR210 SHANGHAI and GAITHERSBURG, Md. , Nov. 11, 2020 /PRNewswire/ -- I-Mab (the "Company") (Nasdaq: IMAB), a clinical stage biopharmaceutical company committed to the discovery,
Pipeline Strategy and Progress
https://www.i-mabbiopharma.com/en/Pipeline.aspx
LB-001 for the treatment methylmalonic acidemia (MMA)
LB-001 for the treatment methylmalonic acidemia (MMA)
LB-001 for the treatment methylmalonic acidemia (MMA)
LogicBio Therapeutics Receives FDA Fast Track Designation for LB-001 for the Treatment of Methylmalonic Acidemia (MMA)
Wed November 4, 2020 7:45 AM|GlobeNewswire|About: LOGCGlobeNewswire
LEXINGTON, Mass., Nov. 04, 2020 (GLOBE NEWSWIRE) -- LogicBio Therapeutics, Inc. (LOGC) (LogicBio),
LB-001 for the treatment methylmalonic acidemia (MMA)
For more information, please visit www.logicbio.com.
https://seekingalpha.com/symbol/LOGC
https://www.logicbio.com/pipeline/
Nov 04, 2020
vupanorsen (AKCEA-ANGPTL3-LRx)
LB-001 for the treatment methylmalonic acidemia (MMA)
TSHA-104, an AAV9-based gene therapy
Ionis and Akcea announce that Pfizer has initiated a Phase 2b clinical study of vupanorsen (AKCEA-ANGPTL3-LRx)
Tue November 3, 2020 6:00 PM|PR Newswire|About: IONS, PFEQ3: 10-27-20 Earnings Summary
EPS of $0.72 beats by $0.01 Revenue of $12.13B (-4.33% Y/Y) misses by $-166.74M
CARLSBAD, Calif. and BOSTON, Nov. 3, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (IONS) and its wholly owned subsidiary Akcea Therapeutics, Inc. (AKCA), today announced that Pfizer Inc. (PFE)
vupanorsen (AKCEA-ANGPTL3-LRx)
To learn more about Ionis visit www.ionispharma.com
For more information about Akcea, please visit www.akceatx.com. For more information about Akcea, please visit www.akceatx.com.
View original content to download multimedia:http://www.prnewswire.com/news-releases/ionis-and-akcea-announce-that-pfizer-has-initiated-a-phase-2b-clinical-study-of-vupanorsen-akcea-angptl3-lrx-301166067.html
The Ionis antisense pipeline
https://www.ionispharma.com/ionis-innovation/pipeline/
The study, Targeting ANGPTL3 with an antisense oligonucleotide in adults with dyslipidemia (TRANSLATE-TIMI 70), will evaluate various doses of vupanorsen to inform potential future development.
https://seekingalpha.com/symbol/IONS
https://seekingalpha.com/symbol/AKCA
https://seekingalpha.com/symbol/PFE
TSHA-104, an AAV9-based gene therapy
TSHA-104, an AAV9-based gene therapy
TSHA-104, an AAV9-based gene therapy
Taysha Gene Therapies Receives Rare Pediatric Disease Designation and Orphan Drug Designation for TSHA-104 to Treat SURF1-Associated Leigh Syndrome
Tue October 27, 2020 7:00 AM|Business Wire|About: TSHA
Taysha anticipated to submit Investigational New Drug Application for TSHA-104 to FDA in 2021
Rare pediatric disease and orphan drug designations now obtained in multiple pipeline programs, including TSHA-101 for GM2 gangliosidosis, TSHA-102 for Rett syndrome and TSHA-118 for CLN1
DALLAS--(BUSINESS WIRE)-- Taysha Gene Therapies Inc. (TSHA)
More information is available at www.tayshagtx.com.
TSHA-104, an AAV9-based gene therapy
https://seekingalpha.com/symbol/TSHA
https://tayshagtx.com/pipeline/
efavaleukin alfa (formerly AMG 592)
TSHA-104, an AAV9-based gene therapy
efavaleukin alfa (formerly AMG 592)
Amgen Announces Participation Of Systemic Lupus Erythematosus (SLE) Adaptive Clinical Trial In The FDA Complex Innovative Trial Designs (CID) Pilot Program
Tue October 27, 2020 4:00 PM|PR Newswire|About: AMGNPR Newswire
THOUSAND OAKS, Calif., Oct. 27, 2020 /PRNewswire/ -- Amgen (AMGN)
Efavaleukin alfa
For more information, visit www.amgen.com
View original content to download multimedia:http://www.prnewswire.com/news-releases/amgen-announces-participation-of-systemic-lupus-erythematosus-sle-adaptive-clinical-trial-in-the-fda-complex-innovative-trial-designs-cid-pilot-program-301160908.html
https://seekingalpha.com/symbol/AMGN
Amgen Announces Participation Of Systemic Lupus Erythematosus (SLE) Adaptive Clinical Trial In The FDA Complex Innovative Trial Designs (CID) Pilot Program
Amgen and FDA Collaborate on Novel Clinical Trial Design to Advance Development of Potential Treatment for Patients With Uncontrolled SLE
THOUSAND OAKS, Calif., Oct. 27, 2020 /PRNewswire/ -- Amgen (NASDAQ:AMGN)
https://seekingalpha.com/symbol/AMGN
Mavrilimumab
TSHA-104, an AAV9-based gene therapy
efavaleukin alfa (formerly AMG 592)
Kiniksa Announces New Data from Phase 2 Trial of Mavrilimumab in Giant Cell Arteritis to be Presented at Late-Breaking Abstracts Session of American College of Rheumatology Convergence 2020
Mon October 26, 2020 8:00 AM|GlobeNewswire|About: KNSA
- Mavrilimumab reduced time-to-flare and increased sustained remission in Phase 2 giant cell arteritis clinical trial -
- Primary and secondary efficacy endpoints achieved statistical significance -
- Results were consistent across new onset and relapsing/refractory cohorts -
HAMILTON, Bermuda, Oct. 26, 2020 (GLOBE NEWSWIRE) -- Kiniksa Pharmaceuticals, Ltd. (KNSA) (Nasdaq: KNSA)
Mavrilimumab
https://www.kiniksa.com/our-pipeline/mavrilimumab/
10/26/20
For more information, please visit www.kiniksa.com.
https://seekingalpha.com/news/3625814-kiniksa-reports-additional-mavrilimumab-data
https://seekingalpha.com/symbol/KNSA
https://www.kiniksa.com/our-pipeline/
Adagrasib (MRTX849)
Adagrasib (MRTX849)
Adagrasib (MRTX849)
Mirati Therapeutics Reports Investigational Adagrasib (MRTX849) Preliminary Data Demonstrating Tolerability and Durable Anti-Tumor Activity as well as Initial MRTX1133 Preclinical Data
Sun October 25, 2020 10:45 AM|PR Newswire|About: MRTX
- Median duration of treatment >8 months with 50% of patients and 83% of responders still on treatment in the Phase 1/1b monotherapy cohort in patients with advanced NSCLC
- Well-tolerated safety profile across monotherapy and combination trials
- Initial preclinical data for MRTX1133, the Company's potential first-in-class KRAS G12D selective inhibitor, demonstrate significant tumor regression in mutant animal models; IND filing planned for 1H 2021
- Mirati to host virtual investor event today at 1:30 p.m. PT / 4:30 p.m. ET
PR Newswire
SAN DIEGO, Oct. 25, 2020 /PRNewswire/ -- Mirati Therapeutics, Inc. (MRTX)
Adagrasib (MRTX849)
https://www.mirati.com/pipeline/
10/25/2020
For more information, visit www.mirati.com.
View original content to download multimedia:http://www.prnewswire.com/news-releases/mirati-therapeutics-reports-investigational-adagrasib-mrtx849-preliminary-data-demonstrating-tolerability-and-durable-anti-tumor-activity-as-well-as-initial-mrtx1133-preclinical-data-301159166.html
https://seekingalpha.com/symbol/MRTX
Olutasidenib
Adagrasib (MRTX849)
Adagrasib (MRTX849)
Forma Therapeutics Announces Positive Top-line Olutasidenib Data From a Planned Interim Analysis of a Registrational Phase 2 Clinical Trial in Acute Myeloid Leukemia (AML)
Mon October 26, 2020 7:05 AM|Business Wire|About: FMTX
FT2102-HEM-101 clinical trial data demonstrate a 30% CR and 3% CRh rate with olutasidenib monotherapy in 123 relapsed or refractory IDH1m AML patients
While median duration of CR/CRh has not been reached, sensitivity analysis indicates the median duration of CR/CRh to be 13.8 months
Favorable tolerability profile consistent with Phase 1 study results
Data will be submitted for discussion at an upcoming medical meeting
WATERTOWN, Mass.--(BUSINESS WIRE)-- Forma Therapeutics Holdings, Inc. (FMTX) (Nasdaq: FMTX)
Forma Therapeutics Holdings (NASDAQ:FMTX) announces positive preliminary data from a planned interim analysis of a Phase 2 clinical trial evaluating olutasidenib (FT-2102) in patients with isocitrate dehydrogenase 1 (IDH1) mutation-positive acute myeloid leukemia (AML).
Olutasidenib
https://www.formatherapeutics.com/pipeline/olutasidenib/
For more information, please visit www.FormaTherapeutics.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20201026005229/en/
Oct 26, 2020
https://seekingalpha.com/symbol/FMTX
Vadadustat
Adagrasib (MRTX849)
voclosporin
Akebia Presents Results from its INNO2VATE Global Phase 3 Program; Demonstrated Efficacy and Cardiovascular Safety of Vadadustat for the Treatment of Anemia due to Chronic Kidney Disease in Adult Patients on Dialysis
Thu October 22, 2020 5:05 PM|PR Newswire|About: AKBA
- Presentation expands on previously announced positive top-line data showing vadadustat achieved clear and consistent results across primary and secondary efficacy and safety endpoints
- Company on track to submit New Drug Application
- Company to host investor briefing webcast at 4:10 p.m. ET on October 23, 2020
PR Newswire
CAMBRIDGE, Mass., Oct. 22, 2020 /PRNewswire/ -- Akebia Therapeutics, Inc. (AKBA) (Nasdaq: AKBA)
Vadadustat
Akebia Therapeutics (NASDAQ:AKBA) has reported additional data from the INNO2VATE Phase 3 trial comparing its vadadustat to darbepoetin alfa (similar to erythropoietin) in adult patients on dialysis with chronic kidney disease-related anemia. Data were presented at the American Society of Nephrology Kidney Week.
For more information, please visit our website at www.akebia.com
View original content to download multimedia:http://www.prnewswire.com/news-releases/akebia-presents-results-from-its-inno2vate-global-phase-3-program-demonstrated-efficacy-and-cardiovascular-safety-of-vadadustat-for-the-treatment-of-anemia-due-to-chronic-kidney-disease-in-adult-patients-on-dialysis-301158444.html
https://seekingalpha.com/symbol/AKBA
October 22, 2020
voclosporin
SGX301 (synthetic hypericin)
voclosporin
Aurinia Presents Voclosporin Efficacy and Pharmacokinetic Data from Integrated Analysis of AURA-LV and AURORA Pivotal Trials at ASN Kidney Week 2020
Thu October 22, 2020 4:15 PM|Business Wire|About: AUPH
- Integrated analysis confirms statistically superior efficacy and safety of voclosporin in combination with MMF and steroids over standard-of-care –
- Voclosporin pharmacokinetic data supports consistent dose-response, potentially eliminating the need for therapeutic drug monitoring –
VICTORIA, British Columbia--(BUSINESS WIRE)-- Aurinia Pharmaceuticals Inc. (AUPH)
View source version on businesswire.com: https://www.businesswire.com/news/home/20201022006147/en/
https://seekingalpha.com/symbol/AUPH
voclosporin
OCTOBER 22, 2020
Nedosiran PHYOX™3 Trial
SGX301 (synthetic hypericin)
SGX301 (synthetic hypericin)
Dicerna Presents Positive New Interim Data From PHYOX™3 Long-Term, Open-Label Extension Study of Nedosiran for Treatment of Primary Hyperoxaluria (PH)
Thu October 22, 2020 10:05 AM|Business Wire|About: DRNA
– All Participants Receiving Nedosiran, Regardless of Subtype, Achieved Normalization or Near-Normalization of Urinary Oxalate, a Key PH Measure, by Day 180 in Ongoing, Open-Label Trial –
– Nedosiran Was Generally Well Tolerated With No Serious Safety Concerns Identified in This Ongoing Study –
LEXINGTON, Mass.--(BUSINESS WIRE)-- Dicerna Pharmaceuticals, Inc. (DRNA) (Nasdaq: DRNA)
Nedosiran PHYOX™3 Trial
The PHYOX™3 trial (ClinicalTrials.gov: NCT04042402) is an ongoing, open-label extension study evaluating nedosiran’s long-term safety and efficacy in participants with primary hyperoxaluria (PH), a family of ultra-rare, life-threatening genetic disorders that initially manifest with recurrent renal stones and can lead to kidney failure.
For more information, please visit www.dicerna.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20201022005764/en/
Dicerna's nedosiran shows positive effect in rare kidney stone disorder
Oct. 22, 2020 11:24 AM ET|About: Dicerna Pharmaceutical... (DRNA)|By: Douglas W. House, SA News Editor
https://seekingalpha.com/symbol/DRNA
Oct 22, 2020
SGX301 (synthetic hypericin)
SGX301 (synthetic hypericin)
SGX301 (synthetic hypericin)
Soligenix Announces Phase 3 FLASH Study Continues to Demonstrate Positive Benefits in Patients with Cutaneous T-Cell Lymphoma
Thu October 22, 2020 9:00 AM|PR Newswire|About: SNGX
- SGX301 remains safe and well tolerated with no systemic exposure through 6 months including the optional, final cycle of the trial (Cycle 3)
- SGX301 demonstrates statistically significant response in both patch and plaque lesions through 12 weeks of treatment (Cycle 2), highlighting the unique benefits of deeper skin penetration
PR Newswire
PRINCETON, N.J., Oct. 22, 2020 /PRNewswire/ -- Soligenix, Inc. (SNGX)
These results are consistent with the positive findings highlighted in a recently reported case study of folliculotropic mycosis fungoides, a hard to treat variant of CTCL where lesions are associated with the hair follicles deep in the skin and more resistant to phototherapy."
SGX301 is a novel first-in-class photodynamic therapy utilizing safe visible light for activation
For further information regarding Soligenix, Inc., please visit the Company's website at www.soligenix.com.
View original content:http://www.prnewswire.com/news-releases/soligenix-announces-phase-3-flash-study-continues-to-demonstrate-positive-benefits-in-patients-with-cutaneous-t-cell-lymphoma-301157831.html
SGX301 (synthetic hypericin)
Soligenix's SGX301 shows sustained benefit in late-stage lymphoma study
Oct. 22, 2020 12:18 PM ET|About: Soligenix, Inc. (SNGX)|By: Vandana Singh, SA News Editor
Additional data from Phase 3 Flash trial evaluating Soligenix's (SNGX +6.4%) SGX301 (synthetic hypericin) in patients with cutaneous T-cell lymphoma (CTCL), has reinforced positive primary endpoint treatment response demonstrated in treatment cycle 1, with further improved response rates in Cycle 3.
https://seekingalpha.com/symbol/SNGX
https://www.soligenix.com/pipeline-programs/
Soligenix Announces Phase 3 FLASH Study Continues to Demonstrate Positive Benefits in Patients with Cutaneous T-Cell Lymphoma- Nearly half of all patients in Phase 3 trial continue to see sustained and statistically significant improvement in their response rates when treated with SGX301 through 18 weeks (Cycle 3), reinforcing positive SGX301 primary endpoint treatment response- SGX301 remains safe and well tolerated with no systemic exposure through 6 months including the optional, final cycle of the trial (Cycle 3)- SGX301 demonstrates statistically significant response in both patch and plaque lesions through 12 weeks of treatment (Cycle 2), highlighting the unique benefits of deeper skin penetration
PRINCETON, N.J., Oct. 22, 2020 /PRNewswire/ -- Soligenix, Inc. (Nasdaq: SNGX)
biotecHOPE™ July/August/September 2020
onvansertib, combined with J&J's Zytiga (abiraterone acetate) and prednisone
onvansertib, combined with J&J's Zytiga (abiraterone acetate) and prednisone
onvansertib, combined with J&J's Zytiga (abiraterone acetate) and prednisone
Cardiff Oncology's onvansertib may overcome Zytiga resistance in prostate cancer
Oct. 20, 2020 9:16 AM ET|About: Cardiff Oncology, Inc. (CRDF)|By: Vandana Singh, SA News Editor
New efficacy data from ongoing Phase 2 trial of Cardiff Oncology's (NASDAQ:CRDF) onvansertib in combination with J&J's Zytiga (abiraterone) and prednisone for metastatic castration-resistant prostate cancer, achieves the primary endpoint of disease control in patients showing initial resistance to J&J's Zytiga (abiraterone).
https://cardiffoncology.com/pipeline/
OCT 20, 2020
-- Trial on track to meet prespecified criteria for success on its primary endpoint, with 31% (8/26) disease control rate in evaluable patients in cohorts A and B. Patients eligible for the trial have two consecutive rises in PSA levels indicating initial resistance to abiraterone (Zytiga®)
https://seekingalpha.com/symbol/CRDF
In February this year, interim results from a Phase 2 trial evaluating onvansertib, combined with J&J's Zytiga (abiraterone acetate) and prednisone, demonstrated lower PSA levels in 86% (n=6/7) of patients.
lenzilumab
onvansertib, combined with J&J's Zytiga (abiraterone acetate) and prednisone
onvansertib, combined with J&J's Zytiga (abiraterone acetate) and prednisone
National Institutes of Health Launches its ACTIV-5 “Big Effect Trial” Evaluating Humanigen’s Lenzilumab™ as Potential COVID-19 Therapy
Tue October 13, 2020 11:00 AM|Business Wire|About: HGEN
- Lenzilumab among the promising agents selected for ACTIV program from pool of approximately 400 candidate agents
BURLINGAME, Calif.--(BUSINESS WIRE)-- Humanigen, Inc. (HGEN), (Nasdaq: HGEN) (“Humanigen”)
More details on Humanigen’s programs in COVID-19 can be found on the company’s website at www.humanigen.com under the COVID-19 tab, and details on ACTIV-5/BET can be found at www.clinicaltrials.gov using Identifier NCT04583969.
For more information, visit www.humanigen.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20201013005813/en/
https://seekingalpha.com/symbol/HGEN
CLINICAL-STAGE PIPELINE: LENZILUMAB™ IN COVID-19
https://www.humanigen.com/pipeline
National Institutes of Health Launches its ACTIV-5 “Big Effect Trial” Evaluating Humanigen’s Lenzilumab™ as Potential COVID-19 Therapy
October 13, 2020
- Lenzilumab among the promising agents selected for ACTIV program from pool of approximately 400 candidate agents
Burlingame, CA – October 13, 2020 – Humanigen, Inc., (Nasdaq: HGEN)
pentavalent NanoFlu/NVX-CoV2373 vaccine
onvansertib, combined with J&J's Zytiga (abiraterone acetate) and prednisone
pentavalent NanoFlu/NVX-CoV2373 vaccine
Novavax Appoints Leadership Team to Advance NanoFlu through Regulatory Licensure
Tue October 13, 2020 8:00 AM|GlobeNewswire|About: NVAX
- Team will focus on global NanoFlu licensure and evaluation of post-pandemic influenza/COVID-19 combination vaccine with NVX-CoV2373
- Russell (Rip) Wilson promoted to Executive Vice President and NanoFlu General Manager
GAITHERSBURG, Md., Oct. 13, 2020 (GLOBE NEWSWIRE) -- Novavax, Inc. (NVAX) Novavax, Inc.
pentavalent NanoFlu/NVX-CoV2373 vaccine
Novavax to explore combined influenza/COVID-19 vaccine for post-pandemic setting
Oct. 13, 2020 11:03 AM ET|About: Novavax, Inc. (NVAX)|By: Vandana Singh, SA News Editor
https://seekingalpha.com/symbol/NVAX
https://novavax.com/our-pipeline#nvx-cov2373
Oct 13, 2020
Novavax Appoints Leadership Team to Advance NanoFlu through Regulatory Licensure
Team w ill focus on global NanoFlu licensure and evaluation of post-pandemic influenza/COVID-19 combination vaccine with NVX-CoV2373 Russell (Rip) Wilson promoted to Executive Vice President and NanoFlu General Manager GAITHERSBURG, Md. , Oct. 13, 2020 (GLOBE NEWSWIRE) -- Novavax, Inc....
VAC2
Nirogacestat in Combination with PF‐06863135
pentavalent NanoFlu/NVX-CoV2373 vaccine
Lineage Cell Therapeutics and Cancer Research UK Announce Encouraging Preliminary Phase 1 Study Results With VAC2 for the Treatment of Non-small Cell Lung Cancer
Tue October 13, 2020 9:00 AM|Business Wire|About: LCTX
- Potent Induction of Immune Responses Observed with VAC2 Vaccine
- Peripheral Antigen-specific Immunogenicity Above 3% Observed at Multiple Timepoints
- VAC2 Appears Well Tolerated with No Unexpected Adverse Events
CARLSBAD, Calif. & LONDON--(BUSINESS WIRE)-- Lineage Cell Therapeutics, Inc. (LCTX) (NYSE American and TASE: LCTX)
VAC2
https://lineagecell.com/products-pipeline/vac2/
For further information about Cancer Research UK's work or to find out how to support the charity, please call 0300 123 1022 or visit www.cancerresearchuk.org.
For more information, please visit www.lineagecell.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20201013005118/en/
Phase 1 study of Lineage Cell Therapeutics's (LCTX) VAC2 in non-small cell lung cancer, demonstrated potent induction of immune responses in all patients dosed to date, with high levels of peripheral antigen-specific immunogenicity observed at multiple time points and confirmed by multimer staining.
https://seekingalpha.com/symbol/LCTX
IONIS-ENAC-2.5Rx
Nirogacestat in Combination with PF‐06863135
Nirogacestat in Combination with PF‐06863135
Ionis' inhaled antisense candidate shows encouraging action in cystic fibrosis study
Oct. 13, 2020 7:31 AM ET|About: Ionis Pharmaceuticals, Inc. (IONS)|By: Vandana Singh, SA News Editor
A Phase 1/2a Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single and Multiple Doses of IONIS-ENaCRx in Healthy Volunteers and Patients With Cystic Fibrosis
https://clinicaltrials.gov/ct2/show/NCT03647228
IONIS-ENAC-2.5Rx
https://www.ionispharma.com/ionis-innovation/pipeline/
Ionis' inhaled antisense medicine demonstrates potential as a novel treatment for cystic fibrosis
October 13, 2020 at 7:05 AM EDT- Data from the first clinical study of IONIS-ENAC-2.5 Rx to be presented at North American Cystic Fibrosis Conference
CARLSBAD, Calif., Oct. 13, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS)
https://seekingalpha.com/symbol/IONS
Nirogacestat in Combination with PF‐06863135
Nirogacestat in Combination with PF‐06863135
Nirogacestat in Combination with PF‐06863135
SpringWorks Therapeutics Announces Clinical Collaboration with Pfizer Inc. to Evaluate Nirogacestat in Combination with PF‐06863135 in Patients with Relapsed or Refractory Multiple Myeloma
Mon October 5, 2020 6:30 AM|GlobeNewswire|About: PFE, SWTXGlobeNewswire
STAMFORD, Conn., Oct. 05, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (SWTX)
For more information, visit www.springworkstx.com
https://seekingalpha.com/symbol/PFE
https://seekingalpha.com/symbol/SWTX
https://www.springworkstx.com/
SpringWorks Therapeutics Announces Clinical Collaboration with Pfizer Inc. to Evaluate Nirogacestat in Combination with PF‐06863135 in Patients with Relapsed or Refractory Multiple Myeloma
October 5, 2020 at 6:30 AM EDTPDF Version
Fifth industry collaboration to evaluate nirogacestat as a BCMA potentiator across modalities
STAMFORD, Conn., Oct. 05, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (Nasdaq: SWTX),
ION373
Lumasiran is an investigational, subcutaneously administered RNAi therapeutic
Soticlestat (OV935/TAK-935)
Ionis treatment for Alexander disease receives orphan drug designation from U.S. FDA
Wed September 30, 2020 4:05 PM|PR Newswire|About: IONSPR Newswire
CARLSBAD, Calif., Sept. 30, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (IONS)
To learn more about Ionis visit www.ionispharma.com
View original content to download multimedia:http://www.prnewswire.com/news-releases/ionis-treatment-for-alexander-disease-receives-orphan-drug-designation-from-us-fda-301142267.html
https://www.ionispharma.com/ionis-innovation/pipeline/
Ionis treatment for Alexander disease receives orphan drug designation from U.S. FDA
September 30, 2020 at 4:05 PM EDT- U.S. orphan drug status follows similar designation by the European Medicines Agency
CARLSBAD, Calif., Sept. 30, 2020 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (NASDAQ: IONS)
Soticlestat (OV935/TAK-935)
Lumasiran is an investigational, subcutaneously administered RNAi therapeutic
Soticlestat (OV935/TAK-935)
Ovid Therapeutics Provides Soticlestat (OV935/TAK-935) Results from ARCADE and ENDYMION Studies Showing Seizure Reduction in Rare Epilepsies
Wed September 30, 2020 8:00 AM|GlobeNewswire|About: OVID
- Results from signal-finding, pilot Phase 2 open-label ARCADE study and ENDYMION long-term extension study in CDKL5 deficiency disorder (CDD) and Dup15q syndrome (Dup15q) show seizure frequency reduction over time
- In CDD patients, median motor seizure frequency reduction was 24% in the ARCADE study, increasing to a 50% reduction in ENDYMION long-term extension study
- Clinical Global Impression of Change (CGI-C) and Caregiver Global Impression of Change (Care GI-C) suggest improvements beyond motor seizure frequency reduction in both CDD and Dup15q patients
- Soticlestat was generally well tolerated in both studies and continues to demonstrate a favorable safety profile
NEW YORK, Sept. 30, 2020 (GLOBE NEWSWIRE) -- Ovid Therapeutics Inc. (OVID),
Takeda and Ovid are sharing in the development and commercialization costs of soticlestat on a 50/50 basis, and if successful, the companies will share in the profits on a 50/50 basis.
For more information on Ovid, please visit www.ovidrx.com.
Ovid Therapeutics (NASDAQ:OVID) reported results from Phase 2 ARCADE and ENDYMION studies evaluating soticlestat (OV935/TAK-935) in patients with CDKL5 deficiency disorder (CDD) and Dup15q syndrome (Dup15q), two rare developmental and epileptic encephalopathies.
https://seekingalpha.com/symbol/OVID
Ovid's soticlestat shows positive effect in rare epilepsies
Sep. 30, 2020 9:00 AM ET|About: Ovid Therapeutics Inc. (OVID)|By: Vandana Singh, SA News Editor
https://seekingalpha.com/news/3618388-ovids-soticlestat-shows-positive-effect-in-rare-epilepsies
https://ovidrx.com/science/pipeline/
Sep 30, 2020
Lumasiran is an investigational, subcutaneously administered RNAi therapeutic
Lumasiran is an investigational, subcutaneously administered RNAi therapeutic
Lumasiran is an investigational, subcutaneously administered RNAi therapeutic
Alnylam Reports Positive Topline Results from ILLUMINATE-B Phase 3 Study of Lumasiran for the Treatment of Primary Hyperoxaluria Type 1 in Children Under the Age of Six
Wed September 30, 2020 7:00 AM|Business Wire|About: ALNY
– First-Ever Study to Have Evaluated the Safety and Efficacy of an Investigational RNAi Therapeutic in Infants and Children Under the Age of Six –
– Lumasiran Demonstrated Clinically Significant Reduction in Urinary Oxalate Levels Relative to Baseline in Children as Young as Four Months Old –
– Safety and Tolerability Profile Consistent with That Observed in ILLUMINATE-A Phase 3 Pivotal Study –
– Full Results Planned to be Presented at the American Society of Nephrology Annual Meeting in October 2020 –
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Alnylam Pharmaceuticals, Inc. (ALNY) (Nasdaq: ALNY)
ILLUMINATE-B (NCT03905694)
ILLUMINATE-A (NCT03681184)
For more information about our people, science and pipeline, please visit www.alnylam.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20200930005038/en/
Alnylam's lumasiran shows positive effect in young children with rare kidney disorder
Sep. 30, 2020 7:44 AM ET|About: Alnylam Pharmaceutical... (ALNY)|By: Douglas W. House, SA News Editor
eganelisib (IPI-549)
OTL-203 for the Treatment of MPS-I
Lumasiran is an investigational, subcutaneously administered RNAi therapeutic
Infinity Receives Fast Track Designation for Eganelisib in Combination with a Checkpoint Inhibitor and Chemotherapy for First-Line Treatment of Advanced TNBC
Tue September 29, 2020 7:35 AM|Business Wire|About: INFI
CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Infinity Pharmaceuticals, Inc. (INFI) (NASDAQ: INFI)
Infinity is currently enrolling patients in MARIO-3, the Company’s ongoing Phase 2 study in collaboration with Roche/Genentech to evaluate eganelisib in a triple combination front-line regimen with Tecentriq and Abraxane in TNBC.
For more information on Infinity, please refer to Infinity's website at www.infi.com.
Infinity Receives Fast Track Designation for Eganelisib in Combination with a Checkpoint Inhibitor and Chemotherapy for First-Line Treatment of Advanced TNBC
September 29, 2020 at 7:35 AM EDT
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sep. 29, 2020-- Infinity Pharmaceuticals, Inc. (NASDAQ: INFI
View source version on businesswire.com: https://www.businesswire.com/news/home/20200929005325/en/
https://seekingalpha.com/symbol/INFI
Our Pipeline
http://www.infi.com/home/our-development-program/our-pipeline/
OTL-203 for the Treatment of MPS-I
OTL-203 for the Treatment of MPS-I
OTL-203 for the Treatment of MPS-I
Orchard Therapeutics Receives EMA PRIME Designation for OTL-203 for the Treatment of MPS-I
Mon September 28, 2020 7:00 AM|GlobeNewswire|About: ORTX
BOSTON and LONDON, Sept. 28, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (ORTX)
For more information please visit the EMA website at www.ema.europa.eu.
https://www.orchard-tx.com/focus/
For more information, please visit www.orchard-tx.com
Orchard Therapeutics Receives EMA PRIME Designation for OTL-203 for the Treatment of MPS-I
BOSTON and LONDON, Sept. 28, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX)
https://seekingalpha.com/symbol/ORTX
Resiniferatoxin (RTX)
OTL-203 for the Treatment of MPS-I
OTL-203 for the Treatment of MPS-I
Sorrento Releases Positive Results of Phase 1B Trial of Resiniferatoxin (RTX) In Reduction of OsteoArthritis (OA) Knee Pain
Mon September 28, 2020 9:00 AM|GlobeNewswire|About: SRNE
- Phase 1b safety data at 6 month post-administration and Day 84 efficacy data available for all patients enrolled.
- No dose limiting toxicities at any of the doses tested (up to 30 ug) at 6 months post-administration. The study is scheduled to complete the last patient follow-up (1 year) in 1Q2021.
- Dose of 12.5ug selected as the likely optimal intra-articular therapeutic dose for future clinical trials. Durable signal at 12 weeks post-injection justifies initiation of Phase 2 (dose confirmation against active drug and saline control).
- Pain control in patients with advanced disease (Kellgren-Lawrence grade 3/4) show persistence of relief beyond 6 months and composite WOMAC A+C suggests that RTX could be a promising alternative for control of refractory pain in patients candidates for elective joint replacement.
SAN DIEGO, Sept. 28, 2020 (GLOBE NEWSWIRE) -- Sorrento Therapeutics, Inc. (SRNE)
Phase 1b trial data (clinicaltrials.gov NCT03542838) of resiniferatoxin (RTX)
Sorrento (SRNE +7.3%) reports positive results from a Phase 1b clinical trial evaluating resiniferatoxin (RTX) in patients with moderate-to-severe pain due to osteoarthritis of the knee.
https://seekingalpha.com/symbol/SRNE
For more information visit www.sorrentotherapeutics.com
https://sorrentotherapeutics.com/research/pain/
Sorrento Releases Positive Results of Phase 1B Trial of Resiniferatoxin (RTX) In Reduction of OsteoArthritis (OA) Knee Pain
September 28, 2020 at 9:00 AM EDTDownload PDF
- Phase 1b safety data at 6 month post-administration and Day 84 efficacy data available for all patients enrolled.
- No dose limiting toxicities at any of the doses tested (up to 30 ug) at 6 months post-administration. The study is scheduled to complete the last patient follow-up (1 year) in 1Q2021.
- Dose of 12.5ug selected as the likely optimal intra-articular therapeutic dose for future clinical trials. Durable signal at 12 weeks post-injection justifies initiation of Phase 2 (dose confirmation against active drug and saline control).
- Pain control in patients with advanced disease (Kellgren-Lawrence grade 3/4) show persistence of relief beyond 6 months and composite WOMAC A+C suggests that RTX could be a promising alternative for control of refractory pain in patients candidates for elective joint replacement.
SAN DIEGO, Sept. 28, 2020 (GLOBE NEWSWIRE) -- Sorrento Therapeutics, Inc. (NASDAQ: SRNE
biotecHOPE™ July/August/September 2020
Ampligen (rintatolimod)
Ampligen (rintatolimod)
Ampligen (rintatolimod)
AIM ImmunoTech Receives Statistically Significant Positive Survival Results in Pancreatic Cancer from Erasmus University Medical Center, Rotterdam, Netherlands
Tue September 22, 2020 6:50 AM|Accesswire|About: AIM
Median survival approximately two-fold higher in Ampligen arm compared to historical controls; AIM to seek FDA Fast Track designation and orphan drug status in late stage pancreatic cancer
OCALA, FL / ACCESSWIRE / September 22, 2020 / AIM ImmunoTech Inc. (AIM)
AIM ImmunoTech Receives Statistically Significant Positive Survival Results in Pancreatic Cancer from Erasmus University Medical Center, Rotterdam, Netherlands
Tuesday, September 22, 2020 6:50 AM
https://aimimmuno.com/pipeline/
https://seekingalpha.com/symbol/AIM
PDF September 22, 2020AIM ImmunoTech Receives Statistically Significant Positive Survival Results in Pancreatic Cancer from Erasmus University Medical Center, Rotterdam, Netherlands
Median survival approximately two-fold higher in Ampligen arm compared to historical controls; AIM to seek FDA Fast Track designation and orphan drug status in late stage pancreatic cancer
OCALA, FL / September 22, 2020 / AIM ImmunoTech Inc. (NYSE American:AIM)
AIM ImmunoTech's Ampligen extends survival in pancreatic cancer patients
Sep. 22, 2020 7:29 AM ET|About: AIM ImmunoTech Inc. (AIM)|By: Douglas W. House, SA News Editor
AYVAKIT™ (avapritinib)
Ampligen (rintatolimod)
Ampligen (rintatolimod)
Blueprint Medicines Announces Positive Top-line Results from EXPLORER and PATHFINDER Trials of AYVAKIT™ (avapritinib) in Patients with Advanced Systemic Mastocytosis
Tue September 22, 2020 7:00 AM|PR Newswire|About: BPMC
-- 75% confirmed ORR in PATHFINDER, with a median duration of response not reached --
-- AYVAKIT safety profile reinforced at 200 mg QD dose in advanced SM --
-- Plan to submit supplemental new drug application to FDA for advanced SM in fourth quarter of 2020 --
-- Blueprint Medicines to host investor conference call today at 8:30 a.m. ET --
PR Newswire
CAMBRIDGE, Mass., Sept. 22, 2020 /PRNewswire/ -- Blueprint Medicines Corporation (BPMC)
AYVAKIT is currently being evaluated in two ongoing, registrational clinical trials for advanced SM: the EXPLORER trial (ClinicalTrials.gov Identifier: NCT02561988) and the PATHFINDER trial (ClinicalTrials.gov Identifier: NCT03580655).
Additional information is available at www.BlueprintClinicalTrials.com and www.clinicaltrials.gov.
For more information, visit www.BlueprintMedicines.com
https://www.blueprintmedicines.com/pipeline/
https://seekingalpha.com/symbol/BPMC
Blueprint Medicines' avapritinib shows clinical benefit in late-stage systemic mastocytosis studies
Sep. 22, 2020 7:32 AM ET|About: Blueprint Medicines Corpo... (BPMC)|By: Vandana Singh, SA News Editor
GAVRETO™ (Pralsetinib)
Ampligen (rintatolimod)
lemzoparlimab (also known as TJC4)
Blueprint Medicines Reports ARROW Trial Data at ESMO Virtual Congress 2020 Demonstrating Durable Clinical Benefits of GAVRETO™ (Pralsetinib) in Patients with Advanced RET-Mutant Medullary Thyroid Cancer
Sun September 20, 2020 8:25 AM|PR Newswire|About: BPMC
-- Median DOR and median PFS not reached across lines of therapy --
-- New drug application under review for RET-mutant medullary thyroid cancer and RET fusion-positive thyroid cancer --
-- NCCN guidelines now include GAVRETO as a preferred treatment option for RET fusion-positive NSCLC --
PR Newswire
CAMBRIDGE, Mass., Sept. 20, 2020 /PRNewswire/ -- Blueprint Medicines Corporation (BPMC)
Blueprint Medicines' website at www.BlueprintMedicines.com.
The Phase 1/2 ARROW trial (ClinicalTrials.gov Identifier: NCT03037385) is designed to evaluate the safety, tolerability and efficacy of GAVRETO in adults with RET-altered cancers.
Additional information is available at www.BlueprintClinicalTrials.com/ARROW
https://www.blueprintmedicines.com/
https://www.blueprintclinicaltrials.com/clinical-trial/arrow/
For more information, visit www.BlueprintMedicines.com
https://seekingalpha.com/symbol/BPMC
Blueprint Medicines says durable clinical benefits observed for Gavreto in thyroid cancer
Sep. 21, 2020 11:22 AM ET|About: Blueprint Medicines Corpo... (BPMC)|By: Vandana Singh, SA News Editor
lemzoparlimab (also known as TJC4)
Moderna Announces Progress Across Broad Portfolio and all Three Clinical Stage Therapeutic Areas
lemzoparlimab (also known as TJC4)
I-Mab Announces China NMPA Clearance for Phase 1 Clinical Trial of Lemzoparlimab in Relapsed or Refractory Advanced Lymphoma
Mon September 21, 2020 8:00 AM|PR Newswire|About: IMAB
- Second IND approval from China NMPA for lemzoparlimab
PR Newswire
SHANGHAI and GAITHERSBURG, Md., Sept. 21, 2020 /PRNewswire/ -- I-Mab (IMAB) (the "Company") (Nasdaq: IMAB)
please visit http://ir.i-mabbiopharma.com
https://www.i-mabbiopharma.com/en/Pipeline.aspx
https://seekingalpha.com/symbol/IMAB
Earlier this month, I-Mab announced lemzoparlimab pact with AbbVie. I-Mab retains all rights in mainland China, Macau and Hong Kong. The collaboration also allows for potential collaboration on future CD47-related therapeutic agents.
https://seekingalpha.com/symbol/ABBV
https://www.i-mabbiopharma.com/en/
sotorasib (proposed INN for AMG 510)
Moderna Announces Progress Across Broad Portfolio and all Three Clinical Stage Therapeutic Areas
Moderna Announces Progress Across Broad Portfolio and all Three Clinical Stage Therapeutic Areas
Clinical Data From Full Phase 1 Cohort Of Investigational Sotorasib Published In New England Journal Of Medicine
Sun September 20, 2020 8:39 AM|PR Newswire|About: AMGN
NSCLC Data Featured in Proffered Presentation Session at Virtual ESMO 2020
Manuscript Represents First Phase 1 Results Published for a KRAS G12C Inhibitor
PR Newswire
THOUSAND OAKS, Calif., Sept. 20, 2020 /PRNewswire/ -- Amgen (AMGN)
Additional information about CodeBreaK clinical trials can be found at http://www.codebreaktrials.com.
please visit AmgenOncology.com. For more information
For more information, visit www.amgen.com
https://seekingalpha.com/symbol/AMGN
Amgen's sotorasib shows encouraging clinical benefit in KRAS G12C-mutant tumors
Sep. 21, 2020 5:37 AM ET|About: Amgen Inc. (AMGN)|By: Mamta Mayani, SA News Editor
Moderna Announces Progress Across Broad Portfolio and all Three Clinical Stage Therapeutic Areas
Moderna Announces Progress Across Broad Portfolio and all Three Clinical Stage Therapeutic Areas
Moderna Announces Progress Across Broad Portfolio and all Three Clinical Stage Therapeutic Areas
The company wants to ensure trust in the immunizations, the CEO says.
Covid-19 Live Updates: Drugmaker Shares Details of Its Vaccine Trial
In releasing the blueprint, Moderna hopes to earn the trust of the public and of scientists who have clamored for details of its study.
https://www.nytimes.com/2020/09/17/world/covid-19-coronavirus.html
https://seekingalpha.com/symbol/MRNA
Enrollment Update:
25,296 participants enrolled
in the COVE Phase 3 Study as of Wednesday, September 16, 2020 at 5:00 pm ET
https://www.modernatx.com/cove-study
Moderna highlights pipeline programs at R&D Day
Sep. 17, 2020 10:42 AM ET|About: Moderna, Inc. (MRNA)|By: Douglas W. House, SA News Editor
Notes from Moderna's (NASDAQ:MRNA) R&D Day event:
https://seekingalpha.com/news/3614900-moderna-highlights-pipeline-programs-r-and-d-day
Mirvetuximab Soravtansine in Combination With Carboplatin and Avastin®
Mirvetuximab Soravtansine in Combination With Carboplatin and Avastin®
Mirvetuximab Soravtansine in Combination With Carboplatin and Avastin®
ImmunoGen Presents Final Data From Phase 1b FORWARD II Triplet Cohort Evaluating Mirvetuximab Soravtansine in Combination With Carboplatin and Avastin® at ESMO
Thu September 17, 2020 3:01 AM|Business Wire|About: IMGN
Triplet Combination Demonstrates Encouraging Anti-Tumor Activity and Tolerability in Recurrent Platinum-Sensitive Ovarian Cancer
WALTHAM, Mass.--(BUSINESS WIRE)-- ImmunoGen, Inc. (IMGN), (Nasdaq: IMGN)
https://seekingalpha.com/symbol/IMGN
Learn more about who we are, what we do, and how we do it at www.immunogen.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20200917005172/en/
https://www.immunogen.com/category/mirvetuximab-soravtansine/
ImmunoGen Presents Final Data From Phase 1b FORWARD II Triplet Cohort Evaluating Mirvetuximab Soravtansine in Combination With Carboplatin and Avastin® at ESMO
September 17, 2020 at 3:01 AM EDTPDF Version
Triplet Combination Demonstrates Encouraging Anti-Tumor Activity and Tolerability in Recurrent Platinum-Sensitive Ovarian Cancer
WALTHAM, Mass.--(BUSINESS WIRE)--Sep. 17, 2020-- ImmunoGen, Inc., (Nasdaq: IMGN
LY-CoV555 is a potent, neutralizing IgG1 monoclonal antibody (mAb)
Mirvetuximab Soravtansine in Combination With Carboplatin and Avastin®
Mirvetuximab Soravtansine in Combination With Carboplatin and Avastin®
Lilly announces proof of concept data for neutralizing antibody LY-CoV555 in the COVID-19 outpatient setting
Wed September 16, 2020 6:45 AM|PR Newswire|About: LLY
- Rate of hospitalizations and ER visits was 1.7 percent (5/302) for LY-CoV555 versus 6 percent (9/150) for placebo--a 72 percent risk reduction in this limited population
- LY-CoV555 was well-tolerated across all doses with no drug-related serious adverse events reported
PR Newswire
INDIANAPOLIS, Sept. 16, 2020 /PRNewswire/ -- Eli Lilly and Company (LLY)
About BLAZE-1
BLAZE-1 (NCT04427501) is a randomized, double-blind, placebo-controlled Phase 2 study designed to assess the efficacy and safety of LY-CoV555 and LY-CoV016 for the treatment of symptomatic COVID-19 in the outpatient setting. Across all treatment arms, the trial will enroll an estimated 800 participants.
Lilly has successfully completed enrollment and primary safety assessments of LY-CoV555 in a Phase 1 study of hospitalized patients with COVID-19 (NCT04411628) and long-term follow-up is ongoing. A Phase 2 study in people recently diagnosed with COVID-19 in the ambulatory setting (NCT04427501) is ongoing. Lilly recently initiated a Phase 3 study for the prevention of COVID-19 in residents and staff at long-term care facilities (NCT04497987). In addition, LY-CoV555 is being tested in the National Institutes of Health-led ACTIV-2 and ACTIV-3 studies of ambulatory and hospitalized COVID-19 patients.
To learn more about Lilly, please visit us at www.lilly.com
https://seekingalpha.com/symbol/LLY
Etrasimod (APD334)
Mirvetuximab Soravtansine in Combination With Carboplatin and Avastin®
LY3819253 (LY-CoV555) and LY3832479 (LY-CoV016)
Arena Pharmaceuticals Announces First Subject Dosed in ELEVATE UC 12 Global Phase 3 Trial Evaluating Etrasimod in Ulcerative Colitis
Wed September 16, 2020 8:30 AM|PR Newswire|About: ARNA
- Significant unmet need for new effective oral therapies in UC
PR Newswire
SAN DIEGO, Sept. 16, 2020 /PRNewswire/ -- Arena Pharmaceuticals, Inc. (ARNA) (Nasdaq: ARNA)
ELEVATE UC 12 global Phase 3 trial evaluating etrasimod, as the potential treatment of moderately to severely active ulcerative colitis (UC).
https://www.arenapharm.com/pipeline/etrasimod/
https://seekingalpha.com/symbol/ARNA
LY3819253 (LY-CoV555) and LY3832479 (LY-CoV016)
Relugolix is a once-daily, oral gonadotropin-releasing hormone (GnRH) receptor antagonist
LY3819253 (LY-CoV555) and LY3832479 (LY-CoV016)
Lilly COVID-19 antibody shows positive effect in mid-stage study
Sep. 16, 2020 7:21 AM ET|About: Eli Lilly and Company (LLY)|By: Douglas W. House, SA News Editor
A Study of LY3819253 (LY-CoV555) and LY3832479 (LY-CoV016) in Participants With Mild to Moderate COVID-19 Illness (BLAZE-1)
https://clinicaltrials.gov/ct2/show/NCT04427501?term=ly3819253&draw=2&rank=4
Phase 2 clinical trial, BLAZE-1, evaluating LY-CoV555, a SARS-CoV-2 neutralizing antibody, in COVID-19 outpatients with mild-to-moderate symptoms.
https://seekingalpha.com/symbol/LLY
Lilly announces proof of concept data for neutralizing antibody LY-CoV555 in the COVID-19 outpatient setting
September 16, 2020
https://www.abcellera.com/news/2020-03-abcellera-and-lilly-codevelopment
Ganaxolone, a positive allosteric modulator of GABAA receptors
Relugolix is a once-daily, oral gonadotropin-releasing hormone (GnRH) receptor antagonist
Relugolix is a once-daily, oral gonadotropin-releasing hormone (GnRH) receptor antagonist
Ganaxolone Achieves Primary Endpoint in Phase 3 Trial for CDKL5 Deficiency Disorder (CDD), a Rare Form of Genetic Epilepsy
Mon September 14, 2020 4:01 PM|Business Wire|About: MRNS
- Trial met primary endpoint, median 28-day major motor seizure frequency reduction of 32.2 percent compared to 4.0 percent for placebo (p=0.002)
- Ganaxolone was generally well tolerated and the discontinuation rate in the active treatment arm was less than 5 percent
- New Drug Application (NDA) submission planned for mid-2021; commercial launch targeted for 1H 2022
- Conference call scheduled for September 14 at 4:30pm EDT
RADNOR, Pa.--(BUSINESS WIRE)-- Marinus Pharmaceuticals, Inc (MRNS). (Nasdaq: MRNS)
https://www.marinuspharma.com/
https://www.marinuspharma.com/our-science-pipeline/our-pipeline
Phase 3 clinical trial (Marigold Study) evaluating the use of oral ganaxolone in children and young adults with CDKL5 deficiency disorder (CDD), a rare, genetic epilepsy with refractory seizures.
Marinus Pharmaceuticals Receives Rare Pediatric Disease Designation from FDA for Ganaxolone for the Treatment of CDKL5 Deficiency Disorder (CDD)
RADNOR, Pa.--(BUSINESS WIRE)--July 30, 2020 -- Marinus Pharmaceuticals, Inc. (Nasdaq: MRNS)
For more information visit www.marinuspharma.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20200914005828/en/
https://seekingalpha.com/symbol/MRNS
Relugolix is a once-daily, oral gonadotropin-releasing hormone (GnRH) receptor antagonist
Relugolix is a once-daily, oral gonadotropin-releasing hormone (GnRH) receptor antagonist
Relugolix is a once-daily, oral gonadotropin-releasing hormone (GnRH) receptor antagonist
Myovant Sciences Presents Additional Data on Bone Mineral Density in Women with Uterine Fibroids from Phase 3 LIBERTY Program and from Prospective Observational Study
Mon September 14, 2020 8:30 AM|GlobeNewswire|About: MYOV
- Relugolix combination therapy maintained bone mineral density through one year, consistent with bone mineral density changes observed in untreated women
- Findings presented at the American Society for Bone and Mineral Research (ASBMR) 2020 Annual Meeting Virtual Event
BASEL, Switzerland, Sept. 14, 2020 (GLOBE NEWSWIRE) -- Myovant Sciences (MYOV)
The findings were presented at the American Society for Bone and Mineral Research (ASBMR) 2020 Annual Meeting Virtual Event, held September 11-15, 2020.
please visit our website at www.myovant.com.
https://seekingalpha.com/symbol/MYOV
LIBERTY 1 and LIBERTY 2 met the primary endpoint with 73.4% and 71.2%, respectively, of women in the treatment group achieving the responder criteria compared to 18.9% and 14.7%, respectively, of women receiving placebo at week 24 (p < 0.0001).
biotecHOPE™ July/August 2020
EPI-7386 for the Treatment of Metastatic Castration-Resistant Prostate Cancer
EPI-7386 for the Treatment of Metastatic Castration-Resistant Prostate Cancer
EPI-7386 for the Treatment of Metastatic Castration-Resistant Prostate Cancer
ESSA Pharma Announces Fast Track Designation Granted by the FDA to EPI-7386 for the Treatment of Metastatic Castration-Resistant Prostate Cancer
Mon September 14, 2020 7:00 AM|PR Newswire|About: EPIX
HOUSTON and VANCOUVER, BC, Sept. 14, 2020 /PRNewswire/ - ESSA Pharma Inc. (NASDAQ: EPIX) (TSXV: EPI)
For more information, please visit www.essapharma.com
https://www.essapharma.com/pipeline/
investigational anti-MOSPD2 (motile sperm domain-containing protein 2) mAbs
EPI-7386 for the Treatment of Metastatic Castration-Resistant Prostate Cancer
EPI-7386 for the Treatment of Metastatic Castration-Resistant Prostate Cancer
VBL +14% on encouraging antibody activity in multiple sclerosis
Sep. 11, 2020 9:29 AM ET|About: Vascular Biogenics Ltd. (VBLT)|By: Mamta Mayani, SA News Editor
https://seekingalpha.com/symbol/VBLT
https://www.vblrx.com/vb-600-platform/targeting-mospd2-for-inflammation/
VBL Presents Human Proof-of-Concept Data That Show the Potential of its Novel anti-MOSPD2 Monoclonal Antibodies for Multiple Sclerosis at the MS Virtual 2020 Meeting
September 11, 2020
TEL AVIV, Israel, Sept. 11, 2020 (GLOBE NEWSWIRE) -- VBL Therapeutics (Nasdaq: VBLT)
https://seekingalpha.com/symbol/VBLT
BIVV001 in people with severe hemophilia A
EPI-7386 for the Treatment of Metastatic Castration-Resistant Prostate Cancer
STRO-002 is a folate receptor alpha (FolRα)-targeting ADC
New England Journal of Medicine publishes positive final results from Phase 1/2a study of BIVV001 in people with severe hemophilia A
Thu September 10, 2020 1:00 AM|GlobeNewswire|About: SNY
- BIVV001 is the first investigational factor VIII therapy independent of von Willebrand Factor and has the potential to transform replacement therapy for people with hemophilia A
- It is uniquely designed to deliver near-normal factor activity levels for the majority of the week, extending bleed protection in a once-weekly dose
- Results from the Phase 1/2a study showed that a single dose of BIVV001 achieved high sustained factor activity and a three- to four-fold increase in half-life when compared to conventional factor VIII replacement therapies
PARIS and STOCKHOLM – September 10, 2020 –The New England Journal of Medicine today published positive final results from the Phase 1/2a trial evaluating the safety, tolerability and pharmacokinetics of BIVV001 (rFVIIIFc-VWF-XTEN) in adult patients with severe hemophilia A. BIVV001 is an investigational factor VIII therapy designed to provide higher bleed protection in a once-weekly prophylactic treatment regimen. Sanofi (SNY) and Sobi™ (STO:SOBI) collaborate on the development and commercialization of BIVV001.
BIVV001 Fusion Protein as Factor VIII Replacement Therapy for Hemophilia A
https://www.nejm.org/doi/full/10.1056/NEJMoa2002699
You can find more information about Sobi at www.sobi.com.
https://seekingalpha.com/symbol/SNY
STRO-002 is a folate receptor alpha (FolRα)-targeting ADC
STRO-002 is a folate receptor alpha (FolRα)-targeting ADC
STRO-002 is a folate receptor alpha (FolRα)-targeting ADC
Sutro Biopharma Presents Promising STRO-002 Interim Phase 1 Clinical Data in Ovarian Cancer at the 2020 IGCS Annual Global Meeting
Wed September 9, 2020 4:01 PM|PR Newswire|About: STRO
- Conference Call Today Scheduled for 5:30 p.m. Eastern Time
PR Newswire
SOUTH SAN FRANCISCO, Calif., Sept. 9, 2020 /PRNewswire/ -- Sutro Biopharma, Inc. (STRO)
The trial is registered with clinicaltrials.gov identifier NCT03748186.
https://www.sutrobio.com/pipeline/
https://seekingalpha.com/symbol/STRO
Study of STRO-002, an Anti-Folate Receptor Alpha (FolRα) Antibody Drug Conjugate in Ovarian & Endometrial Cancers
https://clinicaltrials.gov/ct2/show/NCT03748186?term=sutro+biopharma&draw=2&rank=1
Leronlimab (PRO 140)
STRO-002 is a folate receptor alpha (FolRα)-targeting ADC
Leronlimab (PRO 140)
CytoDyn to discuss accelerated approval of leronlimab for COVID-19 with UK health authorities September 9
Sep. 2, 2020 4:31 PM ET|About: CytoDyn Inc. (CYDY)|By: Douglas W. House, SA News Editor
U.K. MHRA Grants Meeting to CytoDyn to Discuss Fast Track Approval of Leronlimab for COVID-19 Patients
Wed September 2, 2020 4:01 PM|GlobeNewswire|About: CYDY
U.S. FDA schedules Type A meeting with CytoDyn (CYDY) to discuss BLA filing for HIV
VANCOUVER, Washington, Sept. 02, 2020 (GLOBE NEWSWIRE) -- CytoDyn Inc.
https://seekingalpha.com/symbol/CYDY
More information is at www.cytodyn.com.
https://www.cytodyn.com/pipeline/covid-19
U.K. MHRA Grants Meeting to CytoDyn to Discuss Fast Track Approval of Leronlimab for COVID-19 Patients
Download as PDFSeptember 02, 2020 4:01pm EDT
U.S. FDA schedules Type A meeting with CytoDyn to discuss BLA filing for HIV
VANCOUVER, Washington, Sept. 02, 2020 (GLOBE NEWSWIRE) -- CytoDyn Inc. (OTC.QB: CYDY),
Lenzilumab
STRO-002 is a folate receptor alpha (FolRα)-targeting ADC
Leronlimab (PRO 140)
Lenzilumab COVID-19 Case-Control Study Published in Mayo Clinic Proceedings Journal
Tue September 1, 2020 7:00 AM|Business Wire|About: HGEN
- 80% reduction in relative risk of invasive mechanical ventilation and/or death in patients treated with lenzilumab compared to the control group
- Median time to resolution of acute respiratory distress syndrome (ARDS) reduced to one day for patients treated with lenzilumab versus eight days in control group
- Lenzilumab patients discharged from the hospital in less than half the time compared with control group
BURLINGAME, Calif.--(BUSINESS WIRE)-- Humanigen, Inc. (HGEN)
https://www.businesswire.com/news/home/20200901005342/en/
https://www.humanigen.com/pipeline
Humanigen's lenzilumab shows positive effect in COVID-19 in case-control study
Sep. 1, 2020 7:59 AM ET|About: Humanigen, Inc. (HGEN)|By: Douglas W. House, SA News Editor
https://seekingalpha.com/symbol/HGEN
Lenzilumab COVID-19 Case-Control Study Published in Mayo Clinic Proceedings Journal
September 1, 2020
- 80% reduction in relative risk of invasive mechanical ventilation and/or death in patients treated with lenzilumab compared to the control group
- Median time to resolution of acute respiratory distress syndrome (ARDS) reduced to one day for patients treated with lenzilumab versus eight days in control group
- Lenzilumab patients discharged from the hospital in less than half the time compared with control group
Burlingame, CA, August 31, 2020 – Humanigen, Inc., (HGEN)
PRV-015 (also known as AMG 714)
Phase II study of LNP023 in patients with paroxysmal nocturnal hemoglobinuria (PNH)
Phase II study of LNP023 in patients with paroxysmal nocturnal hemoglobinuria (PNH)
Provention Bio Announces Initiation of Phase 2b PROACTIVE Study of PRV-015 (anti-IL-15) in Non-responsive Celiac Disease
Mon August 31, 2020 7:30 AM|PR Newswire|About: PRVB
PRV-015 (anti-interleukin-15) is the first investigational product to show both a reduction in gluten-induced symptoms and in markers of intestinal inflammation in a placebo-controlled trial in celiac subjects
PR Newswire
OLDWICK, N.J., Aug. 31, 2020 /PRNewswire/ -- Provention Bio, Inc. (PRVB)
More information on the study is available at https://proactiveceliac.com/.
https://seekingalpha.com/symbol/PRVB
https://proventionbio.com/pipeline/
Provention Bio Announces Initiation of Phase 2b PROACTIVE Study of PRV-015 (anti-IL-15) in Non-responsive Celiac DiseasePotential to be the first approved therapeutic for the treatment of Non-responsive Celiac DiseasePRV-015 (anti-interleukin-15) is the first investigational product to show both a reduction in gluten-induced symptoms and in markers of intestinal inflammation in a placebo-controlled trial in celiac subjects
OLDWICK, N.J., Aug. 31, 2020 /PRNewswire/ -- Provention Bio, Inc. (Nasdaq: PRVB
Phase II study of LNP023 in patients with paroxysmal nocturnal hemoglobinuria (PNH)
Phase II study of LNP023 in patients with paroxysmal nocturnal hemoglobinuria (PNH)
Phase II study of LNP023 in patients with paroxysmal nocturnal hemoglobinuria (PNH)
LNP023 as an add-on to Alexion's Soliris (eculizumab)
Novartis announces positive results from Phase II study of LNP023 in patients with paroxysmal nocturnal hemoglobinuria (PNH)
Aug 29, 2020
- Oral, investigational complement factor B inhibitor LNP023 substantially improved hematological response as add-on therapy to eculizumab
- Seven of ten patients discontinued eculizumab and remained on LNP023 as monotherapy, retaining hemoglobin (Hb) levels with no changes in biomarkers of disease activity and with no signs or symptoms of breakthrough hemolysis
- Phase II results are promising for treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare and life-threatening blood disorder;1,2 second Phase II study with LNP023 monotherapy in anti-C5 naïve PNH patients ongoing
- First-in-class LNP023 – which potently and selectively targets factor B, part of the immune system’s alternative complement pathway3,4,5 – is also in development for several renal conditions with complement system involvement
- FDA and EMA have granted orphan drug designations to LNP023 for PNH and the rare renal disease complement 3 glomerulopathy (C3G)
Basel, August 29, 2020 —
Alexion up 8% on Soliris performance bump from Novartis' LNP023
Aug. 31, 2020 11:37 AM ET|About: Alexion Pharmaceutical... (ALXN)|By: Douglas W. House, SA News Editor
https://seekingalpha.com/news/3610155-alexion-up-8-on-soliris-performance-bump-from-novartis-lnp023
https://seekingalpha.com/symbol/ALXN
https://seekingalpha.com/symbol/NVS
BCX9930
Phase II study of LNP023 in patients with paroxysmal nocturnal hemoglobinuria (PNH)
Aldafermin (formerly NGM282)
FDA Grants Orphan Drug Designation for BCX9930 in PNH
Mon August 31, 2020 7:00 AM|GlobeNewswire|About: BCRX
RESEARCH TRIANGLE PARK, N.C., Aug. 31, 2020 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (BCRX)
Further information about PNH is available from the National Institutes of Health at https://ghr.nlm.nih.gov/condition/paroxysmal-nocturnal-hemoglobinuria
For more information, please visit the Company's website at www.BioCryst.com.
https://seekingalpha.com/symbol/BCRX
https://www.biocryst.com/our-program/
FDA Grants Orphan Drug Designation for BCX9930 in PNH
RESEARCH TRIANGLE PARK, N.C., Aug. 31, 2020 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX)
Aldafermin (formerly NGM282)
Investigational Hepatitis B Therapeutic JNJ-3989
Aldafermin (formerly NGM282)
NGM Bio Presents Comprehensive Findings from 24-Week Phase 2 Study (Cohort 4) of Aldafermin in Oral Plenary Presentation at The Digital International Liver Congress™ (ILC) 2020 and Announces Enrollment Completed in Phase 2b ALPINE 2/3 Study
Sat August 29, 2020 7:20 AM|GlobeNewswire|About: NGM
- Late-breaker presentation selected as “Best of ILC”
- The 24-week Phase 2 study met its primary endpoint, achieving a statistically significant reduction in liver fat content (LFC), and robust fibrosis improvement and resolution of NASH, with a favorable safety profile
- New analysis shows 30% placebo corrected anti-fibrotic response rate among NASH patients with more advanced liver fibrosis (F3)
- NGM Bio anticipates Phase 2b ALPINE 2/3 study topline data readout in Q2 2021
SOUTH SAN FRANCISCO, Calif., Aug. 29, 2020 (GLOBE NEWSWIRE) -- NGM Biopharmaceuticals, Inc. (NGM)
Evaluation of Efficacy, Safety and Tolerability of Aldafermin in a Phase 2b, Randomized, Double-blind, Placebo-controlled, Multi-center Study In Subjects With Nonalcoholic Steatohepatitis and Stage 2/3 Fibrosis (ALPINE 2/3)
https://clinicaltrials.gov/ct2/show/NCT03912532?lead=ngm+biopharmaceuticals
Aldafermin (formerly NGM282) is an engineered variant of a hormone called fibroblast growth factor 19 (FGF19) secreted by the gall bladder that plays a key role protecting the liver.
NGM Bio's aldafermin shows positive effect in NASH patients with advanced disease
Aug. 31, 2020 8:25 AM ET|About: NGM Biopharmaceuticals... (NGM)|By: Douglas W. House, SA News Editor
AUGUST 29, 2020 AT 7:20 AM EDT
NGM Bio Presents Comprehensive Findings from 24-Week Phase 2 Study (Cohort 4) of Aldafermin in Oral Plenary Presentation at The Digital International Liver Congress™ (ILC) 2020 and Announces Enrollment Completed in Phase 2b ALPINE 2/3 Study
https://seekingalpha.com/symbol/NGM
ARO-APOC3 and ARO-ANG3
Investigational Hepatitis B Therapeutic JNJ-3989
Investigational Hepatitis B Therapeutic JNJ-3989
Arrowhead Presents Positive New Phase 1/2 Clinical Data on Cardiometabolic Candidates ARO-APOC3 and ARO-ANG3 at European Society of Cardiology Congress 2020
Mon August 31, 2020 4:50 AM|Business Wire|About: ARWR
PASADENA, Calif.--(BUSINESS WIRE)-- Arrowhead Pharmaceuticals Inc. (ARWR)
A copy of the presentation materials may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website.
View source version on businesswire.com: https://www.businesswire.com/news/home/20200831005208/en/
https://arrowheadpharma.com/pipeline/
Arrowhead Presents Positive New Phase 1/2 Clinical Data on Cardiometabolic Candidates ARO-APOC3 and ARO-ANG3 at European Society of Cardiology Congress 2020
Aug 31, 2020 at 4:50 AM EDTPASADENA, Calif. --(BUSINESS WIRE)--Aug. 31, 2020-- Arrowhead Pharmaceuticals Inc. (NASDAQ: ARWR)
https://seekingalpha.com/symbol/ARWR
Investigational Hepatitis B Therapeutic JNJ-3989
Investigational Hepatitis B Therapeutic JNJ-3989
Investigational Hepatitis B Therapeutic JNJ-3989
Arrowhead and Collaborator Janssen Present Phase 2 Clinical Data on Investigational Hepatitis B Therapeutic JNJ-3989 at The Digital Liver Congress
Fri August 28, 2020 7:30 AM|Business Wire|About: ARWR
PASADENA, Calif.--(BUSINESS WIRE)-- Arrowhead Pharmaceuticals Inc. (ARWR)
For more information, please visit www.arrowheadpharma.com
https://www.businesswire.com/news/home/20200828005039/en/
Target the Gene
Silence the Disease
Aug 18, 2020 -Arrowhead Pharmaceuticals to Participate in Upcoming August 2020 ConferencesView More »Oct 18, 2019 8:00 AM EDT
Arrowhead Analyst R&D Day 2019View
https://arrowheadpharma.com/pipeline/
https://seekingalpha.com/symbol/ARWR
https://seekingalpha.com/symbol/JNJ
second part of a Phase 1/2 clinical trial evaluating RNAi therapeutic candidate JNJ-3989 (formerly ARO-HBV)
Janssen/Arrowhead RNAi candidate shows positive action in early-stage hepatitis B study
Aug. 28, 2020 9:18 AM ET|About: Arrowhead Pharmaceutic... (ARWR)|By: Douglas W. House, SA News Editor
biotecHOPE™ July/August 2020
CUTX-101 (Copper Histidinate)
RP-L401 for Fast Track review for Infantile Malignant Osteopetrosis (IMO)
RP-L401 for Fast Track review for Infantile Malignant Osteopetrosis (IMO)
Fortress Biotech Announces Positive Topline Clinical Efficacy Results for CUTX-101, Copper Histidinate, for the Treatment of Menkes Disease
Fri August 28, 2020 8:30 AM|GlobeNewswire|About: FBIO
Statistically Significant Improvement in the Primary Endpoint of Overall Survival in Menkes Disease Patients who Received Early Treatment with CUTX-101, Compared to an Untreated Historical Control, with a Nearly 80% Reduction in the Risk of Death (Hazard Ratio = 0.21, p<0.0001)
Rolling Submission of New Drug Application to the FDA for CUTX-101 on Track to Begin in the Fourth Quarter of 2020
NEW YORK, Aug. 28, 2020 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (FBIO)
https://www.fortressbiotech.com/
CUTX-101 (Copper Histidinate)
https://www.cypriumtx.com/pipeline/cutx-101-copper-histidinate/
https://www.fortressbiotech.com/programs
Molecular Bases of Response to Copper Treatment in Menkes Disease, Related Phenotypes, and Unexplained Copper Deficiency
https://clinicaltrials.gov/ct2/show/NCT00811785
Copper Histidinate Treatment for Menkes Disease
https://clinicaltrials.gov/ct2/show/NCT04074512
Cyprium Therapeutics' copper histidinate shows positive effect in kids with rare copper disorder
Aug. 28, 2020 9:49 AM ET|About: Fortress Biotech, Inc. (FBIO)|By: Douglas W. House, SA News Editor
https://seekingalpha.com/symbol/FBIO
For more information, visit www.fortressbiotech.com.
RP-L401 for Fast Track review for Infantile Malignant Osteopetrosis (IMO)
RP-L401 for Fast Track review for Infantile Malignant Osteopetrosis (IMO)
RP-L401 for Fast Track review for Infantile Malignant Osteopetrosis (IMO)
Rocket Pharmaceuticals Receives FDA Fast Track Designation for RP-L401 Gene Therapy for Infantile Malignant Osteopetrosis
Thu August 27, 2020 7:00 AM|Business Wire|About: RCKT
—Rocket’s Fifth Gene Therapy Program Receives Fast Track Designation—
NEW YORK--(BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (RCKT)
For more information about Rocket, please visit www.rocketpharma.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20200827005255/en/
https://seekingalpha.com/symbol/RCKT
RP-L401 was in-licensed from Lund University and Medizinische Hochschule Hannover.
Current Portfolio
Rocket’s pipeline is comprised of first-in-class gene therapies for rare and devastating inherited diseases.
https://www.rocketpharma.com/pipeline/
Rocket Pharmaceuticals Receives FDA Fast Track Designation for RP-L401 Gene Therapy for Infantile Malignant Osteopetrosis
Aug 27,2020
Moderna Vaccine Produces Antibodies in Trial of Older People
RP-L401 for Fast Track review for Infantile Malignant Osteopetrosis (IMO)
Moderna Vaccine Produces Antibodies in Trial of Older People
Prognosis
Moderna Vaccine Produces Antibodies in Trial of Older People
By Robert LangrethAugust 26, 2020, 9:28 AM EDT Updated on August 26, 2020, 12:25 PM EDT
- New data from early-stage trial presented to CDC advisors
- High antibody levels to virus seen in older adults after shots
Moderna to Present New Interim Clinical Data About mRNA Vaccine Against COVID-19 (mRNA-1273) at Advisory Committee on Immunization Practices (ACIP) Meeting
August 26, 2020 at 8:53 AM EDTPDF Version
Following the ACIP presentation today at 10:30 a.m. ET, management will host a conference call to be held today at 4:30 p.m. ET
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Aug. 26, 2020-- Moderna, Inc., (Nasdaq: MRNA)
https://seekingalpha.com/symbol/MRNA
Enrollment Update:
13,194 participants enrolled
in the COVE Phase 3 Study as of Friday, August 21, 2020 at 5:00 pm ET
https://www.modernatx.com/cove-study
glucose kinase activator (AZD1656)
glucose kinase activator (AZD1656)
Moderna Vaccine Produces Antibodies in Trial of Older People
25-Aug-2020
FIRST PATIENT DOSED WITH POTENTIAL NEW THERAPEUTIC FOR TREATMENT OF COVID-19 IN DIABETES SUFFERERS
A new therapy that could treat people with diabetes suffering from COVID-19 is to undergo an advanced clinical trial in the UK. The first patient will be dosed this week.
https://openinnovation.astrazeneca.com/
UK study to assess AstraZeneca drug in diabetic COVID-19 patients
Aug. 25, 2020 11:59 AM ET|About: AstraZeneca PLC (AZN)|By: Douglas W. House, SA News Editor
https://seekingalpha.com/symbol/AZN
Soticlestat (TAK-935/OV935)
glucose kinase activator (AZD1656)
Soticlestat (TAK-935/OV935)
Phase 2 ELEKTRA Study of Soticlestat (TAK-935/OV935) Meets Primary Endpoint Reducing Seizure Frequency in Children with Dravet Syndrome or Lennox-Gastaut Syndrome
Tue August 25, 2020 7:00 AM|Business Wire|About: OVID, TAK
− Primary endpoint achieved, demonstrating a statistically significant reduction of seizures from baseline compared to placebo (p=0.0007) in the combined Dravet syndrome and Lennox-Gastaut syndrome study population
− Statistically significant reduction in seizure frequency from baseline in Dravet syndrome cohort compared to placebo (p=0.0007); based on strong efficacy results, Takeda (TKPHF) and Ovid plan to initiate a Phase 3 registrational program of soticlestat in Dravet syndrome
− Data from Lennox-Gastaut syndrome cohort demonstrated numeric reductions in seizure frequency compared to placebo but did not achieve statistical significance (p=0.1279); data analysis ongoing for the Lennox-Gastaut syndrome patients
− Soticlestat was well-tolerated and demonstrated a safety profile consistent with the findings of previous studies with no new safety signals identified
− Ovid to host conference call and webcast today at 8:00 a.m. EDT
NEW YORK & OSAKA, Japan--(BUSINESS WIRE)-- Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (“Takeda”) and Ovid Therapeutics Inc. (OVID) (NASDAQ: OVID) (“Ovid”)
View the full release here: https://www.businesswire.com/news/home/20200825005303/en/
For more information, visit https://www.takeda.com.
https://seekingalpha.com/symbol/TAK
For more information on Ovid, please visit www.ovidrx.com.
https://seekingalpha.com/symbol/OVID
View source version on businesswire.com: https://www.businesswire.com/news/home/20200825005303/en/
Ovid's soticlestat successful in mid-stage study in severe forms of childhood epilepsy
Aug. 25, 2020 8:14 AM ET|About: Ovid Therapeutics Inc. (OVID)|By: Douglas W. House, SA News Editor
Phase 2 clinical trial, ELEKTRA, evaluating soticlestat in children with Dravet syndrome (DS) or Lennox-Gastaut syndrome (LGS), rare, treatment-resistant, severe forms of epilepsy.
CDX-527 in Solid Tumors
glucose kinase activator (AZD1656)
Soticlestat (TAK-935/OV935)
Celldex Therapeutics Initiates Phase 1 Study of New Bispecific Product Candidate CDX-527 in Solid Tumors
Mon August 24, 2020 8:01 AM|GlobeNewswire|About: CLDX
HAMPTON, N.J., Aug. 24, 2020 (GLOBE NEWSWIRE) -- Celldex Therapeutics, Inc. (CLDX)
More information about this study is available on www.clinicaltrials.gov (Identifier: NCT04440943).
Aug 24, 2020
Celldex Therapeutics Initiates Phase 1 Study of New Bispecific Product Candidate CDX-527 in Solid Tumors
HAMPTON, N.J., Aug. 24, 2020 (GLOBE NEWSWIRE) -- Celldex Therapeutics, Inc. (Nasdaq:CLDX)
https://seekingalpha.com/symbol/CLDX
Celldex launches early-stage study for bispecific candidate in solid tumors
Aug. 24, 2020 1:28 PM ET|About: Celldex Therapeutics, Inc. (CLDX)|By: Vandana Singh, SA News Editor
COVID-19 vaccine candidate, Ad26.COV2.S
COVID-19 vaccine candidate, Ad26.COV2.S
COVID-19 vaccine candidate, Ad26.COV2.S
For Covid-19 Vaccine, J&J Plans 60,000-Subject Pivotal Trial
The planned phase 3 trial would have double the number of subjects expected to enroll in late-stage studies of other vaccines
By Peter Loftus
Updated Aug. 20, 2020 3:56 pm ET
J&J planning 60K-subject study to test COVID-19 vaccine
Aug. 20, 2020 11:31 AM ET|About: Johnson & Johnson (JNJ)|By: Douglas W. House, SA News Editor
https://seekingalpha.com/symbol/JNJ
Leronlimab (PRO 140)
COVID-19 vaccine candidate, Ad26.COV2.S
COVID-19 vaccine candidate, Ad26.COV2.S
CytoDyn Submits its Top-line Report from its Phase 2 COVID-19 Trial to the U.S. FDA and Requests Emergency Use Approval
Mon August 17, 2020 6:00 AM|GlobeNewswire|About: CYDY
The Top-line Report has been sent to the regulatory authorities in Mexico, and will be provided to U.K. MHRA, and E.U. EMA, with requests for emergency use approval
CytoDyn (CYDY) is preparing a Phase 3 protocol for leronlimab use in long-hauler COVID-19 individuals
VANCOUVER, Washington, Aug. 17, 2020 (GLOBE NEWSWIRE) -- CytoDyn Inc.,
CytoDyn files for emergency use of leronlimab for COVID-19
Aug. 17, 2020 6:12 AM ET|About: CytoDyn Inc. (CYDY)|By: Douglas W. House, SA News Editor
https://seekingalpha.com/symbol/CYDY
https://www.cytodyn.com/pipeline
atuzaginstat (COR388)
COVID-19 vaccine candidate, Ad26.COV2.S
Viltepso (viltolarsen)
Cortexyme Announces GAIN Trial of Atuzaginstat in Alzheimer’s Disease Has Reached Enrollment Milestone of 500 Patients
Fri August 14, 2020 8:00 AM|Business Wire|About: CRTX
- Open label extension is currently active for GAIN Trial completers in the United States with robust conversion of eligible patients
- Interim analysis of the Phase 2/3 GAIN Trial expected to occur before year-end 2020
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Cortexyme, Inc. (CRTX)
For more information on the trial, visit www.gaintrial.com.
o learn more about Cortexyme, visit www.cortexyme.com
https://www.businesswire.com/news/home/20200814005238/en/
Cortexyme Announces GAIN Trial of Atuzaginstat in Alzheimer’s Disease Has Reached Enrollment Milestone of 500 Patients
August 14, 2020PDF Version
- Open label extension is currently active for GAIN Trial completers in the United States with robust conversion of eligible patients
- Interim analysis of the Phase 2/3 GAIN Trial expected to occur before year-end 2020
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Aug. 14, 2020-- Cortexyme, Inc. (Nasdaq: CRTX)
Cortexyme advancing study of lead drug in Alzheimer's
Aug. 14, 2020 8:27 AM ET|About: Cortexyme, Inc. (CRTX)|By: Douglas W. House, SA News Editor
https://seekingalpha.com/news/3605694-cortexyme-advancing-study-of-lead-drug-in-alzheimers
https://seekingalpha.com/symbol/CRTX
Viltepso (viltolarsen)
investigational cannabinoid formulation - IGC-AD1
Viltepso (viltolarsen)
FDA Approves Targeted Treatment for Rare Duchenne Muscular Dystrophy Mutation
Wed August 12, 2020 2:16 PM|PR Newswire
SILVER SPRING, Md., Aug. 12, 2020 /PRNewswire/
Muscular Dystrophy Information Page
https://www.ninds.nih.gov/Disorders/All-Disorders/Muscular-Dystrophy-Information-Page
Additional Resources:
NS Pharma’s VILTEPSO™ (viltolarsen) injection Now FDA-Approved in the U.S. for the Treatment of Duchenne Muscular Dystrophy in Patients Amenable to Exon 53 Skipping Therapy Patients taking VILTEPSO showed an increase in dystrophin expression to an average of 5.9% of normal after 20-24 weeks of treatment. Overall, in a pivotal study of VILTEPSO 100% of patients showed an increase in dystrophin levels after treatment and 88% of patients showed dystrophin levels of 3% of normal or greater. PARAMUS, NJ: August 12, 2020 – NS Pharma, Inc. (NS Pharma; President, Tsugio Tanaka)
https://www.nspharma.com/pdfs/Viltepso_Approval_Press_Release.pdf
ofranergene obadenovec (VB-111)
investigational cannabinoid formulation - IGC-AD1
investigational cannabinoid formulation - IGC-AD1
VBL Therapeutics Announces Second Successful Pre-planned Interim Analysis with a Positive Data Safety Monitoring Committee Review Looking at OS - the Primary Endpoint of the OVAL Phase 3 Potential Registration Study of VB-111 in Ovarian Cancer
Wed August 12, 2020 8:00 AM|GlobeNewswire|About: VBLT
TEL AVIV, Israel, Aug. 12, 2020 (GLOBE NEWSWIRE) -- VBL Therapeutics (Nasdaq: VBLT)
About the OVAL study (NCT03398655)
Phase 2 clinical trials in radioiodine-refractory thyroid cancer and recurrent platinum-resistant ovarian cancer (NCT01711970).
https://seekingalpha.com/symbol/VBLT
Phase 3 ((OVAL)) trial in platinum-resistant ovarian cancer and recommended that the study continue unmodified.
investigational cannabinoid formulation - IGC-AD1
investigational cannabinoid formulation - IGC-AD1
investigational cannabinoid formulation - IGC-AD1
FDA Approves Initiation of IGC’s Cannabinoid Trial on Alzheimer’s Patients
Tue August 11, 2020 5:03 PM|Business Wire|About: IGC
IGC Announces FDA Removal of Clinical Hold for Multiple Ascending Dose Study of IGC-AD1, Targeting Patients Suffering from Alzheimer’s-related Dementia
POTOMAC, Md.--(BUSINESS WIRE)-- India Globalization Capital (IGC) (NYSE American: IGC)
https://www.businesswire.com/news/home/20200811005841/en/
https://www.igcpharma.com/alzheimers
https://seekingalpha.com/symbol/IGC
FDA gives green signal to IGC's cannabinoid early-stage trial in Alzheimer’s disease
Aug. 11, 2020 5:32 PM ET|About: India Globalization Capit... (IGC)|By: Vandana Singh, SA News Editor
biotecHOPE™ July/August 2020
LTX-315 is an oncolytic peptide is injected directly into a tumor to induce immunogenic cell death
LTX-315 is an oncolytic peptide is injected directly into a tumor to induce immunogenic cell death
LTX-315 is an oncolytic peptide is injected directly into a tumor to induce immunogenic cell death
Verrica Pharmaceuticals Licenses LTX-315, A First-in-Class Oncolytic Peptide Based Immunotherapy, from Lytix Biopharma AS, for the Treatment of Dermatologic Oncology Indications
Tue August 11, 2020 7:00 AM|GlobeNewswire|About: VRCAQ2: 08-05-20 Earnings Summary
WEST CHESTER, Pa., Aug. 11, 2020 (GLOBE NEWSWIRE) -- Verrica Pharmaceuticals Inc. (VRCA)(“Verrica”) (NASDAQ: VRCA)
For more information, visit www.verrica.com.
https://verrica.com/pipeline-vp-102/
http://www.lytixbiopharma.com/
http://www.lytixbiopharma.com/research-development/pipeline.html
Verrica Pharma in-licenses Lytix Bio's LTX-315 for dermatologic cancers
Aug. 11, 2020 8:46 AM ET|About: Verrica Pharmaceuticals Inc. (VRCA)|By: Mamta Mayani, SA News Editor
https://seekingalpha.com/symbol/VRCA
For more information, visit www.lytixbiopharma.com.
Key Statistics for Basal and Squamous Cell Skin Cancers
https://www.cancer.org/cancer/basal-and-squamous-cell-skin-cancer/about/key-statistics.html
Leronlimab (PRO 140)
LTX-315 is an oncolytic peptide is injected directly into a tumor to induce immunogenic cell death
LTX-315 is an oncolytic peptide is injected directly into a tumor to induce immunogenic cell death
CytoDyn Announces Clinically Significant Top-line Results from its Phase 2 Trial in Mild-to-Moderate COVID-19 Patients
Tue August 11, 2020 9:15 AM|GlobeNewswire|About: CYDY
Primary endpoint shows early clinical improvement in symptom score at Day 3 in patients receiving leronlimab
Leronlimab also demonstrated statistically significant improvement versus placebo in key secondary efficacy endpoint, National Early Warning Score 2 scale (NEWS2)
Results will be reported to the United States FDA, United Kingdom MHRA, and European Union regulatory agency, EMA
Management to hold conference call on August 12 at 1:00 pm PT - - details to follow
VANCOUVER, Washington, Aug. 11, 2020 (GLOBE NEWSWIRE) -- CytoDyn Inc. (CYDY)
https://www.cytodyn.com/pipeline/hiv
More information is at www.cytodyn.com.
CytoDyn announces "positive" results from mid-stage COVID-19 study
Aug. 11, 2020 9:51 AM ET|About: CytoDyn Inc. (CYDY)|By: Douglas W. House, SA News Editor
https://seekingalpha.com/symbol/CYDY
Study to Evaluate the Efficacy and Safety of Leronlimab for Mild to Moderate COVID-19
https://clinicaltrials.gov/ct2/show/NCT04343651?cond=Covid19&lead=cytodyn&phase=12&draw=2&rank=1
CytoDyn bullish on leronlimab in COVID-19 based on success of objective endpoint
Aug. 12, 2020 4:23 PM ET|About: CytoDyn Inc. (CYDY)|By: Douglas W. House, SA News Editor
CS1001 (Anti-PD-L1 mAb)
LTX-315 is an oncolytic peptide is injected directly into a tumor to induce immunogenic cell death
PRS-343 With Ramucirumab and Paclitaxel in Gastric Cancer
CStone antibody successful in late-stage lung cancer study
Aug. 7, 2020 9:24 AM ET|About: CSTONE PHARMACEUTICALS (CSPHF)|By: Douglas W. House, SA News Editor
A Study of CS1001 in Subjects With Stage IV Non-Small Cell Lung Cancer
https://clinicaltrials.gov/ct2/show/NCT03789604?lead=cstone+pharmaceuticals&phase=2&draw=2&rank=4
A Phase 3 clinical trial evaluating CStone Pharmaceuticals' (OTCPK:CSPHF) PD-L1 inhibitor CS1001 for the first-line treatment of metastatic squamous and nonsquamous non-small cell lung cancer (NSCLC) met the primary endpoint of progression-free survival (PFS).
http://www.cstonepharma.com/en/
http://www.cstonepharma.com/en/develop/p3.html
CStone Announces CS1001 (Anti-PD-L1 mAb) Phase III Trial Met the Primary Endpoint as First-Line Treatment in Stage IV Squamous and Non-squamous Non-Small Cell Lung Cancer and Plans to Submit a New Drug Application
Times：2020.08.06 Author：CStone
- First anti-PD-L1 mAb to demonstrate overwhelming efficacy as 1L treatment of Stage IV squamous and non-squamous NSCLC in a randomized, double-blind phase III trial
PRS-343 With Ramucirumab and Paclitaxel in Gastric Cancer
Narsoplimab, also known as “OMS721,” is an investigational human monoclonal antibody
PRS-343 With Ramucirumab and Paclitaxel in Gastric Cancer
Pieris and Lilly Enter Into a Clinical Trial Collaboration to Evaluate Combination of PRS-343 With Ramucirumab and Paclitaxel in Gastric Cancer
Mon August 10, 2020 6:30 AM|Accesswire|About: PIRS
BOSTON, MA / ACCESSWIRE / August 10, 2020 / Pieris Pharmaceuticals, Inc. (PIRS),
For more information, visit www.pieris.com.
https://seekingalpha.com/symbol/PIRS
https://seekingalpha.com/symbol/LLY
Narsoplimab, also known as “OMS721,” is an investigational human monoclonal antibody
Narsoplimab, also known as “OMS721,” is an investigational human monoclonal antibody
Narsoplimab, also known as “OMS721,” is an investigational human monoclonal antibody
Omeros Corporation Reports Recovery and Survival of All Patients in Study Evaluating Narsoplimab for Treatment of COVID-19-Associated Acute Respiratory Distress Syndrome
Mon August 10, 2020 7:30 AM|Business Wire|About: OMER
-- All six patients, requiring mechanical ventilation prior to treatment, recovered, survived and were discharged from the hospital
-- Narsoplimab treatment was associated with rapid and sustained improvement across all assessed markers of endothelial/cellular damage and/or inflammation
-- Omeros is in discussions with U.S. government agencies regarding acceleration of narsoplimab manufacturing for use in COVID-19 patients
-- Conference call and webcast today at 8:30 a.m. ET, 5:30 a.m. PT
SEATTLE--(BUSINESS WIRE)-- Omeros Corporation (OMER)
Endothelial Injury and Thrombotic Microangiopathy in COVID-19: Treatment with the Lectin-Pathway Inhibitor Narsoplimab
https://www.sciencedirect.com/science/article/pii/S0171298520304459
https://www.omeros.com/omidria/
https://www.businesswire.com/news/home/20200810005280/en/
https://seekingalpha.com/symbol/OMER
Favipiravir is an antiviral
Narsoplimab, also known as “OMS721,” is an investigational human monoclonal antibody
Narsoplimab, also known as “OMS721,” is an investigational human monoclonal antibody
FDA Clears Appili Therapeutics to Expand its Phase 2 Clinical Trial of Favipiravir for the Potential Prevention of COVID-19 at U.S. Long-Term Care Facilities
Mon August 10, 2020 7:17 AM|Business Wire|About: APLIF
U.S. expansion of antiviral outbreak control trial in long-term care facilities
Favipiravir already approved in India and Russia for emergency use against COVID-19 and as a pandemic influenza antiviral medication in Japan
First oral antiviral that may provide protection for elderly and vulnerable populations without need for intravenous administration or injections
Over 40% of deaths in the United States involve nursing home residents and staff i
HALIFAX, Nova Scotia--(BUSINESS WIRE)-- Appili Therapeutics Inc. (APLIF)
Appili is expanding its Phase 2 clinical trial into the U.S. to evaluate the safety and efficacy of favipiravir tablets in controlling outbreaks following exposure to COVID-19 in long-term care (LTC) facilities.
https://www.appilitherapeutics.com/favipiravir
https://www.appilitherapeutics.com/
https://pubchem.ncbi.nlm.nih.gov/compound/Favipiravir
For more information, visit www.AppiliTherapeutics.com.
Appili receives regulatory clearance to expand mid-stage study of Favipiravir for COVID-19
Aug. 10, 2020 9:25 AM ET|About: Appili Therapeutics Inc. (APLIF)|By: Vandana Singh, SA News Editor
https://seekingalpha.com/symbol/APLIF
https://www.businesswire.com/news/home/20200810005098/en/
Evaluating the Combination of Lucitanib and Opdivo in Gynecologic Cancers
Evaluating the Combination of Lucitanib and Opdivo in Gynecologic Cancers
Evaluating the Combination of Lucitanib and Opdivo in Gynecologic Cancers
Clovis Oncology Announces First Patient Enrolled in the Phase 2 Portion of the LIO-1 Trial Evaluating the Combination of Lucitanib and Opdivo in Gynecologic Cancers
Wed August 5, 2020 8:05 AM|Business Wire|About: CLVS
- Initial Phase 1b data from LIO-1 to be presented at the ESMO Virtual Congress 2020
- The LIO-1 trial is part of Clovis Oncology’s broad clinical collaboration with Bristol Myers Squibb
BOULDER, Colo.--(BUSINESS WIRE)-- Clovis Oncology, Inc. (CLVS)
More information about the LIO-1 trial (NCT04042116) is available here.
Phase 2 portion of a Phase 1/2 clinical trial, LIO-1, evaluating Clovis Oncology's (NASDAQ:CLVS) lucitanib, combined with Bristol Myers Squibb's (NYSE:BMY) Opdivo (nivolumab), in patients with gynecologic cancers.
please visit www.clovisoncology.com for more information
https://www.businesswire.com/news/home/20200805005359/en/
Clovis launches mid-stage study of lucitanib in gynecologic cancers
Aug. 5, 2020 8:59 AM ET|About: Clovis Oncology, Inc. (CLVS)|By: Douglas W. House, SA News Editor
https://seekingalpha.com/symbol/CLVS
https://seekingalpha.com/symbol/BMY
Lucitanib Clinical Development
AXS-12 (reboxetine)
Evaluating the Combination of Lucitanib and Opdivo in Gynecologic Cancers
Evaluating the Combination of Lucitanib and Opdivo in Gynecologic Cancers
Axsome Therapeutics Receives FDA Breakthrough Therapy Designation for AXS-12 for the Treatment of Narcolepsy
Wed August 5, 2020 7:00 AM|GlobeNewswire|About: AXSM
Designation offers potential for expedited development and review
FDA Orphan Drug Designation previously granted to Axsome for AXS-12 in narcolepsy
Third FDA Breakthrough Therapy designation received by Axsome
NEW YORK, Aug. 05, 2020 (GLOBE NEWSWIRE) -- Axsome Therapeutics, Inc. (AXSM)
please visit the Company’s website at axsome.com.
https://axsome.com/axs-pipeline/about-axs-12/
https://seekingalpha.com/symbol/AXSM
Axsome nabs accelerated review in U.S. for narcolepsy candidate AXS-12
Aug. 5, 2020 7:54 AM ET|About: Axsome Therapeutics, Inc. (AXSM)|By: Vandana Singh, SA News Editor
NVX-CoV2373
Evaluating the Combination of Lucitanib and Opdivo in Gynecologic Cancers
RGX-314 is being developed as a potential one-time treatment for wet AMD
Novavax rebounds from vaccine data-stoked selloff on clarified safety profile
Aug. 4, 2020 4:34 PM ET|About: Novavax, Inc. (NVAX)|By: Douglas W. House, SA News Editor
Novavax Announces Positive Phase 1 Data for its COVID-19 Vaccine Candidate
Tue August 4, 2020 4:05 PM|GlobeNewswire|About: NVAX
- Phase 1 portion of the Phase 1/2 clinical trial evaluated two doses of Novavax’ COVID-19 vaccine across two dose levels (5 and 25 µg) in 131 healthy adults ages 18-59 years
- NVX-CoV2373 was generally well-tolerated and had a reassuring safety profile
- The vaccine induced neutralization titers in 100% of participants
- Both 5 µg and 25 µg adjuvanted doses generated peak geometric mean titer (GMT) greater than 1:3,300
- Matrix-M™ adjuvant induced robust polyfunctional CD4+ T cell responses
- Conference call to be held on Tuesday, August 4 at 5:00 p.m. ET. Detailed data slides will be posted at 4:30 p.m. ET on novavax.com.
GAITHERSBURG, Md., Aug. 04, 2020 (GLOBE NEWSWIRE) -- Novavax, Inc. (NVAX)
https://novavax.com/download/files/2020.08.04NVXCoV2373Phase1ClinicalResults.pdf
https://novavax.com/our-pipeline#nvx-cov2373
Novavax website (novavax.com)
visit www.novavax.com
Novavax’s Covid-19 vaccine shows promising immune response, early data show
AUGUST 4, 2020
Update: Shares have rebounded in after-hours trading, now down only 2%, after STAT News issued a correction to its story after communicating with the company. No participants required hospitalization.
https://seekingalpha.com/symbol/NVAX
Novavax Announces Positive Phase 1 Data for its COVID-19 Vaccine Candidate
Aug 04, 2020 at 4:05 PM EDT
Prognosis
Novavax’s Covid-19 Vaccine Results Send Investors on Wild Ride
By Cristin FlanaganAugust 4, 2020, 4:08 PM EDT Updated on August 4, 2020, 5:10 PM EDT
RGX-314 is being developed as a potential one-time treatment for wet AMD
RGX-314 is being developed as a potential one-time treatment for wet AMD
RGX-314 is being developed as a potential one-time treatment for wet AMD
REGENXBIO Announces Additional Positive Interim Phase I/IIa Trial Update and Program Updates for RGX-314 for the Treatment of Wet AMD
Tue August 4, 2020 7:00 AM|PR Newswire|About: RGNX
ROCKVILLE, Md., Aug. 4, 2020 /PRNewswire/ --
- Company on track to initiate RGX-314 subretinal delivery pivotal program by the end of 2020
- RGX-314 was generally well-tolerated in 42 patients at all dose levels in Phase I/IIa trial
- Positive interim update from Cohorts 4 and 5 at one year informs pivotal program
- Durable treatment effect observed with stable to improved visual acuity and retinal thickness
- Demonstrated meaningful reductions in anti-VEGF treatment burden over one year
- 61% and 85% reduction of anti-VEGF injections in Cohorts 4 and 5, respectively
- 73% of patients (8/11) in Cohort 5 remain anti-VEGF injection-free
- Intraocular RGX-314 protein expression levels are dose-dependent at one year
- Phase II trial for RGX-314 for the treatment of wet AMD using suprachoroidal delivery (AAVIATE) is active
- Enrollment expected to begin in Q3 2020; interim data update from first cohort expected by end of 2020
- Company to host conference call and webcast on Tuesday, August 4 at 8:30a.m. ET, featuring wet AMD Key Opinion Leaders, Robert Avery, M.D., Dante Pieramici, M.D., and Peter Kaiser, M.D.
REGENXBIO Inc. (RGNX)
RGX-314 Gene Therapy for Neovascular AMD Trial
https://clinicaltrials.gov/ct2/show/NCT03066258?lead=regenxbio&draw=2&rank=9
https://www.regenxbio.com/rgx-314/
https://seekingalpha.com/symbol/RGNX
Regenxbio announces positive one-year data on wet AMD gene therapy
Aug. 4, 2020 7:52 AM ET|About: REGENXBIO Inc. (RGNX)|By: Douglas W. House, SA News Editor
REGENXBIO (NASDAQ:RGNX) announces positive one-year data from cohorts 4 & 5 of its Phase 1/2a clinical trial evaluating gene therapy RGX-314 in patients with wet age-related macular degeneration (wet AMD)
ELX-02 for the Treatment of Cystic Fibrosis
RGX-314 is being developed as a potential one-time treatment for wet AMD
ALRN-6924, an investigational first-in-class MDM2/MDMX dual inhibitor
Eloxx Pharmaceuticals Receives U.S. Orphan Drug Designation for ELX-02 for the Treatment of Cystic Fibrosis
Tue August 4, 2020 7:30 AM|GlobeNewswire|About: ELOXGlobeNewswireWALTHAM, Mass., Aug. 04, 2020 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (ELOX),
please visit www.eloxxpharma.com.
https://seekingalpha.com/symbol/ELOX
https://www.eloxxpharma.com/pipeline/
Eloxx Pharmaceuticals Receives U.S. Orphan Drug Designation for ELX-02 for the Treatment of Cystic Fibrosis
August 4, 2020PDF Version ELX-02 had previously been granted orphan medicinal product designation for the treatment of Cystic Fibrosis by the European Medicines AgencyWALTHAM, Mass., Aug. 04, 2020 (GLOBE NEWSWIRE) -- Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX), https://investors.eloxxpharma.com/news-
releases/news-release-details/eloxx-pharmaceuticals-receives-us-orphan-drug-designation-elx-02
ALRN-6924, an investigational first-in-class MDM2/MDMX dual inhibitor
RGX-314 is being developed as a potential one-time treatment for wet AMD
ALRN-6924, an investigational first-in-class MDM2/MDMX dual inhibitor
Aileron Therapeutics Announces Completion of Enrollment in Dose Optimization Expansion Cohort of Proof-of-Concept Phase 1b Study of ALRN-6924
Mon August 3, 2020 8:00 AM|GlobeNewswire|About: ALRN
- Study is evaluating ALRN-6924 to protect against chemotherapy-induced bone marrow toxicities in patients with p53-mutated small cell lung cancer (SCLC) treated with topotecan
- Previously reported positive interim data from dose optimization part of study demonstrated 0.3 mg/kg dose of ALRN-6924, now being evaluated in expansion cohort, resulted in most robust chemoprotective effects
- Data readouts anticipated in Q4 2020: Final dose optimization data, including data from expansion cohort as well as pharmacodynamic biomarker and tumor efficacy data; preliminary data from recently initiated schedule optimization part of study
WATERTOWN, Mass., Aug. 03, 2020 (GLOBE NEWSWIRE) -- Aileron Therapeutics (ALRN)
Visit us at aileronrx.com to learn more.
https://seekingalpha.com/symbol/ALRN
https://www.aileronrx.com/science/pipeline/
Aileron Therapeutics Announces Completion of Enrollment in Dose Optimization Expansion Cohort of Proof-of-Concept Phase 1b Study of ALRN-6924
August 03, 2020 at 8:00 AM EDTPDF Version
- Study is evaluating ALRN-6924 to protect against chemotherapy-induced bone marrow toxicities in patients with p53-mutated small cell lung cancer (SCLC) treated with topotecan
- Previously reported positive interim data from dose optimization part of study demonstrated 0.3 mg/kg dose of ALRN-6924, now being evaluated in expansion cohort, resulted in most robust chemoprotective effects
- Data readouts anticipated in Q4 2020: Final dose optimization data, including data from expansion cohort as well as pharmacodynamic biomarker and tumor efficacy data; preliminary data from recently initiated schedule optimization part of study
WATERTOWN, Mass., Aug. 03, 2020 (GLOBE NEWSWIRE) -- Aileron Therapeutics (NASDAQ:ALRN
biotecHOPE™ July/August 2020
TDP-43 (TAR DNA-binding protein 43)
SM-88 (racemetyrosine) for pancreatic cancer.
TDP-43 (TAR DNA-binding protein 43)
AC Immune launches preclinical trials for anti-TDP-43 antibody in neurodegenerative diseases
Aug. 3, 2020 8:55 AM ET|About: AC Immune SA (ACIU)|By: Vandana Singh, SA News Editor
https://www.acimmune.com/en/pipeline-overview/
AC Immune Initiates IND-Enabling Studies for First-in-Class Antibody Targeting TDP-43 to Treat Neurodegeneration
August 03, 2020
https://seekingalpha.com/symbol/ACIU
Mavacamten (MYK-461)
SM-88 (racemetyrosine) for pancreatic cancer.
TDP-43 (TAR DNA-binding protein 43)
MyoKardia Doses First Patient in Phase 3 VALOR-HCM Trial of Mavacamten
Mon August 3, 2020 8:30 AM|GlobeNewswire|About: MYOKGlobeNewswire
BRISBANE, Calif., Aug. 03, 2020 (GLOBE NEWSWIRE) -- MyoKardia, Inc. (MYOK) announced that the first patient has been dosed in the Phase 3 VALOR-HCM clinical trial.
CORRECTION: MyoKardia Doses First Patient in Phase 3 VALOR-HCM Trial of Mavacamten
August 3, 2020 at 9:52 AM EDTPDF VersionVALOR-HCM Designed to Generate Direct Evidence of Improved Outcomes for People with HCM by Reducing the Need for Invasive Septal Reduction Therapy
BRISBANE, Calif., Aug. 03, 2020 (GLOBE NEWSWIRE) -- In a release issued under the same headline on Monday, August 3rd by MyoKardia, Inc. (Nasdaq: MYOK), please note that the second quote by Jeffrey B. Geske, M.D. has changed. The corrected release follows:
MyoKardia commences late-stage study of mavacamten in treatment-resistant heart disorder
Aug. 3, 2020 9:36 AM ET|About: MyoKardia, Inc. (MYOK)|By: Douglas W. House, SA News Editor
A Study to Evaluate Mavacamten in Adults With Symptomatic Obstructive HCM Who Are Eligible for Septal Reduction Therapy (VALOR-HCM)
SM-88 (racemetyrosine) for pancreatic cancer.
SM-88 (racemetyrosine) for pancreatic cancer.
SM-88 (racemetyrosine) for pancreatic cancer.
TYME Announces Orphan Drug Designation for SM-88 as Potential Treatment for Patients with Pancreatic Cancer
Mon August 3, 2020 8:00 AM|Business Wire|About: TYME
- SM-88 has demonstrated encouraging tumor responses in 15 different cancers across four separate studies with minimal serious grade 3 or higher adverse events
- TYME-88-Panc pivotal trial enrolling patients using oral SM-88 as a potential treatment for third-line pancreatic cancer.
- PanCAN enrolling patients in its Precision PromiseSM adaptive randomized Phase II/III registration-intent trial evaluating oral SM-88 as second-line monotherapy for pancreatic cancer
- TYME & Joseph Ahmed Foundation’s HopES Sarcoma study enrolling patients for the investigator-initiated Phase II trial studying oral SM-88 as maintenance monotherapy in previously treated metastatic Ewing’s sarcoma and salvage monotherapy in clinically advanced sarcomas
- SM-88 mechanism of action supported by new preclinical data presented at AACR 2020
BEDMINSTER, N.J.--(BUSINESS WIRE)-- Tyme Technologies, Inc. (TYME)
visit www.tymeinc.com
https://www.businesswire.com/news/home/20200803005153/en/
https://seekingalpha.com/symbol/TYME
Tyme's racemetyrosine an Orphan Drug in U.S. for pancreatic cancer
Aug. 3, 2020 8:37 AM ET|About: Tyme Technologies, Inc. (TYME)|By: Vandana Singh, SA News Editor
https://seekingalpha.com/news/3598896-tymes-racemetyrosine-orphan-drug-in-u-s-for-pancreatic-cancer
https://www.tymeinc.com/our-science/sm-88-overview/default.aspx
TYME Pancreatic Cancer Clinical Trial TYME is an emerging biotechnology company developing nextgeneration Cancer Metabolism-Based Therapies (CMBTs™) for patients with advanced cancers
https://s22.q4cdn.com/265040820/files/doc_downloads/2020/TYME_88_Panc-Trial_vJune.pdf
Seladelpar
LY-CoV555 is a potent, neutralizing IgG1 monoclonal antibody (mAb) vs the spike protein SARS-CoV-2
SM-88 (racemetyrosine) for pancreatic cancer.
CymaBay Announces Positive Topline Results from ENHANCE for Seladelpar in Patients with Primary Biliary Cholangitis
Mon August 3, 2020 7:00 AM|GlobeNewswire|About: CBAY
- The study demonstrated seladelpar to be efficacious, safe and well-tolerated
- 78.2% of patients on seladelpar 10 mg versus 12.5% on placebo achieved the primary composite outcome after only 3 months (p<0.0001)
- 27.3% of patients on seladelpar 10 mg versus zero on placebo normalized ALP by 3 months (p<0.0001)
- Statistically significant improvement in pruritus (p<0.05) for patients with moderate-to-severe itch demonstrated for seladelpar 10 mg versus placebo
- Strongly supports re-initiation of Phase 3 study to confirm the potential of seladelpar to be a best-in-class treatment for PBC
- Conference call today at 8:00 a.m. ET
NEWARK, Calif., Aug. 03, 2020 (GLOBE NEWSWIRE) -- CymaBay Therapeutics, Inc. (CYMA) (NASDAQ: CBAY)
visit www.cymabay.com.
https://seekingalpha.com/symbol/CBAY
http://www.cymabay.com/pipeline.html
CymaBay's seladelpar successful in late-stage study in bile duct disorder
Aug. 3, 2020 7:39 AM ET|About: CymaBay Therapeutics, ... (CBAY)|By: Douglas W. House, SA News Editor
LY-CoV555 is a potent, neutralizing IgG1 monoclonal antibody (mAb) vs the spike protein SARS-CoV-2
LY-CoV555 is a potent, neutralizing IgG1 monoclonal antibody (mAb) vs the spike protein SARS-CoV-2
LY-CoV555 is a potent, neutralizing IgG1 monoclonal antibody (mAb) vs the spike protein SARS-CoV-2
Lilly Initiates Phase 3 Trial of LY-CoV555 for Prevention of COVID-19 at Long-Term Care Facilities in Partnership with the National Institute of Allergy and Infectious Diseases (NIAID)
Mon August 3, 2020 6:45 AM|PR Newswire|About: LLY
- First-of-its-kind study will enroll up to 2,400 residents and staff at long-term care facilities; will utilize customized mobile research units to conduct the study on-site
PR Newswire
INDIANAPOLIS, Aug. 3, 2020 /PRNewswire/ -- Eli Lilly and Company (LLY)
BLAZE-2, a Phase 3 trial studying LY-CoV555 for the prevention of SARS-CoV-2 infection and COVID-19 in residents and staff at long-term care facilities in the U.S.
visit Lilly TrialGuide for information regarding eligibility for ongoing trials.
(NCT04427501),
please visit us at www.lilly.com and www.lilly.com/news.
https://seekingalpha.com/symbol/LLY
IMU-838 is an orally available, next-generation selective immune modulator
LY-CoV555 is a potent, neutralizing IgG1 monoclonal antibody (mAb) vs the spike protein SARS-CoV-2
LY-CoV555 is a potent, neutralizing IgG1 monoclonal antibody (mAb) vs the spike protein SARS-CoV-2
Immunic, Inc. Reports Positive Top-line Data from Phase 2 EMPhASIS Trial of IMU-838 in Patients with Relapsing-Remitting Multiple Sclerosis
Sun August 2, 2020 3:00 PM|PR Newswire|About: IMUX
- Statistically Significant Reduction of 62% and 70% in Combined Unique Active Magnetic Resonance Imaging Lesions in 45mg and 30mg Patient Cohorts, As Compared to Placebo -
- Data Supports Previously Observed Favorable Safety Profile of IMU-838 in Relapsing-Remitting Multiple Sclerosis Patient Population -
- Company Also Reports Second Quarter 2020 Financial Results With $48.6 Million in Cash and Cash Equivalents -
- Conference Call and Webcast to be Held on August 3, 2020 at 8:30am ET -
PR Newswire
NEW YORK, Aug. 2, 2020 /PRNewswire/ -- Immunic, Inc. (IMUX)
Immunic's website at: ir.imux.com.
https://www.immunic-therapeutics.com/
please visit: www.imux.com.
https://www.immunic-therapeutics.com/imu838/
Immunic's IMU-838 successful in mid-stage multiple sclerosis study
Aug. 3, 2020 2:26 AM ET|About: Immunic, Inc. (IMUX)|By: Mamta Mayani, SA News Editor
phase 2 EMPhASIS trial of lead asset, IMU-838, the selective oral DHODH inhibitor, in patients with relapsing-remitting multiple sclerosis (RRMS).
https://seekingalpha.com/symbol/IMUX
RLF-100 (aviptadil)
oral antiviral therapy/clinical-trials-2/ and two vaccines for COVID-19 ---- EIDD-2801
oral antiviral therapy/clinical-trials-2/ and two vaccines for COVID-19 ---- EIDD-2801
RLF-100 (aviptadil) clinical trial showed rapid recovery from respiratory failure and inhibition of coronavirus replication in human lung cells
Sun August 2, 2020 4:00 PM|PR Newswire|About: RLFTF
- Aviptadil is being developed as the first COVID therapeutic to block replication of the SARS-CoV-2 virus in human lung cells and monocytes
- RLF-100 is a patented formulation of aviptadil (synthetic human Vasoactive Intestinal Polypeptide VIP), which has been granted FDA Fast Track Designation, FDA emergency use IND authorization, and an expanded access protocol
PR Newswire
RADNOR, Pa. and GENEVA, Aug. 2, 2020 /PRNewswire/ -- NeuroRx, Inc. and Relief Therapeutics Holdings AG (SIX:RLF, OTC:RLFTF)
clinicaltrials.gov NCT04311697.
https://seekingalpha.com/symbol/RLFTF
https://relieftherapeutics.com/
https://www.neurorxpharma.com/
Relief Therapeutics' Aviptadil shows positive action in COVID-19 patients
Aug. 3, 2020 12:30 AM ET|About: Relief Therapeutics Holdin... (RLFTF)|By: Mamta Mayani, SA News Editor
oral antiviral therapy/clinical-trials-2/ and two vaccines for COVID-19 ---- EIDD-2801
oral antiviral therapy/clinical-trials-2/ and two vaccines for COVID-19 ---- EIDD-2801
oral antiviral therapy/clinical-trials-2/ and two vaccines for COVID-19 ---- EIDD-2801
Merck & Co. joins race for COVID-19 vaccines and therapies
The firm will develop viral vector vaccines from IAVI and Themis Bioscience, and the experimental EIDD-2801 antiviral compound developed by Ridgeback Biotherapeutics
by Ryan Cross
MAY 26, 2020 | APPEARED IN VOLUME 98, ISSUE 21
An emerging antiviral takes aim at COVID-19
EIDD-2801 wasn’t designed to fight the novel coronavirus, but its chemistry might make it an ideal weapon in this pandemic and the next
by Bethany Halford
MAY 5, 2020
COVID-19 First In Human Study to Evaluate Safety, Tolerability, and Pharmacokinetics of EIDD-2801 in Healthy Volunteers
https://clinicaltrials.gov/ct2/show/NCT04392219?cond=EIDD-2801&draw=2&rank=1
https://www.merck.com/index.html
IAVI and Merck Collaborate to Develop Vaccine Against SARS-CoV-2
Tuesday, May 26, 2020 6:49 am ET
Ridgeback Aims Antiviral at COVID-19 in Two Phase II Studies
Published: Jun 22, 2020 By Alex Keown
Ridgeback Biotherapeutics Announces Launch of Phase 2 Trials Testing EIDD-2801 as Potential Treatment for COVID-19
Two Randomized Double-Blind Placebo Controlled Studies In COVID-19 Patients Follow Phase 1 Trials Showing Safety & Promising Exposures in Humans
June 19, 2020 10:00 AM Eastern Daylight Time
MIAMI--(BUSINESS WIRE)--Ridgeback Biotherapeutic
CUTX-101 (Copper Histidinate)
oral antiviral therapy/clinical-trials-2/ and two vaccines for COVID-19 ---- EIDD-2801
Bucillamine in patient with mild-to-moderate COVID-19.
Fortress Biotech Announces Positive Opinion on Orphan Drug Designation Received from the European Medicines Agency for CUTX-101, Copper Histidinate, for the Treatment of Menkes Disease
Fri July 31, 2020 7:30 AM|GlobeNewswire|About: FBIOGlobeNewswire
NEW YORK, July 31, 2020 (GLOBE NEWSWIRE) -- Fortress Biotech, Inc. (FBIO)
For more information, visit www.cypriumtx.com.
https://www.fortressbiotech.com/
https://www.cypriumtx.com/wp-content/uploads/2020/07/2020-07-31-Fortress-Cyprium-EMA-ODD-FINAL.pdf
For more information, visit www.fortressbiotech.com.
A Diversified Pipeline Spanning Several Large Markets
https://www.fortressbiotech.com/programs
https://seekingalpha.com/symbol/FBIO
Bucillamine in patient with mild-to-moderate COVID-19.
Stereotaxis & Acutus Medical = First Integrated Cardiac Ablation Procedure w/ TeleRobotic Support
Bucillamine in patient with mild-to-moderate COVID-19.
Revive Therapeutics Announces U.S. FDA Approval of Confirmatory Phase 3 Clinical Trial for Bucillamine in COVID-19
Fri July 31, 2020 8:39 AM|GlobeNewswire|About: RVVTF
TORONTO, July 31, 2020 (GLOBE NEWSWIRE) -- Revive Therapeutics Ltd. (RVVTF)
https://www.revivethera.com/pipeline.html
For more information, visit www.ReviveThera.com.
https://seekingalpha.com/symbol/RVVTF
Stereotaxis & Acutus Medical = First Integrated Cardiac Ablation Procedure w/ TeleRobotic Support
Stereotaxis & Acutus Medical = First Integrated Cardiac Ablation Procedure w/ TeleRobotic Support
Stereotaxis & Acutus Medical = First Integrated Cardiac Ablation Procedure w/ TeleRobotic Support
Stereotaxis and Acutus Medical Announce First Integrated Cardiac Ablation Procedure with TeleRobotic Support
Fri July 31, 2020 8:05 AM|GlobeNewswire|About: STXS
ST. LOUIS and CARLSBAD, Calif., July 31, 2020 (GLOBE NEWSWIRE) -- Stereotaxis (STXS) (NYSE: STXS) and Acutus Medical (AFIB)
Stereotaxis (STXS) (NYSE: STXS) and Acutus Medical (AFIB)
For more information, please visit www.stereotaxis.com.
https://www.acutusmedical.com/us/platform/#products
http://www.stereotaxis.com/products/
https://seekingalpha.com/symbol/STXS
https://seekingalpha.com/symbol/AFIB
integrated cardiac ablation procedure
Ganaxolone, a positive allosteric modulator of GABAA receptors
Stereotaxis & Acutus Medical = First Integrated Cardiac Ablation Procedure w/ TeleRobotic Support
Stereotaxis & Acutus Medical = First Integrated Cardiac Ablation Procedure w/ TeleRobotic Support
Marinus Pharmaceuticals Receives Rare Pediatric Disease Designation from FDA for Ganaxolone for the Treatment of CDKL5 Deficiency Disorder (CDD)
Thu July 30, 2020 7:30 AM|Business Wire|About: MRNS
RADNOR, Pa.--(BUSINESS WIRE)-- Marinus Pharmaceuticals, Inc. (MRNS) (Nasdaq: MRNS)
For more information visit www.marinuspharma.com.
https://www.businesswire.com/news/home/20200730005259/en/
https://seekingalpha.com/symbol/MRNS
biotecHOPE™ July 2020
FixVac cancer vaccine program BNT111
atogepant, an orally available calcitonin gene-related peptide (CGRP) receptor antagonist
encouraging preclinical results on COVID-19 vaccine
BioNTech Publishes Data from mRNA-based BNT111 FixVac Melanoma Trial in Nature
Thu July 30, 2020 3:00 AM|GlobeNewswire|About: BNTX
- Preliminary Phase 1 results from Lipo-MERIT trial with data from 89 patients highlight favorable tolerability profile of BNT111 in advanced melanoma patients
- Efficacy analysis in a subset of 42 checkpoint-inhibitor (CPI)-experienced metastatic melanoma patients shows that BNT111 mediates durable responses both as a single agent and in combination with anti-PD-1 antibodies
- Durable objective responses by BNT111 are associated with activation and strong expansion of tumor-antigen-specific CD4+ and CD8+ T cells
- Phase 2 trial with registrational potential planned to commence in 2020
MAINZ, Germany, July 30, 2020 (GLOBE NEWSWIRE) -- BioNTech SE (BNTX) (NASDAQ: BNTX,
Phase 1 trial (NCT02410733) to evaluate safety and tolerability of vaccinated patients with stage IIIB-C and stage IV melanoma. The publication titled “An RNA vaccine drives immunity in checkpoint-inhibitor-treated melanoma”
Further FixVac cancer vaccine candidates are currently investigated in Phase 1 clinical trials for prostate cancer (BNT112) (Clinicaltrials.gov Identifier NCT04382898), HPV16-positive cancers (BNT113) (Clinicaltrials.gov Identifier NCT03418480), triple negative breast cancer (BNT114) (Clinicaltrials.gov Identifier NCT02316457) and ovarian cancer (BNT115) (Clinicaltrials.gov Identifier NCT04163094).
For more information, please visit www.BioNTech.de.
https://biontech.de/science/pipeline
clinical trial, Lipo-MERIT, evaluating FixVac mRNA cancer vaccine candidate BNT111 in patients with advanced melanoma. The results were just published in the journal Nature.
https://seekingalpha.com/symbol/BNTX
encouraging preclinical results on COVID-19 vaccine
atogepant, an orally available calcitonin gene-related peptide (CGRP) receptor antagonist
encouraging preclinical results on COVID-19 vaccine
INOVIO's COVID-19 DNA Vaccine INO-4800 Provides Protection with Memory Immune Responses In Non-Human Primates Challenged with SARS-CoV-2 Virus
Thu July 30, 2020 8:00 AM|PR Newswire|About: INO
- INO-4800 is the only vaccine to demonstrate long-term protection in non-human primates challenged with SARS-CoV-2 virus 13 weeks from vaccination
- Memory T and B cell responses resulted in reduced viral loads and faster viral clearance in macaques' lungs and nasal passages
- INO-4800 vaccination generated antibodies neutralizing both the earlier strain of virus as well as the mutant variant (D614G) that has emerged with greater infectivity, and now accounts for >80% of newly circulating virus
- No antibody-dependent enhanced disease events were reported
PR Newswire
PLYMOUTH MEETING, Pa., July 30, 2020 /PRNewswire/ -- INOVIO (NASDAQ:INO)
https://www.inovio.com/our-focus-serving-patients/covid-19/
https://seekingalpha.com/symbol/INO
atogepant, an orally available calcitonin gene-related peptide (CGRP) receptor antagonist
atogepant, an orally available calcitonin gene-related peptide (CGRP) receptor antagonist
atogepant, an orally available calcitonin gene-related peptide (CGRP) receptor antagonist
AbbVie's atogepant successful in late-stage migraine study
Jul. 29, 2020 9:37 AM ET|About: AbbVie Inc. (ABBV)|By: Douglas W. House, SA News Editor
Phase 3 clinical trial, ADVANCE, evaluating atogepant, an orally available calcitonin gene-related peptide (CGRP) receptor antagonist, for the prevention of migraine.
https://seekingalpha.com/symbol/ABBV
July 29, 2020
AbbVie Announces Positive Phase 3 Data for Atogepant in Migraine Prevention
RGLS4326 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Arrowhead Pharmaceuticals Earns $20 Million Milestone Payment from Amgen for Phase 2 of AMG 890
atogepant, an orally available calcitonin gene-related peptide (CGRP) receptor antagonist
Regulus Therapeutics Announces FDA Orphan Drug Designation of RGLS4326 for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Wed July 29, 2020 8:07 AM|PR Newswire|About: RGLS
LA JOLLA, Calif., July 29, 2020 /PRNewswire/ -- Regulus Therapeutics Inc (RGLS). (Nasdaq: RGLS)
http://regulusrx.com/programs/pipeline/
https://seekingalpha.com/symbol/RGLS
Arrowhead Pharmaceuticals Earns $20 Million Milestone Payment from Amgen for Phase 2 of AMG 890
Arrowhead Pharmaceuticals Earns $20 Million Milestone Payment from Amgen for Phase 2 of AMG 890
Arrowhead Pharmaceuticals Earns $20 Million Milestone Payment from Amgen for Phase 2 of AMG 890
Arrowhead Pharmaceuticals Earns $20 Million Milestone Payment from Amgen for Start of Phase 2 Trial of AMG 890
Wed July 29, 2020 7:30 AM|Business Wire|About: ARWR
PASADENA, Calif.--(BUSINESS WIRE)-- Arrowhead Pharmaceuticals Inc. (ARWR)
The clinical study (NCT04270760)
please visit www.arrowheadpharma.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20200729005483/en/
https://seekingalpha.com/symbol/ARWR
https://seekingalpha.com/symbol/AMGN
TARA-002 is an investigational cell-based therapy based on the broad immunopotentiator OK-432
Arrowhead Pharmaceuticals Earns $20 Million Milestone Payment from Amgen for Phase 2 of AMG 890
Arrowhead Pharmaceuticals Earns $20 Million Milestone Payment from Amgen for Phase 2 of AMG 890
Protara Therapeutics Receives Rare Pediatric Disease Designation for TARA-002 for the Treatment of Lymphatic Malformations
Tue July 28, 2020 7:00 AM|GlobeNewswire|About: TARA
NEW YORK, July 28, 2020 (GLOBE NEWSWIRE) -- Protara Therapeutics, Inc. (TARA)
For more information, visit www.protaratx.com
https://protaratx.com/pipeline/
https://seekingalpha.com/symbol/TARA
Protara cell therapy nabs Rare Pediatric Disease tag for lymphatic disorder
Jul. 28, 2020 7:56 AM ET|About: Protara Therapeutics, ... (TARA)|By: Douglas W. House, SA News Editor
TC-210 Phase 1/2 trial for mesothelin-expressing solid tumors.
TC-210 Phase 1/2 trial for mesothelin-expressing solid tumors.
TC-210 Phase 1/2 trial for mesothelin-expressing solid tumors.
TCR² Therapeutics Announces RECIST Responses with First TC-210 Dose Tested in Advanced Mesothelin-Expressing Solid Tumors
Sun July 26, 2020 5:00 PM|GlobeNewswire|About: TCRR
- TC-210 TRuC-T cell monotherapy induced tumor regression in first five patients
- Two RECIST unconfirmed partial responses and two patients with stable disease through six months
- Manageable toxicity profile, with only one patient exhibiting TC-210-related non-hematologic Grade >2 toxicity
- Translational data demonstrated T cell expansion and cytokine production
- TCR2 to host conference call today starting at 8:00am E.T. with live webcast available online
CAMBRIDGE, Mass., July 26, 2020 (GLOBE NEWSWIRE) -- TCR2 Therapeutics Inc. (TXMD) (Nasdaq: TCRR)
https://seekingalpha.com/symbol/TCRR
TCR2 Therapeutics' TC-210 successful in mid-stage cancer study
Jul. 27, 2020 3:52 AM ET|About: TCR2 Therapeutics Inc. (TCRR)|By: Mamta Mayani, SA News Editor
FUROSCIX®
TC-210 Phase 1/2 trial for mesothelin-expressing solid tumors.
TC-210 Phase 1/2 trial for mesothelin-expressing solid tumors.
scPharmaceuticals Announces FDA Acceptance of FUROSCIX® New Drug Application Resubmission
Mon July 27, 2020 8:00 AM|Business Wire|About: SCPH
FDA sets PDUFA date of December 30, 2020
BURLINGTON, Mass.--(BUSINESS WIRE)-- scPharmaceuticals Inc. (SCPH)
please visit www.scPharmaceuticals.com.
https://www.businesswire.com/news/home/20200727005117/en/
https://seekingalpha.com/symbol/SCPH
CiVax, its heat-stable subunit COVID-19 vaccine candidate.
TC-210 Phase 1/2 trial for mesothelin-expressing solid tumors.
Savolitinib is an oral, potent, and highly selective small molecule inhibitor of MET
Soligenix Announces Publication of Positive Pre-clinical Results for a Novel COVID-19 Vaccine
Tue July 28, 2020 7:00 AM|PR Newswire|About: SNGX
- Key Attributes including Generation of Neutralizing Antibodies, Th1 Antibody and Cell Mediated Responses Established
PR Newswire
PRINCETON, N.J., July 28, 2020 /PRNewswire/ -- Soligenix, Inc. (SNGX)
https://www.soligenix.com/pipeline-programs/
Soligenix Announces Publication of Positive Pre-clinical Results for a Novel COVID-19 Vaccine- Rapid Immune Responses Demonstrated with CoVaccine HT™ Adjuvant in a Prototype Vaccine- Key Attributes including Generation of Neutralizing Antibodies, Th1 Antibody and Cell Mediated Responses Established
PRINCETON, N.J., July 28, 2020 /PRNewswire/ -- Soligenix, Inc. (Nasdaq: SNGX)
https://seekingalpha.com/symbol/SNGX
visit the Company's website at www.soligenix.com.
Savolitinib is an oral, potent, and highly selective small molecule inhibitor of MET
Savolitinib is an oral, potent, and highly selective small molecule inhibitor of MET
Savolitinib is an oral, potent, and highly selective small molecule inhibitor of MET
Chi-Med’s NDA for Savolitinib in Non-Small Cell Lung Cancer Granted Priority Review in China
Tue July 28, 2020 2:00 AM|GlobeNewswire|About: AZN, HCM
HONG KONG and FLORHAM PARK, N.J., July 28, 2020 (GLOBE NEWSWIRE) -- Hutchison China MediTech Limited (HCM)
Phase II in MET Exon 14 mutation NSCLC (NCT02897479)
MET-driven papillary renal cell carcinoma (“RCC”) (NCT03091192)
For more information, please visit: www.chi-med.com.
https://seekingalpha.com/symbol/HCM
In 2011, Chi-Med entered into a global licensing and joint development and commercialization agreement with AstraZeneca (LSE, STO, NYSE: AZN) for savolitinib. Savolitinib’s global development plan includes NSCLC and kidney cancer, and additional MET-driven tumors are being explored.
SAVANNAH Phase II study of savolitinib in combination with Tagrisso® in patients who have progressed following Tagrisso® due to MET amplification or overexpression (NCT03778229)
Zenocutuzumab (Zeno)
Savolitinib is an oral, potent, and highly selective small molecule inhibitor of MET
Zenocutuzumab (Zeno)
Merus announces FDA Orphan Drug Designation of Zenocutuzumab for the Treatment of Pancreatic Cancer
Mon July 27, 2020 8:00 AM|GlobeNewswire|About: MRUS
UTRECHT, The Netherlands and CAMBRIDGE, Mass., July 27, 2020 (GLOBE NEWSWIRE) -- Merus N.V. (Nasdaq: MRUS)
Zeno is now being evaluated in a global phase 1/2 clinical trial called the eNRGy trial.
www.nrg1.com or by calling 1-833-NRG-1234
https://seekingalpha.com/symbol/MRUS
Merus announces FDA Orphan Drug Designation of Zenocutuzumab for the Treatment of Pancreatic Cancer
July 27, 2020 at 8:00 AM EDT
MYCAPSSA (octreotide)
Savolitinib is an oral, potent, and highly selective small molecule inhibitor of MET
Zenocutuzumab (Zeno)
Chiasma Announces 48-Week Safety and Efficacy Data from the Open-Label Extension Study of its CHIASMA OPTIMAL Phase 3 Trial Evaluating MYCAPSSA® in Patients with Acromegaly
Mon July 27, 2020 4:01 PM|GlobeNewswire|About: CHMA
-- The mean of the IGF-1 levels for the population of all MYCAPSSA treated patients that completed the 36-week core CHIASMA OPTIMAL trial and continued into the open-label extension (OLE) (n=19) was maintained within normal limits at the end of the 48-week OLE period --
-- All MYCAPSSA responders (IGF-1 within normal limits) who enrolled into the OLE completed the 48-week period; 93% maintained their response at the end of this period --
NEEDHAM, Mass., July 27, 2020 (GLOBE NEWSWIRE) -- Chiasma, Inc. (CHMA)
The full Prescribing Information for MYCAPSSA is available at www.MYCAPSSA.com.
website at www.chiasma.com.
https://seekingalpha.com/symbol/CHMA
Chiasma's octreotide shows sustained benefit in acromegaly extension study
Jul. 27, 2020 4:31 PM ET|About: Chiasma, Inc. (CHMA)|By: Douglas W. House, SA News Editor
biotecHOPE™ July 2020
ARIKAYCE® (amikacin liposome inhalation suspension)
ARIKAYCE® (amikacin liposome inhalation suspension)
ARIKAYCE® (amikacin liposome inhalation suspension)
Insmed Receives Positive CHMP Opinion for ARIKAYCE Liposomal 590 mg Nebuliser Dispersion for the Treatment of NTM Lung Infections Caused by MAC in Non-CF Patients with Limited Treatment Options
Fri July 24, 2020 7:36 AM|PR Newswire|About: INSM
- Decision from European Commission Expected Second Half of 2020 -
PR Newswire
BRIDGEWATER, N.J., July 24, 2020 /PRNewswire/ -- Insmed Incorporated (INSM)
Please see Full Prescribing Information.
For more information, visit www.insmed.com.
ADP-A2M4 for the Treatment of Synovial Sarcoma
ARIKAYCE® (amikacin liposome inhalation suspension)
ARIKAYCE® (amikacin liposome inhalation suspension)
Adaptimmune Granted Access to PRIority MEdicines (PRIME) Regulatory Support by the European Medicines Agency for ADP-A2M4 for the Treatment of Synovial Sarcoma
Thu July 23, 2020 4:00 PM|GlobeNewswire|About: ADAP
PHILADELPHIA and OXFORDSHIRE, United Kingdom, July 23, 2020 (GLOBE NEWSWIRE) -- Adaptimmune Therapeutics plc (ADAP)
https://www.adaptimmune.com/pipeline/adp-a2m4
Adaptimmune Granted Access to PRIority MEdicines (PRIME) Regulatory Support by the European Medicines Agency for ADP-A2M4 for the Treatment of Synovial Sarcoma
https://seekingalpha.com/symbol/ADAP
Synovial Sarcoma
What is synovial sarcoma?
You can help speed up the development of new treatments by giving researchers the tools they need.
Synovial sarcoma is a cancer that can come from different types of soft tissue, such as muscle or ligaments. It is often found in the arm, leg, or foot, and near joints such as the wrist or ankle. It can also form in soft tissues in the lung or abdomen. Synovial sarcoma may also be called malignant synovioma.
Mavacamten (MYK-461)
ARIKAYCE® (amikacin liposome inhalation suspension)
Phase 2 trial of relacorilant combined with nab-paclitaxel (Abraxane®)
MyoKardia Announces Receipt of Breakthrough Therapy Designation from FDA for Mavacamten for the Treatment of Symptomatic, Obstructive Hypertrophic Cardiomyopathy
Thu July 23, 2020 4:05 PM|GlobeNewswire|About: MYOK
BRISBANE, Calif., July 23, 2020 (GLOBE NEWSWIRE) -- MyoKardia, Inc. (MYOK)
https://myokardia.com/programs
https://seekingalpha.com/symbol/MYOK
MyoKardia Announces Receipt of Breakthrough Therapy Designation from FDA for Mavacamten for the Treatment of Symptomatic, Obstructive Hypertrophic Cardiomyopathy
Phase 2 trial of relacorilant combined with nab-paclitaxel (Abraxane®)
RGLS4326 is a novel oligonucleotide designed to inhibit miR-17 & designed to target the kidney
Phase 2 trial of relacorilant combined with nab-paclitaxel (Abraxane®)
Corcept Therapeutics Completes Enrollment in Controlled, Phase 2 Trial of Relacorilant Plus Nab-Paclitaxel in Patients with Metastatic Ovarian Cancer
Thu July 23, 2020 8:30 AM|GlobeNewswire|About: CORT
MENLO PARK, Calif., July 23, 2020 (GLOBE NEWSWIRE) -- Corcept Therapeutics Incorporated (CORT)
2 For additional information, see Clinicaltrials.gov (trial identifier: NCT03776812)
https://www.corcept.com/research-pipeline/pipeline/
https://seekingalpha.com/symbol/CORT
Corcept Therapeutics (CORT -0.2%) has completed enrollment of 177 metastatic ovarian cancer patients in a Phase 2 clinical trial assessing the combination of relacorilant and Bristol Myers Squibb's Abraxane (nab-paclitaxel) in patients who have failed to respond to platinum-based chemo
Microbiome Metabolic Therapies (MMT™)
RGLS4326 is a novel oligonucleotide designed to inhibit miR-17 & designed to target the kidney
RGLS4326 is a novel oligonucleotide designed to inhibit miR-17 & designed to target the kidney
Kaleido Biosciences Initiates Controlled Clinical Study with Massachusetts General Hospital of Microbiome Metabolic Therapy KB109 in Outpatients with Mild-to-Moderate COVID-19
Thu July 23, 2020 7:00 AM|GlobeNewswire|About: KLDO
- Second Study Launched in Kaleido’s COVID-19 Clinical Development Program for this Rapidly Growing Patient Population -
LEXINGTON, Mass., July 23, 2020 (GLOBE NEWSWIRE) -- Kaleido Biosciences, Inc. (KLDO)
https://kaleido.com/our-pipeline/
https://seekingalpha.com/symbol/KLDO
Kaleido Biosciences launches study of KB109 in COVID-19 patients
Jul. 23, 2020 8:13 AM ET|About: Kaleido Biosciences, Inc. (KLDO)|By: Douglas W. House, SA News Editor
RGLS4326 is a novel oligonucleotide designed to inhibit miR-17 & designed to target the kidney
RGLS4326 is a novel oligonucleotide designed to inhibit miR-17 & designed to target the kidney
RGLS4326 is a novel oligonucleotide designed to inhibit miR-17 & designed to target the kidney
Regulus Therapeutics Completes Dosing in Phase 1 Multiple Ascending Dose Study of RGLS4326 in Healthy Volunteers for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)
Wed July 22, 2020 8:07 AM|PR Newswire|About: RGLS
LA JOLLA, Calif., July 22, 2020 /PRNewswire/ -- Regulus Therapeutics Inc. (RGLS) (Nasdaq: RGLS)
https://seekingalpha.com/symbol/RGLS
Regulus Therapeutics concludes early-stage study of RGLS4326 in genetic kidney disease
Jul. 22, 2020 11:35 AM ET|About: Regulus Therapeutics Inc. (RGLS)|By: Vandana Singh, SA News Editor
Port Delivery System with ranibizumab (PDS) Lucentis® (ranibizumab injection)
tetracycline (including minocycline) for dermatological use (European Patent Application No. 1671416
tetracycline (including minocycline) for dermatological use (European Patent Application No. 1671416
Phase III Data Show Port Delivery System With Ranibizumab Enabled Over 98% of Patients to Go Six Months Between Treatments for Neovascular Age-Related Macular Degeneration
Wed July 22, 2020 12:00 PM|Business Wire|About: RHHBY
- In the Archway study, Port Delivery System with ranibizumab (PDS) demonstrated non-inferior and equivalent visual acuity outcomes compared with monthly ranibizumab eye injections, and a favorable benefit-risk profile
- PDS is a permanent refillable eye implant that continuously delivers a customized formulation of ranibizumab over a period of months, potentially reducing the treatment burden associated with frequent eye injections
- Data from the study will be discussed at the upcoming 38th Annual Meeting of the American Society of Retina Specialists (ASRS)
SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY)
Roche eye implant shows sustained benefit in wet AMD study
Jul. 22, 2020 12:20 PM ET|About: Roche Holding AG (RHHBY)|By: Douglas W. House, SA News Editor
https://seekingalpha.com/news/3593706-roche-eye-implant-shows-sustained-benefit-in-wet-amd-study
A Phase III Study to Evaluate the Port Delivery System With Ranibizumab Compared With Monthly Ranibizumab Injections in Participants With Wet Age-Related Macular Degeneration (Archway)
https://clinicaltrials.gov/ct2/show/NCT03677934?term=NCT03677934&draw=2&rank=1
Lucentis® (ranibizumab injection)
Archway (NCT03677934)
https://seekingalpha.com/symbol/RHHBY
https://www.businesswire.com/news/home/20200722005151/en/
tetracycline (including minocycline) for dermatological use (European Patent Application No. 1671416
tetracycline (including minocycline) for dermatological use (European Patent Application No. 1671416
tetracycline (including minocycline) for dermatological use (European Patent Application No. 1671416
Timber Pharmaceuticals Announces European Patent Office Intends to Grant Patent for BPX-01 and BPX-04
Wed July 22, 2020 8:00 AM|GlobeNewswire|About: TMBR
Stable topical composition has potential to reduce side effects of orally delivered compositions in treatment of acne and rosacea
WOODCLIFF LAKE, NJ, July 22, 2020 (GLOBE NEWSWIRE) -- via NEWMEDIAWIRE -- Timber Pharmaceuticals, Inc. (TMBR)
For more information, visit https://www.timberpharma.com/.
https://www.timberpharma.com/pipeline-1
https://seekingalpha.com/symbol/TMBR
https://www.timberpharma.com/tmb-001-topical-isotretinoin
https://www.timberpharma.com/tmb-002-topical-rapamycin
https://www.timberpharma.com/tmb-003-topical-subcutaneous-et-a-r
stabilized pre-fusion spike protein (CoV-2 S-2P)
tetracycline (including minocycline) for dermatological use (European Patent Application No. 1671416
True Human™ Therapeutic Antibodies anti-IL-1⍺ antibody
Preclinical Study Shows SARS-CoV-2 Spike Protein Licensed by Oragenics from the NIH Produces Neutralizing Antibodies
Wed July 22, 2020 7:30 AM|Business Wire|About: OGEN
Recently published paper supports the Company’s approach to COVID-19 vaccine development
TAMPA, Fla.--(BUSINESS WIRE)-- Oragenics, Inc. (OGEN)
SARS-CoV-2 mRNA Vaccine Development Enabled by Prototype Pathogen Preparedness
https://www.biorxiv.org/content/10.1101/2020.06.11.145920v1
visit www.oragenics.com
https://www.businesswire.com/news/home/20200722005308/en/
https://seekingalpha.com/symbol/OGEN
True Human™ Therapeutic Antibodies anti-IL-1⍺ antibody
nirogacestat, an investigational gamma secretase inhibitor
True Human™ Therapeutic Antibodies anti-IL-1⍺ antibody
XBiotech Achieves Milestone for Production of its New Potential Blockbuster Anti-IL-1⍺ Therapy
Wed July 22, 2020 9:20 AM|GlobeNewswire|About: XBITGlobeNewswire
AUSTIN, Texas, July 22, 2020 (GLOBE NEWSWIRE) -- XBiotech Inc. (XBIT)
For more information, visit www.xbiotech.com.
https://seekingalpha.com/symbol/XBIT
nirogacestat, an investigational gamma secretase inhibitor
nirogacestat, an investigational gamma secretase inhibitor
nirogacestat, an investigational gamma secretase inhibitor
SpringWorks Therapeutics Announces Full Enrollment of Phase 3 DeFi Trial Evaluating Nirogacestat in Adult Patients with Desmoid Tumors
Wed July 22, 2020 6:30 AM|GlobeNewswire|About: SWTX
STAMFORD, Conn., July 22, 2020 (GLOBE NEWSWIRE) -- SpringWorks Therapeutics, Inc. (SWTX)
www.clinicaltrials.gov under the identifier NCT03785964
https://www.springworkstx.com/
https://seekingalpha.com/symbol/SWTX
https://www.springworkstx.com/pipeline/nirogacestat/
Nirogacestat for Adults With Desmoid Tumor/Aggressive Fibromatosis (DT/AF) (DeFi)
https://clinicaltrials.gov/ct2/show/NCT03785964?term=NCT03785964&draw=1&rank=1
full enrollment in its Phase 3 DeFi trial evaluating nirogacestat, an investigational gamma secretase inhibitor, in adult patients with progressing desmoid tumors.
voclosporin, as a potential treatment for lupus nephritis
nirogacestat, an investigational gamma secretase inhibitor
nirogacestat, an investigational gamma secretase inhibitor
Aurinia Announces U.S. Food and Drug Administration Acceptance of the Filing of New Drug Application and Priority Review for Voclosporin for the Treatment of Lupus Nephritis
Tue July 21, 2020 5:27 PM|Business Wire|About: AUPH
- FDA grants Priority Review and sets PDUFA date of January 22, 2021 -
VICTORIA, British Columbia & ROCKVILLE, Md.--(BUSINESS WIRE)-- Aurinia Pharmaceuticals Inc. (AUPH)
https://www.businesswire.com/news/home/20200721005950/en/
https://seekingalpha.com/symbol/AUPH
https://www.auriniapharma.com/our-programs/about-voclosporin
biotecHOPE™ July 2020
Verdiperstat (BHV-3241)
Abivertinib is a novel small molecule tyrosine kinase inhibitor (TKI)
Abivertinib is a novel small molecule tyrosine kinase inhibitor (TKI)
Biohaven Completes Enrollment Ahead of Timelines in International Phase 3 Clinical Trial of Verdiperstat in Multiple System Atrophy
Tue July 21, 2020 7:30 AM|PR Newswire|About: BHVN
- Verdiperstat is a potential first-in-class, brain-penetrant inhibitor of myeloperoxidase (MPO), an enzyme implicated in neuroinflammation and neurodegeneration
- Approximately 300 patients enrolled in the 48-week trial and topline data anticipated by the end of 2021
PR Newswire
NEW HAVEN, Conn., July 21, 2020 /PRNewswire/ -- Biohaven Pharmaceutical Holding Company Ltd. (BHVN)
VERDIPERSTAT
For Initial Indications in Multiple System Atrophy (MSA) and Amyotrophic Lateral Sclerosis (ALS
https://www.biohavenpharma.com/science-pipeline/mpo/verdiperstat
https://www.biohavenpharma.com/
https://seekingalpha.com/symbol/BHVN
Biohaven completes enrollment in late-stage verdiperstat study in rare neurological disorder
Jul. 21, 2020 8:23 AM ET|About: Biohaven Pharmaceutica... (BHVN)|By: Douglas W. House, SA News Editor
Abivertinib is a novel small molecule tyrosine kinase inhibitor (TKI)
Abivertinib is a novel small molecule tyrosine kinase inhibitor (TKI)
Abivertinib is a novel small molecule tyrosine kinase inhibitor (TKI)
FDA clears Abivertinib for Phase 2 safety and efficacy study in hospitalized patients with moderate to severe COVID-19
Mon July 20, 2020 9:00 AM|PR Newswire|About: SRNE
- The compound demonstrated in-vitro the ability to simultaneously lower multiple critical inflammatory cytokines associated with cytokine storm and poor prognosis in COVID-19 patients.
- It is to be tested next in a Phase 2 trial in moderate to severe COVID-19 patients, hospitalized with developing cytokine storm in the lungs.
PR Newswire
SAN DIEGO, July 20, 2020 /PRNewswire/ -- Sorrento Therapeutics, Inc. (SRNE)
For more information visit www.sorrentotherapeutics.com
https://seekingalpha.com/symbol/SRNE
https://www.aceatherapeutics.com/
Enzastaurin
Abivertinib is a novel small molecule tyrosine kinase inhibitor (TKI)
RPGR Gene Therapy
FDA Grants Fast Track Designation For DB102 in Patients with Newly-Diagnosed Glioblastoma (GBM)
Fri July 17, 2020 8:00 AM|PR NewswirePR Newswire
SAN DIEGO, July 17, 2020 /PRNewswire/ -- Denovo Biopharma LLC,
Enzastaurin is an orally available small molecule serine/threonine kinase inhibitor of the PKC beta, PI3K and AKT pathways. The company in-licensed global rights from Eli Lilly (NYSE:LLY).
https://www.denovobiopharma.com/
https://seekingalpha.com/symbol/LLY
Glioblastoma
Denovo Biopharma's enzastaurin Fast Track'd for brain tumor
Jul. 17, 2020 8:18 AM ET|About: Eli Lilly and Company (LLY)|By: Douglas W. House, SA News Editor
https://seekingalpha.com/news/3592221-denovo-biopharmas-enzastaurin-fast-trackd-for-brain-tumor
RPGR Gene Therapy
Phase 1 trial evaluating eprenetapopt, in TP53 mutant acute myeloid leukemia (AML).
RPGR Gene Therapy
Interim Six-Month Data of RPGR Gene Therapy Shows Significant Vision Improvement in Patients Living with X-Linked Retinitis Pigmentosa
Fri July 17, 2020 7:30 AM|PR Newswire
Johnson & Johnson to review interim Phase 1/2 clinical trial findings in pre-recorded webcast
Company anticipates progressing the program to Phase 3
PR Newswire
RARITAN, N.J., July 17, 2020 /PRNewswire/ -- The Janssen Pharmaceutical Companies of Johnson & Johnson
Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) Retinitis Pigmentosa GTPase Regulator (RPGR)
Phase 1/2 trial (NCT03252847)
https://clinicaltrials.gov/ct2/show/NCT03252847?.
Retinitis pigmentosa
https://ghr.nlm.nih.gov/condition/retinitis-pigmentosa
J&J gene therapy shows encouraging action in vision loss disorder
Jul. 17, 2020 7:51 AM ET|About: Johnson & Johnson (JNJ)|By: Douglas W. House, SA News Editor
https://seekingalpha.com/symbol/JNJ
PB1046 in hospitalized COVID-19 patients
Phase 1 trial evaluating eprenetapopt, in TP53 mutant acute myeloid leukemia (AML).
Phase 1 trial evaluating eprenetapopt, in TP53 mutant acute myeloid leukemia (AML).
PhaseBio Doses First Patient in VANGARD Phase 2 Clinical Trial to Evaluate PB1046 for Hospitalized COVID-19 Patients
Thu July 16, 2020 4:05 PM|Business Wire|About: PHAS
Recently launched Phase 2 “VANGARD” trial to assess the efficacy and safety of PB1046 in hospitalized COVID-19 patients at high risk for rapid clinical deterioration and acute respiratory distress syndrome
PB1046, a long-acting analog of vasoactive intestinal peptide (VIP), has the potential to modulate several proinflammatory cytokines that are believed to be key drivers of the inflammatory response to COVID-19
MALVERN, Pa. & SAN DIEGO--(BUSINESS WIRE)-- PhaseBio Pharmaceuticals, Inc. (PHAS) (Nasdaq: PHAS)
PhaseBio Pharmaceuticals, Inc. (PHAS) (Nasdaq: PHAS)
PB1046, a Long-acting, Sustained Release Human VIP Analogue, Intended to Provide Clinical Improvement to Hospitalized COVID-19 Patients at High Risk for Rapid Clinical Deterioration and Acute Respiratory Distress Syndrome (ARDS). (VANGARD)
https://clinicaltrials.gov/ct2/show/NCT04433546
businesswire.com: https://www.businesswire.com/news/home/20200716006010/en/
Phase 2 clinical trial, VANGUARD, evaluating PhaseBio Pharmaceuticals' (NASDAQ:PHAS)
https://phasebio.com/pipeline/
Phase 1 trial evaluating eprenetapopt, in TP53 mutant acute myeloid leukemia (AML).
Phase 1 trial evaluating eprenetapopt, in TP53 mutant acute myeloid leukemia (AML).
Phase 1 trial evaluating eprenetapopt, in TP53 mutant acute myeloid leukemia (AML).
Aprea Therapeutics Announces Expansion of Clinical Trial Evaluating Eprenetapopt for the Front-Line Treatment of TP53 Mutant Acute Myeloid Leukemia (AML)
Thu July 16, 2020 7:00 AM|GlobeNewswire|About: APRE
- Commenced expansion cohort in combination with venetoclax and azacitidine
- Added cohort to be activated in combination with azacitidine
BOSTON, July 16, 2020 (GLOBE NEWSWIRE) -- Aprea Therapeutics (APRE), Inc. (Nasdaq: APRE)
https://seekingalpha.com/symbol/APRE
https://www.aprea.com/our-pipeline/
Oxford University's COVID-19 vaccine candidate ChAdOx1 nCoV-19 [ADZ1222
Oxford University's COVID-19 vaccine candidate ChAdOx1 nCoV-19 [ADZ1222
Oxford University's COVID-19 vaccine candidate ChAdOx1 nCoV-19 [ADZ1222
A Study of a Candidate COVID-19 Vaccine (COV001)
Phase 1 clinical trial evaluating Oxford University's COVID-19 vaccine candidate ChAdOx1 nCoV-19 [ADZ1222 by licensee AstraZeneca (NYSE:AZN)]
Oxford vaccine produces "double defence" against SARS-CoV-2
Jul. 15, 2020 4:59 PM ET|About: AstraZeneca PLC (AZN)|By: Douglas W. House, SA News Editor
Coronavirus vaccine hopes rise after strong trial results
Vaccine by Oxford scientists may offer 'double defence' against the virus
ByBill Gardner ; Victoria Ward ; Sarah Newey and Henry Bodkin, HEALTH AND SCIENCE CORRESPONDENT15 July 2020 • 9:55pm
licensee AstraZeneca (NYSE:AZN)]
oral insulin capsule (ORMD-0801) for type 2 diabetics
Oxford University's COVID-19 vaccine candidate ChAdOx1 nCoV-19 [ADZ1222
Oxford University's COVID-19 vaccine candidate ChAdOx1 nCoV-19 [ADZ1222
Oramed Reports Positive End of Phase 2 Meeting With the FDA for Oral Insulin
Wed July 15, 2020 8:55 AM|PR Newswire|About: ORMP
NEW YORK, July 15, 2020 /PRNewswire/ -- Oramed Pharmaceuticals Inc. (ORMP) (TASE: ORMP) (www.oramed.com)
ORMD 0801 – Oral Insulin Capsule for T2DM
https://www.oramed.com/pipeline/ormd-0801-type-2/
ORMD 0901 – Oral GLP-1 for T2DM
https://www.oramed.com/pipeline/ormd-0901/
https://seekingalpha.com/symbol/ORMP
AT-301, its proprietary COVID-19 nasal spray drug candidate
Oxford University's COVID-19 vaccine candidate ChAdOx1 nCoV-19 [ADZ1222
AT-301, its proprietary COVID-19 nasal spray drug candidate
Atossa Therapeutics Announces Successful In Vitro Testing of Nasal Spray Formulation: AT-301 Inhibits SARS-CoV-2 Infectivity of VERO Cells in Laboratory Culture
Wed July 15, 2020 9:30 AM|GlobeNewswire|About: ATOSGlobeNewswire
SEATTLE, July 15, 2020 (GLOBE NEWSWIRE) -- Atossa Therapeutics, Inc. (ATOS) (Nasdaq: ATOS)
https://atossatherapeutics.com/
https://seekingalpha.com/symbol/ATOS
Atossa Therapeutics up 24% on encouraging COVID-19 nasal spray data
Jul. 15, 2020 10:00 AM ET|About: Atossa Therapeutics, Inc. (ATOS)|By: Mamta Mayani, SA News Editor
GC4419 (Avasopasem Manganese)
aprepitant in early hospitalized adult COVID-19 patients
AT-301, its proprietary COVID-19 nasal spray drug candidate
Dose Escalation Trial of Stereotactic Body Radiation Therapy (SBRT) in Combination With GC4419 in Pancreatic Cancer
Galera Therapeutics Completes Enrollment of Phase 1b/2a Clinical Trial of GC4419 in Combination with Radiotherapy for Locally Advanced Pancreatic Cancer
Mon July 13, 2020 5:46 PM|GlobeNewswire|About: GRTXGlobeNewswire
MALVERN, Pa., July 13, 2020 (GLOBE NEWSWIRE) -- Galera Therapeutics, Inc. (GRTX)
https://seekingalpha.com/symbol/GRTX
Galera completes enrollment in mid-stage study of lead drug in pancreatic cancer
Jul. 13, 2020 6:06 PM ET|About: Galera Therapeutics, Inc. (GRTX)|By: Vandana Singh, SA News Editor
GOCOVRI® (amantadine) extended release capsules
aprepitant in early hospitalized adult COVID-19 patients
aprepitant in early hospitalized adult COVID-19 patients
Adamas presents new post-hoc Phase 3 data analysis for GOCOVRI® in Parkinson’s disease at the 2020 American Academy of Neurology (AAN) Science Highlights Platform
Wed July 15, 2020 8:00 AM|GlobeNewswire|About: ADMS
EMERYVILLE, Calif., July 15, 2020 (GLOBE NEWSWIRE) -- Adamas Pharmaceuticals, Inc. (ADMS)
GOCOVRI is indicated for the treatment of dyskinesia in people with Parkinson's disease receiving levodopa-based therapy. The abstract and poster presentation are available on the 2020 AAN Science Highlights Platform.
Please see full Prescribing Information for additional important safety information at https://www.gocovri.com/assets/pdfs/Gocovri_Prescribing_Information.pdf.
Adamas announces post-hoc data supporting benefit of Gocovri in Parkinson's
Jul. 15, 2020 11:39 AM ET|About: Adamas Pharmaceuticals, Inc. (ADMS)|By: Vandana Singh, SA News Editor
aprepitant in early hospitalized adult COVID-19 patients
aprepitant in early hospitalized adult COVID-19 patients
aprepitant in early hospitalized adult COVID-19 patients
Aprepitant Injectable Emulsion in Patients With COVID-19 (GUARDS-1)
ClinicalTrials.gov of a Phase 2 clinical trial, GUARDS-1, evaluating aprepitant in early hospitalized adult COVID-19 patients.
Heron Therapeutics readies mid-stage study of aprepitant for COVID-19
Jul. 14, 2020 1:07 PM ET|About: Heron Therapeutics, Inc. (HRTX)|By: Douglas W. House, SA News Editor
https://seekingalpha.com/symbol/HRTX
http://www.herontx.com/cinvanti
Heron Therapeutics Announces Initiation of Phase 2 Clinical Study of CINVANTI® for the Treatment of COVID-19
Thu July 16, 2020 8:30 AM|PR Newswire|About: HRTX
SAN DIEGO, July 16, 2020 /PRNewswire/ -- Heron Therapeutics, Inc. (HRTX)