BiotecHOPE™ Every Victory
May 23, 2021 2:00 PM ETABBV, AMGN...
Mar. 14, 2021 3:08 PM ET. AZN, BNTX, JNJ, LLY, MRK, MRNA, NVAX, PFE, VIR
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.
•Premiered Jan 29, 2021 Danny Gokey
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
•Jul 20, 2020 Elevation Worship
Written by Steven Furtick, Chris Brown
Aug. 26, 2020 5:37 PM ET|
"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""
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:
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.
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.
Aug. 25, 2021 7:51 AM ET
By: Mamta Mayani, SA News Editor
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.
Aug. 11, 2021 4:06 PM ET
By: Aakash Babu, SA News Editor
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
Jul. 21, 2021 9:55 AM ET
By: Aakash Babu, SA News Editor
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
Jul. 19, 2021 9:45 AM ET
Download as PDFJuly 19, 2021 6:00am EDT
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:
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.
Jul. 19, 2021 6:19 AM ET
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.
Jul. 15, 2021 8:21 AM ET
By: Aakash Babu, SA News Editor
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.”
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
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/.
Jul. 14, 2021 7:30 AM ET
By: Mamta Mayani, SA News Editor
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:
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.
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 email@example.com or call 877-256-9526 in North America and +31 20 369 7861 in Europe.
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.
June 11, 2021 at 4:11 PM EDTDownload PDF
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.
In addition to the intravenous formulation of COVI-AMG™ neutralizing antibody, Sorrento is developing COVIDROPS which will be the intranasal formulation of COVI-AMG™.
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.
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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:
Additionally, Innovent currently has regulatory submissions under review in China for TYVYT® (sintilimab Injection):
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
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/
Jun. 05, 2021 10:12 AM ET
By: Jonathan M Block, SA News Editor
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:
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/
By: Mamta Mayani, SA News Editor
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.
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
May 19, 2021 8:53 AM ET
By: Mamta Mayani, SA News Editor
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.
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.
May 19, 2021 8:36 AM ET
By: Mamta Mayani, SA News Editor
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
May 14, 2021 at 7:05 AM EDT
ROCKVILLE, Md., May 14, 2021 /PRNewswire/ --
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 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.
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.
May 14, 2021 7:38 AM ET
By: Mamta Mayani, SA News Editor
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:
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).
May 07, 2021 8:22 AM ETVaxart, Inc. (VXRT)
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:
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
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SOURCE Compugen Ltd.
Apr. 27, 2021 8:15 AM ET
By: Mamta Mayani, SA News Editor
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.
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.
Apr. 26, 2021 5:03 PM ET
By: Aakash Babu, SA News Editor
Apr 22, 2021
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.
Apr. 22, 2021 8:28 AM ET
By: Dulan Lokuwithana, SA News Editor
Apr 22, 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)
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.
For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com.
Apr. 22, 2021 8:42 AM ET
By: Mamta Mayani, SA News Editor
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
First in class, anti-SEMA4D therapy for neuroinflammatory/neurodegenerative
BAVENCIO is a prescription medicine used to treat:
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.
It is not known if BAVENCIO is safe and effective in children under the age of 12.
You may also report side effects to EMD Serono at 1-800-283-8083 ext.5563.
Apr 19, 2021Download PDF
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.
For more information, visit www.xbiotech.com.
Apr. 19, 2021 4:27 AM ET XBiotech Inc. (XBIT)
Apr 19, 2021 DownloadPDF Format (opens in new window)
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
Apr. 19, 2021 9:37 AM ET
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.
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
Apr. 15, 2021 7:38 AM ET
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:
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.
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
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.”
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.
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.
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.
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.
April 10, 2021 Download this Press ReleasePDF Format (opens in new window)
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/
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/.
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.
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.
"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.
For more information about the Company, please visit http://www.moleculin.com.
Mar. 30, 2021 8:04 AM ET
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.
Copiktra should be prescribed by physicians experienced in the treatment of cancer.
19 Studies found for: CH5126766 OR defactinib | Recruiting, Not yet recruiting, Active, not recruiting, Completed, Enrolling by invitation Studies
Mar. 26, 2021 7:53 AM ET
Oncology / Immunology, Press Releases, RNS Announcements | 24 Mar 2021
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.
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.
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 and SANET-p 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. 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, 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.
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 (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.
For more information, please visit: www.hutch-med.com.
Mar. 24, 2021 3:17 AM ET
By: Mamta Mayani, SA News Editor
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.
SOURCE Oramed Pharmaceuticals Inc.
Mar 23, 2021
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.
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.
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.
Mar. 23, 2021 4:07 AM ET
By: Mamta Mayani, SA News Editor
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.
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
Mar. 16, 2021 8:35 AM ET
By: Mamta Mayani, SA News Editor
AC Immune Announces New Clinical Results in Down Syndrome and Plans for Future Development of Anti-Amyloid-Beta Vaccine
March 16, 2021
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
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
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.
Mar. 16, 2021 8:22 AM ET
By: Aakash Babu, SA News Editor
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.
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/
Mar. 16, 2021 7:07 AM ET
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.
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.
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/
Mar. 16, 2021 7:01 AM ET
By: Gaurav Batavia, SA News Editor
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 firstname.lastname@example.org 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/
Mar. 15, 2021 6:31 AM ET
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.
Mar. 13, 2021 11:59 PM ET
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 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
AC Immune Presents New Preclinical Data for Therapeutic and Diagnostic Candidates Addressing Novel Targets for Neurodegenerative Diseases
March 09, 2021
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
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.
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,
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.
Tue March 9, 2021 1:00 AM| Accesswire
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.
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
Mar 8, 2021PDF Version
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.
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.
Monday, March 08, 2021 - 08:30amEST
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/
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.
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
Mar. 03, 2021 5:02 PM ET
News March 2, 2021argenx Announces FDA Acceptance of BLA Filing for Efgartigimod for the Treatment of Generalized Myasthenia Gravis
Breda, the Netherlands
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
Mar. 02, 2021 2:32 AM ET
By: Mamta Mayani, SA News Editor
February 27, 2021 at 6:25 PM EST
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.
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
Feb. 27, 2021 9:04 PM ET
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’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.
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.
Feb. 24, 2021 7:27 AM ET
By: Dulan Lokuwithana, SA News Editor
Feb 22, 2021
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.
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.
February 16, 2021 at 7:05 AM EST
ROCKVILLE, Md., Feb. 16, 2021 /PRNewswire/ --
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 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.