Moorestown, New Jersey, United States
biotecHOPE™ 2022
biotecHOPE™ 2022
RELYVRIO™ (sodium phenylbutyrate and taurursodiol) (AMX0035)
RELYVRIO™ (sodium phenylbutyrate and taurursodiol) (AMX0035)
RELYVRIO™ (sodium phenylbutyrate and taurursodiol) (AMX0035)
RELYVRIO™ (sodium phenylbutyrate and taurursodiol) (AMX0035)
RELYVRIO™ (sodium phenylbutyrate and taurursodiol) (AMX0035)
AMYLYX PHARMACEUTICALS ANNOUNCES FDA APPROVAL OF RELYVRIO™ FOR THE TREATMENT OF ALS
September 29, 2022 at 6:30 PM EDT
- RELYVRIO (previously known as AMX0035 in the U.S.) is an oral, fixed-dose combination therapy for the treatment of adults with ALS
- RELYVRIO significantly slowed loss of physical function in a randomized, placebo-controlled clinical trial in ALS
- Detailed data from the CENTAUR clinical trial were published in the New England Journal of Medicine, Muscle & Nerve, and the Journal of Neurology, Neurosurgery and Psychiatry
- Amylyx to host investor conference call tomorrow, September 30 at 8:00 a.m. ET
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sep. 29, 2022--
Amylyx Pharmaceuticals, Inc. (NASDAQ: AMLX) (“Amylyx” or the “Company”) today announced that the U.S. Food and Drug Administration (FDA) has approved RELYVRIO™ (sodium phenylbutyrate and taurursodiol) for the treatment of adults with amyotrophic lateral sclerosis (ALS). RELYVRIO (previously known as AMX0035 in the U.S.) significantly slowed the loss of physical function in people living with ALS in a randomized, placebo-controlled clinical trial. RELYVRIO can be taken as a monotherapy or with existing approved treatments.
“Today’s FDA approval of RELYVRIO is an exciting milestone for the ALS community and is a major step toward achieving our mission to one day end the suffering caused by neurodegenerative diseases,” said Joshua Cohen and Justin Klee, Co-CEOs of Amylyx. “We want to give a heartfelt thank you to the broader ALS community, including healthcare professionals and those living with ALS, for their guidance, support of our clinical programs, and for sharing their experiences with us. Their stories inspired us and helped our team to better understand the ALS clock, instilling in us a deep sense of urgency that will continue to drive us forward. This is just the beginning and there is much more to be done.”
ALS is a relentlessly progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis and eventually, death. More than 90% of people with ALS have sporadic disease, showing no clear family history. ALS affects approximately 29,000 people in the U.S.
Leading U.S. ALS advocacy organizations including The ALS Association, Answer ALS Foundation, I AM ALS, Les Turner ALS Foundation and Team Gleason said in a statement, “Our organizations have been on a mission to create a world free of ALS. With today’s approval, we are encouraged that RELYVRIO can offer people living with ALS and their families the potential of more time with functional independence. This is especially important for a rapidly progressive disease with a median survival time from diagnosis of just two to three years. This is significant for people living with ALS, their loved ones, caregivers, clinicians, researchers, and advocacy, as we now have a new treatment option that could be a big step forward for the future of ALS care.”
The approval of RELYVRIO is based on data from CENTAUR, a multicenter Phase 2 clinical trial in 137 participants with ALS encompassing a 6-month randomized, placebo-controlled phase and an open-label extension (OLE) long-term follow-up phase. Detailed data from CENTAUR were published in the New England Journal of Medicine, Muscle & Nerve, and the Journal of Neurology, Neurosurgery, and Psychiatry.
The most common adverse events occurring with RELYVRIO (at least 15% and at least 5% greater than placebo) were diarrhea, abdominal pain, nausea, and upper respiratory tract infection. Gastrointestinal-related adverse reactions occurred throughout the study but were more frequent during the first three weeks of treatment.
“Any time we have a new tool to slow the progression of this disease represents an important milestone in how we battle ALS. The published data on both function and survival in a randomized trial – and what this means for people living with ALS – are a step forward for the ALS community,” said Sabrina Paganoni, M.D., Ph.D., principal investigator of the CENTAUR trial, investigator at the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital, and Associate Professor of Physical Medicine and Rehabilitation at Harvard Medical School and Spaulding Rehabilitation Hospital.
“The approval of a new treatment that helps slow the progression of ALS, preserve physical function and potentially extend survival has the potential to greatly impact the hundreds of people living with ALS who I currently treat,” said Merit Cudkowicz, M.D., co-principal investigator of the CENTAUR trial and co-founder of the Northeast ALS Consortium, Chief of Neurology and the Director of the Healey & AMG Center for ALS and Chief of Neurology at Massachusetts General Hospital, and the Julieanne Dorn Professor of Neurology at Harvard Medical School. “There are too few options to target this uniformly fatal and rapid illness, and I am encouraged at this outcome and what it represents for my patients and their families.”
“Our priority now is to ensure that adults living with ALS in the U.S. whose doctors have prescribed RELYVRIO can access it as quickly as possible,” said Margaret Olinger, Global Head of Commercial and Chief Commercial Officer of Amylyx. “Physicians will be able to prescribe immediately, and we anticipate specialty pharmacies will be able to start to fill prescriptions and ship RELYVRIO to people with ALS in the next four to six weeks.”
After considering the input of many stakeholders throughout the ALS community in the U.S., Amylyx made the decision to price RELYVRIO below the latest FDA-approved product available to people with ALS. To ensure that every eligible person who can benefit from RELYVRIO will have access, Amylyx will provide support to healthcare professionals, people living with ALS, and their loved ones through the Amylyx Care Team (ACT) Support Program. ACT provides people living with ALS who have been prescribed RELYVRIO, and their loved ones, with a dedicated, single point of contact to guide their treatment journey. ACT works tirelessly to aid with navigating through insurance in an effort to overcome potential barriers to access. ACT helps confirm coverage and provides financial assistance options to eligible individuals with out-of-pocket costs. ACT also provides education, support, and resources to help adults living with ALS get started and continue with RELYVRIO treatment, as prescribed by their healthcare provider. People with ALS, their caregivers, and healthcare professionals in the U.S. can now call 1-866-318-2989 or email amylyxcareteam@amylyx.com to speak with an ACT team member.
About the CENTAUR Trial
CENTAUR was a multicenter Phase 2 clinical trial in 137 participants with ALS encompassing a 6-month randomized placebo-controlled phase and an open-label extension (OLE) long-term follow-up phase. The trial met its primary efficacy endpoint.
Detailed safety and functional efficacy data from CENTAUR were published in the New England Journal of Medicine. Data from additional analyses from the CENTAUR trial were published in Muscle & Nerve in 2020 and 2022, and the Journal of Neurology, Neurosurgery and Psychiatry in 2022.
The CENTAUR trial was funded, in part, by the ALS ACT grant and the ALS Ice Bucket Challenge, and was supported by The ALS Association, ALS Finding a Cure (a program of The Leandro P. Rizzuto Foundation), the Northeast ALS Consortium, and the Sean M. Healey & AMG Center for ALS at Mass General.
About RELYVRIO™ (previously known as AMX0035 in the U.S.)
RELYVRIO™ (sodium phenylbutyrate and taurursodiol) is an oral, fixed-dose medication approved to treat amyotrophic lateral sclerosis (ALS) in adults in the U.S. and approved with conditions as ALBRIOZA™ for the treatment of ALS in Canada. Additionally, the European Medicines Agency (EMA) is reviewing the Company’s Marketing Authorisation Application for AMX0035 for the treatment of ALS in Europe. AMX0035 is being explored for the potential treatment of other neurodegenerative diseases.
INDICATION AND IMPORTANT SAFETY INFORMATION FOR RELYVRIO (sodium phenylbutyrate/taurursodiol), for oral suspension
INDICATION
RELYVRIO is indicated for the treatment of amyotrophic lateral sclerosis (ALS) in adults.
Please click here to read the full Prescribing Information for RELYVRIO™.
About Amylyx Pharmaceuticals
Amylyx Pharmaceuticals, Inc. is committed to supporting and creating more moments for the neurodegenerative community through the discovery and development of innovative new treatments. Amylyx is headquartered in Cambridge, Massachusetts and has operations in Canada and EMEA. For more information, visit amylyx.com and follow us on LinkedIn and Twitter. For investors, please visit investors.amylyx.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220929005962/en/
Source: Amylyx Pharmaceuticals, Inc.
Amylyx wins FDA approval of ALS drug Relyvrio; shares up 12% after hours
Sep. 29, 2022 5:40 PM ET
Amylyx Pharmaceuticals, Inc. (AMLX)
By: Jonathan Block, SA News Editor2 Comments
The US FDA approved Amylyx Pharmaceuticals (NASDAQ:AMLX) Relyvrio for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease.
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CFT1946
RELYVRIO™ (sodium phenylbutyrate and taurursodiol) (AMX0035)
Lecanemab
C4 Therapeutics Receives Study May Proceed Letter from U.S. FDA to Initiate Phase 1/2 Clinical Trial of CFT1946, an Orally Bioavailable BiDAC™ Degrader, in BRAF-V600 Mutant Solid Cancers
Phase 1/2 Clinical Trial Will Study CFT1946 in BRAF-V600 Mutant Solid Cancers Including Lung, Colorectal and Melanoma; Trial Initiation Expected by Year End 2022
WATERTOWN, Mass., Sept. 29, 2022 (GLOBE NEWSWIRE) -- C4 Therapeutics, Inc. (C4T) (Nasdaq: CCCC), a clinical-stage biopharmaceutical company dedicated to advancing targeted protein degradation science to develop a new generation of small-molecule medicines and transform how disease is treated, today announced it has received a Study May Proceed Letter from the United States Food and Drug Administration (FDA) to begin a Phase 1/2 trial for CFT1946, an orally bioavailable mutant-selective BiDAC™ degrader for the treatment of BRAF-V600 mutant solid tumors.
“The Study May Proceed Letter from the FDA, which marks C4T’s third successful oncology investigational new drug application in less than two years, demonstrates the power of our TORPEDO platform to build an exciting portfolio of degrader medicines that have the potential to transform how diseases are treated,” said Andrew Hirsch, president and chief executive officer of C4 Therapeutics. “We designed CFT1946 to be a potent and selective degrader of mutant BRAF-V600, which accounts for approximately 50,000 cancer diagnoses annually. We believe our innovative therapeutic candidate may overcome some limitations of BRAF inhibitor treatments to offer cancer patients the potential for deeper and more durable responses.”
The Phase 1/2 clinical trial will primarily investigate safety, tolerability, and anti-tumor activity, with secondary and exploratory objectives to characterize the pharmacokinetic and pharmacodynamic profile of CFT1946. The initial arm of the Phase 1 portion of the study will evaluate CFT1946 as a single agent in patients with BRAF-V600 mutant solid tumors. As the Phase 1 trial progresses, an additional arm of the trial will evaluate CFT1946 in combination with trametinib in patients with BRAF-V600 mutant solid tumors. Following the identification of the recommended dose, the Phase 2 portion of the trial is expected to expand to three investigational arms to evaluate: (1) CFT1946 monotherapy in patients with V600 mutant melanoma or non-small cell lung cancer (NSCLC) after prior BRAF inhibitor treatment; (2) CFT1946 in combination with trametinib in patients with V600 mutant melanoma or NSCLC after prior BRAF inhibitor treatment; and (3) CFT1946 in combination with trametinib in patients with V600 mutant NSCLC who have not previously been treated with a BRAF inhibitor.
About C4 Therapeutics
C4 Therapeutics (C4T) (Nasdaq: CCCC) is a clinical-stage biopharmaceutical company dedicated to delivering on the promise of targeted protein degradation science to create a new generation of medicines that transform patients’ lives. C4T is leveraging its TORPEDO® platform to efficiently design and optimize small-molecule medicines that harness the body’s natural protein recycling system to rapidly degrade disease-causing proteins, offering the potential to overcome drug resistance, drug undruggable targets and improve patient outcomes. C4T is advancing multiple targeted oncology programs to the clinic and expanding its research platform to deliver the next wave of medicines for difficult-to-treat diseases. For more information, please visit www.c4therapeutics.com.
About CFT1946
CFT1946 is an orally bioavailable BiDAC degrader designed to be potent and selective against BRAF-V600 mutant targets. In pre-clinical studies, CFT1946 is active in vivo and in vitro in models with BRAF-V600E-driven disease and in the escape mutant BRAF models. C4T is advancing CFT1946 to the clinic to study treatment for BRAF-V600 mutant solid tumors including lung, colorectal or melanoma.
C4 Therapeutics wins FDA nod to study cancer candidate in solid tumors
Sep. 29, 2022 8:14 AM ET
By: Dulan Lokuwithana, SA News Editor
- C4 Therapeutics, Inc. (NASDAQ:CCCC) added ~7% pre-market Thursday after the clinical-stage biotech announced that the FDA issued a Study May Proceed Letter to start a Phase 1/2 trial for its oral solid tumor candidate CFT1946.
- https://seekingalpha.com/news/3887093-cccc-stock-on-watch-after-fda-nod-to-study-cancer-candidate-in-solid-tumors
- https://seekingalpha.com/symbol/CCCC
- https://c4therapeutics.com/pipeline
- https://c4therapeutics.com/
Lecanemab
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Lecanemab
LECANEMAB CONFIRMATORY PHASE 3 CLARITY AD STUDY MET PRIMARY ENDPOINT, SHOWING HIGHLY STATISTICALLY SIGNIFICANT REDUCTION OF CLINICAL DECLINE IN LARGE GLOBAL CLINICAL STUDY OF 1,795 PARTICIPANTS WITH EARLY ALZHEIMER’S DISEASE
SEPTEMBER 27, 2022 • INVESTOR RELATIONS
- ALL KEY SECONDARY ENDPOINTS ALSO MET, DEMONSTRATING HIGHLY STATISTICALLY SIGNIFICANT RESULTS
- PROFILE OF AMYLOID-RELATED IMAGING ABNORMALITIES (ARIA) INCIDENCE WAS WITHIN EXPECTATIONS
- EISAI AIMS TO FILE FOR TRADITIONAL APPROVAL IN THE U.S., AND TO SUBMIT MARKETING AUTHORIZATION APPLICATIONS IN JAPAN AND EUROPE BY THE END OF EISAI FY2022, WHICH ENDS ON MARCH 31, 2023
TOKYO and CAMBRIDGE, Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, "Eisai") and Biogen Inc. (Nasdaq: BIIB, Corporate headquarters: Cambridge, Massachusetts, CEO: Michel Vounatsos, "Biogen") announced positive topline results from Eisai’s large global Phase 3 confirmatory Clarity AD clinical trial of lecanemab (development code: BAN2401), an investigational anti-amyloid beta (Aβ) protofibril antibody for the treatment of mild cognitive impairment (MCI) due to Alzheimer’s disease (AD) and mild AD (collectively known as early AD) with confirmed presence of amyloid pathology in the brain. Lecanemab met the primary endpoint (CDR-SB: Clinical Dementia Rating-Sum of Boxes*) and all key secondary endpoints with highly statistically significant results. Eisai will discuss this data with regulatory authorities in the U.S., Japan and Europe with the aim to file for traditional approval in the US and for marketing authorization applications in Japan and Europe by the end of Eisai’s FY2022, which ends March 31, 2023. Additionally, Eisai will present the Clarity AD study results on November 29, 2022, at the Clinical Trials on Alzheimer’s Congress (CTAD), and publish the findings in a peer-reviewed medical journal.
* CDR-SB is a numeric scale used to quantify the various severity of symptoms of dementia. Based on interviews of people living with AD and family/caregivers, qualified healthcare professionals assess cognitive and functional performance in six areas: memory, orientation, judgment and problem solving, community affairs, home and hobbies, and personal care. The total score of the six areas is the score of CDR-SB, and CDR-SB is also used as an appropriate item for evaluating the effectiveness of therapeutic drugs targeting the early stages of AD.
Lecanemab treatment met the primary endpoint and reduced clinical decline on the global cognitive and functional scale, CDR-SB, compared with placebo at 18 months by 27%, which represents a treatment difference in the score change of -0.45 (p=0.00005) in the analysis of Intent-to-treat (ITT) population. Starting as early as six months, across all time points, the treatment showed highly statistically significant changes in CDR-SB from baseline compared to placebo (all p-values are less than 0.01). All key secondary endpoints were also met with highly statistically significant results compared with placebo (p<0.01). Key secondary endpoints were the change from baseline at 18 months compared with placebo of treatment in amyloid levels in the brain measured by amyloid positron emission tomography (PET), the AD Assessment Scale-cognitive subscale14 (ADAS-cog14), AD Composite Score (ADCOMS) and the AD Cooperative Study-Activities of Daily Living Scale for Mild Cognitive Impairment (ADCS MCI-ADL).
The incidence of amyloid-related imaging abnormalities-edema/effusion (ARIA-E), an adverse event associated with anti-amyloid antibodies, was 12.5% in the lecanemab group and 1.7% in the placebo group. The incidence of symptomatic ARIA-E was 2.8% in the lecanemab group and 0.0% in the placebo group. The ARIA-H (ARIA cerebral microhemorrhages, cerebral macrohemorrhages, and superficial siderosis) rate was 17.0% in the lecanemab group and 8.7% in the placebo group. The incidence of symptomatic ARIA-H was 0.7% in the lecanemab group and 0.2% in the placebo group. There was no imbalance in isolated ARIA-H (i.e., ARIA-H in patients who did not also experience ARIA-E) between lecanemab (8.8%) and placebo (7.6%). The total incidence of ARIA (ARIA-E and/or ARIA-H) was 21.3% in the lecanemab group and 9.3% in the placebo group. Overall, lecanemab’s ARIA incidence profile was within expectations.
Clarity AD was a global confirmatory Phase 3 placebo-controlled, double-blind, parallel-group, randomized study in 1,795 people with early AD. The treatment group was administered a dosage of 10 mg/kg bi-weekly of lecanemab, with participants allocated in a 1:1 ratio to receive either placebo or lecanemab. The baseline characteristics of both placebo and lecanemab groups are similar and well balanced. Eligibility criteria allowed patients with a broad range of comorbidities/comedications: hypertension, diabetes, heart disease, obesity, renal disease and anti-coagulants, etc. Eisai’s recruitment strategy for the Clarity AD clinical trial ensured greater inclusion of ethnic and racial populations in the U.S., resulting in approximately 25% of the total U.S. enrollment including Hispanic and African American persons living with early AD. Due to the inclusive eligibility criteria and the successful recruitment of diverse ethnic and racial populations in the U.S., Clarity AD’s population is generally comparable to the country’s Medicare population.
“Since Eisai launched Aricept in the U.S. and Japan in the late 1990s and obtained its approval in over 100 countries, Eisai has provided the drug to people living with dementia while building empathy for them and their families through disease education efforts and community involvement. The positive result of the lecanemab, an anti-Aβ protofibril antibody, pivotal study after almost 25 years since Aricept’s launch is an important milestone for Eisai in fulfilling our mission to meet the expectations of the Alzheimer’s disease community. Alzheimer’s disease not only presents a great challenge for patients and their families, but it also negatively impacts society, including decreased productivity, increased social costs and disease-related anxiety. We believe that helping to alleviate these burdens will positively impact society as a whole,” said Haruo Naito, Chief Executive Officer at Eisai. “Additionally, the lecanemab Clarity AD study results prove the amyloid hypothesis, in which the abnormal accumulation of Aβ in the brain is one of the main causes of Alzheimer’s disease, when targeted with a protofibril-binding therapy. Eisai believes these findings will create new horizons in the diagnosis and treatment of Alzheimer’s disease as well as further activate innovation for new treatment options. The successful results of the Clarity AD clinical trial would not be possible without the truly inspiring dedication of the study’s participants, their families and caregivers and the clinical investigators around the world. We thank all the people involved in the study for their invaluable contributions.”
“Today’s announcement gives patients and their families hope that lecanemab, if approved, can potentially slow the progression of Alzheimer’s disease, and provide a clinically meaningful impact on cognition and function,” said Michel Vounatsos, Chief Executive Officer at Biogen. “Importantly, the study shows that removal of aggregated amyloid beta in the brain is associated with a slowing of disease in patients at the early stage of the disease. We want to thank the many patients who participated in this groundbreaking global study and want to acknowledge the clinical investigators who worked tirelessly to increase the enrollment of traditionally underrepresented populations. As pioneers in neuroscience, we believe defeating this disease will require multiple approaches and treatment options, and we look forward to continuing the discussion about the significance of these findings with the patient, scientific, and medical communities.”
In July 2022, the U.S. Food and Drug Administration (FDA) accepted Eisai’s Biologics License Application (BLA) for lecanemab under the accelerated approval pathway and granted Priority Review. The Prescription Drugs User Fee Act action date (PDUFA) is set for January 6, 2023. The FDA has agreed that the results of Clarity AD can serve as the confirmatory study to verify the clinical benefit of lecanemab. In an effort to secure traditional FDA approval for lecanemab as soon as possible, Eisai submitted the BLA through the FDA’s Accelerated Approval Pathway so that the agency could complete its review of all lecanemab data with the exception of the data from the confirmatory Clarity AD study.
In March 2022, Eisai began submitting application data, with the exception of Clarity AD data, to Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) under the prior assessment consultation system with the aim of obtaining early approval for lecanemab so that people living with early AD may have access to the therapy as soon as possible.
Eisai serves as the lead of lecanemab development and regulatory submissions globally with both Eisai and Biogen co-commercializing and co-promoting the product and Eisai having final decision-making authority.
2. About Lecanemab
Lecanemab is an investigational humanized monoclonal antibody for AD that is the result of a strategic research alliance between Eisai and BioArctic. Lecanemab selectively binds to neutralize and eliminate soluble, toxic amyloid-beta (Aβ) aggregates (protofibrils) that are thought to contribute to the neurodegenerative process in AD. As such, lecanemab may have the potential to have an effect on disease pathology and to slow down the progression of the disease. Currently, lecanemab is being developed as the only anti- Aβ antibody that can be used for the treatment of early AD without the need for titration. With regard to the results from pre-specified analysis at 18 months of treatment, Study 201 demonstrated reduction of brain Aβ accumulation (P<0.0001) and slowing of disease progression measured by ADCOMS (P<0.05) in early AD subjects. The study did not achieve its primary outcome measure* at 12 months of treatment. The Study 201 open-label extension was initiated after completion of the Core period and a Gap period off treatment of 9-59 months (average of 24 months, n=180 from core study enrolled) to evaluate safety and efficacy, and is underway.
Since July 2020 the Phase 3 clinical study (AHEAD 3-45) for individuals with preclinical AD, meaning they are clinically normal and have intermediate or elevated levels of amyloid in their brains, is ongoing. AHEAD 3-45 is conducted as a public-private partnership between the Alzheimer’s Clinical Trial Consortium that provides the infrastructure for academic clinical trials in AD and related dementias in the U.S, funded by the National Institute on Aging, part of the National Institutes of Health, Eisai and Biogen.
Since January 2022, the Tau NexGen clinical study for Dominantly Inherited AD (DIAD), that is conducted by Dominantly Inherited Alzheimer Network Trials Unit (DIAN-TU), led by Washington University School of Medicine in St. Louis, is ongoing.
Furthermore, Eisai has initiated a lecanemab subcutaneous dosing Phase 1 study.
* An 80% or higher estimated probability of demonstrating 25% or greater slowing in clinical decline at 12 months treatment measured by ADCOMS from baseline compared to placebo.
4. About the Collaboration between Eisai and Biogen for AD
Eisai and Biogen have been collaborating on the joint development and commercialization of AD treatments since 2014. Eisai serves as the lead of lecanemab development and regulatory submissions globally with both companies co-commercializing and co-promoting the product and Eisai having final decision-making authority.
5. About the Collaboration between Eisai and BioArctic for AD
Since 2005, Eisai and BioArctic have had a long-term collaboration regarding the development and commercialization of AD treatments. Eisai obtained the global rights to study, develop, manufacture and market lecanemab for the treatment of AD pursuant to an agreement with BioArctic in December 2007. The development and commercialization agreement on the antibody lecanemab back-up was signed in May 2015.
6. About Eisai Co., Ltd.
Eisai’s Corporate Concept is “to give first thought to patients and people in the daily living domain, and to increase the benefits that health care provides.” Under this Concept (also known as human health care (hhc) Concept), we aim to effectively achieve social good in the form of relieving anxiety over health and reducing health disparities. With a global network of R&D facilities, manufacturing sites and marketing subsidiaries, we strive to create and deliver innovative products to target diseases with high unmet medical needs, with a particular focus in our strategic areas of Neurology and Oncology.
In addition, we demonstrate our commitment to the elimination of neglected tropical diseases (NTDs), which is a target (3.3) of the United Nations Sustainable Development Goals (SDGs), with working on various activities together with global partners.
For more information about Eisai, please visit www.eisai.com (for global headquarters: Eisai Co., Ltd.), and connect with us on Twitter @Eisai_SDGs.
7. About Biogen
As pioneers in neuroscience, Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological diseases as well as related therapeutic adjacencies. As one of the world’s first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Sir Kenneth Murray, and Nobel Prize winners Walter Gilbert and Phillip Sharp. Today, Biogen has a leading portfolio of medicines to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, and developed the first and only approved treatment to address a defining pathology of Alzheimer’s disease. Biogen is also commercializing biosimilars and focusing on advancing one of the industry’s most diversified pipelines in neuroscience that will transform the standard of care for patients in several areas of high unmet need.
In 2020, Biogen launched a bold 20-year, $250 million initiative to address the deeply interrelated issues of climate, health, and equity. Healthy Climate, Healthy Lives™ aims to eliminate fossil fuels across the company’s operations, build collaborations with renowned institutions to advance the science to improve human health outcomes, and support underserved communities.
We routinely post information that may be important to investors on our website at www.biogen.com. Follow us on social media - Twitter, LinkedIn, Facebook, YouTube.
Biogen, Eisai drive rally in Alzheimer’s stocks as lecanemab succeeds in late-stage trial
Sep. 28, 2022 6:43 AM ET
Biogen Inc. (BIIB)RHHBY, LLY, SAVA, ESALY, AVXL, ESALF, RHHBF, ANVS, PRTA, ACIU, ABOS, CGTX
By: Dulan Lokuwithana, SA News Editor7 Comments
Biogen (NASDAQ:BIIB) and Japanese drugmaker Eisai (OTCPK:ESALF) (OTCPK:ESALY) announced their experimental Alzheimer's medication lecanemab met the main goal in a large-stage trial, sparking pre-market gains Wednesday in companies with similar drugs in development.
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SKYSONA® (elivaldogene autotemcel)
20-Valent Pneumococcal Conjugate Vaccine
20-Valent Pneumococcal Conjugate Vaccine
bluebird bio Receives FDA Accelerated Approval for SKYSONA® Gene Therapy for Early, Active Cerebral Adrenoleukodystrophy (CALD)
SKYSONA is the first FDA approved therapy shown to slow the progression of neurologic dysfunction in boys with this devastating and fatal neurodegenerative disease
Management team to host conference call Monday, September 19, at 8:00 a.m. ET
SOMERVILLE, Mass.--(BUSINESS WIRE)--Sep. 16, 2022-- bluebird bio, Inc. (Nasdaq: BLUE) today announced the U.S. Food and Drug Administration (FDA) has granted Accelerated Approval of SKYSONA® (elivaldogene autotemcel), also known as eli-cel, to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD). The Company also confirmed that the previous clinical hold on the eli-cel clinical development program has been lifted.
CALD is a rare, progressive, neurodegenerative disease that primarily affects young boys and causes irreversible, devastating neurologic decline, including major functional disabilities such as loss of communication, cortical blindness, requirement for tube feeding, total incontinence, wheelchair dependence, or complete loss of voluntary movement. Nearly half of patients who do not receive treatment die within five years of symptom onset. Prior to the approval of SKYSONA treatment, effective options were limited to allogeneic hematopoietic stem cell transplant (allo-HSCT), which is associated with the risk of serious potential complications including death, that can increase dramatically in patients without a human leukocyte antigen (HLA) matched donor.
“Children with CALD and their families have been at the heart of bluebird’s mission since the company was founded more than a decade ago,” said Andrew Obenshain, chief executive officer, bluebird bio. “For the ALD community, this long-awaited approval represents significant hope and offers families a new option where, for many, there had been none. We are grateful to every individual who was involved in the development of SKYSONA and are committed to working with providers and payers to make this important treatment option available to patients and their families.”
“The agony of watching your child slip away is something no parent should have to bear,” said Elisa Seeger, co-founder, ALD Alliance. “We have made significant strides in providing children diagnosed with CALD the best chance at life with early identification of ALD through expanded newborn screening. Yet with limited treatment options, early diagnosis is still cause for despair instead of hope for many families. Today, parents whose boys receive a CALD diagnosis can have renewed hope for the future.”
“CALD strikes young boys in the prime of their development, robbing them of core neurologic functions necessary for survival. That is an unimaginable reality for any parent, and as a clinician, it is heartbreaking to have limited treatment options for these children and their families,” said David A. Williams, MD, Chief, Division of Hematology/Oncology, Boston Children’s Hospitali. “After supporting the clinical development of SKYSONA for nearly a decade as a study site, Boston Children’s Hospital is extremely pleased that an FDA-approved therapy is now available for children who urgently need new therapies.”
“As one of the largest and most experienced pediatric gene therapy and stem cell transplant programs in the world, the University of Minnesota is committed to expanding access and advancing care and research for patients with rare diseases like ALD,” said Paul Orchard, MD, a pediatric blood and marrow transplant physician at the University of Minnesota Medical School and M Health Fairview Masonic Children’s Hospital. “It’s crucial for these patients and families to have another therapeutic option for cerebral ALD beyond blood stem cell transplantation utilizing cells from another donor, and we’ve seen firsthand the impact that gene therapy has on our patients. We are encouraged by progress we’re making to treat these rare and devastating diseases.”
As a condition of the SKYSONA Accelerated Approval, bluebird has agreed to provide confirmatory long-term clinical data to the FDA. bluebird anticipates that this will include data from the ongoing long-term follow-up study (LTF-304), which follows patients treated in clinical trials for 15 years, and from commercially treated patients.
bluebird anticipates that commercial product will be available by the end of 2022 through a limited number of Qualified Treatment Centers (QTCs) in the United States, including Boston Children’s Hospital and Children’s Hospital of Philadelphia.
bluebird has set the wholesale acquisition cost of SKYSONA in the U.S. at $3.0M. Additional information is available through bluebird’s patient support program, my bluebird support, which will provide personalized support for patients and their families related to all aspects of the gene therapy journey. Caregivers of patients with CALD can visit mybluebirdsupport.com or call 833-888-NEST (833-888-6378) Monday-Friday between 8 a.m. and 8 p.m. ET to ask questions and enroll.
The SKYSONA Biologics License Application (BLA) was reviewed by the U.S. FDA under Priority Review, and bluebird received a rare pediatric priority review voucher upon approval. SKYSONA was previously granted Orphan Drug designation, Rare Pediatric Disease designation, and Breakthrough Therapy designation.
SKYSONA Clinical Data
The approval of SKYSONA is based on data from bluebird bio’s Phase 2/3 study ALD-102 (Starbeam) (N=32) and Phase 3 ALD-104 (N=35) study.
Both open-label, single-arm studies enrolled patients with early, active CALD who had elevated very long chain fatty acid (VLCFA) values, a Loes score between 0.5 and 9 (inclusive), and gadolinium enhancement on magnetic resonance imaging (MRI) of demyelinating lesions. Additionally, patients were required to have a neurologic function score (NFS) of ≤ 1, indicating limited changes in neurologic function. The efficacy of SKYSONA was compared to a natural history population.
Per protocol, patients treated with SKYSONA were assessed using the NFS and monitored for the emergence of six Major Functional Disabilities (MFDs) associated with CALD progression including loss of communication, cortical blindness, requirement for tube feeding, total incontinence, wheelchair dependence, or complete loss of voluntary movement.
The Accelerated Approval of SKYSONA is based on 24-month MFD-free survival. A post-hoc enrichment analysis in symptomatic patients assessed MFD-free survival from onset of symptoms (NFS ≥ 1) in SKYSONA treated (N=11) and untreated patients (N=7). SKYSONA treated patients had an estimated 72 percent likelihood of MFD-free survival at 24 months from time of first NFS ≥ 1, compared to untreated patients who had only an estimated 43 percent likelihood of MFD-free survival.
The most common non-laboratory adverse reactions (incidence ≥ 20%) are mucositis, nausea, vomiting, febrile neutropenia, alopecia, decreased appetite, abdominal pain, constipation, pyrexia, diarrhea, headache, and rash. The most common Grade 3 or 4 laboratory abnormalities (≥40%) include leukopenia, lymphopenia, thrombocytopenia, neutropenia, anemia, and hypokalemia. Please see SKYSONA Important Safety Information below, including a Boxed Warning for Hematologic Malignancy.
Enrollment is complete and all patients have been treated in both studies; follow-up in ALD-104 is ongoing. All patients who complete 24 months of follow-up in studies ALD-102 or ALD-104 are encouraged to participate in a long-term follow-up study (LTF-304) to continue monitoring safety and efficacy outcomes in boys treated with SKYSONA through 15 years post-treatment. On September 15, 2022, the FDA lifted the clinical hold that was put in place August 2021, prior to the completion of its review of the SKYSONA Biologics License Application.
Indication
SKYSONA is indicated to slow the progression of neurologic dysfunction in boys 4-17 years of age with early, active cerebral adrenoleukodystrophy (CALD). Early, active cerebral adrenoleukodystrophy refers to asymptomatic or mildly symptomatic (neurologic function score, NFS ≤ 1) boys who have gadolinium enhancement on brain magnetic resonance imaging (MRI) and Loes scores of 0.5-9.
This indication is approved under accelerated approval based on 24-month Major Functional Disability (MFD)- free survival. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
Please see full Prescribing Information for SKYSONA, including BOXED WARNING and Medication Guide.
About SKYSONA® (elivaldogene autotemcel), also known as eli-cel
SKYSONA is a one-time gene therapy custom-designed to treat the underlying cause of cerebral adrenoleukodystrophy (CALD). SKYSONA uses ex-vivo transduction with the Lenti-D lentiviral vector (LVV) to add functional copies of the ABCD1 gene into a patient’s own hematopoietic stem cells (HSCs). The addition of the functional ABCD1 gene allows patients to produce the ALD protein (ALDP), which can then participate in the local degradation of very long-chain fatty acids (VLCFAs). This degradation of VLCFAs is believed to slow or possibly prevent further inflammation and demyelination.
About bluebird bio, Inc.
bluebird bio is pursuing curative gene therapies to give patients and their families more bluebird days. With a dedicated focus on severe genetic diseases, bluebird has industry-leading clinical and research programs for sickle cell disease, beta-thalassemia and cerebral adrenoleukodystrophy and is advancing research to apply new technologies to these and other diseases. We custom design each of our therapies to address the underlying cause of disease and have developed in-depth and effective analytical methods to understand the safety of our lentiviral vector technologies and drive the field of gene therapy forward.
Founded in 2010, bluebird has the largest and deepest ex-vivo gene therapy data set in the world—setting the standard for industry. Today, bluebird continues to forge new paths, combining our real-world experience with a deep commitment to patient communities and a people-centric culture that attracts and grows a diverse flock of dedicated birds.
For more information, visit bluebirdbio.com or follow us on social media at @bluebirdbio, LinkedIn, Instagram and YouTube.
SKYSONA and bluebird bio are trademarks of bluebird bio, Inc.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220916005595/en/
Source: bluebird bio, Inc.
Bluebird bio jumps 17% on FDA approval of new gene therapy
Sep. 19, 2022 5:52 AM ET
By: Dulan Lokuwithana, SA News Editor4 Comments
- The commercial-stage biotech bluebird bio, Inc. (NASDAQ:BLUE) added ~17% pre-market Monday after the company announced the FDA approval of its gene therapy Skysona to address the impact of a rare neurodegenerative disease called cerebral adrenoleukodystrophy (CALD).
- https://seekingalpha.com/news/3883642-blue-stock-jumps-on-fda-approval-of-new-gene-therapy
- https://seekingalpha.com/symbol/BLUE
- https://www.bluebirdbio.com/
- https://www.bluebirdbio.com/-/media/bluebirdbio/Corporate%20COM/Files/Skysona/SKYSONA_Medication_Guide.pdf
20-Valent Pneumococcal Conjugate Vaccine
20-Valent Pneumococcal Conjugate Vaccine
20-Valent Pneumococcal Conjugate Vaccine
Pfizer Announces Positive Top-Line Results from Phase 3 Study in 20-Valent Pneumococcal Conjugate Vaccine in Infants in the European Union
Monday, September 19, 2022 - 06:45am
- Positive pivotal top-line data demonstrates 20-valent pneumococcal conjugate vaccine candidate (20vPnC), if approved, can likely help protect against all 20 vaccine serotypes in three-dose series and potentially offer the broadest serotype coverage of any available pneumococcal conjugate vaccine (PCV)
- The safety profile of 20vPnC was favorable and similar to Prevenar 13® (or Prevnar 13® in the U.S.) in this schedule, and concomitant use with common pediatric vaccines was supported and well tolerated
- 20vPnC also showed robust functional antibody responses to the vaccine serotypes post Dose 2 and 3 similar to Prevenar® and Prevenar 13®
- The company intends to file for regulatory approval in the EU in the next few months
NEW YORK--(BUSINESS WIRE)-- Pfizer today announced positive top-line results from its pivotal E.U. Phase 3 study in infants (NCT04546425) evaluating its 20-valent pneumococcal conjugate vaccine candidate (20vPnC) for the prevention of invasive pneumococcal disease (IPD), pneumonia, and acute otitis media caused by the 20 Streptococcus pneumoniae (pneumococcus) serotypes contained in the vaccine for the pediatric population.
The study had three coprimary outcomes, associated with immunogenicity responses one month after the second and third doses of a three-dose vaccination series given at approximately 2, 4, and 11-12 months of age of 20vPnC compared to Prevenar 13®. For the non-inferiority (NI) co-primary objective of immunoglobulin G (IgG) geometric mean concentrations (GMCs) one month after Dose 3 at 11-12 months of age, 19 of the 20 serotypes met the NI criteria with only one serotype narrowly missing. For the NI co-primary objective of IgG GMCs one month after Dose 2, 16 of the 20 serotypes met NI. Finally, for the third NI co-primary objective of the percentage of participants with predefined serotype-specific IgG concentrations one month after Dose 2, nine of the 20 serotypes met the NI criteria. All 20 serotypes showed increased booster responses from post dose 2 to post dose 3 which are indicative of immunological memory and long-term protection. All 20 vaccine serotypes also showed strong functional antibody responses as measured by the opsonophagocytic assay (OPA) post-dose 2 and post dose 3 similar to Prevenar® and Prevenar 13®. The totality of data is therefore directionally consistent with prior clinical experience with Prevenar® and Prevenar 13® after 2 and 3 infant doses, both of which have demonstrated effectiveness in a three-dose schedule against the serotypes contained in the vaccine in post-licensure studies.
In summary, the totality of these positive 20vPnC data, combined with the experience with Prevenar 13® in this schedule, demonstrates that the 20vPnC candidate, if approved, is likely to help protect against all 20 vaccine serotypes in a three-dose vaccine series.
“Today marks another important milestone in the 20vPnC pediatric program, with these data demonstrating 20vPnC’s potential to provide the most comprehensive pneumococcal serotype coverage of any available pneumococcal conjugate vaccine,” said Annaliesa Anderson, Ph.D., Senior Vice President and Chief Scientific Officer, Vaccine Research and Development, Pfizer. “Based on the totality of immunogenicity and safety data, we feel confident that 20vPnC is likely to be protective against all vaccine serotypes in a three-dose series. We are thankful to everyone who worked on or participated in this study, and we look forward to hopefully being able to provide infants with more robust and meaningful protection against more pneumococcal disease-causing serotypes in the near future.”
The safety profile of 20vPnC was similar to Prevenar 13® in this schedule, and concomitant use with common pediatric vaccines were supported.
Pfizer plans to file these data by the end of this year with the European Medicines Agency (EMA). These positive data mark the conclusion of pivotal topline readouts for the 20vPnC pediatric program. Pfizer will also seek to present and publish outcomes from this clinical trial at a future date once safety and immunogenicity data have been fully analyzed.
About the 20vPnC Phase 3 Pediatric Program
In 2020, Pfizer initiated the Phase 3 clinical trial program for the pediatric indication for 20vPnC. Four core Phase 3 pediatric studies will help expand the data on the safety, tolerability, and immunogenicity of 20vPnC. These studies collectively enrolled approximately 4,700 infants and 800 toddlers and children of all ages including:
- A Phase 3 study describing the tolerability and safety and comparing immunogenicity of 20vPnC to Prevenar 13® in infants vaccinated at 2, 4, 6, and 12-15 months of age in the U.S. (NCT04382326)
- A Phase 3 study describing the tolerability and safety of 20vPnC, with Prevenar 13® serving as the control in infants vaccinated at 2, 4, 6, and 12-15 months of age in multiple countries. (NCT04379713)
- A Phase 3 study describing the tolerability and safety and comparing immunogenicity of 20vPnC to Prevenar 13® in infant vaccination at approximately 2, 4, and 11-12 months of age in Europe and Australia (NCT04546425)
- A Phase 3 study in children 15 months through <18 years of age receiving a single dose of 20vPnC in the U.S. (NCT04642079).
About 20vPnC
Pfizer’s 20vPnC pediatric vaccine candidate includes 13 serotypes already included in Prevenar 13® – 1, 3, 4, 5, 6A, 6B, 7F, 9V, 14, 18C, 19A, 19F, and 23F. The seven new serotypes included in 20vPnC are global causes of invasive pneumococcal disease (IPD), and are associated with high case-fatality rates, antibiotic resistance, and/or meningitis. Together, the 20 serotypes included in 20vPnC are responsible for the majority of currently circulating pneumococcal disease in the EU and globally.
On Feb 14, 2022, the European Commission Decision was adopted for APEXXNAR ® (20vPnC) for the prevention of invasive disease and pneumonia caused by the 20 Streptococcus pneumoniae (pneumococcus) serotypes in the vaccine in adults ages 18 years and older.
INDICATIONS FOR PREVENAR 13®
- Active immunisation for the prevention of invasive disease, pneumonia and acute otitis media caused by Streptococcus pneumoniae in infants, children and adolescents from 6 weeks to 17 years of age.
- Active immunisation for the prevention of invasive disease and pneumonia caused by Streptococcus pneumoniae in adults ≥18 years of age and the elderly.
- The use of Prevenar 13 should be determined on the basis of official recommendations taking into consideration the risk of invasive disease and pneumonia in different age groups, underlying comorbidities as well as the variability of serotype epidemiology in different geographical areas.
INDICATIONS FOR APEXXNAR®
- Active immunisation for the prevention of invasive disease and pneumonia caused by Streptococcus pneumoniae in individuals 18 years of age and older. APEXXNAR ® should be used in accordance with official recommendations.
About Pfizer: Breakthroughs That Change Patients’ Lives
At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 170 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at www.Pfizer.com. In addition, to learn more, please visit us on www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220919005238/en/
Source: Pfizer Inc.
Pfizer posts topline
data for pneumococcal vaccine from late-stage EU trial for children
Sep. 19, 2022 7:24 AM ET
By: Dulan Lokuwithana, SA News Editor
Pfizer (NYSE:PFE) announced Monday preliminary results from a pivotal EU Phase 3 study designed to evaluate its 20-valent pneumococcal conjugate vaccine candidate 20vPnC for the prevention of invasive pneumococcal disease (IPD), pneumonia, and acute otitis media (inflammation of the middle ear).
https://seekingalpha.com/symbol/PFE
https://prevnar20.pfizerpro.com/
biotecHOPE™ 2022
KT-333
Rigosertib In Combination With Opdivo (Nivolumab)
Rigosertib In Combination With Opdivo (Nivolumab)
Kymera Therapeutics Receives FDA Orphan Drug Designation for KT-333, a First-in-Class, Investigational STAT3 Degrader for the Treatment of Cutaneous T-Cell Lymphoma
Sep 15, 2022
Cutaneous T-Cell Lymphoma is the second orphan drug designation for KT-333
WATERTOWN, Mass., Sept. 15, 2022 (GLOBE NEWSWIRE) -- Kymera Therapeutics, Inc. (NASDAQ: KYMR), a clinical-stage biopharmaceutical company advancing targeted protein degradation to deliver novel small molecule protein degrader medicines, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to KT-333 for the treatment of Cutaneous T-cell Lymphoma (CTCL).
KT-333 is a first-in-class degrader of the transcriptional regulator STAT3. Deregulation of STAT3 signaling has been implicated in the pathogenesis of a variety of cancers, including CTCL. There are currently no approved therapies for CTCL that target this pathway. KT-333 received orphan drug designation for the treatment of Peripheral T-cell Lymphoma (PTCL) earlier this year.
“This second orphan drug designation reinforces the potential of KT-333 to impact the lives of a broad range of patients with hematological and solid tumors by targeting STAT3, a protein that has been considered undruggable,” said Nello Mainolfi, Co-Founder, President and CEO of Kymera Therapeutics. “We have a significant opportunity to deliver an important new medicine with this first-in-class heterobifunctional degrader, and we look forward to working with the lymphoma community to rapidly advance KT-333 in CTCL and exploring its potential in other cancers.”
The safety, tolerability and PK/PD of escalating doses of KT-333 are currently being evaluated in an ongoing Phase 1 clinical trial in adult patients with relapsed/refractory liquid and solid tumors, including aggressive lymphomas.
The FDA's Orphan Drug Designation program provides orphan status to drugs defined as those intended for the treatment, diagnosis or prevention of rare diseases that affect fewer than 200,000 people in the United States. Orphan drug designation qualifies the sponsor of the drug for certain development incentives, including tax credits for qualified clinical testing, prescription drug user fee exemptions and seven-year marketing exclusivity upon FDA approval.
About Cutaneous T-Cell Lymphoma
Cutaneous T-cell lymphoma (CTCL) is a general term for non-Hodgkin’s T-cell lymphomas that are primarily characterized by an abnormal accumulation of T-cells in the skin and which account for approximately 25 percent of T-cell lymphomas in the U.S. CTCL can involve the blood, lymph nodes and other internal organs. CTCL is a rare and typically slow-growing cancer with symptoms such as dry skin, potentially severe itching, rashes and enlarged lymph nodes. Since symptoms and skin biopsy findings are similar to other skin conditions, early-stage diagnosis can be difficult.
About Kymera Therapeutics
Kymera Therapeutics (Nasdaq: KYMR) is a biopharmaceutical company pioneering the field of targeted protein degradation, a transformative approach to address disease targets and pathways inaccessible with conventional therapeutics. Kymera’s Pegasus platform is a powerful drug discovery engine, advancing novel small molecule therapies that harness the body’s innate protein recycling machinery to degrade dysregulated, disease-causing proteins. With a focus on undrugged nodes in validated pathways, Kymera is advancing a pipeline of novel therapeutics designed to address the most intractable pathways and provide new treatments for patients. Kymera’s initial programs target IRAK4, IRAKIMiD, and STAT3 within the IL-1R/TLR or JAK/STAT pathways, providing the opportunity to treat patients with a broad range of immune-inflammatory diseases, hematologic malignancies, and solid tumors. For more information, visit www.kymeratx.com.
Founded in 2016, Kymera is headquartered in Watertown, Mass. Kymera has been named a “Fierce 15” biotechnology company by Fierce Biotech and has been recognized by the Boston Business Journal as one of Boston’s “Best Places to Work.” For more information about our people, science, and pipeline, please visit www.kymeratx.com or follow us on Twitter or LinkedIn.
Kymera KT-333 gets FDA orphan drug status for blood cancer subtype
Sep. 15, 2022 8:25 AM ET
Kymera Therapeutics, Inc. (KYMR)
By: Ravikash, SA News Editor
- The U.S. Food and Drug Administration (FDA) granted orphan drug designation to Kymera Therapeutics' (NASDAQ:KYMR) KT-333 to treat Cutaneous T-cell Lymphoma (CTCL).
- https://seekingalpha.com/news/3882983-kymera-kt-333-gets-fda-orphan-drug-status-for-blood-cancer-subtype
- https://seekingalpha.com/symbol/KYMR
- https://www.kymeratx.com/
- https://clinicaltrials.gov/ct2/show/NCT05225584
- https://www.kymeratx.com/wp-content/uploads/2021/12/1865.pdf
Rigosertib In Combination With Opdivo (Nivolumab)
Rigosertib In Combination With Opdivo (Nivolumab)
Rigosertib In Combination With Opdivo (Nivolumab)
Sep 12, 2022
Onconova Therapeutics Announces Updated Data From Investigator-Sponsored Phase 1/2a Trial Evaluating Rigosertib In Combination With Nivolumab In Advanced KRAS-Mutated Non-Small Cell Lung Cancer At The ESMO Congress 2022
- Data show an early signal of efficacy in an extensively pre-treated population with 1 complete response and 2 partial responses achieved in 14 evaluable patients
- Responses achieved in patients with 3 distinct and different KRAS mutations, confirming the MOA of rigosertib being KRAS+ agnostic
- 4 of 14 (29%) evaluable patients demonstrated disease control
- The combination of rigosertib and nivolumab has been well tolerated with no synergistic toxicities observed to-date
NEWTOWN, Pa., Sept. 12, 2022 (GLOBE NEWSWIRE) -- Onconova Therapeutics, Inc. (NASDAQ: ONTX), (“Onconova”), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, today announced updated data from an investigator-sponsored Phase 1/2a trial of oral rigosertib plus the anti-PD-1 immune checkpoint inhibitor (ICI) nivolumab in advanced KRAS-mutated (KRAS+) non-small cell lung cancer (NSCLC). The data, which are featured in a poster at the European Society for Medical Oncology (ESMO) Congress 2022, show an early and encouraging signal of efficacy in the trial’s extensively pre-treated population. The studied doublet has been well tolerated to-date.
"The emerging data being presented at ESMO are encouraging, as treatment with rigosertib plus nivolumab led to both complete and partial responses in patients with KRAS-mutated lung cancers who failed prior ICI therapy,” said Dr Rajwanth Veluswamy, the principal investigator of the study. “Objective responses showcased rigosertib’s KRAS mutation-agnostic mechanism of action, as each responding patient had a tumor with a different underlying variant. This differentiates rigosertib from agents targeting a single KRAS mutation variant, and positions it to potentially address the unmet needs of a much broader patient population. In addition, the ESMO data demonstrated activity in multiple patients with both low PD-L1 expression at diagnosis and STK11/LKB1 co-mutations, both poor predictive features for current lung cancer treatments."
Key data from the presentation include:
Demographics:
- All enrolled patients failed at least one line of prior therapy with a PD-1 checkpoint inhibitor (includes evaluable and non-evaluable patients)
- 80% of enrolled patients failed at least two lines of prior therapy
Response results (as of August 15th, 2022-data cutoff date):
- 3 of 14 evaluable patients achieved an objective response
- 1 patient achieved a complete response (CR) as per RECIST Criteria, with complete resolution of the primary lung tumor as well as sites of metastatic disease.
- 2 patients achieved a partial response (PR)
- Responses were achieved in patients with 3 distinct KRAS mutations (CR: KRAS G12V; PRs: KRAS G12C/STK11 and Q61H/STK11)
- The mean duration of response is 6.75 months
- 4 of 14 evaluable patients achieved disease control (CR, PR, or stable disease)
About the Investigator-sponsored Phase 1/2a Trial
This Phase 1/2a trial is designed to evaluate the combination of rigosertib and nivolumab in advanced KRAS+ metastatic NSCLC patients who have progressed on standard-of-care with anti-PD-1 monotherapy or anti-PD-1 in combination with chemotherapy. It includes a dose-escalating Phase 1 portion followed by a Phase 2a dose-expansion portion. Patients in the trial receive oral rigosertib twice daily on days 1-21, and intravenous nivolumab on days 1 and 15 of 28-day cycles. The primary endpoints of the trial are safety assessments to determine maximum tolerated dose, and overall response rate. Secondary endpoints include progression-free survival and overall survival. For more information on the trial, see ClinicalTrials.gov Identifier: NCT04263090.
About Onconova Therapeutics, Inc.
Onconova Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer. The Company has proprietary targeted anti-cancer agents designed to disrupt specific cellular pathways that are important for cancer cell proliferation.
Onconova’s novel, proprietary multi-kinase inhibitor narazaciclib (formerly ON 123300) is being evaluated in two separate and complementary Phase 1 dose-escalation and expansion studies. These trials are currently underway in the United States and China.
Onconova’s product candidate rigosertib is being studied in an investigator-sponsored study program, including in a dose-escalation and expansion Phase 1/2a investigator-sponsored study with oral rigosertib in combination with nivolumab for patients with KRAS+ non-small cell lung cancer.
For more information, please visit www.onconova.com.
Onconova rigosertib/Opdivo combo shows promise in advanced lung cancer in trial
Sep. 12, 2022 12:19 PM ET
Onconova Therapeutics, Inc. (ONTX), BMY
By: Ravikash, SA News Editor
Onconova Therapeutics (NASDAQ:ONTX) said updated data from a phase 1/2a trial of oral rigosertib and Bristol-Myers Squibb's (NYSE:BMY) Opdivo (nivolumab) showed early signs of efficacy in certain extensively pre-treated patients with non-small cell lung cancer (NSCLC).
https://seekingalpha.com/symbol/ONTX
https://seekingalpha.com/symbol/BMY
https://www.onconova.com/pipeline/
HyBryte™ (research name SGX301)
Rigosertib In Combination With Opdivo (Nivolumab)
HyBryte™ (research name SGX301)
U.S. Food and Drug Administration Awards $2.6 Million Orphan Products Development Grant for Expanded Study of HyBryte™ in the Treatment of Cutaneous T-Cell Lymphoma
Grant supports study of expanded HyBryte™ treatment, including in the Home Use Setting
PRINCETON, N.J., Sept. 6, 2022 /PRNewswire/ -- Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced today that the U.S. Food and Drug Administration (FDA) has awarded an Orphan Products Development grant to support the evaluation of HyBryte™ (synthetic hypericin) for expanded treatment in patients with early-stage CTCL. The grant, totaling $2.6 million over 4 years, was awarded to a prestigious academic institution that was a leading enroller in the recently published positive Phase 3 FLASH (Fluorescent Light Activated Synthetic Hypericin) study in the treatment of early stage cutaneous T-cell lymphoma (CTCL).
"We are pleased the FDA is supporting the HyBryte™ program and giving patients an opportunity to access the therapy in an open-label setting," stated Christopher J. Schaber, President and CEO of Soligenix, Inc. "CTCL is an incredibly difficult to treat orphan disease and remains an area of unmet medical need with a very limited number of safe and effective treatment options. The Phase 3 results provide the basis for our upcoming marketing application and this study will serve to embark on potential home-use of the therapy, augment the safety database as well as provide further real-world evidence into the practical use of HyBryte™ once commercially available."
The clinical study RW-HPN-MF-01, "Assessment of Treatment with Visible Light Activated Synthetic Hypericin Ointment in Mycosis Fungoides Patients" is designed as an open-label, multicenter clinical trial enrolling approximately 50 patients at up to six of the highest enrolling clinical centers that participated in the Phase 3 FLASH study. Patients have the potential to be treated for up to 12 months with twice a week dosing (visible light activation to follow ointment application by 24 ± 6 hours). The primary endpoint for the study will be evaluating the number of treatment successes defined as ≥50% reduction in the cumulative CAILS (Composite Assessment of Index Lesion Severity) score from baseline to end of the treatment. Study initiation is planned for the fourth quarter of 2022.
About HyBryte™
HyBryte™ (research name SGX301) is a novel, first-in-class, photodynamic therapy utilizing safe, visible light for activation. The active ingredient in HyBryte™ is synthetic hypericin, a potent photosensitizer that is topically applied to skin lesions that is taken up by the malignant T-cells, and then activated by visible light 16 to 24 hours later. The use of visible light in the red-yellow spectrum has the advantage of penetrating more deeply into the skin (much more so than ultraviolet light) and therefore potentially treating deeper skin disease and thicker plaques and lesions. This treatment approach avoids the risk of secondary malignancies (including melanoma) inherent with the frequently employed DNA-damaging drugs and other phototherapy that are dependent on ultraviolet exposure. Combined with photoactivation, hypericin has demonstrated significant anti-proliferative effects on activated normal human lymphoid cells and inhibited growth of malignant T-cells isolated from CTCL patients. In a published Phase 2 clinical study in CTCL, patients experienced a statistically significant (p=0.04) improvement with topical hypericin treatment whereas the placebo was ineffective. HyBryte™ has received orphan drug and fast track designations from the FDA, as well as orphan designation from the European Medicines Agency (EMA).
The recently published Phase 3 FLASH trial enrolled a total of 169 patients (166 evaluable) with Stage IA, IB or IIA CTCL. The trial consisted of three treatment cycles. Treatments were administered twice weekly for the first 6 weeks and treatment response was determined at the end of the 8th week of each cycle. In the first double-blind treatment cycle, 116 patients received HyBryte™ treatment (0.25% synthetic hypericin) and 50 received placebo treatment of their index lesions. A total of 16% of the patients receiving HyBryte™ achieved at least a 50% reduction in their lesions (graded using a standard measurement of dermatologic lesions, the CAILS score) compared to only 4% of patients in the placebo group at 8 weeks (p=0.04) during the first treatment cycle (primary endpoint). HyBryte™ treatment in the first cycle was safe and well tolerated.
In the second open-label treatment cycle (Cycle 2), all patients received HyBryte™ treatment of their index lesions. Evaluation of 155 patients in this cycle (110 receiving 12 weeks of HyBryte™ treatment and 45 receiving 6 weeks of placebo treatment followed by 6 weeks of HyBryte™ treatment), demonstrated that the response rate among the 12-week treatment group was 40% (p<0.0001 vs the placebo treatment rate in Cycle 1). Comparison of the 12-week and 6-week treatment groups also revealed a statistically significant improvement (p<0.0001) between the two groups, indicating that continued treatment results in better outcomes. HyBryte™ continued to be safe and well tolerated. Additional analyses also indicated that HyBryte™ is equally effective in treating both plaque (response 42%, p<0.0001 relative to placebo treatment in Cycle 1) and patch (response 37%, p=0.0009 relative to placebo treatment in Cycle 1) lesions of CTCL, a particularly relevant finding given the historical difficulty in treating plaque lesions in particular.
The third (optional) treatment cycle (Cycle 3) was focused on safety and all patients could elect to receive HyBryte™ treatment of all their lesions. Of note, 66% of patients elected to continue with this optional compassionate use / safety cycle of the study. Of the subset of patients that received HyBryte™ throughout all 3 cycles of treatment, 49% of them demonstrated a positive treatment response (p<0.0001 vs patients receiving placebo in Cycle 1). Moreover, in a subset of patients evaluated in this cycle, it was demonstrated that HyBryte™ is not systemically available, consistent with the general safety of this topical product observed to date. At the end of Cycle 3, HyBryte™ continued to be well tolerated despite extended and increased use of the product to treat multiple lesions.
Overall safety of HyBryte™ is a critical attribute of this treatment and was monitored throughout the three treatment cycles (Cycles 1, 2 and 3) and the 6-month follow-up period. HyBryte's™ mechanism of action is not associated with DNA damage, making it a safer alternative than currently available therapies, all of which are associated with significant and sometimes fatal, side effects. Predominantly these include the risk of melanoma and other malignancies, as well as the risk of significant skin damage and premature skin aging. Currently available treatments are only approved in the context of previous treatment failure with other modalities and there is no approved front-line therapy available. Within this landscape, treatment of CTCL is strongly motivated by the safety risk of each product. HyBryte™ potentially represents the safest available efficacious treatment for CTCL. With no systemic absorption, a compound that is not mutagenic and a light source that is not carcinogenic, there is no evidence to date of any potential safety issues.
The Phase 3 CTCL clinical study was partially funded by the National Cancer Institute via a Phase II SBIR grant (#1R44CA210848-01A1) awarded to Soligenix, Inc.
About Soligenix, Inc.
Soligenix is a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need. Our Specialized BioTherapeutics business segment is developing and moving toward potential commercialization of HyBryte™ (SGX301 or synthetic hypericin) as a novel photodynamic therapy utilizing safe visible light for the treatment of cutaneous T-cell lymphoma (CTCL). With a successful Phase 3 study completed, regulatory approval is being sought and commercialization activities for this product candidate are being advanced initially in the U.S. Development programs in this business segment also include our first-in-class innate defense regulator (IDR) technology, dusquetide (SGX942) for the treatment of inflammatory diseases, including oral mucositis in head and neck cancer, and proprietary formulations of oral beclomethasone 17,21-dipropionate (BDP) for the prevention/treatment of gastrointestinal (GI) disorders characterized by severe inflammation including pediatric Crohn's disease (SGX203).
Our Public Health Solutions business segment includes active development programs for RiVax®, our ricin toxin vaccine candidate, and SGX943, our therapeutic candidate for antibiotic resistant and emerging infectious disease, and our vaccine programs targeting filoviruses (such as Marburg and Ebola) and CiVax™, our vaccine candidate for the prevention of COVID-19 (caused by SARS-CoV-2). The development of our vaccine programs incorporates the use of our proprietary heat stabilization platform technology, known as ThermoVax®. To date, this business segment has been supported with government grant and contract funding from the National Institute of Allergy and Infectious Diseases (NIAID), the Defense Threat Reduction Agency (DTRA) and the Biomedical Advanced Research and Development Authority (BARDA).
For further information regarding Soligenix, Inc., please visit the Company's website at https://www.soligenix.com and follow us on LinkedIn and Twitter at @Soligenix_Inc.
SOURCE Soligenix, Inc.
U.S. FDA awards $2.6M grant for expanded study of Soligenix's HyBryte for skin cancer
Sep. 12, 2022 9:28 AM ET
By: Dania Nadeem, SA News Editor
- Soligenix (NASDAQ:SNGX) is trading 3.2% higher after the U.S. Food and Drug Administration awarded a $2.6M grant to support a study of expanded HyBryte treatment to treat T-cell lymphoma, including at-home usage.
- https://seekingalpha.com/news/3882003-us-fda-awards-26m-grant-for-expanded-study-of-soligenixs-hybryte-for-skin-cancer
- https://seekingalpha.com/symbol/SNGX
- https://www.hybryte.com/
XMT-1660
Sotyktu™ (deucravacitinib)
HyBryte™ (research name SGX301)
Mersana Therapeutics Announces FDA Fast Track Designation Granted to XMT-1660 for the Treatment of Triple-Negative Breast Cancer
September 12, 2022
XMT-1660 currently being studied in a Phase 1 trial enrolling patients with breast, endometrial and ovarian cancers
CAMBRIDGE, Mass., Sept. 12, 2022 (GLOBE NEWSWIRE) -- Mersana Therapeutics, Inc. (NASDAQ: MRSN), a clinical-stage biopharmaceutical company focused on discovering and developing a pipeline of antibody-drug conjugates (ADCs) targeting cancers in areas of high unmet medical need, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to XMT-1660 for the treatment of adult patients with advanced or metastatic triple-negative breast cancer (TNBC). XMT-1660 is a B7-H4-directed Dolasynthen antibody drug conjugate with a precise, target-optimized drug-to-antibody ratio (DAR 6) and Mersana’s clinically validated DolaLock microtubule inhibitor payload with controlled bystander effect.
“While breast cancer remains an area of high unmet need, TNBC is associated with particularly poor outcomes and very limited treatment options,” said Anna Protopapas, President and Chief Executive Officer of Mersana Therapeutics. “XMT-1660 has demonstrated promising anti-tumor effects in preclinical studies, and we are excited to have recently initiated our Phase 1 trial to investigate its safety and clinical activity. This Fast Track designation allows for a potentially accelerated regulatory review of XMT-1660 as we seek to offer a new therapy for patients living with a range of B7-H4 expressing tumors.”
Mersana’s ongoing multicenter Phase 1 trial is investigating the safety, tolerability and anti-tumor activity of XMT-1660 in patients with solid tumors, including in breast, endometrial and ovarian cancers. The initial dose escalation portion of this trial will evaluate the safety and tolerability of XMT-1660. The dose expansion portion of the trial will evaluate the safety, tolerability and efficacy of XMT-1660.
The FDA’s Fast Track program is designed to facilitate the development and expedite the review of drug candidates to treat serious conditions and fill an unmet medical need. A product candidate that is granted Fast Track designation may be eligible for several benefits, including more frequent meetings and communications with the FDA and, if certain criteria are met, the potential for Accelerated Approval, Priority Review or Rolling Review of a Biologics License Application (BLA) by the FDA.
About Mersana Therapeutics
Mersana Therapeutics is a clinical-stage biopharmaceutical company using its differentiated and proprietary ADC platforms to rapidly develop novel ADCs with optimal efficacy, safety and tolerability to meaningfully improve the lives of people fighting cancer. Mersana’s lead product candidate, upifitamab rilsodotin (UpRi), is a Dolaflexin ADC targeting NaPi2b that is being studied in UPLIFT, a single-arm registrational trial in patients with platinum-resistant ovarian cancer; UPGRADE, a Phase 1/2 umbrella trial evaluating UpRi in combination with other ovarian cancer therapies; and UP-NEXT, a Phase 3 clinical trial of UpRi as monotherapy maintenance following treatment with platinum doublets in recurrent platinum-sensitive ovarian cancer. Mersana is also advancing XMT-1660, a Dolasynthen ADC targeting B7-H4, and XMT-2056, an Immunosynthen ADC targeting a novel epitope of human epidermal growth factor receptor 2 (HER2), in addition to other earlier-stage assets. In addition, multiple partners are using Mersana’s platforms to advance their ADC pipelines. Mersana Therapeutics was named among the 2021 Top Places to Work in Massachusetts by The Boston Globe. Mersana routinely posts information that may be useful to investors on the “Investors and Media” section of its website at www.mersana.com.
Mersana gains as FDA grants fast track status for breast cancer drug
Sep. 12, 2022 8:22 AM ET
Mersana Therapeutics, Inc. (MRSN)
By: Dulan Lokuwithana, SA News Editor
- Clinical-stage biotech Mersana Therapeutics, Inc. (NASDAQ:MRSN) added ~8% in the pre-market trading on the announcement that the FDA issued its Fast Track designation for the company’s antibody-drug conjugate XMT-1660 as a treatment for adults with triple-negative breast cancer (TNBC).
- https://seekingalpha.com/news/3881943-mrsn-stock-gains-fda-grants-fast-track-status-breast-cancer-drug
- https://seekingalpha.com/symbol/MRSN
- https://www.mersana.com/xmt-1660/
Sotyktu™ (deucravacitinib)
Sotyktu™ (deucravacitinib)
Sotyktu™ (deucravacitinib)
Bristol Myers Squibb Announces New Sotyktu™ (deucravacitinib) Long-Term Data Showing Clinical Efficacy Maintained for Up to Two Years with Continuous Treatment in Moderate-to-Severe Plaque Psoriasis
09/10/2022CATEGORY:
Results add to the growing body of evidence and reinforce the efficacy profile of Sotyktu, a once-daily, oral, selective, allosteric tyrosine kinase 2 (TYK2) inhibitor for the treatment of moderate-to-severe plaque psoriasis
New analysis to be presented at the 2022 European Academy of Dermatology and Venereology Congress as part of 26 company-sponsored scientific presentations, demonstrating ongoing commitment to dermatology research
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE:BMY) today announced new two-year results from the POETYK PSO long-term extension (LTE) trial demonstrating clinical efficacy was maintained with continuous Sotyktu™ (deucravacitinib) treatment in adult patients with moderate-to-severe plaque psoriasis. This analysis assessed patients from the pivotal POETYK PSO-1 trial who transitioned into the LTE trial. At 112 weeks of Sotyktu treatment, modified non-responder imputation (mNRI) response rates were 82.4% for Psoriasis Area and Severity Index (PASI) 75, 55.2% for PASI 90 and 66.5% for static Physician's Global Assessment (sPGA) 0/1.
These data (Presentation #D3T01.1F) and 25 additional abstracts demonstrating Bristol Myers Squibb’s robust body of research in dermatology are being presented at the European Academy of Dermatology and Venereology (EADV) Congress, taking place September 7-10, 2022.
“The reality we are facing is that dermatologists and individuals with psoriasis alike have identified the need for more effective and tolerable oral therapies, as psoriasis is a chronic, systemic, immune-mediated disease that is associated with serious comorbidities,” said Dr. Mark Lebwohl, MD, Dean of Clinical Therapeutics at the Kimberly and Eric J. Waldman Department of Dermatology at the Icahn School of Medicine, Mount Sinai (New York); Dr. Lebwohl was also a participant in the Publication Steering Committee for this study. “These new long-term results showing durable efficacy through up to two years of continuous treatment further support the use of once-daily Sotyktu for people with moderate-to-severe plaque psoriasis and address the need for more effective oral treatment options.”
Of the 262 Sotyktu patients in the analysis, 171 had achieved PASI 75 at Week 16 of the POETYK PSO-1 trial, and among these patients, efficacy was maintained for up to 112 weeks, including response rates for PASI 75 (Week 16, 100%; Week 52, 90.1%; Week 112, 91.0%), PASI 90 (Week 16, 62.6%; Week 52, 64.9%; Week 112, 63.0%) and sPGA 0/1 (Week 16, 84.2%; Week 52, 73.7%; Week 112, 73.5%).
“These two-year follow-up data demonstrate the durable efficacy offered by Sotyktu and its potential to provide long-term, clinically relevant improvement for individuals with moderate-to-severe plaque psoriasis,” said Jonathan Sadeh, MD, MSc, senior vice president of Immunology and Fibrosis Development, Bristol Myers Squibb. “At Bristol Myers Squibb, we are committed to exploring pathbreaking science to elevate care for people burdened by serious immune-mediated diseases and are focused on continuing our research with Sotyktu and other novel molecules in our deep and differentiated portfolio.”
Bristol Myers Squibb thanks the patients and investigators involved in the POETYK PSO clinical trial program.
About the POETYK PSO Clinical Trial Program
PrOgram to Evaluate the efficacy and safety ofdeucravacitinib, a selective TYK2 inhibitor (POETYK) PSO-1 (NCT03624127) and POETYK PSO-2 (NCT03611751) were global Phase 3 studies designed to evaluate the safety and efficacy of deucravacitinib compared to placebo and Otezla® (apremilast) in patients with moderate-to-severe plaque psoriasis. Both POETYK PSO-1, which enrolled 666 patients, and POETYK PSO-2, which enrolled 1,020 patients, were multi-center, randomized, double-blind trials that evaluated deucravacitinib (6 mg once daily) compared with placebo and Otezla (30 mg twice daily). POETYK PSO-2 included a randomized withdrawal and retreatment period after Week 24.
The co-primary endpoints of both POETYK PSO-1 and POETYK PSO-2 were the percentage of patients who achieved Psoriasis Area and Severity Index (PASI) 75 response and those who achieved static Physician's Global Assessment (sPGA) score of 0 or 1 (clear/almost clear) at Week 16 versus placebo. Key secondary endpoints of the trials included the percentage of patients who achieved PASI 75 and sPGA 0/1 compared to Otezla at Week 16 and other measures evaluating deucravacitinib versus placebo and Otezla.
Following the 52-week POETYK PSO-1 and POETYK PSO-2 trials, patients could enroll in the ongoing POETYK PSO-LTE trial (NCT04036435) and receive open-label deucravacitinib 6 mg once-daily. 1,221 patients enrolled in the long-term extension trial and received at least one dose of deucravacitinib. Efficacy was analyzed utilizing treatment failure rules (TFR) method of imputation, along with sensitivity analyses using modified non-responder imputation and as-observed analysis, which have been used in similar analyses with other agents. In addition to POETYK PSO-1, POETYK PSO-2 and POETYK PSO-LTE, Bristol Myers Squibb has evaluated deucravacitinib in two other Phase 3 studies in psoriasis: POETYK PSO-3 (NCT04167462) and POETYK PSO-4 (NCT03924427).
The Journal of the American Academy of Dermatology recently published primary analysis results from the POETYK PSO-1 study.
About Sotyktu™
Sotyktu™ (deucravacitinib) is a selective, allosteric inhibitor of tyrosine kinase 2 (TYK2). TYK2 is a member of the Janus kinase (JAK) family. Sotyktu binds to the regulatory domain of TYK2, stabilizing an inhibitory interaction between the regulatory and the catalytic domains of the enzyme. This results in allosteric inhibition of receptor-mediated activation of TYK2 and its downstream activation of Signal Transducers and Activators of Transcription (STATs) as shown in cell-based assays. Janus kinases function as pairs of homo- or heterodimers in the JAK-STAT pathways. TYK2 pairs with JAK1 to mediate multiple cytokine pathways and also pairs with JAK2 to transmit signals as shown in cell-based assays. The precise mechanism linking inhibition of TYK2 enzyme to therapeutic effectiveness in the treatment of adults with moderate-to-severe plaque psoriasis is not currently known.
The U.S. Food and Drug Administration approved Sotyktu for the treatment of adults with moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy on September 9, 2022. Sotyktu is also under regulatory review by the European Medicines Agency and other health authorities around the world for the treatment of moderate-to-severe plaque psoriasis and by Japan's Ministry of Health, Labour and Welfare for the treatment of adults with moderate-to-severe plaque psoriasis, pustular psoriasis and erythrodermic psoriasis.
INDICATION
SOTYKTU™ (deucravacitinib) is indicated for the treatment of moderate-to-severe plaque psoriasis in adults who are candidates for systemic therapy or phototherapy.
Limitations of Use:
SOTYKTU is not recommended for use in combination with other potent immunosuppressants.
Please see U.S. Full Prescribing Information, including Medication Guide, for SOTYKTU.
About Bristol Myers Squibb
Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.
Source: Bristol Myers Squibb
Bristol Myers stock rises on FDA approval of psoriasis therapy Sotyktu; presents long-term efficacy data
Sep. 12, 2022 6:22 AM ET
Bristol-Myers Squibb Company (BMY)
By: Ravikash, SA News Editor3 Comments
The U.S. Food and Drug Administration (FDA) on Sept. 9 approved Bristol Myers Squibb's (NYSE:BMY) Sotyktu (deucravacitinib) to treat adults with moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy.
https://seekingalpha.com/symbol/BMY
Ritlecitinib
Sotyktu™ (deucravacitinib)
Sotyktu™ (deucravacitinib)
FDA and EMA Accept Regulatory Submission for Pfizer’s Ritlecitinib for Individuals 12 Years and Older with Alopecia Areata
Friday, September 09, 2022 - 06:45am
– Filings based on clinical data demonstrating significant scalp hair regrowth versus placebo –
NEW YORK--(BUSINESS WIRE)-- Pfizer Inc. (NYSE: PFE) announced today that the U.S. Food and Drug Administration (FDA) accepted for filing the New Drug Application (NDA) for ritlecitinib for adults and adolescents 12 years of age and older with alopecia areata. The FDA is expected to make a decision in the second-quarter 2023. The European Medicines Agency (EMA) has also accepted the Marketing Authorization Application (MAA) for ritlecitinib in the same patient population with a decision anticipated in the fourth-quarter 2023. Ritlecitinib is an investigational oral once daily treatment that is the first in a new class of oral highly selective kinase inhibitors that is a dual inhibitor of the TEC family of tyrosine kinases and of Janus kinase 3 (JAK3).
Alopecia areata is an autoimmune disease that has an underlying immuno-inflammatory pathogenesis and develops when the immune system attacks the body’s hair follicles, causing hair to fall out. This hair loss often occurs on the scalp, but it can also affect eyebrows, eyelashes, facial hair and other areas of the body. Alopecia areata affects approximately 6.8 million people in the U.S. and around 147 million people globally.
“Alopecia areata is an autoimmune disease that can impact people of all ages, genders, and ethnicities, often having an impact on day-to-day life that goes beyond the hair loss itself,” said Michael Corbo, PhD, Chief Development Officer, Inflammation & Immunology, Pfizer Global Product Development. “We believe that ritlecitinib, if approved, will be an important new treatment option, and we are continuing to work closely with regulatory authorities to bring ritlecitinib to adults and adolescents in the U.S. and the EU.”
The submissions were based on previously announced topline results from the pivotal and dose-ranging Phase 2b/3 ALLEGRO (NCT03732807) study and an ongoing Phase 3 ALLEGRO-LT (NCT04006457) open-label, long-term study. Results from the ALLEGRO Phase 2b/3 study were presented at the 2021 European Academy of Dermatology and Venereology (EADV) Congress and the 2022 American Academy of Dermatology Annual Meeting. Initial data from the ongoing ALLEGRO-LT study will be presented at the 2022 EADV Congress.
Pfizer has also completed regulatory submissions for ritlecitinib in the United Kingdom, China and Japan, and expects decisions in 2023.
About the ALLEGRO Program
The randomized, placebo-controlled, double-blind ALLEGRO Phase 2b/3 study investigated ritlecitinib in patients 12 years of age and older with alopecia areata (n=718). Patients included in the study had 50% or more scalp hair loss, as measured by the Severity of Alopecia Tool (SALT), including patients with alopecia totalis (complete scalp hair loss) and alopecia universalis (complete scalp, face, and body hair loss), and were experiencing a current episode of alopecia areata that had lasted between six months and ten years. Patients were randomized to receive once daily ritlecitinib 30 mg or 50 mg (with or without one month of initial treatment with once daily ritlecitinib 200 mg), ritlecitinib 10 mg or placebo.
In this study, statistically significantly higher proportions of patients treated with ritlecitinib 30 mg and 50 mg (with or without the loading dose) had 80% or more scalp hair coverage (SALT<=20) after six months of treatment versus placebo. The overall safety data demonstrated that ritlecitinib was well tolerated both in adult and adolescent patients. Adverse events (AEs) and serious AEs (SAEs) through Week 48 were experienced by 82% and 2% of patients, respectively, with similar rates across active treatment groups. The most common AEs were headache, nasopharyngitis, and upper respiratory tract infections. Cases of herpes zoster (8), pulmonary embolism (1), and breast cancer (2) were reported; no cardiac events, deaths or opportunistic infections were reported through Week 48.
More information about the ALLEGRO 2b/3 trial can be found at https://www.clinicaltrials.gov.
ALLEGRO-LT is an ongoing Phase 3 open-label, long-term study to investigate the safety and efficacy of ritlecitinib in adults with alopecia areata with 25% or greater hair loss and adolescents from 12 years of age with alopecia areata with 50% or greater hair loss.
About Pfizer: Breakthroughs That Change Patients’ Lives
At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 170 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at www.Pfizer.com. In addition, to learn more, please visit us on www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220908006121/en/
Source: Pfizer Inc.
PFE Dividend History
https://www.nasdaq.com/market-activity/stocks/pfe/dividend-history
https://seekingalpha.com/symbol/PFE
UNI-494
Group B Streptococcus (GBS)
Group B Streptococcus (GBS)
Unicycive Reports Key Findings of UNI-494 Efficacy in Preclinical Animal Model of Acute Kidney Injury (AKI)
September 07, 2022 7:00am EDT Download as PDF
Established animal model of AKI showed a statistically significant reduction of a key biomarker of kidney injury when treated with UNI-494
AKI remains a serious unmet medical need with no FDA approved drugs for its treatment
On track to file regulatory submission by year end 2022 to initiate first-in-humans Phase 1 study with UNI-494
LOS ALTOS, Calif., Sept. 07, 2022 (GLOBE NEWSWIRE) -- Unicycive Therapeutics, Inc. (Nasdaq: UNCY), a clinical stage biotechnology company developing therapies for patients with kidney disease, today announced key findings of UNI-494 efficacy from a preclinical study in rodent model of Acute Kidney Injury.
UNI-494 is a mitochondrial ATP sensitive potassium (mitoKATP) channel activator that is in development for the treatment of AKI. The Company evaluated the effect of UNI-494 on ischemia-reperfusion induced acute kidney injury (IR-AKI) in rats. Animals (n=10/group) were divided into 4 groups: sham, vehicle control, low dose of UNI-494 and high dose of UNI-494. Kidney injury was induced by 45-minute ischemia of bilateral kidneys. UNI-494 was administered orally 1-hour before the induction of ischemia and kidney function was monitored by measuring urinary creatinine, total urinary albumin, and β-2 microglobulin (β-2 MG). Treatment of animals with the higher dose of UNI-494 resulted in a statistically significant reduction of β-2 MG levels in urine.
“We are excited to report this key finding from the IR-AKI model showing treatment with UNI-494 significantly reduced this key biomarker of kidney injury that is a well-accepted biomarker of proximal renal tubule damage. We look forward to presenting these data, along with other data from the study, at an upcoming scientific meeting,” said Shalabh Gupta, M.D., Chief Executive Officer of Unicycive. “We remain on track to file a regulatory submission by the end of 2022 that will allow us to initiate our Phase 1 study with UNI-494.”
“These are very encouraging results for UNI-494. The rat IR-AKI model is a widely published model of AKI and β-2 MG is an established clinical biomarker of proximal renal tubule damage in AKI. The ability of UNI-494 to significantly reduce β-2 MG in these animals provides compelling support for the clinical development of this new drug for AKI," said Ravi Mehta, M.D., Prof. Emeritus of Medicine at University of California San Diego School of Medicine, and a world-renowned expert in AKI research.
About UNI-494
UNI-494 is a novel proprietary drug that selectively binds to the SUR2B subunit of the mitochondrial KATP channel and activates it to restore mitochondrial function and reduce oxidative stress. UNI-494 is cleaved by esterase enzymes to form nicorandil which is the active metabolite. Nicorandil has extensive safety and efficacy data from multiple clinical trials including a 5,000-patient randomized controlled trial (IONA Study) and there is a consensus in the literature that the activation of mitoKATP channel is the biological basis for the observed cardio-protection and reno-protection in multiple clinical trials.
Unicycive has completed pharmacokinetic, safety pharmacology, genetic toxicity and ADME studies on UNI-494. Repeat dose toxicity studies in two species are on track to be completed in the third quarter. The Company is on track to file a regulatory submission in the second half of 2022 to initiate the Phase I healthy volunteer study.
While Unicycive’s initial focus is on acute kidney injury (AKI), UNI-494’s novel mechanism of action may also hold promise for indications in which mitochondrial dysfunction is implicated such as chronic kidney disease, liver disease (alcoholic hepatitis, hepatic encephalopathy) and ophthalmic disease (dry AMD, macular degeneration, etc).
UNI-494 is protected by issued patent(s) in the U.S. and Europe and a wide range of patent applications worldwide.
About Unicycive Therapeutics
Unicycive Therapeutics is a biotechnology company developing novel treatments for kidney diseases. Unicycive’s lead drug, Renazorb, is a novel phosphate binding agent being developed for the treatment of hyperphosphatemia. UNI-494 is a patent-protected new chemical entity in late preclinical development for the treatment of acute kidney injury. For more information, please visit www.unicycive.com.
SOURCE: Unicycive Therapeutics, Inc.
Unicycive stock soars 25% as kidney drug UNI-494 shows promise in preclinical study in rats
Sep. 07, 2022 8:01 AM ET
Unicycive Therapeutics, Inc. (UNCY)
By: Ravikash, SA News Editor
- Unicycive Therapeutics (NASDAQ:UNCY) said treatment with UNI-494 showed a statistically significant reduction of a key biomarker of kidney injury in a preclinical study of an animal model of acute kidney injury (AKI).
- https://seekingalpha.com/news/3880758-unicycive-stock-soars-25-as-kidney-drug-uni-494-shows-promise-in-preclinical-study-in-rats
- https://seekingalpha.com/symbol/UNCY
- https://unicycive.com/focus/uni-494/
- https://unicycive.com/
Group B Streptococcus (GBS)
Group B Streptococcus (GBS)
Group B Streptococcus (GBS)
FDA Grants Breakthrough Therapy Designation to Pfizer’s Group B Streptococcus Vaccine Candidate to Help Prevent Infection in Infants Via Immunization of Pregnant Women
Wednesday, September 07, 2022 - 06:45am
NEW YORK--(BUSINESS WIRE)-- Pfizer Inc. (NYSE:PFE) today announced that its investigational Group B Streptococcus (GBS) vaccine candidate, GBS6 or PF-06760805, received Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA) for the prevention of invasive GBS disease due to the vaccine serotypes in newborns and young infants by active immunization of their mothers during pregnancy.
The FDA decision is informed by the interim analysis of a placebo-controlled Phase 2 study (NCT03765073), evaluating the safety and immunogenicity of GBS6 in healthy pregnant women aged 18 to 40 years, who were vaccinated during the second or early third trimester of pregnancy. The study remains ongoing, and Pfizer will publish outcomes from this clinical trial when it is completed.
“GBS infections can have a devastating effect on newborns and their families. While prenatal screening and antibiotics during childbirth help provide protection against GBS in developed countries, this approach is not fully protective in the first week of life; presents multiple challenges in low- and middle-income countries; and has not been shown effective in preventing disease globally in infants beyond the first week of life and through the vulnerable first three months of life,” said Annaliesa Anderson, Ph.D., Senior Vice President and Head of Vaccine Research & Development, Pfizer. “If approved for pregnant women, GBS6 could help protect newborns from the serious illnesses caused by this disease like meningitis, pneumonia, and sepsis – fulfilling a critical global public health need. We are encouraged by today’s decision and look forward to discussing GBS6 with the FDA and other regulatory agencies to potentially reduce neonatal deaths and positively impact the existing global disease burden of GBS.”
The FDA’s Breakthrough Therapy Designation is designed to expedite the development and review of drugs and vaccines that are intended to treat or prevent serious conditions, and preliminary clinical evidence indicates that the drug or vaccine may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).1 This decision follows the FDA’s March 2017 decision to grant Fast Track status to GBS6. Fast Track status is a process designed to facilitate the development and expedite the review of new drugs and vaccines intended to treat or prevent serious conditions and address an unmet medical need.2
About GBS6
Hexavalent anti capsular polysaccharide (CPS) / cross reactive material 197 glycoconjugate (GBS6) is an investigational maternal vaccine being developed to help prevent invasive Group B Streptococcus (GBS) in newborns. GBS6 is designed to offer protection against the six most prominent GBS serotypes, which account for 98% of disease worldwide.3 It is being evaluated in an ongoing Phase 2, placebo-controlled study in pregnant women and their infants in South Africa, U.K., and the U.S. and is assessing the safety and immunogenicity of a single dose administered by intramuscular injection during the second or early third trimester of pregnancy to prevent disease in infants. Pfizer is pursuing a clinical development strategy in high-, middle- and low-income countries with the intent to make a successfully developed vaccine available globally as quickly as possible.
In April 2022, GBS6 was granted PRIME designation by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP). This designation provides enhanced support for the development of medicines that target an unmet medical need.4
In 2016, Pfizer received a grant from the Bill & Melinda Gates Foundation, which supported the ongoing Phase 2 clinical trial of GBS6 as well as a parallel non-interventional natural history study.
About Group B Streptococcus (GBS)
Group B Streptococcus (GBS) is a common bacterium that can cause potentially devastating diseases in infants, including sepsis, pneumonia and meningitis, during the first three months of life. About one in four pregnant women carry GBS bacteria in their body and may pass it along to their baby during or prior to birth.5 Annually, there are an estimated 410,000 GBS cases worldwide, which cause at least 147,000 stillbirths and infant deaths each year.6
About Pfizer: Breakthroughs That Change Patients’ Lives
At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 170 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at www.Pfizer.com. In addition, to learn more, please visit us on www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.
Source: Pfizer Inc.
Pfizer's GBS vaccine for pregnant women to protect infants gets FDA breakthrough therapy status
Sep. 07, 2022 7:15 AM ET
By: Ravikash, SA News Editor1 Comment
- The U.S. Food and Drug Administration (FDA) granted breakthrough therapy designation to Pfizer's (NYSE:PFE) vaccine candidate GBS6 (PF-06760805) for preventing invasive Group B Streptococcus (GBS) disease in newborns by immunization of their mothers during pregnancy.
- https://seekingalpha.com/news/3880731-pfizers-gbs-vaccine-for-pregnant-women-to-protect-infants-gets-fda-breakthrough-therapy-status
- https://seekingalpha.com/symbol/PFE
- https://www.pfizer.com/
- https://www.cdc.gov/groupbstrep/index.html
Sarconeos (BIO101)
Group B Streptococcus (GBS)
Sarconeos (BIO101)
Biophytis Announces Very Promising Top Line Results of its Phase 2-3 COVA Clinical Study in COVID-19-Related Respiratory Failure
Wednesday, September 7, 2022 2:20 AM
Biophytis
https://www.biophytis.com/en/
Topic:
Company Update
PARIS, FRANCE and CAMBRIDGE, MA / ACCESSWIRE / September 7th, 2022 / Biophytis SA (NasdaqCM:BPTS)(Euronext Growth Paris:ALBPS) (the "Company" or "Biophytis"), a clinical-stage biotechnology company focused on the development of therapeutics that slow the degenerative processes associated with aging, including severe respiratory failure in patients suffering from COVID-19, today released top-line results from its phase 2-3 COVA clinical study evaluating Sarconeos (BIO101) in the treatment of COVID-19-related respiratory failure.
The objective of the study was to investigate the efficacy and safety of Sarconeos (BIO101), 350 mg BID in hospitalized COVID-19 patients with hypoxemia, at risk of respiratory failure requiring high flow oxygen or mechanical ventilation, and death. The proportion and time to onset of these negative events were studied for 28 days in the primary analysis, with follow-up of mortality and safety until 90 days after start of treatment. The 233 treated patients were 63 years old on average, 64% of the patients were male, recruited in centers in Europe, the US and Brazil between Q3 2020 and Q1 2022, infected with the main SARS-Cov-2 variants
In the primary analysis, Sarconeos (BIO101) reduced by 39% the risk of respiratory failure or early death at 28 days (primary endpoint) compared to placebo (15.8% vs 26.0%, adjusted difference 11.8% in favor of treatment, p=0.07). Sarconeos (BIO101) reduced both the proportion of patients with respiratory failure (12.7% vs 21.5%) and early death (0.8% vs 2.8%). Sarconeos (BIO101) also significantly (p=0.03) delayed the progression of respiratory failure or early death over 28 days maximum treatment period:
In addition, in this primary analysis, Sarconeos (BIO101) reduced the risk of death at 28 days compared to placebo, in similar proportion to the observed reduction in the risk of respiratory failure or early death and delayed its occurrence within 90 days. These effects are nonetheless not statistically significant.
Sarconeos (BIO101) presents a good safety profile, with a similar proportion of adverse events compared to placebo, particularly severe adverse events (25% vs 31%). In addition, more patients in the placebo group experienced respiratory adverse events than patients in the Sarconeos (BIO101) group (32.7% vs 22.7%, respectively), supporting the main efficacy results.
Further data analysis is still ongoing. Results of which will be communicated to the market and presented in details during a major international scientific conference in the next months.
Stanislas Veillet, CEO of Biophytis, said: "The results obtained with Sarconeos (BIO101) in the fight against COVID-19 are very encouraging. It is the only innovative drug candidate in Europe or the United States directly targeting respiratory failure that has demonstrated clinical efficacy in hospitalized patients with hypoxemia caused by COVID-19. It could be used in combination with certain anti-viral and/or anti-inflammatory drugs, which are now part of the medical practice. We will be sharing these results in the coming months with regulatory agencies, health authorities and our partners in Europe, the United States and Brazil to determine under what conditions we could pursue the development of Sarconeos (BIO101) in COVID-19. I would like to sincerely thank all the patients, caregivers, advisors, and finally the shareholders and employees of Biophytis for their hard work, trust and dedication throughout this study, the results of which come at a time when the fear of an epidemic rebound this winter is growing".
Dr Girish Balachandran Nair, Associate Professor of Medicine in Respiratory and Critical Care, Beaumont Hospital, Royal Oak, MI, USA and Principal Investigator of the COVA study in the USA, adds: "These results are extremely exciting and of public interest. This is great news for the medical community and the general population at risk, especially elderly patients. Indeed, COVID-19 is becoming endemic and will persist for a long time. Sarconeos (BIO101) could provide a real therapeutic alternative for elderly patients, hospitalized with hypoxemia and at risk of respiratory failure."
Based on these very promising results, the Company intends to make this new therapeutic solution available to COVID-19 hospitalized patients at risk of respiratory failure and early death. Actually, according to the Johns Hopkins Coronavirus Resource Center, during the last month in the US, Brazil and France, 2.6 million, 0.5 million and 0.6 million people respectively were infected by COVID-19 and 13.9 thousand, 4.5 thousand and 1.7 thousand died from it. Beyond COVID-19 infection, other infections such as flu can also generate Acute Respiratory Distress Syndrome (ARDS).
Biophytis is currently evaluating the possibility of amending and continuing the Early Access Program (EAP) to make Sarconeos (BIO101) available as soon as this winter to COVID-19 hospitalized patients and at risk of respiratory failure and death. An EAP has already been authorized in Brazil to treat patients in intensive care and further EAP might be filed in other territories including the USA and Europe. This is a first step towards market authorization, the conditions of which will be discussed in the coming months with the relevant regulatory authorities (FDA, EMA and ANVISA).
About the COVA study
As a reminder, the COVA clinical programme (identifier clinicaltrials.gov: NCT04472728) is an international, multi-centre, double-blind, placebo-controlled, group-sequential and adaptive two-part study. It is a phase 2-3 study evaluating Sarconeos (BIO101) in patients aged 45 years and older, hospitalised with severe respiratory manifestations of COVID-19. Part 1 of the COVA study is an exploratory Phase 2 proof-of-concept study designed to provide preliminary data on the safety, tolerability and efficacy of Sarconeos (BIO101) in 50 hospitalised patients with severe respiratory failure in patients suffering from COVID-19. Part 2 of the COVA study is a randomised phase 3 study investigating the safety and efficacy of Sarconeos (BIO101) on respiratory function in patients. Due to the evolution of the pandemia, the company decided
in April 2022 to stop enrolment at 237 patients.
About BIOPHYTIS
Biophytis SA is a clinical-stage biotechnology company specialized in the development of therapeutics that are aimed at slowing the degenerative processes associated with aging and improving functional outcomes for patients suffering from age-related diseases, including severe respiratory failure in patients suffering from COVID-19. Sarconeos (BIO101), our leading drug candidate, is a small molecule, administered orally, being developed as a treatment for sarcopenia in a Phase 2 clinical trial in the United States and Europe (SARA-INT). It has also been studied in a clinical two-part Phase 2-3 study (COVA) for the treatment of severe respiratory manifestations of COVID-19 in Europe, Latin America, and the US. A pediatric formulation of Sarconeos (BIO101) is being developed for thetreatment of Duchenne Muscular Dystrophy (DMD). The Company is based in Paris, France, and Cambridge, Massachusetts. The Company's ordinary shares are listed on Euronext Growth (Ticker: ALBPS -ISIN: FR0012816825) and ADSs (American Depositary Shares) are listed on Nasdaq Capital Market (Ticker BPTS - ISIN: US09076G1040). For more information visit www.biophytis.com.
SOURCE: Biophytis
Biophytis stock soars 45% as COVID drug cuts risk of death, respiratory failure in trial
Sep. 07, 2022 5:44 AM ET
By: Ravikash, SA News Editor
Biophytis (NASDAQ:BPTS) said its drug Sarconeos was better at reducing the risk of respiratory failure or early death in patients with COVID-19,compared to placebo.
https://seekingalpha.com/symbol/BPTS
https://www.biophytis.com/en/pipeline/covid19-programme-clinique-cova/
https://www.biophytis.com/en/our-science/sarcopenie/
Eplontersen
XenpozymeTM (olipudase alfa-rpcp)
Sarconeos (BIO101)
Ionis presents positive results from Phase 3 NEURO-TTRansform study at International Symposium on Amyloidosis
September 7, 2022 at 2:30 AM EDT
- Ionis and AstraZeneca's eplontersen demonstrated a statistically significant and clinically meaningful change from baseline for co-primary and secondary endpoints at 35 weeks compared to the external placebo group
- Eplontersen achieved an 81.2% reduction in the co-primary endpoint of serum transthyretin (TTR) concentration from baseline, demonstrating reduced TTR protein production
- Eplontersen demonstrated a favorable safety and tolerability profile
CARLSBAD, Calif., Sept. 7, 2022 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today presented positive results from a planned 35-week interim analysis of the Phase 3 NEURO-TTRansform study of Ionis and AstraZeneca's eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). In the study, eplontersen demonstrated a statistically significant and clinically meaningful change from baseline for its co-primary and secondary efficacy endpoints compared to the external placebo group. Eplontersen demonstrated a favorable safety and tolerability profile. The study results were presented today at the International Symposium on Amyloidosis (ISA) in Heidelberg, Germany.
In the study, eplontersen achieved an 81.2% (p<0.0001) mean reduction in the co-primary endpoint of serum transthyretin (TTR) concentration compared to baseline, demonstrating reduced TTR protein production. Eplontersen also demonstrated a significant treatment effect on the co-primary endpoint of modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression, with a statistically significant difference in mean change from baseline versus the external placebo group (p<0.0001). The study also met its key secondary endpoint of change from baseline in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN), showing that treatment with eplontersen significantly improved patient-reported quality of life compared to the external placebo group (p<0.0001).
Eplontersen demonstrated a favorable safety and tolerability profile. In the study, the rate of treatment emergent adverse events (TEAEs) in the eplontersen group was either lower or similar compared to placebo across all major categories. There were no TEAEs of special interest leading to drug discontinuation.
The study data are consistent with the clinical profile seen across Ionis' other LICA programs, further validating how the company's LIgand-Conjugated Antisense technology positions Ionis to deliver potentially transformative treatments for a broad range of unmet medical needs.
"Eplontersen showed clinically meaningful improvement in neuropathy impairment and quality of life measures relative to baseline. The significant efficacy, combined with a favorable safety and tolerability profile, indicate that eplontersen has the potential to be an important therapeutic option for patients living with this debilitating and fatal disease," said Teresa Coelho, M.D., a neurologist and neurophysiologist at Hospital Santo António, Centro Hospitalar Universitário do Porto, Portugal and an investigator for the NEURO-TTRansform study. Dr. Coelho presented data from the interim analysis at ISA.
"The promising results from NEURO-TTRansform show that eplontersen had a positive impact on disease progression and improved quality of life in a substantial number of patients. We are excited about the potential for delivering a new treatment option to patients living with this relentless and devastating disease," said Eugene Schneider, M.D., Ionis' executive vice president and chief clinical development officer.
Based on the study results, Ionis and AstraZeneca will seek regulatory approval for eplontersen for ATTRv-PN and plan to file a new drug application with the U.S. Food and Drug Administration this year.
As part of a global development and commercialization agreement with AstraZeneca, eplontersen is being jointly developed and commercialized by both companies in the U.S. and will be developed and commercialized in the rest of the world by AstraZeneca (with the exception of Latin America).
Eplontersen was granted Orphan Drug Designation in the U.S. It is also currently being evaluated in the Phase 3 CARDIO-TTRansform study for amyloid transthyretin cardiomyopathy (ATTR-CM), a systemic, progressive and fatal condition that leads to progressive heart failure and death within four years from diagnosis.
About NEURO-TTRansform (NCT04136184)
NEURO-TTRansform is a global, open-label, randomized study evaluating the efficacy and safety of eplontersen in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN). The study has enrolled adult patients with stage 1 or stage 2 polyneuropathy and will compare efficacy of eplontersen to the historical placebo arm from the TEGSEDI® (inotersen) NEURO-TTR registrational study that Ionis completed in 2017. The final primary endpoint analysis will be completed at week 66 and all patients will be followed until week 85 when they will have the option to transition into the open label extension study.
About Eplontersen
Eplontersen is an investigational antisense medicine that uses Ionis' advanced LIgand-Conjugated Antisense, or LICA, technology designed to inhibit the production of the transthyretin (TTR) protein at its source. Eplontersen, which is planned to be delivered to patients via a self-administered autoinjector, is in development to treat all types of ATTR, a systemic, progressive and fatal disease.
About Ionis Pharmaceuticals, Inc.
For more than 30 years, Ionis has been the leader in RNA-targeted therapy, pioneering new markets and changing standards of care with its novel antisense technology. Ionis currently has three marketed medicines and a premier late-stage pipeline highlighted by industry-leading cardiovascular and neurological franchises. Our scientific innovation began and continues with the knowledge that sick people depend on us, which fuels our vision of becoming a leading, fully integrated biotechnology company.
To learn more about Ionis, visit www.ionispharma.com and follow us on Twitter @ionispharma.
View original content to download multimedia:https://www.prnewswire.com/news-releases/ionis-presents-positive-results-from-phase-3-neuro-ttransform-study-at-international-symposium-on-amyloidosis-301618729.html
SOURCE Ionis Pharmaceuticals, Inc.
AstraZeneca/Ionis present data for rare disease drug meeting trial's main goal
Sep. 07, 2022 6:47 AM ET
Ionis Pharmaceuticals, Inc. (IONS), AZN
By: Ravikash, SA News Editor2 Comments
AstraZeneca (NASDAQ:AZN) and Ionis Pharmaceuticals (NASDAQ:IONS) presented detailed results from a 35-week interim analysis of a phase 3 trial of eplontersen, which had met its main goal, in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).
The company presented the data at the International Symposium on Amyloidosis in Germany. Broad results were reported in June.
https://seekingalpha.com/symbol/IONS
https://seekingalpha.com/symbol/AZN
https://www.ionispharma.com/medicines/akcea-ttr-l/
XenpozymeTM (olipudase alfa-rpcp)
XenpozymeTM (olipudase alfa-rpcp)
XenpozymeTM (olipudase alfa-rpcp)
August 31, 2022
Press Release: XenpozymeTM (olipudase alfa-rpcp) approved by FDA as first disease-specific treatment for ASMD (non-CNS manifestations)
Press Release: XenpozymeTM (olipudase alfa-rpcp) approved by FDA as first disease-specific treatment for ASMD (non-CNS manifestations)
XenpozymeTM (olipudase alfa-rpcp) approved by FDA as first disease-specific treatment for ASMD (non-CNS manifestations)
Paris, August 31, 2022. The U.S. Food and Drug Administration (FDA) has approved XenpozymeTM (olipudase alfa-rpcp) for the treatment of non-central nervous system (non-CNS) manifestations of acid sphingomyelinase deficiency (ASMD) in adult and pediatric patients. Xenpozyme is the first therapy indicated specifically for the treatment of ASMD, and is currently the only approved treatment for this disease.
Bill Sibold
Executive Vice President, Head, Specialty Care at Sanofi
“Sanofi teams have been dedicated to bringing hope to patients living with ASMD and their families. This is a devastating and extremely rare disease that affects both children and adults. The approval of Xenpozyme represents the culmination of bold work done in research and development, and our unwavering commitment to this historically overlooked community.”
ASMD, historically known as Niemann-Pick disease types A, A/B, and B, is an extremely rare, progressive genetic disease with significant morbidity and mortality. It has been estimated that there are fewer than 120 patients diagnosed with ASMD in the U.S. Approximately two-thirds of patients with ASMD in the U.S. are pediatric. Signs and symptoms of ASMD can present in infancy, childhood, or adulthood, and may include enlarged spleen or liver, difficulty breathing, lung infections, and unusual bruising or bleeding, among other disease manifestations. Until now, management of ASMD included supportive care to address the impact of individual symptoms and careful monitoring to detect potential disease complications.
David Guy
Parent to Kaila, age 16, living with ASMD
“As young parents, it was initially devastating to me and my wife when our daughter, Kaila, received her diagnosis of ASMD. We faced so many unknowns when we first heard the diagnosis: what does this mean, how will this affect her, and most importantly what hope is there for a treatment option? We were grateful to find hope when we enrolled Kaila in the clinical trials for olipudase alfa.”
In the U.S., Xenpozyme received Breakthrough Therapy designation, which expedites the development and review of drugs intended to treat serious or life-threatening diseases and conditions. The FDA evaluated Xenpozyme under Priority Review, which is reserved for medicines that represent potentially significant improvements in efficacy or safety in treating serious conditions.. In March 2022, Xenpozyme was approved in Japan under the SAKIGAKE (or “pioneer”) designation, marking the first approval for olipudase alfa anywhere in the world. In June 2022, the European Commission (EC) approved Xenpozyme for use in Europe.
ASMD represents a spectrum of disease, with two types that may represent opposite ends of a continuum referred to as ASMD type A and ASMD type B. ASMD type A/B is an intermediate form that includes varying degrees of central nervous system (CNS) involvement.
ASCEND and ASCEND-Peds clinical trials showed that Xenpozyme improved lung function and reduced spleen and liver volumes in adults and children
The approval is based on positive data from the ASCEND and ASCEND-Peds clinical trials, in which Xenpozyme showed clinically relevant improvement in lung function (as measured by diffusing capacity of the lung for carbon monoxide, or DLco) and platelet count, and reduction of spleen and liver volumes, with a demonstrated safety profile.
Melissa Wasserstein
MD, Pediatric Genetic Medicine, Albert Einstein College of Medicine and the Children’s Hospital at Montefiore
“ASMD is an extremely rare, progressive, and potentially fatal genetic disease that impacts children and adults around the world. Until now, those living with ASMD have had no FDA-approved treatment to combat this devastating condition. I’m proud of the work that has been done and look forward to witnessing the impact that this treatment may have on those living with ASMD.”
The ASCEND trial evaluated the efficacy and safety of Xenpozyme; 31 adult patients with ASMD type A/B or type B were randomized to receive Xenpozyme or placebo for 52 weeks (primary analysis). In the trial, Xenpozyme improved lung function, assessed as the percent change from baseline to Week 52 in predicted diffusing capacity of the lung for carbon monoxide (DLco), and reduced spleen volume, evaluated as percent change from baseline in multiples of normal (MN).
- Twelve (12) patients treated with Xenpozyme had a mean change in percent predicted DLco from baseline (49.1%) to Week 52 (59.4%). This change represents a 23.9% relative improvement compared to a 3% improvement in DLco from baseline in the 17 patients from the placebo group (48.5%) to Week 52 (49.9%). The difference between the two arms (20.9%) was nominally statistically significant (p=0.0003).
- Thirteen (13) patients treated with Xenpozyme had a mean reduction in spleen volume by 38.9% from baseline (11.5 MN) to Week 52 (7.2 MN) compared to a mean increase by 0.5% for the 17 patients in the placebo group from baseline (11.2 MN) to Week 52 (11.2 MN). The difference between the two arms (39.4%) was nominally statistically significant (p<0.0001).
- Twelve (12) patients treated with Xenpozyme had a mean reduction in liver volume by 26.5% from baseline (1.4 MN) to Week 52 (1.0 MN) compared to a mean decrease of 1.8% for the 17 patients in the placebo group from baseline (1.6 MN) to Week 52 (1.6 MN). The difference between the two arms (24.7%) was nominally statistically significant (p<0.0001).
- Thirteen (13) patients treated with Xenpozyme had a mean improvement in platelet count by 18.3% from baseline (109.3x109/L) to Week 52 (126.4x109/L) compared to increase by 2.7% for the 16 patients in the placebo group from baseline (115.6x109/L) to Week 52 (120.2x109/L). The difference between the two arms (15.6%) was nominally statistically significant (p=0.0280).
- All ASCEND patients treated with Xenpozyme showed improvement in key endpoints (DLco and spleen and liver volume).
- Most frequently reported adverse drug reactions in adults (incidence ≥10%) were headache, cough, diarrhea, hypotension, and ocular hyperemia.
The single-arm ASCEND-Peds trial studied 8 pediatric patients younger than 12 years of age with ASMD type A/B or type B who all received Xenpozyme, with a primary objective of evaluating the safety and tolerability of Xenpozyme for 64 weeks. All patients completed the study and continued in an extension trial. The ASCEND-Peds trial also explored efficacy endpoints of progressive lung disease, spleen and liver enlargement, and platelet count. After one year of treatment (52 weeks):
- Three (3) patients who were able to perform the test at baseline treated with Xenpozyme had a mean relative improvement of 45.9% in percent predicted DLco from baseline (48.5%) to Week 52 (70.9%) (children over the age of five were assessed if they were able to perform the test).
- Eight (8) patients treated with Xenpozyme had mean reduction in spleen volume by 46.7% from baseline (18.3 MN) to Week 52 (9.5 MN).
- Eight (8) patients treated with Xenpozyme had a mean reduction in liver volume by 38.1% from baseline (2.5 MN) to Week 52 (1.6 MN).
- Seven (7) patients treated with Xenpozyme had a mean improvement in platelet count by 37.6% from baseline (136.7x109/L; n=8) to Week 52 (184.5x109/L).
- Serious adverse reactions of anaphylactic reaction were reported in 2 (25%) Xenpozyme-treated pediatric patients.
- Treatment-related serious adverse reactions, hypersensitivity reactions including anaphylaxis, and infusion associated reactions occurred within 24 hours of infusion and were observed in a higher percentage of pediatric patients than in adult patients.
- Most frequently reported adverse drug reactions in pediatric patients (incidence ≥20%) were pyrexia, cough, diarrhea, rhinitis, abdominal pain, vomiting, headache, urticaria, nausea, rash, arthralgia, pruritus, fatigue, and pharyngitis.
A scientific innovation for patients living with ASMD
Xenpozyme, a hydrolytic lysosomal sphingomyelin-specific enzyme replacement therapy, is designed to replace deficient or defective acid sphingomyelinase (ASM), an enzyme that allows for the breakdown of the lipid sphingomyelin. In individuals with ASMD, the deficiency in the ASM enzyme leads to sphingomyelin accumulation in various tissues. Xenpozyme is not expected to cross the blood-brain barrier or modulate CNS manifestations of ASMD. Xenpozyme has not been studied in patients with ASMD type A.
Xenpozyme is adminstered intravenously every two weeks, and its administration requires a dose escalation phase followed by a maintenance phase.
Xenpozyme is expected to be available in the U.S. in the coming weeks. The U.S. list price, or wholesale acquisition cost, of Xenpozyme is $7,142.00 per vial. Actual patient out-of-pocket costs may be lower, as the list price does not reflect insurance coverage, co-pay support for eligible patients, or financial assistance from patient support programs.
Sanofi is committed to helping eligible U.S. patients access the support they need and to help reduce barriers throughout their treatment journey. As part of its commitment to treatment access and affordability for innovative therapies, Sanofi provides disease education, financial and co-pay assistance programs, and other support services to eligible patients. For more information, patients can call 1-800-745-4447 and select Option 3, contact Info@CareConnectPSS.com, or visit www.Xenpozyme.com.
About Sanofi
We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives. Our team, across some 100 countries, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions.
Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY
FDA approves Sanofi's Xenpozyme for the treatment of rare genetic disease ASMD
Aug. 31, 2022 2:09 PM ET
By: Anuron Mitra, SA News Editor
- The U.S. Food and Drug Administration (FDA) on Wednesday approved Genzyme's Xenpozyme drug for intravenous infusion for the treatment of kids and adults with acid sphingomyelinase deficiency (ASMD), a rare genetic disease.
- Genzyme is a Sanofi (NASDAQ:SNY) company.
- https://seekingalpha.com/news/3879049-fda-approves-sanofis-xenpozyme-for-the-treatment-of-rare-genetic-disease-asmd
- https://seekingalpha.com/symbol/SNY
- https://www.fda.gov/news-events/press-announcements/fda-approves-first-treatment-acid-sphingomyelinase-deficiency-rare-genetic-disease
- https://www.sanofi.com/en/
Lomecel-B™
XenpozymeTM (olipudase alfa-rpcp)
XenpozymeTM (olipudase alfa-rpcp)
U.S. Food and Drug Administration (FDA) Grants Fast Track Designation for Longeveron’s Lomecel-B™ Product for Treatment of Hypoplastic Left Heart Syndrome (HLHS) in Infants
08/31/2022New designation may expedite FDA review and potential approval to address this life-threatening heart condition affecting approximately 1,000 babies per year
MIAMI, Aug. 31, 2022 (GLOBE NEWSWIRE) -- Longeveron Inc. (NASDAQ: LGVN) ("Longeveron" or "Company"), a clinical stage biotechnology company developing cellular therapies for chronic aging-related and life-threatening conditions, today announced the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to Lomecel-B™ for the treatment of Hypoplastic Left Heart Syndrome (HLHS), a rare and life-threatening congenital heart defect affecting approximately 1,000 infants per year. Lomecel-B™, an investigational allogeneic, bone marrow-derived medicinal signaling cell (MSC) product, is currently in a Phase 2a trial for HLHS.
Fast Track Designation is intended to facilitate development and expedite the review of drugs that treat serious conditions and fill an unmet medical need so a product can potentially be approved and reach patients more quickly. Fast Track Designation enables the company to have more frequent interactions with the FDA throughout the drug development process and allows for eligibility for priority review and accelerated approval if certain criteria are met, as well as a rolling review. The Fast Track Designation must continue to be met or FDA can withdraw the designation. The FDA previously granted Longeveron’s Lomecel-B™ Orphan Drug and Rare Pediatric Disease designations in November of 2021 for HLHS.
“Fast Track Designation represents a significant milestone in our efforts to develop Lomecel-B™ as a treatment for infants with HLHS,” said Chris Min, M.D., Ph.D., Longeveron’s Interim Chief Executive Officer and Chief Medical Officer. “Fast Track Designation underscores the urgent need in HLHS, and we look forward to continuing to work closely with the FDA to bring this potential new therapy to infants as expeditiously as possible.”
HLHS is a rare congenital heart defect that affects approximately 1,000 babies per year in the U.S. Infants born with HLHS have an underdeveloped or absent left ventricle, which impairs the heart’s ability to pump adequate amounts of blood throughout the body. Patients require three reconstructive heart surgeries within the first five years of life, and many require a heart transplant. Without treatment, the condition is always fatal. Even with reconstructive surgical interventions, HLHS is still associated with high mortality. Overall survival from birth to adolescence is estimated to be 50% to 60%.
Longeveron is currently evaluating Lomecel-B™ in ELPIS II, an ongoing 38-patient, randomized (1:1), blinded, controlled Phase 2a clinical trial intended to evaluate the safety and efficacy of intramyocardial (directly into the heart) injection of the Lomecel-B™ product in infants with HLHS who are undergoing Stage II reconstructive cardiac surgery. In ELPIS, a Phase 1 open-label trial in ten HLHS patients, Lomecel-B™ was found to be well tolerated with no major adverse cardiac events one-year post-surgery, and/or treatment-related infections.
One hundred percent of the infants enrolled in the ELPIS Phase 1 trial (n=10) were alive and had not required a transplant between 2-3.5 years post-surgery. Normally, approximately 20% of patients require heart transplants to survive within one year of Stage II reconstructive surgery. Longeveron recently announced the release of a manuscript entitled “Intramyocardial cell-based therapy during bidirectional cavopulmonary anastomosis for hypoplastic left heart syndrome: The ELPIS phase I trial” on MedRxiv, a preprint server that posts papers before they are peer reviewed. The paper will now undergo peer review prior to publication of the final study report.
About Longeveron Inc.
Longeveron is a clinical stage biotechnology company developing cellular therapies for specific aging-related and life-threatening conditions. The Company’s lead investigational product is the Lomecel-B™ cell-based therapy product, which is derived from culture-expanded medicinal signaling cells (MSCs) that are sourced from bone marrow of young, healthy adult donors. Longeveron believes that by using the same cells that promote tissue repair, organ maintenance, and immune system function, it can develop safe and effective therapies for some of the most difficult disorders associated with the aging process and other medical disorders. Longeveron is currently sponsoring Phase 1 and 2 clinical trials in the following indications: Alzheimer’s disease, hypoplastic left heart syndrome (HLHS), Aging Frailty, and Acute Respiratory Distress Syndrome (ARDS). Additional information about the Company is available at www.longeveron.com.
Source: Longeveron
Longeveron spikes after FDA’s Fast Track Designation for lead candidate
Aug. 31, 2022 8:45 AM ET
By: Dulan Lokuwithana, SA News Editor
- The shares of clinical-stage biotech Longeveron Inc. (NASDAQ:LGVN) jumped in the pre-market trading Wednesday after the company announced that the FDA issued Fast Track Designation for its lead asset Lomecel-B as a treatment for Hypoplastic Left Heart Syndrome (HLHS).
- https://seekingalpha.com/news/3878887-lgvn-stock-spikes-after-fdas-fast-track-designation-for-lead-candidate
- https://seekingalpha.com/symbol/LGVN
- https://www.longeveron.com/lomecel-b
- https://www.longeveron.com/
biotecHOPE™ 2022
RGX-121
Cemdisiran (ALN-CC5)
RGX-121
REGENXBIO PRESENTS ADDITIONAL POSITIVE INTERIM DATA FROM THE PHASE I/II/III CAMPSIITE™ TRIAL OF RGX-121 FOR THE TREATMENT OF MPS II (HUNTER SYNDROME) AT THE SOCIETY FOR THE STUDY OF INBORN ERRORS OF METABOLISM (SSIEM) ANNUAL SYMPOSIUM
August 31, 2022 at 7:05 AM EDT
- New data in Cohort 3 using pivotal program dose level demonstrates largest median reduction in CSF GAGs, continuing to approach normal levels at 48 weeks
- RGX-121, a one-time gene therapy for MPS II, continues to be well-tolerated with no drug-related SAEs across three dose levels
- Positive interim data supports recently announced plan to file Biologics License Application in 2024 using the accelerated approval pathway
ROCKVILLE, Md., Aug. 31, 2022 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced additional positive interim data from the Phase I/II/III CAMPSIITE™ 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 results were presented at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium.
"We are pleased to share new, positive interim data from our Phase I/II/III CAMPSIITE trial that continues to demonstrate encouraging reductions of CSF GAGs and supports the selection of dose level 3 for our pivotal program," said Steve Pakola, M.D., Chief Medical Officer of REGENXBIO. "This interim data of Cohorts 1-3 demonstrate dose-dependent reductions in CSF GAGs, key biomarkers of I2S enzyme activity, in additional patients and at longer timepoints than previously presented. RGX-121 has also demonstrated positive impact on neurodevelopmental function. We believe the data supports our plan to advance RGX-121 as quickly as possible using the accelerated approval pathway with the aim of providing a much-needed new treatment option for the MPS II community."
"RGX-121 continues to demonstrate compelling data showing its potential to impact the neurodevelopmental decline of MPS II patients, which remains an unmet need for most affected individuals," said Roberto Giugliani, M.D., Ph.D., Professor, Department of Genetics, UFRGS, Medical Genetics Service, HCPA, Porto Alegre, Brazil. "A successful gene therapy with the potential to provide advantages over current standard of care is something that could provide a meaningful treatment option to the MPS II patient community. I look forward to continuing to follow this trial as the recently announced pivotal program progresses."
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. Data presented at SSIEM was from the Phase I/II portion of the CAMPSIITE trial where the primary endpoint is to evaluate the safety of RGX-121. Secondary and exploratory endpoints include cerebral spinal fluid (CSF) glycosaminoglycans (GAGs), neurodevelopmental assessments, caregiver reported outcomes and systemic biomarkers. RGX-121 is administered directly to the central nervous system (CNS). As of August 1, 2022, patients had been treated across three dose levels, 1.3x1010 genome copies per gram (GC/g) of brain mass (n=3), 6.5x1010 GC/g of brain mass (n=7), and 2.9x1011 GC/g of brain mass (n=4).
Data Summary and Safety Update
As of August 1, 2022, RGX-121 is reported to be well-tolerated across all cohorts with no drug-related serious adverse events (SAEs) in 14 patients dosed with RGX-121. Time of post-administration follow-up ranges from eight weeks to two years. Eleven patients have completed the 48-week immunosuppression regimen per study protocol. Twelve patients were receiving weekly, intravenous enzyme replacement therapy (ERT) at the time of enrollment, per standard of care; three of these patients have discontinued ERT, per investigator discretion, as allowed in the protocol.
CSF GAGs Data
Biomarker data from patients in all three cohorts indicate encouraging, dose-dependent reductions of CSF GAGs following one-time administration of RGX-121. Heparan sulfate (HS) and D2S6, a component of HS closely correlated with severe MPS II, are GAGs that are key biomarkers of I2S enzyme activity and are being measured in the CSF at baseline and after administration of RGX-121. GAGs in the CSF have the potential to be considered a surrogate biomarker that is reasonably likely to predict clinical benefit in MPS II disease under the accelerated approval pathway, as buildup of GAGs in the CSF of MPS II patients correlates with clinical manifestations, including neurodevelopmental deficits.
The majority of patients in all three cohorts demonstrated reductions of HS in the CSF following RGX-121 administration at the last time point available with dose-dependent reductions seen at Weeks 8, 24, and 48 post RGX-121 administration. At Week 48, median reduction of CSF HS from baseline was 33.5% in Cohort 1, 52.9% in Cohort 2 and 62.5% in Cohort 3.
Similarly, dose-dependent reductions of CSF HS D2S6 were observed at last time point available following RGX-121 administration in the majority of patients, with Cohort 3 patients approaching normal levels at 48 weeks. All three cohorts demonstrated reduction in HS D2S6 with dose-dependent reductions seen at Weeks 8, 24, and 48. Median reduction from baseline of 31.9% in Cohort 1, 69.4% in Cohort 2 and 83.1% in Cohort 3 was seen at Week 48.
In addition, I2S protein concentration in the CSF, which was undetectable in all patients prior to dosing, was measurable in Cohort 2 and 3 patients after RGX-121 administration.
Neurodevelopmental and Systemic Data
As previously reported at the 2022 WORLDSymposium™, improvements in neurodevelopmental function and caregiver reported outcomes in Cohorts 1 and 2 demonstrated CNS activity up to 2 years after RGX-121 administration. Additionally, evidence of systemic enzyme expression and biomarker activity was observed in patients across all three cohorts following RGX-121 administration. The majority of patients demonstrated increases in I2S protein concentration levels in plasma following administration of RGX-121. Total urine GAG measures demonstrated evidence of a systemic effect of RGX-121, independent of ERT treatment.
The study findings presented at the SSIEM Annual Symposium will be available under the Presentations & Publications page in the Media section of REGENXBIO's website located at www.regenxbio.com.
About the CAMPSIITE™ Trial
CAMPSIITE is a multicenter, open-label trial enrolling boys with MPS II, aged 4 months up to five years of age. CAMPSIITE is expected to enroll up to 10 MPS II patients to support the BLA filing using the accelerated approval pathway, with the potential to enroll additional patients. These patients will receive a dose of 2.9x1011 GC/g of brain mass of RGX-121, which is the same dose being evaluated in Cohort 3 of the Phase I/II trial. The pivotal program is using commercial-scale cGMP material from REGENXBIO's proprietary, high-yielding suspension-based manufacturing process, named NAVXPress™. In addition to measuring GAGs in the CSF, the trial will continue to collect neurodevelopmental data and caregiver-reported outcomes.
CAMPSIITE is a global trial, which is expected to include sites in the United States, Brazil and Canada. REGENXBIO has begun dosing patients in the pivotal program.
About RGX-121
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.
About REGENXBIO Inc.
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates, including late-stage and commercial programs, in multiple therapeutic areas. REGENXBIO is committed to a "5x'25" strategy to progress five AAV Therapeutics from our internal pipeline and licensed programs into pivotal-stage or commercial products by 2025.
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SOURCE REGENXBIO Inc.
REGENXBIO shares more data for Hunter Syndrome candidate
Aug. 31, 2022 8:07 AM ET
By: Dulan Lokuwithana, SA News Editor
REGENXBIO Inc. (NASDAQ:RGNX), a biotech focused on gene therapies, announced Wednesday new data supporting the pivotal program for RGX-121, its candidate for the genetic disorder Hunter Syndrome.
https://seekingalpha.com/news/3878866-regenxbio-shares-more-data-for-hunter-syndrome-candidate
https://seekingalpha.com/symbol/RGNX
https://regenxbio.com/rgx-121/
BCX9250
Cemdisiran (ALN-CC5)
RGX-121
FDA Grants Orphan Drug Designation for BioCryst’s ALK-2 Inhibitor, BCX9250, for the Treatment of Fibrodysplasia Ossificans Progressiva
RESEARCH TRIANGLE PARK, N.C., Aug. 31, 2022 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for BCX9250 for the treatment of fibrodysplasia ossificans progressiva (FOP).
The FDA has the option to grant orphan drug designation to applicants for the purpose of supporting the development of a drug or biologic intended to treat a disease or condition that affects fewer than 200,000 individuals in the U.S. Companies that receive this designation are eligible for incentives, including tax credits for qualified clinical trials, exemption from user fees and up to seven years of market exclusivity after approval.
“We appreciate the FDA’s decision to grant orphan drug designation to BCX9250 as we work toward our goal of bringing this important oral investigational therapy to FOP patients. The benefits available to us through this designation – and the designations we previously received from the FDA and the EMA – have the potential to advance our ALK-2 inhibitor program as efficiently as possible as we move towards beginning trials in FOP patients next year,” said Dr. Helen Thackray, chief research and development officer of BioCryst.
FOP is an ultra-rare, severely disabling genetic disorder characterized by heterotopic ossification (HO), or the irregular formation of bone outside the normal skeleton. HO can occur in muscles, tendons, ligaments and other connective tissues. Patients with FOP become bound by this irregular ossification over time, with restricted movement and fused joints, resulting in deformities, restricted mobility and premature mortality.
Earlier this year, BioCryst announced that BCX9250 has received Fast Track designation from the FDA, in addition to orphan drug designation and PRIME designation from the European Medicines Agency (EMA).
BioCryst will present three posters featuring data from non-clinical studies and first-in-human Phase 1 safety, tolerability and pharmacokinetics study in healthy subjects from the BCX9250 program at the American Society for Bone and Mineral Research Annual Meeting, which is being held in Austin, Texas, from Sept. 9-12, 2022. As the company previously reported in the Phase 1 study, BCX9250 was safe and well tolerated at all doses studied, with linear and dose-proportional exposure supporting the potential for once-daily dosing.
About BCX9250
BCX9250 is a novel, oral activin receptor-like kinase-2 (ALK-2) inhibitor designed to inhibit the ALK-2 enzyme, which is a part of the normal signaling pathway for bone formation and responds to binding its specific ligands (bone morphogenic proteins, BMPs) by stimulating normal bone growth and renewal in healthy children and adults. Specific activating mutations of the ALK-2 gene are seen in all cases of fibrodysplasia ossificans progressiva (FOP). An activating mutation in ALK-2 is necessary for the disease to occur, making the ALK-2 enzyme an ideal drug target for treatment of FOP. In a Phase 1 clinical trial in healthy subjects, BCX9250 was safe and well tolerated at all doses studied, with linear and dose-proportional exposure supporting the potential for once-daily dosing.
About BioCryst Pharmaceuticals
BioCryst Pharmaceuticals discovers novel, oral, small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease. Oral, once-daily ORLADEYO® (berotralstat) is approved in the United States and multiple global markets. BioCryst has several ongoing development programs including BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases, BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva, and galidesivir, a potential treatment for Marburg virus disease and yellow fever. RAPIVAB® (peramivir injection) is approved in the U.S. and multiple global markets, with post-marketing commitments ongoing. For more information, please visit the company’s website at www.biocryst.com.
BioCryst wins FDA’s Orphan Drug status for bone disorder therapy
Aug. 31, 2022 7:34 AM ET
BioCryst Pharmaceuticals, Inc. (BCRX)
By: Dulan Lokuwithana, SA News Editor1 Comment
- Commercial-stage biotech BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) announced Wednesday that the FDA granted the orphan drug designation for its experimental drug BCX9250 for the treatment of the rare genetic disorder fibrodysplasia ossificans progressiva (FOP).
- https://seekingalpha.com/news/3878846-biocryst-wins-fdas-orphan-drug-status-for-bone-disorder-therapy
- https://seekingalpha.com/symbol/BCRX
- https://www.biocryst.com/our-pipeline/
- https://www.biocryst.com/
Cemdisiran (ALN-CC5)
Cemdisiran (ALN-CC5)
Cemdisiran (ALN-CC5)
Alnylam Reports Positive Results from Phase 2 Study of Investigational Cemdisiran for the Treatment of IgA Nephropathy
Aug 29, 2022
-Treatment with Cemdisiran Resulted in a Higher Proportion of Patients Achieving Clinically Meaningful Reductions in 24-Hour Urine Protein to Creatinine Ratio, as Compared to Patients on Placebo -
- Consistent Proteinuria Reduction Seen with Secondary Endpoints -
- Cemdisiran Was Generally Well-Tolerated in Patients with IgA Nephropathy -
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Aug. 29, 2022-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY) today announced positive results from a Phase 2 study of cemdisiran, an investigational RNAi therapeutic targeting the C5 component of the complement pathway, in development in collaboration with Regeneron Pharmaceuticals for the treatment of adult patients with immunoglobulin A nephropathy (IgAN). The results were presented at the 18th European Meeting on Complement in Human Disease (EMCHD) being held in Bern, Switzerland on August 26-28.
“We are pleased to present additional data from our Phase 2 study demonstrating that cemdisiran had favorable effects on different measures of proteinuria – a strong risk factor for disease progression in patients with IgAN,” said Sonalee Agarwal, Ph.D., Vice President and Program Leader for the Cemdisiran program at Alnylam. “IgAN is an inflammatory disease that can lead to severe loss of kidney function. Thus, given the persistent unmet need in the treatment landscape of this progressive disease, we, together with our partners at Regeneron, are working expeditiously to advance this investigational RNAi therapeutic into Phase 3 clinical development.”
The data showed that at Week 32, as previously reported, cemdisiran demonstrated a clinically meaningful reduction in 24-hour urine protein to creatinine ratio (UPCR) – the primary endpoint of this Phase 2 study – with a 37 percent (90 percent CI: -0.5, 61) reduction in 24-hour UPCR observed relative to placebo. New results that were presented demonstrated an equivalent (36 percent [90 percent CI: -6, 62]) reduction in 24-hour urine total protein and a higher proportion, 32 versus 13 percent, of patients treated with cemdisiran as compared to those on placebo, respectively, achieving greater than or equal to 50 percent reduction in 24-hour UPCR. Spot urine data were consistent with 24-hour urine data, with the initial onset of treatment effect emerging as early as Week 8 and remaining stable over time. Specifically, patients on cemdisiran achieved a 46 percent (90 percent CI: 26, 60) placebo-adjusted reduction from baseline in spot UPCR at 32 weeks.
Furthermore, the results showed that cemdisiran was generally well tolerated in patients with IgAN with no adverse events (AEs) leading to treatment or study discontinuation during the double-blind treatment period. One death occurred in the cemdisiran arm due to cardiorespiratory collapse; this was not considered related to study drug by the study investigator. AEs reported by greater than or equal to 10 percent of patients in the cemdisiran arm were injection site reactions (41 percent) and peripheral edema (14 percent). There were no drug-related serious or severe AEs.
To view all results presented by Alnylam and collaborators at EMCHD please visit Capella.
About the Phase 2 Study
The Phase 2 trial is a randomized, double-blind, placebo-controlled multicenter study to evaluate the efficacy and safety of cemdisiran in adult patients with immunoglobulin A nephropathy (IgAN). Thirty-one adult patients (≥18 years and ≤ 65 years of age) with a clinical diagnosis of primary IgAN were randomized in a 2:1 cemdisiran to placebo ratio. The study was conducted in three periods. The first was an observational 14-week run-in period during which patients’ blood pressure, kidney function, degree of hematuria, and proteinuria were measured, and the standard of care remained unchanged. Patients did not receive study drug (cemdisiran or placebo) during this time. The second period was a 32-week treatment period, during which patients were dosed with 600 mg of cemdisiran or placebo every 4 weeks in combination with standard of care (angiotensin converting enzyme (ACE) inhibitors or angiotensin II receptor blockers (ARB)). The third period is a 52-week open-label extension (OLE) period to further evaluate the long-term safety and clinical activity of cemdisiran. During the OLE, all patients (including those initially on placebo) are treated with cemdisiran in combination with standard of care. The primary endpoint of the study is the percent change from baseline in 24-hour urine protein to creatinine ratio at week 32. Secondary endpoints include additional measures of proteinuria, changes in hematuria, percent of patients with partial clinical remission, and frequency of adverse events.
About Cemdisiran
Cemdisiran (ALN-CC5) is a subcutaneously administered, investigational RNAi therapeutic targeting the C5 component of the complement pathway. It is being developed in partnership with Regeneron Pharmaceuticals for the treatment of complement-mediated diseases. Cemdisiran utilizes Alnylam’s Enhanced Stabilization Chemistry (ESC)-GalNAc delivery platform.
About Alnylam Pharmaceuticals
Alnylam (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding 20 years ago, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam’s commercial RNAi therapeutic products are ONPATTRO® (patisiran), GIVLAARI® (givosiran), OXLUMO® (lumasiran), AMVUTTRA® (vutrisiran) and Leqvio® (inclisiran) being developed and commercialized by Alnylam’s partner, Novartis. Alnylam has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P5x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam, on LinkedIn, or on Instagram.
Source: Alnylam Pharmaceuticals, Inc.
Alnylam says trial of RNAi therapeutic cemdisiran achieves goals for IgA nephropathy
Aug. 29, 2022 11:18 AM ET
Alnylam Pharmaceuticals, Inc. (ALNY)
By: Jonathan Block, SA News Editor
Alnylam Pharmaceuticals (NASDAQ:ALNY) said that a phase 2 trial of its RNAi therapeutic cemdisiran for immunoglobulin A nephropathy achieved positive results.
- https://seekingalpha.com/news/3877481-alnylam-says-trial-of-rnai-therapeutic-cemdisiran-achieves-goals-for-iga-nephropathy
- https://seekingalpha.com/symbol/ALNY
- https://clinicaltrials.gov/ct2/show/NCT03841448
- https://www.alnylam.com/alnylam-rnai-pipeline
- https://www.alnylam.com/
BCX9930
ROCTAVIAN™ (valoctocogene roxaparvovec)
Cemdisiran (ALN-CC5)
BioCryst Presents Data Demonstrating >99 Percent Suppression of Alternative Pathway Complement Activity with BCX9930 in C3G Patients
Study represents first data with BCX9930 in C3G patients
RESEARCH TRIANGLE PARK, N.C., Aug. 26, 2022 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced that its oral Factor D inhibitor, BCX9930, demonstrated >99 percent suppression of the alternative pathway (AP), and that >98 percent suppression was maintained for 24 hours post-dosing, in patients with C3 glomerulopathy (C3G), a rare renal disease that is characterized by dysregulation of the AP of the complement system.
The data, which are the first data with BCX9930 in patients with C3G, are being presented in a poster session at the 18th European Meeting on Complement in Human Disease, which is being held in Bern, Switzerland, from August 26-29, 2022.
“The analyses from this study add to the body of evidence that show Factor D inhibition can correct dysregulation of the alternative pathway and has the potential to be a meaningful treatment approach for multiple complement-mediated diseases. The data are consistent using both commercially available assays and sophisticated proprietary assays our team has developed, which we can also utilize with our next-generation pipeline programs to quickly assess complement activation in vivo early in development,” said Dr. Yarlagadda S. Babu, chief discovery officer of BioCryst.
BioCryst EMCHD 2022 Presentation Highlights
BCX9930, an Oral Factor D Inhibitor, Suppresses Complement Alternative Pathway C3 Convertase Activity in vitro, and in Patients with Complement 3 Glomerulopathy (Poster #80); Sunday, August 28, 2022; 11:45am-1:30pm CET; Hintere Gasse, Building Fab8
- The study assessed the effects of oral BCX9930 on AP activity in ex vivo activated serum from healthy volunteers (n=16) and C3G patients (n=6) who participated in a phase 1, open-label study to evaluate the pharmacokinetics and pharmacodynamics of a single dose of BCX9930.
- One assay was used to assess the ability of BCX9930 to inhibit formation of AP C3 convertase in vitro (Elisa-detecting properdin (P)-bound C3 convertase), two assays were used to assess the ability of BCX9930 to inhibit AP complement activity ex vivo (AP Wieslab kit and multiplex assays), and another assay was used to assess the ability of BCX9930 to inhibit formation of AP C3 convertase ex vivo (immunofixation electrophoresis).
- The analyses found that a single oral dose (600 mg) of BCX9930:
- Demonstrated >99 percent (median) suppression of the AP, and that >98 percent (median) suppression was maintained for 24 hours post-dosing, in C3G patients.
- Achieved rapid (within two hours) and maximal (median >99 percent relative to pre-dose levels) suppression of the generation of C3a, a product of C3 convertase activity, in both healthy volunteers and C3G patients.
- Demonstrated >99 percent (median) suppression of the AP, and that >98 percent (median) suppression was maintained for 24 hours post-dosing, in C3G patients.
- These data support further development of Factor D inhibitors for the treatment of complement-mediated diseases, including C3G and other diseases driven by dysregulation of the AP.
In the study, a single dose of BCX9930 600 mg was generally safe and well-tolerated. Based on prior work with BCX9930, the proposed clinical dose of 400 mg (bid) provides a similar level of AP suppression as the 600 mg single dose used in this study in C3G patients.
Currently, BioCryst is conducting the RENEW proof-of-concept basket study evaluating BCX9930 in C3G, immunoglobulin A nephropathy (IgAN) and primary membranous nephropathy (PMN), all rare renal diseases caused by dysregulation of the alternative pathway.
BioCryst is also currently conducting the REDEEM-1 and REDEEM-2 pivotal trials evaluating BCX9930 in paroxysmal nocturnal hemoglobinuria (PNH).
About BioCryst Pharmaceuticals
BioCryst Pharmaceuticals discovers novel, oral, small-molecule medicines that treat rare diseases in which significant unmet medical needs exist and an enzyme plays a key role in the biological pathway of the disease. Oral, once-daily ORLADEYO® (berotralstat) is approved in the United States and multiple global markets. BioCryst has several ongoing development programs including BCX9930, an oral Factor D inhibitor for the treatment of complement-mediated diseases, BCX9250, an ALK-2 inhibitor for the treatment of fibrodysplasia ossificans progressiva, and galidesivir, a potential treatment for Marburg virus disease and yellow fever. RAPIVAB® (peramivir injection) is approved in the U.S. and multiple global markets, with post-marketing commitments ongoing. For more information, please visit the company’s website at www.biocryst.com.
BioCryst BCX9930 for rare kidney disorder shows promise in early-stage study
Aug. 26, 2022 7:44 AM ET
BioCryst Pharmaceuticals, Inc. (BCRX)
By: Ravikash, SA News Editor
BioCryst Pharmaceuticals (NASDAQ:BCRX) said that a single oral dose of its Factor D inhibitor BCX9930 showed >99% suppression of alternative pathway (AP) in a phase 1 trial in patients with C3 glomerulopathy (C3G).
https://seekingalpha.com/symbol/BCRX
Lifileucel
ROCTAVIAN™ (valoctocogene roxaparvovec)
ROCTAVIAN™ (valoctocogene roxaparvovec)
Iovance Biotherapeutics Initiates Biologics License Application (BLA) Submission for Lifileucel in Advanced Melanoma
First TIL Therapy BLA Submission Initiated with U.S. Food and Drug Administration
Complete BLA Submission on Track for Fourth Quarter 2022
SAN CARLOS, Calif., Aug. 25, 2022 (GLOBE NEWSWIRE) -- Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a late-stage biotechnology company developing novel T cell-based cancer immunotherapies, today announced that it has initiated a rolling Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA) for lifileucel, a tumor infiltrating lymphocyte (TIL) therapy, in patients with advanced (unresectable or metastatic) melanoma who progressed on or after prior anti-PD-1/L1 therapy, and if BRAF mutation positive, also prior BRAF or BRAF/MEK inhibitor therapy. There are no FDA approved therapies in this treatment setting.
Frederick Vogt, Ph.D., J.D., Interim President and Chief Executive Officer of Iovance, stated, “Initiating our rolling BLA submission for lifileucel is a significant step towards our goal to deliver the first individualized, one-time cell therapy for melanoma patients with significant unmet need. In parallel, we are executing our on-boarding and personnel training at authorized treatment centers, education and awareness initiatives, internal capacity planning, and launch readiness activities to prepare for commercialization. The FDA is supportive of our regulatory approach, and we look forward to continuing this collaboration throughout the submission and review process.”
A rolling BLA allows Iovance to submit portions of the BLA to the FDA on an ongoing basis, which enables the FDA to begin review as early as possible while documents are received. Iovance expects to complete the BLA submission in the fourth quarter of 2022. The rolling BLA submission and eligibility for priority review are benefits available under the FDA’s guidance on expedited programs for serious conditions. The FDA previously granted a regenerative medicine advanced therapy (RMAT) designation for lifileucel in advanced melanoma.
“Lifileucel represents hope and a new treatment for thousands of people with advanced melanoma who have very limited options after they progress on available standard of care,” said Kyleigh LiPira, CEO, Melanoma Research Foundation. “Cell immunotherapies are revolutionizing cancer treatment, and we are excited about the potential for the first FDA-approved TIL cell therapy for the treatment of melanoma, which helps us take another step towards finding a cure.”
The BLA submission for lifileucel is supported by positive clinical data from the C-144-01 clinical trial in patients with advanced melanoma. Iovance plans to present additional results from the C-144-01 trial at a medical meeting later this year.
About Iovance Biotherapeutics, Inc.
Iovance Biotherapeutics aims to be the global leader in innovating, developing and delivering tumor infiltrating lymphocyte (TIL) therapies for patients with cancer. We are pioneering a transformational approach to cure cancer by harnessing the human immune system’s ability to recognize and destroy diverse cancer cells in each patient. Our lead late-stage TIL product candidate, lifileucel for metastatic melanoma, has the potential to become the first approved one-time cell therapy for a solid tumor cancer. The Iovance TIL platform has demonstrated promising clinical data across multiple solid tumors. We are committed to continuous innovation in cell therapy, including gene-edited cell therapy, that may extend and improve life for patients with cancer. For more information, please visit www.iovance.com.
Source: Iovance Biotherapeutics, Inc.
Iovance submits rolling license application to FDA for skin cancer treatment lifileucel
Aug. 25, 2022 9:21 AM ET
Iovance Biotherapeutics, Inc. (IOVA)
By: Anuron Mitra, SA News Editor1 Comment
- Iovance Biotherapeutics (NASDAQ:IOVA) on Thursday said it had submitted a rolling biologics license application (BLA) to the U.S. FDA for its cancer therapy lifileucel for the treatment of advanced melanoma, the most serious type of skin cancer.
- IOVA said it expects to complete the BLA submission in Q4.
- https://seekingalpha.com/news/3876644-iovance-submits-rolling-license-application-to-fda-for-skin-cancer-treatment-lifileucel
- https://www.iovance.com/clinical-pipeline/
- https://www.iovance.com/
- https://seekingalpha.com/symbol/IOVA
ROCTAVIAN™ (valoctocogene roxaparvovec)
ROCTAVIAN™ (valoctocogene roxaparvovec)
ROCTAVIAN™ (valoctocogene roxaparvovec)
First Gene Therapy for Adults with Severe Hemophilia A, BioMarin's ROCTAVIAN™ (valoctocogene roxaparvovec), Approved by European Commission (EC)
Aug 24, 2022
Maintains Orphan Drug Designation (ODD) in the EU Providing 10-years of Market Exclusivity
Significant Benefit Over Existing Therapies for Patients with Severe Hemophilia A in EU Based on EMA Determination of ODD
Conference Call and Webcast to be Held Wed., Aug. 24th at 8:00 pm Eastern
SAN RAFAEL, Calif., Aug. 24, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that the European Commission (EC) has granted conditional marketing authorization (CMA) to ROCTAVIAN™ (valoctocogene roxaparvovec) gene therapy for the treatment of severe hemophilia A (congenital Factor VIII deficiency) in adult patients without a history of Factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5). The EC also endorsed EMA's recommendation for Roctavian to maintain orphan drug designation, thereby granting a 10-year period of market exclusivity. The EMA recommendation noted that, even in light of existing treatments, Roctavian may potentially offer a significant benefit to those affected with severe Hemophilia A. The one-time infusion is the first approved gene therapy for hemophilia A and works by delivering a functional gene that is designed to enable the body to produce Factor VIII on its own without the need for continued hemophilia prophylaxis, thus relieving patients of their treatment burden relative to currently available therapies. People with hemophilia A have a mutation in the gene responsible for producing Factor VIII, a protein necessary for blood clotting.
It is estimated that more than 20,000 adults are affected by severe hemophilia A across more than 70 countries in Europe, the Middle East, and Africa. Of the 8,000 adults with severe hemophilia A in the 24 countries within BioMarin's footprint covered by today's EMA approval, there are an estimated 3,200 patients who will be indicated for Roctavian. BioMarin anticipates additional access to ROCTAVIAN™ for patients outside of the EU through named patient sales based on the European Medicines Agency (EMA) approval in countries in the Middle East, Africa and Latin America and expects additional market registrations to be facilitated by the EMA license.
"This approval in the EU represents a medical breakthrough in the treatment of patients with severe hemophilia A that expands the conversation between a patient and physician on treatment choices to now include a one-time infusion that protects from bleeds for several years," said Professor Johannes Oldenburg, Director of the Institute of Experimental Haematology and Transfusion Medicine and the Haemophilia Centre at the University Clinic in Bonn, Germany. "It is exciting to imagine the possibilities of this approved gene therapy, which has demonstrated a substantial and sustained reduction in bleeding for patients, who potentially could be freed from the burden of regular infusions."
"Roctavian approval in Europe is a historic milestone in medicine and is built upon almost four decades of scientific discovery, innovation, and perseverance. We thank the European Commission for recognizing Roctavian's value as the first gene therapy for hemophilia A, a feat that we believe will transform how healthcare professionals and the patient community think about caring for bleeding disorders," said Jean-Jacques Bienaimé, Chairman and Chief Executive Officer of BioMarin. "We are grateful to the patients, investigators and community, who dedicated their time and effort to this achievement and whose aspirations provided the driving force behind making this one-time therapy a reality."
The EC based its decision on a significant body of data from the Roctavian clinical development program, the most extensively studied gene therapy for hemophilia A, including two-year outcomes from the global GENEr8-1 Phase 3 study. The GENEr8-1 Phase 3 study demonstrated stable and durable bleed control, including a reduction in the mean annualized bleeding rate (ABR) and the mean annualized Factor VIII infusion rate. In addition, the data included five and four years of follow-up from the 6e13 vg/kg and 4e13 vg/kg dose cohorts, respectively, in the ongoing Phase 1/2 dose escalation study. BioMarin has committed to continue working with the broader community and the EMA to monitor the long-term effects of treatment. The Product Information will be available shortly on the EMA website under the Medicines tab. Search for "ROCTAVIAN" and select "Human medicine European public assessment report (EPAR): Roctavian. Then select "Product Information" in the Table of Contents and then select "Roctavian: EPAR – Product Information."
A Conditional Marketing Authorization (CMA) recognizes that the medicine fulfils an unmet medical need based on a positive benefit-risk assessment, and that the benefit to public health of the immediate availability on the market outweighs the uncertainties inherent to the fact that additional data are still required. BioMarin will provide further data from ongoing studies within defined timelines to confirm that the benefits continue to outweigh the risks, building on what already constitutes the largest clinical data package for gene therapy in hemophilia A. Conversion to a standard marketing authorization will be contingent on the provision of additional data from currently ongoing Roctavian clinical studies, including longer-term follow up of patients enrolled in the pivotal trial GENEr8-1, as well as a study investigating efficacy and safety of ROCTAVIAN with prophylactic use of corticosteroids (Study 270-303), for which enrollment is now complete.
Orphan drug designation is reserved for medicines treating rare (affecting not more than five in 10,000 people in the EU), life-threatening or chronically debilitating diseases. Authorized orphan medicines benefit from ten years of market exclusivity, protecting them from competition with similar medicines with the same therapeutic indication, which cannot be marketed during the exclusivity period.
BioMarin remains committed to bringing Roctavian to eligible patients with severe hemophilia A in the United States and is targeting a Biologics License Application (BLA) resubmission for Roctavian by the end of September 2022. Typically, BLA resubmissions are followed by a six-month review procedure. However, the Company anticipates three additional months of review may be necessary based on the number of data read-outs that will emerge during the procedure.
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 also conducting a Phase 3B, single arm, open-label study to evaluate the efficacy and safety of Roctavian at a dose of 6e13 vg/kg with prophylactic corticosteroids in people with hemophilia A (Study 270-303). Also ongoing are a Phase 1/2 Study with the 6e13 vg/kg dose of Roctavian in people with hemophilia A with pre-existing AAV5 antibodies (Study 270-203) and aa Phase 1/2 Study with the 6e13 vg/kg dose of Roctavian in people with hemophilia A with active or prior Factor VIII inhibitors (Study 270-205).
About BioMarin
BioMarin is a global biotechnology company that develops and commercializes innovative therapies for people with serious and life-threatening genetic diseases and medical conditions. The Company selects product candidates for diseases and conditions that represent a significant unmet medical need, have well-understood biology and provide an opportunity to be first-to-market or offer a significant benefit over existing products. The Company's portfolio consists of eight commercial products and multiple clinical and preclinical product candidates for the treatment of various diseases. For additional information, please visit www.biomarin.com.
SOURCE BioMarin Pharmaceutical Inc.
BioMarin to resubmit hemophilia therapy for FDA approval after EU nod
Aug. 25, 2022 6:53 AM ET
BioMarin Pharmaceutical Inc. (BMRN)
By: Dulan Lokuwithana, SA News Editor6 Comments
- Announcing the EU approval of Roctavian for hemophilia A, BioMarin Pharmaceutical (NASDAQ:BMRN) disclosed its plans to resubmit the U.S. marketing application for the gene therapy to the FDA by the end of next month.
- https://seekingalpha.com/news/3876536-bmrn-stock-watch-plans-to-resubmit-hemophilia-therapy-for-fda-approval
- https://seekingalpha.com/symbol/BMRN
- https://www.biomarin.com/our-treatments/pipeline/valoctocogene-roxaparvovec-for-severe-hemophilia-a/
MYTESI® (crofelemer) delayed-release tablets
FUJOVEE™ (Abivertinib) (Cytokine Storm – STI 5656)
FUJOVEE™ (Abivertinib) (Cytokine Storm – STI 5656)
Jaguar Health Announces that Orphan Drug Designation Application Submitted to the FDA for Crofelemer for a Severe Congenital Diarrheal Disorder (CDD) Has Been Accepted for Review
Microvillus Inclusion Disease (MVID), a CDD, Is a Life-Threatening and Rare Autosomal Recessive Disease That Affects Newborns and Children and Leads to Significant Morbidity and Mortality From Severe Secretory Diarrhea
Replay Link for Jaguar's August 22, 2022 Investor Webcast Can Be Accessed by Clicking Here; Q2 2022 Represented the Fourth Consecutive Quarter of Growth in Mytesi® Net Revenue
SAN FRANCISCO, CA / ACCESSWIRE / August 23, 2022 / Jaguar Health (NASDAQ:JAGX) today announced that its Orphan Drug Designation (ODD) application for crofelemer submitted to the U.S. Food and Drug Administration for a rare congenital diarrheal disorder called microvillus inclusion disease (MVID) has been accepted by the FDA for review. A response from the FDA on the application is expected by the end of September 2022.
Jaguar today also provided the replay link for the company's August 22, 2022 investor webcast, which can be accessed by clicking here.
"Although our message may have been lost with the overall market turndown yesterday, we were very pleased to announce that our prescription product net revenue was approximately $2.9 million in the second quarter of 2022, representing an increase of approximately 12% over Mytesi net revenue in the first quarter of 2022, which totaled approximately $2.6 million, and an increase of approximately 641% over Mytesi net revenue in the second quarter of 2021, which totaled approximately $0.4 million," said Lisa Conte, Jaguar's president and CEO. "This is the FOURTH consecutive quarter of growth in Mytesi net revenue - which largely represents the funds collected after various insurance and government chargebacks. The continued improvement in net revenue - a key reportable financial measure of commercial performance under GAAP (generally accepted accounting principles) - is largely a result of the efficiencies realized by the transition we completed this past January to a closed network of specialty pharmacies - an initiative that resulted in a meaningful reduction in Mytesi distribution costs as well as a higher average net price."
As announced, Napo Therapeutics, the Italian corporation Jaguar established in Italy in 2021 that focuses on expanding crofelemer access in Europe with an initial focus on orphan designated diseases, submitted an ODD application for crofelemer this past May for MVID to the European Medicines Agency (EMA).
"CDDs are a group of inherited chronic enteropathies characterized by heterogeneous etiology, and each type of CDD is thus a different disease with a different pathogenetic mechanism," said Martire Particco, MD, Chief Medical Officer of Napo Therapeutics. "MVID is a life-threatening and rare autosomal recessive disease that affects newborns and children and leads to significant morbidity and even death from severe secretory diarrhea."
CDDs share a primary common symptom: chronic diarrhea, and therefore secondary symptoms associated with diarrhea, including significant dehydration, metabolic acidosis or alkalosis and malnutrition, among other secondary symptoms, and these symptoms expeditiously emerge and become life-threatening.
Napo Therapeutics' mission is to provide access to Jaguar's proprietary first-in-class plant-based medicine crofelemer in Europe to address such significant rare disease indications. Under its license to crofelemer from Jaguar, Napo Therapeutics' initial focus is on clinical development and future registration in Europe of crofelemer for debilitating orphan disease target indications, starting with short bowel syndrome (SBS) and CDD.
Crofelemer has received ODD from the EMA and the FDA for SBS, which is a complex condition characterized by severe malabsorption of fluids and nutrients due to surgical resection of bowel segments, congenital anomalies, or disease-associated loss of absorption. For SBS patients who endure the catastrophic loss of their bowel, the resulting excessive intestinal fluid output and lifelong restriction and adjustment of oral intake of food and liquids leads to the requirement to receive intravenous fluids for most of every day (parenteral nutrition). This challenges their ability to carry out activities of daily living, or to attend school or work, and has a significant impact on their daily quality of life. Furthermore, lifelong parenteral nutrition leads to potentially life-threatening complications like sepsis and organ failure. SBS affects approximately 10,000 to 20,000 people in the United States1, according to the Crohn's & Colitis Foundation, and it is estimated that the population of SBS patients in Europe is approximately the same size.2
Jaguar anticipates the completion in 2022 of an investigator-initiated proof-of-concept study of crofelemer for SBS, supporting the potential for expanded patient access to crofelemer in Europe in 2023 for this disease. The company has approved this planned trial of crofelemer for SBS, and the third-party investigator is targeting the presentation in December 2022 of results from the SBS study at a global GI conference in Dubai.
About Crofelemer
Crofelemer is a botanical (plant-based) drug extracted and purified from the red bark sap, also referred to as "dragon's blood," of the medicinal Croton lechleri tree in the Amazon Rainforest. Napo Pharmaceuticals, Jaguar Health's wholly owned U.S. subsidiary, has established a sustainable harvesting program, under fair trade practices, for crofelemer to ensure a high degree of quality, ecological integrity, and support for Indigenous communities.
About Jaguar Health, Jaguar Animal Health, Napo Pharmaceuticals, & Napo Therapeutics
Jaguar Health, Inc. is a commercial stage pharmaceuticals company focused on developing novel, plant-based, non-opioid, and sustainably derived prescription medicines for people and animals with GI distress, including chronic, debilitating diarrhea. Jaguar Animal Health is a tradename of Jaguar Health. Jaguar Health's wholly owned subsidiary, Napo Pharmaceuticals, Inc., focuses on developing and commercializing proprietary plant-based human pharmaceuticals from plants harvested responsibly from rainforest areas. Jaguar Health is the majority shareholder of Napo Therapeutics S.p.A. (f/k/a Napo EU S.p.A.), an Italian corporation established by Jaguar Health in Milan, Italy in 2021 that focuses on expanding crofelemer access in Europe.
For more information about Jaguar Health, please visit https://jaguar.health. For more information about Napo Pharmaceuticals, visit www.napopharma.com.
SOURCE: Jaguar Health, Inc.
accesswire.com
https://www.accesswire.com/713157/Jaguar-Health-Announces-that-Orphan-Drug-Designation-Application-Submitted-to-the-FDA-for-Crofelemer-for-a-Severe-Congenital-Diarrheal-Disorder-CDD-Has-Been-Accepted-for-Review
Jaguar's diarrhea disorder therapy gets FDA review for orphan drug status
Aug. 23, 2022 9:42 AM ET
By: Ravikash, SA News Editor
- The U.S. Food and Drug Administration (FDA) accepted to review Jaguar Health's (NASDAQ:JAGX) application seeking orphan drug designation (ODD) for crofelemer to treat microvillus inclusion disease (MVID).
- https://seekingalpha.com/news/3875810-jaguars-diarrhea-disorder-therapy-gets-fda-review-for-orphan-drug-status
- https://seekingalpha.com/symbol/JAGX
- https://mytesi.com/
FUJOVEE™ (Abivertinib) (Cytokine Storm – STI 5656)
FUJOVEE™ (Abivertinib) (Cytokine Storm – STI 5656)
FUJOVEE™ (Abivertinib) (Cytokine Storm – STI 5656)
Sorrento Announces Significant Positive Pivotal Trial Results as Assessed by an Independent Review Committee (IRC) With Matured Long-Term (Over Three Years) Follow-Up Data of Abivertinib for the Treatment of Advanced Non-Small Cell Lung Cancer (NSCLC)
August 23, 2022 at 9:00 AM EDTDownload PDF
- Abivertinib is a novel third-generation epidermal growth factor receptor (EGFR) inhibitor that irreversibly targets mutant forms of EGFR in advanced NSCLC patients who are resistant to first-line EGFR kinase inhibitor therapies.
- In this pivotal study conducted in China, matured data of 209 response evaluable NSCLC patients were assessed by an IRC. The overall response rate (ORR) as confirmed by the IRC was 56.5% (118/209) and among them, 11 patients had complete responses (CR) with a CR rate of 5.3% (11/209) and median overall survival (OS) of 28.2 months.
- Based on these significant positive results from the IRC assessment, Sorrento is closing the study and preparing the materials for a pre-New Drug Application (NDA) meeting with the FDA and potentially submitting for approvals to regulatory agencies of other countries.
SAN DIEGO, Aug. 23, 2022 (GLOBE NEWSWIRE) -- Sorrento Therapeutics, Inc. (Nasdaq: SRNE, "Sorrento") today announced positive results from a pivotal study of Abivertinib on 209 response evaluable, heavily pretreated NSCLC patients by an IRC assessment with matured long-term follow up data.
Abivertinib is a pyrrolopyrimidine-based, third-generation EGFR inhibitor, which is structurally distinct from osimertinib. Abivertinib selectively inhibits EGFR-activating and resistant mutation with nearly 300-fold greater potency as compared with wild-type EGFR. Previously, a positive interim result assessed by investigators was published in the peer-reviewed Clinical Cancer Research journal with interim data (https://clincancerres.aacrjournals.org/content/early/2021/11/04/1078-0432.CCR-21-2595). In these IRC-assessed preliminary topline results with more matured long-term follow up data (previously 16.8 months, now 38.8 months), Abivertinib showed significant treatment benefits in 209 response evaluable, heavily treated NSCLC patients with ORR of 56.5%, and notably a significant CR rate was seen with Abivertinib (5.3%) in comparison with that of osimertinib (Tagrisso) (0.5%)*, while the ORR rate is comparable between the two drugs. This combined data, the ORR of 56.5%, the CR rate of 5.3%, and median OS of 28.2 months (versus Tagrisso’s median OS of 26.8 months)**, is potentially superior to that of the approved third generation EGFR inhibitor (osimertinib). Abivertinib demonstrated significantly efficacious effects in overcoming the resistant mutation in NSCLC. Based on these results, Sorrento is closing the study and preparing the pre-NDA materials and NDA package. Sorrento will request a pre-NDA meeting with the FDA and other regulatory agencies around the world with potential NDA filings pending agreements with the regulatory agencies in different countries.
“We are very encouraged by the significant positive results of Abivertinib assessed by the IRC with long-term follow up data and look forward to meeting with the FDA and other regulatory authorities for the possibility of bringing Abivertinib to the U.S. and global markets,” said Dr. Henry Ji, Chairman and CEO of Sorrento. Abivertinib is one of the multiple clinical and pre-clinical stage assets obtained by Sorrento in the previously completed acquisition of ACEA Therapeutics, Inc. in June of 2021.
*https://www.accessdata.fda.gov/drugsatfda_docs/nda/2015/208065Orig1s000MedR.pdf
** https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/208065s025lbl.pdf
About Sorrento Therapeutics, Inc.
Sorrento is a clinical and commercial stage biopharmaceutical company developing new therapies to treat cancer, pain (non-opioid treatments), autoimmune disease and COVID-19. Sorrento's multimodal, multipronged approach to fighting cancer is made possible by its extensive immuno-oncology platforms, including key assets such as next-generation tyrosine kinase inhibitors (TKIs), fully human antibodies (“G-MAB™ library”), immuno-cellular therapies (“DAR-T™”), antibody-drug conjugates (“ADCs”), and oncolytic virus (“Seprehvec™”). Sorrento is also developing potential antiviral therapies and vaccines against coronaviruses, including STI-1558, COVISHIELD™ and COVIDROPS™; and diagnostic test solutions, including COVIMARK™.
Sorrento's commitment to life-enhancing therapies for patients is also demonstrated by our effort to advance a first-in-class (TRPV1 agonist) non-opioid pain management small molecule, resiniferatoxin (“RTX”), and SP-102 (10 mg, dexamethasone sodium phosphate viscous gel) (SEMDEXA™), a novel, viscous gel formulation of a widely used corticosteroid for epidural injections to treat lumbosacral radicular pain, or sciatica, and to commercialize ZTlido® (lidocaine topical system) 1.8% for the treatment of postherpetic neuralgia (PHN). RTX has been cleared for a Phase II trial for intractable pain associated with cancer and a Phase II trial in osteoarthritis patients. Positive final results from the Phase III Pivotal Trial C.L.E.A.R. Program for SEMDEXA™, its novel, non-opioid product for the treatment of lumbosacral radicular pain (sciatica), were announced in March 2022. ZTlido® was approved by the FDA on February 28, 2018.
For more information visit www.sorrentotherapeutics.com
Source: Sorrento Therapeutics, Inc.
Sorrento achieves positive results in pivotal trial for abivertinib in NSCLC
Aug. 23, 2022 10:45 AM ET
Sorrento Therapeutics, Inc. (SRNE)
By: Jonathan Block, SA News Editor
- Sorrento Therapeutics (NASDAQ:SRNE) said that a pivotal study of its candidate abivertinib in non-small cell lung cancer demonstrated an overall response rate of 56.5%.
- https://seekingalpha.com/news/3875837-sorrento-achieves-positive-results-in-pivotal-trial-for-abivertinib-in-nsclc
- https://seekingalpha.com/symbol/SRNE
- https://sorrentotherapeutics.com/research/immuno-oncology/abivertinib/
- https://sorrentotherapeutics.com/
Quizartinib
FUJOVEE™ (Abivertinib) (Cytokine Storm – STI 5656)
CagriSema (2.4 mg semaglutide and 2.4 mg cagrilintide)
Quizartinib Marketing Authorization Application Validated by EMA for Treatment of Adult Patients with Newly Diagnosed FLT3- ITD Positive Acute Myeloid Leukemia • Submission based on QuANTUM-First results showing quizartinib plus chemotherapy significantly improved overall survival compared to chemotherapy alone Tokyo & Munich –August 23, 2022 – Daiichi Sankyo (TSE: 4568) today announced that the European Medicines Agency (EMA) has validated the marketing authorization application (MAA) for quizartinib in combination with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy, and as continuation monotherapy following consolidation, for the treatment of adult patients with newly diagnosed acute myeloid leukemia (AML) that is FMS-like tyrosine kinase 3 internal tandem duplication (FLT3-ITD) positive. AML is one of the most common forms of leukemia in adults, representing about one-third of all cases. 1 In Europe, approximately 18,000 people are diagnosed with AML each year and the five-year survival rate is reported at 17% for adult patients. 2,3 Of all newly diagnosed cases of AML, approximately 25% carry the FLT3-ITD gene mutation, which is associated with a particularly unfavorable prognosis including increased risk of relapse and shorter overall survival.4 “There is a need to improve survival for the majority of patients with acute myeloid leukemia, particularly those with the FLT3-ITD subtype, which is aggressive and difficult to treat,” said Ken Takeshita, MD, Global Head, R&D, Daiichi Sankyo. “We look forward to working with the EMA to support their review of quizartinib as a potential option for patients with newly diagnosed FLT3-ITD positive acute myeloid leukemia.” Validation confirms that the application is complete and commences the scientific review process by the EMA’s Committee for Medicinal Products for Human Use (CHMP). The application is based on data from the QuANTUM-First phase 3 trial recently presented at the European Hematology Association (#EHA2022) Congress. In QuANTUM-First, quizartinib combined with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy, and continued as monotherapy following consolidation, demonstrated a statistically significant and clinically meaningful improvement in overall survival (OS) in adult patients with newly diagnosed FLT3-ITD positive AML 2 compared to chemotherapy alone. The safety of quizartinib combined with intensive chemotherapy and as continuation monotherapy in QuANTUM-First was generally manageable and consistent with previous clinical trials. The incidence of Grade ≥3 QT prolongation was low, with uncommon ventricular arrythmia events. Overall, the risk of QT prolongation was manageable with ECG monitoring, quizartinib dose modification and correction/elimination of additional risk factors. About QuANTUM-First QuANTUM-First is a randomized, double-blind, placebo-controlled global phase 3 study evaluating quizartinib in combination with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy, and as continuation monotherapy following consolidation, in adult patients aged 18-75 with newly diagnosed FLT3-ITD positive AML. Patients were randomized 1:1 into two treatment groups to receive quizartinib or placebo combined with anthracycline- and cytarabine-based regimens. Eligible patients, including those who underwent hematopoietic stem cell transplant (HSCT), continued with quizartinib or placebo for up to 36 cycles. The primary study endpoint was OS. Secondary endpoints include event-free survival (EFS), postinduction rates of complete remission (CR) and composite complete remission (CRc), and the percentage of patients who achieve CR or CRc with FLT3-ITD minimal residual disease negativity. Safety and pharmacokinetics, along with exploratory efficacy and biomarker endpoints, also were evaluated. QuANTUM-First enrolled 539 patients at 193 study sites across Asia, Europe, North America, Oceania and South America. For more information, visit Clinicaltrialsregister.eu or ClinicalTrials.gov.
About FLT3-ITD FLT3 (FMS-like tyrosine kinase 3) is a tyrosine kinase receptor protein normally expressed by hematopoietic stem cells that plays an important role in cell development, promoting cell survival, growth and differentiation through various signaling pathways. 4 Mutations of the FLT3 gene, which occur in approximately 30% of AML patients, can drive oncogenic signaling.4 FLT3-ITD (internal tandem 3 duplication) is the most common type of FLT3 mutation in AML, occurring in about 25% of all newly diagnosed patients, and is associated with increased risk of relapse and shorter overall survival.4 About Quizartinib Quizartinib is an oral, selective type II FLT3 inhibitor currently in clinical development for treatment of FLT3-ITD positive AML. In addition to QuANTUM-First, the quizartinib development program includes a phase 1/2 trial in pediatric and young adult patients with relapsed/ refractory FLT3-ITD positive AML in Europe and North America. Several phase 1/2 combination studies with quizartinib are also underway at The University of Texas MD Anderson Cancer Center as part of a strategic research collaboration focused on accelerating development of Daiichi Sankyo pipeline therapies for AML. Quizartinib, which is currently not approved in Europe, has been granted Orphan Drug Designation for the treatment of AML in Europe, Japan and the U.S. Quizartinib has received Fast Track Designation from the U.S. Food and Drug Administration for the treatment of adult patients with newly diagnosed AML that is FLT3-ITD positive, in combination with standard cytarabine and anthracycline induction and cytarabine consolidation chemotherapy. Quizartinib is currently approved for use in Japan for the treatment of adult patients with relapsed/refractory FLT3-ITD AML, as detected by an approved test. Quizartinib is an investigational medicine in all countries outside of Japan. About Daiichi Sankyo Daiichi Sankyo is dedicated to creating new modalities and innovative medicines by leveraging our world-class science and technology for our purpose “to contribute to the enrichment of quality of life around the world.” In addition to our current portfolio of medicines for cancer and cardiovascular disease, Daiichi Sankyo is primarily focused on developing novel therapies for people with cancer as well as other diseases with high unmet medical needs. With more than 100 years of scientific expertise and a presence in more than 20 countries, Daiichi Sankyo and its 16,000 employees around the world draw upon a rich legacy of innovation to realize our 2030 Vision to become an “Innovative Global Healthcare Company Contributing to the Sustainable Development of Society.” For more information, please visit www.daiichisankyo.com.
Daiichi Sankyo's quizartinib gets EMA review to treat blood cancer subtype
Aug. 23, 2022 4:48 AM ET
Daiichi Sankyo Company, Limited (DSNKY), DSKYF
By: Ravikash, SA News Editor
- The European Medicines Agency (EMA) validated Daiichi Sankyo's (OTCPK:DSKYF) (OTCPK:DSNKY) application seeking approval of quizartinib in combination with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy, and as continuation standalone therapy after consolidation, to treat adult patients with newly diagnosed acute myeloid leukemia (AML) which is FMS-like tyrosine kinase 3 internal tandem duplication (FLT3-ITD) positive.
- https://seekingalpha.com/news/3875634-daiichi-sankyos-quizartinib-gets-ema-review-to-treat-blood-cancer-subtype
- https://seekingalpha.com/symbol/DSKYF
- https://seekingalpha.com/symbol/DSNKY
- https://www.daiichisankyo.com/
CagriSema (2.4 mg semaglutide and 2.4 mg cagrilintide)
CagriSema (2.4 mg semaglutide and 2.4 mg cagrilintide)
CagriSema (2.4 mg semaglutide and 2.4 mg cagrilintide)
07:49
22 August 2022
Novo Nordisk successfully completes phase 2 trial with CagriSema in people with type 2 diabetes
Bagsværd, Denmark ,
2 2 August 2022 – Novo Nordisk today announced headline results from a phase 2 clinical trial with CagriSema, a once-weekly subcutaneous combination of semaglutide and a novel amylin analogue, cagrilintide.
The trial investigated the efficacy and safety of a fixed dose combination of CagriSema (2.4 mg semaglutide and 2.4 mg cagrilintide) compared to the individual components semaglutide 2.4 mg and cagrilintide 2.4 mg, all administered once weekly, in 92 people with type 2 diabetes and overweight. People were equally randomised among the three treatment arms. In the trial, the mean baseline HbA1c was 8.4% and the mean baseline body weight was 106 kg.
After 32 weeks of treatment, people treated with CagriSema achieved a numerically higher HbA1c reduction of 2.18%-points compared to a reduction of 1.79%-points for people treated with semaglutide and 0.93%-points with cagrilintide alone. People treated with CagriSema achieved a numerically higher body weight reduction of 15.6% compared to a reduction of 5.1% for people treated with semaglutide and 8.1% with cagrilintide alone1. In the trial, CagriSema appeared to have a safe and well-tolerated profile.
“We are encouraged by the impressive phase 2 results for CagriSema in people with type 2 diabetes,” said Martin Holst Lange, executive vice president for Development at Novo Nordisk. “The results indicate that CagriSema reduces blood sugar more than semaglutide alone and the weight loss seen in the trial confirms the substantial weight lowering potential of CagriSema”.
Based on the results, Novo Nordisk is now planning to initiate a phase 3 development programme for CagriSema in people with type 2 diabetes in 2023. The CagriSema (2.4 mg semaglutide and 2.4 mg cagrilintide) phase 3 programme in people with overweight and obesity, REDEFINE, is expected to begin in the fourth quarter of 2022.
About Novo Nordisk
Novo Nordisk is a leading global healthcare company, founded in 1923 and headquartered in Denmark. Our purpose is to drive change to defeat diabetes and other serious chronic diseases such as obesity and rare blood and endocrine disorders. We do so by pioneering scientific breakthroughs, expanding access to our medicines and working to prevent and ultimately cure disease. Novo Nordisk employs about 50 , 8 00 people in 80 countries and markets its products in around 170 countries. Novo Nordisk's B shares are listed on Nasdaq Copenhagen (Novo-B). Its ADRs are listed on the New York Stock Exchange (NVO) . For more information, visit novonordisk.com , Facebook, Twitter , LinkedIn and YouTube.
Novo Nordisk CagriSema helps reduce blood sugar, weight in diabetics/overweight in phase 2 trial
Aug. 23, 2022 5:32 AM ET
By: Ravikash, SA News Editor
Novo Nordisk (NVO) said its medicine CagriSema was better at reducing blood sugar levels and weight compared to its other diabetes drug semaglutide and a novel amylin analogue, cagrilintide, in a phase 2 trial in people with type 2 diabetes and overweight.
https://seekingalpha.com/symbol/NVO
ZYNTEGLO® (betibeglogene autotemcel) or beti-cel
CagriSema (2.4 mg semaglutide and 2.4 mg cagrilintide)
ZYNTEGLO® (betibeglogene autotemcel) or beti-cel
bluebird bio Announces FDA Approval of ZYNTEGLO®, the First Gene Therapy for People with Beta-Thalassemia Who Require Regular Red Blood Cell Transfusions
ZYNTEGLO offers potentially curative benefit across ages and genotypes, through the achievement of durable transfusion independence and normal or near normal total hemoglobin levels
Management team to host conference call Thursday, August 18 at 8:00 am ET
SOMERVILLE, Mass.--(BUSINESS WIRE)--Aug. 17, 2022-- bluebird bio, Inc. (Nasdaq: BLUE) today announced the U.S. Food and Drug Administration (FDA) has approved ZYNTEGLO® (betibeglogene autotemcel), also known as beti-cel, a one-time gene therapy custom-designed to treat the underlying genetic cause of beta‑thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions.
“The FDA approval of ZYNTEGLO offers people with beta-thalassemia the possibility of freedom from burdensome regular red blood cell transfusions and iron chelation, and unlocks new possibilities in their daily lives,” said Andrew Obenshain, chief executive officer, bluebird bio. “After more than a decade of research and clinical development, and through the perseverance of clinicians, patients, and their families, the approval of ZYNTEGLO marks a watershed moment for the field of gene therapy. As the first ex-vivo lentiviral vector gene therapy approved in the U.S. for the treatment of people with beta-thalassemia, we are ushering in a new era in which gene therapy has the potential to transform existing treatment paradigms for diseases that currently carry a lifelong burden of care.”
Beta-thalassemia is a rare, genetic blood disease caused by mutations in the beta-globin gene and characterized by significantly reduced or absent adult hemoglobin production. Patients with the most severe form, sometimes called transfusion-dependent beta-thalassemia or beta-thalassemia major, experience severe anemia and lifelong dependence on regular red blood cell transfusions, a lengthy process that patients typically undergo every 2-5 weeks. Despite advances in treatment and improved transfusion techniques, transfusions only temporarily address symptoms of anemia and people with beta‑thalassemia who require regular transfusions have an increased risk for morbidity and mortality due to complications from treatment-related iron overload. Data from the Cooley’s Anemia Foundation indicate that the median age of death of patients with transfusion-dependent beta‑thalassemia in the U.S. who died during the last decade was just 37 years. bluebird estimates that there are approximately 1,300-1,500 individuals with transfusion-dependent beta-thalassemia in the U.S.
“Transfusion-dependent beta-thalassemia is associated with an intense treatment burden and significant health risks related to regular red blood transfusions and iron management,” said Alexis A. Thompson, MD, MPH, Chief of the Division of Hematology, Children's Hospital of Philadelphia. “As a clinician and an investigator in the ZYNTEGLO clinical development program, I celebrate the therapeutic potential of this treatment for patients and its implications for the field of gene therapy, all made possible through the incredible courage of patients and families who participated in the clinical trials.”
“The Cooley’s Anemia Foundation applauds the FDA’s approval of ZYNTEGLO for people with beta‑thalassemia who require regular red blood cell transfusions. The availability of a one-time gene therapy which offers the possibility of transfusion independence opens up new and exciting opportunities for those who are medically eligible to receive this treatment option,” said Craig Butler, National Executive Director, Cooley’s Anemia Foundation. “While advances in treatment have been of enormous benefit to those with beta-thalassemia, a potentially curative therapy may offer a true life-changing experience.”
The approval of ZYNTEGLO is the culmination of nearly 10 years of clinical research of gene therapy in patients with transfusion-dependent beta-thalassemia. ZYNTEGLO works by adding functional copies of a modified form of the beta-globin gene (βA-T87Q-globin gene) into a patient’s own hematopoietic (blood) stem cells (HSCs) to allow them to make normal to near normal levels of total hemoglobin without regular RBC transfusions. The functional beta-globin gene is added into a patient’s cells outside of the body (ex-vivo), and then infused into the patient. Though ZYNTEGLO is designed to be administered to the patient once, the treatment process is comprised of several steps that may take place over the course of several months.
Due to the complex nature of gene therapy, ZYNTEGLO will be available exclusively at Qualified Treatment Centers (QTCs) which are carefully selected based on their expertise in relevant areas such as stem cell transplantation, cell and gene therapy, and beta-thalassemia; and receive specialized training to administer ZYNTEGLO. Information on bluebird’s QTC network, as well as personalized support focused on the needs of each patient throughout their treatment journey and information on insurance coverage and access will be available through bluebird’s patient support program, my bluebird support. Patients can call 833-888-NEST (833-888-6378) for more information, and additional details will be available at mybluebirdsupport.com in the coming days.
ZYNTEGLO was reviewed under Priority Review, and the Company received a Priority Review voucher upon approval. ZYNTEGLO was previously granted Orphan Drug designation and Breakthrough Therapy designation.
Clinical Data Supporting Approval of ZYNTEGLO
bluebird bio has the longest and most robust clinical program in transfusion-dependent beta‑thalassemia (TDT) in the field of gene therapy. The approval of ZYNTEGLO is based on data from bluebird bio’s Phase 3 studies HGB-207 (Northstar-2) and HGB-212 (Northstar-3), and the long-term follow-up study LTF-303.
The single-arm, open-label, 24-month Phase 3 studies of ZYNTEGLO included 41 patients aged 4 to 34 years with both non-β0/β0 and β0/β0 genotypes, with longest follow up out to 4 years. Eighty-nine percent (32/36) of evaluable patients across ages and genotypes achieved transfusion independence (TI), which is defined as no longer needing red blood cell transfusions for at least 12 months while maintaining a weighted average total hemoglobin of at least 9 g/dL. Results in these patients were durable as of last follow-up.
The most common non-laboratory adverse reactions (≥20%) were mucositis, febrile neutropenia, vomiting, pyrexia, alopecia, epistaxis, abdominal pain, musculoskeletal pain, cough, headache, diarrhea, rash, constipation, nausea, decreased appetite, pigmentation disorder, and pruritus. The most common Grade 3 or 4 laboratory abnormalities (>50%) include neutropenia, thrombocytopenia, leukopenia, anemia, and lymphopenia.
Enrollment is complete and all patients have been treated in the Phase 3 Northstar-2 (HGB-207) and Northstar-3 (HGB-212) studies evaluating ZYNTEGLO. Follow-up in HGB-212 is ongoing. bluebird bio is also conducting a long-term follow-up study, LTF-303, to monitor safety and efficacy for patients with TDT who have participated in bluebird bio-sponsored clinical studies of lentiviral vector (LVV) gene therapy through 15 years post-treatment.
Across all studies, all patients who achieved transfusion independence have remained transfusion-free.
About ZYNTEGLO® (betibeglogene autotemcel) or beti-cel
ZYNTEGLO is a first-in-class, one-time ex-vivo LVV gene therapy approved for the treatment of beta-thalassemia in adult and pediatric patients who require regular red blood cell (RBC) transfusions. ZYNTEGLO works by adding functional copies of a modified form of the beta-globin gene (βA-T87Q-globin gene) into a patient’s own hematopoietic (blood) stem cells to enable the production of a modified functional adult hemoglobin (HbAT87Q). Once a patient has the βA-T87Q-globin gene, they have the potential to increase ZYNTEGLO-derived adult hemoglobin (HbAT87Q) and total hemoglobin to normal or near normal levels that can eliminate the need for regular RBC transfusions.
Indication
ZYNTEGLO is indicated for the treatment of adult and pediatric patients with beta-thalassemia who require regular red blood cell (RBC) transfusions.
Please see full Prescribing Information for ZYNTEGLO.
About bluebird bio, Inc.
bluebird bio is pursuing curative gene therapies to give patients and their families more bluebird days. With a dedicated focus on severe genetic diseases, bluebird has industry-leading clinical and research programs for sickle cell disease, beta-thalassemia and cerebral adrenoleukodystrophy and is advancing research to apply new technologies to these and other diseases. We custom design each of our therapies to address the underlying cause of disease and have developed in-depth and effective analytical methods to understand the safety of our lentiviral vector technologies and drive the field of gene therapy forward.
Founded in 2010, bluebird has the largest and deepest ex-vivo gene therapy data set in the world—setting the standard for industry. Today, bluebird continues to forge new paths, combining our real‑world experience with a deep commitment to patient communities and a people-centric culture that attracts and grows a diverse flock of dedicated birds.
For more information, visit bluebirdbio.com or follow us on social media at @bluebirdbio, LinkedIn, Instagram and YouTube.
ZYNTEGLO and bluebird bio are trademarks of bluebird bio, Inc.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220817005667/en/
Source: bluebird bio, Inc.
FDA approves bluebird bio's Zynteglo gene therapy for beta thalassemia; shares up 7%
Aug. 17, 2022 1:57 PM ET
By: Jonathan Block, SA News Editor3 Comments
Note: This article has been updated with bluebird bio share movement and the company's reimbursement program with payers.
- The U.S. FDA has approved bluebird bio's (NASDAQ:BLUE) Zynteglo (betibeglogene autotemcel), a gene therapy for the treatment of the blood disorder β-thalassemia.
- https://seekingalpha.com/news/3874245-fda-approves-bluebird-bio-zynteglo-beti-cel-gene-therapy-beta-thalassemia
- https://seekingalpha.com/symbol/BLUE
- https://www.bluebirdbio.com/
- https://www.bluebirdbio.com/our-science/pipeline
INBRX-109
CagriSema (2.4 mg semaglutide and 2.4 mg cagrilintide)
ZYNTEGLO® (betibeglogene autotemcel) or beti-cel
European Medicines Agency Grants Orphan Drug Designation to INBRX-109 for the Treatment of Chondrosarcoma
SAN DIEGO, Aug. 15, 2022 /PRNewswire/ --
Inhibrx, Inc. (Nasdaq: INBX), a biotechnology company with four clinical programs in development and a strong emerging pipeline, today announced that the European Commission ("EC"), based on a positive opinion issued by the European Medicines Agency ("EMA"), has granted orphan medicinal product designation to INBRX-109 for the treatment of chondrosarcoma.
"We are highly optimistic about the potential of INBRX-109 in chondrosarcoma to address a high unmet medical need," said Inhibrx Chief Executive Officer, Mark Lappe. "The positive opinion issued by the EMA is excellent news and acknowledges the potential of INBRX-109 as a treatment for patients throughout Europe who suffer from this debilitating, rare condition."
The EC grants orphan designation to drugs and biologics intended for the safe and effective treatment, prevention, or diagnosis of a disease that is life-threatening or chronically debilitating impacting fewer than five in 10,000 patients in the European Union ("EU"). Orphan drug designation in the EU can provide certain benefits, including reduced regulatory fees, clinical protocol assistance and the potential for up to ten years of market exclusivity following regulatory approval.
About INBRX-109
INBRX-109 is a precision-engineered, tetravalent death receptor 5 (DR5) agonist antibody designed to exploit the tumor-biased cell death induced by DR5 activation.
In 2021, the FDA granted Fast Track designation to INBRX-109 for the treatment of patients with unresectable or metastatic conventional chondrosarcoma and orphan-drug designation to INBRX-109 for chondrosarcoma in the United States.
In June 2021, Inhibrx initiated a randomized, blinded, placebo-controlled, potential registration-enabling Phase 2 trial of INBRX-109 in conventional chondrosarcoma, which is currently ongoing.
In November 2021, Inhibrx provided updated results from its ongoing Phase 1 clinical trial evaluating the efficacy and safety of INBRX-109 in patients with conventional chondrosarcoma. Preliminary disease control was observed in 16 of the 18 evaluable patients (89%) measured by RECISTv1.1, with two of the 18 achieving partial responses (11%). Based on preliminary results of the ongoing Phase 1 trial at that time, the median progression-free survival (PFS) was 7.4 months, and the median overall survival had not been reached. Three patients had exceeded 61 weeks on treatment with INBRX-109, with 77 weeks being the longest duration of stable disease observed at that time.
About Inhibrx, Inc.
Inhibrx is a clinical-stage biotechnology company focused on developing a broad pipeline of novel biologic therapeutic candidates in oncology and orphan diseases. Inhibrx utilizes diverse methods of protein engineering to address the specific requirements of complex target and disease biology, including its proprietary sdAb platform. Inhibrx has collaborations with 2seventy bio (formerly bluebird bio), Bristol-Myers Squibb and Chiesi. For more information, please visit www.inhibrx.com.
SOURCE Inhibrx Inc.
Europe drug regulator grants orphan designation to Inhibrx's drug for type of bone cancer
Aug. 15, 2022 4:38 PM ET
By: Anuron Mitra, SA News Editor
- Inhibrx (NASDAQ:INBX) on Monday said the European Commission had granted an orphan drug designation to the biotech's antibody INBRX-109 for the treatment of chondrosarcoma, a type of bonce cancer.
- https://seekingalpha.com/news/3873205-europe-drug-regulator-grants-orphan-designation-to-inhibrxs-drug-for-type-of-bone-cancer
- https://inhibrx.com/inbrx-109/
- https://seekingalpha.com/symbol/INBX
- https://inhibrx.com/
biotecHOPE™ 2022
Picankibart (IBI112)
KarXT (xanomeline-trospium)
Picankibart (IBI112)
Innovent Announces Phase 2 Clinical Study of Picankibart (IBI112) in Chinese Patients with Moderate-to-severe Plaque Psoriasis Met Primary Endpoint
Published on: Aug 10th, 2022
SAN FRANCISCO, US and SUZHOU, China, Aug 10, 2022 — Innovent Biologics, Inc. (“Innovent”) (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high-quality medicines for the treatment of oncology, autoimmune, metabolic, ophthalmology and other major diseases, announces that the primary endpoint was met in a multicenter, randomized, double-blind, placebo-controlled phase 2 study (clinicaltrials.gov, NCT 05003531) of picankibart (R&D code: IBI112), a recombinant anti-interleukin 23p19 subunit antibody injection, in Chinese patients with moderate-to-severe plaque psoriasis. This study (CIBI112A201) was designed to evaluate the efficacy and safety of picankibart under various administration in the treatment of moderate-to-severe plaque psoriasis. A total of 250 subjects were randomized 1:1:1:1:1 to receive 50 mg (once at week 0, 4, then every 12 weeks), 100mg (once at week 0, 4, then every 12 weeks), 100mg (once at week 0, 4, then every 8 weeks), 200mg (once at week 0, 4, then every 12 weeks) of picankibart or placebo. The primary endpoint of the study was the proportion of subjects who achieved a ≥90% improvement in the Psoriasis Area and Severity Index (PASI) at week 16 (PASI 90) and the total study period is 52 weeks. To date, a total of 245 subjects (98.0%) have completed the primary endpoint at week 16, and a total of 236 subjects (94.4%) have completed the visit at week 28.
- In terms of efficacy, 52.0%~54.9% of the subjects achieved PASI 90 after picankibart treatment at week 16, which was significantly higher than that in the placebo group and reached the primary endpoint, in addition, the proportions of subjects who achieved PASI 75 and PASI 100 were also significantly higher than that in the placebo group (all P-values＜0.001). The study results through 28 weeks showed a long-term efficacy of picankibart, with 61.2%~72.5% of the subjects who received picankibart achieving PASI 90, 78.4%~88.0% of the subjects achieved PASI 75, and in one of the groups received picankibart, about 40.0 % of subjects achieved complete skin lesions clearance (PASI 100).
- In terms of safety, picankibart is generally well tolerated, and no new safety signals occurred compared with previous clinical studies. The overall safety profile is similar to other IL-23p19 mAbs. The most frequently reported (incidence ≥10%) treatment-emergent adverse events were upper respiratory tract infection with mild to moderate severity.
- In addition, we observed a significant increase from baseline in the Dermatology Life Quality Index (DLQI) of subjects treated with each dose of picankibart compared to placebo (all P-values＜0.0001).
Clinical study results showed that 50-200mg picankibart administered every 12 or 8 weeks significantly improved the skin lesions and quality of life of subjects with moderate to severe plaque psoriasis, demonstrating the significant improvement effect at long-dose interval. It is worth noting that, at the 16-week primary endpoint assessment, subjects received only two administrations, significantly less than other similar biologics. Moreover, continued improvement in efficacy across the picankibart treatment was observed after reaching the primary endpoint (up to 28 weeks), demonstrating the potential sustained advantage of long interval dosing. The study period is 52 weeks (one year), and currently, subjects in the placebo group have been treated with IBI112, and the dosing and follow-up of the study are ongoing. Full data on the study will be published at future academic conferences or peer-reviewed journals. Professor Jianzhong Zhang of Peking University People’s Hospital, the principal investigator of the study, stated, “Psoriasis is a life-long disease, which has a huge impact on the physical and mental health and quality of life, while the current traditional treatment is not ideal. We are pleased to find significant clinical benefit and improved quality of life in subjects with moderate to severe plaque psoriasis in the phase 2 clinical study of picankibart. Meanwhile, picankibart also shows potential advantages in long dosing intervals and long-term efficacy. I am confident and looking forward to the success of its upcoming phase 3 study, and hope it will provide an alternative treatment option for Chinese psoriasis patients.” Dr. Lei Qian , Vice President of Clinical Development of Innovent, stated: “We are happy that phase 2 study of picankibart in Chinese patients with moderate-to-severe plaque psoriasis achieved primary clinical endpoint. As a new generation of IL-23p19 target drug, picankibart shows clear advantages in the long-dose interval and long-term efficacy. The dosing frequency is only 5 times annually, and it shows a great potential for both efficacy and safety, and could be offered as a more friendly treatment option compared to the current ones on the market whose dosing frequency is 7-16 times annually. Currently, there is no IL-23p19 target drug independently developed by Chinese pharmaceutical companies in the domestic market. We are greatly encouraged by the results of good efficacy, safety and tolerability of picankibart in the phase 2 clinical study. Based on this, we are confident and will actively advance the phase 3 clinical study of picankibart in patients with moderate to severe plaque psoriasis, and we plan to optimize the dosing regimen scientifically based on quantitative pharmacology to further improve efficacy, and synchronize the use of auto-injector in the phase 3 clinical study. We are striving to provide more convenient, user-friendly and effective treatment options for patients with moderate to severe plaque psoriasis as soon as possible and to fulfill our mission of providing high-quality innovative biopharmaceutical products that are accessible to patients.” About Picankibart (IBI112)Picankibart (IBI112) is a monoclonal antibody independently developed by Innovent, with independent intellectual property rights. This product specifically binds to IL-23p19 subunit, thereby preventing IL-23 from binding to cell surface receptors, resulting in the inhibition of the IL-23 receptor-mediated signaling pathway. Preclinical data demonstrated that IBI112 has a clear target and well-elucidated mechanism of action and significant anti-inflammatory effect. Further, good safety and tolerability have been verified in the phase 1 clinical study. At present, the phase 2 clinical studies of IBI112 in the indications of moderate to severe plaque psoriasis and ulcerative colitis are ongoing, and the primary clinical study endpoint has been met in the phase 2 clinical study of psoriasis, suggesting that it may provide a more effective treatment option for patients with psoriasis or other autoimmune diseases.
About Innovent
Inspired by the spirit of "Start with Integrity, Succeed through Action,” Innovent’s mission is to develop, manufacture and commercialize high-quality biopharmaceutical products that are affordable to ordinary people. Established in 2011, Innovent is committed to developing, manufacturing and commercializing high-quality innovative medicines for the treatment of oncology, autoimmune, metabolic, ophthalmology and other major diseases. On October 31, 2018, Innovent was listed on the Main Board of the Stock Exchange of Hong Kong Limited with the stock code: 01801. HK.
Since its inception, Innovent has developed a fully integrated multi-functional platform that includes R&D, CMC (Chemistry, Manufacturing, and Controls), clinical development and commercialization capabilities. Leveraging the platform, the company has built a robust pipeline of 34 valuable assets in the fields of cancer, autoimmune, metabolic, ophthalmology and other major therapeutic areas, among them, 7 varieties were selected for the national "Major New Drug Creation" special project, 7 products approved for marketing in China – TYVYT® (sintilimab injection), BYVASDA® (bevacizumab biosimilar injection), SULINNO® (adalimumab biosimilar injection), HALPRYZA® (rituximab biosimilar injection), Pemazyre® (pemigatinib oral inhibitor) and olverembatinib (BCR-ABL TKI) and Cyramza® (ramucirumab), 3 asset under NMPA NDA review, 4 assets in Phase 3 or pivotal clinical trials, and an additional 20 molecules in clinical studies.
Innovent has built an international team with advanced talent in high-end biological drug development and commercialization, including many global experts. The company has also entered into strategic collaborations with Eli Lilly and Company, Sanofi, Adimab, Incyte, MD Anderson Cancer Center, and other international partners. Innovent strives to work with many collaborators to help advance China’s biopharmaceutical industry, improve drug availability and enhance the quality of the patients’ lives. For more information, please visit: www.innoventbio.com.
Note:
TYVYT® (Sintilimab Injection) is not an approved product in the United States.
BYVASDA® (bevacizumab biosimilar injection), HALPRYZA® (rituximab biosimilar injection), and SULINNO® (adalimumab biosimilar injection) are not approved products in the United States.
TYVYT® (sintilimab injection, Innovent)
BYVASDA® (bevacizumab biosimilar injection, Innovent)
HALPRYZA® (rituximab biosimilar injection, Innovent)
SULINNO® (adalimumab biosimilar injection, Innovent)
Pemazyre® (pemigatinib oral inhibitor, Incyte Corporation). Pemazyre® was discovered by Incyte Corporation and licensed to Innovent for development and commercialization in Mainland China, Hong Kong, Macau and Taiwan.
CYRAMZA® (ramucirumab, Eli Lilly). Cyramza® was discovered by Eli Lilly and licensed to Innovent for commercialization in Mainland China.
Innovent psoriasis drug reduces disease severity, meets main goal in mid-stage study
Aug. 10, 2022 6:01 AM ET
Innovent Biologics, Inc. (IVBIY)
By: Ravikash, SA News Editor
Innovent Biologics (OTCPK:IVBIY) medicine picankibart helped reduce the severity of disease in Chinese patients with moderate-to-severe plaque psoriasis, meeting the main goal of a phase 2 trial.
https://seekingalpha.com/symbol/IVBIY
GFH009
KarXT (xanomeline-trospium)
Picankibart (IBI112)
SELLAS Life Sciences’ GFH009 Demonstrates Cancer Cell Growth Inhibition in Preclinical In Vitro Studies in Solid Cancer and Acute Myeloid Leukemia Cell Lines
PDF Format (opens in new window) (PDF)
- Results Demonstrate Significant Anti-Tumor Effects on All Selected Cell Lines -
- In Three of the Four Cell Lines, GFH009 Inhibited Cancer Growth by 90 to 100 Percent -
NEW YORK, Aug. 09, 2022 (GLOBE NEWSWIRE) --
SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) (“SELLAS" or the “Company”), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, today announced results from preclinical in vitro studies for its highly selective CDK9 inhibitor, GFH009, in solid cancer and acute myeloid leukemia (AML) cell lines. The data shows that GFH009 demonstrated significant anti-tumor effects in all four selected cell lines. In three out of the four cell lines, GFH009 inhibited cancer cell growth by 90 to 100 percent.
The in vitro studies were conducted at an independent, third-party contract research organization, Translational Drug Development (TD2), and the following cell lines were selected for the studies based on their unique characteristics, combined with GFH009’s mechanism of action:
- RH30: a pediatric soft tissue sarcoma cell line that is a model for studying high-risk pediatric rhabdomyosarcoma, an indication for which currently available treatments are limited to high-dose chemotherapy with unsatisfactory results. RH30 cells are driven by PAX3-FOXO1, a transcription factor whose expression is strictly needed for tumor cell survival. RH30 is also dependent on MYCN, a major target of CDK9 inhibition. Treatment with GFH009 resulted in 90 percent or more cancer inhibition at dose levels equivalent to those already demonstrated to be safe in patients in the ongoing Phase 1 trial with no viable cancer cells at the highest dose levels.
- NCI-H209: a small cell lung cancer cell line characterized by the loss of function of two major tumor suppressor genes, RB1 and TP53. This cell line also expresses MCL-1, a major target of CDK9 inhibition. Treatment with GFH009 resulted in 90 percent or more cancer inhibition at dose levels equivalent to those already demonstrated to be safe in patients in the ongoing Phase 1 trial with no viable cancer cells at highest dose levels.
- SKOV-3: an ovarian cancer cell line containing the wild type BRCA1 gene and highly expresses CDK9. Treatment with GFH009 resulted in more than 50 percent cancer inhibition at dose levels equivalent to those already demonstrated to be safe in patients in the ongoing Phase 1 trial.
- OCI-AML-2: an AML cell line that develops resistance to the chemotherapy venetoclax on exposure. Treatment with GFH009 resulted in 90 to 100 percent cancer inhibition at dose levels equivalent to those already demonstrated to be safe in patients in the ongoing Phase 1 trial with no viable cancer cells at the highest dose levels.
“The data from preclinical studies that we are reporting today demonstrates that certain cancer cell lines, whose survival depends on specific changes in the cell, can be identified and treated with GFH009,” said Dragan Cicic, MD, Senior Vice President, Clinical Development, of SELLAS. “Through identifying specific genes in cancer cells, GFH009 has shown in these preclinical studies the ability to stop the transcription and expression from gene to protein, inhibiting cancer cell growth altogether. We will utilize this important information as we design our clinical development program for GFH009 in adult and pediatric tumor types.”
About Translational Drug Development (TD2)
TD2 is an oncology development organization that provides innovative services for oncology-focused companies. Using a dedicated team of professionals with broad experience and understanding in drug development, TD2 is uniquely positioned to support improved and accelerated development of medicines for life-threatening oncology diseases. TD2 applies rigorous and high-throughput translational preclinical development, combined with regulatory affairs expertise, to customize clinical trial design and execution. TD2’s suite of capabilities encourages the timely selection of patient populations who are most likely to benefit from a new agent, and the rapid identification of clinically significant endpoints. TD2 is committed to reducing the risks and uncertainty inherent in the drug development process and to the acceleration of patient access to promising treatments. For more information, visit www.TD2inc.com.
About SELLAS Life Sciences Group, Inc.
SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) is a late-stage clinical biopharmaceutical company focused on the development of novel therapeutics for a broad range of cancer indications. SELLAS’ lead product candidate, galinpepimut-S (GPS), is licensed from Memorial Sloan Kettering Cancer Center and targets the WT1 protein, which is present in an array of tumor types. GPS has potential as a monotherapy or in combination with other therapies to address a broad spectrum of hematologic malignancies and solid tumor indications. The Company is also developing GFH009, a small molecule, highly selective CDK9 inhibitor, which is licensed from GenFleet Therapeutics (Shanghai), Inc., for all therapeutic and diagnostic uses in the world outside of Greater China.
For more information on SELLAS, please visit www.sellaslifesciences.com.
Source: SELLAS Life Sciences Group, Inc.
SELLAS Life Sciences rises 9% after cancer drug shows promising result in animal study
Aug. 09, 2022 9:33 AM ET
SELLAS Life Sciences Group, Inc. (SLS)
By: Dania Nadeem, SA News Editor
- SELLAS Life Sciences (NASDAQ:SLS) is trading 9.2% higher on Tuesday after the company announced results from preclinical studies for its CDK9 inhibitor, GFH009, to treat solid cancer and acute myeloid leukemia (AML) cell lines.
- https://seekingalpha.com/news/3869812-sellas-life-sciences-cancer-drug-shows-promising-result-in-animal-study
- https://seekingalpha.com/symbol/SLS
- https://www.sellaslifesciences.com/pipeline/sellas-pipeline/default.aspx
KarXT (xanomeline-trospium)
KarXT (xanomeline-trospium)
KarXT (xanomeline-trospium)
Karuna Therapeutics Announces Positive Results from Phase 3 EMERGENT-2 Trial of KarXT in Schizophrenia
Aug 8, 2022 at 6:30 AM EDT
Trial met primary endpoint, with KarXT demonstrating a statistically significant 9.6-point reduction in PANSS Total Score compared to placebo at Week 5 (p<0.0001)
Trial also met key secondary endpoints, demonstrating statistically significant reductions in positive and negative symptoms of schizophrenia, as measured by the PANSS positive, PANSS negative and PANSS negative Marder factor subscales
KarXT was generally well tolerated, with a side effect profile substantially consistent with prior trials of KarXT in schizophrenia
The Company plans to submit a New Drug Application (NDA) with the U.S. Food & Drug Administration (FDA) in mid-2023
Conference call and webcast to take place today at 8:00 a.m. ET
BOSTON--(BUSINESS WIRE)--Aug. 8, 2022-- Karuna Therapeutics, Inc. (NASDAQ: KRTX), a clinical-stage biopharmaceutical company driven to create and deliver transformative medicines for people living with psychiatric and neurological conditions, today announced positive topline results from its Phase 3 EMERGENT-2 trial evaluating the efficacy, safety, and tolerability of its lead investigational therapy, KarXT (xanomeline-trospium), in adults with schizophrenia. The trial met its primary endpoint, with KarXT demonstrating a statistically significant and clinically meaningful 9.6-point reduction in the Positive and Negative Syndrome Scale (PANSS) total score compared to placebo (-21.2 KarXT vs. -11.6 placebo, p<0.0001) at Week 5 (Cohen’s d effect size of 0.61). KarXT also demonstrated an early and sustained statistically significant reduction of symptoms, as assessed by PANSS total score, starting at Week 2 and maintained such reduction through all timepoints in the trial.
“We are thrilled that these topline results from the Phase 3 EMERGENT-2 trial confirm what was seen in our Phase 2 EMERGENT-1 trial and underscore the potential for KarXT, with its novel and unique mechanism of action, to redefine what successful treatment looks like for the 21 million people living with schizophrenia worldwide, and potentially usher in the first new class of medicine for these patients in more than 50 years,” said Steve Paul, M.D., chief executive officer, president and chairman of Karuna Therapeutics. “These results represent our second positive registrational trial. We look forward to continuing to gather long-term safety data to support our submission of a New Drug Application with the U.S. Food and Drug Administration for KarXT as a treatment for schizophrenia, which we expect to occur in mid-2023.”
KarXT also met key secondary endpoints in the Phase 3 EMERGENT-2 trial, demonstrating a statistically significant reduction in both positive symptoms (e.g., hallucinations or delusions) and negative symptoms (e.g., difficulty enjoying life or withdrawal from others) of schizophrenia as measured by the PANSS positive, PANSS negative and PANSS negative Marder factor subscales. Results at Week 5 include:
- 2.9-point reduction in the PANSS positive subscale with KarXT compared to placebo (-6.8 KarXT vs. -3.9 placebo, p<0.0001).
- 1.8-point reduction in the PANSS negative subscale with KarXT compared to placebo (-3.4 KarXT vs. -1.6 placebo, p=0.0055).
- 2.2-point reduction in the PANSS negative Marder factor subscale with KarXT compared to placebo (-4.2 KarXT vs. -2.0 placebo, p=0.0022).
KarXT was generally well tolerated. Overall discontinuation rates were similar between KarXT and placebo groups (25% vs. 21%). The overall treatment-emergent adverse events (TEAEs) rate for KarXT and placebo was 75% and 58%, respectively. Discontinuation rates related to TEAEs were similar between KarXT (7%) and placebo (6%). Equal rates of serious TEAEs were observed between KarXT and placebo (2% in each group) and included suicidal ideation, worsening of schizophrenia symptoms, and appendicitis. None of the serious TEAEs were determined to be drug related. The most common TEAEs (>5%) in the KarXT arm were all mild to moderate in severity and included constipation, dyspepsia, nausea, vomiting, headache, increases in blood pressure, dizziness, gastroesophageal reflux disease (acid reflux), abdominal discomfort, and diarrhea. Mean blood pressure measures were similar between KarXT and placebo throughout the trial, and no syncopal events were observed. In the subset of patients with a TEAE of blood pressure increases, mean blood pressure at endpoint was similar to baseline and did not lead to trial discontinuation. Similar to prior trials, an increase in heart rate was associated with KarXT treatment and decreased in magnitude by the end of the trial. Consistent with EMERGENT-1, KarXT was not associated with common problematic side effects of current treatments, including sedation (somnolence), weight gain, and extrapyramidal symptoms.
“Despite the number of available treatment options, there continues to be a tremendous unmet need in the treatment of schizophrenia, placing an immense burden on both patients and their caregivers,” said Rishi Kakar, M.D., chief scientific officer, Segal Trials and lead investigator of the Phase 3 EMERGENT-2 trial. “These data build on the growing body of clinical evidence supporting the potential of KarXT as a new and differentiated approach for schizophrenia, demonstrating notable improvements across both positive and negative symptoms, while not being associated with common problematic side-effects of current therapies, such as weight gain, sedation and movement disorders. This unique profile of KarXT has the potential to provide a new meaningful treatment option for our patients and their families beyond the current standard of care.”
The EMERGENT program consists of the completed positive Phase 2 EMERGENT-1 and Phase 3 EMERGENT-2 trials, as well as three ongoing trials evaluating the acute efficacy and long-term safety of KarXT (EMERGENT-3, EMERGENT-4, and EMERGENT-5). Topline data from the Phase 3 EMERGENT-3 trial are expected in the first quarter of 2023. The data from our EMERGENT program will be used to support submission of an NDA with the U.S. FDA for KarXT as a treatment for schizophrenia, which is expected in mid-2023. Additional analysis of data from the Phase 3 EMERGENT-2 trial is ongoing, with plans to present these results at future medical meetings.
About the Phase 3 EMERGENT-2 Trial
The Phase 3 EMERGENT-2 trial is a double-blind, placebo-controlled, five-week, inpatient trial evaluating the efficacy, safety, and tolerability of our lead investigational therapy, KarXT, as compared to placebo in adults with schizophrenia in the United States. The primary endpoint was change from baseline in Positive and Negative Syndrome Scale (PANSS) total score, a scale for measuring schizophrenia symptom severity, of KarXT compared to placebo at Week 5. Key secondary endpoints included change from baseline in PANSS positive, PANSS negative and PANSS negative Marder factor subscale of KarXT compared to placebo at Week 5.
A total of 252 adults (between the ages of 18-65 years) with a confirmed diagnosis of schizophrenia who were experiencing symptoms of psychosis enrolled in the trial. Patients were randomized 1:1 to receive either a flexible dose of KarXT (n=126) or placebo (n=126) two times a day (BID) for five weeks. On Days 1-2, patients received a dose of 50/20 KarXT (50mg xanomeline/20mg trospium) BID or matching placebo. On Day 3, patients escalated to a dose of 100/20 BID, and starting on Day 8, patients could increase to 125/30 BID based on tolerability. In the trial, 81% of patients on KarXT compared to 90% on placebo titrated to the highest dose level (125/30).
About KarXT
KarXT (xanomeline-trospium) is an oral, investigational M1/M4-preferring muscarinic agonist in development for the treatment of psychiatric and neurological conditions, including schizophrenia and psychosis in Alzheimer’s disease. Comprised of muscarinic agonist xanomeline and muscarinic antagonist trospium, it is designed to preferentially stimulate muscarinic receptors in the central nervous system. KarXT is the first potential medicine of its kind with a truly new and unique dual mechanism that does not rely on the dopaminergic or serotonergic pathway to treat symptoms of serious mental illness. This approach has the potential to provide a differentiated therapy, and, if approved, to beneficially impact the lives of millions of people with serious mental illness.
About Karuna Therapeutics
Karuna Therapeutics is a clinical-stage biopharmaceutical company driven to create and deliver transformative medicines for people living with psychiatric and neurological conditions. At Karuna, we understand there is a need for differentiated and more effective treatments that can help patients navigate the challenges presented by serious mental illness. Utilizing our extensive knowledge of neuroscience, we are harnessing the untapped potential of the brain in pursuit of novel pathways to develop medicines that make meaningful differences in peoples’ lives. For more information, please visit www.karunatx.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220808005222/en/
Source: Karuna Therapeutics, Inc.
Karuna Therapeutics jumps 52% as schizophrenia trial meets key goal
Aug. 08, 2022 8:29 AM ET
Karuna Therapeutics, Inc. (KRTX)ZLAB
By: Dulan Lokuwithana, SA News Editor3 Comments
The shares of Karuna Therapeutics (NASDAQ:KRTX) surged ~52% in the pre-market on Monday after the clinical-stage biotech announced that its late-stage trial for experimental schizophrenia therapy KarXT met the primary endpoint. The ADRs of Chinese biotech Zai Lab Limited (ZLAB), Karuna's (KRTX) partner for the program also added ~15% in reaction.
https://seekingalpha.com/news/3868667-krtx-stock-jumps-as-schizophrenia-trial-meets-key-goals
https://seekingalpha.com/symbol/KRTX
Fruquintinib
TPOXX® (Tecovirimat)
KarXT (xanomeline-trospium)
Announcements & Press Releases, Oncology / Immunology | 8 Aug 2022
HUTCHMED Announces that Fruquintinib Global Phase III FRESCO-2 Study Has Met Its Primary Endpoint in Metastatic Colorectal Cancer
— Trial met primary endpoint of overall survival and all secondary endpoints —
— Overall safety consistent with fruquintinib known profile —
— Plans for regulatory submissions underway in the U.S., Europe and Japan —
— Results to be submitted to an upcoming medical meeting —
Hong Kong, Shanghai & Florham Park, NJ — Monday, August 8, 2022: HUTCHMED (China) Limited (“HUTCHMED”) (Nasdaq/AIM: HCM, HKEX: 13) today announces that the pivotal global Phase 3 FRESCO-2 trial evaluating the investigational use of fruquintinib met its primary endpoint of overall survival (“OS”) in patients with advanced, refractory metastatic colorectal cancer (“CRC”).
The FRESCO-2 study was a multi-regional clinical trial conducted in the U.S., Europe, Japan and Australia that investigated fruquintinib plus best supportive care (“BSC”) vs placebo plus BSC in patients with metastatic CRC who had progressed on standard chemotherapy and relevant biologic agents and who had progressed on, or were intolerant to, TAS-102 and/or regorafenib. In addition to OS, a statistically-significant improvement in progression-free survival (“PFS”), a key secondary endpoint, was observed. The safety profile of fruquintinib in FRESCO-2 was consistent with previously reported studies. Full results will be submitted for presentation at an upcoming medical meeting.
HUTCHMED has been in communication with regulatory agencies globally regarding the FRESCO-2 trial design and conduct and will discuss these data with the agencies in the U.S., Europe and Japan with the intent to submit marketing authorization applications as soon as possible. The U.S. FDA granted Fast Track Designation for the development of fruquintinib for the treatment of patients with metastatic CRC in June 2020.
“We are very happy to see the positive outcomes of the FRESCO-2 study which offers a potential new treatment for patients with advanced metastatic colorectal cancer, where the unmet need is very high and patients have limited treatment options,” said Dr Marek Kania, Executive Vice President, Managing Director and Chief Medical Officer of HUTCHMED International. “Results from the global FRESCO-2 study supplement findings from the original FRESCO study that led to the marketing approval and commercialization of fruquintinib in China. We would like to thank the patients, their families, and the healthcare professionals who participated in this study and helped achieve this important milestone.”
Professor Cathy Eng, MD, FACP, FASCO, David H. Johnson Endowed Chair in Surgical and Medical Oncology and Co-Leader, Gastrointestinal Cancer Research Program, at the Vanderbilt-Ingram Cancer Center, who served as the FRESCO2 co-PI and Steering Committee member said: “Completion of the international FRESCO-2 phase III trial in a timely fashion during the era of COVID-19 isolation demonstrates the unmet need for new therapeutic agents in metastatic colorectal cancer. By meeting the primary endpoint of OS with a secondary endpoint of PFS, fruquintinib provides a significant potential new option for our refractory colorectal cancer patients. As an oral agent, fruquintinib also provides added convenience for our patients. Based on fruquintinib’s profile, we will likely see further exploration in future clinical trials in different settings. This is extremely encouraging, and I look forward to seeing the final results.”
Dr Weiguo Su, Chief Executive Officer and Chief Scientific Officer of HUTCHMED, said: “We are pleased to have successfully completed our first multi-regional clinical trial, FRESCO-2, to support the global registration of fruquintinib. It has already benefited patients with advanced CRC in China since its launch in 2018. It is also being evaluated alone and in combination with other agents in various tumor types in ongoing studies around the world.”
HUTCHMED retains all commercial rights to fruquintinib outside of China. In China, where fruquintinib is marketed under the brand name ELUNATE®, 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. Fruquintinib is not approved for use outside of China.
About Fruquintinib
Fruquintinib is a highly selective and potent oral inhibitor of VEGFR-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.
About Fruquintinib Approval in China
Metastatic CRC in China: Fruquintinib was approved for marketing by the China National Medical Products Administration (NMPA) in September 2018 and commercially launched in China in late November 2018 under the brand name ELUNATE®. It has been included in the China National Reimbursement Drug List (NRDL) since January 2020. ELUNATE® is indicated for the treatment of patients with metastatic CRC who 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). Results of the FRESCO study[iii], a Phase III pivotal registration trial of fruquintinib in 416 patients with metastatic CRC in China, were published in The Journal of the American Medical Association, JAMA, in June 2018 (clinicaltrials.gov identifier: NCT02314819).
About HUTCHMED
HUTCHMED (Nasdaq/AIM: HCM; HKEX: 13) is an innovative, commercial-stage, biopharmaceutical company. It is committed to the discovery and global development and commercialization of targeted therapies and immunotherapies for the treatment of cancer and immunological diseases. It has more than 4,900 personnel across all its companies, at the center of which is a team of about 1,800 in oncology/immunology. Since inception it has advanced 13 cancer drug candidates from in-house discovery into clinical studies around the world, with its first three oncology drugs now approved and marketed in China. For more information, please visit: www.hutch‑med.com or follow us on LinkedIn.
Hutchmed fruquintinib improves survival, meets main goal of colorectal cancer study
Aug. 08, 2022 5:55 AM ETHUTCHMED (China) Limited (HCM), LLYBAYRY, BAYZFBy: Ravikash, SA News Editor
Hutchmed (China) (NASDAQ:HCM) said its medicine fruquintinib met the main goal of overall survival (OS) in patients with advanced, refractory metastatic colorectal cancer (CRC) in a phase 3 trial.
The study called FRESCO-2 evaluated fruquintinib plus best supportive care (BSC) versus placebo plus BSC in patients with metastatic CRC who had progressed on standard chemotherapy and relevant biologic agents and who had progressed on, or were intolerant to, TAS-102 and/or regorafenib, sold as Stivarga by Bayer (OTCPK:BAYRY) (OTCPK:BAYZF).
https://seekingalpha.com/symbol/HCM
https://www.hutch-med.com/pipeline-and-products/our-pipeline/
TPOXX® (Tecovirimat)
TPOXX® (Tecovirimat)
TPOXX® (Tecovirimat)
Siga’s Monkeypox Drug to Start Human Testing in September
Aug. 5, 2022, 4:11 PM
- Studied only in animals, Tpoxx will undergo thorough review
- Therapy is now available through an expanded access protocol
SIGA's Tpoxx antiviral to undergo human testing for monkeypox in September - Bloomberg
Aug. 05, 2022 4:53 PM ET
SIGA Technologies, Inc. (SIGA)
By: Jonathan Block, SA News Editor1 Comment
- Tpoxx (tecovirimat), an antiviral medication from SIGA Technologies (NASDAQ:SIGA), will begin testing in humans as soon as September, Bloomberg reported.
- https://seekingalpha.com/news/3868451-sigas-tpoxx-antiviral-to-undergo-human-testing-for-monkeypox-in-september-bloomberg
- https://seekingalpha.com/symbol/SIGA
- https://www.siga.com/wp-content/themes/sigahba/TPOXX-Fact-Sheet.pdf
SIGA Announces Approximately $28 Million of International Procurement Orders for Oral TPOXX® (Tecovirimat)
July 12, 2022 at 4:15 PM EDT
- Approximately $26 Million of Orders Received from Canada under
Existing Procurement Contracts –
- Approximately $2 Million of Orders Received from
Two New Jurisdictions -
NEW YORK, July 12, 2022 (GLOBE NEWSWIRE) -- SIGA Technologies, Inc. (SIGA) (NASDAQ: SIGA),
Information for Healthcare Providers on Obtaining and Using TPOXX (Tecovirimat) for Treatment of Monkeypox
Updated July 22, 2022Print
CDC, in partnership with FDA, has made it easier for healthcare providers to provide tecovirimat (TPOXX) treatment to patients with monkeypox under the expanded access investigational new drug (EA-IND).
The streamlined process allows healthcare providers to start treatment before the paperwork is submitted, and reduces the number of required forms, patient samples, photos, and gives patients the option to see their doctor virtually.
https://www.cdc.gov/poxvirus/monkeypox/clinicians/obtaining-tecovirimat.html
VAX-24
TPOXX® (Tecovirimat)
TPOXX® (Tecovirimat)
Vaxcyte Provides Positive Regulatory Updates for VAX-24 Adult and Pediatric Programs
August 4, 2022PDF Version
-- Company Receives FDA Fast Track Designation for VAX-24 in Adults --
-- Vaxcyte Completes Successful Pre-IND Meeting with FDA Regarding VAX-24 Pediatric Program, Supporting Path to Proceed Directly into Infants --
-- VAX-24 is 24-Valent Pneumococcal Conjugate Vaccine Designed to Deliver Broad-Spectrum Protection to Prevent Invasive Pneumococcal Disease --
SAN CARLOS, Calif., Aug. 04, 2022 (GLOBE NEWSWIRE) -- Vaxcyte, Inc. (Nasdaq: PCVX), a clinical-stage vaccine innovation company engineering high-fidelity vaccines to protect humankind from the consequences of bacterial diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to VAX-24, the Company’s 24-valent pneumococcal conjugate vaccine (PCV) candidate designed to prevent invasive pneumococcal disease (IPD), in adults ages 18 and older. The Fast Track designation is an FDA process that has been designed to facilitate the development and expedite the review of drugs, including vaccines, that treat or prevent serious conditions and fill an unmet medical need.
The Company also completed a successful pre-Investigational New Drug (IND) meeting with the FDA regarding the VAX-24 pediatric program. Vaxcyte received positive written feedback from the FDA supporting the initiation of a pediatric study that proceeds directly into infants, contingent on satisfactory topline safety, tolerability and immunogenicity results from the ongoing VAX-24 Phase 1/2 clinical proof-of-concept study in adults 18 to 64 years of age. This approach provides the Company with an accelerated clinical path to deliver a potentially best-in-class PCV, VAX-24, to the pediatric population, which represents the largest portion of the pneumococcal vaccine market in the United States.
“We are very pleased with the FDA’s feedback, which we believe provides an expedited path to deliver VAX-24 to adults and children, while also underscoring the need for a PCV that provides broader protection to prevent this serious disease,” said Grant Pickering, Chief Executive Officer and Co-Founder of Vaxcyte. “By leveraging our site-specific technology, the XpressCF™ cell-free protein synthesis platform, VAX-24 is designed to improve upon the standard-of-care PCVs and surpass the coverage of those currently available without compromising overall immune response.”
“Despite high vaccination rates, a broader spectrum PCV remains an important public health need, especially in vulnerable populations such as infants and older adults, due to substantial disease driven by emerging serotypes not covered by currently available vaccines,” said Jim Wassil, Executive Vice President and Chief Operating Officer of Vaxcyte. “With multiple milestones anticipated over the next 12 months for VAX-24, we expect strong, continued momentum.”
About VAX-24 and the Ongoing Adult Clinical Program
VAX-24 is an investigational 24-valent PCV candidate designed to prevent IPD, which can be most serious for infants, young children, older adults and those with immune deficiencies or certain chronic health conditions.
VAX-24 is intended to improve upon the standard-of-care PCVs for both children and adults by covering the serotypes that are responsible for most of the pneumococcal disease currently in circulation. Vaxcyte aims to efficiently create and deliver high-fidelity, broad-spectrum vaccines, such as VAX-24, by using modern synthetic techniques, including advanced chemistry and the XpressCF™ cell-free protein synthesis platform. Vaxcyte is deploying this approach with VAX-24 in order to add more pneumococcal strains without compromising the overall immune response.
VAX-24 is currently being evaluated in two clinical studies in adults:
- A Phase 1/2 Clinical Proof-of-Concept Study in Adults Aged 18-64: The Phase 1/2 clinical proof-of-concept study is now fully enrolled and the Company expects to announce topline safety and tolerability results from the Phase 1 portion of the study and safety, tolerability and immunogenicity results from the Phase 2 portion of the study in October or November 2022.
- The VAX-24 Phase 1/2 clinical proof-of-concept study (VAX-24 Study 101, NCT05266456) is a randomized, observer-blind, dose-finding, controlled study designed to evaluate the safety, tolerability and immunogenicity of VAX-24 in healthy adults 18-64 years of age.
- The Phase 1 portion of the study is evaluating safety and tolerability of a single injection of VAX-24 at three dose levels and compared to Prevnar 20™ in 64 healthy adults 18 to 49 years of age.
- The Phase 2 portion is evaluating the safety, tolerability and immunogenicity of a single injection of VAX-24 at three dose levels and compared to a single injection of Prevnar 20™ in approximately 800 healthy adults 50 to 64 years of age.
- A Separate Phase 2 Clinical Study in Adults Aged 65 and Older: Vaxcyte announced the dosing of the first participants in a separate Phase 2 study in adults 65 years of age and older in July 2022. Topline safety, tolerability and immunogenicity results from this study are expected in the first half of 2023, further building upon the body of clinical evidence to support the potential of VAX-24 as the broadest-spectrum PCV.
- This VAX-24 Phase 2 clinical study (VAX-24 Study 102, NCT05297578) is a randomized, observer-blind, dose-finding, controlled study designed to evaluate the safety, tolerability and immunogenicity of VAX-24 in approximately 200 healthy adults 65 years of age and older.
- The study is evaluating the safety, tolerability and immunogenicity of a single injection of VAX-24 at three dose levels and compared to a single injection of Prevnar 20™.
About Vaxcyte
Vaxcyte is a vaccine innovation company engineering high-fidelity vaccines to protect humankind from the consequences of bacterial diseases. The Company is developing broad-spectrum conjugate and novel protein vaccines to prevent or treat bacterial infectious diseases. Vaxcyte’s lead candidate, VAX-24, is a 24-valent, broad-spectrum pneumococcal conjugate vaccine being developed for the prevention of IPD. Vaxcyte is re-engineering the way highly complex vaccines are made through modern synthetic techniques, including advanced chemistry and the XpressCFTM cell-free protein synthesis platform, exclusively licensed from Sutro Biopharma, Inc. Unlike conventional cell-based approaches, the Company’s system for producing difficult-to-make proteins and antigens is intended to accelerate its ability to efficiently create and deliver high-fidelity vaccines with enhanced immunological benefits. Vaxcyte’s pipeline also includes VAX-XP, a PCV with an expanded breadth of coverage of greater than 30 strains; VAX-A1, a prophylactic vaccine candidate designed to prevent Group A Strep infections; and VAX-PG, a therapeutic vaccine candidate designed to slow or stop the progression of periodontal disease. Vaxcyte is driven to eradicate or treat invasive bacterial infections, which have serious and costly health consequences when left unchecked. For more information, visit www.vaxcyte.com.
Vaxcyte wins FDA’s Fast Track designation for pneumococcal vaccine candidate
Aug. 04, 2022 9:30 AM ET
By: Dulan Lokuwithana, SA News Editor
Clinical-stage biotech Vaxcyte, Inc. (NASDAQ:PCVX) announced on Thursday that the FDA awarded the Fast Track designation to the company's pneumococcal conjugate vaccine (PCV) candidate VAX-24 in adults aged 18 years and older.
https://seekingalpha.com/symbol/PCVX
RGX-121
HyBryte™ (research name SGX301)
ONPATTRO® (Patisiran)
REGENXBIO ANNOUNCES INTENTION TO FILE A BIOLOGICS LICENSE APPLICATION USING THE ACCELERATED APPROVAL PATHWAY FOR RGX-121, AN AAV THERAPEUTIC FOR THE TREATMENT OF MPS II
August 3, 2022 at 7:05 AM EDT
- FDA will consider an accelerated approval pathway for RGX-121; BLA filing expected in 2024
- Pivotal program is active and enrolling patients
- RGX-121 is a potential first-in-class, one-time gene therapy for the treatment of MPS II
- Internal cGMP manufacturing process expected to support BLA
ROCKVILLE, Md., Aug. 3, 2022 /PRNewswire/ -- REGENXBIO Inc. (Nasdaq: RGNX) today announced its intention to file a Biologics License Application (BLA) in 2024 using the FDA's accelerated approval pathway for RGX-121 for the treatment of Mucopolysaccharidosis Type II (MPS II), also known as Hunter Syndrome. The Company also announced that a pivotal program for RGX-121 is active and enrolling patients. RGX-121 is an investigational, one-time AAV Therapeutic using the NAV AAV9 vector to deliver the gene that encodes the iduronate-2-sulfatase (I2S) enzyme.
"We are pleased to share that our recent discussions with the FDA support our plans to submit a BLA for RGX-121 in 2024 using the accelerated approval pathway, which was created to allow for expedited development of drugs that treat serious conditions and provide a meaningful advantage over available therapies based on a surrogate endpoint," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "We believe RGX-121 for the treatment of Hunter Syndrome has demonstrated emerging positive impact on neurodevelopmental function, and we intend to advance this program as quickly as possible with the aim of providing a much-needed new treatment option for the MPS II community."
"The accelerated approval pathway gives patients access to potentially life-changing therapeutics sooner, which is why it is a critical tool for the over 7,000 rare diseases, most of which lack FDA-approved treatments," said Joseph Muenzer, MD, PhD, Bryson Distinguished Professor in Pediatrics Genetics, and Director, MPS Research and Treatment Program, University of North Carolina at Chapel Hill. "Boys with MPS II do not have time to wait. I am encouraged by FDA's commitment to advance therapies for rare diseases and their willingness to consider this important pathway for RGX-121."
The ongoing Phase I/II trial of RGX-121 in children up to five years old has been expanded into a pivotal Phase I/II/III trial, named CAMPSIITE™. A BLA filing in 2024 is expected to be supported by endpoints in the pivotal program, including changes from baseline of glycosaminoglycans (GAGs) in the cerebrospinal fluid (CSF) at four months. GAGs in the CSF have the potential to be considered a surrogate biomarker that is reasonably likely to predict clinical benefit in MPS II disease under the accelerated approval pathway, as buildup of GAGs in the CSF of MPS II patients correlates with clinical manifestations, including neurodevelopmental deficits.
As presented in February 2022, preliminary results from REGENXBIO's Phase I/II trial of RGX-121 indicated that RGX-121 continued to be well-tolerated with dose-dependent reductions of GAGs in the CSF, with patients in Cohort 3 approaching normal levels of certain GAGs in the CSF. Measures of neurodevelopmental function from patients in Cohorts 1 and 2 demonstrated continued developmental skill acquisition up to 2 years after RGX-121 administration.
"MPS II families are in need of a treatment option that can address the neurodevelopmental decline associated with this disease that available enzyme replacement therapies cannot," said Terri Klein, President and CEO of the National MPS Society. "We are encouraged by today's announcement and the hope that a one-time gene therapy offers this community."
Pivotal Program (CAMPSIITE Trial)
CAMPSIITE is a multicenter, open-label trial enrolling boys with MPS II, aged 4 months up to five years of age. CAMPSIITE is now expected to enroll up to 10 MPS II patients to support the BLA filing using the accelerated approval pathway, with the potential to enroll additional patients. These patients will receive a dose of 2.9x1011 GC/g of brain mass of RGX-121, which is the same dose being evaluated in Cohort 3 of the Phase I/II trial. The pivotal program is using commercial-scale cGMP material from REGENXBIO's proprietary, high-yielding suspension-based manufacturing process, named NAVXPress™. In addition to measuring GAGs in the CSF, the trial will continue to collect neurodevelopmental data and caregiver-reported outcomes.
CAMPSIITE is a global trial, which is expected to include sites in the United States, Brazil and Canada. REGENXBIO has begun dosing patients in the pivotal program.
"MPS II patients face irreversible neurodevelopmental decline that significantly impacts a child's daily life and life expectancy," said Paul Harmatz, MD, professor, UCSF Department of Pediatrics, and principal investigator of the CAMPSIITE study. "Current therapies are inadequate, so we are excited to be a part of this study and eager to see the potential impact on patients."
REGENXBIO plans to share new RGX-121 data at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium on August 31, 2022.
About RGX-121
RGX-121 is designed to use the NAV 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 and consequent reduction of GAGs. RGX-121 has received orphan drug product, rare pediatric disease and Fast Track designations from the U.S. Food and Drug Administration.
RGX-121 is being evaluated in two on-going studies, CAMPSIITE, a Phase I/II/III multicenter, open-label clinical trial of patients up to five years old with MPS II and a Phase I/II multicenter, open-label clinical trial in patients over the age of five years old with MPS II.
About REGENXBIO Inc.
REGENXBIO is a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy. REGENXBIO's NAV Technology Platform, a proprietary adeno-associated virus (AAV) gene delivery platform, consists of exclusive rights to more than 100 novel AAV vectors, including AAV7, AAV8, AAV9 and AAVrh10. REGENXBIO and its third-party NAV Technology Platform Licensees are applying the NAV Technology Platform in the development of a broad pipeline of candidates, including late-stage and commercial programs, in multiple therapeutic areas. REGENXBIO is committed to a "5x'25" strategy to progress five AAV Therapeutics from our internal pipeline and licensed programs into pivotal-stage or commercial products by 2025.
View original content to download multimedia:https://www.prnewswire.com/news-releases/regenxbio-announces-intention-to-file-a-biologics-license-application-using-the-accelerated-approval-pathway-for-rgx-121-an-aav-therapeutic-for-the-treatment-of-mps-ii-301598783.html
SOURCE REGENXBIO Inc.
Regenxbio to seek FDA's accelerated approval for Hunter Syndrome treatment, shares rise 8%
Aug. 03, 2022 10:02 AM ET
By: Anuron Mitra, SA News Editor
- Regenxbio (NASDAQ:RGNX) on Wednesday said it intends to file a biologics license application (BLA) in 2024 using the U.S. FDA's accelerated approval process for its RGX-121 treatment candidate for Mucopolysaccharidosis Type II (MPS II).
- https://seekingalpha.com/news/3865767-regenxbio-to-seek-fdas-accelerated-approval-for-hunter-syndrome-treatment-shares-rise-8
- https://seekingalpha.com/symbol/RGNX
- https://regenxbio.com/
ONPATTRO® (Patisiran)
HyBryte™ (research name SGX301)
ONPATTRO® (Patisiran)
Alnylam Reports Positive Topline Results from APOLLO-B Phase 3 Study of Patisiran in Patients with ATTR Amyloidosis with Cardiomyopathy
Aug 03, 2022
– Patisiran Met the Primary Endpoint with a Statistically Significant Improvement in 6-Minute Walk Test Compared to Placebo at 12 Months –
– Patisiran Also Met the First Secondary Endpoint with a Statistically Significant Improvement in Quality of Life, as Measured by the Kansas City Cardiomyopathy Questionnaire, Compared to Placebo at 12 Months –
– Patisiran Demonstrated Encouraging Safety and Tolerability Profile in Patients with ATTR Amyloidosis with Cardiomyopathy –
– Company Plans to File a Supplemental New Drug Application in U.S. in Late 2022 –
– Full Data Will Be Presented at the 18th International Symposium on Amyloidosis –
– Alnylam to Host Conference Call Today at 8:00 am ET –
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Aug. 3, 2022-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the APOLLO-B Phase 3 study of patisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy, met the primary endpoint of change from baseline in the 6-Minute Walk Test (6-MWT) at 12 months compared to placebo (p-value 0.0162). The study also met the first secondary endpoint of change from baseline in quality of life compared to placebo, as measured by the Kansas City Cardiomyopathy Questionnaire (KCCQ) (p-value 0.0397).
The study also included additional secondary composite outcome endpoints to be tested in a hierarchical manner. A non-significant result (p-value 0.0574) was found on the secondary composite endpoint of all-cause mortality, frequency of cardiovascular events, and change from baseline in 6-MWT over 12 months compared to placebo. As a result, formal statistical testing was not performed on the final two composite endpoints, which were not powered for statistical significance given the short duration of the study — all-cause mortality and frequency of all-cause hospitalizations and urgent heart failure visits in patients not on tafamidis at baseline (nominal p-value 0.9888), and in the overall population (nominal p-value 0.5609). Patisiran also demonstrated an encouraging safety and tolerability profile, with deaths numerically favoring the patisiran arm.
“We are thrilled that APOLLO-B successfully met all its major objectives, which we believe for the first time validates the hypothesis that TTR silencing by an RNAi therapeutic can be an effective approach for treating the cardiomyopathy of ATTR amyloidosis,” said Pushkal Garg, M.D., Chief Medical Officer of Alnylam. “ATTR amyloidosis with cardiomyopathy is an increasingly recognized cause of heart failure, affecting greater than 250,000 patients around the world. These patients have limited treatment options, and disease progression is common. As such, we are encouraged to see the potential of patisiran to improve the functional capacity and quality of life of patients living with this fatal, multi-system disease. I want to thank all the patients, caregivers, investigators, and study staff who have and continue to participate in APOLLO-B. We look forward to sharing full results at an upcoming conference in September, and based on these positive results, we plan to submit a supplemental NDA for patisiran with the U.S. Food and Drug Administration in late 2022.”
APOLLO-B is a Phase 3, randomized, double-blind, placebo-controlled multicenter global study designed and powered to evaluate the effects of patisiran on functional capacity and quality of life in patients with ATTR amyloidosis with cardiomyopathy. The study enrolled 360 adult patients with ATTR amyloidosis (hereditary or wild-type) with cardiomyopathy at 69 sites in 21 countries. Patients were randomized 1:1 to receive 0.3 mg/kg of patisiran or placebo intravenously administered every three weeks over a 12-month double-blind treatment period. After 12 months, all patients will receive patisiran in an open-label extension period.
The primary endpoint of APOLLO-B is the change from baseline in the 6-MWT at 12 months compared to placebo. The secondary endpoints evaluate the efficacy of patisiran vs. placebo over 12 months in a hierarchical manner with the following measures:
- Health-related quality of life with the KCCQ change from baseline at 12 months;
- Composite of all-cause mortality, frequency of cardiovascular (CV) events (CV hospitalizations and urgent heart failure (HF) visits) and change from baseline in 6-MWT;
- Composite of all-cause mortality and frequency of all-cause hospitalizations and urgent HF visits in patients not on tafamidis at baseline; and
- Composite of all-cause mortality and frequency of all-cause hospitalizations and urgent HF visits in the overall study population.
Exploratory endpoints included cardiac biomarkers and various imaging tools to further characterize the potential burden of cardiac involvement in these patients.
The overall safety profile of patisiran during the 12-month double-blind period was encouraging.
- 5 patients (2.8 percent) on patisiran and 8 patients (4.5 percent) on placebo died. Furthermore, the number of deaths in the all-cause mortality efficacy analysis was 4 (2.2 percent) in the patisiran arm and 10 (5.6 percent) in the placebo arm, determined in accordance with the pre-defined statistical analysis plan, which excluded death due to COVID-19, and treated cardiac transplant as a death event consistent with other studies in the field.
- The patisiran and placebo arms had similar frequencies of adverse events (AEs) (91.2 percent and 94.4 percent, respectively) and serious adverse events (SAEs) (33.7 percent and 35.4 percent, respectively). AEs reported in greater than or equal to 5 percent of patisiran patients and seen at least 3 percent more frequently with patisiran compared with placebo were infusion-related reactions (12.2 percent vs. 9 percent, respectively), arthralgia (7.7 percent vs. 4.5 percent, respectively), and muscle spasms (6.6 percent vs. 2.2 percent, respectively). No SAEs occurred at least 2 percent more frequently in patisiran versus placebo treated patients.
“I am delighted by the results of the APOLLO-B study, which suggest the potential for patisiran to be a treatment option for patients with ATTR amyloidosis with cardiomyopathy, assuming favorable regulatory review. In addition, the APOLLO-B data further strengthen our confidence in our Phase 3 HELIOS-B study of vutrisiran in ATTR amyloidosis with cardiomyopathy, which is expected to report out in early 2024,” said Yvonne Greenstreet, MBChB, Chief Executive Officer of Alnylam. “Today’s positive results advance our goal to establish an industry leading TTR franchise, which currently includes ONPATTRO® and AMVUTTRATM for the polyneuropathy of hereditary ATTR amyloidosis. We believe these data take us one step closer to achieving our Alnylam P5x25 vision of becoming a leading biopharma company.”
Full results of the APOLLO-B study will be presented as part of a late-breaker session at the 18th International Symposium on Amyloidosis on September 8, 2022, in Heidelberg, Germany.
Patisiran is the established name for ONPATTRO, which is approved in the United States and Canada for the treatment of the polyneuropathy of hATTR amyloidosis in adults. ONPATTRO is also approved in the European Union, Switzerland and Brazil for the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, and in Japan for the treatment of hATTR amyloidosis with polyneuropathy.
About ONPATTRO® (Patisiran)
ONPATTRO is an RNAi therapeutic that is approved in the United States and Canada for the treatment of the polyneuropathy of hATTR amyloidosis in adults. ONPATTRO is also approved in the European Union, Switzerland and Brazil for the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, and in Japan for the treatment of hATTR amyloidosis with polyneuropathy. ONPATTRO is an intravenously administered RNAi therapeutic targeting transthyretin (TTR). It is designed to target and silence TTR messenger RNA, thereby reducing the production of TTR protein before it is made. Reducing the pathogenic protein leads to a reduction in amyloid deposits in tissues. For more information about ONPATTRO, including full Prescribing Information, visit ONPATTRO.com.
ONPATTRO Indication and ISI
Indication
ONPATTRO is indicated for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
Important Safety Information
Infusion-Related Reactions
Infusion-related reactions (IRRs) have been observed in patients treated with ONPATTRO. In a controlled clinical study, 19% of ONPATTRO-treated patients experienced IRRs, compared to 9% of placebo-treated patients. The most common symptoms of IRRs with ONPATTRO were flushing, back pain, nausea, abdominal pain, dyspnea, and headache.
To reduce the risk of IRRs, patients should receive premedication with a corticosteroid, acetaminophen, and antihistamines (H1 and H2 blockers) at least 60 minutes prior to ONPATTRO infusion. Monitor patients during the infusion for signs and symptoms of IRRs. If an IRR occurs, consider slowing or interrupting the infusion and instituting medical management as clinically indicated. If the infusion is interrupted, consider resuming at a slower infusion rate only if symptoms have resolved. In the case of a serious or life-threatening IRR, the infusion should be discontinued and not resumed.
Reduced Serum Vitamin A Levels and Recommended Supplementation
ONPATTRO treatment leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking ONPATTRO. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with ONPATTRO, as serum levels do not reflect the total vitamin A in the body.
Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g. night blindness).
Adverse Reactions
The most common adverse reactions that occurred in patients treated with ONPATTRO were upper respiratory tract infections (29%) and infusion‑related reactions (19%).
About Alnylam Pharmaceuticals
Alnylam (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding 20 years ago, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam’s commercial RNAi therapeutic products are ONPATTRO® (patisiran), GIVLAARI® (givosiran), OXLUMO® (lumasiran), AMVUTTRA™ (vutrisiran) and Leqvio® (inclisiran) being developed and commercialized by Alnylam’s partner, Novartis. Alnylam has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P5x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam, on LinkedIn, or on Instagram.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220803005528/en/
Alnylam Pharmaceuticals, Inc.
Source: Alnylam Pharmaceuticals, Inc.
Alnylam soars 42% as Phase 3 trial for ATTR amyloidosis therapy meets main goal
Aug. 03, 2022 9:33 AM ET
Alnylam Pharmaceuticals, Inc. (ALNY)
By: Dulan Lokuwithana, SA News Editor1 Comment
Alnylam Pharmaceuticals (NASDAQ:ALNY) added nearly 42% in the morning hours Wednesday after the RNAi therapeutics company said that its Phase 3 trial for patisiran reached the primary goal in transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy.
https://seekingalpha.com/symbol/ALNY
HyBryte™ (research name SGX301)
HyBryte™ (research name SGX301)
Tofersen (formerly IONIS-SOD1Rx), also known as BIIB067
Soligenix Receives Agreement from FDA on Initial Pediatric Study Plan for HyBryte™ for the Treatment of Cutaneous T-Cell Lymphoma
PRINCETON, N.J., July 27, 2022 /PRNewswire/ -- Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced today that it has received agreement from the US Food & Drug Administration (FDA) on an initial pediatric study plan (iPSP) for HyBryte™ (synthetic hypericin) for the treatment of cutaneous T-cell lymphoma (CTCL). The agreed iPSP stipulates that Soligenix intends on requesting a full waiver of pediatric studies upon submission of a new drug application (NDA). Agreement with FDA on an iPSP is one of the regulatory requirements that must be met prior to submitting a NDA.
"We are pleased to have FDA's agreement on our proposal to request a full waiver of pediatric studies at the time of our HyBryte™ NDA filing later this year," stated Christopher J. Schaber, PhD, President & Chief Executive Officer of Soligenix. "This is consistent with decisions by the European Medicines Agency (EMA) and Medicines and Healthcare products Regulatory Agency (MHRA) in the United Kingdom which have previously granted product-specific waivers from the requirement for pediatric studies in applications for marketing authorization of HyBryte™ in the UK and Europe."
About HyBryte™
HyBryte™ (research name SGX301) is a novel, first-in-class, photodynamic therapy utilizing safe, visible light for activation. The active ingredient in HyBryte™ is synthetic hypericin, a potent photosensitizer that is topically applied to skin lesions that is taken up by the malignant T-cells, and then activated by visible light 16 to 24 hours later. The use of visible light in the red-yellow spectrum has the advantage of penetrating more deeply into the skin (much more so than ultraviolet light) and therefore potentially treating deeper skin disease and thicker plaques and lesions. This treatment approach avoids the risk of secondary malignancies (including melanoma) inherent with the frequently employed DNA-damaging drugs and other phototherapy that are dependent on ultraviolet exposure. Combined with photoactivation, hypericin has demonstrated significant anti-proliferative effects on activated normal human lymphoid cells and inhibited growth of malignant T-cells isolated from CTCL patients. In a published Phase 2 clinical study in CTCL, patients experienced a statistically significant (p=0.04) improvement with topical hypericin treatment whereas the placebo was ineffective. HyBryte™ has received orphan drug and fast track designations from the FDA, as well as orphan designation from the European Medicines Agency (EMA).
The recently published Phase 3 FLASH trial trial enrolled a total of 169 patients (166 evaluable) with Stage IA, IB or IIA CTCL. The trial consisted of three treatment cycles. Treatments were administered twice weekly for the first 6 weeks and treatment response was determined at the end of the 8th week of each cycle. In the first double-blind treatment cycle, 116 patients received HyBryte™ treatment (0.25% synthetic hypericin) and 50 received placebo treatment of their index lesions. A total of 16% of the patients receiving HyBryte™ achieved at least a 50% reduction in their lesions (graded using a standard measurement of dermatologic lesions, the CAILS score) compared to only 4% of patients in the placebo group at 8 weeks (p=0.04) during the first treatment cycle (primary endpoint). HyBryte™ treatment in the first cycle was safe and well tolerated.
In the second open-label treatment cycle (Cycle 2), all patients received HyBryte™ treatment of their index lesions. Evaluation of 155 patients in this cycle (110 receiving 12 weeks of HyBryte™ treatment and 45 receiving 6 weeks of placebo treatment followed by 6 weeks of HyBryte™ treatment), demonstrated that the response rate among the 12-week treatment group was 40% (p<0.0001 vs the placebo treatment rate in Cycle 1). Comparison of the 12-week and 6-week treatment groups also revealed a statistically significant improvement (p<0.0001) between the two groups, indicating that continued treatment results in better outcomes. HyBryte™ continued to be safe and well tolerated. Additional analyses also indicated that HyBryte™ is equally effective in treating both plaque (response 42%, p<0.0001 relative to placebo treatment in Cycle 1) and patch (response 37%, p=0.0009 relative to placebo treatment in Cycle 1) lesions of CTCL, a particularly relevant finding given the historical difficulty in treating plaque lesions in particular.
The third (optional) treatment cycle (Cycle 3) was focused on safety and all patients could elect to receive HyBryte™ treatment of all their lesions. Of note, 66% of patients elected to continue with this optional compassionate use / safety cycle of the study. Of the subset of patients that received HyBryte™ throughout all 3 cycles of treatment, 49% of them demonstrated a positive treatment response (p<0.0001 vs patients receiving placebo in Cycle 1). Moreover, in a subset of patients evaluated in this cycle, it was demonstrated that HyBryte™ is not systemically available, consistent with the general safety of this topical product observed to date. At the end of Cycle 3, HyBryte™ continued to be well tolerated despite extended and increased use of the product to treat multiple lesions.
Overall safety of HyBryte™ is a critical attribute of this treatment and was monitored throughout the three treatment cycles (Cycles 1, 2 and 3) and the 6-month follow-up period. HyBryte's™ mechanism of action is not associated with DNA damage, making it a safer alternative than currently available therapies, all of which are associated with significant and sometimes fatal, side effects. Predominantly these include the risk of melanoma and other malignancies, as well as the risk of significant skin damage and premature skin aging. Currently available treatments are only approved in the context of previous treatment failure with other modalities and there is no approved front-line therapy available. Within this landscape, treatment of CTCL is strongly motivated by the safety risk of each product. HyBryte™ potentially represents the safest available efficacious treatment for CTCL. With no systemic absorption, a compound that is not mutagenic and a light source that is not carcinogenic, there is no evidence to date of any potential safety issues.
The Phase 3 CTCL clinical study was partially funded by the National Cancer Institute via a Phase II SBIR grant (#1R44CA210848-01A1) awarded to Soligenix, Inc.
About Soligenix, Inc.
Soligenix is a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need. Our Specialized BioTherapeutics business segment is developing and moving toward potential commercialization of HyBryte™ (SGX301 or synthetic hypericin) as a novel photodynamic therapy utilizing safe visible light for the treatment of cutaneous T-cell lymphoma (CTCL). With a successful Phase 3 study completed, regulatory approval is being sought and commercialization activities for this product candidate are being advanced initially in the U.S. Development programs in this business segment also include our first-in-class innate defense regulator (IDR) technology, dusquetide (SGX942) for the treatment of inflammatory diseases, including oral mucositis in head and neck cancer, and proprietary formulations of oral beclomethasone 17,21-dipropionate (BDP) for the prevention/treatment of gastrointestinal (GI) disorders characterized by severe inflammation including pediatric Crohn's disease (SGX203).
Our Public Health Solutions business segment includes active development programs for RiVax®, our ricin toxin vaccine candidate, and SGX943, our therapeutic candidate for antibiotic resistant and emerging infectious disease, and our vaccine programs targeting filoviruses (such as Marburg and Ebola) and CiVax™, our vaccine candidate for the prevention of COVID-19 (caused by SARS-CoV-2). The development of our vaccine programs incorporates the use of our proprietary heat stabilization platform technology, known as ThermoVax®. To date, this business segment has been supported with government grant and contract funding from the National Institute of Allergy and Infectious Diseases (NIAID), the Defense Threat Reduction Agency (DTRA) and the Biomedical Advanced Research and Development Authority (BARDA).
For further information regarding Soligenix, Inc., please visit the Company's website at https://www.soligenix.com and follow us on LinkedIn and Twitter at @Soligenix_Inc.
SOURCE Soligenix, Inc.
Soligenix gets agreement from FDA for photodynamic therapy study in kids for lymphoma
Jul. 27, 2022 8:22 AM ET
By: Ravikash, SA News Editor
- Soligenix (NASDAQ:SNGX) said it received agreement from the U.S. Food & Drug Administration (FDA) on an initial pediatric study plan ((iPSP)) of its photodynamic therapy HyBryte (synthetic hypericin) to treat cutaneous T-cell lymphoma (CTCL).
- https://seekingalpha.com/news/3861272-soligenix-gets-agreement-from-fda-for-photodynamic-therapy-study-in-kids-for-lymphoma
- https://seekingalpha.com/symbol/SNGX
- https://www.soligenix.com/pipeline-programs/
- https://www.soligenix.com/
- https://www.hybryte.com/
Tofersen (formerly IONIS-SOD1Rx), also known as BIIB067
Tofersen (formerly IONIS-SOD1Rx), also known as BIIB067
Tofersen (formerly IONIS-SOD1Rx), also known as BIIB067
FDA Accepts Biogen’s New Drug Application and Grants Priority Review of Tofersen for a Rare, Genetic Form of ALS
JULY 26, 2022 • INVESTOR RELATIONS
- SOD1-ALS is a rare genetic form of ALS that affects approximately 330 people in the U.S.,1 it is progressive, leads to the loss of everyday functions and is uniformly fatal
- If approved, tofersen would be the first treatment to target a genetic cause of ALS
- 12-month data included in the filing show that earlier initiation of tofersen slowed decline across measures of clinical and respiratory function, strength, and quality of life
CAMBRIDGE, Mass., July 26, 2022 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) today announced that the U.S. Food and Drug Administration (FDA) has accepted a New Drug Application (NDA) for tofersen, an investigational drug for superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS). The application has been granted priority review and given a Prescription Drug User Fee Act action date of January 25, 2023. The FDA has noted that it is currently planning to hold an Advisory Committee meeting for this application, on a yet-to-be determined date. The average life expectancy for people with ALS is three to five years from time of symptom onset. There is currently no treatment targeted for SOD1-ALS.2
“The available data show that tofersen has the potential to make a meaningful difference for people with SOD1-ALS,” said Priya Singhal, M.D., M.P.H., Head of Global Safety and Regulatory Sciences and Interim Head of R&D at Biogen. “Pursuing the FDA’s accelerated approval pathway offers the potential to make tofersen available to people living with this fatal, neurodegenerative disease as quickly as possible. If approved, tofersen will be the first treatment to target a genetic cause of ALS and we hope this will pave the way for further advances in this relentless disease.”
Biogen is seeking approval of tofersen under the FDA’s accelerated approval pathway, based on the use of neurofilament as a surrogate biomarker that is reasonably likely to predict clinical benefit. Neurofilaments are normal proteins found in healthy neurons, that are increased in blood and cerebrospinal fluid when damage has been done to neurons or their axons and are a marker of neurodegeneration. In ALS, higher levels of neurofilaments have been found to predict more rapid decline in clinical function and shortened survival.3 Tofersen study results suggest reductions in neurofilament preceded and predicted slowing of decline in measures of clinical and respiratory function, strength, and quality of life. Biogen is committed to ongoing data generation and finalizing the confirmatory data package with the FDA.
“The 12-month results showed that individuals with SOD1-ALS who started tofersen earlier experienced a slower rate of decline in clinical and respiratory function, strength and quality of life. These are critical measures for people living with this devastating disease,” said Timothy Miller, M.D., Ph.D., principal investigator of VALOR and ALS Center co-Director at Washington University School of Medicine, St. Louis. “For people in my clinic living with SOD1-ALS, tofersen may meaningfully slow the rapid progression of their disease and the impact it has on their lives.”
The tofersen NDA included results from a Phase 1 study in healthy volunteers, a Phase 1/2 study evaluating ascending dose levels, the Phase 3 VALOR study, and the open label extension (OLE) study. Also included are the most current 12-month integrated results from VALOR and the OLE study, recently presented at the European Network to Cure ALS (ENCALS) annual meeting.
As previously reported in October 2021, VALOR, a six-month Phase 3 randomized study, did not meet the primary endpoint of change from baseline to week 28 in the Revised Amyotrophic Lateral Sclerosis Functional Rating Scale. However, trends of reduced disease progression across multiple secondary and exploratory endpoints were observed. The 12-month integrated data showed that earlier initiation of tofersen led to sustained reductions in neurofilament, a marker of neurodegeneration and slowed decline across multiple efficacy endpoints.
In the 12-month data, the most common adverse events (AEs) in participants receiving tofersen in VALOR and the OLE study were headache, procedural pain, fall, back pain and pain in extremities. Most AEs in both VALOR and the OLE were mild to moderate in severity. Serious neurologic events including myelitis, radiculitis, aseptic meningitis, and papilledema, were reported in 6.7 percent of participants receiving tofersen in VALOR and its OLE.
During the FDA review period Biogen will maintain its early access program for tofersen, now with participants in over a dozen countries. The open-label extension and Phase 3 ATLAS study in presymptomatic individuals with a SOD1 genetic mutation remain ongoing. Biogen is actively engaging with other regulators around the world and will provide updates when appropriate.
About Tofersen
Tofersen is an antisense drug being evaluated for the potential treatment of SOD1-ALS. Tofersen binds to SOD1 mRNA, allowing for its degradation by RNase-H in an effort to reduce synthesis of SOD1 protein production. In addition to the ongoing open label extension of VALOR, tofersen is being studied in the Phase 3 ATLAS study designed to evaluate whether tofersen can delay clinical onset when initiated in presymptomatic individuals with a SOD1 genetic mutation and biomarker evidence of disease activity. Biogen licensed tofersen from Ionis Pharmaceuticals, Inc. under a collaborative development and license agreement.
About Biogen
As pioneers in neuroscience, Biogen discovers, develops, and delivers worldwide innovative therapies for people living with serious neurological diseases as well as related therapeutic adjacencies. One of the world’s first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Sir Kenneth Murray, and Nobel Prize winners Walter Gilbert and Phillip Sharp. Today, Biogen has a leading portfolio of medicines to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, and developed the first and only approved treatment to address a defining pathology of Alzheimer’s disease. Biogen is also commercializing biosimilars and focusing on advancing one of the industry’s most diversified pipelines in neuroscience that will transform the standard of care for patients in several areas of high unmet need.
In 2020, Biogen launched a bold 20-year, $250 million initiative to address the deeply interrelated issues of climate, health, and equity. Healthy Climate, Healthy Lives™ aims to eliminate fossil fuels across the company’s operations, build collaborations with renowned institutions to advance the science to improve human health outcomes, and support underserved communities.
We routinely post information that may be important to investors on our website at www.biogen.com. Follow us on social media - Twitter, LinkedIn, Facebook, YouTube.
Biogen/Ionis ALS therapy tofersen gets FDA priority review
Jul. 26, 2022 9:14 AM ET
By: Ravikash, SA News Editor1 Comment
The U.S. Food and Drug Administration (FDA) granted priority review to Biogen's (NASDAQ:BIIB) and Ionis' (NASDAQ:IONS) tofersen to treat patients with superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS).
https://seekingalpha.com/news/3860495-biogenionis-als-therapy-tofersen-gets-fda-priority-review
https://seekingalpha.com/symbol/BIIB
https://seekingalpha.com/symbol/IONS
TRUSELTIQ (infigratinib) capsules
Tofersen (formerly IONIS-SOD1Rx), also known as BIIB067
TRUSELTIQ (infigratinib) capsules
BridgeBio Pharma Announces Positive Interim Results from a Phase 2 Trial of Infigratinib in Achondroplasia Demonstrating an Increase in Annualized Height Velocity of 1.52 cm/year in Children 5 Years of Age and Older, and Adds 5th Cohort to Trial
– At the highest dose level evaluated to date (Cohort 4, 0.128 mg/kg once daily), the mean change from baseline in annualized height velocity (AHV) was +1.52 cm/yr (p=0.02, n=11) and the responder rate was 64% in children 5 years and older 1
– Infigratinib was well-tolerated with no serious adverse events (SAE) and no discontinuations due to adverse events (AE) including in Cohort 5 (dose: 0.25 mg/kg once daily) participants dosed to date, with a median duration of follow-up of 48.1 weeks; only a limited number of AEs were assessed as related to study drug and all were Grade 1, the lowest level
– Given infigratinib’s profile to date, and after discussions with regulators, BridgeBio has begun dosing children in Cohort 5 (dose: 0.25 mg/kg once daily)
– Infigratinib is the only known oral product candidate currently under clinical investigation for achondroplasia, with granted and filed patent applications expiring as late as 2041
PALO ALTO, CA – July 26, 2022 — BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced positive interim results from PROPEL 2, a Phase 2 trial of the investigational therapy infigratinib in children with achondroplasia. Achondroplasia (ACH) is the most common cause of disproportionate short stature. Achondroplasia impacts health and can lead to medical complications such as obstructive sleep apnea, middle ear dysfunction, kyphosis, and spinal stenosis. Achondroplasia affects approximately 55,000 people in the United States (US) and Europe, including up to 13,000 children and adolescents with open growth plates. The condition is uniformly caused by an activating mutation in fibroblast growth factor receptor 3 (FGFR3). Infigratinib is an oral small molecule that inhibits FGFR3, therefore designed to target this well-described disease at its source.
At the highest dose level evaluated to date (0.128 mg/kg once daily), mean increase in annualized height velocity (AHV) was 1.52 cm/yr over baseline (p=0.02, n=11) for all follow-up data available at time of data cut in children 5 years of age and older. In this group, 64% were responders (defined with a strict criterion of an increase ≥25% in AHV from baseline) and the average percent change from baseline in AHV was 60%.
Earlier cohorts in PROPEL 2 did not achieve the target efficacious exposure as suggested by our preclinical data and no dose response was observed. An increase in AHV over baseline of 0.22 cm/yr (p=0.54, n=38) was observed across these earlier combined cohorts for children 5 years of age and older.
PROPEL 2 enrolled children as young as 3 years of age in the study. Consistent with other trials in the younger age population, both AHV and height Z-scores were analyzed to help control for greater variability in growth seen by age and sex in children 3 to <5 years old. In this age group, the average increase in ACH height Z-score at 6 months was 0.21 standard deviation score (SDS) and an increase in AHV of 0.61 cm/yr over baseline (n=5) was observed. Across Cohort 4, the median duration of follow-up was 26.9 weeks at time of data cut.
“These interim data demonstrate compelling initial proof-of-concept for an oral FGFR inhibitor in children with achondroplasia. We are encouraged by what we have seen to date, including the overall safety profile and promising initial efficacy data of infigratinib. We look forward to completing enrollment in Cohort 5 with the goal of presenting the full study results next year,” said Professor Ravi Savarirayan, M.D., Ph.D., clinical geneticist and group leader of skeletal therapies research at the Murdoch Children’s Research Institute in Australia, the lead investigator for PROPEL 2.
Median follow-up across the entire study is 48.1 weeks across 62 participants included in the safety population at time of data cut. No treatment-related SAEs have been reported to date in any cohort. 90.3% of participants experienced at least one treatment-emergent adverse event (TEAE), the majority of which were Grade 1, unrelated to study drug, and consistent with a pediatric achondroplasia population. Only 9.7% of participants had a TEAE related to study drug, all of which were Grade 1, and included dyspepsia, decreased appetite, flatulence, hypercholesterolemia, hyperphosphatemia, and vitamin D decrease.
A single case of mild hyperphosphatemia (Grade 1) led to a dose reduction in a participant in Cohort 3 (dose: 0.064 mg/kg once daily), the only dose adjustment made to date in the study. Phosphorus levels for this participant only exceeded upper limit of normal by <10% and returned to and remain normal at the reduced dose (0.032 mg/kg once daily). No other cases of hyperphosphatemia have been observed in any other cohort (including Cohort 5) to date, and no trends in phosphorous by dose have been observed. No other AEs required dose modifications or interruptions.
No children have discontinued treatment as the result of an adverse event. No bone-related AEs were observed to date.
“I am encouraged by this interim efficacy and safety data and thankful, as ever, to be partnered with the healthcare providers, children, and families who are making this study possible. These data, combined with our positive proof-of-concept data in LGMD2i and ADH1 earlier this year, highlight BridgeBio’s ability to efficiently prosecute high value programs in large areas of unmet need. We look forward to exploring the potential of infigratinib in achondroplasia and related skeletal dysplasias in the near future,” said Neil Kumar, Ph.D., founder and CEO of BridgeBio.
BridgeBio, after discussions with regulatory agencies, has begun enrolling Cohort 5. Cohort 5 participants are receiving approximately twice the dose of Cohort 4 (0.25 mg/kg once daily vs 0.128 mg/kg once daily in Cohort 4). At the conclusion of the ongoing trial, BridgeBio intends to present full data at a medical conference in the first half of 2023. Additionally, BridgeBio expects to evaluate development of infigratinib in other FGFR-driven skeletal dysplasias, which affect more than 50,000 people in the US and Europe, building on this positive interim data from PROPEL 2 as well as preclinical data in hypochondroplasia presented at the Endocrine Society (ENDO) 2022 Annual Conference earlier this year.
Infigratinib is not approved in any country for the treatment of children or adults with achondroplasia. For more information on infigratinib, please see the following link.
About BridgeBio Pharma, Inc.
BridgeBio Pharma (BridgeBio) is a commercial-stage biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio’s pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter.
BridgeBio up 11% following positive mid-stage results of infigratinib in achondroplasia
Jul. 26, 2022 9:27 AM ET
By: Jonathan Block, SA News Editor
- Shares of BridgeBio Pharma (NASDAQ:BBIO) are up 11% in premarket trading after reporting positive phase 2 results for infigratinib in children with achondroplasia.
- https://seekingalpha.com/news/3860508-bridgebio-up-11-following-positive-mid-stage-results-of-infigratinib-in-achondroplasia
- https://seekingalpha.com/symbol/BBIO
- https://www.truseltiq.com/
- https://www.truseltiq.com/pdfs/prescribing-information.pdf
- https://bridgebio.com/
TX200
Tofersen (formerly IONIS-SOD1Rx), also known as BIIB067
TRUSELTIQ (infigratinib) capsules
European Orphan Medicinal Product Designation Granted to Sangamo Therapeutics Investigational CAR-Treg Cell Therapy TX200 for Solid Organ Transplantation
July 21, 2022 at 8:05 AM EDTPDF Version
BRISBANE, Calif.--(BUSINESS WIRE)--Jul. 21, 2022-- Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicine company, today announced that the European Commission (EC) has granted Orphan Medicinal Product Designation (OMPD) to TX200, a wholly-owned autologous Chimeric Antigen Receptor Regulatory T Cell (CAR-Treg) cell therapy product candidate for treatment in solid organ transplantation.
“Patients who have received a solid organ transplant require lifelong surveillance and chronic immunosuppressive medications to manage the risk of transplant rejection,” said Rob Schott, MD, MPH, FACC, Head of Development for Sangamo. “Our goal with TX200 is to create a transformative therapy that reduces the risk of organ rejection, while reducing the patient burden from chronic immunosuppressive therapy. Achieving this important regulatory milestone takes us one step closer to realizing that goal for patients.”
Dosing of the first patient in the STEADFAST Phase 1/2 clinical study took place in March 2022, with the second patient dosing planned later this quarter.
The EC granted OMPD to TX200 following a positive opinion from the European Medicines Agency’s Committee for Orphan Medicinal Products. To qualify for orphan designation, a treatment must be intended for a life-threatening or chronically debilitating disease affecting fewer than 5 in 10,000 people. Importantly, no satisfactory method of treatment must exist, or if such a method exists, the treatment must be of significant benefit to patients.
The OMPD status offers a range of incentives to encourage the development of orphan medicines, including protocol assistance on study protocols, potential fee reductions, and 10 years of market exclusivity upon regulatory approval.
About TX200
TX200 is a cell therapy composed of autologous regulatory T cells (Tregs) engineered to express an HLA-A2 Chimeric Antigen Receptor (CAR), with the aim of preventing immune-mediated rejection in HLA-A2 mismatched kidney transplants from a living donor. TX200 is intended to reduce the risk of transplant rejection by suppressing local inflammation and promoting immunological tolerance to the graft. TX200 is being assessed in HLA-A2 negative patients receiving a mismatched HLA-A2 positive kidney from a living donor. TX200 CAR-Treg cells are expected to localize to the graft and activate upon binding to the HLA-A2 antigen. Through their ability to regulate the immune system, TX200 cells may protect the graft from immune-mediated rejection and reduce or eliminate the need for lifelong treatment with immunosuppressants. The first patient in the ongoing STEADFAST Phase 1/2 study was dosed in March 2022.
About Sangamo Therapeutics
Sangamo Therapeutics is a clinical-stage biopharmaceutical company with a robust genomic medicines pipeline. Using ground-breaking science, including our proprietary zinc finger genome engineering technology and manufacturing expertise, Sangamo aims to create new genomic medicines for patients suffering from diseases for which existing treatment options are inadequate or currently don’t exist. For more information about Sangamo, visit www.sangamo.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220721005330/en/
Source: Sangamo Therapeutics, Inc.
Sangamo's TX200 gets orphan drug tag in EU for use in organ transplant
Jul. 21, 2022 8:30 AM ET
Sangamo Therapeutics, Inc. (SGMO)
By: Ravikash, SA News Editor1 Comment
The European Commission (EC) granted orphan medicinal product designation to Sangamo Therapeutics' (NASDAQ:SGMO) CAR-Treg cell therapy TX200 for treatment in solid organ transplantation.
- https://seekingalpha.com/news/3859037-sangamos-tx200-gets-orphan-drug-tag-in-eu-for-use-in-organ-transplant
- https://seekingalpha.com/symbol/SGMO
- https://www.sangamo.com/programs/
biotecHOPE™ 2022
Eltanexor (KPT-8602)
Eltanexor (KPT-8602)
Eltanexor (KPT-8602)
Karyopharm Granted Regulatory Designations for Eltanexor for the Treatment of Myelodysplastic Syndromes
– FDA Fast Track Designation and European Commission Orphan Medicinal Product Designation Underscore the Significant Need for New Treatment Options for MDS –
NEWTON, Mass., July 20, 2022 /PRNewswire/ -- Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, today announced new regulatory designations for eltanexor, a novel oral, Selective Inhibitor of Nuclear Export (SINE) investigational compound being studied for the treatment of myelodysplastic syndromes (MDS): (i) the U.S. Food and Drug Administration (FDA) has granted fast track designation for the development program of eltanexor as monotherapy for the treatment of patients with relapsed or refractory intermediate, high-, or very high-risk MDS; (ii) the European Commission (EC) adopted the Committee for Orphan Medicinal Products (COMP) opinion to designate eltanexor as an orphan medicinal product for the treatment of MDS in the European Union (EU). Karyopharm also received orphan drug designation from the FDA in January 2022. MDS are a group of diseases characterized by ineffective production of the components of the blood due to poor bone marrow function with a risk of progression to acute myeloid leukemia.
Karyopharm is currently investigating eltanexor in an ongoing open-label Phase 1/2 study in patients with relapsed/refractory MDS. Previously, Karyopharm reported initial data from the Phase 1 portion of this study evaluating single-agent eltanexor in patients with hypomethylating agent (HMA)-refractory MDS.
Approximately 15,000 people in the U.S.1 and 14,000 people in the EU2 are expected to be diagnosed with intermediate-to-high risk MDS in 2022. HMAs are the current standard of care for newly diagnosed, higher-risk MDS patients. However, only 40-60% of patients respond, with these responses typically lasting less than two years.3 The prognosis in HMA-refractory disease is poor, with a median overall survival of four to six months.4,5 There are currently no approved therapies for HMA- refractory MDS.
"These recent designations from the FDA and EC reinforce eltanexor's potential to improve clinical outcomes for patients with relapsed/refractory MDS," said Richard Paulson, President and Chief Executive Officer of Karyopharm. "We are dedicated to advancing our ongoing clinical trials and remain committed to bringing eltanexor to these patients and their families as a new treatment option."
Fast track is a process designed by the FDA to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. The purpose is to get important new drugs to the patient earlier. Fast Track addresses a broad range of serious conditions. Once a drug receives Fast Track designation, early and frequent communication between the FDA and the drug company is encouraged throughout the entire drug development and review process.
Orphan Medicinal Product Designation is granted by the EC to promote the development of drugs that target rare (less than 5 in 100,000 people across the EU), seriously debilitating and/or life-threatening diseases, and are expected to provide a significant benefit over existing authorized treatments. Orphan designation qualifies a company for certain incentives that apply across all stages of drug development, including the potential for ten years of market exclusivity following marketing approval, fee reductions, and eligibility for orphan drug grants.
About Eltanexor
Eltanexor (KPT-8602) is an investigational novel SINE compound that functions by binding with, and inhibiting, the nuclear export protein, XPO1, leading to the accumulation of tumor suppressor proteins in the cell nucleus. This reinitiates and amplifies their tumor suppressor function and is believed to lead to the selective induction of apoptosis in cancer cells, while largely sparing normal cells.
In preclinical models, eltanexor has a broad therapeutic window with minimal penetration of the blood brain barrier and, therefore, has the potential to serve as another SINE compound for cancer indications. Following oral administration, animals treated with eltanexor show lower percentage of body weight loss and improved food consumption than animals similarly treated with selinexor. This allows more frequent dosing of eltanexor, enabling a longer period of exposure than is possible with selinexor.
Eltanexor is an investigational medicine and has not been approved by the United States Food and Drug Administration or any other regulatory agency.
About Karyopharm Therapeutics
Karyopharm Therapeutics Inc. (Nasdaq: KPTI) is a commercial-stage pharmaceutical company pioneering novel cancer therapies. Since its founding, Karyopharm has been the industry leader in oral Selective Inhibitor of Nuclear Export (SINE) compound technology, which was developed to address a fundamental mechanism of oncogenesis: nuclear export dysregulation. Karyopharm's lead SINE compound and first-in-class, oral exportin 1 (XPO1) inhibitor, XPOVIO® (selinexor), is approved in the U.S. and marketed by the Company in three oncology indications and has received regulatory approvals in various indications in a growing number of ex-U.S. territories and countries, including Europe and the United Kingdom (as NEXPOVIO®) and China. Karyopharm has a focused pipeline targeting multiple high unmet need cancer indications, including in multiple myeloma, endometrial cancer, myelodysplastic syndromes and myelofibrosis. For more information about our people, science and pipeline, please visit www.karyopharm.com, and follow us on Twitter at @Karyopharm and LinkedIn.
SOURCE Karyopharm Therapeutics Inc.
Karyopharm cancer therapy eltanexor gets FDA fast track status, orphan drug tag in EU
Jul. 20, 2022 8:31 AM ET
Karyopharm Therapeutics Inc. (KPTI)
By: Ravikash, SA News Editor
- Karyopharm Therapeutics (NASDAQ:KPTI) said eltanexor was granted fast track designation by the U.S. Food and Drug Administration (FDA) as monotherapy to treat patients with relapsed or refractory intermediate, high-, or very high-risk myelodysplastic syndromes (MDS).
- https://seekingalpha.com/news/3858479-karyopharm-cancer-drug-eltanexor-gets-fda-fast-track-status-orphan-drug-tag-in-eu
- https://www.karyopharm.com/pipeline/oral-eltanexor/
- https://www.karyopharm.com/
- https://seekingalpha.com/symbol/KPTI
Olverembatinib
Eltanexor (KPT-8602)
Eltanexor (KPT-8602)
Innovent and Ascentage Pharma Announce the China NMPA Accepted and Granted Priority Review Designation to a New Drug Application for Olverembatinib for the Treatment of Drug-Resistant CML
Published on: Jul 20th, 2022
SUZHOU, China, SAN FRANCISCO, CA. and ROCKVILLE, MD., U.S., Jul 19, 2022 — Innovent Biologics, Inc. (“Innovent”) (HKEX: 01801), a world-class biopharmaceutical company that develops, manufactures and commercializes high quality medicines for the treatment of oncology, metabolic, autoimmune, ophthalmology and other major diseases, and Ascentage Pharma (6855.HK), a global biopharmaceutical company engaged in developing novel therapies for cancers, chronic hepatitis B (CHB), and age-related diseases, today announce that the Center for Drug Evaluation (CDE) of China National Medical Products Administration (NMPA) has accepted and granted Priority Review designation (in a CDE’s public notice ended on July 18, 2022) to a New Drug Application (NDA) that will support the full approval of olverembatinib in patients with chronic-phase chronic myeloid leukemia (CML-CP) who are resistant and/or intolerant of first- and second-generation tyrosine kinase inhibitors (TKIs). Following the conditional NDA approval in November 2021, the acceptance for the latest application marks another milestone and will potentially bring olverembatinib to benefit a broader population of patients with CML. Innovent and Ascentage are mutually committed to the commercialization of olverembatinib in the China market.
In November 2021, the NMPA granted a conditional approval to olverembatinib for the treatment of adult patients with TKI-resistant CML-CP or CML-AP harboring the T315I mutation as confirmed by a validated diagnostic test, thus filling an urgent treatment gap that has long hindered the clinical outcome in Chinese patients with TKI-resistant CML harboring the T315I mutation.
The acceptance and Priority Review designation for this application are based on the results from an open-label, randomized, controlled, confirmatory Phase II pivotal study (study code: HQP1351CC203) which previously served as the basis for a Breakthrough Therapy designation to olverembatinib by the CDE in March 2021. The study is designed to evaluate the efficacy and safety of olverembatinib in patients with CML-CP who are resistant and/or intolerant to first- and second-generation TKIs, with the event-free survival (EFS) as its primary endpoint. A total of 144 patients were enrolled and randomized to either receive olverembatinib or the control group to receive the current best available treatment (BAT). Results show that olverembatinib significantly improved the EFS compared to the control group and has met the pre-specified superiority criteria. Detailed results from this study will be released at an upcoming academic conference.
CML is a hematologic malignancy of the white blood cells. The introduction of BCR-ABL TKIs has significantly improved the clinical practice and management of CML. However, acquired resistance to TKIs remains a major challenge in the treatment of CML. BCR-ABL tyrosine kinase mutations represent a key mechanism of acquired drug resistance and there is an urgent unmet medical need for a safe and effective treatment. Olverembatinib is a potentially global best-in-class drug that has been developed by Ascentage Pharma and supported by the National Major New Drug Discovery and Manufacturing Program in China. As the first and only third-generation BCR-ABL inhibitor approved for the treatment of drug-resistant CML in China, olverembatinib is able to effectively target a spectrum of BCR-ABL mutants, including T315I.
The leading principal investigator of olverembatinib in China, Prof. Xiaojun Huang, MD, Director of the Institute of Hematology, Peking University, Director of the Hematology Department at Peking University People’s Hospital, commented: “The approval for olverembatinib last year has brought a breakthrough therapy for patients with drug-resistant CML harboring the T315I mutation. Meanwhile, many patients who are resistant to first- and second-generation TKIs still lack effective treatment and drug resistance to TKIs and this remains a major clinical challenge in the treatment of CML. In recent years, results from multiple clinical studies have validated olverembatinib’s therapeutic efficacy in CML patients. We hope that the second Priority Review designation granted to olverembatinib will allow more patients with CML to soon benefit from this novel drug.”
Dr. Hui Zhou, Senior Vice President of Innovent, stated: “We are pleased about the acceptance and Priority Review designation for the application for olverembatinib, as it will potentially allow a broader population of patients with TKI-resistant CML to benefit from this novel therapeutic. Innovent has established a robust pipeline and franchise in hematology and we are fully committed to bringing forth more ‘high-quality biopharmaceutical products that are affordable to ordinary people’ and serve the unmet medical needs from Chinese patients as early as possible.”
Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma, commented, “As an innovative biopharmaceutical company dedicated to addressing the unmet medical needs of patients in China and around the world, Ascentage Pharma is forging ahead with its clinical development programs to allow more patients to benefit from our innovative drugs. Having this application for olverembatinib accepted and granted the Priority Review designation by the CDE highlights the authority’s commitment to filling the treatment gap in patients with drug-resistant CML and its strong recognition of olverembatinib’s therapeutic efficacy. We will work closely with the CDE to bring this innovative therapeutic to more patients as soon as possible.”
About Chronic Myeloid Leukemia
Chronic myeloid leukemia (CML) is a malignancy caused by the clonal proliferation of hematopoietic stem cell in the bone marrow. Also referred to as chronic myelocytic leukemia, CML is one of the most common subtypes of chronic leukemia, accounting for 15% of all leukemia cases in adults. According to epidemiology data, the onset of CML in Chinese patients happens at a younger age than that in the West; the median age of onset of CML in China is around 45 – 50 years old, while it is 67 years old in the west.
BCR-ABL TKIs have significantly improved the clinical management of CML. However, acquired resistance to TKIs remains a major challenge in the treatment of CML, while BCR-ABL tyrosine kinase mutations represent a key mechanism of acquired drug resistance.
About Olverembatinib
Developed by Ascentage Pharma with support from the National Major New Drug Discovery and Manufacturing Program in China, the orally active, third-generation BCR-ABL inhibitor olverembatinib is the first China-approved third-generation BCR-ABL inhibitor targeting drug-resistant CML. Olverembatinib can effectively target a spectrum of BCR-ABL mutants, including the T315I mutation.
In November 2021, olverembatinib was approved by the NMPA of China for the treatment of adult patients with TKI-resistant CML-CP or CML-AP harboring the T315I mutation as confirmed by a validated diagnostic test. In March 2021, it was granted the Breakthrough Therapy designation by the CDE for the treatment of patients with CML-CP who are resistant and/or intolerant of first- and second-generation TKIs.
In overseas, olverembatinib was cleared by the US FDA in July 2019 to directly enter a Phase Ib study. Since 2018, the clinical results of olverembatinib have been selected for oral presentations at the American Society of Hematology (ASH) Annual Meetings for four consecutive years, and was nominated for “Best of ASH” in 2019. To date, olverembatinib has been granted one Fast Track designation and three Orphan Drug designations from the US FDA for the treatment of CML, acute lymphoblastic leukemia (ALL), and acute myeloid leukemia (AML); and one Orphan Designation from the European Medicines Agency (EMA) of the European Union for the treatment of CML.
In July 2021, Ascentage Pharma (6855.HK) and Innovent Biologics (1801.HK) reached the agreement regarding the joint development and commercialization of olverembatinib in China.
*Olverembatinib has not been approved for any indication in the U.S.
About Innovent
Inspired by the spirit of "Start with Integrity, Succeed through Action,” Innovent’s mission is to develop, manufacture and commercialize high-quality biopharmaceutical products that are affordable to ordinary people. Established in 2011, Innovent is committed to developing, manufacturing and commercializing high-quality innovative medicines for the treatment of cancer, autoimmune, metabolic, ophthalmology and other major diseases. On October 31, 2018, Innovent was listed on the Main Board of the Stock Exchange of Hong Kong Limited with the stock code: 01801.HK.
Since its inception, Innovent has developed a fully integrated multi-functional platform which includes R&D, CMC (Chemistry, Manufacturing, and Controls), clinical development and commercialization capabilities. Leveraging the platform, the company has built a robust pipeline of 32 valuable assets in the fields of cancer, metabolic, autoimmune disease and other major therapeutic areas, with 7 products approved for marketing in China – TYVYT® (sintilimab injection), BYVASDA® (bevacizumab biosimilar injection), SULINNO® (adalimumab biosimilar injection), HALPRYZA® (rituximab biosimilar injection) , Pemazyre® (pemigatinib oral inhibitor) , olverembatinib (BCR-ABL TKI) and Cyramza® (ramucirumab), 3 assets under NMPA NDA review, 3 assets in Phase 3 or pivotal clinical trials, and an additional 19 molecules in clinical studies.
Innovent has built an international team with advanced talent in high-end biological drug development and commercialization, including many global experts. The company has also entered into strategic collaborations with Eli Lilly and Company, Adimab, Incyte, MD Anderson Cancer Center, Hanmi and other international partners. Innovent strives to work with many collaborators to help advance China’s biopharmaceutical industry, improve drug availability and enhance the quality of the patients’ lives. For more information, please visit: www.innoventbio.com. and www.linkedin.com/company/innovent-biologics/.
Note:
TYVYT® (sintilimab injection) is not an approved product in the United States.
BYVASDA® (bevacizumab biosimilar injection), SULINNO®, and HALPRYZA® (rituximab biosimilar injection) are not approved products in the United States.
TYVYT® (sintilimab injection, Innovent)
BYVASDA® (bevacizumab biosimilar injection, Innovent)
HALPRYZA® (rituximab biosimilar injection, Innovent)
SULINNO® (adalimumab biosimilar injection, Innovent)
Pemazyre® (pemigatinib oral inhibitor, Incyte Corporation). Pemazyre® was discovered by Incyte Corporation and licensed to Innovent for development and commercialization in Mainland China, Hong Kong, Macau and Taiwan.
CYRAMZA® (ramucirumab, Eli Lilly). Cyramza® was discovered by Eli Lilly and licensed to Innovent for commercialization in Mainland China.
About Ascentage Pharma
Ascentage Pharma (6855.HK) is a globally focused biopharmaceutical company engaged in developing novel therapies for cancers, chronic hepatitis B, and age-related diseases. On October 28, 2019, Ascentage Pharma was listed on the Main Board of the Stock Exchange of Hong Kong Limited with the stock code 6855.HK.
Ascentage Pharma focuses on developing therapeutics that inhibit protein-protein interactions to restore apoptosis, or programmed cell death. The company has built a pipeline of eight clinical drug candidates, including novel, highly potent Bcl-2, and dual Bcl-2/Bcl-xL inhibitors, as well as candidates aimed at IAP and MDM2-p53 pathways, and next-generation tyrosine kinase inhibitors (TKIs). Ascentage Pharma is also the only company in the world with active clinical programs targeting all three known classes of key apoptosis regulators. The company is conducting more than 50 Phase I/II clinical trials in the US, Australia, Europe, and China. Olverembatinib, the company's core drug candidate developed for the treatment of drug-resistant chronic myeloid leukemia (CML), was granted Priority Review status and a Breakthrough Therapy Designation (BTD) by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) and is already approved for the indication. In addition, olverembatinib was also granted an Orphan Drug Designation (ODD) and a Fast Track Designation (FTD) by the US FDA, and an Orphan Designation by the EU. To date, Ascentage Pharma has obtained a total of 15 ODDs, 2 FTDs, and 2 Rare Pediatric Disease (RPD) designations from the FDA and 1 ODD from the EU for four of the company's investigational drug candidates. Ascentage Pharma has been designated for multiple Major National R&D Projects, including 5 National Major New Drug Discovery and Manufacturing projects, 1 New Drug Incubator status, 4 Innovative Drug Programs, and 1 Major Project for the Prevention and Treatment of Infectious Diseases.
Leveraging its robust R&D capabilities, Ascentage Pharma has built a portfolio of global intellectual property rights and entered into global partnerships with numerous renowned biotechnology and pharmaceutical companies and research institutes such as UNITY Biotechnology, MD Anderson Cancer Center, Mayo Clinic, Dana-Farber Cancer Institute, Merck, AstraZeneca, and Pfizer. The company has built a talented team with global experience in discovering, developing, launching, and commercializing innovative drugs and is setting up world-class commercial manufacturing and Sales & Marketing teams. One pivotal aim of Ascentage Pharma is to continuously strengthen its R&D capabilities and accelerate its clinical development programs, in order to fulfil its mission of addressing unmet clinical needs in China and around the world for the benefit of more patients.
Innovent, Ascentage's cancer drug gets priority review in China for expanded use
Jul. 20, 2022 5:04 AM ET
Innovent Biologics, Inc. (IVBIY)
By: Ravikash, SA News Editor
- China's National Medical Products Administration (NMPA) granted priority review designation to Innovent Biologics (OTCPK:IVBIY) and Ascentage Pharma's application seeking full approval of olverembatinib to treat patients with chronic-phase chronic myeloid leukemia (CML-CP) who are resistant and/or intolerant of first- and second-generation tyrosine kinase inhibitors (TKIs).
- https://seekingalpha.com/news/3858381-innovent-ascentage-cancer-drug-get-priority-review-in-china-for-expanded-use
- https://seekingalpha.com/symbol/IVBIY
- https://www.innoventbio.com/
- https://www.ascentage.com/science/pipeline/
INNOVENT BIOLOGICS, INC. (1801)
https://www.hkex.com.hk/Market-Data/Securities-Prices/Equities/Equities-Quote?sym=1801&sc_lang=en
AVR-RD-05
Eltanexor (KPT-8602)
Cabozantinib in Combination with Nivolumab and Ipilimumab
AVROBIO Receives Orphan Drug Designation from the U.S. Food and Drug Administration for AVR-RD-05, a Gene Therapy for Mucopolysaccharidosis Type II (MPSII) or Hunter Syndrome
AVR-RD-05 is the fourth AVROBIO gene therapy to receive orphan drug designation
FDA previously granted rare pediatric disease designation for AVR-RD-05
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jul. 13, 2022-- AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene therapy company with a shared purpose to free people from a lifetime of genetic disease, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for AVR-RD-05, its gene therapy for the treatment of mucopolysaccharidosis type II (MPSII), or Hunter syndrome, a rare and seriously debilitating lysosomal disorder that primarily affects young boys.
Orphan drug designation is granted by FDA 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 incentives, which may include tax credits towards the cost of clinical trials and prescription drug user fee waivers.
Hunter syndrome is caused by a deficiency in the lysosomal enzyme iduronate-2-sulfatase (IDS), which is essential for breaking down large sugar molecules. The gene therapy uses a patient’s own hematopoietic stem cells (HSCs) that are transduced ex vivo with a lentiviral vector encoding the human IDS enzyme. The company’s planned collaborator-sponsored Phase 1/2 clinical trial for Hunter syndrome is expected to commence in 2023 under the company’s collaboration with the University of Manchester, UK. The program was developed by Brian Bigger, Ph.D., professor of cell and gene therapy at the University of Manchester, who has previously published preclinical data demonstrating that HSC gene therapy deploying an optimized, proprietary tag has the potential to correct peripheral disease and normalize brain pathology.
About AVROBIO
Our vision is to bring personalized gene therapy to the world. We aim to prevent, halt or reverse disease throughout the body with a single dose of gene therapy designed to drive durable expression of therapeutic protein, even in hard-to-reach tissues and organs including brain, muscle and bone. AVROBIO’s pipeline is powered by our industry-leading plato® gene therapy platform, our foundation designed to deliver gene therapy worldwide. It includes clinical programs in cystinosis and Gaucher disease type 1, as well as preclinical programs in Gaucher disease type 3, Hunter syndrome and Pompe disease. We are headquartered in Cambridge, Mass. For additional information, visit avrobio.com, and follow us on Twitter and LinkedIn.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220713005196/en/
Source: AVROBIO, Inc.
AVROBIO granted FDA’s orphan drug designation for metabolic disorder therapy
Jul. 13, 2022 7:25 AM ET
By: Dulan Lokuwithana, SA News Editor
- The shares of gene therapy company AVROBIO (NASDAQ:AVRO) jumped 9% in the pre-market Wednesday after the company announced that the FDA awarded the orphan drug designation for AVR-RD-05, a gene therapy candidate for mucopolysaccharidosis type II (MPSII), or Hunter syndrome.
- https://seekingalpha.com/news/3856326-avro-stock-gains-on-fdas-orphan-drug-status-for-metabolic-disorder-therapy
- https://seekingalpha.com/symbol/AVRO
- https://www.avrobio.com/our-pipeline
Cabozantinib in Combination with Nivolumab and Ipilimumab
Cabozantinib in Combination with Nivolumab and Ipilimumab
Cabozantinib in Combination with Nivolumab and Ipilimumab
Exelixis Announces Cabozantinib in Combination with Nivolumab and Ipilimumab Significantly Improved Progression-Free Survival in Phase 3 COSMIC-313 Pivotal Trial in Patients with Previously Untreated Advanced Kidney Cancer
– Exelixis intends to discuss the results with the U.S. FDA to determine next steps toward a potential regulatory submission –
– Trial to continue until the next analysis of the secondary endpoint of overall survival –
ALAMEDA, Calif.--(BUSINESS WIRE)--Jul. 11, 2022-- Exelixis, Inc. (Nasdaq: EXEL) today announced that COSMIC-313 met its primary endpoint, demonstrating significant improvement in progression-free survival (PFS) at the primary analysis. COSMIC-313 is an ongoing phase 3 pivotal trial evaluating the combination of cabozantinib (CABOMETYX®), nivolumab and ipilimumab versus the combination of nivolumab and ipilimumab in patients with previously untreated advanced intermediate- or poor-risk renal cell carcinoma (RCC). At a prespecified interim analysis for the secondary endpoint of overall survival (OS), the combination of cabozantinib, nivolumab and ipilimumab versus the combination of nivolumab and ipilimumab did not demonstrate a significant benefit. Therefore, the trial will continue to the next analysis of OS.
“As the treatment landscape continues to evolve, resulting in more options for advanced kidney cancer, there is still a need for additional effective first-line treatment options for patients with intermediate- or poor-risk disease,” said Toni Choueiri, M.D., Director of the Lank Center for Genitourinary Oncology at Dana-Farber Cancer Institute and the Jerome and Nancy Kohlberg Professor of Medicine at Harvard Medical School. “These initial findings from COSMIC-313 suggest that the triplet combination of cabozantinib, nivolumab and ipilimumab may have potential to serve as an additional option for this patient population.”
In the primary analysis of PFS per Response Evaluation Criteria in Solid Tumors version 1.1, as assessed by Blinded Independent Radiology Committee, cabozantinib in combination with nivolumab and ipilimumab significantly reduced the risk of disease progression or death compared with the combination of nivolumab and ipilimumab (hazard ratio: 0.73; 95% confidence interval: 0.57-0.94; P=0.01). The safety profile observed in the trial was reflective of the known safety profiles for each single agent, as well as the combination regimens used in this study. No new safety signals were identified.
Exelixis intends to discuss the results with the U.S. Food & Drug Administration (FDA) to determine next steps toward a potential regulatory submission for the combination regimen for patients with previously untreated advanced intermediate- or poor-risk RCC. Detailed findings will be submitted for presentation at a future medical meeting.
“COSMIC-313 is the first trial to show that a tyrosine kinase inhibitor added to dual checkpoint inhibition can improve progression-free survival in patients with advanced kidney cancer,” said Vicki L. Goodman, M.D., Executive Vice President, Product Development & Medical Affairs, and Chief Medical Officer, Exelixis. “With these findings in hand, we look forward discussing the results with the FDA and presenting the data at a future medical meeting.”
About COSMIC-313
COSMIC-313 is a multicenter, randomized, double-blinded, controlled phase 3 pivotal trial that enrolled 855 patients at 177 sites globally. Patients were randomized 1:1 into the experimental or control arms of the study. Patients in the experimental arm received cabozantinib (40 mg, once daily) in combination with nivolumab (3 mg/kg infusion, once every 3 weeks for 4 doses total) and ipilimumab (1 mg/kg infusion, once every 3 weeks for 4 doses total) followed by cabozantinib (40 mg, once daily) and nivolumab (480 mg/kg flat dose infusion, once every 4 weeks for up to 2 years). Patients in the control arm received cabozantinib-matched placebo in combination with nivolumab (3 mg/kg infusion, once every 3 weeks for 4 doses total) and ipilimumab (1 mg/kg infusion, once every 3 weeks for 4 doses total) followed by cabozantinib-matched placebo and nivolumab (480 mg/kg flat dose infusion, once every 4 weeks for up to 2 years). The primary endpoint is PFS; the secondary endpoint is OS. Exelixis is funding the trial, and Bristol Myers Squibb is providing nivolumab and ipilimumab for use in this trial. More information about this trial is available at ClinicalTrials.gov.
About CABOMETYX® (cabozantinib)
In the U.S., CABOMETYX tablets are approved for the treatment of patients with advanced RCC; for the treatment of patients with hepatocellular carcinoma who have been previously treated with sorafenib; for patients with advanced RCC as a first-line treatment in combination with nivolumab; and for adult and pediatric patients 12 years of age and older with locally advanced or metastatic differentiated thyroid cancer that has progressed following prior VEGFR-targeted therapy and who are radioactive iodine-refractory or ineligible. CABOMETYX tablets have also received regulatory approvals in the European Union and additional countries and regions worldwide. In 2016, Exelixis granted Ipsen Pharma SAS exclusive rights for the commercialization and further clinical development of cabozantinib outside of the U.S. and Japan. In 2017, Exelixis granted exclusive rights to Takeda Pharmaceutical Company Limited for the commercialization and further clinical development of cabozantinib for all future indications in Japan. Exelixis holds the exclusive rights to develop and commercialize cabozantinib in the U.S.
CABOMETYX in combination with nivolumab and ipilimumab is not indicated as a treatment for previously untreated advanced RCC.
About Exelixis
Founded in 1994, Exelixis, Inc. (Nasdaq: EXEL) is a commercially successful, oncology-focused biotechnology company that strives to accelerate the discovery, development and commercialization of new medicines for difficult-to-treat cancers. Following early work in model system genetics, we established a broad drug discovery and development platform that has served as the foundation for our continued efforts to bring new cancer therapies to patients in need. Our discovery efforts have resulted in four commercially available products, CABOMETYX® (cabozantinib), COMETRIQ® (cabozantinib), COTELLIC® (cobimetinib) and MINNEBRO® (esaxerenone), and we have entered into partnerships with leading pharmaceutical companies to bring these important medicines to patients worldwide. Supported by revenues from our marketed products and collaborations, we are committed to prudently reinvesting in our business to maximize the potential of our pipeline. We are supplementing our existing therapeutic assets with targeted business development activities and internal drug discovery — all to deliver the next generation of Exelixis medicines and help patients recover stronger and live longer. Exelixis is a member of the Standard & Poor’s (S&P) MidCap 400 index, which measures the performance of profitable mid-sized companies. For more information about Exelixis, please visit www.exelixis.com, follow @ExelixisInc on Twitter or like Exelixis, Inc. on Facebook.
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Source: Exelixis, Inc.
Exelixis says phase 3 trial evaluating its kidney cancer combo treatment met main goal
Jul. 11, 2022 9:54 AM ETExelixis, Inc. (EXEL)By: Anuron Mitra, SA News Editor2 Comments
- Exelixis (NASDAQ:EXEL) on Monday said its phase 3 trial evaluating its cabozantinib medicine in combination with two other cancer drugs for the treatment of kidney cancer met its main goal.
- https://seekingalpha.com/news/3855690-exelixis-says-phase-3-trial-evaluating-its-kidney-cancer-combo-treatment-met-main-goal
- https://seekingalpha.com/symbol/EXEL
- https://www.cabometyx.com/