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Monday, May 2, 2022

Veru Gets Pre-Emergency Use Authorization Meeting Date on Positive Phase 3 COVID-19 Study

 Veru Inc. (NASDAQ: VERU), a biopharmaceutical company, today announced that the FDA has granted the Company a pre-Emergency Use Authorization (EUA) meeting on May 10, 2022, for the positive Phase 3 COVID-19 study for sabizabulin.

“We want to keep our investors informed of the important steps as we proceed through the EUA process. We appreciate FDA’s actions to expedite this process as COVID-19 cases are on the rise again,” said Mitchell S. Steiner MD, Chairman, President and Chief Executive Officer of Veru Inc.

https://finance.yahoo.com/news/fda-granted-veru-pre-emergency-123000144.html

Denali Hits Milestone for Phase 2 Clinical Trial Initiation in ALS by Sanofi

 Denali Therapeutics Inc. (NASDAQ: DNLI), a biopharmaceutical company developing a broad portfolio of product candidates engineered to cross the blood-brain barrier (BBB) for neurodegenerative diseases, today announced that its partner Sanofi has commenced dosing in a Phase 2 study, named HIMALAYA, of SAR443820 (DNL788) in individuals with amyotrophic lateral sclerosis (ALS). SAR443820 is a central nervous system (CNS)-penetrant small molecule inhibitor of RIPK1.

“ALS is a devastating neurodegenerative disease for which effective treatment options are a critical unmet medical need,” said Nazem Atassi, M.D., Sanofi’s Global Head of Early Neuro Development. “We are very encouraged by the results from the Phase 1 trial of SAR443820 in healthy volunteers in which robust target engagement was demonstrated at doses that were generally well tolerated. Based on these results, we are excited to start the HIMALAYA Phase 2 ALS study and look forward to working with Denali and the ALS community to advance the development of this novel investigational therapy.”

“The initiation of this Phase 2 study marks a significant milestone in the SAR443820 development program and our RIPK1 inhibitor collaboration with Sanofi,” said Denali’s Chief Medical Officer, Carole Ho, M.D. “We are excited about the progression of Denali’s portfolio in ALS with this milestone. Denali is committed to collaborating with the ALS community as we work toward a unified goal of developing potentially life-saving therapeutics for people living with ALS.”

https://finance.yahoo.com/news/denali-therapeutics-announces-achievement-ripk1-123000293.html

HilleVax Starts Phase 2b Trial of HIL-214 Norovirus Vaccine

 HilleVax, Inc. (Nasdaq: HLVX), a biopharmaceutical company focused on the development and commercialization of novel vaccine candidates, reported today dosing of the first subjects in its previously announced Phase 2b clinical trial of HIL-214, the company’s virus-like particle (VLP) based vaccine candidate, for the prevention of moderate-to-severe acute gastroenteritis (AGE) caused by norovirus infection in infants.

“We’re pleased to have dosed the first subjects in this Phase 2b study, the first ever field efficacy clinical trial of a norovirus vaccine candidate in infants,” said Rob Hershberg, MD, PhD, Chairman and Chief Executive Officer of HilleVax. “We believe HIL-214 has the potential to address a vast unmet need which includes approximately 700 million cases, 200,000 deaths, and $60 billion of economic burden from norovirus-related AGE worldwide each year.”

The clinical trial is a Phase 2b, randomized, double-blind, placebo-controlled study to evaluate the efficacy, safety, and immunogenicity of HIL-214 in infants of approximately 5 months of age at time of initial vaccination at sites in the United States and Latin America. The study is planned to enroll 3,000 subjects who will be randomized 1:1 to receive a two-dose regimen of either HIL-214 or placebo. The primary objective of the trial is to evaluate the protective efficacy of HIL-214 against the first confirmed moderate or severe AGE event due to GI.1 or GII.4 norovirus strains (excluding certain co-infections) that occurs prior to each subject reaching 12 months of age. A key secondary endpoint is to evaluate the protective efficacy of HIL-214 against any GI or GII norovirus strain. A pre-specified safety and immunogenicity analysis is planned for the first 200 subjects.

https://www.biospace.com/article/releases/hillevax-announces-initiation-of-phase-2b-clinical-trial-of-hil-214-vaccine-candidate-for-the-prevention-of-norovirus-related-acute-gastroenteritis-in-infants/

Alzheimer's Disease Treatment Revolution Supported by Advanced Testing Tools: Quest

 Quest Diagnostics (NYSE: DGX), the world's leading provider of diagnostic information services, today released a new research report, The Coming Alzheimer's Disease Healthcare Revolution: U.S. Physician and Adult Perspectives on the Future of Diagnostics and Treatment, that provides insights into the expectations and hopes of primary care physicians and American adults for the current and future landscape of dementia and AD testing, treatment and care. According to the report, a majority of physicians (66%) believe we are on the precipice of groundbreaking new treatment options for AD.

The Coming Alzheimer’s Disease Healthcare Revolution: U.S. Physician and Adult Perspectives on the Future of Diagnostics and Treatment

While 50% of physicians do not think there will ever be a cure for AD, the surveys indicate that physicians and American adults are optimistic that a new generation of therapies and diagnostics for the disease will improve patient care. In fact, more than 3 in 4 physicians (77%) believe new therapies will transform AD into a chronic, manageable disease–and 84% say testing for early risk of the disease will lead to earlier and improved disease management. Among U.S. adults, 9 in 10 (90%) say they are hopeful that new therapies will cure AD, and 86% believe blood tests for the early detection of AD risk will increasingly become a regular part of preventative care.

Based on insights from online surveys of 501 primary care providers (PCPs) and 2,052 Americans aged 18 years and older, the report also highlights the important role diagnostics may play in the next era of AD healthcare. Quest Diagnostics commissioned The Harris Poll to conduct the surveys in March 2022.

The report suggests cost concerns could impede the adoption of blood tests for AD: more than 8 in 10 physicians (85%) say the value of a blood test for the early detection of AD risk will depend on how widely it is reimbursed. However, 94% of physicians say blood tests would be more cost effective for the healthcare system compared to more invasive methods of detection (e.g., lumbar puncture, imaging studies).

"We are on the cusp of a new generation of therapies for Alzheimer's disease, but the important role of diagnostics has been missing from the conversation. Patients today are typically screened for Alzheimer's disease only after signs of cognitive impairment emerge and often by expensive methods, such as brain imaging and cerebrospinal fluid taps, which only specialists can perform. As new, efficacious therapies come to the forefront, the need for scalable, less invasive and more cost-effective diagnostics, including in primary care settings, will grow," said Michael K. Racke, M.D., Neurology Medical Director, Quest Diagnostics. "Our goal for this report is to help prepare the medical community and engaged patients and caregivers for the transformational healthcare shifts that must occur to unleash the full potential of future treatment and diagnostic innovations to improve outcomes for patients with Alzheimer's disease."

The new Quest report follows the Centers for Medicare and Medicaid Services' decision in April to limit coverage of the AD drug aducanumab to patients who receive it as participants in a clinical trial. The Food and Drug Administration's approval of the drug in June 2021 marked the first authorization of a treatment designed to target AD pathophysiology. In addition, the identification of biomarkers for AD has led to new avenues of pharmaceutical drug research and development. More than 100 disease-modifying therapies are now in clinical trials–nearly 20 in phase 3.i

https://www.biospace.com/article/releases/physicians-foresee-an-alzheimer-s-disease-treatment-revolution-supported-by-advanced-testing-tools-new-quest-diagnostics-report-finds/

VBI Vaccines: EC OKs Marketing Authorisation for 3-Antigen Adult Hepatitis B Vaccine

 

  • PreHevbri™ is the only approved 3-antigen hepatitis B vaccine for adults in the EU and EEA
  • Approval follows the positive opinion granted by European Committee for Medicinal Products for Human Use (CHMP) in February 2022
  • Regulatory review in the United Kingdom is ongoing as part of the European Commission Decision Reliance Procedure

IDEAYA Gets FDA Orphan Designation for Darovasertib in Uveal Melanoma

 IDEAYA Biosciences Inc. on Monday said the U.S. Food and Drug Administration granted orphan-drug designation to darovasertib for the treatment of uveal melanoma, the most common primary intraocular cancer.

The South San Francisco, Calif., precision-medicine oncology company is currently evaluating darovasertib in combination with Pfizer Inc.'s cancer drug Xalkori in patients with metastatic uveal melanoma and in patients with GNAQ or GNA11 mutant solid tumors in a Phase 1/2 clinical trial as part of a collaboration with the New York drugmaker.

IDEAYA said it is targeting a mid-year clinical data update for the combination, including tolerability and clinical efficacy, adding that it also plans to seek FDA regulatory guidance for a potential registration-enabling trial design to evaluate the combination in metastatic uveal melanoma.

The FDA's orphan-drug program gives special status to drugs and biologics for diseases and disorders that affect fewer than 200,000 people in the U.S. and provides for an extended marketing exclusivity period against competition.

https://www.marketscreener.com/quote/stock/IDEAYA-BIOSCIENCES-INC-58457878/news/IDEAYA-Gets-FDA-Orphan-Designation-for-Darovasertib-in-Uveal-Melanoma-40243665/

HUTCHMED : FDA Rejects Surufatinib Application in Neuroendocrine Tumors

 HUTCHMED (China) Ltd. on Monday said the U.S. Food and Drug Administration has turned away its application seeking approval of surufatinib for the treatment of pancreatic and extra-pancreatic neuroendocrine tumors and wants the biopharmaceutical company to conduct another study.

The Hong Kong company said the FDA determined that its current data package, based on two positive Phase III trials in China and one bridging study in the U.S., doesn't support an approval in the U.S., and that a multi-regional clinical trial that includes subjects more representative of the U.S. patient population and aligned to current U.S. medical practice is needed.

HUTCHMED said the FDA issued a so-called complete response letter, which indicates the agency won't approve the application in its current form.

The company noted that the FDA's action isn't related to any safety issues with surufatinib, adding that it is working with the agency to evaluate its next steps.

https://www.marketscreener.com/quote/stock/HUTCHMED-CHINA-LIMITED-59660104/news/HUTCHMED-FDA-Rejects-Surufatinib-Application-in-Neuroendocrine-Tumors-40243754/