Douglas Pharmaceuticals | January 27, 2023
A clinical advisory board has been established to support Douglas Pharmaceuticals' lead program R-107, an oral extended-release dose of racemic ketamine being developed as an adjunctive or stand-alone therapy for treatment-resistant depression.
Douglas Pharmaceuticals is a New Zealand-based pharmaceutical company specializing in the development of specialty generic and repurposed medications (TRD).
Peter Surman, Chief Scientific Officer expressed, "We are very excited to bring together a group of world-class experts in the field of psychiatry, to help guide our late-stage development of R-107." "We believe their deep knowledge in the field, insights and contributions will be instrumental in charting an efficient course through pivotal clinical studies in TRD," he added.
(Source – Cision PR Newswire)
Some of the members of the clinical advisory board are as follows:
• Prof. Paul Glue [MB, ChB (Otago), MD (Bristol), FRCPsych]
• Prof. Alan F. Schatzberg, [MD] • Prof. Sanjay J. Mathew [MD]
• Prof. Allan Young [MB, ChB, MPhil, Ph.D., FRCP (Edin.), FRCPC, FRCPsych]
• Prof. Wiesław J. Cubała, [MD, PhD]
• Prof. Colleen Loo [MBBS (Hons), FRANZCP, MD]
About Douglas Pharmaceuticals
New Zealand's largest private pharmaceutical and healthcare firm, Douglas Pharmaceuticals, employs over 700 people and exports to over 40 countries. The company provides creative, competitive, and high-quality healthcare solutions to 'improve lives'. It designs, manufactures, and distributes unique and generic products, preferring those with significant intellectual property and technological complexity in high-unmet need sectors. The company's presence in international markets continues to grow, exposing New Zealand to the world. It is dedicated to improving people's health all around the globe. Some of the products that the company works with cutting-edge partners to develop, make, market, and distribute are pharmaceuticals, nutraceuticals, automated compliance systems, and consumer healthcare items.
RESEARCH, PHARMA TECH
Ionis Pharmaceuticals, Inc. | March 10, 2023
Ionis Pharmaceuticals, a leading RNA-targeted therapy firm, recently announced that the USFDA has accepted for review a new drug application (NDA) for eplontersen. Eplontersen is an investigational antisense medicine designed to reduce the production of transthyretin (TTR) protein and treat hereditary and non-hereditary forms of ATTR amyloidosis (ATTR).
The NDA has been given a Prescription Drug User Fee Act (PDUFA) action date of December 22, 2023. The FDA has declared that no review concerns were detected and made no new data requests. The FDA also stated that it does not intend to conduct a meeting of the advisory committee to discuss the application.
ATTR amyloidosis is a systemic disease caused by the accumulation of TTR protein in organs, leading to progressive organ damage and eventually death. Patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) experience ongoing debilitating nerve damage throughout their bodies, resulting in the progressive loss of motor function.
The NDA for eplontersen is based on results from the global Phase 3 NEURO-TTRansform study showcased at the International Symposium on Amyloidosis (ISA). In the study, eplontersen showcased a statistically significant and clinically meaningful change relative to the baseline for its co-primary and critical secondary endpoints compared to the external placebo group. In addition, Eplontersen also demonstrated a favorable safety and tolerability profile.
Ionis and AstraZeneca announced a strategic partnership to develop and commercialize eplontersen in December 2021. Eplontersen is being developed and commercialized jointly by both businesses in the United States and will be created and commercialized by AstraZeneca throughout the rest of the globe, except Latin America.
The FDA granted eplontersen Orphan Drug Designation in the United States in January 2022. Eplontersen is also being studied in the worldwide Phase 3 CARDIO-TTRansform trial for transthyretin amyloid cardiomyopathy (ATTR-CM), a systemic, progressive, and deadly disorder leading to progressive heart failure and death within 3 to 5 years after disease start.
About Ionis Pharmaceuticals, Inc.
Ionis Pharmaceuticals, Inc. is a leading biopharmaceutical company headquartered in Carlsbad, California. Its unique antisense technology platform allows them to develop RNA-targeted therapies that modify the production of disease-causing proteins, leading to the development of treatments for rare genetic disorders, cardiovascular diseases, and neurodegenerative diseases. The company's most significant achievement is developing and commercializing Spinraza, the first approved treatment for spinal muscular atrophy. Ionis Pharmaceuticals has also developed drugs for other rare diseases, including transthyretin amyloidosis, Huntington's disease, and myotonic dystrophy type 1.
RESEARCH, PHARMA TECH
Asklepios BioPharmaceutical, Inc. (AskBio) | March 01, 2023
Asklepios BioPharmaceutical, Inc. (AskBio), a subsidiary of Bayer AG, recently announced that the European Commission (EC) has granted orphan drug designation to AB-1003 (also known as LION-101) for the treatment of limb-girdle muscular dystrophy (LGMD). AB-1003 is an investigational recombinant adeno-associated virus (AAV) based gene therapy that is being developed as a one-time intravenous infusion for patients with LGMD type 2I/R9.
The disease affects 4.5 people per million worldwide, with more than 5,000 people affected in the EU and the US. The EC decision was received through AskBio's EU-based subsidiary BrainVectis, following a positive opinion from the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP)
AB-1003 is currently being investigated in a Phase 1/2 multicenter study in the US to evaluate its tolerability, safety, and efficacy in adult subjects with genotypically confirmed LGMD2I/R9.
The EC for medicinal products grants orphan drug designation developed to treat a life-threatening disease that affects no more than five people in 10,000 in the EU, provided there is no other adequate treatment option or the medicine can bring significant value to those affected by a specific condition. This designation will provide special incentives in the EU, including eligibility for protocol assistance and possible reductions or exemptions in certain regulatory fees. Additionally, if the medicine is approved for marketing, this designation will provide ten years of marketing exclusivity.
Limb-girdle muscular dystrophy (LGMD) is a group of diseases that cause progressive weakening and wasting of the muscles in the arms and legs. The severity, features of LGMD and age of onset vary among the many subtypes of the condition and are often inconsistent, even within the same family. Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9) is a form of LGMD caused by mutations in the FKRP gene. Signs and symptoms of LGMD2I/R9 often appear in late childhood and may include difficulty walking and running. The symptoms intensify with time, eventually leading to considerable disability, and afflicted persons typically require a wheelchair for movement roughly 23-26 years after beginning. Unfortunately, there is presently no treatment that slows the advancement of the disease, and management is based on the indications and symptoms that each individual exhibits.
About Asklepios BioPharmaceutical, Inc. (AskBio)
AskBio is a leading, fully integrated gene therapy company dedicated to developing innovative gene therapeutics and life-saving medicines. With operations worldwide, its pipeline includes gene therapy candidates for various diseases, including the central nervous system, neuromuscular, metabolic, and cardiovascular diseases. AskBio's global headquarters are located in RTP, NC, with European headquarters in Edinburgh. In addition, the company operates AAV research centers in Philadelphia, PA; Columbus, OH; and Paris, and modern clinical and commercial facilities in San Sebastian, Spain.