Bayer AskBio receive Pioneering Regenerative Medical Product Designation for gene therapy programs in Japan
Berlin: AskBio, a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, has announced that Japan's Ministry of Health, Labour and Welfare (MHLW) has granted the Pioneering Regenerative Medical Product designation (SAKIGAKE) for two of AskBio’s investigational gene therapy programs: AB-1005, for the treatment of Parkinson’s disease (PD), and AB-1002, for the treatment of non-ischemic heart failure with reduced left ventricular ejection fraction and New York Heart Association (NYHA) Class III heart failure despite appropriate medical therapy.
The designation is awarded to products demonstrating innovativeness (a new mode of action), prominent efficacy or safety data, and the potential to address severe diseases, especially when submitted first or simultaneously with other countries. This recognition offers significant advantages, including priority consultations and accelerated review timelines, thereby facilitating earlier participant access to transformative treatments.
“Having AB-1005 and AB-1002 receive the Pioneering Regenerative Medical Product designation in Japan highlights our dedication to advancing innovative gene therapies for participants facing serious diseases,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio. “This recognition not only accelerates regulatory review but also reaffirms our commitment to delivering advanced treatments to those living with serious chronic diseases that lack therapies targeting root causes.”
AB-1005, currently being evaluated in the Phase II REGENERATE-PD trial, is an investigational gene therapy with adeno-associated viral (AAV) vector-mediated delivery of the glial cell line-derived neurotrophic factor (GDNF) gene for participants with moderate-stage PD. The therapy aims to restore neuronal function and potentially slow disease progression for people with limited treatment options. AB-1005 previously received US Food and Drug Administration (FDA) Regenerative Medicine Advanced Therapy (RMAT), FDA Fast Track, and UK Medicines and Healthcare products Regulatory Agency (MHRA) Innovation Passport designations.
AB-1002 is an investigational AAV gene therapy being studied for the treatment of adults with NYHA Class III heart failure with non-ischemic etiology. It previously received FDA Fast Track designation, and is designed as a one-time gene therapy targeting protein phosphatase 1 inhibition, with the intention of improving cardiac function and addressing the substantial global burden of congestive heart failure.
“Bayer and AskBio’s collaboration continues to drive progress in gene therapy with a robust pipeline targeting central nervous system, cardiovascular, and other disease indications,” said Christian Rommel, PhD and Global Head of Research and Development for Bayer's Pharmaceuticals Division. “Receiving the designation in Japan, which is a first for Bayer, marks an important milestone in expanding global access to pioneering therapies and reinforces our shared commitment to delivering breakthrough science to improve outcomes for patients worldwide.”
FDA RMAT is a designation granted by the FDA to regenerative therapies, including gene therapies, being developed to treat, modify, reverse, or cure serious or life-threatening diseases or conditions. Investigational products receiving this designation must have produced preliminary clinical evidence indicating that they may have the potential to address unmet medical needs for such diseases or conditions. RMAT provides recipients with enhanced access to the FDA, which could include intensive guidance on efficient drug development, rolling Biologics License Application (BLA) review, and other actions to expedite review.
The FDA Fast Track program is designed to facilitate the development and expedite the review of new therapeutics that are intended to treat serious conditions and address unmet medical needs. The purpose of the program is to get important new therapeutics to participants earlier. Therapeutics that receive this designation benefit from eligibility for more frequent meetings with the FDA to discuss the clinical development plan and, if relevant criteria are met, eligibility for Accelerated Approval and Priority Review.
The UK MHRA Innovation Passport is the entry point to the Innovative Licensing and Access Pathway (ILAP), which aims to accelerate time to market, facilitating participants access to innovative medicines. This designation provides Innovation Passport holders with the opportunity to work with the UK MHRA and partners to create product-specific Target Development Profiles (TDP) for new therapies. The TDP will define key regulatory and development features, identify potential pitfalls, offer access to specialist toolkits, and create a roadmap for delivering early participants access.
AB-1005 and AB-1002 are investigational gene therapies that have not been approved by any regulatory health authority, and their efficacy and safety have not been established or fully evaluated.
AskBio is also exploring AB-1005 beyond PD and is currently conducting the MSA-101 trial in the U.S. for participants diagnosed with multiple system atrophy of the parkinsonian subtype (MSA-P) in a Phase I trial to assess the preliminary safety, tolerability, and efficacy of GDNF gene therapy for this rapidly progressing condition for which no treatment options are currently available.
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