AskBio receives FDA Fast Track and MHRA Innovation Passport designations for AB-1005 investigational GDNF gene therapy for Parkinson’s disease

• AB-1005 (formerly known as AAV2-GDNF) is being studied for the treatment of patients with moderate Parkinson’s disease

• AskBio is currently enrolling patients in its Phase II REGENERATE-PD trial in the United States

• European Union and the United Kingdom trial sites planned to open later in 2024

Reading, UK, 11th July 2024 – Bayer AG and Asklepios BioPharmaceutical, Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that the United States (U.S.) Food and Drug Administration (FDA) has granted Fast Track Designation for AB-1005 which is being developed for moderate Parkinson’s disease. AB-1005 has also been awarded the innovative medicine designation, the Innovation Passport by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) for the treatment of Parkinson’s disease.

AB-1005 is an investigational adeno-associated virus 2 glial cell line-derived neurotrophic factor (AAV2-GDNF) neurorestorative gene therapy being studied for the treatment of moderate Parkinson’s disease. Earlier this year, AskBio presented the 18-month Phase Ib clinical trial results for AB-1005, which met its primary objective of evaluating the safety of a one-time bilateral delivery of AB-1005 directly to the putamen.

“These designations clearly underscore the importance of developing innovative therapies for those living with Parkinson’s disease, where a significant unmet need still exists,” said Krystof Bankiewicz, MD, PhD, Scientific Chair, Parkinson’s and MSA, AskBio. “They further highlight the willingness of key regulatory bodies to support accelerated development of AB-1005 with a focus on the potential benefit to patients.”

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 fill unmet medical needs.1 The purpose of the Program is to get important new therapeutics to patients earlier.1 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 patient access. This designation provides Innovation Passport holders with the opportunity to work with the 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 patient access.2,3

“Receiving FDA Fast Track Designation and the UK MHRA Innovation Passport represent important accomplishments for the clinical development of AB-1005 and highlight our goal of bringing a potential neurorestorative treatment to patients with moderate Parkinson’s disease,” said Canwen Jiang, MD, PhD, Chief Development Officer, and Chief Medical Officer, AskBio. “We look forward to advancing our Phase II REGENERATE-PD clinical trial, which is currently enrolling patients in the U.S. with sites in the European Union and the UK planned to open later this year.”

“We are excited about the opportunity to potentially accelerate the development of AB-1005, leveraging the frequent interaction with relevant regulatory bodies,” said Christian Rommel, PhD, Global Head of Research & Development at Bayer’s Pharmaceuticals Division. “The granted designations for AB-1005 highlight the demand to advance novel therapeutic modalities, like gene therapy, for people living with the debilitating effects of Parkinson’s disease.”

AB-1005 is an investigational gene therapy that has not been approved by any regulatory authority, and its efficacy and safety have not been established or fully evaluated.

AskBio is also exploring GDNF therapy beyond Parkinson’s disease and is currently enrolling patients in the U.S. with the parkinsonian subtype of multiple system atrophy (MSA-P) in a Phase I trial to assess the preliminary safety, tolerability, and efficacy of GDNF therapy for this rapidly progressing condition.4