February 19, 2025

Not intended for UK Media Berlin, Germany, and Research Triangle Park, N.C., USA, February 19, 2025 – AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that investigational gene therapy AB-1005 for the treatment of Parkinson’s disease (PD) has been granted Regenerative Medicine Advanced Therapy (RMAT) designation from the United States Food and Drug Administration (FDA). “The FDA’s decision to grant RMAT designation to AB-1005 is exciting news for people living with Parkinson’s disease and their loved ones,” said Gustavo Pesquin, CEO, AskBio. “This milestone could potentially expedite the development of our important investigational gene therapy program, and it highlights our promising data and the potential of AB-1005 for patients and the medical community. We look forward to working closely with the FDA to accelerate our program.” The FDA determined that AB-1005, an investigational gene therapy intended to slow disease progression and improve motor outcomes in patients with PD, met the criteria for RMAT designation. This decision follows a review of information and data provided by AskBio, including clinical evidence from the open label, uncontrolled study Phase Ib trial of AB-1005. AskBio’s 36-month Phase Ib data showed that the administration of AB-1005 was well tolerated with no product-related serious adverse events.1 Further, the moderate PD cohort showed trends for improvement or stability on several PD-relevant clinical scales at 36 months compared to baseline, including Movement Disorder Society-Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) and self-reported PD motor diaries, together with trends in reductions in Parkinson’s medications (levodopa-equivalent daily dose [LEDD]).1 Most participants in the mild PD cohort showed an overall stable clinical status with little change in MDS-UPDRS, the self-reported PD motor diary, or LEDD.1 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.2 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.2 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.2 “The RMAT designation for AB-1005 underscores the high unmet medical need and the potential of this investigational gene therapy to make a difference for patients with Parkinson’s disease,” said Christian Rommel, Executive Vice President, Global Head of Research and Development and Member of the Pharmaceuticals Leadership Team at Bayer. “This is the latest example of what can be achieved through the joint commitment of AskBio and Bayer to deliver breakthrough innovation for patients.” The first participants in the AB-1005 Phase II REGENERATE-PD clinical trial have been randomized in the United States, and the trial is currently recruiting.3 Additional study sites in the United States, Germany, Poland, and the United Kingdom are expected to be opened for enrollment in first half of 2025.3 AB-1005 has not been approved by any ...read the news