AskBio's Gene Therapy Gets FDA Designation

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 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. 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]). 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 .

In the AB-1005 Phase Ib trial, a multi-center, multi-site, open-label, uncontrolled trial, 11 patients were administered AB 1005 to the putamen via one-time bilateral convection-enhanced delivery. Patients were enrolled into two cohorts, mild (6 patients) and moderate (5 patients), based upon the duration and stage of their PD. The objective of this investigation was to evaluate the safety and potential clinical effect of AB 1005 delivered to the putamen in patients with early/mild or moderate PD. The outcomes assessed at 36 months were incidence of Treatment-Emergent Adverse Events (TEAEs) as reported by the patients or assessed clinically by physical and neurological examinations, motor symptoms as reported via the Movement Disorder Society’s Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) and PD Motor Diary self-assessments, non-motor symptoms of PD, and brain dopaminergic network integrity as measured by DaTSCAN. These assessments will continue for up to five years. 

“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 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 REGENERATE-PD (NCT06285643), a Phase II, randomized, double-blind, sham-controlled trial of the efficacy and safety of intraputaminal AB 1005 in the treatment of adults (45-75 years) with moderate-stage Parkinson's disease- have been randomized in the United States and the trial is currently recruiting. 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.. 

See citation- Nicolas M, et al. Preliminary Efficacy of GDNF Gene Therapy (AAV2-GDNF; AB-1005) in Parkinson’s Disease: 36-Month Follow-Up From a Phase 1b Study. Presented at the International Congress of Parkinson’s Disease and Movement Disorders 2024.