First participants randomized in Bayer's AskBio Phase II gene therapy trial

Enrollment ongoing in the United States for REGENERATE-PD clinical trial of AB-1005 (also known as AAV2-GDNF) glial cell line-derived neurotrophic factor (GDNF) investigational gene therapy for the treatment of moderate-stage Parkinson’s disease / Phase Ib 36-month data demonstrated a favorable safety profile and continued positive trends in assessed clinical measures of AB-1005 with no associated serious adverse events

AskBio Inc. (AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, announced that the first participants have been randomized in its Phase II clinical trial in patients with Parkinson’s disease (PD).

“The randomization of the first participants in REGENERATE-PD is positive news for people living with Parkinson’s disease and the physicians treating them,” said Dr Rajesh Pahwa, MD, Director, Parkinson's Disease and Movement Disorder Center, University of Kansas Medical Center, USA, and REGENERATE-PD Principal Investigator. “There is a significant need for neurorestorative therapies in Parkinson’s and seeing the advancement of an important investigational gene therapy in a Phase II clinical trial gives hope to patients and the medical community.”

In 2024, AskBio initiated recruitment in the United States arm of REGENERATE-PD. The objective of this randomized, double-blind, Phase II clinical trial is to evaluate the safety and efficacy of AB-1005 delivered to the putamen in adult participants aged 45–75 years with moderate-stage PD. REGENERATE-PD is estimated to enroll approximately 87 participants across clinical centers that are being opened in the United States, Germany, Poland, and the United Kingdom.

AskBio also presented 36-month Phase Ib data at the International Congress of Parkinson’s Disease and Movement Disorders, which was held in Philadelphia, Pennsylvania, from September 27 to October 1, 2024. The data demonstrated that administration of AB-1005 was well tolerated with no attributed serious adverse events. The Moderate PD cohort showed trends for improvement or stability on several motor scales at 36 months, including Movement Disorder Society-Unified Parkinson's Disease Rating Scale (MDS-UPDRS) and motor diaries, and 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.

“AskBio continues to mark significant milestones in the clinical development of the investigational gene therapy AB-1005 as we strive to bring a safe and effective gene therapy to patients with moderate stage Parkinson’s disease,” said Canwen Jiang, MD, PhD, Chief Development Officer and Chief Medical Officer, AskBio. “With REGENERATE-PD participants now being randomized, we are excited by our progress with AB-1005 and look forward to sharing further updates at an appropriate scientific forum as the program advances through next year and beyond.”

“The advancement of AskBio’s Parkinson’s program is a significant milestone that highlights our commitment to exploring the potential of gene therapy in an area of significant unmet medical need,” said Christian Rommel, Member of the Executive Committee of Bayer’s Pharmaceuticals Division, and Global Head of Research and Development. “We believe that therapeutic modalities like gene therapy can change the treatment landscape for people living with the debilitating effects of Parkinson’s disease and look forward to seeing what we will learn in this next phase of research and development for AB-1005.”

AskBio is also exploring AB-1005 beyond Parkinson’s disease and is currently enrolling participants in the United States with the parkinsonian subtype of multiple system atrophy (MSA-P) in a Phase I clinical trial to assess the preliminary safety, tolerability, and efficacy for this rapidly progressing condition.

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.

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