AskBio's AB-1005 Gene Therapy for Parkinson’s Disease Meets Primary Endpoint in Phase Ib Trial

The Phase Ib clinical trial of AB-1005 (AAV2-GDNF), an investigational gene therapy for Parkinson’s disease (PD), conducted by AskBio, has successfully met its primary endpoint. The trial demonstrated that AB-1005 was well tolerated with no serious adverse events attributed to the therapy in all 11 patients over an 18-month period. The study focused on the safety of a one-time bilateral delivery of AB-1005 directly to the putamen, achieving a target putamen coverage of 63% ± 2%, which exceeds the goal of greater than 50% coverage.

Patients enrolled in the trial were divided into two cohorts based on the severity of their PD symptoms: Mild stage PD (6 patients) and Moderate stage PD (5 patients). Neurosurgical delivery of AB-1005 was well tolerated by all participants. The trial also included 18-month neurological assessments and self-reported questionnaires to evaluate the severity of motor and non-motor symptoms associated with PD, alongside brain imaging to assess safety and potential changes in dopamine handling or abnormal metabolic patterns.

AskBio is planning to present the 18-month study data, including secondary endpoints, at a scientific meeting in Q2 2024. Additionally, planning is underway for a Phase II trial expected to begin screening patients in the first half of 2024. The trial design has been harmonized with feedback from U.S. and European health authorities.

AB-1005 is based on adeno-associated viral vector serotype 2 (AAV2) containing the human glial cell line-derived neurotrophic factor (GDNF) transgene, allowing for stable and continuous expression of GDNF in localized brain regions after direct neurosurgical injection. GDNF has been evaluated as a potential treatment for diseases marked by progressive degeneration of midbrain dopaminergic neurons, such as Parkinson’s disease.

Parkinson’s disease is a progressive neurodegenerative disorder caused by nerve cell damage in the brain, leading to decreased dopamine levels. The disease affects more than 10 million people worldwide, with symptoms including tremor, rigidity, cramping, and slowness of movement.

AskBio, a wholly owned subsidiary of Bayer AG, is dedicated to developing gene therapies for a range of diseases, including Parkinson’s disease. The company’s gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive capsid and promoter library.