Bayer AG and Asklepios BioPharmaceutical, Inc. announced the completion of the 18-month data collection in the Phase Ib clinical trial for AB-1005 (AAV2-GDNF), an investigational gene therapy for treating patients with Parkinson's disease (PD). The study met its primary objective, which was to evaluate the safety of a one-time bilateral delivery of AB-1005 directly to the putamen. Eleven patients were enrolled into two cohorts, Mild stage PD (6 patients) and Moderate stage PD (5 patients), based upon the timing from a PD diagnosis and the severity of their PD symptoms at screening.

Neurosurgical delivery of AB-1005 was well tolerated by all patients with target putamen coverage of 63% ± 2%, exceeding the goal of greater than 50% coverage with AB-1005. No serious adverse events have been attributed to AB-1005, with continued clinical follow-up for up to 5 years post administration ongoing. Patients also completed 18-month neurological assessments and self-reported questionnaires at regular intervals to evaluate the severity of motor and non-motor symptoms associated with PD.

Additionally, brain imaging was performed to longitudinally assess safety and potential changes in dopamine handling or abnormal metabolic patterns associated with PD. AskBio is planning to present the 18-month study data, including secondary endpoints, at a scientific meeting in second quarter 2024. Planning is underway for a Phase II trial that is 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.