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On January 4, 2024, Bayer AG and its wholly-owned, independently operated subsidiary Asklepios BioPharmaceutical, Inc. (AskBio) announced that the 18-month data collection for the Phase Ib clinical trial of AB-1005 (AAV2-GDNF) has been completed.
AB-1005 is an investigational gene therapy based on the adeno-associated virus serotype 2 vector (AAV2), containing the human glial cell line-derived neurotrophic factor (GDNF) transgene. Following direct neurosurgical injection via convection-enhanced delivery monitored by magnetic resonance imaging (MRI), GDNF can achieve stable and continuous expression in localized brain regions. GDNF is a homodimer and a distant member of the transforming growth factor (TGF)-β superfamily. In midbrain neuronal cell cultures, recombinant human GDNF promotes the survival and morphological differentiation of dopaminergic neurons and enhances high-affinity dopamine uptake. For a long time, GDNF has been considered to have the potential to treat diseases characterized by "progressive degeneration of midbrain dopaminergic neurons," such as Parkinson's disease.
The study met its primary endpoint,To evaluate the safety of AB-1005 administered as a one-time bilateral putaminal infusion. Based on the time of Parkinson’s disease diagnosis and symptom severity at screening, 11 patients were divided into two groups: mild Parkinson’s disease (six patients) and moderate Parkinson’s disease (five patients). All patients tolerated the neurosurgical administration of AB-1005 well, with a target putamen coverage rate of 63% ± 2%, exceeding the goal of over 50% coverage. No serious adverse events related to AB-1005 were observed, and clinical follow-up will continue for five years post-administration.Patients also regularly completed neurological assessments and self-rating questionnaires over an 18-month period to evaluate the severity of motor and non-motor symptoms associated with Parkinson's disease. Additionally, longitudinal brain imaging was conducted to assess safety and potential changes in dopamine processing or abnormal metabolic patterns associated with Parkinson's disease.AskBio Plans to Release 18-Month Study Data, Including Secondary Endpoints, at an Academic Conference in Q2 2024. The Phase II Clinical Trial Is in Planning and Expected to Begin Patient Recruitment in the First Half of 2024. The Trial Design Has Been Adjusted Based on Feedback from Health Authorities in the U.S. and Europe.AskBio Parkinson's Disease and Multiple System Atrophy Scientific DirectorKrystof Bankiewicz stated"These early data are encouraging, indicating that AB-1005 has good tolerability in patients with mild to moderate Parkinson's disease in this study. Although this early investigational gene therapy still has much to be further researched, these preliminary findings will inform our work in this area and have the potential to contribute to clinical improvements in AB-1005 for the treatment of Parkinson’s disease."
