Gene Therapy AB-1005 for Parkinson’s Disease Meets Primary Endpoint in Phase 1b Trial

Jan 05, 2024 07:38 CST Updated 07:38

AskBio

AAV Gene Therapy Developer

Bayer

Pharmaceutical Product R&D Developer

▎Edited by the WuXi AppTec content team

BayerBayer AG and its wholly-owned subsidiary Asklepios BioPharmaceutical (AskBio)Tian announced,AB-1005 (AAV2-GDNF), an investigational gene therapy for the treatment of Parkinson's disease (PD), met the primary endpoint in a Phase 1b clinical trial, demonstrating good safety in 11 treated patients.Based on this result, the two companies plan to launch Phase 2 clinical trials in the first half of 2024.

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.Recombinant human GDNF can promote the survival and morphological differentiation of dopaminergic neurons, and increase 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.

In treating central nervous system diseases, how to deliver gene therapy to the brain regions affected by the disease is an important challenge that needs to be addressed. Dr. Amber Van Laar, Vice President of Clinical Development for CNS Gene Therapy at AskBio, stated in an interview with the WuXi AppTec content team,Monitoring drug delivery to the brain through MRI imaging is a significant technological leap, now allowing neurosurgeons to deliver drugs directly, accurately, and consistently to brain regions affected by specific diseases.

The study met its primary endpoint, which was to evaluate the safety of a single bilateral putaminal administration of AB-1005. Based on the time of diagnosis of Parkinson's disease and the severity of symptoms 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.AB-1005 has not shown any serious adverse events, and the 5-year clinical follow-up after administration is currently ongoing.

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 as well as potential changes in dopamine processing or abnormal metabolic patterns related to Parkinson’s disease.

AskBio Prepares to Present 18-Month Study Data, Including Secondary Endpoint Results, at a Scientific Conference in Q2 2024. Phase 2 Clinical Trial is Currently Being Planned, with Patient Screening Expected to Begin in the First Half of 2024.

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References:

[1] AskBio Phase Ib trial of AB-1005 gene therapy in patients with Parkinson’s disease meets primary endpoint. Retrieved January 4, 2024, from https://www.bayer.com/media/en-us/askbio-phase-ib-trial-of-ab-1005-gene-therapy-in-patients-with-parkinsons-disease-meets-primary-endpoint/

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