AskBio, a Bayer subsidiary, has initiated a Phase II clinical trial, REGENERATE-PD, for its Parkinson’s disease gene therapy, AB-1005 (AAV2-GDNF). The trial will evaluate the safety and efficacy of AB-1005 in adults with moderate-stage Parkinson’s disease across clinical sites in the United States, Germany, Poland, and the United Kingdom. Positive 36-month Phase Ib data showcased a favorable safety profile and encouraging trends in clinical measures.
The commencement of the Phase II trial signifies a crucial step towards developing a potentially disease-modifying treatment for Parkinson’s disease. Current therapies primarily manage symptoms, but the progressive nature of the disease necessitates treatments that can slow or halt neuronal degeneration. AB-1005, by delivering the GDNF gene directly to the brain, aims to support the survival of dopamine-producing neurons, potentially altering the disease course and improving long-term outcomes for patients. This approach holds considerable promise for addressing the significant unmet need in Parkinson’s disease treatment.
REGENERATE-PD is a randomized, double-blind, sham-controlled trial that will enroll approximately 87 participants aged 45-75. The primary objective is to evaluate both the safety and efficacy of intraputaminal administration of AB-1005. Previously reported 36-month Phase Ib data indicated no serious adverse events related to AB-1005, and participants exhibited improvements or maintained stability in motor function assessments. AskBio is also investigating AB-1005 for multiple system atrophy, a related neurodegenerative disorder.
The advancement of AB-1005 into Phase II testing reinforces the potential of gene therapy to revolutionize Parkinson’s disease treatment. The results of this trial will be critical in determining the future development and potential regulatory approval of this promising therapeutic. Positive data could not only lead to a new treatment option for patients with moderate-stage Parkinson’s disease but also validate the approach of using GDNF gene therapy for neurodegenerative conditions, stimulating further research and development in this field.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
