Bayer's AskBio checks off another early win for gene therapy platform with Parkinson's safety data

Bayer’s AskBio gene therapy unit has achieved a key safety milestone in an early-stage trial for an investigational Parkinson’s disease treatment that must be delivered surgically into the brain.

The phase 1b study of gene therapy AB-1005 met the key primary endpoint of safety, showing that the one-time medicine can be delivered to the putamen region of the brain, according to a Thursday press release. Delivery of the treatment was well-tolerated by all 11 patients in the small trial. There have been no serious adverse events attributed to the therapy.

Patients also exceeded a goal of greater than 50% putamen coverage at 63%.

“Although there is still much to learn about this early-stage investigational gene therapy, these first findings will inform our work in this space and have the potential to contribute to the clinical advancement of AB-1005 for the treatment of Parkinson’s disease,” said Krystof Bankiewicz, M.D., Ph.D., scientific chair of Parkinson’s and MSA at AskBio in a Thursday statement.

The study featured 11 patients split between two cohorts of mild (six patients) and moderate (five patients) Parkinson’s disease. AB-1005 was administered to the brain via one-time bilateral convection-enhanced delivery. The main goal was to evaluate the safety and potential clinical effect of the treatment. Patients were evaluated at 18 months for treatment-emergent adverse events, motor symptoms and brain dopaminergic network integrity. The study will continue to follow the patients for up to five years.

AskBio will present the 18 month results plus secondary endpoint data at a medical meeting in the second quarter. The company is also moving forward with planning for a phase 2 study of the gene therapy that should begin screening patients this half.

Last year, AskBio’s congestive heart failure gene therapy showed even better uptake in initial human data compared to previous preclinical testing—a result that “confounded” the company’s scientists. The data helped provide early validation of AskBio’s young AAV-focused pipeline. With AB-1005 now advancing, AskBio could have it’s first phase 2 asset.

The early data also provides validation for Bayer, which bought the gene therapy company for $4 billion in October 2020.