ABLi Therapeutics recently completed an End of Phase 2 meeting with the FDA to discuss results from their Parkinson’s disease trial and plan their Phase 3 program. The company’s drug, risvodetinib, showed promising results in Phase 2, achieving nominal significance in key measures of motor skills and daily living activities. Importantly, the FDA supported ABLi’s proposed primary endpoint for the Phase 3 trial, further validating the clinical relevance of their approach.
This progress is particularly encouraging for Parkinson’s patients, as it suggests a potential new treatment option focusing on disease modification. Current therapies primarily address symptoms, leaving a significant unmet need for treatments that slow or halt the disease’s progression. Risvodetinib’s potential to delay the need for levodopa/carbidopa, a standard Parkinson’s treatment, could represent a significant advancement in patient care. This signifies a shift towards targeting the underlying disease mechanisms, offering hope for improved long-term outcomes.
Technically, the FDA accepted ABLi’s proposal to use the change in MDS-UPDRS Part 2 as the primary outcome measure for the Phase 3 trial, aligning with their focus on patient quality of life. The agency also agreed with the novel endpoint of measuring the time to levodopa/carbidopa initiation, which ABLi considers a direct measure of disease modification. Furthermore, the FDA simplified the vision monitoring requirements for the Phase 3 trial based on the safety data observed so far. While the company is exploring skin biopsy as a potential prognostic marker, the FDA advised further evaluation before it can be considered a definitive measure of treatment success.
This positive feedback from the FDA paves the way for a streamlined and focused Phase 3 program for risvodetinib. The alignment on key endpoints and the reduced monitoring requirements increase the efficiency and likelihood of success for the upcoming trials. Positive Phase 3 results could lead to the first disease-modifying therapy for Parkinson’s disease, significantly altering the treatment landscape and offering new hope for patients.
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.
