ABLi Therapeutics has announced final outcomes from its Phase II 201 Trial of risvodetinib in untreated Parkinson’s disease (PD), achieving the primary endpoint of tolerability and safety.
The selective, brain-penetrant c-Abl kinase inhibitor demonstrated a side-effect profile comparable to placebo.
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Results were presented at the 2025 Movement Disorders Society Annual Congress.
The first long-term dosing 201 Trial is designed to examine risvodetinib as a monotherapy. It evaluated doses of 50mg, 100mg and 200mg in 126 subjects randomised 1:1:1:1 to receive either placebo or risvodetinib, administered once daily for 12 weeks.
Human exposure to risvodetinib is between two and 200-times higher than that of any c-Abl inhibitor currently approved for use in humans.
Risvodetinib met its primary safety endpoint, with the incidence and average number of adverse events per individual similar to placebo.
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By GlobalDataABLi noted that 95% of enrolled subjects completed the 12-week period, with dosing compliance of 99%.
Common adverse events including oedema, nausea, vomiting, diarrhoea and cardiovascular incidents were no more frequent in risvodetinib-treated subjects against those receiving placebo.
Key secondary endpoints related to activities of daily living favoured risvodetinib treatment across all dose levels. The MDS-UPDRS Part 1 and 2 and the Schwab & England Activities of Daily Living (SEADL) scales all showed improvements.
Movement Disorders Society Unified Parkinson’s Disease Rating Scale (MDS-UPDRS) Part II and SEADL scales achieved nominal statistical significance at doses of 100mg and 50mg, respectively.
ABLi Therapeutics chairman and CEO Dr Milton Werner said: “We had seen a good safety profile with seven-day dosing but didn’t anticipate the drug would have an adverse event profile similar to placebo at longer dosing durations, with none of the typical side-effects of other drugs in the class.
“We think the most important measure beyond safety is the confirmation that risvodetinib can reduce the alpha-synuclein pathology that drives PD. This outcome is the first measure of an experimental treatment reducing the disease-causing pathology of PD.”
