Exploring Risvidetinib in Phase 2 Trial for Untreated Parkinson Disease: Milton Werner, PhD

WATCH TIME: 5 minutes | Captions are auto-generated and may contain errors.

"When risvodetinib is given orally once daily, it has a very long half-life. It has virtually no adverse effects. It’s hard to distinguish it from placebo, and in key aspects—cardiovascular, gastrointestinal, or fluid retention adverse events common to this drug class—we see none of that. Risvodetinib is more favorable than placebo. It’s really unexpected for this drug class."

Risvodetinib (ABLi Therapeutics) is a potent, selective small-molecule inhibitor of the nonreceptor c-Abl kinases, designed for once-daily oral use to target the underlying biological mechanisms of Parkinson disease (PD). The agent is currently being assessed in the multicenter, double-blind, placebo-controlled 201 Trial (NCT05424276) in patients with early and untreated PD. Within the trial, investigators evaluated a 50-mg, 100-mg or 200-mg dose of risvodetinib or placebo given once daily in 126 participants randomized 1:1:1:1 for 12 weeks.^1,2

Final results from the phase 2 trial of risvodetinib, presented at 2025 International Congress of Parkinson’s Disease and Movement Disorders (MDS), held October 5-9, in Honolulu, Hawaii, showed that the study met its primary safety and tolerability end points in patients with untreated PD. Presented by lead author Milton Werner, PhD, as the “Clinical Breakthrough Lecture” during the keynote session, results demonstrated that risvodetinib was generally well tolerated among participants, with mild to moderate adverse events reported at a frequency and severity comparable to placebo.

In an interview with NeurologyLive^® during the meeting, Werner, PhD, chairman and CEO at ABLi Therapeutics, detailed the research and clinical progress behind risvidetinib as a potential treatment for PD. He highlighted the presented phase 2 data from the trial, showing strong safety and tolerability, as well as early dose-dependent improvements in activities of daily living. He also emphasized that exploratory biomarker data revealed significant reductions in α-synuclein pathology in patient skin biopsies after 12 weeks. With these positive findings, he noted that the development program of risvidetinib may move forward to a phase 3 evaluation.

Click here for more coverage of MDS 2025.

REFERENCES
1. Werner M, Meyer C, Mancino E, et al. A Phase 2 Study of Risvodetinib in Untreated Parkinson’s Disease. Presented at: International Congress of Parkinson’s Disease and Movement Disorders; October 5-9, 2025; Honolulu, HI.
2. ABLi Therapeutics Reports Final Results from the Phase 2 ‘201 Trial’ Evaluating Risvodetinib for the Treatment of Parkinson’s Disease. News release. October 9, 2025. Accessed November 13, 2025. https://www.ablitherapeutics.com/news/detail/9613/abli-therapeutics-reports-final-results-from-the-phase-2-201-trial-evaluating-risvodetinib-for-the-treatment-of-parkinsons-disease