Risvodetinib
Phase 2: mid-stage testing
An oral c-Abl inhibitor aimed at slowing Parkinson's. Its Phase 2 trial showed it was safe and well tolerated, but was not designed to prove it slows the disease.
- Stage: Phase 2 (201 Trial complete)
- Type: Disease-modifying
- Developer: ABLi Therapeutics (formerly Inhibikase Therapeutics)
- Target: c-Abl kinase / alpha-synuclein pathway
- How it works: A brain-penetrant pill that blocks an enzyme called c-Abl, which is thought to help drive the misfolding and spread of alpha-synuclein in Parkinson's.
- Who it's for: People with early, untreated Parkinson's.
- Last reviewed: 2026-08-07
What it is
c-Abl is an enzyme that becomes overactive in Parkinson's and appears to worsen the clumping of alpha-synuclein and to impair the cell's clean-up machinery. Risvodetinib is a newer, more selective c-Abl blocker, chemically related to the cancer drug imatinib (Gleevec), designed to reach the brain and calm this pathway.
Where it stands
The Phase 2 "201 Trial" tested three once-daily doses against placebo in early, untreated Parkinson's. Its main purpose was to check safety and tolerability, and final results were published in early 2026. Larger trials designed to test whether it actually slows the disease would be the next step.
What the data shows so far
The randomized, placebo-controlled 201 Trial enrolled 137 people with early, untreated Parkinson's on 50 mg, 100 mg, 200 mg, or placebo once daily over its dosing period. Its pre-specified primary endpoints were safety and tolerability, which it met, with a side-effect profile similar to placebo. Crucially, it was not powered or designed to prove the drug slows Parkinson's — any change in Parkinson's rating scales was exploratory only — and earlier c-Abl inhibitors (such as nilotinib) failed to show clear benefit in controlled trials.
What families should know
This is an early, encouraging safety result, not evidence that the drug slows Parkinson's. The c-Abl approach as a whole has repeatedly disappointed, so a clean safety read-out is a starting point, not a finish line.
Caveats
Not proven to slow Parkinson's; the Phase 2 trial tested safety and tolerability, not efficacy. Related c-Abl inhibitors have failed in controlled Parkinson's trials. Larger, longer, efficacy-powered trials are needed before any conclusions.
Timeline
Most recent first.
- 2026, Final 201 Trial results published; primary safety/tolerability goals met.
- 2022-2024, Phase 2 "201 Trial" conducted in early untreated Parkinson's.
Sources
Last reviewed: 2026-08-07. Back to the Parkinson's drug pipeline