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A promising new drug for Parkinson's disease may do what current treatments can't — actually slow the disease. Rexaceract, an oral small molecule, kept motor symptom scores stable over 360 days in a phase 1b study, with no new safety signals. Researchers are now gearing up for a placebo-controlled phase 2 trial.
A promising new drug for Parkinson's disease may do what current treatments can't — actually slow the disease.
Current Parkinson's therapies like levodopa and dopamine agonists manage symptoms but don't stop the underlying neurodegeneration. Rexaceract, developed by Gain Therapeutics, takes a different approach: it targets GCase, a key lysosomal enzyme, restoring its function to reduce the toxic buildup of alpha-synuclein — a hallmark of Parkinson's pathology. Notably, the drug appears to work even in patients without the GBA1 gene mutation.
In a phase 1b open-label study, 12 patients with Parkinson's disease completed up to 360 days of treatment. MDS-UPDRS motor scores — which typically climb around 6 points per year — remained flat throughout. Some patients also reported improvements in smell, taste, balance, and concentration. The drug was well-tolerated, with most adverse events classified as mild and fewer safety signals in the extension phase than in the initial dosing period.
By the Numbers:
Why it matters: If confirmed in a phase 2 placebo-controlled trial, rexaceract could become one of the first true disease-modifying therapies for Parkinson's — a significant step forward for the millions living with a condition that current medicine can only partially manage.
