STAT+: Closely watched experimental Parkinson’s drug fails key clinical trial
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- Biogen and Denali Therapeutics said their experimental Parkinson’s therapy failed to meet the primary endpoint in a randomized trial.
- The trial enrolled 648 Parkinson’s patients who received either a placebo or a LRRK2‑targeting pill.
- LRRK2 is a protein whose gene mutations cause a rare inherited form of Parkinson’s, discovered in 2004, and was hypothesized in 2018 to be a therapeutic target for all patients.
- The failure undermines the hypothesis that LRRK2 inhibition could broadly treat Parkinson’s disease.
Why it matters: Patients lose a potential disease‑modifying therapy; Biogen and Denali investors see a setback that delays the launch of a new Parkinson’s drug, and researchers shift focus away from LRRK2 inhibition.
