Corcept Highlights Survival Benefit Despite ALS Trial’s Missed Primary Endpoint
Corcept Therapeutics presented results from the Phase 2 DAZALS study of dazucorilant in amyotrophic lateral sclerosis (ALS) at the ENCALS 2025 meeting123.
The 249-patient DAZALS trial randomized participants to 150 mg, 300 mg dazucorilant, or placebo daily for 24 weeks135.
The trial's primary endpoint (change on the ALS Functional Rating Scale-Revised, ALSFRS-R) was not met; there was no statistically significant slowing of functional decline135.
Despite missing the primary endpoint, the secondary endpoint of survival showed significant improvement:
at 24 weeks, no deaths were reported in the 300 mg dazucorilant group, versus five deaths in the placebo group (p = 0.02)135.
A one-year exploratory survival analysis indicated a pronounced treatment effect, with a hazard ratio of 0.16 (p = 0.0009) for 300 mg dazucorilant versus placebo3.
Patients on dazucorilant experienced more gastrointestinal side effects early in treatment than those on placebo5.
A long-term extension study is ongoing, with full one-year survival results expected by March 20254.
Sources:
1. https://www.thepharmaletter.com/biotechnology/mixed-phase-ii-results-for-corcepts-dazucorilant
2. https://ir.corcept.com/news-releases/news-release-details/corcept-presents-results-phase-2-study-dazucorilant-patients-0
3. https://firstwordpharma.com/story/5969481
4. https://www.ainvest.com/news/corcept-therapeutics-dazucorilant-als-trial-survival-breakthrough-functional-setbacks-2506/
5. https://www.benzinga.com/markets/biotech/25/06/45796638/corcept-therapeutics-therapy-fails-primary-goal-but-extends-patient-survival-in-amyotrophic-lateral-sclerosis-study