Corcept Therapeutics’ dazucorilant did not meet primary endpoint in ALS study

Corcept Therapeutics (CORT) presented results from its DAZALS study of dazucorilant in patients with ALS at the European Network to Cure ALS 2025 annual meeting. DAZALS is a placebo-controlled Phase 2 study in which 249 patients with ALS were randomized to receive either 150 mg of dazucorilant, 300 mg of dazucorilant or placebo, daily for 24 weeks. The primary endpoint in DAZALS was the difference in ALSFRS-R between patients who received dazucorilant and those who received placebo. Overall survival was a secondary endpoint. Although DAZALS did not meet its primary endpoint, patient survival significantly improved. At week 24 of the study, no deaths had occurred in the 83 patients who received 300 mg of dazucorilant, while there were five deaths in the 82-patient placebo group. An exploratory analysis conducted at the one-year mark shows the survival benefit has continued. Patients randomized to 300 mg of dazucorilant lived significantly longer than patients who received placebo and did not switch to 300 mg of dazucorilant in the extension study. The difference between groups was pronounced. A similar survival benefit was observed in patients who received 300 mg of dazucorilant for greater than 24 weeks. The extension study is ongoing. Dazucorilant has demonstrated an acceptable safety profile, with 92% of adverse events being mild to moderate in severity. Mild to moderate, dose-related, transient abdominal pain was the most common adverse effect.