Corcept Therapeutics announced 2-year OS data from Phase 2 dazucorilant study

Corcept Therapeutics (CORT) “announced two-year overall survival data from the Phase 2 DAZALS study of its proprietary, selective cortisol modulator dazucorilant in patients with amyotrophic lateral sclerosis (ALS). DAZALS is a randomized, double-blind, 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. Patients who completed the treatment period were eligible to enter the study’s long-term extension phase, in which all patients received 300 mg of dazucorilant. DAZAL’s primary endpoint was the difference in function, as measured by the ALS Functional Rating Scale – Revised (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, at the end of the 24-week treatment period patients who received 300 mg of dazucorilant daily did exhibit improved overall survival (p-value: 0.02). Exploratory analyses show that this survival benefit has continued. In the two years following the start of treatment, patients who received 300 mg of dazucorilant experienced an 87 percent reduction in the risk of death compared to patients who received placebo and did not switch to 300 mg of dazucorilant in the extension phase. This finding is consistent with the 84 percent reduction in risk of death observed after one year of treatment (hazard ratio: 0.16; p-value: 0.0009). Those one-year data were presented at the European Network to Cure ALS (ENCALS) 2025 annual meeting. A similarly pronounced survival benefit was observed at both the one-year and two-year marks in patients who received 300 mg of dazucorilant for more than 24 weeks, whether in the treatment period or in the long-term extension phase, compared to patients who received either placebo or 150 mg of dazucorilant for more than 24 weeks and did not receive dazucorilant in the extension phase. Between these groups, the reduction in risk of death in patients who received 300 mg of dazucorilant was 64 percent at one year (hazard ratio: 0.36; p-value: 0.02) and 61 percent at two years (hazard ratio: 0.39; p-value: 0.02). Dazucorilant continues to demonstrate an acceptable safety profile, with mild to moderate, dose-related, transient abdominal pain being the most common adverse effect. Corcept is conducting a dose titration study to refine methods of improving dazucorilant’s gastrointestinal tolerability and inform the program’s path forward.”