Corcept Therapeutics Incorporated reports two-year survival data from the DAZALS Phase 2 trial showing significant mortality reduction with Dazucorilant in Amyotrophic Lateral Sclerosis, despite missing the primary functional endpoint.
Written by: Fariha Sameen, Pharm D.
Reviewed By: Pharmacally Editorial Team
Corcept has reported updated two-year overall survival data from its Phase 2 DAZALS study evaluating dazucorilant in patients with Amyotrophic Lateral Sclerosis. The findings show a sustained survival benefit, despite the study not meeting its primary endpoint.
DAZALS is a randomized, double-blind, placebo-controlled trial that enrolled 249 patients with ALS. Participants received either 150 mg or 300 mg of dazucorilant, or placebo, once daily for 24 weeks. Patients completing this phase were eligible for a long-term extension, during which all participants received 300 mg of the drug.
The primary endpoint assessed functional decline using the ALS Functional Rating Scale–Revised (ALSFRS-R). The study did not demonstrate a statistically significant difference between treatment and placebo groups on this measure. However, overall survival was a predefined secondary endpoint.
At 24 weeks, patients treated with 300 mg of dazucorilant showed improved survival compared with placebo (p=0.02). Longer-term follow-up indicated that this benefit persisted. At two years, patients initially treated with 300 mg experienced an 87% reduction in the risk of death compared with those who received placebo and did not switch to active treatment in the extension phase (hazard ratio 0.13; p<0.0001).
This result aligns with earlier one-year data, which showed an 84% reduction in mortality risk (hazard ratio 0.16; p=0.0009), previously presented at the ENCALS 2025 annual meeting.
Additional analyses showed consistent benefit in patients receiving prolonged exposure to the 300 mg dose. Among patients treated for more than 24 weeks, the risk of death was reduced by 64% at one year and 61% at two years compared with those who received placebo or the lower dose and did not transition to active treatment.
Dazucorilant maintained an acceptable safety profile. The most common adverse event was mild to moderate, dose-related abdominal pain, which was transient. The company is currently conducting a dose titration study to improve gastrointestinal tolerability.
Corcept’s Chief Development Officer, Bill Guyer, stated that the data indicate a meaningful reduction in mortality during the early years after diagnosis, when patients retain functional capacity and quality of life. He added that the company is engaging with regulators and plans to initiate a Phase 3 trial later this year.
ALS is a progressive neurodegenerative disorder affecting more than 55,000 people across the United States and Europe. The disease leads to progressive muscle weakness, ultimately impairing speech, movement, and breathing. Median survival following diagnosis is typically two to five years.
Dazucorilant is a selective modulator of the glucocorticoid receptor and does not bind to other hormone receptors. It has received Fast Track designation and orphan drug status from the U.S. Food and Drug Administration for ALS, supporting its continued clinical development.
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