Corcept’s ALS Drug Trial Misses Primary Endpoint, Yet Shows Promising Survival Benefit

Primary Endpoint Missed:
Corcept's Phase 2 trial for dazucorilant in ALS patients failed to meet its primary endpoint, which was to slow the decline in motor skills and other functional criteria as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R)12.

Survival Benefit:
Despite missing the primary endpoint, the study found a significant survival benefit with zero deaths in the 300mg treatment group compared to 5 deaths in the placebo group (p=0.02)12.

Gastrointestinal Side Effects:
Patients who received dazucorilant experienced substantially more gastrointestinal upset at the onset of treatment compared to those who received placebo12.

Fast Track Designation:
Dazucorilant has been granted Fast Track Designation by the FDA, indicating its potential to address unmet medical needs in treating ALS12.

Open-Label Extension Study:
An open-label, long-term extension study is ongoing, with overall survival assessment planned for March 2025 after all patients have had one year pass since the onset of treatment12.

Study Design: The DAZALS study enrolled 249 patients, randomized 1:1:
1 to receive either 150mg of dazucorilant, 300mg of dazucorilant, or placebo daily for 24 weeks12.

Sources:

1. https://www.stocktitan.net/news/CORT/corcept-announces-results-from-phase-2-study-of-dazucorilant-in-ysx3nrep5s6p.html

2. https://www.businesswire.com/news/home/20241211463748/en/Corcept-Announces-Results-From-Phase-2-Study-of-Dazucorilant-in-Patients-With-Amyotrophic-Lateral-Sclerosis-ALS