Corcept’s ALS Modulator Fails to Meet Primary Endpoint in Phase 2 Study

After Amylyx and PTC, Corcept has also decided to withdraw from the amyotrophic lateral sclerosis (ALS) space. Results from its Phase 2 study, designed to assess the efficacy of its selective cortisol modulator in slowing ALS progression, revealed that the treatment failed to meet the primary endpoint of reducing motor skill decline and other functional criteria.
Additionally, participants receiving the treatment experienced significantly more gastrointestinal side effects.
The mid-stage trial included approximately 250 patients from Europe, the U.S., and Canada, who were administered either 150 mg or 300 mg doses of dazucorilant or a placebo over a 24-week period. By the end of the study, it was evident that the treatment neither reduced motor decline progression nor provided any functional benefits. Furthermore, patients in the dazucorilant groups experienced notably higher rates of gastrointestinal upset compared to those in the placebo group, particularly at the start of treatment. This information was disclosed in a post-market release earlier this week.

The results are particularly disappointing given the optimism expressed just months earlier. In October, Bill Guyer, Corcept’s Chief Development Officer, stated that “dazucorilant has great promise in an animal model of ALS, including improving motor performance and reducing neuroinflammation and muscular atrophy.”
Corcept has previously highlighted literature suggesting that ALS patients, especially those with rapid disease progression, often exhibit elevated or abnormal cortisol levels.
Despite the setback, the biotech plans to continue developing dazucorilant as part of an open-label study. A long-term extension trial is also ongoing, with an assessment of overall survival outcomes scheduled for March of next year.
Corcept’s clinical-stage pipeline is currently led by a treatment for Cushing’s syndrome, which demonstrated positive results in a Phase 3 study earlier this year. The anti-glucocorticoid relacorilant is now being prepared for regulatory submission, with an FDA approval application expected before the end of the year.
Other companies, including Amylyx Pharmaceuticals, PTC Therapeutics, and Metagenomi, have also faced challenges in the ALS field. Amylyx withdrew its therapy Relyvrio after a failed confirmatory trial. Similarly, PTC dropped its candidate following Phase 2 trial failures, while Metagenomi opted to discontinue its ALS program, following Biogen and Ionis, who halted their own treatment efforts due to various challenges.

Corcept, headquartered in Redwood City, California, has an FDA-approved cortisol receptor blocker, Korlym (mifepristone), for managing hyperglycemia caused by elevated cortisol levels in the blood.

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