Corcept Therapeutics has tried to cast its recent phase 2 amyotrophic lateral sclerosis (ALS) failure in a more flattering light by pointing to promising survival data.
The biopharma revealed in December 2024 that the drug, called dazucorilant, had failed to demonstrate a reduction in the decline in motor skills and other functional criteria, while being linked to “substantially more gastrointestinal upset.” The midstage study saw 249 patients across the U.S., Canada and Europe receive either 150 mg or 300 mg of dazucorilant or placebo daily for 24 weeks.
In a presentation at the European Network to Cure ALS 2025 annual meeting this week, Corcept included a table showing that the decline as measured by a functionality scale was broadly the same across both dazucorilant cohorts and the placebo group over 24 weeks.
However, Corcept pointed out that patient survival—a secondary endpoint for the study—“significantly improved” among the 83 patients who received the 300 mg dazucorilant dose. None of these patients had died by week 24, Corcept noted, compared to two patients in the 150 mg arm and five patients who received placebo.
The biopharma has since conducted an analysis at 12 months that it said showed this survival benefit has continued. Specifically, 14 patients in the 300 mg group had died by this point, compared to 23 deaths in the 150 mg group and 21 deaths in the placebo arm—equivalent to a hazard ratio of 0.16 for the 300 mg group and placebo groups.
Speaking to Fierce at the American Society of Clinical Oncology meeting in Chicago this week, Adrian Jubb, M.D., Ph.D., Head of Oncology and Neurology Clinical Development at Corcept, said the company had not originally planned “to look at the data this way.”
“Nonetheless, in a disease like this, when you see an endpoint that's really clinically meaningful, like survival, it makes you stop and think,” Jubb told Fierce. “And so we're now looking at the data in a much more positive light than simply a miss on a primary objective.”
When it came to safety, this morning's data showed that 19% of patients in the 300 mg group experienced at least one severe treatment-emergent adverse event (TEAE), compared to 17% of the 150 mg group and 13% of the placebo cohort. No patients died as a result of a TEAE in the 300 mg group, compared to two patients in the 150 mg group and four patients in the placebo group.
Overall, dazucorilant demonstrated “an acceptable safety profile,” according to Corcept, which pointed out that 92% of adverse events were mild to moderate in severity.
“Medications that can extend life for patients with ALS are urgently needed,” Corcept’s Chief Development Officer Bill Guyer said in a June 5 statement. “We are working with regulatory authorities to determine the optimal path for advancing dazucorilant.”
The Redwood City, California-based biotech markets the FDA-approved cortisol receptor blocker Korlym, also known as mifepristone, as a treatment for hyperglycemia caused by high cortisol levels in the blood. Corcept’s clinical-stage pipeline is headed up by relacorilant, an antiglucocorticoid that secured a phase 3 win in Cushing’s syndrome last year and is awaiting an FDA approval decision by the end of the year.
The biotech was up more than 1.4% premarket Thursday morning on a $7.4 billion market cap.