Corcept's ALS Drug Shows Promising Survival Data Despite Phase 2 Failure

Corcept Therapeutics has unveiled new data from its phase 2 amyotrophic lateral sclerosis (ALS) trial, shedding light on the potential of its drug dazucorilant despite initial setbacks. The company presented these findings at the European Network to Cure ALS 2025 annual meeting, offering a glimmer of hope in the challenging landscape of ALS treatment.

Survival Benefit Emerges as Key Finding

While the primary endpoint of reducing decline in motor skills and functional criteria was not met, Corcept has highlighted a significant improvement in patient survival among those receiving the higher dose of dazucorilant. In the 300 mg cohort, no patients had died by week 24, compared to two deaths in the 150 mg arm and five in the placebo group.

This survival benefit appeared to persist in a 12-month follow-up analysis. Corcept reported 14 deaths in the 300 mg group, versus 23 in the 150 mg group and 21 in the placebo arm by this time point. Bill Guyer, Corcept's Chief Development Officer, emphasized the urgent need for life-extending ALS treatments, stating, "We are working with regulatory authorities to determine the optimal path for advancing dazucorilant."

Safety Profile and Adverse Events

The trial data also provided insights into dazucorilant's safety profile. While the drug was associated with more gastrointestinal upset at the onset, Corcept maintains that it demonstrated "an acceptable safety profile" overall. The company noted that 92% of adverse events were mild to moderate in severity.

Severe treatment-emergent adverse events (TEAEs) were observed in 19% of patients in the 300 mg group, compared to 17% in the 150 mg group and 13% in the placebo cohort. Importantly, no patients in the 300 mg group died as a result of a TEAE, whereas two patients in the 150 mg group and four in the placebo group experienced fatal TEAEs.

Market Response and Future Prospects

The market has responded positively to these new findings, with Corcept's stock up more than 1.4% premarket on Thursday, reflecting a $7.4 billion market cap. As the company continues to analyze the data and engage with regulatory authorities, the future of dazucorilant in ALS treatment remains a focal point for both Corcept and the broader pharmaceutical industry.