Corcept Therapeutics announced results from its Phase 2 DAZALS study of dazucorilant in patients with amyotrophic lateral sclerosis (ALS). The study did not achieve its primary endpoint of slowing the decline in motor skills and other functional criteria. However, a noteworthy observation was the lower mortality rate in the higher dose dazucorilant group compared to the placebo group.
This news holds significant implications for the ALS patient community, which currently faces limited treatment options for this devastating and fatal disease. Although the primary endpoint was not met, the survival data offers a potential new avenue for investigation in a disease where extending survival is a critical goal. This unexpected finding could shift the focus of ALS research towards cortisol modulation and its potential role in disease progression. The reduced mortality, even in the absence of functional improvement, could indicate a neuroprotective effect of dazucorilant, warranting further exploration.
The DAZALS study involved 249 patients randomized to receive either 150 mg or 300 mg of dazucorilant, or a placebo, daily for 24 weeks. While the primary endpoint using the ALSFRS-R scale was not met, there were no deaths in the 300 mg dazucorilant arm compared to five deaths in the placebo arm, a statistically significant result. Patients experienced more gastrointestinal side effects with dazucorilant. An open-label extension study, where all participants receive 300 mg of dazucorilant, is ongoing and will assess overall survival in March 2025.
The unexpected survival benefit observed in the DAZALS study, despite the lack of functional improvement, presents a complex but hopeful outlook for ALS research. While further research is crucial to understand the mechanism behind this observation and to confirm these findings in larger studies, it opens up the possibility of a new therapeutic strategy for ALS. The results could lead to renewed interest in cortisol modulation as a potential target for neuroprotection in ALS and potentially other neurodegenerative diseases. This could trigger further investment in research and development of selective cortisol modulators, leading to potential new treatments that focus on slowing disease progression and extending survival for individuals with ALS.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
