Corcept Therapeutics’ selective cortisol modulator failed to slow the progress of amyotrophic lateral sclerosis (ALS) in a phase 2 trial and was also 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. The trial missed the primary endpoint of demonstrating a reduction in the decline in motor skills and other functional criteria, the company explained in a postmarket release Dec. 11.
In addition, patients in the dazucorilant cohorts “experienced substantially more gastrointestinal upset at the onset of treatment than those who received placebo,” the biotech pointed out.
Back in October, Corcept’s chief development officer Bill Guyer had pointed to dazucorilant’s “great promise in an animal model of ALS,” including “improving motor performance and reducing neuroinflammation and muscular atrophy.”
The biotech also alluded to “increasing evidence that patients with ALS, particularly those with rapid disease progression, exhibit elevated or abnormal cortisol levels.”
This year has been a reminder of what a tricky space ALS is to work in. Amylyx Pharmaceuticals pulled its ALS therapy Relyvrio off the market after the drug failed in a confirmatory trial, while PTC Therapeutics dropped its candidate in the wake of a phase 2 fail and Metagenomi was prompted by Biogen and Ionis’ problems to halt its own ataxin-2-focused treatment for ALS.
For now, it appears Corcept will continue to develop dazucorilant as an open-label, long-term extension study will continue, according to the company, with overall survival to be assessed in March 2025.
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 earlier this year and is being lined up for an approval application to the FDA before the end of the year.