Company: CORT
Filing Date: 2025-05-05
Form Type: 10-Q
Source: 0001628280-25-022173
Chunk: 97

Company: CORCEPT THERAPEUTICS INC
Filing Date: 2025-05-05
Form: 10-Q
Item: Part I, Item 2
Chunk 97
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 cortisol modulator dazucorilant improved motor performance and reduced neuroinflammation and muscular atrophy in an animal model of ALS. Following these compelling results, we initiated a Phase 2 trial (“DAZALS”) of dazucorilant in patients with ALS. Two hundred forty-nine patients were randomized on a double-blind basis 1:1:1 to receive either 150 mg of dazucorilant, 300 mg of dazucorilant or placebo daily for 24 weeks. Upon completion of the trial, patients were eligible to enter an open-label, long-term extension study, in which they receive 300 mg of dazucorilant for up to 132 weeks. DAZALS did not meet its primary endpoint, which was the change from baseline in the ALS Functional Rating Scale-Revised (ALSFRS-R) in patients who received dazucorilant compared to those who received placebo. Patients who received dazucorilant also experienced substantially more gastrointestinal upset at the onset of treatment than did those who received placebo. A statistically significant improvement in overall survival was observed at week 24 of the study. An exploratory analysis of overall survival at the one-year mark showed a reduction in deaths in patients who were randomized to receive 300 mg of dazucorilant at baseline, compared to patients who were randomized to receive placebo at baseline and never received 300 mg of dazucorilant, with a 

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hazard ratio of 0.16 (p-value: 0.0009). The open-label, long-term extension study, which enrolled 118 patients, will continue. The FDA has granted dazucorilant Fast Track Designation and orphan drug status for the treatment of ALS in the United States.

Metabolic Diseases

Liver Disease. Metabolic dysfunction-associated steatohepatitis (“MASH”) is an advanced form of metabolic dysfunction-associated fatty liver disease that afflicts millions of patients and is a leading cause of liver-related mortality. Our Phase 1b trial of the selective cortisol modulator miricorilant as a potential treatment for MASH identified a dosing regimen that reduced liver fat, improved liver health and key metabolic and lipid measures and was well-tolerated. Following these compelling results, we initiated a randomized, double-blind, placebo-controlled, Phase 2b trial (“MONARCH”) of miricorilant in patients with MASH in October 2023. MONARCH