REDWOOD CITY, Calif. — Corcept Therapeutics Incorporated (NASDAQ: CORT), a commercial-stage company engaged in the discovery and development of medications to treat severe endocrinologic, oncologic, metabolic and neurologic disorders by modulating the effects of the hormone cortisol, today announced two-year overall survival data from the Phase 2 DAZALS study of its proprietary, selective cortisol modulator dazucorilant in patients with amyotrophic lateral sclerosis (ALS).
DAZALS is a randomized, double-blind, placebo-controlled Phase 2 study in which 249 patients with ALS were randomized to receive either 150 mg of dazucorilant, 300 mg of dazucorilant or placebo, daily for 24 weeks. Patients who completed the treatment period were eligible to enter the study’s long-term extension phase, in which all patients received 300 mg of dazucorilant. DAZAL’s primary endpoint was the difference in function, as measured by the ALS Functional Rating Scale – Revised (ALSFRS-R), between patients who received dazucorilant and those who received placebo. Overall survival was a secondary endpoint.
Although DAZALS did not meet its primary endpoint, at the end of the 24-week treatment period patients who received 300 mg of dazucorilant daily did exhibit improved overall survival (p-value: 0.02). Exploratory analyses show that this survival benefit has continued. In the two years following the start of treatment, patients who received 300 mg of dazucorilant experienced an 87 percent reduction in the risk of death compared to patients who received placebo and did not switch to 300 mg of dazucorilant in the extension phase (hazard ratio: 0.13; p-value: < 0.0001). This finding is consistent with the 84 percent reduction in risk of death observed after one year of treatment (hazard ratio: 0.16; p-value: 0.0009). Those one-year data were presented at the European Network to Cure ALS (ENCALS) 2025 annual meeting.
A similarly pronounced survival benefit was observed at both the one-year and two-year marks in patients who received 300 mg of dazucorilant for more than 24 weeks, whether in the treatment period or in the long-term extension phase, compared to patients who received either placebo or 150 mg of dazucorilant for more than 24 weeks and did not receive dazucorilant in the extension phase. Between these groups, the reduction in risk of death in patients who received 300 mg of dazucorilant was 64 percent at one year (hazard ratio: 0.36; p-value: 0.02) and 61 percent at two years (hazard ratio: 0.39; p-value: 0.02).
Dazucorilant continues to demonstrate an acceptable safety profile, with mild to moderate, dose-related, transient abdominal pain being the most common adverse effect. Corcept is conducting a dose titration study to refine methods of improving dazucorilant’s gastrointestinal tolerability and inform the program’s path forward.
“Our data demonstrate that dazucorilant markedly reduces mortality in the first years following diagnosis, when people with ALS retain meaningful function and quality of life,” said Bill Guyer, PharmD, Corcept’s Chief Development Officer. “We are working with regulators to advance this program as expeditiously as possible and expect to initiate a pivotal Phase 3 study later this year.”
About the DAZALS Study
DAZALS is a randomized, double-blind, placebo-controlled Phase 2 trial in which 249 patients with ALS were randomized 1:1:1 to receive either 150 mg of dazucorilant, 300 mg of dazucorilant or placebo daily for 24 weeks. Patients who completed the treatment period were eligible to enroll in the study’s long-term extension phase, in which all patients received 300 mg of dazucorilant. Baseline patient characteristics, including the ENCALS risk score, time from diagnosis, ALSFRS-R total score, and bulbar onset, were consistent across study arms.
The DAZALS primary endpoint was the difference in change from baseline during the study’s 24-week treatment period in ALSFRS-R score between patients who received dazucorilant and those who received placebo. Key secondary endpoints include overall survival and quality of life. DAZALS was conducted at sites in Europe, the United States and Canada.
About Amyotrophic Lateral Sclerosis
ALS, also known as Lou Gehrig’s disease or motor neuron disease, is a fatal degenerative neurologic disorder that affects more than 55,000 people in the United States and Europe. ALS causes muscles to weaken and, as the disease progresses, severely impairs patients’ ability to speak, eat, move and breathe. There is increasing evidence that patients with ALS, particularly those with rapid disease progression, exhibit elevated or abnormal cortisol levels. A patient’s life expectancy after diagnosis is two to five years.
About Dazucorilant
Dazucorilant is a selective cortisol modulator that binds to the glucocorticoid receptor but not to the body’s other hormone receptors. Corcept is studying dazucorilant as a potential treatment for ALS and other neurologic disorders. The compound is proprietary to Corcept and is protected by composition of matter, method of use and other patents. The U.S. Food and Drug Administration has granted dazucorilant Fast Track Designation and orphan drug status for the treatment of ALS in the United States.
About Corcept Therapeutics
For over 25 years, Corcept has focused on cortisol modulation and its potential to treat patients with a wide variety of serious disorders and has discovered more than 1,000 proprietary selective cortisol modulators and glucocorticoid receptor antagonists. Corcept is conducting advanced clinical trials in patients with Cushing’s syndrome, solid tumors, ALS and liver disease. In 2012, the company introduced Korlym®, the first medication approved by the U.S. Food and Drug Administration (FDA) for the treatment of patients with endogenous Cushing’s syndrome, and in 2026, the company introduced Lifyorli™, approved in combination with nab-paclitaxel, the first FDA-approved selective glucocorticoid receptor antagonist, for adults with platinum-resistant ovarian cancer. Corcept is headquartered in Redwood City, California. For more information, visit Corcept.com.
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