Corcept Announces Results From Phase 2 Study of Dazucorilant in Patients With Amyotrophic Lateral Sclerosis (ALS)

Rhea-AI Summary

Corcept Therapeutics (NASDAQ: CORT) has announced results from its DAZALS Phase 2 trial evaluating dazucorilant in ALS patients. The study tested two doses (150mg and 300mg) of the selective cortisol modulator but did not meet its primary endpoint measuring change in ALS Functional Rating Scale-Revised (ALSFRS-R). While patients experienced increased gastrointestinal upset with dazucorilant, notably, no deaths occurred in the 300mg treatment group (0/83) compared to 5 deaths in the placebo group (5/82), showing statistical significance (p=0.02). The drug has received Fast Track Designation from the FDA. An open-label extension study continues, with overall survival assessment planned for March 2025.

Positive

• Zero deaths in 300mg treatment group vs 5 deaths in placebo group (p=0.02)
• FDA Fast Track Designation granted for dazucorilant
• Open-label extension study continuing with survival assessment in March 2025

Negative

• Failed to meet primary endpoint (ALSFRS-R change from baseline)
• Increased gastrointestinal side effects in treatment group

Insights

Medical Research Analyst

The Phase 2 DAZALS study results for dazucorilant in ALS present a mixed picture. While the trial missed its primary endpoint of improving functional scores (ALSFRS-R), the survival data shows a potentially significant benefit, with zero deaths in the 300mg treatment group compared to 5 deaths in placebo (p=0.02). This survival signal is particularly noteworthy in ALS, a rapidly progressive and fatal disease. However, the increased gastrointestinal side effects in the treatment group warrant careful consideration. The continuation of the open-label extension study through March 2025 will be important in validating the survival benefit. The FDA Fast Track designation remains valuable for potential accelerated development, but the missed primary endpoint may complicate the regulatory pathway forward.

Financial Analyst

The mixed trial results create uncertainty for CORT's ALS program. While missing the primary endpoint typically spells trouble for drug development, the statistically significant survival benefit (p=0.02) could provide a viable path forward, particularly given the FDA Fast Track designation. However, investors should note that additional studies may be required, potentially extending the development timeline and increasing costs. The gastrointestinal side effects could impact patient compliance and commercial potential if approved. The stock may experience volatility as the market weighs the survival benefit against the missed functional endpoint. The March 2025 survival data from the extension study will be a key catalyst for reassessing the program's potential.

REDWOOD CITY, Calif.--(BUSINESS WIRE)-- 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 results from the DAZALS study, a randomized, double-blind, placebo-controlled, Phase 2 trial evaluating two doses (150 mg and 300 mg) of its proprietary selective cortisol modulator dazucorilant in patients with ALS. Upon completion of the trial, patients were eligible to enter an open-label, long-term extension study, in which they received 300 mg of dazucorilant.

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 experienced substantially more gastrointestinal upset at the onset of treatment than those who received placebo. During the 24-week study, no deaths (0/83) were observed in the 300 mg arm, compared to 5 deaths (5/82) in the placebo group (p-value: 0.02). The open-label, long-term extension study will continue and overall survival will be assessed in March 2025 after all patients have had one year pass since the onset of treatment. Dazucorilant has been granted Fast Track Designation by the U.S. Food and Drug Administration.

Complete results from the DAZALS study will be presented at a medical conference next year.

About the DAZALS Study

DAZALS enrolled 249 patients, randomized 1:1:1 to receive either 150 mg of dazucorilant, 300 mg of dazucorilant or placebo daily for 24 weeks. The study’s primary endpoint was to slow the decline in motor skills and other functional criteria, compared to placebo, as measured by the ALSFRS-R. Key secondary endpoints include overall survival and quality of life. Patients were provided the opportunity to receive 300mg of dazucorilant for 132 weeks in the open-label, long-term extension of the DAZALS study. DAZALS is being conducted at sites in Europe, the United States and Canada.

About Amyotrophic Lateral Sclerosis (ALS)

ALS, also known as Lou Gehrig’s 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 does not bind to the body’s other hormone receptors. Corcept is studying it as a potential treatment for ALS and other neurologic disorders. Dazucorilant is proprietary to Corcept and is protected by composition of matter, method of use and other patents.

About Corcept Therapeutics

For over 25 years, Corcept’s focus on cortisol modulation and its potential to treat patients with a wide variety of serious disorders has led to the discovery of more than 1,000 proprietary selective cortisol modulators. Corcept is conducting advanced clinical trials in patients with hypercortisolism, solid tumors, ALS and liver disease. In February 2012, the company introduced Korlym^®, the first medication approved by the U.S. Food and Drug Administration for the treatment of patients with endogenous hypercortisolism. Corcept is headquartered in Redwood City, California. For more information, visit Corcept.com.

Forward-Looking Statements

Statements in this press release, other than statements of historical fact, are forward-looking statements based on our current plans and expectations, which are subject to risks and uncertainties that might cause our actual results to differ materially from those such statements express or imply. These risks and uncertainties are set forth in our SEC filings, which are available at our website and the SEC’s website.

In this press release, forward-looking statements include those concerning the development of dazucorilant as a treatment for patients with ALS, including the pace, conduct, timing and outcome of DAZALS and its associated long-term extension study, as well as oversight or requirements that may be imposed by the FDA or other regulatory authorities. We disclaim any intention or duty to update forward-looking statements made in this press release.

View source version on businesswire.com: https://www.businesswire.com/news/home/20241211463748/en/

Investor inquiries:

ir@corcept.com

Media inquiries:

communications@corcept.com

www.corcept.com

Source: Corcept Therapeutics Incorporated

FAQ

What were the main results of Corcept's DAZALS Phase 2 trial for ALS (CORT)?

The trial did not meet its primary endpoint measuring ALSFRS-R change, but showed zero deaths in the 300mg treatment group compared to 5 deaths in the placebo group, with increased gastrointestinal side effects in the treatment group.

What doses of dazucorilant were tested in Corcept's ALS trial (CORT)?

The DAZALS study tested two doses of dazucorilant: 150mg and 300mg.

When will Corcept (CORT) assess overall survival data for the dazucorilant ALS study?

Overall survival data from the open-label extension study will be assessed in March 2025, after all patients have completed one year of treatment.

What regulatory status has dazucorilant received from the FDA for ALS treatment (CORT)?

Dazucorilant has been granted Fast Track Designation by the U.S. Food and Drug Administration.

What were the mortality rates in Corcept's DAZALS trial for ALS (CORT)?

In the 24-week study, there were 0 deaths out of 83 patients in the 300mg treatment group, compared to 5 deaths out of 82 patients in the placebo group (p=0.02).