Corcept Reports Phase 2 Dazucorilant Study Results in ALS Patients

Corcept Therapeutics Incorporated, based in Redwood City, California, has recently unveiled the outcomes of its DAZALS study. This Phase 2 trial was organized to evaluate the efficacy of dazucorilant, a selective cortisol modulator, in treating individuals with Amyotrophic Lateral Sclerosis (ALS). The study was structured as a randomized, double-blind, placebo-controlled examination, assessing two dosages, 150 mg and 300 mg, of dazucorilant.

A significant element of the DAZALS trial was its primary endpoint, aimed at assessing the change in the ALS Functional Rating Scale-Revised (ALSFRS-R) score from the baseline among patients treated with dazucorilant compared to those on placebo. Unfortunately, the results revealed that dazucorilant did not achieve this primary objective. Additionally, patients administered dazucorilant reported more gastrointestinal issues at the beginning of the treatment than those on placebo.

Despite the primary endpoint not being met, notable findings emerged during the 24-week study concerning patient mortality. No fatalities were recorded in the cohort receiving 300 mg of dazucorilant, whereas five deaths were reported in the placebo group, a statistically significant result with a p-value of 0.02. Following the trial, participants were invited to continue in an open-label, long-term extension study, which provides them with 300 mg of dazucorilant. This extension aims to monitor overall survival, with results expected in March 2025 after all participants have been treated for a year. Furthermore, dazucorilant has been conferred Fast Track Designation by the U.S. Food and Drug Administration (FDA).

The DAZALS study engaged 249 participants, who were evenly distributed into three groups to receive either 150 mg of dazucorilant, 300 mg of dazucorilant, or a placebo daily over a span of 24 weeks. The primary focus was on mitigating the deterioration of motor skills and other functional characteristics, assessed via the ALSFRS-R. Secondary objectives included evaluating overall survival and quality of life metrics. The long-term extension segment allows patients to receive 300 mg of dazucorilant for up to 132 weeks. The study is conducted across various locations in Europe, the United States, and Canada.

Amyotrophic Lateral Sclerosis, also referred to as Lou Gehrig’s disease, is a progressive and fatal neurological condition affecting over 55,000 individuals in the United States and Europe. ALS primarily leads to muscle weakening and severely diminishes the patient’s ability to move, speak, eat, and breathe as the disease advances. There is growing evidence suggesting that ALS patients, especially those with rapid disease progression, may exhibit abnormal or increased levels of cortisol. Life expectancy post-diagnosis typically ranges from two to five years.

Dazucorilant, the investigational drug in question, acts as a selective cortisol modulator by binding specifically to the glucocorticoid receptor. Unlike other substances, it does not interact with the body's other hormone receptors. Corcept Therapeutics is exploring its potential application in treating ALS and various other neurological disorders. Dazucorilant is exclusive to Corcept and is protected under composition of matter, method of use, and other patents.

For over 25 years, Corcept Therapeutics has concentrated on modulating cortisol's effects to develop treatments for numerous severe ailments. With a portfolio of over 1,000 proprietary selective cortisol modulators, the company is actively conducting advanced clinical trials targeting hypercortisolism, solid tumors, ALS, and liver diseases. In 2012, Corcept introduced Korlym, the first medication sanctioned by the FDA for treating endogenous hypercortisolism. The company's headquarters is in Redwood City, California.