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 positive results from the open-label portion of the pivotal Phase 3 GRACE trial of its proprietary selective cortisol modulator relacorilant in patients with all etiologies of endogenous Cushing's syndrome (hypercortisolism).

GRACE has two parts. In the "open-label" phase, 152 patients with Cushing's syndrome and either hypertension, hyperglycemia or both received relacorilant for 22 weeks. Patients who exhibited pre-specified improvements in either or both symptoms were given the opportunity to enter the trial's randomized, double-blind withdrawal phase, in which half of the patients continued to receive relacorilant and half received placebo for 12 weeks.

GRACE's primary endpoint is maintenance of blood pressure control in the "randomized withdrawal" phase, with maintenance of glycemic control as the key secondary endpoint. Other key secondary and exploratory endpoints in the randomized withdrawal phase include changes in weight, waist circumference, cognitive impairment and Cushing's Quality of Life score. The data provided below are from GRACE's open-label phase.

Open-Label Results
Patients in the open-label phase exhibited clinically meaningful and statistically significant improvements in hypertension, hyperglycemia and other key secondary and exploratory endpoints. The drug was well-tolerated, consistent with relacorilant's known safety profile. Due to relacorilant's unique mechanism of action, the observed efficacy was seen without increases in cortisol concentrations and relacorilant-induced hypokalemia. In addition, no cases of relacorilant-induced endometrial hypertrophy with or without vaginal bleeding were seen, nor were there any instances of adrenal insufficiency or QT prolongation (independently confirmed).

"These open-label results are compelling, and they provide important information about the treatment of hypercortisolism," said Richard Auchus, MD, PhD, Professor of Internal Medicine, Division of Metabolism, Endocrinology & Diabetes at the University of Michigan and Chief of the Endocrinology & Metabolism Section at the Ann Arbor VA Medical Center. "Patients showed marked improvement across a broad range of signs and symptoms, without significant safety burden. Due to relacorilant's unique mechanism of action, we are not observing other toxicities seen with current therapies, which positions relacorilant to potentially become a new standard of care for patients with this disease."

"These data improve on the results we observed in our Phase 2 study," said Bill Guyer, PharmD, Corcept's Chief Development Officer. "Cushing's syndrome has many signs and symptoms, which is why it is so important that patients in GRACE's open-label phase exhibited improvements across a broad range of clinically meaningful endpoints. We expect to build on these results in the trial's randomized withdrawal phase. We plan to present data from the open-label and randomized withdrawal phases at a medical conference in June and remain on track to submit our NDA this quarter."