Positive Results for Mifepristone in Treating Hypercortisolism and Type 2 Diabetes: CATALYST Trial

Corcept Therapeutics Incorporated, a commercial-stage company, has announced promising results from the treatment phase of the Phase 4 CATALYST trial. The findings highlight the efficacy of mifepristone in addressing hypercortisolism (Cushing's syndrome) and difficult-to-control type 2 diabetes (T2D).

The randomized, double-blind, placebo-controlled CATALYST trial met its primary endpoint, demonstrating that patients with hypercortisolism and T2D experienced a statistically significant and clinically meaningful reduction in hemoglobin A1c (HbA1c) following treatment with mifepristone. Patients treated with Korlym showed a reduction of 1.47% in HbA1c levels compared to a 0.15% reduction in the placebo group, translating into a placebo-adjusted reduction of 1.32%.

"CATALYST’s first part showed that hypercortisolism is much more common than previously assumed. The results announced today show that Korlym is a safe and effective treatment option," Ralph DeFronzo, MD, chief of the Diabetes Division and professor of medicine at UT Health San Antonio and a CATALYST study investigator, said in a press release. DeFronzo emphasized that these findings are particularly significant given that participants were already receiving advanced diabetes therapies but continued to struggle with their disease.

The CATALYST study was designed as a two-part, Phase 4 investigation. The first part screened 1,057 patients with difficult-to-control type 2 diabetes—defined as having HbA1c levels greater than 7.5% despite optimal treatment with GLP-1 agonists and other therapies. Of these patients, 23.8% were identified as having hypercortisolism and eligible for the treatment phase. A total of 136 patients were randomized in a 2:1 ratio to receive either Korlym or a placebo for 24 weeks.

Bill Guyer, PharmD, Corcept's Chief Development Officer, highlighted the importance of these findings: "One in four patients with difficult-to-control type 2 diabetes have hypercortisolism, and Korlym was highly effective in controlling hyperglycemia in this patient population."

Importantly, the safety profile of Korlym observed in the CATALYST study was consistent with its existing labeling, with no new adverse events or side effects identified. Complete results from the trial are expected to be presented at a medical conference in the coming year.

Corcept’s findings underscore the potential for expanded screening for hypercortisolism and improved treatment strategies to enhance outcomes for patients struggling with these challenging conditions.

About Hypercortisolism (Cushing’s Syndrome)

Hypercortisolism, also known as Cushing’s syndrome, occurs due to excessive levels or activity of the hormone cortisol in the body. The symptoms can vary widely among individuals, but common signs include high blood pressure, central obesity, elevated blood sugar, and challenging-to-control type 2 diabetes. Many patients also report severe fatigue, muscle weakness, irritability, anxiety, depression, and cognitive impairments.

Hypercortisolism has the potential to impact multiple organ systems, making early diagnosis and treatment critical. If left untreated, it can lead to life-threatening complications.