Clinical Trial Will Test Fluasterone in Cushing’s Syndrome

SteroTherapeutics and the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) are teaming up to conduct a Phase 2 clinical trial to determine the safety, efficacy, and dosage regimen for fluasterone as a potential treatment for the metabolic manifestations of Cushing’s syndrome.

“This is an important partnership and opportunity to develop a well-tolerated drug that may target the multiple metabolic manifestations of Cushing syndrome including hypertriglyceridemia, insulin resistance, obesity, and fatty liver disease,” Manohar Katakam, PhD, founder and CEO of SteroTherapeutics, said in a press release.

Cushing’s syndrome is caused by elevated levels of the hormone cortisol. High cortisol levels (hypercortisolism) can result in changes to the body’s metabolism. For example, Cushing’s can cause nonalcoholic fatty liver disease, which can result in scarring and damage to the liver, or metabolic syndrome —  a cluster of conditions such as high blood pressure and body fat levels, which can increase the risk of stroke and heart attack.

Fluasterone is an analogue of dehydroepiandrosterone (DHEA), a hormone that the body uses to make other hormones, including testosterone and estrogen. As a potential Cushing’s treatment, fluasterone was granted orphan drug designation by the U.S. Food and Drug Administration in 2018.

SteroTherapeutics and the NICHD have signed a Cooperative Research and Development Agreement (CRADA) to conduct a Phase 2 clinical trial testing fluasterone in people with Cushing’s. Per the agreement, SteroTherapeutics will provide the study drug and placebo to enable the NICHD, which is a part of the National Institutes of Health, to conduct the clinical study.

The study’s overarching goal will be to gather data to support advancing fluasterone into a larger Phase 3 trial, which will provide data about the therapy’s effectiveness. If trial results are positive, the therapy is expected to be approved in 2023–24.

The clinical trial will be led by Constantine Stratakis, MD, chief of the Section on Endocrinology and Genetics at the NICHD. Stratakis and his research group have been studying Cushing’s syndrome for more than 25 years.

“Over three decades of research on Cushing syndrome, I have come across a number of patients with [Cushing’s syndrome] that are not eligible for the few existing medical therapies; our research seeks to determine whether fluasterone could provide a new therapeutic option for them,” Stratakis said.