SteroTherapeutics, LLC signs CRADA Agreement with NIH to Develop a Novel Treatment for Cushing Syndrome Patients

SPRING HOUSE, Pa., Dec. 02, 2020 (GLOBE NEWSWIRE) -- SteroTherapeutics, LLC, a biopharmaceutical company developing new treatments for patients with rare diseases announced today the signing of a Cooperative Research and Development Agreement (CRADA) with the Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), part of the National Institutes of Health (NIH), to complete a Phase 2 clinical trial to determine the safety, efficacy, and dosage regimen for a non-androgenic analogue of dehydroepiandrosterone (DHEA) in the treatment of the manifestations of Cushing syndrome (also referred to as Cushing’s syndrome) including metabolic syndrome and fatty liver disease which is found in up to 25% of adult Cushing syndrome patients.

Leading the NIH fluasterone Phase 2 study will be Dr. Constantine Stratakis, MD, DSci, Chief, Section on Endocrinology & Genetics at the NICHD. Dr. Stratakis and his research group have been working on Cushing syndrome for more than 25 years and have discovered several genetic defects predisposing to the disease and described animal models of Cushing syndrome that can be used to evaluate future treatments. The need for this new treatment option was best described by Dr. Stratakis, “Over three decades of research on Cushing syndrome, I have come across a number of patients with this condition 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.”

Under the terms of the CRADA, SteroTherapeutics will provide the study drug and placebo to enable the NICHD to conduct the clinical study. The study will be conducted under the rubric of SteroTherapeutics’ Investigational New Drug authorization with the goal of advancing fluasterone to a pivotal Phase 3 study and marketing approval by 2023-24. “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,” said Dr. Manohar Katakam, SteroTherapeutics’ Founder and CEO. These therapeutic outcomes were also delineated in fluasterone’s orphan designation indication, which includes hyperglycemia and nonalcoholic fatty liver disease, making fluasterone unique for both its indication as well as the mechanism of action.

About CRADAs (Cooperative Research and Development Agreements)

The National Institutes of Health (NIH) CRADA is a formal research and development agreement as well as an essential tool for establishing a relationship in which Federal laboratories can work with non-Federal parties. The intent of Congress in establishing the CRADAs was to promote national technological competitiveness and rapid transfer of the fruits of innovation to the marketplace. The CRADA is an agreement in which the NIH laboratories contribute personnel, services, facilities, equipment, and other resources toward the conduct of a specified clinical trial. The goal of the NIH CRADA programs is to move new technologies to market especially if these technologies meet unmet or underserved needs for the general public health and wellness.

About Cushing’s syndrome

Cushing syndrome is a disorder with physical and mental changes that result from having too much cortisol in the blood for a long period of time. Currently it is estimated that 20,000 patients have the syndrome in the United States, and approximately 3,000 new patients are diagnosed annually. Cortisol is a steroid hormone produced by the adrenal glands, located above the kidneys. In normal amounts, cortisol helps the body to respond to stress, maintain blood pressure and cardiovascular function, keep the immune system in check and convert fat, carbohydrates, and proteins into energy. A number of drugs have been advocated for the medical management of Cushing's syndrome but few have gained widespread acceptance. The most reliably effective agents are often plagued by side effects that results in the discountenance of that agent.

About SteroTherapeutics, LLC

SteroTherapeutics is a privately held pharmaceutical company focused on developing novel therapies for diseases with significant unmet needs. SteroTherapeutics is pursuing a novel drug therapy for major diseases related to metabolic syndrome, which afflicts 35% of the US adult population.

The lead molecule is a non-androgenic analogue of dehydroepiandrosterone (DHEA) and is a selective anti-glucocorticoid which suppresses many of glucocorticoid deleterious effects but does not antagonize its anti-inflammatory activity. This unique mechanism of action makes it a suitable molecule for managing in which excess exposure of glucocorticoids (e.g. Cushing syndrome) or chronic administration of exogenous steroids that is the standard of care in certain rare diseases like Duchenne Muscular Dystrophy (DMD).

Corporate Contact Info:

Bo Allen, PhD
Tel: 267-300-0010