Sterotherapeutics LLC announced that the first patient has been successfully dosed in its ongoing Phase 2 clinical trial evaluating ST-002 for the treatment of Cushing's Syndrome. The milestone represents a critical advancement for patients with this rare, debilitating endocrine condition caused by chronic exposure to excess cortisol.
The multicenter European study is designed to assess the safety, efficacy, and tolerability of ST-002, following a successful Investigator Meeting held earlier this year in Athens, Greece, which marked the official trial activation.
Addressing Critical Unmet Medical Need
Cushing's Syndrome presents significant clinical challenges, leading to serious complications including diabetes, cardiovascular disease, osteoporosis and Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD). Currently, no universally effective treatment is available for this condition, highlighting the critical unmet medical need that ST-002 aims to address.
"We are pleased to announce the dosing of the first patient in our Phase 2 trial of ST-002," said Dr. Manohar Katakam, Ph.D., Chief Executive Officer of Sterotherapeutics. "This achievement is a testament to the dedication of our clinical teams and the commitment of our investigators. ST-002 has the potential to change the treatment landscape for patients who currently have limited therapeutic options."
Clinical Trial Design and Objectives
The Phase 2 trial will evaluate ST-002's impact on Cushing's Syndrome and its associated complications, including diabetes and MASLD. The study incorporates advanced assessment methods, with MASLD being evaluated through magnetic resonance imaging and targeted measurements.
Dr. Constantine Stratakis, MD, PhD, Executive Medical Director of Sterotherapeutics and Professor of Pediatrics, Endocrinology and Genetics, emphasized the study's comprehensive approach: "We remain focused on generating high-quality data that will inform the future development of ST-002 and provide hope for patients living with this challenging disorder and its associated complications, including diabetes and MASLD, the latter being assessed by magnetic resonance imaging and targeted measurements in our clinical study."
Regulatory Recognition and Future Expansion
ST-002 has previously received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA), reinforcing its potential value for patients with rare diseases and enabling regulatory incentives for development. The trial is being conducted across multiple sites, with additional sites expected to open in the coming months.
Sterotherapeutics, based in the USA, is a clinical-stage company dedicated to developing novel therapeutics for orphan diseases with significant unmet needs. The company's lead programs include ST-002 for Cushing's Syndrome and ST-003 for primary sclerosing cholangitis, both of which have received Orphan Drug Designation from the U.S. FDA and have demonstrated strong preclinical and early clinical results with favorable safety profiles and well-understood mechanisms of action.
