OMass Therapeutics, a biotechnology company based in Oxford, United Kingdom, announced it will present the first public preclinical data for its lead development candidate OMS1620 at the Annual Endocrine Society Meeting (ENDO 2025) in San Francisco from July 12-15, 2025. The presentation marks a significant milestone for the company's melanocortin-2 (MC2) receptor program targeting congenital adrenal hyperplasia (CAH).
Novel Approach to CAH Treatment
OMS1620 represents a potential best-in-class MC2 receptor antagonist designed specifically for diseases associated with ACTH excess, including CAH. The MC2 receptor is a G-protein coupled receptor (GPCR) for adrenocorticotropic hormone (ACTH), which is released by the pituitary gland and triggers cortisol and androgen production.
In classical CAH, patients are unable to produce cortisol, leading to chronic overproduction of ACTH which drives excess androgen production. Due to the lack of cortisol, CAH patients must receive glucocorticoid supplementation to survive. However, achieving proper ACTH regulation in CAH patients typically requires supraphysiological doses of glucocorticoids, resulting in patients either suffering from effects of hyperandrogenism, over-dosing of glucocorticoids, or both.
Optimized Binding Kinetics for Enhanced Efficacy
OMS1620 has been specifically designed to maximize receptor residency time, making it highly resistant to competition from rising endogenous ACTH that occurs as glucocorticoid doses are reduced. This unique characteristic can support patients in achieving the ultimate treatment goal in CAH of androgen normalization while on physiological dose replacement of glucocorticoids.
The development candidate is currently in IND enabling studies, representing a critical step toward clinical development. According to the company, their preclinical modeling demonstrates that OMS1620 has the potential to be a best-in-class product with a differentiated profile that can increase the number of CAH patients reaching their ultimate treatment goal.
Conference Presentation Details
The poster presentation, titled "Optimizing Binding Kinetics to Develop Insurmountable MC2 Receptor Antagonists for the Treatment of Congenital Adrenal Hyperplasia," will be presented by Mark Soave on July 13, 2025, from 12:00-1:30 PM PDT during Session P55 - ADRENAL (EXCLUDING MINERALOCORTICOIDS): Adrenal Insufficiency and CAH II at the Moscone Convention Centre in San Francisco.
Ros Deegan, Chief Executive Officer of OMass, commented: "We are excited to share the data we have generated for OMS1620 at ENDO 2025 as we continue our progress towards the clinic. Our preclinical modelling demonstrates that OMS1620 has the potential to be a best-in-class product with a differentiated profile, that can further increase the number of CAH patients reaching their ultimate treatment goal."
Company Background and Platform
OMass Therapeutics utilizes its proprietary OdyssION™ drug discovery platform, which comprises next-generation native mass spectrometry with novel biochemistry techniques and custom chemistry. This platform interrogates not just a drug target, but also the interaction of the target with its native ecosystem, separate from the confounding complexity of the cell, resulting in what the company describes as cell-system fidelity with cell-free precision.
The company is advancing a pipeline of small molecule therapeutics in rare diseases and immunological conditions, with the MC2 receptor program representing its lead development effort. OMass has raised over $160 million (£129 million) from a top-tier international investor syndicate including Syncona, Oxford Science Enterprises, GV, Northpond Ventures, Sanofi Ventures and British Patient Capital.
