Orphagen Pharmaceuticals, an early-stage biopharmaceutical company focused on developing and commercializing novel therapies for cancer and other serious diseases with significant unmet need, today announced that data from its preclinical program to develop OR-449 for the treatment of adrenocortical cancer will be shared at the Endocrine Society’s 2021 Annual Meeting (ENDO 2021) taking place virtually from Saturday, March 20 to Tuesday, March 23.

OR-449 is an antagonist to the orphan nuclear receptor steroidogenic factor-1 (SF-1) and the product of a dedicated medicinal chemistry and drug development campaign carried out at Orphagen.

Orphagen ENDO2021 poster

Adrenocortical cancer (ACC) is a rare cancer of the adrenal gland. According to clinical experts, the response rates for the most common treatments are <30% for patients with metastatic disease. The preclinical findings presented demonstrate that OR-449 can be dosed orally and can block tumor growth in a mouse xenograft derived from a pediatric ACC tumor.

Orphagen has set a goal of initiating first-in-human trials for OR-449 in 2022.

A Novel Steroidogenic Factor-1 Antagonist, OR-449, as a Targeted Therapy for Adrenocortical Cancer

Presenter: Paul D Crowe, PhD

Co-Authors: Ray Fox, PhD, Haiyan Tao, PhD, Emily Eastwood, PhD, Neil Raheja, PhD, Raul Ribeiro, MD, Scott Thacher, PhD.

Poster Session: P54, Endocrine Disrupting Compounds: Mechanisms of Action and Clinical Implications

About Orphagen

Orphagen unlocks the power of orphan nuclear receptors. Our scientists excel in discovery and development of small molecule ligands to these largely unexplored drug targets. Starting with a therapeutic area agnostic approach, we are exploring proprietary lead molecules with potential in autoimmune disease and oncology.

Orphagen successfully partnered its first program for ROR-gamma antagonists with a mid-size pharmaceutical company ahead of all competitors in the field. Funding from its partnerships and other non-dilutive sources, including federal grants, has allowed Orphagen to advance its proprietary first-in-class drug discovery programs, including OR-449 for adrenocortical cancer.