Orphagen Pharmaceuticals Receives $2 Million NCI Grant for Novel ACC Therapy

Key Insights

• Orphagen Pharmaceuticals secured a $2 million SBIR Phase II grant from the National Cancer Institute to advance OR-449, a first-in-class drug for adrenocortical carcinoma (ACC).
• OR-449 is a selective inhibitor of steroidogenic factor-1 (SF-1), a key transcription factor crucial for adrenal gland development and implicated in ACC pathogenesis.
• The funding supports preclinical development of OR-449, representing a potential new treatment option for ACC, a rare cancer with limited therapeutic solutions and poor prognosis.

Orphagen Pharmaceuticals has been awarded a $2 million Small Business Innovation Research (SBIR) Phase II grant from the National Cancer Institute (NCI) to support the preclinical development of OR-449, a potential first-in-class therapy for adrenocortical carcinoma (ACC). ACC is a rare and aggressive cancer of the adrenal gland with limited treatment options and a poor prognosis, making the development of new therapies a critical unmet need.

Targeting Steroidogenic Factor-1 (SF-1) in ACC

OR-449 is designed as a potent and selective inhibitor of steroidogenic factor-1 (SF-1 or NR5A1), an orphan nuclear receptor and transcription factor essential for the growth and development of the adrenal gland. Research indicates that SF-1 plays a crucial role in the pathogenesis of ACC, making it a promising therapeutic target. Inhibiting SF-1 could potentially disrupt the growth and survival of ACC cells.

A Novel Approach to ACC Treatment

"Orphagen has a strong platform for nuclear receptor discovery and our technology has allowed us to identify promising antagonists to a previously unexplored nuclear receptor, known as steroidogenic factor-1, or SF-1," said Scott Thacher, CEO of Orphagen and Principal Investigator of this grant. He further added, "The discovery and characterization of OR-449, which led to this prestigious award, was the product of an immensely talented team, with expertise in compound screening, medicinal chemistry, the implementation of novel tumor models, and small molecule drug development."

The development of OR-449 represents a novel approach to treating ACC by targeting SF-1. This strategy could offer a new therapeutic avenue for patients with this rare and aggressive cancer, where current treatment options are limited.

Orphagen's Drug Discovery Platform

Orphagen Pharmaceuticals focuses on discovering drug candidates targeting the nuclear receptor family. Their approach involves identifying and characterizing small molecule ligands for potential drug targets, aiming to initiate preclinical and clinical development with commercial partners. Orphagen previously partnered its ROR-gamma antagonist program with JT Pharma. The current funding, along with other non-dilutive sources, enables Orphagen to advance additional first-in-class drug discovery programs.