The Cancer Prevention & Research Institute of Texas (CPRIT) announced today that it plans to award Orphagen a $10.2 M product development grant over three years to partially fund the final steps towards IND filing and a Phase 1 clinical trial of OR-449, a novel small molecule and orally-bioavailable therapeutic candidate for an intractable cancer, adrenocortical cancer (ACC). OR-449 may be developed subsequently in head & neck and lung squamous cancers as a precision therapy for the 3-10% of these tumors where its target is highly expressed. Details of today’s announcement are here: CPRIT.grants.announcement.Nov.20.2024.
A major site for the Phase 1 trial would be the MD Anderson Cancer Center in Houston, internationally recognized for its expertise in treatment of ACC. To initiate and fully comply with the CPRIT grant, Orphagen will raise additional matching funds and move its headquarters to Texas in 2025.
Scott Thacher, Orphagen’s CEO and founder, stated, “This is an important step forward for our programs. OR-449 is the first of our internal programs that is on the path to a Phase 1 trial.”
“Further OR-449 represents a significant opportunity for improved therapy of ACC patients, whose options for medical therapy are limited. The target of OR-449 is the orphan nuclear receptor SF-1, now recognized as a potential driver of ACC.”
Orphagen Pharmaceuticals is a preclinical stage biopharmaceutical company focused on discovering and developing first-in-class therapeutics for cancer and inflammatory disease. Orphagen is a pioneer in the screening, discovery and development of ligands that modulate orphan or unexplored members of the nuclear receptor family. On a per target basis, the nuclear receptors are one of the most successful target classes known to the pharmaceutical industry.
Its first-in-class SF-1 antagonist (OR-449) has practically completed IND-enabling studies. Potential expansion indications for OR-449 include other cancers where SF-1 expression is highly elevated in such a way to suggest these depend on SF-1 activity for tumor growth.
CONTACTS
Company Contact: Scott Thacher, Ph.D., CEO, Orphagen, smt@orphagen.com