Orphagen Pharmaceuticals, Inc.

Biotechnology ResearchCalifornia, United States2-10 Employees

Orphagen Pharmaceuticals discovers and develops first-in-class small-molecule therapies targeting orphan nuclear receptors—novel pathways with breakthrough potential in immunology, pediatric rare diseases, and cancer.

Orphagen's lead oncology program, OR-449, is a first-in-class breakthrough small molecule for the treatment of a rare and aggressive cancer of the adrenal gland, adrenocortical cancer (ACC), that lacks an effective standard of care. OR-449 is a potent and highly selective antagonist to the orphan nuclear receptor, steroidogenic factor-1 (SF-1 or NR5A1), a cetral transcription factor in the growth and development of ACC. OR-449 is close to IND filing and could receive FDA approval within 5 years for what we estimate is a $1 B global market for ACC. In addition, SF-1 antagonists can be developed as a precision therapy for SF-1(HIGH) head and neck and lung squamous cancers, increasing the treatable patient population for OR-449 several fold.

CPRIT, the Texas State Cancer Agency, will contribute a $10.2 M Product Development Award for a Phase 1 trial of OR-449 in adult ACC once appropriate seed funds have been raised.

Orphagen’s pipeline also includes a novel candidate for treatment of inflammatory bowel disease (OR-812). Development and translational studies are currently supported by an NIH SBIR grant. OR-812 is an antagonist of retinoic acid receptor alpha (RARA). Blocking RAR-alpha represents a highly differentiated approach to suppression of gut inflammation and is supported by extensive preclinical findings.

Based in San Diego, Orphagen is pursuing novel biology to create clinical-stage innovation. For partnership or investor inquiries, contact: info@orphagen.com.

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