Clinical TrialsTildacerfont is an oral, non-steroid, highly selective corticotropin-releasing factor 1 (CRF1) receptor antagonist in development for congenital adrenal hyperplasia (CAH).
Competitive AdvantageRemoving BBIO from the CAH race is an incremental positive for SPRB, which could be poised to have the second novel CAH drug to market.
Market OpportunityThe CAH opportunity has a large enough rare disease market with ~20K-30K patients in the U.S. and ~250 new patients identified via newborn screening annually.