Crinetics receives Orphan Drug designation for atumelnant in CAH

Crinetics (CRNX) Pharmaceuticals announced that the U.S. Food and Drug Administration granted Orphan Drug Designation for atumelnant, a novel, once-daily oral adrenocorticotropic hormone receptor antagonist candidate for the proposed treatment of classic congenital adrenal hyperplasia. Atumelnant is the first and only small molecule ACTH receptor antagonist in clinical development. In January 2025, Crinetics reported topline results from the Phase 2 TouCAHn trial of atumelnant in adults with classic CAH. The study demonstrated substantial, rapid and sustained reductions of key biomarkers across doses, including up to an 80% mean reduction in androstenedione. The study also demonstrated meaningful improvements in multiple clinical signs and symptoms of the disease affecting patient health, including resumption of menses and reduction of adrenal size. Crinetics expects the first participants randomized in the CALM-CAH Phase 3 study in adults and the BALANCE-CAH Phase 2/3 study in pediatrics in the second half of 2025.