Crinetics Pharmaceuticals, Inc. announced on January 10, 2025, positive topline results from its open-label, Phase 2 TouCAHn study of investigational atumelnant in congenital adrenal hyperplasia (CAH). Atumelnant is a novel, once-daily oral adrenocorticotropic hormone (ACTH) receptor antagonist.
The study demonstrated rapid, substantial, and sustained statistically significant reductions in key biomarkers, including up to an 80% mean reduction of androstenedione (A4) levels across all doses. Meaningful improvements were also observed in multiple clinical signs and symptoms of CAH affecting patient health.
Atumelnant was generally well tolerated, with no treatment-related severe or serious adverse events reported. These positive safety and efficacy data support the initiation of a global Phase 3 pivotal trial for adults in CAH, as well as a Phase 2b/3 trial in pediatric patients later in 2025.
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