Crinetics Initiates Phase 2/3 Pediatric Trial Evaluating Atumelnant in Congenital Adrenal Hyperplasia (CAH)

Core Insights - Crinetics Pharmaceuticals has initiated the BALANCE-CAH Phase 2/3 trial for atumelnant, a novel ACTH receptor antagonist aimed at treating classic congenital adrenal hyperplasia (CAH) in children and adolescents [1][2] Group 1: Study Overview - The BALANCE-CAH trial is designed to evaluate the safety, efficacy, and pharmacokinetics of atumelnant in pediatric patients with CAH, addressing a significant unmet medical need [2][3] - The study consists of three parts: Part A is a Phase 2 open-label dose-ranging study, Part B is a Phase 3 double-blind randomized placebo-controlled study, and Part C is an open-label extension [3] Group 2: Atumelnant Details - Atumelnant is the first and only small molecule ACTH receptor antagonist in late-stage clinical development, specifically targeting the melanocortin type 2 receptor (MC2R) to block excess androgen production [2][4] - Preclinical studies have shown that atumelnant has a strong binding affinity for MC2R and has demonstrated significant reductions in key CAH biomarkers, indicating its potential to transform care for individuals with CAH [4] Group 3: Company Background - Crinetics Pharmaceuticals focuses on developing novel therapies for endocrine diseases and related tumors, with expertise in targeting G-protein coupled receptors (GPCRs) [5] - The company’s lead product, PALSONIFY™ (paltusotine), is the first once-daily oral treatment approved for adults with acromegaly, and it has a robust pipeline of over 10 programs, including atumelnant [6]