First in class study of an investigational ACTH receptor antagonist aims to address significant unmet need in children and adolescents with classic CAH
SAN DIEGO, Jan. 22, 2026 (GLOBE NEWSWIRE) -- Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) today announced that the first patient has been dosed in the BALANCE-CAH Phase 2/3 trial evaluating investigational candidate atumelnant, a novel, once-daily oral adrenocorticotropic hormone (ACTH) receptor antagonist candidate for the proposed treatment of classic congenital adrenal hyperplasia (CAH) in children and adolescents.
“BALANCE-CAH is designed to evaluate atumelnant for the treatment of CAH in pediatric age patients – for whom the lifelong burden of this disease begins early,” said Dr. Alan Krasner, M.D., Chief Endocrinologist, Crinetics. “For children living with CAH, correcting elevated androgen levels caused by the condition, while preserving the ability to use low glucocorticoid doses for replacement purposes only, is critical in supporting normal growth, development, and metabolic health. By studying atumelnant in this population, we aim to generate insights that could help children live less encumbered by the challenges of this serious endocrine disorder.”
Atumelnant is the first and only small molecule ACTH receptor antagonist in late-stage clinical development and is designed to block the pathway in the adrenal gland that leads to the production of excess androgens associated with classic CAH. The BALANCE-CAH Phase 2/3 study is a potential registrational study of atumelnant in pediatric patients with CAH and will evaluate safety, efficacy, and pharmacokinetics in children and adolescents, a population with significant unmet medical need.
The study will be conducted in three phases. Part A is a Phase 2, open-label, semi-sequential dose-ranging portion of the study. Part B is the Phase 3, double-blind, randomized, placebo controlled confirmatory portion of the study. Part C is an open-label extension portion of the study.
Crinetics recently received Orphan Drug Designation from the U.S. Food & Drug Administration for atumelnant in the treatment of classic CAH.
For more information, visit https://clinicaltrials.gov/study/NCT07159841
About Atumelnant
Investigational atumelnant is the first in class and only once-daily, oral adrenocorticotropic hormone (ACTH) receptor antagonist that acts selectively at the melanocortin type 2 receptor (MC2R) on the adrenal gland in late-stage clinical development. Diseases associated with excess ACTH can have a significant impact on physical and mental health. Novel atumelnant has exhibited strong binding affinity for MC2R in preclinical models and has demonstrated suppression of adrenally derived glucocorticoids and androgens that are under the control of ACTH. Data from a 12-week Phase 2 study consistently demonstrated compelling treatment benefits of atumelnant, evidenced by the rapid, substantial and sustained statistically significant reductions in key CAH disease related biomarkers, including A4 and 17-hydroxyprogesterone, in a diverse population. Currently in Phase 3 clinical development, atumelnant holds the potential to offer transformational care for individuals living with congenital adrenal hyperplasia and ACTH-dependent Cushing’s syndrome. This breakthrough could revolutionize the management of these conditions, providing hope for unprecedented improvements in quality of life.
About Crinetics Pharmaceuticals
Crinetics Pharmaceuticals is a global pharmaceutical company committed to transforming the treatment of endocrine diseases and endocrine-related tumors through science rooted in patient needs. Crinetics is focused on discovering, developing, and commercializing novel therapies, with a core expertise in targeting G-protein coupled receptors (GPCRs) with small molecules that have specifically tailored pharmacology and properties.
Crinetics’ lead product, PALSONIFY™ (paltusotine), is the first once-daily, oral treatment approved by the U.S. FDA for the treatment of adults with acromegaly who had an inadequate response to surgery and/or for whom surgery is not an option. Paltusotine is also in clinical development for carcinoid syndrome associated with neuroendocrine tumors. Crinetics’ deep pipeline of 10+ disclosed programs includes late-stage investigational candidate atumelnant, which is currently in development for congenital adrenal hyperplasia and ACTH-dependent Cushing’s syndrome, and CRN09682, a nonpeptide drug conjugate candidate that is being developed to treat SST2 expressing neuroendocrine tumors and other SST2 expressing solid tumors. Additional discovery programs address a variety of endocrine conditions such as neuroendocrine tumors, Graves’ disease (including Graves’ hyperthyroidism and Graves’ orbitopathy, or thyroid eye disease), polycystic kidney disease, hyperparathyroidism, diabetes, obesity, and GPCR-targeted oncology indications.
