SAN DIEGO, Calif. — Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) today announced that the first patient has been dosed in the CALM-CAH Phase 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).
“Dosing the first patient in this Phase 3 study underscores our commitment to addressing the unmet needs of people living with CAH and our excitement about the potential of atumelnant,” said Dana Pizzuti, M.D., Chief Medical and Development Officer of Crinetics. “Through the CALM-CAH study, we will evaluate atumelnant’s ability to normalize adrenal androgen levels while simultaneously reducing glucocorticoid levels to the physiologically normal range. With a unique endpoint measuring both of these objectives, CALM-CAH sets a new standard in terms of assessing overall disease control.”
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. In a Phase 2 study in adults with classic CAH, treatment with atumelnant was associated with reductions in key biomarkers, including androstenedione and 17-hydroxyprogesterone, as well as other clinical measures of disease activity including adrenal size and resumption of menses. Based on Phase 2 results, Crinetics has advanced the registrational CALM-CAH Phase 3 trial in adults.
The CALM-CAH Phase 3 study is a randomized, placebo-controlled trial evaluating atumelnant in adults with classic CAH, designed to assess reductions in excess androgens, improvements in glucocorticoid use, and other clinical outcomes that reflect disease control.
Crinetics recently received Orphan Drug Designation from the U.S. Food & Drug Administration (FDA) 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. For more information visit https://crinetics.com/ or follow the company on X.
Media Contact
Natalie Badillo
Head of Corporate Communications
[email protected]
(858) 345-6075
Investors Contact
Gayathri Diwakar
Head of Investor Relations
[email protected]
(858) 345-6340