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

Forward-Looking Statements
This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. All statements other than statements of historical facts contained in this press release are forward-looking statements, including statements regarding the plans and timelines for the Phase 2/3 program for atumelnant for CAH; the plans and timelines for the clinical development of our drug candidates, including the therapeutic potential and clinical benefits or safety profile thereof; the expected timing of additional research pipeline updates or the expected timing of the advancement of those programs. In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplates,” “believes,” “estimates,” “predicts,” “potential,” “upcoming” or “continue” or the negative of these terms or other similar expressions. These forward-looking statements speak only as of the date of this press release and are subject to a number of risks, uncertainties and assumptions, including, without limitation, data that we report may change following completion or a more comprehensive review of the data related to the clinical studies; we may not be able to obtain, maintain and enforce our patents and other intellectual property rights, and it may be prohibitively difficult or costly to protect such rights; geopolitical events may disrupt Crinetics’ business and that of the third parties on which it depends, including delaying or otherwise disrupting its clinical studies and preclinical studies, manufacturing and supply chain, or impairing employee productivity; unexpected adverse side effects or inadequate efficacy of the Company’s product candidates that may limit their development, regulatory approval and/or commercialization; the Company’s dependence on third parties in connection with product manufacturing, research and preclinical and clinical testing; the success of Crinetics’ clinical studies and nonclinical studies; regulatory developments or political changes, including the policies related to pricing and pharmaceutical drug reimbursement in the United States and foreign countries; clinical studies and preclinical studies may not proceed at the time or in the manner expected, or at all; the timing and outcome of research, development and regulatory review is uncertain, and Crinetics’ drug candidates may not advance in development; Crinetics may use its capital resources sooner than expected or our cash burn rate may accelerate; any future impacts to our business resulting from geopolitical developments outside our control; and the other risks and uncertainties described in the Company’s periodic filings with the Securities and Exchange Commission (SEC). The events and circumstances reflected in the company’s forward-looking statements may not be achieved or occur and actual results could differ materially from those projected in the forward-looking statements. Additional information on risks facing Crinetics can be found under the heading “Risk Factors” in Crinetics’ periodic filings with the SEC, including its annual report on Form 10-K for the year ended December 31, 2024. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Except as required by applicable law, Crinetics does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

Media:
Natalie Badillo
Head of Corporate Communications
nbadillo@crinetics.com
(858) 345-6075

Investors:
Gayathri Diwakar
Head of Investor Relations
gdiwakar@crinetics.com
(858) 345-6340


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


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