Crinetics Pharmaceuticals announced that it has dosed the first patient in its CALM‑CAH Phase 3 trial of atumelnant, a once‑daily oral ACTH receptor antagonist designed to treat classic congenital adrenal hyperplasia (CAH). The trial enrolls adults with classic CAH and will evaluate whether atumelnant can normalize adrenal androgen levels while allowing glucocorticoid doses to be reduced to physiologic ranges.
Atumelnant is the first small‑molecule ACTH receptor antagonist in late‑stage development for CAH, a rare disease that currently requires lifelong glucocorticoid therapy at supraphysiologic doses. By blocking the melanocortin type 2 receptor on the adrenal gland, atumelnant aims to reduce excess androgen production and potentially spare patients from the long‑term side effects of high‑dose steroids.
The trial represents a significant expansion of Crinetics’ pipeline beyond its recently approved product, PALSONIFY™ (paltusotine), which received FDA approval for acromegaly on September 25, 2025. The company’s cash position—$1.1 billion in cash, cash equivalents, and investments as of September 30, 2025—provides a robust runway to fund the Phase 3 program through 2029 and supports continued investment in its endocrine portfolio.
Dr. Dana Pizzuti, Chief Medical and Development Officer, said the CALM‑CAH study “will test a novel therapeutic approach that could transform the standard of care for patients with classic CAH, reducing glucocorticoid exposure and improving quality of life.” She added that the study’s innovative endpoints are designed to set a new standard for assessing disease control in this rare population.
The Phase 3 trial is a pivotal step toward regulatory approval. Crinetics already holds Orphan Drug Designation from the FDA for atumelnant in classic CAH, which provides incentives such as market exclusivity and potential tax credits. Successful completion of the trial would position the company to pursue a regulatory filing and expand its commercial footprint in the endocrine space.
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