Atumelnant's promising CAH trial
Crinetics presents positive Phase 2 trial results for atumelnant in congenital adrenal hyperplasia at ENDO 2026
Crinetics Pharmaceuticals presented Phase 2 trial results for atumelnant — a once-daily pill that directly blocks the hormone driving Congenital Adrenal Hyperplasia (CAH) — at the Endocrine Society's annual ENDO 2026 meeting in Chicago on June 14. The drug cut androgen levels by a mean of 67% and allowed 88% of patients to reduce their steroid doses to healthy levels, according to GlobeNewswire.
CAH affects roughly 20,000–30,000 Americans. For over 70 years, the only treatment has been high-dose steroids — which cause weight gain, bone loss, and diabetes. Atumelnant targets the root cause differently, and Crinetics is now moving the drug into Phase 3 trials.
Phase 2 Data Shows Rapid Androgen Cuts and Steroid Reduction
The Phase 2 "TouCAHn" trial tested an 80mg once-daily morning dose in adults with classic CAH. By week 12, morning androstenedione (A4) — a key androgen that signals disease activity — fell by a mean of 67%, according to Barchart. Two other androgens also dropped sharply: 11-OHA4 fell 64% and 11-ketotestosterone fell 56%.
The steroid-sparing result was equally striking. Seven out of eight patients — 88% — successfully cut their glucocorticoid dose down to a normal, healthy replacement level by week 12, GlobeNewswire reported. Trial investigator Dr. Umasuthan Srirangalingam of University College London Hospitals called it "exciting" that steroid dose cuts "did not impact the atumelnant-induced decline in androstenedione."
How Atumelnant Works — and Why It Differs From the Only Approved Rival
In CAH, a genetic defect stops the adrenal glands from making cortisol. The pituitary gland responds by flooding the body with ACTH — a hormone that tells the adrenals to work harder. That flood drives the adrenals to overproduce androgens instead. Atumelnant blocks the ACTH receptor directly on the adrenal gland, cutting off that signal at its destination.
The only approved non-steroid CAH drug is Crenessity (crinecerfont), made by Neurocrine Biosciences and approved by the FDA in December 2024. It works higher up the chain — at the pituitary — blocking the signal before ACTH is even released. Crinetics argues its approach is more direct and may allow deeper steroid tapering than crinecerfont has shown in trials.
Promising Early Cushing's Data Adds a Second Target for the Drug
Crinetics also shared early data from a separate Phase 1b/2a trial in ACTH-dependent Cushing's syndrome — a condition where a tumor drives excess ACTH and cortisol production. A 40mg dose normalized urinary free cortisol in 50% of patients — three out of six — within just 10 days, according to Markets Financial Content.
No serious drug-related side effects were reported in either trial. Mild-to-moderate symptoms linked to adrenal adjustment during steroid tapering were the most common issues. Crinetics Chief Endocrinologist Dr. Alan Krasner said the data "suggest it could represent a uniquely effective and simple oral therapy" for patients who need new options.
Phase 3 Trials Underway as Wall Street Eyes Blockbuster Potential
Crinetics is now enrolling adults in the CALM-CAH Phase 3 trial and children in the BALANCE-CAH Phase 2/3 trial — the first patient in BALANCE-CAH was dosed in January 2026. The FDA granted atumelnant Orphan Drug Designation in August 2025. A successful Phase 3 could support a New Drug Application filing as early as late 2027 or 2028, according to UK Market Screener.
Wall Street is watching closely. Analysts at Citizens and Evercore ISI both rate Crinetics stock "Outperform," with a consensus price target of $86 per share and high estimates reaching $105. The Phase 2 results are open-label with only eight patients in the key cohort, and analysts note that larger placebo-controlled Phase 3 data will be needed to confirm the drug's full profile.
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