Crinetics Pharmaceuticals

The purpose of this study is to evaluate the safety, efficacy, pharmacokinetics (PK), and pharmacodynamics (PD) of atumelnant treatment in pediatric participants with classic congenital adrenal hyperplasia (CAH).

The purpose of this study is to evaluate the efficacy, safety, PK, and PD of atumelnant in adults with classic CAH due to 21-OHD.

The purpose of this study is to evaluate the long-term safety, tolerability, and efficacy of atumelnant (CRN04894).

A Phase 3, randomized, double-blinded, placebo-controlled study to evaluate the efficacy and safety of paltusotine treatment vs placebo as well as the long-term safety of paltusotine in adults with carcinoid syndrome due to well-differentiated neuroendocrine tumors. The purpose of this study is to continue the evaluation of the safety, efficacy, and pharmacokinetics (PK) of paltusotine in participants with carcinoid syndrome.Show more

This Phase 1/2, multicenter, open-label, FIH study aims to evaluate the safety, tolerability, PK, and preliminary antitumor activity of CRN09682 in participants with SST2-expressing NENs and other solid tumors. The study includes a Dose Escalation Phase to determine the MTD and DLTs. Following MTD identification, additional participants will be enrolled at the expansion dose to further assess safety, tolerability, PK, and antitumor activity.Show more

Crinetics Pharmaceuticals is a San Diego biotech with one clear focus. They build oral pills for rare hormone diseases that, for decades, have been treated mostly with injections or imprecise hormone replacement. The company was started in 2008 by four scientists who left Neurocrine Biosciences (Scott Struthers, Stephen Betz, Frank Zhu, and Ana Kusnetzow) and went public on the NASDAQ in 2018 under the ticker CRNX. On July 6, 2026, Crinetics agreed to be acquired by Vertex Pharmaceuticals for $85.00 per share in cash (approximately $10 billion equity value). The transaction closed on September 1, 2026, and Crinetics is now part of Vertex. Its medicines and pipeline programs described below continue under Vertex.

Their first FDA approval came in September 2025, when Palsonify (the brand name for paltusotine) was approved for acromegaly. Acromegaly is a rare disorder where the body makes too much growth hormone, usually because of a small tumor on the pituitary gland. For most adults who needed medicine for acromegaly, the standard had been monthly injections at a doctor's office for years on end. Palsonify is the first oral once-daily pill that works the same way the injections do, and in the Phase 3 PATHFNDR-1 trial, 83% of patients on Palsonify kept their hormone levels in the normal range compared to 4% on placebo. Crinetics runs a patient support program called CrinetiCARE that helps with insurance, copays, and getting started on treatment.

The next major drug in development is atumelnant, which is being studied for two related rare hormone diseases. Both classic congenital adrenal hyperplasia (CAH) and ACTH-dependent Cushing's syndrome have historically forced patients to take very high doses of steroids to keep their disease under control. The high steroid doses do their job but they also cause serious long-term problems including heart disease, weight gain, weakened bones, and metabolic issues. Atumelnant takes a different approach by blocking the signal that tells the adrenal glands to overproduce, which could give patients a way to lower their steroid doses safely. The pivotal Phase 3 CALM-CAH adult trial dosed its first patient in December 2025, a Phase 2/3 pediatric trial called BALANCE-CAH started in January 2026, and the FDA has granted atumelnant Orphan Drug Designation for CAH.

Paltusotine is also being tested for carcinoid syndrome in the Phase 3 CAREFNDR trial (NCT07087054), and CRN09682, a drug built to carry a cell-killing payload to neuroendocrine tumors with the SST2 receptor, is in a Phase 1/2 trial (NCT07129252). Earlier programs, not yet tested in people, target hyperparathyroidism, Graves' disease and thyroid eye disease, and autosomal dominant polycystic kidney disease, according to the company's pipeline page. The throughline across the company is consistent: find rare endocrine diseases where today's treatments rely on injections or blunt hormone replacement, and give patients a precise daily oral alternative that they can take at home instead of in a clinic.

Crinetics Pharmaceuticals has 1 FDA-approved drug with a rare disease use that we track, as of November 2026.

Palsonify (paltusotine), approved 2025: Acromegaly.

Source: the FDA's openFDA label database, matched to the rare diseases on Trial Friend; the list updates automatically and can include older products.

Yes, for 1 of its FDA-approved rare disease medicine that we track, each checked on Crinetics Pharmaceuticals' own website.

Palsonify: CrinetiCARE, 844-276-4357. Offers copay help with private insurance, a free medicine program, a short-term supply while insurance is decided and insurance help. Checked November 9, 2026.

Medicare and Medicaid: drugmaker copay cards are limited to private insurance, so people with government coverage usually look to a free medicine program or a charity fund, both on the help paying page.

Eligibility is decided by each program, so call the number listed before a prescription is filled.