Neurocrine Biosciences sold $53 million of CRENESSITY in the second quarter of 2025. A year later, the same drug brought in $184 million, and a second rare-disease product, acquired in May, added $54 million in its first six weeks on Neurocrine’s books.
Neither drug appeared on the company’s income statement two years ago. They now account for about a quarter of revenue, and they give a company long defined by a single movement-disorder drug a second line of business while it pursues riskier science, including a Phase 2 trial in Alzheimer’s disease that the Food and Drug Administration cleared on Aug. 26.
CRENESSITY and Congenital Adrenal Hyperplasia
Classic congenital adrenal hyperplasia is a genetic disorder, usually caused by a deficiency in the enzyme 21-hydroxylase, in which the adrenal glands cannot make enough cortisol. The body responds by overproducing adrenocorticotropic hormone, which drives excess adrenal androgens. For decades, the standard treatment has been glucocorticoid steroids at doses high enough to suppress that cycle, with side effects that build over a lifetime. Most U.S. cases are identified through newborn screening, and treatment continues for life.
CRENESSITY (crinecerfont) blocks the corticotropin-releasing factor type 1 receptor, which sits upstream of that hormone cascade. The FDA approved it in December 2024. In its Phase 3 trial, 63% of patients reached physiologic steroid doses, according to a summary by BioSpace.
Quarterly sales rose from $14.3 million in the first quarter of 2025 to $53 million in the second and $98 million in the third. Full-year 2025 sales reached $301.2 million. In the second quarter of 2026, sales hit $184 million, up 247%, and about 80% of dispensed prescriptions were reimbursed, Neurocrine said.
Those figures already strain older market estimates. A market research report cited by BioSpace put the entire congenital adrenal hyperplasia treatment market at about $500 million in 2024, rising above $1 billion by 2035. CRENESSITY’s second-quarter sales, multiplied by four, exceed $700 million.
“Approximately 15% of the estimated diagnosed patient population has now been prescribed CRENESSITY,” Chief Commercial Officer Eric Benevich said on the company’s July earnings call. “Importantly, our prescriber base has nearly tripled compared to 1 year ago, providing a strong foundation for continued growth.”
A Competitor Several Years Behind
Crinetics Pharmaceuticals is developing the most advanced rival. Its drug, atumelnant, blocks the receptor for adrenocorticotropic hormone directly. In Phase 2 data released in early 2026, seven of eight patients in one cohort reduced their steroid use after 12 weeks. A later update reported no serious adverse events. Crinetics shares rose about 3% on the day of one later data update, and Neurocrine’s fell by a similar amount.
The comparison with CRENESSITY carries caveats. Patients in the atumelnant study started on 24 milligrams of steroids, against 32 milligrams in CRENESSITY’s Phase 3, and 20% of atumelnant patients dropped out, compared with 4% in the CRENESSITY trial. Counting those dropouts, the share of patients who reduced steroids falls to about 70%, BioSpace reported.
Crinetics raised $350 million in a public offering in January and has started a 32-week Phase 3 trial. Analysts at Truist Securities said at the time that Crinetics was “several years from potential approval” and that by then Neurocrine would likely have penetrated much of the market. The Truist analysts wrote that they “see little rationale for stable Crenessity patients to switch therapies.”
VYKAT XR and the Soleno Deal
Neurocrine’s second rare-disease product came by acquisition. The company bought Soleno Therapeutics for $53 a share in cash, about $2.9 billion, completing the deal through a tender offer on May 18.
Soleno’s VYKAT XR (diazoxide choline) won FDA approval on March 26, 2025, for patients 4 and older with Prader-Willi syndrome, a genetic disorder involving chromosome 15 that disrupts the brain’s signals of fullness. No drug had previously been approved for that hunger, known clinically as hyperphagia.
“Today marks an important advancement in Neurocrine’s mission to deliver life-changing treatments for patients with significant unmet needs,” Chief Executive Kyle Gano said when the transaction closed.
Families have long managed the condition with locked kitchens and close supervision. Neurocrine acquired the product about 14 months after its FDA approval, once the launch was already generating sales.
VYKAT XR contributed $54 million in the six weeks Neurocrine owned it during the second quarter. Had the deal closed at the start of the quarter, the drug would have produced $94 million and total company revenue would have been $998 million, according to unaudited pro forma figures in the earnings release.
The purchase came at a cost to the balance sheet. Cash, equivalents and marketable securities fell to $482 million at June 30 from $2.54 billion at the end of 2025. Neurocrine entered a $1 billion revolving credit facility in May and had not drawn on it by quarter-end. The Soleno price of $53 a share was paid entirely in cash, with no new Neurocrine stock issued. The company’s 2026 guidance now includes $85 million to $90 million in amortization of acquired intangibles, a new line tied to the deal, and its GAAP selling, general and administrative forecast rose to $1.575 billion to $1.6 billion from $1.375 billion to $1.4 billion, reflecting the larger company after the acquisition.
INGREZZA Still Leads
INGREZZA, Neurocrine’s treatment for tardive dyskinesia and Huntington’s disease chorea, remains the largest contributor. It sold $716 million in the second quarter, up 15%, and the company raised its full-year forecast to $2.825 billion to $2.875 billion. Total revenue rose 39% to $959 million. Other revenue, a line that includes payments tied to AbbVie’s elagolix-based women’s health drugs, came to $5 million.
The rare-disease products changed the mix. CRENESSITY and VYKAT XR together produced $238 million, about 25% of second-quarter revenue. A year earlier, CRENESSITY’s $53 million was less than 8% of a $688 million total. On its own, CRENESSITY now accounts for about 19% of quarterly revenue.
“Our second quarter performance demonstrates the power of a strategy designed to compound over time,” Gano said in the release.
What the Revenue Funds
Neurocrine expects GAAP research and development expense of $1.275 billion to $1.325 billion this year. Its late-stage programs include direclidine for schizophrenia and osavampator for major depressive disorder, with Phase 3 results due in 2027. Gano described “multiple important clinical milestones in 2027” in the second-quarter release.
The Alzheimer’s program is earlier. NBI-1117567, an oral drug that favors the M1 muscarinic receptor, is designed to improve cognition, and the FDA’s acceptance of the Phase 2 application triggered a $10 million payment to Nxera Pharma, which discovered the molecule. The 2021 Nxera license that produced it could be worth up to $2.6 billion to Nxera over time, and NBI-1117567 is its third molecule to reach a mid-stage trial.
The program sits beside three other muscarinic compounds from the Nxera collaboration. Direclidine is in Phase 3 for schizophrenia, NBI-1117570 is in Phase 2, and NBI-1117569 is in early testing in Alzheimer’s with the aim of moving into Alzheimer’s psychosis.
Alzheimer’s has turned back most of the drugs aimed at it. One success in roughly 250 attempts was the record for 2002 to 2012, a 99.6% failure rate in Cummings’ 2014 count. From 2004 through 2021, 98 compounds failed in Phase 2 or 3 and two reached a reported success, by a 2022 tally in the Journal of Alzheimer’s Disease. Antibodies against amyloid now on the market slow the disease but leave lost cognition unrestored, and restoring some of it is what NBI-1117567 will be tested on.
The trial will run in a field with more company than a decade ago. The 2026 Alzheimer’s pipeline review led by Jeffrey Cummings of the University of Nevada, Las Vegas, counted 158 drugs in 192 trials. Phase 2 had the most activity, with 84 drugs in 89 trials.
Who Holds the Shares
Institutional investors own about 92.6% of Neurocrine, MarketBeat data show. Among second-quarter filers, State Street held about 4.4 million shares, a stake worth roughly $739 million at the end of June, AQR Capital Management about 3.8 million and Two Sigma Investments about 2.1 million. Invesco started a new position of about 1.1 million shares, and quantitative manager Engineers Gate lifted its stake 64%. MarketBeat’s tally of analyst ratings in September averaged out to “Moderate Buy.”
Armistice Capital, the hedge fund Steven Boyd founded in 2012, reported 535,838 Neurocrine shares worth about $90.3 million as of June 30, according to GuruFocus. The position represented 1.1% of the $8.17 billion Armistice disclosed in its 13F filing, alongside holdings in other drug developers and medical companies. Most of the reported book’s value sits in put options on two index funds, StockZoa data show.
The rare-disease side of Neurocrine’s business has its own dates and numbers ahead of the Alzheimer’s data. CRENESSITY has reached about 15% of the diagnosed patient population, VYKAT XR will report its first full quarter under Neurocrine in the third-quarter results, and Crinetics’ Phase 3 program has only begun.