Neurocrine Biosciences stock jumped Monday after the biotech company won Food and Drug Administration approval for the first new treatment for an adrenal disease in decades.
The approval of Neurocrine's newest drug, Crenessity, came roughly two weeks ahead of expectations. Crenessity, formerly crinecerfont, is treats congenital adrenal hyperplasia, or CAH, in adults and children as young as 4 years old. Neurocrine stock popped 5% to 133.05.
CAH is characterized by an imbalance between hormones called cortisol and androgens. When the body can't make enough of the cortisol hormone, another hormone known as ACTH climbs. This causes the body to overproduce androgens, or male sex hormones like testosterone.
For decades, patients have used glucocorticoids to treat their CAH. But long-term use of these drugs — especially at the high dosages required in CAH — can cause weight gain, cardiovascular issues and bone loss.
Crenessity will be an important change for patients, said Neurocrine Chief Executive Kyle Gano.
"Patients aren't having to make that Sophie's choice anymore between high ACTH, high androgens or high (glucocorticoids) and dealing with the consequences of high-dose (glucocorticoids)," he told Investor's Business Daily. "We think this is going to change the standard of care for patients with CAH."
Neurocrine Biosciences: First-In-Class Drugs
Ahead of the approval, Gano said the company expects to launch Crenessity within a week of its approval. He estimates there are 30,000 patients in the U.S. with CAH. They mirror the country's population: roughly a third are adolescents and two-thirds are adults.
Neurocrine Biosciences has yet to announce the price for Crenessity. William Blair analyst Myles Minter models $1,116 per dose, leading to an estimated net price of $264,784 a year.
"We believe this to be in the orphan disease pricing range that management has qualitatively talked about prior and net price will ultimately be a function of payer contracting/negotiations," he said in a report. "We anticipate a 2025 revenue for Crenessity in the U.S. around $150 million and have modeled peak global Crenessity sales of about $1.47 billion in 2030 pending global approvals."
Minter kept his outperform rating on Neurocrine stock.
The Most Motivated Patients
For children, the earlier they start treatment, the better, said Gano, the company's chief executive. Parents are particularly motivated to get their children off the glucocorticoid roller coaster. William Blair's Minter noted the label for Crenessity isn't restrictive, permitting a broad range of CAH patients.
Crenessity will be Neurocrine Biosciences' third drug on the market. The company is best known for its drug Ingrezza, which treats tardive dyskinesia. Neurocrine also licensed to AbbVie the rights to an endometriosis treatment called Orlissa.
"All of these medicines, when they were approved were the first in class," Gano said. "We do think that Ingrezza and crinecerfont will change the standard of view for patients, and we're hoping that view, externally, will be one that people take away for Neurocrine. They're all first-in-class medicines that can change the standard of care for patients. And that's really our north star, where we point all our R&D."
Follow Allison Gatlin on X, the platform formerly known as Twitter, at @IBD_AGatlin.
