Neurocrine's pediatric phase 3 data are missing piece of puzzle for hyperplasia drug submission

A month after Neurocrine Biosciences’ drug crinecerfont renewed analysts’ hopes of its blockbuster potential by acing a phase 3 study in adults with hyperplasia, the therapy has now scored another win in the pediatric version of the trial.

In the latest readout from the CAHtalyst studies, crinecerfont was shown to induce a statistically significant reduction in daily glucocorticoid dose compared to placebo after 28 weeks, while maintaining androgen control, hitting the primary endpoint. The trial enrolled 103 patients aged 2 to 17 years old with classic congenital adrenal hyperplasia as a result of 21-hydroxylase deficiency.

The results mirrored similar findings from the adult version of the CAHtalyst trial in September. Specifically, the study in children demonstrated a p-value of less than 0.0001 when the reduction in daily glucocorticoid dose was measured at Week 28, which matched the p-value when measured at Week 24 in the adult trial.

The most common adverse events were headache, fever, vomiting, upper respiratory tract infection and nasopharyngitis, Neurocrine noted in the Oct. 5 release. Of the “few” serious adverse events, none were considered to be related to crinecerfont, the biotech added.

“The results from these studies suggest that crinecerfont could represent a new standard of care for CAH patients with the ability to reduce androgen levels through a non-glucocorticoid mechanism,” Neurocrine Chief Medical Officer Eiry Roberts, M.D., said in the release. “I am particularly excited about our positive results in the pediatric patient population and what improved androgen control in the setting of reduced glucocorticoid doses could mean for important outcomes related to growth and development."

Congenital adrenal hyperplasia spans a group of genetic conditions that result in an enzyme deficiency that alters the production of adrenal hormones. Around 95% of cases are caused by a mutation that leads to a deficiency of the enzyme 21-hydroxylase, and there are currently no FDA-approved non-glucocorticoid treatments available.

The latest pediatric data will form part of the package of findings submitted to the FDA next year for crinecerfont’s approval application.

In a note published Monday ahead of the leatest readout, William Blair analysts said they “continue to see crinecerfont as a more than $1 billion peak sales opportunity.”

Should the drug reach the market, analyst Myles Minter, Ph.D., said in the note that crinecerfont would provide “an important option to expand the company’s commercial pipeline.” For now, that pipeline still relies on the tardive dyskinesia drug Ingrezza after Neurocrine called it quits on the approved drug Ongentys in May due to what is described as an “unsustainable” launch.