Neurocrine reports positive phase 3 data for hyperplasia drug in children

Neurocrine reports positive phase 3 data for hyperplasia drug in children

Just a month after the successful outcome of Neurocrine Biosciences’ phase 3 study for crinecerfont in adults with hyperplasia, the drug has achieved another victory in the pediatric version of the trial.

In the recent update from the CAHtalyst studies, crinecerfont demonstrated a statistically significant reduction in daily glucocorticoid dose compared to a placebo after 28 weeks, while maintaining androgen control, achieving the primary endpoint. The trial involved 103 patients aged 2 to 17 years old with classic congenital adrenal hyperplasia due to 21-hydroxylase deficiency.

These results are in line with similar findings from the adult version of the CAHtalyst trial in September. In particular, the study in children showed a p-value of less than 0.0001 when measuring the reduction in daily glucocorticoid dose at Week 28, which matched the p-value at Week 24 in the adult trial.

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The most common adverse events reported were headache, fever, vomiting, upper respiratory tract infection, and nasopharyngitis. Neurocrine noted that of the “few” serious adverse events, none were considered related to crinecerfont.

Congenital adrenal hyperplasia comprises a group of genetic conditions resulting in enzyme deficiency that affects the production of adrenal hormones. About 95% of cases are caused by a mutation leading to a deficiency of the enzyme 21-hydroxylase, and currently, there are no FDA-approved non-glucocorticoid treatments available.

“As a pediatric endocrinologist, I’m highly encouraged by the results from the CAHtalyst Pediatric study and the potential of crinecerfont to shift the treatment paradigm for a disorder that has seen little innovation in many decades. The data suggest that crinecerfont might enable us to smooth out the numerous adjustments we have to make in glucocorticoid doses to manage high androgen levels as children grow, potentially improving clinical outcomes related to androgen excess as well as chronic supraphysiologic glucocorticoid dosing.”

– Kyriakie Sarafoglou, M.D., Professor, Department of Pediatrics and Department of Experimental and Clinical Pharmacology, Divisions of Endocrinology and Genetics & Metabolism, University of Minnesota

The latest pediatric data will be included in the package of findings submitted to the FDA next year as part of the approval application for crinecerfont.

In a recent note, analysts from William Blair expressed their belief that crinecerfont represents a peak sales opportunity of over $1 billion. Analyst Myles Minter, Ph.D., stated that if the drug reaches the market, it would be a valuable addition to Neurocrine’s commercial pipeline. Currently, the company’s pipeline relies on Ingrezza, a drug for tardive dyskinesia, as Neurocrine discontinued the approved drug Ongentys in May due to what was described as an “unsustainable” launch.

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“The CAHtalyst studies represent the largest interventional clinical trial program conducted in both pediatric and adult CAH patients to date and enrolled a patient population that reflects the unmet need in this condition, with both adult and pediatric patients having inadequate androgen control at baseline despite supraphysiologic glucocorticoid dosing. 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. 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.”

– Eiry W. Roberts, M.D., Chief Medical Officer at Neurocrine Biosciences, Inc

The information gathered from the CAHtalyst Pediatric and Adult studies, along with data from the open-label treatment phases, will be used to prepare regulatory applications for submission to the FDA in 2024, followed by submissions to the European Medicines Agency. Further details regarding the outcomes of both Phase 3 CAHtalyst studies will be presented during the Company’s Analyst Day in December 2023 and published in a peer-reviewed medical journal.

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