Neurocrine's hopes for hyperplasia blockbuster a step closer after phase 3 win

Neurocrine Biosciences has a lot riding on its hyperplasia drug crinecerfont, which analysts tout as a potential blockbuster. Plus, the company needs a win after throwing in the towel on a Parkinson’s disease med. Luckily, phase 3 data have delivered the goods.

Crinecerfont was shown to induce a statistically significant reduction in daily glucocorticoid dose compared to placebo after 24 weeks, while maintaining androgen control, hitting the primary endpoint of the phase 3 study. The trial had enrolled 182 patients with classic congenital adrenal hyperplasia as a result of 21-hydroxylase deficiency.

The study also hit secondary endpoints thanks to a statistically significant decrease in androstenedione at week 4. The crinecerfont cohort was more likely to see a reduction in physiologic glucocorticoid dose, with 63% reporting this benefit compared to 18% on placebo, the company explained.

Crinecerfont was generally well tolerated, Neurocrine said in its Tuesday morning release, with the most common adverse events being fatigue, headache and coronavirus infection. No serious adverse events were related to the drug.

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.

"For physicians, the current treatment paradigm is problematic, relying on glucocorticoids for a dual purpose: not only to address the underlying cortisol deficiency but typically at supraphysiologic doses to treat androgen excess resulting in well-known complications over the long-term,” Neurocrine Chief Medical Officer Eiry Roberts, M.D., said in today’s release.

The latest readout keeps the biotech on track to submit an approval application to the FDA in 2024. Data from a phase 3 trial in children is due early in the fourth quarter of this year, and analysts have already raised the prospect of crinecerfont achieving blockbuster status if the drug reaches the market.

For now, the tardive dyskinesia drug Ingrezza remains Neurocrine’s chief revenue generator. The biotech is anticipating total sales of up to $1.7 billion for the med this year, excluding a recent label expansion into Huntington's disease. Work in Parkinson’s disease has proved less successful, with Neurocrine calling it quits on the approved drug Ongentys in May due to an “unsustainable” launch.