Neurocrine Biosciences drug for congenital adrenal hyperplasia succeeds in late-stage trial

SAN DIEGO — Neurocrine Biosciences announced on Tuesday that an experimental drug for congenital adrenal hyperplasia succeeded in a late-stage trial, bringing the company a step closer to treating patients with a rare and serious set of genetic disorders that alters their ability to produce key hormones.

In the Phase 3 study, known as CAHtalyst, 182 adults were randomly assigned to receive a placebo or crinecerfont, an oral drug Neurocrine developed to lower male sex hormones patients with the disease produce in excess. Researchers found that, compared to those on placebo, participants who took crinecerfont needed a significantly lower dose of the current standard-of-care treatment, glucocorticoids, after 24 weeks of treatment that they did at the study’s start. And about 63% of participants who took crinecerfont were able to lower their dose of glucocorticoids to levels researchers think would be safer for prolonged use, compared to 18% of those on the placebo.

Neurocine, a San Diego biotech focused on treating rare neurological and endocrine diseases, believes the drug could allow people living with congenital adrenal hyperplasia, or CAH, to avoid some of the more serious side effects of glucocorticoids by allowing them to take lower doses. Long-term use of high doses of these drugs can cause patients to gain weight and put them at risk of diabetes, heart disease, and osteoporosis.