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Neurocrine’s Rare Disease Drug Generates Positive Clinical Data

Interim clinical trial results were positive for a Neurocrine drug aimed at a rare genetic disorder affecting the adrenal glands, the company reported March 12.

NBI-74788, which underwent a phase 2 proof-of-concept study, was found to lower hormones linked to classic congenital adrenal hyperplasia. The disorder can cause menstrual irregularities in females, fertility problems in both sexes, short stature and be life threatening.

Currently, treatment involves corticosteroids, with potential side effects like bone loss and metabolic issues.

“These data provide encouragement that NBI-74788 has potential as a new treatment option to help patients avoid the complications associated with current therapeutic options for classic CAH,” said Richard Auchus, the study’s lead investigator and professor of medicine at University of Michigan Health System.

Neurocrine will meet with the U.S. Food and Drug Administration to talk next steps. The company, among the largest biotechs based in San Diego, has one drug on the market and several in development.

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