Neurocrine breaks out proof-of-concept data for CAH drug; Akebia, MTPC work toward Japanese NDA after anemia drug scores in twin PhIIIs
→ More than a month after its big ticket deal with Voyager Therapeutics $VYGR, Neurocrine Biosciences $NBIX announced positive interim data from a Phase II proof-of-concept study evaluating its experimental drug, NBI-74788, in adults with a genetic disorder that results in an enzyme deficiency that alters the production of adrenal steroids called classic congenital adrenal hyperplasia (CAH). Data showed a reduction of at least 50% from baseline in 17-hydroxyprogesterone (17-OHP) — used as a diagnostic biomarker in CAH — in more than half the treated patients. “But what does a 50% ‘responder’ rate mean in clinical terms? This study wasn’t really designed to answer this question, and while we don’t know the patients’ 17-OHP baseline values in this study, our guess from looking at other CAH trials (ahead of the data today) is that it was probably very high. Thus, it is unclear to us today how these biomarker changes will or will not translate into an improvement in patient outcomes,” Stifel’s Paul Matteis wrote.
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