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.

Akebia, which merged with Auryxia maker Keryx last year, put out positive late-stage data from twin Phase III Japanese studies evaluating its lead experimental drug vadadustat as an anemia treatment for CKD patients. Partner Mitsubishi Tanabe Pharma Corp (MTPC) expects to submit a Japanese marketing application in 2019. Akebia $AKBA is in a fierce race with FibroGen $FGEN in both Japan and the United States to get their respective anemia drugs on the market. FibroGen and its partner Astellas reported positive 4th Phase III study of their rival roxadustat in Japan in 2018.

→ UK’s Oxford Biomedica is joining forces with tech major Microsoft in a research and development collaboration to develop next-gen gene therapy vectors using the cloud and machine learning.

Lipocine provided updated data from its “liver fat” study evaluating its experimental oral drug — LPCN 1144 — which is being developed for NASH. In January, the company said the drug was being evaluating in 36 hypogonadal males in a 16-week study. Seven of the nine subjects had at least 10% baseline liver fat, which the company said is indicative of subjects with NAFLD with the potential to have NASH. Baseline mean liver fat of these seven subjects was 21.0%, and after treatment there was an average absolute mean reduction from baseline of 7.6% in liver fat after 8 weeks of treatment. On Tuesday, Lipocine said 62% of the of the evaluated subjects had NAFLD, defined as baseline liver fat of at least 5%. At the end, 48% of the treated NAFLD subjects had NAFLD resolution, defined as liver fat <5% post treatment for 16 weeks. The company’s shares $LPCN were up 7.3% in morning trading.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
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Atlas Venture Cambridge, MA

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