Neurocrine fleshes out its PhIII case for valbenazine; Avrobio adds to gene therapy pipeline

Neurocrine has already posted the key late-stage data that it took to the FDA in building a case for Ingrezza (valbenazine) as a new therapy for tardive dyskinesia. But now it’s published the full set of data in its favor, including a note that its drug scored for 40% of the patients in the drug arm who registered at least a 50% improvement in AIMS dyskinesia score, compared to 8.7% of those on placebo. Neurocrine is facing a head-to-head fight with a new drug from Teva, and a number of analysts think the smaller biotech has a clear shot at dominating the sector. The PDUFA date for the breakthrough therapy is April 11.

→ Cambridge, MA-based biotech Avrobio is expanding its gene therapy pipeline. Investigators are adding a preclinical program for Gaucher disease, which will follow their Phase I for Fabry disease. CEO Geoff Mackay called it “an excellent strategic fit.”

Aprea Therapeutics, a Karolinska Development company, struck a deal to collaborate with Memorial Sloan Kettering Cancer Center on the effects of reactivating tumor suppressor protein p53 by APR-246.

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SV Health Investors Boston, MA
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Contrafect Corporation New York, NY
Director, Translational Sciences
Cadent Therapeutics Cambridge, MA

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