WOBURN, Mass.--(BUSINESS WIRE)--ArQule, Inc. (NASDAQ: ARQL) today announced an agreement with the National Human Genome Research Institute (NHGRI) of the National Institutes of Health (NIH) providing for the clinical development of ARQ 092, an orally available, selective small molecule inhibitor of AKT, in Proteus syndrome, a rare disease characterized by overgrowth of the skeleton, skin, adipose tissue and central nervous system.
The NIH Proteus syndrome team is led by researchers who originally discovered the somatic single (point) mutation in the AKT 1 oncogene that causes Proteus syndrome1. On that basis, the NIH entered into a collaboration with ArQule focused on pre-clinical testing of ARQ 092. Results from that pre-clinical research recently presented by the NIH team at the 2014 meeting of the American Society of Human Genetics (ASHG, Abstract # 2180M2) demonstrate that treatment with ARQ 092 caused a rapid shutdown of AKT signaling and a reduction in the viability of Proteus syndrome cells taken from patients compared to untreated diseased cells. These findings represent pre-clinical proof-of-concept for advancement of ARQ 092 into clinical testing in this indication.
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