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news.ArQule has 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.
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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 syndrome.
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.
"This collaboration provides the opportunity to clinically investigate the therapeutic impact of ARQ 092 in a rare, genetic disorder that has no approved therapy and for which the only current treatment is surgery," said Dr. Leslie G. Biesecker, M.D., Chief of the Medical Genomics and Metabolic Genetics Branch at the National Human Genome Research Institute.
"We believe the goal of treatment for Proteus syndrome will be early diagnosis and administration of therapy over years to mitigate the abnormal cell signaling initiated by the point mutation."
"The need for medical treatment of Proteus syndrome is high," said Brian Schwartz, M.D., chief medical officer of ArQule.
"Characterized by non-cancerous overgrowths of tissue, Proteus syndrome is the first non-oncology rare indication to enter clinical testing for ARQ 092. We are hopeful that intervention in a single aberrant pathway responsible for this disease may hold potential for a therapeutic advance."
The NIH is currently developing a protocol for a Phase 1 trial with ARQ 092 in this indication, with input from ArQule. The Company will also be providing compound for the planned NIH clinical trial, which is currently expected to begin in 2015.