Scientific Partners

We are currently collaborating with the National Human Genome Research Institute (NHGRI) of the National Institute of Health (NIH) in Proteus syndrome, a rare disease characterized by overgrowth of the skeleton, skin, adipose tissue and central nervous system. Our proprietary, selective pan-AKT inhibitor, ARQ 092, is being tested in a phase 1 study to establish clinical proof of concept. The study, which is the first ever clinical trial for Proteus syndrome, should begin enrolling shortly.

In 2011, an NHGRI team led by Dr. Leslie G. Biesecker discovered that the somatic single (point) mutation in the AKT1 oncogene causes Proteus syndrome¹. Subsequently, the NHGRI team presented at the 2014 meeting of the American Society of Human Genetics (ASHG, Abstract # 2180M²), data that 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 provided pre-clinical proof-of-concept for advancement of ARQ 092 into clinical testing for Proteus syndrome.

Our agreement, signed with the NIH in November 2014, is built upon pre-clinical data from NIH research and our own research in oncology showing the shutdown of AKT signaling and the reduction of cell viability. The 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.

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1 N Engl J Med 2011; 365:611-619, August 18, 2011


We are collaborating with the University of Texas Southwestern Medical Center on the clinical development of ARQ 761, an intravenously administered prodrug of Beta-lapachone, a naturally occurring substance. ARQ 761 is an NQO1 inhibitor that has demonstrated the potential to enhance the effect of chemotherapy and other cancer therapies.

In vitro testing showed activity against a wide range of tumors. Phase 1a testing with ARQ 761 identified anti-cancer activity as measured by tumor responses that occurred exclusively in a portion of the patient population with high levels of NQO1, the mechanistic target of the compound. Consequently, the phase 1b expansion cohorts for ARQ 761 will focus on patients whose tumors have high levels of NQO1. We expect our collaborator to begin dosing in a Phase 2 study for pancreatic cancer shortly.


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