Small Molecules for Precision Medicine

ArQule is a biopharmaceutical company engaged in the research and development of targeted therapeutics to treat cancers and certain rare diseases.

Our mission is to discover, develop and commercialize novel small molecule drugs that will dramatically extend and improve the lives of patients in areas of high unmet need.

Our prioritized clinical-stage pipeline consists of five drug candidates, all of which are in targeted, biomarker-defined patient populations, making ArQule a leader in precision medicine.  These drug candidates include:

 Derazantinib (ARQ 087)

Derazantinib (ARQ 087) is an investigational, oral, multi-kinase inhibitor designed to preferentially inhibit the FGFR family of kinases with demonstrated activity in FGFR2 genetic alterations, including fusions. The drug has demonstrated favorable clinical data in a biomarker-driven Phase 1/2 trial in iCCA targeting patients with FGFR2 fusions. Both the FDA and EMA have granted ArQule orphan drug designation for this disease. We plan to initiate a registrational biomarker-driven trial in this indication in Q3 2017 and subsequently seek breakthrough therapy designation with the FDA. Learn more »

Miransertib (ARQ 092)

Miransertib (ARQ 092) is an investigational orally available, selective, pan-AKT inhibitor that potently inhibits AKT1, 2 and 3 isoforms. The drug is currently in phase 1b for oncology and phase 1 for Proteus syndrome, a rare over-growth disease, in a trial being conducted by the National Institutes of Health (NIH). Both trials are biomarker-driven and are focused on enrolling patients with the AKT1 mutation. We plan to initiate a company sponsored Phase 1/2 trial in overgrowth syndromes, including PROS (PIK3CA Rare Overgrowth Syndromes) and Proteus syndrome, in Q2 of 2017. Learn more »

ARQ 751

ARQ 751 is an investigational, orally available, selective, next generation pan-AKT inhibitor that potently inhibits AKT1, 2 and 3 isoforms. With certain properties distinct from Miransertib, this second clinical-stage AKT inhibitor could provide development options for the Company as it pursues indications in oncology and rare diseases.  A Phase 1 biomarker driven trial in cancers with AKT mutations commenced in mid-2016. Learn more »

ARQ 531

ARQ 531 is an investigational, orally bioavailable, potent and reversible inhibitor of both wild type and C481S-mutant Bruton’s tyrosine kinase (BTK). We commenced a phase 1 trial in patients with B-cell malignancies refractory to other therapeutic options in July of 2017.  Learn more »

 ARQ 761

ARQ 761 is an intravenously administered analogue of β-Lapachone that targets patients with elevated levels of NQO1; a Phase 2 trial is being conducted by our collaborator, the University of Texas Southwestern Medical Center, in pancreatic cancer patients with elevated levels of NQO1.  Learn more »


With five internally discovered drugs in multiple biomarker guided clinical trials and an enriched proprietary kinase library from which to select additional drug candidates, ArQule is delivering on the potential of precision medicine by providing its patients with novel therapies for multiple hard to treat indications and its shareholders with a focused, highly efficient and cost effective research and development strategy.

ArQule is led by a senior management team with significant clinical and commercial expertise that provides the leadership required to optimize its opportunities for success in a rapidly changing and highly competitive field.

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