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.

Mechanism of Action

Fibroblast growth factors and their receptors tightly regulate key cellular behaviors, such as proliferation, cell differentiation, cell migration, cell survival and angiogenesis. In human cancers, FGFRs have been found to be dysregulated by multiple mechanisms, including aberrant expression, mutations, chromosomal rearrangements, and amplifications. FGFR dysregulation has been identified as a driver in a number of cancers, including iCCA, cholangiocarcinoma, bladder, endometrial, breast, gastric, lung and ovarian. Current scientific literature suggests FGFR alterations exists in anywhere from 5% to 40% of these cancers. Derazantinib is a potent FGFR inhibitor that shows strong anti-proliferative activity in cell lines harboring FGFR2 alterations. In clinical testing the molecule has demonstrated activity in cancerous tumors harboring FGFR2 fusions in iCCA and bladder cancers.

Precision Medicine

Increased FGFR levels were identified as a potential surrogate marker in the phase 1a trial. In our studies with Derazantinib, FGFR2 dysregulation correlates with efficacy. The translation of results from FGFR2 preclinical models into clinical efficacy in patients with FGFR2 fusion driven intrahepatic cholangiocarcinoma is encouraging and shaped the ongoing phase 2 portion of the clinical trial and the design of the planned registrational trial.

Diseases We Are Exploring in Clinical Trials

Intrahepatic Cholangiocarcinoma (iCCA) (bile duct cancer) is a rare and difficult to treat cancer that occurs in the small, tube-like bile ducts within the liver that carry bile to the gallbladder. Current treatment is based on the patient’s stage of the cancer when diagnosed and include resection, chemoradiation and systemic chemotherapy.

Current Clinical Trials

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