ARQ 087 is an investigational oral FGFR inhibitor in phase 1b for solid tumors and phase 2 for intrahepatic cholangiocarcinoma. The phase 2 trial is biomarker-driven and enrolling patients with FGFR translocations. Single agent activity with ARQ 087 was observed in Phase 1a and Phase 1b studies in patients with FGFR2 fusions and amplified FGFR1.
ARQ 087 is an orally bioavailable compound and is a dual kinase inhibitor that binds to inactive form of FGFR1 and FGFR2 and potently inhibits the active form of FGFR1 and FGFR2. ARQ 087 has demonstrated inhibition of tumor growth and downstream signaling in vivo in tumors whose growth is driven by these targets.
Increased FGFR levels were identified as a potential surrogate marker in the phase 1a trial. In our studies with ARQ 087, FGFR2 dysregulation correlates with efficacy. The translation of results from FGFR2 preclinical models into clinical efficacy in patients with FGFR2 fusion driven cholangiocarcinoma is encouraging and shaped the ongoing phase 2 portion of the clinical trial.
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.