Earlier today at the 2017 American Society of Clinical Oncology (ASCO) Annual Meeting, ArQule, Inc. announced that data from a phase 1/2 trial with ARQ 087 exhibits significant clinical benefit to intrahepatic cholangiocarcinoma (iCCA) patients housing FGFR2 fusions.
iCCA is a primary liver malignant tumor, and originates from the intrahepatic biliary ductal system, forming an intrahepatic mass. These data support future development of the multi-kinase inhibitor as second-line treatment.
, titled “ARQ 087, an oral pan-Fibroblast Growth Factor Receptor (FGFR) inhibitor, in patients with advanced intrahepatic cholangiocarcinoma (iCCA) with fibroblast growth factor receptor 2 (FGFR2) genetic aberrations,” displayed data showing a strong response rate and prolonged duration of therapy for patients, well-exceeding that reported for second-line chemotherapy.
“Clinical evidence is accumulating on the role of FGFR inhibitors in cholangiocarcinoma and other FGFR driven tumors such as urothelial and gastric cancers,” said Dr. Brian Schwartz, M.D., Head of Research and Development and Chief Medical Officer at ArQule.
“We are encouraged to see that both the response rate and disease control rate were consistent throughout the trial. Patients with iCCA often have a poor prognosis for front-line treatment with chemotherapy, and there are no currently approved second-line therapeutic options.”
The data is comprised of 35 iCCA patients harboring FGFR2 genetic alterations, of which 29 patients were FGFR2 fusion positive. All 29 of these patients were evaluable for this data presentation, and evaluated using Standard RECIST (Response Evaluation Criteria in Solid Tumors).
The objective response rate among the 29 patients was 21%, and the disease control rate was 83%. Clinical benefit was observed in 72%. The median duration of therapy seen in iCCA FGFR2 fusion positive patients was 182 days, and the median duration of front-line chemotherapy was 119 days.
ARQ 087, designed to preferentially inhibit the fibroblast growth factor receptor (FGFR) family, showed a controllable safety profile with primarily Grade 1 and 2 adverse events (AE). Nine patients with FGFR2 fusions are still continuing in the trial.
A registrational phase 3 trial in iCCA FGFR2 fusion positive patients is planned to begin in the third quarter of 2017.