ArQule announced today that registrational trial of its FGFR inhibitor, derazantinib (ARQ 087) in FGFR2 fusion driven second-line intrahepatic Cholangiocarcinoma (iCCA), has initiated the dosing of patients.
ARQ 087, which was granted orphan drug designation
by the U.S. Food and Drug Administration (FDA) in December 2015, is a multi-kinase inhibitor designed to reduce or prevent the fibroblast growth receptor (FGFR) family. The locations of FGFR have been previously identified as a potential target for tyrosine kinase inhibitor therapies, and derazantinib has exhibited in vivo
inhibition of tumor growth and downstream signaling in tumors whose growth is driven by FGFR.
Intrahepatic Cholangiocarcinoma (iCCA) is the most common biliary malignancy and the second most common hepatic malignancy, behind only hepatocellular carcinoma (HCC). It develops in the biliary ductal system and creates an intrahepatic mass. The condition currently affects approximately 1 in 100,000 people per year between the United States in Europe.
Depending on the anatomic location, CCA can be classified as either intrahepatic (iCCA), perihilar (pCCA), or extrahepatic (eCCA).
“iCCA is a rare and difficult disease to treat,” said Dr. Brian Schwartz, M.D., Head of Research and Development and Chief Medical Officer at ArQule in a press release
. “The durable response rate of 21% we observed in the phase 1/2 iCCA FGFR2 fusion driven trial has lead us to design the first registrational trial with a biomarker for this patient population. We are happy to report that recruitment is off to a great start with four patients already dosed.”
Objective response rate (ORR) will serve as the primary endpoint in the open-label single-arm trial that is recruiting in the U.S. Derazantinib will be dosed orally, once-a-day at 300 mh, and an interim analysis will be performed after the first 40 patients have been enrolled and evaluated for a response.
Previous phase 1a studies have observed signals of single agent activity with the drug, and phase 1b expansion cohorts with derazantinib include patients with cholangiocarcinoma and adrenocortical tumors, in addition to those with FGFR translocations, amplifications and mutations.
ArQule is currently enrolling patients into the Phase 3 trial for FGFR2 Fusion Positive iCCA, and more information can be found on clinicaltrials.gov: NCT0323018
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