On May 17, 2017 ArQule, Inc. (Nasdaq: ARQL) reported that data from the phase 1/2 trial in intrahepatic cholangiocarcinoma (iCCA) with fibroblast growth factor receptor (FGFR) inhibitor, ARQ 087, will be presented on June 3, 2017 at the 2017 ASCO (Free ASCO Whitepaper) Annual Meeting in Chicago, Illinois (Press release, ArQule, MAY 17, 2017, View Source [SID1234519181]). The presentation will include an analysis of iCCA patients with FGFR2 fusions. ARQ 087 is a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth factor receptor (FGFR) family.

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A registrational phase 3 trial with ARQ 087 in second-line iCCA FGFR2 fusion positive patients is planned to begin in the third quarter of 2017. The company has been granted orphan drug designation by the U.S. Food and Drug Administration and European Medicines Agency for ARQ 087 in this indication.

The company will also present at ASCO (Free ASCO Whitepaper) final data from the completed Tivantinib METIV-HCC phase 3 trial for hepatocellular carcinoma.

Data will also be presented for the company sponsored ARQ 092 phase 1b trial in combination with carboplatin plus paclitaxel for oncology, and for the University of Texas Southwest sponsored ARQ 761 phase 1 trial cancer for cell necrosis.

Presentation Details

Saturday, June 3, 2017: Gastrointestinal (Noncolorectal) Cancer

ARQ 087
Abstract 4017/Poster Board: #9
ARQ 087, an oral pan-Fibroblast Growth Factor Receptor (FGFR) inhibitor, in patients (pts) with advanced intrahepatic cholangiocarcinoma (iCCA) with FGFR2 genetic aberrations.
Poster Session
Location: Hall A, 8:00 AM – 11:30 AM CT
Poster Discussion Session
Location: Hall D2, 4:45 PM – 6:00 PM CT

Sunday, June 4, 2017: Gastrointestinal (Noncolorectal) Cancer

Tivantinib (ARQ 197)
Abstract 4000
Second-line tivantinib (ARQ 197) vs placebo in patients (Pts) with MET-high hepatocellular carcinoma (HCC): Results of the METIV-HCC phase III trial.
Oral Abstract Session
Location: Hall D2, 8:00 AM – 8:12 AM CT

Monday, June 5, 2017: Developmental Therapeutics – Clinical Pharmacology and Experimental Therapeutics

ARQ 092
Abstract 2524/Poster Board #16
Results of a phase Ib study of ARQ 092 in combination with carboplatin (C) plus paclitaxel (P), or with P in patients (pts) with solid tumors.
Poster Session
Location: Hall A, 8:00 AM – 11:30 AM CT

ARQ 761
Abstract 2517/ Poster Board #9
Phase 1 study of ARQ 761, a β-lapachone analog that promotes NQO1-mediated programmed cancer cell necrosis.
Poster Session
Location: Hall A, 8:00 AM – 11:30 AM CT
Poster Discussion Session
Location: Arie Crown Theater, 11:30 AM – 12:45 PM CT

About Intrahepatic Cholangiocarcinoma

Cholangiocarcinoma (CCA) is the most common biliary malignancy and the second most common hepatic malignancy after hepatocellular carcinoma (HCC)1. Depending on the anatomic location, CCA is classified as intrahepatic (iCCA), perihilar (pCCA), and extrahepatic (eCCA). iCCA originates from the intrahepatic biliary ductal system and forms an intrahepatic mass. The average age adjusted incidence rate for iCCA is approximately one in 100,000 per year in the United States and Europe2,3.

About FGFR and ARQ 087

ARQ 087 is a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth factor receptor ("FGFR") family with demonstrated efficacy in FGFR2 genetic alterations. The FGFR pathway is disrupted in several ways in human cancer, thus providing numerous therapeutic targets for an inhibitor of this pathway. ARQ 087 has demonstrated in vivo inhibition of tumor growth and downstream signaling in tumors whose growth is driven by FGFR targets.

Signals of single agent activity with this drug were observed in phase 1a testing. Phase 1b expansion cohorts with ARQ 087 include patients with cholangiocarcinoma and adrenocortical tumors, as well as those with FGFR translocations, amplifications and mutations. Clinical development of ARQ 087 advanced into phase 2 for intrahepatic cholangiocarcinoma (iCCA) in patients with FGFR2 fusions following the observation of two confirmed responses in this patient population in the phase 1 portion of the program, and a phase 3 registrational trial is planned to begin in the third quarter of 2017 in this same patient population.

About the AKT Pathway and ARQ 092

ARQ 092 is an orally bioavailable, selective small molecule inhibitor of the AKT kinases. The AKT pathway when abnormally activated is implicated in multiple oncogenic processes such as cell proliferation and apoptosis. This pathway has emerged as a target of potential therapeutic relevance for compounds that inhibit its activity, which has been linked to a variety of cancers as well as to select non-oncology indications.

Dysregulation of AKT is also a driver of certain rare proliferative disorders. For example, the E17K mutation of AKT1 causes Proteus syndrome, a rare non-cancerous segmental overgrowth disorder, and the analogous PIK3CA-Related Overgrowth Spectrum (PROS) is caused by genetic alterations in the PI3K pathway. ARQ 092 has been shown preclinically and clinically to inhibit AKT and PI3K cell signaling and therefore may provide the potential for much-needed treatment options for patients with these diseases.

ARQ 092, the lead compound in ArQule’s AKT program, has completed phase 1a clinical testing and has advanced into phase 1b expansion testing in cohorts of patients with endometrial cancer, lymphomas and tumors harboring either AKT or PI3K mutations. A company sponsored phase 1/2 trial is being conducted in the U.S. and E.U. for Overgrowth Diseases, including PROS and Proteus syndrome. ARQ 092 is also in a phase 1 trial being conducted by the NIH for Proteus syndrome. Collaborators are exploring in preclinical testing other indications for ARQ 092, including sickle cell disease.