On November 10, 2014 ArQule reported positive top-line results from a randomized, double-blind, placebo-controlled Phase 2 clinical trial of tivantinib as a single agent in metastatic prostate cancer (Press release ArQule, NOV 10, 2014, View Source [SID:1234500944]). This trial (clinicaltrials.gov identifier NCT01519414) was conducted under a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute’s Cancer Therapy Evaluation Program (CTEP) (“A Randomized, Double-Blind, Placebo-Controlled Phase 2 Study of ARQ 197 (tivantinib) in Men with Asymptomatic or Minimally Symptomatic Metastatic Castrate Resistant Prostate Cancer,” NCI Protocol # 8986). The principal investigator was J. Paul Monk, M.D. of The Ohio State University, Arthur G. James Cancer Hospital and Solove Research Institute.

In this trial, 78 patients were randomized 2 to 1 to receive either tivantinib as a single agent or placebo. During a pre-planned analysis, it was found that the trial met its primary endpoint of improving median progression-free survival (PFS) with tivantinib alone as compared to placebo. The results were highly statistically significant. Safety data were consistent with those observed in other trials of tivantinib.

The results of the trial are the subject of ongoing analyses and will be submitted by the investigators for presentation at a future medical conference. As final data emerge from this trial, ArQule and its partner, Daiichi Sankyo Company, Limited, will discuss with the National Institutes of Health (NIH) the potential for additional trials in this indication.

“We are pleased that these significant findings from the NIH trial in prostate cancer add to the body of clinical evidence of the anti-cancer activity of tivantinib across multiple tumor types,” said Paolo Pucci, chief executive officer of ArQule.

“Our CRADA reflects our shared commitment with the NCI to continue to explore the clinical potential of tivantinib in tumor types beyond the compound’s lead indication, hepatocellular carcinoma (HCC), currently in Phase 3 clinical trials,” said Mr. Pucci.

Uncontrolled, signal generation data from additional NIH-sponsored trials with tivantinib in breast cancer and multiple myeloma did not meet their primary endpoint of response rate. As a result, the Company does not plan to prioritize development in these indications at this time.