(By Balaseshan) ArQule Inc. (NASDAQ:ARQL) and its partner Daiichi Sankyo have reached a special protocol assessment (SPA) agreement with the U.S. Food and Drug Administration (FDA) for the design of a Phase 3 trial of tivantinib in patients with liver cancer.
The Phase 3 trial will be a randomized, double-blinded study of tivantinib as a single agent therapy in previously treated patients with MET diagnostic-high inoperable hepatocellular carcinoma (HCC). The drug is being evaluated broadly as a monotherapy and as part of combination therapy against a variety of cancers.
The primary endpoint is overall survival in the intent-to-treat population, and the secondary endpoint is progression free survival in the same population. About 300 patients are planned to be enrolled at about 120 centers worldwide.
Tivantinib is an orally administered, selective inhibitor of MET, a receptor tyrosine kinase. In healthy adult cells, MET is present in normal levels to support natural cellular function, but in cancer cells MET is inappropriately and continuously activated for unknown reasons.
When abnormally activated, MET plays multiple roles in aspects of human cancer, including cancer cell growth, survival, angiogenesis, invasion and metastasis. Tivantinib has not yet been approved for any indication in any country.
The SPA process is a procedure by which the FDA provides official evaluation and written guidance on the design and size of proposed protocols that are intended to form the basis for a new drug application. Final marketing approval depends on the results of the trial.
Globally, liver cancer is the sixth most common cancer (749,000 new cases), accounting for 7 percent of all cancers, and is the third cause of cancer related death (692,000 cases). HCC represents more than 90 percent of primary liver cancers.
Earlier in October, the company and Daiichi Sankyo discontinued a Phase 3 trial evaluating the selective MET inhibitor tivantinib (ARQ 197) in patients with nonsquamous non-small cell lung cancer (NSCLC). This follows an interim analysis from the Marquee study's independent data monitoring committee, which concluded the trial wouldn't meet its primary endpoint of improved overall survival.
ARQL is trading up 4.91 percent at $2.78 on Tuesday. The stock has been trading between $1.98 and $8.32 for the past 52 weeks.