- Clinical-stage drugmaker ArQule announced a blow to its pipeline on Friday as its lead candidate failed in a late-stage study testing the drug in patients with liver cancer.
- The randomized, double-blind, placebo-controlled study enrolled 368 patients who have inoperable hepatocellular carcinoma and who either can't receive or have already received chemotherapy. The drug being tested, tivantinib, didn't meet its primary endpoint: significantly improving the overall survival rate of those patients.
- ArQule wasn't optimistic about further investigation of the drug for that indication during a Feb. 17 investor call. "Although we will keep an eye on any further developments for tivantinib, our primary goal – our almost exclusive goal now – is to support the pipeline development," the company's CEO Paolo Pucci said.
The last chance tivantinib has for treating liver cancer is in specific ethnic populations. The drug is currently in another Phase 3 trial testing it in liver cancer patients in Japan. That study, which comes from a partnership between ArQule and Japanese R&D specialist Kyowa Hakko Kirin Co., enrolled 160 patients and should wrap up in July, with an initial readout most likely to come early in the second quarter, according to Pucci.
That's not to say tivantinib is being shelved altogether. Pucci said his company is planning a third Phase 3 trial, though did not mention for what indication, and will conduct the trial without a partner. ArQule had nearly $15 million in cash and cash equivalents as of Sept. 30 and spent $5.2 million on research and development during the third quarter, according to its most recent 10-K filing with the Securities and Exchange Commission.
This isn't the first time tivantinib has disappointed. In 2012, an independent data monitoring committee advised stopping a Phase 3 trial testing the drug in patients with non-small cell lung cancer after interim results raised doubts that the study's primary endpoint would be reached.
Tivantinib inhibits c-MET receptor tyrosine kinase, a protein that helps control the longevity of a cell. When mutated, c-MET can lead to cancer.
Daiichi and ArQule first linked up in 2008, when the former company paid $75 million upfront for access to two of the latter's programs. Licensing, co-development and co-commercialization rights to tivantinib made up $60 million of that total. In late 2015, the companies agreed to co-commercialize the drug in the U.S.
Following news of the trial failure, ArQule stock plunged more than 18% to $1.20 per share in Friday morning trading.