Daiichi, ArQule’s struggling tivantinib fails in phase 3 liver cancer test

After fluffing its lines in two mid-to-late stage trials in lung and colorectal cancer over the past few years, Daiichi and ArQule had hoped that tivantinib could produce the goods in a phase 3 liver cancer test, but it was not to be.

In data released this morning, the partners said their MET-inhibitor med did not meet its primary endpoint of improving overall survival in hepatocellular carcinoma (the most common form of liver cancer) in 340 patients.

The trial, dubbed METIV-HCC, was set up as a biomarker-selected, double-blind, placebo-controlled, phase 3 study that looked at tivantinib against best supportive care in patients with MET-overexpressing, inoperable HCC who cannot take, or were previously treated, with systemic therapy.

The primary endpoint of the study was overall survival, but the pair said in a brief update that it did not meet this target, although detailed data on how much is missed by were not published in the release.

Secondary endpoints included progression-free survival and safety, but again no data were given. The pair said more information will be shared “an upcoming scientific forum.”

“HCC is a disease with high unmet need, especially in the second-line setting, so these results are disappointing for the patients as well as the investigators and the companies,” said Paolo Pucci, CEO of ArQule.

“Despite the negative outcome of this study, we remain committed to applying rigorous science to unmet needs for patients with cancer,” added Antoine Yver, MD, EVP and global head of oncology R&D at Daiichi Sankyo.

The company has seen a string of negative results for this drug, but there was a ray of light in late 2014, when it posted a positive outcome in a phase 2 metastatic prostate cancer study after hitting the primary endpoint for progression-free survival compared to a placebo.

But this has not helped relieve pressure from the biotech, and this latest setback will only ramp up the questions on the drug’s clinical future across the board. The biotech's shares were down more than 4% yesterday, before the news, at less than $1.50 a share and with a market cap of $111 million. 

Daiichi, which trades in Japan, was down 0.5% today on the data. The company has been swinging the R&D ax of late, cutting down research sites in Japan, India, the U.K. and Germany, but has also been keen to pen new cancer R&D pacts, most recently with Kite Pharma and its potential first-to-market CAR-T candidate.