Liver cancer miss adds to litany of trial failures for ArQule's tivantinib

Biotech is now pushing its FGFR inhibitor, heading into phase 3

ArQule's c-MET inhibitor tivantinib has failed yet another late-stage trial, drawing a line under the program as the biotech tries to move beyond what CEO Paolo Pucci has called the company's "tivantinib cycle."

The serially failing drug missed its primary target in the phase 3 JET-HCC  trial run by Japanese licensee Kyowa Hakko involving patients with a form of liver cancer known as hepatocellular carcinoma HCC) who had previously been treated with Bayer's Nexavar (sorafenib), Arqule confirmed in a statement.

Tivantinib failed to improve progression-free survival (PFS) in the trial, which is not a particularly surprising result given that last month ArQule and partner Daiichi Sankyo reported similarly disappointing results from the METIV-HCC study in the same form of liver cancer. In that case, tivantinib failed to show an improvement in overall survival, with the result wiping a fifth off of ArQule's share price on the day.


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At last count, tivantinib had already been discontinued for gastric cancer, non-small cell lung cancer, pancreatic cancer, soft tissue sarcoma and germ cell/embryonal neoplasms, and according to ArQule's website HCC was the final indication being sought for the drug. Shares in the company were on the slide again pre-market today, suggesting some investors at least were hoping for some good news on the program.

After the METIV-HCC trial missed the mark ArQule said it would take some time to go through the entire dataset and try to work out why the drug did so well in phase 2 but failed in larger trials. The company already seemed keen to move onto more enticing prospects, however, spending little time discussing tivantinib on a conference call with investors. Instead, Pucci stressed that the company is moving into a "new cycle that's going to be underpinned by [our] proprietary pipeline."

With tivantinib being read the last rites, ArQule's next most advanced project is ARQ 087, a fibroblast growth factor receptor (FGFR) inhibitor that is in a phase 2 trial for intrahepatic cholangiocarcinoma (ICCA) or bile duct cancer—the second most common primary liver cancer after HCC—as well as early-stage testing in other solid tumors.

A phase 3 trial in ICCA is due to get underway later this year, with Pucci describing the indications as a "small but fast-to-market" opportunity that could be followed up by trials in more common cancers characterized by FGFR mutations. That list includes bladder, lung, breast, thyroid, oral, and prostate cancers.

Also in the pipe are a pan-AKT inhibitor code-named ARQ 092 in phase 1 testing for cancers and Proteus syndrome and NQO1 inhibitor ARQ 761 in phase 2 for pancreatic cancer and phase 1 for various solid tumors.

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