Eximias Pharmaceutical Corp.'s lead product, Thymitaq, missed its primary endpoint in a pivotal study evaluating its survival benefit in comparison with doxorubicin in inoperable primary liver cancer.
The two-arm, randomized ETHECC (Evaluation of Thymitaq [nolatrexed] in Hepatocellular Carcinoma) study completed enrollment in April at sites in North America, Europe and South Africa. Although detailed results from the 446-patient study were not released, the Berwyn, Pa.-based company said it intends to conduct additional analyses to determine future activities.
Eximias did not immediately return calls seeking comment.
Thymitaq's Phase III failure could spell trouble for the privately held company, which has only two products in its development pipeline: the intravenously administered Thymitaq and an oral version of nolatrexed called Orataq that is in early clinical development for the treatment of lung, head and neck, rectal, colon and pancreatic cancers. The drugs are designed to act as direct inhibitors of the thymidylate synthase enzyme, which is needed for the normal production of DNA.
Eximias, which licensed Thymitaq from La Jolla, Calif.-based Agouron Pharmaceuticals Inc. in 1998, has raised $94 million to date, including an April 2004 private round that brought in $63.5 million specifically for the Phase III trials. At the time of that financing, the company said it had planned to build its own U.S. sales force, while partnering Thymitaq for markets outside of the U.S. In 2002, the company agreed to license rights for the sale of the drug in Korea with LG Life Sciences, a company headquartered in Seoul.
Eximias had estimated that about 19,000 new cases of liver cancer would be diagnosed in the U.S. last year, with worldwide figures reaching upward of 500,000. As one of the most advanced products in development for liver cancer, for which there is no FDA-approved standard of care, Thymitaq was granted orphan status in both the U.S. and Europe, and fast-track status in the U.S.
In addition to liver cancer, Thymitaq also has been studied in colon, lung, liver, head and neck and pancreatic cancers.
Eximias named a new president and CEO in February. Gail Schulze replaced Elizabeth Corsi, who had been with the company since 1998 and had served as president since 2001.