Hepatocellular, or liver cancer, is one of the most common tumors in the world, and there are currently no satisfactory treatments available. In the U.S., there are approximately 17,000 new cases per year providing the opportunity for orphan drug status. Adherex has already received orphan drug designation from the U.S. Food and Drug Administration for the use of eniluracil in combination with fluoropyrimidines (including 5-FU) to treat liver cancer. Our rationale for targeting liver cancer as our lead indication includes: 1) two Phase II studies previously conducted by GSK in liver cancer showed extended periods of stable disease and survival, even though those studies were conducted using what we now know to be a suboptimal dose and schedule, and 2) liver cancer has naturally high levels of DPD, making it resistant to treatment with 5-FU. Eniluracil irreversibly inhibits DPD and, therefore, may make liver cancer susceptible to therapy with 5-FU.
