Staff Writer

The European Commission has granted full marketing authorization to EpiCept Corp.'s Ceplene for use in preventing relapse of acute myeloid leukemia (AML), one of the most deadly forms of the disease.

Ceplene, believed to be the first such therapy for preventing the recurrence of AML, will be available in 30 countries, including three outside the EU: Iceland, Liechtenstein and Norway.

EpiCept CEO Jack Talley called the drug "a medical breakthrough to help the prognosis of thousands of AML patients." The Tarrytown, N.J.-based company hopes to secure a European partner for Ceplene in early 2009, he told BioWorld Today.

The approval is based on a single Phase III trial showing that Ceplene, combined with interleukin-2 (Proleukin, Novartis) immunotherapy, prolonged the period of time in which adult patients remained leukemia-free.

In the majority of cases, AML patients achieved complete remission after treatment with chemotherapy. That is, they have no hematological evidence of the disease. However, about 50 percent of untreated AML patients relapse within a year, and that first relapse often ends in death, according to EpiCept.

There are some investigational drugs in Phase II testing that are being developed to prevent the relapse. But Talley said those drug candidates - Genzyme Corp.'s clofarabine and Wyeth's Mylotarg - are believed to be "considerably more toxic."

EpiCept also plans to seek Ceplene approval in the U.S. and Canada. The company hopes to use the same studies submitted to EU regulators to file for approval in the U.S. and Canada, Talley said. The product was studied internationally, including in North America.

A pre-new drug application submission meeting with Health Canada has been scheduled during the fourth quarter, and will be followed by a pre-new drug application meeting with the FDA in first-quarter 2009. Leukemia-free survival is an accepted study endpoint in leukemia studies, one that the FDA has experience evaluating, Talley said. Historically, products approved in Europe generally are approved in the U.S. and vice versa, he said. Though, there certainly is no guarantee of Ceplene's U.S. approval, he added.

Although EpiCept is seeking a marketing partner in Europe, a U.S. partner may not be needed, Talley said. However, he said, that "doesn't mean we won't have one."

Several potential commercial partners with established sales presence in hematology and oncology in Europe have expressed interest in marketing Ceplene and are in various stages of due diligence, Talley said in a company statement. Further details on the company's commercialization strategy are expected later this year, the statement noted.

As an "orphan" product, Ceplene will have 10 years of marketing exclusivity in Europe. In addition, the company has control of the worldwide supply of the drug's main ingredient, histamine dihydrochloride. And it also holds the manufacturing purification patent through 2021.

Ceplene is not believed to be potent enough to work as a potential treatment for the disease itself, but it is expected to have broader application beyond AML as a possible prevention therapy in other indications. Those other indications will be explored in clinical trials that the company hopes to start in 2009, Talley said.

Likely targets for those studies include myelodysplastic syndrome, chronic myelogenous leukemia and multiple myeloma, which has a much larger potential patient population than the currently approved indication.

As part of its EU marketing authorization, EpiCept will perform two post-approval clinical studies of Ceplene. One will assess certain biomarkers in AML patients in first remission. The other study will assess the effect of Ceplene/IL-2 on the development of minimal residual disease in the same patient population. EpiCept is considering combining those studies into a single clinical trial.

AML is the most common type of leukemia in adults. There are about 40,000 AML patients in the EU, with 16,000 new cases occurring each year.

Shares in EpiCept (NASDAQ:EPCT) closed at 82 cents Thursday, up 7 cents, or 9.4 percent.