EpiCept Corporation, an emerging specialty pharmaceutical company, has submitted a Market Authorization Application (MAA) to the European Medicines Agency for the Evaluation of Medicinal Products (EMEA) for Ceplene (histamine dihydrochloride), the company's lead oncology product candidate, administered in conjunction with interleukin-2 (IL-2), for the maintenance of first remission in patients with Acute Myeloid Leukaemia (AML).

"The filing of an MAA for Ceplene represents a critical step forward in our efforts to fill a specific void of available drug treatment options for AML patients," stated Jack Talley, President and chief executive officer, EpiCept Corporation. "We look forward to working with the European regulatory authorities to obtain a positive opinion for the approval of this important product candidate."

The MAA submission for Ceplene will be reviewed under the EU centralized procedure, and if approved, would provide a marketing authorization valid in all EU member states. The European Commission has previously granted orphan drug status to Ceplene for use in the treatment of AML.

The pivotal efficacy and safety data for this MAA submission is from a Phase III clinical trial for Ceplene in conjunction with interleukin-2. This study met its primary endpoint of preventing relapse as shown by increased leukaemia-free survival for AML patients in remission. The study was conducted in eleven countries and included 320 randomized patients. The data demonstrated that patients with AML in complete remission who received 18 months of treatment with Ceplene plus low dose interleukin-2 experienced a significantly improved leukaemia-free survival compared to the current standard of care, which is no treatment after successful induction of remission. The improvement in leukaemia-free survival achieved by Ceplene/IL-2 was highly statistically significant (p=0.0096, analyzed according to Intent-to-Treat).

Even more striking was the benefit observed in patients in their first remission (CR1). In this subgroup, the patients had a 55% improvement in leukaemia free survival. This represented an absolute improvement of more than 22 weeks in terms of delayed progression of the disease. This benefit was also highly statistically significant, (p=0.0113) and is the intended patient population (CR1) under consideration for this application. The results of this trial were published in Blood, a leading scientific journal in haematology, (Blood; The Journal of the American Society of Haematology, volume 108, number 1, July 1, 2006).

Professor Kristoffer Hellstrand, M.D., Ph.D., Sahlgrenska University Hospital, Goteborg, Sweden, an author of the Blood article and responsible for the design of the Phase III study of Ceplene, remarked, "There is a distinctive need for new treatment options to improve long-term leukaemia free survival among AML patients. The majority of AML patients in complete remission will experience a relapse of leukaemia with a progressively poor prognosis. These study results indicate that Ceplene, combined with low dose interleukin-2, significantly improves long-term leukaemia-free survival among these patients."

EpiCept retains full marketing rights for Ceplene worldwide. Talley commented; "EpiCept's strategy is to concentrate its future marketing efforts in the US market. Consistent with that strategy, we anticipate selecting a partner to market and sell Ceplene in Europe following regulatory approval. In this regard, we will continue our discussions with prospective partners who have appropriate European distribution capabilities with a view towards identifying a partner for Ceplene next year. At a comparable cost of therapy relative to other cancer treatments, the market opportunity for Ceplene for this use alone in the European Union could exceed $300 million annually."

EpiCept's proprietary apoptosis screening technology can efficiently identify new cancer drug candidates and molecular targets that selectively induce apoptosis in cancer cells through the use of chemical genetics and its proprietary live cell high-throughput caspase-3 screening technology. Chemical genetics is a research approach investigating the effect of small molecule drug candidates based on the cellular activity of a protein, enabling researchers to determine the protein's function. With the combination of chemical genetics and caspase-3 screening, EpiCept's researchers identify and test the effect of small molecules on pathways and molecular targets crucial to apoptosis and gain insights into their potential as new anticancer agents. The Company's screening technology is particularly versatile and can be adapted for many cell types that can be cultured, and it can measure caspase activation inside multiple cell types (e.g. cancer cells, immune cells, or cell lines from different organ systems or genetically engineered cells). This allows researchers to find potential drug candidates that are selective for specific cancer types, which may help identify candidates that provide increased therapeutic benefit and reduced toxicity.