EpiCept announces opinion by European Regulatory Authority on Ceplene

EpiCept Corporation has announced that the European Committee for Medicinal Products for Human Use (CHMP), the scientific committee of the European Medicines Agency (EMEA), has issued, as expected following the trend vote announced last month, a negative opinion regarding the marketing authorization application (MAA) for Ceplene (histamine dihydrochloride) for the remission maintenance and prevention of relapse of patients with Acute Myeloid Leukemia (AML) in first remission.

Ceplene is designated as an orphan medicinal product in the European Union with respect to this indication.

In reaching this opinion, the CHMP stated that further data are required to support the results from EpiCept ' s Phase III clinical study of Ceplene. The CHMP indicated that data from the single pivotal AML trial was not adequately statistically compelling. Specifically, the CHMP seeks additional mechanistic data on Ceplene to elucidate further the pharmacological rationale for the proposed use of Ceplene ® in conjunction with interleukin-2. The company believes that sufficient pre-clinical mechanistic data has already been supplied to the CHMP. EpiCept also believes that additional mechanistic clinical data could be generated post-approval. Importantly, based on input from the CHMP, EpiCept believes we will be able to obtain approval without having to conduct an additional confirmatory Phase III trial.

There were no major safety concerns with the application. A divergent opinion from the co-rapporteur indicated that the results of the pivotal study were sufficiently robust to support full marketing authorization in part due to the public health need for AML treatment.

In accordance with the rules governing the European Centralized procedure, EpiCept has already requested a re-examination of this opinion through the appeal procedure. No formal decision will be taken by the European Commission, as the European licensing authority, until the appeal procedure has been completed. EpiCept intends to submit a document setting out the “ Detailed Grounds for Re-Examination ” within 60 days of receipt of the negative opinion and expects its appeal proceeding to take place in the third quarter of this year.

“ While not surprising in light of the negative trend vote announced last month, we are nonetheless disappointed at the opinion reached by the CHMP, ” remarked Jack Talley, President and CEO of EpiCept. “ We believe the strength of the clinical data provided in our application has amply demonstrated the benefits of Ceplene in prolonging leukemia free survival and preventing relapse in AML patients when used in conjunction with low-dose interleukin-2 (IL-2), the only therapy ever to produce this degree of therapeutic benefit. Ceplene ' s Phase III clinical trial results showed that it achieved a greater than 50 percent improvement in long-term prognosis, in essence conferring in excess of an extra year of life to these seriously-ill patients. We remain optimistic that we will be successful in the re-examination of our application and we will continue to be steadfast in our efforts to bring this desperately needed therapy to AML patients in Europe. ”

About Acute Myeloid Leukemia (AML)

AML is the most common type of leukemia in adults. There are approximately 40,000 AML patients in the EU, with 14,000 new cases occurring each year. Once diagnosed with AML, patients are typically treated with induction chemotherapy and consolidation therapy, with the majority achieving complete remission. However, about 75-80% of patients who achieve first remission will relapse, with the median time in remission before relapse being only 12 months with current treatments. Less than 5% of relapsed patients survive long term.

About Ceplene

Ceplene is EpiCept's registration-stage compound for the treatment of AML. Ceplene is designed to protect lymphocytes responsible for immune-mediated destruction of residual leukemic cells. Laboratory research has demonstrated that Ceplene reduces formation of oxygen radicals from phagocytes, inhibiting NADPH oxidase and protecting IL-2-activated NK-cells and T-cells.

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