EntreMed Inc announced that its new derivative of thalidomide, ENMD 0995, received Orphan Drug designation from the Food and Drug Administration (FDA) for the treatment of patients with multiple myeloma. Multiple myeloma, the second most prevalent form of blood cancer among Americans, causes 11,000 deaths annually. FDA Orphan Drug designation encourages the development of therapeutics to treat diseases affecting fewer than 200,000 Americans by providing tax credits and marketing exclusivity incentives to companies.

In animal studies, ENMD 0995 exhibited significant activity in primary and in metastatic human tumor models of multiple myeloma and was more effective than thalidomide, a drug now widely used for the investigational treatment of multiple myeloma.

Preclinical investigators reported that the progression of metastatic multiple myeloma disease was inhibited markedly in ENMD 0995-treated animals when compared with thalidomide-treated and control groups. Survival of the animals was significantly prolonged in the ENMD 0995 treated group. ENMD 0995's ability to inhibit angiogenesis and B-cell specific proliferation may be key to its success against multiple myeloma.

Dr. John Holaday, Chairman and Chief Scientific Officer, commented, "Building on our previous in-house research and development work with thalidomide as a possible cancer treatment, EntreMed scientists have successfully identified a new version of thalidomide that is more active and shows no signs of the toxic side effects associated with thalidomide in preclinical models. Receiving Orphan Drug status from the FDA reinforces the strength of EntreMed's scientific team and furthers our strategy of the successful and timely clinical development of ENMD 0995." Dr. Holaday continued, "This designation also increases our hope that ENMD 0995 will be a better treatment over therapies currently available to multiple myeloma patients."

The Company will also develop other thalidomide analogs to treat cancer and other diseases.EntreMed has an orphan drug grant application pending with the Food and Drug Administration (FDA) to support the clinical costs associated with future ENMD 0995 clinical trials. In September, the FDA awarded EntreMed an orphan drug grant in the amount of $300,000 for three consecutive years to support the Company's Phase II Endostatin trial for neuroendocrine tumor patients. The FDA orphan drug grant program funds clinical research to accelerate the development of products that demonstrate promise to treat such rare diseases.

ENMD 0995 is a small molecule analog of thalidomide with improved angiogenesis inhibitor activity that in animal models does not show evidence of the toxic side effects reported for thalidomide. ENMD 0995 is the 3-amino derivative of thalidomide with two mirror-image forms known as the S(-) enantiomer and the R(+) enantiomer. EntreMed scientists demonstrated in a number of animal models that the S(-) enantiomer form is more active than both the R(+) enantiomer form and the R,S mixture (racemate). Based on those data, EntreMed has chosen to move the S(-) enantiomer - designated as ENMD 0995 - towards the clinic.

EntreMed scientists first identified that ENMD 0995 has not only angiogenesis-inhibitory activity, but also B-cell specific anti-proliferative activity. Therefore, ENMD 0995 may be an ideal candidate for the treatment of B-cell tumors such as multiple myeloma and some lymphomas, as well as some solid tumors.

Multiple myeloma is the second most prevalent blood cancer and represents approximately 1% of all cancers and 2% of all cancer deaths. More than 40,000 Americans currently have multiple myeloma, including former Congresswoman Geraldine Ferraro. Over 14,000 new patients are diagnosed each year, with median survival ranging from 3 to 4.5 years. The cancer is twice as common in males as females. African Americans and Native Pacific Islanders have the highest reported incidence of this disease. Among African Americans, multiple myeloma is one of the top ten leading causes of cancer death.