ActivBiotics Receives FDA Orphan Drug Designation for Superoxide Dismutase Mimetic, M40403, in Oral Mucositis of Cancer

Thursday, March 6, 2008 General News
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WELLESLEY HILLS, Mass., March 6 Joseph F. Finn, Jr., CPA,the Assignee for the Benefit of Creditors of ActivBiotics, announced todaythat the Office of Orphan Products Development of the Food and DrugAdministration (FDA) has granted Orphan Drug Designation for M40403 for theprevention of radiation -- or chemotherapy -- induced oral mucositis (OM) incancer patients.

The development of oral mucositis is a major, debilitating side-effect inthe radiation and chemotherapy of cancer. Oral mucositis is one of the mainreasons for poor compliance with and early discontinuation of therapy bypatients being treated for their condition. Nearly half a million patientsdevelop oral mucositis each year across all cancer indications, and of these90% are associated with solid tumors. Although the estimated potential of thetotal oral mucositis market is $1- 2 billion, this therapeutic area is one ofhigh unmet medical need, with few successful treatment options available.

By targeting various early stage inflammatory pathways implicated in thedevelopment of oral mucositis, M40403 may have particularly unique potentialas a successful therapeutic in this disorder.

FDA's Orphan Drug Act was designed to encourage the development ofproducts that demonstrate promise for the diagnosis, prevention and/ortreatment of life-threatening or very serious conditions that are rare andaffect not more than 200,000 persons in the United States. Orphan DrugDesignation provides an important economic incentive for the development ofnew products in the cancer field. US Orphan Designation provides for 7 yearsof market exclusivity, reduction regulatory fees, and additional regulatorysupport for R&D initiatives.

About M40403

This investigational small molecule mimics the action of a naturalcomponent of the cells, an enzyme called superoxide dismutase. Superoxidedismutase is thought to have a double pharmacologic effect during the earlystages of tissue damage, first through the reduction of inflammation, andsecondarily through the decreased rate of programmed cell death (apoptosis).As mucositis involves inflammation, the positive effect of the M40403 overthis process is expected to have a beneficial effect in the condition. M40403has shown efficacy in an animal model where oral mucositis was induced byacute irradiation. The compound has been evaluated in approximately 700subjects in several Phase I and II clinical trials, and was shown to be safeand well-tolerated.

About Oral Mucositis

Oral mucositis is a side effect experienced by 20-30% of cancer patientsundergoing chemotherapy or radiation therapy. Patients receiving radiationtherapy are at particular risk of developing oral mucositis, with essentiallyall cases developing oral mucositis. In addition to killing tumor cells,radiation therapy also adversely affects surrounding tissues including theinner lining (mucosa) of the mouth. The resulting "oral mucositis;" is thenumber one patient-reported debilitating side effect of cancer therapy. Thecondition is debilitating, resulting in ulcers, severe pain, dryness of themouth, and inability to swallow. Patients with severe oral mucositis andreduced immunity, such as cancer patients receiving radio and chemotherapy,frequently develop serious opportunistic infections leading to furthercomplications in patient care. Oral mucositis often leads to breaks inantineoplastic therapy, reducing the effectiveness of cancer treatment. Insevere cases, patients will also need parenteral nutrition, and may requireopiates for pain control of the mucositis. Currently, there are nosatisfactory methods for the treatment of oral mucositis in patients withsolid tumors in the US.

About ActivBiotics

ActivBiotics is currently in the process of selling its proprietaryassets, including M40403, through an Assignment for the Benefit of Creditors.The bidding for

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