Protalex Awarded Grant from FDA Office of Orphan Products Development for PRTX-100

Protalex announced the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) has awarded the Company a $403,000 grant to support future clinical development activity of PRTX-100 as a treatment for Immune Thrombocytopenia (ITP). The goal of FDA's OOPD Orphan Products Clinical Trials Grants Program is to encourage the clinical development of new drugs for use in rare diseases or conditions where no current therapy exists, or where the candidate drug will be superior to the existing therapy.

PRTX-100 is a highly purified form of Staphylococcal protein A (SpA), which is an immunomodulatory protein known to modify aspects of the human immune system. PRTX-100 is a new generation immunomodulatory therapy and has been granted Orphan Drug Designation as a potential treatment for ITP in both the U.S. and Europe.

Protalex is currently enrolling patients into two Phase 1/2 dose-escalating studies of PRTX-100 as a potential new treatment for ITP at several sites in the U.S. (the 202 Study) and in Europe (the 203 Study) and has so far seen patients in each completed lower dose cohort achieve a protocol-defined platelet response.

"We are delighted to have been granted this funding from the FDA’s OOPD as it underscores the great need for innovative, effective treatments for this rare autoimmune disease, and recognizes the potential benefits that PRTX-100 may provide for patients with ITP," stated Richard J. Francovitch, Ph.D., Protalex’s Vice President, ITP Programs. "We recently opened new dose cohorts in both the 202 and 203 studies and look forward to the results as the studies move forward evaluating higher doses of PRTX-100."

ITP is an autoimmune-mediated condition characterized by bruising and increased bleeding as a result of immune-mediated accelerated destruction of platelets and impaired production of platelets. The diagnosis of ITP is based upon a low platelet count, usually less than 100,000 per microliter of blood, in the absence of other possible causes of reduced platelet numbers such as an underlying illness or medication.

PRTX-100, a new generation immunomodulatory therapy, is a highly purified form of SpA, an immunomodulatory protein known to modify aspects of the human immune system. PRTX-100 has the ability, at very low concentrations, to bind to human B-lymphocytes and macrophages and to modulate immune processes. Pre-clinical data indicate that PRTX-100 may have the potential to treat ITP by reducing the immune-mediated destruction of the platelets. The safety, tolerability and pharmacokinetics of PRTX-100 have been characterized in six clinical studies and was recently granted Orphan Drug Designation in the U.S. and Europe for the treatment of ITP. In two Phase 1b clinical trials in adult patients with active Rheumatoid Arthritis (RA), PRTX-100 was generally safe and well tolerated at all dose levels, and at certain higher doses more patients showed improvement in measures of RA disease activity than did patients at the lower dose or placebo cohorts. PRTX-100 is given as a short intravenous infusion.