Protalex, Inc. (OTCQB:PRTX), a clinical-stage biopharmaceutical company, today announced that it has dosed the first patient in its U.S. Phase 1/2 study of PRTX-100 in adults with persistent/chronic Immune Thrombocytopenia (ITP) (PRTX-100-202 Study). PRTX-100, Protalex's lead drug candidate, is a highly purified form of Staphylococcal protein A (SpA), which was recently granted Orphan Drug Designation in the U.S. and in Europe for the treatment of ITP. PRTX-100 is also the subject of ongoing clinical development in Rheumatoid Arthritis (RA).
The 202 Study is an open-label, dose escalating study that can enroll up to 36 patients in as many as six cohorts. Each patient will receive four weekly intravenous doses of PRTX-100 and will be monitored for up to 48 weeks thereafter. The primary study endpoint of the 202 Study is a platelet response to PRTX-100. Secondary endpoints include safety, immunogenicity, and pharmacokinetics. Enrollment is currently taking place at two study sites in the U.S.
"We are delighted that patients are beginning to receive treatment in the U.S. with PRTX-100 in the 202 Study and look forward shortly to the initiation of treatment in the European based ITP study (PRTX-100-203 Study)," stated Richard J. Francovitch, Ph.D., Protalex's Vice President ITP Programs. "We are also pleased that results from pre-clinical studies of PRTX-100 as a treatment for ITP will be presented at the American Society of Hematology (ASH) meeting in Orlando in early December (Session 311. Disorders of Platelet Number or Function: Poster I, Saturday, December 5). These results from a sophisticated animal model of ITP demonstrated that PRTX-100 raises platelet counts by inhibiting processes that destroy platelets and support our enthusiasm for the Phase 1/2 studies of PRTX-100 in patients with ITP."
About Immune Thrombocytopenia (ITP)
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.
About PRTX-100
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 two most recently approved drugs used to treat ITP, Nplate® (romiplostin) and Promacta®/Revolade™ (eltrombopag) both increase the production of platelets but do not appear to affect the underlying platelet destruction process. The safety, tolerability and pharmacokinetics of PRTX-100 have been characterized in five 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.
