Protalex Receives Orphan Drug Designation from FDA for PRTX-100 to Treat Immune Thrombocytopenia

FLORHAM PARK, N.J.-- Protalex, Inc. (OTCQB:PRTX), a clinical-stage biopharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development (OOPD) has granted Orphan Drug Designation to PRTX-100 for the treatment of immune thrombocytopenia (ITP). The OOPD is tasked with evaluating the scientific and clinical data submissions from sponsors to identify and designate products as promising for rare diseases and to further advance scientific development of such promising medical products. PRTX-100, Protalex’s lead product, is a highly purified form of Staphylococcal Protein A. The FDA previously accepted the Company’s Investigational New Drug (IND) application for a Phase I/II open-label, dose-escalating study of PRTX-100 in adults with persistent/chronic ITP. Protalex expects to enroll its first patient in an ITP study in the third quarter of 2015.

Orphan drug designation provides certain exclusivity benefits, tax credits for certain research and a waiver of the New Drug Application user fee. ITP is recognized by the FDA as an orphan disease, usually defined as a condition that affects fewer than 200,000 people nationwide.

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.

“The FDA’s timely approval of our request for orphan drug designation for PRTX-100 to treat ITP is a key milestone that supports our broader strategy of bringing this potentially life-saving therapy to patients with ITP,” stated William E. Gannon, M.D., Chief Medical Officer of Protalex. “ITP is a chronic, debilitating disease. There are patients who do not respond to current treatment options or cannot maintain an increased platelet count requiring ongoing or additional treatment. Patients may also experience adverse events or are unable to tolerate current therapies, limiting their ability to continue treatment. So there is a need for new agents that treat the underlying mechanisms of ITP and provide effective, safe and well tolerated treatments that are also convenient for patients with ITP. We look forward to enrolling patients in our Phase I/II clinical trials of PRTX-100 to treat patients with ITP and expect to have top-line data in 2016.”

The two most recently approved drugs used to treat ITP, Nplate® (romiplostin) and Promacta®(eltrombopag) both increase the production of platelets but do not appear to affect the underlying platelet destruction process. In contrast, pre-clinical data indicate that PRTX-100 may have the potential to treat ITP by reducing the immune-mediated destruction of the platelets. Furthermore, PRTX-100 has established an acceptable safety profile based on data from patients treated in five clinical studies including patients with Rheumatoid Arthritis (RA), another autoimmune disease.

About Protalex, Inc.

Protalex, Inc. is a clinical-stage biopharmaceutical company focused on the development of a class of drugs for treating autoimmune and inflammatory diseases including Rheumatoid Arthritis (RA) and Immune Thrombocytopenia (ITP). Protalex’s lead product, PRTX-100, is a formulation of a proprietary, highly purified form of Staphylococcal Protein A, which is an immunomodulatory protein produced by bacteria. PRTX-100 has the ability, at very low concentrations, to bind to human B-lymphocytes and macrophages and to modulate immune processes. The safety, tolerability and pharmacokinetics of PRTX-100 have been characterized in five clinical studies. In two Phase 1b clinical trials in adult patients with active 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.

Forward-Looking Statements

Statements in this press release that are not statements of historical or current fact constitute "forward-looking statements." Such forward-looking statements involve known and unknown risks, uncertainties and other unknown factors that could cause the Company's actual operating or clinical results to be materially different from any historical results or from any future results expressed or implied by such forward-looking statements. In addition to statements that explicitly describe these risks and uncertainties, readers are urged to consider statements that contain terms such as "believes," "belief," "expects," "expect," "intends," "intend," "anticipate," "anticipates," "plans," "plan," to be uncertain and forward-looking. The forward-looking statements contained herein are also subject generally to other risks and uncertainties that are described from time to time in the Company's filings with Securities and Exchange Commission.

LHA
Anne Marie Fields, 212-838-3777
afields@lhai.com

Source: Protalex, Inc.