LAVAL, QUEBEC, CANADA, ROCKVILLE, MARYLAND, USA and TAIPEI, TAIWAN – March 14, 2013 – ProMetic Life Sciences Inc. (TSX: PLI) ("ProMetic" or the "Corporation") announced today that an orphan drug designation status has been granted for its plasma purified human plasminogen drug to ProMetic BioTherapeutics, Inc., its US based subsidiary, by the American Food and Drug Administration ("FDA"). The orphan drug designation is for the treatment of hypoplasminogenemia, or type I plasminogen deficiency ("T1PD").
ProMetic is currently developing a systemic formulation for the treatment of T1PD in partnership with Hematech Biotherapeutics Inc. ("Hematech") and has already successfully scaled up the manufacturing process.
T1PD is a rare autosomal recessive disorder that leads to severe clinical manifestations, primarily associated with fibrous depositions on mucous membranes throughout the body. It is a multisystem disease that affects the eyes, ears, sinuses, tracheobronchial tree, genitourinary tract and gums. There is no effective replacement therapy currently available for this disease.
"The conditions resulting from the deficiency of plasminogen require that several medical specialties combine their efforts to provide care to these patients", stated Dr Tom Chen, Senior Vice President, Product & Asian Market Development. "We are very proud to bring this tangible therapeutic solution enabled by our proprietary manufacturing platform and our strategic partner, Hematech Biotherapeutics", added Dr Chen.
"Given the multitude of organs and tissues that are affected by such type of disorders, it is critical to be able to provide a safe and reliable source of plasminogen for these patients in need", stated Pierre Laurin, CEO of ProMetic. "The scale-up of our proven manufacturing platform at our Laval, Quebec facility will enable the global supply of plasminogen as well as several other therapeutics targeting serious unmet medical conditions".
Orphan Drug Designation is granted to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven year period of U.S. marketing exclusivity upon marketing approval for the designated indication, as well as with tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and the waiver of prescription drug user fees.
Plasminogen is a naturally occurring protein that is synthesized by the liver and circulates in the blood. Activated plasminogen, plasmin, is an enzymatic component of the fibrinolytic system and is the main enzyme involved in the lysis of clots and clearance of extravasated fibrin. Plasminogen is therefore involved in wound healing, cell migration, tissue remodeling, angiogenesis and embryogenesis.
About Hypoplasminogenemia or Type 1 Plasminogen Deficiency (T1PD):
One of the most well-defined conditions associated with plasminogen deficiency is ligneous conjunctivitis, which is characterized by thick, woody (ligneous) growths on the conjunctiva of the eye, and if left untreated, can lead to blindness. Most affected cases are infants and children showing their first clinical manifestation at a median age of approximately 10 months.
While ligneous conjunctivitis is the most well characterized lesion of plasminogen deficiency, hypoplasminogenemia is actually a multisystem disease that can also affect the ears, sinuses, tracheobronchial tree, genitourinary tract, and gingiva. Tracheobronchial lesions including hyperviscous secretions can result in respiratory failure. Hydrocephalus has occurred in approximately 10% of children with severe hypoplasminogenemia, apparently related to the deposition of fibrin in the cerebral ventricular system; treatment may require implantation of a shunt which can be complicated by repeated occlusions, and which can lead to death (Schott, 1998). Severely impaired skin wound healing has also been reported (Schott, 1998). Other organ systems affected by T1PD with less frequency include the gastrointestinal tract, renal collecting system and a malformation of the cerebellar vermis called the Dandy-Walker malformation (Schott, 1998).
About ProMetic Life Sciences Inc.
ProMetic Life Sciences Inc. (http://prometic.com/) is a global biopharmaceutical company specializing in the design of small molecules that mimic unique and specific interactions between proteins, research, development, manufacture and marketing of a variety of commercial applications derived from its proprietary Mimetic LigandTM technology. This technology is used in large-scale purification of biologics, drug development, proteomics and the elimination of pathogens. ProMetic is also active in developing its own novel therapeutic products targeting unmet medical needs in the field of fibrosis, anemia, neutropenia, cancer and autoimmune diseases/inflammation as well as certain nephropathies Headquartered in Laval (Canada), ProMetic has R&D facilities in the UK, the U.S. and Canada, manufacturing facilities in the UK and business development activities in the U.S., Europe, Asia.
Forward Looking Statements
This press release contains forward-looking statements about ProMetic's objectives, strategies and businesses that involve risks and uncertainties. These statements are "forward-looking" because they are based on our current expectations about the markets we operate in and on various estimates and assumptions. Actual events or results may differ materially from those anticipated in these forward-looking statements if known or unknown risks affect our business, or if our estimates or assumptions turn out to be inaccurate. Such risks and assumptions include, but are not limited to, ProMetic's ability to develop, manufacture, and successfully commercialize value-added pharmaceutical products, the availability of funds and resources to pursue R&D projects, the successful and timely completion of clinical studies, the ability of ProMetic to take advantage of business opportunities in the pharmaceutical industry, uncertainties related to the regulatory process and general changes in economic conditions. You will find a more detailed assessment of the risks that could cause actual events or results to materially differ from our current expectations on page 24 of ProMetic's Annual Information Form for the year ended December 31, 2011, under the heading "Risk and Uncertainties related to ProMetic's business". As a result, we cannot guarantee that any forward-looking statement will materialize. We assume no obligation to update any forward-looking statement even if new information becomes available, as a result of future events or for any other reason, unless required by applicable securities laws and regulations. All amounts are in Canadian dollars unless indicated otherwise.