- Ryplazim™ is Prometic’s Second Orphan Drug Designation (ODD) Granted for IPF by the FDA
- Two Late Stage Clinical Assets targeting IPF: Ryplazim™ under BLA review and PBI-4050 entering pivotal Phase 2/3 clinical trials.
LAVAL, QUEBEC, CANADA – Dec. 18, 2017 – Prometic Life Sciences Inc. (TSX: PLI) (OTCQX: PFSCF) (Prometic) announced today that an orphan drug designation status has been granted to its Plasminogen (Ryplazim™) for the treatment of idiopathic pulmonary fibrosis (“IPF”) by the US Food and Drug Administration (FDA).
In a gold-standard animal model proven to emulate pulmonary fibrosis in humans, Prometic’s Plasminogen (Ryplazim™) performed favorably compared to recently- approved IPF drugs to treat this condition. Plasminogen (Ryplazim™) significantly reduced tissue scarring in the lungs that was observed in non-treated animals, indicating the potential for providing clinically significant improvement and stabilization in lung function.
“The fibrinolytic systems play a central role in wound healing and tissue repair, a process believed to be abnormal within the IPF affected lung,” explained Dr. John Moran, Chief Medical Officer of Prometic. “Animal models of pulmonary fibrosis have demonstrated an imbalance between thrombosis and fibrinolysis within the alveolar compartment, a finding that is also observed in IPF patients. We plan to evaluate whether Plasminogen (Ryplazim™) can help lung function of IPF patients during acute exacerbation episodes which would be both complementary to anti-fibrotic chronic therapy and addressing an unmet medical need in the IPF patient population.”
Pierre Laurin, President and Chief Executive Officer of Prometic, commented: “We are pleased to have secured a second IPF orphan drug designation from the FDA with our Plasminogen, Ryplazim™, for the treatment of this devastating disease following the initial orphan drug designation received for PBI-4050, our small molecule. This designation supports our decision to aggressively pursue the development of Plasminogen in additional acute care medical conditions where the healing and fibrinolysis process is impaired.”
Orphan Drug Designation is granted to 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.
More about Idiopathic Pulmonary Fibrosis (IPF) and Acute Exacerbation
Idiopathic pulmonary fibrosis (IPF) is a chronic, devastating, and ultimately fatal disease characterized by a progressive decline in lung function. It is a specific type of interstitial lung disease in which the small air sacs of the lung, the “alveoli,” gradually become replaced by fibrotic (scar) tissue and is the cause of worsening dyspnea (shortness of breath). IPF is usually associated with a poor prognosis. The term “idiopathic” is used because the cause of pulmonary fibrosis is still unknown. IPF usually occurs in adult individuals of between 50 and 70 years of age, particularly those with a history of cigarette smoking, and affects men more often than women. IPF affects about 130,000 people in the United States, with about 48,000 new cases diagnosed annually. Approximately 40,000 people with IPF die each year, a similar number of deaths to those due to breast cancer. The 5-year mortality rate for patients with IPF is estimated to range from 50% to 70%. Acute exacerbation of IPF (AE-IPF) is defined as a sudden acceleration of the disease or an idiopathic acute injury superimposed on diseased lung that leads to a significant decline in lung function. An AE-IPF is associated with a mortality rate as high as 85% with mean survival periods of a few months.
Plasminogen is a naturally occurring protein that is synthesized by the liver and circulates in the blood. Activated plasminogen, plasmin, is a fundamental component of the fibrinolytic system and is the main enzyme involved in the lysis of blood clots and clearance of extravasated fibrin. Plasminogen is therefore vital in wound healing, cell migration, tissue remodeling, angiogenesis and embryogenesis.
About Prometic Life Sciences Inc.
Prometic Life Sciences Inc. (www.prometic.com) is a long-established biopharmaceutical company with globally recognized expertise in bioseparations, plasma-derived therapeutics and small-molecule drug development. Prometic is active in developing its own novel small molecule therapeutic products targeting unmet medical needs in the field of fibrosis, cancer and autoimmune diseases/inflammation. A number of plasma-derived and small molecule products are under development for orphan drug indications. Prometic also offers its state-of-the-art technologies for large-scale purification of biologics, drug development, proteomics and the elimination of pathogens to a growing base of industry leaders and uses its own affinity technology that provides for highly efficient extraction and purification of therapeutic proteins from human plasma in order to develop best-in-class therapeutics and orphan drugs. Headquartered in Laval (Canada), Prometic has R&D facilities in the UK, the U.S. and Canada, manufacturing facilities in the UK and commercial activities in the U.S., Canada, Europe and 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 in Prometic’s Annual Information Form for the year ended December 31, 2016, 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.