Prometic has previously reported data from this pivotal Phase 2/3 trial, which showed that RyplazymTM treatment consistently replaced and maintained the plasminogen concentration at an appropriate level and that it resolved all lesions in all 10 patients treated for 12 weeks. These data fulfilled the clinical information required for the Biologics License Application (BLA) filing with the US Food and Drug Administration (FDA) for the Accelerated Regulatory Pathway Approval. Under the same pivotal Phase 2/3 protocol, these 10 patients have been treated for an additional 36 weeks, for a total drug exposure period of 48 weeks. In addition to the dossier filed with the FDA, the 48-week clinical efficacy data will form the basis for the upcoming regulatory filing with Health Canada in the fourth quarter of 2017.
"This pivotal Phase 2/3 trial of RyplazymTM has successfully met all primary and secondary endpoints, and we are pleased to announce these follow-up results which confirm the durability of the positive clinical efficacy and safety profile out to 48 weeks", stated John Moran, M.D., Chief Medical Officer of Prometic. "The 48-week clinical data will be submitted as a supplement to our BLA filing, after RyplazymTM receives its expected accelerated approval in the fourth quarter of 2017."
Pursuant to the BLA process, the FDA's inspection of Prometic's RyplazymTM manufacturing facility is scheduled to take place this summer. Also as part of the rolling BLA process, the Corporation is responding diligently to all FDA requests which to date are not related to underlying clinical data for RyplazymTM and will provide further updates on the exact expected timeline of regulatory approval as soon as available.
The RyplazymTM Phase 2/3 trial is still underway, and the dataset for all 15 patients that will have been treated over a period of 48 weeks is expected to be an acceptable demonstration of the efficacy of plasminogen therapy in patients with plasminogen congenital deficiency for full licensure, without the need for any additional clinical trials.
Prometic is also in discussions with the European Medicines Agency (EMA) with regards to the clinical information which would be required to secure regulatory approval of RyplazymTM in Europe and expects to be able to provide an update thereon in the fall of 2017.
Finally, Prometic has provided further data to the FDA to support its claims that the serious and life-threatening clinical manifestations associated with congenital plasminogen deficiency occurs predominantly in pediatric patients, as required to qualify for a rare pediatric disease designation.
Ryplazym Phase 2/3 Clinical Data Presented at ISTH Congress in Berlin
Clinical data for the 10 patients with 12 weeks of plasminogen treatment were featured in a poster session yesterday, July 10, at the International Society on Thrombosis and Hemostasis (ISTH) Congress, currently taking place in Berlin. To view a copy of the poster, refer to http://prometic.com/wp-content/uploads/2017/07/ISTH-plasminogen-phase-2-3-poster-final-submitted-19-Jun.pdf
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 Plasminogen Deficiency
The most common condition 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 corneal damage and blindness. Ligneous growths tend to recur after surgical excision, thereby requiring multiple surgeries.
While ligneous conjunctivitis is the best characterized lesion of plasminogen deficiency, hypoplasminogenemia is a multi-systemic 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 also been reported in children with severe hypoplasminogenemia, thought to be related to the deposition of fibrin in the cerebral ventricles.
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, Russia, Australia 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, our ability to develop, manufacture, and successfully commercialize value-added pharmaceutical products, obtaining regulatory approvals, the availability of funds and resources to pursue research and development projects, the successful and timely completion of clinical studies, our ability to take advantage of business opportunities in the pharmaceutical industry, our reliance on key personnel, collaborative partners and third parties, our patents and proprietary technology, our ability to access capital, the use of certain hazardous materials, the availability and sources of raw materials, our manufacturing capabilities, currency fluctuations, the value of our intangible assets, negative operating cash flow, legal proceedings, uncertainties related to the regulatory process, increased data security costs, costs related to environmental safety regulations, competition from existing treatments, as well as from current and future competitors, developing products for the indications we are targeting, market acceptance of our product candidates by patients, doctors and clinicians, including as it relates to the availability of third-party reimbursement, general changes in economic conditions and other risks related to our industry. 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 our 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.
SOURCE ProMetic Life Sciences Inc.
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