- Severe clinical manifestations (including acute lung complications) significantly improved in a 20-month old infant
- Recommended plasminogen dose regimen provided efficacious clinical result
LAVAL, QUEBEC, CANADA – November 4, 2015 – ProMetic Life Sciences Inc. (TSX: PLI) (OTCQX: PFSCF)
(“ProMetic” or the “Corporation”) announced today that its plasma-derived plasminogen replacement therapy, currently in a Phase 1 clinical trial in the USA, has been successfully used to treat a plasminogen-deficient infant in critical condition in an intensive care unit at the Altona Children’s Hospital in Hamburg, Germany. The plasminogen was administered by a team from the Department of Pediatric Haematology and Oncology at the University Medical Center, Hamburg-Eppendorf, under the direction of Professor Reinhard Schneppenheim.
Diagnosed with severe plasminogen deficiency at 4 weeks of age, the patient had experienced numerous medical complications, ultimately leading to respiratory failure requiring ventilatory and circulatory support in the ICU. Administration of plasma was not successful in raising his plasminogen to an effective level. ProMetic’s plasminogen was provided under the Named Patient Program, a special patient access program in Germany which enables physicians, healthcare regulators and manufacturers to coordinate the provision of therapeutics that are not yet commercially available.
Professor Schneppenheim commented: “This young patient was in a very critical condition. The plasminogen and the protocol for its use recommended by ProMetic enabled us to quickly reach an efficacious concentration of plasminogen in the blood. Within a few days we observed reduction of the lesions, and after six weeks of therapy the lesions have markedly improved”.
“This case provides a clear demonstration of the efficacy of our plasminogen product in a very serious clinical situation. Within a few days the patient was able to breathe without ventilatory support”, said Dr. John Moran, ProMetic’s Chief Medical Officer, who added, “We are very pleased to have been able to provide the plasminogen therapy and to share our clinical insights with Professor Schneppenheim and his team, and in doing so, supporting their efforts to save this profoundly ill child”.
ProMetic is currently developing its plasminogen therapy for the management of patients with plasminogen deficiency and has received orphan drug designation from both the FDA and EMA. Pierre Laurin, ProMetic’s President and Chief Executive Officer commented: “Our mission is to bring to patients life-saving therapeutics such as plasminogen. There is nothing more gratifying than to be part of such a remarkable turnaround situation with a critically-ill patient. I am proud of our team’s accomplishment and congratulate the medical experts in Germany for their diligent intervention and wish a continued, strong recovery to the patient and his family”.
ProMetic is currently completing its single ascending dose clinical trial to demonstrate the safety, tolerability and pharmacokinetics of ProMetic’s plasma-derived plasminogen in patients with plasminogen deficiency. The clinical program will then cross-over to a phase II-III trial where the same plasminogen deficient patients will be administered multiple doses to define the optimal treatment regimen to achieve the targeted blood concentration of plasminogen. The FDA has agreed to an accelerated regulatory approval pathway given the rarity of the condition and the related unmet medical need. To secure an accelerated pathway approval, a drug must treat a serious condition, provide a meaningful advantage over available therapies and demonstrate an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit.
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 blindness. The 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 multiorgan 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 children with severe hypoplasminogenemia, apparently related to the deposition of fibrin in the cerebral ventricular system.
About ProMetic Life Sciences Inc.
ProMetic Life Sciences Inc. (prometic.com) is a long established biopharmaceutical company with globally recognized expertise in bioseparations, plasma-derived therapeutics and small-molecule drug development. ProMetic 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. ProMetic is also active in developing its own novel small-molecule 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 commercial activities in the U.S., 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, 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, 2014, 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.