» Second case of broncho-tracheal blockage and multiple organ lesions shows immediate benefit
» Established dose regimen supports acute treatment use in both pediatric and adult patients
» Safety and tolerability confirmed by phase I trial results
» Significant clinical improvement observed with both acute and chronic lesions
» Safety, pharmacokinetic & clinical data presented at the American Society of Hematology annual conference in Orlando, Florida (ASH 2015)
LAVAL, QUEBEC, CANADA – December 7, 2015 – ProMetic Life Sciences Inc. (TSX: PLI) (OTCQX: PFSCF) (“ProMetic” or the “Corporation”) announced today new data from its plasma-derived plasminogen replacement therapy Phase I clinical trial for the treatment of Congenital Plasminogen Deficiency (“CPD”). The results from the two cohorts of patients enrolled in the Phase I trial confirm that ProMetic’s plasminogen replacement therapy is safe, well tolerated and without any related serious adverse events. Moreover, there were no plasminogen antibodies detected and the results confirm the established therapeutic dose of 6 mg/kg.
Dramatic and rapid improvements were observed in a severely affected patient, a 36 year-old woman with a plasminogen level of 4% with involvement of multiple organ systems, including the lungs, nasal passages, eyes, gums and urinary tract. . Within minutes of completing the plasminogen infusion, she noted improvement in her breathing, and within 2 hours blew a piece of tissue from her nose and coughed up soft tissue lesion previously blocking her airway. This improvement in the lung was also coupled with a reduction in the size of her conjunctival lesions and improvement in her prominent gum lesions. She had a single episode of passing blood and lesional material in the urine 5 hours after the infusion. On the day following infusion, her respiratory status continued to be substantially improved and she did not require her typical nebulizer treatment in the morning.
Dr John Moran, ProMetic’s Chief Medical Officer, commented “This is the second time we have observed such a dramatic and rapid positive effect in broncho-tracheal blockage. The reduction of co-existing chronic lesions in multiple organs is also remarkable. These effects were achieved without clinical complications. This case supports our belief that even chronic lesions can be resolved with repeated administration, as we saw in the first case in Germany”.
According to Pierre Laurin, President and Chief Executive Officer of ProMetic Life Sciences: “The pharmacokinetic profile of our plasminogen drug has been established to the point that our team was able to provide the necessary clinical insight to enable the dramatic rescue of the critically ill 20 month old infant in Germany announced last month. We are also very pleased to see a second patient immediately respond following infusion of the drug”.
The phase II/III trial is scheduled to enroll 12 – 15 plasminogen deficient patients, most of whom have participated in the phase I trial, using multiple doses to define the optimal treatment regimen to achieve the targeted blood concentration of plasminogen. ProMetic expect to update the FDA on the PK data and safety profile from the completed phase I in the coming weeks and anticipate starting the enrollment of patients in the Phase II/III trial during the first quarter of 2016 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.
ProMetic’s plasminogen has received orphan drug designation in both the US and Europe.
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 blood clots and clearance of extravasated fibrin. Plasminogen is therefore involved in wound healing, cell migration, tissue remodeling, angiogenesis and embryogenesis. For more information, please visit: http://plasminogen.com
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 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 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 and orphan drugs. 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., Canada, Europe, Russia, Asia and Australia.
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.
For further information please contact:
President and CEO
ProMetic Life Sciences Inc.
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ProMetic Life Sciences Inc.