- 100% success rate on meeting clinical trial’s primary end points required for Accelerated Approval Regulatory Pathway
- 100% clinical response rate with healing of lesions within weeks of treatment
- Successful pre-BLA meeting held with FDA
- ProMetic to commence filing plasminogen BLA modules with the FDA as planned in coming weeks
LAVAL, QUEBEC, CANADA, – October 26, 2016 – ProMetic Life Sciences Inc. (TSX: PLI) (OTCQX: PFSCF) (“ProMetic” or the “Corporation”) announced today that its pivotal Phase 2/3 clinical trial in patients with plasminogen deficiency has met its primary and secondary endpoints with the intravenous plasminogen treatment.
In addition to being safe, well tolerated and without any drug related serious adverse events, ProMetic’s plasminogen treatment achieved a 100% success rate of its primary end point, namely, a targeted increase in the blood plasma concentration level of plasminogen as a surrogate target. Moreover, all patients who had active visible lesions when enrolled in the trial had complete healing of their lesions within weeks of treatment, a 100% response rate for this secondary end point.
Following the pre-BLA meeting recently held with the Food and Drug Administration (“FDA”), it was agreed that ProMetic would continue along the Accelerated Approval Regulatory Pathway and file the pharmacokinetic safety data on 10 plasminogen deficient patients along with efficacy data available for each of these patients that have completed 12 weeks of treatment.
“The clinical response is impressive to the point that we are not expected to run additional clinical trials to demonstrate efficacy for a full licensure for this indication”, stated Dr John Moran, Chief Medical Officer of ProMetic. “We will continue to treat the patients for an additional period of 36 weeks to demonstrate the durability of the positive clinical activity observed to date. This additional clinical data will be submitted as a supplement to our BLA after our plasminogen receives the expected accelerated approval in 2017”. Dr Moran added; “The data we have seen so far indicates that a significant number of patients will need prophylactic treatment for their entire life. Most if not all patients will in addition require more aggressive treatment ahead of critical events such as elective surgery or following unexpected events such as injuries, or intercurrent illnesses.”
Pierre Laurin, CEO of ProMetic stated: “In accordance with the Fast Track Designation granted by the FDA, we will commence filing modules for the plasminogen BLA in the coming weeks. This means that we are on target for an expected commercial launch of plasminogen in the USA mid-next year. We look forward to efficiently reaching out to the patients and to the healthcare professionals attending to their well-being.”
In addition to the 100% response rate observed with the patients currently enrolled in the pivotal Phase 2/3 trial, ProMetic also generated similar data on 3 patients treated under a compassionate use program in the USA and in Germany. The USA Phase 2/3 study was also extended to include patients in Norway. All these patients will contribute to the pharmacokinetic, safety, and efficacy databases.
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 in 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. (www.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, 2015, 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.