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ProMetic's PBI-4050 Receives Promising Innovative Medicine Designation From the UK MHRA for the Treatment of Alström Syndrome

PR Newswire

LAVAL, QC, January 24, 2017 /PRNewswire/ --

ProMetic Life Sciences Inc. (TSX: PLI) (OTCQX: PFSCF), ("ProMetic" or the "Corporation") announced today that its orally active lead drug candidate, PBI-4050, has been issued a Promising Innovative Medicine ("PIM") designation by the UK Medicines and Healthcare Products Regulatory Agency ("MHRA") for the treatment of Alström Syndrome ("AS").

A PIM designation is an early indication that a medicinal product is a promising candidate for the Early Access to Medicines Scheme ("EAMS"), intended for the treatment, diagnosis or prevention of a life-threatening or seriously debilitating condition with the potential to address an unmet medical need following an early review of the clinical data by the agency. To obtain a PIM designation, the following criteria must be fulfilled:

  1. The condition should be life-threatening or seriously debilitating with a high unmet medical need (no method of treatment or existing methods have serious limitations)
  2. The medicinal product is likely to offer major advantage over methods currently used in the UK
  3. The potential adverse effects of the medicinal product are likely to be outweighed by the benefits

Such a designation is a prerequisite to enter the EAMS scientific opinion assessment step.

Dr John Moran, ProMetic's Chief Medical Officer, commented on the PIM designation: "We are very proud to have received the PIM designation. We are pleased to see the MHRA agree that the data generated so far with PBI-4050 in the Alström Syndrome phase 2 clinical trial demonstrates that it has the potential to address this very serious and debilitating medical condition for which there is basically no complete medical solution available at the moment". 

The EAMS provides an opportunity for important drugs to be used in UK clinical practice in parallel with the later stages of the regulatory process. It is anticipated that medicines with a positive EAMS opinion could be commercially made available to patients 12 - 18 months before receiving marketing authorisation.

Mr. Pierre Laurin, President and Chief Executive Officer of ProMetic stated: "We are very enthusiastic for patients afflicted with this disease with MHRA's decision. We believe PBI-4050 to have first-in-class anti-fibrotic properties and look forward to continue working closely with the MHRA to advance PBI-4050 through its clinical program and provide Alström Syndrome patients with a new treatment option as soon as possible".

The PIM designation follows the recent grant of an orphan drug designation status for the treatment of Alström Syndrome by the European Commission.

More about Alström Syndrome:  

Alström Syndrome is a rare inherited autosomal recessive syndrome characterized by the onset of obesity in childhood or adolescence, Type 2 diabetes with severe insulin resistance, dyslipidemia, hypertension and severe multi-organ fibrosis, involving the liver, kidney and heart.

Alström Syndrome is also characterized by a progressive loss of vision and hearing, a form of heart disease that enlarges and weakens the heart muscle (dilated cardiomyopathy), and short stature. This disorder can also cause serious or life-threatening medical problems involving the liver, kidneys, bladder, and lungs. Some individuals with Alström Syndrome have a skin condition called acanthosis nigricans, which causes the skin in body folds and creases to become thick, dark, and velvety. The signs and symptoms of Alström Syndrome vary in severity, and not all affected individuals have all of the characteristic features of the disorder.

More about PBI-4050  

PBI-4050 is an orally active lead drug candidate with excellent safety and efficacy profiles confirmed in several in vivo experiments targeting fibrosis. Fibrosis is a very complex process by which continuing inflammation causes vital organs to lose their function as normal tissue is replaced by fibrotic scar tissue. The proof of concept data generated to date confirms our lead drug candidates' anti-fibrotic activity in several key organs including the kidneys, the heart, the lungs and the liver. Twenty six million patients in the U.S. alone are believed to suffer from chronic kidney diseases ("CKD"). Patients with severe CKD stages (3 and 4) suffer from a progressive loss of their renal function leading to end-stage renal disease and the need for dialysis or kidney transplant. Cardiovascular complications are the most common cause of death in dialysis patients.

About ProMetic Life Sciences Inc.          

ProMetic Life Sciences Inc. (http://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.

Media contact
Pierre Laurin
President and CEO, ProMetic Life Sciences Inc.
p.laurin@prometic.com
+1-450-781-0115

Frédéric Dumais
Senior Director, Communications and Investor Relations, ProMetic Life Sciences Inc.
f.dumais@prometic.com
+1-450-781-0115



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