RE:RE:FDA APPROVES PLI ! I got this in an Email from prometic.
US FDA GRANTS ORPHAN DRUG DESIGNATION TO PROMETIC’S PBI-4050 DRUG FOR THE TREATMENT OF ALSTRM SYNDROME
LAVAL, QUEBEC, CANADA, – March 3, 2017 – Prometic Life Sciences Inc. (TSX: PLI) (OTCQX: PFSCF) (“Prometic” or the “Corporation”) announces that an orphan drug designation status has been granted, by the United States Food and Drug Administration, for its orally active, anti-fibrotic, lead drug candidate, PBI-4050, for the treatment of Alstrm Syndrome (“AS”).
This condition is chronically debilitating due to permanent blindness, deafness, type 2 diabetes and life-threatening due to progressive organ failure. To this day, no satisfactory method of treatment has been authorised in the USA for patients affected by AS. Prometic is currently investigating the effects of PBI-4050 on multiple organs in AS patients in an ongoing, open label, Phase 2, clinical study in the United Kingdom (UK) with plans to expand the clinical program, both in the USA and elsewhere in Europe, once an optimal regulatory pathway has been defined with the FDA and the European Medicines Agency, respectively.
Dr. John Moran, Prometic’s Chief Medical Officer commented: “The positive results in patients with AS complement those we have observed in both our other trials in idiopathic pulmonary fibrosis and metabolic syndrome with type 2 diabetes, since AS includes features of both those conditions. AS leads to severe fibrosis in vital organs, including the heart, liver, lungs, and kidneys, with progressive multi-organ failure, and PBI-4050 has successfully reversed fibrosis in all those organs in numerous preclinical studies. We are very encouraged that PBI-4050 may offer a real long-term benefit for these patients”.
Pierre Laurin, Chief Executive Officer of Prometic commented, “Our next step will be to meet with the regulatory agencies to define the approval pathway to enable a promising treatment for these patients”.
Alstrm Syndrome is a rare inherited condition with progressive fibrosis involving multiple organs such as the liver, the kidneys and the heart. Idiopathic infantile dilated cardiomyopathy (ICM) presents acutely in nearly half of all AS subjects within the first few weeks of life. In survivors, cardiomyopathy recurs or develops de novo in up to 65% of adolescents and adults with high rates of morbidity and mortality, at which stage coarse fibrosis is evident on post-mortem.
Orphan Drug Designation is granted to drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven-year period of U.S. marketing exclusivity upon marketing approval for the designated indication, as well as with tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and the waiver of prescription drug user fees.
More about Alstrm syndrome:
Alstrm 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.
Alstrm 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 Alstrm 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 Alstrm 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. (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.