EUROPEAN COMMISSION GRANTS ORPHAN DRUG DESIGNATION TO PROMETIC’S PBI-4050 DRUG FOR THE TREATMENT OF ALSTRÖM SYNDROME
LAVAL, QUEBEC, CANADA, – January 18, 2017 –
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 granted an orphan drug designation status for the treatment of Alström Syndrome (“AS”) by the European Commission.
The European Medicines Agency (“EMA”) determined that the intention to treat AS with PBI-4050 was justified based on the preliminary clinical data generated in AS patients showing an improvement in liver fibrosis. 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 UK.
“The positive results in patients with AS nicely complement those already observed in our other trials in idiopathic pulmonary fibrosis and in metabolic syndrome and type 2 diabetes” commented Dr. John Moran, ProMetic’s Chief Medical Officer. “AS leads to severe fibrosis in vital organs, including the heart, liver, and kidneys, with progressive multi organ failure. PBI-4050 has successfully reversed fibrosis in the heart, lungs, kidneys, pancreas and liver in several preclinical studies. We are very excited at the idea that PBI-4050 may well offer a real long-term benefit for these patients”.
This condition is chronically debilitating due to permanent blindness, deafness, and type 2 diabetes and life-threatening due to progressive organ failure. No satisfactory method of treatment has been authorised so far in the European Union for patients affected by AS.
Pierre Laurin, Chief Executive Officer of ProMetic commented: “We will continue to work diligently with medical experts and with the regulatory agencies to define an optimal regulatory pathway to provide a treatment for these patients affected by AS”.
Alström 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.
European Orphan Drug Designation is granted to novel drugs or biologics that treat a rare disease or condition affecting fewer than 250,000 patients in the European Union. The designation provides the drug developer with a ten year period of marketing exclusivity upon marketing approval for the designated indication, as well as reduced fees for regulatory activities, the ability to apply for marketing authorisation centrally in the European Union and protocol assistance, a form of scientific advice specifically for orphan medicines.
To view the Alstrom Syndrome related video, please use the following URL address: prometiclifesciencesinc.cmail19.com
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.'
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