PROMETIC’S PBI-4050 RECEIVES PROMISING INNOVATIVE MEDICINE DESIGNATION FROM THE UK MHRA FOR THE TREATMENT OF ALSTROM SYNDROME.
LAVAL, QUEBEC, CANADA, – January 24, 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 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. '
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