• Sustained safety observed over 48 weeks of treatment
• Beneficial clinical effects in liver sustained over 48 weeks of treatment
• Positive results support requests for meetings with both FDA and EMA to define the clinical-regulatory pathway for approval of PBI-4050 for the treatment of Alström syndrome
LAVAL, QUEBEC, CANADA – September 19, 2017 – Prometic Life Sciences Inc. (TSX: PLI) (OTCQX: PFSCF) (Prometic)
today announced that longer-term data from its on-going phase 2 open label clinical trial in subjects suffering from Alström Syndrome in the United Kingdom confirm that the beneficial clinical effects previously observed are sustained during prolonged treatment.
The clinical study has now enrolled 12 subjects. Given the evidence of clinical benefit and continuing safety and tolerability, the Data Safety Monitoring Board (DSMB) and Medicines and Healthcare products Regulatory Agency (MHRA) have allowed for 2 successive extensions of the duration of treatment. The duration of treatment has been extended from the original 24 weeks for an additional 36 weeks, and then once more for a further 12 weeks (total of 72 weeks). This last extension was to ensure that subjects could remain on treatment while the regulatory authorities are reviewing a rollover protocol which, if approved, would allow subjects to remain on treatment for an additional period of up to 96 weeks, or until regulatory approval is obtained in the UK.
“We have previously reported the encouraging results in the first 5 subjects who had reached 24 weeks of treatment. We now have results on 8 subjects who have had 36 weeks or more of treatment, up to 60 weeks” said Dr John Moran, Prometic’s Chief Medical Officer. “The safety and tolerability has been confirmed over this extended period , with no drug-related serious adverse events. The best-accepted non-invasive technique for assessment of liver fibrosis is the FibroScan. In these 8 subjects there was a significant improvement in this measure of liver stiffness, from a median of 8.5 at baseline to 5.8 at last measurement, an absolute decrease of 2.7 kPa (31%), p = 0.0098. Accompanying this, we saw a normalization of liver enzymes in those subjects whose levels were elevated at baseline. Furthermore, we observed a major reduction of key biomarkers in the urine of the 9 subjects in whom the 24 week results are available. Elevated levels of these biomarkers indicate ongoing kidney injury.”
“Pierre Laurin, Prometic’s President and Chief Executive Officer, stated “We believe we now have enough convincing clinical data to trigger meetings with both the Euopean and the US regulatory authories to determine the clinical-regulatory pathway for these patients, who are in dire need. We intend to hold such meetings in the fall of this year to determine whether this ultra-rare pediatric unmet medical need could formally become an indication granted priority review”.
|Markers of kidney damage in the urine||Change 24 weeks vs. baseline||N=9|
|Kim-1||↓ 43 %||P=0.01|
|Clusterin||↓ 47 %||P=0.02|
|Cystatin C||↓ 28 %||P=0.03|
|MCP-1||↓ 26 %||P=0.02|
|NGAL||↓ 31 %||P=0.03|
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, often 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 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. The clinical manifestations of Alström syndrome vary in severity, and not all affected individuals have all of the features associated with 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’ the anti-fibrotic activity of PBI-4050 in several key organs including the kidneys, the heart, the lungs and the liver. It is also effective in improving glucose control in animal models of diabetes. Open label Phase 2 studies in idiopathic pulmonary fibrosis and in Type 2 diabetes have provided preliminary evidence that the results seen in preclinical models translate into the corresponding human diseases.
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 is active in developing its own novel small-molecule therapeutic products targeting unmet medical needs in the field of fibrosis, cancer and autoimmune diseases/inflammation. A number of plasma-derived and small molecule products are under development for orphan drug indications. Prometic also 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. 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 and Asia.
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, 2016, 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.
For further information please contact:
President and CEO
Prometic Life Sciences Inc
Senior Director, Communications & Investor Relations
Prometic Life Sciences Inc.
Matt Middleman, M.D.
LifeSci Public Relations