LAVAL, QC, April 12, 2018
The clinical study, which has enrolled 12 patients, demonstrated improved liver function and reduced fibrosis in fat tissues as evidenced from fat biopsies in patients treated with PBI-4050. Clinical activity and tolerability of PBI-4050 were sustained with prolonged treatment. The average treatment duration of PBI-4050 for the 12 patients has reached 52 weeks and further anti-fibrotic clinical activity in the heart, liver and fat tissue was observed with longer treatment exposure. Prometic had previously reported positive heart and liver data from this PBI-4050 Phase 2 study on March 28th.
Dr. John Moran, Prometic's Chief Medical Officer, stated, "In addition to improved FibroScan and MRI measurements in the liver, we now have direct evidence of a significant improvement of liver physiology in these patients. By using a demanding and complex technique called "insulin clamp" we were able to demonstrate that PBI-4050 significantly reduced the liver resistance to insulin. Analysis of data comparing fat biopsies taken at baseline and after 24 weeks of PBI-4050 treatment clearly show a significant clinical improvement. Importantly, PBI-4050's clinical activity and tolerability have been confirmed over this extended period, with no drug-related serious adverse events."
"The clinical activity of PBI-4050 has now been confirmed in the liver, the heart, the kidney, and in the fat tissue in the AS patients, stated Pierre Laurin, President and Chief Executive Officer of Prometic. "We look forward to presenting these detailed trial results this summer at meetings scheduled with European and U.S. regulatory authorities to determine the clinical-regulatory pathway for PBI-4050 in Alström syndrome."
Summary of Fat Biopsies and Liver Data
Dysfunctional adipose tissue involving enlargement of fat cells, is known to increase cardiometabolic risk. In AS patients, fat tissue is characterized with significant enlargement and coalescence of adipocytes forming giant vesicular vacuolation/steatosis. After 24 weeks of treatment with PBI-4050, adipocytes were more distinct, were smaller in size and no coalescence was observed.
Analysis of the biopsies also revealed that dystrophy and homogenized blurred cytoplasm in smooth muscle cells of arteries appeared normal after 24 weeks of treatment with PBI-4050.
A key metabolic effect of insulin is to suppress the production of glucose by the liver (endogenous glucose production (EGP)). Improvement of the liver function in AS patients was measured by the insulin clamp technique which confirmed a significant reduction of EGP and reduction of hepatic liver resistance after 24 weeks of PBI-4050 treatment.
Previous clinical findings from this trial, announced in a press release on March 28, 2018, demonstrated statistically significant reductions in cardiac fibrosis, liver stiffness (as measured by FibroScan) and liver fibrosis measured by MRI. This press release, which includes a detailed review of the data, can be found on the Company's website (CLICK HERE).
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.
PBI-4050 is an orally active lead drug candidate with excellent safety and efficacy profiles demonstrated in a large number of animal models of fibrosis affecting different organs, including the lung, liver, heart, kidney, and pancreas. The effects of PBI-4050 demonstrated in animal models have been replicated in Phase 2 studies in IPF, in metabolic syndrome with type 2 diabetes and in Alström syndrome. PBI-4050 is entering pivotal placebo-controlled phase 3 clinical trials for the treatment of IPF.
Prometic Life Sciences Inc. (www.prometic.com) is a biopharmaceutical corporation with two drug discovery platforms focusing on unmet medical needs in the field of fibrosis and orphan diseases. The first platform, small molecule therapeutics, stems from the discovery of two receptors GPR40/GPR84 acting as "dual master switches" which are at the core of the healing process as opposed to fibrosis. The second platform, plasma-derived therapeutics, leverages Prometic's vast experience in bioseparation technologies to address unmet medical needs with therapeutic proteins not currently commercially available, such as Ryplazim™ (plasminogen human). Prometic is also leveraging the second platform higher recovery yield advantage to develop some more established plasma-derived therapeutics with significant growth in demand such as Intravenous Immunoglobulin (IVIG) and provides access to its proprietary bioseparation technologies to enable pharmaceutical companies in their production of non-competing biopharmaceuticals. Globally recognized as a bioseparations expert, the Corporation derives revenue from this activity through sales of affinity chromatography media which contributes to offset the costs of its own R&D investments. 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, 2017, 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.
View original content:http://www.prnewswire.com/news-releases/prometic-presents-new-pbi-4050-clinical-data-from-ongoing-alstrom-syndrome-phase-2-trial-300628679.html
SOURCE ProMetic Life Sciences Inc.
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