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Resverlogix Receives Approval From Health Canada to Proceed With Fabry Disease Clinical Trial With Lead Compound Apabetalone
Calgary, Alberta (ots/PRNewswire) - Apabetalone expands in to first orphan disease clinical trial in patients with Fabry disease
Resverlogix Corp. ("Resverlogix" or the "Company") (TSX:RVX) today announced that it has received approval from Health Canada, Therapeutic Products Directorate, to proceed with a clinical trial with its lead compound apabetalone in patients with Fabry disease.
Donald McCaffrey, President and CEO, stated, "We are extremely pleased to receive approval for this clinical trial in Canada. Canada possesses some of the world's preeminent experts in the orphan Fabry disease field and a well-established patient registry. Working with us throughout the trial are Dr. Michael West, Principal Investigator, Canadian Fabry Disease Initiative, Professor, Department of Medicine, Dalhousie University, Halifax NS and Dr. Aneal Khan, Associate Professor, Departments of Medical Genetics, Paediatrics, Alberta Children's Hospital, Calgary, AB. Apabetalone is known to regulate key biomarkers associated with this rare disease and may potentially offer an alternative for patients with this rare disorder."
Fabry Disease Clinical Trial Summary
This is an open-label, exploratory clinical study to assess the patient safety and effect on key biomarkers of apabetalone in subjects with Fabry disease for up to 16 weeks.
The primary objective of the study is to evaluate the safety and tolerability of apabetalone in patients with Fabry disease. Secondary objectives include evaluating the effect of apabetalone in subjects with Fabry disease as determined by change in key biomarkers including alkaline phosphatase (ALP), high-sensitivity C-reactive protein (hs-CRP), and other well-known markers for chronic kidney disease.
The study population will consist of two cohorts:
Cohort 1: Patients with Fabry disease receiving enzyme replacement therapy (ERT).
Cohort 2: Patients with Fabry disease not receiving ERT
The Company expects the complete trial protocol to be available on http://www.clinicaltrials.gov in due course.
About Fabry Disease
Fabry disease is a rare, X-linked lysosomal storage disease that results from a mutation in the GLA gene leading to a deficiency in the alpha galactosidase-A enzyme that breaks down a fatty substance called Gb3. This leads to abnormal deposits of Gb3 in blood vessel walls and tissues throughout the body that can damage major organs and shorten lifespan. Fabry disease has a reported minimum prevalence of 1 in 17,000.
Patients with the disease experience various heart, kidney, and dermatological complications with stroke, heart disease and kidney complications being the top causes of mortality. Standard of care includes ERT, which introduces functional versions of the alpha-Gal-A enzyme into patients. However, ERT lacks long-term effectiveness, especially in improving longevity and limiting disease progression. Current medications approved for Fabry disease are not sufficient and there remains a large unmet need.
Resverlogix is developing apabetalone (RVX-208), a first-in-class, small molecule that is a selective BET (bromodomain and extra-terminal) inhibitor. BET bromodomain inhibition is an epigenetic mechanism that can regulate disease-causing genes. Apabetalone is the first and only BET inhibitor selective for the second bromodomain (BD2) within the BET protein called BRD4. This selective inhibition of apabetalone on BD2 produces a specific set of biological effects with potentially important benefits for patients with high-risk cardiovascular disease (CVD), diabetes mellitus (DM), chronic kidney disease, dialysis, Alzheimer's disease, Fabry disease, other orphan diseases, and peripheral artery disease, while maintaining a well described safety profile. Apabetalone is the only selective BET bromodomain inhibitor in human clinical trials. Apabetalone is currently being studied in a Phase 3 trial, BETonMACE, in high-risk CVD patients with type 2 DM and low high-density lipoprotein (HDL), and is expected to be initiated in a Phase 2a kidney dialysis trial designed to evaluate biomarker changes and safety parameters in up to 30 patients with end-stage renal disease treated with hemodialysis.
Resverlogix common shares trade on the Toronto Stock Exchange (TSX:RVX).
This news release may contain certain forward-looking information as defined under applicable Canadian securities legislation, that are not based on historical fact, including without limitation statements containing the words "believes", "anticipates", "plans", "intends", "will", "should", "expects", "continue", "estimate", "forecasts" and other similar expressions. In particular, this news release includes forward looking information relating to the potential role of apabetalone in the treatment of CVD, DM, chronic kidney disease, end-stage renal disease treated with hemodialysis, Alzheimer's disease, Alkaline phosphatase (ALP), Fabry disease, and Orphan diseases. Our actual results, events or developments could be materially different from those expressed or implied by these forward-looking statements. We can give no assurance that any of the events or expectations will occur or be realized. By their nature, forward-looking statements are subject to numerous assumptions and risk factors including those discussed in our Annual Information Form and most recent MD&A which are incorporated herein by reference and are available through SEDAR at http://www.sedar.com. The forward-looking statements contained in this news release are expressly qualified by this cautionary statement and are made as of the date hereof. The Company disclaims any intention and has no obligation or responsibility, except as required by law, to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.