Resverlogix Announces the commencement of an Orphan Disease Program specific for Complement Mediated Diseases

Apabetalone (RVX-208) to be tested in a proof-of-concept pilot trial in a complement mediated disease Paroxysmal Nocturnal Hemoglobinuria (PNH) in 2016

CALGARY, Sept. 24, 2015 /CNW/ - Resverlogix Corp. ("Resverlogix" or the "Company") (TSX:RVX) announced today the commencement of an Orphan Disease Program specific for Complement Mediated Diseases. New data generated by Resverlogix has demonstrated that BET inhibition by apabetalone (RVX-208) has effects on multiple biological pathways that underlie disease pathology. Specifically, apabetalone (RVX-208) has been shown to modulate the complement and coagulation pathways, known to play roles in cardiovascular disease and a variety of orphan indications. Based on these findings, Resverlogix plans to pursue a pilot proof-of-concept trial in complement mediated diseases, with the first clinical trial in Paroxysmal Nocturnal Hemoglobinuria (PNH).

Apabetalone (RVX-208) has been shown to downregulate multiple components of the complement and coagulation pathways both in vitro, in vivo and in the plasma of select patients treated with RVX-208 (from the ASSURE clinical trial). "We believe that the next step in testing the potential for apabetalone to downregulate the levels (and possibly the activity) of complement components in humans, is to test it in patients with an overactive complement cascade," stated Dr. Ewelina Kulikowski, vice president of scientific development at Resverlogix. "Based on this data, an initial Phase 2 pilot trial to test the effect of apabetalone treatment in a small group of PNH patients is proposed," she added.

In addition to apabetalone (RVX-208), preclinical testing on other BET inhibitors in the Resverlogix library demonstrates similar effects on important markers in the complement and coagulation cascades. These compounds are under consideration as follow on compounds for complement mediated diseases such as PNH, but also atypical hemolytic uremic syndrome (aHUS), glomerulonephritis, and others.

About PNH

According to the National Organization for Rare Disorders (NORD), PNH is a rare acquired hematopoietic stem cell disorder which results in the production of defective red blood cells that are extremely susceptible to premature destruction by a person's own immune system (complement system).  In PNH, the complement system mistakenly destroys blood cells due to the absence of a GPI-anchored proteins that would normally protect the blood cell from the activity of the complement system. The destruction of red blood cells (hemolysis) by complement leads to episodes of hemoglobin in the urine (hemoglobinuria), as well as repeated, potentially life-threatening blood clots (thromboses). 

Currently, severe cases of PNH are treated with eculizumab (Soliris®), however due to the cost of the drug, widely considered to be one of the most expensive drug in the world, the majority of patients are monitored and treated with symptomatic and supportive approaches such as: folic acid (folate) supplements, anticoagulation therapy, steroids, growth factors, blood transfusions and bone marrow transplants. According to a 2014 report by the International PNH Registry, approximately 75% of PNH patients worldwide are not receiving Solaris® treatment, leaving a large unmet medical need for patients with this severe disease.

A rare disease, also referred to as an orphan disease, is any disease that affects a small percentage of the population.  Most rare diseases are genetic, and thus are present throughout the person's entire life, even if symptoms do not immediately appear. Many rare diseases appear early in life, and about 30 percent of children with rare diseases will die before reaching their fifth birthday. There is no single, widely accepted definition for rare diseases. In the United States it is generally accepted as about 1 in 1,500 people while in Europe it is assumed to be about 1 in 2,000 people. 

About Resverlogix

Resverlogix is developing apabetalone (RVX-208), a first-in-class, small molecule that is a selective BET bromodomain 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 diseases such as high-risk cardiovascular disease (CVD), diabetes mellitus (DM), Alzheimer's disease, peripheral artery disease, and chronic kidney disease while maintaining an excellent safety profile. Apabetalone is the only selective BET bromodomain inhibitor in human clinical trials. Resverlogix's Phase 3 clinical trial BETonMACE in high-risk CVD patients with DM and low HDL is planned to commence in the fall of 2015. Resverlogix's common shares trade on the Toronto Stock Exchange (TSX: RVX). For further information please visit We can be followed on our blog at and via Twitter @Resverlogix_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 a pilot proof-of-concept trial in PNH planned by the Company, a Phase 3 clinical trial planned by the Company, research and development activities and the potential role of RVX-208 in the treatment of CVD, DM, Alzheimer's disease, peripheral artery disease, chronic kidney disease and complement mediated 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 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.

Company Contacts:

Donald J. McCaffrey 
President and CEO 
Resverlogix Corp. 
Phone: 403-254-9252 

Sarah Zapotichny
Director, Investor Relations & Corporate Communications
Resverlogix Corp. 
Phone: 403-254-9252 

SOURCE Resverlogix Corp.


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