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Message: Orphan Disease Announcement

http://www.resverlogix.com/media/press-release.html?id=528#.VgPYuY9VhHw

News Release Issued: Sep 24, 2015 (5:00am MDT)

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 (SolirisĀ®), 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 SolarisĀ® 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.

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