Pabetalone (RVX-208) to be tested in proof-of-concept pilot study in complement mediated diseases

Sep 24 2015

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

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.