On September 24, Resverlogix Corp. announced the commencement of an Orphan Disease Program specific for Complement Mediated Diseases. Data generated by Resverlogix has demonstrated that BET inhibition by apabetalone (RVX-208) has effects on multiple biological pathways that underlie disease pathology. Also, it has been shown to modulate the complement and coagulation pathways, known to play roles in cardiovascular disease and a variety of orphan indications. 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.
