Ra Pharmaceuticals announced today the launch of a Phase 1 study of its C5 complement inhibitor RA101495. From the Ra Pharmaceutical press release, “Ra Pharma is developing RA101495 for paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and other debilitating conditions. The product is designed for convenient, subcutaneous self-administration.” This first step in the clinical development of RA101495 will focus on in-human study to help determine suitable dosing for treating patients within the paroxysmal nocturnal hemoglobinuria (PNH) patient population, which is a rare disease with similar red blood cell destruction as in aHUS patients.
In just a few days clinicians and researchers in the field of hematology will gather for 57th American Society of Hematology Annual Meeting and Exposition (ASH), taking place December 5-8, 2014 in Orlando, Florida. Ra Pharmaceuticals will be presenting preclinical data regarding C5 inhibition related to its suitability as treatment for various complement-mediated disease. Details on the presentations at ASH are as follows:
Abstract Title: Preclinical Evaluation of RA101495, a Potent Cyclic Peptide Inhibitor of C5 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (#939) Session 101, Saturday 12/5
Abstract Title: Complement C5 Inhibition Blocks the Cytokine Storm and Consumptive Coagulopathy By Decreasing Lipopolysaccharide (LPS) Release in E. coli Sepsis (#765) Session 331, Monday 12/7
The Atypical HUS Foundation is hosting an informational booth at this ASH event, to provide an outreach opportunity that expresses the voice and issues of aHUS patients, families, and caregivers. We look forward to hearing more about potential new treatments that may become available in the future for aHUS patients, and to reporting that information to the aHUS community as events unfold in the drug development landscape.