"These data are impressive," stated Markus Sperandio, M.D., professor of cardiovascular physiology and microvascular disorders at the Walter-Brendel-Center of Experimental Medicine, Munich, Germany, where he conducted the TMA studies evaluating OMS721. "OMS721 looks like a promising agent to prevent thrombus formation in the microcirculation. We are planning additional studies to refine the dosing and timing of administration of OMS721 in models across a wide range of thrombotic disorders."
"We are pleased with these results and are excited to initiate the clinical program for OMS721," stated Gregory A. Demopulos, M.D., chairman and chief executive officer of Omeros. "The current treatment options for aHUS are limited and, when compared to those options, we believe that OMS721 carries marked advantages that will translate to significant patient benefits. We are eager to evaluate the drug product in aHUS patients, and we remain on track to begin Phase 1 clinical trials mid-year."
About Atypical Hemolytic Uremic Syndrome (aHUS)
Atypical Hemolytic Uremic Syndrome (aHUS) is a chronic, very rare, life-threatening disease. The condition has the hallmarks of a thrombotic microangiopathy and manifests as hemolytic anemia, thrombocytopenia and renal impairment. aHUS differs from HUS in that an infectious etiology, i.e., E. coli or streptococcal infection, is absent. Dysregulation of the complement system lies at the heart of aHUS pathogenesis, and genetic abnormalities in complement genes have been identified in greater than 60 percent of all aHUS patients. It is thought that certain precipitating factors are needed to trigger aHUS in a genetically predisposed individual. Many of the potential precipitating factors are linked to endothelial cell activation, stress or injury; all of these processes activate the lectin pathway. If untreated, most aHUS patients die or develop end-stage renal disease within one year of diagnosis. Current treatment options include plasma therapy and eculizumab, which have limitations in terms of feasibility, safety and patient convenience.About Omeros' MASP-2 Program Omeros controls the worldwide rights to MASP-2 and all therapeutics targeting MASP-2, a novel pro-inflammatory protein target involved in activation of the complement system, which is an important component of the immune system. The complement system plays a role in the inflammatory response and becomes activated as a result of tissue damage or microbial infection. MASP-2 appears to be unique to, and required for the function of, one of the principal complement activation pathways, known as the lectin pathway. Importantly, inhibition of MASP-2 does not appear to interfere with the antibody-dependent classical complement activation pathway, which is a critical component of the acquired immune response to infection, and its abnormal function is associated with a wide range of autoimmune disorders. MASP-2 is generated by the liver and is then released into the circulation. Adult humans who are genetically deficient in one of the proteins that activate MASP-2 do not appear to be detrimentally affected by the deficiency. Therefore, Omeros believes that it may be possible to deliver MASP-2 antibodies systemically.