A discovery by researchers at the University of Leicester led to the identification of mannan-binding lectin-associated serine protease-2 (MASP-2), a novel pro-inflammatory protein target in the complement system. Omeros holds worldwide exclusive licenses to rights related to MASP-2, the antibodies targeting MASP-2 and the therapeutic applications for those antibodies from the University of Leicester and from its collaborator, Medical Research Council at Oxford University, as well as Helion Biotech ApS.
MASP-2 is a key protein involved in activation of the complement system, which is a critical component of the immune system. The complement system plays a role in the inflammatory response and becomes activated as a result of tissue damage (e.g., trauma) or infection by microbial pathogens. 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, a central component of the acquired immune response to infection. If the classical pathway is caused to function abnormally, a wide range of autoimmune disorders can result.
Our preclinical data suggest that antibody-blockade of MASP-2 may have a preventive or therapeutic effect in the treatment of a wide range of complement-related diseases and disorders, including thrombotic microangiopathies (TMAs, e.g., atypical hemolytic uremic syndrome (aHUS), thrombotic thrombocytopenic purpura (TTP), etc.) and neovascular age-related macular degeneration (AMD).
OMS721 has received Orphan Drug Designation from the FDA for prevention of complement-mediated TMAs. We are completing a Phase 1 trial in Europe evaluating the safety, tolerability, pharmacodynamics and pharmacokinetics of OMS721 in healthy subjects. In this trial, OMS721 administration was well tolerated in all subjects, there were no drug-related adverse events, and no clinically significant abnormalities on laboratory tests or electrocardiograms have been observed. In the Phase 1 trial, both subcutaneous and intravenous routes of administration resulted in a high degree of lectin-pathway inhibition and successfully achieved the pharmacologic target of sustained inhibition for at least one week. We submitted an Investigational New Drug application to the FDA in March 2014 for our Phase 2 clinical program, and we expect begin enrolling subjects in the Phase 2 trial in the third quarter of 2014 to evaluate OMS721 for the prevention of complement-mediated TMAs.
We also believe that we have identified the proteins that activate the complement system's alternative pathway in humans, which is linked to a wide range of immune-related disorders, and we are establishing a broad intellectual property position around these discoveries. Therefore, in addition to our lectin pathway inhibitors, such as OMS721, we are advancing the development of antibodies that would block activation of the alternative pathway alone or in combination with the lectin pathway.
As of February 15, 2014, we exclusively controlled seven issued patents and 17 pending patent applications in the U.S. and 16 issued patents and 90 pending patent applications in foreign markets related to our MASP pr