Omeros Submits IND to Advance OMS721 Phase 2 Clinical Program -- Successful Phase 1 Results Set the Stage for Trials in Patients with Life-Threatening Blood Disorders --
SEATTLE, March 4, 2014 /PRNewswire/ -- Omeros Corporation (NASDAQ: OMER) today announced that it has submitted to the U.S. Food and Drug Administration (FDA) an investigational new drug (IND) application to initiate the Phase 2 clinical program for OMS721, the company's lead human monoclonal antibody targeting mannan-binding lectin‑associated serine protease-2 (MASP-2), the key regulator of the lectin pathway of the immune system. In the Phase 2 clinical program, Omeros will evaluate OMS721 in the treatment of thrombotic microangiopathies (TMAs), a family of orphan disorders, including atypical hemolytic uremic syndrome (aHUS) and thrombotic thrombocytopenic purpura (TTP), that occur in the microcirculation of the body's organs, most commonly the kidney and brain.
Last month Omeros announced positive data following completion of dosing in the Phase 1 clinical trial of OMS721, which was conducted in the Netherlands under a Clinical Trial Application. In that study OMS721, whether administered by subcutaneous injection or intravenous infusion, was well tolerated and achieved a high degree of sustained lectin pathway inhibition, consistent with the significant efficacy of OMS721 seen in animal models of TMA, age-related macular degeneration and other lectin pathway-related disorders. The lectin pathway is one of the principal complement activation pathways and plays a central role in the innate immune response.
The initial Phase 2 clinical trial is an open-label, two-stage ascending-dose-escalation trial in adult subjects with TMA. The objectives of the trial are to evaluate efficacy, safety, tolerability, pharmacokinetics, pharmacodynamics and immunogenicity of OMS721 in patients with TMAs, including aHUS, TTP and transplant-related TMA. Design of the trial is based on the final data from the Phase 1 clinical trial of OMS721 and on discussions with the FDA's Division of Hematology Products at a pre-IND meeting held earlier this year. The pre-IND meeting also included representation from the FDA's Office of Orphan Products Development given that OMS721 has been granted Orphan Drug designation from the FDA for prevention of complement-mediated TMAs. Enrollment into the Phase 2 clinical trial is expected to begin following FDA's clearance of the IND.
"We are excited to be initiating the Phase 2 program for OMS721 to evaluate the molecule across the family of TMAs," stated Gregory A. Demopulos, M.D., chairman and chief executive officer of Omeros. "Based on our preclinical data, the FDA awarded OMS721 Orphan Drug designation for all TMAs, broadening beyond our initial submission for aHUS only. Our team worked effectively to expand the scope of our program while maintaining our timeline, and we look forward to assessing the clinical activity of our drug in patients suffering from these life-threatening hematologic disorders."
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 and OMS721, its lead MASP-2 antibody, is designed to be self-administered by subcutaneous injection.
Omeros also believes that it has identified the proteins that activate the complement system's alternative pathway in humans, which is linked to a wide range of immune-related disorders. In addition to its lectin pathway inhibitors, the Company is advancing the development of antibodies that would block activation of the alternative pathway alone or in combination with the lectin pathway.
About Omeros Corporation
Omeros is a clinical-stage biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics targeting inflammation, coagulopathies and disorders of the central nervous system. Derived from its proprietary PharmacoSurgery® platform, the Company's lead drug product, OMS302 for lens replacement surgery, is currently under review for marketing approval by both the US Food and Drug Administration and the European Medicines Agency with commercial launch planned for 2014. Omeros' five other clinical programs are focused on schizophrenia, Huntington's disease and cognitive impairment; addictive and compulsive disorders; complement-related diseases; and preventing problems associated with surgical procedures. Omeros also has a proprietary GPCR platform, which is making available an unprecedented number of new GPCR drug targets and corresponding compounds to the pharmaceutical industry for drug development.
This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, which are subject to the "safe harbor" created by those sections for such statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. Omeros' actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with Omeros' unproven preclinical and clinical development activities, regulatory oversight, product commercialization, intellectual property claims, and the risks, uncertainties and other factors described under the heading "Risk Factors" in the Company's Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 7, 2013. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and the Company assumes no obligation to update these forward-looking statements, even if new information becomes available in the future.
SOURCE Omeros Corporation