Omeros Corporation announced that it has obtained regulatory clearance to start the Phase 1 clinical trial evaluating OMS721, the company's lead human monoclonal antibody from its mannan-binding lectin-associated serine protease-2 (MASP-2) program. The first clinical trial with OMS721 will be conducted in Europe and will evaluate the safety, tolerability, pharmacodynamics and pharmacokinetics of OMS721 administered subcutaneously in healthy subjects. Omeros plans to follow this study with a clinical trial evaluating OMS721 in patients with atypical hemolytic uremic syndrome (aHUS), a rare but life-threatening form of thrombotic microangiopathy (TMA). Enrollment in the initial clinical trial is scheduled to begin next month. Omeros has filed for orphan drug designation in the United States for the use of OMS721 to treat aHUS and will request orphan drug designation in Europe as well. Omeros controls the worldwide rights to MASP-2 and all therapeutics targeting MASP-2. Omeros' previously reported preclinical findings indicate that blockade of MASP-2 by OMS721 may have a preventive or therapeutic effect in the treatment of aHUS and other life-threatening TMAs, such as hemolytic uremic syndrome and thrombotic thrombocytopenic purpura, as well as a wide range of additional disorders, including age-related macular degeneration, ischemia-reperfusion injury and transplant-related complications.

Omeros Corporation announced positive results from its Phase 1 clinical program evaluating OMS824, the lead compound from its phosphodiesterase 10 (PDE10) program. This study measured the extent to which OMS824 binds to PDE10 in the basal ganglia, a region of the brain that has been linked to a wide range of diseases that affect cognition. The results show that the selected dose of OMS824 achieved approximately 50% occupancy of PDE10 without triggering the extrapyramidal symptoms (loss of muscle control, e.g., muscle rigidity, tremors, or involuntary muscle movements) reported as side effects with other PDE10 inhibitors that achieved similar or significantly lower occupancy levels. OMS824 is Omeros' proprietary compound that selectively inhibits PDE10, and the Company plans to advance into Phase 2 clinical trials in Huntington's disease and schizophrenia later in 2013. The OMS824 Phase 1 clinical program previously evaluated the tolerability and pharmacokinetics of single and multiple doses of OMS824 in healthy subjects. OMS824 was well tolerated by all subjects, and the only apparent drug-related adverse event was mild somnolence at the dose evaluated. In the clinical trial reported today, positron emission tomography (PET) scans were used to measure the binding activity of OMS824 to PDE10 in the brains of healthy subjects who received once daily dosing of OMS824 for seven days. Quantitation of PET images showed that approximately 50% occupancy of PDE10 in the basal ganglia was achieved by this dosing regimen. OMS824 was well tolerated and, consistent with earlier studies, mild somnolence was the only apparent adverse effect. Other PDE10 inhibitors under development have been associated with extrapyramidal symptoms in humans at similar or substantially lower PDE10 occupancy levels than that observed with OMS824. In contrast, these adverse side effects have not been seen at any of the OMS824 dose levels studied to date. This difference suggests that OMS824 has a better clinical therapeutic index or "safety factor" than other PDE10 inhibitors in development. Omeros plans to measure OMS824's binding to PDE10 in additional dose cohorts. Omeros' PDE10 Program: PDE10 is an enzyme that is expressed in areas of the brain linked to diseases that affect cognition and psychomotor functions, including Huntington's disease and schizophrenia. Huntington's disease is a hereditary neurodegenerative disorder that leads to movement, cognition, and behavioral abnormalities and premature death. Cognitive dysfunction is responsible for substantial disability in these diseases and is not improved by current medications. Omeros' proprietary compound OMS824 inhibits PDE10 and is being developed for the treatment of cognitive disorders. In addition to potential benefits on cognition, OMS824 could also improve psychiatric manifestations, such as the positive (e.g., hallucinations) and negative (e.g., flat affect) symptoms of schizophrenia.

Omeros Corporation announced that it has filed a Clinical Trial Application (CTA) with European regulators to initiate clinical trials evaluating OMS721, the company's lead human monoclonal antibody from its mannan-binding lectin-associated serine protease-2 (MASP-2) program. The lead indication for OMS721 will be atypical hemolytic uremic syndrome (aHUS), a rare but life-threatening form of thrombotic microangiopathy (TMA). Assuming positive regulatory review of its CTA, Omeros plans to initiate a Phase 1 clinical trial evaluating OMS721 early next quarter. The Phase 1 clinical trial will be a placebo-controlled, double-blind, single-ascending-dose study to evaluate the safety, tolerability, pharmacodynamics and pharmacokinetics of OMS721 administered subcutaneously in healthy subjects. Immediately following this study, Omeros plans to advance OMS721 into a Phase 2 clinical trial in aHUS patients. Omeros controls the worldwide rights to MASP-2 and all therapeutics targeting MASP-2, a novel pro-inflammatory protein involved in activation of the complement system -- an important component of the immune system. The complement system plays a role in the inflammatory response to tissue damage or microbial infection. OMS721 selectively inhibits MASP-2, blocking the lectin pathway of the complement system while leaving intact the classical pathway, which represents the acquired immune response to infection. Soliris(R) (eculizumab), the only approved complement-targeting antibody on the market and the only currently approved therapy for aHUS, inhibits microbial killing by the classical pathway, increasing the risk of infection for the patient. By targeting only the lectin pathway and leaving the classical pathway intact, OMS721 should not have this increased infection risk. In addition, Soliris requires a 20-minute to 2-hour intravenous infusion in a medical facility, while OMS721 is designed to be self-administered by subcutaneous injection, which would be more convenient for patients.

Omeros Corporation announced that its Investigational New Drug Application (IND) to evaluate OMS824 in Huntington's disease has been cleared by the U.S. Food and Drug Administration (FDA). OMS824 selectively inhibits phosphodiesterase 10 (PDE10), an enzyme expressed in areas of the brain linked to a wide range of diseases that affect cognition, including Huntington's disease and schizophrenia. OMS824 has shown promising results in animal models directly relevant to Huntington's disease and, as previously announced, OMS824 was well tolerated and exhibited favorable pharmacokinetic properties in a Phase 1 clinical program. Omeros plans to advance OMS824 into Phase 2 clinical trials for Huntington's disease next quarter and for schizophrenia later this year, the OMS824 IND for use in patients with schizophrenia having already been cleared by the FDA. Omeros also announced that it has requested Orphan Drug Designation from the FDA for OMS824 in the treatment of Huntington's disease. Orphan-designated drugs are eligible for incentives such as a faster approval process and additional market exclusivity. This designation is granted to drugs that are expected to provide significant therapeutic advantage over existing treatments and that target conditions affecting 200,000 or fewer U.S. patients annually. Huntington's disease is estimated to affect approximately 31,000 U.S. patients annually, and the only FDA--approved treatment for the disease is tetrabenazine, which is indicated for Huntington's-related movement disorders. Omeros recently announced that it has requested Fast Track designation for OMS824 in the treatment for Huntington's disease. Fast Track designation is reserved for drugs being developed to treat life-threatening conditions with the potential to address unmet medical needs and typically provides priority review status.

Omeros Corporation Presents at Jefferies 2013 Global Healthcare Conference, Jun-06-2013 01:00 PM. Venue: Grand Hyatt Hotel, 109 East 42nd Street at Grand Central, New York, New York, United States. Speakers: Gregory A. Demopulos, Co-Founder, Chairman, Chief Executive Officer, President, Chief Financial Officer, Principal Accounting Officer and Treasurer.