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Press Release

FDA Grants Orphan Drug Designation to Omeros’ OMS721 for Treatment of IgA Nephropathy

- Initiating Phase 3 Program for OMS721 in IgA Nephropathy with Both Breakthrough and Orphan Designations -

SEATTLE--(BUSINESS WIRE)--Aug. 4, 2017-- Omeros Corporation (NASDAQ:OMER) today announced that OMS721 has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of Immunoglobulin A (IgA) nephropathy. OMS721 is Omeros’ lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), the effector enzyme of the lectin pathway of the complement system. IgA nephropathy is the most common primary glomerulopathy globally, with an estimated 120,000 to 180,000 cases in the U.S. alone, and accounts for up to 10 percent of all dialysis patients. Up to 40 percent of individuals with the disease develop end-stage renal disease, a life-threatening condition, within 20 years following diagnosis.

As previously reported, Phase 2 clinical trial results with OMS721 in IgA nephropathy patients show unprecedented reductions in urine protein levels during and following treatment with OMS721. Elevated urinary protein is highly correlated with poor outcomes in patients with IgA nephropathy. Following review of these data, FDA in June granted OMS721 breakthrough therapy designation for the treatment of IgA nephropathy. Omeros plans to begin enrolling patients in its Phase 3 registration trial in IgA nephropathy later this year.

“Working with FDA, we are initiating another Phase 3 program for OMS721 – this one in IgA nephropathy, which has been granted both breakthrough and orphan designations,” stated Gregory A. Demopulos, M.D., chairman and chief executive officer of Omeros. “This marks the second Phase 3 program for OMS721, joining our ongoing aHUS program that already has received fast track status from FDA. A third Phase 3 program for OMS721 could be added as well this year in stem cell transplant-associated thrombotic microangiopathy that, together with aHUS, has been granted orphan designation. Focused on bringing OMS721 to market as quickly as possible, we are excited about its prospects and the benefits that we expect OMS721 will provide for patients across a wide range of serious and life-threatening disorders.”

FDA grants orphan designation to promote the development of a drug that is expected to have significant therapeutic advantage over existing treatments that target a condition affecting 200,000 or fewer U.S. patients annually. It qualifies a company for benefits that apply across all stages of drug development, including seven years of market exclusivity following marketing approval, tax credits on U.S. clinical trials, eligibility for orphan drug grants, and waiver of certain administrative fees.

To date, more than 150 subjects worldwide have received OMS721, and no safety concerns have been noted.

About Omeros’ MASP Programs

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 is the effector enzyme of the lectin pathway, one of the principal complement activation pathways. 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 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. OMS721 is Omeros’ lead human MASP-2 antibody.

Following discussions with both the FDA and the European Medicines Agency, a Phase 3 clinical program for OMS721 in atypical hemolytic uremic syndrome (aHUS) is in progress. Also, two Phase 2 trials are ongoing. One is evaluating OMS721 in glomerulonephropathies, which has generated positive data in patients with immunoglobulin A (IgA) nephropathy and with lupus nephritis; the other has reported positive data both in patients with hematopoietic stem cell transplant-associated thrombotic microangiopathy (TMA) and in those with aHUS. Based on the positive Phase 2 data, a second Phase 3 clinical program for OMS721 is being initiated in IgA nephropathy and a third Phase 3 program could begin later this year in stem cell transplant-associated TMA. OMS721 can be administered intravenously, and Omeros also expects to commercialize OMS721 for one or more therapeutic indications as a subcutaneous injection. In parallel, Omeros is developing small-molecule inhibitors of MASP-2. Based on requests from treating physicians, Omeros has established a compassionate-use program for OMS721, which is active in both the U.S. and Europe. The FDA has granted OMS721 breakthrough therapy designation for IgA nephropathy, orphan drug status for the prevention (inhibition) of complement-mediated TMAs and for the treatment of IgA nephropathy, and fast track designation for the treatment of patients with aHUS.

Omeros also has identified MASP-3 as responsible for the conversion of pro-factor D to factor D and as a critical activator of the human complement system’s alternative pathway. The alternative pathway is linked to a wide range of immune-related disorders. In addition to its lectin pathway inhibitors, the company is advancing its development of antibodies and small-molecule inhibitors against MASP-3 to block activation of the alternative pathway. Omeros is preparing to initiate manufacturing scale-up of its MASP-3 antibodies in advance of clinical trials.

About Omeros Corporation

Omeros is a biopharmaceutical company committed to discovering, developing and commercializing both small-molecule and protein therapeutics for large-market as well as orphan indications targeting inflammation, coagulopathies and disorders of the central nervous system. Part of its proprietary PharmacoSurgery® platform, the company’s first drug product, OMIDRIA® (phenylephrine and ketorolac injection) 1% / 0.3%, was broadly launched in the U.S. in April 2015. OMIDRIA is the first and only FDA-approved drug (1) for use during cataract surgery or intraocular lens (IOL) replacement to maintain pupil size by preventing intraoperative miosis (pupil constriction) and to reduce postoperative ocular pain and (2) that contains an NSAID for intraocular use. In the European Union, the European Commission has approved OMIDRIA for use in cataract surgery and lens replacement procedures to maintain mydriasis (pupil dilation), prevent miosis (pupil constriction), and to reduce postoperative eye pain. Omeros has multiple Phase 3 and Phase 2 clinical-stage development programs focused on: complement-associated thrombotic microangiopathies; complement-mediated glomerulonephropathies; Huntington’s disease and cognitive impairment; and addictive and compulsive disorders. In addition, Omeros has a proprietary G protein-coupled receptor (GPCR) platform and controls 54 new GPCR drug targets and corresponding compounds, a number of which are in preclinical development. The company also exclusively possesses a novel antibody-generating platform.

Forward-Looking Statements

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 and variations thereof. 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 product commercialization and commercial operations, unproven preclinical and clinical development activities, regulatory oversight, intellectual property claims, competitive developments, litigation, 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 May 10, 2017. 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

Cook Williams Communications, Inc.
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