Omeros (OMER) Requests FDA Fast-Track Designation for OMS721 as IgA Nephropathy Treatment

October 31, 2016 7:02 AM EDT

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Omeros Corporation (Nasdaq: OMER) announced that it has requested fast track designation from the U.S. Food and Drug Administration (FDA) for the development of OMS721 for the treatment of patients with immunoglobulin A (IgA) nephropathy (also known as Berger’s disease). The company recently reported positive data (p = 0.017) from the company’s Phase 2 clinical trial of OMS721 for the treatment of kidney disorders, including IgA nephropathy, none of which currently have an approved treatment and all of which frequently lead to end-stage renal disease and dialysis. OMS721 significantly improved key endpoints of renal function and patients achieved partial remission with just 12 weeks of dosing. With similar outcomes in additional patients, the company plans to pursue accelerated approval. 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.

FDA’s fast track program streamlines the development of drugs intended to treat serious or life-threatening conditions and that have the potential to address unmet medical needs. Fast track drugs are eligible for more frequent and timely meetings with FDA to discuss the development plan and to ensure that data needed for approval are collected appropriately. Drugs in the fast track program typically are granted priority review status and their respective New Drug Applications are accepted and reviewed by the FDA as rolling submissions. If granted, fast track designation is expected to expedite Omeros’ Phase 3 program for OMS721 in IgA nephropathy, which is planned to initiate next year.

FDA already has granted fast track designation for OMS721 in patients with atypical hemolytic uremic syndrome (aHUS) and orphan designation for OMS721 in patients with thrombotic microangiopathies (TMAs), including aHUS and hematopoietic stem cell-associated TMAs (HSCT-TMAs). In addition to positive Phase 2 data in IgA nephropathy and membranous nephropathy, Omeros has reported positive data from Phase 2 clinical trials in both aHUS and HSCT-TMAs. Following guidance from FDA and from the European Medicines Agency, Omeros plans to open enrollment for its single Phase 3 trial in patients with aHUS later this year. Based on discussions with the FDA, the company is also pursuing breakthrough therapy designation for OMS721 in IgA nephropathy and in HSCT-TMAs, neither of which has any approved treatment.

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 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. Omeros has received both Orphan Drug status and Fast Track designation from the U.S. FDA for its lead human MASP-2 antibody OMS721. Following discussions with both the FDA and the European Medicines Agency, a Phase 3 program for OMS721 in atypical hemolytic uremic syndrome is in progress. Also, two Phase 2 trials are ongoing with one evaluating OMS721 in glomerulonephropathies, which has generated positive data in patients with immunoglobulin A (IgA) nephropathy and with membranous nephropathy, and the other in thrombotic microangiopathies (TMAs), with positive data reported in patients with hematopoietic stem cell transplant-associated TMA. In addition to potential intravenous administration, Omeros plans to commercialize OMS721 for one or more therapeutic indications as a subcutaneous injection and is also 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.

Omeros also has identified MASP-3 as the protein that is critical to the activation of 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 its development of antibodies and small-molecule inhibitors against MASP-3 to block activation of the alternative pathway.

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