Research Areas

Through our innovative and cutting-edge research, our world-class team has achieved scientific advances that no other group has accomplished, delivering new classes of therapeutics to treat patients with severe and life-threatening diseases and disorders.

Omeros is advancing novel research and development programs for small-molecule and protein therapeutics to address both orphan and large-market indications. With the successful commercialization of OMIDRIA® (phenylephrine and ketorolac intraocular solution) 1%/0.3%, our current areas of focus include complement-mediated diseases, central nervous system disorders and immuno-oncology. Tackling challenging diseases and disorders, we are targeting and exclusively control a broad range of previously untapped receptors and enzymes. The end result is first-in-class therapeutics designed to improve and save the lives of patients.

Complement-Mediated Disease

The complement system is an important component of the immune system, playing a role in the inflammatory response and becoming activated primarily as a result of tissue damage or microbial infection. Excessive activation or insufficient control of complement activation leads to immune imbalance that can fuel a cycle between complement, inflammatory cells and mediators, and tissue damage. Therapeutic modulation of complement activity is a promising area of research and development with the exciting potential to treat a large and expanding array of immunological and inflammatory conditions.

Narsoplimab (also known as OMS721) is being actively studied in a number of complement-mediated diseases including stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), IgA nephropathy, atypical hemolytic uremic syndrome (aHUS), and lupus nephritis.

Endothelial Injury Syndrome

Endothelial injury syndrome encompasses a range of disorders associated with hematopoietic stem cell transplantation. Stem cell transplantation is used to treat a wide range of blood-related cancers, severe types of anemia and a variety of life-threatening immune disorders. Across all of these, the pretransplant chemotherapy regimens cause significant inflammation and endothelial damage. Then, as part of the stem cell transplant procedure, patients must undergo conditioning drug regimens followed by immunosuppressive therapy, each causing even more inflammation and endothelial damage. Components of endothelial injury syndrome associated with stem cell transplantation include thrombotic microangiopathy (TMA), veno-occlusive disease (VOD) and diffuse alveolar hemorrhage (DAH). Graft-versus-host disease (GvHD), a common complication of stem cell transplant, also causes significant endothelial damage.

Endothelial injury syndrome
Stem cell transplantation for blood-related cancer

Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy (HSCT-TMA)

Hematopoietic stem cell transplant-associated thrombotic microangiopathy is a significant and often lethal complication of stem cell transplants. This condition is a systemic, multifactorial disorder caused by endothelial cell damage induced by infection, GvHD, and therapies used as part of stem cell transplantation. Activation of the complement system – primarily the lectin complement pathway – by endothelial damage plays a central role in the development of HSCT-TMA.

There are currently no approved products for the treatment or prevention of HSCT-TMA. We are developing narsoplimab, our antibody against MASP-2 (the effector enzyme of the lectin pathway), to treat HSCT-TMA.

In late-stage clinical trials compared to an historical control, narsoplimab improved the estimated median overall survival in high-risk HSCT-TMA patients by an order of magnitude and improved 100-day survival by approximately six-fold.


IgA Nephropathy

IgA nephropathy is the most common form of primary glomerulonephritis globally and is responsible for 10% of all dialysis patients worldwide. Forty percent of IgA nephropathy pateints develop end-stage renal disease and require dialysis within 20 years of diagnosis. There currently is no approved treatment for IgA nephropathy.

We are developing narsoplimab, our antibody against MASP-2 (the effector enzyme of the lectin complement pathway), to treat IgA nephropathy. In a Phase 2 clinical trial, narsoplimab reduced proteinuria in IgA nephropathy patients by 50%-90% and stabilized or increased glomerular filtration rates – effects unmatched by any other drug in development for IgA nepropathy.


Atypical hemolytic uremic syndrome (aHUS) therapeutic development

Atypical Hemolytic Uremic Syndrome (aHUS)

Atypical hemolytic uremic syndrome is a rare, chronic genetic disease characterized by microangiopathic hemolytic anemia, thrombocytopenia, and progressive kidney injury. While aHUS manifests most commonly in the kidney, the central nervous and gastrointestinal systems are also affected. The prognosis is poor, with progression to end-stage renal disease in up to half of aHUS patients.

We are developing narsoplimab, our antibody against MASP-2 (the effector enzyme of the lectin pathway), to treat aHUS. Phase 2 clinical results are positive, and a Phase 3 clinical program is underway.


Lupus Nephritis

Lupus nephritis is an inflammatory kidney disease caused by systemic lupus erythematosus (SLE), an autoimmune disorder in which the body’s immune system attacks its own cells and organs. Kidney damage is common in SLE patients and may lead to kidney failure over time.

In a Phase 2 clincial trial, narsoplimab, our antibody against MASP-2 (the effector enzyme of the lectin pathway), reduced proteinuria by an unprecedented 70%. We plan to initiate a Phase 3 program for narsoplimab in lupus nephritis.

Narsoplimab – nearing Phase 3 program for treatment of lupus nephritis

Leading Translational Research in Complement-Mediated Disease

Leading Translational Research in Complement-Mediated Disease

In 2018, Omeros entered into a research collaboration with the University of Cambridge to establish the Omeros Center at Cambridge for Complement and Inflammation Research (OC3IR). One of the OC3IR’s priorities will be to characterize further the role of the complement system in endothelial injury, which is implicated in a wide range of diseases, including thrombotic microangiopathies and glomerulonephropathies. The Center will also facilitate collaborative links between Omeros and other leading academic research laboratories in the field of complement and inflammation research.

Central Nervous System Disorders

Addiction

Substance addiction, including nicotine, cocaine, opioid and alcohol dependence, has an estimated societal cost of nearly $1 trillion annually in the US alone. Currently marketed drugs for addiction are, at best, only partially effective.

We discovered – and exclusively control – the link between phosphodiesterase 7 (PDE7) and any form of addiction or compulsive disorder. We have shown that our PDE7 inhibitor OMS527 blocks both craving and relapse across animal models of nicotine, cocaine, opioid and alcohol addiction as well as binge eating. PDE7 inhibitors also appear to avoid a major drawback of all currently marketed anti-addiction drugs – the depression of pleasure derived from other activities (e.g., social interaction, sex, sports, etc.) – which is responsible for poor patient compliance. Also, PDE7 inhibitors have been shown to be nonaddictive.

OMS527 – currently being evaluated for nicotine addiction

Immuno-oncology

Omeros is evaluating a number of novel compounds targeting oncology while also exploring immunotherapies that harness the body’s own immune system to fight cancer. Our GPCR program, currently including 54 distinct targets, holds the potential to utilize unique pathways to fight a broad range of solid tumors and hematologic malignancies. GPR174 and GPR161 oncology programs are advancing toward human trials.

Explore our GPCR program
Omeros’ GPCR program explores immunotherapies fighting solid tumors and hematologic malignancies