Omeros Corporation (OMER)

Omeros is a Seattle-based biopharmaceutical company committed to discovering, developing, and commercializing small-molecule and protein therapeutics for large-market as well as orphan indications targeting inflammation, coagulopathies, and disorders of the central nervous system.

Our most advanced product candidates are derived from our proprietary PharmacoSurgery^® platform designed to improve clinical outcomes of patients undergoing ophthalmological, arthroscopic, urological and other surgical and medical procedures. Our PharmacoSurgery platform is based on low-dose combinations of therapeutic agents delivered directly to the surgical site throughout the duration of the procedure to inhibit preemptively inflammation and other problems caused by surgical trauma and to provide clinical benefits both during and after surgery.

Omidria™ (phenylephrine and ketorolac injection) 1%/0.3%, the first commercial product from Omeros’ PharmacoSurgery platform, was approved by the FDA in May 2014. Omidria is also currently under review for marketing approval by the European Medicines Agency (EMA).

Omeros’ six 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. Two additional programs are expected to advance into the clinic next year – one for the control of blood loss during surgery or resulting from trauma and the second for the treatment of a wide range of addictions and compulsions as well as any movement disorder. 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.

Omeros is developing a deep pipeline of small-molecule and protein therapeutic candidates targeting inflammation, coagulopathies, and disorders of the central nervous system. Our twelve programs include those focused on inflammation, coagulopathies, and multiple CNS disorders, as well as our three platform programs: PharmacoSurgery^®, antibody and G protein-coupled receptor all targeting both large-market and exciting orphan opportunities.

Products from our proprietary PharmacoSurgery platform, which yielded our first commercial product Omidria™ (phenylephrine and ketorolac injection) 1%/0.3%, for use during cataract surgery and other lens replacement procedures, are designed to improve the clinical outcomes of patients undergoing arthroscopic, urological, and other surgical and medical procedures.

Our MASP program is in clinical development to treat thrombotic microangiopathies, including atypical hemolytic uremic syndrome, and a wide range of inflammatory disorders.

Our two PDE10 clinical programs for the treatment of schizophrenia and Huntington's disease, our clinical program for the treatment and prevention of addictions and compulsions and our preclinical programs targeting other CNS disorders and coagulopathies further strengthen our pipeline and help create multiple opportunities for commercial success.

Our GPCR platform is making available an unprecedented number of new GPCR drug targets and corresponding compounds to the pharmaceutical industry for drug development, and our antibody platform enables the discovery of novel, high-affinity monoclonal antibodies.

For each of our product candidates and programs, we have retained all manufacturing, marketing and distribution rights.

PharmacoSurgery^®

Our proprietary PharmacoSurgery^® products are designed to improve the clinical outcomes of patients undergoing ophthalmological, arthroscopic, urological and other surgical and medical procedures. Omidria™ (phenylephrine and ketorolac injection) 1%/0.3%, is the first approved drug from our PharmacoSurgery platform

We have multiple programs focused on central nervous system (CNS) disorders, all targeting large markets.

Our most advanced CNS programs include our phosphodiesterase 10 (PDE10) program, focused on developing drugs for the treatment of schizophrenia, Huntington's disease and other cognitive disorders; our PPARγ program, focused on developing proprietary compositions that include peroxisome proliferator-activated receptor gamma (PPARγ) agonists for the treatment and prevention of addiction to substances of abuse (e.g., opioids, nicotine and alcohol); and our PDE7 program for the development of drugs for the treatment of movement disorders, such as Parkinson's d

We are developing antifibrinolytic agents for the control of blood loss during surgery or resulting from trauma. Excessive bleeding during cardiac surgery is known to increase overall morbidity and mortality. In an attempt to control this bleeding, patients undergoing cardiac and other extensive surgery often receive antifibrinolytic compounds. These drugs inhibit plasmin, an enzyme present in blood that degrades fibrin clots. Because plasmin degrades fibrin clots, an agent that inhibits plasmin may have potential utility for reducing blood loss due to trauma or surgery.

Prior to withdrawal from the market in 2008 for safety concerns, the antifibrinolytic Trasylol® (aprotinin) had been shown in a number of studies to be more effective at reducing blood loss than the other two most commonly used antifibrinolytics on the market today, tranexamic acid and epsilon aminocaproic acid. While Trasylol® is a potent inhibitor of plasmin, it is non-selective. In addition to plasmin, it significantly inhibits kallikrein and Factor XIa, two enzymes important in promoting clotting, and their inhibition can increase bleeding. Trasylol® was found to be associated with a number of safety issues, including increased mortality. Further, it is a bovine protein associated with anaphylactic reactions. While the specific cause of increased death remains unknown, an often-cited explanation is the lack of specificity of Trasylol®.

Our proprietary agents also inhibit plasmin but, unlike Trasylol®, they do not significantly inhibit kallikrein and Factor XIa. Additionally, our agents are derived from human protein, which may reduce immunological side effects. The properties of our proprietary agents are described in a peer-reviewed article titled "Engineering Kunitz Domain 1 (KD1) of Human Tissue Factor Pathway Inhibitor-2 to Selectively Inhibit Fibrinolysis: Properties of KD1-L17R Variant" that was published in the February 11, 2011 issue of the Journal of Biological Chemistry. We believe the efficacy and improved selectivity of our proprietary agents provide a novel approach to the control of bleeding from surgery and trauma.

We have selected a lead clinical candidate and are manufacturing pre-clinical supplies to enable the initiation of GLP toxicology studies intended to support the submission of an IND or clinical trial application and subsequent clinical trials. We plan to be in clinical trials with our anti-plasmin molecule in 2015.

Patent Position

As of February 15, 2014, we owned one issued patent and three pending patent applications in the U.S. and seven issued patents and 26 pending patent applications in foreign markets directed to our recent discoveries linking PPAR? and

G protein-coupled receptors (GPCRs), which mediate key physiological processes in the body, are one of the most valuable families of drug targets. According to Insight Pharma Reports, GPCR-targeting drugs represent 30 to 40 percent of marketed pharmaceuticals. Examples include Claritin® (allergy), Zantac® (ulcers and reflux), OxyContin® (pain), Lopressor® (high blood pressure), Imitrex® (migraine headache), Reglan® (nausea) and Abilify® (schizophrenia, bipolar disease and depression) as well as all other antihistamines, opioids, alpha and beta blockers, serotonergics and dopaminergics.

The industry focuses its GPCR drug discovery efforts mostly on non-sensory GPCRs. Of the 363 total non-sensory GPCRs, approximately 240 have known ligands (molecules that bind the receptors) with nearly half of those targeted either by marketed drugs (46 GPCRs) or by drugs in development (about 70 GPCRs). There are approximately 120 GPCRs with no known ligands, which are termed "orphan GPCRs." Without a known ligand, drug development for a given receptor is extremely difficult.

Omeros uses its proprietary high-throughput cellular redistribution assay (CRA) to identify small-molecule agonists and antagonists for orphan GPCRs, unlocking them to drug development. Omeros believes that it is the first to possess the capability to unlock orphan GPCRs in high-throughput, and that currently there is no other comparable technology. Unlocking these receptors could lead to the development of drugs that act at these new targets. There is a broad range of indications linked to orphan GPCRs including cardiovascular disease, asthma, diabetes, pain, obesity, Alzheimer's disease, Parkinson's disease, multiple sclerosis, schizophrenia, learning and cognitive disorders, autism, osteoporosis, osteoarthritis and several forms of cancer.

Omeros has begun screening orphan GPCRs against its small-molecule chemical libraries using its proprietary, high-throughput CRA. In addition to Class A orphan GPCRs, we have also begun screening orphan and non-orphan Class B receptors. Class B GPCRs have large extracellular domains and their natural ligands are generally large peptides, making the development of orally active, small-molecule drugs against these receptors, such as glucagon and parathyroid hormone, a persistent challenge. Omeros has announced that it has identified and confirmed sets of compounds that interact selectively with the following orphan receptors:

In parallel, Omeros is executing on its intellectual property strategy to protect each unlocked target through a multipronged approach directed to compound structures, uniquely identified signaling pathways and associated therapeutic indications. Collectively, this approach provides Omeros the opportunity to establish broad and enforceable protection for each unlocked receptor.

GPR17

We are optimizing compounds against GPR17, a G protein-coupled receptor (GPCR) which is linked to myelin formation. Myelin is an insulating layer rich in lipids and proteins that forms a sheath around the nerve fibers, which is essential for the proper functioning of the nervous system. Loss of the myelin sheath is the hallmark of several diseases, including multiple sclerosis, acute disseminated encephalomyelitis, Neuromyelitis Optica, transverse myelitis, chronic inflammatory demyelinating polyneuropathy, Guillain-Barré syndrome, central pontine myelinosis, inherited demyelinating diseases such as leukodystrophy, and Charcot-Marie-Tooth disease. We believe GPR17 inhibitors have the potential to promote remyelination and improve the outcome of these diseases as well as traumatic brain injury and spinal cord injury, conditions that have been associated with GPR17.

Discovering GPR17 inhibitors has previously been challenging to the pharmaceutical industry because this receptor is an orphan GPCR. However, using our proprietary CRA, we have been able to identify over 100 compounds that functionally interact with GPR17. We are now in the process of developing lead molecules targeting GPR17, which we intend to evaluate in remyelination assays in cell culture systems as well as in animal models.

Patent Position

As of February 15, 2014, we owned five issued patents and 10 pending patent applications in the U.S., and 43 issued patents and eight pending patent applications in foreign markets , which are directed to previously unknown links between specific molecular targets in the brain and a series of CNS disorders, our cellular redistribution assay and other research tools that are used in our GPCR program and to orphan GPCRs and other GPCRs for which we have identified functionally interacting compounds using our cellular redistribution as

Our proprietary ex vivo platform for the discovery of novel, high-affinity monoclonal antibodies utilizes a chicken B-cell lymphoma cell line and has demonstrated potential for the generation of diverse antibodies that can be readily engineered. This platform offers several advantages over other antibody platforms. The ex vivo immunizations of our proprietary cell line are significantly more rapid than whole animal immunizations and conventional hybridoma technology. By avoiding immunization of mice or other animals, we believe that the antibodies we generate from this platform are not limited by immunological tolerance.

Our platform is capable of producing novel antibodies against difficult targets, such as highly homologous proteins, enzymes, and receptors with short extracellular domains. Chicken antibodies also have unique features that enable binding capabilities distinct from mammalian antibodies. We have generated antibodies to several clinically significant targets, and our platform continues to add antibodies against additional important targets to our pipeline.

Patent Position

As of February 15, 2014, we owned and/or held worldwide exclusive license rights from the University of Washington to three pending U.S. Patent Applications, four foreign patent applications and one International Patent Cooperation Treaty Patent Application directed to our antibody platform. Additionally, we owned one issued U.S. Patent, two pending U.S. Patent Applications and eight pending foreign applications directed to antibodies generated