Omeros’ Narsoplimab Pivotal Trial Data to Be Shared as an Oral Presentation at the European Hematology Association Congress

Rhea-AI Summary

Omeros Corporation (Nasdaq: OMER) announced that data from its pivotal trial of narsoplimab for HSCT-TMA will be presented at the 2021 EHA Virtual Congress. Dr. Miguel-Angel Perales will present findings on the drug's impact on organ function improvement in high-risk patients. Narsoplimab, a human monoclonal antibody, is currently under priority review by the U.S. FDA for HSCT-TMA treatment and has received numerous designations, including breakthrough therapy. The condition HSCT-TMA is serious, with up to 90% mortality in severe cases, and no approved therapies currently exist.

Positive

• Narsoplimab shows potential in improving organ function in HSCT-TMA, a critical health issue.
• The drug is under priority review by the U.S. FDA for HSCT-TMA, indicating its importance.
• Narsoplimab has received breakthrough therapy and orphan drug designations, enhancing its market entry prospects.

Negative

• HSCT-TMA has a mortality rate exceeding 90% in severe cases, highlighting the condition's severity.
• No existing approved therapies for HSCT-TMA could indicate high market risk for Omeros.

Omeros Corporation (Nasdaq: OMER) today announced that data on organ function improvement from Omeros’ pivotal trial of narsoplimab for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) will be shared as an oral presentation at the 2021 European Hematology Association (EHA) Virtual Congress. The presentation, entitled Narsoplimab (OMS721) Treatment Contributes to Improvements in Organ Function in Adult Patients with High-Risk Transplant-Associated Thrombotic Microangiopathy, will be delivered by Miguel-Angel Perales, M.D., Chief of Adult Bone Marrow Transplant Service at Memorial Sloan Kettering Cancer Center.

Dr. Perales’ oral presentation will be available on demand through the EHA Virtual Congress platform to registered meeting attendees beginning Friday, June 11, 2021 at 9:00 am CEST / 3:00 am EDT. The presentation abstract (S241) can be accessed on EHA’s website.

About Hematopoietic Stem Cell Transplant-associated Thrombotic Microangiopathy

Hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) is a significant and often lethal complication of stem cell transplantation. This condition is a systemic, multifactorial disorder caused by endothelial cell damage induced by conditioning regimens, immunosuppressant therapies, infection, graft-versus-host disease, and other factors associated with stem cell transplantation. Endothelial damage, which activates the lectin pathway of complement, plays a central role in the development of HSCT-TMA. The condition occurs in both autologous and allogeneic transplants but is more common in the allogeneic population. In the United States and Europe, approximately 25,000 to 30,000 allogeneic transplants are performed annually. Recent reports in both adult and pediatric allogeneic stem cell transplant populations have found an approximately 40-percent incidence of HSCT-TMA, and high-risk features may be present in up to 80 percent of these patients. In severe cases of HSCT-TMA, mortality can exceed 90 percent and, even in those who survive, long-term renal sequalae (e.g., dialysis) are common. There is no approved therapy or standard of care for HSCT-TMA.

About Narsoplimab

Narsoplimab, also known as “OMS721,” is an investigational human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), a novel pro-inflammatory protein target and the effector enzyme of the lectin pathway of complement. 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. Omeros controls the worldwide rights to MASP-2 and all therapeutics targeting MASP-2.

A biologics license application (BLA) is under priority review by the U.S. FDA for use of narsoplimab in the treatment of HSCT-TMA, and the drug is in Phase 3 clinical programs for immunoglobulin A (IgA) nephropathy and atypical hemolytic uremic syndrome (aHUS). Narsoplimab is also being evaluated for the treatment of COVID-19 as a part of the I-SPY-COVID-19 platform trial sponsored by Quantum Leap Healthcare Collaborative. The FDA has granted narsoplimab breakthrough therapy designations for HSCT-TMA and for IgA nephropathy; orphan drug status for the prevention (inhibition) of complement-mediated thrombotic microangiopathies, for the treatment of HSCT-TMA and for the treatment of IgA nephropathy; and fast track designation for the treatment of patients with aHUS. The European Medicines Agency has granted orphan drug designation to narsoplimab for treatment in HSCT and for treatment of primary IgA nephropathy.

About Omeros Corporation

Omeros is a commercial-stage biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market and orphan indications targeting inflammation, immunologic diseases (e.g., complement-mediated diseases and cancers) and central nervous system disorders. Its commercial product OMIDRIA® (phenylephrine and ketorolac intraocular solution) 1%/0.3% continues to gain market share in cataract surgery. Omeros’ lead MASP-2 inhibitor narsoplimab targets the lectin pathway of complement and is the subject of a biologics license application under priority review by FDA for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy. Narsoplimab is also in multiple late-stage clinical development programs focused on other complement-mediated disorders, including IgA nephropathy, atypical hemolytic uremic syndrome and COVID-19. OMS906, Omeros’ inhibitor of MASP-3, the key activator of the alternative pathway of complement, is in a Phase 1 clinical trial, and the company’s PDE7 inhibitor program OMS527, targeting addiction and movement disorders, has successfully completed a Phase 1 trial. Omeros’ pipeline holds a diverse group of preclinical programs including a proprietary-asset-enabled antibody-generating technology and a proprietary GPCR platform through which it controls 54 GPCR drug targets and their corresponding compounds. One of these novel targets, GPR174, modulates a new cancer immunity axis recently discovered by Omeros, and the company is advancing GPR174-targeting antibodies and small-molecule inhibitors. For more information about Omeros and its programs, visit www.omeros.com.

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FAQ

What data will Omeros present at the 2021 EHA Virtual Congress?

Omeros will present data on organ function improvement from its pivotal trial of narsoplimab for HSCT-TMA.

Who is presenting the narsoplimab data at the EHA Congress?

Dr. Miguel-Angel Perales, Chief of Adult Bone Marrow Transplant Service at Memorial Sloan Kettering Cancer Center, will present.

What is the significance of narsoplimab for HSCT-TMA?

Narsoplimab is an investigational drug under FDA priority review, showing potential in treating a severe, often lethal condition.

What designations has narsoplimab received?

Narsoplimab has received breakthrough therapy, orphan drug status, and fast track designation for multiple conditions.

What is HSCT-TMA, and why is it important?

HSCT-TMA is a serious complication of stem cell transplants with high mortality rates and no approved therapies, underscoring the need for effective treatments.