Omeros’ Narsoplimab Meets its Pivotal Trial Primary Endpoint – Statistical Analysis Shows Survival Superiority Over External Control in Patients with TA-TMA
- Primary statistical analysis of overall survival compared TA-TMA patients treated with narsoplimab in its pivotal trial to a cohort of over 100 TA-TMA patients not treated with narsoplimab in an external control stem cell transplant registry; FDA-agreed analysis was conducted by an independent external statistical group
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Narsoplimab-treated TA-TMA patients had an over 3-fold reduction in risk of mortality (hazard ratio = 0.32 [
95% confidence interval: 0.23, 0.44]; p < 0.00001) compared to similarly at-risk patients without narsoplimab treatment - Omeros is preparing and will resubmit a BLA to FDA for narsoplimab to become the first approved therapeutic for TA-TMA, a life-threatening complication of hematopoietic stem cell transplantation
“The results of this comparative survival analysis for narsoplimab in TA-TMA are truly outstanding,” stated Alessandro Rambaldi, M.D., Professor of Hematology at the University of
Late last month, Omeros announced that it had received FDA’s recommendations on the statistical analysis plan (SAP) for the primary analysis comparing overall survival from time of first dosing in the 28 narsoplimab-treated TA-TMA patients in the pivotal trial OMS721-TMA-001 to overall survival, adjusted for immortal time bias, of the more than 100 TA-TMA patients in the external control registry, none of whom received narsoplimab. The two cohorts had similar demographics, diagnostic criteria, baseline characteristics, underlying diseases, conditioning regimens, and transplant procedures. All patients in both cohorts met the criteria for high risk of death as defined by an international expert panel tasked with reaching consensus on diagnostic and prognostic criteria and representing the American Society for Transplantation and Cellular Therapy, the Center for International Bone Marrow Transplant Research, the Asia-Pacific Blood and Marrow Transplantation Group, and the European Society for Blood and Marrow Transplantation. Following receipt of FDA’s recommendations, the independent statistical group incorporated them into the final SAP, conducted and validated the primary analysis, and then shared the results with Omeros.
“TA-TMA is an increasingly recognized and devastating complication of stem cell transplantation, often leading to multi-organ failure and death,” stated Rafael Duarte, M.D., Ph.D., F.R.C.P.(Lon), Associate Professor, Head of Department of Hematology and the Hematopoietic Transplantation Program, University Hospital Puerta de Hierro Majadahonda,
Additional analyses are being conducted by the independent statistical group, including an analysis similar to the primary analysis but comparing survival in the high-risk TA-TMA patients enrolled in the global narsoplimab expanded access program (EAP) to that of similarly at-risk TA-TMA registry patients. Sensitivity analyses related to each of the primary and EAP comparisons are also pending. These are expected to support the results of the primary analysis and will be included in the narsoplimab BLA for TA-TMA. Representative analyses will be shared publicly.
Across all its clinical trials in various indications to date, narsoplimab has been well tolerated and has shown no safety signal of concern.
“We are very pleased with the results of the primary analysis,” said Gregory A. Demopulos, M.D., Omeros’ Chairman and Chief Executive Officer. “Our team worked collaboratively with FDA over many months and incorporated FDA’s recommendations on the statistical analysis plan comparing overall survival in OMS721-TMA-001 to that of the external control. Given the primary analysis results, we will resubmit the narsoplimab BLA for TA-TMA as quickly as possible followed by our planned submission of the corresponding European marketing authorisation application in the second quarter of 2025. Additional sensitivity and other analyses are pending and should be available over the next few weeks. The Omeros team and clinical investigators have seen the benefits of narsoplimab in both adults and children. As demonstrated by the primary analysis, narsoplimab markedly improves survival, and we look forward to enabling broad accessibility for TA-TMA patients and their physicians.”
Two manuscripts – one comparing survival between OMS721-TMA-001 and TA-TMA registry patients and the other comparing survival in the EAP to that of TA-TMA registry patients – will be authored by international groups of transplant experts and submitted for publication to peer-reviewed journals in the first part of the coming year.
About Narsoplimab
Narsoplimab, also known as “OMS721,” is an investigational fully 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 has been demonstrated to leave intact the antibody-dependent classical complement activation pathway, which is a critical component of the acquired immune response to infection. A biologics license application (BLA) is pending before the FDA for use of narsoplimab in the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA). Omeros will resubmit the BLA for narsoplimab in TA-TMA followed by our planned submission of the corresponding European marketing authorisation application (MAA) in 2025. FDA has granted narsoplimab breakthrough therapy and orphan drug designations for TA-TMA and orphan drug status for the prevention (inhibition) of complement-mediated thrombotic microangiopathies as well as for the treatment of TA-TMA. The European Medicines Agency (EMA) has granted orphan drug designation to narsoplimab for treatment in hematopoietic stem-cell transplant.
About Hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-TMA)
Hematopoietic stem cell transplant-associated thrombotic microangiopathy (TA-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 TA-TMA. The condition occurs in both autologous and allogeneic transplants but is more common in the allogeneic population. In
About Omeros Corporation
Omeros is an innovative biopharmaceutical company committed to discovering, developing and commercializing first-in-class small-molecule and protein therapeutics for large-market and orphan indications targeting immunologic disorders, including complement-mediated diseases and cancers, as well as addictive and compulsive disorders. Omeros’ lead MASP-2 inhibitor narsoplimab targets the lectin pathway of complement and is the subject of a biologics license application pending before FDA for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy. Omeros’ long-acting MASP-2 inhibitor OMS1029 has successfully completed Phase 1 single- and multiple-ascending dose clinical studies. Zaltenibart, Omeros’ inhibitor of MASP-3, the key activator of the alternative pathway of complement, is advancing toward Phase 3 clinical trials for paroxysmal nocturnal hemoglobinuria and complement 3 glomerulopathy. Funded by the National Institute on Drug Abuse, Omeros’ lead phosphodiesterase 7 inhibitor OMS527 is in clinical development for the treatment of cocaine use disorder. Omeros also is advancing a broad portfolio of five novel cellular and molecular immuno-oncology programs. For more information about Omeros and its programs, visit www.omeros.com.
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 “aim,” “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “likely,” “look forward to,” “may,” “objective,” “plan,” “potential,” “predict,” “project,” “should,” “slate,” “target,” “will,” “would” and similar expressions and variations thereof. Forward-looking statements, including statements regarding the anticipated resubmission of the biologics license application for narsoplimab in
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Jennifer Cook Williams
Cook Williams Communications, Inc.
Investor and Media Relations
IR@omeros.com
Source: Omeros Corporation