Omeros Corporation (NASDAQ: OMER) has announced significant results from the primary statistical analysis comparing the overall survival of patients with hematopoietic stem cell transplant-associated thrombotic microangiopathy (ta-tma) treated with narsoplimab to an external control group. The analysis revealed that narsoplimab-treated ta-tma patients experienced over a 3-fold reduction in the risk of mortality compared to similarly at-risk patients without narsoplimab treatment, with a hazard ratio of 0.32 and a p-value of less than 0.00001.
The pivotal trial, OMS721-TMA-001, demonstrated clinically meaningful and statistically significant superiority in overall survival, prompting Omeros to prepare and resubmit a biologics license application (BLA) to the FDA for narsoplimab. This aims to make narsoplimab the first approved therapeutic for ta-tma, a life-threatening complication of hematopoietic stem cell transplantation.
The comparative survival analysis for narsoplimab in ta-tma has been described as "truly outstanding" by Alessandro Rambaldi, M.D., a professor of hematology at the University of Milan. The results of the analysis have also garnered positive feedback from Rafael Duarte, M.D., Ph.D., who emphasized the compelling nature of the comparative survival results and the potential therapeutic option presented by narsoplimab for conditions characterized by endothelial dysfunction.
In response to the primary analysis results, Omeros plans to resubmit the narsoplimab BLA for ta-tma as quickly as possible, with additional analyses pending to further support the primary analysis results. The company's Chairman and CEO, Gregory A. Demopulos, M.D., expressed satisfaction with the primary analysis results, noting that the team worked collaboratively with the FDA and incorporated the agency's recommendations on the statistical analysis plan.
Narsoplimab, also known as "OMS721," is an investigational fully human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (masp-2), with a pending BLA before the FDA for use in the treatment of ta-tma. Omeros has also received breakthrough therapy and orphan drug designations for narsoplimab from the FDA, along with orphan drug status for the prevention of complement-mediated thrombotic microangiopathies.
Hematopoietic stem cell transplant-associated thrombotic microangiopathy (ta-tma) is a significant and often lethal complication of stem cell transplantation, with no approved therapy or standard of care. The condition affects both adult and pediatric populations undergoing stem cell transplantation, and the incidence of ta-tma is a cause for concern, with severe cases potentially leading to mortality exceeding 90 percent.
Omeros Corporation is an innovative biopharmaceutical company focused on 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. The company's lead masp-2 inhibitor narsoplimab is at the forefront of its efforts, with other programs in development targeting a broad spectrum of diseases and conditions. The market has reacted to these announcements by moving the company's shares 39.6% to a price of $10.41. If you want to know more, read the company's complete 8-K report here.
