Omeros Corporation announced the recent presentation of a case report describing resolution of gastrointestinal hematopoietic stem cell transplant-associated thrombotic microangiopathy following narsoplimab treatment under a compassionate-use protocol. The case was presented at the 2019 Annual Meeting of the European Society for Blood and Marrow Transplantation. Omeros is reporting the case to ensure broad availability of the information presented. Narsoplimab, Omeros’ antibody to inhibit the lectin pathway of complement, has received breakthrough therapy designation from FDA and is in Phase 3 development for the treatment of HSCT-TMA. The case was presented by Rafael Duarte M.D., Ph.D., F.R.C.P., Associate Professor, Head of Hematopoietic Transplantation and Hemato-oncology Section, University Hospital Puerta de Hierro Majadahonda, Madrid, Spain, and Secretary of the EBMT. Dr. Duarte described an 18-year-old patient with biopsy-proven HSCT-TMA of the gastrointestinal tract causing severe gastrointestinal bleeding requiring transfusions. The patient received narsoplimab, her TMA resolved, and all transfusions have been discontinued. The patient continues to do well after cessation of narsoplimab treatment. Hematopoietic stem cell transplant-associated TMA was a focus of the EBMT meeting program. In addition to the educational course on early transplant complications that included Dr. Duarte’s presentation, the program included a review session on renal complications of HSCT-TMA and an Omeros-sponsored symposium entitled “How Do I Diagnose HSCT-TMA.” The meeting program also contained several other podium and poster presentations directed to complications related to endothelial injury, which include HSCT-TMA, graft-versus-host disease and diffuse alveolar hemorrhage. Omeros is preparing both a U.S. biologics license application and a European marketing authorization application for narsoplimab in the treatment of HSCT-TMA. The FDA has agreed that a rolling BLA submission is appropriate and discussions with European regulators are ongoing.
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