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Pivotal trial data for narsolimab to treat hematopoietic stem cell transplant-associated thrombotic microangiopathy HSCT-TMA).- Omeros Corporation.

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Published:17th Jun 2021
Omeros Corporation announced that data on organ function improvement from its pivotal trial of narsoplimab for the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) were shared during an oral presentation at the virtual edition of the 26th Congress of the European Hematology Association (EHA) .

The presentation, entitled "Narsoplimab (OMS721) Treatment Contributes to Improvements in Organ Function in Adult Patients with High-Risk Transplant-Associated Thrombotic Microangiopathy", was delivered by Miguel-Angel Perales, M.D., Chief of Adult Bone Marrow Transplant Service at Memorial Sloan Kettering Cancer Center. The organ function improvement data presented underscore the potential of narsoplimab as a significant advance in the treatment of often fatal HSCT-TMA.

The pivotal trial’s findings include : The study population was high-risk, with 93 percent having multiple risk factors for poor outcomes, and highly reflective of “real-world” clinical practice. At baseline: i. 75% of patients had kidney dysfunction. ii. 57% had neurologic dysfunction. iii. 18% had pulmonary dysfunction. iv. 50% had multiple organ TMA involvement. v. 86% had significant infection. vi. 68% had graft versus host disease (GVHD). vii. 61% of the intent-to-treat (ITT) population (any patient receiving at least 1 dose of narsoplimab) and 74% of the per-protocol (PP) population (those patients receiving ? 4 weeks of dosing) responded to narsoplimab based on improvement in laboratory TMA markers (platelet count improvement and reduction in LDH levels) and clinical status (organ function or freedom from transfusion).viii.74% of eligible patients in the ITT population experienced improvement in organ function (67%, 50% and 100% in kidney, neurologic, or gastrointestinal function, respectively); 77% of eligible patients in the PP population experienced organ function improvement. ix.48% of eligible patients in the ITT population and 55% in the PP population experienced freedom from transfusion.

Narsoplimab was well tolerated in this very sick population . The most common adverse events were pyrexia, diarrhea, vomiting, nausea, neutropenia, fatigue, and hypokalemia, all common in HSCT. Six patients died during the core study period due to causes common in HSCT. There were no study discontinuations due to non-fatal adverse events. Detailed data and findings from the study are being submitted to a peer-reviewed scientific journal for publication.

Condition: Thrombotic Microangiopathies /HSCT- TMA
Type: drug

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