Investigational treatment substantially improves hematological response in rare blood disorder

An investigational oral treatment for paroxysmal nocturnal hemoglobinuria (PNH) improved hematological response in patients with active hemolysis despite treatment with eculizumab, according to data from a Phase II study presented at 2020 European Society for Blood and Marrow Transplantation (EBMT) Annual Meeting.

PNH is a rare disorder in which red blood cells break apart prematurely, according to NORD.

In the open-label Phase II study, patients who were treated with LNP023 as an add-on to eculizumab had significant reductions in lactate dehydrogenase levels and improvement in hemoglobin (Hb) levels. LNP023 increased Hb by a clinically relevant 2.87 g/dL when compared to the baseline values on patients receiving eculizumab alone.

Without red blood cell (RBC) transfusions, 80% of patients achieved Hb levels >12 g/dL. All patients required RBC transfusions before starting treatment with LNP023.

“In the study, LNP023 given orally resulted in transfusion avoidance and meaningful clinical benefit in PNH patients who remained anemic and dependent on transfusions despite standard of care anti-complement treatment,” said Prof. Antonio Risitano, Federico II University, Naples, and Head of Hematology and the BMT Unit, Ospedale Moscati, Avellino, Italy, in a press release.

Read the full press release here.