Novartis, a renowned Swiss pharmaceutical company, has reportedly released Phase II data of its investigational oral therapy for PNH (paroxysmal nocturnal hemoglobinuria), LNP023, at the 2020 EBMT (European Society for Blood and Marrow Transplantation) Annual Meeting.

In the Phase II trial, LNP023 showed enhancements in hematological response as well as biomarkers of disease activity in patients suffering from active hemolysis despite being treated with eculizumab. Patients administered with LNP023 like an add-on to the conventional eculizumab treatment witnessed substantial reductions in the lactate dehydrogenase levels, which is a intravascular hemolysis biomarker.

Additionally, the patients also saw significant improvement in their Hb (hemoglobin) levels. In comparison with the baseline values solely on eculizumab, LNP023 therapy grew Hb by a significant 2.87 g/dL ((p<0.001), and all except two patients reached Hb levels>12 g/dL when there were no RBC (red blood cell) transfusions. All patients needed RBC transfusions before initiating LNP023 therapy.

Professor Antonio Risitano of Federico II University, Naples, and the Head of Hematology and the BMT Unit at Ospedale Moscati, Avellino in Italy and Professor Régis Peffault de Latour, Chief of French Reference Centre studying Aplastic Anemia as well as PNH, in Saint Louis Hospital, as well as University of Paris, are both the principal investigators of the LNP023 trial.

Professor Risitano stated that in the trial LNP023 administered orally showed meaningful clinical advantage and transfusion avoidance in PNH patients who had stayed anemic and were dependent on transfusions regardless of the standard of care anti-complement therapy.

Professor Peffault de Latour stated that the new insights majorly highlight that LNP023 is capable of controlling the hemolysis mechanism in the PNH disease. LNP023 might also change the therapy completely for PNH, added Peffault de Latour.

PNH is a life-threatening and rare blood disorder that is characterized by impaired bone marrow function, thrombosis, and complement driven hemolysis, leading to debilitating symptoms that might affect the quality of life of a patient.

Source credit: https://www.novartis.com/news/media-releases/novartis-announces-positive-results-from-phase-ii-study-lnp023-patients-paroxysmal-nocturnal-hemoglobinuria-pnh