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Hairy nocturnal hemoglobinuria (PNH) is a rare, complex and deadly blood disease. Stem cells in
patients with partial hematopoietic bone marrow have mutations in the PIG-A gene on the X chromosome, and abnormal hyperpluration of mutant cells results in deficiency of GPI-anchored proteins translated by the gene, and red blood cells lack PIG-A enzymes, resulting in the absence of two surface proteins, CD55 and CD59.
LNP023 is an oral research drug for PNH.
Today, Novarma released data on a Phase II clinical trial of LNP023 (NCT03439839), which showed that patients in the LNP023 group had a reduced risk of active hemolysis, significantly lower levels of lactic acid dehydrogenase, and significantly improved levels of biomarkers and hemoglobin (Hb) in blood vessels.
LNP023 increases Hb by 2.87 g / dL (p .lt;0.001) compared to using eculizumab alone.
S. Professor Peffault de Latour, of the University of Paris, said: "These new data clearly show that LNP023 can control the hemolysis mechanism of the disease and may change the way PNH is treated."
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