This is a Phase II, open-label study designed to evaluate the safety, efficacy, and immunogenicity of NM8074 administered intravenously to adult patients with Paroxysmal Nocturnal Hemoglobinuria (PNH).
Patients ≥ 18 years (males and females), weight ≥ 45 kg at the time of consent
Confirmation of PNH diagnosis by flow cytometry evaluation of white blood cells (WBCs), with neutrophil, granulocyte and/or monocyte clone size of ≥10%
Evidence of ongoing hemolysis
≥1 packed red blood cell (pRBC) transfusion within 12 months prior to screening
History of bone marrow, hematopoietic stem cell, or solid organ transplantation
History of splenectomy
Participation in any other investigational drug trial within 5 elimination half-lives of enrollment, or within 30 days, whichever is longer
Subjects currently or previously under other complement inhibitor treatments less than 3 months prior to study Day 1