Paroxysmal Nocturnal Hemoglobinuria (PNH)

Understanding Paroxysmal Nocturnal Hemoglobinuria

Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic, life-threatening blood disease that is associated with abnormally low (below normal) hemoglobin levels. PNH can appear at any age and in any race or gender, and is most often diagnosed in people in their early 30s.1,2

Low hemoglobin levels indicate ongoing hemolysis and can result in frequent transfusions and debilitating symptoms like severe fatigue and chest pain for individuals with PNH.

In PNH, blood cells lack complement regulatory proteins, so the body recognizes these healthy red blood cells as damaged.1 This leads to uncontrolled activation of the complement cascade, initiated at C3, and results in the destruction of oxygen-carrying red blood cells through a process called hemolysis.3 The complement cascade is a part of the immune system that is responsible for recognizing and eliminating pathogens and damaged cells in the blood.

Published data suggest that even with eculizumab treatment, approximately two-thirds of people with PNH have low hemoglobin levels, and 20% require three or more transfusions a year.4,5,6

Pegcetacoplan (APL-2) in PNH

Pegcetacoplan is a targeted C3-inhibitor designed to regulate uncontrolled or excessive complement activation.

With this approach, we believe that pegcetacoplan may have the potential to provide broad control of the red blood cell destruction (hemolysis) that drives the symptoms of PNH.

Interested in our PNH clinical trials?

For clinical trial information, please email us at



  1. Paroxysmal nocturnal hemoglobinuria (PNH). The Sidney Kimmel Comprehensive Cancer Center Web site. Accessed November 20, 2019.
  2. Besa EC. Paroxysmal nocturnal hemoglobinuria (PNH). MedScape 2017; Accessed November 20, 2019.
  3. Rosse WF, Ware RE. The molecular basis of paroxysmal nocturnal hemoglobinuria. Blood. 1995;86(9):3277-3286. Accessed November 20, 2019.
  4. McKinley CE, et al. Blood. 2017;130(suppl 1):3471.
  5. Hillmen P, et al. Br J Haematol. 2013;162(1):62-73.
  6. Risitano AM, Marotta S, Ricci P, Marano L, Frieri C, Cacace F, Sica M, Kulasekararaj A, Calado RT, Scheinberg P, Notaro R and Peffault de Latour R (2019) Anti-complement Treatment for Paroxysmal Nocturnal Hemoglobinuria: Time for Proximal Complement Inhibition? A Position Paper From the SAAWP of the EBMT. Front. Immunol. 10:1157. doi: 10.3389/fimmu.2019.01157