A Study to Evaluate the Efficacy and Safety of APL-2 in Patients With PNH

Inclusion Criteria:

• Be at least 18 years old (inclusive).
• Have LDH ≥1.5 x ULN at the screening visit.
• Have PNH diagnosis, confirmed by high sensitivity flow cytometry (granulocyte or monocyte clone >10%).
• Have Hb less than the lower limit of normal (LLN) at the screening visit.
• Have ferritin greater than/equal to the LLN, or total iron binding capacity (TIBC) less than/equal to ULN at the screening visit, based on central laboratory reference ranges. If a subject is receiving iron supplements at screening, the Investigator must ensure that the subject's dose has been stable for 4 weeks prior to screening, and it must be maintained throughout the study. Subjects not receiving iron at screening must not start iron supplementation during the course of the study.
• Body mass index (BMI) ≤ 35 kg/m2 at the screening visit.
• Have a platelet count of >50,000/mm3 at the screening visit.
• Have an absolute neutrophil count >500/mm3 at the screening visit.

Exclusion Criteria:

• Treatment with any complement inhibitor (eg, eculizumab) within 3 months prior to screening.
• Hereditary complement deficiency.
• History of bone marrow transplantation.

• Concomitant use of any of the following medications is prohibited if not on a stable regimen for the time period indicated below prior to screening:

  • Erythropoietin or immunosuppressants for at least 8 weeks
  • Systemic corticosteroids for at least 4 weeks
  • Vitamin K antagonists (eg, warfarin) with a stable international normalized ratio (INR) for at least 4 weeks
  • Iron supplements, vitamin B12, or folic acid for at least 4 weeks
  • Low-molecular-weight heparin for at least 4 weeks