A Phase I Study to Assess the Safety of Pegcetacoplan (APL-2) as an Add-On to Standard of Care in Subjects With PNH

Inclusion Criteria:

Male or Female
At least 18 years of age
Weigh >55 kg
Diagnosed with PNH
On treatment with eculizumab (Soliris®) for at least 3 months
Hb < 10 g/dL at screening OR have received at least one transfusion within 12 months prior to screening
Platelet count of >30,000/mm3
Absolute neutrophil count > 500/mm3
Women of child-bearing potential (WOCBP) must have a negative pregnancy test at screening and must agree to use protocol defined methods of contraception for the duration of the study (see below)
Males with female partners of child bearing potential must agree to use protocol defined methods of contraception (see below) and agree to refrain from donating sperm for the duration of the study
Willing and able to give informed consent

Exclusion Criteria:

Active bacterial infection
Known infection with hepatitis B, C or HIV
Hereditary complement deficiency
History of bone marrow transplantation
Participation in any other investigational drug trial or exposure to other investigational agent, device or procedure within 30 days
Evidence of QTcF prolongation defined as > 450 ms for males and > 470 ms for females at screening
Creatinine clearance (CrCl) < 50 mL/min (Cockcroft-Gault formula) at screening
Breast-feeding women
History of meningococcal disease
No vaccination against N. meningitidis types A, C, W, Y and B (administered as two separate vaccinations), Pneumococcal conjugate vaccine or Pneumococcal polysaccharide vaccine 23 (PCV13 or PPSV23, respectively) and Haemophilus influenzae Type B (Hib) vaccination within 2 years prior to Day 1 (Visit 2) dosing.