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

Inclusion Criteria:

1. Male or Female

2. At least 18 years of age

3. Weigh >55 kg

4. Diagnosed with PNH

5. On treatment with eculizumab (Soliris®) for at least 3 months

6. Hb < 10 g/dL at screening OR have received at least one transfusion within 12 months
prior to screening

7. Platelet count of >30,000/mm3

8. Absolute neutrophil count > 500/mm3

9. 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)

10. 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

11. Willing and able to give informed consent

Exclusion Criteria:

1. Active bacterial infection

2. Known infection with hepatitis B, C or HIV

3. Hereditary complement deficiency

4. History of bone marrow transplantation

5. Participation in any other investigational drug trial or exposure to other
investigational agent, device or procedure within 30 days

6. Evidence of QTcF prolongation defined as > 450 ms for males and > 470 ms for females
at screening

7. Creatinine clearance (CrCl) < 50 mL/min (Cockcroft-Gault formula) at screening

8. Breast-feeding women

9. History of meningococcal disease

10. 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.