BCX9930 for the Treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH) in Participants Not Receiving Other Complement Inhibitor Therapy

Inclusion Criteria:

• Male or female, aged ≥ 18 years old

• Body weight ≥ 40 kg

• Documented diagnosis of PNH

• No complement inhibitor therapy for ≥ 12 months prior to screening

• Contraindication to or no access to approved (C3 or C5) complement inhibitortherapies

• Documentation of current vaccinations against Neisseria meningitidis andStreptococcus pneumoniae or willingness to start vaccination series

• At screening: PNH clone of ≥ 10%, hemoglobin ≤ 10.5 g/dL and lactate dehydrogenase ≥ 2 × upper limit of normal

Exclusion Criteria:

• Known history of or existing diagnosis of hereditary complement deficiency

• History of hematopoietic cell transplant or solid organ transplant or anticipatedcandidate for transplantation

• Myocardial infarction or cerebrovascular accident within 30 days prior to screening,or current and uncontrolled clinically significant cardiovascular or cerebrovascularcondition

• History of malignancy within 5 years prior to the screening visit

• Active bacterial, viral, or fungal infection or any other serious infection within 14 days prior to screening

• Treatment with anti-thymocyte globulin within 180 days prior to screening

• Initiation of treatment with an erythrocyte or platelet growth factor, or danazolwithin 28 days prior to screening

• Receiving iron supplementation with an unstable dose in the 28 days prior toscreening