Study of Efficacy and Safety of Twice Daily Oral Iptacopan (LNP023) in Adult PNH Patients Who Are Naive to Complement Inhibitor Therapy

Inclusion Criteria:

• Male and female participants ≥ 18 years of age with a diagnosis of PNH confirmed byhigh-sensitivity flow cytometry with RBCs and WBCs clone size ≥ 10%

• Mean hemoglobin level <10 g/dL

• LDH > 1.5 x Upper Limit of Normal (ULN)

• Vaccination against Neisseria meningitidis infection is required prior to the startof study treatment

• If not received previously, vaccination against Streptococcus pneumoniae andHaemophilus influenzae infections should be given

Exclusion Criteria:

• Prior treatment with a complement inhibitor, including anti-C5 antibody

• Known or suspected hereditary complement deficiency

• History of hematopoietic stem cell transplantation

• Patients with laboratory evidence of bone marrow failure (reticulocytes <100x109/L;platelets <30x109/L; neutrophils <0.5x109/L).

• Active systemic bacterial, viral (incl. COVID-19)or fungal infection within 14 daysprior to study drug administration.

• History of recurrent invasive infections caused by encapsulated organisms, e.g.meningococcus or pneumococcus.

• Major concurrent comorbidities including but not limited to severe kidney disease (e.g., dialysis), advanced cardiac disease (e.g., NYHA class IV heart failure),severe pulmonary disease (e.g., severe pulmonary hypertension (WHO class IV)), orhepatic disease (e.g., active hepatitis) that in the opinion of the investigatorprecludes participant's participation in the study.