Study of SGB-9768 in Patients With Paroxysmal Nocturnal Hemoglobinuria

Prior treatment

• Active systemic bacterial, viral, or fungal infection within 14 days before randomization.

Other illness

• History of congenital asplenia or splenectomy.
• Active or recurrent invasive infections caused by encapsulated bacteria (e.g., Neisseria meningitidis, Streptococcus pneumoniae, or Haemophilus influenzae).
• Evidence or history of tuberculosis infection (except adequately treated inactive tuberculosis with negative screening results).
• Recurrent chronic infections within 1 year before screening.
• Positive virology tests indicating active Hepatitis B Virus (HBV), Hepatitis C Virus (HCV), Human Immunodeficiency Virus (HIV), or syphilis infection.

Lab values

• Patients with reticulocytes \<100x10⁹/L; platelets \<30x10⁹/L; neutrophils \<0.5x10⁹/L.

Other

• Known or suspected hereditary or acquired complement deficiencies/abnormalities.

• Male and female participants ≥ 18 years of age;
• Diagnosis of PNH confirmed by flow cytometry, with a PNH clone size \>10% in granulocytes and/or monocytes;
• Mean hemoglobin level \<100 g/L at screening.
• Lactate Dehydrogenase (LDH) \> 1.5 x Upper Limit of Normal (ULN) at screening.
• Complement inhibitor-naïve, or previous complement inhibitor therapy discontinued for more than 5 drug half-lives or 3 months before randomization.
• Vaccination against Neisseria meningitidis infection is required prior to the start of study treatment. If not received previously, vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given.