Pharmacokinetics and Pharmacodynamics Study of SEG101 (Crizanlizumab) in Sickle Cell Disease (SCD) Patients With Vaso- Occlusive Crisis (VOC)

Inclusion Criteria:

- Male and non-pregnant female patients 16-70 years of age (inclusive)

- Confirmed diagnosis of sickle cell disease by hemoglobin electrophoresis or
high-performance liquid chromatography (HPLC) [performed locally]. All sickle cell
disease genotypes are eligible.

- Experienced at least 1 VOC within the preceding 12 months prior to Screening, as
determined by medical history.

- If receiving HU/HC or erythropoietin stimulating agent, must have been receiving the
drug for at least 6 months prior to Screening

- Hemoglobin ≥4.0 g/dL. Absolute neutrophil count ≥1.0 x 109/L and platelet count ≥75 x
109/L

- Adequate renal and hepatic function as defined:

- GFR ≥45 mL/min/1.73 m2 calculated by CKD-EPI

- ALT ≤3 x ULN

- Direct (conjugated) bilirubin ≤2 x ULN

- ECOG performance status ≤2

- Written informed consent (or assent/ parental consent for minor subjects) prior to any
screening procedures

Exclusion Criteria:

- History of stem cell transplant.

- Acute VOC ending 7 days prior to first dosing

- Ongoing hospitalization prior to Screening

- Received blood products within 30 days to first dosing

- Participating in a chronic transfusion program (pre-planned series of transfusions for
prophylactic purposes)

- History of severe hypersensitivity reactions to other monoclonal antibodies

- Received a monoclonal antibody or immunoglobulin -based agent within 1 year of
Screening, or has documented immunogenicity to a prior biologic.

- Received active treatment on another investigational trial within 30 days (or 5
half-lives of that agent, whichever is greater) prior to Screening

- Significant active infection or immune deficiency (including chronic use of
immunosuppressive drugs)

- Resting QTcF ≥470 msec at pretreatment (baseline) or other cardiac or cardiac
repolarization abnormality

Other protocol-defined Inclusion/Exclusion criteria may apply.