A Study of FT-4202 in Adults and Adolescents With Sickle Cell Disease (Hibiscus Study)

Inclusion Criteria:

• • Provision of consent
• Patient has a confirmed diagnosis of sickle cell disease
• At least 2 episodes of vaso-occlusive crises in the past 12 months
• Hemoglobin 5.5 and 10 g/dL (55 and 100 g/L) during screening
• Patients taking hydroxyurea, must demonstrate a stable dose for at least 90 days prior to start of study treatment
• Female patients of childbearing potential must use highly effective methods of contraception; male patients are willing to use barrier methods of contraception

Exclusion Criteria:

• • More than 10 vaso-occlusive crises within the past 12 months
• Female who is breast feeding or pregnant
• Hepatic dysfunction characterized by alanine aminotransferase (ATL) > 4.0 upper limit of normal (ULN)
• Hepatic dysfunction characterized by direct bilirubin > 3.0 upper limit of normal (ULN)
• Known HIV positive
• Active hepatitis B or hepatitis C infection
• Severe renal dysfunction or on chronic dialysis
• History of unstable or deteriorating cardiac or pulmonary disease within 6 months prior to consent including but not limited to: a.) Unstable angina pectoris or myocardial infarction or elective coronary intervention; b.) Congestive heart failure requiring hospitalization; c.) Uncontrolled clinically significant arrhythmias; d.) Symptomatic pulmonary hypertension
• History of overt clinical stroke within previous 2 years or any history of an intracranial hemorrhage
• Prior/concomitant therapy
• Patients receiving regularly scheduled blood (RBC) transfusion therapy (also termed chronic, prophylactic, or preventative transfusion)
• Receiving or use of concomitant medications that are strong inducers or moderate/strong inhibitors of CYP3A4/5 within 2 weeks of starting study treatment or anticipated need for such agents during the study
• Use of voxelotor within 28 days prior to starting study treatment or anticipated need for this agent during the study
• Use of a selectin antagonist (eg, crizanlizumab or other monoclonal antibody or small molecule) within 28 days of starting study treatment or anticipated need for such agents during the study
• Use of erythropoietin or other hematopoietic growth factor treatment within 28 days of starting study treatment or anticipated need for such agents during the study
• Receipt of prior cellular-based therapy (eg, hematopoietic cell transplant, gene modification therapy)