Study to Assess Efficacy and Safety of VIT-2763 (Vamifeport) in Subjects With Sickle Cell Disease

Inclusion Criteria:

• Male or female subjects with confirmed diagnosis of SCD, including only HbS/S orHbS/βT0 genotype.

• Subjects who had at least 1 and no more than 10 vaso-occlusive crises (VOC) episodesreported within 12 months prior to screening.

• Body weight ≥40 kg and ≤120 kg at screening and baseline.

• Subjects on concomitant hydroxyurea must be on a stable dose (mg/kg) for ≥3 monthsprior to screening Visit V1

• Female subjects of childbearing potential, must have negative pregnancy, must havestopped breastfeeding as of first dose, and must either commit to true abstinencefrom heterosexual contact or must be willing to use adequate contraceptiveprecautions.

• Male subjects must practice true abstinence or agree to use a condom during sexualcontact with a pregnant female or a female of childbearing potential

Exclusion Criteria:

• Hb level <6.0 g/dl or >10.4 g/dl for female participants and >11.0 g/dl for maleparticipants, at screening Visit V1

• Having received red blood cell (RBC) transfusion therapy within 4 weeks prior toscreening, or ongoing or planned RBC transfusion therapy during the course of thestudy

• Low levels of Ferritin or transferrin saturation or total iron-binding capacity atscreening

• Subjects being hospitalized for SCD-related events within 14 days before thescreening visit

• Chronic liver disease or history of liver cirrhosis, and/or high levels of alanineaminotransferase or aspartate aminotransferase at baseline

• Low estimated glomerular filtration rate, and/or significant high urinaryalbumin/creatinine ratio at screening or on chronic dialysis.

• Newly diagnosed folate deficiency anemia, which is considered clinically relevant bythe Investigator at screening

• Any history or clinically important finding of cardiac or pulmonary disorders

• Family history of long-QT syndrome or sudden death without a preceding diagnosis ofa condition that could be causative of sudden death

• Clinically significant bacterial, fungal, parasitic, or viral infection whichrequires therapy. Note: A subject meeting this criterion should delay screeningand/or enrolment for a minimum of 2 weeks, or if excluded can be re-screened at alater time point.

• Concomitant use of certain hormonal contraceptives as defined in the study protocol,are not allowed within 4 weeks prior to screening and until 1 week after the lastadministration of the study drug and the use of progesterone-only hormonalcontraception as the sole measure to prevent pregnancy.

• Pregnant or females currently breastfeeding.

• History or known concomitant solid tumours and/or haematological malignancies unlessresolved in the ≥2 past years, except for basal or squamous cell carcinoma of theskin, carcinoma in situ of the cervix or breast, incidental histologic finding ofprostate cancer

• Unable to take and absorb oral medications

• Acute peptic stomach or duodenal ulcer in the previous 6 months before screeningand/or healed after 3 months of treatment.

• Uncontrolled hemorrhages