HIBISCUS

Title of Protocol:

An Adaptive, Randomized, Placebo-controlled, Double-blind, Multi-center Study of Oral FT-4202, a Pyruvate Kinase Activator in Patients with Sickle Cell Disease

Purpose/Objective of Study:

The primary purpose of this study is to assess the efficacy and safety of FT-4202 in subjects with sickle cell disease as compared to placebo as measured by improvement in hemoglobin.

Study Agent(s) and Mechanism of Drug Action/Device Description:

FT-4202 is the investigational drug for this clinical trial. FT-4202 may help treat sickle cell disease by acting to lower the rate of red blood cell sickling.

Toxicities/Side Effects:

Possible side effects are headaches.

Time Committment: 52 weeks

VIT-2763-SCD-202

Title of Protocol:

A Phase 2a, double-blind, randomised, placebo-controlled, ascending dose and maintenance dose, efficacy, and safety study of multiple doses of VIT-2763 in subjects with sickle cell disease.

Purpose/Objective of Study:

The primary purpose of this study is to explore the effect of VIT-2763 on markers of haemolysis vs placebo. VIT-2763 is the investigational drug for this clinical trial.

Study Agent(s) and Mechanism of Drug Action/Device Description:

VIT-2763 is the investigational drug for this clinical trial. VIT-2763 directly targets and blocks the iron transporter protein in the body called ferroportin. Iron is important for the production of hemoglobin, the protein that transports oxygen in red blood cells. It is expected that, by blocking ferroportin, VIT-2763 will lower the iron level in your blood, which might reduce the concentration of diseased hemoglobin (e.g., HbS) in your red blood cells. Subsequently, this reduction can prevent hemolysis (destruction of red blood cells) and pain crisis in patients with SCD.

Toxicities/Side Effects:

Possible side effects are headache, decrease in white blood cells, decrease in the number of neutrophils, somnolence, and dizziness.

Time Committment: Approximately 16 weeks