Robert Clark Brown, MD, PhD, Children’s Healthcare of Atlanta, Atlanta, GA, gives an update on a randomized Phase I study (NCT03815695) of FT-4202 versus placebo in patients with sickle cell disease (SCD). FT-4202 is a small molecule activator of erythrocyte pyruvate kinase (PKR), which acts to decrease 2,3-DPG and increase ATP in red blood cells. This Phase I study aims to evaluate the safety, pharmacokinetics, and pharmacodynamics of FT-4202 in healthy volunteers and patients with SCD. Treatment-related adverse events to FT-4202 included nausea and headache. All adverse events in patients treated with FT-4202 were less than or equal to grade two. Dr Brown reports that of eight patients enrolled on the 8-week open-label extension, 7/8 have reached the target of increasing their hemoglobin to greater than 1g per dL, with a mean increase of 1.5g per dL above baseline. This interview took place at the virtual European Hematology Association (EHA) Congress 2021.
Robert Clark Brown, MD, PhD, has participated in consultancy work with Global Blood Therapeutics, Imara and Novo Nordisk; and has received research support from Forma Therapeutics, Global Blood Therapeutics, Imara and Novartis.