Forma Therapeutics Holdings, Inc. announced new data from its ongoing randomized, placebo-controlled, multi-center Phase 1 trial of FT-4202 in patients with sickle cell disease (SCD) that further support the development of this novel investigational agent, a selective red blood cell (RBC) pyruvate kinase-R (PKR) activator, as a potential disease-modifying therapy. Data previously presented at the 2020 American Society of Hematology (ASH) Annual Meeting were based on the first cohort of patients in the Phase 1 trial dosed with 300 mg of FT-4202 or placebo once daily for 14 days and a 7-day follow up period. The new findings include an analysis of the blinded data from the second cohort of patients randomly assigned to receive 600 mg of FT-4202 or placebo once daily for 14 days and a 7-day follow up period. Aggregate findings from the placebo-controlled cohorts of the Phase 1 trial demonstrated 10 of 14 patients (71%) who received FT-4202 achieved a hemoglobin increase of greater than or equal to 1 g/dL from baseline with once-daily dosing of FT-4202 during 14 days of treatment. Based on a trend toward increasing response over the treatment period, the potential exists for additional benefit when dosing beyond 14 days; this is being explored in the ongoing open label extension, which is dosing patients at 400 mg daily for 12 weeks. While the data from the 600 mg cohort of patients remain blinded, initial analysis of the cohort suggests FT-4202 has a similar safety and tolerability profile as the 300 mg cohort, despite the doubling of the dose. No dose-limiting toxicities or treatment-related adverse events (AE) were reported, and the overall AE profile of the 600 mg cohort was consistent with the 300 mg cohort. Unblinded 600 mg cohort data are expected to be reported at an upcoming medical conference in Summer 2021, in addition to initial results from the ongoing open label extension. The blinded, randomized, placebo-controlled portion of the ongoing Phase 1 study is now complete. People with SCD are now directly enrolling into the 12-week open label cohort receiving 400 mg of FT-4202 daily.