Forma Therapeutics Presents Clinical Proof-of-Concept Data at the 62nd Annual ASH Meeting Supporting the Potential of its Novel Investigational PKR Activator, FT-4202, to Treat Sickle Cell Disease (SCD)

WATERTOWN, Mass.--(BUSINESS WIRE)--Forma Therapeutics Holdings, Inc. (Nasdaq: FMTX), a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, today announced that clinical proof-of-concept in patients with sickle cell disease (SCD) has been observed in the ongoing randomized, placebo-controlled, multi-center Phase 1 trial of FT-4202. FT-4202 is a novel, investigational, selective red blood cell (RBC) pyruvate kinase-R (PKR) activator in development as a potential disease-modifying therapy for SCD. In a cohort of nine patients, six of seven patients (86%) who received FT-4202 achieved a hemoglobin increase of greater than 1 g/dL from baseline with once-daily dosing at 300 mg for 14 days. The data, being presented today at the 62nd American Society of Hematology (ASH) Annual Meeting, also demonstrate improvements in hemolytic parameters showing that activation of PKR by FT-4202 decreases reticulocyte counts and sickle RBC hemolysis in patients with SCD.