FORMA Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on rare hematologic diseases and cancers, announced the U.S. Food and Drug Administration (FDA) has granted Fast Track designation and Rare Pediatric Disease designation for its lead clinical asset, FT-4202, in development as a disease-modifying treatment for sickle cell disease (SCD). FT-4202 is a selective pyruvate kinase-R (PKR) activator that exhibits the potential to beneficially impact both anemia and vaso-occlusive crises for people living with SCD.
“These designations for FORMA’s lead clinical asset underscore the FDA’s continued recognition of the needs of patients with SCD,” said Frank Lee, chief executive officer of FORMA Therapeutics. “With more than 100,000 individuals living with SCD in the U.S., and with relatively few treatment options, we believe significant unmet medical needs persist. These FDA designations give FORMA the opportunity to accelerate the development of a new treatment for people impacted by SCD.”
FORMA is currently enrolling patients with SCD in a Phase 1 study to evaluate the safety and pharmacokinetics/pharmacodynamics (PK/PD) of FT-4202. FORMA plans to initiate a registrational trial within the next year. For more information on eligibility and study sites for the open Phase 1 study, please visit clinicaltrials.gov/NCT03815695.