Fulcrum Therapeutics Announces Preclinical Proof-of-Concept Data for FTX-6058 at the Virtual 14th Annual Sickle Cell Disease Research & Educational Symposium and 43rd National Sickle Cell Disease Scientific Meeting

- Fetal Hemoglobin expression in human cellular models increased up to ~30% by FTX-6058 for the potential treatment of sickle-cell disease

- Company plans to initiate Phase 1 trial in healthy volunteers by year-end

- Non-provisional composition of matter patent application covering FTX-6058 published

CAMBRIDGE, Mass., Sept. 25, 2020 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on improving the lives of patients with genetically defined rare diseases, today announced preclinical proof-of-concept data supporting the development of FTX-6058 to treat sickle cell disease and beta-thalassemia. FTX-6058, a small molecule designed to increase expression of fetal hemoglobin, demonstrated target engagement and good tolerability in multiple preclinical rodent models with once-a-day oral dosing. The Company presented these data today at the 14^th Annual Sickle Cell Disease Research & Educational Symposium and 43^rd National Sickle Cell Disease Scientific Meeting being held virtually. Slides from the presentation will be available on Fulcrum’s website at ir.fulcrumtx.com/events-and-presentations.

“Despite newly approved therapies for sickle cell disease, a significant unmet need remains,” said Martin H. Steinberg, MD, Professor of Medicine at Boston University School of Medicine. “An orally available small molecule therapeutic acting through a novel mechanism to induce increased pancellular HbF should be an important disease-modifying agent.”

Fetal Hemoglobin (HbF) is a key modulator of sickle cell disease. Increasing HbF levels has the potential to prevent or reduce disease-related pathophysiology, resulting in reduction of recurring events such as vaso-occlusive crises (VOCs) and hemolysis. In some cases, sickle cell patients with high HbF levels have asymptomatic disease, underscoring the protective effect of HbF. Fulcrum has identified FTX-6058, a highly potent small molecule inhibitor of Embryonic Ectoderm Development (EED) capable of inducing robust HbF protein expression in cell and murine models. Additionally, Fulcrum believes that pharmacokinetics and human dose simulations support FTX-6058 may be given as a once daily oral compound.