Press release
Fulcrum Therapeutics Presents Published Structure of Investigational Small Molecule FTX-6058 at the American Chemical Society (ACS) Spring 2021 Virtual Conference
Company initiates dosing in Phase 1 healthy volunteer multiple ascending dose (MAD) cohort CAMBRIDGE, Mass., April 09, 2021 (GLOBE NEWSWIRE) -- Fulcrum
About this update from Fulcrum Therapeutics, Inc.
[{"type":"text","content":"Company initiates dosing in Phase 1 healthy volunteer multiple ascending dose (MAD) cohort\nCAMBRIDGE, Mass., April 09, 2021 (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 presented the medicinal chemistry strategy for FTX-6058 at the First Time Disclosure Session at the American Chemical Society (ACS) Spring 2021 National Meeting. FTX-6058 is a highly potent orally bioavailable small molecule EED inhibitor for the potential treatment of select hemoglobinopathies, including sickle cell disease and β-thalassemia. The validation of EED as a fetal hemoglobin (HbF) inducer target for sickle cell disease was conducted using FulcrumSeek, Fulcrum’s proprietary product engine. “We are pleased to report progress on our development of FTX-6058 including the first publication of the structure of this compelling EED inhibitor,” said Chris Moxham, Ph.D., Fulcrum’s chief scientific officer. “We believe that this oral, once-a-day therapy with an impressive preclinical pharmacological profile has the potential to provide a meaningful therapeutic benefit to patients with sickle cell disease and β-thalassemia. We are also excited to report initial PK results from the SAD cohort and that our Phase 1 trial in healthy volunteers continues to progress with initiation of the multiple ascending dose cohorts. We expect to report the full data from this Phase 1 trial mid-year.” FTX-6058 inhibits PRC2 via binding to EED, which induces robust HbF protein expression in both cell and murine models. Increasing HbF has the potential to prevent or reduce disease-related pathophysiology and reduce the risk of recurring events such as vaso-occlusive crises and hemolysis. Preclinical data with FTX-6058 showed an increase in HbF levels up to approximately 30% of total hemoglobin, demonstrating the potential to have a significant impact in patients with sickle cell disease. Human genetic data further indicate that individuals with the sickle cell mutation and high HbF levels may have asymptomatic disease, underscoring the protective effect of increased HbF. Fulcrum’s Phase 1 trial in healthy volunteers is evaluating the safety, tolerability and pharmacokinetics of FTX-6058. Dosing has been initiated in the randomized, do...