Press release
Fulcrum Therapeutics® Announces Additional HBG mRNA Induction from Higher Dose Cohorts in Phase 1 Healthy Adult Volunteer Trial of FTX-6058 for Sickle Cell Disease and New Preclinical Mechanism Data
Achieved mean 5.6-fold HBG mRNA induction at 20mg and mean 6.2-fold at 30mg after 14 days of once-daily dosing, further supporting potential of FTX-6058 to
About this update from Fulcrum Therapeutics, Inc.
[{"type":"text","content":"Achieved mean 5.6-fold HBG mRNA induction at 20mg and mean 6.2-fold at 30mg after 14 days of once-daily dosing, further supporting potential of FTX-6058 to provide a functional cure Continues to be well-tolerated at higher doses with no serious adverse events observed to date New mechanism data demonstrate potent downregulation of BCL11A and MYB, key repressors of fetal hemoglobin On track to initiate enrollment in Phase 1b clinical trial in people with sickle cell disease and to submit an IND for treatment of other hemoglobinopathies by year-end 2021 Company to review results on conference call, including guest KOL Dr. Gerd Blobel, at 8:00 am ET today CAMBRIDGE, Mass., Dec. 06, 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 announced positive results from the 20mg and 30mg dose cohorts in healthy adult volunteers in its Phase 1 clinical trial of FTX-6058. The company also shared new preclinical mechanism data showing that FTX-6058 downregulated known repressors of fetal hemoglobin (HbF). FTX-6058 is an investigational oral HbF inducer that is being developed for the treatment of sickle cell disease and other hemoglobinopathies, such as beta-thalassemia. Data from the 20mg and 30mg dose cohorts demonstrated a mean 5.6-fold induction and a mean 6.2-fold induction in HBG mRNA, respectively, at day 14. These increases were higher than those observed in the previously reported 2, 6 and 10mg dose cohorts. In preclinical studies of FTX-6058, increases in HBG mRNA have consistently translated to the same fold increases in HbF protein. Notably, human genetics show that 2-3-fold increases in HbF are associated with significantly improved outcomes, and even functional cures, in people with sickle cell disease. FTX-6058 has now demonstrated greater than a mean 2-fold induction starting with the 6mg dose. “Despite progress in the treatment of sickle cell disease, existing therapies either offer limited benefit or, in the case of gene therapy, are not amenable to the great majority of patients and carry certain risks,” said Gerd Blobel, MD, PhD, Frank E. Weise III Endowed Chair in Pediatric Hematology at Children’s Hospital of Philadelphia. “The strategy of increasing the levels of fetal hem...