Benzinga | Dec 6, 2021 07:03AM EST

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 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