Benzinga | Aug 10, 2021 07:08AM EDT

Fulcrum Therapeutics Announces Positive Interim Results from Phase 1 Healthy Adult Volunteer Trial of FTX-6058 for Sickle Cell Disease

* Achieved dose proportional pharmacodynamic changes in HBG mRNA and F-reticulocytes in whole blood Mean 4.5-fold induction in HBG mRNA at 10mg after 14 days of once-daily dosing Mean 4.2-fold increase in F-reticulocytes at 10mg after 14 days of once-daily dosing

* Achieved maximal target engagement by day seven in 6mg and 10mg cohorts

* FTX-6058 was generally well tolerated with no serious adverse events observed to date

* Company plans to initiate enrollment in a Phase 1b clinical trial in sickle cell patients in 4Q 2021

* Company plans to submit Investigational New Drug (IND) application in non-sickle cell hemoglobinopathies (e.g., beta-thalassemia) by year-end 2021

* Company to review clinical results on second quarter earnings call today at 8:00am ET