- Fulcrum Therapeutics Inc FULC has announced positive interim results from the Phase 1 trial with FTX-6058 in healthy adult volunteers.
- FTX-6058 is a potent and selective small-molecule inhibitor of EED designed to increase fetal hemoglobin (HbF) expression with the potential to treat hemoglobinopathies, such as sickle cell disease and beta-thalassemia.
- Results from the MAD portion of the trial demonstrated proof of biology as evidenced by a dose-proportional induction in HBG mRNA and accompanying increases in HbF-containing reticulocytes (F-reticulocytes).
- At 10mg, the highest dose studied to date, the mean changes were 4.5-fold and 4.2-fold, respectively.
- Additionally, all FTX-6058 doses in the MAD portion of the trial achieved maximal target engagement by day seven in 6mg and 10mg cohorts.
- Fulcrum plans to initiate a Phase 1b trial enrollment in sickle cell patients in 4Q 2021 and submit Investigational New Drug (IND) application in non-sickle cell hemoglobinopathies by year-end 2021.
- As of 30 June, cash and marketable securities were $125.6 million, which will be sufficient to fund Fulcrum's expenses into Q1 of 2023.
- Price Action: FULC shares are up 60.90% at $13.50 during the premarket session on the last check Tuesday.
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