Fulcrum Therapeutics Shares Additional Data From Phase 1 Trial Of Sickle Cell Disease Treatment

Fulcrum Therapeutics Shares Additional Data From Phase 1 Trial Of Sickle Cell Disease Treatment

Fulcrum Therapeutics Inc FULC has announced results from the 20mg and 30mg dose cohorts in healthy adult volunteers in its Phase 1 trial of FTX-6058 for sickle cell disease and other hemoglobinopathies, such as beta-thalassemia.

  • The Company also shared new preclinical mechanism data showing that FTX-6058 downregulated known repressors of fetal hemoglobin (HbF).
  • 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. 
  • The Company remains on track to begin enrolling people with sickle cell disease in Phase 1b trial by year-end 2021 to report initial data, including HbF protein levels, in Q2 of 2022.
  • Fulcrum also announced new preclinical data demonstrating a new mechanism of potent downregulation of BCL11A and MYB, key repressors of fetal hemoglobin.
  • Related Link: Fulcrum's Candidate For Inherited Blood Disorders Shows Proof Of Mechanism, Biology In Early-Stage Study.
  • Price Action: FULC shares are down 6.81% at $13.06 during the market session on the last check Monday.

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