Fulcrum’s rise suggests high interest in small molecule sickle cell therapies

While newer modalities from gene editing to gene silencing are making headway for hemoglobinopathies, investor enthusiasm for Fulcrum’s Phase I sickle cell disease program indicates that there is still high demand for oral small molecule options. 

Fulcrum Therapeutics Inc. (NASDAQ:FULC) gained 133% to $19.38 in mid-day trading Tuesday after reporting interim data from a Phase I trial of FTX-6058 in healthy volunteers showing the EED inhibitor led to a dose-dependent increase in HBG mRNA and fetal hemoglobin-containing reticulocytes (immature red blood cells). In the multiple ascending dose portion of the study, participants received oral FTX-6058 once-daily for 14 consecutive days. ...