Fulcrum has positive interim results in Phase 1 sickle cell trial

 

• 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

Fulcrum will host a conference call and webcast today at 8:00 am ET to discuss these results and the company’s second quarter financial results. The webcast will be accessible through the Investor Relations section of Fulcrum’s website at www.fulcrumtx.com. Following the live webcast, an archived replay will also be available.

Dial-in Number
U.S./Canada Dial-in Number: 800-527-6973
International Dial-in Number: 470-495-9162
Conference ID: 3291056

Replay Dial-in Number: 855-859-2056
Replay International Dial-in Number: 404-537-3406
Conference ID: 3291056

https://finance.yahoo.com/news/fulcrum-therapeutics-announces-positive-interim-110000744.html