Fulcrum Therapeutics is advancing its oral therapy, pociredir, for sickle cell disease (SCD) in the PIONEER #trial (NCT05169580). The study is testing safety, effectiveness, and how the drug works in the body. After positive early data, enrollment is now open for the 20 mg dose group across U.S. and global sites, including Nigeria and South Africa. Earlier results showed dose-dependent increases in fetal hemoglobin with no serious side effects. CEO Alex Sapir called the progress “encouraging,” with full 12 mg cohort data expected mid-2025. Patient retention remains high, with 90%+ adherence. If successful, pociredir could become a once-daily oral option for SCD patients who don’t respond to current therapies.

OxyDial supports innovation for underserved SCD communities, where new treatments are urgently needed. Pociredir’s approach of reactivating fetal hemoglobin is a promising way to improve the outcomes for SCD #patients in dire need of high-quality #treatment.

Read more: https://sicklecellanemianews.com/news/sickle-cell-disease-patients-being-recruited-pioneer-trial-pociredir/

#SickleCell #SCD #RareDisease #Pociredir #Hemoglobinopathies