Fulcrum Therapeutics announced positive initial results from its Phase 1b trial of pociredir for sickle cell disease. In the 20mg dose cohort, patients saw a significant increase in hemoglobin F (HbF) levels, with an average increase of 9.9% at six weeks. This represents a substantial improvement over baseline levels, which ranged from 5-7%. Additionally, more than 50% of patients achieved HbF levels above 20%, and there was evidence of early progression toward pan-cellular HbF induction. The company also reported reduced VOC (vascular occlusion) frequency compared to the period before treatment.
The Phase 1b trial found a clear dose-response relationship, with increased mean HbF induction at higher doses. This suggests pociredir may be effective in increasing hemoglobin levels and reducing VOCs in patients with sickle cell disease. The results are promising for Fulcrum Therapeutics and its lead candidate, pociredir.
Source: https://www.tipranks.com/news/the-fly/fulcrum-announces-results-from-20-mg-dose-cohort-of-phase-1b-pioneer-trial-thefly?mod=news_archive