Engasertib versus Placebo for Bleeding in Hereditary Hemorrhagic Telangiectasia

The safety profile of engasertib was similar to that of placebo except for mild-to-moderate rash, which resolved in most patients who continued to receive the drug. Engasertib treatment was associated with decreases in epistaxis frequency and duration. (Funded by Vaderis Therapeutics; ClinicalTrials.gov number, NCT05406362.).