N Engl J Med
Investigational oral drug safely reduces nosebleeds in hereditary hemorrhagic telangiectasia
In a multicenter, double-blind trial (NCT05406362), 75 adults with hereditary hemorrhagic telangiectasia (HHT) received oral engasertib (30 mg or 40 mg) or placebo daily for 12 weeks. Safety was comparable across groups, except for mild-to-moderate rash (21% at 30 mg; 42% at 40 mg) and reversible hyperglycemia (12% at 40 mg). Epistaxis duration decreased by 29.9% (30 mg), 41.4% (40 mg), and 23.8% (placebo); frequency reductions were similar. An open-label extension is ongoing.
Source:
Al-Samkari H, et al. (2025, November 26). N Engl J Med. Engasertib versus Placebo for Bleeding in Hereditary Hemorrhagic Telangiectasia. https://pubmed.ncbi.nlm.nih.gov/41297007/