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– In a multicenter, double-blind, placebo-controlled proof-of-concept trial (NCT05406362), oral engasertib (30 mg or 40 mg daily), an allosteric selective AKT inhibitor, was evaluated for safety and effects on epistaxis in patients with hereditary hemorrhagic telangiectasia (HHT).
– Engasertib produced modest mean reductions in epistaxis frequency (26.5% with 30 mg; 27.8% with 40 mg) and duration (29.9% with 30 mg; 41.4% with 40 mg) over 12 weeks versus 18.0% and 23.8% reductions with placebo, respectively; variability was large and formal statistical comparisons were not reported in the summary.
