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This randomized, double-blind, placebo-controlled phase 1b trial of engasertib (an oral AKT1/2 inhibitor) in patients with HHT with moderate-to-severe epistaxis, showing dose-dependent reductions in epistaxis duration and frequency over 12 weeks, with further improvement through 12 months in the open-label extension. The 40 mg dose reduced monthly epistaxis duration by 41.4% and frequency by 27.8% vs placebo, and increased epistaxis-free days by 4.6/month. Safety was manageable and most notably, no thromboembolic signals were observed. Larger studies are needed, but this could be a game changer for our HHT patients sent to us for management of bleeding and anemia.