Oral Engasertib Safe for Hereditary Hemorrhagic Telangiectasia

THURSDAY, Dec. 4, 2025 (HealthDay News) -- For adult patients with hereditary hemorrhagic telangiectasia (HHT), a new, allosteric, selective activin receptor-like kinase 1 inhibitor, oral engasertib, is safe and is associated with decreases in epistaxis frequency and duration, according to a study published in the Nov. 27 issue of the New England Journal of Medicine.Hanny Al‑Samkari, M.D., from Massachusetts General Hospital in Boston, and colleagues examined the safety and efficacy of oral engasertib in patients with HHT. A total of 75 adult patients were randomly assigned to receive engasertib 30 mg, engasertib 40 mg, or placebo (24, 25, and 26 patients, respectively) once daily for 12 weeks.The researchers found that the most common on-target adverse events associated with engasertib included mild-to-moderate rash (21, 42, and 8 percent of patients in the 30-mg engasertib group, 40-mg engasertib group, and placebo group, respectively) and mild-to-moderate hyperglycemia (12 percent in the 40-mg engasertib group and no patients in the other groups); these events were reversible. The incidence of serious adverse events was similar in each of the two engasertib groups and the placebo group. The mean decrease in epistaxis frequency from baseline to week 12 was 26.5 ± 26.5, 27.8 ± 35.1, and 18.0 ± 36.0 percent, respectively, in the 30-mg engasertib group, 40-mg engasertib group, and placebo group; the corresponding decreases in epistaxis duration were 29.9 ± 53.2, 41.4 ± 41.0, and 23.8 ± 53.4 percent."The observed improvements in epistaxis variables with engasertib treatment in the double-blind period and the open-label extension are encouraging and support the continued development of engasertib to address the need for a therapy in these patients," the authors write.Several authors disclosed ties to biopharmaceutical companies, including Vaderis Therapeutics, which is developing engasertib and funded the study.Abstract/Full Text (subscription or payment may be required).Sign up for our weekly HealthDay newsletter

Oral Engasertib Safe for Hereditary Hemorrhagic Telangiectasia

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