A new study found that a drug specifically designed to target hereditary hemorrhagic telangiectasia (HHT) was safe and effective.
HHT is the “second most common inherited bleeding disorder worldwide, affecting 1 in 3,800 persons. HHT’s hallmark symptom is chronic nosebleeds, which often occur alongside other internal bleeding and vascular malformations that impact quality of life and longevity.” The disorder causes “serious vascular abnormalities throughout the body, often leading to dangerous, abnormal blood vessels in the brain, lungs, and liver, which bring with them major complications including stroke, heart failure, and brain hemorrhage.”
The drug, engasertib, targets a protein called AKT that is made in excess in people with HHT. The drug was evaluated in a study with 75 participants, who were split into three groups – one receiving 30mg of engasertib, one receiving 40mg of engasertib, and one receiving a placebo. The patients who received the drug “experienced fewer and shorter nosebleeds relative to the placebo group. Additionally, 61% of the 40-milligram group and 37% of the 30-milligram group reported feeling ‘much better’ at the end of 12 weeks, while only 27% of the placebo group said the same.”
The drug was deemed safe. The most common side effect was a “mild and reversible rash” and serious adverse events did not differ significantly between the treatment and placebo groups.
