Sotatercept was associated with a substantial reduction in serious clinical events among high-risk patients with pulmonary arterial hypertension, according to a recent study.
Researchers conducted a phase III randomized, double-blind, placebo-controlled trial that investigated sotatercept in patients with pulmonary arterial hypertension (PAH) who were at high risk for death. The study was published in The New England Journal of Medicine.
The trial enrolled 172 patients with WHO functional class III or IV PAH and a REVEAL Lite 2 risk score of nine or greater. Patients were randomly assigned in a 1:1 ratio to receive sotatercept, initiated at 0.3 mg/kg and escalated to 0.7 mg/kg every 3 weeks, or placebo, in addition to stable background therapy. The primary end point evaluated the time to the first occurrence of lung transplantation, hospitalization of at least 24 hours due to worsening PAH, or death from any cause.
The trial—led by Marc Humbert of Université Paris-Saclay, Hôpital Bicêtre (Assistance Publique–Hôpitaux de Paris), and the European Reference Network for Rare Respiratory Diseases, Le Kremlin-Bicêtre in France—was stopped early following an interim analysis that demonstrated efficacy. A primary end point event occurred in 17.4% of patients who received sotatercept compared with 54.7% of those who received placebo. Death from any cause occurred in 8.1% of the sotatercept group and 15.1% of the placebo group. Lung transplantation occurred in 1.2% and 7% of patients, respectively. Hospitalization for worsening PAH was reported in 9.3% of patients in the sotatercept group compared with 50% in the placebo group.
Serious adverse events were reported in 53.5% of patientswho received sotatercept and 64% of those who received placebo. Adverse events that were more common with sotatercept included epistaxis, which occurred in 44.2% of patients vs 9.3% in the placebo group, and telangiectasia, which occurred in 25.6% vs 3.5%, respectively. Researchers concluded that sotatercept reduced the risk of major clinical outcomes in patients with advanced PAH who were at high risk for death when added to background therapy.
Full disclosures can be found in the published study.