Patients with heart failure with preserved ejection fraction (HFpEF) and pulmonary hypertension experienced significant improvements after taking the drug sotatercept, according to a study presented at the American College of Cardiology's Annual Scientific Session on Mar. 29.
This development is important because there are currently no approved therapies for Group 2 pulmonary hypertension, including combined post and precapillary pulmonary hypertension (CpcPH) associated with HFpEF. These conditions can cause severe symptoms such as shortness of breath, fatigue, and swelling due to increased pressure in the lungs and impaired heart function.
The Phase 2 trial enrolled 164 patients averaging 75 years old, most of whom were female. Participants received either two different doses of sotatercept or a placebo over a period of 24 weeks. Researchers measured changes in pulmonary vascular resistance as the primary outcome. The results showed that sotatercept led to a statistically significant reduction in this resistance compared to placebo. Patients also demonstrated better six-minute walking distances, improved right heart function, and fewer clinical worsening events.
Mardi Gomberg, MD, MSc, lead author of the study and professor at George Washington School of Medicine and Health Sciences said: "Patients and the scientific community have been waiting a long time for a treatment that shows benefit in CpcPH associated with HFpEF, as previous trials have failed and there are no approved therapies." Gomberg added: "It's really exciting because we see improvements in heart failure clinical markers as well as biomarkers of heart function in both the right side and the left side. The left atrial volume and left atrial pressure (measured as wedge pressure) improved, features that can put you at risk for a variety of bad outcomes with HFpEF. Seeing these improvements in addition to improvements in the pulmonary vasculature makes this a big deal."
The safety profile was favorable across both doses tested; initial data suggest that lower doses may offer an optimal balance between benefits and risks for this patient group. Some participants who had previously undergone valve repair procedures also benefited from sotatercept without increased risk.
Because this was a Phase 2 study with limited sample size and duration (24 weeks), researchers plan further studies to assess long-term safety and effectiveness through an upcoming Phase 3 trial.
