Sotatercept for Pulmonary Artery Hypertension

What is the role of sotatercept in patients with PAH?

Study Design

Multi-center
Double blinded
Randomized Controlled Trial (RCT)

Population

323 patients (256 female, 67 male) with PAH receiving stable background therapy, excluding PAH subtypes associated with portopulmonary disease, schistosomiasis, HIV infection, or VOD.

Interventions

Sotatercept: N=163, starting dose of 0.3 mg/kg with a target dose of 0.7 mg/kg every 3 weeks
Placebo: N=160, matching placebo every 3 weeks

Primary Outcome

Significant increase in improvement in the median 6-minute walk distance at week 24 (34.4 m vs. 1 m; AD 33.4 m, 95% CI 13.58 to 53.22)

Secondary Outcomes

6-minute walk distance, NT-proBNP level, and WHO functional class: 38.9% vs. 10.1%; AD 28.8%, 95% CI 11.71 to 45.89
Pulmonary vascular resistance reduction: 165.1 dyn.sec.cm-5 vs. 32.8 dyn.sec.cm-5; AD 197.9 dyn.sec.cm-5, 95% CI 80.47 to 315.33
NT-proBNP level reduction: 230.3 pg/mL vs. -58.6 pg/mL; AD 288.9 pg/mL, 95% CI 117.48 to 460.32

Safety Outcomes

No significant difference in adverse events.

Conclusion

In adult patients with PAH who were receiving stable background therapy, sotatercept was superior to placebo with respect to improvement in the median 6-minute walk distance at week 24.