Sotatercept is becoming a treatment option for patients with newly diagnosed pulmonary arterial hypertension

Sotatercept versus placebo showed a reduction in clinical deterioration in patients with newly diagnosed pulmonary arterial hypertension (PAH). However, this was associated with an increased incidence of bleeding events. 

HYPERION (NCT04811092) is a randomised, double-blind, phase 3 study of sotatercept versus placebo in participants with symptomatic, newly diagnosed PAH at moderate-to-high risk of death. The primary endpoint was the time-to-clinical worsening (a composite of the first occurrence of all-cause death, PAH-related hospitalisation, atrial septostomy, lung transplantation, or PAH deterioration). A total of 320 participants were enrolled.

Prof. Vallerie McLaughlin (University of Michigan, MI, USA) presented the results. The primary endpoint was met for sotatercept versus placebo, with a 76% reduction in time-to-clinical worsening (HR 0.24; 95% CI 0.14-0.41; P<0.0001). The benefit was consistent across subgroups characterised by age (<65 vs ≥65 years), sex, type of PAH, baseline WHO functional class (II vs III), and COMPERA 2.0 risk score. Discontinuation rates were low (3.1% with sotatercept vs 0% with placebo). Adverse events were more common with sotatercept, including bleeding events (41.2% vs 16.2%), telangiectasia (26.2% vs 11.2%), increased haemoglobin (11.2% vs 1.2%), and severe bleeding events (3.8% vs 1.9%) [1].

“Sotatercept initiated within the first year of diagnosis and shortly after initiation of background therapy reduced clinical worsening events by 76%,” concluded Prof. McLaughlin. “HYPERION expands the evidence-based positive risk-benefit of sotatercept from patients with prevalent PAH to patients with a new diagnosis.”

1. McLaughlin V, et al. Efficacy and safety of sotatercept in patients with newly diagnosed pulmonary arterial hypertension at intermediate-to-high risk of mortality: Results from Phase 3 HYPERION study. ERS Congress, 27 September–1 October 2025, Amsterdam, the Netherlands.

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Posted on 30 October 202530 October 2025