A long-term follow-up study of sotatercept for treatment of pulmonary arterial hypertension: interim results of SOTERIA
Ioana R. Preston, David B. Badesch, Hossein-Ardeschir Ghofrani, J. Simon R. Gibbs, Mardi Gomberg‐Maitland, Marius M. Hoeper, Marc Humbert, Vallerie V. McLaughlin, Aaron B. Waxman, Solaiappan Manimaran, Elina Mikhailova, Madhavi Reddy, Anna Lau, Janethe de Oliveira Pena, Rogério Souza
Abstract
BACKGROUND: SOTERIA (ClinicalTrials.gov: NCT04796337) is an ongoing open-label study evaluating long-term safety, tolerability and efficacy of sotatercept in participants with pulmonary arterial hypertension (PAH). METHODS: once every 21 days). Safety and tolerability (primary objective) were assessed by adverse events (AEs), vital signs and laboratory assessments. Efficacy (secondary objective) was assessed by 6-min walk distance (6MWD), N-terminal pro-brain natriuretic peptide (NT-proBNP) levels, World Health Organization (WHO) Functional Class, clinical worsening events and simplified French risk score (SFRS). The data cut-off date was 8 November 2023. RESULTS: Altogether, 426 participants were included in the analyses. Mean±sd duration of exposure to sotatercept and follow-up in SOTERIA was 448.6±172.93 days (range 21-923 days; 523 patient-years). Of 426 participants, 387 (90.8%) experienced AEs, 15 (3.5%) discontinued treatment, 129 (30.3%) had serious AEs and 11 (2.6%) had serious AEs related to treatment. There were 12 deaths (2.8%). Among AEs of interest, epistaxis (22.1%) and telangiectasia (16.9%) were the most frequently reported individual events. 22 (5.2%) participants had serious bleeding events, including two (0.5%) with serious bleeding leading to death (not related to treatment by investigator judgement). Improvements in 6MWD, NT-proBNP, WHO Functional Class and SFRS achieved from baseline of SOTERIA were largely maintained at 1 year, including in the placebo-crossed group. CONCLUSION: Interim results of SOTERIA support the favourable benefit-risk of add-on sotatercept treatment in adults with PAH. Follow-up reports from this study will provide additional information on benefit-risk.