Long-term treatment of patients with pulmonary arterial hypertension (PAH) with sotatercept for 12 to 18 months proved efficacious and safe, according to study findings presented at the American Thoracic Society (ATS) 2022 International Conference, held in San Francisco, CA, May 13 to 18.
Sotatercept, a reverse-remodeling agent, rebalances the anti-proliferative and pro-proliferative signaling imbalance in PAH that results in the hyperproliferation of vessel wall cells. Treatment of adult patients with PAH with sotatercept was found to be safe and effective over a 24-week period in the phase 2, randomized, double-blind PULSAR study (ClinicalTrials.gov Identifier: NCT03496207) In the current study, an open-label extension (OLE) study following the placebo-controlled treatment period of PULSAR, researchers sought to determine the long-term effectiveness and safety of sotatercept among patients with PAH.
Of the 97 participants who completed the 24-week PULSAR treatment period, those previously treated with sotatercept continued with the same dose (continued cohort), while those in the placebo cohort were rerandomized into sotatercept 0.3mg/kg or 0.7mg/kg placebo-crossed cohorts, with sotatercept administered subcutaneously every 3 weeks for 18 to 24 months.
From baseline to extension study conclusion, placebo-crossed cohorts showed statistically significant gains in pulmonary vascular resistance (PVR) and 6-minute walk distance (6MWD) tests, and 55% of patients showed gains in World Health Organization functional class (WHO FC). Previous gains were preserved or enhanced in the continued cohort in the PVR and 6MWD tests, and 41% of patients showed gains in the WHO FC. Treatment-emergent adverse events were reported in more than 40% of the participants (32 serious; 11 nonserious; 10 forced to leave the study; 3 died [for reasons considered unrelated to the study]).
Researchers said their study showed the clinical value of long-term use of sotatercept among patients with PAH. They concluded that “significant clinical improvement across multiple endpoints was achieved following 12-18 months of sotatercept treatment in previous placebo participants; clinical efficacy was maintained or further enhanced in those with longer-term use.”
Disclosure: Some study authors declared affiliations with biotech, pharmaceutical, and/or device companies. Please see the original reference for a full list of authors’ disclosures.
Humbert M, Mclaughlin VV, Gibbs SR, et al. PULSAR open-label extension: long-term efficacy and safety of sotatercept for the treatment of pulmonary arterial hypertension (PAH). Presented at: the American Thoracic Society (ATS) 2022 International Conference; May 13-18, 2022; San Francisco, CA. Session A96.