Efficacy and safety of balovaptan for posttraumatic stress disorder: A randomized, placebo-controlled trial.

Abstract

Posttraumatic stress disorder (PTSD) has a significant impact on quality of life and affects more than 13 million individuals in the United States, with limited treatments available. EXUVIA (NCT05401565) was a Phase 2, randomized, placebo-controlled, double-blind trial, conducted across eight sites in the United States. The study aimed to assess the efficacy, safety, and pharmacokinetics of balovaptan, a highly selective vasopressin 1a receptor antagonist, in adults with PTSD. Between August 2022 and October 2023, a total of 57 adult participants (aged 18-60 years) were screened, and 29 participants were randomly allocated (1:1) to receive either balovaptan (13/29 [44.8%]) or placebo (16/29 [55.2%]). No meaningful differences were observed for balovaptan (-17.2 [± 10.7]) versus placebo (-15.6 [± 10.7]) as measured by the primary endpoint of change from baseline at Week 12 in Clinician-Administered PTSD Scale for Diagnostic and Statistical Manual of Mental Disorders, Fifth Edition total symptom severity score. No meaningful differences for balovaptan versus placebo were observed at Week 12 for any secondary endpoints. Balovaptan was well tolerated with no new safety findings. The number of participants with at least one adverse event of any intensity was 9/13 (69.2%) in the balovaptan group and 7/16 (43.8%) in the placebo group. (PsycInfo Database Record (c) 2025 APA, all rights reserved).