RG7314 · program

Balovaptan for Post-traumatic stress disorder

DiscontinuedDiscontinuedF. Hoffmann-La Roche Ltd (ROG)

Indications for RG7314: Autism spectrum disorder · Discontinued Post-traumatic stress disorder · Discontinued

EXUVIA (NCT05401565), a Phase 2 randomised, double-blind, placebo-controlled trial at eight US sites (Aug 2022 - Oct 2023), randomised 29 adults to balovaptan 10 mg daily or placebo for 12 weeks. No meaningful difference on the primary endpoint (CAPS-5 total change at week 12: -17.2 balovaptan vs -15.6 placebo) or any secondary endpoint; well tolerated.

Data current as of

Development timeline

Phase 2Aug 2022 – Nov 2024
  1. missedEXUVIA Phase 2 in adult PTSD: balovaptan 10 mg did not separate from placebo on CAPS-5 total severity at week 12 (-17.2 vs -15.6) or on any secondary endpoint.↗
  2. EXUVIA completed (actual primary completion and study completion 2023-10-05); 29 randomised.
  3. Trial (portion) completedPhase 2 EXUVIA trial of balovaptan in PTSD completed
  4. EXUVIA Phase 2 in adult PTSD started (registry actual start 2022-08-02).

Readouts

  • 2024-11-11ReportedFull resultsmissedNCT05401565

    EXUVIA Phase 2 in adult PTSD: balovaptan 10 mg did not separate from placebo on CAPS-5 total severity at week 12 (-17.2 vs -15.6) or on any secondary endpoint. ↗

Clinical trials in Post-traumatic stress disorder

NCT05401565BN43546Phase 2Completedn=29

Study To Evaluate The Efficacy And Safety Of Balovaptan In Adults With Post-Traumatic Stress Disorder (PTSD) (EXUVIA)

Started Aug 2022· Primary completion Oct 2023· 📍 15 sites across 1 country (United States)

Formulations

FormulationRouteRegimenPharmacokinetics
Balovaptan oral 10 mg once daily
10 mg PO once daily in V1aduct (adult ASD Phase 3) and EXUVIA (PTSD Phase 2, 12 weeks); paediatric aV1ation used age-adjusted 4 mg and 10 mg adult-equivalent doses; VANILLA tested 1.5, 4 and 10 mg daily for 12 weeks.
OralOnce daily—

Mechanism of action (compound-wide)

Selective, brain-penetrant antagonist of the arginine vasopressin V1a receptor (AVPR1A), a Gq-coupled class-A GPCR implicated in social behaviour. Human V1a Ki 0.7 nM ([3H]AVP displacement) and IC50 3 nM in a functional assay, with ~1,700-fold lower affinity at the V2 receptor (Ki 1,200 nM) and Kb-based selectivity ratios of ~2,700-3,900x over oxytocin and V2 receptors. The clinical hypothesis was that V1a blockade improves socialisation and communication in autism; it was later tested for PTSD symptom severity.

TargetActionAffinity
V1A receptorprimaryAVPR1AAntagonistKi 0.7 nMⓘ

← Full RG7314 compound page (identity, identifiers, all indications)

Sources

  1. Balovaptan, ChEMBL CHEMBL4297183 - bioactivities (human V1a Ki 0.7 nM; V2 Ki 1200 nM) — ChEMBL (EMBL-EBI)
  2. Derks M, et al. Bioavailability and pharmacokinetic profile of balovaptan in healthy volunteers. Expert Opin Investig Drugs. 2021;30(8):893-901 — Expert Opinion on Investigational Drugs / PubMed
  3. Marler S, et al. Efficacy and safety of balovaptan for posttraumatic stress disorder: A randomized, placebo-controlled trial. Exp Clin Psychopharmacol. 2025;33(1):43-52 — Experimental and Clinical Psychopharmacology (APA) / PubMed
  4. NCT05401565 (BN43546) - Study To Evaluate The Efficacy And Safety Of Balovaptan In Adults With PTSD (results posted 2024-04-18) — ClinicalTrials.gov (U.S. National Library of Medicine)