Small Molecule · VQW-765

VQW-765

Phase 3Vanda Pharmaceuticals Inc. (VNDA)

An orally administered, selective alpha-7 nicotinic acetylcholine receptor (alpha7-nAChR) partial agonist in development by Vanda Pharmaceuticals for the on-demand treatment of acute anxiety in social anxiety disorder. Formerly known as AQW-051, it was originated by Novartis and in-licensed by Vanda. It exhibits an inverted U-shaped dose-response attributed to receptor desensitization at higher exposures.

Also known as: VQW-765, AQW-051, AQW051, (3R)-3-{[6-(4-methylphenyl)pyridin-3-yl]oxy}-1-azabicyclo[2.2.2]octane, 669770-29-0

Data current as of

Key facts

Modality
Small molecule
Chemical class
quinuclidine, arylpyridine
Chemistry
Single enantiomer
Mechanism
alpha7 nAChR Partial agonist
Highest phase
Phase 3
Lead indication
Social anxiety disorder
Developer
Vanda Pharmaceuticals Inc. (VNDA)
Trials
2 tracked · 1 recruiting · 45 sites
Next catalyst
End of 2026 — Topline data (Social anxiety disorder)

Mechanism of action#

VQW-765 is a potent and selective partial agonist of the alpha-7 nicotinic acetylcholine receptor (alpha7-nAChR, gene CHRNA7). It is being developed for on-demand acute use, leveraging fast-onset cholinergic modulation of the social-stress response.

TargetActionAffinity
alpha7 nAChRprimaryCHRNA7Partial agonistEC50 7.5 uMⓘ

Formulations#

FormulationRouteRegimenPharmacokinetics
VQW-765 oral capsule
10 mg oral capsule, single on-demand dose (Phase II performance-anxiety trial VP-VQW-765-2201)
OralSingle doset½ 20.75 h · Tmax 6 h

Development timeline#

201620182020202220242026TodayPhase 331 Dec 2026 — upcoming — Phase 3 VQW-765 social anxiety disorder topline results expected by end of 202622 Sept 2025 — phase change — Phase 3 social anxiety disorder study (NCT07221578) initiated; first study start date on ClinicalTrials.gov.31 Dec 2014 — Licensing deal — Vanda in-licenses VQW-765 (AQW051) alpha-7 nicotinic agonist from NovartisPhase 22 Dec 2022 — readout (missed) — Phase 2 VQW-765 performance-anxiety study missed primary endpoint overall; significant in females2 Aug 2022 — phase change — Phase 2 performance-anxiety study (NCT04800237) completed; primary SUDS endpoint missed in overall ITT population (P=0.1443) but significant in the female subgroup (P=0.034).
Phase change Readout Event UpcomingHover a marker for details.
Phase 3Dec 2014 – Dec 2026
  1. UpcomingPhase 3 VQW-765 social anxiety disorder topline results expected by end of 2026↗
  2. Phase 3 social anxiety disorder study (NCT07221578) initiated; first study start date on ClinicalTrials.gov.
  3. Licensing dealVanda in-licenses VQW-765 (AQW051) alpha-7 nicotinic agonist from Novartis↗
Phase 2Aug 2022 – Dec 2022
  1. missedPhase 2 VQW-765 performance-anxiety study missed primary endpoint overall; significant in females↗
  2. Phase 2 performance-anxiety study (NCT04800237) completed; primary SUDS endpoint missed in overall ITT population (P=0.1443) but significant in the female subgroup (P=0.034).↗

VQW-765 for Social anxiety disorder#

Phase 3RecruitingSocial anxiety disorder indication →

Vanda's Phase 3 program for VQW-765 in social anxiety disorder evaluates a single, on-demand oral dose for acute anxiety provoked by a standardized psychosocial stress test. The pivotal study (NCT07221578) is recruiting ~500 adults with SAD; topline results are guided for end of 2026. The approach follows a Phase 2 performance-anxiety study (NCT04800237) that missed its primary endpoint overall but showed a significant effect in female participants.

Readouts

  • End of 2026AnticipatedTopline dataNCT07221578

    Phase 3 VQW-765 social anxiety disorder topline results expected by end of 2026 ↗

  • 2022-12-02ReportedTopline datamissedNCT04800237

    Phase 2 VQW-765 performance-anxiety study missed primary endpoint overall; significant in females ↗

Clinical trials#

NCT07221578Phase 3Recruitingn=500

A Phase III, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of VQW-765 for the On-Demand Treatment of Acute Anxiety in Patients With Social Anxiety Disorder

Started Sept 2025· Primary completion Dec 2026· 30 sites across 1 country

United States

NCT04800237VP-VQW-765-2201Phase 2Completedn=230

A Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate Efficacy and Safety of VQW-765 in Patients With Performance Anxiety

Started Feb 2021· Primary completion Aug 2022· 15 sites across 1 country

United States

missedprimarySubjective Units of Distress Scale (SUDS) during the Trier Social Stress Test performance phase (0.1443)

A single 10 mg oral dose of VQW-765 produced numerically lower distress (SUDS) than placebo during the TSST performance phase but did not reach statistical significance in the overall intention-to-treat population (P=0.1443).

metsecondarySUDS during TSST performance phase, female subgroup (0.034)

Female participants (~69% of the sample) receiving VQW-765 showed a larger-magnitude and statistically significant reduction in performance-phase anxiety versus placebo (P=0.034).

Sources#

  1. Efficacy and safety of an alpha7-nAChR agonist, VQW-765, in subjects with performance anxiety (RCT) - oral 10 mg single dose — The British Journal of Psychiatry (PMC)
  2. Evaluating the Effects of VQW-765 vs. Placebo in Performance Anxiety — ClinicalTrials.gov
  3. Feuerbach et al. 2015 - AQW051, a novel, potent and selective alpha7 nAChR partial agonist: pharmacological characterization and phase I evaluation — British Journal of Pharmacology (PMC)
  4. NCT07221578 - VQW-765 for on-demand treatment of social anxiety disorder — ClinicalTrials.gov
  5. Vanda Pharmaceuticals Inc. Form 10-K (FY2020) — U.S. Securities and Exchange Commission (EDGAR)
  6. Vanda Pharmaceuticals Reports Fourth Quarter and Full Year 2025 Financial Results — Vanda Pharmaceuticals (Nasdaq)
  7. Vanda Pharmaceuticals Reports Results in a Phase II Clinical Study of VQW-765 in the Treatment of Acute Performance Anxiety — Vanda Pharmaceuticals (PR Newswire)