Washington, D.C., USA, October 14, 2026
Vanda Pharmaceuticals Inc. has announced results from its Phase III clinical trial of VQW-765, an investigational small-molecule drug being developed for the on-demand treatment of acute anxiety in adults with social anxiety disorder (SAD). The study did not meet its primary endpoint, and the results did not confirm findings from an earlier Phase II trial. However, Vanda reported that VQW-765 was safe and well tolerated, consistent with previous studies. The company has placed the VQW-765 development program on hold while it conducts further analysis of the complete clinical dataset. The announcement highlights the challenges of translating early-stage clinical findings into successful late-stage outcomes and underscores the importance of confirmatory trials in evaluating investigational treatments for psychiatric disorders.
Phase III Trial Evaluates VQW-765 for Social Anxiety
The Phase III study, identified as VP-VQW-765-3201 (Study 3201), was a multicenter, randomized, double-blind, placebo-controlled clinical trial conducted at approximately 30 clinical sites across the United States. The study enrolled 546 adults aged 18 to 65 years who had a diagnosis of social anxiety disorder and elevated baseline scores on standardized anxiety assessments. Participants were randomly assigned to receive a single oral dose of VQW-765 or placebo before completing the Trier Social Stress Test (TSST), a standardized procedure designed to induce social and performance-related stress. During the test, participants prepared and delivered an interview-style presentation to a panel that provided no feedback or encouragement. The trial’s primary endpoint measured distress during the presentation phase among female participants using the Subjective Units of Distress Scale (SUDS), a self-reported measure ranging from zero to 100, with higher scores indicating greater distress. The study also evaluated distress scores across the combined male and female population as a key secondary endpoint. The results showed that VQW-765 did not demonstrate a statistically significant improvement over placebo on either measure. These findings mean the trial did not establish the intended benefit of the investigational treatment under the conditions evaluated.
Clinical Results Do Not Confirm Earlier Findings
In the primary analysis of female participants, the mean adjusted distress score was 65.6 for VQW-765 compared with 66.0 for placebo. The difference was not statistically significant, with a reported p-value of 0.8899. In the combined male and female population, the corresponding scores were 64.7 for VQW-765 and 63.8 for placebo, with a p-value of 0.6605. These findings indicate that the Phase III trial failed to meet both its primary endpoint and its key secondary endpoint. The numerical differences observed between the treatment and placebo groups did not demonstrate a statistically significant reduction in distress during the performance phase of the stress test. Study 3201 was designed to confirm findings from Vanda’s earlier Phase II study, VP-VQW-765-2201. In that earlier study, participants with a history of performance anxiety who received VQW-765 reported numerically lower stress levels during the TSST than participants receiving placebo. The Phase III results did not reproduce those earlier findings, highlighting the importance of confirmation in larger, later-stage clinical studies. Vanda expressed disappointment with the outcome and acknowledged the contributions of trial participants, investigators and clinical site staff. The company emphasized that the safety and tolerability findings were consistent with its previous experience with the investigational compound.
Advertisement
VQW-765 Development Program Placed on Hold
VQW-765 is a small-molecule partial agonist of the alpha-7 nicotinic acetylcholine receptor (α7-nAChR), a receptor involved in neurological signaling. The compound is being investigated for its potential application in psychiatric disorders, including acute anxiety associated with social and performance situations. Vanda licensed the compound, previously known as AQW-051, from Novartis. Performance anxiety can affect activities such as public speaking, examinations and professional presentations. Developing an on-demand treatment that reduces acute anxiety without unwanted sedative effects remains an area of clinical interest. Nevertheless, the latest trial results do not establish VQW-765 as an effective treatment for social anxiety disorder. Following the Phase III outcome, Vanda has placed the VQW-765 clinical development program on hold pending further analysis of the full dataset. Additional evaluation may help the company understand the results and determine whether any further development steps are warranted. No new development timeline has been established in the announcement. The findings represent a setback for the investigational program but provide important information about the compound’s performance in a controlled clinical setting. Further conclusions will depend on the company’s analysis of the complete dataset and any subsequent development decisions.
Source: Vanda Pharmaceuticals press release
