Vanda Pharmaceuticals has put its social anxiety disorder program on hold after VQW-765 failed a phase 3 trial. The setback came after investigators found that women who received the small molecule alpha 7 nicotinic acetylcholine receptor partial agonist did no better than placebo on the study's primary distress-scale endpoint.
The miss also extended to a secondary endpoint that analyzed both men and women. Vanda said it will pause the program pending further analysis of the full dataset, a decision that reflects how dependent the late-stage plan had become on a narrower signal from earlier testing.
The Data
Investigators randomized 546 adults with social anxiety disorder to receive a single oral dose of VQW-765 or placebo. Patients were then put through a psychosocial stress test that required them to prepare and deliver an interview-style presentation to a panel that offered no feedback or encouragement.
Vanda limited the primary analysis to women because midphase data had suggested they responded better to the drug. In the phase 2 trial, the company reported a significant response in women but not in the overall population. The phase 3 failure to reproduce that effect in the subgroup that had informed the registration strategy is the central readout from this event.
For development programs built around subgroup findings, replication matters more than the initial hint. Here, the late-stage result did not confirm the earlier distinction between women and the broader study population, leaving Vanda without a clear efficacy signal to advance.
The Road Here
VQW-765 has had a long path through development. Novartis originally developed the asset in schizophrenia and transferred rights to Vanda in 2014. By that point, the candidate was already in phase 2 development.
Vanda later redirected the program and started a phase 2 anxiety trial in 2021. The latest miss adds another setback to a field that has struggled to deliver late-stage wins. The source notes that Vistagen's candidate failed a phase 3 trial in December and again in June, although that company has continued toward discussions with the FDA after a post-hoc subpopulation analysis found a nominally significant improvement.
For Vanda, the immediate implication is narrower: the company is no longer treating the earlier female-subgroup signal as sufficient support for continued execution. That is a more conservative read than trying to push forward on an unconfirmed effect, and it leaves the future of VQW-765 dependent on whether the full dataset reveals a more persuasive explanation for the divergence between phase 2 and phase 3.
