A phase 3 trial of Vanda Pharmaceuticals’ social anxiety disorder drug candidate has missed its primary endpoint, prompting the company to put the program on hold.
Investigators randomized (PDF) 546 adults with social anxiety disorder to receive a single oral dose of VQW-765—a small molecule alpha 7 nicotinic acetylcholine receptor partial agonist—or placebo. Patients were then challenged with a psychosocial stress test, which required them to prepare and deliver an interview-style presentation to a panel that gave no feedback or encouragement.
Scores on a distress scale were no better in women who received the drug candidate than in their counterparts on placebo, causing the trial to miss its primary endpoint. The study also missed a secondary endpoint that included men and women in the analysis.
Vanda limited its primary analysis to women based on midphase data that suggested they responded better to the drug. In the phase 2 trial, the biotech found a significant response in women but not in the overall population.
The failure to replicate the midphase efficacy signal led Vanda to put the program on hold pending further analysis of the full dataset.
Vanda’s setback is another blow to efforts to develop treatments for social anxiety disorder. Vistagen’s own candidate failed a phase 3 trial in December and flopped again in June. Yet with a post-hoc subpopulation analysis finding a nominally significant improvement, the biotech pushed ahead with plans to talk to the FDA.
Vanda's setback adds another twist to VQW-765’s long, winding path through development. Novartis originally developed the asset in schizophrenia but transferred rights to Vanda in 2014. By then, the drug candidate was in phase 2 development, before Vanda started a phase 2 anxiety trial in 2021.