Tonix Pharmaceuticals to initiate PWS Phase 2 trial of TNX-2900 in 2026

Tonix Pharmaceuticals (TNXP) announced plans to progress its TNX-2900 program for the treatment of Prader-Willi syndrome into a Phase 2 clinical trial. TNX-2900 is a proprietary magnesium-potentiated intranasal oxytocin formulation designed to improve receptor binding and decrease dose-related inconsistencies in receptor activity. The program has received both Orphan Drug and Rare Pediatric Disease designations from the U.S. Food and Drug Administration, which would make Tonix eligible for a transferable Priority Review Voucher, upon approval. The FDA has cleared the Investigational New Drug application for TNX-2900 to progress into Phase 2 development. Tonix plans to conduct a Phase 2 randomized, double-blind, placebo-controlled, parallel-design study to evaluate the safety, tolerability, and efficacy of TNX-2900 in male and female participants with PWS, ages 8 to 17.5 years. Eligible participants will be randomized to receive 12-weeks of treatment with TNX-2900 at one of three dose levels, or placebo, in a 1:1:1:1 ratio. The primary efficacy endpoint will be the change from baseline in the validated Hyperphagia Questionnaire for Clinical Trials, a widely used measure of hyperphagia severity in PWS. Secondary objectives will include assessments of behavior, caregiver burden, and quality of life measures, as well as safety and tolerability outcomes.