Tonix Pharmaceuticals Plans to Initiate Prader-Willi Syndrome Phase 2 Trial of TNX-2900 (Intranasal Potentiated Oxytocin) in 2026

Core Insights - Tonix Pharmaceuticals is advancing its TNX-2900 program into a Phase 2 clinical trial for the treatment of Prader-Willi Syndrome (PWS), a rare genetic disorder with significant unmet medical needs [1][2] - TNX-2900 is a magnesium-potentiated intranasal oxytocin formulation aimed at improving receptor binding and reducing dose-related inconsistencies [1][4] - The FDA has granted TNX-2900 Orphan Drug and Rare Pediatric Disease designations, which may provide a Priority Review Voucher upon approval [1][2] Group 1: Clinical Trial Details - The Phase 2 trial will be randomized, double-blind, placebo-controlled, and will include participants aged 8 to 17.5 years [2] - Participants will receive 12 weeks of treatment with TNX-2900 at one of three dose levels or placebo in a 1:1:1:1 ratio [2] - The primary efficacy endpoint is the change from baseline in the Hyperphagia Questionnaire for Clinical Trials (HQ-CT) [2] Group 2: Prader-Willi Syndrome Overview - PWS affects approximately 1 in 10,000 to 1 in 30,000 births and is characterized by severe hyperphagia, behavioral challenges, and obesity [3][5] - The average life expectancy for individuals with PWS is less than 30 years, highlighting the urgent need for effective treatments [2][6] - Current interventions for PWS are often inadequate and difficult to sustain [3] Group 3: Mechanism of Action - Research indicates that PWS is associated with a functional deficiency of oxytocin, which regulates feeding behaviors [4][6] - TNX-2900 aims to enhance oxytocin receptor binding and signaling while minimizing off-target effects related to vasopressin [4][7] - Previous studies have shown that intranasal oxytocin therapy can improve behaviors associated with PWS [6] Group 4: Company Background - Tonix Pharmaceuticals is a fully-integrated biotechnology company with a focus on central nervous system disorders, immunology, and infectious diseases [8] - The company has recently received FDA approval for Tonmya, a non-opioid analgesic for fibromyalgia, marking a significant milestone in its product development [8] - Tonix is also developing other candidates targeting various conditions, including TNX-801 for mpox and smallpox [8]