Tonix Pharmaceuticals Holdings CEO Dr. Seth Lederman shared with Steve Darling from Proactive to announce a major milestone for the company’s rare disease pipeline: the advancement of 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 reduce inconsistencies in receptor activity often observed with standard oxytocin dosing. The program has received both Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA). Importantly, these designations could make Tonix eligible for a transferable Priority Review Voucher (PRV) if the therapy is successfully approved, a potential asset of significant strategic and financial value. Dr. Lederman explained that the FDA has already cleared Tonix’s Investigational New Drug (IND) application, enabling the initiation of Phase 2 development. The planned trial will be a randomized, double-blind, placebo-controlled, parallel-design study, evaluating the safety, tolerability, and efficacy of TNX-2900 in boys and girls with PWS aged 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 Hyperphagia Questionnaire for Clinical Trials (HQ-CT), a validated tool widely used to measure hyperphagia severity in PWS. Secondary endpoints will include assessments of behavior, caregiver burden, quality of life, and additional safety outcomes. Prader-Willi syndrome is a rare genetic disorder and the leading cause of life-threatening childhood obesity, affecting an estimated 1 in 10,000 to 1 in 30,000 births. Infants often present with poor muscle tone and feeding difficulties, while older children and adolescents develop hyperphagia (insatiable hunger), behavioral challenges, and severe obesity with associated metabolic complications. Current interventions remain limited, difficult to sustain, and often inadequate to manage the condition effectively. With FDA clearance, multiple regulatory designations, and a well-defined clinical pathway, Tonix is now positioned to advance TNX-2900 toward becoming a potential first-in-class treatment for PWS. #proactiveinvestors #tonixpharmaceuticalsholdingcorp #nasdaq #tnxp #Biotech #MPOXVaccine #Smallpox #TNX801 #VaccineDevelopment #ClinicalTrials #PharmaceuticalNews #MedicalResearch #WHO #GlobalHealth #InfectiousDiseases #Biopharma #ProactiveInvestors