Tonix’s Intranasal Oxytocin Candidate TNX-2900 Moves Into Phase 2 With FDA Orphan And Rare Pediatric Designations

Tonix Pharmaceuticals Holding Corp. announced that it will advance its TNX-2900 program into a Phase 2 clinical trial for the treatment of Prader-Willi syndrome (PWS). TNX-2900 is a proprietary intranasal oxytocin formulation, potentiated with magnesium, designed to enhance receptor binding while reducing inconsistencies often associated with oxytocin dosing.

The TNX-2900 program has already been granted Orphan Drug and Rare Pediatric Disease designations by the U.S. Food and Drug Administration (FDA), which positions Tonix to potentially receive a transferable Priority Review Voucher upon regulatory approval. These designations reflect the significant unmet need for effective therapies for PWS, a rare genetic disorder characterized by insatiable hunger, behavioral challenges, and metabolic complications.

“We are pleased to advance TNX-2900 into a Phase 2 trial for PWS, a condition with unmet needs for new medicines with activity and tolerability.  Families caring for children with PWS face significant challenges and burdens. Among them is hyperphagia which drives persistent food-seeking behaviors that require constant supervision and often result in obesity and serious medical complications. With an average life expectancy of less than 30 years, treatment of PWS remains an urgent and unmet need. By addressing limitations of traditional oxytocin delivery, we believe TNX-2900 has the potential to become an FDA-approved therapy targeting the oxytocin receptor in PWS and provide meaningful benefit for patients and families living with this rare disorder,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. 

Tonix also confirmed that the FDA has cleared its Investigational New Drug (IND) application for TNX-2900, paving the way for clinical trials in patients with PWS. The planned Phase 2 trial represents a significant step in the company’s strategy to expand its pipeline of rare disease therapies, leveraging its expertise in intranasal drug delivery systems.