TNX-2900

TNX-2900 (intranasal potentiated oxytocin) is being developed for Prader-Willi syndrome (“PWS”), a rare genetic disorder and the leading cause of life-threatening childhood obesity, affecting about 1 in 10,000 to 1 in 30,000 births. Infants often present with poor muscle tone and feeding difficulties, while children and adolescents develop hyperphagia, behavioral challenges, and severe obesity and metabolic disease. Current interventions are difficult to sustain and often inadequate.

The formulation technology for TNX-2900 was acquired from Trigemina, Inc. and licensed from Stanford University. The potentiated formulation includes magnesium, which has been shown in animal studies to potentiate binding of oxytocin to the oxytocin receptor. The therapeutic technology was licensed from Inserm, the French National Institute of Health and Medical Research.

TNX-2900 was granted Orphan-Drug Designation by the FDA in the second half of 2023, the IND was cleared by the FDA in the fourth quarter of 2023, and Rare Pediatric Disease Designation was received in March 2024, makeing Tonix eligible for a transferable Priority Review Voucher upon approval.

Tonix plans to progress the TNX-2900 program for the treatment of PWS into 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 (HQ-CT), 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. Tonix plans to initiate enrollment in this study in the first quarter of 2027.

TNX-2900 is an investigational new drug and has not been approved for any indication.