Tonix Pharmaceuticals (TNXP) Investor presentation summary

Disease overview and unmet need

• Prader-Willi syndrome (PWS) is the most common genetic cause of life-threatening childhood obesity, affecting 1 in 10,000 to 1 in 30,000 births.

• Symptoms include poor muscle strength, feeding difficulties in infancy, hyperphagia, intellectual disabilities, and behavioral problems.

• There is no cure and limited FDA-approved treatments for PWS, creating a high unmet need for effective therapies.

• Hyperphagia in PWS leads to severe obesity, caretaker burden, and significant health risks such as type 2 diabetes and cardiovascular disease.

Oxytocin biology and rationale

• Oxytocin plays a key role in satiety, appetite suppression, and behavioral regulation.

• PWS patients show abnormalities in the oxytocin system, including increased plasma oxytocin but decreased receptor expression and abnormal neurons.

• Oxytocin requires magnesium ions for optimal receptor binding and activation, which is critical for its satiety signaling.

Therapeutic approach and formulation

• TNX-2900 is a patented intranasal formulation combining oxytocin with magnesium to enhance receptor activity.

• Intranasal oxytocin has been studied for various neuropsychiatric and metabolic disorders and is generally well tolerated.

• Previous studies in PWS showed mixed efficacy, with improvements mainly in younger patients and challenges at higher doses.

• Addition of magnesium expands the effective dose range and rescues oxytocin efficacy at higher doses in animal models.