Tonix Pharmaceuticals has announced plans for a Phase 2 trial of its TNX-2900 intranasal magnesium-potentiated oxytocin in patients with Prader-Willi syndrome (PWS). According to Tonix, the FDA has already cleared the company’s IND for a Phase 2 trial of TNX-2900. TNX-2900 has received both Rare Disease Designation from the FDA for the treatment of PWS in children and adolescents and orphan drug designation for PWS.
The news comes shortly after Acadia Pharmaceuticals announced that it was stopping development of its ACP-101 carbetocin nasal spray for PWS after a Phase 3 trial failed to meet a single endpoint. The Tonix announcement notes that oxytocin has different effects than carbetocin and says that the company believes that the magnesium formulation will “further enhance oxytocin receptor binding and signaling, with the goal of providing more consistent and selective receptor activation while minimizing off-target vasopressin effects.”
The Phase 2 trial is expected to enroll pediatric PWS patients between ages 8 and 17.5 years who will receive one of 3 dose levels of TNX-2900 or placebo over 12 weeks. The primary endpoint will be the change from baseline in the severity of overeating, as measured by a validated hyperphagia questionnaire.
Tonix CEO Seth Lederman commented, “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. . . . 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.”
Read the Tonix Pharmaceuticals press release
