Tonix Pharmaceuticals Holding Corp. announced on September 30, 2025 that it will initiate a Phase 2, randomized, double‑blind, placebo‑controlled study of its magnesium‑potentiated intranasal oxytocin formulation, TNX‑2900, for the treatment of Prader‑Willi syndrome (PWS). The trial will enroll male and female participants aged 8 to 17.5 years and will evaluate safety, tolerability, and efficacy over a 12‑week treatment period with three dose levels versus placebo.
The primary endpoint is the change from baseline in the Hyperphagia Questionnaire for Clinical Trials (HQ‑CT), a validated measure of hyperphagia severity in PWS. Secondary objectives include assessments of behavior, caregiver burden, and quality‑of‑life metrics, as well as safety and tolerability outcomes. The study design follows a 1:1:1:1 randomization to three active doses or placebo, with the goal of identifying a dose that maximizes clinical benefit while maintaining an acceptable safety profile.
TNX‑2900 has already received FDA orphan drug and rare pediatric disease designations, qualifying Tonix for a transferable priority review voucher upon approval. The magnesium‑potentiated formulation is intended to enhance oxytocin receptor binding and reduce dose‑related inconsistencies, addressing a critical unmet need in a population with limited therapeutic options. By advancing to Phase 2, Tonix seeks to generate pivotal data that could support regulatory approval and open a new revenue stream in a high‑impact rare disease market.
The announcement underscores Tonix’s strategy of expanding its pipeline beyond its commercial migraine products and the fibromyalgia candidate TNX‑102 SL. It also highlights the company’s focus on innovative delivery platforms and rare disease indications, positioning it to capitalize on both therapeutic and financial incentives such as the priority review voucher. The Phase 2 initiation represents a significant step toward diversifying Tonix’s product portfolio and strengthening its long‑term growth prospects.
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