Tonix Pharmaceuticals Advances TNX-2900 for Prader-Willi Syndrome Into Phase 2 Clinical Trial

The U.S. Food and Drug Administration has cleared Tonix Pharmaceuticals Holding Corp.'s Investigational New Drug application for TNX-2900, allowing the company to advance its proprietary magnesium-potentiated intranasal oxytocin formulation into Phase 2 clinical trials for Prader-Willi syndrome. This regulatory milestone represents a significant step forward in addressing PWS, a rare genetic disorder recognized as a leading cause of life-threatening childhood obesity. The program's progression carries substantial implications for patients and families affected by this challenging condition, potentially offering new therapeutic options where limited treatments currently exist.

TNX-2900 has received both Orphan Drug and Rare Pediatric Disease designations from the FDA, regulatory classifications that acknowledge the medication's potential to address unmet medical needs in rare conditions. These designations provide Tonix Pharmaceuticals with important incentives, including eligibility for a transferable Priority Review Voucher upon approval of TNX-2900. Such vouchers can accelerate the review process for future drug applications and represent valuable assets in the pharmaceutical industry, potentially generating significant financial value that could support further research and development efforts.

The advancement of TNX-2900 comes at a time when Tonix Pharmaceuticals has achieved other significant regulatory successes, including recent FDA approval for Tonmya, a first-in-class, non-opioid analgesic medicine for fibromyalgia treatment. This approval marked the first new prescription medicine for fibromyalgia in more than 15 years, demonstrating the company's capability to navigate complex regulatory pathways and bring innovative treatments to market. Additional information about Tonix's development programs and corporate activities can be found at https://www.tonixpharma.com.

Beyond TNX-2900, Tonix maintains a diverse development portfolio focused on central nervous system disorders, immunology, immuno-oncology and infectious diseases. The company's immunology pipeline includes TNX-1500, an Fc-modified humanized monoclonal antibody targeting CD40-ligand being developed for preventing allograft rejection and treating autoimmune diseases. In infectious diseases, Tonix is developing TNX-801 as a vaccine candidate for mpox and smallpox, while TNX-4200 represents a small molecule broad-spectrum antiviral agent under development through a contract with the U.S. Department of Defense's Defense Threat Reduction Agency valued at up to $34 million over five years.

The progression of TNX-2900 into Phase 2 trials holds particular significance for the rare disease community, where treatment options remain limited despite advances in genetic understanding. Prader-Willi syndrome presents complex challenges including insatiable appetite, metabolic abnormalities, and behavioral issues that significantly impact quality of life and require comprehensive management strategies. The development of targeted therapies like TNX-2900 represents hope for improved symptom management and potentially better long-term outcomes for individuals living with this condition.