Tonix Pharmaceuticals Reports Positive Phase 1 Results for TNX-1500 in Kidney Transplant and Autoimmune Treatments

Tonix Pharmaceuticals Holding Corp., a fully-integrated biopharmaceutical company, has announced promising results from its Phase 1 trial of TNX-1500, a Fc-modified humanized anti-CD40L monoclonal antibody. This study aimed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of TNX-1500 in healthy volunteers, with an eye towards its potential application in preventing kidney transplant rejection and treating autoimmune diseases.

During the Phase 1 trial, TNX-1500 was administered intravenously in single ascending doses of 3 mg/kg, 10 mg/kg, and 30 mg/kg. The study involved 26 participants who were monitored over a period extending to 120 days. Results indicated that TNX-1500 generally exhibited a favorable safety profile, with the only common treatment-emergent adverse event being mild aphthous ulcers. Importantly, no serious adverse events or thromboembolic events, identified as events of special interest, were reported.

A key component of the trial involved an antigen challenge using Keyhole Limpet Hemocyanin (KLH) to assess the immune modulation capabilities of TNX-1500. Participants received KLH injections on Day 2 and Day 29 of the study. TNX-1500 effectively blocked antibody responses at the 10 mg/kg and 30 mg/kg doses, demonstrating both primary and secondary antibody response inhibition. At the 3 mg/kg dose, TNX-1500 reduced the secondary antibody response significantly compared to the placebo.

Pharmacokinetic analysis revealed that TNX-1500 has a mean half-life ranging from 19.6 days at the 3 mg/kg dose to approximately 37.8 days at the 10 mg/kg dose, supporting the feasibility of monthly dosing in future efficacy trials. These findings support the potential for TNX-1500 to move forward into a Phase 2 trial, focusing on its efficacy in kidney transplant recipients.

Dr. Seth Lederman, CEO of Tonix Pharmaceuticals, highlighted the significance of these findings, noting that TNX-1500 could represent a next-generation alternative to earlier anti-CD40L therapies, which were associated with an increased risk of thrombosis. Tonix designed TNX-1500 by modifying the Fc region of a previous anti-CD40L monoclonal antibody to improve safety without compromising efficacy.

Dr. Gregory Sullivan, Chief Medical Officer of Tonix Pharmaceuticals, expressed optimism about TNX-1500's potential in improving graft survival and reducing long-term toxicity compared to current immunosuppressive therapies. The data from this Phase 1 trial provide a strong foundation for advancing TNX-1500 into further clinical development.

Tonix Pharmaceuticals plans to discuss these encouraging results with the U.S. Food and Drug Administration (FDA) in an upcoming meeting to align on the next steps for a Phase 2 trial. TNX-1500 is in development not only for preventing allograft rejection but also for addressing graft-versus-host disease following hematopoietic stem cell transplantation and treating autoimmune disorders.

This promising data adds to the body of evidence supporting TNX-1500, as previous studies on non-human primates have shown its efficacy in preventing organ rejection and preserving graft function, either alone or in combination with other therapies.

While TNX-1500 is currently an investigational new biologic and not approved for any indication, these results signal a significant step forward in the quest to enhance transplant and autoimmune treatment options, offering hope for improved outcomes and quality of life for affected patients.