Tonix Pharmaceuticals Holding Corp. has secured worldwide licensing rights to TNX-4800, a long-acting human monoclonal antibody designed for seasonal prevention of Lyme disease, from UMass Chan Medical School. The single-dose prophylactic treatment represents a novel approach to preventing the most common vector-borne infection in the United States, which affects millions of Americans annually.
Novel Mechanism Targets Bacterial Maturation in Ticks
TNX-4800 is a fully human monoclonal antibody with an engineered extended half-life that targets the outer-surface protein A (OspA) on Lyme-causing Borrelia burgdorferi bacteria. By binding OspA, the antibody blocks the maturation of Borrelia burgdorferi in the mid-gut of infected deer ticks, inactivating the bacteria before it reaches the skin at the site of the tick bite.
"TNX-4800's novel mechanism of blocking the maturation of Borrelia in the midgut of infected ticks is consistent with Tonix's focus on innovation," said Seth Lederman, MD, Chief Executive Officer of Tonix Pharmaceuticals. The treatment sidesteps the multidose schedules required for OspA vaccines in development and addresses concerns about autoimmunity that led to the withdrawal of previous FDA-approved vaccines.
Single Annual Dose Provides Season-Long Protection
Administered once yearly in early spring before ticks become most active, TNX-4800 is designed to maintain protective antibody titers for the entire tick season. The treatment provides pre-exposure prophylaxis against Lyme disease without relying on the recipient's immune system to generate antibodies, offering immediate protection within two days of administration.
"Unlike vaccine programs in development, TNX-4800 is a single dose and provides immediate immunity to the bacteria that causes Lyme disease," said Mark Klempner, MD, professor of medicine at UMass Chan and leader of the research team that discovered and developed the preventive medicine.
Positive Phase 1 Results Support Advanced Development
The monoclonal antibody has completed a positive Phase 1 study that demonstrated safety, tolerability, and a linear pharmacokinetic-pharmacodynamic-efficacy relationship. Tonix is now planning an adaptive Phase 2/3 study with the goal of submitting a Biologics Licensing Application.
"More than thirty years of clinical experience confirm that monoclonal antibodies can be delivered safely and can be effective in preventing infections," Klempner noted. The approach follows successful precedents in respiratory syncytial virus (RSV) prevention, where two long-acting monoclonal antibody products have received FDA approval for prophylaxis.
Addressing Growing Public Health Challenge
Lyme disease remains the most common vector-borne infection in the United States, with incidence climbing each year. The disease occurs most commonly in the Northeast, mid-Atlantic, and upper-Midwest regions. Approximately 70 million people live in areas of the U.S. where Lyme disease is endemic and could be eligible for treatment.
Typical symptoms of Lyme disease include fever, headache, fatigue, and a characteristic skin rash called erythema migrans. If left untreated, infection can spread to joints, the heart, and the nervous system. Even with antibiotic treatment, up to 20% of cases may progress to Post-Treatment Lyme Disease Syndrome, a chronic, debilitating condition characterized by joint and muscle pain, fatigue, and other symptoms.
Strategic Partnership for Translation
The licensing agreement was facilitated by BRIDGE Innovation and Business Development at UMass Chan, which received funding in 2023 to advance the technology toward commercialization. "This collaboration reflects UMass Chan's enduring commitment to translational research that addresses unmet medical needs," said Terence R. Flotte, MD, Provost, Dean and Executive Deputy Vice Chancellor of UMass Chan Medical School.
Currently, the only way to prevent Lyme disease infection is to prevent tick bites through insect repellent use and prompt tick removal. There are currently no FDA-approved vaccines or prophylactics to protect against Lyme disease, making TNX-4800 a potentially significant advancement in prevention strategies for millions of people who live, work, and vacation in endemic regions.
