8-K: Tonix Pharmaceuticals Presents Promising Preclinical Data for TNX-801 Mpox Vaccine at World Vaccine Congress

Tonix Pharmaceuticals presented data at the World Vaccine Congress highlighting the potential of its TNX-801 single-dose mpox vaccine, demonstrating protection and tolerability in preclinical studies.

Summary

• Tonix Pharmaceuticals presented preclinical data on TNX-801, a single-dose, live virus vaccine candidate for mpox and smallpox, at the World Vaccine Congress Washington 2025.
• The data showed that TNX-801 protected animals, including immunocompromised ones, from mpox and rabbitpox and was well-tolerated.
• TNX-801 demonstrated durable six-month protection against a lethal challenge with rabbitpox virus.
• The vaccine also protected immunocompromised animals against a lethal challenge with monkeypox clade IIa virus.
• These findings build on previous studies showing protection against a lethal challenge with intratracheal clade Ia mpox virus.
• TNX-801 prevented both clinical disease and lesion formation in all studies after a single-dose vaccination.
• The company believes TNX-801 has the potential to impact the prevention of mpox and control future epidemics, aligning with the WHO's target product profile for mpox vaccines.
• TNX-801 is based on synthesized horsepox and has shown better tolerability than 20th-century vaccinia live-virus vaccines in animals.
• Tonix has a collaboration with the Kenya Medical Research Institute (KEMRI) to plan a Phase I clinical study of TNX-801 in Kenya.
• The FDA has granted Fast Track designation to TNX-102 SL for the management of fibromyalgia, with a PDUFA goal date of August 15, 2025.

Sentiment

Score: 8

Explanation: The document presents positive preclinical data for TNX-801, suggesting a promising future for the vaccine candidate. The alignment with WHO's target product profile and the potential for single-dose administration further contribute to a positive outlook.

Positives

• TNX-801 shows promise as a potential mpox and smallpox vaccine by providing protective immunity to animals with a single dose.
• TNX-801 was generally well tolerated, even in immunocompromised animals.
• The vaccine offers durable six-month protection against a lethal challenge with rabbitpox virus.
• TNX-801 prevented both clinical disease and lesion formation in all studies after a single-dose vaccination.
• The WHO's preferred target product profile aligns with the characteristics of TNX-801.
• TNX-801 is based on synthesized horsepox, believed to be more closely related to Dr. Jenner's vaccine than 20th-century vaccinia viruses.
• Tonix has a collaboration with the Kenya Medical Research Institute (KEMRI) to plan a Phase I clinical study of TNX-801 in Kenya.

Risks

• The forward-looking statements are subject to risks related to obtaining FDA clearances or approvals, successfully marketing products, the timing and progress of clinical development, the need for additional financing, patent protection and litigation, government or third-party payor reimbursement, limited research and development efforts, dependence upon third parties, and substantial competition.
• As with any pharmaceutical under development, there are significant risks in the development, regulatory approval, and commercialization of new products.

Future Outlook

Tonix is focused on advancing TNX-102 SL for fibromyalgia, with a PDUFA goal date of August 15, 2025. The company is also developing TNX-1300 for cocaine intoxication and TNX-1500 for organ transplant rejection and autoimmune diseases. Tonix is also working on TNX-4200 for the prevention or treatment of infections to improve the medical readiness of military personnel in biological threat environments.

Management Comments

• Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals, stated that TNX-801 shows promise as a potential mpox and smallpox vaccine by providing protective immunity to animals with a single-dose.
• Dr. Lederman noted that the new data show durable six-month protection against a lethal challenge with rabbitpox virus and protection of immunocompromised animals against a lethal challenge with monkeypox clade IIa virus.
• Dr. Lederman believes TNX-801 has the potential to make an impact towards preventing mpox and controlling future mpox epidemics.

Industry Context

The ongoing mpox epidemics highlight the need for additional vaccine options, particularly single-dose options. The WHO's preferred target product profile for mpox vaccines aligns with the characteristics of TNX-801, indicating a potential market for this type of vaccine.

Comparison to Industry Standards

• The FDA-approved non-replicating mpox vaccine Jynneos requires two doses and provides a relatively short duration of protection, while TNX-801 is being developed as a single-dose vaccine.
• FDA also recently approved ACAM2000, a live, replicating vaccinia vaccine for prevention of mpox, while TNX-801 is a minimally replicative, live-virus vaccine based on synthesized horsepox.
• Pre-clinical results from an mRNA vaccine recently showed some protection from a Clade I monkeypox challenge, but with multiple break-through lesions in vaccinated animals, while TNX-801 provides 100% protection against lesions in animal models.

Stakeholder Impact

• Positive preclinical results for TNX-801 could benefit shareholders through potential stock price appreciation.
• Successful development and commercialization of TNX-801 could improve public health by providing a new vaccine option for mpox and smallpox.
• The collaboration with KEMRI could strengthen Tonix's relationships with international research institutions.

Next Steps

• Tonix plans to design, plan, and seek regulatory approval for a Phase I clinical study of TNX-801 in Kenya in collaboration with KEMRI.
• Tonix will continue to advance TNX-102 SL for fibromyalgia, with a PDUFA goal date of August 15, 2025.

Key Dates

Keywords

TNX-801, mpox vaccine, smallpox vaccine, vaccine, Tonix Pharmaceuticals, preclinical data, immunocompromised, horsepox, KEMRI, single-dose