Tonix Pharmaceuticals Holding Corp.

TNX-801 is a single-dose, live virus vaccine in development to protect against mpox and smallpox TNX-801 protects immunocompromised animals from a lethal challenge with clade IIa monkeypox virus Durability of TNX-801 vaccination shown by six-month protection of animals from a lethal challenge with rabbitpox Tolerability of TNX-801 demonstrated in immunocompromised animals by no spreading to blood or tissues, even at high doses April 24, 2025 -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a fully-integrated biopharmaceutical company with marketed products and a pipeline of development candidates, presented data in an oral presentation at the World Vaccine Congress Washington 2025, held April 21-24, 2025, in Washington, D.C. The presentation titled, “A Novel Single-Dose, Attenuated Live, Minimally Replicative Mpox Vaccine”, highlighted positive preclinical efficacy data, demonstrating that TNX-801 protected animals from mpox and rabbitpox and was well tolerated, even in immunocompromised animals. A copy of the Company’s presentation is available under the Scientific Presentations tab of the Tonix website at www.tonixpharma.com. “TNX-801 shows promise as a potential mpox and smallpox vaccine by providing protective immunity to animals with a single-dose,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “TNX-801 was generally well tolerated, even in immunocompromised animals. 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. These new data build upon prior studies showing protection of animals against a lethal challenge with intratracheal clade Ia mpox virus. In all of these studies, after a single dose vaccination, TNX-801 prevented both clinical disease and formation of lesions.” Dr. Lederman continued, “The ongoing clade IIb mpox epidemic that started in 2022, and the more recent and ongoing clade Ib mpox epidemic, highlight the need for additional vaccine options, particularly single-dose options. Both the 2022 clade IIb and the 2024 clade Ib mpox epidemics have been declared by the World Health Organization (WHO) to be Public Health Emergencies of International Concern (PHEICs). We believe TNX-801 has the potential to make an impact towards preventing mpox and controlling future mpox epidemics.” TNX-801 is a minimally replicative, live-virus vaccine based on synthesized horsepox that has been shown to provide single-dose immune protection against a monkeypox challenge with better tolerability than 20th century vaccinia live-virus vaccines in animals. In September 2024, Tonix announced that the WHO’s preferred target product profile (TPP), released at the WHO sponsored Mpox Research and Innovation Scientific Conference, aligns with the characteristics of TNX-801. Key elements of the WHO draft TPP include single-dose, durable protection, administration without special equipment, and stability at ambient temperature. Other potential beneficial characteristics include the ability to limit forward transmission, use in case-contact vaccination strategies and suitability for use in immunocompromised individuals. TNX-801 (recombinant horsepox virus) is a single-dose, attenuated, minimally replicative, live virus vaccine based on horsepox in pre-clinical development to prevent mpox and smallpox. Tonix reported positive preclinical efficacy data, demonstrating that TNX-801 vaccination protected non-human primates against lethal challenge with monkeypox. After a single dose vaccination, TNX-801 prevented clinical disease and lesions and decreased shedding in the mouth and lungs of non-human primates. The findings are consistent with mucosal immunity and suggest the ability to block forward transmission, similar to Dr. Edward Jenner’s vaccine, which eradicated smallpox and kept mpox out of the human population. TNX-801 is based on synthesized horsepox which is believed to be more closely related to Dr. Jenner’s vaccine than 20th century vaccinia viruses.6 Smallpox vaccines descended from Jenner’s vaccine used prior to 1900 would be called horsepox by modern nomenclature. TNX-801 is delivered percutaneously with only one dose and therefore may achieve higher rates of community protection than two-dose vaccines by eliminating drop-out between doses and limiting forward transmission. Tonix has received official written response from a Type B pre-Investigational New Drug Application (IND) meeting with the U.S. Food and Drug Administration (FDA) to develop TNX-801 as a potential vaccine to protect against mpox disease and smallpox. Tonix has announced a collaboration with the Kenya Medical Research Institute (KEMRI) to design, plan and seek regulatory approval for a Phase I clinical study of TNX-801 in Kenya. The Company believes TNX-801 has the potential to make a global impact on mpox and the risk of smallpox because of its durable T-cell immune response, the potential to manufacture at scale, and the use of a lower dose than non-replicating vaccines. The FDA-approved non-replicating mpox vaccine Jynneos® requires two doses and provides a relatively short duration of protection. FDA also recently approved ACAM2000, a live, replicating vaccinia vaccine for prevention of mpox. ACAM200 is a clone from DryVax®, a 20th century vaccinia vaccine derived from the NYCBH strain. 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. Tonix is a fully integrated biopharmaceutical company focused on transforming therapies for pain management and vaccines for public health challenges. Tonix’s development portfolio is focused on central nervous system (CNS) disorders. Tonix’s priority is to advance TNX-102 SL, a product candidate for the management of fibromyalgia, for which an NDA was submitted based on two statistically significant Phase 3 studies for the management of fibromyalgia and for which a PDUFA (Prescription Drug User Fee act) goal date of August 15, 2025 has been assigned for a decision on marketing authorization. The FDA has also granted Fast Track designation to TNX-102 SL for the management of fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction and acute stress disorder under a Physician-Initiated IND at the University of North Carolina in the OASIS study funded by the U.S. Department of Defense (DoD). Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase), a biologic in Phase 2 development designed to treat cocaine intoxication that has FDA Breakthrough Therapy designation, and its development is supported by a grant from the National Institute on Drug Abuse. Tonix’s immunology development portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is an Fc-modified humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft rejection and for the treatment of autoimmune diseases. Tonix’s infectious disease portfolio includes TNX-801, a vaccine in development for mpox and smallpox, as well as TNX-4200 for which Tonix has a contract with the U.S. Department of Defense’s (DoD’s) Defense Threat Reduction Agency (DTRA) for up to $34 million over five years. TNX-4200 is a small molecule broad-spectrum antiviral agent targeting CD45 for the prevention or treatment of infections to improve the medical readiness of military personnel in biological threat environments. Tonix owns and operates a state-of-the art infectious disease research facility in Frederick, Md. Tonix Medicines, our commercial subsidiary, markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan nasal spray) 10 mg for the treatment of acute migraine with or without aura in adults. * Tonix’s product development candidates are investigational new drugs or biologics. Their efficacy and safety have not been established and have not been approved for any indication. Zembrace SymTouch and Tosymra are registered trademarks of Tonix Medicines. All other marks are property of their respective owners. The content above comes from the network. if any infringement, please contact us to modify.

Plus, news about the Novo Nordisk Foundation and Tonix: AbbVie’s Rinvoq gets label expansion: The blockbuster JAK inhibitor won approval in the US to treat adults with giant cell arteritis, an autoimmune disease that causes inflammation of the arteries, often in the scalp and head. Rinvoq was approved in Europe for the same condition earlier this month. — Nicole DeFeudis #AUA2025: Relmada Therapeutics’ interim Phase 2 data in bladder cancer: The company said NDV-01, a reformulation of two chemo drugs, achieved an 85% overall response rate in 20 patients with non-muscle invasive bladder cancer. Two patients with carcinoma in situ achieved a complete response. Already below $1.00, Relmada’s stock price $RLMD was down 18% on Tuesday morning. — Max Gelman Novo Nordisk Foundation expands certain grants beyond Denmark: The grants, which are awarded through the foundation’s annual Challenge Programme, will now be available to researchers in most of Europe. The new area includes Europe’s Schengen area — this accounts for most EU member countries plus a handful of non-member countries — Ireland and the UK. The overall budget for grants this year is DKK 600 million (€80 million). — Max Gelman Tonix discontinues cocaine intoxication trial: The biotech discontinued the Phase 2 study for TNX-1300 due to slower-than-projected enrollment, it said in an SEC filing last week. — Max Gelman Hello, Mark Smith You’re an essential part of the biopharma world. Share your voice on this important question to help shape our understanding of the industry. Can you help us out? Hello, Mark Smith You’re an essential part of the biopharma world. Share your voice on this important question to help shape our understanding of the industry. Can you help us out?

Tonix Pharmaceuticals\' study was set up in an emergency department setting but was hampered by slow enrollment. \n Tonix Pharmaceuticals has no tonic for cocaine intoxication, with the biopharma terminating a midstage trial for the condition amid poor trial enrollment.The investigational drug in question, TNX-1300, had snagged an FDA breakthrough therapy designation to treat cocaine intoxication and overdose.In a Securities and Exchange Commission filing made public Friday, however, Tonix said it has discontinued enrollment and terminated the study, dubbed Catalyst. The trial was set up in an emergency department, but enrollment in this setting “was slower than projected,” Tonix said in the filing, noting that the related trial termination was not due to safety or efficacy concerns. The investigational therapy works as a recombinant protein enzyme via a non-disease-producing strain of E. coli bacteria.Tonix said that there is no specific pharmacotherapy indicated for cocaine intoxication, which can prompt life-threatening risks including heart attacks, respiratory failure and seizures.It’s not curtains yet for TNX-1300, as Tonix added that it will be “evaluating new study designs and new endpoints for further development” of the drug, though the company did not give specifics. In 2022, the biopharma had received financing for the study from the U.S. government via a pact with the National Institute on Drug Abuse, part of the National Institutes of Health. Tonix is no stranger to drug and trial setbacks. At the end of 2023, the company dropped a major depression disorder therapy after the drug flamed out of a phase 2 trial.A few months prior, Tonix abandoned testing out a cyclobenzaprine drug for fibromyalgia-like long COVID after the asset failed to show a statistically significant improvement in pain symptoms.There however may be reprieve for that therapy, called TNX-102 SL, on the horizon. Tonix is gearing up for what it hopes will be an FDA approval for the med in fibromyalgia, with an FDA decision date expected before or by Aug. 15.