Tonix Pharmaceuticals Reports Fourth Quarter and Full Year 2024 Financial Results and Operational Highlights
August 15, 2025, is the FDA PDUFA goal date for TNX-102 SL for the management of fibromyalgia; If approved, TNX-102 SL would become the first new drug for treating fibromyalgia in more than 15 years
Company expects to have sufficient cash to fund planned operations beyond the FDA PDUFA goal date and anticipated fourth quarter 2025 launch of TNX-102 SL for fibromyalgia; $98.8 million in cash as of December 31, 2024
Announced positive topline results from Phase 1 study of TNX-1500, a next generation anti-CD40L mAb candidate for prevention of kidney transplant rejection and treatment of autoimmune diseases
Received government grant for potential mpox vaccine, TNX-801, which has demonstrated single-dose immune protection against a monkeypox challenge in non-human primates
Received first payments from U.S. Department of Defense (DoD) contract for up to $34 million over five years to develop a broad-spectrum antiviral drug program
CHATHAM, N.J., March 18, 2025 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (NASDAQ:TNXP) (Tonix or the Company), a fully-integrated biopharmaceutical company with marketed products and a pipeline of development candidates, today announced financial results for the fourth quarter and full year ended December 31, 2024, and provided an overview of recent operational highlights.
"With commercial preparations underway, we believe we are well positioned to launch TNX-102 SL for the management of fibromyalgia in the fourth quarter of this year if approved by the U.S. Food and Drug Administration," said Seth Lederman, M.D., Chief Executive Officer of Tonix. "We believe that TNX-102 SL has the potential to be the first in a new class of non-opioid analgesic medicines for the management of fibromyalgia, and the first new drug for treating fibromyalgia in more than 15 years."
Fibromyalgia is a debilitating chronic pain condition affecting over 10 million adults in the United States, most of whom are women. Data from our pivotal Phase 3 trials indicate that TNX-102 SL can provide fibromyalgia patients with significant reduction in pain. TNX-102 SL was generally well tolerated and has no known addictive properties.
Dr. Lederman continued, "Tonix is debt free and expects to have sufficient cash to fund operations through the PDUFA target date of August 15, 2025, and the anticipated commercial launch of TNX-102 SL in the fourth quarter of this year. We continue to meaningfully add to our commercial team and are engaged in pre-launch activities. We look forward to continuing discussions with the FDA throughout the review period in advance of the PDUFA goal date to bring patients a potential new treatment option."
"Beyond TNX-102 SL for fibromyalgia, we are encouraged by the continued development of our pipeline in a capital efficient manner. Positive results from a Phase 1 study evaluating the tolerability and pharmacokinetics of TNX-1500, a next generation anti-CD40L mAb for prevention of kidney transplant rejection, support advancing to a planned Phase 2 trial in kidney transplant recipients with monthly dosing. In addition, we continue to advance TNX-801 vaccine for preventing mpox and smallpox towards the clinic. With the ongoing global mpox epidemic continuing to spread, TNX-801's ability to protect animals from lethal challenge with Clade Ia monkeypox virus and its tolerability in immune-compromised animals is encouraging and further supports testing in humans. We look forward to providing additional updates to each of these promising programs in 2025."
Key Product Candidates* -- Recent Highlights
Central Nervous System (CNS) Pipeline
TNX-102 SL (cyclobenzaprine HCl sublingual tablets): 5.6 mg, once-daily at bedtime small molecule for the management of fibromyalgia (FM), a centrally-acting, non-opioid analgesic.
In December 2024, the Company announced U.S. Food and Drug Administration (FDA) acceptance of its New Drug Application (NDA) for TNX-102 SL for fibromyalgia, with a Prescription Drug User Fee Act (PDUFA) goal date of August 15, 2025. The NDA was based upon two Phase 3 studies of TNX-102 SL in fibromyalgia that showed statically significant reduction in the chronic, widespread pain associated with fibromyalgia. It was well tolerated and has no known addictive properties. If approved by the FDA, TNX-102 SL would be the first member of a new class of tertiary amine tricyclic (TAT) non-opioid analgesic drugs for fibromyalgia and the first new drug available for treating fibromyalgia in more than 15 years. Fibromyalgia affects more than 10 million adults in the U.S., most of whom are women.
In March 2025, Tonix presented data and analyses of TNX-102 SL treatment and effects on fibromyalgia at the 7th International Congress on Controversies in Fibromyalgia, held in Vienna, Austria, in an oral presentation titled, "Transmucosal Sublingual Cyclobenzaprine (TNX-102 SL) Treatment of Fibromyalgia at Bedtime to Target Non-Restorative Sleep Showed Durable Pain Reduction in Two Double-Blind Randomized Phase 3 Studies." TNX-102 SL, a sublingual formulation of cyclobenzaprine designed for transmucosal delivery and durable activity, has demonstrated statistically significant, durable activity (three months) in reducing fibromyalgia pain in two double-blind randomized Phase 3 studies.
In November 2024, at the American College of Rheumatology (ACR) Convergence 2024 Annual Meeting, Tonix announced additional data and analyses of TNX-102 SL for the management of fibromyalgia. TNX-102 SL had met the pre-specified primary endpoint in the Phase 3 RESILIENT study, significantly reducing daily pain compared to placebo (p-value=0.00005) in participants with fibromyalgia with statistically significant improvement in all six pre-specified key secondary endpoints, including those related to improving sleep quality, reducing fatigue, and improving patient global ratings and overall fibromyalgia symptoms and function. TNX-102 SL was well tolerated with an adverse event profile comparable to prior studies and no new safety signals were observed.
In September 2024, at the 11th Global Conference on Pharmaceutics and Novel Drug Delivery Systems (PDDS 2024), the Company announced data highlighting the proprietary formulation technology and pharmacokinetic properties of TNX-102 SL, including composition and methods patents based on the proprietary eutectic formulation of TNX-102 SL that are expected to provide market exclusivity until at least 2034 in the U.S., EU, Japan, China and other jurisdictions. The eutectic protects cyclobenzaprine HCl from interacting with the basifying agent that is also part of the formulation and required for efficient transmucosal absorption. The formulation of TNX-102 SL was designed specifically for sublingual administration and transmucosal absorption for bedtime dosing to target disturbed sleep, relieve pain and other fibromyalgia symptoms, while reducing the risk of daytime somnolence.
TNX-102 SL for the treatment of acute stress reaction (ASR) and acute stress disorder (ASD), and prophylaxis against development of posttraumatic stress disorder (PTSD)
The DoD-funded Optimizing Acute Stress Reaction Interventions (OASIS) trial will be conducted by the University of North Carolina under an investigator-initiated investigational new drug (IND) application. The OASIS trial will examine the safety and efficacy of TNX-102 SL to reduce adverse posttraumatic neuropsychiatric sequelae among patients in the emergency department (ED) after a motor vehicle collision. Fourteen days of bedtime TNX-102 SL will be dosed and tested in the immediate aftermath of motor vehicle collision. The study will test the potential for TNX-102 SL to target trauma-related sleep disturbance and its ability to facilitate recovery from ASR and to prevent PTSD. The program has the potential to provide military personnel with a new treatment option that improves warfighter performance and resilience when administered in the early aftermath of a traumatic event, The study is expected to be initiated in the first half of 2025.
TNX-1300 (recombinant double mutant cocaine esterase): biologic for cocaine intoxication
The National Institutes of Health (NIH)'s National Institute of Drug Abuse (NIDA) previously awarded Tonix a Cooperative Agreement grant for approximately $5 million to support development of TNX-1300.
TNX-1300 has been granted Breakthrough Therapy designation by the FDA.
The Phase 2 CATALYST study of TNX-1300 for the treatment of cocaine intoxication began enrolling in August 2024. CATALYST is a Phase 2 single-blind, placebo-controlled, proof-of-concept study in patients presenting to the emergency department. Because of the challenges of recruiting eligible patients into this study, we are not guiding to a timeline for completion of enrollment or topline data.
Immunology Pipeline
TNX-1500 (anti-CD40L Fc-modified humanized monoclonal antibody): third generation anti-CD40L monoclonal antibody for prophylaxis for organ transplant rejection and treatment of autoimmune disorders.
In February 2025, Tonix announced positive topline results from its Phase 1, single ascending dose (SAD) first-in-human trial of TNX-1500 in healthy participants. The objectives of the Phase 1 trial were to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of intravenous TNX-1500, as well as to support dosing in a planned Phase 2 trial in kidney transplant recipients, pending alignment with the FDA. All objectives were met and support proceeding to a Phase 2 trial. TNX-1500 blocked the primary and secondary antibody responses to a test antigen at the 10 mg/kg and 30 mg/kg i.v. doses, showed mean half-life of 34-38 days for the 10 mg/kg and 30 mg/kg doses (supporting monthly dosing for future efficacy trials) and was generally well-tolerated with a favorable safety profile.
The first proposed indication for TNX-1500 is prophylaxis of organ rejection in adult patients receiving a kidney transplant; but multiple additional indications are possible, including the treatment of autoimmune diseases. Preclinical studies have shown that TNX-1500 maintains the activity of first-generation monoclonal antibodies (mAbs), yet with reduced risk of thrombotic complications. Pharmacokinetic data support a monthly i.v. dosing regimen. This analysis together with TNX-1500's activity and tolerability in animals, suggests that the protein engineering of TNX-1500's Fc region has achieved its design goals.
Infectious Disease Pipeline
TNX-801 (recombinant horsepox virus, minimally replicative live vaccine): potential vaccine to protect against mpox and smallpox.
In March 2025, Tonix announced it was awarded a grant from the Medical CBRN Defense Consortium (MCDC) to support the development of TNX-801. The grant will allow Tonix to develop a commercialization plan for TNX-801.
In November 2024, Tonix announced that it has entered into a sponsored research agreement with the Kenya Medical Research Institute (KEMRI) to design, plan and seek regulatory approval for a Phase I clinical study in Kenya to test the safety, tolerability, and immunogenicity of TNX-801 (horsepox, live virus) as a vaccine to prevent mpox and smallpox. Tonix is expected to be the sponsor and KEMRI is expected to lead the execution of the proposed clinical trial.
In November 2024, the Company announced the publication of a peer-reviewed paper highlighting the tolerability of TNX-801 in immune-compromised animals. The publication describes data in which TNX-801 was compared with older vaccinia vaccine strains used in the eradication of smallpox for tolerability in both in vitro and in vivo models. Together, TNX-801 was shown to be greater than 10- to 1,000-fold less virulent (or more attenuated) compared to the older vaccinia smallpox vaccines. Previously, single-dose vaccination with TNX-801 was shown to protect animals from a lethal challenge with Clade Ia monkeypox.
In September 2024, at the DoD's MHSRS conference and in October 2024 at the World Vaccine Congress in Barcelona, Spain, Tonix presented new data on potential mpox vaccine, TNX-801, demonstrating tolerability and no evidence of spreading to blood or tissues, even at high doses, in immunocompromised animals. 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. After a single-dose vaccination, TNX-801 prevented clinical disease and lesions, and also decreased shedding in the mouth and lungs of non-human primates after a lethal challenge with Clade Ia monkeypox. These findings are consistent with TNX-801 inducing mucosal immunity and suggest TNX-801 has the ability to block forward transmission.
In September 2024, the Company announced that the World Health Organization's (WHO) preferred target product profile (TPP) 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. In August 2024, the WHO determined that the upsurge of mpox in a growing number of countries in Africa constitutes a public health emergency of international concern (PHEIC), the second such declaration in the past two years in response to transmission of the virus. Mpox cases of the new Clade Ib mpox have since also been detected in many countries outside of Africa including at least three cases in the U.S.
Corporate and Partnerships, Recent Highlights
In February 2025, the Company announced the promotion of Siobhan Fogarty to Chief Technical Officer from Executive Vice President, Product Development. Ms. Fogarty originally joined Tonix in 2016 and has over 25 years of experience in pharmaceutical and biotech product development, manufacturing and quality, for both small and large molecules, at notable pharmaceutical and biotech companies.
In January 2025, Tonix announced the appointment of Gary Ainsworth as its new Vice President, Market Access. With over two decades of industry and market access experience, Mr. Ainsworth offers a significant track record of success building market access functions, developing launch-ready access and reimbursement strategies and payer-focused resources, including those for fibromyalgia and migraine treatment options.
In December 2024, the Company announced the expansion of its leadership team with the appointment of two strategic hires: Bradley Raudabaugh, MBA, joined Tonix as Vice President, Marketing, and Errol Gould, Ph.D., joined the company as Vice President, Medical Affairs. Mr. Raudabaugh brings over 25 years of marketing, sales and product planning experience to Tonix and Dr. Gould offers over 25 years of experience in R&D and medical affairs across a wide range of therapeutic areas, including fibromyalgia.
In October 2024, the Company announced the receipt of the first contract payment from the previously awarded contract by the U.S. Department of Defense (DoD) for accelerated development of broad-spectrum antivirals with the Defense Threat Reduction Agency (DTRA). The contract, awarded in July 2024, has the potential for up to $34 million over five years with an objective to develop small molecule broad-spectrum antiviral agents for the prevention or treatment of infections to improve the medical readiness of military personnel in biological threat environments. Tonix's program is focusing on optimization and development of its TNX-4200 program, to develop an orally available CD45 antagonist, with broad-spectrum efficacy against a range of viral families through preclinical evaluation. The program is expected to establish physicochemical properties, pharmacokinetics, and safety attributes to support an IND submission and to fund a first-in-human Phase 1 clinical study.
In October 2024, the Company announced it entered into an artificial intelligence and machine learning drug discovery collaboration with X-Chem, Inc., a leader in small molecule drug discovery, to accelerate the development of small molecules as orally available host-targeted broad-spectrum medical countermeasures. Tonix's TNX-4200 antiviral program focuses on the development of oral CD45 phosphatase inhibitors, with broad-spectrum activity against a range of viral families.
In September 2024, Tonix announced the appointment of Thomas Englese as its new Executive Vice President, Commercial Operations. Mr. Englese brings significant leadership to Tonix across several functions, including commercial operations, sales and marketing, and launching and managing major brands through all stages of commercialization.
Marketed Products, Recent Highlights
In September 2024, Tonix announced that the United States Patent and Trademark Office (USPTO) issued U.S. Patent No. 12,097,183 to the Company, claiming use of a pre-filled autoinjector comprising a composition of Zembrace® SymTouch® for treating migraines via subcutaneous administration. This patent, excluding possible patent term extensions, is expected to fortify protection and market exclusivity into 2036.
Tonix announced that the USPTO issued U.S. Patent No. 12,090,139 to the Company, claiming a pharmaceutical composition, a method of treating migraine via intranasal administration, and an intranasal delivery system for Tosymra®. This patent is expected to fortify protection and market exclusivity into 2030.
In September 2024, Tonix Medicines launched a national educational campaign focusing on the link between migraine, gastroparesis, and the need for non-oral acute migraine therapies. Tonix Medicines is the only manufacturer with both a branded injectable and nasal spray indicated for the acute treatment of migraine with or without aura in adults.
Financial - Recent Highlight
Tonix had approximately $98.8 million of cash and cash equivalents as of December 31, 2024, compared to approximately $24.9 million as of December 31, 2023. Net cash used in operations was approximately $60.9 million for the full year ended December 31, 2024, compared to $102.0 million for the same period in 2023. Net cash used in investing activities for the full year ended ...
