Tonix Pharmaceuticals Announces That its Single Dose Mpox Vaccine Candidate TNX-801 Aligns with WHO’s Newly Issued Preferred Target Product Profile for Mpox Vaccines in Global Health Emergency
Tonix Pharmaceuticals (Nasdaq: TNXP) announced that its single-dose mpox vaccine candidate TNX-801 aligns with the World Health Organization's (WHO) newly issued preferred target product profile (TPP) for mpox vaccines. The WHO released its TPP criteria at the Mpox Research and Innovation Scientific Conference held August 29-30, 2024. Key elements of the WHO draft TPP include single-dose, durable protection, administration without special equipment, and stability at ambient temperature.
TNX-801 has shown single dose protection against a lethal challenge of Clade I monkeypox virus in animal studies. It is designed for percutaneous administration using a bifurcated needle and is expected to have stability at ambient temperature, eliminating the need for ultra-cold storage. The vaccine may also be suitable for use in immunocompromised individuals.
Tonix recently announced a collaboration with Bilthoven Biologics to develop GMP manufacturing processes for its mpox vaccine. The WHO's declaration of mpox as a Public Health Emergency of International Concern (PHEIC) underscores the urgent need for new vaccines to control the outbreak.
Tonix Pharmaceuticals (Nasdaq: TNXP) ha annunciato che il suo candidato vaccinale monodose per mpox TNX-801 è in linea con il profilo di prodotto target preferito (TPP) recentemente emesso dall'Organizzazione Mondiale della Sanità (OMS) per i vaccini mpox. L'OMS ha rilasciato i criteri TPP durante la Conferenza Scientifica di Ricerca e Innovazione su mpox tenutasi il 29-30 agosto 2024. I principali elementi del TPP provvisorio dell'OMS includono monodose, protezione duratura, somministrazione senza attrezzature speciali e stabilità a temperatura ambiente.
TNX-801 ha mostrato protezione con una sola dose contro una sfida letale del virus della monkeypox Clade I negli studi sugli animali. È progettato per la somministrazione percutanea utilizzando un ago biforcato ed è previsto che abbia stabilità a temperatura ambiente, eliminando la necessità di stoccaggio a ultra-freddo. Il vaccino potrebbe anche essere adatto per l'uso in individui immunocompromessi.
Tonix ha recentemente annunciato una collaborazione con Bilthoven Biologics per sviluppare processi di produzione GMP per il suo vaccino mpox. La dichiarazione dell'OMS su mpox come Emergenza Sanitaria Pubblica di Preoccupazione Internazionale (PHEIC) sottolinea l'urgenza di nuovi vaccini per controllare l'epidemia.
Tonix Pharmaceuticals (Nasdaq: TNXP) anunció que su candidato a vacuna monodosis para mpox TNX-801 se alinea con el perfil de producto objetivo preferido (TPP) recientemente emitido por la Organización Mundial de la Salud (OMS) para las vacunas mpox. La OMS publicó sus criterios TPP en la Conferencia Científica de Investigación e Innovación sobre mpox celebrada del 29 al 30 de agosto de 2024. Los elementos clave del TPP provisional de la OMS incluyen monodosis, protección duradera, administración sin equipo especial y estabilidad a temperatura ambiente.
TNX-801 ha mostrado protección de una sola dosis contra un desafío letal del virus de la monkeypox Clade I en estudios con animales. Está diseñado para la administración percutánea utilizando una aguja bifurcada y se espera que tenga estabilidad a temperatura ambiente, eliminando la necesidad de almacenamiento a ultra frío. La vacuna también podría ser adecuada para su uso en individuos inmunocomprometidos.
Tonix recientemente anunció una colaboración con Bilthoven Biologics para desarrollar procesos de fabricación GMP para su vacuna mpox. La declaración de la OMS sobre mpox como Emergencia de Salud Pública de Preocupación Internacional (PHEIC) subraya la urgente necesidad de nuevas vacunas para controlar el brote.
Tonix Pharmaceuticals (Nasdaq: TNXP)는 단일 투여량 mpox 백신 후보 TNX-801가 세계보건기구(WHO)가 최근 발표한 mpox 백신을 위한 선호 제품 목표 프로필(TPP)과 일치한다고 발표했습니다. WHO는 2024년 8월 29일과 30일에 열린 Mpox 연구 및 혁신 과학 회의에서 TPP 기준을 발표했습니다. WHO 초안 TPP의 핵심 요소에는 단일 투여량, 지속적인 보호, 특수 장비 없이 투여 가능 및 실온에서의 안정성이 포함됩니다.
TNX-801은 동물 연구에서 Clade I 몽키폭스 바이러스의 치명적인 도전에 대한 단일 투여량 보호 효과를 나타낸 바 있습니다. 이는 두 갈래 바늘을 이용한 경피적 투여를 위해 설계되었으며, 상온에서 안정성을 유지할 것으로 예상되어 초저온 저장이 필요 없습니다. 이 백신은 면역 억제 개인에게 사용하기에도 적합할 수 있습니다.
Tonix는 최근 Bilthoven Biologics와 협력하여 mpox 백신의 GMP 제조 공정을 개발하기로 발표했습니다. WHO가 mpox를 국제적 관심의 공공 보건 비상사태(PHEIC)로 선언한 것은 발병을 통제하기 위한 새로운 백신의 긴급한 필요성을 강조합니다.
Tonix Pharmaceuticals (Nasdaq: TNXP) a annoncé que son candidat vaccin monodose pour mpox TNX-801 est en adéquation avec le profil de produit cible préféré (TPP) récemment publié par l'Organisation mondiale de la santé (OMS) pour les vaccins mpox. L'OMS a publié ses critères TPP lors de la conférence scientifique de recherche et d'innovation sur mpox qui s'est tenue les 29 et 30 août 2024. Les éléments clés du TPP provisoire de l'OMS comprennent monodose, protection durable, administration sans équipements spéciaux et stabilité à température ambiante.
TNX-801 a montré une protection par dose unique contre un défi létal du virus monkeypox de Clade I dans des études animales. Il est conçu pour une administration par voie percutanée à l'aide d'une aiguille bifurquée et devrait avoir une stabilité à température ambiante, éliminant ainsi le besoin de stockage à ultra-froid. Le vaccin pourrait également convenir à une utilisation chez les personnes immunodéprimées.
Tonix a récemment annoncé une collaboration avec Bilthoven Biologics pour développer des processus de fabrication GMP pour son vaccin mpox. La déclaration de l'OMS sur mpox en tant qu'urgence de santé publique de portée internationale (PHEIC) souligne l'urgence de nouveaux vaccins pour contrôler l'épidémie.
Tonix Pharmaceuticals (Nasdaq: TNXP) gab bekannt, dass sein monodosen Impfstoffkandidat für mpox TNX-801 mit dem neu veröffentlichten bevorzugten Zielproduktprofil (TPP) der Weltgesundheitsorganisation (WHO) für mpox-Impfstoffe übereinstimmt. Die WHO veröffentlichte ihre TPP-Kriterien während der Mpox-Forschungs- und Innovationskonferenz, die am 29. und 30. August 2024 stattfand. Zu den wichtigsten Elementen des vorläufigen TPP der WHO gehören einmalige Dosis, dauerhafte Schutzwirkung, Verabreichung ohne spezielle Ausrüstung und Stabilität bei Raumtemperatur.
TNX-801 hat Schutz mit einer einzigen Dosis gegen eine tödliche Herausforderung des Clade I Monkeypox-Virus in Tierstudien gezeigt. Es ist für die perkutanen Verabreichung mit einer gegabelten Nadel konzipiert und wird voraussichtlich bei Raumtemperatur stabil sein, wodurch die Notwendigkeit einer ultrakalten Lagerung entfällt. Der Impfstoff könnte auch für immun geschwächte Personen geeignet sein.
Tonix kündigte kürzlich eine Zusammenarbeit mit Bilthoven Biologics zur Entwicklung von GMP-Herstellungsverfahren für seinen mpox-Impfstoff an. Die Erklärung der WHO zu mpox als Notfall für die öffentliche Gesundheit von internationaler Bedeutung (PHEIC) unterstreicht den dringenden Bedarf an neuen Impfstoffen zur Bekämpfung des Ausbruchs.
- TNX-801 aligns with WHO's preferred target product profile for mpox vaccines
- TNX-801 has shown single dose protection against lethal Clade I monkeypox virus in animal studies
- TNX-801 is expected to have stability at ambient temperature, eliminating need for ultra-cold storage
- Collaboration with Bilthoven Biologics to develop GMP manufacturing processes for TNX-801
- Potential suitability of TNX-801 for use in immunocompromised individuals
- TNX-801 is still in development and not yet approved for human use
- Efficacy and safety in humans yet to be demonstrated through clinical trials
Insights
The alignment of Tonix's TNX-801 vaccine with WHO's preferred target product profile for mpox vaccines is a significant development. Key advantages of TNX-801 include:
- Single-dose protection, potentially improving vaccination coverage and enabling efficient "ring vaccination" strategies
- Potential to block forward transmission, important for controlling outbreaks
- Stability at ambient temperature, eliminating the need for ultra-cold storage
- Possible suitability for immunocompromised individuals, addressing a critical population in mpox-affected regions
These features could position TNX-801 as a valuable tool in combating the global mpox emergency, particularly in resource- settings. However, it's important to note that while animal studies are promising, human clinical trials are necessary to confirm efficacy and safety.
The mpox vaccine market is evolving rapidly due to the recent WHO declaration of a Public Health Emergency of International Concern. Tonix's TNX-801 could potentially capture a significant market share if it proves successful in human trials. Key market factors include:
- competition, with only two FDA-approved vaccines currently available
- Growing demand, especially in Africa and emerging hotspots
- Potential for government contracts and stockpiling
- Collaboration with Bilthoven Biologics, part of the world's largest vaccine manufacturer, which could streamline production and distribution
However, Tonix faces challenges from established players like Bavarian Nordic and potential new entrants like Moderna. The company's ability to quickly advance TNX-801 through clinical trials and regulatory approval will be important for market success.
The World Health Organization (WHO) released its preferred target product profile (TPP) criteria for mpox vaccines at its Mpox Research and Innovation Scientific Conference held August 29-30
TNX-801, Tonix’s attenuated live-virus vaccine candidate, has characteristics that align closely with WHO’s TPP
On August 14, 2024, the WHO determined that the upsurge of mpox in a growing number of countries in Africa constitutes a public health emergency of international concern1-4: cases of the potentially lethal new Clade I mpox also detected in Sweden, Thailand and Singapore
CHATHAM, N.J., Sept. 16, 2024 (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, announced today that the World Health Organization’s (WHO’s) preferred target product profile (TPP), released at the WHO sponsored Mpox Research and Innovation Scientific Conference held August 29-30, 2024, aligns with the characteristics of TNX-801 (horsepox, live virus) vaccine, which is being developed for preventing mpox (formerly known as monkeypox). 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.
“The characteristics of TNX-801 align with the draft TPP released at the WHO sponsored Mpox Research and Innovation Scientific Conference,” said Seth Lederman, M.D., Chief Executive Officer of Tonix. “In animal studies TNX-801 has shown single dose protection against a lethal challenge of Clade I monkeypox virus administered by intratracheal route.5 In addition, protected animals did not produce any infectious virus suggesting TNX-801 has the potential to block forward transmission as expected with live-virus vaccines. TNX-801 is designed for percutaneous administration using a bifurcated needle, like the products and delivery used in WHO’s accelerated smallpox eradication project. Since TNX-801 is a live-virus vaccine, we expect the stability of lyophilized TNX-801 at ambient temperature to be similar to live vaccinia virus vaccines including ACAM2000. We believe TNX-801 can be shipped and stored without the need for a costly and cumbersome ultra-cold supply chain, a particular advantage in lesser developed parts of the world. The stability of live virus vaccines eliminates the need for ultra-cold storage which complicates the widespread use of mRNA vaccines in Africa, where they are needed most right now. Finally, studies on immunocompromised animals6 suggest that TNX-801 may be given to persons with immunocompromising conditions such as HIV, which is another property that will be essential for public health.”
Dr. Lederman continued, “The recent WHO declaration of a Public Health Emergency of International Concern (PHEIC) underscores the urgent need for new vaccines to control this outbreak and save lives. We have been motivated to develop TNX-801 because single-dose vaccines simplify logistics of administration, achieve higher coverage by reducing vaccinee dropout between doses and allow for case-contact or “ring” strategies to vaccinate the contacts of confirmed mpox patients.7,8 Ring vaccination is deemed essential for controlling mpox but requires single-dose vaccines that interrupt forward transmission.”7,8
On August 26, 2024, Tonix announced a collaboration with Bilthoven Biologics (Bbio) to develop GMP manufacturing processes for its mpox vaccine. Bbio is part of the world’s largest vaccine manufacturer, the Cyrus Poonawalla Group, which also includes the Serum Institute of India.
The U.S. Food and Drug Administration (FDA) approved vaccines for mpox are a two-dose non-replicating vaccine called Jynneos® from Bavarian Nordic9 and a one-dose live-virus vaccine from Emergent for people at high risk for mpox infection.10 WHO recently authorized Jynneos for use in adults.11 Recently data in animals have been reported for a two-dose mRNA vaccine from Moderna.12
About TNX-801*
TNX-801 is a live replicating attenuated vaccine based on horsepox that is believed to provide immune protection with better tolerability than 20th Century vaccinia viruses. As previously disclosed, TNX-801 protected animals against lethal challenge with intratracheal Clade I monkeypox virus.5 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.6 The Findings are consistent with mucosal immunity and suggest the ability to block forward transmission, similar to Dr. Edward Jenner’s vaccinia vaccine, which eradicated smallpox and kept mpox out of the human population. TNX-801 combines immune protection with improved tolerability compared to other vaccines based on orthopoxviruses and is administered with a single dose which has advantages over two-dose regimens. The focus on single-dose vaccines confirms early recommendations by the Bipartisan Commission on Biodefense, 7 and the U.S. National Academies of Science.7,8 The National Academies of Science (NAS) report highlights the difficulty of a ring vaccination strategy with even a two-dose regimen. 7 The U.S. National Institutes of Health (NIH) selected Tonix’s COVID-19 vaccine, TNX-1800 for Project NextGen. TNX-1800 is an engineered version of horsepox that expresses the spike protein of SARS-CoV-2. 13,14
About Mpox*
On August 14, 2024, the WHO determined that the upsurge of mpox in a growing number of countries in Africa constitutes a PHEIC the second such declaration in the past two years called in response to an mpox outbreak.1 The current outbreak is caused by Clade I monkeypox virus, while the 2022 outbreak was Clade 2 monkeypox virus. The global mpox outbreak, which commenced in 2022 has affected over 90,000 persons in countries where mpox had previously not been endemic, including Europe and the US. The spread of Clade IIb strain mpox in 2022 underscores the pandemic potential of mpox. Unlike Clade IIb mpox, the Clade I strain of mpox appears to be spreading to countries neighboring the Democratic Republic of the Congo, including Burundi, Rwanda, Uganda and Kenya. Clade I mpox is typically associated with approximately twenty times the case fatality rates than Clade IIb mpox in Africa. According to the U.S. Centers for Disease Control and Prevention (CDC), and other experts, there is a significant risk that the deadlier Clade I strain may appear in the U.S.2,3
Tonix Pharmaceuticals Holding Corp.*
Tonix is a fully-integrated biopharmaceutical company focused on developing, licensing and commercializing therapeutics to treat and prevent human disease and alleviate suffering. Tonix’s priority is to submit a New Drug Application (NDA) to the FDA in October of 2024 for TNX-102 SL, a product candidate for which two statistically significant Phase 3 studies have been completed for the management of fibromyalgia. The FDA has 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. Tonix recently announced the U.S. Department of Defense (DoD), Defense Threat Reduction Agency (DTRA) awarded it a contract for up to
*Tonix’s product development candidates are investigational new drugs or biologics 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.
This press release and further information about Tonix can be found at www.tonixpharma.com.
1WHO Press Release August 14, 2024. “WHO Director-General declares mpox outbrfeak a public health emergency of international concern”. URL: www.who.int/news/item/14-08-2024-who-director-general-declares-mpox-outbreak-a-public-health-emergency-of-international-concern (accessed 8-15-24)
2McQuiston JH, et al. U.S. Preparedness and Response to Increasing Clade I Mpox Cases in the Democratic Republic of the Congo. 2024, MMWR Morbi Mortal Wkly Rep: United States. p. 435-440
3CDC. 2022-2023 Mpox: US Map and Case Count. https://www.cdc.gov/poxvirus/mpox/response/2022/us-map.html
4World Health Organization SAGE meeting highlights on updated mpox vaccine recommendations. 2024, March
5Noyce RS, et al. Viruses. 2023 Jan 26;15(2):356. Doi: 10.3390/v15020356. PMID: 36851570; PMCID: PMC9965234
6Trefry, SV et al. bioRxiv 2023.10.25.564033; doi: https://doi.org/10.1101/2023.10.25.564033
7Bipartisan Commission on Biodefense. Box the Pox: Reducing the risk of Smallpox and Other Ortho poxviruses, Washington:2024
8U.S. National Academies of Science. Future State of Smallpox Medical Countermeasures. Washington:2024
9Zaeck LM, et al. Low levels of monkeypox virus-neutralizing antibodies after MVA-BN vaccination in healthy individuals. Nat Med. 2023 Jan;29(1):270-278. doi: 10.1038/s41591-022-02090-w. Epub 2022 Oct 18. PMID: 36257333; PMCID: PMC9873555.
10August 30, 2024. Reuters. “US FDA approves Emergent's smallpox vaccine for people at high risk of mpox”. https://www.msn.com/en-us/health/other/us-fda-approves-emergent-s-smallpox-vaccine-for-people-at-high-risk-of-mpox/
11Keaton, J. Sept. 13, 2024. Associated Press. “WHO grants first mpox vaccine approval to ramp up response to disease in Africa.” URL: https://bit.ly/4e4yyeb
12Mucker et al., (in press) Comparison of protection against mpox following mRNA or modified vaccinia Ankara vaccination in nonhuman primates, Cell (2024), https://doi.org/10.1016/j.cell.2024.08.043
13Awasthi M, et al. Viruses. 2023 Oct 21;15(10):2131. Doi: 10.3390/v15102131. PMID: 37896908; PMCID: PMC10612059.
14Awasthi M, et al. Vaccines (Basel). 2023 Nov 2;11(11):1682. Doi: 10.3390/vaccines11111682.PMID: 38006014
Forward Looking Statements
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FAQ
What are the key features of Tonix Pharmaceuticals' mpox vaccine candidate TNX-801?
How does TNX-801 align with WHO's target product profile for mpox vaccines?
What collaboration has Tonix Pharmaceuticals (TNXP) announced for TNX-801 manufacturing?
When did WHO declare mpox a Public Health Emergency of International Concern (PHEIC)?
