Full Press Release Details
Tonix Pharmaceuticals Holding Corp. 8-K
Tonix Pharmaceuticals Presents Data from its
Vaccine Development Program at the World Vaccine Congress
Preclinical data demonstrate the efficacy
of TNX-801 vaccination against mpox virus challenge in a non-human primate model
Phase 1 trial with TNX-801 for the prevention
of mpox and smallpox is expected to start in the second half of 2023
CHATHAM, N.J., April 6, 2023 (GLOBE NEWSWIRE)
- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, presented
data from its live virus vaccine platform development program in two oral presentations at the World Vaccine Congress being held in Washington
D.C., April 3-6, 2023. Copies of the Company's presentations are available under the Scientific
Presentations tab of the Tonix website at www.tonixpharma.com.
"Tonix's live virus vaccine technology
is designed to help protect against emerging infectious diseases by providing durable protection, and can be widely deployed without the
need for sterile injection or ultra-cold shipping and storage," said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals.
"Our lead vaccine candidate, TNX-801, is a live virus vaccine in development to protect against monkeypox ( mpox') and
smallpox. We believe TNX-801 is closer in genetic structure and properties to the smallpox vaccines used in the U.S. and Europe before
1900 than the modern vaccinia smallpox vaccines. Relative to modern vaccinia vaccines, TNX-801 has reduced virulence in animals, and we
believe it has the potential for widespread use to protect against mpox and smallpox."
The presentation, titled, "TNX-801:
A Live Attenuated Orthopoxvirus (Horsepox) Vaccine for Mpox and Smallpox," describes the history of live virus vaccines and
the rationale for the development of the Company's Recombinant Pox Virus (RPV) platform. The presentation demonstrates that non-human
primates vaccinated with TNX-801 were fully protected with sterilizing immunity from a lethal challenge.
The presentation, titled, "The Development
of Horsepox virus as a vaccine platform: Evaluation of TNX-1800 as a SARS-CoV-2 Vaccine," describes TNX-1800, a live virus vaccine
based on the horsepox viral vector platform, which was developed to protect against COVID-19. The presentation shows that in animal testing,
TNX-1800 protected upper and lower airways after challenge with SARS-CoV-2, suggesting an ability to block forward transmission. TNX-1800
is an example of the ability of Tonix's adaptable vaccine platform to protect against emerging threats and future pandemics.
TNX-801 is a live virus vaccine based on
horsepox2,3. Tonix is developing TNX-801 for percutaneous administration as a vaccine to protect against mpox and smallpox.
Tonix's TNX-801 is based on the sequence of the 1976 natural isolate Mongolian horsepox clone MNR-763.2 Molecular
analysis of DNA sequences suggests that TNX-801 is closer than modern smallpox vaccines to the vaccine discovered and disseminated by
Dr. Edward Jenner in 17984-6. For example, recent studies7,8 have shown approximately 99.7% colinear identity
between TNX-801 and the circa 1860 U.S. smallpox vaccine VK05.9 The small plaque size in culture of TNX-801 appears identical
to the U.S. Centers for Disease Control publication of the natural isolate10. Relative to vaccinia, horsepox has substantially
decreased virulence in mice2. Dr. Edward Jenner invented vaccination in 1798 and the procedure was called "vaccination"
because cow' is vacca' in Latin and the inoculum material was initially obtained from lesions on the udders
of cows affected by a mild disease known as cowpox. However, Dr. Jenner suspected that cowpox originated from horses6. Subsequently,
Dr. Jenner and others immunized against smallpox using material directly obtained from horses. The use of vaccines from horses was sometimes
called equination' from the Latin equus' which means horse'11. Equination and vaccination
were practiced side-by-side in Europe11,12.
About the Recombinant Pox Virus (RPV)
Horsepox virus and vaccines based on its
use as a vector are live replicating viruses that elicit strong immune responses. Live replicating orthopoxviruses, like vaccinia or horsepox,
can be engineered to express foreign genes and have been exploited as platforms for vaccine development because they possess; (1) large
packaging capacity for exogenous DNA inserts, (2) precise virus-specific control of exogenous gene insert expression, (3) lack of persistence
or genomic integration in the host, (4) strong immunogenicity as a vaccine, (5) ability to rapidly generate vector/insert constructs,
(6) manufacturable at scale, and (7) ability to provide direct antigen presentation. Horsepox-based vaccines are designed to be single
dose, vial-sparing vaccines, that can be manufactured using conventional cell culture systems, with the potential for mass scale production
and packaging in multi-dose vials. Tonix's TNX-801 and RPV vaccine candidates are administered percutaneously using a two-pronged,
or "bifurcated" needle. The major cutaneous reaction or "take" to vaccinia vaccine was described by Dr. Edward
Jenner in 1796 and has been used since then as a biomarker for protective immunity to smallpox, including in the World Health Organization's
(WHO) accelerated smallpox eradication program that successfully eradicated smallpox in the 1960's. The "take" is a
measure of functional T cell immunity validated by the eradication of smallpox, a respiratory-transmitted disease caused by variola.
About Mpox and Smallpox
Mpox13 and smallpox14
are diseases in humans caused by the mpox and smallpox (or variola) viruses, respectively. Mpox and variola are closely related orthopox
viruses. Vaccination against smallpox with live virus vaccines based on horsepox or vaccinia protects against mpox. After routine smallpox
vaccination was stopped in about 1970, mpox has become a growing problem in Africa. Since May of 2022, approximately 30,000 cases have
been identified in the United States15,16. There are two distinct clades of the mpox virus: the central African (Congo Basin)
clade, and the west African clade which is associated with the recent outbreak. Historically, the Congo Basin clade has caused more severe
disease than the west African clade. In recent times, the case fatality ratio for the virus is about 3-6%17. In November
2022, the WHO began using a new preferred term "mpox" as a synonym for monkeypox18. Smallpox is considered eradicated,
but there are concerns about malicious reintroduction.
Tonix Pharmaceuticals Holding Corp.1
Tonix is a clinical-stage
biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics to treat and prevent human disease
and alleviate suffering. Tonix's portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious
disease product candidates. Tonix's CNS portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric
and addiction conditions. Tonix's lead CNS candidate, TNX-102 SL (cyclobenzaprine HCl sublingual tablet), is in mid-Phase 3 development
for the management of fibromyalgia with interim data expected in the second quarter of 2023. TNX-102 SL is also being developed to treat
Long COVID, a chronic post-acute COVID-19 condition. Enrollment in a Phase 2 study has been completed, and topline results are expected
in the third quarter of 2023. TNX-1900 (intranasal potentiated oxytocin), in development for chronic migraine, is currently enrolling
with interim data expected in the fourth quarter of 2023. TNX-601 ER (tianeptine hemioxalate extended-release tablets), a once-daily formulation
being developed as a treatment for major depressive disorder (MDD), is also currently enrolling with interim data expected in the fourth
quarter of 2023. TNX-1300 (cocaine esterase) is a biologic designed to treat cocaine intoxication and has been granted Breakthrough Therapy
designation by the FDA. A Phase 2 study of TNX-1300 is expected to be initiated in the second quarter of 2023. Tonix's rare disease
portfolio includes TNX-2900 (intranasal potentiated oxytocin) for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan
Drug designation by the FDA. Tonix's immunology portfolio includes biologics to address organ transplant rejection, autoimmunity
and cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the
prevention of allograft and xenograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 is expected
to be initiated in the second quarter of 2023. Tonix's infectious disease pipeline includes TNX-801, a vaccine in development to
prevent smallpox and mpox, for which a Phase 1 study is expected to be initiated in the second half of 2023. TNX-801 also serves as the
live virus vaccine platform or recombinant pox vaccine platform for other infectious diseases. The infectious disease portfolio also includes
TNX-3900 and TNX-4000, classes of broad-spectrum small molecule oral antivirals.
This press release and further information about
Tonix can be found at www.tonixpharma.com.
of Tonix's product candidates are investigational new drugs or biologics and have not been approved for any indication.
2Noyce RS, et al. (2018) PLoS One. 13(1):e0188453
ER, et al. (2006) J Virol. 80(18):9244-58.PMID:16940536
L et al. (2017) N Engl J Med. 377:1491.
et al. (2015) J. Virol. 89:1809.
E. "An Inquiry Into the Causes and Effects of the Variolae Vaccinae: A Disease Discovered in Some of the Western Counties of England,
Particularly Gloucestershire, and Known by the Name of the Cow Pox." London: Sampson Low, 1798.
A et al, Genome Biology (2020) 21:286 https://doi.org/10.1186/s13059-020-02202-0
A et al. Genome Biology (2020) 21:175 https://doi.org/10.1186/s13059-020-02079-z
GS et al. (2016) Viruses (12). Pii: E328. PMID:27973399
E, et al (2017) Vaccine. 35(52):7222-7230.
J et al. (2020) Vaccine.; 38(30):4773-4779.