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 $34 million over five years in an
Other Transaction Agreement (OTA) to develop TNX-4200 small
molecule broad-spectrum antiviral agents 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. The company’s Good
Manufacturing Practice (GMP)-capable advanced manufacturing
facility in Dartmouth, MA was purpose-built to manufacture TNX-801
and the GMP suites are ready to be reactivated in case of a
national or international emergency. Tonix’s development portfolio
is focused on central nervous system (CNS) disorders. Tonix’s CNS
portfolio includes TNX-1300 (cocaine esterase), a biologic in Phase
2 development designed to treat cocaine intoxication that has
Breakthrough Therapy designation. Tonix’s immunology development
portfolio consists of 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 rejection
and for the treatment of autoimmune diseases. Tonix also has
product candidates in development in the areas of rare disease and
infectious disease. 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 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. 435-4403CDC. 2022-2023 Mpox: US Map and Case Count.
https://www.cdc.gov/poxvirus/mpox/response/2022/us-map.html4World
Health Organization SAGE meeting highlights on updated mpox vaccine
recommendations. 2024, March5Noyce RS, et al. Viruses. 2023 Jan
26;15(2):356. Doi: 10.3390/v15020356. PMID: 36851570; PMCID:
PMC99652346Trefry, SV et al.
bioRxiv 2023.10.25.564033; doi: https://doi.org/10.1101/2023.10.25.5640337Bipartisan
Commission on Biodefense. Box the Pox: Reducing the risk of
Smallpox and Other Ortho poxviruses, Washington:20248U.S. National
Academies of Science. Future State of Smallpox Medical
Countermeasures. Washington:20249Zaeck 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/4e4yyeb12Mucker 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.2024.08.04313Awasthi 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 Certain statements in this press release
are forward-looking within the meaning of the Private Securities
Litigation Reform Act of 1995. These statements may be identified
by the use of forward-looking words such as “anticipate,”
“believe,” “forecast,” “estimate,” “expect,” and “intend,” among
others. These forward-looking statements are based on Toni’s
current expectations and actual results could differ materially.
There are a number of factors that could cause actual events to
differ materially from those indicated by such forward-looking
statements. These factors include, but are not limited to, risks
related to the failure to obtain FDA clearances or approvals and
noncompliance with FDA regulations; risks related to the failure to
successfully market any of our products; risks related to the
timing and progress of clinical development of our product
candidates; our need for additional financing; uncertainties of
patent protection and litigation; uncertainties of government or
third party payor reimbursement; limited research and development
efforts and 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. Tonix does not undertake an
obligation to update or revise any forward-looking statement.
Investors should read the risk factors set forth in the Annual
Report on Form 10-K for the year ended December 31, 2023, as filed
with the Securities and Exchange Commission (the “SEC”) on April 1,
2024, and periodic reports filed with the SEC on or after the date
thereof. All of Toni’s forward-looking statements are expressly
qualified by all such risk factors and other cautionary statements.
The information set forth herein speaks only as of the date
thereof.
Investor Contact
Jessica MorrisTonix
Pharmaceuticalsinvestor.relations@tonixpharma.com (862)
904-8182
Peter VozzoICR Westwickepeter.vozzo@westwicke.com (443)
213-0505
Media Contact
Ray JordanPutnam Insightsray@putnaminsights.com (949)
245-5432
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