Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the
Company), a clinical-stage biopharmaceutical company, today
announced it received the official written response from a Type B
pre-Investigational New Drug (IND) meeting with the U.S. Food and
Drug Administration (FDA) to develop TNX-18001 (recombinant
horsepox virus, live vaccine) as a potential SARS-CoV-2 vaccine to
protect against COVID-19.
Tonix believes the written response provides a
path to agreements on the design of a Phase 1 study and the overall
clinical development plan to qualify TNX-1800 as a vaccine to
prevent COVID-19. Based on the response, the Company expects to
begin a Phase 1 study in the first half of 2022.
TNX-1800 is a live virus vaccine based on the
horsepox viral vector platform designed to express the SARS-CoV-2
spike protein and to protect against COVID-19, primarily by
eliciting a T cell response. Tonix reported positive efficacy data
from animal studies of TNX-1800 in the first quarter of 2021. The
horsepox virus is closely related to the vaccine developed by Dr.
Edward Jenner more than 200 years ago that led to the eradication
of smallpox.
Seth Lederman, M.D., President and Chief
Executive Officer of Tonix, stated, “The pre-IND meeting written
response marks an important milestone in the development of
TNX-1800. We have obtained FDA concurrence and clear guidance on
the proposed manufacturing, nonclinical pharmacology and toxicology
studies, and the Phase 1 clinical design.” Dr. Lederman continued,
“Operation Warp Speed (OWS) vaccines were available very rapidly
and have made a huge contribution to the health of the U.S.
population, but they have limitations, particularly in terms of the
short duration of protection and the likely requirement for
boosters. Concerns about durability of protection have led Pfizer
and Moderna, the innovators of the two most widely used COVID-19
vaccines in U.S., to file or plan to file for approval of booster
shots within eight months after the administration of each
vaccine’s second dose.”
“It’s taken longer to develop live virus
vaccines relative to the OWS vaccines,” Dr. Lederman added, “but
live virus vaccines for other viruses have proven to induce durable
T cell immunity, prevent serious illness after infection and block
forward transmission. These properties have been demonstrated with
vaccines against smallpox, chickenpox, mumps, measles, and rubella,
among others. We designed TNX-1800 as a potential single dose
vaccine using a virus that is closely related to Dr. Jenner’s
vaccine, which provided long term, even lifetime T cell immunity to
smallpox, prevented forward transmission of the smallpox virus, and
eradicated that disease.”
Dr. Lederman continued, “Unlike smallpox, we do
not expect COVID-19 to be eradicated because there are asymptomatic
spreaders, a relatively long period when people are infectious
before they become symptomatic and numerous animal reservoirs. Like
Jenner’s smallpox vaccine, we expect TNX-1800 can potentially be
scaled up for manufacturing and will not require a costly and
cumbersome cold chain for distribution and storage. We expect it
will also be glass-sparing, with 100 doses filled per vial.”
“Many believe that after the COVID pandemic
passes COVID will become endemic, and it will likely remain a
long-term concern,” Dr. Lederman added. “Because of this, we
believe the need for new vaccine technologies will be ongoing and
may lead to vaccines that are tailored to each person’s health,
genetics or age using precision medicine. Ultimately, a childhood
vaccination like the MMR for measles, mumps and rubella with long
lasting protection may be what’s needed to control COVID in the
future. Together with an expected strong and durable immune
response, ability to manufacture at scale, store and ship in
standard refrigeration, a live virus vaccine like TNX-1800 could
become a global product.”
Anthony Macaluso, Ph.D., Executive Vice
President of Strategic Development of Tonix, commented, “We
previously reported the positive results of TNX-1800 in animals
after a live SARS-CoV-2 challenge. Animals vaccinated with TNX-1800
had undetectable SARS-CoV-2 in their upper and lower airways six
days after challenge with SARS-CoV-2. Animals vaccinated with
TNX-1800 manifested both neutralizing antibodies and a ‘take’,
which is a skin reaction to horsepox vaccination that also serves
as a validated biomarker of functional T cell immunity.”
Dr. Macaluso continued, “The ’take‘ is
considered important because it is otherwise difficult and costly
to measure the T cell response to a vaccine. Vaccines that elicit a
strong T cell response, like horsepox and closely related vaccinia
virus vaccine, have been established to provide long-term, durable
immunity and to block forward transmission. In the successful
campaign to eradicate smallpox, which was also spread by the
respiratory route like COVID-19, the ‘take’ was used as a biomarker
for protective immunity. We believe the absence of detectable CoV-2
in the upper airways shows the potential for TNX-1800 to decrease
shedding of virus and is consistent with decreased forward
transmission.”
Dr. Lederman added, “The reliable durability of
vaccine protection from the OWS vaccines is under review1, leading
the makers of the two mRNA vaccines to take steps towards seeking
approval of booster shots and setting up the possible need for
boosters on a regular basis in the future. The U.S. government has
pledged to make available booster shots for the mRNA vaccines
available starting on September 20, pending FDA approval. The
prospect of boosters poses a challenging and expensive public
health policy implementation in the U.S. In contrast to the short
duration of protection of the OWS COVID-19 vaccines, live virus
vaccines typically provide decades or life-long protective immunity
against those diseases. Live virus vaccines activate the immune
system in ways that scientists do not completely understand and
have not yet been able to recreate with other technologies.”
About TNX-18001TNX-1800 is a
live modified horsepox virus vaccine for percutaneous
administration that is designed to express the Spike protein of the
SARS-CoV-2 virus and to elicit a predominant T cell response.
TNX-1800 is based on a horsepox vector, which is a live
replicating, attenuated virus that elicits a strong immune
response. Live replicating orthopoxviruses, like vaccinia or
horsepox, can be engineered to express foreign genes and have been
explored 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) readily 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. Horsepox and vaccinia are closely related
orthopoxviruses that are believed to share a common ancestor
horsepox3-7. Relative to vaccinia, horsepox has substantially
decreased virulence in mice7. Molecular analysis shows that
horsepox is closer than modern vaccinia vaccines in DNA sequence to
the vaccine discovered and disseminated by Dr. Edward Jenner, which
protected against smallpox, a respiratory-transmitted disease
caused by the orthopox virus, variola.4-7 Vaccine genome
researchers have established the contemporaneous use of horsepox
and horsepox-related viruses in the United States as smallpox
vaccines in the 1860’s, and found a remarkable degree of identity
with the circa 1860 U.S. smallpox vaccine VK05 and the 1976
Mongolian horsepox isolate called MNR-76, upon which Tonix’s
TNX-801 is based.3,8-10 Tonix’s proprietary horsepox vector is
believed to be more closely related to Jenner’s vaccinia vaccine
than modern vaccinia vaccines, which appear to have evolved by
deletions and mutations to a phenotype of larger plaque size in
tissue culture and greater virulence in mice. The small plaque size
in culture of TNX-801 appears identical to the U.S. Centers for
Disease Control publication of the natural isolate11. Tonix’s
TNX-1800 vaccine candidate is 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
179612 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. Tonix reported that immunization with a single dose of
TNX-1800 induced “takes” and neutralizing anti-SARS-CoV-2
antibodies in non-human primates. 13
1TNX-1800 is in the pre-IND stage and has not been approved for
any indication.2Joint Statement from HHS Public Health and Medical
Experts on COVID-19 Booster Shots Statement. URL:
www.cdc.gov/media/releases/2021/s0818-covid-19-booster-shots.html3Tulman
ER, et al. (2006) J Virol. 80(18):9244-58.PMID:169405364Qin et al.
J. Virol. 89:1809 (2015).5Esparza E, et al Vaccine. (2017)
35(52):7222-7230.5Schrick L et al N Engl J Med (2017);
377:1491-14927Noyce RS, et al. (2018) PLoS One.
13(1):e01884538Brinkmann A et al, Genome Biology (2020) 21:286
https://doi.org/10.1186/s13059-020-02202-09Duggan A et al. Genome
Biology (2020) 21:175
https://doi.org/10.1186/s13059-020-02079-z10Esparza J et al.
Vaccine. (2020); 38(30):4773-4779.11Trindale GS et al. Viruses
(2016) (12). pii: E328. PMID:2797339912Jenner E. “An Inquiry
Into the Causes and Effects of the Variole 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, 1798.13Tonix Press Release: “Tonix
Pharmaceuticals Reports Positive COVID-19 Vaccine Efficacy Results
in Non-Human Primates Vaccinated with TNX-1800 and Challenged with
Live SARS-CoV-2. URL:
ir.tonixpharma.com/press-releases/detail/1255/tonix-pharmaceuticals-reports-positive-covid-19-vaccine”
About Tonix Pharmaceuticals Holding
Corp.
Tonix is a clinical-stage biopharmaceutical
company focused on discovering, licensing, acquiring and developing
small molecules and biologics to treat and prevent human disease
and alleviate suffering. Tonix’s portfolio is primarily composed of
central nervous system (CNS) and immunology product candidates. The
Company’s CNS portfolio includes both small molecules and biologics
to treat pain, neurologic, psychiatric and addiction conditions.
Tonix’s lead CNS candidate, TNX-102 SL1, is in mid-Phase 3
development for the management of fibromyalgia. Tonix’s immunology
portfolio includes vaccines to prevent infectious diseases and
biologics to address immunosuppression, cancer, and autoimmune
diseases. Tonix’s lead vaccine candidate, TNX-18002, is a live
replicating vaccine based on the horsepox viral vector platform to
protect against COVID-19, primarily by eliciting a T cell response.
Tonix reported positive efficacy data from animal studies of
TNX-1800 in the first quarter of 2021. TNX-8012, live horsepox
virus vaccine for percutaneous administration, is in development to
protect against smallpox and monkeypox. TNX-35003 (sangivamycin) is
a small molecule antiviral drug for COVID-19 in the pre-IND stage
of development.
1TNX-102 SL is an investigational new drug and
has not been approved for any indication.2TNX-1800 and TNX-801 are
investigational new biologics at the pre-IND stage of development
and have not been approved for any indication.3TNX-3500 is an
investigational new drug at the pre-IND stage of development and
has not been approved for any indication.
This press release and further information about
Tonix can be found at www.tonixpharma.com.
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 Tonix'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
failure to obtain FDA clearances or approvals and noncompliance
with FDA regulations; delays and uncertainties caused by the global
COVID-19 pandemic; 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, 2020, as filed with the Securities and
Exchange Commission (the “SEC”) on March 15, 2021, and periodic
reports filed with the SEC on or after the date thereof. All
Tonix'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.
Jessica Morris (corporate)Tonix
Pharmaceuticalsinvestor.relations@tonixpharma.com(862) 904-8182
Olipriya Das, Ph.D.
(media)Russo PartnersOlipriya.Das@russopartnersllc.com
(646) 942-5588
Peter Vozzo
(investors)Westwickepeter.vozzo@westwicke.com (443)
213-0505
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