Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP), a
biopharmaceutical company with marketed products and a pipeline of
development candidates, today announced the completion of the
clinical phase of the Phase 2 proof-of-concept, double-blind,
randomized, placebo-controlled PREVENTION1 study of TNX-19002
(intranasal potentiated oxytocin) as a potential treatment for the
prevention of migraine headaches in chronic migraineurs. A total of
88 patients were enrolled in this multi-site study in the U.S.
Topline results are expected in early December 2023.
TNX-1900 is a proprietary formulation of
oxytocin that contains magnesium, which Tonix has shown in animal
models potentiates the action of oxytocin at oxytocin receptors and
potentially improves the consistency of treatment by reducing
paradoxical high-dose inhibition. Its unique, multimodal mechanism
of action includes an ability to inhibit the release of the
trigeminal neuropeptide calcitonin gene-related peptide (CGRP) in
blood vessels within the brain, its lining and the brainstem, and
to suppress signaling in pain neurons. Tonix believes that by
engaging and activating oxytocin receptors in the trigeminal
ganglia, TNX-1900 has the potential to help those suffering from
chronic migraine.
“TNX-1900 is being developed as a novel,
non-CGRP antagonist approach to treatment for chronic migraineurs,”
said Seth Lederman, M.D., Chief Executive Officer of Tonix
Pharmaceuticals. “There are an estimated four million individuals
in the U.S. suffering from this debilitating condition, and despite
several classes of migraine preventives available, there remains an
unmet need for novel approaches.”
“With the last patient now treated, we look
forward to analysis of the results of this proof-of-concept study,
which will help to inform our plans as we discuss next steps with
the U.S. Food and Drug Administration (FDA),” said Gregory
Sullivan, M.D., Chief Medical Officer of Tonix Pharmaceuticals. “We
would like to thank the participants, their families, and all the
investigators and researchers who have been an important part of
this journey so far.”
1Clinical Trials.gov I.D. NCT056799082TNX-1900 is
an investigational new drug and is not approved for any
indication.
About the Phase 2 PREVENTION
StudyThe Phase 2 PREVENTION study is a double-blind,
randomized, multicenter, placebo-controlled study to evaluate the
efficacy and safety of TNX-1900 taken prophylactically on a daily
basis to prevent chronic migraine. There are three arms: two
treatment regimens of TNX-1900 and one placebo in a 1:1:1 ratio in
a total of 88 participants across approximately 26 U.S. sites.
After a four-week Run-In phase to confirm chronic migraine criteria
are met, there are 12-weeks of a double-blind Treatment phase with
study drug, followed by a 2-week safety Follow-Up phase. The
primary efficacy endpoint is mean change in the number of migraine
headache days between the 28-day Run-In phase and the last 28-days
of the Treatment phase. Key secondary efficacy endpoints include
proportion of patients experiencing a ≥ 50% reduction in the number
of migraine headache days, mean change in the number of days using
migraine abortive medications, and mean change in a
migraine-specific quality of life measure.
For more information, see ClinicalTrials.gov
Identifier: NCT05679908.
About MigraineMigraine is a
neurological condition that typically manifests in a throbbing
moderate to severe headache which lasts at least four hours, often
on one side of the head and aggravated by routine physical
activity. It can also be accompanied by nausea, vomiting, visual
disturbances, and sensitivity to bright light and loud noises1.
Epidemiological studies indicate that globally, approximately 1.2
billion individuals suffer from migraines annually2. In the
U.S., approximately 39 million Americans suffer from migraines and
among these individuals, approximately four million experience
chronic migraines (15 or more headache days per month, at least
eight of which are migraines)2. The current FDA approved drugs
for migraine prevention in chronic migraine include
Botox® (onabotulinumtoxinA), and the anti-CGRP/CGRP-R
monoclonal antibodies Aimovig® (erenumab),
Vyepti® (eptinezumab), Ajovy® (fremanezumab) and
Emgality® (galcanezumab).
1The International Classification of Headache
Disorders, 3rd Edition. Cephalalgia. 2018. 38(1):1-211.
2Burch et al., Migraine: Epidemiology, Burden,
and Comorbidity. Neurol Clin. 2019. 631–649.
About TNX-1900TNX-1900
(intranasal potentiated oxytocin) is a proprietary formulation of
oxytocin in development as a candidate for prevention in chronic
migraine and other conditions. In 2020, TNX-1900 was acquired from
Trigemina, Inc. who had licensed the technology underlying the
composition and method from Stanford University. TNX-1900 is a
drug-device combination product, based on an intranasal actuator
device that delivers oxytocin into the nasal cavity. Oxytocin is a
naturally occurring human peptide hormone that also acts as a
neurotransmitter in the brain. Oxytocin has no recognized addiction
potential. It has been observed that low oxytocin levels in the
body are associated with increases in migraine headache frequency,
and that increased oxytocin levels are associated with fewer
migraine headaches. Certain other chronic pain conditions are also
associated with decreased oxytocin levels. Migraine attacks are
caused, in part, by the activity of pain-sensing trigeminal neurons
which, when activated, release of calcitonin gene-related peptide
(CGRP) which binds to receptors on other nerve cells and starts a
cascade of events that is believed to result in headache. Oxytocin,
when delivered via the nasal route, concentrates in the trigeminal
system1 resulting in binding of oxytocin to receptors on
neurons in the trigeminal system, inhibiting the release of CGRP
and transmission of pain signals returning from the site of CGRP
release2. Blocking CGRP release is a distinct mechanism
compared with CGRP antagonist and anti-CGRP antibody drugs, which
block the binding of CGRP to its receptor. With TNX-1900, the
addition of magnesium to the oxytocin formulation enhances oxytocin
receptor binding3 as well as its inhibitory effects on
trigeminal neurons and resultant craniofacial analgesic effects, as
demonstrated in animal models4. Intranasal oxytocin has been shown
to be well tolerated in several clinical trials in both adults and
children5. Targeted nasal delivery results in low systemic exposure
and lower risk of non-nervous system, off-target effects, which
could potentially occur with systemic CGRP antagonists such as
anti-CGRP antibodies6. For example, CGRP has roles in dilating
blood vessels in response to ischemia, including in the heart. The
Company believes nasally targeted delivery of oxytocin could
translate into selective blockade of CGRP release from neurons in
the trigeminal ganglion and not throughout the body, which could be
a potential safety advantage over systemic CGRP inhibition. In
addition, daily dosing is more rapidly reversible, in contrast to
monthly or quarterly dosing, as is the case with anti-CGRP
antibodies, giving physicians and their patients greater control.
In addition to chronic migraine, TNX-1900 will be developed for
treatment of episodic migraine, binge eating disorder, craniofacial
pain conditions, and insulin resistance. Tonix also has a license
with the University of Geneva to use TNX-1900 for the treatment of
insulin resistance and related conditions.
1Yeomans DC, et al. Transl Psychiatry. 2021.
11(1):388.
2Tzabazis A, et al. Cephalalgia. 2016.
36(10):943-50.
3Antoni FA and Chadio SE. Biochem J. 1989.
257(2):611-4.
4Cai Q, et al., Psychiatry Clin Neurosci. 2018.
72(3):140-151.
5Yeomans, DC et al. 2017. US patent
US2017368095.
6MaassenVanDenBrink A, et al. Trends Pharmacol
Sci. 2016. 37(9):779-788.
Tonix Pharmaceuticals Holding
Corp.*Tonix is a biopharmaceutical
company focused on commercializing, developing, discovering and
licensing therapeutics to treat and prevent human disease and
alleviate suffering. Tonix Medicines, our commercial subsidiary,
markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and
Tosymra® (sumatriptan nasal spray) 10 mg under a transition
services agreement with Upsher-Smith Laboratories, LLC from whom
the products were acquired on June 30, 2023. Zembrace SymTouch and
Tosymra are each indicated for the treatment of acute migraine with
or without aura in adults. Tonix’s development portfolio is
composed of central nervous system (CNS), rare disease, immunology
and infectious disease product candidates. Tonix’s CNS development
portfolio includes both small molecules and biologics to treat
pain, neurologic, psychiatric and addiction conditions. Tonix’s
lead development CNS candidate, TNX-102 SL (cyclobenzaprine HCl
sublingual tablet), is in mid-Phase 3 development for the
management of fibromyalgia, having completed enrollment of a
potentially confirmatory Phase 3 study in the third quarter of
2023, with topline data expected in late December 2023. TNX-102 SL
is also being developed to treat fibromyalgia-type Long COVID, a
chronic post-acute COVID-19 condition. Enrollment in a Phase 2
proof-of-concept study has been completed, and topline results were
reported in the third quarter of 2023. TNX-601 ER (tianeptine
hemioxalate extended-release tablets) is a once-daily oral
formulation being developed as a treatment for major depressive
disorder (MDD), that completed enrollment in a Phase 2 study in the
third quarter of 2023, with topline results expected in early
November of 2023. TNX-4300 (estianeptine) is a single isomer
version of TNX-601, a small molecule oral therapeutic in
preclinical development to treat MDD, Alzheimer’s disease and
Parkinson’s disease. Relative to tianeptine, estianeptine lacks
activity on the mu-opioid receptor while maintaining activity and
the ability to activate PPAR-β/δ and neuroplasticity in tissue
culture. TNX-1900 (intranasal potentiated oxytocin), is in
development as a preventive treatment in chronic migraine, and the
clinical phase of a Phase 2 proof-of-concept study is now completed
with topline data expected in early December 2023. TNX-1900 is also
being studied in binge eating disorder, pediatric obesity and
social anxiety disorder by academic collaborators under
investigator-initiated INDs. 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 fourth quarter
of 2023. Tonix’s rare disease development 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 development 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 rejection and for the
treatment of autoimmune diseases. A Phase 1 study of TNX-1500 was
initiated in the third quarter of 2023. Tonix’s infectious disease
pipeline includes TNX-801, a vaccine in development to prevent
smallpox and mpox. TNX-801 also serves as the live virus vaccine
platform or recombinant pox vaccine platform for other infectious
diseases, including TNX-1800, in development as a vaccine to
protect against COVID-19. The infectious disease development
portfolio also includes TNX-3900 and TNX-4000, which are classes of
broad-spectrum small molecule oral antivirals.
*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. Intravail is a registered trademark
of Aegis Therapeutics, LLC, a wholly owned subsidiary of Neurelis,
Inc. All other marks are property of their respective owners.
This press release and further information about
Tonix can be found at www.tonixpharma.com.
Forward-Looking
StatementsCertain 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 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, 2022, as filed
with the Securities and Exchange Commission (the “SEC”) on March
13, 2023, and periodic reports filed with the SEC on or after the
date thereof. All of 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.
Investor ContactJessica
MorrisTonix Pharmaceuticalsinvestor.relations@tonixpharma.com(862)
904-8182
Peter VozzoICR Westwickepeter.vozzo@westwicke.com (443)
213-0505
Media ContactBen ShannonICR
Westwickeben.shannon@westwicke.com443-213-0495
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