Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP), a
biopharmaceutical company with marketed products and a pipeline of
development candidates, today announced the U.S. Food and Drug
Administration (FDA) has cleared the Investigational New Drug (IND)
application to support clinical development of TNX-2900 (intranasal
potentiated oxytocin), a proprietary magnesium (Mg2+)-enhanced
formulation of intranasal oxytocin, to treat Prader-Willi syndrome
(PWS) in children and adolescents. TNX-2900 for the treatment of
PWS was granted Orphan Drug designation by the FDA in 2022.
The Phase 2 study approved by the IND is a
dose-finding study involving approximately 36 PWS patients divided
into four groups with approximately nine PWS patients per group.
One group will receive placebo and three groups will receive
different dosage regimens of TNX-2900. Tonix intends to seek a
partner to advance TNX-2900 for PWS in clinical development.
“We are pleased that TNX-2900 is cleared for
clinical studies for the treatment of PWS in children and
adolescents as there remains a significant need for new therapies,
particularly for PWS hyperphagia, which currently has no approved
treatments,” said Seth Lederman, M.D., Chief Executive Officer of
Tonix Pharmaceuticals. “PWS is the most common genetic cause of
life-threatening childhood obesity.3,4 We believe adding Mg2+ to
the formulation has the potential to improve intranasal oxytocin’s
therapeutic action.”
The IND application for TNX-2900 was supported
by preclinical data demonstrating that Mg2+ enhances the potency of
oxytocin. Oxytocin is a naturally-occurring hormone that reduces
appetite and eating and regulates hunger, anxiety and prosocial
behavior. PWS is a genetic disorder associated with abnormalities
of the oxytocin system5. Several previous clinical studies in PWS
of intranasal oxytocin without Mg2+-potentiation have shown trends
toward improvement, but the results have been inconsistent.1,2
Tonix believes that Mg2+-potentiation of intranasal oxytocin in PWS
may improve consistency in clinical trials because in animal
studies Mg2+-potentiation appears to eliminate the high-dose
suppression of oxytocin’s inverted “U”-shaped dose response.6
Gregory Sullivan, M.D., Chief Medical Officer of
Tonix Pharmaceuticals added, “Recent reports show Mg2+ is necessary
for oxytocin to fully activate the oxytocin receptor.3,6 Oxytocin
has potent effects in adult mice correcting behavioral
characteristics of the Magel2 knock-out mouse model for PWS and
autism.4 Oxytocin has many potential therapeutic roles in reducing
appetite, eating, weight, migraine pain and autistic spectrum
behaviors. Tonix recently completed enrollment in a Phase 2 study
of TNX-1900, a related Mg2+-potentiated intranasal oxytocin
candidate, for the prevention of migraine headaches, and is also
studying TNX-1900 through external collaborations for the treatment
of obesity in adolescents, binge eating disorder, bone health in
autism, and social anxiety disorder.”
About Prader-Willi Syndrome
(PWS)PWS is recognized as the most common genetic cause of
life-threatening childhood obesity and affects males and
females with equal frequency and all races and ethnicities. PWS
results from the absence of expression of a group of genes on the
paternally acquired chromosome 15. The hallmarks of PWS are
lack of suckling in newborns and, in children and adolescents,
severe hyperphagia, an overriding physiological drive to eat,
leading to severe obesity and other complications associated with
significant mortality. A systematic review of the morbidity and
mortality as a consequence of hyperphagia in PWS found that the
average age of death in PWS was 22.1 years.7 There is no approved
medication to treat poor feeding in newborns or hyperphagia in
children and adolescents with PWS. Given these serious or
life-threatening manifestations of these conditions, there is a
critical need for effective treatments to decrease morbidity and
mortality, improve quality of life, and increase life expectancy in
people with PWS. Oxytocin has potent effects in adult mice
correcting behavioral characteristics of the Magel2 knock-out mouse
model for PWS and autism.4 In addition, oxytocin has potent effects
in correcting behavioral characteristics of the neonatal Magel2
knock-out mouse model for PWS and autism8 and intriguing effects in
a clinical trial of neonates with PWS.9
About TNX-2900 and Tonix’s Potentiated
Oxytocin PlatformTNX-2900 is based on Tonix’s patented
intranasal potentiated oxytocin formulation intended for use by
adults and adolescents. Tonix’s patented potentiated oxytocin
formulation is believed to increase specificity for oxytocin
receptors relative to vasopressin receptors as well as to enhance
the potency of oxytocin. Tonix is also developing a different
intranasal formulation, designated TNX-1900, for prophylaxis of
chronic migraine as well as for adolescent obesity, binge eating
disorder, bone health in autism and social anxiety disorder.
Oxytocin is a naturally occurring human hormone that acts as a
neurotransmitter in the brain. Oxytocin is believed to be more than
600 million years old and is present in vertebrates including
mammals, birds, reptiles, amphibians and fish.10,11 It was
originally approved by the U.S. Food and Drug Administration as
Pitocin®*, an intravenous infusion or intramuscular injection drug,
for use in pregnant women to induce labor. An intranasal
formulation of oxytocin is marketed in some European countries to
assist in the production of breast milk as Syntocinon®** (oxytocin
nasal 40 units/ml). *Pitocin® is a trademark of Par Pharmaceutical,
Inc.
**Syntocinon® is a trademark of BGP Products
Operations GmbH
Citations
- Shalma NM, et al. Diabetes Metab
Syndr. 2023. 17(2):102711.
- Rice LJ, et al. Curr Opin
Psychiatry. 2018. 31(2):123-127.
- Meyerowitz JG, et al. Nat Struct Mol
Biol. 2022. 29(3):274-281.
- Meziane H, et al. Biol Psychiatry.
2015. 78(2):85-94.
- Correa-da-Silva F, et al. J
Neuroendocrinol. 2021. 33(7):e12994.
- Bharadwaj VN, et al. Pharmaceutics.
2022. 14(5):1105.
- Bellis SA, et
al. Eur J Med Genet. 2022. 65(1):104379.
- Bertoni A, et al. Mol Psychiatry.
2021. 26(12):7582-7595.
- Tauber M, et al. Pediatrics. 2017.
139(2):e20162976.
- Oxytocin in Wikipedia
https://en.wikipedia.org/wiki/Oxytocin (accessed 8-8-23)
- Gruber CW. Exp Physiol. 2014.
99(1):55-61. doi: 10.1113/expphysiol.2013.072561.
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 the clinical phase of
a potentially confirmatory Phase 3 study in the fourth 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, and topline results were
reported in the third quarter of 2023. TNX-1900 (intranasal
potentiated oxytocin), is in development as a preventive treatment
in chronic migraine, and enrollment has completed in a Phase 2
proof-of-concept study 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. During the fourth quarter of 2023,
TNX-1800 was selected by the U.S. National Institutes of Health
(NIH), National Institute of Allergy and Infectious Diseases
(NIAID) Project NextGen for inclusion in Phase 1 clinical trials.
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 Contact
Jessica MorrisTonix
Pharmaceuticalsinvestor.relations@tonixpharma.com (862)
904-8182
Peter VozzoICR Westwickepeter.vozzo@westwicke.com (443)
213-0505
Media Contact
Ben ShannonICR Westwickeben.shannon@westwicke.com(443)
213-0495
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