Full Press Release Details
TONIX PHARMACEUTICALS HOLDING CORP. 8-K
Tonix Pharmaceuticals Plans to Initiate Prader-Willi
Syndrome Phase 2 Trial of TNX-2900 (Intranasal Potentiated Oxytocin) in 2026
Phase 2 randomized, double-blind, placebo-controlled
trial planned to evaluate TNX-2900 in children and adolescents (ages 8 to 17.5 years) with Prader-Willi Syndrome under a cleared IND
TNX-2900 granted Orphan Drug and Rare Pediatric
Disease Designations by the FDA, providing the potential for a Priority Review Voucher upon approval
Magnesium-potentiated
intranasal oxytocin formulation designed to reduce dose-related inconsistencies in receptor activity
CHATHAM, N.J., September 29, 2025 - Tonix Pharmaceuticals
Holding Corp. (Nasdaq: TNXP), a fully-integrated commercial-stage biotechnology company with innovative marketed products and a pipeline
of development candidates, today announced plans to progress its TNX-2900 program for the treatment of Prader-Willi syndrome (PWS) into
a Phase 2 clinical trial. TNX-2900 is a proprietary magnesium-potentiated intranasal oxytocin formulation designed to improve receptor
binding and decrease dose-related inconsistencies in receptor activity. The program has received both Orphan Drug and Rare Pediatric Disease
designations from the U.S. Food and Drug Administration (FDA), which would make Tonix eligible for a transferable Priority Review Voucher,
upon approval. The FDA has cleared the Investigational New Drug (IND) application for TNX-2900 to progress into Phase 2 development.
"We are pleased to advance TNX-2900 into a Phase
2 trial for PWS, a condition with unmet needs for new medicines with activity and tolerability," said Seth Lederman, M.D., Chief
Executive Officer of Tonix Pharmaceuticals. "Families caring for children with PWS face significant challenges and burdens. Among
them is hyperphagia which drives persistent food-seeking behaviors that require constant supervision and often result in obesity and serious
medical complications. With an average life expectancy of less than 30 years, treatment of PWS remains an urgent and unmet need. By addressing
limitations of traditional oxytocin delivery, we believe TNX-2900 has the potential to become an FDA-approved therapy targeting the oxytocin
receptor in PWS and provide meaningful benefit for patients and families living with this rare disorder."
Tonix plans to conduct a Phase 2 randomized, double-blind,
placebo-controlled, parallel-design study to evaluate the safety, tolerability, and efficacy of TNX-2900 in male and female participants
with PWS, ages 8 to 17.5 years. Eligible participants will be randomized to receive 12-weeks of treatment with TNX-2900 at one of three
dose levels, or placebo, in a 1:1:1:1 ratio. The primary efficacy endpoint will be the change from baseline in the validated Hyperphagia
Questionnaire for Clinical Trials (HQ-CT), a widely used measure of hyperphagia severity in PWS. Secondary objectives will include assessments
of behavior, caregiver burden, and quality of life measures, as well as safety and tolerability outcomes.
Prader-Willi syndrome (PWS) is a rare genetic disorder
and the leading cause of life-threatening childhood obesity, affecting about 1 in 10,000 to 1 in 30,000 births. Infants often present
with poor muscle tone and feeding difficulties, while children and adolescents develop hyperphagia, behavioral challenges, and severe
obesity and metabolic disease. Current interventions are difficult to sustain and often inadequate.
Research suggests PWS is associated with a functional
deficiency of oxytocin, a neuropeptide that regulates satiety and feeding behaviors through the oxytocin receptor. Oxytocin treatment
addresses several key features of PWS expressed in the MAGEL2 (MAGE-like 2) knock-out mouse.1 Intranasal oxytocin therapy
has shown benefits in infants with PWS.2 Carbetocin has a different spectrum of activity on oxytocin and vasopressin receptors
than oxytocin and carbetocin has not been tested to our knowledge in the MAGEL2 knock-out mouse.3 Oxytocin has dose-related
inconsistencies in receptor activity that have been described as "high-dose suppression" or an "inverted "U"
dose response.4 TNX-2900 is formulated with magnesium to further enhance oxytocin receptor binding and signaling, with the
goal of providing more consistent and selective receptor activation while minimizing off-target vasopressin effects. In vitro and
in vivo in animals Mg++- containing formulations reduce these inconsistencies.4
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, specifically related to the MAGE (melanoma antigen) gene family on the Prader-Willi
critical region (15q11-q13) on the paternally acquired chromosome. 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.5 Given the 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 correcting behavioral characteristics of the MAGEL2 knock-out mouse model for
PWS and autism.1,6,7 Six clinical trials have investigated intranasal oxytocin as a treatment in pediatric patients with PWS.
Four clinical studies showed evidence for improvement in PWS-related behaviors/symptoms/2,810 Three of these clinical studies
reported evidence for improvement in hyperphagia8-10 and one showed an improvement in sucking in infants.2
About TNX-2900 and Tonix's Potentiated Oxytocin
TNX-2900 is based on Tonix's patented intranasal
Mg2+-potentiated oxytocin formulation intended for use by children and adolescents. This formulation is believed to enhance
the potency of oxytocin as well as increase specificity for oxytocin receptors relative to vasopressin receptors, potentially reducing
unwanted side effects from activating vasopressin receptors. In collaboration with academic investigators, Tonix is also testing a different
intranasal formulation, designated TNX-1900 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. It was initially 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 and control postpartum bleeding or hemorrhage. An intranasal formulation of oxytocin is marketed in some European countries
to assist in breast milk production as Syntocinon (oxytocin nasal 40 international units/ml).
Tonix Pharmaceuticals Holding Corp.*
Tonix Pharmaceuticals is a commercial-stage, fully-integrated
biotechnology company with marketed products and a pipeline of development candidates. Tonix recently received FDA approval for TonmyaTM,
a first-in-class, non-opioid analgesic medicine for the treatment of fibromyalgia, a chronic pain condition that affects millions of adults.
This marks the first approval for a new prescription medicine for fibromyalgia in more than 15 years. Tonix also markets two treatments
for acute migraine in adults. Tonix's development portfolio is focused on central nervous system (CNS) disorders, immunology, immuno-oncology
and infectious diseases. TNX-102 SL is being developed to treat acute stress reaction and acute stress disorder under a Physician-Initiated
IND at the University of North Carolina in the OASIS study funded by the U.S. Department of Defense (DoD). Tonix's immunology development
portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is an Fc-modified
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's infectious disease portfolio includes TNX-801, a vaccine in development for mpox and
smallpox, as well as TNX-4200 for which Tonix has a contract with the U.S. DoD's Defense Threat Reduction Agency (DTRA) for up to
$34 million over five years. TNX-4200 is a small molecule broad-spectrum antiviral agent 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.
* Tonix's product development candidates are investigational
new drugs or biologics; their efficacy and safety have not been established and have not been approved for any indication.
This press release and further information about Tonix can be found
Forward Looking Statements
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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 successfully launch and commercialize Tonmya
and any of our approved products; risks related to the failure to obtain FDA clearances or approvals and noncompliance with FDA regulations;
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, 2024, as filed with the Securities and Exchange Commission (the "SEC") on March 18, 2025, 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