Tonix製藥MDD二期HORIZON研究首位患者入組

EX-99.01
2
ex99-01.htm
EX-99.01

 

 

Exhibit 99.01

 

 

Tonix
Pharmaceuticals Announces First Patient Enrolled in Phase 2 HORIZON Study of TNX-102 SL for the Treatment of Major Depressive Disorder
(MDD)

 

HORIZON
is a potentially pivotal, randomized, double-blind, placebo-controlled Phase 2 study evaluating TNX-102 SL (cyclobenzaprine HCl sublingual
tablets) 5.6 mg as a first-line monotherapy in adults with MDD

 

Primary
endpoint is change from baseline in the Montgomery-Åsberg Depression Rating Scale (MADRS) total score at Week 6

 

TNX-102-SL
was approved by the U.S. Food and Drug Administration (FDA) in August 2025 and launched commercially as TONMYA® for the
treatment of fibromyalgia in adults

 

BERKELEY
HEIGHTS, N.J., June 29, 2026 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (“Tonix” or
the “Company”), a fully integrated, commercial-stage biotechnology company, today announced that the first patient has been
enrolled in HORIZON, a potentially pivotal Phase 2 study evaluating TNX-102 SL 5.6 mg as a first-line monotherapy in adults with major
depressive disorder (MDD).

 

“We
are committed to extending the science of TNX-102 SL into other conditions in which disturbed sleep quality plays important roles, including
MDD,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “TNX-102 SL for MDD is designed to target
poor sleep quality. Bedtime TNX-102 SL showed signals for improving depressive symptoms and subjective sleep quality in both a prior
Phase 2 posttraumatic stress disorder study and two Phase 3 fibromyalgia studies. We look forward to evaluating TNX-102 SL in the Phase
2 HORIZON trial to learn if these promising signals will translate into benefits in MDD.”

 

Dr.
Gregory Sullivan, M.D., Chief Medical Officer of Tonix Pharmaceuticals added, “More than 21 million U.S. adults1 experience
a major depressive episode each year, and many current therapies are associated with tolerability and adherence challenges. TNX-102 SL
for MDD is designed to target disturbed sleep through potent antagonism at four neuronal receptors that influence sleep quality. The
quality of sleep, particularly deep slow wave sleep, is a critical factor in the nightly homeostatic neuro-restoration critical for recovery
from stress-associated conditions such as MDD.”

 

HORIZON
is a 6-week, randomized, double-blind, placebo-controlled Phase 2 study evaluating TNX-102 SL 5.6 mg as a first-line monotherapy in adults
with MDD. Approximately 360 patients are expected to enroll at approximately 30 sites across the United States. Eligible participants
are 18 years of age or older and currently experiencing a moderate to severe major depressive episode. Participants will receive TNX-102
SL 5.6 mg taken sublingually at bedtime or matching placebo. The primary endpoint of the study is the change from baseline in MADRS total
score at Week 6. Secondary endpoints include global impression scores, anxiety ratings, and measures of sleep quality and disturbance.

 

  

  

 

 

 

About
Major Depressive Disorder (MDD)

 

MDD
is a prevalent and serious psychiatric illness that affects adults of all ages, races, and backgrounds. It is characterized by persistent
feelings of sadness or loss of interest, along with symptoms such as sleep and appetite disturbances, fatigue, difficulty concentrating,
and thoughts of worthlessness or suicide. These symptoms must last at least two weeks and significantly impair daily functioning. In
the United States, more than 21 million adults experience a major depressive episode each year. While several antidepressant medications
are available, many individuals do not achieve adequate relief or discontinue treatment due to side effects such as weight gain, sleep
disruption, cognitive impairment, and sexual dysfunction. MDD is associated with an increased risk of suicide and substantial impairment
in quality of life, underscoring the urgent need for new, first-line therapies that are demonstrated to be both effective and well tolerated.

 

About
TNX-102 SL

 

TNX-102
SL is a novel sublingual tablet formulation of cyclobenzaprine hydrochloride that enables rapid transmucosal absorption and reduces production
of the persistent active metabolite, norcyclobenzaprine, by bypassing first-pass hepatic metabolism. TNX-102 SL is a tertiary amine tricyclic
(TAT) and multifunctional agent with potent binding and antagonist activities at the 5-HT2A serotonergic, α1-adrenergic,
H1-histaminergic, and M1-muscarinic receptors. It is currently FDA approved in the U.S. as a once-daily bedtime
treatment for fibromyalgia in adults under the brand name TONMYA® (cyclobenzaprine HCl sublingual tablets). TNX-102 SL
is also in development as a daily bedtime treatment for acute stress disorder (ASD)/acute stress reaction (ASR) under an investigator-initiated
IND. In addition to MDD, Tonix also holds active INDs for the following indications for TNX-102 SL: Long COVID (post-acute sequelae of
COVID-19), PTSD, alcohol use disorder, and agitation in Alzheimer’s disease. The United States Patent and Trademark Office issued
United States Patent No. 9636408 in May 2017, Patent No. 9956188 in May 2018, Patent No. 10117936 in November 2018, Patent No. 10357465
in July 2019, and Patent No. 10736859 in August 2020. The Protectic™ protective eutectic and Angstro-Technology™ formulation
claimed in the patents are important elements of Tonix’s proprietary composition. These patents are expected to provide TNX-102
SL U.S. market exclusivity until 2034. Pending patent applications related to method of use could extend exclusivity until 2044. The
potential use of TNX-102 SL in MDD, ASD/ASR, and other unapproved indications is currently under clinical development, and the safety
and efficacy have not been evaluated by any regulatory authority.

 

Citations

 

1Substance
Abuse and Mental Health Services Administration (SAMHSA). 2024. Key Substance Use and Mental Health Indicators in the United States:
Results from the 2024 National Survey on Drug Use and Health.

 

Tonix
Pharmaceuticals Holding Corp.

 

Tonix
Pharmaceuticals* is a fully integrated, commercial-stage biotechnology company focused on central nervous system (CNS) disorders, infectious
diseases, immunology conditions, and rare diseases where there exists high unmet medical need. TONMYA® (cyclobenzaprine
HCl sublingual tablets 2.8mg), the Company’s flagship internally conceived and developed medicine, is the first new treatment for
fibromyalgia in more than 15 years. Tonix’s CNS commercial infrastructure supports its marketed products, including its acute migraine
products, Zembrace® SymTouch® (sumatriptan injection 3 mg) and Tosymra® (sumatriptan nasal
spray 10 mg). Tonix is extending the science behind TONMYA in Phase 2 clinical studies to evaluate its potential in major depressive
disorder and acute stress disorder/acute stress reaction. Tonix is also advancing a pipeline of infectious disease programs, including
monoclonal antibody TNX-4800 (anti-OspA mAb) for Lyme disease prevention in the U.S. and TNX-801 (horsepox, live virus vaccine), a vaccine
in development for the prevention of mpox and smallpox. Within immunology, Tonix is developing TNX-1500 (anti-CD40L mAb), a third-generation
CD40 ligand inhibitor for the prevention of kidney transplant rejection. Finally, the Company’s rare disease portfolio includes
TNX-2900, which is Phase 2 ready for the treatment of Prader-Willi syndrome. To learn more, visit www.tonixpharma.com.

 

  

  

 

 

 

*Tonix’s
product development candidates, including TONMYA for unapproved indications, are investigational new drugs or biologics. Their efficacy
and safety have not been established and have not been approved for any indication.

 

Zembrace
SymTouch and Tosymra are registered trademarks of Tonix Medicines. TONMYA is a registered trademark of Tonix Pharma Limited. All other
marks are property of their respective owners.

 

Forward
Looking Statements

 

Certain
statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995 including
those relating to the completion of the offering, the satisfaction of customary closing conditions, the intended use of proceeds from
the offering and other statements that are predictive in nature. These statements may be identified by the use of forward-looking words
such as “anticipate,” “believe,” “forecast,” “estimate,” “expect,” and “intend,”
among others. 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 in the Company’s Annual Report on Form 10-K
for the year ended December 31, 2025, as filed with the SEC on March 12, 2026, and periodic reports filed with the SEC on or after the
date thereof. Tonix does not undertake an obligation to update or revise any forward-looking statement. 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.

 

Contacts

 

Deborah
Elson (Investors/Media)

Tonix
Pharmaceuticals

[email protected]

[email protected]

 

Brian
Korb (Investors)

astr
partners

(917)
653-5122

[email protected]

 

Ray
Jordan (Media)

Putnam
Insights

[email protected]

 

  

  

 

 

 

INDICATION

 

TONMYA
is indicated for the treatment of fibromyalgia in adults.

 

CONTRAINDICATIONS

 

TONMYA
is contraindicated:

 

In
patients with hypersensitivity to cyclobenzaprine or any inactive ingredient in TONMYA. Hypersensitivity reactions may manifest as an
anaphylactic reaction, urticaria, facial and/or tongue swelling, or pruritus. Discontinue TONMYA if a hypersensitivity reaction is suspected.

 

With
concomitant use of monoamine oxidase (MAO) inhibitors or within 14 days after discontinuation of an MAO inhibitor. Hyperpyretic crisis
seizures and deaths have occurred in patients who received cyclobenzaprine (or structurally similar tricyclic antidepressants) concomitantly
with MAO inhibitors drugs.

 

During
the acute recovery phase of myocardial infarction, and in patients with arrhythmias, heart block or conduction disturbances, or congestive
heart failure.

 

In
patients with hyperthyroidism.

 

WARNINGS
AND PRECAUTIONS

 

Embryofetal
toxicity: Based on animal data, TONMYA may cause neural tube defects when used two weeks prior to conception and during the first trimester
of pregnancy. Advise females of reproductive potential of the potential risk and to use effective contraception during treatment and
for two weeks after the final dose. Perform a pregnancy test prior to initiation of treatment with TONMYA to exclude use of TONMYA during
the first trimester of pregnancy.

 

Serotonin
syndrome: Concomitant use of TONMYA with selective serotonin reuptake inhibitors (SSRIs), serotonin norepinephrine reuptake inhibitors
(SNRIs), tricyclic antidepressants, tramadol, bupropion, meperidine, verapamil, or MAO inhibitors increases the risk of serotonin syndrome,
a potentially life-threatening condition. Serotonin syndrome symptoms may include mental status changes, autonomic instability, neuromuscular
abnormalities, and/or gastrointestinal symptoms. Treatment with TONMYA and any concomitant serotonergic agent should be discontinued
immediately if serotonin syndrome symptoms occur and supportive symptomatic treatment should be initiated. If concomitant treatment with
TONMYA and other serotonergic drugs is clinically warranted, careful observation is advised, particularly during treatment initiation
or dosage increases.

 

Tricyclic
antidepressant-like adverse reactions: Cyclobenzaprine is structurally related to TCAs. TCAs have been reported to produce arrhythmias,
sinus tachycardia, prolongation of the conduction time leading to myocardial infarction and stroke. If clinically significant central
nervous system (CNS) symptoms develop, consider discontinuation of TONMYA. Caution should be used when TCAs are given to patients with
a history of seizure disorder, because TCAs may lower the seizure threshold. Patients with a history of seizures should be monitored
during TCA use to identify recurrence of seizures or an increase in the frequency of seizures.

 

  

  

 

 

 

Atropine-like
effects: Use with caution in patients with a history of urinary retention, angle-closure glaucoma, increased intraocular pressure, and
in patients taking anticholinergic drugs.

 

CNS
depression and risk of operating a motor vehicle or hazardous machinery: TONMYA monotherapy may cause CNS depression. Concomitant use
of TONMYA with alcohol, barbiturates, or other CNS depressants may increase the risk of CNS depression. Advise patients not to operate
a motor vehicle or dangerous machinery until they are reasonably certain that TONMYA therapy will not adversely affect their ability
to engage in such activities.

 

Oral
mucosal adverse reactions: In clinical studies with TONMYA, oral mucosal adverse reactions occurred more frequently in patients treated
with TONMYA compared to placebo. Advise patients to moisten the mouth with sips of water before administration of TONMYA to reduce the
risk of oral sensory changes (hypoesthesia). Consider discontinuation of TONMYA if severe reactions occur.

 

ADVERSE
REACTIONS

 

The
most common adverse reactions (incidence ≥2% and at a higher incidence in TONMYA-treated patients compared to placebo-treated patients)
were oral hypoesthesia, oral discomfort, abnormal product taste, somnolence, oral paresthesia, oral pain, fatigue, dry mouth, and aphthous
ulcer.

 

DRUG
INTERACTIONS

 

MAO
inhibitors: Life-threatening interactions may occur.

 

Other
serotonergic drugs: Serotonin syndrome has been reported.

 

CNS
depressants: CNS depressant effects of alcohol, barbiturates, and other CNS depressants may be enhanced.

 

Tramadol:
Seizure risk may be enhanced.

 

Guanethidine
or other similar acting drugs: The antihypertensive action of these drugs may be blocked.

 

USE
IN SPECIFIC POPULATIONS

 

Pregnancy:
Based on animal data, TONMYA may cause fetal harm when administered to a pregnant woman. The limited amount of available observational
data on oral cyclobenzaprine use in pregnancy is of insufficient quality to inform a TONMYA-associated risk of major birth defects, miscarriage,
or adverse maternal or fetal outcomes. Advise pregnant women about the potential risk to the fetus with maternal exposure to TONMYA and
to avoid use of TONMYA two weeks prior to conception and through the first trimester of pregnancy. Report pregnancies to the Tonix Medicines,
Inc., adverse-event reporting line at 1-888-869-7633 (1-888-TNXPMED).

 

  

  

 

 

 

Lactation:
A small number of published cases report the transfer of cyclobenzaprine into human milk in low amounts, but these data cannot be confirmed.
There are no data on the effects of cyclobenzaprine on a breastfed infant, or the effects on milk production. The developmental and health
benefits of breastfeeding should be considered along with the mother’s clinical need for TONMYA and any potential adverse effects
on the breastfed child from TONMYA or from the underlying maternal condition.

 

Pediatric
use: The safety and effectiveness of TONMYA have not been established.

 

Geriatric
patients: Of the total number of TONMYA-treated patients in the clinical trials in adult patients with fibromyalgia, none were 65 years
of age and older. Clinical trials of TONMYA did not include sufficient numbers of patients 65 years of age and older to determine whether
they respond differently from younger adult patients.

 

Hepatic
impairment: The recommended dosage of TONMYA in patients with mild hepatic impairment (HI) (Child Pugh A) is 2.8 mg once daily at bedtime,
lower than the recommended dosage in patients with normal hepatic function. The use of TONMYA is not recommended in patients with moderate
HI (Child Pugh B) or severe HI (Child Pugh C). Cyclobenzaprine exposure (AUC) was increased in patients with mild HI and moderate HI
compared to subjects with normal hepatic function, which may increase the risk of TONMYA-associated adverse reactions.

 

Please
see additional safety information in the full Prescribing Information.

 

To
report suspected adverse reactions, contact Tonix Medicines, Inc. at 1-888-869-7633, or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.