Full Press Release Details
Tonix Pharmaceuticals Holding Corp. 8-K
Pharmaceuticals Completes Enrollment in Potentially NDA-Enabling Phase 3 RESILIENT Trial of TNX-102 SL for Management of Fibromyalgia
Data Expected Fourth Quarter 2023
Trial Required for Submission of a New Drug Application, if Successful
N.J., August 1, 2023 - Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a biopharmaceutical company,
today announced that it has completed enrollment of its potentially final, confirmatory Phase 3 RESILIENT trial of TNX-102 SL (cyclobenzaprine
HCL sublingual tablets) 5.6 mg in fibromyalgia and expects topline data next quarter. A total of 457 participants were randomized. TNX-102
SL is in development as a non-opioid, centrally acting analgesic, to be taken daily at bedtime for the management of fibromyalgia. If
successful, we believe this will be the final, well-controlled efficacy trial required for submission of a New Drug Application (NDA)
for approval by the U.S. Food and Drug Administration (FDA).
completion of enrollment in our Phase 3 RESILIENT trial is a significant milestone for both Tonix and the fibromyalgia community,"
said Seth Lederman, M.D., Chief Executive Officer of Tonix. "Currently-approved treatments have not fully met the needs of fibromyalgia
patients and there has not been a new FDA-approved therapy for the condition since 2009. TNX-102 SL has the potential to be a new non-addictive,
non-opioid bedtime medication with broad spectrum symptom coverage and which can be used on a chronic basis for the management of fibromyalgia.
With all other clinical, nonclinical and CMC requirements for an NDA submission achieved, we are looking forward to the upcoming data
readout and an expeditious filing of an NDA."
December 2020, Tonix reported positive results from the first Phase 3 study (RELIEF) of TNX-102 SL 5.6 mg for the management of fibromyalgia.
TNX-102 SL met its pre-specified primary endpoint in the Phase 3 RELIEF trial, significantly reducing daily pain compared to placebo
(p=0.01) in participants with fibromyalgia. Also, when the primary endpoint was analyzed as a 30% pain responder analysis, there
was a higher rate of responders to TNX-102 SL (47%) than to placebo (35%; p=0.006). TNX-102 SL at 5.6 mg also showed activity in key
secondary endpoints demonstrating improvements in sleep quality, mitigation of fatigue, and fibromyalgia-specific global symptomatic
and functional recovery. TNX-102 SL was generally safe and well tolerated in patients with fibromyalgia, with overall adverse event profile
comparable to prior fibromyalgia studies. The most common treatment-emergent adverse events were oral hypoesthesia, oral paresthesia,
and product taste abnormal.
the Phase 3 RESILIENT Study
RESILIENT study is a double-blind, randomized, placebo-controlled trial designed to evaluate the efficacy and safety of TNX-102 SL (cyclobenzaprine
HCl sublingual tablets) in the management of fibromyalgia. The two-arm trial randomized 457 participants in the U.S. The first two weeks
of treatment consist of a run-in period in which participants start on TNX-102 SL 2.8 mg (1 tablet) or placebo. Thereafter, all participants
increase their dose to TNX-102 SL 5.6 mg (2 x 2.8 mg tablets) or two placebo tablets for the remaining 12 weeks. The primary endpoint
is the daily diary pain severity score change (TNX-102 SL 5.6 mg vs. placebo) from baseline to Week 14 (using the weekly averages of
the daily numerical rating scale scores), analyzed by mixed model repeated measures with multiple imputation.
more information, see ClinicalTrials.gov Identifier: NCT05273749
is a chronic pain disorder that is understood to result from amplified sensory and pain signaling within the central nervous system.
Fibromyalgia afflicts an estimated 6-12 million adults in the U.S., approximately 90% of whom are women. Symptoms of fibromyalgia include
chronic widespread pain, nonrestorative sleep, fatigue, and morning stiffness. Other associated symptoms include cognitive dysfunction
and mood disturbances, including anxiety and depression. Individuals suffering from fibromyalgia struggle with their daily activities,
have impaired quality of life, and frequently are disabled. Physicians and patients report common dissatisfaction with currently marketed
SL is a patented sublingual tablet formulation of cyclobenzaprine hydrochloride which provides rapid transmucosal absorption and reduced
production of a long half-life active metabolite, norcyclobenzaprine, due to bypass of first-pass hepatic metabolism. As a multifunctional
agent with potent binding and antagonist activities at the 5-HT2A-serotonergic, 1-adrenergic, H1-histaminergic, and M1-muscarinic
receptors, TNX-102 SL is in development as a daily bedtime treatment for fibromyalgia, Long COVID (formally known as post-acute sequelae
of COVID-19 [PASC]), alcohol use disorder and agitation in Alzheimer's disease. The United States Patent and Trademark Office (USPTO)
issued United States Patent No. 9636408 in May 2017, Patent No. 9956188 in May 2018, Patent No. 10117936 in November 2018, Patent No.
10,357,465 in July 2019, and Patent No. 10736859 in August 2020. The Protectic protective eutectic and Angstro-Technology
formulation claimed in the patent are important elements of Tonix's proprietary TNX-102 SL composition. These patents are expected
to provide TNX-102 SL, upon NDA approval, with U.S. market exclusivity until 2034/2035.
Pharmaceuticals Holding Corp.*
is a biopharmaceutical company focused on commercializing, developing, discovering and licensing therapeutics to treat and prevent human
disease and alleviate suffering. Tonix markets Zembrace SymTouch (sumatriptan injection) 3 mg and Tosymra (sumatriptan
nasal spray) 10 mg. 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 CNS candidate, TNX-102 SL (cyclobenzaprine HCl sublingual tablet), is in mid-Phase 3 development
for the management of fibromyalgia, having completed enrollment in a potentially registration-enabling study, with topline data expected
in the fourth quarter of 2023. TNX-102 SL is also being developed to treat Long COVID, a chronic post-acute COVID-19 condition. Enrollment
in a Phase 2 study has been completed, and topline results are expected in the third quarter of 2023. TNX-601 ER (tianeptine hemioxalate
extended-release tablets), a once-daily formulation being developed as a treatment for major depressive disorder (MDD). Enrollment has
now completed in the UPLIFT trial of TNX-601 ER in MDD, with topline results expected in the fourth quarter of 2023. TNX-4300 (estianeptine)
is a small molecule oral therapeutic in preclinical development to treat MDD, Alzheimer's disease and Parkinson's disease.
TNX-1900 (intranasal potentiated oxytocin), in development for chronic migraine, has completed enrollment with topline data expected
in the fourth quarter of 2023. 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 third 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 is expected to be 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. The infectious disease development portfolio also includes TNX-3900 and TNX-4000, classes of broad-spectrum
small molecule oral antivirals.
product development candidates are investigational new drugs or biologics and have not been approved for any indication.
Medicines has contracted to acquire the Zembrace SymTouch and Tosymra registered trademarks. Intravail is a registered trademark of Aegis
Therapeutics, LLC, a wholly owned subsidiary of Neurelis, Inc.
press release and further information about Tonix can be found at www.tonixpharma.com.
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.
SymTouch (sumatriptan Injection): IMPORTANT SAFETY INFORMATION
SymTouch (Zembrace) can cause serious side effects, including heart attack and other heart problems, which may lead to death. Stop use
and get emergency help if you have any signs of a heart attack:
is not for people with risk factors for heart disease (high blood pressure or cholesterol, smoking, overweight, diabetes, family history
of heart disease) unless a heart exam shows no problem.
not use Zembrace if you have:
your provider about all of your medical conditions and medicines you take, including vitamins and supplements.
can cause dizziness, weakness, or drowsiness. If so, do not drive a car, use machinery, or do anything where you need to be alert.
may cause serious side effects including: