Tonix Pharmaceuticals Announces Outcome of Interim Analysis of Phase 3 RALLY Study of TNX-102 SL for the Management of Fibromyalgia
24 July 2021 - 6:40AM
Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the
Company), a clinical-stage biopharmaceutical company, announced
today that the Company has decided to stop enrollment in the Phase
3 RALLY study of TNX-102 SL (cyclobenzaprine HCl sublingual
tablets) 5.6 mg for the management of fibromyalgia following an
unblinded, pre-planned interim analysis by the Independent Data
Monitoring Committee (IDMC) of the RALLY study. Based on interim
analysis results of the first 50% (n=337) enrolled participants,
the IDMC recommended stopping the trial for futility as TNX-102 SL
is unlikely to demonstrate a statistically significant improvement
in the primary endpoint of overall change from baseline in daily
diary pain severity scores between those treated with TNX-102 SL
5.6 mg (2x 2.8 mg tablets) and those receiving placebo. Tonix
remains blinded to the detailed interim analysis results and only
received the recommendation made by the IDMC. Preliminary blinded
safety data from these participants did not reveal any new safety
signals, and the decision to discontinue enrolling new participants
is not related to safety. The Company intends to continue studying
those participants currently enrolled until completion and then
proceed with a full analysis of the unblinded data, with the
topline results expected to be reported in the fourth quarter of
2021, to determine the next steps in this program.
“We are surprised and disappointed that the
interim analysis did not support continued enrollment in this Phase
3 RALLY study, especially considering the previous Phase 3 RELIEF
study, which had a similar design and achieved statistical
significance on the primary endpoint. After the currently enrolled
participants complete the study, we will proceed with a full
analysis of the unblinded data from the study to determine the next
steps in this program,” commented Seth Lederman, M.D., President
and Chief Executive Officer. “These results underscore the
difficulty in managing and treating fibromyalgia. We thank the
patients, caregivers and investigators who participated in the
RALLY study.”
About the Phase 3 RALLY
Study
The RALLY study is a double-blind, randomized,
placebo-controlled trial designed to evaluate the efficacy and
safety of TNX-102 SL (cyclobenzaprine HCl sublingual tablets). The
two-arm trial has enrolled 514 participants across approximately 40
U.S. sites. For the first two weeks of treatment, there is a run-in
period in which participants start on TNX-102 SL 2.8 mg (1 tablet)
or placebo. After the first two weeks, all participants have the
dose increased to TNX-102 SL 5.6 mg (2 x 2.8 mg tablets) or two
placebo tablets for 12 weeks. The primary endpoint is daily diary
pain severity score change (TNX-102 SL 5.6 mg vs. placebo) from
baseline (using the weekly averages of the daily numerical rating
scale scores), analyzed by mixed model repeated measures with
multiple imputation.
Additional details about the RALLY study are available at
clinicaltrials.gov (NCT04508621).
About Fibromyalgia
Fibromyalgia 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 products.
About TNX-102 SL
TNX-102 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 serotonin-2A, alpha-1 adrenergic,
histamine-H1, and muscarinic-M1 receptors, TNX-102 SL is in
clinical development as a daily bedtime treatment for fibromyalgia,
PTSD, alcohol use disorder and agitation in Alzheimer’s disease.
The U.S. Patent and Trademark Office (USPTO) has 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 these patents 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.
About Tonix Pharmaceuticals Holding
Corp.
Tonix is a clinical-stage biopharmaceutical
company focused on discovering, licensing, acquiring and developing
small molecules and biologics to treat and prevent human disease
and alleviate suffering. Tonix’s portfolio is primarily composed of
central nervous system (CNS) and immunology product candidates. The
Company’s CNS portfolio includes both small molecules and biologics
to treat pain, neurologic, psychiatric and addiction conditions.
Tonix’s lead CNS candidate, TNX-102 SL1, is in mid-Phase 3
development for the management of fibromyalgia. Tonix’s immunology
portfolio includes vaccines to prevent infectious diseases and
biologics to address immunosuppression, cancer, and autoimmune
diseases. Tonix’s lead vaccine candidate, TNX-18002, is a live
replicating vaccine based on the horsepox viral vector platform to
protect against COVID-19, primarily by eliciting a T cell response.
Tonix reported positive efficacy data from animal studies of
TNX-1800 in the first quarter of 2021. TNX-8012, live horsepox
virus vaccine for percutaneous administration, is in development to
protect against smallpox and monkeypox.
1TNX-102 SL is an investigational new drug and
has not been approved for any indication.2TNX-1800 and TNX-801
are investigational new biologics and have not been approved for
any indication.
This press release and further information about
Tonix can be found at www.tonixpharma.com.
Forward Looking Statements
Certain 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 clinical trials, risks related to failure to obtain FDA
clearances or approvals and noncompliance with FDA regulations;
delays and uncertainties caused by the global COVID-19 pandemic;
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, 2020, as filed with the Securities and Exchange
Commission (the “SEC”) on March 15, 2021, and periodic reports
filed with the SEC on or after the date thereof. All 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.
Jessica Morris (corporate)Tonix
Pharmaceuticalsinvestor.relations@tonixpharma.com(862) 904-8182
Olipriya Das, Ph.D. (media)Russo
PartnersOlipriya.Das@russopartnersllc.com(646) 942-5588
Peter Vozzo (investors)Westwicke/ICRpeter.vozzo@westwicke.com
(443) 213-0505
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