Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the
Company), a fully-integrated biopharmaceutical company with
marketed products and a pipeline of development candidates, today
announced financial results for the first quarter ended March 31,
2024, and provided an overview of recent operational highlights.
“Our near-term priority continues to be the
submission of our New Drug Application (NDA) for Tonmya™
(cyclobenzaprine HCl sublingual tablets) for the management of
fibromyalgia, while continuing to build out our commercial strategy
for the anticipated product launch in the event of FDA approval,
which we currently estimate to occur in the second half of 2025,”
said Seth Lederman, M.D., Chief Executive Officer of Tonix.
Dr. Lederman added, “The well-known
treatment-limiting side effects of the three currently approved
drugs have led to widespread patient dissatisfaction, creating what
we believe is a significant opportunity for a new therapeutic.
Tonmya has a differentiated mechanism of action and is generally
free of common side effects associated with the currently approved
products, including weight gain, fatigue, insomnia, increased blood
pressure, gastrointestinal issues or sexual dysfunction. As such,
we believe Tonmya, if approved, could become the treatment of
choice for the approximately 10 million people in the U.S.
suffering the debilitating effects of fibromyalgia.”
The Company is also advancing other key pipeline
programs including those for immunology, obesity, eating disorders,
infectious and rare diseases, many through a capital efficient
strategy involving partnerships, grants and in-kind
contributions.
Recent Highlights – Key Product
Candidates*
Central Nervous System (CNS) Pipeline
Tonmya (also known as TNX-102 SL;
cyclobenzaprine HCl sublingual tablets): a centrally-acting,
non-opioid, small molecule analgesic taken once-daily at bedtime
for the management of fibromyalgia (FM).
- In January 2024,
Tonix presented additional safety and tolerability data from the
pivotal Phase 3 RESILIENT study that showed Tonmya treatment was
not associated with increases in systolic or diastolic blood
pressure or body weight, nor were there any reported sexual side
effects. The Company had previously announced in December 2023 that
the Phase 3 RESILIENT study, a registration-quality, double-blind,
placebo-controlled study evaluating Tonyma met its pre-specified
primary endpoint in the second of two positive Phase 3 clinical
trials, significantly reducing daily pain compared to placebo
(p-value=0.00005) in participants with fibromyalgia. Statistically
significant and clinically meaningful results were also seen in all
pre-specified key secondary endpoints including those related to
improving sleep quality, reducing fatigue, and improving patient
global ratings and overall fibromyalgia symptoms and function.
Tonmya was well tolerated with an adverse event profile comparable
to prior studies and no new safety signals observed. In addition,
Tonmya therapy showed activity on improving female sexual function
relative to placebo with a nominal p-value=0.010 by the Changes in
Sexual Functioning Questionnaire short-form, female version.
- Tonix plans to
submit an NDA to the FDA in the second half of 2024 for Tonmya for
the management of fibromyalgia. In February 2024, Tonix announced
the engagement of Rho, Inc. as our contract research organization
(CRO) to support NDA submission.
- In February 2024,
Tonix announced statistically significant results from its clinical
pharmacokinetic (PK) bridging study of Tonmya in healthy adult male
and female ethnic Japanese and Chinese volunteers. Results indicate
that key PK parameters of cyclobenzaprine are comparable in ethnic
Japanese and Chinese volunteers to Caucasian volunteers from a
prior PK study. Tonmya was generally well tolerated in the ethnic
Japanese and Chinese healthy volunteers. The company expects these
data to fulfill the requirement for a bridging study, and enables
Tonix to rely on Phase 3 studies RESILIENT and RELIEF results to
support regulatory filings for clinical studies in Japan and China
where cyclobenzaprine is a new chemical entity (NCE). Tonix holds
issued patents for market exclusivity rights of Tonmya in Japan,
China, Hong Kong and Taiwan.
- In March 2024,
Tonix announced the selection of two contract manufacturing
organizations (CMOs), including Almac Pharma Services, as dual
supply sources for the potential launch and commercialization of
Tonmya in the U.S.
- In March 2024,
Tonix selected EVERSANA, a leading provider of commercialization
services to the global life sciences industry, to support the
launch strategy and commercial planning of Tonmya in the U.S.
- Tonix presented
additional efficacy data from RESILIENT at the 6th International
Congress on Controversies in Fibromyalgia in Brussels, Belgium,
March 7-8, 2024. The data showed that Tonmya treatment resulted in
an improvement in cognitive dysfunction, or ‘brain fog’, measured
by the change in the Fibromyalgia Impact Questionnaire-Revised
(FIQ-R) memory item. The FIQ-R cognitive item showed nominal
improvement in Tonmya-treated patients vs placebo-treated patients
with a nominal p-value=0.001 and effect size of 0.31.
TNX-102 SL for the treatment of acute stress
reaction (ASR) and acute stress disorder (ASD), and prophylaxis
against development of posttraumatic stress disorder (PTSD)
- In February 2024,
the Company announced the FDA cleared the Investigational New Drug
(IND) application for the Phase 2 investigator-initiated OASIS
trial to evaluate TNX-102 SL in reducing the severity of ASR and
the frequency of ASD and PTSD. The trial is sponsored by the
University of North Carolina Institute for Trauma Recovery and
supported by a $3 million grant from the U.S. Department of
Defense, which was awarded in September 2023. The proposed Phase 2,
Optimizing Acute Stress Reaction Interventions with TNX-102 SL
(OASIS) study will examine the safety and efficacy of TNX-102 SL to
reduce adverse posttraumatic neuropsychiatric sequelae among
patients presenting to the emergency department (ED) after a motor
vehicle collision. The study will enroll approximately 180 trauma
survivors at ED study sites in the U.S. Participants will be
randomized in the ED to receive a two-week course of either TNX-102
SL 5.6 mg or placebo.
- Tonix anticipates
the Phase 2 OASIS trial will initiate in the second quarter of
2024.
TNX-102 SL for the treatment of
Fibromyalgia-Type Long COVID, also known as Post-Acute Sequelae of
COVID-19 (PASC)
- In January 2024,
the Company announced the online publication of a research paper in
the Journal Pain. The article titled, “Chronic Overlapping Pain
Conditions Increase the Risk of Long COVID Features, Regardless of
Acute COVID Status,” by Bergmans, et al.1, found that patients
with pre-existing chronic overlapping pain conditions (COPCs) had
an increased risk of being diagnosed with symptoms of Long COVID1.
Faculty at the University of Michigan directed the research.
Commentary on the article titled, “A step towards better
understanding chronic overlapping pain conditions” by
Fitzcharles, et al,2 is in the same issue of the journal.
COPCs include fibromyalgia, chronic fatigue syndrome, migraine
headache, irritable bowel syndrome, endometriosis and low back
pain. These results contribute to a growing body of evidence that
common symptoms of Long COVID in many patients are at least partly
driven by central nervous system mechanisms.
TNX-1300 (recombinant double mutant cocaine
esterase): biologic for life-threatening cocaine intoxication
- Tonix expects to
initiate a Phase 2 clinical study of TNX-1300 for the treatment of
cocaine intoxication in emergency rooms in the second quarter of
2024. In 2022, Tonix was awarded a Cooperative Agreement grant from
the National Institutes of Health (NIH)’s National Institute of
Drug Abuse (NIDA) to support development of TNX-1300.
- TNX-1300 has been
granted Breakthrough Therapy designation by the FDA.
TNX-1900 (intranasal potentiated oxytocin):
small peptide in development through investigator-initiated studies
for adolescent obesity, binge eating disorder, bone health in
autism and social anxiety disorder (SAD).
- TNX-1900 continues
to be studied in four ongoing investigator-initiated Phase 2
studies as follows: Massachusetts General Hospital (MGH): (1) Phase
2 study for binge-eating disorder (BED); (2) Phase 2 study for
adolescent obesity; (3) Phase 2 study for improving bone health in
children with autism spectrum disorder (BOX); and at University of
Washington, (4) Phase 2 study for social anxiety disorder (SAD).
The BED study and the adolescent obesity study will investigate
whether TNX-1900 has effects on eating behaviors in specialized
populations.
Rare Disease Pipeline
TNX-2900 (intranasal potentiated oxytocin):
small peptide for the treatment of Prader-Willi syndrome (PWS)
- In March 2024,
Tonix announced that it received Rare Pediatric Disease designation
from the FDA for TNX-2900 for the treatment of PWS. Tonix has an
IND to support clinical development of TNX-2900 to treat PWS in
children and adolescents. The planned Phase 2 study is a
dose-finding study involving approximately 36 PWS patients divided
into four groups with approximately nine per group. One group will
receive placebo and three groups will receive different dosage
regimens of TNX-2900. TNX-2900 for the treatment of PWS was granted
Orphan Drug designation by the FDA in 2022. PWS is a genetic
disorder that affects several body systems, with cognitive and
behavioral symptoms including pathological over-eating beginning in
childhood and leading to severe metabolic sequelae.
Immunology Pipeline
TNX-1500 (anti-CD40L Fc-modified humanized
monoclonal antibody): third generation anti-CD40L monoclonal
antibody for prophylaxis of organ transplant rejection and
treatment of autoimmune disorders.
- The first proposed
indication for TNX-1500 is prophylaxis of organ rejection in adult
patients receiving a kidney transplant; but multiple additional
indications are possible, including autoimmune diseases. Two peer
reviewed publications described the work with TNX-1500 at the
Massachusetts General Hospital (MGH) on allogeneic transplants in
animals.3,4
- Preclinical
studies have shown that TNX-1500 maintains the activity of
first-generation monoclonal antibodies (mAbs), yet with reduced
risk of thrombotic complications.3-5 Modeling studies from animal
pharmacokinetic data3 predict a half-life of greater than three
weeks for TNX-1500 in humans, which supports a monthly i.v. dosing
regimen. This analysis together with TNX-1500’s activity and
tolerability in animals, suggests that the protein engineering of
TNX-1500’s Fc region has achieved its design goals.
- In February 2024,
Tonix announced the completion of the clinical stage of its Phase 1
single ascending dose study of TNX-1500 in healthy volunteers. The
primary objectives of the study are to assess the safety,
tolerability, pharmacokinetics and pharmacodynamics of intravenous
TNX-1500. This first-in-human study is intended to support dosing
in a planned Phase 2 trial in kidney transplant recipients.
- In March of 2024,
the MGH announced the first transplant of a genetically modified
pig kidney into a living patient in collaboration with eGenesis,
which produced the pig donors and used an anti-CD40L mAb from
another company.5 Some of the pre-clinical work that supported the
living human transplant was performed in collaboration with Tonix
and used TNX-1500.6 The patient was able to return home after the
transplant, but died after approximately two months.7
Marketed Products – Recent
Highlights
- As of
April 1, 2024, Tonix completed the transition to becoming a fully
integrated pharmaceutical company. Tonix Pharmaceuticals has
implemented personnel, systems and contracts required to support a
commercial organization and has assumed responsibility for
distribution, selling and marketing of Zembrace SymTouch and
Tosymra, as well as supply chain, regulatory and quality control of
the two products.
Facilities – Recent
Highlights
- In the fourth
quarter of 2023, Tonix engaged CBRE, an international real estate
brokerage firm, to potentially find a strategic partner for, or
buyer of, its Advanced Development Center (ADC) to align with the
Company’s current business objectives and priorities. At this time,
the Company does not have a commitment in place to sell the
building. ADC, located in the New Bedford business park in
Dartmouth, Massachusetts, is an approximately 45,000 square foot
BSL-2 facility intended for clinical scale manufacturing of
live-virus vaccines and biologics.
*All of Tonix’s product candidates are
investigational new drugs or biologics and none have been approved
for any indication.
Tonmya™ is conditionally accepted by the U.S.
Food and Drug Administration (FDA) as the tradename for TNX-102 SL
for the management of fibromyalgia. Tonmya has not been approved
for any indication.
1 Bergmans RS, et al. PAIN. 2023. DOI:
10.1097/j.pain.0000000000003110.
2 Fitzcharles M-A, et al. PAIN. 2023. DOI:
10.1097/j.pain.0000000000003129.
3 Lassiter G., et al. Am J Transplantation.
2023. https://doi.org/10.1016/j.ajt.2023.03.022
4 Miura S., et al. Am J Transplantation. 2023.
https://doi.org/10.1016/j.ajt.2023.03.025
5 Massachusetts General Hospital press release.
March 21, 2024. “World’s First Genetically Edited Pig Kidney
Transplant into Living Recipient Performed at Massachusetts General
Hospital.”
www.massgeneral.org/news/press-release/worlds-first-genetically-edited-pig-kidney-transplant-into-living-recipient
(accessed March 29, 2024)
6 Anand, R.P., et al Nature. 622, 393–401
(2023). https://doi.org/10.1038/s41586-023-06594-4
7 Stoico, N. Boston Globe. May 11, 2023. “Mass
Man who received first kidney transplant from genetically
engineered pig has died, family says”.
Recent Highlights –
Financial
As of March 31, 2024, Tonix had $7.0 million of
cash and cash equivalents, compared to $24.9 million as of December
31, 2023. Net cash used in operations was approximately $17.6
million for first quarter 2024, compared to net cash used in
operations of $32.9 million for the same period in 2023.
On April 1, 2024, the Company closed a financing
with existing healthcare-focused institutional investors for
upfront gross proceeds of approximately $4.4 million through a
registered direct offering.
First Quarter 2024 Financial Results
Net product revenue for the first quarter 2024
was approximately $2.5 million. Net product revenue consisted of
combined net sales of Zembrace® SymTouch® and Tosymra®, which were
acquired from Upsher-Smith Laboratories, LLC on June 30, 2023. Cost
of Sales for the first quarter 2024 was approximately $1.7
million.
Research and development expenses for the first
quarter 2024 were $12.9 million, compared to $26.5 million for the
same period in 2023. This decrease is predominantly due to
decreased clinical, non-clinical and manufacturing expenses.
General and administrative expenses for the
first quarter 2024 were $9.3 million, compared to $7.4 million for
the same period in 2023. The increase was primarily due to sales
and marketing and the transition services expenses associated with
the Company’s recently acquired marketed products offset by a
decrease in financial reporting expenses.
Net loss was $(14.9) million, or $(0.18) per
share, basic and diluted, for the first quarter 2024, compared to
net loss of $(33.0) million, or $(3.21) per share, basic and
diluted, for the same period in 2023. The basic and diluted
weighted average common shares outstanding for the first quarter
2024 was 80,879,108 compared to 10,268,500 shares for the same
period in 2023.
Tonix Pharmaceuticals Holding
Corp.*
Tonix is a fully-integrated biopharmaceutical
company focused on developing, licensing and commercializing
therapeutics to treat and prevent human disease and alleviate
suffering. Tonix’s development portfolio is focused on central
nervous system (CNS) disorders. Tonix’s priority is to submit a New
Drug Application (NDA) to the FDA in the second half of 2024 for
Tonmya1, a product candidate for which two statistically
significant Phase 3 studies have been completed for the management
of fibromyalgia. TNX-102 SL is also being developed to treat acute
stress reaction as well as fibromyalgia-type Long COVID. Tonix’s
CNS portfolio includes TNX-1300 (cocaine esterase), a biologic
designed to treat cocaine intoxication that has Breakthrough
Therapy designation. Tonix’s immunology development portfolio
consists of 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. Tonix also has product candidates
in development in the areas of rare disease and infectious disease.
Tonix Medicines, our commercial subsidiary, markets Zembrace®
SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan
nasal spray) 10 mg for the treatment of acute migraine with or
without aura in adults.
*Tonix’s product development candidates are
investigational new drugs or biologics and have not been approved
for any indication.
1Tonmya™ is conditionally accepted by the U.S.
Food and Drug Administration (FDA) as the tradename for TNX-102 SL
for the management of fibromyalgia. Tonmya has not been approved
for any indication.
Zembrace SymTouch and Tosymra are registered
trademarks of Tonix Medicines. 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 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 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, 2023, as filed
with the Securities and Exchange Commission (the “SEC”) on April 1,
2024, 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.
|
TONIX PHARMACEUTICALS HOLDING
CORP.CONDENSED CONSOLIDATED STATEMENTS OF
OPERATIONS(In Thousands, Except Share and Per
Share Amounts)(unaudited) |
|
|
|
Three months ended March 31, |
|
|
|
2024 |
|
|
2023 |
|
REVENUES: |
|
|
|
|
|
|
|
|
Product revenue, net |
|
$ |
2,482 |
|
|
$ |
— |
|
|
|
|
|
|
|
|
|
|
COSTS AND EXPENSES: |
|
|
|
|
|
|
|
|
Cost of sales |
|
|
1,660 |
|
|
|
— |
|
Research and development |
|
|
12,863 |
|
|
|
26,511 |
|
General and
administrative |
|
|
9,310 |
|
|
|
7,391 |
|
Total Operating Expenses |
|
|
23,833 |
|
|
|
33,902 |
|
|
|
|
|
|
|
|
|
|
Operating loss |
|
|
(21,351 |
) |
|
|
(33,902 |
) |
|
|
|
|
|
|
|
|
|
Gain on change in fair value
of warrant liabilities |
|
|
7,005 |
|
|
|
— |
|
Other (expense) income,
net |
|
|
(593 |
) |
|
|
897 |
|
|
|
|
|
|
|
|
|
|
Net loss available to common
stockholders |
|
$ |
(14,939 |
) |
|
$ |
(33,005 |
) |
|
|
|
|
|
|
|
|
|
Net loss to common
stockholders per common share, basic and diluted |
|
$ |
(0.18 |
) |
|
$ |
(3.21 |
) |
|
|
|
|
|
|
|
|
|
Weighted average common shares
outstanding, basic and diluted |
|
|
80,879,108 |
|
|
|
10,268,500 |
|
See the accompanying notes to the condensed
consolidated financial statements
|
TONIX PHARMACEUTICALS HOLDING
CORP.CONDENSED CONSOLIDATED BALANCE
SHEETS (In
Thousands)(Unaudited) |
|
|
March 31, 2024 |
|
December 31, 20231 |
Assets |
|
|
|
|
|
|
|
Cash and cash equivalents |
$ |
7,049 |
|
|
$ |
24,948 |
|
Inventory |
|
12,351 |
|
|
|
13,639 |
|
Prepaid expenses and
other |
|
10,698 |
|
|
|
9,181 |
|
Total current assets |
|
30,098 |
|
|
|
47,768 |
|
Other non-current assets |
|
105,245 |
|
|
|
106,689 |
|
Total assets |
$ |
135,343 |
|
|
$ |
154,457 |
|
|
|
|
|
|
|
|
|
Liabilities and
stockholders' equity |
|
|
|
|
|
|
|
Total liabilities |
$ |
27,200 |
|
|
$ |
48,932 |
|
Stockholders' equity |
|
108,143 |
|
|
|
105,525 |
|
Total liabilities and
stockholders' equity |
$ |
135,343 |
|
|
$ |
154,457 |
|
1The condensed consolidated balance sheet for the year ended
December 31, 2023 has been derived from the audited financial
statements but do not include all of the information and footnotes
required by accounting principles generally accepted in the United
States for complete financial statements.
Investor Contact
Jessica MorrisTonix
Pharmaceuticalsinvestor.relations@tonixpharma.com (862)
904-8182
Peter VozzoICR Westwickepeter.vozzo@westwicke.com (443)
213-0505
Media Contact
Katie
DodgeLaVoieHealthSciencekdodge@lavoiehealthscience.com(978)
360-3151
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