Interim Analysis of Registration-Enabling Phase 3 Fibromyalgia Trial of TNX-102 SL Expected Second Quarter 2023; Topline Data Expected Fourth Quarter 2023

Potentially Pivotal Phase 2 Trials of TNX-1900 in Chronic Migraine and TNX-601 ER in Depression Scheduled for Interim Analyses in Fourth Quarter 2023

Potentially Pivotal Phase 2 Fibromyalgia-Type Long COVID Study Enrolling

Cash and Cash Equivalents Totaled Approximately $120.2 Million at December 31, 2022

CHATHAM, N.J., March 13, 2023 (GLOBE NEWSWIRE) -- Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced financial results for the fourth quarter and full year ended December 31, 2022, and provided an overview of recent operational highlights.

“Our clinical activity is at a high point in the Company’s history, and we believe it is setting the stage for a year of significant accomplishments across an expanded portfolio of novel pharmaceutical candidates designed to serve major unmet medical needs,” said Seth Lederman, M.D., Chief Executive Officer of Tonix.

“We are pleased with the progress of our current Phase 3 program in fibromyalgia, and we are looking forward to the results of a planned interim analysis due next quarter, followed by topline results in the fourth quarter of this year. If successful, we believe it will be the second and final adequate and well-controlled efficacy trial required for filing a New Drug Application (NDA) for approval by the U.S. Food and Drug Administration (FDA)”, he added. “Moreover, we believe we have satisfied all the other clinical and non-clinical requirements for an NDA.”

Dr. Lederman added, “Patients and caregivers alike report widespread dissatisfaction with the three currently approved drugs for fibromyalgia – Lyrica®, Cymbalta®, and Savella®, and generic pregabalin and duloxetine – switching back and forth between them, and too often taking off-label products, including addictive opiates. Fibromyalgia affects between six and 12 million adults in the U.S. according to the American Pain Association, and there hasn’t been a new FDA drug approval in the category in more than a dozen years.”

Dr Lederman continued, “Our recently expanded late-stage clinical programs include four potentially pivotal Phase 2 trials. Two are currently enrolling, one in Long COVID and the other in chronic migraine. The two others – one in depression and the other in cocaine intoxication – are due to start enrolling. We expect to initiate enrollment in the depression study by the end of March, followed by the cocaine intoxication study in the second quarter of this year.”

“In summary”, he concluded, “these programs, together with several others in earlier development, represent a diverse portfolio of programs with multiple opportunities for value creation in 2023 and beyond.”

Recent Highlights—Key Product Candidates*

Central Nervous System (CNS) Pipeline

TNX-102 SL (cyclobenzaprine HCl sublingual tablet): small molecule for the management of fibromyalgia (FM)

TNX-102 SL for the treatment of Fibromyalgia-Type Long COVID, also known as Post-Acute Sequelae of COVID-19 (PASC)

TNX-1900 (intranasal potentiated oxytocin): small peptide for migraine, craniofacial pain, insulin resistance and related disorders, and obesity-associated binge eating disorder

TNX-601 ER (tianeptine hemioxalate extended-release tablets): a once-daily orally-administered small molecule for the treatment of major depressive disorder (MDD), Posttraumatic Stress Disorder (PTSD), and neurocognitive dysfunction associated with corticosteroid use.

TNX-1300 (recombinant double mutant cocaine esterase): biologic for life-threatening cocaine intoxication

Rare Disease Pipeline

TNX-2900 (intranasal potentiated oxytocin): small peptide for the treatment of Prader-Willi syndrome (PWS)

Immunology Pipeline

TNX-1500 (anti-CD40L monoclonal antibody): third generation anti-CD40L monoclonal antibody for prophylaxis of organ transplant rejection and treatment of autoimmune disorders.

Infectious Disease Pipeline

TNX-801 (live horsepox virus vaccine for percutaneous administration): vaccine to protect against smallpox and mpox designed as a single-administration vaccine to elicit T cell immunity

*All of Tonix’s product candidates are investigational new drugs or biologics and none have been approved for any indication.

Recent Highlights—Corporate and Other

      Recent Highlights—Financial

As of December 31, 2022, Tonix had $120.2 million of cash and cash equivalents, compared to $178.7 million as of December 31, 2021. Net proceeds from financing activities were approximately $87.8 million for full year 2022, compared to $212.5 million for the full year 2021.

Since January 1, 2023, the Company repurchased 15,700,269 shares of common stock under a $12.5 million share purchase program at prices ranging from $0.44 to $1.38 for a gross aggregate cost of approximately $12.5 million. 

In January 2023, the Board of Directors approved a new $12.5 million share repurchase program. Since January 1, 2023, the Company repurchased 1,000,000 shares of common stock under this share repurchase program at $1.14 for a gross aggregate cost of $1.1 million.

Cash used in operations was approximately $98.1 million for the full year 2022, compared to $75.6 million for the full year 2021. The increase in cash outlays was primarily due to an increase in research and development (R&D) and general and administrative (G&A) activities, described below.

Cash used by investing activities for the years ended December 31, 2022, and 2021 was approximately $48.1 million and $35.3 million, respectively, related to the purchase of property and equipment.

Fourth Quarter 2022 Financial Results

R&D expenses for the fourth quarter 2022 were $24.7 million, compared to $22.3 million for the same period in 2021. The increase is predominately due to increased employee-related, facility and laboratory expenses. We expect R&D expenses to increase during 2023 as we move our clinical development programs forward and invests in our development pipeline.

G&A expenses for the fourth quarter 2022 were $8.1 million, compared to $7.3 million for the same period in 2021. The increase is primarily due to increased employee-related and financial reporting expenses.

Net loss available to common stockholders was $34.1 million, or $0.56 per share, basic and diluted, for the fourth quarter 2022, compared to net loss of $29.6 million, or $2.08 per share, basic and diluted, for the same period in 2021. The basic and diluted weighted average common shares outstanding for the fourth quarter 2022 was 61,379,692 compared to 14,230,897 shares for the same period in 2021.

Full Year 2022 Financial Results

R&D expenses for the full year 2022 were $81.9 million, compared to $68.8 million for the same period in 2021. The increase is predominately due to increased employee-related, facility and laboratory expenses. We expect R&D expenses to increase during 2023 as we move our clinical development programs forward and invest in our development pipeline.

G&A expenses for the full year 2022 were $30.2 million, compared to $23.5 million for the same period in 2021. The increase is primarily due to increased employee-related and financial reporting expenses.

Net loss available to common stockholders was $116.9 million, or $3.27 per share, basic and diluted, for the full year 2022, compared to net loss of $92.3 million, or $8.10 per share, basic and diluted, for the same period in 2021. The basic and diluted weighted average common shares outstanding for full year 2022 was 35,739,057 compared to 11,387,308 shares for the same period in 2021.

Tonix Pharmaceuticals Holding Corp.*

Tonix is a clinical-stage biopharmaceutical company focused on discovering, licensing, acquiring and developing therapeutics to treat and prevent human disease and alleviate suffering. Tonix’s portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious disease product candidates. Tonix’s CNS 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 with a new Phase 3 study launched in the second quarter of 2022 and interim data expected in the second quarter of 2023. TNX-102 SL is also being developed to treat fibromyalgia-type Long COVID, a chronic post-acute COVID-19 condition. Tonix initiated a Phase 2 study in Long COVID in the third quarter of 2022. TNX-1900 (intranasal potentiated oxytocin), a small molecule in development for chronic migraine, is being studied in a potential pivotal Phase 2 study that initiated enrollment in the first quarter of 2023 and for which interim data is expected in the fourth quarter of 2023. TNX-601 ER (tianeptine hemioxalate extended-release tablets) is a once-daily formulation of tianeptine being developed as a potential treatment for major depressive disorder (MDD) with a Phase 2 study expected to be initiated in the first 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 second quarter of 2023. Tonix’s rare disease 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 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 and xenograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 is expected to be initiated in the second quarter of 2023. Tonix’s infectious disease pipeline includes a vaccine in development to prevent smallpox and mpox, TNX-801; a next-generation vaccine to prevent COVID-19, TNX-1850; a platform to make fully human and murine monoclonal antibodies to treat COVID-19, TNX-3600 and TNX-4100, respectively; and humanized anti-SARS-CoV-2 monoclonal antibodies, TNX-3800; and a class of broad-spectrum small molecule oral antivirals, TNX-3900. TNX-801, Tonix’s vaccine in development to prevent smallpox and mpox, also serves as the live virus vaccine platform or recombinant pox vaccine (RPV) platform for other infectious diseases. A Phase 1 study of TNX-801 is expected to be initiated in the second half of 2023.

*All of Tonix’s product candidates are investigational new drugs or 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 the failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; delays and uncertainties caused by the global COVID-19 pandemic and social and economic unrest; 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 as filed with the Securities and Exchange Commission (the “SEC”) 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)

                 
                 
  Year Ended
December 31,
  Three Months Ended
December 31,
 
  2022  2021  2022  2021 
COSTS AND EXPENSES:                
Research and development $81,876  $68,838  $24,674  $22,296 
General and administrative  30,215   23,474   8,054   7,183 
   112,091   92,312   32,728   29,479 
                 
Operating loss  (112,091)  (92,312)  (32,728)  (29,479)
                 
Interest income, net  1,873   25   1,048   (74)
                 
Net loss  (110,218)  (92,287)  (31,680)  (29,553)
                 
Preferred stock deemed dividend  6,659      2,404    
                 
Net loss available to common stockholders $(116,877) $(92,287) $(34,084) $(29,553)
                 
Net loss per common share, basic and diluted $(3.27) $(8.10) $(0.56) $(2.08)
                 
Weighted average common shares outstanding, basic and diluted  35,739,057   11,387,308   61,379,692   14,230,897 


TONIX PHARMACEUTICALS HOLDING CORP.
CONDENSED CONSOLIDATED BALANCE SHEETS
(In Thousands)
(Unaudited)

 December 31, 20221  December 31, 20211
Assets  
Cash and cash equivalents$120,229 $178,660
Prepaid expenses and other 10,548  10,389
Total current assets 130,777  189,049
Other non-current assets 94,913  51 ,851
Total assets$225,690 $240,900
   
Liabilities and stockholders' equity  
Total liabilities$18,508 $22,183
Stockholders' equity 207,182  218,717
Total liabilities and stockholders' equity$225,690 $240,900

1The condensed consolidated balance sheet for the years ended December 31, 2022 and December 31, 2021 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.

Contacts

Jessica Morris (corporate)
Tonix Pharmaceuticals
investor.relations@tonixpharma.com
(862) 904-8182

Olipriya Das, Ph.D. (media)
Russo Partners
Olipriya.Das@russopartnersllc.com
(646) 942-5588

Peter Vozzo (investors)
ICR Westwicke
peter.vozzo@westwicke.com
(443) 213-0505