CHATHAM, N.J., Nov. 13, 2024 (GLOBE NEWSWIRE) — 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 that Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals, will deliver a poster presentation at the ACR Convergence 2024 Annual Meeting, which will be held in Washington, D.C., November 14-19, 2024. A copy of the Company’s presentation will be available under the Scientific Presentations tab of the Tonix website at www.tonixpharma.com following the conference. Additional meeting information can be found on the ACR website here.
Presentation Details
Presenter:
Seth Lederman, M.D.
Title:
Randomized, Double-Blind, Placebo-Controlled Confirmatory Phase 3 Trial of Bedtime Sublingual Cyclobenzaprine (TNX-102 SL) in Fibromyalgia
Poster No.:
1218
Session:
Poster Session B, Pain in Rheumatic Disease Including Fibromyalgia Poster
Location:
Walter E. Washington Convention Center, Washington, D.C.
Date:
Sunday, November 17, 2024
Time:
10:30 a.m. – 12:30 p.m. ET
Tonix Pharmaceuticals Holding Corp.*
Tonix is a fully-integrated biopharmaceutical company focused on transforming therapies for pain management and vaccines for public health challenges. Tonix’s development portfolio is focused on central nervous system (CNS) disorders. Tonix’s priority is to advance TNX-102 SL, a product candidate for the management of fibromyalgia, for which an NDA was submitted based on two statistically significant Phase 3 studies for the management of fibromyalgia. The FDA has granted Fast Track designation to TNX-102 SL for the management of fibromyalgia. We expect an FDA decision on the acceptance of the NDA for review and a PDUFA date in December and if accepted, a decision on NDA approval in 2025. TNX-102 SL is also being developed to treat acute stress reaction and acute stress disorder under a Physician-Initiated IND at the University of North Carolina in the OASIS study funded by the U.S. Department of Defense (DoD). Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase), a biologic in Phase 2 development designed to treat cocaine intoxication that has FDA Breakthrough Therapy designation and its development is supported by a grant from the U.S. National Institute of Drug Abuse and Addiction. Tonix’s immunology development portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is an Fc-modified 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, including TNX-2900 for Prader-Willi syndrome, and infectious disease, including a vaccine for mpox, TNX-801. Tonix recently announced a contract with the U.S. DoD’s Defense Threat Reduction Agency (DTRA) for up to $34 million over five years to develop TNX-4200, small molecule broad-spectrum antiviral agents targeting CD45 for the prevention or treatment of infections to improve the medical readiness of military personnel in biological threat environments. Tonix owns and operates a state-of-the art infectious disease research facility in Frederick, MD. 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; 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. 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 Toni’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 Toni’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.
Leading heart failure specialist features tecarfarin data and Cadrenal’s proposed clinical trial protocol at 8th EACTS Mechanical Circulatory Support Summit
PONTE VEDRA, Fla., Nov. 12, 2024 — Cadrenal Therapeutics, Inc., (Nasdaq: CVKD), a late-stage biopharmaceutical company developing tecarfarin, a new vitamin K antagonist (VKA) anticoagulant, today highlighted a key opinion leader presentation at the November 2024 European Association for Cardio-thoracic Surgery (EACTS) Mechanical Circulatory Support Summit that featured tecarfarin historical data and Cadrenal’s proposed clinical trial protocol to evaluate tecarfarin versus warfarin in patients with the Abbott HeartMate3 (HM3) left ventricular assist device (LVAD).
The presentation, titled Tecarfarin and Hemocompatibility with LVAD Therapy (TECH-LVAD), took place in Prague, Czech Republic and outlined Cadrenal’s proposed clinical trial protocol recently submitted to the U.S. Food and Drug (FDA). Dr. Mandeep R. Mehra, who holds the William Harvey Chair in Advanced Cardiovascular Medicine and is Executive Director, Center for Advanced Heart Disease, Brigham and Women’s Hospital, developed and delivered the TECH-LVAD presentation. In the presentation Dr. Mehra highlighted data from past trials demonstrating the inverse relationship between bleeding rates and time in therapeutic range (TTR) for HM3 patients, and evidence from prior studies indicating tecarfarin’s potential ability to improve TTR. He included data from a trial in end-stage kidney disease (ESKD) patients showing that ESKD does not alter tecarfarin exposure while warfarin exposure is increased, explaining that this is one of the critical differentiators for tecarfarin because many LVAD patients have kidney impairment.
Dr. Mehra, who also chaired Abbott’s ARIES-HM3 study in LVAD patients and is a Professor of Medicine, Harvard University, commented, “In the proposed TECH-LVAD trial, we plan to study a much-needed VKA option with the expectation of reducing bleeding events that accompany use of the HM3 LVAD in advanced heart failure. Tecarfarin could potentially be an important therapy for patients with LVADs who all require chronic anticoagulation.”
“As our team progresses discussions with the FDA and Abbott about a tecarfarin study in LVAD patients, increasing tecarfarin data visibility will help us to continue accelerating our development as we plan for investigator outreach and patient recruitment for our tecarfarin trial,” said Quang X. Pham, Chief Executive Officer of Cadrenal Therapeutics.
ABOUT CADRENAL THERAPEUTICS, INC.
Cadrenal Therapeutics is a late-stage biopharmaceutical company developing tecarfarin, a new vitamin K antagonist (VKA) designed to offer safer, more effective chronic anticoagulation for patients with implanted cardiac devices or rare cardiovascular conditions. Tecarfarin is anticipated to result in fewer adverse events such as strokes, heart attacks, bleeds, and deaths than warfarin, the most commonly used anticoagulant for these patients, despite its prevalent adverse events, drug-to-drug interactions, and frequent dosing changes. Cadrenal is focused on evaluating tecarfarin’s superiority to warfarin in these patients where direct oral anticoagulants (DOACs) are not recommended in the treatment guidelines of leading cardiology associations. Tecarfarin received an orphan drug designation for advanced heart failure patients with implanted left ventricular assist devices (LVADs) as well as both orphan drug and fast-track status for end-stage kidney disease patients with atrial fibrillation. Cadrenal is opportunistically planning pivotal clinical trials and pursuing clinical and commercial partnerships to advance tecarfarin. The company’s plans also include studying tecarfarin in patients with mechanical heart valves experiencing anticoagulation difficulties. Visit www.cadrenal.com to learn more.
Safe Harbor Statement
Any statements contained in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements.” These statements include statements regarding our planned pivotal trial to evaluate tecarfarin’s effectiveness for LVAD patients, tecarfarin’s potential ability to improve TTR; the plan to study a much-needed VKA option with the expectation to reduce bleeding events that accompany use of the HM3 LVAD in advanced heart failure; tecarfarin potentially being an important therapy for patients with LVADs who all require chronic anticoagulation and advancing the visibility of tecarfarin data helping to continue to accelerate the Company’s development as it plans for investigator outreach and patient recruitment for its tecarfarin trial. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including the ability of tecarfarin to improve TTR and anticoagulation treatment in patients, the ability of the Company to advance tecarfarin with patients with left ventricular assist devices (LVADs) and those with ESKD with AFib, the collaborative efforts with Abbott being successful and the other risk factors described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023, and the Company’s subsequent filings with the Securities and Exchange Commission, including subsequent periodic reports on Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. Any forward-looking statements contained in this press release speak only as of the date hereof and, except as required by federal securities laws, the Company specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events, or otherwise.
For more information, please contact:
Cadrenal Therapeutics: Matthew Szot, CFO 858-337-0766 press@cadrenal.com
Investors: Lytham Partners, LLC Robert Blum, Managing Partner 602-889-9700 CVKD@lythampartners.com
Submitted New Drug Application (NDA) to FDA for TNX-102 SL for fibromyalgia based on two statistically significant Phase 3 studies
Granted Fast Track Designation by FDA in July 2024 for TNX-102 SL, a centrally-acting, non-opioid analgesic; Fibromyalgia is a common chronic pain condition that affects mostly women
Expect FDA decision in December 2024 on TNX-102 SL NDA acceptance for review and 2025 PDUFA date; If FDA-approved in 2025, TNX-102 SL would be the first new drug for fibromyalgia in more than 15 years
Presented new data on potential mpox vaccine, TNX-801, in September and October 2024, demonstrating tolerability in immunocompromised animals; Previously reported studies showed a single-dose provided immune protection against a monkeypox challenge
Awarded U.S. Department of Defense (DoD) contract for up to $34 million over five years in July 2024 to develop a broad-spectrum antiviral drug; Received first payment from DTRA
CHATHAM, N.J., Nov. 12, 2024 (GLOBE NEWSWIRE) — 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 third quarter ended September 30, 2024, and provided an overview of recent operational highlights.
“With our recent NDA submission to the U.S. Food and Drug Administration (FDA), Tonix is excited about the potential of TNX-102 SL to become the first new drug treatment option in more than 15 years for the roughly 10 million adults in the U.S. suffering from fibromyalgia,” said Seth Lederman, M.D., Chief Executive Officer of Tonix. “FDA awarded TNX-102 SL Fast Track designation in the third quarter of 2024, which is intended to expedite FDA review of important new drugs to fill unmet needs for serious conditions. We look forward to next steps with FDA. If the NDA filing is accepted in December, we expect a decision on the marketing approval of TNX-102 SL for fibromyalgia in 2025.”
Dr. Lederman continued, “As we continue to advance key pipeline products through a capital efficient strategy, we are excited to have announced collaborations with world-class institutions to advance the development of TNX-801, a potential mpox vaccine whose single-dose administration and other characteristics align closely with The World Health Organization’s preferred target product profile (TPP) criteria for mpox vaccines. The World Health Organization (WHO) previously announced the growing number of mpox cases constitutes a public health emergency of international concern (PHEIC), with clade 1b mpox strains now detected in 16 countries in Africa as well as in Sweden, Thailand, Singapore, India, England and Germany.”
Key Product Candidates* — Recent Highlights
Central Nervous System (CNS) Pipeline
TNX-102 SL (cyclobenzaprine HCl sublingual tablets): 5.6 mg, once-daily at bedtime small molecule for the management of fibromyalgia (FM) – a centrally-acting, non-opioid analgesic.
In October 2024, Tonix announced submission of the TNX-102 SL New Drug Application (NDA) for fibromyalgia to the FDA. The submission was based upon two Phase 3 studies of TNX-102 SL in fibromyalgia that showed statically significant reduction in the chronic, widespread pain associated with fibromyalgia. If approved by the FDA, TNX-102 SL would be the first member of a new class of analgesic drugs for fibromyalgia and the first new drug available for treating fibromyalgia in more than 15 years. Fibromyalgia affects more than 10 million adults in the U.S., most of whom are women.
In September 2024, at the 11th Global Conference on Pharmaceutics and Novel Drug Delivery Systems (PDDS 2024), the Company announced data highlighting the proprietary formulation technology and pharmacokinetic properties of TNX-102 SL, including composition and methods patents based on the proprietary eutectic1 formulation of TNX-102 SL that are expected to provide market exclusivity until at least 2034 in the U.S., EU, Japan, China and other jurisdictions. The eutectic protects cyclobenzaprine HCl from interacting with the basifying agent that is also part of the formulation and required for efficient transmucosal absorption. The formulation of TNX-102 SL was designed specifically for sublingual administration and transmucosal absorption for bedtime dosing to target disturbed sleep, improve pain and other fibromyalgia symptoms, while reducing the risk of daytime somnolence.
In August 2024, at both the DoD’s 2024 Military Health System Research Symposium (MHSRS), and at the International Association for the Study of Pain’s (IASP’s) 2024 World Congress on Pain, Tonix announced additional data and analyses of TNX-102 SL for the management of fibromyalgia. TNX-102 SL had met the pre-specified primary endpoint in the Phase 3 RESILIENT study, significantly reducing daily pain compared to placebo (p-value=0.00005) in participants with fibromyalgia while demonstrating broad syndromal benefits with statistically significant improvement in all six 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. TNX-102 SL was well tolerated with an adverse event profile comparable to prior studies and no new safety signals were observed.
In July 2024, Tonix noted that, based on the new definition of Long COVID by the U.S. National Academies of Sciences, Engineering and Medicine (NASEM), fibromyalgia is a ‘diagnosable condition’ in people suffering from Long COVID. The Company believes that diagnosing fibromyalgia in Long COVID patients will increase the potential market for TNX-102 SL following approval as compared to market estimates from before the COVID-19 pandemic.
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 August 2024 at the DoD’s MHSRS conference, the Company presented clinical data and rationale supporting the potential for TNX-102 SL to be studied for the treatment of ASR and prevention of PTSD. Prior studies showed that treatment with TNX-102 SL showed effects on sleep and PTSD symptoms in PTSD patients at two and four weeks1. This supportive data on the effects of TNX-102 SL on reducing PTSD symptoms suggest early intervention immediately after trauma using TNX-102 SL has the potential to reduce ASR/ASD symptoms which are similar to those of PTSD2,3. Data from these trials support testing of TNX-102 SL within 24 hours of index trauma for effects on ASR symptoms and the subsequent incidence of PTSD.
The DoD-funded Optimizing Acute Stress Reaction Interventions (OASIS) trial will be conducted by the University of North Carolina under an investigator-initiated investigational new drug (IND) application. The OASIS trial will examine the safety and efficacy of TNX-102 SL to reduce adverse posttraumatic neuropsychiatric sequelae among patients in the emergency department (ED) after a motor vehicle collision. Fourteen days of bedtime TNX-102 SL will be dosed and tested in the immediate aftermath of motor vehicle collision. The study will test the potential for TNX-102 SL to target trauma-related sleep disturbance and its ability to facilitate recovery from ASR and to prevent PTSD. The results may ultimately provide military personnel with a new treatment option that, when administered in the early aftermath of a traumatic event to individuals with ASR symptoms, improves warfighter function.
Tonix announced the first patient in the Phase 2 CATALYST study of TNX-1300 for the treatment of cocaine intoxication was enrolled in August 2024. CATALYST is a Phase 2 single-blind, placebo-controlled, proof-of-concept study in patients presenting to the emergency department. Topline results are expected in the first half of 2025.
The National Institutes of Health (NIH)’s National Institute of Drug Abuse (NIDA) previously awarded Tonix a Cooperative Agreement grant for approximately $5 million from to support development of TNX-1300.
TNX-1300 has been granted Breakthrough Therapy designation by the FDA.
Infectious Disease Pipeline
TNX-801 (recombinant horsepox virus, live vaccine): potential vaccine to protect against mpox disease and smallpox.
In September 2024, at the DoD’s MHSRS conference and in October 2024 at the World Vaccine Congress in Barcelona, Spain, Tonix presented new data on potential mpox vaccine, TNX-801, demonstrating tolerability and no evidence of spreading to blood or tissues, even at high doses, in immunocompromised animals. TNX-801 is an attenuated live-virus vaccine based on synthesized horsepox that has been shown to provide single-dose immune protection against a monkeypox challenge. After a single-dose vaccination, TNX-801 prevented clinical disease and lesions, and also decreased shedding in the mouth and lungs of non-human primates after a lethal challenge with Clade Ia monkeypox. These findings are consistent with TNX-801 inducing mucosal immunity and suggest TNX-801 has the ability to block forward transmission.
In August 2024, the WHO determined that the upsurge of mpox in a growing number of countries in Africa constitutes a public health emergency of international concern (PHEIC), the second such declaration in the past two years in response to transmission of the virus. Mpox cases of the new clade 1b mpox have since also been detected in Sweden, Thailand, Singapore, India, Germany and England.
In September 2024, the Company announced that the WHO’s preferred TPP aligns with the characteristics of TNX-801. Key elements of the WHO draft TPP include single-dose, durable protection, administration without special equipment, and stability at ambient temperature. Other potential beneficial characteristics include the ability to limit forward transmission, use in case-contact vaccination strategies and suitability for use in immunocompromised individuals.
Footnotes:
The TNX-102 SL eutectic is a composition of matter based on co-penetration of cyclobenzaprine HCl and mannitol crystals and protected by 5 issued U.S patents: Nos. 9,636,408; 9,956,188; 10,117,936; 10,864,175; 11,839,594; 9,918,948; 11,826,321.
Sullivan GM, et al. Randomized clinical trial of bedtime sublingual cyclobenzaprine (TNX-102 SL) in military-related PTSD and the role of sleep quality in treatment response. Psychiatry Res. 2021 Jul;301:113974.
Parmenter ME, et al. A phase 3, randomized, placebo-controlled, trial to evaluate the efficacy and safety of bedtime sublingual cyclobenzaprine (TNX-102 SL) in military-related posttraumatic stress disorder. Psychiatry Res. 2024 (In Press). https://doi.org/10.1016/j.psychres.2024.115764
Corporate and Partnerships – Recent Highlights
In November 2024, the Company announced that it has entered into a sponsored research agreement with the Kenya Medical Research Institute (KEMRI) to design, plan and seek regulatory approval for a Phase I clinical study in Kenya to test the safety, tolerability, and immunogenicity of TNX-801 (horsepox, live virus) as a vaccine to prevent mpox and smallpox. Tonix is expected be the sponsor and KEMRI is expected to lead the execution of the proposed clinical trial.
In October 2024, the Company announced it entered into an artificial intelligence and machine learning drug discovery collaboration with X-Chem, Inc., a leader in small molecule drug discovery, to accelerate the development of small molecules as orally available host-targeted broad-spectrum medical countermeasures. Tonix’s TNX-4200 antiviral program focuses on the development of oral CD45 phosphatase inhibitors, with broad-spectrum activity against a range of viral families.
In September 2024, Tonix announced the appointment of Thomas Englese as its new Executive Vice President, Commercial Operations. Mr. Englese brings significant leadership to Tonix across several functions, including commercial operations, sales and marketing, and launching and managing major brands through all stages of commercialization.
In August 2024, the Company announced a collaboration with Bilthoven Biologics (Bbio) to develop GMP manufacturing processes for TNX-801 as a potential mpox vaccine. Bbio is part of the world’s largest vaccine manufacturer, the Cyrus Poonawalla Group, which also includes the Serum Institute of India.
In July 2024, Tonix announced it had been awarded a DoD contract with a potential for up to $34 million over five years by DoD’s Defense Threat Reduction Agency (DTRA). The objective of the contract is to develop small molecule broad-spectrum antiviral agents for the prevention or treatment of infections to improve the medical readiness of military personnel in biological threat environments. Tonix’s program will focus on optimization and development of its TNX-4200 program, to develop an orally available CD45 antagonist, with broad-spectrum efficacy against a range of viral families through preclinical evaluation. The program is expected to establish physicochemical properties, pharmacokinetics, and safety attributes to support an IIND submission and to fund a first-in-human Phase 1 clinical study.
Marketed Products – Recent Highlights
In September 2024, Tonix announced that the United States Patent and Trademark Office (USPTO) issued U.S. Patent No. 12,097,183 to the Company, claiming use of a pre-filled autoinjector comprising a composition of Zembrace® SymTouch® for treating migraines via subcutaneous administration. This patent, excluding possible patent term extensions, is expected to fortify protection and market exclusivity into 2036.
Tonix announced that the USPTO issued U.S. Patent No. 12,090,139 to the Company, claiming a pharmaceutical composition, a method of treating migraine via intranasal administration, and an intranasal delivery system for Tosymra®. This patent is expected to fortify protection and market exclusivity into 2030.
Tonix Medicines launched a national educational campaign focusing on the link between migraine, gastroparesis, and the need for non-oral acute migraine therapies. Tonix Medicines is the only manufacturer with both a branded injectable and nasal spray indicated for the acute treatment of migraine with or without aura in adults.
Financial – Recent Highlight
As of September 30, 2024, Tonix had approximately $28.2 million of cash and cash equivalents, compared to approximately $24.9 million as of December 31, 2023. Additionally, Tonix had inventory totaling approximately $7.9 million as of September 30, 2024. Net cash used in operations was approximately $46.3 million for the nine months ended September 30, 2024, compared to approximately $79.7 million for the same period in 2023. Cash used in investing activities for the nine months ended September 30, 2024 was approximately $117,000 compared to $28.6 million for the same period in 2023.
In July 2024, Tonix received net proceeds of approximately $3.5 million in a securities offering with certain institutional and retail investors. Additionally, during the three months ended September 30, 2024, Tonix sold approximately 134.5 million shares of common stock under the 2024 ATM Sales Agreement for net proceeds of approximately $41.8 million.
Third Quarter 2024 Financial Results
Net product revenue for the third quarter 2024 was approximately $2.8 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 third quarter 2024 was approximately $1.6 million.
During the three months ended September 30, 2024, Tonix received its first payment from DTRA as part of its previously announced award from DTRA for up to $34 million over five years.
Research and development expenses for the third quarter 2024 were approximately $9.1 million, compared to $21.0 million for the same period in 2023. This decrease is predominantly due to lower clinical, non-clinical and manufacturing expenses aligned with the Company’s capital efficient strategy.
Selling, general and administrative expenses for the third quarter 2024 were approximately $7.7 million, compared to $8.7 million for the same period in 2023. The decrease was primarily due to lower employee-related expenses, transactional services and sales and marketing expenses partially offset by an increase in professional fees.
Net loss available to common stockholders was approximately $14.2 million, or $0.23 per share, basic and diluted, for the third quarter 2024, compared to net loss available to common stockholders of $28.0 million, or $38.63 per share, basic and diluted, for the same period in 2023. The basic and diluted weighted average common shares outstanding for the third quarter 2024 was 62,122,283 compared to 724,190 shares for the same period in 2023.
Tonix Pharmaceuticals Holding Corp.* Tonix is a fully-integrated biopharmaceutical company focused on transforming therapies for pain management and vaccines for public health challenges. Tonix’s development portfolio is focused on central nervous system (CNS) disorders. Tonix’s priority is to advance TNX-102 SL, a product candidate for the management of fibromyalgia, for which an NDA was submitted based on two statistically significant Phase 3 studies for the management of fibromyalgia. The FDA has granted Fast Track designation to TNX-102 SL for the management of fibromyalgia. We expect an FDA decision on the acceptance of the NDA for review and a PDUFA date in December and if accepted, a decision on NDA approval in 2025. TNX-102 SL is also being developed to treat acute stress reaction and acute stress disorder under a Physician-Initiated IND at the University of North Carolina in the OASIS study funded by the U.S. Department of Defense (DoD). Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase), a biologic in Phase 2 development designed to treat cocaine intoxication that has FDA Breakthrough Therapy designation and its development is supported by a grant from the U.S. National Institute of Drug Abuse and Addiction. Tonix’s immunology development portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is an Fc-modified 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, including TNX-2900 for Prader-Willi syndrome, and infectious disease, including a vaccine for mpox, TNX-801. Tonix recently announced a contract with the U.S. DoD’s Defense Threat Reduction Agency (DTRA) for up to $34 million over five years to develop TNX-4200, small molecule broad-spectrum antiviral agents targeting CD45 for the prevention or treatment of infections to improve the medical readiness of military personnel in biological threat environments. Tonix owns and operates a state-of-the art infectious disease research facility in Frederick, MD. 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; 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. 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.
PONTE VEDRA, Fla., Nov. 7, 2024 — Cadrenal Therapeutics, Inc., (Nasdaq: CVKD) (the “Company” or “Cadrenal”), a late-stage biopharmaceutical company developing tecarfarin, a new vitamin K antagonist (VKA) designed to provide safer and superior anticoagulation for patients with implanted cardiac devices or rare cardiovascular conditions, today provided a corporate update coinciding with the filing of its Quarterly Report on Form 10-Q for the quarter ended September 30, 2024.
Recent Highlights
In early September, Cadrenal leadership met with the U.S. Food and Drug Administration (FDA) to discuss its tecarfarin Phase 3 clinical trial protocol in left ventricular assist device (LVAD) patients and is continuing these discussions.
Cadrenal advanced Abbott collaboration discussions regarding Cadrenal’s pivotal clinical trial in patients with the Abbott LVAD HeartMate 3, the only LVAD available in the U.S.
Also, in October 2024, Cadrenal joined the Corporate Council of the Anticoagulation Forum (AC Forum), the largest professional organization of anticoagulation specialists committed to advancing the quality and safety of chronic anticoagulation care globally. Through participation in the Corporate Council, Cadrenal will collaborate with the AC Forum as it works to educate and engage the organization’s 15,000 healthcare professional members to improve outcomes for patients on anticoagulants.
Cadrenal and its pharmaceutical contract development and manufacturing organization (CDMO) completed the operational readiness activities necessary to supply active pharmaceutical ingredients and clinical trial materials in accordance with current good manufacturing principles (cGMP).
On October 24, 2024, Cadrenal announced that it successfully raised approximately $5.1 million through its at-the-market facility (ATM).
On November 1, 2024, Cadrenal announced the exercise of warrants generating gross proceeds of approximately $4.7 million.
Recent financing transactions totaling $9.8 million increased its cash balance to approximately $11.3 million and strengthened its balance sheet. The net proceeds provide Cadrenal with additional working capital as it advances tecarfarin toward a pivotal Phase 3 trial.
Q3 2024 operating expenses were $2.5 million, including $0.3 million of non-cash expenses.
Cash used in operating activities totaled $2.2 million during Q3 2024.
Cash and cash equivalent balance of $11.3 million as of November 7, 2024.
“Momentum is building from our achievement of several critical milestones toward beginning a pivotal clinical trial to evaluate tecarfarin’s superiority to warfarin in LVAD patients,” said Quang X. Pham, Founder, Chairman, and Chief Executive Officer of Cadrenal Therapeutics. “These accomplishments span finance, operations, partner relations, and clinical development and enhance our ability to execute our strategic plan going into 2025.
“Efficiently raising nearly $10 million in recent weeks bolsters funds for operational and clinical development needs. At the same time, we are progressing our dialogue with the FDA and Abbott and moving ahead with our CDMO to manufacture tecarfarin for our Phase 3 trial,” continued Pham.
Tecarfarin is the only anticoagulant in development worldwide for patients with implanted cardiac devices and other rare cardiovascular conditions. The oral and reversible drug has been uniquely designed to overcome many of the challenges patients experience with warfarin and to fill a need unmet by direct oral anticoagulants (DOACs) that are contraindicated or not recommended by leading cardiology associations for these individuals. If approved, tecarfarin may be a safer and more effective chronic anticoagulant for LVAD patients in the U.S.
In addition, tecarfarin may prove valuable for other patients where warfarin is not providing recommended anticoagulation because of genetic warfarin resistance or renal impairment making warfarin metabolism difficult. These include individuals with end-stage renal disease and atrial fibrillation or those with mechanical heart valves and hard-to-control anticoagulation, as determined by International Normalized Ratio (INR) measurements of how long it takes the blood to clot.
ABOUT CADRENAL THERAPEUTICS, INC.
Cadrenal Therapeutics is a late-stage biopharmaceutical company developing tecarfarin, a new vitamin K antagonist (VKA) designed to offer safer, more effective chronic anticoagulation for patients with implanted cardiac devices or rare cardiovascular conditions. Tecarfarin is anticipated to result in fewer adverse events such as strokes, heart attacks, bleeds, and deaths than warfarin, the most commonly used anticoagulant for these patients, despite its prevalent adverse events, drug-to-drug interactions, and frequent dosing changes. Cadrenal is focused on evaluating tecarfarin’s superiority to warfarin in these patients where DOACs are not recommended in the treatment guidelines of leading cardiology associations. Tecarfarin received an orphan drug designation for advanced heart failure patients with implanted LVADs as well as both orphan drug and fast-track status for end-stage kidney disease patients with atrial fibrillation. Cadrenal is opportunistically planning pivotal clinical trials and pursuing clinical and commercial partnerships to advance tecarfarin. The company’s plans also include studying tecarfarin in patients with mechanical heart valves experiencing anticoagulation difficulties. Visit www.cadrenal.com to learn more.
About Tecarfarin
Tecarfarin is a Phase 3-ready drug candidate that Cadrenal is developing to overcome many of warfarin’s challenges and fill the need for a safer and more effective VKA chronic anticoagulant. Tecarfarin is anticipated to improve outcomes and result in fewer major events for warfarin-dependent patients. Extensive data indicates that the efficacy of tecarfarin, metabolized via a different pathway than warfarin, is not affected by drug-drug interactions and kidney impairment, which are common in these patients. Phase 2/3 clinical trials show that tecarfarin may offer enhanced stability and time in therapeutic range (TTR) that inversely correlate with major events. Tecarfarin is the only new anticoagulant being developed for patients with implanted cardiac devices or rare cardiovascular conditions. Treatment with tecarfarin aims to improve anticoagulation for these underserved patients and their healthcare providers who face difficulties in managing warfarin’s wide variability and risk of gastrointestinal bleeds.
Safe Harbor Statement
Any statements contained in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, may constitute “forward-looking statements.” These statements include statements regarding our planned pivotal trial to evaluate tecarfarin’s effectiveness for LVAD patients, the success of the Company’s collaborative efforts with Abbott, tecarfarin potentially being a safer and more effective chronic anticoagulant for patients with LVADs, tecarfarin filling a need unmet by DOACs that are contraindicated or not recommended, the commencement of trials to evaluate tecarfarin’s superiority to warfarin in LVAD patients and potentially bring the Company’s better anticoagulation solution to those in need, and tecarfarin proving valuable for other patients where warfarin is not providing recommended anticoagulation because of genetic warfarin resistance or renal impairment making warfarin metabolism difficult. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “will,” “would” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including the ability of tecarfarin to improve anticoagulation treatment in patients, the ability of the Company to advance tecarfarin with patients with left ventricular assist devices (LVADs), the collaborative efforts with Abbott being successful and those with AFib and ESKD, the collaboration with Abbott being successful and the other risk factors described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023, and the Company’s subsequent filings with the Securities and Exchange Commission, including subsequent periodic reports on Quarterly Reports on Form 10-Q and Current Reports on Form 8-K. Any forward-looking statements contained in this press release speak only as of the date hereof and, except as required by federal securities laws, the Company specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events, or otherwise.
For more information, please contact:
Cadrenal Therapeutics: Matthew Szot, CFO 858-337-0766 press@cadrenal.com
Investors: Lytham Partners, LLC Robert Blum, Managing Partner 602-889-9700 CVKD@lythampartners.com
Key Points: – Avid Bioservices will be acquired by GHO Capital and Ampersand Capital in a $1.1 billion deal, with a 13.8% per-share premium. – Acquisition to enhance Avid’s biologics CDMO services with expanded resources for development and manufacturing. – Expected to close in Q1 2025, enabling Avid to operate privately and accelerate its service offerings for the biotechnology sector.
Avid Bioservices, Inc. (NASDAQ: CDMO), a major biologics contract development and manufacturing organization (CDMO), announced its acquisition by GHO Capital Partners LLP and Ampersand Capital Partners for approximately $1.1 billion. The all-cash transaction positions Avid for substantial growth, with the backing of experienced healthcare-focused investors to expand its development and manufacturing capabilities in the biotechnology sector. This acquisition marks a strategic move for GHO and Ampersand, leveraging Avid’s expertise to strengthen their portfolios in the life sciences industry.
Under the agreement terms, GHO and Ampersand will acquire Avid’s outstanding shares for $12.50 each in cash, reflecting a 13.8% premium over Avid’s closing share price and a 21.9% premium over its 20-day average. This values the transaction at $1.1 billion, enhancing Avid’s growth potential within a private company framework where it can develop its offerings with the support of dedicated capital and expanded industry networks.
Alan MacKay and Mike Mortimer, Managing Partners of GHO, expressed their excitement to work with Avid’s team to realize the company’s full potential, calling Avid “an ideal addition” that exemplifies their mission to improve healthcare access through efficient manufacturing and high-quality innovation. The deal will enable Avid to maintain its focus on serving the biotechnology and pharmaceutical sectors, helping it meet growing demand for complex biologics at clinical and commercial stages. Additionally, the acquisition aligns with GHO and Ampersand’s broader healthcare strategies, aiming to optimize Avid’s impact in global markets.
Nick Green, President and CEO of Avid, noted that partnering with GHO and Ampersand comes at an opportune time, as Avid has been strategically expanding to meet a broader range of customer needs. He emphasized that this partnership will further Avid’s impact, positioning it to better serve biopharma innovators by utilizing GHO and Ampersand’s resources. “After years of investment and expansion, now is the right time to move forward as a private company,” Green stated, expressing confidence that this move will significantly enhance Avid’s capabilities.
The acquisition is expected to support Avid’s future projects, including cell line development, CGMP clinical and commercial manufacturing, analytical testing, and expanded services for early-stage programs. This additional support allows Avid to optimize customer offerings, broaden its expertise, and develop new industry capabilities that align with its dedication to quality and regulatory standards.
The acquisition, unanimously approved by Avid’s board of directors, is expected to close in Q1 2025, pending customary approvals. Avid’s shares will no longer be publicly traded after the acquisition, and the company will continue to operate under its current name and brand identity, with its headquarters remaining in Tustin, California. Financial and legal advising for Avid are being handled by Moelis & Company LLC and Cooley LLP, respectively, while GHO and Ampersand are advised by William Blair & Company and Ropes & Gray LLP.
This partnership reflects GHO and Ampersand’s strategic investment approach, enabling Avid to strengthen its leadership in biologics manufacturing and develop solutions that respond to high-growth demand in the industry.
Proposed clinical study in Kenya intended to test the safety, tolerability, and immunogenicity of TNX-801, a vaccine being developed to prevent mpox (formerly called monkeypox)
The World Health Organization (WHO) declared mpox a public health emergency of international concern (PHEIC)1-4: cases of the new Clade Ib mpox detected in Sweden, Thailand, Singapore, India, Germany and England
In preclinical data, TNX-801, Tonix’s attenuated live-virus vaccine candidate, demonstrated efficacy as a vaccination against mpox in animal models
CHATHAM, N.J., Nov. 04, 2024 (GLOBE NEWSWIRE) — 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 that it has entered into a sponsored research agreement with the Kenya Medical Research Institute (KEMRI) to design, plan and seek regulatory approval for a Phase I clinical study in Kenya to test the safety, tolerability, and immunogenicity of TNX-801 (horsepox, live virus) as a vaccine to prevent mpox and smallpox. Tonix will be the sponsor and KEMRI will lead the execution of the proposed clinical trial.
“We are excited to advance development of TNX-801 under this research agreement with KEMRI,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “There is an urgent need for the worldwide availability of a single dose mpox vaccine with durable protection. TNX-801 has demonstrated encouraging preclinical data and was recently shown to align with the World Health Organization’s (WHO’s) newly issued preferred target product profile (TPP) for mpox vaccines. Further, TNX-801 can be scaled up for manufacturing, distribution and storage without a costly supply chain. We believe TNX-801 has the potential to address and help interrupt the spread of mpox worldwide.”
In September 2024, Tonix announced that the preferred target product profile (TPP), released at the WHO sponsored Mpox Research and Innovation Scientific Conference, aligns with the potential characteristics of TNX-801. Key elements of the WHO draft TPP include single-dose, durable protection, administration without special equipment, and stability at ambient temperature. Other potential beneficial characteristics include the ability to limit forward transmission, use in case-contact vaccination strategies and suitability for use in immunocompromised individuals.
In August 2024, Tonix announced a collaboration with Bilthoven Biologics (Bbio) to develop GMP manufacturing processes for TNX-801. Bbio is part of the world’s largest vaccine manufacturer, the Cyrus Poonawalla Group, which also includes the Serum Institute of India.
About TNX-801*
TNX-801 is a live minimally-replicating attenuated horsepox vaccine that is believed to provide immune protection against mpox in animals with better tolerability than 20th century vaccinia viruses. Vaccinia vaccines from the 20th century are descendants of Edward Jenner’s circa 1800 that have become more virulent in connection with losing regulatory elements in their genomes. Given the modern understanding that Jenner’s circa 1800 vaccine would be called “horsepox” today, TNX-801 was designed to be similar to Jenner’s vaccine. After a single dose vaccination, TNX-801 protected animals against lethal challenge with intratracheal Clade Ia monkeypox virus.5 In this experiment, TNX-801 vaccination prevented clinical disease and skin lesions and also decreased shedding in the mouth and lungs. The findings are consistent with mucosal immunity and suggest the ability to block forward transmission. In addition, TNX-801 has decreased virulence in immunocompromised animals relative to 20th Century vaccinia viruses.6 Based on animal studies, TNX-801 combines immune protection with improved tolerability compared to live-virus vaccinia vaccines. TNX-801 is administered with a single dose which has advantages over two-dose regimens. The focus on single-dose vaccines confirms early recommendations by the Bipartisan Commission on Biodefense, 7 and the U.S. National Academies of Science.7,8 The National Academies of Science (NAS) report highlights the difficulty of a ring vaccination strategy with even a two-dose regimen.7 TNX-1800 is an engineered version of horsepox that expresses the spike protein of SARS-CoV-2. 9,10 The U.S. National Institutes of Health (NIH) selected Tonix’s COVID-19 vaccine, TNX-1800 for Project NextGen.
About Mpox* On August 14, 2024, the WHO determined that the upsurge of mpox in a growing number of countries in Africa constitutes a PHEIC, the second such declaration in the past two years called in response to an mpox outbreak.1 The current outbreak is caused by Clade Ib monkeypox virus while the 2022 outbreak was caused by Clade IIb monkeypox virus. The 2022 global mpox outbreak, which is ongoing, has affected over 90,000 persons in countries where mpox had previously not been endemic, including Europe and the US. The rapid spread of Clade IIb strain mpox in 2022 underscores the pandemic potential of mpox. Unlike Clade IIb mpox, the Clade Ib strain of mpox appears to be spreading in Africa, particularly those neighboring the Democratic Republic of the Congo, including Burundi, Rwanda, and Uganda, but now affecting 16 countries. According to the U.S. Centers for Disease Control and Prevention (CDC), and other experts, there is a significant risk that the Clade Ib monkeypox may appear in the U.S.2,3
Tonix Pharmaceuticals Holding Corp.* Tonix is a fully integrated biopharmaceutical company focused on transforming therapies for pain management and modernizing solutions for public health challenges. Tonix’s development portfolio is focused on central nervous system (CNS) disorders, and its priority is to progress TNX-102 SL, a product candidate for which an NDA was submitted based on two statistically significant Phase 3 studies for the management of fibromyalgia. The FDA has granted Fast Track designation to TNX-102 SL for the management of fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction and acute stress disorder under a Physician-Initiated IND at the University of North Carolina in the OASIS study funded by the U.S. Department of Defense (DoD). Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase), a biologic in Phase 2 development designed to treat cocaine intoxication that has FDA Breakthrough Therapy designation and its development is supported by a grant from the U.S. National Institude of Drug Abuse and Addiction. Tonix’s immunology development portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is an Fc-modified 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, including TNX-2900 for Prader-Willi syndrome, and infectious disease, including a vaccine for mpox, TNX-801. Tonix has a contract with the U.S. DoD’s Defense Threat Reduction Agency (DTRA) for up to $34 million over five years to develop TNX-4200, small molecule broad-spectrum antiviral agents targeting CD45 for the prevention or treatment of infections to improve the medical readiness of military personnel in biological threat environments. Tonix owns and operates a state-of-the art infectious disease research facility in Frederick, MD. 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; 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. All other marks are property of their respective owners.
This press release and further information about Tonix can be found at www.tonixpharma.com.
1WHO Press Release August 14, 2024. “WHO Director-General declares mpox outbrfeak a public health emergency of international concern”. URL: www.who.int/news/item/14-08-2024-who-director-general-declares-mpox-outbreak-a-public-health-emergency-of-international-concern (accessed 8-15-24) 2McQuiston JH, et al. U.S. Preparedness and Response to Increasing Clade I Mpox Cases in the Democratic Republic of the Congo. 2024, MMWR Morbi Mortal Wkly Rep: United States. p. 435-440 3CDC. 2022-2023 Mpox: US Map and Case Count. https://www.cdc.gov/poxvirus/mpox/response/2022/us-map.html 4World Health Organization SAGE meeting highlights on updated mpox vaccine recommendations. 2024, March 5Noyce RS, et al. Viruses. 2023 Jan 26;15(2):356. Doi: 10.3390/v15020356. PMID: 36851570; PMCID: PMC9965234 6Trefry, SV et al. bioRxiv 2023.10.25.564033; doi: https://doi.org/10.1101/2023.10.25.564033 7Bipartisan Commission on Biodefense. Box the Pox: Reducing the risk of Smallpox and Other Ortho poxviruses, Washington:2024 8U.S. National Academies of Science. Future State of Smallpox Medical Countermeasures. Washington:2024 9Awasthi M, et al. Viruses. 2023 Oct 21;15(10):2131. Doi: 10.3390/v15102131. PMID: 37896908; PMCID: PMC10612059. 10Awasthi M et al Vaccines (Basel). 2023 Nov 2;11(11):1682. Doi: 10.3390/vaccines11111682.PMID: 38006014
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 Toni’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 Toni’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.
The Company’s antiviral candidate for seasonal and pandemic influenza shows in vitro activity against the avian influenza A PB2 protein, whereas current influenza vaccines offer no protection against pandemic avian influenza
BOTHELL, Wash., Oct. 31, 2024 (GLOBE NEWSWIRE) — As the fall seasonal influenza and COVID season gets underway in the Northern Hemisphere, Cocrystal Pharma, Inc. (Nasdaq: COCP) (“Cocrystal” or the “Company”) highlights the ability of the Company’s innovative structure-based drug discovery platform technology to discover and develop novel broad-spectrum antivirals therapeutics to treat a wide range of viral diseases, including newly emerging pandemic strains such as recent H5N1 avian influenza identified in the US.
“As our nation enters the fall flu and COVID season and learns of the emerging highly pathogenic avian H5N1 influenza A strain in dairy cattle and humans, the need for more effective antivirals is clear. Current flu vaccines are developed for seasonal influenza strains, not for pandemic avian influenza strains. Also, treatment-emergent resistance to approved antivirals and transmission of resistant viruses has been a challenging issue,” said Sam Lee, PhD, President and co-CEO of Cocrystal. “For example, the widespread oseltamivir (Tamiflu®) resistance of the pandemic avian influenza strains could create a serous public health situation. Clearly, there continues to be an unmet need for therapeutics with a high barrier to resistance.
“We believe our approach makes it possible to develop highly effective therapeutics for noroviruses, coronaviruses and influenza A because we target the highly conserved, essential function of viral enzymes, regardless of whether the strain is seasonal or pandemic,” he added. “We recently revealed the high-resolution cocrystal structure of the avian influenza PB2 protein complexed with CC-42344, further confirming that our PB2 inhibitor CC-42344 binds to its highly conserved PB2 region, indicating activity against this strain.”
Cocrystal’s platform utilizes Nobel Prize-winning technology to develop a new class of direct-acting antivirals that work against enzymes that are essential for viral replication. The Company is evaluating its oral CC-42344 in a Phase 2a study in healthy subjects infected with a seasonal influenza A strain. Topline safety and tolerability results from this trial are expected by the end of 2024, and preparations are underway for an Investigational New Drug (IND) application to conduct a late-stage clinical study with CC-42344 in the U.S.
The Company is also conducting a Phase 1 study with its oral protease inhibitor CDI-988, the first pan-viral drug candidate in clinical evaluation for both noroviruses and coronaviruses. Topline safety and tolerability results of the multiple-ascending cohorts are expected late this year or early next year. There is no approved vaccine or antiviral for norovirus. Norovirus is highly contagious and is the most common cause of acute gastroenteritis, which has gained notoriety for outbreaks in closed quarters such as on cruise ships and in nursing homes. According to the Centers for Disease Control and Prevention (CDC), an estimated 685 million cases and an estimated 200,000 deaths are attributed to norovirus each year worldwide, with an estimated societal cost of approximately $60 billion.
“We view the next viral pandemic as a question of timing as seasonal viruses like flu and COVID continue to evolve,” said James Martin, CFO and co-CEO of Cocrystal. “Our proprietary technology platform allows us to efficiently discover and develop potent, broad-spectrum, effective antiviral drug candidates for pandemic and seasonal outbreaks relatively quickly and far less costly than traditional approaches to drug development. As a small company, Cocrystal is highly efficient in utilizing our groundbreaking technology to develop differentiated antivirals for high-value indications with the goal of improving people’s lives.”
Avian Influenza A multistate outbreak of highly pathogenic avian influenza in dairy cows was initially reported in March 2024. This is the first time that avian flu viruses were found in cows, with outbreaks now confirmed in herds in 14 states. In April 2024 the CDC confirmed an avian flu infection in a person exposed to dairy cows that were presumed to be infected with the virus. This is believed to be the first instance of likely mammal to human spread of this virus. In September 2024 the CDC reported the first human case of avian influenza without a known occupational exposure to sick or infected animals. As of October 30, 2024, the CDC has reported 36 human cases of this highly pathogenic avian influenza A in the U.S. during 2024.
The CDC analyzed blood collected from people of all ages in all 10 Health & Human Services regions during the 2022-2023 and 2021-2022 flu seasons. These samples were challenged with the avian flu subtype H5N1 virus to determine whether there was an antibody reaction. Data from this study suggest that there is extremely low to no population immunity to clade 2.3.4.4b A (H5N1) viruses in the U.S. Antibody levels remained low regardless of whether or not participants received a seasonal flu vaccination, meaning that seasonal flu vaccination did not produce antibodies to avian flu H5N1 viruses.
Antiviral Influenza Candidate CC-42344 CC-42344 is Cocrystal’s novel, broad-spectrum investigational antiviral candidate for the treatment of pandemic and seasonal influenza A. CC-42344 inhibits the first step in influenza A’s viral replication by binding to a highly conserved PB2 site of the influenza polymerase complex that is essential to replication and was discovered using Cocrystal’s proprietary structure-based drug discovery platform technology.
Cocrystal is evaluating safety, viral and clinical measures of oral CC-42344 in healthy volunteers who are challenged with influenza A in a Phase 2a human challenge study underway in the United Kingdom. CC-42344 was advanced into this study following favorable safety and tolerability results reported in a Phase 1 study in healthy volunteers conducted in Australia. In vitro testing showed CC-42344’s excellent antiviral activity against influenza A strains, including pandemic and seasonal strains, as well as against strains resistant to Tamiflu® and Xofluza®, while also demonstrating favorable pharmacokinetic and safety profiles.
Cocrystal used its structure-based platform to determine the high resolution X-ray crystal structure of the recent avian influenza A (H5N1) PB2 protein, and confirmed activity of CC-42344 in vitro (NIH GeneBank ID:influenza A/Texas/37/2024(H5N1). The crystal structure of the avian influenza A (H5N1) PB2 protein showed new mutations located outside the PB2 active site. Subsequent studies showed that CC-42344 binds to the active site of the avian influenza A (H5N1) PB2 protein as previously demonstrated with the pandemic and seasonal influenza A PB2.
Structure-Based Platform Technology Cocrystal’s proprietary structural biology, along with its expertise in enzymology and medicinal chemistry, enable its development of novel antiviral agents. The Company’s platform provides a three-dimensional structure of inhibitor complexes at near-atomic resolution, providing immediate insight to guide Structure Activity Relationships. This helps to identify novel binding sites and allows for a rapid turnaround of structural information through highly automated X-ray data processing and refinement. The goal of this technology is to facilitate the development of best-in-class antiviral therapies that have fast onset of action and/or shortened treatment time, are safe, well tolerated and easy to administer, are effective against all viral subtypes that cause disease and have a high barrier to viral resistance.
About Cocrystal Pharma, Inc. Cocrystal Pharma, Inc. is a clinical-stage biotechnology company discovering and developing novel antiviral therapeutics that target the replication process of influenza viruses, coronaviruses (including SARS-CoV-2), noroviruses, and hepatitis C viruses. Cocrystal employs unique structure-based technologies and Nobel Prize-winning expertise to create first- and best-in-class antiviral drugs. For further information about Cocrystal, please visit www.cocrystalpharma.com.
Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the potential efficacy of Cocrystal’s product candidates against certain viruses, ongoing research and development efforts including the expected timing of clinical studies and topline results for such product candidates, and the potential market for such product candidates. The words “believe,” “may,” “estimate,” “continue,” “anticipate,” “intend,” “should,” “plan,” “could,” “target,” “potential,” “is likely,” “will,” “expect” and similar expressions, as they relate to us, are intended to identify forward-looking statements. We have based these forward-looking statements largely on our current expectations and projections about future events. Some or all of the events anticipated by these forward-looking statements may not occur. Important factors that could cause actual results to differ from those in the forward-looking statements include, but are not limited to, risks relating to our ability to obtain regulatory authority for and proceed with clinical trials including the recruiting of volunteers for such studies by our clinical research organizations and vendors, the results of such studies, our collaboration partners’ technology and software performing as expected, general risks arising from clinical studies, receipt of regulatory approvals, regulatory changes, and potential development of effective treatments and/or vaccines by competitors, including as part of the programs financed by the U.S. government, and potential mutations in a virus we are targeting that may result in variants that are resistant to a product candidate we develop. Further information on our risk factors is contained in our filings with the SEC, including our Annual Report on Form 10-K for the year ended December 31, 2023. Any forward-looking statement made by us herein speaks only as of the date on which it is made. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to publicly update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by law.
Participation Reinforces Cadrenal’s Commitment to Innovation, Education and Improved Patient Outcomes with Anticoagulation Therapy
PONTE VEDRA, Fla., Oct. 23, 2024 — Cadrenal Therapeutics, Inc. (Nasdaq: CVKD), a late-stage biopharmaceutical company developing tecarfarin, a new vitamin K antagonist (VKA) designed to improve anticoagulation for patients with implanted cardiac devices or rare cardiovascular conditions, today announced its membership in the Corporate Council of the Anticoagulation Forum (AC Forum).
The AC Forum is the largest professional organization of anticoagulation specialists, committed to advancing the quality and safety of chronic anticoagulation care globally. Through participation in the Corporate Council, Cadrenal Therapeutics will collaborate with the AC Forum as it works to identify and address unmet clinical needs, share cutting-edge research, and promote advocacy and educational initiatives for the organization’s 15,000 healthcare professional members aimed at improving outcomes for patients on anticoagulants.
“Our membership in the AC Forum’s Corporate Council reflects our shared commitment to transforming anticoagulation care,” said Quang X. Pham, Chief Executive Officer of Cadrenal Therapeutics. “We look forward to partnering with the AC Forum and contributing to programs that advance safer, more effective care and inform about ongoing research and anticoagulation best practice guidelines.”
Darren Triller, PharmD, Director of Strategic Initiatives at the Anticoagulation Forum, welcomed Cadrenal Therapeutics to the Corporate Council, saying: “We are excited to have Cadrenal Therapeutics join us in our mission to improve the quality of care for patients receiving antithrombotic therapies. We look forward to a productive partnership that will help us address the challenges in the anticoagulation landscape together.”
About Cadrenal Therapeutics, Inc.
Cadrenal Therapeutics is a late-stage biopharmaceutical company developing tecarfarin, a new vitamin K antagonist (VKA) designed to offer safer, superior chronic anticoagulation for patients with implanted cardiac devices or rare cardiovascular conditions. Tecarfarin is anticipated to result in fewer adverse events such as strokes, heart attacks, bleeds and deaths than warfarin, the most commonly used anticoagulant for these patients despite its prevalent side effects, drug-to-drug interactions and frequent dosing changes. Tecarfarin received an orphan drug designation for advanced heart failure patients with implanted left ventricular assist devices (LVADs) as well as both orphan drug and fast-track status for end-stage kidney disease patients with atrial fibrillation. Cadrenal is planning pivotal clinical trials and pursuing clinical and commercial partnerships. The company’s plans also include studying tecarfarin in patients with mechanical heart valves experiencing anticoagulation difficulties. Visit www.cadrenal.com to learn more.
About the Anticoagulation Forum
The Anticoagulation Forum has led advancements in thrombosis-related care for over three decades. With more than 15,000 members across 3,000 healthcare institutions, the AC Forum is dedicated to enhancing the safety and quality of care for over 1 million patients annually. Through education, thought leadership, and partnerships with industry leaders, the AC Forum shapes the future of anticoagulation management to ensure the best outcomes for patients.
For more information, please contact: Cadrenal Therapeutics: Matthew Szot, CFO 858-337-0766 press@cadrenal.com
Investors: Lytham Partners, LLC Robert Blum, Managing Partner 602-889-9700 CVKD@lythampartners.com
First payment received under Tonix’s contract with the Defense Threat Reduction Agency (DTRA), for up to $34 million over five years
CHATHAM, N.J., Oct. 17, 2024 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a fully-integrated biopharmaceutical company with marketed products and a pipeline of development candidates, received its first payment from the Defense Threat Reduction Agency (DTRA), an agency within the U.S. Department of Defense (DoD), to develop small molecule broad-spectrum antiviral agents for the prevention or treatment of infections to improve the medical readiness of military personnel in biological threat environments. The previously announced award from DTRA is for up to $34 million over five years.
“This award provides important validation and substantial non-dilutive funding for our ongoing research to advance our antiviral discovery program,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals “With biological adaptations and mutations happening to viruses rapidly across the globe, this research will be crucial in order to protect lives in the event biological threats are introduced onto the battlefield.”
The $34.0 million five-year contract will help fund and accelerate the development of the Company’s broad-spectrum antiviral program, which has the potential to reduce viral load and allow the adaptive immune system to alert the other arms of the immune system to mount a protective response. Tonix plans to leverage previous research on phosphatase inhibitors, specifically compounds that target CD45, to optimize lead compounds for therapeutic intervention of biothreat agents and provide the government with a complete and cost-effective solution for a broad-spectrum medical countermeasure. Tonix’s premise is that partial inhibition of CD45 will provide optimal antiviral protection while requiring lower plasma drug concentrations, a lower dose, and a better safety profile.
Tonix will utilize its state-of-the-art research laboratory capabilities, including a Biosafety Level 3 (BSL-3) lab at its research and development center (RDC) facility located in Frederick, Md., as well as experienced personnel in-house. The RDC is located in Maryland’s ‘I-270 biotech corridor’ and is close to the center of the U.S. biodefense research community.
About Defense Threat Reduction Agency (DTRA) The Defense Threat Reduction Agency (DTRA), an agency within the United States Department of Defense (DoD) is both a Defense Agency and Combat Support Agency with two distinct yet highly integrated roles countering Weapons of Mass Destruction (WMD) and emerging threats. Its origins stretch back to World War II and the Manhattan Project, but today the agency encompasses a wide variety of strategic and operational functions that deter, prevent, and ultimately prevail against these unique threats. DTRA enables the Department of Defense (DoD), the United States Government and international partners to counter and deter weapons of mass destruction (WMD) and emerging threats. DTRA provides cross-cutting solutions to enable the Department of Defense, the United States Government, and international partners to deter strategic attack against the United States and its allies; prevent, reduce, and counter WMD and emerging threats; and prevail against WMD-armed adversaries in crisis and conflict. DTRA’s continued effort to enhance the combat support mission also advances public health services by developing innovative technologies that protect against biological threats. For more information, visit www.dtra.mil.
Tonix Pharmaceuticals Holding Corp.** Tonix is a fully integrated biopharmaceutical company focused on transforming therapies for pain management and modernizing solutions for public health challenges. Tonix’s development portfolio is focused on central nervous system (CNS) disorders, and its priority is to progress TNX-102 SL, a product candidate for which an NDA was submitted based on two statistically significant Phase 3 studies for the management of fibromyalgia. The FDA has granted Fast Track designation to TNX-102 SL for the management of fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction and acute stress disorder under a Physician-Initiated IND at the University of North Carolina in the OASIS study funded by the U.S. Department of Defense (DoD). Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase), a biologic in Phase 2 development designed to treat cocaine intoxication that has FDA Breakthrough Therapy designation and its development is supported by a grant from the U.S. National Institude of Drug Abuse and Addiction (NIDA). Tonix’s immunology development portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is an Fc-modified 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, including TNX-2900 for Prader-Willi syndrome, and infectious disease, including a vaccine for mpox, TNX-801. Tonix recently announced a contract with the U.S. DoD’s Defense Threat Reduction Agency (DTRA) for up to $34 million over five years to develop TNX-4200, small molecule broad-spectrum antiviral agents targeting CD45 for the prevention or treatment of infections to improve the medical readiness of military personnel in biological threat environments. Tonix owns and operates a state-of-the art infectious disease research facility in Frederick, MD. 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; 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. 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.
Lead indication for ZyVersa’s Inflammasome ASC Inhibitor IC 100 is obesity with metabolic complications.
WESTON, Fla., Oct. 17, 2024 (GLOBE NEWSWIRE) — ZyVersa Therapeutics, Inc. (Nasdaq: ZVSA, or “ZyVersa”), a clinical stage specialty biopharmaceutical company developing first-in-class drugs for treatment of inflammatory and renal diseases, announces that Stephen C. Glover, Co-Founder, Chairman, Chief Executive Officer, and President, has issued a Letter to Shareholders highlighting obesity development plans for Inflammasome ASC Inhibitor IC 100 with anticipated milestones over the next nine months. The full text of the letter follows.
A MESSAGE FROM OUR PRESIDENT AND CHIEF EXECUTIVE OFFICER
To my fellow shareholders,
As announced in late July, ZyVersa selected obesity with metabolic complications as the lead indication for Inflammasome ASC Inhibitor IC 100. Selection of this indication is consistent with our mission to restore health and transform patient lives through innovation. Although GLP-1 agonist therapy has changed the treatment paradigm for obesity with unsurpassed weight loss, significant unmet medical needs remain. A critical unmet need is for therapies to address the chronic systemic inflammation of obesity that causes life-altering metabolic comorbidities such as cardiovascular diseases, type 2 diabetes, non-alcoholic fatty liver disease, and even neurodegenerative diseases such as Parkinson’s and Alzheimer’s diseases. Based on IC 100’s highly differentiated mechanism of action, it is anticipated that IC 100 will control the chronic systemic inflammation of obesity, attenuate development and/or progression of associated metabolic comorbidities, and augment weight loss when added to GLP-1 agonist therapy.
Selection of obesity with metabolic complications as the lead indication for IC 100 is also consistent with ZyVersa’s goal to drive shareholder value. The obesity drug revolution has led to a 10-fold increase in private venture dollars flowing into the category over the last 5 years and high deal volume. This is expected to continue with investment opportunities in R&D innovation to develop GLP-1 agonist add-on therapies to address unmet needs. This is exemplified by some of the latest transactions:
Oct 10, 2024: Lilly announced up to $1.4 billion plus upfront payment and royalties for expanded collaboration with KeyBioscience for rights to dual amylin calcitonin receptor agonists for obesity and complications (e.g., osteoarthritis).
September 25, 2024: Bioage announced an upsized $198 Million IPO driven by their APJ agonist in development for muscle preservation and improved metabolism to be used in combination with GLP-1 agonist therapy.
September 23, 2024: Sanofi announced a $27 Million strategic investment in Ventyx Biosciences’ NLRP3 inhibitor VTX3232, which is phase 2 for obesity with cardiovascular complications and Parkinson’s disease.
September 4, 2024: Lilly announced a collaboration with Haya Therapeutics for up to $1 Billion for drug discovery in obesity and related metabolic conditions.
August 10, 2024: Novo Nordisk announced acquisition of Inversago Pharma, valued up to $1.075 Billion. Inversago develops CB1 receptor-based therapies for obesity, diabetes, and complications associated with metabolic disorders.
We are excited about the potential for Inflammasome ASC Inhibitor IC 100 as an add-on therapy to GLP-1 agonists to help restore health and transform the lives of patients living with obesity and its associated comorbidities. We recently reviewed the preclinical data supporting IC 100 with our newly formed Obesity, Metabolic, and Inflammatory Disease Scientific Advisory Board. Advisory Board members indicated that IC 100’s mechanism of action supports its rationale for use as an add-on treatment to GLP-1 agonists to address the chronic inflammation of obesity and its associated metabolic complications. They also provided excellent input on our planned DIO obesity model studies to strengthen their design and endpoints for proof-of-concept of IC 100’s potential as a treatment for obesity with metabolic complications.
Following are key IC 100 short-term obesity development milestones that we expect to achieve:
Q4-2024: Initiate IC 100 monotherapy study using semaglutide as a comparator in DIO mice.
Q1- 2025: Initiate IC 100 combination study with semaglutide in DIO mice.
Q2-2025: File IND.
Q3-2025: Initiate IC 100 phase 1 trial in healthy overweight subjects with a BMI 27 – 30.
We appreciate your current and ongoing support that enables us to progress IC 100’s development program to drive shareholder value.
About Inflammasome ASC Inhibitor IC 100
IC 100 is a novel humanized IgG4 monoclonal antibody that inhibits the inflammasome adaptor protein ASC. IC 100 was designed to attenuate both initiation and perpetuation of the inflammatory response. It does so by binding to a specific region of the ASC component of multiple types of inflammasomes, including NLRP1, NLRP2, NLRP3, NLRC4, AIM2, and Pyrin. Intracellularly, IC 100 binds to ASC monomers, inhibiting inflammasome formation, thereby blocking activation of IL-1β early in the inflammatory cascade. IC 100 also binds to ASC in ASC Specks, both intracellularly and extracellularly, further blocking activation of IL-1β and the perpetuation of the inflammatory response that is pathogenic in inflammatory diseases. Because active cytokines amplify adaptive immunity through various mechanisms, IC 100, by attenuating cytokine activation, also attenuates the adaptive immune response. The lead indication for IC 100 is obesity and its associated metabolic complications. To review a white paper summarizing the mechanism of action and preclinical data for IC 100, Click Here.
About ZyVersa Therapeutics, Inc.
ZyVersa (Nasdaq: ZVSA) is a clinical stage specialty biopharmaceutical company leveraging advanced proprietary technologies to develop first-in-class drugs for patients with inflammatory or kidney diseases with high unmet medical needs. We are well positioned in the rapidly emerging inflammasome space with a highly differentiated monoclonal antibody, Inflammasome ASC Inhibitor IC 100, and in kidney disease with phase 2 Cholesterol Efflux Mediator™ VAR 200. The lead indication for IC 100 is obesity and its associated metabolic complications, and for VAR 200, focal segmental glomerulosclerosis (FSGS). Each therapeutic area offers a “pipeline within a product,” with potential for numerous indications. The total accessible market is over $100 billion. For more information, please visit www.zyversa.com.
Certain statements contained in this press release regarding matters that are not historical facts, are forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995. These include statements regarding management’s intentions, plans, beliefs, expectations, or forecasts for the future, and, therefore, you are cautioned not to place undue reliance on them. No forward-looking statement can be guaranteed, and actual results may differ materially from those projected. ZyVersa Therapeutics, Inc (“ZyVersa”) uses words such as “anticipates,” “believes,” “plans,” “expects,” “projects,” “future,” “intends,” “may,” “will,” “should,” “could,” “estimates,” “predicts,” “potential,” “continue,” “guidance,” and similar expressions to identify these forward-looking statements that are intended to be covered by the safe-harbor provisions. Such forward-looking statements are based on ZyVersa’s expectations and involve risks and uncertainties; consequently, actual results may differ materially from those expressed or implied in the statements due to a number of factors, including ZyVersa’s plans to develop and commercialize its product candidates, the timing of initiation of ZyVersa’s planned preclinical and clinical trials; the timing of the availability of data from ZyVersa’s preclinical and clinical trials; the timing of any planned investigational new drug application or new drug application; ZyVersa’s plans to research, develop, and commercialize its current and future product candidates; the clinical utility, potential benefits and market acceptance of ZyVersa’s product candidates; ZyVersa’s commercialization, marketing and manufacturing capabilities and strategy; ZyVersa’s ability to protect its intellectual property position; and ZyVersa’s estimates regarding future revenue, expenses, capital requirements and need for additional financing.
New factors emerge from time-to-time, and it is not possible for ZyVersa to predict all such factors, nor can ZyVersa assess the impact of each such factor on the business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. Forward-looking statements included in this press release are based on information available to ZyVersa as of the date of this press release. ZyVersa disclaims any obligation to update such forward-looking statements to reflect events or circumstances after the date of this press release, except as required by applicable law.
This press release does not constitute an offer to sell, or the solicitation of an offer to buy, any securities.
Corporate, Media, and IR Contact: Karen Cashmere Chief Commercial Officer kcashmere@zyversa.com 786-251-9641
NDA based on two Phase 3 studies of TNX-102 SL in fibromyalgia with statistically significant results on the primary endpoint of reducing widespread pain; generally well tolerated
TNX-102 SL is a non-opioid, centrally acting analgesic, granted Fast Track designation by FDA
Fibromyalgia affects more than 10 million adults in the U.S. who are mostly women
If approved by FDA, TNX-102 SL would be the first member of a new class of analgesic drugs for fibromyalgia and the first new drug for treating fibromyalgia in more than 15 years
CHATHAM, N.J., Oct. 16, 2024 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for TNX-102 SL (cyclobenzaprine HCl sublingual tablets) 5.6 mg, a non-opioid, centrally-acting analgesic that showed statistically significant reduction in the chronic, widespread pain associated with fibromyalgia in two Phase 3 studies and was generally well tolerated. TNX-102 SL was granted Fast Track designation for fibromyalgia by the FDA in July of 2024. Fast Track is designed to expedite FDA review of important new drugs to treat serious conditions and fill an unmet medical need.
“With the submission of this NDA, Tonix has achieved a critical milestone in potentially bringing a new first-line treatment option to the large and dissatisfied fibromyalgia population that has not had a new pharmacotherapy in over 15 years,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “TNX-102 SL would be the first member of a new class of medicines for treating fibromyalgia. TNX-102 SL was designed and developed as a bedtime treatment to be taken daily on a chronic basis. Tonix believes bedtime TNX-102 SL relieves fibromyalgia pain by targeting the non-restorative sleep that is characteristic of fibromyalgia.”
The NDA submission is supported by data from two 14-week double-blind, randomized, placebo-controlled Phase 3 clinical trials evaluating the safety and efficacy of TNX-102 SL 5.6 mg as a bedtime treatment for fibromyalgia. The prior Phase 3 RELIEF trial of TNX-102 SL in fibromyalgia, completed in December 2020, met its pre-specified primary endpoint of significantly reducing daily pain compared to placebo (p=0.010). In the confirmatory Phase 3 RESILIENT study in fibromyalgia, completed in December 2023, TNX-102 SL met the pre-specified primary endpoint of significantly reducing daily pain compared to placebo (p =0.00005). In both trials, TNX-102 SL was generally well tolerated with an adverse event profile comparable to prior studies and with no new safety signals observed. In both pivotal studies, the most common treatment-emergent adverse event was tongue or mouth numbness at the administration site, which was temporally related to dosing, self-limited, never rated as severe, and rarely led to study discontinuation (one participant in each study). Therefore, Tonix believes the submitted dossier contains the requisite safety and efficacy data from two adequate and well-controlled studies to support NDA submission.
“Despite three FDA-approved medications, representing two different classes of medicines, there remains a need for new treatment options for fibromyalgia patients,” commented Gregory Sullivan, M.D., Chief Medical Officer of Tonix Pharmaceuticals. “If approved by FDA, TNX-102 SL would be the first of a new tricyclic class of medicines for treating fibromyalgia. The existing FDA-approved drugs for fibromyalgia include the gabapentinoid class, represented by Pfizer’s Lyrica® (pregabalin) approved in 2008, and the SNRI class, represented by Lilly’s Cymbalta® (duloxetine) and AbbVie’s Savella® (milnacipran) approved in 2007 and 2009, respectively. The TNX-102 SL tablet is based on a proprietary eutectic formulation of cyclobenzaprine HCl and mannitol that provides a stable product which dissolves rapidly and efficiently delivers cyclobenzaprine by the transmucosal route into the bloodstream. I would like to thank all the participants in our clinical trials, as well as the trial investigators and staff, who worked together over many years to help make this important milestone possible.”
The FDA typically has a 60-day filing review period to determine whether the submitted NDA is complete and accepted for review. If FDA accepts the NDA for review, the Company expects a 2025 date for an FDA decision on approval, based on the Prescription Drug User Fee Act (PDUFA).
About Fibromyalgia
Fibromyalgia is a common chronic pain disorder that is understood to result from amplified sensory and pain signaling within the central nervous system, called central sensitization. Brain imaging studies have localized the functional disorder to the brain’s insular and anterior cingulate cortex. Fibromyalgia afflicts more than 10 million adults in the U.S., the majority of whom are women. Symptoms of fibromyalgia include chronic widespread pain, non-restorative sleep, fatigue, and brain fog (or cognitive dysfunction). Other associated symptoms include mood disturbances, including depression, anxiety, headaches, and abdominal pain or cramps. Individuals suffering from fibromyalgia often struggle with their daily activities, have impaired quality of life, and frequently are disabled. Physicians and patients report common dissatisfaction with currently marketed products. Fibromyalgia is now recognized as the prototypic nociplastic syndrome. Nociplastic pain is the third primary type of pain in addition to nociceptive pain and neuropathic pain. Many patients present with pain syndromes that are a spectrum of mixtures of the three primary types of pain. Nociplastic syndromes are associated with central and peripheral sensitization. Fibromyalgia can occur without any identifiable precipitating event. However, many fibromyalgia cases follow one or more precipitating event(s) including: chronic nociceptive or neuropathic pain states; recovery from an infectious illness; a cancer diagnosis or cancer treatment; a metabolic or endocrine stress; or a traumatic event. In the cases of recovery from an infectious illness, fibromyalgia is considered an Infection-Associated Chronic Condition. In addition to fibromyalgia cases associated with other conditions or stressors, the U.S. National Academies of Sciences, Engineering, and Medicine, has concluded that fibromyalgia is a diagnosable condition that occurs after recovery from COVID-19 in the context of Long COVID. Fibromyalgia is also recognized as a Chronic Overlapping Pain Condition, due to shared symptoms with chronic fatigue syndrome/myalgic encephalomyelitis (CFS/ME), irritable bowel syndrome, endometriosis, low back pain, post-concussive syndrome (also known as mild traumatic brain injury), chronic Lyme Disease, chronic diabetic neuropathy and chronic post-herpetic neuralgia.
About TNX-102 SL
TNX-102 SL is a centrally acting, non-opioid bedtime investigational drug, designed for chronic use. The tablet is a patented sublingual formulation of cyclobenzaprine hydrochloride developed for bedtime dosing for the management of fibromyalgia. Cyclobenzaprine interacts as an antagonist at four different receptors in the brain: serotonergic-5-HT2A, adrenergic-α1, histaminergic-H1, and muscarinic-M1-cholinergic receptors. Together, these interactions are believed to target the non-restorative sleep characteristic of fibromyalgia that was identified by Professor Harvey Moldofsky in 1975. Cyclobenzaprine is not associated with risk of addiction or dependence. The TNX-102 SL tablet is based on a eutectic formation of cyclobenzaprine HCl and mannitol that provides a stable product which dissolves rapidly and delivers cyclobenzaprine by the transmucosal route efficiently into the bloodstream. The eutectic protects cyclobenzaprine HCl from interacting with the basifying agent that is also part of the formulation and required for efficient transmucosal absorption. Patents based on TNX-102 SL’s eutectic composition and its properties have issued in the U.S., E.U., Japan, China and many other jurisdictions around the world and provide market protection into 2034. The European Patent Office’s Opposition Division maintained Tonix’s European Patent EP 2 968 992 in unamended form after an Opposition was filed against it by a Sandoz subsidiary, Hexal AG. Hexal AG did not appeal that decision. The formulation of TNX-102 SL was designed specifically for sublingual administration and transmucosal absorption for bedtime dosing to target disturbed sleep, while reducing the risk of daytime somnolence. Clinical pharmacokinetic studies indicated that the addition of a basifying agent was necessary for efficient transmucosal absorption which results in higher levels of exposure during the first 2 hours after dosing and in deceased levels of the long-lived active metabolite, norcyclobenzaprine in both single dose and multiple dose studies, consistent with bypassing first pass hepatic metabolism. At steady state after 20 days of dosing TNX-102 SL, the dynamic peak level of cyclobenzaprine is higher than the background level of norcyclobenzaprine. In contrast, after 20 days of dosing oral cyclobenzaprine, the simulated peak level of cyclobenzaprine is lower than the simulated background level of norcyclobenzaprine.
Tonix Pharmaceuticals Holding Corp.*
Tonix is a fully integrated biopharmaceutical company focused on transforming therapies for pain management and modernizing solutions for public health challenges. Tonix’s development portfolio is focused on central nervous system (CNS) disorders, and its priority is to progress TNX-102 SL, a product candidate for which an NDA was submitted based on two statistically significant Phase 3 studies for the management of fibromyalgia. The FDA has granted Fast Track designation to TNX-102 SL for the management of fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction and acute stress disorder under a Physician-Initiated IND at the University of North Carolina in the OASIS study funded by the U.S. Department of Defense (DoD). Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase), a biologic in Phase 2 development designed to treat cocaine intoxication that has FDA Breakthrough Therapy designation and its development is supported by a grant from the U.S. National Institude of Drug Abuse and Addiction. Tonix’s immunology development portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is an Fc-modified 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, including TNX-2900 for Prader-Willi syndrome, and infectious disease, including a vaccine for mpox, TNX-801. Tonix recently announced a contract with the U.S. DoD’s Defense Threat Reduction Agency (DTRA) for up to $34 million over five years to develop TNX-4200, small molecule broad-spectrum antiviral agents targeting CD45 for the prevention or treatment of infections to improve the medical readiness of military personnel in biological threat environments. Tonix owns and operates a state-of-the art infectious disease research facility in Frederick, MD. 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; 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. 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.
Lead indication for ZyVersa’s Inflammasome ASC Inhibitor IC 100 is obesity with metabolic complications.
WESTON, Fla., Oct. 15, 2024 (GLOBE NEWSWIRE) — ZyVersa Therapeutics, Inc. (Nasdaq: ZVSA, or “ZyVersa”), a clinical stage specialty biopharmaceutical company developing first-in-class drugs for treatment of inflammatory and renal diseases, announces that Stephen C. Glover, Co-Founder, Chairman, Chief Executive Officer, and President, was interviewed on the Big Biz Show to address current trends in the obesity drug market and the potential role of inflammasome inhibitors.
Key Points
Obesity is an international health crisis, with over 50% of the world’s population predicted to be overweight or obese within the next ten years.
Launch of incretin therapy (GLP-1 agonists) for obesity has resulted in substantial and unsurpassed weight loss, stimulating a drug revolution that has led to a 10-fold increase in venture dollars flowing into the category.
High levels of investment in the obesity category are expected to continue due to the trend toward development of add-on therapies to GLP-1 agonists to preserve muscle during weight loss and to attenuate obesity-related inflammation that leads to chronic complications such as cardiovascular disease, type 2 diabetes, and even neurodegenerative diseases like Parkinson’s and Alzheimer’s disease. This is reflected in the billion-dollar acquisitions recently made by Novo and Lilly, and Sanofi’s $27 Million strategic investment in Ventyx’s NLRP3 inhibitor to address the complications of obesity.
ZyVersa’s Inflammasome ASC Inhibitor IC 100 is being developed for use in combination with GLP-1 agonists to control obesity-associated inflammation, attenuate the associated complications, and enhance weight loss. The company is working with renowned experts in the field who are providing guidance to the development program.
About Inflammasome ASC Inhibitor IC 100
IC 100 is a novel humanized IgG4 monoclonal antibody that inhibits the inflammasome adaptor protein ASC. IC 100 was designed to attenuate both initiation and perpetuation of the inflammatory response. It does so by binding to a specific region of the ASC component of multiple types of inflammasomes, including NLRP1, NLRP2, NLRP3, NLRC4, AIM2, and Pyrin. Intracellularly, IC 100 binds to ASC monomers, inhibiting inflammasome formation, thereby blocking activation of IL-1β early in the inflammatory cascade. IC 100 also binds to ASC in ASC Specks, both intracellularly and extracellularly, further blocking activation of IL-1β and the perpetuation of the inflammatory response that is pathogenic in inflammatory diseases. Because active cytokines amplify adaptive immunity through various mechanisms, IC 100, by attenuating cytokine activation, also attenuates the adaptive immune response. The lead indication for IC 100 is obesity and its associated metabolic complications. To review a white paper summarizing the mechanism of action and preclinical data for IC 100, Click Here.
About ZyVersa Therapeutics, Inc.
ZyVersa (Nasdaq: ZVSA) is a clinical stage specialty biopharmaceutical company leveraging advanced proprietary technologies to develop first-in-class drugs for patients with inflammatory or kidney diseases with high unmet medical needs. We are well positioned in the rapidly emerging inflammasome space with a highly differentiated monoclonal antibody, Inflammasome ASC Inhibitor IC 100, and in kidney disease with phase 2 Cholesterol Efflux MediatorTM VAR 200. The lead indication for IC 100 is obesity and its associated metabolic complications, and for VAR 200, focal segmental glomerulosclerosis (FSGS). Each therapeutic area offers a “pipeline within a product,” with potential for numerous indications. The total accessible market is over $100 billion. For more information, please visit www.zyversa.com.
Certain statements contained in this press release regarding matters that are not historical facts, are forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995. These include statements regarding management’s intentions, plans, beliefs, expectations, or forecasts for the future, and, therefore, you are cautioned not to place undue reliance on them. No forward-looking statement can be guaranteed, and actual results may differ materially from those projected. ZyVersa Therapeutics, Inc (“ZyVersa”) uses words such as “anticipates,” “believes,” “plans,” “expects,” “projects,” “future,” “intends,” “may,” “will,” “should,” “could,” “estimates,” “predicts,” “potential,” “continue,” “guidance,” and similar expressions to identify these forward-looking statements that are intended to be covered by the safe-harbor provisions. Such forward-looking statements are based on ZyVersa’s expectations and involve risks and uncertainties; consequently, actual results may differ materially from those expressed or implied in the statements due to a number of factors, including ZyVersa’s plans to develop and commercialize its product candidates, the timing of initiation of ZyVersa’s planned preclinical and clinical trials; the timing of the availability of data from ZyVersa’s preclinical and clinical trials; the timing of any planned investigational new drug application or new drug application; ZyVersa’s plans to research, develop, and commercialize its current and future product candidates; the clinical utility, potential benefits and market acceptance of ZyVersa’s product candidates; ZyVersa’s commercialization, marketing and manufacturing capabilities and strategy; ZyVersa’s ability to protect its intellectual property position; and ZyVersa’s estimates regarding future revenue, expenses, capital requirements and need for additional financing.
New factors emerge from time-to-time, and it is not possible for ZyVersa to predict all such factors, nor can ZyVersa assess the impact of each such factor on the business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. Forward-looking statements included in this press release are based on information available to ZyVersa as of the date of this press release. ZyVersa disclaims any obligation to update such forward-looking statements to reflect events or circumstances after the date of this press release, except as required by applicable law.
This press release does not constitute an offer to sell, or the solicitation of an offer to buy, any securities.
Corporate, Media, and IR Contact: Karen Cashmere Chief Commercial Officer kcashmere@zyversa.com 786-251-9641
MAIA seeks to advance two new THIO-derived telomere-targeting molecules to clinical trials
CHICAGO–(BUSINESS WIRE)– MAIA Biotechnology, Inc., (NYSE American: MAIA) (“MAIA”, the “Company”), a clinical-stage biopharmaceutical company developing targeted immunotherapies for cancer, today announced that an abstract related to the company’s second generation of proprietary telomere-targeting THIO prodrugs has been accepted for poster presentation during the upcoming 36th EORTC-NCI-AACR Symposium on Molecular Targets and Cancer Therapeutics, taking place October 23-25, 2024, in Barcelona, Spain. The Symposium is hosted by the European Organisation for Research and Treatment of Cancer (EORTC), the National Cancer Institute (NCI) and the American Association for Cancer Research (AACR).
MAIA will present the results of preclinical studies for its new THIO-based prodrugs, designated as MAIA-2021-20 and MAIA-2022-12, which are lipid-conjugated compounds derived from THIO. These second-generation compounds belong to a new chemical class of molecules called telomere targeting divalent dinucleotides. These preclinical studies were designed to evaluate the efficacy of the new molecules alone and in combination with or without immune checkpoint-blocking antibodies (anti-PD-L1).
MAIA’s Chief Scientific Officer Sergei Gryaznov, Ph.D. commented, “Our presentation will demonstrate that treatment with each of the prodrugs studied was highly efficacious and was shown to overcome immunotherapy resistance. Our results have shown marked activity in advanced tumors and resulted in tumor growth inhibition in preclinical models of lung cancer, colorectal carcinoma, melanoma and hepatocellular carcinoma. We are actively working to advance these candidates toward clinical development.”
Presentation details:
Title:
New Dual-Pharmacophore Dinucleotide Prodrugs as Potent Telomere Targeting Anticancer Molecules
Abstract number:
ENA24-0044
Session title:
New Drugs
Date and time:
Wednesday, October 23, 2024, at noon CEST
Presenting author:
Ilgen Mender, Ph.D., Director of Biology Research, MAIA Biotechnology
MAIA has designed and evaluated more than 80 THIO-like compounds to date and has submitted three U.S. patent applications for its second-generation telomere targeting agents.
About THIO
THIO (6-thio-dG or 6-thio-2’-deoxyguanosine) is a first-in-class investigational telomere-targeting agent currently in clinical development to evaluate its activity in Non-Small Cell Lung Cancer (NSCLC). Telomeres, along with the enzyme telomerase, play a fundamental role in the survival of cancer cells and their resistance to current therapies. The modified nucleotide 6-thio-2’-deoxyguanosine (THIO) induces telomerase-dependent telomeric DNA modification, DNA damage responses, and selective cancer cell death. THIO-damaged telomeric fragments accumulate in cytosolic micronuclei and activates both innate (cGAS/STING) and adaptive (T-cell) immune responses. The sequential treatment with THIO followed by PD-(L)1 inhibitors resulted in profound and persistent tumor regression in advanced, in vivo cancer models by induction of cancer type–specific immune memory. THIO is presently developed as a second or later line of treatment for NSCLC for patients that have progressed beyond the standard-of-care regimen of existing checkpoint inhibitors.
About MAIA Biotechnology, Inc.
MAIA is a targeted therapy, immuno-oncology company focused on the development and commercialization of potential first-in-class drugs with novel mechanisms of action that are intended to meaningfully improve and extend the lives of people with cancer. Our lead program is THIO, a potential first-in-class cancer telomere targeting agent in clinical development for the treatment of NSCLC patients with telomerase-positive cancer cells. For more information, please visit www.maiabiotech.com.
Forward Looking Statements
MAIA cautions that all statements, other than statements of historical facts contained in this press release, are forward-looking statements. Forward-looking statements are subject to known and unknown risks, uncertainties, and other factors that may cause our or our industry’s actual results, levels or activity, performance or achievements to be materially different from those anticipated by such statements. The use of words such as “may,” “might,” “will,” “should,” “could,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify forward looking statements. However, the absence of these words does not mean that statements are not forward-looking. For example, all statements we make regarding (i) the initiation, timing, cost, progress and results of our preclinical and clinical studies and our research and development programs, (ii) our ability to advance product candidates into, and successfully complete, clinical studies, (iii) the timing or likelihood of regulatory filings and approvals, (iv) our ability to develop, manufacture and commercialize our product candidates and to improve the manufacturing process, (v) the rate and degree of market acceptance of our product candidates, (vi) the size and growth potential of the markets for our product candidates and our ability to serve those markets, and (vii) our expectations regarding our ability to obtain and maintain intellectual property protection for our product candidates, are forward looking. All forward-looking statements are based on current estimates, assumptions and expectations by our management that, although we believe to be reasonable, are inherently uncertain. Any forward-looking statement expressing an expectation or belief as to future events is expressed in good faith and believed to be reasonable at the time such forward-looking statement is made. However, these statements are not guarantees of future events and are subject to risks and uncertainties and other factors beyond our control that may cause actual results to differ materially from those expressed in any forward-looking statement. Any forward-looking statement speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. In this release, unless the context requires otherwise, “MAIA,” “Company,” “we,” “our,” and “us” refers to MAIA Biotechnology, Inc. and its subsidiaries.
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