Key Points: – Novartis has agreed to acquire Anthos Therapeutics for up to $3.1 billion, expanding its presence in the cardiovascular space. – Anthos’ lead drug candidate, abelacimab, has demonstrated significant potential in reducing bleeding risks compared to current anticoagulants. – The acquisition highlights the success of Blackstone Life Sciences’ investment strategy in building and scaling innovative biopharmaceutical companies.
Novartis has entered into a definitive agreement to acquire Anthos Therapeutics, a clinical-stage biopharmaceutical company specializing in innovative therapies for cardiometabolic diseases, for up to $3.1 billion. The deal, announced by Blackstone Life Sciences and Anthos, represents a major step forward in the development of abelacimab, a next-generation Factor XI inhibitor designed to prevent strokes and blood clots with superior safety benefits.
Anthos was founded in 2019 as a collaboration between Blackstone Life Sciences and Novartis, securing exclusive global rights from Novartis to develop, manufacture, and commercialize abelacimab. The acquisition reflects Novartis’ confidence in abelacimab’s potential to become a leader in the growing class of Factor XI anticoagulants, which aim to reduce the risk of major bleeding while maintaining strong stroke prevention efficacy.
“Abelacimab has the potential to be an important treatment option for the millions of patients globally with atrial fibrillation at high risk of stroke, and we could not have more conviction in the potential of this asset,” said Bill Meury, Chief Executive Officer of Anthos. “With its deep roots in the cardiovascular space, Novartis is especially well positioned to advance abelacimab’s clinical development and bring this innovative product to healthcare providers and patients.”
The drug has already demonstrated promising results in the AZALEA-TIMI 71 trial, where abelacimab showed a 62% reduction in major bleeding or clinically relevant non-major bleeding compared to rivaroxaban (Xarelto), a 67% reduction in major bleeding, and an 89% reduction in gastrointestinal bleeding. These impressive findings prompted the Independent Data Monitoring Committee to discontinue the study early due to clear clinical benefits. The results were recently published in the New England Journal of Medicine.
Anthos is currently conducting three phase 3 clinical trials for abelacimab: LILAC-TIMI 76 for patients with atrial fibrillation at high risk of stroke or systemic embolism, and ASTER and MAGNOLIA for patients with cancer-associated thrombosis. Data from these trials are expected in the second half of 2026, and Novartis is expected to continue these efforts to bring abelacimab to market.
Blackstone Life Sciences has played a crucial role in Anthos’ growth, investing in its development, assembling a world-class team, and designing the clinical plan. “This transaction is an affirmation of Blackstone Life Sciences’ ownership investment strategy, where we seek to find innovative products and build companies around them to meet unmet patient needs,” said Dr. Nicholas Galakatos, Global Head of Blackstone Life Sciences.
The acquisition deal includes an upfront payment of $925 million, with additional payments contingent on meeting regulatory and commercial milestones. The transaction is expected to close in the first half of 2025, pending regulatory approvals.
James J. Ferguson, MD, FACC, FAHA, joins as Chief Medical Officer
Extensive experience provides strong support for advancing specialized cardiovascular assets, including leading the late-stage clinical development of tecarfarin and other business development opportunities
PONTE VEDRA, Fla. – Cadrenal Therapeutics, Inc. (Nasdaq: CVKD), a late-stage biopharmaceutical company focused on the development of specialized cardiovascular therapies, with the late-stage asset tecarfarin, a new Vitamin K antagonist, today announced a leadership transition appointing James J. Ferguson, MD, FACC, FAHA, as its new Chief Medical Officer, effective immediately. Dr. Ferguson is a distinguished medical expert with over 25 years of leadership in the cardiovascular field and deep expertise in clinical development. Dr. Ferguson will lead the late-stage clinical development of tecarfarin to include the pivotal trial in LVAD patients and other indications in rare cardiovascular conditions requiring life-long anticoagulation therapy as well as other business development opportunities to build the Company’s pipeline.
Dr. Ferguson replaces Douglas W. Losordo, MD. Cadrenal thanks Dr. Douglas Losordo for his contributions to advancing the development of our tecarfarin program.
“We welcome Dr. Ferguson to our team and are confident that he will play a critical role in driving the late-stage clinical development of tecarfarin and the prioritization of indications. Dr. Ferguson brings expertise and strong relationships with cardiovascular clinical and scientific thought leadership, which will be instrumental as we prepare for late-stage clinical development of our tecarfarin program,” said Quang X. Pham, Chief Executive Officer, Cadrenal Therapeutics, Inc.
Dr. Ferguson joins Cadrenal after serving as Chief Medical Officer at Matinas BioPharma. Previously, Dr. Ferguson was Head of U.S. Cardiovascular Medical Affairs at Amgen and held several senior positions at AstraZeneca, including Vice President of US Cardiovascular Medical and Scientific External Relations, Therapeutic Area Vice President of Cardiovascular Global Medical Affairs, and US Development brand leader for BRILINTA.
“I’m truly honored to be joining Cadrenal at this exciting and transformative time. I look forward to advancing the late-stage clinical development of tecarfarin and bringing forward the first innovation in vitamin K-targeted anticoagulation in 70 years. This product could have a truly meaningful impact for patients in whom there continues to be major unmet medical needs with standard warfarin therapy,” said Dr. Ferguson.
About Cadrenal Therapeutics, Inc.
Cadrenal Therapeutics, Inc. is a late-stage biopharmaceutical company focused on developing specialized therapeutics for rare cardiovascular conditions. The Company is developing tecarfarin, a vitamin K antagonist (VKA) designed to be a better and safer anticoagulant than warfarin for individuals with implanted cardiac devices. Cadrenal strives to improve outcomes and reduce major adverse events for these patients. Although warfarin is widely used off-label for several rare cardiovascular diseases, extensive clinical and real-world data have shown it to have significant serious side effects. With its innovation, Cadrenal aims to meet the unmet needs of this patient population by relieving them and their healthcare providers of some of warfarin’s greatest clinical challenges.
Cadrenal is pursuing a product in a pipeline approach with tecarfarin. Tecarfarin received Orphan Drug designation (ODD) for advanced heart failure patients with implanted left ventricular assist devices (LVADs). In 2025, the Company plans to initiate a pivotal Phase 3 trial evaluating tecarfarin versus warfarin for LVAD patients. The Company also received ODD and fast-track status for tecarfarin in end-stage kidney disease and atrial fibrillation (ESKD+AFib).
Cadrenal is opportunistically pursuing business development initiatives with a longer-term focus to build a pipeline of specialized cardiovascular therapies. For more information, visit www.cadrenal.com and connect with us on LinkedIn.
Safe Harbor
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.” The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potentially,” “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. These statements include statements regarding the contributions to be made by Dr. Ferguson including leading the late-stage clinical development of tecarfarin to include the pivotal trial in LVAD patients and other indications in rare cardiovascular conditions requiring life-long anticoagulation therapy as well as other business development opportunities to build the Company’s pipeline, as well as advancing other indications in rare cardiovascular conditions requiring chronic anticoagulation; playing a critical role in driving the late-stage clinical development of tecarfarin and the prioritization of indications; Dr. Ferguson’s expertise and strong relationships with cardiovascular clinical and scientific thought leadership being instrumental as the Company prepares for the for late-stage clinical development of our tecarfarin programs; advancing the late-stage clinical development of tecarfarin and bringing forward the first innovation in vitamin K-targeted anticoagulation in 70 years; the product having a truly meaningful impact for patients in whom there continues to be major unmet medical needs with standard warfarin therapy; the Company striving to improve outcomes and reduce major adverse events for patients; the Company aiming to meet the unmet needs of the patient population by relieving them and their healthcare providers of some of warfarin’s greatest clinical challenges; and the Company initiating in 2025 a pivotal Phase 3 trial evaluating tecarfarin versus warfarin for LVAD patients. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including the ability to derive benefits from the contributions expected to be made by Dr. Ferguson; the ability to initiate the pivotal Phase 3 clinical trial for tecarfarin in LVAD patients in 2025 and provide improved outcomes; the ability to enter into collaborations with development partners; the ability of tecarfarin to provide a safer alternative to LVAD patients 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.
CHATHAM, N.J., Feb. 03, 2025 (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 will effect a 1-for-100 reverse stock split of its outstanding common stock. The reverse stock split will be effective for trading purposes as of the commencement of trading on February 5, 2025.
The reverse stock split is intended to increase the per share trading price of Tonix’s common stock to satisfy the $1.00 minimum bid price requirement for continued listing on The NASDAQ Capital Market (Rule 5550(a)(1)). Tonix’s common stock will continue to trade on the NASDAQ Capital Market under the symbol “TNXP” and under a new CUSIP number, 890260839. As a result of the reverse stock split, every one hundred pre-split shares of common stock outstanding will become one share of common stock. The reverse split will also apply to common stock issuable upon the exercise of Tonix’s outstanding warrants and stock options. The reverse stock split will not proportionately reduce the number of shares of authorized common stock, as permitted under Nevada law, as shareholder approval for the reverse stock split was obtained on October 30, 2024.
Tonix’s transfer agent, VStock Transfer LLC, which is also acting as the exchange agent for the reverse split, will provide instructions to shareholders regarding the process for exchanging share certificates. Any fractional shares of common stock resulting from the reverse stock split will be rounded up to the nearest whole post-split share and no shareholders will receive cash in lieu of fractional shares.
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 and for which a PDUFA (Prescription Drug User Fee act) goal date of August 15, 2025 has been assigned for a decision on marketing authorization. The FDA has previously 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 National Institute on Drug Abuse. 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 infectious disease, including a vaccine for mpox, TNX-801. In July 2024, Tonix 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.
Indication and Usage Zembrace® SymTouch® (sumatriptan succinate) injection (Zembrace) and Tosymra® (sumatriptan) nasal spray are prescription medicines used to treat acute migraine headaches with or without aura in adults who have been diagnosed with migraine. Zembrace and Tosymra are not used to prevent migraines. It is not known if Zembrace or Tosymra are safe and effective in children under 18 years of age.
Important Safety Information Zembrace and Tosymra can cause serious side effects, including heart attack and other heart problems, which may lead to death. Stop use and get emergency help if you have any signs of a heart attack:
discomfort in the center of your chest that lasts for more than a few minutes or goes away and comes back
severe tightness, pain, pressure, or heaviness in your chest, throat, neck, or jaw
pain or discomfort in your arms, back, neck, jaw or stomach
shortness of breath with or without chest discomfort
breaking out in a cold sweat
nausea or vomiting
feeling lightheaded
Zembrace and Tosymra are not for people with risk factors for heart disease (high blood pressure or cholesterol, smoking, overweight, diabetes, family history of heart disease) unless a heart exam shows no problem.
Do not use Zembrace or Tosymra if you have:
history of heart problems
narrowing of blood vessels to your legs, arms, stomach, or kidney (peripheral vascular disease)
uncontrolled high blood pressure
hemiplegic or basilar migraines. If you are not sure if you have these, ask your provider.
had a stroke, transient ischemic attacks (TIAs), or problems with blood circulation
severe liver problems
taken any of the following medicines in the last 24 hours: almotriptan, eletriptan, frovatriptan, naratriptan, rizatriptan, ergotamines, or dihydroergotamine. Ask your provider for a list of these medicines if you are not sure.
are taking certain antidepressants, known as monoamine oxidase (MAO)-A inhibitors or it has been 2 weeks or less since you stopped taking a MAO-A inhibitor. Ask your provider for a list of these medicines if you are not sure.
an allergy to sumatriptan or any of the components of Zembrace or Tosymra
Tell your provider about all of your medical conditions and medicines you take, including vitamins and supplements. Zembrace and Tosymra can cause dizziness, weakness, or drowsiness. If so, do not drive a car, use machinery, or do anything where you need to be alert. Zembrace and Tosymra may cause serious side effects including:
changes in color or sensation in your fingers and toes
sudden or severe stomach pain, stomach pain after meals, weight loss, nausea or vomiting, constipation or diarrhea, bloody diarrhea, fever
cramping and pain in your legs or hips; feeling of heaviness or tightness in your leg muscles; burning or aching pain in your feet or toes while resting; numbness, tingling, or weakness in your legs; cold feeling or color changes in one or both legs or feet
increased blood pressure including a sudden severe increase even if you have no history of high blood pressure
medication overuse headaches from using migraine medicine for 10 or more days each month. If your headaches get worse, call your provider.
serotonin syndrome, a rare but serious problem that can happen in people using Zembrace or Tosymra, especially when used with anti-depressant medicines called SSRIs or SNRIs. Call your provider right away if you have: mental changes such as seeing things that are not there (hallucinations), agitation, or coma; fast heartbeat; changes in blood pressure; high body temperature; tight muscles; or trouble walking.
hives (itchy bumps); swelling of your tongue, mouth, or throat
seizures even in people who have never had seizures before
The most common side effects of Zembrace and Tosymra include: pain and redness at injection site (Zembrace only); tingling or numbness in your fingers or toes; dizziness; warm, hot, burning feeling to your face (flushing); discomfort or stiffness in your neck; feeling weak, drowsy, or tired; application site (nasal) reactions (Tosymra only) and throat irritation (Tosymra only). Tell your provider if you have any side effect that bothers you or does not go away. These are not all the possible side effects of Zembrace and Tosymra. For more information, ask your provider. This is the most important information to know about Zembrace and Tosymra but is not comprehensive. For more information, talk to your provider and read the Patient Information and Instructions for Use. You can also visit https://www.tonixpharma.com or call 1-888-869-7633. You are encouraged to report adverse effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.
Key Points: – Novo Nordisk’s amycretin led to 22% average weight loss in a 36-week trial. – Shares rose 7.13%, marking the best single-day gain since March 2024. – Amycretin targets dual hormones to tackle hunger and appetite, showcasing groundbreaking innovation.
Novo Nordisk shares surged Friday after the pharmaceutical giant announced promising early-stage trial results for amycretin, a groundbreaking weight-loss drug administered through a once-weekly injection. The Danish company revealed that the treatment led to an average weight reduction of 22% in overweight and obese patients over a 36-week trial, marking a significant advancement in the fight against obesity. This compares to a 2% weight gain observed in patients receiving a placebo, showcasing the drug’s potential to reshape the treatment landscape for weight management.
The trial involved 125 participants and highlighted amycretin’s innovative mechanism of action. The drug targets GLP-1, a gut hormone that regulates appetite, and amylin, a hormone produced by the pancreas that influences hunger. This dual-action approach is a step forward from Novo Nordisk’s flagship products, Wegovy, which mimics GLP-1, and Ozempic, its well-known diabetes treatment. Amycretin’s ability to address weight loss through multiple pathways underscores its potential to provide life-changing results for patients struggling with obesity.
Novo Nordisk’s stock rose by 7.13% on Friday, reaching its best single-day performance since March 2024. The initial gains peaked at nearly 14% before settling, reflecting strong investor confidence in the company’s ability to expand its market dominance in obesity therapeutics. Fellow Danish drugmaker Zealand Pharma also benefited from the announcement, with shares climbing 4.7%, while rival Eli Lilly, the maker of obesity drug Zepbound, saw a slight dip in premarket trading.
The pharmaceutical industry has been increasingly focused on developing more effective weight-loss solutions, with obesity affecting millions worldwide and posing significant health risks. Novo Nordisk’s continued innovation in this space has made it a frontrunner, and the results from the amycretin trial further solidify its position. The company is already exploring oral formulations of the drug, which, in a separate early-stage trial announced last September, demonstrated a 13.1% weight reduction over 12 weeks.
Safety and tolerability are key considerations for any obesity treatment, and amycretin appears to meet these benchmarks. The most common side effects observed during the trial were gastrointestinal issues, but most were mild to moderate in severity. These findings align with patient tolerability seen in previous trials for similar drugs, making amycretin a promising addition to Novo Nordisk’s portfolio.
Martin Lange, executive vice president for development at Novo Nordisk, expressed optimism about the trial results. “We are very encouraged by the subcutaneous phase 1b/2a results for amycretin in people living with overweight or obesity,” Lange said in a statement. “The results seen in the trial support the weight-lowering potential of this novel unimolecular GLP-1 and amylin receptor agonist.”
As Novo Nordisk invests further in amycretin, the drug has the potential to transform the obesity treatment market, which is projected to grow substantially in the coming years. The company’s strategic focus on innovative, science-driven solutions positions it to maintain a competitive edge while addressing a critical global health challenge.
Key Points: – Beta Bionics’ iLet Bionic Pancreas is the first FDA-approved device to autonomously determine insulin doses using adaptive algorithms. – The company plans to raise $112.5 million by offering 7.5 million shares at a price range of $14-$16, achieving a potential market cap of $577.35 million. – Beta Bionics is part of a surge in biotech IPOs, reflecting investor confidence in transformative medical technologies.
Beta Bionics, the California-based innovator behind the iLet Bionic Pancreas, disclosed plans for a $112.5 million IPO, marking a pivotal moment in healthcare technology. The IPO terms, filed on January 22, 2025, outline the offering of 7.5 million shares priced between $14.00 and $16.00. At the midpoint of $15.00 per share, the company would achieve a market cap of approximately $577.35 million. Trading is set to commence on January 30, 2025, under the proposed ticker symbol “BBNX” on the NASDAQ.
The iLet Bionic Pancreas represents a groundbreaking advancement in diabetes management, being the first FDA-approved insulin delivery device to use adaptive closed-loop algorithms. This innovation enables the device to autonomously determine every insulin dose without requiring users to count carbohydrates, offering a significant improvement in the quality of life for individuals with Type 1 diabetes (T1D).
T1D affects approximately 1.8 million people in the U.S., all of whom rely on daily insulin replacement. The iLet system, cleared by the FDA for patients aged six and older in May 2023, targets this market with a vision to transform diabetes care. Despite its groundbreaking potential, Beta Bionics is currently unprofitable, reporting a net loss of $55.4 million on $53.1 million in revenue for the 12 months ending September 30, 2024.
The IPO, led by BofA Securities, Piper Sandler, Leerink, and Stifel, will provide the funding necessary for Beta Bionics to expand commercialization efforts and further develop its innovative technology. This initiative positions the company at the forefront of the intersection between healthcare and technology, emphasizing the growing demand for automated and personalized solutions in chronic disease management.
Beta Bionics’ IPO is part of a broader trend highlighting the growing prominence of biotech companies in public markets. With the rapid advancements in medical technology and increasing regulatory approvals, the biotech sector has emerged as a key driver of innovation. Biotech IPOs have gained momentum, reflecting strong investor interest in companies addressing critical healthcare needs with cutting-edge solutions.
In particular, biotech firms are increasingly leveraging public funding to accelerate the development and distribution of transformative therapies and devices. The promise of addressing unmet medical needs, coupled with advancements in artificial intelligence and biotechnology, has fueled optimism in the sector. Companies like Beta Bionics exemplify how public markets can empower medical innovation to scale, potentially improving millions of lives.
Investors are drawn to biotech IPOs not only for their market potential but also for their societal impact, as these companies strive to tackle some of the world’s most pressing healthcare challenges. Beta Bionics’ iLet device is a prime example of this trend, offering a glimpse into the future of automated, patient-centric care.
FDA is expected to assign a Prescription Drug User Fee Act (PDUFA) target action date and announce whether Priority Review has been granted in the Day 74 Letter
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
TNX-102 SL has the potential to 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, if approved by FDA
NDA based on two statistically significant Phase 3 studies of TNX-102 SL for the management of fibromyalgia; in which TNX-102 SL was generally well tolerated
CHATHAM, N.J., Dec. 17, 2024 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), today announced that the U.S. Food and Drug Administration (FDA) has accepted the filing of its New Drug Application (NDA) for TNX-102 SL (cyclobenzaprine HCl sublingual tablets), a 5.6 mg, non-opioid, centrally-acting analgesic, for the management of fibromyalgia. The FDA is expected to assign the NDA a Prescription Drug User Fee Act (PDUFA) target action date in a Day 74 Letter. At that time, the FDA will also communicate to Tonix whether Priority Review has been granted. 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.
“The FDA’s acceptance of our NDA represents another step forward as we pursue our goal of delivering the first member of a new class of medicines for the management of fibromyalgia, a condition affecting over 10 million adults in the U.S.,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “The fibromyalgia community, comprised of patients and their families and support groups, has been waiting for a new drug for over 15 years. Analysis of insurance claims in the U.S., commissioned by Tonix, have shown that 18 months after diagnosis, fibromyalgia patients were more likely to be prescribed addictive opioids than all three of the FDA-approved drugs combined.”
Dr. Lederman continued, “We look forward to working closely with the FDA throughout the NDA review period with the goal of bringing TNX-102 SL to the market to address the significant unmet needs of the fibromyalgia community as quickly as possible. Furthermore, this is an important milestone as we advance our commercial preparations in anticipation of a potential approval in 2025 with an accomplished commercial leadership team already in place, supporting our marketed products 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.”
The NDA 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 as a bedtime treatment for fibromyalgia. The first Phase 3 trial, RELIEF, of TNX-102 SL 5.6 mg 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 again 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). Excluding COVID-19, systemic adverse events in each of the two studies was lower than 4.0%. Tonix believes the submitted dossier contains the requisite safety and efficacy data from two adequate and well-controlled studies to support NDA approval.
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 insula 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: post-operative pain, acute or 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 can occur after recovery from COVID-19 in the context of Long COVID. Fibromyalgia is also recognized as a Chronic Overlapping Pain Condition, which is a group of related conditions including, 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 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 potently binds and acts as an antagonist at four different post-synaptic neuroreceptor subtypes: 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 formulation 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 relative to oral cyclobenzaprine, TNX-102 SL 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 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. The FDA has accepted the NDA filing for TNX-102 SL for 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 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. In July 2024, Tonix 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.
Zembrace® SymTouch® (sumatriptan succinate) injection (Zembrace) and Tosymra® (sumatriptan) nasal spray are prescription medicines used to treat acute migraine headaches with or without aura in adults who have been diagnosed with migraine. Zembrace and Tosymra are not used to prevent migraines. It is not known if Zembrace or Tosymra are safe and effective in children under 18 years of age.
Important Safety Information
Zembrace and Tosymra can cause serious side effects, including heart attack and other heart problems, which may lead to death. Stop use and get emergency help if you have any signs of a heart attack:
discomfort in the center of your chest that lasts for more than a few minutes or goes away and comes back
severe tightness, pain, pressure, or heaviness in your chest, throat, neck, or jaw
pain or discomfort in your arms, back, neck, jaw or stomach
shortness of breath with or without chest discomfort
breaking out in a cold sweat
nausea or vomiting
feeling lightheaded
Zembrace and Tosymra are not for people with risk factors for heart disease (high blood pressure or cholesterol, smoking, overweight, diabetes, family history of heart disease) unless a heart exam shows no problem.
Do not use Zembrace or Tosymra if you have:
history of heart problems
narrowing of blood vessels to your legs, arms, stomach, or kidney (peripheral vascular disease)
uncontrolled high blood pressure
hemiplegic or basilar migraines. If you are not sure if you have these, ask your provider.
had a stroke, transient ischemic attacks (TIAs), or problems with blood circulation
severe liver problems
taken any of the following medicines in the last 24 hours: almotriptan, eletriptan, frovatriptan, naratriptan, rizatriptan, ergotamines, or dihydroergotamine. Ask your provider for a list of these medicines if you are not sure.
are taking certain antidepressants, known as monoamine oxidase (MAO)-A inhibitors or it has been 2 weeks or less since you stopped taking a MAO-A inhibitor. Ask your provider for a list of these medicines if you are not sure.
an allergy to sumatriptan or any of the components of Zembrace or Tosymra
Tell your provider about all of your medical conditions and medicines you take, including vitamins and supplements.
Zembrace and Tosymra can cause dizziness, weakness, or drowsiness. If so, do not drive a car, use machinery, or do anything where you need to be alert.
Zembrace and Tosymra may cause serious side effects including:
changes in color or sensation in your fingers and toes
sudden or severe stomach pain, stomach pain after meals, weight loss, nausea or vomiting, constipation or diarrhea, bloody diarrhea, fever
cramping and pain in your legs or hips; feeling of heaviness or tightness in your leg muscles; burning or aching pain in your feet or toes while resting; numbness, tingling, or weakness in your legs; cold feeling or color changes in one or both legs or feet
increased blood pressure including a sudden severe increase even if you have no history of high blood pressure
medication overuse headaches from using migraine medicine for 10 or more days each month. If your headaches get worse, call your provider.
serotonin syndrome, a rare but serious problem that can happen in people using Zembrace or Tosymra, especially when used with anti-depressant medicines called SSRIs or SNRIs. Call your provider right away if you have: mental changes such as seeing things that are not there (hallucinations), agitation, or coma; fast heartbeat; changes in blood pressure; high body temperature; tight muscles; or trouble walking.
hives (itchy bumps); swelling of your tongue, mouth, or throat
seizures even in people who have never had seizures before
The most common side effects of Zembrace and Tosymra include: pain and redness at injection site (Zembrance only); tingling or numbness in your fingers or toes; dizziness; warm, hot, burning feeling to your face (flushing); discomfort or stiffness in your neck; feeling weak, drowsy, or tired; application site (nasal) reactions (Tosymra only) and throat irritation (Tosymra only).
Tell your provider if you have any side effect that bothers you or does not go away. These are not all the possible side effects of Zembrace and Tosymra. For more information, ask your provider.
This is the most important information to know about Zembrace and Tosymra but is not comprehensive. For more information, talk to your provider and read the Patient Information and Instructions for Use. You can also visit https://www.tonixpharma.com or call 1-888-869-7633.
You are encouraged to report adverse effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
CHATHAM, N.J., Nov. 26, 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, announced today that Jessica Morris, Chief Operating Officer of Tonix Pharmaceuticals, will present and conduct investor meetings at NobleCon20, Noble Capital Markets’ Twentieth Annual Emerging Growth Equity Conference, being held December 3-4 in Boca Raton, Fla.
Investors interested in arranging a meeting with the Company’s management during the conference should contact the NobleCon conference coordinator. A video webcast of the presentation will be available the day following the presentation under the IR Events tab of the Tonix website at www.tonixpharma.com and as part of a complete catalog of presentations available at Noble Capital Markets’ Conference website at www.nobleconference.com and on Channelchek, the investor portal created by Noble Capital Markets, at www.channelchek.com.
Details of the Tonix Pharmaceuticals Presentation
Event:
NobleCon20, Noble Capital Markets’ Twentieth Annual Emerging Growth Equity Conference
Date:
Tuesday, December 3, 2024
Time:
12:00 p.m. ET
Location:
Florida Atlantic University, College of Business Executive Education (COBEE) Complex
Track:
Presentation Room 3
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.
Key Points: – Poseida Therapeutics to be acquired by Roche for up to $1.5 billion, advancing the field of allogeneic CAR-T therapies. – Poseida’s non-viral, TSCM-rich CAR-T therapies aim to enhance clinical outcomes and expand treatment access. – Roche’s capabilities in late-stage development and commercialization will bring Poseida’s advancements to patients worldwide.
Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage biopharmaceutical company pioneering allogeneic cell and genetic therapies, announced that it has entered into a definitive agreement to be acquired by Roche Holdings, Inc. in a transaction valued at up to $1.5 billion. Under the agreement, Poseida stockholders will receive $9.00 per share in cash upon closing, along with a contingent value right (CVR) for up to $4.00 per share upon achieving specified milestones. The acquisition has been unanimously approved by Poseida’s Board of Directors, which recommends that stockholders tender their shares.
The merger will bring Poseida’s advanced platform technologies and innovative therapies into Roche’s Pharmaceuticals Division. This acquisition marks a significant expansion of Roche’s capabilities in allogeneic cell therapy, a cutting-edge area of biotech innovation focused on creating “off-the-shelf” treatments. This collaboration builds on an ongoing strategic partnership between Poseida and Roche, particularly in developing CAR-T therapies for hematologic malignancies. The expanded scope will also include CAR-T programs targeting solid tumors and autoimmune diseases.
Poseida’s proprietary technology platform, which utilizes non-viral, T stem cell memory (TSCM)-rich CAR-T therapies, is at the forefront of innovation in cell therapy. TSCM cells are considered highly advantageous due to their long-lived, self-renewing, and multipotent properties, enabling potentially safer and more effective therapies. These characteristics differentiate Poseida’s therapies from other CAR-T approaches, which often rely on more differentiated cells with reduced regenerative capacity.
Highlighting Poseida’s potential, interim clinical data for P-BCMA-ALLO1, a CAR-T candidate for multiple myeloma, have shown compelling results. This reinforces the transformative potential of Poseida’s platform in addressing high unmet needs across various therapeutic areas. Kristin Yarema, Ph.D., President and CEO of Poseida Therapeutics, emphasized the importance of the acquisition: “Poseida has demonstrated the unique ability of its non-viral platform to deliver TSCM-rich CAR-T therapies with the potential to improve clinical outcomes and expand access. Joining Roche will allow us to accelerate development and bring our therapies to patients worldwide.”
The transaction terms include a tender offer for all outstanding Poseida shares, priced at $9.00 per share in cash at closing. The CVR, valued at up to $4.00 per share, will be contingent upon achieving specific clinical, regulatory, and commercial milestones. Roche plans to acquire any remaining shares not tendered during the initial offer through a second-step merger, ensuring full ownership of Poseida.
The deal is expected to close in the first quarter of 2025, subject to regulatory approvals and customary closing conditions. Centerview Partners LLC and Cooley LLP advised Poseida on the transaction, while Citi and Sidley Austin LLP served as advisors to Roche.
This acquisition represents a major milestone for Poseida and Roche alike. For Roche, it establishes a new core capability in allogeneic cell therapy and genetic medicine, areas that hold immense promise for the future of personalized healthcare. For Poseida, the merger offers an opportunity to leverage Roche’s global resources in late-stage development, commercialization, and market access, enabling its therapies to reach patients worldwide.
As the biotech industry increasingly focuses on transformative therapies, this deal underscores the growing importance of partnerships in accelerating innovation. With Poseida’s cutting-edge technology and Roche’s global expertise, the acquisition is poised to reshape the landscape of cell and genetic therapies, delivering groundbreaking treatments to patients across the globe.
Recognized as innovative biopharma developing a potentially safer and superior anticoagulant for patients with implanted cardiac devices and rare cardiovascular conditions
PONTE VEDRA, Fla., Nov. 19, 2024 — Cadrenal Therapeutics, Inc. (Nasdaq: CVKD), a late-stage biopharmaceutical company developing tecarfarin, a new vitamin K antagonist (VKA) anticoagulant, has been recognized as the 2024 “Anticoagulation Therapy Company of the Year” by Pharma Tech Outlook, an industry publication focused on breakthrough pharmaceutical technologies. The award underscores Cadrenal’s commitment to addressing the significant unmet needs of patients with implanted left ventricular assist devices (LVADs) and other rare cardiovascular conditions who require chronic anticoagulation. Cadrenal aims to develop a better VKA blood thinner for these warfarin-dependent patients.
In addition to receiving the award, Cadrenal was featured in the current edition of Pharma Tech Outlook. Entitled Pioneering Innovation in Anticoagulation for Rare Cardiovascular Conditions, the article highlights tecarfarin, Cadrenal’s lead candidate that is being developed to potentially overcome many of the challenges associated with warfarin anticoagulation therapy including drug-drug interactions and wide variability requiring frequent dosing changes. Unlike VKA warfarin, tecarfarin uses a unique metabolic pathway that is less affected by drug-drug interactions and kidney impairment. By providing a stable, once-daily, and reversible therapeutic, Cadrenal seeks to improve patient outcomes and ease the burden on patients and healthcare providers who face the complex anticoagulation management needs of this population.
“We are honored to receive this recognition from Pharma Tech Outlook,” said Quang X. Pham, founder and CEO of Cadrenal Therapeutics. “Our goal is to provide a safer and superior blood thinner option for patients who rely on chronic anticoagulation but are underserved by current treatments. Warfarin, the first VKA anticoagulant, was approved more than 70 years ago yet no significant advancements in oral VKA drugs have occurred since then.”
“Cadrenal Therapeutics has demonstrated a commitment to filling a serious gap in anticoagulation therapy,” said Lisa Winget, Managing Editor at Pharma Tech Outlook. “We are pleased to recognize Cadrenal’s innovative approach to developing a new treatment to serve patients with LVADs and other rare cardiovascular conditions.”
With orphan drug and fast-track designations, tecarfarin is uniquely positioned to fill the gap in chronic anticoagulation treatment options for patients with LVADs and rare cardiovascular conditions such as end-stage kidney disease (ESKD) with atrial fibrillation.
About Cadrenal Therapeutics, Inc. Cadrenal Therapeutics is a late-stage biopharmaceutical company developing tecarfarin, a new vitamin K antagonist (VKA) to provide potentially safer and superior chronic anticoagulation for patients with implanted cardiac devices or rare cardiovascular conditions. Cadrenal is focused on evaluating tecarfarin versus warfarin in reducing adverse events such as strokes, heart attacks, and bleeds in these patients. With tecarfarin, Cadrenal aims to address many of warfarin’s challenges such as drug interactions, frequent dosing adjustments, and kidney impairment effects, which are common in these patients. Tecarfarin has an orphan drug designation for left ventricular assist device (LVAD) patients and both orphan drug and fast-track designations for end-stage kidney disease patients with atrial fibrillation. Cadrenal is advancing a pivotal trial and pursuing clinical and commercial partnerships, with plans to study mechanical heart valve patients facing anticoagulation difficulties. Visit www.cadrenal.com to learn more.
Safe Harbor
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 Cadrenal’s anticoagulant being a potentially safer and superior anticoagulant for patients with implanted cardiac devices and rare cardiovascular conditions; Cadrenal developing a better VKA blood thinner for warfarin-dependent patients; Cadrenal’s lead candidate potentially overcoming many of the challenges associated with warfarin anticoagulation therapy including drug-drug interactions and wide variability requiring frequent dosing changes; Cadrenal improving patient outcomes and easing the burden on patients and healthcare providers who face the complex anticoagulation management needs of this population; Cadrenal providing a safer and superior blood thinner option for patients who rely on chronic anticoagulation but are underserved by current treatments; and tecarfarin being uniquely positioned to fill the gap in chronic anticoagulation treatment options for patients with LVADs and rare cardiovascular conditions such as end-stage kidney disease (ESKD) with atrial fibrillation; and advancing a pivotal trial and pursuing clinical and commercial partnerships, with plans to study mechanical heart valve patients facing anticoagulation difficulties. The words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potentially,” “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 be a safer and superior anticoagulant for patients with implanted cardiac devices and rare cardiovascular conditions and being a better VKA blood thinner for warfarin-dependent patients, the ability of the Company to advance tecarfarin in a pivotal trial and to advance tecarfarin with patients with LVADs and those with AFib and ESKD, the ability to advance commercial partnerships 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
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
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