Onconova Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer. The Company has proprietary targeted anti-cancer agents designed to disrupt specific cellular pathways that are important for cancer cell proliferation. Onconova’s novel, proprietary multi-kinase inhibitor narazaciclib (formerly ON 123300) is being evaluated in a combination trial with estrogen blockade in advanced endometrial cancer. Based on preclinical and clinical studies of CDK 4/6 inhibitors, Onconova is also evaluating opportunities for combination studies with narazaciclib in additional indications. Onconova’s product candidate rigosertib is being studied in multiple investigator-sponsored studies. These studies include a dose-escalation and expansion Phase 1/2a study of oral rigosertib in combination with nivolumab in patients with KRAS+ non-small cell lung cancer, a Phase 2 program evaluating rigosertib monotherapy in advanced squamous cell carcinoma complicating recessive dystrophic epidermolysis bullosa (RDEB-associated SCC), and a Phase 2 trial evaluating rigosertib in combination with pembrolizumab in patients with metastatic melanoma.
Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
Phase 1 Topline Data Shows Safety and Tolerability. Traws Pharma announced results from its Phase 1 trial testing ratutrelvir, its oral protease inhibitor for COVID-19. The trial was designed to determine pharmacokinetics and safety, with results showing consistent plasma levels within the expected range with no adverse events. A Phase 2a trial is being planned to begin in 1H2025.
Ratutrelvir Could Become A Category-Leading Drug. Ratutrelvir is an inhibitor of the SARS-CoV-2 Main protease (Mpro or 3CL protease). It has demonstrated in vitro activity against the original viral strain and numerous variants, including delta and omicron. Ratutrelvir does not require co-administration of ritonavir as a metabolic inhibitor and avoids the drug-drug interactions and potential side effects. In comparison, Pfizer’s Paxlovid is a combination of its Mpro inhibitor, nirmatrelvir, with ritonavir as a metabolic (cytochrome P450) inhibitor.
Equity Research is available at no cost to Registered users of Channelchek. Not a Member? Click ‘Join’ to join the Channelchek Community. There is no cost to register, and we never collect credit card information.
This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).
*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision.
Ratutrelvir was well-tolerated for 10 days and achieved consistent plasma levels in the predicted therapeutic window, without the need for co-administration of ritonavir
Phase 2a study expected to begin in H1 2025 in patients with COVID
Improving COVID care is an ongoing need, with approximately 50,000 US deaths in 2023
NEWTOWN, Pa., Sept. 30, 2024 (GLOBE NEWSWIRE) — Traws Pharma, Inc. (NASDAQ: TRAW) (“Traws Pharma”, “Traws” or “the Company”), a clinical-stage biopharmaceutical company developing potential oral small molecule therapies for the treatment of respiratory viral diseases, today announced positive topline Phase 1 results for its potential best-in-class COVID (SARS-CoV-2) candidate, ratutrelvir, an oral inhibitor of the Main protease (Mpro).
“Topline data indicate that administration of ratutrelvir, our product candidate for COVID, as monotherapy, for 10 days to healthy volunteers, showed no treatment related adverse events and demonstrated consistent plasma drug levels in the predicted therapeutic window. We are especially pleased by ratutrelvir’s ability to achieve plasma concentrations that are considerably above the EC90 against a comprehensive panel of SARS-CoV-2 viruses, without the need for ritonavir co-administration that can be a source of drug-drug interactions and potential severe side effects,” said Werner Cautreels, PhD, Chief Executive Officer of Traws Pharma. “These data provide us with further indication that ratutrelvir has the potential to be a potent, best-in-class, once-a-day, single-dose, 10-day antiviral therapy for COVID. This profile contrasts with Paxlovid™, an approved Mpro inhibitor, which requires co-administration of ritonavir, a metabolic inhibitor. We believe that ratutrelvir’s ritonavir-free regimen has the potential to reduce the burden of treatment, especially for patients with underlying medical conditions. Based on the Phase 1 data, we have selected the dose for our Phase 2a study, expected to begin in H1 2025.”
“As we near the fifth anniversary of the pandemic, it is notable that, despite the wide availability of approved antiviral therapies, COVID was a major cause of mortality in the US in 2023, with approximately 50,000 deaths1,” said Robert R. Redfield, MD, Chief Medical Officer for Traws Pharma and former Director of the U.S. Centers for Disease Control and Prevention (CDC). “We believe it is important for new COVID therapies to have a simple treatment regimen that can be used broadly, especially in patients who are at higher risk of serious symptoms or who are over 65 years of age with underlying medical conditions including heart disease, kidney disease, and chronic lung disease2. In addition, we consider activity against resistant viruses and low risk of clinical rebound to be important features of potential new COVID therapies. Our enthusiasm for ratutrelvir’s potential to meet the need for improved COVID treatment has been enhanced by the Phase 1 data.”
“Our goal for ratutrelvir is to effectively treat COVID, limit the symptoms of infection, and lower the risk for clinical rebound,” said C. David Pauza, PhD, Chief Scientific Officer for Traws Pharma. “Preclinical studies, presented at the annual International Conference on Antiviral Research in 2024 (ICAR2024), showed that ratutrelvir monotherapy has differentiated activity compared to nirmatrelvir against a range of drug-resistant viruses. Also, preclinical testing in animal models showed that levels of ratutrelvir in the lung were higher than in plasma. Phase 1 data show that ratutrelvir achieved consistent plasma levels in the predicted therapeutic window, with a pharmacokinetic profile that may reduce the likelihood of clinical rebound.”
Topline Phase 1 Results The Phase 1 trial (NCT06402136, conducted in Australia) was designed as a randomized, double-blind, placebo-controlled study to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of single- and multiple ascending doses (SAD and MAD) of ratutrelvir, administered as a capsule formulation in 56 healthy, COVID-negative, adult volunteers, randomized three-to-one (eight subjects per dosing group). The SAD portion of the study evaluated five ratutrelvir dose levels; the MAD segment evaluated two ratutrelvir dose levels, administered once daily for 10 days.
This first-in-human study showed no treatment related adverse events reported up to the highest dose. Topline data also showed that once-daily administration of ratutrelvir maintained plasma drug levels within the predicted therapeutic window. Preclinical studies, presented at the annual International Conference on Antiviral Research in 2024 (ICAR2024), showed that ratutrelvir demonstrated differentiated activity compared to nirmatrelvir against a range of SARS-CoV-2 variants, based on a comparison of EC50 values.
About Ratutrelvir Industry data indicate that COVID treatment represents a potential multi-billion dollar market opportunity. Ratutrelvir (also previously known as 83-0060 or TRX-01) was designed as an inhibitor of the SARS-CoV-2 Main protease (Mpro or 3CL protease). It has demonstrated in vitro activity against the original strain of the virus as well as the delta and omicron variants, and is more active than nirmatrelvir (Pfizer’s Mpro inhibitor, co-packaged with ritonavir as PAXLOVID™) in preclinical studies. Based on preclinical studies, ratutrelvir did not require co-administration with a metabolic inhibitor, such as ritonavir, which inhibits human cytochrome P450 (CYP) 3A4. Because of this, ratutrelvir is expected to avoid ritonavir-associated drug-drug interactions and potential resulting severe side effects, which may permit wider patient use. The drug candidate’s pharmacokinetic (PK) profile, including the ability to achieve plasma levels within the predicted therapeutic window, as demonstrated in the Phase 1 study, may also enable a once daily treatment regimen and reduce the likelihood of clinical rebound.
About Traws Pharma, Inc. Traws Pharma is a clinical stage biopharmaceutical company developing potential oral small molecule therapies for the treatment of respiratory viral diseases and cancer. The viral respiratory disease program includes two Phase 1 potentially best-in-class oral small molecules in development: tivoxavir marboxil, a novel oral antiviral drug candidate for influenza and avian flu, targeting the influenza cap-dependent endonuclease, and ratutrelvir, targeting Mpro (3CL protease) for COVID.
In the cancer program, Traws is utilizing a partnering strategy, supported by investigator sponsored studies, to advance two novel proprietary multi-kinase inhibitors, narazaciclib, targeting CDK4+, and rigosertib, targeting cell cycle proteins including PLK-1.
Traws is committed to delivering novel compounds for unmet medical needs using state-of-the-art drug development technology. With a focus on product safety and a commitment to patients in need or that are specifically vulnerable, we aim to build solutions for important medical challenges and alleviate the burden of viral infections and cancer.
Forward-Looking Statements
Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995, and involve risks and uncertainties including statements regarding the Company, its business and product candidates, including the potential opportunity, benefits and Traws regulatory plans for ratutrelvir. The Company has attempted to identify forward-looking statements by terminology including “believes”, “estimates”, “anticipates”, “expects”, “plans”, “intends”, “may”, “could”, “might”, “will”, “should”, “preliminary”, “encouraging”, “approximately” or other words that convey uncertainty of future events or outcomes. Although Traws believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including the success and timing of Traws’ clinical trials, collaborations, merger integration, market conditions and those discussed under the heading “Risk Factors” in Traws’ filings with the U.S. Securities and Exchange Commission (SEC). Any forward-looking statements contained in this release speak only as of its date. Traws undertakes no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events.
Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
Client Meetings Answered Questions About Tegoprubart. We held a Virtual Non-Deal Road Show with Eledon’s CEO, Dr. DA Gros. The discussions with clients covered tegoprubart history of development, mechanism of action, clinical trials, and its upcoming milestones. Some of the points raised and common questions are highlighted below.
The Phase 2 BESTOW Trial Completed Enrollment In August. Eledon announced the completion of enrollment for its Phase 2 BESTOW trial that tests tegoprubart against tacrolimus for prevention of kidney transplant rejection. The enrollment was completed ahead of the expected YE2024 timeframe due to higher than expected interest from the transplant surgeons.
Equity Research is available at no cost to Registered users of Channelchek. Not a Member? Click ‘Join’ to join the Channelchek Community. There is no cost to register, and we never collect credit card information.
This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).
*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision.
New patent expected to provide market exclusivity into 2036
Zembrace® SymTouch® (sumatriptan succinate injection) 10mg is indicated for the acute treatment of migraine in adults
CHATHAM, N.J., Sept. 27, 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 the United States Patent and Trademark Office issued U.S. Patent No. 12,097,183 to the Company on September 24, 2024. The patent, entitled “Pharmaceutical Composition for Treating Migraine”, claims 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 provide protection into 2036.
“We are excited to announce the issuance of this additional patent, providing additional protection for our exclusive marketing and sale of FDA-approved Zembrace® for the treatment of migraines,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “We believe Zembrace® is a compelling non-oral option for people who suffer with migraines.”
Tonix recently launched a new educational campaign, “Does Your Migraine Pill Work Every Time?” The goal of the campaign is to educate patients and their healthcare providers on the benefits of non-oral migraine medications including nasal and injectable treatment options. Non-oral migraine medications, such as injectables and nasal sprays, do not rely on the digestive system to be absorbed and can offer the potential for faster relief from migraine symptoms in as little as 10 minutes.
Migraine often requires patients to advocate for themselves to develop an effective migraine treatment plan. Empowering patients to understand why they are experiencing delayed or inconsistent relief from oral medications and educating them on other migraine treatment options could ultimately improve their management of migraine symptoms and ultimately enhance their quality of life.
For example, gastroparesis is common before, during, and sometimes in between migraine attacks. Gastroparesis can slow or even block the absorption of oral medications causing delayed, incomplete, or no migraine symptom relief. Tonix will launch a new disease education website, www.gpmigraine.com, for patients who want to learn more about gastroparesis and migraine and why their oral medications do not work.
Dr. Lederman continued, “Tonix is dedicated to educating patients and their healthcare providers on gastroparesis and how non-oral medicines including nasal and injectable medications can help patients manage their migraines. We hope to inspire patients to optimize their migraine treatment plan with non-oral medications.”
About Migraine
Nearly 40 million people in the United States suffer from migraine1 and it has been recognized as the second leading cause of disability in the world2,3. Migraine is characterized by debilitating attacks lasting four to 72 hours with multiple symptoms, including pulsating headaches of moderate to severe pain intensity often associated with nausea or vomiting, and/or sensitivity to sound (phonophobia) and sensitivity to light (photophobia)4.
1Law, H. Z., Chung, M. H., Nissan, G., Janis, J. E., & Amirlak, B. (2020). Hospital Burden of Migraine in United States Adults: A 15-year National Inpatient Sample Analysis. Plastic and reconstructive surgery. Global open, 8(4), e2790. https://doi.org/10.1097/GOX.0000000000002790
2GBD 2016 Headache Collaborators. Global, regional, and national burden of migraine and tension-type headache, 1990-2016: a systematic analysis for the Global Burden of Disease Study 2016. Lancet Neurol2018;17(11):954-976.
3Steiner, T.J., Stovner, L.J., Jensen, R. et al. Lifting the Burden: the Global Campaign against Headache. Migraine remains second among the world’s causes of disability, and first among young women: findings from GBD2019. J Headache Pain 21, 137 (2020).
4Headache Classification Committee of the International Headache Society (IHS). The international classification of headache disorders, 3rd edition. Cephalalgia. 2018;38(1):1–211.
Tonix Pharmaceuticals Holding Corp.*
Tonix is a fully integrated biopharmaceutical company focused on transforming therapies for pain management and modernizing solutions for public health challenges. Tonix’s development portfolio is focused on central nervous system (CNS) disorders, and its priority is to submit a New Drug Application (NDA) to the FDA in October 2024 for TNX-102 SL, a product candidate for which two statistically significant Phase 3 studies have been completed for the management of fibromyalgia. The FDA has granted Fast Track designation to TNX-102 SL for the management of fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction. Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase), a biologic in Phase 2 development designed to treat cocaine intoxication that has Breakthrough Therapy designation. Tonix’s immunology development portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft rejection and for the treatment of autoimmune diseases. Tonix also has product candidates in development in the areas of rare disease, including TNX-2900 for Prader-Willi syndrome, and infectious disease, including a vaccine for mpox, TNX-801. Tonix recently announced the U.S. Department of Defense (DoD), Defense Threat Reduction Agency (DTRA) awarded it a contract for up to $34 million over five years in an Other Transaction Agreement (OTA) 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, instrumental in progressing this development. Tonix Medicines, our commercial subsidiary, markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan nasal spray) 10 mg for the treatment of acute migraine with or without aura in adults.
*Tonix’s product development candidates are investigational new drugs or biologics and have not been approved for any indication.
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) and TOSYMRA® (sumatriptan spray) are indicated for the acute treatment of migraine with or without aura in adults. ZEMBRACE SymTouch and TOSYMRA should only be used where a clear diagnosis of migraine has been established. ZEMBRACE SymTouch and TOSYMRA are not indicated for the prevention of migraine attacks or for the treatment of cluster headache.
Important Safety Information CONTRAINDICATED IN PATIENTS WITH:
Ischemic coronary artery disease (CAD) or coronary artery vasospasm, including Prinzmetal’s angina
Wolff-Parkinson-White syndrome or arrhythmias associated with other cardiac accessory conduction pathway disorders
History of stroke, transient ischemic attack (TIA), or hemiplegic or basilar migraine
Peripheral vascular disease
Ischemic bowel disease
Uncontrolled hypertension
Recent (i.e., within 24 hours) use of ergotamine-containing or ergot-type medication, or another 5-HT1 agonist
Concurrent or recent (within 2 weeks) use of a MAO-A inhibitor
Hypersensitivity to sumatriptan (angioedema and anaphylaxis seen)
Severe hepatic impairment
WARNINGS AND PRECAUTIONS
Myocardial ischemia/infarction, Prinzmetal’s angina: These events may occur even in patients without known cardiovascular disease. Perform cardiac evaluation in triptan-naïve patients with multiple risk factors and, if satisfactory, administer first dose of ZEMBRACE SymTouch and TOSYMRA in a medically-supervised setting
Arrhythmias: Life-threatening disturbances of cardiac rhythm, including ventricular tachycardia and ventricular fibrillation leading to death, have been reported within a few hours following the administration of 5-HT1 agonists. Discontinue ZEMBRACE SymTouch and TOSYMRA if these disturbances occur
Sensations of chest/throat/neck/jaw pain, tightness, pressure, or heaviness: Commonly occur after treatment with 5-HT1 agonists and are usually non-cardiac in origin. Perform a cardiac evaluation in patients with cardiac risk
Cerebrovascular Events: Cerebral hemorrhage, subarachnoid hemorrhage, and stroke have occurred in patients treated with 5-HT1 agonists, and some have resulted in fatalities. Discontinue ZEMBRACE SymTouch and TOSYMRA if a cerebrovascular event occurs. Before treating headaches in patients not previously diagnosed as migraineurs, and in migraineurs who present with atypical symptoms, exclude other potentially serious neurological conditions
Other Vasospasm Reactions: 5-HT1 agonists, including ZEMBRACE SymTouch and TOSYMRA, may cause non-coronary vasospastic reactions, such as peripheral vascular ischemia, gastrointestinal vascular ischemia and infarction, splenic infarction, and Raynaud’s syndrome. In patients who experience symptoms or signs suggestive of a vasospastic reaction following the use of any 5-HT1 agonist, rule out a vasospastic reaction before using ZEMBRACE SymTouch and TOSYMRA
Medication Overuse Headache: Overuse of acute migraine drugs may lead to exacerbation headache (medication overuse head- ache). Detoxification of patients, including withdrawal of the overused drugs, and treatment of withdrawal symptoms may be necessary
Serotonin Syndrome: May occur with triptans, including ZEMBRACE SymTouch and TOSYMRA, particularly during co-administration with selective serotonin reuptake inhibitors (SSRIs), serotonin norepinephrine reuptake inhibitors (SNRIs), tricyclic antidepressants (TCAs), and monoamine oxidase inhibitors (MAOIs). The onset of symptoms usually occurs within minutes to hours of receiving a new or greater dose of a serotonergic medication. Discontinue ZEMBRACE SymTouch and TOSYMRA if serotonin syndrome is suspected
Increases in Blood Pressure: Significant elevation in blood pressure, including hypertensive crisis with acute impairment of organ systems, has been reported in patients treated with 5-HT1 agonists. Monitor blood pressure in patients treated with ZEMBRACE SymTouch and TOSYMRA
Hypersensitivity Reactions: Hypersensitivity reactions, including angioedema and anaphylaxis, have occurred in patients receiving sumatriptan. Such reactions can be life threatening or fatal. ZEMBRACE SymTouch and TOSYMRA are contraindicated in patients with a history of hypersensitivity reaction to sumatriptan
Seizures: Seizures have been reported following administration of sumatriptan, with or without predisposing factors. ZEMBRACE SymTouch and TOSYMRA should be used with caution in patients with a history of epilepsy or conditions associated with a lowered seizure threshold
Local Irritation (TOSYMRA only): Local irritative symptoms were reported in approximately 46% of patients with TOSYMRA in an open-label trial which allowed repeated use of TOSYMRA over the course of 6 months. The most common of which were application site reaction (eg., burning sensations in the nose), dysgeusia, and throat irritation. Approximately 0.5% of the cases were reported as severe.
ADVERSE REACTIONS
The most common adverse reactions (≥5% and > placebo) were injection site reactions (ZEMBRACE SymTouch only), tingling, dizziness/vertigo, warm/hot sensation, burning sensation, feeling of heaviness, pressure sensation, flushing, feeling of tightness, and numbness/paresthesia.
To report SUSPECTED ADVERSE REACTIONS, contact TONIX Medicines, Inc, at 1-888-869-7633 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
Please see the full Prescribing Information, including Instructions for Use, for ZEMBRACE SymTouch and TOSYMRA.
Joe Gomes, CFA, Managing Director, Equity Research Analyst, Generalist , Noble Capital Markets, Inc.
Joshua Zoepfel, Research Associate, Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
Update and Presentation. On Tuesday, we had the opportunity to participate in a webinar presentation and interview with MustGrow’s management providing an update on the current progress of the Company’s pipeline and its future. Key takeaways of the update, in our view, were the progress towards sales of its TerraSante product, advancing towards commercialization of its TerraMG product, and the expansion of the Company’s intellectual property portfolio.
TerraSante. The Company has been underway in its planning for larger-scale production on TerraSante. The Company had its initial commercial production run in 2023 and had its first sales during the third quarter this year. Management is seeing strong initial interest in the product, primarily from California, and has the opportunity for multiple applications per year for various crop markets, which could potentially triple acre coverage. We believe that we will continue to see more state approvals for the product in the near term as management has focused on getting such approvals.
Equity Research is available at no cost to Registered users of Channelchek. Not a Member? Click ‘Join’ to join the Channelchek Community. There is no cost to register, and we never collect credit card information.
This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).
*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision.
Tosymra® (sumatriptan nasal spray) 10mg is indicated and marketed for the acute treatment of migraine in adults
CHATHAM, N.J., Sept. 19, 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 the United States Patent and Trademark Office issued U.S. Patent No. 12,090,139 to the Company on September, 17, 2024. The patent, entitled “Formulations Comprising Triptan Compounds”, claims a pharmaceutical composition, a method of treating migraine via intranasal administration, and an intranasal delivery system for Tosymra®. This patent, excluding possible patent term extensions, is expected to expire in 2030.
“We believe this patent further solidifies Tosymra® in the market as a differentiated drug with a differentiated administration method,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “This new patent targets specific delivery and composition. We are thrilled to have these additional issued patent claims, which add to the intellectual property protection existing for Tosymra®.”
About Migraine
Nearly 40 million people in the United States suffer from migraine1 and it has been recognized as the second leading cause of disability in the world2,3. Migraine is characterized by debilitating attacks lasting four to 72 hours with multiple symptoms, including pulsating headaches of moderate to severe pain intensity often associated with nausea or vomiting, and/or sensitivity to sound (phonophobia) and sensitivity to light (photophobia)4.
1Law, H. Z., Chung, M. H., Nissan, G., Janis, J. E., & Amirlak, B. (2020). Hospital Burden of Migraine in United States Adults: A 15-year National Inpatient Sample Analysis. Plastic and reconstructive surgery. Global open, 8(4), e2790. https://doi.org/10.1097/GOX.0000000000002790
2GBD 2016 Headache Collaborators. Global, regional, and national burden of migraine and tension-type headache, 1990-2016: a systematic analysis for the Global Burden of Disease Study 2016. Lancet Neurol 2018;17(11):954-976.
3Steiner, T.J., Stovner, L.J., Jensen, R. et al. Lifting the Burden: the Global Campaign against Headache. Migraine remains second among the world’s causes of disability, and first among young women: findings from GBD2019.J Headache Pain 21, 137 (2020).
4Headache Classification Committee of the International Headache Society (IHS). The international classification of headache disorders, 3rd edition. Cephalalgia. 2018;38(1):1–211.
Tonix Pharmaceuticals Holding Corp.*
Tonix is a fully integrated biopharmaceutical company focused on transforming therapies for pain management and modernizing solutions for public health challenges. Tonix’s development portfolio is focused on central nervous system (CNS) disorders, and its priority is to submit a New Drug Application (NDA) to the FDA in October 2024 for TNX-102 SL, a product candidate for which two statistically significant Phase 3 studies have been completed for the management of fibromyalgia. The FDA has granted Fast Track designation to TNX-102 SL for the management of fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction. Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase), a biologic in Phase 2 development designed to treat cocaine intoxication that has Breakthrough Therapy designation. Tonix’s immunology development portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft rejection and for the treatment of autoimmune diseases. Tonix also has product candidates in development in the areas of rare disease, including TNX-2900 for Prader-Willi syndrome, and infectious disease, including a vaccine for mpox, TNX-801. Tonix recently announced the U.S. Department of Defense (DoD), Defense Threat Reduction Agency (DTRA) awarded it a contract for up to $34 million over five years in an Other Transaction Agreement (OTA) 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, instrumental in progressing this development. Tonix Medicines, our commercial subsidiary, markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan nasal spray) 10 mg for the treatment of acute migraine with or without aura in adults.
*Tonix’s product development candidates are investigational new drugs or biologics and have not been approved for any indication.
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.
Tosymra can cause serious side effects, including heart attack and other heart problems, which may lead to death. Stop Tosymra and get emergency medical help if you have any signs of 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
Tosymra is 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 is done and shows no problem.
Do not use 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
severe liver problems
hemiplegic or basilar migraines. If you are not sure if you have these, ask your healthcare provider.
had a stroke, transient ischemic attacks (TIAs), or problems with blood circulation
taken any of the following medicines in the last 24 hours: almotriptan, eletriptan, frovatriptan, naratriptan, rizatriptan, ergotamines, or dihydroergotamine. Ask your provider if you are not sure if your medicine is listed above.
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 ingredient in Tosymra
Tell your provider about all of your medical conditions and medicines you take, including vitamins and supplements.
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.
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 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 Tosymra include: tingling, dizziness, feeling warm or hot, burning feeling, feeling of heaviness, feeling of pressure, flushing, feeling of tightness, numbness, application site (nasal) reactions, abnormal taste, and throat irritation.
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 Tosymra. For more information, ask your provider.
This is the most important information to know about 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 www.tonixpharma.com or call 1-888-650-3789.
You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
INDICATION AND USAGE Tosymra is a prescription medicine used to treat acute migraine headaches with or without aura in adults.
Tosymra is not used to treat other types of headaches such as hemiplegic or basilar migraines or cluster headaches.
Tosymra is not used to prevent migraines. It is not known if Tosymra is safe and effective in children under 18 years of age.
Joe Gomes, CFA, Managing Director, Equity Research Analyst, Generalist , Noble Capital Markets, Inc.
Joshua Zoepfel, Research Associate, Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
New Funding. MustGrow announced yesterday that the Company is receiving funding from Agriculture and Agri-Food Canada (AAFC) to investigate its mustard-derived ingredient. The ingredient could potentially be used in human and animal health and food applications. The funding amount was not disclosed. The Company will continue to focus on its core business outside of the program, with the anticipation of more state approvals for TerraSante, in our view.
The Program. Under the program, the Company and AAFC will look at bioactive components in mustard seed and assess yellow mustard extracts for potential antibiotic and gut health applications in both humans and animals. They will also test the selected bioactive components for antifungal and antibacterial activities against food spoilage microbes and disease pathogens and for use as ingredients in functional foods and nutraceuticals for improved immunity and gut health in both humans and animals.
Equity Research is available at no cost to Registered users of Channelchek. Not a Member? Click ‘Join’ to join the Channelchek Community. There is no cost to register, and we never collect credit card information.
This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).
*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision.
Organon, a global healthcare company focused on improving women’s health, announced a major acquisition of Dermavant Sciences Ltd., a subsidiary of Roivant. This acquisition includes Dermavant’s innovative dermatologic therapy, VTAMA® (tapinarof) cream, 1%, which is approved for treating plaque psoriasis and is currently under FDA review for atopic dermatitis.
The acquisition enhances Organon’s presence in the U.S. dermatology market, adding to their international portfolio. This move aligns with Organon’s mission to provide treatments for conditions that disproportionately affect women. The inclusion of VTAMA cream, which addresses psoriasis and potentially atopic dermatitis, fits into their strategic goal of expanding access to effective therapies. Dermavant’s established commercial team will integrate with Organon’s market capabilities, further extending the product’s reach.
Dermavant’s VTAMA cream has been a game changer in the dermatology space. Approved in May 2022, it provides a non-steroidal, once-daily treatment option for plaque psoriasis, a condition that impacts over 8 million Americans. Unlike traditional steroid treatments, VTAMA cream is free of safety warnings, making it an appealing option for long-term use. The product is also under review to extend its use to treat atopic dermatitis, a common inflammatory skin condition affecting over 16.5 million adults and 9.6 million children in the U.S.
Organon’s acquisition of Dermavant not only strengthens its foothold in the U.S. market but also provides a new channel for global growth. Organon CEO Kevin Ali emphasized that this acquisition is part of their commitment to improving women’s health and that integrating Dermavant’s operations would accelerate VTAMA’s availability to patients. With Organon’s commercial scale, they expect to significantly increase patient access to this novel therapy globally.
The acquisition is structured with an upfront payment of $175 million, a milestone payment of $75 million contingent upon FDA approval for atopic dermatitis, and additional payments of up to $950 million tied to commercial milestones. Dermavant shareholders will also receive tiered royalties on net sales. The deal, subject to regulatory approvals, is expected to close by the fourth quarter of 2024.
The acquisition complements Organon’s portfolio of women’s health solutions, biosimilars, and established medicines, bringing a much-needed dermatological therapy into its fold. VTAMA cream has been a success, becoming the top branded topical for plaque psoriasis within just two months of its launch. Organon expects that the therapy will continue to grow, especially as the FDA considers its use for atopic dermatitis.
For Dermavant, the deal provides an opportunity for continued growth, with the support and scale of Organon to take VTAMA to new markets and potentially reach millions of patients globally.
With this acquisition, Organon is well-positioned to further expand its impact in dermatology, providing innovative treatments for plaque psoriasis and potentially atopic dermatitis. The company continues to focus on improving the lives of patients, particularly women, by offering accessible and effective healthcare solutions.
PDS Biotech is a clinical-stage immunotherapy company developing a growing pipeline of molecularly targeted cancer and infectious disease immunotherapies based on the Company’s proprietary Versamune® and Infectimune™ T-cell activating technology platforms. Our Versamune®-based products have demonstrated the potential to overcome the limitations of current immunotherapy by inducing in vivo, large quantities of high-quality, highly potent polyfunctional tumor specific CD4+ helper and CD8+ killer T-cells. PDS Biotech has developed multiple therapies, based on combinations of Versamune® and disease-specific antigens, designed to train the immune system to better recognize diseased cells and effectively attack and destroy them. The Company’s pipeline products address various cancers including HPV16-associated cancers (anal, cervical, head and neck, penile, vaginal, vulvar) and breast, colon, lung, prostate and ovarian cancers.
Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
Additional Clinical Details Presented At ESMO Conference. PDS Biotech presented an update to its Phase 2 VERSATILE-002 trial at the European Society for Medical Oncology (ESMO), adding detail to the data announced in June 2024. We see these Phase 2 data as highly encouraging for the Phase 3 VERSATILE-003 trial testing the doublet combination and the triplet combination (Versamune HPV with PDS01ADC and Keytruda) against Keytruda alone.
Trial Continues As Patient Survival Continues To Surpass Other Treatments. The VERSATILE-002 enrolled 53 patients with recurrent or metastatic head and neck squamous cell carcinoma (R/M HNSCC). As of the May 17, 2024, analysis date, 27 patients remain alive with some reaching the 3-year survival point. Out of those alive, 10 remain on treatment and 17 are followed for survival.
Equity Research is available at no cost to Registered users of Channelchek. Not a Member? Click ‘Join’ to join the Channelchek Community. There is no cost to register, and we never collect credit card information.
This Company Sponsored Research is provided by Noble Capital Markets, Inc., a FINRA and S.E.C. registered broker-dealer (B/D).
*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision.
Median Overall Survival remains at 30 months; Objective Response Rate of 36% and Disease Control Rate of 77%
11/53 (21%) of patients experienced 90-100% tumor shrinkage
VERSATILE-003 Phase 3 clinical trial planned to begin this year
PRINCETON, N.J., Sept. 16, 2024 (GLOBE NEWSWIRE) — PDS Biotechnology Corporation (Nasdaq: PDSB) (“PDS Biotech” or the “Company”), a late-stage immunotherapy company focused on transforming how the immune system targets and kills cancers and the development of infectious disease vaccines, today announced updated data from the VERSATILE-002 trial evaluating Versamune® HPV (formerly PDS0101) in combination with KEYTRUDA® (pembrolizumab) as a first-line (1L) treatment for patients with HPV16-positive recurrent/metastatic (R/M) head and neck squamous cell carcinoma (HNSCC). The data were presented during a poster session on September 14 at the European Society for Medical Oncology (ESMO) Congress 2024 in Barcelona, Spain.
As of the latest data cut of the VERSATILE-002 single-arm, Phase 2 trial on May 17, 2024, Versamune® HPV plus pembrolizumab continued to be well tolerated in this 1L R/M HPV16-positive HNSCC population. Enrollment in the trial (n=53) is complete, 10 patients remain on study treatment and 27 patients (including the 10 on treatment) continue to be followed for survival. Median patient follow-up is 16 months. The data demonstrated the following:
Median Overall Survival (mOS) was 30 months with a lower 95% confidence interval of 19.7 months; Published mOS for pembrolizumab is 12-18 months1,2
Objective Response Rate (ORR) of 36% (19/53); Published ORR for pembrolizumab is 19-25%1,2
Disease Control Rate (DCR) is 77% (41/53)
21% (11/53) of patients had deep tumor responses and shrinkage of 90-100%
9% (5/53) of patients had a complete response
Treatment-related adverse events of Grade ≥3 were seen in 9 patients (Grade 3, n=8 and Grade 4, n=1)
“The updated response data we presented at ESMO show the strong clinical activity and durability of Versamune® HPV plus pembrolizumab,” said Jared Weiss, M.D., Section Chief of Thoracic and Head/Neck Oncology, Professor of Medicine at the University of North Carolina, and principal investigator of the VERSATILE-002 clinical trial. “Continued evaluation shows the promise of this combination in improving survival for patients with HPV16-positive HNSCC.”
A global, randomized, controlled Phase 3 clinical trial, VERSATILE-003, that will evaluate Versamune® HPV plus pembrolizumab vs. pembrolizumab monotherapy as 1L treatment in patients with HPV16-positive R/M HNSCC with CPS ≥1 is planned to start this year.
“We’re encouraged to see that as the data from our VERSATILE-002 clinical trial have matured, responses continue to improve, suggesting durability of the Versamune® HPV induced anti-tumor immune response,” said Dr. Kirk Shepard, M.D., Chief Medical Officer of PDS Biotech. “The encouraging patient survival and clinical responses coupled with promising tolerability as seen in the VERSATILE-002 trial underscore our belief in the potential of the combination to be the first HPV-targeted immunotherapy for HNSCC, and a significant advancement in the treatment of the growing population of patients with HPV16-positive HNSCC. We are working toward initiating the VERSATILE-003 Phase 3 study this year.”
Versamune® HPV has been granted Fast Track designation by the FDA.
Licitra L. et al. 2024, International Journal of Radiation Oncology Volume 118, Issue 5e2-e3April 01
No head-to-head studies have been performed comparing Versamune® HPV with other treatments
About PDS Biotechnology PDS Biotechnology is a late-stage immunotherapy company focused on transforming how the immune system targets and kills cancers and the development of infectious disease vaccines. The Company plans to initiate a pivotal clinical trial in 2024 to advance its lead program in advanced HPV16-positive head and neck squamous cell cancers. PDS Biotech’s lead investigational targeted immunotherapy Versamune® HPV is being developed in combination with a standard-of-care immune checkpoint inhibitor, and also in a triple combination including PDS01ADC, an IL-12 fused antibody drug conjugate (ADC), and a standard-of-care immune checkpoint inhibitor.
Forward Looking Statements This communication contains forward-looking statements (including within the meaning of Section 21E of the United States Securities Exchange Act of 1934, as amended, and Section 27A of the United States Securities Act of 1933, as amended) concerning PDS Biotechnology Corporation (the “Company”) and other matters. These statements may discuss goals, intentions and expectations as to future plans, trends, events, results of operations or financial condition, or otherwise, based on current beliefs of the Company’s management, as well as assumptions made by, and information currently available to, management. Forward-looking statements generally include statements that are predictive in nature and depend upon or refer to future events or conditions, and include words such as “may,” “will,” “should,” “would,” “expect,” “anticipate,” “plan,” “likely,” “believe,” “estimate,” “project,” “intend,” “forecast,” “guidance”, “outlook” and other similar expressions among others. Forward-looking statements are based on current beliefs and assumptions that are subject to risks and uncertainties and are not guarantees of future performance. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: the Company’s ability to protect its intellectual property rights; the Company’s anticipated capital requirements, including the Company’s anticipated cash runway and the Company’s current expectations regarding its plans for future equity financings; the Company’s dependence on additional financing to fund its operations and complete the development and commercialization of its product candidates, and the risks that raising such additional capital may restrict the Company’s operations or require the Company to relinquish rights to the Company’s technologies or product candidates; the Company’s limited operating history in the Company’s current line of business, which makes it difficult to evaluate the Company’s prospects, the Company’s business plan or the likelihood of the Company’s successful implementation of such business plan; the timing for the Company or its partners to initiate the planned clinical trials for PDS01ADC, Versamune® HPV (formerly PDS0101), PDS0203 and other Versamune® and Infectimune® based product candidates; the future success of such trials; the successful implementation of the Company’s research and development programs and collaborations, including any collaboration studies concerning PDS01ADC, Versamune® HPV, PDS0203 and other Versamune® and Infectimune® based product candidates and the Company’s interpretation of the results and findings of such programs and collaborations and whether such results are sufficient to support the future success of the Company’s product candidates; the success, timing and cost of the Company’s ongoing clinical trials and anticipated clinical trials for the Company’s current product candidates, including statements regarding the timing of initiation, pace of enrollment and completion of the trials (including the Company’s ability to fully fund its disclosed clinical trials, which assumes no material changes to the Company’s currently projected expenses), futility analyses, presentations at conferences and data reported in an abstract, and receipt of interim or preliminary results (including, without limitation, any preclinical results or data), which are not necessarily indicative of the final results of the Company’s ongoing clinical trials; any Company statements about its understanding of product candidates mechanisms of action and interpretation of preclinical and early clinical results from its clinical development programs and any collaboration studies; the Company’s ability to continue as a going concern; and other factors, including legislative, regulatory, political and economic developments not within the Company’s control. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the other risks, uncertainties, and other factors described under “Risk Factors,” “Management’s Discussion and Analysis of Financial Condition and Results of Operations” and elsewhere in the documents we file with the U.S. Securities and Exchange Commission. The forward-looking statements are made only as of the date of this press release and, except as required by applicable law, the Company undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether as a result of new information, future events or otherwise.
Versamune® and Infectimune® are registered trademarks of PDS Biotechnology Corporation.
Keytruda® is a registered trademark of Merck Sharp and Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, N.J., USA.
The World Health Organization (WHO) released its preferred target product profile (TPP) criteria for mpox vaccines at its Mpox Research and Innovation Scientific Conference held August 29-30
TNX-801, Tonix’s attenuated live-virus vaccine candidate, has characteristics that align closely with WHO’s TPP
On August 14, 2024, the WHO determined that the upsurge of mpox in a growing number of countries in Africa constitutes a public health emergency of international concern1–4: cases of the potentially lethal new Clade I mpox also detected in Sweden, Thailand and Singapore
CHATHAM, N.J., Sept. 16, 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 the World Health Organization’s (WHO’s) preferred target product profile (TPP), released at the WHO sponsored Mpox Research and Innovation Scientific Conference held August 29-30, 2024, aligns with the characteristics of TNX-801 (horsepox, live virus) vaccine, which is being developed for preventing mpox (formerly known as monkeypox). 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.
“The characteristics of TNX-801 align with the draft TPP released at the WHO sponsored Mpox Research and Innovation Scientific Conference,” said Seth Lederman, M.D., Chief Executive Officer of Tonix. “In animal studies TNX-801 has shown single dose protection against a lethal challenge of Clade I monkeypox virus administered by intratracheal route.5 In addition, protected animals did not produce any infectious virus suggesting TNX-801 has the potential to block forward transmission as expected with live-virus vaccines. TNX-801 is designed for percutaneous administration using a bifurcated needle, like the products and delivery used in WHO’s accelerated smallpox eradication project. Since TNX-801 is a live-virus vaccine, we expect the stability of lyophilized TNX-801 at ambient temperature to be similar to live vaccinia virus vaccines including ACAM2000. We believe TNX-801 can be shipped and stored without the need for a costly and cumbersome ultra-cold supply chain, a particular advantage in lesser developed parts of the world. The stability of live virus vaccines eliminates the need for ultra-cold storage which complicates the widespread use of mRNA vaccines in Africa, where they are needed most right now. Finally, studies on immunocompromised animals6 suggest that TNX-801 may be given to persons with immunocompromising conditions such as HIV, which is another property that will be essential for public health.”
Dr. Lederman continued, “The recent WHO declaration of a Public Health Emergency of International Concern (PHEIC) underscores the urgent need for new vaccines to control this outbreak and save lives. We have been motivated to develop TNX-801 because single-dose vaccines simplify logistics of administration, achieve higher coverage by reducing vaccinee dropout between doses and allow for case-contact or “ring” strategies to vaccinate the contacts of confirmed mpox patients.7,8 Ring vaccination is deemed essential for controlling mpox but requires single-dose vaccines that interrupt forward transmission.”7,8
On August 26, 2024, Tonix announced a collaboration with Bilthoven Biologics (Bbio) to develop GMP manufacturing processes for its mpox vaccine. Bbio is part of the world’s largest vaccine manufacturer, the Cyrus Poonawalla Group, which also includes the Serum Institute of India.
The U.S. Food and Drug Administration (FDA) approved vaccines for mpox are a two-dose non-replicating vaccine called Jynneos® from Bavarian Nordic9 and a one-dose live-virus vaccine from Emergent for people at high risk for mpox infection.10 WHO recently authorized Jynneos for use in adults.11 Recently data in animals have been reported for a two-dose mRNA vaccine from Moderna.12
About TNX-801*
TNX-801 is a live replicating attenuated vaccine based on horsepox that is believed to provide immune protection with better tolerability than 20th Century vaccinia viruses. As previously disclosed, TNX-801 protected animals against lethal challenge with intratracheal Clade I monkeypox virus.5 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.6 The Findings are consistent with mucosal immunity and suggest the ability to block forward transmission, similar to Dr. Edward Jenner’s vaccinia vaccine, which eradicated smallpox and kept mpox out of the human population. TNX-801 combines immune protection with improved tolerability compared to other vaccines based on orthopoxviruses and is administered with a single dose which has advantages over two-dose regimens. The focus on single-dose vaccines confirms early recommendations by the Bipartisan Commission on Biodefense, 7 and the U.S. National Academies of Science.7,8 The National Academies of Science (NAS) report highlights the difficulty of a ring vaccination strategy with even a two-dose regimen. 7 The U.S. National Institutes of Health (NIH) selected Tonix’s COVID-19 vaccine, TNX-1800 for Project NextGen. TNX-1800 is an engineered version of horsepox that expresses the spike protein of SARS-CoV-2. 13,14
About Mpox* On August 14, 2024, the WHO determined that the upsurge of mpox in a growing number of countries in Africa constitutes a PHEIC the second such declaration in the past two years called in response to an mpox outbreak.1 The current outbreak is caused by Clade I monkeypox virus, while the 2022 outbreak was Clade 2 monkeypox virus. The global mpox outbreak, which commenced in 2022 has affected over 90,000 persons in countries where mpox had previously not been endemic, including Europe and the US. The spread of Clade IIb strain mpox in 2022 underscores the pandemic potential of mpox. Unlike Clade IIb mpox, the Clade I strain of mpox appears to be spreading to countries neighboring the Democratic Republic of the Congo, including Burundi, Rwanda, Uganda and Kenya. Clade I mpox is typically associated with approximately twenty times the case fatality rates than Clade IIb mpox in Africa. According to the U.S. Centers for Disease Control and Prevention (CDC), and other experts, there is a significant risk that the deadlier Clade I strain may appear in the U.S.2,3
Tonix Pharmaceuticals Holding Corp.* Tonix is a fully-integrated biopharmaceutical company focused on developing, licensing and commercializing therapeutics to treat and prevent human disease and alleviate suffering. Tonix’s priority is to submit a New Drug Application (NDA) to the FDA in October of 2024 for TNX-102 SL, a product candidate for which two statistically significant Phase 3 studies have been completed for the management of fibromyalgia. The FDA has granted Fast Track designation to TNX-102 SL for the management of fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction. Tonix recently announced the U.S. Department of Defense (DoD), Defense Threat Reduction Agency (DTRA) awarded it a contract for up to $34 million over five years in an Other Transaction Agreement (OTA) 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. The company’s Good Manufacturing Practice (GMP)-capable advanced manufacturing facility in Dartmouth, MA was purpose-built to manufacture TNX-801 and the GMP suites are ready to be reactivated in case of a national or international emergency. Tonix’s development portfolio is focused on central nervous system (CNS) disorders. Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase), a biologic in Phase 2 development designed to treat cocaine intoxication that has Breakthrough Therapy designation. Tonix’s immunology development portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft rejection and for the treatment of autoimmune diseases. Tonix also has product candidates in development in the areas of rare disease and infectious disease. Tonix Medicines, our commercial subsidiary, markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan nasal spray) 10 mg for the treatment of acute migraine with or without aura in adults.
*Tonix’s product development candidates are investigational new drugs or biologics and have not been approved for any indication.
Zembrace SymTouch and Tosymra are registered trademarks of Tonix Medicines. All other marks are property of their respective owners.
This press release and further information about Tonix can be found at www.tonixpharma.com.
1WHO Press Release August 14, 2024. “WHO Director-General declares mpox outbrfeak a public health emergency of international concern”. URL: www.who.int/news/item/14-08-2024-who-director-general-declares-mpox-outbreak-a-public-health-emergency-of-international-concern (accessed 8-15-24) 2McQuiston JH, et al. U.S. Preparedness and Response to Increasing Clade I Mpox Cases in the Democratic Republic of the Congo. 2024, MMWR Morbi Mortal Wkly Rep: United States. p. 435-440 3CDC. 2022-2023 Mpox: US Map and Case Count. https://www.cdc.gov/poxvirus/mpox/response/2022/us-map.html 4World Health Organization SAGE meeting highlights on updated mpox vaccine recommendations. 2024, March 5Noyce RS, et al. Viruses. 2023 Jan 26;15(2):356. Doi: 10.3390/v15020356. PMID: 36851570; PMCID: PMC9965234 6Trefry, SV et al. bioRxiv 2023.10.25.564033; doi: https://doi.org/10.1101/2023.10.25.564033 7Bipartisan Commission on Biodefense. Box the Pox: Reducing the risk of Smallpox and Other Ortho poxviruses, Washington:2024 8U.S. National Academies of Science. Future State of Smallpox Medical Countermeasures. Washington:2024 9Zaeck LM, et al. Low levels of monkeypox virus-neutralizing antibodies after MVA-BN vaccination in healthy individuals. Nat Med. 2023 Jan;29(1):270-278. doi: 10.1038/s41591-022-02090-w. Epub 2022 Oct 18. PMID: 36257333; PMCID: PMC9873555. 10August 30, 2024. Reuters. “US FDA approves Emergent’s smallpox vaccine for people at high risk of mpox”. https://www.msn.com/en-us/health/other/us-fda-approves-emergent-s-smallpox-vaccine-for-people-at-high-risk-of-mpox/ 11Keaton, J. Sept. 13, 2024. Associated Press. “WHO grants first mpox vaccine approval to ramp up response to disease in Africa.” URL: https://bit.ly/4e4yyeb 12Mucker et al., (in press) Comparison of protection against mpox following mRNA or modified vaccinia Ankara vaccination in nonhuman primates, Cell (2024), https://doi.org/10.1016/j.cell.2024.08.043 13Awasthi M, et al. Viruses. 2023 Oct 21;15(10):2131. Doi: 10.3390/v15102131. PMID: 37896908; PMCID: PMC10612059. 14Awasthi M, et al. Vaccines (Basel). 2023 Nov 2;11(11):1682. Doi: 10.3390/vaccines11111682.PMID: 38006014
Forward Looking Statements Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as “anticipate,” “believe,” “forecast,” “estimate,” “expect,” and “intend,” among others. These forward-looking statements are based on Toni’s current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, risks related to the failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; risks related to the failure to successfully market any of our products; risks related to the timing and progress of clinical development of our product candidates; our need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party payor reimbursement; limited research and development efforts and dependence upon third parties; and substantial competition. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Tonix does not undertake an obligation to update or revise any forward-looking statement. Investors should read the risk factors set forth in the Annual Report on Form 10-K for the year ended December 31, 2023, as filed with the Securities and Exchange Commission (the “SEC”) on April 1, 2024, and periodic reports filed with the SEC on or after the date thereof. All of Toni’s forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date thereof.
Key Points: – MBX Biosciences raised $163.2 million, focusing on metabolic and endocrine disorders. – Bicara Therapeutics and Zenas BioPharma raised $315M and $225M, respectively. – These IPOs reflect renewed investor interest in biotech amid a sluggish broader market.
In a significant boost to the biotech IPO market, three emerging biotech companies—MBX Biosciences, Bicara Therapeutics, and Zenas BioPharma—collectively raised over $700 million through initial public offerings (IPOs). This surge in biotech IPOs, after a quiet summer, underscores the sector’s ability to attract investor attention despite broader market challenges.
MBX Biosciences successfully raised $163.2 million by pricing 10.2 million shares at $16 each, the high end of its expected range. MBX is developing peptide-based therapies for treating metabolic and endocrine disorders, including its lead candidate, MBX 2109, which targets chronic hypoparathyroidism. The company is also developing a preclinical therapy, MBX 4291, aimed at treating obesity by mimicking the effects of gut hormones GLP-1 and GIP. These advances in weight-loss therapies have garnered significant investor interest, especially as obesity treatments like Eli Lilly’s Zepbound and Novo Nordisk’s Wegovy continue to show potential for reducing risks such as stroke and heart attacks.
Another notable IPO, Bicara Therapeutics, raised $315 million, positioning itself as the third-largest biotech IPO of the year. Bicara is focused on developing bifunctional antibody drugs for treating cancers, including head and neck cancers. With plans to launch a late-stage trial alongside Merck’s Keytruda, Bicara is well-positioned to explore treatments for other solid tumors as well.
Zenas BioPharma raised $225 million through its IPO and is gaining traction in the immunology space. Zenas is developing a dual-targeting antibody currently in Phase 3 testing for treating IgG4-related diseases and anemia. With potential applications for multiple sclerosis and lupus, the company is riding a wave of enthusiasm for immune therapies, contributing to its successful public offering.
These IPOs reflect a growing interest in later-stage biotech companies, with all three firms advancing drugs already in human testing. The renewed confidence in the sector could also signal more biotech IPOs on the horizon, particularly as companies look to capitalize on robust investor demand for novel therapies in metabolic diseases, cancer, and immunology.
In a market that has been challenging for biotech firms, these successful IPOs highlight the resilience of companies with strong pipelines and innovative approaches to medical treatment. With MBX Biosciences set to trade under the symbol “MBX” on the Nasdaq Global Select Market, investors are closely watching the sector, hopeful that this uptick in activity is a sign of better things to come for biotech in 2025.
Joe Gomes, CFA, Managing Director, Equity Research Analyst, Generalist , Noble Capital Markets, Inc.
Joshua Zoepfel, Research Associate, Noble Capital Markets, Inc.
Refer to the full report for the price target, fundamental analysis, and rating.
Another Approval. Following the approval from the state of Arizona late last month, MustGrow announced receipt from the Florida Department of Agriculture and Consumer Services (FDACS) for the registration approval of its TerraSante product. Florida now becomes the sixth state to approve the Company’s product alongside Arizona, Idaho, California, Oregon, and Washington.
Florida Market. The state consists of around 47,300 farms which utilized 9.7 million acres, 5,000 farms of which had sales exceeding $100,000 according to the FDACS. Florida ranked first in 2022 in value of the production of bell peppers, Valencia oranges, grapefruit, sugarcane, fresh market tomatoes, and watermelons. The average farm size in the state is around 205 acres. The state adds plenty of opportunity to MustGrow’s expanding pipeline and we expect more states to be added in the future.
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*Analyst certification and important disclosures included in the full report. NOTE: investment decisions should not be based upon the content of this research summary. Proper due diligence is required before making any investment decision.
