Historic kidney xenotransplantation procedure conducted at Massachusetts General Hospital
Tegoprubart administration has now been usedinvestigationally to prevent rejection in both kidney and heart pig-to-human xenotransplantations, as well as in human-to-human kidney transplantation
Eledon recently presented results from its ongoing Phase 1b kidney transplantation study which demonstrated that tegoprubart was generally safe and well tolerated and successfully prevented rejection with post-transplant kidney function above historical averages
IRVINE, Calif., March 21, 2024 (GLOBE NEWSWIRE) — Eledon Pharmaceuticals, Inc. (“Eledon”) (NASDAQ: ELDN) today announced that tegoprubart, the company’s investigational anti-CD40L antibody, was used as a component of the immunosuppressive treatment regimen following the first-ever transplant of a kidney from a genetically modified pig to a human. The procedure was completed on March 16, 2024, at Massachusetts General Hospital on a 62-year-old man living with end-stage kidney disease.
“This first-ever kidney xenotransplant marks a pivotal moment for the transplant community and provides hope that this option may one day help solve the current shortage of available organs,” said David-Alexandre C. Gros, M.D., Eledon Chief Executive Officer. “Eledon has now participated in both heart and kidney xenotransplant procedures, further demonstrating tegoprubart’s broad potential in transplant. We are thankful to the patient, the entire medical team at Massachusetts General Hospital, and our partner eGenesis for the privilege to participate in this landmark procedure as we work to achieve our goal of developing tegoprubart as a new and better immunosuppressive option for transplant patients.”
Tegoprubart is being administered to the patient investigationally as part of a regimen designed to suppress the immune system and prevent the body from rejecting the transplanted pig organ. Tegoprubart has been observed to be safe and well-tolerated in multiple studies and in multiple indications, including for the prevention of rejection following kidney transplantation.
“It is exciting to see the clinical application of xenotransplantation to a patient with end stage renal disease,” said Andrew Adams, MD, PhD, Chief, Division of Transplant Surgery, University of Minnesota. “Based on all of the studies performed in preclinical models to date, it is clear that therapies targeting CD40L, like tegoprubart, are critical to controlling the immune response to the xenograft, potentially leading to superior long-term outcomes compared to other immunosuppressive therapies. CD40L sits at the interface of the adaptive and innate immune responses which may explain why therapies designed to block it have such potent effects in xenotransplantation.”
“This procedure represents a significant milestone in the transplantation field and a promising step to address a medical crisis: the worldwide shortage of available organs,” said Leonardo V. Riella, MD, PhD, Medical Director for Kidney Transplantation at Massachusetts General Hospital. “Xenotransplantation represents a unique approach with the potential to provide patients with additional options to access life-saving treatments in a timely manner. We commend the courage of our patient and the skill of the entire team involved in the operation, and I look forward to continued advancements in research with the hope that we can make this novel treatment option available to more patients in the future.”
Multiple clinical and preclinical research efforts are currently underway to evaluate the ability of tegoprubart to reduce the risk of rejection in organ transplant. Eledon is advancing preclinical studies in which tegoprubart is being used as a part of the immunosuppression regimen designed to reduce the risk of rejection in nonhuman primate recipients in xenotransplant procedures. In parallel, Eledon is running two global clinical studies evaluating tegoprubart for the prevention of organ rejection in persons receiving a de novo kidney transplant. The company recently presented results from 11 participants enrolled in its ongoing Phase 1b kidney transplantation study, which demonstrated that tegoprubart, as part of a calcineurin inhibitor free immunosuppressive regimen, was generally safe and well tolerated and both successfully prevented rejection as well as permitted above historical average post-transplant kidney function. The company’s Phase 2 BESTOW study, assessing tegoprubart head-to-head with tacrolimus for the prevention of rejection in kidney transplantation, is currently recruiting participants, and plans to complete enrollment at the end of 2024.
About Eledon Pharmaceuticals and tegoprubart
Eledon Pharmaceuticals, Inc. is a clinical stage biotechnology company that is developing immune-modulating therapies for the management and treatment of life-threatening conditions. The Company’s lead investigational product is tegoprubart, an anti-CD40L antibody with high affinity for the CD40 Ligand, a well-validated biological target that has broad therapeutic potential. The central role of CD40L signaling in both adaptive and innate immune cell activation and function positions it as an attractive target for non-lymphocyte depleting, immunomodulatory therapeutic intervention. The Company is building upon a deep historical knowledge of anti-CD40 Ligand biology to conduct preclinical and clinical studies in kidney allograft transplantation, xenotransplantation, and amyotrophic lateral sclerosis (ALS). Eledon is headquartered in Irvine, California. For more information, please visit the Company’s website at www.eledon.com.
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Forward-Looking Statements
This press release contains forward-looking statements that involve substantial risks and uncertainties. Any statements about the company’s future expectations, plans and prospects, including statements about planned clinical trials, the development of product candidates, expected timing for initiation of future clinical trials, expected timing for receipt of data from clinical trials, as well as other statements containing the words “believes,” “anticipates,” “plans,” “expects,” “estimates,” “intends,” “predicts,” “projects,” “targets,” “looks forward,” “could,” “may,” and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are inherently uncertain and are subject to numerous risks and uncertainties, including: risks relating to the safety and efficacy of our drug candidates; risks relating to clinical development timelines, including interactions with regulators and clinical sides, as well as patient enrollment; risks relating to costs of clinical trials and the sufficiency of the company’s capital resources to fund planned clinical trials; and risks associated with the impact of the ongoing coronavirus pandemic. Actual results may differ materially from those indicated by such forward-looking statements as a result of various factors. These risks and uncertainties, as well as other risks and uncertainties that could cause the company’s actual results to differ significantly from the forward-looking statements contained herein, are discussed in our quarterly 10-Q, annual 10-K, and other filings with the U.S. Securities and Exchange Commission, which can be found at www.sec.gov. Any forward-looking statements contained in this press release speak only as of the date hereof and not of any future date, and the company expressly disclaims any intent to update any forward-looking statements, whether as a result of new information, future events or otherwise.
Tonmya™ is a potential new first-line, centrally acting non-opioid analgesic for the management of fibromyalgia, supported by positive results from two Phase 3 studies
New Drug Application (NDA) submission to the FDA planned for second half of 2024
CHATHAM, N.J., March 20, 2024 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a biopharmaceutical company with marketed products and a pipeline of development candidates, today announced it has selected two contract manufacturing organizations (CMOs), one of which is Almac Pharma Services, a member of the privately owned Almac Group, as dual supply sources for the potential launch and commercialization of Tonmya™ (also known as TNX-102 SL, cyclobenzaprine HCl sublingual tablets) in the U.S.
“Dual sourcing is a critical element for the successful commercial launch and supply chain management of a product,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “We are excited to advance our first internally developed program toward NDA submission and to work with two well-established CMOs for commercial supply and potential launch of Tonmya.”
“Having supported the development and clinical trial supply of this drug, we’re thrilled to be continuing our partnership with Tonix to support the commercial launch and ongoing supply of this important new non-opioid analgesic to patients with fibromyalgia, a chronic debilitating disease,” said Mark English, VP Operations, Almac Pharma Services.
Tonmya is a centrally acting, non-opioid medication. As previously announced, Tonix’s second positive Phase 3 study, RESILIENT, met its pre-specified primary endpoint, significantly reducing daily pain compared to placebo (p=0.00005) in participants with fibromyalgia. Statistically significant and clinically meaningful results (p=0.001 or better) were also seen in all key secondary endpoints related to improving sleep quality, reducing fatigue, and improving 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.
Tonix plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration in the second half of 2024 for Tonmya for the management of fibromyalgia.
About Tonmya* (also known as TNX-102 SL)
TNX-102 SL is a tablet containing 2.8 mg cyclobenzaprine HCl that will be administered sublingually once daily at bedtime for the first 2 weeks, titrating subsequently to 2 tablets (5.6 mg total per day) at bedtime, as tolerated, for chronic, long-term use. The sublingual tablet is formulated using a patented Protectic™ eutectic formulation including a basifying agent for transmucosal absorption with rapid systemic exposure pharmacokinetic properties suitable for bedtime administration. The eutectic properties enhance the stability with a predicted shelf life of greater than 48 months, at room temperature conditions. The planned commercial distribution will be a 14, 60 and 90 count tablet bottles allowing for titration, flexible and three-month chronic supply. Tonmya is a centrally acting, non-opioid, non-addictive, bedtime medication. In December 2023, the Company announced highly statistically significant and clinically meaningful topline results in RESILIENT, a second positive Phase 3 clinical trial of Tonmya for the management of fibromyalgia. RELIEF, the first positive Phase 3 trial of Tonmya in fibromyalgia, was completed in December 2020. It met its pre-specified primary endpoint of daily pain reduction compared to placebo (p=0.010) and showed activity in key secondary endpoints.
*Tonmya™ is conditionally accepted by the U.S. Food and Drug Administration (FDA) as the tradename for TNX-102 SL for the management of fibromyalgia. Tonmya has not been approved for any indication.
About Almac Pharma Services
Tailormade pharmaceutical development and commercial solutions
With over 50 years’ experience, Almac Pharma Services is a world leading outsourcing partner to the global pharmaceutical and biotechnology industry.
Employing over 1,600 highly skilled individuals across 4 locations in Europe and the US, the company provides tailored, quality-led and timely solutions from early and late phase pharmaceutical development, clinical and commercial drug product manufacture, product launch through to commercial packaging and global distribution.
On March 6, 2024, the company announced the completion of a custom-built, high-volume facility that significantly increases commercial manufacturing and packaging of sachet drug product presentations forming part of the Group’s ongoing global expansion investment now totaling over £400 million.
To keep up to date with latest news, follow us on LinkedIn or visit our website.
About Almac Group
The Almac Group is an established contract development and manufacturing organisation providing an extensive range of integrated services across the drug development lifecycle to the pharmaceutical and biotech sectors globally. Its innovative services range from R&D, biomarker discovery development and commercialisation, API manufacture, analytical services, formulation development, clinical trial supply, IRT (IVRS/IWRS) through to commercial-scale manufacture.
The international company is a privately owned organisation which has grown organically, now employing 7,200 highly skilled personnel across 18 facilities including Europe, the USA and Asia.
Tonix is a biopharmaceutical company focused on developing, licensing and commercializing therapeutics to treat and prevent human disease and alleviate suffering. Tonix’s development portfolio is focused on central nervous system (CNS) disorders. Tonix’s priority is to submit a New Drug Application (NDA) to the FDA in the second half of 2024 for Tonmya, a product candidate for which two positive Phase 3 studies have been completed for the management of fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction as well as fibromyalgia-type Long COVID. Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase) a biologic designed to treat cocaine intoxication with 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.
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, 2022, as filed with the Securities and Exchange Commission (the “SEC”) on March 13, 2023, 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.
Feedback provides greater clarity on regulatory requirements for planned Phase 2b trial
BOTHELL, Wash., March 19, 2024 (GLOBE NEWSWIRE) — Cocrystal Pharma, Inc. (Nasdaq: COCP) (“Cocrystal” or the “Company”) announces it has received Pre-Investigational New Drug (Pre-IND) feedback from the U.S. Food and Drug Administration (FDA) regarding CC-42344 as a potential oral treatment for pandemic and seasonal influenza A. A Pre-IND review provides the opportunity to obtain FDA guidance and clarification on critical steps such as the proposed clinical trial design, as well as clinical drug manufacturing and nonclinical studies deemed necessary before filing the trial design. The feedback was provided in a written response to a Pre-IND package and questions submitted by the Company in January 2024.
“We value the FDA guidance as we prepare to file the IND for our Phase 2b trial and open enrollment of patients in the U.S.,” said Sam Lee, Ph.D., Cocrystal’s President and co-CEO. “This is an important milestone that provides greater clarity on the regulatory requirements and our planned oral CC-42344 clinical program.”
A Phase 2a challenge study of oral CC-42344 is underway in the United Kingdom to evaluate safety, and viral and clinical measures in healthy volunteers who are infected with the influenza A virus. The Company expects to report topline results from this study in the second half of this year.
In addition, preparations are underway to begin a Phase 1 study in Australia with the Company’s inhaled formulation of CC-42344 as a potential influenza A treatment and prophylaxis for those exposed to the virus. Recent preclinical data showed that inhaled CC-42344 exhibited highly effective delivery into the lung, superior lung exposure, efficacy in influenza-infected human lung epithelia, and a favorable safety profile.
CC-42344 Influenza A PB2 Inhibitor CC-42344 is a novel, broad-spectrum, investigational antiviral candidate for the treatment of pandemic and seasonal influenza A. CC-42344 inhibits the first step in the viral replication process of influenza A by binding to a highly conserved PB2 site of the polymerase complex that is essential to replication. In vitro testing with CC-42344 showed excellent antiviral activity against influenza A strains, including pandemic and seasonal strains, as well as against strains resistant to Tamiflu® and Xofluza®, while also demonstrating favorable pharmacokinetic and safety profiles. In addition, oral CC-42344 demonstrated favorable safety and tolerability results in a Phase 1 study in Australia. This antiviral candidate was discovered using the Company’s proprietary structure-based drug discovery platform technology.
About Cocrystal Pharma, Inc. Cocrystal Pharma, Inc. is a clinical-stage biotechnology company discovering and developing novel antiviral therapeutics that target the replication process of influenza viruses, coronaviruses (including SARS-CoV-2), noroviruses and hepatitis C viruses. Cocrystal employs unique structure-based technologies and Nobel Prize-winning expertise to create first- and best-in-class antiviral drugs. For further information about Cocrystal, please visit www.cocrystalpharma.com.
Cautionary Note Regarding Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the expected results of the Phase 2a trial for CC-42344 for the oral treatment of influenza A in the second half of 2024, efforts in preparation for an anticipated Phase 2b trial for oral treatment of influenza A following the Phase 2a trial, efforts to begin a Phase 1 study in Australia for an inhaled formulation of CC-42344 as a potential influenza A treatment and prophylaxis, and the potential efficacy and clinical benefits of, and market for, such product candidates. The words “believe,” “may,” “estimate,” “continue,” “anticipate,” “intend,” “should,” “plan,” “could,” “target,” “potential,” “is likely,” “will,” “expect” and similar expressions, as they relate to us, are intended to identify forward-looking statements. We have based these forward-looking statements largely on our current expectations and projections about future events. Some or all of the events anticipated by these forward-looking statements may not occur. Important factors that could cause actual results to differ from those in the forward-looking statements include, but are not limited to, risks relating to our ability to proceed with the studies including recruiting volunteers and procuring materials for such studies by our clinical research organizations and vendors, the results of such studies and our ability to obtain FDA approval to initiate the Phase 2b study. Further information on our risk factors is contained in our filings with the SEC, including our Annual Report on Form 10-K for the year ended December 31, 2022. Any forward-looking statement made by us herein speaks only as of the date on which it is made. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to publicly update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by law.
Phase 2a trial is on track to begin in the first half of 2024.
Cholesterol Efflux MediatorTM VAR 200 is in development to ameliorate renal lipid accumulation that damages the kidneys’ filtration system, leading to chronic kidney disease and its progression.
WESTON, Fla., March 18, 2024 (GLOBE NEWSWIRE) — ZyVersa Therapeutics, Inc. (Nasdaq: ZVSA, or “ZyVersa”), a clinical stage specialty biopharmaceutical company developing first-in-class drugs for the treatment of renal and inflammatory diseases with high unmet medical needs, announces Institutional Review Board (IRB) approval of the Phase 2a clinical trial protocol to evaluate the efficacy and safety of Cholesterol Efflux Mediator VAR 200 in patients with diabetic kidney disease. The clinical trial is on track to begin in the first half of 2024.
Initiation of this clinical trial is a key milestone for ZyVersa. It is the first in human trial for VAR 200 intended to substantiate that the promising preclinical results demonstrated in three different animal models of kidney disease (diabetic kidney disease, focal segmental glomerulosclerosis, and Alport syndrome) translate to patients with kidney disease. The preclinical data demonstrated across all three kidney disease models that VAR 200:
Reduced cholesterol and lipid levels in the kidneys’ filtration system.
Protected against kidney injury and fibrosis.
Significantly reduced protein in the urine (proteinuria).
“A large body of evidence reinforces the importance of addressing kidney accumulation of cholesterol and lipids to help attenuate progression of chronic kidney disease, which affects over 35 million adults in the United States,” commented Stephen C. Glover, ZyVersa’s Co-founder, Chairman, CEO, and President. “Currently, over 130,000 patients with kidney disease progress to renal failure each year, and more than 800,000 patients are living with renal failure requiring dialysis or transplant to sustain life. We are excited about the potential of Cholesterol Efflux MediatorTM VAR 200 to protect against kidney injury and reduce kidney disease progression.”
About ZyVersa Therapeutics, Inc.
ZyVersa (Nasdaq: ZVSA) is a clinical stage specialty biopharmaceutical company leveraging advanced, proprietary technologies to develop first-in-class drugs for patients with renal and inflammatory diseases who have significant unmet medical needs. The Company is currently advancing a therapeutic development pipeline with multiple programs built around its two proprietary technologies – Cholesterol Efflux Mediator™ VAR 200 for treatment of kidney diseases, and Inflammasome ASC Inhibitor IC 100, targeting damaging inflammation associated with numerous CNS and peripheral inflammatory diseases. For more information, please visit www.zyversa.com.
Certain statements contained in this press release regarding matters that are not historical facts, are forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995. These include statements regarding management’s intentions, plans, beliefs, expectations, or forecasts for the future, and, therefore, you are cautioned not to place undue reliance on them. No forward-looking statement can be guaranteed, and actual results may differ materially from those projected. ZyVersa Therapeutics, Inc (“ZyVersa”) uses words such as “anticipates,” “believes,” “plans,” “expects,” “projects,” “future,” “intends,” “may,” “will,” “should,” “could,” “estimates,” “predicts,” “potential,” “continue,” “guidance,” and similar expressions to identify these forward-looking statements that are intended to be covered by the safe-harbor provisions. Such forward-looking statements are based on ZyVersa’s expectations and involve risks and uncertainties; consequently, actual results may differ materially from those expressed or implied in the statements due to a number of factors, including ZyVersa’s plans to develop and commercialize its product candidates, the timing of initiation of ZyVersa’s planned preclinical and clinical trials; the timing of the availability of data from ZyVersa’s preclinical and clinical trials; the timing of any planned investigational new drug application or new drug application; ZyVersa’s plans to research, develop, and commercialize its current and future product candidates; the clinical utility, potential benefits and market acceptance of ZyVersa’s product candidates; ZyVersa’s commercialization, marketing and manufacturing capabilities and strategy; ZyVersa’s ability to protect its intellectual property position; and ZyVersa’s estimates regarding future revenue, expenses, capital requirements and need for additional financing.
New factors emerge from time-to-time, and it is not possible for ZyVersa to predict all such factors, nor can ZyVersa assess the impact of each such factor on the business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. Forward-looking statements included in this press release are based on information available to ZyVersa as of the date of this press release. ZyVersa disclaims any obligation to update such forward-looking statements to reflect events or circumstances after the date of this press release, except as required by applicable law.
This press release does not constitute an offer to sell, or the solicitation of an offer to buy, any securities.
Corporate, Media, and IR Contact: Karen Cashmere Chief Commercial Officer kcashmere@zyversa.com 786-251-9641
Phase 3 RESILIENT study of Tonmya met its primary endpoint of daily pain reduction (p=0.00005) and achieved statistically significant improvement on all six key pre-specified secondary endpoints with effect sizes on sleep, fatigue, FIQ-R symptoms and FIQ-R function ranging from 0.3 to 0.5
Cognitive dysfunction, or “brain fog,” nominally improved on FIQ-R memory item (p=0.001) where the patients rated their level of memory problems
NDA submission expected in the second half of 2024 following pre-NDA meeting with FDA scheduled for the second quarter of 2024
CHATHAM, N.J., March 11, 2024 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a biopharmaceutical company with marketed products and a pipeline of development candidates, today announced the presentation of additional efficacy data from RESILIENT, the second positive Phase 3 study evaluating Tonmya (also known as TNX-102 SL, cyclobenzaprine HCl sublingual tablets) for the management of fibromyalgia, at the 6th International Congress on Controversies in Fibromyalgia in Brussels, Belgium, March 7-8, 2024.
In presenting more detailed data from the RESILIENT study, Seth Lederman, M.D., President and Chief Executive Officer of Tonix Pharmaceuticals, said, “We previously reported statistically significant and clinically meaningful results in all six key secondary endpoints related to improving sleep quality, reducing fatigue, and improving overall fibromyalgia symptoms and function. We now report that the effect sizes of the five continuous key secondary outcomes measures ranged from 0.3 to 0.5. The results also showed that Tonmya treatment resulted in an improvement in cognitive dysfunction, or ‘brain fog’, measured by the change in the Fibromyalgia Impact Questionnaire-Revised (FIQ-R) memory item. The FIQ-R cognitive item showed nominal improvement in Tonmya-treated patients vs placebo-treated patients with a p=0.001 and effect size of 0.31. Together, we believe the activity of Tonmya on pain, sleep quality, fatigue and brain fog are indicative of broad-spectrum activity of Tonmya and suggest that Tonmya treats fibromyalgia at a syndromal level.”
As previously announced, RESILIENT met its pre-specified primary endpoint, significantly reducing daily pain compared to placebo (p=0.00005) in participants with fibromyalgia. RELIEF, the first Phase 3 trial of Tonmya 5.6 mg in fibromyalgia, was completed in December 2020. It also met its pre-specified primary endpoint of daily pain reduction compared to placebo (p=0.010). Tonix plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the second half of 2024 and has scheduled a pre-NDA meeting with FDA in the second quarter of 2024.
Tonmya was not associated with increases in systolic or diastolic blood pressure or body weight, nor were there any reported sexual side effects in the RESILIENT trial. In addition, when systematically investigated using the Changes in Sexual Functioning Questionnaire short form (CSFQ-14), women who received study drug had a higher CSFQ-14 total score relative to those who received placebo, which is consistent with improved sexual function.
Dr. Gregory Sullivan, Chief Medical Officer of Tonix Pharmaceuticals said, “These are important tolerability factors for fibromyalgia patients on long-term treatment with the three FDA-approved drugs, since weight gain and fatigue are associated with gabapentinoids, and negative sexual side effects, increased blood pressure and insomnia are associated with SNRIs.”
Dr. Lederman added, “We believe that the data from our two positive Phase 3 studies, with clinically meaningful separation from placebo on pain, sleep disturbance, and fatigue, supports the conclusion that fibromyalgia may be successfully treated with Tonmya 5.6 mg, and may provide the opportunity for Tonix to launch the first FDA-approved drug for fibromyalgia in more than a decade. We are excited to bring forward a new first-line treatment to fibromyalgia patients that offers broad symptom relief with favorable tolerability attributes for chronic use and adherence, which provides hope for the 6-12 million affected adults in the U.S.”
Dr. Sullivan added, “We believe that these broad-spectrum efficacy results will be important to fibromyalgia patients who struggle not only with pain, but also multiple other symptoms. We also believe the favorable tolerability and side effect profiles will be important to patients and doctors managing this debilitating condition on a long-term basis.”
About the Phase 3 RESILIENT Study
The RESILIENT study is a double-blind, randomized, placebo-controlled trial designed to evaluate the efficacy and safety of Tonmya (cyclobenzaprine HCl sublingual tablets) in the management of fibromyalgia. The two-arm trial randomized 457 participants in the U.S. across 33 sites. The first two weeks of treatment consist of a run-in period in which participants start on Tonmya 2.8 mg (1 tablet) or placebo. Thereafter, all participants increase their dose to Tonmya 5.6 mg (2 x 2.8 mg tablets) or two placebo tablets for the remaining 12 weeks. The primary endpoint is the daily diary pain severity score change (Tonmya 5.6 mg vs. placebo) from baseline to Week 14 (using the weekly averages of the daily numerical rating scale scores), analyzed by mixed model repeated measures with multiple imputation. The results showed that Tonmya treatment resulted in an improvement in cognitive dysfunction or ‘brain fog’ measured by the change in the FIQ-R memory item. The FIQ-R cognition item showed improvement in Tonmya treated patients vs placebo treated patients (LS mean (SE) difference of −0.8 (0.23); nominal p=0.001; effect size 0.31, no correction for multiple comparisons, mixed model repeated measures analysis). The Cohen’s d effect sizes (ESs) of the five continuous key secondary outcomes measures were: Fibromyalgia Impact Questionnaire-Revised (FIQ-R) – Symptoms domain ES = 0.44, FIQ-R-Function ES =0.30, PROMIS sleep disturbance ES =0.50, PROMIS Fatigue ES = 0.37 and Daily Sleep quality rating ES = 0.32. The most common adverse events were local administration site reactions that were transient and self-limited.
For more information, see ClinicalTrials.gov Identifier: NCT05273749.
About Fibromyalgia
Fibromyalgia is a chronic pain disorder that is understood to result from amplified sensory and pain signaling within the central nervous system. Fibromyalgia afflicts an estimated 6 million to 12 million adults in the U.S., the majority of whom are women. Symptoms of fibromyalgia include chronic widespread pain, nonrestorative sleep, fatigue, and morning stiffness. Other associated symptoms include cognitive dysfunction and mood disturbances, including anxiety and depression. Individuals suffering from fibromyalgia struggle with their daily activities, have impaired quality of life, and frequently are disabled. Physicians and patients report common dissatisfaction with currently marketed products.
About Tonmya* (also known as TNX-102 SL)
Tonmya is a centrally acting, non-opioid, non-addictive, bedtime medication. The tablet is a patented sublingual formulation of cyclobenzaprine hydrochloride developed for the management of fibromyalgia. In December 2023, the company announced highly statistically significant and clinically meaningful topline results in RESILIENT, a second positive Phase 3 clinical trial of Tonmya for the management of fibromyalgia. In the study, Tonmya met its pre-specified primary endpoint, significantly reducing daily pain compared to placebo (p=0.00005) in participants with fibromyalgia. Statistically significant and clinically meaningful results were also seen in all key secondary endpoints related to improving sleep quality, reducing fatigue and improving overall fibromyalgia symptoms and function. RELIEF, the first positive Phase 3 trial of Tonmya in fibromyalgia, was completed in December 2020. It met its pre-specified primary endpoint of daily pain reduction compared to placebo (p=0.010) and showed activity in key secondary endpoints.
*Tonmya™ is conditionally accepted by the U.S. Food and Drug Administration as the tradename for TNX-102 SL for the management of fibromyalgia. Tonmya has not been approved for any indication.
Tonix Pharmaceuticals Holding Corp.*
Tonix is a biopharmaceutical company focused on developing, licensing and commercializing therapeutics to treat and prevent human disease and alleviate suffering. Tonix’s development portfolio is focused on central nervous system (CNS) disorders. Tonix’s priority is to submit a New Drug Application (NDA) to the FDA in the second half of 2024 for Tonmya, a product candidate for which two positive Phase 3 studies have been completed for the management of fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction as well as fibromyalgia-type Long COVID. Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase) a biologic designed to treat cocaine intoxication with 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
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, 2022, as filed with the Securities and Exchange Commission (the “SEC”) on March 13, 2023, 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.
Data support evaluation of the effects of two weeks of TNX-102 SL therapy on severity of acute stress reaction (ASR) and frequency of acute stress disorder (ASD) and PTSD after civilian motor vehicle collision in upcoming U.S. DoD-FundedPhase 2 investigator-initiated OASIS trial
Nominal improvement in PTSD severity and measures of sleep quality at Week 4 in the HONOR study support development of bedtime TNX-102 SL therapy in the immediate aftermath of trauma
TNX-102 SL (Tonmya™) is also in late-stage development for the management of fibromyalgia for which NDA preparation is ongoing
CHATHAM, N.J., March 07, 2024 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a biopharmaceutical company with marketed products and a pipeline of development candidates, today announced the publication of a research paper in the Journal Psychiatry Research. The article titled, “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,” by Parmenter, et al. found that bedtime TNX-102 SL* treatment is well-tolerated and showed nominal improvement in PTSD severity and sleep quality measures in the first four weeks in military-related posttraumatic stress disorder (PTSD).1 The Company believes these findings suggest a potential role for short-term bedtime TNX-102 SL treatment in the immediate aftermath of traumatic events.
The data support the U.S. Department of Defense (DoD)-funded Phase 2 investigator-initiated OASIS trial to evaluate bedtime TNX-102 SL2 in reducing the severity of acute stress reaction (ASR) and the frequency of acute stress disorder (ASD) and PTSD. The IND supporting the OASIS trial was recently cleared,3 and the trial is expected to begin enrolling in the second quarter. The trial is sponsored by The University of North Carolina Institute for Trauma Recovery and supported by a $3 million grant from DoD. In the OASIS study, 14 days of bedtime TNX-102 SL 5.6 mg will be tested in the immediate aftermath of motor vehicle collision. The study will test the potential for TNX-102 SL treatment initiated within 24 hours of index trauma to target trauma-related sleep disturbance and other ASR symptoms to facilitate recovery from ASR and to prevent PTSD.
“There is an urgent need for interventions to reduce rates of ASD and PTSD in the immediate aftermath of trauma,”4 said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “We believe the results in the published paper suggest that bedtime TNX-102 SL has short-term activity on improving PTSD symptom severity and sleep quality in military-related PTSD. Poor sleep after trauma is a risk factor for progressing from ASD to PTSD. Therefore, poor sleep is not only a symptom of ASR, ASD and PTSD, but also a potential target of therapy.”
Dr. Gregory Sullivan, Chief Medical Officer of Tonix said, “Sleep disturbances are known to play a critical role in the development and maintenance of PTSD. The upcoming OASIS trial will test a 14-day short-course of bedtime TNX-102 SL therapy beginning within 24 hours of index trauma for effects on ASR symptoms and incidence of PTSD development. We are excited to test bedtime TNX-102 SL in the immediate aftermath of trauma to learn whether drug intervention reorients the trajectory of posttraumatic pathology from acute trauma to early recovery in the first few weeks.”
About TNX-102 SL (also known as Tonmya™ for the management of fibromyalgia)
PTSD: The Phase 3 HONOR study described in the published article was performed in military-related PTSD with the primary endpoint of improvement from baseline in Clinician-Administered PTSD Scale for DSM-5 (CAPS-5) total score at Week 12 comparing TNX-102 SL 5.6 mg and placebo. The study did not reach statistical significance on the primary endpoint. While there was nominal improvement by the Week 4 visit on CAPS-5 (p=0.019), the improvement relative to placebo was not sustained at Weeks 8 and 12. The CAPS-5 “sleep disturbance” item also showed nominal improvement at Week 4 (p=0.002), as well as at Week 8 (p=0.026), but not thereafter. The PROMIS Sleep Disturbance T-score also showed early nominal improvement with TNX-102 SL 5.6 mg at Week 4 (p=0.015). It is also notable that when the primary endpoint was analyzed for responder rate, defined as ≥50% improvement on CAPS-5 total score at Week 4, 38.4% of those on TNX-102 SL were responders versus 24.4% on placebo (p=0.019). TNX-102 SL was well-tolerated and the adverse events reported were similar to those seen in prior TNX-102 SL studies. There were three participants with serious adverse events (SAEs) reported during the study: two in the placebo group and one in the active group. None were deemed related to study drug. Administration site reactions were similar in profile to prior studies with TNX-102 SL, with oral numbness (hypoaesthesia) at the highest rate. These oral sensory adverse events (AE), oral numbness, oral tingling, and tongue discomfort were temporally-related to dosing and were rated as mild and transient (<60 min) in the majority of cases. No new safety signals were observed.
In addition to the Phase 3 HONOR study described in the published article1, Tonix has also studied TNX-102 SL in a Phase 2 (‘AtEase’) trial in military PTSD5 and in a Phase 3 (‘RECOVERY’) trial in civilian PTSD.6 Both studies were performed with the primary endpoint of CAPS-5 improvement at Week 12. AtEase compared bedtime TNX-102 SL at two doses (2.8 mg & 5.6 mg) and placebo. RECOVERY compared TNX-102 SL 5.6 mg and placebo. Neither study reached statistical significance on the primary endpoint.
Fibromyalgia: TNX-102 SL has shown positive results in two Phase 3 clinical trials for the management of fibromyalgia. Tonix plans to submit a New Drug Application to the U.S. Food and Drug Administration in the second half of 2024 under the 505(b)(2) regulatory pathway for Tonmya for the management of fibromyalgia.
Formulation Technology and Patents: TNX-102 SL is a patented sublingual tablet formulation of cyclobenzaprine hydrochloride which is designed for daily administration at bedtime with a proposed mechanism of improving sleep quality in fibromyalgia. TNX-102 SL provides rapid transmucosal absorption and reduced production of a long half-life active metabolite, norcyclobenzaprine, due to bypass of first-pass hepatic metabolism. As a multifunctional agent with potent binding and antagonist activities at the 5-HT2A-serotonergic, α1-adrenergic, H1-histaminergic, and M1-muscarinic cholinergic receptors, TNX-102 SL is in development as a daily bedtime treatment for fibromyalgia. TNX-102 SL is also in development for fibromyalgia-type Long COVID (formally known as post-acute sequelae of COVID-19 [PASC]), alcohol use disorder, and agitation in Alzheimer’s disease. The United States Patent and Trademark Office (USPTO) issued United States Patent No. 9636408 in May 2017, Patent No. 9956188 in May 2018, Patent No. 10117936 in November 2018, Patent No. 10,357,465 in July 2019, and Patent No. 10736859 in August 2020. The Protectic™ protective eutectic and Angstro-Technology™ formulation claimed in the patent are important elements of Tonix’s proprietary TNX-102 SL composition. These patents are expected to provide Tonmya, upon NDA approval, with U.S. market exclusivity until 2034/2035. In addition, Tonix has pending but not issued U.S. patent applications directed to the transmucosal absorption of cyclobenzaprine HCl, with U.S. market exclusivity expected until 2033, for treating depressive symptoms in fibromyalgia, with U.S. market exclusivity expected until 2032, and for treating pain in fibromyalgia with U.S. market exclusivity expected until 2041.
*TNX-102 SL has not been approved for any indication; name conditionally approved by FDA as Tonmya™ for the management of fibromyalgia
Tonix Press Release – September 27, 2023. “Tonix Pharmaceuticals Announces Department of Defense Grant to Support the University of North Carolina’s Proposed Investigator Sponsored OASIS Trial of TNX-102 SL for Treatment of Acute Stress Reaction, Acute Stress Disorder, and Posttraumatic Stress Disorder”. https://bit.ly/3T1LyIl
Tonix Press Release – Feb 12, 2024. “Tonix Pharmaceuticals Announces FDA IND Clearance for DoD Funded Trial of TNX-102 SL for the Reduction of Acute Stress Reaction and Prevention of PTSD” https://bit.ly/3TiQOsj.
Tonix Press Release – December 21, 2020, “Tonix Pharmaceuticals Reports Topline Results from Phase 3 RECOVERY Study of TNX-102 SL in PTSD and Outlines Future Development Plans” https://bit.ly/3uOgUu8
Tonix Pharmaceuticals Holding Corp.*
Tonix is a biopharmaceutical company focused on developing, licensing and commercializing therapeutics to treat and prevent human disease and alleviate suffering. Tonix’s development portfolio is focused on central nervous system (CNS) disorders. Tonix’s priority is to submit a New Drug Application (NDA) to the FDA in the second half of 2024 for Tonmya, a product candidate for which two positive Phase 3 studies have been completed for the management of fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction as well as fibromyalgia-type Long COVID. Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase) a biologic designed to treat cocaine intoxication with 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.
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, 2022, as filed with the Securities and Exchange Commission (the “SEC”) on March 13, 2023, 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.
Tonmya is a potential new first-line, centrally acting non-opioid analgesic for the management of fibromyalgia, supported by statistically significant results from two Phase 3 trials
New Drug Application (NDA) submission to the FDA planned for the second half of 2024
CHATHAM, N.J., March 06, 2024 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a biopharmaceutical company with marketed products and a pipeline of development candidates, announced today that EVERSANA®, a leading provider of commercialization services to the global life sciences industry, has been selected to support the launch strategy and commercial planning of Tonmya (also known as TNX-102 SL, cyclobenzaprine HCl sublingual tablets) in the U.S. Specifically, EVERSANA will work with Tonix to assess the fibromyalgia landscape and help plan an efficient go-to-market strategy.
“EVERSANA shares our commitment to delivering novel therapeutics to patients in need,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “We are excited to further refine our business strategy for the anticipated launch of Tonmya in 2025. It has been over a decade since patients suffering with fibromyalgia have been provided a new therapeutic option.”
“Today’s dynamic market requires experts that can help customers navigate the complexities of launch and commercial planning,” said Jim Lang, CEO, EVERSANA. “Together, we look forward to bringing a new treatment option to market for the millions of Americans suffering from this chronic disorder.”
Tonmya is a centrally acting, non-opioid medication. As previously announced, Tonix’s second positive Phase 3 study, RESILIENT, met its pre-specified primary endpoint, significantly reducing daily pain compared to placebo (p=0.00005) in participants with fibromyalgia. Statistically significant and clinically meaningful results (p=0.001 or better) were also seen in all key secondary endpoints related to improving sleep quality, reducing fatigue, and improving overall fibromyalgia symptoms and function.
Tonix plans to submit a New Drug Application to the U.S. Food and Drug Administration in the second half of 2024 for Tonmya for the management of fibromyalgia and has scheduled a Type B pre-NDA meeting with FDA for the second quarter of 2024.
About Tonmya* (also known as TNX-102 SL)
Tonmya is a centrally acting, non-opioid, non-addictive, bedtime medication. The tablet is a patented sublingual formulation of cyclobenzaprine hydrochloride developed for the management of fibromyalgia. In December 2023, the company announced highly statistically significant and clinically meaningful topline results in RESILIENT, a second positive Phase 3 clinical trial of Tonmya for the management of fibromyalgia. In the study, Tonmya met its pre-specified primary endpoint, significantly reducing daily pain compared to placebo (p=0.00005) in participants with fibromyalgia. Statistically significant and clinically meaningful results were also seen in all key secondary endpoints related to improving sleep quality, reducing fatigue and improving overall fibromyalgia symptoms and function. RELIEF, the first positive Phase 3 trial of Tonmya in fibromyalgia, was completed in December 2020. It met its pre-specified primary endpoint of daily pain reduction compared to placebo (p=0.010) and showed activity in key secondary endpoints.
*Tonmya™ is conditionally accepted by the U.S. Food and Drug Administration as the tradename for TNX-102 SL for the management of fibromyalgia. Tonmya has not been approved for any indication.
About EVERSANA®
EVERSANA is a leading independent provider of global services to the life sciences industry. The company’s integrated solutions are rooted in the patient experience and span all stages of the product life cycle to deliver long-term, sustainable value for patients, prescribers, channel partners and payers. The company serves more than 650 organizations, including innovative start-ups and established pharmaceutical companies, to advance life sciences solutions for a healthier world. To learn more about EVERSANA, visit eversana.com or connect through LinkedIn and X.
Tonix Pharmaceuticals Holding Corp.*
Tonix is a biopharmaceutical company focused on developing, licensing and commercializing therapeutics to treat and prevent human disease and alleviate suffering. Tonix’s development portfolio is focused on central nervous system (CNS) disorders. Tonix’s priority is to submit a New Drug Application (NDA) to the FDA in the second half of 2024 for Tonmya, a product candidate for which two positive Phase 3 studies have been completed for the management of fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction as well as fibromyalgia-type Long COVID. Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase) a biologic designed to treat cocaine intoxication with 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.
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, 2022, as filed with the Securities and Exchange Commission (the “SEC”) on March 13, 2023, 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.
TNX-1500, a third generation anti-CD40L mAb, was Fc-modified to preserve the activity and bioavailability of first generation mAbs while addressing their thrombosis risk
Sanofi projects its Fc-modified humanized anti-CD40L mAb frexalimab will exceed €5B per year in peak sales1 based on Phase 2 multiple sclerosis data recently published in the New England Journal of Medicine2
Topline results of TNX-1500 Phase 1 trial expected in the third quarter of 2024; clinical stage completed last month
TNX-1500 has multiple potential indications including solid organ and bone marrow transplantation and the treatment of autoimmune diseases: potential ‘pipeline in a product’
CHATHAM, N.J., March 05, 2024 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP), a biopharmaceutical company with marketed products and a pipeline of development candidates, today announced the results of modeling key human pharmacokinetic (PK) properties for TNX-1500 (Fc-modified humanized anti-CD40L monoclonal antibody, or mAb)* from animal studies. TNX-1500 is in development for the prevention of rejection in solid organ and bone marrow transplantation and for the treatment of autoimmune disorders.
“For more than 30 years, anti-CD40L therapy has shown promise in transplantation and the treatment of autoimmunity, but first-generation humanized mAbs were associated with an increased risk of thrombosis and second-generation agents had poor PK properties or reduced activity,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “Preclinical studies in non-human primates have shown that TNX-1500 maintains the activity of first generation mAbs, with reduced risk of thrombotic complications.3-5 Today we are announcing that modeling studies from animal PK data3, predict that a half-life of approximately three weeks for TNX-1500 in humans6,7, which supports monthly dosing. This PK analysis together with TNX-1500’s activity and tolerability in animals, suggests that the protein engineering of TNX-1500’s Fc region has achieved its design goals.”
Dr. Lederman continued, “Recently, positive clinical data have been reported by Sanofi, with its Fc-modified humanized anti-CD40L mAb frexalimab in treating relapsing multiple sclerosis with monthly i.v. or biweekly s.c. dosing regimens.2 Based on its results in multiple sclerosis, Sanofi projects that frexalimab will exceed €5B per year in peak sales1. TNX-1500 was designed to reduce binding to the Fc-receptor for IgG type 2a, or FcγR2a, which has been shown to play a role in the thrombosis associated with first-generation anti-CD40L mAbs, similar to frexalimab. In addition, Eledon Pharmaceuticals is in Phase 2 development for the prevention of rejection of kidney transplants with tegoprubart, a non-covalent dimer antibody with no heavy-light or heavy-heavy interchain disulfide bridges for the prevention of rejection of kidney transplants.”8
“Anti-CD40L therapy has multiple possible indications in addition to solid organ and bone marrow transplantation, including autoimmune diseases,” Dr. Lederman stated, “We look forward to the results of our Phase 1 PK and pharmacodynamic trial in the third quarter of 2024 and to advancing TNX-1500 as a promising candidate for prevention of organ and bone marrow transplant rejection and for treating autoimmune conditions.”
About the Translation of Human Pharmacokinetic Parameters from Animal Data
Results of a single dose PK study in animals were analyzed to predict human PK parameters. The PK study was conducted in six healthy cynomolgus monkeys at 30, 100 and 300 mg/kg and revealed linear PK across those doses, consistent with an antibody with no target mediated drug disposition.6,7 The half-life in cynomolgus monkeys is approximately 14 days. Human half-life prediction for TNX-1500 was based on allometric scaling for mAbs with linear PK.6 Clearance in cynomolgus animals was 6.24 ml/day (26.6% C.V.) and the predicted clearance in humans was 141 mL/day (C.V. 22.9%).6,7 The predicted human half-life for TNX 1500 is 23.8 days (range of 18.3 to 27.6 days) which supports monthly dosing. 6,7
About TNX-1500
TNX-1500 (Fc-modified humanized anti-CD40L mAb) is a humanized monoclonal antibody that binds and blocks the CD40-ligand (CD40L), also known as CD154. TNX-1500 is being developed for the prevention of allograft and xenograft rejection, for the prevention of graft-versus-host disease (GvHD) after hematopoietic stem cell transplantation (HCT) and for the treatment of autoimmune diseases. A first-in-human Phase 1 trial of TNX-1500 has completed the clinical phase. Topline results are expected in the third quarter of 2024. The primary objective of the Phase 1 trial is to assess the safety, tolerability, PK, and pharmacodynamics of intravenous (i.v.) TNX-1500. Eligible participants enrolled in the Phase 1 trial were distributed across three dosing cohorts (3 mg/kg, 10 mg/kg, and 30 mg/kg, respectively) and evaluated regularly over a 120-day period after dosing. The Phase 1 trial is intended to support dosing in a planned Phase 2 trial in kidney transplant recipients. Two published articles in the American Journal of Transplantation demonstrate TNX-1500 prevents rejection, prolongs survival and preserves graft function as a single agent or in combination with other drugs in non-human primate renal and heart allografts.3,4
About anti-CD40L Therapeutics in Development
No anti-CD40L mAb has been approved in any jurisdiction. In addition to TNX-1500, frexalimab and tegoprubart, tn03 fusion protein dazodalibep is being developed by Amgen (formerly Horizon Therapeutics Public Limited Company) for the treatment of Sjögren’s Syndrome.9,10 Dapirolizumab pegol, an anti-CD40L pegylated Fab, is being developed by UCB for the treatment of systemic lupus erythematosus.11
*TNX-1500 is an investigational new biologic and is not approved for any indication
Tonix is a biopharmaceutical company focused on developing, licensing and commercializing therapeutics to treat and prevent human disease and alleviate suffering. Tonix’s development portfolio is focused on central nervous system (CNS) disorders. Tonix’s priority is to submit a New Drug Application (NDA) to the FDA in the second half of 2024 for Tonmya1, a product candidate for which two positive Phase 3 studies have been completed for the management of fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction as well as fibromyalgia-type Long COVID. Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase) a biologic designed to treat cocaine intoxication with Breakthrough Therapy designation. Tonix’s immunology development portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft rejection and for the treatment of autoimmune diseases. Tonix also has product candidates in development in the areas of rare disease and infectious disease. Tonix Medicines, our commercial subsidiary, markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan nasal spray) 10 mg for the treatment of acute migraine with or without aura in adults.
*Tonix’s product development candidates are investigational new drugs or biologics and have not been approved for any indication.
1Tonmya™ is conditionally accepted by the U.S. Food and Drug Administration (FDA) as the tradename for TNX-102 SL for the management of fibromyalgia. Tonmya has not been approved for any indication.
Zembrace SymTouch and Tosymra are registered trademarks of Tonix Medicines. All other marks are property of their respective owners.
This press release and further information about Tonix can be found at www.tonixpharma.com.
Forward Looking Statements
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as “anticipate,” “believe,” “forecast,” “estimate,” “expect,” and “intend,” among others. These forward-looking statements are based on Tonix’s current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, risks related to the failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; risks related to the failure to successfully market any of our products; risks related to the timing and progress of clinical development of our product candidates; our need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party payor reimbursement; limited research and development efforts and dependence upon third parties; and substantial competition. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Tonix does not undertake an obligation to update or revise any forward-looking statement. Investors should read the risk factors set forth in the Annual Report on Form 10-K for the year ended December 31, 2022, as filed with the Securities and Exchange Commission (the “SEC”) on March 13, 2023, 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.
PDF Version•Up to 16 million adults in the US are living with a neurological disease, the leading cause of physical and cognitive disability, and 1.2 million new cases are diagnosed annually.•The review article summarizes data demonstrating that inflammation resulting from activation of more than one type of inflammasome contributes to development of neurological diseases and that ASC specks lead to their progression.•ZyVersa is developing IC 100, a monoclonal antibody targeting inflammasome ASC and ASC specks from multiple types of inflammasomes to block initiation and perpetuation of damaging inflammation.•IC 100 preclinical data demonstrate that it penetrates the brain, and that it has promising therapeutic potential for neurological diseases based on preclinical studies representative of multiple sclerosis, Alzheimer’s disease, traumatic brain injury, and spinal cord injury.WESTON, Fla., Feb. 14, 2024 (GLOBE NEWSWIRE) — ZyVersa Therapeutics, Inc. (Nasdaq: ZVSA or “ZyVersa”), a clinical stage specialty biopharmaceutical company developing first-in-class drugs for treatment of inflammatory and renal diseases, highlights data from a review article published in Nature Reviews Neurology. This article provides increasing evidence that activation of several types of inflammasomes and extracellular ASC specks contribute to the development and progression of multiple neurological diseases including Alzheimer’s disease (AD), Parkinson’s disease (PD), multiple sclerosis (MS), amyotrophic lateral sclerosis (ALS), epilepsy, traumatic brain injury (TBI), and stroke.In the paper titled, “Inflammasomes in neurological disorders — mechanisms and therapeutic potential,” the authors reviewed 297 published papers that included data from cell cultures, preclinical disease models, and human tissue analysis to summarize the current evidence for and understanding of inflammasome activation in various neurological diseases. Key learnings include:•Many neurological conditions involve an underlying chronic inflammasome-mediated inflammatory process that worsens the trajectory of these conditions.•Beyond NLRP3, evidence suggests that other inflammasomes, including but not restricted to NLRP1, NLRC4, and AIM2, and their downstream effectors contribute to neuropathology in AD, PD, ALS, MS, stroke, epilepsy, and TBI.•Inflammasome-induced release of ASC specks into the extracellular space seems to be important in the speed of neurodegeneration in conditions such as AD and PD.The authors concluded, “Use of inflammasome-targeted therapeutic approaches could improve existing therapeutic strategies for multiple neurological conditions.” To review the publication, Click Here.“We are thrilled to see the large number of studies summarized in the review article published in Nature Reviews Neurology that reinforce the role of multiple types of inflammasomes and extracellular ASC specks in the development and progression of numerous neurological diseases,” commented Stephen C. Glover, ZyVersa’s Co-founder, Chairman, CEO, and President. These studies, in combination with our own preclinical program, substantiate the rationale for targeting inflammasome ASC to inhibit multiple inflammasome pathways and to disrupt the function of ASC specks to control inflammation in numerous neurological diseases.” To review a white paper summarizing the mechanism of action and preclinical data for IC 100, Click Here.About Inflammasome ASC Inhibitor IC 100IC 100 is a novel humanized IgG4 monoclonal antibody that inhibits the inflammasome adaptor protein ASC. IC 100 was designed to attenuate both initiation and perpetuation of the inflammatory response. It does so by binding to a specific region of the ASC component of multiple types of inflammasomes, including NLRP1, NLRP2, NLRP3, NLRC4, AIM2, and Pyrin. Intracellularly, IC 100 binds to ASC monomers, inhibiting inflammasome formation, thereby blocking activation of IL-1β early in the inflammatory cascade. IC 100 also binds to ASC in ASC Specks, both intracellularly and extracellularly, further blocking activation of IL-1β and the perpetuation of the inflammatory response that is pathogenic in inflammatory diseases. Because active cytokines amplify adaptive immunity through various mechanisms, IC 100, by attenuating cytokine activation, also attenuates the adaptive immune response.About ZyVersa Therapeutics, Inc.ZyVersa (Nasdaq: ZVSA) is a clinical stage specialty biopharmaceutical company leveraging advanced, proprietary technologies to develop first-in-class drugs for patients with renal and inflammatory diseases who have significant unmet medical needs. The Company is currently advancing a therapeutic development pipeline with multiple programs built around its two proprietary technologies – Cholesterol Efflux Mediator™ VAR 200 for treatment of kidney diseases, and Inflammasome ASC Inhibitor IC 100, targeting damaging inflammation associated with numerous CNS and other inflammatory diseases. For more information, please visit www.zyversa.com.Cautionary Statement Regarding Forward-Looking StatementsCertain statements contained in this press release regarding matters that are not historical facts, are forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995. These include statements regarding management’s intentions, plans, beliefs, expectations, or forecasts for the future, and, therefore, you are cautioned not to place undue reliance on them. No forward-looking statement can be guaranteed, and actual results may differ materially from those projected. ZyVersa Therapeutics, Inc (“ZyVersa”) uses words such as “anticipates,” “believes,” “plans,” “expects,” “projects,” “future,” “intends,” “may,” “will,” “should,” “could,” “estimates,” “predicts,” “potential,” “continue,” “guidance,” and similar expressions to identify these forward-looking statements that are intended to be covered by the safe-harbor provisions. Such forward-looking statements are based on ZyVersa’s expectations and involve risks and uncertainties; consequently, actual results may differ materially from those expressed or implied in the statements due to a number of factors, including ZyVersa’s plans to develop and commercialize its product candidates, the timing of initiation of ZyVersa’s planned preclinical and clinical trials; the timing of the availability of data from ZyVersa’s preclinical and clinical trials; the timing of any planned investigational new drug application or new drug application; ZyVersa’s plans to research, develop, and commercialize its current and future product candidates; the clinical utility, potential benefits and market acceptance of ZyVersa’s product candidates; ZyVersa’s commercialization, marketing and manufacturing capabilities and strategy; ZyVersa’s ability to protect its intellectual property position; and ZyVersa’s estimates regarding future revenue, expenses, capital requirements and need for additional financing.New factors emerge from time-to-time, and it is not possible for ZyVersa to predict all such factors, nor can ZyVersa assess the impact of each such factor on the business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. Forward-looking statements included in this press release are based on information available to ZyVersa as of the date of this press release. ZyVersa disclaims any obligation to update such forward-looking statements to reflect events or circumstances after the date of this press release, except as required by applicable law.This press release does not constitute an offer to sell, or the solicitation of an offer to buy, any securities.Corporate, Media, and IR Contact:Karen CashmereChief Commercial Officerkcashmere@zyversa.com786-251-9641
IRVINE, Calif., Feb. 13, 2024 (GLOBE NEWSWIRE) — Eledon Pharmaceuticals, Inc. (“Eledon”) (NASDAQ: ELDN) today announced that the Company’s Compensation Committee granted 42,500 restricted stock units (RSUs) and stock options to purchase an aggregate of 90,000 common shares, at a per share exercise price of $1.77, the closing price of Eledon’s common stock on the grant date, to one employee. The RSUs and stock options were granted as inducements material to the new employee entering employment with Eledon in accordance with Nasdaq Listing Rule 5635(c)(4).
The RSUs vest fully on the one-year anniversary of the grant date. The stock options have a ten-year term and vest over four years, with 25% of the original number of shares vesting on the one-year anniversary of the grant date and 6.25% of the stock options vesting quarterly thereafter until fully vested on the fourth anniversary of the grant date. In each case, vesting is subject to the relevant employee’s continued service with Eledon on the applicable vesting date.
About Eledon Pharmaceuticals and tegoprubart
Eledon Pharmaceuticals, Inc. is a clinical stage biotechnology company that is developing immune-modulating therapies for the management and treatment of life-threatening conditions. The Company’s lead investigational product is tegoprubart, an anti-CD40L antibody with high affinity for CD40 Ligand, a well-validated biological target within the costimulatory CD40/CD40L cellular pathway. The central role of CD40L signaling in both adaptive and innate immune cell activation and function positions it as an attractive target for non-lymphocyte depleting, immunomodulatory therapeutic intervention. The Company is building upon a deep historical knowledge of anti-CD40 Ligand biology to conduct preclinical and clinical studies in kidney allograft transplantation, xenotransplantation, and amyotrophic lateral sclerosis (ALS). Eledon is headquartered in Irvine, California. For more information, please visit the Company’s website at www.eledon.com.
Follow Eledon Pharmaceuticals on social media: LinkedIn; Twitter
WESTON, Fla., Jan. 03, 2024 (GLOBE NEWSWIRE) — ZyVersa Therapeutics, Inc. (Nasdaq: ZVSA; “ZyVersa”), a clinical stage specialty biopharmaceutical company developing first-in-class drugs for treatment of patients with renal and inflammatory diseases who have unmet medical needs, announces that Stephen C. Glover, Co-Founder, Chairman, Chief Executive Officer, and President, has issued a Letter to Shareholders providing a corporate outlook with anticipated milestones for 2024. The full text of the letter follows.
A MESSAGE FROM OUR PRESIDENT AND CHIEF EXECUTIVE OFFICER
To my fellow shareholders,
Like so many in the biotech and pharmaceutical industry, we are looking forward to 2024 with great optimism for improved conditions in the financial markets and a milestone-rich year in the development of our lead renal and anti-inflammatory therapeutic candidates, which is expected to drive value for our shareholders. Our renal and inflammatory disease pipelines each have potential to address multiple indications. Combined, our two proprietary product platforms target a global total addressable market of around $75 billion.
On the financial front, various market watchers are seeing signals that the biotech and pharma investment environment is set to trend positively in 2024. Capital is again starting to flow to companies that are seeking to innovate through differentiated technologies or novel mechanisms of action. Near the end of 2023, we began to witness an increase in financings and an uptick in M&A across multiple sectors from inflammatory diseases to oncology. These were driven by investors targeting high upside opportunities and large pharma seeking to re-energize their product pipelines. Our team at ZyVersa shares this optimism, anticipating improved conditions as we focus our efforts on developing innovative, disease-modifying treatments to help improve patients’ quality of life.
We are looking forward to an active first quarter of this new year, including initiation of our first clinical trial for our Cholesterol Efflux MediatorTM VAR 200. VAR 200 is designed to ameliorate renal lipid accumulation that damages the kidney’s filtration system. Currently there are no approved treatments that target lipid accumulation, which is known to contribute to structural damage, proteinuria, and progression of kidney disease. The current standard of care addresses glomerular hypertension and inflammation.
We are on schedule to initiate our Phase 2a clinical trial with VAR 200 in patients with diabetic kidney disease (DKD) in the first quarter of 2024. This will be an open label trial for real-time data reads. Initial data is anticipated to be available mid-year. DKD represents a significant area of unmet need, as it is the leading cause of end-stage kidney disease, requiring dialysis or kidney transplant for survival. Although substantial progress has been made in slowing progression of DKD with introduction of ACE inhibitors, ARBs, and most recently SGLT2 inhibitors, patients are still progressing to end-stage renal disease. We are optimistic that the protection against kidney injury, fibrosis, and disease progression that was demonstrated with VAR 200 in animal models of three different kidney diseases will translate to humans. Data from our initial Phase 2a trial will not only provide proof-of-concept for VAR 200 in renal patients, it will also provide valuable insights for designing future trials with VAR 200 in other planned renal indications, including two rare kidney diseases, focal segmental glomerulosclerosis (FSGS) and Alport syndrome.
We are equally excited about our progress in the development of our proprietary Inflammasome ASC Inhibitor IC 100, which is designed to block initiation and perpetuation of damaging inflammation that contributes to a multitude of inflammatory diseases. A growing body of scientific literature reinforces the central role of multiple types of inflammasomes in the development and progression of inflammatory diseases, and we continue to report on these developments as they are published. By inhibiting Inflammasome ASC rather than a sensor molecule such as NLRP3, IC 100 targets multiple types of inflammasome pathways (including NLRP3, NLRP1, and AIM2), which is expected to provide better control of damaging inflammation and its perpetuation.
The IC 100 preclinical program is nearing completion and IND submission is planned for the fourth quarter of 2024, with Phase 1 trial initiation expected shortly thereafter.
Two Platforms, Singular Vision
We view 2024 as a potentially transformative year for ZyVersa based on the value-building milestones that we expect to achieve over the next 12 to 15 months. I look forward to working with my leadership team and fellow Board members to execute a business and clinical strategy that has potential to position ZyVersa as a leading and innovative company developing transformative drugs for underserved patients with renal and inflammasome-mediated inflammatory diseases.
Though our platforms are independent from each other, our mission is singular – to develop drug therapies that can restore health and improve quality of life for patients living with the often debilitating symptoms of renal and inflammatory diseases. We look forward to embarking on what we believe will be a productive 2024. We thank you for your continued support.
Sincerely, Stephen C. Glover Co-Founder, Chairman, Chief Executive Officer, and President ZyVersa Therapeutics
About ZyVersa Therapeutics, Inc.
ZyVersa is a clinical stage specialty biopharmaceutical company leveraging advanced, proprietary technologies to develop first-in-class drugs. Our focus is on patients with renal or inflammatory diseases who have significant unmet medical needs. Our development pipeline includes phase clinical stage Cholesterol Efflux MediatorTM VAR 200 in development to alleviate damaging accumulation of cholesterol and lipids in the filtering system of the kidneys. The lead indication is treatment of rare kidney disease, focal segmental glomerulosclerosis. VAR 200 has potential to treat other kidney diseases, including Alport syndrome and diabetic kidney disease. ZyVersa’s pipeline also includes proprietary inflammasome ASC inhibitor IC 100 that blocks initiation and perpetuation of damaging inflammation associated with a multitude of inflammatory diseases. IC 100 has potential to treat many different CNS and other inflammatory diseases. For more information, please visit www.zyversa.com.
Certain statements contained in this press release regarding matters that are not historical facts, are forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995. These include statements regarding management’s intentions, plans, beliefs, expectations, or forecasts for the future, and, therefore, you are cautioned not to place undue reliance on them. No forward-looking statement can be guaranteed, and actual results may differ materially from those projected. ZyVersa Therapeutics, Inc (“ZyVersa”) uses words such as “anticipates,” “believes,” “plans,” “expects,” “projects,” “future,” “intends,” “may,” “will,” “should,” “could,” “estimates,” “predicts,” “potential,” “continue,” “guidance,” and similar expressions to identify these forward-looking statements that are intended to be covered by the safe-harbor provisions. Such forward-looking statements are based on ZyVersa’s expectations and involve risks and uncertainties; consequently, actual results may differ materially from those expressed or implied in the statements due to a number of factors, including ZyVersa’s plans to develop and commercialize its product candidates, the timing of initiation of ZyVersa’s planned preclinical and clinical trials; the timing of the availability of data from ZyVersa’s preclinical and clinical trials; the timing of any planned investigational new drug application or new drug application; ZyVersa’s plans to research, develop, and commercialize its current and future product candidates; the clinical utility, potential benefits and market acceptance of ZyVersa’s product candidates; ZyVersa’s commercialization, marketing and manufacturing capabilities and strategy; ZyVersa’s ability to protect its intellectual property position; and ZyVersa’s estimates regarding future revenue, expenses, capital requirements and need for additional financing. A discussion of these and other factors, including risks and uncertainties with respect to ZyVersa, is set forth in ZyVersa’s filings with the Securities and Exchange Commission, including ZyVersa’s Annual Report on Form 10-K and its Quarterly Reports on Form 10-Q.
New factors emerge from time-to-time, and it is not possible for ZyVersa to predict all such factors, nor can ZyVersa assess the impact of each such factor on the business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. Forward-looking statements included in this press release are based on information available to ZyVersa as of the date of this press release. ZyVersa disclaims any obligation to update such forward-looking statements to reflect events or circumstances after the date of this press release, except as required by applicable law.
Corporate and IR Contact Karen Cashmere Chief Commercial Officer kcashmere@zyversa.com 786-251-9641
Media Contacts Casey McDonald cmcdonald@tiberend.com 646-577-8520
Dave Schemelia Dschemelia@tiberend.com 609-468-9325
Phase 3 RESILIENT study of TNX-102 SL successfully demonstrated daily pain reduction over placebo (primary endpoint, p = 0.00005)
All six key secondary endpoints, including patient global impression, fibromyalgia-specific symptoms and dysfunction, fatigue and sleep measures were significantly improved (all p ≤ 0.001)
Positive results support planned New Drug Application (NDA) submission to the FDA in the second half of 2024 An estimated 6 million to 12 million adults in the U.S. are living with fibromyalgia, the majority of whom are women
CHATHAM, N.J., Dec. 20, 2023 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a biopharmaceutical company with marketed products and a pipeline of development candidates, today announced that the Phase 3 RESILIENT study evaluating TNX-102 SL (cyclobenzaprine HCl sublingual tablets) met its pre-specified primary endpoint in the second of two positive Phase 3 clinical trials, significantly reducing daily pain compared to placebo (p=0.00005) in participants with fibromyalgia (Table 1). Statistically significant and clinically meaningful results were also seen in all key secondary endpoints related to improving sleep quality, reducing fatigue, and improving overall fibromyalgia symptoms and function. Additionally, as it relates to improving daily pain, treatment with TNX-102 SL showed a robust and clinically meaningful analgesic effect size of 0.38, with rapid onset of action, separating from placebo for each week of the study. TNX-102 SL was well tolerated with an adverse event profile comparable to prior studies, and no new safety signals were observed. Tonix plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the second half of 2024 for TNX-102 SL for the management of fibromyalgia. An estimated 6 million to 12 million U.S. adults are living with fibromyalgia, the majority of whom are women.
TNX-102 SL is a tablet formulation containing 2.8 mg cyclobenzaprine HCl and is a novel, centrally-acting, non-opioid analgesic, designed to be taken once daily at bedtime for the management of fibromyalgia. RESILIENT was a 14-week randomized, double-blind, placebo-controlled trial of TNX-102 SL 5.6 mg, in which 457 participants with fibromyalgia were randomized in a 1:1 ratio to TNX-102 SL or placebo across 33 sites in the U.S. All participants received one 2.8 mg tablet of TNX-102 SL (2.8 mg) or placebo for the first 2 weeks, which was increased to two 2.8 mg tablets of TNX-102 SL (5.6 mg) or placebo for the remaining 12 weeks.
In December 2020, Tonix reported positive results from the first Phase 3 RELIEF study of TNX-102 SL 5.6 mg for the management of fibromyalgia. The RELIEF study met its pre-specified primary endpoint, significantly reducing daily pain compared to placebo (p=0.010) in participants with fibromyalgia, and showing activity in key secondary endpoints.
“We believe that the positive results of RESILIENT and RELIEF show that fibromyalgia can be successfully treated by TNX-102 SL 5.6 mg and may provide the opportunity for Tonix to have the first FDA-approved drug for fibromyalgia in more than a decade,” said Seth Lederman, M.D., President and Chief Executive Officer of Tonix Pharmaceuticals. “We are now an important step closer to bringing a new, first-line treatment to fibromyalgia patients that offers broad symptom relief and favorable tolerability for chronic use and adherence. We believe that we are well positioned to submit an NDA to the FDA under the 505(b)(2) regulatory approval pathway in the second half of 2024, and are on track to supply the U.S. market upon FDA approval.”
Table 1. Results of Primary and Secondary Endpoints for the Phase 3 RESILIENT Study of TNX-102 SL
Outcome Measure at Week 14
Intent-to-Treat Analysis1
P-value
Primary Endpoint
Daily Pain Diary, NRS
Mean Change from Baseline2
0.00005*
Key Secondary Endpoints
Non-specific
Patient Global Impression of Change
Proportion “Much” or “Very Much Improved”3
<0.001*
Fibromyalgia Syndrome-Related
FIQ-R Symptom Domain
Mean Change from Baseline
<0.001*
FIQ-R Function Domain
Mean Change from Baseline
0.001*
PROMIS Sleep Disturbance
Mean Change from Baseline
<0.001*
PROMIS Fatigue
Mean Change from Baseline
<0.001*
Daily Sleep Quality Diary, NRS
Mean Change from Baseline
<0.001*
Abbreviations: FIQ-R = Fibromyalgia Impact Questionnaire – Revised; NRS = Numeric Rating Scale; PROMIS = Patient-Reported Outcomes Measurement Information System
*Statistically significant; to control for overall type 1 error, a pre-specified, serial gatekeeping procedure was utilized.
1Analysis by mixed model repeated measures with multiple imputation unless otherwise indicated.
2Primary endpoint analysis for FDA approvals of Cymbalta®and Lyrica® in fibromyalgia.
3Pearson’s chi-squared test responder analysis, with missing data considered non-responders
“These data are terrific news for patients with fibromyalgia,” said Daniel J. Clauw, M.D., Professor of Anesthesiology, Medicine and Psychiatry at the University of Michigan. “Despite approved medications, there remains a need for new treatment options to better address the quality of life impacts many fibromyalgia patients experience on a chronic basis. TNX-102 SL is a non-opioid, centrally-acting analgesic, the active ingredient of which has a known, favorable safety profile from decades of use. The fact that cyclobenzaprine was also beneficial in many other key symptom domains, including sleep quality, sleep disturbance and fatigue, will be appreciated by fibromyalgia patients that struggle with not just pain but multiple other symptoms.”
“These positive data from RESILIENT and previously with RELIEF, with remarkable separation from placebo on pain, sleep, and fatigue, add support to TNX-102 SL’s proposed mechanism of improving sleep quality to improve the syndromal effects of fibromyalgia,” commented Gregory Sullivan, M.D., Chief Medical Officer of Tonix Pharmaceuticals. “The sublingual formulation of TNX-102 SL, which uses our proprietary Protectic® and Angstro® technologies, is integral to our treatment paradigm. These technologies enable transmucosal delivery of cyclobenzaprine with distinctive pharmacokinetic properties that include rapid absorption after dosing and bypass of first-pass hepatic metabolism. I would like to thank the RESILIENT study participants and their families and caregivers, as well as the investigators and their hard-working staff who all made this a highly successful trial.”
Summary of Topline Results of the RESILIENT Study
The RESILIENT study achieved statistical significance on the pre-specified primary efficacy endpoint: change from baseline in the weekly average of daily diary pain severity numerical rating scale (NRS) scores for TNX-102 SL 5.6 mg (LS mean [SE]: -1.8 [0.12] units) versus placebo (-1.2 [0.12] units), analyzed by mixed model repeated measures with multiple imputation (LS mean [SE] difference: -0.7 [0.16] units, p=0.00005, Table 1). In addition, all pre-specified sensitivity analyses of the primary endpoint were statistically significant (p<0.001). Figure 1 shows reduction in pain across all weeks of the 14-week study, with nominal p<0.01 for every week. Note the rapid onset of action with separation from placebo at Week 1 was sustained throughout all weeks of dosing.
Abbreviations: LS = least squares; NRS = numerical rating scale; SE = standard error
The statistically significant improvement in pain is further substantiated when diary pain was analyzed by another standard statistical approach, a 30 percent responder analysis, with 45.9% on active and 27.1% on placebo having a 30 percent or greater reduction in pain (Pearson Chi-Squared Test; difference in proportions [95% CI]: 18.8% [10.1%, 27.4%]; nominal p<0.001).
TNX-102 SL showed statistical significance (p≤0.001) on all six pre-specified key secondary efficacy outcome measures (Table 1).
Consistent with the proposed mechanism that TNX-102 SL acts in fibromyalgia through improving sleep quality, TNX-102 SL showed statistically significant improvement of sleep by two main measures. For the daily diary sleep quality ratings, improvement in sleep quality for TNX-102 SL (-1.8 [0.12] units) was significantly greater than that of placebo (-1.2 [0.12] units; LS mean [SE] difference from placebo: -0.6 [0.17] units; p<0.001). For the PROMIS Sleep Disturbance instrument, TNX-102 SL also demonstrated significantly greater improvement over placebo on T-scores (LS mean [SE] difference from placebo: -4.2 [0.79] units; p<0.001). Fatigue is another cardinal symptom of fibromyalgia and has a major impact on quality of life. TNX-102 SL showed significant improvement over placebo on the PROMIS Fatigue instrument T-scores (-3.0 [0.77] units; p<0.001).
The Fibromyalgia Impact Questionnaire – Revised (FIQ-R) is a 21-item self-rated instrument that assesses level of function, overall impact, and symptoms due to fibromyalgia, and the symptoms and function domains were key secondary endpoints in RESILENT. At Week 14 on the FIQ-R Symptoms domain, there was significantly greater improvement with TNX-102 SL than with placebo (LS mean [SE] difference from placebo: -7.7 [1.62], p<0.001). Similarly, TNX-102 SL resulted in greater improvement on FIQ-R Function (LS mean [SE] difference from placebo: -5.4 [1.66], p=0.001). Although not a key secondary efficacy endpoint, TNX-102 SL also separated from placebo on the FIQ-R Impact domain (nominal p=0.001). These results, along with the robust effects on improving sleep and fatigue, suggests broad symptomatic coverage of the syndrome of fibromyalgia.
Safety Results of the Phase 3 RESILIENT Study
In the RESILIENT study, TNX-102 SL was well tolerated and consistent with prior trials, with no new safety signals observed. Among participants randomized to the TNX-102 SL and placebo arms, 81.0% and 79.2%, respectively, completed the 14-week dosing period. As expected based on prior TNX-102 SL studies, administration site reactions were the most commonly reported adverse events and were higher in the TNX-102 SL treatment group (Table 2). Hypoaesthesia oral and paraesthesia oral, or tongue and mouth numbness or tingling, product taste abnormal (typically a bitter aftertaste upon dosing), and tongue discomfort were local effects nearly always temporally related to dose administration and transiently expressed (<60 minutes) in most occurrences. The only treatment-emergent adverse events that occurred at a rate of 3.0% or greater in either arm were these four oral adverse events, along with COVID-19, somnolence, and headache (Table 2). Adverse events resulted in premature study discontinuation in 6.1% of those who received TNX-102 SL compared with 3.5% of placebo recipients. There were a total of seven serious adverse events in five patients, five of which were experienced by three patients in the placebo arm, and two of which were in the TNX-102 SL arm. Of the two in the TNX-102 SL arm, one was renal cancer, deemed unrelated to study drug, and the other was acute pancreatitis with onset 14 days after dosing was completed and reported as possibly related to study drug.
Table 2. Treatment-Emergent Adverse Events at a Rate of 3% or Greater in Either Treatment Arm
TNX-102 SL (N=231)
Placebo (N=226)
Total (N=457)*
Administration Site Reactions
N
%
N
%
N
%
Hypoaesthesia oral
55
23.8%
1
0.4%
56
12.3%
Product taste abnormal
27
11.7%
2
0.9%
29
6.3%
Paraesthesia oral
16
6.9%
2
0.9%
18
3.9%
Tongue discomfort
16
6.9%
0
0.0%
16
3.5%
Systemic
Adverse Events
N
%
N
%
N
%
COVID-19
10
4.3%
7
3.1%
17
3.7%
Somnolence
7
3.0%
3
1.3%
10
2.2%
Headache
7
3.0%
4
1.8%
11
2.4%
*Safety Population
The Changes in Sexual Functioning Questionnaire short form (CSFQ-14) served as a safety measure for assessing potential adverse effects on sexual functioning. In females, the total score on the CSFQ-14 at Week 14 improved (indicating better sexual functioning) in the TNX-102 SL group compared with placebo (nominal p=0.010 by analysis of covariance). This potentially indicates an important tolerability advantage over pharmacotherapeutics which potently inhibit reuptake of serotonin. The low percentage of males in the safety population (<5%) did not allow meaningful analysis of the CSFQ-14 data.
About the Phase 3 RESILIENT Study
The RESILIENT study is a double-blind, randomized, placebo-controlled trial designed to evaluate the efficacy and safety of TNX-102 SL (cyclobenzaprine HCl sublingual tablets) in the management of fibromyalgia. The two-arm trial randomized 457 participants in the U.S. across 33 sites. The first two weeks of treatment consist of a run-in period in which participants start on TNX-102 SL 2.8 mg (1 tablet) or placebo. Thereafter, all participants increase their dose to TNX-102 SL 5.6 mg (2 x 2.8 mg tablets) or two placebo tablets for the remaining 12 weeks. The primary endpoint is the daily diary pain severity score change (TNX-102 SL 5.6 mg vs. placebo) from baseline to Week 14 (using the weekly averages of the daily numerical rating scale scores), analyzed by mixed model repeated measures with multiple imputation.
For more information, see ClinicalTrials.gov Identifier: NCT05273749.
About Fibromyalgia
Fibromyalgia is a chronic pain disorder that is understood to result from amplified sensory and pain signaling within the central nervous system. Fibromyalgia afflicts an estimated 6 million to 12 million adults in the U.S., the majority of whom are women. Symptoms of fibromyalgia include chronic widespread pain, nonrestorative sleep, fatigue, and morning stiffness. Other associated symptoms include cognitive dysfunction and mood disturbances, including anxiety and depression. Individuals suffering from fibromyalgia struggle with their daily activities, have impaired quality of life, and frequently are disabled. Physicians and patients report common dissatisfaction with currently marketed products.
About TNX-102 SL
TNX-102 SL is a patented sublingual tablet formulation of cyclobenzaprine hydrochloride which provides rapid transmucosal absorption and reduced production of a long half-life active metabolite, norcyclobenzaprine, due to bypass of first-pass hepatic metabolism. As a multifunctional agent with potent binding and antagonist activities at the 5-HT2A-serotonergic, α1-adrenergic, H1-histaminergic, and M1-muscarinic cholinergic receptors, TNX-102 SL is in development as a daily bedtime treatment for fibromyalgia, fibromyalgia-type Long COVID (formally known as post-acute sequelae of COVID-19 [PASC]), alcohol use disorder and agitation in Alzheimer’s disease. Dr. Harvey Moldofsky, Professor Emeritus of Psychiatry and Medicine at the University of Toronto, founding Director of the University of Toronto Center for Sleep and Chronobiology, first recognized the central role of non-restorative sleep in the pathogenesis of fibromyalgia1,2. Our program is based on the subsequent pioneering work of Dr. Iredell W. Iglehart III, who recognized that a sleep-focused cyclobenzaprine treatment protocol had the potential to target non-restorative sleep and lead to improvement of fibromyalgia at the syndromal level3. Teams led by Giorgio Reiner at APR Applied Pharma Research S.A., a wholly-owned subsidiary of Relief Therapeutics Holding AG, and Professor Marino Nebuloni and Patrizia Colombo at Redox Analytical Science Srl invented and developed these underlying technologies in collaboration with Tonix. The United States Patent and Trademark Office (USPTO) issued United States Patent No. 9636408 in May 2017, Patent No. 9956188 in May 2018, Patent No. 10117936 in November 2018, Patent No. 10,357,465 in July 2019, and Patent No. 10736859 in August 2020. The Protectic™ protective eutectic and Angstro-Technology™ formulation claimed in the patent are important elements of Tonix’s proprietary TNX-102 SL composition. These patents are expected to provide TNX-102 SL, upon NDA approval, with U.S. market exclusivity until 2034/2035. In addition, Tonix has pending but not issued U.S. patent applications directed to the transmucosal absorption of CBP-HCl, with U.S. market exclusivity expected until 2033, for treating major depressive disorder in fibromyalgia, with U.S. market exclusivity expected until 2032, and for treating pain in fibromyalgia with U.S. market exclusivity expected until 2041.
1Moldofsky H et al, Psychosom Med 1975;37:341-51. 2Moldofsky H and Scarisbrick P. Psychosom Med 1976;38:35-44. 3Iglehart IW. 2003; US Patent 6,541,523.
Tonix Pharmaceuticals Holding Corp.*
Tonix is a biopharmaceutical company focused on commercializing, developing, discovering and licensing therapeutics to treat and prevent human disease and alleviate suffering. Tonix Medicines, our commercial subsidiary, markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan nasal spray) 10 mg under a transition services agreement with Upsher-Smith Laboratories, LLC from whom the products were acquired on June 30, 2023. Zembrace SymTouch and Tosymra are each indicated for the treatment of acute migraine with or without aura in adults. Tonix’s development portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious disease product candidates. Tonix’s CNS development portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead development CNS candidate, TNX-102 SL (cyclobenzaprine HCl sublingual tablet), has completed two positive Phase 3 studies for the management of fibromyalgia. Tonix intends to meet with the FDA and submit an NDA for the approval of TNX-102 SL for the management of fibromyalgia in the second half of 2024. TNX-102 SL is also being developed to treat fibromyalgia-type Long COVID, a chronic post-acute COVID-19 condition, and topline results were reported in the third quarter of 2023. TNX-1900 (intranasal potentiated oxytocin) is being studied in binge eating disorder, pediatric obesity, bone health in autism and social anxiety disorder by academic collaborators under investigator-initiated INDs. TNX-1300 (cocaine esterase) is a biologic designed to treat cocaine intoxication and has been granted Breakthrough Therapy designation by the FDA. A Phase 2 study of TNX-1300 is expected to be initiated in the first quarter of 2024 Tonix’s rare disease development portfolio includes TNX-2900 (intranasal potentiated oxytocin) for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan Drug designation by the FDA. Tonix’s immunology development portfolio includes biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 was initiated in the third quarter of 2023. Tonix’s infectious disease pipeline includes TNX-801, a vaccine in development to prevent smallpox and mpox. TNX-801 also serves as the live virus vaccine platform or recombinant pox vaccine platform for other infectious diseases, including TNX-1800, in development as a vaccine to protect against COVID-19. During the fourth quarter of 2023, TNX-1800 was selected by the U.S. National Institutes of Health (NIH), National Institute of Allergy and Infectious Diseases (NIAID) Project NextGen for inclusion in Phase 1 clinical trials. The infectious disease development portfolio also includes TNX-3900 and TNX-4000, which are classes of broad-spectrum small molecule oral antivirals.
*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.
About Redox – Analytical Science Srl
Redox is an independent CRO company headquartered in Monza- Italy with R&D activities and customer analytical support to pharmaceutical companies for more than 30 years. From more than 25 years the analytical activities have been certified by national and international agencies (European Medicines Agency, the Italian Medicines Agency (AIFA), FDA, and etc). One of the main activities is the development of new drug products in order to improve the pharmaceutical actions and at the same time improve the stability and reducing the cost of the new drug substance. Several unique and sophisticated analytical techniques and equipment are used in support to research and development strategies with the focus to reach the best and effective pharmaceutical formulation in a short time frame. More than 30 professional people are dedicated to our efforts and many projects are ongoing in collaboration with the pharmaceutical industry as well as with Italian and international Universities.
Further information about Redox can be found at www.labredox.com.
About APR Applied Pharma Research S.A., a wholly-owned subsidiary of Relief Therapeutics Holding AG
Relief Therapeutics is a commercial-stage biopharmaceutical company committed to advancing treatment paradigms and delivering improvements in efficacy, safety, and convenience to benefit the lives of patients living with select specialty and rare diseases. Relief Therapeutics’ portfolio offers a balanced mix of marketed, revenue-generating products, our proprietary, globally patented Physiomimic™ and TEHCLO™ platform technologies and a targeted clinical development pipeline consisting of risk-mitigated assets focused in three core therapeutic areas: rare metabolic disorders, rare skin diseases and rare respiratory diseases. In addition, Relief Therapeutics is commercializing several legacy products via licensing and distribution partners. Relief Therapeutics’ mission is to provide therapeutic relief to those suffering from rare diseases and is being advanced by an international team of well-established, experienced biopharma industry leaders with extensive research, development and rare disease expertise. Relief Therapeutics is headquartered in Geneva, with additional offices in Balerna, Switzerland, Offenbach am Main, Germany and Monza, Italy. Relief Therapeutics is listed on the SIX Swiss Exchange under the symbol RLF.
Further information about APR can be found at www.relieftherapeutics.com or by following Relief Therapeutics on LinkedIn and Twitter.
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, 2022, as filed with the Securities and Exchange Commission (the “SEC”) on March 13, 2023, 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.
CHATHAM, N.J., Dec. 11, 2023 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP), a biopharmaceutical company with marketed products and a pipeline of development candidates, announced today that Seth Lederman M.D., Chief Executive Officer of Tonix Pharmaceuticals, will present at the National Academies of Sciences, Engineering, and Medicine Committee on the Current State of Research, Development, and Stockpiling of Smallpox Medical Countermeasures public meeting on Thursday, December 14, 2023 via Zoom. Dr. Lederman will participate in a panel discussion on Vaccine Research & Development taking place from 2:00 – 2:45 p.m. ET.
Discussions will explore lessons learned from the recent COVID-19 pandemic and mpox multi-country outbreak to inform an evaluation of the current state of research, development, and stockpiling of smallpox readiness and response measures.
A webcast of the meeting can be found here and will be available under the IR Events tab of the Tonix website at www.tonixpharma.com following the presentation.
Tonix Pharmaceuticals Holding Corp.*
Tonix is a biopharmaceutical company focused on commercializing, developing, discovering and licensing therapeutics to treat and prevent human disease and alleviate suffering. Tonix Medicines, our commercial subsidiary, markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan nasal spray) 10 mg under a transition services agreement with Upsher-Smith Laboratories, LLC from whom the products were acquired on June 30, 2023. Zembrace SymTouch and Tosymra are each indicated for the treatment of acute migraine with or without aura in adults. Tonix’s development portfolio is composed of central nervous system (CNS), rare disease, immunology and infectious disease product candidates. Tonix’s CNS development portfolio includes both small molecules and biologics to treat pain, neurologic, psychiatric and addiction conditions. Tonix’s lead development CNS candidate, TNX-102 SL (cyclobenzaprine HCl sublingual tablet), is in mid-Phase 3 development for the management of fibromyalgia, having completed the clinical phase of a potentially confirmatory Phase 3 study in the fourth quarter of 2023, with topline data expected in late December 2023. TNX-102 SL is also being developed to treat fibromyalgia-type Long COVID, a chronic post-acute COVID-19 condition, and topline results were reported in the third quarter of 2023. TNX-1900 (intranasal potentiated oxytocin) is in development as a preventive treatment in chronic migraine, with analysis of topline data from a Phase 2 proof-of-concept study expected to be completed late December 2023. TNX-1900 is also being studied in binge eating disorder, pediatric obesity and social anxiety disorder by academic collaborators under investigator-initiated INDs. TNX-1300 (cocaine esterase) is a biologic designed to treat cocaine intoxication and has been granted Breakthrough Therapy designation by the FDA. A Phase 2 study of TNX-1300 is expected to be initiated in the fourth quarter of 2023. Tonix’s rare disease development portfolio includes TNX-2900 (intranasal potentiated oxytocin) for the treatment of Prader-Willi syndrome. TNX-2900 has been granted Orphan Drug designation by the FDA. Tonix’s immunology development portfolio includes biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is a humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft rejection and for the treatment of autoimmune diseases. A Phase 1 study of TNX-1500 was initiated in the third quarter of 2023. Tonix’s infectious disease pipeline includes TNX-801, a vaccine in development to prevent smallpox and mpox. TNX-801 also serves as the live virus vaccine platform or recombinant pox vaccine platform for other infectious diseases, including TNX-1800, in development as a vaccine to protect against COVID-19. During the fourth quarter of 2023, TNX-1800 was selected by the U.S. National Institutes of Health (NIH), National Institute of Allergy and Infectious Diseases (NIAID) Project NextGen for inclusion in Phase 1 clinical trials. The infectious disease development portfolio also includes TNX-3900 and TNX-4000, which are classes of broad-spectrum small molecule oral antivirals.
*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. Intravail is a registered trademark of Aegis Therapeutics, LLC, a wholly owned subsidiary of Neurelis, Inc. 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, 2022, as filed with the Securities and Exchange Commission (the “SEC”) on March 13, 2023, 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.
