Tag: Biopharmaceutical

Onconova Therapeutics (ONTX) – Onconova To Present Data At American Association of Cancer Research Meeting

Posted on by cpereira@noblefcm.com


Monday, March 11, 2024

Onconova Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer. The Company has proprietary targeted anti-cancer agents designed to disrupt specific cellular pathways that are important for cancer cell proliferation. Onconova’s novel, proprietary multi-kinase inhibitor narazaciclib (formerly ON 123300) is being evaluated in a combination trial with estrogen blockade in advanced endometrial cancer. Based on preclinical and clinical studies of CDK 4/6 inhibitors, Onconova is also evaluating opportunities for combination studies with narazaciclib in additional indications. Onconova’s product candidate rigosertib is being studied in multiple investigator-sponsored studies. These studies include a dose-escalation and expansion Phase 1/2a study of oral rigosertib in combination with nivolumab in patients with KRAS+ non-small cell lung cancer, a Phase 2 program evaluating rigosertib monotherapy in advanced squamous cell carcinoma complicating recessive dystrophic epidermolysis bullosa (RDEB-associated SCC), and a Phase 2 trial evaluating rigosertib in combination with pembrolizumab in patients with metastatic melanoma.

Robert LeBoyer, Senior Vice President, Equity Research Analyst, Biotechnology, Noble Capital Markets, Inc.

Refer to the full report for the price target, fundamental analysis, and rating.

Onconova Announces Presentation At AACR. Onconova announced the scheduling of a poster presentation at the American Association for Cancer Research Annual Meeting scheduled for April 8, 2024, in San Diego. The abstract provides additional details on rigosertib’s multiple mechanisms of action that include effects on cell signaling, interruption of cell division, and inflammation within the tumor environment to improve efficacy of checkpoint inhibitors.

Mechanisms Are Consistent With Rigosertib Clinical Trials. Rigosertib is currently in a Phase 1/2 trial testing the combination of rigosertib with nivolumab (Opdivo, an anti-PD-1 checkpoint inhibitor) in patients with KRAS-positive non-small cell lung cancer (NSCLC) and in a Phase 2 trial with pembrolizumab (Keytruda, an anti-PD-1 checkpoint inhibitor) in metastatic melanoma.


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Release – Onconova Therapeutics’ Rigosertib Poster Selected for AACR 2024

Posted on by aweinsoff@channelchek.com

Research News and Market Data on ONTX

Mar 08, 2024

PDF Version

Translational science to characterize pathways impacted by rigosertib may help to guide future clinical studies and combination treatment regimen for difficult-to-treat cancers

NEWTOWN, Pa., March 08, 2024 (GLOBE NEWSWIRE) — Onconova Therapeutics, Inc. (NASDAQ: ONTX), (“Onconova” or “the Company”), a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer, today announced that the Company will present an abstract related to preclinical studies conducted by the Company and its collaborators to further characterize the mechanism of rigosertib at the American Association for Cancer Research Annual Meeting 2024 (AACR 2024), taking place April 5-10, 2024 in San Diego, CA.

“The ability to impact multiple targets is an important characteristic of Onconova’s two lead clinical programs, rigosertib and narazaciclib. We are pleased to present new preclinical studies that explore the molecular targets for rigosertib, in clinical development through a series of investigator initiated studies (IIS),” commented Steven Fruchtman, M.D., President and Chief Executive Officer of Onconova. “The studies provide additional prospective on the main cellular pathways impacted by rigosertib, including RAS-MAPK signaling and reactive oxygen species (ROS)-related proteins. In addition, the studies highlight rigosertib’s impact on the tumor microenvironment through the activation of inflammation–related targets, such as an NLRP3. We hope to apply this translational science to help guide the clinical program to enable the potential use of rigosertib in difficult-to-treat cancers.”

Poster Information:
Poster Title:Rigosertib promotes anti-tumor activity of cancer cells via CETSA revealed novel targets and activates NLRP3-dependent inflammatory responses (2033/16)
Session:Microenvironment, Immunity, and DNA Repair in Therapeutic Response (PO.ET05.02)
Date/Time:Monday, April 8 9:00a-12:30p PT


Brief Overview: Onconova and its collaborators conducted a series of biochemistry and molecular and cell biology assays to study rigosertib’s molecular and inflammation-based targets. The team used a specialized mass spectrometry assay (a Cellular Thermal shift Assay or CETSA-MS) as one of the tools to identify new targets which were then validated as novel targets of rigosertib.

Conclusions: This work identified a series of cellular and inflammatory targets that may be affected by rigosertib. In particular, the research highlighted target activity through RAS-MAPK signaling, ROS-mediation JNK activation, and tumor microenvironment reprogramming through NLRP3 activation, which may contribute to preclinical and clinical synergistic effects with checkpoint inhibitors. The identification of these targets and signaling pathways may be helpful in the design of clinical trials to address difficult-to-treat cancers.

About Onconova Therapeutics, Inc.

Onconova Therapeutics is a clinical-stage biopharmaceutical company focused on discovering and developing novel products for patients with cancer. The Company’s product candidates, narazaciclib and rigosertib, are proprietary targeted anti-cancer agents designed to disrupt specific cellular pathways that are important for cancer cell proliferation.

Narazaciclib, Onconova’s novel, multi-kinase inhibitor (formerly ON 123300), is being evaluated in a Phase 1/2 combination trial with the estrogen blocker letrozole, in advanced endometrial cancer (NCT05705505). Based on preclinical and clinical studies of CDK4/6 inhibitors, Onconova believes narazaciclib has broad potential and is also evaluating opportunities for combination studies with narazaciclib and letrozole in additional indications, including breast cancer, ovarian cancer, multiple myeloma, and mantle cell lymphoma.

Rigosertib is being studied through an IIS strategy to evaluate the product candidate in multiple indications, including a dose-escalation and expansion Phase 1/2a study of oral rigosertib in combination with nivolumab in patients with KRAS+ non-small cell lung cancer (NCT04263090), a Phase 2 program evaluating oral or IV rigosertib monotherapy in advanced squamous cell carcinoma complicating recessive dystrophic epidermolysis bullosa (RDEB-associated SCC) (NCT03786237, NCT04177498), and a Phase 2 trial evaluating rigosertib in combination with pembrolizumab in patients with metastatic melanoma (NCT05764395).

For more information, please visit www.onconova.com.

Forward Looking Statements

Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995, and involve risks and uncertainties. These statements relate to Onconova’s expectations regarding its clinical development and trials, its product candidates, its business and financial position including the potential to initiate IISs in metastatic breast cancer and relapsed/refractory multiple myeloma, and initiate enrollment in H2 2024 and potential that the results will provide data on new treatment options for patients with metastatic breast cancer and relapsed/refractory multiple myeloma. Onconova has attempted to identify forward-looking statements by terminology including “believes,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” “preliminary,” “encouraging,” “approximately” or other words that convey uncertainty of future events or outcomes. Although Onconova believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including the success and timing of Onconova’s clinical trials, investigator-initiated trials and regulatory agency and institutional review board approvals of protocols, Onconova’s collaborations, market conditions and those discussed under the heading “Risk Factors” in Onconova’s most recent Annual Report on Form 10-K and quarterly reports on Form 10-Q. Any forward-looking statements contained in this release speak only as of its date. Onconova undertakes no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events.

Company Contact:
Mark Guerin
Onconova Therapeutics, Inc.
267-759-3680
ir@onconova.us
https://www.onconova.com/contact/

Investor Contact:
Bruce Mackle
LifeSci Advisors, LLC
646-889-1200
bmackle@lifesciadvisors.com

Release – ZyVersa Therapeutics Generated Approximately $2.7 Million From Exercise of Warrants Following Excitement Around News That Inflammasome Inhibitors Have Potential to Effectively Treat Obesity

Posted on by aweinsoff@channelchek.com

Research News and Market Data on ZVSA

Feb 29, 2024

PDF Version

  • ZyVersa is developing Inflammasome ASC Inhibitor IC 100, which is designed to inhibit formation of multiple types of inflammasomes, including NLRP3, and their associated ASC specks to attenuate initiation and perpetuation of damaging inflammation associated with obesity and numerous other conditions.

WESTON, Fla., Feb. 29, 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 inflammatory and renal diseases with high unmet medical needs, announces that on February 26, 2024, the company generated approximately $2.7 Million from existing investors’ exercise of previously issued warrants. This was following excitement around news issued by NodThera on February 19, 2024, that their NLRP3 inflammasome inhibitors have potential to treat obesity with weight loss efficacy similar to the GLP-1 receptor agonist, Wegovy, but with added cardiovascular benefits. This news became viral among financial reporters, driving high volume trading and increased stock prices for companies developing inflammasome inhibitors, including ZyVersa.

About Inflammasome ASC Inhibitor IC 100

IC 100 is a novel humanized IgG4 monoclonal antibody that inhibits the inflammasome adaptor protein ASC. IC 100 was designed to attenuate both initiation and perpetuation of the inflammatory response. It does so by binding to a specific region of the ASC component of multiple types of inflammasomes, including NLRP1, NLRP3, and AIM2, to address inflammatory diseases in which multiple inflammasome pathways are activated. 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 spread and 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. To review a white paper summarizing the mechanism of action and preclinical data for IC 100, Click Here.

About ZyVersa Therapeutics, Inc.

ZyVersa 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.

Cautionary Statement Regarding Forward-Looking Statements

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     

Release – Tonix Pharmaceuticals Completes Clinical Stage of Phase 1 Trial for TNX-1500 (Fc-modified humanized anti-CD40L mAb) in Healthy Volunteers

Posted on by aweinsoff@channelchek.com

Research News and Market Data on TNXP

February 28, 2024 8:00am ESTDownload as PDF

Topline results expected in the third quarter of 2024; planning a Phase 2 Trial for prevention of kidney transplant rejection

Anti-CD40L has multiple possible indications in addition to solid organ and bone marrow transplantation including autoimmune diseases: potential pipeline in a product

Sanofi recently published results on their Fc-modified humanized anti-CD40L mAb, frexalimab, for multiple sclerosis in the New England Journal of Medicine1

CHATHAM, N.J., Feb. 28, 2024 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP), a biopharmaceutical company with marketed products and a pipeline of development candidates, today announced the completion of the clinical stage of its Phase 1, single ascending dose escalation trial of TNX-1500 (Fc-modified humanized anti-CD40L monoclonal antibody, or mAb)* in healthy volunteers. TNX-1500 is in development for the prevention of rejection in solid organ and bone marrow transplant and for the treatment of autoimmune disorders.

“Despite advancements in the field of solid organ transplantation, there remains a significant need for new treatments with improved activity and tolerability,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “Anti-CD40L modulates T cell function and has the potential to promote tolerance of transplanted organs. We are excited to have completed the clinical stage of this Phase 1 trial of TNX-1500, a third-generation Fc-modified anti-CD40L mAb that has been designed by protein engineering to eliminate the risk of thrombosis associated with first-generation anti-CD40L mAbs. Preclinical studies in non-human primates did not result in any thrombotic complications, suggesting that the protein engineering of TNX-1500’s Fc region has achieved its design goals.”

Dr. Lederman continued, “Recently, positive clinical data with other CD40L blockers have been reported by Sanofi, with its Fc-modified humanized anti-CD40L mAb frexalimab in treating relapsing multiple sclerosis.2 Eledon Pharmaceuticals is developing tegoprubart, a non-covalent dimer antibody with no heavy-light or heavy-heavy interchain disulfide bridges for the prevention of rejection of kidney transplants3.”

Dr. Lederman concluded, “We believe TNX-1500 has the potential to prevent organ transplant rejection and improve graft survival with reduced long-term toxicity burden of current immunosuppressive regimens. In addition, TNX-1500 has the potential to address multiple other indications, including several autoimmune diseases. We look forward to the results of this Phase 1 trial, which are expected in the third quarter of this year, and to continuing the development of TNX-1500 as a promising candidate in an important therapeutic space.”

About TNX-1500

TNX-1500 (Fc-modified humanized anti-CD40L mAb) is a humanized monoclonal antibody that interacts with 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. The first-in-human Phase 1 trial of TNX-1500 was initiated in the third quarter of 2023. The primary objective of the Phase 1 trial is to assess the safety, tolerability, pharmacokinetics, 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. This first-in-human 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.4,5

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 .6,7 Dapirolizumab pegol, an anti-CD40L pegylated Fab, is being developed by UCB for the treatment of systemic lupus erythematosus.8

*TNX-1500 is an investigational new biologic and is not approved for any indication

  1. Vermersch P., et al. N Engl J Med. 2024. 390(7):589-600 https://doi.org/10.1056/nejmoa2309439
  2. Sanofi press release. May 31, 2023. https://www.sanofi.com/en/media-room/press-releases/2023/2023-05-31-05-00-00-2678991 (accessed August 2023)
  3. Eledon press release. November 2, 2023. https://ir.eledon.com/news-releases/news-release-details/eledon-reports-updated-data-ongoing-phase-1b-trial-evaluating (accessed February 2024)
  4. Lassiter G., et al. Am J Transplantation. 2023. https://doi.org/10.1016/j.ajt.2023.03.022
  5. Miura S., et al. Am J Transplantation. 2023. https://doi.org/10.1016/j.ajt.2023.03.025
  6. BioSpace. September 12, 2022. https://www.biospace.com/article/releases/horizon-therapeutics-plc-announces-phase-2-trial-evaluating-dazodalibep-for-the-treatment-of-sjoegren-s-syndrome-meets-primary-endpoint (accessed February 2024)
  7. BioSpace. January 18, 2023. https://www.biospace.com/article/horizon-bags-second-phase-ii-win-in-sjoegren-s-syndrome (accessed February 2024)
  8. https://www.ucb.com/our-science/pipeline (accessed August 2023)

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 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.

Investor Contact

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

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

Media Contact

Ben Shannon
ICR Westwicke
ben.shannon@westwicke.com
443-213-0495

Source: Tonix Pharmaceuticals Holding Corp.

Released February 28, 2024

Release – ZyVersa Therapeutics Highlights Data from Review Article Published in Nature Reinforcing IC 100’s Rationale for Inhibiting ASC and ASC Specks to Attenuate Damaging Inflammation Associated with Various Conditions, Including Obesity and Its Complications

Posted on by aweinsoff@channelchek.com

Research News and Market Data on ZVSA

Feb 28, 2024

PDF Version

  • Obesity, which affects over 1.9 billion people worldwide, is a state of low-grade chronic inflammation that causes an array of different metabolic disorders, including insulin resistance, type 2 Diabetes, hypertension, cardiovascular disease, dyslipidemia, and even cancer.
  • The article summarizes data demonstrating that inflammasome activation in fat tissue triggers cell death, known as pyroptosis. Pyroptosis results in systemic release of inflammatory cytokines, IL-1β and IL-18, and ASC specks, which perpetuates and spreads inflammation to other tissues leading to the metabolic disturbances associated with obesity.
  • ZyVersa is developing Inflammasome ASC Inhibitor IC 100, designed to inhibit formation of multiple types of inflammasomes and their associated ASC specks to attenuate initiation and perpetuation of damaging inflammation.

WESTON, Fla., Feb. 28, 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 published in Nature reinforcing IC 100’s rationale for inhibiting ASC and ASC Specks to attenuate damaging inflammation associated with various conditions, including obesity and its complications.

Studies dating back to 2011 have implicated a pathogenic role for inflammasome activation in initiation of obesity and its metabolic complications. Authors of the review paper published in Nature titled, “Cell death and inflammation during obesity: Know my methods, WAT(son),” reviewed 111 papers, which demonstrated that the state of low-grade chronic inflammation in obesity combined with activated macrophages in adipose tissue result in a vicious cycle of inflammation, cell death, and metabolic dysbalance that together cause metabolic syndromes. This also promotes a pro-tumorigenic microenvironment that induces or supports tumor growth in cancers linked to obesity such as breast, liver, and colon carcinomas.

The authors concluded, “There is no question that macrophage-inflammasome activation triggers systemic inflammation during obesity and it is one of the main culprits of metabolic syndromes.” To read the article, Click Here.

“Obesity, a well-established risk factor for array of different metabolic disorders, including insulin resistance, type 2 Diabetes, hypertension, cardiovascular disease, and cancer, has reached pandemic proportions, and may affect up to two-thirds of the adult population in developed countries,” stated Stephen C. Glover, ZyVersa’s Co-founder, Chairman, CEO and President. “The research published in Nature demonstrates that inflammasome activation in fat tissue triggers cell death. Cell death results in systemic release of inflammatory cytokines, IL-1β and IL-18, and ASC specks, which can perpetuate and spread inflammation to other tissues leading to the metabolic disturbances associated with obesity. This provides support for Inflammasome ASC Inhibitor IC 100 as a potential therapeutic option. By inhibiting ASC, IC 100 blocks formation of NLRP3 and other types of inflammasomes to block initiation of the inflammatory cascade. Likewise, IC 100 uniquely inhibits ASC specks to attenuate spread and perpetuation of damaging inflammation.” To review a white paper summarizing the mechanism of action and preclinical data for IC 100, Click Here.

About Inflammasome ASC Inhibitor IC 100

IC 100 is a novel humanized IgG4 monoclonal antibody that inhibits the inflammasome adaptor protein ASC. IC 100 was designed to attenuate both initiation and perpetuation of the inflammatory response. It does so by binding to a specific region of the ASC component of multiple types of inflammasomes, including NLRP1, NLRP2, NLRP3, NLRC4, AIM2, 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 Statements

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, IR, and Media Contact:
Karen Cashmere
Chief Commercial Officer
kcashmere@zyversa.com
786-251-9641

Release – Tonix Pharmaceuticals Announces Positive Results from Clinical Pharmacokinetic Bridging Study of Tonmya™ to Support Development and Partnering in Japan and China

Posted on by aweinsoff@channelchek.com

Research News and Market Data on TNXP

February 27, 2024 8:00am ESTDownload as PDF

Tonix plans to File a Clinical Trial Notification (CTN) in Japan and Investigational New Drug (IND) application in China to support a registration-enabling Asian Phase 3 study without dosage adjustment based on U.S. registrational Phase 3 data

Tonmya’s market exclusivity is supported by issued patents in Japan, China, Hong Kong and Taiwan; cyclobenzaprine is a new chemical entity in Japan and China

New Drug Application (NDA) submission to the U.S. FDA for the approval of Tonmya for the management of fibromyalgia planned for second half of 2024

CHATHAM, N.J., Feb. 27, 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 positive results from its clinical pharmacokinetic (PK) bridging study of Tonmya™ (also known as TNX-102 SL, cyclobenzaprine HCl sublingual tablets) in healthy adult male and female ethnic Japanese and Chinese volunteers. Results indicate that key pharmacokinetic parameters of cyclobenzaprine are comparable in ethnic Japanese and Chinese volunteers to Caucasian volunteers from a prior PK study. Tonmya was generally well tolerated in the ethnic Japanese and Chinese healthy volunteers. The company expects these data to fulfill the requirement for a bridging study, and to support regulatory filings for clinical studies in Japan and China where cyclobenzaprine is a new chemical entity (NCE). Tonix holds issued patents for market exclusivity rights of Tonmya in Japan, China, Hong Kong and Taiwan.

This study characterized the PK profile and dose proportionality of Tonmya following administration in 20 healthy volunteers of documented Japanese or Chinese ancestry, and compared these findings to an existing PK dataset conducted under similar conditions in Caucasian volunteers.

“This bridging study is an important first step as we begin evaluating the potential for approval and marketing Tonmya in Japan and China. The results show a similar pharmacokinetic profile in ethnic Japanese and Chinese volunteers with a Caucasian comparator group,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “As a result, we believe that these data, with supporting results recently reported from the positive Phase 3 RESILIENT study, are the only clinical data needed to support regulatory filings in Japan and China.”

Dr. Lederman continued, “With patents issued in Japan, China, Hong Kong and Taiwan expected to provide market exclusivity into 2034, we believe that Tonmya would be a welcome addition to the therapeutic options for fibromyalgia patients in East Asia and an attractive asset for the right development and commercialization partners in these markets. Cyclobenzaprine is an NCE in both of these countries. We plan to meet with Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) and China’s National Medical Products Administration (NMPA) to seek agreement on the development of Tonmya in Japan and China, respectively.”

About the Asia Bridging Study

The study was a randomized, single-dose, open-label, 2-way, crossover study design in ethnic Japanese (N=10) and Chinese (N=10) healthy male and female volunteers. The primary objective of the study was to characterize the PK profile and dose proportionality of Tonmya following administration of 2.8 mg and 5.6 mg (one and two 2.8 mg tablets) under fasting conditions in Japanese and Chinese volunteers, and to retrospectively compare these PK data with existing PK data of both cyclobenzaprine and norcyclobenzaprine from a prior Phase 1 study in Caucasian volunteers dosed under the same conditions. Safety and tolerability were also assessed. A 2.8 mg or 5.6 mg dose (2 X 2.8 mg tablet) of Tonmya was administered sublingually in the morning under fasted conditions. Blood samples were collected pre-dose and up to 15 days post-dose for analyte measurements, with a 28-day washout between periods. The primary PK endpoints were the total amount of cyclobenzaprine and metabolite norcyclobenzaprine in the blood (expressed as the area under the curve (AUC0-T)) and maximum concentration (expressed as Cmax).

Study Results

Pharmacokinetics
Ethnic Japanese and Chinese volunteers were considered comparable on PK parameters for cyclobenzaprine following a 2.8 mg and 5.6 mg dose of Tonmya, and dose proportionality was demonstrated in both samples. Given that the similarity in PK profile between Japanese and Chinese volunteers was confirmed, the PK data from the two ethnic groups were pooled for the comparison between Asian (n=20) and Caucasian (n=16) volunteers. The PK parameters of cyclobenzaprine for Japanese, Chinese, and Caucasian groups were similar, with geometric mean ratios falling within the 90% confidence interval.

Safety

  • Tonmya was shown to be safe and well-tolerated at doses up to 5.6 mg as single sublingual administrations in healthy adult Japanese and Chinese volunteers.
  • The incidence of adverse events (AEs) and investigational product-related AEs was low. No volunteer discontinued due to an AE.
  • No clinically significant abnormal findings in laboratory parameters, ECGs, or other safety assessments were noted during the study. No severe AEs and no deaths were reported during the study.

Issued Patents in Japan, China, Hong Kong and Taiwan

EUTECTIC FORMULATIONS









CountryPatent NumberExpected Expiry
ChinaZL 201480024011.103/14/2034
ChinaZL201910263541.603/14/2034
Hong KongHK121872703/14/2034
Japan631054203/14/2034
Taiwan R.O.C.I66182503/14/2034
TRANSMUCOSAL ABSORPTION







Japan625945206/14/2033
Taiwan R.O.C.I59082006/14/2033
Taiwan R.O.C.I68366006/14/2033
Taiwan R.O.C.I64242906/14/2033
Hong Kong120936106/14/2033

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.

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 Tonmya for the management of fibromyalgia.

*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.

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.

Investor Contact

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

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

Media Contact

Ben Shannon
ICR Westwicke
ben.shannon@westwicke.com
443-213-0495

Source: Tonix Pharmaceuticals Holding Corp.

Released February 27, 2024

Release – ZyVersa Therapeutics Publishes New White Paper Detailing the Role of Inflammasomes and ASC Specks in Neurological Diseases, and Data Supporting Inflammasome ASC Inhibitor IC 100 as a Potential Therapeutic Option

Posted on by aweinsoff@channelchek.com

Research News and Market Data on ZVSA

Feb 22, 2024

PDF Version

  • Neurological diseases, which affect up to 16 million people over 18 years old in the US, are the leading cause of physical and cognitive disability.
  • A growing body of evidence supports that activation of more than one type of inflammasome contributes to development of common neurological diseases, and that extracellular ASC specks are involved in disease progression.
  • The white paper summarizes the preclinical research of leading inflammasome experts, Drs. Robert W. Keane and Juan Pablo de Rivero Vaccari at University of Miami Miller School of Medicine, demonstrating that Inflammasome ASC Inhibitor IC 100 crosses the blood brain barrier, and that it displays strong pharmacologic and mechanistic proof-of-concept in a variety of neurological conditions.

WESTON, Fla., Feb. 22, 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 inflammatory and renal diseases who have significant unmet medical needs, announces availability of a new white paper titled, “Inflammasome ASC Inhibitor IC 100, Promising Therapeutic Potential For Neurological Diseases.” The white paper highlights data demonstrating that activation of more than one type of inflammasome and associated release of ASC specks leads to development and progression of common neurological diseases. The white paper then summarizes data from preclinical research led by Drs. Robert W. Keane and Juan Pablo de Rivero Vaccari at the University of Miami Miller School of Medicine demonstrating strong pharmacologic and mechanistic proof-of-concept for Inflammasome ASC Inhibitor IC 100 in animal models and tissue cultures representative of a variety of neurological conditions: multiple sclerosis, age-related inflammation, Alzheimer’s disease, traumatic brain injury, and spinal cord injury. Drs. Keane, de Rivero Vaccari, and their colleagues currently have research underway in Parkinson’s disease that was funded through a grant with the Michael J. Fox Foundation. To access a copy of the white paper, Click Here.

“We are excited about the potential of targeting multiple inflammasome pathways and ASC specks with Inflammasome ASC Inhibitor I00 to control the damaging inflammation contributing to neurological diseases that negatively impact the quality of life in millions of people,” said Stephen C. Glover, Co-founder, Chairman, CEO, and President of ZyVersa. “We are nearing completion of our preclinical program for IC 100 and expect to file an IND in the fourth quarter of 2024, with plans to initiate a phase 1 clinical trial in early 2025.”

About Inflammasome ASC Inhibitor IC 100

IC 100 is a novel humanized IgG4 monoclonal antibody that inhibits the inflammasome adaptor protein ASC. IC 100 attenuates 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 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 developed to ameliorate renal lipid accumulation that damages the kidneys’ filtration system in patients with glomerular 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 Statements

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 and IR Contact:

Karen Cashmere
Chief Commercial Officer
kcashmere@zyversa.com
786-251-9641        

Novavax Stock Surges Over 20% on Positive Gavi Settlement

Posted on by slightbourne@noblecapitalmarkets.com

Shares of vaccine maker Novavax jumped over 20% on Thursday after the company announced it had reached a settlement agreement with Gavi, the Vaccine Alliance. The settlement resolves a dispute between the two organizations over a canceled COVID-19 vaccine order and provides a boost to the small cap pharmaceutical company.

In May 2021, Novavax signed an advance purchase agreement with Gavi for 350 million doses of its COVID vaccine. Gavi is a public-private global health partnership focused on increasing access to immunization in lower-income countries. It was planning to distribute Novavax’s shots globally through the COVAX initiative.

However, in 2022, Novavax terminated the agreement due to Gavi’s failure to procure any of the planned vaccine doses. Gavi sought a refund on $700 million in advance payments it had made to Novavax, but the company claimed these payments were non-refundable.

The dispute went to arbitration, with Gavi demanding full repayment of the $700 million in 2023. This presented a major financial risk for the small cap Novavax, which has a market capitalization under $5 billion.

Under the new settlement, Novavax will pay Gavi a total of up to $475 million, but in installments over 5 years. An initial $75 million payment has already been made. The remaining payments of $80 million annually through 2028 can potentially be reduced based on any future Novavax vaccine orders Gavi makes.

Gavi also has the option to order discounted Novavax vaccines over the next 5 years using “vaccine credits” provided under the settlement terms. This means that if demand arises, Novavax has the opportunity to supply more of its shots to Gavi for use in lower-income countries.

The flexible settlement terms are highly positive for Novavax’s business outlook. Instead of facing a risky $700 million payment in 2023, the company can spread payments over time while potentially recouping some of the amounts through future vaccine orders.

Many analysts viewed the Gavi arbitration as one of the largest overhangs on the beaten-down stock. Resolving this dispute eliminates a major uncertainty just as Novavax is struggling with low demand for its COVID vaccine. It also ensures Novavax can still participate in serving lower-income markets through partnerships like COVAX.

As a small cap player in the competitive vaccine space, Novavax relies heavily on such partnerships. The Gavi settlement provides the company with much-needed cash flow relief and keeps the door open to future deals. Novavax can now focus its resources on boosting sales and advancing other vaccines in its pipeline.

All told, the settlement comes as a major win for Novavax and its investors. While risks remain for the small vaccine developer, removing the Gavi arbitration cloud and securing continued market access is the optimistic boost Novavax needed right now. The company still faces challenges but has bought itself more time to strategically get back on track.

Take a look at more small cap biotech companies by taking a look at Noble Capital Markets’ Senior Research Analyst Robert LeBoyer’s coverage universe.

AstraZeneca Completes $1.1 Billion Buyout of Seattle Biotech Icosavax

Posted on by slightbourne@noblecapitalmarkets.com

UK pharmaceutical giant AstraZeneca has finalized its $1.1 billion acquisition of Icosavax, a Seattle-based biotechnology company specializing in virus-like particle (VLP) vaccines. This buyout provides key insights into AstraZeneca’s pipeline strategy and the ongoing consolidation in the biopharma sector.

Icosavax was founded in 2017 as a spinout from the University of Washington’s Institute for Protein Design. The company leverages computationally designed VLPs to induce robust and durable immune responses against respiratory viruses, including COVID-19, respiratory syncytial virus (RSV), and human metapneumovirus (hMPV).

Since its founding, Icosavax has raised over $150 million in private funding and completed a successful IPO in 2021. However, the company caught the eye of pharma giant AstraZeneca, who sees Icosavax’s VLP platform and talented research team as a strategic fit.

For AstraZeneca, this acquisition provides access to a versatile new vaccine modality with broad applicability beyond Icosavax’s current clinical programs. It also bolsters AstraZeneca’s pipeline with a Phase 1/2 COVID-19 vaccine candidate, IVX-411, which produced robust neutralizing antibody titers in early clinical testing.

Broader Implications for Investors and the Biopharma Industry

The buyout has several key implications for biotech investors and industry dynamics. Firstly, it highlights that platform technologies with versatile applications across disease areas remain highly valued, even in the ongoing biotech market downturn. Vaccines also continue to see strong corporate interest after the pandemic spotlight.

Secondly, it reflects Big Pharma’s pursuit of emerging biotech innovation to replenish pipelines and access cutting-edge modalities like VLPs. With the Icosavax deal, AstraZeneca gains talented scientists and potential new products without costly in-house R&D.

Thirdly, from a structure standpoint, the deal provides an upfront cash payout to Icosavax investors but leaves upside through future contingent payments on pipeline advancement. This highlights a flexible model to balance the high valuations sought by biotechs with the risk management needs of acquirers.

Finally, the buyout continues the wave of consolidation between large and small biopharma players. With the market downturn squeezing biotech funding, more mergers and acquisitions are likely on the horizon. Investors should watch for other innovative biotechs with promising science that become acquisition targets.

What Drove AstraZeneca’s Interest in Icosavax

AstraZeneca has been one of the more active Big Pharmas on the M&A front, and the Icosavax deal provides strategic rationale. The VLP technology adds a promising new platform to AstraZeneca’s vaccine capabilities, already bolstered by its previous acquisitions of drug delivery player MedImmune and biotech Sobi.

Icosavax’s potential COVID-19 and RSV vaccine candidates can be added to AstraZeneca’s pipeline as it looks to expand beyond its core oncology portfolio. Additionally, Icosavax’s team and VLP engineering expertise will be valuable assets for the company.

By acquiring Icosavax while still early-stage compared to more established biopharmas, AstraZeneca secures access to the technology at a reasonable price. The $1.1 billion price tag is well below the multi-billion deals that some commercial-stage biotechs have commanded.

Overall, Icosavax represented an opportunity for AstraZeneca to obtain cutting-edge vaccine technology and talent to boost its R&D capabilities in new directions. It highlights that Big Pharmas are willing to buy innovation at early stages rather than develop it internally.

Take a moment to take a look at emerging growth healthcare and biotech companies by taking a look at Noble Capital Markets’ Senior Research Analyst Robert LeBoyers’s coverage universe

The Future for Icosavax’s Programs

While the buyout places Icosavax’s pipeline under AstraZeneca’s control, active development of the VLP programs is expected to continue. Lead COVID-19 vaccine candidate IVX-411 recently began Phase 1/2 trials, and its RSV and hMPV programs are progressing towards clinical stages as well.

AstraZeneca has expressed interest in advancing Icosavax’s full portfolio of vaccines leveraging the versatility of the VLP platform. Its resources and late-stage development expertise can help progress these experimental vaccines through clinical trials and regulatory approval pathways.

Meanwhile, Icosavax will continue operations as an AstraZeneca subsidiary based in Seattle. Keeping its operations separate allows Icosavax to retain its innovative biotech culture while benefiting from AstraZeneca’s financial backing and synergies.

In summary, AstraZeneca’s acquisition of Icosavax underscores its strategy of looking to smaller biotechs to supplement its pipeline with cutting-edge science. The deal rewards Icosavax investors for their early backing while retaining upside potential through milestone payments. For the biopharma industry, it exemplifies the ongoing consolidation between pharmas and biotechs amidst market pressures. Investors should watch for other emerging biotechs that may become tomorrow’s M&A targets.

Release – Tonix Pharmaceuticals Hosted KOL Webinar on Positive Phase 3 Fibromyalgia Trial for Tonmya™, Plans to File NDA for FDA Approval in Second Half of 2024

Posted on by aweinsoff@channelchek.com

Research News and Market Data on TNXP

February 14, 2024 8:00am EST

Clinical results show that Tonyma™ has broad-spectrum activity, addressing the three core fibromyalgia symptoms: pain, fatigue, and sleep disturbance

Tonmya™ has a favorable tolerability profile well-suited for chronic treatment

CHATHAM, N.J., Feb. 14, 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, held a key opinion leader (KOL) webinar to discuss the positive Phase 3 data of Tonmya™ (also known as TNX-102 SL, cyclobenzaprine HCl sublingual tablets) for the management of fibromyalgia, and the path to file for FDA approval in the second half of 2024. The webinar was held on January 31, 2024 and hosted by Alliance Global Partners (A.G.P.).

The webinar featured two thought leaders in the field of fibromyalgia: Daniel Clauw, M.D., Professor of Anesthesiology, Medicine and Psychiatry, Director Chronic Pain & Fatigue Research Center, University of Michigan, and Lesley Arnold, M.D., Professor of Psychiatry and Behavioral Neuroscience, University of Cincinnati College of Medicine.

A replay of the webinar is available here.

Tonmya™ is a centrally acting, non-opioid, non-addictive, bedtime 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 (NDA) to the U.S. Food and Drug Administration (FDA) in the second half of 2024 for Tonmya™ for the management of fibromyalgia.

“These data offer new hope for patients with fibromyalgia,” said Dr. Arnold. “Many of my patients have required combination treatments to combat their multiple fibromyalgia-related symptoms. We haven’t seen a medication that can treat fatigue, sleep, and pain all together. Having a compound like Tonmya™ that may address all three is very exciting.”

“The fact that cyclobenzaprine was beneficial in many other key symptom domains, including sleep quality, is important to fibromyalgia patients,” said Dr. Clauw. “We’re really beginning to understand how crucial sleep is in alleviating and potentially preventing fibromyalgia symptoms.”

Dr. Arnold stated that most patients are open to new treatment options, citing lack of efficacy in some cases, tolerability issues and unwanted side effects associated with the currently approved fibromyalgia treatments. “Since patients typically need to be treated for prolonged periods of time, it is important to develop a therapeutic which is well-tolerated.”

“We believe that these positive results show that fibromyalgia can be successfully treated by Tonmya™ 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.”

About the Phase 3 RESILIENT Study
The RESILIENT study was a double-blind, randomized, placebo-controlled trial designed to evaluate the efficacy and safety of Tonmya™ (TNX-102 SL: cyclobenzaprine HCl sublingual tablets) for the management of fibromyalgia. The two-arm trial randomized 457 participants in the U.S. across 33 sites. The first two weeks of treatment consisted of a run-in period in which participants started on TNX-102 SL 2.8 mg (1 tablet) or placebo. Thereafter, all participants increased 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 study met the pre-specified primary endpoint of 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 (p=0.00005).

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 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. Tonmya 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, Tonmya is in development as a daily bedtime treatment for fibromyalgia. TNX-102 SL is also in development 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 Tonmya 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 CBP-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.

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’s development portfolio is focused on central nervous system disorders. Tonix’s priority is to submit a New Drug Application (NDA) to the FDA for Tonmya, which has completed two positive Phase 3 studies for the management of fibromyalgia. Tonix intends to meet with the FDA in the first half of 2024 and submit an NDA for the approval of Tonmya for the management of fibromyalgia in the second half of 2024. TNX-102 SL is being developed to reduce the severity of acute stress reaction and the frequency of acute stress disorder and posttraumatic stress disorder. This trial is being sponsored by the University of North Carolina and received funding support from the U.S. Department of Defense. TNX-102 SL is also being developed to treat fibromyalgia-type Long COVID, a chronic post-acute COVID-19 condition, and topline results from a proof-of-concept study were reported in the third quarter of 2023. TNX-1300 (cocaine esterase) is a biologic designed to treat cocaine intoxication and has been granted Breakthrough Therapy designation by the FDA and received funding from the National Institute on Drug Abuse (NIDA). 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 (PWS). TNX-2900 has been granted Orphan Drug designation by the FDA and an investigational new drug (IND) application has been cleared to support a Phase 2 study in PWS patients. 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 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 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.

Investor Contact

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

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

Media Contact

Ben Shannon
ICR Westwicke
ben.shannon@westwicke.com
443-213-0495

Source: Tonix Pharmaceuticals Holding Corp.

Released February 14, 2024

Release – YS Biopharma Announces Appointment of New Directors

Posted on by aweinsoff@channelchek.com

Research News and Market Data on YS

GAITHERSBURG, Md., Feb. 14, 2024 /PRNewswire/ — YS Biopharma Co., Ltd. (Nasdaq: YS) (“YS Biopharma” or the “Company”, and together with its subsidiaries, “YS Group”), a global biopharmaceutical company dedicated to discovering, developing, manufacturing, and delivering new generations of vaccines and therapeutic biologics for infectious diseases and cancer, today announced the appointment of six new members to its Board of Directors (the “Board”), effective February 13, 2024. The six new directors are: Dr. Yuntao Cui; Dr. Jin Wang; Mr. Henry Chen; Mr. Haitao Zhao; Mr. Pierson Yue Pan; and Ms. Brenda Chunyuan Wu.

In addition to the new appointments, the Board has elected Dr. Ajit Shetty, who had served as the Company’s Interim Chairperson of the Board since December 9, 2023, as Chairperson of the Board, effective February 13, 2024.

Dr. Yuntao Cui has over 15 years of law-related work experience, and has previously worked as a Senior Lawyer at Zhong Lun Law Firm, one of the top law firms in China, and as General Manager of Legal Affairs at Wanda Group, a top conglomerate in the real estate, finance, and hospitality industries. Dr. Cui is currently a Senior Vice President and General Counsel at Juventas Cell Therapy Ltd., a leading immune cell therapy company. Dr. Cui holds a JD from Renmin University of China.

Dr. Jin Wang is Founding Partner and CEO of Manhattan Capital Investment Consulting Group (“MCG”), and has managed the venture capital investment fund Nuokang Venture Fund since 2014. Dr. Wang has more than 27 years of experience finding, building, and strategizing for biotech companies, including his co-founding and angel investment in leading medical professional networking and service firm DXY. Dr. Wang previously worked as an analyst and Asia/Greater China Regional Director for Paramount Capital Investment, LLC, and co-founded the Sino-American Pharmaceutical Professionals Association (“SAPA”) in 1993. Dr. Wang holds a PhD in Biomedical Science from Worcester Polytechnic Institute.

Mr. Henry Chen is the CEO of Monument Pacific Development Corp., a California-based company dedicated to developing, managing, and operating residential and commercial properties in Northern and Southern California. Mr. Chen received his MBA from the University of California, Berkeley’s Hass School of Business in 2012, and a Bachelor of Laws in Business Administration and Management from the Beijing University of Chemical Technology in 2005.

Mr. Haitao Zhao has served as a Partner of the Shanxi Tie Niu law firm since 2016, where he is responsible for handling corporate law related to intellectual property protection, corporate internal governance, and financing-related legal services. Previously, Mr. Zhao worked as an Attorney for the Beijing Ding En law firm from 2012-2016. He holds an MA in International Law from Shanxi University.

Mr. Pierson Yue Pan has served as a Vice President of Monument Pacific Development Corp. since 2018. Previously, Mr. Pan worked as Vice President of Project Development at real estate developer Propriis from 2012-2018, and as General Manager of the Africa Market with the China Civil Engineering Construction Company Nigeria Limited from 2004-2011. Mr. Pan holds an MBA from the University of California, Berkeley’s Hass School of Business.

Ms. Chunyuan (Brenda) Wu currently serves as the CFO of YS Group, a position she has held since December 31, 2020. Previously, Ms. Wu served as CFO of YS Group Biopharma from 2018-2020, and as Financial Controller for the same company from 2013-2018. Ms. Wu has also previously worked as a Senior Auditor at Ernst & Young. Ms. Wu holds degrees in Accounting and Finance from Washington State University.

Dr. Ajit Shetty, Chairperson of the Board of Directors of the Company, commented, “We are pleased to welcome each of our new directors to the Board. Each of these talented individuals brings valuable expertise in various aspects of corporate governance, legal affairs, business management, and strategic decision-making. We expect that their diverse experience and commitment to success will serve our company well going forward.”

About YS Biopharma

YS Biopharma is a global biopharmaceutical company dedicated to discovering, developing, manufacturing, and commercializing new generations of vaccines and therapeutic biologics for infectious diseases and cancer. It has developed a proprietary PIKA® immunomodulating technology platform and a series of preventive and therapeutic biologics with a potential for improved Rabies, Coronavirus, Hepatitis B, Influenza, and Shingles vaccines. YS Biopharma operates in China, the United States, Singapore and the Philippines, and is led by a management team that combines rich local expertise and global experience in the bio-pharmaceutical industry. For more information, please visit investor.ysbiopharm.com.

Cautionary Statement Regarding Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933, as amended, Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995. All statements other than statements of historical or current fact included in this press release are forward-looking statements, including but not limited to statements regarding the expected growth of the Company, the development progress of all product candidates, the progress and results of all clinical trials, the Company’s ability to source and retain talent, and the cash position of the Company following the closing of the Business Combination. Forward-looking statements may be identified by the use of words such as “estimate,” “plan,” “project,” “forecast,” “intend,” “will,” “expect,” “anticipate,” “believe,” “seek,” “target” or other similar expressions that predict or indicate future events or trends or that are not statements of historical matters. These statements are based on various assumptions, whether identified in this press release, and on the current expectations of YS Biopharma’s management and are not predictions of actual performance.

These statements involve risks, uncertainties and other factors that may cause actual results, levels of activity, performance, or achievements to be materially different from those expressed or implied by these forward-looking statements. Although YS Biopharma believes that it has a reasonable basis for each forward-looking statement contained in this press release, YS Biopharma cautions you that these statements are based on a combination of facts and factors currently known and projections of the future, which are inherently uncertain. In addition, there are risks and uncertainties described in the documents filed by YS Biopharma from time to time with the U.S. Securities and Exchange Commission (“SEC”). These filings may identify and address other important risks and uncertainties that could cause actual events and results to differ materially from those contained in the forward-looking statements.

YS Biopharma cannot assure you that the forward-looking statements in this press release will prove to be accurate. These forward-looking statements are subject to a number of risks and uncertainties, including, among others, the outcome of any potential litigation, government or regulatory proceedings, the sales performance of the marketed vaccine product and the clinical trial development results of the product candidates of YS Biopharma, and other risks and uncertainties, including those included under the heading “Risk Factors” in the post-effective amendment No. 2 to Form F-1 filed with the SEC on January 23, 2024 which became effective on January 25, 2024, and other filings with the SEC. There may be additional risks that YS Biopharma does not presently know or that YS Biopharma currently believes are immaterial that could also cause actual results to differ from those contained in the forward-looking statements. In light of the significant uncertainties in these forward-looking statements, nothing in this press release should be regarded as a representation by any person that the forward-looking statements set forth herein will be achieved or that any of the contemplated results of such forward-looking statements will be achieved. The forward-looking statements in this press release represent the views of YS Biopharma as of the date of this press release. Subsequent events and developments may cause those views to change. However, while YS Biopharma may update these forward-looking statements in the future, there is no current intention to do so, except to the extent required by applicable law. You should, therefore, not rely on these forward-looking statements as representing the views of YS Biopharma as of any date subsequent to the date of this press release. Except as may be required by law, YS Biopharma does not undertake any duty to update these forward-looking statements.

Investor Relations Contact

Robin Yang
Partner, ICR, LLC
Tel: +1 (212) 537-4035
Email: YSBiopharma.IR@icrinc.com 

View original content to download multimedia:https://www.prnewswire.com/news-releases/ys-biopharma-announces-appointment-of-new-directors-302061795.html

SOURCE YS Biopharma Co., Ltd.

Release – Tonix Pharmaceuticals Announces Engagement of Rho as CRO to Support Submission of New Drug Application to the FDA for Approval of Tonmya™ for the Management of Fibromyalgia

Posted on by aweinsoff@channelchek.com

Research News and Market Data on TNXP

February 13, 2024 8:00am ESTDownload as PDF

Tonmya™ is a potential new first-line therapy, 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 second half of 2024

Fibromyalgia is a chronic pain condition affecting approximately 10 million U.S. adults that typically persists for years or decades

CHATHAM, N.J., Feb. 13, 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 that it has engaged Rho, Inc. (Rho), a global contract research organization (CRO), to support Tonix’s preparation and planned submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the approval of Tonmya™ (cyclobenzaprine HCl sublingual tablets) for the management of fibromyalgia.

“We are excited by our most recent positive Phase 3 study results of Tonmya and look forward to Rho’s support in preparing our NDA,” said Seth Lederman, M.D., President and Chief Executive Officer of Tonix Pharmaceuticals. “We intend to meet with the FDA in the first half of this year and to submit our NDA in the second half of the year. We believe we have completed all development work required for the NDA, including CMC requirements and the completion of two positive Phase 3 studies.”

Tonmya is a patented sublingual tablet 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.

“We are delighted in our continued support of Tonix and their Tonmya program,” said Tara Gladwell, President and COO at Rho. “We are hopeful that this work may result in a new treatment for the millions of Americans that suffer from this debilitating, chronic disorder.”

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 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. Tonmya 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, Tonmya is in development as a daily bedtime treatment for fibromyalgia. TNX-102 SL is also in development 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 Tonmya 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 CBP-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.

About Rho

Rho is a global, privately held contract research organization (CRO) headquartered in Research Triangle Park, a biotech hub in North Carolina, US. Rho provides a full range of drug development services, from program strategy through to clinical trials and marketing applications. Since 1984, Rho has been a trusted partner to some of the most innovative pharmaceutical, biotechnology and medical device companies as well as academic and government organizations. Dedicated to service excellence and cross-functional collaboration, Rho’s therapeutic expertise, employee focus and commitment to strong site relationships change what it means to work with a CRO – accelerating time to market, maximizing ROI, and delivering consistent, smarter and more efficient programs. Experience Rho by following the company on LinkedIn.

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’s development portfolio is focused on central nervous system disorders. Tonix’s priority is to submit a New Drug Application (NDA) to the FDA for Tonmya, which has completed two positive Phase 3 studies for the management of fibromyalgia. Tonix intends to meet with the FDA in the first half of 2024 and submit an NDA for the approval of Tonmya for the management of fibromyalgia in the second half of 2024. TNX-102 SL is being developed to reduce the severity of acute stress reaction and the frequency of acute stress disorder and posttraumatic stress disorder. This trial is being sponsored by the University of North Carolina and received funding support from the U.S. Department of Defense. TNX-102 SL is also being developed to treat fibromyalgia-type Long COVID, a chronic post-acute COVID-19 condition, and topline results from a proof-of-concept study were reported in the third quarter of 2023. TNX-1300 (cocaine esterase) is a biologic designed to treat cocaine intoxication and has been granted Breakthrough Therapy designation by the FDA and received funding from the National Institute on Drug Abuse (NIDA). 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 (PWS). TNX-2900 has been granted Orphan Drug designation by the FDA and an investigational new drug (IND) application has been cleared to support a Phase 2 study in PWS patients. 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 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 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.

Investor Contact

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

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

Media Contact

Ben Shannon
ICR Westwicke
ben.shannon@westwicke.com
443-213-0495

Source: Tonix Pharmaceuticals Holding Corp.

Released February 13, 2024

Gilead Sciences Acquires CymaBay Therapeutics for $4.3 Billion in Cash

Posted on by slightbourne@noblecapitalmarkets.com

Gilead Sciences announced Monday that it will acquire clinical-stage biotech CymaBay Therapeutics for $4.3 billion, gaining seladelpar, an investigational treatment for primary biliary cholangitis (PBC) that is currently under FDA priority review.

PBC is a progressive autoimmune disease that damages the bile ducts in the liver, causing bile acid buildup that can lead to irreversible scarring and liver failure. An estimated 130,000 Americans live with PBC, which mostly affects women over 40.

CymaBay’s seladelpar is an oral selective peroxisome proliferator-activated receptor delta (PPARδ) agonist that targets metabolic and inflammatory pathways involved in PBC. Data from late-stage studies demonstrate seladelpar’s potential as a best-in-class therapy for second-line PBC patients who don’t respond adequately to first-line treatment with ursodeoxycholic acid (UDCA).

The drug received breakthrough therapy and orphan drug designations from the FDA and EMA based on positive mid-stage results. In December 2022, CymaBay submitted a new drug application to the FDA, which was granted priority review last month. An approval decision is expected by August 14, 2024.

According to the phase 3 RESPONSE trial, seladelpar achieved significant improvements in reducing alkaline phosphatase levels and relieving itch symptoms compared to placebo. Nearly 62% of patients on seladelpar attained a biochemical response versus 20% on placebo.

Gilead aims to leverage its extensive experience in developing treatments for liver diseases like hepatitis C and nonalcoholic steatohepatitis (NASH) to advance seladelpar. The deal expands Gilead’s presence in the PBC space, complementing its existing medicine Ocaliva, which is approved as a second-line option.

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“We are looking forward to advancing seladelpar by leveraging Gilead’s long-standing expertise in treating and curing liver diseases,” commented Gilead CEO Daniel O’Day. “Building on the strong R&D work by the CymaBay team, we have the potential to address a significant unmet need for people with PBC.”

Under the terms of the agreement, Gilead will commence a tender offer to acquire all outstanding CymaBay shares at $32.50 per share in cash, representing a 27% premium over the stock’s closing price on February 9. Following the tender offer, Gilead will mop up any untendered shares via a second-step merger at the same price.

The deal is anticipated to close in the first quarter of 2024, subject to customary closing conditions and regulatory clearances. Once the transaction is completed, CymaBay will become a wholly owned subsidiary of Gilead.

Gilead expects the buyout will boost its top-line revenue growth, while being neutral to earnings per share in 2025 before turning significantly accretive thereafter.

For CymaBay, the takeover marks the culmination of years of effort advancing seladelpar into late-stage testing and regulatory review. “Now that seladelpar has achieved priority review with the FDA, we are excited that Gilead can apply its expertise to bring seladelpar as quickly as possible to people with PBC,” noted CymaBay CEO Sujal Shah.

The profitable exit provides a major return for CymaBay investors, as the purchase price represents a substantial premium over the stock’s pre-announcement valuation. CymaBay shares have languished below $4 for much of the past two years.

Gilead has actively pursued M&A to augment its pipeline and product portfolio across therapeutic areas like oncology, inflammation, and antivirals. The company faces looming patent expiries on flagship HIV medicines. New growth drivers like seladelpar could help offset that impact.

PBC currently affects a relatively small patient population, but analysts project seladelpar could generate peak annual sales above $1 billion. Gilead likely sees potential to expand seladelpar’s utility to other cholestatic liver diseases.

Nonetheless, the deal does carry risks for Gilead. Seladelpar’s broad mechanism regulating gene expression raises safety concerns about potential side effects. Patients in late-stage testing experienced elevations in low-density lipoprotein cholesterol.

By acquiring CymaBay outright, Gilead shoulders all future R&D costs rather than opting for a partnership deal to share expenses. If seladelpar encounters any regulatory or commercial setbacks, Gilead lacks an immediate fallback option for its PBC program.

But with priority review underway and approval expected within six months, Gilead moved aggressively to lock up rights to a promising PBC candidate. Adding seladelpar provides another growth avenue beyond HIV and bolsters Gilead’s mission of delivering transformative medicines for underserved diseases.