Category: Health

ZyVersa Therapeutics, Inc. (ZVSA) – FY23 Reported With Reiteration of Milestones For Kidney and Inflammasome Trials

Posted on by cpereira@noblefcm.com


Tuesday, March 26, 2024

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.

ZyVersa Reported FY2023. ZyVersa Reported a FY2023 loss of $106.2 million or $(108.97) per share, including write-downs of In-process Research and Development of $81.4 million and Goodwill of $11.9 million that were recorded with the reverse merger in December 2022. Excluding these items, the operating loss would have been $14.4 million. Cash on hand was $3.1 million on December 31, and does not include $2.7 million from warrant exercise reported during February 2024.

ZyVersa Has Made Progress Toward Starting VAR 200 Clinical Trials. ZyVersa announced the selection of a CRO and approval by an Institutional Review Board (IRB). These are steps required to start the Phase 2a clinical trial for VAR 200, its lead molecule for modulating cholesterol and lipid accumulation in the kidney that starts damage and declining function pathways. VAR 200 is expected to begin treating patients with diabetic kidney disease in 1H24.


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Nuvation Bio Acquires AnHeart Therapeutics, Gains Promising Oncology Assets

Posted on by slightbourne@noblecapitalmarkets.com

In a strategic move to strengthen its oncology pipeline, Nuvation Bio Inc. (NYSE: NUVB), a biopharmaceutical company focused on developing novel cancer therapies, has announced its acquisition of AnHeart Therapeutics Ltd. in an all-stock transaction. This acquisition promises to transform Nuvation Bio into a late-stage global oncology company, positioning it as a potential commercial organization by the end of 2025.

The deal, which is subject to approval by AnHeart’s shareholders and other customary closing conditions, is expected to close in the second quarter of 2024. Upon completion, the former shareholders of AnHeart will own approximately 33% of Nuvation Bio on a fully diluted basis, while the current stockholders of Nuvation Bio will retain a 67% stake.

The primary asset driving this acquisition is taletrectinib, AnHeart’s lead investigational therapy and a next-generation ROS1 inhibitor for the treatment of ROS1-positive non-small cell lung cancer (NSCLC). Taletrectinib has already received Breakthrough Therapy Designations from both the U.S. Food and Drug Administration (FDA) and China’s National Medical Products Administration (NMPA).

Notably, taletrectinib is currently completing two pivotal Phase 2 studies, TRUST-I in China and TRUST-II, a global pivotal study, potentially positioning it as a best-in-class treatment option for patients with ROS1-positive NSCLC. The NMPA has also granted Priority Review Designation to New Drug Applications for taletrectinib, further underscoring its potential.

In addition to taletrectinib, the acquisition also brings safusidenib, a potentially best-in-class mutant IDH1 inhibitor, into Nuvation Bio’s pipeline. Safusidenib is currently being evaluated in a global Phase 2 study for the treatment of patients with grades 2 and 3 IDH1-mutant glioma.

“This transaction represents a significant milestone for our company and reflects Nuvation Bio’s continued commitment to developing therapies for patients with the most difficult-to-treat cancers,” said David Hung, M.D., Founder, President, and Chief Executive Officer of Nuvation Bio. “AnHeart’s lead asset, taletrectinib, which will become our lead asset as it completes two pivotal studies, is a differentiated, next-generation ROS1 inhibitor with a potentially best-in-class profile that may overcome the significant limitations of existing therapies.”

For Nuvation Bio, this all-stock acquisition preserves the company’s robust cash balance, enabling the development of both the newly acquired assets and its existing pipeline without the immediate need to raise additional capital. This financial strength positions Nuvation Bio to execute its development strategy effectively and advance its combined portfolio of differentiated oncology therapeutic candidates.

The acquisition also brings together the talented teams from both companies, with Nuvation Bio’s current management team, including Dr. Hung, remaining at the helm. Additionally, Min Cui, Ph.D., Founder and Managing Director of Decheng Capital, an investor in AnHeart, and Junyuan Jerry Wang, Ph.D., Co-Founder and Chief Executive Officer of AnHeart, will join Nuvation Bio’s board of directors.

As the demand for innovative cancer therapies continues to grow, Nuvation Bio’s acquisition of AnHeart Therapeutics represents a strategic move to bolster its oncology pipeline and position itself as a potential commercial organization in the near future. With taletrectinib and safusidenib as promising additions to its portfolio, Nuvation Bio is poised to make significant strides in addressing the unmet needs of patients with challenging forms of cancer.

Release – Eledon Pharmaceuticals Announces 12th Participant Enrolled in Phase 2 BESTOW Trial Evaluating Tegoprubart for the Prevention of Organ Rejection

Posted on by aweinsoff@channelchek.com

Research News and Market Data on ELDN

March 25, 2024

PDF Version

IRVINE, Calif., March 25, 2024 (GLOBE NEWSWIRE) — Eledon Pharmaceuticals, Inc. (“Eledon”) (NASDAQ: ELDN) today announced the enrollment of the 12th participant on March 23, 2024, in the Company’s ongoing Phase 2 BESTOW trial assessing tegoprubart head-to-head with tacrolimus for the prevention of rejection in kidney transplantation.

“We are pleased with the strong pace of enrollment in our Phase 2 BESTOW trial and believe it speaks to the underlying demand for a new immunosuppressive regimen for the tens of thousands of patients each year who undergo kidney transplantation,” said David-Alexandre C. Gros, M.D., Chief Executive Officer.

The Risk of Organ Failure in Transplantation

In transplantation procedures, organ rejection is a major cause of graft failure, which can be a life-threatening condition. Rejection occurs due to allorecognition, wherein the recipient’s immune system identifies the transplanted organ as foreign tissue, triggering an immune response against the transplanted organ. To reduce the risk of rejection, patients are treated with immunosuppressive therapies for life. Calcineurin inhibitors (“CNIs”) are a critical component of most immunosuppressive regimens to prevent acute and long-term organ transplant rejection. However, chronic exposure to CNIs (tacrolimus is the drug most commonly used) is associated with nephrotoxicity, hypertension, new onset diabetes due to pancreatic beta cell toxicity, as well as central nervous system side effects like tremor. Regarding kidney transplantation, the toxicity associated with CNIs causes 30-50% of kidney transplants to fail with 10-15 years of transplantation. Strategies to better and more safely protect transplanted organs and thus increase how long they function represent a significant area of unmet need in organ transplantation.

About Eledon Pharmaceuticals and tegoprubart

Eledon Pharmaceuticals, Inc. is a clinical stage biotechnology company that is developing immune-modulating therapies for the management and treatment of life-threatening conditions. The Company’s lead investigational product is tegoprubart, an anti-CD40L antibody with high affinity for the CD40 Ligand, a well-validated biological target that has broad therapeutic potential. The central role of CD40L signaling in both adaptive and innate immune cell activation and function positions it as an attractive target for non-lymphocyte depleting, immunomodulatory therapeutic intervention. The Company is building upon a deep historical knowledge of anti-CD40 Ligand biology to conduct preclinical and clinical studies in kidney allograft transplantation, xenotransplantation, and amyotrophic lateral sclerosis (ALS). Eledon is headquartered in Irvine, California. For more information, please visit the Company’s website at www.eledon.com.

Follow Eledon Pharmaceuticals on social media: LinkedIn; Twitter

Forward-Looking Statements

This press release contains forward-looking statements that involve substantial risks and uncertainties. Any statements about the demand for a new and better immunosuppressive regimen for patients who undergo kidney transplantation, or statements about the Company’s other future expectations, plans and prospects, as well as other statements containing the words “believes,” “anticipates,” “plans,” “expects,” “estimates,” “intends,” “predicts,” “projects,” “targets,” “looks forward,” “could,” “may,” and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are inherently uncertain and are subject to numerous risks and uncertainties, including: risks relating to the safety and efficacy of our drug candidates; risks relating to clinical development timelines, including interactions with regulators and clinical sides, as well as patient enrollment; risks relating to costs of clinical trials and the sufficiency of the company’s capital resources to fund planned clinical trials; and risks associated with the impact of the ongoing coronavirus pandemic. Actual results may differ materially from those indicated by such forward-looking statements as a result of various factors. These risks and uncertainties, as well as other risks and uncertainties that could cause the company’s actual results to differ significantly from the forward-looking statements contained herein, are discussed in our quarterly 10-Qs, annual 10-K, and other filings with the U.S. Securities and Exchange Commission, which can be found at www.sec.gov. Any forward-looking statements contained in this press release speak only as of the date hereof and not of any future date, and the company expressly disclaims any intent to update any forward-looking statements, whether as a result of new information, future events or otherwise. 

Investor Contact:

Stephen Jasper
Gilmartin Group
(858) 525 2047
stephen@gilmartinir.com

Media Contact:

Jenna Urban
Berry & Company Public Relations
(212) 253 8881
jurban@berrypr.com

Source: Eledon Pharmaceuticals

AbbVie’s Acquisition of Landos Biopharma Highlights Potential in Small-Cap Biotech

Posted on by slightbourne@noblecapitalmarkets.com

In a strategic move that could have significant implications for the small-cap biotech sector, pharmaceutical giant AbbVie Inc. (NYSE: ABBV) announced its acquisition of Landos Biopharma, Inc. (NASDAQ: LABP), a clinical-stage biopharmaceutical company focused on developing novel oral therapeutics for autoimmune diseases. The deal, valued at approximately $212.5 million including contingent value rights, underscores the growing interest and potential in the small-cap biotech space, particularly in the field of inflammatory and autoimmune diseases.

Under the terms of the agreement, AbbVie will acquire Landos at $20.42 per share in cash upon closing, plus a contingent value right of up to $11.14 per share, subject to the achievement of a clinical development milestone. The acquisition is expected to close in the second quarter of 2024, subject to customary closing conditions, including approval by Landos’ stockholders.

The primary asset driving this deal is NX-13, Landos’ lead investigational asset and a first-in-class, oral NLRX1 agonist with a bimodal mechanism of action. NX-13 is currently in Phase 2 clinical trials for the treatment of ulcerative colitis (UC), a chronic inflammatory bowel disease affecting millions worldwide.

“With this acquisition, we aim to advance the clinical development of NX-13, a differentiated, first-in-class, oral asset with the potential to make a difference in the lives of people living with ulcerative colitis and Crohn’s disease,” said Roopal Thakkar, M.D., AbbVie’s senior vice president and chief medical officer, global therapeutics.

NX-13’s unique bimodal mechanism of action, which is both anti-inflammatory and facilitates epithelial repair, could provide a novel approach to treating UC and other inflammatory bowel diseases. If successful, it could address a significant unmet need in this therapeutic area.

The acquisition underscores AbbVie’s commitment to strengthening its portfolio in inflammatory and autoimmune diseases, which represent a substantial market opportunity. According to estimates, the global inflammatory bowel disease treatment market is projected to reach $8.6 billion by 2027, driven by factors such as increasing prevalence, rising healthcare expenditure, and a growing focus on developing targeted therapies.

For small-cap investors, this deal highlights the potential value and attractiveness of emerging biotech companies with promising pipeline candidates. As larger pharmaceutical companies seek to bolster their portfolios and drive innovation, strategic acquisitions of small-cap biotechs with compelling assets can provide attractive exit opportunities and significant returns for investors.

This acquisition also comes at an opportune time, as Noble Capital Markets’ upcoming virtual healthcare event on April 17-18 will showcase emerging growth companies in the healthcare, biotech, and medical device industries. Investors interested in exploring opportunities in the small-cap biotech space should mark their calendars for this event, which promises to provide valuable insights and potential investment prospects in this dynamic sector.

With the rising interest in novel therapies for inflammatory and autoimmune diseases, the AbbVie-Landos deal serves as a reminder of the potential value that can be unlocked in the small-cap biotech realm. As larger players seek to fortify their pipelines, the spotlight on promising small-cap innovators is likely to intensify, presenting exciting opportunities for investors in this space.

Release – ZyVersa Therapeutics Reports Full Year 2023 Financial Results and Provides Business Update

Posted on by aweinsoff@channelchek.com

Research News and Market Data on ZVSA

Mar 25, 2024

Key Highlights:

  • Cholesterol Efflux MediatorTM VAR 200 on target to begin Phase 2a clinical trial in patients with diabetic kidney disease H1-2024.
  • Inflammasome ASC Inhibitor IC 100 preclinical program nearing completion, with planned Investigational New Drug (IND) submission Q4-2024, and Phase 1 clinical trial initiation shortly thereafter.
  • Inflammasome ASC Inhibitor IC 100 preclinical research funded by The Michael J. Fox Foundation for Parkinson’s Research (MJFF) nearing completion, with potential for a second MJFF grant for further research.
  • Scientific collaboration to assess Inflammasome ASC Inhibitor IC 100 as a potential treatment for atherosclerosis expected to conclude H1-2024.
  • Scientific collaboration to assess Inflammasome ASC Inhibitor IC 100 as a potential treatment for obesity and metabolic syndrome expected to begin Q2-2024.

WESTON, Fla., March 25, 2024 (GLOBE NEWSWIRE) — ZyVersa Therapeutics, Inc. (Nasdaq: ZVSA, or “ZyVersa”), a clinical-stage specialty biopharmaceutical company developing first-in-class drugs for the treatment of renal and inflammatory diseases with high unmet medical needs, reports financial results for full year ending December 31, 2023, and provides business update.

“Throughout 2023 and early 2024, ZyVersa achieved considerable progress in advancing development of our two lead candidates. A Phase 2a clinical trial with Cholesterol Efflux MediatorTM VAR 200 is on target to be initiated in the first half of this year in patients with diabetic kidney disease; preclinical studies for indication expansion are underway for Parkinson’s disease, atherosclerosis, and obesity with Inflammasome ASC Inhibitor IC 100; and IND submission for IC 100 is expected by end of the year,” stated Stephen C. Glover, ZyVersa’s Co-founder, Chairman, CEO, and President. “We view 2024 as a potentially transformative year for the company based on the value-building milestones that we expect to achieve over the next 12 to 15 months. I look forward to working with my leadership team and fellow Board members to execute a business and clinical strategy that has potential to position ZyVersa as a leading, innovative company developing transformative drugs for underserved patients with renal and inflammasome-mediated inflammatory diseases.” 

BUSINESS Update

CHOLESTEROL EFFLUX MEDIATORTM VAR 200 FOR RENAL DISEASE

  • Phase 2a clinical trial in patients with diabetic kidney disease is on target to begin in the first half of 2024.
  • CRO, George Clinical was engaged in December 2023 to initiate and manage the trial.
  • An IND amendment to study diabetic kidney disease was filed on February 16, 2024.
  • A central Institutional Review Board (IRB) approved the clinical trial protocol for trial initiation.

INFLAMMASOME ASC INHIBITOR IC 100 FOR INFLAMMATORY DISEASES

  • Inflammasome ASC Inhibitor IC 100’s preclinical program is nearing completion, with a planned IND submission in Q4-2024, followed by initiation of a Phase 1 clinical trial shortly thereafter.
  • Preclinical research funded by MJFF and conducted by researchers at University of Miami Miller School of Medicine to determine the potential of Inflammasome ASC Inhibitor IC 100 to block the damaging neuroinflammation that induces neural degeneration in Parkinson’s disease is nearing completion.
  • Research update and key findings were provided to MJFF project team on March 8, 2024.
  • Based on the findings, the MJFF project team suggested that the team apply for a second grant to further the research in an established animal model.
  • A scientific collaboration was initiated with an undisclosed partner to assess the potential of Inflammasome ASC Inhibitor IC 100 as a treatment for atherosclerosis in a well-established animal model.
  • Study is expected to conclude in H1-2024.
  • A scientific collaboration with inflammasome and neurology experts at University of Miami Miller School of Medicine to assess the potential of Inflammasome ASC Inhibitor IC 100 as a treatment for obesity and metabolic syndrome is expected to begin in Q2-2024.

YEAR END 2023 FINANCIAL RESULTS

Net losses were $98.3 million for the year ended December 31, 2023 (the “2023 Period”), an increase of $84.2 million compared to a net loss of $14.1 million for the “2022 Period,” which is comprised of the financial results of the company containing our operations prior to our business combination from January 1, 2022 through December 12, 2022, and our financial results after the business combination, from December 13, 2022 through December 31, 2022. A deferred tax benefit of $9.5 million for the 2023 Period, compared to $0.7 million tax benefit during the 2022 Period, resulted from the impairment of the in-process research and development.

Pre-tax losses were $107.8 million for the 2023 Period, an increase of $92.9 million compared to a pre-tax loss of $14.9 million for the 2022 Period. The higher net loss reported for the 2023 Period is primarily due to the impairment of in-process research and development and impairment of goodwill of $81.4 million and $11.9 million, respectively, compared to none for the 2022 Period. The impairment is a result of the decline in stock value and the resulting market capitalization of ZyVersa during the 2023 Period.

Based on its current operating plan, ZyVersa expects its cash of $3.1 million as of December 31, 2023 will be sufficient to fund its operating expenses and capital expenditure requirements on a month-to-month basis. ZyVersa will need additional financing to support its continuing operations. ZyVersa will seek to fund its operations through public or private equity or debt financings or other sources, which may include government grants and collaborations with third parties. 

Research and development expenses for the 2023 Period were $3.2 million, a decrease of $2.6 million or 44.8% from $5.8 million for the 2022 Period. The decrease in research and development expenses was primarily due to spending for materials supplies for manufacturing in 2022 that were not required in 2023 as manufacturing was completed in 2022. 

General and administrative expenses for the 2023 Period were $11.2 million, an increase of $3.2 million or 39.7% from $8.0 million for the 2022 Period. The increase is primarily attributed to increased costs for legal and professional fees of $2.7 million, investor and public relations fees of $1.2 million, and increased director and officer insurance of $1.3 million, all due to increased costs associated with being a public company. These were offset by reduced business combination transaction costs of $2.2 million.

About ZyVersa Therapeutics, Inc.

ZyVersa (Nasdaq: ZVSA) is a clinical stage specialty biopharmaceutical company leveraging advanced, proprietary technologies to develop first-in-class drugs for patients with renal and inflammatory diseases who have significant unmet medical needs. The Company is currently advancing a therapeutic development pipeline with multiple programs built around its two proprietary technologies – Cholesterol Efflux Mediator™ VAR 200 for treatment of kidney diseases, and Inflammasome ASC Inhibitor IC 100, targeting damaging inflammation associated with numerous CNS and peripheral inflammatory diseases. For more information, please visit www.zyversa.com.

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, IR Contact

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

ZYVERSA THERAPEUTICS, INC. 
CONSOLIDATED BALANCE SHEETS 
  
    Successor 
    December 31, 
     2023   2022  
        
Assets       
        
Current Assets:     
 Cash $3,137,674  $5,902,199  
 Prepaid expenses and other current assets 215,459   225,347  
 Vendor deposits  –   235,000  
  Total Current Assets 3,353,133   6,362,546  
Equipment, net  6,933   17,333  
In-process research and development 18,647,903   100,086,329  
Goodwill  –   11,895,033  
Security deposit  98,476   46,659  
Operating lease right-of-use asset 7,839   98,371  
        
  Total Assets$22,114,284  $118,506,271  
        
Liabilities, Temporary Equity and Stockholders’ Equity    
        
Current Liabilities:     
 Accounts payable $8,431,583  $6,025,645  
 Accrued expenses and other current liabilities 1,754,533   2,053,559  
 Operating lease liability 8,656   108,756  
  Total Current Liabilities 10,194,772   8,187,960  
Deferred tax liability  844,914   10,323,983  
  Total Liabilities 11,039,686   18,511,943  
        
Commitments and contingencies    
        
 Redeemable common stock, subject to possible redemption,    
 0 and 1,880 shares outstanding as of December 31, 2023 and    
 2022, respectively –   331,331  
Stockholders’ Equity:     
 Preferred stock, $0.0001 par value, 1,000,000 shares authorized:    
 Series A preferred stock, 8,635 shares designated, 50 and 8,635 shares issued    
 and outstanding as of December 31, 2023 and 2022, respectively –   1  
 Series B preferred stock, 5,062 shares designated, 5,062 shares issued    
 and outstanding as of December 31, 2023 and 2022, respectively 1   1  
 Common stock, $0.0001 par value, 250,000,000 shares authorized;    
 4,052,119 and 257,604 shares issued at December 31, 2023 and 2022,    
 respectively, and 4,052,057 and 257,604 shares outstanding as of    
 December 31, 2023 and 2022, respectively 405   26  
 Additional paid-in-capital 114,300,484   104,584,147  
 Accumulated deficit (103,219,124)  (4,921,178) 
 Treasury stock, at cost, 62 and 0 shares at December 31, 2023    
 and 2022, respectively (7,168)  –  
  Total Stockholders’ Equity 11,074,598   99,662,997  
        
  Total Liabilities, Temporary Equity and Stockholders’ Equity$22,114,284  $118,506,271  
        
ZYVERSA THERAPEUTICS, INC. 
CONSOLIDATED STATEMENTS OF OPERATIONS 
  
          
   Successor  Predecessor 
   For the For the period  For the period 
   Year Ended December 13 through  January 1 through 
   December 31, December 31,  December 12, 
    2023   2022    2022  
          
          
          
Operating Expenses:       
 Research and development$3,207,573  $399,894   $5,407,859  
 General and administrative 11,213,201   420,174    7,605,205  
 Impairment of in-process research and development 81,438,426   –    –  
 Impairment of goodwill 11,895,033   –    –  
  Total Operating Expenses 107,754,233   820,068    13,013,064  
          
  Loss From Operations (107,754,233)  (820,068)   (13,013,064) 
          
Other (Income) Expense:       
 Interest (income) expense (457)  –    427,542  
 Change in fair value of derivative liabilities –   –    607,001  
          
  Pre-Tax Net Loss (107,753,776)  (820,068)   (14,047,607) 
  Income tax benefit 9,455,830   745,050    –  
  Net Loss (98,297,946)  (75,018)   (14,047,607) 
  Deemed dividend to preferred stockholders (7,948,209)  –    (10,015,837) 
  Net Loss Attributable to Common Stockholders$(106,246,155) $(75,018)  $(24,063,444) 
          
          
  Net Loss Per Share       
  – Basic and Diluted$(108.97) $(0.29)  $(0.99) 
          
  Weighted Average Number of       
  Common Shares Outstanding       
  – Basic and Diluted 975,035   257,604    24,194,270  

Release – Tonix Pharmaceuticals Receives Rare Pediatric Disease Designation from the FDA for TNX-2900 for the Treatment of Prader-Willi Syndrome

Posted on by aweinsoff@channelchek.com

Research News and Market Data on TNXP

March 25, 2024 8:00am EDT

TNX-2900 is a proprietary magnesium-potentiated formulation of intranasal oxytocin, a naturally occurring hormone that reduces appetite and eating

Prader Willi syndrome is the most common genetic cause of life-threatening childhood obesity

CHATHAM, N.J., March 25, 2024 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a biopharmaceutical company with marketed products and a pipeline of development candidates, today announced the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to TNX-2900* (intranasal potentiated oxytocin), a proprietary magnesium (Mg2+)-potentiated formulation of intranasal oxytocin, to treat Prader-Willi syndrome (PWS) in children and adolescents. TNX-2900 was previously granted Orphan Drug designation by the FDA in 2022 for the treatment of PWS and the investigational new drug (IND) application was cleared by the FDA in 2023. The Company may be eligible to receive a transferable Priority Review Voucher if TNX-2900 for PWS is approved for marketing. Recently, vouchers have sold for approximately $100 million.

“The Rare Pediatric Disease Designation is an important regulatory milestone in the development of TNX-2900. With PWS being the most common genetic cause of life-threatening childhood obesity, we are excited that the FDA has recognized this significant unmet need in children and adolescents, particularly for PWS hyperphagia, which currently has no approved treatments1,2,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “As PWS is a genetic disorder associated with abnormalities of the oxytocin system, Tonix believes TNX-2900’s unique formulation has the potential to improve intranasal oxytocin’s therapeutic action by addressing limitations in efficacy observed at high-dose intranasal oxytocin that is not Mg2+-potentiated3,4.”

The FDA defines a rare pediatric disease as a serious or life-threating disease that primarily affects individuals aged from birth to 18 years and affects under 200,000 people in the United States.

About FDA’s Rare Pediatric Disease Priority Voucher Program

The FDA’s Rare Pediatric Disease Priority Voucher Program is intended to encourage the development of new drugs to treat certain rare pediatric diseases. Under the FDA’s Rare Pediatric Disease Designation and Voucher Program, if TNX-2900 is approved for marketing, Tonix may qualify for a priority review voucher that can be redeemed to receive priority review of a subsequent marketing application for a different product. Priority review vouchers may also be sold or transferred to another sponsor. The new sponsor can redeem the voucher to receive priority review for a different product, which reduces the review time of NDAs from 10 months to six months. There is no limit on the number of times a priority review voucher can be transferred. A 2020 U.S. Government Accounting Office analysis5 of the voucher program found that in the ten years since launch of the program in 2009, the price of buying priority review vouchers ranged from $67 million to $350 million. More recently, priority review vouchers were acquired by Novo Nordisk for $110 million in June of 2022, and by Novartis for $100 million from Marinus in July of 2022.6   Bluebird Bio sold vouchers for $102 million, $95 million and $103 million in November 2022, January 2023, and October 2023, respectively.7-9 In June of 2023, Novartis bought a priority review voucher from Pharming for $21 million, a price that had been negotiated as part of a purchase agreement when Pharming acquired the asset from Novartis.5

About Prader-Willi Syndrome (PWS)

PWS is recognized as the most common genetic cause of life-threatening childhood obesity and affects males and females with equal frequency and all races and ethnicities. PWS results from the absence of expression of a group of genes on the paternally acquired chromosome 15. The hallmarks of PWS are lack of suckling in newborns and, in children and adolescents, severe hyperphagia – an overriding physiological drive to eat, leading to severe obesity and other complications associated with significant mortality. A systematic review of the morbidity and mortality as a consequence of hyperphagia in PWS found that the average age of death in PWS was 22.1 years.10 There is no approved medication to treat poor feeding in newborns or hyperphagia in children and adolescents with PWS. Given the serious or life-threatening manifestations of these conditions, there is a critical need for effective treatments to decrease morbidity and mortality, improve quality of life, and increase life expectancy in people with PWS. Oxytocin has potent effects in correcting behavioral characteristics of the Magel2 knock-out mouse model for PWS and autism.11-13 Six clinical trials have investigated intranasal oxytocin as a treatment in pediatric patients with PWS. Four studies showed evidence for improvement in PWS-related behaviors/symptoms14-17; three clinical studies reported evidence for improvement in hyperphagia14,15,17; and one clinical study showed an improvement in sucking in infants16.

About TNX-2900 and Tonix’s Potentiated Oxytocin Platform

TNX-2900 is based on Tonix’s patented intranasal Mg2+-potentiated oxytocin formulation intended for use by children and adolescents. This formulation is believed to enhance the potency of oxytocin as well as increase specificity for oxytocin receptors relative to vasopressin receptors, potentially reducing unwanted side effects from activating vasopressin receptors. Tonix is also developing a different intranasal formulation, designated TNX-1900 for adolescent obesity, binge eating disorder, bone health in autism, and social anxiety disorder. Oxytocin is a naturally occurring human hormone that acts as a neurotransmitter in the brain. Oxytocin is believed to be more than 600 million years old and is present in vertebrates including mammals, birds, reptiles, amphibians, and fish.18 It was initially approved by the U.S. Food and Drug Administration as Pitocin®**, an intravenous infusion or intramuscular injection drug, for use in pregnant women to induce labor and control postpartum bleeding or hemorrhage. An intranasal formulation of oxytocin is marketed in some European countries to assist in breast milk production as Syntocinon®*** (oxytocin nasal 40 units/ml).

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. Tonmya™ is conditionally accepted by the U.S. Food and Drug Administration as the tradename for TNX-102 SL for the management of fibromyalgia.

**Pitocin® is a trademark of Par Pharmaceutical, Inc.

***Syntocinon® is a trademark of BGP Products Operations GmbH

Citations

  1. Meyerowitz JG, et al. Nat Struct Mol Biol. 2022. 29(3):274-281.
  2. Meziane H, et al. Biol Psychiatry. 2015. 78(2):85-94.
  3. Correa-da-Silva F, et al. J Neuroendocrinol. 2021. 33(7):e12994.
  4. Bharadwaj VN, et al. Pharmaceutics. 2022. 14(5):1105.
  5. U.S. Government Accounting Office, Jan 31, 2020. “Drug Development: FDA’s Priority Review Voucher Programs”. GAO-20-251. https://www.gao.gov/products/gao-20-251.
  6. Waldron, J. Fierce Biotech. June 1, 2023. “Novartis buys priority review voucher from Pharming for discount price of $21 M.” www.fiercebiotech.com/biotech/novartis-buys-priority-review-voucher-pharming-discount-price-21m
  7. BlueBird bio Press Release. Nov. 30, 2022 “bluebird bio Sells Priority Review Voucher for $102 Million”. https://investor.bluebirdbio.com/news-releases/news-release-details/bluebird-bio-sells-priority-review-voucher-102-million
  8. Kansteiner, F. Fierce Pharma. Jan 6, 2023 “Bluebird scores $95M nest egg after selling second FDA priority review voucher to BMS”. http://www.fiercepharma.com/pharma/bluebird-scores-another-nest-egg-after-selling-second-fda-review-voucher-bms-95m 
  9. Business Wire. October 30, 2023. “bluebird bio Enters into Advance Agreement to Sell Priority Review Voucher, if Granted, for $103 Million.” http://finance.yahoo.com/news/bluebird-bio-enters-advance-agreement-120000768.html
  10. Bellis SA, et al. Eur J Med Genet. 2022. 65(1):104379.
  11. Bertoni A, et al. Mol Psychiatry. 2021. 26(12):7582-7595.
  12. Schaller F, et al. Hum Mol Genet. 2010. 19:4895-4905.
  13. Meziane H, et al. Biol Psychiatry. 2015. 78: 85-94.
  14. Kuppens RJ, et al. Clin Endocrinol. 2016. 85:979-987.
  15. Miller JL et al. Am J Med Genet A. 2017. 173: 1243-1250.
  16. Tauber M, et al. Pediatrics. 2017. 139(2):e20162976.
  17. Damen L, et al. Clin Endocrinol. 2020. 94:774-785.
  18. Gruber CW. Exp Physiol. 2014. 99(1):55-61. doi: 10.1113/expphysiol.2013.072561.

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 March 25, 2024

Release – Unicycive Therapeutics to Be Featured In Multiple Presentations At The Upcoming European Renal Association Congress

Posted on by aweinsoff@channelchek.com

Research News and Market Data on UNCY

March 25, 2024 7:03am EDT Download as PDF

LOS ALTOS, Calif., March 25, 2024 (GLOBE NEWSWIRE) — Unicycive Therapeutics, Inc. (Nasdaq: UNCY), a clinical-stage biotechnology company developing therapies for patients with kidney disease (the “Company or “Unicycive”), today announced that multiple presentations will be delivered on the Company’s product candidates, oxylanthanum carbonate (OLC) and UNI-494, at the 61st European Renal Association (ERA) Congress taking place May 23-26, 2024, in Stockholm, Sweden.

Shalabh Gupta, MD, Chief Executive Officer of Unicycive, commented, “The ERA Congress is one of the most prominent nephrology meetings of the year, and we are excited to deliver presentations on both OLC and UNI-494. In addition to presenting preclinical data supporting both of our programs, we will also be reporting on our two clinical trials in progress. We look forward to participating in this important event.”

Oxylanthanum Carbonate (OLC)

Title:Enhanced Urinary Phosphorous Reduction: Comparative Study of Oxylanthanum Carbonate and Tenapanor in Rats
Lead Author:Satya Medicherla, Ph.D., Vice President, Preclinical Pharmacology, Unicycive
Type:Focused Oral Presentation
Dates/Times:May 25, 2024 from 12:10 p.m. – 12:15 p.m. CEST
  
Title:Oxylanthanum Carbonate for Hyperphosphatemia in End Stage Kidney Disease (ESKD): Tolerability Trial in Progress
Lead Author:Pablo E. Pergola, M.D., Ph.D., Renal Associates, P.A.
Type:ePoster
Date/Time:Available throughout the conference
  

UNI-494

Title:Oral Administration of UNI-494 Ameliorates Acute Kidney Injury in a Rat Model of Delayed Graft Function
Lead Author: Satya Medicherla, Ph.D., Vice President, Preclinical Pharmacology, Unicycive
Type:Focused Oral Presentation
Dates/Times:May 25, 2024 from 12:00 p.m. – 12:05 p.m. CEST
  
Title:UNI-494 Phase I Tolerability and Pharmacokinetics: Trial in Progress
Lead Author:Guru Reddy, Ph.D., Vice President of Preclinical R&D, Unicycive
Type:Focused Oral Presentation
Dates/Times:May 25, 2024 from 12:45 p.m. – 12:50 p.m. CEST
  

About Oxylanthanum Carbonate (OLC)

Oxylanthanum carbonate is a next-generation lanthanum-based phosphate binding agent utilizing proprietary nanoparticle technology being developed for the treatment of hyperphosphatemia in patients with chronic kidney disease (CKD). OLC has over forty issued and granted patents globally. Its potential best-in-class profile may have meaningful patient adherence benefits over currently available treatment options as it requires a lower pill burden for patients in terms of number and size of pills per dose that are swallowed instead of chewed. Based on a survey conducted in 2022, Nephrologists stated that the greatest unmet need in the treatment of hyperphosphatemia with phosphate binders is a lower pill burden and better patient compliance.1 The global market opportunity for treating hyperphosphatemia is projected to be in excess of $2.5 billion in 2023, with the United States accounting for more than $1 billion of that total. Despite the availability of several FDA-cleared medications, 75 percent of U.S. dialysis patients fail to achieve the target phosphorus levels recommended by published medical guidelines.

Unicycive is seeking FDA approval of OLC via the 505(b)(2) regulatory pathway. As part of the clinical development program, two clinical studies were conducted in over 100 healthy volunteers. The first study was a dose-ranging Phase I study to determine safety and tolerability. The second study was a randomized, open-label, two-way crossover bioequivalence study to establish pharmacodynamic bioequivalence between OLC and Fosrenol. Based on the topline results of the bioequivalence study, pharmacodynamic (PD) bioequivalence of OLC to Fosrenol was established.

Fosrenol® is a registered trademark of Shire International Licensing BV.
1Reason Research, LLC 2022 survey. Results here.

About Hyperphosphatemia

Hyperphosphatemia is a serious medical condition that occurs in nearly all patients with End Stage Renal Disease (ESRD). If left untreated, hyperphosphatemia leads to secondary hyperparathyroidism (SHPT), which then results in renal osteodystrophy (a condition similar to osteoporosis and associated with significant bone disease, fractures and bone pain); cardiovascular disease with associated hardening of arteries and atherosclerosis (due to deposition of excess calcium-phosphorus complexes in soft tissue). Importantly, hyperphosphatemia is independently associated with increased mortality for patients with chronic kidney disease on dialysis. Based on available clinical data to date, over 80% of patients show signs of cardiovascular calcification by the time they become dependent on dialysis.

Dialysis patients are already at an increased risk for cardiovascular disease (because of underlying diseases such as diabetes and hypertension), and hyperphosphatemia further exacerbates this. Treatment of hyperphosphatemia is aimed at lowering serum phosphate levels via two means: (1) restricting dietary phosphorus intake; and (2) using, on a daily basis, and with each meal, oral phosphate binding drugs that facilitate fecal elimination of dietary phosphate rather than its absorption from the gastrointestinal tract into the bloodstream.

About UNI-494

UNI-494 is a novel nicotinamide ester derivative and a selective ATP-sensitive mitochondrial potassium channel activator. Mitochondrial dysfunction plays a critical role in the progression of acute kidney injury and chronic kidney disease. UNI-494 has a novel mechanism of action that restores mitochondrial function and may be beneficial for the treatment of several diseases including kidney disease. Unicycive is currently conducting a Phase 1 dose-ranging safety study in healthy volunteers in the United Kingdom that is expected to complete in 2H of 2024. UNI-494 is protected by issued patent(s) in the U.S. and Europe and a wide range of patent applications worldwide. UNI-494 has been granted orphan drug designation (ODD) by the U.S. Food and Drug Administration (FDA) for the prevention of Delayed Graft Function (DGF) in kidney transplant patients.

About Delayed Graft Function

Delayed Graft Function (DGF) refers to the acute kidney injury (AKI) that occurs in the first week after kidney transplantation, which necessitates dialysis intervention. As the name indicates, DGF can result in sub-optimal or impaired graft function and is one of the most common and serious complications of kidney transplantation. Poor kidney function in the first week of graft life is detrimental to the longevity of the allograft. DGF is also associated with higher rates of tissue rejection and decreased patient survival. Currently, there are no FDA approved drugs for the treatment of DGF.

Ischemia/reperfusion injury (IRI) is known to be a major causative factor for the AKI that results in DGF during kidney transplantation. Ischemic preconditioning, that works by activating KATP channels in mitochondria, is a natural endogenous mechanism which protects cells from IRI in the heart, kidney, liver, and other organs. UNI-494 is a pharmacological approach that emulates and enhances this natural phenomenon of ischemic preconditioning.

About Unicycive Therapeutics

Unicycive Therapeutics is a biotechnology company developing novel treatments for kidney diseases. Unicycive’s lead drug candidate, oxylanthanum carbonate (OLC), is a novel investigational phosphate binding agent being developed for the treatment of hyperphosphatemia in chronic kidney disease patients on dialysis. UNI-494 is a patent-protected new chemical entity in late preclinical development for the treatment of acute kidney injury. For more information, please visit Unicycive.com and follow us on LinkedIn and YouTube.

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 using words such as “anticipate,” “believe,” “forecast,” “estimated” and “intend” or other similar terms or expressions that concern Unicycive’s expectations, strategy, plans or intentions. These forward-looking statements are based on Unicycive’s current expectations and actual results could differ materially. There are several 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, clinical trials involve a lengthy and expensive process with an uncertain outcome, and results of earlier studies and trials may not be predictive of future trial results; our clinical trials may be suspended or discontinued due to unexpected side effects or other safety risks that could preclude approval of our product candidates; risks related to business interruptions, which could seriously harm our financial condition and increase our costs and expenses; dependence on key personnel; substantial competition; uncertainties of patent protection and litigation; dependence upon third parties; and risks related to failure to obtain FDA clearances or approvals and noncompliance with FDA regulations. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties related to market conditions and other factors described more fully in the section entitled ‘Risk Factors’ in Unicycive’s Annual Report on Form 10-K for the year ended December 31, 2022, and other periodic reports filed with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and Unicycive specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

Investor Contact:

ir@unicycive.com 
(650) 543-5470

SOURCE: Unicycive Therapeutics, Inc

Released March 25, 2024

Eledon Pharmaceuticals (ELDN) – First Transplant Of A Pig Kidney Uses Tegoprubart To Prevent Immune Rejection

Posted on by cpereira@noblefcm.com


Friday, March 22, 2024

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.

First Transplant Of A Genetically Modified Kidney To A Human. Eledon announced that tegoprubart, its drug in development to prevent organ transplant rejection, was used in the first transplant of an engineered pig kidney into a human patient. The kidney was genetically engineered by eGenesis, Inc, a company that has been collaborating with Eledon. Tegoprubart is currently in a Phase 2 clinical trial testing it against tacrolimus in prevention of kidney transplant rejection.

Eledon Has A Research Collaborated With eGenesis. The pig kidney was engineered by eGenesis, Inc, a company that formed a research collaboration with Eledon in January 2023. eGenesis has proprietary technology for engineering genetic changes to animal organs to make them compatible for transplantation into humans. As described in our Research Note on October 16, these genetic changes reduce the cross-species immunological barriers. The agreement allows eGenesis to use tegoprubart in its xenotransplantation research.


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Release – MAIA Biotechnology Welcomes Prominent Medical Oncology Scientist Dr. Saadettin Kilickap to Its Scientific Advisory Board

Posted on by aweinsoff@channelchek.com

Research News and Market Data on MAIA

March 21, 2024 3:00pm EDT

  • Acclaimed academic researcher has led 40+ multicenter phase 2 and phase 3 clinical studies

CHICAGO–(BUSINESS WIRE)– MAIA Biotechnology, Inc., (NYSE American: MAIA) (“MAIA”, the “Company”), a clinical-stage biopharmaceutical company developing targeted immunotherapies for cancer, today announced the appointment of Professor Saadettin Kilickap, M.D. to its Scientific Advisory Board (SAB).

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20240321173734/en/

Professor Saadettin Kilickap, M.D., Scientific Advisor to MAIA Biotechnology (Photo: Business Wire)

Dr. Kilickap is a professor at the Istinye University Faculty of Medicine, Department of Medical Oncology, Liv Hospital in Turkey. His research focuses on medical oncology and cancer epidemiology, including solid tumors such as lung cancer, breast cancer, melanoma, and gastrointestinal system cancers, as well as targeted therapies and immunotherapy.

“Saadettin has served as principal or sub-investigator in more than 40 national and international multi-center phase 2 and phase 3 studies, many of which were related to lung cancer,” said Chief Executive Officer Vlad Vitoc, M.D. “He is a prominent voice on medical oncology and cancer epidemiology, with a special interest in quality of life for cancer patients. We are delighted to welcome him as Scientific Advisor as we begin to clear major clinical inflection points this year and progress with our groundbreaking cancer research.”

Prior to his current appointment, Dr. Kilickap was a professor at the Preventive Oncology Department of Hacettepe University Cancer Institute of Turkey. Earlier he worked in the Department of Hematology-Oncology at Regensburg University in Germany and was a faculty member at the Sivas Cumhuriyet University Faculty of Medicine, Department of Internal Medicine, in Turkey.

Dr. Kilickap graduated with honors from Gazi University Faculty of Medicine and went on to complete his internal medicine residency training at Hacettepe University Faculty of Medicine, both located in Ankara, Turkey. He completed his fellowship training at Hacettepe and became a medical oncology specialist in 2009. In the same year, he graduated from the Cancer Epidemiology Master’s Program at the Hacettepe University Oncology Institute, Department of Preventive Oncology.

Dr. Kilickap has authored more than 240 scientific articles published in international peer-reviewed journals, and delivered more than 50 poster presentations at international congresses.

About MAIA Biotechnology, Inc.

MAIA is a targeted therapy, immuno-oncology company focused on the development and commercialization of potential first-in-class drugs with novel mechanisms of action that are intended to meaningfully improve and extend the lives of people with cancer. Our lead program is THIO, a potential first-in-class cancer telomere targeting agent in clinical development for the treatment of NSCLC patients with telomerase-positive cancer cells. For more information, please visit www.maiabiotech.com.

Forward Looking Statements

MAIA cautions that all statements, other than statements of historical facts contained in this press release, are forward-looking statements. Forward-looking statements are subject to known and unknown risks, uncertainties, and other factors that may cause our or our industry’s actual results, levels or activity, performance or achievements to be materially different from those anticipated by such statements. The use of words such as “may,” “might,” “will,” “should,” “could,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify forward looking statements. However, the absence of these words does not mean that statements are not forward-looking. For example, all statements we make regarding (i) the initiation, timing, cost, progress and results of our preclinical and clinical studies and our research and development programs, (ii) our ability to advance product candidates into, and successfully complete, clinical studies, (iii) the timing or likelihood of regulatory filings and approvals, (iv) our ability to develop, manufacture and commercialize our product candidates and to improve the manufacturing process, (v) the rate and degree of market acceptance of our product candidates, (vi) the size and growth potential of the markets for our product candidates and our ability to serve those markets, and (vii) our expectations regarding our ability to obtain and maintain intellectual property protection for our product candidates, are forward looking. All forward-looking statements are based on current estimates, assumptions and expectations by our management that, although we believe to be reasonable, are inherently uncertain. Any forward-looking statement expressing an expectation or belief as to future events is expressed in good faith and believed to be reasonable at the time such forward-looking statement is made. However, these statements are not guarantees of future events and are subject to risks and uncertainties and other factors beyond our control that may cause actual results to differ materially from those expressed in any forward-looking statement. Any forward-looking statement speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. In this release, unless the context requires otherwise, “MAIA,” “Company,” “we,” “our,” and “us” refers to MAIA Biotechnology, Inc. and its subsidiaries.

View source version on businesswire.com: https://www.businesswire.com/news/home/20240321173734/en/

Investor Relations Contact
+1 (872) 270-3518
ir@maiabiotech.com

Source: MAIA Biotechnology, Inc.

Released March 21, 2024

Breakthrough: First Genetically Modified Pig Kidney Transplanted into Human

Posted on by slightbourne@noblecapitalmarkets.com

In a groundbreaking medical procedure, surgeons at Massachusetts General Hospital have successfully transplanted a genetically modified pig’s kidney into a human patient – the first xenotransplantation of its kind. This historic operation, performed on March 16th, 2024, provides hope that xenotransplantation could one day help alleviate the chronic shortage of human organs available for those desperately awaiting a life-saving transplant.

The recipient was a 62-year-old man suffering from end-stage kidney disease. After his body initially accepted the pig kidney, a drug regimen was immediately administered to prevent rejection, including the experimental anti-rejection therapy tegoprubart developed by Eledon Pharmaceuticals.

“This first-ever kidney xenotransplant marks a pivotal moment for the transplant community,” stated David-Alexandre C. Gros, M.D., CEO of Eledon Pharmaceuticals. “We are thankful for the opportunity to participate in this landmark procedure as we work to develop tegoprubart as a new and potentially superior immunosuppressive option for transplant patients.”

Preventing Rejection Key to Xenotransplant Success
The greatest challenge in xenotransplantation is preventing the recipient’s body from violently rejecting the foreign organ. Tegoprubart targets a protein called CD40 ligand (CD40L) that plays a central role in activating the immune system’s responses against the transplanted organ.

“Therapies targeting CD40L, like tegoprubart, are critical to controlling the immune response to the xenograft, potentially leading to superior long-term outcomes compared to other immunosuppressive therapies,” explained Andrew Adams, M.D., Ph.D., Chief of Transplant Surgery at the University of Minnesota.

Tegoprubart has already shown promise in preventing rejection in previous pig-to-human heart xenotransplants as well as in ongoing clinical trials for human-to-human kidney transplants.

“We have seen tegoprubart be safe and well-tolerated while successfully preventing rejection and enabling above-average kidney function post-transplant when used as part of an immunosuppressive regimen in our Phase 1b kidney transplant study,” said Dr. Gros.

A Pivotal Moment of Hope
For the medical team at Massachusetts General Hospital, this pioneering procedure represents a significant milestone with profound implications for the future of transplant medicine.

“Xenotransplantation represents a unique approach with the potential to provide patients with additional options to access life-saving treatments in a timely manner,” said Dr. Leonardo V. Riella, Medical Director for Kidney Transplantation at MGH. “We commend the courage of our patient and look forward to continued advancements to make this option available to more patients.”

More than 90,000 Americans are currently on the national waiting list for a kidney transplant, with thousands more awaiting other life-saving organ donations. The ability to use genetically engineered animal organs could dramatically increase the supply available for transplant.

“This procedure provides hope that xenotransplantation may one day help solve the current shortage of available organs,” noted Dr. Gros. “While still at an experimental stage, it’s an exciting development for the entire transplant field.”

Critical Next Steps
Eledon is conducting further preclinical studies evaluating tegoprubart’s ability to prevent rejection in non-human primate recipients of xenotransplanted organs. The company is also running a Phase 2 clinical trial called BESTOW directly comparing tegoprubart head-to-head against the standard anti-rejection drug tacrolimus for preventing kidney rejection in human-to-human transplants.

While this first xenotransplant procedure is an incredible medical achievement, significant additional research is still needed to prove the technique can lead to consistently successful long-term outcomes. However, this unprecedented operation has opened up a new frontier of possibilities for making organ transplants more widely available to the hundreds of thousands in need.

Take a moment to watch an intriguing panel discussion on xenotransplantation with CEO of Eledon Pharmaceuticals, Dr. DA Gros and other prominent figures in the field at NobleCon19.

Release – Eledon Pharmaceuticals Announces Use of Tegoprubart in First-ever Transplant of Genetically Modified Kidney from a Pig to a Human

Posted on by aweinsoff@channelchek.com

Research News and Market Data on ELDN

March 21, 2024

Historic kidney xenotransplantation procedure conducted at Massachusetts General Hospital

Tegoprubart administration has now been used investigationally to prevent rejection in both kidney and heart pig-to-human xenotransplantations, as well as in human-to-human kidney transplantation

Eledon recently presented results from its ongoing Phase 1b kidney transplantation study which demonstrated that tegoprubart was generally safe and well tolerated and successfully prevented rejection with post-transplant kidney function above historical averages

IRVINE, Calif., March 21, 2024 (GLOBE NEWSWIRE) — Eledon Pharmaceuticals, Inc. (“Eledon”) (NASDAQ: ELDN) today announced that tegoprubart, the company’s investigational anti-CD40L antibody, was used as a component of the immunosuppressive treatment regimen following the first-ever transplant of a kidney from a genetically modified pig to a human. The procedure was completed on March 16, 2024, at Massachusetts General Hospital on a 62-year-old man living with end-stage kidney disease.

“This first-ever kidney xenotransplant marks a pivotal moment for the transplant community and provides hope that this option may one day help solve the current shortage of available organs,” said David-Alexandre C. Gros, M.D., Eledon Chief Executive Officer. “Eledon has now participated in both heart and kidney xenotransplant procedures, further demonstrating tegoprubart’s broad potential in transplant. We are thankful to the patient, the entire medical team at Massachusetts General Hospital, and our partner eGenesis for the privilege to participate in this landmark procedure as we work to achieve our goal of developing tegoprubart as a new and better immunosuppressive option for transplant patients.”

Tegoprubart is being administered to the patient investigationally as part of a regimen designed to suppress the immune system and prevent the body from rejecting the transplanted pig organ. Tegoprubart has been observed to be safe and well-tolerated in multiple studies and in multiple indications, including for the prevention of rejection following kidney transplantation.

“It is exciting to see the clinical application of xenotransplantation to a patient with end stage renal disease,” said Andrew Adams, MD, PhD, Chief, Division of Transplant Surgery, University of Minnesota. “Based on all of the studies performed in preclinical models to date, it is clear that therapies targeting CD40L, like tegoprubart, are critical to controlling the immune response to the xenograft, potentially leading to superior long-term outcomes compared to other immunosuppressive therapies. CD40L sits at the interface of the adaptive and innate immune responses which may explain why therapies designed to block it have such potent effects in xenotransplantation.”

“This procedure represents a significant milestone in the transplantation field and a promising step to address a medical crisis: the worldwide shortage of available organs,” said Leonardo V. Riella, MD, PhD, Medical Director for Kidney Transplantation at Massachusetts General Hospital. “Xenotransplantation represents a unique approach with the potential to provide patients with additional options to access life-saving treatments in a timely manner. We commend the courage of our patient and the skill of the entire team involved in the operation, and I look forward to continued advancements in research with the hope that we can make this novel treatment option available to more patients in the future.”

Multiple clinical and preclinical research efforts are currently underway to evaluate the ability of tegoprubart to reduce the risk of rejection in organ transplant. Eledon is advancing preclinical studies in which tegoprubart is being used as a part of the immunosuppression regimen designed to reduce the risk of rejection in nonhuman primate recipients in xenotransplant procedures. In parallel, Eledon is running two global clinical studies evaluating tegoprubart for the prevention of organ rejection in persons receiving a de novo kidney transplant. The company recently presented results from 11 participants enrolled in its ongoing Phase 1b kidney transplantation study, which demonstrated that tegoprubart, as part of a calcineurin inhibitor free immunosuppressive regimen, was generally safe and well tolerated and both successfully prevented rejection as well as permitted above historical average post-transplant kidney function. The company’s Phase 2 BESTOW study, assessing tegoprubart head-to-head with tacrolimus for the prevention of rejection in kidney transplantation, is currently recruiting participants, and plans to complete enrollment at the end of 2024.

About Eledon Pharmaceuticals and tegoprubart

Eledon Pharmaceuticals, Inc. is a clinical stage biotechnology company that is developing immune-modulating therapies for the management and treatment of life-threatening conditions. The Company’s lead investigational product is tegoprubart, an anti-CD40L antibody with high affinity for the CD40 Ligand, a well-validated biological target that has broad therapeutic potential. The central role of CD40L signaling in both adaptive and innate immune cell activation and function positions it as an attractive target for non-lymphocyte depleting, immunomodulatory therapeutic intervention. The Company is building upon a deep historical knowledge of anti-CD40 Ligand biology to conduct preclinical and clinical studies in kidney allograft transplantation, xenotransplantation, and amyotrophic lateral sclerosis (ALS). Eledon is headquartered in Irvine, California. For more information, please visit the Company’s website at www.eledon.com.

Follow Eledon Pharmaceuticals on social media: LinkedIn; Twitter

Forward-Looking Statements

This press release contains forward-looking statements that involve substantial risks and uncertainties. Any statements about the company’s future expectations, plans and prospects, including statements about planned clinical trials, the development of product candidates, expected timing for initiation of future clinical trials, expected timing for receipt of data from clinical trials, as well as other statements containing the words “believes,” “anticipates,” “plans,” “expects,” “estimates,” “intends,” “predicts,” “projects,” “targets,” “looks forward,” “could,” “may,” and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are inherently uncertain and are subject to numerous risks and uncertainties, including: risks relating to the safety and efficacy of our drug candidates; risks relating to clinical development timelines, including interactions with regulators and clinical sides, as well as patient enrollment; risks relating to costs of clinical trials and the sufficiency of the company’s capital resources to fund planned clinical trials; and risks associated with the impact of the ongoing coronavirus pandemic. Actual results may differ materially from those indicated by such forward-looking statements as a result of various factors. These risks and uncertainties, as well as other risks and uncertainties that could cause the company’s actual results to differ significantly from the forward-looking statements contained herein, are discussed in our quarterly 10-Q, annual 10-K, and other filings with the U.S. Securities and Exchange Commission, which can be found at www.sec.gov. Any forward-looking statements contained in this press release speak only as of the date hereof and not of any future date, and the company expressly disclaims any intent to update any forward-looking statements, whether as a result of new information, future events or otherwise.

Investor Contact:

Stephen Jasper
Gilmartin Group
(858) 525 2047
stephen@gilmartinir.com

Media Contact:

Jenna Urban
Berry & Company Public Relations
(212) 253 8881
jurban@berrypr.com

Source: Eledon Pharmaceuticals

Source: Eledon Pharmaceuticals, Inc.

Release – Tonix Pharmaceuticals Announces Poster Presentation Describing Discovery of Novel Next-Generation Oxytocin Analogues at the American Chemistry Society (ACS) Spring 2024 Meeting

Posted on by aweinsoff@channelchek.com

Research News and Market Data on TNXP

March 21, 2024 8:00am EDT

Four Phase 2 investigator-initiated studies of TNX-1900 (intranasal potentiated oxytocin) are ongoing for pediatric obesity, binge eating disorder, bone health in autism and social anxiety disorder

TNX-2900 (intranasal potentiated oxytocin) is being developed under an IND as a treatment for Prader-Willi Syndrome, an Orphan Disease characterized by excessive eating

TNX-1900 and TNX-2900 may serve as novel neuroendocrine treatments for certain pain, eating and endocrine disorders 

CHATHAM, N.J., March 21, 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 announces a poster presentation at the American Chemistry Society (ACS) Spring 2024 Meeting, held March 17-21, 2024, in New Orleans, Louisiana. A copy of the poster is available under the scientific presentations page of the Tonix website at www.tonixpharma.com.

The poster presentation titled, Oxytocin Analogs with Enhanced Craniofacial Antinociceptive Effects in Low Magnesium Formulations, describes the discovery and characterization of novel oxytocin analogues that are candidate treatments for craniofacial pain, excessive eating (including Prader Willi Syndrome), and endocrinological conditions including bone health in autism and insulin resistance.

“Intranasal oxytocin has several potential therapeutic applications,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “Preclinical studies have shown that oxytocin, a hypothalamic peptide hormone, simultaneously reduces food intake and increases energy expenditure, leading to weight loss.1-3 Intranasal oxytocin is well-tolerated and in published studies of adults, results in reduced caloric intake, increased fat burning and improved insulin sensitivity.1-3”

Dr. Lederman continued, “There is preclinical evidence that the activity of intranasal oxytocin is dependent on magnesium (Mg++) concentration.4-6 Our current intranasal oxytocin formulations of TNX-1900 and TNX-2900 contain Mg++ to augment the activity. We believe the new oxytocin analogues described in the poster have enhanced binding to Mg++ and consequently their activity does not require Mg++ augmentation.”

Four Phase 2 investigator-initiated studies of TNX-1900 are currently ongoing; three at the Massachusetts General Hospital (MGH) and one at the University of Washington. The Phase 2 ‘POWER’ study at MGH is investigating the efficacy and safety of TNX-1900 as a novel therapeutic agent to induce weight loss and improve indicators of cardiometabolic risk in adolescent patients with obesity. The Phase 2 ‘STROBE’ study at MGH is investigating the efficacy and safety of TNX-1900 as a novel therapeutic agent to reduce binge eating frequency in adults with binge-eating disorder. The Department of Defense (DoD)-funded Phase 2 ‘BOX’ study at MGH is investigating the efficacy and safety of TNX-1900 as a novel therapeutic agent to improve bone health in children with autism spectrum disorder. In addition, a Phase 2 study at the University of Washington is investigating the potential role of TNX-1900 in enhancing vicarious extinction learning in social anxiety disorder, compared to healthy controls.

About TNX-1900 and TNX-2900

TNX-1900 and TNX-2900 (intranasal potentiated oxytocin) are proprietary formulations of oxytocin. TNX-1900 is in Phase 2 development under investigator-initiated INDs as a candidate for adolescent obesity, binge eating disorder, bone health in autism and social anxiety disorder. TNX-1900 is also planned for development in treating insulin resistance. TNX-2900 is in development as a treatment for Prader Willi Syndrome. TNX-2900 has received orphan drug designation from the U.S. Food and Drug Administration (FDA) and its IND has been cleared. In 2020, TNX-1900 was acquired from Trigemina, Inc. who had licensed the technology underlying the composition and method from Stanford University. TNX-1900 is a drug-device combination product, based on an intranasal actuator device that delivers oxytocin into the nasal cavity. Tonix’s patented intranasal potentiated oxytocin formulation intended for use by adults and adolescents. Tonix’s patented potentiated oxytocin formulation is believed to increase specificity for oxytocin receptors relative to vasopressin receptors as well as to enhance the potency of oxytocin. Oxytocin is a naturally occurring human hormone that acts as a neurotransmitter in the brain. Oxytocin is believed to be more than 600 million years old and is present in vertebrates including mammals, birds, reptiles, amphibians and fish.7 It was originally approved by the U.S. Food and Drug Administration as Pitocin®*, an intravenous infusion or intramuscular injection drug, for use in pregnant women to induce labor. An intranasal formulation of oxytocin is marketed in some European countries to assist in the production of breast milk as Syntocinon®** (oxytocin nasal 40 units/ml). Oxytocin has no recognized addiction potential. Oxytocin, when delivered via the nasal route, concentrates in the trigeminal system1 resulting in binding of oxytocin to receptors on neurons in the trigeminal system. With TNX-1900 and TNX-2900, the addition of magnesium to the oxytocin formulation enhances oxytocin receptor binding8 as well as its inhibitory effects on trigeminal neurons and resultant craniofacial analgesic effects, as demonstrated in animal models9. Intranasal oxytocin has been shown to be well tolerated in several clinical trials in both adults and children10. Targeted nasal delivery results in low systemic exposure and lower risk of non-nervous system, off-target effects. Tonix also has a license with the University of Geneva to use TNX-1900 for the treatment of insulin resistance and related conditions.

About Prader-Willi Syndrome (PWS)

PWS is recognized as the most common genetic cause of life-threatening childhood obesity and affects males and females with equal frequency and all races and ethnicities. PWS results from the absence of expression of a group of genes on the paternally acquired chromosome 15. The hallmarks of PWS are lack of suckling in newborns and, in children and adolescents, severe hyperphagia, an overriding physiological drive to eat, leading to severe obesity and other complications associated with significant mortality. A systematic review of the morbidity and mortality as a consequence of hyperphagia in PWS found that the average age of death in PWS was 22.1 years.11 There is no approved medication to treat poor feeding in newborns or hyperphagia in children and adolescents with PWS. Given these serious or life-threatening manifestations of these conditions, there is a critical need for effective treatments to decrease morbidity and mortality, improve quality of life, and increase life expectancy in people with PWS. Oxytocin has potent effects in adult mice correcting behavioral characteristics of the Magel2 knock-out mouse model for PWS and autism.12 In addition, oxytocin has potent effects in correcting behavioral characteristics of the neonatal Magel2 knock-out mouse model for PWS and autism13 and intriguing effects in a clinical trial of neonates with PWS.14

*Pitocin® is a trademark of Par Pharmaceutical, Inc.

**Syntocinon® is a trademark of BGP Products Operations GmbH

References

1Lawson EA, et al. J Neuroendocrinol 2020;32(4):e12805. doi: 10.1111/jne.12805.
2Niu J, et al. Front Neurosci 2021;15:743546. doi: 10.3389/fnins.2021.743546.
3Maejima Y, et al. Neuroendocrinology 2018;107(1):91-104.
4Yeomans DC, et al. Transl Psychiatry. 2021. 11(1):388.
5Tzabazis A, et al. Cephalalgia. 2016. 36(10):943-50.
6Meyerowitz JG, et al. Nat Struct Mol Biol. 2022. 29(3):274-281.
7Gruber CW. Exp Physiol. 2014. 99(1):55-61. doi: 10.1113/expphysiol.2013.072561.
8Antoni FA and Chadio SE. Biochem J. 1989. 257(2):611-4.
9Cai Q, et al., Psychiatry Clin Neurosci. 2018. 72(3):140-151.
10Yeomans, DC et al. 2017. US patent US2017368095.
11Bellis SA, et al. Eur J Med Genet. 2022. 65(1):104379.
12Meziane H, et al. Biol Psychiatry. 2015. 78(2):85-94.
13Bertoni A, et al. Mol Psychiatry. 2021. 26(12):7582-7595.
14Tauber M, et al. Pediatrics. 2017. 139(2):e20162976.

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. Tonmya™ is conditionally accepted by the U.S. Food and Drug Administration as the tradename for TNX-102 SL for the management of fibromyalgia.

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
(919) 360-3039

Source: Tonix Pharmaceuticals Holding Corp.

Released March 21, 2024

Release – Tonix Pharmaceuticals Announces Selection of Two Contract Manufacturing Organizations for the Launch and Commercial Manufacture of Tonmya™ for the Management of Fibromyalgia

Posted on by aweinsoff@channelchek.com

Research News and Market Data on TNXP

March 20, 2024 8:00am EDTDownload as PDF

Tonmya™ is a potential new first-line, centrally acting non-opioid analgesic for the management of fibromyalgia, supported by positive results from two Phase 3 studies

New Drug Application (NDA) submission to the FDA planned for second half of 2024

CHATHAM, N.J., March 20, 2024 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a biopharmaceutical company with marketed products and a pipeline of development candidates, today announced it has selected two contract manufacturing organizations (CMOs), one of which is Almac Pharma Services, a member of the privately owned Almac Group, as dual supply sources for the potential launch and commercialization of Tonmya™ (also known as TNX-102 SL, cyclobenzaprine HCl sublingual tablets) in the U.S.

“Dual sourcing is a critical element for the successful commercial launch and supply chain management of a product,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “We are excited to advance our first internally developed program toward NDA submission and to work with two well-established CMOs for commercial supply and potential launch of Tonmya.”

“Having supported the development and clinical trial supply of this drug, we’re thrilled to be continuing our partnership with Tonix to support the commercial launch and ongoing supply of this important new non-opioid analgesic to patients with fibromyalgia, a chronic debilitating disease,” said Mark English, VP Operations, Almac Pharma Services.

Tonmya is a centrally acting, non-opioid medication. As previously announced, Tonix’s second positive Phase 3 study, RESILIENT, met its pre-specified primary endpoint, significantly reducing daily pain compared to placebo (p=0.00005) in participants with fibromyalgia. Statistically significant and clinically meaningful results (p=0.001 or better) were also seen in all key secondary endpoints related to improving sleep quality, reducing fatigue, and improving overall fibromyalgia symptoms and function. TNX-102 SL was well tolerated with an adverse event profile comparable to prior studies, and no new safety signals were observed.

Tonix plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration in the second half of 2024 for Tonmya for the management of fibromyalgia.

About Tonmya* (also known as TNX-102 SL)

TNX-102 SL is a tablet containing 2.8 mg cyclobenzaprine HCl that will be administered sublingually once daily at bedtime for the first 2 weeks, titrating subsequently to 2 tablets (5.6 mg total per day) at bedtime, as tolerated, for chronic, long-term use. The sublingual tablet is formulated using a patented Protectic™ eutectic formulation including a basifying agent for transmucosal absorption with rapid systemic exposure pharmacokinetic properties suitable for bedtime administration. The eutectic properties enhance the stability with a predicted shelf life of greater than 48 months, at room temperature conditions. The planned commercial distribution will be a 14, 60 and 90 count tablet bottles allowing for titration, flexible and three-month chronic supply. Tonmya is a centrally acting, non-opioid, non-addictive, bedtime medication. In December 2023, the Company announced highly statistically significant and clinically meaningful topline results in RESILIENT, a second positive Phase 3 clinical trial of Tonmya for the management of fibromyalgia. RELIEF, the first positive Phase 3 trial of Tonmya in fibromyalgia, was completed in December 2020. It met its pre-specified primary endpoint of daily pain reduction compared to placebo (p=0.010) and showed activity in key secondary endpoints.

*Tonmya™ is conditionally accepted by the U.S. Food and Drug Administration (FDA) as the tradename for TNX-102 SL for the management of fibromyalgia. Tonmya has not been approved for any indication.

About Almac Pharma Services

Tailormade pharmaceutical development and commercial solutions

With over 50 years’ experience, Almac Pharma Services is a world leading outsourcing partner to the global pharmaceutical and biotechnology industry.

Employing over 1,600 highly skilled individuals across 4 locations in Europe and the US, the company provides tailored, quality-led and timely solutions from early and late phase pharmaceutical development, clinical and commercial drug product manufacture, product launch through to commercial packaging and global distribution.

On March 6, 2024, the company announced the completion of a custom-built, high-volume facility that significantly increases commercial manufacturing and packaging of sachet drug product presentations forming part of the Group’s ongoing global expansion investment now totaling over £400 million.

To keep up to date with latest news, follow us on LinkedIn or visit our website.

About Almac Group

The Almac Group is an established contract development and manufacturing organisation providing an extensive range of integrated services across the drug development lifecycle to the pharmaceutical and biotech sectors globally. Its innovative services range from R&D, biomarker discovery development and commercialisation, API manufacture, analytical services, formulation development, clinical trial supply, IRT (IVRS/IWRS) through to commercial-scale manufacture.

The international company is a privately owned organisation which has grown organically, now employing 7,200 highly skilled personnel across 18 facilities including Europe, the USA and Asia.

To keep up to date with latest news, follow us on X and LinkedIn or visit almacgroup.com.

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 March 20, 2024