Health Archives - Page 51 of 225

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

Posted on March 21, 2024March 21, 2024 by aweinsoff@channelchek.com

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).

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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.

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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 March 21, 2024March 21, 2024 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 March 21, 2024March 21, 2024 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 March 21, 2024March 21, 2024 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 March 20, 2024March 20, 2024 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’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

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

Release – Tonix Pharmaceuticals Announces Transition to Fully Integrated Biopharmaceutical Company Expected on April 1, 2024

Posted on March 19, 2024March 19, 2024 by aweinsoff@channelchek.com

Research News and Market Data on TNXP

March 19, 2024 8:00am EDTDownload as PDF

Zembrace^® SymTouch^® and Tosymra^® will be marketed by Tonix Medicines, Tonix’s wholly-owned commercial subsidiary

Commercial capabilities prepare Tonix for the potential launch in 2025 of Tonmya™ for the management of fibromyalgia, assuming FDA approval

CHATHAM, N.J., March 19, 2024 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a biopharmaceutical company with marketed products and a pipeline of development candidates, today announced that it will achieve its goal of transitioning to a fully integrated pharmaceutical company on April 1, 2024. Since the acquisition of Zembrace^® SymTouch^® (sumatriptan injection) 3 mg and Tosymra^® (sumatriptan nasal spray) 10 mg on June 30, 2023, Tonix Pharmaceuticals has been putting in place the personnel, systems and contracts required to support a commercial organization. Both products are indicated for the treatment of acute migraine with or without aura in adults.

“Tonix will become a fully integrated biopharmaceutical company with a mission of developing and marketing innovative, high-value therapeutics,” said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. “Tonix will assume responsibility for the distribution, selling and marketing of Zembrace SymTouch and Tosymra, as well as supply chain, regulatory and quality control of the two products.”

Dr. Lederman added, “The assumption of commercial activities by Tonix represents an important milestone in the evolution of Tonix as we continue to build and strengthen our commercial organization for the potential launch of Tonmya™ (cyclobenzaprine HCl sublingual tablets) for fibromyalgia in 2025, assuming approval by the U.S. Food and Drug Administration (FDA).”

About Zembrace^® SymTouch^® and Tosymra^®

Zembrace SymTouch is the only actively promoted brand of sumatriptan autoinjector in the United States (other sumatriptan autoinjector products on the market are Imitrex^® and generics to Imitrex^®). It has a unique low dose and has demonstrated onset of migraine pain relief in as few as 10 minutes (17% of patients vs. 5% for placebo).¹ Zembrace SymTouch also demonstrated migraine pain freedom for 46% of patients (vs 27% for placebo) at 2 hours in a single-attack, double-blind study (N=230).¹Zembrace SymTouch currently has patent protection to 2036. Tosymra employs Intravail^® permeation enhancer technology and is pharmacokinetically equivalent to 4 mg subcutaneous sumatriptan.² Tosymra delivers migraine pain relief in as little as 10 minutes with just one spray for some patients (13% vs. 5% for placebo).² Tosymra currently has patent protection to 2031.

About Migraine

Nearly 40 million people in the United States suffer from migraine³ and it has been recognized as the second leading cause of disability in the world.⁴ Migraine is characterized by debilitating attacks lasting four to 72 hours with multiple symptoms, including pulsating headaches of moderate to severe pain intensity often associated with nausea or vomiting, and/or sensitivity to sound (phonophobia) and sensitivity to light (photophobia).³

References:

Zembrace SymTouch [package insert]. Maple Grove, MN: Upsher-Smith Laboratories, LLC: February 2021.
Tosymra [package insert]. Maple Grove, MN: Upsher-Smith Laboratories, LLC: Feb 2021.
Headache Classification Committee of the International Headache Society (IHS). The international classification of headache disorders, 3rd edition. Cephalalgia. 2018;38(1):1–211.
GBD 2016 Headache Collaborators. Global, regional, and national burden of migraine and tension-type headache, 1990-2016: a systematic analysis for the Global Burden of Disease Study 2016. Lancet Neurol 2018;17(11):954-976.

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

Tonmya is a centrally acting, non-opioid, non-addictive, bedtime medication. The tablet is a patented sublingual formulation of cyclobenzaprine hydrochloride developed for the management of fibromyalgia. In December 2023, the company announced highly statistically significant and clinically meaningful topline results in RESILIENT, a second positive Phase 3 clinical trial of Tonmya for the management of fibromyalgia. In the study, Tonmya met its pre-specified primary endpoint, significantly reducing daily pain compared to placebo (p=0.00005) in participants with fibromyalgia. Statistically significant and clinically meaningful results were also seen in all key secondary endpoints related to improving sleep quality, reducing fatigue and improving overall fibromyalgia symptoms and function. RELIEF, the first positive Phase 3 trial of Tonmya in fibromyalgia, was completed in December 2020. It met its pre-specified primary endpoint of daily pain reduction compared to placebo (p=0.010) and showed activity in key secondary endpoints.

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

Tonix Pharmaceuticals Holding Corp.^*

*Tonix’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

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

Zembrace^® SymTouch^® (sumatriptan Injection): IMPORTANT SAFETY INFORMATION

Zembrace SymTouch (Zembrace) can cause serious side effects, including heart attack and other heart problems, which may lead to death. Stop use and get emergency help if you have any signs of a heart attack:

discomfort in the center of your chest that lasts for more than a few minutes or goes away and comes back
severe tightness, pain, pressure, or heaviness in your chest, throat, neck, or jaw
pain or discomfort in your arms, back, neck, jaw or stomach
shortness of breath with or without chest discomfort
breaking out in a cold sweat
nausea or vomiting
feeling lightheaded

Zembrace is not for people with risk factors for heart disease (high blood pressure or cholesterol, smoking, overweight, diabetes, family history of heart disease) unless a heart exam shows no problem.

Do not use Zembrace if you have:

history of heart problems
narrowing of blood vessels to your legs, arms, stomach, or kidney (peripheral vascular disease)
uncontrolled high blood pressure
hemiplegic or basilar migraines. If you are not sure if you have these, ask your provider.
had a stroke, transient ischemic attacks (TIAs), or problems with blood circulation
severe liver problems
taken any of the following medicines in the last 24 hours: almotriptan, eletriptan, frovatriptan, naratriptan, rizatriptan, ergotamines, dihydroergotamine.
are taking certain antidepressants, known as monoamine oxidase (MAO)-A inhibitors or it has been 2 weeks or less since you stopped taking a MAO-A inhibitor. Ask your provider for a list of these medicines if you are not sure.
an allergy to sumatriptan or any of the components of Zembrace

Tell your provider about all of your medical conditions and medicines you take, including vitamins and supplements.

Zembrace can cause dizziness, weakness, or drowsiness. If so, do not drive a car, use machinery, or do anything where you need to be alert.

Zembrace may cause serious side effects including:

changes in color or sensation in your fingers and toes
sudden or severe stomach pain, stomach pain after meals, weight loss, nausea or vomiting, constipation or diarrhea, bloody diarrhea, fever
cramping and pain in your legs or hips; feeling of heaviness or tightness in your leg muscles; burning or aching pain in your feet or toes while resting; numbness, tingling, or weakness in your legs; cold feeling or color changes in one or both legs or feet
increased blood pressure including a sudden severe increase even if you have no history of high blood pressure
medication overuse headaches from using migraine medicine for 10 or more days each month. If your headaches get worse, call your provider.
serotonin syndrome, a rare but serious problem that can happen in people using Zembrace, especially when used with anti-depressant medicines called SSRIs or SNRIs. Call your provider right away if you have: mental changes such as seeing things that are not there (hallucinations), agitation, or coma; fast heartbeat; changes in blood pressure; high body temperature; tight muscles; or trouble walking.
hives (itchy bumps); swelling of your tongue, mouth, or throat
seizures even in people who have never had seizures before

The most common side effects of Zembrace include: pain and redness at injection site; tingling or numbness in your fingers or toes; dizziness; warm, hot, burning feeling to your face (flushing); discomfort or stiffness in your neck; feeling weak, drowsy, or tired.

Tell your provider if you have any side effect that bothers you or does not go away. These are not all the possible side effects of Zembrace. For more information, ask your provider.

This is the most important information to know about Zembrace but is not comprehensive. For more information, talk to your provider and read the Patient Information and Instructions for Use. You can also visit www.tonixpharma.com or call 1-888-869-7633.

You are encouraged to report adverse effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

INDICATION AND USAGE

Zembrace is a prescription medicine used to treat acute migraine headaches with or without aura in adults who have been diagnosed with migraine.

Zembrace is not used to prevent migraines. It is not known if it is safe and effective in children under 18 years of age.

Tosymra^® (sumatriptan nasal spray): IMPORTANT SAFETY INFORMATION

Tosymra can cause serious side effects, including heart attack and other heart problems, which may lead to death. Stop Tosymra and get emergency medical help if you have any signs of heart attack:

discomfort in the center of your chest that lasts for more than a few minutes or goes away and comes back
severe tightness, pain, pressure, or heaviness in your chest, throat, neck, or jaw
pain or discomfort in your arms, back, neck, jaw, or stomach
shortness of breath with or without chest discomfort
breaking out in a cold sweat
nausea or vomiting
feeling lightheaded

Tosymra is not for people with risk factors for heart disease (high blood pressure or cholesterol, smoking, overweight, diabetes, family history of heart disease) unless a heart exam is done and shows no problem.

Do not use Tosymra if you have:

history of heart problems
narrowing of blood vessels to your legs, arms, stomach, or kidney (peripheral vascular disease)
uncontrolled high blood pressure
severe liver problems
hemiplegic or basilar migraines. If you are not sure if you have these, ask your healthcare provider.
had a stroke, transient ischemic attacks (TIAs), or problems with blood circulation
taken any of the following medicines in the last 24 hours: almotriptan, eletriptan, frovatriptan, naratriptan, rizatriptan, ergotamines, or dihydroergotamine. Ask your provider if you are not sure if your medicine is listed above.
are taking certain antidepressants, known as monoamine oxidase (MAO)-A inhibitors or it has been 2 weeks or less since you stopped taking a MAO-A inhibitor. Ask your provider for a list of these medicines if you are not sure.
an allergy to sumatriptan or any ingredient in Tosymra

Tell your provider about all of your medical conditions and medicines you take, including vitamins and supplements.

Tosymra can cause dizziness, weakness, or drowsiness. If so, do not drive a car, use machinery, or do anything where you need to be alert.

Tosymra may cause serious side effects including:

changes in color or sensation in your fingers and toes
sudden or severe stomach pain, stomach pain after meals, weight loss, nausea or vomiting, constipation or diarrhea, bloody diarrhea, fever
cramping and pain in your legs or hips, feeling of heaviness or tightness in your leg muscles, burning or aching pain in your feet or toes while resting, numbness, tingling, or weakness in your legs, cold feeling or color changes in one or both legs or feet
increased blood pressure including a sudden severe increase even if you have no history of high blood pressure
medication overuse headaches from using migraine medicine for 10 or more days each month. If your headaches get worse, call your provider.
serotonin syndrome, a rare but serious problem that can happen in people using Tosymra, especially when used with anti-depressant medicines called SSRIs or SNRIs. Call your provider right away if you have: mental changes such as seeing things that are not there (hallucinations), agitation, or coma; fast heartbeat; changes in blood pressure; high body temperature; tight muscles; or trouble walking.
hives (itchy bumps); swelling of your tongue, mouth, or throat
seizures even in people who have never had seizures before

The most common side effects of Tosymra include: tingling, dizziness, feeling warm or hot, burning feeling, feeling of heaviness, feeling of pressure, flushing, feeling of tightness, numbness, application site (nasal) reactions, abnormal taste, and throat irritation.

Tell your provider if you have any side effect that bothers you or does not go away. These are not all the possible side effects of Tosymra. For more information, ask your provider.

This is the most important information to know about Tosymra but is not comprehensive. For more information, talk to your provider and read the Patient Information and Instructions for Use. You can also visit www.tonixpharma.com or call 1-888-869-7633.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

INDICATION AND USAGE
Tosymra is a prescription medicine used to treat acute migraine headaches with or without aura in adults.

Tosymra is not used to treat other types of headaches such as hemiplegic or basilar migraines or cluster headaches.

Tosymra is not used to prevent migraines. It is not known if Tosymra is safe and effective in children under 18 years of age.

Source: Tonix Pharmaceuticals Holding Corp.

Released March 19, 2024

Release – Cocrystal Pharma Receives Pre-IND Responses from the FDA on Oral CC-42344 for Treating Influenza A

Posted on March 19, 2024March 19, 2024 by aweinsoff@channelchek.com

Research News and Market Data on COCP

MARCH 19, 2024

Feedback provides greater clarity on regulatory requirements for planned Phase 2b trial

BOTHELL, Wash., March 19, 2024 (GLOBE NEWSWIRE) — Cocrystal Pharma, Inc. (Nasdaq: COCP) (“Cocrystal” or the “Company”) announces it has received Pre-Investigational New Drug (Pre-IND) feedback from the U.S. Food and Drug Administration (FDA) regarding CC-42344 as a potential oral treatment for pandemic and seasonal influenza A. A Pre-IND review provides the opportunity to obtain FDA guidance and clarification on critical steps such as the proposed clinical trial design, as well as clinical drug manufacturing and nonclinical studies deemed necessary before filing the trial design. The feedback was provided in a written response to a Pre-IND package and questions submitted by the Company in January 2024.

“We value the FDA guidance as we prepare to file the IND for our Phase 2b trial and open enrollment of patients in the U.S.,” said Sam Lee, Ph.D., Cocrystal’s President and co-CEO. “This is an important milestone that provides greater clarity on the regulatory requirements and our planned oral CC-42344 clinical program.”

A Phase 2a challenge study of oral CC-42344 is underway in the United Kingdom to evaluate safety, and viral and clinical measures in healthy volunteers who are infected with the influenza A virus. The Company expects to report topline results from this study in the second half of this year.

In addition, preparations are underway to begin a Phase 1 study in Australia with the Company’s inhaled formulation of CC-42344 as a potential influenza A treatment and prophylaxis for those exposed to the virus. Recent preclinical data showed that inhaled CC-42344 exhibited highly effective delivery into the lung, superior lung exposure, efficacy in influenza-infected human lung epithelia, and a favorable safety profile.

CC-42344 Influenza A PB2 Inhibitor
CC-42344 is a novel, broad-spectrum, investigational antiviral candidate for the treatment of pandemic and seasonal influenza A. CC-42344 inhibits the first step in the viral replication process of influenza A by binding to a highly conserved PB2 site of the polymerase complex that is essential to replication. In vitro testing with CC-42344 showed excellent antiviral activity against influenza A strains, including pandemic and seasonal strains, as well as against strains resistant to Tamiflu^® and Xofluza^®, while also demonstrating favorable pharmacokinetic and safety profiles. In addition, oral CC-42344 demonstrated favorable safety and tolerability results in a Phase 1 study in Australia. This antiviral candidate was discovered using the Company’s proprietary structure-based drug discovery platform technology.

About Seasonal Influenza
Each year there are approximately 1 billion cases of seasonal influenza worldwide, with 3-5 million severe illnesses and up to 650,000 deaths, according to the World Health Organization. On average about 8% of the U.S. population contracts influenza each season. In addition to the health risk, influenza is responsible for approximately $10.4 billion in direct costs for hospitalizations and outpatient visits for adults in the U.S. annually.

About Cocrystal Pharma, Inc.
Cocrystal Pharma, Inc. is a clinical-stage biotechnology company discovering and developing novel antiviral therapeutics that target the replication process of influenza viruses, coronaviruses (including SARS-CoV-2), noroviruses and hepatitis C viruses. Cocrystal employs unique structure-based technologies and Nobel Prize-winning expertise to create first- and best-in-class antiviral drugs. For further information about Cocrystal, please visit www.cocrystalpharma.com.

Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding the expected results of the Phase 2a trial for CC-42344 for the oral treatment of influenza A in the second half of 2024, efforts in preparation for an anticipated Phase 2b trial for oral treatment of influenza A following the Phase 2a trial, efforts to begin a Phase 1 study in Australia for an inhaled formulation of CC-42344 as a potential influenza A treatment and prophylaxis, and the potential efficacy and clinical benefits of, and market for, such product candidates. The words “believe,” “may,” “estimate,” “continue,” “anticipate,” “intend,” “should,” “plan,” “could,” “target,” “potential,” “is likely,” “will,” “expect” and similar expressions, as they relate to us, are intended to identify forward-looking statements. We have based these forward-looking statements largely on our current expectations and projections about future events. Some or all of the events anticipated by these forward-looking statements may not occur. Important factors that could cause actual results to differ from those in the forward-looking statements include, but are not limited to, risks relating to our ability to proceed with the studies including recruiting volunteers and procuring materials for such studies by our clinical research organizations and vendors, the results of such studies and our ability to obtain FDA approval to initiate the Phase 2b study. Further information on our risk factors is contained in our filings with the SEC, including our Annual Report on Form 10-K for the year ended December 31, 2022. Any forward-looking statement made by us herein speaks only as of the date on which it is made. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to publicly update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by law.

Investor Contact:
LHA Investor Relations
Jody Cain
310-691-7100
jcain@lhai.com

Media Contact:
JQA Partners
Jules Abraham
917-885-7378
Jabraham@jqapartners.com

# # #

Source: Cocrystal Pharma, Inc.

Released March 19, 2024

AstraZeneca Makes $2.4 Billion Bet on Next-Gen Cancer Radioconjugates

Posted on March 19, 2024March 19, 2024 by slightbourne@noblecapitalmarkets.com

In a bold move to fortify its oncology pipeline, pharmaceutical giant AstraZeneca (NASDAQ: AZN) has agreed to acquire clinical-stage biotech Fusion Pharmaceuticals (NASDAQ: FUSN) for up to $2.4 billion. The deal gives AstraZeneca full access to Fusion’s pioneering radioconjugate (RC) therapies and expertise as it aims to transform cancer treatment and unseat traditional chemotherapy and radiation regimens.

Fusion specializes in developing a promising new class of precision oncology drugs called RCs, which dispense powerful, targeted radiation directly to cancer cells via targeting molecules like antibodies. By delivering potent radioisotope payloads to tumors in this manner, RCs may improve upon external beam radiation’s limitations and indiscriminately toxic effects.

Under the agreed terms, AstraZeneca will pay $21 per share in cash upfront to acquire all outstanding Fusion shares, valuing the biotech at approximately $2 billion. This headline price represents a staggering 97% premium over Fusion’s closing price prior to deal announcement. AstraZeneca has also committed up to $3 per share in additional contingent value rights tied to a future regulatory milestone, which could push the total deal value to $2.4 billion if achieved.

For AstraZeneca, the acquisition paves the way for a major expansion into promising RC therapeutics, which could revolutionize how cancers are treated in the future. The crown jewel of the deal is FPI-2265, Fusion’s lead RC candidate that uses the alpha particle-emitting isotope actinium-225 to target PSMA proteins in metastatic castration-resistant prostate cancer. FPI-2265 is already in Phase 2 clinical trials with early data expected in 2024.

Beyond this advanced asset, AstraZeneca gains control of Fusion’s broader pipeline of RC programs and candidates across multiple solid tumor types. Just as importantly, the transaction provides AstraZeneca with Fusion’s specialized R&D capabilities, manufacturing infrastructure, and actinium-225 supply chain specifically tailored for developing these next-wave radiotherapeutics.

This strategic acquisition doubles down on AstraZeneca’s burgeoning radio-isotope therapy initiatives. The pharma giant already has a collaboration with Fusion exploring lung cancer applications using one of its RC molecules. By bringing Fusion’s RC therapy capabilities fully in-house, AstraZeneca can now swiftly integrate and scale up this cutting-edge treatment modality across its industry-leading oncology portfolio.

For Fusion investors, the buyout represents a lucrative exit at a substantial premium, especially compared to the company’s $300 million market cap prior to deal reports. While always bittersweet to see a promising biotech get absorbed, Fusion’s technology and team are now set to be fueled by abundant AstraZeneca resources in pursuing RC breakthroughs.

The transaction, expected to close in Q2 2024 pending customary approvals, foreshadows a future where RCs could potentially supplant chemotherapy and radiotherapy as more precise, less toxic foundational cancer regimens. While still an emerging field, AstraZeneca’s bold multi-billion dollar investment signals its confidence in harnessing RCs’ unique advantages over traditional oncology treatments.

As AstraZeneca executes an ambitious pivot toward next-generation RC therapies, biotech and pharma investors would be wise to monitor this rapidly evolving space. The acquisition of Fusion continues to position the pharma titan at the vanguard of replacing archaic cancer protocols with targeted radioconjugate precision medicines.

WaveDancer’s Acquisition of AI Neuroscience Firm Firefly Poised to Shake Up Healthcare Tech

Posted on March 19, 2024March 19, 2024 by slightbourne@noblecapitalmarkets.com

The financial markets are abuzz over WaveDancer, Inc.’s approved merger deal to acquire Firefly Neuroscience, an artificial intelligence company pioneering brain health diagnostics and analytics software. With stockholders from both companies green-lighting the transaction, the stage is set for a new player to emerge in the rapidly evolving neurological healthcare technology space.

WaveDancer (NASDAQ: WAVD), currently an IT services provider to government and commercial clients, is essentially acquiring the capabilities and pipeline of private AI neuroscience firm Firefly through a merger of one of its subsidiaries into the latter. Once the deal closes in Q2 2024, the combined entity will rebrand as Firefly Neuroscience, Inc. and trade under the new ticker AIFF on the Nasdaq Capital Market.

The new Firefly Neuroscience will be laser-focused on commercializing and advancing the development of Firefly’s innovative Brain Network Analytics (BNA) software platform, cleared by the U.S. Food and Drug Administration. Leveraging AI, machine learning, and a massive database of over 17,000 EEG brain scans, the BNA Platform aims to revolutionize the diagnosis and treatment of mental health conditions like depression and anxiety, as well as neurological disorders such as dementia, concussions, and ADHD.

“The WaveDancer favorable shareholder vote is an important step in consummating the merger and reinventing WaveDancer as an AI-enabled neurological health platform,” stated Jamie Benoit, Chairman and CEO of WaveDancer. The company plans to divest its legacy IT services operations to fully concentrate on scaling up Firefly’s neuroscience technology business post-merger.

Firefly’s unique AI platform has already garnered significant attention and investment from the medical community, with the company raising approximately $60 million to date to build out its EEG database, develop the software, secure patents, and attain FDA clearance. Now poised to transition into a publicly-traded commercial entity, Firefly Neuroscience could rapidly accelerate adoption of its solutions among pharmaceutical companies, clinical trials, and medical practitioners globally.

The combined firm will hit the ground running, with Firefly management slated to present at the Noble Capital Markets Emerging Growth Virtual Healthcare Equity Conference on April 17-18, 2024. This high-profile investor forum will provide an ideal platform to lay out Firefly Neuroscience’s growth strategy and market opportunity to Wall Street.

The Emerging Growth Virtual Healthcare Conference will feature 2 days of corporate presentations from up to 50 innovative public healthcare, biotech, and medical device companies, showcasing their latest advancements and investment opportunities. Each presentation will be followed by a fireside-style…Read Mo re

While the technology is highly specialized, analysts forecast significant potential upside for Firefly’s brain mapping and analytics capabilities across a range of healthcare markets struggling with neurological and mental health challenges. The company’s AI edge in these areas could position it to drive improved patient outcomes, lower costs through earlier interventions, and open new avenues for drug development and therapies.

For WaveDancer shareholders, the transaction marks a bold pivot into a cutting-edge industry riding powerful tailwinds of AI adoption in healthcare settings. Assuming a seamless merger and transition, the company could quickly morph into a promising pure-play on validated, scalable neurotechnology solutions backed by substantial technical assets and IP.

As the deal approaches the finish line, all eyes will be on Firefly Neuroscience’s market debut and ability to execute on the immense growth prospects its AI brain analytics platform presents. Healthcare investors would be wise to keep a close watch as this under-the-radar neuroscience firm prepares to seize a starring role on Wall Street.

Release – ZyVersa Therapeutics Announces IRB Approval of Phase 2a Clinical Trial Protocol to Evaluate Cholesterol Efflux Mediator™ VAR 200 in Patients with Diabetic Kidney Disease

Posted on March 18, 2024March 18, 2024 by aweinsoff@channelchek.com

Research News and Market Data on ZVSA

Mar 18, 2024

PDF Version

Phase 2a trial is on track to begin in the first half of 2024.
Cholesterol Efflux Mediator^TM VAR 200 is in development to ameliorate renal lipid accumulation that damages the kidneys’ filtration system, leading to chronic kidney disease and its progression.

WESTON, Fla., March 18, 2024 (GLOBE NEWSWIRE) — ZyVersa Therapeutics, Inc. (Nasdaq: ZVSA, or “ZyVersa”), a clinical stage specialty biopharmaceutical company developing first-in-class drugs for the treatment of renal and inflammatory diseases with high unmet medical needs, announces Institutional Review Board (IRB) approval of the Phase 2a clinical trial protocol to evaluate the efficacy and safety of Cholesterol Efflux Mediator VAR 200 in patients with diabetic kidney disease. The clinical trial is on track to begin in the first half of 2024.

Initiation of this clinical trial is a key milestone for ZyVersa. It is the first in human trial for VAR 200 intended to substantiate that the promising preclinical results demonstrated in three different animal models of kidney disease (diabetic kidney disease, focal segmental glomerulosclerosis, and Alport syndrome) translate to patients with kidney disease. The preclinical data demonstrated across all three kidney disease models that VAR 200:

Reduced cholesterol and lipid levels in the kidneys’ filtration system.
Protected against kidney injury and fibrosis.
Significantly reduced protein in the urine (proteinuria).

For more information about VAR 200, Click Here.

“A large body of evidence reinforces the importance of addressing kidney accumulation of cholesterol and lipids to help attenuate progression of chronic kidney disease, which affects over 35 million adults in the United States,” commented Stephen C. Glover, ZyVersa’s Co-founder, Chairman, CEO, and President. “Currently, over 130,000 patients with kidney disease progress to renal failure each year, and more than 800,000 patients are living with renal failure requiring dialysis or transplant to sustain life. We are excited about the potential of Cholesterol Efflux Mediator^TM VAR 200 to protect against kidney injury and reduce kidney disease progression.”

About ZyVersa Therapeutics, Inc.

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

Cautionary Statement Regarding Forward-Looking Statements

Certain statements contained in this press release regarding matters that are not historical facts, are forward-looking statements within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995. These include statements regarding management’s intentions, plans, beliefs, expectations, or forecasts for the future, and, therefore, you are cautioned not to place undue reliance on them. No forward-looking statement can be guaranteed, and actual results may differ materially from those projected. ZyVersa Therapeutics, Inc (“ZyVersa”) uses words such as “anticipates,” “believes,” “plans,” “expects,” “projects,” “future,” “intends,” “may,” “will,” “should,” “could,” “estimates,” “predicts,” “potential,” “continue,” “guidance,” and similar expressions to identify these forward-looking statements that are intended to be covered by the safe-harbor provisions. Such forward-looking statements are based on ZyVersa’s expectations and involve risks and uncertainties; consequently, actual results may differ materially from those expressed or implied in the statements due to a number of factors, including ZyVersa’s plans to develop and commercialize its product candidates, the timing of initiation of ZyVersa’s planned preclinical and clinical trials; the timing of the availability of data from ZyVersa’s preclinical and clinical trials; the timing of any planned investigational new drug application or new drug application; ZyVersa’s plans to research, develop, and commercialize its current and future product candidates; the clinical utility, potential benefits and market acceptance of ZyVersa’s product candidates; ZyVersa’s commercialization, marketing and manufacturing capabilities and strategy; ZyVersa’s ability to protect its intellectual property position; and ZyVersa’s estimates regarding future revenue, expenses, capital requirements and need for additional financing.

New factors emerge from time-to-time, and it is not possible for ZyVersa to predict all such factors, nor can ZyVersa assess the impact of each such factor on the business or the extent to which any factor, or combination of factors, may cause actual results to differ materially from those contained in any forward-looking statements. Forward-looking statements included in this press release are based on information available to ZyVersa as of the date of this press release. ZyVersa disclaims any obligation to update such forward-looking statements to reflect events or circumstances after the date of this press release, except as required by applicable law.

This press release does not constitute an offer to sell, or the solicitation of an offer to buy, any securities.

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

Release – Ocugen, Inc. Appoints Huma Qamar, MD, MPH As Chief Medical Officer

Posted on March 18, 2024March 18, 2024 by aweinsoff@channelchek.com

Research News and Market Data on OCGN

March 18, 2024

MALVERN, Pa., March 18, 2024 (GLOBE NEWSWIRE) — Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies and vaccines, today announced the appointment of Dr. Huma Qamar as Chief Medical Officer (CMO).

“I’m very pleased to name Dr. Qamar as the CMO at Ocugen,” said Dr. Shankar Musunuri, Chairman, Chief Executive Officer, and Co-Founder of Ocugen. “This appointment recognizes her invaluable contributions to building a strong clinical team, overseeing a well-conducted Phase 1/2 gene therapy trial, and establishing relationships with leading medical and academic institutions and key opinion leaders.”

Dr. Qamar is a distinguished healthcare professional with a rich background in clinical research, having contributed her expertise to esteemed Ivy League institutions including Yale University, Harvard University, and University of Pennsylvania. Her expertise encompasses the development of Phase I-IV clinical protocols and execution of clinical studies, FDA inspections, and effective leadership of medical affairs teams.

“Having been a part of Ocugen’s mission and vision for more than three years now, it’s a pleasure to lead Ocugen’s clinical development at this pivotal time,” said Dr. Qamar. “I am committed to addressing patients’ unmet medical needs and bringing novel modifier gene therapy products to the market globally.”

Dr. Qamar has expertise in multiple therapeutic areas, including gene and cell therapy, vaccines, oncology (Heme-Onc, CAR-T, rare tumors, sarcoma, melanoma, women’s health, GU & GI, fetal oncology), rheumatology, dermatology, neurology, cardiology, hepatology, and infectious diseases.

“Dr. Qamar has a deep understanding of Ocugen’s ongoing clinical trials and a close working relationship with the clinical and regulatory teams, both of which are fundamentally important to the success of Ocugen’s programs,” said Dr. Prabhavathi Fernandes, Independent Lead Director, Ocugen board. “I’m confident that her determination and dedication will bring the Company’s first-in-class candidates through the upcoming clinical milestones to deliver on Ocugen’s long-term strategy.”

Prior to joining Ocugen, Dr. Qamar was Senior Vice President, Head of R&D at FSD Pharma—demonstrating her commitment to advancing scientific innovation and pharmaceutical development.

About Ocugen, Inc.

Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patients’ lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on X and LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, which are subject to risks and uncertainties. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should,” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks, and uncertainties that may cause actual events or results to differ materially from our current expectations. These and other risks and uncertainties are more fully described in our periodic filings with the Securities and Exchange Commission (SEC), including the risk factors described in the section entitled “Risk Factors” in the quarterly and annual reports that we file with the SEC. Any forward-looking statements that we make in this press release speak only as of the date of this press release. Except as required by law, we assume no obligation to update forward-looking statements contained in this press release whether as a result of new information, future events, or otherwise, after the date of this press release.

Contact:
Tiffany Hamilton
Head of Communications
Tiffany.Hamilton@ocugen.com 

Titan Medical Announces Transformative Merger with Conavi Medical

Posted on March 18, 2024March 18, 2024 by slightbourne@noblecapitalmarkets.com

Toronto-based medical device company Titan Medical Inc. (TSX: TMD) unveiled a definitive agreement to merge with Conavi Medical Inc. in an all-stock transaction that will create a new publicly-traded leader in hybrid intravascular imaging.

The deal, announced on March 18, 2024, will see Titan acquire all of the outstanding shares of Conavi, a commercial-stage firm that has developed the Novasight Hybrid System for guiding minimally invasive coronary procedures. In exchange, Conavi shareholders will receive newly issued shares of Titan.

The merger will constitute a reverse takeover of Titan by Conavi. Upon completion, the combined entity plans to change its name to Conavi Medical Inc. and apply to list its shares on the TSX Venture Exchange after delisting from the Toronto Stock Exchange.

“This planned merger comes at a pivotal moment as we advance the Novasight Hybrid System, unlocking its full potential in the U.S. and globally,” said Thomas Looby, CEO of Conavi. “Gaining public company status will enhance our financial strength and growth strategy.”

Conavi’s Novasight Hybrid system is the first to combine intravascular ultrasound (IVUS) and optical coherence tomography (OCT) imaging modalities, enabling simultaneous and co-registered imaging of coronary arteries. It has regulatory approvals in the U.S., Canada, China and Japan.

Paul Cataford, Interim CEO and Board Chair of Titan, said “Conavi is an exciting commercial-stage company with groundbreaking technology. We are confident in their ability to drive adoption of the Novasight Hybrid System.”

As part of the transaction, Conavi will complete a concurrent equity financing raising between $15-20 million before the deal closes around July 15th. The financing is expected to attract support from institutional investors.

Take a moment to take a look at Noble Capital Markets’ Senior Research Analyst Robert Leboyer’s coverage universe.

Key benefits anticipated for the combined company include a strong balance sheet following the financing, established product capabilities, a proven coronary imaging product being commercialized, a large market opportunity, and increasing user traction.

Under the agreement terms, Titan will consolidate its shares on an agreed ratio prior to the merger. A Titan subsidiary will then amalgamate with Conavi, with Titan issuing new consolidated shares to Conavi shareholders based on an exchange ratio valuing Conavi at $69.84 million pre-money. This ratio will ensure existing Titan shareholders own at least 10% of the combined company.

All officers and certain Titan directors will resign upon closing and be replaced by Conavi nominees. The merger requires approval from shareholders of both companies as well as regulatory approvals. Titan’s board unanimously recommends shareholders vote in favor based on a fairness opinion from its financial advisor.

The transaction marks the culmination of a strategic review process for Titan over the past 15 months. The company previously halted work on its surgical robotics program to conserve cash before pursuing asset sales and IP licensing deals.

With Conavi’s commercial hybrid imaging technology and anticipated financial resources from the merger and financing, the combined company aims to drive market penetration in the fast-growing field of intravascular imaging for coronary procedures. The deal transforms Titan from an R&D-stage firm into a revenue-generating medtech leader.

Geron Stock Skyrockets on Pivotal FDA Panel Backing for Blood Disorder Drug

Posted on March 18, 2024March 18, 2024 by slightbourne@noblecapitalmarkets.com

In a major vindication for Geron Corporation’s therapeutic pipeline, the biotech company’s shares skyrocketed nearly 90% in after-hours trading Thursday after receiving a critical green light from U.S. drug regulators.

In a 12-2 vote, the Food and Drug Administration’s Oncologic Drugs Advisory Committee (ODAC) ruled that the clinical benefits of Geron’s investigational drug imetelstat outweigh its risks for treating a serious blood disorder. Specifically, the independent panel of experts endorsed imetelstat as a potential new therapy for transfusion-dependent anemia in certain adult patients with low to intermediate-risk myelodysplastic syndromes.

Myelodysplastic syndromes (MDS) are a group of malignant bone marrow disorders that disrupt the production of healthy blood cells. In the lower-risk forms of MDS being targeted by imetelstat, anemia caused by a lack of red blood cells is one of the most problematic complications, often requiring regular blood transfusions.

Imetelstat, an innovative first-in-class drug candidate, works by inhibiting the enzyme telomerase. This mechanism of action allows imetelstat to potentially restore normal red blood cell production and alleviate the need for transfusions in these MDS patients.

In Geron’s pivotal phase 3 IMerge study involving over 200 patients, those treated with imetelstat showed significantly higher rates of achieving red blood cell transfusion independence for at least 8 weeks compared to placebo. The study also found 28% of imetelstat patients achieved transfusion independence for 24 weeks or longer – with a median duration of 80 weeks. Only 3% of those on placebo matched that level of durable response.

“There are few treatment options and significant unmet medical need remains for these patients, particularly among those with difficult-to-treat subtypes of this blood cancer,” said Faye Feller, Geron’s chief medical officer. “We believe that imetelstat has the potential to be an important new medicine.”

Wall Street clearly agrees with that assessment. Geron shares, which had already surged over 60% year-to-date in anticipation of Thursday’s advisory meeting, ripped nearly 90% higher in extended trading after the result was announced.

While not binding, the FDA typically follows the recommendations of its advisory panels when making final approval decisions. The agency has set a target action date of June 16 to decide on whether to greenlight imetelstat for MDS patients based on the totality of evidence – including the IMerge trial data and ODAC’s favorable risk-benefit evaluation.

Approval appears likely after the decisive ODAC vote, providing a tremendous boost for Geron as it pushes ahead with commercialization plans for imetelstat. In addition to seeking FDA approval, the company is pursuing European regulatory clearance after submitting its marketing application to health authorities there last year.

Analysts see imetelstat generating hundreds of millions in peak sales if approved for this initial MDS population with unmet needs. But Geron is also evaluating the drug’s potential utility in other blood and bone marrow disorders like myelofibrosis, significantly expanding imetelstat’s commercial opportunity.

Even after Thursday’s enormous stock spike, Geron remains modestly valued at around $1.5 billion in market capitalization. While certainties remain around pricing, reimbursement and ultimate market penetration, the positive ODAC outcome drastically improves the outlook for this longtime drugmaker to finally bring its first product to market after years of development setbacks.

For a company that has relied primarily on partnerships, licensing deals and equity raises to sustain its operations, having a wholly-owned product with multi-billion dollar sales potential could completely transform Geron’s outlook – validating the bold decision to double down on imetelstat’s high-risk, high-reward proposition in recent years.

While challenges still lie ahead, investors are clearly salivating over imetelstat’s bright future after the pivotal FDA panel vote removed a major roadblock on the path to potential commercialization. The overwhelmingly positive outcome sent an unmistakable signal that Geron may finally be nearing the lucrative promised land after getting mired in drug development purgatory for decades.