Dr. Christian Lunetta to Present “From PARADIGM to PARAGON: Advancing PrimeC for ALS through Phase 2 Clinical and Biomarker Insights toward a Global Phase 3 Trial”
CAMBRIDGE, Mass., March 18, 2026 /PRNewswire/ — NeuroSense Therapeutics Ltd. (NASDAQ: NRSN) (“NeuroSense”), a late-stage clinical biotechnology company focused on developing disease-modifying treatments for neurodegenerative diseases, today announced that Dr. Christian Lunetta will present new data and insights on the development of PrimeC, the company’s investigational therapy for amyotrophic lateral sclerosis (ALS), at the AD/PD™ 2026 International Conference on Alzheimer’s and Parkinson’s Diseases and Related Neurological Disorders, to be held in Copenhagen, Denmark, on March 19, 2026.
Dr. Lunetta’s presentation, titled “From PARADIGM to PARAGON: Advancing PrimeC for ALS through Phase 2 Clinical and Biomarker Insights toward a Global Phase 3 Trial,” will take place during the symposium “Mechanisms and Therapeutics in the ALS-FTD Spectrum (SOD-1, TDP-43, C9ORF72 and TMEM106B).”
The presentation will review key clinical and biomarker findings from the Phase 2b PARADIGM trial, which evaluated PrimeC in people living with ALS. The data provide important insights into disease mechanisms and treatment effects that helped inform the design of the company’s global Phase 3 PARAGON trial, currently being advanced to further evaluate PrimeC’s safety and efficacy.
“ALS is one of the most complex neurodegenerative diseases, and advancing therapeutic development requires the integration of rigorous clinical research with deeper biological insight,” said Dr. Christian Lunetta. “The clinical findings emerging from the PARADIGM trial, together with the expanding biomarker analyses, provide an important scientific foundation as we advance toward the PARAGON Phase 3 study. I look forward to sharing these results with the scientific community at AD/PD 2026 and to contributing, together with fellow investigators, to the next stage of clinical development as we work to advance meaningful therapeutic options for people living with ALS.”
“We are deeply appreciative of Dr. Lunetta’s role in presenting these findings and of his contribution as part of the clinical investigator community behind this work,” said Dr. Shiran Zimri, NeuroSense VP of Research and Development and Canada Country Lead. “The timing of this presentation, coming just days after the publication of the PARADIGM results in JAMA Neurology and at such a highly regarded scientific forum, is especially meaningful. It creates a unique moment where robust, peer-reviewed data can immediately be brought into scientific exchange and critical discussion. For us, this is not only about sharing results, but about engaging the field in a deeper understanding of the clinical and biomarker insights from PARADIGM as we advance with urgency toward our global Phase 3 PARAGON study.”
The PARAGON Phase 3 trial is planned as a multinational, randomized, double-blind, placebo-controlled study designed to further evaluate PrimeC’s potential to slow disease progression in people living with ALS.
NeuroSense continues active engagement with regulatory authorities to advance PrimeC toward potential marketing authorization.
About NeuroSense
NeuroSense Therapeutics is a clinical-stage biotechnology company focused on discovering and developing treatments for patients suffering from debilitating neurodegenerative diseases. NeuroSense believes that these diseases, which include amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and Parkinson’s disease, among others, represent one of the most significant unmet medical needs of our time, with limited effective therapeutic options available for patients to date. Due to the complexity of neurodegenerative diseases and based on strong scientific research on a large panel of related biomarkers, NeuroSense’s strategy is to develop combined therapies targeting multiple pathways associated with these diseases.
For additional information, we invite you to visit our website and follow us on LinkedIn, YouTube and X. Information that may be important to investors may be routinely posted on our website and these social media channels.
About PrimeC
PrimeC, NeuroSense’s lead drug candidate, is a novel extended-release oral formulation composed of a unique fixed-dose combination of two FDA-approved drugs: ciprofloxacin and celecoxib. PrimeC is designed to synergistically target several key mechanisms of ALS and AD, that contribute to neuron degeneration, inflammation, iron accumulation and impaired ribonucleic acid (“RNA”) regulation to potentially inhibit the progression of ALS and AD.
About ALS
Amyotrophic lateral sclerosis (“ALS”) is an incurable neurodegenerative disease that causes complete paralysis and death within 2-5 years from diagnosis. Every year, more than 5,000 people are diagnosed with ALS in the U.S. alone, with an annual disease burden of $1 billion. The number of people living with ALS is expected to grow by 24% by 2040 in the U.S. and EU.
Forward-Looking Statements
This press release contains “forward-looking statements” that are subject to substantial risks and uncertainties. All statements, other than statements of historical fact, contained in this press release are forward-looking statements. Forward-looking statements contained in this press release may be identified by the use of words such as “anticipate,” “believe,” “contemplate,” “could,” “estimate,” “expect,” “intend,” “seek,” “may,” “might,” “plan,” “potential,” “predict,” “project,” “target,” “aim,” “should,” “will” “would,” or the negative of these words or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on NeuroSense Therapeutics’ current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict and include statements regarding the timing of regulatory filings, meetings and regulatory decisions. Further, certain forward-looking statements, including statements regarding future development of PrimeC, are based on assumptions as to future events that may not prove to be accurate. The future events and trends may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward looking statements. These risks include the uncertainty regarding outcomes and the timing of current and future clinical trials; the risk the PrimeC will not advance towards later-stage development, timing for reporting data, including from the study of PrimeC in Alzheimer’s disease; that the study will not be successful; the ability of NeuroSense to remain listed on Nasdaq; and other risks and uncertainties set forth in NeuroSense’s filings with the Securities and Exchange Commission (SEC). You should not rely on these statements as representing our views in the future. More information about the risks and uncertainties affecting NeuroSense is contained under the heading “Risk Factors” in the Annual Report on Form 20-F filed with the Securities and Exchange Commission on April 7, 2025 and NeuroSense’s subsequent filings with the SEC. Forward-looking statements contained in this announcement are made as of this date, and NeuroSense undertakes no duty to update such information except as required under applicable law.
BERKELEY HEIGHTS, N.J., March 17, 2026 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (“Tonix” or the “Company”), a fully integrated, commercial biotechnology company, today announced an oral presentation and two poster presentations on its preclinical immuno-oncology portfolio at the American Association for Cancer Research (AACR) Annual Meeting 2026, being held April 17–22, 2026, in San Diego, California.
Oral Presentation Details
Title: TFF2 deficiency amplifies IL-1β-driven inflammation and promotes aging-associated gastric tumor progression Abstract #: 6822 Date and Time: April 21, 2026, 2:30–4:30 p.m. PT (5:30-7:30 p.m. ET) Session Category: Tumor Biology Session Title: Aging Micro- and Macro-Environments in Tumor Progression and Therapy Presenters: Shuang Li, MD, PhD, and Timothy C. Wang, MD, Herbert Irving Comprehensive Cancer Center, Columbia University Medical Center (Tonix co-authors: Seth Lederman, MD, Chief Executive Officer, and Bruce L. Daugherty, PhD, MBA, Executive Vice President of Research)
Poster Presentation Details
Title: In vitro characterization of fully human antagonistic anti-BTLA monoclonal antibodies Poster #: 6550 Date and Time: April 21, 2026, 2:00-5:00 p.m. PT (5:00-8:00 p.m. ET) Session Category: Clinical Research Session Title: Immune Checkpoint Blockade Location: Poster Section 44, Board 16 Presenter: Bruce Daugherty, PhD, MBA, Executive Vice President of Research, Tonix
Title: Pharmacokinetics of TNX-1700 in non-human primates and human FcRn/serum albumin transgenic mice Poster #: 7940 Date and Time: April 22, 2026, 9:00 a.m.–12:00 p.m. PT (12:00-3:00 p.m. ET) Session Category: Clinical Research Session Title: Tumor Microenvironment Modulators Location: Poster Section 49, Board 15 Presenter: Bruce Daugherty, PhD, MBA, Executive Vice President of Research, Tonix
About TNX-1700 TNX-1700, a fusion protein of TFF2 and albumin, is in preclinical development for the treatment of gastric and colorectal cancer in combination with PD-1 blockade. TNX-1700, in-licensed from Columbia University, is in the pre-Investigational New Drug (IND) stages of development.
Tonix Pharmaceuticals Holding Corp. Tonix Pharmaceuticals* is a fully-integrated, commercial-stage biotechnology company focused on central nervous system (CNS) and immunology treatments in areas of high unmet medical need. TONMYA® (cyclobenzaprine HCl sublingual tablets 2.8 mg), is the first new treatment for fibromyalgia in adults in more than 15 years. Tonix’s CNS commercial infrastructure supports its marketed products, including its acute migraine products, Zembrace® Symtouch® (sumatriptan injection 3 mg) and Tosymra® (sumatriptan nasal spray 10 mg). Tonix is investigating TONMYA in Phase 2 clinical trials to evaluate its potential in major depressive disorder and acute stress disorder. In addition, the company’s CNS portfolio includes TNX-2900, which is Phase 2 ready for the treatment of Prader-Willi syndrome, a rare disease. Tonix is also advancing a pipeline of immunology programs, including monoclonal antibody TNX-4800 for Lyme disease prophylaxis and TNX-1500, a third-generation CD40 ligand inhibitor for the prevention of kidney transplant rejection. To learn more, visit www.tonixpharma.com and follow the Company on LinkedIn and X.
*Tonix’s product development candidates are investigational new drugs or biologics; their efficacy and safety have not been established and have not been approved for any indication.
Zembrace SymTouch and Tosymra are registered trademarks of Tonix Medicines. TONMYA is a trademark of Tonix Pharma Limited. All other marks are property of their respective owners.
Forward Looking Statements Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995 including those relating to the completion of the offering, the satisfaction of customary closing conditions, the intended use of proceeds from the offering and other statements that are predictive in nature. 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 as a result of a number of factors, including the ability of the Company to satisfy the conditions to the closing of the offering and the timing thereof, as well as those described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025, as filed with the SEC on March 12, 2026, and periodic reports filed with the SEC on or after the date thereof. Tonix does not undertake an obligation to update or revise any forward-looking statement. 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.
The Journal of the American Medical Association (JAMA Neurology) publication highlights consistent clinical and biomarker findings from NeuroSense PARADIGM Phase 2b trial in ALS, including slower functional decline, reduced risk of ALS-related complications, and modulation of disease-relevant biomarkers
CAMBRIDGE, Mass., March 16, 2026 /PRNewswire/ — NeuroSense Therapeutics Ltd. (NASDAQ: NRSN) (“NeuroSense”), a late-stage clinical biotechnology company focused on developing disease-modifying treatments for neurodegenerative diseases, today announced that results from the Phase 2b clinical trial (PARADIGM) evaluating PrimeC in people living with amyotrophic lateral sclerosis (ALS) have been published in JAMA Neurology.
Earlier today, Mass General Brigham (MGB) and Barrow Neurological Institute (BNI) issued a joint announcement highlighting the publication and the contributions of the investigators involved in the PARADIGM trial. NeuroSense is honored to collaborate with these leading ALS research centers of excellence and their distinguished clinical teams in advancing research for people living with ALS.
PARADIGM was a randomized, double-blind, placebo-controlled trial that evaluated the safety, biological activity, and potential clinical effects of PrimeC during a 6-month blinded treatment period followed by a 12-month open-label extension.
PrimeC is a novel fixed-dose oral combination formulated in a synchronized extended-release composition designed to concurrently address three key mechanisms implicated in ALS progression: neuroinflammation, iron dysregulation, and microRNA-mediated regulatory pathways.
The publication was authored by a distinguished international group of ALS investigators, including Merit Cudkowicz, MD, MSc (Mass General Brigham and Harvard Medical School), Vivian Drory, MD (Tel-Aviv Sourasky Medical Centre), Adriano Chiò, MD, PhD (University of Torino), Christian Lunetta, MD (IRCCS Maugeri), Christen Shoesmith, MD (London Health Sciences Centre), Ruben van Eijk,MD, PhD (UMC Utrecht), Jeffrey Rosenfeld, MD, PhD (Loma Linda University), and Jeremy Shefner, MD, PhD (Barrow Neurological Institute), together with additional collaborators across North America, Europe, and Israel.
Key highlights from the PARADIGM phase 2b study published
Strong safety and tolerability profile: PrimeC demonstrated a safety profile comparable to placebo over 18 months, with most treatment-related adverse events mild to moderate and transient.
Clinically meaningful functional outcomes: Continuous treatment with PrimeC was associated with slower functional decline and improved long-term outcomes, including a 7.92-point advantage in ALSFRS-R at 18 months, which represents over 36% benefit of slowing disease progression (p=0.007) and significant improvement in bulbar function (P=0.001).
Reduction in risk of major ALS complications: Early initiation of PrimeC was associated with a 64% relative reduction in risk of ALS-related complications (p=0.02), including respiratory failure, hospitalization, or death.
Biological evidence supporting disease modification: PrimeC treatment was associated with significant modulation of ALS-related microRNAs and iron-regulatory biomarkers, supporting its biological activity and reinforcing the potential for disease-modifying effects.
Supports advancement to Phase 3: These findings support the continued clinical development of PrimeC and its advancement to a confirmatory Phase 3 clinical trial in ALS.
“ALS is one of the most serious neurological diseases, and there is an urgent need for therapies that can meaningfully alter its course,” said Merit Cudkowicz, MD, MSc, Director of the Healey & AMG Center for ALS at Mass General Brigham and Julieanne Dorn Professor of Neurology at Harvard Medical School. “What is particularly noteworthy about the PARADIGM results is the consistency of the findings across clinical outcomes and disease-relevant biomarkers, in addition to good safety. These results provide a strong scientific rationale for continuing the clinical development of PrimeC and support its evaluation in a larger confirmatory Phase 3 trial.”
“What stands out about the PARADIGM study is the multiple clinical endpoints suggest the same level of clinical benefit and that multiple biomarkers are consistent with clinical endpoints,” said Jeremy M. Shefner, MD, PhD, Professor of Neurology at the Barrow Neurological Institute and corresponding author of the publication. “Together, these findings provide a strong scientific foundation for advancing PrimeC into a Phase 3 trial designed to validate its impact for patients.”
“The publication of the PARADIGM results in the prestigious JAMA Neurology represents an important milestone for NeuroSense and for people living with ALS,” said Ferenc Tracik, MD, Chief Medical Officer of NeuroSense Therapeutics. “This article integrates PrimeC’s safety, clinical, and biomarker data over 18 months. The consistent findings of slower functional decline, reduced risk of ALS-related complications, and modulation of iron-regulatory and microRNA biomarkers strengthen confidence in PrimeC’s potential as a disease-modifying therapy. These findings directly informed the design of our Phase 3 trial, which has received FDA clearance to proceed.”
PARADIGM Study Overview
PARADIGM was a multinational Phase 2b randomized, double-blind, placebo-controlled clinical trial conducted at leading ALS referral centers in Italy, Canada, and Israel.
The study enrolled 68 participants with definite or probable ALS, randomized in a 2:1 ratio to receive PrimeC or placebo for six months. The double-blind phase was followed by a 12-month open-label extension, during which all participants received PrimeC while maintaining blinding to original treatment assignment.
Even though the trial was not powered to demonstrate definitive efficacy, participants who received PrimeC from the outset maintained a clinically meaningful 7.92-point functional advantage in ALSFRS-R at 18 months, which represents over 36% benefit of slowing disease progression (p=0.007), with the largest improvement observed in the bulbar domain (p=0.001), a key determinant of speech, swallowing, and quality of life in ALS.
In addition, ALS complication-free survival demonstrated a 64% relative risk reduction favoring early treatment (p=0.02), suggesting potential long-term clinical benefit.
Biomarker Findings
The trial also demonstrated biological activity consistent with PrimeC’s proposed multi-pathway mechanism.
Markers of iron metabolism, which are frequently dysregulated in ALS, showed favorable significant changes, including preservation of transferrin levels and stabilization of ferritin.
In parallel, plasma microRNA analysis revealed statistically significant downregulation of ALS-associated microRNAs miR-199a-3p, miR-199a-5p, miR-181a-5p, and miR-181b-5p, which have been linked to disease severity and prognosis.
Together, these findings provide biological evidence that PrimeC engages disease-relevant pathways and support its continued development as a potential disease-modifying therapy.
About PrimeC
PrimeC is an investigational fixed-dose oral combination therapy composed of celecoxib and ciprofloxacin in a synchronized extended-release formulation. The therapy is designed to simultaneously target multiple biological mechanisms implicated in ALS progression, including neuroinflammation, iron dysregulation, and microRNA dysregulation.
The therapeutic rationale for PrimeC is supported by preclinical evidence across ALS disease models and human induced pluripotent stem cell–derived motor neuron systems and has now been evaluated in a randomized Phase 2b clinical trial.
About NeuroSense Therapeutics
NeuroSense Therapeutics is a clinical-stage biopharmaceutical company focused on developing disease-modifying therapies for people living with ALS and other neurodegenerative diseases. The company’s lead program, PrimeC, is designed to simultaneously target multiple biological pathways implicated in ALS progression.
Forward-Looking Statements
This press release contains “forward-looking statements” that are subject to substantial risks and uncertainties. All statements, other than statements of historical fact, contained in this press release are forward-looking statements. Forward-looking statements contained in this press release may be identified by the use of words such as “anticipate,” “believe,” “contemplate,” “could,” “estimate,” “expect,” “intend,” “seek,” “may,” “might,” “plan,” “potential,” “predict,” “project,” “target,” “aim,” “should,” “will” “would,” or the negative of these words or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on NeuroSense Therapeutics’ current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict and include statements regarding the timing of regulatory filings, meetings and regulatory decisions. Further, certain forward-looking statements, including statements regarding future development of PrimeC, are based on assumptions as to future events that may not prove to be accurate. The future events and trends may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward looking statements. These risks include the uncertainty regarding outcomes and the timing of current and future clinical trials; the risk the PrimeC will not advance towards later-stage development, timing for reporting data, including from the study of PrimeC in Alzheimer’s disease; that the study will not be successful; the ability of NeuroSense to remain listed on Nasdaq; and other risks and uncertainties set forth in NeuroSense’s filings with the Securities and Exchange Commission (SEC). You should not rely on these statements as representing our views in the future. More information about the risks and uncertainties affecting NeuroSense is contained under the heading “Risk Factors” in the Annual Report on Form 20-F filed with the Securities and Exchange Commission on April 7, 2025 and NeuroSense’s subsequent filings with the SEC. Forward-looking statements contained in this announcement are made as of this date, and NeuroSense undertakes no duty to update such information except as required under applicable law.
– 12-patient cohort fully enrolled with an average time since transplant of approximately 8 months
– 100% insulin independence achieved in 10 patients who are over 4 weeks post-transplant
– No signs of graft rejection or de novo donor-specific HLA antibodies
– Tegoprubart continues to demonstrate a favorable safety and tolerability profile
IRVINE, Calif, March 16, 2026 (GLOBE NEWSWIRE) — Eledon Pharmaceuticals, Inc. (“Eledon”) (NASDAQ: ELDN) today announced updated results from an investigator-initiated trial conducted at the University of Chicago Medicine Transplant Institute and presented by Piotr Witkowski, M.D., Ph.D., Director of the Pancreas and Islet Transplant Program at UChicago Medicine, at the Advanced Technologies and Treatments for Diabetes (ATTD) conference, held March 11-14, 2026 in Barcelona, Spain.
The investigator-initiated pilot study enrolled 12 adults with long-standing type 1 diabetes undergoing allogeneic islet transplantation at UChicago Medicine. Patients had a median duration of diabetes of approximately 33 years and mean hemoglobin A1C (“HbA1C”) of approximately 8.0% prior to transplantation. Participants received tegoprubart, Eledon’s anti-CD40L monoclonal antibody, as part of a calcineurin inhibitor-free immunosuppression regimen.
The data demonstrated rapid improvement in glycemic control following islet transplantation, with stable islet graft function observed across the cohort. All 10 patients who were more than four weeks post-transplant achieved both insulin independence and a most recent HbA1c below 6.0%, with a mean most recent HbA1c across the 10 patients of approximately 5.35%. Tegoprubart-based immunosuppression was generally well tolerated with reported post-transplant immunosuppression-related adverse events successfully treated by lowering the mycophenolic acid dose, if necessary. There were no rejection episodes, and no patients developed de novo donor-specific HLA antibodies. Additionally, no evidence of nephrotoxicity, hypertension or neurotoxicity, which are commonly associated with tacrolimus-based immunosuppression regimens, was observed. These findings further support the potential of CD40L blockade to enable effective islet graft protection while avoiding the toxicities of calcineurin inhibitors.
“T1D patients have been waiting decades for a potential functional cure, and it is very encouraging to see meaningful progress in that direction through the emerging promise of tegoprubart,” said David-Alexandre C. Gros, M.D., Chief Executive Officer of Eledon. “These latest findings support the potential of tegoprubart to enable effective islet graft protection while avoiding the toxicities often associated with calcineurin inhibitors, and potentially enable access to islet cell transplantation for individuals living with T1D. We are proud to contribute to these important ongoing research efforts and support the work of Dr. Witkowski and the team at UChicago Medicine. We also look forward to working closely with the FDA towards our goal of receiving regulatory guidance on a path to market for tegoprubart in islet cell transplantation later this year.”
“Breakthrough T1D is proud to fund the University of Chicago’s clinical trial testing tegoprubart as a novel immunosuppression alternative for use in islet cell transplants and we are very encouraged by the early data,” said Aaron Kowalski, Ph.D., CEO of Breakthrough T1D. “It is exciting to see islet transplant recipients in this trial who no longer need to administer insulin and who are experiencing fewer side effects than with traditional immunosuppressive regimens.”
This UChicago Medicine-initiated clinical trial is funded by Breakthrough T1D, with initial support from The Cure Alliance. Breakthrough T1D has also committed to fund a second study evaluating tegoprubart as part of a calcineurin inhibitor-free immunosuppression drug regimen to prevent islet transplant rejection in individuals with T1D and chronic kidney disease.
About Islet Transplantation for Type 1 Diabetes
Pancreatic islet transplantation is a minimally invasive procedure developed to provide blood glucose control for subjects with type 1 diabetes and minimize or eliminate dependence on insulin. During the procedure, pancreatic islets containing insulin-producing beta cells are isolated from the pancreas of a deceased organ donor and infused through a small catheter into the patient’s liver. The islet cells lodge in small blood vessels in the liver and release insulin. After the procedure, subjects remain on immunosuppression therapy to prevent transplant rejection.
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, islet cell transplantation, liver allograft transplantation 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 planned clinical trials, the development of product candidates, expected or future results of tegoprubart trials and its ability to prevent rejection in connection with islet cell transplantation, 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 sites, as well as patient enrollment; and risks relating to costs of clinical trials and the sufficiency of the company’s capital resources to fund planned clinical trials. 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.
Peer-Reviewed Article Describes Clinical and Immunologic Rationale for Dual-Antigen MVA-Based Vaccine Designed to Address Limitations of First-Generation COVID-19 Vaccines in Highly Vulnerable Populations
ATLANTA, GA – March 12, 2026 – GeoVax Labs, Inc. (Nasdaq: GOVX), a clinical-stage biotechnology company developing vaccines and immunotherapies for infectious diseases and solid tumors, today announced the publication of a peer-reviewed article describing its next-generation COVID-19 vaccine candidate, GEO-CM04S1, in Medical Research Archives, the journal of the European Society of Medicine.
The article, titled “GEO-CM04S1: A Dual-Antigen COVID-19 Vaccine for Immunocompromised Patients,” provides a comprehensive review of the vaccine’s scientific rationale, preclinical studies, and clinical findings supporting its development as a vaccine designed specifically to protect immunocompromised individuals who often respond poorly to currently authorized COVID-19 vaccines.
The publication highlights how GEO-CM04S1’s dual-antigen design (Spike + Nucleocapsid) delivered via a Modified Vaccinia Ankara (MVA) viral vector is intended to generate antibody and T-cell responses that are both broad and durable, addressing limitations in such vulnerable populations of single-antigen vaccines that primarily target the spike protein.
The publication discusses how next-generation vaccines designed to stimulate more robust and durable cellular immunity may offer improved protection for these high-risk populations.
Scientific Highlights from the Publication
Key findings summarized in the publication include:
1. Dual-Antigen Design to Enhance Immune Breadth: GEO-CM04S1 expresses both the spike (S) and nucleocapsid (N) proteins of SARS-CoV-2, allowing the vaccine to stimulate immune responses against conserved viral targets that are less susceptible to mutation and immune escape.
2. Robust T-Cell Responses: Preclinical and clinical data show the vaccine induces strong CD4+ and CD8+ T-cell responses, which are critical for controlling viral infection and reducing progression to severe disease.
3. Favorable Safety and Immunogenicity: Early clinical studies demonstrated a benign safety profile and strong immunologic responses, including seroconversion and cellular immune activation across multiple dose levels.
4. Encouraging Results in Immunocompromised Patients: Early readouts from ongoing Phase 2 clinical trials in patients with hematologic malignancies receiving cell transplants, and individuals with chronic lymphocytic leukemia, indicate the vaccine can generate durable immune responses even in patients with impaired immune systems.
David Dodd, Chairman and Chief Executive Officer of GeoVax, stated: “This publication reinforces the scientific rationale for GEO-CM04S1 as a purpose-built vaccine for immunocompromised populations that remain inadequately protected by current COVID-19 vaccines. An estimated 40+ million patients in the U.S. are considered immunocompromised, including patients with cancer, transplant recipients, individuals receiving immunosuppressive therapies, and those with chronic diseases. These individuals may fail to mount adequate immune responses following vaccination and remain at higher risk of severe COVID-19 outcomes. Worldwide, an estimated 400 million patients have such weakened immune systems, rendering them at risk of severe infection, hospitalization and potential death.”
Mark J. Newman, PhD, Chief Scientific Officer of GeoVax and co-author of the publication, added: “A growing body of evidence demonstrates that strong and early T-cell responses play a critical role in controlling SARS-CoV-2 infection and preventing severe disease. GEO-CM04S1 was designed specifically to stimulate these responses, which may be particularly important for immunocompromised individuals who often fail to generate adequate antibody responses to existing vaccines. The MVA vector platform provides an ideal backbone for next-generation vaccines due to its ability to safely induce durable humoral and cellular immunity. Our dual-antigen strategy also expands immune recognition beyond the spike protein, and data from small animal studies indicates efficacy against variants is induced, reducing the need to continually update vaccines.”
About GEO-CM04S1
GEO-CM04S1 is a dual-antigen Modified Vaccinia Ankara (MVA)-vectored COVID-19 vaccine designed to induce durable T-cell and antibody responses against SARS-CoV-2.
The vaccine is currently being evaluated in multiple Phase 2 clinical trials, including:
Primary vaccination in immunocompromised individuals
Booster vaccination in patients with chronic lymphocytic leukemia (CLL)
The vaccine’s multi-antigen design and viral vector platform are intended to provide broader, more durable immune protection and improved efficacy in populations where first-generation vaccines have demonstrated reduced effectiveness.
About GeoVax
GeoVax Labs, Inc. is a clinical-stage biotechnology company focused on the development of vaccines and immunotherapies addressing high-consequence infectious diseases and solid tumor cancers. GeoVax’s priority program is GEO-MVA, a Modified Vaccinia Ankara (MVA)–based vaccine targeting mpox and smallpox. The program is advancing under an expedited regulatory pathway, with plans to initiate a pivotal Phase 3 clinical trial in the second half of 2026, to address critical global needs for expanded orthopoxvirus vaccine supply and biodefense preparedness. In oncology, GeoVax is developing Gedeptin®, a gene-directed enzyme prodrug therapy (GDEPT) designed to enhance immune checkpoint inhibitor activity. Gedeptin has completed a multicenter Phase 1/2 clinical trial in advanced head and neck cancer and is being advanced into combination strategies, including planned neoadjuvant and first-line settings. GeoVax’s broader pipeline includes the development of GEO-CM04S1, a next-generation COVID-19 vaccine candidate being evaluated in immunocompromised and other patient populations. GeoVax maintains a global intellectual property portfolio supporting its infectious disease and oncology programs and continues to evaluate strategic partnerships and funding opportunities aligned with its development priorities. For more information, visit www.geovax.com.
Forward-Looking Statements
This release contains forward-looking statements regarding GeoVax’s business plans. The words “believe,” “look forward to,” “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 and financial trends that we believe may affect our financial condition, results of operations, business strategy and financial needs. Actual results may differ materially from those included in these statements due to a variety of factors, including whether: GeoVax is able to obtain acceptable results from ongoing or future clinical trials of its investigational products, GeoVax’s immuno-oncology products and preventative vaccines can provoke the desired responses, and those products or vaccines can be used effectively, GeoVax’s viral vector technology adequately amplifies immune responses to cancer antigens, GeoVax can develop and manufacture its immuno-oncology products and preventative vaccines with the desired characteristics in a timely manner, GeoVax’s immuno-oncology products and preventative vaccines will be safe for human use, GeoVax’s vaccines will effectively prevent targeted infections in humans, GeoVax’s immuno-oncology products and preventative vaccines will receive regulatory approvals necessary to be licensed and marketed, GeoVax raises required capital to complete development, there is development of competitive products that may be more effective or easier to use than GeoVax’s products, GeoVax will be able to enter into favorable manufacturing and distribution agreements, and other factors, over which GeoVax has no control.
Further information on our risk factors is contained in our periodic reports on Form 10-Q and Form 10-K that we have filed and will file with the SEC. 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.
Program Reaches Strategic Inflection Point as GEO-MVA Advances Toward Pivotal Phase 3 Study
Atlanta, GA – March 11, 2026 – GeoVax Labs, Inc. (Nasdaq: GOVX), a clinical-stage biotechnology company developing vaccines and immunotherapies against infectious diseases and cancer, today announced that it has begun engaging with global health and preparedness organizations to explore potential future procurement of its GEO-MVA vaccine candidate for mpox and smallpox preparedness programs.
GeoVax has initiated discussions and is soliciting interest from international organizations that influence or directly procure mpox/smallpox vaccines. These organizations play key roles in recommending or directly procuring vaccines for national preparedness stockpiles and international outbreak response programs.
The outreach reflects growing global recognition of the need to diversify supply of Modified Vaccinia Ankara (MVA) vaccines used for protection against mpox and smallpox. Currently, global supply of MVA vaccines is concentrated in a single commercial manufacturer.
Global preparedness programs for smallpox and mpox vaccines represent a potential multi-billion-dollar procurement market supported by national stockpiles, military preparedness programs, and international health organizations. Governments have invested billions of dollars in medical countermeasure stockpiles over the past two decades, and demand for MVA-based vaccines is expected to further expand as mpox continues to emerge as a recurring global health threat.
GeoVax believes that initiating engagement with procurement and preparedness organizations represents an important transition from development toward commercialization planning, reflecting growing confidence in the GEO-MVA program and the potential for the vaccine to contribute to global preparedness stockpiles following successful completion of the planned Phase 3 study, scheduled to initiate in the second half of 2026.
Advancing GEO-MVA Through an Expedited Regulatory Pathway
GeoVax’s GEO-MVA program has progressed through an extensive regulatory dialogue with the European Medicines Agency (EMA), culminating in scientific advice in support of an expedited development pathway based on a single immunobridging trial to the licensed MVA vaccine.
“The EMA carefully evaluated the scientific evidence over an extended period before confirming an expedited pathway. We believe their guidance validates both the scientific foundation of GEO-MVA and the growing recognition that expanding the global MVA vaccine supply is an important public health priority,” said David Dodd, Chairman and Chief Executive Officer of GeoVax.
“We have also been encouraged by the positive response we are receiving from global health organizations and preparedness agencies as we initiated these discussions,” Dodd continued. “We view this engagement as an endorsement of the progress we have made, our commitment to advancing GEO-MVA, and the potential of GEO-MVA to help address the current worldwide need for expanded MVA vaccine supply. Importantly, it also represents a critical step toward commercialization of GEO-MVA and the potential generation of meaningful revenues through government and global health procurement programs as the program advances through its final development stages.”
Implications for Global Preparedness and Biodefense Readiness
The discussions initiated by GeoVax occur amid growing policy dialogue regarding the strategic importance of expanding global MVA vaccine manufacturing capacity. GeoVax believes GEO-MVA has the potential to become a strategically important medical countermeasure supporting both global public health preparedness and national biodefense initiatives.
Recent mpox outbreaks have reinforced the understanding that mpox is not a single episodic event, but rather an evolving infectious disease threat with potential for continued geographic expansion and recurrence. As a result, governments and international health organizations are increasingly emphasizing the need for manufacturing diversification and supply redundancy for vaccines used in outbreak response and biodefense preparedness.
In the United States, policymakers and defense stakeholders have increasingly recognized that no domestic manufacturing capability currently exists for MVA vaccines, a gap with implications for both civilian preparedness and military readiness.
“Establishing additional MVA manufacturing capability is increasingly viewed as an important component of global preparedness,” said Dodd. “We are seeing growing interest from public health organizations, governments, and defense stakeholders who recognize the strategic importance of supply diversification.”
About GEO-MVA
GEO-MVA is GeoVax’s candidate vaccine for protection against mpox and smallpox based on the Modified Vaccinia Ankara (MVA) platform. Pending successful completion of the planned immunobridging trial, GEO-MVA could represent an important additional source of MVA vaccine supply for global preparedness, outbreak response, and biodefense programs.
About GeoVax
GeoVax Labs, Inc. is a clinical-stage biotechnology company focused on the development of vaccines and immunotherapies addressing high-consequence infectious diseases and solid tumor cancers. GeoVax’s priority program is GEO-MVA, a Modified Vaccinia Ankara (MVA)–based vaccine targeting mpox and smallpox. The program is advancing under an expedited regulatory pathway, with plans to initiate a pivotal Phase 3 clinical trial in the second half of 2026, to address critical global needs for expanded orthopoxvirus vaccine supply and biodefense preparedness. In oncology, GeoVax is developing Gedeptin®, a gene-directed enzyme prodrug therapy (GDEPT) designed to enhance immune checkpoint inhibitor activity. Gedeptin has completed a multicenter Phase 1/2 clinical trial in advanced head and neck cancer and is being advanced into combination strategies, including planned neoadjuvant and first-line settings. GeoVax’s broader pipeline includes the development of GEO-CM04S1, a next-generation COVID-19 vaccine candidate being evaluated in immunocompromised and other patient populations. GeoVax maintains a global intellectual property portfolio supporting its infectious disease and oncology programs and continues to evaluate strategic partnerships and funding opportunities aligned with its development priorities. For more information, visit www.geovax.com.
Forward-Looking Statements
This release contains forward-looking statements regarding GeoVax’s business plans. The words “believe,” “look forward to,” “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 and financial trends that we believe may affect our financial condition, results of operations, business strategy and financial needs. Actual results may differ materially from those included in these statements due to a variety of factors, including whether: GeoVax is able to obtain acceptable results from ongoing or future clinical trials of its investigational products, GeoVax’s immuno-oncology products and preventative vaccines can provoke the desired responses, and those products or vaccines can be used effectively, GeoVax’s viral vector technology adequately amplifies immune responses to cancer antigens, GeoVax can develop and manufacture its immuno-oncology products and preventative vaccines with the desired characteristics in a timely manner, GeoVax’s immuno-oncology products and preventative vaccines will be safe for human use, GeoVax’s vaccines will effectively prevent targeted infections in humans, GeoVax’s immuno-oncology products and preventative vaccines will receive regulatory approvals necessary to be licensed and marketed, GeoVax raises required capital to complete development, there is development of competitive products that may be more effective or easier to use than GeoVax’s products, GeoVax will be able to enter into favorable manufacturing and distribution agreements, and other factors, over which GeoVax has no control.
Further information on our risk factors is contained in our periodic reports on Form 10-Q and Form 10-K that we have filed and will file with the SEC. 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.
Company launched TONMYA, approved by the FDA as a treatment for fibromyalgia, in November 2025
In post hoc analysis of the pivotal RESILIENT study, TONMYA produced rapid pain relief as early as Day 2 of treatment, with durable pain reduction and significant improvements in all key secondary endpoints as compared to placebo
In pooled post hoc analysis of the pivotal RELIEF and RELISIENT studies, TONMYA showed favorable benefit-risk profile using number needed to treat, number needed to harm, and likelihood to be helped or harmed
BERKELEY HEIGHTS, N.J., March 10, 2026 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (“Tonix” or the “Company”), a fully integrated, commercial biotechnology company, announced two oral presentations on TONMYATM, which was investigated as TNX-102 SL (cyclobenzaprine HCl sublingual tablets) at the 8th International Congress on Controversies in Fibromyalgia held on March 9-10, 2026, in Krakow, Poland.
“Phase 3 post hoc analyses reinforce the potential of TONMYA to provide a benefit to the approximately 10 million adults in the U.S. living with fibromyalgia,” said Gregory Sullivan, M.D., Chief Medical Officer of Tonix Pharmaceuticals. “In a post hoc analysis of RESILIENT, the data show rapid and early onset of pain relief. In a post hoc analysis of two pivotal studies, TONMYA showed a favorable benefit-risk profile that suggests treatment benefit is nearly four times more likely than discontinuation of treatment due to an adverse event. Together, these findings underscore TONMYA’s profile as a differentiated, generally well tolerated, and effective medicine that may address the unmet medical needs of those with fibromyalgia. TONMYA is the first medication approved for fibromyalgia in over 15 years.”
Oral Presentation One: “Cyclobenzaprine HCl Sublingual Tablets (CBP SL) Provide Rapid Pain Relief in Adults with Fibromyalgia”
In the RESILIENT trial, a 14-week, randomized, placebo-controlled Phase 3 study evaluating 457 adults with fibromyalgia as defined by 2016 American College of Rheumatology (ACR) criteria, a post hoc mixed-model repeated-measures analysis demonstrated that TONMYA produced a rapid reduction in pain, with improvements versus placebo observed as early as Day 2 of treatment and statistically significant pain relief at each week over Weeks 1–14. The primary endpoint, change from baseline to Week 14 in weekly average daily numeric rating scale (NRS) pain scores, was met with high statistical significance (p<0.001), with a least-squares mean treatment difference of -0.65. All key secondary endpoints were also statistically significant in favor of TONMYA.
TONMYA was generally well tolerated, with 6.1% of participants discontinuing due to adverse events versus 3.5% with placebo. The most common treatment-emergent adverse events were oral cavity reactions, including oral hypoesthesia (23.8%) and abnormal product taste (11.7%), which were typically mild, transient, and self-limited.
Oral Presentation Two: “Cyclobenzaprine HCl Sublingual Tablets for the Treatment of Fibromyalgia: Number Needed to Treat and Number Needed to Harm”
The data from a pooled post hoc analysis of 959 participants (783 completed the studies) from the RELIEF and RESILIENT Phase 3 trials was utilized to further clarify the benefit-risk profile of TONMYA using number needed to treat (NNT), number needed to harm (NNH), and likelihood to be helped or harmed (LHH). The NNT for achieving a clinically meaningful ≥30% pain reduction over placebo at Week 14 was 7 (95% confidence interval (CI): 5–12) while the NNH for discontinuation due to an adverse event was 26 (95% CI: 14–110). Based on these values, the LHH was 3.7, indicating that TONMYA provides a nearly four-fold greater likelihood of clinical benefit than adverse event-related discontinuation.
The pooled safety data were consistent with the known profile of TONMYA, with no new or unexpected safety signals. The most common treatment-emergent adverse events were oral cavity reactions that were typically mild, transient, and self-limited.
Copies of the Company’s presentations are available under the Scientific Presentations tab on the Tonix website at www.tonixpharma.com.
About Fibromyalgia
Fibromyalgia is a chronic pain disorder that is understood to result from amplified sensory and pain signaling within the central nervous system. Fibromyalgia afflicts an estimated 6-12 million adults in the U.S., approximately 90% of whom are women. Symptoms of fibromyalgia include chronic widespread pain, nonrestorative sleep, fatigue, and morning stiffness. Other associated symptoms include cognitive dysfunction and mood disturbances, including anxiety and depression. Individuals suffering from fibromyalgia struggle with their daily activities, have impaired quality of life, and frequently are disabled. Physicians and patients report common dissatisfaction with currently marketed products.
About TONMYA™ (cyclobenzaprine HCl sublingual tablets)
TONMYA (cyclobenzaprine HCl sublingual tablets) is a patented sublingual tablet formulation of cyclobenzaprine hydrochloride which provides rapid transmucosal absorption and reduced production of a long half-life active metabolite, norcyclobenzaprine, due to bypass of first-pass hepatic metabolism. As a multifunctional agent with potent binding and antagonist activities at the 5-HT2A serotonergic, α1-adrenergic, H1-histaminergic, and M1-muscarinic receptors, TONMYA was approved on August 15, 2025, by the FDA for the treatment of fibromyalgia in adults. TONMYA is the first new prescription medicine approved for fibromyalgia in more than 15 years. TONMYA was investigated as TNX-102 SL. TNX-102 SL is also being developed to treat acute stress reaction (ASR)/acute stress disorder (ASD), and major depressive disorder (MDD). The United States Patent and Trademark Office (USPTO) issued United States Patent No. 9636408 in May 2017, Patent No. 9956188 in May 2018, Patent No. 10117936 in November 2018, Patent No. 10,357,465 in July 2019, and Patent No. 10736859 in August 2020. The Protectic™ protective eutectic and Angstro-Technology™ formulation claimed in the patent are important elements of Tonix’s proprietary TONMYA composition. These patents are expected to provide TONMYA with U.S. market exclusivity until 2034/2035.
Tonix Pharmaceuticals Holding Corp.*
Tonix Pharmaceuticals* is a fully-integrated, commercial-stage biotechnology company focused on central nervous system (CNS) and immunology treatments in areas of high unmet medical need. TONMYATM (cyclobenzaprine HCl sublingual tablets 2.8mg), the Company’s recently approved flagship medicine, is the first new treatment for fibromyalgia in more than 15 years. Tonix’s CNS commercial infrastructure supports its marketed products, including its acute migraine products, Zembrace® SymTouch® and Tosymra®. Tonix is maximizing the science behind TONMYA in Phase 2 clinical trials to evaluate its potential in major depressive disorder and acute stress disorder. In addition, the company’s CNS portfolio includes TNX-2900, which is Phase 2 ready for the treatment of Prader-Willi syndrome, a rare disease. Tonix is also advancing a pipeline of immunology programs, including monoclonal antibody TNX-4800 for Lyme disease prophylaxis and TNX-1500, a third-generation CD40 ligand inhibitor for the prevention of kidney transplant rejection. To learn more, visit www.tonixpharma.com and follow the Company on LinkedIn and X.
* Tonix’s product development candidates are investigational new drugs or biologics; their efficacy and safety have not been established and have not been approved for any indication.
Zembrace SymTouch and Tosymra are registered trademarks of Tonix Medicines.
Forward Looking Statements
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995 including those relating to the completion of the offering, the satisfaction of customary closing conditions, the intended use of proceeds from the offering and other statements that are predictive in nature. 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 as a result of a number of factors, including the ability of the Company to satisfy the conditions to the closing of the offering and the timing thereof, as well as those described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2024, as filed with the SEC on March 18, 2025, and periodic reports filed with the SEC on or after the date thereof. Tonix does not undertake an obligation to update or revise any forward-looking statement. All of Tonix’s forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date thereof.
TONMYA is indicated for the treatment of fibromyalgia in adults.
CONTRAINDICATIONS
TONMYA is contraindicated: In patients with hypersensitivity to cyclobenzaprine or any inactive ingredient in TONMYA. Hypersensitivity reactions may manifest as an anaphylactic reaction, urticaria, facial and/or tongue swelling, or pruritus. Discontinue TONMYA if a hypersensitivity reaction is suspected. With concomitant use of monoamine oxidase (MAO) inhibitors or within 14 days after discontinuation of an MAO inhibitor. Hyperpyretic crisis seizures and deaths have occurred in patients who received cyclobenzaprine (or structurally similar tricyclic antidepressants) concomitantly with MAO inhibitors drugs. During the acute recovery phase of myocardial infarction, and in patients with arrhythmias, heart block or conduction disturbances, or congestive heart failure. In patients with hyperthyroidism.
WARNINGS AND PRECAUTIONS
Embryofetal toxicity: Based on animal data, TONMYA may cause neural tube defects when used two weeks prior to conception and during the first trimester of pregnancy. Advise females of reproductive potential of the potential risk and to use effective contraception during treatment and for two weeks after the final dose. Perform a pregnancy test prior to initiation of treatment with TONMYA to exclude use of TONMYA during the first trimester of pregnancy.
Serotonin syndrome: Concomitant use of TONMYA with selective serotonin reuptake inhibitors (SSRIs), serotonin norepinephrine reuptake inhibitors (SNRIs), tricyclic antidepressants, tramadol, bupropion, meperidine, verapamil, or MAO inhibitors increases the risk of serotonin syndrome, a potentially life-threatening condition. Serotonin syndrome symptoms may include mental status changes, autonomic instability, neuromuscular abnormalities, and/or gastrointestinal symptoms. Treatment with TONMYA and any concomitant serotonergic agent should be discontinued immediately if serotonin syndrome symptoms occur and supportive symptomatic treatment should be initiated. If concomitant treatment with TONMYA and other serotonergic drugs is clinically warranted, careful observation is advised, particularly during treatment initiation or dosage increases.
Tricyclic antidepressant-like adverse reactions: Cyclobenzaprine is structurally related to TCAs. TCAs have been reported to produce arrhythmias, sinus tachycardia, prolongation of the conduction time leading to myocardial infarction and stroke. If clinically significant central nervous system (CNS) symptoms develop, consider discontinuation of TONMYA. Caution should be used when TCAs are given to patients with a history of seizure disorder, because TCAs may lower the seizure threshold. Patients with a history of seizures should be monitored during TCA use to identify recurrence of seizures or an increase in the frequency of seizures.
Atropine-like effects: Use with caution in patients with a history of urinary retention, angle-closure glaucoma, increased intraocular pressure, and in patients taking anticholinergic drugs.
CNS depression and risk of operating a motor vehicle or hazardous machinery: TONMYA monotherapy may cause CNS depression. Concomitant use of TONMYA with alcohol, barbiturates, or other CNS depressants may increase the risk of CNS depression. Advise patients not to operate a motor vehicle or dangerous machinery until they are reasonably certain that TONMYA therapy will not adversely affect their ability to engage in such activities. Oral mucosal adverse reactions: In clinical studies with TONMYA, oral mucosal adverse reactions occurred more frequently in patients treated with TONMYA compared to placebo. Advise patients to moisten the mouth with sips of water before administration of TONMYA to reduce the risk of oral sensory changes (hypoesthesia). Consider discontinuation of TONMYA if severe reactions occur.
ADVERSE REACTIONS
The most common adverse reactions (incidence ≥2% and at a higher incidence in TONMYA-treated patients compared to placebo-treated patients) were oral hypoesthesia, oral discomfort, abnormal product taste, somnolence, oral paresthesia, oral pain, fatigue, dry mouth, and aphthous ulcer.
DRUG INTERACTIONS
MAO inhibitors: Life-threatening interactions may occur. Other serotonergic drugs: Serotonin syndrome has been reported. CNS depressants: CNS depressant effects of alcohol, barbiturates, and other CNS depressants may be enhanced. Tramadol: Seizure risk may be enhanced. Guanethidine or other similar acting drugs: The antihypertensive action of these drugs may be blocked.
USE IN SPECIFIC POPULATIONS
Pregnancy: Based on animal data, TONMYA may cause fetal harm when administered to a pregnant woman. The limited amount of available observational data on oral cyclobenzaprine use in pregnancy is of insufficient quality to inform a TONMYA-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. Advise pregnant women about the potential risk to the fetus with maternal exposure to TONMYA and to avoid use of TONMYA two weeks prior to conception and through the first trimester of pregnancy. Report pregnancies to the Tonix Medicines, Inc., adverse-event reporting line at 1-888-869-7633 (1-888-TNXPMED). Lactation: A small number of published cases report the transfer of cyclobenzaprine into human milk in low amounts, but these data cannot be confirmed. There are no data on the effects of cyclobenzaprine on a breastfed infant, or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for TONMYA and any potential adverse effects on the breastfed child from TONMYA or from the underlying maternal condition. Pediatric use: The safety and effectiveness of TONMYA have not been established. Geriatric patients: Of the total number of TONMYA-treated patients in the clinical trials in adult patients with fibromyalgia, none were 65 years of age and older. Clinical trials of TONMYA did not include sufficient numbers of patients 65 years of age and older to determine whether they respond differently from younger adult patients. Hepatic impairment: The recommended dosage of TONMYA in patients with mild hepatic impairment (HI) (Child Pugh A) is 2.8 mg once daily at bedtime, lower than the recommended dosage in patients with normal hepatic function. The use of TONMYA is not recommended in patients with moderate HI (Child Pugh B) or severe HI (Child Pugh C). Cyclobenzaprine exposure (AUC) was increased in patients with mild HI and moderate HI compared to subjects with normal hepatic function, which may increase the risk of TONMYA-associated adverse reactions.
Please see additional safety information in the full Prescribing Information.
To report suspected adverse reactions, contact Tonix Medicines, Inc. at 1-888-869-7633, or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
IRVINE, Calif., March 10, 2026 (GLOBE NEWSWIRE) — Eledon Pharmaceuticals, Inc. (“Eledon”) (NASDAQ: ELDN) today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to tegoprubart for the prevention of allograft rejection in liver transplantation. Tegoprubart has previously received orphan drug designation from the FDA for the prevention of allograft rejection in pancreatic islet cell transplantation and for the treatment of amyotrophic lateral sclerosis (ALS).
“Clinical studies in kidney transplantation have demonstrated that tegoprubart has the potential to improve graft survival and function while reducing the side effects associated with calcineurin inhibitors, supporting its promise as a novel immunosuppressive therapy across multiple organ transplant settings,” said David-Alexandre C. Gros, MD, Chief Executive Officer of Eledon. “Based on the encouraging preclinical evidence we have generated to date, we believe liver transplantation represents a significant incremental opportunity for tegoprubart, and we look forward to evaluating its potential in the clinical setting through an anticipated investigator sponsored trial initiating later this year.”
Orphan Drug Designation is intended to support the development of therapies for rare diseases, defined as conditions affecting fewer than 200,000 people in the United States or fewer than 5 in 10,000 individuals in the European Union. These designations provide sponsors with a range of incentives intended to encourage the development of medicines for diseases with high unmet medical needs.
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, islet cell transplantation, liver transplantation 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 planned clinical trials, the development of product candidates, expected or future results of tegoprubart trials and its ability to prevent rejection in connection with liver transplantation, 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 sites, as well as patient enrollment; and risks relating to costs of clinical trials and the sufficiency of the company’s capital resources to fund planned clinical trials. 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.
Follow Eledon Pharmaceuticals on social media: LinkedIn; Twitter
TONMYA (cyclobenzaprine HCl sublingual tablets) for the treatment of fibromyalgia in adults, was commercially launched in November 2025
Treatment with TONMYA provided statistically significant pain reduction in two Phase 3 trials and was generally well tolerated
BERKELEY HEIGHTS, N.J., March 09, 2026 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (“Tonix” or the “Company”), a fully integrated, commercial biotechnology company, presented data on TONMYATM (cyclobenzaprine HCl sublingual tablets), at the 2026 American Academy of Pain Medicine (AAPM) PainConnect Annual Meeting, in Salt Lake City, Utah.
“The data presented at the AAPM PainConnect Annual Meeting support TONMYA’s role as a safe and effective non-opioid analgesic for daily use at bedtime in fibromyalgia,1,2” said Gregory Sullivan, M.D., Chief Medical Officer of Tonix Pharmaceuticals. “TONMYA is designed to target nonrestorative sleep and is a first-in-class tertiary amine tricyclic for long-term use. As the first approved therapy for fibromyalgia in over 15 years, TONMYA is an alternative to three currently FDA-approved medicines, which are limited by side effects, adherence, and high discontinuation rates. TONMYA’s unique sublingual formulation enables cyclobenzaprine to bypass first-pass hepatic metabolism. Relative to off-label oral swallowed cyclobenzaprine, TONMYA treatment results in reduced formation of the active, persistent metabolite norcyclobenzaprine, which we believe interferes with the durability of cyclobenzaprine’s treatment effect in fibromyalgia with long term dosing. Oral cyclobenzaprine failed in development because it provided only short-term (one month) benefit3, which is not sufficient for the treatment of fibromyalgia, a chronic condition that requires a sustained treatment effect.”
Data presented at the AAPM PainConnect Annual Meeting are from the Phase 3 RESILIENT trial, a 14-week randomized, double-blind, placebo-controlled study that assessed the safety and efficacy of TONMYA in 457 patients who met the 2016 American College of Rheumatology (ACR) diagnostic criteria for fibromyalgia. In addition to showing a statistically significant reduction in mean daily pain at 14 weeks, TONMYA treatment resulted in an increase over placebo in the number of individuals with a 30% reduction in daily pain, which is considered a clinically meaningful response. The most common adverse events were mild and self-limited oral cavity reactions that uncommonly led to study withdrawal. TONMYA was approved by the FDA in August 2025 in part based on the Phase 3 RESILIENT trial results and was commercially launched in the U.S. in November 2025.
A copy of the Company’s poster presentation, “Treatment with TNX-102 SL Produces Clinically Meaningful Improvements in Patient-Centered Outcomes in Fibromyalgia,” is available under the Presentations tab of the Tonix website at https://ir.tonixpharma.com/presentations.
Citations
1Carette S, et al. Arthritis Rheum. 1994. 37(1):32-40. doi: 10.1002/art.1780370106. 2Lederman S, et al. Arthritis Care Res (Hoboken). 2023. 75(11):2359-2368. doi: 10.1002/acr.25142. 3Lederman S, et al. Pain Med. 2026. 27(1):86-94. doi: 10.1093/pm/pnaf089.
About Fibromyalgia
Fibromyalgia is a chronic pain disorder that is understood to result from amplified sensory and pain signaling within the central nervous system. Fibromyalgia afflicts an estimated 6-12 million adults in the U.S., approximately 90% of whom are women. Symptoms of fibromyalgia include chronic widespread pain, nonrestorative sleep, fatigue, and morning stiffness. Other associated symptoms include cognitive dysfunction and mood disturbances, including anxiety and depression. Individuals suffering from fibromyalgia struggle with their daily activities, have impaired quality of life, and frequently are disabled. Physicians and patients report common dissatisfaction with currently marketed products.
About TONMYA™ (cyclobenzaprine HCl sublingual tablets)
TONMYA (cyclobenzaprine HCl sublingual tablets) is a patented sublingual tablet formulation of cyclobenzaprine hydrochloride which provides rapid transmucosal absorption and reduced production of a long half-life active metabolite, norcyclobenzaprine, due to bypass of first-pass hepatic metabolism. As a multifunctional agent with potent binding and antagonist activities at the 5-HT2A serotonergic, α1-adrenergic, H1-histaminergic, and M1-muscarinic receptors, TONMYA was approved on August 15, 2025, by the FDA for the treatment of fibromyalgia in adults. TONMYA is the first new prescription medicine approved for fibromyalgia in more than 15 years. TONMYA was investigated as TNX-102 SL. TNX-102 SL is also being developed to treat acute stress reaction (ASR)/acute stress disorder (ASD), and major depressive disorder (MDD). The United States Patent and Trademark Office (USPTO) issued United States Patent No. 9636408 in May 2017, Patent No. 9956188 in May 2018, Patent No. 10117936 in November 2018, Patent No. 10,357,465 in July 2019, and Patent No. 10736859 in August 2020. The Protectic™ protective eutectic and Angstro-Technology™ formulation claimed in the patent are important elements of Tonix’s proprietary TONMYA composition. These patents are expected to provide TONMYA with U.S. market exclusivity until 2034/2035.
Tonix Pharmaceuticals Holding Corp.
Tonix Pharmaceuticals* is a fully-integrated, commercial-stage biotechnology company focused on central nervous system (CNS) and immunology treatments in areas of high unmet medical need. TONMYATM (cyclobenzaprine HCl sublingual tablets 2.8 mg), the Company’s recently approved flagship medicine, is the first new treatment for fibromyalgia in more than 15 years. Tonix’s CNS commercial infrastructure supports its marketed products, including its acute migraine products, Zembrace® SymTouch® and Tosymra®. Tonix is maximizing the science behind TONMYA in Phase 2 clinical trials to evaluate its potential in major depressive disorder and acute stress disorder. In addition, the company’s CNS portfolio includes TNX-2900, which is Phase 2 ready for the treatment of Prader-Willi syndrome, a rare disease. Tonix is also advancing a pipeline of immunology programs, including monoclonal antibody TNX-4800 for Lyme disease prophylaxis and TNX-1500, a third-generation CD40 ligand inhibitor for the prevention of kidney transplant rejection. To learn more, visit www.tonixpharma.com and follow the Company on LinkedIn and X.
*Tonix’s product development candidates are investigational new drugs or biologics; their efficacy and safety have not been established and have not been approved for any indication.
Tonmya, Zembrace SymTouch and Tosymra are registered trademarks of Tonix Medicines.
Forward Looking Statements
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995 including those relating to the completion of the offering, the satisfaction of customary closing conditions, the intended use of proceeds from the offering and other statements that are predictive in nature. 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 as a result of a number of factors, including the ability of the Company to satisfy the conditions to the closing of the offering and the timing thereof, as well as those described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2024, as filed with the SEC on March 18, 2025, and periodic reports filed with the SEC on or after the date thereof. Tonix does not undertake an obligation to update or revise any forward-looking statement. All of Tonix’s forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date thereof.
TONMYA is indicated for the treatment of fibromyalgia in adults.
CONTRAINDICATIONS
TONMYA is contraindicated: In patients with hypersensitivity to cyclobenzaprine or any inactive ingredient in TONMYA. Hypersensitivity reactions may manifest as an anaphylactic reaction, urticaria, facial and/or tongue swelling, or pruritus. Discontinue TONMYA if a hypersensitivity reaction is suspected. With concomitant use of monoamine oxidase (MAO) inhibitors or within 14 days after discontinuation of an MAO inhibitor. Hyperpyretic crisis seizures and deaths have occurred in patients who received cyclobenzaprine (or structurally similar tricyclic antidepressants) concomitantly with MAO inhibitors drugs. During the acute recovery phase of myocardial infarction, and in patients with arrhythmias, heart block or conduction disturbances, or congestive heart failure. In patients with hyperthyroidism.
WARNINGS AND PRECAUTIONS
Embryofetal toxicity: Based on animal data, TONMYA may cause neural tube defects when used two weeks prior to conception and during the first trimester of pregnancy. Advise females of reproductive potential of the potential risk and to use effective contraception during treatment and for two weeks after the final dose. Perform a pregnancy test prior to initiation of treatment with TONMYA to exclude use of TONMYA during the first trimester of pregnancy.
Serotonin syndrome: Concomitant use of TONMYA with selective serotonin reuptake inhibitors (SSRIs), serotonin norepinephrine reuptake inhibitors (SNRIs), tricyclic antidepressants, tramadol, bupropion, meperidine, verapamil, or MAO inhibitors increases the risk of serotonin syndrome, a potentially life-threatening condition. Serotonin syndrome symptoms may include mental status changes, autonomic instability, neuromuscular abnormalities, and/or gastrointestinal symptoms. Treatment with TONMYA and any concomitant serotonergic agent should be discontinued immediately if serotonin syndrome symptoms occur and supportive symptomatic treatment should be initiated. If concomitant treatment with TONMYA and other serotonergic drugs is clinically warranted, careful observation is advised, particularly during treatment initiation or dosage increases.
Tricyclic antidepressant-like adverse reactions: Cyclobenzaprine is structurally related to TCAs. TCAs have been reported to produce arrhythmias, sinus tachycardia, prolongation of the conduction time leading to myocardial infarction and stroke. If clinically significant central nervous system (CNS) symptoms develop, consider discontinuation of TONMYA. Caution should be used when TCAs are given to patients with a history of seizure disorder, because TCAs may lower the seizure threshold. Patients with a history of seizures should be monitored during TCA use to identify recurrence of seizures or an increase in the frequency of seizures.
Atropine-like effects: Use with caution in patients with a history of urinary retention, angle-closure glaucoma, increased intraocular pressure, and in patients taking anticholinergic drugs.
CNS depression and risk of operating a motor vehicle or hazardous machinery: TONMYA monotherapy may cause CNS depression. Concomitant use of TONMYA with alcohol, barbiturates, or other CNS depressants may increase the risk of CNS depression. Advise patients not to operate a motor vehicle or dangerous machinery until they are reasonably certain that TONMYA therapy will not adversely affect their ability to engage in such activities. Oral mucosal adverse reactions: In clinical studies with TONMYA, oral mucosal adverse reactions occurred more frequently in patients treated with TONMYA compared to placebo. Advise patients to moisten the mouth with sips of water before administration of TONMYA to reduce the risk of oral sensory changes (hypoesthesia). Consider discontinuation of TONMYA if severe reactions occur.
ADVERSE REACTIONS
The most common adverse reactions (incidence ≥2% and at a higher incidence in TONMYA-treated patients compared to placebo-treated patients) were oral hypoesthesia, oral discomfort, abnormal product taste, somnolence, oral paresthesia, oral pain, fatigue, dry mouth, and aphthous ulcer.
DRUG INTERACTIONS
MAO inhibitors: Life-threatening interactions may occur. Other serotonergic drugs: Serotonin syndrome has been reported. CNS depressants: CNS depressant effects of alcohol, barbiturates, and other CNS depressants may be enhanced. Tramadol: Seizure risk may be enhanced. Guanethidine or other similar acting drugs: The antihypertensive action of these drugs may be blocked.
USE IN SPECIFIC POPULATIONS
Pregnancy: Based on animal data, TONMYA may cause fetal harm when administered to a pregnant woman. The limited amount of available observational data on oral cyclobenzaprine use in pregnancy is of insufficient quality to inform a TONMYA-associated risk of major birth defects, miscarriage, or adverse maternal or fetal outcomes. Advise pregnant women about the potential risk to the fetus with maternal exposure to TONMYA and to avoid use of TONMYA two weeks prior to conception and through the first trimester of pregnancy. Report pregnancies to the Tonix Medicines, Inc., adverse-event reporting line at 1-888-869-7633 (1-888-TNXPMED). Lactation: A small number of published cases report the transfer of cyclobenzaprine into human milk in low amounts, but these data cannot be confirmed. There are no data on the effects of cyclobenzaprine on a breastfed infant, or the effects on milk production. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for TONMYA and any potential adverse effects on the breastfed child from TONMYA or from the underlying maternal condition. Pediatric use: The safety and effectiveness of TONMYA have not been established. Geriatric patients: Of the total number of TONMYA-treated patients in the clinical trials in adult patients with fibromyalgia, none were 65 years of age and older. Clinical trials of TONMYA did not include sufficient numbers of patients 65 years of age and older to determine whether they respond differently from younger adult patients. Hepatic impairment: The recommended dosage of TONMYA in patients with mild hepatic impairment (HI) (Child Pugh A) is 2.8 mg once daily at bedtime, lower than the recommended dosage in patients with normal hepatic function. The use of TONMYA is not recommended in patients with moderate HI (Child Pugh B) or severe HI (Child Pugh C). Cyclobenzaprine exposure (AUC) was increased in patients with mild HI and moderate HI compared to subjects with normal hepatic function, which may increase the risk of TONMYA-associated adverse reactions.
Please see additional safety information in the full Prescribing Information.
To report suspected adverse reactions, contact Tonix Medicines, Inc. at 1-888-869-7633, or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
CDI-988 is a direct-acting, oral antiviral being developed for norovirus
Norovirus challenge study is underway at Emory University School of Medicine to evaluate efficacy and safety of CDI-988
No approved treatments or vaccines are available for norovirus treatment and prevention, posing a significant unmet need and contributing to a global economic burden of $60 billion annually
BOTHELL, Wash., March 09, 2026 (GLOBE NEWSWIRE) — Cocrystal Pharma, Inc. (Nasdaq: COCP) (“Cocrystal” or the “Company”) announces the first subjects have been dosed in a Phase 1b norovirus challenge study (NCT07198139) to evaluate CDI-988 as both a preventive and treatment for norovirus infections. This cohort is to assess the infectivity rate of the challenge inoculum, GII.2 (Snow Mountain Virus). CDI-988 is a direct-acting, oral antiviral designed to inhibit a highly conserved region of the viral 3CL protease present in all known norovirus strains, including GII.2, GII.4 and recently re-emerging GII.17 variants. It is the first oral antiviral drug candidate developed for norovirus acute gastroenteritis.
“Commencement of this study is a significant milestone for Cocrystal and a critical step toward addressing a serious global unmet medical need, given the debilitating symptoms and high societal cost of norovirus outbreaks,” said Sam Lee, Ph.D., President and co‑CEO of Cocrystal Pharma. “CDI-988 has particular potential in high‑risk environments such as hospitals, nursing homes, cruise ships, schools and military facilities. The human challenge model is designed to provide proof‑of‑concept for our compound in a tightly controlled setting.”
The Phase 1b randomized, double-blind, placebo-controlled study is being conducted at Emory University School of Medicine and will enroll up to 40 healthy subjects ages 18–49. All participants will be infected with the norovirus GII.2 (Snow Mountain Virus) strain.
The first cohort will evaluate the infectivity rate of the challenge inoculum, GII.2 norovirus
Subsequent cohorts will be orally administered CDI-988 or placebo
The primary endpoint is efficacy versus placebo in reducing the incidence of clinical symptoms
Secondary endpoints include reduction of viral shedding and disease severity, and safety and pharmacokinetic profiles
“This challenge study is the first clinical trial involving a direct-acting antiviral specifically targeting norovirus infections. The efficacy and safety data from this study are expected to provide a strong rationale for further clinical advancement of CDI-988, and validate our proprietary structure-based drug discovery platform technology,” added Dr. Lee. “We would like to thank the volunteers for the norovirus challenge study and staff from Emory University School of Medicine who are currently participating in the study.”
CDI-988 previously demonstrated favorable safety and tolerability in a Phase 1 study across all dose levels, including the highest dose of 1200 mg being administered in the Phase 1b human challenge study. In September 2025 Cocrystal received a Study May Proceed Letter from the FDA and in December 2025 received Institutional Review Board approval from Emory University School of Medicine.
Cocrystal Pharma’s Structure-Based Drug Discovery Platform Technology
Cocrystal’s proprietary structural biology, along with its expertise in enzymology and medicinal chemistry, enable its development of novel antiviral agents. The Company’s platform provides a three-dimensional structure of inhibitor complexes at near-atomic resolution, providing immediate insight to guide Structure Activity Relationships. This helps identify novel binding sites and enables a rapid turnaround of structural information through highly automated X-ray data processing and refinement. The goal of this technology is to facilitate the development of novel broad-spectrum antivirals for the treatment of acute, chronic and potentially pandemic viral diseases.
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 to create viable antiviral drugs. For further information about Cocrystal, please visit www.cocrystalpharma.com.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding our norovirus study, the potential of CDI-988 for treatment and prevention of norovirus infections, and expectations that the outcome of the study will provide proof-of-concept and validation for further clinical advancement of our CDI-988 product candidate. 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, the risks and uncertainties arising from inflation, affordability, the possibility of a recession, the impact of future interest rate changes on the economy, uncertainty surrounding and impacts arising from tariffs and litigation and developments relating thereto, and geopolitical conflicts including those in the Middle East and Ukraine on our Company, our collaboration partners, and on the U.S. and global economies, including manufacturing and research delays arising from raw materials and labor shortages, supply chain disruptions and other business interruptions including any adverse impacts on our ability to obtain raw materials for and otherwise proceed with the norovirus study or subsequent studies as well as similar problems with our vendors and our current and any future clinical research organizations (CROs) and contract manufacturing organizations (CMOs), the progress and results of the studies including any adverse findings or delays, the ability of us and our CROs to recruit volunteers for, and to otherwise proceed with, clinical studies, our and our collaboration partners’ technology and software performing as expected, financial difficulties experienced by certain partners, the results of any current and future preclinical and clinical studies, general risks arising from clinical studies, receipt of regulatory approvals, regulatory changes and any adverse developments which may arise therefrom, potential mutations in a virus we are targeting that may result in variants that are resistant to a product candidate we develop, the potential for the development of effective treatments by competitors which could reduce or eliminate a prospective future market share commercializing any product candidates we may develop in the future, and our ability to meet our future liquidity needs. Further information on our risk factors is contained in our filings with the SEC, including the “Risk Factors” in Item 1A of our Annual Report on Form 10-K for the year ended December 31, 2024 and the Prospectus dated September 25, 2025. 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.
Financing included participation by healthcare-dedicated investors alongside existing shareholders
CHICAGO, IL, March 04, 2026 (GLOBE NEWSWIRE) — MAIA Biotechnology, Inc., (NYSE American: MAIA) (“MAIA”, the “Company”), a clinical-stage biopharmaceutical company developing targeted immunotherapies for cancer, today announced the closing of its previously announced underwritten public offering of 20,000,000 shares of its common stock at a public offering price of $1.50 per share for aggregate gross proceeds of $30 million, prior to deducting underwriting discounts and other offering expenses. In addition, the Company has granted the underwriters a 30-day option to purchase up to an additional 3,000,000 shares of common stock at the public offering price per share, less the underwriting discounts to cover over-allotments, if any.
The offering was structured as a straightforward common stock only investment with no warrant coverage and was led by healthcare-dedicated investors alongside existing shareholders.
Konik Capital Partners, LLC, a division of T.R. Winston & Company acted as the sole book-running manager for the offering.
MAIA intends to use the net proceeds from the offering to conduct clinical trials and for working capital and general corporate purposes.
The securities described above were offered and sold pursuant to a “shelf” registration statement on Form S-3 (File No. 333-273984), including a base prospectus, filed with the U.S. Securities and Exchange Commission (the “SEC”) on August 15, 2023, and declared effective on August 23, 2023.
The offering was made only by means of a prospectus supplement and accompanying prospectus that form a part of the registration statement. A prospectus supplement describing the terms of the public offering has been filed with the SEC and forms a part of the effective registration statement.
Copies of the prospectus supplement and the accompanying prospectus relating to this offering may be obtained, on the SEC’s website at http://www.sec.gov or by contacting Konik Capital Partners LLC, a division of T.R. Winston & Company, at 7 World Trade Center, 46th Floor, New York, NY 10007, Attention: Capital Markets Team, Email: capmarkets@konikcapitalpartners.com.
This press release shall not constitute an offer to sell or the solicitation of an offer to buy any of the securities described herein, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.
About MAIA Biotechnology
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 ateganosine (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.
MAIA cautions that all statements, other than statements of historical facts contained in this press release, are forward-looking statements (including within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and Section 27A of the Securities Act of 1933, as amended). 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, (vii) our expectations regarding our ability to obtain and maintain intellectual property protection for our product candidates, and (viii) the use of proceeds of our underwritten public offering of common stock, 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. A detailed discussion of these uncertainties and risks that affect our business is contained in our SEC filings, including our reports on Form 10-K and Form 10-Q, particularly under the heading “Risk Factors.” 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.
Enrollment for the OCU400 Phase 3 liMeliGhT clinical trial—the first and largest gene therapy registrational trial for broad retinitis pigmentosa patients—was completed. Topline Phase 3 data expected in the first quarter 2027, advancing OCU400 towards potential approval in 2027.
OCU410ST Phase 2/3 pivotal confirmatory trial nearing enrollment completion. Interim data expected in the third quarter 2026, followed by topline Phase 2/3 data in the second quarter 2027 in advance of the BLA submission.
OCU410 positive preliminary Phase 2 data announced in January. Full Phase 2 data expected in March 2026.
First regional licensing agreement for OCU400 in 2025 initiates strategic partnership strategy ahead of commercialization
Rounded out executive leadership team with top talent in business development, commercial, finance, and operations to encompass all required expertise for upcoming growth
MALVERN, Pa., March 04, 2026 (GLOBE NEWSWIRE) — Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today reported fourth quarter and full year 2025 financial results along with a general business update.
“Considerable development across all our modifier gene therapy programs, notable licensing and financing agreements to strengthen our financial position, and meaningful appointments to our leadership team made 2025 a transformative year for Ocugen,” said Dr. Shankar Musunuri, Chairman, CEO, Co-founder of Ocugen. “We are poised to leverage upcoming catalysts and advance the business as we near the first of our three BLA filings.”
Enrollment is now complete for the OCU400 Phase 3 liMeliGhT clinical trial for retinitis pigmentosa (RP). As a one-year clinical trial, topline data will be available in the first quarter of 2027. These data are anticipated to support the Biologics License Application (BLA) filing for OCU400 and potential approval in 2027. The liMeliGhT clinical trial enrolled 140 patients who were randomized 2:1 into the treatment group (2.5× vg per eye 250 µL) and untreated control group across mutations (RHO and gene-agnostic arms). The target population included patients with early- to late-stage disease among a broad RP population, including pediatrics (3+ years). The primary endpoint is 12-month change in visual function assessed by LDNA (luminance dependent navigation assessment) with improvement in Lux Level from baseline to 12 months. The OCU400 Phase 3 liMeliGhT clinical trial is the only broad RP gene-agnostic trial and the largest known Phase 3 orphan gene therapy trial.
The OCU410ST Phase 2/3 GARDian clinical trial for Stargardt disease (ST) remains ahead of schedule in preparation for the 2027 BLA filing. In January, the Company announced publication of Phase 1 GARDian1 trial results for OCU410ST in EYE. The study supports the favorable safety, tolerability and efficacy profile of OCU410ST and its potential to provide clinically meaningful functional and structural benefits in ST patients. OCU410ST holds the potential to address the unmet medical need that remains for approximately 100,000 Stargardt patients in the U.S. and Europe who have no treatment option available.
Recently, Ocugen announced positive preliminary 12-month data (~50% of patients evaluated to date) from the Phase 2 ArMaDa clinical trial evaluating OCU410 (AAV5-RORA), its novel modifier gene therapy for geographic atrophy (GA) secondary to dry age-related macular degeneration (dAMD). Key findings from Phase 2 include 46% lesion growth reduction (medium + high dose vs. control; p=0.015; N=23) at 12 months and 50% responder rate with patients achieving >50% lesion size reduction vs. control. A subgroup analysis of patients with a baseline GA size ≥7.5 mm²—representing advanced atrophy—demonstrated a 57% reduction in lesion growth in treated eyes for medium dose and a 56% reduction in high dose compared with control eyes. This reduction in lesion size in medium and high doses suggests OCU410 may be more effective in patients with substantial disease burden.
The latest OCU410 data set also included encouraging 12-month Phase 1 findings where OCU410-treated eyes demonstrated 60% slower loss of the ellipsoid zone (a structural and functional exploratory endpoint) compared to untreated fellow eyes. The 60% reduction in ellipsoid zone (EZ) loss rate indicates that OCU410 treatment is substantially slowing the rate of photoreceptor degeneration compared to the natural history observed in the untreated fellow eye of the same patient.
“With approximately 2 to 3 million GA patients in the U.S. and Europe combined, OCU410 represents a significant market opportunity. Current therapies have notable limitations, and there are no treatments approved for GA in Europe, as existing FDA-approved options fail to demonstrate meaningful functional outcomes,” said Dr. Musunuri. “OCU410 is therefore well-positioned to address this critical unmet need, and we look forward to reporting full data from the OCU410 Phase 2 clinical trial this month and initiating Phase 3 in 2026.”
The licensing agreement with Kwangdong Pharmaceutical, Co., Ltd. for the exclusive Korean rights to OCU400—with upfront fees and near-term development milestone payments, along with royalties—was a critical step in Ocugen’s business development strategy, affirming a regional partnership approach for OCU400 that preserves the Company’s rights to larger geographies while also generating a potential return for shareholders.
To extend the cash runway into the fourth quarter of 2026, in January 2026 the Company secured $22.5 million in gross proceeds through an underwritten registered direct offering of common stock led by RTW Investments, with additional participation from new and existing investors. This raise follows the $20 million registered direct offering of common stock and warrants with Janus Henderson Investors in August 2025. The Company may receive up to $30 million of additional gross proceeds from the August 2025 registered direct offering if the warrants are exercised in full.
“I am proud of our accomplishments in 2025, as they accelerate our drive to achieve even more significant clinical and pre-commercial objectives in 2026,” said Dr. Musunuri. “With a full bench of experienced leadership across the organization, I am confident that we have the resources and know-how to take Ocugen to the next level.”
Business Updates
Novel Modifier Gene Therapy Platform—Targeting Three BLA Filings in the Next Three Years
OCU400 – Completed enrollment in the Phase 3 liMeliGhT clinical trial for OCU400 and are on track to file the rolling BLA in the third quarter of 2026. Subjects will be followed for a year after dosing for primary endpoint analyses. Positive long-term, 3-year Phase 1/2 durable, safety and tolerability data demonstrates sustained clinically meaningful, approximately 2-line LLVA gain, reinforcing durable gene-agnostic benefit.
OCU410ST – The Phase 2/3 GARDian3 pivotal confirmatory trial is progressing ahead of schedule with anticipated enrollment completion in the first quarter of 2026. Interim data is expected in the third quarter of 2026.
OCU410 – In January 2026, Ocugen announced positive preliminary 12-month data for Phase 2 subjects from the ArMaDa clinical trial for GA secondary to dAMD. The complete data set for the ArMaDa trial is expected to be available in March 2026.
Other Programs
OCU200 – No serious adverse events (SAEs) or adverse events (AEs) related to OCU200 reported to date across the dose-escalation cohorts and trial enrollment is expected to be completed by the first quarter of 2026.
OCU500 – NIAID intends to initiate the OCU500 Phase 1 clinical trial in the second quarter of 2026.
NeoCart – Created OrthoCellix as a wholly-owned subsidiary of Ocugen for the regenerative cell therapy assets with a goal of obtaining independent financing.
Financial Results
Fourth quarter — Research and development expenses for the three months ended December 31, 2025, were $10.7 million compared to $8.3 million for the three months ended December 31, 2024. General and administrative expenses for the three months ended December 31, 2025, were $6.1 million compared to $6.3 million for the three months ended December 31, 2024. Ocugen reported a $0.06 net loss per common share for the three months ended December 31, 2025, compared to a $0.05 net loss per common share for the three months ended December 31, 2024.
Full year — Research and development expenses for the year ended December 31, 2025, were $39.8 million compared to $32.1 million for the year ended December 31, 2024. General and administrative expenses for the year ended December 31, 2025, were $27.6 million compared to $26.7 million for the year ended December 31, 2024. Ocugen reported a $0.23 net loss per common share for the year ended December 31, 2025, compared to a $0.20 net loss per common share for the year ended December 31, 2024.
Ocugen’s cash and restricted cash, totalled $18.9 million as of December 31, 2025, compared to $58.8 million as of December 31, 2024. The Company estimates that additional proceeds from the $22.5 million financing in January 2026 will enable it to fund its operations into the fourth quarter of 2026. If the Janus Henderson warrants are fully exercised this year, it is expected that cash runway will be extended into the second quarter of 2027. The Company had 312.4 million shares of common stock outstanding as of December 31, 2025.
Conference Call and Webcast Details
Ocugen has scheduled a conference call and webcast for 8:30 a.m. ET today to discuss the financial results and recent business highlights. Ocugen’s leadership team will host the call, which will be open to all listeners. There will also be a question-and-answer session following the prepared remarks.
Attendees are invited to participate on the call or webcast using the following details:
Dial-in Numbers: (800) 715-9871 for U.S. callers and (646) 307-1963 for international callers Conference ID: 3029428 Webcast: Available on the events section of the Ocugen investor site
A replay of the call and archived webcast will be available for approximately 45 days following the event on the Ocugen investor site.
About Ocugen, Inc. Ocugen, Inc. is a pioneering biotechnology leader in gene therapies for blindness diseases. Our breakthrough modifier gene therapy platform has the potential to address significant unmet medical need for large patient populations through our gene-agnostic approach. Unlike traditional gene therapies and gene editing, Ocugen’s modifier gene therapies address the entire disease—complex diseases that are potentially caused by imbalances in multiple gene networks. Currently we have programs in development for inherited retinal diseases and blindness diseases affecting millions across the globe, including retinitis pigmentosa, Stargardt disease, and geographic atrophy—late stage dry age-related macular degeneration. Discover more at www.ocugen.com and follow us on X and LinkedIn.
Cautionary Note on Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995, including, but not limited to, strategy, business plans and objectives for Ocugen’s clinical programs, plans and timelines for the preclinical and clinical development of Ocugen’s product candidates, including the therapeutic potential, clinical benefits and safety thereof, expectations regarding timing, success and data announcements of current ongoing preclinical and clinical trials, including the timing of enrollment and data readouts, the ability to initiate new clinical programs, Ocugen’s financial condition and expected cash runway into the fourth quarter of 2026, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines, statements regarding potential market size and commercial possibilities of Ocugen’s product candidates, and Ocugen’s projections under its license agreement with Kwangdong Pharmaceutical Co., Ltd., 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, including, but not limited to, the risks that preliminary, interim and top-line clinical trial results may not be indicative of, and may differ from, final clinical data; that unfavorable new clinical trial data may emerge in ongoing clinical trials or through further analyses of existing clinical trial data; that earlier non-clinical and clinical data and testing of may not be predictive of the results or success of later clinical trials; and that that clinical trial data are subject to differing interpretations and assessments, including by regulatory authorities. These and other risks and uncertainties are more fully described in our annual and 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.
Financing included participation by healthcare-dedicated investors alongside existing shareholders
CHICAGO, IL, March 02, 2026 (GLOBE NEWSWIRE) — MAIA Biotechnology, Inc., (NYSE American: MAIA) (“MAIA”, the “Company”), a clinical-stage biopharmaceutical company developing targeted immunotherapies for cancer, today announced the pricing of its underwritten public offering of 20,000,000 shares of its common stock at a public offering price of $1.50 per share for aggregate gross proceeds of $30 million, prior to deducting underwriting discounts and other offering expenses. In addition, the Company has granted the underwriters a 45-day option to purchase up to an additional 3,000,000 shares of common stock at the public offering price per share, less the underwriting discounts to cover over-allotments, if any. The offering is expected to close on March 4, 2026, subject to satisfaction of customary closing conditions.
The offering was structured as a straightforward common stock only investment with no warrant coverage and was led by healthcare-dedicated investors alongside existing shareholders.
Konik Capital Partners, LLC, a division of T.R. Winston & Company is acting as the sole book-running manager for the offering.
MAIA intends to use the net proceeds from the offering to conduct clinical trials and for working capital and general corporate purposes.
The securities described above are being offered and sold pursuant to a “shelf” registration statement on Form S-3 (File No. 333-273984), including a base prospectus, filed with the U.S. Securities and Exchange Commission (the “SEC”) on August 15, 2023, and declared effective on August 23, 2023.
The offering is being made only by means of a prospectus supplement and accompanying prospectus that form a part of the registration statement.. A prospectus supplement describing the terms of the public offering will be filed with the SEC and will form a part of the effective registration statement. A preliminary prospectus supplement and accompanying prospectus relating to this offering have been filed with the SEC.
Copies of the prospectus supplement and the accompanying prospectus relating to this offering may be obtained, when available, on the SEC’s website at http://www.sec.gov or by contacting Konik Capital Partners LLC, a division of T.R. Winston & Company, at 7 World Trade Center, 46th Floor, New York, NY 10007, Attention: Capital Markets Team, Email: capmarkets@konikcapitalpartners.com.
This press release shall not constitute an offer to sell or the solicitation of an offer to buy any of the securities described herein, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction.
About MAIA Biotechnology
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 ateganosine (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.
MAIA cautions that all statements, other than statements of historical facts contained in this press release, are forward-looking statements (including within the meaning of Section 21E of the Securities Exchange Act of 1934, as amended, and Section 27A of the Securities Act of 1933, as amended). 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, (vii) our expectations regarding our ability to obtain and maintain intellectual property protection for our product candidates and (viii) the closing of our underwritten public offering of common stock, 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. A detailed discussion of these uncertainties and risks that affect our business is contained in our SEC filings, including our reports on Form 10-K and Form 10-Q, particularly under the heading “Risk Factors.” 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.
