BOTHELL, Wash., March 31, 2025 (GLOBE NEWSWIRE) — Cocrystal Pharma, Inc. (Nasdaq: COCP) (“Cocrystal” or the “Company”) reports financial results for the 12 months ended December 31, 2024, and provides updates on its antiviral product pipeline, upcoming milestones and business activities.
“Our novel, potent antiviral compounds for norovirus, influenza and coronavirus address critical gaps in global health where effective treatments or vaccines are currently lacking,” said Sam Lee, Ph.D., President and co-CEO of Cocrystal. “We plan to initiate a norovirus human challenge study in the U.S. later this year to evaluate our pan-viral protease inhibitor CDI-988 for the potential treatment and prevention of norovirus infection. The big surge in reported norovirus outbreaks is possibly due to norovirus variants switching from GII.4 to GII.17, as well as increased social gathering after the COVID-19 pandemic. Norovirus is the most common cause of acute gastroenteritis and there are no approved therapeutics or vaccines, making it a compelling target.
“We are optimistic that our oral PB2 inhibitor CC-42344 will have potential as a treatment for seasonal influenza A infection and pandemic avian influenza,” he added. “Following the unexpected low infection rate from the Phase 2a challenge study that precluded us from obtaining meaningful human efficacy data, we plan to continue the influenza challenge study.”
“News coverage in recent months of norovirus and avian flu outbreaks underscore the urgent need for new antiviral solutions,” said James Martin, CFO and co-CEO of Cocrystal. “We are advancing our first- and best-in-class antiviral drug compounds that were designed using our Nobel Prize-winning structure-based technologies for these high-value viral targets that address multibillion-dollar markets.”
Antiviral Product Pipeline Overview
We apply our proprietary structure-based drug discovery platform technology for developing broad-spectrum antivirals that inhibit viral replication. By designing and selecting candidates that target highly conserved regions of the viral enzymes, we seek to develop drugs that are effective against the virus and mutations of the virus, while reducing off-target interactions that may cause undesirable side effects. Our drug discovery process differs from traditional, empirical medicinal chemistry approaches that often require iterative high-throughput compound screening and lengthy hit-to-lead processes.
Oral CC-42344 for the treatment of pandemic and seasonal influenza A
Our novel PB2 inhibitor CC-42344 showed excellent in vitro antiviral activity against pandemic and seasonal influenza A strains, as well as strains that are resistant to Tamiflu® and Xofluza®.
In December 2022 we reported favorable safety and tolerability results from the oral CC-42344 Phase 1 study.
In December 2023 we began a randomized, double-blind, placebo-controlled Phase 2a human challenge study to evaluate the safety, tolerability, viral and clinical measurements of CC-42344 in influenza A-infected subjects in the United Kingdom, following authorization from the UK Medicines and Healthcare Products Regulatory Agency (MHRA).
In May 2024 we completed enrollment in the Phase 2a human challenge study.
In June 2024 we reported that in vitro studies demonstrated CC-42344 inhibits the activity of the highly pathogenic avian influenza A (H5N1) PB2 protein identified in humans exposed to infected dairy cows.
In December 2024 we announced a plan to extend the CC-42344 human challenge study due to unexpectedly low influenza infection among study participants.
Inhaled CC-42344 as prophylaxis and treatment for pandemic and seasonal influenza A
Our preclinical testing showed superior pulmonary pharmacology with CC-42344 including high exposure to drug and a long half-life.
We have completed CC-42344 inhalation formulation development and GLP toxicology studies.
Influenza A/B program
Our efforts to develop a preclinical lead of novel influenza replication inhibitors are ongoing.
Oral pan-viral protease inhibitor CDI-988 for the treatment of noroviruses and coronaviruses
Our novel, broad-spectrum protease inhibitor CDI-988 is being evaluated as a potential oral treatment for noroviruses and coronaviruses.
CDI-988 has shown in vitro pan-viral activity against multiple norovirus strains.
In May 2023 we announced approval of our application to the Australian regulatory agency for a randomized, double-blind, placebo-controlled Phase 1 study to evaluate the safety, tolerability and pharmacokinetics (PK) of oral CDI-988 in healthy volunteers.
In August 2023 we announced our selection of CDI-988 as our lead compound for the oral treatment for noroviruses, in addition to coronaviruses.
In July 2024 we reported favorable safety and tolerability results from the single-ascending dose cohorts in the Phase 1 study.
In December 2024 we reported favorable safety and tolerability results from the multiple-ascending dose cohorts of the Phase 1 study and the addition of a higher-dose cohort.
We expect to report topline results from the high-dose healthy volunteer cohort with CDI-988 in the second quarter of 2025.
We plan to initiate a human challenge study in the U.S. in 2025 to evaluate CDI-988 as a norovirus treatment and prophylaxis.
SARS-CoV-2 and Other Coronavirus Programs
By targeting viral replication enzymes and proteases, we believe it is possible to develop effective treatments for all diseases caused by coronaviruses including SARS-CoV-2 and its variants, Severe Acute Respiratory Syndrome (SARS) and Middle East Respiratory Syndrome (MERS). CDI-988 showed potent in vitro pan-viral activity against common human coronaviruses, rhinoviruses and respiratory enteroviruses, as well as against noroviruses. The global COVID-19 therapeutics market is estimated to exceed $16 billion annually by the end of 2031.
Oral pan-viral protease inhibitor CDI-988 for the treatment of coronaviruses and noroviruses
CDI-988 exhibited superior in vitro potency against SARS-CoV-2 and demonstrated a favorable safety profile and PK properties.
In September 2023 we dosed the first healthy subject in our dual pan-norovirus/pan-coronavirus oral CDI-988 study, which is expected to serve as a Phase 1 study for both indications.
In July 2024 we reported favorable safety and tolerability results from the single-ascending dose cohort in the Phase 1 study.
In December 2024 we reported favorable safety and tolerability results from the multiple-ascending dose cohorts of the Phase 1 study and the addition of a higher-dose cohort.
We expect to report topline results from the higher dose cohort in the CDI-988 Phase 1 study in the second quarter of 2025.
2024 Financial Results
Research and development (R&D) expenses for 2024 were $12.5 million, compared with $15.2 million for 2023. The decrease was primarily due to the timing of clinical study costs. General and administrative (G&A) expenses for 2024 were $5.3 million, compared with $6.0 million for 2023, with the $0.7 million decrease primarily due to a reduction of insurance costs and other expenses.
During 2023 the Company received $2.6 million related to litigation with an insurer.
The net loss for 2024 was $17.5 million, or $1.72 per share, compared with the net loss for 2023 of $18.0 million, or $1.87 per share. For 2024 the year over-year-net loss was reduced by $3.1 million exclusive of the $2.6 million received in 2023 noted above.
Cocrystal reported unrestricted cash as of December 31, 2024 of $9.9 million, compared with $26.4 million as of December 31, 2023. Net cash used in operating activities for 2024 was $16.5 million, compared with $14.7 million for 2023. The Company had working capital of $9.2 million and 10.2 million common shares outstanding as of December 31, 2024.
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. 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 plans for the future development of preclinical and clinical product candidates, our expectations regarding future characteristics of the product candidates we develop, the expected time of achieving certain value-driving milestones in our programs, including preparation, commencement and advancement of clinical studies for certain product candidates in 2025, our plans regarding further clinical development of such product candidates, and the viability and efficacy of potential treatments for diseases our product candidates are designed to treat, and expectations for the markets for certain therapeutics. 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 our need for additional capital to fund our operations over the next 12 months, inflation, the possibility of a recession, interest rate increases, imposed and threated tariffs, and geopolitical conflicts including those in Ukraine and Israel on our Company, our collaboration partners, and on the U.S., UK, Australia 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 studies as well as similar problems with our vendors and our current and any future clinical research organization (CROs) and contract manufacturing organizations (CMOs), the progress and results of the studies for CC-42344 and CDI-988 including the delay of the Phase 2a study for CC-42344 which may require us to incur substantial additional costs, 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 including potential downward pressure on government spending on the biopharmaceutical and healthcare industry based on policies and actions taken by the Trump Administration in the U.S., the impact of the Trump Administration’s policies and actions on regulation affecting the FDA and other healthcare agencies and potential staffing issues resulting therefrom, potential mutations in a virus we are targeting that may result in variants that are resistant to a product candidate we develop, and 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. 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. 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.
CHATHAM, N.J., March 27, 2025 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a biopharmaceutical company with marketed products and a pipeline of development candidates, today announced that Seth Lederman, M.D., Chief Executive Officer, will present a poster at the AAPM 2025 Annual Meeting, PainConnect, in Austin, Texas on April 5, 2025, at 11:45 a.m. CT. The meeting will take place from April 3-6, 2025.
Tonix is a fully-integrated biopharmaceutical company focused on transforming therapies for pain management and vaccines for public health challenges. Tonix’s development portfolio is focused on central nervous system (CNS) disorders. Tonix’s priority is to advance TNX-102 SL, a product candidate for the management of fibromyalgia, for which an NDA was submitted based on two statistically significant Phase 3 studies for the management of fibromyalgia and for which a PDUFA (Prescription Drug User Fee act) goal date of August 15, 2025 has been assigned for a decision on marketing authorization. The FDA has also granted Fast Track designation to TNX-102 SL for the management of fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction and acute stress disorder under a Physician-Initiated IND at the University of North Carolina in the OASIS study funded by the U.S. Department of Defense (DoD). Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase), a biologic in Phase 2 development designed to treat cocaine intoxication that has FDA Breakthrough Therapy designation, and its development is supported by a grant from the National Institute on Drug Abuse. Tonix’s immunology development portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is an Fc-modified humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft rejection and for the treatment of autoimmune diseases. Tonix also has product candidates in development in infectious disease, including a vaccine for mpox, TNX-801. Tonix recently announced a contract with the U.S. DoD’s Defense Threat Reduction Agency (DTRA) for up to $34 million over five years to develop TNX-4200, small molecule broad-spectrum antiviral agents targeting CD45 for the prevention or treatment of infections to improve the medical readiness of military personnel in biological threat environments. Tonix owns and operates a state-of-the art infectious disease research facility in Frederick, Md. Tonix Medicines, our commercial subsidiary, markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan nasal spray) 10 mg for the treatment of acute migraine with or without aura in adults.
* Tonix’s product development candidates are investigational new drugs or biologics; 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. All other marks are property of their respective owners.
This press release and further information about Tonix can be found at www.tonixpharma.com.
Forward Looking Statements
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as “anticipate,” “believe,” “forecast,” “estimate,” “expect,” and “intend,” among others. These forward-looking statements are based on Tonix’s current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, risks related to the failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; risks related to the failure to successfully market any of our products; risks related to the timing and progress of clinical development of our product candidates; our need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party payor reimbursement; limited research and development efforts and dependence upon third parties; and substantial competition. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Tonix does not undertake an obligation to update or revise any forward-looking statement. Investors should read the risk factors set forth in the Annual Report on Form 10-K for the year ended December 31, 2024, as filed with the Securities and Exchange Commission (the “SEC”) on March 18, 2025, and periodic reports filed with the SEC on or after the date thereof. All of Tonix’s forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date thereof.
Zembrace® SymTouch® (sumatriptan succinate) injection (Zembrace) and Tosymra® (sumatriptan) nasal spray are prescription medicines used to treat acute migraine headaches with or without aura in adults who have been diagnosed with migraine.
Zembrace and Tosymra are not used to prevent migraines. It is not known if Zembrace or Tosymra are safe and effective in children under 18 years of age.
Important Safety Information
Zembrace and Tosymra can cause serious side effects, including heart attack and other heart problems, which may lead to death. Stop use and get emergency help if you have any signs of a heart attack:
discomfort in the center of your chest that lasts for more than a few minutes or goes away and comes back
severe tightness, pain, pressure, or heaviness in your chest, throat, neck, or jaw
pain or discomfort in your arms, back, neck, jaw or stomach
shortness of breath with or without chest discomfort
breaking out in a cold sweat
nausea or vomiting
feeling lightheaded
Zembrace and Tosymra are not for people with risk factors for heart disease (high blood pressure or cholesterol, smoking, overweight, diabetes, family history of heart disease) unless a heart exam shows no problem.
Do not use Zembrace or Tosymra if you have:
history of heart problems
narrowing of blood vessels to your legs, arms, stomach, or kidney (peripheral vascular disease)
uncontrolled high blood pressure
hemiplegic or basilar migraines. If you are not sure if you have these, ask your provider.
had a stroke, transient ischemic attacks (TIAs), or problems with blood circulation
severe liver problems
taken any of the following medicines in the last 24 hours: almotriptan, eletriptan, frovatriptan, naratriptan, rizatriptan, ergotamines, or dihydroergotamine. Ask your provider for a list of these medicines if you are not sure.
are taking certain antidepressants, known as monoamine oxidase (MAO)-A inhibitors or it has been 2 weeks or less since you stopped taking a MAO-A inhibitor. Ask your provider for a list of these medicines if you are not sure.
an allergy to sumatriptan or any of the components of Zembrace or Tosymra
Tell your provider about all of your medical conditions and medicines you take, including vitamins and supplements.
Zembrace and Tosymra can cause dizziness, weakness, or drowsiness. If so, do not drive a car, use machinery, or do anything where you need to be alert.
Zembrace and Tosymra may cause serious side effects including:
changes in color or sensation in your fingers and toes
sudden or severe stomach pain, stomach pain after meals, weight loss, nausea or vomiting, constipation or diarrhea, bloody diarrhea, fever
cramping and pain in your legs or hips; feeling of heaviness or tightness in your leg muscles; burning or aching pain in your feet or toes while resting; numbness, tingling, or weakness in your legs; cold feeling or color changes in one or both legs or feet
increased blood pressure including a sudden severe increase even if you have no history of high blood pressure
medication overuse headaches from using migraine medicine for 10 or more days each month. If your headaches get worse, call your provider.
serotonin syndrome, a rare but serious problem that can happen in people using Zembrace or Tosymra, especially when used with anti-depressant medicines called SSRIs or SNRIs. Call your provider right away if you have: mental changes such as seeing things that are not there (hallucinations), agitation, or coma; fast heartbeat; changes in blood pressure; high body temperature; tight muscles; or trouble walking.
hives (itchy bumps); swelling of your tongue, mouth, or throat
seizures even in people who have never had seizures before
The most common side effects of Zembrace and Tosymra include: pain and redness at injection site (Zembrace only); tingling or numbness in your fingers or toes; dizziness; warm, hot, burning feeling to your face (flushing); discomfort or stiffness in your neck; feeling weak, drowsy, or tired; application site (nasal) reactions (Tosymra only) and throat irritation (Tosymra only).
Tell your provider if you have any side effect that bothers you or does not go away. These are not all the possible side effects of Zembrace and Tosymra. For more information, ask your provider.
This is the most important information to know about Zembrace and Tosymra but is not comprehensive. For more information, talk to your provider and read the Patient Information and Instructions for Use. You can also visit https://www.tonixpharma.com or call 1-888-869-7633.
You are encouraged to report adverse effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
SAN DIEGO, March 27, 2025 (GLOBE NEWSWIRE) — Gyre Therapeutics (“Gyre”) (Nasdaq: GYRE), an innovative, commercial-stage biotechnology company with clinical development programs focusing on organ fibrosis, today announced the publication of the manuscript titled “Hydronidone for the Treatment of Liver Fibrosis Associated with Chronic Hepatitis B: Protocol for a Phase 3 Randomized Trial” in the Journal of Clinical and Translational Hepatology. This publication details the full protocol for the pivotal Phase 3 trial to support the use of hydronidone in Chinese patients with liver fibrosis associated with chronic hepatitis B (“CHB”). The published protocol outlines patient inclusion criteria, randomization and blinding processes, key assessments, and the statistical analysis plan.
The randomized, double-blind, placebo-controlled, multicenter Phase 3 trial (NCT05115942) completed the enrollment of 248 patients across 44 clinical research hospitals in the People’s Republic of China (“PRC”) in October 2024. Patients were randomized 1:1 to receive either F351 or placebo in addition to entecavir antiviral basic therapy for CHB. The primary endpoint is a decrease in liver fibrosis (as measured by the Ishak Scoring System) by at least one stage after 52 weeks of treatment relative to baseline.
The PRC’s National Medical Products Administration (“NMPA”) designated F351 as a “Breakthrough Therapy” in 2021. Gyre expects to report topline results from this Phase 3 trial in the second quarter of 2025.
About Hydronidone (F351)
F351 is a next-generation anti-fibrotic compound and a structural analogue of Pirfenidone, the first approved treatment for idiopathic pulmonary fibrosis (“IPF”) in Japan, the European Union, the United States and the PRC. F351’s dual mechanism has been shown to inhibit in vitro p38γ kinase activity and TGF-β1-driven collagen overproduction—both key drivers of liver fibrosis. F351 specifically targets hepatic stellate cells (“HSCs”), which are central to the progression of fibrosis. In preclinical studies, it has shown strong anti-proliferative and anti-fibrotic effects on HSCs. These effects have been validated across multiple in vivo models of liver fibrosis, including CCl4-induced liver fibrosis in mice, DMN- and HSA-induced liver fibrosis in rats, and a mouse model of MASH fibrosis (a form of MASH with fibrosis) induced by CCl4 plus a Western high-fat diet. Together, these results highlight F351’s potential as a differentiated treatment for liver fibrosis, with a unique mechanism and strong preclinical and clinical evidence supporting its advancement into later-stage development.
About Gyre Pharmaceuticals
Gyre Pharmaceuticals is a commercial-stage biopharmaceutical company committed to the research, development, manufacturing and commercialization of innovative drugs for organ fibrosis. Its flagship product, ETUARY® (Pirfenidone capsule), was the first approved treatment for IPF in the PRC in 2011 and has maintained a prominent market share (2024 net sales of $105.8 million). In addition, Gyre Pharmaceuticals is evaluating F351 in a Phase 3 clinical trial in CHB-associated liver fibrosis in the PRC, which is expected to readout topline data by Q2 2025. F351 received Breakthrough Therapy designation by the NMPA Center for Drug Evaluation in March 2021. Gyre Pharmaceuticals is also developing treatments for PD, DKD, COPD, PAH and ALF/ACLF. In October 2023, Gyre Therapeutics acquired an indirect majority interest in Gyre Pharmaceuticals (also known as Beijing Continent Pharmaceuticals Co., Ltd.).
About Gyre Therapeutics
Gyre Therapeutics is a biopharmaceutical company headquartered in San Diego, CA, with a primary focus on the development and commercialization of F351 (Hydronidone) for the treatment of MASH-associated fibrosis in the U.S. Gyre’s development strategy for F351 in MASH is based on the company’s experience in MASH rodent model mechanistic studies and CHB-induced liver fibrosis clinical studies. Gyre is also advancing a diverse pipeline in the PRC through its indirect controlling interest in Gyre Pharmaceuticals, including ETUARY therapeutic expansions, F573, F528, and F230.
Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995, which statements are subject to substantial risks and uncertainties and are based on estimates and assumptions. All statements, other than statements of historical facts included in this press release, are forward-looking statements, including statements concerning: the expectations regarding Gyre’s research and development efforts and timing of expected clinical readouts, including timing of topline data from Gyre Pharmaceuticals’ Phase 3 clinical trial evaluating F351 for the treatment of CHB-associated liver fibrosis in the PRC. In some cases, you can identify forward-looking statements by terms such as “may,” “might,” “will,” “objective,” “intend,” “should,” “could,” “can,” “would,” “expect,” “believe,” “design,” “estimate,” “predict,” “potential,” “plan” or the negative of these terms, and similar expressions intended to identify forward-looking statements. These statements reflect our plans, estimates, and expectations, as of the date of this press release. These statements involve known and unknown risks, uncertainties and other factors that could cause our actual results to differ materially from the forward-looking statements expressed or implied in this press release. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation: Gyre’s ability to execute on its clinical development strategies; positive results from a clinical trial may not necessarily be predictive of the results of future or ongoing clinical trials; the timing or likelihood of regulatory filings and approvals; competition from competing products; the impact of general economic, health, industrial or political conditions in the United States or internationally; the sufficiency of Gyre’s capital resources and its ability to raise additional capital. Additional risks and factors are identified under “Risk Factors” in Gyre’s Annual Report on Form 10-K for the year ended December 31, 2024 filed on March 17, 2025 and in other filings the Company may make with the Securities and Exchange Commission.
Gyre expressly disclaims any obligation to update any forward-looking statements whether as a result of new information, future events or otherwise, except as required by law.
Poster highlights potential predictive biomarker for therapeutic response in advanced non-small cell lung cancer (NSCLC)
CHICAGO–(BUSINESS WIRE)– MAIA Biotechnology, Inc., (NYSE American: MAIA) (“MAIA”, the “Company”), a clinical-stage biopharmaceutical company developing targeted immunotherapies for cancer, today announced that an abstract detailing a potential predictive biomarker for THIO treatment was selected for poster presentation at the European Lung Cancer Congress 2025 (ELCC 2025) taking place, March 26-29, in Paris, France. ELCC is a program of the European Society for Medical Oncology (ESMO).
“We are proud to join ELCC 2025, a premier conference focused directly on the science of thoracic oncology,” said Vlad Vitoc, M.D., CEO of MAIA. “Our poster features our latest findings on cytokine Interleukin-6 (IL-6) as a potential predictive immune response biomarker for THIO sequenced with a checkpoint inhibitor. Predictive biomarkers can further illuminate THIO’s unique mechanisms of action which have shown exceptional efficacy in our Phase 2 clinical trial.”
Presentation details:
Title:
Phase 2 Study of Telomere-Targeting Agent THIO Sequenced by Cemiplimab in Immune Checkpoint Inhibitor-Resistant Advanced NSCLC: Interleukin-6 as a Potential Predictive Biomarker
Abstract number:
997
Date:
March 28, 2025
Time:
12:00 p.m. CET
Presenter:
Tomasz Jankowski, M.D., Ph.D. – Lead investigator for THIO-101 Phase 2 clinical trial
The European Lung Cancer Congress is a collaborative effort of the most important multidisciplinary societies representing thoracic oncology specialists, working together to advance science, disseminate education and improve the practice of lung cancer specialists worldwide.
About Ateganosine
Ateganosine (THIO, 6-thio-dG or 6-thio-2’-deoxyguanosine) is a first-in-class investigational telomere-targeting agent currently in clinical development to evaluate its activity in Non-Small Cell Lung Cancer (NSCLC). Telomeres, along with the enzyme telomerase, play a fundamental role in the survival of cancer cells and their resistance to current therapies. The modified nucleotide 6-thio-2’-deoxyguanosine induces telomerase-dependent telomeric DNA modification, DNA damage responses, and selective cancer cell death. Ateganosine-damaged telomeric fragments accumulate in cytosolic micronuclei and activates both innate (cGAS/STING) and adaptive (T-cell) immune responses. The sequential treatment with ateganosine followed by PD-(L)1 inhibitors resulted in profound and persistent tumor regression in advanced, in vivo cancer models by induction of cancer type–specific immune memory. Ateganosine is presently developed as a second or later line of treatment for NSCLC for patients that have progressed beyond the standard-of-care regimen of existing checkpoint inhibitors.
About MAIA Biotechnology, Inc.
MAIA is a targeted therapy, immuno-oncology company focused on the development and commercialization of potential first-in-class drugs with novel mechanisms of action that are intended to meaningfully improve and extend the lives of people with cancer. Our lead program is ateganosine, a potential first-in-class cancer telomere targeting agent in clinical development for the treatment of NSCLC patients with telomerase-positive cancer cells. For more information, please visit www.maiabiotech.com.
Forward Looking Statements
MAIA cautions that all statements, other than statements of historical facts contained in this press release, are forward-looking statements. Forward-looking statements are subject to known and unknown risks, uncertainties, and other factors that may cause our or our industry’s actual results, levels or activity, performance or achievements to be materially different from those anticipated by such statements. The use of words such as “may,” “might,” “will,” “should,” “could,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify forward looking statements. However, the absence of these words does not mean that statements are not forward-looking. For example, all statements we make regarding (i) the initiation, timing, cost, progress and results of our preclinical and clinical studies and our research and development programs, (ii) our ability to advance product candidates into, and successfully complete, clinical studies, (iii) the timing or likelihood of regulatory filings and approvals, (iv) our ability to develop, manufacture and commercialize our product candidates and to improve the manufacturing process, (v) the rate and degree of market acceptance of our product candidates, (vi) the size and growth potential of the markets for our product candidates and our ability to serve those markets, and (vii) our expectations regarding our ability to obtain and maintain intellectual property protection for our product candidates, are forward looking. All forward-looking statements are based on current estimates, assumptions and expectations by our management that, although we believe to be reasonable, are inherently uncertain. Any forward-looking statement expressing an expectation or belief as to future events is expressed in good faith and believed to be reasonable at the time such forward-looking statement is made. However, these statements are not guarantees of future events and are subject to risks and uncertainties and other factors beyond our control that may cause actual results to differ materially from those expressed in any forward-looking statement. Any forward-looking statement speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. In this release, unless the context requires otherwise, “MAIA,” “Company,” “we,” “our,” and “us” refers to MAIA Biotechnology, Inc. and its subsidiaries.
ATLANTA, GA, March 24, 2025 – GeoVax Labs, Inc. (Nasdaq: GOVX), a clinical-stage biotechnology company developing immunotherapies and vaccines against cancer and infectious diseases, today announced that it has entered into a securities purchase agreement with a single healthcare-focused institutional investor for the purchase and sale of 3,435,115 shares of its common stock (or common stock equivalents in lieu thereof) and warrants to purchase up to an aggregate of 3,435,115 shares of common stock in a registered direct offering (the “Offering”) at a combined purchase price of $1.31 per share and accompanying warrant, priced at-the-market under Nasdaq rules. The warrants will have an exercise price of $1.31 per share, will be exercisable upon shareholder approval and will expire 5 years from shareholder approval.
The closing of the Offering is expected to occur on or about March 25, 2025, subject to the satisfaction of customary closing conditions. The gross proceeds from the Offering are expected to be approximately $4.5 million, before deducting placement agent fees and other estimated offering expenses. The Company intends to use the net proceeds from the Offering for working capital and general corporate purposes.
A.G.P./Alliance Global Partners is acting as sole placement agent for the Offering.
The Offering is being made pursuant to an effective shelf registration statement on Form S-3 (File No. 333-277585) previously filed with the U.S. Securities and Exchange Commission (the “SEC”). A prospectus supplement describing the terms of the proposed Offering will be filed with the SEC and will be available on the SEC’s website located at http://www.sec.gov. Electronic copies of the prospectus supplement may be obtained, when available, from A.G.P./Alliance Global Partners, 590 Madison Avenue, 28th Floor, New York, NY 10022, or by telephone at (212) 624-2060, or by email at prospectus@allianceg.com.
In connection with the offering, the Company also agreed to amend existing warrants to purchase up to 2,675,610 shares of the Company’s common stock, with an exercise price of $5.00 per share, that were previously issued to the investor participating in this offering. Effective upon closing of this Offering, such existing warrants will be amended to reduce the exercise price to $1.31 per share and the term of such existing warrants will be amended to 5 years from the closing date of the offering.
This press release shall not constitute an offer to sell or a solicitation of an offer to buy these securities, nor shall there be any sale of these securities in any state or other 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 other jurisdiction.
About GeoVax
GeoVax Labs, Inc. is a clinical-stage biotechnology company developing novel vaccines for many of the world’s most threatening infectious diseases and therapies for solid tumor cancers. The company’s lead clinical program is GEO-CM04S1, a next-generation COVID-19 vaccine for which GeoVax was recently awarded a BARDA-funded contract to sponsor a 10,000-participant Phase 2b clinical trial to evaluate the efficacy of GEO-CM04S1 versus an approved COVID-19 vaccine. In addition, GEO-CM04S1 is currently in three Phase 2 clinical trials, being evaluated as (1) a primary vaccine for immunocompromised patients such as those suffering from hematologic cancers and other patient populations for whom the current authorized COVID-19 vaccines are insufficient, (2) a booster vaccine in patients with chronic lymphocytic leukemia (CLL) and (3) a more robust, durable COVID-19 booster among healthy patients who previously received the mRNA vaccines. In oncology the lead clinical program is evaluating a novel oncolytic solid tumor gene-directed therapy, Gedeptin®, having recently completed a multicenter Phase 1/2 clinical trial for advanced head and neck cancers. A Phase 2 clinical trial in first recurrent head and neck cancer, evaluating Gedeptin® combined with an immune checkpoint inhibitor is planned. GeoVax has a strong IP portfolio in support of its technologies and product candidates, holding worldwide rights for its technologies and products. The Company has a leadership team who have driven significant value creation across multiple life science companies over the past several decades. For more information about the current status of our clinical trials and other updates, visit our website: 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.
TNX-102 SL is a non-opioid analgesic; if approved, TNX-102 SL would become the first new drug for treating fibromyalgia in more than 15 years
Commercial planning for TNX-102 SL underway for launch in the fourth quarter of 2025
CHATHAM, N.J., March 24, 2025 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a fully-integrated biopharmaceutical company with marketed products and a pipeline of development candidates, today announced that the U.S. Food and Drug Administration (FDA) will not require an Advisory Committee meeting to discuss the Company’s New Drug Application (NDA) for TNX-102 SL for the management of fibromyalgia. If approved, TNX-102 SL (cyclobenzaprine HCl sublingual tablets) would be the first new treatment option for fibromyalgia patients in 15 years.
“We are pleased that FDA will not require an advisory committee meeting as part of the regulatory review process for TNX-102 SL,” said Seth Lederman, M.D., President, and Chief Executive Officer of Tonix Pharmaceuticals. “We believe that TNX-102 SL has the potential to be the first member of a new class of medicines for the management of fibromyalgia, a debilitating condition affecting over 10 million adults in the U.S. We believe we are well positioned to launch TNX-102 SL for the management of fibromyalgia in the fourth quarter of 2025 if approved by the FDA.”
The FDA previously granted Fast Track designation to TNX-102 SL for the management of fibromyalgia in 2024, a designation intended to expedite FDA review of important new drugs to treat serious conditions and fill an unmet medical need.
About Fibromyalgia
Fibromyalgia is a common chronic pain disorder that is understood to result from amplified sensory and pain signaling within the central nervous system, called central sensitization. Brain imaging studies have localized the functional disorder to the brain’s insula and anterior cingulate cortex. Fibromyalgia afflicts more than 10 million adults in the U.S., the majority of whom are women. Symptoms of fibromyalgia include chronic widespread pain, non-restorative sleep, fatigue, and brain fog (or cognitive dysfunction). Other associated symptoms include mood disturbances, including depression, anxiety, headaches and abdominal pain or cramps. Individuals suffering from fibromyalgia often struggle with their daily activities, have impaired quality of life, and frequently are disabled. Physicians and patients report common dissatisfaction with currently marketed products. Fibromyalgia is now recognized as the prototypic nociplastic syndrome and as a chronic overlapping pain condition (COPC) 1–3. Nociplastic pain is the third primary type of pain in addition to nociceptive pain and neuropathic pain. Many patients present with pain syndromes that are mixtures of the three primary types of pain. Nociplastic syndromes are associated with central and peripheral sensitization. Fibromyalgia can occur without any identifiable precipitating event. However, many fibromyalgia cases follow one or more precipitating event(s) including: post-operative pain, acute or chronic nociceptive or neuropathic pain states; recovery from an infectious illness; a cancer diagnosis or cancer treatment; a metabolic or endocrine stress; or a traumatic event. In the cases of recovery from an infectious illness, fibromyalgia is considered an Infection-Associated Chronic Condition. In addition to fibromyalgia cases associated with other conditions or stressors, the U.S. National Academies of Sciences, Engineering, and Medicine, has concluded that fibromyalgia is a diagnosable condition that can occur after recovery from COVID-19 in the context of Long COVID. Fibromyalgia is also recognized as a Chronic Overlapping Pain Condition, which is a group of related conditions that include chronic fatigue syndrome/myalgic encephalomyelitis (CFS/ME), irritable bowel syndrome, endometriosis, low back pain, post-concussive syndrome (also known as mild traumatic brain injury), chronic Lyme Disease, chronic diabetic neuropathy and chronic post-herpetic neuralgia.
About TNX-102 SL
TNX-102 SL is a centrally acting, non-opioid investigational drug, designed for chronic use. The tablet is a patented sublingual formulation of cyclobenzaprine hydrochloride developed for bedtime dosing for the management of fibromyalgia. Cyclobenzaprine potently binds and acts as an antagonist at four different post-synaptic neuroreceptor subtypes: serotonergic-5-HT2A, adrenergic-α1, histaminergic-H1, and muscarinic-M1-cholinergic receptors. Together, these interactions are believed to target the non-restorative sleep characteristic of fibromyalgia identified by Professor Harvey Moldofsky in 1975. Cyclobenzaprine is not associated with risk of addiction or dependence. The TNX-102 SL tablet is based on a eutectic formulation of cyclobenzaprine HCl and mannitol that provides a stable product which dissolves rapidly and delivers cyclobenzaprine by the transmucosal route efficiently into the bloodstream. The eutectic protects cyclobenzaprine HCl from interacting with the basifying agent that is also part of the formulation and required for efficient transmucosal absorption. Patents based on TNX-102 SL’s eutectic composition and its properties have issued in the U.S., E.U., Japan, China and many other jurisdictions around the world and provide market protection into 2034. The European Patent Office’s Opposition Division maintained Tonix’s European Patent EP 2 968 992 in unamended form after an Opposition was filed against it by a Sandoz subsidiary, Hexal AG. Hexal AG did not appeal that decision. The formulation of TNX-102 SL was designed specifically for sublingual administration and transmucosal absorption for bedtime dosing to target disturbed sleep, while reducing the risk of daytime somnolence. Clinical pharmacokinetic studies indicated that relative to oral cyclobenzaprine, TNX-102 SL results in higher levels of exposure during the first 2 hours after dosing and in deceased levels of the long-lived active metabolite, norcyclobenzaprine in both single dose and multiple dose studies, consistent with bypassing first pass hepatic metabolism. At steady state after 20 days of dosing TNX-102 SL, the dynamic peak level of cyclobenzaprine is higher than the background level of norcyclobenzaprine. In contrast, after 20 days of dosing oral cyclobenzaprine, the simulated peak level of cyclobenzaprine is lower than the simulated background level of norcyclobenzaprine.
1Fitzcharles MA, et al. Lancet. 2021;397:2098-110
2Clauw DJ. Ann Rheum Dis. Published Online First: 2024
3Kaplan CM, et al. Nat Rev Neurol. 2024;20, 347–363
Tonix Pharmaceuticals Holding Corp.*
Tonix is a fully-integrated biopharmaceutical company focused on transforming therapies for pain management and vaccines for public health challenges. Tonix’s development portfolio is focused on central nervous system (CNS) disorders. Tonix’s priority is to advance TNX-102 SL, a product candidate for the management of fibromyalgia, for which an NDA was submitted based on two statistically significant Phase 3 studies for the management of fibromyalgia and for which a PDUFA (Prescription Drug User Fee act) goal date of August 15, 2025 has been assigned for a decision on marketing authorization. The FDA has also granted Fast Track designation to TNX-102 SL for the management of fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction and acute stress disorder under a Physician-Initiated IND at the University of North Carolina in the OASIS study funded by the U.S. Department of Defense (DoD). Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase), a biologic in Phase 2 development designed to treat cocaine intoxication that has FDA Breakthrough Therapy designation, and its development is supported by a grant from the National Institute on Drug Abuse. Tonix’s immunology development portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is an Fc-modified humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft rejection and for the treatment of autoimmune diseases. TNX-1500 has completed a positive Phase I trial. Tonix’s infectious disease portfolio includes TNX-801, a vaccine in development for mpox and smallpox, as well as TNX-4200 for which Tonix has a contract with the U.S. DoD’s Defense Threat Reduction Agency (DTRA) for up to $34 million over five years. TNX-4200 is a small molecule broad-spectrum antiviral agent targeting CD45 for the prevention or treatment of infections to improve the medical readiness of military personnel in biological threat environments. Tonix owns and operates a state-of-the art infectious disease research facility in Frederick, Md. Tonix Medicines, our commercial subsidiary, markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan nasal spray) 10 mg for the treatment of acute migraine with or without aura in adults.
* Tonix’s product development candidates are investigational new drugs or biologics; 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. All other marks are property of their respective owners.
This press release and further information about Tonix can be found at www.tonixpharma.com.
Forward Looking Statements
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as “anticipate,” “believe,” “forecast,” “estimate,” “expect,” and “intend,” among others. These forward-looking statements are based on Tonix’s current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, risks related to the failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; risks related to the failure to successfully market any of our products; risks related to the timing and progress of clinical development of our product candidates; our need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party payor reimbursement; limited research and development efforts and dependence upon third parties; and substantial competition. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Tonix does not undertake an obligation to update or revise any forward-looking statement. Investors should read the risk factors set forth in the Annual Report on Form 10-K for the year ended December 31, 2024, as filed with the Securities and Exchange Commission (the “SEC”) on March 18, 2025, and periodic reports filed with the SEC on or after the date thereof. All of Tonix’s forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date thereof.
Zembrace® SymTouch® (sumatriptan succinate) injection (Zembrace) and Tosymra® (sumatriptan) nasal spray are prescription medicines used to treat acute migraine headaches with or without aura in adults who have been diagnosed with migraine.
Zembrace and Tosymra are not used to prevent migraines. It is not known if Zembrace or Tosymra are safe and effective in children under 18 years of age.
Important Safety Information
Zembrace and Tosymra can cause serious side effects, including heart attack and other heart problems, which may lead to death. Stop use and get emergency help if you have any signs of a heart attack:
discomfort in the center of your chest that lasts for more than a few minutes or goes away and comes back
severe tightness, pain, pressure, or heaviness in your chest, throat, neck, or jaw
pain or discomfort in your arms, back, neck, jaw or stomach
shortness of breath with or without chest discomfort
breaking out in a cold sweat
nausea or vomiting
feeling lightheaded
Zembrace and Tosymra are not for people with risk factors for heart disease (high blood pressure or cholesterol, smoking, overweight, diabetes, family history of heart disease) unless a heart exam shows no problem.
Do not use Zembrace or Tosymra if you have:
history of heart problems
narrowing of blood vessels to your legs, arms, stomach, or kidney (peripheral vascular disease)
uncontrolled high blood pressure
hemiplegic or basilar migraines. If you are not sure if you have these, ask your provider.
had a stroke, transient ischemic attacks (TIAs), or problems with blood circulation
severe liver problems
taken any of the following medicines in the last 24 hours: almotriptan, eletriptan, frovatriptan, naratriptan, rizatriptan, ergotamines, or dihydroergotamine. Ask your provider for a list of these medicines if you are not sure.
are taking certain antidepressants, known as monoamine oxidase (MAO)-A inhibitors or it has been 2 weeks or less since you stopped taking a MAO-A inhibitor. Ask your provider for a list of these medicines if you are not sure.
an allergy to sumatriptan or any of the components of Zembrace or Tosymra
Tell your provider about all of your medical conditions and medicines you take, including vitamins and supplements.
Zembrace and Tosymra can cause dizziness, weakness, or drowsiness. If so, do not drive a car, use machinery, or do anything where you need to be alert.
Zembrace and Tosymra may cause serious side effects including:
changes in color or sensation in your fingers and toes
sudden or severe stomach pain, stomach pain after meals, weight loss, nausea or vomiting, constipation or diarrhea, bloody diarrhea, fever
cramping and pain in your legs or hips; feeling of heaviness or tightness in your leg muscles; burning or aching pain in your feet or toes while resting; numbness, tingling, or weakness in your legs; cold feeling or color changes in one or both legs or feet
increased blood pressure including a sudden severe increase even if you have no history of high blood pressure
medication overuse headaches from using migraine medicine for 10 or more days each month. If your headaches get worse, call your provider.
serotonin syndrome, a rare but serious problem that can happen in people using Zembrace or Tosymra, especially when used with anti-depressant medicines called SSRIs or SNRIs. Call your provider right away if you have: mental changes such as seeing things that are not there (hallucinations), agitation, or coma; fast heartbeat; changes in blood pressure; high body temperature; tight muscles; or trouble walking.
hives (itchy bumps); swelling of your tongue, mouth, or throat
seizures even in people who have never had seizures before
The most common side effects of Zembrace and Tosymra include: pain and redness at injection site (Zembrace only); tingling or numbness in your fingers or toes; dizziness; warm, hot, burning feeling to your face (flushing); discomfort or stiffness in your neck; feeling weak, drowsy, or tired; application site (nasal) reactions (Tosymra only) and throat irritation (Tosymra only).
Tell your provider if you have any side effect that bothers you or does not go away. These are not all the possible side effects of Zembrace and Tosymra. For more information, ask your provider.
This is the most important information to know about Zembrace and Tosymra but is not comprehensive. For more information, talk to your provider and read the Patient Information and Instructions for Use. You can also visit https://www.tonixpharma.com or call 1-888-869-7633.
You are encouraged to report adverse effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
Tegoprubart used as a key component of immunosuppression regimen in the second transplant of a genetically modified pig kidney into a human conducted at Massachusetts General Hospital
Announced positive initial data from first three subjects with type 1 diabetes treated with tegoprubart as part of immunosuppression regimen following islet transplantation in investigator-initiated trial at UChicago Medicine
Topline results from Phase 2 BESTOW trial of tegoprubart in kidney transplantation expected in fourth quarter of 2025
Proceeds from oversubscribed $85 million underwritten offering extend cash runway to end of 2026
IRVINE, Calif., March 20, 2025 (GLOBE NEWSWIRE) — Eledon Pharmaceuticals, Inc. (“Eledon”) (Nasdaq: ELDN) today reported its fourth quarter and full year 2024 operating and financial results and reviewed recent business highlights.
“We have recently made great strides in expanding our role in bringing new options in organ transplantation to patients. Tegoprubart was a cornerstone immunosuppression component in recent historic procedures including kidney xenotransplant and islet transplants in patients with type 1 diabetes,” said David-Alexandre C. Gros, M.D., Chief Executive Officer of Eledon. “The results from these landmark studies together with the encouraging allograft kidney transplant clinical data we have shared continue to reinforce tegoprubart’s broad potential to protect transplanted organs and cells, regardless of the transplant type and the organ source. We are entering 2025 from a position of balance sheet strength and we are on track to deliver on multiple key milestones in the coming months, including topline results from our Phase 2 BESTOW trial in the fourth quarter of 2025.”
Fourth Quarter 2024 and Recent Business Highlights
Announced the use of tegoprubart as a lead component of the immunosuppression treatment regimen following the second transplant of a genetically modified pig kidney into a human in a study conducted at Massachusetts General Hospital. Following the successful transplant on January 25, 2025, the patient was discharged from the hospital without need for continued treatment with dialysis for the first time in over two years.
Reported positive initial data for the first three islet transplant recipients treated with tegoprubart as part of an immunosuppression regimen for the prevention of islet transplant rejection in subjects with type 1 diabetes in an investigator-initiated trial at the University of Chicago Medicine’s Transplant Institute. The data demonstrated potentially the first in human cases of insulin independence achieved using an anti-CD40L monoclonal antibody immunosuppression therapy without the use of tacrolimus, the current standard for care for prevention of transplant rejection.
Completed an oversubscribed, underwritten offering of common stock and pre-funded warrants for total gross proceeds of $85.0 million and net proceeds of approximately $79.5 million after deducting underwriting discounts, commissions, and offering expenses. The offering, which priced at a premium, included participation from both new and existing investors.
Anticipated Upcoming Milestones
Summer 2025: Report updated interim clinical data from the ongoing Phase 1b open-label trial evaluating tegoprubart for the prevention of organ rejection in kidney transplant patients.
4Q 2025: Report topline results from the Phase 2 BESTOW trial of tegoprubart in kidney transplantation.
2025: Report updated interim clinical data from the investigator-led clinical trial with UChicago Medicine for pancreatic islet transplantation in subjects with type 1 diabetes.
Fourth Quarter 2024 Financial Results
Cash, cash equivalents and short-term investments totaled $140.2 million as of December 31, 2024 compared to $78.2 million at September 30, 2024. The company expects current cash, cash equivalents and short-term investments to fund operations to the end of 2026.
Research and development (R&D) expenses for the fourth quarter of 2024 were $17.9 million, including $2.7 million of non-cash stock-based compensation expense, compared to $7.1 million, including $0.3 million of non-cash stock-based compensation expense, for the comparable period in 2023.
General and administrative expenses for the fourth quarter of 2024 were $6.8 million, including $3.9 million of non-cash stock-based compensation expense, compared to $3.3 million, including $1.4 million of non-cash stock-based compensation expense, for the comparable period in 2023.
Net loss for the fourth quarter of 2024 was $44.6 million, or $0.64 per basic share, compared with a net loss of $30.1 million, or $1.00 per basic share, for the comparable period in 2023. Both periods included a non-cash loss from changes in the fair value of warrant liabilities, totaling $20.9 million in 2024 and $20.5 million in 2023.
Full Year 2024 Financial Results
Research and development (R&D) expenses for the year ended December 31, 2024 were $52.0 million, including $4.3 million of non-cash stock-based compensation expense, compared to $30.3 million, including $1.5 million of non-cash stock-based compensation expense, for the comparable period in 2023. The increase was primarily driven by a rise in clinical development expenses related to the Phase 1b, Phase 2 BESTOW and Phase 2 open-label extension trials for kidney transplantation, an increase in manufacturing costs for drug substance and drug product clinical trial supply, an increase in stock-based compensation expense and employee compensation and benefits related to increased headcount.
General and administrative expenses for the year ended December 31, 2024 were $18.6 million, including $8.8 million of non-cash stock-based compensation expense, compared to $12.7 million, including $5.0 million of non-cash stock-based compensation expense, for the comparable period in 2023. The increase was primarily driven by an increase in stock-based compensation expense, professional services and employee compensation and benefits.
Net loss for the year ended December 31, 2024 was $36.2 million, or $0.75 per basic share, compared with a net loss of $116.5 million, or $4.73 per basic share, in 2023. The 2024 net loss included a non-cash gain of $30.9 million from changes in the fair value of warrant liabilities, whereas the 2023 net loss included a non-cash loss of $76.2 million from such changes. Excluding the non-cash items related to changes in the fair value of warrant liabilities, the company would have recorded a net loss of $67.1 million for the year ended December 31, 2024 and a net loss of $40.3 million for the year ended December 31, 2023.
About Eledon Pharmaceuticals and tegoprubart
Eledon Pharmaceuticals, Inc. is a clinical stage biotechnology company that is developing immune-modulating therapies for the management and treatment of life-threatening conditions. The Company’s lead investigational product is tegoprubart, an anti-CD40L antibody with high affinity for the CD40 Ligand, a well-validated biological target that has broad therapeutic potential. The central role of CD40L signaling in both adaptive and innate immune cell activation and function positions it as an attractive target for non-lymphocyte depleting, immunomodulatory therapeutic intervention. The Company is building upon a deep historical knowledge of anti-CD40 Ligand biology to conduct preclinical and clinical studies in kidney allograft transplantation, xenotransplantation, and amyotrophic lateral sclerosis (ALS). Eledon is headquartered in Irvine, California. For more information, please visit the Company’s website at www.eledon.com.
Follow Eledon Pharmaceuticals on social media: LinkedIn; Twitter
Forward Looking Statements
This press release contains forward-looking statements that involve substantial risks and uncertainties. Any statements about the company’s future expectations, plans and prospects, including statements about planned clinical trials, the development of product candidates, expected timing for initiation of future clinical trials, expected timing for receipt of data from clinical trials, the company’s capital resources and ability to finance planned clinical trials, as well as other statements containing the words “believes,” “anticipates,” “plans,” “expects,” “estimates,” “intends,” “predicts,” “projects,” “targets,” “looks forward,” “could,” “may,” and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements are inherently uncertain and are subject to numerous risks and uncertainties, including: risks relating to the safety and efficacy of our drug candidates; risks relating to clinical development timelines, including interactions with regulators and clinical sites, as well as patient enrollment; risks relating to costs of clinical trials and the sufficiency of the company’s capital resources to fund planned clinical trials; and risks associated with the impact of the ongoing coronavirus pandemic. Actual results may differ materially from those indicated by such forward-looking statements as a result of various factors. These risks and uncertainties, as well as other risks and uncertainties that could cause the company’s actual results to differ significantly from the forward-looking statements contained herein, are discussed in our quarterly 10-Q, annual 10-K, and other filings with the U.S. Securities and Exchange Commission, which can be found at www.sec.gov. Any forward-looking statements contained in this press release speak only as of the date hereof and not of any future date, and the company expressly disclaims any intent to update any forward-looking statements, whether as a result of new information, future events or otherwise.
Novel THIO dimer shows promise as a new compound with a dual mechanism of action for enhancing standard cancer treatments and overcoming resistance
CHICAGO–(BUSINESS WIRE)– MAIA Biotechnology, Inc., (NYSE American: MAIA) (“MAIA”, the “Company”), a clinical-stage biopharmaceutical company developing targeted immunotherapies for cancer, today announces the publication of preclinical data for its lead proprietary telomere-targeting THIO dimer in the peer-reviewed scientific journal Naunyn-Schmiedeberg’s Archives of Pharmacology.
In a preclinical study, THIO and its new described dimer form were found to be potent inhibitors of Glutathione S-transferase Pi (GSTP1), a key enzyme implicated in cancer progression and chemoresistance and a highly important factor for the detoxification of cancer cells. The findings suggest that the dimerized form of THIO could enhance chemotherapeutic efficacy by effectively targeting GSTP1 and reducing drug resistance. The article, titled “Investigation of the inhibitory effects of the telomere-targeted compounds on glutathione S-transferase P1,” was published on February 15, 2025.
“The esteemed Archives of Pharmacology has published our first peer-reviewed paper about the unique potential of the lead molecule in our second-generation THIO program,” said Vlad Vitoc, M.D., CEO of MAIA. “Preclinical findings illuminate the superior GSTP1 binding affinity and inhibitory potency of this novel prodrug and support continued development of this new strategy for cancer therapy.”
MAIA’s second generation research and discovery platform seeks to identify new telomere-targeting THIO-like compounds with potentially improved specificity towards cancer cells relative to normal cells and with potentially increased anticancer activity. More than 80 THIO-like compounds have been developed as part of the second-generation telomere targeting program.
“Our manuscript highlights the potential of THIO’s dimer as a potent GSTP1 inhibitor and a promising new strategy for enhancing cancer treatment and overcoming drug resistance,” said Chief Scientific Officer Sergei Gryaznov, Ph.D. “Further exploration of the combinatorial effects of THIO with standard chemotherapeutic agents could provide valuable insights for optimizing standard cancer treatment protocols. These efforts could pave the way for novel, targeted strategies in cancer therapy, offering new hope in the fight against drug-resistant cancers.”
About Naunyn–Schmiedeberg’s Archives of Pharmacology
Naunyn–Schmiedeberg’s Archives of Pharmacology, founded in 1873, is the oldest existing pharmacological journal and a dedicated platform for new and significant information on drug action and toxicity of chemical compounds. The peer-reviewed scientific journal covers all fields of experimental and clinical pharmacology as well as toxicology and includes studies in neuropharmacology and cardiovascular pharmacology and those describing drug actions at the cellular, biochemical and molecular levels.
About Ateganosine
Ateganosine (THIO, 6-thio-dG or 6-thio-2’-deoxyguanosine) is a first-in-class investigational telomere-targeting agent currently in clinical development to evaluate its activity in Non-Small Cell Lung Cancer (NSCLC). Telomeres, along with the enzyme telomerase, play a fundamental role in the survival of cancer cells and their resistance to current therapies. The modified nucleotide 6-thio-2’-deoxyguanosine induces telomerase-dependent telomeric DNA modification, DNA damage responses, and selective cancer cell death. Ateganosine-damaged telomeric fragments accumulate in cytosolic micronuclei and activates both innate (cGAS/STING) and adaptive (T-cell) immune responses. The sequential treatment with ateganosine followed by PD-(L)1 inhibitors resulted in profound and persistent tumor regression in advanced, in vivo cancer models by induction of cancer type–specific immune memory. Ateganosine is presently developed as a second or later line of treatment for NSCLC for patients that have progressed beyond the standard-of-care regimen of existing checkpoint inhibitors.
About MAIA Biotechnology, Inc.
MAIA is a targeted therapy, immuno-oncology company focused on the development and commercialization of potential first-in-class drugs with novel mechanisms of action that are intended to meaningfully improve and extend the lives of people with cancer. Our lead program is ateganosine, a potential first-in-class cancer telomere targeting agent in clinical development for the treatment of NSCLC patients with telomerase-positive cancer cells. For more information, please visit www.maiabiotech.com.
Forward Looking Statements
MAIA cautions that all statements, other than statements of historical facts contained in this press release, are forward-looking statements. Forward-looking statements are subject to known and unknown risks, uncertainties, and other factors that may cause our or our industry’s actual results, levels or activity, performance or achievements to be materially different from those anticipated by such statements. The use of words such as “may,” “might,” “will,” “should,” “could,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify forward looking statements. However, the absence of these words does not mean that statements are not forward-looking. For example, all statements we make regarding (i) the initiation, timing, cost, progress and results of our preclinical and clinical studies and our research and development programs, (ii) our ability to advance product candidates into, and successfully complete, clinical studies, (iii) the timing or likelihood of regulatory filings and approvals, (iv) our ability to develop, manufacture and commercialize our product candidates and to improve the manufacturing process, (v) the rate and degree of market acceptance of our product candidates, (vi) the size and growth potential of the markets for our product candidates and our ability to serve those markets, and (vii) our expectations regarding our ability to obtain and maintain intellectual property protection for our product candidates, are forward looking. All forward-looking statements are based on current estimates, assumptions and expectations by our management that, although we believe to be reasonable, are inherently uncertain. Any forward-looking statement expressing an expectation or belief as to future events is expressed in good faith and believed to be reasonable at the time such forward-looking statement is made. However, these statements are not guarantees of future events and are subject to risks and uncertainties and other factors beyond our control that may cause actual results to differ materially from those expressed in any forward-looking statement. Any forward-looking statement speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. In this release, unless the context requires otherwise, “MAIA,” “Company,” “we,” “our,” and “us” refers to MAIA Biotechnology, Inc. and its subsidiaries.
Key Points: – FOXO Technologies has signed a non-binding agreement to acquire Vector Biosource, a biospecimen sourcing provider. – Vector is expected to generate $800,000 in revenue in 2025 without additional capital. – The acquisition involves Series D Preferred Stock and milestone-based earnout payments.
FOXO Technologies Inc. (NYSE American: FOXO) has announced the execution of a non-binding agreement to acquire Vector Biosource Inc., a provider of information and biospecimen sourcing services for the biotechnology, clinical research, and pharmaceutical industries. The acquisition aligns with FOXO’s strategy of expanding its footprint in healthcare and biotechnology sectors.
The proposed transaction includes an initial payment of $750,000 in Series D Cumulative Redeemable Preferred Stock, with an additional $750,000 in Series D Preferred Stock contingent on Vector meeting specific revenue and cash collection milestones in 2025. Further earnout payments in Series D Preferred Stock are structured based on Vector’s performance in 2026 and 2027. The deal remains subject to definitive agreements, due diligence, and the provision of $1 million in working capital.
Seamus Lagan, CEO of FOXO Technologies, emphasized the strategic benefits of the deal, stating, “We are excited to have reached agreement with Vector to move forward with this strategic acquisition. We were attracted to Vector’s unique position in this healthcare sector and its growth profile, and we are focused on working closely with Vector senior leadership to aggressively expand the Vector platform.”
Vector’s CEO, Frank Dias, Jr., highlighted the advantages of the partnership, noting, “We believe the partnership with FOXO will allow Vector to achieve its near and long-term growth plans by providing growth capital, corporate infrastructure, and potential synergies with other FOXO subsidiaries. We anticipate a significant increase in expected revenues with the provision of growth capital and corporate infrastructure by FOXO.”
FOXO Technologies operates through three subsidiaries:
Rennova Community Health, Inc.: Owner and operator of Scott County Community Hospital (Big South Fork Medical), a critical access hospital in East Tennessee.
Myrtle Recovery Centers, Inc.: A 30-bed behavioral health facility offering inpatient detox, residential treatment, and outpatient services.
Foxo Labs, Inc.: A biotechnology company dedicated to advancing health and lifespan through innovative technology and product solutions.
The acquisition of Vector Biosource marks another step in FOXO’s broader growth strategy as it continues to integrate specialized healthcare and biotechnology services under its corporate umbrella. The deal is expected to close within the next 45 days, subject to regulatory approvals and standard closing conditions.
Nonproprietary drug name approval marks essential step in FDA approval process
CHICAGO–(BUSINESS WIRE)– MAIA Biotechnology, Inc., (NYSE American: MAIA) (“MAIA”, the “Company”), a clinical-stage biopharmaceutical company developing targeted immunotherapies for cancer, today announced that the United States Adopted Names (USAN) Council has approved “ateganosine” as the nonproprietary (generic) name for its lead molecule THIO, a telomere-targeting anticancer agent in clinical development as a first-in-class treatment for advanced non-small cell lung cancer (NSCLC).
The USAN Council is responsible for selecting standardized, informative and unique nonproprietary (generic) drug names. It is made up of experts from the American Medical Association (AMA), the U.S. Pharmacopeial Convention (USP) and the U.S Food and Drug Administration (FDA).
“The designation of a new nonproprietary name for THIO is a key step along our development and regulatory pathway as we move forward with Phase 2 and 3 clinical trials,” said Vlad Vitoc, M.D., CEO of MAIA. “We chose a name inspired by the mechanism of action of our molecule: altering telomeric guanosine of the cancer cells. The generic name ateganosine is a unique and consistent identity that will support clear communication between healthcare providers, patients and researchers.”
Generic drug names are used in product information, drug regulation, labelling and prescribing as for promotional materials and scientific literature.
MAIA will retain the name THIO in its clinical trial designations (THIO-101, THIO-102, THIO-103, THIO-104).
About Ateganosine
Ateganosine (THIO, 6-thio-dG or 6-thio-2’-deoxyguanosine) is a first-in-class investigational telomere-targeting agent currently in clinical development to evaluate its activity in Non-Small Cell Lung Cancer (NSCLC). Telomeres, along with the enzyme telomerase, play a fundamental role in the survival of cancer cells and their resistance to current therapies. The modified nucleotide 6-thio-2’-deoxyguanosine induces telomerase-dependent telomeric DNA modification, DNA damage responses, and selective cancer cell death. Ateganosine-damaged telomeric fragments accumulate in cytosolic micronuclei and activates both innate (cGAS/STING) and adaptive (T-cell) immune responses. The sequential treatment with ateganosine followed by PD-(L)1 inhibitors resulted in profound and persistent tumor regression in advanced, in vivo cancer models by induction of cancer type–specific immune memory. Ateganosine is presently developed as a second or later line of treatment for NSCLC for patients that have progressed beyond the standard-of-care regimen of existing checkpoint inhibitors.
About MAIA Biotechnology, Inc.
MAIA is a targeted therapy, immuno-oncology company focused on the development and commercialization of potential first-in-class drugs with novel mechanisms of action that are intended to meaningfully improve and extend the lives of people with cancer. Our lead program is ateganosine, a potential first-in-class cancer telomere targeting agent in clinical development for the treatment of NSCLC patients with telomerase-positive cancer cells. For more information, please visit www.maiabiotech.com.
Forward Looking Statements
MAIA cautions that all statements, other than statements of historical facts contained in this press release, are forward-looking statements. Forward-looking statements are subject to known and unknown risks, uncertainties, and other factors that may cause our or our industry’s actual results, levels or activity, performance or achievements to be materially different from those anticipated by such statements. The use of words such as “may,” “might,” “will,” “should,” “could,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,” “future,” “potential,” or “continue,” and other similar expressions are intended to identify forward looking statements. However, the absence of these words does not mean that statements are not forward-looking. For example, all statements we make regarding (i) the initiation, timing, cost, progress and results of our preclinical and clinical studies and our research and development programs, (ii) our ability to advance product candidates into, and successfully complete, clinical studies, (iii) the timing or likelihood of regulatory filings and approvals, (iv) our ability to develop, manufacture and commercialize our product candidates and to improve the manufacturing process, (v) the rate and degree of market acceptance of our product candidates, (vi) the size and growth potential of the markets for our product candidates and our ability to serve those markets, and (vii) our expectations regarding our ability to obtain and maintain intellectual property protection for our product candidates, are forward looking. All forward-looking statements are based on current estimates, assumptions and expectations by our management that, although we believe to be reasonable, are inherently uncertain. Any forward-looking statement expressing an expectation or belief as to future events is expressed in good faith and believed to be reasonable at the time such forward-looking statement is made. However, these statements are not guarantees of future events and are subject to risks and uncertainties and other factors beyond our control that may cause actual results to differ materially from those expressed in any forward-looking statement. Any forward-looking statement speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law. In this release, unless the context requires otherwise, “MAIA,” “Company,” “we,” “our,” and “us” refers to MAIA Biotechnology, Inc. and its subsidiaries.
August 15, 2025, is the FDA PDUFA goal date for TNX-102 SL for the management of fibromyalgia; If approved, TNX-102 SL would become the first new drug for treating fibromyalgia in more than 15 years
Company expects to have sufficient cash to fund planned operations beyond the FDA PDUFA goal date and anticipated fourth quarter 2025 launch of TNX-102 SL for fibromyalgia; $98.8 million in cash as of December 31, 2024
Announced positive topline results from Phase 1 study of TNX-1500, a next generation anti-CD40L mAb candidate for prevention of kidney transplant rejection and treatment of autoimmune diseases
Received government grant for potential mpox vaccine, TNX-801, which has demonstrated single-dose immune protection against a monkeypox challenge in non-human primates
Received first payments from U.S. Department of Defense (DoD) contract for up to $34 million over five years to develop a broad-spectrum antiviral drug program
CHATHAM, N.J., March 18, 2025 (GLOBE NEWSWIRE) — Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a fully-integrated biopharmaceutical company with marketed products and a pipeline of development candidates, today announced financial results for the fourth quarter and full year ended December 31, 2024, and provided an overview of recent operational highlights.
“With commercial preparations underway, we believe we are well positioned to launch TNX-102 SL for the management of fibromyalgia in the fourth quarter of this year if approved by the U.S. Food and Drug Administration,” said Seth Lederman, M.D., Chief Executive Officer of Tonix. “We believe that TNX-102 SL has the potential to be the first in a new class of non-opioid analgesic medicines for the management of fibromyalgia, and the first new drug for treating fibromyalgia in more than 15 years.”
Fibromyalgia is a debilitating chronic pain condition affecting over 10 million adults in the United States, most of whom are women. Data from our pivotal Phase 3 trials indicate that TNX-102 SL can provide fibromyalgia patients with significant reduction in pain. TNX-102 SL was generally well tolerated and has no known addictive properties.
Dr. Lederman continued, “Tonix is debt free and expects to have sufficient cash to fund operations through the PDUFA target date of August 15, 2025, and the anticipated commercial launch of TNX-102 SL in the fourth quarter of this year. We continue to meaningfully add to our commercial team and are engaged in pre-launch activities. We look forward to continuing discussions with the FDA throughout the review period in advance of the PDUFA goal date to bring patients a potential new treatment option.”
“Beyond TNX-102 SL for fibromyalgia, we are encouraged by the continued development of our pipeline in a capital efficient manner. Positive results from a Phase 1 study evaluating the tolerability and pharmacokinetics of TNX-1500, a next generation anti-CD40L mAb for prevention of kidney transplant rejection, support advancing to a planned Phase 2 trial in kidney transplant recipients with monthly dosing. In addition, we continue to advance TNX-801 vaccine for preventing mpox and smallpox towards the clinic. With the ongoing global mpox epidemic continuing to spread, TNX-801’s ability to protect animals from lethal challenge with Clade Ia monkeypox virus and its tolerability in immune-compromised animals is encouraging and further supports testing in humans. We look forward to providing additional updates to each of these promising programs in 2025.”
Key Product Candidates* — Recent Highlights
Central Nervous System (CNS) Pipeline
TNX-102 SL (cyclobenzaprine HCl sublingual tablets): 5.6 mg, once-daily at bedtime small molecule for the management of fibromyalgia (FM) – a centrally-acting, non-opioid analgesic.
In December 2024, the Company announced U.S. Food and Drug Administration (FDA) acceptance of its New Drug Application (NDA) for TNX-102 SL for fibromyalgia, with a Prescription Drug User Fee Act (PDUFA) goal date of August 15, 2025. The NDA was based upon two Phase 3 studies of TNX-102 SL in fibromyalgia that showed statically significant reduction in the chronic, widespread pain associated with fibromyalgia. It was well tolerated and has no known addictive properties. If approved by the FDA, TNX-102 SL would be the first member of a new class of tertiary amine tricyclic (TAT) non-opioid analgesic drugs for fibromyalgia and the first new drug available for treating fibromyalgia in more than 15 years. Fibromyalgia affects more than 10 million adults in the U.S., most of whom are women.
In March 2025, Tonix presented data and analyses of TNX-102 SL treatment and effects on fibromyalgia at the 7th International Congress on Controversies in Fibromyalgia, held in Vienna, Austria, in an oral presentation titled, “Transmucosal Sublingual Cyclobenzaprine (TNX-102 SL) Treatment of Fibromyalgia at Bedtime to Target Non-Restorative Sleep Showed Durable Pain Reduction in Two Double-Blind Randomized Phase 3 Studies.” TNX-102 SL, a sublingual formulation of cyclobenzaprine designed for transmucosal delivery and durable activity, has demonstrated statistically significant, durable activity (three months) in reducing fibromyalgia pain in two double-blind randomized Phase 3 studies.
In November 2024, at the American College of Rheumatology (ACR) Convergence 2024 Annual Meeting, Tonix announced additional data and analyses of TNX-102 SL for the management of fibromyalgia. TNX-102 SL had met the pre-specified primary endpoint in the Phase 3 RESILIENT study, significantly reducing daily pain compared to placebo (p-value=0.00005) in participants with fibromyalgia with statistically significant improvement in all six pre-specified key secondary endpoints, including those related to improving sleep quality, reducing fatigue, and improving patient global ratings and overall fibromyalgia symptoms and function. TNX-102 SL was well tolerated with an adverse event profile comparable to prior studies and no new safety signals were observed.
In September 2024, at the 11th Global Conference on Pharmaceutics and Novel Drug Delivery Systems (PDDS 2024), the Company announced data highlighting the proprietary formulation technology and pharmacokinetic properties of TNX-102 SL, including composition and methods patents based on the proprietary eutectic formulation of TNX-102 SL that are expected to provide market exclusivity until at least 2034 in the U.S., EU, Japan, China and other jurisdictions. The eutectic protects cyclobenzaprine HCl from interacting with the basifying agent that is also part of the formulation and required for efficient transmucosal absorption. The formulation of TNX-102 SL was designed specifically for sublingual administration and transmucosal absorption for bedtime dosing to target disturbed sleep, relieve pain and other fibromyalgia symptoms, while reducing the risk of daytime somnolence.
TNX-102 SL for the treatment of acute stress reaction (ASR) and acute stress disorder (ASD), and prophylaxis against development of posttraumatic stress disorder (PTSD)
The DoD-funded Optimizing Acute Stress Reaction Interventions (OASIS) trial will be conducted by the University of North Carolina under an investigator-initiated investigational new drug (IND) application. The OASIS trial will examine the safety and efficacy of TNX-102 SL to reduce adverse posttraumatic neuropsychiatric sequelae among patients in the emergency department (ED) after a motor vehicle collision. Fourteen days of bedtime TNX-102 SL will be dosed and tested in the immediate aftermath of motor vehicle collision. The study will test the potential for TNX-102 SL to target trauma-related sleep disturbance and its ability to facilitate recovery from ASR and to prevent PTSD. The program has the potential to provide military personnel with a new treatment option that improves warfighter performance and resilience when administered in the early aftermath of a traumatic event, The study is expected to be initiated in the first half of 2025.
TNX-1300 (recombinant double mutant cocaine esterase): biologic for cocaine intoxication
The National Institutes of Health (NIH)’s National Institute of Drug Abuse (NIDA) previously awarded Tonix a Cooperative Agreement grant for approximately $5 million to support development of TNX-1300.
TNX-1300 has been granted Breakthrough Therapy designation by the FDA.
The Phase 2 CATALYST study of TNX-1300 for the treatment of cocaine intoxication began enrolling in August 2024. CATALYST is a Phase 2 single-blind, placebo-controlled, proof-of-concept study in patients presenting to the emergency department. Because of the challenges of recruiting eligible patients into this study, we are not guiding to a timeline for completion of enrollment or topline data.
Immunology Pipeline
TNX-1500 (anti-CD40L Fc-modified humanized monoclonal antibody): third generation anti-CD40L monoclonal antibody for prophylaxis for organ transplant rejection and treatment of autoimmune disorders.
In February 2025, Tonix announced positive topline results from its Phase 1, single ascending dose (SAD) first-in-human trial of TNX-1500 in healthy participants. The objectives of the Phase 1 trial were to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of intravenous TNX-1500, as well as to support dosing in a planned Phase 2 trial in kidney transplant recipients, pending alignment with the FDA. All objectives were met and support proceeding to a Phase 2 trial. TNX-1500 blocked the primary and secondary antibody responses to a test antigen at the 10 mg/kg and 30 mg/kg i.v. doses, showed mean half-life of 34-38 days for the 10 mg/kg and 30 mg/kg doses (supporting monthly dosing for future efficacy trials) and was generally well-tolerated with a favorable safety profile.
The first proposed indication for TNX-1500 is prophylaxis of organ rejection in adult patients receiving a kidney transplant; but multiple additional indications are possible, including the treatment of autoimmune diseases. Preclinical studies have shown that TNX-1500 maintains the activity of first-generation monoclonal antibodies (mAbs), yet with reduced risk of thrombotic complications. Pharmacokinetic data support a monthly i.v. dosing regimen. This analysis together with TNX-1500’s activity and tolerability in animals, suggests that the protein engineering of TNX-1500’s Fc region has achieved its design goals.
Infectious Disease Pipeline
TNX-801 (recombinant horsepox virus, minimally replicative live vaccine): potential vaccine to protect against mpox and smallpox.
In March 2025, Tonix announced it was awarded a grant from the Medical CBRN Defense Consortium (MCDC) to support the development of TNX-801. The grant will allow Tonix to develop a commercialization plan for TNX-801.
In November 2024, Tonix announced that it has entered into a sponsored research agreement with the Kenya Medical Research Institute (KEMRI) to design, plan and seek regulatory approval for a Phase I clinical study in Kenya to test the safety, tolerability, and immunogenicity of TNX-801 (horsepox, live virus) as a vaccine to prevent mpox and smallpox. Tonix is expected to be the sponsor and KEMRI is expected to lead the execution of the proposed clinical trial.
In November 2024, the Company announced the publication of a peer-reviewed paper highlighting the tolerability of TNX-801 in immune-compromised animals. The publication describes data in which TNX-801 was compared with older vaccinia vaccine strains used in the eradication of smallpox for tolerability in both in vitro and in vivo models. Together, TNX-801 was shown to be greater than 10- to 1,000-fold less virulent (or more attenuated) compared to the older vaccinia smallpox vaccines. Previously, single-dose vaccination with TNX-801 was shown to protect animals from a lethal challenge with Clade Ia monkeypox.
In September 2024, at the DoD’s MHSRS conference and in October 2024 at the World Vaccine Congress in Barcelona, Spain, Tonix presented new data on potential mpox vaccine, TNX-801, demonstrating tolerability and no evidence of spreading to blood or tissues, even at high doses, in immunocompromised animals. TNX-801 is a minimally replicative live-virus vaccine based on synthesized horsepox that has been shown to provide single-dose immune protection against a monkeypox challenge. After a single-dose vaccination, TNX-801 prevented clinical disease and lesions, and also decreased shedding in the mouth and lungs of non-human primates after a lethal challenge with Clade Ia monkeypox. These findings are consistent with TNX-801 inducing mucosal immunity and suggest TNX-801 has the ability to block forward transmission.
In September 2024, the Company announced that the World Health Organization’s (WHO) preferred target product profile (TPP) aligns with the characteristics of TNX-801. Key elements of the WHO draft TPP include single-dose, durable protection, administration without special equipment, and stability at ambient temperature. Other potential beneficial characteristics include the ability to limit forward transmission, use in case-contact vaccination strategies and suitability for use in immunocompromised individuals. In August 2024, the WHO determined that the upsurge of mpox in a growing number of countries in Africa constitutes a public health emergency of international concern (PHEIC), the second such declaration in the past two years in response to transmission of the virus. Mpox cases of the new Clade Ib mpox have since also been detected in many countries outside of Africa including at least three cases in the U.S.
Corporate and Partnerships – Recent Highlights
In February 2025, the Company announced the promotion of Siobhan Fogarty to Chief Technical Officer from Executive Vice President, Product Development. Ms. Fogarty originally joined Tonix in 2016 and has over 25 years of experience in pharmaceutical and biotech product development, manufacturing and quality, for both small and large molecules, at notable pharmaceutical and biotech companies.
In January 2025, Tonix announced the appointment of Gary Ainsworth as its new Vice President, Market Access. With over two decades of industry and market access experience, Mr. Ainsworth offers a significant track record of success building market access functions, developing launch-ready access and reimbursement strategies and payer-focused resources, including those for fibromyalgia and migraine treatment options.
In December 2024, the Company announced the expansion of its leadership team with the appointment of two strategic hires: Bradley Raudabaugh, MBA, joined Tonix as Vice President, Marketing, and Errol Gould, Ph.D., joined the company as Vice President, Medical Affairs. Mr. Raudabaugh brings over 25 years of marketing, sales and product planning experience to Tonix and Dr. Gould offers over 25 years of experience in R&D and medical affairs across a wide range of therapeutic areas, including fibromyalgia.
In October 2024, the Company announced the receipt of the first contract payment from the previously awarded contract by the U.S. Department of Defense (DoD) for accelerated development of broad-spectrum antivirals with the Defense Threat Reduction Agency (DTRA). The contract, awarded in July 2024, has the potential for up to $34 million over five years with an objective to develop small molecule broad-spectrum antiviral agents for the prevention or treatment of infections to improve the medical readiness of military personnel in biological threat environments. Tonix’s program is focusing on optimization and development of its TNX-4200 program, to develop an orally available CD45 antagonist, with broad-spectrum efficacy against a range of viral families through preclinical evaluation. The program is expected to establish physicochemical properties, pharmacokinetics, and safety attributes to support an IND submission and to fund a first-in-human Phase 1 clinical study.
In October 2024, the Company announced it entered into an artificial intelligence and machine learning drug discovery collaboration with X-Chem, Inc., a leader in small molecule drug discovery, to accelerate the development of small molecules as orally available host-targeted broad-spectrum medical countermeasures. Tonix’s TNX-4200 antiviral program focuses on the development of oral CD45 phosphatase inhibitors, with broad-spectrum activity against a range of viral families.
In September 2024, Tonix announced the appointment of Thomas Englese as its new Executive Vice President, Commercial Operations. Mr. Englese brings significant leadership to Tonix across several functions, including commercial operations, sales and marketing, and launching and managing major brands through all stages of commercialization.
Marketed Products – Recent Highlights
In September 2024, Tonix announced that the United States Patent and Trademark Office (USPTO) issued U.S. Patent No. 12,097,183 to the Company, claiming use of a pre-filled autoinjector comprising a composition of Zembrace® SymTouch® for treating migraines via subcutaneous administration. This patent, excluding possible patent term extensions, is expected to fortify protection and market exclusivity into 2036.
Tonix announced that the USPTO issued U.S. Patent No. 12,090,139 to the Company, claiming a pharmaceutical composition, a method of treating migraine via intranasal administration, and an intranasal delivery system for Tosymra®. This patent is expected to fortify protection and market exclusivity into 2030.
In September 2024, Tonix Medicines launched a national educational campaign focusing on the link between migraine, gastroparesis, and the need for non-oral acute migraine therapies. Tonix Medicines is the only manufacturer with both a branded injectable and nasal spray indicated for the acute treatment of migraine with or without aura in adults.
Financial – Recent Highlight
Tonix had approximately $98.8 million of cash and cash equivalents as of December 31, 2024, compared to approximately $24.9 million as of December 31, 2023. Net cash used in operations was approximately $60.9 million for the full year ended December 31, 2024, compared to $102.0 million for the same period in 2023. Net cash used in investing activities for the full year ended December 31, 2024, was approximately $0.1 million compared to $29.1 million for the same period in 2023.
Following the repayment of a mortgage (Loan and Guaranty Agreement) with JGB Capital and related parties in February 2025, the Company is now debt-free.
Tonix continues to expect that its cash resources at December 31, 2024, and the net proceeds of approximately $46.3 million raised from the sale of common stock under an at-the-market (ATM) facility in the first quarter of 2025, will be sufficient to fund its planned operations into the first quarter of 2026, beyond the August 15, 2025 PDUFA goal date assigned by the FDA for a decision on marketing authorization for TNX-102 SL for management of fibromyalgia.
Subsequent to December 31, 2024, the Company repurchased 250,000 of its shares of common stock outstanding under the 2024 share repurchase program.
Fourth Quarter 2024 Financial Results
Net product revenue for the fourth quarter 2024 was approximately $2.6 million, compared to $3.8 million for the same period in 2023, and consisted of combined net sales of Zembrace® SymTouch® and Tosymra®. Cost of Sales for the fourth quarter 2024 was approximately $1.2 million, compared to $2.4 million for the same period in 2023.
R&D expenses for the fourth quarter 2024 were approximately $8.3 million, compared to $17.1 million for the same period in 2023. This decrease is predominantly due to decreased clinical expenses resulting from fewer clinical trials and pipeline prioritization efforts.
SG&A expenses for the fourth quarter 2024 were $15.6 million, compared to $11.6 million for the same period in 2023. The increase was primarily due to an increase in financial reporting expenses, sales and marketing, and professional fees associated with TNX-102 SL’s NDA submission.
Net loss available to common stockholders was $22.1 million, or $9.77 per basic and diluted share, for the fourth quarter 2024, compared to net loss available to common stockholders of $27.3 million, or $2,179.83 per basic and diluted share, for the same period in 2023. The basic and diluted weighted average common shares outstanding for the fourth quarter 2024 was 2,263,535 compared to 12,534 shares for the same period in 2023.
Full Year 2024 Financial Results
Net product revenue for the full year 2024 was approximately $10.1 million. Cost of sales for the full year 2024 was approximately $7.8 million.
R&D expenses for the full year 2024 were approximately $40.0 million, compared to $86.7 million in 2023. This decrease is predominantly due to fewer clinical trials and from pipeline prioritization efforts, which further decreased non-clinical, manufacturing, employee-related and professional expenses as well.
SG&A expenses for the full year 2024 were $40.1 million, compared to $34.8 million in 2023. The increase was primarily due to an increase in financial reporting expenses, sales and marketing expenses associated with the Company’s recently acquired marketed products, and professional fees associated with TNX-102 SL’s NDA submission.
Net loss available to common stockholders was $130.0 million, or $176.60 per basic and diluted share, for the full year 2024, compared to net loss available to common stockholders of $116.7 million, or $14,720.25 per basic and diluted share, in 2023. The basic and diluted weighted average common shares outstanding for the full year 2024 was 736,339 compared to 7,925 shares for 2023.
Tonix Pharmaceuticals Holding Corp.*
Tonix is a fully integrated biopharmaceutical company focused on transforming therapies for pain management and vaccines for public health challenges. Tonix’s development portfolio is focused on central nervous system (CNS) disorders. Tonix’s priority is to advance TNX-102 SL, a product candidate for the management of fibromyalgia, for which an NDA was submitted based on two statistically significant Phase 3 studies for the management of fibromyalgia and for which a PDUFA (Prescription Drug User Fee act) goal date of August 15, 2025 has been assigned for a decision on marketing authorization. The FDA has also granted Fast Track designation to TNX-102 SL for the management of fibromyalgia. TNX-102 SL is also being developed to treat acute stress reaction and acute stress disorder under a Physician-Initiated IND at the University of North Carolina in the OASIS study funded by the U.S. Department of Defense (DoD). Tonix’s CNS portfolio includes TNX-1300 (cocaine esterase), a biologic in Phase 2 development designed to treat cocaine intoxication that has FDA Breakthrough Therapy designation, and its development is supported by a grant from the National Institute on Drug Abuse. Tonix’s immunology development portfolio consists of biologics to address organ transplant rejection, autoimmunity and cancer, including TNX-1500, which is an Fc-modified humanized monoclonal antibody targeting CD40-ligand (CD40L or CD154) being developed for the prevention of allograft rejection and for the treatment of autoimmune diseases. Tonix’s infectious disease portfolio includes TNX-801, a vaccine in development for mpox and smallpox, as well as TNX-4200 for which Tonix has a contract with the U.S. DoD’s Defense Threat Reduction Agency (DTRA) for up to $34 million over five years. TNX-4200 is a small molecule broad-spectrum antiviral agent targeting CD45 for the prevention or treatment of infections to improve the medical readiness of military personnel in biological threat environments. Tonix owns and operates a state-of-the art infectious disease research facility in Frederick, Md. Tonix Medicines, our commercial subsidiary, markets Zembrace® SymTouch® (sumatriptan injection) 3 mg and Tosymra® (sumatriptan nasal spray) 10 mg for the treatment of acute migraine with or without aura in adults.
* Tonix’s product development candidates are investigational new drugs or biologics; 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. All other marks are property of their respective owners.
This press release and further information about Tonix can be found at www.tonixpharma.com.
Forward Looking Statements
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking words such as “anticipate,” “believe,” “forecast,” “estimate,” “expect,” and “intend,” among others. These forward-looking statements are based on Tonix’s current expectations and actual results could differ materially. There are a number of factors that could cause actual events to differ materially from those indicated by such forward-looking statements. These factors include, but are not limited to, risks related to the failure to obtain FDA clearances or approvals and noncompliance with FDA regulations; risks related to the failure to successfully market any of our products; risks related to the timing and progress of clinical development of our product candidates; our need for additional financing; uncertainties of patent protection and litigation; uncertainties of government or third party payor reimbursement; limited research and development efforts and dependence upon third parties; and substantial competition. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. Tonix does not undertake an obligation to update or revise any forward-looking statement. Investors should read the risk factors set forth in the Annual Report on Form 10-K for the year ended December 31, 2023, as filed with the Securities and Exchange Commission (the “SEC”) on April 1, 2024, and periodic reports filed with the SEC on or after the date thereof. All of Tonix’s forward-looking statements are expressly qualified by all such risk factors and other cautionary statements. The information set forth herein speaks only as of the date thereof.
1The condensed consolidated balance sheets for the years ended December 31, 2024 and 2023 has been derived from the audited financial statements but do not include all of the information and footnotes required by accounting principles generally accepted in the United States for complete financial statements.
1The condensed consolidated balance sheets for the years ended December 31, 2024 and 2023 has been derived from the audited financial statements but do not include all of the information and footnotes required by accounting principles generally accepted in the United States for complete financial statements.
Zembrace® SymTouch® (sumatriptan succinate) injection (Zembrace) and Tosymra® (sumatriptan) nasal spray are prescription medicines used to treat acute migraine headaches with or without aura in adults who have been diagnosed with migraine.
Zembrace and Tosymra are not used to prevent migraines. It is not known if Zembrace or Tosymra are safe and effective in children under 18 years of age.
Important Safety Information
Zembrace and Tosymra can cause serious side effects, including heart attack and other heart problems, which may lead to death. Stop use and get emergency help if you have any signs of a heart attack:
discomfort in the center of your chest that lasts for more than a few minutes or goes away and comes back
severe tightness, pain, pressure, or heaviness in your chest, throat, neck, or jaw
pain or discomfort in your arms, back, neck, jaw or stomach
shortness of breath with or without chest discomfort
breaking out in a cold sweat
nausea or vomiting
feeling lightheaded
Zembrace and Tosymra are not for people with risk factors for heart disease (high blood pressure or cholesterol, smoking, overweight, diabetes, family history of heart disease) unless a heart exam shows no problem.
Do not use Zembrace or Tosymra if you have:
history of heart problems
narrowing of blood vessels to your legs, arms, stomach, or kidney (peripheral vascular disease)
uncontrolled high blood pressure
hemiplegic or basilar migraines. If you are not sure if you have these, ask your provider.
had a stroke, transient ischemic attacks (TIAs), or problems with blood circulation
severe liver problems
taken any of the following medicines in the last 24 hours: almotriptan, eletriptan, frovatriptan, naratriptan, rizatriptan, ergotamines, or dihydroergotamine. Ask your provider for a list of these medicines if you are not sure.
are taking certain antidepressants, known as monoamine oxidase (MAO)-A inhibitors or it has been 2 weeks or less since you stopped taking a MAO-A inhibitor. Ask your provider for a list of these medicines if you are not sure.
an allergy to sumatriptan or any of the components of Zembrace or Tosymra
Tell your provider about all of your medical conditions and medicines you take, including vitamins and supplements.
Zembrace and Tosymra can cause dizziness, weakness, or drowsiness. If so, do not drive a car, use machinery, or do anything where you need to be alert.
Zembrace and Tosymra may cause serious side effects including:
changes in color or sensation in your fingers and toes
sudden or severe stomach pain, stomach pain after meals, weight loss, nausea or vomiting, constipation or diarrhea, bloody diarrhea, fever
cramping and pain in your legs or hips; feeling of heaviness or tightness in your leg muscles; burning or aching pain in your feet or toes while resting; numbness, tingling, or weakness in your legs; cold feeling or color changes in one or both legs or feet
increased blood pressure including a sudden severe increase even if you have no history of high blood pressure
medication overuse headaches from using migraine medicine for 10 or more days each month. If your headaches get worse, call your provider.
serotonin syndrome, a rare but serious problem that can happen in people using Zembrace or Tosymra, especially when used with anti-depressant medicines called SSRIs or SNRIs. Call your provider right away if you have: mental changes such as seeing things that are not there (hallucinations), agitation, or coma; fast heartbeat; changes in blood pressure; high body temperature; tight muscles; or trouble walking.
hives (itchy bumps); swelling of your tongue, mouth, or throat
seizures even in people who have never had seizures before
The most common side effects of Zembrace and Tosymra include: pain and redness at injection site (Zembrace only); tingling or numbness in your fingers or toes; dizziness; warm, hot, burning feeling to your face (flushing); discomfort or stiffness in your neck; feeling weak, drowsy, or tired; application site (nasal) reactions (Tosymra only) and throat irritation (Tosymra only).
Tell your provider if you have any side effect that bothers you or does not go away. These are not all the possible side effects of Zembrace and Tosymra. For more information, ask your provider.
This is the most important information to know about Zembrace and Tosymra but is not comprehensive. For more information, talk to your provider and read the Patient Information and Instructions for Use. You can also visit https://www.tonixpharma.com or call 1-888-869-7633.
You are encouraged to report adverse effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.
OCU200 has a very favorable safety and tolerability profile
No serious adverse events related to the study drug have been reported
Dosing of the second cohort has been approved
MALVERN, Pa., March 18, 2025 (GLOBE NEWSWIRE) — Ocugen, Inc. (“Ocugen” or the “Company”) (NASDAQ: OCGN), a pioneering biotechnology leader in gene therapies for blindness diseases, today announced that the Data and Safety Monitoring Board (DSMB) for the OCU200 clinical trial recently convened and reviewed safety data following dosing of the first cohort in the dose-escalation portion of the Phase 1 study and approved continuation of dosing in the second cohort. OCU200 is a novel fusion protein consisting of two human proteins, tumstatin and transferrin, with the potential to treat diabetic macular edema (DME).
“OCU200 is given intravitreally,” said Peter Chang, MD, FACS, Co-President and Partner of the Massachusetts Eye Research and Surgery Institution (MERSI). “No serious adverse events related to OCU200 have been reported to date.”
The OCU200 Phase 1 clinical trial is a multicenter, open-label, dose-escalation study to assess drug safety via intravitreal injection in three cohorts: low dose (0.025 mg), medium dose (0.05 mg), and high dose (0.1 mg). All subjects will receive two doses six weeks apart, and patients will be followed for up to 6 months.
“It is encouraging that we have successfully completed dosing in the low dose cohort for OCU200, a novel biologic that has a very favorable safety and tolerability profile,” said Dr. Huma Qamar, Chief Medical Officer at Ocugen. “There remains a considerable unmet medical need for the 30% to 40% of DME patients who do not respond to current anti-VEGF therapies. OCU200 holds the promise of potentially benefiting all DME, diabetic retinopathy (DR), and wet age-related macular degeneration (wet AMD) patients.”
Approximately 12 million people in the United States and 130 million people worldwide are affected by DME, DR, or wet AMD. Patients affected by them share common symptoms, such as blurriness in vision and progressive vision loss, as the diseases progress. The formation of fragile and leaky new blood vessels leads to fluid accumulation in and around the retina, causing damage to vision.
OCU200 has the potential to change the treatment landscape for DME, DR, and wet AMD with its unique mechanism of action, binding the active component—tumstatin—to integrin receptors on active endothelial cells that play a crucial role in disease pathogenesis.
OCU200 brings an innovative biologic candidate to Ocugen’s ophthalmology portfolio targeting blindness diseases. The Company intends to complete the Phase 1 OCU200 clinical trial in the second half of 2025 and to provide preliminary safety and efficacy updates throughout the year.
About OCU200
OCU200 is a recombinant fusion protein that consists of two parts connected by a linker: tumstatin, the active component, acts as an anti-inflammatory, anti-VEGF agent by binding to integrin receptors; and transferrin, which targets the drug to the choroid and retina by binding transferrin receptors on endothelial cells. These features will potentially enable OCU200 to reduce the vascular permeability, inflammation, and neovascularization that drive the pathophysiology of DME, DR, and wet AMD at a significantly lower dose compared to currently approved therapies.
About Ocugen, Inc.
Ocugen, Inc. is a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines that improve health and offer hope for patients across the globe. We are making an impact on patients’ lives through courageous innovation—forging new scientific paths that harness our unique intellectual and human capital. Our breakthrough modifier gene therapy platform has the potential to treat multiple retinal diseases with a single product, and we are advancing research in infectious diseases to support public health and orthopedic diseases to address unmet medical needs. Discover more at www.ocugen.com and follow us on X and LinkedIn.
Cautionary Note on Forward-Looking Statements Thispressreleasecontainsforward-lookingstatementswithinthemeaningofThePrivateSecuritiesLitigationReformActof1995,including,butnot limited to, statements regarding qualitative assessments of available data, potential benefits, expectations for ongoing clinical trials, anticipated regulatory filings and anticipated development timelines,whicharesubjecttorisksanduncertainties.Wemay,insomecases,usetermssuchas “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,butnotlimitedto,therisksthatpreliminary,interimandtop-lineclinicaltrialresultsmaynotbeindicativeof,andmaydifferfrom,finalclinical data;the ability of OCU200 to perform in humans in a manner consistent with nonclinical or preclinical study data;thatunfavorablenewclinicaltrialdatamayemergeinongoingclinicaltrialsorthroughfurtheranalysesofexistingclinicaltrialdata;thatearlier non-clinicalandclinicaldataandtestingofmaynotbepredictiveoftheresultsorsuccessoflaterclinicaltrials;andthatthatclinicaltrialdataare subject to differing interpretations and assessments, including by regulatory authorities.Theseandotherrisksanduncertaintiesaremorefully describedinourperiodicfilingswiththeSecuritiesandExchangeCommission(SEC),includingtheriskfactorsdescribedinthesectionentitled“Risk Factors”inthequarterlyandannualreportsthatwefilewiththeSEC.Anyforward-lookingstatementsthatwemakeinthispressreleasespeakonlyas ofthedateofthispressrelease.Exceptasrequiredbylaw,weassumenoobligationtoupdateforward-lookingstatementscontainedinthispress release whether as a result of new information, future events, or otherwise, after the date of this press release.
Data from pivotal Phase 3 trial in CHB-associated liver fibrosis expected in Q2 2025
Commercial launch in the PRC of generic nintedanib for the treatment of IPF and avatrombopag maleate tablets for the treatment of CLD-associated thrombocytopenia expected in 2025
Initiation of U.S. Phase 2 trial of F351 in MASH-associated liver fibrosis expected in 2025
Full year 2025 total revenue guidance of $118 to $128 million
SAN DIEGO, March 17, 2025 (GLOBE NEWSWIRE) — Gyre Therapeutics (“Gyre”) (Nasdaq: GYRE), a self-sustainable, commercial-stage biotechnology company with clinical development programs focusing on organ fibrosis, today announced financial results for the fourth quarter and full year ended December 31, 2024 and provided a business update.
“2025 is shaping up to be a pivotal year for Gyre across both our commercial-stage and clinical-stage portfolios. We plan to expand and enhance our commercial product offerings through the additions of nintedanib for IPF, SSc-ILD and PF-ILD, as well as avatrombopag for CLD-associated thrombocytopenia and chronic idiopathic thrombocytopenia (“ITP”). Given our proven track record and extensive sales and marketing platform, we are confident in our ability to successfully launch and expand these two products in the PRC,” said Han Ying, Ph.D., Chief Executive Officer of Gyre Therapeutics. “In parallel, we expect to share topline data from our pivotal Phase 3 trial in CHB-associated liver fibrosis in the second quarter of 2025, which will help inform our U.S. Phase 2 proof-of-concept trial of F351 in MASH-associated liver fibrosis.”
Full Year 2024 Business Highlights and Upcoming Milestones
Commercial-Stage Updates
ETUARY (Pirfenidone) sales update: For the year ended December 31, 2024, Gyre Pharmaceuticals generated $105.0 million primarily in sales of ETUARY.
Nintedanib: In May 2024, Gyre Pharmaceuticals executed a comprehensive agreement with Jiangsu Wangao Pharmaceuticals Co., Ltd. to obtain the drug registration certificate for and became the marketing authorization holder of nintedanib, the other product approved for the treatment of treatment of idiopathic pulmonary fibrosis (“IPF”). In addition, it has also been approved for the treatment of SSc-ILD and PF-ILD. Gyre Pharmaceuticals plans to initiate commercialization of the nintedanib product in the PRC in 2025.
Avatrombopag: In June 2024, Gyre Pharmaceuticals received approval from China’s National Medical Products Administration (“NMPA”) for avatrombopag maleate tablets for the treatment of thrombocytopenia associated with chronic liver disease (“CLD”) and chronic idiopathic thrombocytopenia (“ITP”) in adult patients undergoing elective diagnostics procedures or therapy. Gyre Pharmaceuticals plans to begin commercialization of avatrombopag in 2025.
Pipeline Development Updates
F351 (Hydronidone):
All patients completed 52-week pivotal Phase 3 trial in chronic hepatitis B (“CHB”)-associated liver fibrosis in the PRC. In October 2024, Gyre Pharmaceuticals announced the last patient completed the 52-week pivotal Phase 3 trial. The trial is evaluating 248 patients with CHB-associated liver fibrosis in the PRC with a primary endpoint of the reduction of the liver fibrosis score (Ishak Scoring System) by at least one stage after taking F351 in combination with entecavir. Gyre expects to report topline data in the second quarter of 2025.
Plans to initiate a Phase 2 clinical trial in metabolic dysfunction-associated steatohepatitis (“MASH”)-associated liver fibrosis in 2025. Pending the results from the pivotal Phase 3 trial in CHB-associated liver fibrosis, Gyre intends to initiate a Phase 2 proof-of-concept trial in the U.S. to evaluate F351 for the treatment of MASH-associated liver fibrosis in 2025.
F573:
F573 is a caspase inhibitor and a potential Category 1 new drug for the treatment of acute/acute on-chronic liver failure (“ALF/ACLF”). Completion of the Phase 2 clinical trial of F573 as a treatment for ALF/ACLF is expected by the end of 2026.
F230:
F230, a selective endothelin receptor agonist for the treatment of pulmonary arterial hypertension (“PAH”), is expected to begin a Phase 1 trial in 2025.
F528:
F528, a novel anti-inflammation agent with the potential to modify the progression of chronic obstructive pulmonary disease (“COPD”), is undergoing preclinical studies as a potential first-line therapy for the treatment of COPD. Gyre plans to submit an IND application in 2026.
Corporate Updates
In January 2025, appointed Ping Zhang to the Company’s Board of Directors as the lead independent director and member of the Nominating Committee. In addition, Ying Luo, Ph.D., resigned as Chairman and member of the Board of Directors of Gyre and Gyre Pharmaceuticals, Gyre’s majority indirectly owned subsidiary in the People’s Republic of China (“PRC”), to focus on other responsibilities at GNI Group Ltd. Songjiang Ma has been appointed Chairman of the Board of Directors of Gyre Pharmaceuticals.
Financial Results
Cash Position
As of December 31, 2024, Gyre had cash, cash equivalents, short-term and long-term bank deposits of $51.2 million.
Financial Results for the Three Months Ended December 31, 2024
Revenues: Revenues for the three months ended December 31, 2024 were $27.9 million, compared to $27.1 million for the same period in 2023. The $0.8 million increase was primarily driven by a $1.0 million increase in ETUARY’s revenue and a $0.2 million decrease in generic drug revenue. The growth in ETUARY sales was attributed to the active expansion of the IPF treatment market, increased market penetration, and a stronger focus on ETUARY sales. To support future revenue growth, Gyre Pharmaceuticals plans to commercially launch two new products, nintedanib and avatrombopag, in 2025, which will be supported by its extensive sales and marketing platform in the PRC.
Cost of Revenues: For the three months ended December 31, 2024, cost of revenues was $1.2 million, compared to $1.3 million for the same period in 2023. The $0.1 million decrease was primarily driven by a $0.2 million decrease in generic drug cost due to the decrease in sales and a $0.1 million decrease in factory stoppage loss due to factory renovation in 2023, offset by a $0.2 million increase due to the increase of ETUARY’s cost due to the increase in sales.
Selling and Marketing Expense: For the three months ended December 31, 2024, selling and marketing expense was $16.9 million, compared to $16.5 million for the same period in 2023. The increase was primarily driven by a $2.1 million increase in promotion expense and conference expenses, offset by a $1.1 million decrease in selling and marketing payroll costs, a $0.3 million decrease in stock-based compensation expense and a $0.3 million decrease in travel and miscellaneous expenses.
Research and Development Expense: For the three months ended December 31, 2024, research and development expense was $3.7 million, compared to $4.6 million for the same period in 2023. The decrease was primarily driven by a $0.5 million decrease in pre-clinical and clinical research expenses and a $0.5 million decrease in stock-based compensation expense, offset by a $0.1 million increase in miscellaneous expense.
General and Administrative Expense: For the three months ended December 31, 2024, general and administrative expense was $5.5 million, compared to $10.1 million for the same period in 2023. The decrease was primarily driven by a $5.8 million decrease in stock-based compensation cost, offset by a $0.8 million increase in the functional and administrative department’s personnel cost and a $0.4 million increase in professional expense, including legal and consulting fees.
Income (Loss) from Operations: For the three months ended December 31, 2024, income from operations was $0.7 million, compared to $91.1 million loss from operation for the same period in 2023. The increase in income from operations was driven primarily by acquired in-process research and development expense recognized in the fourth quarter of 2023 and there was no such expense in the same period in 2024.
Net Income (Loss): For the three months ended December 31, 2024, net income was $0.6 million, compared to $101.0 million net loss for the same period in 2023.
Non-GAAP Adjusted Net Income: For the three months ended December 31, 2024, non-GAAP adjusted net income was $1.1 million, compared to $2.1 million for the same period in 2023. The decrease was primarily driven by the costs of being a public company for three months in 2024, as compared to two months in 2023.
Financial Results for the Full Year Ended December 31, 2024
Revenues: Revenues for the full year ended December 31, 2024 were $ 105.8 million, compared to $113.5 million for the same period in 2023. The $7.7 million decrease was primarily driven by a $7.1 million decrease in ETUARY’s revenue and a $0.6 million decrease in generic drug revenue as a result of decreased sales volumes. The decrease in ETUARY and generic drug sales volumes was due to fluctuations in the Chinese economy that significantly affected demand for anti-fibrosis drugs and decreasing healthcare spending generally. To support future revenue growth, Gyre plans to commercially launch two new products, nintedanib and avatrombopag, in 2025, which will be supported by Gyre Pharmaceuticals’ extensive sales and marketing platform across the PRC.
Cost of Revenues: For the full year ended December 31, 2024, cost of revenues was $3.9 million, compared to $4.6 million for the same period in 2023. The $0.7 million decrease was primarily driven by a $0.5 million factory stoppage loss due to factory renovation in 2023, which did not occur in 2024, and a $0.2 million decrease due to decreased sales volumes.
Selling and Marketing Expense: For the full year ended December 31, 2024, selling and marketing expense was $57.5 million, compared to $61.2 million for the same period in 2023. The decrease was primarily driven by a $2.4 million decrease in conference costs and promotion expense due to decreased sales activities, a $0.9 million decrease in selling and marketing payroll costs due to the decrease of sales of ETUARY in 2024, a $0.3 million decrease in share base compensation expense, and a $0.1 million decrease in miscellaneous expenses.
Research and Development Expense: For the full year ended December 31, 2024, research and development expense was $12.0 million, compared to $13.8 million for the same period in 2023. The decrease was primarily from Gyre Pharmaceuticals, and was driven by a $0.3 million decrease in materials and utilities, a $1.3 million decrease in pre-clinical research expense due to several research and development projects advancing to the clinical trials stage or reaching the application phase in 2024, and a $0.4 million decrease in staff cost due to reduced headcount, and a $0.5 million decrease in stock-based compensation, related to options being fully vested in 2023, which did not occur in 2024, This overall decrease was partially offset by a 0.7 million increase in general research and development expense from Gyre Therapeutics due to increased consulting fees.
General and Administrative Expense: For the full year ended December 31, 2024, general and administrative expense was $16.1 million, compared to $14.7 million for the same period in 2023. The increase was primarily driven by costs associated with being a public company, including a $1.9 million increase in professional expense, a $2.1 million increase in miscellaneous expenses and a $3.0 million increase in the functional and administrative department’s personnel cost, offset by a $5.6 million decrease in stock-based compensation cost.
Income (loss) from Operations: For the full year ended December 31, 2024, income from operations was $16.2 million, compared to $67.2 million loss for the same period in 2023. The increase in income from operations was driven primarily by acquired in-process research and development expense recognized in 2023 and there was no such expense in the same period in 2024.
Net Income (loss): For the full year ended December 31, 2024, net income was $17.9 million, compared to $85.5 million net loss for the same period in 2023.
Non-GAAP Adjusted Net Income: For the full year ended December 31, 2024, non-GAAP adjusted net income was $16.9 million, compared to $25.4 million for the same period in 2023. The decrease was primarily driven by a $7.7 million decline in revenue and a $1.1 million increase in operating expenses. Despite these changes, the gross profit margin remained consistent.
Full Year 2025 Financial Guidance
For the full year 2025, the Company expects to generate revenues of $118 to $128 million, representing growth of 11.3% to 20.8% over 2024 revenue, primarily driven by the anticipated commercial launches of nintedanib and avatrombopag and sales of ETUARY.
Guidance Range
Total Revenue
$118 to $128 million
Please note the following regarding the total revenue guidance:
Guidance assumes a constant foreign currency exchange rate.
Guidance assumes no significant economic disruption or downturn.
Use of Non-GAAP Financial Measures by Gyre Therapeutics, Inc.
Gyre reports financial results in accordance with accounting principles generally accepted in the United States (“GAAP”). This release presents the financial measure “adjusted net income,” which is not calculated in accordance with GAAP. The most directly comparable GAAP measure for this non-GAAP financial measure is “net income.” Adjusted net income presents Gyre’s results of operations after excluding gain from change in fair value of warrants, stock-based compensation, and provision for income taxes. This is meant to supplement, and not substitute, Gyre’s financial information presented in accordance with GAAP. Adjusted net income as defined by Gyre may not be comparable to similar non-GAAP measures presented by other companies. Management believes that presenting adjusted net income provides investors with additional useful information in evaluating the Gyre’s performance and valuation. See the reconciliation of adjusted net income to net income in the section titled “Reconciliation of GAAP to Non-GAAP Financial Measures” below.
About Hydronidone (F351)
F351 is a structural analogue of the approved anti-fibrotic (IPF) drug Pirfenidone and has been shown to inhibit in vitro both p38γ kinase activity and TGF-β1-induced excessive collagen synthesis in hepatic stellate cells (“HSCs”), which are recognized as critical event in the development and progression of fibrosis in the liver. This is further supported by its anti-proliferative effects on the HSCs in the liver. In vitro anti-fibrotic effects of F351 were also confirmed in several established in vivo models of liver fibrosis such as CCI4-induced liver fibrosis mouse model, DMN-induced liver fibrosis rat model, and HSA-induced liver rat model, as well as mouse model of MASH fibrosis (CCI4+Western High Fat Diet).
About Gyre Pharmaceuticals
Gyre Pharmaceuticals is a commercial-stage biopharmaceutical company committed to the research, development, manufacturing and commercialization of innovative drugs for organ fibrosis. Its flagship product, ETUARY® (Pirfenidone capsule), was the first approved treatment for IPF in the PRC in 2011 and has maintained a prominent market share (2024 net sales of $105.0 million). In addition, Gyre Pharmaceuticals is evaluating F351 in a Phase 3 clinical trial in CHB-associated liver fibrosis in the PRC, which is expected to readout topline data by Q2 2025. F351 received Breakthrough Therapy designation by the NMPA Center for Drug Evaluation in March 2021. Gyre Pharmaceuticals is also developing treatments for PD, DKD, COPD, PAH and ALF/ACLF. In October 2023, Gyre Therapeutics acquired an indirect majority interest in Gyre Pharmaceuticals (also known as Beijing Continent Pharmaceuticals Co., Ltd.).
About Gyre Therapeutics
Gyre Therapeutics is a biopharmaceutical company headquartered in San Diego, CA, with a primary focus on the development and commercialization of F351 (Hydronidone) for the treatment of MASH-associated fibrosis in the U.S. Gyre’s development strategy for F351 in MASH is based on the company’s experience in MASH rodent model mechanistic studies and CHB-induced liver fibrosis clinical studies. Gyre is also advancing a diverse pipeline in the PRC through its indirect controlling interest in Gyre Pharmaceuticals, including ETUARY therapeutic expansions, F573, F528, and F230.
Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995, which statements are subject to substantial risks and uncertainties and are based on estimates and assumptions. All statements, other than statements of historical facts included in this press release, are forward-looking statements, including statements concerning: the expectations regarding Gyre’s research and development efforts, timing of expected clinical readouts, including timing of topline data from Gyre Pharmaceuticals’ Phase 3 clinical trial evaluating F351 for the treatment of CHB-associated liver fibrosis in the PRC, initiation of Gyre’s Phase 2 trial in the U.S. for F351 for the treatment of MASH-associated liver fibrosis, timing of completion of Gyre’s Phase 2 clinical trial in the PRC of F573 for ALF/ACLF, initiation of Phase 1 trial of F230 for the treatment of PAH and IND submission of F528 in COPD, the expectations regarding commercial launch of nintedanib and avatrombopag maleate tablets, interactions with regulators, expectations regarding future product sales, and Gyre’s financial position and cash resources. In some cases, you can identify forward-looking statements by terms such as “may,” “might,” “will,” “objective,” “intend,” “should,” “could,” “can,” “would,” “expect,” “believe,” “design,” “estimate,” “predict,” “potential,” “plan” or the negative of these terms, and similar expressions intended to identify forward-looking statements. These statements reflect our plans, estimates, and expectations, as of the date of this press release. These statements involve known and unknown risks, uncertainties and other factors that could cause our actual results to differ materially from the forward-looking statements expressed or implied in this press release. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation: Gyre’s ability to execute on its clinical development strategies; positive results from a clinical trial may not necessarily be predictive of the results of future or ongoing clinical trials; the timing or likelihood of regulatory filings and approvals; competition from competing products; the impact of general economic, health, industrial or political conditions in the United States or internationally; the sufficiency of Gyre’s capital resources and its ability to raise additional capital. Additional risks and factors are identified under “Risk Factors” in Gyre’s Annual Report on Form 10-K for the year ended December 31, 2023 filed on March 27, 2024 and in other filings with the Securities and Exchange Commission.
Gyre expressly disclaims any obligation to update any forward-looking statements whether as a result of new information, future events or otherwise, except as required by law.
