Tonix’s TNX-1500 Shows Promise in Preventing Organ Transplant Rejections of Either Human or Pig Organs; Autoimmune Diseases Also a Target
Tonix Pharmaceuticals (NASDAQ:TNXP) has reported promising results for TNX-1500, a humanized monoclonal antibody designed to prevent organ transplant rejections and treat autoimmune diseases. The company recently announced topline results from its Phase 1 trial, demonstrating TNX-1500's ability to block immune responses in healthy volunteers with a half-life supporting monthly dosing.
TNX-1500 represents a third-generation CD40L blocker that aims to maintain the effectiveness of first-generation blockers while improving tolerability. The drug shows potential in preventing rejection of both human and pig organ transplants, addressing a critical need as over 100,000 people in the U.S. await organ transplants, with approximately 17 deaths daily due to organ shortages.
The organ transplant immunosuppressant drug market, valued at $5.5 billion in 2023, is projected to reach $7.17 billion by 2030. TNX-1500's development targets both traditional human organ transplants and xenotransplants, with potential expansion into autoimmune conditions. Animal studies have shown promising results with reduced thrombotic events, suggesting successful protein engineering modifications.
Tonix Pharmaceuticals (NASDAQ:TNXP) ha riportato risultati promettenti per TNX-1500, un anticorpo monoclonale umanizzato progettato per prevenire il rigetto degli organi trapiantati e trattare malattie autoimmuni. L'azienda ha recentemente annunciato i risultati preliminari del suo studio di Fase 1, dimostrando la capacità di TNX-1500 di bloccare le risposte immunitarie in volontari sani, con una emivita che supporta somministrazioni mensili.
TNX-1500 rappresenta un bloccante CD40L di terza generazione che mira a mantenere l'efficacia dei bloccanti di prima generazione, migliorando al contempo la tollerabilità. Il farmaco mostra potenziale nel prevenire il rigetto sia di trapianti di organi umani che di organi di maiale, affrontando un bisogno critico poiché oltre 100.000 persone negli Stati Uniti aspettano trapianti di organi, con circa 17 decessi al giorno a causa della carenza di organi.
Il mercato dei farmaci immunosoppressori per il trapianto di organi, valutato 5,5 miliardi di dollari nel 2023, è previsto raggiungere 7,17 miliardi di dollari entro il 2030. Lo sviluppo di TNX-1500 mira sia ai tradizionali trapianti di organi umani che ai xenotrapianti, con potenziale espansione nelle condizioni autoimmuni. Studi sugli animali hanno mostrato risultati promettenti con eventi trombotici ridotti, suggerendo modifiche ingegneristiche di proteine di successo.
Tonix Pharmaceuticals (NASDAQ:TNXP) ha reportado resultados prometedores para TNX-1500, un anticuerpo monoclonal humanizado diseñado para prevenir el rechazo de trasplantes de órganos y tratar enfermedades autoinmunes. La compañía anunció recientemente resultados preliminares de su ensayo de Fase 1, demostrando la capacidad de TNX-1500 para bloquear las respuestas inmunitarias en voluntarios sanos, con una vida media que respalda la dosificación mensual.
TNX-1500 representa un bloqueador de CD40L de tercera generación que busca mantener la efectividad de los bloqueadores de primera generación mientras mejora la tolerabilidad. El fármaco muestra potencial para prevenir el rechazo tanto de trasplantes de órganos humanos como de órganos de cerdo, abordando una necesidad crítica, ya que más de 100,000 personas en los EE. UU. esperan trasplantes de órganos, con aproximadamente 17 muertes diarias debido a la escasez de órganos.
El mercado de fármacos inmunosupresores para trasplantes de órganos, valorado en 5.5 mil millones de dólares en 2023, se proyecta que alcanzará 7.17 mil millones de dólares para 2030. El desarrollo de TNX-1500 se dirige tanto a los trasplantes de órganos humanos tradicionales como a los xenotrasplantes, con una posible expansión en condiciones autoinmunes. Los estudios en animales han mostrado resultados prometedores con eventos trombóticos reducidos, sugiriendo modificaciones exitosas en la ingeniería de proteínas.
Tonix Pharmaceuticals (NASDAQ:TNXP)는 장기 이식 거부 반응을 예방하고 자가 면역 질환을 치료하기 위해 설계된 인간화 단클론 항체 TNX-1500에 대한 유망한 결과를 보고했습니다. 회사는 최근 건강한 자원봉사자를 대상으로 한 1상 시험의 주요 결과를 발표했으며, TNX-1500이 면역 반응을 차단할 수 있는 능력을 보여주었고, 월간 투여를 지원하는 반감기를 가지고 있습니다.
TNX-1500은 1세대 블로커의 효과를 유지하면서 내약성을 개선하는 것을 목표로 하는 3세대 CD40L 블로커입니다. 이 약물은 인간과 돼지 장기 이식 모두에서 거부 반응을 예방할 수 있는 잠재력을 보여주며, 미국에서 100,000명 이상의 사람들이 장기 이식을 기다리고 있고, 매일 약 17명이 장기 부족으로 사망하는 심각한 필요를 해결합니다.
장기 이식 면역억제제 시장은 2023년 55억 달러로 평가되며, 2030년까지 71억 7천만 달러에 이를 것으로 예상됩니다. TNX-1500의 개발은 전통적인 인간 장기 이식과 이종 이식을 모두 목표로 하며, 자가 면역 질환으로의 확장 가능성도 있습니다. 동물 연구에서는 혈전 사건이 감소한 유망한 결과를 보여주어 성공적인 단백질 공학 수정이 이루어졌음을 시사합니다.
Tonix Pharmaceuticals (NASDAQ:TNXP) a rapporté des résultats prometteurs pour TNX-1500, un anticorps monoclonal humanisé conçu pour prévenir les rejets de greffes d'organes et traiter les maladies auto-immunes. L'entreprise a récemment annoncé les résultats préliminaires de son essai de phase 1, démontrant la capacité de TNX-1500 à bloquer les réponses immunitaires chez des volontaires en bonne santé, avec une demi-vie soutenant une posologie mensuelle.
TNX-1500 représente un bloqueur CD40L de troisième génération qui vise à maintenir l'efficacité des bloqueurs de première génération tout en améliorant la tolérance. Le médicament montre un potentiel pour prévenir le rejet tant des greffes d'organes humains que des greffes d'organes de porc, répondant à un besoin critique alors que plus de 100 000 personnes aux États-Unis attendent des greffes d'organes, avec environ 17 décès par jour dus à la pénurie d'organes.
Le marché des médicaments immunosuppresseurs pour les greffes d'organes, évalué à 5,5 milliards de dollars en 2023, devrait atteindre 7,17 milliards de dollars d'ici 2030. Le développement de TNX-1500 cible à la fois les greffes d'organes humains traditionnels et les xénogreffes, avec une éventuelle expansion vers les maladies auto-immunes. Des études sur des animaux ont montré des résultats prometteurs avec des événements thrombotiques réduits, suggérant des modifications réussies en ingénierie des protéines.
Tonix Pharmaceuticals (NASDAQ:TNXP) hat vielversprechende Ergebnisse für TNX-1500, einen humanisierten monoklonalen Antikörper, der entwickelt wurde, um Organtransplantationsabstoßungen zu verhindern und Autoimmunerkrankungen zu behandeln, gemeldet. Das Unternehmen gab kürzlich die vorläufigen Ergebnisse seiner Phase-1-Studie bekannt, die die Fähigkeit von TNX-1500 zeigt, Immunreaktionen bei gesunden Freiwilligen zu blockieren, mit einer Halbwertszeit, die monatliche Dosierungen unterstützt.
TNX-1500 stellt einen CD40L-Blocker der dritten Generation dar, der darauf abzielt, die Wirksamkeit von Blockern der ersten Generation aufrechtzuerhalten und gleichzeitig die Verträglichkeit zu verbessern. Das Medikament zeigt Potenzial zur Verhinderung von Abstoßungen sowohl bei menschlichen als auch bei Schweineorgantransplantationen und adressiert einen kritischen Bedarf, da über 100.000 Menschen in den USA auf Organtransplantationen warten, wobei täglich etwa 17 Todesfälle aufgrund von Organmangel auftreten.
Der Markt für immunsuppressive Medikamente zur Organtransplantation, der im Jahr 2023 mit 5,5 Milliarden US-Dollar bewertet wird, wird voraussichtlich bis 2030 auf 7,17 Milliarden US-Dollar anwachsen. Die Entwicklung von TNX-1500 zielt sowohl auf traditionelle menschliche Organtransplantationen als auch auf Xenotransplantationen ab, mit der Möglichkeit einer Erweiterung auf Autoimmunerkrankungen. Tierversuche haben vielversprechende Ergebnisse mit reduzierten thrombotischen Ereignissen gezeigt, was auf erfolgreiche Proteinengineering-Modifikationen hindeutet.
- Successful Phase 1 trial results showing effective immune response blocking
- Drug demonstrates potential for convenient monthly dosing schedule
- Effective in both human and pig organ transplant prevention in animal studies
- Reduced thrombotic events in animal studies compared to earlier generations
- Large market opportunity with $5.5B current market size, growing to $7.17B by 2030
- Product still in early development stages with only Phase 1 completion
- Faces competition from other CD40L blockers in development
- Will require extensive additional clinical trials for multiple indications
Insights
The Phase 1 trial results for Tonix's TNX-1500 represent a meaningful milestone in the company's clinical development pipeline. This third-generation CD40L blocker demonstrated its ability to inhibit immune response in healthy volunteers with a pharmacokinetic profile supporting monthly dosing, addressing key limitations of earlier CD40L blockers.
The market opportunity is substantial, with the organ transplant immunosuppressant sector projected to grow from
TNX-1500's differentiation lies in its engineered design that maintains efficacy while reducing thrombotic complications. If successful, this could position Tonix in multiple high-value markets including both human and xenotransplantation, plus potential expansion into autoimmune conditions where other CD40L blockers have shown promise.
While promising, investors should recognize that substantial clinical development remains, with years before potential commercialization. The company will need sufficient capital to advance through more expensive later-stage trials across multiple indications.
TNX-1500 represents an important scientific advancement in transplant medicine through its targeted approach to immune modulation. Unlike traditional immunosuppressants that broadly suppress immunity, CD40L blockers like TNX-1500 more selectively inhibit T-cell responses to transplanted organs.
The successful Phase 1 results validate the protein engineering approach used to modify the Fc region, apparently achieving the important balance of maintaining immunomodulatory efficacy while reducing thrombotic risk. This addresses the fatal flaw of first-generation CD40L antibodies that showed promise but caused dangerous blood clots.
The clinical significance extends beyond conventional transplantation. With 17 people dying daily while awaiting organs, xenotransplantation (pig-to-human) represents a potential solution to organ shortages. However, cross-species rejection presents formidable immunological barriers that TNX-1500 might help overcome.
The data from animal studies showing efficacy in both same-species and pig organ transplants is scientifically compelling, though human transplant studies will present more complex challenges. The half-life supporting monthly dosing provides a practical advantage over non-antibody CD40L blockers requiring more frequent administration.
The validation of CD40L inhibition as a therapeutic pathway through positive trials by Sanofi, Biogen and Amgen in autoimmune conditions strengthens the scientific rationale behind Tonix's approach.
This post was written and published as a collaboration between the in-house editorial team at Benzinga and Tonix Pharmaceuticals Holding Corp. with financial support from Tonix. The two organizations work to ensure that any and all information contained within is true and accurate as of the date hereof to the best of their knowledge and research. This content is for informational purposes only and not intended to be investing advice.
CHATHAM, NJ / ACCESS Newswire / March 12, 2025 / On any given day, there are over 100,000 people in the U.S. alone waiting for an organ transplant. Of that group, approximately 17 will succumb to their illness each day without a transplant. That's why doctors and researchers have turned to non-human organs to make up for the shortfall. In March 2024 doctors performed the first ever transplant of a modified pig kidney into a living human. However, the rejection rate for non-human organs, otherwise known as xenotransplants, is much higher than in human transplants. Even with human organs, as many as
Those are worrying statistics that Tonix Pharmaceuticals Holding Corp. (NASDAQ:TNXP), the biopharmaceutical company, is trying to change with TNX-1500,* a humanized monoclonal antibody that blocks the action of CD40-ligand (CD40L), also known as CD154. The promise of CD40L blockers is to modulate the immune system rather than suppress it. Traditional immunosuppression drugs work for transplant and autoimmune diseases, but they carry a number of liabilities related to how broadly they suppress immunity and also to how they damage kidneys and other organs at the doses required for efficacy.
"Despite advancements in the field of solid organ transplantation, there remains a significant need for new treatments with improved activity and tolerability," said Seth Lederman, M.D., Chief Executive Officer of Tonix Pharmaceuticals. "Anti-CD40L modulates T cell function and has the potential to promote tolerance of transplanted organs."
TNX-1500 is a third-generation CD40L blocker that has been designed by protein engineering to maintain the activity of first generation CD40L blockers, while improving their tolerability. The hope is that agents like TNX-1500 may someday replace immunosuppressant drugs or at least reduce the dose of immunosuppressants to a point where they are safer.
The first generation of CD40L blockers were monoclonal antibodies that showed promise in reducing organ rejection and addressing autoimmune conditions, but their use was associated with an increased risk of thrombosis, or blood clots. The second generation of CD40L blockers included some biologics that were not monoclonal antibodies. Most of these reduced thrombosis risk, but sacrificed activity. The third generation of anti-CD40L blockers are based on learnings from the first two generations and are designed to retain the activity of the first generation, but with the reduced risk thrombosis. The promise of third-generation CD40L blockers is to translate the science into better patient outcomes with lower rates of rejection, and less reliance on traditional broad-spectrum immunosuppressants.
Proof is in the Studies
In February, 2025, Tonix reported the topline results of a first-in-human Phase 1 trial of TNX-1500. This was a single ascending dose escalation trial of TNX-1500 in healthy volunteers. Results of the Phase 1 trial are encouraging because TNX-1500 treatment blocked the ability of volunteers to respond to a test immunization. Also, the half-life of TNX-1500 in the blood stream supports monthly dosing in trials aimed towards FDA approval.
Earlier animal studies indicated that TNX-1500 is active at preventing rejection of organ grafts and preserving graft function, either as a single agent or in combination with low doses of traditional immunosuppressants. TNX-1500 is active whether the organ comes from the same species or from genetically engineered pigs. So far, TNX-1500 treatment in these animal transplantation studies has shown a dramatic reduction in thrombotic events, indicating that the protein engineering of TNX-1500's Fc region achieved its design goals.
Although the lead indication for Tonix's TNX-1500 product candidate is the prevention of rejection of transplanted human kidneys, Tonix is also pursuing development of TNX-1500 for preventing rejection of genetically engineered pig organs. Ultimately, Tonix also plans to develop TNX-1500 as a treatment for autoimmune diseases.
Recently, encouraging clinical data in three different autoimmune conditions have been reported with other CD40L blockers. Sanofi SA reported an encouraging Phase 2 study in relapsing multiple sclerosis with frexalimab1, Biogen reported a positive Phase 3 study in systemic lupus erythematosus with dapirolizumab pegol2,3, and Amgen reported positive results in two patient populations in a Phase 2 study of Sjogren's Syndrome with dazodalibep4,5. Like TNX-1500, Sanofi's frexalimab has traditional characteristics of a humanized monoclonal antibody. In contrast, Biogen's dapirolizumab pegol and Amgen's dazodalibep are different types of biologics that block CD40L. The half-lives of TNX-1500 and frexalimab are greater than the reported half-lives of the non-antibodies, which provides for convenient monthly dosing.
Growing Potential Multi-Billion Dollar Market
Reducing rejection rates of human and non-human organs would not only save lives, it is also a potential big opportunity for Tonix. According to one estimate, the organ transplant immunosuppressant drug market was valued at
One other CD40L blocker is in development for preventing organ transplant rejection, tegoprubart from Eledon Pharmaceuticals Inc.. A Phase 2 study of tegoprubart in kidney transplant has completed dosing of 120 patients6 and data are expected in 2026. Tegoprubart is a non-covalent antibody with no heavy-light or heavy-heavy interchain disulfide bridges that is being studied with i.v. dosing every three weeks. Tonix's TNX-1500 has disulfide bridges and a half-life that supports i.v. dosing monthly.
With the number of needy people in the U.S. in excess of 100,000 and with the medical community turning to non-human organs to fill the void, developing drugs to make sure these organs take is imperative. Tonix believes it has a solution with TNX-1500. With Phase 1 trial results recently announced and with the organ transplant market seemingly poised to grow, Tonix's drug development is bringing hope to patients on the transplant waiting lists.
"Organ transplant is a stringent test of a CD40L blocker to modulate the immune system. The activity of TNX-1500 that we've observed in animal studies of human and pig transplants, and on blocking the response to a test antigen in the Phase 1 study encourages us to develop TNX-1500 for allotransplantation, xenotransplantation and also for the treatment of autoimmune conditions," said Dr. Lederman.
*TNX-1500 (Fc-modified humanized anti-CD40L monoclonal antibody) is an investigational new drug and is not approved for any indication
1Vermersch P, et al. N Engl J Med. 2024 Feb 15;390(7):589-600. doi: 10.1056/NEJMoa2309439. PMID: 38354138
2Gelman, M. Endpoints. "Biogen, UCB detail response rates in Phase 3 lupus trial after surprising Success" Nov. 19, 2024
3Biogen Press Release, November 19, 2024. https://investors.biogen.com/news-releases/news-release-details/dapirolizumab-pegol-phase-3-data-presented-american-college
4Horizon Press Release. September 12, 2022. https://www.biospace.com/article/releases/horizon-therapeutics-plc-announces-phase-2-trial-evaluating-dazodalibep-for-the-treatment-of-sjoegren-s-syndrome-meets-primary-endpoint/
5Horizon Press Release. January 18, 2023.
6www.nasdaq.com/articles/eledon-pharmaceuticals-completes-enrollment-phase-2-bestow-trial-tegoprubart-kidney
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Contact Information
Jessica Morris
Tonix Pharmaceuticals
investor.relations@tonixpharma.com
862.904.8182
SOURCE: Tonix Pharmaceuticals Holding
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FAQ
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