Traws Pharma Announces Completion of Phase I Studies with Tivoxavir Marboxil, a Single Dose Oral Investigational Drug for the Treatment and Prevention of H5N1 Bird Flu
Traws Pharma (NASDAQ: TRAW) has announced the completion of Phase I clinical studies for tivoxavir marboxil, their investigational one-dose therapy for H5N1 bird flu treatment and prevention. The randomized, double-blind, placebo-controlled study evaluated safety, tolerability, and pharmacokinetics in healthy adult volunteers across four dose levels.
The topline results showed no significant treatment-related adverse events, and pharmacokinetic data supported dosing for both therapeutic use and prevention. Preclinical studies demonstrated that tivoxavir marboxil effectively inhibited drug-resistant influenza viruses and highly pathogenic bird flu viruses both in vitro and in vivo.
The company views these results as supportive of accelerated development in response to growing bird flu threats, particularly following the first U.S. death and spread into commercial poultry farms. The combined clinical and animal model data will guide upcoming regulatory meetings.
Traws Pharma (NASDAQ: TRAW) ha annunciato il completamento degli studi clinici di Fase I per tivoxavir marboxil, la loro terapia sperimentale a dose unica per il trattamento e la prevenzione dell'influenza aviaria H5N1. Lo studio randomizzato, in doppio cieco e controllato con placebo ha valutato la sicurezza, la tollerabilità e la farmacocinetica in volontari adulti sani su quattro livelli di dosaggio.
I risultati principali hanno mostrato che non ci sono stati eventi avversi significativi legati al trattamento, e i dati farmacocinetici hanno supportato il dosaggio sia per l'uso terapeutico che per la prevenzione. Gli studi preclinici hanno dimostrato che il tivoxavir marboxil inibisce efficacemente i virus dell'influenza resistenti ai farmaci e i virus altamente patogeni dell'influenza aviaria sia in vitro che in vivo.
L'azienda considera questi risultati come supporto per lo sviluppo accelerato in risposta alla crescente minaccia dell'influenza aviaria, in particolare dopo la prima morte negli Stati Uniti e la diffusione nelle fattorie avicole commerciali. I dati clinici combinati con quelli del modello animale guideranno i prossimi incontri con le autorità regolatorie.
Traws Pharma (NASDAQ: TRAW) ha anunciado la finalización de los estudios clínicos de Fase I para tivoxavir marboxil, su terapia experimental de una sola dosis para el tratamiento y la prevención de la gripe aviar H5N1. El estudio aleatorizado, doble ciego y controlado con placebo evaluó la seguridad, la tolerabilidad y la farmacocinética en voluntarios adultos sanos en cuatro niveles de dosis.
Los resultados principales mostraron que no hubo eventos adversos significativos relacionados con el tratamiento, y los datos farmacocinéticos respaldaron la dosificación tanto para el uso terapéutico como para la prevención. Los estudios preclínicos demostraron que el tivoxavir marboxil inhibe eficazmente los virus de la influenza resistentes a los medicamentos y los virus de la gripe aviar altamente patógenos tanto in vitro como in vivo.
La empresa considera estos resultados como un apoyo para el desarrollo acelerado en respuesta a la creciente amenaza de la gripe aviar, especialmente tras la primera muerte en EE.UU. y la propagación en granjas avícolas comerciales. Los datos clínicos combinados con los de modelos animales guiarán las próximas reuniones regulatorias.
트라우스 파마 (NASDAQ: TRAW)는 H5N1 조류 독감 치료 및 예방을 위한 단회 투여 요법인 티폭사비르 마르복실의 1상 임상 연구 완료를 발표했습니다. 무작위 이중 맹검 위약 대조 연구는 건강한 성인 자원봉사자를 대상으로 4개의 용량 수준에서 안전성, 내약성 및 약리학을 평가했습니다.
주요 결과는 치료와 관련된 중대한 부작용이 없음을 보여주었고, 약리학적 데이터는 치료 및 예방 사용을 위한 용량 지지를 뒷받침했습니다. 전임상 연구는 티폭사비르 마르복실이 약물 내성 인플루엔자 바이러스와 고병원성 조류 독감 바이러스를 시험관 내외에서 효과적으로 억제했음을 확인했습니다.
회사는 이러한 결과를 조류 독감 위협 증가에 대응하기 위한 가속 개발의 지지 증거로 보고 있으며, 특히 미국에서 발생한 첫 사망과 상업적인 가금류 농장으로의 확산 이후 그렇습니다. 결합된 임상 및 동물 모델 데이터는 향후 규제 회의를 안내할 것입니다.
Traws Pharma (NASDAQ: TRAW) a annoncé l'achèvement des études cliniques de Phase I pour tivoxavir marboxil, leur thérapie expérimentale à dose unique pour le traitement et la prévention de la grippe aviaire H5N1. L'étude randomisée, en double aveugle et contrôlée par placebo a évalué la sécurité, la tolérance et la pharmacocinétique chez des volontaires adultes en bonne santé à quatre niveaux de dosage.
Les résultats principaux ont montré qu'il n'y avait pas d'événements indésirables significatifs liés au traitement, et les données pharmacocinétiques ont soutenu le dosage tant pour l'utilisation thérapeutique que pour la prévention. Des études précliniques ont démontré que le tivoxavir marboxil inhibe efficacement les virus de la grippe résistants aux médicaments et les virus de la grippe aviaire hautement pathogènes, tant in vitro qu'in vivo.
L'entreprise considère ces résultats comme un soutien à un développement accéléré en réponse à la menace croissante de la grippe aviaire, notamment après le premier décès aux États-Unis et la propagation dans les exploitations avicoles commerciales. Les données cliniques combinées avec celles des modèles animaliers guideront les prochaines réunions de réglementation.
Traws Pharma (NASDAQ: TRAW) hat den Abschluss der Phase-I-Studien für Tivoxavir Marboxil, ihre experimentelle Einzeldosistherapie zur Behandlung und Vorbeugung der H5N1-Vogelgrippe, bekannt gegeben. Die randomisierte, doppelblinde, placebokontrollierte Studie bewertete die Sicherheit, Verträglichkeit und Pharmakokinetik bei gesunden erwachsenen Freiwilligen in vier Dosierungsstufen.
Die wichtigsten Ergebnisse zeigten keine signifikanten unerwünschten Behandlungseffekte, und die pharmakokinetischen Daten unterstützten die Dosierung sowohl für therapeutische als auch für präventive Zwecke. Präklinische Studien haben gezeigt, dass Tivoxavir Marboxil sowohl in vitro als auch in vivo gegen medikamentenresistente Influenza-Viren und hochpathogene Vogelgrippeviren wirksam inhibiert.
Das Unternehmen betrachtet diese Ergebnisse als Unterstützung für eine beschleunigte Entwicklung in Reaktion auf die wachsenden Bedrohungen durch Vogelgrippe, insbesondere nach dem ersten Todesfall in den USA und der Ausbreitung in kommerzielle Geflügelfarmen. Die kombinierten klinischen und tierexperimentellen Daten werden die bevorstehenden regulatorischen Treffen leiten.
- Successful completion of Phase I trials with no significant treatment-related adverse events
- Positive pharmacokinetic data supporting both therapeutic and preventive dosing
- Demonstrated potent inhibition of drug-resistant influenza and bird flu viruses in preclinical studies
- Early-stage development (Phase I only completed)
- Further animal testing required before regulatory approval
Insights
The successful completion of Phase I trials for tivoxavir marboxil represents a significant milestone in addressing the growing threat of H5N1 bird flu. The key differentiator is its single-dose oral administration, which could provide substantial advantages over existing multi-dose treatments in both therapeutic and prophylactic settings.
The clean safety profile across four dose levels is particularly noteworthy, as it may accelerate the development timeline. However, investors should understand that Phase I success, while important, primarily demonstrates safety rather than efficacy. The pharmacokinetic data supporting both therapeutic and preventive applications could substantially expand the drug's market potential.
From a regulatory perspective, Traws Pharma may benefit from expedited pathways given the urgent public health need and lack of effective H5N1 treatments. The involvement of former CDC Director Robert Redfield adds credibility to the program and suggests potential support from regulatory authorities.
The preclinical efficacy against resistant strains is particularly valuable, as antiviral resistance is a growing concern in influenza treatment. This positions tivoxavir marboxil as a potential second-line therapy even if it doesn't immediately capture first-line market share.
For a micro-cap company (
Phase I completed in healthy volunteers with pharmacokinetic data supporting dosing for both therapeutic and further development for bird flu prevention
"We believe that our clinical data in healthy volunteers support accelerated development of tivoxavir marboxil in response to the growing threat of bird flu," said Werner Cautreels, PhD, Chief Executive Officer of Traws Pharma.
"The first
"Topline data from the Phase I study demonstrated safety and tolerability of tivoxavir marboxil in humans at levels of exposure that have demonstrated potent inhibition of H5N1 and informs dose selections for further testing in relevant animal models of bird flu," said C. David Pauza, PhD, Chief Science Officer for Traws Pharma. "The combined clinical and animal model data package will guide our upcoming regulatory meetings as we advance this product and, hopefully, provide an important medical countermeasure against bird flu."
Topline Phase I Results
The Phase I trial was a randomized, double-blind, placebo-controlled study to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of ascending doses of one-time tivoxavir marboxil treatment in healthy, influenza-negative, adult volunteers.
No significant treatment related adverse events were reported during the Phase I study, which evaluated four dose levels. Preclinical studies showed that tivoxavir marboxil demonstrated potent inhibition of drug-resistant influenza viruses, as well as potent inhibition of highly pathogenic bird flu viruses1, both in vitro and in vivo.
About Tivoxavir Marboxil
Seasonal influenza represents an estimated multi-billion antiviral market opportunity, largely driven by global health organizations, practice guidelines and government tenders1, with upside potential from pandemic flu outbreaks. Tivoxavir marboxil was designed as an inhibitor of the highly conserved influenza protein, CAP-dependent endonuclease (CEN). It has demonstrated potent in vitro activity against a range of influenza strains in preclinical studies, including the highly pathogenic avian flu H5N1 (bird flu). Completed and ongoing animal model studies are assessing the impact of a single dose of tivoxavir marboxil on lethal bird flu infection. We believe that these data support further development of tivoxavir marboxil as a one-time treatment for bird flu.
Source information:
- TRAWS data on file
- https://www.cdc.gov/media/releases/2025/m0106-h5-birdflu-death.html
- http://dx.doi.org/10.15585/mmwr.mm7344a3
About Traws Pharma, Inc. Antiviral Programs
Traws Pharma is a clinical stage biopharmaceutical company developing potential oral small molecule therapies for the treatment of respiratory viral diseases. The viral respiratory disease program includes two oral, novel, Phase I, potentially best-in-class, small molecule drug candidates: tivoxavir marboxil, in development for flu and pandemic flu, targeting the influenza cap-dependent endonuclease (CEN); and ratutrelvir, in development as a COVID treatment, targeting the Mpro (3CL protease), without the need for co-administration of ritonavir.
Traws is committed to delivering novel compounds for unmet medical needs using state-of-the-art drug development technology. With a commitment to patients in need and vulnerable populations, we aim to build solutions for important medical challenges and alleviate the burden of viral infectious disease.
Forward-Looking Statements
Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, Section 21E of the Securities Exchange Act of 1934, as amended, and the Private Securities Litigation Reform Act of 1995, and involve risks and uncertainties including statements regarding the Company, its business and product candidates, including the potential opportunity, benefits and the regulatory plans for tivoxavir marboxil. The Company has attempted to identify forward-looking statements by terminology including "believes", "estimates", "anticipates", "expects", "plans", "intends", "may", "could", "might", "will", "should", "preliminary", "encouraging", "approximately" or other words that convey uncertainty of future events or outcomes. Although Traws believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including the success and timing of Traws' clinical trials, collaborations, market conditions, regulatory requirements, the extent of the spread and threat of the bird flu, and those discussed under the heading "Risk Factors" in Traws' filings with the
Traws Pharma Contact:
Mark Guerin
Traws Pharma, Inc.
267-759-3680
www.trawspharma.com
View original content:SOURCE Traws Pharma, Inc.
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