Traws Pharma Announces Positive Topline Phase 1 Data for Flu Candidate, Tivoxavir Marboxil
Traws Pharma (NASDAQ: TRAW) announced positive topline Phase 1 safety and pharmacokinetic results for its investigational one-dose influenza therapy, tivoxavir marboxil. The study demonstrated good overall tolerability and a pharmacokinetic profile supporting potential use as a one-time treatment for flu, including pandemic flu. Key findings include:
1. A single dose maintained plasma drug levels above the EC90 for over 5 days.
2. Preclinical data showed potent inhibition of drug-resistant and bird flu viruses.
3. The company plans to advance to a Phase 2 study in H1 2025.
Tivoxavir targets the highly-conserved influenza protein CAP-dependent endonuclease (CEN), potentially effective against a broad range of flu viruses. The Phase 1 trial identified the Phase 2 dose, with no treatment-related adverse events reported at this level.
Traws Pharma (NASDAQ: TRAW) ha annunciato risultati positivi preliminari di sicurezza e farmacocinetica della Fase 1 per la sua terapia antivirale monodose contro l'influenza, tivoxavir marboxil. Lo studio ha dimostrato una buona tollerabilità complessiva e un profilo farmacocinetico che supporta un potenziale utilizzo come trattamento unico per l'influenza, compresa l'influenza pandemica. I principali risultati includono:
1. Una singola dose ha mantenuto i livelli plasmatici del farmaco sopra l'EC90 per oltre 5 giorni.
2. I dati preclinici hanno mostrato un'inibizione potente dei virus resistenti ai farmaci e dell'influenza aviaria.
3. L'azienda prevede di passare a uno studio di Fase 2 nel primo semestre del 2025.
Tivoxavir colpisce la proteina altamente conservata dell'influenza CEN (endonucleasi dipendente da CAP), potenzialmente efficace contro un'ampia gamma di virus influenzali. La sperimentazione di Fase 1 ha identificato la dose per la Fase 2, senza eventi avversi correlati al trattamento segnalati a questo livello.
Traws Pharma (NASDAQ: TRAW) anunció resultados positivos preliminares de seguridad y farmacocinética de la Fase 1 para su terapia única contra la influenza, tivoxavir marboxil. El estudio demostró una buena tolerabilidad general y un perfil farmacocinético que respalda su uso potencial como tratamiento único para la gripe, incluida la gripe pandémica. Los hallazgos clave incluyen:
1. Una sola dosis mantuvo los niveles plasmáticos del fármaco por encima del EC90 durante más de 5 días.
2. Los datos preclínicos mostraron una potente inhibición de los virus resistentes a medicamentos y de la gripe aviar.
3. La empresa planea avanzar a un estudio de Fase 2 en el primer semestre de 2025.
Tivoxavir se dirige a la proteína de la influenza, la endonucleasa dependiente de CAP (CEN), que puede ser efectiva contra un amplio rango de virus de la gripe. El ensayo de Fase 1 identificó la dosis para la Fase 2, sin eventos adversos relacionados con el tratamiento reportados a este nivel.
트라우스 제약 (NASDAQ: TRAW)은 1상 안전성 및 약동학의 긍정적인 topline 결과를 발표했습니다. 이는 단일 용량 인플루엔자 치료제인 티복사비르 마르복실에 대한 것입니다. 연구 결과는 우수한 전반적인 내약성과 팬데믹 인플루엔자를 포함한 독감에 대한 단일 치료법으로서의 잠재적 사용을 지원하는 약동학적 프로필을 보여주었습니다. 주요 발견 사항은 다음과 같습니다:
1. 단일 용량이 5일 이상 EC90 이상의 혈장 약물 수준을 유지했습니다.
2. 비임상 데이터는 약물 내성 및 조류 인플루엔자 바이러스에 대한 강력한 억제를 보여주었습니다.
3. 회사는 2025년 상반기에 2상 연구로 나아갈 계획입니다.
티복사비르는 인플루엔자 단백질인 CAP 의존성 엔도뉴클레아제를 표적으로 하며, 이는 광범위한 독감 바이러스에 대해 효과적일 수 있습니다. 1상 시험은 2상 용량을 확인했으며, 이 수준에서 치료 관련 부작용은 보고되지 않았습니다.
Traws Pharma (NASDAQ: TRAW) a annoncé des résultats positifs préliminaires de sécurité et de pharmacocinétique de la Phase 1 pour sa thérapie unique contre la grippe, tivoxavir marboxil. L'étude a démontré une bonne tolérance globale et un profil pharmacocinétique soutenant une utilisation potentielle comme traitement unique de la grippe, y compris celle pandémique. Les principales conclusions incluent :
1. Une seule dose a maintenu les niveaux plasmatiques du médicament au-dessus de l'EC90 pendant plus de 5 jours.
2. Les données précliniques ont montré une inhibition puissante des virus résistants aux médicaments et de la grippe aviaire.
3. La société prévoit de passer à une étude de Phase 2 au premier semestre 2025.
Tivoxavir cible la protéine de la grippe, l'endonucléase dépendante de CAP (CEN), potentiellement efficace contre un large éventail de virus grippaux. L'essai de Phase 1 a identifié la dose de la Phase 2, sans événements indésirables liés au traitement signalés à ce niveau.
Traws Pharma (NASDAQ: TRAW) hat positive vorläufige Ergebnisse zur Sicherheit und Pharmakokinetik der Phase 1 für seine experimentelle Einzeldosis-Grippe-Therapie tivoxavir marboxil bekannt gegeben. Die Studie zeigte eine gute allgemeine Verträglichkeit und ein pharmakokinetisches Profil, das eine potenzielle Verwendung als einmalige Behandlung für Grippe, einschließlich pandemischer Grippe unterstützt. Die wichtigsten Ergebnisse umfassen:
1. Eine Einzelgabe hielt die Plasmaspiegel des Medikaments über 5 Tage über dem EC90.
2. Präklinische Daten zeigten eine starke Hemmung von medikamentenresistenten und Vogelgrippe-Viren.
3. Das Unternehmen plant, in der ersten Hälfte des Jahres 2025 in eine Phase-2-Studie überzugehen.
Tivoxavir zielt auf das hochkonservierte Influenza-Protein CEN (CAP-abhängige Endonuklease) ab, das potenziell gegen eine Vielzahl von Grippeviren wirksam ist. Die Phase-1-Studie identifizierte die Phase-2-Dosis, wobei keine behandlungsbedingten unerwünschten Ereignisse auf diesem Niveau berichtet wurden.
- Positive topline Phase 1 safety and pharmacokinetic results for tivoxavir marboxil
- Good overall tolerability and pharmacokinetic profile supporting one-time treatment potential
- Single dose maintained plasma drug levels above EC90 for over 5 days
- Preclinical data showed potent inhibition of drug-resistant and bird flu viruses
- Phase 2 study planned for H1 2025
- No treatment-related adverse events reported at the identified Phase 2 dose
- None.
Insights
The Phase 1 results for tivoxavir marboxil are promising, showing good tolerability and a favorable pharmacokinetic profile. Key points include:
- Single dose maintained plasma levels above EC90 for >5 days
- No treatment-related adverse events at the identified Phase 2 dose
- Preclinical data showed potent inhibition of drug-resistant and bird flu viruses
These results support tivoxavir's potential as a one-dose treatment for both seasonal and pandemic influenza. The drug's ability to inhibit the highly conserved CEN protein across various flu strains is particularly noteworthy. The >15X higher accumulation in lungs compared to plasma in preclinical studies is also encouraging for respiratory infections.
However, it's important to note that Phase 1 trials primarily assess safety and pharmacokinetics, not efficacy. The true potential of tivoxavir will be better understood after the planned Phase 2 study in H1 2025. The market for influenza treatments is competitive, so tivoxavir will need to demonstrate superior efficacy or convenience to existing options to gain significant market share.
The development of tivoxavir marboxil is timely given the current influenza landscape:
- Seasonal flu remains a substantial public health burden, especially for older adults
- Recent flu season data showed
~300% higher hospitalization and mortality rates in people 65+ - Increasing concerns about bird flu (H5N1) potentially evolving into a pandemic strain
Tivoxavir's broad-spectrum activity against drug-resistant and highly pathogenic strains addresses a critical need in pandemic preparedness. Its potential as a one-dose treatment could significantly improve patient compliance and reduce transmission in outbreak scenarios.
However, the true impact on public health will depend on real-world effectiveness, which is yet to be determined in larger clinical trials. The planned Phase 2 study in H1 2025 will be important in assessing tivoxavir's potential to address these unmet needs in influenza treatment and prevention.
Investigational agent in development as a one dose treatment or prevention of seasonal and pandemic influenza
A dose ranging, Phase 1 study in healthy volunteers demonstrated positive tolerability results and plasma levels in the predicted therapeutic window, enabling selection of Phase 2 dose
Preclinical data showed potent inhibition of drug-resistant and bird influenza viruses
Phase 2 study expected to begin in H1 2025
Improved therapy is an important need for both seasonal and pandemic flu
NEWTOWN, Pa., Oct. 08, 2024 (GLOBE NEWSWIRE) -- Traws Pharma, Inc. (NASDAQ: TRAW) (“Traws Pharma”, “Traws” or “the Company”), a clinical-stage biopharmaceutical company developing oral small molecule therapies for the treatment of respiratory viral diseases, today announced positive topline Phase 1 safety and pharmacokinetic results for its investigational one-dose influenza (flu) therapy, tivoxavir marboxil (tivoxavir). Tivoxavir was designed as a potential best-in-class inhibitor of the highly-conserved influenza protein,
CAP-dependent endonuclease (CEN), intended for use across a broad range of flu viruses.
“Topline data from the Phase 1 healthy volunteer study showed good overall tolerability and a pharmacokinetic profile that appears to support tivoxavir’s potential use as a one-time treatment for flu, including pandemic flu. We are especially pleased with data showing that a single dose of tivoxavir maintained plasma drug levels above the EC90 for greater than 5 days1,” said Werner Cautreels, PhD, Chief Executive Officer of Traws Pharma. “In addition, preclinical data show that tivoxavir is a potent inhibitor of drug-resistant influenza and bird flu viruses1. Together, these initial results suggest that tivoxavir has the potential to be developed as a best-in-class agent for influenza. With these data, we plan to advance the program to a Phase 2 study in H1 2025.”
“Influenza is a substantial public health burden in the US2, with a disproportionate impact on older adults and vulnerable populations. Data from the last influenza season showed that flu-related hospitalizations3 and mortality4 were highest, by approximately three times, among people 65 years of age or older5. Recent bird flu outbreaks, and the risk that this highly pathogenic virus (H5N1) poses for evolving into a pandemic outbreak6, also signal the need for new antiviral treatments,” said Robert R. Redfield, MD, Chief Medical Officer for Traws Pharma and former Director of the U.S. Centers for Disease Control and Prevention (CDC). “Our enthusiasm for tivoxavir is based on the data announced today and the need for novel therapeutics, especially as valuable resources in case of an avian flu outbreak or a pandemic, and for potential use to prevent virus spread in households and congregant settings.”
“We selected CEN as the target for our flu program because it is conserved among human and avian influenza viruses, including the highly pathogenic H5N1. As a result, we believe tivoxavir has the potential to suppress a wide range of viruses. We are very pleased that tivoxavir demonstrated broad activity in laboratory studies against drug-resistant viruses and highly pathogenic strains such as avian flu1. In addition, we believe that preferential uptake in the lung is another important feature of a candidate agent. Preclinical data indicate that a single dose of tivoxavir has more than 15X higher accumulation in the lung compared to plasma1,” said C. David Pauza, PhD, Chief Science Officer for Traws Pharma. “Positive Phase 1 data showing that tivoxavir achieved plasma blood levels in healthy subjects that are consistently above the EC90 and within the predicted therapeutic window for more than five days lead to the identification of our Phase 2 dose, supporting further development of the program.”
Topline Phase 1 Results
The Phase 1 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.
At the identified Phase 2 dose, no treatment related adverse events were reported during the Phase 1 study. Topline data from this study showed that a single dose of tivoxavir marboxil maintained plasma drug levels consistently above the EC90 for more than five days and within the predicted therapeutic window. 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.
About Tivoxavir Marboxil
Seasonal influenza is estimated to represent a 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 (also known as 27-5116 or TRX-100) 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, including the highly pathogenic avian flu, in preclinical studies. The drug candidate’s Phase 1 pharmacokinetic (PK) profile in healthy subjects, including the ability to achieve plasma levels that are consistently above the EC90 (as determined in laboratory studies), for more than five days and within the predicted therapeutic window, may enable a single dose treatment regimen. These data, combined with good overall tolerability results in healthy subjects, support further development of tivoxavir marboxil as a one-time treatment for influenza.
Source information:
About Traws Pharma, Inc.
Traws Pharma is a clinical stage biopharmaceutical company developing potential oral small molecule therapies for the treatment of respiratory viral diseases and cancer. The viral respiratory disease program includes two oral, novel, Phase 1, 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.
In the cancer program, Traws is utilizing a partnering strategy, supported by investigator sponsored studies, to advance two novel proprietary multi-kinase inhibitors, narazaciclib, targeting CDK4+, and rigosertib, targeting cell cycle proteins including PLK-1.
Traws is committed to delivering novel compounds for unmet medical needs using state-of-the-art drug development technology. With a focus on product safety and a commitment to patients in need or that are specifically vulnerable, we aim to build solutions for important medical challenges and alleviate the burden of viral infections.
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 and those discussed under the heading “Risk Factors” in Traws’ filings with the U.S. Securities and Exchange Commission (SEC). Any forward-looking statements contained in this release speak only as of its date. Traws undertakes no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events.
Traws Pharma Contacts:
Mark Guerin
Traws Pharma, Inc.
267-759-3680
www.trawspharma.com
Investor Contact:
Bruce Mackle
LifeSci Advisors, LLC
646-889-1200
bmackle@lifesciadvisors.com
FAQ
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