Traws Pharma, in collaboration with Expert Systems, has announced promising progress in its H5N1 antiviral program with Tivoxavir Marboxil, an investigational drug showing potential as a single-dose treatment for avian influenza. The completion of Phase 1 trials and plans for Phase 2 studies in early 2025 mark a significant step forward in addressing this global health threat.
Tivoxavir Marboxil: A Novel Approach to Influenza Treatment
Tivoxavir Marboxil is an oral cap-dependent endonuclease inhibitor designed to target a highly conserved influenza protein. This mechanism of action allows for broad-spectrum efficacy against various flu strains, including avian influenza. Preclinical studies have demonstrated the drug's potent activity against drug-resistant and highly pathogenic bird flu viruses in both in vitro and in vivo models.
Phase 1 Trial Results
The Phase 1 clinical trial was a randomized, double-blind, placebo-controlled study that evaluated the safety, tolerability, and pharmacokinetics of single ascending doses of Tivoxavir Marboxil in healthy, influenza-negative adult volunteers. The trial reported no treatment-related adverse events. Notably, a single dose maintained plasma drug levels above the effective concentration (EC90) for over 23 days, suggesting the potential for sustained efficacy with a one-time administration.
Expert Commentary
Dr. Robert R. Redfield, Chief Medical Officer for Traws Pharma and former Director of the U.S. Centers for Disease Control and Prevention (CDC), emphasized the importance of this development, stating, "The spread of avian influenza in wild and domestic animal populations, including mammals, brings increasing risk for adaptation to humans and subsequent spread in the population. With increasing numbers of human infections and recent reports of severe cases, we should be alert to the rising potential for epidemic or pandemic spread of bird flu."
The Role of AI in Drug Discovery
Expert Systems has played a crucial role in the ideation, design, and support of the Tivoxavir Marboxil program, leveraging its hybrid human and AI-based drug discovery platform. This collaboration highlights the effectiveness of integrating artificial intelligence with human expertise and computer-aided drug design to accelerate the development of innovative therapies.
Addressing a Critical Unmet Need
The development of Tivoxavir Marboxil addresses a critical need for effective treatments against avian influenza, particularly as the virus continues to evolve and spread. The potential for a single-dose oral therapy offers significant advantages in terms of ease of administration and patient compliance, especially in the event of a pandemic outbreak.
Next Steps: Phase 2 Trials
With the completion of Phase 1 trials, Traws Pharma is planning to initiate Phase 2 efficacy studies in the first half of 2025. These studies will further evaluate the safety and efficacy of Tivoxavir Marboxil in patients infected with H5N1 bird flu, bringing the drug closer to potential regulatory approval and commercialization.
