Traws Pharma Reports Positive Phase 1 Results for Flu Drug Tivoxavir Marboxil

Traws Pharma, Inc., a biopharmaceutical company focused on developing treatments for respiratory viral diseases, announced promising results from a Phase 1 clinical trial of its investigational flu therapy, tivoxavir marboxil. This experimental drug aims to provide a one-dose treatment for both seasonal and pandemic influenza.

Tivoxavir marboxil is designed to inhibit the CAP-dependent endonuclease (CEN) protein, which is highly conserved across various influenza viruses. The Phase 1 trial was a randomized, double-blind, placebo-controlled study involving healthy adult volunteers who were free of influenza. The primary objectives were to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of increasing doses of tivoxavir.

The results from the Phase 1 study indicated that a single dose of tivoxavir was well-tolerated by participants, with no treatment-related adverse events reported. The pharmacokinetic data showed that plasma drug levels remained consistently above the effective concentration (EC90) for more than five days, suggesting a potential for a single-dose treatment regimen.

Werner Cautreels, PhD, CEO of Traws Pharma, highlighted the importance of these findings, stating, "The Phase 1 data demonstrate good overall tolerability and a pharmacokinetic profile that supports tivoxavir's use as a one-time treatment for influenza, including pandemic flu. We are especially pleased that a single dose maintained plasma drug levels above the EC90 for more than five days."

Preclinical studies further supported the potential of tivoxavir, showing its ability to inhibit drug-resistant influenza strains and highly pathogenic avian flu viruses. These findings suggest that tivoxavir could be an effective treatment across a broad spectrum of flu viruses.

Robert R. Redfield, MD, Chief Medical Officer for Traws Pharma and former Director of the CDC, emphasized the need for new antiviral treatments. He noted, "Influenza poses a significant public health burden, especially for older adults and vulnerable populations. Recent bird flu outbreaks underline the urgent need for novel therapeutics to manage both seasonal and pandemic flu. The data on tivoxavir are encouraging and highlight its potential as a valuable resource in managing virus spread."

C. David Pauza, PhD, Chief Science Officer for Traws Pharma, explained the strategic selection of CEN as the target for tivoxavir. "CEN is conserved among human and avian influenza viruses, including the highly pathogenic H5N1 strain. Our preclinical data indicate that tivoxavir demonstrates broad activity against drug-resistant and highly pathogenic strains. Additionally, the preferential uptake in the lung is a critical feature, with preclinical data showing significantly higher lung accumulation compared to plasma levels."

The Phase 1 results have paved the way for a Phase 2 study, which is expected to commence in the first half of 2025. This next phase will further evaluate the efficacy and safety of tivoxavir in a larger group of participants.

Traws Pharma aims to address the substantial public health impact of influenza, which disproportionately affects older adults and vulnerable populations. The company is optimistic that tivoxavir could become a best-in-class treatment for both seasonal and pandemic flu, providing a much-needed solution in the fight against these pervasive viruses.