AV-001 Advances to High-Dose Cohort in Phase 2a Study

Vasomune Therapeutics Inc., a clinical-stage biopharmaceutical company, has announced that its leading drug candidate, AV-001, has received a positive recommendation from the Independent Data and Safety Monitoring Board (IDSMB) to proceed to the high-dose cohort in their AV001-004 Phase 2a Study. This study, registered as NCT05123755, explores the potential of AV-001 in treating patients hospitalized with pneumonia.

Shahid Ahmad, the Vice-President of Operations and Planning at Vasomune, expressed satisfaction with the IDSMB's recommendation to move forward with the study. Ahmad highlighted the importance of AV-001 in potentially improving outcomes for patients at risk of acute respiratory distress syndrome (ARDS), a condition often precipitated by pneumonia. He noted that AV-001 works by restoring Tie2 signaling, which is crucial for maintaining endothelial stability, enhancing barrier defense, and preventing vascular leakage. The continuation of this Phase 2a study marks a critical milestone for AV-001, thanks to the dedication of clinical investigators and the support from the United States Department of Defense Congressionally Directed Medical Research Programs award #PR203503.

Ei Yamada, President & CEO of AnGes, also expressed optimism about the IDSMB's decision to advance the study to its final cohort. Yamada underscored the commitment to completing this essential clinical study, which could significantly impact the treatment of ARDS, a condition with no current standard of care.

AV-001 was initially discovered and designed at the Sunnybrook Research Institute at Sunnybrook Hospital in Toronto. Vasomune Therapeutics Inc. is developing the drug under a co-development agreement with AnGes Inc. AV-001 is a novel investigational medicine targeting the Tie2 receptor, a protein on endothelial cell surfaces within the vascular system. The activation of the Tie2-Angiopoietin signaling axis by AV-001 helps normalize the vasculature by enhancing endothelial cell stability, restoring barrier defense, and preventing vascular leakage. This process addresses the underlying disease mechanisms in conditions such as bacterial and viral ARDS, sepsis, hemorrhagic shock, acute kidney injury, stroke, and vascular dementia. Pre-clinical studies have shown that AV-001 significantly improves survival by tightening cell junctions, reducing pulmonary edema, and enhancing lung function.

Vasomune Therapeutics Inc., founded in 2014, is dedicated to developing the next generation of medicines that leverage the body's natural defense mechanisms. The company focuses on strategies that normalize vascular function, and AV-001 represents its lead candidate, currently progressing from research to clinical application. Vasomune's headquarters and laboratories are located in Toronto, Canada, with additional offices in Raleigh, NC.

AnGes Inc., founded in December 1999, is a biopharmaceutical company that develops gene-based medicines. AnGes' first clinical product, Collategene®, is a gene therapy for Chronic Limb-Threatening Ischemia that was granted Breakthrough designation by the FDA in 2024. AnGes is also developing AV-001, a Tie2 tyrosine kinase receptor agonist, for the treatment of COVID-19, viral and bacterial pneumonia, and an NF-κB decoy oligonucleotide for chronic discogenic lumbar back pain.