Vasomune's AV-001 Gets US FDA Fast Track Status

Vasomune Therapeutics, Inc., headquartered in Toronto, has achieved a significant regulatory milestone with the US Food and Drug Administration (FDA) granting Fast Track designation for their drug candidate AV-001. This designation is for the prevention or treatment of moderate-to-severe acute respiratory distress syndrome (ARDS) in patients hospitalized due to viral or bacterial respiratory infections. The prominence of ARDS, a condition marked by high mortality rates, underscores the urgency and importance of this advancement.

AV-001 is a pioneering synthetic peptide, PEGylated to enhance stability, targeting the Tie2 receptor. This receptor is crucial for maintaining vascular stability and barrier integrity, particularly within the lungs. The Fast Track designation from the FDA is a testament to AV-001’s potential to meet the significant unmet medical needs in treating ARDS.

Data from Phase 1 clinical trials demonstrated that AV-001 is safe and effective with once-daily dosing. The trials also showed successful activation of the Tie2 receptor, confirming the drug’s targeted action. Vasomune Therapeutics is collaborating with AnGes Inc. in the drug’s development, and AV-001 is currently in a Phase 2a clinical trial targeting ARDS in pneumonia patients, with ongoing participant recruitment.

Dr. Brian Jahns, President and COO of Vasomune, emphasized the company’s dedication to addressing the persisting needs of patients suffering from ARDS and other conditions related to vascular endothelial instability. He expressed gratitude for the support received from the United States Department of Defense’s Congressionally Directed Medical Research Programs, which have provided significant funding for AV-001’s development. Additionally, the National Research Council of Canada’s Industrial Research Assistance Program has been instrumental in funding the Phase 1 study.

AnGes Inc.’s President & CEO, Ei Yamada, highlighted the importance of the FDA’s expedited development support, marking a critical milestone in their efforts to revolutionize ARDS treatment with AV-001. Both companies are optimistic about the ongoing Phase 2a study and the future potential of AV-001.

AV-001, initially discovered at Sunnybrook Research Institute in Toronto, is being developed under a co-development agreement between Vasomune and AnGes. The drug targets the Tie2 receptor, predominantly expressed on endothelial cells in blood vessels. By activating the Tie2-Angiopoietin signaling pathway, AV-001 enhances endothelial cell stability, fortifies barrier defenses, and prevents vascular leaks. This mechanism is particularly relevant for conditions like ARDS, sepsis, hemorrhagic shock, acute kidney injury, stroke, and vascular dementia.

Preclinical studies have shown that AV-001 fortifies endothelial cell junctions, promotes cell survival, reduces pulmonary edema, and improves lung function, resulting in significantly better survival rates compared to untreated control groups.

Vasomune Therapeutics is a private clinical-stage biopharmaceutical firm established in 2014, dedicated to developing innovative treatments leveraging the body’s natural defense mechanisms. The company’s focus on vascular normalization strategies has led to the development of their leading candidate, AV-001, from initial research to clinical trials. Vasomune operates out of Toronto, Canada, and has additional offices in Raleigh, NC.

AnGes Inc., founded in 1999, is a biopharmaceutical company specializing in gene-based therapies. The company has made significant strides with its conditional approval and commercialization of Collategene®, a plasmid-based gene therapy for ischemic ulcers. AnGes continues to explore new treatments, including AV-001 for ARDS related to COVID-19 and other infections, and an NF-κB decoy oligonucleotide for chronic discogenic lumbar back pain. The acquisition of EmendoBio in 2020 has further expanded AnGes' expertise in genome-editing technologies.