Evaxion Unveils AI-Designed Cancer Vaccine EVX-04 Targeting Acute Myeloid Leukemia
Key Insights
Evaxion (search) has developed EVX-04 (search), a novel "off-the-shelf" therapeutic cancer vaccine candidate designed to target acute myeloid leukemia using AI-identified endogenous retrovirus antigens from the dark genome.
The vaccine leverages the company's proprietary AI-Immunology™ platform to identify ERV tumor antigens that are overexpressed in cancer but absent in healthy tissue, making them highly attractive immunotherapy targets.
Preclinical data demonstrates that EVX-04 (search) induces strong T-cell responses and kills cancer cells, with results to be presented at the American Society of Hematology Annual Meeting on December 6, 2025.
Evaxion (search) A/S has expanded its research and development pipeline with EVX-04 (search), an artificial intelligence-designed precision cancer vaccine candidate targeting acute myeloid leukemia (AML). The clinical-stage TechBio company announced the addition of this novel therapeutic vaccine, currently in preclinical development, which represents a completely new approach to cancer immunotherapy.
AI-Powered Vaccine Design Targets Dark Genome
EVX-04 (search) is designed to target non-conventional endogenous retrovirus (ERV) tumor antigens from the dark genome. These ERVs are remnants of ancient viruses lying dormant in the human genome that are often overexpressed in cancer but not in healthy tissue, making them visible to the immune system and promising targets for cancer vaccines.
Leveraging Evaxion (search)'s proprietary AI-Immunology™ platform, the company has identified ERV antigens in patient tumor sequencing data. The platform then selects optimal fragments from these antigens based on their potential to be effective vaccine targets across a wide range of patients.
"We are very excited to select a lead candidate for our ERV-based precision cancer vaccine concept. Representing a completely novel approach, EVX-04 (search) is a great example of how AI-Immunology™ enables us to design and develop new therapies that could lead to better outcomes for patients," says Birgitte Rønø, CSO and interim CEO of Evaxion (search).
Off-the-Shelf Vaccine Approach
By including multiple ERV antigen fragments in EVX-04 (search), the vaccine is designed to be effective in all patients regardless of immune and tumor ERV antigen differences. This makes EVX-04 a so-called "off-the-shelf" vaccine that can be preproduced and ready for immediate administration after diagnosis.
The concept behind EVX-04 (search) is broadly applicable with potential across cancers where immunotherapies remain inadequate. This approach could enable broader use of cancer vaccines, including for patients who do not respond to conventional immunotherapies.
Promising Preclinical Results
New preclinical data demonstrates that EVX-04 (search) induces strong T-cell responses and kills cancer cells. These results will be presented at an oral session at the American Society of Hematology (ASH) Annual Meeting and Exposition in Florida on December 6, 2025.
"The ASH meeting is a fantastic opportunity for us to present the data and introduce EVX-04 (search) to both scientists, doctors and potential business partners. We are thrilled to have been selected to do an oral presentation and are looking very much forward to share and discuss the data and concept," says Rønø.
The presentation, titled "Dark genome interrogation identifies novel antigens in acute myeloid leukemia – developing an off-the-shelf vaccine from machine learning to clinic," will be delivered by Rasmus Villebro, Bioinformatics Manager at Evaxion (search), during the session on Emerging Tools, Techniques, and Artificial Intelligence in Hematology.
Addressing Critical Medical Need
AML is an aggressive hematologic malignancy characterized by the clonal expansion of undifferentiated myeloid precursor cells (AML blasts) in the bone marrow. The malignant proliferation leads to suppression of normal hematopoiesis, resulting in cytopenia, increased susceptibility to infections, bleeding, and fatigue.
If left untreated, the disease is fatal, often within weeks to months, with patients typically succumbing to infections. The urgent need for new therapeutic approaches makes EVX-04 (search)'s novel mechanism of action particularly significant for this patient population.