AI-Powered Precision: Evaxion's EVX-04 Targets Hidden Cancer Antigens in AML Treatment

Evaxion has unlocked a new frontier in immunotherapy by deploying its proprietary AI-Immunology™ technology to identify previously untapped cancer targets. The company’s latest addition to its therapeutic pipeline, EVX-04, represents a distinct departure from conventional cancer vaccine approaches by focusing on endogenous retroviruses (ERVs) embedded within the “dark genome”—the overlooked segments of our DNA that harbor dormant viral fragments.

The Science Behind ERV-Based Cancer Vaccines

What makes Evaxion’s approach distinctive is its targeting strategy. ERVs are ancient viral remnants that typically remain inactive in healthy cells but become abnormally active in cancer tissues, particularly in acute myeloid leukemia (AML). This selective activation creates an opportunity: ERV-derived antigens are visible to immune cells in tumor environments while remaining invisible in normal tissue. By concentrating on these non-conventional antigens, EVX-04 circumvents the traditional challenge of distinguishing cancer cells from healthy ones.

The AI-Immunology™ platform analyzes patient tumor sequencing data to identify therapeutically relevant ERV fragments, then selects optimal pieces capable of triggering effective immune responses across diverse patient populations. Rather than requiring personalized production for each individual, EVX-04 functions as an “off-the-shelf” therapeutic—manufactured in advance and ready for immediate deployment following diagnosis.

Clinical Evidence Takes Center Stage

Preliminary research demonstrates that EVX-04 successfully activates robust T-cell responses and induces cancer cell death in preclinical models. These findings will be showcased during an oral presentation at the American Society of Hematology (ASH) Annual Meeting and Exposition scheduled for December 6, 2025, in Florida, delivering a platform for discussing both the mechanistic insights and practical implications with the hematology community and industry stakeholders.

According to Birgitte Rønø, Chief Scientific Officer and interim CEO, the candidate exemplifies how artificial intelligence can facilitate the design of therapies addressing treatment gaps. “This approach could broaden cancer vaccine applications to patients who remain unresponsive to conventional immunotherapies,” she noted, underscoring the potential impact for populations with limited treatment options.

Positioning for Broader Impact

AML represents a particularly aggressive hematologic malignancy, characterized by rapid expansion of immature myeloid cells that overwhelm normal blood production. Without intervention, the disease typically proves fatal within weeks to months due to infection, hemorrhage, and organ complications. Current therapeutic approaches leave significant unmet needs, particularly for relapsed or refractory cases.

Evaxion’s methodology for ERV antigen discovery and vaccine design extends conceptually beyond AML, suggesting applicability across multiple cancer types where immune evasion remains a fundamental challenge. The company’s strategic expansion of its R&D pipeline reflects confidence in AI-guided immunotherapy design as a scalable model for precision oncology.