EVX-04: An Off-the-Shelf AML Vaccine Built with AI-Immunology™
Evaxion A/S announced preclinical data for EVX-04, an off-the-shelf therapeutic cancer vaccine for acute myeloid leukemia (AML). Built with the company's AI-Immunology™ platform, EVX-04 targets multiple non-conventional endogenous retrovirus (ERV) tumor antigens and showed strong, specific T-cell responses while preventing tumor growth in preclinical models.
The data was presented at the American Society of Hematology (ASH) Annual Meeting in Orlando. For product teams, the signal is clear: a data-first antigen strategy can enable a ready-to-administer vaccine with broad patient coverage.
Why this matters for product development
- Off-the-shelf by design: Preproduced dosing enables immediate use after diagnosis, avoiding the delays and cost of bespoke manufacturing.
- Data-driven coverage: Target sets were selected for cross-patient relevance, aiming to reduce patient-to-patient variability in antigen expression.
- Scalable concept: The same ERV-based approach can extend to other hard-to-treat cancers where conserved, immunogenic antigens are present.
- Evidence in models: EVX-04 induced specific T-cell responses and blocked tumor growth in preclinical studies.
How EVX-04 was built
Using sequencing data from AML patients, the platform identified ERV tumor antigens from the "dark genome." From there, it mined smaller fragments with potential for immune recognition.
- Antigen discovery: ~5 million ERV antigen fragments evaluated from patient data.
- Selection engine: The platform combined and selected 16 optimal ERV fragment sets based on cross-patient relevance and immunogenic potential.
- Validation signal: All 16 fragments included in EVX-04 triggered a specific immune response in preclinical testing.
This selection process is the product core: prioritize breadth across patients while preserving immunogenicity at the fragment level. That balance is what supports an off-the-shelf strategy.
Preclinical readout at ASH
EVX-04 produced strong, antigen-specific T-cell responses and prevented tumor growth in preclinical models. These findings were presented in an oral session at the ASH Annual Meeting, with follow-up discussions planned with researchers, clinicians, and partners.
For context on the meeting, see the ASH Annual Meeting site: American Society of Hematology.
Product strategy takeaways
- Speed to treatment: Preproduced lots enable same-day or near-term dosing after AML diagnosis.
- Manufacturing focus: Standardized composition (16 ERV fragments) supports consistent release criteria and quality controls.
- Biomarker plan: ERV expression profiling can guide patient selection and response monitoring.
- Combination logic: Pairing with standard AML regimens or checkpoint inhibitors may be evaluated as the program advances.
- Platform reuse: The selection framework can be reapplied to other tumor types with ERV overexpression.
AML context: the problem to solve
AML is an aggressive leukemia that suppresses normal blood formation, leading to infections, bleeding, and fatigue. Median age at diagnosis is 68, and many patients are not eligible for intensive treatment.
Long-term survival remains limited, especially in older adults. For a concise overview, see the NCI's AML resource: NCI: Adult AML Treatment (PDQ).
Why ERVs are attractive targets
ERVs are remnants of ancient viruses within human DNA. They are often overexpressed in tumors but silent in healthy tissue, making them visible to the immune system.
The challenge has been finding which ERV fragments matter in each cancer. AI-Immunology™ addresses that by ranking candidates from patient sequencing data and selecting sets with the highest potential benefit across patients.
What to watch next
- Clinical path: First-in-human study design, endpoints, and patient selection criteria.
- Manufacturing scale-up: Lot sizes, stability data, and CMC milestones for fixed-composition vaccines.
- Regulatory feedback: Positions on ERV targets, assay validation, and safety monitoring.
- Expansion: Indication selection beyond AML based on ERV expression maps and preclinical evidence.
About Evaxion
Evaxion is a clinical-stage TechBio company using AI-Immunology™ to build vaccines for cancer, bacterial diseases, and viral infections. The company is advancing a clinical oncology pipeline of personalized vaccines and a preclinical infectious disease pipeline addressing high unmet needs.
Forward-looking note
This article references future plans and expectations that involve risk and uncertainty, including clinical development, regulatory outcomes, financing, and market adoption. Actual results may differ due to factors such as clinical data, manufacturing, partnerships, and macro conditions. Refer to company filings with the SEC for a full discussion of risks.
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