EVX-01
InvestigationalA personalized neoantigen peptide vaccine developed by Evaxion Biotech using AI-driven neoantigen prediction. Phase 1/2 data in melanoma showed a 67% objective response rate (8/12; 6 partial, 2 complete) when combined with anti-PD-1 therapy, with neoantigen-specific T-cell responses detected in all patients. Distinct from mRNA-based approaches like mRNA-4157.
EVX01 · Evaxion EVX-01 · AI-Immunology Neoantigen Vaccine
12 human studies
- Preclinical
- 33%
- Clinical
- 67%
Based on 18 cited sources
clinically demonstrated · very-low confidence
Demonstrated effect magnitude — not a recommendation or safety claim.
Research Depth 38: single uncontrolled open-label Phase 1/2a (NCT03715985, reported in 35036074 interim n=5 and 38782542 dose-escalation n=12, 1A=15); high risk of bias from single-arm historical-control design (1B=5); total human N ~17 well under 50 (1C=2); direct match to melanoma population/route/ outcome (1D=12); modest peptide+CAF09b adjuvant preclinical base (1E=4). Mechanism 77: patient-specific neoantigens presented on MHC-I/II with functional confirmation via measured CD4+/CD8+ ELISpot responses, no knockout (2A=30); class antigen->DC->MHC->T-cell->tumor pathway mapped (2B=24); IFN-gamma magnitude correlated with peptide dose in vivo in humans (2C=13); confirmed in vivo human (2D=10). Plausibility 85: mechanism-> surrogate (T-cell response) established and surrogate->clinical ORR observed though inferred (3A=30); fully coherent with tumor immunology (3B=24); multiple same-class neoantigen vaccines show the analogous effect (Ott 2017 28678778; Sahin 2017 28678784) (3C=18); tightly scoped single-indication claim (3D=13). Global Coverage 33: EVX-01 itself is a single Evaxion-led program/one pivotal trial (4A=10); multi-country clinical sites incl. KEYNOTE-D36 protocol 36047545 / NCT05309421 (4B=12); <5 EVX-01-specific publications (4C=6); investigational trial registration (4D=5). Community Experience 17: clinical-only investigational agent with negligible documented real-world use (5A=3), short track record (5B=6), no notable recurring adverse signal over limited trial exposure (5D=8). Effectiveness basis clinical (very-low): ORR 67% (8/12) with 17% CR (2/12) vs historical ~40% pembrolizumab monotherapy, patient-relevant response endpoints, but a single-arm n=12 open-label trial with historical control only and no head-to-head comparator caps confidence. Reconciled 2026-06-27 to corrected evidence (PMID 38782542): CR rate corrected 25%->17% and fabricated multi-year durability stats removed (now "most responses not durable, one ongoing CR past 16mo"); effectiveness 58->55 (E1/E3 secondary-outcome strength down, primary 67% ORR intact). Evidence axis unchanged — corrections were citation-link fixes to the same n=12 trial at the same tier; overall 51 holds.
The proposed mechanisms of EVX-01 are based on Phase 1/2a clinical data and established neoantigen vaccine immunology. The personalized approach uses AI-driven prediction to identify patient-specific tumor targets.
How It Works (Simplified)
EVX-01 is a personalized cancer vaccine that trains the immune system to attack tumor-specific targets:
PIONEER platform analyzes tumor DNA to predict which mutations will generate immunogenic neoantigens unique to your cancer.
Long peptides are taken up by dendritic cells at injection site, processed, and presented on MHC-I and MHC-II molecules.
Both CD8+ cytotoxic and CD4+ helper T cells are activated, creating a coordinated immune response against tumor neoantigens.
Combined with anti-PD-1 therapy, vaccine-primed T cells can effectively attack tumor cells without immunosuppressive brakes.
Key Research: The Phase 1/2 trial (NCT03715985) demonstrated 100% T-cell immunogenicity and a 67% objective response rate (8/12; 6 partial, 2 complete) in melanoma when combined with anti-PD-1 therapy.
Important Limitations
- Single-arm Phase 1/2a trial with only 12 evaluable patients
- No randomized comparison to pembrolizumab alone
- Historical control comparison has inherent limitations
- Personalized manufacturing adds 6-8 weeks from biopsy to first dose
- Scalability and cost-effectiveness not yet demonstrated
Initial vaccination series (weeks 1, 2, 3, 4). Based on Phase 1/2a protocol, neoantigen-specific T-cell responses begin developing. Early immune priming occurs.
NCT03715985Continued vaccination schedule (weeks 6, 8, 11, 14). T-cell expansion and maturation. Clinical responses may begin to emerge in combination with checkpoint inhibitor.
NCT03715985Maintenance vaccinations (weeks 20, 26). In Phase 1/2a, objective responses were typically evident by week 12-24. Durable T-cell memory formation.
NCT03715985In the Phase 1/2 trial, most responses were not durable: one patient had an ongoing complete response (continuing past 16 months) while the remaining responders eventually progressed. Vaccine-induced T-cell responses were still detectable up to 14 months after the last vaccination, though their frequency declined over time.
NCT03715985Research-based observations
This timeline reflects observations from published clinical and preclinical studies. Individual responses may vary significantly. This is not a guarantee of effects or a dosing schedule. Consult qualified healthcare providers for personalized guidance.
Good Signs (6 indicators)
Warning Signs (4 indicators)
Bad Signs (6 indicators)
For Research Evaluation Only
These quality indicators are general guidelines based on typical peptide characteristics. Professional laboratory testing (HPLC, mass spectrometry) provides definitive quality verification. This checklist is for initial visual evaluation only.
Pembrolizumab
SynergisticEVX-01 is designed for combination with anti-PD-1 therapy. Vaccine primes neoantigen-specific T cells while checkpoint inhibitor removes immunosuppressive brakes. Phase 1 data from this combination.
Mrna-4157
CompatibleAlternative neoantigen vaccine platform (mRNA vs peptide). Both target personalized tumor neoantigens through different delivery mechanisms. Not typically combined.
Research Note: Interaction data is based on published literature, mechanistic understanding, and theoretical considerations. Most peptide combinations lack direct clinical study. This information is for educational purposes only and does not constitute medical advice. Always consult qualified healthcare providers.
Key Studies Cited
Full reference list available on request. All citations link to PubMed for verification.
This dossier synthesizes available evidence from peer-reviewed literature, regulatory documents, and clinical trial registries. Evidence strength ratings follow a modified GRADE approach.
For complete methodology details, see our Methodology page.
Important Disclaimer
This dossier is for educational purposes only and does not constitute medical advice. Always consult a qualified healthcare provider before making health decisions.
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