EVX-01
InvestigationalEVX-01 is an investigational, patient-specific neoantigen peptide vaccine studied with anti-PD-1 therapy in advanced melanoma. Two small single-arm cohorts show vaccine-specific T-cell responses and observed tumor responses, but they cannot establish how much EVX-01 adds to checkpoint treatment.
EVX01 · Evaxion EVX-01
EVX-01-specific T-cell responses are documented in small human cohorts; a clinical benefit added to anti-PD-1 treatment has not been established.
- Preclinical
- 0%
- Human research
- 25%
Based on 8 cited sources
Eight linked records are three PubMed publications, two official trial registrations, one 2026 AACR sponsor poster, and two dated sponsor updates. The 2022 first-five-person report and 2024 twelve-person report concern overlapping NCT03715985 participants; the 2022 KEYNOTE-D36 publication is a phase 2 design paper, not results. The 2025 sponsor release and 2026 poster describe the same NCT05309421 cohort. The displayed Human 2 (25% of eight records) counts the two overlapping Phase 1 outcome publications, not two independent cohorts. The selected records describe two distinct clinical cohorts overall; the Phase 2 cohort appears in registration, conference-poster and sponsor-update records rather than a peer-reviewed outcome publication. The poster's 101/118 individual-neoantigen analysis covered 13 of its 15 immune-assayed participants. Two outcome-publication full texts were confirmed free in PMC; openAccess is that verified minimum, not an exhaustive audit of all eight records. The inherited 18/12/6/14 counts could not be reconstructed from linked distinct records and have been withdrawn.
Evidence score pending review
The inherited numeric evidence and effectiveness scores use unverified source counts, overlap the first-five-person and twelve-person reports, and compare uncontrolled combination outcomes with historical anti-PD-1 results. A current payload-bound rubric audit is needed before scoring.
This is a limitation of the numeric assessment, not a measured absence of benefit. It does not re-review the separately recorded catalogue classification or establish efficacy or safety.
- Study identity requires review
- Assessment incomplete
- Correction unresolved
- Community provenance requires review
- Scoring method unresolved
Status date:
Scoring context and limitationsEVX-01 is a personalized peptide mixture, not a single fixed-sequence peptide. Evaxion’s PIONEER platform selects tumor-specific neoantigens from each person’s tumor and normal-tissue data. The selected peptides are formulated with an adjuvant for clinical investigation. EVX-01 is a peptide platform; the related mRNA-4157 page describes a different platform, not a head-to-head comparator. (First-in-human study, phase 2 design paper)
What the peer-reviewed study found
NCT03715985 was a first-in-human, open-label phase 1/2 study of EVX-01 added to anti-PD-1 treatment. Safety and tolerability were its primary objective. A 2022 paper described the first five participants; the 2024 dose-escalation publication expanded the report to 12, so those papers do not represent independent cohorts. The first-five-person report recorded vaccine manufacture in 48–55 days and treatment initiation within 60 days of baseline biopsy; this is observed feasibility in that study, not a guaranteed turnaround. In the later report, all 12 had vaccine-specific T-cell responses measured in laboratory assays; 8 of 12 had an observed objective tumor response, including six partial and two complete responses. Without a concurrent anti-PD-1-only arm, that 8/12 observation cannot quantify EVX-01’s added clinical benefit. (2022 interim report, 2024 dose-escalation report, official study record)
What is newer, and how strong is it?
The separate KEYNOTE-D36 phase 2 registration, NCT05309421, describes an open-label, single-arm study in adults with unresectable or metastatic melanoma who were checkpoint-inhibitor naïve. Pembrolizumab preceded the first EVX-01 administration by 12 weeks; the registered primary endpoint concerns improvement in best overall response after that initial treatment. The official record lists 17 actual enrollees, is marked active, not recruiting, and was last posted on September 22, 2025. It has no posted results section in the checked API record. (Trial design paper, official study record)
In an April 2026 AACR poster, Evaxion reported 12 objective responses among 16 clinically analyzed phase 2 participants (75%) and vaccine-specific immune responses in 15 assayed participants. An individual-neoantigen analysis in 13 of those 15 found 101 immunogenic among 118 tested neoantigens. These are sponsor-reported conference results, not a peer-reviewed phase 2 outcomes publication or a randomized comparison. Evaxion’s October 2025 release explicitly said the registered primary response-conversion endpoint was not formally analyzed because enrollment fell below the original plan. Its August 2026 business update discussed planned further follow-up rather than a new published outcome analysis. Do not pool the 12-person phase 1 results with the 16-person phase 2 response subset or treat immune and tumor-response denominators as interchangeable. (AACR 2026 poster, October 2025 sponsor release, August 2026 sponsor update)
Why the response numbers need restraint
These studies show that EVX-01 can be manufactured for individual participants and is associated with measurable neoantigen-specific immune responses. They do not establish that it raises response rates, extends survival, or is safer than anti-PD-1 treatment alone. The 2024 publication did not use a randomized control arm; the phase 2 data currently cited are from a sponsor conference poster and company releases. A historical pembrolizumab response rate cannot substitute for a concurrent comparison. (2024 dose-escalation report, official phase 2 record, AACR 2026 poster)
Sources and record boundaries
The eight distinct linked records are the 2022 first-five-person report, 2024 dose-escalation report, 2022 KEYNOTE-D36 design paper, NCT03715985, NCT05309421, AACR 2026 sponsor poster, October 2025 sponsor release, and August 2026 sponsor update. The papers and sponsor reports reuse participants within each study. A linked record is not an independent efficacy trial.
This is an evidence summary of investigational research, not medical advice or a treatment recommendation.
- 001
No cited randomized comparison separates EVX-01's contribution from anti-PD-1 therapy. Observed response rates are not a treatment-effect estimate for the vaccine.
- 002
Phase 1 was designed primarily for safety and tolerability. The later phase 2 trial is open-label and single-arm; its response and immune data cited here are conference and sponsor reports rather than a peer-reviewed phase 2 outcomes publication.
- 003
The phase 2 registry records 17 enrolled participants, while the cited clinical poster reports response analysis in 16, immune analysis in 15, and individual-neoantigen analysis in 13 of those 15. These denominators have different scopes.
- 004
Personalized vaccine composition varies by patient; there is no single EVX-01 peptide sequence or fixed molecular weight to display.
| NCT ID | Title | Peptide | Phase | Status | Completion |
|---|---|---|---|---|---|
| NCT03715985 | Personalized Neo-antigen Vaccine in Advanced Solid Tumors (NeoPepVac) Malignant Melanoma, Metastatic, Non Small Cell Lung Cancer Metastatic +1 more | EVX-01 | Phase 1/2 | Unknown | Dec 2022 (estimated) |
| NCT05309421 | A Single Arm Trial Evaluating the Efficacy and Safety of EVX-01 in Combination With Pembrolizumab in Adults With Unresectable or Metastatic Melanoma Melanoma Stage IV, Unresectable Melanoma | EVX-01 | Phase 2 | Active not recruiting | Mar 2026 (estimated) |
Personalized Neo-antigen Vaccine in Advanced Solid Tumors (NeoPepVac)
A Single Arm Trial Evaluating the Efficacy and Safety of EVX-01 in Combination With Pembrolizumab in Adults With Unresectable or Metastatic Melanoma
Key Studies Cited
Use the source links in the research discussion above to inspect the referenced records.
This dossier synthesizes available evidence from peer-reviewed literature, regulatory documents, and clinical trial registries. Evidence strength labels summarize the cited material for this entry and should be read alongside its population, model, and study limitations.
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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