Personalized therapy with peptide-based neoantigen vaccine (EVX-01) including a novel adjuvant, CAF®09b, in patients with metastatic melanoma

Sofie Kirial Mørk, Mohammad Kadivar, Kalijn Fredrike Bol, Arianna Draghi, Marie Christine Wulff Westergaard, Signe Koggersbøl Skadborg, Nana Overgaard, Anders Bundgård Sørensen, Ida Svahn Rasmussen, Lars Vibe Andreasen, Christina Westmose Yde, Thomas Trolle, Christian Garde, Jens Friis-Nielsen, Nis Nørgaard, Dennis Christensen, Jens Vindahl Kringelum, Marco Donia, Sine Reker Hadrup, Inge Marie Svane*

*Corresponding author for this work

Research output: Contribution to journalJournal articleResearchpeer-review

70 Downloads (Pure)


The majority of neoantigens arise from unique mutations that are not shared between individual patients, making neoantigen-directed immunotherapy a fully personalized treatment approach. Novel technical advances in next-generation sequencing of tumor samples and artificial intelligence (AI) allow fast and systematic prediction of tumor neoantigens. This study investigates feasibility, safety, immunity, and anti-tumor potential of the personalized peptide-based neoantigen vaccine, EVX-01, including the novel CD8+ T-cell inducing adjuvant, CAF®09b, in patients with metastatic melanoma (NTC03715985). The AI platform PIONEERTM was used for identification of tumor-derived neoantigens to be included in a peptide-based personalized therapeutic cancer vaccine. EVX-01 immunotherapy consisted of 6 administrations with 5–10 PIONEERTM-predicted neoantigens as synthetic peptides combined with the novel liposome-based Cationic Adjuvant Formulation 09b (CAF®09b) to strengthen T-cell responses. EVX-01 was combined with immune checkpoint inhibitors to augment the activity of EVX-01-induced immune responses. The primary endpoint was safety, exploratory endpoints included feasibility, immunologic and objective responses. This interim analysis reports the results from the first dose-level cohort of five patients. We documented a short vaccine manufacturing time of 48–55 days which enabled the initiation of EVX-01 treatment within 60 days from baseline biopsy. No severe adverse events were observed. EVX-01 elicited long-lasting EVX-01-specific T-cell responses in all patients. Competitive manufacturing time was demonstrated. EVX-01 was shown to be safe and able to elicit immune responses targeting tumor neoantigens with encouraging early indications of a clinical and meaningful antitumor efficacy, warranting further study.
Original languageEnglish
Article number2023255
Issue number1
Number of pages17
Publication statusPublished - 2022


  • Personalized therapy
  • Neoantigen
  • Immune response
  • Cancer vaccine
  • Immunotherapy


Dive into the research topics of 'Personalized therapy with peptide-based neoantigen vaccine (EVX-01) including a novel adjuvant, CAF®09b, in patients with metastatic melanoma'. Together they form a unique fingerprint.

Cite this