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Published on: July 7, 2023
Clinical phase I study of elpamotide, a peptide vaccine for vascular endothelial growth factor receptor 2, in
Isamu Okamoto1, Tokuzo Arao, Masaki Miyazaki
1Department of Medical Oncology, Kinki University Faculty of Medicine, Osaka, Japan.
Abstract:
Targeting of tumor angiogenesis with vaccines is a potentially valuable approach to cancer treatment. Elpamotide is an immunogenic peptide derived from vascular endothelial growth factor receptor 2, which is expressed at a high level in vascular endothelial cells. We have now carried out a phase I study to evaluate safety, the maximum tolerated dose, and potential pharmacodynamic biomarkers for this vaccine. Ten HLA-A*24:02-positive patients with advanced refractory solid tumors received elpamotide s.c. at dose levels of 0.5, 1.0, or 2.0 mg once a week on a 28-day cycle. Five patients experienced an injection site reaction of grade 1 and 2, which was the most frequent adverse event. In the 1.0 mg cohort, one patient experienced proteinuria of grade 1 and another patient developed both hypertension and proteinuria of grade 1. No adverse events of grade 3 or higher were observed, and the maximum tolerated dose was therefore not achieved. The serum concentration of soluble vascular endothelial growth factor receptor 2 decreased significantly after elpamotide vaccination. Microarray analysis of gene expression in PBMCs indicated that several pathways related to T cell function and angiogenesis were affected by elpamotide vaccination, supporting the notion that this peptide induces an immune response that targets angiogenesis in the clinical setting. In conclusion, elpamotide is well tolerated and our biomarker analysis indicates that this anti-angiogenic vaccine is biologically active. Clinical trial registration no. UMIN000008336.
Insights
Elpamotide, a novel cancer vaccine targeting tumor angiogenesis, demonstrated good tolerability in a Phase I trial. Biomarker analysis confirmed its biological activity, showing a decrease in soluble vascular endothelial growth factor receptor 2 levels.
Area of Science:
- Oncology
- Immunology
- Vaccine Development
Background:
- Tumor angiogenesis is a critical target for cancer treatment.
- Elpamotide is a peptide vaccine targeting vascular endothelial growth factor receptor 2 (VEGFR2).
Purpose of the Study:
- To evaluate the safety, maximum tolerated dose, and pharmacodynamic biomarkers of elpamotide in patients with advanced solid tumors.
- To assess the biological activity of elpamotide as an anti-angiogenic vaccine.
Main Methods:
- Phase I clinical trial involving ten HLA-A*24:02-positive patients.
- Elpamotide administered subcutaneously at escalating doses (0.5, 1.0, 2.0 mg) weekly.
- Safety assessments, serum VEGFR2 measurements, and gene expression analysis in peripheral blood mononuclear cells (PBMCs).
Main Results:
- Elpamotide was well-tolerated, with most frequent adverse events being grade 1-2 injection site reactions.
- No grade 3 or higher adverse events were observed; maximum tolerated dose was not reached.
- Significant decrease in serum soluble VEGFR2 levels and modulation of T cell and angiogenesis-related gene pathways in PBMCs.
Conclusions:
- Elpamotide is a safe and well-tolerated anti-angiogenic vaccine.
- Biomarker data support elpamotide's biological activity and its potential to induce an immune response targeting tumor angiogenesis.
