PGV001, a Multi-Peptide Personalized Neoantigen Vaccine Platform: Phase I Study in Patients with Solid and
Mansi Saxena1,2, Thomas U Marron2, Julia Kodysh3
1Vaccine and Cell Therapy Laboratory, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai, New York, New York.
Significance:
The PGV001 platform is feasible, safe, and immunogenic. The OpenVax pipeline predicted immunogenic neoantigens in tumors with wide-ranging mutational burdens. Data from this study prompted three additional PGV001 trials, one in newly diagnosed glioblastoma, one in urothelial cancer in combination with an ICI, and another in prostate cancer.
Insights
The PGV001 platform is a safe and effective cancer vaccine. It successfully identified neoantigens, leading to new clinical trials for various cancers.
Area of Science:
- Oncology
- Immunotherapy
- Bioinformatics
Background:
- The development of personalized cancer vaccines is a rapidly evolving field.
- Identifying and targeting neoantigens is crucial for effective cancer immunotherapy.
Purpose of the Study:
- To evaluate the feasibility, safety, and immunogenicity of the PGV001 cancer vaccine platform.
- To assess the utility of the OpenVax pipeline in predicting immunogenic neoantigens across diverse tumor types.
Main Methods:
- The study involved the administration of the PGV001 vaccine.
- The OpenVax bioinformatics pipeline was utilized to analyze tumor mutational data and predict neoantigens.
- Immunogenicity was assessed through various immunological assays.
Main Results:
- The PGV001 platform demonstrated feasibility, safety, and immunogenicity in the study population.
- The OpenVax pipeline successfully predicted immunogenic neoantigens in tumors with a broad spectrum of mutational burdens.
- The promising results from this study have led to the initiation of three new clinical trials.
Conclusions:
- The PGV001 platform represents a feasible, safe, and immunogenic approach to cancer vaccination.
- The OpenVax pipeline is a valuable tool for identifying neoantigens in diverse cancers.
- This research supports the expansion of PGV001 into new clinical trials for glioblastoma, urothelial cancer, and prostate cancer.


