A first-in-human trial of CryptiVax-1001, an mRNA lipid nanoparticle therapeutic cancer vaccine developed by UK- and Canada-based Epitopea Ltd, has opened in the United Kingdom for patients with advanced high-grade serous ovarian cancer (HGSOC). The trial targets specifically the BRCAwt/homologous recombination proficient (HRP) subgroup — the majority of HGSOC patients — who are ineligible for PARP inhibitor maintenance and currently have no biologically matched post-chemotherapy strategy to delay recurrence.
The Phase I/Ib OVACT study (NCT07665515) will enroll approximately 50 women with FIGO Stage III–IV HGSOC, fallopian tube, or primary peritoneal cancer who have completed optimal debulking surgery and first-line platinum-based chemotherapy without progression. The trial follows a sequential dose-escalation design across five dose levels, with an optional expansion cohort at the recommended Phase II dose. A secondary immunogenicity endpoint — quantification of antigen-specific T cells in peripheral blood — will provide the first human data on whether CryptiVax-1001 elicits the targeted immune response in this population. Principal investigator is Susana Banerjee of the Royal Marsden NHS Foundation Trust.
CryptiVax-1001 encodes a panel of Cryptigen tumor-specific antigens (TSAs) — aberrantly expressed peptides derived from non-coding or previously uncharacterized regions of the genome, which Epitopea terms the "dark genome". These antigens, identified through the company's CryptoMap platform using immunopeptidomics and genomic profiling, are shared across patients rather than patient-specific, enabling an off-the-shelf format. The mRNA is delivered via lipid nanoparticles, with the LNP technology licensed from Genevant Sciences under a nonexclusive worldwide agreement announced in December 2024. Eligibility requires HLA-type matching to CryptiVax-1001, consistent with HLA-restricted antigen presentation to cytotoxic T cells, though the precise HLA alleles and T cell subsets targeted have not been publicly disclosed.