PDS Biotech's PDS0101 combo shows 39-month survival in HNSCC trial

VERSATILE-002 Phase 2 results, published in JAMA Oncology, showed 39.3 months median overall survival in HPV16-positive head and neck cancer, more than

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A brightly lit medical imaging room contains a large white and gray CT scanner with an attached patient table, and blurred medical equipment and a desk with monitors in the background.

PDS Biotechnology has announced the peer-reviewed publication of Phase 2 data for its HPV16-targeted immunotherapy PDS0101, used in combination with Merck's pembrolizumab in patients with recurrent or metastatic head and neck squamous cell carcinoma (R/M HNSCC). The results appeared in the 17 September 2026 issue of JAMA Oncology.

The VERSATILE-002 trial enrolled 88 patients with histologically confirmed HPV16-positive R/M disease across 26 oncology centres in the US, UK and Ireland. The key efficacy population comprised 53 immune checkpoint inhibitor (ICI)-naïve patients with PD-L1 combined positive score (CPS) of 1 or above. In that group, the combination produced a median overall survival (mOS) of 39.3 months (95% CI, 23.9 to not estimable). The company cites published standard-of-care data showing pembrolizumab alone or combined with chemotherapy achieves a best mOS of 17.9 months in a comparable population, noting that no direct head-to-head comparison has been conducted.

Efficacy and safety data

Objective response rate (ORR) in the overall CPS 1-or-above population reached 36% by investigator review, with five of 53 patients achieving a complete response. Central review confirmed an ORR of 34%, with six complete responders. This compares with a published ORR of 19% for pembrolizumab monotherapy in the same PD-L1-unselected population, according to a 2022 reference cited by the company.

The safety profile appeared manageable. Grade 3 or higher treatment-related adverse events occurred in 13.8% of patients, below the 17% rate reported for pembrolizumab alone in the comparable published dataset. There was one Grade 4 event, occurring more than a year after PDS0101 dosing, and no Grade 5 events.

Kirk Shepard, Chief Medical Officer of PDS Biotech, said the publication was "consistent with promising survival outcomes reported in two other recently published studies," referencing the IMMUNOCERV study and an NCI-led trial in HPV16-positive R/M cancers, though neither dataset was summarised in the release.

Pipeline strategy and market context

PDS Biotech is positioning PDS0101 for out-licensing rather than self-funded Phase 3 development. The company recently completed an initial closing of a PIPE financing of up to $22.3 million led by Nant Capital, alongside an exclusive one-year negotiating window for NantWorks, an affiliate of Nant Capital, on an exclusive licence to PDS0101. The company's internal pipeline priority has shifted to PDS0301, an IL-12 immunocytokine being advanced in metastatic colorectal cancer.

HPV16-positive HNSCC is a distinct and growing segment within head and neck oncology. Unlike tobacco-associated HNSCC, HPV-driven disease typically affects a younger demographic and has historically shown better responses to immune checkpoint blockade, making it an attractive target for combination immunotherapy strategies. Several academic and commercial groups are investigating tumour-infiltrating lymphocyte therapies and therapeutic vaccines in this indication, creating a competitive backdrop against which PDS0101's data will need to be assessed at the Phase 3 level.

The non-randomised design of VERSATILE-002 limits the strength of cross-trial comparisons, a point likely to feature in any regulatory dialogue. PDS Botech has said it intends to pursue a Phase 3 partnership; the NantWorks negotiating window and the JAMA Oncology publication together represent a clear effort to strengthen its licensing position ahead of that conversation.

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