PDS0101 Plus Pembrolizumab Posts 39.3-Month Median OS in HPV16-Positive Head and Neck Cancer in JAMA Oncology
核心洞察
PDS Biotechnology's Phase 2 VERSATILE-002 results for PDS0101 plus pembrolizumab in HPV16-positive recurrent or metastatic head and neck cancer were published in JAMA Oncology.
Median overall survival reached 39.3 months in immune checkpoint inhibitor-naive patients with PD-L1 (搜索) CPS of 1 or higher, versus 17.9 months reported with standard of care.
Objective response rate was 36% by investigator review and 34% by central review, including five and six complete responders respectively.
PDS Biotechnology has published Phase 2 results for its HPV16-targeted immunotherapy PDS0101 combined with pembrolizumab in HPV16-positive recurrent or metastatic head and neck squamous cell carcinoma (搜索) (R/M HNSCC). The data appeared in the September 17, 2026 issue of JAMA Oncology, a peer-reviewed journal of the American Medical Association, under the title "VERSATILE-002 - A Phase 2 Study of PDS0101 HPV16-targeted T Cell Immunotherapy and Pembrolizumab Combination in HPV16-Positive Recurrent/Metastatic Head and Neck Squamous Cell Carcinoma: A Nonrandomized Clinical Trial."
Survival and Response Data
In the immune checkpoint inhibitor (ICI)-naive modified intention-to-treat (mITT) cohort with PD-L1 (搜索) combined positive score (CPS) of 1 or higher (n = 53), median overall survival (mOS) was 39.3 months (95% CI, 23.9 to NE). The company states the best published result with standard of care pembrolizumab, or pembrolizumab plus chemotherapy, is 17.9 months. No head-to-head trials have been performed.
Among these ICI-naive participants, 62.3% had low CPS of 1 to 19 and 37.7% had high CPS of 20 or higher.
Objective response rate (ORR) for the CPS 1 or higher population was 36%, of which 5 of 53 were complete responders by investigator review. By central review, ORR was 34%, of which 6 of 53 were complete responders. The published ORR for the overall CPS 1 or higher population treated with standard of care pembrolizumab is 19%.
Safety Profile
The combination was well tolerated. Grade 3 or higher treatment-related adverse events (TRAEs) occurred in 13.8% of patients, with one Grade 4 event reported one year after PDS0101 therapy and no Grade 5 TRAEs. The published safety profile for the overall CPS 1 or higher population treated with standard of care pembrolizumab reports 17% Grade 3 or higher TRAEs.
Trial Design and Population
VERSATILE-002 was an open-label, non-randomized Phase 2 trial conducted at twenty-six oncology centers across the United States, the United Kingdom and Ireland. Eighty-eight patients with histologically confirmed R/M HPV16-positive disease were enrolled; 87 received treatment and comprised the safety population. Seventy-five patients formed the mITT population used for efficacy analyses: 53 ICI-naive patients with PD-L1 (搜索) CPS of 1 or higher and 22 ICI-resistant patients. Mean age was 64.5 years, 94.7% were male and 92.0% were White.
The trial was led by Dr. Jared Weiss of the Lineberger Comprehensive Cancer Center, University of North Carolina, Chapel Hill. It was designed to determine the efficacy and safety of PDS0101 plus pembrolizumab, with separate evaluation of ICI-naive and ICI-resistant cohorts.
"We're very pleased with the publication of the Phase 2 clinical results of the VERSATILE-002 clinical trial in a leading peer reviewed oncology journal," said Dr. Kirk Shepard, Chief Medical Officer of PDS Biotech. "These results support the durable clinical effect of PDS0101, consistent with promising survival outcomes reported in two other recently published studies, the IMMUNOCERV study, and the NCI-led study in HPV16-positive recurrent and/or metastatic cancers. We believe PDS0101 has the potential to bring new hope to the rapidly growing population of HPV16-positive head and neck cancer patients."
Development Strategy
PDS0101 is based on the company's Versamune cancer vaccine platform and has been evaluated in HPV16-positive cancers. PDS Biotechnology intends to pursue strategic partnerships or other externally funded opportunities for Phase 3 development of PDS0101. The company recently announced the initial closing of a PIPE financing of up to $22.3 million led by Nant Capital, alongside an agreement granting NantWorks, LLC, an affiliate of Nant Capital, a one-year exclusive right to negotiate an exclusive license to PDS0101.
The company's lead development program, PDS0301 (also referred to as PDS01ADC and NHS-IL12), is an investigational tumor-targeted interleukin-12 (IL-12 (搜索)) immunocytokine designed to deliver IL-12 preferentially to the tumor microenvironment, with the goal of enhancing anti-tumor immune activity while limiting systemic exposure. PDS0301 has been clinically evaluated across multiple solid tumors, including metastatic colorectal cancer (搜索) and prostate cancer. PDS Biotechnology said it is focused on advancing PDS0301 in indications where its tumor-targeted mechanism may help address and overcome mechanisms of resistance to immunotherapy, and continues to prioritize the program in metastatic colorectal cancer.