VERSATILE-002 Trial to Enroll Previously Treated HPV+ HNSCC Per Protocol Amendment


A new treatment arm of a phase 2 trial examining PDS0101 plus pembrolizumab in patients with HPV-related head and neck cancers will be opened to patients with immune checkpoint inhibitor–pretreated disease.

A protocol amendment to the VERSATILE-002 trial (NCT04260126) of PDS0101 plus pembrolizumab (Keytruda) for certain patients with head and neck cancers will allow enrollment of patients who have failed prior checkpoint inhibitor therapy to an additional study arm, according to PDS Biotechnology Corporation.1

The liposomal nanoparticle-based PDS0101 vaccine encapsulating 6 human papillomavirus virus 16 (HPV-16) peptides is for the treatment of patients with HPV-related cancers.2 Initially, the nonrandomized, multicenter, open-label, phase 2 trial was opened to examine efficacy of the combination in patients with treatment-naïve HPV-related head and neck malignancies.

“There is an enormous unmet medical need in advanced head and neck cancer patients who have failed multiple therapies, including chemotherapy, radiation, and checkpoint inhibitor therapy. We believe the combination of PDS0101 and Keytruda has the potential to significantly improve clinical outcomes for these patients who have limited treatment options,” Lauren Wood, MD, Chief Medical Officer of PDS Biotech, said in a press release.

The additional study arm will evaluate the objective response rate (ORR) resulting from PDS0101 plus pembrolizumab treatment in roughly 40 patients with refractory head and neck cancers who have failed multiple therapies, including immunotherapy. The ORR will be measured by radiographic tumor response per RECIST 1.1. The first 21 patients in the treatment arm will be evaluated for safety and response rate before progressing to full enrollment.

To be eligible for enrollment, patients must be at least 18 years of age; have histologically or cytologically confirmed squamous cell carcinoma of the head and neck that is recurrent, metastatic, or persistent with HPV16 infection or PD-L1 expression. Additionally, adequate organ function and an ECOG performance status of 0 or 1.

Patients receiving prior radiotherapy within 2 weeks or live vaccines or any prior systemic anticancer within 30 days are excluded from the trial. Other known malignancies within 3 years and hematopoietic stem cell transplant within 5 years are not allowed.

Results of a phase 1/2 trial of PDS0101 in combination with 2 other investigational immunotherapies, M9241 and bintrafusp a, in 14 patients will be reported at the upcoming 2021 American Society of Clinical Oncology Annual Meeting.3 The trial population included those with cervical, vaginal/vulvar, anal, and oropharyngeal cancers with checkpoint inhibitor–refractory and -naïve disease.

Promising responses were noted in those with and without prior experience with an immunotherapy agent. In those whose disease had failed on chemotherapy, radiation, and checkpoint inhibitors (n = 8), the ORR was 63%. Additionally, the combination showed a manageable safety profile in the examined patient population.

VERSATILE-002 is currently being run at approximately 20 sites in the United States with Jared Weiss, MD, of University of North Carolina at Chapel Hill School of Medicine and Lineberger Comprehensive Cancer Center, serving as principal investigator.


1. PDS Biotechnology Expands VERSATILE-002 study of PDS0101 and KEYTRUDA in Advanced Head and Neck Cancer to Include Patients Who Have Failed Prior Treatment with Checkpoint Inhibitors. News release. PDS Biotechnology Corporation. June 3, 2021. Accessed June 3, 2021.

2. Liposomal HPV-16 E6/E7 multipeptide vaccine PDS0101. National Cancer Institute. Accessed June 3, 2021.

3. Strauss J, Floudas CS, Sater HA, et al. Phase II evaluation of the triple combination of PDS0101, M9241, and bintrafusp alfa in patients with HPV 16 positive malignancies. J Clin Oncol. 2021;39(suppl 15):2501. doi:10.1200/JCO.2021.39.15_suppl.2501

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