PDS0101/Pembrolizumab Achieves Preliminary Objective Response End Point for Advanced Head and Neck Cancer

PDS Biotechnology Corporation announced that the combination of PDS0101 and pembrolizumab achieved preliminary response efficacy for patients with advanced human papillomavirus–associated head and neck cancer.

Results from the phase 2 VERSATILE-002 trial (NCT04260126), investigating PDS0101 in combination with pembrolizumab (Keytruda) for patients with advanced human papillomavirus (HPV)–associated head and neck cancer, met its preliminary objective response end points, according to a news release from developer PDS Biotechnology Corporation.1

The trial will now progress to full enrollment and feature 54 patients with head and neck cancer in the checkpoint inhibitor–naïve arm based on a pre-specified clinical design to confirm achievement of objective responses. The clinical-stage immunotherapy company that develops novel cancer therapies and vaccines for infectious disease expects that the full enrollment data will be presented at an upcoming medical conference.

“The achievement of this important milestone in the VERSATILE-002 phase 2 clinical trial strengthens the evidence of our novel Versamune® platform’s potential ability to induce high levels of tumor-specific CD8+ killer T-cells that attack the cancer to achieve tumor regression,” Lauren V. Wood, MD, chief medical officer at PDS Biotech, said in the news release. “The initial data solidifies our belief that PDS0101 [has] demonstrated preclinical efficacy when combined with [pembrolizumab] has the potential to significantly improve clinical outcomes for patients with advanced HPV16-positive head and neck cancers.”

Achievement of objective response was measured by radiographic tumor responses according to RECIST 1.1 criteria. Response needed to be confirmed by 2 different measurements among at least 4 of the first 17 patients in the checkpoint inhibitor–naïve arm to advance the study to full enrollment.

PDS0101 is a liposomal nanoparticle-based vaccine that encapsulates 6 HPV16 peptides.2

VERSATILE-002 is focused on 2 groups of patients with HPV16-positive head and neck cancer with disease that has spread or returned, one of which was added via a protocol amendment in the summer of 2021. The first group featured patients who did not previously receive checkpoint inhibitors. The second group included 21 patients who were refractory to checkpoint inhibitors, with assessment currently ongoing.

Eligibility criteria required patients to be aged 18 years or older with histologically confirmed recurrent, metastatic, or persistent head and neck squamous cell carcinoma with HPV16 infection or PD-L1 expression. Patients also needed to have adequate organ function and an ECOG performance status of 0 or 1.

Exclusion criteria stated that on patients who received prior radiotherapy within 2 weeks, or those who received live vaccines or any previous systemic anticancer therapy within 30 days could not be included in the study.


  1. PDS Biotech announces preliminary efficacy achievement in VERSATILE-002 phase 2 trial of PDS0101 in combination with KEYTRUDA® (pembrolizumab) in advanced head and neck cancer. News release. PDS Biotechnology Corporation. February 2, 2022. Accessed February 7, 2022. https://tinyurl.com/5y54jwpc
  2. 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 February 7, 2022. https://bit.ly/34ILuVd
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