Author: Brielle Benyon

Results from a phase 2 clinical trial demonstrated promise for the combination of the novel agent PDS0101 plus Keytruda (pembrolizumab) in treating human papillomavirus (HPV)-associated head and neck cancer. As such, the trial will now progress to full enrollment of 54 patients who have not been previously treated with a checkpoint inhibitor.

The trial, VERSATILE-002, involves two groups of HPV16-positive patients with head and neck cancer that is either metastatic or has returned after treatment. One group consists of patients who have no prior treatment with checkpoint inhibition immunotherapy, while the other group is made up of 21 patients whose disease failed checkpoint inhibition — assessment for this group is still ongoing.

In the checkpoint inhibitor-naïve group, four or more of the 17 patients achieved an objective response, which was classified by a 30% or more reduction in tumor size.

“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,” commented Dr. Lauren V. Wood, Chief Medical Officer of PDS Biotech, the developer of PFS101, in a statement. “The initial data solidifies our belief that PDS0101’s demonstrated preclinical efficacy when combined with Keytruda has the potential to significantly improve clinical outcomes for patients with advanced HPV16-positive head and neck cancers.”

PDS0101 works by inducing large quantities of CD4+ helper and CD8+ killer T cells, a type of white blood cells that are key in the functioning of the immune system. This works in tandem with checkpoint inhibitors like Keytruda, which help unmask the cancer cells to the immune system, allowing the body to find and fight the cancer.

Upcoming research will determine which regimen is better at fighting cancer: Keytruda plus PDS0101 or Keytruda alone. Eligible patients must have HPV16-positive head and neck cancer that has come back or spread, have recovered from complications from previous major surgeries or radiation and have good overall health, as determined by a blood test.

Patients on the trial are set to receive intravenous (IV) Keytruda every three weeks for up to 35 treatments (about two years). Those in the PDS0101 group are also anticipated to receive the drug, via IV, in combination with Keytruda for the first four cycles of treatment and again at cycle 12. The plan is for bloodwork to be conducted before and after each treatment, and patients should also undergo imaging studies every nine weeks for the first year so that their clinicians can evaluate the status of their cancer.

Treatment on the trial is planned to be administered until a patient experiences disease progression or until they received the maximum limit of 35 treatments.

The lead investigator of the expanded trial is Dr. Jared Weiss from the University of Carolina at Chapel Hill School of Medicine and Lineberger Comprehensive Cancer Center, but that does not mean that patients who are interested must be nearby. There are 27 sites across the U.S. that are involved in the trial. Patients interested in enrolling can email: