FDA Grants Fast Track Status to PDS0101 Plus Pembrolizumab for Recurrent/Metastatic HPV16+ HNSCC


The FDA has granted fast track designation to the combination of PDS0101 and pembrolizumab (Keytruda) for the treatment of patients with recurrent or metastatic HPV16-positive head and neck squamous cell carcinoma (HNSCC), according to a press release issued by PDS Biotechnology Corporation.1


Trial Name: A Phase II, Open-Label, Multi-Center Study of PDS0101 and Pembrolizumab (KEYTRUDA®) Combination Immunotherapy in Subjects With Recurrent and/or Metastatic HNSCC and High-Risk HPV16 Infection 

Indentifier: NCT04260126

Completion Date: July 2024

Sponsor: PDS Biotechnology Corp. and Merck Sharp & Dohme LLC

Recruitment Contact: Lauren V Wood, MD of PDS Biotechnology Corporation

PDS0101 in combination with pembrolizumab is currently being assessed in the phase 2 VERSATILE-002 clinical trial, and preliminary findings show that a tumor reduction rate of 30% or more was achieved with the therapy in at least 4 of the first 17 patients, making the study eligible to proceed to full enrollment.

“We are thrilled that the FDA has granted fast track designation for PDS0101 in combination with Keytruda,” said Frank Bedu-Addo, PhD, chief executive officer of PDS Biotech, in a press release. “The HPV-associated head and neck cancer prevalence continues to rise, leaving this affected group with limited treatment options to date. Receiving this designation underscores the potential of the Versamune platform and the need for a new therapy that may improve outcomes for those with this devastating disease.”

In VERSATILE-002, an open-label, multicenter study (NCT04260126), approximately 95 patients with recurrent and/or metastatic HNSCC will be included. All patients will be treated with pembrolizumab 200 mg via intravenous infusion plus 2 0.5 mL subcutaneous injections of PDS0101 on cycles 1, 2, 3, 4, and 12. Pembrolizumab monotherapy will also be administered to patients in the study during cycles 5 through 11 and 13 through 35.2

The primary end point of the study is best overall response within a 24-month time frame, per RECIST v1.1 criteria. The secondary end points of the study include progression-free survival (PFS) at 12 and 24 months per RECIST v1.1, overall survival, and the incidence of treatment-emergent adverse events. Other outcome measures to be explored include duration of response, and anti-HPV-16 E6 and E7 immune response induced by the combination of PDS0101 and pembrolizumab.

Patients with histological or cytologically confirmed HNSCC aged 18 years or older are eligible to enroll given they have confirmed HPV16 infection, confirmed tumor PD-L1 expression defined by a combined positive score of ≥ 1, and have not received prior immunological therapy for metastatic disease. Patients who received prior treatment with a checkpoint inhibitor must have received at least 2 doses of the agent or underwent treatment for at least 6 weeks and have documented clinical progression or recurrence that has been radiologically confirmed.

All patients are also required to have recurrent and/or metastatic measurable disease, and adequate organ function. In addition, all patients are required to have recovered from any toxicities or complications related to major surgery or radiation therapy of > 30 Gy before they can be eligible for enrollment.

The VERSATILE-002 is actively recruiting patients at 22 locations across the United States. The study is being led by Jared Weiss, MD, associate professor, Division of Oncology and section chief of Thoracic and Head and Neck Oncology at the University of Carolina at Chapel Hill School of Medicine and Lineberger Comprehensive Cancer Center.


1. PDS Biotechnology granted FDA fast track designation for lead candidate PDS0101. News release. June 2, 2022. Accessed June 2, 2022. https://bit.ly/3Qaladh

2. Study of PDS0101 and pembrolizumab combination i/o in subjects with HPV16 + recurrent and/or metastatic HNSCC (VERSATILE002). Clinicaltrials.gov. Updated April 29, 2022. Accessed June 2, 2022. https://clinicaltrials.gov/ct2/show/NCT04260126

Related Videos
Related Content