PDS Biotechnology Corporation announced on October 29, 2025, that it has requested a meeting with the FDA to explore an expedited approval pathway for its lead immunotherapy candidate PDS0101 in HPV16-positive head and neck cancer. The request follows compelling final results from the company's VERSATILE-002 Phase 2 trial, which demonstrated unprecedented survival outcomes in this challenging patient population.
Breakthrough Survival Data Drives Regulatory Strategy
The final VERSATILE-002 trial data revealed a median overall survival of 39.3 months and median progression-free survival of 6.3 months in patients with unresectable, recurrent or metastatic HPV16-positive head and neck cancer. According to Frank Bedu-Addo, PhD, President and Chief Executive Officer of PDS Biotech, "It's the first time that a trial in the recurrent/metastatic Head and Neck Cancer population has reported a mOS of almost 40 months."
The VERSATILE-002 trial (NCT04260126) was an open-label, multi-center Phase 2 study evaluating PDS0101, an HPV16-targeted immunotherapy, in combination with pembrolizumab. The trial assessed the combination therapy's impact on patients who were either naive to or refractory to immune checkpoint inhibitors.
Proposed Trial Amendment to Accelerate Approval Timeline
Based on these results, PDS Biotechnology has proposed amending its ongoing VERSATILE-003 Phase 3 trial to include progression-free survival as an earlier primary endpoint alongside median overall survival. The proposed changes would also reduce the number of patients while maintaining statistical power. If the PFS endpoint is achieved, it would enable the company to submit for accelerated FDA approval.
"The fact that our Phase 3 study has mOS as its primary endpoint and PFS as a secondary endpoint inherently lengthens the duration of the trial," explained Bedu-Addo. "To shorten the trial duration, we believe a meeting with the FDA to discuss changes to the current trial protocol to include PFS as an earlier primary endpoint independent of mOS is warranted."
The VERSATILE-003 trial has been temporarily paused pending FDA review of the proposed protocol amendments, though treatment for currently enrolled patients will continue during this pause.
Addressing Growing Clinical Need
The regulatory strategy reflects the increasing clinical importance of HPV16-positive head and neck cancer, which is projected to become the dominant type of head and neck cancer in the United States by the mid-2030s. Dr. Kirk Shepard, Chief Medical Officer, emphasized the urgency: "Based on the increasing incidence of HPV16-positive head and neck cancer, our goal is to seek the fastest and most cost-effective regulatory pathway to approval."
The company aims to provide patients with a well-tolerated treatment option without chemotherapy for a disease where effective therapies are currently limited. PDS0101 is being developed as a targeted immunotherapy that works by enhancing the immune system's ability to target and eliminate cancer cells.
Company Pipeline and Development Strategy
PDS Biotechnology is a late-stage immunotherapy company focused on transforming immune system targeting of cancers. Beyond PDS0101, the company is developing PDS01ADC, an IL-12 fused antibody drug conjugate, which is being evaluated in multiple Phase 2 trials across various cancer indications in combination with standard of care treatments.
The full VERSATILE-002 dataset is mature and expected to be submitted for publication later this year, providing the broader medical community with detailed insights into the treatment's efficacy and safety profile in this patient population.
