PDS Biotechnology Corporation announced updated results from its VERSATILE-002 Phase 2 clinical trial, revealing promising outcomes for Versamune® HPV (formerly PDS0101) in combination with KEYTRUDA® (pembrolizumab) as a first-line treatment for patients with HPV16-positive recurrent/metastatic (R/M) head and neck squamous cell carcinoma (HNSCC). The data, presented at the European Society for Medical Oncology (ESMO) Congress 2024, indicate a significant improvement in survival and response rates compared to historical data with pembrolizumab alone.
Key Findings from VERSATILE-002
The VERSATILE-002 trial, a single-arm Phase 2 study, enrolled 53 patients with HPV16-positive R/M HNSCC. As of the data cut on May 17, 2024, the combination of Versamune® HPV and pembrolizumab demonstrated:
- Median Overall Survival (mOS): 30 months (95% CI: 19.7 months to not reached), compared to 12-18 months historically observed with pembrolizumab.
- Objective Response Rate (ORR): 36% (19/53 patients), versus 19-25% with pembrolizumab alone.
- Disease Control Rate (DCR): 77% (41/53 patients).
- Deep Tumor Responses: 21% (11/53 patients) experienced 90-100% tumor shrinkage.
- Complete Response Rate: 9% (5/53 patients).
These results suggest a clinically meaningful benefit for patients with HPV16-positive HNSCC, a population with significant unmet medical needs. The treatment-related adverse events of Grade ≥3 were observed in 9 patients (Grade 3, n=8 and Grade 4, n=1), indicating manageable tolerability.
Expert Commentary
"The updated response data we presented at ESMO show the strong clinical activity and durability of Versamune® HPV plus pembrolizumab," said Dr. Jared Weiss, Section Chief of Thoracic and Head/Neck Oncology, Professor of Medicine at the University of North Carolina, and principal investigator of the VERSATILE-002 clinical trial. "Continued evaluation shows the promise of this combination in improving survival for patients with HPV16-positive HNSCC."
VERSATILE-003: A Phase 3 Trial on the Horizon
Building on these encouraging Phase 2 results, PDS Biotech is planning to initiate the VERSATILE-003, a global, randomized, controlled Phase 3 clinical trial. This trial will evaluate Versamune® HPV plus pembrolizumab versus pembrolizumab monotherapy as a first-line treatment in patients with HPV16-positive R/M HNSCC with CPS ≥1.
Implications for HNSCC Treatment
Head and neck squamous cell carcinoma is a challenging disease, and HPV16-positive tumors represent a distinct and growing subset. The current standard of care, pembrolizumab, offers limited survival benefits, highlighting the need for more effective therapies. Versamune® HPV, with its unique mechanism of action, has the potential to address this unmet need by enhancing the immune response against HPV16-positive cancer cells.
"We’re encouraged to see that as the data from our VERSATILE-002 clinical trial have matured, responses continue to improve, suggesting durability of the Versamune® HPV induced anti-tumor immune response," said Dr. Kirk Shepard, M.D., Chief Medical Officer of PDS Biotech. "The encouraging patient survival and clinical responses coupled with promising tolerability as seen in the VERSATILE-002 trial underscore our belief in the potential of the combination to be the first HPV-targeted immunotherapy for HNSCC, and a significant advancement in the treatment of the growing population of patients with HPV16-positive HNSCC. We are working toward initiating the VERSATILE-003 Phase 3 study this year."
