PDS Biotech Announces Submission of Phase 3 Protocol to FDA to Initiate VERSATILE-003 Trial

PDS Biotechnology Corporation recently announced the submission to the US FDA of an updated Chemistry, Manufacturing and Controls (CMC) package and a Phase 3 multicenter registrational protocol to the company’s Investigational New Drug (IND) submission to evaluate the combination of PDS0101 and KEYTRUDA (pembrolizumab), Merck’s anti-PD-1 therapy, for the treatment of recurrent or metastatic human papillomavirus (HPV) 16-positive head and neck squamous cell carcinoma (HNSCC). The protocol was developed in accordance with guidance from the FDA on key elements of the Phase 3 program to support the eventual submission of a Biologics License Application (BLA).

The Phase 3 trial, named VERSATILE-003, is a randomized, active comparator-controlled study designed to investigate the safety and efficacy of PDS0101 combined with KEYTRUDA compared to KEYTRUDA monotherapy in immune checkpoint inhibitor (ICI)-naïve patients with recurrent or metastatic HPV16-positive HNSCC. The primary efficacy endpoint for VERSATILE-003, per the protocol, is overall survival (OS). The Phase 3 study is expected to involve approximately 90-100 clinical sites globally. PDS Biotech anticipates initiating the VERSATILE-003 Phase 3 trial in the fourth quarter of 2023.

“Submission of the protocol and supportive CMC documents for this Phase 3 registrational trial is an important milestone for PDS Biotech and our VERSATILE-003 program investigating PDS0101 in combination with KEYTRUDA as a potential treatment for recurrent or metastatic HPV16-positive HNSCC,” stated Dr. Lauren V. Wood, PDS Biotech’s Chief Medical Officer. “Interim data from our ongoing VERSATILE-002 Phase 2 clinical trial have been very encouraging, with impressive interim OS and PFS results. With VERSATILE-003, we have an opportunity to confirm the Phase 2 results from VERSATILE-002 in a controlled, Phase 3 clinical trial comparing the combination of PDS0101 and KEYTRUDA to KEYTRUDA monotherapy.”

PDS0101, PDS Biotech’s lead candidate, is a novel investigational human papillomavirus (HPV)-targeted immunotherapy that stimulates a potent targeted T cell attack against HPV-positive cancers. PDS0101 is given by subcutaneous injection alone or in combination with other immunotherapies and cancer treatments. In a Phase 1 study of PDS0101 in monotherapy, the treatment demonstrated the ability to generate multifunctional HPV16-targeted CD8 and CD4 T cells with minimal toxicity. Interim data suggest PDS0101 generates clinically effective immune responses, and the combination of PDS0101 with other treatments can demonstrate significant disease control by reducing or shrinking tumors, delaying disease progression and/or prolonging survival. The combination of PDS0101 with other treatments does not appear to compound the toxicity of other agents.

VERSATILE-002 is a single-arm Phase 2 trial evaluating the safety and efficacy of PDS0101, an HPV16-targeted investigational T cell-activating immunotherapy that leverages PDS Biotech’s proprietary Versamune technology, in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab). The combination is being evaluated in immune checkpoint inhibitor (ICI)-naïve and ICI-refractory patients with recurrent/metastatic HPV16-positive head and neck squamous cell carcinoma (HNSCC) and was granted Fast Track designation by the FDA in June 2022.

Interim efficacy and safety data were presented at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting for ICI-naïve patients. Preliminary data from the first 34 patients demonstrated a 12-month overall survival rate of 87% and median progression free survival of 10.4 months. No Grade 4 or higher treatment related adverse events were observed, and Grade 3 treatment related adverse events were observed in 8% of patients.

VERSATILE-003 is a randomized, controlled Phase 3 trial evaluating the safety and efficacy of PDS0101 in combination with Merck’s anti-PD-1 therapy, KEYTRUDA (pembrolizumab) versus KEYTRUDA monotherapy. The combination is being evaluated in immune checkpoint inhibitor (ICI)-naïve patients with recurrent/metastatic HPV16-positive head and neck squamous cell carcinoma (HNSCC) and was granted Fast Track designation by the FDA in June 2022.

PDS Biotech is a clinical-stage immunotherapy company developing a growing pipeline of targeted cancer and infectious disease immunotherapies based on our proprietary Versamune, Versamune plus PDS0301, and Infectimune T cell-activating platforms. We believe our targeted immunotherapies have the potential to overcome the limitations of current immunotherapy approaches through the activation of the right type, quantity and potency of T cells. To date, our lead Versamune clinical candidate, PDS0101, has demonstrated the ability to reduce and shrink tumors and stabilize disease in combination with approved and investigational therapeutics in patients with a broad range of HPV16-associated cancers in multiple Phase 2 clinical trials and will be advancing into a Phase 3 clinical trial in combination with KEYTRUDA for the treatment of recurrent/metastatic HPV16-positive head and neck cancer in 2023. Our Infectimune-based vaccines have also demonstrated the potential to induce not only robust and durable neutralizing antibody responses, but also powerful T cell responses, including long-lasting memory T cell responses in pre-clinical studies to date. For more information, visit www.pdsbiotech.com.