PDS Biotechnology Corporation (Nasdaq: PDSB) announced today that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for a novel combination therapy targeting metastatic colorectal cancer (mCRC). The therapy combines Versamune® MUC1 (formerly PDS0103), a MUC1-targeted immunotherapy candidate, with PDS01ADC to treat MUC1-positive unresectable, metastatic colorectal carcinoma in patients who have failed previous treatment.
The IND application, filed in January 2025, represents a significant advancement for PDS Biotech's immunotherapy platform and its potential applications beyond HPV-related cancers. The National Cancer Institute (NCI) will lead the Phase 1/2 clinical trial under a Cooperative Research and Development Agreement (CRADA) with PDS Biotech.
Addressing a Critical Unmet Need
Colorectal cancer remains one of the leading causes of cancer-related deaths worldwide, with limited effective options for patients with metastatic disease who have progressed on initial therapies. The new combination specifically targets MUC1-positive tumors in patients with Proficient Mismatch Repair/Microsatellite Stable mCRC, which accounts for approximately 95% of metastatic colorectal cancer cases.
"MUC1 is over-expressed in multiple solid tumors including colon, pancreatic, ovarian, breast, and NSCLC, and is associated with drug resistance and poor patient outcomes," explained Kirk Shepard, M.D., PDS Biotech's Chief Medical Officer. "This results in an unmet need for more effective, safer, better-tolerated targeted treatment options."
Dr. Shepard highlighted that these tumors are typically more resistant to current immunotherapies, such as immune checkpoint inhibitors, and second-line chemotherapy. "Targeting MUC1 with an immunotherapy that elicits a strong and durable tumor-infiltrating T-cell response could represent a major advancement in cancer treatment," he added.
Mechanism of Action and Patent Protection
The Versamune® platform is designed to activate the immune system to target specific cancer antigens. In this case, the therapy targets MUC1, a protein that is abnormally expressed in various cancer types and associated with aggressive disease.
PDS Biotech recently secured U.S. Patent #12,201,685, which covers the methods of using the proprietary combination of Versamune® and cytokines to overcome immune suppression in the tumor microenvironment and improve anti-tumor immune responses. This patent protection strengthens the company's intellectual property position as it advances its pipeline.
Strategic Focus and Pipeline Development
While this IND clearance marks an important milestone, PDS Biotech's primary focus remains on its recently initiated VERSATILE-003 Phase 3 clinical trial in HPV16-positive head and neck squamous cell carcinoma.
"The IND clearance of Versamune® MUC1 marks progress for our Versamune® platform and its potential to expand beyond HPV-related cancers," said Frank Bedu-Addo, PhD, President and Chief Executive Officer of PDS Biotech. "Though our focus remains on our recently initiated VERSATILE-003 Phase 3 clinical trial in HPV16-positive head and neck squamous cell carcinoma, we are pleased to support the NCI investigation of a therapy that shows promise in driving strong, durable anti-tumor immune responses."
Trial Design and Patient Population
The Phase 1/2 clinical trial will evaluate the combination of Versamune® MUC1 and PDS01ADC in patients with recurrent or metastatic colorectal cancer. The study will focus on patients whose tumors express the MUC1 protein and who have failed previous treatment regimens.
This patient population represents a significant unmet medical need, as current immunotherapy approaches have shown limited efficacy in microsatellite stable colorectal cancer, which comprises the vast majority of cases.
Broader Implications for Cancer Immunotherapy
The development of this combination therapy highlights the evolving landscape of cancer immunotherapy, particularly for tumor types that have historically been resistant to immune-based approaches. By targeting MUC1, which is expressed across multiple solid tumor types, this platform could potentially have applications beyond colorectal cancer.
The collaboration with the NCI further validates the scientific approach and provides access to significant clinical research expertise as the therapy moves into human trials.
As PDS Biotech advances this program alongside its lead HPV-targeted therapies, the company continues to expand its portfolio of immunotherapies designed to transform how the immune system targets and eliminates cancer cells.
