Diakonos Oncology Corp. presented encouraging preliminary data from its Phase 1 study of DOC1021, a patient-specific dendritic cell immunotherapy, in pancreatic ductal adenocarcinoma (PDAC) at the Society for Immunotherapy of Cancer (SITC) 40th Annual Meeting. The Houston-based clinical-stage biotechnology company reported that five of seven treated patients remain alive with post-operative survival times ranging from 12.9 to 45.3 months, with three patients remaining relapse-free.
Treatment Safety and Tolerability
The study demonstrated a favorable safety profile for DOC1021, with no dose-limiting toxicities observed across the patient cohort. The most common adverse events were mild flu-like symptoms, supporting the therapy's potential for outpatient administration. This safety profile is particularly significant given the challenging nature of pancreatic cancer treatment and the typically poor prognosis associated with the disease.
Biomarker Evidence of Immune Activation
The trial revealed promising biomarker signatures indicating enhanced immune system activity following DOC1021 treatment. Researchers observed post-vaccination CD127 upregulation on circulating T-cells and Granzyme B upregulation in circulating CD8+ cells, suggesting the therapy successfully activated the patient's immune response against cancer cells.
"Pancreatic cancer patients face a poor prognosis with few effective treatment options," said Jay Hartenbach, President and COO of Diakonos Oncology. "We are encouraged by these results for DOC1021, a novel and highly targeted approach which activates the patient's own immune system in a specific manner against their cancer."
Novel Double-Loading Approach
DOC1021 represents a first-in-class, patient-derived double-loaded dendritic cell therapy that uniquely combines tumor lysate and amplified tumor-derived mRNA. The immunotherapy is manufactured using a patient's dendritic cells combined with mRNA and proteins prepared from freshly obtained patient tumor specimens.
The unique double-loading approach mimics a viral infection, unlocking what the company describes as a synergistic and exponentially more powerful tumor killing TH1 response driven by dual protein and RNA antigen sourcing. This methodology allows targeting of the complete cancer antigen pool without requiring molecular modification of the patient's immune cells for manufacturing or preconditioning chemotherapy and high dose IL-2 for administration.
Expanding Clinical Program
The initial patient group received DOC1021 after neoadjuvant chemotherapy, surgery, and adjuvant chemotherapy. The company is now enrolling a second group of patients where DOC1021 will be administered after surgery but before adjuvant chemotherapy, representing a modified treatment sequence approach.
"Given the ongoing need for and growing excitement surrounding personalized tumor vaccination following resection of pancreatic cancer, we are very excited to continue enrolling patients in this very important trial," said Dr. Benjamin Leon Musher, Professor of Medicine-Hematology & Oncology and Medical Director of Medical Oncology of the Dan L Duncan Comprehensive Cancer Center at Baylor College of Medicine.
Regulatory Recognition and Broader Pipeline
Diakonos has received significant regulatory support for its DOC1021 program, including FDA Fast Track designations for both pancreatic cancer (May 2024) and glioblastoma programs (October 2023). The company also received Orphan Drug Designation for the glioblastoma program in January 2024.
Beyond the Phase 1 pancreatic cancer study (NCT04157127), Diakonos recently opened a Phase 2 glioblastoma study with DOC1021 (NCT06805305), building on promising results previously observed in that indication. The therapy's design allows for simple outpatient administration and broad reach via community cancer centers, potentially expanding access to this personalized immunotherapy approach.
