Candel Therapeutics Inc (NASDAQ:CADL) announced that the European Medicines Agency (EMA) has granted Orphan Designation to its investigational immunotherapy CAN-2409 for the treatment of pancreatic cancer.
The designation adds to CAN-2409’s existing Orphan Drug Designation and Fast Track Designation from the US Food and Drug Administration for pancreatic ductal adenocarcinoma.
The EMA grants Orphan Designation to therapies for rare and serious conditions affecting fewer than five in 10,000 people in the European Union.
The designation provides incentives, including regulatory fee reductions, protocol assistance, research grants, and up to 10 years of market exclusivity upon approval.
“Receiving EMA Orphan Designation for CAN-2409 represents a significant regulatory milestone for CAN-2409 in this disease,” Candel CEO Dr Paul Peter Tak said.
“This designation, alongside our existing FDA Orphan Drug and Fast Track Designations for CAN-2409 in PDAC, underscores the promise of our novel multimodal immunotherapy approach. We look forward to continuing our development efforts and working with regulatory authorities across the world to make this innovative therapy available to patients in need.”
CAN-2409 is a replication-defective adenovirus engineered to deliver the herpes simplex virus thymidine kinase (HSV-tk) gene to tumor cells.
When combined with a prodrug such as valacyclovir or acyclovir, the HSV-tk enzyme converts the prodrug into compounds that induce immunogenic cell death. This process results in the release of tumor antigens into the tumor microenvironment.
The mechanism is designed to trigger a systemic immune response against multiple therapy-resistant solid tumors.
Candel is investigating CAN-2409 in cancers including prostate cancer, non-small cell lung cancer, and pancreatic ductal adenocarcinoma.
The company previously reported positive survival data from a Phase 2a trial in borderline resectable pancreatic cancer.
In that trial, patients receiving CAN-2409 and valacyclovir showed a median overall survival of 31.4 months. The control arm receiving standard care showed a median overall survival of 12.5 months.
At the time of data cut-off in February 2025, three of seven patients treated with CAN-2409 remained alive with survival durations of 66.0, 63.6, and 35.8 months.
“Pancreatic cancer remains one of the most challenging malignancies to treat, particularly in patients whose disease is unresectable,” Candel’s chief medical officer Dr Garrett Nichols said.
“We observed that CAN-2409 profoundly remodels the tumor microenvironment, transforming it from ‘cold’ (non-inflamed and immunosuppressive) to ‘hot’ (inflamed with active immune infiltration) and extends survival.”