FDA Grants Orphan Drug Designation for CAN-2409 to Treat Pancreatic Cancer


The designation was granted based on overall survival data from an ongoing randomized phase 2 clinical trial.

The FDA has granted Orphan Drug Designation (ODD) to CAN-2409 (Candel Therapeutics, Inc.), a multimodal biological immunotherapy candidate for the treatment of pancreatic cancer.1

“Obtaining Orphan Drug Designation marks a significant milestone for Candel, as we continue to develop CAN-2409 for pancreatic cancer,” said Garrett Nichols, MD, MS, Chief Medical Officer at Candel, in a press release. “We are excited by this FDA designation, which further supports Candel’s efforts in the development of medicines to cure less prevalent yet challenging to treat cancers. The evidence base for CAN-2409 is growing, as we read out clinical trials in patients with difficult-to-treat cancers, such as our recent results in pancreatic ductal adenocarcinoma, and non-small cell lung cancer later in the current quarter.”1

3d rendered medically accurate illustration of pancreas cancer - Image credit: Sebastian Kaulitzki | stock.adobe.com

Image credit: Sebastian Kaulitzki | stock.adobe.com

As the fourth leading cause of cancer-related death in the US in both men and women, pancreatic cancer is anticipated to account for 3.3% of all new cancer cases, according to research from the National Cancer Institute, Surveillance, Epidemiology and End Results (SEER) database. Furthermore, the research estimated 64,050 new cases and 50,550 deaths caused by the disease in 2023. The study authors noted that pancreatic ductal adenocarcinoma (PDAC) makes up 90% of pancreatic carcinomas. PDAC typically occurs due to a lack of early diagnosis and limited response to treatment, leading to an urgent need for new therapies.1,2

The most common recommended treatment for individuals diagnosed with resectable pancreatic cancer is pancreaticoduodenectomy, or the Whipple procedure, based on the location of the tumor. The study authors noted that other options, like adjuvant chemotherapy, have displayed only slight improvements in survival rates, leading to an increased use of neoadjuvant chemotherapy among individuals with resectable PDAC.1

CAN-2409 could be a new treatment option for individuals as an off-the-shelf replication-defective adenovirus. The study authors noted that the HSV-tk gene could provide a systemic anti-tumor immune response as it “locally converts orally administered valacyclovir into a toxic metabolite that kills nearby cancer cells, resulting in the release of a wide variety of cancer antigens,” according to study authors. However, with an adenoviral serotype 5 capsid protein, individuals can induce a specific CD8+ T cell mediated response against the injected tumor for anti-tumor activity. Following, the study authors noted that CAN-2409 provides this individualized anti-tumor response that could treat solid tumors.1

The ODD was granted based on updated results of overall survival from an ongoing randomized phase 2 clinical trial that assessed CAN-2409 with valacyclovir (Valtrex) in combination with standard of care chemoradiation. This was then followed by resection for borderline resectable PDAC.1

“We recently reported data from the phase 2 randomized clinical trial of CAN-2409 in borderline resectable pancreatic cancer, showing that CAN-2409, when added to standard of care, more than doubled the median overall survival obtained with standard of care alone,” said Paul Peter Tak, MD, PhD, FMedSci, President and Chief Executive Officer of Candel, in a press release.1

The median overall survival improved to 28.8 months after treatment with CAN-2409, compared to 12.5 months in the control groups, according to study authors. At 24 months, the survival rate was 71.4% with CAN-2409 compared to 16.7% in the control group after chemoradiation.1

The study authors noted that no new safety signals were observed in this phase 2 study, inferring that multiple injections of CAN-2409 were tolerated.1

“We are pleased that the FDA has now granted Candel with both Orphan Drug and Fast Track Designation to this program, as we seek to reshape the treatment paradigm in pancreatic cancer,” said Tak, in a press release.1

1. Candel Therapeutics Receives FDA Orphan Drug Designation for CAN-2409 for the Treatment of Pancreatic Cancer. Candel Therapeutics. News release. April 11, 2024. Accessed April 12, 2024. https://ir.candeltx.com/news-releases/news-release-details/candel-therapeutics-receives-fda-orphan-drug-designation-can.
2. Pancreatic ductal adenocarcinoma: Treatment hurdles, tumor microenvironment and immunotherapy. National Library of Medicine. News release. February 15, 2020. Accessed April 12, 2024. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7031151/#:~:text=Pancreatic%20ductal%20adenocarcinoma%20(PDAC)%20is%20a%20highly%20aggressive%20lethal%20malignancy,90%25%20of%20pancreatic%20cancer%20cases.
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