CAN-2409 Receives FDA Orphan Drug Designation in Pancreatic Cancer


CAN-2409 has received orphan drug designation from the FDA for the treatment of patients with pancreatic cancer.

Paul Peter Tak, MD, PhD, FMedSci

Paul Peter Tak, MD, PhD, FMedSci

The FDA has granted orphan drug designation to the multimodal biological immunotherapy candidate aglatimagene besadenovec (CAN-2409) for the treatment of patients with pancreatic cancer.1

On April 4, 2024, Candel Therapeutics—the developer of CAN-2409— announced interim findings from the phase 2 PaTK02 trial (NCT02446093) of CAN-2409 in combination with valacyclovir and standard-of-care (SOC) chemoradiation followed by resection vs the SOC alone in patients with resectable pancreatic ductal adenocarcinoma (PDAC). At a data cutoff of March 29, 2024, patients in the CAN-2409 arm (n = 7) achieved an estimated median overall survival (OS) of 28.8 months compared with 12.5 months for those in the SOC arm (n = 6). Moreover, the 24-month OS rates following chemoradiation were 71.4% vs 16.7%, respectively, and the 36-month OS rates were 47.6% vs 16.7%, respectively.2

“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 SOC, more than doubled the median OS obtained with SOC alone,” Paul Peter Tak, MD, PhD, FMedSci, president and chief executive officer of Candel, said in a press release.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.”

PaTK02 was an investigational, open-label study of CAN-2409 in adult patients with borderline resectable pancreatic adenocarcinoma. To be eligible for the study, patients needed to have an ECOG performance status of 0 or 1; be in adequate health to undergo major surgery; have received a FOLFIRINOX (leucovorin, fluorouracil, irinotecan, and oxaliplatin)–based induction chemotherapy regimen for pancreatic adenocarcinoma for at least 4 months; and have adequate blood counts.3

Patients were randomly assigned 2:1, and those in the experimental arm received 3 courses of CAN-2409 plus valacyclovir after induction chemotherapy, during chemoradiation or after completion of stereotactic body radiation therapy, and at the time of surgery. Up to 2 additional courses of CAN-2409 plus valacyclovir were permitted for patients who experienced disease progression or metastases. In both arms, chemoradiation was administered no more than 2 months after the completion of induction chemotherapy and surgery was performed within 8 weeks of completion of chemoradiation.

The coprimary end points were safety and 24-month OS rate. Secondary end points included OS, progression-free survival, resection rate, disease-free survival, and biomarker characterization.

Additional findings from the study revealed that 4 patients in the investigational arm were still alive at data cutoff, including 2 patients who survived for over 50 months following enrollment. Comparatively, only 1 patient in the standard-of-care arm was alive at 50.6 months.2

In terms of safety, no new signals were observed, and there were no dose-limiting toxicities or instances of pancreatitis. Resected tumor analysis revealed that patients who received CAN-2409 experienced a modified tumor microenvironment with the local recruitment and activation of cytotoxic lymphocytes and increased levels of proinflammatory cytokines. This activity indicates the activation of a significant antitumor immune response with CAN-2409.1

In 2023, CAN-2409 plus valacyclovir was granted fast track designation by the FDA for the treatment of patients with PDAC based on data from this phase 2 clinical trial.4

“Obtaining orphan drug designation marks a significant milestone for Candel, as we continue to develop CAN-2409 for pancreatic cancer,” Garrett Nichols, MD, MS, chief medical officer at Candel said in the press release.1 “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 PDAC, and non-small cell lung cancer later in the current quarter.”


  1. Candel Therapeutics receives FDA orphan drug designation for CAN-2409 for the treatment of pancreatic cancer. News release. April 11, 2024. Accessed April 11, 2024.
  2. Candel Therapeutics announces positive interim data from randomized phase 2 clinical trial of CAN-2409 in non-metastatic pancreatic cancer. News release. Candel Therapeutics, Inc. April 4, 2024. Accessed April 11, 2024.
  3. Neoadjuvant CAN-2409 in combination with chemoradiation or SBRT for borderline resectable pancreatic adenocarcinoma (PaTK02). Updated January 8, 2024. Accessed April 11, 2024.
  4. Candel Therapeutics receives FDA fast track designation for CAN-2409 in pancreatic cancer. News release. Candel Therapeutics, Inc. December 12, 2023. Accessed April 11, 2024.
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