CAN-2409 By Candel Therapeutics Receives FDA Regenerative Medicine Advanced Therapy Status For Prostate Cancer

Candel Therapeutics, Inc., a clinical-stage biopharmaceutical company focused on developing multimodal biological immunotherapies to help patients fight cancer, has announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its lead candidate, CAN-2409 (aglatimagene besadenovec). This designation applies to the treatment of newly diagnosed localized prostate cancer in patients classified as intermediate-to-high-risk. CAN-2409 had previously received FDA Fast Track designation for the same indication.

The RMAT designation is designed to accelerate the development and review of regenerative medicine therapies aimed at serious or life-threatening conditions, where preliminary clinical evidence suggests that the therapy may address unmet medical needs. This status provides enhanced opportunities for frequent interaction with the FDA, organizational support to speed development, and eligibility for expedited regulatory mechanisms such as rolling reviews and priority review during the Biologics License Application (BLA) process.

Candel received this designation based on positive results from its Phase 3 randomized, placebo-controlled clinical trial, which evaluated the efficacy and safety of CAN-2409 combined with valacyclovir (a prodrug) alongside the standard of care external beam radiation therapy (SoC) in patients with newly diagnosed localized intermediate-to-high-risk prostate cancer.

In December 2024, Candel announced that the Phase 3 trial successfully met its primary endpoint. The study showed a statistically significant improvement in disease-free survival (DFS), with a p-value of 0.0155 and a 30% reduction (hazard ratio of 0.70) in the risk of prostate cancer recurrence or death from any cause for patients treated with CAN-2409 plus prodrug and SoC radiotherapy (n=496) compared to those receiving placebo plus SoC radiotherapy (n=249). Additionally, CAN-2409 demonstrated a 38% risk reduction in prostate-specific DFS compared to placebo (hazard ratio 0.62, p=0.0046).

Paul Peter Tak, MD, PhD, FMedSci, President and Chief Executive Officer of Candel, said in a statement, “Receiving the FDA’s RMAT designation underscores the critical unmet need in patients with early, localized prostate cancer and validates the promising clinical activity observed with CAN-2409. This designation further supports the design of our phase 3 study, including the DFS primary endpoint agreed upon with the FDA during the SPA negotiation.”

He further continued, “We look forward to collaborating with the FDA to pursue an expeditious approval of CAN-2409 once we submit our BLA—currently anticipated at the end of 2026. Our aim is to introduce a new treatment option for patients at the early stages of prostate cancer, a disease that has seen minimal innovation over the past two decades. We expect the RMAT designation to facilitate the BLA filing process and bring us closer to achieve this objective.”

The trial also revealed a significant increase in the proportion of patients achieving a prostate-specific antigen (PSA) nadir below 0.2 ng/ml in the CAN-2409 arm compared to the placebo group (67.1% versus 58.6%, p=0.0164). Furthermore, pathological complete response rates from 2-year post-treatment biopsies were 80.4% in the CAN-2409 group versus 63.6% in the control group, a statistically significant difference (p=0.0015).

The safety profile observed for CAN-2409 was consistent with prior studies, and no new safety concerns were identified. Key elements of the study design, including the primary endpoint, had been agreed upon with the FDA through a Special Protocol Assessment (SPA), supporting the robustness of the clinical trial results and the regulatory pathway moving forward.