The U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to biological immunotherapy candidate, CAN-2409, for the treatment of newly diagnosed localized prostate cancer in patients with intermediate-to-high-risk disease.
This designation follows CAN-2409’s prior FDA Fast Track designation for the same indication and is intended to expedite the development and review of therapies addressing serious diseases with unmet medical needs. The RMAT status provides enhanced FDA guidance, organizational commitment, and eligibility for accelerated approval pathways, including rolling and priority review of the Biologics License Application (BLA).

The RMAT designation was awarded based on positive results from a pivotal phase 3 randomized, placebo-controlled clinical trial involving 745 patients, which evaluated CAN-2409 plus valacyclovir (a prodrug) in combination with standard-of-care external beam radiation therapy.
The trial met its primary endpoint by demonstrating a statistically significant 30% reduction in the risk of prostate cancer recurrence or death (hazard ratio 0.70; p=0.0155) compared to placebo plus standard care. CAN-2409 also showed a 38% reduction in prostate-specific disease-free survival risk (HR 0.62; p=0.0046) and significantly increased the proportion of patients achieving a prostate-specific antigen (PSA) nadir below 0.2 ng/ml (67.1% vs. 58.6%; p=0.0164). Additionally, 80.4% of patients in the CAN-2409 arm had a pathological complete response in 2-year post-treatment biopsies compared to 63.6% in the control group (p=0.0015). The safety profile was consistent with previous studies, with no new safety concerns identified.

CAN-2409 is an off-the-shelf, replication-defective adenovirus designed to deliver the herpes simplex virus thymidine kinase (HSV-tk) gene directly to tumor cells. When combined with orally administered valacyclovir, HSV-tk converts the prodrug into a cytotoxic agent that kills nearby cancer cells and stimulates a systemic CD8+ T cell immune response against tumor antigens. This in situ immunization approach aims to generate broad anti-tumor activity, and CAN-2409 has shown encouraging results in combination with radiotherapy, chemotherapy, surgery, and immune checkpoint inhibitors across multiple tumor types. Over 1,000 patients have been treated with CAN-2409 to date, demonstrating a favorable tolerability profile.

Source.