Advanced penile cancer has long been overlooked in oncology research — but a new phase 2 trial is pushing for change. The EPIC-A trial enrolled 29 patients with locally advanced or metastatic penile cancer and tested cemiplimab (a PD-1 inhibitor) combined with platinum-based chemotherapy, followed by cemiplimab maintenance for up to 24 months. The results, published in JCO Oncology Advances, showed a strong efficacy and safety profile with no new safety signals.

Lead investigator Dr. Amit Bahl of Bristol Cancer Institute hopes the findings will prompt major bodies like NCCN and the European Association of Urology to add this regimen to their guidelines — unlocking broader funding and access for patients outside of clinical trials. The team is also pursuing a biomarker analysis and a neoadjuvant study to explore whether the combo can improve surgical outcomes.

By the Numbers:

• 62.1% clinical benefit rate at 12 weeks
• 51.7% objective response rate at 12 weeks
• 6.3 months median progression-free survival
• 15.7 months median overall survival
• 48.3% clinical benefit rate maintained at 21 weeks

Why it matters: Penile cancer is rare and historically underfunded, leaving patients with few treatment options. EPIC-A not only offers a promising new therapy but also proves that rigorous clinical trials for rare cancers are achievable — potentially opening the door to more research and better outcomes for this underserved population.