Dr Hayne on the Implications of the ANZUP 1301 Trial of Mitomycin Plus BCG in NMIBC

“This [regimen] is a way that we can deliver a safe and effective therapy to our patients with high-risk NMIBC up front whilst preserving BCG supplies. Globally, [patients] have been treated with unproven agents and have been given non-ideal BCG regimens [with] not enough maintenance BCG, [or they’re] using third-dose BCG even though it’s not as effective as the full dose.”

Dickon Hayne, MD, FRCS, MBBS, a professor of urology at The University of Western Australia Medical School, as well as a consultant urological surgeon and the head of Urology for the South Metropolitan Health Service, discussed the clinical implications of the phase 3 ANZUP 1301 trial (NCT02948543) evaluating mitomycin in combination with BCG in patients with high-risk non–muscle-invasive bladder cancer (NMIBC).

During the 2025 ASCO Annual Meeting, Hayne presented data from ANZUP 1301, which demonstrated that patients who received mitomycin plus BCG (n = 249) achieved a disease-free survival benefit compared with those who received BCG monotherapy (n = 252; HR, 0.87; 95% CI, 0.65-1.16; P = .34). The regimens produced comparable 3-month complete response rates, at 90% (95% CI, 85%-93%) and 86% (95% CI, 81%-90%), respectively.

Safety outcomes were similar between the 2 arms, with 2 deaths during treatment in the monotherapy arm and 1 in the combination arm. In terms of serious adverse effects, 1 patient in the monotherapy arm experienced further mycotic aortic aneurysm and another in the combination arm had myasthenia gravis. Notably, comparable safety and efficacy outcomes were achieved with 40% less doses of BCG being needed in the combination arm.

The lower dose of BCG used in the ANZUP 1301 regimen could help to alleviate the ongoing global BCG shortage, Hayne said. In the past, patients with NMIBC have been treated with unproven agents and non-ideal BCG regimens, including regimens without enough maintenance BCG and third-dose BCG, due to the shortage, he added. There is an approximately 30% to 50% decrease in BCG supply; wider adoption of mitomycin plus BCG could help to address this, he concluded.