At EAU26: 41st Annual EAU Congress, held 13–16th March 2026 in London, Professor Dickon Hayne (Fiona Stanley Hospital, University of West Australia, Perth, WA, Australia) presented findings from the ANZUP 1301 (BCG+MM) trial, a phase III study exploring whether alternating BCG with mitomycin C could redefine the treatment landscape for high-risk non–muscle-invasive bladder cancer (NMIBC). With global BCG shortages and ongoing challenges around tolerability, the study offers timely insights into how combination intravesical strategies may deliver comparable efficacy while improving patient experience and resource utilisation.
BCG has been the cornerstone of treatment for high-risk non–muscle-invasive bladder cancer (NMIBC) for decades, but it’s far from perfect. We face two key challenges: tolerability and supply. Many patients struggle to complete the full course due to side effects, and, more recently, global shortages have made access increasingly difficult. The rationale behind ANZUP 1301 – also known as the BCG+MM trial – was to explore whether alternating BCG with mitomycin C could maintain efficacy while reducing BCG exposure and potentially improving tolerability.
This was a phase III randomized trial enrolling patients with high-risk NMIBC. Participants were randomized one-to-one to receive either standard BCG – six weeks of induction followed by ten months of maintenance – or a combination regimen of alternating BCG and mitomycin C. The combination arm involved a nine-week induction followed by nine months of maintenance. Eligible patients included those with high-grade Ta, T1 disease or carcinoma in situ. The primary endpoints included disease-free survival (DFS), recurrence-free survival, and progression-free survival.
In terms of efficacy, the results were very reassuring. Outcomes such as DFS, recurrence-free survival, and progression-free survival were numerically better in the combination arm, although not statistically significant. However, a Bayesian analysis suggested an 83% probability of improved DFS and a 94% likelihood of non-inferiority compared to BCG alone.
Importantly, the combination arm used 39% less BCG – a major advantage in the context of shortages. We also saw significantly better treatment completion rates, suggesting improved tolerability. Serious adverse events attributable to treatment were lower in the combination group, largely because these are typically driven by BCG.
In subgroup analyses, particularly among higher-risk patients – those with T1 disease or carcinoma in situ—we observed signals suggesting improved efficacy with the combination approach. Altogether, this positions BCG plus mitomycin as a highly viable alternative to standard BCG, potentially offering similar or even improved outcomes with better tolerability and reduced resource use.
Patient-reported outcomes were a critical component of the study. We assessed urinary symptoms using tools like the AUA symptom index, alongside broader quality-of-life measures. What we found was a modest but meaningful reduction in symptom burden in the combination arm.
While overall quality-of-life scores were broadly similar between groups over five years, there was a consistent trend favouring the combination therapy – particularly earlier in treatment. These differences didn’t reach statistical significance at five years, but they do suggest that patients may experience a more tolerable journey, especially during the active treatment phase.
This trial opens the door to more flexible and potentially more effective treatment strategies. The idea of combining chemotherapy with immunotherapy intravesically is clearly viable, and that concept could extend further. We’re already seeing interest in alternatives like gemcitabine and docetaxel, with ongoing trials comparing these directly to BCG in treatment-naïve patients. If those regimens prove highly active, the next logical step could be combining them with BCG in a chemo-immunotherapy approach.
In parallel, we’re undertaking extensive translational work from the BCG+MM trial, including collaborations using AI-driven predictive tools to identify which patients are most likely to benefit from BCG-based therapies. With a growing biobank and ongoing biomarker research, the future is likely to be more personalized—selecting the right intravesical strategy for the right patient at the right time.
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Cite: ANZUP 1301: A new approach to high-risk non–muscle-invasive bladder cancer. touchONCOLOGY. March 25th, 2026
Disclosure: Dickon Hayne has no financial or non-financial conflicts of interest to declare in relation to this article. This short article was prepared by touchONCOLOGY in collaboration with Prof Dickon Hayne. Views expressed are the author’s own and do not necessarily reflect the views of Touch Medical Media.
Editor: Sophie Nickelson (Editorial Director)
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