09 May 2025
Imfinzi
regimen
demonstrated statistically significant and clinically meaningful
improvement in disease-free survival for high-risk
non-muscle-invasive bladder cancer in POTOMAC Phase III
trial
Patients lived significantly longer
without high-risk disease recurrence or progression after one year
of Imfinzi treatment plus Bacillus Calmette-Guérin (BCG) induction
and maintenance therapy vs. BCG alone
Positive high-level results from the POTOMAC
Phase III trial showed one year of treatment with AstraZeneca's
Imfinzi (durvalumab) plus
standard-of-care BCG induction and maintenance therapy demonstrated
a statistically significant and clinically meaningful improvement
in disease-free survival (DFS) for patients with high-risk
non-muscle-invasive bladder cancer (NMIBC) compared to BCG
induction and maintenance therapy alone.
The trial was not statistically powered to
formally test overall survival (OS) however a descriptive analysis
demonstrated no detriment.
More than 70% of bladder cancer patients are
diagnosed with NMIBC, an early-stage cancer where the tumour is in
the tissue that lines the inner surface of the bladder but has not
invaded the muscle wall.1-2 About half of patients with NMIBC are
classified as high-risk for disease progression or recurrence
because of certain characteristics of their cancer, such as tumour
grade, stage and specific tumour features.3
Maria De Santis, MD, Head of the
Interdisciplinary Uro-Oncology Section at Charité
Universitätsmedizin Berlin, Germany, and a principal investigator
in the POTOMAC trial, said: "These exciting data show that adding
one year of durvalumab to the current standard treatment
significantly extends the time patients live without high-risk
disease recurrence or progression. While most patients with
non-muscle invasive bladder cancer are treated with curative
intent, 80 per cent see their disease return and almost half may
require life-altering surgery to remove the bladder, underscoring
the urgent need to improve treatment."
Cristian Massacesi, Chief Medical Officer and
Oncology Chief Development Officer, AstraZeneca, said: "The
positive results for Imfinzi in the POTOMAC trial represent
a significant advance that will potentially allow more patients
with early-stage bladder cancer to benefit from this important
immunotherapy. Building on the NIAGARA data, this outcome
demonstrates our strategy of bringing novel therapies to patients
with early-stage disease where there is the greatest potential for
long-term benefit."
The safety and tolerability of Imfinzi plus BCG induction and
maintenance therapy was consistent with the known safety profiles
of the individual medicines, with no new safety concerns
identified. The addition of Imfinzi did not compromise patients'
ability to complete BCG induction and maintenance
therapy.
The second experimental arm evaluating
Imfinzi plus BCG
induction-only therapy compared to BCG induction and maintenance
therapy alone did not meet the endpoint of DFS.
These data will be presented at a forthcoming
medical meeting and shared with global regulatory
authorities.
Imfinzi is approved in the US
and other countries for patients with muscle-invasive bladder
cancer (MIBC) based on results from the NIAGARA Phase III trial and
continues to be investigated across early and late-stage bladder
cancer in various treatment combinations, including in patients
with MIBC who are ineligible or refuse to take cisplatin (VOLGA)
and in locally advanced or metastatic disease
(NILE).
Notes
Bladder
cancer
Bladder cancer is the 9th most common cancer in
the world, with more than 614,000 cases diagnosed each
year.4 The most
common type is urothelial carcinoma, which begins in the urothelial
cells of the urinary tract.2
In 2024, an estimated 125,000 patients were
treated for high-risk NMIBC, for which the current standard of care
is transurethral resection of bladder tumour (TURBT) followed by
administration of BCG directly into the
bladder.5-6 Up to
80% of patients experience disease recurrence within five years,
and rates of progression in high-risk patients can be as high as
45%.2 There is a
critical need for treatment options in this curative-intent
setting.
POTOMAC
POTOMAC is a randomised, open-label,
multi-centre, global Phase III trial evaluating Imfinzi in combination with BCG
therapy as a treatment for 1,018 patients with high-risk, BCG-naïve
NMIBC who have undergone TURBT prior to randomisation. Patients
were randomised 1:1:1 to receive Imfinzi plus BCG induction and
maintenance therapy, or Imfinzi plus BCG induction-only
therapy, versus standard-of-care BCG induction and maintenance
therapy.
The trial was conducted in more than 120
centres across 12 countries including Canada and others across
Europe and Asia. The primary endpoint was DFS, defined as time from
randomisation to date of first recurrence of high-risk disease or
death from any cause, for Imfinzi plus BCG induction and
maintenance therapy compared to BCG induction and maintenance
therapy alone. Secondary endpoints included DFS for Imfinzi plus BCG induction only
therapy versus the comparator arm, as well as OS at five years and
safety across both experimental arms of the trial.
Imfinzi
Imfinzi
(durvalumab) is a human monoclonal antibody that binds to the
PD-L1 protein and blocks the interaction of PD-L1 with the PD-1 and
CD80 proteins, countering the tumour's immune-evading tactics and
releasing the inhibition of immune responses.
In addition to its indication in MIBC,
Imfinzi is the global
standard of care based on OS in the curative-intent setting of
unresectable, Stage III non-small cell lung cancer (NSCLC) in
patients whose disease has not progressed after chemoradiotherapy
(CRT). Additionally, Imfinzi is approved as a perioperative
treatment in combination with neoadjuvant chemotherapy in
resectable NSCLC, and in combination with a short course of
Imjudo (tremelimumab) and
chemotherapy for the treatment of metastatic NSCLC. Imfinzi is also approved for
limited-stage small cell lung cancer (SCLC) in patients whose
disease has not progressed following concurrent platinum-based CRT;
and in combination with chemotherapy for the treatment of
extensive-stage SCLC.
Imfinzi is also
approved in combination with chemotherapy in locally advanced or
metastatic biliary tract cancer and in combination with
Imjudo in unresectable
hepatocellular carcinoma (HCC). Imfinzi is also approved as a
monotherapy in unresectable HCC in Japan and the European Union
(EU).
In March 2025, perioperative Imfinzi added to standard-of-care
chemotherapy met the primary endpoint of event-free survival in the
MATTERHORN Phase III trial in resectable gastric and
gastroesophageal junction cancers.
Imfinzi in
combination with chemotherapy followed by Imfinzi monotherapy is approved as a
1st-line treatment for primary advanced or recurrent endometrial
cancer (mismatch repair deficient disease only in US and EU).
Imfinzi in combination
with chemotherapy followed by Lynparza (olaparib) and Imfinzi is approved for patients with
mismatch repair proficient advanced or recurrent endometrial cancer
in EU and Japan.
Since the first approval in May 2017, more than
374,000 patients have been treated with Imfinzi. As part of a broad
development programme, Imfinzi is being tested as a single
treatment and in combinations with other anti-cancer treatments for
patients with NSCLC, bladder cancer, breast cancer, ovarian cancer
and several gastrointestinal cancers.
AstraZeneca in
immuno-oncology (IO)
AstraZeneca is a pioneer in introducing the
concept of immunotherapy into dedicated clinical areas of high
unmet medical need. The Company has a comprehensive and diverse IO
portfolio and pipeline anchored in immunotherapies designed to
overcome evasion of the anti-tumour immune response and stimulate
the body's immune system to attack tumours.
AstraZeneca strives to redefine cancer care and
help transform outcomes for patients with Imfinzi as a monotherapy and in
combination with Imjudo as
well as other novel immunotherapies and modalities. The Company is
also investigating next-generation immunotherapies like bispecific
antibodies and therapeutics that harness different aspects of
immunity to target cancer, including cell therapy and T-cell
engagers.
AstraZeneca is pursuing an innovative clinical
strategy to bring IO-based therapies that deliver long-term
survival to new settings across a wide range of cancer types. The
Company is focused on exploring novel combination approaches to
help prevent treatment resistance and drive longer immune
responses. With an extensive clinical programme, the Company also
champions the use of IO treatment in earlier disease stages, where
there is the greatest potential for cure.
AstraZeneca in oncology
AstraZeneca is leading a revolution in oncology
with the ambition to provide cures for cancer in every form,
following the science to understand cancer and all its complexities
to discover, develop and deliver life-changing medicines to
patients.
The Company's focus is on some of the most
challenging cancers. It is through persistent innovation that
AstraZeneca has built one of the most diverse portfolios and
pipelines in the industry, with the potential to catalyse changes
in the practice of medicine and transform the patient
experience.
AstraZeneca has the vision to redefine cancer
care and, one day, eliminate cancer as a cause of
death.
AstraZeneca
AstraZeneca (LSE/STO/Nasdaq: AZN) is a global,
science-led biopharmaceutical company that focuses on the
discovery, development, and commercialisation of prescription
medicines in Oncology, Rare Diseases, and BioPharmaceuticals,
including Cardiovascular, Renal & Metabolism, and Respiratory
& Immunology. Based in Cambridge, UK, AstraZeneca's innovative
medicines are sold in more than 125 countries and used by millions
of patients worldwide. Please visit astrazeneca.com and
follow the Company on social media @AstraZeneca.
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References
1. Fuge O, et al.
Immunotherapy for bladder cancer. Res Rep Urol.
2015;7:65-79.
2. American Cancer
Society. What Is Bladder Cancer? Available at:
https://www.cancer.org/cancer/bladder-cancer/about/what-is-bladder-cancer.html.
Accessed May 2025.
3. Porten SP, Cooperberg
MR. High-risk nonmuscle invasive bladder cancer: definition and
epidemiology. Curr Opin
Urol. 2012;22:385-389.
4. World Health
Organization. International Agency for Research on Cancer. Bladder
Fact Sheet. Available at:
https://gco.iarc.who.int/media/globocan/factsheets/cancers/30-bladder-fact-sheet.pdf.
Accessed May 2025.
5. AstraZeneca PLC.
Investor Relations Epidemiology Spreadsheet. Available at:
https://www.astrazeneca.com/investor-relations.html.
Accessed May 2025.
6. Gontero P, et al. EAU
Guidelines on Non-muscle-invasive Bladder Cancer (TaT1 and CIS).
2025. Edn. presented at the EAU Annual Congress Madrid 2025. ISBN
978-94-92671-29-5.
Matthew Bowden
Company Secretary
AstraZeneca PLC
