Ferring Unveils New Data with ADSTILADRIN ® (nadofaragene firadenovec-vncg) at 112 th Annual Meeting of the Japanese Urological Association

Business Wire

21-04-2025

PARSIPPANY, N.J.--(BUSINESS WIRE)--Ferring Pharmaceuticals presented the initial results from an ongoing Phase 3 trial (NCT05704244) in Japan assessing the efficacy and safety of ADSTILADRIN ® (nadofaragene firadenovec-vncg) in patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors (±Ta/T1). 1 ADSTILADRIN is the first and only intravesical non-replicating gene therapy approved by the U.S. Food and Drug Administration (FDA) in this patient population. ADSTILADRIN is not approved for use in Japan.
The data – presented at the 112th Annual Meeting of the Japanese Urological Association (JUA 2025) held April 17-19 in Fukuoka, Japan – showed a complete response (CR) rate at three months of 75% (15/20) among participants in Japan with CIS ± high-grade Ta/T1. 1 Overall, 80% of participants (16 patients) experienced a drug–related adverse event (AE), with 76 total AEs recorded. 1 All reported AEs were either Grade 1 (64 AEs in 15 participants, 84.2%) or Grade 2 (12 AEs in 5 participants, 15.8%). 1 No grade 3, 4, or 5 AEs were reported, demonstrating a clinically manageable and tolerable safety profile. 1 The data from Japan add to the growing body of evidence, including recent real-world data presented by the Mayo Clinic that showed a 79% CR rate, demonstrating consistent effectiveness and safety when patients are treated with ADSTILADRIN at three months (19/24 patients with CIS +/- Ta/T1). 2
"At Ferring, we are committed to meeting the unmet needs in bladder cancer care and equipping uro-oncologists with critical evidence they need to deliver effective and life-changing treatment," said Joern Jakobsen, M.D., Ph.D., Vice President and Head of Global Research and Medical for Uro-Oncology and Urology, Ferring Pharmaceuticals. "These initial Phase 3 findings from Japan affirm the safety profile of ADSTILADRIN, demonstrating a three-month efficacy that appears to be higher than previously reported in our Phase 3 clinical trial, and consistent with results from an ongoing independent real-world study presented earlier this year. Collectively, the data are broadening our understanding of the value that ADSTILADRIN offers, furthering our journey to establish ADSTILADRIN as the new standard of care and backbone therapy across the urothelial cancer disease spectrum.'
The ongoing Phase 3 Japanese trial is evaluating the efficacy and safety of ADSTILADRIN in two cohorts of high-risk BCG-unresponsive NMIBC patients: those with CIS ± HG Ta/T1, and patients with papillary tumors (Ta/T1) only. 1 Results from the cohort with CIS ± HG Ta/T1 were presented on April 19 at JUA 2025 in the oral presentation, 'Efficacy and Safety of Nadofaragene Firadenovec for BCG-Unresponsive Non–Muscle-Invasive Bladder Cancer: Initial Results From an Ongoing Japanese Phase 3 Trial.' 1
About ADSTILADRIN
ADSTILADRIN ® (nadofaragene firadenovec-vncg) is the first and only FDA-approved intravesical non-replicating gene-therapy for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors. It is a non-replicating adenovirus vector-based therapy containing the gene interferon alfa-2b, administered locally as a monotherapy by catheter directly into the bladder once every three months. The vector enters the cells of the bladder wall, releasing the active gene and causing the bladder's cell walls to secrete high and transient local expression of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer. This approach essentially turns the bladder wall cells into interferon microfactories, enhancing the body's own natural defenses against the cancer.
ADSTILADRIN has been studied in a clinical trial program that includes 157 patients with high-risk, BCG-unresponsive NMIBC who had been treated with adequate BCG previously and did not see benefit from additional BCG treatment. 3 The pivotal Phase 3 study met its primary endpoint, with more than half (51%) of the 98 patients (95% CI [41%, 61%]) with CIS with or without papillary tumors (±Ta/T1) achieving a complete response (CR) by three months (full inclusion criteria published on clinicaltrials.gov: NCT02773849). 3
Patients in the pivotal Phase 3 study who had no evidence of high-grade recurrence at 12 months following initial ADSTILADRIN therapy continued receiving treatment once every three months at the discretion of their treating physician, entering an additional four-year treatment and monitoring phase. Final 60-month follow-up data demonstrated an 80% overall survival rate and 49% cystectomy-free survival rate in adult patients with high-risk BCG-unresponsive NMIBC with CIS with or without papillary tumors (±Ta/T1), and in patients with high-grade Ta/T1 without CIS. 4 Most treatment emergent AEs (TEAEs) at five years were transient Grade 1 or 2 (66% of all patients studied), and <4% of patients experienced Grade 3 TEAEs with the most common being discharge around the catheter during instillation, fatigue, bladder spasm, urgency to urinate, chills, dysuria, pyrexia, and urinary incontinence. There were no Grade 4 or 5 TEAEs, no treatment-related deaths, and no new safety signals reported with long-term follow-up.
Ferring is leading the future in uro-oncology treatment with ADSTILADRIN at the center, while expanding access with the support of new, state-of-the-art manufacturing facilities. As announced in January 2024, ADSTILADRIN is fully available and accessible in the U.S. ADSTILADRIN has confirmed 99 percent coverage for commercial and government-insured patients. As of April 1, 2024, in accordance with the Centers for Medicare and Medicaid Services (CMS), ADSTILADRIN established an Average Sales Price (ASP). Since the establishment of ASP, all covered claims submitted for reimbursement have received payment within an average of 25 days. 5
About Ferring Uro-Oncology
Ferring is committed to investing in novel therapies, developing life-changing solutions that address unmet medical needs, and aiding the uro-oncology community in helping patients live better lives. More information is available in the U.S. at FerringUroOncology.com and on the dedicated Ferring Uro-Oncology channels on LinkedIn and X.
About Non-Muscle Invasive Bladder Cancer (NMIBC)
NMIBC is a form of bladder cancer that is found in the inner layer cells of the bladder and does not invade into or beyond the muscle wall. 6 In the United States, bladder cancer is the sixth most common cancer, 7 fourth among men, 8 and it is estimated that there will be approximately 84,870 new cases of bladder cancer in the U.S. in 2025. 8 Historically, 75% of bladder cancer presents as NMIBC. 9 In patients with high-risk NMIBC, intravesical BCG remains the first-line standard-of-care. However, approximately one third of patients with NMIBC will not respond to BCG therapy and 50% of those with an initial response will experience recurrence or progression of their disease. 10 Current treatment options for BCG-unresponsive patients are very limited, and National Comprehensive Cancer Network (NCCN) guidelines recommend cystectomy (partial or complete removal of the bladder). 11
INDICATION
ADSTILADRIN is a non-replicating adenoviral vector-based gene therapy indicated for the treatment of adult patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.
IMPORTANT SAFETY INFORMATION
CONTRAINDICATIONS: ADSTILADRIN is contraindicated in patients with prior hypersensitivity reactions to interferon alfa or to any component of the product.
WARNINGS AND PRECAUTIONS:
Risk with delayed cystectomy: Delaying cystectomy in patients with BCG-unresponsive CIS could lead to development of muscle invasive or metastatic bladder cancer, which can be lethal. If patients with CIS do not have a complete response to treatment after 3 months or if CIS recurs, consider cystectomy.
Risk of disseminated adenovirus infection: Persons who are immunocompromised or immunodeficient may be at risk for disseminated infection from ADSTILADRIN due to low levels of replication-competent adenovirus. Avoid ADSTILADRIN exposure to immunocompromised or immunodeficient individuals.
DOSAGE AND ADMINISTRATION: Administer ADSTILADRIN by intravesical instillation only. ADSTILADRIN is not for intravenous use, topical use, or oral administration.
USE IN SPECIFIC POPULATIONS: Advise females of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 6 months after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during ADSTILADRIN treatment and for 3 months after the last dose.
ADVERSE REACTIONS: The most common (>10%) adverse reactions, including laboratory abnormalities (>15%), were glucose increased, instillation site discharge, triglycerides increased, fatigue, bladder spasm, micturition (urination urgency), creatinine increased, hematuria (blood in urine), phosphate decreased, chills, pyrexia (fever), and dysuria (painful urination).
You are encouraged to report negative side effects of prescription drugs to FDA. Visit www.FDA.gov/medwatch or call 1-800-332-1088. You may also contact Ferring Pharmaceuticals at 1-888-FERRING.
Please click to see the full Prescribing Information.
About Ferring Pharmaceuticals
Ferring Pharmaceuticals is a privately-owned, research-driven, specialty biopharmaceutical group committed to building families and helping people live better lives. In the United States, Ferring is a leader in reproductive medicine, and in areas of gastroenterology and orthopaedics. We are at the forefront of innovation in microbiome-based therapeutics and uro-oncology intravesical gene therapy. Our company was founded in 1950 and is headquartered in Saint-Prex, Switzerland. Ferring employs more than 7,000 people worldwide and markets its medicines in over 100 countries. Ferring USA is based in Parsippany, New Jersey, and employs more than 900 employees.
For more information, please visit www.ferringusa.com, call 1-888-FERRING (1-888-337-7464), or connect with us on LinkedIn, and X.
References:
Inoue K, Kikuchi E, Nishiyama H, Nasu Y, et al. Efficacy and Safety of Nadofaragene Firadenovec for BCG-Unresponsive Non–Muscle-Invasive Bladder Cancer: Initial Results From an Ongoing Japanese Phase 3 Trial. Presented at the 112 th Annual Meeting of the Japanese Urological Association, April 19, 2025. Available at: https://www.micenavi.jp/jua2025/search/detail_program/id:2055
Moyer J, Durant A, Nguyen M. Real-world outcomes of nadofaragene firadenovec in BCG-unresponsive non-muscle invasive bladder cancer. Presented at the Annual Meeting of the American Society of Clinical Oncology GU, February 2025.
ADSTILADRIN in Patients With High-Grade, Bacillus Calmette-Guerin (BCG) Unresponsive Non-Muscle Invasive Bladder Cancer (NMIBC). Gov Identifier: NCT02773849. Available at: https://clinicaltrials.gov/ct2/show/NCT02773849. Accessed April 1, 2025.
Ferring Pharmaceuticals. Final Five-Year Analysis of Phase 3 Data With ADSTILADRIN® (nadofaragene firadenovec-vncg) Shows Durable Bladder Preservation and Consistent Long-Term Safety in BCG-unresponsive NMIBC. Published May 6, 2024. https://www.ferringusa.com/us-press-releases-final-five-year-analysis-of-phase-3-data-with-adstiladrin-nadofaragene-firadenovec-vncg-shows-durable-bladder-preservation-and-consistent-long-term-safety-in-bcg-unresponsive-nmibc/. Accessed April 1, 2025.
Ferring Access Support benefits investigations for commercial and government-insured patients through March 15, 2024.
Urology Care Foundation. Non-muscle Invasive Bladder Cancer. Available at: https://www.urologyhealth.org/urology-a-z/n/non-muscle-invasive-bladder-cancer. Accessed April 1, 2025.
National Cancer Institute. Cancer Statistics. Available at: https://www.cancer.gov/about-cancer/understanding/statistics#:~:text=The%20most%20common%20cancers%20%28listed%20in%20descending%20order,pancreatic%20cancer%2C%20leukemia%2C%20thyroid%20cancer%2C%20and%20liver%20cancer. Accessed April 1, 2025.
American Cancer Society. Cancer Facts & Figures 2025. Available at: https://www.cancer.org/content/dam/cancer-org/research/cancer-facts-and-statistics/annual-cancer-facts-and-figures/2025/2025-cancer-facts-and-figures-acs.pdf. Accessed April 1, 2025.
Babjuk M, Burger M, Capoun O, et al. European Association of Urology Guidelines on Non-muscle-invasive Bladder Cancer. Eur Urol. 2022 Jan;81(1):75-94.
Lidagoster S, et al. BCG and Alternative Therapies to BCG Therapy for Non-Muscle-Invasive Bladder Cancer. Curr Oncol. 2024 Feb 16;31(2):1063-1078. doi: 10.3390/curroncol31020079.
National Comprehensive Cancer Network. Bladder Cancer (Version 4.2024). Available at: https://www.nccn.org/professionals/physician_gls/pdf/bladder.pdf. Accessed April 1, 2025.
More information is available at the following: