Phase 4 Study Evaluating Use of ADSTILADRIN® (nadofaragene firadenovec-vncg) in Real-World Setting

Ferring Pharmaceuticals announced today it is presenting the methodology for the ADSTILADRIN in BLadder cancEr (ABLE-41) Real World Evidence (RWE) study at the 20^th Annual American Society of Clinical Oncologists Genitourinary Cancers Symposium (ASCO GU). ABLE-41 (NCT06026332) is an ongoing Phase 4 observational study evaluating the effectiveness, overall experiences, patterns of use, and safety of ADSTILADRIN^® (nadofaragene firadenovec-vncg) in a U.S. real-world setting.

ADSTILADRIN is the first and only intravesical gene therapy approved by the U.S. Food and Drug Administration (FDA) in 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 (±Ta/T1).

“Many NMIBC patients who initially respond to BCG therapy experience disease recurrence or progression, so developing a local, effective, bladder-preserving treatment option like ADSTILADRIN has long been needed,” said Kristen Litchfield, DHSc, Vice President, Medical Affairs, Uro-Oncology, Ferring Pharmaceuticals, U.S. “In our pivotal Phase 3 trial, ADSTILADRIN demonstrated long-term efficacy and tolerability starting from the first dose. With this new post-marketing study, we are looking to build an additional body of evidence in the real-world setting.”

ABLE-41 is a non-interventional study following NMIBC patients aged 18 years or older who are being treated with ADSTILADRIN in a clinical setting and had not previously received this therapy in a clinical trial. In September 2023, Ferring announced the first patient enrolled in ABLE-41. Eligible patients include those who were prescribed and scheduled to receive treatment, per their physician’s discretion, or patients who received their first instillation (per physician discretion) after September 5, 2023, but before the site was activated in the trial. The primary objective is to assess whether patients achieve a complete response (CR) at three months and/or at any time within a year of their first instillation.

“ABLE-41 is a unique real-world registry that will provide important evidence on what patients and physicians can expect from intravesical gene therapy in a clinical setting,” said Siamek Daneshmand, M.D., who is affiliated with USC/Norris Comprehensive Cancer Center and the University of Southern California, and a lead ABLE-41 investigator. “I am thrilled to be part of this ongoing research and among the first uro-oncologists to be treating patients with ADSTILADRIN in a clinical setting. This is an innovative therapy that is already changing how we manage NMIBC patients who no longer respond to standard BCG therapy, providing an alternative to radical cystectomy, or bladder removal surgery.”

Participants in ABLE-41 will be followed for 24 months, or until study discontinuation or withdrawal. Key secondary outcomes include the following: treatment patterns of use; duration of CR; recurrence-free survival, cystectomy-free survival, progression-free survival, overall survival, and bladder cancer–specific mortality; patient, caregiver, and physician experiences; adjunctive use of molecular markers; and safety. Patient experiences will be assessed with a commonly used quality of life questionnaire (EuroQol 5 Dimension 5 Level), measuring mobility, self-care, usual activities, pain/discomfort, and anxiety/depression. Caregiver experiences will be measured with the Work Productivity and Activity Impairment questionnaire, adapted for caregiving, which assesses the impact of health problems on paid and unpaid work.

Final results from this prospective, multi-institutional study are expected at the end of 2026. Learn more at www.clinicaltrials.gov/study/NCT06026332.

Poster Presentation Info:

Trials in Progress Poster Session B: Urothelial Carcinoma

“ABLE-41: Nadofaragene firadenovec-vncg early utilization and outcomes in a real-world setting in the United States.”

Abstract #TPS705, Friday, Jan. 26 at 5:45 - 6:45 p.m. PST

Presenters: Neal Shore, Sia Daneshmand, Kristen Litchfield, Amy Guo, Kristian Juul, Sandra Guedes, Dalila Delattre, and Yair Lotan

About ADSTILADRIN

ADSTILADRIN® (nadofaragene firadenovec-vncg) is the first and only FDA-approved intravesical 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 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 quantities 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-grade, BCG-unresponsive NMIBC who had been treated with adequate BCG previously and did not see benefit from additional BCG treatment (full inclusion criteria published on clinicaltrials.gov: NCT02773849).¹

About Non-Muscle Invasive Bladder Cancer (NMIBC)

NMIBC is a form of bladder cancer which is present in the superficial layer of the bladder and has not invaded deeper into the bladder or spread to other parts of the body.² Bladder cancer is the sixth most common cancer in the U.S., and it is estimated that there were approximately 81,180 new cases of bladder cancer in the U.S. in 2022,³ 75% of which present as NMIBC.⁴ In patients with high-risk NMIBC, intravesical BCG remains the first-line standard of care. However, more than 50% of patients who receive initial treatment with BCG will experience disease recurrence and progression within one year, with many developing BCG-unresponsive disease.³ 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).⁵

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 maternal health, 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 (Twitter).

References:

1. ADSTILADRIN in Patients With High Grade, Bacillus Calmette-Guerin (BCG) Unresponsive Non-Muscle Invasive Bladder Cancer (NMIBC). Gov Identifier: NCT02773849. https://clinicaltrials.gov/ct2/show/NCT02773849.
2. Boorjian SA, Alemozaffar M, Konety BR, et al. Intravesical nadofaragene firadenovec gene therapy for BCG-unresponsive non-muscle-invasive bladder cancer: a single-arm, open-label, repeat-dose clinical trial. Lancet Oncol 2021; 22: 107–17.
3. American Cancer Society. Key Statistics for Bladder Cancer. https://www.cancer.org/cancer/bladder-cancer/about/key-statistics.html. Updated January 13,2023. Accessed October 15, 2023.
4. Burger M, Catto JW, Dalbagni G, et al. Epidemiology and risk factors of urothelial bladder cancer. Eur Urol. 2013;63(2):234-41.
5. NCCN Guideline Insights. Bladder Cancer, Version 3.2023. www.nccn.org/patients/guidelines/content/PDF/bladder-patient.pdf. Accessed November 7, 2023.

More information is available at the following:

• Health Care Providers: If you are interested in ordering ADSTILADRIN, please sign up for information and updates at ADSTILADRINHCP.com.
• Patients and Consumers: For more information about ADSTILADRIN, please call 1-888-FERRING (888-337-7464), and select option number one.

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