Phase 4 study evaluating real-world use of ADSTILADRIN® (nadofaragene filadenovec-vncg)

• Ferring to present design and methodology of ABLE-41 real-world evidence study at ASCO’s 20th Annual Genitourinary Cancer Symposium in San Francisco this week

• Study evaluating the efficacy, overall experience, usage patterns, and safety of the first and only intravesical gene therapy approved by the FDA

• Final results from ongoing post-market studies are expected by the end of 2026

Parsippany, NJ, January 24, 2024–(BUSINESS WIRE)–Fering Pharmaceuticals announced today that aDistilladrin BLadder cannotEr (ABLE-41) 20th World Evidence (RWE) Study^th American Society of Clinical Oncology Annual Genitourinary Cancers Symposium (ASCO GU). ABLE-41 (NCT06026332) is an ongoing Phase 4 observational study evaluating the efficacy, overall experience, usage patterns, and safety of ADSTILADRIN.^® (nadofaragene firadenovec-vncg) Real-world setting in the United States.

Adstiladrine has been approved by the U.S. Food and Drug Administration (FDA) for adult patients with high-risk Bacillus Calmette-Guérin (BCG) nonresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ. The first and only intravesical gene therapy. (CIS) Presence or absence of papillary tumor (±Ta/T1).

“Many NMIBC patients who initially respond to BCG therapy experience disease recurrence or progression, so the development of localized, effective bladder-sparing treatment options like Adstiladrine has long been needed. .” Kristen Litchfield, DHSc, medical vice president, urinary tract oncology, Ferring Pharmaceuticals, USA. “In our pivotal Phase 3 trial, Adstiladrine demonstrated long-term efficacy and tolerability from the first dose. With this new post-marketing study, we are building additional evidence in a real-world setting. ”

ABLE-41 is a non-interventional study of NMIBC patients aged 18 years and older who have been treated with Adstiladrine in a clinical setting and who have not previously received this treatment in a clinical trial. In September 2023, Dr. Ferring announced the first patient enrolled in ABLE-41. Eligible patients include those who have been prescribed and scheduled to receive treatment at their physician’s discretion, or who received their first infusion after September 5, 2023 and before the site is activated in the trial (as determined by the physician’s discretion). (at its discretion). The primary objective is to assess whether the patient reaches a complete response (CR) at any time within 3 months and/or 1 year after the first infusion.

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“ABLE-41 is a unique real-world registry that provides important evidence of what patients and physicians can expect from intravesical gene therapy in the clinical setting.” said Siamek Daneshmand, MD, ABLE-41 principal investigator at USC/Noris Comprehensive Cancer Center and the University of Southern California. “I am excited to be part of this ongoing study and to be one of the first uro-oncologists to treat patients with Adstiladrine in a clinical setting. “This is an innovative treatment that is already changing the way patients with NMIBC are managed.”It offers an alternative to radical cystectomy or cystectomy. ”

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

Final results of this prospective multicenter study are expected to be published in late 2026. For more information, visit www.clinicaltrials.gov/study/NCT06026332.

Poster presentation information:

Ongoing Clinical Trials Poster Session B: Urothelial Carcinoma
“ABLE-41: Initial Utilization and Outcomes of Nadofaragene Filadenovec-VNCG in the Real World in the United States.”
Abstract #TPS705, Friday, January 26th, 5:45-6:45pm PST
Presenter: Neil Shore, Thea Daneshmand, Kristen Litchfield, Amy Guo, Christian Juhl, Sandra Guedes, Dalila Delatorre, Yair Lotan

About Adstiladrin
ADSTILADRIN® (nadofaragene firadenovec-vncg) is the first and only FDA-approved intravesical drug for the treatment of adult patients with high-risk Bacillus Calmette-Guerin (BCG) non-responsive non-muscle invasive bladder cancer (NMIBC) with carcinoma. It’s gene therapy. in situ (CIS) with or without papillary tumors. This is a non-replicating adenoviral vector-based treatment containing the gene interferon alpha-2b, which is administered directly into the bladder via catheter once every three months. This vector enters cells in the bladder wall and releases an active gene, causing the bladder cell wall to secrete large amounts of interferon alpha-2b protein, a natural protein used by the body to fight cancer. This approach essentially turns bladder wall cells into interferon microfactories, boosting the body’s own natural defenses against cancer.

ADSTILADRIN is being studied in a clinical trial program that included 157 patients with high-grade, BCG-unresponsive NMIBC who had previously been treated with adequate BCG and who did not respond to additional BCG therapy. Complete inclusion criteria published on .gov: NCT02773849).¹

About non-muscle invasive bladder cancer (NMIBC)
NMIBC is a type of bladder cancer that lies on the surface of the bladder and does not invade deeper into the bladder or spread to other parts of the body.² Bladder cancer is the sixth most common cancer in the United States, and it is estimated that there will be approximately 81,180 new cases of bladder cancer in the United States in 2022.³ 75% of them exist as NMIBC.^Four In high-risk NMIBC patients, intravesical BCG remains the first-line standard treatment. However, more than 50% of patients treated initially with BCG experience disease recurrence and progression within 1 year, and many develop BCG-unresponsive disease.³ Current treatment options for BCG-nonresponsive patients are very limited, and the National Comprehensive Cancer Network (NCCN) guidelines recommend cystectomy (partial or complete removal of the bladder).^Five

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Adstiladrine is a non-replicating adenoviral vector-based gene therapy for adults with high-risk Bacillus Calmette-Guérin (BCG)-refractory non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS). Indicated for patient treatment. No papillary tumors.

Important safety information

Contraindications: Adstiladrine is contraindicated in patients who have had a history of hypersensitivity reactions to interferon alfa or any of the components of the product.

Warnings and precautions:

• Risks of delayed cystectomy: Delaying cystectomy in patients with BCG-unresponsive CIS can lead to the development of muscle-invasive or metastatic bladder cancer, which can be fatal. If a patient with CIS does not fully respond to treatment after 3 months or if CIS recurs, consider cystectomy.

• Risk of disseminated adenovirus infection: Immunocompromised or immunocompromised individuals may be at risk of disseminated infection with ADSTILADRIN due to low levels of replication-competent adenovirus. Avoid exposure of Adstiladrine to immunocompromised or immunocompromised individuals.

Medication and management: Adstiladrine should be administered by intravesical instillation only. Adstiladrine is not for intravenous use, topical use, or oral administration.

Use in specific populations: Advise women of reproductive potential to use effective contraception during Adstiladrine treatment and for 6 months after the last dose. Advise male patients with a female partner of reproductive potential to use effective contraception during Adstiladrine treatment and for 3 months after the last dose.

Side effects: The most common side effects (>10%) include abnormal laboratory values ​​(>15%): increased blood sugar, IV site drainage, increased triglycerides, fatigue, bladder spasms, urination (urgency), and creatinine There was an increase in hematuria (haematuria). ), decreased phosphate, chills, fever (fever), and difficulty urinating (painful urination).

We encourage you to report negative side effects of your prescription medications to the FDA.visit www.FDA.gov/medwatch Or call 1-800-332-1088. You can also contact Ferring Pharmaceuticals at 1-888-FERRING.

Click for complete prescribing information.

About Ferring Pharmaceuticals
Ferring Pharmaceuticals is a privately held, research-driven biopharmaceutical group committed to building families and helping people live better lives. In the United States, Ferring is a leader in reproductive and maternal health, gastroenterology and orthopedics. We are at the forefront of innovation in microbiome-based therapeutics and uro-oncology intravesical gene therapy. The company was founded in 1950 and is headquartered in Saint-Preix, Switzerland. Ferring worldwide employs more than 7,000 people and sells pharmaceutical products in more than 100 countries. Ferring USA is based out of Parsippany, NJ and employs more than 900 people.

For more information, visit www.ferringusa.com, call 1-888-FERRING (1-888-337-7464), or contact us on LinkedIn. X (Twitter).

References:

1. Adostiladrine in patients with high-grade Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC). Government Identifier: NCT02773849. https://clinicaltrials.gov/ct2/show/NCT02773849.

2. Boorjian SA, Alemozaffar M, Konety BR, et al. Intravesical nadofalagen filadenovec gene therapy for BCG-nonresponsive nonmuscle-invasive bladder cancer: a single-arm, open-label, repeated-dose clinical trial. Lancet Oncol 2021; 22:107-17.

3. American Cancer Society. Key statistics about bladder cancer. https://www.cancer.org/cancer/bladder-cancer/about/key-statistics.html. Updated January 13, 2023. Accessed October 15, 2023.

4. Berger M, Cato JW, Dalbani G, Epidemiology and risk factors of other urothelial bladder cancers. Eurol. 2013;63(2):234-41.

5. Insights into the NCCN Guidelines. Bladder Cancer, version 3.2023. www.nccn.org/patients/guidelines/content/PDF/bladder-patient.pdf. Accessed November 7, 2023.

See below for more information.

• Healthcare provider: If you are interested in ordering ADSTILADRIN, please visit ADSTILADRINHCP.com for information and updates.

• Patients and consumers: For more information about ADSTILADRIN, please call 1-888-FERRING (888-337-7464) and select option 1.

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contact address

• media: Media may contact Carol Ready, Contact Director of Brand Communications at (973) 765-7307 or by email at carol.ready@ferring.com.