First Bladder Cancer Patient Dosed with Commercially Available Intravesical Gene Therapy ADSTILADRIN® (nadofaragene firadenovec-vncg)

• Urologists treating eligible high-risk bladder cancer patients at participating early experience program clinics in the U.S. can now prescribe the first and only FDA-approved intravesical gene therapy

• Ferring launches ABLE-41 Real World Evidence Study, a patient registry to explore early use, experiences and outcomes of ADSTILADRIN in a real-world setting

• Ferring on track to increase manufacturing supplies of new gene therapy ADSTILADRIN

Ferring Pharmaceuticals today announced the first patient in the United States with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) was dosed with the commercially available intravesical gene therapy ADSTILADRIN^® (nadofaragene firadenovec-vncg) as part of the ADSTILADRIN Early Experience Program announced earlier this year.

ADSTILADRIN was approved by the U.S. Food & Drug Administration (FDA) in December 2022 for the treatment of adult patients with high-risk, Bacillus Calmette-Guérin (BCG)-unresponsive NMIBC with carcinoma in situ (CIS) with or without papillary tumors. It is the first and only FDA-approved intravesical gene therapy for adults with NMIBC who no longer respond to standard therapy.

“The first patient dosed with ADSTILADRIN marks an incredible milestone for Ferring, the bladder cancer community and the patients we aim to serve,” said Shetal Vyas, Vice President, General Manager, Uro-Oncology at Ferring Pharmaceuticals. “Making this novel and efficacious intravesical gene therapy commercially available for patients underscores our commitment to changing the trajectory of NMIBC. We will continue bringing available doses of ADSTILADRIN forward responsibly as we scale up manufacturing.”

The patient, a 78-year-old male treated at a clinic within the mid-west area also was enrolled in the non-interventional study, the “ADSTILADRIN in BLadder CancEr” (ABLE-41) U.S. Real World Evidence (RWE) Study. The research study, launched in tandem with participating Early Experience Program clinics, will explore early utilization, experiences, and outcomes of ADSTILADRIN in the routine care setting. Learn more at www.clinicaltrials.gov/study/NCT06026332.

“ADSTILADRIN represents a major advancement in the current treatment landscape for people living with high-risk NMIBC who may be facing removal of the bladder,” said Elizabeth Garner, MD, MPH, Chief Scientific Officer at Ferring Pharmaceuticals. “The ADSTILADRIN Early Experience Program and ABLE-41 US RWE Study allow us to address pressing patient needs while collecting data on its use in a real-world setting, further expanding on what we’ve learned in our Phase 3 clinical program.”

Earlier this month, Ferring made doses of ADSTILADRIN commercially available through an Early Experience Program to urologists at the clinical trial sites that participated in the Phase 3 study¹ and a mix of community clinics with the highest number of eligible high-risk patients with NMIBC. The Early Experience Program is designed to ensure every patient who starts on therapy will continue to receive future doses of ADSTILADRIN for the duration of their treatment.

The ADSTILADRIN Early Experience Program is temporary, and more clinics will be offered the opportunity to participate as manufacturing volumes steadily increase over time.

About the ABLE-41 (Adstiladrin in BLadder CancEr) US RWE Study

The ABLE-41 US RWE Study is a real-world observational study of ADSTILADRIN focused on patient treatment outcomes and early use experiences in U.S. routine care settings. Up to 800 patients enrolled in the ADSTILADRIN Early Experience Program who previously have not received ADSTILADRIN will be followed for a minimum of 24 months. The effectiveness of ADSTILADRIN will be measured as complete response rates. Data analyzed will examine patterns of ADSTILADRIN use, and overall experience among patients, caregivers and healthcare providers.

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 encoding interferon alfa-2b protein, 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 82,290 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.² Current treatment options for BCG-unresponsive patients are very limited, and often result in a highly invasive life-changing procedure of radical cystectomy (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 hypersensitivity to interferon alfa or 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.

About Ferring Pharmaceuticals

Ferring Pharmaceuticals is a research-driven, specialty biopharmaceutical group committed to helping people around the world build families and live better lives. In the United States, Ferring is a leader in reproductive medicine and maternal health, uro-oncology and in specialty areas within orthopaedics and gastroenterology, including microbiome therapeutics. For more information, call 1-888-FERRING (1-888-337-7464) or visit www.ferringusa.com.

References:

1. ADSTILADRIN in Patients With High Grade, Bacillus Calmette-Guérin (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 12,2022. Accessed November 22, 2022.
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 2.2022. J Natl Compr Canc Netw. 2022;20(8):866–878.

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