New Data Show Durable Response Following Treatment with ADSTILADRIN® (nadofaragene firadenovec-vncg)

  • 36-month follow-up data from the Phase 3 study demonstrate one-quarter of patients with high-risk, BCG-unresponsive NMIBC remained free of high-grade recurrence
  • Trial showed a 90% three-year overall survival rate, with more than half of cystectomy-free patients at 36 months
  • Follow-up analysis of the Phase 3 study continues with five-year treatment and monitoring
Reno, Nevada (UroToday.com) -- Ferring Pharmaceuticals announced the presentation of new 36-month follow-up data from the Phase 3 study at the 24th Annual Meeting of the Society of Urologic Oncology (SUO) demonstrating a sustained durability of response with ADSTILADRIN® (nadofaragene firadenovec-vncg) 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). ADSTILADRIN is the first and only intravesical gene therapy approved by the U.S. Food and Drug Administration (FDA) in this patient population.

“These new data continue to build on the body of evidence supporting the efficacy and safety of ADSTILADRIN, adding to the data on its sustained durable response following treatment,” said Elizabeth Garner, M.D., M.P.H., Chief Scientific Officer, Ferring Pharmaceuticals, U.S. “Patients with BCG-unresponsive NMIBC are at high risk for recurrence and disease progression, which can often lead to bladder removal surgery, or cystectomy. ADSTILADRIN is an innovative therapy that we believe will transform the current standard of care for these patients, providing an effective alternative therapy.”
The new interim data are from a 36-month follow-up analysis from the Phase 3 study, which is ongoing with a planned five-year treatment and monitoring phase. From the outset, patients with NMIBC CIS±Ta/T1 received ADSTILADRIN 75 mL intravesical instillation (3 x 1011 vp) once every three months for up to 12 months (four doses) or until unacceptable toxicity or recurrent high-grade (HG) NMIBC. Patients without evidence of HG recurrence were allowed to continue ADSTILADRIN treatment every three months as part of an ongoing follow-up analysis. Among patients who continued treatment after the 12-month Phase 3 period, 25.5% (14/55 patients) remained HG recurrence-free at 36 months following the start of treatment. The Kaplan-Meier (KM)-estimated probability of duration of complete response (CR) for at least 12, 24, and 36 months was 46.5%, 36.6%, and 34.2%, respectively.1

In the overall CIS±Ta/T1 cohort of participants (N=103), the KM-estimated median (95% confidence interval [CI]) duration of HG recurrence-free survival was six months (3.4, 8.3), with a 30.1% (21.55%, 39.2%) probability of (95% CI) of HG recurrence-free survival for at least 12 months. At 36 months, the KM-estimated cystectomy-free survival (95% CI) was 53.8% (43.3%, 63.1%), and the three-year overall survival was 90.4% (82.3%, 94.9%). Two patients (1.9%) discontinued ADSTILADRIN treatment due to adverse events, while four (3.9%) experienced progression to muscle-invasive disease.1

“The management of patients with continued high-grade NMIBC after treatment with BCG remains challenging,” said Stephen A. Boorjian M.D., who is the Carl Rosen Professor and David and Anne Luther Chair of the Department of Urology at Mayo Clinic, and was the lead investigator on the ADSTILADRIN clinical trial presented at the conference. “These new ADSTILADRIN data emphasize the importance of obtaining long-term follow up from novel therapies to establish treatment expectations.”
Abstract and Presentation Time:
Efficacy of Intravesical Nadofaragene Firadenovec for Patients with BCG-Unresponsive Carcinoma in Situ of the Bladder: 36-Month Follow-Up From a Phase 3 Trial
Poster #164, Thursday, Nov. 30 from 4:15 – 5:15 p.m. EST 



References:

  1. Boorjian SA, Narayan VM, Konety BR, et. al. Efficacy of intravesical nadofaragene firadenovec for patients with BCG-unresponsive carcinoma in situ of the bladder: 36-month follow-up from a Phase 3 trial. Presented at 24th Annual Meeting of the Society of Urologic Oncology. 
Source: Ferring Pharmaceuticals. (2023). New Data Show Durable Response Following Treatment with ADSTILADRIN® (nadofaragene firadenovec-vncg) [Press release]. https://www.ferring.com/new-data-show-durable-response-following-treatment-with-adstiladrin-nadofaragene-firadenovec-vncg/.

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