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Efficacy Of Intravesical Nadofaragene Firadenovec-Vncg For Patients With Bacillus Calmette-Guérin-Unresponsive Non-Muscle-Invasive Bladder Cancer: 36-Month Follow-Up From A Phase 3 Trial
Leonard G. Gomella, MD1, Stephen A. Boorjian, MD2, Vikram M. Narayan, MD3, Badrinath R. Konety, MD4, Viraj A. Master, MD, PhD3, Neal D. Shore, MD5, Ashish M. Kamat, MD6, Trinity J. Bivalacqua, MD, PhD7, Max R. Kates, MD8, Jeffrey S. Montgomery, MD9, Paul L. Crispen, MD10, Gary D. Steinberg, MD11, Piyush K. Agarwal, MD12, Anne Schuckman, MD13, Lawrence I. Karsh, MD, FACS14, Marc A. Bjurlin, DO, MSc, FACOS15, Gordon A. Brown, DO16, Yair Lotan, MD17, Brant A. Inman, MD18, Michael B. Williams, MD19, Michael S. Cookson, MD, FACS20, Sam S. Chang, MD, MBA21, Eric H. Kim, MD22, Alexander I. Sankin, MD23, Colin P N Dinney, MD6
1Sidney Kimmel Cancer Center, Thomas Jefferson University, Philadelphia, PA, USA, 2Mayo Clinic, Rochester, MN, USA, 3Emory University School of Medicine, Atlanta, GA, USA, 4University of Minnesota and Allina Health Cancer Institute, Minneapolis, MN, USA, 5Carolina Urologic Research Center, Myrtle Beach, SC, USA, 6University of Texas MD Anderson Cancer Center, Houston, TX, USA, 7University of Pennsylvania, Philadelphia, PA, USA, 8The James Buchanan Brady Urological Institute and Greenberg Bladder Cancer Institute, Johns Hopkins University School of Medicine, Baltimore, MD, USA, 9University of Michigan, Ann Arbor, MI, USA, 10University of Florida, Gainesville, FL, USA, 11Rush University, Chicago, IL, USA, 12University of Chicago, Chicago, IL, USA, 13USC Institute of Urology, Norris Comprehensive Cancer Center, Los Angeles, CA, USA, 14The Urology Center of Colorado, Denver, CO, USA, 15University of North Carolina, Chapel Hill, NC, USA, 16New Jersey Urology, Cherry Hill, NJ, USA, 17University of Texas Southwestern Medical Center, Dallas, TX, USA, 18Schulich School of Medicine & Dentistry, Western University, London, ON, Canada, 19Urology of Virginia, Virginia Beach, VA, USA, 20University of Oklahoma Health Sciences Center, Oklahoma City, OK, USA, 21Vanderbilt University, Nashville, TN, USA, 22Siteman Cancer Center Barnes-Jewish Hospital, Washington University School of Medicine, Saint Louis, MO, USA, 23Montefiore Medical Center, Bronx, NY, USA

Introduction:
Local bladder-preserving treatment options are needed for patients with Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC). Nadofaragene firadenovec-vncg, a non-replicating adenovirus vector-based gene therapy, is approved by the US Food and Drug Administration for patients with BCG-unresponsive NMIBC with carcinoma in situ (CIS) with/without papillary tumors (±Ta/T1). The primary endpoint of the open-label, multicenter, phase 3 study (NCT02773849) of nadofaragene firadenovec was met, as 53.4% (95% confidence interval [CI]: 43.3, 63.3) of patients with CIS ± Ta/T1 achieved complete response (CR) at 3 months. Thirty-six-month follow-up results of this study are reported here.

Methods:
Patients with BCG-unresponsive NMIBC (N=157) were enrolled; 107 and 50 patients were in the CIS ± Ta/T1 (CIS) and Ta/T1 without CIS (papillary disease [PD]) cohorts, respectively. Efficacy analysis included 103 and 48 patients in the CIS and PD cohorts, respectively, who met the protocol definition of BCG-unresponsive NMIBC. Patients received nadofaragene firadenovec once every 3 months with cystoscopy and cytology assessments. Biopsies were taken at 12 months and patients who remained high-grade recurrence-free (HGRF) were offered continued treatment at the investigator’s discretion.

Results:
All patients completed the 36-month visit or discontinued by 09 September 2021. The mean (standard deviation) duration of follow-up was 42.6 (12.2) months; 13/107 (12.1%) and 10/50 (20.0%) patients in the CIS and PD cohorts received nadofaragene firadenovec at month 36, respectively. Among patients with CIS who achieved CR at 3 months, 14/55 (25.5%) remained HGRF at 36 months. Eleven (22.9%) patients with PD were HGRF at 36 months. Four (3.9%) patients with CIS and 1 (2.1%) patient with PD experienced progression to muscle-invasive disease documented by transurethral resection of bladder tumor at the time of high-grade recurrence as collected in the electronic case report form; 54 patients (35.8%) in total underwent cystectomy. The Kaplan-Meier-estimated cystectomy-free survival rate at month 36 was 53.8% (95% CI: [43.3, 63.1]) and 63.6% (95% CI: [48.0, 75.6]) in the CIS and PD cohorts, respectively. Two patients (1.9%) with CIS and 1 patient (2%) with PD discontinued treatment due to an adverse event.

Conclusions:
Intravesical nadofaragene firadenovec, administered once every 3 months, demonstrated a durable response in patients with BCG-unresponsive CIS ± Ta/T1 and high-grade BCG-unresponsive papillary NMIBC. Nadofaragene firadenovec is a novel and safe intravesical treatment option for BCG-unresponsive NMIBC.

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