MP16-02 PHASE III TRIAL OF INTRAVESICAL NADOFARAGENE FIRADENOVEC IN PATIENTS WITH HIGH-GRADE, BCG-UNRESPONSIVE, NON-MUSCLE INVASIVE BLADDER CANCER: TWO YEAR FOLLOW-UP IN THE TA/T1 COHORT

Abstract

You have accessJournal of UrologyBladder Cancer: Non-invasive II (MP16)1 Sep 2021MP16-02 PHASE III TRIAL OF INTRAVESICAL NADOFARAGENE FIRADENOVEC IN PATIENTS WITH HIGH-GRADE, BCG-UNRESPONSIVE, NON-MUSCLE INVASIVE BLADDER CANCER: TWO YEAR FOLLOW-UP IN THE TA/T1 COHORT Yair Lotan, Anne K. Schuckman, Stephen A. Boorjian, Katherine E. Cilwa, and Colin P. N. Dinney Yair LotanYair Lotan More articles by this author , Anne K. SchuckmanAnne K. Schuckman More articles by this author , Stephen A. BoorjianStephen A. Boorjian More articles by this author , Katherine E. CilwaKatherine E. Cilwa More articles by this author , and Colin P. N. DinneyColin P. N. Dinney More articles by this author View All Author Informationhttps://doi.org/10.1097/JU.0000000000002001.02AboutPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareFacebookLinked InTwitterEmail Abstract INTRODUCTION AND OBJECTIVE: Patients (pts) with high-grade (HG) non-muscle invasive bladder cancer (NMIBC) who develop BCG-unresponsive disease are faced with limited therapeutic options. Nadofaragene firadenovec is a novel gene-mediated therapy that confers the human IFNα2b gene to bladder urothelium following intravesical instillation and results in prolonged, localized expression of IFNα2b. Phase 3 primary efficacy, safety, and durable responses were demonstrated in the published, initial findings. Herein, 24 month (mos) follow-up data are presented for the HG Ta/T1 cohort. METHODS: A multicenter, open-label Phase 3 trial enrolled pts into two cohorts with BCG-unresponsive NMIBC: carcinoma in situ (CIS±Ta/T1) and HG Ta/T1 alone. For the Ta/T1 cohort, 48 pts and 50 were included in the efficacy and safety analyses. Nadofaragene (3x1011 vp/mL [75 mL]) was administered once every 3 mos for up to 4 doses. The protocol mandated a 5-site (dome, trigone, right and left lateral walls, posterior wall) biopsy at 12 mos. All pts with an absence of HG disease recurrence at 12 mos were offered continued treatment once every 3 mos at the investigator’s discretion. Pts who did not continue treatment beyond 12 mos were followed. RESULTS: As of the September 2020 data cutoff, 9/48 (18.8%) pts had received 24 mos of treatment, and 33/48 (68.8%) remained on study with restricted-mean follow-up of 23.5 mos (95% CI 22.9-24.1). Of 48 pts, 35 (72.9%), 21 (43.8%), 16 (33.3%) were HGRF at 3, 12, and 24 mos. Of those HGRF at 3 mos, 16/35 (45.7%) were HGRF at 24 mos and the median duration of HGRFS was 19.8 months. Cystectomy-free survival was 69.8% (95% CI 54.3-80.9) and overall survival was 93.2% (95% CI 80.4–97.8) when estimated via KM at 24 mos, with 11/48 (22.9%) pts having underwent cystectomy. The most common drug-related adverse events (AEs) were 26% instillation site discharge, 16% dysuria, 14% bladder spasm, and 14% micturition urgency. The majority were Grades 1/2. One (2%) pt had any AE leading to discontinuation. There was one Grade 4 AE (not related to study drug/procedure) and no adverse events occurred leading to pt death. CONCLUSIONS: Nadofaragene firadenovec was well tolerated and demonstrates durability of response in pts with HG Ta/T1, BCG-unresponsive NMIBC in 24 mos follow-up after first intravesical treatment. Clinical trial information: NCT02773849. Source of Funding: FKD Therapies Oy © 2021 by American Urological Association Education and Research, Inc.FiguresReferencesRelatedDetails Volume 206Issue Supplement 3September 2021Page: e296-e296 Advertisement Copyright & Permissions© 2021 by American Urological Association Education and Research, Inc.MetricsAuthor Information Yair Lotan More articles by this author Anne K. Schuckman More articles by this author Stephen A. Boorjian More articles by this author Katherine E. Cilwa More articles by this author Colin P. N. Dinney More articles by this author Expand All Advertisement Loading ...