MP16-01 EFFICACY OF INTRAVESICAL NADOFARAGENE FIRADENOVEC FOR PATIENTS WITH CARCINOMA IN SITU (CIS), BCG-UNRESPONSIVE NON-MUSCLE INVASIVE BLADDER CANCER (NMIBC): LONGER-TERM FOLLOW-UP FROM THE PHASE III TRIAL

Abstract

You have accessJournal of UrologyBladder Cancer: Non-invasive II (MP16)1 Sep 2021MP16-01 EFFICACY OF INTRAVESICAL NADOFARAGENE FIRADENOVEC FOR PATIENTS WITH CARCINOMA IN SITU (CIS), BCG-UNRESPONSIVE NON-MUSCLE INVASIVE BLADDER CANCER (NMIBC): LONGER-TERM FOLLOW-UP FROM THE PHASE III TRIAL Anne K. Schuckman, Yair Lotan, Stephen A. Boorjian, Katherine E. Cilwa, and Colin P. N. Dinney Anne K. SchuckmanAnne K. Schuckman More articles by this author , Yair LotanYair Lotan 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.0000000000002001.01AboutPDF ToolsAdd to favoritesDownload CitationsTrack CitationsPermissionsReprints ShareFacebookLinked InTwitterEmail Abstract INTRODUCTION AND OBJECTIVE: Despite optimal treatment (tx), patients (pts) with BCG-unresponsive NMIBC remain at significant risk for recurrence and progression and thus an unmet medical need exists for local, effective, bladder-preserving tx options. Nadofaragene firadenovec is a non-replicating recombinant type 5 adenovirus vector-based gene therapy that delivers a copy of the human IFNα2b gene. The Phase 3 study assessed its safety and efficacy in 157 pts with high-grade (HG), BCG-unresponsive NMIBC and met its primary endpoint with 53.4% of pts with CIS±Ta/T1 achieving a complete response (CR), all by 3 mos. Longer-term follow-up results for the CIS±Ta/T1 cohort is reported here. METHODS: The open-label Phase 3 study enrolled 107 BCG-unresponsive, CIS±Ta/T1 (CIS with/without HG Ta or T1) pts. Efficacy analysis included 103 pts. Nadofaragene (3x1011 vp/mL [75 mL]) was administered once every 3 mos for up to 4 doses. The protocol mandated a 5-site biopsy at 12 mos. All pts without HG recurrence at 12 mos were offered continued tx once every 3 mos beyond 12 mos at the investigator’s discretion. Pts who did not receive further tx were followed. RESULTS: As of the data cutoff in September 2020, 64/103 (62.1%) of the cohort remained on study with restricted-mean follow up of 23.5 mos (95% CI 22.9-24.0) and 18/103 (17.5%) pts had received 24 mos of tx. At 24 mos after the first dose, 20/103 (19.4%) pts continued to remain free of HG recurrence. Of the 55 CIS±Ta/T1 pts who achieved a CR following treatment, 20 (36.4%) remained free of HG recurrence at 24 months and the median duration of HGRFS was 12.2 months. By 24 mos 33/103 (32%) pts had undergone cystectomy, for a KM cystectomy-free survival of 64.6% (95% CI 54.1-73.3) and overall survival of 94.4% (95% CI 87.0-97.6). Nadofaragene firadenovec was well tolerated. Overall, 1 grade 4 non-tx-related AE (sepsis) was reported and there were no grade 5 AEs. The most common drug-related AEs were instillation site discharge 24.3%, fatigue 23.4%, bladder spasm 17.8%, and micturition urgency 16.8%. The majority were Grades 1/2. Two (1.9%) pts discontinued due to drug-related AEs (1 instillation site discharge, 1 bladder spasm). CONCLUSIONS: Nadofaragene firadenovec instilled intravesically once every 3 mos demonstrates sustained durability of response with longer follow-up in pts with HG, BCG-unresponsive NMIBC. 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 Anne K. Schuckman More articles by this author Yair Lotan 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 ...