ABLE-41, a real-world evidence study for bladder cancer patients treated with nadofaragene firadenovec: Baseline patient characteristics and demographics.

Abstract

e16608 Background: Nadofaragene firadenovec-vncg is the first FDA-approved intravesical nonreplicating adenoviral vector-based gene therapy which delivers interferon alpha 2b into the bladder urothelial cells for treatment of high-risk Bacillus Calmette-Guerin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) ± papillary tumors. “ADSTILADRIN in BLadder cancEr” (ABLE)-41 (NCT06026332) is a phase 4 multicenter non-interventional study examining nadofaragene utilization and outcomes in the real-world setting. While the primary outcome is complete response rate, secondary outcomes include patterns of use as well as patient, caregiver and prescribing physician experience which can provide key insights beyond registrational trials. In this study we present our baseline demographic findings for the initial cohort of patients. Methods: Adult patients receiving nadofaragene firadenovec-vncg in routine clinical practice were offered enrollment following informed consent to share data. The study included optional surveys for patient and caregiver experience. Results: As of October 2024, 54 patients have been enrolled from 17 US centers. Median age was 79 years (range 50-92, IQR 73-85) which is higher than our Phase 3 trial (71 years IQR 66-77). Patients were mostly male (87.0%) and White (96.3%). While 97.4% were ECOG ≤2 one patient had an ECOG status of 3 (excluded in the phase 3 trial). For the 47 patients with complete pathology data at first dose, 30 (65.2%) had CIS ± papillary tumors, and 5 (10.6%) were CIS only. 17 (36.1%) had papillary tumors only which included 3 low grade tumors and one T2 tumor. Of 47 patients with documented BCG unresponsive disease, 34 (72.3%) were BCG refractory and 13 (27.1%) were BCG relapsed. Interestingly, 2 patients had received prior radiotherapy for their NMIBC. For 32 patients who chose to fill out the baseline survey, 27 (50%) had no or slight concerns about side effects, 24 (75%) had no issues with activities of daily living, 31 (96.9%) had no or slight anxiety or depression and 27 (84.4%) had no or slight pain. Conclusions: Early data from the real-world usage of nadofaragene firadenovec in this study demonstrates patients are older than those in the phase 3 trial but despite having 1 subject with ECOG status > 2, they self-report low levels of pain and limitations on daily living activities. Inclusion of patients outside the FDA label and clinical guidelines may provide unique insights and hypothesis generation. Clinical trial information: NCT06026332 .