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A quarter patients with high-risk, BCG-unresponsive disease were recurrence free at 3 years.
WASHINGTON -- The gene therapy nadofaragene firadenovec (Adstiladrin) continued to demonstrate clinical activity in patients with high-risk bladder cancer, researchers reported.
The long-term follow-up data came from a phase III study of patients with Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) with carcinoma in situ (CIS), with or without papillary tumors. The data showed that of 55 patients who had achieved a complete response (CR) to the therapy at 3 months, 14 (25.5%) were still high-grade recurrence free at 3 years, according to Stephen A. Boorjian, MD, of the Mayo Clinic in Rochester, Minnesota, and colleagues, at the Society of Urologic Oncologyopens in a new tab or window (SUO) annual meeting. The findings emphasize the importance of obtaining long-term data from novel therapies in order to set treatment expectations, he said. Primary trialopens in a new tab or window results, reported at the 2019 SUOopens in a new tab or window meeting, demonstrated that the 55 patients who achieved a CR at 3 months represented 53.4% of 103 patients eligible for analysis. Here, Boorjian and colleagues reported that the long-term high-grade recurrence free results were:
- 40.8% (n=42 patients) at 6 months
- 35.0% (n=36) at 9 months
- 24.3% (n=25) at 1 year
- 19.4% (n=20) at 2 years
- 13.6% (n=14) at 3 years
In addition to the 3-year results, of those 55 patients who achieved a CR, the percentage who maintained that response was:
- 76.4% (n=42) at 6 months
- 65.5% (n=36) at 9 months
- 45.5% (n=25) at 12 months
- 36.4% (n=20) at 24 months
The median duration of CR was 9.7 months, and the probability of duration of CR for at least 36 months was 34.2%. The cystectomy-free survival rate at 36 months was 53.8% (95% CI 43.3-63.1), and the 3-year overall survival rate was 90.4% (95% CI 82.3-94.9).
Nadofaragene firadenovecopens in a new tab or window is an adenoviral vector-based product that the FDA approvedopens in a new tab or window in 2022 as the first gene therapy for the treatment of high-risk BCG-unresponsive NMIBC for adult patients with CIS, with or without papillary tumors. The single-arm, open-label, multicenter trialopens in a new tab or window enrolled 107 patients, 103 of whom met the protocol definition of BCG-unresponsive NMIBC and were included in an efficacy analysis. Patients received nadofaragene 75 mL intravesical instillation (3 x 1011 viral particles/mL) once every 3 months for up to 12 months (4 doses) or until unacceptable toxicity or recurrent high-grade NMIBC. Per protocol, a five-site biopsy of the bladder was performed at 12 months, and patients who were high-grade recurrence free were offered continued treatment. The trial is ongoing with a planned 5-year treatment and monitoring phase. The median age of the entire cohort of 107 patients was 72, 89% were male, and 99% had received at least two BCG courses before enrolling in the study. Boorjian and colleagues reported that 3.9% (n=4) of patients experienced progression to muscle-invasive disease as documented by transurethral resection of bladder tumor at the time of high-grade recurrence. Two patients discontinued treatment due to adverse events (AE), including a grade 3 bladder spasm and a grade 2 instillation site discharge. Neither event was considered a serious AE.
