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Nadofaragene firadenovec maintains efficacy in NMIBC after 5 years of follow-up

The gene therapy nadofaragene firadenovec-vncg (Adstiladrin) continued to demonstrate durable clinical activity in patients with high-risk, BCG-unresponsive non-muscle-invasive bladder cancer (NMIBC), according to five-year data from the phase 3 CS-003 study ( NCT02773849) presented at the 2024 AUA Annual Meeting.1

The open-label, multicenter study included 157 patients enrolled in 2 cohorts: patients with carcinoma in situ (CIS) ± Ta/T1 (CIS cohort; n = 107) and patients with high-grade Ta/T1 without CIS (papillary disease) . PD) cohort; n = 50). The five-year efficacy analysis included 103 patients from the CIS cohort and 48 patients from the PD cohort.

At a median follow-up of 50.8 months (interquartile range, 39.1-60.0) for all treated patients, the 5-year survival rates were 76.3% (95% CI: 64.6-84.5) and 85% .9% (95% CI: 70.9). -93.5) in the CIS and PD cohorts, respectively. At 60 months, the cystectomy-free survival rate was 43.2% (95% CI: 32.2-53.7) in the CIS cohort and 58.7% (95% CI: 43.1-71.4) in the PD -cohort.

In the CIS cohort, 10.9% (n = 6) of patients who had a complete response at 3 months (n = 55) remained free of high-grade recurrence (HGRF) at 57 months. In the PD cohort, 20% (n=7) of the 35 patients who were HGRF at 3 months remained HGRF at 57 months.

Based on previously reported findings from this study, the FDA approved nadofaragene firadenovec for use in this setting in December 2022.2

“At 60 months, nadofaragen enabled bladder preservation in nearly half of patients in the CIS cohort and in two-thirds of patients in the Ta/T1 (PD) cohort,” said Vikram Narayan, MD, when presenting the findings at the AUA meeting .

“Nadofaragene represents a new treatment option for BCG-unresponsive NMIBC, especially for patients who are unwilling or unable to undergo cystectomy, with a convenient dosing pattern for patients (once every three months),” said Narayan, director of Urological Oncology at Grady Memorial Hospital and assistant professor in the Department of Urology at Emory University School of Medicine at Winship Cancer Institute.

The total study population was heavily pretreated, with a median of 12 previous BCG doses. Patients were treated with 75 ml nadofaragene firadenovec (3 x 1011 virus particles/ml) once every 3 months for up to 4 doses. The study protocol stipulated that patients would receive a 5-site biopsy at 12 months and that patients who had no evidence of high-grade recurrence could continue to receive nadofaragene firadenovec every 3 months.

Regarding safety, nadofaragen was well tolerated and most treatment-emergent adverse events (TEAEs) were grade 1/2 and resolved rapidly. A total of 66.2% of patients had a TEAE grade 1/2 and 3.8% had a TEAE grade 3. Grade 3 TEAEs included 2 cases of micturition urgency and 1 case of bladder spasm, syncope, hypertension and urinary incontinence. At the five-year follow-up, no new safety signals were reported, as well as no Grade 4 TEAEs or treatment-related deaths.

Ferring Pharmaceuticals, the developer of nadofaragene, announced in January 2024 that nadofaragene firadenovec is now fully available in the United States to eligible patients.3

References

1. Efficacy of nadofaragene firadenovec-vncg for patients with Bacillus Calmette-Guérin unresponsive non-muscle invasive bladder cancer: final results from a phase 3 study. Presented at: American Urological Association Annual Meeting 2024. May 2-6, San Antonio, Texas. Summary PD48-01.

2. FDA approves first gene therapy to treat high-risk, non-muscle-invasive bladder cancer. News item. FDA. December 16, 2022. Accessed January 16, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-high-risk-non-muscle-invasive – bladder cancer

3.Ferring announces full availability of ADSTILADRIN (nadofaragene firadenovec-vncg) in US news release. Ferring pharmaceutical products. Published and accessed online January 16, 2024. https://www.businesswire.com/news/home/20240116546395/en/Ferring-Announces-Full-Availability-of-ADSTILADRIN%C2%AE-nadofaragene-firadenovec-vncg-in -The United States