Ferring Pharmaceuticals has launched 3 new clinical trials for nadofaragene firadenovec (Adstiladrin), a marketed adeno-associated virus (AAV) vector-based gene therapy currently approved for the treatment of adult patients with high-risk, Bacillus Calmette-Guerin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.1 The 3 new clinical trials will take place in the United States and are intended to increase the amount of accumulated data supporting the use of Adstiladrin in NMIBC.
One of these studies, ABLE-22, will evaluate Adstiladrin alone or in combination with a chemotherapy regimen consisting of gemcitabine and docetaxel or an immunotherapy regimen consisting of pembrolizumab. ABLE-22, which is a phase 2 randomized, multicenter, open-label study, will seek to enroll adults with high-grade BCG-unresponsive NMIBC. Another of the 3 new studies is ABLE-32, a randomized controlled trial that will evaluate the gene therapy against observation in participants with intermediate-risk NMIBC. The other new trial is ABLE-42, a phase 4 clinical trial that will investigate retreatment with Adstiladrin in patients that were previously treated with Adstiladrin under its current indication, but whose disease did not respond to the gene therapy at their first 3-month evaluation. ABLE-42 is an open-label, multicenter study.
“Adstiladrin is an established monotherapy treatment option for patients with BCG-unresponsive NMIBC disease, an area where patients’ treatment options have been severely limited for decades,” Pierre-Yves Berclaz, MD, PhD, the executive vice president and chief science and medical officer of Ferring Pharmaceuticals, said in a statement.1 “These new trials underscore Ferring's commitment to create solutions for patients by enlarging the body of evidence for this novel nonreplicating gene therapy.”
Key Takeaways
- Ferring Pharmaceuticals has launched 3 new clinical trials for nadofaragenefiradenovec (Adstiladrin), a marketed adeno-associated virus (AAV) vector-based gene therapy currently approved for the treatment of adult patients with high-risk, Bacillus Calmette-Guerin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) with or without papillary tumors.
- The 3 new clinical trials will take place in the United States and are intended to increase the amount of accumulated data supporting the use of Adstiladrin in NMIBC.
- These 3 new trials are additions to an ongoing set of clinical trials for Adstiladrin that also includes the ongoing noninterventional real-world evidence study ABLE-41 (NCT06026332) and the Japan Phase 3B Study (000381; NCT05704244).
- Adstiladrin was originally approved by the FDA for its current indication in December 2022.
These 3 new trials are additions to an ongoing set of clinical trials for Adstiladrin that also includes the ongoing noninterventional real-world evidence study ABLE-41 (NCT06026332) and the Japan Phase 3B Study (000381; NCT05704244). ABLE-41, which enrolled its first patient in September of last year, is observing US patients who are receiving Adstiladrin for the first time in the commercial setting under its FDA-approved indication, with a focus on learning about experiences and outcomes with the gene therapy in this setting. The Japan Phase 3B Study, on the other hand, is a single arm bridging study for Adstiladrin in patients in Japan who are aged 20 years or older and have high-grade, BCG-unresponsive NMIBC.
Adstiladrin was originally approved by the FDA for its current indication in December 2022.2 The FDA’s decision was based on data from an open-label phase 3 clinical trial (NCT02773849) that included 157 participants. Results reported prior to the approval indicated that more than half of patients treated (51%; 95% CI, 41-61) achieved a complete response by 3 months.3 Among those who reached an initial complete response, 46% remained free of high-grade recurrence at 12 months (median duration, 9.7 months).
Following the FDA’s approval, Ferring announced that the first patient had been treated with Adstiladrin in the commercial setting as part of an Early Experience Program.4 In January 2024, Ferring reported that the gene therapy had been produced in sufficient product supply levels to allow for wide availability to patients in need.5 As a result, Ferring put a wrap on the Early Experience Program.
“Adstiladrin is the foundation of Ferring's leadership mission in the uro-oncology therapeutic category and our ambition is for it to become the new standard of care and the backbone therapy for patients across the NMIBC disease spectrum,” Bipin Dalmia, the senior vice president and the global head of the Uro-Oncology Franchise at Ferring Pharmaceuticals, added to the statement.1 “Embarking on a broad research and geographic expansion program is yet another demonstration of our commitment to the uro-oncology community, together with our significant manufacturing investments that recently enabled full availability of Adstiladrinto every appropriate US patient who needs it.”
REFERENCES
1. Ferring adds three new studies to non-muscle invasive bladder cancer clinical trial program with Adstiladrin® (nadofaragenefiradenovec-vncg). News release. Ferring Pharmaceuticals. April 16, 2024. Accessed April 18, 2024.https://www.businesswire.com/news/home/20240415649389/en/Ferring-Adds-Three-New-Studies-to-Non-Muscle-Invasive-Bladder-Cancer-Clinical-Trial-Program-With-ADSTILADRIN%C2%AE-nadofaragene-firadenovec-vncg
2. FDA Approves First Gene Therapy for the Treatment of High-Risk, Non-Muscle-Invasive Bladder Cancer. News release. FDA. December 16, 2022. Accessed April 18, 2024. https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-high-risk-non-muscle-invasive-bladder-cancer
3. Ferring Receives Approval from U.S. FDA for Adstiladrin for High-Risk, BCG-Unresponsive Non-Muscle Invasive Bladder Cancer. News release. Ferring. December 16, 2022. Accessed April 18, 2024. https://www.ferring.com/ferring-receives-approval-from-u-s-fda-for-adstiladrin-for-high-risk-bcg-unresponsive-non-muscle-invasive-bladder-cancer/
4. First bladder cancer patient dosed with commercially available intravesical gene therapy ADSTILADRIN® (nadofaragenefiradenovec-vncg). News release. September 12, 2023. Accessed April 18, 2024. Ferring Pharmaceuticals. https://www.businesswire.com/news/home/20230912802578/en/First-Bladder-Cancer-Patient-Dosed-with-Commercially-Available-Intravesical-Gene-Therapy-ADSTILADRIN%C2%AE-nadofaragene-firadenovec-vncg
5. Ferring announces full availability of ADSTILADRIN® (nadofaragene firadenovec-vncg) in the U.S. News release. Ferring Pharmaceuticals. January 16, 2024. Accessed April 18, 2024. https://www.ferring.com/ferring-announces-full-availability-of-adstiladrin-nadofaragene-firadenovec-vncg-in-the-u-s/
