Ferring Advances Three ADSTILADRIN® Clinical Studies

Ferring Pharmaceuticals has progressed with three clinical studies involving ADSTILADRIN (nadofaragene firadenovec-vncg), targeting non-muscle invasive bladder cancer (NMIBC) and upper tract urothelial cancer (UTUC). The company revealed that they will present the study protocol for patients with intermediate-risk NMIBC and new data from a Phase 3 study at the Society of Urologic Oncology's 25th Annual Meeting. This data shows that patients with high-risk Bacillus Calmette-Guérin (BCG)-unresponsive NMIBC experienced significant cystectomy-free survival over five years after three months of complete response with ADSTILADRIN.

ADSTILADRIN, which is the first FDA-approved gene therapy for high-risk BCG-unresponsive NMIBC with carcinoma in situ (CIS) with or without papillary tumors, is a non-replicating gene therapy delivered intravesically.

In the U.S., Ferring activated study sites for two clinical trials in the ABLE (ADSTILADRIN in Bladder Cancer) program for NMIBC. The Phase 2 ABLE-22 trial evaluates ADSTILADRIN's efficacy and safety as a monotherapy and in combination with chemotherapy or an immune checkpoint inhibitor for high-risk BCG-unresponsive NMIBC. A notable aspect of this study is the potential for re-induction for patients not achieving a complete response initially. The Phase 3B ABLE-32 trial assesses ADSTILADRIN's safety and efficacy in patients with intermediate-risk NMIBC, a group that currently lacks FDA-approved treatments in the U.S.

Additionally, the LUNAR (Low-Grade UTUC Treated with Nadofaragene Firadenovec Administered to Renal Pelvis) study, a Phase 1-2 trial, has started. This multi-center, multi-national study aims to evaluate the safety, tolerability, and efficacy of ADSTILADRIN instilled in the renal pelvis for patients with low-grade UTUC.

Dr. Joern Jakobsen, Vice President and Head of Global Research and Medical for Uro-Oncology and Urology at Ferring Pharmaceuticals, emphasized the potential of ADSTILADRIN to offer organ-sparing treatment options for NMIBC and UTUC patients. Dr. Jakobsen noted that expanding the ADSTILADRIN clinical program to include intermediate-risk NMIBC and low-grade UTUC, along with generating data on monotherapy and combination treatments for high-risk BCG-unresponsive NMIBC, is crucial for understanding how ADSTILADRIN could benefit more patients.

Dr. Colin P.N. Dinney from the University of Texas MD Anderson Cancer Center, a lead investigator of the Phase 3 study, highlighted that the trial has significantly advanced the understanding of treating BCG-unresponsive NMIBC. He noted that the study paved the way for re-induction strategies in subsequent trials.

Ferring is prioritizing the collection of ADSTILADRIN re-induction data for high-risk BCG-unresponsive NMIBC patients who did not achieve a complete response to the initial dose. This data is being collected from clinical settings through studies like ABLE-22 and ABLE-41, while the previously announced ABLE-42 clinical trial has been discontinued.

The ABLE-41 study, currently enrolling patients, is a non-interventional study gathering real-world evidence on the use of ADSTILADRIN in routine care for high-risk BCG-unresponsive NMIBC patients. The LUNAR study will assess the safety and efficacy of ADSTILADRIN in patients with low-grade UTUC, with a safety lead-in period for the first patients.

ADSTILADRIN (nadofaragene firadenovec-vncg) is a non-replicating adenovirus vector-based gene therapy approved for high-risk BCG-unresponsive NMIBC with carcinoma in situ. The therapy involves administering the gene interferon alfa-2b directly into the bladder every three months, enabling the bladder cells to produce interferon, which helps combat cancer. This approach turns bladder wall cells into microfactories of interferon, boosting the body's natural defenses against cancer.

Ferring Pharmaceuticals continues to drive forward in uro-oncology treatment by expanding access to ADSTILADRIN and investing in new therapies. The company, founded in 1950 and headquartered in Switzerland, is committed to addressing unmet medical needs and improving patients' lives.