Landmark Study in Non-Muscle Invasive Bladder Cancer Evaluating Breakthrough Investigational Gene Therapy Nadofaragene Firadenovec Published in The Lancet Oncology

• Novel intravesical gene therapy given once every three months studied in patients with high-grade, BCG-unresponsive non-muscle invasive bladder cancer
• Phase 3 clinical trial met its primary endpoint with results demonstrating more than half of patients (53.4% of CIS ± Ta/T1 cohort) achieving a complete response (CR), all by three months¹
• 45.5% of patients in the CIS ± Ta/T1 cohort who achieved CR continued to remain free of high-grade recurrence at 12 months¹

CAMBRIDGE, Mass.–(BUSINESS WIRE)–FerGene Inc., today announced that The Lancet Oncology published the Phase 3 data from the landmark U.S. clinical trial evaluating an investigational gene therapy, nadofaragene firadenovec (rAd-IFN/Syn3), for the treatment of patients with high-grade, Bacillus Calmette-Guérin (BCG) unresponsive non-muscle invasive bladder cancer (NMIBC). In the study, patients received nadofaragene firadenovec, an intravesical therapy given once every three months that is believed to target the patient’s own bladder wall cells to enhance the body’s natural defenses to fight cancer.¹

The Phase 3 study of 157 patients from 33 U.S. sites met its primary endpoint with more than half (53.4%) of CIS ± Ta/T1 patients (carcinoma in situ; with or without concomitant high-grade Ta or T1 disease) achieving a complete response (CR), all by three months. Of the patients who achieved a CR, 45.5% continued to remain free of high-grade recurrence at 12 months. In the study, nadofaragene firadenovec was administered directly into the patient’s bladder once every three months by a healthcare professional. The long-term follow-up phase of the four-year study is ongoing, and patients are continuing to be monitored. (Read more…)¹

The most common adverse events (AEs) observed in the study that occurred in patients in order of decreasing frequency were: instillation site discharge, fatigue, bladder spasm, micturition urgency, and hematuria. The discontinuation rate due to AEs was 1.9%.¹

“Once patients with high-grade, non-muscle invasive bladder cancer no longer benefit from their initial BCG treatments, patients often make an informed decision to decline cystectomy – a highly complex and life-altering bladder removal surgery – or are often medically ineligible for this complex operation, leaving them with limited options,” said Colin P. N. Dinney, MD, Chairman, Department of Urology, Division of Surgery, University of Texas MD Anderson Cancer Center and senior author of the publication. “These data published in The Lancet Oncology show that nadofaragene firadenovec, a first-of-its-kind therapy, may be an effective treatment option for BCG-unresponsive non-muscle invasive bladder cancer patients.”

“As a practicing urologist, I’m encouraged by these efficacy and safety data which demonstrate the potential for a novel treatment option that fits within the urology practice and gives patients the choice of receiving treatment once every three months – which may be a particularly important consideration in this evolving healthcare environment,” said Gennady Bratslavsky, MD, President of the Society of Urologic Oncology Clinical Trials Consortium (SUO-CTC). “Our organization is proud to have played a key role in the mid- and late-stage clinical studies for nadofaragene firadenovec.”

Bladder cancer is the sixth most common cancer in the U.S., with NMIBC representing approximately 75% of all new bladder cancer cases.^2,3 BCG remains the first-line standard of care for people living with high-grade NMIBC, however up to 50% of high-grade patients who receive initial treatment with BCG will experience recurrence and disease progression within one year – becoming BCG-unresponsive.^4,5

“We believe the important clinical findings highlighted in The Lancet Oncology with this novel gene therapy may fulfill a significant unmet need for patients and have the potential to be practice-changing,” said Vijay Kasturi, MD, Vice President of Medical Affairs at FerGene Inc. “We are extremely grateful to the investigators, the patients who participated in the study, FKD Therapies and the SUO-CTC for the important findings highlighted in this publication.”

A Biologics License Application (BLA) for nadofaragene firadenovec is currently with the U.S. Food and Drug Administration (FDA).

About Nadofaragene Firadenovec

Nadofaragene firadenovec (rAd-IFN/Syn3) is an investigational gene therapy being developed as a treatment for patients with high-grade, BCG-unresponsive NMIBC. It is a non-replicating adenovirus vector-based gene therapy containing the gene interferon alfa-2b, administered by catheter into the bladder once every three months. The vector enters the cells of the bladder wall, releasing the active gene to do its work. The internal gene/DNA machinery of the cells ‘picks up’ the gene and translates its DNA sequence, resulting in the cells secreting high quantities of interferon alfa-2b protein, a naturally occurring protein the body uses to fight cancer. This novel gene therapy approach thereby turns the patient’s own bladder wall cells into interferon microfactories, enhancing the body’s natural defenses against the cancer. Nadofaragene firadenovec has been studied in a clinical trial program that includes 221 patients with high-grade, BCG-unresponsive NMIBC who had been treated with adequate BCG previously and did not see benefit from additional BCG treatment.

About Non-Muscle Invasive Bladder Cancer (NMIBC)

NMIBC is a form of bladder cancer which is present in the superficial layer of the bladder and has not invaded deeper into the bladder or spread to other parts of the body.⁴ It is estimated that there will be approximately 81,000 new cases of bladder cancer in the U.S. in 2020⁶; 75% of these cases present as NMIBC.³ In patients with high-grade NMIBC, intravesical BCG is the recommended treatment; however, up to 50% of high-grade patients will experience disease recurrence within one year.^4,5 The outcome for BCG-unresponsive patients is poor, with chemotherapy and radiation or total cystectomy (complete removal of the bladder) often being the recommended next treatment options.⁷

About the Society of Urologic Oncology Clinical Trials Consortium (SUO-CTC)

Created, owned and operated by its members, the SUO-CTC is a clinical research investigator network of over 500 members from more than 200 clinical sites in the U.S. and Canada. This national alliance of leading academic and community based uro-oncologists is committed to furthering urology research. The SUO-CTC is a registered 501c3 not-for-profit corporation and has a cooperative relationship with the Society of Urologic Oncology (SUO). The SUO-CTC pursues clinical trials, in concert with sponsors, to investigate therapeutic interventions which address urological cancers including, but not restricted to: Bladder Cancer, Prostate Cancer and Renal Cancer. Together with industry, the SUO-CTC offers enhanced research options for ultimately delivering better quality of life to our patients.

About FerGene Inc.

FerGene Inc. is a gene therapy company committed to revolutionizing the treatment of bladder cancer through its innovative science and unparalleled commitment to patient care. Founded in 2019, as a result of a collaboration between Blackstone Life Sciences and Ferring Pharmaceuticals, FerGene Inc. is singularly focused on evolving the bladder cancer treatment landscape through its novel approach to gene therapy. A trusted partner to medical and advocacy communities, FerGene Inc. is dedicated to bringing new hope to a patient population which has seen little improvement in their standard of care over the past twenty years. For more information, please visit www.fergene.com or engage with us on Twitter at @FerGeneBio or on LinkedIn.

© 2020 FerGene Inc. 20/09 US- ADST-2000114

¹ Boorjian, S., Alemozaffar, M., Konety, B., Shore, N., Gomella, L., Kamat, A. et al. Intravesical nadofaragene firadenovec gene therapy for BCG-unresponsive non-muscle-invasive bladder cancer: a single-arm, open-label, repeat-dose clinical trial. Lancet Oncol. 2020;2045(20)30540-4. doi:10.1016/S1470.

² National Cancer Institute SEER Program. Cancer Stat Facts: Common Cancer Sites. https://seer.cancer.gov/statfacts/html/common.html. Accessed March 5, 2020.

³ Burger M, Catto JW, Dalbagni G, et al. Epidemiology and risk factors of urothelial bladder cancer. Eur Urol. 2013;63(2):234-41. doi:10.1016/j.eururo.2012.07.033.

⁴ Sanli, O., Dobruch, J., Knowles, M. et al. Bladder cancer. Nat Rev Dis Primers. 3,17022 (2017) doi:10.1038/nrdp.2017.22.

⁵ Kamat AM, Li R, O’Donnell MA, et al. Predicting response to intravesical Bacillus Calmette-Guérin immunotherapy: Are we there yet? A systematic review. Eur Urol. 2018;73(5):738-748. doi:10.1016/j.eururo.2017.10.003

⁶ American Cancer Society. Key Statistics for Bladder Cancer. https://www.cancer.org/cancer/bladder-cancer/about/key-statistics.html. Updated 2020. Accessed March 5, 2020.

⁷ Marqueen K, et al. Identifying high surgical risk in muscle-invasive bladder cancer (MIBC) patients undergoing radical cystectomy (RC). JNCI Cancer Spectr. 2018 Oct; 2(4): pky075.

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