Adstiladrin (nadofaragene firadenovec-vncg) / Ferring, FKD Therapies, Royalty - LARVOL DELTA

Intravesical instillation of a TME engineering viral vector increases survival in an orthotropic bladder cancer model (AACR 2025) - "Non-muscle invasive urinary bladder cancer is currently treated with bacillus Calmette-Guérin (BCG) instillations followed by nadofaragene firadenovec-vncg for BCG refractory patients...We hypothesize that LOAd703, the human equivalent to Adm703, may be an interesting option for these patients...In conclusion, TME gene engineering with TMZ-CD40L and 4-1BBL increased survival of mice with orthotopic bladder cancer. Further studies investigating Adm703's effect on the immune system are planned."

Preclinical • Viral vector • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • CD40LG

ABLE-22: A Trial to Evaluate Intravesical Nadofaragene Firadenovec Alone or in Combination With Chemotherapy or Immunotherapy in Participants With High-grade BCG Unresponsive Non-muscle Invasive Bladder Cancer (clinicaltrials.gov) - P2 | N=150 | Recruiting | Sponsor: Ferring Pharmaceuticals | Trial completion date: Jul 2028 ➔ Dec 2030 | Trial primary completion date: Jul 2026 ➔ Dec 2030

Trial completion date • Trial primary completion date • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

U.S. FDA Approves Second Drug Product Manufacturing Facility for ADSTILADRIN (nadofaragene firadenovec-vncg) (Businesswire) - "Ferring Pharmaceuticals announced today the U.S. Food and Drug Administration (FDA) approved a state-of-the-art drug product manufacturing hub in Parsippany, NJ, for its intravesical non-replicating gene therapy ADSTILADRIN (nadofaragene firadenovec-vncg). The approval expands Ferring’s manufacturing capabilities to three cutting-edge sites dedicated to bringing ADSTILADRIN to patients and secures a final $200 million payment from Royalty Pharma (Nasdaq: RPRX) as part of a royalty-based financing agreement announced in 2023....'This expansion and diversification of our manufacturing footprint will further ensure stable and sustainable supply of ADSTILADRIN to meet the anticipated growth in global demand.'"

Commercial • Bladder Cancer

ABLE-41, a real-world evidence study for bladder cancer patients treated with nadofaragene firadenovec: Baseline patient characteristics and demographics. (ASCO 2025) - P | "Clinical Trial Registration Number: NCT06026332 The abstract will be released to the public on May 22, 2025 at 5:00 PM EDT"

Clinical • HEOR • Real-world • Real-world evidence • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

ABLE-22: Safety and efficacy evaluation of nadofaragene firadenovec alone or in combination with chemotherapy or immunotherapy—A randomized, open-label, phase 2 study. (ASCO 2025) - P2 | "Clinical Trial Registration Number: NCT06545955 The abstract will be released to the public on May 22, 2025 at 5:00 PM EDT"

Clinical • Combination therapy • P2 data • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

ABLE-32: A randomized, controlled, phase 3b clinical trial of nadofaragene firadenovec-vncg versus observation in patients with intermediate-risk non–muscle-invasive bladder cancer. (ASCO 2025) - P3 | "Clinical Trial Registration Number: NCT06510374 The abstract will be released to the public on May 22, 2025 at 5:00 PM EDT"

Clinical • P3 data • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

Final Five-Year Analysis of Phase 3 Data With ADSTILADRIN (nadofaragene firadenovec-vncg) Shows Durable Bladder Preservation and Consistent Long-Term Safety in BCG-unresponsive NMIBC (Businesswire) - P3 | N=157 | NCT02773849 | Sponsor: Ferring Pharmaceuticals | "Ferring Pharmaceuticals today announced the final 60-month follow-up data from the Phase 3 study of ADSTILADRIN (nadofaragene firadenovec-vncg) were presented at the American Urological Association (AUA) 2024 Annual Meeting....Median follow-up was 50.8 months (IQR 39.1-60.0), with 26.8% receiving five instillations and 7.6% receiving treatment for 57 months. Among patients who were high-grade recurrence free (HGRF) at Month 3, 13 patients were followed and remained HGRF at month 57....The overall survival at 60 months was 76.3% in the CIS cohort and 85.9% in the PD cohort. The cystectomy-free survival rate at Month 60 was 43.2.% and 58.7% in the CIS and PD cohorts, respectively. Clinical progression to muscle invasion was rare in the trial population and seen in only 3.3% of all patients (four with CIS and one with Ta/T1) over the five-year follow-up period."

P3 data • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

Ferring Unveils New Data with ADSTILADRIN (nadofaragene firadenovec-vncg) at 112th Annual Meeting of the Japanese Urological Association (Businesswire) - P3 | N=25 | NCT05704244 | "Results showed 75% complete response rate at three months in high-risk Bacillus Calmette-Guérin (BCG)-unresponsive NMIBC patients receiving ADSTILADRIN (n=20) and is in addition to the independent real-world data presented earlier this year that showed a 79% complete response rate. Preliminary safety profile shows all treatment-related adverse events were Grade 1 (84.2%) or Grade 2 (15.8%), with no Grade 3, 4, or 5 adverse events reported."

P3 data • Bladder Cancer • Urothelial Cancer

Use of Rectal Diazepam to Prevent Bladder Spasms and Leakage of Medication During Intravesical Administration of Nadofaragene Firadenovec in BCG-Unresponsive Non-Muscle-Invasive Bladder Cancer (AUA 2025) - "Median number of prior BCG instillations was 12 (IQR, 9-15), while 29 (64.4%) patients had previously received intravesical chemotherapy and 11 (24.4%) had previously received pembrolizumab. Rectal diazepam pretreatment reduces bladder spasms and improves nadofaragene retention during intravesical instillation, thereby enhancing patient comfort and medication delivery. Further investigation regarding the impact on treatment efficacy is warranted through larger, prospective studies."

Bladder Cancer • Fatigue • Genito-urinary Cancer • Oncology • Solid Tumor

Nursing Considerations for the Use of Intravesical Nadofaragene Firadenovec-vncg in Patients With Bacillus Calmette-Guérin–Unresponsive Non–Muscle-Invasive Bladder Cancer (ONS 2025) - "Discussion Healthcare providers administering intravesical nadofaragene firadenovec help with visit coordination, drug administration, monitoring, supportive care, and education. Understanding best practices will help optimize safety and efficacy of nadofaragene firadenovec instillation and improve patient and caregiver experiences."

Clinical • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer

Operationalizing a Breakthrough Bladder Cancer Medication with a Multidisciplinary Approach: Insights from the Nursing Team (ONS 2025) - "Purpose Adstiladrin is a gene therapy, unique compared to traditional chemotherapy or immunotherapy...This collaborative approach set a replicable model for other institutions. Sharing these best practices can empower more facilities to successfully adopt this new therapy, ultimately providing greater access to essential care for patients in need."

Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

A Study Comparing Standard Treatments in People With Non-Muscle Invasive Bladder Cancer (NMIBC) (clinicaltrials.gov) - P3 | N=125 | Recruiting | Sponsor: Memorial Sloan Kettering Cancer Center

New P3 trial • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

Novel combination therapy using lentiviral interferon and immune checkpoint blockade for treatment of bladder cancer in a murine model (AUA 2025) - "Combination therapy with LV-IFNα and anti-PD-1 eradicated bladder tumors in immune-competent mice and improved OS. Combination therapy resulted in upregulation of IFN and immune-related pathways. These findings suggest a therapeutic role for immune checkpoint inhibitors in BCG-unresponsive NMIBC patients treated with nadofaragene."

Checkpoint block • Checkpoint inhibition • Combination therapy • IO biomarker • Preclinical • Bladder Cancer • Gene Therapies • Genito-urinary Cancer • Oncology • Solid Tumor • IFNA1 • IL6 • PD-L1

Ferring achieved record sales driven by Reproductive Medicine combined with the emergence of Adstiladrin (Businesswire) - "Ferring achieved total revenues of €2.3 billion in 2024, an increase of 7% over the previous year at actual exchange rates (AER) and 8% at constant exchange rates (CER). This above market growth reflected the continued strong performance in Reproductive Medicine, which exceeded €1 billion in global sales for the first time. In addition, 2024 was the first full year of U.S. sales of Adstiladrin, our novel gene-based therapy for bladder cancer. Operating expenses were carefully managed while prioritising investment in growth opportunities, such as Adstiladrin."

Commercial • Bladder Cancer

Safety of nadofaragene firadenovec-vncg: review of data from phase 2 and phase 3 studies. (PubMed, Can J Urol) - P2, P3 | "Nadofaragene firadenovec demonstrated a favorable and tolerable safety profile across its clinical study program, allowing for broad patient selection among those with high-risk BCG-unresponsive NMIBC."

Journal • P2 data • P3 data • Review • Bladder Cancer • Fatigue • Gene Therapies • Genito-urinary Cancer • Oncology • Solid Tumor

Matching-Adjusted Indirect Comparisons (MAIC) of TAR-200 vs FDA Approved Novel Agents in Bacillus Calmette-Guerin (BCG) Unresponsive High-Risk (HR) Non-Muscle Invasive Bladder Cancer (NMIBC) with Carcinoma in Situ (CIS) (ISPOR 2025) - "OBJECTIVES: FDA has approved pembrolizumab (pembro), nadofaragene firadenovec-vncg (nadofaragene), and nogapendekin alfa inbakicept-pmln (NAI) + BCG for the treatment of patients with BCG-unresponsive HR NMIBC with CIS. TAR-200 demonstrated statistically significant clinical benefit in CR rate at any time over FDA approved novel agents in BCG-unresponsive HR NMIBC with CIS and at first disease assessment compared to NAI + BCG. Further analyses will be conducted once additional follow-up is available for TAR-200."

Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

ABLE-32: Trial of Nadofaragene Firadenovec Vs. Observation in Participants with Intermediate Risk Non-Muscle Invasive Bladder Cancer (clinicaltrials.gov) - P3 | N=454 | Recruiting | Sponsor: Ferring Pharmaceuticals | Trial completion date: Jan 2031 ➔ Jun 2031

Trial completion date • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

Nadofaragene Firadenovec Is Effective With Beneficial Dosing Schedule in Real-World for BCG-Unresponsive NMIBC (OncLive) - "The favorable dosing schedule of nadofaragene firadenovec-vncg (Adstiladrin) makes it a suitable option for patients with BCG-unresponsive non–muscle-invasive bladder cancer (NMIBC) who have to travel far for care, and patient selection must be done carefully as not to include those who are likely to leak the drug...Data from a real-world study conducted by Moyer, Tyson, and colleagues at Mayo Clinic locations revealed that, at a median follow-up of 8.2 months, patients who received the intravesical therapy (n = 43) achieved a cystectomy-free survival rate of 95% and all patients were alive at the data cutoff."

Real-world • Bladder Cancer • Urothelial Cancer

Nadofaragene Firadenovec Shows CR and Tolerability in Patients With NMIBC (Pharmacy Times) - "Real-world evidence from a multisite, single-institution cohort of patients with Bacillus Calmette-Guérin (BCG)-unresponsive non-muscle invasive bladder cancer (NMIBC) found thatnadofaragene firadenovec (Adstiladrin; Ferring Pharmaceuticals) was effective at inducing a complete response (CR) and a sustained duration of response (DOR) and featured a tolerable safety profile. The data were presented by Mayo Clinic at the 2025 American Society of Clinical Oncologists Genitourinary Cancers (ASCO GU) Symposium in San Francisco, California....There were 24 patients with CIS with or without papillary disease who were evaluable for efficacy. Of these, 79% had a CR observed at 3 months. Importantly, the median DOR was not reached; at a median follow-up point of about 7.3 months, 84% (16/19) of treatment responders continued to report CRs."

Real-world evidence • Bladder Cancer

Advancements in the Diagnosis, Treatment, and Risk Stratification of Non-Muscle Invasive Bladder Cancer. (PubMed, Curr Oncol Rep) - "Advances in immunotherapy, including the use of pembrolizumab and nadofaragene firadenovec, offer options for BCG-unresponsive NMIBC, though challenges like cost and adverse effects remain. Future directions highlight the need for biomarker-driven models to refine treatment paradigms, reduce overtreatment, and improve long-term outcomes. Continued clinical trials are essential to validate these approaches and address unmet needs in NMIBC care."

IO biomarker • Journal • Review • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • FGFR3 • TERT

Practical use of nadofaragene firadenovec-vncg for pharmacists. (PubMed, J Oncol Pharm Pract) - "Pharmacists are positioned to be key contributors to the care team regarding the effective use of nadofaragene firadenovec. Further awareness and education will continue to optimize the clinical use of nadofaragene firadenovec in the high-risk BCG-unresponsive NMIBC setting."

Journal • Review • Bladder Cancer • Gene Therapies • Genito-urinary Cancer • Oncology • Solid Tumor

ABLE-22: Safety and efficacy evaluation of nadofaragene firadenovec alone or in combination with chemotherapy or immunotherapy—A randomized, open-label, phase 2 study. (ASCO-GU 2025) - P2 | "Bladder-preserving treatment options for patients with BCG-unresponsive NMIBC with CIS ± Ta/T1 include intravesical gene therapy (nadofaragene firadenovec-vncg), intravesical chemotherapy (gemcitabine and docetaxel), and immunotherapy (intravenous pembrolizumab and intravesical nogapendekin alfa inbakicept-pmln). Exploratory endpoints include changes in expression of potential biomarkers in blood and urine. Results from this investigational, randomized, multicenter, open-label study evaluating the safety and efficacy of nadofaragene firadenovec alone or in combination with chemotherapy or immunotherapy are expected July 2028."

Clinical • Combination therapy • IO biomarker • P2 data • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

ABLE-32: A randomized, controlled, phase 3b clinical trial of nadofaragene firadenovec-vncg versus observation in patients with intermediate-risk non–muscle-invasive bladder cancer. (ASCO-GU 2025) - P3 | "Exploratory endpoints include effect on potential biomarkers and health-related quality of life. Final results are expected in 2031."

Clinical • P3 data • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor

Real-world outcomes of nadofaragene firadenovec in BCG-unresponsive non-muscle invasive bladder cancer. (ASCO-GU 2025) - "Early real-world data demonstrates encouraging clinical complete response rates in patients with BCG-unresponsive NMIBC and a favorable safety profile. Further investigation with larger cohorts and longer follow-up is warranted. Data are median (IQR) or % [95% CI]."

Clinical • Real-world • Real-world evidence • Bladder Cancer • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer

New Treatment Options for Non-Muscle-Invasive Bladder Cancer. (PubMed, Am Soc Clin Oncol Educ Book) - "Emerging approaches such as TAR-200 and UGN-102 offer novel intravesical delivery systems that enhance therapeutic efficacy while minimizing systemic adverse effects. Viral therapies, including nadofaragene firadenovec and CG0070, deliver immune-activating and oncolytic agents directly to urothelial tumor cells. Additionally, immune checkpoint inhibitors such as pembrolizumab and durvalumab have demonstrated potential for systemic treatments in BCG-unresponsive NMIBC and may show even more promise in combinations...For low-grade NMIBC, efforts are underway to de-escalate care through active surveillance and novel adjuvant therapies, reducing the need for repeated TURBT procedures. Together, these advancements highlight a promising shift toward personalized, bladder-preserving strategies that prioritize patient quality of life while addressing unmet needs in NMIBC management."

Journal • Review • Bladder Cancer • Gene Therapies • Genito-urinary Cancer • Oncology • Solid Tumor • Urothelial Cancer