This phase II trial tests the effect of intravesical nadofaragene firadenovec in combination with gemcitabine, cisplatin and durvalumab before (neoadjuvant) radical cystectomy (RC) in treating patients with muscle invasive bladder cancer. The combination of gemcitabine, cisplatin and durvalumab are already considered standard of care in the treatment of muscle invasive bladder cancer. This trial attempts to determine whether the addition of nadofaragene firadenovec to the current standard regiment is safe and can improve oncological outcomes for those with muscle invasive bladder cancer.
Nadofaragene firadenovec, a type of intravesical gene therapy, is a weakened adenovirus that carries a copy of the gene for interferon alfa-2b. This medication gets absorbed by the bladder and stimulates the bladder to naturally create interferon alfa-2b, which is thought to kill bladder cancer. Nadofaragene firadenovec is given in a solution that is placed directly into the bladder (intravesical) using a thin tube called a catheter. It is a medication that is already FDA approved for the treatment of non-muscle invasive bladder cancer. Gemcitabine is a chemotherapy drug that blocks the cells from making deoxyribonucleic acid and may kill tumor cells. Cisplatin is in a class of medications known as platinum-containing compounds. It works by killing, stopping or slowing the growth of tumor cells. Immunotherapy with monoclonal antibodies, such as durvalumab, may help the body's immune system attack the tumor, and may interfere with the ability of tumor cells to grow and spread.

Start Date01 Jun 2026

Sponsor / Collaborator

University of Washington

[+1]

NCT06668493

/ RecruitingPhase 1/2

A Phase 1/2, Single-arm, Open-Label Trial to Evaluate the Safety and Efficacy of Nadofaragene Firadenovec Instilled to the Renal Pelvis in Adult Subjects With Low-grade Upper Tract Urothelial Carcinoma (LG-UTUC)

The primary purpose of this trial is to evaluate the safety & tolerability of Nadofaragene Firadenovec in subjects with LG-UTUC. To help with this evaluation, a safety lead-in period will be conducted for the first 6 subjects. Complete response is at 3 or 6 months defined as absence of any UTUC in the renal pelvis.

Start Date12 Jun 2025

Sponsor / Collaborator

Ferring Pharmaceuticals SA

NCT06929286

/ RecruitingPhase 3IIT

COMparative Prospective Assessment Through Clinically Integrated Randomized Trials Evaluating Intravesical Treatments: The COMPARE IT Trial

The purpose of this study is to compare the effectiveness of different FDA-approved/NCCN-recommended drug treatments for NMIBC. In particular, the FDA-approved drug nadofaragene firadenovec will be compared to usual care with other NCCN-recommended standard treatments for NMIBC (gemcitabine with or without docetaxel, mitomycin, re-treatment with BCG, or pembrolizumab).

Start Date11 Apr 2025

Sponsor / Collaborator

Memorial Sloan Kettering Cancer Center

100 Clinical Results associated with Nadofaragene firadenovec-vncg

100 Translational Medicine associated with Nadofaragene firadenovec-vncg

100 Patents (Medical) associated with Nadofaragene firadenovec-vncg

Literatures (Medical) associated with Nadofaragene firadenovec-vncg

01 Feb 2026·UROLOGIC ONCOLOGY-SEMINARS AND ORIGINAL INVESTIGATIONS

Real-world outcomes of nadofaragene firadenovec for BCG-unresponsive non–muscle-invasive bladder cancer: A multicenter experience

Article

Author: Durant, Adri M ; Mi, Lanyu ; Nguyen, Mimi V ; Moyer, Jacob A ; Boorjian, Stephen A ; Tyson, Mark D ; Zganjar, Andrew J ; Shah, Paras H ; Lyon, Timothy D

BACKGROUND:

Nadofaragene firadenovec-vncg received US Food and Drug Administration approval for bacille Calmette-Guérin (BCG)-unresponsive non-muscle-invasive bladder cancer (NMIBC) based on prospective efficacy and safety results. However, postmarketing data are lacking. This study evaluates outcomes in a real-world setting.

METHODS:

We analyzed data for patients treated at Mayo Clinic from November 2023 through December 2024. Outcomes included complete response (CR) rate, duration of response, adverse events, and high-grade recurrence-free (HGRFS), cystectomy-free, and overall survival.

RESULTS:

Forty-six patients were treated with nadofaragene firadenovec; 3 with pending posttreatment cystoscopy were excluded. Of 24 evaluable patients with carcinoma in situ with/without papillary disease, 79% had a CR at 3 months. The median response duration was not reached; 68% (13/19) of responders still had CRs with median follow-up of 9.3 months. Of 19 patients with papillary-only disease (median follow-up of 7.6 months), HGRFS at 3, 6, and 12 months was 68%, 53%, and 26%, respectively. With median follow-up of 13.9 months, cystectomy-free and overall survival were 93% and 95%, respectively. Progression occurred in 5 patients. The most common adverse events were grade 1-2 bladder spasms (61%) and failure to fully retain the instillation (33%). Four patients had grade 3 events; there were no grade 4 or 5 events. Limitations include the small sample size and limited follow-up.

CONCLUSIONS:

Real-world data confirm the efficacy and safety of nadofaragene firadenovec, clarifying its role in the management of BCG-unresponsive NMIBC.

01 Jan 2026·Gan to kagaku ryoho. Cancer & chemotherapy

[Evolving Pharmacotherapy for Bladder Cancer-From Non-Muscle-Invasive to Metastatic Disease-Development of Novel Therapies for BCG-Unresponsive NMIBC].

Article

Author: Inoue, Keiji ; Shigehisa, Ryu ; Fukata, Satoshi

Bladder cancer is a common malignancy in the elderly, with approximately 75% of cases diagnosed as non-muscle-invasive bladder cancer(NMIBC).Although transurethral resection of bladder tumor(TURBT)is the standard initial treatment, recurrence rates remain high, and intravesical Bacillus Calmette-Guérin(BCG)instillation has long been used as an adjuvant therapy.However, about 30% of patients develop BCG-unresponsive disease despite adequate BCG administration.Radical cystectomy is recommended in such cases, but its invasiveness and impact on quality of life(QOL)often make it impractical in real-world settings.Thus, the development of novel bladder-preserving treatments is urgently needed.In recent years, diverse approaches including immune checkpoint inhibitors(ICI), gene therapy, drug delivery systems(DDS), and oncolytic virus-based therapies have been actively investigated, with numerous clinical trials ongoing worldwide.Importantly, pembrolizumab, nadofaragene firadenovec, and N-803 in combination with BCG have received FDA approval, representing major advances in the management of BCG-unresponsive NMIBC.In addition, promising agents such as TAR-200, cretostimogene grenadenorepvec, and detalimogene voraplasmid are under clinical evaluation.These advances indicate the potential for novel strategies that enable bladder preservation while achieving tumor control.Future challenges include validation of long-term outcomes, establishment of evidence through randomized controlled trials, and regulatory approval and reimbursement in Japan.Expansion of therapeutic options is expected to improve both prognosis and QOL in this high-risk population.

01 Nov 2025·JOURNAL OF UROLOGY

Outcomes of Reinduction With Nadofaragene Firadenovec

Author: Daneshmand, Siamak ; Luckenbaugh, Amy N. ; Dickstein, Rian ; Narayan, Vikram M. ; Lotan, Yair ; Agarwal, Piyush K. ; Tyson, Mark D. ; Horns, Joshua J. ; Schmidt, Bogdana ; Linehan, Jennifer A.

News (Medical) associated with Nadofaragene firadenovec-vncg

10 Mar 2026

·www.biospace.com

For the First Time, Ferring Reports Revenue of Over €2.5 Billion in 2025

Ferring's total revenues for 2025 exceeded €2.5 billion, an increase of 10% from 2024, mainly driven by our flagship product Menopur ® Continued ramp-up in the US for Adstiladrin ® , our novel gene-based therapy for bladder cancer, confirming its position as Ferring’s second major growth driver Commitment to sustainability demonstrated by SBTi approval of our targets to reduce greenhouse gas emissions, and by our programme to reduce maternal deaths in low- and lower middle-income countries SAINT-PREX, Switzerland--(BUSINESS WIRE)--Ferring today published its 2025 Annual Report and Sustainability Report . The company achieved total revenues of over €2.5 billion in 2025, an increase of 7% over the previous year at actual exchange rates (AER) and 10% at constant exchange rates (CER). These were mainly driven by our flagship product Menopur ® (menotropins for injection) in reproductive medicine, and the ramp-up in the US of our breakthrough gene-based therapy for non-muscle invasive bladder cancer, Adstiladrin ® (nadofaragene firadenovec-vncg). Operating expenses were contained to an increase of €61 million year-on-year (i.e. +5% at AER and +7% at CER), and this includes significant non-recurring items (notably impairment charges and restructuring provisions). Underlying operating expenses remained well-controlled, with increased investments targeted to support the growth of Adstiladrin and other opportunities. Thus, operating profit for the year reached €167 million, a decrease of -€24 million (‑13%) versus the prior year at AER, while remaining flat at CER - with the difference being driven by the weaker US dollar. Following a focus on improving cash conversion after several heavy investments, free cash flow generation approached neutral despite currency headwinds from the weaker US dollar. This represents a substantial improvement compared to the previous year, and a significant step towards sustainable cash generation. Jean-Frédéric Paulsen, Chairman of the Board of Directors and Chief Executive Officer, said: “This was a pivotal year as we continued evolving our business to become stronger, more agile and more resilient, and importantly we got back to free cash flow neutral. Moreover, in 2025, we introduced an enterprise model designed to create greater value for patients and customers while supporting sustainable growth. This reflects our long-term commitment to serving patients’ need, and fostering an environment where people can learn, grow and perform at their best.” Ferring has always conducted business responsibly by seeking to protect the environment, create value for society, and uphold our high standards of ethics and governance. In 2025, we passed a major milestone when our targets for reducing greenhouse gas (GHG) emissions were approved by the Science Based Targets initiative (SBTi). This globally recognised standard ensures corporate goals are aligned with international climate policy. During the year, we succeeded in reducing our Scope 1 and 2 GHG emissions by 4.3% and Scope 3 emissions by 19%. Access to affordable healthcare is embedded in Ferring’s purpose and strategic priorities. The Project Family™: Safe Birth initiative aims to reduce maternal deaths in low- and lower middle-income countries by enabling wider access to Carbetocin Ferring (carbetocin, room-temperature stable formulation). In 2025, we worked with partners to supply around 1.7 million doses of this life-saving medicine at an affordable access price, while gaining further approvals in seven low- and lower middle-income countries. About Ferring Pharmaceuticals Ferring Pharmaceuticals is a privately owned specialty biopharmaceutical group committed to building families and helping people live better lives. We are leaders in reproductive medicine with a strong heritage in gastroenterology and urology and are at the forefront of innovation in uro-oncology gene therapy. Ferring was founded in 1950 and employs more than 7,500 people worldwide. The company is headquartered in Saint-Prex, Switzerland, and has operating subsidiaries in more than 50 countries which market its medicines in over 100 countries. Learn more at , or connect with us on LinkedIn , Instagram and YouTube . Contacts For more information, please contact Carine Julen Senior Manager, Corporate Communications & Public Affairs carine.julen@ferring.com

Gene TherapyDrug ApprovalFinancial Statement

10 Mar 2026

·www.ferring.com

For the first time, Ferring reports revenue of over €2.5 billion in 2025

Saint-Prex, Switzerland – 10 March 2026 – Ferring today published its 2025 Annual Report and Sustainability Report. The company achieved total revenues of over €2.5 billion in 2025, an increase of 7% over the previous year at actual exchange rates (AER) and 10% at constant exchange rates (CER). These were mainly driven by our flagship product Menopur® (menotropins for injection) in Reproductive Medicine, and the ramp-up in the US of our breakthrough gene therapy for non-muscle invasive bladder cancer Adstiladrin® (nadofaragene firadenovec-vncg). Operating expenses were contained to an increase of €61 million year-on-year (i.e. +5% at AER and +7% at CER), and this includes significant non-recurring items (notably impairment charges and restructuring provisions). Underlying operating expenses remained well-controlled, with increased investments targeted to support the growth of Adstiladrin and other opportunities. Thus operating profit for the year reached €167 million, a decrease of -€24 million (‑13%) versus the prior year at AER, while remaining flat at CER – with the difference being driven by the weaker US dollar. Following a focus on improving cash conversion after several heavy investments, free cash flow generation approached neutral despite currency headwinds from the weaker US dollar. This represents a substantial improvement compared to the previous year, and a significant step towards sustainable cash generation. Jean-Frédéric Paulsen, Chairman of the Board of Directors and Chief Executive Officer, said: “This was a pivotal year as we continued evolving our business to become stronger, more agile and more resilient, and importantly we got back to free cash flow neutral. Moreover, in 2025, we introduced an enterprise model designed to create greater value for patients and customers while supporting sustainable growth. This reflects on our long-term commitment to serving patients’ need, and fostering an environment where people can learn, grow and perform at their best.” Ferring has always conducted business responsibly by seeking to protect the environment, create value for society, and uphold our high standards of ethics and governance. In 2025, we passed a major milestone when our targets for reducing greenhouse gas (GHG) emissions were approved by the Science Based Targets initiative (SBTi). This globally recognised standard ensures corporate goals are aligned with international climate policy. During the year, we succeeded in reducing our Scope 1 and 2 GHG emissions by 4.3% and Scope 3 emissions by 19%. Access to affordable healthcare is embedded in Ferring’s purpose and strategic priorities. The Project Family™: Safe Birth initiative aims to reduce maternal deaths in low- and lower middle-income countries by enabling wider access to Carbetocin Ferring (carbetocin, room-temperature stable formulation). In 2025, we worked with partners to supply around 1.7 million doses of this life-saving medicine at an affordable access price, while gaining further approvals in seven low- and lower middle-income countries. Ferring Pharmaceuticals is a privately owned specialty biopharmaceutical group committed to building families and helping people live better lives. We are leaders in reproductive medicine with a strong heritage in gastroenterology and urology and are at the forefront of innovation in uro-oncology gene therapy. Ferring was founded in 1950 and employs more than 7,500 people worldwide. The company is headquartered in Saint-Prex, Switzerland, and has operating subsidiaries in more than 50 countries which market its medicines in over 100 countries. Learn more at www.ferring.com, or connect with us on LinkedIn, Instagram and YouTube. Carine Julen Senior Manager, Corporate Communications & Public Affairs carine.julen@ferring.com

Gene TherapyFinancial Statement

27 Feb 2026

·allsci.com

Pfizer withdraws EMA filing for sc PD-1 Zumrad in high-risk bladder cancer

Pfizer Europe MA EEIG withdrew its EU marketing authorization application for Zumrad (sasanlimab), a subcutaneous PD-1–blocking monoclonal antibody being developed with bacillus Calmette-Guérin (BCG) for BCG-naïve, high-risk non-muscle invasive bladder cancer (NMIBC), according to an EMA questions and answers document. The EMA said Pfizer withdrew the application on February 13, 2026, after the agency had evaluated the initial dossier and was assessing the company’s responses to CHMP questions. In its provisional view at the time of withdrawal, the EMA said Zumrad could not have been authorized based on the available data. The agency cited concerns that major changes were made while the pivotal study was ongoing, and that the statistical method used to determine efficacy was also changed, which the EMA said increased the chance of concluding benefit. The EMA also questioned whether the primary effectiveness measure—assessed by investigators in a setting where “researchers and participants knew which treatment was being given”—was appropriate, and said it was unclear whether the effect size represented a meaningful patient benefit. The agency added that outcomes such as overall survival did not support the primary endpoint finding, and that adding sasanlimab to BCG resulted in more side effects, including immune-related events, plus more BCG interruptions and discontinuations. Pfizer told the EMA it withdrew the application to allow for additional data collection and analyses to address the agency’s questions and concerns, and said there were no consequences for patients in ongoing clinical trials using Zumrad. The application was supported by a main study in 1,055 adults with high-risk NMIBC who were BCG-naïve or had not received BCG within the prior two years. In the study, Zumrad plus BCG was compared with BCG alone. Sasanlimab was administered once every four weeks for up to two years, while BCG was given weekly for six weeks followed by maintenance across five cycles, the EMA said. The primary measure was time free of events including recurrence or worsening, presence of cancer cells in the bladder lining, or death from any cause; overall survival was also evaluated. Pfizer has previously publicly disclosed positive Phase III results for sasanlimab plus BCG in high-risk NMIBC (the CREST program), including publication of CREST data in Nature Medicine, but the EMA document indicates the agency’s review focused on whether the evidence package was sufficient for authorization as filed. From a treatment-landscape perspective, NMIBC drug development has accelerated in BCG-unresponsive disease, where the US FDA has approved multiple non-surgical options, including pembrolizumab (Keytruda) for BCG-unresponsive high-risk NMIBC with carcinoma in situ (CIS) (2020), nadofaragene firadenovec (Adstiladrin) gene therapy (2022), and nogapendekin alfa inbakicept-pmln (Anktiva) with BCG (2024). Mechanistically, Zumrad targets the PD-1 checkpoint on T cells, aiming to prevent tumor-driven immune suppression via PD-L1/PD-L2 binding and thereby enhance anti-tumor immune activity—an approach that has reshaped systemic oncology but has faced design and endpoint challenges in earlier-stage, bladder-sparing NMIBC trials where recurrence-based measures, investigator assessment, and intravesical therapy schedules can meaningfully influence interpretability.

Phase 3Clinical ResultDrug ApprovalGene Therapy

100 Deals associated with Nadofaragene firadenovec-vncg

R&D Status

Approved

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Indication	Country/Location	Organization	Date
Non-Muscle Invasive Bladder Neoplasms	United States	Ferring Pharmaceuticals A/S	16 Dec 2022

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Malignant Pleural Mesothelioma	Phase 3	United States	-	21 Jan 2019
Malignant Pleural Mesothelioma	Phase 3	Australia	-	21 Jan 2019
Malignant Pleural Mesothelioma	Phase 3	Canada	-	21 Jan 2019
Malignant Pleural Mesothelioma	Phase 3	France	-	21 Jan 2019
Malignant Pleural Mesothelioma	Phase 3	Germany	-	21 Jan 2019
Malignant Pleural Mesothelioma	Phase 3	Italy	-	21 Jan 2019
Malignant Pleural Mesothelioma	Phase 3	Poland	-	21 Jan 2019
Malignant Pleural Mesothelioma	Phase 3	Russia	-	21 Jan 2019
Malignant Pleural Mesothelioma	Phase 3	United Kingdom	-	21 Jan 2019
Muscle Invasive Bladder Carcinoma	Phase 2	United States	Ferring Pharmaceuticals SA	01 Jun 2026

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT06668493 (LUNAR, ASCO_GU2026)	Phase 1/2	Renal Pelvis and Ureter Urothelial Carcinoma	20	Nadofaragene firadenovec	dsutubtcoh(gvdltaqxyv) = fsqagvaqig ouskrcaile (keueevkfzn )	Positive	26 Feb 2026
BusinessWire Manual	Not Applicable	Non-Muscle Invasive Bladder Neoplasms	13	Nadofaragene firadenovec	vmspijdiaz(fgoajhwacn) = irkoocdxet bjltmpafov (mkptayvdeh )	Positive	16 Oct 2025
BusinessWire Manual	Not Applicable	Non-Muscle Invasive Bladder Neoplasms	13	Nadofaragene firadenovec (CIS± papillary tumors)	vmspijdiaz(fgoajhwacn) = pmomvujkna bjltmpafov (mkptayvdeh )	Positive	16 Oct 2025
NCT06026332 (ABLE-41, ASCO2025)	Phase 4	Bladder Cancer CIS \| papillary tumors	54	Nadofaragene firadenovec-vncg	rypuyuponj(lkvcevlaob) = udmovgrvzx szprjmhldg (ekgqfqgplb ) View more	Positive	30 May 2025
NCT06510374 (ABLE-32, ASCO2025)	Phase 3	Non-Muscle Invasive Bladder Neoplasms Maintenance	454	Nadofaragene firadenovec-vncg	xbakxzwkqh(bauhvzzrtt) = no grade 4/5 study drug-related AEs bhipggfhxn (kebnstmtws )	Positive	30 May 2025
NCT03710876 (INFINITE, CTgov)	Phase 3	Malignant Pleural Mesothelioma	53	rAd-IFN+Gemcitabine+Celecoxib Oral Product (Treatment Group)	oyxfypneac(xihrgdtsgv) = qdznmvjsxg daemnmrqnd (bchghndgpg, geyftyibtj - pbqpvosrfy) View more	-	20 May 2025
NCT03710876 (INFINITE, CTgov)	Phase 3	Malignant Pleural Mesothelioma	53	Gemcitabine+Celecoxib Oral Product (Control Group)	oyxfypneac(xihrgdtsgv) = twznsjmewu daemnmrqnd (bchghndgpg, jmbbqmtkgc - fualvllmus) View more	-	20 May 2025
NCT05704244 (BusinessWire) Manual	Phase 3	Non-Muscle Invasive Bladder Neoplasms	20	Nadofaragene firadenovec	cofhkxssfp(yqynwmpjkm) = isxswcxgub puwfotgbmh (lixbmakhpe )	Positive	21 Apr 2025
NCT06545955 (ABLE-22, ASCO_GU2025)	Phase 2	Non-Muscle Invasive Bladder Neoplasms First line	150	Nadofaragene firadenovec	zgskkaogpb(tsjzrfwacr) = lmyefinrsv jkauuirrou (isneotqwhv )	Positive	13 Feb 2025
NCT02773849 (PD48-01, AUA2024)	Phase 3	Non-Muscle Invasive Bladder Neoplasms BCG-unresponsive \| CIS \| Ta/T1	-	Nadofaragene firadenovec	vldtqpwcvg(csrwcqcmzk) = jrtpjqonny tmymyckkkj (mxsammitfy ) View more	Positive	01 May 2024
NCT02773849 (PD48-01, AUA2024)	Phase 3		-	Placebo	vldtqpwcvg(csrwcqcmzk) = vqtzemaulj tmymyckkkj (mxsammitfy ) View more	Positive	01 May 2024
MP16-07 (AUA2024)	Phase 2	Non-Muscle Invasive Bladder Neoplasms TP53 \| TERT \| PIK3CA ... View more	43	Nadofaragene firadenovec	nifjscsmoz(nlltpdsidg) = lgfnhosuxz olxtstrdyi (uflzrevocf )	Positive	01 May 2024
NCT06026332 (ABLE-41, ASCO_GU2024)	Phase 3	-	800	Nadofaragene firadenovec-vncg	dberwqawlo(cnkitdmemz) = gjquhnpoux ktbddexcbp (dfmuewktno ) View more	-	25 Jan 2024

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