On October 2, 2025 UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company focused on developing and commercializing innovative solutions for urothelial and specialty cancers, reported the publication of a comprehensive review of the clinical development program for ZUSDURI (mitomycin) for intravesical solution, formerly known as UGN-102, the first and only FDA-approved medicine for adults with recurrent, low-grade, intermediate-risk non–muscle-invasive bladder cancer (LG-IR-NMIBC) (Press release, UroGen Pharma, OCT 2, 2025, View Source [SID1234656404]). The article titled: "Review of UGN-102: A Reverse Thermal Gel Containing Mitomycin for the Treatment of Recurrent, Low-Grade, Intermediate-Risk Non-Muscle Invasive Bladder Cancer" is published in the peer-reviewed journal Reviews in Urology, the official journal of LUGPA.

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"ZUSDURI is an FDA-approved, non-surgical treatment that has consistently demonstrated robust, clinically significant, and durable complete responses in patients with recurrent LG-IR-NMIBC," said publication author and ENVISION principal investigator Sandip Prasad, M.D., M.Phil., Director of Genitourinary Surgical Oncology and Vice Chair of Urology at Morristown Medical Center/Atlantic Health System, NJ. "With an acceptable safety profile manageable in routine urologic practice, ZUSDURI is administered in an outpatient setting without the need for general anesthesia. This review article highlights the clinical evidence supporting ZUSDURI’s role as an innovative option for patients with recurrent LG-IR-NMIBC."

Key Highlights from the Review:

High Complete Response Rates: Across late-stage clinical trials (OPTIMA II, ATLAS, and ENVISION), UGN-102 demonstrated complete response (CR) rates between 64.8% and 79.6% at three months.
Durable Disease Response: In the ENVISION trial, 80.6% of patients remained disease-free at 18 months following CR. Among the 41 patients who achieved CR in the OPTIMA II study, the median duration of response (DOR) was 24.2 months; among the 17 patients in the long-term follow-up study, the median DOR was 42.1 months. The median follow-up for the 17 patients in the long-term follow-up study was 50.4 months.
Manageable Safety Profile: Adverse events were primarily localized to the lower urinary tract. The most common (≥10%) adverse reactions (ARs) with ZUSDURI, including laboratory abnormalities, that occurred in patients were dysuria, increased potassium, increased creatinine, decreased hemoglobin, increased eosinophils, increased aspartate aminotransferase, increased alanine aminotransferase, decreased lymphocytes, urinary tract infection, decreased neutrophils, and hematuria. ARs were mainly mild to moderate. Serious ARs occurred in 12% of patients, including urinary retention (0.8%) and urethral stenosis (0.4%).
No Adverse Impact from Patient-Reported Outcomes: Clinical trials show that ZUSDURI did not adversely affect functionality, symptom burden, and quality of life in patients with LG-IR-NMIBC.
"This comprehensive review clearly explains the body of evidence supporting ZUSDURI as an innovative treatment option for patients with recurrent LG-IR-NMIBC," said Mark Schoenberg, M.D., Chief Medical Officer, UroGen. "By offering a non-surgical and durable solution, ZUSDURI represents a major advance in the way we care for these patients."

The clinical development program for ZUSDURI includes three late-phase clinical trials that investigated the safety and efficacy of ZUSDURI in patients with LG-IR-NMIBC: OPTIMA II, ATLAS, and ENVISION.

The newly diagnosed and recurrent patients with LG-IR-NMIBC included in the ZUSDURI clinical development program tended to be elderly (median ages 67-70 years across the studies). They also had a relatively high disease burden, with 60.3-82.8% having multiple tumors, 18.6-49.3% having aggregate tumor size greater than 3 cm, and 24.6-51.7% having LG recurrence within the year prior to their current diagnosis.

About ZUSDURI

ZUSDURI (mitomycin) for intravesical solution is an innovative drug formulation of mitomycin approved for the treatment of adults with recurrent LG-IR-NMIBC. Utilizing UroGen’s proprietary RTGel technology (a sustained release, hydrogel-based formulation), ZUSDURI is delivered directly into the bladder by a trained healthcare professional using a urinary catheter in an outpatient setting, thereby enabling the treatment of tumors by non-surgical means.

About Non-Muscle Invasive Bladder Cancer (NMIBC)
LG-IR-NMIBC affects around 82,000 people in the U.S. every year and of those, an estimated 59,000 are recurrent. Bladder cancer primarily affects older populations with increased risk of comorbidities, with the median age of diagnosis being 73 years. Guideline recommendations for the management of NMIBC include transurethral resection of bladder tumor (TURBT) as the standard of care. Up to 70 percent of NMIBC patients experience at least one recurrence, and LG-IR-NMIBC patients are even more likely to recur and face repeated TURBT procedures. Learn more about non-muscle invasive bladder cancer at www.BladderCancerAnswers.com.

About ENVISION
The Phase 3 ENVISION trial is a single-arm, multinational, multicenter pivotal study evaluating the efficacy and safety of ZUSDURI (mitomycin) for intravesical solution as a chemoablative therapy in adult patients with recurrent LG-IR-NMIBC. The Phase 3 ENVISION trial completed target enrollment with 240 patients across 56 sites. Study participants received six once-weekly intravesical instillations of ZUSDURI. The primary endpoint evaluated the CR rate at three months after the first instillation, and the key secondary endpoint evaluates durability over time in patients who achieved a CR at the three-month assessment. Learn more about the Phase 3 ENVISION trial at www.clinicaltrials.gov (NCT05243550).

About the Phase 2b OPTIMA II Trial

OPTIMA II (OPTimized Instillation of Mitomycin for Bladder Cancer Treatment) was an open-label, single-arm, multi-center Phase 2b clinical trial of ZUSDURI evaluating the safety and efficacy in patients with LG-IR-NMIBC. The trial enrolled 63 patients across 20 sites in the US and Israel. Learn more about the OPTIMA II trial at www.clinicaltrials.gov (NCT03558503).

About ATLAS

ATLAS was a global, open-label, randomized controlled Phase 3 trial designed to assess the efficacy and safety of UGN-102, with or without TURBT, vs. TURBT alone in patients diagnosed with LG-IR-NMIBC. The trial enrolled 282 patients in clinical sites in the U.S., Europe and Israel. UroGen halted patient enrollment in the ATLAS study in November 2021 to shift to an alternative development strategy, primarily focusing on the pivotal ENVISION trial for ZUSDURI. Learn more about the ATLAS trial at www.clinicaltrials.gov (NCT04688931).

On October 2, 2025 Phost’in Therapeutics, a clinical-stage biotechnology company focused on the development of N-glycosylation inhibitors and the Fondazione Gianni Bonadonna, which provides scientific contribution and independent support, reported that it will jointly present a poster on interim clinical data from the PhAST trial at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Annual Congress 2025, held October 17–21 in Berlin, Germany (Press release, Phost’in, OCT 2, 2025, View Source [SID1234656403]).

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On October 2, 2025 Kazia Therapeutics Limited (NASDAQ: KZIA), an oncology-focused drug development company, reported a substantial reduction in tumor burden from a single-patient expanded access case in triple-negative breast cancer (TNBC) treated with a combination regimen that included the Company’s investigational pan-PI3K/mTOR inhibitor, paxalisib (Press release, Kazia Therapeutics, OCT 2, 2025, View Source [SID1234656402]). After three weeks of combination immunotherapy/chemotherapy plus paxalisib, imaging performed at the treating institution showed an 86% reduction in overall tumor burden.

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The patient is a 40+ year old female initially diagnosed with TNBC in April 2023. She received neoadjuvant chemotherapy and immunotherapy, followed by bilateral mastectomy six weeks post-treatment with no residual cancer detected, followed by radiation therapy. Approximately two years later, the patient was diagnosed with metastatic disease to bone and lungs. She was then treated under a single-patient expanded-access protocol with an immunotherapy/chemotherapy regimen plus paxalisib. After three weeks of therapy, imaging demonstrated an 86% overall tumor reduction. The patient’s profile and treatment approach are highly similar to the inclusion and combination strategy being evaluated in Kazia’s Phase 1b TNBC study, which is assessing paxalisib in combination with Keytruda (pembrolizumab) and chemotherapy.

"Although this is a single-patient expanded-access case, the speed and magnitude of tumor regression are highly encouraging and align with our scientific rationale for combining paxalisib with immune checkpoint blockade," said Dr. John Friend, M.D., Chief Executive Officer of Kazia Therapeutics. "This experience reinforces our commitment to our ongoing, company sponsored Phase 1b trial in advanced breast cancer, and it echoes the recent ex-vivo findings showing disruption of circulating tumor cell clusters with paxalisib."

About the ongoing Phase 1b TNBC trial

Kazia is conducting a company-sponsored multi-centered Phase 1b study in Australia evaluating paxalisib in combination with Keytruda and chemotherapy for advanced breast cancer, including TNBC. The study is designed to assess safety, preliminary anti-tumor activity, and translational biomarkers, including effects on circulating tumor cells and cluster dynamics to better understand how paxalisib may enhance immunotherapy responsiveness in this population.

On October 2, 2025 Greenwich LifeSciences, Inc. (Nasdaq: GLSI) (the "Company"), a clinical-stage biopharmaceutical company focused on its Phase III clinical trial, FLAMINGO-01, which is evaluating GLSI-100, an immunotherapy to prevent breast cancer recurrences, reported the expansion of FLAMINGO-01 clinical trial to Belgium (Press release, Greenwich LifeSciences, OCT 2, 2025, View Source [SID1234656401]).

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The Company’s application to European regulators has been formally approved, adding Belgium as an approved country in FLAMINGO-01 in addition to Spain, France, Germany, Italy, Poland, Romania, Ireland, Portugal, and the US.

According to the latest data collected by the European Cancer Information System (click here), a total of 11,366 new cases of breast cancer were diagnosed in Belgium in 2022, which is the most common cancer diagnosed in women, representing approximately 33% of all cancers in women. Breast cancer is the leading cause of death from cancer in women in Belgium with 2,324 deaths in 2022.

The Company is collaborating with Patrick Neven, MD, PhD, at UZ Leuven, who will be serving as the national principal investigator in Belgium for FLAMINGO-01. Dr Neven is Full Professor at the Department of Gynecological Oncology, University Hospitals Leuven, and is a staff member of the Multidisciplinary Breast Centre. His research focuses on breast oncology, particularly endocrine therapy and quality of life. He has served as principal investigator in multiple clinical trials, published over 300 peer-reviewed papers, and lectured widely at international meetings. He is president of the Belgian Society of Senology, active in several scientific societies, and has mentored numerous doctors and PhD students in breast cancer care.

CEO Snehal Patel commented, "We thank our steering committee for introducing us to Dr. Neven and look forward to working with him and his colleagues. We are planning start-up activities in Leuven in the coming month. Leuven is centrally located in Belgium and is an ideal location to cover large parts of the country, including Brussels and Antwerp."

About FLAMINGO-01 and GLSI-100

FLAMINGO-01 (NCT05232916) is a Phase III clinical trial designed to evaluate the safety and efficacy of GLSI-100 (GP2 + GM-CSF) in HER2 positive breast cancer patients who had residual disease or high-risk pathologic complete response at surgery and who have completed both neoadjuvant and postoperative adjuvant trastuzumab based treatment. The trial is led by Baylor College of Medicine and currently includes US and European clinical sites from university-based hospitals and academic and cooperative networks with plans to open up to 150 sites globally. In the double-blinded arms of the Phase III trial, approximately 500 HLA-A*02 patients will be randomized to GLSI-100 or placebo, and up to 250 patients of other HLA types will be treated with GLSI-100 in a third arm. The trial has been designed to detect a hazard ratio of 0.3 in invasive breast cancer-free survival, where 28 events will be required. An interim analysis for superiority and futility will be conducted when at least half of those events, 14, have occurred. This sample size provides 80% power if the annual rate of events in placebo-treated subjects is 2.4% or greater.

For more information on FLAMINGO-01, please visit the Company’s website here and clinicaltrials.gov here. Contact information and an interactive map of the majority of participating clinical sites can be viewed under the "Contacts and Locations" section. Please note that the interactive map is not viewable on mobile screens. Related questions and participation interest can be emailed to: [email protected]

About Breast Cancer and HER2/neu Positivity

One in eight U.S. women will develop invasive breast cancer over her lifetime, with approximately 300,000 new breast cancer patients and 4 million breast cancer survivors. HER2 (human epidermal growth factor receptor 2) protein is a cell surface receptor protein that is expressed in a variety of common cancers, including in 75% of breast cancers at low (1+), intermediate (2+), and high (3+ or over-expressor) levels.

On October 2, 2025 Clarity Pharmaceuticals (ASX: CU6) ("Clarity" or "Company"), a clinical-stage radiopharmaceutical company with a mission to develop next-generation products that improve treatment outcomes for patients with cancer, reported that data on Clarity’s pan-cancer theranostic, 64/67Cu-SAR-bisFAP, will be presented at the upcoming World Molecular Imaging Conference (WMIC) 2025 from the 29th September to October 3rd in Anchorage, Alaska by Dr. Michele De Franco, a research fellow at the Memorial Sloan Kettering Cancer Center (MSK) and Clarity’s collaborator (Press release, Clarity Pharmaceuticals, OCT 2, 2025, View Source [SID1234656399]).

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Clarity is developing 64/67Cu-SAR-bisFAP as potential pan-cancer theranostics targeting fibroblast activation protein (FAP), which is expressed on cancer associated fibroblasts (CAFs), a particular cell type found in the tumour microenvironment (cancer ‘infrastructure’ called the tumour stroma). FAP is found to be highly expressed in a broad range of cancers (e.g. breast, colorectal, pancreatic, lung, brain and ovarian cancers), but only minimally in normal tissue, making it a promising pan-cancer target for both imaging and treatment of cancers1. CAFs form part of the environment surrounding the cancer cells, and they can promote cancer growth and the spread of the tumour throughout the body2. Targeting the tumour stroma is an alternative way to treat cancer whereby the architecture of the tumour mass is targeted rather than the tumour cells directly.

As part of the optimisation process, Clarity developed and assessed two versions of the FAP-targeted product, one with a singular targeting molecule, SAR-FAP, and a dimeric version of the same molecule, SAR-bisFAP. Whilst both molecules have shown high tumour-specific uptake and targeting, the dual-targeting SAR-bisFAP has shown superior tumour targeting and retention in FAP-expressing mouse models.

Comparison between the ex vivo biodistribution of 64Cu-SAR-FAP and 64Cu-SAR-bisFAP in FAP-positive U-87 MG (human glioblastoma) xenografts, showing how much product accumulated in each location, expressed as the percentage of the injected activity (%IA/g), at 1, 4, and 24 h post-injection.

Consistent with the enhanced tumour uptake observed using the dual-targeting 64Cu-SAR-bisFAP, 67Cu-SAR-bisFAP also showed improved efficacy in therapeutic mice studies, with a doubling in the median survival time of the mice who received 30 MBq of 67Cu-SAR-bisFAP compared to those who received 30 MBq of the 67Cu-SAR-FAP monomer or an industry benchmark, 177Lu-FAP-2286 (median survival time was 28.5, 14.5, and 11.5 days, respectively).

Based on this data and results from previously completed pre-clinical studies, Clarity is aiming to progress the dual-targeting SAR-bisFAP theranostic products into human clinical studies, with a focus on the diagnostic in the first instance.

Dr Alan Taylor, Executive Chairperson of Clarity Pharmaceuticals, commented: "FAP-targeted products represent an exciting new generation of radiopharmaceuticals with the potential to target a range of cancer indications with high unmet needs. Consistent with our approach to high quality research and science, we continue to explore and optimise the most promising products in the field and going the extra mile to maximise their viability and success in the clinic. Starting from the benchtop, we work with thought leaders in the field to overcome the shortcomings of the existing products in development and generate strong data to support this. As a result of this commitment to excellence, we are now excited for our colleagues at MSK to share the results of the SAR-bisFAP products at one of the leading conferences in the field as they pave the way for clinical research in the near future.

"The diagnostic products using copper-64 and the therapeutics using copper-67 have shown high tumour targeting and retention in FAP-expressing xenograft mice models of cancer, with the dual-targeting 64Cu-SAR-bisFAP showing improved retention compared to the monomer alone, and 67Cu-SAR-bisFAP displaying improved efficacy compared to both 67Cu-SAR-FAP and an industry benchmark, 177Lu-FAP-2286. We believe these benefits are enabled by the optimised "bis" structure of our FAP product as well as the advantages offered by the perfect pairing of copper-64 and copper-67, securely held by our proprietary SAR chelator.

"Based on this data, we are currently conducting product development to enable a Phase I clinical trial in 2026 with 64Cu-SAR-bisFAP, which will be followed by exploring potential treatment opportunities of cancers based on their unmet medical needs using 67Cu-SAR-bisFAP in subsequent clinical trials."