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 1, 2025 PhotonPharma , a cancer immunotherapy company developing a scalable autologous therapy platform, Innocell, reported that CEO and Chief Scientific Officer Raymond Goodrich, PhD, will present at the 2nd Annual Personalized Cancer Vaccine Summit taking place November 18-20, 2025, in Boston, Massachusetts (Press release, PhotonPharma, OCT 1, 2025, View Source [SID1234656385]).

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Dr. Goodrich will deliver two featured presentations showcasing PhotonPharma’s innovative approach to personalized cancer immunotherapies:

"Developing a Practical & Scalable Autologous Vaccine Manufacturing Platform Using Whole Tumor Cells to Enable Real-World Application of Personalized Immunotherapy" – This presentation will detail how PhotonPharma is adapting pathogen reduction technology to manufacture metabolically active, non-replicating tumor cells for autologous immunotherapy, significantly simplifying production while preserving immunogenic function.

"Activating the Immune System with Whole-Tumor Vaccines: Harnessing Shared & Neoantigens to Remodel the Tumor Microenvironment" – Dr. Goodrich will present preclinical data demonstrating how PhotonPharma’s whole tumor cell platform stimulates immune responses by combining shared tumor-associated antigens and patient-specific neoantigens in a single vaccine format.

"We’re excited to share our progress in developing a truly scalable manufacturing platform to potentially deliver personalized cancer immunotherapies" said Dr. Terry Opgenorth, Chairman of the Board. "Our approach addresses the key challenges facing the field – manufacturing speed, cost-effectiveness, and broad tumor coverage – while maintaining the personalized benefits that make these therapies so promising."

The presentations highlight PhotonPharma’s unique manufacturing workflow that streamlines the process from tumor resection to reinjection, reducing turnaround time and improving reproducibility. The technology platform’s initial indication targets patients with relapsed ovarian cancer.

About the Personalized Cancer Vaccine Summit

The 2nd Annual Personalized Cancer Vaccine Summit brings together 80+ senior executives from leading pharmaceutical and biotechnology companies to address critical challenges in personalized cancer vaccine development. The event features presentations from industry leaders including Moderna, Merck, BioNTech, and Genentech, alongside innovative companies developing next-generation platforms across mRNA, peptide, viral, and cellular modalities.

On October 1, 2025 Humanetics Corporation, an advanced clinical-stage specialty pharmaceutical company pioneering novel approaches to tissue protection in oncology-related radiation exposure and mitigating the inflammatory response in pulmonary disease, reported that the Company will be hosting a virtual panel on October 08, 2025, 1:30 pm EDT, entitled "Mitigating Tissue Damage in Radiation Oncology and Inflammation in Pulmonary Disease: Targeting Pathways of Tissue Injury and Inflammatory Response (Press release, Humanetics, OCT 1, 2025, View Source [SID1234656386])."

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Panel members include:

Rany Condos, MD, Director, Interstitial Lung Disease Program and Post-COVID Program, NYU Langone
Pranshu Mohindra, MD, MMM, Professor and Vice Chair of Operations and Quality for the Department of Radiation Oncology and Director of the University Hospitals Proton Therapy Center, University Hospitals Cleveland Medical Center
Colin G. Chinn, MD, MHS, FACP, RADM MC USN (Ret), Chief Medical Officer, Humanetics
Michael D. Kaytor, PhD, Vice President, Research & Development, Humanetics
Hannah Olson, PhD, Research & Development Scientist, Humanetics
This panel will explore advances in addressing unmet medical needs related to oncology and radiation exposure, as well as pulmonary disease and inflammation. Leading oncology and pulmonary medicine clinicians, together with researchers, will share perspectives on emerging data, trial design, clinical practice challenges, and insights into potential therapeutic targets.

A live Q&A session will follow the panel discussion.

Humanetics convened an expert panel on August 12, 2025, that explored its Medical Countermeasure development program. As discussed, protecting Warfighters from tissue damage due to accidental or intentional ionizing radiation exposure remains a priority for the United States as well as its allies and partners. Likewise, safeguarding emergency medical personnel, first responders, and civilian populations is among the domestic imperatives for Strategic National Stockpile decision makers. Access to the video recording of the August 12 session is available on the Humanetics Corporation website under "News & Events."

On October 1, 2025 Ensem Therapeutics, Inc. (ENSEM) reported the U.S. Food and Drug Administration (FDA) granted Fast Track designation to its clinical stage pan mutant-specific allosteric PI3Kα inhibitor and degrader, ETX-636, for the treatment of adult patients with PIK3CA-mutant, hormone receptor positive (HR+)/human epidermal growth factor negative (HER2-) advanced breast cancer (Press release, ENSEM Therapeutics, OCT 1, 2025, View Source [SID1234656387]).

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ETX-636 was designed using ENSEM’s unique Kinetic Ensemble platform to optimally fit into a specific allosteric binding site in p110α, the catalytic subunit of PI3Kα and can selectively inhibit multiple activating mutant forms of PI3Kα, while sparing wildtype PI3Kα. The selectivity of ETX-636 for mutant PI3Kα greatly reduces the risk for hyperglycemia and other wildtype PI3Kα-related adverse events compared to non-mutant selective PI3Kα inhibitors. In addition, ETX-636 also leads to proteasome-dependent degradation of mutant PI3Kα, while sparing wildtype protein (a feature not seen with other pan-mutant allosteric inhibitors).

Activating mutations or alterations of the PI3Kα pathway are known to promote cancer cell growth and survival in a variety of tumor types, including breast cancer. In HR+/HER2- breast cancer (approximately 70% of all breast cancers), PIK3CA mutations are particularly prevalent, occurring in up to 40% of the cases.

"Patients with advanced HR+/HER2- breast cancer harboring PIK3CA mutations have poor prognosis, and there is an unmet need for therapies targeting this population that are safer and more efficacious than the current FDA approved non-mutant selective treatments," said Dr. Shengfang Jin, CEO and Co-Founder of ENSEM. "We are appreciative that the FDA has recognized ETX-636 as a potentially important treatment for this indication and we remain laser-focused on demonstrating its benefit to patients in our current clinical trials."

ETX-636 is currently being studied in an initial first-in-human, Phase 1/2 study which is evaluating the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary antitumor activity of ETX-636 in participants with advanced solid tumors harboring a PIK3CA mutation. ETX-636 will be administered alone or in combination with fulvestrant, a selective estrogen receptor degrader approved for the treatment of advanced hormone receptor HR+/HER2- breast cancer NCT06993844.

Fast Track designation is a program provided by the FDA to expedite the development and review of drugs and biologics that treat serious conditions with unmet medical needs. It provides the opportunity for frequent and early communication and collaboration with the FDA, as well as the potential for accelerated approval and priority review eligibility to allow faster access to promising treatment to patients.

On October 1, 2025 NUCLIDIUM AG, a clinical-stage radiopharmaceutical company developing a proprietary copper-based theranostic platform, reported that it has received a Notice of Allowance from the U.S. Patent and Trademark Office (USPTO) for a patent covering the company’s KaliosTM-program (Press release, NUCLIDIUM, OCT 1, 2025, https://nuclidium.com/nuclidium-receives-notice-of-allowance-for-composition-of-matter-patent-covering-fap-inhibitor-based-radiodiagnostics-and-therapeutics-in-the-united-states/ [SID1234656372]). Once issued, the composition-of-matter patent will protect a variety of small molecule inhibitors of the fibroblast activation protein (FAP) that can be used in combination with a range of radionuclides bound with a variety of chelating moieties for the diagnosis and treatment of diseases, including solid tumors. The KaliosTM-program consists of a tumor-targeting FAP inhibitor labelled with a pre-selected copper radioisotope: Copper-61 for diagnostics and Copper-67 for therapeutics. The program is currently in development for the diagnosis and treatment of certain breast and lung cancer indications.

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"The Notice of Allowance from the U.S. Patent and Trademark Office is an important milestone for NUCLIDIUM as we continue to advance our best-in-class copper-based radiotheranostics. This initial action from the U.S. Patent office strengthens our intellectual property position in a highly competitive field. It also recognizes the innovation inherent in our approach and supports our long-term goal of addressing unmet needs in oncology, especially in women’s health, through innovative copper-based radiopharmaceuticals. We are committed to building a robust patent portfolio that protects our discoveries and to advancing our copper-based theranostics into clinical evaluation," said Leila Jaafar, PhD, CEO and Co-Founder of NUCLIDIUM.

The expected patent covers a diverse range of inhibitors that target FAP, representing broad protection for Nuclidium’s current and future radiopharmaceuticals and theranostics in its KaliosTM-program. FAP is a membrane-bound serine protease (dipeptidyl peptidase) present in epithelial tumors that make up 80-90% of all cancer cases. It is highly expressed on cancer-associated fibroblasts in the tumor stroma as well as in degenerative tissues or inflamed tissues such as bursitis and fibrosis. These characteristics render FAP an attractive target for cancer diagnosis and treatment. Currently approved FAP-targeted radioligands, labelled with either 68Ga or 18F, have a short half-life, which restricts their ability for delayed imaging to identify and treat smaller metastases and, in addition, limits their possible distribution range after production.

Nuclidium and its collaborators recently published a preclinical study in the European Journal of Nuclear Medicine and Molecular Imaging – Radiopharmacy and Chemistry, highlighting the unique potential of copper-based FAP-targeting PET radiotracers. In the study, [61Cu]Cu-Kalios PET radioligands showed favorable pharmacokinetics, high tumor uptake, and strong tumor-to-background ratios with delayed 4h imaging when compared to established FAP-targeting radiotracers. These findings underscore the clinical potential of 61-copper-based radiopharmaceuticals to improve the temporal imaging range for precise detection of FAP-expressing tumors.