PRINCETON, N.J.--(BUSINESS WIRE)--UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, today announced U.S. Food and Drug Administration (FDA) acceptance of the New Drug Application (NDA) for investigational drug UGN-102 (mitomycin) for intravesical solution.

新泽西州普林斯顿市（商业新闻短讯）——UroGen Pharma Ltd.（纳斯达克：URGN），一家致力于开发和商业化治疗尿路上皮癌和特殊癌症的创新解决方案的生物技术公司，今天宣布美国食品和药物管理局（FDA）接受用于膀胱内溶液的研究药物UGN-102（丝裂霉素）的新药申请（NDA）。

UGN-102 could become the first FDA-approved medicine for the treatment of low-grade intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). The FDA has assigned a Prescription Drug User Fee Act (PDUFA) goal date of June 13, 2025..

UGN-102可能成为FDA批准的第一种用于治疗低度中度风险非肌层浸润性膀胱癌（LG-IR-NMIBC）的药物。美国食品和药物管理局（FDA）已将处方药使用费法案（PDUFA）的目标日期指定为2025年6月13日。。

'The FDA acceptance of our NDA is a pivotal moment in our journey to bring UGN-102 to patients,” said Liz Barrett, President and Chief Executive Officer of UroGen. “UGN-102 could be the first FDA-approved medicine for LG-IR-NMIBC, offering a novel approach that could expand treatment options and address unmet needs.

UroGen总裁兼首席执行官利兹·巴雷特（LizBarrett）说：“FDA接受我们的NDA是我们将UGN-102带给患者的旅程中的关键时刻。”。“UGN-102可能是FDA批准的第一种用于LG-IR-NMIBC的药物，提供了一种新的方法，可以扩大治疗选择并解决未满足的需求。

There is an urgent need for innovative solutions in this space, and we are dedicated to collaborating with the FDA as we prepare for a potential launch of UGN-102 in 2025.”.

在这个领域迫切需要创新的解决方案，我们致力于与FDA合作，为2025年可能推出的UGN-102做好准备。”。

Dr. Mark Schoenberg, Chief Medical Officer of UroGen, stated, “The NDA for UGN-102 is backed by a robust data set demonstrating impressive durability of response across three clinical trials and a favorable safety profile. Notably, the ENVISION trial successfully met its primary endpoint, showing a 79.6% complete response rate at three months after the first instillation of UGN-102.

UroGen首席医疗官Mark Schoenberg博士表示：“UGN-102的NDA得到了强大数据集的支持，该数据集显示了三项临床试验中令人印象深刻的反应持久性和良好的安全性。值得注意的是，ENVISION试验成功达到了其主要终点，首次滴注UGN-102后三个月的完全缓解率为79.6%。

Additionally, the latest results from that trial revealed an 82.3% 12-month duration of response by Kaplan-Meier estimate in patients who achieved a complete response at 3 months. The most common treatment-emergent adverse events in the ENVISION trial were dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention.

此外，该试验的最新结果显示，Kaplan-Meier估计，在3个月时达到完全缓解的患者中，12个月的反应持续时间为82.3%。ENVISION试验中最常见的治疗紧急不良事件是排尿困难，血尿，尿路感染，pollakiuria，疲劳和尿潴留。

Additionally, the safety profile observed in the ENVISION trial was consistent with that seen in other studies of UGN-102. We believe that, if approved, UGN-102’s ability to achieve durable complete responses and potentially reduce recurrence rates while extending treatment-free intervals will represent a significant advance in managing LG-IR-NMIBC.”.

此外，ENVISION试验中观察到的安全性与UGN-102其他研究中观察到的一致。我们相信，如果获得批准，UGN-102能够实现持久的完全缓解，并可能降低复发率，同时延长无治疗间隔时间，这将是管理LG-IR-NMIBC的重大进步。”。

About UGN-102

关于UGN-102

UGN-102 (mitomycin) for intravesical solution is an innovative drug formulation of mitomycin, currently under regulatory review for approval in the treatment of LG-IR-NMIBC. Utilizing UroGen’s proprietary RTGel® technology, a sustained release, hydrogel-based formulation, UGN-102 is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means.

用于膀胱内溶液的UGN-102（丝裂霉素）是丝裂霉素的创新药物制剂，目前正在接受监管审查，以批准用于治疗LG-IR-NMIBC。UGN-102利用UroGen专有的RTGel®技术，一种基于水凝胶的缓释制剂，旨在使膀胱组织更长时间暴露于丝裂霉素，从而能够通过非手术手段治疗肿瘤。

UGN-102 is delivered to patients using a standard urinary catheter in an outpatient setting by a trained healthcare professional. An NDA for UGN-102 is currently under review by the FDA with a potential decision expected by June 13, 2025. The U.S. market for LG-IR-NMIBC that UGN-102 can address, if approved, is valued at approximately $5 billion..

UGN-102由训练有素的医疗保健专业人员在门诊使用标准导尿管输送给患者。FDA目前正在审查UGN-102的NDA，预计将于2025年6月13日做出决定。如果UGN-102获得批准，LG-IR-NMIBC在美国的市场价值约为50亿美元。。

About ENVISION

关于ENVISION

The Phase 3 ENVISION trial is a single-arm, multinational, multicenter study evaluating the efficacy and safety of UGN-102 (mitomycin) for intravesical solution as primary chemoablative therapy in patients with LG-IR-NMIBC. The Phase 3 ENVISION trial completed target enrollment with approximately 240 patients across 56 sites.

ENVISION 3期试验是一项单臂，多国，多中心研究，评估UGN-102（丝裂霉素）作为LG-IR-NMIBC患者主要化学消融治疗膀胱内溶液的有效性和安全性。第三阶段ENVISION试验完成了56个地点约240名患者的目标登记。

Study participants received six once-weekly intravesical instillations of UGN-102. The primary endpoint evaluated the CR rate at the three-month assessment after the first instillation, and the key secondary endpoint evaluated durability over time in patients who achieved a CR at the three-month assessment.

研究参与者每周接受六次膀胱内滴注UGN-102。主要终点在第一次滴注后的三个月评估中评估CR率，关键次要终点评估在三个月评估中达到CR的患者随时间的耐久性。

About Non-Muscle Invasive Bladder Cancer (NMIBC)

关于非肌层浸润性膀胱癌（NMIBC）

In the U.S. bladder cancer is the second most common urologic cancer in men. LG-IR-NMIBC represents approximately 22,000 newly diagnosed bladder cancer patients each year and an estimated 60,000 recurrences annually among patients diagnosed from previous years. Bladder cancer primarily affects older populations with the median age of diagnosis 73 years and an increased risk of comorbidities.

在美国，膀胱癌是男性第二常见的泌尿系统癌症。LG-IR-NMIBC每年代表大约22000名新诊断的膀胱癌患者，在前几年诊断的患者中，估计每年有60000例复发。膀胱癌主要影响中位诊断年龄为73岁且合并症风险增加的老年人群。

Guideline recommendations for the management of NMIBC include 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..

NMIBC管理的指南建议包括TURBT作为护理标准。高达70%的NMIBC患者至少有一次复发，LG-IR-NMIBC患者更有可能复发并面临反复的TURBT手术。。

About UroGen Pharma Ltd.

关于UroGen制药有限公司

UroGen is a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers because patients deserve better options. UroGen has developed RTGel® reverse-thermal hydrogel, a proprietary sustained-release, hydrogel-based platform technology that has the potential to improve the therapeutic profiles of existing drugs.

UroGen是一家生物技术公司，致力于开发和商业化治疗尿路上皮癌和特殊癌症的创新解决方案，因为患者应该得到更好的选择。UroGen开发了RTGel®反向热水凝胶，这是一种专有的缓释水凝胶平台技术，有可能改善现有药物的治疗效果。

UroGen’s sustained release technology is designed to enable longer exposure of the urinary tract tissue to medications, making local therapy a potentially more effective treatment option. Our first product to treat low-grade upper tract urothelial cancer and investigational treatment UGN-102 (mitomycin) for intravesical solution for patients with LG-IR-NMIBC are designed to ablate tumors by non-surgical means.

我们的第一个治疗低度上尿路上皮癌的产品和用于LG-IR-NMIBC患者膀胱内溶液的研究性治疗UGN-102（丝裂霉素）旨在通过非手术手段消融肿瘤。

UroGen is headquartered in Princeton, NJ with operations in Israel. Visit www.UroGen.com to learn more or follow us on X (Twitter), @UroGenPharma..

UroGen总部位于新泽西州普林斯顿，在以色列运营。