PRINCETON, N.J.--(

新泽西州普林斯顿--(

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)--UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, today announced results from the subgroup analyses of the pivotal ENVISION study evaluating the impact of tumor burden and focality on the response to UGN-102, an investigational drug in development for treatment of LG-IR-NMIBC.

)--UroGen Pharma Ltd.（纳斯达克：URGN）是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，今天宣布了关键ENVISION研究的亚组分析结果，该研究评估了肿瘤负荷和病灶对UGN-102（一种正在开发的用于治疗LG-IR-NMIBC的研究药物）反应的影响。

Analyses of pre-specified subgroups showed comparable CR rates and DOR patterns across different levels of tumor burden (defined as the sum of the diameters of all visible tumors) and number of tumors at baseline in patients treated with UGN-102. The poster presentation titled, .

对预先指定的亚组的分析显示，在接受UGN-102治疗的患者中，不同水平的肿瘤负荷（定义为所有可见肿瘤直径的总和）和基线肿瘤数量的CR率和DOR模式相当。海报展示名为。

Impact of Tumor Burden or Focality in Recurrent Low-Grade Intermediate-Risk Non-Muscle-Invasive Bladder Cancer on Response to Treatment with UGN-102: A Substudy of the Phase 3 ENVISION Trial

复发性低级别中危非肌肉浸润性膀胱癌的肿瘤负荷或病灶对UGN-102治疗反应的影响：3期ENVISION试验的子研究

was presented today at ASCO GU 2025.

今天在ASCO GU 2025上发表。

'In this subgroup analysis on tumor burden and focality from the ENVISION study, the overall CR rate was consistent, and a majority of patients maintained their response 12 months after achieving CR at three months,' said Sandip M. Prasad, M.D., Director of Urology at Morristown Medical Center and lead author of the study.

莫里斯敦医学中心泌尿科主任兼该研究的主要作者桑迪普·普拉萨德（SandipM.Prasad）医学博士说：“在ENVISION研究中对肿瘤负荷和病灶的亚组分析中，总体CR率是一致的，大多数患者在三个月达到CR后12个月仍保持反应。”。

'These results provide evidence of UGN-102’s potential to become an effective and durable treatment option for patients with recurrent LG-IR-NMIBC, regardless of tumor size or focality.'.

“这些结果提供了UGN-102有潜力成为复发性LG-IR-NMIBC患者的有效和持久的治疗选择的证据，无论肿瘤大小或病灶如何。”。

The ENVISION study included 240 patients with recurrent LG-IR-NMIBC who received at least one dose of UGN-102 and 95% (228 patients) received all six weekly intravesical instillations of UGN-102. In this pre-specified analysis CR rates were 82.8% for patients with a tumor burden ≤3 cm (n=149/180) compared to 73.2% for those with a tumor burden >3 cm (n=30/41).

ENVISION研究包括240名复发性LG-IR-NMIBC患者，他们接受了至少一剂UGN-102，95%（228名患者）接受了所有六周一次的UGN-102膀胱内滴注。在这个预先指定的分析中，肿瘤负荷≤3 cm（n=149/180）患者的CR率为82.8%，而肿瘤负荷>3 cm（n=30/41）患者的CR率为73.2%。

Of the patients with CR at three months, 15.4% vs 20.0% experienced recurrence of low-grade disease, progression (either in stage or grade), or death (unrelated to treatment) by 15 months. In the 191 patients with multiple vs single tumors who achieved a CR, the three-month CR was 79.3% vs 82.9%, with recurrence rates of 18.5% vs 11.8%..

在三个月的CR患者中，15.4%比20.0%的患者在15个月内出现低度疾病复发，进展（分期或分级）或死亡（与治疗无关）。在1911例多发性和单发性肿瘤患者中，三个月的CR分别为79.3%和82.90%，复发发生概率分别为18.5%和11.8%…的患者中，CR的发生与复发的发生有密切关系。的患者中，CR的发生与复发的发生有密切关系。的患者中，CR的发生与复发的发生有密切关系。。的患者中。。

“We are encouraged by these results, which highlight the potential of UGN-102 to provide durable responses in patients with recurrent LG-IR-NMIBC,” said Mark Schoenberg, M.D., Chief Medical Officer of UroGen. “These pre-specified subgroup analyses further strengthen the growing body of evidence supporting UGN-102 as a treatment option for patients dealing with the challenges of disease recurrence and the need for repeated surgeries.”.

UroGen首席医学官马克·勋伯格（Mark Schoenberg）医学博士说：“我们对这些结果感到鼓舞，这些结果突显了UGN-102在复发性LG-IR-NMIBC患者中提供持久反应的潜力。”。“这些预先指定的亚组分析进一步加强了越来越多的证据支持UGN-102作为应对疾病复发挑战和需要重复手术的患者的治疗选择。”。

The ENVISION trial’s primary efficacy and safety results, previously presented, showed that UGN-102 delivered a 79.6% CR rate at three months, with an unprecedented 82.3% probability of remaining in response 12 months later by Kaplan-Meier estimates.

先前介绍的ENVISION试验的主要疗效和安全性结果显示，UGN-102在三个月时的CR率为79.6%，Kaplan-Meier估计在12个月后仍有82.3%的可能性是前所未有的。

These findings further support UGN-102’s potential to alter the treatment landscape for patients with LG-IR-NMIBC, a condition where recurrence and progression remain significant clinical challenges.

这些发现进一步支持了UGN-102改变LG-IR-NMIBC患者治疗前景的潜力，这种情况下复发和进展仍然是重大的临床挑战。

UroGen initiated the submission of a rolling new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for UGN-102 as a treatment for LG-IR-NMIBC in January 2024 and completed the NDA submission in August, ahead of schedule. The FDA accepted the NDA for UGN-102 with a Prescription Drug User Fee Act (PDUFA) goal date of June 13, 2025..

UroGen于2024年1月开始向美国食品和药物管理局（FDA）提交UGN-102的滚动新药申请（NDA），作为LG-IR-NMIBC的治疗方法，并于8月提前完成了NDA提交。FDA接受了UGN-102的NDA，处方药用户费用法案（PDUFA）的目标日期为2025年6月13日。。

The most common treatment-emergent adverse events (TEAEs) in the ENVISION trial were dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention. The TEAEs were typically mild-to-moderate in severity and either resolved or were resolving. The ENVISION trial demonstrated a similar safety profile to that observed in other studies of UGN-102..

ENVISION试验中最常见的治疗紧急不良事件（TEAE）是排尿困难，血尿，尿路感染，pollakiuria，疲劳和尿潴留。。ENVISION试验证明了与UGN-102其他研究中观察到的相似的安全性。。

Study limitations include the small sample size of the comparator groups and the single arm design.

研究局限性包括比较组的样本量小和单臂设计。

The analysis in another presentation titled,

另一篇题为，

Treatment of Low-Grade Intermediate-Risk Non-Muscle-Invasive Bladder Cancer with UGN-102: Results of the Phase 3 ATLAS and ENVISION Studies

UGN-102治疗低度中度风险非肌层浸润性膀胱癌：3期ATLAS和ENVISION研究的结果

highlights that in both studies, the CR rate in patients initially treated with UGN-102 was robust, with the majority of patients remaining event-free at 12 months after a three-month CR. A new analysis from the ATLAS trial highlights 54% decreased risk of recurrence, progression or death with UGN-102 compared to TURBT.

强调在这两项研究中，最初接受UGN-102治疗的患者的CR率都很高，大多数患者在三个月的CR后12个月仍无事件发生。ATLAS试验的一项新分析显示，与TURBT相比，UGN-102的复发，进展或死亡风险降低了54%。

The hazard ratio for DOR in ATLAS was 0.46 with a 95% confidence interval (0.24, 0.86) favoring the UGN-102 arm. Median DOR was not estimable in any arm due to low recurrence rates; the most common adverse event with UGN-102 in both studies was dysuria, occurring in 22.5% in ENVISION and 30.4% in ATLAS..

ATLAS中DOR的风险比为0.46，95%置信区间（0.24，0.86）有利于UGN-102组。由于复发率低，任何手臂的中位DOR都不可估计；。。

About UGN-102

关于UGN-102

UGN-102 (mitomycin) for intravesical solution is an innovative drug formulation of mitomycin, currently in Phase 3 development for the treatment of LG-IR-NMIBC. Utilizing UroGen’s proprietary

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，目前处于用于治疗LG-IR-NMIBC的3期开发中。利用UroGen专有

RTGel

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. UGN-102 is delivered to patients using a standard urinary catheter in an outpatient setting by a trained healthcare professional.

UGN-102是一种缓释水凝胶制剂，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。

UroGen completed the NDA submission in August, ahead of schedule. The FDA accepted the NDA for UGN-102 and assigned a PDUFA goal date of June 13, 2025..

UroGen于8月提前完成了NDA提交。FDA接受了UGN-102的NDA，并将PDUFA的目标日期定为2025年6月13日。。

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 23,000 newly diagnosed bladder cancer patients each year and an estimated 59,000 recurrences annually among patients diagnosed in previous years. Bladder cancer primarily affects older populations with increased risk of comorbidities, with the median age of diagnosis being 73 years.

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23000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59000例复发。膀胱癌主要影响合并症风险增加的老年人群，中位诊断年龄为73岁。

Guideline recommendations for the management of NMIBC include trans-urethral 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..

。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 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 a 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。

Learn more about the Phase 3 ENVISION trial at .

了解有关第3阶段ENVISION试用的更多信息，请访问。

www.clinicaltrials.gov

www.clinicaltrials.gov

(NCT05243550).

（NCT05243550）。

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

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。

RTGel

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’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.

反向热水凝胶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的缓释技术旨在使尿路组织更长时间暴露于药物，使局部治疗成为潜在的更有效的治疗选择。

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. UroGen is headquartered in Princeton, NJ with operations in Israel. To learn more, visit .

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总部位于新泽西州普林斯顿，在以色列运营。要了解更多信息，请访问。

www.UroGen.com

www.UroGen.com

or follow us on X (Twitter), @UroGenPharma.

或者在X（推特），@UroGenPharma上关注我们。

Forward-Looking Statements

前瞻性声明

This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including, without limitation, statements regarding: the ENVISION subgroup analyses providing evidence of UGN-102's potential to become an effective and durable treatment option for patients with recurrent LG-IR-NMIBC, regardless of tumor size or focality and further strengthening the growing body of evidence supporting UGN-102 as a treatment option for patients dealing with the challenges of disease recurrence and repeated surgeries; the potential benefits to patients and opportunities for UGN-102, if approved, including to alter the treatment landscape for patients with LG-IR-NMIBC; statements related to UroGen’s NDA submission and expected PDUFA target action date for UGN-102; the estimated annual U.S.

本新闻稿包含1995年《私人证券诉讼改革法案》中定义的前瞻性声明，包括但不限于以下声明：ENVISION亚组分析提供了UGN-102有可能成为复发性LG-IR-NMIBC患者的有效和持久治疗选择的证据，无论肿瘤大小或病灶如何，并进一步加强了越来越多的证据支持UGN-102作为应对疾病复发和重复手术挑战的患者的治疗选择；如果获得批准，UGN-102对患者的潜在益处和机会，包括改变LG-IR-NMIBC患者的治疗环境；有关UroGen NDA提交和UGN-102预期PDUFA目标行动日期的声明；估计每年的美国。

patient population and demographics for LG-IR-NMIBC; the potential of UroGen’s proprietary .

LG-IR-NMIBC的患者人群和人口统计学；UroGen专有技术的潜力。

RTGel

RTGel

technology to improve therapeutic profiles of existing drugs; and UroGen’s sustained release technology making local delivery potentially more effective as compared to other treatment options. These statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to: prior results may not be indicative of results that may be observed in the future; even though the NDA for UGN-102 has been accepted for filing by the FDA, there is no guarantee that such NDA will be sufficient to support approval of UGN-102 on the timeframe expected, or at all; the ability to maintain regulatory approval; complications associated with commercialization activities; the labeling for any approved product; competition in UroGen’s industry; the scope, progress and expansion of developing and commercializing UroGen’s product candidates; the size and growth of the market(s) therefor and the rate and degree of market acceptance thereof vis-à-vis alternative therapies; UroGen’s ability to attract or retain key management, members of the board of directors and other personnel; UroGen’s .

改进现有药物治疗概况的技术；与其他治疗方案相比，UroGen的缓释技术使局部分娩可能更有效。这些报表受到许多风险、不确定性和假设的影响，包括但不限于：先前的结果可能并不表明未来可能观察到的结果；尽管UGN-102的NDA已被FDA接受备案，但不能保证此类NDA足以支持在预期的时间范围内或根本不支持UGN-102的批准；保持监管批准的能力；与商业化活动相关的并发症；任何批准产品的标签；尿激素行业的竞争；UroGen候选产品开发和商业化的范围、进展和扩展；因此，市场的规模和增长以及相对于替代疗法的市场接受率和程度；UroGen吸引或留住关键管理人员、董事会成员和其他人员的能力；尿原的。

RTGel

RTGel

technology may not perform as expected; and UroGen may not successfully develop and receive regulatory approval of any other product that incorporates

技术可能无法按预期运行；UroGen可能无法成功开发并获得任何其他包含

RTGel

RTGel

technology. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section of UroGen’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, filed with the SEC on November 6, 2024 (which is available at

技术。鉴于这些风险和不确定性，以及UroGen于2024年11月6日向SEC提交的截至2024年9月30日的季度报告表10-Q中风险因素部分所述的其他风险和不确定性（可在

www.sec.gov

www.sec.gov

), the events and circumstances discussed in such forward-looking statements may not occur, and UroGen’s actual results could differ materially and adversely from those anticipated or implied thereby. Any forward-looking statements speak only as of the date of this press release and are based on information available to UroGen as of the date of this release..

)，此类前瞻性声明中讨论的事件和情况可能不会发生，UroGen的实际结果可能与预期或暗示的结果存在重大不利差异。任何前瞻性声明仅在本新闻稿发布之日起生效，并基于UroGen截至本新闻稿发布之日可获得的信息。。