A Phase 3, Single-arm, Multicenter Study to Evaluate the Efficacy and Safety of UGN-103, a Novel Formulation of UGN-102, for the Treatment of Patients With Low-grade (LG) Non-muscle Invasive Bladder Cancer (NMIBC) at Intermediate-risk (IR) of Recurrence

This Phase 3, single-arm, multicenter study will evaluate the efficacy and safety of UGN-103, a novel formulation of UGN-102, instilled in the urinary bladder of patients with low-grade non-muscle invasive bladder cancer (LG-NMIBC).

开始日期2024-08-29

申办/合作机构

UroGen Pharma Ltd.

NCT05874921 / RecruitingN/A

uTRACT Jelmyto Registry: A Multicenter, Prospective and Retrospective Registry to Evaluate Real World Experience and Outcomes of Patients With Upper Tract Urothelial Cancer (UTUC) Treated With Jelmyto

The purpose of this registry is to evaluate real world experience and outcomes of patients with Upper Tract Urothelial Cancer (UTUC) treated with Jelmyto in the United States.

开始日期2023-11-17

申办/合作机构

UroGen Pharma Ltd.

EUCTR2021-005948-29-AT / Unknown status临床3期

A Phase 3, Single-Arm, Multicenter Study to Evaluate the Efficacy and Safety of UGN-102 as Primary Chemoablative Therapy in Patients with Low Grade (LG) Non-Muscle-Invasive Bladder Cancer (NMIBC) at Intermediate Risk (IR) of Recurrence - A Phase 3 Single-Arm Study of UGN-102 for Treatment of LG IR NMIBC

开始日期2022-09-14

申办/合作机构

UroGen Pharma Ltd.

100 项与 UroGen Pharma Ltd. 相关的临床结果

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项与 UroGen Pharma Ltd. 相关的文献（医药）

2023-10-01·The Journal of urology

Treatment of Low-grade Intermediate-risk Nonmuscle-invasive Bladder Cancer With UGN-102 ± Transurethral Resection of Bladder Tumor Compared to Transurethral Resection of Bladder Tumor Monotherapy: A Randomized, Controlled, Phase 3 Trial (ATLAS).

Article

作者: Nosov, Alexander ; Kopyltsov, Evgeny ; Raju, Sunil ; Burger, Brent ; Zubarev, Vadim ; Brown, Gordon ; Schoenberg, Mark ; Genov, Pencho ; Komlev, Dmitrii ; Huang, William C ; Marchev, Dobri ; Prasad, Sandip M ; Khuskivadze, Alexandre ; Orlov, Igor ; Hu, Brian ; Ganev, Tosho ; Shishkov, Dimitar ; Shore, Neal D ; Bjurlin, Marc ; Meads, Andrew

PURPOSELow-grade intermediate-risk nonmuscle-invasive bladder cancer is a chronic illness commonly treated by repetitive transurethral resection of bladder tumor. We compared the efficacy and safety of intravesical chemoablation with UGN-102 (a reverse thermal gel containing mitomycin), with or without subsequent transurethral resection of bladder tumor, to transurethral resection of bladder tumor alone in patients with low-grade intermediate-risk nonmuscle-invasive bladder cancer.MATERIALS AND METHODSThis prospective, randomized, phase 3 trial recruited patients with new or recurrent low-grade intermediate-risk nonmuscle-invasive bladder cancer to receive initial treatment with either UGN-102 once weekly for 6 weeks or transurethral resection of bladder tumor. Patients were followed quarterly by endoscopy, cytology, and for-cause biopsy. The primary end point was disease-free survival. All patients were followed for adverse events.RESULTSTrial enrollment was halted by the sponsor to pursue an alternative development strategy after 282 of a planned 632 patients were randomized to UGN-102 ± subsequent transurethral resection of bladder tumor (n=142) or transurethral resection of bladder tumor monotherapy (n=140), rendering the trial underpowered to perform hypothesis testing. Patients were predominantly male and ≥65 years of age. Tumor-free complete response 3 months after initial treatment was achieved by 92 patients (65%) who received UGN-102 and 89 patients (64%) treated by transurethral resection of bladder tumor. The estimated probability of disease-free survival 15 months after randomization was 72% for UGN-102 ± transurethral resection of bladder tumor and 50% for transurethral resection of bladder tumor (hazard ratio 0.45). The most common adverse events (incidence ≥10%) in the UGN-102 group were dysuria, micturition urgency, nocturia, and pollakiuria.CONCLUSIONSPrimary, nonsurgical chemoablation with UGN-102 for the management of low-grade intermediate-risk nonmuscle-invasive bladder cancer offers a potential therapeutic alternative to immediate transurethral resection of bladder tumor monotherapy and warrants further investigation.

2022-01-01·PDA Journal of Pharmaceutical Science and Technology

Points to Consider When Using KPIs/Metrics

Review

作者: Orszula, Timothy E ; Taylor, Daniel ; Esteban Nunez, Ana ; Nezlin, Alexander ; Moritz, Sven Alexander ; Hinsch, Bernhard

Pharmaceutical quality systems according to ICH Q10 require the identification of processes that need to be measured. Designing and maintaining a metrics system in a way that serves its purpose well requires careful consideration of various aspects in order to meet expectations. The purpose of using key performance indicators/metrics can be defined by main aspects that include: 1) supporting the achievement of objectives by making them measurable, 2) reaching comparability and/or standardization of performance for organizational units, and 3) achieving objectiveness in assessing performance. The first two aspects are closely linked to management responsibilities. Therefore, management needs to be strongly involved with the activities of setting up and maintaining a metrics system. This article provides an overview of the points to be considered when setting up and managing a quality-related metrics system.

2021-06-01·Cancer Chemotherapy and Pharmacology4区 · 医学

Pharmacokinetics of UGN-101, a mitomycin-containing reverse thermal gel instilled via retrograde catheter for the treatment of low-grade upper tract urothelial carcinoma

4区 · 医学

Article

作者: Smith, Angela B ; Seltzer, Elyse ; Scherr, Douglas ; Shabsigh, Ahmad ; Kleinmann, Nir ; Schoenberg, Mark ; Lerner, Seth P

AbstractPurposeTo evaluate the pharmacokinetic properties of UGN-101, a mitomycin-containing reverse thermal gel used as primary chemoablative treatment for low-grade upper tract urothelial carcinoma (UTUC), in a subset of patients participating in a phase 3 clinical trial.MethodsPharmacokinetic parameters (Cmax, Tmax, AUC(0–6), λz, t½, and AUCinf) were evaluated in six participants (male or female, ≥ 18 years) with biopsy-proven, low-grade UTUC who received the first of 6 once-weekly instillations of UGN-101 to the renal pelvis and calyces via retrograde ureteral catheter. Plasma samples were collected prior to instillation and 30 min, 1, 2, 3, 4, 5, and 6 h post-instillation. Safety was assessed by laboratory evaluations, physical exam, and adverse event monitoring.ResultsThe mean age of the six participants was 69 years; most were male (5/6) and Caucasian (5/6). Mean (SD) Cmax was 6.24 (4.11) ng/mL and mean Tmax was 1.79 (1.89) hours after instillation. Mean apparent t½ following instillation was 1.27 (0.63) hours. Mean total systemic exposure to mitomycin up to 6 h post-instillation was 20.30 (19.69) ng h/mL. At 6 h post-instillation, mitomycin plasma concentrations of 5/6 participants were < 2 ng/mL. There were no clinically important adverse events or changes in laboratory values in any participant after a single instillation of UGN-101.ConclusionThe reverse thermal gel formulation of UGN-101 is associated with higher concentration and extended dwell time of mitomycin in contact with the urothelium of the upper urinary tract while limiting systemic absorption of mitomycin.RegistrationNCT02793128; registered June 8, 2016.

117

项与 UroGen Pharma Ltd. 相关的新闻（医药）

2024-10-29

·凯莱英药闻

超82.3%患者持久一年无癌！潜在首款LG-IR-NMIBC药物预计将于明年中旬获批

近日，UroGen Pharma宣布，公司针对UGN-102在低级别中等风险非肌层浸润性膀胱癌（LGIR-NMIBC）患者中开展的3 期 ENVISION 研究的长期持久性反应结果在《泌尿外科杂志》上在线发表。 UGN-102是一种创新的丝裂霉素（mitomycin）药物，其用于LGIR-NMIBC的新药申请(NDA)于2024年8月提交，指定的PDUFA 目标日期为 2025 年 6月 13 日。 3期ENVISION 试验是一项单组、跨国、多中心研究，旨在评估 UGN-102膀胱内灌注溶液作为 LG-IR-NMIBC 患者的化学消融疗法的有效性和安全性。患者每周接受一次 UGN-102 膀胱内灌注，共六次；主要终点是评估首次灌注后三个月评估的完全缓解(CR)率，关键次要终点是在三个月评估中达到 CR患者的长期疗效。结果显示：在首次灌注UGN-102三个月达到完全缓解 (CR) 的患者中，82.3%显示了12个月持续反应(DOR)。 3个月CR后，15个月（n=43）和18个月（n=9）的DOR估计值均为80.9%（95% CI, 73.9%, 86.2%）。研究达到了主要终点，即首次滴注后三个月的CR率为79.6%。最常见的治疗引起的不良事件 (TEAE) 是排尿困难、血尿、尿路感染、尿频、疲劳和尿潴留。TEAE 的严重程度通常为轻度至中度。 UGN-102利用 UroGen 专有的 RTGel® 技术（一种缓释水凝胶配方），旨在使膀胱组织更长时间地接触丝裂霉素，从而能够通过非手术方式治疗肿瘤。UGN-102 可借由受过培训的医疗专业人员在门诊环境中通过标准导尿管给药。美国 FDA 尚未批准用于 LG-IR-NMIBC 初级治疗的药物；UGN-102有望成为首款获批产品，作为重复 TURBT 的非手术替代方案。目前，该药物在两项3期临床试验中，均显示出令人信服的临床结果。膀胱癌是泌尿系统常见的恶性肿瘤，根据癌细胞是否进入侵至肌肉层，分为非肌层浸润性膀胱癌（NMIBC）和肌层浸润性膀胱癌（MIBC），NMIBC 约占膀胱癌的80%。经尿道膀胱肿瘤切除手术（TURBt）是NMIBC 的标准治疗方式，但术后肿瘤残余会导致复发。NMIBC 患者经过标准治疗后1 年复发率15-61%，5 年复发率为31-78%，复发风险较高。 NMIBC 患者经过TURBt后的辅助治疗为BCG 灌注治疗，复发后只能重复灌注或切除膀胱，切除膀胱后患者需要终生携带尿袋，严重影响生活质量。因此灌注治疗后对新疗法需求迫切。 2020 年，FDA 批准K 药作为单药，治疗不符合或已选择不进行膀胱切除术的BCG 无应答、高风险、伴CIS、伴或不伴乳头状肿瘤的NMIBC 患者；除此以外，2024 年4 月，FDA再次批准mmunityBio 公司的IL-15药物Anktiva（N-803）联合卡介苗（BCG），用于治疗卡介苗不响应的NMIBC伴原位癌（CIS），伴或不伴乳头状瘤患者。 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和特殊癌症的创新解决方案。公司首发的两个资产Jelmyto 和 UGN-102均是现有活性药物丝裂霉素C(Mitomycin C，MMC) 与RTGel结合的新型缓释制剂。公司核心平台RTGel ®是一种反向热凝胶技术。与大多数在较低温度下变稠的液体不同，RTGel 在较低温度下的粘度会降低。在水的冰点下，RTGel与水相似，而在体温 37 摄氏度下，RTGel会凝固并表现出果冻的粘度。RTGel 聚合物基于FDA归类为安全(GRAS) 的非活性成分。 UroGen 的策略是利用 RTGel 的这些独特特性来改善药物的递送，其反向热凝胶技术叠加灌注的药品，进入泌尿系统后10min后就会凝固，通过增加停留时间和药物暴露从而提高临床效果。参考资料公司官网、ImpromptuThoughts 内容来源于网络，如有侵权，请联系删除。

临床结果申请上市

2024-10-17

·PipelineReview

UroGen Announces FDA Acceptance of its New Drug Application for UGN-102

PRINCETON, NJ, USA I October 15, 2024 I 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 . 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. “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. 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.” 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. 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. 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.” About 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. 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. About 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. 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. Learn more about the Phase 3 ENVISION trial at www.clinicaltrials.gov (NCT05243550). About Non-Muscle Invasive Bladder Cancer (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. 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. About UroGen Pharma Ltd. 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’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. UroGen is headquartered in Princeton, NJ with operations in Israel. Visit www.UroGen.com to learn more or follow us on X (Twitter), @UroGenPharma. SOURCE: UroGen Pharma

临床3期临床结果申请上市

2024-10-16

·药明康德

肿瘤3个月后完全消失，超80%患者持久一年无癌，FDA接受创新疗法上市申请！

▎药明康德内容团队编辑日前，UroGen Pharma宣布美国FDA已接受其在研疗法UGN-102膀胱内灌注液的新药申请（NDA），用于治疗低级别中等风险非肌层浸润性膀胱癌（LG-IR-NMIBC）患者。FDA预计在2025年6月13日以前公布审评结果。根据新闻稿，如果获批，UGN-102将成为美国FDA批准用于治疗LG-IR-NMIBC患者的首款药物。该新药申请主要基于ENVISION试验的积极数据。这是一项单臂、多中心的3期研究，旨在评估UGN-102膀胱内灌注液作为主要化学消融疗法，在LG-IR-NMIBC患者中的疗效和安全性，共有约240名患者入组。受试者接受每周一次UGN-102灌注共6次。主要终点为首次灌注后三个月的完全缓解（CR）率，关键次要终点则评估达到CR患者随时间推移的疗效持续性。结果显示，该试验达到主要终点，接受UGN-102治疗患者在首次灌注后三个月的CR率为79.6%（95% CI：73.9-84.5）。而通过Kaplan-Meier分析（n=108）的12个月缓解持续时间（DOR）数据显示，达到CR的患者中有82.3%（95% CI：75.9-87.1）在首次灌注UGN-102后持续缓解12个月。此外，在第15个月（n=43）和18个月（n=9）的DOR估计值均为80.9%（95% CI：73.9-86.2）。图片来源：123RF 最常见的治疗伴发不良事件（TEAE）包括排尿困难、血尿、尿路感染、尿频、疲劳和尿潴留，TEAE通常为轻度至中度。 UGN-102膀胱灌注液是一种创新的丝裂霉素（mitomycin）药物。该疗法利用UroGen专有的RTGel技术，这种基于水凝胶的持续释放制剂可使膀胱组织能够更长时间地暴露于丝裂霉素之下，从而通过非手术手段治疗肿瘤。UGN-102可借由受过培训的医疗专业人员在门诊环境中通过标准导尿管给药。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料： [1] UroGen Announces FDA Acceptance of its New Drug Application for UGN-102. Retrieved October 16, 2024 from https://www.businesswire.com/news/home/20241015921667/en 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

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