UGN-102 Showed Promising Long-Term Results in the Phase 3 ENVISION Trial, Potentially Paving the Way for First FDA-Approved Treatment for LG-IR-NMIBC
UGN-102 Showed Promising Long-Term Results in the Phase 3 ENVISION Trial, Potentially Paving the Way for First FDA-Approved Treatment for LG-IR-NMIBC
The side effect profile of UGN-102 was consistent with previous clinical trials, further supporting its potential as a new treatment option for patients with LG-IR-NMIBC.- UGN-102 demonstrated 82.3% duration of response (DOR) at 12 months in patients who achieved complete response at 3 months
- 79.6% complete response rate at 3 months in patients treated with UGN-102
- Safety profile consistent with prior clinical trials of UGN-102
- UGN-102在12個月時顯示出82.3%的反應持續時間(DOR)，對於在3個月內完成反應的患者
- 用UGN-102治療的患者在3個月時，完全反應率爲79.6%
- 安全性與UGN-102先前臨床試驗一致
PRINCETON, N.J.--(BUSINESS WIRE)--Dec. 5, 2024--UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers, today announced the presentation of the Phase 3 ENVISION trial's efficacy and safety results at the Society of Urologic Oncology (SUO) annual meeting in Dallas, TX. These results, published online in the Journal of Urology in October, demonstrate that treatment with investigational therapy UGN-102, a mitomycin-based intravesical solution, resulted in a high and clinically meaningful complete response rate that was durable in patients with recurrent low-grade intermediate-risk non-muscle-invasive bladder cancer (LG-IR-NMIBC).
新澤西州普林斯頓--(商業新聞)--2024年12月5日--烏龍製藥（納斯達克：URGN），一家致力於開發和商業化治療尿路上皮和特種癌症的創新解決方案的生物科技公司，今天在德克薩斯州達拉斯召開的人類泌尿學腫瘤學會（SUO）年會上發佈了三期ENVISION試驗的療效和安全性結果。這些結果於10月在《泌尿學雜誌》上在線發佈，表明使用UGN-102（以絲裂黴素爲基礎的膀胱灌注液）治療，患者的複發性低級別中度風險非肌肉侵襲性膀胱癌（LG-IR-NMIBC）完成回應率高且具有臨床意義，並且在患者中持久有效。
"Finding options for patients with recurrent low-grade bladder cancer continues to be a major unmet need," says Max Kates, R. Christian B. Evensen Professor and an Associate Professor of Urology and Oncology, and Director, Division of Urologic Oncology at the Brady Urological Institute at Johns Hopkins and ENVISION study investigator. "We are excited by the overall results of the ENVISION trial, especially the durability of response data, that support the potential of UGN-102 as a viable treatment option for these patients."
"爲複發性低級別膀胱癌患者尋找選擇仍然是一個重大未滿足的需求，"約翰霍普金斯大學布雷迪泌尿學研究所泌尿科和腫瘤學副教授、泌尿學腫瘤學主任Max Kates教授說，ENVISION研究的調查員之一。"我們對ENVISION試驗的整體結果感到興奮，特別是響應數據的持久性，支持UGN-102成爲這些患者的可行治療選擇。"
In the ENVISION study, UGN-102 treatment showed an impressive 82.3% (95% CI: 75.9%, 87.1%) duration of response (DOR) at 12 months, according to the Kaplan-Meier estimate, in patients who achieved a complete response (CR) at 3 months following the initial treatment with UGN-102. The DOR at 15 months (n=43) and 18 months (n=9) remained robust, both at 80.9% (95% CI: 73.9%, 86.2%) according to the Kaplan-Meier estimates. These results build upon the trial's positive primary endpoint, a 79.6% (95% CI: 73.9%, 84.5%) CR rate 3 months after the first instillation of UGN-102.
在ENVISION研究中，UGN-102治療在12個月時顯示出令人印象深刻的82.3%（95% CI：75.9%，87.1%）反應持續時間（DOR），根據卡普蘭-梅耶估計，患者在UGN-102首次治療後3個月實現完全應答（CR）。在15個月（n=43）和18個月（n=9）時，DOR仍保持強勁，均爲80.9%（95% CI：73.9%，86.2%），根據卡普蘭-梅耶估計。這些結果建立在試驗的積極主要終點之上，首次灌注UGN-102後3個月CR率爲79.6%（95% CI：73.9%，84.5%）。
The side effect profile of UGN-102 was consistent with previous clinical trials, further supporting its potential as a new treatment option for patients with LG-IR-NMIBC.
UGN-102的副作用特徵與之前的臨床試驗一致，進一步支持其作爲LG-IR-NMIBC患者新治療期權的潛力。
"We are excited by the progress made in advancing UGN-102 as a potential treatment for LG-IR-NMIBC and securing a PDUFA goal date of June 13, 2025, from the FDA," said Mark Schoenberg, M.D., Chief Medical Officer, UroGen. "The strong durability of response observed in the ENVISION study highlights UGN-102's promising potential for patients. Given that many LG-IR-NMIBC patients are elderly and endure multiple surgeries under general anesthesia for their condition that impact their health and quality of life, there is an urgent need for alternative treatment options that can prolong recurrence-free periods and enhance patient outcomes."
「我們對推進UGN-102作爲LG-IR-NMIBC潛在治療方案的進展感到興奮，並且獲得FDA認證的PDUFA目標日期爲2025年6月13日，」烏龍製藥首席醫療官Mark Schoenberg萬.D.說。「在ENVISION研究中觀察到的強反應持久性凸顯了UGN-102對患者的前景潛力。考慮到許多LG-IR-NMIBC患者年紀較大並且在他們的控件下經歷多次全身麻醉手術，這影響了他們的健康和生活質量，因此迫切需要其他治療選擇，以延長無復發期並改善患者的結果。」
The most common treatment-emergent adverse events (TEAEs) in the ENVISION trial were dysuria, hematuria, urinary tract infection, pollakiuria, fatigue, and urinary retention. TEAEs were typically mild-to-moderate in severity and resolved or resolving. The ENVISION trial demonstrated a similar safety profile to that observed in other studies of UGN-102.
ENVISION試驗中的最常見治療相關不良事件（TEAE）是排尿困難、血尿、尿路感染、尿頻、疲勞和尿瀦留。TEAE通常爲輕度至中度，且已消退或正在消退。ENVISION試驗證明與UGN-102的其他研究中觀察到的安全性脫穎而出。
About UGN-102
關於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 RTGel technology, a sustained release, hydrogel-based formulation, UGN-102 is designed to treat tumors by enabling longer exposure of bladder tissue to mitomycin. UGN-102 is delivered to patients using a standard urinary catheter in an outpatient setting by a trained healthcare professional. UroGen completed the NDA submission for UGN-102 in August, ahead of schedule. The FDA accepted the NDA for UGN-102 and assigned a PDUFA goal date of June 13, 2025.
UGN-102（絲裂黴素）爲膀胱灌注溶液是一種絲裂黴素的創新藥物製劑，目前正在進行III期開發，以治療LG-IR-NMIBC。利用烏龍製藥的專有RTGel科技，這是一種持續釋放的水凝膠基製劑，UGN-102旨在通過延長膀胱組織與絲裂黴素的接觸時間來治療腫瘤。UGN-102通過受過訓練的醫療專業人員在門診環境中使用標準尿管輸送給患者。烏龍製藥於8月提前完成了UGN-102的NDA提交。FDA接受了UGN-102的NDA並指定了PDUFA目標日期爲2025年6月13日。
About Non-Muscle Invasive Bladder Cancer (NMIBC)
在美國，膀胱癌是男性最常見的泌尿系統癌症。 LG-IR-NMIBC每年約有22,000名新診斷的膀胱癌患者，並估計在之前年份被診斷的患者中每年復發約60,000次。膀胱癌主要影響年齡較大的人群，診斷的中位年齡爲73歲，伴有併發症的風險增加。 NMIBC的管理指南建議TURBT作爲標準護理。多達70％的NMIBC患者至少經歷一次復發，而LG-IR-NMIBC患者則更有可能復發並面臨重複的TURBT手術。
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 increased risk of comorbidities, with the median age of diagnosis being 73 years. 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. LG-IR-NMIBC is characterized by early or frequent recurrences, multiple tumors, or a solitary tumor larger than three centimeters.
在美國，膀胱癌是男性第二常見的泌尿系統癌症。LG-IR-NMIBC每年代表大約22,000例新診斷的膀胱癌患者，以及從前幾年診斷的患者中預計每年有60,000例復發。膀胱癌主要影響年長群體，並伴有增加的合併症風險，診斷的中位年齡爲73歲。NMIBC的管理指南推薦包括經尿道膀胱腫瘤切除術（TURBT）作爲標準護理。多達70%的NMIBC患者至少經歷一次復發，而LG-IR-NMIBC患者更容易復發，並面臨重複的TURBT手術。LG-IR-NMIBC的特點是早期或頻繁複發，多發腫瘤或單個腫瘤大於三厘米。
About ENVISION
關於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 therapy for 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 (NCT05243550).
第三階段ENVISION試驗是一項單臂、多國、多中心的研究，評估UGN-102（美託咪啶）作爲治療LG-IR-NMIBC患者的膀胱內溶液的療效和安全性。第三階段ENVISION試驗已完成目標招募，共招募約240名患者，分佈在56個地點。研究參與者接受了六次每週一次的UGN-102膀胱內灌注。主要終點評估在第一次灌注後的三個月評估中的完全緩解（CR）率，關鍵次要終點評估了在三個月評估中達成CR的患者的耐受性。欲了解更多關於第三階段ENVISION試驗的信息，請訪問（NCT05243550）。
About UroGen Pharma Ltd.
關於烏龍製藥有限公司
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'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 to learn more or follow us on X (Twitter), @UroGenPharma.
烏龍製藥是一家專注於開發和商業化治療尿路上皮和特殊癌症的創新解決方案的生物技術公司，因爲患者應該擁有更好的選擇。烏龍製藥已經開發出RTGel反熱水凝膠，這是一種潛在的持續釋放的水凝膠基平台技術，可以提高現有藥物的治療特性。烏龍的持續釋放技術旨在延長泌尿道組織接受藥物的時間，從而使局部治療成爲可能更有效的治療選擇。我們的第一個產品用於治療低級上尿路上皮癌（Joe Biden）和絲裂黴素的研究性治療UGN-102（絲裂黴素）膀胱內溶液用於LG-IR-NMIBC患者是無需手術消融腫瘤的治療手段。烏龍製藥總部位於新澤西州普林斯頓，以色列也有運營。請訪問該網站以了解更多信息或在X（Twitter）、@UroGenPharma上關注我們。
Forward-Looking Statements
前瞻性聲明
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 potential for UGN-102 as the first FDA-approved treatment for LG-IR-NMIBC; the long-term results in the ENVISION trial supporting the potential of UGN-102 as a viable treatment option for LG-IR NMIBC; the estimated annual U.S. patient population and demographics for LG-IR-NMIBC; the potential benefits to patients and opportunities for UGN-102, if approved; statements related to the PDUFA goal date for UGN-102 and the potential approval and timing thereof; the potential of UroGen's proprietary 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: 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 RTGel technology may not perform as expected; and UroGen may not successfully develop and receive regulatory approval of any other product that incorporates 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 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.
本新聞稿包含前瞻性聲明，按照1995年私人證券訴訟改革法案中的定義，包括但不限於關於以下內容的聲明：UGN-102作爲FDA首個批准的LG-IR-NMIBC治療方法的潛力；ENVISION試驗的長期結果支持UGN-102作爲LG-IR NMIBC可行治療選項的潛力；LG-IR-NMIBC的美國患者年人群和人口統計數據的估算；如果獲得批准，UGN-102對患者的潛在益處和機會；關於UGN-102的PDUFA目標日期及其潛在批准和時間的相關聲明；UroGen的專有RTGel技術有可能改善現有藥物的治療特性；以及UroGen的持續釋放技術使局部給藥比其他治療選項可能更有效。這些聲明受到許多風險、不確定性和假設的制約，包括但不限於：儘管UGN-102的NDA已被FDA接受提交，但不能保證該NDA將在預期的時間內，或根本上能夠支持UGN-102的批准；維持監管批准的能力；與商業化活動相關的複雜情況；任何已批准產品的標籤；UroGen行業的競爭；開發和商業化UroGen產品候選者的範圍、進展和擴展；相應市場的規模和增長以及市場接受率與替代療法相比的比率和程度；UroGen吸引或留住關鍵管理人員、董事會成員和其他人員的能力；UroGen的RTGel技術可能無法按預期表現；UroGen可能無法成功開發並獲得任何其他融入RTGel技術的產品的監管批准。考慮到這些風險和不確定性，以及在UroGen截至2024年9月30日的季度報告的風險因素部分中描述的其他風險和不確定性（該報告於2024年11月6日向SEC提交，可在www.sec.gov獲得），這些前瞻性聲明中討論的事件和情況可能不會發生，UroGen的實際結果可能與所預期或暗示的結果在實質上和不利上存在重大差異。任何前瞻性聲明僅在本新聞稿發佈日期生效，並基於UroGen在本發佈日可獲得的信息。
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INVESTOR CONTACT:
Vincent Perrone
Senior Director, Investor Relations
vincent.perrone@UroGen.com
609-460-3588 ext. 1093
投資者聯繫方式：
文森特·佩羅尼
投資者關係高級總監
vincent.perrone@烏龍製藥.com
609-460-3588 ext. 1093
MEDIA CONTACT:
Cindy Romano
Director, Communications
cindy.romano@urogen.com
609-460-3583 ext. 1083
媒體聯繫人：
Cindy Romano
通信-半導體
cindy.romano@urogen.com
609-460-3583 ext. 1083
Source: UroGen Pharma Ltd.
來源：烏龍製藥有限公司。
譯文內容由第三人軟體翻譯。