Urogen Pharma Ltd (URGN) 2025 Q1 法說會逐字稿

Hello and welcome to UroGen Pharma first-quarter 2025 earnings conference call. (Operator Instructions)

您好，歡迎參加 UroGen Pharma 2025 年第一季財報電話會議。（操作員指示）

I would now like to turn the conference over to Vincent Perrone. You may begin.

現在我想將會議交給 Vincent Perrone。你可以開始了。

Thank you, operator. Good morning, everyone, and welcome to UroGen Pharma's first-quarter 2025 financial results and business update conference call. Earlier this morning, we issued a press release providing an overview of our recent corporate highlights and financial results for the quarter ended March 31, 2025. The press release can be accessed on the investors portion of our website at investors.urogen.com.

謝謝您，接線生。大家早安，歡迎參加 UroGen Pharma 2025 年第一季財務業績和業務更新電話會議。今天早些時候，我們發布了一份新聞稿，概述了我們最近的公司亮點和截至 2025 年 3 月 31 日的季度的財務業績。您可在我們網站的投資者部分（網址為 investors.urogen.com）上查閱該新聞稿。

Joining me on the call today are Liz Barrett, President and Chief Executive Officer; Dr. Mark Schoenberg, Chief Medical Officer; David Lin, Chief Commercial Officer; and Chris Degnan, Chief Financial Officer.

今天與我一起參加電話會議的還有總裁兼執行長 Liz Barrett、首席醫療官 Mark Schoenberg 博士、商務長 David Lin 和財務長 Chris Degnan。

During today's call, we will be making certain forward-looking statements. These may include statements regarding our ongoing pre-commercialization activities related to UGN-102, regulatory meetings and decisions, our commercialization strategy and expectations, as well as potential future commercialization activities for UGN-102 if approved, market and revenue opportunities, commercialization activities related to Jelmyto, our ongoing and planned clinical trials, commercial and clinical milestones, UGN-102 being the primary growth driver for UroGen, future R&D efforts, and our goals in 2025 financial guidance, among other things.

在今天的電話會議中，我們將做出一些前瞻性的陳述。這些可能包括有關我們正在進行的與 UGN-102 相關的商業化前活動的聲明、監管會議和決定、我們的商業化策略和預期，以及如果獲得批准，UGN-102 未來的潛在商業化活動、市場和收入機會、與 Jelmyto 相關的商業化活動、我們正在進行和計劃中的臨床發展、市場和收入機會、與 Jelmyto 相關的商業化活動、我們正在進行和計劃中的臨床發展、商業和臨床里程碑、Gen.102 2025 成長年財務指導中的目標等。

These forward-looking statements are based on current information, assumptions, and expectations that are subject to change. The description of potential risks can be found in our earnings press release and latest SEC disclosure documents. You are cautioned not to place undue reliance on these forward-looking statements, and UroGen disclaims any obligation to update these statements.

這些前瞻性陳述基於當前資訊、假設和預期，可能會發生變化。潛在風險的描述可以在我們的收益新聞稿和最新的 SEC 揭露文件中找到。請注意不要過度依賴這些前瞻性陳述，UroGen 不承擔更新這些陳述的任何義務。

I'll now turn the call over to Liz Barrett, Chief Executive Officer. Liz?

現在我將把電話轉給執行長 Liz Barrett。莉茲？

Thank you, Vincent, and thank you all for joining us this morning. The new drug application for our lead development stage candidate, UGN-102, is now in the final stages of FDA review with a PDUFA target date of June 13. We designed the UGN-102 as a novel and innovative treatment for patients with low-grade intermediate risk, non-muscle invasive bladder cancer.

謝謝你，文森特，也謝謝大家今天早上加入我們。我們處於領先開發階段的候選藥物 UGN-102 的新藥申請目前正處於 FDA 審查的最後階段，PDUFA 目標日期為 6 月 13 日。我們設計了 UGN-102 作為針對低度中度風險、非肌肉浸潤性膀胱癌患者的新穎創新治療方法。

We believe UGN-102 has the potential to change the treatment paradigm and meaningfully improve the standard of care in this patient population. If approved, UGN-102 will be the primary growth driver for our company, and alongside Jelmyto could solidify our leadership in the urothelial cancer space, advancing our mission to bring innovative, patient-centered solutions to urologic cancers.

我們相信 UGN-102 有潛力改變治療模式並顯著提高該患者群體的護理標準。如果獲得批准，UGN-102 將成為我們公司的主要成長動力，並且與 Jelmyto 一起鞏固我們在泌尿道上皮癌領域的領導地位，推進我們為泌尿系統癌症帶來創新的、以患者為中心的解決方案的使命。

We have been informed by the FDA that an Oncologic Drugs Advisory Committee, or ODAC, meeting has been scheduled for UGN-102 on May 21. This is consistent with our expectations, and we look forward to the opportunity to showcase the strength and consistency of our clinical data to the members of the panel and the public. We have been preparing for the ODAC meeting and believe we are well prepared to present a clear, compelling, and scientifically robust case supporting the approval of UGN-102.

我們已收到 FDA 的通知，腫瘤藥物諮詢委員會 (ODAC) 會議已安排於 5 月 21 日為 UGN-102 舉行。這與我們的預期一致，我們期待有機會向專家組成員和公眾展示我們臨床數據的強度和一致性。我們一直在為 ODAC 會議做準備，並相信我們已經做好充分準備，可以提出清晰、令人信服且科學有力的案例來支持批准 UGN-102。

In parallel, our regulatory team continues to engage regularly with the agency and has been responding to their information request. To date, we've encountered no resource or policy related issues that concern us. The UGN-102 NDA is supported by a robust development program, demonstrating meaningful complete response rate, durable responses, and an acceptable safety profile across three late-stage clinical trials.

同時，我們的監管團隊繼續定期與該機構合作，並一直在回應他們的資訊請求。到目前為止，我們還沒有遇到任何令我們擔憂的資源或政策相關問題。UGN-102 NDA 得到了強大的開發計劃的支持，在三個後期臨床試驗中展示了有意義的完全緩解率、持久的反應和可接受的安全性。

In March, we reported updated data from the pivotal ENVISION trial, demonstrating that 80.6% of patients who achieved a CR at three months remained in response at 18 months per Kaplan-Meier estimate. It's important to highlight that these robust results are with UGN-102 alone and not following a transurethral resection of bladder tumor.

3 月份，我們報告了關鍵性 ENVISION 試驗的最新數據，顯示根據 Kaplan-Meier 估計，80.6% 在 3 個月時達到 CR 的患者在 18 個月時仍保持療效。值得強調的是，這些可靠的結果是單獨使用 UGN-102 獲得的，而不是經尿道膀胱腫瘤切除術後所獲得的。

Additionally, there are six weekly installations. Then patients are treatment-free until recurrence. As Mark will highlight, these results, along with the broader update on UGN-102 and Jelmyto, were shared with the urology community at this year's AUA meeting, underscoring our clinical leadership and commitment to advancing innovation in neuro-encology.

此外，每週還有六次安裝。然後患者無需接受治療，直到復發。正如馬克所強調的，這些結果以及 UGN-102 和 Jelmyto 的更廣泛更新已在今年的 AUA 會議上與泌尿科界分享，強調了我們的臨床領導力和推動神經內科創新的承諾。

With the PDUFA date goal approximately one month away, a commercial team has been actively preparing for the potential launch of UGN-102. This launch would mark a pivotal moment in UroGen's evolution from a rare disease focused company to a scaled multi-product team, positioned to serve a significantly broader patient population.

距離 PDUFA 日期目標大約還有一個月的時間，商業團隊一直在積極為 UGN-102 的潛在推出做準備。此次發布標誌著 UroGen 從一家專注於罕見疾病的公司發展成為一家規模龐大的多產品團隊的關鍵時刻，旨在為更廣泛的患者群體提供服務。

We're expanding our commercial footprint accordingly with plans to grow our sales force from approximately 50 reps today to over 80 at launch. Our medical affairs and market access teams are also deeply engaged in pre-launch planning. We are targeting commercial readiness by June, and we will be ready to promote immediately following approval with product availability in July.

我們正在相應地擴大我們的商業足跡，計劃將我們的銷售隊伍從目前的約 50 名代表擴大到推出時的 80 多名。我們的醫療事務和市場准入團隊也深入參與了上市前的規劃。我們的目標是在 6 月實現商業化，並在 7 月產品獲批並上市後立即進行推廣。

UGN-102 represents a transformative growth opportunity for UroGen. We estimate an addressable population of approximately 60,000 patients annually, with recurrent, low-grade intermediate risk non-muscle invasive bladder cancer, translating to a market opportunity of over $5 billion. This is nearly 10 times larger than the Jelmyto market.

UGN-102 代表 UroGen 的一個變革性成長機會。我們估計，每年可治療的複發性、低度、中度風險非肌肉層浸潤性膀胱癌患者人數約為 6 萬人，這意味著超過 50 億美元的市場機會。這幾乎是 Jelmyto 市場的 10 倍。

Critically, this market is highly accessible. Unlike the more fragmented UTUC setting. In NMIBC, patients are widely distributed and primarily managed by community urologists across the country. UGN-102 is well aligned with current clinical workflows. It's easy to administer, does not require specialized equipment, and can be delivered by a nurse with minimal additional training.

至關重要的是，這個市場非常容易進入。與更分散的 UTUC 設定不同。在北米利班，患者分佈廣泛，主要由全國各地的社區泌尿科醫師管理。UGN-102 與目前的臨床工作流程非常吻合。它易於管理，不需要專門的設備，只需經過少量額外培訓的護士即可完成。

We believe these advantages position UGN-102 to become a foundational therapy in the management of low-grade intermediate risk non-muscle invasive bladder cancer and a significant driver of long-term value creation for UroGen.

我們相信，這些優勢將使 UGN-102 成為治療低度中度風險非肌肉層浸潤性膀胱癌的基礎療法，並成為 UroGen 長期價值創造的重要驅動力。

Turning to Jelmyto, we reported $20.3 million in first-quarter sales, and 8% year-over-year growth compared to the first quarter of 2024, driven by underlying demand growth of 12%. We continue to advance our pipeline across multiple fronts, including our next generation programs for Jelmyto and UGN-102, as well as our emerging immuno-oncology initiatives.

談到 Jelmyto，我們報告第一季銷售額為 2,030 萬美元，與 2024 年第一季相比同比增長 8%，這得益於 12% 的潛在需求增長。我們繼續在多個方面推進我們的產品線，包括我們的下一代 Jelmyto 和 UGN-102 項目，以及我們新興的免疫腫瘤學計劃。

In February, we acquired product candidate, ICVB-1042, a next-generation investigational oncolytic virus from IconOVir, which we have assigned an internal code name of UGN-501. This is an important step in expanding our presence in immune-based therapies for urologic cancers. In parallel, we also have multiple strategic research collaborations in place aimed at leveraging our proprietary RTGel technology to enhance the delivery and effectiveness of various existing drugs.

今年 2 月，我們從 IconOVir 收購了候選產品 ICVB-1042，這是新一代研究性溶瘤病毒，我們已為其分配了內部代號 UGN-501。這是擴大我們在泌尿系統癌症免疫治療領域影響力的重要一步。同時，我們也進行了多項策略研究合作，旨在利用我們專有的 RTGel 技術來增強各種現有藥物的輸送和有效性。

UroGen is executing with focus and discipline. We remain committed to transforming the treatment landscape in UroGen oncology and are supported by a strong balance sheet with just over $200 million in cash, cash equivalents, and marketable securities as of March 31. We are investing in innovation with purpose, driven by the opportunity to make a meaningful impact on patients while delivering value to our shareholders.

UroGen 正在專注而有紀律地執行。我們始終致力於改變 UroGen 腫瘤學的治療格局，並擁有強大的資產負債表支持，截至 3 月 31 日，現金、現金等價物和有價證券略高於 2 億美元。我們有目的地投資創新，旨在為患者帶來有意義的影響，同時為股東創造價值。

I will turn the call over to Mark Schoenberg who will provide a clinical update. Mark?

我將把電話轉給馬克·勳伯格，他將提供臨床更新資訊。標記？

Thank you, Liz. I would like to echo what Liz shared regarding the strength of the clinical data supporting UGN-102. We've assembled a compelling and comprehensive clinical package that we believe demonstrates both the safety and efficacy of UGN-102. This gives us a high level of confidence as we approach the upcoming ODAC meeting and the PDUFA target date.

謝謝你，莉茲。我想附和 Liz 分享的關於支持 UGN-102 的臨床數據的強度。我們已經制定了一套令人信服且全面的臨床方案，我們相信這可以證明 UGN-102 的安全性和有效性。這讓我們在即將舉行的 ODAC 會議和 PDUFA 目標日期到來時充滿信心。

This year's AUA was an important event for UroGen. It provided a valuable platform to present the latest data on UGN-102 and Jelmyto with the broader urologic community. We were proud of the six abstracts accepted, reflecting the growing body of evidence behind our programs and the continued momentum of our clinical efforts.

今年的 AUA 對 UroGen 來說是一個重要的活動。它提供了一個寶貴的平台，向更廣泛的泌尿科界展示有關 UGN-102 和 Jelmyto 的最新數據。我們為被接受的六篇摘要感到自豪，這反映了我們計畫背後越來越多的證據和我們臨床努力的持續勢頭。

The highlight was the updated 18-month duration of response data from the Phase 3 ENVISION trial, which, as is the pivotal trial that is the foundation of our NDA for UGN-102. The data were featured in a podium presentation by Dr. Sandip Prasad, the principal investigator on the study. We are highly encouraged by the continued durability of response observed in the Phase 3 ENVISION trial.

亮點是來自第 3 階段 ENVISION 試驗的更新的 18 個月持續時間的反應數據，該試驗是我們針對 UGN-102 的 NDA 的基礎，也是關鍵試驗。該研究的首席研究員桑迪普·普拉薩德博士在演講中介紹了這些數據。我們對第三階段 ENVISION 試驗中觀察到的持續反應感到非常鼓舞。

Among patients who achieved a complete response, the duration of response at 18 months remains strong at 80.6% by Kaplan-Meier estimate. Mediann follow-up time has now extended to 18.7 months post three-month CR, up from 13.8 months at the previous data cut, and the median duration of response has still not been reached.

在完全緩解的患者中，根據 Kaplan-Meier 估計，18 個月時的緩解持續時間仍高達 80.6%。目前，中位追蹤時間已延長至三個月 CR 後的 18.7 個月，高於上次數據截斷時的 13.8 個月，但中位反應持續時間仍未達到。

For reference, the 12-month duration of response was previously reported at 82.5%. These results continue to reinforce the potential of UGN-102 to offer a durable non-surgical treatment approach for patients with recurrent low-grade intermediate risk NMIBC.

作為參考，先前報告的 12 個月回應持續時間為 82.5%。這些結果繼續強化了 UGN-102 為複發性低度中度風險 NMIBC 患者提供持久非手術治療方法的潛力。

We also presented a poster featuring patient reported outcomes from the three late stage UGN-102 studies: OPTIMA II, ATLAS, and ENVISION. These assessments use the EORTC 24-item quality of life questionnaire specific to NMIBC to evaluate the symptom burden and overall health status and function. The findings were consistent across trials, showing that treatment with UGN-102 did not negatively impact symptom burden, patient function, or quality of life, an important consideration for a novel therapy intended for use in routine clinical practice.

我們還展示了一張海報，其中展示了三項後期 UGN-102 研究（OPTIMA II、ATLAS 和 ENVISION）的患者報告結果。這些評估使用針對 NMIBC 的 EORTC 24 項生活品質問卷來評估症狀負擔和整體健康狀況和功能。各項試驗的結果一致，顯示使用 UGN-102 治療不會對症狀負擔、患者功能或生活品質產生負面影響，這對於旨在用於常規臨床實踐的新療法來說是一個重要的考慮因素。

At the AUA, we also shared data from the Phase 1 dose escalation study of UGN-301, our investigational anti-CTLA-4 antibody delivered via RTGel. These results were previously presented at SUO in late 2024 and continue to support a favorable safety profile for UGN-301, both as monotherapy and in combination with UGN-201, our TLR7 agonist, and with gemcitabine.

在 AUA，我們也分享了 UGN-301（我們透過 RTGel 遞送的試驗性抗 CTLA-4 抗體）的 1 期劑量遞增研究的數據。這些結果之前已於 2024 年底在 SUO 上公佈，並繼續支持 UGN-301 的良好安全性，無論是作為單一療法還是與 UGN-201（我們的 TLR7 激動劑）和吉西他濱聯合使用。

We observed clinical responses in both the monotherapy and combination arms with follow up on the combination arms ongoing to evaluate durability of response. We expect to share updated data from this program later this year. At that point, we anticipate being in a position to make a go no-go decision on whether to advance UGN-301 into Phase 2 development.

我們觀察了單一療法和聯合療法的臨床反應，並對聯合療法進行了持續跟踪，以評估反應的持久性。我們預計將在今年稍後分享該計劃的更新數據。屆時，我們預計將能夠做出是否將 UGN-301 推進到第 2 階段開發的決定。

Our next-generation candidates are actively advancing through development. Enrollment is ongoing in the Phase 3 UTOPIA trial, which is evaluating UGN-103 in patients with recurrent low-grade intermediate risk NMIBC.

我們的下一代候選人正在積極發展。目前，UTOPIA 第三階段試驗的招募工作正在進行中，該試驗旨在評估 UGN-103 對復發性低度中度風險 NMIBC 患者的作用。

UTOPIA is a single arm multicenter study modeled on the ENVISION trial. Efficacy will be measured by complete response of three months following treatment, with follow up focused on assessing durability. Enrollment is progressing ahead of plan, and we expect a complete enrollment by the middle of this year with top-line data anticipated in 2026. We are taking a similar approach with UGN-104, our next-generation formulation of Jelmyto, and expect to commence a single arm Phase 3 study by mid-year this year.

UTOPIA 是一項以 ENVISION 試驗為藍本的單組多中心研究。療效將透過治療後三個月的完全反應來衡量，後續追蹤將重點評估持久性。招生工作正在按計劃提前進行，我們預計今年年中將完成招生，並預計在 2026 年獲得頂線數據。我們對 Jelmyto 的下一代製劑 UGN-104 採取了類似的方法，並預計將於今年年中開始單臂 3 期研究。

Now, over to David Lin for a commercial update.

現在，請 David Lin 介紹商業更新。

Thank you, Mark. Our organization is now fully engaged in the final stages of pre-launch activities for UGN-102. The goal is to deliver a seamless and impactful launch that will ensure timely access for patients as soon as possible following approval.

謝謝你，馬克。我們的組織目前正全力投入 UGN-102 發射前活動的最後階段。目標是實現無縫且有影響力的啟動，以確保患者在獲得批准後儘快及時獲得治療。

Our team is highly energized, and we have the necessary experience, resources, and talent to drive adoption. Our clinical data supports our conviction that UGN-102 can be a transformative product that will change the standard of care in recurrent low grade, intermediate risk, non-muscle invasive bladder cancer.

我們的團隊充滿活力，擁有推動採用所需的經驗、資源和人才。我們的臨床數據支持我們的信念：UGN-102 可以成為一種變革性產品，它將改變復發性低級別、中度風險、非肌肉浸潤性膀胱癌的治療標準。

There are three ongoing activities that I want to highlight. First, our medical affairs team is leading an educational effort, engaging directly with urologists to highlight the unmet needs in recurrent low-grade intermediate risk NMIBC and ensure strong awareness of the clinical evidence supporting UGN-102.

我想強調三項正在進行的活動。首先，我們的醫療事務團隊正在領導一項教育工作，直接與泌尿科醫生合作，強調復發性低度中度風險 NMIBC 中未滿足的需求，並確保對支持 UGN-102 的臨床證據有很強的認識。

Second, we are scaling the organization to capture a larger opportunity in low-grade intermediate risk non-muscle invasive bladder cancer. This includes expanding our sales force from approximately 50 reps today to over 80 at launch. We are also building out the rest of the commercial infrastructure, including a robust patient support and distribution network.

其次，我們正在擴大組織規模，以抓住低度中度風險非肌肉層浸潤性膀胱癌的更大機會。這包括將我們的銷售隊伍從目前的約 50 名銷售代表擴大到推出時的 80 多名。我們也正在建造其餘的商業基礎設施，包括強大的患者支援和分銷網絡。

Third, we are executing a focused payer engagement strategy. Our market access and medical affairs teams are actively engaging with payers and formulary decision makers to communicate the clinical data supporting UGN-102.

第三，我們正在實施有針對性的付款人參與策略。我們的市場准入和醫療事務團隊正在積極與付款人和處方決策者合作，以傳達支持 UGN-102 的臨床數據。

We recognize that timely access can make a meaningful difference for patients, and our goal is to ensure coverage decisions are aligned closely with approval to support rapid adoption. If approved, UGN-102 will launch with a temporary miscellaneous J-code, and we anticipate securing a permanent product specific J-code by January 2026, which will be particularly important for broad adoption in the community setting.

我們認識到及時獲得治療可以為患者帶來重大改變，我們的目標是確保覆蓋決策與批准緊密結合，以支持快速採用。如果獲得批准，UGN-102 將以臨時雜項 J 代碼啟動，我們預計到 2026 年 1 月將獲得永久產品特定的 J 代碼，這對於在社區環境中的廣泛採用尤為重要。

In the first six to nine months, our strategy is to focus on a defined group of urologists who have demonstrated a willingness to adopt new therapies during the miscellaneous J-code period. We are also working to identify affiliate sites of care for these physicians to help facilitate access and to support product administration.

在最初的六到九個月裡，我們的策略是專注於一組特定的泌尿科醫生，他們在雜項 J 代碼期間表現出願意採用新療法。我們也致力於為這些醫生尋找附屬護理站點，以幫助促進訪問並支持產品管理。

Our experience with Jelmyto along with recent customer insights tells us that many providers prefer to initiate use of new therapies in the hospital outpatient setting where pharmacy budgets are often managed as separate cost centers. During this initial phase, our goal is to establish a strong foundation for UGN-102 adoption, positioning us for broader expansion once the permanent J-code is in place.

我們使用 Jelmyto 的經驗以及最近的客戶洞察告訴我們，許多供應商更喜歡在醫院門診環境中開始使用新療法，因為藥房預算通常作為單獨的成本中心進行管理。在此初始階段，我們的目標是為 UGN-102 的採用奠定堅實的基礎，以便在永久 J 程式碼到位後實現更廣泛的擴展。

As we enter the final phase of pre-launch execution, we do so with a confidence and momentum. The interest from the healthcare community in UGN-102 has been very encouraging, and we are implementing a robust strategy to support its introduction.

當我們進入發布前執行的最後階段時，我們充滿信心和動力。醫療保健界對 UGN-102 的興趣非常令人鼓舞，我們正在實施強有力的策略來支持它的推出。

Now turning to Jelmyto, first-quarter sales were $20.3 million with demand continuing to grow at a double-digit pace. We remain focused on high frequency engagement with our top performing accounts. As we scale our commercial organization in preparation for the anticipated launch of UGN-102, our expanded sales force will also support continued promotion of Jelmyto. This integrated effort will allow us to deliver broader utilization across both products while maximizing the impact of our commercial infrastructure.

現在來看看 Jelmyto，第一季的銷售額為 2,030 萬美元，需求繼續以兩位數的速度成長。我們將繼續專注於與表現最佳的帳戶進行高頻互動。隨著我們擴大商業組織規模，為即將推出的 UGN-102 做準備，我們擴大的銷售團隊也將支持 Jelmyto 的持續推廣。這項整合的努力將使我們能夠更廣泛地利用這兩種產品，同時最大限度地發揮我們商業基礎設施的影響力。

I will now turn the call over to Chris Degnan to review our financial results.

現在我將把電話轉給克里斯·德格南 (Chris Degnan) 來審查我們的財務結果。

Thank you, David. Jelmyto net product revenues were $20.3 million and $18.8 million for the three months ended March 31, 2025, and 2024 respectively. Year-over-year revenue growth of 8% was driven by underlying demand growth of 12%, partially offset by higher 340B chargebacks.

謝謝你，大衛。截至 2025 年 3 月 31 日及 2024 年 3 月 31 日的三個月，Jelmyto 淨產品收入分別為 2,030 萬美元及 1,880 萬美元。營收年增 8%，這得益於基礎需求成長 12%，但 340B 退款增加部分抵消了這一成長。

The gross to net rate for Jelmyto has stabilized in recent quarters, and we expect resulting headwinds on year-over-year growth to be less impactful going forward. R&D expenses for the first quarter of 2025 were $19.9 million, including non-cash share-based compensation expense of $0.6 million as compared to $15.5 million, including non-cash share-based compensation expense of $0.5 million for the same period in 2024.

Jelmyto 的毛淨利率在最近幾季已經趨於穩定，我們預計未來同比成長所面臨的阻力將不會太大。2025 年第一季的研發費用為 1,990 萬美元，其中包括 60 萬美元的非現金股權激勵費用，而 2024 年同期的研發費用為 1,550 萬美元，其中包括 50 萬美元的非現金股權激勵費用。

The year-over-year increase in R&D expenses was primarily driven by the equity consideration issued to IconOVir for the acquisition of UGN-501, which was expense in the quarter, higher manufacturing costs, and costs associated with the Phase 3 UTOPIA trial for UGN-103, partially offset by lower clinical trial costs and regulatory expenses in connection with UGN-102.

研發費用年增主要由於向 IconOVir 發行的用於收購 UGN-501 的股權對價（該對價為本季度費用）、更高的製造成本以及與 UGN-103 的 3 期 UTOPIA 試驗相關的成本，但與 UGN-102 相關的臨床試驗成本和監管費用的降低部分抵消了這一增長。

Selling, general, and administrative expenses for the first quarter of 2025 were $35 million, including non-cash share-based compensation expense of $2.5 million. This compares to $27.3 million, including non-cash share-based compensation expense of $2.2 million for the same period in 2024. The year-over-year increase was primarily a result of UGN-102 commercial preparation activities.

2025 年第一季的銷售、一般和行政費用為 3,500 萬美元，其中包括 250 萬美元的非現金股權激勵費用。相較之下，2024 年同期為 2,730 萬美元，其中包括 220 萬美元的非現金股權激勵費用。年比成長主要源自於 UGN-102 商業化準備活動。

We reported non-cash financing expense related to the pre-paid forward obligation to RTW Investments of $4.6 million in the first quarter of 2025, compared to $5.7 million in the same period in 2024. Interest expense related to the $125 million term loan facility with funds managed by Pharmakon Advisors was $4.1 million in the first quarter of 2025, compared to $2.4 million in the same period in 2024. The increase was primarily driven by interest expense related to the $25 million third tranche of the loan that was funded in September 2024.

我們報告稱，2025 年第一季與預付給 RTW Investments 的遠期債務相關的非現金融資費用為 460 萬美元，而 2024 年同期為 570 萬美元。2025 年第一季度，由 Pharmakon Advisors 管理的基金提供的 1.25 億美元定期貸款相關的利息支出為 410 萬美元，而 2024 年同期為 240 萬美元。成長的主要原因是 2024 年 9 月發放的 2,500 萬美元第三筆貸款的利息支出。

Net loss was $43.8 million or $0.92 per basic and diluted share in the first quarter of 2025 compared with a net loss of $32.3 million or $0.87 per basic and diluted share in the same period in 2024. As of March 30, 2025, cash, cash equivalents, and marketable securities totaled $200.4 million.

2025 年第一季淨虧損為 4,380 萬美元，即每股基本虧損和稀釋虧損均為 0.92 美元，而 2024 年同期淨虧損為 3,230 萬美元，即每股基本虧損和稀釋虧損均為 0.87 美元。截至 2025 年 3 月 30 日，現金、現金等價物及有價證券總額為 2.004 億美元。

Turning now to guidance, we last provided financial guidance with our year-end 2024 results in March, and that guidance remains unchanged. We continue to expect full-year 2025 net product revenues from Jelmyto to be in the range of $94 million to $98 million. And this implies a year-over-year growth rate of approximately 8% to 12% over the $87.4 million in demand driven Jelmyto sales in 2024, which excludes the $3 million in CREATES Act sales reported in 2024. Guidance on full-year 2025 operating expenses is also unchanged. It is expected to be in the range of $215 to $225 million, including non-cash share-based compensation expense of $11 million to $14 million.

現在談到指導，我們上次在 3 月提供了 2024 年年終業績的財務指導，並且該指導保持不變。我們繼續預計 Jelmyto 2025 年全年淨產品收入將在 9,400 萬美元至 9,800 萬美元之間。這意味著 2024 年 Jelmyto 的銷售額將比需求驅動的 8,740 萬美元成長約 8% 至 12%，其中還不包括 2024 年報告的 300 萬美元的 CREATES 法案銷售額。2025 年全年營運費用指引也維持不變。預計在 2.15 億美元至 2.25 億美元之間，其中包括 1,100 萬美元至 1,400 萬美元的非現金股權激勵費用。

We're now ready to open the call for questions. Operator?

我們現在準備開始回答問題。操作員？

(Operator Instructions) Tara Bancroft, TD Cowen

（操作員指示）Tara Bancroft，TD Cowen

So I was hoping that, in light of today's events, can you possibly give us the breakdown of Medicare and Medicaid exposure that you anticipate for UGN-102 and currently for Jelmyto? Really how you think pricing dynamics could play out? Thanks so much.

因此，我希望，鑑於今天的事件，您能否向我們提供您預計的 UGN-102 和目前 Jelmyto 的醫療保險和醫療補助風險明細？您真的認為定價動態會如何發揮作用嗎？非常感謝。

Yeah, hi Tara. It's Liz, and I'll ask David to comment on that in a moment when I think about the percent of our business, but keep in mind that we're only a US-focused company. So we don't have any issues from a best price perspective, favored nation perspective, outside. So we don't have any risk associated with that.

是的，你好，塔拉。我是 Liz，當我考慮我們業務的百分比時，我會請 David 對此發表評論，但請記住，我們只是一家以美國為中心的公司。因此，從最優價格角度、最惠國角度來看，我們沒有遇到任何問題。所以我們不會面臨任何與此相關的風險。

But David, can you just talk about the Medicare?

但是大衛，你能談談醫療保險嗎？

Yeah, hi Tara. It's David. We anticipate that the Medicare population will comprise about 70% of our business, very consistent with the overall patient demographic in low-grade intermediate risk NMIBC. And as we think about launch, our principal priority will also be to drive reimbursement confidence with the providers who treat these patients. Thanks very much for the question. I appreciate it.

是的，你好，塔拉。是戴維。我們預計，醫療保險人口將占我們業務的 70% 左右，這與低中度風險 NMIBC 的整體患者人口結構非常一致。當我們考慮推出產品時，我們的首要任務也是增強治療這些患者的醫療服務提供者對報銷的信心。非常感謝您的提問。我很感激。

Kelsey Goodwin, Guggenheim.

凱爾西·古德溫，古根漢美術館。

Congrats on getting one step closer to this exciting approval. In terms of preparing for the ODAC, I guess how are you preparing and where do you expect the most pushback from the panel and what do you think are your strongest arguments there? Thank you.

恭喜您距離這項令人興奮的批准又近了一步。在準備 ODAC 方面，我想您是如何準備的，您預計專家小組在哪些方面會提出最大的反對意見，您認為您最有力的論點是什麼？謝謝。

Yeah, hi Kelsey. It's Liz. Thanks for the question. We have been preparing for quite a few months, frankly, since last fall. We have had our mock ODAC panels. We've had several now. We've had them with a distinguished guest that are medical oncologists, that had been on ODAC, statisticians. So what we've tried to do is really have a situation where we are -- it's just like if we were at the ODAC. So a lot of the members have been ODA members in the past. Some have even been leaders of the ODAC. So we feel like we've really put ourselves through the paces and we will continue to do so up until the day of the ODAC.

是的，你好，凱爾西。是莉茲。謝謝你的提問。坦白說，自去年秋天以來，我們已經準備了好幾個月了。我們已經有類比的 ODAC 面板。現在我們已經有好幾個了。我們邀請了一位尊貴的嘉賓，他們是腫瘤內科醫生、曾在 ODAC 任職的統計學家。因此，我們嘗試做的是真正實現我們所處的情形 — — 就像我們在 ODAC 一樣。很多成員過去都曾是 ODA 成員。有些人甚至還擔任過 ODAC 的領導。因此，我們感覺我們已經真正地完成了各項任務，並且我們會繼續這樣做直到 ODAC 到來的那一天。

I'm going to give my perspective and then I'm going to turn it over to Mark and ask him to comment as well. So my perspective is that the biggest question is around the fact that ENVISION is a single arm study. It's the basis for an approval, for our approval and how did the results -- how do you compare those results? How do you put them in context?

我將給出我的觀點，然後將其交給馬克並請他也發表評論。因此我的觀點是，最大的問題在於 ENVISION 是否是單臂研究。這是批准的基礎，也是我們批准的基礎，結果如何——您如何比較這些結果？您如何將它們放在上下文中？

So in other words, it sounds great, right? You're 80%, sounds great and you're 80%, it sounds great, but I don't have anything to compare it to. And unfortunately with the exception of our own study, ATLAS, and we can talk about sort of the challenges with ATLAS, but except for our own study, ATLAS, there hasn't been a lot of peer-reviewed studies or data published in this specific patient population. The low-grade intermediate risk, non-muscle invasive bladder cancer. So it's really about putting it in context and how do you take a single arm study and put the data in context.

換句話說，聽起來很棒，對吧？你有 80%，聽起來很棒，你有 80%，聽起來很棒，但我沒有什麼可以比較。不幸的是，除了我們自己的研究 ATLAS 之外，我們可以談論 ATLAS 面臨的挑戰，但除了我們自己的研究 ATLAS 之外，還沒有很多針對這一特定患者群體的同行評審研究或發表的數據。低度中度風險、非肌肉層浸潤性膀胱癌。因此，這實際上是關於將其置於上下文中，以及如何進行單臂研究並將數據置於上下文中。

Having said that, we obviously feel really good about it, and I can tell you that in our mock ODAC, we've gotten a positive vote. That's where it comes down to. And we are not dealing with people who are being easy on us, trust me, and we give our presentation, we give an FDA presentation. We give them a briefing book. So again, we try to simulate an ODAC meeting. And ultimately, in those situations we have come out with a positive vote.

話雖如此，我們顯然對此感到非常高興，我可以告訴你，在我們的模擬 ODAC 中，我們獲得了積極的投票。這就是事情的真相。我們不會與那些對我們寬容的人打交道，相信我，我們會做我們的演示，我們會向 FDA 做演示。我們給了他們一本簡報。因此，我們再次嘗試模擬 ODAC 會議。最終，在這種情況下，我們投了贊成票。

But Mark, will you comment, one, at the second part of Kelsey's question, which is why we feel good about where we are and what our arguments are, and any other comments you have around you think potential challenges.

但是馬克，你能否評論一下，第一，凱爾西問題的第二部分，這就是為什麼我們對自己的現狀和論點感到滿意，以及你認為潛在的挑戰的任何其他評論。

Sure. Thanks, Liz. So we've got, for starters, and actually this came out of extensive conversations with the FDA in preparation for this meeting, are the fact that, as everybody listening in probably remembers, we are working in a recurrent population. So the population we're treating and talking about in this meeting are patients who've already had surgery and who have recurred.

當然。謝謝，莉茲。首先，事實上，這是在為這次會議做準備時與 FDA 進行廣泛對話後得出的結論，事實是，正如所有聽眾可能記得的那樣，我們正在針對複發人群開展工作。因此，我們在這次會議上治療和討論的人群是已經接受手術並且病情復發的患者。

So the agency identified this as the greatest unmet medical need, and we completely agree. These are patients who have failed the standard of care and then they received treatment, primary treatment, as Liz said, with UGN-102 and achieve an 80% complete response rate [treatment] profile, and 18 months later have an over 80% durability of that complete response. These are, from a clinical perspective, remarkable results.

因此，該機構將此認定為最大的未滿足的醫療需求，我們完全同意。這些患者未能達到標準治療，然後他們接受了治療，主要治療，正如 Liz 所說，使用 UGN-102 並實現了 80% 的完全緩解率 [治療]，並且 18 個月後完全緩解的持久性超過 80%。從臨床角度來看，這些都是顯著的成果。

I think one of our strongest arguments is that we have great results in a population of patients who didn't do well with the standard of care. So that's number one, and I think our experience with the mock panels, as Liz has pointed out, has been favorable precisely because of the encouraging safety profile and the sort of remarkable results of the ENVISION trial and the support of data from ATLAS and OPTIMA.

我認為我們最有力的論點之一是，我們在那些沒有很好地接受標準治療的患者群體中取得了巨大的成果。這是第一點，我認為我們在模擬小組方面的經驗是有利的，正如 Liz 指出的那樣，正是因為令人鼓舞的安全性和 ENVISION 試驗的顯著成果以及 ATLAS 和 OPTIMA 的數據支持。

So I think Liz has articulated where we think the conversation is going to go, the importance of the -- how to interpret the single arm trial. We have done -- we've scoured the literature and we've got all the supporting data you can possibly have for the single arm trial, the meaningfulness of ATLAS, the safety profile, and then the unmet medical need and how to articulate that. And we believe those are going to be the areas of conversation. And as Liz pointed out, we have been preparing for months to answer those questions.

所以我認為 Liz 已經清楚地表達了我們認為對話將會走向的方向，即如何解讀單臂試驗的重要性。我們已經完成了——我們已經仔細查閱了文獻，並且獲得了所有可能的支持數據，包括單臂試驗、ATLAS 的意義、安全性概況，以及未滿足的醫療需求以及如何表達這些需求。我們相信這些將成為討論的領域。正如利茲指出的那樣，我們已經準備了好幾個月來回答這些問題。

You know what, one thing I'll just add as well is we have two very strong KOLs that are joining us. They're part of our presentation. They'll be giving a presentation and they'll be also answering some of the questions. They have a lot of credibility. They're well known. Again, a lot of credibility.

你知道嗎，我還要補充一點，我們有兩位非常強大的 KOL 加入我們。它們是我們演示的一部分。他們將進行演示並回答一些問題。他們很有信譽。他們很有名。再次強調，這非常具有可信度。

So when questions do get asked, and they are the ones who are standing up answering it, it's very helpful for us. And so I'm really, really pleased with the two KOLs that are joining us for our presentation during the ODAC.

因此，當有人提出問題時，他們站出來回答，這對我們非常有幫助。因此，我真的非常高興有兩位 KOL 在 ODAC 期間加入我們的演講。

Leland Gershell, Oppenheimer.

利蘭·格謝爾，奧本海默。

Two from us. First, I just want to ask, in the FDA review process we're about a month away from the PDUFA date. Just wondering, given the [adcom] is still pending, just want to ask if you could share any color on your recent directions. Presumably you've had your late cycle meeting. Have you had the chance to discuss proposed labeling? Just want to ask if you're able to share any details there.

我們有兩個。首先，我只想問一下，在 FDA 審查過程中，我們距離 PDUFA 日期還有大約一個月的時間。只是想知道，鑑於[adcom]仍在等待中，只是想問您是否可以分享您最近的指示。假設你已經參加了後期週期會議。您有機會討論擬議的標籤嗎？只是想問一下您是否可以分享一些詳細資訊。

And then the second question I guess for Mark or David. You want to choose position to become, as you say, a foundational therapy and low-grade intermediate risk at the same time. Urologist adoption may begin with a certain type of patient profile. Just wondering what you see as kind of that most likely patient profile for urologists to start using 102 out of the 60,000 patients. What fraction might that be. Thank you.

第二個問題我想問的是馬克或大衛。正如您所說，您想要選擇的職位同時成為基礎治療和低級中級風險。泌尿科醫師的採用可能始於某種類型的病患概況。我只是想知道，您認為泌尿科醫生最有可能使用哪一種患者資料，從而在 60,000 名患者中選取 102 名。那可能是多大比例？謝謝。

Sure. So thanks Leland, for the question. I would say we feel really good about kind of where we are with the FDA. We've had continuous interactions with them. All them asking questions. That's right you can tell from where we are and the questions that they are asking where they are in the review.

當然。感謝 Leland 提出這個問題。我想說，我們對與 FDA 的合作感到非常滿意。我們一直與他們保持互動。他們都在問問題。沒錯，您可以從我們所處的位置以及他們在評論中提出的問題中看出他們所處的位置。

And so we have no concerns about the PDUFA date, and we don't want to get into a very specific conversation about our conversations with the FDA, but suffice it to say that it's very clear that they're at the end of their review, right, and we are in a position in a -- we'll be in a really good position post ODAC for that to move very quickly. So, again, no concerns there.

因此，我們並不擔心 PDUFA 日期，我們也不想就與 FDA 的對話進行非常具體的討論，但我可以說，很明顯他們的審查已經結束了，對吧，我們處於 - 我們將在 ODAC 之後處於非常有利的位置，以便快速推進。所以，再說一次，不用擔心。

I think the conversation -- we had the mid-cycle review around the label and switching it to the recurrent patient population has really made things -- really simplified things both from our presentation as well as any of the labeling or discussions with the FDA. And so I think that has helped a tremendous amount.

我認為，我們圍繞標籤進行了中期審查，並將其轉向復發患者群體，這確實使事情變得簡單，無論是從我們的演示還是從任何標籤或與 FDA 的討論來看都是如此。所以我認為這有很大幫助。

We did not have a late cycle meeting and we're not going to have a late cycle meeting. So when we met with them, I don't know if you recall, the mid-cycle review meeting was really late, later than it was supposed to be. There hasn't been a need for a late cycle meeting and they informed us at that time that there would not be a late cycle review. So we won't have that.

我們沒有舉行過後期週期會議，也不會舉行後期週期會議。所以當我們與他們會面時，我不知道你是否還記得，中期審查會議真的很晚，比預計的時間要晚。沒有必要舉行後期週期會議，他們當時通知我們不會進行後期週期審查。所以我們不會有這樣的情況。

I'm going to ask David to talk to you about the populations in which we expect will be the kind of low-hanging fruit and the first patients that physicians will use this on. So David?

我將請大衛向您介紹我們預期會成為這種療法最容易獲得療效的人群以及醫生將對其使用這種療法的首批患者。那麼大衛？

Yeah, thanks, Liz, and thanks, Leland, for the question. Coming out of the gates, first I'll just share we've done extensive market research with physicians, and suffice it to say they are really pleased with the clinical data we've shared on UGN-102. They find it very compelling and they see a need for it in their practices because of the multiple recurrences that their patients experience.

是的，謝謝 Liz 和 Leland 提出這個問題。首先，我要說的是，我們已經與醫生進行了廣泛的市場調查，可以說他們對我們分享的 UGN-102 臨床數據感到非常滿意。他們發現這種方法非常引人注目，並且由於患者會經歷多次復發，因此他們認為這種方法在實踐中是必要的。

Coming out of the gates, there's really three segments that we've teased out in talking to physicians. First, it's those patients who have multiple recurrences. As you know, 70% of the patients have multiple recurrences. The second group are patients who are early recurrers. And then finally, there's a small set of patients that are just not able to have surgery for one reason or the other.

從一開始，我們在與醫生的交談中就明確了三個部分。第一類是多次復發的患者。大家知道，70%的患者會出現多次復發。第二組是早期復發的患者。最後，還有一小部分患者因為這樣或那樣的原因無法接受手術。

It could be because of polypharmacy, or they can't handle anesthesia, but those are the primary patient populations that we think are very, very ripe for uptake when we launch UGN-102. Importantly though, one of the things around UGN-102 is that it fits into the workflow of the urologist office. So, we're very pleased that with minimal training, we can help onboard UGN-102 into practice and make it a seamless experience. Appreciate the question.

這可能是因為多種藥物合併使用，或者他們無法接受麻醉，但我們認為，當我們推出 UGN-102 時，這些是最適合接受治療的主要患者群。但重要的是，UGN-102 的特點之一是它適合泌尿科醫生辦公室的工作流程。因此，我們很高興，只需最少的培訓，我們就可以幫助將 UGN-102 付諸實踐，並使其成為一種無縫體驗。感謝你的提問。

Raghuram Selvaraju, H.C. Wainwright.

拉古拉姆‧塞爾瓦拉朱 (Raghuram Selvaraju)，H.C.溫賴特。

Just in furtherance of what has already been asked on this call regarding the ODAC meeting, I was wondering if you could comment on two aspects in particular. Firstly, to what extent there has been communication regarding the purpose of the ODAC that pertains specifically to the lack of specific precedent for products being approved in low-grade intermediate risk NMIBC. If you expect that to be a meaningful topic of discussion at the ODAC meeting with regard to UGN-102.

為了進一步回答本次電話會議中關於 ODAC 會議提出的問題，我想知道您是否可以特別就兩個方面發表評論。首先，關於 ODAC 的目的，已經進行了多大程度的溝通，具體涉及缺乏在低級中等風險 NMIBC 中批准產品的具體先例。如果您希望這成為 ODAC 會議上關於 UGN-102 的一個有意義的討論主題。

And then secondly, to what extent do you expect the precedent case of Jelmyto to aid and facilitate the discussion around UGN-102 to effectively put the committee in a position to be familiar with how UGN-102 works, what its benefit level is, and effectively facilitate potentially direction towards a favorable panel vote because of this precedent example that utilizes the same active ingredient and the same fundamental principle of delivery. Thank you.

其次，您認為 Jelmyto 的先例案例在多大程度上有助於和促進圍繞 UGN-102 的討論，從而使委員會能夠熟悉 UGN-102 的工作原理、其益處水平，並有效地促進潛在的有利小組投票，因為這個先例使用了相同的活性成分和相同的基本傳遞原理。謝謝。

Okay, great points, Ram. And Mark, why don't you comment and then I'll add some color as well.

好的，Ram，你的觀點很棒。馬克，你為什麼不評論一下，然後我也會添加一些顏色。

Yeah, sure. Thanks for the insightful comments. With respect to the first question, I think it's implicit in your observation that this is a (technical difficulty) the therapy that we're presenting to the agency, that the FDA would like a public conversation about what the meaningfulness of this approach to this disease would be in the setting of an expert panel.

是的，當然。感謝您的深刻評論。關於第一個問題，我認為您的觀察中隱含著我們向 FDA 提出的治療方法存在（技術難題），FDA 希望在專家小組的背景下就這種方法對這種疾病的意義進行公開討論。

So we've always thought that that was part of the reason for the ODAC that the FDA has promised us for a very long time would be the case in the approval process for UGN-102 because it is a different way of treating patients, because it is a primary therapy, because it does (technical difficulty) with an option that does not involve surgery. We think a robust discussion about that is going to take place within the context of what Liz was referring to earlier, namely the [implementation] trial. That said, we believe we have ample data to support the meaningfulness of the clinical outcome we've observed in our trials, as well as the benefit to patients of having an option.

因此，我們一直認為，這是 FDA 長期以來向我們承諾的 ODAC 的原因之一，因為它是一種治療患者的不同方式，因為它是一種主要療法，因為它確實存在（技術難度）不涉及手術的選擇。我們認為，關於這一問題的深入討論將在 Liz 之前提到的背景下進行，即[實施]試驗。話雖如此，我們相信我們有充足的數據來支持我們在試驗中觀察到的臨床結果的意義，以及患者擁有選擇權的益處。

It is interesting to remember that this would be the only urologic cancer, to my knowledge, where patients really don't have a choice of therapies. They only have one therapy. So UGN-102 would present to this population with the type of option that many patients with urologic cancers have in other settings, whether they be prostate or kidney cancer or more advanced forms of bladder cancer. So we think we can address that, and we think that is going to be a part of the conversation overall.

有趣的是，據我所知，這是唯一患者沒有治療選擇的泌尿系統癌症。他們只有一種療法。因此，UGN-102 將為這類族群提供許多泌尿系統癌症患者在其他情況下可以選擇的治療方案，無論他們是前列腺癌、腎癌或更晚期的膀胱癌。因此我們認為我們可以解決這個問題，而且我們認為這將成為整個對話的一部分。

And then, sorry, the second question is?

那麼，抱歉，第二個問題是？

It was around the precedent case of Jelmyto.

這是圍繞 Jelmyto 的先例案件。

Oh, I'm sorry, yes. And so we actually do, as part of our presentation, we actually remind or acquaint the committee with Jelmyto and its history and the fact that it has informative with respect to thinking about upper tract disease.

噢，對不起，是的。因此，作為演示的一部分，我們實際上提醒或讓委員會了解 Jelmyto 及其歷史，以及它在思考上泌尿道疾病方面具有資訊量的事實。

So we have in fact put forward a segment of our presentation to specifically familiarize the advisory committee with Jelmyto and its history and the similarities with respect to approach and active ingredient to reassure them that this is in fact a follow on conceptually to what we've already had approved by the FDA.

因此，我們實際上提出了一個演示片段，專門讓諮詢委員會熟悉 Jelmyto 及其歷史以及方法和活性成分方面的相似之處，以向他們保證，這實際上是對我們已經獲得 FDA 批准的概念的延續。

But Liz may want to comment as well.

但 Liz 可能也想發表評論。

Yeah, no, I think that's great. I think the only other comment I'll make is one of the reasons we want to do that is because the ODAC panel will be made up mostly of medical oncologists, right? So they will have adhoc members, urologists, but because medical oncologists are not familiar with, either this disease or our treatments, we felt like it was important. And Mark put that into the presentation so.

是的，不，我認為這很棒。我認為我要說的唯一其他評論是，我們想要這樣做的原因之一是 ODAC 小組主要由腫瘤內科醫生組成，對嗎？因此他們會有臨時成員、泌尿科醫生，但由於腫瘤內科醫生不熟悉這種疾病或我們的治療方法，我們覺得這很重要。馬克就把這一點融入了演講中。

But great questions because those are exactly why we're actually going to the ODAC and exactly the right questions to ask. So thank you, Ram.

但這些問題很棒，因為這正是我們前往 ODAC 的原因，也是我們應該問的正確問題。所以謝謝你，拉姆。

Just one quick follow up if I may. Can you give us an update on the current status of the UGN-103 clinical development program and when you expect to report the next material update on the progress of that trial? Thank you.

如果可以的話，我只想快速跟進。您能否向我們介紹一下 UGN-103 臨床開發計畫的當前狀態，以及您預計何時報告該試驗進展的下一個重要更新？謝謝。

Yeah, we're almost fully enrolled and so we'll be able to share that soon. So as soon as over the next couple of months, as soon as we're finished enrollment, we'll be able to provide. The fact that we've finished enrollment, we'll be by the end of the summer, and then we'll go from there as far as the timing for the CR. All patients at CR and then all patients, and durability so those would be the next steps. You'll start to see data in '26.

是的，我們的招生人數已經基本滿了，所以我們很快就能分享這個消息。因此，在接下來的幾個月內，一旦我們完成招生，我們就能提供服務。事實上，我們已經完成了招生工作，我們將在夏季結束前完成，然後我們將從那裡開始確定 CR 的時間。所有患者均處於 CR，然後是所有患者，以及耐久性，因此這些將是下一步。您將在 26 年開始看到數據。

Paul Choi, Goldman Sachs

高盛集團 Paul Choi

I had a question about how maybe you're thinking about the market for 102 here. David provided some nice color on the population size and opportunity as well as expected greater patient population in the community setting.

我有一個問題，關於您可能如何考慮這裡的 102 市場。David 對人口規模和機會給出了一些很好的解釋，並預計社區環境中的患者人數會更多。

But I was just curious, given the trend you're seeing with 340B utilization for Jelmyto, is that something structural you also expect for the 102 market? Just some color there would be helpful and just thinking about hospital pharmacy pricing.

但我只是好奇，鑑於您看到的 Jelmyto 340B 利用率趨勢，您是否也預期 102 市場也會出現這種結構性趨勢？只要有一些顏色就會有幫助，只要考慮醫院藥局的定價。

And then my second question is for Mark, just in terms of your earlier comments on thinking about advancing 301, sort of maybe can you provide us how you're thinking about maybe the bar for the go or no-go decision for that program for the CTLA-4. Thank you very much.

然後我的第二個問題是針對馬克的，就您之前關於考慮推進 301 的評論而言，也許您能否向我們提供一下，您是如何考慮對 CTLA-4 計劃進行通過或不通過的決定標準。非常感謝。

Yeah, the three -- the good news for UGN-102 is the 340B we expect the discounts not to be as great as they are for Jelmyto because, to your point, we do expect that ultimately, not at the beginning, but ultimately, that it will be a sort of 70% of the business will be in the community based off the comments that David made around that.

是的，這三個 - 對於 UGN-102 來說，好消息是 340B，我們預計折扣不會像 Jelmyto 那麼大，因為正如你所說，我們確實預計最終，不是一開始，而是最終，根據 David 對此發表的評論，70% 的業務將在社區中。

So we're really hopeful and expecting that the 340B would be will be much less in in UGN-102 than it is for -- but we have been more conservative with our assumptions also as we go into UGN-102 because as you know, Jelmyto, that's been one of the headwinds we've shared because our data, our actual patient demand, has been much greater but we've seen some real challenges with the 340B.

因此，我們真的充滿希望並期望 340B 在 UGN-102 中的應用會比現在少得多 - 但當我們進入 UGN-102 時，我們的假設也更加保守，因為正如你所知，Jelmyto，這是我們分享的阻力之一，因為我們的數據，我們實際的患者需求要大得多，但我們已經看到 340B 面臨的一些真正的挑戰。

Mark, do you want to talk about Paul's second question?

馬克，你想談談保羅的第二個問題嗎？

Sure. Thanks, Paul. So as the audience will undoubtedly remember, 301 is our Intravesical immunotherapy approach to high grade disease, and it's a Phase 1 study, both as Liz said earlier, a monotherapy study and in combination with our TLR7 agonist [in 201] as well as with the gemcitabine. So as a Phase 1 study, it's primarily focused on tolerability and safety and not on efficacy.

當然。謝謝，保羅。因此，觀眾無疑會記得，301 是我們針對高級別疾病的膀胱內免疫治療方法，這是一項 1 期研究，正如 Liz 之前所說，它既是一項單一療法研究，也是與我們的 TLR7 激動劑 [在 201 中] 以及吉西他濱聯合使用的研究。因此，作為第一階段的研究，它主要關注耐受性和安全性，而不是功效。

That said, we have said publicly that we've seen a few responses, and we are very interested in these responses for obvious reasons. But in a Phase 1 study, it would be a little bit difficult to give you a numeric bar that we are expecting to be meaningful. What we are doing now is following patients to look for the durability of these responses.

話雖如此，我們已經公開表示，我們已經看到了一些回應，並且出於顯而易見的原因，我們對這些回應非常感興趣。但在第一階段的研究中，給出一個我們期望有意義的數字標準會有點困難。我們現在正在做的是追蹤患者，尋找這些反應的持久性。

And later this year, we anticipate presenting those data probably at the SUO at the end of the year, at which time we'll have had a chance to assess both the durability and quality of those responses in the combination arms. And at that point, we'll probably be in a position to make a go, no-go decision about a trial that would be able to answer the question that you're asking, namely what numeric bar would be meaningful for us in comparison to the other assets out there currently addressing this population.

今年晚些時候，我們預計將在年底的 SUO 上展示這些數據，屆時我們將有機會評估聯合治療方案中這些反應的持久性和品質。到那時，我們可能就能夠對試驗做出進行或不進行的決定，該決定將能夠回答您所問的問題，即與目前針對該人群的其他資產相比，哪個數字標準對我們來說有意義。

So I guess my short answer is it's probably premature for me to give you a number, but we are encouraged that we're seeing responses. We're tracking those, and we'll be able to talk later this year about what would constitute a signal to us for go, no-go for Phase 2, and Liz may want to comment as well.

所以我想我的簡短回答是，現在給你一個數字可能還為時過早，但我們很高興看到了回應。我們正在追蹤這些情況，並將在今年稍後討論什麼將構成我們進入第二階段或不進入第二階段的訊號，Liz 可能也想發表評論。

Yeah. My comments are really around it's very clear that the bar for efficacy and safety but particularly for efficacy is definitely higher than it was when we started the program given the results that you've seen. I do think there's still a lot of opportunity in high-grade disease for several reasons.

是的。我的評論實際上是圍繞著這一點，很明顯，從您所看到的結果來看，功效和安全性的標準，特別是功效的標準肯定比我們開始該計劃時要高。我確實認為，由於多種原因，高級別疾病仍然有很多機會。

One, these patients are not cured unfortunately, so they continue to see recurrences and so they need more treatments because it's not -- these aren't cures. Two, I think all of the current programs that are out there that are seeing the higher complete response rates and durability are because you're seeing continuous dosing.

首先，不幸的是，這些病人並沒有被治愈，所以他們會不斷復發，所以他們需要更多的治療，因為這不是——這些都不是治愈方法。二，我認為目前所有現有的計畫之所以能夠獲得更高的完全緩解率和持久性，是因為採用了持續給藥。

So, almost all of them are requiring re-inductions, continuous dosing, and so I still think that there's opportunity. If you can have a situation like we have with UGN-102 and intermediate risk, we're actually -- we're given six weeks and then the patients don't have treatment. And I think that's very important for patients to not only be recurrence free but also treatment free.

因此，幾乎所有這些都需要重新誘導、持續給藥，所以我仍然認為有機會。如果出現像我們遇到的 UGN-102 和中度風險的情況，我們實際上會給患者六週的時間，然後患者就不會接受治療。我認為這對患者來說非常重要，不僅可以避免復發，而且無需治療。

And so being able to have a more simple administration and administration schedule, although I do believe that in high grade, you're more likely to need to have some sort of maintenance or retreatment, I do think that there's still a lot of opportunity. But we will be very diligent and we'll be very critical before we launch into a very expensive Phase 2 or Phase 3 study with UGN-301.

因此，能夠擁有更簡單的管理和管理計劃，儘管我確實相信在高級別中，您更有可能需要進行某種維護或再治療，但我確實認為仍然有很多機會。但在啟動 UGN-301 的非常昂貴的第 2 階段或第 3 階段研究之前，我們會非常勤勉，並且會非常謹慎。

And also I just want to mention 501, right? We're very excited about that, so we will have to make a determination for 301 and 501 about where we move and when we move and how we move. And so those are all things that we're working on right now. And so as Mark said, a little too early to comment except to understand that the bar is definitely higher, and we believe that both of those approaches actually can easily reach that bar and potentially simplify the administration.

而且我只想提一下 501，對嗎？我們對此感到非常興奮，因此我們必須為 301 和 501 做出決定，確定我們將搬到哪裡、何時搬到以及如何搬到那裡。這些都是我們現在正在努力的事情。正如馬克所說，現在評論還為時過早，但要明白標準肯定更高，我們相信這兩種方法實際上都可以輕鬆達到這個標準，並可能簡化管理。

George Farmer, Scotiabank.

加拿大豐業銀行的喬治法默。

A few for me. Can you confirm that the FDA representatives you've been speaking with are still employed with the agency? That's number one.

對我來說有幾個。您能否確認與您交談過的 FDA 代表仍在該機構任職？這是第一點。

Number two, this 18-month data that you presented at AUA, is that going to be included during the discussion at ODAC? And number three, do you see a need or are you planning for any sort of direct-to-consumer advertising campaign? Thanks.

第二，您在 AUA 上展示的這 18 個月的數據是否會被納入 ODAC 的討論中？第三，您是否認為有必要或是否計劃開展任何形式的直接面向消費者的廣告活動？謝謝。

Great question, George. Thank you very much. The FDA team, and knock on wood, is still in place, the same team that have been there since Jelmyto, frankly. So hopefully, over the next few weeks, that team stays in place, but there's still the same people that we've been working with all along, and we are -- we can confirm that they're still employed in there.

喬治，問得好。非常感謝。FDA 團隊仍在原地，坦白說，自 Jelmyto 以來就一直在那裡的團隊是同一支。因此，希望在接下來的幾週內，團隊能夠繼續留任，但與我們一直合作的人員仍然是相同的，我們可以確認他們仍在那裡工作。

The 18-month data has already been incorporated. The FDA received that before it was even public. So all that data is with the FDA and has been and is part of all of our discussions and presentation with them.

18 個月的數據已經納入。FDA 在消息公開之前就收到了它。因此，所有這些數據都屬於 FDA，並且一直是我們與他們進行的所有討論和演示的一部分。

And I'm sorry I forgot the last question. Oh, direct to consumer. I wouldn't expect broad-based direct to consumer, but we will absolutely have programs that engage the patient. It's really important. I think you've heard several times we talked about the fact that 90% of patients prefer UGN-102 to TURBT. These are patients that have had a TURBT, and they've had UGN-102. And 90% of them prefer UGN-102.

很抱歉我忘了最後一個問題。哦，直接面向消費者。我不會期望廣泛的直接面向消費者，但我們絕對會有讓患者參與的計劃。這真的很重要。我想您已經聽過我們多次討論過這樣一個事實：90% 的患者更喜歡 UGN-102 而不是 TURBT。這些患者都曾接受過 TURBT 治療，並使用過 UGN-102。其中 90% 的人喜歡 UGN-102。

So we really need to ensure that we engage the patient because we want that physician, we want them to be part of the discussion and part of the treatment decision. So when a physician gives them the option, that they understand and know. So we will again engage a patient but not expected initially to be in broad-based DTC.

因此，我們確實需要確保與患者互動，因為我們希望醫生、我們希望他們參與討論和治療決策。因此，當醫生給他們選擇時，他們能夠理解並知道。因此，我們將再次與患者接觸，但最初預計不會涉及廣泛的 DTC。

Aydin Huseynov, Ladenburg.

拉登堡的艾登‧胡賽諾夫 (Aydin Huseynov)。

I got a couple. Maybe this is a little bit unusual question, but curious what would happen to patients who progress on UGN-102, with 20 or 25, whatever, percentage. So would they switch back to TURBT or would you redose them with UGN-102?

我有一對。也許這是一個有點不尋常的問題，但令人好奇的是，對於使用 UGN-102 的患者，如果病情有 20% 或 25% 出現進展，會發生什麼情況。那麼他們會改回 TURBT 嗎？還是會重新使用 UGN-102？

Yeah, Mark, do you want to take that?

是的，馬克，你想接受這個嗎？

Sure, so let me start out by saying that, thankfully, very few patients did what's called progress during treatment during [UGCR] program. And when we say progression, what we're talking about is a patient with low grade disease turning into someone with high grade disease or even more unusually turning into someone with invasive disease.

當然，首先我要說的是，值得慶幸的是，在 [UGCR] 計劃期間，很少有患者在治療過程中取得所謂的進展。當我們說進展時，我們談論的是低度疾病患者轉變為高度疾病患者，或更不尋常的是轉變為侵襲性疾病患者。

So that eventuality, that rarely occurred in our program. That said, in patients (technical difficulty) responded and then recurred or didn't respond completely after treatment with UGN-102, the use of transurethral resection to remove any residual or recurrent tumor was not associated with complications or problems that were exceptional or unusual. So the standard of care currently, which is our experience in our clinical trials program, is that it's not complicated by prior use or treatment with the UGN-102.

所以這種情況在我們的程序中很少發生。也就是說，對於接受 UGN-102 治療後出現反應但隨後復發或未完全反應的患者（技術難度），使用經尿道切除術切除任何殘留或復發的腫瘤不會引發併發症或異常或不尋常的問題。因此，目前的治療標準（也是我們在臨床試驗計畫中的經驗）是，先前使用或接受 UGN-102 治療不會使其變得複雜。

As for what you're anticipating, which is, if approved and patients are successfully treated initially with UGN-102, how would they subsequently be treated assuming they have the same disease. I think I'm going to defer to Liz as to how she thinks that would happen. We don't currently have clinical trials data to tell us specifically about what retreatment would mean or look like. But Liz, I don't know if you want to postulate what you think would happen once approved.

至於您的預期，即如果獲得批准並且患者最初使用 UGN-102 成功治療，那麼假設他們患有相同的疾病，隨後將如何治療。我想我會聽從 Liz 的意見，看看她認為這會如何發生。我們目前沒有臨床試驗數據來具體告訴我們再治療意味著什麼或是什麼樣子。但是 Liz，我不知道您是否想假設一旦獲得批准會發生什麼。

Yeah, I think it's very similar to what you would see in other cancers. If a patient gets a good response and a durable response and then they recur, physicians are likely to retreat with what they used the first time. And so we expect that to happen. We will absolutely either launch a Phase 4 study or have a registry or some way to generate data that shows what happens when patients recur and get retreated.

是的，我認為它與其他癌症的表現非常相似。如果患者的反應良好且持久，但隨後病情復發，醫生很可能會重新採用他們第一次使用的治療方法。因此我們期待這種情況發生。我們絕對會啟動第四階段研究，或建立登記冊或以某種方式產生數據，以顯示患者復發和再次治療時發生的情況。

And then the other patient population that will want to study as well are partial responders. So I don't know if you remember but when we did the announcement of the durability, one of the doctors talked about the fact that one patient had so much tumor and UGN-102 got 90% of the tumor, but they couldn't be considered a complete responder.

然後，另一個想要研究的患者群體是部分響應者。所以我不知道您是否記得，但是當我們宣布耐久性時，其中一位醫生談到這樣一個事實：一名患者的腫瘤很大，而 UGN-102 獲得了 90% 的腫瘤，但他們不能被視為完全反應者。

So what would happen if you gave that patient a couple of additional treatments? Could you take these partial responders and put them into complete response? So we will be looking at all of those questions through either IRRs, Phase 4, registry, but we will have a way to generate data that will support the use of UGN-102 once a patient has recurred either on our drug or after a TURBT. So very much expect that you would be able to retreat very much like you do, like I said in other cancers.

那麼，如果你給該患者幾次額外的治療，會發生什麼事？您能將這些部分響應者轉變為完全響應者嗎？因此，我們將透過 IRR、第 4 階段、註冊研究所有這些問題，但一旦患者在服用我們的藥物或接受 TURBT 後出現復發，我們將有辦法產生支持使用 UGN-102 的數據。因此非常希望您能夠像我在其他癌症中所說的那樣順利退卻。

Thank you. I appreciate that. And another question is commercial question. Could you clarify the [GTN] gross [tone] for Jelmyto, and what should be the expectation of GTN for UGN-102?

謝謝。我很感激。另一個問題是商業問題。您能否澄清一下 Jelmyto 的 [GTN] 整體 [語氣]，以及對 UGN-102 的 GTN 預期是什麼？

So we've been kind of in the mid-70%s net of growth for Jelmyto, and the one thing we said on the call is we do expect that the headwinds we've been experiencing for 340B, some of those headwinds are started to annualize a bit. So from a year-over-year growth perspective as we think about the rest of this year, we would expect that impact to be less impactful, in terms of our growth rate.

因此，Jelmyto 的淨成長率一直處於 70% 左右，我們在電話會議上說的一件事是，我們確實預計，我們在 340B 方面遇到的阻力，其中一些阻力已經開始有點年度化。因此，從同比增長的角度來看，當我們考慮今年剩餘時間時，我們預計對我們的成長率的影響會較小。

In terms of UGN-102, as Liz mentioned, over time, we do expect the gross in net profile to be more favorable for UGN-12 as compared to Jelmyto, mainly because mix of business to be more heavily weighted towards community. Initially at launch, we do expect more utilization in the hospital setting, but over time that that should shift over to the community.

就 UGN-102 而言，正如 Liz 所提到的，隨著時間的推移，我們確實預計 UGN-102 的淨利潤總額將比 Jelmyto 更有利，主要是因為業務組合將更加偏向社區。在最初推出時，我們確實期望在醫院環境中有更多利用率，但隨著時間的推移，這一目標應該轉移到社區。

Thank you. Ladies and gentlemen, I'm showing no further questions in the queue. I would now like to return the call back to Liz for closing remarks.

謝謝。女士們、先生們，隊列中沒有其他問題了。現在我想回電給 Liz 請她做最後發言。

Well, thanks to everybody. As you can imagine, a lot of excitement over here as we prepare and get ready for the ODAC next week. We appreciate all of your support. It's been a long time coming. We're excited about it though. We feel really good about it. And most importantly, these patients need new options.

好吧，謝謝大家。正如您所想像的，當我們為下週的 ODAC 做準備時，這裡充滿了興奮。我們感謝您的所有支持。已經等了很久了。但我們對此感到很興奮。我們對此感覺非常好。最重要的是，這些患者需要新的選擇。

And so we're very much looking forward to next week, and then following that the PDUFA. So thanks, and we'll keep you guys informed of any happenings. So thanks a lot. We'll talk to you guys soon. Bye-bye.

因此，我們非常期待下週以及隨後的 PDUFA。謝謝您，我們隨時會向大家通報最新情況。非常感謝。我們很快會和你們交談。再見。

Ladies and gentlemen, that concludes today's conference call. Thank you for your participation. You may now disconnect.

女士們、先生們，今天的電話會議到此結束。感謝您的參與。您現在可以斷開連線。