Nuvation Bio Inc (NUVB) 2025 Q2 法說會逐字稿

Hello and welcome to the new Nuvation Bio's second-quarter 2025 financial results and corporate update call. Today's call is being recorded, and our play will be available. (Operator Instructions)

您好，歡迎參加 Nuvation Bio 2025 年第二季財務業績和公司最新情況電話會議。今天的通話正在錄音，我們的劇本將會播出。（操作員指示）

Now I would like to turn the call over to JR DeVita, Executive Director of Corporate Development and Investor Relations at Nuvation Bio. Please go ahead.

現在我想將電話轉給 Nuvation Bio 公司企業發展和投資者關係執行董事 JR DeVita。請繼續。

Thank you and good morning, everyone. Welcome to the Nuvation Bio's second-quarter 2025 earnings conference call. Earlier today we released financial results for the quarter ending June 30, 2025 and provided a business update.

謝謝大家，早安。歡迎參加 Nuvation Bio 2025 年第二季財報電話會議。今天早些時候，我們發布了截至 2025 年 6 月 30 日的季度財務業績並提供了業務更新。

The press release is available on the investor section of our website at newfishandbio.com. A recording of this conference call can also be found on the investor section of our website following its completion.

新聞稿可在我們網站 newfishandbio.com 的投資者專區查看。電話會議結束後，您也可以在我們網站的投資人專區查看本次電話會議的錄音。

I'd like to remind you that today's call includes forward looking statements about IBTROZI's launch, our pipeline progress, and our cash runway.

我想提醒您，今天的電話會議包括有關 IBTROZI 的發布、我們的管道進度和我們的現金流的前瞻性陳述。

Because such statements deal with future events and are subject to many risks and uncertainties, actual results may differ materially from those in the forward-looking statements. For a full discussion of these risks and uncertainties, please review our annual report on form 10-K and our quarterly reports on form 10-Q that are filed with the US Securities and Exchange Commission.

由於此類陳述涉及未來事件，並受許多風險和不確定性的影響，實際結果可能與前瞻性陳述中的結果有重大差異。有關這些風險和不確定性的完整討論，請查看我們向美國證券交易委員會提交的 10-K 表年度報告和 10-Q 表季度報告。

Joining me on today's call to discuss our quarterly results are our Founder, President, and Chief Executive Officer, Dr. David Hung; and our Chief Financial Officer, Philippe Sauvage. David will provide an overview of the IBTROZI commercial launch and additional pipeline updates and Philippe will discuss our financial and operating updates for the quarter. David will then conclude with closing remarks.

今天與我一起參加電話會議討論季度業績的有我們的創始人、總裁兼首席執行官 David Hung 博士和首席財務官 Philippe Sauvage。David 將概述 IBTROZI 的商業發布和其他管道更新，而 Philippe 將討論我們本季的財務和營運更新。大衛將以結束語結束演講。

Now I'll turn the call over to Dr. David Hung. David?

現在我將把電話轉給 David Hung 博士。戴維？

Thanks, JR, and good morning, everyone. Thank you for joining us today. I'm excited to share a series of updates that mark an important new era for Nuvation Bio, namely as a commercial stage entity. Less than two months ago, on June 11, we achieved our most important company milestone to date, receiving full FDA approval for IBTROZI, our next generation, highly selective ROS1 tyrosine kinase inhibitor, or TKI.

謝謝，JR，大家早安。感謝您今天加入我們。我很高興與大家分享一系列更新，這些更新標誌著 Nuvation Bio 的一個重要新時代，即作為商業階段實體。不到兩個月前，即 6 月 11 日，我們實現了迄今為止最重要的公司里程碑，即我們的下一代高選擇性 ROS1 酪胺酸激酶抑制劑（TKI）IBTROZI 獲得了 FDA 的全面批准。

Per our label, IBTROZI is indicated for adults with locally advanced or metastatic ROS1+ non-small cell lung cancer, or NSCLC, in the line agnostic setting. Since then, the Nuvation Bio team has been executing on multiple fronts to bring this therapy to patients in need.

根據我們的標籤，IBTROZI 適用於治療線未知的局部晚期或轉移性 ROS1+ 非小細胞肺癌（NSCLC）成人患者。自那時起，Nuvation Bio 團隊一直在多個方面努力為有需要的患者提供這種療法。

Our next key achievement occurred on June 20, just a week following FDA approval, when the National Comprehensive Cancer Network, or NCCN, added IBTROZI as a preferred agent for both first line and subsequent lines of therapy for ROS1+ non-small cell lung cancer patients, consistent with our line agnostic label. The NCCN guidelines also included specific recommendations for IBTROZI for patients with brain metastases and resistance mutations.

我們的下一個關鍵成就發生在 6 月 20 日，即 FDA 批准後僅一周，當時美國國家綜合癌症網絡 (NCCN) 將 IBTROZI 添加為 ROS1 + 非小細胞肺癌患者一線和後續治療的首選藥物，這與我們的線不可知標籤一致。NCCN 指引也針對腦轉移和抗藥性突變患者使用 IBTROZI 提出了具體建議。

The NCCN guidelines are a highly regarded resource in clinical decision-making, especially in the community setting and play a critical role in the development of formulary and treatment pathway decisions. We believe the rapidity with which IBTROZI was added as a preferred agent in the NCCN guidelines gives credibility to its differentiated efficacy and safety profile, while also highlighting IBTROZI as a new and important treatment option for patients with ROS1+ NSCLC.

NCCN 指南是臨床決策中備受推崇的資源，尤其是在社區環境中，在處方集和治療途徑決策的製定中發揮關鍵作用。我們相信，IBTROZI 如此迅速地被添加為 NCCN 指南中的首選藥物，證明了其差異化的療效和安全性，同時也凸顯了 IBTROZI 作為 ROS1+ NSCLC 患者的一種新的、重要的治療選擇。

This viewpoint, along with an associated urgency to utilize ROS1 targeted agents for these patients instead of immunotherapy and chemotherapy, has been echoed by oncologists in our post-approval marketing conversations in both the academic and community settings. This NCCN endorsement strengthens IBTROZI's competitive position, and we believe it has been and will continue to be important in spreading awareness of IBTROZI among prescribers and payers.

在學術和社區環境中的上市後行銷對話中，腫瘤學家也表達了這種觀點，並迫切需要為這些患者使用 ROS1 標靶藥物，而不是免疫療法和化療。NCCN 的認可增強了 IBTROZI 的競爭地位，我們相信，它在處方者和付款人中傳播 IBTROZI 意識方面一直並將繼續發揮重要作用。

On the access front, early dialog with national and regional plans has been favorable. Major payers were engaged immediately following approval, and the initial coverage landscape is looking strong. As of the end of July, we already have confirmed coverage of IBTROZI from payers representing 58% of covered lives.

在准入方面，與國家和地區計劃的早期對話是有利的。批准後，主要付款人立即參與其中，初步覆蓋前景看好。截至 7 月底，我們已確認 IBTROZI 的承保範圍涵蓋了佔受保人數 58% 的付款人。

In addition, our patient support program, Nuvation Connect, is fully operational, answering questions and helping patients and caregivers navigate reimbursement and access programs. It is still early days, but the launch is building promising momentum.

此外，我們的患者支援計劃 Nuvation Connect 已全面投入運營，可以解答問題並幫助患者和照護者了解報銷和訪問計劃。雖然現在還處於早期階段，但此次發布正在積聚良好的勢頭。

By the end of July, we had engaged with over 90% of our Tier 1 accounts and over 85% of our Tier 2 accounts across both community and academic centers, which combined are where we expect the majority, though not all, IBTROZI prescriptions to come from. This engagement is critical to our launch as it ensures prescribers understand IBTROZI's compelling efficacy and safety, convenient once-daily dosing, and simple path to patient access through our specialty pharmacy and distribution partners, in addition to our previously mentioned Nuvation Connect patient support program.

截至 7 月底，我們已經與社區和學術中心的 90% 以上的一級帳戶和 85% 以上的二級帳戶進行了接觸，我們預計 IBTROZI 處方的大部分（儘管不是全部）都來自這些帳戶。這種參與對於我們的產品發布至關重要，因為它確保處方者了解 IBTROZI 令人信服的功效和安全性、方便的每日一次給藥方式，以及透過我們的專業藥房和分銷合作夥伴以及我們之前提到的 Nuvation Connect 患者支援計劃，患者可以輕鬆獲得 IBTROZI。

Our engagements with key customers are already translating into early adoption of IBTROZI. Doctors have written prescriptions in 100% of our six sales regions and 75% of our 47 sales territories, including over 50 different prescribers across community centers, academic centers, and integrated delivery networks. In addition, 80% of our top 10 target accounts have prescribed IBTROZI.

我們與主要客戶的合作已轉化為對 IBTROZI 的早期採用。我們六個銷售區域的 100% 和 47 個銷售地區的 75% 均有醫生開立處方，其中包括來自社區中心、學術中心和綜合配送網絡的 50 多名不同的開處方者。此外，我們前 10 個目標帳戶中有 80% 都已開立 IBTROZI。

As a result of this positive traction, we are excited to report that 70 patients have started IBTROZI as of the end of July, which constitutes roughly seven weeks of commercial effort given our mid-June FDA approval and July being our first full month of commercialization. And given the Juneteenth and July Fourth holidays, these first seven weeks of commercial launch constituted only 34 full business days.

由於這種積極的推動，我們很高興地報告，截至 7 月底，已有 70 名患者開始使用 IBTROZI，考慮到我們在 6 月中旬獲得 FDA 批准，而 7 月是我們商業化的第一個完整月份，這大約相當於七週的商業努力。考慮到六月節和七月四日假期，商業發布的前七週僅有 34 個完整工作日。

So out of the gate, we are already achieving slightly over two new patients starting on IBTROZI per business day, and we expect that pace to accelerate.

因此，從一開始，我們每個工作日就已接待了略多於兩名的新患者開始使用 IBTROZI，我們預計這一速度將會加快。

Furthermore, more than 90% of the patients on IBTROZI were new patients started after FDA approval, as only six patients were enrolled in our early access program. As we have previously indicated, we consider the number of patients on IBTROZI to be the best metric for future success, independent of their reimbursement status.

此外，超過 90% 接受 IBTROZI 治療的患者都是 FDA 批准後開始接受治療的新患者，因為只有六名患者參加了我們的早期使用計畫。正如我們之前所指出的，我們認為使用 IBTROZI 的患者數量是衡量未來成功的最佳指標，與他們的報銷狀況無關。

We put our 70 patients on IBTROZI by the end of its first full month of launch into some context. AUGTYRO was also approved mid-month. By the end of AUGTYRO's first full month of launch, there was only one patient on drug as reported by IQVIA. By the end of AUGTYRO's second full month of launch, there were still only 18 patients on drug as reported by IQVIA, which we are well aware does not capture patients on free drug or even all patients on commercial drug.

在 IBTROZI 上市的第一個整月結束時，我們為 70 名患者安排了該藥物治療。AUGTYRO 也在月中獲得批准。根據 IQVIA 報告，AUGTYRO 上市第一個月結束時，只有一名患者服用該藥物。截至 AUGTYRO 推出第二個月末，IQVIA 報告顯示，仍有 18 名患者在服用該藥物，我們很清楚，這並未包括服用免費藥物的患者，甚至沒有包括服用商業藥物的所有患者。

While some of our 70 IBTROZI patients were enrolled in our free trial program, this program typically runs for only one month to allow immediate access to IBTROZI or access issues like prior authorizations are resolved. Therefore, most patients on this program would be expected to generate full commercial sales starting in the second month.

雖然我們的 70 名 IBTROZI 患者中有一些參加了我們的免費試用計劃，但該計劃通常只運行一個月，以允許立即使用 IBTROZI 或解決事先授權等訪問問題。因此，預計該計劃的大多數患者將從第二個月開始產生全部商業銷售額。

We are thrilled with the robust rate of IBTROZI adoption thus far in our launch, and we believe that it is a clear reflection of IBTROZI's highly differentiated efficacy and safety profile. We have previously commented that based on pooled data as published in the Journal of Clinical Oncology, IBTROZI's confirmed overall response rate of 89% and median duration of response, or DOR, of nearly four years in the first line setting have not, to our knowledge, previously been shown by any approved cancer drug in any solid tumor indication.

我們對 IBTROZI 在我們推出以來的強勁採用率感到非常興奮，我們相信這清楚地反映了 IBTROZI 高度差異化的功效和安全性。我們先前曾評論說，根據《臨床腫瘤學雜誌》上發表的匯總數據，IBTROZI 確認的總體緩解率為 89%，中位緩解持續時間（DOR）為近四年，據我們所知，此前還沒有任何已批准的抗癌藥物在任何實體瘤適應症中顯示出這種療效。

And with almost five months of additional maturity for the efficacy data set that comprise the IBTROZI label, the median DOR metrics for the individual TRUST-I and TRUST-II studies are now no longer in reach. This durability is a key factor in giving doctors confidence to prescribe IBTROZI, not only in the first line setting, but across all lines of therapy.

而且，由於 IBTROZI 標籤的功效資料集還有近五個月的額外成熟期，單一 TRUST-I 和 TRUST-II 研究的中位 DOR 指標現已無法達到。這種持久性是讓醫生有信心開出 IBTROZI 的關鍵因素，不僅作為第一線治療，而且作為所有治療方案。

And given that our pivotal clinical trials for IBTROZI were started over five years ago, it will take many years for a ROS1 TKI in development to reach the length of follow-up time used to evaluate IBTROZI in clinical studies. Therefore, we believe that our head start and IBTROZI's robust median DOR will give IBTROZI an important competitive edge in influencing physicians' use of ROS1 therapy.

鑑於我們對 IBTROZI 的關鍵臨床試驗是在五年前開始的，因此，正在開發的 ROS1 TKI 需要很多年才能達到用於在臨床研究中評估 IBTROZI 的追蹤時間長度。因此，我們相信，我們的領先優勢和 IBTROZI 強勁的中位 DOR 將使 IBTROZI 在影響醫生使用 ROS1 療法方面具有重要的競爭優勢。

IBTROZI's robust efficacy comes with a safety profile that shows it is generally well-tolerated, including adverse events that are generally low-grade, transient, and manageable. While we will not walk through all adverse events detailed in our prescribing information today, I'd like to address the two most common safety events.

IBTROZI 的強大功效與安全性相伴而生，表明其通常耐受性良好，包括通常較低級別、短暫且可控的不良事件。雖然我們今天不會詳細介紹處方資訊中的所有不良事件，但我想談談兩個最常見的安全事件。

First, the most common lab-based adverse events associated with IBTROZI use are increased aspartate aminotransferase, or AST, and increased alanine aminotransferase, or ALT. These laboratory abnormalities are not clinically apparent to patients, as they generally cause no symptoms, even though they do require appropriate monitoring.

首先，與 IBTROZI 使用相關的最常見的實驗室不良事件是天冬氨酸氨基轉移酶 (AST) 升高和丙氨酸氨基轉移酶 (ALT) 升高。這些實驗室異常對於患者來說在臨床上並不明顯，因為它們通常不會引起任何症狀，儘管它們確實需要適當的監測。

And when we look at how often the adverse events of AST and ALT increases led to treatment discontinuation in our safety database, only one out of 337 patients with ROS1+ NSCLC discontinued IBTROZI due to these events.

當我們查看安全資料庫中 AST 和 ALT 升高的不良事件導致治療停止的頻率時，337 名 ROS1+ NSCLC 患者中只有 1 名因這些事件而停止使用 IBTROZI。

The most common clinical adverse event associated with IBTROZI use is diarrhea. The vast majority of which is Grade 1, occurs within about two days of starting therapy, and resolves in about 24 hours. We have previously stated that we believe IBTROZI's efficacy and safety profile will result in wider adoption and longer use of IBTROZI and therefore create a larger commercial opportunity than has been seen to date for other ROS1 TKIs.

與 IBTROZI 使用相關的最常見臨床不良事件是腹瀉。其中絕大多數為 1 級，發生在開始治療後約兩天內，並在約 24 小時內消退。我們之前曾表示，我們相信 IBTROZI 的功效和安全性將使其得到更廣泛的採用和更長的使用，從而創造比迄今為止其他 ROS1 TKI 更大的商業機會。

So far, our early launch results appear to be consistent with our belief that IBTROZI will become the treatment of choice for physicians and patients.

到目前為止，我們早期推出的結果似乎與我們的信念一致，即 IBTROZI 將成為醫生和患者的首選治療方法。

Now I'd like to provide a bit more detail on the types of patients that have been prescribed IBTROZI, which shows the broad potential of our therapy. The makeup of these patients has been quite diverse and includes both TKI-naive and TKI pre-treated populations, and patients from both academic and community settings across the country.

現在我想更詳細地介紹接受 IBTROZI 治療的患者類型，這顯示了我們這種療法的廣泛潛力。這些患者的組成相當多樣化，包括未接受 TKI 治療的人群和接受過 TKI 治療的人群，以及來自全國各地學術和社區環境的患者。

We are delighted to see IBTROZI being prescribed in the frontline settings, as IBTROZI's nearly four-year median DOR suggests that this indication will generate the longest and highest revenue stream. In the second-line settings, some patients have started IBTROZI following the progression of a prior TKI, as we expected.

我們很高興看到 IBTROZI 被應用於一線治療，因為 IBTROZI 近四年的中位 DOR 表明該適應症將產生最長和最高的收入流。在二線治療中，正如我們預期的那樣，一些患者在先前的 TKI 治療進展後開始使用 IBTROZI。

However, of note, a portion of TKI pre-treated patients have switched to IBTROZI due to tolerability issues with other prior ROS1 therapies. This indicates to us that both prescribers and patients feel IBTROZI can provide not only an efficacious, but a more tolerable treatment option as well.

然而，值得注意的是，由於其他先前 ROS1 療法的耐受性問題，一部分接受 TKI 預先治療的患者已改用 IBTROZI。這向我們表明，處方醫生和患者都認為 IBTROZI 不僅是一種有效的治療選擇，而且還是一種更耐受的治療選擇。

Additionally, and somewhat unexpectedly, we have also seen some switches from crizotinib to IBTROZI that were not due to disease progression or tolerability issues. We believe this may reflect the appreciation by oncologists that crizotinib does not penetrate the blood-brain barrier to prevent or treat brain metastases, while IBTROZI has strong CNS activity.

此外，有些出乎意料的是，我們也看到一些從克唑替尼到 IBTROZI 的轉換並不是由於疾病進展或耐受性問題。我們認為這可能反映了腫瘤學家的認識，即克唑替尼不能穿透血腦屏障來預防或治療腦轉移，而 IBTROZI 具有強大的中樞神經系統活性。

In our experience, it is remarkable for oncologists to switch therapies for patients who have not yet progressed. The crizotinib switches that we have already seen are promising, and we believe the right thing to do for patients given IBTROZI's strong CNS activity and the fact that the brain is the primary site for disease progression.

根據我們的經驗，腫瘤科醫師為病情尚未進展的患者更換治療方法是非常了不起的。我們已經看到的克唑替尼轉換療法很有前景，我們相信，考慮到 IBTROZI 強大的中樞神經系統活性以及大腦是疾病進展的主要部位這一事實，這對患者來說是正確的做法。

In summary, we are seeing broad adoption of IBTROZI in both the first and second-line settings, and in the latter, we are seeing switches to IBTROZI for disease progression and tolerability issues with other TKIs, and even switches for neither disease progression nor tolerability issues with other TKIs.

總之，我們看到 IBTROZI 在一線和二線治療中都得到了廣泛的應用，而在二線治療中，我們看到有人因為疾病進展和其他 TKI 的耐受性問題而改用 IBTROZI，甚至有人因為疾病進展和其他 TKI 的耐受性問題而改用 IBTROZI。

We are gratified that IBTROZI has the potential to positively impact the lives of so many different types of patients with ROS1 and positive NSCLC this early in the year. We are also excited to see the positive outcomes of IBTROZI in our launch.

我們感到欣慰的是，IBTROZI 有可能在今年年初對如此多不同類型的 ROS1 和陽性 NSCLC 患者的生活產生積極影響。我們也很高興看到 IBTROZI 在我們推出後取得了積極成果。

We view these positive updates, NCCN inclusion, constructive engagement with payers and providers, and most importantly, various types of new patient starts as encouraging indicators of how our launch will progress. When combined with IBTROZI's balanced and differentiated efficacy and safety profile, these signals gives us confidence that IBTROZI can become the standard of care and market leader in ROS1+ NSCLC.

我們認為這些積極的更新、NCCN 納入、與付款人和提供者的建設性接觸，以及最重要的是各種類型的新患者的開始，都是我們發布會進展的令人鼓舞的指標。結合 IBTROZI 均衡且差異化的療效和安全性，這些訊號讓我們有信心 IBTROZI 可以成為 ROS1+ NSCLC 的治療標準和市場領導者。

Looking ahead, we see considerable opportunities to increase the size of the ROS1+ NSCLC market. While our team is focused on adoption of IBTROZI, we are also focused on increasing the awareness of the importance of testing for oncogenic drivers, like ROS1, and ensuring patients are treated with the appropriate targeted therapy. There are approximately 3,000 advanced ROS1+ NSCLC patients who could be diagnosed in the US every year by DNA-based testing.

展望未來，我們看到了擴大 ROS1+ NSCLC 市場規模的巨大機會。雖然我們的團隊專注於採用 IBTROZI，但我們也致力於提高人們對檢測 ROS1 等致癌驅動因素的重要性的認識，並確保患者接受適當的標靶治療。美國每年約有 3,000 名晚期 ROS1+ NSCLC 患者可透過基於 DNA 的檢測進行診斷。

Looking further ahead, we would expect an eventual shift to RNA-based testing to increase the total addressable patient population to approximately 4,000 new patients per year, as publications have shown that RNA-based testing will detect roughly 30% more ROS1 infusions than DNA testing.

展望未來，我們預計最終轉向基於 RNA 的檢測將使可尋址患者總數增加到每年約 4,000 名新患者，因為出版物表明，基於 RNA 的檢測將比 DNA 檢測多檢測到約 30% 的 ROS1 輸注。

Therefore, given IBTROZI's last published median DOR and median progression-free survival or PFS of nearly four years, we would expect the theoretical maximum number of first line patients treated with IBTROZI over this period to equilibrate at roughly 16,000 patients in year four.

因此，考慮到 IBTROZI 最新公佈的中位 DOR 和中位無惡化存活期或 PFS 接近四年，我們預計在此期間接受 IBTROZI 治療的一線患者的理論最大數量將在第四年平衡至約 16,000 名患者。

The prolonged durability of IBTROZI creates a patient stacking phenomenon that turns a small instance population into a large prevalence population, thus generating an expanded market opportunity. Again, this example is based on first-line patients only and does not account for the pre-treated population that further increases the addressable population over this illustrative time frame.

IBTROZI 的長期抗藥性產生了患者堆積現象，將小規模病例群體轉變為大規模患病群體，從而產生了擴大的市場機會。再次強調，此範例僅基於第一線患者，並未考慮在此說明性時間範圍內進一步增加可尋址人群的預處理人群。

Our 47 account managers, supported by regional medical, access, and commercial specialists, remain focused on removing barriers and expanding IBTROZI's reach, particularly in the community setting, to maximize the commercial opportunity of IBTROZI.

我們的 47 名客戶經理在區域醫療、准入和商業專家的支持下，始終致力於消除障礙並擴大 IBTROZI 的覆蓋範圍，特別是在社區環境中，以最大限度地發揮 IBTROZI 的商業機會。

While we are now a commercial-stage company, let us not overlook the other exciting programs in development in our pipeline. Safusidenib, our mutant IDH1 inhibitor, is being developed for diffuse IDH1 mutant glioma, a devastating brain cancer for which there are very few treatment options available today. As a reminder, there are approximately 2,400 new cases of IDH mutant glioma per year, split almost evenly between low-grade and high-grade.

雖然我們現在是一家商業階段的公司，但我們不要忽視正在開發的其​​他令人興奮的項目。我們的突變型 IDH1 抑制劑 Safusidenib 正在開髮用於治療瀰漫性 IDH1 突變型神經膠質瘤，這是一種毀滅性的腦癌，目前可用的治療選擇非常少。提醒一下，每年大約有 2,400 例新的 IDH 突變型膠質瘤病例，低度和高級別病例幾乎各佔一半。

While the incidence is smaller than ROS1+ NSCLC, the market opportunity is materially larger because patients with low-grade and high-grade IDH mutant glioma live approximately 10 to 15 and 3 to 7 years, respectively. Therefore, the opportunity in IDH mutant glioma is expected to potentially be significantly larger than in ROS1+ NSCLC, already a sizable commercial opportunity.

雖然發生率低於 ROS1+ NSCLC，但市場機會卻大得多，因為低度和高等級 IDH 突變型膠質瘤患者的壽命分別約為 10 至 15 年和 3 至 7 年。因此，IDH 突變型膠質瘤的機會預計可能比 ROS1+ NSCLC 大得多，這已經是一個相當大的商業機會。

The only treatment option available for patients with IDH1 mutant glioma is vorasidenib, which was approved by the US FDA in August 2024, and only for low-grade glioma. In this pivotal INDIGO study, vorasidenib demonstrated a progression-free survival of PFS of 27.7 months and an overall response rate of ORR of 11%.

IDH1突變型膠質瘤患者唯一可用的治療選擇是vorasidenib，該藥物於2024年8月獲得美國FDA批准，且僅適用於低度膠質瘤。在這項關鍵的 INDIGO 研究中，vorasidenib 顯示出 27.7 個月的無惡化存活期 PFS 和 11% 的整體反應率 ORR。

Safusidenib showed an ORR of 33% in a clinical study of patients with recurrent low-grade IDH1 mutant glioma. Furthermore, there are no agents approved in high-grade IDH1 mutant glioma, where safusidenib showed an ORR of 17%, including two complete responses, lasting multiple years in a clinical study.

在針對復發性低度 IDH1 突變型膠質瘤患者的臨床研究中，Safusidenib 的 ORR 為 33%。此外，目前尚無藥物核准用於治療高等級 IDH1 突變型膠質瘤，而沙夫西地尼在臨床研究中的 ORR 為 17%，其中包括兩次完全緩解，且持續時間長達數年。

To our knowledge, no other IDH inhibitors have demonstrated responses of this kind in this indication. And we believe that the next step in the study is to develop a new, more comprehensive, and we believe this speaks to the impressive clinical profile of safusidenib. We plan to disclose more data evaluating safusidenib in the low-grade population post-surgical reception by year end.

據我們所知，沒有其他 IDH 抑制劑在此適應症中表現出此類反應。我們相信，研究的下一步是開發一種新的、更全面的藥物，我們相信這證明了沙夫西地尼令人印象深刻的臨床特性。我們計劃在年底前披露更多評估沙夫西地尼在低級別人群術後接受治療的數據。

Based on data generated to date, we have modified the ongoing Phase 2 study of safusidenib in the United States to evaluate maintenance treatment of safusidenib against placebo in high-grade IDH1 mutant glioma. Please refer to clinicaltrials.gov for additional details on the study design.

根據迄今為止產生的數據，我們修改了正在進行的美國沙夫西地尼 2 期研究，以評估沙夫西地尼與安慰劑對高級別 IDH1 突變型膠質瘤的維持治療效果。有關研究設計的更多詳細信息，請參閱 clinicaltrials.gov。

Finally, we are in active discussions with the FDA regarding the development of safusidenib in IDH1 mutant glioma. These discussions cover the expansion of the ongoing study in high-grade glioma with registrational intent and the potential design of a pivotal study of safusidenib in low-grade IDH1 mutant glioma. We look forward to providing updates on our development plan later this year.

最後，我們正在與 FDA 積極討論沙夫西地尼在 IDH1 突變型膠質瘤中的發展。這些討論涵蓋了正在進行的具有註冊意圖的高級別膠質瘤研究的擴展以及對低級別 IDH1 突變型膠質瘤進行 safusidenib 關鍵研究的潛在設計。我們期待在今年稍後提供有關我們開發計劃的最新資訊。

NUV-1511 is the first clinical candidate of our drug-drug conjugate, or DDC, platform and represents a new modality in targeted cancer therapy. We plan to provide an update from our Phase 1 dose escalation study in difficult-to-treat solid tumors later this year. We remain confident that we have the team, strategy, and mindset to execute our program successfully, build lasting value, and most importantly, serve patients in need.

NUV-1511 是我們藥物-藥物偶聯物（DDC）平台的首個臨床候選藥物，代表了標靶癌症治療的一種新方式。我們計劃在今年稍後提供針對難治性實體瘤的 1 期劑量遞增研究的最新進展。我們堅信，我們擁有團隊、策略和思維方式來成功執行我們的計劃，創造持久的價值，最重要的是，為有需要的患者提供服務。

With that, I'll turn it over to Philippe to provide an update on our operations and financials for the quarter. Philippe?

接下來，我將把時間交給菲利普，讓他提供本季的營運和財務狀況的最新情況。菲利普？

Thanks, David, and good morning, everyone. For detailed second-quarter 2025 financials, please refer to the press release we issued this morning, which is available on our website.

謝謝，大衛，大家早安。有關 2025 年第二季的詳細財務狀況，請參閱我們今天上午發布的新聞稿，該新聞稿可在我們的網站上查閱。

Now let me bring your attention to a few highlights from the quarter. This is our first quarter reporting as a commercial company, and I'm pleased to inform you that we've generated $4.8 million in total revenue. This includes $1.2 million in net product revenue from the trade and investment of our product revenue from IBTROZI during the first 13 business days from FDA approval to the end of June.

現在，讓我提請大家關注本季的一些亮點。這是我們作為商業公司的第一季報告，我很高興地通知大家，我們的總收入為 480 萬美元。其中包括從 FDA 批准到 6 月底的前 13 個工作天內，來自 IBTROZI 產品收入的貿易和投資的 120 萬美元淨產品收入。

As anticipated, most of the IBTROZI net revenue comes from channel stocking, so we do not expect this to be a significant component of our sales in the future and currently have more than enough demand to convert supply to new patient stocks.

正如預期的那樣，IBTROZI 的大部分淨收入來自通路庫存，因此我們預計這不會成為我們未來銷售的重要組成部分，並且目前有足夠的需求將供應轉化為新的患者庫存。

As a reminder, our limited distribution model will limit initial stock buildup and drug in channel, but will also provide better long-term efficiency and access. The remaining product revenue comes from new patient stocks over this short period, some of which were enrolled in our free trial program, which typically runs for only one month to allow patients to receive needed treatment immediately without waiting for reimbursement approval.

提醒一下，我們的有限分銷模式將限制初始庫存累積和通路藥物，但也會提供更好的長期效率和管道。剩餘的產品收入來自於這段短時間內新增的患者，其中一些患者參加了我們的免費試用計劃，該計劃通常只運行一個月，以便患者能夠立即接受所需的治療，而無需等待報銷批准。

We would expect most patients on this program to generate full commercial revenue in their second month on IBTROZI.

我們預計該計劃中的大多數患者在使用 IBTROZI 的第二個月就能獲得全部商業收入。

The remaining revenue comes from our collaboration and license agreement, including product supply, royalty revenue, and research and development services. While our current royalty revenue comes from our own commercialization partner in China, Innovent Biologics, we expect to begin receiving additional royalty revenue from our partner in Japan, Nippon Kayaku, following the anticipated approval of taletrectinib in Japan later this year. Notably, approval and reimbursement listing in Japan will result in a $25 million milestone payment from Nippon Kayaku to Nuvation Bio.

其餘收入來自我們的合作和授權協議，包括產品供應、特許權使用費收入以及研發服務。雖然我們目前的專利權使用費收入來自於我們在中國的商業化合作夥伴信達生物製藥，但我們預計，隨著塔雷替尼預計在今年晚些時候在日本獲得批准，我們將開始從我們在日本的合作夥伴日本化藥那裡獲得額外的專利權使用費收入。值得注意的是，在日本獲得批准和報銷將導致日本化藥向 Nuvation Bio 支付 2500 萬美元的里程碑付款。

As we mentioned on our last quarterly call, the real metric of success is the number of patients we help with our differentiated therapy. Given this, we will focus on providing quarterly updates on the number of new and continuing patients prescribed in IBTROZI. This will offer key insights into how our launch is evolving and show how we can build a sustained revenue stream given the prolonged durability and high response rate demonstrated by IBTROZI in clinical studies.

正如我們在上次季度電話會議上提到的那樣，成功的真正衡量標準是我們透過差異化治療幫助的患者數量。有鑑於此，我們將重點放在每季更新 IBTROZI 處方的新患者和繼續服用 IBTROZI 的患者數量。這將為我們的上市過程提供關鍵見解，並展示我們如何在 IBTROZI 在臨床研究中表現出的持久性和高反應率下建立持續的收入來源。

David already mentioned that we have 70 patients on IBTROZI at the end of July, which we believe is a very strong commercial start.

David 已經提到，截至 7 月底，我們有 70 名患者使用 IBTROZI，我們相信這是一個非常強勁的商業開端。

On the expense side, R&D expenses for the quarter were $27.4 million as we continued investment in our lead asset, IBTROZI, in our clinical stage pipeline, including safusidenib and NUV-1511.

在費用方面，本季的研發費用為 2,740 萬美元，因為我們繼續投資於我們臨床階段管道中的領先資產 IBTROZI，包括 safusidenib 和 NUV-1511。

SG&E expenses were $38.5 million, primarily driven by our continued commercial build-out. This includes personal related expenses tied to commercial operations, as well as the strategic investment in medical education, payer engagement, patient support programs, and marketing.

SG&E 支出為 3850 萬美元，主要由於我們持續的商業建設。這包括與商業運營相關的個人相關費用，以及對醫學教育、付款人參與、患者支持計劃和行銷的策略性投資。

As a reminder, we have right sized our sales force with 47 oncology accounts managers. We do not expect to increase our sales force or materially increase other parts of our commercial team.

提醒一下，我們已將銷售團隊規模調整到 47 位腫瘤科客戶經理。我們並不打算增加銷售人員或大幅增加商業團隊的其他部分。

Turning to the balance sheet, we ended the quarter with $607.7 million in cash, cash equivalents, and marketable securities. This figure includes the proceeds received to date from a recently announced financing agreement with Sagard Healthcare Partners, which provides up to $250 million in non-dilutive capital.

談到資產負債表，我們在本季末擁有 6.077 億美元的現金、現金等價物和有價證券。這一數字包括迄今為止與 Sagard Healthcare Partners 達成的最近宣布的融資協議所獲得的收益，該協議提供了高達 2.5 億美元的非稀釋性資本。

To be precise, shortly after the FDA approval of IBTROZI, we received $200 million from Sagard, including $150 million of royalty financing and $50 million of non-dilutive capital. Under a term loan, an additional $50 million under the term loan is available at our auction until June 30, 2026.

確切地說，在 FDA 批准 IBTROZI 後不久，我們從 Sagard 獲得了 2 億美元，其中包括 1.5 億美元的特許權使用費融資和 5000 萬美元的非稀釋性資本。根據定期貸款，我們拍賣的另外 5,000 萬美元定期貸款有效期至 2026 年 6 月 30 日。

As we have stated previously, this transaction solidified our capital position. We already believe our cash balance prior to these non-dilutive financing with Sagard was sufficient to fund operations for profitability, including the US launch of IBTROZI and advancement of our pipeline. Now, our ability to reach profitability without additional funding is even more clear.

正如我們之前所說，這筆交易鞏固了我們的資本地位。我們已經相信，在與 Sagard 進行這些非稀釋性融資之前，我們的現金餘額足以為盈利運營提供資金，包括在美國推出 IBTROZI 和推進我們的管道。現在，我們在沒有額外資金的情況下實現盈利的能力更加明顯。

Operationally, we remain an agile organization with the flexibility to redirect resources as insights emerge into commercial launch, development of our pipeline, and evaluation of other exciting external opportunities. That discipline, combined with early IBTROZI performance and a robust cash balance, positions us extremely well to execute the remainder of our 2025 objectives. We have the right team, structure, resources, and preserved flexibility to continue to grow responsibly.

在營運方面，我們仍然是一個敏捷的組織，隨著對商業發布、管道開發和其他令人興奮的外部機會的評估的深入了解，我們可以靈活地重新分配資源。這種紀律，加上 IBTROZI 的早期表現和強勁的現金餘額，使我們能夠很好地實現 2025 年剩餘的目標。我們擁有合適的團隊、結構、資源和保留的靈活性，可以繼續負責任地發展。

With that, I'll hand it back to David.

說完這些，我就把它交還給大衛。

Thanks, Philippe.

謝謝，菲利普。

The early momentum we're seeing with IBTROZI, including strong uptake, constructive payer and provider discussions, and enthusiastic physician feedback, makes us confident we're on the right trajectory. It's still early days, and biotechnologies are always complex, but these first signs reinforce our conviction that IBTROZI can potentially become the standard of care for patients with ROS1+ NSCLC, and more broadly, demonstrate how effectively our team can execute.

我們看到 IBTROZI 的早期發展勢頭，包括強勁的接受度、付款人和提供者的建設性討論以及醫生的熱情反饋，這些讓我們相信我們正走在正確的軌道上。現在還處於早期階段，生物技術總是很複雜，但這些初步跡象增強了我們的信念，即 IBTROZI 有可能成為 ROS1 + NSCLC 患者的治療標準，更廣泛地說，證明了我們的團隊能夠有效地執行。

At the same time, we're advancing a pipeline designed to tackle some of the toughest challenges in cancer treatment. Each program reflects the same urgency and scientific rigor that brought IBTROZI to market, and together they position the Nuvation Bio to make a long-term impact.

同時，我們正在推動旨在解決癌症治療中一些最棘手挑戰的研發項目。每個項目都體現了將 IBTROZI 推向市場的同樣的緊迫性和科學嚴謹性，它們共同使 Nuvation Bio 產生長期影響。

We're energized by the work ahead, and most importantly, by the opportunity to improve the lives of patients in need.

我們對未來的工作充滿信心，最重要的是，我們有機會改善有需要的患者的生活。

With that, Operator, please open the lines for questions.

接線員，請打開熱線來回答問題。

(Operator Instructions)

（操作員指示）

Kaveri Pohlman, Clear Street.

卡弗里·波爾曼 (Kaveri Pohlman)，清晰街。

Congrats on the progress, and thanks for taking my question. I was wondering if you can provide any guidance on the next quarter sales or the number of patients on treatment you expect to see. Do you expect the rate of enrollment to remain the same or perhaps increase? And any additional insight into first line versus second line use, if you can provide any breakdown there? And what percentage of patients had drugs switching from crizotinib in first line?

恭喜您取得進展，並感謝您回答我的問題。我想知道您是否可以對下一季的銷售額或預計接受治療的患者數量提供任何指導。您預計入學率會維持不變還是增加？您對於一線和二線的使用還有什麼其他見解嗎，能否提供一些細分？有多少比例的患者將一線用藥從克唑替尼換成了其他藥物？

Hi, Kaveri. So in answer to the first part of your question, the 70 patients that we have on drug at the end of July reflected 34 business days, so about two patients per day.

你好，卡弗里。因此，回答您問題的第一部分，截至 7 月底，我們有 70 名患者接受藥物治療，這反映了 34 個工作日，因此每天大約有兩名患者接受藥物治療。

And there's only four business days in August, but so far in those four business days, we've now put about another three patients per day on drugs. So we do think that it's very early. It's hard to really know that all sustained, but it looks to us like there is a slight uptick, and we would expect this to accelerate, as most launches do for a successful drug.

八月只有四個工作日，但到目前為止，在這四個工作日裡，我們每天為另外三名患者提供藥物。所以我們確實認為現在還為時過早。很難真正知道這一切是否能夠持續下去，但在我們看來，似乎有一個小幅上升，我們預計這一趨勢會加速，就像大多數成功藥物的上市一樣。

So I think that we feel pretty good about where we are, and as you know, it still takes a while to get the drug on the formularies of all the aggregators that you want, and so we think that there are a number of events coming up that will further accelerate that curve, but I would say so far, just from the early days of August, we're already seeing an uptick from what we saw in July.

所以我認為我們對目前的狀況感到很滿意，而且正如你所知，要讓這種藥物進入你想要的所有聚合器的處方集還需要一段時間，所以我們認為即將發生的一系列事件將進一步加速這一曲線，但我想說到目前為止，僅從 8 月初開始，我們已經看到了比 7 月份有所上升的跡象。

On the second side, on the breakdown of the patients, it's so early in our launch that I think it's going to be difficult for us to comment on that. We don't get to see a lot of that information because the vast majority of our patients are actually in specialty distributors, which is blind to us.

第二方面，關於患者的具體情況，我們推出這項服務還處於初期階段，因此我認為我們很難對此發表評論。我們看不到很多這樣的訊息，因為我們的絕大多數患者實際上都在專業經銷商處，這對我們來說是盲目的。

We actually don't see the characteristics there, and so for me to speculate on what they will end up being when the vast majority of that data is blind to us, I think would -- I just don't want to say something that doesn't end up being correct, and so I don't think we have enough visibility to make a comment on that.

我們實際上並沒有看到那裡的特徵，因此，當絕大多數數據對我們來說都是盲目的時，我推測它們最終會是什麼樣，我認為——我只是不想說一些最終不正確的話，所以我認為我們沒有足夠的能見度來對此發表評論。

Got it. That's fair, and can you tell us about the expected data at the Lung Cancer Conference and ESMO? What can we expect to see? Will it include the updated data cutoff for PFS and duration of response from TRUST-I and TRUST-II studies, and could this lead to any changes to the label and its use?

知道了。這很公平，您能告訴我們肺癌會議和 ESMO 的預期數據嗎？我們能期待看到什麼？它是否會包括 TRUST-I 和 TRUST-II 研究的 PFS 和反應持續時間的更新數據截止值，這是否會導致標籤及其使用發生任何變化？

So at World Lung, we're going to provide an update on the TRUST-I and TRUST-II that we used for our NDA submission, both on the efficacy and safety side, but we have not yet decided whether we're going to do another cut of the data, so I don't know when that will be.

因此，在世界肺臟大會上，我們將提供 NDA 提交的 TRUST-I 和 TRUST-II 的更新，包括功效和安全性方面，但我們尚未決定是否要再次對數據進行審查，所以我不知道什麼時候會是那樣。

At ESMO, we're going to provide additional data on patients treated with IBTROZI following entrectinib, and that's important, as you know, because entrectinib is one of the first gen TKIs that does cross the blood-brain barrier, and so we're going to show data on what IBTROZI does in patients following entrectinib treatment, so I think that'll be important.

在 ESMO，我們將提供更多關於接受恩曲替尼治療後接受 IBTROZI 治療的患者的數據，這一點很重要，因為恩曲替尼是第一代能夠穿過血腦屏障的 TKI 之一，因此我們將展示關於 IBTROZI 對接受恩曲替尼治療的患者的作用的數據，所以我認為這很重要。

Got it. I appreciate it. Thank you, and congrats again.

知道了。我很感激。謝謝，再次恭喜。

Soumit Roy, Jones Research.

蘇米特·羅伊（Soumit Roy），瓊斯研究公司。

Congratulations on a solid start. Curious, when you're talking to the community center physicians, are you seeing any direct effect by IBTROZI being the preferred [MCCN] agent? And the second is -- yeah, are you getting any sense from the ground, like, RNA testing, if the academic or community centers are thinking about it, when do you think this could start picking up?

恭喜您有一個好的開始。好奇的是，當您與社區中心的醫生交談時，您是否看到 IBTROZI 作為首選 [MCCN] 藥物有任何直接影響？第二點是——是的，您是否從實際情況中了解到，例如 RNA 檢測，如果學術機構或社區中心正在考慮這個問題，您認為什麼時候可以開始普及？

Okay. So on the first question, yes, we've spent a fair amount of time on the road talking to KOLs and also, the management at aggregators around the country, and I would say, we said in our script that we believe that the rapid inclusion into the MCCN guidelines in literally a week is quite unusual, and we think it speaks to what an important option this drug is for patients and how we believe our profile is differentiated.

好的。因此，關於第一個問題，是的，我們花了相當多的時間在路上與 KOL 以及全國各地聚合器的管理層交談，我想說，我們在腳本中說過，我們認為在短短一周內迅速納入 MCCN 指南是非常不尋常的，我們認為這說明了這種藥物對患者來說是一個多麼重要的選擇，以及我們認為我們的產品有何不同。

And we've had that echoed by most, virtually all the people we've spoken to on the road. So we've spoken to quite a few thought leaders, quite a few practices, quite a few aggregators, and we're hearing the same thing. So I think that it's a pretty consistent message we're receiving, and it makes us feel good because we believe this drug is differentiated and really important for patients.

我們在路上採訪過的大多數人，幾乎所有人都有同樣的感受。因此，我們與許多思想領袖、許多實踐者、許多聚合者進行了交談，我們聽到了同樣的事情。所以我認為我們收到的訊息非常一致，這讓我們感到高興，因為我們相信這種藥物與眾不同，對患者來說非常重要。

With regard to RNA testing, we do see a lot more people talking about moving there. I think that there's a recognition that genetic testing is really important because lung cancer only a few decades ago, especially when the association with smoking was first discovered, was really thought to be a death sentence.

關於 RNA 檢測，我們確實看到越來越多的人談論搬到那裡。我認為人們意識到基因檢測非常重要，因為僅僅幾十年前，尤其是當肺癌與吸菸首次被發現有關時，人們還認為它無異於死刑。

And suddenly, really in the last -- in a few decades, lung cancer, especially the ones that have genetic mutations like EGFR, ALK, RET, and now ROS1, have suddenly become some of the most treatable cancers on Earth.

突然之間，在過去的幾十年裡，肺癌，尤其是那些具有 EGFR、ALK、RET 和現在的 ROS1 等基因突變的肺癌，突然成為地球上最可治療的癌症之一。

And so it's really important to identify those mutations, and if you can identify it with a more sensitive technique like RNA testing versus DNA testing, I think it's really becoming rapidly more appreciated how important that is to find.

因此，識別這些突變非常重要，如果你能用更敏感的技術（如 RNA 測試而不是 DNA 測試）來識別它，我認為人們很快就會意識到發現它的重要性。

So we are seeing people talking about a switch to RNA. You never can predict how fast that'll happen, but we are hearing people recognize that and talk about that. I do anticipate a switch to RNA. I just can't predict the time frame.

因此我們看到人們正在談論轉向 RNA。你永遠無法預測這會以多快的速度發生，但我們聽到人們認識到這一點並談論這一點。我確實期待轉向 RNA。我只是無法預測時間框架。

Is that something, that kind of information you would provide us in, like, a couple quarters from now as you start seeing RNA versus DNA testing? Just curious.

當您開始看到 RNA 與 DNA 測試的對比時，您會在幾個季度後向我們提供這類資訊嗎？只是好奇。

If we see some more data that we can speak to, I'm really delighted to talk about that. I don't know how visible that will be to us, because we're not the makers of the test, and we don't really control that data. So if we can point out any data that comes, that's brought to our attention, we'd be happy to do that. But I don't know how much control we'll have over that data.

如果我們看到更多可以談論的數據，我會非常高興地談論它。我不知道這對我們來說有多明顯，因為我們不是測試的製定者，我們實際上也無法控制這些數據。因此，如果我們可以指出任何引起我們注意的數據，我們會很樂意這樣做。但我不知道我們對這些數據的控製程度有多大。

Totally understandable. One last question on safusidenib. If you can talk us through the decision making to switch to the maintenance setting, and is the enrollment criteria expanding to all mutations, all types of IDH1 mutations?

完全可以理解。關於沙夫西尼的最後一個問題。您能否向我們介紹轉換到維護設定的決策過程，以及入組標準是否擴展到所有突變、所有類型的 IDH1 突變？

So our drug, safusidenib, is an IDH1 inhibitor, and IDH1 is 95%-plus of glioma -- IDH1-mutated glioma. So we're not an IDH2 drug, but as I said, the very vast majority of these tumors are IDH1-mutated. So that's what we're going to be focused on.

因此，我們的藥物 safusidenib 是一種 IDH1 抑制劑，而 IDH1 佔神經膠質瘤（IDH1 突變神經膠質瘤）的 95% 以上。因此，我們不是 IDH2 藥物，但正如我所說，絕大多數的腫瘤都是 IDH1 突變。這就是我們要關注的重點。

No, I was meaning like [R132HC], is that expanding to the other subtypes, [GLS]?

不，我的意思是像[R132HC]那樣，它是否擴展到其他亞型，[GLS]？

Yeah, we're targeting all of them.

是的，我們的目標就是他們所有人。

Leonid Timashev, RBC Capital Markets.

加拿大皇家銀行資本市場 (RBC Capital Markets) 的 Leonid Timashev。

Hey, guys. It's Anish on for Leo. Congrats on the progress this quarter and for taking our question. Just a quick one from us. Would you tell us how many of the 70 patients are from clinical trials on a free drug program on EAP versus paid drug commercial patients? Just if you could give us the breakdown there.

嘿，大家好。阿尼什 (Anish) 取代利奧 (Leo)。恭喜您本季的進展並回答我們的問題。我們只想簡單說一下。您能告訴我們這 70 名患者中有多少是來自 EAP 免費藥物計畫臨床試驗的患者，有多少是來自付費藥物商業患者的？如果您能提供我們詳細情況就好了。

Yeah, so 90% of those were not EAP patients. As I said, we only had six patients on the EAP program. And I would say the majority of patients are paying patients. The number of patients on a free drug is still a relatively small minority. And we've kept those patients on free drug to convert to full commercial drug by the next month.

是的，其中 90% 不是 EAP 患者。正如我所說，我們只有六名患者參加 EAP 計劃。我想說大多數病人都是付費病人。享受免費藥物的患者數量仍然相對較少。我們將繼續為這些患者提供免費藥物，下個月將轉為完全商業化藥物。

Yaron Werber, Cowen.

亞倫·韋伯，考恩。

Great. Congrats on this side. And I don't know if you can hear this fire alarm in the background. I have three questions. Maybe the first one, what's the percentage of (technical difficulty) who came or converting from clinical studies? And if you can remind us in the [ENTRUST] program, how many patients were in the US? I thought you had sort of more than 130 patients in the US, but I could be wrong. And then in terms of the one sum, your partner in Japan gets approval for a [sufa], would you record the $25 million as a revenue line or is that below the line in your amortized payment? Thank you.

偉大的。這邊恭喜一下。我不知道你是否能聽到背景中的火警警報。我有三個問題。也許是第一個問題，來自臨床研究或從臨床研究轉變而來的人員比例是多少（技術難度）？您能否提醒我們一下，[ENTRUST] 計劃中美國有多少患者？我以為美國有超過 130 名患者，但我可能錯了。然後就這筆款項而言，當您在日本的合作夥伴獲得 [sufa] 批准時，您會將 2500 萬美元記錄為收入線還是低於您的攤銷付款線？謝謝。

Okay, Yaron, I'm sorry. I could not hear the first part of your question. Could you repeat that?

好的，亞倫，我很抱歉。我聽不清楚你問題的第一部分。你能再說一次嗎？

Yeah, sorry. I got a fire alarm. Stop. So what percentage of the 70 patients were clinical study patients? And then how many patients in ENTRUST came from the US?

是的，抱歉。我收到了火警警報。停止。那麼 70 名患者中有多少比例是臨床研究患者？那麼 ENTRUST 中有多少患者來自美國？

So the number of patients, there were zero patients from clinical trials. There were not clinical study patients, none of them.

因此，就患者數量而言，臨床試驗中的患者為零。沒有臨床研究患者，一個也沒有。

Okay, great. And then just remind us, how many patients did you have in the US in the ENTRUST program?

好的，太好了。然後提醒我們一下，在美國，ENTRUST 計劃中有多少患者？

135 from Europe, North America, and 50 from the US.

其中 135 人來自歐洲和北美，50 人來自美國。

All right, so 135 were US, Europe, and 50 were from the US.

好的，其中 135 人來自美國、歐洲，50 人來自美國。

One would imagine those will convert to commercial product, right? You don't guarantee them jobs for life under clinical study, is that correct?

人們會想像這些會轉化為商業產品，對嗎？您不能保證他們在臨床研究中終身就業，對嗎？

Can you repeat that again? You're kind of garbled, sorry.

你能再說一次嗎？你有點混亂了，抱歉。

Yeah, sorry, it's a bad reception. The patients who are in clinical studies, one would imagine they will convert to commercial product, right? Or do they have sort of unrestricted clinical study drug until they get reimbursed?

是的，很抱歉，接待不太好。人們會想像，參與臨床研究的患者會轉向商業產品，對嗎？或者他們是否有某種不受限制的臨床研究藥物直到他們獲得報銷？

Well, we are tracking these. The DOR of this drug is so long that it is to our benefit to continue to track them and record the duration of response. So they'll stay on trial for a long period. I mean, the longer, the better for us. And we have so many patients who still are on drug and haven't progressed. So we just, it's really in our interest to keep them on trial as long as we can.

嗯，我們正在追蹤這些。這種藥物的 DOR 很長，因此繼續追蹤它們並記錄反應持續時間對我們有益。因此他們將接受長期審判。我的意思是，時間越長，對我們越好。我們有許多患者仍在服用藥物，但病情沒有改善。因此，我們只是盡可能長時間地對他們進行審判，這確實符合我們的利益。

Got it. And I just thought the question in terms of the milestones from the Japanese approval. Would you record that as revenues or revenues? Would you record that as revenues or that's going to obviously hit your inclusive cash position, but it'll be amortized?

知道了。我只是從日本批准的里程碑角度思考了這個問題。您會將其記錄為收入或收益嗎？您會將其記錄為收入嗎？或者這顯然會影響您的現金狀況，但會被攤提？

Yeah, let me take that, Yaron. So we will assess if there are remaining performance obligations, but at that stage, we expect it to recognize a response in revenue line.

是的，讓我來接受，亞倫。因此，我們將評估是否還有剩餘的履約義務，但在那個階段，我們預計它將在收入線中確認回應。

And that's the second half this year, would you say?

您說這就是今年下半年的情況嗎？

That would be, yeah, later part of the year, like we said in the call.

是的，就像我們在電話中說的那樣，那將是在今年晚些時候。

David Nierengarten, Wedbush Securities.

大衛‧尼倫加滕 (David Nierengarten)，韋德布希證券公司 (Wedbush Securities)。

Most of my commercial-related questions have been asked. So one on something. Did the FDA recommend the addition of the maintenance evaluation or look at your arm, whatever you want to call it in the study? I'm just wondering on registration designs, if you're thinking of a typical PFS study or some other metric to measure in that study.

我的大部分商業相關問題都已經被問過了。所以就某件事而言。FDA 是否建議增加維護評估或檢查您的手臂（無論您在研究中想如何稱呼它）？我只是想知道關於註冊設計，您是否正在考慮典型的 PFS 研究或在該研究中測量的其他指標。

Yeah, so we're still in discussions with the FDA. So once we've concluded that, we'll put all of that out on clinicaltrials.gov. The FDA did not make that recommendation. That was our decision. But when we get more visibility and finish those discussions, we'll make that all public on clinicaltrials.gov.

是的，我們仍在與 FDA 進行討論。因此，一旦我們得出結論，我們會將所有內容發佈到clinicaltrials.gov網站。 FDA並沒有提出這項建議。這是我們的決定。但是，當我們獲得更多關注並完成這些討論時，我們會在 clinicaltrials.gov 上公開所有內容。

Silvan Tuerkcan, Citizens.

Silvan Tuerkcan，公民。

Congrats on the first quarter with the launch here. Just a question. Since the changes to the NCCN guidelines that require switching to a ROS1 agent from upon finding that mutation, and obviously, you have small patient numbers here, but have you seen any switching from, for example, pembro or any other checkpoint inhibitor to frontline IBTROZI in any of your patients? And then I have a follow-up.

恭喜第一季的發布。只是一個問題。自從 NCCN 指南發生變化，要求在發現突變後改用 ROS1 藥物，顯然，您這裡的患者數量很少，但是您是否看到任何患者從例如 pembro 或任何其他檢查點抑製劑轉換為一線 IBTROZI？然後我有一個後續問題。

I think that's really hard for us to know. I think that we've spoken previously about the pretty wide recognition that you look at the PFS of biochemo, we're still talking about a PFS of 6 to 12 months. And if you look at our duration response, and we're just now approaching four years and still counting, I think it's going to become increasing more difficult to justify anything other than a ROS1 agent for a ROS1 cancer.

我認為這對我們來說真的很難知道。我認為我們之前已經討論過，人們對生物化學治療的 PFS 的認識相當廣泛，我們仍在討論 6 至 12 個月的 PFS。如果你看一下我們的持續時間反應，我們現在已經接近四年了，而且還在繼續計算，我認為除了 ROS1 藥物之外，任何其他藥物治療 ROS1 癌症的合理性都將變得越來越困難。

So I think the NCCN guidelines are consistent with that. [IO] is now contraindicated in ROS1 lung cancer. And I think that we've already seen in a number of large aggregators, we've seen that contraindication now incorporated into their own internal guidelines. So that's going to make it very difficult for anything other than a ROS1 agent to be prescribed for those patients, which is certainly the right thing to do clinically.

所以我認為NCCN指南與此一致。 [IO]目前禁用於ROS1肺癌。我認為我們已經在許多大型聚合器中看到，禁忌症現在已納入他們自己的內部指南中。因此，除了 ROS1 藥物之外，給這些患者其他藥物都會變得非常困難，但從臨床上來說，這無疑是正確的做法。

Great. Thank you. And then on safu here, obviously FDA discussion is still ahead of you, but maybe big picture, what's your thinking about high-grade and low-grade pivotal plans? Is there any way you can maybe file with data on hand in the high-grade or two pivotal trials, or would it be a joint pivotal trial?

偉大的。謝謝。然後關於安全問題，顯然 FDA 的討論仍在進行中，但也許從大局來看，您對高級和低級關鍵計劃有何看法？您是否可以透過某種方式在高級或兩個關鍵試驗中提交現有數據，或將其作為聯合關鍵試驗？

Thank you. Yeah, so we've made the point that if you look at low-grade glioma, which is where vorasitinib is approved, their response rate is about 11%. In our first study with Daiichi, we showed a low-grade response rate of about 33%, but we have a new trial with Daiichi that's going to be presented sometime later this year. And you'll see for the first time not only a response rate, but for the first time, we'll actually see PFS. So we're excited about that.

謝謝。是的，所以我們指出，如果你看看低度膠質瘤，沃拉西替尼已獲批准，其反應率約為 11%。在我們與第一日本製藥株式會社合作的第一次研究中，我們顯示出約 33% 的低級反應率，但我們與第一日本製藥株式會社將進行一項新的試驗，並將於今年稍後公佈。您不僅會首次看到回應率，而且我們還會首次真正看到 PFS。所以我們對此感到很興奮。

So we think that there are potentially advantages of safusidenib over vorasitinib in the low-grade setting. In the high-grade setting, there is the response rate of vorasitinib is zero. And the response rate of safusidenib, as we mentioned, is 17%. But more interestingly, we're seeing in some cases very durable, deep responses, CRs that go out 2 and 3, 3.5 years.

因此我們認為，在低度腫瘤中，沙夫西替尼比沃拉西替尼具有潛在優勢。在高級環境中，沃拉西替尼的反應率為零。正如我們所提到的，沙夫西地尼的反應率為 17%。但更有趣的是，在某些情況下，我們看到非常持久、深刻的反應，CR 會持續 2 年、3 年或 3.5 年。

So with our FDA discussions, we're really focused on what we need to do to make these trials registrational. And so that's what our discussions are focused on. And when we conclude those discussions, we're going to put those trial designs, you know, make those public on clinicaltrials.gov. And then you'll get an idea of what the end point should be, what our design is.

因此，在與 FDA 的討論中，我們真正關注的是我們需要做什麼才能使這些試驗獲得註冊。這就是我們討論的重點。當我們結束這些討論後，我們會把這些試驗設計公佈在clinicaltrials.gov網站上。這樣你就能知道最終結果是什麼，以及我們的設計是什麼樣的。

And you can also probably have a better estimate of what time frame it would take to get this drug across approval finish line.

而且您可能還可以更好地估計讓這種藥物通過審批需要多長時間。

Congrats again.

再次恭喜。

There are no questions waiting at this time. And I'll pass the conference back over to David for any further remarks.

目前沒有問題。我將把會議交還給大衛，以便他可以發表進一步的評論。

Well, we're very excited about our launch so far. We think it's going extremely well. We are very optimistic about the future. The feedback we've gotten has been really very encouraging. So we'll keep you updated as we get more data in the next quarter. And we're also equally excited about the rest of our pipeline. So stay tuned and we'll talk to you in another quarter. Thanks so much for joining.

嗯，到目前為止，我們對我們的發布感到非常興奮。我們認為一切進展非常順利。我們對未來非常樂觀。我們收到的回饋確實非常令人鼓舞。因此，我們將在下個季度獲得更多數據時向您通報最新情況。我們對其餘的產品線也同樣感到興奮。請繼續關注，我們將在下一季與您交談。非常感謝您的加入。

Thank you. That concludes today's call. Thank you for your participation. You may now disconnect your line.

謝謝。今天的電話會議到此結束。感謝您的參與。現在您可以斷開線路了。