Syndax Pharmaceuticals Inc (SNDX) 2024 Q2 法說會逐字稿

Good day, everyone, and welcome to the Syndax second quarter 2024 earnings conference call. Today's call is being recorded. (Operator Instructions)

大家好，歡迎參加 Syndax 2024 年第二季財報電話會議。今天的通話正在錄音。（操作員說明）

At this time, I would like to turn the call over to Sharon Klahre, Head of Investor Relations at Syndax Pharmaceuticals.

現在，我想將電話轉給 Syndax Pharmaceuticals 投資者關係主管 Sharon Klahre。

Thank you, operator. Welcome, and thank you all for joining us today for a review of Syndax's second quarter 2024 financial and operating results. I'm Sharon Klahre, and with me this afternoon to provide an update on the company's progress and discuss financial results are Michael Metzger, Chief Executive Officer; Dr. Neil Gallagher, President and Head of R&D; Steve Closter, Chief Commercial Officer; and Keith Goldan, Chief Financial Officer. Also joining us on the call today for the question-and-answer session are Dr. Peter Ordentlich, Chief Scientific Officer; and Dr. Anjali Ganguli, Chief Business Officer.

謝謝你，接線生。歡迎並感謝大家今天加入我們，回顧 Syndax 的 2024 年第二季財務與營運表現。我是 Sharon Klahre，今天下午與我一起介紹公司最新進展並討論財務業績的是執行長 Michael Metzger； Neil Gallagher 博士，總裁兼研發主管；史蒂夫‧克洛斯特，首席商務官；和財務長基思戈爾丹。首席科學官 Peter Ordentlich 博士也參加了今天的電話問答環節。以及首席商務官 Anjali Ganguli 博士。

This call is accompanied by a slide deck that has been posted on the Investor page of the company's website. You can now turn to our forward-looking statements on Slide 2. Before we begin, I would like to remind you that any statements made during the call that are not historical are considered to be forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995.

這次電話會議還附有幻燈片，該幻燈片已發佈在該公司網站的投資者頁面上。現在您可以參閱投影片 2 上的前瞻性陳述。在開始之前，我想提醒您，電話會議期間所做的任何非歷史性陳述均被視為 1995 年《私人證券訴訟改革法案》含義內的前瞻性陳述。

Actual results may differ materially from those indicated by these statements as a result of various important factors, including those discussed in the risk factors section in the company's most recent quarterly reports on Form 10-Q as well as other reports filed with the SEC. Any forward-looking statements made represent our views as of today, August 1, 2024, only. A replay of this call will be available on the company's website, www.syndax.com, following its completion.

由於各種重要因素的影響，實際結果可能與這些聲明中所示的結果有重大差異，包括公司最新 10-Q 表格季度報告以及向 SEC 提交的其他報告中風險因素部分討論的因素。所做的任何前瞻性陳述僅代表我們截至今天（2024 年 8 月 1 日）的觀點。本次電話會議結束後，將在本公司網站 www.syndax.com 上重播。

With that, I'm pleased to turn the call over to Michael Metzger, Chief Executive Officer of Syndax.

至此，我很高興將電話轉給 Syndax 執行長 Michael Metzger。

Thank you, Sharon. Good afternoon, everyone, and thank you for joining us today. Starting with Slide 3. This is a very exciting year for Syndax as we stand on the cusp of becoming a commercial stage company with the anticipated upcoming FDA approvals of revumenib and axatilimab, new first-in-class treatments for diseases with high unmet medical needs.

謝謝你，莎倫。大家下午好，感謝您今天加入我們。從投影片 3 開始。對於 Syndax 來說，這是非常激動人心的一年，因為我們正處於成為商業階段公司的風口浪尖，預計 FDA 即將批准 revumenib 和 axatilimab，這是針對醫療需求未得到滿足的疾病的新型一流療法。

I'm pleased to report that we have achieved several clinical milestones in the second quarter that support our strategic priorities. In June, we presented updated positive revumenib combination data from the BEAT AML and AUGMENT-102 trials at the European Hematology Association, or EHA Congress. These data, which Neil will review shortly, continue to support revumenib's potential to enhance current standard of care agents.

我很高興地報告，我們在第二季度實現了幾個臨床里程碑，以支持我們的策略重點。6 月，我們在歐洲血液學協會 (EHA) 大會上展示了來自 BEAT AML 和 AUGMENT-102 試驗的最新 Revumenib 組合陽性數據。尼爾很快就會審查這些數據，繼續支持瑞維美尼提高目前護理藥物標準的潛力。

At EHA, we also presented additional positive data from the AGAVE-201 pivotal trial evaluating axatilimab in patients with refractory chronic graft versus host disease or GVHD. Reinforcing the previous organ response data that we had shared, this latest data set shows that responses were noted in all fibrosis-dominant organs that had clinical activity supported by clinician reported and patient reported changes in organ specific symptoms such as improvements in swallowing, shortness of breath, skin and joints, and sclerotic skin.

在 EHA 上，我們也展示了 AGAVE-201 關鍵試驗的更多正面數據，該試驗評估了 axatilimab 對難治性慢性移植物抗宿主疾病或 GVHD 患者的療效。這一最新數據集強化了我們先前分享的器官反應數據，顯示在所有以纖維化為主的器官中均出現了反應，這些器官的臨床活動得到了臨床醫生報告的支持，並且患者報告了器官特異性性症狀的變化，例如吞嚥改善、呼吸困難等。

Looking ahead, we are on track for a historic year with the anticipated FDA approval of revumenib and axatilimab coming soon. With axatilimab, our anti CSF-1 antibody for patients with chronic GVHD, we have a PDUFA action date of August 28, 2024 and anticipate launching in the fourth quarter of this year with our partner Incyte.

展望未來，我們將迎來歷史性的一年，預計 FDA 即將批准 revumenib 和 axatilimab。我們針對慢性 GVHD 患者的抗 CSF-1 抗體 axatilimab 的 PDUFA 行動日期為 2024 年 8 月 28 日，並預計與我們的合作夥伴 Incyte 於今年第四季推出。

With revumenib, our menin inhibitor being reviewed by the FDA for patients with relapse or refractory KMT2A-rearranged acute leukemias, we have a PDUFA action date of December 26, 2024. As we announced earlier this week, the FDA recently extended PDUFA action date for revumenib NDA from September 26 to December 26, 2024, a standard three-month extension to allow for additional time to conduct a full review of supplemental information we provided in response to their requests.

FDA 正在審查我們的 menin 抑制劑 revumenib 用於治療復發或難治性 KMT2A 重排急性白血病的患者，我們的 PDUFA 行動日期為 2024 年 12 月 26 日。正如我們本週早些時候宣布的那樣，FDA 最近將revumenib NDA 的PDUFA 行動日期從2024 年9 月26 日延長至12 月26 日，這是一個標準的三個月延期，以便有更多時間對我們為回應而提供的補充資訊進行全面審查他們的要求。

Importantly, we are confident that the information we have provided supports approval and continues to demonstrate the meaningful benefit revumenib brings to patients. We look forward to continuing to closely engage with the FDA as they complete the review under the Real Time Oncology Review Program, or RTOR, and we stand ready to launch revumenib with strength as soon as we receive the anticipated approval.

重要的是，我們相信我們提供的資訊支持批准，並繼續證明瑞維美尼為患者帶來的有意義的益處。我們期待在 FDA 完成實時腫瘤學審查計劃（RTOR）下的審查時繼續與 FDA 密切合作，並且我們準備在收到預期批准後立即強力推出 revumenib。

Later in the call, Steve will provide some additional color on our preparations for these two anticipated launches. We also have several other meaningful milestones approaching, such as the anticipated readout of pivotal top line revumenib data in relapsed or refractory NPM1 AML in the fourth quarter, which could serve as the basis for a supplemental NDA filing in the first half of 2025.

在稍後的電話會議中，史蒂夫將就我們為這兩個預期發布所做的準備工作提供一些額外的資訊。我們還有其他幾個有意義的里程碑即將到來，例如預計在第四季度讀出復發或難治性 NPM1 AML 的關鍵頂線 revumenib 數據，這可以作為 2025 年上半年補充 NDA 備案的基礎。

Additionally, we expect to make further progress advancing our clinical trials of revumenib and axatilimab that are underway or plan to initiate in the coming months. Positive results could support the future use of these agents in combination with standard-of-care medicines earlier in a patient's treatment and thereby expand their utility significantly.

此外，我們預計正在推進或計劃在未來幾個月啟動的 revumenib 和 axatilimab 臨床試驗以取得進一步進展。正面的結果可以支持未來在患者治療早期將這些藥物與標準護理藥物結合使用，從而顯著擴大其效用。

We are in a strong financial position with $455 million in cash as of June 30th that we expect will provide sufficient capital through 2026. Our current balance sheet not only supports our planned commercial launches and clinical trials, but also sets us up to expand beyond our first registration indications and to pursue early-stage business development opportunities.

截至 6 月 30 日，我們的財務狀況強勁，擁有 4.55 億美元現金，我們預計將在 2026 年之前提供充足的資本。我們目前的資產負債表不僅支持我們計劃的商業啟動和臨床試驗，而且還使我們能夠超越我們的首次註冊適應症並尋求早期業務發展機會。

I will now ask Neil to review some of our recent data presentations and pipeline. Neil?

我現在請尼爾回顧我們最近的一些數據演示和管道。尼爾？

Thanks, Michael. Turning to Slide 4, in revumenib, we have a robust clinical development strategy underway that will support the use of revumenib in KMT2A-rearranged and mutant NPM1 acute leukemias across the treatment paradigm. The development plan extends beyond the initial relapsed or refractory indications with trials of revumenib in combination with standards of care that potentially support use of the frontline relapse, refractory and post-transplant maintenance settings.

謝謝，麥可。轉向幻燈片 4，在 revumenib 中，我們正在進行一項強有力的臨床開發策略，該策略將支持在整個治療模式中使用 revumenib 在 KMT2A 重排和突變 NPM1 急性白血病中使用。該開發計劃超越了最初的複發或難治性適應症，將 revumenib 與可能支持一線復發、難治性和移植後維持設置的護理標準相結合的試驗相結合。

On the next two slides, I will briefly review the data recently presented at EHA from the two combination trials that you see listed, BEAT AML and AUGMENT-102. On Slide 5, at EHA in June, we and our collaborators at the Leukemia Lymphoma Society presented updated data from the BEAT AML trial of revumenib in combination with venetoclax, azacitidine or Ven/Aza in patients newly diagnosed with mutant NPM1 or KMT2A-rearranged AML.

在接下來的兩張投影片中，我將簡要回顧最近在 EHA 上展示的來自您看到的列出的兩項組合試驗（BEAT AML 和 AUGMENT-102）的數據。在幻燈片5 上，6 月在EHA 上，我們和白血病淋巴瘤協會的合作者展示了revumenib 聯合Venetoclax、阿扎胞苷或Ven/Aza 在新診斷患有突變NPM1 或KMT2A 重排AML 患者中進行的BEAT AML 試驗的最新數據。

This updated data set with a cut off of the of May 1, 2024, includes 24 efficacy of valuable patients, 96% of patients had a composite complete response for CRc and 92% had no residual disease and were therefore MRD-negative.

此更新資料集截止日期為 2024 年 5 月 1 日，包括 24 名有價值患者的療效，其中 96% 的患者對 CRc 具有複合完全緩解，92% 的患者沒有殘留疾病，因此為 MRD 陰性。

As in all the combination trials conducted so far, two doses of revumenib are being investigated, 113 milligrams and 163 milligrams administered every 12 hours. The dose limiting toxicity or DLT window for each was cleared last year on this trial, and both cohorts are currently being expanded in order to identify the dose for Phase 3 development.

與迄今為止進行的所有聯合試驗一樣，正在研究兩種劑量的 revumenib，每 12 小時給藥 113 毫克和 163 毫克。去年，該試驗的劑量限制毒性或 DLT 窗口已被清除，目前正在擴大這兩個隊列，以確定 3 期開發的劑量。

The combination is well tolerated, and the safety profile reported at EHA is consistent with what has been previously reported. Of note, the rates of adverse events associated with the combination of revumenib with Ven/Aza observed in the study are consistent with data for Ven/Aza from historical controls. For example, the addition of revumenib to the doublet does not appear to exacerbate the modest associated with Ven/Aza alone.

該組合具有良好的耐受性，EHA 報告的安全性與先前報告的一致。值得注意的是，研究中觀察到的與 revumenib 與 Ven/Aza 組合相關的不良事件發生率與歷史對照組中的 Ven/Aza 數據一致。例如，在雙聯體中添加 revumenib 似乎不會加劇與單獨使用 Ven/Aza 相關的適度影響。

Additionally, the BEAT AML investigators concluded that the data suggests that adjusting the dose of venetoclax while maintaining the dose of revumenib may be the most logical strategy for future development. As we previously communicated, we intend to initiate a pivotal trial with this triplet combination in newly diagnosed patients by yearend.

此外，BEAT AML 研究人員得出的結論是，數據表明，在維持 revumenib 劑量的同時調整 Venetoclax 劑量可能是未來開發最合理的策略。正如我們之前所傳達的，我們打算在年底前在新診斷的患者中啟動一項針對這種三聯體組合的關鍵試驗。

Turning to Slide 6, at EHA, we also presented updated data from the AUGMENT-102 trial of revumenib in combination with fludarabine/cytarabine or FLA in a predominantly pediatric population with relapsed refractory mutant NPM1, NUP98-rearranged or KMT2A-rearranged AML.

轉向幻燈片6，在EHA，我們還提供了revumenib 聯合氟達拉濱/阿糖胞苷或FLA 在患有復發難治性突變NPM1、NUP98 重排或KMT2A 重排AML 的主要兒科人群中進行的AUGMENT-102 試驗的最新數據。

As of the data cutoff of 15th of January, 2024, 27 patients received FLAPS as recommended at 113 milligrams or 163 milligrams. Patients included in the trial were heavily pre-treated with a median of three prior lines of therapy. 67% had prior FLAPS-containing regimens. 52% of patients achieved composite complete remission or CRC, the MRD negative rate among CRC patients is 71%. Furthermore, seven patients or 50% of responders proceeded to transplant once they had entered remission.

截至 2024 年 1 月 15 日數據截止，有 27 名患者按照建議接受了 113 毫克或 163 毫克的 FLAPS。參與試驗的患者接受了中位數為三線治療的大量預先治療。 67% 之前接受過包含 FLAPS 的治療方案。 52%的患者達到複合完全緩解或CRC，CRC患者中MRD陰性率為71%。此外，7 名患者或 50% 的反應者在進入緩解期後開始進行移植。

As with the Ven/Aza triplet we just discussed, revumenib does not appear to alter the tolerability profile of the standard of care regimen. These two updated data sets presented at EHA provide further support for combining revumenib with different standards of care in the newly diagnosed as well as relapsed or refractory settings.

與我們剛剛討論的 Ven/Aza 三聯體一樣，revumenib 似乎不會改變標準護理方案的耐受性。EHA 上發布的這兩個更新的數據集為在新診斷以及復發或難治性情況下將 revumenib 與不同護理標準相結合提供了進一步的支持。

Turning to Slide 7, in March, we completed enrollment in the pivotal cohort of the AUGMENT-101 trial with the enrollment of 64 adults and up to 20 pediatric relapsed refractory NPM1 mutant AML patients. We are on track -- our acute leukemia patients. We are on track to report data in the fourth quarter.

轉向幻燈片 7，3 月份，我們完成了 AUGMENT-101 試驗關鍵隊列的入組，招募了 64 名成人和最多 20 名兒童復發難治性 NPM1 突變 AML 患者。我們的急性白血病患者正在步入正軌。我們預計在第四季度報告數據。

On this slide, you can see the results from 14 mutant NPM1 patients in the Phase 1 portion of AUGMENT-101, which showed that 50% of patients achieved a response and 36% achieved a complete remission or CR, with partial hematological recovery.

在這張投影片上，您可以看到AUGMENT-101 1 期部分14 名突變NPM1 患者的結果，其中顯示50% 的患者實現了緩解，36% 的患者實現了完全緩解或CR，並有部分血液學恢復。

Notably, all patients with the CRC rates were MRD negative. And consistent with the KMT2A population, revumenib also enabled patients in remission to proceed to transplant with a durable response despite many of the patients having failed prior venetoclax therapy and cell transplant. These data also indicate that revumenib is well tolerated in patients with mutant NPM1 with an overall safety portfolio that is consistent with what has been previously reported in the KMT2A-rearranged population.

值得注意的是，所有 CRC 患者的 MRD 均為陰性。與 KMT2A 族群一致，儘管許多患者先前的 Venetoclax 治療和細胞移植失敗，但 Revumenib 也使緩解期患者能夠繼續進行移植，並獲得持久的反應。這些數據還表明，revumenib 在 NPM1 突變患者中具有良好的耐受性，其整體安全性組合與先前在 KMT2A 重排族群中報告的一致。

Before I hand over to Steve, I'd like to provide a quick overview on Slide 8 of the preparations we've made and the medical side of the organization to support the upcoming anticipated launches. We've built a highly experienced medical affairs team that is driving deep scientific engagement and robust evidence generation.

在交給 Steve 之前，我想在幻燈片 8 上快速概述我們所做的準備工作以及該組織為支持即將到來的預期發布而進行的醫療方面的工作。我們建立了一支經驗豐富的醫療事務團隊，推動深入的科學參與和強有力的證據生成。

As you've seen from our participation in ASH and EHA as well as various papers and top tier journals such as Nature, we have an ambitious conference and publication plan underway to increase awareness of the compelling data from our two agents.

正如您從我們參與ASH 和EHA 以及各種論文和《自然》等頂級期刊中看到的那樣，我們正在進行一項雄心勃勃的會議和出版計劃，以提高人們對我們兩個代理商的令人信服的數據的認識。

We also continue to prepare for NCCN guideline submissions for revumenib that we anticipate will include the prescribing information as well as peer-review publications and congress presentation. Of note, once the top line data in mutant NPM1 AML are available, we plan to publish as soon as it's feasible to support potential guideline inclusion following the approval of revumenib and KMT2A.

我們也繼續準備向 NCCN 提交 revumenib 指南，我們預計其中將包括處方資訊以及同行評審出版物和大會演示。值得注意的是，一旦獲得突變型 NPM1 AML 的主要數據，我們計劃在可行的情況下盡快發布，以支持在 revumenib 和 KMT2A 獲得批准後潛在的指南納入。

Additionally, we have a team of seasoned medical science liaisons in the field. They've made tremendous progress driving scientific exchange, which positions major centers of excellence across the country. We've also built a health economics and real-world evidence team focused on supporting the value of our portfolio, and we continue to increase engagement with patient advocacy groups.

此外，我們在該領域擁有一支經驗豐富的醫學科學聯絡團隊。他們在推動科學交流方面取得了巨大進展，為全國各地的主要卓越中心奠定了基礎。我們還建立了一個健康經濟學和現實世界證據團隊，專注於支持我們產品組合的價值，並繼續加強與患者倡導團體的接觸。

With that, I'll now turn it over to Steve to talk about our commercial launch plans and the market opportunities. Steve?

現在，我將把它交給史蒂夫來討論我們的商業發布計劃和市場機會。史蒂夫？

Thank you, Neil, and thanks, everyone, for joining us today. Really appreciate it. It's really a pleasure to be with you to talk about the extensive preparations we're making to position Syndax as the leading commercial stage organization serving patients and healthcare providers. We are well prepared to execute on the exciting opportunities in front of us.

謝謝尼爾，也謝謝大家今天加入我們。真的很感激。很高興與您一起談論我們為將 Syndax 定位為服務患者和醫療保健提供者的領先商業階段組織所做的廣泛準備工作。我們已做好充分準備，抓住眼前令人興奮的機會。

Turning to revumenib preparations in Slide 8. Our goal simply is a strong launch, and we are ready to hit the ground running with revumenib as soon as we receive the anticipated approval from FDA. We've hired and trained a sales team with extensive experience and pre-existing relationships in the hematology-oncology space and a demonstrated track record of success.

轉向幻燈片 8 中的 revumenib 製劑。我們的目標只是大力推出，一旦收到 FDA 的預期批准，我們就準備好立即啟動 revumenib。我們聘請並培訓了一支銷售團隊，他們在血液腫瘤學領域擁有豐富的經驗和現有的關係，並且擁有良好的成功記錄。

This team is in the field right now profiling accounts and understanding the patient treatment journey so we can meet the needs of the different stakeholders involved in their care. This robust pre-launch preparation will let us rapidly begin meeting patient needs upon the anticipated approval.

團隊目前正在現場分析帳戶並了解患者的治療歷程，以便我們能夠滿足參與其護理的不同利害關係人的需求。這種強有力的上市前準備工作將使我們能夠在獲得預期批准後迅速開始滿足患者的需求。

Our customer-facing team has an average of 22 years of experience, primarily in hematology, oncology and an average of six product launches each. With an efficient field force footprint in the range of 30 to 50 individuals, we believe that we can effectively reach the relevant academic and community-based centers, and meet the needs of physicians and patients.

我們面向客戶的團隊平均擁有 22 年的經驗，主要在血液學、腫瘤學領域，平均每個領域有 6 個產品發表。憑藉 30 至 50 人的高效現場人員足跡，我們相信我們能夠有效到達相關學術和社區中心，並滿足醫生和患者的需求。

We plan to call on approximately 2,000 centers with roughly 200 of those accounts representing more than two-thirds of the opportunity, enabling a concentrated effort. With our plan, we believe we should reach centers with 98% or more of potential relapsed/refractory KMT2Ar patients receive treatment.

我們計劃拜訪大約 2,000 個中心，其中大約 200 個帳戶代表了三分之二以上的機會，從而能夠集中精力。根據我們的計劃，我們相信我們應該讓 98% 或更多的潛在復發/難治性 KMT2Ar 患者接受治療。

Now another important thing we've done in preparation for the launch is develop advanced data mining capabilities to appropriately identify patients need. Because these are high-risk patients that require rapid identification of the treatment options, we've developed capabilities that will help us initiate very targeted physician engagement based on where our tools indicate there are appropriate patients.

現在，我們為發布做準備的另一件重要事情是開發先進的資料探勘功能，以正確識別患者的需求。由於這些患者是高風險患者，需要快速識別治療方案，因此我們開發了一些功能，可以幫助我們根據我們的工具表明存在合適患者的位置，啟動非常有針對性的醫生參與。

Now with respect to market access, we've built an accomplished team with extensive experience working with payers and other trade partners to facilitate access to new products. Together with the medical affairs team, our payer field team continues their pre-approval information exchange work with payers, and we're on track to reach plan, covering more than 90% of all covered lives both commercial and [Part B] prior to the anticipated approval.

現在，在市場准入方面，我們已經建立了一支經驗豐富的團隊，在與付款人和其他貿易夥伴合作以促進新產品的准入方面擁有豐富的經驗。我們的付款人現場團隊與醫療事務團隊一起繼續與付款人進行批准前資訊交換工作，我們預計將實現計劃，涵蓋 90% 以上的商業和 [B 部分] 之前的所有承保生活預期的批准。

Payers tell us that they recognize the unmet need and appreciate the value that revumenib provides. We believe plans will make their formulary decisions within 6 to 12 months of approval, and we'll work with them to expedite the review when possible.

付款人告訴我們，他們認識到未滿足的需求並欣賞 revumenib 提供的價值。我們相信計劃將在批准後 6 至 12 個月內做出正式決定，我們將與他們合作，盡可能加快審查速度。

Importantly, given the urgent patient needs, we expect that plans will provide patients access to the product at launch through the medical exception process to support providers and patients we've partnered with leading best-in-class specialty pharmacies who are well recognized for their ability to help providers and patients navigate access to new oncology medicines.

重要的是，考慮到患者的迫切需求，我們預計該計劃將透過醫療例外流程為患者提供在產品推出時使用該產品的機會，以支持我們與領先的一流專業藥房合作的提供者和患者，這些藥房因其幫助提供者和患者獲得新的腫瘤藥物的能力。

Through a network of specialty pharmacies and specialty distributors, we're prepared to have product and channel very quickly right after we receive approval. We're also ready to launch a dedicated patient support program that will provide a level of support on par with what you see from leading oncology company.

透過專業藥局和專業經銷商網絡，我們準備在獲得批准後立即提供產品和通路。我們還準備啟動專門的患者支援計劃，該計劃將提供與您從領先的腫瘤學公司看到的同等水平的支援。

Turning to Slide 9 in the market opportunity. KMT2Ar and NPM1 acute leukemia represent up to 40% of all AML patients. There are no FDA approved targeted therapies for this population. We believe relapsed or refractory KMT2Ar acute leukemia alone represents a total addressable market opportunity of approximately $750 million in the US.

轉向投影片 9 的市場機會。KMT2Ar 和 NPM1 急性白血病佔所有 AML 患者的比例高達 40%。目前尚無 FDA 批准針對該族群的標靶療法。我們相信，僅復發性或難治性 KMT2Ar 急性白血病在美國就代表了約 7.5 億美元的潛在市場機會。

The annual incidence of KMT2Ar acute leukemia is about 2,600 patients with the majority of these patients, about 2,000 experiencing relapsed or refractory disease. We estimate a median duration of therapy across the treated population of approximately nine months. And we believe the clinical data supports pricing competitively to other targeted therapies in AML, such as FLIP3 or IDH inhibitors.

KMT2Ar急性白血病每年發病約2,600名患者，其中大多數患者，約2,000名患有復發或難治性疾病。我們估計治療族群的中位治療持續時間約為九個月。我們相信，臨床數據支持其定價與其他 AML 標靶療法（例如 FLIP3 或 IDH 抑制劑）相比具有競爭力。

Physicians we've spoken with indicate an eagerness to prescribe revumenib early during an eligible patients treatment journey to bring more patients to transplant and then extend responses by continuing with revumenib monotherapy following transplant engraftment.

我們訪談的醫生表示，他們渴望在符合條件的患者治療過程中儘早開出瑞維美尼(revumenib) 處方，以便讓更多患者接受移植，然後在移植後繼續使用瑞維美尼(revumenib)單藥治療來延長療效。

We expect that our first mover advantage and the early experience physicians will gain treating patients with revumenib will be vitally important for the long-term success of our brand. Our significant market share is likely to extend meaningfully beyond KMT2Ar, especially as we will be the first to deliver meaningful pivotal data in other indications, such as NPM1 AML.

我們預計，我們的先發優勢和醫生在使用瑞維美尼治療患者時獲得的早期經驗對於我們品牌的長期成功至關重要。我們重要的市場佔有率可能會有意義地擴展到 KMT2Ar 之外，特別是因為我們將成為第一個在其他適應症（例如 NPM1 AML）中提供有意義的關鍵數據的公司。

We estimate that the two distinct market segments in acute leukemias, KMT2Ar and NPM1 equal a combined accessible population of 5,000 to 6,500 patients in the relapsed or refractory setting and an addressable market opportunity that approaches $2 billion in the US.

我們估計，急性白血病的兩個不同細分市場 KMT2Ar 和 NPM1 相當於 5,000 至 6,500 名復發或難治性患者的總可用人群，以及在美國接近 20 億美元的潛在市場機會。

During the various meetings that I've had the chance to participate in with clinicians, from advisory boards to field business and more, I've seen tremendous excitement and support for revumenib creating a momentum that I look forward to building on to our commitment to delivering an absolute best-in-class experience for healthcare providers and patients. Moving to axatilimab on Slide 10.

在我有機會與臨床醫生一起參加的各種會議中，從諮詢委員會到現場業務等，我看到了對revumenib 的巨大興奮和支持，這創造了一種勢頭，我期待著在我們的承諾的基礎上再接再厲。前往幻燈片 10 上的 axatilimab。

The anticipated commercialization of axatilimab will be led by the Incyte team and we'll benefit from the deep experience and long-standing relationship that they've established through their work, building the GVHD market with Jakafi. We'll provide 30% of the sales effort, leveraging our own field force that we anticipate will carry two products with highly overlapping call points.

axatilimab 的預期商業化將由 Incyte 團隊領導，我們將受益於他們透過工作建立的豐富經驗和長期關係，與 Jakafi 共同打造 GVHD 市場。我們將提供 30% 的銷售工作，利用我們自己的現場人員，我們預計他們將攜帶兩種呼叫點高度重疊的產品。

Paved the way for a successful launch, Incyte is continuing to drive engagement with payers and raise awareness of the distinct pathway that axatilimab targets and the compelling outcomes observed in the AGAVE-201 trial, which we recently detailed at the 2023 ASH Congress.

為成功推出鋪平了道路，Incyte 正在繼續推動與付款人的互動，並提高人們對axatilimab 靶向的獨特途徑以及AGAVE-201 試驗中觀察到的引人注目的結果的認識，我們最近在2023 年ASH 大會上詳細介紹了這項試驗。

Turning to Slide 11. We estimate there are approximately 17,000 patients on treatment for chronic GVHD at any one time, the majority of whom are refractory and cycle through therapies for better symptom control as their disease progresses. We believe there are approximately 6,500 patients progressing to later lines of treatment after two previous lines of treatment, which would be our target population for our first indication and represents an attractive initial opportunity.

轉到投影片 11。我們估計，任何時候都有大約 17,000 名慢性 GVHD 患者接受治療，其中大多數患者是難治性的，隨著疾病的進展，需要循環治療以更好地控制症狀。我們相信，大約有 6,500 名患者在接受了前兩線治療後，正在進入後續治療線，這將是我們第一個適應症的目標人群，並且代表著一個有吸引力的初始機會。

For instance, in the three years since the launch of REZUROCK and other drug with a third line indication, net sales continue to grow, and they're annualizing at nearly $500 million. We estimate that the total addressable market for third line treatment in the US is between $1.5 billion to $2 billion, which assumes that patients will remain on therapy for over 12 months and assuming axatilimab is priced at a premium to approved agents for chronic GVHD, based on its product profile and Part B reimbursement.

例如，自 REZUROCK 和其他第三線適應症藥物推出以來的三年裡，淨銷售額持續成長，年銷售額接近 5 億美元。我們估計，美國第三線治療的潛在市場總額在 15 億至 20 億美元之間，假設患者將繼續接受治療超過 12 個月，並假設 axatilimab 的定價高於已批准的慢性 GVHD 藥物，基於關於其產品簡介和B 部分報銷。

Beyond the third line setting, we plan to study axatilimab in earlier line settings for chronic GVHD and other diseases where we believe its anti-fibrotic and anti-inflammatory mechanism is relevant, such as IPF, which represents a large opportunity.

除了三線治療之外，我們還計劃在慢性 GVHD 和其他我們認為其抗纖維化和抗發炎機制相關的疾病（例如 IPF）的早期治療中研究 axatilimab，這代表著一個巨大的機會。

I'll now turn the call over to Keith to review our financial results.

我現在將電話轉給基思，以審查我們的財務表現。

Thank you, Steve. Turning to Slide 12. As Michael mentioned earlier, the $455 million in cash equivalents and short and long-term investments on our balance sheet as of June 30, we expected to provide runway through 2026. Our financial strength allows us to fund the anticipated commercialization of two drugs and appropriately invest to continue to realize the value of our pipeline.

謝謝你，史蒂夫。轉到投影片 12。正如邁克爾之前提到的，截至 6 月 30 日，我們資產負債表上的 4.55 億美元現金等價物以及短期和長期投資預計將提供到 2026 年的跑道。我們的財務實力使我們能夠為兩種藥物的預期商業化提供資金，並進行適當投資以繼續實現我們的管道價值。

Turning to the income statement. Operating expenses in the second quarter were $77.7 million and included $48.7 million of research and development expense and $29.1 million of selling, general and administrative expense. Let me provide financial guidance for the third quarter and full year 2024.

轉向損益表。第二季營運費用為 7,770 萬美元，其中包括 4,870 萬美元的研發費用以及 2,910 萬美元的銷售、一般和管理費用。讓我提供 2024 年第三季和全年的財務指引。

For the third quarter, the company expects research and development expense to be $70 million to $75 million and total operating expenses to be $105 million to $110 million. For the full year of 2024, there is no change to the existing guidance, and the company expects research and development expenses to be $240 million to $260 million and total operating expenses to be between $355 million to $375 million.

該公司預計第三季的研發費用為7,000萬至7,500萬美元，總營運費用為1.05億至1.1億美元。2024年全年，現有指引沒有變化，公司預計研發費用為2.4億美元至2.6億美元，總營運費用為3.55億美元至3.75億美元。

Please note that the guidance range for operating expenses for the full year includes an estimated $43 million of noncash stock compensation expense and that research and development expense guidance includes any milestones owed to our partners' potential drug approvals. Ahead of the upcoming launch of axatilimab, I want to briefly review how we will recognize revenue for that partner product.

請注意，全年營運費用指導範圍包括估計 4,300 萬美元的非現金股票補償費用，研發費用指導包括我們合作夥伴潛在藥物批准的任何里程碑。在 axatilimab 即將推出之前，我想簡要回顧一下我們將如何確認該合作夥伴產品的收入。

Slide 13 provides an illustrative example of accounting for sales of axatilimab and is not intended to provide any margin or any other guidance. We will record 50% of the commercial profit, defined as net product revenue minus the cost of sales and commercial expenses. During a period where there is net commercial profit for axatilimab, as in the top example of this slide, our 50% share of the net profit will be recognized on our P&L as collaboration revenue.

投影片 13 提供了 axatilimab 銷售額的說明性範例，並非旨在提供任何利潤或任何其他指引。我們將記錄 50% 的商業利潤，定義為淨產品收入減去銷售成本和商業費用。在 axatilimab 產生淨利潤的時期，如本投影片頂部的範例所示，我們 50% 的淨利潤份額將在我們的損益表中確認為合作收入。

During a period where there is a net commercial loss for axatilimab, as in the example on the bottom of the slide, our 50% share of the net commercial loss would be included in the operating expenses designated as a separate line item called share of collaboration loss. The milestone revenue from various global commercial and regulatory milestones that we received from Incyte will be recorded as a milestone revenue on our income statement.

在 axatilimab 出現淨商業損失的時期（如幻燈片底部的範例所示），我們 50% 的淨商業損失份額將包含在營運費用中，指定為單獨的行項目，稱為合作份額損失。我們從 Incyte 收到的各種全球商業和監管里程碑的里程碑收入將作為里程碑收入記錄在我們的損益表中。

As a reminder, research and development expenses under our partnership, including regulatory and CMC expenses were shared 55-45 in the US, and our 45% share is included in the income statement as part of our R&D expense. Outside of the United States, Incyte is responsible for 100% of the development and regulatory expenses, and we are entitled to receive milestones with a double-digit royalty on ex-US sales.

需要提醒的是，我們合作夥伴關係下的研發費用，包括監管和 CMC 費用，在美國以 55-45 分攤，其中 45% 的份額作為研發費用的一部分計入損益表。在美國以外的地區，Incyte 負責 100% 的開發和監管費用，我們有權獲得美國以外銷售的兩位數特許權使用費。

With that, let me turn the call back over to Michael.

接下來，讓我把電話轉回給麥可。

Thank you, Keith. As you heard during our call, we have made tremendous progress over the past few months and are well prepared for the transformational period ahead of us with the anticipated FDA approval and launch of our first two medicines. With the momentum we have built and our robust clinical development strategy designed to explore the full potential of revumenib and axatilimab, we are excited about the path ahead and the opportunity we have to make a major impact for patients.

謝謝你，基斯。正如您在我們的電話會議中聽到的那樣，我們在過去幾個月中取得了巨大進展，並為我們即將面臨的轉型期做好了充分準備，預計FDA 會批准並推出我們的前兩種藥物。憑藉我們所建立的勢頭以及旨在探索 revumenib 和 axatilimab 全部潛力的穩健臨床開發策略，我們對未來的道路以及我們為患者產生重大影響的機會感到興奮。

On Slide 14, you can see a recap of our upcoming anticipated milestones that we believe we will continue to fuel our momentum and drive value. As always, I want to express my gratitude to the Syndax team and our partners for their hard work and dedication to our mission.

在幻燈片 14 上，您可以看到我們即將到來的預期里程碑的回顧，我們相信我們將繼續推動我們的勢頭並推動價值。一如既往，我想對 Syndax 團隊和我們的合作夥伴表示感謝，感謝他們的辛勤工作和對我們使命的奉獻。

Most importantly, I want to thank all of the patients, families, trial sites and investigators who have participated in our trials and inspire us with their tenacity and commitment to finding new ways to improve the lives of patients with cancer. I'd also like to thank our committed long-term investors who continue to share in our vision and support our work building Syndax.

最重要的是，我要感謝所有參與我們試驗的患者、家屬、試驗地點和研究人員，他們的堅韌和承諾激勵我們尋找改善癌症患者生活的新方法。我還要感謝我們忠誠的長期投資者，他們繼續分享我們的願景並支持我們建立 Syndax 的工作。

With that, I would like to open the call for questions, operator.

話務員，我想開始提問。

(Operator Instructions) Anupam Rama, JPMorgan.

（操作員指令）Anupam Rama，摩根大通。

Hi, thanks for taking the question. This is actually now from Malcolm Kuno on for Anupam. Can you remind us of the size and scope of the field force? And then what sort of medical education efforts will you have ongoing ahead of the potential axatilimab and revumenib approval?

您好，感謝您提出問題。這其實是 Malcolm Kuno 為 Anupam 創作的。您能否提醒我們現場部隊的規模和範圍？那麼，在 axatilimab 和 revumenib 可能獲得批准之前，您將進行什麼樣的醫學教育工作？

Thanks for the question, Malcolm. Let me turn it over to Steve to address it.

謝謝你的提問，馬爾科姆。讓我把這個問題交給史蒂夫來解決。

Yes. Thanks, Malcolm, for the question. In terms of size, I think like comments in the opening was roughly 30 to 50, sorry for the wide range. We haven't given an exact number. But it's adequate to cover the audience. We've got overlapping field teams.

是的。謝謝馬爾科姆提出的問題。就數量而言，我認為開頭的評論大約有 30 到 50 條，抱歉範圍很廣。我們還沒有給出確切的數字。但足以覆蓋觀眾。我們有重疊的現場團隊。

I mean it's a complex treatment journey for patients. They've got treatment centers that have physicians and nursing staff and lab in pathology and formulary. So we've got different customer-facing teams to cover that. We'll cover, I think I said 98% of the opportunity. So it's in place. The field team is activating.

我的意思是，這對患者來說是一個複雜的治療過程。他們擁有配備醫生和護理人員的治療中心以及病理學和處方實驗室。因此，我們有不同的面向客戶的團隊來解決這個問題。我們會涵蓋，我想我說的是 98% 的機會。所以它就位了。現場團隊正在啟動。

I think your other question was around education and what are we doing. I'd say for the field right now as an organization, we're really trying to do two things. One of them is profiling accounts, I mentioned that the complex treatment journey. We want to understand for any given institution that we call on, how they do business, how they see patients, how they're treated. There's a ton for us to learn from understanding how they test KMT2Ar, how those patients get highlighted for clinicians, how they like to deliver drug, the role of nursing staff.

我認為你的另一個問題是關於教育以及我們在做什麼。我想說，作為一個組織，我們現在正在努力做兩件事。其中之一是分析帳戶，我提到了複雜的治療旅程。我們希望了解我們所拜訪的任何特定機構，他們如何開展業務、他們如何看診患者以及他們如何接受治療。透過了解他們如何測試 KMT2Ar、臨床醫生如何突出顯示這些患者、他們喜歡如何輸送藥物以及護理人員的角色，我們可以學到很多東西。

So of the 2,000 accounts that I mentioned, we've already been to more than half of them. I think I mentioned that we call it about 200 that are the priority accounts. We've been to probably at this point, 85% of them and in some of them we've made multiple calls. So that's one thing we're doing, and that's profiling accounts. The goal is to, at the time of launch, know everything that we need to know in order to get patients on drug and treat it. Second piece is the education piece.

因此，在我提到的 2,000 個帳戶中，我們已經訪問過其中一半以上。我想我提到過我們稱之為大約 200 個優先帳戶。到目前為止，我們可能已經去過其中 85% 的企業，其中一些我們已經多次致電。這就是我們正在做的一件事，那就是分析帳戶。我們的目標是在發佈時了解我們需要知道的一切，以便讓患者接受藥物並進行治療。第二部分是教育部分。

There is a little bit of a lift here for menin inhibition from the mechanism of disease standpoint. So that is something else that our field teams are delivering. We've also got a [ non ] personal effort really to raise awareness. Literally not talking about the drug, at least from a commercial standpoint, but are raising awareness of the mechanism. We're the only one that's going to be out, so the awareness level should be tied by the time we get to launch.

從疾病機制的角度來看，menin 抑制有一點提升。這就是我們的現場團隊正在提供的其他東西。我們也做出了[非]個人努力來真正提高人們的認識。從字面上看，至少從商業角度來看，並不是談論這種藥物，而是在提高人們對機制的認識。我們是唯一一個即將退出的公司，因此在我們推出時，意識水平應該是一致的。

So between profiling, raising awareness, we'll be in great shape once the drug gets approved to pull it through.

因此，在分析和提高認識之間，一旦藥物獲得批准，我們將處於良好狀態。

Great, thank you.

太好了，謝謝。

Thanks, Malcolm.

謝謝，馬爾科姆。

Chris Shibutani, Goldman Sachs.

克里斯·澀谷，高盛。

Hi, this is Kevin Sterling for Chris. Thanks for taking my question. Just wanted to talk about the combination studies for revumenib in the second half. You said you'd have data updates. Could you just highlight in bookend what those will be? I believe there's going to be an update from the safe trial?

大家好，我是克里斯的凱文‧史特林。感謝您提出我的問題。只是想談談下半年revumenib的聯合研究。你說你會有數據更新。你能在書擋上強調一下這些是什麼嗎？我相信安全試驗將會有更新？

And then also for the BEAT AML trial, can you talk about where you are with respect to establishing a recommended Phase II dose there? And then what the rate-limiting steps are to start that pivotal trial by the end of the year? And then potentially, how many doses you could bring forward into that trial?

對於 BEAT AML 試驗，您能談談您在建立建議的 II 期劑量方面的進展嗎？那麼，在今年底前開始這項關鍵試驗的速率限制步驟是什麼？然後，您可能可以將多少劑量帶入該試驗？

Kevin, thank you. Maybe I'll address that. So for the BEAT AML trial -- the BEAT AML trial, I'll start there. As you know, we're looking for a recommended Phase II dose. We've made tremendous progress at EHA, we had presented the data on both of those doses. We have in the process of filling out, at least those two doses, just filling out the expected number of patients, and we're going to be making a choice at some point soon between the two doses, which is the recommended Phase II dose, we'll be working through that over the next weeks.

凱文，謝謝你。也許我會解決這個問題。因此，對於 BEAT AML 試驗——BEAT AML 試驗，我將從這裡開始。如您所知，我們正在尋找建議的 II 期劑量。我們在 EHA 取得了巨大進展，我們已經提供了這兩種劑量的數據。我們正在填寫，至少這兩個劑量，只是填寫預期的患者數量，我們將很快在兩個劑量之間做出選擇，這是建議的 II 期劑量，我們將在接下來的幾週內解決這個問題。

So we're in very good shape, ultimately, to start a trial we believe by the end of this year, pivotal trial. And so stay tuned for additional updates on that as we get through the year.

因此，我們的狀態非常好，最終我們相信將在今年年底開始一項關鍵的試驗。因此，請繼續關注這一年的更多更新。

In the safe trial, again, this is Dr. Guise's trial at MD Anderson. This is in relapsed/refractory patients. Combination of ven and [covi] plus revumenib. We presented -- data presented last year at ASH. So the investigators told us that there will be some updated presentations in the latter half of this year. But of course, we're not at liberty to say exactly where that is at this time. But we do expect there to be more patients, additional follow-up. And as you know, it was exciting data. So we're eagerly anticipating that as well.

再次，在安全試驗中，這是吉斯博士在 MD 安德森的試驗。這是在復發/難治性患者。ven 和 [covi] 加上 revumenib 的組合。我們提供了去年在 ASH 上提供的數據。所以調查人員告訴我們，今年下半年會有一些更新的介紹。當然，目前我們還不能透露具體位置。但我們確實預期會有更多的患者、更多的追蹤。如您所知，這是令人興奮的數據。所以我們也熱切期待這一點。

Great. Thank you.

偉大的。謝謝。

Thanks, Kevin.

謝謝，凱文。

Next question is from the line of Phil Nadeau from TD Cowen. Your line is now open.

下一個問題來自 TD Cowen 的 Phil Nadeau。您的線路現已開通。

This is Ernie Rodriguez for Phil. Thanks for taking our questions. I have two brief ones. The first one is, even though the lab in the target treatment centers between revu and axa, and all the prep work -- commercial prep work that you've been doing, do you see any benefit to the now sort of like delay or longer time between the two launches? Can you see perhaps that maybe a benefit or any learnings that you can use to later accelerate the launch? It came too late relative to what your internal estimates were?

我是菲爾的厄尼·羅德里格斯。感謝您回答我們的問題。我有兩個簡短的。第一個是，即使revu和axa之間的目標治療中心的實驗室，以及您一直在做的所有準備工作——商業準備工作，您認為現在延遲或更長時間有什麼好處嗎？之間的時間間隔？您是否能看到這可能是一個好處，或者您可以用來加速發布的任何經驗教訓？相對於您的內部估計來說，這來得太晚了？

And then the second question is, for the Intercept trial of revumenib in monotherapy in the maintenance setting, I believe that trial has been ongoing for a while. I was wondering if there's any updates there, or when could we see data from that trial?

第二個問題是，對於維持治療中瑞維美尼單藥治療的攔截試驗，我相信該試驗已經進行了一段時間。我想知道是否有任何更新，或者我們什麼時候可以看到該試驗的數據？

Thanks for the questions. First I'll ask Neil to address the trial question that you asked and then Steve will adress your commercial question.

感謝您的提問。首先，我將請尼爾解決您提出的試用問題，然後史蒂夫將解決您的商業問題。

Thanks, I think you were asking about the Intercept trial. So you're right, the Intercept trial has been ongoing for some time. The trial is progressing well, as you are also well aware, it is a third-party firm, therefore we don't control disclosure of data. But our understanding is that recruitment is going relatively well. And we expect that the investigators will present data at a medical meeting in the future, but we don't have an exact planning on that.

謝謝，我想您是在詢問 Intercept 試驗。所以你是對的，Intercept 試驗已經進行了一段時間。試驗進展順利，您也很清楚，這是一家第三方公司，因此我們不控制資料的揭露。但我們的理解是，招募進展相對順利。我們預計研究人員將在未來的醫學會議上提供數據，但我們對此沒有確切的計劃。

With that, I'll pass it over to Steve for your first question

這樣，我將把你的第一個問題轉交給史蒂夫

Yes. So just to reiterate, the first question was really sort of benefiting the extra time, particularly the overlap of the two compounds. So I think just speaking, and really speaking from experience, there's never been a launch where someone -- in terms of the pre-launch period, that they wish they had less time or more time in advance of an approval. I think the answer is always, they wish they had more time. So we obviously have more time in revumenib than we would have otherwise anticipated.

是的。所以重申一下，第一個問題確實有利於額外的時間，特別是兩種化合物的重疊。所以我認為，只是說，從經驗來看，從來沒有一個產品發佈時，在發布前的一段時間內，有人希望在獲得批准之前有更少或更多的時間。我認為答案總是，他們希望有更多時間。因此，我們在 revumenib 上的時間顯然比我們預期的要多。

The activity that I've described before will enable us, all the profiling and the mechanisms of disease and disease awareness work we're doing, we'll put to good use over the -- however long it takes us to get to approval. And the 2,000 accounts that I mentioned, there's a good chance we will be at all of them, if not most of them, and we'll be there multiple times. So I think that we'll be able to leverage all that time. And I think the point made, there is tremendous overlap with the axatilimab opportunity.

我之前描述的活動將使我們能夠充分利用我們正在進行的疾病和疾病意識工作的所有分析和機制，無論我們需要多長時間才能獲得批准。我提到的 2,000 個帳戶，我們很有可能會存取所有這些帳戶（如果不是大多數），而且我們會多次存取這些帳戶。所以我認為我們將能夠充分利用這段時間。我認為所提出的觀點與 axatilimab 機會有巨大的重疊。

I don't think I shared the number of treatment centers for chronic GVHD, but we essentially covered them all by covering all of the revumenib opportunities. What I mean by that is 2,000 centers, both academic and community, that we're covering for AML in revumenib. There's under 200 that you'd cover for transplant to support axatilimab.

我不認為我分享了慢性 GVHD 治療中心的數量，但我們基本上透過涵蓋所有 revumenib 機會來涵蓋所有這些中心。我的意思是，我們正在為 2,000 個學術中心和社區中心提供 revumenib 的 AML 治療。您需要支付不到 200 筆移植費用來支援 axatilimab。

So we're early in those, essentially. So we're profiling those accounts as well. There's a lot of connectivity between hema and transplanters, and there's actually some advantages with rev, particularly as patients hopefully move to our transplant and potentially go on continued treatment. So we've always seen that strategically, both drugs are highly aligned to what we're trying to do as an organization.

所以本質上我們還處於早期階段。因此，我們也在對這些帳戶進行分析。血液和移植機之間有很多聯繫，而且轉速實際上有一些優勢，特別是當患者希望轉移到我們的移植並可能繼續接受治療時。因此，我們一直認為，從戰略上講，這兩種藥物與我們作為一個組織正在努力做的事情高度一致。

The call point -- it's the same call point. So we can really accomplish both. And we'll, like I said, make use and leverage the time we have from now until either product comes to market.

呼叫點－這是同一個呼叫點。所以我們確實可以實現這兩點。正如我所說，我們將利用並利用從現在到任一產品上市之前的時間。

Peter Lawson, Barclays.

彼得·勞森，巴克萊銀行。

Hey, good afternoon. This is Alex on for Peter from Barclays. Thanks for taking our questions. And just a quick clarification for me on the BEAT AML study. Could we see more data from this study later this year? And then a separate question is, could we see initial Phase I data from your seven + three combination in 2024.

嘿，下午好。我是來自巴克萊銀行的亞歷克斯 (Alex) 替補彼得 (Peter)。感謝您回答我們的問題。請向我簡單介紹一下 BEAT AML 研究。今年稍後我們能看到這項研究的更多數據嗎？然後一個單獨的問題是，我們能否在 2024 年看到七+三組合的初始第一階段資料。

Alex, thanks for the question. So for BEAT AML, I think we had obviously mentioned that this is not our trial. We are collaborating closely with the sponsor. But the last update was done at EHA, so that was a pretty comprehensive update. We don't have knowledge yet when that's going to be updated. It's possible it could come in the second half of this year at one of the medical meetings.

亞歷克斯，謝謝你的提問。因此，對於 BEAT AML，我認為我們已經明確提到這不是我們的試驗。我們正在與贊助商密切合作。但最後一次更新是在 EHA 完成的，所以這是相當全面的更新。我們還不知道何時會更新。它有可能在今年下半年的醫學會議上出現。

But we don't have perfect information there. So stay tuned on that. And then for the seven + three 3 Phase I that we've initiated, we haven't given guidance yet as to when we're going to be putting together the initial data for that. So stay tuned. I wouldn't expect that we would have it in 2024. That's likely to come in 2025.

但我們那裡沒有完整的資訊。所以請繼續關注。然後，對於我們啟動的 7 + 3 3 第一階段，我們還沒有給出關於何時匯總初始數據的指導。所以請繼續關注。我沒想到我們會在 2024 年擁有它。這可能會在 2025 年實現。

Okay, great. And just if I may, a quick follow-up. Do you have to enroll sort of adverse risk patients here initially? What types of patients are being enrolled in the Phase I?

好的，太好了。如果可以的話，請快速跟進。您是否必須先在這裡招募某種不良風險患者？I 期試驗招募了哪些類型的病人？

Yes. I don't think we've actually talked about the exact type of patients that we're enrolling in the trial. I think they're just newly diagnosed patients that -- with seven + 3 in combination.

是的。我認為我們實際上並沒有討論過我們參加試驗的患者的確切類型。我認為他們只是新診斷的患者——七+三的組合。

Thank you.

謝謝。

Thank you.

謝謝。

Brad, Stifel.

布拉德、斯蒂菲爾.

Hi. Thank you. This is Brad. I think it would be good to clear up some of the sequence around revumenib review. I think a lot of us presumed that with RTOR and the closed FDA collaboration of that revised plus you're ramping commercial and vocal about it, that you had really good visibility about what you needed to get this across the line, either at or well before the PDUFA. So can you help us understand a bit of what happened here?

你好。謝謝。這是布拉德.我認為澄清一些有關 revumenib 審查的順序會很好。我想我們很多人都認為，透過RTOR 和FDA 對該修訂版的封閉合作，再加上你們正在加大商業力度並大聲疾呼，你們對實現這一目標所需的東西有很好的了解，無論是在還是在很好。那麼你能幫助我們了解這裡發生的事情嗎？

I think we were able to catch up with many of you earlier in the week when the news came out. Look, I think this is unfortunate in the sense that we were in receipt of a few RFIs from -- and this is an ongoing process under RTOR that's been going on since last year where we submitted our NDA. And since then, we've been receiving requests for information from the agency that the two -- I'd say two latest RFIs were -- resulted in quite a bit of information going back to the agency.

我想我們在本週早些時候新聞發佈時就能夠採訪到你們中的許多人。聽著，我認為這是不幸的，因為我們收到了一些 RFI，這是 RTOR 下的持續過程，自去年我們提交 NDA 以來一直在進行。從那時起，我們一直收到該機構索取資訊的請求，這兩份資訊請求——我想說的是最新的兩份 RFI——導致我們向該機構返回了相當多的信息。

Once they received that information, they'll let us know. As of last Friday -- late last Friday that, that would require them to actually spend more time on the supplement information and they were going to extend the clock under a major amendment.

一旦他們收到該訊息，他們就會通知我們。截至上週五 - 上週五晚些時候，這將要求他們實際上花更多時間在補充資訊上，並且他們將根據一項重大修正案延長時鐘。

That's a three-month delay. So that was -- and you're right, we were actually quite collaborative over many months with the agency and have been working very closely with them. The information that we provided, we believe, could have been reviewed within the time of the PDUFA. And so for us, we were quite confident that that was the process that we were following. We were a little bit surprised that the agency would extend the clock, but they did.

也就是延遲了三個月。所以，你是對的，我們實際上與該機構合作了好幾個月，並且一直與他們密切合作。我們相信，我們提供的資訊可以在 PDUFA 期限內得到審查。因此，對我們來說，我們非常有信心這就是我們正在遵循的流程。我們對該機構會延長時間感到有點驚訝，但他們確實這麼做了。

That's up to them. That's their decision. So look, this is information that, as I previously said, this is information that we did not have in the NDA. It was not requested of us at the time of the submission of the NDA. It was new information, new clinical information that they wanted to review, and so we provided that.

這取決於他們。這是他們的決定。所以看，正如我之前所說，這是我們在保密協議中沒有的資訊。在提交 NDA 時，我們並未提出這樣的要求。他們想要審查的是新資訊、新臨床訊息，所以我們提供了這些資訊。

This was something that we were prepared for, but they didn't ask for it and we provided it when they did ask for it. The only unfortunate part of this is they -- not that they asked for it, but we learned about it sort of late in the review cycle. We had gone through our mid-cycle and our late-cycle review, and it was without event. So we were feeling quite confident in everything that we had not only been through with the agency, but what we submitted in first half.

這是我們準備好的，但他們沒有要求，當他們要求時我們就提供了。唯一不幸的是他們——不是他們要求這樣做，而是我們在審查週期的後期才了解這一點。我們已經完成了中期和後期的審查，沒有發生任何事件。因此，我們不僅對我們與該機構所經歷的一切，而且對我們上半年提交的內容都感到非常有信心。

So that's the -- those are the sequence of events, and we feel very confident that this drug will get approved on the December time line as they've extended the date. So we're, I think, in a very good position to launch the drug before the end of the year.

這就是 - 這些是事件的順序，我們非常有信心這種藥物將在 12 月的時間表上獲得批准，因為他們已經延長了日期。因此，我認為我們處於非常有利的位置，可以在今年年底前推出該藥物。

And maybe just to follow on that, because I think many of us are now looking at a situation where there's a potential for heightened asymmetry of information. You've got the FDA potentially looking at data that we haven't been able to vet. I guess, what assurance can you give that the overall profile that we know publicly from revumenib is consistent and that prior data cut remains reliable?

也許只是為了跟進這一點，因為我認為我們中的許多人現在都在關註一種可能存在資訊不對稱加劇的情況。FDA 可能會查看我們無法審查的數據。我想，您能提供什麼保證來確保我們公開了解的 revumenib 的整體概況是一致的，並且之前的數據削減仍然可靠？

Yes. Thanks, Brad. Look, I think the information -- and I can -- we don't get into the specifics of anyone's RFI and what's being asked of us in an ongoing review, but what I can say at the late-cycle review, we did learn that there's going to be no AdCom for the product, and they assured us no REMS program for -- that will be required as well.

是的。謝謝，布拉德。聽著，我認為這些資訊——我可以——我們沒有深入了解任何人的RFI 的具體情況，也沒有在正在進行的審查中對我們提出什麼要求，但我可以在後期審查中說的是，我們確實了解到該產品不會有 AdCom，他們向我們保證不會有 REMS 計劃——這也是必要的。

This is not manufacturing information that they've looked for. It's a clinical package that is in our mind completely consistent and supportive of approval. This is not information that changes the profile, the risk-benefit, at all. And so, we feel actually it's even more robust of a package than as previously worked on.

這不是他們尋找的製造資訊。在我們看來，這是一個完全一致並支持批准的臨床方案。這些資訊根本不會改變概況、風險收益。因此，我們實際上認為它比以前的軟體包更強大。

It's basically what the agency had asked for, and they asked for supplemental information. So we feel like it doesn't change anything at all having to do with the profile or the risk-benefit. And for that reason, that's why we're so confident that the drug will not only get approved, but it'll get approved on the time line as proposed. So I think that's as detailed as I could get, Brad.

這基本上就是該機構所要求的，他們要求提供補充資訊。因此，我們認為它根本不會改變與概況或風險收益有關的任何內容。出於這個原因，這就是為什麼我們如此有信心該藥物不僅會獲得批准，而且會按照提議的時間表獲得批准。所以我認為這是我能得到的最詳細的信息，布拉德。

Michael Schmidt, Guggenheim.

邁克爾·施密特，古根漢。

Thanks for taking my question. Maybe just a quick follow-up. So given the revumenib NDA, obviously, is still under RTOR review, and with the extended PDUFA date until end of September now giving ample time for the FDA, just wondering what your view is on the potential for an approval to come ahead of that extended PDUFA date? Is that still on the table, in your opinion? And then I had an unrelated follow-up.

感謝您提出我的問題。也許只是快速跟進。因此，鑑於 revumenib NDA 顯然仍在 RTOR 審查中，並且 PDUFA 日期延長至 9 月底，現在為 FDA 提供了充足的時間，只是想知道您對在延長之前獲得批准的可能性有何看法PDUFA 日期？您認為這仍然在桌面上嗎？然後我進行了一個不相關的後續行動。

Yes, Michael, thanks for the question. It's not unprecedented for -- either with the extensions. We've seen a couple of RTOR products, REZUROCK being one that got approved under RTOR, got extended and actually got approved about six weeks earlier than their extended time frame or their extended PDUFA. So we've seen RTOR products get extended and get approved. Many products under priority review, same thing.

是的，邁克爾，謝謝你的提問。對於擴展而言，這並不是史無前例的。我們已經看到了一些 RTOR 產品，其中 REZUROCK 是在 RTOR 下獲得批准的產品，並得到了延期，並且實際上比其延長的時間範圍或延長的 PDUFA 早了大約六週。因此，我們看到 RTOR 產品已擴展並獲得批准。許多產品正在優先審查，同樣的事情。

Most of the time they go the distance, meaning that they're approved on or around their extended PDUFA date. It's very hard to know if that will or will not be the case for us. We do expect it to be on or before, of course, but we can't -- it's very hard to judge whether that's very removed from the extended date itself. So our expectation is that it will come on or before, on or around the date that they have specified.

大多數情況下，他們會堅持到底，這意味著他們會在 PDUFA 延長日期或前後獲得批准。很難知道我們的情況是否會如此。當然，我們確實希望它能在當天或之前舉行，但我們不能——很難判斷這是否與延長的日期本身相去甚遠。所以我們的期望是它會在他們指定的日期或之前、當天或前後出現。

Super helpful. And then, unrelated follow-up, just sort of thinking about the upcoming registration data in NPM1 patients later this year. Perhaps relative to KMT2Ar, how should we think about the transplant dynamics in the NPM1 subset? And related to that, what's a good modeling assumption for duration of therapy for that market subset relative to KMT2Ar?

超有幫助。然後，無關的後續行動，只是思考今年稍後即將發布的 NPM1 患者的登記數據。也許相對於KMT2Ar，我們該如何思考NPM1子集中的移植動態？與此相關的是，相對於 KMT2Ar，該市場子集的治療持續時間的良好建模假設是什麼？

Thanks for the question. Look, I think NPM1, our view has been all along that the NPM1 data that we've seen in the Phase I trial looks very similar to what we've seen with KMT2A and the consistency across all the measures between the datasets. So that also includes what we've seen in transplant. And although the patients in the NPM1 cohort tend to be a little older, perhaps considerably so, I mean, versus KMT2A, we've also seen a good proportion of those patients get transplanted. So while the way the patients are treated by physicians, there may be more of an urgency to take patients to transplant who have KMT2A versus NPM1.

謝謝你的提問。看，我認為 NPM1，我們一直認為我們在第一階段試驗中看到的 NPM1 數據看起來與我們在 KMT2A 中看到的非常相似，並且數據集之間所有度量的一致性。這也包括我們在移植中看到的情況。儘管 NPM1 群組中的患者年齡往往稍大一些，我的意思是，與 KMT2A 相比，年齡可能要大一些，但我們也看到其中很大一部分患者接受了移植。因此，儘管醫生治療患者的方式不同，但與攜帶 NPM1 的患者相比，攜帶 KMT2A 的患者可能更迫切需要進行移植。

It doesn't preclude a physician from thinking in the same way, that they're healthy enough and they've cleared their tumor to get them to a transplant. So again, that dynamic has seen -- and we've seen that with NPM1 as well as we have with KMT2A. So our view is that the modeling assumptions, and of course this is your own model, but we would model them very similarly in terms of percentage of patients who go on to transplant and then potentially even go back on drug post-transplant, even though they happen to be a slightly older patient population. So I think that's to the best of our knowledge based on information that we've seen from the Phase I and now in our pivotal trial for KMT2A.

這並不妨礙醫生以同樣的方式思考，他們足夠健康，並且已經清除了腫瘤，可以進行移植。再說一次，這種動態已經出現——我們在 NPM1 和 KMT2A 中都看到了這一點。所以我們的觀點是建模假設，當然這是你自己的模型，但我們會根據繼續移植甚至可能在移植後重新接受藥物的患者百分比來對它們進行非常相似的建模，即使他們恰好是年齡稍大的患者群。因此，我認為，根據我們從第一階段以及現在 KMT2A 關鍵試驗中看到的信息，這是我們所知的最佳結果。

Kelly ShiJefferies.

凱莉·希杰弗里斯.

Hi, this is Clare on for Kelly. Thanks for taking our question. Now that you will likely have pivotal data readout in NPM1 that had approval in KMT2A, how would that possibly change your discussion with NCCN Committee for the guideline inclusion? And maybe one step forward also, what will be the conversation with payers going to be like upon this update as well?

大家好，我是凱莉的克萊爾。感謝您提出我們的問題。既然您可能會在 NPM1 中讀出並在 KMT2A 中獲得批准的關鍵數據，那麼這將如何改變您與 NCCN 委員會關於納入指南的討論？也許還向前邁出了一步，在這次更新中與付款人的對話也會是什麼樣的？

Yes, Clare, thanks for the question. Look, I think the cadence of events here in our mind seems to be that we will have NPM1 data now on our pivotal trial ahead of our KMT2A approval. They'll be in close proximity to one another, but it's no longer the other way around. So having NPM1 data first, our priority will be to get that data published as quickly as we can. And once the drug is approved in KMT2A, we can proceed with potential guideline inclusion for NPM1.

是的，克萊爾，謝謝你的提問。看，我認為我們腦海中的事件節奏似乎是，在 KMT2A 獲得批准之前，我們現在將在關鍵試驗中獲得 NPM1 數據。他們將彼此非常接近，但不再相反。因此，首先擁有 NPM1 數據，我們的首要任務是盡快發布該數據。一旦該藥物在 KMT2A 中獲得批准，我們就可以繼續將 NPM1 納入潛在的指南。

So that's the sort of cadence here. And there'll be some urgency to move in order to affect the fact that we have a drug on the market and then another indication in NPM1, which is moving through quickly through an SMDA process at the same time, but could be included in guidelines ahead of approval. So there will be quite a bit of activity related to not only getting the drug approved, but also moving guidelines through publications and conversations with thought leaders who work on an ad hoc basis, if you will, to get new drugs included in guidelines. Maybe I'll turn it over to Steve to talk about the other questions.

這就是這裡的節奏。為了影響我們在市場上有一種藥物以及 NPM1 中的另一個適應症這一事實，將會有一些緊迫性，該適應症正在同時快速通過 SMDA 流程，但可以包含在指南中提前批准。因此，將會有相當多的活動不僅與藥物獲得批准有關，而且還會透過出版物和與思想領袖的對話來推動指南，如果你願意的話，這些思想領袖會臨時工作，將新藥納入指南。也許我會把它交給史蒂夫討論其他問題。

Yes. I think part two is what is the impact on payers of NPM1? So, in my opening comments I talked about our activity with payers, which has been expensive. We'll have talked to payers that cover 90% of managed care lives in both commercial and Part B. I think payers, they recognize the unmet need in KMT2Ar, and they recognize this in NPM1.

是的。我認為第二部分是NPM1對付款人有什麼影響？因此，在我的開場白中，我談到了我們與付款人的活動，這些活動的成本很高。我們將與涵蓋商業和 B 部分中 90% 的管理式醫療生活的付款人進行交談。

They like what they've seen with revumenib, and given the price point in the case of roughly $27,000 to $32,000 a month, we believe we'll be at a slight premium to those agents. It's this obvious connection between NCCN guidelines payers. Payers like to see what's in the public domain. They want to see data published. They want to see the guidelines.

他們喜歡 revumenib 的效果，考慮到每月約 27,000 至 32,000 美元的價格，我們相信我們的價格會比這些藥物略高。這是 NCCN 指南支付者之間這種明顯的聯繫。付款人喜歡查看公共領域的內容。他們希望看到數據被公佈。他們希望看到指導方針。

Clearly, the entree point is KMT2Ar. That's where we'll set price. I think payers want to service patients. They'll produce some hurdles and obstacles upfront and barriers, which they always do with products like this. We've got fantastic specialty pharmacy partners and distributors and a patient portal when needed to usher patients through the early days of a launch when products are not yet on formulary.

顯然，主線是 KMT2Ar。這就是我們設定價格的地方。我認為付款人希望為患者提供服務。他們會預先設置一些障礙和障礙，他們總是對此類產品這樣做。我們擁有出色的專業藥房合作夥伴和分銷商，以及在需要時引導患者度過產品尚未進入處方集的上市初期的患者門戶。

So we understand the medical exception process as well as our preferred pharmacy partners as well as treatments and institutions. So we think either way we'll be able to set the right price, and we'll be able to get patients on drugs with some challenge, but we'll get there, and we're prepared to do it.

因此，我們了解醫療例外流程以及我們首選的藥房合作夥伴以及治療方法和機構。因此，我們認為無論哪種方式，我們都將能夠設定合適的價格，並且我們將能夠讓患者接受一些挑戰的藥物，但我們會實現這一目標，並且我們已經準備好這樣做。

Yigal Nochomovitz, Citi.

伊格爾·諾霍莫維茨，花旗銀行。

This is Ashiq Mubarack on for Yigal. Thanks for taking my questions. I just wanted to ask one on axatilimab. Sounds like everything is on track with the PDUFA, which is coming up pretty soon. I just wanted to know if you could share any color on maybe where things are in the sort of ELA process? Have you reached label negotiations?

我是阿希克·穆巴拉克 (Ashiq Mubarack)，替補伊加爾 (Yigal)。感謝您回答我的問題。我只是想問一個關於 axatilimab 的問題。聽起來 PDUFA 一切都步入正軌，而且很快就會推出。我只是想知道您是否可以分享有關 ELA 流程中的情況的任何顏色？你們已經達成標籤談判了嗎？

Was there anything of value with the mid-cycle review? Anything to sort of confirm that ELA is on track would be very helpful.

中期審查有什麼價值嗎？任何能夠確認 ELA 步入正軌的事情都會非常有幫助。

Thanks for the question. So we don't comment on any specific part of our negotiations or discussions with the agency during the process. It is under priority review, as you remember. As we said along, it's going well. We do expect the drug to be approved around the PDUFA date, which is the end of August.

謝謝你的提問。因此，我們不會對此過程中與該機構的談判或討論的任何具體部分發表評論。如您所知，它正在接受優先審查。正如我們所說，一切進展順利。我們確實預計該藥物將在 PDUFA 日期（即 8 月底）左右獲得批准。

Nothing that we've encountered in the review process changes that point of view. I won't comment specifically around whether we are or we are not in label discussion, but we are feeling very confident that the drug will be approved and will be approved on its time line.

我們在審查過程中遇到的任何事情都不會改變這一觀點。我不會具體評論我們是否在標籤討論中，但我們非常有信心該藥物將獲得批准，並將在其時間表上獲得批准。

Maybe one follow-up on that then. Should we sort of expect the commercial launch to take place maybe immediately after a potential approval that's coming up? Or is there a reason to sort of think there might be a little bit of a lag maybe waiting for a revumenib? So there's some synergy with that commercial launch as well?

也許接下來會有一個後續行動。我們是否應該期望在即將獲得潛在批准後立即進行商業發布？或者是否有理由認為等待 revumenib 可能會有一點延遲？那麼與商業發布也有一些協同作用嗎？

So no, thanks for the follow-up, Ashiq. We said that the launch is expected in the fourth quarter. So that's our guidance. We're working with our partner to get ready to do that, and we'll launch the drug when it's ready.

所以不，感謝您的跟進，Ashiq。我們表示預計將於第四季推出。這就是我們的指導。我們正在與我們的合作夥伴合作，做好準備，準備好後我們將推出該藥物。

Kalpesh Patel, B. Riley.

卡爾佩什·帕特爾，B.萊利。

Yes, hey, good afternoon and thanks for taking the question. Maybe one for revumenib. Is there anything that you learned from the KMT2Ar regulatory filing here that could be beneficial for the NPM1 filing and help streamline that? I know that the time line says first half '25 and sort of maybe first quarter '25. Is there a reason for that? And then I have a follow-up.

是的，嘿，下午好，感謝您提出問題。也許是 revumenib 的一種。您從此處的 KMT2Ar 監管備案中學到的東西是否有利於 NPM1 備案並有助於簡化流程？我知道時間軸是 25 年上半年，也可能是 25 年第一季。這是有原因的嗎？然後我有一個後續行動。

Yes, thanks, Kal. Let me turn it over to Neil to maybe take a crack at that.

是的，謝謝，卡爾。讓我把它交給尼爾也許可以嘗試。

Yes, thanks. Thanks for the question. Yes. Thanks for the question. So the straightforward answer to your question is yes. So I think that we've learned a lot about the process. And, of course, it's important to draw the distinction between the NDA and the SNDA.

是的，謝謝。謝謝你的提問。是的。謝謝你的提問。所以你的問題的簡單答案是肯定的。所以我認為我們在這個過程中學到了很多。當然，區分 NDA 和 SNDA 也很重要。

So in the NDA, we do a lot of the heavy lifting is done, and any subsequent application -- supplementary application is much more straightforward. So we're obviously excited and looking forward to getting the NPM1 data during the fourth quarter, and you can be sure that we will be filing or submitting those data as expeditiously as possible. There was probably a question I missed. Did I address all of your questions, or was there something I missed?

因此，在 NDA 中，我們做了很多繁重的工作，任何後續申請——補充申請都更加簡單。因此，我們顯然很興奮並期待在第四季度獲得 NPM1 數據，您可以確信我們將盡快歸檔或提交這些數據。可能有一個問題我錯過了。我是否回答了您所有的問題，或者我遺漏了什麼？

Yes. It's just that -- it's just first half '25 instead of first quarter '25 for filing for the NPM1 cohort?

是的。只是 - NPM1 隊列的申請只是 25 年上半年，而不是 25 年第一季？

Yes. We haven't been that specific with our guidance here, actually. But you can be assured that we will be working pretty hard at it. I'll pass it back to Michael.

是的。實際上，我們在這裡的指導並沒有那麼具體。但您可以放心，我們將為此付出很大努力。我會把它傳回給麥可。

The second question is sort of related here. Was there -- the new information that was requested by the FDA, I guess, was any part of the NPM1 cohort data set that was sent to the FDA for this update?

第二個問題與此相關。我猜 FDA 要求的新資訊是否是發送給 FDA 進行更新的 NPM1 隊列資料集的任何部分？

No, Kal. But I would actually just focus on the KMT2A submission. That's what we're working with the FDA right now. NPM1 data hasn't even been -- we're not even through the trial. All of it hasn't been done yet.

不，卡爾。但實際上我只會關注 KMT2A 提交的內容。這就是我們目前正在與 FDA 合作的內容。NPM1 數據甚至還沒有——我們甚至還沒有完成試驗。所有這一切還沒有完成。

So when that trial is available or when the data is available, we'll publish it, and that will be in the fourth quarter. So you should -- the information that we're focused on with the agency relates to KMT2A.

因此，當試驗可用或數據可用時，我們將發布它，這將在第四季度進行。所以你應該——我們與該機構關注的資訊與 KMT2A 相關。

Justin Allen, BTIG. Your line is now open.

賈斯汀·艾倫，BTIG。您的線路現已開通。

Thanks for taking my question. Michael, I'd like to clarify regarding your confidence on the revumenib approval by the end of the year. Should we continue to expect a traditional approval here, or could there be a scenario in the review cycle where revumenib could be approved under an accelerated approval basis?

感謝您提出我的問題。邁克爾，我想澄清一下您對今年年底前批准 revumenib 的信心。我們是否應該繼續期待傳統的批准，還是在審查週期中可能存在一種情況，即 revumenib 可以在加速批准的基礎上獲得批准？

Yes, Justin, thanks for the question. I don't think you should assume for a second that this is going to be approved in any other way other than full approval. All of the precedent drugs that have gotten approved in AML as monotherapy-targeted therapies have been in the -- through the exact same process.

是的，賈斯汀，謝謝你的提問。我認為你不應該假設除了完全批准之外，這將以任何其他方式獲得批准。所有在 AML 中作為單一療法獲得批准的先例藥物都經過了完全相同的過程。

And so, that's never been discussed with the agency that we would -- there is no accelerated approval pathway, and that's not the pathway we're pursuing here. So I would not assume that that would change at all. And we have high confidence that this is just a delay of three months. It doesn't change the process at all in terms of what we're filing for and what we're likely to have at approval.

因此，我們從未與該機構討論過這一問題——沒有加速批准途徑，這也不是我們在這裡追求的途徑。所以我認為這根本不會改變。我們非常有信心這只是三個月的延遲。就我們正在申請的內容以及我們可能獲得批准的內容而言，它根本不會改變流程。

Jason, Zemansky from Bank of America.

美國銀行的傑森·澤曼斯基。

This is Cameron Bozdog on for Jason. Congrats on the progress, and thanks so much for taking our question. So looking ahead to the potential near-term launch of axatilimab, can you maybe comment on your internal market analysis and what you've been hearing from prescribers? I mean, do you expect uptake, at least initially, to be largely driven by academic prescribers centered at transplant centers versus community docs, especially when you think about (inaudible) administration?

我是卡梅倫·博茲多格 (Cameron Bozdog) 替傑森 (Jason) 發言。恭喜您的進展，非常感謝您提出我們的問題。因此，展望近期可能推出的 axatilimab，您能否對您的內部市場分析以及您從處方醫生那裡聽到的情況發表評論？我的意思是，您是否預計，至少在最初，採用主要是由以移植中心為中心的學術處方者而不是社區醫生推動的，特別是當您考慮（聽不清楚）管理時？

Great. Thanks for the question. I'm going to turn it over to Steve to address.

偉大的。謝謝你的提問。我要把它交給史蒂夫處理。

Thanks for the question. I'd start off saying, it's a pretty small audience and there's a lot of high science and experts in the field of transplantation. So from the treatment center standpoint, I think I may have provided the numbers earlier, 10% of the centers. It's under 200. So it was all driven by that. I think in the part of who are the drivers, academic versus community, it's the biggest centers are going to drive all this.

謝謝你的提問。我首先要說的是，觀眾很少，但移植領域有許多高科技和專家。所以從治療中心的角度來看，我想我可能之前已經提供了數字，10%的中心。還不到200呢所以這一切都是由這個驅動的。我認為就誰是驅動者而言，學術界還是社區，最大的中心將推動這一切。

For the most part, certainly at launch, there'll be some awareness outside of that. And even within the 200 center, there's probably 35 that see half the patients in the country. So it is -- I wouldn't say it's the smallest physician audience to call in, but it's one of the smallest. And it's a tight community. I think speaking to market opportunity, there's a lot of business here.

在大多數情況下，當然是在發佈時，除此之外還會有一些意識。即使在 200 個中心內，也可能有 35 個中心收治全國一半的患者。所以，我不會說這是拜訪的醫生觀眾人數最少的，但它是最少的之一。這是一個緊密的社區。我認為就市場機會而言，這裡有很多生意。

There's a lot of unmet need and dissatisfaction in the current treatment GVHD population. They want more. They need more agents. It's a really insidious disease. So it'll be I'd say early uptake and a group of patients probably that are waiting for something like this.

目前 GVHD 治療族群中存在許多未滿足的需求和不滿。他們想要更多。他們需要更多的代理人。這真是一種陰險的疾病。所以我想說的是早期的吸收和一群患者可能正在等待這樣的事情。

It's not a huge number, but there's some number of patients waiting. So there will be some form of bolus, but the market's ready and will be ready shortly too and I think it'll be well received by physicians and patients.

人數雖然不多，但等待的病人還是有一定數量的。因此，將會有某種形式的推注，但市場已經準備好，並且很快就會準備好，我認為它將受到醫生和患者的好評。

George Farmer,Scotiabank.

喬治法默，豐業銀行。

Hi. This is Chloe on for George. So two from us. So based on your initial data package for revumenib, you said that FDA did its thing and ODAC was necessary. Do you still believe now to be the case? So how confident are you that the additional information you submitted that prompted the proof extension will not be subject of an ODAC review in the next six months?

你好。這是克洛伊為喬治代言的。所以我們有兩個。因此，根據您最初的 revumenib 資料包，您說 FDA 做了它的事情，ODAC 是必要的。現在你還相信是這樣嗎？那麼，您對您提交的促使證明延期的附加資訊在未來六個月內不會接受 ODAC 審核的信心有多大？

And the second question is, Michael, you mentioned BD remark. So I'm just curious what types of BD deals you had in mind, any examples of areas of interest, those types of assets you're eyeing? So at the moment, those are the vital or even a target. And if you could maybe put it in context to do the Incyte partnership for us and tell us kind of how you see that evolving over time?

第二個問題是，Michael，你提到了 BD 的言論。所以我只是好奇您想到的是哪些類型的 BD 交易、您感興趣的領域的任何示例、您正在關注的資產類型？所以目前來說，這些都是至關重要的，甚至是一個目標。您是否可以將其放在為我們開展因塞特合作夥伴關係的背景中，並告訴我們您如何看待這種關係隨著時間的推移而演變？

Chloe, hanks for the question. So first, in terms of ODAC, I think high degree of confidence that an ODAC will not be required. The agency on more than one occasion has told us this. The data that we submitted, as I referenced earlier, is only in our minds and very clearly supportive of approval and only augments the package that we've submitted. So there's really -- there is, in our minds and based on what we've heard from the agency, there doesn't seem to be any risk of an ODAC that we know of.

克洛伊，謝謝你的提問。首先，就 ODAC 而言，我認為非常有信心不需要 ODAC。該機構不只一次告訴我們這一點。正如我之前提到的，我們提交的數據僅在我們的腦海中，並且非常明確地支持批准，並且只會增強我們提交的一攬子計劃。因此，在我們看來，根據我們從該機構聽到的情況，據我們所知，ODAC 似乎不存在任何風險。

So I feel quite confident about that. And then in terms of BD, which is an interesting question and essentially relates to how do we grow the pipeline and continue doing what we do well at Syndax in part, which is in licensing or acquiring new molecules to add to the pipeline. We've said in the past that we continue to work on adding or backfilling the pipeline as early-stage molecules in the targeted therapy space in oncology, focused on oncology.

所以我對此非常有信心。然後就 BD 而言，這是一個有趣的問題，本質上涉及我們如何發展管道並繼續做我們在 Syndax 擅長的部分，即許可或獲取新分子以添加到管道中。我們過去曾說過，我們將繼續致力於在腫瘤學標靶治療領域（重點是腫瘤學）中添加或回填作為早期分子的管道。

We don't speak specifically about which mechanisms we're going after or the status of any of these discussions and what -- when we conclude our deals, we usually bring -- we do bring them forward and talk about them publicly. But at this stage, we are very actively looking and feel quite good about the potential.

我們不會具體談論我們正在尋求哪些機製或任何這些討論的狀態，以及當我們達成交易時，我們通常會提出什麼，我們確實會提出並公開討論它們。但在現階段，我們非常積極地尋找潛力，並對潛力感到非常滿意。

But at this point, it's hard to speculate on business development transactions until they're done and closed. But whatever we do will be a -- it seems of an earlier variety, so we're not looking to take on late-stage projects. Earlier projects are the type of the -- type and complexion of what we feel like we can accommodate at this time with all the other interesting things we have going on in our pipeline for axatilimab and for revumenib. So it's a balanced strategy and one that we are actively engaged in.

但目前，在業務開發交易完成並結束之前很難對其進行推測。但無論我們做什麼，都將是一個早期的項目，所以我們不打算承擔後期項目。早期的專案是我們認為目前可以適應的類型和複雜性，以及我們在 axatilimab 和 revumenib 管道中正在進行的所有其他有趣的事情。因此，這是一項平衡的策略，也是我們正在積極參與的策略。

Okay, thanks. With respect to the partnership with Incyte we have been seeing, any thoughts on how you see that evolving in the future?

好的，謝謝。關於我們與 Incyte 的合作關係，您對未來的發展有何想法？

Our partnership with Incyte is a good one. We conceived it back in 2021 when we announced that. They've been good partners to us. And as Steve outlined and we've talked about, we're keen to launch that, get that product approved and launched, and certainly expand the utilization of axatilimab in a variety of indications. Earlier in the treatment course for GVHD, we're pursuing IPF.

我們與 Incyte 的合作關係非常好。我們在 2021 年宣布這項消息時就開始構思它。他們一直是我們的好夥伴。正如 Steve 所概述和我們所討論的，我們熱衷於推出該產品，獲得該產品的批准和推出，並肯定會擴大 axatilimab 在各種適應症中的使用。在 GVHD 治療過程的早期，我們正在尋求 IPF。

So that's -- these are -- this is a global partnership with Incyte and so it's gone quite well. And we continue to believe that it will expand and continue in flexibility in the way we envisioned it to start. So we're very happy with that.

這是與 Incyte 的全球合作夥伴關係，進展順利。我們仍然相信，它將按照我們設想的開始方式擴展並繼續保持靈活性。所以我們對此非常滿意。

This concludes the question-and-answer session. I'll now turn the floor over to Mr. Michael Metzger for any additional comments or closing remarks.

問答環節到此結束。現在請邁克爾·梅茨格先生發表補充意見或結束語。

Thank you all. We appreciate you all tuning in today to discuss the progress we have made and the exciting milestones ahead for the company. We look forward to seeing you at our planned investor events, including the upcoming BTIG conference in August. And with that, we wish you a good day. Thank you so much.

謝謝大家。我們感謝大家今天收聽來討論我們所取得的進展以及公司未來令人興奮的里程碑。我們期待在我們計劃的投資者活動中見到您，包括即將於 8 月舉行的 BTIG 會議。在此，我們祝福您有美好的一天。太感謝了。