Syndax Pharmaceuticals Inc (SNDX) 2025 Q3 法說會逐字稿

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  • Operator

    Operator

  • Good day, everyone, and welcome to the Syndax third-quarter 2025 earnings conference call. Today's call is being recorded. (Operator Instructions)

    大家好,歡迎參加 Syndax 2025 年第三季財報電話會議。今天的通話將會被錄音。(操作說明)

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

    此時,我想把電話交給 Syndax Pharmaceuticals 的投資人關係主管 Sharon Klahre。

  • Sharon Klahre - VP of Investor Relations & Communications

    Sharon Klahre - VP of Investor Relations & Communications

  • Thank you, operator. Welcome, and thank you all for joining us today for a review of Syndax's third-quarter 2025 financial and operating results. I'm Sharon Klahre. With me this afternoon to provide an update on the company's progress and discuss financial results are Michael Metzger, Chief Executive Officer; Steve Closter, Chief Commercial Officer; Dr. Nick Botwood, Head of R&D and Chief Medical Officer; and Keith Goldan, Chief Financial Officer.

    謝謝接線生。歡迎各位,感謝大家今天與我們一起回顧 Syndax 2025 年第三季的財務與營運表現。我是莎倫·克拉雷。今天下午與我一起向大家報告公司進展並討論財務業績的有:首席執行官 Michael Metzger;首席商務官 Steve Closter;研發主管兼首席醫療官 Nick Botwood 博士;以及首席財務官 Keith Goldan。

  • 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 Strategy Officer. 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.

    今天參加電話會議問答環節的還有首席科學官 Peter Ordentlich 博士和首席策略官 Anjali Ganguli 博士。本次電話會議附帶一份幻燈片,該幻燈片已發佈在公司網站的投資者關係頁面上。現在您可以翻到第 2 頁查看我們的前瞻性聲明。

  • Before we begin, I'd like to remind you that any statements made during this call that are not historical are considered to be forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. 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 report on Form 10-Q as well as other reports filed with the SEC.

    在開始之前,我想提醒各位,本次電話會議中任何非歷史事實的陳述均屬於 1995 年《私人證券訴訟改革法案》所界定的前瞻性陳述。由於各種重要因素的影響,實際結果可能與這些聲明所指出的結果有重大差異,這些因素包括公司最新季度報告(10-Q 表格)「風險因素」部分討論的因素以及向美國證券交易委員會提交的其他報告中討論的因素。

  • Any forward-looking statements made represent our views as of today, November 3, 2025, only. A replay of this call will be available on the company's website, www.syndax.com, following its completion. With that, I am pleased to turn the call over to Michael Metzger, Chief Executive Officer of Syndax.

    文中所有前瞻性陳述僅代表我們截至 2025 年 11 月 3 日的觀點。本次電話會議結束後,您可以在本公司網站 www.syndax.com 上收聽本次電話會議的錄音。接下來,我很高興將電話交給 Syndax 的執行長 Michael Metzger。

  • Michael Metzger - Chief Executive Officer, Director

    Michael Metzger - Chief Executive Officer, Director

  • Thank you, Sharon. Good afternoon, and thank you all for joining us. Starting with slide 3. The third-quarter was another outstanding period of commercial and portfolio execution for Syndax. Importantly, the progress we made advances us on the road to profitability and furthers our leadership position in menin inhibition, an exciting new category that Syndax is uniquely positioned to lead across the relapsed/refractory and frontline setting.

    謝謝你,莎倫。下午好,感謝各位的參與。從第3張投影片開始。Syndax 第三季在商業和產品組合執行方面又取得了卓越的成就。重要的是,我們取得的進展使我們朝著獲利的目標邁進了一步,並進一步鞏固了我們在menin抑制劑領域的領先地位。 menin抑制劑是一個令人興奮的新類別,Syndax 在該領域擁有獨特的優勢,可以引領復發/難治性和一線治療。

  • Starting with our commercial results for the quarter. We reported $45.9 million in total revenue for the third-quarter, representing strong 21% growth over the prior quarter. We are very encouraged by the launch metrics for both Revuforj and Niktimvo, two first and best-in-class medicines that are addressing major unmet patient needs.

    首先來看我們本季的商業業績。第三季總營收為 4,590 萬美元,較上一季強勁成長 21%。Revuforj 和 Niktimvo 的上市指標令我們倍感鼓舞,這兩款首創且同類最佳的藥物正在解決患者尚未滿足的重大需求。

  • With both medicines, a robust base of new patients are starting each quarter and a growing number are continuing on therapy, building a foundation for sustained long-term growth. Net revenue for Revuforj was $32 million in the third-quarter, up 12% from the prior quarter, even with approximately a third of patients temporarily pausing treatment to receive a stem cell transplant.

    這兩種藥物每季都有大量新患者開始接受治療,而且越來越多的患者正在繼續接受治療,這為持續的長期成長奠定了基礎。Revuforj 第三季淨收入為 3,200 萬美元,比上一季增加 12%,儘管約有三分之一的患者暫時停止治療以接受幹細胞移植。

  • Importantly, all indicators of demand remain strong and with approximately 25% growth in total prescriptions and new patient starts in the third-quarter compared to the prior quarter. Revuforj has become -- rapidly become the standard of care for relapsed/refractory KMT2A and is widely being used early in the treatment paradigm with approximately 50% of usage in the second line.

    重要的是,所有需求指標依然強勁,第三季處方總量和新患者開戶量比上一季成長了約 25%。Revuforj 已迅速成為復發/難治性 KMT2A 的標準治療方法,並在治療模式的早期廣泛使用,約 50% 的使用率用於二線治療。

  • A growing number of KMT2A patients are proceeding to a potentially curative stem cell transplant after receiving Revuforj, a fantastic outcome for patients and clinicians. The use of Revuforj in the post-transplant maintenance setting also continues to build as physicians put their patients back on therapy. This dynamic will become an important growth driver in the fourth-quarter and beyond as the number of patients receiving extended maintenance treatment begins to meaningfully offset and then exceed the number who paused therapy each quarter to receive a transplant.

    越來越多的 KMT2A 患者在接受 Revuforj 治療後,正在進行可能治癒的幹細胞移植,這對患者和臨床醫生來說都是一個極好的結果。隨著醫生讓患者重新接受治療,Revuforj 在移植後維持治療的應用也持續成長。這種動態將在第四季度及以後成為重要的成長驅動力,因為接受長期維持治療的患者人數將開始顯著抵消並超過每季暫停治療接受移植的患者人數。

  • In the third-quarter and recent weeks, we have made major strides advancing another important Revuforj growth driver. On September 18, Revuforj was added to the NCCN Guidelines as a recommended treatment option for relapsed/refractory NPM1 mutated AML ahead of the subsequent FDA approval, which speaks to the strength of our clinical data and physicians' enthusiasm for Revuforj.

    在第三季和最近幾週,我們在推動另一個重要的 Revuforj 成長驅動因素方面取得了重大進展。9 月 18 日,Revuforj 被列入 NCCN 指南,作為復發/難治性 NPM1 突變 AML 的建議治療方案,隨後獲得 FDA 批准,這體現了我們臨床數據的實力以及醫生對 Revuforj 的熱情。

  • On October 24, we received FDA approval for Revuforj in relapsed/refractory NPM1 mutated AML, tripling the size of our addressable patient population. This approval makes Revuforj the first and only menin inhibitor that is FDA approved for multiple acute leukemia subtypes in adult and children one year of age or older. The breadth of our indicated population highlights the compelling and consistent efficacy and tolerability of Revuforj across different patient populations.

    10 月 24 日,我們獲得了 FDA 對 Revuforj 用於治療復發/難治性 NPM1 突變 AML 的批准,使我們可治療的患者群體規模擴大了三倍。此次獲準使 Revuforj 成為首個也是唯一一個獲得 FDA 批准用於治療成人和一歲及以上兒童多種急性白血病亞型的 menin 抑制劑。我們所針對的族群範圍之廣,凸顯了 Revuforj 在不同患者族群中具有令人信服且一致的療效和耐受性。

  • Nick will unpack the unique aspects of the Revuforj product profile when he reviews the key abstracts we will have this year at ASH. These data sets will add to the growing body of efficacy data that differentiate Revuforj from other menin inhibitors. Our expansion into NPM1 is in full swing, and we are pleased with our early progress driving awareness of the new indication and generating demand among physicians who treat NPM1 patients.

    Nick 將在審查我們今年在 ASH 會議上發表的關鍵摘要時,詳細介紹 Revuforj 產品概況的獨特之處。這些數據集將補充不斷增長的療效數據,這些數據將使 Revuforj 與其他 menin 抑制劑區分開來。我們正在全面拓展 NPM1 領域,並且很高興看到我們在提高人們對這一新適應症的認識以及在治療 NPM1 患者的醫生中產生需求方面取得的早期進展。

  • Importantly, these are the same physicians who treat KMT2A patients and have already built familiarity and trust with Revuforj and Syndax. In the one week since approval, we have already engaged with hundreds of physicians and feedback has been very positive.

    重要的是,這些醫生也是治療 KMT2A 患者的醫生,他們已經對 Revuforj 和 Syndax 建立了熟悉度和信任度。自獲批以來的一周內,我們已經與數百名醫生進行了交流,反饋非常積極。

  • They are enthusiastic to have Revuforj as the first highly efficacious targeted therapy indicated for relapsed/refractory NPM1. We are well positioned for success with best-in-class efficacy and at least a one-year first-mover advantage over any other company. Physician decision-making is driven by efficacy in acute leukemia, and we have differentiated efficacy data in multiple acute leukemia subtypes.

    他們非常高興Revuforj成為第一個針對復發/難治性NPM1的高效標靶治療藥物。我們擁有業內領先的功效,並且比任何其他公司至少有一年的先發優勢,因此我們具備成功的良好條件。醫生在製定急性白血病治療方案時,主要考慮療效,而我們針對多種急性白血病亞型,已獲得了不同的療效數據。

  • In relapsed/refractory NPM1 specifically, we have shown unmatched data, including an approximately 50% overall response rate, five-month median duration of CR/CRh, 17% transplant rate and a two-year median overall survival observed among responders. While CR/CRh is an important regulatory endpoint, ORR is of utmost importance from a clinical standpoint. A higher overall response rate gives clinicians the ability to bring more patients into remission and the best chance of bringing their eligible patients to a potentially curative stem cell transplant.

    特別是對於復發/難治性 NPM1,我們展示了無與倫比的數據,包括約 50% 的總體緩解率、5 個月的 CR/CRh 中位數持續時間、17% 的移植率以及緩解者中觀察到的 2 年的中位總生存期。雖然 CR/CRh 是一個重要的監管終點,但從臨床角度來看,ORR 更為重要。更高的整體緩解率使臨床醫生能夠使更多患者病情緩解,並為符合條件的患者提供最佳機會進行可能治癒的幹細胞移植。

  • Moving to Niktimvo. In the second-quarter of launch, our partner, Incyte, reported $45.8 million in Niktimvo net revenue, a robust 27% increase over the prior quarter. Within just the first eight months of launch, Niktimvo is annualizing at nearly $200 million and tracking in line with first year sales of Sanofi's REZUROCK, which reached over $500 million in annual US net sales within the first three years of launch in the same indication.

    搬到尼克蒂姆沃。在產品發布後的第二季度,我們的合作夥伴 Incyte 報告 Niktimvo 淨收入為 4,580 萬美元,比上一季強勁成長了 27%。Niktimvo上市僅八個月,年銷售額就接近2億美元,與賽諾菲的REZUROCK上市第一年的銷售額持平。 REZUROCK在同一適應症下,上市三年內,其在美國的年淨銷售額就超過了5億美元。

  • Importantly, Niktimvo is profitable to Syndax with our 50% share of the Niktimvo product contribution amounting to $13.9 million for the third-quarter. As sales continue to ramp, we expect the proportion of net revenue retained by Syndax to materially grow over time. We remain on the road to profitability with growing contributions from Revuforj and Niktimvo, a solid balance sheet and an operating expense base that will remain stable over the next few years while fully funding our strategic priorities.

    重要的是,Niktimvo 為 Syndax 帶來了利潤,我們佔 Niktimvo 產品貢獻的 50%,第三季貢獻額達 1,390 萬美元。隨著銷售額持續成長,我們預期 Syndax 保留的淨收入比例將隨著時間的推移而大幅成長。在 Revuforj 和 Niktimvo 的貢獻不斷增長、資產負債表穩健以及未來幾年將保持穩定的營運支出基礎的共同作用下,我們正朝著盈利的目標穩步前進,同時為我們的戰略重點提供充足的資金。

  • Most notably, our strategic priorities include the expansion of Revuforj and Niktimvo into the frontline setting, which would unlock a combined market opportunity exceeding $10 billion. Enrollment is well underway in EVOLVE-2, the first pivotal frontline trial for a menin inhibitor to start enrolling patients.

    最值得注意的是,我們的策略重點包括將 Revuforj 和 Niktimvo 擴展到一線環境,這將釋放超過 100 億美元的綜合市場機會。EVOLVE-2 的招募工作正在順利進行中,這是首個開始招募患者的關鍵性一線試驗,針對的是 menin 抑制劑。

  • We have the right strategy and partnerships to flawlessly execute this trial and be the first to frontline with a menin inhibitor. With Niktimvo, two frontline trials are ongoing in combination with standard of care therapies that could transform the treatment of chronic GVHD. With that, I will turn the call over to Steve to discuss our commercial progress in more detail. Steve?

    我們擁有正確的策略和合作關係,能夠完美地執行這項試驗,並成為第一個將menin抑制劑應用於第一線治療的團隊。目前,Niktimvo 正在進行兩項第一線試驗,與標準治療方案相結合,有望改變慢性 GVHD 的治療方式。接下來,我將把電話交給史蒂夫,讓他更詳細地討論我們的商業進展。史蒂夫?

  • Steven Closter - Chief Commercial Officer

    Steven Closter - Chief Commercial Officer

  • Thank you, Michael. Starting with Revuforj on slide 4. We're on track for a strong first year of sales with continued growth in KMT2A and a solid foundation in place for a successful launch into NPM1 and our future expansion into the frontline setting. In the first 10 months of sales, we've generated nearly $90 million in Revuforj net revenue, exceeding by a wide margin the launch benchmark set by other AML therapies. These impressive results reflect the rapid adoption of Revuforj as the standard of care in relapsed/refractory KMT2A and physicians' positive experience with the drug.

    謝謝你,麥可。從第 4 張投影片上的 Revuforj 開始。我們預計在第一年實現強勁的銷售成長,KMT2A 持續成長,並為成功推出 NPM1 以及未來擴展到第一線環境奠定了堅實的基礎。在上市後的前 10 個月裡,Revuforj 的淨收入接近 9,000 萬美元,遠遠超過了其他 AML 療法設定的上市基準。這些令人印象深刻的結果反映了 Revuforj 迅速被採納為復發/難治性 KMT2A 的標準療法,以及醫生們對該藥物的積極體驗。

  • Sales of Revuforj were strong in the third-quarter with $32 million in net revenue, up from $28.6 million in the prior quarter. Importantly, key demand indicators increased even more significantly with total prescription and new patient starts for the quarter both increasing approximately 25% over the prior quarter. This robust increase in demand speaks to Revuforj's compelling product profile and the strong and durable business that we are building.

    Revuforj 第三季的銷售額表現強勁,淨收入達 3,200 萬美元,高於上一季的 2,860 萬美元。值得注意的是,關鍵需求指標的成長更為顯著,本季處方總量和新患者開戶量均比上一季增加了約 25%。需求的強勁成長表明 Revuforj 具有強大的產品吸引力,並且我們正在建立一個強大而持久的業務。

  • The delta between demand and net revenue growth this quarter was due to variability in gross to net and channel inventory as you often see period-to-period, especially in the first year of the launch. Since launch in late 2024 through the end of September of this year, approximately 2,200 prescriptions have been written for 750 patients, with an estimated 90% of usage in KMT2A.

    本季需求與淨收入成長之間的差異是由於毛利潤與淨利潤以及通路庫存的波動造成的,這種情況經常在不同時期出現,尤其是在產品上市的第一年。自 2024 年底推出以來,截至今年 9 月底,已為 750 名患者開立了約 2,200 張處方,其中 KMT2A 的使用率估計為 90%。

  • With this momentum, we remain on track to treat 1,000 KMT2A patients by year's end or more. This would represent 50% penetration of the annual 2,000-patient KMT2A incidents within the first year of launch, and that is a fantastic result. The use of Revuforj continues to migrate to earlier lines of therapy with claims data showing approximately 70% of usage concentrated in the second and third line with 50% coming from the second line or first relapse patients alone.

    憑藉這一勢頭,我們仍有望在年底前治療 1,000 名 KMT2A 患者,甚至更多。這將意味著在推出後的第一年內,每年 2,000 名患者的 KMT2A 事件的滲透率達到 50%,這是一個了不起的結果。Revuforj 的使用正逐漸擴展到更早期的治療方案,索賠數據顯示,約 70% 的使用集中在二線和三線治療中,其中 50% 來自二線或首次復發患者。

  • Claims data is also showing significant combination use with one-third of patients receiving Revuforj in combination with another standard of care therapy, venetoclax being the most common. This trend highlights physicians' comfort with the Revuforj profile and the potential for average treatment durations to extend over time as treatment patterns mature.

    索賠數據也顯示,合併用藥的情況十分普遍,三分之一的患者在接受 Revuforj 治療時會與其他標準療法聯合使用,其中最常見的是與維奈托克聯合使用。這一趨勢凸顯了醫生們對 Revuforj 治療方案的信心,以及隨著治療模式的成熟,平均治療持續時間可能會隨時間延長。

  • Consistent with last quarter, an estimated one-third of KMT2A patients treated with Revuforj have proceeded to a stem cell transplant, and we continue to see patients being put back on Revuforj by their physicians after a three to four month pause for engraftment.

    與上一季一致,估計有三分之一接受 Revuforj 治療的 KMT2A 患者進行了乾細胞移植,我們繼續看到患者在停用 Revuforj 三到四個月以進行植入後,由醫生重新開始使用 Revuforj。

  • We estimate that 35% to 40% of transplant patients have restarted Revuforj with that percentage expected to build over time as more patients clear the engraftment period and physicians gain more experience using Revuforj post-transplant. As we've seen in our clinical trial and expanded access program experience, we expect patients could stay on therapy for one to two years post-transplant, given the high risk of relapse and the favorable tolerability of Revuforj.

    我們估計有 35% 到 40% 的移植患者重新開始使用 Revuforj,隨著更多患者度過植入期,醫生在移植後使用 Revuforj 獲得更多經驗,預計這一比例會隨著時間的推移而增加。正如我們在臨床試驗和擴大使用計劃的經驗中所看到的,考慮到復發風險高以及 Revuforj 的良好耐受性,我們預計患者在移植後可以繼續接受治療一到兩年。

  • As Revuforj is used earlier in the treatment paradigm and more patients restart after transplant, we expect this will translate into a significant increase in the overall average duration of therapy. Based on our experience to date, we anticipate the average duration of therapy for KMT2A patients will be 4 to 6 months this year and 6 to 12 months in 2026 as treatment patterns further mature.

    由於 Revuforj 在治療方案中更早使用,並且有更多患者在移植後重新開始治療,我們預計這將導致治療的平均持續時間顯著增加。根據我們目前的經驗,我們預計今年 KMT2A 患者的平均治療時間為 4 至 6 個月,到 2026 年隨著治療模式的進一步成熟,治療持續時間將為 6 至 12 個月。

  • Let's turn to NPM1, the next important growth driver for Revuforj. As shown on slide 5, the second indication approved for Revuforj expands our annual total addressable US population from approximately 2,000 to 6,500 incident patients across both genetic subtypes in the relapsed/refractory setting, a $2 billion-plus market opportunity.

    讓我們來看看 NPM1,這是 Revuforj 的下一個重要成長驅動因素。如幻燈片 5 所示,Revuforj 核准的第二個適應症使我們每年在美國可接觸的總患者人數從大約 2,000 人增加到 6,500 人,涵蓋復發/難治性兩種基因亞型,這是一個超過 20 億美元的市場機會。

  • Moving to slide 6. Our promotional expansion into NPM1 began quickly once we received approval on Friday, October 24, with broad communication outreach to all relevant treatment centers and health care practitioners.

    切換到第6張投影片。10 月 24 日星期五獲得批准後,我們迅速開始了對 NPM1 的推廣擴展,並向所有相關的治療中心和醫療保健從業人員進行了廣泛的溝通。

  • The very next day, we had members of our field team trained and promoting the new indication at an oncology conference. and our engagement with HCPs has only expanded from there. We are pleased with the early progress we have made driving awareness of the expanded indication and generating demand in NPM1. Physicians are enthusiastic to have Revuforj as a new effective option for their NPM1 patients. Our success in NPM1 is going to be driven by two main factors: First, the breadth and strength of the Revuforj efficacy data and the overall product profile.

    第二天,我們就安排了現場團隊成員接受培訓,並在腫瘤學會議上推廣這項新適應症。此後,我們與醫療保健專業人員的合作範圍不斷擴大。我們對在提高人們對 NPM1 擴大適應症的認識和創造需求方面取得的早期進展感到滿意。醫生們很高興能將 Revuforj 視為 NPM1 患者的一種新的有效治療選擇。我們在 NPM1 的成功將取決於兩個主要因素:首先是 Revuforj 功效數據的廣度和強度以及整體產品概況。

  • With unmatched efficacy data across multiple patient subtypes, we are positioned to serve patients and secure dominant market share, given that physicians consider efficacy the most important factor driving their prescribing decisions. We are also the only company now and for the foreseeable future with a menin inhibitor that is FDA approved for multiple acute leukemia subtypes in patients one year and older.

    憑藉著在多種患者亞型中無可比擬的療效數據,我們有能力服務患者並獲得市場主導地位,因為醫生將療效視為影響其處方決策的最重要因素。目前,在可預見的未來,我們是唯一一家擁有經 FDA 批准用於治療一歲及以上患者多種急性白血病亞型的 menin 抑制劑的公司。

  • The ability to use one efficacious and generally well-tolerated drug across 40% to 45% of patients with AML is a huge benefit to practitioners and payers. Second, we have a solid commercial foundation that we're leveraging, including a large prescriber base that has already seen excellent clinical results with Revuforj and has experienced how easy we've made it for their patients to gain access to the drug. Physicians have already treated well over 1,000 patients with Revuforj across nearly one year of commercial use, clinical trials, and our EAP.

    對於 40% 至 45% 的 AML 患者而言,能夠使用一種療效顯著且耐受性良好的藥物,這對醫生和支付方來說都是一個巨大的好處。其次,我們擁有堅實的商業基礎,並正在充分利用這一基礎,包括龐大的處方醫生群體,他們已經看到了 Revuforj 的出色臨床效果,並且體驗到了我們讓他們的患者更容易獲得該藥物的過程。在近一年的商業使用、臨床試驗和我們的 EAP 計劃中,醫生們已經使用 Revuforj 治療了超過 1000 名患者。

  • From launch through the end of September, 70% of Tier 1 and Tier 2 accounts in the US have started using Revuforj on a regular basis. Physicians tell us it typically takes two or three patients to develop loyalty and habit with a new oncology medicine, and most of the major centers have already built up that comfort and muscle memory with Revuforj. Beyond the largest institutions, adoption is also increasing across all other sizes of accounts, including community practices. Our broad and growing prescriber base gives us a significant competitive advantage as we expand into NPM1.

    從上線到 9 月底,美國 70% 的一級和二級帳戶已開始定期使用 Revuforj。醫生告訴我們,通常需要兩到三名患者才能對一種新的腫瘤藥物產生忠誠度和習慣,而大多數主要中心已經對 Revuforj 建立了這種舒適感和肌肉記憶。除了規模最大的機構之外,其他各種規模的帳戶(包括社區實踐)的採用率也在不斷提高。我們龐大且不斷成長的處方醫生群體為我們拓展 NPM1 業務帶來了顯著的競爭優勢。

  • The positive experience accounts have had with Revuforj reflects the world-class commercial organization and infrastructure we have built to deliver to patients. We have an efficient limited distribution model with an average time from prescription to first fill of less than four days.

    客戶對 Revuforj 的積極體驗反映了我們為服務患者而建立的世界一流的商業組織和基礎設施。我們採用高效率的有限分銷模式,從開處方到首次配藥的平均時間不到四天。

  • Our highly experienced customer engagement team has long-standing relationships with key prescribers and accounts. Formulary coverage for KMT2A is already in place for 97% of covered lives and is expected to build rapidly for NPM1. While it builds, we expect the reimbursement rate to be high given the NCCN Guideline listing for NPM1 and the existing KMT2A coverage. We have everything we need for a successful expansion into NPM1 and look forward to providing further updates as this exciting launch progresses.

    我們經驗豐富的客戶關係團隊與主要處方醫生和客戶建立了長期合作關係。KMT2A 的處方集涵蓋範圍已涵蓋 97% 的受保人,預計 NPM1 的覆蓋範圍也將迅速擴大。雖然還在建設中,但鑑於 NPM1 已列入 NCCN 指南,且 KMT2A 已覆蓋,我們預計報銷率會很高。我們已具備成功擴展至 NPM1 所需的一切條件,並期待隨著這一激動人心的發布進程提供更多更新資訊。

  • Turning to Niktimvo on slide 7. We saw robust Niktimvo growth in the third-quarter with $45.8 million in net revenue, up 27% from the prior quarter. We continue to receive excellent feedback from HCPs on the rapid and durable improvements they are observing with Niktimvo across some of the most difficult-to-treat organs associated with chronic GVHD. We are steadily adding new patients and patients are staying on therapy.

    前往幻燈片 7 中的 Niktimvo。Niktimvo 第三季實現了強勁成長,淨收入達 4,580 萬美元,比上一季成長 27%。我們不斷收到來自醫療保健專業人員的極好回饋,他們觀察到 Niktimvo 在一些與慢性 GVHD 相關的最難治療的器官中取得了快速而持久的改善。我們不斷增加新患者,患者也都在堅持接受治療。

  • From the start of the launch through the end of the third-quarter, 8,500 infusions have been administered to 1,100 patients. Usage has been mostly in the fourth line, but it is growing in the third line, with the recent decrease in REZUROCK sales corresponding with increased adoption of Niktimvo. Of the patients who started Niktimvo in Q1, approximately 80% remain on therapy today. The breadth and depth of prescribing continues to grow with 90% of bone marrow transplant centers in the US prescribing Niktimvo, with all centers placing repeat orders year-to-date.

    從計畫啟動到第三季末,已為 1100 名患者進行了 8500 次輸液。該產品主要在第四線使用,但在第三線的使用量正在增長,最近 REZUROCK 銷售的下降與 Niktimvo 的採用率增加相對應。第一季開始接受 Niktimvo 治療的患者中,約有 80% 目前仍在接受治療。Niktimvo 的處方範圍和深度持續成長,美國 90% 的骨髓移植中心都在開立 Niktimvo 處方,所有中心今年迄今都下了重複訂單。

  • While we've made excellent progress in the first eight months, we still have significant room to continue growing given the scale of the unmet need with approximately 6,500 patients in the US requiring three or more lines of therapy, representing a $2 billion market opportunity, as shown on slide 8.

    儘管我們在前八個月取得了卓越的進展,但鑑於美國約有 6,500 名患者需要三種或更多種療法,我們仍有很大的成長空間,這代表著 20 億美元的市場機會,如幻燈片 8 所示。

  • I'll close by saying that, I'm thrilled by the progress we have made with Revuforj and Niktimvo. Both medicines are delivering for patients and on blockbuster trajectories. Achieving success as a commercial organization takes great products, great plans and great execution, and we have all three, positioning Syndax for sustained growth for years to come.

    最後我想說,我對我們與 Revuforj 和 Niktimvo 的進展感到非常興奮。這兩種藥物都對患者有效,並且有望成為暢銷藥物。作為一家商業機構,要想取得成功,需要優秀的產品、出色的計劃和卓越的執行力,而我們三者兼備,這使得 Syndax 在未來幾年能夠持續成長。

  • With that, I'll hand the call over to Nick to discuss our upcoming data presentations at ASH. Nick?

    接下來,我將把電話交給 Nick,讓他來討論我們即將在 ASH 大會上進行的數據展示。缺口?

  • Nicholas Botwood - Head - Research and Development, Chief Medical Officer

    Nicholas Botwood - Head - Research and Development, Chief Medical Officer

  • It's a pleasure to be on the call today. Thank you, Steve, and to discuss the strong presence Syndax will have at ASH with 23 abstracts accepted for presentation, including 6 oral presentations, highlighting our scientific leadership in menin inhibition and CSF-1R inhibition. Starting with Revuforj or revumenib. Collectively, the abstracts highlight the remarkable activity and tolerability of revumenib in multiple genetic subtypes, both as a monotherapy and in combination with standard of care therapies across the acute leukemia treatment continuum.

    今天能參加這次電話會議真是太好了。謝謝史蒂夫,接下來我們將討論 Syndax 在 ASH 大會上的強大影響力,我們共有 23 篇摘要被接受進行展示,其中包括 6 篇口頭報告,這突顯了我們在 menin 抑制和 CSF-1R 抑制方面的科學領先地位。首先使用Revuforj或revumenib。總的來說,這些摘要強調了revumenib在多種基因亞型中具有顯著的活性和耐受性,無論是作為單藥療法還是與急性白血病治療過程中的標準療法聯合使用。

  • Slide 9 summarizes the first real-world evidence for menin inhibitor. Data from the first 18 patients treated commercially with Revuforj at Moffitt Cancer Center show favorable tolerability and excellent clinical activity across multiple genetic subtypes and settings. Patients with NPM1, KMT2A and NUP98 acute leukemias are included in the data set. 15 patients received Revuforj in the relapsed/refractory setting, 2 in frontline and 1 after stem cell transplant without prior Revuforj treatment. Notably, nearly 80% of the patients received Revuforj in combination with standard of care regimens, most commonly venetoclax plus HMA. At the time of the abstract data cutoff, median follow-up was relatively short at about four months.

    第 9 張投影片總結了 menin 抑制劑的第一個真實世界證據。來自莫菲特癌症中心使用 Revuforj 進行商業治療的前 18 名患者的數據顯示,該藥物在多種基因亞型和治療環境中均具有良好的耐受性和優異的臨床活性。本資料集納入了NPM1、KMT2A和NUP98急性白血病患者。其中15例患者在復發/難治性治療中接受了Revuforj治療,2例為第一線治療,1例為幹細胞移植後未接受過Revuforj治療的患者。值得注意的是,近 80% 的患者接受了 Revuforj 與標準治療方案的聯合治療,最常見的是維奈托克加 HMA。在摘要數據截止時,中位追蹤時間相對較短,約四個月。

  • 16 patients were efficacy evaluable. Among 14 patients treated for morphological marrow disease relapse, 79% achieved an overall response. Rates of MRD negativity by flow cytometry were high at 86% and 67% for KMT2A and NPM1 responders, respectively. Four patients or 29% of the population treated for morphological disease proceeded to a stem cell transplant. Three patients received Revuforj as maintenance post-transplant, including two who resumed Revuforj post-transplant and who started post-transplant without prior Revuforj treatment.

    16 名患者可進行療效評估。在接受形態學骨髓疾病復發治療的 14 名患者中,79% 的患者達到了整體緩解。KMT2A 和 NPM1 應答者的 MRD 陰性率分別為 86% 和 67%,透過流式細胞儀檢測得出。接受形態學疾病治療的患者中有 4 例(佔總人口的 29%)進行了幹細胞移植。三名患者在移植後接受了 Revuforj 維持治療,其中兩名患者在移植後重新開始接受 Revuforj 治療,而這兩名患者在移植後開始接受 Revuforj 治療之前沒有接受過 Revuforj 治療。

  • It's also noteworthy that there were two additional patients who were treated with Revuforj NPM1 MRD positivity with one of the patients achieving MRD negativity at the data cutoff. The potential use of revumenib as an MRD eraser in HOX/MEIS-driven tumors is an area of high clinical interest with multiple ongoing studies exploring this area.

    值得注意的是,還有兩名患者接受了 Revuforj NPM1 MRD 陽性治療,其中一名患者在數據截止時達到了 MRD 陰性。revumenib 作為 HOX/MEIS 驅動腫瘤的 MRD 清除劑的潛在用途是一個備受臨床關注的領域,目前有多項研究正在探索這一領域。

  • Importantly, Revuforj was well tolerated in this real-world cohort, consistent with what we have observed among more than 1,000 patients treated across our broader clinical trial compassionate use and commercial experience. There was a low rate of revumenib dose reductions and no AEs led to revumenib discontinuation. DS and QTC were well managed with no events of either above Grade 3.

    重要的是,Revuforj 在這個真實世界隊列中耐受性良好,這與我們透過更廣泛的臨床試驗同情用藥和商業經驗治療的 1000 多名患者所觀察到的情況一致。revumenib 劑量減少率很低,沒有不良事件導致 revumenib 停藥。DS 和 QTC 都得到了很好的管理,沒有發生任何 3 級以上的事件。

  • The first real-world data set provides important insight into the breadth of Revuforj usage we are observing at leading academic institutions like Moffitt. The use in KMT22A, NPM1 and NUP98 underscores the clinical value of the data we have presented, showing activity in multiple genetic subtypes, one of the several differentiating features of the revumenib profile.

    第一個真實世界的資料集為我們深入了解像莫菲特這樣的領先學術機構對 Revuforj 的使用範圍提供了重要見解。KMT22A、NPM1 和 NUP98 的應用凸顯了我們所展示數據的臨床價值,表明其對多種基因亞型具有活性,這是 revumenib 特性的幾個區別特徵之一。

  • The high rate of combination therapy observed highlights physicians' comfort with revumenib's safety profile and their desire to combine therapies with the hope of achieving deeper and more durable responses. We look forward to the presentation of longer-term follow-up data from Moffitt at ASH. This presentation will be the first in a series of real-world data sets we will be collecting and presenting in partnership with leading physicians and centers.

    觀察到的高聯合治療率凸顯了醫生們對瑞維尼安全性的信心,以及他們希望透過聯合治療獲得更深層、更持久療效的願望。我們期待在 ASH 會議上看到 Moffitt 公佈更長期的追蹤數據。本次演講將是我們與頂尖醫師和中心合作收集和展示的一系列真實世界資料集中的第一個。

  • Turning to slide 10 and the frontline setting. We are pleased to share data from the first 17 patients enrolled in the newly diagnosed cohort of the SAVE trial. This trial is evaluating revumenib in combination with venetoclax and decitabine/cedazuridine in the relapsed/refractory and frontline settings. These new data show the combination was well tolerated in newly diagnosed patients with high rates of complete remission or CR and MRD negativity. Among newly diagnosed patients with NPM1 or KMT2A, 88% of evaluable patients achieved a CR. 100% of patients with CR were MRD negative by flow cytometry.

    翻到第 10 張投影片,了解前線狀況。我們很高興與大家分享 SAVE 試驗新診斷群組中首批 17 名患者的數據。該試驗正在評估 revumenib 與 venetoclax 和 decitabine/cedazuridine 合併用於復發/難治性和一線治療的療效。這些新數據顯示,新確診的患者對此聯合療法耐受性良好,完全緩解率(CR)和微小殘留病灶(MRD)陰性率很高。在新診斷的 NPM1 或 KMT2A 患者中,88% 可評估的患者達到完全緩解。 100% 的完全緩解患者以流式細胞儀檢測 MRD 為陰性。

  • Five patients or 29% proceeded to transplant. Two of these patients had resumed revumenib as post-transplant maintenance at the time of the data cutoff. At a median follow-up of six months, median OS and EFS were not reached. The combination was well tolerated. DS and QTC were well managed with no events of QTC above Grade 2 and no events of DS above Grade 3.

    五名患者(佔 29%)接受了移植手術。截至數據截止時,其中兩名患者已恢復使用瑞維美尼作為移植後維持治療。中位追蹤時間為 6 個月,中位總存活期 (OS) 和無事件存活期 (EFS) 尚未達到。此組合療法耐受性良好。DS 和 QTC 都得到了很好的控制,沒有發生 2 級以上的 QTC 事件,也沒有發生 3 級以上的 DS 事件。

  • This is an important data set that builds on the encouraging results observed in the BEAT-AML trial of revumenib with venetoclax and azacitidine in newly diagnosed patients with AML. The concordance of the results from two different studies and different centers bolsters our confidence in the potential for revumenib in combination with low-intensity therapy to transform the treatment paradigm for newly diagnosed NPM1 or KMT2A AML.

    這是一個重要的數據集,它建立在 BEAT-AML 試驗中觀察到的令人鼓舞的結果之上,該試驗研究了 revumenib 與 venetoclax 和 azacitidine 聯合治療新診斷的 AML 患者的療效。兩項不同研究和不同中心的結果的一致性增強了我們對revumenib與低強度療法聯合治療改變新診斷的NPM1或KMT2A AML治療模式的潛力的信心。

  • To realize the full therapeutic potential of Revuforj, we are laser-focused on advancing our frontline trials, including the pivotal EVOLVE -2 trial of revumenib with venaza that was initiated in collaboration with HOVON in the first-quarter of 2025, the first pivotal trial of a menin inhibitor to start enrolling in the frontline setting.

    為了充分發揮 Revuforj 的治療潛力,我們正全力推進一線試驗,包括與 HOVON 合作於 2025 年第一季啟動的 revumenib 與 venaza 的關鍵性 EVOLVE-2 試驗,這是首個在一線治療中開始招募患者的 menin 抑制劑關鍵性試驗。

  • Moving now to slide 11 and preliminary Phase I data supporting revumenib in combination with intensive chemotherapy or 7+3 in newly diagnosed patients with NPM1 or KMT2A AML. Data from two ongoing trials will be presented at ASH, including one led by the National Cancer Institute, or NCI, and one led by Syndax.

    現在來看看第 11 張投影片,初步 I 期數據支持 revumenib 與強化化療或 7+3 聯合用於治療新診斷的 NPM1 或 KMT2A AML 患者。ASH 將公佈兩項正在進行的試驗的數據,其中一項由美國國家癌症研究所 (NCI) 牽頭,另一項由 Syndax 牽頭。

  • Collectively, the early data from these trials show the tolerability of revumenib in combination with 7+3, along with high rates of CR, MRD negativity transplant and rapid count recovery. Both trials evaluated two dose levels of revumenib in combination with 7+3 induction and cytarabine consolidation.

    這些試驗的早期數據總體上表明,revumenib 與 7+3 聯合用藥具有良好的耐受性,且 CR 率高,MRD 陰性移植率高,細胞計數恢復快。這兩項試驗均評估了兩種劑量等級的瑞維美尼合併 7+3 誘導治療和阿糖胞苷鞏固治療。

  • Dose level 1 was revumenib at 110 or 220 milligrams every 12 hours with or without strong CYP3A4 inhibitor, respectively. Dose level 2 was at the FDA-approved monotherapy dose. No maximum tolerated dose has been identified and the adverse events reported were consistent with the known AE profile of intensive chemotherapy and revumenib.

    劑量等級 1 為每 12 小時給予 110 或 220 毫克 revumenib,分別與強效 CYP3A4 抑制劑合併或不合併使用。劑量等級 2 為 FDA 核准的單藥治療劑量。目前尚未確定最大耐受劑量,所報告的不良事件與強化化療和瑞維美尼的已知不良事件特徵一致。

  • In the NCI trial, one investigator-assessed dose-limiting toxicity or DLT was reported at dose level 2. This was one Grade 5 event of typhlitis or severe inflammation of the intestine, a complication that is known to occur in patients receiving intensive chemotherapy. There were no reports of DS or QTC prolongation of any grade. The NCI investigators concluded that revumenib appears to be well tolerated both with 7+3 induction and consolidation.

    在 NCI 試驗中,一名研究者評估的劑量限制性毒性 (DLT) 報告於劑量水準 2。這是一例 5 級盲腸炎或腸道嚴重發炎事件,已知這種併發症會發生在接受強化化療的患​​者身上。沒有報告任何級別的 DS 或 QTc 延長。美國國家癌症研究所的研究人員得出結論,revumenib 在 7+3 誘導和鞏固治療中似乎都具有良好的耐受性。

  • In the Syndax study, one DLT of Grade 3 QTc prolongation was reported at dose level 1. This patient discontinued revumenib during the first cycle. Notably, at the end of the first cycle, the patient had achieved an MRD-negative CR and went on to receive a stem cell transplant.

    在 Syndax 研究中,劑量等級 1 報告了一例 3 級 QTc 間期延長的劑量限制性毒性 (DLT)。該患者在第一個療程期間停止服用瑞伐美尼。值得注意的是,在第一個療程結束時,患者達到了 MRD 陰性 CR,並接受了幹細胞移植。

  • Turning to the promising clinical activity observed among nine efficacy evaluable NPM1 and KMT2A patients in the NCI trial at the dose level 1 or 2 at the time of the abstract data cutoff, 89% achieved a CR and 44% proceeded to transplant following treatment with revumenib. The median time to full count recovery, including both neutrophils and platelets was 25.5 days among patients with CR.

    在 NCI 試驗中,9 名可評估療效的 NPM1 和 KMT2A 患者在劑量水平 1 或 2 時觀察到了令人鼓舞的臨床活性,截至摘要數據截止時,89% 的患者達到了 CR,44% 的患者在接受 revumenib 治療後進行了移植。CR 患者中,嗜中性球和血小板數完全恢復的中位數時間為 25.5 天。

  • Among seven efficacy evaluable KMT2A patients in the Syndax trial at the time of the data cutoff, 100% achieved a CR and the MRD negativity rate was 100% among evaluable patients, 57% proceeded to transplant. At ASH, data from additional patients and follow-up will be presented from both trials.

    在 Syndax 試驗中,截至資料截止時,共有 7 名 KMT2A 患者可評估療效,其中 100% 的患者達到完全緩解,可評估患者的 MRD 陰性率為 100%,57% 的患者接受了移植。在 ASH 會議上,將公佈這兩項試驗的更多患者和追蹤數據。

  • Seeing positive early data from these two trials is very encouraging as we near the initiation of the registration-directed REVEAL program, which will evaluate revumenib in combination with intensive chemotherapy in newly diagnosed fit patients with NPM1 or KMT22A. We remain on track to initiate REVEAL by the end of '25 and look forward to providing further updates in due course.

    看到這兩項試驗的早期積極數據,我們感到非常鼓舞,因為我們即將啟動以註冊為導向的 REVEAL 項目,該項目將評估 revumenib 與強化化療聯合用於新診斷的 NPM1 或 KMT22A 患者。我們仍按計劃在 2025 年底前啟動 REVEAL 項目,並期待在適當的時候提供更多最新消息。

  • Turning to Slide 12. This abstract provides insights into the growing usage of revumenib we are observing in the post-transplant setting. In a retrospective review of 10 pediatric patients with KMT2A or NUP98r acute leukemia who received revumenib maintenance post-transplant at MD Anderson, revumenib was well tolerated with promising early efficacy.

    翻到第12張投影片。本文摘要深入分析了我們在移植後環境中觀察到的瑞維尼使用量不斷增長的現象。回顧性研究顯示,在 MD 安德森癌症中心接受移植後 revumenib 維持治療的 10 名 KMT2A 或 NUP98r 急性白血病兒童中,revumenib 耐受性良好,早期療效令人鼓舞。

  • Patients received a median of two cycles of revumenib prior to transplant and revumenib was initiated at a median of 111 days or roughly three to four months post-transplant, consistent with what we have observed in other data sets. The study planned for continuation of revumenib post-transplant for up to one year. Patients had completed a median of 11 cycles post-transplant at the time of the data cutoff. One patient continued for two years due to parental preference.

    患者在移植前平均接受了兩個週期的瑞維尼治療,瑞維尼治療在移植後平均 111 天(或大約三到四個月)開始,這與我們在其他數據集中觀察到的情況一致。研究計劃在移植後繼續使用revumenib長達一年。截至數據截止時,患者移植後平均已完成 11 個療程。一名患者因父母意願而繼續接受治療兩年。

  • This highlights the tolerability of Revuforj and reinforces prior feedback we have received from patients and families on the strong desire to stay on therapy that induced remission. At the last follow-up, all 10 patients were alive with no relapses, yielding an estimated one-year event-free survival of 100%. This is very encouraging results in a population with a high risk of relapse within the first year. The use of revumenib in the post-transplant setting is an area of high clinical interest.

    這凸顯了 Revuforj 的耐受性,並強化了我們先前從患者和家屬收到的回饋,即他們強烈希望繼續接受能夠誘導緩解的治療。在最後一次追蹤中,所有 10 名患者均存活且無復發,估計一年無事件存活率為 100%。對於第一年內復發風險較高的族群來說,這是一個非常令人鼓舞的結果。瑞維美尼在移植後治療的應用是一個備受臨床關注的領域。

  • In addition to the abstract just discussed, investigators from a different study will present a trial in progress poster describing a Phase I trial evaluating the safety and preliminary efficacy of revumenib as post-transplant maintenance in adult and pediatric patients with NPM1 or KMT2A. This trial, which is actively recruiting at City of Hope and Dana-Farber Cancer Institute is planning to continue revumenib for two years post-transplant.

    除了剛才討論的摘要之外,另一項研究的研究人員將展示一份正在進行的試驗海報,描述一項 I 期試驗,該試驗評估 revumenib 作為 NPM1 或 KMT2A 成人和兒童患者移植後維持治療的安全性和初步療效。這項試驗目前正在希望之城和丹娜-法伯癌症研究所積極招募受試者,並計劃在移植後繼續使用revumenib治療兩年。

  • Turning now to axatilimab on slide 13. I will briefly highlight three axatilimab abstracts that underscore the potential for long-term benefit in recurrent refractory chronic GVHD and the feasibility of combining ruxolitinib in newly diagnosed chronic GVHD. The first abstract shows that 33 of the 239 patients in the pivotal AGAVE-201 trial of axatilimab were still on therapy as of March 2025, with a median of 2.8 years on axatilimab.

    現在就來看看第 13 張投影片上的阿沙替利單抗。我將簡要介紹三篇關於阿沙替利單抗的摘要,這些摘要強調了阿沙替利單抗在復發性難治性慢性 GVHD 中具有長期獲益的潛力,以及在初診慢性 GVHD 中聯合魯索替尼治療的可行性。第一份摘要顯示,截至 2025 年 3 月,在 axatilimab 的關鍵性 AGAVE-201 試驗中,239 名患者中有 33 名仍在接受治療,接受 axatilimab 治療的中位數為 2.8 年。

  • Long-term data show a continued tolerable safety profile. The second abstract reports the safety and feasibility of axatilimab in patients who had a response at the FDA-approved dose of 0.3 milligrams per kilogram every two weeks and then transitioned to a double dose every four weeks. Among the 19 patients who switched, the four-week dosing was well tolerated with a median of 1.7 years on therapy after the dosing change.

    長期數據顯示,該產品具有持續可接受的安全性。第二篇摘要報告了阿沙利單抗在患者中的安全性和可行性,這些患者在接受 FDA 批准的每兩週每公斤 0.3 毫克劑量治療後有反應,然後過渡到每四周雙倍劑量。在 19 名轉換治療方案的患者中,四週一次的給藥方案耐受性良好,給藥方案改變後,患者接受治療的中位數為 1.7 年。

  • The third abstract reports interim safety data from 44 patients enrolled in the ongoing Phase II trial of axatilimab with ruxolitinib in newly diagnosed chronic GVHD. The data showed the combination was well tolerated, paving the way for the further development of this potentially steroid-sparing regimen.

    第三篇摘要報告了正在進行的阿沙替利單抗合併魯索替尼治療新診斷的慢性 GVHD 的 II 期試驗中 44 名患者的中期安全性數據。數據顯示,該組合療法耐受性良好,為進一步開發這種可能減少類固醇用量的療法鋪平了道路。

  • Importantly, this is one of two ongoing trials that have the potential to expand axatilimab into the frontline setting in combination with standard of care therapies. In summary, this year's ASH will be another exciting meeting for Syndax. After watching the clinical community's enthusiasm for revumenib and axatilimab grow over the year, it's a pleasure to have the opportunity to share the next wave of data that will help drive forward the next phase of progress for patients. And with that, I will hand over the call to Keith to discuss our financials.

    重要的是,這是兩項正在進行的試驗之一,這兩項試驗有可能將阿沙利單抗推廣到一線治療中,並與標準治療方案聯合使用。總之,今年的 ASH 大會對 Syndax 來說將是另一個令人興奮的會議。在過去一年裡,我們看到臨床界對瑞維美尼和阿沙利單抗的熱情日益高漲,很高興有機會分享下一波數據,這將有助於推動下一階段的患者治療進展。接下來,我將把電話交給基思,讓他來討論我們的財務狀況。

  • Keith Goldan - Chief Financial Officer, Treasurer

    Keith Goldan - Chief Financial Officer, Treasurer

  • Thanks, Nick. Earlier this afternoon, we reported detailed third-quarter 2025 financial results. I will touch on a few key points on slide 14. For the third-quarter of 2025, we reported Revuforj net revenue of $32 million. Quarter-over-quarter sales growth was driven by demand as inventory levels remained at two to three weeks.

    謝謝你,尼克。今天下午早些時候,我們公佈了 2025 年第三季的詳細財務業績。我將在第 14 張投影片中簡要提及幾個重點。2025年第三季度,我們公佈Revuforj淨收入為3,200萬美元。季增長銷售成長主要受需求驅動,庫存水準保持在兩到三週的範圍內。

  • While prescription demand increased 25% quarter-over-quarter, net sales grew 12% over the prior quarter. The primary reason for this delta was an increase in Revuforj's gross to net adjustments in the third-quarter versus 2Q, while still within the 20% to 25% guidance range we previously provided. The increase was due to higher proportion of 340B business in the quarter as well as higher exposure to Medicare and Medicaid, all of which mandate statutory discounts.

    雖然處方藥需求較上季成長了 25%,但淨銷售額比上一季成長了 12%。造成這一差異的主要原因是 Revuforj 第三季毛利與淨利調整額較第二季有所增加,但仍在我們先前提供的 20% 至 25% 的指導範圍內。該季度增長是由於 340B 業務佔比增加,以及對聯邦醫療保險和醫療補助的敞口增加,所有這些都強制要求法定折扣。

  • There was also a slight drawdown of inventory in the channel this quarter, while still within the two to three week range that we previously guided. Looking ahead, we expect sales growth to meaningfully accelerate over the coming quarters with the approval in NPM1 and an increasing average duration of therapy in KMT2A as more patients receive Revuforj as long-term maintenance therapy post-transplant.

    本季通路庫存略有下降,但仍在我們先前預測的兩到三週的範圍內。展望未來,隨著 NPM1 的批准以及 KMT2A 患者接受 Revuforj 作為移植後長期維持治療,平均治療持續時間的增加,我們預計未來幾季的銷售成長將顯著加速。

  • Turning to Niktimvo. Syndax reported $13.9 million in collaboration revenue after deducting the cost of sales and commercial expenses. Importantly, Niktimvo continues to be positive cash flow contributor to Syndax. We continue to expect the Niktimvo margin contribution, defined as collaboration revenue recorded by Syndax as a percentage of Niktimvo net sales to be in the 25% to 30% range in the near term and increase longer term as sales grow and the partnership leverages a largely fixed expense base. We expect continued robust growth given GVHD is a chronic disease where there is a high response rate to Niktimvo and the average patient will likely remain on therapy for years.

    轉向尼克提姆沃。Syndax 報告稱,扣除銷售成本和商業費用後,合作收入為 1,390 萬美元。重要的是,Niktimvo 繼續為 Syndax 帶來正向現金流。我們繼續預計,Niktimvo 的利潤貢獻(定義為 Syndax 記錄的合作收入佔 Niktimvo 淨銷售額的百分比)在短期內將達到 25% 至 30%,並且隨著銷售額的增長以及合作關係利用了主要固定的費用基礎,長期來看這一貢獻將會增加。我們預計 GVHD 將繼續保持強勁成長,因為 GVHD 是一種慢性疾病,對 Niktimvo 的反應率很高,而且普通患者可能需要接受治療數年。

  • Turning to the balance sheet. We continue to maintain a strong financial position with $456 million in cash, equivalents and short- and long-term investments as of September 30. As I've said in the past and reiterate today, we expect Syndax will reach profitability with current funds on hand. In fact, my confidence is higher today given that both drugs are outperforming our original forecasts. We are confident we can execute commercially, and also deliver on our integrated clinical development plans for both drugs while keeping operating expenses at today's levels.

    接下來看一下資產負債表。截至9月30日,我們擁有4.56億美元的現金、現金等價物以及短期和長期投資,財務狀況依然穩健。正如我過去所說,今天我再次重申,我們預計 Syndax 將利用現有資金獲利。事實上,鑑於這兩種藥物的療效都超過了我們最初的預測,我今天的信心更加堅定了。我們有信心在商業上取得成功,並實現兩種藥物的綜合臨床開發計劃,同時將營運費用維持在目前的水平。

  • Our cash, combined with increasing Revuforj and Niktimvo cash flow contributions alongside an expected fixed expense base will drive our path to profitability. Michael?

    我們的現金,加上 Revuforj 和 Niktimvo 不斷增長的現金流貢獻,以及預期的固定支出基礎,將推動我們走向獲利之路。麥可?

  • Michael Metzger - Chief Executive Officer, Director

    Michael Metzger - Chief Executive Officer, Director

  • Thank you, Keith. Turning to slide 15. Syndax has never been in a stronger position than we are today. We have two first and best-in-class therapies on blockbuster trajectories with plenty of room for growth in the front line and beyond. We have an outstanding team that is consistently executing at the highest level, culminating in three FDA approvals and launches within roughly one year, a remarkable achievement.

    謝謝你,基斯。翻到第15張投影片。Syndax 目前的處境從未像今天這樣強勢。我們擁有兩種一流且前景廣闊的療法,預計將成為重磅炸彈級產品,在一線及其他領域都擁有巨大的成長空間。我們擁有一支傑出的團隊,他們始終保持著最高水準的執行力,最終在一年左右的時間內獲得了三項 FDA 批准並成功上市,這是一項了不起的成就。

  • With two exceptional product launches underway, a strong balance sheet and stable expense outlook, we are on the road to profitability and fulfilling our mission as a company. I would like to close by thanking everyone who has made it possible for us to make a major impact for patients, especially our talented Syndax employees and long-term investors. With that, I would like to open the call for questions.

    憑藉兩款卓越的產品正在推出、強勁的資產負債表和穩定的支出前景,我們正走在盈利的道路上,並實現我們作為一家公司的使命。最後,我要感謝所有幫助我們為患者帶來重大影響的人,特別是我們才華橫溢的 Syndax 員工和長期投資者。接下來,我想接受大家的提問。

  • Operator?

    操作員?

  • Operator

    Operator

  • (Operator Instructions)

    (操作說明)

  • Anupam Rama, JPMorgan.

    Anupam Rama,摩根大通。

  • Priyanka Grover - Analyst

    Priyanka Grover - Analyst

  • This is Priyanka on for Anupam. Can you review how Revuforj's place in lines of therapy has evolved in the commercial setting during the launch? And how do you think this will translate for the NPM1 setting? Would physicians with experience with Revuforj be more willing to use it in earlier lines of therapy?

    這是普里揚卡為阿努帕姆主持節目。您能否回顧一下Revuforj在上市期間,在商業環境中,其在治療領域中的地位是如何演變的?你認為這對於 NPM1 設定會有什麼影響?有使用Revuforj經驗的醫師是否更願意在早期治療中使用它?

  • Michael Metzger - Chief Executive Officer, Director

    Michael Metzger - Chief Executive Officer, Director

  • Yes. Thanks, Priyanka, for the question. I'll take that. So, look, lines of therapy, I think the question is relating to how is it being used in clinical practice. For KMT2A, we have said that about 70% of our business is second or third line, so that's first relapse or second relapse.

    是的。謝謝 Priyanka 的提問。我接受。所以,你看,治療方案,我認為問題在於它在臨床上是如何應用的。對於 KMT2A,我們說過,我們大約 70% 的業務是二線或三線治療,也就是第一次復發或第二次復發。

  • That's a stark change from what we've seen in our clinical trial where third and fourth line was the average patient. And so, what the meaning of that is that it enables patients to be treated earlier. They tend to do better, stay on treatment longer. We've seen an uptick in the amount of patients going to transplant as a result. We've seen in our clinical trial, we saw 25% of patients go to transplant.

    這與我們臨床試驗中觀察到的情況截然不同,在臨床試驗中,三線和四線治療的患者是平均值。因此,這意味著患者可以更早接受治療。他們往往治療效果更好,接受治療的時間也更長。因此,我們看到接受器官移植的患者數量增加。我們在臨床試驗中發現,25% 的患者最終接受了移植手術。

  • In our commercial experience, we've seen about a third go to transplant. So, we've actually seen quite a shift and that we think will manifest in patients staying on drug longer over time in KMT2A. So that's been very meaningful. And we expect with NPM1, these patients are getting to transplant as well. We're also seeing high rates of response.

    根據我們的商業經驗,大約有三分之一的患者最終接受了移植手術。所以,我們實際上已經看到了相當大的轉變,我們認為這將體現在 KMT2A 患者隨著時間的推移而延長用藥時間。所以這非常有意義。我們預計,有了 NPM1,這些患者也能接受移植手術。我們也看到了很高的回應率。

  • About half of the patients get to response. We do expect them to be treated earlier and earlier in the treatment journey. And as we've talked about, patients are also being treated in combination. So that will drive patients to earlier utilization within their journey. So, this is, I think, a trend that will continue not just for KMT2A, but for NPM1 and ultimately should lead to better utilization, longer utilization for patients.

    大約一半的患者會出現療效。我們預計他們在治療過程中會越來越早接受治療。正如我們之前討論過的,患者也在接受聯合治療。這樣就能促使患者在治療過程中更早接受治療。所以,我認為這種趨勢不僅適用於 KMT2A,也適用於 NPM1,最終應該會為患者帶來更好的利用率和更長的利用期。

  • Operator

    Operator

  • Corinne Johnson, Goldman Sachs.

    科琳·約翰遜,高盛集團。

  • Corinne Johnson - Analyst

    Corinne Johnson - Analyst

  • You spoke about a 6- to 12-month range for duration of therapy in 2026. Could you help us think about the key factors that you're looking to understand in order to narrow that range? And when could that start to be reflected in the revenue trajectory?

    您曾提到,2026 年的治療時間為 6 至 12 個月。您能否幫助我們思考一下,為了縮小範圍,您希望了解的關鍵因素有哪些?那麼,這種情況何時才能開始反映在營收成長軌跡上呢?

  • Michael Metzger - Chief Executive Officer, Director

    Michael Metzger - Chief Executive Officer, Director

  • Thanks, Corinne. Great question. Look, I think 2026, we said that in 2025, as we started with new patients staying on therapy in the range of four to six months, and that was really reflective of new patient starts and some patients coming back from -- on maintenance therapy post-transplant. But the impact of that in terms of duration of therapy won't be felt really until 2026, where more patients will be returning from transplant and receiving maintenance. We expect, obviously, to have a launch now with NPM1.

    謝謝你,科琳。問得好。你看,我認為 2026 年,我們在 2025 年說過,當我們開始讓新患者接受治療四到六個月時,這確實反映了新患者的開始治療以及一些患者在移植後接受維持治療的情況。但就治療持續時間而言,這種影響要到 2026 年才會真正顯現,屆時將有更多患者從移植手術中恢復並接受維持治療。我們顯然希望現在能與 NPM1 一起發布。

  • So additional patients will be receiving therapy. And then we'll have some of those patients go to transplant as well. But I think the mix of patients between KMT2A where you'll have slightly longer duration of treatment based on the fact that more patients in KMT2A will go to transplant than NPM1. That mix of patients will be -- will have a slightly longer duration of therapy. NPM1, larger patient population.

    因此,將有更多患者接受治療。然後,我們會讓其中一些病人接受移植手術。但我認為,KMT2A 患者的混合情況,由於 KMT2A 患者接受移植的比例高於 NPM1 患者,因此治療時間會稍長。這部分患者的治療時間會稍長。NPM1,患者族群較大。

  • So, we expect more patients to be on drug than perhaps what we'll see with KMT2A ultimately, but a slightly shorter duration based on the fact that fewer patients will go to transplant, although some will. So, it's the mix of those two patient populations that we believe will drive to 6 to 12 months in the second year.

    因此,我們預期接受藥物治療的患者人數可能會比最終接受 KMT2A 治療的患者人數更多,但治療持續時間可能會略短一些,因為接受移植的患者人數較少,儘管有些患者最終會接受移植。因此,我們認為正是這兩類患者族群的混合,將使第二年的治療時間延長至 6 到 12 個月。

  • Operator

    Operator

  • Brad Canino, Guggenheim.

    布拉德卡尼諾,古根漢美術館。

  • Bradley Canino - Analyst

    Bradley Canino - Analyst

  • Nice commercial momentum on the quarter. First question for you. Have you looked at all where the maintenance restart rate is for the patients who started revumenib during the first few months of launch? Because obviously, the 35% to 40% you're reporting is weighed down by the bolus of recent patients getting transplants, but not yet undergoing the ability to get maintenance. So, were you able to do a longitudinal analysis at all to understand where that restart rate number can grow to?

    本季商業發展勢頭良好。第一個問題問你。您是否查看過在瑞伐美尼上市最初幾個月開始接受治療的患者的維持治療重啟率?顯然,你報告的 35% 到 40% 的比例被近期接受移植手術但尚未獲得維持治療的患者人數拉低了。那麼,您是否能夠進行縱向分析,以了解重啟率可能會成長到什麼程度?

  • Michael Metzger - Chief Executive Officer, Director

    Michael Metzger - Chief Executive Officer, Director

  • Excellent question, Brad. I think we've seen some progress this quarter in the restart rate where we saw last quarter about a third of patients restarting maintenance therapy. Now it's up to about 35%, 40%. We do believe that will grow over time additional patients, steady flow.

    問得好,布拉德。我認為本季我們在重啟率方面取得了一些進展,上個季度我們看到大約三分之一的患者重新開始維持治療。現在大約是 35%、40%。我們相信隨著時間的推移,患者數量會不斷增加,流量會穩定成長。

  • We've seen this quarter going to transplant, again, not fully offset by the patients coming back. We do think that, that will build in the next quarter and the quarters beyond. We don't have an upper limit of what percentage of patients will come back, although what we've heard from physicians is that they're very keen to put them back on therapy. And so, what we've heard is as many as 80%, 90%, they've given figures that they would say almost all their patients hard to estimate what the upper limit is of what percentage of patients will come back.

    我們看到本季移植手術的數量再次增加,但並沒有完全被康復患者的數量所抵消。我們認為,這種情況會在接下來的一個季度以及之後的幾個季度持續改善。我們沒有病患復診率的上限,不過我們從醫師那裡了解到,他們非常渴望讓病患重新接受治療。因此,我們聽到的消息是,高達 80%、90% 的患者會再次就診,他們給出的數據表明,幾乎所有患者都會再次就診,很難估計患者復診率的上限是多少。

  • It is impacted by other factors that are beyond the control of physician, if a patient has extenuating circumstances. But I think the inclination is to bring them back and put them on therapy. So, we'll just have to wait and see how that manifests. But it's a good sign that even now we're starting to see more patients come back on.

    如果患者有特殊情況,則可能受到其他醫生無法控制的因素的影響。但我認為目前的趨勢是把他們帶回來接受治療。所以,我們只能拭目以待,看看事情會如何發展。但令人欣慰的是,即使現在我們也開始看到越來越多的患者重新開始接受治療。

  • Operator

    Operator

  • Clara Dong, Jefferies.

    克拉拉‧董,傑富瑞。

  • Clara Dong - Equity Analyst

    Clara Dong - Equity Analyst

  • So, as we think about the relationship between prescription growth and revenue growth, so could you provide some perspective in terms of how revenue per prescription might evolve as the patient mix shifts from predominantly KMT2A in the third-quarter to include more NPM1 patients going forward?

    因此,當我們思考處方成長與收入成長之間的關係時,您能否就每張處方的收入如何變化提供一些見解,以說明隨著患者組成從第三季度以 KMT2A 患者為主轉變為未來納入更多 NPM1 患者,每張處方的收入可能會如何變化?

  • Michael Metzger - Chief Executive Officer, Director

    Michael Metzger - Chief Executive Officer, Director

  • Yes. Keith, do you want to take that question?

    是的。基思,你想回答這個問題嗎?

  • Keith Goldan - Chief Financial Officer, Treasurer

    Keith Goldan - Chief Financial Officer, Treasurer

  • Yes, Clara, thanks for the question. We really don't expect much of a change in terms of average revenue per prescription as more and more NPM1 patients start to make their way into our prescribing base.

    是的,克拉拉,謝謝你的提問。隨著越來越多的 NPM1 患者加入我們的處方群體,我們預計每張處方的平均收入不會有太大變化。

  • Operator

    Operator

  • Peter Lawson, Barclays.

    Peter Lawson,巴克萊銀行。

  • Peter Lawson - Analyst

    Peter Lawson - Analyst

  • Just on the delta between quarter-over-quarter growth results versus the Rx rate. And is there any way to break down that gap between gross to net versus inventory timing that we should be thinking about or any changes that we should be thinking about going forward? And then I've got a question just on if there's been any friction again NPM1 authorizations and payer access.

    僅考慮季度環比增長結果與處方藥價格之間的差異。那麼,我們應該考慮哪些方法來縮小毛利與淨利和庫存時間之間的差距,或者我們應該考慮哪些未來的變化?然後我還有一個問題,關於 NPM1 授權和付款人存取方面是否出現了任何摩擦。

  • Michael Metzger - Chief Executive Officer, Director

    Michael Metzger - Chief Executive Officer, Director

  • Peter, thanks for the question regarding quarter-over-quarter growth and the breakdown between what we're seeing in those metrics. But Keith, why don't you take that question?

    Peter,感謝你提出的關於季度環比增長以及我們從這些指標中看到的細分情況的問題。但是基思,你為什麼不回答這個問題呢?

  • Keith Goldan - Chief Financial Officer, Treasurer

    Keith Goldan - Chief Financial Officer, Treasurer

  • Yes, Peter, thank you. I'd start off by saying that generally, when you see a disconnect in a quarterly result between net revenue and prescription growth as is often the case, especially in launches, there's generally two factors that play into that, and it's generally gross differences in gross to net and differences in inventory stocking. And as I said, both can fluctuate quarter-to-quarter. In this period, as I said in my prepared remarks, the delta was primarily driven by higher gross to net adjustments. I want to emphasize it's still within the range we provided.

    是的,彼得,謝謝你。首先我想說的是,通常情況下,當季度業績中淨收入與處方增長出現脫節時(這種情況經常發生,尤其是在產品上市初期),通常有兩個因素會導致這種情況,分別是毛利與淨利之間的差異以及庫存差異。正如我所說,這兩者都可能出現季度波動。正如我在事先準備好的演講稿中所說,在此期間,差額主要是由於毛利與淨利之間的調整幅度增大所致。我想強調的是,它仍然在我們提供的範圍內。

  • So a very tight range of 20% to 25%, but we did have slightly higher 340B chargebacks and slightly higher Medicaid to Medicare utilization. As I said, both remain within the guidance range as does inventory. The two to three weeks, which is very typical of specialty launches, rare disease launches using the type of distribution network that we do. But we did see a slight drawdown in inventory, which those two factors combined to explain the disconnect between prescription growth of 25% and revenue growth of 12%.

    因此,範圍非常窄,在 20% 到 25% 之間,但我們的 340B 退款略高,醫療補助轉醫保的使用率也略高。正如我所說,這兩項指標以及庫存均在指導範圍內。兩到三週的時間,對於我們這種分銷網絡下的特藥上市、罕見疾病藥物上市來說非常典型。但我們確實看到庫存略有下降,這兩個因素加在一起解釋了處方增長 25% 與收入增長 12% 之間的脫節。

  • Michael Metzger - Chief Executive Officer, Director

    Michael Metzger - Chief Executive Officer, Director

  • Yes. And I would just add that, again, just to remind you, Peter, that we had about a third of revenue go away, if you will, for patients who were going to transplant. And we had an offset of only about 35% to 40% of those patients coming back. So that will build over time, but that was, of course, is a factor in what could have been a different quarter from a top line perspective.

    是的。我還要補充一點,再次提醒你,彼得,我們大約有三分之一的收入因為病人需要器官移植而消失了。而且,只有大約 35% 到 40% 的患者再次復發。所以隨著時間的推移,這種情況會逐漸改善,但這當然也是導致本季營收狀況有所不同的因素。

  • Steven Closter - Chief Commercial Officer

    Steven Closter - Chief Commercial Officer

  • And I think the other part of Peter's question was just on the payer side.

    我認為彼得問題的另一部分只是關於付款方的問題。

  • Michael Metzger - Chief Executive Officer, Director

    Michael Metzger - Chief Executive Officer, Director

  • Yes.

    是的。

  • Steven Closter - Chief Commercial Officer

    Steven Closter - Chief Commercial Officer

  • So, as you know, payer access formulary coverage for Revuforj really since launch has been simply outstanding. By month five, we hit 97% formulary coverage. So, in essence, unfettered access for commercial Part D and Medicaid patients. There has, of course, been some off-label prescriptions outside of KMT2A. We know that it's been about 10% since launch, and that will obviously -- the usage will grow with the indication.

    如您所知,Revuforj 自上市以來,其在支付方處方集覆蓋方面一直表現出色。到第五個月,我們的藥品目錄覆蓋率達到了 97%。因此,從本質上講,商業保險D部分和醫療補助患者可以不受限制地獲得醫療服務。當然,KMT2A 之外也存在一些超適應症用藥的情況。我們知道自推出以來,使用率約為 10%,而且很明顯,隨著指標的出現,使用率將會成長。

  • But we haven't had any pushback from payers for the most part, even in advance of the NPM1 indication. Once the publication came out in May in blood, obviously, the NCCN listing was in the third week of September. That's really what payers need to get products covered. Even when they're not yet indicated, obviously, the indication is going to accelerate that. So, the payer team has been talking to payers since we submitted the sNDA earlier this year, coverage will build quickly.

    但總的來說,我們還沒有收到來自支付方的任何反對意見,甚至在 NPM1 適應症公佈之前也是如此。該出版物於 5 月以血腥的方式出版,顯然,NCCN 的收錄是在 9 月的第三週。這正是支付方需要用來確保產品獲得健保覆蓋的條件。即使目前還沒有跡象表明存在這種情況,但很明顯,這種跡象將會加速這一進程。因此,自從我們今年稍早提交補充新藥申請以來,支付方團隊一直在與支付方進行溝通,覆蓋範圍將迅速擴大。

  • But in the interim, Peter, as coverage builds, the claims will be adjudicated and paid for. So, patients will still enjoy open access to Revuforj moving forward until that coverage is permanent.

    但在此期間,彼得,隨著保險覆蓋範圍的擴大,索賠將會得到裁決和賠償。因此,在保險覆蓋範圍永久化之前,患者仍可繼續免費使用 Revuforj。

  • Operator

    Operator

  • Ellen Horste, TD Cowen.

    艾倫·霍斯特,TD Cowen。

  • Ellen Horste - Analyst

    Ellen Horste - Analyst

  • Congrats on the quarter and all the exciting abstracts. Just wondering a couple of things about the NPM1 launch. One, if you noticed any modest uptick in the final days of Q3 where you did have that inclusion NCCN Guidelines? And then more broadly, wondering how we should think about the launch trajectory in the NPM1 population in terms of market penetration relative to the launch in the KMT2A market, given that, as you said, it's a larger population, but it's likely to face some competition. Any thoughts there would be helpful.

    恭喜你本季取得的成績以及所有精彩的論文摘要。關於NPM1的發布,我有幾個問題想請教一下。第一,您是否注意到在第三季最後幾天,納入 NCCN 指南的病例數量略有上升?更廣泛地說,考慮到NPM1人群規模更大,但可能會面臨一些競爭,我們應該如何看待NPM1人群的上市軌跡,以及相對於KMT2A市場的上市軌跡,就市場滲透率而言。任何想法都將不勝感激。

  • Michael Metzger - Chief Executive Officer, Director

    Michael Metzger - Chief Executive Officer, Director

  • Yes, Ellen, thanks for the question. I'll start off with some comments about the quarter, and then I'll turn it over to Steve to talk about the launch trajectory. So first, strong start to the quarter. I'd say HCPs are excited. As you'd imagine, awareness is quite high.

    是的,艾倫,謝謝你的提問。我先就本季做一些評論,然後把麥克風交給史蒂夫,讓他談談發射軌跡。首先,本季開局強勁。我覺得醫護人員都很興奮。正如你所料,人們的意識水平相當高。

  • Increase in prescriptions, we're seeing it, accounts are ordering and have expanded. The setup for the forward, I think, is quite positive. We had guidelines as you know, late September. So that didn't impact the quarter too much, but sets us up well for this quarter coming and approval in October. So, the combination really sets our launch at a very -- we think, in a very good way.

    我們看到處方量增加了,帳戶數量增加了,而且訂單也增加了。我認為,前鋒線的配置相當積極。如您所知,我們在九月下旬制定了指導方針。所以這件事對本季影響不大,但為即將到來的本季和10月份的批准奠定了良好的基礎。所以,這種組合確實讓我們的發表會處於一個非常好的狀態——我們認為,這是一個非常好的狀態。

  • So, we expect a solid Q4, and we expect this to add meaningfully to the book of business that we have in KMT2A. And we talked about the factors that will drive KMT2A business, which is new patient starts steady as well as maintenance and patients coming back from -- on to maintenance therapy, which will grow. So, we're expecting a good Q4. Maybe I'll turn it over to Steve to talk about launch trajectory in NPM1.

    因此,我們預計第四季度業績穩健,並預計這將顯著增加我們在 KMT2A 的業務量。我們討論了推動 KMT2A 業務發展的因素,包括新患者的穩定成長、維持治療以及從維持治療中恢復的患者,這些因素都將成長。所以,我們預計第四季業績會不錯。也許我會把話題交給史蒂夫,讓他談談 NPM1 中的發射軌跡。

  • Steven Closter - Chief Commercial Officer

    Steven Closter - Chief Commercial Officer

  • Yes. Just to add on to Michael's comments. I mean, awareness and excitement around the new indication is incredibly high. Our field force was trained within days, and we were talking to customers the day after approval, I think I mentioned in my prior comments. We're excited about the launch.

    是的。我補充一下邁克爾的評論。我的意思是,人們對這項新適應症的認知度和興奮度都非常高。我想我在之前的評論中提到過,我們的現場團隊在幾天內就接受了培訓,並在批准後的第二天就開始與客戶洽談。我們對此次發布感到非常興奮。

  • I know physicians are as well. There's three main drivers as we think about this. We'll see if and when, there is competition, we prepare as though there is, which is why we operate at a very high level and execute as best as we can. First is product profile. We think we have an unsurpassed profile, really best-in-class, two indications covering nearly half of the population, adults and peds, AML, ALL.

    我知道醫生們也是如此。我們在思考這個問題時,主要有三個驅動因素。我們會看看何時會有競爭,如果有競爭,我們會做好應對的準備,所以我們會以非常高的水平運作,並盡我們所能做到最好。首先是產品概況。我們認為我們擁有無與倫比的優勢,真正是同類最佳,兩種適應症涵蓋了近一半的人口,包括成人和兒童,即急性髓性白血病 (AML) 和急性淋巴細胞白血病 (ALL)。

  • We've talked about this efficacy is the most important attribute for any cancer oncology heme or indication. And we believe we have the best data, and that's what physicians tell us. The drug is well tolerated, range of doses. Physicians have proven that they can use it in KMT2A very widely as well as in NPM1, and they'll have more experience doing that.

    我們已經討論過,療效是任何癌症腫瘤血液或適應症最重要的屬性。我們相信我們擁有最好的數據,醫生也這樣告訴我們。此藥物耐受性良好,劑量範圍廣泛。醫生們已經證明,他們可以在 KMT2A 和 NPM1 中廣泛使用它,而且他們在這方面會有更多經驗。

  • Second piece is really just around relationships and ability to execute. We've been in the market for selling for almost a year, but our field team was in place even six months in advance of that. We've got great relationships. The experience that physicians have had has been excellent around the drug. We've talked about 1,000 patients treated to date.

    第二點其實就是關於人際關係和執行能力。我們進入市場銷售已經將近一年了,但我們的現場團隊甚至提前六個月就已經到位。我們關係很好。醫生在使用這種藥物方面的經驗非常好。我們已經談到了迄今為止接受治療的1000名患者。

  • We'll be over 1,000 patients treated commercially. That means a lot. We've got a growing account base and not just large accounts, it's been medium-sized accounts as well as community practices, really showing unmet need and also how easy it is to use the drug. And that experience accounts have it is meaningful. It's really two to three patients to gain some serious muscle memory in use.

    我們將有超過1000名患者接受商業治療。這意義重大。我們的客戶群不斷增長,而且不僅僅是大客戶,還有中型客戶以及社區診所,這充分錶明了未被滿足的需求,同時也表明了這種藥物的使用是多麼容易。而且,這種經驗說明它很有意義。確實需要兩到三個病人才能在使用過程中形成明顯的肌肉記憶。

  • And the last piece, which we highlighted on this call is really the ongoing clinical development program of the data that we've been supporting and whether they're collaborative studies, ISTs, and health economic work. That data set will build over time, giving physicians the kind of data sets and data points they need to continue to use this drug widely. So, we feel we're in a great position moving forward.

    最後一點,也是我們在這次電話會議上重點提到的,是我們一直在支持的數據的持續臨床開發計劃,無論是合作研究、IST 還是衛生經濟學工作。隨著時間的推移,該數據集將會不斷積累,為醫生提供他們繼續廣泛使用這種藥物所需的數據集和數據點。所以,我們感覺我們未來發展前景非常有利。

  • Michael Metzger - Chief Executive Officer, Director

    Michael Metzger - Chief Executive Officer, Director

  • Yes. And I would just add that, I think we have -- it's quite simple in terms of our view on competition, Revuforj has the broadest and strongest efficacy profile. This is a very much of an efficacy-driven market where you have physicians looking to get patients to remission. It's -- they're very sick and they need, I think, a very strong drug to drive home and get patients into remission. And Revuforj is that and it has the broadest profile to achieve that in all types of patients.

    是的。我還要補充一點,我認為——就我們對競爭的看法而言,這很簡單,Revuforj 具有最廣泛、最強的功效。這是一個非常注重療效的市場,醫生們希望幫助患者達到緩解期。他們病情非常嚴重,我認為他們需要一種非常強大的藥物來幫助患者康復並達到緩解狀態。Revuforj 正是這樣一種藥物,它具有最廣泛的適用性,能夠幫助所有類型的患者達到這一目標。

  • So, I think we're in a very good position going into this launch. And we have a pretty simple view on how the competitive dynamic will play out. We should dominate this market.

    所以,我認為我們這次發表會處於非常有利的位置。我們對競爭格局將如何演變有著非常簡單的看法。我們應該主導這個市場。

  • Operator

    Operator

  • Stephen Willey, Stifel.

    Stephen Willey,Stifel。

  • Stephen Willey - Analyst

    Stephen Willey - Analyst

  • Just one, I guess, on the soon to initiate REVEAL trial in frontline patients with intensive chemo. I know we don't have protocol details yet, but I was just curious about how you're philosophically thinking about specifically evaluating the contribution of maintenance therapy within the protocol itself, just given what J&J now appears to be doing in the frontline setting and whether you think trial design differences may have some kind of competitive implications on the labeling front as it pertains to maintenance therapy explicitly?

    我想,只有一位患者參與了即將啟動的針對第一線接受強化化療患者的 REVEAL 試驗。我知道我們目前還沒有方案細節,但我只是好奇,鑑於強生公司目前在一線治療領域所採取的措施,您在方案本身中是如何從哲學角度考慮評估維持治療的作用的?您是否認為試驗設計上的差異可能會對維持治療相關的標籤標註產生某種競爭影響?

  • Michael Metzger - Chief Executive Officer, Director

    Michael Metzger - Chief Executive Officer, Director

  • Steve, thanks. A great and important question about how do we think about evaluating or how are we evaluating maintenance therapy in our REVEAL trial. So maybe I'll turn it over to Nick to take that.

    史蒂夫,謝謝。這是一個非常重要的問題,即我們如何看待或如何評估 REVEAL 試驗中的維持治療。所以也許我會把它交給尼克來做。

  • Nicholas Botwood - Head - Research and Development, Chief Medical Officer

    Nicholas Botwood - Head - Research and Development, Chief Medical Officer

  • Yes, it's an important question. It's something we've thought a lot about, and we're looking forward to, as we've indicated, starting our REVEAL program soon this quarter and very encouraged actually by the data that we've already presented and we'll follow up more in terms of combinations with intensive chemotherapy, which looks very encouraging.

    是的,這是一個重要的問題。我們已經對此進行了深入思考,並且正如我們之前所指出的,我們期待著在本季度盡快啟動我們的 REVEAL 項目,事實上,我們已經公佈的數據讓我們非常鼓舞,我們將進一步跟進與強化化療的聯合應用,這看起來非常令人鼓舞。

  • So, maintenance is an important question. And the way we're thinking about this is that we have a number of studies that will generate data that support maintenance, looking at different doses, different approaches that will support treatment practice. So, maintenance is obviously a consideration within the pivotal studies themselves.

    所以,維護保養是一個重要議題。我們正在考慮的問題是,我們有許多研究將產生支持維持治療的數據,研究不同的劑量、不同的方法,以支持治療實踐。因此,維護顯然是關鍵研究本身需要考慮的因素。

  • All of our studies allow for maintenance after transplant, and we'll be able to ascertain some data from that. But in terms of the overall profile, we'll be looking at a broad body of evidence to support use in the maintenance setting in the front line.

    我們所有的研究都允許在移植後進行維持治療,我們將能夠從中獲得一些數據。但就整體情況而言,我們將考察大量證據,以支持在一線維護環境中使用該技術。

  • Operator

    Operator

  • Yigal Nochomovitz, Citigroup.

    Yigal Nochomovitz,花旗集團。

  • Yigal Nochomovitz - Analyst

    Yigal Nochomovitz - Analyst

  • I was just curious, when you look at the trends between the community practices and academic, are you seeing any differences there in terms of the percent going to transplant? And then related to that, are you seeing any differences in those segments in the percent returning to maintenance post-transplant?

    我只是好奇,當你觀察社區診所和學術機構之間的趨勢時,你是否發現移植百分比有任何差異?與此相關的是,您是否觀察到這些族群在移植後恢復維持治療的比例上有任何差異?

  • Michael Metzger - Chief Executive Officer, Director

    Michael Metzger - Chief Executive Officer, Director

  • Yigal, thanks for the questions. I'm going to turn it over to Steve to kind of to address that. First question relates to, are we seeing differences in transplant between community practices and academic practices?

    Yigal,謝謝你的提問。我將把這個問題交給史蒂夫來解答。第一個問題是,我們是否發現社區診所和學術診所在器官移植上有差異?

  • Steven Closter - Chief Commercial Officer

    Steven Closter - Chief Commercial Officer

  • Yes. So, we know there's usage across academia as well as community. Majority is in academics, and these patients are very sick. That's not uncommon, whether it's for KMT2A, we expect that early for NPM1. So, the majority has been in academia.

    是的。所以,我們知道學術界和社群都有人使用。大部分患者來自學術界,而且這些患者病情非常嚴重。這並不罕見,無論是KMT2A還是NPM1,我們都預期這種情況會很早發生。因此,大多數人都在學術界工作。

  • We're not able to peel apart the rates -- the treatment rates and maintenance. We do have some claims analysis, which trails. Some of that data that we've been shared today is from that claims analysis. Perhaps as the data set grows, we'll be able to pull apart that dynamic. But for now, it's -- the rates we've shared are really across the spectrum.

    我們無法將治療費用和維持費用分開來看。我們確實有一些索賠分析,但結果並不理想。我們今天分享的部分數據就來自理賠分析。或許隨著資料集的成長,我們就能剖析這種動態關係。但就目前而言,我們分享的利率確實涵蓋了各種範圍。

  • Yigal Nochomovitz - Analyst

    Yigal Nochomovitz - Analyst

  • Okay. And I know you mentioned, obviously, for NPM1, you'll have less transplants. But nonetheless, since the drug was just approved in the expanded label, -- is there a situation where some patients that did have transplants that were NPM1 could still get Revuforj as maintenance even if they didn't get it before or that wouldn't happen?

    好的。我知道你提到過,對於 NPM1 來說,移植次數肯定會減少。但是,由於該藥物剛剛獲準擴大適應症,是否存在這樣一種情況:一些接受過 NPM1 移植的患者,即使之前沒有接受過 Revuforj 治療,現在仍然可以接受該藥物作為維持治療,或者這種情況不會發生?

  • Michael Metzger - Chief Executive Officer, Director

    Michael Metzger - Chief Executive Officer, Director

  • Excellent question, Yigal. I'll turn it over to Nick.

    問得好,伊加爾。我把它交給尼克。

  • Nicholas Botwood - Head - Research and Development, Chief Medical Officer

    Nicholas Botwood - Head - Research and Development, Chief Medical Officer

  • Actually, yes. Interestingly, we are seeing that. And in fact, there was a reported case in the real-world series that I mentioned from Moffitt, you'll see one patient actually start on Revuforj having I assume as a function of timing, not had treatment prior to the transplant. So, there is now -- and we've heard from some other centers as well that there is a desire if they haven't had Revuforj prior to the transplant that they would start on it as a maintenance therapy afterwards.

    實際上,是的。有趣的是,我們正在看到這種情況。事實上,在我提到的莫菲特真實世界系列報導中,有一個病例,你會看到一名患者實際上開始服用Revuforj,我推測這是由於時間關係,他在移植之前沒有接受過治療。所以現在——我們也從其他一些中心了解到——如果患者在移植前沒有接受過 Revuforj 治療,他們希望在移植後開始接受 Revuforj 作為維持治療。

  • Operator

    Operator

  • Justin Zelin, BTIG.

    Justin Zelin,BTIG。

  • Justin Zelin - Equity Analyst

    Justin Zelin - Equity Analyst

  • Congrats on the quarter. Just wondering if you could give us an update on how the safety profile has been faring in the real world. Do you see patients discontinuing the drug at all for any adverse events?

    恭喜你本季取得佳績。我想了解一下,您能否向我們介紹一下該安全性能指標在實際應用中的表現如何?您是否觀察到有患者因不良反應而停止服用該藥物?

  • Michael Metzger - Chief Executive Officer, Director

    Michael Metzger - Chief Executive Officer, Director

  • Justin, thanks. I'm going to turn it to Nick for a safety profile.

    賈斯汀,謝謝。我要把它交給尼克做個安全評估。

  • Nicholas Botwood - Head - Research and Development, Chief Medical Officer

    Nicholas Botwood - Head - Research and Development, Chief Medical Officer

  • Yes. I would say -- I mean, we -- I would say we probably spoke to 1,000 physicians since the launch and the reception has been very favorable. Again, we've talked about very consistent safety profile. They're very familiar with managing it, in particular, very low rates of serious cardiac complications across our clinical trial program of over 1,000 patients. So, it's very well managed.

    是的。我想說——我的意思是,我們——我想說,自推出以來,我們可能已經與 1000 名醫生進行了交談,反應非常好。我們再次強調,安全性非常穩定。他們非常熟悉如何管理這種情況,尤其是在我們超過 1000 名患者的臨床試驗項目中,嚴重心臟併發症的發生率非常低。所以,管理得非常好。

  • We actually see, as you can see from the data -- the extensive data we're going to be presented at ASH, you see very low rates of discontinuations from therapy. The adverse event profile is well managed, and that's consistent with what we see in the commercial use as well. They're very experienced in using the drug, and it's well managed.

    實際上,正如您從數據中看到的那樣——我們將在 ASH 上展示的大量數據表明,治療中斷率非常低。不良事件控制良好,這與我們從商業應用中看到的情況一致。他們使用該藥物經驗非常豐富,而且管理得井井有條。

  • Operator

    Operator

  • Salim Syed, Mizuho.

    Salim Syed,瑞穗銀行。

  • Salim Syed - Analyst

    Salim Syed - Analyst

  • Great. Congrats on the quarter, guys. Just one for me as well on the safety side. I know people are focused and you guys mentioned this on the approval call, the one case of Torsades, it's now listed in the black box there. I know one case, and you mentioned previously, you don't expect things to really change with it.

    偉大的。恭喜各位,本季業績圓滿完成。出於安全考慮,我也只需要一個。我知道大家都很關注這件事,你們在審批電話會議上也提到了這一點,那例Torsades病例現在已經列入黑框中了。我知道一個案例,你之前也提到過,你不期待它能真正改變現狀。

  • And I get that. But as you kind of think about first line here where there are more patients and you are treating thousands of patients per year, is it not reasonable to think here that you're going to get more cases like that, and that's something that you're going to have to educate or manage around, especially if zifto does not end up with that on the label?

    我明白。但是,考慮到第一線治療中患者更多,每年要治療成千上萬的患者,難道不應該考慮會遇到更多類似的情況嗎?如果 Zifto 的標籤上最終沒有包含這些內容,那麼你就需要對病人進行教育或進行相應的管理。

  • Michael Metzger - Chief Executive Officer, Director

    Michael Metzger - Chief Executive Officer, Director

  • Nick, do you want to?

    尼克,你願意嗎?

  • Nicholas Botwood - Head - Research and Development, Chief Medical Officer

    Nicholas Botwood - Head - Research and Development, Chief Medical Officer

  • Well, I think actually, the answer is no because the rates in the frontline setting actually seem to be lower. This may have something to do with patients may be fitter newly diagnosed. They've had low -- they haven't had exposure to prior anthracyclines and things. So we're seeing low rates. The other benefit of our frontline studies, of course, is that they will be randomized.

    嗯,我覺得答案是否定的,因為一線崗位的感染率似乎實際上更低。這可能與新確診的患者身體狀況較好有關。他們接觸過蒽環類藥物等藥物的風險很低。所以我們看到利率很低。當然,我們一線研究的另一個好處是,它們是隨機的。

  • There'll be a control arm. It will be much easier to ascertain the true rates of drug-related side effects with the control. So, I think it will be much more informed. And based on what we've seen to date, we're really seeing very low rates of serious cardiac events or discontinuations.

    會有一個控制臂。有了對照組,就更容易確定藥物相關副作用的真實發生率。所以,我認為它會更加資訊豐富。根據我們目前所看到的,嚴重心臟事件或停藥的發生率確實非常低。

  • Michael Metzger - Chief Executive Officer, Director

    Michael Metzger - Chief Executive Officer, Director

  • Yes. I'd just add on top of that, I mean, we -- as Nick mentioned, we've talked to hundreds of physicians since launch. And here's the takeaway. I mean, they're excited about the profile. Revuforj's efficacy sort of stands out for sure, very manageable safety profile.

    是的。我還要補充一點,正如尼克所提到的,自從產品推出以來,我們已經與數百名醫生進行了交流。這就是重點。我的意思是,他們對這個個人資料感到很興奮。Revuforj 的療效確實非常突出,而且安全性也很好控制。

  • They're not changing the way they practice based on the label and what they've seen. There's no new monitoring. So, they've been doing the same thing they've been doing for a year as we've launched KMT2A, and they've also treated NPM1 patients successfully during this time. So, I think they see Rev as a game changer for their patients and efficacy really matters the most here, and that's what they're focused on. So that's really where we lead things.

    他們不會因為標籤和所見所聞而改變自己的訓練方式。沒有新的監測措施。所以,自從我們推出 KMT2A 以來,他們一年來一直在做同樣的事情,並且在此期間也成功地治療了 NPM1 患者。所以,我認為他們把Rev視為病人的治療方案,療效在這裡才是最重要的,這也是他們關注的重點。所以,這就是我們真正想要引導的方向。

  • Operator

    Operator

  • David Dai, UBS.

    David Dai,瑞銀集團。

  • David Dai - Analyst

    David Dai - Analyst

  • Just a question on the 35% to 40% patients who resumed Revuforj post transplantation in third-quarter . Could you maybe provide some additional detail around the timing of the maintenance use? How long is the drug holiday before we'll see them coming back to the maintenance therapy?

    關於第三季移植後恢復使用Revuforj的35%至40%的患者,我有個問題。您能否提供一些關於維護使用時間的更多詳細資訊?停藥期有多長,才會重新開始維持治療?

  • Michael Metzger - Chief Executive Officer, Director

    Michael Metzger - Chief Executive Officer, Director

  • Great, David. Thanks for the question. Very simple. So, what we're seeing in our clinical experience, our commercial experience is reminiscent of what we've seen in our clinical experience, which is patients get about two to three months of therapy. For the ones who go to transplant, they usually get their response in that time frame.

    太好了,大衛。謝謝你的提問。非常簡單。所以,我們在臨床經驗和商業經驗中看到的情況,與我們在臨床經驗中看到的情況類似,那就是患者接受大約兩到三個月的治療。對於接受移植手術的患者來說,他們通常會在那個時間範圍內得到療效。

  • They go to transplant, so they're off Revuforj for a period of time, and then they resume about three to four months later. So it's, call it, six -- in the range of six months from start to resumption of therapy in the maintenance setting.

    它們需要進行移植手術,因此會有一段時間無法服用 Revuforj,然後大約三到四個月後才能恢復服用。所以,姑且稱之為六個月——從開始治療到在維持治療環境中恢復治療,大約需要六個月的時間。

  • Operator

    Operator

  • Mayank Mamtani, B. Riley.

    Mayank Mamtani,B. Riley。

  • Mayank Mamtani - Analyst

    Mayank Mamtani - Analyst

  • Congrats on strong momentum. Actually, a lot of demand indicators aligned with our recent physician survey. So, on the KMT2A versus NPM1 revenue split being obviously 90 to 10 right now, are you able to comment on when you'd expect that to be a little bit more balanced or even NPM be a bit more dominant from a timing standpoint?

    恭喜你們取得如此強勁的發展勢頭。事實上,許多需求指標都與我們最近的醫生調查結果相符。所以,目前KMT2A與NPM1的收入比例顯然是90比10,您能否評論一下,您預計這種情況何時會更加平衡,或者從時間角度來看,NPM何時會佔據主導地位?

  • I obviously recognize there are different dynamics here in play in terms of what you just commented on treatment duration and obviously, transplant dynamics and obviously, the larger starting patient population. And then I have a quick follow-up.

    我當然意識到,正如你剛才提到的,治療持續時間、移植動態以及更廣泛的初始患者群體,這裡存在著不同的動態因素。然後我還有一個簡短的後續問題。

  • Michael Metzger - Chief Executive Officer, Director

    Michael Metzger - Chief Executive Officer, Director

  • Yes. Thanks for the question. I think you answered it yourself. It's two different populations of patients. KMT2A is smaller than NPM1. We're expanding the population two to three times the size with NPM1. More patients do go to transplant that are KMT2A will have a slightly longer duration of treatment based on the fact that there's a group of patients -- a growing group of patients in KMT2A going to transplant and then coming back on for maintenance. NPM1 will have its own dynamics. But we do have the best profile, we believe, in both those segments. We're widely indicated for AML, ALL for KMT2A, adults and pediatrics, and we extend adults and pediatrics with NPM1.

    是的。謝謝你的提問。我覺得你自己已經回答這個問題了。這是兩類不同的患者群。KMT2A 比 NPM1 小。透過 NPM1,我們將人口規模擴大兩到三倍。接受移植的 KMT2A 患者數量較多,他們的治療時間會稍長一些,因為有一部分 KMT2A 患者接受移植後會回來接受維持治療。NPM1 將有其自身的運作機制。但我們相信,我們在這兩個領域都擁有最佳的優勢。我們的產品廣泛適用於 AML、KMT2A 型 ALL、成人和兒童,我們也透過 NPM1 擴大了成人和兒童的適用範圍。

  • So, we do have the broadest profile. We do expect to capture the largest share in NPM1 and dominate for both segments. So, I think this is -- it's a little difficult to tease out what's the contribution of parts at this point, but we do think that we'll have a majority share in both segments.

    所以,我們的客戶群最為廣泛。我們預計將在 NPM1 中佔據最大份額,並在兩個細分市場中都佔據主導地位。所以,我認為——目前很難區分各個部分的貢獻,但我們確實認為我們將在兩個細分市場中都佔多數。

  • Mayank Mamtani - Analyst

    Mayank Mamtani - Analyst

  • Okay. And on the impressive frontline AML data set from the save all-oral regimen, I believe that was and then the intensive chemo combination. We also saw a couple of peer frontline data sets that were released this morning. Any updated thoughts on how you're thinking of competitive positioning and even maybe regulatory strategy with different combination regimen trials in frontline based on some of this data? And the post-transplant one-year relapse-free rate was 100%, if I heard one-year EFS.

    好的。而從令人印象深刻的一線 AML 資料集來看,全口服方案的療效顯著,我相信這就是後來的強化化療組合方案。我們也看到了今天早上發布的一些同行前線資料集。基於這些數據,您對第一線不同聯合治療方案試驗的競爭定位,甚至是監管策略,有什麼新的思考嗎?移植後一年無復發率達到 100%,如果我聽到的是一年 EFS 的話。

  • Is that not something you could include in the label at some point? Or would you have to do that post maintenance frontline trial to get there?

    難道你不能在某個時候把這個內容加到標籤裡嗎?或者說,你必須先進行維修後的前線試驗才能達到這個目標?

  • Michael Metzger - Chief Executive Officer, Director

    Michael Metzger - Chief Executive Officer, Director

  • Yes. Great questions, I'm going to turn it over to Nick to maybe address each of those if you can.

    是的。問得好,我打算把這個問題交給尼克,讓他來逐一解答,如果你方便的話。

  • Nicholas Botwood - Head - Research and Development, Chief Medical Officer

    Nicholas Botwood - Head - Research and Development, Chief Medical Officer

  • Yes, I would be happy to. And I think we're going to have a very dominant presence at ASH, given the data we're going to be presenting across 12 abstracts. And as you say, very compelling data in the frontline setting from both our SAVE oral data, but also now our emerging data from intensive chemotherapy, which we will be updating. You'll see more numbers and more follow-up from both of the studies that we'll be presenting. When we look across, I would say, the competitive landscape, there's going to be a lot of combination data presented at ASH.

    是的,我很樂意。我認為,鑑於我們將在 12 篇摘要中展示的數據,我們將在 ASH 大會上佔據主導地位。正如您所說,無論是我們的 SAVE 口服數據,還是我們現在正在更新的強化化療數據,都提供了非常有說服力的前線治療數據。我們將展示這兩項研究的更多數據和後續結果。當我們放眼整個競爭格局時,我認為ASH大會上將會展示很多組合數據。

  • I think that overall, the profile for revumenib really is quite compelling, both in terms of the efficacy that we're showing consistently now, particularly when you look at the subsets of patients like KMT2A, where we're seeing a 100% response rate and 100% MRD negativity in what we've reported. It's really quite unparalleled. And again, it speaks to the depth and the breadth of the profile that we see with revumenib.

    我認為總體而言,revumenib 的療效確實非常顯著,無論是在我們目前持續展示的療效方面,還是在像 KMT2A 這樣的患者亞組中,我們報告的數據顯示,其緩解率和 MRD 陰性率均為 100%。這真是獨一無二。這再次說明了瑞維尼的療效特徵的深度和廣度。

  • So, I think we're very well positioned in the data we're going to be presenting. And when you think that, that's adding on to data that we've already presented like the BEAT-AML study previously in frontline in combination with venaza, it bodes very well for all of our frontline programs, both the REVEAL programs with intensive chemotherapy and also the EVOLVE-2 study that we're doing in collaboration with HOVON, which is well underway and has been enrolling since earlier this year.

    所以,我認為我們即將展示的數據非常有優勢。考慮到這一點,再加上我們已經公佈的數據,例如之前一線治療中與維納扎聯合使用的 BEAT-AML 研究,這對我們所有的一線治療項目來說都是一個非常好的預兆,包括採用強化化療的 REVEAL 項目,以及我們正在與 HOVON 合作開展的 EVOLVE-2 研究,該研究開始招募工作,並且從今年年初就開始招募。

  • So yes, I think a strong profile and nothing that looks differentiating in any of the other combination data we've looked at to date at ASH.

    所以,是的,我認為這是一個很強的指標,而且在我們迄今為止在 ASH 上看到的任何其他組合數據中,都沒有任何差異化的特徵。

  • Operator

    Operator

  • Jason Zemansky, Bank of America.

    傑森‧澤曼斯基,美國銀行。

  • Jason Zemansky - Analyst

    Jason Zemansky - Analyst

  • Congrats on the progress. I was hoping you could speak to the impact of the NPM1 approval on your gross to net and inventory trends as we head into the fourth-quarter and early next year. Given the size of the population relative to the KMT2A, I have to imagine that some of the headwinds may pivot to tailwinds and at a much more substantive impact. And then I guess, secondarily, if a patient returns to Revuforj following a transplant, how challenging is that, at least from an administrative or payer perspective? How difficult is it sort of restarting a patient like that?

    恭喜你取得進展。我希望您能談談 NPM1 批准對貴公司毛利淨利和庫存趨勢的影響,尤其是在我們即將進入第四季和明年年初的時候。考慮到國民黨第二屆政府的人口規模,我認為一些不利因素可能會轉變為有利因素,並產生更實質的影響。其次,如果患者在移植手術後返回 Revuforj,從行政或支付方的角度來看,這會有多大的挑戰?像這樣重新開始治療病人有多難?

  • Michael Metzger - Chief Executive Officer, Director

    Michael Metzger - Chief Executive Officer, Director

  • Jason, thanks for the questions. First question, impact of on GTN for NPM1. Keith, I'll address that.

    傑森,謝謝你的提問。第一個問題,NPM1 對 GTN 的影響。基思,我會處理這件事。

  • Keith Goldan - Chief Financial Officer, Treasurer

    Keith Goldan - Chief Financial Officer, Treasurer

  • Yes. The first one is a two-part question. With respect to inventory, Jason, we don't expect any changes. The two to three week guidance that we've given is going to grow in absolute terms as volumes grow. So, it's based on a trailing period of demand.

    是的。第一個問題包含兩個部分。傑森,關於庫存方面,我們預計不會有任何變化。我們給出的兩到三週的預測,隨著銷量的成長,絕對值也會成長。所以,它是基於過去一段時間的需求數據而得出的。

  • So, we expect inventory levels in our specialty distribution channel to stay at two to three weeks. With respect to your question on gross to net, the NPM1 indication may shift the mix in terms of payers. As you know, we offer no commercial rebates. There's the statutory rebates that I mentioned earlier in response to another question. But we have pretty good visibility, and we expect to remain in that 20% to 25% gross to net range.

    因此,我們預計我們在專業分銷管道的庫存水準將保持在兩到三週的水平。關於您提出的毛利與淨利的問題,NPM1 指標可能會改變付款方的組成。如您所知,我們不提供任何商業折扣。還有我之前在回答另一個問題時提到的法定退稅。但我們對獲利前景有相當清晰的預期,預計毛利率將維持在 20% 到 25% 的範圍內。

  • Michael Metzger - Chief Executive Officer, Director

    Michael Metzger - Chief Executive Officer, Director

  • Steve, do you want to take the second question?

    史蒂夫,你想回答第二個問題嗎?

  • Steven Closter - Chief Commercial Officer

    Steven Closter - Chief Commercial Officer

  • Yes. And maybe just a comment on the gross to net. I mean, NPM1 patients tend to be older, so Keith write the mix, it could go to more Medicare Part D from commercial. So, there might be some slight impact. In terms of, Jason, the question on impacting payers and this is patients coming back post a successful transplant as a restart or as we call maintenance treatment, we don't expect any pushback from payers.

    是的。或許還可以就毛利潤與淨利的關係提一點意見。我的意思是,NPM1 患者往往年齡較大,所以 Keith 寫道,混合方案可能會從商業保險轉向更多 Medicare Part D。所以,可能會有一些輕微的影響。傑森,就影響支付方的問題而言,這是患者在成功移植後重新開始治療或我們稱之為維持治療的情況,我們預計支付方不會有任何阻力。

  • There's been so few pushback from payers at this point really throughout the launch. And a lot of this is payers understand the unmet need, the value the drug brings. They've accepted the price. So, a vast majority of prescriptions are being paid for regardless of if they're KMT2A, NPM1 maintenance or even for other off-label use. So, we don't expect any pushback from payers on patients returning to a maintenance therapy.

    到目前為止,在整個發布過程中,付費用戶幾乎沒有提出任何反對意見。很大程度是因為支付者了解未被滿足的需求,以及這種藥物帶來的價值。他們接受了這個價格。因此,絕大多數處方藥無論用於 KMT2A、NPM1 維持治療或其他非適應症用途,都能獲得報銷。因此,我們預期支付方不會對患者恢復維持治療提出任何反對意見。

  • Operator

    Operator

  • This concludes our question-and-answer session. I will now turn the floor over to Mr. Michael Metzger for any closing remarks.

    我們的問答環節到此結束。現在我將把發言權交給麥可‧梅茲格先生,請他作總結發言。

  • Michael Metzger - Chief Executive Officer, Director

    Michael Metzger - Chief Executive Officer, Director

  • Thank you all. We appreciate you all tuning in today to discuss our recent progress and the exciting milestones ahead. We look forward to seeing many of you at the upcoming UBS, Guggenheim, Stifel, Jefferies and Encore Conferences -- Evercore Conferences as well as our ASH investor event in December. And with that, have a great evening. Thank you.

    謝謝大家。感謝各位今天收看我們的節目,共同探討我們近期的進展以及未來令人興奮的里程碑。我們期待在即將舉行的瑞銀集團、古根漢集團、史蒂費爾集團、傑富瑞集團和安可集團會議(即 Evercore 會議)以及 12 月的 ASH 投資者活動中見到各位。祝您有個美好的夜晚。謝謝。