亚盛医药2026年摩根大通医疗保健会议演讲实录（中英逐段对照）

$亚盛医药-B(06855)$ $亚盛医药(AAPG)$
一、会议核心要点总结

本次J.P. Morgan Healthcare Conference（2026年1月14日）是亚盛医药（AAPG）首次登上主会议舞台的关键亮相，核心围绕“全球商业化阶段的血液学/肿瘤学创新药企”定位，传递以下关键信息：

1. 公司基本盘：财务稳健+全球布局

成立16年（2009年），全球商业化阶段血液学/肿瘤学公司，双上市（纳斯达克+港交所）；

现金储备4.2亿美元，支撑研发至2027年；

全球员工近800人，总部覆盖中国苏州、美国马里兰州、澳大利亚悉尼；

知识产权：478项全球授权专利+500+待审申请，多篇论文发表于《JAMA Oncology》等顶级期刊。

2. 核心资产：2款商业化产品+强后期管线

公司已实现2款新型商业化产品（中国获批），并拥有“世界级创新、高度低风险”的后期管线：

奥雷巴替尼（Olverembatinib，第三代BCR-ABL抑制剂）： 中国2021年获批CML-CP（伴/不伴突变、2种TKI不耐受）； 全球进展：2项III期研究（FDA/EMA批准）、POLARIS-1一线Ph+ ALL（ASH数据：64% MRD阴性CR率，是普纳替尼的2倍）、SDH缺陷型GIST单药注册试验； 临床优势：覆盖T315I突变/复合突变（全球晚期CML首选潜力）、重度预处理耐药患者仍有效（失败普纳替尼/asciminib后单药MMR率达40%/30%）、4年随访安全性良好（血液学AE随时间减少）。

Lisaftoclax（APG-2575，新型口服Bcl-2抑制剂）： 中国6个月前获批BTK失败后CLL/SLL（全球首个BTK后单药获批）； 全球进展：4项注册试验（2项FDA/EMA批准）、GLORA-4一线高危MDS（全球唯一MDS III期研究）； 临床优势：安全性优于维奈克拉（0 TLS、无DDI、患者友好 dosing）、维奈克拉失败患者仍有效（AML/MDS ORR 31%）、中国获批“每日剂量递增”独特设计。

3. 管线亮点：First-in-class+差异化机制

BTK降解剂（APG-3288）：2025年快速推进（PCC到IND<2年），获FDA批准全球研究，可覆盖共价/非共价/突变BTK抑制剂耐药，未来可与Bcl-2联合用于淋巴瘤；

EED抑制剂（APG-5918，靶向PRC2）：覆盖血液瘤+实体瘤（前列腺癌）+贫血，中国完成地中海贫血患者研究，临床前显示与IMiD联合治骨髓瘤的潜力；

三激酶抑制剂（APG-2449，FAK/ALK/ROS1）：中国获批ALK/ROS1 III期，FAK可解决ALK抑制剂耐药；

MDM2-p53抑制剂：10年研究，多项II期，瞄准ACC/儿童肉瘤未满足需求。

4. 2025年关键里程碑

纳斯达克上市；

Lisaftoclax获中国NDA批准；

2项FDA/EMA注册试验批准（一线患者）；

推进POLARIS-1（Ph+ ALL）、GLORA-4（MDS）等关键注册试验入组。

5. 2026年战略重点

聚焦执行：完成所有注册研究入组；

商业化准备：为Lisaftoclax美国上市招CCO，2027年拟NDA；

管线推进：BTK降解剂、EED抑制剂（肿瘤+贫血）的美国/中国进展；

合作开放：不排除晚期/早期管线的合作。

二、后续关键催化剂（按时间&重要性排序）

1. 短期（2026年上半年）

BTK降解剂Phase I启动：全球首款BTK降解剂，FDA/中国同步入组，关注剂量递增安全性/PK/初步疗效（尤其淋巴瘤/CLL）；

注册试验入组加速：POLARIS-1（Ph+ ALL一线）、GLORA-4（MDS一线）、奥雷巴替尼SDH缺陷型GIST等注册试验的入组进度更新；

F-3资格落地：下月生效，通过ATM等方式吸引美欧投资者，提升流动性。

2. 中期（2026年下半年-2027年）

Lisaftoclax美国商业化准备：CCO到位、团队搭建完成，为2027年NDA做准备；

奥雷巴替尼全球数据读出：POLARIS-2（CML-CP III期）、SDH缺陷型GIST注册试验的顶线数据（有效性/安全性）；

EED抑制剂贫血数据：中国地中海贫血患者的临床概念验证（POC）数据更新；

MDM2-p53注册路径明确：针对ACC/儿童肉瘤的注册试验方案获批。

3. 长期（2027年及以后）

NDA申报与获批： 奥雷巴替尼：全球III期试验（如POLARIS-1）的NDA申报（美国/欧盟）； Lisaftoclax：美国NDA申报（基于GLORA-4等试验）；

商业化落地：Lisaftoclax美国上市（若获批）、奥雷巴替尼全球商业化（武田合作）；

联合疗法数据：奥雷巴替尼+Lisaftoclax（Ph+ ALL）、Bcl-2+MDM2-p53（AML/DLBCL）等的临床数据读出；

新靶点突破：BTK降解剂的Phase I数据、EED抑制剂肿瘤适应症的临床进展。

综上，亚盛医药的核心逻辑是“全球后期管线的强执行力+差异化产品的商业化潜力”，后续催化剂集中在注册试验入组/数据读出、NDA申报、商业化准备，以及新靶点（如BTK降解剂）的早期临床进展，持续验证其“超级后期药企”的价值。
亚盛医药2026年摩根大通医疗保健会议演讲实录（中英逐段对照）

【开场介绍】

中文：大家下午好。非常感谢大家参加第44届摩根大通医疗保健会议的另一场环节。我是Brian Cheng。我是公司的高级生物科技分析师之一。台上的嘉宾是亚盛医药。现在我把话筒交给首席执行官杨大钧博士，进行简短演讲，随后是现场观众问答。杨博士，欢迎。舞台交给你。
英文：Good afternoon, everyone. Thank you so much for joining us for another session at the 44th JPMorgan Healthcare Conference. I'm Brian Cheng. I'm one of the senior biotech analysts here at the firm. On stage, we have Ascentage Pharma. I'll now pass the mic to the CEO, Dr. Dajun Yang, for a short presentation, followed by a live audience Q&A. Dr. Yang, welcome. The stage is yours.

【杨大钧开场：首次主会议演讲+IPO回顾】

中文：谢谢。很荣幸首次在主会议演讲上发言。我们原本打算去年做演讲，但由于去年摩根大通会议后立即计划IPO，所以不得不取消去年的演讲。所以今天能来这里真的很荣幸。今年我们取得了很多进展。正如我所说，我们在去年摩根大通会议后完成了一次成功的IPO，而且也是由摩根大通去年牵头完成的。
英文：Thank you. Really honored to be here at the main conference presentation for the first time. And we were supposed to do our presentation last year, but due to the planned IPO immediately after the JPMorgan meeting last year, so we had to cancel our presentation last year. So it's a great honor to be here. And this year, we have made a lot of progress. And as I said, we did a great IPO last year following the JPMorgan meeting and also led by JPMorgan last year as well.

【公司概况：成立背景+核心属性】

中文：这是我们亚盛的概况。对于之前可能不了解我们的朋友，我来详细介绍一下。亚盛成立于约16年前——2009年，我们是真正的全球商业化阶段血液学/肿瘤学公司。我们有2款针对BCR-ABL和Bcl-2的新型商业化产品。我们是少数同时在纳斯达克和香港联交所双重上市的公司之一。截至最近一份财务报告，我们有4.2亿美元现金，足以支持当前研发计划至2027年。
英文：This is actually the overview of Ascentage. For some of you who may not know us before, let me go through this in a little bit more detail. Ascentage actually founded about 16 years ago, in 2009, and we are really truly global and commercial stage hematology/oncology company. We have 2 novel commercial products targeting BCR-ABL and also Bcl-2. We are one of the few dual listed on NASDAQ and the Hong Kong Stock Exchange. And up to the last financial report, we have $420 million of cash. So that will -- can support our current R&D plans through 2027.

【管线与知识产权：临床阶段+专利+学术】

中文：我们有一个非常强大的管线：7个新型临床阶段化合物，针对10多个适应症，拥有约30多项FDA批准的IND（ investigational New Drug，新药临床试验申请），还获得了FDA和EMA（欧洲药品管理局）颁发的17项孤儿药资格认定、4项快速通道资格认定（其中2项是儿科适应症）。目前我们在全球开展超过40项临床试验。我认为亚盛脱颖而出的一个原因是：我们拥有478项全球授权专利，还有500多项待审专利申请。我们定期在《JAMA Oncology》《Clinical Cancer Research》等全球同行评审期刊上发表论文。
英文：We have a very robust pipeline. We have 7 novel active clinical stage compounds targeting more than 10 indications, with about 30-plus FDA-cleared INDs, and we actually received 17 orphan drug designation by FDA and also by EMA, 4 Fast Track designation, 2 of them are pediatric. We run at the time, more than 40 clinical trials globally. I think one thing really makes Ascentage standing out is that we have global IP protection over 478 global issued patents, about 500-plus more pending applications. We publish regularly on JAMA Oncology, Clinical Cancer Research, among other peer-reviewed journals globally.

【全球布局与总部】

中文：目前我们在全球有近800名员工。总体而言，我们已完成或正在进行13项全球注册试验，包括4项FDA批准的III期注册试验。公司在开曼群岛注册，总部位于中国苏州、美国马里兰州罗克维尔及澳大利亚悉尼。这是我们苏州总部的研发大楼。
英文：Currently, we have close to 800 employees worldwide. Overall, we did or ongoing 13 global registration trials, including 4 FDA-cleared Phase III registration trials. Company is incorporated in Cayman Islands with headquarter in China, Suzhou, Rockville, Maryland and also Sydney. This is actually our headquarter in Suzhou building, the R&D center.

【两款商业化产品：奥雷巴替尼】

中文：以下是我们两款具有全球机会的新型商业化阶段产品：奥雷巴替尼（Olverembatinib）：第三代BCR-ABL抑制剂，已在中国获批用于伴或不伴突变的慢性髓性白血病慢性期（CML-CP）及对2种酪氨酸激酶抑制剂（TKI）不耐受的患者。我们正在开展2项经FDA和EMA批准的全球III期研究。
英文：So these are 2 novel commercial stage products with global opportunity. Olverembatinib is the third-generation BCR-ABL inhibitor approved in China for CML-CP with or without mutation and about 2 TKI, including the intolerant. And we are running 2 global Phase III studies cleared by FDA and EMA.

【两款商业化产品：Lisaftoclax】

中文：右侧是Lisaftoclax：新型口服选择性Bcl-2抑制剂，6个月前刚在中国获批用于BTK治疗后复发/难治的慢性淋巴细胞白血病（CLL）和小淋巴细胞淋巴瘤（SLL）。我认为就这一适应症而言，我们是全球首个在BTK治疗后用单药治疗CLL的公司——因为维奈克拉（Venetoclax）仅获批17p缺失的适应症。我们还有4项全球注册试验，其中2项经FDA和EMA批准。
英文：On the right, we have Lisaftoclax, the novel orally active selective Bcl-2 inhibitor approved just 6 months ago in China for the CLL and SLL after BTK therapy. I think for this indication, we are actually the first one globally after BTK in CLL with a single agent as Venetoclax was only 17p at deletion indications. We also have 4 global registration trials with 2 cleared by FDA and EMA.

【后期管线：First-in-class+未满足需求】

中文：我们还有4项全球注册试验，其中2项经FDA和EMA批准。这是我们真正世界级创新且高度低风险的后期管线。如您所见，前两款是商业化阶段产品，针对各类血液恶性肿瘤；我们还有3个潜在**first-in-class（同类首创）**的新型化合物，成功推进至市场——它们聚焦于FAK、MDM2-p53、Bcl-2/Bcl-xl靶点，目前全球尚无获批产品。
英文：We also have 4 global registrational trials with 2 cleared by FDA and EMA. This is our really the world-class innovative and a highly derisked late-stage pipeline. As you can see, the first 2 are commercial stage, targeting the variety of heme malignancies. We also have 3 other novel compounds potentially the first-in-class, these were successfully developed to the market. Those focus on FAK, focus on MDM2-p53, Bcl-2, Bcl-xl. There's no currently approved product yet globally.

【PRC2/EED项目+BTK降解剂最新进展】

中文：我们还有两个激动人心的项目：通过EED靶向PRC2（多梳抑制复合物2），有望同时用于肿瘤和贫血。就在上周，我们宣布BTK降解剂获FDA批准开展全球研究。
英文：I think there are also 2 exciting programs targeting the PRC2 through the EED, have potential both for oncology anemia. Just about last week, we announced clearance of BTK degrader cleared by FDA for the global study.

【奥雷巴替尼全球项目：POLARIS-2/POLARIS-1】

中文：以下是关于奥雷巴替尼（HQP1351）的更多信息：该产品自2021年起在中国获批——这就是为什么我说它“高度低风险”。我们已经治疗了数万名CML（伴或不伴突变）及Ph+急性淋巴细胞白血病（Ph+ ALL）患者。我们有3个全球项目：POLARIS-2：单药治疗CML，对照臂为博舒替尼（bosutinib），已获FDA和EMA批准；POLARIS-1：一线治疗Ph+ ALL患者——这是非常棒的结果。我们上个月在ASH（美国血液学会年会）上展示了POLARIS-1的A部分数据：3个月CMR（完全分子缓解）率比同人群中的普纳替尼（ponatinib）翻倍，还获得了CD（中国国家药监局）的突破性疗法认定。
英文：Here's a little more information about the Olverembatinib, HQP1351. As you can see, we have this product approved in China since 2021. That's why I said it's highly derisked. We already treated over tens of thousands of patients, CML with and without mutation and also Ph+ ALL. We have 3 global programs, POLARIS-2 targeting the CML with a single agent with the control arm bosutinib cleared by FDA and EMA and also POLARIS-1, first-line Ph+ for ALL patients. This is actually a great result. With the Part A of POLARIS-1 data we presented at ASH just last month, we doubled the 3-month CMR rate over like ponatinib in the same patient population. And we also received breakthrough designation by CD.

【SDH缺陷型GIST小适应症】

中文：我们还有一个针对**SDH缺陷型胃肠道间质瘤（GIST）**的小适应症——这是单药关键注册试验，该人群全球无有效疗法。我认为真正令人兴奋的是：即使在这个会议上，至少有2家同行公司也在 targeting 同样的CML患者群体，但我们的产品在业内备受关注。

英文：We also have one small indication targeting SDH-deficient GIST. This is a monotherapy for pivotal registration trial. This is a group of patients which has no effective therapy globally. I think really exciting to see is that even that this is a conference where there are at least 2 other peer companies are targeting the same like a CML patient population received great attention at Street.

【CML市场定位+Ph+ ALL潜力】

中文：目前CML市场规模超70亿或80亿美元，asciminib（阿斯利康的BCR-ABL抑制剂）的峰值销售额已从30亿美元增至40亿美元。奥雷巴替尼在T315I突变和复合突变中占据强定位，有望成为全球晚期CML的首选药物。另一个适应症是Ph+ ALL——在亚洲国家（如中国、日本）非常普遍，目前全球无有效、安全的小分子药物，而奥雷巴替尼正在开展一线Ph+ ALL的全球注册试验。
英文：So overall CML market currently is over 7billionor7 billion or 7billionor8 billion and asciminib peak sales already increased from 3billionto3 billion to 3billionto4 billion. And Olverembatinib is strongly positioned for T315 mutation and compound mutations and could be the first choice for the late-line CML globally. There's another indication is the Ph+ ALL. It's very prevalent in Asian countries as China, Japan. Currently, there's no effective, safe small molecule drug and Olverembatinib is a global registration in the first-line Ph+ for ALL.

【指南纳入+长期随访+ASH数据】

中文：我想，过去近10年的临床开发中，我们的数据已进入NCCN（美国国家综合癌症网络）指南和中国CSCO（中国临床肿瘤学会）指南。作为单药，我们展现出真正差异化的疗效特征，且在Ph+ ALL患者中显示出良好的安全性。使用我们药物时间最长的患者自2016年I期试验以来已近10年；我们对I期后CML患者进行了5年随访，对加速期（AP）患者进行了约6年随访。上个月在ASH上，我们展示了在骨髓瘤（MM）、FGFR重排、CML、VP等其他罕见病中的强活性数据。
英文：I think the data so far in the last almost 10 years development in the clinic with the 2 guidance that we entered, NCCN guidelines and also CSCO in China. I think you can see we have a really truly differentiated efficacy profile as a single agent. We show strong -- also really safety profile in Ph+ ALL patients. The longest time patients use our drug is almost 10 years since the Phase I in 2016. And we have a 5-year follow-up for the CML patients after Phase I and also about 6 years follow-up for those in the AP patient population. And we actually had several presentations at ASH last month with strong activity in other rare disease like [MM], also FGFR rearrangement, CML, VP, et cetera.

【重度预处理CML患者数据：MD Anderson主导】

中文：简言之，奥雷巴替尼展现出强活性——尤其是这张幻灯片（由MD安德森癌症中心的Kantarjian博士和Jabbour博士主导）。这是全美国患者群体：初始结果已发表在ASH和《JAMA Oncology》上。如您所见，在这些重度预处理、真正耐药的CML患者（即失败过普纳替尼、asciminib，且超30%有T315I突变）中，作为单药，我们看到了更优的疗效：- 失败普纳替尼或asciminib的患者，单药MMR（主要分子缓解）率约为40%或30%；- 甚至少数同时失败普纳替尼和asciminib的患者，仍能看到单药获益。
英文：So basically, Olverembatinib demonstrates strong activity, especially in this slide, leading by the Dr. Kantarjian and Dr. Jabbour at MD Anderson. This is actually all U.S. patient population. The initial result already published on the ASH and JAMA Oncology. As you can see in this heavily pretreated, truly failed resistant CML patients, those who failed ponatinib, who failed asciminib and also over 30% of them have [T31] mutation, okay? This is a really different patient population, as you see from some of the peers presentation at ASH or this conference. So with this heavily pretreated then almost the last line after 4, 5 line patient population, we see a greater efficacy as a single agent. You can see that in those who failed ponatinib or asciminib, will receive a single agent about 40% or 30% MMR rate. There are few patients actually fail both, ponatinib and asciminib. We still see the single-agent benefit.

【Ph+ ALL初诊数据：POLARIS-1 A部分】

中文：另一个非常激动人心的 data 是我们上个月在ASH发布的Ph+ ALL初诊患者数据（POLARIS-1全球III期试验的A部分）：该人群的64% MRD阴性完全缓解（CR）率——几乎是同人群中普纳替尼（34%）的两倍；当然，对照臂伊马替尼（imatinib）仅能达到16%-17%的CMR率。我们还证明了良好的安全性。
英文：I think another very exciting data is what we released last month at ASH that in the Ph+ ALL, the newly diagnosed patients, this is part A of our global Phase III trial, the POLARIS-1. As you can see, in this population patients, we can have 64% MRD-negative CR rate, okay? This is almost double the same patient population ponatinib did about 34%. Of course, the control arm, the imatinib only can do about 16%, 17% CMR rate. We also have demonstrated very good safety profile.

【二线CML-CP数据】

中文：另一个激动人心的疗效数据是二线治疗CML-CP患者：即使用伊马替尼或第二代TKI（达沙替尼、尼洛替尼）作为一线治疗后，立即使用奥雷巴替尼的患者——MMR率超40%；且一线使用第二代化合物的患者响应更好。
英文：Another exciting data is really on the second-line treatment. This is in the CML-CP patients, patients who use either imatinib or one of the second-generation dasatinib or nilotinib and then immediately after the first line using the Olverembatinib. You can see MMR rate is over 40%. And actually, in the patients who in the first line use the second-generation compound do receive even better response.

【FGFR重排骨髓瘤数据】

中文：另一个非常激动人心的疗效数据来自伴FGFR重排的罕见骨髓瘤患者：目前全球无有效治疗，我们努力入组了约20例患者，结果显示出强活性（包括CMR率和移植后的完全缓解）。
英文：Another very exciting efficacy data is in this rare MM patient population with FGFR rearrangement. Currently, there's really no effective treatment. And we take quite a few efforts to enroll about 20 patients. And as you can see, they have a really robust activity, including the CMR rate and also a complete response after the transplant.

【急变期CML+安全性：4年随访】

中文：另一个深度缓解数据来自急变期CML患者——因时间有限，我不展开，但需强调：奥雷巴替尼对几乎所有形式的CML都有广泛且强的活性。关于安全性：过去几年（包括我忘了提的2024年左右与武田的option协议合作——武田是全球CML和ALL领域的优秀合作伙伴），我们做了注册试验的4年随访。我想提一点：中国的CD标准非常高——该人群必须失败所有3种TKI（全球多数注册试验仅需失败2种）。这项在中国开展的研究纳入140多例患者，大多数失败过伊马替尼、达沙替尼和尼洛替尼这3种TKI。当然，这是以EFS（无事件生存期）为终点的优秀数据。4年随访的安全性数据显示：- 血液学不良事件（3级及以上）：随着治疗清除骨髓癌细胞，血小板减少、中性粒细胞减少的情况随时间减少；- 非血液学事件多为1级或2级——这是4年随访下的良好安全性。
英文：Another really deep remission is in the blast phase CML patients. I think in the interest of time, I will not go into detail, but I think it is important to note is that the Olverembatinib has really broad and strong activity against almost all forms of the CML. The important data is on the safety. I think over the last couple of years, including actually, I forgot to mention, we entered the partnership under the option agreement with Takeda in about 2024, I think. Takeda is a great partner in CML and ALL globally. I think this is actually a 4-year follow-up of the registration trial we did. One thing I want to mention in China, actually, CD have a very high bar. This patient population, they have to fail all 3 TKI, okay? Majority, almost globally, all the registration trial is after 2 TKI. And this particular study, over 140 patients, majority of them had to fail all 3 TKIs, not just imatinib or dasatinib and also nilotinib in China. Of course, this is looking for the EFS as a great data. And the 4-year follow-up, as you can see, this is the safety profile. In the hematological AE, this is all grade 3 and above. Actually, over the time, because of the benefit of treatment to clear the cancer cells in bone marrow, those with thrombocytopenia, neutropenia actually reduced. The nonhematologic event are mostly grade 1 or 2. I think this is a great safety with a 4-year follow-up.

【奥雷巴替尼+Bcl-2联合：Ph+ ALL化疗-free】

中文：另一个激动人心的显著数据是奥雷巴替尼联合Bcl-2抑制剂治疗Ph+ ALL：由于维奈克拉已上市，我们用其开展化疗-free方案，疗效显著；更重要的是，该治疗方案的最长患者随访超800天。当然，我们会继续推进奥雷巴替尼与**Lisaftoclax（我们的Bcl-2抑制剂）**的联合PI（研究者发起的试验）。
英文：Another exciting, I think, notable data is actually combination of Olverembatinib with Bcl-2 in Ph+ ALL. In this case, they use the Venetoclax because that's already approved on the market. Remarkable efficacy in the chemo-free setting. And more importantly, the longest patient follow-up in this particular treatment is over 800 days, okay? Of course, we will continue with the PI, the combination of Olverembatinib with Lisaftoclax now.

【Lisaftoclax（APG-2575）：基本信息+全球试验】

中文：以下是关于我们的APG-2575（Lisaftoclax）的更多细节：Lisaftoclax代表“life-saving [难以辨认] class”，我们简称其为Lisa。如您所见，我们首个关键II期单药研究针对BTK失败的患者群体，6个月前刚在中国获批；我们还在开展4项全球研究（尤其是GLORA和GLORA-4），均获EMA、FDA及日本等主要监管机构批准。
英文：These are more details of our APG-2575, Lisaftoclax. Lisaftoclax standing for life-saving [indiscernible] class and briefly always just called Lisa, okay? So as you can see, we have the first pivotal Phase II study with monotherapy in -- in the BTK failed patient population approved in China just about 6 months ago. And we are running 4 global studies, especially GLORA and GLORA-4, all cleared by EMA and FDA and major regulatory agency around the world, including Japan.

【Lisaftoclax的Bcl-2靶点优势与未满足需求】

中文：我想提醒大家：Bcl-2选择性抑制剂实际上针对广泛的多种血液恶性肿瘤——这里仅列出3种：CLL、急性髓性白血病（AML）和骨髓增生异常综合征（MDS）。目前全球近20年无FDA批准的MDS靶向药物，这是真正的未满足医疗需求和未被开发的市场。

英文：I want to bring your attention to that the Bcl-2 selective inhibitor actually have really broad multiple heme malignancies. This is just list of 3 here, the CLL, AML and also the MDS. I think the MDS right now, globally no targeted drug approved by FDA in the last 20 years and it's really truly unmet medical need globally and untapped market.

【Lisaftoclax的疗效与安全性优势】

中文：我们的解决方案是：Lisaftoclax展现出强疗效，首次进入中国统计指南，还获得FDA的5项孤儿药资格认定。我想重点讲疗效数据，但更重要的是：它与市场上唯一获批9年的Bcl-2抑制剂维奈克拉的差异在于安全性。在所有临床试验中（超600-700例CLL患者，近2000例患者），我们真正证明了其安全性优于维奈克拉：- 从设计之初，我们就采用患者友好的每日给药方案；- 整体肿瘤溶解综合征（TLS）发生率极低（部分研究中为0）；- 无药物相互作用（DDI）——这与维奈克拉不同（维奈克拉与伊布替尼有DDI，且最近公开数据显示某药物的DDI风险更高）。这对患者和护理人员来说非常方便。
英文：So our solution to those issues is that Lisaftoclax demonstrate great efficacy, entered the statistical guideline, the first time in China and also granted 5 ODD by FDA, okay? I think the notable efficacy data, I will present more details. But more importantly, I think, differentiate the first Bcl-2 inhibitor on the market in the last 9 years, Venetoclax is really the safety profile, okay? I think in all the clinical trials, over 600, 700 CLL patients and overall near 2,000 patients, we really demonstrate this safety profile over the current or only one on the market in U.S., Venetoclax. We designed from day 1, the patient-friendly daily dosing schedule, okay? And we received approval. Overall have a very low tumor lysis syndrome, some studies with 0. And then more importantly, there's no drug-drug interaction observed. This is really different than the Venetoclax even have a DDI with ibrutinib. Actually, if you know, they recently just released public data, [indiscernible] even have higher risk DDI over Venetoclax. So I think this is highly convenient for patients and caregivers.

【GLORA-4 MDS试验：全球唯一III期】

中文：我认为一项非常重要的研究是GLORA-4全球试验：针对一线高危MDS，获FDA和EMA批准。我们都知道，VERONA试验（维奈克拉联合阿扎胞苷治疗MDS）自去年7月报道以来结果为阴性——这对全球患者来说确实不幸。我们很幸运邀请到MD安德森的Garcia Manero博士（主要研究者）和黄晓军博士领导这项全球研究。我认为你会看到，我们的数据与VERONA试验（维奈克拉单药 vs 联合阿扎胞苷）有很大不同。
英文：I think one of the very exciting important study is this GLORA-4 global trial in the first-line high-risk MDS cleared by FDA and EMA. We all know that VERONA trial had a negative result about -- since reported last July. And of course, that's really unfortunate for patients globally. And we are very fortunate to have Dr. Garcia Manero, the leading PI from MD Anderson and also Dr. Xiaojun Huang to lead this global study. I think you will see the data actually really different than the VERONA trial in terms of Venetoclax versus the combo with AZA.

【Lisaftoclax在AML/MDS的疗效数据（美国+澳大利亚研究）】

中文：我们在美国研究中证明：Lisaftoclax在AML、MDS及维奈克拉失败的患者中具有强临床活性——尤其是MDS、CMML（慢性粒单核细胞白血病）患者，客观缓解率（ORR）可达80%，且多数是完全缓解（CR）；在暴露于AML/MDS的患者中，ORR也能达到约31%。这真正展示了其在AML和高危MDS患者中的差异化与强活性——这些数据来自美国和澳大利亚的研究。
英文：We demonstrated the data in the U.S. study actually that Lisaftoclax has strong clinical activity in AML, MDS as well as those patients who failed Venetoclax, okay? Especially in the MDS, CML, CMML patients, they -- ORR can be 80%, majority actually is a CR. In the exposed AML/MDS patients, we can also achieve about 31% ORR, okay? I think this really demonstrates truly differentiation and strong activity in AML and high-risk MDS patients. These are all U.S. and Australia studies.

【Lisaftoclax中国CLL标签：独特剂量设计】

中文：以下是我们Lisaftoclax在中国的CLL标签总结：我们是唯一采用“每日剂量递增设计”的产品，仅有3种剂量强度，5天递增至目标剂量后维持——这是我们的独特优势，对患者非常方便。作为BTK治疗后CLL的单药，这是全球首个获批的适应症。

英文：This is the summary of our label in China for the CLL, okay? As you can see, we are the first one, the only one with this daily dose ramp-up design, okay! We have only 3 dose strengths, okay, 5 days combination to the target dose and maintain, okay? I think this is the unique strength and also really convenient for patients with CLL. With a single agent after BTK in CLL, actually, this is the first indication approved globally.

【Lisaftoclax关键II期CLL数据（BTK失败患者）】

中文：我想分享一些数据：在该患者群体中，我们开展了关键II期试验——当时CD设定的门槛很高（4年前给我们的试验设计要求每个患者都必须失败BTK，而当时BTK并非一线常用）。我们花了很长时间才招募到77例患者，但这些患者确实病情严重（存在TP53突变、17p缺失、复杂染色体）。结果显示：我们实现了超62%的ORR，以及有意义的MRD阴性和无进展生存期（PFS）。目前仍在观察PFS和总生存期（OS），数据上个月在ASH上做了口头报告，安全性也很好（因时间有限，不展开ASH的细节）。
英文：I want to also share with you some of the data. Actually, in this patient population, we did a pivotal Phase II. Again, CD has a high bar. They gave us this pivotal Phase II trial design 4 years ago, but a really high bar. They want -- every single patient at that time had to fail the BTK, okay? At that time, BTK was not very commonly used in terms of first line, right? It took us a while to recruit these 77 patients. But also, you can see the data, these patients are really sick patients, right, in terms of [TP53] mutation, 17p deletion and chromosome -- complex chromosome. And then we demonstrate that we can achieve over 62% ORR and also really meaningful MRD negativity and the PFS as well. So -- and then they -- currently, they still observed in the PFS and OS. The data also had an oral presentation at ASH last month. The safety is also really good with those patients. But in the interest of time, I will not go through the details already presented at ASH.

【Bcl-2作为backbone的联合疗法潜力】

中文：更重要的是，未来Bcl-2可作为许多B细胞恶性肿瘤的backbone（核心骨架）——例如，我们可与自身小分子药物（如奥雷巴替尼、MDM2-p53抑制剂）联合治疗AML或弥漫大B细胞淋巴瘤（DLBCL）。我想指出：大多数Bcl-2抑制剂耐药源于Mcl-1过表达，而非突变——因此，任何能间接抑制Mcl-1的药物都会有协同效应。而我们是全球唯一拥有这类小分子药物的公司。

英文：But more importantly, I think, Bcl-2 is really -- can serve as a backbone across major -- many B-cell malignancies. This is also an example, we can do the combination with our own small molecule drug, such as with Olverembatinib or our MDM2-p53 inhibitors, either in AML or DLBCL, okay? I want to point it out that majority of Bcl-2 inhibitor resistance actually is due to the Mcl overexpression, not due to the mutation, right? So any drug can combine, indirectly reduce -- inhibit Mcl-1 will have a synergistic effect. And we are the only company that has those small molecule drug globally.

【其他管线项目：BTK降解剂】

中文：我们还有许多其他有趣的新型管线：这是我们的BTK降解剂（APG-3288）。我想指出，实际上我们将这个产品推进得非常快——从决定启动项目到PCC（临床前候选化合物）再到IND获批不到2年；从PCC声明到IND-enabling研究再到IND获批约8个月。

英文：We also have many other interesting novel pipeline. This is our BTK degrader, APG-3288. I want to point it out, actually, we moved this product very quickly from this decision to go to the program and from the PCC to the IND clearance in less than 2 years. And actually from the PCC declaration, the IND-enabling study to the IND clearance in about 8 months.

【其他管线项目：EED抑制剂（APG-5918）】

中文：我们还有其他项目如APG-5918——这是表观遗传治疗项目，通过EED靶向PRC2（多梳抑制复合物2）。它有多个适应症：除了淋巴瘤、骨髓瘤等血液恶性肿瘤，还包括前列腺癌等实体瘤，以及贫血。

英文：And we also have other program like APG-5918. This is the epigenetic therapeutic program targeting the PRC2 via the EED. There's multiple indications in heme malignancies besides lymphoma, myeloma and also solid tumor in prostate cancer and more also additional indication in anemia.

【EED抑制剂的临床前数据：骨髓瘤+贫血】

中文：这是我们在ASH上月展示的临床前数据：与IMiD（免疫调节剂）联合治疗多发性骨髓瘤——表观遗传靶点的好处之一是纠正失调，恢复其他重要化合物的功能（包括前列腺癌中的化合物）。这是贫血模型中PRC2/EED活性的一个例子：在中国，我们已经完成了健康志愿者的SAD（单次给药毒性）、MAD（多次给药毒性），完成了地中海贫血患者3个剂量组的研究（中国镰状细胞贫血患者较少，因此我们最初在贫血患者中看到了临床概念验证）。

英文：This is the data we presented at ASH last month, preclinical data demonstrated the combination with IMiD in the multiple myeloma. I think one of the benefit of this epigenetic target is really to correct this deregulation and restore the function of other important compound, including those in the prostate cancer. This is one example of the activity in CKD anemia model. In China, we already finished healthy volunteer SAD, MAD, completed about the 3-dose cohort in patients with thalassemia. China doesn't have much sickle cell anemia patients. So that's where we see initially clinical proof of concept in the anemia patients.

【其他管线项目：三激酶抑制剂（APG-2449）】

中文：最后一个项目是APG-2449——这是一款三激酶抑制剂，同时靶向FAK、ALK和ROS1。针对ALK、ROS1，我们实际上已获中国III期注册试验批准；但我认为更令人兴奋的可能是FAK。在非小细胞肺癌中，这种三激酶抑制也可能带来益处——因为FAK过表达与ALK抑制剂耐药相关。

英文：The last one is the APG-2449. This is a triple kinase inhibitor targeting both FAK, ALK and ROS1. For the ALK, ROS1, we actually received clearance for Phase III registration trial in China. But I think more exciting is maybe on the FAK as well. But in terms of non-small cell lung cancer, this particular triple kinase inhibition may also offer the benefit because FAK overexpression is related to the ALK inhibitor resistance.

【其他管线项目：MDM2-p53抑制剂】

中文：最后一个我们长期投入的项目是MDM2-p53——这是一个艰难的靶点，但我们持续努力了10年，包括多项II期研究。目前我们正在寻找注册试验的路径，针对ACC（肾上腺皮质癌）或某些儿童肉瘤患者的真正临床未满足需求。

英文：And the last one is a tough target, but we have continued our effort is the MDM2-p53. There are many companies working on that, and we have been also working on that for the last 10 years, including multiple Phase II studies. And currently, we are looking for the path for the registration trial with some really truly clinical unmet medical need in the ACC or some of the pediatric sarcoma patients.

【其他管线项目：双Bcl-2/xL抑制剂】

中文：最后一个是双Bcl-2/xL抑制剂——开发耗时较长。我认为同行药物navitoclax因血小板毒性停止了所有研究，我们通过前药在临床前解决了这个问题，但仍需更多研究来确定NDA的监管路径。因时间有限，我不会深入细节。

英文：But in terms of non-small cell lung cancer, this particular triple kinase inhibition may also offer the benefit because FAK overexpression is related to the ALK inhibitor resistance. And the last one is a tough target, but we have continued our effort is the MDM2-p53. There are many companies working on that, and we have been also working on that for the last 10 years, including multiple Phase II studies. And currently, we are looking for the path for the registration trial with some really truly clinical unmet medical need in the ACC or some of the pediatric sarcoma patients. So the last one is we also work a long time is the dual Bcl-2, xL inhibitor. This takes a while to develop in the clinic. I think the other peer drug, navitoclax actually stopped all the studies due to some of the target toxicity in the platelets. We solved this problem preclinically by making the prodrug, but still need more study in terms of regulatory path to the NDA. So in the interest of time, I will not go to that much detail.

【2025年里程碑总结】

中文：我认为最后，总结我们去年的成就：2025年对亚盛来说是极其重要的一年。我们实现了第一个目标——在纳斯达克上市；还获得了Bcl-2选择性抑制剂Lisaftoclax的NDA批准；此外，我们从FDA和EMA获得了2项重要批准，因为这两项试验针对的是一线患者。对于MDS，我们是全球唯一开展MDS患者III期研究的公司，而且我们的GLORA-4入组进展非常快。再次强调，POLARIS-1是针对一线Ph+ ALL患者的试验。当然，我们尽一切努力推进所有这些POLARIS和GLORA注册试验的入组。

英文：I think finally, this is a very exciting time to summarize our achievement last year. I think 2025 has been tremendous important year for Ascentage. We achieved the first goal being listed on NASDAQ and also get NDA approval for the Bcl-2 selective inhibitor, Lisaftoclax and also received 2 important clearance from FDA and EMA because these two trials are the first-line patients. And for MDS, we are globally the only Phase III studies for the MDS patients, and we are really moving fast on the GLORA-4 enrollment. And POLARIS-1 again, is Ph+ for ALL patients for the first line. And of course, we do everything we can to advance enrollment all these POLARIS and the GLORA registration trial studies.

【团队致谢+2026年战略】

中文：感谢我的团队——我们幕后努力工作，我今天展示的所有重大成就都来自团队的努力。展望2026年，我认为首要且最重要的是聚焦执行：完成所有注册研究的入组；当然，我们也会持续增长，在商业化努力中触达更多患者。我们将为Lisaftoclax争取NRDL（国家医保目录）覆盖，推进BTK蛋白降解剂和EED抑制剂在肿瘤及贫血领域的研究（覆盖美国和中国）。我认为今年亚盛团队会带来很多惊喜。

英文：I think thanks to my team, we work very hard behind the scene. As I'm presenting here, all the major achievements come from our team's effort. Looking forward to the 2026, I think the first and most important one is focus on execution, complete the enrollment for all the registration studies. And also, of course, we will have a continued growth and reach out to more patients in our commercialization effort. And we're looking for the NRDL coverage for Lisaftoclax and advance our BTK protein degrader and also the EED inhibitor in oncology and also anemia, both U.S. and China. I think there's a lot of excitement to come from Ascentage team this year.

【核心定位：7个卓越小分子+超级后期】

中文：最后，我想展示这张幻灯片：从第一天起，我们就聚焦患者、聚焦全球市场。现在你可以看到，我们有一个“7 magnificent（卓越）小分子药物”的组合，其中2个已进入后期。有位分析师曾说我们应被称为“super late stage（超级后期）”公司——我们有2个超级后期全球机会产品。事实上，通过BTK降解剂，我们将靶向血液恶性肿瘤的3大谱系：CML、ALL、CLL、AML、MDS、多发性骨髓瘤、DLBCL，以及贫血。更重要的是，我们可能是唯一拥有化疗-free口服活性小分子药物并可开展联合疗法的公司——我们的Bcl-2抑制剂、奥雷巴替尼、BTK降解剂，加上EED抑制剂、MDM2-p53抑制剂，我们可能是唯一能提供新型单药及联合疗法帮助全球患者的公司。最后，感谢大家出席，我很乐意回答问题。谢谢。
英文：And finally, I think I want to enter this slide that from day 1, we focus on the patient, focus on the global market. Right now, actually, you can see there's one missing we have 7, what we call 7 magnificent small molecule drug with 2 already late stage. One day, one of the analysts told me we should refer us as a super late stage. So we have 2 super late-stage global opportunity products. And actually, with the BTK degrader, we're going to target all major 3 lineages of heme malignancies from the CML, ALL, CLL, AML, MDS, multiple myeloma, DLBCL and also anemia. I think more importantly, we are probably the only company have this chemo-free oral active small molecule drug can do the combination, right? So the combination of our Bcl-2, Olverembatinib, BTK degrader plus other one like EED inhibitor and the MDM2-p53, we are probably the only company who will be able to have this novel single-agent compound and also combination to help the patients globally. Finally, I thank you all for attending, and I'd be happy to answer the questions. Thank you.