Revumenib

本文梳理了近一个月（2024年10月16日～2024年11月15日）在研药物的审批情况，同时，对部分药物的研究情况进行简述和分析，供研发人员参考。 据统计，在2024年10月16日～2024年11月15日期间，共有5个药物获批（表1）。这些药物涉及的适应症包括白血病、淋巴瘤、非小细胞肺癌、泌尿系统感染、慢性乙肝。以下对重点药物进行介绍和分析。 表1：近一个月（2024年10月16日～2024年11月15日）的药物批准情况 01 Revumenib 2024年11月15日，美国食品药品监督管理局（FDA）批准Revumenib（商品名：Revuforj，图1），用于治疗携带赖氨酸甲基转移酶2A（KMT2A）易位的复发或难治性急性白血病成人患者和1岁及以上的儿童患者。 Revumenib是由美国Syndax Pharmaceuticals生产的首款获批Menin抑制剂。Revumenib是一种针对Menin-KMT2A相互作用的强效、选择性小分子抑制剂，正在开发用于治疗KMT2A重排急性白血病和NPM1突变型（mNPM1）急性髓系白血病（AML）。Revumenib用于治疗KMT2A重排急性白血病的新药申请曾获FDA的优先审评资格以及突破性疗法认定。 KMT2A是一种在人类中编码蛋白质的基因，位于第11号染色体上，主要涉及基因表达的调控，其突变与多种疾病相关。Menin抑制剂是一种能够破坏Menin-KMT2A蛋白与蛋白相互作用的小分子药物，主要用于治疗由KMT2A重排或NPM1突变引起的急性白血病。 图1：Revumenib的化学结构式 KMT2A基因重排（KMT2Ar）可能会导致侵袭性急性白血病，其预后极差且复发率高。据估计，超过95%的KMT2Ar急性白血病患者携带KMT2A易位，这是一种染色体的一部分断裂后与另一染色体融合的重排类型。超过一半的KMT2Ar急性白血病患者在接受传统一线治疗后会复发，其中位总生存期（OS）不到一年。在接受三线或后续治疗时，仅5%的患者可达到完全缓解，其中位OS不足三个月。 Revuforj的批准主要基于FDA对104名携带KMT2A易位的复发/难治性（R/R）急性白血病患者在Ⅰ/Ⅱ期AUGMENT-101试验中接受Revuforj治疗的分析。在疗效评估人群中，完全缓解（CR）加部分血液学恢复的完全缓解（CRh）的比例为21%（22/104名患者；95%CI：13.8-30.3）。CR/CRh患者的中位缓解持续时间为6.4个月（95%CI：2.7-无法估计），达到CR或CRh的中位时间为1.9个月（范围：0.9至5.6个月）。23%（24/104名患者）在接受Revuforj治疗后接受了造血干细胞移植（HSCT）。这104名患者疗效分析的结果与此前在Journal of Clinical Oncology上发表的针对R/R KMT2Ar急性白血病患者（n=57）的Ⅱ期中期分析结果一致。 Revuforj的安全性评估基于FDA对135名携带KMT2A易位的R/R急性白血病患者的数据分析。最常见的不良反应（≥20%）包括实验室检测异常，包括出血、恶心、磷酸盐升高、肌肉骨骼疼痛、感染、天冬氨酸氨基转移酶升高、发热性中性粒细胞减少症、丙氨酸氨基转移酶升高、甲状旁腺激素完整升高、细菌感染、腹泻、分化综合征、心电图QT间期延长、磷酸盐降低、甘油三酯升高、钾降低、食欲下降、便秘、水肿、病毒感染、疲劳和碱性磷酸酶升高。导致剂量减少或永久停药的不良反应分别较低，为10%和12%的患者。 02 欧贝妥基奥仑赛 2024年11月8日，Autolus Therapeutics公司的CAR-T细胞疗法欧贝妥基奥仑赛（商品名：Aucatzyl，通用名为Obecabtagene autoleucel或Obe-cel）已获得FDA批准，用于治疗R/R B细胞前体急性淋巴细胞白血病（B-ALL）成人患者。Aucatzyl是获FDA批准的首个无需风险评估缓解策略计划（REMS）的CAR-T疗法，同时也是FDA批准的第五款CD19 CAR-T疗法。 此次FDA的批准主要是基于Aucatzyl在R/R B-ALL成人患者中所进行的FELIX临床试验的结果。在94名至少接受过一次Aucatzyl输注的患者中，其中65名患者符合疗效可评估的标准。其中，63%的患者获得了总完全缓解（OCR），其中包括51%的患者在任何时间达成CR，12%患者在任何时间达成CR伴不完全的血液学恢复（CRi）。此外，主要疗效终点是在3个月内达成完全缓解，共有42%的患者达成，中位缓解持续时间（DOR）为14.1个月。 安全性方面，Aucatzyl表现出低水平的细胞因子释放综合征（CRS），3级事件发生率为3%，无4级或5级事件。7%的患者出现≥3级免疫效应细胞相关神经毒性综合征（ICANS）。 Aucatzyl是一款靶向CD19抗原的自体CAR-T细胞疗法。它旨在克服当前CAR-T细胞疗法在临床活性和安全性方面的局限性。它的独特之处在于，设计的嵌合抗原受体在与CD19结合后具有较快的解离速度（Fast off-rate），从而能够尽量减少T细胞的过度激活，在降低毒副作用的同时，不易发生T细胞耗竭，提高CAR-T疗法的持久性。此前，该疗法已被FDA和欧洲药品管理局（EMA）授予孤儿药资格，还获得FDA授予的再生医学先进疗法（RMAT）认定以及EMA授予治疗成人R/R B细胞急性淋巴细胞性白血病的PRIME认定。 03 硫培南/丙磺舒 2024年10月25日，Iterum Therapeutics宣布，FDA已批准Iterum的新药申请，批准Orlynvah（硫培南酯/丙磺舒）用于治疗由指定微生物大肠杆菌、肺炎克雷伯菌或变形杆菌引起的女性成人无并发症的尿路感染（uUTIs），这些患者的口服抗菌治疗选择有限或无可选方案。这是Orlynvah首次获得批准的适应症，也是Iterum首个获得FDA批准的产品。 FDA批准Orlynvah是基于一个临床开发计划，该计划得到了强有力的数据支持，包括两个关键的Ⅲ期临床试验（称为SURE 1和REASSURE），这些试验评估了Orlynvah与环丙沙星（SURE 1）和阿莫西林/克拉维酸（Augmentin，REASSURE）在治疗成人女性uUTI方面的安全性和有效性。SURE 1显示在氟喹诺酮耐药感染中，Orlynvah优于环丙沙星，而REASSURE则在对阿莫西林/克拉维酸敏感的人群中显示出非劣效和统计学上的优越性。Orlynvah在SURE 1和REASSURE临床试验中总体耐受性良好。 硫培南（Sulopenem etzadroxil，研发代码：PF-03709270，图2）是一种有口服活性的酯类活性分子，对大多数革兰氏阳性菌和革兰氏阴性菌具有广泛的抗菌活性。硫培南的化学结构具有青霉素、头孢菌素和碳青霉烯类药物的特性。硫培南以口服前药形式存在，该前药通过肠道酯酶水解，形成活性硫培南。 图2：硫培南的化学结构式 丙磺舒（Probenecid，图3）主要用在抗痛风药，通过抑制尿酸盐在近曲肾小管的主动再吸收，增加尿酸盐的排泄而降低血中尿酸盐的浓度，可缓解或防止尿酸盐结节的生成，减少关节的损伤，亦可促进已形成的尿酸盐溶解，无抗炎、镇痛作用。丙磺舒可以竞争性抑制弱有机酸（如青霉素、头孢菌素）在肾小管的分泌，故可以增加这些抗生素的血药浓度和延长它们的作用时间。 图3：丙磺舒的化学结构式 【参考资料】 1. Informa数据库, 检索日期: 2024年11月19日. 2. 药渡数据库, 检索日期: 2024年11月30日. 3. 药明康德、医药魔方、学术经纬、医药观澜、医麦客等网络公开资源. 本文其它内容请见《全球药物创新快讯》2024年第10期（总第146期）。

– Subgroup analyses from Ph 2 protocol-defined R/R mNPM1 AML efficacy population (N=64) show responses across all major subgroups, including heavily pretreated patients – – 26% CR+CRh (20/77) and 48% ORR (37/77) in all enrolled patients who met the efficacy evaluable criteria in Ph 2 R/R mNPM1 AML cohort – – 100% ORR (37/37) and 95% CRc (35/37) in BEAT AML trial exploring revumenib in combination with venetoclax/azacitidine in newly diagnosed mNPM1 or KMT2Ar AML – – BEAT AML data highlight the potential for revumenib to advance the current standard of care – WALTHAM, Mass., Dec. 9, 2024 /PRNewswire/ -- Syndax Pharmaceuticals (Nasdaq: SNDX), a commercial-stage biopharmaceutical company developing an innovative pipeline of cancer therapies, today announced additional positive data from the AUGMENT-101 trial of Revuforj® (revumenib) in relapsed or refractory (R/R) mutant NPM1 (mNPM1) acute myeloid leukemia (AML) and the BEAT AML trial of revumenib in combination with venetoclax and azacitidine in newly diagnosed AML patients. Revuforj is the Company's oral, first-in-class menin inhibitor that is FDA approved for the treatment of relapsed or refractory (R/R) acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients one year and older. "These new data continue to highlight the exciting potential for Revuforj as both a monotherapy and in combination with other therapies," said Michael A. Metzger, Chief Executive Officer of Syndax. "The recent approval of Revuforj for R/R acute leukemia with a KMT2A translocation, coupled with the consistency of the results we have reported across KMT2Ar and mNPM1 within the different trials and populations, continues to bolster our confidence in its practice-changing and blockbuster potential." Additional Results from R/R mNPM1 AML Patients in Pivotal Phase 2 Portion of AUGMENT-101 Syndax recently announced that the primary endpoint was met with a complete remission (CR) plus CR with partial hematological recovery (CRh) rate of 23% (15/64; 95% confidence interval [CI]: 14%, 36%; one-sided p-value =0.0014) in the protocol-defined efficacy population of 64 adults with R/R mNPM1 AML in the Phase 2 cohort of the AUGMENT-101 trial of revumenib (DCO: September 2024). The median duration of CR/CRh responses was 4.7 months at the time of the data cutoff with three patients remaining in response. Minimal residual disease (MRD) status was assessed in 14 of 15 patients who achieved CR/CRh, 64% (9/14) of whom were MRD negative. The overall response rate (ORR)1 was 47% (30/64). The safety profile observed with revumenib in the 84 patients enrolled in the cohort was consistent with previously reported data. Syndax announced today additional results from the Phase 2 cohort of R/R mNPM1 AML patients in the AUGMENT-101 trial, including data generated from the protocol-defined efficacy population of 64 adults and a post-hoc efficacy analysis based on all patients who met the efficacy evaluable criteria. Subgroup analyses from the Phase 2 protocol-defined R/R mNPM1 efficacy population (N=64) show that CR/CRh responses were observed across all major subgroups, including patients with multiple prior lines of therapy and prior venetoclax exposure, although the trial was not powered to evaluate differences among subgroups. The CR+CRh rate was 25% (4/16) among patients with 1 prior line of therapy, 20% (5/25) among patients with 2 prior lines of therapy, and 26% (6/23) among patients who had received three or more prior lines of therapy. The CR+CRh rate was 44% (7/16) among patients without prior venetoclax exposure and 17% (8/48) among patients with prior venetoclax exposure. Historically, AML patients who have failed prior treatment with venetoclax are unlikely to respond to subsequent therapy, with a CR rate of 6% reported for other targeted therapies after prior venetoclax therapy.2 Syndax also shared results from an expanded analysis of the R/R mNPM1 AML patients who enrolled into the Phase 2 cohort of AUGMENT-101. Among the 84 patients enrolled in the cohort, 77 met the efficacy evaluable criteria requiring patients to have blast counts >5% measured within 28 days prior to treatment and a centrally confirmed NPM1 mutation. In this expanded post-hoc efficacy analysis, 48% (37/77; 95% CI: 37%, 60%) achieved an overall response, and 26% (20/77; 95% CI: 17%, 37%) achieved a CR/CRh. The median duration of CR/CRh response was 4.7 months as of the September 2024 DCO. Minimal residual disease (MRD) status was assessed in 19 of 20 patients who achieved CR/CRh, 63% (12/19) of whom were MRD negative. Updated Data from BEAT-AML Trial of Revumenib in Combination with Venetoclax and Azacitidine in Newly Diagnosed AML Patients Today the company announced an update from the Phase 1 BEAT-AML trial evaluating the combination of revumenib with venetoclax and azacitidine in newly diagnosed mNPM1 or KMT2A-rearranged (KMT2Ar) AML patients aged 60 years or older. The trial is being conducted as part of the Leukemia & Lymphoma Society's Beat AML® Master Clinical Trial. Today's update builds on the BEAT AML data that was presented in June at the European Hematology Association (EHA) 2024 Congress from 24 efficacy evaluable patients showing a composite complete remission (CRc) rate of 96% (23/24) as of a May 2024 data cutoff. As of a November 2024 data cutoff, 46 newly diagnosed mNPM1 (n=37) or KMT2Ar (n=9) patients have been enrolled in BEAT AML across two dose levels of revumenib (113 mg q12 or 163 mg q12h with azoles) in combination with venetoclax and azacitidine. The median age of patients enrolled was 71 years (range: 60-92). The efficacy evaluable population includes 37 patients across both dose levels with an ORR1 of 100% (37/37) and CRc rate of 95% (35/37). The rate of MRD negativity was 95% (35/37). 27% (10/37) of patients proceeded to hematopoietic stem cell transplant (HSCT). Revumenib was generally well tolerated at both the 113 mg and 163 mg q12h dose in combination with venetoclax and azacitidine. In the safety population (N=46), 15% (7/46) of patients experienced differentiation syndrome with two (4%) Grade 3 or greater events. 43% (20/46) of patients experienced QTc prolongation with five (11%) Grade 3 or greater events. DS and QTc prolongations were self-limiting and did not cause any discontinuations. Analysis of the onset and extent of hematologic toxicities suggest a similar experience to what has been reported for the venetoclax/azacitidine doublet alone. Overall, there were no new or increased safety signals observed when revumenib was included in this triplet combination. "These are very exciting data that highlight the potential for revumenib to enhance the responses typically observed with venetoclax/azacitidine in newly diagnosed patients with mNPM1 or KMT2Ar who are unfit to receive intensive chemotherapy," said Joshua F. Zeidner, M.D., Chief, Leukemia Research at the University of North Carolina, Lineberger Comprehensive Cancer Center. "These new data continue to show that revumenib has a safety profile that could enable it to be combined with venetoclax/azacitidine and, importantly, we are observing high rates of response and MRD negativity that underscore the potential for revumenib to become an integral component of frontline treatment for KMT2Ar and mNPM1 AML patients." Enrollment in the expansion cohort is ongoing at both dose levels. The Company plans to initiate a pivotal trial with this combination in front-line newly diagnosed patients by year-end 2024. Syndax Corporate Event The new data described above, along with other data presented through today at the 66th ASH Annual Meeting being held in San Diego, CA for both the Revuforj (revumenib) and Niktimvo (axatilimab-csfr) clinical programs, will be highlighted at the Company's investor event on Monday, December 9, 2024 at 7:00 a.m. PT/10:00 a.m. ET. The live audio webcast and accompanying slides for the event may be accessed through the Events & Presentations page in the Investors section of the Company's website or directly through the meeting link here. For those unable to participate in the conference call or webcast for the event, a replay will be available on the Investors section of the Company's website at for a limited time. About Revuforj® (revumenib) Revuforj (revumenib) is an oral, first-in-class menin inhibitor that is FDA approved for the treatment of relapsed or refractory (R/R) acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients one year and older. Revumenib is in development for the treatment of R/R acute myeloid leukemia (AML) with a nucleophosmin 1 mutation (mNPM1). Positive pivotal data from the AUGMENT-101 trial in this population with revumenib as a monotherapy were recently reported. The Company expects to file a supplemental NDA filing for revumenib in R/R mNPM1 AML in the first half of 2025. Additionally, multiple trials of revumenib in combination with standard-of-care agents in mNPM1 AML or KMT2A-rearranged acute leukemia are ongoing across the treatment landscape, including in newly diagnosed patients. Revumenib was previously granted Orphan Drug Designation for the treatment of AML, ALL and acute leukemias of ambiguous lineage (ALAL) by the U.S. FDA and for the treatment of AML by the European Commission. The U.S. FDA also granted Fast Track designation to revumenib for the treatment of adult and pediatric patients with R/R acute leukemias harboring a KMT2A rearrangement or NPM1 mutation and Breakthrough Therapy Designation for the treatment of adult and pediatric patients with R/R acute leukemia harboring a KMT2A rearrangement. IMPORTANT SAFETY INFORMATION WARNING: DIFFERENTIATION SYNDROME Differentiation syndrome, which can be fatal, has occurred with Revuforj. Signs and symptoms may include fever, dyspnea, hypoxia, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain or peripheral edema, hypotension, and renal dysfunction. If differentiation syndrome is suspected, immediately initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution. WARNINGS AND PRECAUTIONS Differentiation syndrome: Revuforj can cause fatal or life-threatening differentiation syndrome (DS). Symptoms of DS, including those seen in patients treated with Revuforj, include fever, dyspnea, hypoxia, peripheral edema, pleuropericardial effusion, acute renal failure, and/or hypotension. In clinical trials, DS occurred in 39 (29%) of 135 patients treated with Revuforj. DS was Grade 3 or 4 in 13% of patients and fatal in one. The median time to onset was 10 days (range 3-41 days). Some patients experienced more than 1 DS event. Treatment interruption was required for 7% of patients, and treatment was withdrawn for 1%. Reduce the white blood cell count to less than 25 Gi/L prior to starting Revuforj. If DS is suspected, immediately initiate treatment with systemic corticosteroids (e.g., dexamethasone 10-mg IV every 12 hours in adults or dexamethasone 0.25-mg/kg/dose IV every 12 hours in pediatric patients weighing less than 40 kg) for a minimum of 3 days and until resolution of signs and symptoms. Institute supportive measures and hemodynamic monitoring until improvement. Interrupt Revuforj if severe signs and/or symptoms persist for more than 48 hours after initiation of systemic corticosteroids, or earlier if life-threatening symptoms occur such as pulmonary symptoms requiring ventilator support. Restart steroids promptly if DS recurs after tapering corticosteroids. QTc interval prolongation: In the clinical trials, QTc interval prolongation was reported as an adverse reaction in 39 (29%) of 135 patients treated with Revuforj. QTc interval prolongation was Grade 3 in 12% of patients. The heart-rate corrected QT interval (using Fridericia's method) (QTcF) was greater than 500 msec in 8%, and the increase from baseline QTcF was greater than 60 msec in 18%. Revuforj dose reduction was required for 5% of patients due to QTc interval prolongation. QTc prolongation occurred in 16% of the 31 patients less than 17 years old, 33% of the 88 patients 17 years to less than 65 years old, and in 50% of the 16 patients 65 years or older. Correct electrolyte abnormalities, including hypokalemia and hypomagnesemia, prior to treatment with Revuforj. Perform an electrocardiogram (ECG) prior to initiation of Revuforj, and do not initiate Revuforj in patients with QTcF >450 msec. Perform an ECG at least once weekly for the first 4 weeks and at least monthly thereafter. In patients with congenital long QTc syndrome, congestive heart failure, electrolyte abnormalities, or those who are taking medications known to prolong the QTc interval, more frequent ECG monitoring may be necessary. Concomitant use with drugs known to prolong the QTc interval may increase the risk of QTc interval prolongation. Interrupt Revuforj if QTcF increases >480 msec and <500 msec, and restart Revuforj at the same dose twice daily after the QTcF interval returns to ≤480 msec Interrupt Revuforj if QTcF increases >500 msec or by >60 msec from baseline, and restart Revuforj twice daily at the lower-dose level after the QTcF interval returns to ≤480 msec Permanently discontinue Revuforj in patients with ventricular arrhythmias and in those who develop QTc interval prolongation with signs or symptoms of life-threatening arrhythmia. Embryo-fetal toxicity: Revuforj can cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential and males with female partners of reproductive potential to use effective contraception during treatment with Revuforj and for 4 months after the last dose of Revuforj. ADVERSE REACTIONS Fatal adverse reactions occurred in 4 (3%) patients who received Revuforj, including 2 with differentiation syndrome, 1 with hemorrhage, and 1 with sudden death. Serious adverse reactions were reported in 99 (73%) patients. The most frequent serious adverse reactions (≥5%) were infection (24%), febrile neutropenia (19%), bacterial infection (17%), differentiation syndrome (12%), hemorrhage (9%), and thrombosis (5%). The most common adverse reactions (≥20%) including laboratory abnormalities, were hemorrhage (53%), nausea (51%), phosphate increased (50%), musculoskeletal pain (42%), infection (41%), aspartate aminotransferase increased (37%), febrile neutropenia (35%), alanine aminotransferase increased (33%), parathyroid hormone intact increased (33%), bacterial infection (31%), diarrhea (30%), differentiation syndrome (29%), electrocardiogram QT prolonged (29%), phosphate decreased (25%), triglycerides increased (25%), potassium decreased (24%), decreased appetite (24%), constipation (23%), edema (23%), viral infection (23%), fatigue (22%), and alkaline phosphatase increased (21%). DRUG INTERACTIONS Drug interactions can occur when Revuforj is concomitantly used with: Strong CYP3A4 inhibitors: reduce Revuforj dose Strong or moderate CYP3A4 inducers: avoid concomitant use with Revuforj QTc-prolonging drugs: avoid concomitant use with Revuforj. If concomitant use is unavoidable, obtain ECGs when initiating, during concomitant use, and as clinically indicated. Withhold Revuforj if the QTc interval is >480 msec. Restart Revuforj after the QTc interval returns to ≤480 msec. SPECIFIC POPULATIONS Lactation: advise lactating women not to breastfeed during treatment with Revuforj and for 1 week after the last dose. Pregnancy and testing: Revuforj can cause fetal harm when administered to a pregnant woman. Verify pregnancy status in females of reproductive potential within 7 days prior to initiating Revuforj. Pediatric: monitor bone growth and development in pediatric patients. Geriatric: compared to younger patients, the incidences of QTc prolongation and edema were higher in patients 65 years and older. Infertility: based on findings in animals, Revuforj may impair fertility. The effects on fertility were reversible. To report SUSPECTED ADVERSE REACTIONS, contact Syndax Pharmaceuticals at 1-888-539-3REV or FDA at 1-800-FDA-1088 or . Please see Full Prescribing Information , including BOXED WARNING. About Syndax Syndax Pharmaceuticals is a commercial-stage biopharmaceutical company developing an innovative pipeline of cancer therapies. Highlights of the Company's pipeline include Revuforj® (revumenib), an FDA-approved menin inhibitor, and Niktimvo™ (axatilimab-csfr), an FDA-approved monoclonal antibody that blocks the colony stimulating factor 1 (CSF-1) receptor. Fueled by our commitment to reimagining cancer care, Syndax is working to unlock the full potential of its pipeline and is conducting several clinical trials across the continuum of treatment. For more information, please visit or follow the Company on X (formerly Twitter) and LinkedIn. Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "anticipate," "believe," "could," "estimate," "expects," "intend," "may," "plan," "potential," "predict," "project," "should," "will," "would" or the negative or plural of those terms, and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Syndax's expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties. Actual results may differ materially from these forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to, statements about the progress, timing, clinical development and scope of clinical trials, the reporting of clinical data for Syndax's product candidates, the acceptance of Syndax and its partners' products in the marketplace, sales, marketing, manufacturing and distribution requirements, and the potential use of its product candidates to treat various cancer indications and fibrotic diseases. Many factors may cause differences between current expectations and actual results, including: unexpected safety or efficacy data observed during preclinical or clinical trials; clinical trial site activation or enrollment rates that are lower than expected; changes to Revuforj's commercial availability, changes in expected or existing competition; changes in the regulatory environment; failure of Syndax's collaborators to support or advance collaborations or product candidates; and unexpected litigation or other disputes. Other factors that may cause Syndax's actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Syndax's filings with the U.S. Securities and Exchange Commission, including the "Risk Factors" sections contained therein. Except as required by law, Syndax assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available. References 1. Overall response rate (ORR) includes CR, CRh, CRp, CRi, MLFS, and PR; Composite complete remission (CRc) includes CR, CRh, CRp, and CRi. CR = Complete remission CRh = Complete remission with partial hematologic recovery CRp = Complete remission with incomplete platelet recovery CRi = Complete remission with incomplete count recovery MLFS = Morphologic leukemia-free state PR = Partial response 2. Bewersdorf JP, Shallis RM, Derkach A, et al. Efficacy of FLT3 and IDH1/2 inhibitors in patients with acute myeloid leukemia previously treated with venetoclax. Leuk Res. 2022;122:106942. doi:10.1016/j.leukres.2022.106942 Contact Sharon Klahre Syndax Pharmaceuticals, Inc. [email protected] Tel 781.684.9827 SNDX-G SOURCE Syndax Pharmaceuticals, Inc. 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