Decitabine

HONG KONG, Sept. 15, 2025 /PRNewswire/ -- Akeso Inc. (9926.HK) today announced that its proprietary next-generation humanized IgG4 monoclonal antibody targeting CD47, ligufalimab (AK117), has been granted Orphan Drug Designation (ODD) by the U.S. FDA for the treatment of acute myeloid leukemia (AML). The Orphan Drug Designation is a program established by the FDA to incentivize the development of therapies for rare diseases. Drugs with this designation benefit from comprehensive FDA guidance during development, tax incentives, and up to seven years of market exclusivity upon approval. Akeso is actively advancing the international clinical development for ligufalimab, which is being evaluated in both hematologic malignancies and solid tumors. In addition to its application in AML, patient enrollment has been completed in a randomized, double-blind, multicenter Phase II study assessing ligufalimab combined with azacitidine in higher-risk myelodysplastic syndromes (HR-MDS). Ligufalimab is also the first CD47 monoclonal antibody to enter registrational Phase III trials in solid tumors. Two Phase III studies are currently ongoing: one evaluating the combination of ligufalimab and ivonescimab as first-line treatment for PD-L1-positive head and neck squamous cell carcinoma (HNSCC), and another study assessing this combination as first-line therapy for pancreatic cancer. Acute Myeloid Leukemia (AML) is a heterogeneous hematologic malignancy characterized by the clonal proliferation of myeloid blasts in the bone marrow, peripheral blood, and extramedullary tissues. It is the most common type of acute leukemia in adults. Treatment strategies for AML, as outlined in the NCCN Guidelines®, are primarily based on whether patients are eligible for intensive induction chemotherapy. For those ineligible for such chemotherapy, treatment options remain limited. The FDA has currently approved venetoclax in combination with azacitidine, decitabine, or low-dose cytarabine for newly diagnosed AML patients aged 75 years or older, or for those with comorbidities that preclude intensive chemotherapy. However, more than half of these patients relapse within 6–9 months, with a median overall survival of approximately one year, highlighting a significant unmet clinical need. Ligufalimab is a humanized IgG4 monoclonal antibody that binds specifically to CD47 expressed on tumor cells, blocking its interaction with the SIRPα receptor. This disrupts the "don't eat me" signal, thereby enhancing macrophage-mediated phagocytosis of tumor cells and inhibiting tumor growth. Ligufalimab's unique design prevents red blood cell agglutination and demonstrates significantly improved safety and efficacy compared to other CD47-targeting agents. Preclinical studies have shown that ligufalimab, when combined with azacitidine or venetoclax, synergistically enhances the expression of "eat me" signals (such as calreticulin), leading to more efficient activation of phagocytic immune responses. This combination may thus offer a promising treatment option for AML patients ineligible for standard induction chemotherapy. Clinical trials have demonstrated that ligufalimab combined with azacitidine shows a favorable safety profile and promising efficacy in first-line AML treatment. Even at high doses (up to 45 mg/kg, administered biweekly), ligufalimab was well tolerated, with no significant safety differences observed across patient groups. The complete remission (CR) rate at the target dose reached 50%, and the composite complete remission (cCR) rate was 55%. Building on these encouraging results, Akeso has launched a Phase II study to further investigate the safety and efficacy of ligufalimab in combination with venetoclax and azacitidine for first-line AML patients ineligible for intensive chemotherapy. Forward-Looking Statement of Akeso, Inc. This announcement by Akeso, Inc. (9926.HK, "Akeso") contains "forward-looking statements". These statements reflect the current beliefs and expectations of Akeso's management and are subject to significant risks and uncertainties. These statements are not intended to form the basis of any investment decision or any decision to purchase securities of Akeso. There can be no assurance that the drug candidate(s) indicated in this announcement or Akeso's other pipeline candidates will obtain the required regulatory approvals or achieve commercial success. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements. Risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and health care legislation in P.R.China, the United States and internationally; global trends toward health care cost containment; technological advances, new products and patents attained by competitors; challenges inherent in new product development, including obtaining regulatory approval; Akeso's ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of the Akeso's patents and other protections for innovative products; and the exposure to litigation, including patent litigation, and/or regulatory actions. Akeso does not undertake any obligation to publicly revise these forward-looking statements to reflect events or circumstances after the date hereof, except as required by law. About Akeso Akeso (HKEX: 9926.HK) is a leading biopharmaceutical company committed to the research, development, manufacturing and commercialization of the world's first or best-in-class innovative biological medicines. Founded in 2012, the company has created a unique integrated R&D innovation system with the comprehensive end-to-end drug development platform (ACE Platform) and bi-specific antibody drug development technology (Tetrabody) as the core, a GMP-compliant manufacturing system and a commercialization system with an advanced operation mode, and has gradually developed into a globally competitive biopharmaceutical company focused on innovative solutions. With fully integrated multi-functional platform, Akeso is internally working on a robust pipeline of over 50 innovative assets in the fields of cancer, autoimmune disease, inflammation, metabolic disease and other major diseases. Among them, 24 candidates have entered clinical trials (including 15 bispecific/multispecific antibodies and bispecific ADCs. Additionally, 7 new drugs are commercially available. Through efficient and breakthrough R&D innovation, Akeso always integrates superior global resources, develops the first-in-class and best-in-class new drugs, provides affordable therapeutic antibodies for patients worldwide, and continuously creates more commercial and social values to become a global leading biopharmaceutical enterprise. For more information, please visit and follow us on Linkedin. SOURCE Akeso, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

近日，美国食品药品监督管理局（FDA）授予了康方生物（9926.HK）自主研发的新一代抗CD47人源化IgG4单克隆抗体莱法利单抗（AK117）孤儿药资格认定（Orphan Drug Designation，ODD），适应症为急性髓系白血病（AML）。 孤儿药资格认定是FDA为鼓励针对罕见病治疗药物研发而设立的特殊激励政策。将有助于该药物在美国的后续研发及商业化开展等方面享受众多积极的政策支持，包括享有临床试验费用税收减免、免除NDA申请费用、获得研发资助，以及该适应症批准上市后可获得美国市场7年独占权等。获得该资格认定，是FDA对莱法利单抗临床价值潜力的认可和期许。 目前，康方生物围绕莱法利单抗在血液肿瘤和实体瘤领域的临床开发均处于国际领先阶段。除AML外，莱法利单抗联合阿扎胞苷治疗较高危骨髓增生异常综合征（HR-MDS）的随机、双盲、国际多中心Ⅱ期临床研究已完成患者入组。 与此同时，莱法利单抗是全球首个进入实体瘤注册性Ⅲ期临床阶段的CD47单抗。目前，其联合依沃西一线治疗PD-L1阳性头颈部鳞状细胞癌（HNSCC）及联合依沃西一线治疗胰腺癌的两项Ⅲ期临床试验正在高效推进中。 AML是一种异质性血液系统恶性肿瘤，以骨髓、外周血及髓外组织中髓系原始细胞异常克隆增殖为主要特征，是成人中最常见的急性白血病类型。根据美国国家综合癌症网络（NCCN）指南，AML的治疗策略取决于患者是否适合接受强化诱导化疗。而对于不适合强化疗的患者，目前仍缺乏有效治疗手段。 FDA目前已批准维奈克拉联合阿扎胞苷、地西他滨或低剂量阿糖胞苷方案，用于75岁及以上或因合并症无法接受强化诱导化疗的新诊断AML患者。然而，超过半数患者会在6-9个月内复发，中位总生存期仅一年左右，临床仍存在巨大未满足需求。 莱法利单抗是一种抗CD47人源化IgG4单克隆抗体，它可特异性结合肿瘤细胞表面表达的CD47，阻断其与SIRPα受体的相互作用，解除“别吃我”信号，增强巨噬细胞对肿瘤细胞的吞噬作用，从而抑制肿瘤生长。 值得注意的是，得益于独特的药物设计，莱法利单抗不诱导红细胞凝集，药物安全性和有效性均显著优于其他同靶点药物，疗效、安全性和用药便利性显著提高。 临床前研究显示，与阿扎胞苷或维奈克拉联用时，莱法利单抗可协同增强“吃我”信号（如钙网蛋白）的表达，从而更高效地激活吞噬免疫反应。因此，莱法利单抗联合阿扎胞苷与维奈克拉的方案，有望为不适合标准诱导化疗的AML患者提供更优治疗选择。 临床研究显示，莱法利单抗联合阿扎胞苷方案安全性良好，并在AML一线治疗中展现出鼓舞人心的疗效：在高达45 mg/kg每两周给药一次的剂量下仍耐受良好，各组之间未见显著安全性差异。目标剂量的完全缓解（CR）率达50.0%，复合完全缓解（cCR）率更高达55.0%。 基于该积极结果，为进一步提升疗效，康方生物正在开展一项Ⅱ期临床研究，评估莱法利单抗联合维奈克拉与阿扎胞苷方案，用于一线不适合强化疗的AML患者的安全性与有效性。 说明：本文作为康方生物的新闻发布，用于披露公司最新进展，并非产品推广广告，不构成康方生物的信息披露或投资建议。 关于莱法利 （AK117，CD47单抗） 莱法利单抗是康方生物自主研发的新一代人源化lgG4 单克隆抗体，可与肿瘤细胞上表达的CD47结合，阻断CD47与其受体SIRPα的相互作用，增强吞噬细胞对肿瘤细胞的吞噬活性，从而抑制肿瘤生长。 莱法利单抗目前正在全球同步推进实体瘤和血液瘤临床试验，莱法利单抗也是全球首个进入实体瘤注册性Ⅲ期临床阶段的CD47单抗。莱法利联合依沃西对比帕博利珠单抗一线治疗PD-L1阳性头颈鳞癌的III期临床研究，以及莱法利联合依沃西一线治疗胰腺癌的III期临床研究均在入组中。莱法利联合维奈克拉和阿扎胞苷一线治疗unfit AML的中国II期临床研究、莱法利治疗MDS的国际多中心II期临床研究均在入组中。此前，莱法利联合阿扎胞苷治疗血液瘤，莱法利单药或联合依沃西和卡度尼利治疗多种实体瘤的II期临床研究初步研究疗效数据优异，未观察到剂量限制性毒性事件，表现出优越的安全性。 关于康方生物 康方生物（9926.HK）是一家集研究、开发、生产及商业化全球首创或同类最佳创新生物新药于一体的领先企业。自2012年成立以来，公司始终以患者为中心，以临床价值为导向，打造了独有的端对端康方全方位新药研究开发平台，建立了以Tetrabody双特异性抗体开发技术、抗体偶联（ADC）技术、siRNA/mRNA技术及细胞治疗技术为核心的研发创新体系，国际化标准的GMP生产体系和运作模式先进的商业化体系，成为了在全球范围内具有竞争力的生物医药创新公司。 公司已开发了50个以上用于治疗肿瘤、自身免疫、炎症、代谢疾病等重大疾病的创新候选药物，24个候选药已进入临床（包括15个双抗/多抗/双抗ADC），7个新药已在商业化销售，3个新药3个适应症的上市申请处于审评审批阶段。 康方生物是全球迄今唯一拥有2个肿瘤免疫双抗新药的制药公司： 2022年6月，公司全球首创的PD-1/CTLA-4双抗卡度尼利获批上市，是全球首个获批的肿瘤免疫治疗双抗新药，也是中国第一个双特异性抗体新药。目前卡度尼利一线治疗胃癌、一线治疗宫颈癌和治疗复发/转移性宫颈癌的适应症已获批，10个注册性/III期临床正在开展。 2024年5月，公司全球首创的PD-1/VEGF双抗新药依沃西获批上市，用于EGFR-TKI治疗进展的局部晚期或转移性nsq-NSCLC，成为全球首个获批的“肿瘤免疫+抗血管生成”机制双抗新药。同期，依沃西在与全球“药王”帕博利珠单抗的随机、双盲、对照一线治疗PD-L1阳性NSCLC的“头对头”III期临床研究中获得显著阳性结果，依沃西可降低患者疾病进展/死亡风险达49%，依沃西成为全球首个在头对头III期临床研究中证明疗效显著优于“药王”帕博利珠单抗的药物。基于该研究结果，依沃西该项适应症于2025年4月获批上市，成为一线肺癌治疗新的标准治疗方案，为患者提供全新更优的“去化疗”选择。目前依沃西有13项注册性/III期临床已开展，包括3项国际多中心注册性III期临床研究。 2024年12月，卡度尼利和依沃西均通过医保谈判被纳入国家新版医保目录。 国际市场开发是康方生物核心发展战略，继2015年开中国先河将自主研发的CTLA-4单抗权益许可给默沙东公司之后，2022年12月，公司再次创造中国纪录，以50亿美金+2位数百分比销售提成的方案，将依沃西部分海外权益许可给美国Summit公司，该合作创下了彼时中国新药对外许可的最高交易金额纪录。2025年2月，Summit 与美国辉瑞（Pfizer）公司达成临床试验合作，共同推进依沃西联合辉瑞多款抗体偶联药物（ADCs）在多种实体瘤中的联合治疗疗法。卡度尼利联合方案一线治疗转移性鼻咽癌和单药用以铂类为基础的化疗治疗失败的转移性鼻咽癌两项适应症于2025年4月获得美国食品药品监督管理局（FDA）批准上市。 2024年9月，公司自主研发的伊努西单抗（PCSK9）治疗高胆固醇血症两项适应症获批，成为非肿瘤领域的首个获批产品。2025年4月，公司独立自主研发的依若奇单抗（IL-12/IL-23“双靶向”单抗）获得批准上市，用于中重度斑块状银屑病成人患者的治疗。公司非肿瘤领域的产品协同效应和战略组合力进一步提升。 康方生物期望通过高效及突破性的研发创新，开发国际首创及同类药物最佳疗法的新药，为全球患者提供更优疾病解决方案，成为全球领先的生物制药企业。 往期推荐 临床 进展 HARMONi-A OS最终分析显示：依沃西达到OS临床终点，取得具有临床意义和统计学显著的OS获益 产品动态 依沃西联合化疗1L治疗sq-NSCLC的上市申请获NMPA受理 数据发布 HARMONi研究发布：PFS HR=0.52，OS HR=0.79  依沃西2L+ EGFRm NSCLC国际多中心III期 产品动态 卡度尼利获批第三项适应症：一线宫颈癌全人群 产品动态 依沃西一线治疗NSCLC获批上市（全球首个对比帕博利珠单抗获显著阳性结果的III期研究） 产品动态 派安普利2个适应症美国重磅上市！获FDA批准用于晚期鼻咽癌治疗 临床 进展 康方首个双抗ADC（Trop2/Nectin4 ADC）临床入组，“IO+ADC”2.0 迭代战略加速推进 临床 进展 康方非肿瘤领域首个双抗IL-4Rα/ST2（AK139）IND获受理，剑指呼吸系统及皮肤疾病领域 产品动态 卡度尼利、依沃西纳入2024年国家医保目录 临床 进展 头对头度伐利尤单抗方案，依沃西方案一线治疗胆道肿瘤III期临床完成首例患者入组 临床 进展 全球首个CD47单抗实体瘤III期临床首例入组：依沃西联合莱法利一线治疗HNSCC（对比帕博利珠单抗） 数据发布 IGCS 2024 LBA & 《柳叶刀》主刊，卡度尼利1L宫颈癌III期研究PFS和OS双阳性结果重磅发布 产品动态 卡度尼利第二个适应症获批：一线胃癌全人群 数据发布 全球首个对比帕博利珠单抗取得显著阳性结果的随机对照大III期研究——依沃西HARMONi-2重磅研究成果在WCLC发表 临床 进展 卡度尼利联合方案治疗uHCC的III期临床研究完成首例患者入组 临床 进展 针对PD-1/L1治疗进展晚期胃癌，卡度尼利+普络西（VEGFR-2）联合疗法Ⅲ期临床完成首例入组 数据发布 ASCO Oral & JAMA主刊丨Ⅲ期HARMONi-A研究重磅公布，依沃西疗法有望改变EGFR-TKI进展肺癌全球治疗标准 临床 进展 国际多中心注册性III期研究HARMONi-3中国启动：依沃西单抗联合化疗对比帕博利珠单抗联合化疗1L治疗sq-NSCLC 产品动态 高达50亿美金！康方生物与Summit就PD-1/VEGF双抗依沃西达成合作和许可协议 产品动态 全球首个肿瘤免疫治疗双抗——开坦尼®（PD-1/CTLA-4双抗，卡度尼利）获批上市