Prifetrastat

新药信息发布/展位/招商合作： 洪先生19957626573（微信同号） 扫码加入新药交流群 扫码预约 商务合作 3 月 4 日，国家药监局药品审评中心（CDE）官网最新公示，复宏汉霖自主研发的 1 类新药 HLX97 临床试验申请正式获得默示许可，拟用于晚期 / 转移性实体瘤的治疗。 这款新型口服组蛋白乙酰转移酶（KAT6A/B）抑制剂的获批，不仅让复宏汉霖在实体瘤创新管线布局再落关键一子，更使其成为国内第三家将 KAT6A/B 抑制剂推入临床阶段的本土药企，标志着这一表观遗传领域的明星靶点，正式迎来国产创新药的三足鼎立竞争格局。 潜在同类最优！HLX97 直击同靶点研发核心痛点 作为表观遗传调控的核心靶点，KAT6A/B 隶属于 MYST 家族的组蛋白乙酰转移酶，通过催化组蛋白 H3K23 乙酰化调控基因转录，其异常扩增、突变或易位与乳腺癌、非小细胞肺癌、胰腺癌等多种实体瘤，以及急性髓系白血病等血液肿瘤的发生发展、侵袭耐药密切相关。尤其在 ER+/HER2 - 乳腺癌中，KAT6A 高频扩增 / 过表达，是驱动肿瘤进展和内分泌治疗耐药的关键元凶，也让该靶点成为突破乳腺癌耐药困境的核心方向。 此次获批临床的 HLX97，正是复宏汉霖针对这一潜力靶点打造的差异化创新成果。作为一款高选择性口服 KAT6A/B 抑制剂，其核心作用机制是通过精准阻断 KAT6A/B 的酶活性，从表观遗传层面调控肿瘤细胞的增殖、分化与凋亡程序，从根源上遏制肿瘤的恶性进展。 而区别于全球同靶点在研药物，HLX97 最核心的竞争力，在于其直击了当前 KAT6A/B 抑制剂研发的最大行业痛点 —— 血液毒性。从全球进展最快的辉瑞 Prifetrastat（PF-07248144）临床数据来看，该产品最主要的 3 级及以上不良反应为中性粒细胞减少症，发生率最高接近 40%，成为限制药物剂量提升和临床获益的关键瓶颈。 复宏汉霖在 HLX97 的研发中，通过结构优化实现了对 KAT6A/B 的高选择性抑制，在临床前研究中展现出快速起效、快速清除的优异药代动力学特性，既能在肿瘤组织中快速达到有效药物浓度发挥抗肿瘤活性，又能通过快速代谢降低体内蓄积风险，在最大化药效的同时，显著减轻了血液毒性，为其冲击 “同类最优”（Best-in-Class）奠定了核心基础，也为后续临床开发中更宽的剂量窗口和联合用药方案提供了可能。 从靶点验证到国产竞速，KAT6A/B 为何成创新药企必争之地？ KAT6A/B 抑制剂赛道的持续升温，核心源于其明确的成药性验证与巨大的未满足临床需求。 在靶点成药性方面，辉瑞 Prifetrastat 的临床数据已完成了关键的概念验证。在针对经 CDK4/6 抑制剂和内分泌治疗失败的 ER+/HER2 - 转移性乳腺癌患者的 I 期临床中，Prifetrastat 联合氟维司群的客观缓解率（ORR）达 30.2%，中位无进展生存期（mPFS）达 10.7 个月，其中三线及以上治疗患者的 ORR 更是高达 40%，证实了 KAT6A/B 抑制剂在耐药乳腺癌中的显著临床获益，彻底打开了该靶点的临床应用空间。 而从临床需求来看，仅乳腺癌领域，ER+/HER2 - 亚型占比超 70%，其中超过 30% 的患者会在内分泌治疗联合 CDK4/6 抑制剂治疗后出现耐药，后续治疗选择极为有限。除此之外，KAT6A/B 在胰腺癌、非小细胞肺癌、NUP98 融合阳性急性髓系白血病等难治性肿瘤中均展现出治疗潜力，甚至可与 menin 抑制剂联用克服耐药，市场空间极为广阔NCBI。 更重要的是，该靶点全球研发尚处早期，目前全球尚无 KAT6A/B 抑制剂获批上市，进展最快的辉瑞 Prifetrastat 仅处于 II 期临床阶段，国内药企的研发进度与全球顶尖水平差距极小，具备实现弯道超车、拿下同类首创 / 同类最优的绝佳机会，这也是国内头部药企纷纷布局的核心原因。 国产三足鼎立成型，赛道竞争进入差异化突围新阶段 截至 HLX97 获批，国内已有 4 款 KAT6A/B 抑制剂进入临床阶段，其中进口产品 1 款（辉瑞 Prifetrastat），国产产品 3 款，分别为齐鲁制药的 QLS1304、百济神州的 BG-75202，以及此次获批的复宏汉霖 HLX97，国产药企正式形成三足鼎立的竞争格局，与跨国巨头同台竞技。 从各家布局来看，头部企业均已明确差异化的临床开发策略：百济神州的 BG-75202优先聚焦 HR+/HER2 - 乳腺癌适应症，目前已在澳大利亚启动 I 期临床，同步探索单药与 CDK 抑制剂联合用药方案，临床前研究中已展现出优于同类产品的有效性与安全性；齐鲁制药的 QLS1304 为国内首款进入临床的国产 KAT6A/B 抑制剂，率先完成了赛道卡位，在临床前研究中展现出强效的体内外抗肿瘤活性；复宏汉霖 HLX97 则以 “减血液毒性 + 高选择性” 为核心差异化优势，瞄准同靶点药物的安全性短板，有望在后续临床中展现出更优的耐受性和患者依从性。 值得注意的是，除了已进入临床的三家企业，英矽智能、人福医药、康辰药业等多家企业均已在 KAT6A/B 抑制剂领域布局，其中英矽智能的 ISM5043 已实现海外权益授权，交易总金额超 5 亿美元，印证了该靶点的全球商业价值。 从跟随到引领，国产创新药的细分赛道突围 HLX97 的获批临床，不仅是复宏汉霖从生物类似药向全球创新药转型的又一重要里程碑，更是国内创新药行业向高质量转型的缩影。 过去数年，国内创新药研发经历了从 me-too 类药物扎堆，到聚焦细分赛道、追求 me-better/best-in-class 的深度转型。KAT6A/B 抑制剂赛道的火热，本质上是国内药企研发能力升级的体现 —— 不再局限于成熟靶点的同质化竞争，而是转向全球研发尚处早期、临床需求明确的新兴靶点，通过结构优化和差异化设计，实现对进口产品的追赶甚至超越。 但同时我们也需清醒认识到，KAT6A/B 抑制剂的研发仍面临诸多挑战：如何通过生物标志物筛选精准获益人群，实现真正的精准治疗；如何优化联合用药方案，进一步提升实体瘤治疗应答率；如何在临床中验证临床前的安全性优势，都是本土药企需要攻克的难题。 可以预见的是，随着复宏汉霖、百济神州、齐鲁制药等企业的临床推进，国产 KAT6A/B 抑制剂的赛道竞速将持续升级。谁能率先拿出更优的临床数据，突破现有治疗的疗效与安全性瓶颈，谁就能在这一新兴赛道中抢占全球话语权。而对于广大晚期实体瘤患者而言，更多创新药的入局，终将带来更多治疗希望，填补耐药后治疗的临床空白。 免责声明：网上信息整理，若有涉及侵权请予以告知，我们会尽快在24小时内删除相关内容 资源对接、沟通交流 找项目、找供应商、找资源 1V1供需对接专员服务 群聊: 资源共享群 仅为公众号粉丝供需交流群 添加公众号专员加入 活动推荐 找批文、找项目、找CRO/CMO、找场地 欢迎会议现场深度对接交流~ 点击了解详情 ▼ 展位/招商合作：洪先生19957626573（微信同号） 【关于药融圈】 【药融圈PRHub】流量渠道覆盖150万+垂直用户，已完成了百余场线下千人规模的生物医药研发类会议，涵盖创新药，改良型新药，仿制药，MAH，合成生物学等领域，以及生物医药上下游研发到产业端全覆盖的【中国制药工业博览会 CMC-CHINA】，服务了百余家上市/独角兽/生物技术/制药企业。 【生态圈合作伙伴· CXO企业】 善渊制药、美迪西、富祥药业、睿智医药、华仁医学、康日百奥、珠海亿胜、倍特药业、杭州澳亚、华阳制药、亚邦生缘、华威医药、汉康医药、国标医药、斯坦德生物医药、艾奇西医药、百奥信康、慧聚药业、瀚合瑞、四川科伦、汇宇制药、广东星昊、兄弟药业、普利制药、大连美创、深圳药欣、FLAMMA、中肽生化、昂博生物、青木制药、常熟神隆、上海玉函、星诺医药、天晟药业、皓元/药源、常州阳光、湖北澳格森、杭州肽佳、世纪迈劲、南京哈柏、宏昇药业、健元医药、木槿化学、维亚生物、蓬勃生物 【生态圈合作伙伴· 制药装备&仪器】 深圳华溶、ATS安拓思、齐蓁科技、赛默飞、安捷伦、黄海药检、宣健仪器、德衡纳米、河北华胜、博奥思、莱顿科学、思勃分离、泰灵佳、相流科技、诺泽流体、康乐辉、上海汉尧 【生态圈合作伙伴·综合服务企业】 仅三生物、宝利包材、桐晖医药、壹生科、STD标准药物、双峰格雷斯海姆、嘉树医疗、齐鲁MAH创新创业基地、青岛明月海藻、盛德伟业、诚利恩、泰矶林、青松医药、奥普迪诗、Gempex、星诚达、艾里奥斯、 【生态圈合作伙伴·创新合作伙伴企业】 迈威生物、士泽生物、云顶新耀、英矽智能、拓领博泰 点分享 点收藏 点在看 点点赞

2026年3月5日，复星医药子公司复宏汉霖（2696.HK）宣布，公司自研的口服创新赖氨酸乙酰转移酶6A/B（KAT6A/B）小分子抑制剂HLX97的新药临床试验（IND）申请已获得国家药品监督管理局（NMPA）批准，拟用于晚期或转移性实体瘤的治疗，有望为乳腺癌等实体瘤耐药患者带来新的治疗方案。潜在同类最优：精准抑制KAT6A/B，挑战后线治疗KAT6A（Lysine Acetyltransferase 6A）及其旁系同源物KAT6B（Lysine Acetyltransferase 6B）是MYST 家族的组蛋白赖氨酸乙酰转移酶(HAT)，在多种肿瘤的发生发展和耐药性产生中发挥着关键作用。临床前及临床研究显示，在既往接受过多种治疗的ER+/HER2-转移性乳腺癌患者中，KAT6A基因的扩增/过表达与内分泌治疗耐药性的产生密切相关。此外，也观察到 KAT6A/B基因的异常表达出现在卵巢癌、子宫颈癌、结直肠癌、前列腺癌、肺癌和脑胶质瘤中等多种实体瘤中1-3，提示KAT6A/B抑制剂在多类肿瘤的治疗中具有潜在的应用前景。HLX97 是一款具有同类最优潜力的创新型口服KAT6A/B抑制剂。通过精准抑制KAT6A/B的活性，HLX97能够调控肿瘤细胞的增殖，诱导肿瘤细胞凋亡，具有“快速起效、快速清除”药代动力学特性和高选择性，旨在最大化药物抗肿瘤活性的同时减轻产品的血液毒性。非临床研究显示，HLX97具有良好的抗肿瘤效果和安全性。与同类产品PF-07248144相比，HLX97展现出更强的KAT6A/B抑制活性和对KAT5/7/8更高的选择性，其中，HLX97在更低剂量下即展现出与同类产品相当或更优的抗肿瘤活性；在相近抑瘤水平时，HLX97引发的血液学毒性显著更低，预示其在临床上可能拥有更宽的治疗窗口。同时，HLX97显示出良好的ADME特性，包括高口服生物利用度和优异的药代动力学特征4-5。全域全程全球：构建乳腺癌全生命周期治疗生态乳腺癌是全球第二以及女性最高发的恶性肿瘤，成为导致女性癌症死亡的主要原因。其中，ER+乳腺癌是最常见的乳腺癌亚型，约占所有乳腺癌病例的70% 6。尽管抑制雌激素生成、直接靶向ER的干预措施等内分泌治疗已成为其基石疗法，显著降低了ER+乳腺癌的复发率和死亡率，但新生耐药和获得性耐药仍然是一项重大挑战。鉴于乳腺癌中KAT6A基因的扩增/过表达已被证实与内分泌治疗耐药性的产生密切相关。因此，靶向抑制KAT6A/B活性有望成为一种克服耐药的有效策略7-9。HLX97成功获批临床，标志着复宏汉霖已经构建了完整贯通的小分子药物研发能力。从成熟靶点的差异化开发到新兴靶点的战略布局，公司正加速实现大分子与小分子药物的深度融合与优势互补。通过自主研发与战略引进，复宏汉霖已在乳腺癌领域建立了“全域全程全球”的完整治疗生态，产品管线包括中美欧等全球50多个国家和地区获批上市曲妥珠单抗生物类似药汉曲优(美国商品名：HERCESSI，欧洲商品名：Zercepac)，首个美国获批的帕妥珠单抗生物类似药POHERDY，早期强化辅助治疗药物汉奈佳（奈拉替尼），创新CDK4/6抑制剂复妥宁（伏维西利）等上市产品，以及新表位抗HER2单抗HLX22、新型内分泌疗法拉索昔芬片HLX78、LIV-1靶点ADC HLX41、HER2xHER2双表位ADC HLX49、HER2 ADC HLX87等高潜创新分子，持续推进覆盖乳腺癌各分型分期的单药及联合疗法临床研究，通过强化管线协同效应，持续提升覆盖提升治疗价值。未来，复宏汉霖将继续坚持“以患者为中心”的研发理念，依托平台化、系统化的创新体系，加速推进具有全球竞争力的差异化创新管线布局，为全球患者提供更可及、更有效的治疗选择。关于复宏汉霖复宏汉霖（2696.HK）是一家国际化创新生物制药企业，致力于为全球患者提供高品质、可负担的生物药，产品覆盖肿瘤、自身免疫疾病、眼科疾病等领域。自2010年成立以来，公司已构建涵盖全球研发、临床、注册、生产及商业化的全产业链平台，拥有全球员工近4,000人，并在中国、美国和日本等多地设有运营及分支机构。依托生物类似药形成的稳健现金流反哺创新研发，复宏汉霖正稳步迈入“全球化2.0”阶段，持续打造可复制、可持续的全球增长模式。截至2026年初，公司共有10款产品在全球60个国家和地区获批上市，其中7款已在中国获批。在欧美主流生物药市场，复宏汉霖亦取得多项里程碑式突破，已有4款产品获得美国FDA批准、4款产品获得欧盟EMA上市授权，充分体现了公司在研发体系、质量管理及生产能力方面已全面对标国际最高标准。在创新驱动方面，复宏汉霖依托上海、美国等多地协同布局的研发体系，构建了多元化、平台化的创新技术矩阵，覆盖免疫检查点抑制剂、免疫细胞衔接器（包括多特异性TCE）、抗体偶联药物（ADC）以及AI驱动的早期研发平台等前沿方向。目前，公司拥有50余项处于早期阶段的创新资产，其中约70%具备同类最佳（Best-in-Class）潜力，并在全球同步推进30余项临床研究。核心产品H药 汉斯状（斯鲁利单抗，欧洲商品名：Hetronifly）作为全球首个获批一线治疗小细胞肺癌的抗PD-1单抗，正加速全球布局，已在全球40余个市场获批上市；同时，多款潜力创新资产，包括PD-L1 ADC HLX43及新表位HER2单抗HLX22正全面推进全球关键性临床研究。依托通过中、欧、美三地GMP认证的生产体系，复宏汉霖已建成总产能达84,000升的生物药生产平台，形成覆盖全球六大洲的稳定供应网络。未来，复宏汉霖将始终坚持以患者为中心，聚焦未满足的临床需求，持续推动创新成果向临床价值与患者可及转化，在全球生物医药创新生态中创造长期而稳健的价值。Henlius Receives IND Approval for Its Potential BIC Novel Oral KAT6A/B Small Molecule Inhibitor HLX97 Shanghai, China – March 5, 2026 – Shanghai Henlius Biotech, Inc. (2696.HK) today announced that the NMPA has approved the IND application for HLX97, a novel oral small molecule inhibitor targeting Lysine Acetyltransferase 6A/B (KAT6A/B) independently developed by the company, for the treatment of advanced or metastatic solid tumors. It is expected to provide a new therapeutic option for patients with breast cancer and other solid tumors who have progressed on standard therapies.A Potential Best-in-Class KAT6A/B Inhibitor for Later-line Treatment of Solid TumorsKAT6A (Lysine Acetyltransferase 6A) and its paralog KAT6B (Lysine Acetyltransferase 6B) are histone lysine acetyltransferases (HATs) belonging to the MYST family. Together with other chromatin-associated proteins, they acetylates lysine residues on histone H3 and plays a key role in the development and drug resistance of various tumors. Preclinical and clinical studies demonstrate that, amplification or overexpression of KAT6A is closely associated with endocrine therapy in heavily pretreated patients with estrogen receptor-positive(ER+)/human epidermal growth factor receptor 2-negative (HER2-) metastatic breast cancer. In addition, dysregulation of KAT6A/B has been observed in various solid tumors, including ovarian, cervical, colorectal, prostate, lung cancers, and glioma1-3, underscoring the broad therapeutic potential of KAT6A/B inhibitors in oncology.HLX97 is a novel, oral KAT6A/B inhibitor with best-in-class potential. By precisely inhibiting KAT6A/B activity, HLX97 inhibits tumor cell proliferationand induces apoptosis. It is characterized by a "fast-on/fast-off" pharmacokinetic profile and high selectivity, designed to maximize anti-tumor efficacy while minimizing hematologic toxicity. Nonclinical studies have demonstrated that HLX97 exhibits potent anti-tumor activity and a favorable safety profile. In comparisons with PF-, HLX97 exhibited superior enzymatic inhibition and enhanced selectivity against KAT5/7/8. Notably, in preclinical studies, HLX97 demonstrated anti-tumor activity comparable or superior to the reference molecule at lower doses. Furthermore, at similar efficacy levels, HLX97 induced significantly less hematological toxicity, suggesting a potentially broader therapeutic window in the clinical setting. Additionally, HLX97 shows favorable ADME characteristics, such as high oral bioavailability and excellent pharmacokinetics 4-5.Breast cancer is the second most common cancer globally and ranks as both the most frequently diagnosed malignancy and the leading cause of cancer death among women. Among its subtypes, estrogen receptor-positive (ER+) breast cancer is the most prevalent, accounting for approximately 70% of all breast cancer cases 6. Treatment strategies for ER+ breast cancer include inhibition of estrogen production and interventions directly targeting the ER, collectively referred to as endocrine therapy. Although endocrine therapy has significantly reduced recurrence and mortality rates in breast cancer, de novo and acquired resistance remain major clinical challenges. Given the established role of KAT6A amplification/overexpression in driving endocrine therapy resistance in breast cancer, inhibition of KAT6A/B activity represents a promising strategy to fulfill this umet medical needs7-9.Building a Full Continuum Care Ecosystem for Breast CancerThe IND approval of HLX97 marks a key milestone in the establishment of Henlius’ fully integrated small-molecule drug R&D capabilities. Building on this, the company is executing a differentiated strategy across its pipeline—pursuing both innovation on established targets and moves into emerging ones—to fully harness the potential of its integrated small-molecule and biologics platforms. Henlius has built a comprehensive pipeline covering the full continuum of breast cancer care through internal innovation and strategic collaborations. Its marketed products include HANQUYOU (trade name: HERCESSI in the U.S., Zercepac in Europe), a trastuzumab biosimilar approved in over 50 countries and regions including China, the U.S., and Europe; POHERDY, the first pertuzumab biosimilar approved in the U.S.; HANNAIJIA (neratinib), an extended adjuvant therapy for early-stage breast cancer, and CDK4/6 inhibitor FUTUONING (fovinaciclib). Meanwhile, Henlius is advancing next-generation molecules such as novel epitope anti-HER2 antibody HLX22, oral selective estrogen receptor modulator (SERM) lasofoxifene (HLX78), KAT6A/B inhibitor HLX97, LIV-1-targeting ADC HLX41, HER2×HER2 bispecific epitope ADC HLX49 and HER2 ADC HLX87 through its robust innovation platforms and collaborative R&D. Building on this foundation, Henlius remains committed to advancing monotherapies and combination regimens across all breast cancer subtypes, enhancing therapeutic value through strengthened pipeline synergies.Looking ahead, Henlius will continue to adhere to its "patient-centric" R&D philosophy. Leveraging its integrated, platform‑based innovation engine, the company is committed to accelerating the development of a differentiated, globally competitive innovative pipeline, striving to provide more accessible and effective treatment options for patients worldwide.About HenliusShanghai Henlius Biotech, Inc. (2696.HK) is a global, innovation-driven biopharmaceutical company committed to delivering high-quality, affordable biologic therapies to patients worldwide. The Company focuses on major disease areas including oncology, autoimmune diseases, and ophthalmic diseases. Founded in 2010, Henlius has established an integrated, end-to-end biopharmaceutical platform encompassing global R&D, clinical operations, regulatory affairs, manufacturing, and commercialisation. The Company employs nearly 4,000 people globally and operates across multiple regions, including China, the United States, and Japan. Leveraging the stable cash flow generated from its biosimilar portfolio to support innovation, Henlius is steadily advancing into its “Globalisation 2.0” phase, building a scalable and sustainable global growth model. As of early 2026, Henlius has achieved regulatory approvals for 10 products across 60 countries and regions worldwide, including seven approvals in China. The Company has also reached multiple milestones in major biopharmaceutical markets, with four products approved by the U.S. Food and Drug Administration (FDA) and four products authorized by the European Medicines Agency (EMA), reflecting its globally aligned R&D capabilities, quality systems, and manufacturing standards.Driven by innovation, Henlius has built a diversified, platform-based technology ecosystem through coordinated R&D efforts across Shanghai, the United States, and other regions. Its innovation platforms span immune checkpoint inhibitors, immune cell engager technologies (including multispecific T cell engagers), antibody-drug conjugates (ADCs), and AI-enabled early discovery platforms. The Company currently has more than 50 early-stage innovative assets, approximately 70% of which are expected to be best-in-class, with over 30 clinical trials ongoing globally. Henlius’ core product, serplulimab (trade name: Hetronifly in Europe), is the world’s first anti–PD-1 mAb approved for first-line treatment of small cell lung cancer and has been approved in more than 40 markets worldwide with an accelerated globalisation process. In parallel, multiple high-potential innovative assets—including the PD-L1 ADC HLX43 and the novel epitope anti-HER2 mAb HLX22—are advancing through global pivotal clinical development. Supported by a biologics manufacturing network with a total capacity of 84,000L and GMP certifications from regulatory authorities in China, Europe, and the United States, Henlius has established a stable global supply system serving six continents. Guided by a patient-centred mission, Henlius remains focused on addressing unmet medical needs and translating scientific innovation into meaningful clinical value and patient access, contributing sustainably to the global biopharmaceutical ecosystem.To learn more about Henlius, visit and connect with us on LinkedIn at J, et al. Mutations of Chromatin Structure Regulating Genes in Human Malignancies. Curr Protein Pept. Sci. 2016;17(5):411-437.Partynska A, et al. The Expression of Histone Acetyltransferase KAT6A in Non-small Cell Lung Cancer. Anticancer Res. 2022;42(12):5731-5741.Lv D, et al. Histone Acetyltransferase KAT6A Upregulates PI3K/AKT Signaling through TRIM24 Binding. Cancer Res. 2017;77(22):6190-6201.Liu R, et al. Abstract 6976: Identification of novel KAT6A/B inhibitors with enhanced antitumor activity and reduced hematologic toxicity. Cancer Res (2025) 85 (8_Supplement_1): 6976. AACR Annual Meeting 2025.2026年第44届摩根大通医疗健康年会复宏汉霖投资者推介材料Nolan E, et al. Deciphering breast cancer: from biology to the clinic. Cell. 2023;186(8):1708-1728.Sharma S, et al. Discovery of a highly potent, selective, orally bioavailable inhibitor of KAT6A/B histone acetyltransferases with efficacy against KAT6A-high ER+ breast cancer. Cell Chem Biol. 2023;30(10):1191-1210.e20.Yu L, et al. Identification of MYST3 as a novel epigenetic activator of ERα frequently amplified in breast cancer. Oncogene. 2017;36(20):2910-2918.Yu L, et al. Identification of MYST3 as a novel epigenetic activator of ERα frequently amplified in breast cancer. Oncogene. 2017;36(20):2910-2918. doi:10.1038/onc.2016.433(复星医药 动态宝)