Gilteritinib in Combination With Azacitidine and Venetoclax Compared to Induction Chemotherapy "7+3" in Combination With a FLT3-inhibitor in Fit, Newly Diagnosed, FLT3-ITD Mutated Adult AML Patients: a Randomized Trial of the EORTC Leukemia Group and GIMEMA.

This international, multicenter, randomized (1:1), open-label phase II/III trial will evaluate the efficacy and safety of gilteritinib combined with azacitidine and venetoclax (experimental arm) versus standard "7+3" induction plus a FLT3inhibitor (quizartinib or midostaurin) (control arm) in newly diagnosed FLT3-ITD mutated AML patients eligible for intensive chemotherapy.

开始日期2026-12-01

申办/合作机构

European Organisation for Research & Treatment of Cancer

[+2]

NCT06992934

/ Not yet recruitingN/AIIT

Non Interventional Study Aiming to Assess the Immunological Impact of a Post Cell Therapy Treatment With FLT3 Inhibitors on the Phenotype, the Metabolism, the Function of T-cells and Monocytes and Macrophages

Allogeneic hematopoietic stem cell transplantation (aHSCT) is the only curative option for many hematological maligancies. The main cause of death following HSCT is the relapse of the original disease.
Few strategies have been developed to prevent relapse after bone marrow transplantation. New prophylactic strategies are needed to decrease the relapse incidence without increasing the non-relapse mortality in the post-transplant area. Several drugs are currently being explored as maintenance in several AML subgroups such as FLT3 ITD/mutated AML.
A specific group of AML patients display the FMS-like tyrosine kinase 3 (FLT3) internal tandem duplication (ITD) or mutation. These recurrent genetic abnormalities account for 30-35% of all AML patients. Because FLT3 is a tyrosine kinase, it can be targeted using tyrosine kinase inhibitors (TKI). Originally limited to first generation sorafenib and Midostaurin, the FLT3 TKI now include second generation Gilteritinib, crenolanib and quizartinib13. Because many FLT3 AML patients would relapse after aHSCT, the use of sorafenib, the only FLT3 TKI available at that time, in a post-transplant setting started to be evaluated.
Sorafenib is a multi-kinase inhibitor that not only inhibit FLT3 but also the RAS, RAF, KIT, and the VEGF and platelet-derived growth factor receptor. Several retrospective trials reported the safety and efficiency of a sorafenib maintenance.
In recent years, a mounting piece of evidence suggests that sorafenib may have an impact on several immune cells as T cell populations, dendritic cells, macrophages and myeloid-derived immunosuppressor cells (MDSC). Other more potent and more specific FLT3 inhibitors are currently under investigation both in combination with chemotherapy before transplantation and as post transplantation maintenance. In this line, crenolanib and Gilteritinib are two potent and more specific TKI that proved more effective than sorafenib in the treatment of FLT3 ITD/TKD relapsed, refractory AML patients. These drugs demonstrated a higher response rate in R/R patients. These two drugs are part of the future standard of care in FLT3 AML but their immunological impact has never been studied.
At a time where cellular therapies (allogeneic stem cell transplantation but also CAR T cells) and targeted therapies are becoming the central point of cancer treatment, it appears mandatory to better understand the interactions between the two. The goal of our research project which covers fundamental and clinical aspects of AML post-transplant treatments is devoted to a better understanding of the impact of Gilteritinib on the immune cells in order to rationalize their use after aHSCT. A better characterization of the action of these drugs on the immune system is urgently needed to develop new prophylactic strategies which could decrease the relapse incidence without increasing the non-relapse mortality in the post-transplant area. This program is proposed for a period of 3 years, time necessary to perform all the in vitro and ex vivo approaches and to recruit a sufficient number of patients eligible for aHSCT.

开始日期2026-09-01

申办/合作机构

Centre Hospitalier Universitaire de Nice

NCT07755202

/ Not yet recruiting临床2期IIT

A Phase 2 Trial of Gilteritinib in Combination With Azacitidine and Venetoclax to Overcome Venetoclax Resistance in Patients With Relapsed/Refractory FLT3-wild Type Acute Myeloid Leukemia

The goal of this clinical trial is to learn if gilteritinib-azacitidine-venetoclax combination drug therapy works to treat adults with FLT3-wt acute myeloid leukemia (AML). It will also learn about the safety and efficacy of this treatment. The main question this clinical trial aims to answer are:
* Will adding gilteritinib to standard-of-care therapy azacitidine-venetoclax show be more effective in treating AML FLT3-wt patients who have previously been treated with azacitidine and/or venetoclax for their disease and have had their cancer come back (relapsed) or have stopped responding to treatment (refractory)?
All participants in this trial will receive the combination therapy. Participants will be on repeated 28-day cycles, for a minimum of 2 cycles, while on the study
Participants will:
* Take the gilteritinib once daily, every day
* Take azacitidine and venetoclax on days 1-7 of each cycle
* Keep a daily dose diary of the days and times they have taken their treatments
* Visit the clinic every 4 weeks for checkups and tests

开始日期2026-09-01

申办/合作机构

British Columbia Cancer Agency

100 项与富马酸吉瑞替尼相关的临床结果

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100 项与富马酸吉瑞替尼相关的转化医学

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100 项与富马酸吉瑞替尼相关的专利（医药）

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700

项与富马酸吉瑞替尼相关的文献（医药）

2026-12-01·Blood Research

Real-world experience with gilteritinib maintenance following allogeneic transplantation in relapsed/refractory AML patients harboring FLT3 mutations

Article

作者: Ko, Bor-Sheng ; Tsai, Xavier Cheng-Hong ; Tseng, Mei-Hsuan ; Lin, Chien-Chin ; Cheng, Chieh-Lung ; Hou, Hsin-An ; Kuo, Yuan-Yeh ; Tien, Feng-Ming ; Liu, Jia-Hau ; Tien, Hwei-Fang ; Lin, Yun-Tzu ; Yao, Ming ; Chou, Wen-Chien ; Lin, Ming-En ; Chou, Sheng-Chieh ; Chuang, Ming-Kai

Abstract:

Background:

Gilteritinib is an established FLT3 inhibitor used to treat patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) harboring FLT3 mutations. Prior studies have predominantly evaluated gilteritinib as a posttransplant maintenance therapy in patients who undergo allogeneic hematopoietic stem cell transplantation (allo-HSCT) in complete remission. Nonetheless, evidence remains limited for patients who receive gilteritinib as salvage therapy in a relapsed or refractory setting, proceed to allo-HSCT, and subsequently continue gilteritinib maintenance. This real-world study aimed to address this knowledge gap in an Asian cohort.

Method and Result:

Patients with FLT3 -mutated R/R AML received gilteritinib before transplantation, and those achieving engraftment without grade ≥ 2 acute graft-versus-host disease were offered posttransplant maintenance between days 30 and 90. The cohort (median age, 48 years; 92.0% FLT3 -ITD) achieved a complete response (CR) rate of 76.2%. At a median follow-up of 46.2 months, gilteritinib maintenance therapy was associated with longer relapse-free and overall survival and a significantly lower 1-year cumulative incidence of relapse. Exploratory measurable residual disease (MRD) subgroup analyses suggested a numerically greater survival difference in patients without flow cytometry-detectable MRD before or after transplantation; however, the findings are limited by the small sample size and lack of FLT3 -targeted next-generation sequencing-based MRD assessment.

Conclusion:

Gilteritinib was generally well-tolerated, with no observed increase in cytomegalovirus (CMV)-related or graft-versus-host complications. This study provides real-world evidence in a clinically relevant scenario and supports the use of gilteritinib maintenance in patients with FLT3 -mutated AML who undergo transplantation during R/R disease.

2026-12-01·Blood Research

Recent advances in the treatment of FLT3-mutated acute myeloid leukemia

Letter

作者: Koh, Jeong Suk ; Song, Ik-Chan

FMS-like tyrosine kinase 3 (FLT3) mutations are among the most common driver alterations in acute myeloid leukemia (AML), occurring in approximately 25-30% of patients, and have historically been associated with high relapse rates and poor survival. The therapeutic landscape for FLT3-mutated AML has evolved substantially with the development of FLT3-targeted agents. In newly diagnosed AML, the addition of midostaurin or quizartinib to intensive induction chemotherapy has demonstrated significant improvements in overall survival (OS), event-free survival, and relapse-free survival, establishing these regimens as current standards of care. In the relapsed or refractory setting, gilteritinib has become the preferred treatment based on superior survival and remission outcomes compared with salvage chemotherapy. Molecular studies have identified genomic factors associated with response and resistance to FLT3 inhibition, highlighting the importance of co-occurring mutations in treatment selection and prognosis. Maintenance therapy following allogeneic hematopoietic stem cell transplantation has emerged as a promising strategy, with evidence supporting the use of sorafenib and gilteritinib to reduce relapse risk and improve posttransplant outcomes. Recent clinical investigations have focused on novel combination approaches, particularly triplet regimens, incorporating FLT3 inhibitors, venetoclax, and hypomethylating agents, which have demonstrated encouraging responses and survival outcomes. Next-generation FLT3 inhibitors, including crenolanib and dapolsertib, are being evaluated in ongoing studies. These advances continue to improve outcomes in patients with FLT3-mutated AML and are expected to further refine treatment strategies across disease settings.

2026-08-01·LEUKEMIA

Sphingosine-1-phosphate receptor modulators resensitize FLT3-ITD acute myeloid leukemia cells with NRAS mutations to FLT3 inhibitors

Article

作者: Mustafa Ali, Moaath K ; Baer, Maria R ; Liu, Yuchen ; Traer, Elie ; Bailey, Christopher M ; Wang, Yin ; Chatterjee, Aditi ; Smith, Catherine C ; Small, Donald ; Silvestri, Giovannino

FLT3 inhibitor efficacy in AML with FLT3-ITD is short-lived, frequently due to new mutations, most commonly in NRAS. Sphingosine kinase 1 (SPHK1), which phosphorylates sphingosine to generate sphingosine-1-phosphate (S1P), is upregulated and localized to the plasma membrane in RAS-mutated cells. We studied S1P and FLT3 co-targeting to overcome FLT3 inhibitor resistance in NRAS-mutated FLT3-ITD AML cells. NRAS-mutated FLT3-ITD AML cell lines and patient blasts were treated with FLT3 inhibitors and/or S1P receptor (S1PR) modulators. FLT3 inhibitor sensitivity was assessed by immunoblotting, cytotoxicity, apoptosis and colony formation. Co-treatment was also assessed in vivo in an orthotopic mouse model. Downstream RAS and SPHK1 effectors were measured by immunoblotting and qRT-PCR. The S1PR modulators fingolimod (FTY720) and mocravimod (KRP-203) resensitized FLT3-ITD-expressing MOLM-14 and MV4-11 human AML cells with G12D, G12S, Q61K or Q61H, but not G12C, and patient blasts with G13D, G13V or G12D NRAS mutations to FLT3 inhibitors. Moreover, FTY720 co-treatment resensitized G12D NRAS-mutated M14(R)701 cells to gilteritinib in vivo. Co-treatment inactivated ERK, transcriptionally downregulated SPHK1, and inactivated downstream AKT, p70 S6K and BAD, with inactivation abrogated by constitutive SPHK1 expression. The clinically applicable S1PR modulators fingolimod and mocravimod resensitize NRAS-mutated FLT3-ITD AML cells to FLT3 inhibitors, supporting potential clinical efficacy.

378

项与富马酸吉瑞替尼相关的新闻（医药）

2026-09-02

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【IPO前哨】邦顺制药二度闯关港股：核心产品刚获批，营收却归零

财华社讯，8月30日，杭州邦顺制药股份有限公司（下称“邦顺制药”）向港交所主板递交上市申请，中信建投国际担任独家保荐人。该公司曾于2026年1月22日首次递表，此次为第二次递表，寻求根据上市规则第18A章上市。核心产品贝泽昔替尼刚刚获得NMPA批准上市，但公司的财务数据却呈现出“产品获批、营收归零”的诡异局面。招股书显示，邦顺制药是一家处于商业化阶段的生物科技公司，专注于研发肿瘤及自身免疫性疾病创新药。核心产品贝泽昔替尼已于2026年获NMPA批准用于骨髓纤维化（MF），并被CSCO临床指南推荐用于原发性血小板增多症（ET）和MF。然而，产品获批并未立即转化为收入，公司2026年上半年营收直接归零，亏损持续扩大。此次二度递表，邦顺制药能否获得资本市场的认可？ 01 营收归零与亏损扩大的悖论邦顺制药的财务数据，勾勒出一家典型18A生物科技公司的困境。2024年，公司实现营业收入94.3万元，期内亏损8844.4万元；2025年，营业收入降至50.9万元，同比下滑46%，期内亏损却大幅扩大至2.99亿元，同比增幅达237.6%；2026年上半年，营业收入直接归零，期内亏损8015.5万元。收入持续萎缩至归零，主要源于公司技术转让业务的完全停滞。招股书显示，公司往绩记录期收入主要来自临床前项目的技术转让，且仅有一名客户，客户集中度达100%。随着技术转让业务的结束，公司收入来源彻底中断，而核心产品贝泽昔替尼虽已获批，但商业化尚处于起步阶段，尚未形成规模销售。亏损持续扩大的背后，是研发投入的刚性增长。2024年、2025年、2026年上半年，公司研发成本分别为6453万元、8650万元、4398万元，呈持续增长态势。对于一家创新药企业而言，研发投入是维持管线竞争力的核心，但在收入归零的情况下，持续增长的研发投入意味着公司需要不断融资来维持运营。更令人担忧的是公司的现金储备。截至最新数据，邦顺制药剩余现金储备不足1亿元。以2025年2.99亿元的亏损规模计算，现有现金储备仅能维持不到半年的运营。这意味着，若IPO募资不及预期或后续融资受阻，公司可能面临流动性危机。这也是邦顺制药急于二度递表的核心原因。 02 贝泽昔替尼：商业化前景与竞争格局尽管财务数据惨淡，但邦顺制药的核心资产贝泽昔替尼仍具备一定的市场潜力。贝泽昔替尼是一种新型JAK抑制剂，用于治疗骨髓纤维化（MF）。骨髓纤维化是一种罕见的血液系统恶性肿瘤，中国患者群体约有数十万人，目前治疗选择有限。招股书显示，中国骨髓增殖性肿瘤药物(MPN)市场于2024年达到9亿美元，预计于2030年增加至15亿美元，2024年至2030年复合年增长率约为10%。贝泽昔替尼作为新一代JAK抑制剂，在疗效和安全性方面具备一定优势，被CSCO指南推荐用于ET和MF，有望在这一快速增长的市场中占据一席之地。然而，骨髓纤维化赛道的竞争正在加剧。目前，诺华的芦可替尼（Ruxolitinib）是骨髓纤维化的标准治疗药物，占据市场主导地位。此外，吉瑞替尼、菲达替尼等同类产品也在加速布局。贝泽昔替尼作为后来者，需要在疗效、安全性、价格等方面证明自身的差异化优势，才能在激烈的市场竞争中突围。商业化能力是邦顺制药面临的另一大挑战。公司目前尚未建立成熟的销售团队，核心产品刚获批，医院准入、医保谈判、渠道建设等工作都需要时间。对于一家18A生物科技公司而言，从“产品获批”到“规模销售”之间存在巨大的鸿沟。邦顺制药能否跨越这一鸿沟，取决于公司的商业化战略和执行能力。 03 股权结构与估值逻辑 2019年6月，年逾七旬的李邦良从华东医药（000963.SZ）董事长一职退居二线，次年5月便与吕裕斌携手创立邦顺制药。此外，2000年6月至2023年11月期间，李邦良曾在九源基因（02566.HK）出任董事兼董事长。如今已届80岁的李邦良依然老当益壮，携邦顺制药冲刺港交所，目前仍以31.25%的总持股比例担任公司实际控制人，同时兼任董事长及执行董事，统筹整体业务管理与战略发展；45岁的吕裕斌持股27.76%，出任执行董事兼总经理，负责整体战略规划、创新药研发与业务运营。另外，李邦良54岁的儿子李阅东担任执行董事兼副总经理，主理公司产品商业化、政府及监管事务和投融资事宜。估值方面，邦顺制药去年底完成C轮融资后，估值达23.87亿元。这一估值在18A生物科技公司中处于中等水平。然而，考虑到公司核心产品刚获批、营收归零、现金储备不足等因素，约24亿元的估值是否合理，市场存在争议。此次二度递表，邦顺制药的估值逻辑将面临市场的重新检验。一方面，核心产品贝泽昔替尼的获批为公司提供了商业化的基础，骨髓纤维化市场的增长潜力为估值提供了支撑；另一方面，营收归零、亏损扩大、现金储备不足等问题，又对估值形成了压制。投资者需要在“产品潜力”与“财务风险”之间做出权衡。对于邦顺制药而言，此次IPO不仅是一次融资，更是一场“生死考验”。若成功上市，公司将获得宝贵的资金支持，推进贝泽昔替尼的商业化和后续管线的研发；若上市失败，公司可能面临资金链断裂的风险。核心产品刚获批，营收却已归零，邦顺制药的港股之路，注定不会平坦。 ✍ 作者丨无为 ✍ 编辑丨Danna 往期精彩推荐一图解码：港股IPO一周回顾 21家公司递表绿的谐波筹划港股上市【IPO追踪】尴尬登场！“具身眼脑手第一股”梅卡曼德（09615.HK）盘中破发【IPO前哨】演唱会“卖水人”兴明亚洲递表：为何利润承压？【IPO前哨】迪哲医药（688192.SH）再赴港：大额授权交易落地，成色几何

2026-09-01

·今日头条

邦顺制药二度闯关港股：核心产品刚获批，营收却归零

财华社讯，8月30日，杭州邦顺制药股份有限公司（下称“邦顺制药”）向港交所主板递交上市申请，中信建投国际担任独家保荐人。该公司曾于2026年1月22日首次递表，此次为第二次递表，寻求根据上市规则第18A章上市。核心产品贝泽昔替尼刚刚获得NMPA批准上市，但公司的财务数据却呈现出“产品获批、营收归零”的诡异局面。招股书显示，邦顺制药是一家处于商业化阶段的生物科技公司，专注于研发肿瘤及自身免疫性疾病创新药。核心产品贝泽昔替尼已于2026年获NMPA批准用于骨髓纤维化（MF），并被CSCO临床指南推荐用于原发性血小板增多症（ET）和MF。然而，产品获批并未立即转化为收入，公司2026年上半年营收直接归零，亏损持续扩大。此次二度递表，邦顺制药能否获得资本市场的认可？一、营收归零与亏损扩大的悖论邦顺制药的财务数据，勾勒出一家典型18A生物科技公司的困境。2024年，公司实现营业收入94.3万元，期内亏损8844.4万元；2025年，营业收入降至50.9万元，同比下滑46%，期内亏损却大幅扩大至2.99亿元，同比增幅达237.6%；2026年上半年，营业收入直接归零，期内亏损8015.5万元。收入持续萎缩至归零，主要源于公司技术转让业务的完全停滞。招股书显示，公司往绩记录期收入主要来自临床前项目的技术转让，且仅有一名客户，客户集中度达100%。随着技术转让业务的结束，公司收入来源彻底中断，而核心产品贝泽昔替尼虽已获批，但商业化尚处于起步阶段，尚未形成规模销售。亏损持续扩大的背后，是研发投入的刚性增长。2024年、2025年、2026年上半年，公司研发成本分别为6453万元、8650万元、4398万元，呈持续增长态势。对于一家创新药企业而言，研发投入是维持管线竞争力的核心，但在收入归零的情况下，持续增长的研发投入意味着公司需要不断融资来维持运营。更令人担忧的是公司的现金储备。截至最新数据，邦顺制药剩余现金储备不足1亿元。以2025年2.99亿元的亏损规模计算，现有现金储备仅能维持不到半年的运营。这意味着，若IPO募资不及预期或后续融资受阻，公司可能面临流动性危机。这也是邦顺制药急于二度递表的核心原因。二、贝泽昔替尼：商业化前景与竞争格局尽管财务数据惨淡，但邦顺制药的核心资产贝泽昔替尼仍具备一定的市场潜力。贝泽昔替尼是一种新型JAK抑制剂，用于治疗骨髓纤维化（MF）。骨髓纤维化是一种罕见的血液系统恶性肿瘤，中国患者群体约有数十万人，目前治疗选择有限。招股书显示，中国骨髓增殖性肿瘤药物(MPN)市场于2024年达到9亿美元，预计于2030年增加至15亿美元，2024年至2030年复合年增长率约为10%。贝泽昔替尼作为新一代JAK抑制剂，在疗效和安全性方面具备一定优势，被CSCO指南推荐用于ET和MF，有望在这一快速增长的市场中占据一席之地。然而，骨髓纤维化赛道的竞争正在加剧。目前，诺华的芦可替尼（Ruxolitinib）是骨髓纤维化的标准治疗药物，占据市场主导地位。此外，吉瑞替尼、菲达替尼等同类产品也在加速布局。贝泽昔替尼作为后来者，需要在疗效、安全性、价格等方面证明自身的差异化优势，才能在激烈的市场竞争中突围。商业化能力是邦顺制药面临的另一大挑战。公司目前尚未建立成熟的销售团队，核心产品刚获批，医院准入、医保谈判、渠道建设等工作都需要时间。对于一家18A生物科技公司而言，从“产品获批”到“规模销售”之间存在巨大的鸿沟。邦顺制药能否跨越这一鸿沟，取决于公司的商业化战略和执行能力。三、股权结构与估值逻辑 2019年6月，年逾七旬的李邦良从华东医药（000963.SZ）董事长一职退居二线，次年5月便与吕裕斌携手创立邦顺制药。此外，2000年6月至2023年11月期间，李邦良曾在九源基因（02566.HK）出任董事兼董事长。如今已届80岁的李邦良依然老当益壮，携邦顺制药冲刺港交所，目前仍以31.25%的总持股比例担任公司实际控制人，同时兼任董事长及执行董事，统筹整体业务管理与战略发展；45岁的吕裕斌持股27.76%，出任执行董事兼总经理，负责整体战略规划、创新药研发与业务运营。另外，李邦良54岁的儿子李阅东担任执行董事兼副总经理，主理公司产品商业化、政府及监管事务和投融资事宜。估值方面，邦顺制药去年底完成C轮融资后，估值达23.87亿元。这一估值在18A生物科技公司中处于中等水平。然而，考虑到公司核心产品刚获批、营收归零、现金储备不足等因素，约24亿元的估值是否合理，市场存在争议。此次二度递表，邦顺制药的估值逻辑将面临市场的重新检验。一方面，核心产品贝泽昔替尼的获批为公司提供了商业化的基础，骨髓纤维化市场的增长潜力为估值提供了支撑；另一方面，营收归零、亏损扩大、现金储备不足等问题，又对估值形成了压制。投资者需要在“产品潜力”与“财务风险”之间做出权衡。对于邦顺制药而言，此次IPO不仅是一次融资，更是一场“生死考验”。若成功上市，公司将获得宝贵的资金支持，推进贝泽昔替尼的商业化和后续管线的研发；若上市失败，公司可能面临资金链断裂的风险。核心产品刚获批，营收却已归零，邦顺制药的港股之路，注定不会平坦。

2026-08-31

·BioSpace

ORYZON Announces Notice of Allowance for U.S. Patent Application Covering Iadademstat Combination With Gilteritinib

Iadademstat is under clinical development in acute myeloid leukemia (AML), including in combination with gilteritinib in the Phase Ib FRIDA trial in patients with FLT3-mutated relapsed/refractory (R/R) AML FRIDA data presented at EHA-2026 showed a high composite complete remission (CRc) rate of 67% in heavily pretreated FLT3-mutated R/R AML patients, together with a favorable safety profile MADRID and CAMBRIDGE, Mass., Aug. 31, 2026 (GLOBE NEWSWIRE) -- Oryzon Genomics, S.A. (ISIN Code: ES0167733015, ORY), a clinical-stage biopharmaceutical company and a global leader in epigenetics, today announced that the United States Patent and Trademark Office (USPTO) has issued a Notice of Allowance for U.S. patent application No. US 18/554,241, entitled “Combinations of LSD1 inhibitors for treating myeloid cancers”. The allowed claims cover combinations of iadademstat, or certain other LSD1 inhibitors, with gilteritinib and their use for the treatment of myeloid cancers, including acute myeloid leukemia (AML). Once granted, the U.S. patent is expected to expire in 2042, excluding any potential patent term adjustment or patent term extension. A corresponding patent has recently also been granted in Taiwan, with additional patent applications pending in other jurisdictions. “This U.S. Notice of Allowance further strengthens the intellectual property protection around iadademstat and its use in combination with gilteritinib in AML,” said Neus Virgili, Oryzon’s Chief IP Officer. “Together with our broader portfolio of patents covering iadademstat combinations with other AML therapies, this allowance reinforces the long-term protection of our iadademstat franchise.” Iadademstat is currently being evaluated in seven ongoing oncology clinical trials, including the Phase Ib FRIDA study in combination with gilteritinib in FLT3-mutated relapsed/refractory AML and the Phase Ib ALICE-2 study in combination with venetoclax and azacitidine in first-line AML. Updated positive clinical data from both studies were presented at the European Hematology Association (EHA) 2026 Annual Congress in June. In FRIDA, iadademstat in combination with gilteritinib achieved a composite complete remission (CRc) rate of 67% at the dose level selected for expansion in a heavily pretreated population with relapsed/refractory FLT3-mutated AML, together with a favorable safety profile. These results are particularly encouraging when compared with contemporary real-world data for gilteritinib monotherapy in a broadly comparable population, which reported a CR/CRi rate of 28% and a median overall survival of 7.1 months. In ALICE-2, iadademstat in combination with venetoclax and azacitidine achieved a 100% overall response rate, an 89% CRc rate and a 78% complete remission rate. These findings compare favorably with historical outcomes for venetoclax plus azacitidine, where approximately 36% of patients failed to achieve a clinical response. Additional, more mature data from both studies are expected to be presented by year-end. In addition to this patent family covering combinations with gilteritinib, Oryzon has patent protection covering combinations of iadademstat with venetoclax, azacitidine and other AML therapies, including granted patents in the United States and other major markets. About Oryzon Founded in 2000 and headquartered in Barcelona, Spain, Oryzon (ISIN: ES0167733015) is a clinical-stage biopharmaceutical company and a European leader in epigenetics, with a strong focus on personalized medicine for central nervous system (CNS) disorders and oncology. Oryzon’s team comprises highly experienced pharmaceutical professionals based in Barcelona, Boston, and New Jersey. The Company has an advanced clinical portfolio built around two LSD1 inhibitors: iadademstat, its oncology/hematology program, with several ongoing Phase I and II studies and which has demonstrated strong preliminary clinical activity in acute myeloid leukemia, including a 100% overall response rate (ORR) in first-line AML; and vafidemstat, its lead CNS program, which is Phase III–ready in borderline personality disorder (BPD). In addition, Oryzon is advancing a broader epigenetics pipeline targeting other mechanisms, including HDAC6, for which the Company has nominated ORY-4001 as a clinical candidate for potential development in Charcot–Marie–Tooth disease (CMT), amyotrophic lateral sclerosis (ALS), and other neurological disorders. The Company also operates a robust platform for biomarker identification and target validation across malignant and neurological diseases. For more information, visit About Iadademstat Iadademstat (ORY-1001) is an oral, highly selective inhibitor of the epigenetic enzyme LSD1, with potent differentiating effect in hematologic cancers. Iadademstat has shown encouraging safety and strong clinical activity in combination with azacitidine in a Phase IIa trial in elder 1L acute myeloid leukemia (AML) patients (ALICE trial). Iadademstat is currently being evaluated in combination with azacitidine and venetoclax in 1L AML in the ALICE-2 trial, an investigator-initiated study (IIS) led by OHSU, and in combination with gilteritinib in the company-sponsored Phase Ib FRIDA trial in relapsed/refractory FLT3-mutant AML, with highly encouraging preliminary safety and efficacy data in both trials reported at EHA-2026: 100% overall response rate (ORR) and 89% composite complete remission rate (CRc), with 78% strict CR in 1L AML, and 67% CRc in R/R Flt3-mut AML. Subject to positive final data in the ALICE-2 study, the company has announced plans to conduct a potentially registrational study in 1L AML. Additional studies in hematologic malignancies include an IIS in myelodysplastic syndrome (MDS) and National Cancer Institute (NCI)-sponsored trials in myeloproliferative neoplasms and 1L AML conducted under the Cooperative Research and Development Agreement (CRADA) between Oryzon and the NCI. Beyond hematological cancers, iadademstat is being evaluated in extensive stage small cell lung cancer (ED-SCLC) in a Phase I/II randomized trial in 1L in combination with immune checkpoint inhibition (ICI) sponsored by NCI and led by the Memorial Sloan Kettering Cancer Center, and an IIS trial in combination with ICI and radiotherapy. Oryzon has also expanded iadademstat into non-oncological hematology indications, with ongoing trials in sickle cell disease and essential thrombocythemia. Iadademstat has orphan drug designation for AML in the US and EU and for SCLC in the US. FORWARD-LOOKING STATEMENTS This communication contains, or may contain, forward-looking information and statements about Oryzon, including financial projections and estimates and their underlying assumptions, statements regarding plans, objectives, and expectations with respect to future operations, capital expenditures, synergies, products and services, and statements regarding future performance. Forward-looking statements are statements that are not historical facts and are generally identified by the words “expects,” “anticipates,” “believes,” “intends,” “estimates” and similar expressions. Although Oryzon believes that the expectations reflected in such forward-looking statements are reasonable, investors and holders of Oryzon shares are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Oryzon that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include those discussed or identified in the documents sent by Oryzon to the Spanish Comisión Nacional del Mercado de Valores (CNMV), which are accessible to the public. Forward-looking statements are not guarantees of future performance and have not been reviewed by the auditors of Oryzon. You are cautioned not to place undue reliance on the forward-looking statements, which speak only as of the date they were made. All subsequent oral or written forward-looking statements attributable to Oryzon or any of its members, directors, officers, employees, or any persons acting on its behalf are expressly qualified in their entirety by the cautionary statement above. All forward-looking statements included herein are based on information available to Oryzon on the date hereof. Except as required by applicable law, Oryzon does not undertake any obligation to publicly update or revise any forward‐looking statements, whether as a result of new information, future events, or otherwise. This document does not constitute an offer or invitation to purchase or subscribe shares in accordance with the provisions of Regulation (EU) 2017/1129 of the European Parliament and of the Council of 14 June 2017, and/or the restated text of the Securities Market Law, approved by Law 6/2023 of 17 March, and its implementing regulations. Nothing in this document constitutes investment advice. In addition, this document does not constitute an offer of purchase, sale or exchange, nor a request for an offer of purchase, sale or exchange of securities, nor a request for any vote or approval in any jurisdiction. The shares of Oryzon Genomics, S.A. may not be offered or sold in the United States of America except pursuant to an effective registration statement under the Securities Act of 1933 or pursuant to a valid exemption from registration. Spain Oryzon IR & Media, Europe & US Patricia Cobo/Mario Cordera Emili Torrell Sandya von der Weid Atrevia Chief BD Officer LifeSci Advisors, LLC +34 91 564 07 25 +34 673 33 97 65 +34 93 515 1313 +41 78 680 05 38 pcobo@atrevia.com mcordera@atrevia.com etorrell@oryzon.com svonderweid@lifesciadvisors.com

临床结果临床1期临床2期孤儿药临床3期

100 项与富马酸吉瑞替尼相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D10709 D10800	-	L01XE54	DB12141

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
急性髓性白血病	加拿大	Astellas Pharma Canada, Inc.	2019-12-23
FLT3阳性急性髓性白血病	日本	Astellas Pharma, Inc.	2018-09-21

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
难治性贫血伴原始细胞过多	临床3期	澳大利亚	Astellas Pharma Global Development, Inc.	2019-12-20
难治性贫血伴原始细胞过多	临床3期	奥地利	Astellas Pharma Global Development, Inc.	2019-12-20
难治性贫血伴原始细胞过多	临床3期	比利时	Astellas Pharma Global Development, Inc.	2019-12-20
难治性贫血伴原始细胞过多	临床3期	芬兰	Astellas Pharma Global Development, Inc.	2019-12-20
难治性贫血伴原始细胞过多	临床3期	法国	Astellas Pharma Global Development, Inc.	2019-12-20
难治性贫血伴原始细胞过多	临床3期	德国	Astellas Pharma Global Development, Inc.	2019-12-20
难治性贫血伴原始细胞过多	临床3期	爱尔兰	Astellas Pharma Global Development, Inc.	2019-12-20
难治性贫血伴原始细胞过多	临床3期	立陶宛	Astellas Pharma Global Development, Inc.	2019-12-20
难治性贫血伴原始细胞过多	临床3期	荷兰	Astellas Pharma Global Development, Inc.	2019-12-20
难治性贫血伴原始细胞过多	临床3期	挪威	Astellas Pharma Global Development, Inc.	2019-12-20

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临床结果

适应症

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT06225427 (WCLC2026)	临床1期	ALK阳性非小细胞肺癌 ALK+	11	Gilteritinib 120 mg/day	簾憲遞積夢觸淵觸糧簾(觸艱壓衊壓窪選遞襯衊) = 範艱選淵艱願願觸憲觸艱壓蓋製憲壓積膚膚積 (醖蓋構獵壓網鹹選襯鹹 ) 更多	积极	2026-09-12
NCT04027309 (HOVON156, EHA2026)	临床3期	伴有 FLT3/ITD 突变的急性髓系白血病一线 FLT3 mutation	768	Gilteritinib	窪構築餘憲築鹽鹹網製(製顧範獵糧積觸鏇範壓) = 壓淵遞觸糧餘壓醖範遞鹹憲蓋顧選夢獵廠鹽積 (願糧鑰繭鬱遞繭廠齋蓋 ) 更多	不佳	2026-06-02
				Midostaurin	窪構築餘憲築鹽鹹網製(製顧範獵糧積觸鏇範壓) = 襯醖夢鏇築蓋醖範壓夢鹹憲蓋顧選夢獵廠鹽積 (願糧鑰繭鬱遞繭廠齋蓋 ) 更多
RATIFY (ASCO2026)	临床3期	伴有 FLT3/ITD 突变的急性髓系白血病一线 FLT3-mutated	-	Midostaurin+chemotherapy	簾築衊觸網壓餘遞繭憲(糧獵蓋遞襯憲淵窪糧淵): HR = 0.78 (95.0% CI, 0.63 ~ 0.96) 更多	积极	2026-05-29
				Quizartinib+chemotherapy
EHA2026	N/A	急性髓性白血病	12	Venetoclax+Gilteritinib	網網醖齋繭憲衊鏇觸醖(鏇願糧鹹選簾積鏇鑰觸) = No QTc prolongation 觸遞襯顧顧鑰壓淵願餘 (選構獵製襯繭積醖壓艱 ) 更多	积极	2026-05-12
EHA2026	N/A	FLT3阳性急性髓性白血病 FLT3-mutated	29	concomitant triplet therapy	膚襯餘製餘壓衊蓋廠構(艱顧憲繭範夢膚襯鑰壓) = 蓋鬱糧築鑰鹹網鏇繭製艱衊鬱艱範壓襯遞膚壓 (衊製襯鬱鏇襯網糧繭壓 ) 更多	积极	2026-05-12
				AZA and VEN followed by concomitant VEN and GILT	齋鬱壓餘鹹憲築選廠鹽(壓糧選蓋壓構壓憲鏇製) = 夢窪壓選範構襯夢淵憲簾憲艱顧獵蓋選膚繭壓 (鏇淵繭鹹簾簾鏇繭願蓋 ) 更多
EHA2026	临床1/2期	伴有 FLT3/ITD 突变的急性髓系白血病 FLT3-MUTATED	13	Gilteritinib + Momelotinib	膚築壓網觸襯鹽積鏇獵(襯襯廠鏇繭鹹淵願夢選) = not observed at any dose level 簾鏇鑰鹹積鹽選選鏇遞 (繭糧膚齋廠夢醖蓋廠醖 ) 更多	积极	2026-05-12
EHA2026	N/A	FLT3阳性急性髓性白血病 FLT3 mutations	54	Gilteritinib plus venetoclax (Gil+Ven)	壓願醖築觸襯製糧選醖(衊憲廠糧網鹹夢齋製鬱) = 鏇齋構鹹網遞構齋遞顧製範範鬱窪襯餘選廠網 (繭壓糧獵繭簾壓憲艱窪 ) 更多	积极	2026-05-12
				Gilteritinib plus venetoclax and "X" (gilteritinib plus venetoclax and azacitidine, gilteritinib plus venetoclax, azacitidine and homoharringtonine) (Gil+Ven+X)	壓願醖築觸襯製糧選醖(衊憲廠糧網鹹夢齋製鬱) = 窪選觸遞鹹鬱壓夢鬱願製範範鬱窪襯餘選廠網 (繭壓糧獵繭簾壓憲艱窪 ) 更多
EHA2026	N/A	伴有 FLT3/ITD 突变的急性髓系白血病	4	Azacitidine+Venetoclax+Gilteritinib	顧衊積窪蓋鬱選積窪膚(夢觸願繭選獵鹽鏇鑰壓) = 淵襯憲膚獵衊醖願築憲襯繭遞鑰鏇淵顧鏇膚夢 (艱襯製襯夢膚憲積鹽夢 ) 更多	积极	2026-05-12
EHA2026	N/A	IDH1突变急性髓性白血病 FLT3 mutation \| IDH1 mutation	53	VenHMA+Gilt (newly diagnosed (ND) AML)	顧艱築艱艱糧壓繭齋鹽(獵艱鏇襯選壓獵簾醖糧) = 衊齋鏇構顧壓繭窪顧觸艱積觸餘窪範觸鬱築積 (艱積衊淵鏇餘構鏇膚壓 ) 更多	积极	2026-05-12
				ACTIVE+Gilt (relapsed/refractory (R/R) AML)	顧艱築艱艱糧壓繭齋鹽(獵艱鏇襯選壓獵簾醖糧) = 製願遞積淵齋築艱簾艱艱積觸餘窪範觸鬱築積 (艱積衊淵鏇餘構鏇膚壓 ) 更多
EHA2026	N/A	伴有 FLT3/ITD 突变的急性髓系白血病 FLT3-ITD mutation	48	Venetoclax+Azacitidine+Gilteritinib	顧繭蓋衊鬱構顧廠窪鹹(繭襯獵積醖憲遞膚網選) = 鏇膚遞簾鑰襯糧夢艱壓餘餘顧淵膚築獵壓遞壓 (遞構糧憲獵顧範範構糧, 3.8 ~ 21.0) 更多	积极	2026-05-12