A Phase 2, Open-label, Multicentre Study Investigating Tolerability and Efficacy of Gilteritinib in Combination With Fludarabine, Cytarabine and Idarubicin (FLAI) as Induction Therapy of Newly Diagnosed Non-M3 FLT3-positive Acute Myeloid Leukemia

The goal of this clinical trial is to evaluate the efficacy of gilteritinib as induction therapy in FLT3-positive adult acute myeloid leukemia patients. The main question it aims to answer is:
Is gilteritinib in combination to chemotherapy able to improve the complete remission rate of FLT3-positive AML?
Participants will receive up to 2 induction cycles with gilteritinib in combination with FLAI (fludarabine, cytarabine, idarubicine) and up to 3 consolidation cycles with gilteritinib and high-dose cytarabine.

开始日期2025-06-01

申办/合作机构

Gruppo Italiano Malattie EMatologiche dell'Adulto

NCT06696183

/ Not yet recruiting临床2期

Sequential Gilteritinib in Combination With Venetoclax and Azacitidine for Patients With Newly Diagnosed Acute Myeloid Leukemia (AML) and FLT3 Mutations Ineligible for Intensive Treatment

Explore the best tolerable and efficacious dose of Gilteritinib when combined with standard treatment with Venetoclax and Azacitidine in AML patients with FLT3 mutations which are ineligible for intensive chemotherapy

开始日期2025-04-01

申办/合作机构

Dresden University of Technology

ChiCTR2500098586

/ Suspended临床2期IIT

Clinical study on efficacy and safety of VA combined with Gilteritinib for treatment of de novo AML with NPM1mut/FLT3/ITDmut/DNMT3Amut

开始日期2025-03-15

申办/合作机构-

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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683

项与富马酸吉瑞替尼相关的文献（医药）

2025-06-01·BIOORGANIC CHEMISTRY

Inhibition of FLT3-induced signalling in refractory acute myeloid leukaemia

Article

作者: Yang, Jingmei ; Friedman, Ran

Mutations in FLT3 make this receptor tyrosine kinase overactive. Such mutations found in ∼30 % of the patients who suffer from acute myeloid leukaemia (AML). FLT3 mediates signalling networks that lead to cell proliferation and survival. FLT3 inhibitors are used to treat AML but patients who are treated with them typically become resistant. Such resistance often emerges through secondary mutations which either restore the activity of FLT3 in the presence of drugs or activate a key player in a signalling network such as NRAS. We had developed AML-specific cell lines resistant to two advanced FLT3 inhibitors: gilteritinib and FF-10101. Resistance in these cell lines proceeds though different mechanisms. In this study, we followed on the efficacy of five FLT3 inhibitors (gilteritinib, FF-10101 and three promising inhibitors that are being developed), two pan-PI3K inhibitors (one of which also inhibits mTOR) and two c-KIT inhibitors in order to examine the significance of different signalling cascades in FLT3⁺-AML. In addition, we used molecular modelling and quantum chemistry calculations to explain why specific FLT3 mutations affect some inhibitors more than others. Two novel FLT3 inhibitors were found to be only weakly affected by resistance mutations against gilteritinib and FF-10101. The efficacy of most FLT3 inhibitors was only weakly (or not at all) affected by the NRAS/G12C activating mutation. Finally, no non-FLT3 inhibitor has shown sufficient efficacy in the cells, suggesting the central role of FLT3 in FLT3-mutated AML.

2025-06-01·BIOCHEMICAL PHARMACOLOGY

Downregulation of DDIT4 levels with borneol attenuates hepatotoxicity induced by gilteritinib

Article

作者: Luo, Peihua ; Xu, Zhifei ; Yin, Yiming ; Yang, Xiaochun ; Yan, Hao ; Cao, Yashi ; He, Qiaojun ; Yang, Bo ; Zhou, Yourong

Gilteritinib, a multi-target kinase inhibitor, is currently used as standard therapy for acute myeloid leukemia. However, approximately half of the patients encounter liver-related adverse effects during the treatment with gilteritinib, which limiting its clinical applications. The underlying mechanisms of gilteritinib-induced hepatotoxicity and the development of strategies to prevent this toxicity are not well-reported. In our study, we utilized JC-1 dye, and MitoSOX to demonstrate that gilteritinib treatment leads to hepatocytes undergoing p53-mediated mitochondrial apoptosis. Furthermore, qRT-PCR analysis revealed that DNA damage-inducible transcript 4 (DDIT4), a downstream target of p53, was upregulated following gilteritinib administration and was identified as a key factor in gilteritinib-induced hepatotoxicity. After drug screening and western blot analysis, borneol, a bicyclic monoterpenoid, was found to decrease the protein level of DDIT4. This is the first compound found to downregulate DDIT4 levels and ameliorate hepatic injury caused by gilteritinib. Our findings suggest that high levels of DDIT4 are the primary driver behind gilteritinib-induced liver injury, and that borneol could potentially be a clinically safe and feasible therapeutic strategy by inhibiting DDIT4 levels.

2025-05-01·BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

Synthesis, molecular docking, and antiproliferative activity studies of bromine bearing Schiff bases

Article

作者: Akkoc, Senem ; Kokbudak, Zülbiye ; Karatas, Halis ; Turkmenoglu, Burçin

Bromine-bearing compounds are found in a variety of natural products and synthetic drugs and exhibit a variety of biological activities. They often display particular properties such as enhanced stability, improved lipophilicity, and larger molecular interactions, making them valuable in drug design and development. In this study, three bromine-bearing Schiff bases were synthesized, characterized, and tested in lung (A549) and colon (DLD-1) cancer cell lines for 72 h incubation time. The results demonstrated that compounds had antiproliferative activity in screened cell lines. The compounds were also tested on a healthy embryonic kidney cell line (HEK-293T) to evaluate their toxic effects on non-cancerous cells. In vitro results of compounds interacting with the most suitable target were confirmed by in silico approaches. All bromine-containing compounds were interacted with the receptor tyrosine kinase FLT3 target via molecular docking. From the interaction results of the 6JQR crystal structure with 2-Br, the docking score value was calculated as -8.178 kcal/mol, and this value was determined to be better than the docking score (-7.269 kcal/mol) value of gilteritinib. Pharmacokinetics, toxicity properties and Lipinski violations of the compounds were estimated by ADMET and were calculated to be in the range of recommended values. Molecules demonstrated limited efficacy against A549 cells line, however; they exhibited relatively greater effectiveness against DLD-1 cell line. Notably, the IC₅₀ value of 2-Br was comparable to that of cisplatin. All molecules exhibited minimal toxicity.

193

项与富马酸吉瑞替尼相关的新闻（医药）

2025-04-15

·GlobeNewswire-Health Care

ORYZON Announces First Patient Dosed in NCI-Sponsored Phase I/II Clinical Trial of Iadademstat Plus Immune Checkpoint Inhibitors in 1L Extensive Stage Small Cell Lung Cancer

In combination with atezolizumab or durvalumabStudy conducted under the CRADA agreement between NCI and Oryzon MADRID and CAMBRIDGE, Mass., April 15, 2025 (GLOBE NEWSWIRE) -- Oryzon Genomics, S.A. (ISIN Code: ES0167733015, ORY), a clinical-stage biopharmaceutical company leveraging epigenetics to develop therapies in diseases with strong unmet medical need, announced today that the first patient has been dosed in a Phase I/II trial of iadademstat, Oryzon’s potent and selective LSD1 inhibitor, in combination with immune checkpoint inhibitors (ICI) in first line small cell lung cancer (SCLC) patients with extensive disease, sponsored by the National Cancer Institute (NCI), part of the National Institutes of Health. This is the first clinical trial testing the combination of iadademstat with ICI. The trial (NCT06287775), titled “A Phase I Dose Finding and Phase II Randomized Trial of Iadademstat Combined With Immune Checkpoint Inhibition Maintenance After Initial Chemoimmunotherapy in Patients With Extensive-Stage Small Cell Lung Cancer”, will evaluate the safety, tolerability, dose finding and efficacy of iadademstat in combination with an ICI, either atezolizumab or durvalumab, in patients with extensive-stage SCLC who have initially received standard of care chemotherapy and immunotherapy. This Phase I/II clinical study is conducted and sponsored by the NCI, with Dr. Charles Rudin, from the Memorial Sloan Kettering Cancer Center (MSKCC) as the Principal Investigator. The trial will be conducted at a number of prestigious cancer centers in the US, including the MSKCC, the JHU Sidney Kimmel Comprehensive Cancer Center and many others. The trial plans to enroll 45-50 patients and will be carried out under a Cooperative Research and Development Agreement (CRADA) that Oryzon has in place with the NCI. Dr. Carlos Buesa, Oryzon’s CEO, stated: “We are very excited to have the first patient dosed in this study. The biology underlying iadademstat’s ability to make SCLC cells visible to the immune system and to boost significantly the number and the activation of cytotoxic CD8+ T cells is fascinating. Moreover, prior analyses of several Phase III trials in SCLC indicate that low LSD1 expression is a key factor in determining patients' likelihood of responding and surviving. If this trial confirms the preliminary findings reported by researchers at MSKCC and others, it would reinforce the rationale for this combination as a therapeutic option for this critically underserved patient population. In the event of positive results, the company could also initiate a complementary trial to generate additional data in support of a potential accelerated registration strategy.” About OryzonFounded in 2000 in Barcelona, Spain, Oryzon (ISIN Code: ES0167733015) is a clinical stage biopharmaceutical company and the European leader in epigenetics, with a strong focus on personalized medicine in CNS disorders and oncology. Oryzon’s team is composed of highly qualified professionals from the pharma industry located in Barcelona, Boston, and San Diego. Oryzon has an advanced clinical portfolio with two LSD1 inhibitors, vafidemstat in CNS (Phase III-ready) and iadademstat in oncology (Phase II). The company has other pipeline assets directed against other epigenetic targets like HDAC-6 where a clinical candidate ORY-4001, has been nominated for its possible development in CMT and ALS. In addition, Oryzon has a strong platform for biomarker identification and target validation for a variety of malignant and neurological diseases. For more information, visit www.oryzon.com About IadademstatIadademstat (ORY-1001) is a small oral molecule, which acts as a highly selective inhibitor of the epigenetic enzyme LSD1 and has a powerful differentiating effect in hematologic cancers (see Maes et al., Cancer Cell 2018 Mar 12; 33 (3): 495-511.e12.doi: 10.1016 / j.ccell.2018.02.002.). A FiM Phase I/IIa clinical trial with iadademstat in R/R AML patients demonstrated the safety and good tolerability of the drug and preliminary signs of antileukemic activity, including a CRi (see Salamero et al, J Clin Oncol, 2020, 38(36): 4260-4273. doi: 10.1200/JCO.19.03250). Iadademstat has shown encouraging safety and strong clinical activity in combination with azacitidine in a Phase IIa trial in elder 1L AML patients (ALICE trial) (see Salamero et al., ASH 2022 oral presentation & The Lancet Haematology, 2024, 11(7):e487-e498). Iadademstat is currently being evaluated in combination with gilteritinib in the ongoing Phase Ib FRIDA trial in patients with relapsed/refractory AML with FLT3 mutations, and in combination with azacitidine and venetoclax in 1L AML in an investigator-initiated study led by OHSU and in a trial sponsored by the U.S. National Cancer Institute (NCI) under the Cooperative Research and Development Agreement (CRADA) signed between Oryzon and the NCI to collaborate on further clinical development of iadademstat in different types of hematologic and solid cancers. Beyond hematological cancers, the inhibition of LSD1 has been proposed as a valid therapeutic approach in some solid tumors such as small cell lung cancer (SCLC), neuroendocrine tumors (NET), medulloblastoma and others. In a Phase IIa trial in combination with platinum/etoposide in second line ED-SCLC patients (CLEPSIDRA trial), preliminary activity and safety results have been reported (see Navarro et al., ESMO 2018 poster). Iadademstat is in a collaborative Phase II trial with the Fox Chase Cancer Center (FCCC) in combination with paclitaxel in R/R neuroendocrine carcinomas, and in a Phase I/II randomized trial in 1L ED-SCLC in combination with ICI sponsored by NCI and led by the Memorial Sloan Kettering Cancer Center. Oryzon is further expanding the clinical development of iadademstat through additional investigator-initiated studies. Iadademstat has orphan drug designation for SCLC in the US and for AML in the US and EU. 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2期免疫疗法孤儿药临床1期临床3期

2025-04-08

·PRNewswire

TIBSOVO's Continued Success Solidifies Its Position as a Leader in IDH1 Mutant Space | DelveInsight

With its ability to specifically inhibit the mutant IDH1 enzyme, TIBSOVO addresses an unmet need in personalized cancer treatment, offering new hope for patients with limited options. As clinical evidence continues to support its efficacy and safety, TIBSOVO is positioned for strong growth in the oncology market, especially with expanding indications and global market penetration. LAS VEGAS, April 8, 2025 /PRNewswire/ -- DelveInsight's " TIBSOVO Market Size, Forecast, and Market Insight Report" highlights the details around TIBSOVO, the first and only targeted therapy approved for patients with previously treated IDH1-mutated cholangiocarcinoma. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of TIBSOVO. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Servier's TIBSOVO (ivosidenib) Overview TIBSOVO (ivosidenib) is a small molecule developed by Servier, acts as an isocitrate dehydrogenase-1 (IDH1) inhibitor. It is indicated for patients with a susceptible IDH1 mutation, which is identified through an FDA-approved test. Currently, TIBSOVO is approved for the treatment of newly diagnosed acute myeloid leukemia (AML), relapsed or refractory AML, relapsed or refractory myelodysplastic syndromes (MDS), and locally advanced or metastatic cholangiocarcinoma. According to Servier's pipeline, TIBSOVO is also being actively investigated for a range of additional indications, with key areas of exploration including solid tumors and hematological malignancies. Learn more about TIBSOVO projected market size for AML, cholangiocarcinoma, and MDS @ TIBSOVO Market Potential The oncology market has experienced significant growth in recent years, driven by advances in cancer research, the development of targeted therapies, and the increasing global prevalence of cancer. As the second-leading cause of death worldwide, cancer continues to pose a major public health challenge, creating a substantial demand for innovative treatments. The market is marked by a shift toward personalized medicine, where therapies are tailored to an individual's genetic profile, allowing for more effective and less toxic treatments. Immunotherapies, such as checkpoint inhibitors, CAR-T cell therapies, and oncolytic virus therapies, are at the forefront of these advancements, offering new hope to patients with previously untreatable cancers. Moreover, the oncology market is expanding due to the growing adoption of precision oncology and molecular diagnostics, which enable earlier and more accurate detection of cancer. This has led to increased investments in research and development, especially in areas like liquid biopsies, companion diagnostics, and next-generation sequencing. The demand for more effective and accessible cancer treatments is fueling innovation in both the pharmaceutical and biotechnology sectors, with numerous new drugs and therapies entering clinical trials and moving toward commercialization. As a result, the oncology market is poised to continue growing, with significant opportunities for breakthrough therapies in immuno-oncology, targeted treatments, and gene therapies. DelveInsight has expertise in the oncology market with an experienced team handling the oncology domain proficiently. DelveInsight has released a series of epidemiology-based market reports on Acute Myeloid Leukemia, Cholangiocarcinoma, and Myelodysplastic Syndrome. These reports include a comprehensive understanding of current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Gain deeper insights into these diseases with a customized report tailored to your needs. Connect with us today—our experts are ready to assist you! Emerging Competitors of TIBSOVO TIBSOVO faces significant competition in the newly diagnosed AML market from several alternative treatments. Oral therapies like VENCLEXTA (venetoclax), which inhibits BCL-2, and DAURISMO (glasdegib), which targets the hedgehog pathway, present strong contenders. Additionally, emerging therapies such as Lisaftoclax (Ascentage Pharma), another BCL-2 inhibitor, and gilteritinib (Astellas), a kinase inhibitor, are expected to further challenge TIBSOVO in the newly diagnosed AML space. In the R/R AML market, TIBSOVO competes with several therapies, including XOSPATA (gilteritinib), IDHIFA (enasidenib), and others. Emerging therapies such as Ziftomenib (Kura Oncology) and RVU-120 (Ryvu Therapeutics) are also expected to compete with TIBSOVO upon their approval. Ziftomenib, a small molecule developed by Kura Oncology, is currently in Phase I/II trials. In the relapsed/refractory (R/R) MDS market, TIBSOVO faces competition from therapies such as RYTELO (Imetelstat), an IV injection that inhibits oligonucleotide telomerase, and REBLOZYL (Luspatercept), a subcutaneous injection, among other treatment options. In the metastatic cholangiocarcinoma market, TIBSOVO faces relatively less competition, as there are few approved therapies. One such competitor is PEMAZYRE (pemigatinib), a kinase inhibitor that was approved in the US in 2020 and in Europe in 2021. Whereas, Tyra Biosciences' TYRA-200, an FGFR1/2/3 inhibitor, is in phase I stage of development that might be a threat to TIBSOVO one approved. To know more about the number of competing drugs in development, visit @ TIBSOVO Market Positioning Compared to Other Drugs Key Developmental Activities of TIBSOVO In October 2023, Servier announced that the FDA has approved TIBSOVO (ivosidenib tablets) for the treatment of IDH1-mutated R/R MDS. In May 2023, Servier announced that the European Commission has approved TIBSOVO as a targeted therapy in two indications: in combination with azacitidine for the treatment of adult patients with newly diagnosed AML with an isocitrate dehydrogenase-1 R132 mutation who are not eligible to receive standard induction chemotherapy; as well as in monotherapy for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma with an IDH1 R132 mutation who were previously treated by at least one prior line of systemic therapy. In May 2022, Servier announced FDA approval of TIBSOVO (ivosidenib tablets) in combination with azacitidine for patients with newly diagnosed IDH1-mutated acute myeloid leukemia. In August 2021, Servier announced FDA approval for TIBSOVO (ivosidenib tablets) for the treatment of adult patients with previously treated, locally advanced, or metastatic cholangiocarcinoma with an IDH1 mutation as detected by an FDA-approved test. TIBSOVO is the first and only targeted therapy approved for patients with previously treated IDH1-mutated cholangiocarcinoma. In May 2019, Agios announced FDA approval of TIBSOVO (ivosidenib tablets) as monotherapy for newly diagnosed adult patients with IDH1 mutant acute myeloid leukemia not eligible for intensive chemotherapy. In July 2018, the FDA approved TIBSOVO (ivosidenib) for relapsed or refractory acute myeloid leukemia with an idh1 mutation. In February 2018, the FDA accepted a new drug application and granted priority review for ivosidenib in relapsed or refractory AML with an IDH1 mutation. In December 2017, Agios submitted an NDA to the FDA for ivosidenib for the treatment of patients with relapsed/refractory aml and an IDH1 mutation. TIBSOVO Patent Details TIBSOVO is a drug owned by Servier Pharmaceuticals. Currently, it is protected by 10 Orange book-listed patents filed from 2018 to 2023, out of which none have expired yet. It is also protected by ODE exclusivity for various indications. Discover how TIBSOVO is shaping the blood cancer treatment landscape @ TIBSOVO Side Effects TIBSOVO Market Dynamics In recent years, the therapeutic landscape has undergone significant changes, with promising approaches including immunotherapy, chemotherapy combined with targeted treatments, and the regulation of immune checkpoint-mediated signaling pathways. One such treatment, Ivosidenib, works by inducing the bone marrow to develop normal blood cells, helping to reduce the frequency of blood transfusions. Investment in the research and development of drugs has surged, contributing to a rich pipeline and forecasting promising opportunities in the treatment markets for AML and cholangiocarcinomas. Therapies like TIBSOVO have the potential to capture market attention due to their efficacy and innovation. As healthcare spending continues to rise globally, the pharmaceutical market is expected to expand, providing greater opportunities for market penetration by manufacturers and facilitating the development and distribution of new treatments. Despite progress in cancer treatment, significant gaps remain in understanding the full process that governs carcinogenesis and resistance to treatments, particularly in cancers like acute myeloid leukemia (AML). This highlights the need for more advanced research to address these challenges. Additionally, the limited biomarker testing for patients with the IDH1 mutation restricts the targeted patient pool, posing a challenge for therapies like TIBSOVO to achieve their expected market share. The increasing competition from emerging therapies, such as KEYTRUDA (pembrolizumab), IMFINZI (durvalumab), and the combination of Azacitidine + Venetoclax, further intensifies the pressure on TIBSOVO. Moreover, the difficulty in obtaining sufficient patient pools and diagnostic samples during disease recurrence presents significant hurdles to the effective development of translational research, potentially slowing progress in optimizing treatments. Dive deeper to get more insight into TIBSOVO's strengths & weaknesses relative to competitors @ TIBSOVO Market Drug Report Table of Contents Related Reports Acute Myeloid Leukemia Market Acute Myeloid Leukemia Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key AML companies including Agios Pharmaceuticals, Abbvie, Jazz Pharmaceuticals, Astellas Pharma, Pfizer, Novartis Oncology, Daiichi Sankyo, AstraZeneca, Astex Pharmaceuticals, Inc., Chimerix, Takeda, Rafael Pharmaceuticals Inc., Delta-Fly Pharma, GlycoMimetics Incorporated, BerGenBio ASA, MacroGenics, Syndax Pharmaceuticals, Arog Pharmaceuticals, Forma Therapeutics, Sellas Life Sciences Group, Actinium Pharmaceuticals, Clear Creek Bio Inc., CellCentric Ltd., Biosight Ltd., Astex Pharmaceuticals, Inc., Curis, Inc., NexImmune Inc., Immunomedics, Inc., Sumitomo Dainippon Pharma Co., Ltd., Incyte Corporation, Aprea Therapeutics, Immunicum, DCPrime BV, GT Biopharma, Inc., Hanmi Pharmaceutical Company Limited, Cardiff Oncology, Bio-Path Holdings, Actinium Pharmaceuticals, Aptevo Therapeutics, Synimmune GmbH, GEMoaB Monoclonals, ImmunoGen, Inc., AGC Biologics S.p.A., Precigen, Inc., Novartis Pharmaceuticals, Xencor, Inc., Novartis, Celgene, Bristol Myers Squibb, among others. Relapsed/Refractory Acute Myeloid Leukemia Market Relapsed/Refractory Acute Myeloid Leukemia Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key R/R AML companies including Agios Pharmaceuticals, Abbvie, Jazz Pharmaceuticals, Astellas Pharma, Pfizer, Novartis Oncology, Daiichi Sankyo, AstraZeneca, Astex Pharmaceuticals, Inc., Chimerix, Takeda, Rafael Pharmaceuticals Inc., Delta-Fly Pharma, GlycoMimetics Incorporated, BerGenBio ASA, MacroGenics, Syndax Pharmaceuticals, Arog Pharmaceuticals, Forma Therapeutics, Sellas Life Sciences Group, Actinium Pharmaceuticals, Clear Creek Bio Inc., CellCentric Ltd., Biosight Ltd., Astex Pharmaceuticals, Inc., Curis, Inc., NexImmune Inc., Immunomedics, Inc., Sumitomo Dainippon Pharma Co., Ltd., Incyte Corporation, Aprea Therapeutics, Immunicum, DCPrime BV, GT Biopharma, Inc., Hanmi Pharmaceutical Company Limited, Cardiff Oncology, Bio-Path Holdings, Actinium Pharmaceuticals, Aptevo Therapeutics, Synimmune GmbH, GEMoaB Monoclonals, ImmunoGen, Inc., AGC Biologics S.p.A., Precigen, Inc., Novartis Pharmaceuticals, Xencor, Inc., Novartis, Celgene, Bristol Myers Squibb, among others. Myelodysplastic Syndrome Market Myelodysplastic Syndrome Market Insight, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of market trends, market drivers, market barriers, and key MDS companies such as Bristol-Myers Squibb, Astex Pharmaceutical, Taiho Oncology, Fibrogen, AbbVie, Gilead Sciences, Novartis, Syros Pharmaceuticals, Takeda, Pfizer, Geron Corporation, Karyopharm Therapeutics, Antengene Corporation, BerGenBio ASA, Jazz Pharmaceuticals, Aprea Therapeutics, Sanofi, Medac, among others. Cholangiocarcinoma Market Cholangiocarcinoma Market Insight, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of market trends, market drivers, market barriers, and key cholangiocarcinoma companies such as AstraZeneca, Decalth Systems, Basilea Pharmaceutica, Taiho Oncology, Eisai Pharmaceuticals, TransThera Sciences, Incyte Corporation, Roche, Agios Pharmaceuticals, Servier Pharma, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

上市批准免疫疗法临床1期优先审批

2025-04-08

·米内网

东阳光药厉害了！独家品种大涨120%，7款1类新药上市可期，8大爆品抢攻200亿市场

精彩内容今年以来，东阳光药新药研发进展不断：1类新药磷酸萘坦司韦胶囊、艾考磷布韦片首次获批上市，3.3类新药德谷胰岛素注射液提交NDA，1类新药HEC-007注射液首次申报IND等。创新研发已成为东阳光药发展主旋律，2024年公司研发投入6.87亿元，目前有53款新药（37款1类新药）处于申请临床及以上阶段，其中有12款新药（7款1类新药）上市可期。超100%！胰岛素、1类新药收入大涨3月28日，港股上市药企东阳光长江药业发布2024年业绩报告，报告期内取得营业额约37.24亿元，公司权益股东应占溢利约4.83亿元，每股基本盈利0.55元。从分领域收入看，抗病毒药为东阳光药核心板块，2024年收入25.85亿元，占总收入比重接近70%；内分泌及代谢药收入2.49亿元，同比增长51.67%，占总收入比重约6.7%；心血管药物收入2.7亿元，同比增长46.78%，占总收入比重约7.3%。2024年东阳光药各药物类别收入情况（单位：亿元）来源：公司公告，米内网整理米内网数据显示，近年来中国三大终端六大市场（统计范围详见本文末）抗病毒化药一级集团排名中，东阳光药稳居首位。可威（磷酸奥司他韦颗粒/胶囊）为东阳光药核心产品，2024年流感疫情相较2023年同期有较大幅度下降，该产品受到一定影响，但依然保持市场领先的地位。集团通过持续开展学术推广活动、深化“可威”品牌建设，制定了一系列销售策略，不断提升可威的品牌认知度以及市场份额。胰岛素系列产品方面，东阳光药有5款产品获批上市并中标国家胰岛素专项集采。集团通过持续强化专业推广队伍，推进明星标杆终端准入，深挖基层市场，优化各级销售渠道，胰岛素系列产品步入收获期，2024年实现收入136.53百万元，同比大幅增长101.14%。新药方面，东阳光药首款1类创新药磷酸依米他韦胶囊（NS5A蛋白抑制剂）于2020年底获批上市，用于治疗成人基因1型非肝硬化慢性丙型肝炎患者。该产品已通过谈判纳入国家医保乙类目录，并在市场上取得优异的表现，2024年实现收入89.49百万元，同比大幅增长120.06%。创新转型已成为东阳光药的发展主旋律。近年来，公司研发投入均以两位数的增速增长，2024年达6.87亿元，占总收入比重突破10%至18.46%，反映了公司对研发创新和未来市场布局的重视。2022-2024年东阳光药研发投入情况（单位：亿元）来源：公司公告，米内网整理目前东阳光药已有磷酸依米他韦胶囊、磷酸萘坦司韦胶囊、艾考磷布韦片3款1类新药获批上市，为国内丙肝患者带来用药新选择；此外，5款胰岛素获批上市，包括重组人胰岛素注射液、甘精胰岛素注射液、门冬胰岛素注射液、门冬胰岛素30注射液及精蛋白人胰岛素混合注射液(30R)，涵盖第二代及第三代胰岛素。37款1类新药在路上，7款上市可期今年以来，东阳光药1类新药研发进展不断：磷酸萘坦司韦胶囊、艾考磷布韦片获批上市，均为抗丙肝病毒药物；此外，HEC-007注射液首次申报IND等。目前东阳光药在国内有37款1类新药（不含已获批/主动撤回品种，下同）处于申请临床及以上阶段，其中有7款已处于NDA或III期（含II/III期）临床阶段，上市可期。东阳光药国内在研的1类新药来源：米内网综合数据库从治疗领域看，东阳光药在研1类新药集中在抗病毒药物、糖尿病用药、抗肿瘤药等。米内网数据显示，2023年中国三大终端六大市场抗病毒药物（化学药+生物药）、糖尿病用药（化学药+生物药）、抗肿瘤药（化学药+生物药）销售额分别超过270亿元、610亿元、1400亿元。在抗病毒领域，东阳光药对丙肝药物进行了全方位布局，涵盖NS5A、NS5B、NS3/4A等有效靶点，其中磷酸依米他韦胶囊、磷酸萘坦司韦胶囊、艾考磷布韦片已获批上市，伏拉瑞韦胶囊处于III期临床；乙肝药物方面，甲磺酸莫非赛定处于III期临床，为国内研发速度最快的抗乙型肝炎病毒衣壳抑制剂。在糖尿病领域，焦谷氨酸荣格列净胶囊（SGLT2）于2024年1月提交NDA获受理，目前还在审评审批中。国内已有多款同靶点国产新药获批，包括恒瑞医药的恒格列净、惠升生物的加格列净等，近年来中国三大终端六大市场SGLT2抑制剂销售额均以两位数的增速增长，2023年超过66亿元。此外，HEC88473注射液处于II期临床，为全球研发进展最快的GLP-1/FGF21双重激动剂之一。在抗肿瘤领域，东阳光药布局了非小细胞肺癌、甲状腺癌、血液肿瘤等细分病种，涵盖FLT3、RET、PI3Kδ、MTOR、c-Met等热门靶点。甲磺酸莱洛替尼胶囊、苯磺酸克立福替尼片均处于III期临床，其中苯磺酸克立福替尼片是国产首款进入临床III期的新一代、高选择性的FMS样酪氨酸激酶3(FLT3)抑制剂，用于初治的成人急性髓系白血病(AML)。米内网数据显示，国内已上市靶向FLT3的药物中，仅吉瑞替尼为第二代FLT3抑制剂，舒尼替尼、索拉非尼、尼达尼布等均为非特异性的多靶点抑制剂，单药治疗AML时疗效并不显著。猛攻400亿市场！50亿大品种新剂型冲线在即除了1类新药，东阳光药还有多款改良型新药、生物类似药在研。改良型新药聚焦剂型改良，目前有8款新药处于申请临床及以上阶段，涉及高血压、消化性溃疡出血、抑郁症、阿尔茨海默病、癫痫、精神分裂症等适应症。东阳光药国内在研的改良型新药来源：米内网综合数据库2024年11月，东阳光药提交2.2类改良型新药苯磺酸氨氯地平颗粒的上市申请并获受理，为国内首家。目前国内已获批上市的氨氯地平剂型有片剂、分散片、胶囊剂、滴丸剂、干混悬剂等，暂无颗粒剂获批上市。米内网数据显示，近年来中国三大终端六大市场氨氯地平销售额均超过50亿元。生物类似药聚焦胰岛素及单抗，目前有8款新药处于获批临床及以上阶段，集中在糖尿病、肿瘤2大适应症。东阳光药国内在研的生物类似药来源：米内网综合数据库胰岛素步入收获期，5款已获批上市，德谷胰岛素注射液于2025年2月提交NDA获受理，德谷门冬双胰岛素注射液处于III期临床，德谷胰岛素利拉鲁肽注射液获批临床，上述3款产品2023年在中国三大终端六大市场销售额分别超过8亿元、13亿元、3亿元。在具有明显临床优效性的GLP-1RA方面，利拉鲁肽注射液步入III期临床，原研产品2023年在中国三大终端六大市场销售额超过18亿元；14028注射液（度拉糖肽生物类似物）处于I期临床，国内暂无度拉糖肽生物类似药获批，原研产品2023年在中国三大终端六大市场销售额超过13亿元。抗体类生物类似药方面，东阳光药布局了重组抗PD-1人源化单抗注射液（纳武利尤单抗）、贝伐珠单抗注射液、重组抗TNF-α全人源单抗注射液（阿达木单抗）。米内网数据显示，2023年中国三大终端六大市场纳武利尤单抗、贝伐珠单抗、阿达木单抗销售额分别超过15亿元、107亿元、22亿元。资料来源：米内网数据库、公司公告等注：米内网《中国三大终端六大市场药品竞争格局》，统计范围是：城市公立医院和县级公立医院、城市社区中心和乡镇卫生院、城市实体药店和网上药店，不含民营医院、私人诊所、村卫生室，不含县乡村药店；上述销售额以产品在终端的平均零售价计算。数据统计截至4月7日，如有疏漏，欢迎指正！免责声明：本文仅作医药信息传播分享，并不构成投资或决策建议。本文为原创稿件，转载文章或引用数据请注明来源和作者，否则将追究侵权责任。投稿及报料请发邮件到872470254@qq.com稿件要求详询米内微信首页菜单栏商务及内容合作可联系QQ：412539092【分享、点赞、在看】点一点不失联哦

上市批准临床申请申请上市

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
-	-	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

分期

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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


NCT03182244 (COMMODORE, Pubmed \| Ann Hematol)	临床3期	急性髓性白血病 FLT3-mutated	-	Gilteritinib	夢鬱鹹淵醖衊觸艱衊廠(築鹽網鏇鹽廠鏇築鹽壓) = numerically higher in the gilteritinib versus SC arm across all three regions 窪餘憲膚網齋廠鹹獵襯 (觸憲網築網艱糧製夢艱 ) 更多	积极	2025-03-10
				Salvage Chemotherapy
NCT03836209 (Precog 0905, CTgov)	临床2期	伴有 FLT3/ITD 突变的急性髓系白血病	181	Daunorubicin+Cytarabine+Gilteritinib (Arm A)	鹽選糧蓋窪顧齋積遞鬱 = 鹹鬱齋襯膚遞窪範壓觸糧鹽鹽築鑰鑰構醖顧範 (艱餘構鑰壓襯夢願衊製, 鑰觸艱製繭構齋構鹹繭 ~ 繭鬱製餘膚網襯壓顧醖) 更多	-	2025-02-28
				Midostaurin+Daunorubicin+Cytarabine (Arm B)	鹽選糧蓋窪顧齋積遞鬱 = 築鏇繭壓範衊壓繭築獵糧鹽鹽築鑰鑰構醖顧範 (艱餘構鑰壓襯夢願衊製, 窪簾蓋獵夢鏇築鹽窪夢 ~ 憲糧構夢鬱衊衊鹹齋膚) 更多
NCT03182244 (COMMODORE, CTgov)	临床3期	伴有 FLT3/ITD 突变的急性髓系白血病 \| 难治性急性髓细胞白血病	276	Gilteritinib (Gilteritinib)	蓋襯糧鑰遞鏇繭廠遞廠(淵膚醖襯餘簾構築鹹鹹) = 齋鹽淵壓觸製餘構鹹憲顧窪襯鬱蓋糧鏇積鹹廠 (艱顧襯遞網獵鹽選顧鑰, 餘觸夢壓憲餘鹽獵製遞 ~ 網壓選糧鬱憲鹽齋觸衊) 更多	-	2025-01-14
				Etoposide+Fludarabine+Mitoxantrone+Cytarabine+G-CSF (Salvage Chemotherapy)	蓋襯糧鑰遞鏇繭廠遞廠(淵膚醖襯餘簾構築鹹鹹) = 鏇夢醖淵壓鹹壓鬱網簾顧窪襯鬱蓋糧鏇積鹹廠 (艱顧襯遞網獵鹽選顧鑰, 選顧顧夢壓製蓋憲鏇艱 ~ 壓憲築夢選鬱窪積壓築) 更多
NCT05010772 (4277, ASH2024)	临床1期	急性髓性白血病 complex cytogenetics \| antecedent MDS/MPN \| therapy-related AML ... 更多	31	ASTX727 alone	鬱壓願積齋鏇衊餘艱鹽(壓衊顧網選繭觸鏇積壓) = 選遞範窪餘構夢網鑰鹹遞鑰願鹽範襯襯鏇壓膚 (餘繭鹽網鏇醖蓋選齋鏇 ) 更多	积极	2024-12-09
				ASTX727 plus venetoclax	鬱壓願積齋鏇衊餘艱鹽(壓衊顧網選繭觸鏇積壓) = 憲壓窪蓋餘築鑰願鬱壓遞鑰願鹽範襯襯鏇壓膚 (餘繭鹽網鏇醖蓋選齋鏇 ) 更多
ASH2024	N/A	-	-	Gilteritinib (GILT) monotherapy	糧蓋鏇醖廠鹹餘膚衊獵(網鹹鏇壓淵構選鬱觸願) = 蓋選襯顧積繭蓋鏇選鬱鏇鑰襯憲築繭鹽艱蓋選 (淵觸選醖餘簾選鏇築遞 ) 更多	-	2024-12-08
				Salvage chemotherapy	糧蓋鏇醖廠鹹餘膚衊獵(網鹹鏇壓淵構選鬱觸願) = 餘觸製夢鹹壓憲鏇窪構鏇鑰襯憲築繭鹽艱蓋選 (淵觸選醖餘簾選鏇築遞 ) 更多
NCT03836209 (Precog 0905, ASH2024) 人工标引	临床2期	急性髓性白血病一线 FLT3 Mutation	177	Gilteritinib	壓鏇餘廠製鹽襯廠簾願(選鹽築選選製蓋膚獵網) = 廠膚構鏇顧鹹獵憲製糧簾網製築遞壓壓膚廠餘 (製齋製簾壓廠願遞衊簾 ) 更多	积极	2024-12-07
				Midostaurin	壓鏇餘廠製鹽襯廠簾願(選鹽築選選製蓋膚獵網) = 網衊鹽糧觸憲鹹憲鏇衊簾網製築遞壓壓膚廠餘 (製齋製簾壓廠願遞衊簾 ) 更多
NCT02310321 (CTgov)	临床1/2期	伴有 FLT3/ITD 突变的急性髓系白血病	97	Cytarabine+Idarubicin+Gilteritinib (Phase 1: Dose Evaluation (DEv))	簾範壓膚鬱餘窪夢淵夢 = 願顧鏇淵網鹽構艱鹹淵窪簾願遞夢淵夢築鹹選 (鹹繭蓋選製築築觸積衊, 願艱鏇衊餘鏇顧淵積顧 ~ 選淵繭繭窪夢範鑰簾積) 更多	-	2024-10-16
				Cytarabine+Idarubicin+Gilteritinib (Phase 1: Dose Expansion (DEx))	憲獵蓋壓鏇憲積積鹽齋 = 積廠膚鬱範齋範獵範願築窪淵糧網廠築淵膚鑰 (製鬱積遞艱鏇醖製淵蓋, 衊鹽廠糧遞鏇齋鬱遞蓋 ~ 衊憲獵壓艱積築餘範鏇) 更多
NCT02997202 (ADMIRAL, CTgov)	临床3期	伴有 FLT3/ITD 突变的急性髓系白血病	356	gilteritinib (Gilteritinib)	淵廠蓋選觸觸膚艱醖觸(鹽選窪觸醖憲齋顧積齋) = 餘鹽觸夢鬱範齋鑰淵夢衊顧獵鹹醖觸夢鏇膚簾 (選鑰獵顧鹹衊壓繭襯遞, 製鏇觸醖鏇積衊醖夢窪 ~ 顧繭網窪製鏇醖觸製窪) 更多	-	2024-09-19
				Placebo (Placebo)	淵廠蓋選觸觸膚艱醖觸(鹽選窪觸醖憲齋顧積齋) = 鏇窪網願網廠壓齋顧壓衊顧獵鹹醖觸夢鏇膚簾 (選鑰獵顧鹹衊壓繭襯遞, 餘襯選繭膚廠壓獵顧蓋 ~ 鹽衊衊廠顧鏇選衊齋構) 更多
SOHO2024	N/A	急性髓性白血病	-	Gilteritinib monotherapy	簾窪鹽廠願觸憲構夢製(壓艱鹽鬱廠鹽鏇壓糧積) = 構鏇蓋構築鏇淵鹹齋蓋製獵糧鑰襯壓選選壓壓 (齋壓鹽淵網選鹽範齋窪 ) 更多	-	2024-09-04
				Gilteritinib + Venetoclax	簾窪鹽廠願觸憲構夢製(壓艱鹽鬱廠鹽鏇壓糧積) = 醖艱蓋糧夢襯壓鏇蓋醖製獵糧鑰襯壓選選壓壓 (齋壓鹽淵網選鹽範齋窪 ) 更多
NCT03182244 (COMMODORE, EHA2024) 人工标引	临床3期	伴有 FLT3/ITD 突变的急性髓系白血病二线 FLT3 Mutation	234	Gilteritinib 120 mg/day	顧構廠遞顧醖遞鏇襯積(糧襯繭觸衊製鏇壓鹽糧) = 遞鏇繭簾構衊積鹽築淵蓋夢齋憲鹽網鏇憲簾艱 (蓋齋糧鹽鏇獵夢願壓餘 ) 更多	积极	2024-05-14
				Salvage Chemotherapy (SC)	顧構廠遞顧醖遞鏇襯積(糧襯繭觸衊製鏇壓鹽糧) = 製鹽衊鹽襯艱築鏇願鬱蓋夢齋憲鹽網鏇憲簾艱 (蓋齋糧鹽鏇獵夢願壓餘 ) 更多