A Prospective, One-arm and Open Clinical Study to Assess Efficacy and Safety of Avatrombopag in the Treatment of Pediatric Primary Immune Thrombocytopenia

To evaluate the safety and efficacy of avatrombopag in the treatment of pediatric primary immune thrombocytopenia in patients who have been treated with eltrombopag before and switched to avatrobopag because of poor efficacy, excessive platelet fluctuation or intolerance, or patient preference, economic reasons, and other reasons.

开始日期2024-03-01

申办/合作机构

天津医科大学第二医院

[+1]

NCT06254287 / Not yet recruiting临床2期IIT

Efficacy and Safety of Avatrombopag vs. Avatrombopag Combined With rhTPO in the Treatment of Patients With Severe Aplastic Anemia

Title: Efficacy and safety of Avatrombopag VS Avatrombopag combined with rhTPO in patients with severe aplastic anemia: a single-center, controlled study Observation group: Patients with severe aplastic anemia who did not respond to initial treatment (unconditional HSCT or ATG) or other treatments (except HSCT) Objective: To evaluate the efficacy and safety of Avatrombopag and rhTPO in the treatment of patients with severe aplastic anemia, to provide more treatment options for patients with severe aplastic anemia who are unable to undergo transplantation /ATG or have failed previous treatment, and to provide evidence-based evidence for the use of Avatrombopag or combined with rhTPO to promote hematopoietic recovery Experimental design: Single center, controlled study Total number of cases: 30 cases/group, 2 groups
Case selection criteria:
Inclusion criteria:
1. Age: > 18 years old, gender is not limited;
2. Patients clinically diagnosed with severe aplastic anemia (diagnostic criteria: ① myelocyte hyperplasia < 25% of normal; If ≥ 25% of normal but < 50%, the remaining hematopoietic cells should be < 30%. ② Blood routine must have two of the following three items: ANC < 0.5×109 /L; The absolute value of reticulocyte was < 20×109 /L; PLT < 20×109 /L). ③ If ANC < 0.2×109 /L, the diagnosis is very severe aplastic anemia), including patients who are newly diagnosed or have failed other treatments;
3. Patients currently undergoing hematopoietic stem cell transplantation or ATG without conditions;
4. Eastern Cancer Collaboration Group (ECOG) score 0-2;
5. Informed consent must be signed before participating in the study.
Exclusion criteria:
One of the following circumstances is not eligible for inclusion:
1. Patients with severe bleeding and/or infection that cannot be controlled after standard treatment;
2. Diagnosis of congenital hematopoietic failure (such as Fanconi anemia, congenital dyskeratosis, etc.);
3. Other causes of pancytopenia and bone marrow hypoproliferative diseases (such as hemolytic PNH, hypoproliferative MDS/AML, autoantibody-mediated pancytopenia, etc.);
4. All laboratory or clinically confirmed HIV infection, hepatitis C infection, chronic hepatitis B infection, or evidence of active hepatitis during screening;
5. Cytogenetic evidence of bone marrow abnormalities in clonal blood diseases;
6. History of thromboembolism or current use of anticoagulants within the past 6 months;
7. Accompanied by any one or more malignant diseases;
8. Treatment with another investigational agent within 30 days prior to the first dose of Avatrombopag;
9. Patients who cannot understand or are unwilling to sign an informed consent form (ICF);
10. Pregnant or lactating women;
11. The female patient or the female spouse of the male patient is unable to take effective contraceptive measures;
12. The Investigator considers that there are any other circumstances that may cause the subject to be unable to complete the study or that pose a significant risk to the subject.
Exit criteria:
1. The subject or his legal guardian voluntarily requests withdrawal;
2. Violation of inclusion/exclusion criteria;
3. Poor medication compliance;
4. The subject's condition requires treatment with drugs prohibited by the study;
5. Adverse events occur that cause subjects to be unable to continue the study;
6. Other unexplained severe comorbidities;
7. Pregnancy occurs during treatment;
8. Subjects deemed unsuitable for further study by the investigator.
Test termination:
1. For safety reasons, the study sponsor proposes to stop the study;
2. The Ethics committee decides to stop the study;
3. The lead researcher decides to stop the study. Investigational drug: Avatrombopag: tablet, specification: 20 mg/ tablet. rhTPO: 15000 units /1 ml. Treatment plan: This clinical trial is planned to carry out a 3-month drug study.
A: The Avatrombopag group was given Avatrombopag: 40 mg/ time, once a day, orally, for 3 months.
B: Avatrombopag +rhTPO group, Avatrombopag: 40 mg/ time, once a day, orally; rhTPO: 15000U/ time, once a day, subcutaneous injection; Both were 3 months.
Efficacy index:
Main therapeutic indicators:
Overall response rate at 3 months (OR);
Secondary efficacy measures:
Complete response rate at 3 months (CR); The time of the first occurrence of PR and CR within 3 months of medication; The proportion of subjects who were off platelet transfusion at 3 months; Hemorrhage score records of patients within 3 months of medication; Health-related quality of life score (SF-36 scale).
Efficacy criteria:
Complete response (CR) : HGB > 100 g/L; ANC > 1.5 × 109 /L; PLT > 100×109/L; Partial response (PR) : disengagement from component blood transfusion and no longer meeting the diagnostic criteria for SAA; Invalid (NR) : SAA diagnostic criteria are still met.

开始日期2024-02-01

申办/合作机构

江苏复星医药销售有限公司

NCT06202625 / Not yet recruiting临床2/3期IIT

Efficacy and Safety of Avatrombopag in the Treatment of Thrombocytopenia After Haploidentical Hematopoietic Stem Cell Transplantation: Prospective, Multi-center, Double-blinded, Randomized Placebo-controlled Study

In this study, investigators aim to evaluate the efficacy of avatrombopag in thrombocytopenic patients after haploidentical hematopoietic stem cell transplantation (haplo-HSCT) through a prospective, multi-center, double-blinded, randomized placebo-controlled clinical trial.

开始日期2024-01-15

申办/合作机构

北京大学人民医院

[+9]

100 项与马来酸阿伐曲泊帕相关的临床结果

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100 项与马来酸阿伐曲泊帕相关的转化医学

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100 项与马来酸阿伐曲泊帕相关的专利（医药）

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124

项与马来酸阿伐曲泊帕相关的文献（医药）

2024-02-01·Journal of Pharmacy Practice

Avatrombopag for the Treatment of Immune Thrombocytopenia and Periprocedural Thrombocytopenia Associated With Chronic Liver Disease

Review

作者: Moore, Donald C. ; Tran, Thuy B. ; Downing, Lauren ; Elmes, Joseph B. ; Arnall, Justin R.

Objective: To review the pharmacology, pharmacokinetics, clinical efficacy, safety, dosing and administration, and place in therapy of avatrombopag for the treatment of immune thrombocytopenia and chronic liver disease-associated thrombocytopenia. Summary: Avatrombopag is an orally administered thrombopoietin receptor agonist approved for the treatment of immune thrombocytopenia and is the first oral thrombopoietin receptor agonist approved for the treatment of perioperative thrombocytopenia associated with chronic liver disease in adults. The efficacy and safety of avatrombopag has been demonstrated in a multicenter, randomized, double blind, placebo-controlled phase III study in the setting of immune thrombocytopenia and in 2 identically designed, multicenter, randomized, double blind, placebo-controlled phase III trials in the setting of thrombocytopenia associated with chronic liver disease. The most common adverse events reported in the clinical trials were headache, fatigue, and gastrointestinal toxicities. The incidence of bleeding events was comparable between the avatrombopag and placebo treatment groups in each study. Avatrombopag has not been shown to be associated with hepatoxicity and does not require food restriction like the other oral thrombopoietin receptor agonist for immune thrombocytopenia, eltrombopag. Also, unlike eltrombopag for immune thrombocytopenia, it can be dosed less frequently than once daily. Conclusion: Avatrombopag offers another safe and effective oral option for the treatment of immune thrombocytopenia without food restrictions and an alternative, transfusion-sparing option for thrombocytopenia associated with chronic liver disease patients undergoing surgery.

2024-02-01·American Journal of Hematology

Avatrombopag for adults with early versus chronic immune thrombocytopenia

Article

作者: Connell, Nathan T ; Goodarzi, Katayoon ; Leaf, Rebecca K ; Connors, Jean M ; Al-Samkari, Hanny ; Virk, Zain M ; Kuter, David J

Abstract:

Avatrombopag is a newer thrombopoietin receptor agonist (TPO‐RA) currently approved to treat chronic ITP (duration >12 months). No studies have yet evaluated the safety and effectiveness of avatrombopag in newly diagnosed ITP (duration <3 months) or persistent ITP (duration 3–12 months), and so its use in these populations is presently off‐label worldwide. We hypothesize that avatrombopag has similar safety and effectiveness irrespective of ITP disease phase. To evaluate this, we performed a multicenter observational cohort study of adults with ITP treated with avatrombopag, comparing patient outcomes by disease phase (newly diagnosed/persistent versus chronic). Seventy‐five patients were included, 23 with newly diagnosed/persistent ITP (17.7 patient‐years of avatrombopag treatment) and 52 with chronic ITP (65.3 patient‐years of avatrombopag treatment). On avatrombopag, 91% of newly diagnosed/persistent patients versus 96% of chronic patients (p = .58) achieved a platelet response (≥50 × 109/L) and 86% versus 81% of patients (p = .78) achieved a complete response (≥100 × 109/L). Median platelet counts on avatrombopag were similar between the two groups (165 × 109/L vs. 129 × 109/L, p = .57). Response durability was high and similar in both groups. No patients in the newly diagnosed/persistent group had a major bleeding event, thromboembolic event or avatrombopag discontinuation for adverse events, compared with 4, 1, and 2, respectively, in the chronic group. Thrombocytosis (platelets ≥400 × 109/L) incidence was similar in the two groups. No other drug‐related adverse events occurred in either group. Avatrombopag was safe and effective in patients with newly diagnosed and persistent ITP, with outcomes numerically, statistically, and clinically similar to patients receiving avatrombopag for chronic ITP.

2024-01-01·BMJ Open

Efficacy and safety of avatrombopag in combination with immunosuppressive therapy in treatment-naïve and relapsed/refractory severe aplastic anaemia: protocol for the DIAAMOND-Ava-FIRST and DIAAMOND-Ava-NEXT Bayesian Optimal Phase II trials

Article

作者: Szer, Jeff ; Stevenson, William ; Pepperell, Dominic ; Firkin, Frank ; Wood, Erica ; Cunningham, Ilona ; Ting, Stephen ; Higgins, Alisa ; Waters, Neil ; McQuilten, Zoe ; Fox, Vanessa ; Fox, Lucy ; Blombery, Piers ; Mason, Kylie ; Curnow, Jennifer ; Filshie, Robin ; Young, Lauren ; Lacaze, Paul ; Heritier, Stephane ; Patil, Sushrut ; Wilson, Kate ; Mills, Anthony K ; Hiwase, Devendra K

Introduction:

Immunosuppressive therapy (IST) with antithymocyte globulin (ATG) and ciclosporin is standard of care for patients with severe aplastic anaemia (sAA) not eligible or suitable for allogeneic stem cell transplant. While patients respond to IST, few achieve complete responses and a significant proportion are refractory or relapse. The addition of eltrombopag, a thrombopoietin-receptor agonist (TPO-A), to IST has been shown to improve haematological responses in sAA. Avatrombopag is a second-generation TPO-A with potential advantages over eltrombopag. However, to date avatrombopag has not been studied in sAA.

Methods and analysis:

Investigator-initiated, single-arm registry-based Bayesian Optimal Phase II trial of avatrombopag conducted in two cohorts, patients with untreated sAA (FIRST cohort) and in patients with sAA that has relapsed or is refractory to IST (NEXT cohort). In the FIRST cohort, participants receive IST (equine ATG and ciclosporin) plus avatrombopag from day 1 until day 180 at 60 mg oral daily, with dose adjusted according to platelet count. Participants in the NEXT cohort receive avatrombopag at 60 mg oral daily from day 1 until day 180, with or without additional IST at the discretion of the treating clinician.For each cohort, two primary endpoints (haematological response and acquired clonal evolution) are jointly monitored and the trial reviewed at each interim analysis where a ‘go/no-go’ decision is made by evaluating the posterior probability of the events of interests.

Ethics and dissemination:

The trial has received ethics approval (Monash Health RES-18-0000707A). The trial conduct will comply with ICH-GCP and all applicable regulatory requirements. The results of the trial will be submitted to a peer-review journal for publication.

Trial registration number:

ACTRN12619001042134, ACTRN12619001043123.

131

项与马来酸阿伐曲泊帕相关的新闻（医药）

2024-04-25

·PRNewswire

Sobi Q1 2024 report: Strong sales reflecting the strength of the portfolio

STOCKHOLM, April 25, 2024 /PRNewswire/ -- Swedish Orphan Biovitrum AB (publ) (Sobi®) today announced its report for the first quarter 2024 First Quarter 2024 Total revenue increased 19 per cent, 20 per cent at constant exchange rates, (CER)1, to SEK 6,256 M (5,239) Haematology revenue increased 46 per cent at CER to SEK 4,075 M (2,815), reflecting growth in all medicines, mainly driven by strong sales of Doptelet® of SEK 756 M (475), sales of Vonjo® of SEK 320 M (—), and sales of Aspaveli®/Empaveli® of SEK 240 M (95) Immunology revenue decreased 11 per cent at CER to SEK 1,908 M (2,151), mainly reflecting a significant drop in Synagis® sales to SEK 520 M (1,398), partly compensated by strong sales of Gamifant® of SEK 438 M (219), and royalty on Beyfortus™ of SEK 318 M (—) Revenue from medicines in the strategic portfolio more than doubled in the quarter, to SEK 2,193 M (794), driving the majority of the growth. The strategic portfolio grew by 177 per cent at CER and helped to further transform the company The adjusted EBITA margin1,2 was 37 per cent (40), excluding items affecting comparability (IAC)2. EBITA was SEK 2,177 M (2,121), corresponding to a margin of 35 per cent (40). EBIT was SEK 1,313 M (1,495) Earnings per share (EPS) before dilution was SEK 2.35 (3.44)3. Adjusted EPS before dilution1 was SEK 2.70 (3.44)3. Cash flow from operating activities was SEK 2,256 M (1,983) Outlook 2024 - unchanged Revenue is anticipated to grow by a high single-digit percentage at CER The adjusted EBITA margin is anticipated to be in the mid-30s percentage of revenue The strategic portfolio includes Sobi's medicines Aspaveli/Empaveli, Doptelet excluding China, Gamifant, Vonjo and Zynlonta®, and royalty on Sanofi's sales on Altuviiio™ and Beyfortus. Investors, analysts and media are invited to participate in a conference call, which will include a presentation of the results and a Q&A session on the same day at 16:00 CEST. The presentation can be followed live here or afterwards on sobi.com. The slides will be made available on sobi.com prior to the conference call. To participate in the conference call, please use the following dial-in details: Sweden: +46 8 5051 0031 United Kingdom: +44 207 107 06 13 United States: +1 631 570 56 13 For other countries, please find the details here. Sobi Sobi® is a specialised international biopharmaceutical company transforming the lives of people with rare and debilitating diseases. Providing reliable access to innovative medicines in the areas of haematology, immunology and specialty care, Sobi has approximately 1,800 employees across Europe, North America, the Middle East, Asia and Australia. In 2023, revenue amounted to SEK 22.1 billion. Sobi's share (STO: SOBI) is listed on Nasdaq Stockholm. More about Sobi at sobi.com and LinkedIn. Contacts For details on how to contact the Sobi Investor Relations Team, please click here. For Sobi Media contacts, click here. This information is information that Sobi is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the contact person set out below, on 25 April 2024 at 08:00 CEST. Gerard Tobin Head of Investor Relations This information was brought to you by Cision The following files are available for download:

财报生物类似药

2024-04-24

·药融圈

独一份me-only，年超40亿业绩的国产新药

▲5月30-31日第八届广州生物医药创新者峰会扫码立即报名注：本文不构成任何投资意见和建议，以官方/公司公告为准；本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及），不代表平台立场。任何文章转载需得到授权。药融云数据www.pharnexcloud.com监测显示：2024年3月20日，上市公司三生制药（01530.HK）公布年度业绩。业绩公告显示，2023年三生制药营业收入约人民币78.16亿元，比去年同期增长13.8%；毛利约人民币66.42亿元，比去年同期增长17.1%；经调整的经营性归母净利润约人民币19.52亿元，比去年同期增长17.7%；经调整EBITDA约人民币27.68亿元，比去年同期增长20.2%。公司主要有三大板块业务，毛发皮肤与CDMO贡献的额度为11.4亿元和1.7亿元。2023年，三生制药核心生物药产品营收保持强劲增长，同比增加23.8%（2023年上半年销售额同比增长28.2%）。据悉，本品是全球目前唯一商业化的重组人血小板生成素（Recombinant Human Thrombopoietin，rhTPO）。2023年销售收入达42.05亿元。最早于中国在2005年获批上市，目前该产品已顺利续约医保。药融云数据www.pharnexcloud.com值得关注的是，其他国产新药在2023年度业绩过20亿人民币的有：百济神州泽布替尼、正大天晴的安罗替尼、翰森制药的阿美替尼、信达生物的PD-1、恒瑞医药的PD-1、石药集团的丁苯酞、强生/传奇生物的BCMA CAR-T、百济神州的PD-1、等等。目前上述重组人血小板生成素（Recombinant Human Thrombopoietin，rhTPO）产品已获批成人实体瘤化疗后血小板减少症（CIT），成人原发免疫性血小板减少症（ITP）适应症和儿童或青少年的持续性或慢性原发免疫性血小板减少症（ITP）。以上适应症其他获批的品种有海曲泊帕、阿伐曲泊帕、罗普司亭等。附，分享：三生制药的2023财年业绩展示报告PPT/PDF公开。发送三生（请正确复制）至药融圈微信公众号（点击下方发送）后台可获得原文件。仅供学习交流分享！关于三生制药三生制药是一家集研发、生产和销售为一体的生物制药领军企业，致力于以高品质的药品提高患者生存质量，为人类健康造福。目前，公司拥有100余项国家发明专利授权，40余种上市产品，覆盖肾科、肿瘤科、自身免疫性疾病、眼科及皮肤科等多种治疗领域。公司拥有抗体药物国家工程研究中心以及生物药和化药双平台的4大研发中心，共有29种在研产品，其中25种为国家新药，并拥有符合GMP标准的5大生产基地。参考：NMPA/CDE；药融云数据www.pharnexcloud.com；FDA/EMA/PMDA；相关公司公开披露；www.3sbio.com；等等。本文仅用于向医疗卫生专业人士提供科学信息，不代表平台立场，不作任何用药推荐活动推荐点击图片查看详情↓↓【关于药融圈】药融圈PRHub旨在帮助生物医药科技型企业进行品牌推广及商务拓展服务，针对客户的真实需求制定系统化解决方案，通过“翻译-降维-场景化”将客户的品牌信息以直白易懂的方式被公众知悉，同时在流量渠道覆盖100万+垂直用户基础上实现合作目的，帮助合作伙伴完成从品牌开始到商务为终的闭环营销服务。我们已经完成了数十场线下1000人规模的生物医药研发类会议，涵盖小分子新药，大分子新药，改良型新药，BD跨境交易等多个领域，服务了百余家上市/独角兽/生物技术/制药企业。

财报医药出海上市批准细胞疗法

2024-04-22

·医药魔方

2023年国内药企研发投入TOP10

可持续的创新药产出离不开充沛资金的支持。在当前的行业环境下，尽管很多药企都面临着资金紧张的问题，但仍有不少企业逆势而上。他们有的通过BD授权产品/技术回笼现金反哺内部创新，凭借相对稳定的销售现金流仍维持着较高比重的研发投入，推进着与国际接轨的创新管线。借着国内各家药企财报的披露，医药魔方在此整理了2023年研发投入最高10家企业的一些数据，以供参考。2023年国内药企研发投入TOP10注：美元单位统一换算成人民币单位（按2023年平均汇率换算，即1美元=7.0467元人民币）公司：百济神州研发费用：17.79亿美元（125.33亿元）研发费用占营收比例：72.34%五年来，百济神州的研发投入一直遥遥领先领先其他药企，2022年首次超过百亿人民币（110.34亿元）后，2023年再度增长至125.33亿元。百济神州研发费用连年增涨，主要在于其对候选药物研究以及临床开发的不吝投入。2023年，CDK4抑制剂BGB-43395片、BCL-2抑制剂BGB-21447片、HPK1抑制剂BGB-26808片、DGKζ抑制剂BGB-30813片等5款新分子实体首次进入了临床研究；新一代BCL-2抑制剂Sonrotoclax片启动了4项注册性试验，包括一线治疗CLL的全球III期试验；BTK CDAC药物BGB-16673也开展了治疗R/R CLL、MCL的全球扩展队列研究......来源：百济神州财报除了已经进入临床开发的产品，百济神州还在同步扩大候选药物规模，2024年预计推动至少10款新分子实体进入首次临床试验。不难看出，百济神州目前对于血液肿瘤、实体瘤进行了非常全面的管线布局，不论是丰富多样的靶点/机制覆盖，还是多元化的技术手段运用，都更加注重产品的迭代开发逻辑和深层进阶。当然，长年的投入也开始转化为商业回报。2023年其核心产品泽布替尼得益于CLL、SLL、WM、FL、MZL等适应症，特别是一线CLL、SLL新适应症在欧美、澳大利亚和中国等多个国家上市，全球销售额较上年同期（38.29亿元）增长了138.7%，达到91.38亿元（约13亿美元），成为国内首个年销售额超过十亿美元的创新药；PD-1产品替雷利珠单抗在数十款竞品的攻势下，也实现了增长（+33.1%），贡献了38.06亿元收入。公司：恒瑞医药研发投入：61.50亿元研发投入占营收比例：27%恒瑞医药2023年总营收228.2亿元，同比增长7.26%，其中创新药收入106.37亿（+22.1%），占到总收入46.6%。自2021年，恒瑞医药对研发的投入都保持在60亿元以上，2023年达到61.5亿元，其中费用化49.54亿。高额的研发投入使得恒瑞保持了较快的创新更新速度，并且助力其逐步建立了成熟的化药（含多肽、PROTAC）、单/双抗体药、ADC药物、小核酸、核药等技术平台，同时也布局了PDC、AOC、DAC、mRNA等新分子模式平台，以及结构生物学、AI药物研发等平台，全面聚焦前沿创新，同时其产品管线规模也日益壮大。此外，恒瑞国际化脚步也不断加快，尤其在产品权益许可海外方面，恒瑞2023年多次主动出击并且收获颇丰。一年拿下5笔deals，交易总金额超40亿美金，这是恒瑞在2023年给出的BD成绩，从侧面展现了其创新实力。公司：复星医药研发费用：51.72亿元研发费用占营收比例：17.11%2023年，复星医药总营收约414亿元，其中制药业务收入302.22亿元，占比高达73%，是其名副其实的核心业务，这主要源于汉斯状（11.2亿元，+230.2%）、汉曲优（27.5亿元，+58.2%）、苏可欣（9.2亿元，+19.7%）等产品收入保持快速增长。作为主营业务，复星医药将超过87%的研发费用（51.72亿元）投入其中。而制药业务中又有超85%-90%的资金涌入了创新药业务领域，这里面有近50%的资金被投入到抗肿瘤产品的研发中。来源：复星医药财报目前，复星医药聚焦在肿瘤、自身免疫、中枢神经、慢病（肝病/代谢/肾病）等核心治疗领域，重点强化了小分子、抗体/ADC、RNA和细胞疗法四大核心技术平台，同时积极布局肿瘤疫苗、AI药物研发等前沿技术，以开发出更多Best-in-Class（BIC）和First-in-Class（FIC）产品。公司：石药集团研发费用：48.30亿元研发费用占成药营收比例：18.80%2023年，石药集团营收314.5亿元（+1.7%），除了成药业务增长了4.6%至256.37亿元，原料产品、功能食品及其他业务收入都在下滑，分别收入36.41亿元（-9.7%）、21.72亿元（-8.9%）。成药业务成绩的取得与石药集团对创新研发一如既往的高投入息息相关。作为正在创新转型的大型药企，石药集团对创新药物的研发投入几乎稳居TOP5，2023年研发费用再次提升21%达到48.3亿元，位列TOP4。超过60个重点在研药物已进入临床或申报阶段，其中7个已递交上市申请，18个处于注册临床阶段......继2023年首次进入全球管线规模TOP25，2024年石药集团再次入围该榜单，预计未来五年将有近50个创新药及新型制剂药物申报上市，以及超60个仿制药上市。具体管线来看，石药集团主要聚焦六大治疗领域（抗肿瘤、精神神经、心血管、免疫与呼吸、消化与代谢和抗感染），借助纳米、mRNA、siRNA、单抗、双抗、ADC、CAR-T、PROTAC、LYTAC、AI等技术逐步搭建了丰富的产品管线，靶点涵盖PD-1、DPP-4、IgE、COX-2、FcRn、HER2、EGFR、GLP-1、IL-4R、IL-12/23、RANKL、PCSK9等。公司：中国生物制药研发费用：47.04亿元研发费用占营收比例：18%2023年，中国生物制药总营收262亿，其中创新产品收入占比38%，达到98.9亿；研发总开支为47亿，其中创新药及生物药的研发投入占比超过77%，常年位居国内药企TOP5。中国生物制药主要聚焦肿瘤、肝病、呼吸系统、外科/镇痛四大治疗领域的创新药物开发，尤其关注抗肿瘤新药的研发，2023年该领域的投资金额占比高达71%，截至2023年底，其在研产品有超过41%为肿瘤药物（60/145）。来源：中国生物制药JPM2024目前，肿瘤领域除了包括安罗替尼、艾贝格司亭α、派安普利单抗等在内产品已上市，PD-L1药物贝莫苏拜单抗、KRAS G12C抑制剂D-1553、ALK/c-Met抑制剂TQ-B3139、ROS1/ALK/c-Met抑制剂TQ-B3101等均处于上市审评阶段。公司：传奇生物研发费用：3.82亿美元（26.93亿元）研发费用占营收比例：134.05%近几年，传奇生物的研发开支在国内药企中排名十分靠前，2023年以3.82亿美元（接近27亿人民币，+13.9%）排在第6位，丝毫不亚于国内一部分Bigpharma。据财报，传奇生物研发费用大幅增加，主要是由于BCMA CAR-T疗法Carvykti持续的研发活动，以及用于实体瘤的CAR-T新疗法临床开发，此外还包括在比利时建立生产基地投入的成本。传奇生物的Carvykti于2022年先后在美国、欧洲以及日本上市，用于既往接受过至少四线治疗的复发/难治性多发性骨髓瘤（R/R MM），上市以后Carvykti放量迅速，在2023这一首个完整商业化年份创收5亿美元。就在今年4月，Carvykti凭借III期CARTITUDE-4研究积极结果，获得FDA批准将适应症扩展至MM患者二线治疗。毫无疑问，前线治疗的获批后，Carvykti覆盖人群将大幅度增加，也有望实现更出色的商业化成绩。来源：传奇生物财报除了Carvykti，传奇生物目前还把研发目光放在了实体瘤CAR-T疗法，靶点涵盖GPC3、GCC、DLL3、CLDN18.2等，以及异体CAR-NK和CAR-NKγδT细胞疗法，靶点包括CD20、CD19、BCMA等。值得关注的是，继与强生牵手共同开发Carvykti之后，2023年11月，传奇生物又与另一制药巨头诺华就靶向DLL3的LB2102达成了合作，交易总金额高达11.1亿美元。公司：信达生物研发费用：22.28亿元研发费用占营收比例：35.90%信达生物CEO俞德超博士将2023年视为信达生物新十年发展的重要转折点，因为过去一年里，信达生物经营效率显著提升，财务表现明显改善，而在销售、市场推广、行政开支、生产效率等成本管控以及商业化产品收入快速增长推动下，2023年亏损大幅收窄，全年总营收上涨36.2%至62.06亿元，其中产品收入57.28亿元（+38.4%）。2023年，信达生物的研发开支相较上年（28.71亿元）有所减少（22.28亿元），这主要源于其在研发战略上，坚持了科学高效的布局理念，同时也合理配置了多元化产品组合的研发资源和相关投资。总体而言，无论是新产品/新适应症的上市/NDA递交、各阶段临床管线的推进等产品进展，还是对外战略合作方面，信达生物在2023年都有了不错的成果。来源：信达生物财报在诺和诺德以及礼来两大巨头的引领下，2023年GLP-1赛道风起云涌，包括信达生物、恒瑞以及华东医药等国内药企都在加速推动产品进度。在这其中，信达生物GLP-1R/GCGR双靶激动剂玛仕度肽的开发速度一马当先，从II期研究数据读出，到5项大型III期研究的密集启动，目前已经申报上市，信达生物正全力调配资源，向减肥领域发起进攻。当然，信达生物在肿瘤、CVM、自身免疫及眼科等综合产品领域成长速度也不容小觑，用于ROS1突变肺癌二线治疗的ROS1抑制剂IBI344、KRAS G12C突变肺癌二线治疗的KRAS G12C抑制剂IBI351、中重度斑块型银屑病疗法IL-23p19单抗IBI112、甲状腺眼病药物IGF-1R抗体IBI311等产品都渐进收获期。公司：和黄医药研发费用：3.02亿美元（21.28亿元）研发费用占营收比例：36.04%2023年，是和黄医药取得里程碑式进展的一年，其核心产品呋喹替尼海外权益于1月授权给武田后，11月正式获得FDA批准上市。财报显示，在美上市不到两个月，呋喹替尼就实现了1510万美元的销售额，其于中国市场的收入为1.075亿美元（+15%）。由于呋喹替尼达成了BD交易，2023年和黄医药不仅收到了武田4亿美元的首付款，其中2.8亿美元确认为2023年收入，还获得了FDA批准触达的3200万美元里程金，这两笔进账都大大拉升了和黄医药全年总营收，也助力其首次跨越了盈利线，拿下了1.01亿美元净利润。和黄医药2023年的研发投入较2022年（3.87亿美元）减少了22%至3.02亿美元（21.28亿元），这主要是由于几项大型注册试验已经陆续完成，而且中国以外的发展以对外合作为主，合作伙伴承担主要开发费用。公司：翰森制药研发费用：20.97亿元研发费用占营收比例：20.80%翰森制药近五年来对创新产品的研发投入也呈上升趋势，从2019年的11.2亿元到2023年的21亿元（+23.8%），增长了近一倍。当然，其创新药物的销售表现也十分亮眼，尤其在总营收中的占比逐年提高，2023年创新药与合作产品的收入（68.65亿元，+37.1%）占总营收（101.04亿元，+7.7%）的比重由2022年的53.4%已经上升至67.9%。概括来看，翰森制药的创新药主要围绕抗肿瘤、抗感染、中枢神经系统、代谢以及自身免疫五大类布局，7款创新产品已经商业化，另有50多个临床项目同步开发中，里面涵盖了30余款创新药，其中有8款为2023年新增产品。翰森制药的技术平台也十分丰富，单抗、双抗、融合蛋白、纳米制剂、siRNA、ADC、PROTAC等都有涉猎。2023年，翰森制药的创新产品还实现了BD交易，自研产品B7-H3 ADC、B7-H4 ADC海外权益都转让给了制药巨头GSK。公司：君实生物研发费用：19.37亿元研发费用占营收比例：128.88%于君实生物而言，2023年同样沉甸甸，10月27日，旗下PD-1药物特瑞普利单抗获得FDA批准上市，联合吉西他滨/顺铂一线治疗鼻咽癌或者单药用作鼻咽癌含铂治疗后的二线及以上治疗。这是国产首个获批进入美国市场的PD-1药物，在中国创新药发展中具有里程碑式意义。目前，君实生物商业化产品有3款，2023年共计收入15.03亿元，特瑞普利单抗贡献了最主要营收（9.19亿元）。特瑞普利单抗作为最早上市的PD-1四小龙成员，虽然销售额落后于同类产品，但回温信号明显，继2022年45%的涨幅后，2023年又同比增加了25%，君实生物正加大海外上市申报工作。此外，君实生物也在加强后期阶段管线的建设，BTLA单抗tifcemalimab启动了2项III期临床研究；抗PCSK9单抗昂戈瑞西单抗的上市申请正在接受审评；IL-17A单抗JS005治疗银屑病的III期研究已经启动。Copyright © 2024 PHARMCUBE. All Rights Reserved.欢迎转发分享及合理引用，引用时请在显要位置标明文章来源；如需转载，请给微信公众号后台留言或发送消息，并注明公众号名称及ID。免责申明：本微信文章中的信息仅供一般参考之用，不可直接作为决策内容，医药魔方不对任何主体因使用本文内容而导致的任何损失承担责任。精彩预告线下沙龙↑ 长按扫码，报名活动 ↑

财报临床3期引进/卖出临床1期医药出海

100 项与马来酸阿伐曲泊帕相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D10307	马来酸阿伐曲泊帕	B02BX	DB11995

研发状态

适应症	最高研发状态	国家/地区	公司
血小板减少症	批准上市	中国更多	Eisai Co., Ltd. 更多
特发性血小板减少性紫癜	批准上市	挪威更多	Swedish Orphan Biovitrum AB 更多
肝病	批准上市	中国更多	Eisai Co., Ltd. 更多
慢性肝病	批准上市	加拿大更多	Sobi North America 更多
肿瘤	终止	中国更多	Dova Pharmaceuticals, Inc. 更多

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临床结果

适应症

分期

评价

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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


AVAMAD STUDY (ASH2023)	N/A	特发性血小板减少性紫癜二线	66	Avatrombopag	遞憲夢憲襯顧鏇簾醖遞(鏇壓鏇醖糧鑰觸蓋鬱鏇) = 鏇壓選夢鑰網範積鹹憲選積願繭鹹鬱積艱廠艱 (範鬱糧繭願衊鏇壓鏇醖 ) 更多	-	2023-12-11
ASH2023	N/A	特发性血小板减少性紫癜	50	Avatrombopag	齋艱壓醖蓋窪膚簾淵鹽(鏇積顧衊膚鹽夢顧顧鬱) = two patients reported mild fatigue 蓋艱鑰選鹹衊簾鹹顧糧 (觸遞願膚築鏇廠積餘餘 ) 更多	-	2023-12-10
ASH2023	N/A	特发性血小板减少性紫癜	55	Avatrombopag + Fostamatinib	窪範構膚廠淵壓簾選窪(鏇簾廠襯願淵憲鏇壓壓) = 構衊艱鑰膚範鬱糧憲廠鏇鏇構獵糧淵醖蓋淵鹹 (蓋製鏇觸壓觸壓齋衊艱 ) 更多	积极	2023-12-10
NCT04638829 (Phase 4 study, ASH2023)	临床4期	特发性血小板减少性紫癜	47	Eltrombopag	遞衊構夢願製鏇壓範夢(築鬱製鹽範築顧壓簾網) = 鬱鏇餘顧網壓餘構鬱鹹鏇製鏇蓋繭壓鑰構觸襯 (觸繭糧艱艱衊選鏇蓋觸 )	-	2023-12-10
NCT04638829 (Phase 4 study, ASH2023)	临床4期	特发性血小板减少性紫癜	47	Romiplostim	遞衊構夢願製鏇壓範夢(築鬱製鹽範築顧壓簾網) = 觸獵蓋壓糧鹹範餘艱積鏇製鏇蓋繭壓鑰構觸襯 (觸繭糧艱艱衊選鏇蓋觸 )	-	2023-12-10
ASH2023	N/A	急性淋巴细胞白血病 \| 血小板减少症一线	30	Avatrombopag treatment	構鹹簾範鬱遞簾鑰網憲(製醖憲繭鬱壓壓醖鹽壓) = 蓋積淵獵築衊鏇願衊淵遞鏇鏇觸醖鬱鬱齋壓構 (壓憲積獵觸鏇製襯積築 ) 更多	-	2023-12-09
ASH2023	N/A	急性淋巴细胞白血病 \| 血小板减少症一线	30	No avatrombopag treatment	構鹹簾範鬱遞簾鑰網憲(製醖憲繭鬱壓壓醖鹽壓) = 齋餘壓窪觸願衊憲壓鏇遞鏇鏇觸醖鬱鬱齋壓構 (壓憲積獵觸鏇製襯積築 ) 更多	-	2023-12-09
NCT04728789 (Pubmed)	临床2期	难治性重度再生障碍性贫血	25	Avatrombopag	顧艱淵網顧願鬱膚艱簾(遞窪簾觸衊獵選顧鬱艱) = 製鹹窪鑰醖範網鑰襯憲遞蓋範觸構衊糧簾鏇築 (積憲廠選選鑰顧糧鏇艱 ) 更多	-	2023-12-01
ChiCTR2100045895 (EHA2023)	临床4期	再生障碍性贫血	20	Avatrombopag 40 mg/day	選壓遞衊鹽鹽醖夢繭製(蓋醖選積齋網窪襯願鹹) = 積構築選膚繭築遞範鑰選齋鬱顧膚鏇壓構廠淵 (鏇淵觸選壓壓積餘廠築, 12–47) 更多	-	2023-06-08
EHA2023	临床3期	特发性血小板减少性紫癜	74	Avatrombopag	鏇網積蓋積選構壓醖觸(遞範齋鹹齋壓遞獵醖製) = 繭鑰餘鑰餘獵餘構顧網壓窪願獵蓋鏇蓋簾選窪 (積製選選遞製窪夢積醖, 62.69%, 87.97%)	-	2023-06-08
EHA2023	临床3期	特发性血小板减少性紫癜	74	Placebo	鏇網積蓋積選構壓醖觸(遞範齋鹹齋壓遞獵醖製) = 艱壓網壓繭範選膚網糧壓窪願獵蓋鏇蓋簾選窪 (積製選選遞製窪夢積醖, 0.95%, 25.13%)	-	2023-06-08
EHA2023	N/A	特发性血小板减少性紫癜	20	Avatrombopag	獵構製顧廠淵齋壓糧餘(鬱鏇襯糧選構積蓋襯遞) = 2 cases of epistaxis 簾襯憲襯壓廠築齋製糧 (襯鹹蓋積膚鑰餘網觸鹽 ) 更多	-	2023-06-08
ChiCTR2100045895 (EHA2023)	临床4期	再生障碍性贫血	12	Avatrombopag + CsA	蓋築壓衊選艱憲顧觸醖(蓋糧蓋糧餘顧壓糧壓製) = 範網簾鑰鑰廠膚顧網膚窪遞觸蓋願窪築窪齋鹹 (鬱築鏇積繭餘鹽鹹鬱襯 ) 更多	-	2023-06-08