B7-2/GM-CSF cancer gene therapy(Radient Consulting)

Updated data presented at ASH include CAPTIVATE and GLOW studies and real-world evidence abstracts NORTH CHICAGO, Ill. , Dec. 10, 2022 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced new and updated data across clinical and real-world studies in chronic lymphocytic leukemia (CLL). Presentations featured during the 64 th American Society of Hematology (ASH) Annual Meeting and Exposition will include two clinical trials investigating once daily, fixed-duration treatment with IMBRUVICA ® (ibrutinib) plus VENCLEXTA ® /VENCLYXTO ® (venetoclax) (I+V) in adults with CLL and several analyses from real-word data evaluating front-line treatment in CLL. "As the only Bruton's Tyrosine Kinase inhibitor (BTKi) studied across many clinical trials, including up to eight years of follow-up data in CLL and small lymphocytic lymphoma (SLL), these latest data add to overall evidence for IMBRUVICA," said James Dean , M.D., Ph.D., global development lead and executive medical director, Pharmacyclics LLC, an AbbVie company. "These findings further inspire our commitment to the continued investigation of IMBRUVICA in the treatment of patients with CLL." Presentations will include the five-year median follow-up efficacy and safety data from the minimal residual disease (MRD) Cohort of the Phase 2 CAPTIVATE study ( Abstract #92 ) 1 and data on MRD kinetics from the randomized, open-label Phase 3 GLOW study ( Abstract #93 ). 2 Additionally, new data from three studies evaluating the impact of IMBRUVICA ® treatment in the real-world setting will be presented (Abstracts #797 , #1809 , #3132 ). 3,4,5 Five-Year Update from the CAPTIVATE Study Adds to Clinical Data Investigating the Potential of a Fixed-Duration Regimen The MRD Cohort of the Phase 2 CAPTIVATE ( NCT02910583 ) study evaluated 164 patients age 70 years or younger with previously untreated CLL; those who achieved confirmed undetectable minimal residual disease (uMRD) after completion of I+V fixed-duration treatment were randomly assigned to placebo (PBO) (n=43), or continued IMBRUVICA ® treatment (n=43). 1 For patients with confirmed uMRD, median time on study was 56 months (PBO arm range, 40‒65 months; IMBRUVICA ® arm range, 25‒68 months); median post-randomization follow-up was 41 months in both arms. 1 At four years of follow-up, estimated progression-free survival (PFS) was 88 percent (95 percent Confidence Interval [CI], 74-95, seven progressive disease events) with PBO compared to 95 percent (95 percent CI, 82-99, two progressive disease events) with continued IMBRUVICA ® treatment. 1 Additionally, at year four, patients in the PBO arm of the study achieved an estimated overall survival (OS) rate of 100 percent compared to 98 percent (95 percent CI, 84-99.7) in the IMBRUVICA ® treatment arm. 1 No new atrial fibrillation (AF) or Grade three or higher hemorrhage events occurred in the PBO arm during the three year post-randomization period, and one patient in the IMBRUVICA ® arm had AF in the second year post-randomization. 1 During the three-year post-randomization period, adverse events (AEs) of any grade for patients treated with IMBRUVICA ® were arthralgia (4/41), hypertension (2/41), neutropenia (1/41) and diarrhea (1/41). No new grade three or higher hemorrhage events occurred in either arm. 1 The Data of IMBRUVICA ® in the Treatment of CLL Through Real-World Studies An oral presentation ( Abstract #797 ) will report findings comparing time to next treatment (TTNT) in patients with CLL who received first-line treatment with IMBRUVICA ® or acalabrutinib based on a real-world study utilizing electronic medical records. 3 A poster presentation ( Abstract #1809 ) will highlight results from a pooled analysis of the RESONATE-2 ( NCT01722487 ), ECOG1912 ( NCT02048813 ), and iLLUMINATE ( NCT02264574 ) clinical studies investigating the comparison of OS in previously untreated CLL patients treated with IMBRUVICA ® to that of the available age-matched general population, as well as comparative results of OS in patients treated with IMBRUVICA ® versus chemotherapy and chemoimmunotherapy. 4 Thirdly, the final analysis from the informCLL™ real-world (RW) registry, which assessed RW outcomes with first-line IMBRUVICA ® versus chemoimmunotherapy (CIT) in patients with CLL and SLL, will be presented as a poster ( Abstract #3132 ). 5 The informCLL™ registry enrolled 1,459 patients, of whom 59 percent were previously untreated. 5 First-line treatment with IMBRUVICA ® was associated with longer TTNT than with CIT and sustained benefit, with up to four years of follow-up. 5 In the IMBRUVICA ® cohort (n=383), serious adverse events (AEs) occurred in 144 patients (38 percent), most commonly (greater or equal to three percent of patients) pneumonia (six percent) and atrial fibrillation (five percent); AEs led to discontinuation of IMBRUVICA ® in 135 patients (35 percent), most commonly atrial fibrillation (five percent) and fatigue (three percent). In the CIT cohort (n=363), serious AEs occurred in 85 patients (23 percent), most commonly febrile neutropenia (four percent) and pneumonia (three percent). AEs led to discontinuation of CIT in 61 patients (17 percent), and no single AE term led to discontinuation in three percent or more of patients (most common was anemia, two percent). 5 The spectrum and frequency of AEs observed with first-line treatment appeared consistent with data from clinical studies and other RWE studies. 5 About IMBRUVICA ® IMBRUVICA ® (ibrutinib) is a once-daily oral medication that is jointly developed and commercialized by Janssen Biotech, Inc. and Pharmacyclics LLC, an AbbVie company. IMBRUVICA ® blocks the Bruton's tyrosine kinase (BTK) protein, which is needed by normal and abnormal B cells, including specific cancer cells, to multiply and spread. By blocking BTK, IMBRUVICA ® may help move abnormal B cells out of their nourishing environments and inhibits their proliferation. 6,7,8 IMBRUVICA ® is approved in more than 100 countries and has been used to treat more than 270,000 patients worldwide. There are more than 50 company-sponsored clinical trials, including 18 Phase 3 studies, over 11 years evaluating the efficacy and safety of IMBRUVICA ® . IMBRUVICA ® was first approved by the U.S. Food and Drug Administration (FDA) in November 2013, and today is indicated for adult patients in six disease areas, including five hematologic cancers. These include indications to treat adults with CLL/SLL with or without 17p deletion (del17p), adults with Waldenström's macroglobulinemia (WM), adults with previously treated mantle cell lymphoma (MCL)*, adult patients with previously treated marginal zone lymphoma (MZL) who require systemic therapy and have received at least one prior anti-CD20-based therapy*, as well as adult and pediatric patients one year of age and older with previously treated chronic graft versus host disease (cGVHD) after failure of one or more lines of systemic therapy. 9 *Accelerated approval was granted for MCL and MZL based on overall response rate. Continued approval for MCL and MZL may be contingent upon verification and description of clinical benefit in confirmatory trials. For more information, visit www.IMBRUVICA.com . IMPORTANT SIDE EFFECT INFORMATION Before taking IMBRUVICA ® , tell your healthcare provider about all of your medical conditions, including if you: have had recent surgery or plan to have surgery. Your healthcare provider may stop IMBRUVICA ® for any planned medical, surgical, or dental procedure. have bleeding problems have or had heart rhythm problems, smoke, or have a medical condition that increases your risk of heart disease, such as high blood pressure, high cholesterol, or diabetes have an infection have liver problems are pregnant or plan to become pregnant. IMBRUVICA ® can harm your unborn baby. If you are able to become pregnant, your healthcare provider will do a pregnancy test before starting treatment with IMBRUVICA ® . Tell your healthcare provider if you are pregnant or think you may be pregnant during treatment with IMBRUVICA ® . Females who are able to become pregnant should use effective birth control (contraception) during treatment with IMBRUVICA ® and for 1 month after the last dose. Males with female partners who are able to become pregnant should use effective birth control, such as condoms, during treatment with IMBRUVICA ® and for 1 month after the last dose. are breastfeeding or plan to breastfeed. Do not breastfeed during treatment with IMBRUVICA ® and for 1 week after the last dose. Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking IMBRUVICA ® with certain other medicines may affect how IMBRUVICA ® works and can cause side effects. How should I take IMBRUVICA ® ? Take IMBRUVICA ® exactly as your healthcare provider tells you to take it. Take IMBRUVICA ® 1 time a day at about the same time each day. IMBRUVICA ® comes as capsules, tablets, and oral suspension. If your healthcare provider prescribes IMBRUVICA ® capsules or tablets: Swallow IMBRUVICA ® capsules or tablets whole with a glass of water. Do not open, break, or chew IMBRUVICA ® capsules. Do not cut, crush, or chew IMBRUVICA ® tablets. If your healthcare provider prescribes IMBRUVICA ® oral suspension: See the detailed Instructions for Use that comes with IMBRUVICA ® oral suspension for information about the correct way to give a dose to your child. If you have questions about how to give IMBRUVICA ® oral suspension, talk to your healthcare provider. Do not use if the carton seal is broken or missing. If you miss a dose of IMBRUVICA ® take it as soon as you remember on the same day. Take your next dose of IMBRUVICA ® at your regular time on the next day. Do not take extra doses of IMBRUVICA ® to make up for a missed dose. If you take too much IMBRUVICA ® call your healthcare provider or go to the nearest hospital emergency room right away. What should I avoid while taking IMBRUVICA ® ? You should not drink grapefruit juice, eat grapefruit, or eat Seville oranges (often used in marmalades) during treatment with IMBRUVICA ® . These products may increase the amount of IMBRUVICA ® in your blood. What are the possible side effects of IMBRUVICA ® ? IMBRUVICA ® may cause serious side effects, including: Bleeding problems (hemorrhage) are common during treatment with IMBRUVICA ® , and can also be serious and may lead to death. Your risk of bleeding may increase if you are also taking a blood thinner medicine. Tell your healthcare provider if you have any signs of bleeding, including: blood in your stools or black stools (looks like tar), pink or brown urine, unexpected bleeding, or bleeding that is severe or that you cannot control, vomit blood or vomit looks like coffee grounds, cough up blood or blood clots, increased bruising, dizziness, weakness, confusion, change in your speech, or a headache that lasts a long time or severe headache. Infections can happen during treatment with IMBRUVICA ® . These infections can be serious and may lead to death. Tell your healthcare provider right away if you have fever, chills, weakness, confusion, or other signs or symptoms of an infection during treatment with IMBRUVICA ® . Heart problems. Serious heart rhythm problems (ventricular arrhythmias, atrial fibrillation and atrial flutter), heart failure and death have happened in people treated with IMBRUVICA ® , especially in people who have an infection, an increased risk for heart disease, or have had heart rhythm problems in the past. Your heart function will be checked before and during treatment with IMBRUVICA ® . Tell your healthcare provider if you get any symptoms of heart problems, such as feeling as if your heart is beating fast and irregular, lightheadedness, dizziness, shortness of breath, swelling of the feet, ankles or legs, chest discomfort, or you faint. If you develop any of these symptoms, your healthcare provider may do tests to check your heart and may change your IMBRUVICA ® dose. High blood pressure (hypertension). New or worsening high blood pressure has happened in people treated with IMBRUVICA ® . Your healthcare provider may start you on blood pressure medicine or change current medicines to treat your blood pressure. Decrease in blood cell counts. Decreased blood counts (white blood cells, platelets, and red blood cells) are common with IMBRUVICA ® , but can also be severe. Your healthcare provider should do monthly blood tests to check your blood counts. Second primary cancers. New cancers have happened during treatment with IMBRUVICA ® , including cancers of the skin or other organs. Tumor lysis syndrome (TLS). TLS is caused by the fast breakdown of cancer cells. TLS can cause kidney failure and the need for dialysis treatment, abnormal heart rhythm, seizure, and sometimes death. Your healthcare provider may do blood tests to check you for TLS. The most common side effects of IMBRUVICA ® in adults with B-cell malignancies (MCL, CLL/SLL, WM and MZL) include: · diarrhea · tiredness · muscle and bone pain · rash · bruising The most common side effects of IMBRUVICA ® in adults or children 1 year of age and older with cGVHD include: · tiredness · low red blood cell count (anemia) · bruising · diarrhea · low platelet count · muscle and joint pain · fever · muscle spasms · mouth sores (stomatitis) · bleeding · nausea · stomach pain · pneumonia · headache Diarrhea is a common side effect in people who take IMBRUVICA ® . Drink plenty of fluids during treatment with IMBRUVICA ® to help reduce your risk of losing too much fluid (dehydration) due to diarrhea. Tell your healthcare provider if you have diarrhea that does not go away. These are not all the possible side effects of IMBRUVICA ® . Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. General information about the safe and effective use of IMBRUVICA ® Medicines are sometimes prescribed for purposes other than those listed in a Patient Information leaflet. Do not use IMBRUVICA ® for a condition for which it was not prescribed. Do not give IMBRUVICA ® to other people, even if they have the same symptoms that you have. It may harm them. You can ask your pharmacist or healthcare provider for information about IMBRUVICA ® that is written for health professionals. Please click here for full Prescribing Information. About VENCLYXTA ® (venetoclax) VENCLYXTA ® (venetoclax) is a first-in-class medicine that selectively binds and inhibits the B-cell lymphoma-2 (BCL-2) protein. In some blood cancers, BCL-2 prevents cancer cells from undergoing their natural death or self-destruction process, called apoptosis. VENCLYXTA targets the BCL-2 protein and works to help restore the process of apoptosis. VENCLYXTA is being developed by AbbVie and Roche. It is jointly commercialized by AbbVie and Genentech, a member of the Roche Group, in the U.S. and by AbbVie outside of the U.S. Together, the companies are committed to BCL-2 research and to studying venetoclax in clinical trials across several blood and other cancers. Venetoclax is approved in more than 80 countries, including the U.S. Approved Uses of VENCLEXTA VENCLEXTA is a prescription medicine used: to treat adults with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL). in combination with azacitidine, or decitabine, or low-dose cytarabine to treat adults with newly diagnosed acute myeloid leukemia (AML) who: are 75 years of age or older, or have other medical conditions that prevent the use of standard chemotherapy. It is not known if VENCLEXTA is safe and effective in children. Important Safety Information What is the most important information I should know about VENCLEXTA? VENCLEXTA can cause serious side effects, including: Tumor lysis syndrome (TLS). TLS is caused by the fast breakdown of cancer cells. TLS can cause kidney failure, the need for dialysis treatment, and may lead to death. Your healthcare provider will do tests to check your risk of getting TLS before you start taking VENCLEXTA. You will receive other medicines before starting and during treatment with VENCLEXTA to help reduce your risk of TLS. You may also need to receive intravenous (IV) fluids into your vein. Your healthcare provider will do blood tests to check for TLS when you first start treatment and during treatment with VENCLEXTA. It is important to keep your appointments for blood tests. Tell your healthcare provider right away if you have any symptoms of TLS during treatment with VENCLEXTA, including fever, chills, nausea, vomiting, confusion, shortness of breath, seizures, irregular heartbeat, dark or cloudy urine, unusual tiredness, or muscle or joint pain. Drink plenty of water during treatment with VENCLEXTA to help reduce your risk of getting TLS. Drink 6 to 8 glasses (about 56 ounces total) of water each day, starting 2 days before your first dose, on the day of your first dose of VENCLEXTA, and each time your dose is increased. Your healthcare provider may delay, decrease your dose, or stop treatment with VENCLEXTA if you have side effects. When restarting VENCLEXTA after stopping for 1 week or longer, your healthcare provider may again check for your risk of TLS and change your dose. Who should not take VENCLEXTA? Certain medicines must not be taken when you first start taking VENCLEXTA and while your dose is being slowly increased because of the risk of increased TLS. Tell your healthcare provider about all the medicines you take, including prescription and over-the- counter medicines, vitamins, and herbal supplements. VENCLEXTA and other medicines may affect each other causing serious side effects. Do not start new medicines during treatment with VENCLEXTA without first talking with your healthcare provider. Before taking VENCLEXTA, tell your healthcare provider about all of your medical conditions, including if you: have kidney or liver problems. have problems with your body salts or electrolytes, such as potassium, phosphorus, or calcium. have a history of high uric acid levels in your blood or gout. are scheduled to receive a vaccine. You should not receive a "live vaccine" before, during, or after treatment with VENCLEXTA, until your healthcare provider tells you it is okay. If you are not sure about the type of immunization or vaccine, ask your healthcare provider. These vaccines may not be safe or may not work as well during treatment with VENCLEXTA. are pregnant or plan to become pregnant. VENCLEXTA may harm your unborn baby. If you are able to become pregnant, your healthcare provider should do a pregnancy test before you start treatment with VENCLEXTA, and you should use effective birth control during treatment and for 30 days after the last dose of VENCLEXTA. If you become pregnant or think you are pregnant, tell your healthcare provider right away. are breastfeeding or plan to breastfeed. It is not known if VENCLEXTA passes into your breast milk. Do not breastfeed during treatment with VENCLEXTA and for 1 week after the last dose. What should I avoid while taking VENCLEXTA? You should not drink grapefruit juice or eat grapefruit, Seville oranges (often used in marmalades), or starfruit while you are taking VENCLEXTA. These products may increase the amount of VENCLEXTA in your blood. What are the possible side effects of VENCLEXTA? VENCLEXTA can cause serious side effects, including: Low white blood cell counts (neutropenia). Low white blood cell counts are common with VENCLEXTA, but can also be severe. Your healthcare provider will do blood tests to check your blood counts during treatment with VENCLEXTA and may pause dosing. Infections. Death and serious infections such as pneumonia and blood infection (sepsis) have happened during treatment with VENCLEXTA. Your healthcare provider will closely monitor and treat you right away if you have a fever or any signs of infection during treatment with VENCLEXTA. Tell your healthcare provider right away if you have a fever or any signs of an infection during treatment with VENCLEXTA. The most common side effects of VENCLEXTA when used in combination with obinutuzumab or rituximab or alone in people with CLL or SLL include low white blood cell counts; low platelet counts; low red blood cell counts; diarrhea; nausea; upper respiratory tract infection; cough; muscle and joint pain; tiredness; and swelling of your arms, legs, hands, and feet. The most common side effects of VENCLEXTA in combination with azacitidine or decitabine or low-dose cytarabine in people with AML include nausea; diarrhea; low platelet count; constipation; low white blood cell count; fever with low white blood cell count; tiredness; vomiting; swelling of arms, legs, hands, or feet; fever; infection in lungs; shortness of breath; bleeding; low red blood cell count; rash; stomach (abdominal) pain; infection in your blood; muscle and joint pain; dizziness; cough; sore throat; and low blood pressure. VENCLEXTA may cause fertility problems in males. This may affect your ability to father a child. Talk to your healthcare provider if you have concerns about fertility. These are not all the possible side effects of VENCLEXTA. Call your doctor for medical advice about side effects. You are encouraged to report side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088. If you cannot afford your medication, contact genentech-access.com/patient/brands/venclexta for assistance. About AbbVie in Oncology At AbbVie, we are committed to transforming standards of care for multiple blood cancers while advancing a dynamic pipeline of investigational therapies across a range of cancer types. Our dedicated and experienced team joins forces with innovative partners to accelerate the delivery of potentially breakthrough medicines. We are evaluating more than 20 investigational medicines in over 300 clinical trials across some of the world's most widespread and debilitating cancers. As we work to have a remarkable impact on people's lives, we are committed to exploring solutions to help patients obtain access to our cancer medicines. For more information, please visit https://www.abbvie.com/oncology and our Blood Cancer Press kit page . About AbbVie AbbVie's mission is to discover and deliver innovative medicines that solve serious health issues today and address the medical challenges of tomorrow. We strive to have a remarkable impact on people's lives across several key therapeutic areas: immunology, oncology, neuroscience, eye care, virology, women's health and gastroenterology, in addition to products and services across its Allergan Aesthetics portfolio. For more information about AbbVie, please visit us at www.abbvie.com . Follow @abbvie on, Facebook , Instagram , YouTube and LinkedIn . Forward-Looking Statements Some statements in this news release are, or may be considered, forward-looking statements for purposes of the Private Securities Litigation Reform Act of 1995. The words "believe," "expect," "anticipate," "project" and similar expressions, among others, generally identify forward-looking statements. AbbVie cautions that these forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those indicated in the forward-looking statements. Such risks and uncertainties include, but are not limited to, failure to realize the expected benefits from AbbVie's acquisition of Allergan plc ("Allergan"), failure to promptly and effectively integrate Allergan's businesses, competition from other products, challenges to intellectual property, difficulties inherent in the research and development process, adverse litigation or government action, changes to laws and regulations applicable to our industry and the impact of public health outbreaks, epidemics or pandemics, such as COVID-19. Additional information about the economic, competitive, governmental, technological and other factors that may affect AbbVie's operations is set forth in Item 1A, "Risk Factors," of AbbVie's 2021 Annual Report on Form 10-K, which has been filed with the Securities and Exchange Commission, as updated by its subsequent Quarterly Reports on Form 10-Q. AbbVie undertakes no obligation to release publicly any revisions to forward-looking statements as a result of subsequent events or developments, except as required by law. IMBRUVICA ® is a registered trademark of Pharmacyclics LLC. 1 Allen J., et al. Treatment Outcomes After Undetectable MRD With First-Line Ibrutinib (Ibr) Plus Venetoclax (Ven): Fixed Duration Treatment (Placebo) Versus Continued Ibr With Up to 5 Years Median Follow-up in the CAPTIVATE Study. 2022 American Society of Hematology (ASH) Annual Meeting. December 11, 2022. 2 Neimann C., et al. Residual Disease Kinetics Among Patients with High-Risk Factors Treated with First-Line Fixed-Duration Ibrutinib plus Venetoclax (Ibr+Ven) versus Chlorambucil plus Obinutuzumab (Clb+O): the GLOW Study. 2022 American Society of Hematology Annual Meeting. December 11, 2022. 3 Jacobs R., et. al. Real-World Comparison of Time to Next Treatment for Patients with CLL Initiated on First-line Treatment with Ibrutinib versus Acalabrutinib. American Society of Hematology Congress 2022. 4 Ghia P., et. al. Initiating First-Line (1L) Ibrutinib (Ibr) in Patients (pts) with Chronic Lymphocytic Leukemia (CLL) Improves Overall Survival (OS) Outcomes to Rates Approximating an Age-Matched Population of ≥65 Years. American Society of Hematology Congress 2022. 5 Ghosh N., et. al. Real-World Outcomes With First-Line Ibrutinib (Ibr) Versus Chemoimmunotherapy (CIT) in Patients With Chronic Lymphocytic Leukemia (CLL)/Small Lymphocytic Lymphoma (SLL): Final Analysis Results From the informCLL Registry. American Society of Hematology Congress 2022. 6 Genetics Home Reference. Isolated growth hormone deficiency. http://ghr.nlm.nih.gov/condition/isolated-growth-hormone-deficiency. Accessed November 2022. 7 Turetsky A, et al. Single cell imaging of Bruton's tyrosine kinase using an irreversible inhibitor. Scientific Reports. 2014;6:4782. 8 de Rooij MF, Kuil A, Geest CR, et al. The clinically active BTK inhibitor PCI-32765 targets B-cell receptor- and chemokine-controlled adhesion and migration in chronic lymphocytic leukemia. Blood. 2012;119(11):2590-2594. 9 IMBRUVICA® U.S. Prescribing Information, August 2022. SOURCE AbbVie

秋高气爽，世间清朗，初遇秋风，如闻天籁。2021年9月4日，在美丽的“人间天堂”杭州，来自五湖四海的血液及肿瘤领域专家学者齐聚一堂，在现场和线上共同出席了恒曲®全国上市会。由恒瑞自主研发的一类创新药物恒曲®（海曲泊帕乙醇胺片），已于2021年6月获批正式上市，是我国首个自主研发且具有ITP和SAA双适应症的TPO-RA， 恒曲®的问世，为国内的ITP、SAA以及其他血小板减少患者提供了一个崭新的治疗选择，以恒曲®为代表的中国创新药物，也将在世界舞台上，奏响中国原研的最强曲！ 本次大会很荣幸地邀请到华中科技大学同济医学院附属协和医院胡豫教授、 北京大学血液研究所黄晓军教授、哈尔滨血液病肿瘤研究所马军教授、浙江大学医学院附属邵逸夫医院潘宏铭教授、南京金陵医院秦叔逵教授、上海交通大学医学院附属瑞金医院沈志祥教授、中国医学科学院肿瘤医院石远凯教授、苏州大学附属第一医院吴德沛教授、中国医学科学院血液病医院王建祥教授、中山大学肿瘤防治中心徐瑞华教授和北京大学肿瘤医院朱军教授共同担任大会主席团成员。 第一乐章 声升奏鸣 第一乐章由哈尔滨血液病肿瘤研究所马军教授担任特邀主持人，马军教授指出，患者的健康幸福是我们奋斗的目标，海曲泊帕的上市，对我国的ITP、SAA以及CIT治疗，意义重大。 山东省肿瘤医院于金明院士致辞道，我们国家这些年在大力推进科技创新，创新不应该是奢侈品，而是必需品。近年来以恒瑞为代表的民族创新企业，打破了发达国家的技术壁垒，让更多的创新好药触手可及。海曲泊帕作为全新一代的小分子非肽类TPO-RA，疗效与安全性俱佳，未来将惠及我国乃至全世界更多血液及肿瘤患者。 南京金陵医院秦叔逵教授赞叹道，海曲泊帕是中国第一个自主研发的小分子非肽类TPO-RA，通过结构改良升级，大大降低了免疫原性，起效快速、效果卓越、安全性高，是TPO-RA类药物中的Best in class，给我国血小板减少疾病的治疗注入了新的活力。 苏州大学附属第一医院吴德沛教授致辞道，在中国两个一百年的交汇点上，海曲泊帕这样的国产原研创新药物的上市，是非常值得庆贺的事情。临床上血小板减少相当常见，希望我国的医务工作者能够用好这个药物，造福更多的老百姓。 浙江大学医学院附属邵逸夫医院潘宏铭教授对来杭州参会的同道们表示感谢，潘宏铭教授对海曲泊帕长达十几年的曲折经历和坚持不懈表示赞赏，并预祝本次全国上市会圆满举办。 华中科技大学同济医学院附属协和医院胡豫教授指出，海曲泊帕克服了同类药物价格高、负担重等缺点，希望恒瑞可以生产出更多更好的创新药物，让更多的血液病患者有药可医。 中山大学肿瘤防治中心徐瑞华教授认为海曲泊帕的成功上市，对我国的健康事业发展具有积极推动作用，也将助力健康中国2030伟大战略行动的推进。 恒瑞医药股份有限公司董事长孙飘扬先生代表恒瑞医药对现场和线上专家表示最热烈欢迎和最衷心感谢。孙飘扬先生感慨地说道，作为创新性民族制药企业，恒瑞一直秉承科技为本、为人类创造健康生活的使命。海曲泊帕是恒瑞自主研发的1类创新药物，也是公司自主研发上市的第8个新药。经过十余年的努力，在国家政府以及领域内各位专家的大力支持之下，海曲泊帕如今终于正式上市，无论是更优化的分子结构、还是更丰富的样本量和中国临床数据，海曲泊帕无疑都是best in class。未来，恒瑞将不断研发更多血液病创新药物，为造福人类健康而努力。 第二乐章 强音溯源 本环节由浙江大学医学院附属第一医院金洁教授、中国科学院大学附属肿瘤医院范云教授、哈尔滨医科大学附属肿瘤医院张清媛教授以及河南省肿瘤医院罗素霞教授共同主持。 中山大学肿瘤防治中心黄慧强教授为大家分享了促血小板生成药物的发展历程。 恒瑞医药副总经理兼首席医学官邹建军博士，为大家介绍了了首个中国自主研发TPO-RA（海曲泊帕）的过去、现在和未来。 第二部分的名家访谈环节由广州南方医科大学南方医院冯茹教授和中国科学院大学附属肿瘤医院王晓稼教授共同主持。江苏省人民医院何广胜教授，中国科学院大学附属肿瘤医院楼寒梅教授、河北医科大学第四医院史健教授以及河南省肿瘤医院周虎教授共同参与讨论。 随后，大会主席团成员北大血研所黄晓军、中国医学科学院血液病医院王建祥教授和北大肿瘤医院朱军教授先后为海曲泊帕上市给予了殷切寄语，几位专家一致认为，海曲泊帕疗效好、副反应少，疗效快，为我国广大患者提供了新的治疗选择，并希望未来能有更多用得起的中国优质原研药物，惠及更多中国患者。 第三乐章 三重合奏 第三环节由山东大学齐鲁医院侯明教授、广西医科大学附属肿瘤医院李力教授、四川大学华西医院牛挺教授、陆军军医大学第二附属医院张曦教授和兰州大学第二医院张连生教授共同主持。 中国医学科学院血液病医院杨仁池教授为大家分享了海曲泊帕ITP注册临床研究解读。 中国医学科学院血液病医院张凤奎教授为大家分享了SAA的研究解读。 哈尔滨血液肿瘤研究所赵东陆教授为大家分享了CIT的疾病特点与研究进展。 本环节的大咖共话由浙江大学医学院附属第二医院钱文斌教授和福建医科大学附属协和医院沈建箴教授共同主持，由中南大学湘雅三医院李昕教授、中国人民解放军总医院靖彧教授、华中科技大学同济医学院附属协和医院梅恒教授、浙江中医药大学附属第一医院周郁鸿教授共同参与讨论。 第四乐章 曲艺新程 最后一个环节，马军教授为大家进行了海曲泊帕CIT注册研究的背景介绍，马教授指出， CIT防治现状目前还存在很多不足，临床亟需一种突破传统治疗、迅速提升血小板计数、安全且服用方便的促血小板生成药物。 会议接近尾声，两位血液及肿瘤领域的大咖发表了简明扼要的大会总结。中国医学科学院肿瘤医院石远凯教授总结道，今天的大会通过海曲泊帕的研发历程和应用疾病领域，我们看到了这样一个创新药物，如何从实验室走向临床，为ITP、SAA等疾病带来新的治疗选择，也正是恒瑞的坚持，我们才看到了越来越多中国创新药物走向临床造福患者。 上海交通大学医学院附属瑞金医院沈志祥教授总结了三个重点。第一点，医生最怕患者颅内或中枢出血，这类出血非常危急，可能迅速夺走病人的生命，是目前面临的最严重的一个问题；第二点，海曲泊帕上市了，疗效非常理想，ITP连用8周，三分之二可以好转，SAA、CIT治疗效果及安全性都很好，肝毒性很低，现在我们最大的任务就是让它广泛应用，多进行真实世界的研究，使所有需要的病人都能快速得到救治；第三点，这是恒瑞第8个一类新药，恒瑞是国内创新药企的领头羊，但现在的创新药物还是远远不够的，我们要有雄心，去开发更多更好的中国原研药物，希望中国新药的声音可以响彻世界。