Plerixafor

作者 | 张自然博士 编辑 | 相宜 6月28日，国家医保局关于公布《2024年国家基本医疗保险、工伤保险和生育保险药品目录调整工作方案》及申报指南等文件的公告（下文简称《方案》）发布，标志着2024年医保谈判正式启动。 按《方案》目录外西药和中成药条件1（2019.1.1～2024.6.30批准上市且尚未国谈的新通用名药品），截至6月23日，共有213个上市新药符合条件1，其中，独家品种189个（占89%）、非独家品种24个（11%）。这213个新品种共涉及388张批文，其中，化药260张（占2/3）、生物制品115张（占近1/3）、中药13张（占1/30）。 注：本文中的企业均指上市许可持有人。 01 批准时间 这388个批文，共分布在6个年头批准，最多的当然是2023年，尚有132个当年批准上市的新药尚未国谈，有资格参与2024年国谈，占了1/3。 最少的是2019年，只有19个新药。2020～2022年依次是38个、46个、83个新药。2024.1.1～24.6.23，批准上市新药70个，将首次参加2024年国谈。 02 非独家品种 符合目录外条件1的24个非独家品种，共涉及130个批文、96家企业。 涉及企业最多的品种是注射用尼可地尔，共涉及15家企业，即石家庄四药、仁合益康、扬子江、成都苑东、北京四环科宝、苏州特瑞、陕西丽彩、天士力之骄、天大药业、海口天行健、江苏博欣医药科技、南京恒道医药科技、云南先施药业、重庆德诚永道医药等14家本土药企，以及1家外企（日本中外制药株式会社）； 其次是涉及10～15家企业的有3个品种，即注射用比伐芦定（11家）、盐酸非索非那定片（11家）、普卢利沙星片（10家）； 涉及5～10家企业的有4个品种，即四价流感病毒裂解疫苗（8家）、富马酸卢帕他定片（6家）、ACYW135群脑膜炎球菌多糖疫苗（6家）、普乐沙福注射液（5家）； 涉及3家企业的有2个品种，即盐酸决奈达隆片、ω-3脂肪酸乙酯90软胶囊。 03 Top企业 一、独家品种Top企业 189个独家品种共涉及132家企业（本土企业69家、外企63家），中、外企业数量基本持平。其中，拥有2家独家品种的企业共有27家，外企20家（约占3/4）、本土7家（约占1/4）。 拥有独家品种数最多的企业是罗氏，共有9个独家新药符合目录外条件1，如法瑞西单抗注射液、格菲妥单抗注射液、可伐利单抗注射液、利妥昔单抗注射液（皮下注射）、玛巴洛沙韦干混悬剂、帕妥珠曲妥珠单抗注射液(皮下注射)、注射用维泊妥珠单抗、阿替利珠单抗注射液、托珠单抗注射液(皮下注射)； 有5个独家品种的有4家企业，即武田、施贵宝、恒瑞、百特； 有4个独家品种的有2家企业，即礼来、辉瑞； 有3个独家品种的有7家企业，即浙江我武生物、日本协和发酵麒麟、强生、诺华、默沙东、美国健赞、Swedish Orphan Biovitrum AB； 有2个独家品种的有13家企业，即兆科、远大在澳洲设立的创新药研发中心、卫材、康希诺、江西青峰药业、葛兰素、东丽株式会社、北京明海生物、安进、阿斯利康、ViiV Healthcare B.V.、NextWave Pharmaceuticals Inc、Napp Pharmaceuticals Limited。 二、非独家品种Top企业 24个非独家品种共涉及96家企业，以本土企业为主，涉及本土企业79家（82%）、外企17家（占18%）。 其中，拥有非独家品种数≥2个的企业共10家，本土企业8家，外企2家（赛诺菲和雅培），除扬子江有3个品种外，其他都有2个品种。 扬子江的3个品种分别是注射用尼可地尔、富马酸卢帕他定片、注射用比伐芦定。 其他如北京四环科宝（注射用尼可地尔、富马酸卢帕他定片）、成都盛迪（盐酸非索非那定片、ω-3脂肪酸乙酯90软胶囊）、华兰生物（四价流感病毒裂解疫苗、ACYW135群脑膜炎球菌多糖疫苗）、厦门恩成制药（艾司奥美拉唑镁碳酸氢钠胶囊、兰索拉唑碳酸氢钠胶囊）、石药集团欧意（盐酸决奈达隆片、盐酸伊立替康脂质体注射液）、四川国为制药（ω-3脂肪酸乙酯90软胶囊、二十碳五烯酸乙酯软胶囊）、重庆科瑞制药（普卢利沙星片、盐酸非索非那定片）。 END 内容沟通：郑瑶（13810174402） 医药代理商产品交流群 扫描下方二维码加入 医药代表交流群 扫描下方二维码加入 左下角「关注账号」，右下角「在看」，防止失联

- Investigator-initiated study includes leading researchers in SCD gene therapy clinical development from St. Jude Children's Research Hospital, Inc. and two other clinical sites - - New trial to expand ongoing clinical research of motixafortide for mobilization of HSCs in patients with SCD - TEL AVIV, Israel, May 30, 2024 /PRNewswire/ -- BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a commercial stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases, today announced a multi-center Phase 1 clinical trial sponsored by St. Jude Children's Research Hospital, Inc. to evaluate motixafortide for the mobilization of CD34+ hematopoietic stem cells (HSCs) used in the development of gene therapies for patients with sickle cell disease (SCD). Investigators in the trial from St. Jude Children's Research Hospital, Inc. and two other clinical sites have extensive SCD gene therapy clinical development experience and are recognized leaders in the field. Hematopoietic stem cell transplantation after genetic modification is potentially curative for patients with SCD. Significant quantities of HSCs (minimum 16.5-20 million cells/kg) are required for genetic manipulation and transplant success, however, the most commonly used drug for collection of stem cells, granulocyte colony-stimulating factor (G-CSF), is contraindicated in patients with SCD. Peripheral blood mobilization of stem cells using the mobilization agent plerixafor is the current strategy to collect HSCs for SCD gene therapies, however limitations exist including the need for multiple collection cycles to achieve the necessary HSC yields. For some, gene therapy may be prohibitive by the failure to obtain adequate numbers of HSCs. "The recent FDA approvals of two gene therapies for sickle cell disease in the U.S. is an exciting development for the sickle cell community," said Ella Sorani, PhD, Chief Development Officer at BioLineRx. "Through this new trial with St. Jude, and our ongoing collaboration with investigators at Washington University School of Medicine in St. Louis, we are excited to be working with leaders in gene therapy and stem cell mobilization to evaluate potential new mobilization options for patients with SCD." Enrollment in the St. Jude Children's Research Hospital, Inc. study is expected to begin in the next few months. Initial data from the Washington University School of Medicine in St. Louis sponsored clinical trial (ClinicalTrials.gov Identifier: NCT05618301) is anticipated in the second half of 2024. Motixafortide, BioLineRx's lead therapeutic candidate, was approved by the U.S. Food & Drug Administration (FDA) in September 2023, in combination with filgrastim (G-CSF), to mobilize HSCs for collection and subsequent autologous transplantation in patients with multiple myeloma, under the brand name APHEXDA®. About SCDSTEMM Clinical Trial with St. Jude Children's Research Hospital Inc. The SCDSTEMM (Sickle Cell Disease Stem Cell Mobilization and Apheresis Using Motixafortide) Phase 1 clinical trial is an open-label, multi-center study evaluating the safety, tolerability, and feasibility of single-agent motixafortide (CXCR4 inhibitor) for the mobilization and collection of CD34+ HSCs in 12 patients (aged 18 and older) with SCD. The trial's primary objective is to assess the safety and tolerability of motixafortide in SCD patients, as determined by the incidence of adverse events. Secondary objectives include understanding CD34+ kinetics after motixafortide administration in patients with SCD and determining the number of CD34+ HSCs collected via leukapheresis. The study is designed in two parts: Part A (N=6) will evaluate single dose motixafortide mobilization followed by one apheresis session; Part B (N=6) will evaluate daily motixafortide administration over a two-day mobilization and apheresis regimen. Additional objectives include phenotype and cell function characterization, as well as assessment of the gene modifying potential and senescence of CD34+ cells. About Sickle Cell Disease SCD is one of the most common genetic diseases globally, affecting millions of people throughout the world and disproportionately impacting persons of color. SCD arises from mutations in the hemoglobin gene, ultimately leading to the production of abnormally shaped (sickle) red blood cells. The clinical manifestations of SCD include anemia and blood vessel occlusion which can lead to both acute and chronic pain, as well as tissue ischemia across multiple organ systems (e.g., brain, lungs, heart, kidneys, spleen, liver, bones), ultimately compromising end organ function. The cumulative impact of these complications significantly impacts morbidity and mortality for patients with SCD. About BioLineRx BioLineRx Ltd. (NASDAQ/TASE: BLRX) is a commercial stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases. The company's first approved product is APHEXDA® (motixafortide) with an indication in the U.S. for stem cell mobilization for autologous transplantation in multiple myeloma. BioLineRx is advancing a pipeline of investigational medicines for patients with sickle cell disease, pancreatic cancer, and other solid tumors. Headquartered in Israel, and with operations in the U.S., the company is driving innovative therapeutics with end-to-end expertise in development and commercialization, ensuring life-changing discoveries move beyond the bench to the bedside. Learn more about who we are, what we do, and how we do it at , or on Twitter and LinkedIn.  Forward Looking Statement Various statements in this release concerning BioLineRx's future expectations constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include words such as "anticipates," "believes," "could," "estimates," "expects," "intends," "may," "plans," "potential," "predicts," "projects," "should," "will," and "would," and describe opinions about future events. These include statements regarding management's expectations, beliefs and intentions regarding, among other things, expectations with regard to clinical trials of motixafortide. These forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause the actual results, performance or achievements of BioLineRx to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Factors that could cause BioLineRx's actual results to differ materially from those expressed or implied in such forward-looking statements include, but are not limited to: the initiation, timing, progress and results of BioLineRx's preclinical studies, clinical trials, and other therapeutic candidate development efforts; BioLineRx's ability to advance its therapeutic candidates into clinical trials or to successfully complete its preclinical studies or clinical trials; whether BioLineRx's collaboration partners will be able to execute on collaboration goals in a timely manner; whether the clinical trial results for APHEXDA will be predictive of real-world results; BioLineRx's receipt of regulatory approvals for its therapeutic candidates, and the timing of other regulatory filings and approvals; the clinical development, commercialization and market acceptance of BioLineRx's therapeutic candidates, including the degree and pace of market uptake of APHEXDA for the mobilization of hematopoietic stem cells for autologous transplantation in multiple myeloma patients; whether access to APHEXDA is achieved in a commercially viable manner and whether APHEXDA receives adequate reimbursement from third-party payors; BioLineRx's ability to establish, operationalize and maintain corporate collaborations; BioLineRx's ability to integrate new therapeutic candidates and new personnel; the interpretation of the properties and characteristics of BioLineRx's therapeutic candidates and of the results obtained with its therapeutic candidates in preclinical studies or clinical trials; the implementation of BioLineRx's business model and strategic plans for its business and therapeutic candidates; the scope of protection BioLineRx is able to establish and maintain for intellectual property rights covering its therapeutic candidates and its ability to operate its business without infringing the intellectual property rights of others; estimates of BioLineRx's expenses, future revenues, capital requirements and its needs for and ability to access sufficient additional financing, including any unexpected costs or delays in the commercial launch of APHEXDA; risks related to changes in healthcare laws, rules and regulations in the United States or elsewhere; competitive companies, technologies and BioLineRx's industry; statements as to the impact of the political and security situation in Israel on BioLineRx's business; and the impact of the COVID-19 pandemic, the Russian invasion of Ukraine, the declared war by Israel against Hamas and the military campaigns against Hamas and other terrorist organizations, which may exacerbate the magnitude of the factors discussed above. These and other factors are more fully discussed in the "Risk Factors" section of BioLineRx's most recent annual report on Form 20-F filed with the Securities and Exchange Commission on March 26, 2024. In addition, any forward-looking statements represent BioLineRx's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. BioLineRx does not assume any obligation to update any forward-looking statements unless required by law. Contacts: United States John Lacey BioLineRx [email protected] Israel Moran Meir LifeSci Advisors, LLC [email protected] Logo - SOURCE BioLineRx Ltd.