BeOne Medicines Ltd.

Preliminary results highlight anti-tumor activity and favorable safety profiles of both B7-H4-targeting ADC and CDK2 inhibitor Data underscore strength of emerging breast cancer pipeline as part of BeOne’s global transformation with next wave of innovation SAN CARLOS, CA, USA I June 02, 2025 I BeOne Medicines Ltd. (NASDAQ: ONC; HKEX: 06160; SSE: 688235), a global oncology company, announced new clinical data from its emerging breast cancer pipeline at the American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago. Poster presentations feature preliminary results of the dose escalation studies of two investigational molecules: BG-C9074, a novel B7-H4-targeting antibody-drug conjugate (ADC) in patients with advanced solid tumors, including breast cancer, and BG-68501, a cyclin-dependent kinase-2 inhibitor (CDK2i), in HR+/HER2- breast cancer patients with prior CDK4/6i exposure. “Presenting the first clinical data for two novel breast cancer candidates at ASCO 2025 marks a pivotal moment for BeOne,” said Mark Lanasa, M.D., Ph.D., Chief Medical Officer, Solid Tumors at BeOne. “These early results highlight the strong potential of our B7-H4-targeting ADC and CDK2 inhibitor to address critical gaps in breast cancer treatment. Alongside our advancing CDK4 inhibitor, they represent just the beginning of a pipeline built on targeted, biology-driven innovation. As we debut our new identity as BeOne, this milestone reflects the momentum behind our science and our commitment to delivering impactful therapies to cancer patients worldwide.” BeOne is advancing a robust pipeline of differentiated investigational medicines for breast cancer that may both effectively combat the disease and potentially improve quality of life for patients receiving treatment. BG-C9074, a B7-H4-targeting ADC ( Abstract #3033 ) BeOne presented initial results of the ongoing first-in-human, Phase 1a dose escalation study of BG-C9074 monotherapy in 78 patients with advanced solid tumors, of which more than a quarter were breast cancer patients. BG-C9074, an investigational topoisomerase I inhibitor ADC that targets the B7-H4 protein, which is broadly expressed in breast and gynecologic cancers, is designed with an innovative drug linker to deliver a potent cancer-killing drug directly to the cancer cells. With limited follow-up among the 56 efficacy-evaluable patients, preliminary clinical responses were observed at multiple dose levels across various tumor types without selection for B7-H4 expression in these heavily pretreated patients. Confirmed overall response rate (ORR) was 16.1% (9/56; 95% CI: 7.6%–28.3%), with 9 confirmed partial responses; unconfirmed ORR was 25.0% (14/56; 14.4%-38.4%) (n=14 partial responses). Confirmed disease control rate (DCR) was 73.2% (59.7%-84.2%) and confirmed clinical benefit rate (CBR) was 17.9% (8.9%-30.4%). Pharmacokinetics (PK) were observed to be approximately dose-proportional across dose levels. BG-C9074 showed a manageable safety and tolerability profile in patients with B7-H4 advanced solid tumors, including breast cancer. There were 5 dose-limiting toxicities (DLTs) reported among 3 dose levels, all related to treatment: grade 3 fatigue (n=1); grade 3 febrile neutropenia (n=2); and grade 4 platelet count decreased (n=2). The most common treatment-emergent adverse events (TEAEs) were nausea, fatigue, and neutropenia*. The most common grade ≥3 TEAEs were neutropenia and thrombocytopenia†. There were no TEAEs leading to treatment discontinuation or death. These data support the continued development of BG-C9074 in patients with advanced solid tumors. ( NCT06233942 ) BG-68501, a CDK2 inhibitor ( Abstract# 3115 ) Dose-escalation data from the first-in-human, Phase 1a study of a novel CDK2 inhibitor, BG-68501, were presented as a poster today. BG-68501 is designed to address elevated CDK2 activity as well as cyclin E1-driven upregulation, two key resistance mechanisms that often limit the effectiveness of CDK4/6 inhibitors in treating HR+/HER2- breast cancer. CDK inhibitors target checkpoint proteins that control cell division to stop the growth of cancer cells. A total of 57 enrolled patients with advanced solid tumors, including 19 patients with HR+/HER2- metastatic breast cancer, received BG-68501 as monotherapy or in combination with fulvestrant in escalating dose cohorts (all received prior CDK4/6i). Of the 37 efficacy-evaluable patients (all with monotherapy), unconfirmed overall response rate (ORR) was 5.4% (2/37; 95% CI: 0.7%–18.2%). Two extensively pretreated patients (5.4%) experienced unconfirmed partial response (PR), 15 patients (40.5%) had stable disease (SD), 15 patients (40.5%) had progressive disease (PD), and 5 patients (13.5%) were not evaluable/not assessed. Of the 2 patients with PR, both were breast cancer patients, and one was ongoing with treatment at the time of data cutoff, while the other had discontinued treatment. Unconfirmed clinical benefit rate (CBR) was 8.1% (3/37; 95% CI: 1.7%-21.9%) and unconfirmed disease control rate (DCR) was 45.9% (17/37; 95% CI: 29.5%-63.1%). BG-68501 demonstrated a linear PK profile consistent with preclinical data and signs of pharmacodynamic responses. BG-68501 demonstrated a manageable safety and tolerability profile, with no DLTs observed to date during dose escalation. The most common TEAEs were vomiting, nausea, and fatigue, and TEAEs leading to treatment discontinuation occurred in 4 patients (7%) across all dose levels. There were no TEAEs leading to death. The data support continued development of BG-68501 as a next-line option for tumors with CDK2 dependency. ( NCT06257264 ) For additional information about our presence at the 2025 ASCO Annual Meeting, please visit our meeting hub: congress.beonemedicines.com . BeOne will host an investor R&D Day on June 26 at 8:30 am ET covering its deep and broad global innovation pipeline and platforms, as well as the Company’s vision, differentiated capabilities, and value creation drivers. A live webcast will be accessible from the investors section of BeOne’s website at https://ir.beonemedicines.com , https://hkexir.beonemedicines.com , or https://sseir.beonemedicines.com . An archived replay will be available for 90 days following the event. About Our Breast Cancer Pipeline BeOne is advancing a robust portfolio of investigational medicines for breast cancer, including three molecules in clinical development – two cyclin-dependent kinase (CDK) inhibitors, BGB-43395, a CDK4 inhibitor, and BG-68501, a CDK2 inhibitor, and an antibody-drug conjugate (ADC), BG-C9074. BeOne also plans to evaluate the potential of BCL2 inhibition in breast cancer, with next-generation BCL2 inhibitor, BGB-21447, expected to begin clinical testing in solid tumor indications soon. Multispecific antibodies and targeted protein degraders with potential applications in breast cancer are among the preclinical assets being developed. About Breast Cancer Breast cancer accounts for close to one in four cancer cases and one in six cancer deaths in women worldwide. 1 Globally, breast cancer is the second most common cancer and the fourth highest cause of cancer mortality as well as the leading cause of cancer death in women. 1 More than 2.3 million patients were diagnosed with breast cancer in 2022, and over 666,000 deaths were reported globally. 1 Approximately two-thirds of breast cancer cases are the HR+/HER2- subtype. 2 About BeOne BeOne Medicines is a global oncology company domiciled in Switzerland that is discovering and developing innovative treatments that are more affordable and accessible to cancer patients worldwide. With a portfolio spanning hematology and solid tumors, BeOne is expediting development of its diverse pipeline of novel therapeutics through its internal capabilities and collaborations. With a growing global team of more than 11,000 colleagues spanning six continents, the Company is committed to radically improving access to medicines for far more patients who need them. To learn more about BeOne, please visit www.beonemedicines.com and follow us on LinkedIn , X , Facebook and Instagram . To access BeOne media resources, please visit our News & Media site. *Neutropenia was defined by a custom MedDRA basket with neutropenia and neutrophil count decrease preferred terms. †Thrombocytopenia was defined by a custom MedDRA basket with thrombocytopenia and platelet count decreased preferred terms. 1 Bray F, et al. Global cancer statistics 2022: GLOBOCAN estimates of incidence and mortality worldwide for 36 cancers in 185 countries. CA Cancer J Clin . April 4, 2024. https://doi-org.libproxy1.nus.edu.sg/10.3322/caac.21834 2 National Cancer Institute, SEER. Cancer Stat Facts: Female Breast Cancer Subtypes. https://seer.cancer.gov/statfacts/html/breast-subtypes.html Accessed November 11, 2024. SOURCE: Be One Medicines

Phase 2 trial results continue to show clinically meaningful efficacy and durable responses, including 36.5-month median overall survival after four years of follow-up, with a manageable safety profile Findings presented today at ASCO 2025 and concurrently published in The Lancet Oncology For U.S. media and investors only DUBLIN, June 2, 2025 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced long-term data, including the first report of median overall survival (OS) from the Phase 2 trial evaluating Ziihera® (zanidatamab-hrii), a dual HER2-targeted bispecific antibody, in combination with chemotherapy for the investigational use in first-line HER2-positive (IHC 3+ or IHC 2+/FISH+) locally advanced nonresectable gastroesophageal adenocarcinoma (mGEA). The data were featured as a rapid oral presentation at the American Society of Clinical Oncology (ASCO) Annual Meeting, and the results were concurrently published in The Lancet Oncology. Among 41 patients with centrally confirmed HER2-positive tumors, treatment with Ziihera in combination with physician's choice of chemotherapy resulted in a median progression-free survival (PFS) of 15.2 months [95% CI: 9.5, 33.4], and a median overall survival (OS) of 36.5 months [95% CI: 23.6, not estimable (NE)]. Median PFS remained stable with the additional four-year follow-up, consistent with previously reported results. Among all 46 patients in the study with HER2-expressing mGEA, median PFS was 12.5 months [95% CI: 8.2, 21.8], and median OS also reached 36.5 months [95% CI: 23.6, NE], with the longest observed survival at 57.9 months (censored at data cutoff). Long-term follow-up also demonstrated low discontinuation rates, with no new safety signals observed. "Gastroesophageal adenocarcinoma remains a highly aggressive cancer with a poor prognosis, even with currently available treatment options," said Dr. Elena Elimova, lead trial investigator and a medical oncologist at Princess Margaret Cancer Centre, Toronto, Canada. "The long-term survival outcomes presented today at ASCO demonstrate the sustained antitumor activity achieved with zanidatamab plus chemotherapy over four years of follow-up. These results are especially encouraging given the high unmet need for better first-line treatment options for this patient population." "These long-term survival data from our Phase 2 trial build on previously reported results and further strengthen our belief in Ziihera as a transformative treatment option for patients with HER2-positive disease," said Rob Iannone, M.D., M.S.C.E., executive vice president, global head of research and development, and chief medical officer of Jazz Pharmaceuticals. "An estimated median overall survival of 36.5 months in this patient cohort is very encouraging given recent observations with standard of care regimens in similar populations, where median survival has typically ranged from 15 to 20 months. The sustained 15.2-month progression-free survival in the centrally confirmed HER2-positive subgroup after four years is a meaningful indicator of durable clinical benefit. We look forward to the top line results of the pivotal Phase 3 HERIZON-GEA-01 trial later this year and remain committed to advancing Ziihera across multiple tumor types." Phase 2 mGEA Trial Results The data include four-year follow-up and the first report of median OS from an ongoing, open-label Phase 2 trial (NCT03929666) evaluating Ziihera in combination with chemotherapy as a first-line treatment for patients with HER2-expressing mGEA, which includes gastric, esophageal and gastroesophageal junction (GEJ) adenocarcinomas. Patients had not received prior HER2-targeted agents nor systemic treatment for mGEA. A total of 46 patients with HER2-expressing mGEA (41 patients with centrally confirmed HER2-positive mGEA) were enrolled from 14 sites across the United States, Canada and South Korea. Patients received Ziihera with physician's choice of chemotherapy, including fluoropyrimidine maintenance regimens. Chemotherapy-based regimens remain the current standard first-line treatment for mGEA. The longer-term data (median duration of follow-up of 48 months [range, 29-59]) demonstrate the promising antitumor activity of Ziihera combined with chemotherapy as a first-line treatment for HER2-positive mGEA. In a post-hoc subgroup analysis of the 41 treated patients with centrally confirmed HER2-positive tumors, median PFS was 15.2 months [95% CI: 9.5, 33.4], and median OS was 36.5 months [95% CI: 23.6, NE]. These survival outcomes were consistent with prior analyses, with PFS durability maintained at the four-year follow-up. The confirmed objective response rate (cORR), the study's primary endpoint, was 83.8% [95% CI: 68.0, 93.8], and median duration of response (DOR) was 20.4 months [95% CI: 8.3, 44.1]. These results further support the observed clinical benefit in this centrally confirmed population. Among all 46 patients in the study, median PFS was 12.5 months [95% CI: 8.2, 21.8], and the estimated 24-month PFS rate was 31% [95% CI: 17%, 46%]. Median OS was also 36.5 months [95% CI: 23.6, NE], with an estimated 24-month OS rate of 65% [95% CI: 49%, 77%]. The cORR was 76.2% [95% CI: 60.5, 87.9], and median DOR was 18.7 months [95% CI: 10.4, 44.1]. With additional follow-up, the safety and tolerability profile of Ziihera plus chemotherapy showed low discontinuation rates, with no new safety signals identified. Diarrhea (39%) and hypokalemia (22%) were the most common Grade 3-4 treatment-related adverse events (TRAEs); the incidence of Grade 3 diarrhea was reduced from 52% to 24% for patients enrolled after the implementation of mandated antidiarrheal prophylaxis. There were no treatment-related deaths. Five patients discontinued Ziihera due to TRAEs. Ongoing Phase 3 Trial The Phase 3 randomized clinical trial, HERIZON-GEA-01 (NCT05152147), evaluating Ziihera in combination with standard of care chemotherapy with and without the addition of a PD-1 agent as a first-line treatment for HER2-expressing mGEA is currently underway. This is an events-based trial, and top-line results are expected to read out in the second half of 2025. About Gastroesophageal Adenocarcinoma Gastroesophageal adenocarcinoma (GEA) is the fifth most common cancer worldwide, and approximately 20% of patients have HER2-positive disease.i,ii,iii HER2-positive GEA has high morbidity and mortality, and patients are urgently in need of new treatment options. The overall prognosis for patients with GEA remains poor, with a global five-year survival rate of less than 30 percent for gastric cancer and about 19 percent for GEA.iv About Ziihera® (zanidatamab-hrii) Ziihera (zanidatamab-hrii) is a bispecific HER2-directed antibody that binds to two extracellular sites on HER2. Binding of zanidatamab-hrii with HER2 results in internalization leading to a reduction in HER2 expression of the receptor on the tumor cell surface. Zanidatamab-hrii induces complement-dependent cytotoxicity (CDC), antibody-dependent cellular cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP). These mechanisms result in tumor growth inhibition and cell death in vitro and in vivo.v In the United States, Ziihera is indicated for the treatment of adults with previously treated, unresectable or metastatic HER2-positive (IHC 3+) biliary tract cancer (BTC), as detected by an FDA-approved test.v The U.S. Food and Drug Administration (FDA) granted accelerated approval for this indication based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s). v Zanidatamab is being developed in multiple clinical trials as a targeted treatment option for patients with solid tumors that express HER2. Zanidatamab is being developed by Jazz and BeiGene, Ltd. (BeiGene) under license agreements from Zymeworks, which first developed the molecule. The FDA granted Breakthrough Therapy designation for zanidatamab development in patients with previously treated HER2 gene-amplified BTC, and two Fast Track designations for zanidatamab: one as a single agent for refractory BTC and one in combination with standard-of-care chemotherapy for 1L gastroesophageal adenocarcinoma (GEA). Additionally, zanidatamab has received Orphan Drug designations from FDA for the treatment of BTC and GEA, as well as Orphan Drug designation from the European Medicines Agency for the treatment of BTC and gastric cancer.  Important Safety Information for ZIIHERA WARNINGS AND PRECAUTIONS Embryo-Fetal Toxicity ZIIHERA can cause fetal harm when administered to a pregnant woman. In literature reports, use of a HER2-directed antibody during pregnancy resulted in cases of oligohydramnios and oligohydramnios sequence manifesting as pulmonary hypoplasia, skeletal abnormalities, and neonatal death. Verify the pregnancy status of females of reproductive potential prior to the initiation of ZIIHERA. Advise pregnant women and females of reproductive potential that exposure to ZIIHERA during pregnancy or within 4 months prior to conception can result in fetal harm. Advise females of reproductive potential to use effective contraception during treatment with ZIIHERA and for 4 months following the last dose of ZIIHERA. Left Ventricular Dysfunction ZIIHERA can cause decreases in left ventricular ejection fraction (LVEF). LVEF declined by >10% and decreased to <50% in 4.3% of 233 patients. Left ventricular dysfunction (LVD) leading to permanent discontinuation of ZIIHERA was reported in 0.9% of patients. The median time to first occurrence of LVD was 5.6 months (range: 1.6 to 18.7). LVD resolved in 70% of patients. Assess LVEF prior to initiation of ZIIHERA and at regular intervals during treatment. Withhold dose or permanently discontinue ZIIHERA based on severity of adverse reactions. The safety of ZIIHERA has not been established in patients with a baseline ejection fraction that is below 50%. Infusion-Related Reactions ZIIHERA can cause infusion-related reactions (IRRs). An IRR was reported in 31% of 233 patients treated with ZIIHERA as a single agent in clinical studies, including Grade 3 (0.4%), and Grade 2 (25%). IRRs leading to permanent discontinuation of ZIIHERA were reported in 0.4% of patients. IRRs occurred on the first day of dosing in 28% of patients; 97% of IRRs resolved within one day. Prior to each dose of ZIIHERA, administer premedications to prevent potential IRRs. Monitor patients for signs and symptoms of IRR during ZIIHERA administration and as clinically indicated after completion of infusion. Have medications and emergency equipment to treat IRRs available for immediate use. If an IRR occurs, slow, or stop the infusion, and administer appropriate medical management. Monitor patients until complete resolution of signs and symptoms before resuming. Permanently discontinue ZIIHERA in patients with recurrent severe or life-threatening IRRs. Diarrhea ZIIHERA can cause severe diarrhea. Diarrhea was reported in 48% of 233 patients treated in clinical studies, including Grade 3 (6%) and Grade 2 (17%). If diarrhea occurs, administer antidiarrheal treatment as clinically indicated. Perform diagnostic tests as clinically indicated to exclude other causes of diarrhea. Withhold or permanently discontinue ZIIHERA based on severity. ADVERSE REACTIONS Serious adverse reactions occurred in 53% of 80 patients with unresectable or metastatic HER2-positive BTC who received ZIIHERA. Serious adverse reactions in >2% of patients included biliary obstruction (15%), biliary tract infection (8%), sepsis (8%), pneumonia (5%), diarrhea (3.8%), gastric obstruction (3.8%), and fatigue (2.5%). A fatal adverse reaction of hepatic failure occurred in one patient who received ZIIHERA. The most common adverse reactions in 80 patients with unresectable or metastatic HER2-positive BTC who received ZIIHERA (≥20%) were diarrhea (50%), infusion-related reaction (35%), abdominal pain (29%), and fatigue (24%). USE IN SPECIFIC POPULATIONS Pediatric Use Safety and efficacy of ZIIHERA have not been established in pediatric patients. Geriatric Use Of the 80 patients who received ZIIHERA for unresectable or metastatic HER2-positive BTC, there were 39 (49%) patients 65 years of age and older. Thirty-seven (46%) were aged 65-74 years old and 2 (3%) were aged 75 years or older. No overall differences in safety or efficacy were observed between these patients and younger adult patients. The full U.S. Prescribing Information for ZIIHERA, including BOXED Warning, is available at: About Jazz Pharmaceuticals Jazz Pharmaceuticals plc (Nasdaq: JAZZ) is a global biopharma company whose purpose is to innovate to transform the lives of patients and their families. We are dedicated to developing potentially life-changing medicines for people with serious diseases — often with limited or no therapeutic options. We have a diverse portfolio of marketed medicines, including leading therapies for sleep disorders and epilepsy, and a growing portfolio of cancer treatments. Our patient-focused and science-driven approach powers pioneering research and development advancements across our robust pipeline of innovative therapeutics in oncology and neuroscience. Jazz is headquartered in Dublin, Ireland with research and development laboratories, manufacturing facilities and employees in multiple countries committed to serving patients worldwide. Please visit for more information. Jazz Pharmaceuticals plc Caution Concerning Forward-Looking Statements This press release contains forward-looking statements, including, but not limited to, statements related to zanidatamab's potential as a transformative treatment option for patients with HER2-positive disease, expected timing of top-line results of the pivotal Phase 3 HERIZON-GEA-01 and other statements that are not historical facts. These forward-looking statements are based on Jazz Pharmaceuticals' current plans, objectives, estimates, expectations and intentions and inherently involve significant risks and uncertainties. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, risks and uncertainties associated with the successful completion of regulatory activities and uncertain regulatory approval, risks related to failure or delays in successfully initiating or completing clinical trials and assessing patients and other risks and uncertainties affecting Jazz Pharmaceuticals and its development programs, including those described from time to time under the caption "Risk Factors" and elsewhere in Jazz Pharmaceuticals plc's Securities and Exchange Commission filings and reports (Commission File No. 001-33500), including Jazz Pharmaceuticals' Annual Report on Form 10-K for the year ended December 31, 2024, as supplement by Jazz Pharmaceuticals' Quarterly Report on Form 10-Q for the quarter ended March 31, 2025, and future filings and reports by Jazz Pharmaceuticals. Other risks and uncertainties of which Jazz Pharmaceuticals is not currently aware may also affect Jazz Pharmaceuticals' forward-looking statements and may cause actual results and the timing of events to differ materially from those anticipated. The forward-looking statements herein are made only as of the date hereof or as of the dates indicated in the forward-looking statements, even if they are subsequently made available by Jazz Pharmaceuticals on its website or otherwise. Jazz Pharmaceuticals undertakes no obligation to update or supplement any forward-looking statements to reflect actual results, new information, future events, changes in its expectations or other circumstances that exist after the date as of which the forward-looking statements were made. Contacts: Media Contact: Kristin Bhavnani Head of Global Corporate Communications Jazz Pharmaceuticals plc [email protected] Ireland +353 1 637 2141 U.S. +1 215 867 4948 Investors: Jeff Macdonald Executive Director, Investor Relations Jazz Pharmaceuticals plc [email protected] Ireland +353 1 634 3211 U.S. +1 650 496 2717 i Abrahao-Machado I.F., et al. HER2 testing in gastric cancer: An update WorldJGastroenterol. 2016;22(19):4619-4625. ii Van Custem E., et al. HER2 screening data from ToGA: targeting HER2 in gastric and gastroesophageal junction cancer. Gastric Cancer. 2015;18(3):476-484. iii Stroes, C.I., et al. A systematic review of HER2 blockade for the curative treatment of gastroesophageal adenocarcinoma: Successes achieved and opportunities ahead. CancerTreatRev. 2021;99:102249. iv Battaglin F, et al. Molecular biomarkers in gastro-esophageal cancer: recent developments, current trends and future directions. Cancer Cell International. 2018;18(99). v ZIIHERA (zanidatamab-hrii) Prescribing Information. Palo Alto, CA: Jazz Pharmaceuticals, Inc.). SOURCE Jazz Pharmaceuticals plc WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

上周，重磅消息很多。一边，国产创新药“霸屏”，尤其是各类国产ADC和PD-L1/VEGF双抗，就在今日，BMS就普米斯PD-L1/VEGF双抗与BioNTech达成合作，首付款高达15亿美元。在政策先行，资本助力和创新积累推动下，国家药监局一天连批11个创新药，集体进入爆发时代。同期，石药、翰森制药领衔，有多款国产新药成功出海。紧接着，2025年ASCO大会召开，中国创新药力量席卷，值得一提的是，中国生物制药宣布其“得福组合”击败了K药。另一端，医药行业合规反腐持续深化，“宇宙最大医院”原院长阚全程涉嫌严重违纪违法，目前正接受河南省纪委监委纪律审查和监察调查；医保局发文，药师“挂证”整治力度全面升级，近2.4万家定点药店限期核查，此次整治是医药零售行业从“资质合规”迈向“实质服务合规”的关键转折节点。一起看看上周还有哪些大事发生？政策动态国家医保局发布《关于对定点零售药店药师“挂证”等情况开展核查的公告》：通过对全国定点零售药店4月份以来的医保结算数据进行筛查分析发现，部分药师姓名出现在多家定点零售药店的药品费用明细中，可能存在药师信息被假冒或药师“挂证”的违规情况，共涉24个省份、23997家定点零售药店、9563名药师。国家医保局将通过日常巡查、专项检查、数据筛查等，不定期对定点零售药店药师配备及在岗履职情况、医保基金使用情况等进行监督检查。药品追溯码入库环节信息采集时间压缩至1分钟左右：国家医保局等四部门联合发布《关于加强药品追溯码在医疗保障和工伤保险领域采集应用的通知》，要求推动药品追溯码在医疗保障各环节全量采集、全场景应用，逐步实现医药机构全覆盖。湖南省医疗保障局会同卫健、药监部门，创新推出药品耗材全域智慧管理模式（H-SPD），打破“三医”数据壁垒，在药品入库环节，追溯信息采集时间已经压缩至1分钟左右，效率极大提升。11个创新药集中获批：5月29日，11款创新药获国家药监局批准上市（9款国内自主研发创新药），包括恒瑞瑞康曲妥珠单抗（HER2-ADC），百济泽尼达妥单抗（HER2双抗），复星医药枸橼酸伏维西利（CDK4/6）等。有机构指出，2025年将成为医药行业“三个元年”，医保谈判后产品进入收入放量期、成批企业迎来盈利跨越、支付端改善推动估值抬升。广东省启动带量采购，含170个药品、248个品规：广东省药品交易中心发布通知，联盟内22省（含兵团）将对此前品种中除国采（含全国联采）重叠的药品剂型、国家医保谈判药品（含竞价药品）和国家基本医保药品目录外的170个品种、248个品规药品展开接续采购。此次采购取消了A/B采购单，分组竞价。小儿感冒宁颗粒和蒲公英颗粒转换为非处方药：根据《处方药与非处方药分类管理办法（试行）》（原国家药品监督管理局令第10号）规定，经国家药监局组织论证和审核，小儿感冒宁颗粒和蒲公英颗粒由处方药转换为非处方药。品种名单及非处方药说明书范本一并发布。自补充申请备案之日起生产的药品，不得继续使用原药品说明书。大型制药翰森制药授予再生元在研GLP-1/GIP双受体激动剂HS-20094海外独占许可：6月2日，翰森制药宣布授予再生元开发、生产及商业化HS-20094的全球独占许可（不含中国内地、香港及澳门）。HS-20094是一款在研GLP-1/GIP双受体激动剂，已成功完成多项Ⅱ期临床试验。根据协议，翰森制药将获得8000万美元首付款，并有资格根据该产品开发、注册审批和商业化进展收取最高19.3亿美元里程碑付款，以及未来潜在产品销售的双位数百分比特许权使用费。中国生物制药“得福组合”头对头战胜K药：当地时间6月1日，中国生物制药以最新重磅摘要（LBA）形式披露CAMPASS研究数据，“得福组合”——贝莫苏拜单抗（安得卫）联合安罗替尼（福可维）头对头战胜K药，这是“得福组合”继头对头战胜替雷联合化疗用于一线鳞状非小细胞肺癌（sq-NSCLC）后，在非小细胞肺癌治疗领域再次展示出极具潜力的临床价值。石药有望达成三项潜在交易，合计达50亿美元：5月30日，石药集团发布一则公告，透露目前正与若干独立第三方就三项潜在交易进行磋商，涉及有关石药若干产品（ 包括表皮生长因子受体抗体药物偶联物（ EGFR ADC ）及由石药技术平台开发的其他药品 ）在开发、生产及商业化方面的授权及合作（潜在交易）。每项潜在交易项下，可能应付予石药的潜在首付款、潜在开发里程碑付款及潜在商业化里程碑付款 ， 合计可能达到约50亿美元 。 三 项 潜 在 交 易 中 的 其 中 一 项 目 前 已 处 于后 期 阶段，预计将于2025年6月完成。信立泰引进国为医药高血压siRNA新药：5月27日，成都国为医药与信立泰共同签署新药研发合作协议，国为医药将在研的AGT-siRNA小干扰核酸药物GW906的原料药及制剂在中国市场的相关权利独家授予信立泰，包括但不限于研发、注册、生产及其商业化等。上药控股与诺华中国签署多款眼科产品战略合作协议：此次合作中，诺华中国将借助上药控股全渠道整合营销服务优势和广泛的市场覆盖能力，加速其抗感染和青光眼成熟产品下沉至非目标市场终端；而上药控股则凭借诺华中国的品牌影响力与产品优势，进一步完善眼科领域的服务体系，强化在专科用药领域的市场竞争力。超90亿美元，BMS就普米斯PD-L1/VEGF双抗与BioNTech达成合作：6月2日，BMS和BioNTech宣布双方已达成协议，将在全球范围内共同开发和商业化BioNTech的双抗候选药物BNT327（PM8002，收购自普米斯），用于多种实体瘤类型。据悉，BMS将向BioNTech支付15亿美元的首付款。默沙东中国区换帅：默沙东全球高级副总裁、默沙东中国总裁田安娜将卸任中国总裁一职，默沙东日本总裁Kyle Tattle将接替其任职，自7月1号开始生效。Kyle Tattle曾在中国、日本和亚太地区领导默沙东的肿瘤业务。他在领导默沙东日本公司五年时间里，将默沙东日本的市场排名从第8位提升至第3位，且业绩卓著。默沙东/第一三共撤回HER3 ADC上市申请：5月29日，双方宣布自愿撤回全球首个申报上市的HER3 ADC新药Patritumab deruxtecan（HER3-DXd）治疗EGFR突变NSCLC的上市申请，原因主要在于验证性三期临床HERTHENA-Lung02没有达到OS终点。该产品上市并非首次遇阻，去年6月，基于第三方生产原因，HER3-DXd被FDA延迟批准用于既往至少接受过两种系统治疗的EGFR突变型局部晚期或转移性非小细胞肺癌。礼来收购非阿片类疼痛疗法公司SiteOne：5月27日，礼来和SiteOne Therapeutics宣布达成确定性协议，礼来将收购SiteOne。SiteOne是一家致力于开发钠通道小分子抑制剂以治疗疼痛和其他神经元过度兴奋性疾病的初创生物技术公司。根据协议条款，SiteOne股东将获得高达10亿美元的现金，包括前期付款以及在实现某些监管和商业里程碑后支付的后续款项。卫材失眠新药在中国获批上市：5 月 27 日，卫材药业宣布其莱博雷生片（商品名：达卫眠）在中国获批上市，用于治疗成人失眠。莱博雷生是国内首款获批上市的双重食欲素受体拮抗剂，通过竞争性结合两种食欲素受体亚型（OX1R 和 OX2R），抑制食欲素神经传递，调节睡眠-觉醒节律。生物科技超15亿美元，信诺维CLDN18.2 ADC授权安斯泰来： 5月30 日，信诺维和安斯泰来宣布，双方就XNW27011（一款靶向CLDN18.2，处于临床阶段的新一代抗体偶联药物）达成一项独家许可协议，授予安斯泰来独家拥有XNW27011在全球（除中国大陆、香港、澳门和台湾地区外）开发和商业化的权利。百济神州和恒瑞医药两款靶向HER2新药获批上市：5月29日，百济神州HER2双抗泽尼达妥单抗上市申请已获批准。2023年11月，该产品被纳入优先审评，适应症为治疗既往接受过全身治疗的HER2高表达的不可切除局部晚期或转移性胆道癌患者。此外，恒瑞医药的注射用瑞康曲妥珠单抗在国内获批，单药适用于治疗存在HER2（ERBB2）激活突变且既往接受过至少一种系统治疗的不可切除的局部晚期或转移性非小细胞肺癌成人患者。岸迈生物就KLK2 T细胞接合分子达成授权合作：5月27日，岸迈生物宣布与TCG Labs Soleil投资组合公司Juri Biosciences签署全球许可协议。该协议授予Juri Biosciences用于治疗转移性前列腺癌的靶向激肽释放肽相关肽酶2（KLK2）和CD3开发的T细胞接合分子的全球独家权利。海创药业1类氘代新药获批上市：5月29日，海创药业自主研发的Ⅰ类新药海纳安（通用名：氘恩扎鲁胺软胶囊）获得国家药监局批准上市，用于治疗接受醋酸阿比特龙及化疗后出现疾病进展，且既往未接受新型雄激素受体抑制剂的转移性去势抵抗性前列腺癌（mCRPC）成人患者。氘恩扎鲁胺软胶囊是国内首款获批上市治疗该适应症的国产创新药物。荣昌生物泰它西普获批重症肌无力新适应症：5月27日，荣昌生物的注射用泰它西普已获NMPA批准新适应症，用于治疗全身型重症肌无力（gMG）。此前，泰它西普已有系统性红斑狼疮（SLE）、类风湿关节炎（RA）两项适应症在国内获批上市。君实生物昂戈瑞西单抗新适应症获批：5月27日，君实生物宣布，昂戈瑞西单抗注射液新适应症获批，用于杂合子型家族性高胆固醇血症以及他汀类药物不耐受或禁忌使用的患者中，单独或与依折麦布联合用药用于非家族性高胆固醇血症和混合型血脂异常的成人患者。爱尔康干眼病药物获FDA批准上市：5月28日，爱尔康宣布美国FDA已批准Tryptyr（acoltremon滴眼液）0.003%用于治疗干眼症（DED）的体征和症状。Tryptyr是首个TRPM8受体激动剂（神经调节剂），通过刺激角膜感觉神经，快速增加自然泪液产生。资本市场95亿美元，赛诺菲收购Blueprint Medicines：6月2日，赛诺菲宣布收购Blueprint Medicines公司，以扩展其在罕见免疫疾病的布局，扩充早期免疫管线。据悉，赛诺菲以129美元/股现金收购Blueprint，股权总价值约91亿美元，此外，Blueprint的股东还将获得针对两项里程碑的CVR付款，交易总价值约95亿美元。“新型减肥药第一股”派格生物港股上市：5月27日，派格生物正式登陆港交所，中金公司担任独家保荐人，其发行价为15.60港元/股，发行市值60.21亿港元，募资3亿港元。上市首日，派格生物开盘下跌13.46%。先通医药港股递表：5月26日，港交所官网显示，先通医药递交港交所上市申请，中金公司和中信证券为联席保荐人。根据灼识咨询报告，该公司是中国首家获得创新放射性药物上市批准的企业；以药品上市许可持有人身份获得创新放射性药物生产许可的企业；完成治疗用放射性配体注册临床试验并获国家药监局受理新药申请的企业。兴齐眼药募资8.5亿元：5月28日，兴齐眼药抛出一则定增募资计划，拟向特定对象发行股票，募集不超过8.5亿元，其中6.5亿元用于研发中心建设项目，2亿元用于补充流动资，旨在构建眼科领域标杆案例，打造集智能实验室矩阵、跨国研发协作中枢、产学研协同创新平台为一体的研发中心，搭建精准制剂技术、多维药理评价、整合式药代评价、药物包材研究等平台。嘉应制药因信披违规被立案：嘉应制药披露，公司于5月28日收到中国证监会的《立案告知书》，因公司涉嫌信息披露违法违规，根据相关法律法规，中国证监会决定对公司立案。公司将积极配合中国证监会的相关工作，并严格按照规定及监管要求履行信息披露义务。一审| 黄佳二审| 李芳晨三审| 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