Exatecan mesilate

Approval based on TROPION-Breast02 phase 3 trial results where Daiichi Sankyo and AstraZeneca’s Datroway showed a statistically significant and clinically meaningful improvement for the dual primary endpoints of overall survival and progression-free survival Datroway now approved for two breast cancer indications in the EU TOKYO--(BUSINESS WIRE)--Datroway ® (datopotamab deruxtecan) has been approved in the European Union (EU) as monotherapy for the first-line treatment of adult patients with unresectable or metastatic triple negative breast cancer (TNBC) who are not candidates for PD-1/PD-L1 inhibitor therapy. Datroway is a specifically engineered TROP2 directed DXd antibody drug conjugate (ADC) discovered by Daiichi Sankyo (TSE: 4568) and being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca (LSE/STO/NYSE: AZN). The approval by the European Commission follows the positive opinion of the Committee for Medicinal Products for Human Use of the European Medicines Agency and is based on results from the TROPION-Breast02 phase 3 trial, which were presented at the 2025 European Society for Medical Oncology Congress and subsequently published in Annals of Oncology . In TROPION-Breast02, which included patients with metastatic TNBC who experienced early relapse following prior treatment, Datroway demonstrated a statistically significant and clinically meaningful 5.0 month improvement in median overall survival (OS) versus investigator’s choice of chemotherapy (hazard ratio [HR]=0.79; 95% confidence interval [CI]: 0.64-0.98; p=0.0291). Median OS was 23.7 months for patients treated with Datroway versus 18.7 months for those treated with chemotherapy. Datroway reduced the risk of disease progression or death by 43% compared to chemotherapy (HR=0.57; 95% CI: 0.47-0.69; p<0.0001) as assessed by blinded independent central review (BICR). Median progression-free survival (PFS) was 10.8 months for patients treated with Datroway versus 5.6 months for those treated with chemotherapy in patients with metastatic TNBC who are not candidates for PD-1/PD-L1 inhibitor therapy. Datroway also was associated with more robust treatment responses compared to chemotherapy, with an objective response rate (ORR) of 62.5% versus 29.3% for those treated with chemotherapy. ezstandalone.cmd.push(function () { ezstandalone.showAds(131); }); “For people living with metastatic triple negative breast cancer, every new treatment option matters. Despite recent advances, more than two thirds of patients are not candidates for immunotherapy and have had limited options beyond chemotherapy,” said Giuseppe Curigliano, MD, PhD, Director of the Early Drug Development Division, European Institute of Oncology, Professor of Medical Oncology, University of Milan, Italy and investigator for the TROPION-Breast02 trial. “In my practice, I see firsthand the devastating impact this aggressive disease has on patients and their families. This approval of datopotamab deruxtecan provides a new treatment option for eligible patients and represents meaningful progress.” The safety pro Datroway (6 mg/kg) was evaluated in 319 patients with TNBC who received Datroway in TROPION-Breast02. The most common (≥20%) adverse reactions, including laboratory abnormalities, were stomatitis, increased amylase, nausea, alopecia, decreased hemoglobin, decreased white blood cells, constipation, decreased calcium, decreased lymphocytes, fatigue, decreased neutrophils, increased alanine aminotransferase, increased aspartate aminotransferase, dry eye, keratitis, decreased albumin, vomiting, musculoskeletal pain, decreased sodium and increased blood alkaline phosphatase. Serious adverse reactions occurred in 17% of patients who received Datroway. Serious adverse reactions in more than 1% of patients who received Datroway included pneumonia, vomiting, COVID-19 and anemia. One patient fatality was attributed to interstitial lung disease/pneumonitis. “With this approval, Datroway is the only TROP2 directed antibody drug conjugate approved in the EU that has demonstrated an overall survival benefit in the first-line setting, representing a meaningful advance for the treatment of patients with metastatic triple negative breast cancer,” said Ken Keller, Global Head of Oncology Business, and President and CEO, Daiichi Sankyo, Inc. “We look forward to bringing Datroway to patients in the EU as an additional treatment option with the potential to extend survival, reflecting our commitment to advancing innovative medicines that address unmet needs for people living with cancer.” “Every year, more than 80,000 people in Europe are diagnosed with triple negative breast cancer, a disease that often affects younger women and has limited treatment options in the metastatic setting,” said Dave Fredrickson, Executive Vice President, Oncology Hematology Business Unit, AstraZeneca. “Today’s approval of Datroway brings an antibody drug conjugate with a differentiated clinical pro by a strong survival benefit to people with this aggressive disease.” Based on the results of TROPION-Breast02, Datroway has been included in the ESMO Clinical Practice Guidelines as a Category IA first-line treatment option for patients with metastatic TNBC who are not candidates for immunotherapy, and it is the preferred option for patients who have relapsed within six months of completing adjuvant therapy. 1 Datroway also received a score of four out of five on the ESMO Magnitude of Clinical Benefit Scale (ESMO-MCBS), recognizing the clinically meaningful benefit demonstrated in TROPION-Breast02. 2 Datroway (6 mg/kg) was approved in the U.S. in May 2026 for the treatment of adult patients with unresectable or metastatic TNBC who are not candidates for PD-1/PD-L1 inhibitor therapy. Additional reviews are underway in China and Japan, as well as Australia, Canada, Singapore and Switzerland as part of Project Orbis. About TROPION-Breast02 TROPION-Breast02 is a global, multicenter, randomized, open-label phase 3 trial evaluating the efficacy and safety of Datroway versus investigator’s choice of chemotherapy (paclitaxel, nab-paclitaxel, capecitabine, carboplatin or eribulin) in patients with previously untreated locally recurrent inoperable or metastatic TNBC for whom immunotherapy was not an option. This included patients whose tumors did not express PD-L1 as well as patients with PD-L1 expressing tumors who could not receive immunotherapy due to prior exposure in early-stage disease, comorbidities or immunotherapy not being accessible in their geography. Enrollment included patients with de novo or recurrent disease, regardless of disease-free interval, and those with poor prognostic factors such as stable brain metastases. The dual primary endpoints of TROPION-Breast02 are OS and PFS as assessed by BICR. Secondary endpoints include PFS as assessed by investigator, ORR, duration of response, disease control rate, pharmacokinetics and safety. TROPION-Breast02 enrolled 644 patients at sites in Africa, Asia, Europe, North America and South America. For more information visit ClinicalTrials.gov . About Triple Negative Breast Cancer TNBC accounts for approximately 15% of all breast cancer cases, with an estimated 365,000 diagnoses globally each year. 3,4 In Europe, there are an estimated 81,000 diagnoses of TNBC each year. 3,5 TNBC is diagnosed more frequently in younger and premenopausal women, and is more prevalent in Black and Hispanic women. 6,7,8 Metastatic TNBC is the most aggressive type of breast cancer and has one of the worst prognoses, with median OS of just 12 to 18 months and only about 15% of patients living five years following diagnosis. 6,9,10 While some breast cancers may test positive for estrogen receptors, progesterone receptors or overexpression of HER2, TNBC tests negative for all three. 6 Due to its aggressive nature and absence of common breast cancer receptors, TNBC is characteristically difficult to treat. 6 For patients with metastatic disease with PD-L1 expressing tumors, the addition of immunotherapy to chemotherapy has improved outcomes in the first-line setting. 11,12 However, for approximately 70% of patients with metastatic TNBC who are not candidates for immunotherapy, chemotherapy was the standard first-line treatment. 13 TROP2 is a protein broadly expressed in several solid tumors, including TNBC. 14 TROP2 is associated with increased tumor progression and poor survival in patients with breast cancer. 15,16 About Datroway Datroway (datopotamab deruxtecan; datopotamab deruxtecan-dlnk in the U.S. only) is a TROP2 directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC Technology, Datroway is one of seven DXd ADCs in the oncology pipeline of Daiichi Sankyo, and one of the most advanced programs in AstraZeneca’s ADC scientific platform. Datroway is comprised of a humanized anti-TROP2 IgG1 monoclonal antibody, developed in collaboration with Sapporo Medical University, attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers. Datroway (6 mg/kg) is approved in more than 30 countries/regions worldwide for the treatment of adult patients with unresectable or metastatic TNBC who are not candidates for PD-1/PD-L1 inhibitor therapy based on the results from the TROPION-Breast02 trial. Datroway (6 mg/kg) is approved in more than 45 countries/regions worldwide for the treatment of adult patients with unresectable or metastatic HR positive, HER2 negative (IHC 0, IHC 1+ or IHC 2+/ISH-) breast cancer who have received prior endocrine-based therapy and chemotherapy for unresectable or metastatic disease based on the results from the TROPION-Breast01 trial. Datroway (6 mg/kg) is approved in Brazil, Russia, Singapore and the U.S. for the treatment of adult patients with locally advanced or metastatic EGFR-mutated non-small cell lung cancer (NSCLC) who have received prior EGFR-directed therapy and platinum-based chemotherapy, based on the results from TROPION-Lung05 and TROPION-Lung01 trials. Continued approval for this indication in the U.S. may be contingent upon verification and description of clinical benefit in a confirmatory trial. About the Datroway Clinical Development Program A comprehensive global clinical development program is underway with more than 20 trials evaluating the efficacy and safety of Datroway across multiple cancers, including NSCLC, TNBC and urothelial cancer. The program includes eight phase 3 trials in lung cancer, five phase 3 trials in breast cancer, and one phase 2/3 trial in urothelial cancer evaluating Datroway as a monotherapy and in combination with other cancer treatments in various settings. About the Daiichi Sankyo and AstraZeneca Collaboration Daiichi Sankyo and AstraZeneca entered into a global collaboration to jointly develop and commercialize Enhertu ® in March 2019 and Datroway in July 2020 , except in Japan where Daiichi Sankyo maintains exclusive rights for each ADC. Daiichi Sankyo is responsible for the manufacturing and supply of Enhertu and Datroway. About the ADC Portfolio of Daiichi Sankyo The Daiichi Sankyo ADC portfolio consists of eight ADCs in clinical development crafted from ADC technology discovered in-house by Daiichi Sankyo. The DXd ADC Technology platform of Daiichi Sankyo consists of seven ADCs in clinical development where each ADC is comprised of a monoclonal antibody attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers. The DXd ADCs include Enhertu and Datroway, which are being jointly developed and commercialized globally with AstraZeneca, and ifinatamab deruxtecan (I-DXd), raludotatug deruxtecan (R-DXd) and patritumab deruxtecan (HER3-DXd), which are being jointly developed and commercialized globally with Merck & Co., Inc, Rahway, NJ, USA. DS-3939 and DS3790 are being developed by Daiichi Sankyo. An additional ADC being developed by Daiichi Sankyo is DS3610, which consists of an antibody attached to a novel payload that acts as an agonist of STING. Ifinatamab deruxtecan, raludotatug deruxtecan, patritumab deruxtecan, DS-3939, DS3610 and DS3790 are investigational medicines that have not been approved for any indication in any country. Safety and efficacy have not been established. About Daiichi Sankyo Daiichi Sankyo (TSE: 4568) is a global healthcare company committed to becoming a trusted healthcare innovator, transforming the lives of people through its strength in science and technology. The company discovers and develops new standards of care to address diverse medical needs to fulfill its purpose of contributing to the enrichment of quality of life around the world. With a strategic focus on oncology, Daiichi Sankyo is advancing an industry-leading antibody drug conjugate portfolio along with identifying new breakthrough generating technologies to deliver practice-changing medicines to patients, healthcare professionals and society. For more information, please visit . ____________________ REFERENCES: 1 de Azambuja E, et al., Annals of Oncology . 2026 2 ESMO. ESMO-MCBS Scorecard: Datopotamab deruxtecan . Accessed July 2026 3 O’Reilly D, et al. World J Clin Oncol . 2021;12(3):164-182. 4 World Health Organization. Global Status Report on Cancer 2026 . Accessed July 2026. 5 World Health Organization. Global Cancer Observatory: Europe . Accessed July 2026. 6 American Cancer Society. Triple-Negative Breast Cancer . Accessed July 2026. 7 Martinez M, et al. Breast Cancer Res Treat . 2017;166(1):185-193. 8 Vargas L, et al. Cancer Epidemiol Biomarkers Prev . 2019;28(11):1771-1783. 9 National Cancer Institute . SEER Cancer Stat Facts: Female Breast Cancer Subtypes . Accessed July 2026. 10 Huppert L, et al. Ther Adv Med Oncol . 2022;14:1-25. 11 Cortes J, et al. N Engl J Med . 2022;387:217-226. 12 Geurts V, et al. Curr Treat Options Oncol . 2023;24:628-643. 13 Punie K, et al. Oncologist . 2025;30(3):oyaf034. 14 Rossi V, et al. Front Immunol . 2024;15:1447280. 15 Lin H, et al. Exp Mol Pathol . 2013:94(1):73-78. 16 Goldenberg D, et al. Oncotarget . 2018;9(48):28989-29006. Contacts MEDIA CONTACTS: Global: Jennifer Brennan jennifer.brennan@daiichisankyo.com +1 908 900 3183 (mobile) EU: Simone Jendsch-Dowé simone.jendsch-dowe@daiichisankyo.com +49 (89) 78080 (office) Japan: DS-PR_jp@daiichisankyo.com INVESTOR RELATIONS CONTACT: DaiichiSankyoIR_jp@daiichisankyo.com

报告摘要    　　眼科基因疗法双产品同步推进，全球可比性进一步增强。公司作为国内眼科龙头，围绕眼底病长效化同时布局高剂量康柏西普、KH631 和KH658，产品形态由持续给药的生物药延伸至一次给药的基因疗法。法瑞西单抗和高剂量阿柏西普持续放量，证明延长给药间隔能够转化为真实的商业价值；sura-vec、4D-150 等头部项目进入Ⅲ期尾声，则进一步验证了“一次给药、长期表达抗VEGF”的临床开发路径。公司在ARVO2026 更新KH631、KH658 人体数据，两款产品均呈现减少补救注射的趋势，治疗后视功能和视网膜厚度总体稳定，早期安全性支持继续开发。KH631 采用视网膜下给药，随访积累相对充分；KH658 采用脉络膜上腔给药，具备给药方式层面的全球稀缺性。    　　全球先发项目进入Ⅲ期尾声，抗VEGF 基因疗法开始接受注册性验证， sura-vec 治疗nAMD 的注册临床ATMOSPHERE 和ASCENT 已完成超过1,200 例入组，预计2026Q4 读出顶线数据，有望通过大样本验证基因疗法在眼底病变领域的视功能保持、补救注射负担的稳定性和长期安全性，4D-150 两项Ⅲ期均完成入组，“一次给药、长期表达抗VEGF”已由早期概念验证走向大样本临床验证。KH631 和KH658 均已取得中美临床许可，并披露了人体减针、视功能和安全性结果，横向对比竞争力明显，具备BIC 潜力。先发项目逐步验证赛道成药性，康弘药业则通过两个产品具备竞争力的临床数据形成全球稀缺的资产组合，后续数据成熟有望进一步提升其出海确定性。    　　合成生物学打开第二增长曲线。KH617（来自公司合成生物平台的榄香烯）通过干预肿瘤代谢并调节MGMT 表达，探索恢复肿瘤对替莫唑胺的敏感性，ASCO 2026 披露的Ⅰ期研究在复发GBM 患者中观察到积极的早期生存信号，整体耐受性支持继续开发；AACR 2026 公布的多项研究又从代谢调节、MGMT 下调和替莫唑胺再增敏三个层面补充了机制依据。与此同时，公司已利用合成生物工艺完成活性成分的规模化生产验证，使天然产物药物从“有活性”进一步走向“可稳定制造”。已登记的随机Ⅱ期将聚焦首次复发、MGMT 启动子未甲基化患者，同时考察KH617 单药活性和与替莫唑胺联用的再增敏价值。KH815 采用TROP2+雷公藤甲素（来自公司合成生物学平台）+依沙替康双载荷设计，希望通过两类作用机制不同的载荷同时作用于RNA 和DNA 层面，改善单一喜树碱类载荷可能面临的耐药问题，项目已在中国和澳大利亚进入Ⅰ期；第二款双毒素ADC KHN922 于2026 年取得临床试验许可，说明公司已具备从同一平台持续产生候选物的能力。公司选择了成熟TROP2 靶点与前沿载荷组合，既具备靶点层面的成药确定性，也保留产品差异化空间，若临床研究数据积极，价值有望由KH815 单品进一步延伸到平台及后续海外合作。    　　创新能力多点延伸，多条管线站上全球研发热点。KH607 抑郁症Ⅱ期已经完成、产后抑郁症处于Ⅱ期；KHN702 布局已被FDA 批准药物验证的NaV1.8 非阿片镇痛机制；KHN707 则切入食欲素受体失眠药赛道。公司已不再只是依靠单一眼科产品推进创新，而是逐步形成“成熟产品贡献现金流、核心临床资产建立全球可比性、技术平台持续输出新项目”的研发体系。    　　基药目录扩容改善主业预期，成熟产品和创新管线形成双重支撑。2026 年版国家基本药物目录中，公司共有13 个主要在销产品在列，新增舒肝解郁胶囊、右佐匹克隆片、枸橼酸托法替布缓释片以及康柏西普预充式产品。康柏西普西林瓶剂型早在2018年版已进入目录，本轮增加预充式规格，有利于进一步完善终端使用场景；舒肝解郁等独家或优势品种进入目录，也有望提升基层覆盖和患者可及性。    　　盈利预测及投资评级：我们预测公司2026-2028 年有望实现营业收入46/51/53 亿元（2026-2027 年的前值54.6/57.0 亿元，因2026 年新医保价格略低于预测值故调低收入预测），归母净利润10/12/13 亿元（2026-2027 年的前值15.1/16.3 亿元，因2026年收入预计低于前期预测值）。考虑到公司主业稳健，创新药研发管线同时布局基因疗法、双毒素ADC、小分子平台，近期在数据读出方面进入兑现期，显现创新转型成效，维持“买入”评级。    　　风险提示：临床试验失败的风险；竞争格局恶化的风险；资本市场行情波动的风险；国家基本药物目录落地、医疗机构配备及产品放量不及预期的风险；研究报告使用的公开资料可能存在信息滞后或更新不及时的风险。