Zemcelpro 泽姆塞尔普罗® (dorocubicel) achieved remarkable and clinically meaningful survival results in patients with high- and very high-risk acute leukemias and myelodysplastic syndromes ®(dorocubicel)在高危和极高危急性白血病及骨髓增生异常综合征患者中取得了显著且具有临床意义的生存结果。Topline results from two Phase 2 studies to be presented on March 23, 2026 at the EBMT Annual Meeting in Madrid, Spain 两项二期研究的顶线结果将于2026年3月23日在西班牙马德里举行的EBMT年会上公布。MONTREAL 蒙特利尔, ,March 10, 2026 2026年3月10日/PRNewswire/ -- ExCellThera Inc. (ExCellThera), a world leader in blood stem cell expansion and metabolic fitness, and its wholly owned subsidiary Cordex Biologics (Cordex), announce positive results from recently completed Phase 2 studies of Zemcelpro® (dorocubicel), also known as UM171 Cell Therapy, in 60 adult patients with high- and very high-risk acute leukemias (AL) and myelodysplastic syndromes (MDS), a population known to experience high relapse rates and poor survival on conventional allogeneic stem cell transplantation (allo-HSCT). /PRNewswire/ -- 血液干细胞扩增和代谢适应性领域的全球领导者 ExCellThera Inc.(ExCellThera)及其全资子公司 Cordex Biologics(Cordex)宣布,近期完成的 Zemcelpro®(多罗库贝西尔),也称为 UM171 细胞疗法的 2 期研究取得了积极结果。该研究针对 60 名高危和极高危急性白血病(AL)及骨髓增生异常综合征(MDS)成人患者,这类人群在传统异基因造血干细胞移植(allo-HSCT)中复发率高且生存率低。Topline results will be presented on March 23, 2026 at the 52. 顶线结果将于2026年3月23日在第52届会议上公布。nd ndEBMT Annual Meeting in Madrid, Spain. 西班牙马德里EBMT年会。Continue Reading 继续阅读ExCellThera logo ExCellThera 标志At 24 months post-transplant, overall survival (OS), progression-free survival (PFS) and relapse-free survival (RFS) were 63.7%, 57.0% and 60%, respectively, among Zemcelpro® (dorocubicel)-treated patients. The cumulative incidences of relapse and non-relapse mortality (NRM) were 22.5% and 20.5%, respectively. 移植后24个月时,接受Zemcelpro®(多洛库贝)治疗的患者总生存率(OS)、无进展生存率(PFS)和无复发生存率(RFS)分别为63.7%、57.0%和60%。复发和非复发死亡率(NRM)的累积发生率分别为22.5%和20.5%。Age was the strongest predictor of NRM: patients <43 years experienced 0% NRM, with 24-month OS and PFS of 82.8% and 79.3%, respectively. Older patients (≥43 years) had comparable relapse rates but higher NRM. Grade III-IV acute graft-versus-host disease (GVHD) occurred in 20%, while moderate to severe chronic GVHD was infrequent at 7% at two years.. 年龄是NRM的最强预测因子:43岁以下患者NRM为0%,24个月总生存率(OS)和无进展生存率(PFS)分别为82.8%和79.3%。年龄较大(≥43岁)的患者复发率相似,但NRM更高。III-IV级急性移植物抗宿主病(GVHD)发生率为20%,而两年内中度至重度慢性GVHD的发生率较低,为7%。'This represents a remarkable and clinically meaningful achievement for Zemcelpro®(dorocubicel), especially given the historical overall survival of only 25–40% “这对Zemcelpro®(多洛库比利)而言是一项显著且具有临床意义的成就,尤其是考虑到历史上仅25%-40%的总体存活率。1 1for patients of similar disease risk receiving standard of care allogeneic grafts,' said Dr Guy Sauvageau, Chief Scientific Officer. 'Results are particularly promising in adults under 43 years of age, showing even greater benefits in both OS and PFS, with no cases of NRM.' “对于接受标准护理的同种异体移植、具有相似疾病风险的患者来说,结果特别有希望,尤其是在43岁以下的成年人中,显示出更大的总生存期和无进展生存期益处,且没有出现非复发死亡病例。”首席科学官Guy Sauvageau博士说道。'These findings suggest that Zemcelpro® (dorocubicel) could redefine the standard of care for patients with high- and very high-risk blood cancers,' said David Millette, CEO. 'Based on its safety and efficacy profile, this positions Zemcelpro® (dorocubicel) as a potentially transformative therapy, challenging the existing treatment paradigm and offering renewed hope for difficult-to-cure high- and very high-risk patients facing unfavorable outcomes with conventional approaches.'. “这些研究结果表明,Zemcelpro®(多罗库比利)可能重新定义高风险和极高风险血液癌症患者的标准治疗,”首席执行官大卫·米莱特表示。“基于其安全性和有效性,这使Zemcelpro®(多罗库比利)成为一种潜在的变革性疗法,挑战现有的治疗模式,并为那些使用传统方法效果不佳的难治高风险和极高风险患者带来新的希望。”About the studies 关于这些研究Outcomes were assessed in 60 adults with high- or very high–risk AL or MDS who received Zemcelpro® (dorocubicel) in two prospective phase 2 trials conducted in the United States, Canada and Europe. High-risk AL/MDS was defined as disease with an expected 2-year PFS <40% after conventional allo-HSCT. 在美国、加拿大和欧洲进行的两项前瞻性 2 期试验中,评估了 60 名接受 Zemcelpro®(多罗库比利)治疗的高危或极高危 AL 或 MDS 成人患者的结局。高危 AL/MDS 被定义为在传统异基因造血干细胞移植后预计 2 年无进展生存率(PFS)<40% 的疾病。Very high-risk disease was defined as a second allo-HSCT, AL with active disease, acute myeloid leukemia (AML) with TP53 mutation and complex karyotype, AML with EVI1 mutation, or MDS with multi-hit TP53 mutation. The primary endpoints were safety and NRM; secondary endpoints included PFS, OS, relapse incidence, engraftment, and GVHD.. 极高危疾病被定义为接受第二次异基因造血干细胞移植(allo-HSCT)、伴有活动性疾病的急性白血病(AL)、携带TP53突变和复杂核型的急性髓系白血病(AML)、携带EVI1突变的AML,或携带多击TP53突变的骨髓增生异常综合征(MDS)。主要终点为安全性和非复发死亡率(NRM);次要终点包括无进展生存期(PFS)、总生存期(OS)、复发率、植入情况和移植物抗宿主病(GVHD)。Sixty (60) patients (median age 43 years; range 19–66) were transplanted; 87% had AL (63% myeloid, 37% lymphoid) and 13% had MDS. Thirty percent (30%) had failed a prior allogeneic transplant and 57% met very high-risk criteria. All patients completed planned 2-3 years of follow-up. 六十名患者(中位年龄43岁;范围19-66岁)接受了移植;87%患有AL(63%为髓系,37%为淋巴系),13%患有MDS。30%的患者之前接受过同种异体移植失败,57%符合极高风险标准。所有患者均完成了计划中的2至3年随访。About Zemcelpro® 关于Zemcelpro®Zemcelpro® (dorocubicel), also known as UM171 Cell Therapy, is a novel personalized cryopreserved haematopoietic stem cell transplantation product containing two components, namely UM171-expanded CD34+ cells (dorocubicel) and unexpanded CD34- cells, each derived from the same cord blood unit. Zemcelpro®(多罗库贝西尔),又称UM171细胞疗法,是一种新型的个性化冷冻保存造血干细胞移植产品,包含两个组分,即UM171扩增的CD34+细胞(多罗库贝西尔)和未扩增的CD34-细胞,两者均源自同一个脐带血单位。Zemcelpro® (dorocubicel) has recently received conditional marketing authorization from the European Commission for the treatment of adults with haematological malignancies requiring allogeneic haematopoietic stem cell transplantation following myeloablative conditioning, for whom no other suitable donor cells are available.. Zemcelpro®(多罗库比利)近日获得欧洲委员会的附条件上市许可,用于治疗需要在骨髓清除性预处理后进行同种异体造血干细胞移植的血液系统恶性肿瘤成人患者,这些患者没有其他合适的供体细胞可用。For complete product information, including warnings and precautions for use and adverse reactions (and their appropriate management), please refer to the EU Summary of Product Characteristics (SmPC) for Zemcelpro® (dorocubicel). 有关完整的产品信息,包括使用警告和注意事项以及不良反应(及其适当管理),请参阅 Zemcelpro®(多柔比星)的欧盟产品特性摘要 (SmPC)。Additional regulatory filings are planned for Zemcelpro® (dorocubicel) with other health authorities, including in the US, Canada, the UK, and Switzerland. Cordex is also actively seeking strategic partnerships to support and accelerate the commercialization of Zemcelpro® (dorocubicel) in Europe and other international markets.. 计划向包括美国、加拿大、英国和瑞士在内的其他健康监管机构提交Zemcelpro®(多罗库比利)的更多监管文件。Cordex还在积极寻求战略合作伙伴,以支持并加速Zemcelpro®(多罗库比利)在欧洲及其他国际市场的商业化进程。Zemcelpro® (dorocubicel) has been evaluated in 120 patients with haematologic malignancies in clinical trials in the United States, Europe and Canada. Zemcelpro® (dorocubicel) has received orphan drug designation and regenerative medicine advanced therapy (RMAT) designations from the FDA as well as orphan medicinal product designation, advanced therapy medicinal product (ATMP) classification and priority medicines (PRIME) designation from the EMA.. Zemcelpro®(多罗库比利)已在美、欧、加的临床试验中对120名血液系统恶性肿瘤患者进行了评估。Zemcelpro®(多罗库比利)获得了FDA授予的孤儿药资格和再生医学先进疗法(RMAT)资格,以及EMA授予的孤儿药产品资格、先进治疗药品(ATMP)分类和优先药物(PRIME)资格。Zemcelpro® (dorocubicel) has been tested in Phase 2 trials in patients with high- and very high-risk acute leukemias and myelodysplasias who have limited treatment options with low survival outcomes and high incidence of relapse under the current standard of care, including patients with patients with TP53 mutations or other genetic abnormalities, patients requiring a second transplant, and patients with refractory or active disease. Zemcelpro®(多罗库比利)已在二期临床试验中对高危和极高危急性白血病及骨髓增生异常综合征患者进行了测试,这些患者治疗选择有限,在当前标准治疗下生存率低、复发率高,其中包括携带TP53突变或其他基因异常的患者、需要二次移植的患者以及难治性或活动性疾病的患者。A pivotal Phase 3 trial in this patient population will be initiated as soon as possible.. 这一患者群体的关键性III期试验将尽快启动。The use of Zemcelpro® (dorocubicel) in other patient populations, including pediatric patients and patients with non-malignant haematological diseases, is also being investigated. Zemcelpro®(多罗库比利)在其他患者群体中的应用,包括儿科患者和非恶性血液病患者,也正在研究中。The product safety and efficacy have not yet been established by other regulatory agencies, such as the U.S. FDA, the MHRA and Health Canada. 产品的安全性和有效性尚未由其他监管机构(如美国 FDA、MHRA 和加拿大卫生部)确定。About ExCellThera and UM171 Technology 关于ExCellThera和UM171技术ExCellThera is a world leader in enhanced blood stem cell therapies. ExCellThera's proprietary Enhance ExCellThera 是增强型血液干细胞疗法的全球领导者。ExCellThera 专有的增强技术TM 商标platform for cell expansion and metabolic fitness is designed to deliver a greater dose of functional therapeutic stem cells by expanding haematopoietic stem cells (HSCs) from any source and counteracting the effects of culture or gene editing induced stress. ExCellThera partners with biopharmas to help them develop best-in-class cell and gene therapies by leveraging the technologies that form the Enhance. 用于细胞扩增和代谢适应性的平台旨在通过扩增来自任何来源的造血干细胞(HSCs)并抵消培养或基因编辑诱导的压力影响,提供更高剂量的功能性治疗干细胞。ExCellThera与生物制药公司合作,利用构成Enhance的技术,帮助他们开发一流的细胞和基因疗法。TM 商标platform, including the proprietary molecule UM171 which has a first-in-class mechanism of action for 平台,包括具有首创作用机制的专有分子UM171,ex vivo 离体expansion and metabolic fitness of HSCs. For additional information, visit HSC的扩增和代谢适应性。欲了解更多信息,请访问 excellthera.com excellthera.com, and follow us on LinkedIn. ,并在LinkedIn上关注我们。Zemcelpro®is a registered trademark of ExCellThera or its related companies. Zemcelpro® 是 ExCellThera 或其相关公司的注册商标。1 1Armand P, Kim HT, Logan BR, Wang Z, Alyea EP, Kalaycio ME, et al. Validation and refinement of the Disease Risk Index for allogeneic stem cell transplantation. Blood. 2014;123(23):3664–367 阿曼德 P, 金 HT, 洛根 BR, 王 Z, 艾利亚 EA, 卡拉伊乔 ME, 等。异基因干细胞移植的疾病风险指数的验证与优化。《血液》。2014;123(23):3664–367。Photo - 照片 -https://mma.prnewswire.com/media/2928711/ExCellThera_Zemcelpro_%C2%A0_dorocubicel__Positive_Results_in_High__a.jpg https://mma.prnewswire.com/media/2928711/ExCellThera_Zemcelpro_®_dorocubicel__Positive_Results_in_High__a.jpg21 21% %more press release views with 更多新闻发布视图与 Request a Demo 请求演示