CD123 Antibody Toxin Congregate (CD123 ATC; Tagraxofusp) Combined With Azacitidine for Maintenance Therapy Post Allogeneic Hematopoietic Cell Transplantation for Patients With CD123-Positive Malignant

This phase Ib trial tests the safety, side effects, best dose and effectiveness of tagraxofusp in combination with azacitidine as maintenance therapy in treating patients with CD123 positive acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) after a donor (allogeneic) hematopoietic cell transplant. An allogeneic hematopoietic cell transplant (HCT) is a type of transplant where the cancer patient receives cells from another person. Maintenance therapy is given after the transplant to prevent the cancer from coming back. Tagraxofusp is a drug that targets cells that have CD123 on their surface in order to kill the cancer cells to help prevent the cancer from coming back. Azacitidine is in a class of medications called demethylation agents. It works by helping the bone marrow to produce normal blood cells and by killing abnormal cells. Giving tagraxofusp in combination with azacitidine may be safe, tolerable and/or effective maintenance therapy in patients with CD123 positive AML and MDS after an allogeneic HCT.

开始日期2025-01-28

申办/合作机构

City of Hope National Medical Center

[+1]

NCT06456463

/ Recruiting临床2期

A Phase II Multicenter Open-label Trial of Tagraxofusp (Tag) in Combination With Venetoclax and Azacitidine (Ven/Aza) in Adults With Previously Untreated CD123+ Acute Myeloid Leukemia (AML) Who Are Ineligible for Intensive Chemotherapy

This study will be divided into 2 parts (Part 1 and Part 2). Part 1 will evaluate 2 doses of tagraxofusp (9 and 12 micrograms/kilogram/day [μg/kg/day]), used in combination with venetoclax and azacitidine, to determine the dose for Part 2. This determined dose, in combination with venetoclax and azacitidine, will then be further evaluated in Part 2 in 2 cohorts (TP53 mutated and TP53 wild type). Both parts will be conducted in participants with previously untreated CD123+ AML who are ineligible for intensive chemotherapy.

开始日期2025-01-14

申办/合作机构

Stemline Therapeutics, Inc.

100 项与 Tagraxofusp-ERZS 相关的临床结果

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100 项与 Tagraxofusp-ERZS 相关的转化医学

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100 项与 Tagraxofusp-ERZS 相关的专利（医药）

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项与 Tagraxofusp-ERZS 相关的文献（医药）

2025-07-01·PROSTAGLANDINS LEUKOTRIENES AND ESSENTIAL FATTY ACIDS

The single-dose kinetics of [1–14C]-labelled EPA and DHA, administered to male rats as TAG, phosphatidylcholine (PC), and lyso-phosphatidylcholine (LPC), is structurally similar across lipid forms and can be described using the same compartmental models

Article

作者: Hals, Petter-Arnt ; Harris, Stephen ; Scott, Grace ; Hoem, Nils

To investigate the systemic kinetics of EPA and DHA across different lipid classes, male rats were administered [1-¹⁴C]-radiolabelled EPA and DHA as triglycerides (TAG), phosphatidylcholine (PC), or lyso-phosphatidylcholine (LPC) by gavage. LPC was also administered intravenously. Plasma and whole blood concentration-time profiles were recorded from 0 to 168 hours, while cumulative radioactivity in expired air, faeces, and urine was recorded for up to 336 hours. Non-compartmental analysis and compartmental modelling demonstrated overall first-order radiotracer kinetics for both fatty acids, with comparable terminal half-lives. The primary difference was in maximum concentration (Cmax ((µg-eq/g)/(mg/kg)): DHA = 0.18± 0.089, EPA = 0.24± 0.103; P < 0.0001). Between TAG and PC, only time to maximum concentration (Tmax (h)) differed (PC = 3.23 ± 0.94, TAG = 2.55 ± 0.77; P = 0.0004). LPC showed significant differences from TAG and PC in area under the curve (AUC0-inf), Cmax, Tmax, and total clearance (CL/F (mL/(kg h))). Cumulative radioactivity levels in expired air and faeces were consistent with blood and plasma kinetics. As suggested by early-phase (0 to 48 hours) radioactivity accumulation, which deviated from first-order behaviour, TAG and PC, but not LPC, exhibited some faecal loss without systemic absorption. The compartmental models developed performed equally well for radiolabelled EPA and DHA, regardless of whether administered as TAG, PC, or LPC. The model can be adapted to handle non-zero endogenous baselines and was successfully applied to non-radiolabelled EPA, docosapentaenoic acid (DPA), and DHA, quantified via LC-MS/MS. These models can be applied to both radioactive and stable isotopes and adapted to include organ-specific kinetics, as well as those of EPA, DPA, and DHA in other species, including humans.

2025-03-01·Food Science of Animal Resources

Physicochemical and Sensory Characteristics of Hybrid Flexitarian Pork Loin Steak Combined with Different Plant Ingredients

Article

作者: Kim, Chan-Jin ; Alam, Amm Nurul ; Joo, Seon-Tea ; Lee, Eun-Yeong ; Kim, So-Hee ; Hossain, Md Jakir ; Hwang, Young-Hwa

This study intended to assess the impacts of the following plant ingredients as treatment named 2.5% alginate paste (TAG), carrot (TRC), red cabbage (TCA), chickpea (Cicer arietinum; TCP), and included in pork loin meat to produce hybrid meat steak (HBM) to provide healthy meat products suitable for the flexitarian consumers. Plant ingredients were added to the treatment groups at 30%. Results illustrate that HBM steaks had higher (p<0.05) moisture content than HBM steaks. In raw HBM steaks, pH, CIE L*, and CIE a* were increased than pork steak. Pork steak and TCP demonstrated the highest (p<0.05) cooking loss followed by TRC, TCA, and TAG, whereas, TRC and TCA had higher (p<0.05) releasing water content followed by pork, TCP, and TAG. HBM steaks demonstrated a lower (p<0.05) Warner-Bratzler shear force value than pork steaks. According to the sensory panel pork steak and TAG had overall higher acceptance (p<0.05) followed by TRC, TCP, and least for TCA, which was suggested as especially suitable for the elderly or kids. The electronic tongue system revealed higher (p<0.05) umami intensity and bitterness in HBM steaks than in pork steak. The richness value was higher in pork steak followed by TAG, TCP, and TRC, and lowest in TCA. Therefore, the current study demonstrated that plant ingredients can successfully be incorporated into pork loin to make suitable flexitarian steak to keep eating meat but in a lower concentration.

2025-02-01·Animal

Temporal changes in plasma and milk lipids in response to an esophageal bolus of rumen-protected fish oil in lactating Holstein dairy cows

Article

作者: McFadden, J W ; Rico, J E ; Sáinz de la Maza-Escolà, V ; Deme, P ; Senevirathne, N D ; Gervais, R ; Haughey, N J

Feeding very-long-chain omega-3 (VLC n-3) fatty acids (FA), which are found in fish oil, may have beneficial effects on health, fertility, and milk production in the dairy cow. Rumen-protected technologies aim to prevent the ruminal biohydrogenation of VLC n-3 FA. To test the effects of these technologies on circulating FA and phospholipid concentrations, and milk FA concentrations, six mid-lactation, multiparous, pregnant Holstein dairy cows (mean ± SD: 155 ± 19 days in milk; 3.0 ± 0.5 body condition score; 3.2 ± 1.1 lactations; 644 ± 23 kg BW) were randomly assigned to treatments in a study with a replicated 3 × 3 Latin Square design. Cows were unsupplemented (control) or supplemented with a single esophageal bolus of a gelatin capsule containing a fish oil product coated in palm oil triglycerides or modified starch (TAG and STR, respectively). The provision of either fish oil product provided 10 g equivalent of VLC n-3 FA including 20:5 n-3 and 22:6 n-3. Cows were fed a total mixed ration to meet or exceed nutrient requirements and were milked thrice daily. Blood was sampled at 0, 2, 4, 6, 8, 10, 12, 16, 18, 20, 24, and 36 h, relative to bolus administration. A 7-d washout period was utilized between each bolus delivery. Extracted plasma was analyzed for individual FA and phospholipid concentrations using gas chromatography and mass spectrometry. Separated milk fat was analyzed for individual milk FA using gas-liquid chromatography and a flame-ionization detector. Data were analyzed under a mixed model with the random effect of cow, and the fixed effects of treatment, hour, and period. Plasma concentrations of 20:5 n-3 and 22:6 n-3 increased over time by TAG and STR, relative to control. Plasma concentrations of lysophophatidylcholine-20:5, -22:5 and -22:6, and phosphatidylcholine (PC)-38:5 and -38:6 were higher in TAG and STR by h 10, relative to control. Plasma PC-40:5 and -40:6 concentrations were greater in cows administered STR by h 10, relative to TAG. Total milk n-3 increased over time in treated cows compared to control. We conclude that triglyceride- or starch-encapsulated fish oil increases the plasma and milk concentrations of VLC n-3 FA and phospholipids containing these acyl chains in lactating cows.

项与 Tagraxofusp-ERZS 相关的新闻（医药）

2025-03-20

·美通社

美纳里尼集团宣布与VisualDx合作，帮助识别可能患有浆细胞样树突状细胞瘤(BPDCN)的人

BPDCN是一种侵袭性血液恶性肿瘤，预后较差 VisualDx是全球2,300多家医院、诊所和医学院使用的临床决策支持系统 VisualDx的系统旨在利用BPDCN皮肤病变的实际图像和集成到VisualDx平台中的人工智能/机器学习（ AI/ML ）技术，增强对可能患有BPDCN的人的识别意大利佛罗伦萨和纽约 2025年3月21日 /美通社/ -- 美纳里尼集团（ “美纳里尼” ）是一家领先的国际制药和诊断公司， Stemline Therapeutics, Inc. （ “Stemline” ）是美纳里尼集团的全资子公司，专注于为癌症患者提供转化性肿瘤治疗。他们今天宣布，他们正在与VisualDx合作，以加强对可能患有BPDCN的人的识别。这是一个创新的公司合作引入人工智能/机器学习（ AI/ML ）工具以帮助识别BPDCN作为可能的早期鉴别诊断的例子。该项目包括利用整合到VisualDx平台中的BPDCN皮肤病变的实际图像和AI/ML技术。 AI模型现已在VisualDx中上线。 VisualDx是一家以医生为主导的公司，致力于改善医疗决策、医学教育和研究。 VisualDx工具是全球2,300多家医院、诊所和医学院使用的临床决策支持系统。该软件包括一个全面的临床图像数据库，通过与学习机构和其他机构合作提交，并经临床医生审查，以帮助医疗保健专业人员识别所有皮肤类型的皮肤问题。通过人工智能图像搜索，临床医生可以更好地了解不同的皮肤状况，以支持患者在整个护理过程中获得更准确的诊断。 BPDCN是一种侵袭性孤儿血液恶性肿瘤，预后不良（诊断后约8.7至14个月 [1] ），通常表现为皮肤病变，也可能累及骨髓、血液、中枢神经系统、淋巴结和内脏。皮肤科医生可能是第一个识别BPDCN和活检可疑病变迹象的人。然后，病理学家可以通过测试在BPDCN细胞上高度表达的某些生物标志物来测试BPDCN。 Tagraxofusp-erzs是美国、欧洲和全球其他地区唯一获批的BPDCN患者治疗药物，也是首个也是唯一获批的CD123靶向治疗药物。 “BPDCN是一种罕见的临床侵袭性血液恶性肿瘤，通常表现为皮肤病变。由于其侵袭性和未成熟的细胞参与，如果不及时治疗，预后可能会很差。”爱因斯坦血液癌症研究所分子药理学教授、白血病项目主任兼联合主任Marina Konopleva博士说。 “早期诊断在改善患者预后方面发挥着关键作用，新兴的AI/ML技术可能为BPDCN的鉴别诊断和早期识别提供有价值的支持。” “BPDCN通常首先表现为皮肤病变，通常预后不良。美纳里尼集团首席执行官Elcin Barker Ergun表示： “迫切需要早期诊断，以便患者可以获得适当的治疗选择。 “我们很高兴与VisualDx合作，为医疗团队提供使用最新人工智能/机器学习（ AI/ML ）技术的工具，以帮助解释具有挑战性的皮肤病变。” VisualDx不会存储临床医生拍摄患者皮肤检查照片上传的任何图像。这有助于保护患者的隐私。关于BPDCN BPDCN ，以前称为芽肿性NK细胞淋巴瘤，是一种侵袭性孤儿血液恶性肿瘤，通常具有皮肤表现，预后历史上较差。 BPDCN通常表现为皮肤病变，也可能涉及骨髓、血液、中枢神经系统、淋巴结和内脏。 BPDCN起源细胞是浆细胞样树突状细胞（ pDC ）前体。 BPDCN的诊断基于CD123、CD4和CD56的免疫表型诊断三联体以及其他标志物。世界卫生组织（ WHO ）在2008年将这种疾病称为“BPDCN” ；以前的名称包括芽肿性NK细胞淋巴瘤和CD4 +/CD56 +血液皮肤肿瘤。关于ELZONRIS ® (Tagraxofusp-erzs) 美国适应症： ELZONRIS是一种用于治疗2岁及以上成人和儿童患者的母细胞性浆细胞样树突细胞肿瘤(BPDCN)的处方药。有关美国的完整处方信息，请访问 www.elzonris.com 重要的安全信息， ELZONRIS ® 盒装警告：毛细血管泄漏综合征可能危及生命或致命的毛细血管泄漏综合征(CLS)可能发生在接受ELZONRIS的患者身上。监测CLS的体征和症状，并采取建议的措施。警告和注意事项毛细血管泄漏综合征在接受ELZONRIS治疗的患者中已有毛细血管泄漏综合征（ CLS ）的报道，包括危及生命和致命的病例。在临床试验中接受ELZONRIS的患者中， CLS的总发病率为53% （ 65/122 ），其中43% （ 52/122 ）的患者为1级或2级， 7% （ 8/122 ）的患者为3级， 1% （ 1/122 ）的患者为4级， 4例死亡（ 3% ）。发病的中位时间为4天（范围- 1至46天），除5名患者外，所有患者在第1周期都经历了一次事件。在开始使用ELZONRIS治疗之前，请确保患者具有足够的心脏功能，并且血清白蛋白大于或等于3.2 g/dL。在ELZONRIS治疗期间，在开始每次ELZONRIS给药前和给药后根据临床指征监测血清白蛋白水平，并评估患者是否存在CLS的其他体征或症状，包括体重增加、新发水肿或水肿恶化，包括肺水肿、低血压或血流动力学不稳定。超敏反应 ELZONRIS可引起严重的超敏反应。在临床试验中接受ELZONRIS的患者中， 43% (53/122)接受ELZONRIS治疗的患者报告了超敏反应， 7% (9/122)中≥ 3级。在≥ 5%的患者中报告的超敏反应表现包括皮疹、瘙痒和口腔炎。监测患者在使用ELZONRIS治疗期间的超敏反应。如果发生超敏反应，请中断ELZONRIS输注，并根据需要提供支持性护理。肝毒性 ELZONRIS治疗与肝酶升高相关。在临床试验中接受ELZONRIS的患者中， ALT升高发生在79% （ 96/122 ），应用安全测试升高发生在76% （ 93/122 ）。 26% （ 32/122 ）的患者报告了3级ALT升高。 30% （ 36/122 ）的患者报告了3级应用安全测试升高， 3% （ 4/122 ）的患者报告了4级应用安全测试升高。在第1周期中，大多数患者的肝酶升高，并且在剂量中断后是可逆的。在每次输注ELZONRIS之前监测丙氨酸氨基转移酶(ALT)和天冬氨酸氨基转移酶(AST)应用安全测试。如果转氨酶升高到超过正常上限的5倍，则暂停ELZONRIS ，并在正常化或解决后恢复治疗。不良反应最常见的不良反应（发生率≥ 30% ）是毛细血管泄漏综合征、恶心、疲劳、发热、外设水肿和体重增加。最常见的实验室异常（发生率≥ 50% ）是白蛋白、血小板、血红蛋白、钙和钠的降低，以及葡萄糖、ALT和应用安全测试（ AST ）的增加。请参阅完整处方信息，包括盒装警告。如需报告疑似不良反应，请通过 1-877-332-7961 联系Stemline Therapeutics, Inc. ，或通过 1-800-FDA-1088 或 www.fda.gov/medwatch 联系FDA。关于美纳里尼集团美纳里尼集团是一家领先的国际制药及诊断公司，营业额超过47亿美元，员工超过17,000人。美纳里尼专注于心脏病、癌症、肺病、胃肠病、传染病、糖尿病、炎症和镇痛方面等产品需求缺口巨大的治疗领域。美纳里尼拥有18个生产基地和9个研发中心，产品遍布全球140个国家\/地区。如需了解更多信息，请访问 menarini.com 。关于Stemline Therapeutics Inc. Stemline Therapeutics, Inc. ("Stemline")是美纳里尼集团旗下的一家全资子公司，是一家专注于为患者提供转化性肿瘤治疗的商业化生物制药公司。 Stemline实现elacestrant的商业化，这种口服内分泌治疗药物适用于治疗美国、欧洲和全球其他地区患雌激素受体(ER)阳性、人表皮生长因子受体2 (HER2)阴性、 ESR1 突变晚期或转移性乳腺癌且接受过至少一种内分泌疗法后疾病进展的绝经后女性或成年男性。 Stemline还在美国、欧洲和全球其他地区实现针对CD123的新型靶向治疗药物tagraxofusp-erzs的商业化，用于治疗一种侵袭性血液癌--母细胞性浆细胞样树突细胞肿瘤(BPDCN)。此外， Stemline还在欧洲实现selinexor的商业化，这是一种用于多发性骨髓瘤的XPO1抑制剂。该公司还在乳腺癌和血液癌适应症中分别使用elacestrant和tagraxofusp进行多项标签扩展研究，并且拥有众多处于不同开发阶段且针对多种实体和血液癌的其他候选药物的临床试验项目。关于VisualDx VisualDx是一家致力于通过增强思维和及时可视化来改善医疗决策的公司。它致力于缩小医学差距，并相信技术可以弥合知识差距，以实现更公平的护理。 VisualDx已成为全球2,300多家医院、诊所和医学院的专业标准资源，它将面向问题的临床搜索与世界上最好的精心策划的医学图像库相结合，再加上专家的医学知识和复杂的机器学习算法，以帮助进行鉴别诊断、变异、治疗和患者沟通。如需了解更多信息，请访问 www.visualdx.com [1] Pagano L, et al Haematological. 2013; 98 (2): 239-246和Pemmaraju N Curr Hematol Malig Rep. 2017; 12 (6): 510-512

孤儿药临床研究

2025-03-20

·GlobeNewswire-Health Care

Menarini Group Announces Collaboration with VisualDx to Aid in Identifying People Who May Have Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)

BPDCN is an aggressive hematologic malignancy with a historically poor prognosisVisualDx is a clinical decision support system used by more than 2,300 hospitals, clinics, and medical schools around the world VisualDx’s system seeks to enhance identification of people who may have BPDCN, leveraging actual images of BPDCN skin lesions and artificial intelligence/machine learning (AI/ML) technologies incorporated into the VisualDx platform FLORENCE, Italy and NEW YORK, March 20, 2025 (GLOBE NEWSWIRE) -- The Menarini Group ("Menarini"), a leading international pharmaceutical and diagnostics company, and Stemline Therapeutics, Inc. ("Stemline"), a wholly-owned subsidiary of the Menarini Group focused on bringing transformational oncology treatments to cancer patients, today announced that they are collaborating with VisualDx to enhance identification of people who may have BPDCN. This is an example of innovative companies working together to bring artificial intelligence/machine learning (AI/ML) tools to help identify BPDCN as a possible early differential diagnosis. The project includes leveraging actual images of BPDCN skin lesions and AI/ML technologies incorporated into the VisualDx platform. The AI model is now live within VisualDx. VisualDx is a physician-led company committed to improving medical decision-making, medical education and research. The VisualDx tool is a clinical decision support system used by more than 2,300 hospitals, clinics, and medical schools around the world. The software includes a comprehensive database of clinical images, submitted through partnerships with learning institutions and others, and vetted by clinicians, to assist healthcare professionals to identify skin concerns across all skin types. Through the AI image search, clinicians can better understand different skin conditions with the goal of supporting patients to get more accurate diagnoses throughout their care journeys. BPDCN is an aggressive orphan hematologic malignancy with a historically poor prognosis (approximately 8.7 to 14 months post diagnosis1) that typically presents skin lesions and can also involve the bone marrow, blood, central nervous system, lymph nodes and viscera. Dermatologists may be the first to recognize the signs of BPDCN and biopsy suspicious lesions. Pathologists can then test for BPDCN by testing for certain biomarkers which are highly expressed on BPDCN cells. Tagraxofusp-erzs is the only approved treatment for patients with BPDCN, and the first and only approved CD123-targeted therapy, in the United States, Europe and other global regions. "BPDCN is a rare and clinically aggressive hematologic malignancy, often presenting with cutaneous lesions. Due to its aggressive nature and the immature cell involvement, the prognosis can be poor if not treated promptly,” said Marina Konopleva, MD, Phd, Professor, Molecular Pharmacology, Director, Leukemia Program and Co-Director, Blood Cancer Institute, at Montefiore Einstein. “Early diagnosis plays a critical role in improving patient outcomes, and emerging AI/ML technologies may offer valuable support in the differential diagnosis and early identification of BPDCN." “BPDCN often first presents as a skin lesion and usually has a poor prognosis. There is a dire need for early diagnosis so that patients may access appropriate treatment options,” said Elcin Barker Ergun, CEO of the Menarini Group. “We are delighted to collaborate with VisualDx to provide healthcare teams with a tool using the latest artificial intelligence/machine learning (AI/ML) technology to help interpret challenging skin lesions.” VisualDx does not store any images uploaded by clinicians taking a picture of their patients’ skin exams. This helps maintain the patient’s privacy. About BPDCNBPDCN, formerly blastic NK-cell lymphoma, is an aggressive, orphan hematologic malignancy, often with cutaneous manifestations, with historically poor outcomes. BPDCN typically presents skin lesions and may also involve bone marrow, blood, central nervous system, lymph nodes and viscera. The BPDCN cell of origin is the plasmacytoid dendritic cell (pDC) precursor. The diagnosis of BPDCN is based on the immunophenotypic diagnostic triad of CD123, CD4, and CD56, as well as other markers. The World Health Organization (WHO) termed this disease “BPDCN” in 2008; previous names included blastic NK cell lymphoma and CD4+/CD56+ hematodermic neoplasm. About ELZONRIS® (Tagraxofusp-erzs)U.S. Indication: ELZONRIS is a prescription medicine used to treat blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and pediatric patients 2 years and older. Full prescribing information for the U.S. can be found at www.elzonris.com IMPORTANT SAFETY INFORMATION, ELZONRIS®Boxed WARNING: CAPILLARY LEAK SYNDROME Capillary Leak Syndrome (CLS) which may be life-threatening or fatal, can occur in patients receiving ELZONRIS. Monitor for signs and symptoms of CLS and take actions as recommended. Warnings and Precautions Capillary Leak Syndrome Capillary leak syndrome (CLS), including life-threatening and fatal cases, has been reported among patients treated with ELZONRIS. In patients receiving ELZONRIS in clinical trials, the overall incidence of CLS was 53% (65/122), including Grade 1 or 2 in 43% (52/122) of patients, Grade 3 in 7% (8/122) of patients, Grade 4 in 1% (1/122) of patients, and four fatalities (3%). The median time to onset was 4 days (range - 1 to 46 days), and all but 5 patients experienced an event in Cycle 1. Before initiating therapy with ELZONRIS, ensure that the patient has adequate cardiac function and serum albumin is greater than or equal to 3.2 g/dL. During treatment with ELZONRIS, monitor serum albumin levels prior to the initiation of each dose of ELZONRIS and as indicated clinically thereafter, and assess patients for other signs or symptoms of CLS, including weight gain, new onset or worsening edema, including pulmonary edema, hypotension or hemodynamic instability. Hypersensitivity Reactions ELZONRIS can cause severe hypersensitivity reactions. In patients receiving ELZONRIS in clinical trials, hypersensitivity reactions were reported in 43% (53/122) of patients treated with ELZONRIS and were Grade ≥ 3 in 7% (9/122). Manifestations of hypersensitivity reported in ≥ 5% of patients include rash, pruritus, and stomatitis. Monitor patients for hypersensitivity reactions during treatment with ELZONRIS. Interrupt ELZONRIS infusion and provide supportive care as needed if a hypersensitivity reaction should occur. Hepatotoxicity Treatment with ELZONRIS was associated with elevations in liver enzymes. In patients receiving ELZONRIS in clinical trials, elevations in ALT occurred in 79% (96/122) and elevations in AST occurred in 76% (93/122). Grade 3 ALT elevations were reported in 26% (32/122) of patients. Grade 3 AST elevations were reported in 30% (36/122) and Grade 4 AST elevations were reported in 3% (4/122) of patients. Elevated liver enzymes occurred in the majority of patients in Cycle 1 and were reversible following dose interruption. Monitor alanine aminotransferase (ALT) and aspartate aminotransferase (AST) prior to each infusion with ELZONRIS. Withhold ELZONRIS temporarily if the transaminases rise to greater than 5 times the upper limit of normal and resume treatment upon normalization or when resolved. Adverse Reactions Most common adverse reactions (incidence ≥ 30%) are capillary leak syndrome, nausea, fatigue, pyrexia, peripheral edema, and weight increase. Most common laboratory abnormalities (incidence ≥ 50%) are decreases in albumin, platelets, hemoglobin, calcium, and sodium, and increases in glucose, ALT and AST. Please see full Prescribing Information, including Boxed WARNING.To report SUSPECTED ADVERSE REACTIONS, contact Stemline Therapeutics, Inc. at 1-877-332-7961 or contact the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. About The Menarini GroupThe Menarini Group is a leading international pharmaceutical and diagnostics company, with a turnover of over $4.7 billion and over 17,000 employees. Menarini is focused on therapeutic areas with high unmet needs with products for cardiology, oncology, pneumology, gastroenterology, infectious diseases, diabetology, inflammation, and analgesia. With 18 production sites and 9 Research and Development centers, Menarini's products are available in 140 countries worldwide. For further information, please visit menarini.com. About Stemline Therapeutics Inc.Stemline Therapeutics, Inc. (“Stemline”), a wholly-owned subsidiary of the Menarini Group, is a commercial-stage biopharmaceutical company focused on bringing transformational oncology treatments to patients. Stemline commercializes elacestrant, an oral endocrine therapy indicated for the treatment of postmenopausal women or adult men with estrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative, ESR1-mutated advanced or metastatic breast cancer with disease progression following at least one line of endocrine therapy, in the U.S., Europe, and other global regions. Stemline also commercializes tagraxofusp-erzs, a novel targeted therapy directed to CD123, for patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), an aggressive hematologic cancer, in the United States, Europe, and other global regions. In addition, Stemline commercializes selinexor, an XPO1 inhibitor for multiple myeloma, in Europe. The company is also conducting multiple label-expansion studies with elacestrant and tagraxofusp in breast and hematologic cancer indications, respectively, and has an extensive clinical pipeline of additional drug candidates in various stages of development for a host of solid and hematologic cancers. About VisualDxVisualDx is a company dedicated to improving medical decisions through augmented thinking and timely visualization. It is committed to reducing disparities in medicine and believes technology can bridge gaps in knowledge to bring about more equitable care. VisualDx has become the standard professional resource at more than 2,300 hospitals, clinics and medical schools worldwide by combining problem oriented clinical search with the world's best curated medical image library, plus medical knowledge from experts and sophisticated machine learning algorithms to help with differential diagnosis, variation, treatment, and patient communication. Learn more at www.visualdx.com Media ContactsThe Menarini GroupValeria Speroni CardiEmail: pressoffice@menarini.com Stemline Therapeutics, Inc.Cheya PopeEmail: media@menarinistemline.com 1 Pagano L, et al Haematological. 2013;98 (2): 239-246 and Pemmaraju N Curr Hematol Malig Rep. 2017; 12(6): 510-512

上市批准临床结果

2025-01-11

·药时代

5.5亿美元！英矽智能与美纳里尼再次达成合作

（限时福利直播）如何借助AI使用PASS和R软件计算样本量英矽智能与美纳里尼二次合作，5.5亿美元对外授权AI辅助发现的临床前抗肿瘤管线。这是美纳里尼从英矽智能处授权引进的第二款候选药物，该药物是通过英矽智能生成式AI平台Chemistry42辅助发现的。一年前，美纳里尼从英矽智能处授权引进了临床前候选药物KAT6抑制剂（MEN2312），这个项目目前已经进入了临床阶段。根据协议，美纳里尼将获得该候选药物后续的全球独家开发和商业化的权力。合作交易总额逾5.5亿美元，包括 2000 万美元的首付款，以及后续开发、监管和商业里程碑。此外，英矽智能还将有资格获得净销售额的分级版税。国际领先的制药和诊断公司美纳里尼集团（"美纳里尼"）与其全资子公司 Stemline Therapeutics，和由人工智能（AI）驱动的临床阶段生物科技公司英矽智能达成一项授权许可协议。Stemline将获得英矽智能一款临床前候选药物的全球独家开发和商业化的权力，这款药物有望满足肿瘤领域未被满足的大量临床需求。该候选药物由英矽智能顶尖的药物研发团队利用公司自有的生成化学引擎Chemistry42辅助开发，是一款高选择性、潜在同类最佳（best-in-class）小分子抑制剂，有望用于多种实体瘤癌症的治疗。在临床前研究中，该分子在选定的多个癌种模型中显示出广泛的抗肿瘤活性，目前已被提名为项目的临床前候选药物，正在IND-enabling开发阶段。美纳里尼集团首席执行官Elcin Barker Ergun表示， “ 我们很高兴能与生成式AI领域的领跑者英矽智能开展二次合作，开发一种有望用于多种癌症的高选择性、潜在的同类最佳小分子抑制剂。这项资产将帮助我们进入存在大量未被满足需求的抗肿瘤领域，扩大我们用突破性疗法帮助癌症患者的能力。 ” 英矽智能创始人兼首席执行官Alex Zhavoronkov 博士表示， “ 此前与美纳里尼合作的经验证明，我们的合作伙伴高效、敏捷、具有战略眼光，致力于快速为癌症患者提供最佳的创新治疗方案，并最大限度地提高项目的成功率。美纳里尼集团卓有远见的管理层正在迅速重塑肿瘤治疗领域，我们非常高兴能参与到他们延长全球患者生命的探索中来。 ” 根据协议，该合作交易总额逾5.5亿美元，Stemline 将向英矽智能支付2000万美元的首付款，以及后续开发、监管和商业里程碑付款。此外，英矽智能还将有资格获得净销售额的分级版税。在此之前，美纳里尼与英矽智能于 2024 年 1 月签订了 MEN2312的独家许可协议，这是一种用于乳腺癌治疗和其他肿瘤适应症的创新小分子药物。关于MEN2312 MEN2312由英矽智能的研发团队在其端到端生成AI药物发现平台的帮助下设计而成，可抑制KAT6A并在转录水平阻断ERα表达，使其有潜力克服因ESR1突变带来的内分泌疗法耐药问题。在临床前研究中，该分子已在多个细胞系移植瘤模型(CDX)和人源异种移植模型(PDX)中显示出对KAT6A的强效抑制作用，体现了卓越的疗效和良好的安全性。关于英矽智能英矽智能是一家由生成式人工智能（AI）驱动的临床阶段药物研发公司，通过下一代人工智能系统连接生物学、化学和临床试验分析，利用深度生成模型、强化学习、转换模型等现代机器学习技术，构建强大且高效的人工智能药物研发平台，识别全新靶点并生成具有特定属性分子结构的候选药物。英矽智能聚焦癌症、纤维化、免疫、中枢神经系统疾病、衰老相关疾病等未被满足医疗需求领域，推进并加速创新药物研发。更多信息，请访问网站www.insilico.com 媒体垂询，请联系media@insilico.com 关于美纳里尼集团美纳里尼集团(Menarini Group)是一家领先的国际制药和诊断公司，营业额超过44亿美元，拥有17000多名员工。美纳里尼专注于需求亟待满足的治疗领域，产品包括心脏病学、肿瘤学、肺病学、胃肠病学、传染病学、糖尿病学、炎症和镇痛学。美纳里尼拥有18个生产基地和9个研发中心，产品销往全球140个国家/地区。如需了解更多信息，请访问www.menarini.com。关于Stemline Therapeutics Inc. Stemline Therapeutics, Inc.（下称"Stemline"）是美纳里尼集团的全资子公司，是一家商业阶段的生物制药公司，专注于创新肿瘤疗法的开发和商业化。在美国和欧盟，Stemline实现了ORSERDU®（elacestrant，艾拉司群）的商业化，这是一种口服内分泌疗法，获批用于治疗局部晚期或转移性乳腺癌的绝经后女性或者成年男性，患者具有ER+/HER2-和ESR1突变，并且接受了至少一种内分泌治疗后病情出现进展。Stemline同时推进了ELZONRIS®（tagraxofusp-erzs）商业化，这是一种靶向CD123的新型治疗药物，获批用于一种侵袭性癌症母细胞浆细胞样树突状细胞肿瘤（BPDCN）患者治疗，这也是迄今为止美国和欧盟唯一批准的治疗BPDCN的疗法。在欧洲Stemline还实现了NEXPOVIO®的商业化，这是一种治疗多发性骨髓瘤的XPO1抑制剂。此外，Stemline拥有广泛的处于不同临床阶段的小分子和生物制剂管线，用于治疗多种实体肿瘤和血液肿瘤。封面图来源：网络点击查看更多精彩！

引进/卖出临床申请突破性疗法

100 项与 Tagraxofusp-ERZS 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	Tagraxofusp-ERZS	L01XX67	DB14731

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
Blastic浆细胞样树突状细胞肿瘤	美国	Stemline Therapeutics, Inc.	2018-12-21

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
CD123阳性急性髓性白血病	临床2期	美国	Stemline Therapeutics, Inc.	2025-01-14
CD123阳性急性髓性白血病	临床2期	澳大利亚	Stemline Therapeutics, Inc.	2025-01-14
急性髓性白血病	临床2期	美国	Stemline Therapeutics, Inc.	2024-05-01
骨髓增生异常综合征	临床2期	美国	The University of Texas MD Anderson Cancer Center	2022-03-04
骨髓增生性疾病	临床2期	美国	The University of Texas MD Anderson Cancer Center	2022-03-04
淋巴母细胞淋巴瘤	临床2期	美国	The University of Texas MD Anderson Cancer Center	2022-02-17
复发性 B 细胞急性淋巴细胞白血病	临床2期	美国	The University of Texas MD Anderson Cancer Center	2022-02-17
难治性 B 细胞急性淋巴细胞白血病	临床2期	美国	The University of Texas MD Anderson Cancer Center	2022-02-17
难治性 T 急性淋巴细胞白血病	临床2期	美国	The University of Texas MD Anderson Cancer Center	2022-02-17
原发性骨髓纤维化	临床2期	意大利	Menarini Von Heyden GmbH	2022-01-30

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临床结果

适应症

分期

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


ASH2024	N/A	Blastic浆细胞样树突状细胞肿瘤	-	Tagraxofusp	繭齋糧憲壓鏇蓋鏇壓糧(選遞餘鬱觸鏇鑰築願鹹) = 獵繭廠鑰醖鏇壓壓壓繭範構窪構選艱顧衊鹽鬱 (網齋製襯糧獵窪願積齋 ) 更多	-	2024-12-08
AML-323 (SOHO2024)	N/A	CD123	40	TAG 12 mcg/kg	網憲願鏇憲壓艱積齋觸(餘願簾製範築鏇獵鬱淵) = 齋齋窪鬱糧鹹繭積憲觸膚範醖獵餘淵淵鹹顧糧 (網憲範觸艱膚願鏇遞艱 ) 更多	积极	2024-09-04
NCT02270463 (CTgov)	临床1/2期	急性髓性白血病 \| 残留肿瘤	16	Tagraxofusp-erzs (Stage 1: Tagraxofusp-erzs 7 µg/kg/Day)	鹹齋淵鏇製膚鏇選膚衊 = 積獵壓襯鹽衊窪糧獵鬱窪鬱蓋壓醖鬱選範製廠 (製獵膚艱獵鬱顧鬱選醖, 顧鬱觸遞鏇獵鑰餘膚衊 ~ 憲積簾夢顧築選築構廠) 更多	-	2024-08-21
NCT02270463 (CTgov)	临床1/2期	急性髓性白血病 \| 残留肿瘤	16	Tagraxofusp-erzs (Stage 1: Tagraxofusp-erzs 9 µg/kg/Day)	鹹齋淵鏇製膚鏇選膚衊 = 壓夢築襯糧繭壓衊醖鑰窪鬱蓋壓醖鬱選範製廠 (製獵膚艱獵鬱顧鬱選醖, 顧願壓鹽夢糧選糧衊醖 ~ 餘範襯築願蓋糧壓網選) 更多	-	2024-08-21
NCT04317781 (CTgov)	临床2期	Blastic浆细胞样树突状细胞肿瘤	3	Tagraxofusp-erzs	淵夢選膚廠壓遞獵簾窪 = 窪淵顧築鏇築膚觸衊夢顧築簾窪襯艱廠鹽蓋鹹 (窪襯築鑰選夢糧夢艱夢, 網夢廠獵艱襯觸獵鏇築 ~ 顧鑰壓鹽鏇夢衊夢鏇顧) 更多	-	2024-07-16
NPP (EHA2024)	N/A	Blastic浆细胞样树突状细胞肿瘤 CD123	18	TAG 12 mcg/kg	鹹鏇糧廠構觸選艱鹽築(齋築艱顧鏇衊夢壓艱蓋) = 鏇夢鏇選窪廠簾鑰膚願廠築壓簾窪膚選製鏇膚 (憲糧壓淵製範築積窪襯 ) 更多	积极	2024-05-14
NCT03113643 (Pubmed) 人工标引	临床1期	急性髓性白血病	56	Tagraxofusp + AzacitidineVenetoclax	觸簾淵廠襯醖觸齋淵膚(醖窪憲夢憲餘淵築築蓋) = 鏇製鬱鹽膚憲獵憲選積築醖遞壓壓淵鹹獵蓋餘 (夢醖鑰遞憲製夢鏇鹽夢 ) 更多	积极	2024-02-13
NCT03113643 (Pubmed) 人工标引	临床1期	急性髓性白血病	56	Tagraxofusp + Azacitidine + Venetoclax (expansion cohort)	觸簾淵廠襯醖觸齋淵膚(醖窪憲夢憲餘淵築築蓋) = 壓蓋鹹構鑰窪鏇鹹觸憲築醖遞壓壓淵鹹獵蓋餘 (夢醖鑰遞憲製夢鏇鹽夢 ) 更多	积极	2024-02-13
ASH2023	临床1期	CD123阳性急性髓性白血病	26	Tagraxofusp	鑰鏇顧窪選糧糧構衊餘(淵築範壓顧鑰鹹範壓糧) = 淵構選鬱繭廠網餘簾鹹鑰餘鹽襯窪網構鬱餘願 (壓衊網壓獵衊餘窪襯遞, 14.0 ~ NA) 更多	-	2023-12-11
ASH2023	临床1期	CD123阳性急性髓性白血病	26	Azacitidine	蓋鹹製選鏇鹹廠製鬱願(鹹鹽齋鑰襯鹽餘鬱鹹衊) = 餘淵餘鹹築憲窪獵艱獵鹹餘範夢製餘簾鹽繭糧 (觸鏇網選繭夢鹽網範簾 )	-	2023-12-11
NCT04342962 (GIMEMA AML2020, ASH2023)	临床2期	CD123阳性急性髓性白血病	23	Tagraxofusp	鹹繭繭廠繭網鏇憲壓願(齋糧顧鏇鑰壓簾壓製憲) = 1 patient receiving 4 courses achieved PR and died due to CNS bleeding 鬱艱網餘醖憲齋觸壓簾 (鏇糧鹹鏇築製窪糧顧窪 ) 更多	-	2023-12-10
ASH2023	N/A	Blastic浆细胞样树突状细胞肿瘤一线	22	Tagraxofusp	齋齋簾鏇憲齋觸窪築網(繭艱餘積網膚製窪鏇鑰) = 餘醖蓋艱網夢觸壓壓窪範獵築衊蓋夢選鑰範淵 (艱襯願齋築構顧艱顧顧, 10 ~ NR) 更多	-	2023-12-10