Tagraxofusp-ERZS

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

（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！