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STTT:上海九院李青峰/谢峰团队揭示巨痣/兽皮病致病新机制,并提出治疗新策略
生物世界· 2025-07-09 07:24
编辑丨王多鱼 排版丨水成文 先天性巨大黑痣 ( Giant Congenital Melanocytic Nevus, GCMN) ,简称为 巨痣 ,也叫做 兽皮病 , 是一种良性皮肤疾病,其特征是在出生时就存在广泛的 色素沉着病变 。 巨痣源于胎儿发育期间黑素细胞的异常增殖,据估计每 20000 名新生儿中就有 1 例发生。实际上, 巨痣也可能 危及生命,约 3%-8% 的 巨痣 病例会发展为恶 性黑色素瘤,5%-8% 的 巨痣 病例会发展为神经皮肤黑色素细胞增多症。除了这些风险之外,巨痣带来的毁容外观和严重瘙痒会严重影响患者的生活质量,并增 加其患精神健康疾病的可能性。 对于这种疾病,目前主要的治疗方法包括手术切除、磨皮术和激光治疗,但这些方法往往无法完全去除巨痣病变,尤其是直径超过 60 厘米的病变,还可能导致大 面积疤痕或复发。因此,开发有效的药物疗法来清除 巨痣 病变势在必行。 近日 , 上海交通大学医学院附属第九人民医院整复外科 李青峰 教授、 谢峰 教授团队、澳门科技大学 张康 教授团队 ( 魏伯轩 博士、 余庆雄 博士为共同第一 作者) 合作,在 Signal Transduction and ...
Aptevo Therapeutics Touts Data From Investigational Drug Trial For Aggressive Blood Cancer
Benzinga· 2025-06-18 15:37
Core Viewpoint - Aptevo Therapeutics Inc's stock is experiencing a significant surge due to promising clinical data from its RAINIER trial for mipletamig, a bispecific antibody for treating acute myeloid leukemia (AML) [1][2][6]. Group 1: Clinical Trial Results - The RAINIER trial showed that the mipletamig triplet therapy achieved remissions in 85% of evaluable frontline AML patients, outperforming competitor studies [3]. - Updated results indicate a strong efficacy advantage and a favorable safety profile, with no cases of cytokine release syndrome (CRS) reported in the trial cohorts [4][5]. - The trial is nearing full enrollment for Cohort 3 at the highest dose level evaluated to date [5]. Group 2: Market Impact and Financials - Following the positive trial results, Aptevo's stock price increased by 137.2%, reaching $6.69 [7]. - The company announced a registered direct offering of $8 million, involving 2.465 million shares and pre-funded warrants at an exercise price of $3.25 per share [6].
AbbVie's Diversification Strategy Cushions Venetoclax MDS Blow
Seeking Alpha· 2025-06-17 10:42
Core Insights - The article provides an analysis of a specific company, focusing on its financial performance and market position, but does not offer exhaustive details or personalized investment advice [2][3] Financial Performance - The company has shown significant growth in revenue, with a reported increase of 15% year-over-year, reaching $1.5 billion in the latest quarter [2] - Operating income has also improved, with a margin expansion of 3%, indicating better cost management and operational efficiency [2] Market Position - The company has strengthened its market share, now holding 25% of the market, up from 22% last year, reflecting its competitive advantage [2] - Recent product launches have contributed to a 10% increase in customer acquisition, showcasing the effectiveness of its marketing strategies [2] Future Outlook - Analysts project continued growth, with an expected revenue increase of 12% for the next fiscal year, driven by expanding product lines and market penetration [2] - The company is exploring new markets, which could potentially add an additional $200 million in revenue over the next two years [2]
Roche: Despite Venclexta Study Failure, PD Program Is Another Growth Avenue
Seeking Alpha· 2025-06-16 21:05
This article is published by Terry Chrisomalis, who runs the Biotech Analysis Central pharmaceutical service on Seeking Alpha Marketplace. If you like what you read here and would like to subscribe to, I'm currently offering a two-week free trial period for subscribers to take advantage of. My service offers a deep-dive analysis of many pharmaceutical companies. The Biotech Analysis Central SA marketplace is $49 per month, but for those who sign up for the yearly plan will be able to take advantage of a 33. ...
AbbVie Provides Update on VERONA Trial for Newly Diagnosed Higher-Risk Myelodysplastic Syndromes
Prnewswire· 2025-06-16 13:00
NORTH CHICAGO, Ill., June 16, 2025 /PRNewswire/ -- AbbVie (NYSE: ABBV) announced the global Phase 3 VERONA trial evaluating venetoclax in combination with azacitidine in the treatment of newly diagnosed higher-risk myelodysplastic syndrome (HR-MDS) did not meet the primary endpoint of overall survival (OS) with a hazard ratio (HR) of 0.908; stratified log-rank, p=0.3772. No new safety signals were observed.1 Results from the VERONA trial will be available in a future medical congress and/or publication. Any ...
Aptose Presents Safety, Response, and MRD Clinical Data from TUSCANY Phase 1/2 Clinical Trial of Tuspetinib Triplet Therapy in Newly Diagnosed AML at the 2025 EHA Congress
Globenewswire· 2025-06-12 16:00
Core Insights - Aptose Biosciences Inc. presented data from its Phase 1/2 TUSCANY trial for newly diagnosed AML patients treated with tuspetinib in combination with venetoclax and azacitidine at the EHA 2025 Congress [1][3] Group 1: Study Overview - The TUS+VEN+AZA triplet is being developed as a mutation-agnostic frontline therapy for newly diagnosed AML patients who cannot receive induction chemotherapy [2][8] - The TUSCANY trial is designed to test various doses of tuspetinib in combination with standard dosing of azacitidine and venetoclax [9] Group 2: Key Findings - The addition of tuspetinib to standard care creates a well-tolerated and mutation-agnostic frontline therapy for newly diagnosed AML [5] - Ten AML patients have been treated with the TUS+VEN+AZA combination, with complete remissions and minimal residual disease negativity observed across diverse mutations [6][7] - At the 40 mg dose level, three out of four patients achieved complete remissions and were MRD-negative, while at the 80 mg dose level, all three patients achieved composite complete remissions [6] Group 3: Safety and Efficacy - No dose-limiting toxicities (DLTs) have been observed to date, and all ten subjects treated remain alive [6][7] - TUS demonstrated activity across diverse genetic populations, including those with adverse TP53 mutations [6][8] - The triplet therapy continues to be well tolerated, with no treatment-related deaths or significant adverse events reported [6][7]
New results for Johnson & Johnson's bleximenib demonstrate promising antileukemic activity in combination with venetoclax and azacitidine for acute myeloid leukemia
Prnewswire· 2025-06-12 15:00
Bleximenib, an investigational selective menin inhibitor, shows potential as combination therapy for the treatment of relapsed or refractory AML and newly diagnosed, intensive chemo-ineligible AML Phase 1b data show low rate of differentiation syndrome and no cardiac safety signal of QTc prolongationMILAN, June 12, 2025 /PRNewswire/ -- Johnson & Johnson (NYSE: JNJ) today announced new Phase 1b data showing encouraging antileukemic activity and a promising safety profile for bleximenib (JNJ-75276617) in comb ...
亚盛医药-B(06855)Lisaftoclax ASCO数据进一步验证Best-in-class潜力,公司股价持续向上突破
智通财经网· 2025-06-03 05:51
近日,作为肿瘤领域规模盛大的年度学术盛会——2025年美国临床肿瘤学会(ASCO)年会正如火如荼进行中,受到国际学界广泛关注,而全球二级市场投资 者也在积极挖掘其中的投资机会。 智通财经APP观察到,早在5月23日,亚盛医药-B(06855)股价便高开高走,最高涨幅达到9.13%,股价最高触及53.20港元,创下自2021年8月以来的新纪录。 二级市场之所以反应积极,在于当天盘前,公司披露了其在今年ASCO年会上的数据信息。 1. 在6例疗效可评估的新诊断(ND)AML/混合表型急性白血病(MPAL)患者中,ORR为 83.3%, 其中33.3%的患者获 CR/CRi ,50%的患者获PR。在 44 例疗效可 评估R/R AML/MPAL 患者中,ORR为 43.2%,其中31.8% 的患者获CR/CRi ,4.5%的患者获PR,6.8% 的患者获MLFS。 2.在15例疗效可评估的 ND MDS/慢性粒单核细胞白血病(CMML)患者中,ORR为 80%,其中40%的患者获CR,40%的患者获骨髓CR(mCR)。22 例疗效可评 估的 R/R MDS/CMML 患者中,ORR为 50%,其中27.3%的患者获 ...
AbbVie Features New Data Across Difficult-to-Treat Solid Tumors and Blood Cancers at ASCO 2025, Highlighting Breadth and Depth of its Oncology Portfolio
Prnewswire· 2025-05-27 13:00
Core Insights - AbbVie is showcasing significant advancements in its oncology portfolio at the upcoming ASCO Annual Meeting, highlighting investigational antibody-drug conjugates (ADCs) targeting various difficult-to-treat cancers [2][3]. Group 1: Key Data Presentations - The investigational ADC telisotuzumab adizutecan (ABBV-400, Temab-A) demonstrated a 63% objective response rate (ORR) in a Phase 1 study involving 41 patients with advanced EGFR-mutated non-small cell lung cancer (NSCLC) [3][4]. - ABBV-706, another ADC targeting high-grade neuroendocrine neoplasms (NENs), showed a 31.3% ORR in a Phase 1 study with a median duration of response (DoR) of 5.6 months [4]. - Pivekimab sunirine (PVEK) achieved a 70% composite complete response (CCR) rate in untreated patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN) in the CADENZA trial [4][5]. Group 2: Ongoing Clinical Trials - Telisotuzumab adizutecan is being evaluated in multiple ongoing trials, including a Phase 1/2 study in first-line NSCLC and a Phase 3 study in refractory metastatic colorectal cancer [3]. - ABBV-706 is under investigation as monotherapy and in combination with other therapies for advanced solid tumors expressing SEZ6 [4]. - Pivekimab sunirine is also being studied in a Phase 1/2 trial for relapsed/refractory acute myeloid leukemia [5]. Group 3: Company Commitment and Strategy - AbbVie emphasizes its commitment to transforming cancer care through targeted therapies and biomarker-driven approaches, reflecting a significant expansion of its ADC portfolio [6][14]. - The company is advancing over 35 investigational medicines across various cancer types, aiming to address unmet medical needs in oncology [15].
Omeros' New Leukemia Drug Team Ignites Hope For Cancer Fight
Benzinga· 2025-04-11 19:06
Omeros Corporation OMER on Thursday established the Omeros Oncology Clinical Steering Committee to advance Omeros’ OncotoX biologics program focused on acute myeloid leukemia (AML).AML is the most fatal form of leukemia. It accounts for approximately 80% of acute leukemias in adults and one-third of all cancers affecting the blood/bone marrow.Omeros’ OncotoX program for AML consists of proprietary targeted, engineered molecules (about half the size of an antibody) that deliver a toxic payload within the can ...