GATE1: A Multicenter Phase II Study of Pirtobrutinib, Rituximab and Venetoclax Combination Therapy for Patients With Previously Untreated Mantle Cell Lymphoma

Primary Objectives:
To estimate the percent of participants who achieve a best response of complete response by the end of the PRV combination therapy in the induction therapy phase in patients with previously untreated MCL.

开始日期2025-12-01

申办/合作机构

M.D. Anderson International España SA

NCT06572618 / Not yet recruiting临床2期

A Phase 2 Study of BTK Inhibitor Nemtabrutinib in Combination With Rituximab in Patients With Previously Untreated Mantle Cell Lymphoma

This phase II trial tests how well nemtabrutinib works with rituximab for the treatment of patients with mantle cell lymphoma. Nemtabrutinib is in a class of medications called kinase inhibitors. It blocks a protein called BTK, which is present on B-cell (a type of white blood cells) cancers such as mantel cell lymphoma at abnormal levels. This may help keep cancer cells from growing and spreading. Rituximab is a monoclonal antibody. It binds to a protein called CD20, which is found on B cells (a type of white blood cell) and some types of cancer cells. This may help the immune system kill cancer cells. Giving nemtabrutinib with rituximab may kill more cancer cells in patients with mantle cell lymphoma.

开始日期2025-02-11

申办/合作机构

Hope Medical Center

[+1]

NCT06563596 / Not yet recruiting临床2期IIT

A Phase 2 Study of Epcoritamab, Zanubrutinib, and Rituximab (EZR) for Treatment of Relapsed or Refractory Follicular Lymphoma

The purpose of this study is to determine how effective and safe the combination of epcoritamab, zanubrutinib, and rituximab is in treating participants with relapse or refractory Follicular Lymphoma (FL).
* The names of the study drugs involved in this research study are:
* Epcoritamab (a type of antibody)
* Zanubrutinib (a type of Bruton tyrosine kinase inhibitor)
* Rituximab (a type of monoclonal antibody)

开始日期2025-02-01

申办/合作机构

Dana-Farber Cancer Institute, Inc.

[+2]

100 项与 BTK 相关的临床结果

登录后查看更多信息

100 项与 BTK 相关的转化医学

登录后查看更多信息

0 项与 BTK 相关的专利（医药）

登录后查看更多信息

3,850

项与 BTK 相关的文献（医药）

2024-12-01·INTERNATIONAL IMMUNOPHARMACOLOGY

Bruton’s tyrosine kinase: A promising target for treating systemic lupus erythematosus

Review

作者: Zhou, Yu ; Sun, Lan ; Feng, Yifan ; Ding, Qiaoyi ; Zhang, Tiantai

Systemic lupus erythematosus (SLE) is a chronic systemic autoimmune disorder involving multiple organs and systems. There is growing evidence that autoreactive B cells occupy a central role in the occurrence and progression of SLE due to their ability to generate pathogenic autoantibodies. Small molecule inhibitors targeting Bruton's tyrosine kinase (BTK), a crucial intracellular kinase regulating B cell development and function, emerge as a new strategy to treat SLE in recent years and are superior to biologic agents depleting B cells in many aspects. Supportive data obtained from lupus-prone mice preliminarily demonstrated the promising therapeutic potential of BTK inhibition. However, these BTK inhibitors, including elsubrutinib, evobrutinib, etc., mostly face with unsatisfactory efficacy and certain safety issues during clinical use, driving the quest for new-generation inhibitors with improved potency and higher selectivity. This paper elaborates the importance of BTK involvement in SLE pathogenesis, reviews the clinical research progress of BTK inhibitors for SLE and discusses limitations and challenges the drugs met in development, in order to contribute to a deeper understanding of disease mechanism and provide a reference for new-generation BTK inhibitor research.

2024-11-01·JOURNAL OF NUTRITIONAL BIOCHEMISTRY

The protective effect of dulcitol on lipopolysaccharide-induced intestinal injury in piglets: mechanistic insights

Article

作者: Zheng, Zi ; Wang, Han ; Chen, Liang ; Yan, Jun ; Zhong, Ruqing ; Li, Ning ; Han, Hui ; Liu, Zhengqun ; Liang, Shiyue ; Mu, Shuqin

This study investigated the protective effect of dulcitol on LPS-induced intestinal injury in piglets and explored the underlying molecular mechanisms. A total of 108 piglets were divided into three groups: CON, LPS, and DUL. The CON and LPS groups were fed a basal diet, the DUL group was fed a diet supplementation with 500 mg/kg dulcitol. On day 29, 6 piglets in the LPS and DUL groups were injected with 100 μg/kg BW of LPS. At 4 h post-challenge, all pigs were slaughtered, and colonic samples were collected. Results showed that dulcitol supplementation boosted intestinal barrier function in LPS-challenged piglets by enhancing intestinal morphology and integrity, and increasing the gene expression of zonula occludens-1, claudin-1, and occludin in the colonic mucosa (P <0.05). Metabolomics showed DUL supplementation mainly increased (P <0.05) the metabolites related to steroid and vitamin metabolism (Cholesterol and Vitamin C). Proteomics showed that dulcitol supplementation altered the protein expression involved in maintaining barrier integrity (FN1, CADM1, and PARD3), inhibiting inflammatory response (SLP1, SFN, and IRF3), and apoptosis (including FAS, ING1, BTK, MTHFR, NOX, and P53BP2) in LPS-challenged piglets (P <0.05). Additionally, dulcitol addition also suppressed the TLR4/NF-κB signaling pathway and apoptosis in mRNA and protein levels. Dulcitol increased the abundance of short-chain fatty acid-producing bacteria (Lactobacillus, Blautia, and Faecalibacterium) at the genus level, but decreased the relative abundance of Proteobacteria at the phylum level and Pseudomonas and Delftia at the genus level in piglets (P < 0.05). In conclusion, these results suggested that the addition of dulcitol alleviated LPS-induced intestinal barrier injury in piglets, probably by maintaining its integrity, inhibiting the TLR4/NF-κB signaling pathways and apoptosis, and modulating the gut microbiota. Therefore, dulcitol can be considered a potential dietary additive for improving intestinal health in pig models.

2024-11-01·FOOD RESEARCH INTERNATIONAL

Rosa roxburghii Tratt (Cili) has a more effective capacity in alleviating DSS-induced colitis compared to Vitamin C through B cell receptor pathway

Article

作者: Deng, Bin ; Wang, Fei ; Yang, RongChang ; Wang, Qi ; Li, Xiang ; Fu, Aikun ; Zhang, Qiao ; Li, Weifen ; Li, Fuyong ; Jin, Qian

Rosa roxburghii Tratt (RRT), a traditional Chinese plant known as the 'King of Vitamin C (VitC; ascorbic acid, AsA)', contains a wealth of nutrients and functional components, including polysaccharides, organic acids, flavonoids, triterpenes, and high superoxide dismutase (SOD) activity. The various functional components of RRT suggest that it may theoretically have a stronger potential for alleviating colitis compared to VitC. This study aims to verify whether RRT has a stronger ability to alleviate colitis than equimolar doses of VitC and to explore the mechanisms underlying this improvement. Results showed that RRT significantly mitigated body weight loss, intestinal damage, elevated inflammation levels, and compromised barriers in mice induced by Dextran sulfate sodium (DSS). Additionally, RRT enhanced the diversity and composition of intestinal microbiota in these DSS-induced mice. Colon RNA sequencing analysis revealed that compared to VitC, RRT further downregulated multiple immune-related signaling pathways, particularly the B cell receptor (BCR) pathway, which is centered around genes like Btk and its downstream PI3K-AKT, NF-κB, and MAPK signaling pathways. Correlation analysis between microbiota and genes demonstrated a significant relationship between the taxa improved by RRT and the key genes in the BCR and its downstream signaling pathways. Overall, RRT exhibited superior capabilities in alleviating DSS-induced colitis compared to VitC by decreasing intestinal inflammation and modulating BCR and its downstream signaling pathways, potentially regulated by the improved intestinal microbiota.

2,776

项与 BTK 相关的新闻（医药）

2024-09-21

·CPHI制药在线

恒瑞医药1类新药申报上市，一线治疗胃癌；达格列净二甲双胍缓释片首仿国内获批 | 制药在线一周药闻复盘

点击蓝字关注我们本周，热点不少。首先是审评审批方面，很值得关注的有两个，一个是达格列净二甲双胍缓释片首仿在中国获批上市，另一个是恒瑞医药一线治疗胃癌的1类新药申报上市；其次是研发方面，多个药取得重要进展，非常值得一说的是，恒瑞帕金森病新药启动Ⅲ期临床以及迪哲医药公布舒沃替尼最新肺癌研究数据，超92%患者靶病灶缩小；最后是交易及投融资方面，欧加农12亿美元收购Dermavant，获得本维莫德乳膏权益。本周盘点包括审评审批、研发、交易及投融资三大板块，统计时间为9.16-9.20，包含27条信息。审评审批 NMPA 上市批准 1、9月18日，NMPA官网显示，宣泰医药的达格列净二甲双胍缓释片获批上市，这是该品种国内首家获批的仿制药。达格列净二甲双胍缓释片是一种SGLT2抑制剂，原研由阿斯利康开发，于2023年6月在国内获批上市，用于治疗2型糖尿病。目前，国内多家药企的该品种仿制药已进入申报上市阶段，包括正大天晴、齐鲁制药、华海药业、石家庄四药及南京方生和等。申请 2、9月18日，CDE官网显示，奥默医药和仙琚制药联合开发的1类新药奥美克松钠注射液申报上市，预测用于治疗拮抗中度肌松和深度肌松。奥美克松钠（Aom0498）是其自主研发的新一代靶向肌松拮抗剂。 3、9月19日，CDE官网显示，恒瑞医药1类新药瑞拉芙普-α注射液（SHR-1701）申报上市，联合氟尿嘧啶类和铂类药物用于局部晚期不可切除、复发或转移性胃及胃食管结合部腺癌的一线治疗。SHR-1701是恒瑞医药自主研发并具有知识产权的抗PD-L1/TGF-βRII双功能融合蛋白，经查询，国内外尚无同类产品获批上市。 4、9月20日，CDE官网显示，EyePoint Pharmaceuticals、欧康维视（Ocumension）等联合开发的地塞米松植入剂（商品名：DEXYCU、OT-502）申报上市。OT-502是一种新型、可生物降解的缓释抗炎药物，旨在为眼科医生提供一种术毕单次注射缓释激素的选择。临床批准 5、9月18日，CDE官网显示，普方生物1类新药注射用PRO1286获批临床，拟用于治疗实体瘤患者。这是一款在研的EGFR与cMET靶向双特异性抗体偶联药物（ADC），2024年5月，Genmab公司已经完成对普方生物的收购，收购金额为18亿美元。 6、9月18日，CDE官网显示，恒瑞医药1类新药SHR-1905注射液获批临床，拟开发用于12-17周岁的青少年哮喘患者。SHR-1905为恒瑞医药研发的TSLP单克隆抗体，此前恒瑞与OneBio公司达成一项高达10.5亿美元的合作，将该产品在大中华区以外的全球范围内开发、生产和商业化独家权利授予OneBio公司。 7、9月18日，CDE官网显示，和记黄埔医药1类新药HMPL-760胶囊获批临床，适应症为联合R-GemOx（利妥昔单抗+吉西他滨+奥沙利铂）治疗复发/难治弥漫大B细胞淋巴瘤（R/RDLBCL）。HMPL-760是一款第三代非共价BTK抑制剂。 8、9月18日，CDE官网显示，上海医药1类新药SPH4336片获批临床，联合奥希替尼治疗KRAS突变型晚期实体瘤。SPH4336是一种新型高选择性口服抑制剂，具有广谱的抗肿瘤作用。该产品对CDK4/6靶点具有较高的选择性，同时具有广谱抗Rb阳性肿瘤活性。 9、9月18日，CDE官网显示，挚盟医药1类新药CB03-154片获批临床，拟开发治疗成人肌萎缩侧索硬化（又称“渐冻症”，ALS）。CB03-154片是一款KCNQ2/3选择性开放剂。该产品此前治疗ALS适应症已经获得美国FDA授予孤儿药资格。 10、9月19日，CDE官网显示，贝达药业的CFT8919胶囊获批临床，拟开发用于携带EGFR突变的非小细胞肺癌患者。这是贝达药业与C4Therapeutics合作开发的一种具有口服生物利用度的变构BiDAC™（双功能蛋白）降解剂。 11、9月19日，CDE官网显示，强生（Johnson&Johnson）1类新药bleximenib胶囊获批临床，拟用于治疗携带组蛋白-赖氨酸N-甲基转移酶2A重排（KMT2Ar）或核磷蛋白1基因突变（NPM1m）的复发/难治性急性髓系白血病（AML）成人患者。bleximenib是一款Menin-KMT2A相互作用抑制剂，正在国际范围内开展Ⅰ/Ⅱ期临床研究。 12、9月20日，CDE官网显示，百济神州1类新药注射用BGB-C354获批临床，拟开发治疗晚期实体瘤患者。BGB-C354是一款靶向B7-H3的抗体偶联药物（ADC），是百济神州在实体瘤领域布局的一款新分子实体，本次为该产品首次在中国获批临床。优先审评 13、9月19日，CDE官网显示，乐普生物的注射用维贝柯妥塔单抗（MRG003）拟纳入优先审评，用于治疗既往经至少二线系统化疗和PD-1/PD-L1抑制剂治疗失败的复发或转移性鼻咽癌（NPC）患者。该药物是进度最快的国产EGFR ADC。 FDA 上市批准 14、9月17日，FDA官网显示，默沙东的PD-1单抗帕博利珠单抗（商品名：Keytruda、K药）获批新适应症，用于联合培美曲塞和铂类化疗一线治疗不可切除的晚期或转移性恶性胸膜间皮瘤（MPM）。目前，全球仅3款创新疗法获批用于治疗MPM，包括纳武利尤单抗（BMS/小野制药）、TTFields（Novocure/再鼎医药）和K药。 15、9月17日，FDA官网显示，诺华的CDK4/6抑制剂瑞波西利与芳香化酶抑制剂(AI)获批，联合用于激素受体阳性/人类表皮生长因子受体2阴性（HR+/HER2-）的Ⅱ期和Ⅲ期早期乳腺癌(EBC)高复发风险患者（包括淋巴结阴性N0患者）的辅助治疗；广泛适用于复发风险较高的HR+/HER2-Ⅱ期和Ⅲ期EBC，将符合CDK4/6抑制剂辅助治疗条件的人群增加了约一倍。 16、9月18日，FDA官网显示，阿斯利康的贝那利珠单抗（Benralizumab、商品名：Fasenra）获批新适应症，用于治疗嗜酸性肉芽肿性多血管炎（EGPA）。贝那利珠单抗是一款抗IL-5受体（IL-5Rα）单抗，2017年11月，首次在美国获批上市，用于12岁及以上的重度嗜酸性粒细胞性哮喘（SEA）患者的附加维持治疗。2019年10月，预填充一次性自动注射笔获批，至此以后患者可自行注射用药。 17、9月18日，FDA官网显示，再生元和赛诺菲的度普利尤单抗（商品名：Dupixent）递交补充生物制品许可申请（sBLA），用于治疗慢性鼻-鼻窦炎伴鼻息肉（CRSwNP）青少年患者。度普利尤单抗是再生元和赛诺菲合作开发的一款抗IL-4Rα全人源单抗，成为了美国第一种针对12-17岁青少年CRSwNP患者的疗法。研发临床状态 18、9月18日，中国药物临床试验登记与信息公示平台官网显示，赛诺菲（Sanofi）启动了amlitelimab注射液的一项新的国际多中心（含中国）Ⅲ期临床研究，旨在评估该产品针对12岁及以上中重度特应性皮炎患者的疗效和安全性。Amlitelimab是一种皮下注射的完全人源化、非耗竭性单克隆抗体，它能与OX40L结合，旨在恢复促炎及调节性T细胞之间的免疫稳态。 19、9月18日，中国药物临床试验登记与信息公示平台官网显示，石药集团启动一项多中心、随机、开放、阳性对照Ⅲ期临床试验，比较其司美格鲁肽注射液和原研药治疗肥胖患者的有效性和安全性。这是国内第四款启动肥胖Ⅲ期临床的司美格鲁肽。 20、9月19日，Clinicaltrials官网显示，恒瑞登记了一项HRG2010治疗帕金森病的Ⅲ期临床（NCT06598735）。HRG2010胶囊是恒瑞自主研发的卡比多巴+左旋多巴新型复方缓释胶囊，以卡比多巴-左旋多巴缓释片为对照，主要终点指标为第21周「停药」时间与基线相比的变化。目前国内外已上市的同类产品主要包括多巴丝肼片（商品名：美多芭）、恩他卡朋双多巴片（商品名：达灵复）和卡左双多巴缓释片（商品名：息宁）。 21、9月19日，药物临床试验登记与信息公示平台显示，艾伯维登记了一项评估Epcoritamab+利妥昔单抗和来那度胺对比化学免疫疗法在既往未接受过治疗的滤泡性淋巴瘤受试者中的Ⅲ期临床试验。Epcoritamab是全球第3款获批的CD20×CD3双抗。临床数据 22、9月17日，默沙东和第一三共公布了patritumab deruxtecan治疗既往接受过EGFR-TKI治疗的局部晚期或转移性EGFR突变NSCLC患者的Ⅲ期HERTHENA-Lung02研究达到了PFS的主要终点。与铂联合培美曲塞诱导化疗后再接受培美曲塞维持化疗相比，该药治疗后患者实现显著的PFS统计学改善。该药是一款采用第一三共技术设计的潜在first-in-class靶向HER3的DXd抗体偶联药物（ADC）。 23、9月17日，迪哲医药公布了高选择性EGFR酪氨酸激酶抑制剂舒沃替尼片（商品名：舒沃哲）国际多中心注册临床研究“悟空1B”（WU-KONG1PartB、WU-KONG1B）临床数据。研究显示，舒沃替尼二/后线治疗EGFR20号外显子插入突变（exon20ins）非小细胞肺癌（NSCLC）各亚组获益与全球整体人群一致。2023年8月，该药在中国获批上市。 24、9月18日，阿斯利康（AstraZeneca）公布了第三代EGFR-TKI奥希替尼治疗Ⅲ期不可切除的EGFR突变非小细胞肺癌的临床（LAURA）结果，与安慰剂相比，奥希替尼将CNS进展或死亡风险降低了83%。此外，奥希替尼治疗的中位至远处转移时间（TTDM）也显示出具有临床意义的改善，12个月时接受奥希替尼治疗组患者TTDM累计发生率为11%，而接受安慰剂组治疗患者为37%。2024年6月，奥希替尼递交了该适应症的上市申请。 25、9月18日，和誉医药公布了小分子FGFR4抑制剂依帕戈替尼（ABSK011）在Ⅰ期临床试验中针对FGF19过表达的晚期肝细胞癌的临床数据。依帕戈替尼220mgBID组在经免疫检查点抑制剂（ICIs）和多靶点小分子酪氨酸激酶抑制剂（mTKIs）治疗的FGF19过表达肝细胞癌患者中展现出优异的有效性，其总体缓解率（ORR）达到44.8%，中位缓解持续时间（mDOR）为7.4个月，中位无进展生存期（mPFS）达到5.5个月。 26、9月19日，Aligos公布了甲状腺激素受体β(THR-β)激动剂ALG-055009在代谢功能障碍相关脂肪性肝炎(MASH)患者中的Ⅱa期HERALD研究的积极顶线结果。第12周，肝脏脂肪相对减少中位数高达46.2%，且剂量反应明显。交易及投融资 27、9月18日，欧加农（Organon）宣布，将收购Roivant子公司Dermavant Sciences。该交易预计将于2024年Q4完成。欧加农预计该交易不会导致其资本配置的优先级发生变化。根据协议条款，收购总交易额约为12亿美元，包括1.75亿美元的首付款、7500万美元的监管里程碑付款以及9.5亿美元的某些商业里程碑付款。 END 【智药研习社直播预告】来源：CPHI制药在线声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。投稿邮箱：Kelly.Xiao@imsinoexpo.com ▼更多制药资讯，请关注CPHI制药在线▼ 点击阅读原文，进入智药研习社~

并购临床3期申请上市抗体药物偶联物上市批准

2024-09-21

·EndPoints

John Carroll on his cancer diagnosis; #ESMO's biggest surprise; Lykos' bad trip; Rate cuts, and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here . What a busy week for news. From ESMO and the Federal Reserve, to the FTC and 23andMe, we’ve got a weekly roundup chock-full of excellent stories. And be sure to sign up for our Endpoints 11 event on Sept. 26 in Boston, celebrating the most promising private biotechs the industry has to offer. — Max Gelman Our founding editor John Carroll was diagnosed with a rare form of cancer a few months ago called Merkel cell carcinoma. After posting semi-regular updates on social media about his search for treatment, John wrote about his diagnosis and the frustrating road he had to take to finally get the right doctors and drugs. If you haven’t already, please take a few minutes out of your weekend to read his article. We’re all rooting for John — Endpoints News wouldn’t be what it is without him. This year’s ESMO conference featured the usual presentations of oncology data, but what stood out the most was a retrospective study showing how mRNA-based Covid-19 vaccines significantly improved lung and skin cancer patients’ survival when given a few months after immunotherapy. Researchers still haven’t pinpointed why this happened, but the results could have big implications for Moderna and BioNTech’s personalized cancer vaccine programs. Check out below for more of our dispatches from Barcelona. Lykos’ quest for a psychedelics drug approval was thwarted earlier this summer, leading to a significant workforce reduction and CEO Amy Emerson eventually stepping down. Max Bayer, who has been reporting on the Lykos story for weeks at Endpoints, took an in-depth look at the company’s journey dating all the way back to 1986 and how long-simmering tensions and the founder’s personal ambitions collided with growing pains as it tried to secure that approval. The Federal Reserve made its long-awaited interest rate cuts this week, lowering the benchmark rate by a half of a percentage point to 4.75% to 5%. While the cut is likely to pressure investors into getting their cash off the sidelines, industry insiders told Endpoints that they hoped Wednesday’s cut would be the start of a trend. Predicting how the recovery will shake out over the coming months, however, will be difficult. All seven of 23andMe’s independent board directors resigned this week over their differing views with CEO Anne Wojcicki and her attempts to take the health tech startup private. The company went public by merging with Richard Branson’s SPAC in 2021, but it hasn’t been able to persuade investors it can tackle genetic testing and drug development simultaneously. The resignations came the same day 23andMe presented data from two clinical programs at ESMO. And, late Friday, a small genetic testing startup said it was interested in acquiring 23andMe. SPOTLIGHT #ESMO24 R&D PHARMA FINANCING FDA+ MANUFACTURING HEALTH TECH DON’T MISS

疫苗并购高管变更IPO

2024-09-20

·药明康德

诺和诺德16周减重14斤的口服减肥疗法；赛诺菲BTK抑制剂全球监管申请递交在即……

▎药明康德内容团队编辑赛诺菲BTK抑制剂3期临床结果积极，即将递交监管申请赛诺菲（Sanofi）公司今日宣布，在研布鲁顿氏酪氨酸激酶（BTK）抑制剂tolebrutinib在名为HERCULES的3期临床试验中获得积极结果。在非复发型继发进展性多发性硬化症（nrSPMS）患者中，tolebrutinib与安慰剂相比，将患者出现6个月确认残疾进展（CDP）的时间延缓31%（HR=0.69；95% CI，0.55-0.88；p=0.0026）。对次要终点的进一步分析显示，tolebrutinib组确认残疾改善的参与者比例增加了两倍，tolebrutinib组为10%，而安慰剂组为5%（HR=1.88；95% CI，1.10-3.21）。赛诺菲公司表示，这些数据显示，tolebrutinib作为一种可渗透大脑的BTK抑制剂，能够解决多发性硬化症的残疾累积问题。该公司预计将于2024年下半年启动全球监管申请的递交。安全性方面，初步分析显示tolebrutinib治疗组某些不良事件的发生率略有增加。肝酶升高在tolebrutinib组中发生率为4.1%，而安慰剂组为1.6%，这一副作用也在其他用于多发性硬化症的BTK抑制剂中被报道。Tolebrutinib组中有0.5%的参与者出现了肝酶峰值超过正常水平20倍的情况，所有这些情况都发生在治疗的前90天内。除了一个病例外，所有肝酶升高的病例都在没有进一步医疗干预的情况下得到了缓解。在实施修订后的更严格监测方案之前，tolebrutinib组有一名参与者接受了肝移植，并因术后并发症去世。迄今为止，通过更频繁的监测已经减轻了此类严重的肝脏不良事件。研究中的其他死亡病例被研究者评估为与治疗无关，安慰剂组和tolebrutinib组的死亡率均为0.3%。诺和诺德公布新机制减肥疗法2期临床结果诺和诺德（Novo Nordisk）今天公布了在研疗法monlunabant的2a期临床试验的主要结果。Monlunabant是一种小分子口服大麻素受体1（CB1）反向激动剂，之前名为INV-202。2023年8月诺和诺德收购Inversago Pharmaceuticals囊获这一疗法。该试验评估了每日一次服用10 mg、20 mg和50 mg剂量的monlunabant与安慰剂相比，在243名患有肥胖症和代谢综合征的人群中，经过16周治疗后对体重的影响及其安全性。试验数据显示，从基线体重110.1公斤开始，所有剂量的monlunabant均显著减轻了体重，与安慰剂组相比有统计学显著差异。经过16周治疗，服用每日一次10 mg剂量monlunabant的参与者平均体重减少了7.1公斤，而安慰剂组仅减少了0.7公斤。较高剂量的monlunabant仅带来了有限的额外减重效果。在试验中，最常见的不良事件为胃肠道反应，绝大多数为轻度至中度，并且与剂量相关。此外，与安慰剂组相比，服用monlunabant的参与者出现轻度至中度神经精神副作用（主要为焦虑、易怒和睡眠障碍）的频率更高，且与剂量相关。未报告与神经精神副作用相关的严重不良事件。基于该结果，诺和诺德计划启动一项更大规模的2b期肥胖症试验，以进一步研究monlunabant的剂量及其在全球人群中更长期的安全性。这项2b期试验预计将在2025年启动。 George Church博士联合创建，新锐致力于开发下一代干细胞疗法 GC Therapeutics（GCTx）日前宣布正式成立，致力于规模化和解锁下一代细胞疗法。该公司的诱导多能干细胞（iPSC）编程平台TFome由哈佛医学院和Wyss研究所的George Church博士团队发明并开发，融合了合成生物学、基因编辑、细胞工程和机器学习领域的最新进展，以克服细胞疗法开发和规模化的复杂性，并提高广泛疾病领域中的患者可及性。GCTx已完成6500万美元的A轮融资。 iPSC分化为不同细胞类型依赖于各种信号通路，这些通路最终汇集到转录因子（TF）上，TF控制细胞中活跃的基因集合。细胞的命运由特定的TF协同作用决定。新闻稿指出，GCTx已经构建了全球首个完整的人类TF库。TFome能够识别优化的TF组合，将iPSCs分化为几乎任何功能性细胞类型。这使得在无需对细胞类型特定的微环境因素进行优化的情况下，实现单步、只需四天的干细胞分化过程，效率超过90%。通过基于TF的细胞分化编程，TFome旨在改进细胞疗法的效力、效率、质量和成本。这一方法比传统细胞分化方法快高达100倍，生产出潜在“first-in-class”和“best-in-class”的现货型细胞治疗产品。GCTx有望成为iPSC细胞疗法领域首个使用类似制造策略，从一个起点开发多个细胞产品的公司，加速产品开发和时间表。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料： [1] GC Therapeutics Launches to Scale and Unlock the Next Generation of Cell Therapy. Retrieved September 19, 2024, from https://www.businesswire.com/news/home/20240918923504/en/GC-Therapeutics-Launches-to-Scale-and-Unlock-the-Next-Generation-of-Cell-Therapy [2] Tolebrutinib demonstrated a 31% delay in time to onset of confirmed disability progression in non-relapsing secondary progressive multiple sclerosis phase 3 study. Retrieved September 20, 2024, from https://www.sanofi.com/en/media-room/press-releases/2024/2024-09-20-09-30-00-2949552 [3] Monlunabant phase 2a trial in obesity successfully completed. Retrieved September 20, 2024, from https://www.novonordisk.com/content/nncorp/global/en/news-and-media/news-and-ir-materials/news-details.html?id=170501 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

临床3期临床结果临床2期并购