A Multicenter, Randomized, Placebo-Controlled, Multiple-Ascending-Dose Investigation of the Oral Anti-Inflammatory Agent BT051 in Subjects With Moderately to Severely Active Ulcerative Colitis (UC)

This is a randomised, double-blind, placebo-controlled study to assess the safety and tolerability of multiple ascending doses of BT051 in subjects with moderately to severely active ulcerative colitis. Subjects will be randomised using a 3 active:1 placebo ratio to 3 ascending dose cohorts of 8 subjects and will be dosed daily for 28 days. The 3 initial dose levels will be 200 mg, 800 mg and 3200 mg per day. Progression to the next cohort will be based on the safety and tolerability of the previous cohort.

开始日期2021-11-30

申办/合作机构

Adiso Therapeutics, Inc.

NCT05103878 / Completed临床1期

A Phase 1, Randomized, Double-Blind, Single-Ascending-Dose Study Evaluating the Safety, Tolerability, and Pharmacokinetics of BT051 in Healthy Subjects

This is a randomized, double-blind, placebo-controlled study to assess the safety and tolerability of single ascending doses of BT051 in healthy male or female volunteers aged 18 to <50 years. A total of 50 subjects will be randomized to receive a single oral dose of BT051 or matching placebo in a ratio of 4 active:1 placebo in 5 ascending dose cohorts (10 subjects per cohort) at active dose levels of 100mg, 300mg, 700mg, 1500mg or 3500mg. The study Safety Review Committee (SRC) will evaluate if any dose-limiting adverse events (AEs) occurred in a cohort through Day 3, as well as review cumulative safety data for all previous cohorts and any available pharmacokinetic (PK) data before proceeding to dosing in the next cohort.

开始日期2020-06-24

申办/合作机构

Bacainn Therapeutics, Inc.初创企业

100 项与 BT-051 相关的临床结果

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100 项与 BT-051 相关的转化医学

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100 项与 BT-051 相关的专利（医药）

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项与 BT-051 相关的文献（医药）

2023-08-01·FEBS open bio

Development of ADS051, an oral, gut-restricted, small molecule neutrophil modulator for the treatment of neutrophil-mediated inflammatory diseases.

Article

作者: Farquhar, Ronald ; Oleson, Frederick B ; Dolle, Roland E ; Murphy, Christopher K ; McCormick, Beth A ; Dixit, Bharat

Neutrophils are an essential component of the innate immune system; however, uncontrolled neutrophil activity can lead to inflammation and tissue damage in acute and chronic diseases. Despite inclusion of neutrophil presence and activity in clinical evaluations of inflammatory diseases, the neutrophil has been an overlooked therapeutic target. The goal of this program was to design a small molecule regulator of neutrophil trafficking and activity that fulfilled the following criteria: (a) modulates neutrophil epithelial transmigration and activation, (b) lacks systemic exposure, (c) preserves protective host immunity, and (d) is administered orally. The result of this discovery program was ADS051 (also known as BT051), a low permeability, small molecule modulator of neutrophil trafficking and activity via blockade of multidrug resistance protein 2 (MRP2)- and formyl peptide receptor 1 (FPR1)-mediated mechanisms. ADS051, based on a modified scaffold derived from cyclosporine A (CsA), was designed to have reduced affinity for calcineurin with low cell permeability and, thus, a greatly reduced ability to inhibit T-cell function. In cell-based assays, ADS051 did not inhibit cytokine secretion from activated human T cells. Furthermore, in preclinical models, ADS051 showed limited systemic absorption (<1% of total dose) after oral administration, and assessment of ADS051 in human, cell-based systems demonstrated inhibition of neutrophil epithelial transmigration. In addition, preclinical toxicology studies in rats and monkeys receiving daily oral doses of ADS051 for 28 days did not reveal safety risks or ADS051-related toxicity. Our results to date support the clinical development of ADS051 in patients with neutrophil-mediated inflammatory diseases.

项与 BT-051 相关的新闻（医药）

2023-12-08

·今日头条

3亿美元！精准调菌消灭痘痘，新锐完成B轮融资

本期看点 ① Eligo Bioscience获得3亿美元B轮融资 ② Abivax正式登陆纳斯达克 ③ Geneoscopy与Adiso Therapeutics就IBD开展临床合作 ④ Lavie Bio与WinField United Canada达成独家销售协议 ⑤ Quazy Foods获得800万欧元种子轮融资 ⑥ 雀巢计划在巴西投资12亿美元 ① Eligo Bioscience获得3亿美元B轮融资作者：Eligo Bioscience 解读：Richard 来源：Yahoo 发布日期：2023-12-05 ■ 内容要点 12月5日，基因编辑公司Eligo Bioscience宣布获得了3亿美元（约合人民币25.1亿元）的B轮融资。赛诺菲旗下投资机构Sanofi Ventures领衔了此次投资，参与投资的还有Bpifrance、Khosla Ventures 和Seventure Partners等新老投资人。资金将用于加速Eligo Bioscience公司治疗痤疮在研管线EB005临床开发，支持其开展新药临床试验申报等相关活动，以及帮助Eligo Bioscience公司扩大研究方向并拓展至肿瘤治疗领域。 Eligo Bioscience公司专注于开展基因编辑疗法来治疗各类炎症性疾病、肿瘤和感染性疾病。在研管线EB005由Eligo Bioscience公司与GSK公司合作开发而来，能够通过抑制皮肤微生物组中的痤疮丙酸杆菌生长，精准地调节皮肤菌群结构，治疗或缓解中重度痤疮。原文链接： https://finance.yahoo.com/news/eligo-bioscience-raises-30-million-110000107.html?guccounter=1 ② Abivax正式登陆纳斯达克作者：奋斗在企业上市辅导解读：Richard 来源：奋斗在企业上市辅导官微发布日期：2023-12-06 ■ 内容要点近日，法国医药公司Abivax正式在美国纳斯达克上市。 Abivax公司此次发行价为11.60美元，共计发行2032.55万股，募资总额为2.36亿美元（约合人民币16.90亿元）。以此发行价计算，Abivax公司市值达到7.29亿美元。 Abivax公司总部位于法国巴黎，专注于开发治疗胃肠道疾病药物，其口服小分子药物Obefazimod正处于3期临床试验中，用于治疗中重度的溃疡性结肠炎。并且，Abivax公司还计划启动该药物治疗克罗恩病的2期临床试验。招股书显示，Abivax公司在2021年、2022年营收分别为1196.10万欧元、458.30万欧元，相应净利润分别为-4245.20万欧元、-6074.00万欧元。在上市前的股东架构中，TCG Crossover持股为11.64%，有9.98%投票权；Truffle Capital持股为10.99%，有17.79%投票权。原文链接： https://mp.weixin.qq.com/s/DANCLT8pknyjVFfBIAh4_Q ③ Geneoscopy与Adiso Therapeutics就IBD开展临床合作作者：Geneoscopy 解读：Richard 来源：Businesswire 发布日期：2023-12-05 ■ 内容要点 12月5日，Geneoscopy公司与Adiso Therapeutics公司双方共同宣布，将在炎症性肠病（IBD）领域达成战略合作。未来，Geneoscopy公司旗下的RNA诊断技术将应用到Adiso Therapeutics公司开展的ADS051治疗IBD 2期临床试验中去。 Geneoscopy公司的RNA诊断技术通过检测粪便样本中的RNA水平，来判断结肠粘膜愈合情况，以及预测患者对治疗药物的响应程度。这一非侵入性的检测方案能够极大提高IBD临床研究效率，比传统的粪便钙卫蛋白检测方案更加精确。候选药物ADS051是一款肠道限制性的口服小分子药物，能够调节中性粒细胞的激活和运输。2期临床试验将在全球开展并计划在2024年开启患者招募。原文链接： https://www.businesswire.com/news/home/20231205408799/en/Geneoscopy-and-Adiso-Therapeutics-Announce-Strategic-Collaboration-to-Investigate-New-Therapeutic-Options-for-Patients-with-Inflammatory-Bowel-Disease ④ Lavie Bio与WinField United Canada达成独家销售协议作者：Lavie Bio 解读：Richard 来源：AgriBusiness GLOBLE 发布日期：2023-12-05 ■ 内容要点近日，农业生物科技公司Lavie Bio宣布与WinField United Canada公司，就其种子包衣产品Yalos达成独家分销协议。此次合作旨在确保2024年Lavie Bio公司在加拿大实现销售收入稳定增长，特别是在加拿大重点农产区，包括阿尔伯塔省、曼尼托巴省和萨斯喀彻温省。 Lavie Bio公司专注于采用微生物组技术和人工智能技术开发生防菌和促生菌产品。Yalos可用于春小麦、硬粒小麦和大麦种子包衣。在一项第三方开展的田间试验中，Yalos可使春小麦、硬粒小麦和大麦每亩增产17-23公斤。根据当前市场价格，对于种植户来说使用Yalos投入产出比可达4:1。此外，Yalos使用方法简单方便，农户使用无须改变现有制种工序。WinField United Canada公司的加入，能有效带动该产品在北美地区的销售。原文链接： https://www.agribusinessglobal.com/agrochemicals/seeds-traits/lavie-bio-forms-distribution-agreement-with-winfield-united-canada-for-its-bio-inoculant-seed-treatment-yalos/ ⑤ Quazy Foods获得800万欧元种子轮融资作者：synbio新农食解读：Richard 来源：FoodTalks 发布日期：2023-12-06 ■ 内容要点近日，德国生物技术初创公司Quazy Foods宣布获得了800万欧元（约合人民币6163.2万元）种子轮融资。ProVeg International、Antler和Sprout & About Ventures领衔了本次投资，其他天使投资人共同参与。资金将用以扩大Quazy Foods公司微藻培养技术，开发功能性植物基食品和其它应用成分。并且，资金还将用推动Quazy Foods公司扩大试点生产，助力其开发首个微生物藻类成分样品。 Quazy Foods公司创立于2021年，专注于培育微藻菌株用于食品工业和其它领域。该公司通过利用生物质发酵和专有微生物藻类菌株，成功生产出高质量蛋白质和功能性复合成分。未来，Quazy Foods公司将积极打造世界一流产品，找到养活全球人口的创新方式，以应对未来几十年人类面临的挑战。原文链接： https://www.foodtalks.cn/news/50101 ⑥ 雀巢计划在巴西投资12亿美元作者：解读：617 来源：FoodTalks 发布日期：2023-12-07 ■ 内容要点 12月7日，雀巢宣布计划在巴西投资60亿雷亚尔（约合美元12亿元）用于食品和饮料生产。上述款项计划于2023年至2025年期间通过一系列项目陆续支付。本次投资资金将主要用于促进业务增长、整合行业新技术、扩大制造设施、转变产品组合，目的是推进雀巢在巴西的巧克力、咖啡、宠物护理和营养健康版块的可持续发展。据报道，这项庞大的投资计划将在巴西南部兴建新的Purina工厂，并在其Cacapava工厂启动全新的KitKat生产线。雀巢方表示，到2024年，公司将继续投资于生产力、创新和运营的可持续性，针对新技术和生产线进行战略布局，加速ESG、创新和数字化转型，支持雀巢巴西业务的持续增长。原文链接： https://www.foodtalks.cn/flash/18257

微生物疗法临床2期临床3期

2023-12-05

·BioSpace

Geneoscopy and Adiso Therapeutics Announce Strategic Collaboration to Investigate New Therapeutic Options for Patients with Inflammatory Bowel Disease

Adiso to Utilize Noninvasive RNA Technology Developed by Geneoscopy as a Precision Immunology Measure in ADS051 Phase 2 IBD Trial ST. LOUIS, Mo. & CONCORD, Mass.--(BUSINESS WIRE)-- Geneoscopy, Inc., a life sciences company focused on developing noninvasive diagnostic and precision medicine tools for gastrointestinal health, and Adiso Therapeutics, Inc., a clinical-stage biotechnology company advancing novel medicines to treat inflammatory diseases, today announced a strategic collaboration to further treatment options available for patients with inflammatory bowel disease (IBD). The agreement allows Geneoscopy’s patented RNA biomarker technology to be used in conjunction with ADS051, a first-in-class, oral, gut-restricted, small molecule modulator of neutrophil trafficking and activation being developed by Adiso for the treatment of IBD. This press release features multimedia. View the full release here: “Utilizing our RNA biomarker platform to assess stool samples from IBD patients and categorize response to treatment with ADS051 is an exciting opportunity,” said Andrew Barnell, CEO of Geneoscopy. “Our technology shows significant promise to enhance IBD clinical trials by identifying patients likely to respond to a particular therapeutic and, through noninvasive methods, determining therapeutic effectiveness through the measurement of RNA expression profiles which signal mucosal healing in the colon.” “The addition of noninvasive stool-based RNA technology from Geneoscopy within our Phase 2 trial of ADS051 is a perfect complement to our neutrophil-targeted, gut-restricted, non-systemic therapy,” said Scott Megaffin, CEO of Adiso. “Employing precision immunology within our trial holds the potential to significantly advance our ability to provide IBD patients with a novel and highly differentiated treatment option that targets their specific needs.” As part of the Adiso ADS051 Phase 2 trial in patients with ulcerative colitis, Geneoscopy will prospectively assess stool-based transcriptome changes in patients treated with ADS051 to identify immunological markers that may predict individual response. Beyond its utility in identifying likely responders to therapy, preliminary research has also indicated that Geneoscopy’s RNA biomarker technology could provide a noninvasive method of determining clinical remission that is more accurate than traditional diagnostics such as fecal calprotectin. In subsequent clinical trials, Adiso will leverage the Geneoscopy biomarker panel to employ a precision immunology approach that uses stool-derived eukaryotic RNA (seRNA) to assess mucosal healing and predict patient response to therapy. This collaboration comes at a critical time for the estimated 3 million Americans living with IBD. Despite an increased focus on improving treatment and patient care, effective tools are lacking to inform therapy selection, and significant opportunities exist to advance and refine methods for disease monitoring. The Adiso ADS051 Phase 2 clinical study will be conducted globally and begin recruiting in 2024. About Geneoscopy, Inc. Geneoscopy Inc. is a life sciences company focused on developing diagnostic tests for gastrointestinal health. Leveraging its proprietary, patented stool-derived eukaryotic RNA (seRNA) biomarker platform, Geneoscopy’s mission is to empower patients and providers to transform gastrointestinal health through innovative diagnostics. Beyond colorectal cancer screening, Geneoscopy is developing diagnostic tests for treatment selection and therapy monitoring in other disease areas in partnership with leading universities and biopharmaceutical companies. For more information, visit and follow the company on LinkedIn. About Adiso Adiso is a clinical-stage biopharmaceutical company dedicated to improving the health of patients suffering from debilitating inflammatory diseases. This dedication is epitomized by our lead clinical candidates, which exemplify 'healthruption' (disruption in healthcare and drug development) with novel mechanisms of action and a distinct approach to the treatment of inflammatory diseases. ADS051, an oral, gut-restricted modulator of neutrophil trafficking and activation via inhibition of MRP2 and FPR1 for the treatment of ulcerative colitis; and ADS032, a dual NLRP1/NLRP3 inflammasome inhibitor initially being developed for inflammatory diseases of the lung. Adiso has built development programs upon a rich history of institutional and academic collaboration, including, the University of Massachusetts Chan Medical School, the Hudson Institute of Medical Sciences Centre for Innate Immunity and Infectious Diseases in Australia, the University of Edinburgh Centre for Inflammation Research and the University College Cork, Ireland, the APC Microbiome Institute. For more information, please visit or our LinkedIn page. About ADS051 ADS051 is a novel first-in-class, oral, gut-restricted, small molecule modulator of neutrophil trafficking and activation via MRP2 and FPR1 receptor inhibition. ADS051 is a locally acting targeted therapy that addresses the underlying pathophysiology of disease while sparing the peripheral role of neutrophil function. Unlike currently available therapies, ADS051 addresses neutrophil-mediated tissue damage, a hallmark of UC. UC is a form of IBD that causes inflammation and ulcers in the colon. Neutrophils are a type of white blood cell that plays a key role in the body's immune response. In UC, neutrophils are recruited to the colon in large numbers, where they release a variety of inflammatory mediators that cause tissue damage. ADS051 gut restriction, safety and tolerability after oral dosing has been demonstrated in a healthy subject Phase 1a clinical study. In a healthy volunteer Single Ascending Dose study and in a moderate to severe UC patient Phase 1b Multiple Ascending Dose study, oral doses of ADS051 were safe and well-tolerated and largely restricted to the gut with limited systemic exposure while demonstrating encouraging signals of pharmacologic activity and clinical benefit.

临床1期临床2期

2023-10-30

·药明康德

治疗阿尔茨海默病的反义寡核苷酸疗法、胰腺癌疾病控制率达85%的溶瘤病毒联合疗法... | 一周盘点

▎药明康德内容团队编辑本期看点1. BioNTech潜在“first-in-class”的CAR-T细胞疗法BNT211治疗CLDN6阳性、难治性/复发性实体肿瘤，接受2级剂量的患者的疾病控制率（DCR）达95%。2. 治疗阿尔茨海默病的反义寡核苷酸（ASO）疗法BIIB080展现积极早期临床结果，2期临床试验已经展开。3. 一线治疗胰腺导管腺癌（PDAC）的溶瘤病毒联合疗法公布积极早期结果，客观缓解率（ORR）达62%，DCR达85%，中位总生存期（OS）比历史结果高出至少25%。4. 百吉生物BST02的IND申请获美国FDA许可，成为首个进入临床阶段用于治疗所有类型肝癌的肿瘤浸润淋巴细胞（TIL）疗法。5. Mustang Bio公司开发的溶瘤病毒/CAR-T组合疗法将于2024年启动针对复发性胶质母细胞瘤和高级别星形细胞瘤的1期临床试验。药明康德内容团队整理BNT211：公布1/2期临床试验的新数据 BioNTech公布其新一代CAR-T疗法BNT211用于治疗CLDN6阳性、难治性/复发性实体肿瘤的1/2期临床试验的积极数据。BNT211是一款潜在“first-in-class”的治疗策略，它将靶向CLDN6抗原的CAR-T疗法与表达CLDN6抗原的CARVac相结合。首先给患者输注低剂量的CLDN6靶向CAR-T疗法。然后给患者注射编码CLDN6的mRNA疫苗CARVac。这种疫苗会在抗原呈递细胞表面表达CLDN6，刺激体内CAR-T细胞的扩增。此次公布的结果显示，CARVac的应用增加了过继转移的自体CAR-T细胞的持久性。38例可进行疗效评估的患者的ORR为45%，DCR为74%。在27名接受2级剂量CAR-T细胞治疗（不论是否使用CARVac）的患者中，13名出现部分缓解（PR），ORR为59%，DCR为95%。此外，在同一队列中，接受CARVac治疗的患者的CAR-T细胞存活时间延长。安全性方面，观察到不良事件呈剂量依赖性增加。在44名安全性可评估的患者中，23名出现了细胞因子释放综合征。大多数情况下，这些不良反应为1级和2级，1名患者为3级，1名患者为4级。两名患者的神经毒性较轻，且有自限性。BIIB080（IONIS-MAPT）：公布1b期临床试验的新数据渤健（Biogen）与Ionis Pharmaceuticals共同宣布其合作开发的在研ASO疗法BIIB080（IONIS-MAPT）在治疗阿尔茨海默病的临床1b试验中展现出积极结果。阿尔茨海默病的主要神经病理学特征是存在细胞外的β-淀粉样蛋白（Aβ）和细胞内过度磷酸化的tau蛋白沉积。病理性tau蛋白的积累已被证明可造成神经元损伤和死亡。BIIB080是Ionis公司发现的一种在研ASO疗法，旨在靶向微管相关蛋白tau（MAPT）的mRNA以阻止tau蛋白的产生。此次公布的结果显示，接受该疗法的患者展现认知下降改善的趋势。根据新闻稿，这是首款显示能够降低tau蛋白聚集并展现对患者临床结局有利趋势的tau蛋白靶向药物。根据此积极结果，渤健已启动相关CELIA临床2期试验，以进一步研究该药物的疗效与安全性，目前病患正在招募中。Pelareorep：公布1/2期临床试验数据 Oncolytics Biotech公布其开发的溶瘤病毒pelareorep与PD-L1抑制剂和化疗的组合疗法，作为一线治疗晚期/转移性PDAC患者的1/2期临床试验积极数据。Pelareorep是一种从常见于环境水域的呼肠孤病毒（reovirus）中分离出来的天然非致病性双链RNA（dsRNA）病毒，有潜力成为治疗实体瘤和血液系统恶性肿瘤的“first-in-class”疗法。研究结果显示，该联合疗法的ORR达62%，DCR为85%，中位缓解持续时间为5.7个月，中位无进展生存期（PFS）为7.2个月，12个月生存率为46%，中位OS为10.6个月，比历史结果高出至少25%。T细胞扩增数据与肿瘤缓解相关，提供了重要的概念验证。安全性方面，该药物组合耐受性良好，未发现安全问题。BST02：IND申请获得FDA许可 BST02是百吉生物开发的一种新型免疫细胞疗法，通过扩增来自患者自身的肿瘤浸润淋巴细胞制备，旨在治疗包括肝细胞癌和胆管癌在内的肝癌。相比于传统的TIL疗法，BST02的临床使用不需要高水平的IL-2，从而减小了安全风险。此外，该疗法还可以通过冷冻保存解决生产地点需在临床中心附近的距离限制。BST02已在探索性的临床试验中初步证明了其安全性和有效性。新闻稿指出，BST02是首个进入临床阶段、用于治疗所有类型肝癌的TIL疗法。MB-109：IND申请获得FDA许可 MB-109是Mustang Bio公司开发的溶瘤病毒/CAR-T组合疗法，该组合疗法用于治疗复发性胶质母细胞瘤（GBM）和高级别星形细胞瘤的IND申请于近日获得FDA许可，预计于2024年启动1期临床试验。MB-101是一种靶向IL13Rα2的CAR-T细胞疗法，IL13Rα2是一种局限在GBM中表达的受体，在大多数GBM肿瘤中大量表达。MB-108属于第二代减毒1型单纯疱疹病毒（HSV-1）溶瘤病毒，能够优先在肿瘤细胞中复制，从而杀死受感染的肿瘤细胞并利用肿瘤细胞增殖性强的特点不断生产新病毒。MB-108还可以诱导促炎信号和趋化性，从而改善CAR-T细胞对肿瘤的浸润。MB-109是上述两种药物联合用药的治疗方案。该组合旨在利用MB-108使冷肿瘤“变热”，从而提高MB-101细胞疗法的疗效。首先注射MB-108溶瘤病毒以感染肿瘤细胞，进而通过募集内源性CD8阳性效应T细胞来重塑肿瘤微环境（TME），发炎的TME允许注射到肿瘤内部和周围的MB-101 CAR-T细胞更好地浸润到整个肿瘤中，激活并杀死肿瘤细胞。在临床前研究中MB-109组合可以在低剂量安全使用并且导致肿瘤缩小。NRTX-1001：公布1/2期临床试验的新数据Neurona Therapeutics公司公布了其候选再生细胞疗法NRTX-1001的1/2期临床试验的长期数据。NRTX-1001是一种来源于人类多能干细胞的再生神经细胞治疗候选药物，用于治疗癫痫。NRTX-1001包括分泌抑制性神经递质γ-氨基丁酸（GABA）的中间神经元。作为一次性给药疗法，该药物能够提供局部长期GABA信号通路抑制，以重新平衡和修复引起癫痫以及神经系统其他疾病过度兴奋的神经网络。接受NRTX-1001一次性治疗后患者需在第一年接受免疫抑制，目的是促进NRTX-1001的整合和长期持久性。迄今为止，正在进行的临床试验尚未报告细胞疗法或免疫抑制方案引起的严重不良反应。临床试验中的首位受试者已按方案完成了免疫抑制治疗，目前已停止免疫抑制治疗，并在接受NRTX-1001治疗16个月后癫痫发作稳定减少>95%。临床试验中的第2名受试者在接受NRTX-1001治疗12个月后癫痫发作也减少了95%以上，并且在过去6个月中一直没有癫痫发作。HPN328：公布1/2期临床试验的中期数据Harpoon Therapeutics公司公布了评估HPN328单药治疗小细胞肺癌（SCLC）和其他神经内分泌肿瘤的最新中期数据。HPN328靶向DLL3，源自Harpoon专有的三特异性T细胞激活结构（TriTAC）平台，旨在招募患者自身的免疫细胞杀死肿瘤细胞。此次公布的结果显示，在使用1 mg起始剂量治疗的所有肿瘤类型和患者组别中，经确认的缓解率为35%（11/31），其中包括3例确认的完全缓解（CR）。SCLC患者的经确认的缓解率为32%（6/19），其中包括1例确认的CR。其他神经内分泌肿瘤患者的经确认的缓解率为42%（5/12），包括2例确认的CR。HPN328耐受性普遍良好，最常见的治疗相关不良事件为细胞因子释放综合征，主要为1级或2级。ELU001：公布1/2期临床试验数据Elucida Oncology公司公布了其C’Dot偶联药物ELU001在过度表达叶酸受体α（FRα）的实体肿瘤患者中的1/2期临床数据。Elucida Oncology致力于利用其名为C’Dot的递送技术精准靶向和肿瘤内递送治疗药物。这一递送技术可以精准递送多种类型的治疗性药物，并且由于它的个体很小，能够在肾脏被有效清除。此次公布的结果显示，ELU001在所有FRα表达水平≥25%的癌症患者中均观察到抗肿瘤活性，且耐受性良好。FRα表达水平≥25%肿瘤细胞的患者的DCR为89%（17/19），其中有21%的患者治疗时间至少长达9个月。此外，一名FRα表达<25%肿瘤细胞的患者也获得了PR。Luveltamab tazevibulin（luvelta）：公布1期临床试验数据Sutro Biopharma公司公布了其靶向FRα的抗体偶联药物（ADC）luveltamab tazevibulin用于治疗子宫内膜癌的1期临床试验数据。在表达FRα的肿瘤比例评分（TPS）＞25%的晚期子宫内膜癌患者中，经确认的PR率为29%，DCR为86%。该候选疗法的安全性与之前对铂类耐药卵巢癌患者的研究数据一致。BDC-1001：公布1期临床试验数据Bolt Biotherapeutics公司公布了其抗体-免疫刺激偶联物（ISAC）BDC-1001针对表达HER2的实体瘤开展的剂量递增研究的积极结果。此次公布的结果显示，BDC-1001无论是作为单一疗法还是与纳武利尤单抗联合使用均具有良好的耐受性。自2023年6月公布数据以来，1名患者由PR转为CR，另外有2名患者保持长期的疾病稳定，3名患者的治疗时间已超过1年。TransCon IL-2 β⁄γ：公布1/2期临床试验数据Ascendis Pharma公司公布了其在研长效IL-2 β/γ选择性类似物TransCon IL-2 β/γ作为单药或与帕博利珠单抗联用的1/2期临床试验数据。结果显示，该疗法作为单药或与免疫检查点抑制剂联用，已显示出临床缓解，接受联合疗法的3名SCLC患者中有两名患者出现缓解。此外，该疗法单药使用或联合用药的耐受性良好，对调节性T细胞和嗜酸性粒细胞无明显影响。HMBD-001：公布1期临床试验数据Hummingbird Bioscience公司公布了其开发的靶向HER3的IgG1抗体HMBD-001用于治疗对获批疗法耐药的实体肿瘤患者的积极早期数据。该候选疗法有可能通过靶向位于HER3与HER2或EGFR形成异二聚体的连接处的关键表位，完全阻断配体依赖性和非依赖性HER3激活和致癌信号。在临床前模型中，该公司观察到HMBD-001与现有的HER3抗体相比具有更好的亲和力和更有效的肿瘤生长抑制作用。此次公布的数据显示，HMBD-001安全且耐受性良好，没有剂量限制性毒性反应，也没有严重的治疗相关不良事件。患者的DCR为43%（9/21），其中一名患者在接受两个周期的HMBD-001单药治疗后获得PR，最佳总缓解为肿瘤缩小51%。Ompenaclid（RGX-202）：公布1b/2期临床试验数据Inspirna公司公布了其潜在“first-in-class”的口服小分子SLC6a8抑制剂ompenaclid（RGX-202）联合FOLFIRI和bevacizumab（BEV）治疗RAS突变（RASm）晚期或转移性结直肠癌（CRC）患者的1b/2期研究数据。结果显示，截至2023年9月18日的数据，30名可评估的RASm患者的ORR为37%，包括8例确认的PR和3例未确认的PR。在意向治疗人群中，所有41名患者的中位PFS为10.2个月，中位OS为19.1个月。9名可评估的RAS野生型患者的ORR为22%，包括1例确认的PR和1例未确认的PR，中位PFS为7.5个月，中位OS为14.5个月。GB1211：公布1b/2a期临床试验数据Galecto公司公布了其口服、高亲和力的小分子碳水化合物基半乳糖凝集素-3（Gal-3）抑制剂GB1211联用PD-L1抑制剂atezolizumab一线治疗转移性/晚期非小细胞肺癌（NSCLC）患者的积极早期数据。结果显示，Gal-3抑制剂与免疫检查点抑制剂联用可以增强免疫检查点抑制剂的效果。该联合疗法的耐受性良好，5名晚期NSCLC患者接受联合治疗至少三周后，其中3人达到PR。ORIC-114：公布1期临床试验数据ORIC Pharmaceuticals公司公布了其候选疗法ORIC-114治疗EGFR或HER2 20号外显子突变的NSCLC患者的1期临床试验的初步数据。ORIC-114是一种高选择性、脑渗透性、口服生物利用度高的不可逆抑制剂，可选择性地靶向EGFR和HER2，对20号外显子插入突变有很强的抑制作用。此次公布的结果显示，在多个剂量水平下均观察到ORIC-114的中枢神经系统（CNS）活性，其中有1名携带未经治疗的脑转移灶的患者获得了中枢神经系统CR。在多个剂量水平下均观察到先前接受过大量治疗的NSCLC患者获得了全身性的缓解，其中81%的患者曾接受过EGFR 20号外显子的靶向药物治疗，86%的患者基线时有CNS转移灶。在潜在的2期推荐剂量下，3名曾接受过amivantamab治疗的EGFR 20号外显子突变患者中有2人观察到缓解（ORR为67%），包括1例确认的CR。在多个剂量水平下均观察到HER2 20号外显子突变患者有缓解，1名患者达到了PR，并且所有目标病灶实现了100%的消退。此外，ORIC-114的安全性良好，主要存在1级和2级治疗相关不良事件。Masofaniten（EPI-7386）：公布1/2期临床试验的新数据ESSA Pharma公布了其第二代抗雄激素药物EPI-7386与恩杂鲁胺（enzalutamide）联合治疗转移性去势抵抗性前列腺癌（mCRPC）患者的1/2期临床试验的最新数据。EPI-7386是一种高选择性的雄激素受体N-末端结构域的口服小分子抑制剂。研究结果显示，该组合疗法的耐受性良好，患者的前列腺特异性抗原（PSA）快速、深度且持久地降低。在所有队列中，69%患者的PSA减少≥90%（PSA90），63%患者在90天内达到PSA90，56%患者达到PSA<0.2 mg/mL的水平。RMC-6236：公布1/1b期临床试验结果Revolution Medicines公司公布其RAS MULTI（ON）抑制剂RMC-6236在既往接受过多种治疗、携带KRAS G12X突变（包括常见的KRAS G12D和G12V突变）的NSCLC和PDAC患者中的抗肿瘤和安全性数据。结果显示，RMC-6236显示出初步的临床活性证据和可接受的安全性和耐受性。在40例疗效可评估的NSCLC患者中，ORR为38%，DCR为85%，其中1例患者获得CR，14例患者获得PR。在46例可进行疗效评估的PDAC患者中，ORR为20%，DCR为87%，其中9例患者获得了PR的最佳缓解（4例为未经确认的PR）。已确认的客观缓解包括携带KRAS G12D、G12V或G12R的肿瘤，所有KRAS突变（包括G12A和G12S）的疾病都得到了控制。ADS051：公布1b期临床试验的新数据Adiso Therapeutics公司公布了其口服中性粒细胞运输和激活小分子调节剂ADS051治疗中度至重度活动性溃疡性结肠炎患者的新早期临床数据。ADS051是一种作用局限于肠道、全身暴露有限的疗法，能够解决中性粒细胞介导的组织损伤——这是溃疡性结肠炎（UC）的一种病理学标志。此次公布的结果显示，ADS051安全且耐受性良好，22%的患者在服药仅28天后就获得了临床缓解。SBI-100：公布1期临床试验的新数据Skye Bioscience公司公布了其潜在“first-in-class”的CB1激动剂SBI-100用于治疗青光眼和眼压增高患者的1期临床试验的新数据。SBI-100眼用乳剂（OE）采用新型分子结构和纳米乳剂配方，使其中的有效成分CB1R激动剂可更好地穿透眼内组织，实现有效的局部给药，对降低眼内压具有良好的疗效。此次公布的数据显示，SBI-100 OE的耐受性良好，充血发生率低（8.4%）。基线眼压较高（定义为≥17 mm汞柱）的健康受试者用药后的眼压平均降低23%。大家都在看作为药明康德旗下专注于细胞和基因疗法的CTDMO，药明生基致力于加速和变革基因和细胞治疗及其他高端治疗的开发、测试、生产和商业化。药明生基能够助力全球客户将更多创新疗法早日推向市场，造福病患。如您有相关业务需求，欢迎点击下方图片填写具体信息。▲如您有任何业务需求，请长按扫描上方二维码，或点击文末“阅读原文/Read more”，即可访问业务对接平台，填写业务需求信息▲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（可上下滑动查看）[1] Adiso Therapeutics Announces Positive Topline Data from Phase 1b Study of ADS051 for the Treatment of Moderate-to-Severe Ulcerative Colitis. Retrieved October 23, 2023, from https://www.prnewswire.com/news-releases/adiso-therapeutics-announces-positive-topline-data-from-phase-1b-study-of-ads051-for-the-treatment-of-moderate-to-severe-ulcerative-colitis-301963581.html[2] Sutro Biopharma Announces Presentation of Data for Luveltamab Tazevibulin (luvelta) from the Phase 1 Dose-Expansion Study in Endometrial Cancers at ESMO 2023. Retrieved October 23, 2023, from https://www.globenewswire.com/news-release/2023/10/22/2764322/0/en/Sutro-Biopharma-Announces-Presentation-of-Data-for-Luveltamab-Tazevibulin-luvelta-from-the-Phase-1-Dose-Expansion-Study-in-Endometrial-Cancers-at-ESMO-2023.html[3] Revolution Medicines Presents Promising Clinical Activity and Safety Data from Phase 1/1b Trial of RMC-6236. Retrieved October 23, 2023, from https://www.globenewswire.com/news-release/2023/10/22/2764319/0/en/Revolution-Medicines-Presents-Promising-Clinical-Activity-and-Safety-Data-from-Phase-1-1b-Trial-of-RMC-6236.html[4] Harpoon Therapeutics Announces Updated Interim Tolerability and Response Data from Phase 1/2 Clinical Trial of T Cell Engager HPN328 at ESMO Congress 2023. Retrieved October 23, 2023, from https://www.globenewswire.com/news-release/2023/10/21/2764299/0/en/Harpoon-Therapeutics-Announces-Updated-Interim-Tolerability-and-Response-Data-from-Phase-1-2-Clinical-Trial-of-T-Cell-Engager-HPN328-at-ESMO-Congress-2023.html[5] Initial Phase 1 Dose Escalation Data of ORIC-114 in Patients with EGFR and HER2 Exon 20 Mutations Demonstrates Potential Best-In-Class Profile. Retrieved October 23, 2023, from https://www.globenewswire.com/news-release/2023/10/21/2764289/0/en/Initial-Phase-1-Dose-Escalation-Data-of-ORIC-114-in-Patients-with-EGFR-and-HER2-Exon-20-Mutations-Demonstrates-Potential-Best-In-Class-Profile.html[6] RIBOSCIENCE ESMO PRESENTATION HIGHLIGHTS SAFETY, PK, PD AND EFFICACY DATA FROM THE FIRST 19 PATIENTS OF THE ENPP1 INHIBITOR RBS2418 PHASE 1 STUDY. Retrieved October 23, 2023, from https://www.prnewswire.com/news-releases/riboscience-esmo-presentation-highlights-safety-pk-pd-and-efficacy-data-from-the-first-19-patients-of-the-enpp1-inhibitor-rbs2418-phase-1-study-301962730.html[7] Neurona Therapeutics Completes Clinical Enrollment of First Cohort and Updates on Long-lasting Impact in First Patients Treated in Ongoing Phase I/II Trial of NRTX-1001 Cell Therapy for Drug-resistant Focal Epilepsy. Retrieved October 23, 2023, from https://www.neuronatherapeutics.com/news/press-releases/101923/[8] Hummingbird Bioscience Announces Positive Phase I Clinical Data for HMBD-001 Monotherapy Trial at the European Society for Medical Oncology Congress 2023. Retrieved October 23, 2023, from https://www.prnewswire.com/news-releases/hummingbird-bioscience-announces-positive-phase-i-clinical-data-for-hmbd-001-monotherapy-trial-at-the-european-society-for-medical-oncology-congress-2023-301964124.html[9] Verve Therapeutics Announces Clearance of Investigational New Drug Application by the U.S. FDA for VERVE-101 in Patients with Heterozygous Familial Hypercholesterolemia. Retrieved October 23, 2023, from https://www.globenewswire.com/news-release/2023/10/23/2764538/0/en/Verve-Therapeutics-Announces-Clearance-of-Investigational-New-Drug-Application-by-the-U-S-FDA-for-VERVE-101-in-Patients-with-Heterozygous-Familial-Hypercholesterolemia.html[10] Oncolytics Presents Positive Updated Pancreatic Cancer Data from GOBLET Phase 1/2 Study at ESMO. Retrieved October 23, 2023, from https://www.prnewswire.com/news-releases/oncolytics-presents-positive-updated-pancreatic-cancer-data-from-goblet-phase-12-study-at-esmo-301964264.html[11] Galecto Presents Updated Clinical Data at ESMO Congress 2023 and Provides Update on Phase 1b/2a GALLANT-1 Trial. Retrieved October 23, 2023, from https://www.globenewswire.com/news-release/2023/10/23/2764667/0/en/Galecto-Presents-Updated-Clinical-Data-at-ESMO-Congress-2023-and-Provides-Update-on-Phase-1b-2a-GALLANT-1-Trial.html[12] Elucida Oncology Presents Encouraging Clinical Data from Phase 1/2 Study of ELU001 in Solid Tumors Overexpressing Folate Receptor Alpha at ESMO Congress 2023. Retrieved October 23, 2023, from https://www.globenewswire.com/news-release/2023/10/23/2764660/0/en/Elucida-Oncology-Presents-Encouraging-Clinical-Data-from-Phase-1-2-Study-of-ELU001-in-Solid-Tumors-Overexpressing-Folate-Receptor-Alpha-at-ESMO-Congress-2023.html[13] Inspirna Announces Clinical Data from Phase 1b/2 Study of Ompenaclid (RGX-202) in Advanced Colorectal Cancer at ESMO Congress 2023. Retrieved October 23, 2023, from https://www.businesswire.com/news/home/20231023708620/en[14] ImmuneOncia Announces Phase 1 Results of CD47 Antibody at ESMO 2023. Retrieved October 23, 2023, from https://www.businesswire.com/news/home/20231023637830/en[15] Bolt Biotherapeutics Presents Updated Clinical Data from Phase 1 Dose-Escalation Trial of BDC-1001 as Monotherapy and in Combination with Nivolumab in HER2-Expressing Tumors at ESMO 2023 Congress. Retrieved October 23, 2023, from https://www.globenewswire.com/news-release/2023/10/23/2764894/0/en/Bolt-Biotherapeutics-Presents-Updated-Clinical-Data-from-Phase-1-Dose-Escalation-Trial-of-BDC-1001-as-Monotherapy-and-in-Combination-with-Nivolumab-in-HER2-Expressing-Tumors-at-ESM.html[16] BioNTech Presents Positive Phase 1/2 Data Update for CAR-T Cell Therapy Candidate BNT211 in Advanced Solid Tumors at ESMO Congress 2023. Retrieved October 23, 2023, from https://www.globenewswire.com/news-release/2023/10/23/2764948/0/en/BioNTech-Presents-Positive-Phase-1-2-Data-Update-for-CAR-T-Cell-Therapy-Candidate-BNT211-in-Advanced-Solid-Tumors-at-ESMO-Congress-2023.html[17] NeuroTherapia Presents Clinical Data from Phase 1b Clinical Trial of NTRX-07 for the Treatment of Alzheimer’s Disease. Retrieved October 24, 2023, from https://www.globenewswire.com/news-release/2023/10/24/2765493/0/en/NeuroTherapia-Presents-Clinical-Data-from-Phase-1b-Clinical-Trial-of-NTRX-07-for-the-Treatment-of-Alzheimer-s-Disease.html[18] Upstream Bio Announces Positive Phase 1b Interim Data in Asthma and Company Progress Toward Phase 2. Retrieved October 24, 2023, from https://www.businesswire.com/news/home/20231024743233/en[19] KaliVir Immunotherapeutics Announces FDA Clearance of Investigational New Drug (IND) for Systemic Oncolytic Virus ASP1012 for Phase I Clinical Trials for Locally Advanced or Metastatic Solid Tumors. Retrieved October 24, 2023, from https://www.businesswire.com/news/home/20231024827071/en[20] TransCode Therapeutics Announces Preliminary Clinical Results in First Patient in Phase 0 Clinical Study with Lead Therapeutic Candidate, TTX-MC138. Retrieved October 24, 2023, from https://www.globenewswire.com/news-release/2023/10/24/2765543/0/en/TransCode-Therapeutics-Announces-Preliminary-Clinical-Results-in-First-Patient-in-Phase-0-Clinical-Study-with-Lead-Therapeutic-Candidate-TTX-MC138.html[21] Ankyra Therapeutics Receives FDA IND and Health Canada CTA Approval to Advance First-In-Class Selective Anchored Immunotherapy Agent, ANK-101, into Human Trials in Solid Tumors. Retrieved October 24, 2023, from https://www.businesswire.com/news/home/20231024848390/en[22] Therini Bio Announces Interim Results from the Phase 1 Trial of THN391 for the Potential Treatment of Dementia. Retrieved October 24, 2023, from https://www.globenewswire.com/news-release/2023/10/24/2765713/0/en/Therini-Bio-Announces-Interim-Results-from-the-Phase-1-Trial-of-THN391-for-the-Potential-Treatment-of-Dementia.html[23] Genascence to Present Full Data from Phase 1 Clinical Trial on GNSC-001, Company's Lead Program in Osteoarthritis, at European Society of Gene & Cell Therapy 30th Annual Congress. Retrieved October 24, 2023, from https://www.prnewswire.com/news-releases/genascence-to-present-full-data-from-phase-1-clinical-trial-on-gnsc-001-companys-lead-program-in-osteoarthritis-at-european-society-of-gene--cell-therapy-30th-annual-congress-301963549.html[24] Excision BioTherapeutics Presents Positive Interim Clinical Data from Ongoing Phase 1/2 Trial of EBT-101 for the Treatment of HIV at ESGCT 30th Annual Congress. Retrieved October 25, 2023, from https://www.globenewswire.com/news-release/2023/10/25/2766525/0/en/Excision-BioTherapeutics-Presents-Positive-Interim-Clinical-Data-from-Ongoing-Phase-1-2-Trial-of-EBT-101-for-the-Treatment-of-HIV-at-ESGCT-30th-Annual-Congress.html[25] Alnylam Reports Additional Positive Interim Phase 1 Results for ALN-APP, in Development for Alzheimer’s Disease and Cerebral Amyloid Angiopathy. Retrieved October 25, 2023, from https://www.businesswire.com/news/home/20231025380274/en[26] Positive Phase 1 Data for Blue Lake Biotechnology’s Intranasal RSV Vaccine Published in Science Advances. Retrieved October 25, 2023, from https://www.businesswire.com/news/home/20231025585836/en[27] Skye Bioscience Announces Positive Phase 1 Trial Results for SBI-100 Ophthalmic Emulsion, Its First-in-Class CB1 Agonist Being Developed for the Treatment of Glaucoma. Retrieved October 25, 2023, from https://www.newsfilecorp.com/release/185167[28] New Data from Biogen’s Investigational Antisense Oligonucleotide (ASO) Targeting Tau Shows Promise for Potential New Generation of Treatments in Early Alzheimer’s Disease. Retrieved October 25, 2023, from https://investors.biogen.com/news-releases/news-release-details/new-data-biogens-investigational-antisense-oligonucleotide-aso[29] Biosyngen's BST02, the World's First TIL Therapy for Liver Cancer, is Granted an IND Approval by FDA. Retrieved October 26, 2023, from https://www.prnewswire.com/news-releases/biosyngens-bst02-the-worlds-first-til-therapy-for-liver-cancer-is-granted-an-ind-approval-by-fda-301968656.html[30] Mustang Bio Announces FDA Acceptance of IND Application for MB-109, a Novel Combination of MB-101 (IL13Rα2‐targeted CAR-T cell therapy) and MB-108 (HSV-1 oncolytic virus), for the Treatment of Recurrent Glioblastoma and High-Grade Astrocytoma. Retrieved October 26, 2023, from https://www.globenewswire.com/news-release/2023/10/26/2767618/0/en/Mustang-Bio-Announces-FDA-Acceptance-of-IND-Application-for-MB-109-a-Novel-Combination-of-MB-101-IL13R%CE%B12-targeted-CAR-T-cell-therapy-and-MB-108-HSV-1-oncolytic-virus-for-the-Treatm.html[31] Ascendis Presents Updated and New TransCon™ IL-2 β⁄γ Monotherapy and Combination Therapy Data Confirming Clinical Activity Across Tumor Types at ESMO 2023. Retrieved October 26, 2023, from https://investors.ascendispharma.com/news-releases/news-release-details/ascendis-presents-updated-and-new-transcontm-il-2-bg-monotherapy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

细胞疗法信使RNA免疫疗法临床申请临床结果

100 项与 BT-051 相关的药物交易

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适应症	最高研发状态	国家/地区	公司	日期
炎症性肠病	临床2期	-	Geneoscopy, Inc.初创企业	-
炎症性肠病	临床2期	-	Adiso Therapeutics, Inc.	-
溃疡性结肠炎	临床1期	美国	Adiso Therapeutics, Inc.	2021-11-30
溃疡性结肠炎	临床1期	格鲁吉亚	Adiso Therapeutics, Inc.	2021-11-30
溃疡性结肠炎	临床1期	摩尔多瓦	Adiso Therapeutics, Inc.	2021-11-30
溃疡性结肠炎	临床1期	波兰	Adiso Therapeutics, Inc.	2021-11-30

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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


NCT05084261 (PRNewswire) 人工标引	临床1期	溃疡性结肠炎	24	ADS051	壓網鹽鏇窪齋獵壓鏇獵(齋醖願齋繭襯選網觸製) = 顧製網淵蓋鹹膚膚製積艱夢衊鹹築願鬱製餘獵 (願鏇糧鹹膚壓願蓋淵網 ) 更多	积极	2023-10-22
				Placebo	壓網鹽鏇窪齋獵壓鏇獵(齋醖願齋繭襯選網觸製) = 憲窪繭淵膚醖顧獵製蓋艱夢衊鹹築願鬱製餘獵 (願鏇糧鹹膚壓願蓋淵網 ) 更多