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更新于：2025-07-28
Briquilimab
布利奎利单抗
更新于：2025-07-28
概要
基本信息
原研机构 |
非在研机构 |
最高研发阶段临床2期 |
首次获批日期- |
最高研发阶段(中国)- |
特殊审评快速通道 (美国)、孤儿药 (美国)、孤儿药 (欧盟)、罕见儿科疾病 (美国) |
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结构/序列
Sequence Code 682557638L
来源: *****
Sequence Code 682558090H
来源: *****
关联
11
项与 布利奎利单抗 相关的临床试验NCT06592768
A Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Safety and Efficacy of Briquilimab in Participants With Allergic Asthma
Study JSP-CP-012 is designed as a proof of concept, parallel-group, single dose, double blind, placebo-controlled study using an allergen challenge model. A single dose proof of concept approach at a dose level shown to be biologically active should inform the impact of briquilimab on allergic asthma as compared to placebo while minimizing exposure to participants, in a new indication.
开始日期2024-11-26 |
申办/合作机构 |
NCT06736262
Open-Label Extension Study to Evaluate Long Term Safety and Clinical Activity of Briquilimab in Chronic Urticaria
Phase 2, open-label, extension study to evaluate the long-term safety, clinical activity, and pharmacodynamics of briquilimab in participants previously enrolled in a Jasper sponsored CU clinical trial.
开始日期2024-11-26 |
申办/合作机构 |
NCT06353971
A Phase 1b/2a, Dose Escalation Trial of Safety, Pharmacokinetic/Pharmacodynamic and Preliminary Clinical Activity of Briquilimab in Adult Patients With Chronic Inducible Urticaria (CIndU) Who Remain Symptomatic Despite Treatment With H1- Antihistamines
This is an open-label, single ascending dose Phase 1b/2a trial to determine the safety and tolerability and assess the preliminary efficacy of briquilimab in adult participants with Cold Urticaria (ColdU) or Symptomatic Dermographism (SD), who remain symptomatic despite treatment with H1 antihistamines. The trial will explore three ascending dose levels which will be tested in three sequential cohorts.
开始日期2024-03-18 |
申办/合作机构 |
100 项与 布利奎利单抗 相关的临床结果
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100 项与 布利奎利单抗 相关的转化医学
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100 项与 布利奎利单抗 相关的专利（医药）
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6
项与 布利奎利单抗 相关的文献（医药）2025-02-01·Transplantation and Cellular Therapy
REMOVED: Allogeneic Hematopoietic Cell Transplantation with Briquilimab (JSP191) Anti-CD117 Antibody-Based Nonmyeloablative Conditioning for GATA2 Deficiency
Article
作者: Calvo, Katherine R ; Holland, Steven M ; Kraft, Ira L ; Long-Boyle, Janel Renee ; Avila, Daniele ; Duncan, Lisa ; Merkel, Roxanne ; Hickstein, Dennis ; Arnold, Danielle E ; Pang, Wendy W
This abstract has been removed: please see Elsevier policy on article withdrawal (https://www.elsevier.com/about/policies-and-standards/article-withdrawal). This article has been removed at the request of the author. This abstract has been removed because it was not presented at the 2025 BMT Tandem Meeting.
2024-09-01·Transplantation and Cellular Therapy
Pharmacokinetics of Briquilimab as a Conditioning Agent for Hematopoietic Stem Cell Transplantation in Patients With Severe Combined Immunodeficiency, Myelodysplastic Syndrome, or Acute Myeloid Leukemia
Article
作者: Pang, Wendy W ; Gobburu, Jogarao V S ; Jung, Dawoon ; Long-Boyle, Janel R
For successful engraftment of donor hematopoietic stem cells (HSC), conditioning with chemotherapy and/or radiation prior to hematopoietic cell transplantation (HCT) has been required to open marrow niche space and minimize the risk of immune rejection. Briquilimab, a humanized IgG1 monoclonal antibody that blocks the interaction between the c-Kit receptor and stem cell factor on various C-Kit expressing tissues including HSC, is a potential nonmyeloablative conditioning agent in clinical development for patients with severe combined immunodeficiency (SCID), myelodysplastic syndromes (MDS), and acute myeloid leukemia (AML). This study aimed to characterize pharmacokinetics (PK) and develop a population PK model of briquilimab after single intravenous infusions of 4 different doses in patients with SCID, MDS, or AML receiving HCT. The PK data was collected from 2 different studies: JAS-BMT-CP-001 and JSP-CP-003. JAS-BMT-CP-001 is a phase 1/2 open-label study of briquilimab as a conditioning agent prior to allogenic HCT in SCID patients. The participants received single intravenous infusions of 0.1, 0.3, 0.6, or 1.0 mg/kg. JSP-CP-003 was a phase 1a/b open-label study of briquilimab in combination with a standard conditioning regimen of low dose total body irradiation and fludarabine in MDS or AML subjects undergoing HCT. The participants received a single intravenous dose of 0.6 mg/kg briquilimab. In both studies, briquilimab PK samples were obtained at pre-treatment, 5 minutes post-end of infusion, 4- and 24-hours post-start of infusion, any time between 2 days and 30 days postinfusion, and on the day of HCT prior to donor cell infusion.The population PK model was developed using the PK data from these 2 clinical studies, and the effect of participants' baseline characteristics on the briquilimab PK was evaluated. PK simulations were performed using the developed PK model to calculate the time to reach target concentrations for HCT. A total of 49 participants (21 SCID adult and pediatric participants with a median age of 12 yr and 28 MDS/AML adult participants with a median age of 70 yr) were included in the PK analysis. A 2-compartment model with combined linear and non-linear elimination best described the PK of briquilimab. Body weight was determined as the sole covariate of the PK parameters among the explored covariates. For a typical subject with a body weight 70 kg, the estimated parameters for clearance, maximum metabolic rate of Michaelis Menten elimination, Michaelis Menten constant, central volume, peripheral volume, and intercompartmental clearance were 17.6 mL/h, 51,434.8 ng/h, 71.5 ng/mL, 3444.0 mL, 1613.3 mL, and 21.2 mL/h, respectively. The median time to reach target concentrations of 500, 1000, and 2000 ng/mL after a single dose of 0.6 mg/kg was calculated as 12.3, 10.4, and 7.7 days, respectively. The PK of intravenous briquilimab was characterized in subjects with SCID, MDS, or AML receiving HCT, and a population PK model was developed to estimate briquilimab clearance to use as a guide to the timing of donor cell infusion post-briquilimab. Body weight was identified as a significant covariate on elimination and volume of distribution of briquilimab.
2022-10-01·CPT: pharmacometrics & systems pharmacology
Physiologically‐based pharmacokinetic modeling of immunoglobulin and antibody coadministration in patients with primary human immunodeficiency
Article
作者: Kakkar, Tarundeep ; Deng, Rong ; Salerno, Sara N.
Abstract:
Intravenous immunoglobulin (IVIG) (2000 mg/kg) increased the clearance of the mouse monoclonal antibody 7E3, directed against platelet integrin IIb/IIIa (alpha IIb beta 3, CD41/CD61) in rodents. We wanted to investigate the effect of IVIG on clearance of monoclonal antibodies in humans as there is extremely limited data regarding this interaction in the literature. Using the tyrosine protein kinase KIT anti‐cluster of differentiation 117 (c‐Kit) humanized monoclonal antibody (JSP191) as a case study, we used physiologically‐based pharmacokinetic (PBPK) modeling to evaluate the pharmacokinetic interaction between monoclonal antibodies and IVIG at doses (300–600 mg/kg) administered to patients with primary human immunodeficiency (PI). We first characterized the interaction between monoclonal antibodies and IVIG in PK‐Sim®/MoBi® using published literature data, including the following: IVIG plus 7E3 in mice and rats and IVIG plus the human anti‐C5 monoclonal antibody tesidolumab in adults with end‐stage renal disease. We next developed a PBPK model using digitized data for JSPI91 alone in older adults with myelodysplastic syndrome and acute myeloid leukemia and in pediatric patients with severe combined immunodeficiency (SCID). Finally, we simulated the impact of IVIG (300–2000 mg/kg) coadministration with JSP191 on the area under the curve of JSP191 in patients with SCID. Model predictions were within 1.5‐fold of observed values for 7E3 plus IVIG and tesidolumab plus IVIG as well as for JSP191 administered alone. Based on our simulations, IVIG doses ≥500 mg exceeded the 80%–125% no‐effect boundaries. IVIG treatment with monoclonal antibodies in patients with PI may result in a clinically significant interaction depending on the IVIG dose administered and the exposure–response relationship for the specific monoclonal antibody.
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项与 布利奎利单抗 相关的新闻（医药）2025-07-27
·生物探索
引言DNA是指导一切生命活动的终极密码。然而，当这本密码本出现“印刷错误”时，身体就会陷入混乱。对于范可尼贫血（Fanconi Anemia, FA）患者而言，他们的DNA修复系统天生存在缺陷，这使得他们不仅面临着骨髓衰竭的威胁，还极易罹患癌症。长期以来，造血干细胞移植（Hematopoietic Stem Cell Transplantation, HSCT）是治愈这一疾病的唯一希望，但其过程却如同一场“与魔鬼的交易”——患者必须先承受大剂量放化疗带来的“焦土政策”，摧毁自身造血和免疫系统，才能为健康的“种子”细胞腾出空间。这种疗法对于DNA本就脆弱的FA患者来说，毒副作用巨大，往往带来长期的健康隐患。有没有一种更温和、更精准的方式，既能治愈疾病，又能最大限度地保护患者？7月22日，《Nature Medicine》上发表的一篇题为 “Irradiation- and busulfan-free stem cell transplantation in Fanconi anemia using an anti-CD117 antibody: a phase 1b trial” 的研究，为我们揭示了一条充满希望的新路径。研究团队报告了一项开创性的1b期临床试验，他们利用一种抗体药物，成功地为三名FA患儿实施了无需辐射和白消安（busulfan）的干细胞移植。这不仅是一次治疗技术的革新，更可能预示着一个全新的、更安全的移植时代的到来。与魔鬼的交易：治愈范可尼贫血，为何必须“杀敌一千，自损八百”？要理解这项新技术的革命性，我们首先走进范可尼贫血（Fanconi Anemia, FA）患者的世界。这是一种罕见的遗传性疾病，其核心问题在于基因突变导致细胞内的DNA修复机制失灵。我们知道，细胞在分裂和应对外界环境（如紫外线、化学物质）时，DNA难免会受到损伤。正常人体内有一套高效的修复系统，像一群勤劳的维修工，随时修补这些损伤。然而，FA患者的“维修工”却处于“罢工”状态，特别是对于一种被称为“链间交联”（interstrand crosslinks）的复杂损伤束手无策。这会导致一系列灾难性的后果。首先是骨髓衰竭（Bone Marrow Failure, BMF）。骨髓是人体的“造血工厂”，持续不断地生产红细胞、白细胞和血小板。FA患者的造血干细胞由于DNA损伤不断累积，会逐渐凋亡或功能丧失。据统计，约80%的FA患者在12岁前就会出现进行性的骨髓衰竭，导致贫血、易感染和出血不止。其次是癌症易感性。DNA修复缺陷意味着基因突变更容易发生和积累，这是癌症发生的温床。FA患者罹患急性髓系白血病（Acute Myeloid Leukemia, AML）和头颈部、妇科鳞状细胞癌的风险比普通人高出数百甚至数千倍。面对如此凶险的疾病，造血干细胞移植（HSCT）成为了唯一的治愈手段。其原理很简单：用健康的供者干细胞，替换掉患者有缺陷的造血系统。然而，实施起来却异常残酷。在移植前，患者必须接受一个名为“预处理”（Conditioning）的环节。传统的预处理方案通常采用全身放疗（Total Body Irradiation, TBI）或大剂量化疗药物如白消安（busulfan）。这种预处理方案的目的有两个：一是清空骨髓，为外来的健康干细胞（“种子”）腾出“生存空间”（“土壤”），使其能够顺利植入、生长；二是免疫抑制，摧毁患者自身的免疫系统，防止其对外来的供者细胞产生排斥反应。这套方案对于普通患者来说已是严峻的考验，而对于DNA修复能力本就脆弱的FA患者，则无异于一场“核打击”。放疗和白消安这类基因毒性（genotoxic）的手段，会造成全身性的DNA损伤。FA患者的身体无法有效修复这些损伤，导致了极其严重的毒副作用，包括严重的短期毒性和长期的后遗症，如器官损伤、不孕不育，以及最令人担忧的——二次肿瘤。研究表明，接受过传统HSCT的FA患者，其罹患实体瘤的风险会再次被急剧放大，并且发病年龄显著提前。多年来，研究人员一直在尝试降低预处理的强度，但完全取消TBI或白消安这类核心的清髓手段，又常常导致移植失败——供者的干细胞无法成功“安家落户”，即植入失败（graft failure）。这使得FA的治疗陷入了一个两难的“魔鬼困境”：要么承受剧毒的治疗以求治愈，要么因无法耐受而放弃希望。如何才能打破这个僵局，找到一种既能有效清髓、又能避免全身性DNA损伤的“鱼与熊掌兼得”之法？这正是该研究试图回答的核心问题。破局之路：一把“细胞钥匙”，如何颠覆百年移植规则？研究团队构想了一个巧妙的替代方案。他们认为，既然“焦土政策”式的无差别攻击太过猛烈，何不换用“精准制导”的战术，只针对需要清除的目标——患者自身的造血干细胞（Hematopoietic Stem and Progenitor Cells, HSPCs）——发起攻击？他们的策略可以分解为三个环环相扣的创新部分。第一部分：精准打击的“生物导弹”——Briquilimab抗体。研究人员找到的“精准制导武器”是一种名为Briquilimab（也称JSP191）的单克隆抗体。它的靶点是造血干/祖细胞表面一个叫做CD117的蛋白。CD117，又名c-KIT，是干细胞因子（stem cell factor）的受体。你可以把它想象成造血干细胞的“能量接收天线”，是维持其生存的关键信号。Briquilimab抗体的作用，就是精确地与CD117结合，像一个“绝缘帽”一样盖住了这个“天线”，阻止生存信号，导致造血干细胞逐渐凋亡。更巧妙的是，研究人员对Briquilimab的结构进行了特殊改造（aglycosylated，去糖基化），使其更像是一种温柔的“劝退”，而非暴力的“强拆”。最关键的一点是，Briquilimab的作用高度特异，只针对表达CD117的造血干/祖细胞，而不会像放化疗那样，对全身其他组织的细胞造成DNA损伤。这就从根本上避免了传统预处理的基因毒性。第二部分：保驾护航的“和平部队”——温和的免疫抑制。仅仅清空骨髓还不够，还必须防止患者的免疫系统攻击新来的供者细胞。为此，研究团队保留了一套不含TBI和白消安的、相对温和的免疫抑制方案，包括兔抗胸腺细胞球蛋白（rATG）、低剂量的氟达拉滨（fludarabine）和环磷酰胺（cyclophosphamide），以及利妥昔单抗（rituximab）。这个组合拳的目标明确：在不造成严重基因毒性的前提下，为供者细胞的植入创造一个和平、宽容的免疫环境。第三部分：精挑细选的“精英援军”——αβ-T细胞去除的半相合移植。为了扩大供者库，研究团队采用了半相合移植（haploidentical HSCT），即使用父母作为供者。为了解决半相合移植中高发的移植物抗宿主病（Graft-versus-Host Disease, GvHD），研究团队在移植前，对移植物进行了体外处理，选择性地去除其中惹是生非的TCRαβ+ T细胞和CD19+ B细胞。经过这番“精挑细选”后，最终输注给患者的移植物，富含高剂量的CD34+造血干细胞，同时TCRαβ+ T细胞的数量被控制在极低的水平。这就像是派遣了一支由“工程师”（干细胞）组成的“精英建设部队”，同时剔除了部队里可能引发冲突的“好斗士兵”。综合来看，这个“Briquilimab精准清髓 + 温和免疫抑制 + αβ-T细胞去除的半相合移植”的新策略，理论上完美地解决了传统疗法的几大痛点：避免了基因毒性，扩大了供者来源，并降低了GvHD的风险。但理论终究是理论，它在真实的人体中表现如何？这正是这项1b期临床试验要回答的问题。初战告捷：三位小勇士的重生之路，数据揭示了什么？这项1b期临床试验招募了三名急需移植的FA患儿，他们分别来自不同的种族背景（白人、亚洲、非洲裔美国人），年龄分别为7岁、10岁和8岁，所携带的FANCA基因突变也各不相同。这为初步评估该方案的普适性提供了宝贵的数据。研究的主要终点是安全性和植入情况。结果令人振奋。1. 安全性：一场没有“硝烟”的战斗三名患儿在接受了单次静脉注射 0.6 mg/kg的Briquilimab 后，均未出现任何与该药物相关的毒副反应。整个预处理和移植期间，最令人担心的肝脏静脉闭塞病（VOD）没有发生。虽然患者们仍然经历了2级或3级的口腔黏膜炎，这可能是由于环磷酰胺等免疫抑制药物引起，但与传统高强度预处理相比，整体的毒性反应显著降低。这证明，用Briquilimab替代TBI和白消安，确实大大减轻了治疗的“痛苦指数”。2. 植入情况：健康“种子”快速生根发芽植入成功是移植最关键的标志。数据显示，三名患儿的植入过程非常迅速且稳健：中性粒细胞恢复到安全水平（>500个/μL）的中位时间仅为 11天（范围11-13天）；摆脱血小板输注依赖的中位时间为 13天（范围11-14天）；所有患儿在移植后的第8天或之前就不再需要输注红细胞。这样的植入速度与既往报道的使用更强预处理方案的移植结果相当，甚至更快，表明Briquilimab的“精准清髓”效果足以媲美传统的“焦土政策”。3. 嵌合率：供者细胞全面“接管”嵌合率（Chimerism）是衡量供者细胞在患者体内占比的金标准。这项研究的嵌合率数据堪称完美。在移植后30天，患者骨髓中所有细胞谱系的供者细胞比例均达到了 92%至100%。更重要的是，在长达两年的持续随访中，这种完全供者嵌合的状态一直稳定维持。在最近一次检测中，三名患者骨髓中所有细胞谱系的供者嵌合率几乎都在 99%至100% 之间。这意味着，健康的供者造血系统已经完全取代了患者原有的缺陷系统，并牢牢地扎下了根。4. 药物代谢与疾病纠正Briquilimab的清除过程稳定且可预测，在移植当天，所有患者体内的药物浓度都顺利降到了安全阈值（<1,500 ng/ml）以下，确保了供者细胞的顺利植入。更令人激动的是，通过经典的DEB诱导的染色体断裂试验发现，移植后所有三名患者的淋巴细胞对DEB的敏感性完全消失，染色体断裂率恢复到了健康人的水平。这从细胞遗传学的层面，证明了FA的疾病表型已经被彻底纠正。骨髓细胞的体外集落形成能力也恢复到了与健康对照组相当的水平。5. 免疫重建与GvHD：可控的“小插曲”在大家最关心的GvHD方面，结果也令人满意。三名患者均未发生急性GvHD。仅有一名患者在后期出现了中度的慢性GvHD，这与患者依从性不佳有关，并得到了有效控制。在半相合移植的背景下，如此低的GvHD发生率，充分体现了αβ-T细胞去除技术的优越性。综合所有数据，这项1b期临床试验证明，Briquilimab导向的无TBI/白消安预处理方案，在FA患者中是安全且高效的。不止于此：从范可尼贫血出发，这场“温和革命”将走向何方？尽管这项研究的样本量很小（仅3人），但其开创性的成果为血液病治疗领域描绘了一幅激动人心的未来图景。它的意义远远超出了范可尼贫血本身。首先，它为一系列遗传性血液病提供了新的治疗范式。FA患者由于其极端的基因毒性敏感，是检验这种“温和”疗法的最佳“试金石”。既然该方案在最脆弱的FA患者身上都取得了成功，那么它很有可能被安全地推广到其他需要干细胞移植的非恶性血液病中，例如镰状细胞病（Sickle Cell Disease）、地中海贫血（Thalassemia），以及其他类型的骨髓衰竭综合征。对于这些患者而言，能够摆脱放化疗的长期阴影，意味着生活质量和远期生存的巨大飞跃。其次，它可能改变移植的时机，实现“防患于未然”。传统移植的高风险性，使得医生和患者往往要等到疾病进展到非常严重的阶段才“下定决心”进行移植。而一个更安全、更温和的移植方案，则可能让早期干预成为现实。我们可以在患者身体状况尚好、器官功能未受损、未发生癌变时就进行移植，从而大大提高移植的成功率。这是一种从“被动挽救”到“主动治愈”的理念转变。再次，它为未来的“全抗体”预处理方案奠定了基础。这项研究虽然去除了TBI和白消安，但仍然使用了环磷酰胺和氟达拉滨等化疗药物进行免疫抑制。这提示我们，通往“完全无毒”的道路还未走完。然而，这项研究的成功，为最终实现完全不含任何基因毒性药物的、纯抗体驱动的预处理方案铺平了道路。未来，我们或许可以开发出更精准、更高效的抗体组合，来分别完成清髓和免疫抑制的任务，将移植的副作用降至最低。当然，我们必须保持严谨的科学态度。这项研究仍处于早期阶段，其1b期试验的主要目的是验证安全性。目前，这项研究的2a期临床试验（ClinicalTrials.gov identifier: NCT04784052）正在进行中，更大规模的患者队列将为我们提供更可靠的有效性数据。关于这种新疗法是否真的能降低二次肿瘤的远期风险，还需要数十年乃至更长时间的随访才能得出结论。尽管如此，这项发表在《自然-医学》上的研究，无疑是精准医学在血液病治疗领域取得的一次里程碑式的胜利。它照亮了那些在黑暗中挣扎的FA患者家庭，也为整个干细胞移植领域指明了一个更人道、更安全、更充满希望的发展方向。这场由一把“细胞钥匙”开启的“温和革命”，或许才刚刚拉开序幕。它让我们有理由相信，在不远的未来，治愈将不再意味着必须承受毁灭性的代价，医学的进步终将让生命在重塑的过程中，绽放出更温柔而坚韧的光芒。参考文献Agarwal, R., Bertaina, A., Soco, C. et al. Irradiation- and busulfan-free stem cell transplantation in Fanconi anemia using an anti-CD117 antibody: a phase 1b trial. Nat Med (2025). https://doi.org/10.1038/s41591-025-03817-1声明：本文仅用于分享，不代表平台立场，如涉及版权等问题，请尽快联系我们，我们第一时间更正，谢谢！往期热文：Nature Biotechnology | 血液中的“肠道私语”：一种颠覆性的癌症检测方法正在浮现Nature | 脂肪的“记忆”与“遗忘”：研究揭示减肥逆转衰老的细胞奥秘Science | 我们的大脑从未停止生长？解密成年人大脑中持续“上新”的神经细胞Cell | 告别“猜谜式”基因研究！“女娲”模型从源头破译调控密码，让DNA序列“开口说话”Nature | 继“人类基因组计划”后，生命科学的下一个宏伟蓝图：绘制人体亿万细胞的“突变天书”Science | 免疫系统的“变形金刚”：一个会“倒立”的酶，如何帮我们抵御病毒入侵？
临床1期
2025-07-13
·药明康德
▎药明康德本期看点1. 单抗疗法briquilimab在针对慢性自发性荨麻疹患者开展的1b/2a期研究中表现亮眼，两个单剂量队列中有89%的患者达成完全缓解（CR）。2. 噬菌体疗法BX004用于治疗囊性纤维化（CF）患者因铜绿假单胞菌引起的慢性肺部感染，在一项早期临床试验中与安慰剂相比显著降低了细菌数量约500倍，无耐药发生，同时有效保护了健康微生物组。Briquilimab：公布1b/2a期临床试验数据Jasper Therapeutics公司公布了其候选单抗briquilimab用于治疗慢性自发性荨麻疹的1b/2a期研究BEACON的初步数据。Briquilimab是一种非糖基化单克隆抗体，可通过靶向细胞表面受体c-Kit（也称为CD117）以避免与干细胞因子结合，从而阻断肥大细胞中的关键生存信号，造成细胞凋亡与耗竭，从而消除肥大细胞驱动疾病（如慢性荨麻疹）中炎症反应的潜在来源。此次公布的结果显示，briquilimab在240 mg和360 mg单剂量队列中表现出深度且快速的疾病控制效果，两组共9名受试者中有8人（89%）实现了CR，7人（78%）在第2周即达到临床缓解。此外，在BEACON研究中完成治疗并转入开放标签扩展研究、接受每8周180 mg剂量治疗的受试者中，也显示出强大的临床疗效：在第12周时，11名受试者中有8人（73%）达到了CR。安全性方面，研究期间未报告任何≥3级的治疗相关不良事件。BX004：公布1b/2a期临床试验数据BiomX公司宣布，其噬菌体疗法BX004的首个人体试验结果已在学术期刊Nature Communications上发表。BX004是一种固定的多噬菌体组合疗法，用于治疗囊性纤维化患者因铜绿假单胞菌引起的慢性肺部感染。铜绿假单胞菌是导致CF患者发病和死亡的主要原因之一。论文中的数据表明，在针对抗生素耐药性铜绿假单胞菌感染的1b/2a期临床试验中，BX004的安全性和耐受性良好，在所有患者中和所有测试剂量水平下均未发生治疗相关安全事件。与接受安慰剂治疗的患者相比，接受BX004治疗患者的细菌数量下降了2.7 log₁₀（约500倍），且未出现耐药现象，同时有效保护了患者的健康微生物组。该公司正在推进BX004的2b期临床试验，预计将于2026年第一季度获得顶线结果。Paxalisib：公布1b期联合治疗试验中首例患者的数据Kazia Therapeutics公司公布了一项1b期临床试验中首例患者的初步结果，该研究旨在评估其可穿越血脑屏障的在研口服PI3K/Akt/mTOR通路抑制剂paxalisib联用帕博利珠单抗和标准化疗的疗效与安全性。该公司于2016年底从基因泰克（Genentech）获得了paxalisib的许可。该疗法此前已获得多项资格，包括美国FDA授予的针对胶质母细胞瘤的孤儿药资格和快速通道资格（FTD），针对携带PI3K通路突变、需联合放疗治疗的实体瘤脑转移适应症的FTD，以及针对弥漫性内生性桥脑胶质瘤（DIPG）和非典型畸胎样/横纹肌样肿瘤的孤儿药资格与罕见儿科疾病认定。此次公布的结果显示，一名61岁、肿瘤已局部转移至左肺上叶的三阴性乳腺癌女性患者在接受治疗仅21天后，其循环肿瘤细胞（CTCs）减少了超过50%，且CTC细胞簇也显著减少。这些早期临床数据与此前公布的临床前数据一致。ELU42：IND申请获得FDA许可Eluciderm公司宣布，美国FDA已批准其针对新药ELU42的IND申请，可开展一项针对糖尿病足溃疡（DFUs）患者的1/2a期开放标签研究，以评估ELU42的安全性和疗效。ELU42是一款新型外用小分子Wnt通路调节剂，具有抑菌特性，旨在治疗慢性开放性伤口。参考资料（可上下滑动查看）[1] Eleva administers first dose of its Factor H biological treatment in C3-Glomerulopathy to healthy volunteers. Retrieved July 11, 2025, from https://elevabiologics.com/eleva-administers-first-dose-of-its-factor-h-biological-treatment-in-c3-glomerulopathy-to-healthy-volunteers/[2] First patient dosed in the Phase I clinical trial of PulseSight Therapeutics’ PST-611 treatment for dry AMD/Geographic Atrophy. Retrieved July 11, 2025, from https://pulsesight.com/2025/07/07/first-patient-dosed-in-the-phase-i-clinical-trial-of-pulsesight-therapeutics-pst-611-treatment-for-dry-amd-geographic-atrophy/[3] Eluciderm, Inc. Announces U.S. FDA Clearance of IND Application for ELU42. Retrieved July 11, 2025, from https://www.businesswire.com/news/home/20250708620364/en/Eluciderm-Inc.-Announces-U.S.-FDA-Clearance-of-IND-Application-for-ELU42[4] Solid Biosciences Announces FDA IND and Health Canada CTA Approval for First-in-Class Cardiac Gene Therapy to Treat Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT). Retrieved July 11, 2025, from https://www.globenewswire.com/news-release/2025/07/08/3112195/0/en/Solid-Biosciences-Announces-FDA-IND-and-Health-Canada-CTA-Approval-for-First-in-Class-Cardiac-Gene-Therapy-to-Treat-Catecholaminergic-Polymorphic-Ventricular-Tachycardia-CPVT.html[5] Sensorium Therapeutics Receives FDA IND Clearance for SNTX-2643, a First-in-Class, Rapid-Acting Anxiolytic. Retrieved July 11, 2025, from https://www.prnewswire.com/news-releases/sensorium-therapeutics-receives-fda-ind-clearance-for-sntx2643-a-firstinclass-rapidacting-anxiolytic-302499309.html[6] BiomX Announces Publication in Nature Communications of Phage Cocktail BX004 Phase 1b/2a Part 1 Data Demonstrating Strong Activity in Cystic Fibrosis. Retrieved July 11, 2025, from https://www.globenewswire.com/news-release/2025/07/08/3111723/0/en/BiomX-Announces-Publication-in-Nature-Communications-of-Phage-Cocktail-BX004-Phase-1b-2a-Part-1-Data-Demonstrating-Strong-Activity-in-Cystic-Fibrosis.html[7] Purespring Therapeutics announce FDA IND clearance for Phase I/II clinical trial for primary IgA nephropathy (IgAN). Retrieved July 11, 2025, from https://www.globenewswire.com/news-release/2025/07/09/3112303/0/en/Purespring-Therapeutics-announce-FDA-IND-clearance-for-Phase-I-II-clinical-trial-for-primary-IgA-nephropathy-IgAN.html[8] Avenzo Therapeutics Announces First Patient Dosed in Phase 1/2 Clinical Study of AVZO-1418, a Potential Best-in-Class EGFR/HER3 Bispecific Antibody-Drug Conjugate. Retrieved July 11, 2025, from https://avenzotx.com/press-releases/avenzo-therapeutics-announces-first-patient-dosed-in-phase-1-2-clinical-study-of-avzo-1418-a-potential-best-in-class-egfr-her3-bispecific-antibody-drug-conjugate/[9] BlueRock Therapeutics announces first patient receives investigational therapy in Phase 1/2a clinical trial of OpCT-001 for the treatment of primary photoreceptor diseases. Retrieved July 11, 2025, from https://www.bayer.com/en/us/news-stories/treatment-of-primary-photoreceptor-diseases-0[10] Jasper Therapeutics Reports Clinical Data Update from Briquilimab Studies in Chronic Spontaneous Urticaria. Retrieved July 11, 2025, from https://ir.jaspertherapeutics.com/news-releases/news-release-details/jasper-therapeutics-reports-clinical-data-update-briquilimab#:~:text=REDWOOD%20CITY%2C%20Calif.%2C%20July%2007%2C%202025%20%28GLOBE%20NEWSWIRE%29,CSU%20and%20providing%20an%20update%20on%20the%20program.[11] Kazia Therapeutics Reports Early Efficacy Data from First Triple-Negative Breast Cancer Patient Receiving Paxalisib Combination Regimen achieving >50% Reduction in Circulating Tumor Cells in Phase 1b Trial. Retrieved July 11, 2025, from https://www.prnewswire.com/news-releases/kazia-therapeutics-reports-early-efficacy-data-from-first-triple-negative-breast-cancer-patient-receiving-paxalisib-combination-regimen-achieving-50-reduction-in-circulating-tumor-cells-in-phase-1b-trial-302500778.html[12] The Institute for Research in Immunology and Cancer (IRIC) of the Université de Montréal and IRICoR Announce initiation of Phase 1 Trial of Licensed Small Molecule Therapy for Solid Tumors. Retrieved July 11, 2025, from https://www.prnewswire.com/news-releases/the-institute-for-research-in-immunology-and-cancer-iric-of-the-universite-de-montreal-and-iricor-announce-initiation-of-phase-1-trial-of-licensed-small-molecule-therapy-for-solid-tumors-302499345.html[13] Neumora Therapeutics Announces Initiation of Phase 1 Clinical Study of M4 Positive Allosteric Modulator NMRA-861. Retrieved July 11, 2025, from https://ir.neumoratx.com/news-releases/news-release-details/neumora-therapeutics-announces-initiation-phase-1-clinical-study[14] Cocrystal Pharma to Present Data from Phase 1 Study of First-in-Class Norovirus Protease Inhibitor CDI-988 at the 9th International Calicivirus Conference. Retrieved July 11, 2025, from https://www.globenewswire.com/news-release/2025/07/10/3113201/0/en/Cocrystal-Pharma-to-Present-Data-from-Phase-1-Study-of-First-in-Class-Norovirus-Protease-Inhibitor-CDI-988-at-the-9th-International-Calicivirus-Conference.html[15] Krystal Biotech Announces First Patient Dosed in Phase 1/2 Trial of KB801 for the Treatment of Neurotrophic Keratitis. Retrieved July 11, 2025, from https://www.globenewswire.com/news-release/2025/07/09/3112425/0/en/Krystal-Biotech-Announces-First-Patient-Dosed-in-Phase-1-2-Trial-of-KB801-for-the-Treatment-of-Neurotrophic-Keratitis.html免责声明：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：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
临床结果微生物疗法临床2期临床1期
2025-07-08
iStock,
Andrii Yalanskyi
After issues with a batch of Jasper Therapeutics’ investigational antibody led to “lower” therapeutic effects in several patients, analysts at BMO Capital Markets said they “believe investors won’t feel comfortable coming back to the story.”
Issues with a batch of the investigational antibody briquilimab “confounded” the readout of Jasper Therapeutics’ Phase Ib/IIa BEACON study in chronic spontaneous urticaria, leading to “lower” therapeutic effects in patients treated with the compromised drugs, the company
reported
Monday.
Jasper’s shares plunged Monday, with the California biotech down 55% at market close. In the aftermath of the drug lot issues, Jasper will implement cost-cutting measures, including a potential strategic restructuring. The biotech had
$48.8 million
in cash and cash equivalents as of March 31.
Results from BEACON “were impacted by an inactive drug product lot,” analysts at William Blair said in a note to investors on Monday, adding that the two affected dose arms—240 mg and 240 mg loading dose followed by 180 mg every eight weeks—“were of high interest as potential doses” for the pivotal development of briquilimab.
For BMO Capital Markets, the lot issue could make Jasper stock a harder sell for investors. “Given the market environment,” the analysts wrote, “we believe investors won’t feel comfortable coming back to the story following today’s update.”
Jasper is developing briquilimab for chronic spontaneous urticaria (CSU), for which it is running
BEACON
, a randomized, double-blinded and placebo-controlled study with around 40 participating patients. The trial administered briquilimab in six ascending doses and Jasper on Monday reported data from 13 patients who were treated with the single 240 mg dose, or 240 mg loading dose followed by 180 mg injections every eight weeks.
Ten of those 13 patients received briquilimab from the compromised lots. Results showed that these patients had “lower than expected” improvements in mean tryptase levels, a key pharmacodynamic outcome of BEACON and a biomarker of CSU. Briquilimab treatment in these 10 patients also showed “no discernable impact” on scores in the Urticaria Activity Score, the study’s primary efficacy outcome.
Meanwhile, two patients treated with briquilimab from a different drug lot achieved a complete response.
The exact issue with the drug lot remains unknown and Jasper is currently looking into the matter. The company is also providing new drug product to transition these 10 patients into a new drug lot that has demonstrated efficacy in earlier cohorts. Jasper will also add 10 to 12 new patients across both the affected dose arms “to ensure a robust data set.”
Jasper expects to report data from these additional BEACON participants in the fourth quarter. Because of Monday’s lot issue, the company has also pushed back the start of a Phase IIb CSU study to mid-2026.
The lot issues also affected an asthma trial of briquilimab. Jasper will no longer pursue this indication for the antibody.
临床结果临床2期临床1期财报临床失败
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研发状态
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| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| 慢性荨麻疹 | 临床2期 | 美国 | 2024-11-26 | |
| 慢性荨麻疹 | 临床2期 | 德国 | 2024-11-26 | |
| 急性胸部综合征 | 临床2期 | 美国 | 2022-05-12 | |
| β地中海贫血 | 临床2期 | 美国 | 2022-05-12 | |
| 镰状细胞遗传性状 | 临床2期 | 美国 | 2022-05-12 | |
| 腺苷脱氨酶缺乏 | 临床2期 | 美国 | 2017-03-20 | |
| 网状细胞发育不全 | 临床2期 | 美国 | 2017-03-20 | |
| 镰状细胞血症 | 临床2期 | 美国 | - | |
| 过敏性哮喘 | 临床1期 | 加拿大 | 2024-11-26 | |
| 慢性肉芽肿病 | 临床1期 | 美国 | 2023-01-02 |
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临床结果
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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 |
|---|
临床1/2期 | 27 | Briquilimab 40mg | 積鹹選鏇壓糧淵鏇襯壓(淵壓鑰繭築餘顧夢鹹壓) = 鏇獵鑰獵衊網築齋鬱願 壓襯鑰艱築製餘壓餘窪 (夢遞齋淵夢顧繭獵繭糧 ) 更多 | 积极 | 2025-06-14 | ||
Briquilimab 120mg | 積鹹選鏇壓糧淵鏇襯壓(淵壓鑰繭築餘顧夢鹹壓) = 鹹鹹積憲觸鏇蓋構齋鏇 壓襯鑰艱築製餘壓餘窪 (夢遞齋淵夢顧繭獵繭糧 ) 更多 | ||||||
临床1/2期 | 49 | Briquilimab 240mg single dose | 蓋憲襯醖夢鏇膚壓壓淵(齋鬱鬱蓋製憲願繭獵憲) = 觸廠獵襯淵餘餘鬱糧選 願簾餘齋蓋窪襯鬱糧獵 (淵遞構築膚範顧繭鏇積 ) 更多 | 积极 | 2025-01-08 | ||
Placebo (single dose) | 蓋憲襯醖夢鏇膚壓壓淵(齋鬱鬱蓋製憲願繭獵憲) = 構構淵鑰築廠構獵顧廠 願簾餘齋蓋窪襯鬱糧獵 (淵遞構築膚範顧繭鏇積 ) 更多 | ||||||
临床1/2期 | 15 | 鏇餘觸糧襯壓鏇憲憲窪(鹹選夢顧憲夢獵繭獵簾) = None 鬱選鹹遞選衊壓鑰繭壓 (淵構獵鏇壓窪構鏇醖淵 ) 更多 | 积极 | 2024-10-14 | |||
Briquilimab 120mg | |||||||
临床1/2期 | 3 | 壓構膚獵艱鹹鏇築築積(醖選糧選襯積構壓簾顧) = 壓廠觸鑰糧遞壓觸繭壓 遞膚築範醖壓醖鬱構夢 (簾廠築獵淵觸鏇鑰製淵 ) 更多 | - | 2023-12-09 | |||
临床1期 | 29 | 鏇衊構鹽糧範衊膚鏇觸(觸憲壓鏇鹹夢衊範鏇廠) = 鏇艱醖壓築餘願繭憲憲 醖襯憲齋範製廠構艱糧 (壓遞鹹蓋築選獵鏇艱鏇 ) 更多 | - | 2023-04-23 | |||
临床1期 | 12 | 衊願積範顧壓遞淵齋積(鏇顧衊選積顧蓋顧鹽衊) = 6 of whom were MRD negative at the latest post-HCT evaluable timepoint 繭積蓋鏇範膚蓋願鑰襯 (觸範構範壓夢鹹鏇鏇遞 ) 更多 | 积极 | 2023-02-01 | |||
Fludarabine 30 mg/m2/day | |||||||
N/A | - | JSP191 + Flu/TBI | 齋觸膚淵顧構襯構網醖(遞蓋膚獵製廠廠獵鏇窪) = were elevated in BM plasma in cGVHD 憲獵衊衊願糧築選襯製 (蓋遞獵遞襯築鏇網顧鹹 ) 更多 | - | 2023-02-01 | ||
临床1期 | 12 | 壓顧觸襯構築鏇壓壓憲(獵窪齋簾淵淵鹹積範壓) = 糧鑰蓋憲鹽鑰淵廠蓋憲 顧鹽顧顧簾壓簾醖構選 (夢憲鹹願鑰窪窪憲觸簾 ) 更多 | 积极 | 2023-02-01 | |||
临床1/2期 | 2 | 鹽鏇糧窪廠齋構餘餘顧(襯範糧夢鑰糧網網衊願) = 鏇糧網鏇糧獵網醖顧繭 觸繭鏇遞鹽鹽遞願簾選 (獵積繭醖艱製壓憲廠鑰 ) | 积极 | 2022-09-26 | |||
临床1期 | 24 | 憲鏇鹽膚壓窪願鹹憲夢(蓋廠鏇窪蓋網蓋廠鹽鏇) = 1pt with grade III-IV acute GI GVHD，1pts with secondary graft failure 窪觸醖壓廠觸鑰襯糧顧 (淵網窪顧繭遞淵窪選築 ) | 积极 | 2022-04-26 |
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