This is a Phase 1, single-center, open-label, single-dose trial. Sixteen participants are planned: 8 participants with moderate renal impairment and 8 matched control participants with normal renal function.

开始日期2025-11-30

申办/合作机构

Sparrow Pharmaceuticals, Inc.

NCT07226635

/ Active, not recruiting临床1期

Pharmacokinetic Interaction Trial of Clofutriben With Midazolam and Prednisolone

Drug: 2 single oral doses of midazolam, 2 single oral doses of prednisolone , and clofutriben once daily for 16 days.

开始日期2025-10-03

申办/合作机构

Sparrow Pharmaceuticals, Inc.

ISRCTN42727091

/ Recruiting临床2期IIT

Dissecting the Contribution of glucocorticoid metabolism in Mild Autonomous Cortisol Secretion: a randomised controlled trial of the 11ß-HSD1 inhibitor SPI-62

开始日期2024-03-04

申办/合作机构

University of Oxford

100 项与克洛曲本相关的临床结果

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100 项与克洛曲本相关的转化医学

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100 项与克洛曲本相关的专利（医药）

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项与克洛曲本相关的文献（医药）

2025-11-01·CTS-Clinical and Translational Science

Human Adipose Tissue 11β‐Hydroxysteroid Dehydrogenase Type 1 Inhibition Without Tachyphylaxis by Clofutriben, a Pseudo‐Irreversible Enzyme Inhibitor

Article

作者: Magers, Tobias ; Rinas, Aimee ; Katz, David A. ; Schmicker, Caleb ; Marmon, Tonya K. ; Lin, Shin‐Kai ; An, Guohua ; Abad‐Zapatero, Cele ; Gutierrez, Julio A. ; Engel, Brian

ABSTRACT:

Inhibitors of 11β‐hydroxysteroid dehydrogenase type 1 (HSD‐1), the pre‐receptor regulator of intracellular active glucocorticoid levels, have promise to treat glucocorticoid excess whether endogenous (neoplastic or physiological elevated cortisol) or iatrogenic (glucocorticoid medicine toxicity). Three structurally distinct HSD‐1 inhibitors have shown tachyphylaxis of pharmacodynamic activity in adipose, a key target tissue, suggesting that the clinical utility of the class might be limited by that pharmacological phenomenon. To determine whether the potent inhibitor clofutriben also showed adipose tachyphylaxis, HSD‐1 activity was measured during daily oral clofutriben administration and infusion of mass‐labeled enzyme substrate (cortisone) via microdialysis catheters placed in subcutaneous abdominal adipose of 12 persons with type 2 diabetes and obesity. During initial dosing, clofutriben was associated with dose‐dependent decreases of mass‐labeled HSD‐1 product (cortisol) in adipose dialysate, from < 25% at 1 mg to > 90% at 6–10 mg. At steady state, all clofutriben doses were associated with > 90% decreases of mass‐labeled cortisol in adipose dialysate with no evidence of tachyphylaxis. By surface plasmon resonance, clofutriben showed fast‐on, slow‐off (residence time 535 s) binding to human HSD‐1. Molecular docking revealed that clofutriben can form a ternary complex with nicotinamide‐adenine dinucleotide phosphate (NADPH) cofactor and the human HSD‐1 active site. The higher activation energy to dissociate that trimolecular interaction, compared to a bimolecular drug‐enzyme interaction characteristic of other HSD‐1 inhibitors, is posited to be responsible for the enzyme kinetic and favorable pharmacological properties of clofutriben.

2024-07-02·EXPERT OPINION ON INVESTIGATIONAL DRUGS

Investigational agents for polymyalgia rheumatica treatment: assessing the critical needs for future development

Review

作者: Padoan, Roberto ; Iorio, Luca ; Bond, Milena ; Dejaco, Christian

INTRODUCTION:

Polymyalgia rheumatica (PMR) is an inflammatory rheumatic disorder characterized by pain and stiffness in the shoulder and pelvic girdles, constitutional symptoms, and elevated acute-phase reactants. Glucocorticoids (GCs) remain the first-choice treatment for PMR, but relapses are common. Identification of steroid-sparing agents is therefore of utmost importance.

AREAS COVERED:

The efficacy of conventional immunosuppressive drugs is controversial. The use of interleukin (IL)-6 receptor inhibitors proved to be effective and safe in treating PMR patients. Currently, there are 12 ongoing clinical trials exploring potential treatments such as leflunomide, low-dose IL-2, rituximab, abatacept, secukinumab, Janus kinase inhibitors, and selective inhibitors like SPI-62 and ABBV 154.

EXPERT OPINION:

The high efficacy of IL-6 R receptor inhibitors as well as the numerous drug trials currently recruiting suggest that several therapeutic options will be available in the near future. Accurate diagnosis and early stratification of PMR patients according to the giant cell arteritis-PMR Spectrum Disease 'GPSD' and potential risk factors for relapsing disease or GC-related adverse events are crucial to identify patients who would benefit most from GC-sparing agents. The development of internationally accepted definitions for remission and relapse is urgently needed. Early referral strategies to specialist settings would improve disease stratification and personalized treatment.

2023-09-01·CLINICAL PHARMACOKINETICS

Population Target-Mediated Pharmacokinetic/Pharmacodynamic Modeling to Evaluate SPI-62 Exposure and Hepatic 11β-Hydroxysteroid Dehydrogenase Type 1 (HSD-1) Inhibition in Healthy Adults

Article

作者: Wu, Nan ; An, Guohua ; Katz, David A. ; Katz, David A

INTRODUCTION:

SPI-62 is a small-molecule 11β-hydroxysteroid dehydrogenase type 1 (HSD-1) inhibitor exhibiting complicated nonlinear pharmacokinetics (PK) in human. Previously, we developed a target-mediated drug disposition (TMDD) model to characterize the substantial nonlinear PK of SPI-62.

OBJECTIVE:

The aim of the current analysis was to perform population PK/PD analysis to further link SPI-62 exposure (i.e., PK) with its response (i.e., inhibition of hepatic HSD-1 activity) to gain a quantitative understanding of the SPI-62 dose-exposure-response relationship.

METHODS:

PK and PD data from the first-in-human (FIH) clinical trials, including single ascending dose (SAD) and multiple ascending dose (MAD) studies, were used for model development. During the model development process, the final model selection was based on biological and physiological plausibility, goodness-of-fit plots, stability of parameter estimates, and objective function value. The nonlinear-mixed effect modeling (NONMEM) software was used for both the implementation of the PK/PD model and model simulation. SPI-62 plasma levels and hepatic HSD-1 inhibition over time following various dose regimens were simulated.

RESULTS:

The final model was a two-compartment TMDD model component for SPI-62 and an inhibitory I_max model component for hepatic HSD-1 activity. The TMDD-hepatic PD model that we established adequately characterized all remarkable PK and PD behaviors of SPI-62, such as extremely low plasma exposures following the first low doses, nonlinear PK turned into linear PK after repeated low doses, and substantial and long-lasting hepatic HSD-1 inhibition following low doses. SPI-62 was estimated to bind to the target with a second-order association rate constant (K_on) of 8.43 nM^-1 h^-1 and first-order dissociation rate constant (K_off) value of 0.229 h^-1, indicating that SPI-62 binds rapidly to, and dissociates slowly from, its pharmacological target. The estimated target capacity (R_tot) of 5460 nmol corresponds to approximately 2.2 mg of SPI-62, which comports well with the dose range in which PK nonlinearity is prominent. Model simulation results reveal that a 6 mg once-daily regimen can lead to long-lasting and substantial hepatic HSD-1 inhibition.

CONCLUSIONS:

A population TMDD-PD model that explains SPI-62 nonlinear PK and hepatic HSD-1 inhibition following different dose regimens in healthy adults was successfully established. Our simulation results provide a solid foundation for model-informed development of SPI-62.

项与克洛曲本相关的新闻（医药）

2025-10-26

·美股滚雪球

资产来自中国：眼科药物新秀完成7亿元融资

今年9月，美国及欧洲的生物医药行业共发生26起融资事件，融资规模达19.62亿美元（约139.83亿人民币），其中值得关注的有：人工智能药物领域仍是热点：9月，多家AI药企业获得大额融资，如融资2.13亿美元的Odyssey ，融资2亿美元的Treeline，融资1.5亿美元的Enveda，延续了今年以来，AI领域作为Biotech行业融资热点的趋势；前沿技术路线与新靶点受到关注：9月，HSD-1抑制剂公司Sparrow、miRNA疗法公司ARTHEx、Menin抑制剂公司CHARM Therapeutics等前沿技术企业均获得大额融资。 1、Odyssey Therapeutics：GSK支持的AI新药企业 9月10日，Odyssey Therapeutics宣布完成2.13亿美元D轮融资。本轮融资的投资人包括Affinity Asset、Dimension Capital、Jeito Capital、Lightspeed Ventures、TPG Life Sciences Innovations和Wedbush Healthcare Partners等机构。 Odyssey Therapeutics是一家专注于发现、开发和商业化下一代免疫调节剂和肿瘤药物的生物技术公司，曾入选BioSpace评选出NextGen Bio“Class of 2023”榜单。目前，Odyssey正在构建一个将人工智能和机器学习相结合用于分子设计的发现引擎，一个包含针对多种氨基酸、分子胶和天然产物的专有共价库的化学平台，以及用于新靶点发现的功能基因组学平台。目前，Odyssey正在迅速推进免疫学和肿瘤学项目，重点关注那些经过有力验证但尚无市场化解决方案的靶点，包括小分子和蛋白质疗法在内的8个项目。在一级市场，Odyssey Therapeutics受到资本追捧，自2021年末成立以来筹集的总资本达到4.87亿美元： 2021年12月，Odyssey Therapeutics完成了2.18亿美元的A轮融资。A轮融资由OrbiMed Advisors与SR One共同领投，Foresite Capital、Woodline Partners、Logos Capital、HBM Healthcare、Colt Ventures、Creacion Ventures等机构跟投。 2022年10月，Odyssey Therapeutics完成了1.68亿美元的B轮融资。B轮融资由General Catalyst领投，Fidelity，GreatPoint Ventures，Walleye Capital，The Healthcare Innovation Investment Fund LLC（与SVB Securities LLC相关的投资基金）等机构跟投。 2024年12月，Odyssey Therapeutics宣布完成1.01亿美元的C轮融资。本轮融资Ascenta Capital领投，OrbiMed、GSK旗下的SR One、Foresite Capital、HBM Healthcare、Fidelity、T.Rowe Price Associates、Catalio Capital 、Alexandria Venture等机构跟投。 2、Crystalys Therapeutics：诺和诺德支持的痛风药企 9月30日，Crystalys Therapeutics宣布完成2.05亿美元的A轮融资。本轮融资由Novo Holdings、SR One、Catalys-Pacific共同领投，Perceptive Xontogeny Venture Funds、Lightstone Ventures、AN Venture、abrdn Inc.、KB Investments、Pontifax、Longwood Fund、Alexandria Venture、Wedbush Healthcare和Prebys Ventures 等机构跟投。 Crystalys成立于2022年，由著名生物医药投资机构Novo Holdings发起设立，是一家专注于代谢疾病新疗法的生物医药企业。 2024年7月，Crystalys Therapeutics从Fortress Biotech控股子公司Urica Therapeutics，引进了药物dotinurad的权益。交易条件是：Crystalys向Urica发行了35%的普通股，还授予Urica 3%的证券化特许权使用费，由Crystalys用于支付dotinurad未来的净销售额，以及有权获得Urica因dotinurad而产生的某些临床和开发成本的名义现金报销。 dotinurad是一款高选择性的尿酸盐转运蛋白1（URAT1）抑制剂，由日本企业富士在苯溴马隆的基础上进行了苯环相邻环结构改造，主要目的是降低肝毒性。dotinurad通过有效抑制肾近端小管URAT1的功能，促进尿酸从肾脏排泄，从而降低血尿酸水平。与非选择性URAT1抑制剂相比，dotinurad不影响尿酸排泄因子ABCG2和OAT1/3的功能，因此具有更高的降血尿酸效率。目前，dotinurad主要用于治疗高尿酸血症和痛风，并于2020年1月23日在日本获批上市。2024年12月10日，中国国家药品监督管理局药品审评中心（CDE）批准dotinurad上市。公司此次募集的资金，主要用于推进两项全球临床三期试验：一项将评估痛风发作次数是否减少，另一项将跟踪痛风石溶解，痛风石是晚期痛风中导致关节变形的大型尿酸晶体沉积物。公司联合创始人兼CEO James Mackay博士在制药行业拥有超过40年的开发和商业化专业知识，包括跨多个治疗领域的6种药物产品批准。在创立Crystalys Therapeutics之前，他是Aristea Therapeutics的首席执行官兼联合创始人，Ardea Biosciences的总裁兼首席执行官，亦曾在AstraZeneca担任高级管理职位超过30年。首席医疗官Nihar曾在Ardea Biosciences（被阿斯利康收购）担任多个关键职位，包括痛风特许经营的全球临床主管。在此职位上，Nihar领导了Duzallo的批准，并领导了Zurampic （lesinurad）的三期项目。此外，他曾担任Aristea Therapeutics的首席医疗官以及百时美施贵宝、罗氏和Gossamer Bio的职位。 3、Treeline Biosciences：基于AI技术的肿瘤新药公司 9月3日，Treeline Biosciences宣布完成2亿美元的A轮融资。Treeline自成立以来共募集约11亿美元。投资者包括：ARCH Venture Partners、GV、OrbiMed、KKR、亿万富翁Len Blavatnik的投资公司Access Industries旗下的AI Life Sciences、Ajax Health、Casdin Capital、T.Rowe Price Associates和Aisling Capital、Rock Springs Capital。 Treeline Biosciences成由Josh Bilenker与Jeff Engelman共同创立于2021年，是一家肿瘤药物企业。Treeline这个名字的意思是暗示科学可能性的外部边缘，那里的条件很有挑战性，但隐喻性的树木仍在生长。公司应用先进的计算工具来优化研发流程、加速项目推进。公司聚焦的治疗领域涵盖癌症、神经疾病以及自身免疫性疾病，研发形式包括小分子、蛋白降解剂，以及靶向治疗抗体偶联药物（TT-ADC）。目前，公司首批进入临床试验的药物有： TLN-121：一款用于治疗淋巴瘤的BCL6蛋白降解剂药物。BCL6是一种天然存在的蛋白，某些淋巴瘤细胞会利用它来沉默本应抑制肿瘤生长和存活的基因。TLN-121可清除癌细胞内的BCL6，同时避免因非特异作用带来的毒性，从而有望与现有标准疗法联合使用。该I期临床试验（NCT07082803）正在招募B细胞与T细胞淋巴瘤患者。 TLN-372：一款用于治疗实体瘤的广谱KRAS抑制剂药物。目前虽已有针对KRAS G12C突变的FDA批准药物，但其他KRAS突变仍缺乏有效的靶向治疗。TLN-372是一款小分子抑制剂，具有全新的化学结构，能够实现持续、深入的广谱KRAS抑制，并具备良好的药物特性。该I期临床试验将招募携带KRAS突变的实体瘤患者。 TLN-254：一款用于治疗淋巴瘤的EZH2抑制剂药物。EZH2是一种调控基因表达的蛋白，在多种癌症中存在高表达或突变。对外合作方面，2023年恒瑞医药与Treeline Biosciences达成协议，将EZH2抑制剂SHR2554（TLN-254，泽美妥司他片）项目除大中华区以外的全球范围内开发、生产和商业化权利有偿许可给Treeline公司。对价方面，恒瑞医药将获得1100万美元首付款，以及不超过4500万美元的开发里程碑款、不超过6.5亿美元的销售里程碑款，还有10%-12.5%年净销售额提成，潜在总价值可达7.06亿美元。联合创始人兼首席执行官Josh Bilenker博士是前Loxo Oncology（被礼来收购）的创始人，曾就职于FDA肿瘤办公室，离职创业前担任礼来的癌症研发总管。另一位联合创始人兼首席科学官Jeff Engelman博士来自诺华，曾任该公司的副总裁兼肿瘤学全球负责人。 Treeline目前拥有超过130位员工，研发团队的背景涵盖生物（大数据遗传学、CRISPR、细胞生物学）、化学（共价修饰、分子胶和异质双功能降解剂、变构）、结构生物学（NMR、X射线晶体学、Cryo EM）、蛋白科学和计算生物学（in silico docking、分子动力学、机器学习/AI）等，研发将优先考虑肿瘤学中已经验证过但难以成药的靶点。 4、Enveda：Baillie Gifford支持的AI药企 9月4日，Enveda宣布完成1.5亿美元D轮融资，本轮融资由Premji Invest领投，Baillie Gifford、Kinnevik、Lingotto Investment、Peakline Partners、FPV、Socium Ventures、Dimension、Level Ventures、Henry Kravis、IA Ventures和Lux Capital跟投。 Enveda成立于2019年，是一家药物发现和开发公司，使用最新的人工智能技术将自然转化为新药。该公司的专有平台首次大规模解决了天然产物药物发现中的长期障碍，包括活性分子鉴定、性质和结构优先级、药物化学的适应性以及大规模材料获取。 Enveda已经开发了世界上最大的可搜索天然化合物库，其中包含超过150万种化合物，并呈指数级增长。公司通过将质谱法与机器学习算法相结合，将必须一次检测一个分子的过程加快到一次检测10,000个分子。基于此，Enveda将化合物转化为候选开发产品的速度比行业平均速度快4倍。在融资方面，Enveda已经完成三轮融资： 2021年6月，宣布完成5100万美元的A轮融资，由Lux Capital领投，Two Sigma Ventures, Hummingbird VC, Catalio Capital, Lifeforce Capital等机构跟投； 2024年6月，Enveda Biosciences宣布完成5500万美元的B轮融资，微软、Premji Invest、Lingotto Investment、The Nature Conservancy、Kinnevik、True Ventures、FPV、Level Ventures和Jazz Venture等机构参与； 2024年11月，Enveda宣布完成1.3亿美元的C轮融资。由Kinnevik和FPV领投，Baillie Gifford、Premji Invest、Lingotto Innovation、Lux Capital、Dimension Capital、True Ventures、Cresset Partners、The nature Conservancy和Henry R.Kravis跟投。 5、Star Therapeutics：血液病领域的“hub-and-spoke”药企 9月30日，Star Therapeutics宣布完成1.25亿美元的D轮融资。本轮融资由Sanofi Ventures和Viking Global共同领投，Janus Henderson、Frazier Life、GordonMD 、Agent Capital、Blue Owl 、Catalio、Cormorant Asset、New Leaf Venture、NYBC、OrbiMed、卡塔尔投资局（QIA）、RA Capital、Redmile Group、Sofinnova 、Soleus Capital和Westlake BioPartners等机构跟投。 Star Therapeutics成立于2018年，致力于解决血液学和免疫学领域的未满足需求。公司采用“hub-and-spoke”模式运营，创建了许多投资组合公司包括去年完成8400万美元B轮融资的Electra Therapeutics，融资4000万美元的Vega Therapeutics。公司的核心药物是2022年底从Vega Therapeutics分拆出来的VGA039。VGA039是一种针对S蛋白以恢复凝血平衡的first-in-class单克隆抗体，用于治疗多种出血性疾病，第一个适应症为von Willebrand病（VWD）。公司已在所有类型的VWD患者中启动了VGA039的关键性3期临床试验，旨在评估每月一次通过皮下注射给予VGA039。 2024年美国血液学会（ASH）年会上呈现的I期VIVID-2试验结果显示，VGA039与当前获批的VWD预防疗法一致，与年出血率大幅降低相关。在该研究中，VGA039在单次皮下注射后维持了多周治疗浓度。公司创始人兼CEO Adam拥有多家生物技术公司的运营经验，并在公司建设、融资、业务发展和战略规划方面拥有广泛的专业知识。在创立Star之前，Adam是Electra Therapeutics的联合创始人兼首席执行官，亦是True North Therapeutics的首席商务官，在那里他筹集了超过1.4亿美元的私人融资，并为该公司被Bioverativ以8.25亿美元收购做出了贡献。他的经历还包括担任iPierian（被BMS以7.25亿美元收购）的副总裁和企业发展主管，以及担任波士顿咨询集团的项目负责人。 6、Ollin Biosciences：资产来自中国的眼科药物新秀 9月17日，Ollin Biosciences宣布完成1亿美元的A轮融资。本轮融资投资人有ARCH Venture Partners、Mubadala Capital和Monograph Capital。 Ollin Biosciences成立于2023年，是一家正在快速崛起的眼科药物公司。目前，公司有两款药物，均为从中国药企引入： OLN324：一款活性更高、摩尔剂量更高的VEGF/Ang2双特异性抗体，正在临床1b期开发中，用于湿性（新生血管）年龄相关性黄斑变性（wAMD）或糖尿病性黄斑水肿（DME）患者，这两种疾病分别是老年人和工作老年人视力丧失的主要原因。 OLN324的抗Ang2活性远高于目前的市场领导者faricimab，再加上较小的蛋白质形式，药物有望获得更大的靶覆盖率和延长治疗耐久性的潜力。这些特征使OLN324有可能成为差异化的一线标准护理方案。目前，公司已经完成了JADE研究中150多名wAMD或DME患者的入组，这是一项基于美国的随机1b期概念验证临床试验，旨在评估OLN324与faricimab在解剖和持久性差异化的潜在领域。该研究的顶线结果预计将在2026年第一季度公布。 OLN102：一款TSHR/IGF-1R双特异性抗体，对甲状腺眼病（TED）以及格雷夫斯病的潜在自身免疫性疾病具有最佳的疾病安全性和疗效。IGF-1R和TSHR都是TED的临床验证靶点，这两种受体之间的相互作用在TED的发病机制和临床后遗症中具有重要作用。通过抑制这两种受体，OLN102可以通过更精确的组织靶向和更广泛的治疗指数，为TED患者提供比现有药物更高的安全性和疗效。此外，OLN102有可能独特地解决TED患者潜在的自身免疫性甲状腺功能障碍（格雷夫斯病）。对外合作方面，公司不仅从信达生物引入了OLN324的海外权益，还从橙帆医药引入了OLN102的海外权益： 2025年5月，橙帆医药（ VelaVigo）宣布与Ollin Biosciences就VBS-102达成全球独家授权协议。根据协议，Ollin公司将获得该药物在大中华区以外的全球开发、生产及商业化权益，橙帆医药保留大中华区权益。 7、Sparrow Pharmaceuticals：HSD-1抑制剂药企 9月24日， Sparrow Pharmaceuticals宣布完成9500万美元的B轮融资。本轮融资由RA Capital Management和Forbion领投，OrbiMed、RiverVest和US Venture Partners跟投。 Sparrow Pharmaceuticals成立于2018年，是一家专注于开发新型皮质醇（cortisol）调节剂，用于治疗代谢和内分泌疾病。公司核心技术是基于HSD-1抑制剂平台，旨在精准调控组织内皮质醇水平，避免传统糖皮质激素受体拮抗剂（如米非司酮）带来的全身性副作用。 clofutriben是一种口服小分子HSD-1抑制剂，已完成I期和IIa期试验。在IIa期试验中，该药显示出良好的耐受性和初步疗效，在降低肝脏和脂肪组织中活性皮质醇的同时，维持了正常的循环皮质醇水平。针对2型糖尿病，该药旨在改善胰岛素敏感性、减少肝糖原生成，并潜在降低心血管风险。此外，公司还在探索clofutriben在库欣综合征和自主性皮质醇分泌（ACS）等适应症的应用。ACS适应症已获得FDA快速通道指定。公司 CEO Robert Jacks在生物制药行业拥有25年的经验。他曾创办了多家公司，筹集了3亿多美元。此前，Robert曾担任inalo Therapeutics的总裁兼首席执行官，以及Symbiomix Therapeutics（被鲁宾医药以1.5亿美元收购）的总裁、首席财务官和联合创始人。Robert此前还担任Tobira Therapeutics公司发展主管、ACT生物技术公司业务发展主管、OrbiMed Advisors驻场企业家。 8、ARTHEx Biotech：RNA疗法新秀 9月17日，ARTHEx Biotech宣布完成8700万美元的B轮融资。本轮融资由Bpifrance领投，AdBio Partners、CDTI Innovación、Columbus Venture Partners、欧洲创新委员会（EIC）、Hadean Ventures、Invivo Partners和Sound Bioventures等机构跟投。 ARTHEx Biotech由InVivo Capital、AdBio Partners和 CDTI-Innvierte 联合创办于2019年，是一家致力于miRNA在肌肉病领域应用的公司，技术源于西班牙瓦伦西亚大学。目前，公司有三款在研管线，其中最核心的管线是ATX-01。 ATX-01是一种anti-miR寡核苷酸，旨在抑制微小RNA23b（miR-23b），与调节DM1病理发生中涉及的MBNL蛋白表达有关。通过抑制miR-23b，ARTHEx已经证明ATX-01可以增加MBNL的产生，减少病灶形成和毒性DMPK mRNA。这种高度差异化的双重作用机制导致游离MBNL的显著增加，改善剪接异常，最终在动物模型中恢复功能。 2022年12月，ARTHEx宣布实现ATX-01研发项目的关键监管里程碑，ATX-01获得美国和欧洲监管机构授予治疗DM1的孤儿药资格。公司CEO Frédéric Legros在生物技术行业拥有超过15年的经验，曾担任Dynacure的首席运营官、Valneva SE的副总裁兼企业发展主管。联合创始人兼首席科学官Beatriz Llamusi曾在瓦伦西亚大学和INCLIVA健康研究所的转化基因组学实验室工作，专注于研究发病机制和潜在治疗方法的分子基础。另一联合创始人Ruben Artero博士是瓦伦西亚大学遗传学教授，紫外线转化基因组学实验室的领导者。Ruben Artero博士发现了肌盲基因，获得了10多项专利，现兼任ARTHEx科学咨询委员会的成员。欢迎加入美股滚雪球的知识星球，查看我们的行业分享以及每周更新的多个投资机会：一、我们专注的领域 1、美股生物医药股投资（核心领域，优势明显）； 2、中概股、美股云计算板块、美股互联网板块投资（重点领域，擅长中长线）； 3、美股IPO、次新股、SPAC、ETF投资（关注领域，水平中等）；二、我们提供的服务 1、每日：医药板块速评（包括板块走势、持仓标的动态、新列入观察标的情况）、每天交易机会提示（不限于医药股）； 2、每周：至少推荐一只值得交易的标的分析、建仓价格和仓位建议等； 3、每周：白马股、成长股、短期交易股、烟蒂股、次新股等五大类，七小类关注标的名单定期更新； 4、每季：核心标的和重点观察标的季报更新； 5、不定期投资逻辑分享，如逆向并购概念股、SPAC公司、中概股打新等；三、联系我们直接获取我们的研究讯息，烦请扫描下方二维码：免费微信交流群，请加微信：meigugunxueqiu 本文内容只供做参考之用，并不构成投资建议，任何人依据本文所做的投资决策所造成的一切后果自负。

2025-09-29

·创鉴汇

亚飞生物、亦立医药获超亿元支持；8家创新药公司获融资，聚焦小分子、核药、CGT等领域 | 一周融资

据创鉴汇不完全统计，上周（9月22日至9月28日）全球大健康领域共披露融资事件26起，总额超9亿美元。小分子药物研发在攻克“不可成药”靶点等前沿技术领域持续突破，上周共有3起融资。针对代谢功能障碍，Sparrow Pharmaceuticals完成9500万美元B轮融资，将推进核心管线clofutriben的2b期临床试验。Clofutriben是一款每日一次的口服HSD-1抑制剂，治疗伴有皮质醇升高的2型糖尿病患者。该群体约占难治性糖尿病患者的近半数，表现出与皮质醇升高相关的心脏代谢风险增加。Clofutriben通过抑制HSD-1，可调节关键代谢组织中细胞内皮质醇生成，旨在改善血糖控制及代谢健康，有可能补充现有的抗糖尿病疗法。肿瘤领域的小分子创新同样获得资金支持。Avenzo Therapeutics凭借其差异化的产品组合，完成6000万美元B轮融资。公司核心小分子资产包括高选择性CDK2抑制剂AVZO-021（临床1期）和CDK4抑制剂AVZO-023（临床1/2期），均针对晚期实体瘤和HR+/HER2-转移性乳腺癌。此外，公司管线还包括EGFR/HER3双特异性ADC和Nectin4/TROP2双特异性ADC等候选药物。 SEED Therapeutics完成3000万美元A3轮融资，其核心项目靶向RBM39的分子胶降解剂ST-01156同步获得美国FDA IND批准，拟用于治疗尤文肉瘤等RBM39依赖性癌症。公司专有的RITE3平台通过重编程泛素-蛋白酶体系统降解致病蛋白，专注于攻克“不可成药”靶点驱动的疾病。该公司已获得来自礼来（Eli Lilly）和卫材（Eisai）近6000万美元的股权，以及合作预付款和里程碑付款。细胞和基因疗法领域上周共获2起融资。 Ensoma完成吉利德等多家机构参投的5300万美元融资，将支持EN-374治疗X连锁慢性肉芽肿病的1/2期临床试验。EN-374是体内造血干细胞（HSC）疗法，采用病毒样颗粒（VLP）体内递送技术，对造血干细胞进行基因工程改造，旨在驱动中性粒细胞中CYBB转基因的持续表达，恢复关键免疫功能。与此同时，神拓生物获数千万元天使+轮融资，专注于开发基于新一代慢病毒载体的体内基因疗法。新型偶联技术也获资本青睐。亲合力母公司亚飞生物完成近3亿元B3轮融资。公司打造了肿瘤微环境激活的多特异性智能偶联药物平台（TMEA-XDC），已开发20余款抗肿瘤候选药物，涵盖多特异性偶联药物（ALDC/SMDC/ADC）及大分子免疫治疗药物，包括抗体、双抗TCE药物和细胞因子等。放射性核药领域，亦立医药完成超亿元天使+轮融资，不到一年已完成两起亿元级融资。公司专注于针对肿瘤及神经系统疾病研发“诊疗一体化”放射性核素药物（RDC），依托配体发现、分子优化及临床转化综合能力，推进多条管线进入临床阶段。除创新药融资外，AI驱动的数字医疗和诊断技术领域的融资同样活跃，例如，Judi Health的2.52亿美元F轮融资、Inspiren的1亿美元B轮融资等，详见下表。读者们请星标⭐创鉴汇，第一时间收到推送免责声明：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发/复制至其他平台。转发授权请在「创鉴汇」微信公众号留言联系我们。更多数据内容推荐点击“在看”，分享创鉴汇健康新动态

抗体药物偶联物临床1期临床申请免疫疗法引进/卖出

2025-09-28

·GlobeNewswire-Health Care

Sparrow Pharmaceuticals Announces $95 Million Series B Financing to Advance Targeted Therapy for Type 2 Diabetes

Sept. 24, 2025 -- Sparrow Pharmaceuticals, a targeted cardiometabolic therapeutics company, today announced the close of a $95 million Series B financing. The round was co-led by RA Capital Management and Forbion with participation from existing investors including OrbiMed, RiverVest, and US Venture Partners. In conjunction with the financing, Zach Scheiner from RA and Nanna Lüneborg from Forbion have joined Sparrow’s Board of Directors. The proceeds will support development of the company’s lead candidate clofutriben for the treatment of type 2 diabetes (T2D) with elevated cortisol (EC). Based on strong biological rationale and rising clinical recognition, Sparrow is deploying a new mechanism to target a distinct population whose poor response to current T2D treatments is linked to excess cortisol. The company has advanced clofutriben into a Phase 2b trial in T2D with EC (CAPTAIN-T2D) with results expected in 2027. “Nearly half of patients with difficult-to-control diabetes display increased cardiometabolic risk associated with elevated cortisol, yet there are no therapies approved for the vast majority of patients with T2D that address this underlying etiology,” said Frank Czerwiec, M.D., Ph.D., Chief Medical Officer. “Prospective studies confirm what endocrinologists have long believed: excess cortisol can drive hyperglycemia by increasing gluconeogenesis, decreasing glucose uptake and disposal, and decreasing insulin synthesis and sensitivity, and thereby also impair the effectiveness of standard-of-care treatments. By directly targeting this causal and multifaceted biology, clofutriben may offer new hope for patients underserved by current therapies.” “We are grateful to our investors whose support reflects strong conviction in Sparrow’s strategy and the clinical promise of clofutriben,” said Robert Jacks, President & Chief Executive Officer. “Elevated cortisol is now recognized as a key driver of disease progression and treatment resistance in millions of patients with type 2 diabetes worldwide. Our decision to focus on this patient population reflects a convergence of emerging biological insights, immense clinical opportunity, and payer and clinician receptivity. This significant financial commitment could enable the next big breakthrough in the management of metabolic syndrome.” Clofutriben is a once-daily, oral HSD-1 inhibitor that modulates intracellular cortisol production in key metabolic tissues, a novel mechanism of action that has the potential to complement existing anti-diabetic therapies. In clinical trials, the candidate has shown improved glycemic control, including in individuals with elevated cortisol, with a favorable safety and tolerability profile and no evidence of adrenal insufficiency or requirement for dose titration. As an added possible benefit, HSD-1 inhibitors, including clofutriben, have also shown potential in clinical trials for improvement in weight and body composition, lipids, blood pressure, and markers of bone metabolism, as well as sleep and quality of life, as elevated cortisol contributes to a spectrum of metabolic dysfunction beyond poor glycemic control. CAPTAIN-T2D is a double-blind, Phase 2b trial evaluating clofutriben, a selective inhibitor of 11β-hydroxysteroid dehydrogenase type-1 (HSD-1), to improve glycemic control and metabolic health in patients who have treatment-resistant type 2 diabetes (T2D) with elevated cortisol (EC). The trial will take place in two parts. Part 1 (Screening) will evaluate patients with T2D and EC risk factors for trial eligibility and the presence of EC. Participants deemed eligible from Part 1 will be randomized to either clofutriben or placebo in the dose-ranging, interventional Part 2 (Treatment). The primary endpoint is change in hemoglobin A1c (HbA1c) at 24 weeks. Exploratory endpoints include changes in body weight, blood pressure, cholesterol, and bone metabolism biomarkers. Sparrow Pharmaceuticals is a targeted cardiometabolic therapeutics company delivering improved outcomes for patients who struggle to control diabetes and related metabolic conditions with current options. The company’s lead candidate, clofutriben, is a once-daily, oral HSD-1 inhibitor in development for the millions of patients with type 2 diabetes and elevated cortisol. HSD-1 inhibition is a novel mechanism that complements existing antidiabetic agents by addressing a cause of disease progression and treatment resistance across a spectrum of metabolic dysfunction. Clofutriben has been associated with improved glycemic control while generally safe and well tolerated in multiple clinical trials without requiring dose titration. Founded in 2004, RA Capital Management is a multi-stage investment manager dedicated to evidence-based investing in public and private healthcare, life sciences, and planetary health companies. RA Capital creates and funds innovative companies, from private seed rounds to public follow-on financings, allowing management teams to drive value creation from inception through commercialization and beyond. RA Capital's knowledge engine is guided by our TechAtlas internal research division, and Raven, RA Capital's company creation team, offers entrepreneurs and innovators a collaborative and comprehensive platform to explore the novel and the re-imagined. RA Capital has over 150 employees and over $10 billion in assets under management. Forbion is a leading venture capital firm that helps companies bridge research and development through the team’s expertise in drug development and company building. Forbion is signatory to the United Nations Principles for Responsible Investment further demonstrating our philosophy that investments in companies should positively impact the health and well-being of patients. https://forbion.com/en OrbiMed is a leading healthcare investment firm, with over $17 billion in assets under management. OrbiMed invests globally across the healthcare industry, from start-ups to large multinational corporations, through private equity funds, public equity funds, and royalty/credit funds. OrbiMed seeks to be a capital provider of choice, providing tailored financing solutions and extensive global team resources to help build world-class healthcare companies. OrbiMed's team of over 130 professionals is based in New York City, London, San Francisco, Shanghai, Hong Kong, Mumbai, Herzliya, and other key global markets. https://www.orbimed.com/ RiverVest Venture Partners® is a leading venture capital firm building life science companies to address significant unmet needs of patients and deliver consistently strong results to investors. https://rivervest.com/ The USVP team foundation is grounded in four generations of high-technology venture investing. We are accomplished venture investors, former CEOs, senior executives and technology company founders that came together to take an exemplary firm into the 21st century. Together, we combine 150 years of operating experience and investment expertise with varied backgrounds in marketing, sales, operations, finance, engineering, computing, research and management. The content above comes from the network. if any infringement, please contact us to modify.

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适应症	最高研发状态	国家/地区	公司	日期
Acrocallosal综合征	临床2期	美国	Sparrow Pharmaceuticals, Inc.	2023-07-01
Acrocallosal综合征	临床2期	罗马尼亚	Sparrow Pharmaceuticals, Inc.	2023-07-01
Acrocallosal综合征	临床2期	英国	Sparrow Pharmaceuticals, Inc.	2023-07-01
ACTH非依赖性大结节样肾上腺增生症	临床2期	美国	Sparrow Pharmaceuticals, Inc.	2023-07-01
ACTH非依赖性大结节样肾上腺增生症	临床2期	罗马尼亚	Sparrow Pharmaceuticals, Inc.	2023-07-01
ACTH非依赖性大结节样肾上腺增生症	临床2期	英国	Sparrow Pharmaceuticals, Inc.	2023-07-01
肾上腺皮质腺瘤	临床2期	美国	Sparrow Pharmaceuticals, Inc.	2023-07-01
肾上腺皮质腺瘤	临床2期	罗马尼亚	Sparrow Pharmaceuticals, Inc.	2023-07-01
肾上腺皮质腺瘤	临床2期	英国	Sparrow Pharmaceuticals, Inc.	2023-07-01
库欣综合征	临床2期	美国	Sparrow Pharmaceuticals, Inc.	2022-09-01

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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


NCT05436652 (BusinessWire) 人工标引	临床2期	风湿性多肌痛	40	SPI-62/prednisolone	憲艱窪觸獵蓋艱願窪選(夢鬱繭糧窪襯鹹鑰餘衊) = A combination of prednisolone 20 mg with clofutriben showed similar efficacy as prednisolone 10 mg with placebo, based on analysis of participants’ symptoms, physical function, and biomarkers of inflammation. 艱糧淵糧鹽範窪構憲膚 (餘願鑰範積願積窪膚醖 ) 更多	积极	2024-09-19
				SPI-62 + placebo
BusinessWire 人工标引	临床2期	风湿性多肌痛	-	Prednisolone 10mg + clofutriben 6mg	鑰鏇鬱艱製範製鹹網築(廠鹽窪鹹齋鬱願艱鏇選) = Prednisolone 10mg combined with clofutriben 6mg demonstrated less efficacy compared to prednisolone 10mg administered with placebo, Prednisolone 20mg combined with clofutriben 6mg demonstrated similar efficacy as determined by symptoms, physical function, and systemic inflammation, 鏇願餘鬱醖鹽鑰膚襯鹽 (築願網餘積簾積衊簾鏇 ) 更多	积极	2024-06-15
				Prednisolone 20mg + clofutriben 6mg
SAT277 (ENDO2023)	临床1期	库欣综合征	-	HSD-1 inhibitor SPI-62	獵繭製築廠醖鹽鑰餘糧(獵遞糧範膚壓獵壓襯鬱) = 築鬱觸構齋艱糧膚鹹積夢襯網襯壓選簾鏇廠獵 (衊艱廠繭糧獵夢簾網網 ) 更多	积极	2023-10-05
				Placebo	獵繭製築廠醖鹽鑰餘糧(獵遞糧範膚壓獵壓襯鬱) = 壓範鬱襯糧鹹網襯築鹽夢襯網襯壓選簾鏇廠獵 (衊艱廠繭糧獵夢簾網網 ) 更多
RF21 \| PSAT100 (ENDO2022)	临床2期	cortisol \| ACTH \| DHEA-S ... 更多	50	SPI-62	網鹽構簾繭觸衊襯淵膚(網淵膚簾鹽繭鏇鏇簾獵) = 廠鹽醖製獵選餘獵鏇衊膚範鑰淵顧鑰糧獵憲網 (簾醖範鏇壓淵鬱膚鹽憲 ) 更多	积极	2022-11-01
				Placebo	網鹽構簾繭觸衊襯淵膚(網淵膚簾鹽繭鏇鏇簾獵) = 積艱製顧衊築鏇獵艱廠膚範鑰淵顧鑰糧獵憲網 (簾醖範鏇壓淵鬱膚鹽憲 ) 更多