Effects of RO7204239 on Insulin Sensitivity, Muscle Composition and Function in Participants Living With Type 2 Diabetes Mellitus and Overweight or Obesity: A Double-blind, Randomized, Placebo-controlled Study

The main purpose of this study is to assess the effect of RO7204239 on insulin sensitivity versus placebo in participants with T2DM and obesity or overweight.

开始日期2025-08-14

申办/合作机构

Roche Holding AG

NCT06965413

/ Recruiting临床2期

A Randomized, Double-blind, Placebo-controlled Phase 2 Trial to Assess Efficacy, Safety, and Tolerability of RO7204239 in Combination With Tirzepatide in Participants With Obesity or Overweight With At Least One Weight-related Comorbidity

The main aim of the study is to assess the effect of RO7204239 in combination with tirzepatide, compared to placebo in combination with tirzepatide, on body weight loss after 48 weeks of treatment in adults with obesity or overweight with at least one weight-related comorbidity, but without diabetes mellitus (DM). The study comprises of a 4-week screening period; a 48-week core treatment period, where all participants will receive tirzepatide as background treatment and will be randomized to one of the 4 treatment arms; a 24-week treatment extension period, where participants will stop treatment with tirzepatide and a 24-week post-treatment follow-up (FU) period.

开始日期2025-05-05

申办/合作机构-

ISRCTN10993304

/ Recruiting临床1期

A single-and multiple-dose, open-label, Phase I study to investigate the safety, tolerability, pharmacokinetics, pharmacodynamics, and immunogenicity of subcutaneously administered RO7204239 in adult participants with high body weight

开始日期2024-05-10

申办/合作机构-

100 项与 Emugrobart 相关的临床结果

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100 项与 Emugrobart 相关的转化医学

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100 项与 Emugrobart 相关的专利（医药）

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项与 Emugrobart 相关的文献（医药）

2023-01-01·The Journal of toxicological sciences

Preclinical <i>in vitro</i> evaluation of immune suppression induced by GYM329, Fc-engineered sweeping antibody

Article

作者: Honda, Masaki ; Ching, Tim Jang ; Mishima, Masayuki ; Okuda, Momoko ; Iwata, Yoshika ; Takeiri, Akira ; Katada, Hitoshi ; Doi, Yoshiaki ; Harada, Asako

Fc-engineering is commonly used to improve the therapeutic potency of antibody (Ab) treatments. Because FcγRIIb is the only inhibitory FcγR that contains an immunoreceptor tyrosine-based inhibition motif (ITIM), Fc-engineered Abs with enhanced binding affinity to FcγRIIb might provide immune suppression in clinical contexts. GYM329 is an anti-latent myostatin Fc-engineered Ab with increased affinity to FcγRIIb which is expected to improve muscle strength in patients with muscular disorders. Cross-linking of FcγRIIb by immune complex (IC) results in phosphorylation of ITIM to inhibit immune activation and apoptosis in B cells. We examined whether the IC of Fc-engineered Abs with enhanced binding affinity to FcγRIIb causes phosphorylation of ITIM or B cell apoptosis using GYM329 and its Fc variant Abs in human and cynomolgus-monkey (cyno) immune cells in vitro. IC of GYM329 with enhanced binding affinity to human FcγRIIb (×5) induced neither ITIM phosphorylation nor B cell apoptosis. As for GYM329, FcγRIIb should work as an endocytic receptor of small IC to sweep latent myostatin, so it is preferable that GYM329 induces neither ITIM phosphorylation nor B cell apoptosis to prevent immune suppression. In contrast, IC of myo-HuCy2b, the Ab with enhanced binding affinity to human FcγRIIb (×4), induced ITIM phosphorylation and B cell apoptosis. The result of the present study demonstrated that Fc-engineered Abs with similar binding affinity to FcγRIIb had different effects. Thus, it is important to also investigate FcγR-mediated immune functions other than binding to fully understand the biological effects of Fc-engineered Abs.

Scientific reports3区 · 综合性期刊

Novel myostatin-specific antibody enhances muscle strength in muscle disease models

3区 · 综合性期刊

ArticleOA

作者: Ueyama, Atsunori ; Ohmine, Ken ; Igawa, Tomoyuki ; Shida-Kawazoe, Meiri ; Muramatsu, Hiroyasu ; Shimizu, Yuichiro ; Hayashi, Madoka ; Katada, Hitoshi ; Ban, Nobuhiro ; Kuramochi, Taichi ; Kitamura, Hidetomo ; Hori, Yuji ; Okuda, Momoko ; Nezu, Junichi ; Haraya, Kenta ; Nonaka, Tatsuya ; Ruike, Yoshinao ; Miyano-Nishizawa, Rie ; Kitazawa, Takehisa ; Honda, Masaki ; Kawabe, Yoshiki ; Hattori, Kunihiro

Abstract:

Myostatin, a member of the transforming growth factor-β superfamily, is an attractive target for muscle disease therapy because of its role as a negative regulator of muscle growth and strength. Here, we describe a novel antibody therapeutic approach that maximizes the potential of myostatin-targeted therapy. We generated an antibody, GYM329, that specifically binds the latent form of myostatin and inhibits its activation. Additionally, via “sweeping antibody technology”, GYM329 reduces or “sweeps” myostatin in the muscle and plasma. Compared with conventional anti-myostatin agents, GYM329 and its surrogate antibody exhibit superior muscle strength-improvement effects in three different mouse disease models. We also demonstrate that the superior efficacy of GYM329 is due to its myostatin specificity and sweeping capability. Furthermore, we show that a GYM329 surrogate increases muscle mass in normal cynomolgus monkeys without any obvious toxicity. Our findings indicate the potential of GYM329 to improve muscle strength in patients with muscular disorders.

项与 Emugrobart 相关的新闻（医药）

2025-08-19

·医药观澜

多款新药冲刺上市！曾无药可医，如今创新疗法如何为SMA患者带来重生曙光？

编者按：每年8月是国际SMA关爱月。脊髓性肌萎缩症（SMA）是一种遗传性神经肌肉病，以肌无力和肌萎缩为主要临床特征。该病致残、致死率高，一度“无药可治”。随着产业界的不懈努力，近年多款新药接连问世，为患者带来“新生”。为了实现更高的治疗甚至治愈目标，科学界还在探索更多的创新疗法。作为全球医药及生命科学行业创新的赋能者，药明康德多年来也一直在支持包括SMA在内的各类罕见病新药的开发，以一体化、端到端的CRDMO服务平台，助力全球合作伙伴加速新药问世、造福病患。脊髓性肌萎缩症（SMA）是一种遗传性神经肌肉病，病症表现为进行性肌肉萎缩、无力和瘫痪。根据今年3月份的文献报道，全球SMA发病率约为每12万人中10例。根据严重程度或发病年龄、基因型等可将SMA分为四种亚型（SMA1-4），最严重的SMA1型为宫内发病，历史数据显示该群体预期寿命不足两年。其他基因型的患者则分别在不同年龄发病。 SMA是由于运动神经元存活基因1（SMN1）基因中出现基因变异，导致无法生成足够的运动神经元生存（SMN）蛋白，而SMN蛋白对维持运动神经元的存活至关重要。得益于疾病机制的确定，SMA新药研发领域在过去10年里不断取得重大突破。目前美国FDA已经批准了3款针对SMA的创新疗法，包括反义寡核苷酸疗法Spinraza、基因疗法Zolgensma、小分子药物Evrysdi。这些疾病修饰疗法改变了以往SMA只能采取姑息治疗的格局，为患者及其家庭带来重生希望。同时为实现更好的治疗效果，当下全球范围内还有数十款SMA新药在研。今年上半年，SMA新药研发领域也迎来多项重要进展，多款新药冲刺或获批上市：图片来源：123RF 2月，罗氏（Roche）宣布FDA已批准其Evrysdi（risdiplam）片剂的新药申请，用于治疗SMA患者。Evrysdi是一种靶向SMN2基因的小分子mRNA剪接调节剂，可提高全身多系统SMN蛋白水平, 缓解SMA患者的症状。该产品的口服液剂型已经于2020年8月获FDA批准治疗2个月以上婴幼儿和成人SMA患者，本次获批的口服药片剂型则为患者用药提供了更多用药便利。《新英格兰医学杂志》还于今年2月发表了一项研究，介绍了全球首例在SMA患儿出生之前，在母体子宫内使用Evrysdi治疗的案例。这名患儿在出生两年多后，尚未观察到任何可识别的SMA病征。研究人员表示，这一研究结果显示了产前治疗SMA的可行性。 3月，Scholar Rock公司宣布其在研SMA抗体疗法apitegromab的生物制品许可申请（BLA）已经获得FDA受理并授予优先审评资格，预计在今年9月22日之前完成审评。Apitegromab通过选择性结合骨骼肌中肌生成抑制蛋白（myostatin）的前体及潜在形式抑制其激活，代表了一种创新的肌肉靶向疗法。myostatin是调节骨骼肌生长和强度的关键负调节因子。同样在3月，诺华（Novartis）宣布其采用鞘内注射的onasemnogene abeparvovec（OAV101 IT），在针对2岁至<18岁SMA患者群体的3期临床研究中显示出积极的安全性和疗效结果。诺华已经在美国和欧盟递交该产品的监管申请。该疗法利用腺相关病毒载体将SMN1转基因递送到运动神经元中，可直接针对疾病的遗传根本原因。此前，采用一次性静脉注射的OAV101（即Zolgensma）已经于2019年5月获FDA批准上市，治疗2岁以下的SMN1双等位基因突变的SMA患者。 6月，渤健（Biogen）公布了反义寡核苷酸（ASO）疗法salanersen治疗SMA的1期临床研究中期分析结果，研究对象为一组既往接受过基因治疗但临床状态仍不理想的SMA患儿。结果显示，该产品可显著降低神经退行性标志物神经丝轻链（NfL）的水平，治疗6个月后基线NfL浓度较高的患者平均NfL下降幅度达70%，且这一效果可持续维持至一年。渤健正与计划推进该产品进入注册性临床研究阶段。这些进展提示SMA治疗有望在近期迎来新突破，患者将有望迎来更多治疗选择。图片来源：123RF 治疗SMA，寡核苷酸疗法率先突破在SMA治疗领域，寡核苷酸药物是率先获批的药物类型。2016年底，SMA治疗迎来重要里程碑：Spinraza获批上市，成为FDA批准的首款治疗SMA的创新疗法。这款由渤健和Ionis Pharmaceuticals联合开发的靶向SMN2基因的ASO类疾病修饰治疗药物，通过鞘内注射直接作用于脊髓运动神经元，通过与SMN2基因转录形成的mRNA相结合，改变RNA的剪接过程，从而增加正常SMN蛋白的表达量。该疗法于2018年获得了国际盖伦奖（Prix Galien USA Awards）最佳生物技术产品奖。于2019年公布的长期疗效数据显示，经过长达45.1个月的随访和数据分析，Spinraza能够帮助婴儿达到疾病自然发展历史上无法达到的运动能力里程碑，其中包括100%的儿童能够不需协助坐起来，88%的儿童能够自主行走。除了Spinraza，全球范围内还有一些寡核苷酸疗法正在被探索治疗SMA，靶点主要包括SMN，还有一些近年来被发现为SMA调控基因的靶点（比如NCALD）。公开资料显示，对于包括SMA在内的神经退行性疾病，治疗困难的主要原因之一在于难以直接靶向引发疾病的遗传因素。ASO疗法通过与编码蛋白的mRNA或mRNA前体结合，促使mRNA降解或调控其前体的剪接过程，为精准靶向神经退行性疾病的遗传根源提供了有效途径。迄今为止，FDA批准的11款ASO疗法中，有4款用于治疗神经退行性疾病，显示出ASO疗法在延缓甚至逆转神经疾病病程方面的巨大潜力。需要提到的是，天然的寡核苷酸极易被人体内的酶降解，因此在临床应用中面临较大挑战。近年来，研发界对ASO的化学修饰显著提高了药物的亲和力、效力和稳定性。然而，这些复杂的修饰工艺也为ASO药物的合成带来了新的技术难题。作为医药创新的赋能者，药明康德旗下WuXi TIDES的寡核苷酸平台针对性地提供从药物发现到商业化生产的一体化CRDMO服务。药物发现阶段的合成服务支持高通量库合成和定制合成，涵盖多种类型的寡核苷酸及其单体、连接子、配体和偶联物，助力合作伙伴快速推进临床前研究。同时，可无缝衔接到工艺开发阶段，放大到任何规模（mmol到mol），充分满足从临床前、临床到商业化阶段的需求。在某项目中，WuXi TIDES各团队高效协作，帮助合作伙伴在12个月内完成了先导化合物的优化、工艺开发及GMP生产，同时帮助合作伙伴基于数据进行快速决策，选出综合效力、稳定性和开发潜力俱佳的ASO候选化合物，为后续临床研究奠定了坚实基础。随着越来越多的ASO药物进入临床开发，这种产业协同模式将成为加快研发步伐的重要推动力。数十款SMA新药在研，多类创新疗法受关注除了ASO寡核苷酸药物，全球范围内还有数十款SMA在研新药，其中少数几款已经进入后期临床研究阶段。这些药物的类型主要涵盖小分子、抗体和基因治疗药物等。在小分子药物领域，有一些新靶点正在被探索SMA治疗潜力。比如NMD Pharma在研的氯离子通道（ClC-1）抑制剂NMD670正在针对SMA开展2期临床研究，临床前研究表明，抑制ClC-1可以增强神经肌肉传递，最终增强骨骼肌功能。在抗体药物领域，肌肉靶向疗法正在被广泛关注。Biohaven公司开发的肌生成抑制蛋白（myostatin）拮抗剂和激活素1型受体抑制剂taldefgrobep alfa目前已进入3期临床研究阶段；罗氏在研的抗myostatin单抗GYM329（RG-70240）联合利司扑兰治疗SMA的3期临床试验正在进行中。此外，来凯医药在研的Act RIIA / IIB双靶点单克隆抗体LAE123也在临床前研究中显示治疗SMA的潜力，研究表明ActRIIA是肌肉增长和脂肪减少的主要调控靶点。突破罕见病新药研发困局，一体化CRDMO平台提供赋能支持 SMA是少数幸运的、已拥有针对性疗法的罕见病类型之一。而放眼更广泛的罕见病新药开发领域，挑战依然存在。根据世界卫生组织统计，全球约有6000多种罕见病，患者总数超3亿人，目前仅不到5%的罕见病有治疗方法。罕见病药物的研发面临患者群体小、研发成本高、技术难度大等诸多挑战，需要创新生物医药生态圈的共同努力。作为创新的推动者、客户信赖的合作伙伴以及全球健康产业的贡献者，药明康德多年来通过其一体化、端到端的CRDMO新药研发平台，通过技术创新、高效协同和全链条服务，为合作伙伴的罕见病新药研发提供强有力的支持。罕见病药物的研发往往始于对疾病机制的深入理解。公开信息显示，药明康德生物学业务早在2016年就上线了罕见病平台，专注于罕见病药物研发的早期阶段。该平台覆盖了罕见病药物研发的全路线，提供包括罕见病体内外模型构建，以及体内、外的药理药效研究一站式服务，涵盖了血液疾病、代谢类疾病、免疫类疾病和精神类疾病等研发领域，助力各类罕见病药物的早期研发。图片来源：123RF 药明康德测试事业部也持续赋能客户开展罕见病新药研发IND工作和非临床研究工作。以神经系统类罕见病疗法“穿透血脑屏障”这一行业挑战为例，在这一领域，药明康德DMPK自2009年起开始进行中枢神经系统相关研究，拥有超过15年的临床前中枢神经系统药物研发经验，建立了针对这类药物体外血脑屏障通透性评价的特色且准确性较高的“漏斗”模型。通过完善的体外、体内测试平台，药明康德DMPK能够快速、准确、高效地对中枢神经系统药物进行全方位药代动力学相关评价，筛选出具有良好的脑通透性及稳定性的中枢神经系统候选药物。根据早前公开信息，该平台已完成包括常规小分子、治疗性蛋白、寡核苷酸等新分子实体类型项目百余项，成功助力全球客户多个中枢神经系统药物进入临床阶段。罕见病药物的研发不仅需要科学突破，还需要高效的工艺开发能力，以应对临床阶段的复杂挑战。药明康德子公司合全药业在这一领域展现了强大的赋能能力。基于质量源于设计（QbD）的理念，合全药业的研发团队能够快速推进药物工艺的开发和安全放大，在保障产品质量的同时，显著降低成本。这些平台共同构成了药明康德在罕见病药物研发领域的强大引擎，助力合作伙伴加速新药研发进程，为全球罕见病患者带来更多的治疗选择。药明康德的赋能不仅限于技术支持，还体现在其全球化的合作网络中。通过与全球药企、生物技术公司和研究机构的紧密合作，药明康德持续助力罕见病新药走向世界。在人类与疾病的漫长斗争中，科学创新始终是照亮希望的火炬。SMA从无药可用到多款突破性疗法相继问世的十年历程，生动诠释了新药研发如何重塑罕见病患者的生命轨迹。我们期待更多罕见病将迎来治疗曙光，更多的罕见病不再“罕治”！参考资料： [1]Nikunja Kishor Mishra.(2025) Spinal Muscular Atrophy (SMA): Treatment strategies, challenges and future prospects.Pharmacological Research - Reports.Doi：https://doi.org/10.1016/j.prerep.2025.100031 版权说明：本文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。转载授权或其他合作需求，请联系wuxi_media@wuxiapptec.com。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。

优先审批寡核苷酸上市批准信使RNA基因疗法

2025-08-06

·BioPlus

Pfizer等6家MNC：2025 Q2管线调整，哪些项目被砍，哪些新增？

作者：Medicina 近期，MNC企业相继发布了其第二季度的财务报告，笔者据此对各企业的管线变动情况进行了整理。 01 Pfizer Programs Discontinued：1. PF-06425090, Prophylactic vaccine – protein subunit, Primary Clostridioides difficile (C. Difficile) Infection （艰难梭菌感染）2. Vepdegestrant (ARV-471) + VERZENIO® (abemaciclib), ER-targeting PROTAC® protein degrader + CDK4/6 inhibitor, ER+/HER2- 2L Metastatic Breast Cancer (VERITAC 4)3. Vedegestrant (ARV-471) + Atimocilib ER-targeting PROTAC® protein degrader + CDK4 inhibitor, ER+/HER2- 1L Metastatic Breast Cancer4. VELSIPITY™ (etrasimod), S1P inhibitor, Crohn's Disease5. PF-06954522, Glucagon-like peptide 1 receptor (GLP-1R) agonist, Chronic Weight Management6. PF-07293893, AMPKγ3 activator, Heart Failure7. PF-07820435, STING agonist, Advanced Solid Tumors8. felmetatug vedotin (PF-08046048) (B7H4V), B7H4-ADC, Advanced Solid Tumors 02 BMS Removed 1. Phase I：IL2-CD25 2. Phase II：afimetrotan（TLR7/8拮抗剂） New to Phase I 1. BMS-986500 in Solid Tumors (有人推测是CDK2 inhibitor?) 2. BMS-986517 in Solid Tumors 3. CD19 HD Allo CAR T in AID 4. RYZ401 in Solid Tumors 03 GSK Removed from Phase III 1. belrestotug (GSK4428859): Anti-TIGIT antibody, Non-small cell lung cancer 2. ibrexafungerp (GSK458448): Antifungal glucan synthase inhibitor, Invasive candidiasis Removed from Phase II 1. GSK3437949: Recombinant protein, adjuvanted, Malaria fractional dose 2. GSK3536852: GMMA, Shigella 3. sanfetrin cilexetil (GV118819): Serine beta lactamase inhibitor, Tuberculosis Removed from Phase I 1. GSK3536867: Bivalent conjugate, Salmonella (typhoid + paratyphoid A) New to Phase I GSK5458514: PSMAxCD3 T cell engaging bispecific antibody, Prostate Cancer 04 Sanofi Removed from Phase 2 1. amitelimab – HS (OX40L单抗，曾报道疗效不佳) 2. SAR444656 – AD/HS（SAR444656也称KT-474，为IRAK4降解剂；曾报道赛诺菲优先考虑临床前IRAK4降解剂KT-485，而不是推进KT-474，新候选药物KT-485在临床前显示出更高的选择性和活性，可能安全性更好） New in Phase 1 1. SAR448501 – inflammatory indication（原研Dren Bio，Dectin-1/CD20） 05 AZ Removed from Phase I 1. AZD5851: GPC3 CAR-T hepatocellular carcinoma (但AZD7003 China GPC3 CAR-T，hepatocellular carcinoma/squamous NSCLC，仍然在管线中，处于I期临床) 2. AZD6422: CLDN18.2 CAR-T solid tumors 3. NT-125: autologous, fully-individualised, multi-specific TCR-T targeting neoantigens solid tumors Removed from Phase II 1. Additional indication: zibotentan/dapagliflozin: endothelin A receptor （ETA）antagonist/SGLT2 inhibitor liver cirrhosis 2. Life cycle management: Ultomiris, anti-C5 mAb proliferative lupus nephritis New to Phase I 1. AZD0240: KRAS G12D armoured TCR-T solid tumours 2. AZD1613: inflammation and metabolism modulation kidney disease (anti-PAPPA单抗) 3. AZD4248: cellular health cardiorenal disease 4. AZD4360: CLDN18.2 ADC solid tumours (这是另一款新的CLDN18.2-ADC;此前从美雅珂/康诺亚获得的CLDN18.2 MMAE ADC AZD0901 现在处于临床III期) 5. AZD4954: Lp(a) inhibitor dyslipidaemia 6. Additional indication: AZD5492 CD20 TITAN T-cell engager systemic lupus erythematosus （CD20/CD8/TCR） 7. volrustomig avDLVF-RCC02: PD-1/CTLA4 bispecific mAb 1L advanced clear cell renal cell carcinoma (ccRCC) 06 Roche Removed from phase I （4 NMES）: 1. RG6279 eciskafusp alfa + T - solid tumors (RG6279为IL-2/PD1抗体融合蛋白) 2. RG6457 WRN covalent inhibitor - solid tumors 3. RG6414 USP1 inhibitor - solid tumors 4. RG7921 NME - RVO Removed from phase III： 1NME: 1. RG6058 tiragolumab + T - stage III unresectable 1L NSCLC （tiragolumab 为anti-TIGIT单抗） 2 AIs: 1. RG6058 tiragolumab + T + Avastin - 1L HCC （tiragolumab 为TIGIT单抗） 2. RG7601 Veneclexta + azacitidine - 1L MDS （Veneclexta为Bcl-2抑制剂） New to phase I (2 NMEs): 1. RG6505 PanRAS inhibitor - solid tumors（2025年3月，启动了I期临床，NCT06884618） 2. RG6327 NME - geographic atrophy （2025年5月，启动了I期临床NCT06961370） New to phase II（1 AI）： 1. RG6237 emugrobart (GYM 329) - obesity（2025年5月启动II期临床，NCT06965413，RG6237 +Tirzepatide vs. Tirzepatide ）欢迎大家加入BioPlus微信交流群，共同探讨。 BioPlus 微信交流群申请入群申请联系人：Lisa 邮箱：lisa.li@bp-bioplus.com 电话：18662346610（同微信）入群申请，请先添加Lisa微信，并分享名片入群画像：Biotech/药厂研发/BD、投资机构、临床医生等入群福利：不定时行业资料包分享拓展阅读 BioPlus-海外寻找资产系列： 1.AACR复盘：9家CDH17-ADC数据对比 2.自免小众靶点APRIL，正在悄悄火起来了！ 3. STEAP1，会成为ADC与TCE的下一个爆款靶点么？ BioPlus国际大会跟踪系列： 1. 2025 ASCO：ADC、双抗/多抗，有哪些看点？（附一览表） 2. 2025 AACR 双抗与ADC有哪些看点？ 3. 小细胞肺癌领域，下一步将如何“破局”，ADC or TCE？ 4. ELCC 讨论：NSCLC的ADCs最新进展 5. 未来ADC发展方向：新靶点，新载荷，双靶点/双表位... 6. 2025 ASCO 重点项目临床数据：有哪些精彩看点？ 7. 2025 ASCO 重点项目临床数据 BioPlus靶点/管线研究系列： 1. “待爆”：CDH17 ADC项目大比拼，谁将脱颖而出? 2. PD-1激动剂在自免疾病项目数据盘点：失败项目的“坑”与启示 3. 上临床就能卖？DLL3 ADC这么好卖？ 4. 胰腺癌三大热门靶点：CLDN18.2、EGFR、RAS 5. TSLP/SCF双抗，下一个自免待爆靶点组合？ 6. GPC3靶点临床屡败，TCE能改写格局么？ 7. 亮相AACR，曾被临床终止的ADC靶点Ly6E，能否卷土重来？ 8. 最高18.45亿美元！赛诺菲为何重金买了两款TL1A双抗？ 9. 2025 Q1：BMS、Sanofi和Roche管线调整，哪些项目被砍？ 10. 现场！2025AACR：KRAS抑制剂的百花齐放 11. IL-17小分子抑制剂，能否杀出重围？ 12.全球首款：华东医药申报MUC17 ADC药物 13.荨麻疹 first-in-class双抗提交临床试验申请 BioPlus技术平台深入剖析系列： 1. TCE 免疫共刺激信号之选：CD2，一颗冉冉升起的新星 2. BD爆发前夜！6家TCE Probody技术平台深度盘点 3. TCE Probody：Amgen携重磅技术“破局“，改写行业格局？ 4. 有人放弃，有人前行！自免TCE全球管线大盘点 5. Immunocore将TCR疗法照进现实，向自免疾病迈进 6. B细胞清除免疫重置：SLE等疾病长效缓解终成现实！ 7. 国内50家CD3平台序列来源汇总 8. CD28共刺激最终章？ZW209 DLL3/CD3/CD28 三抗剑指 Amgen 9. TCE 结构之争落幕：行业用行动给出 “标准答案” 10. CD3 VHH猴交叉：重要吗？ 11.Genentech创新前沿：口服VHH抗体，靶向IL-23R 12.AbbVie专利公开：靶向PSMA/STEAP1 双抗ADC BioPlus人物专访： 1. 专访肖亮：卖青苗不一定是坏事，这是中国Biotech长成参天大树的必经之路 2. 专访王刚，恺佧生物这些年的“出海”之旅？

免疫疗法临床1期细胞疗法抗体药物偶联物疫苗

2025-06-18

·Firstwordpharma

Scholar Rock says its drug helps Zepbound users retain more muscle

Scholar Rock said Wednesday its experimental drug apitegromab helped preserve muscle mass in patients taking Eli Lilly's weight-loss treatment Zepbound (tirzepatide), addressing mounting concerns that powerful new obesity medications may cause patients to lose too much lean tissue alongside unwanted fat.Apitegromab is a monoclonal antibody that inhibits myostatin activation by selectively binding the pro- and latent forms of myostatin in skeletal muscle. Scholar Rock is already developing it for spinal muscular atrophy (SMA), which is the company's primary focus and where the drug succeeded in a Phase III trial last year. However, the company is one of a handful of others like Regeneron Pharmaceuticals and Biohaven that are testing whether drugs originally designed for muscle-wasting diseases can also help maintain muscle mass in people who want to lose weight."GLP therapies have been an effective and important innovation for individuals living with obesity and cardiometabolic disorders; however, these treatments can result in substantial loss of lean muscle mass for patients, leading to unwanted health risks," remarked Akshay Vaishnaw, Scholar Rock's president of R&D.The Phase II proof-of-concept study enrolled 102 patients with obesity or who were overweight with weight-related conditions. Patients receiving apitegromab with Zepbound lost 12.3% of their body weight after 24 weeks versus 13.4% for the arm that took Zepbound and a placebo.However, the combo arm achieved 85.3% of total weight reduction coming from fat mass, with lean mass accounting for just 14.6% of the weight lost. The placebo group saw 69.5% of the total weight reduction coming from fat and 30.2% from lean mass.The results reported Wednesday validate "our hypothesis that our platform of highly selective myostatin inhibitors has the potential to support healthier weight loss for millions of patients on GLP therapies," Vaishnaw said.From a safety perspective, Scholar Rock said its drug demonstrated an "encouraging safety profile," with no serious adverse events or discontinuations considered to be related to apitegromab treatment. When asked for more details during a company call, Vaishnaw said, "I think no news is good news… Essentially the safety profile looks very much like whatever we're seeing, in the balance between the arms, associated with the tirzepatide effect on the GI tract, which are well known for GLPs."The biotech plans to advance a next-generation subcutaneous anti-myostatin antibody called SRK-439, with an IND application expected in the second half of 2025. "We remain focused on preparing for the launch of apitegromab, and following its potential approval in SMA, we look forward to studying it in a range of neuromuscular diseases," Vaishnaw said.Regeneron recently reported that its anti-myostatin antibody trevogrumab helped preserve lean mass in a Phase II study with Novo Nordisk's Wegovy (semaglutide), while Roche began testing its myostatin antibody RO7204239 in a Phase II study with Zepbound last month.Industry observers also await closely watched data from Eli Lilly's bimagrumab, acquired via its $2-billion Versanis buy in 2023, and which is set to be presented at the upcoming American Diabetes Association (ADA) meeting. The drug targets activin type II receptors to inhibit myostatin and activin, and is being evaluated in a Phase II study, with or without Zepbound. For more, see – Vital Signs: Regeneron stacks the deck for myostatin targeting ahead of Eli Lilly's ADA reveal.

临床2期临床结果并购临床3期

100 项与 Emugrobart 相关的药物交易

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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
脊髓性肌萎缩	临床3期	美国	Hoffmann-La Roche, Inc.	2022-06-02
脊髓性肌萎缩	临床3期	日本	Hoffmann-La Roche, Inc.	2022-06-02
脊髓性肌萎缩	临床3期	澳大利亚	Hoffmann-La Roche, Inc.	2022-06-02
脊髓性肌萎缩	临床3期	比利时	Hoffmann-La Roche, Inc.	2022-06-02
脊髓性肌萎缩	临床3期	加拿大	Hoffmann-La Roche, Inc.	2022-06-02
脊髓性肌萎缩	临床3期	克罗地亚	Hoffmann-La Roche, Inc.	2022-06-02
脊髓性肌萎缩	临床3期	意大利	Hoffmann-La Roche, Inc.	2022-06-02
脊髓性肌萎缩	临床3期	荷兰	Hoffmann-La Roche, Inc.	2022-06-02
脊髓性肌萎缩	临床3期	波兰	Hoffmann-La Roche, Inc.	2022-06-02
脊髓性肌萎缩	临床3期	葡萄牙	Hoffmann-La Roche, Inc.	2022-06-02