A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Seamless, Adaptive, Safety, Dose-Finding, and Phase 3 Clinical Study of UX701 AAV-Mediated Gene Transfer for the Treatment of Wilson Disease

The primary objectives of this study are to evaluate the safety of single IV doses of UX701 in patients with Wilson disease, to select the UX701 dose with the best benefit/risk profile based on the totality of safety and efficacy data and to evaluate the effect of UX701 on copper regulation.

开始日期2021-09-27

申办/合作机构

Ultragenyx Pharmaceutical, Inc.

100 项与 Rivunatpagene Miziparvovec 相关的临床结果

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100 项与 Rivunatpagene Miziparvovec 相关的转化医学

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100 项与 Rivunatpagene Miziparvovec 相关的专利（医药）

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项与 Rivunatpagene Miziparvovec 相关的新闻（医药）

2024-10-12

·药明康德

使多名患者停止标准治疗的基因疗法；过半患者癌细胞完全消失的CAR-T疗法…… | CGT周报

▎药明康德内容团队编辑近期，全球细胞和基因疗法（CGT）领域迎来系列进展。最新数据显示，与标准疗法相比，嵌合抗原受体（CAR）T细胞疗法Carvykti可显著延长对来那度胺耐药的二线多发性骨髓瘤患者的总生存期（OS），也是首个能够改善此类患者OS的细胞疗法。非病毒基因疗法detalimogene voraplasmid在一项关键研究中使约70%的非肌层浸润性膀胱癌（NMIBC）患者达成完全缓解（CR）。基因疗法UX701在治疗Wilson病的1/2/3期临床试验中已成功使6名患者停止标准治疗。多款细胞治疗产品在中国获批进入临床，针对胰腺癌和肝癌。本文将节选其中部分重要进展做简单介绍，仅供读者参阅。图片来源：123RF ———✦研发进展✦——— ◇ 强生（Johnson & Johnson）和传奇生物公布了3期临床试验CARTITUDE-4的最新3年随访数据。研究结果显示，在既往至少接受过一种前期治疗的复发或来那度胺耐药的多发性骨髓瘤患者中，单次输注CAR-T细胞疗法Carvykti（西达基奥仑赛，cilta-cel）可显著延长其OS。数据显示，随访30个月时，Carvykti治疗组患者的总生存率为76%，而接受标准疗法治疗的患者总生存率为64%。与泊马度胺、硼替佐米和地塞米松（PVd）或达雷妥尤单抗、泊马度胺和地塞米松（DPd）等标准疗法相比，患者的死亡风险降低了45%。新闻稿指出，与标准疗法相比，Carvykti是首个能够改善对来那度胺耐药的二线多发性骨髓瘤患者总生存期的细胞疗法。 Carvykti是一种靶向B细胞成熟抗原（BCMA）的CAR-T细胞疗法。BCMA主要表达于恶性多发性骨髓瘤B细胞、晚期B细胞和浆细胞的表面。Carvykti的嵌合抗原受体蛋白具有两种靶向BCMA单域抗体，对表达BCMA的细胞具有高亲和力，在与表达BCMA的细胞结合后，CAR可促进T细胞活化、扩增，继而清除靶细胞。 ◇ enGene公司公布了其主打非病毒基因疗法detalimogene voraplasmid（也称为detalimogene）在高风险、对卡介苗（BCG）无应答、伴有原位癌（Cis）的非肌层浸润性膀胱癌患者中进行的LEGEND关键性试验积极结果。分析显示，高达71%的患者达成CR。患者对detalimogene的耐受性通常良好，没有患者因治疗相关不良事件而停药。 Detalimogene（曾用名EG-70）是enGene公司的DDX平台利用壳聚糖（chitosan）衍生物将DNA或RNA递送到粘膜组织中的一种新型的非病毒基因疗法。该疗法利用质粒编码两种RIG-I激动剂刺激先天免疫系统，以及IL-12刺激适应性免疫系统。通过刺激免疫系统的两个分支，detalimogene膀胱内给药在膀胱癌临床前模型中产生了显著的肿瘤消退，并诱导有效的免疫记忆。公司预计在2026年年中为detalimogene提交生物制品许可申请（BLA）。 ◇ Ultragenyx Pharmaceutical公司宣布了其在研基因疗法UX701在治疗肝豆状核变性（又名Wilson病）的1/2/3期临床试验中的最新结果。试验数据显示，UX701显示出有意义的临床活性，改善了患者的铜代谢。15名患者接受了不同剂量UX701的治疗并且随访时间超过24周，其中6名患者已经完全停止接受标准治疗（包括螯合剂和/或锌疗法）。截至今年8月，第7名患者也已经开始逐步减少标准治疗的使用。在停止标准治疗的患者中，体内铜元素水平稳定在正常的健康水平。 UX701是一款基于腺相关病毒9（AAV9）载体的基因疗法，旨在通过一次静脉输注，让患者稳定表达ATP7B蛋白。在临床前研究中，它能够让铜元素运输和排出正常化。UX701已经获得美国FDA和欧盟监管机构授予的孤儿药资格，它还获得FDA授予的快速通道资格。该公司计划进一步探索UX701的最佳剂量和优化免疫调节治疗方案，增强基因疗法的效果和效率，目标为让大部分接受治疗的患者能够停止使用标准治疗。 ◇ Immatics公布了其在研T细胞受体T细胞（TCR-T）疗法IMA203用以治疗转移性黑色素瘤患者的最新1b期临床数据，这些患者先前接受过大量治疗。分析显示，92%患者的疾病获得控制且患者的中位缓解持续时间（DOR）超过一年。根据此积极数据，Immatics预定在今年12月启动SUPRAME注册性3期试验，预计病患招募将于2026年完成，并在2026年初进行预定的中期分析。 IMA203是由Immatics专有ACTengine平台所开发的TCR-T细胞疗法，靶向由HLA-A*02呈递的黑素瘤抗原PRAME。PRAME是一种在各种实体瘤中经常表达的蛋白质，因此IMA203具潜力治疗广泛的癌症患者群体。 ◇ Aurigene Oncology公司宣布其新型BCMA靶向自体CAR-T细胞疗法Ribrecabtagene autoleucel（DRL-1801）用于治疗复发/难治性多发性骨髓瘤的1期临床试验中首批8名患者的初步结果。所有患者都接受过大量的前期治疗，中位治疗线数为5.5线。大多数患者此前也曾接受过移植手术，并在移植后出现疾病进展。结果显示，100%的患者获得了临床缓解，其中有5名（62.5%）患者获得了严格的CR。安全性方面，所有患者均未出现高级别的细胞因子释放综合征（CRS）或神经毒性。 ◇ 驯鹿生物在2024年国际骨髓瘤学会（IMS）年会上以壁报形式展示了其全人源靶向BCMA的CAR-T疗法——伊基奥仑赛注射液（商品名：福可苏）用于治疗复发难治性多发性骨髓瘤合并肾功能损伤患者的1b/2期研究FUMANBA-1的预后研究数据。结果显示，接受伊基奥仑赛注射液治疗的复发难治性多发性骨髓瘤合并肾功能受损患者也能实现快速、深入且持久的缓解，且不影响安全性。部分患者由于伊基奥仑赛输注后清除了骨髓瘤细胞，肾功能得到了改善。该研究结果为真实世界中中重度肾损伤患者的CAR-T治疗提供了参考。 ◇ 中国国家药监局药品审评中心（CDE）官网公示，镔铁生物1类新药IX001 TCR-T注射液获批临床试验默示许可，拟开发适应症为基因型为HLA-A*11:01，肿瘤抗原KRAS G12V表达为阳性的晚期胰腺癌。根据镔铁生物公开资料，这是该公司首个申报注册临床试验的在研产品，是一款针对KRAS G12V突变的TCR-T细胞疗法。该产品通过基因修饰技术，将能够特异性识别KRAS G12V突变的TCR基因序列转入T细胞，使其能够精准识别并有效杀伤胰腺癌细胞。 ◇ 臻知医学宣布，其自主研发生产的IPM001注射液新药的1期IND申请已获国家药品监督管理局（NMPA）默示许可。IPM001注射液是臻知医学研发的多抗原靶点自体工程化T细胞注射液，基于臻知医学IMMURITHMS肿瘤抗原表位筛选平台与高效抗原特异性T细胞扩增体系开发而来，可靶向数十种中国人群肝癌相关抗原与新生抗原。在前期研究者发起的临床试验（IIT）中，IPM001注射液对于经多线系统性抗肿瘤治疗失败的进展期原发性肝细胞癌患者表现出良好的临床疗效，有望成为中国晚期肝癌患者的重要治疗产品。 ◇ 基因启明宣布其细胞治疗产品GKL-006注射液用于治疗不可切除局部晚期或转移性胰腺导管腺癌的IND申请获得CDE受理。该公司还透露，GKL-006注射液用于不可切除的原发性肝细胞癌的注册性临床试验已取得了重要进展。此外，GKL-006注射液用于原发性肝细胞癌患者根治性切除术后预防高危复发的注册性临床试验正在启动中。 ———✦融资、合作、M&A✦——— 图片来源：123RF ◇ Prime Medicine公司宣布与百时美施贵宝（Bristol Myers Squibb）达成战略研发合作和授权协议，共同开发下一代T细胞疗法。这一合作将结合Prime Medicine精准的多重基因编辑能力与百时美施贵宝在开发和商业化创新细胞疗法方面的专长。Prime Medicine的PASSIGE技术平台将先导编辑与整合酶（integrase）或其它位点特异性重组酶相结合，能够将大型基因片段整合进细胞的DNA中，导致稳定的蛋白表达。PASSIGE通过非病毒载体递送，不引入DNA双链断裂和脱靶编辑，有望带来更精准和有效的基因修饰。根据协议，Prime Medicine将获得5500万美元前期付款和5500万美元的股权投资，并有资格获得超过35亿美元的里程碑付款，包括高达14亿美元的开发里程碑和超过21亿美元的商业化里程碑付款。 ◇ AviadoBio公司和安斯泰来（Astellas Pharma）宣布就AVB-101达成一项独家选择权和许可协议，安斯泰来将获得基于AAV的基因疗法AVB-101在GRN相关额颞叶痴呆（FTD-GRN）和其他潜在适应症的全球独家开发和商业化许可。AVB-101目前正在进行1/2期临床研究。根据协议，AviadoBio将获得2000万美元的股权投资和最高3000万美元的预付款。如果安斯泰来公司行使其选择权，AviadoBio公司还有资格获得21.8亿美元的许可费和里程碑付款以及特许权使用费。图片来源：123RF ◇ Purespring Therapeutics公司日前宣布，已完成1.05亿美元的B轮融资，获得资金将用于推进其基于AVV的基因疗法PS-002进入1/2期临床试验，用于治疗IgA肾病。新闻稿指出，该公司是首个利用AAV基因疗法，在肾病模型中成功直接靶向足细胞（podocyte）的公司。足细胞在约60%的肾病中受到影响，该公司的技术平台能够将转基因高效特异性递送到足细胞中，为多种肾病提供了一种具有差异性的治疗模式。 ◇ Resolution Therapeutics宣布完成6350万英镑的B轮融资。此次融资由Syncona领投，融资所得资金主要将用于推进其在研主打项目巨噬细胞疗法RTX001进入1/2期临床试验，用于治疗终末期肝病患者。所得款项的其他用途包括投资公司的生产平台，和将其管线扩展到其他炎症和纤维化适应症，包括移植物抗宿主病（GVHD）和肺纤维化。RTX001是一款潜在“first-in-class”的工程化自体巨噬细胞疗法，采用已知在巨噬细胞中表达的独特基因的组合设计，以增强肝脏的再生能力，从而实现卓越的疗效和持久性。 ◇ Cartherics公司宣布在一轮超额认购融资中筹集了超过1500万澳元的资金，该资金将用于支持其细胞疗法CTH-401用于治疗卵巢癌的临床试验，并将其管线扩展到其他疾病。CTH-401是一种CAR自然杀伤（NK）细胞产品，靶向腺癌特异性抗原TAG-72。TAG-72是一种经过验证的肿瘤标志物，在卵巢癌、胃癌、结直肠癌、前列腺癌和胰腺癌等多种实体瘤中广泛表达。在组织培养和动物模型中，CTH-401都能非常有效地杀死卵巢癌细胞。该公司计划于明年启动首次临床试验。 ◇ Somagenetix公司宣布完成1000万瑞士法郎的A轮融资，该资金将用于推进其治疗p47慢性肉芽肿病（CGD）的候选基因疗法SGX-001的临床试验。慢性肉芽肿病是一组会反复发生危及生命的感染的遗传性免疫缺陷病。这种疾病是由于基因缺陷导致吞噬细胞（一种白细胞）功能受损，从而无法杀死病原体。目前，CGD的治疗标准是干细胞移植，但只有找到免疫相容的捐赠者才能进行干细胞移植。p47-CGD基因疗法的临床前开发得到了Wyss Zurich Translational Center的支持。 ▲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] Ultragenyx Provides Update on Stage 1 Cohorts in Pivotal Phase 1/2/3 Cyprus2+ Study Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease. Retrieved October 4, 2024, from https://www.globenewswire.com/news-release/2024/10/03/2958032/0/en/Ultragenyx-Provides-Update-on-Stage-1-Cohorts-in-Pivotal-Phase-1-2-3-Cyprus2-Study-Evaluating-UX701-Gene-Therapy-for-the-Treatment-of-Wilson-Disease.html [2] 2024 IMS | CARTITUDE-4最新数据公布，CARVYKTI®显著延长二线多发性骨髓瘤患者OS. Retrieved September 30, 2024, from https://www.prnasia.com/story/462671-1.shtml [3] Detalimogene Demonstrates 71% Complete Response Rate at Any Time in Preliminary Analysis of LEGEND Pivotal Cohort. Retrieved September 26, 2024, from https://www.businesswire.com/news/home/20240926869206/en [4] Aurigene Oncology Limited Announces Promising Results of Phase 1 Study for India’s First Trial for Novel Autologous CAR-T Cell Therapy for Multiple Myeloma. Retrieved October 11, 2024, from https://www.businesswire.com/news/home/20241008755325/en/Aurigene-Oncology-Limited-Announces-Promising-Results-of-Phase-1-Study-for-India%E2%80%99s-First-Trial-for-Novel-Autologous-CAR-T-Cell-Therapy-for-Multiple-Myeloma/ [5] Immatics Announces Updated Phase 1b Clinical Data on ACTengine® IMA203 TCR-T Targeting PRAME in Melanoma Patients and Provides Update on Upcoming SUPRAME Phase 3 Trial. Retrieved October 10, 2024, from https://www.globenewswire.com/news-release/2024/10/10/2961129/0/en/Immatics-Announces-Updated-Phase-1b-Clinical-Data-on-ACTengine-IMA203-TCR-T-Targeting-PRAME-in-Melanoma-Patients-and-Provides-Update-on-Upcoming-SUPRAME-Phase-3-Trial.html [6] 中国国家药监局药品审评中心（CDE）官网. Retrieved October 9，2024, From https://www.cde.org.cn/main/xxgk/listpage/4b5255eb0a84820cef4ca3e8b6bbe20c [7] 驯鹿生物在2024年IMS年会上壁报展示伊基奥仑赛注射液（福可苏®）治疗有肾功能损伤的复发难治性多发性骨髓瘤（R/R MM）患者的预后研究. Retrieved October 11, 2024, from https://www.prnasia.com/story/462510-1.shtml [8] 臻知医学IPM001注射液正式获得国家药品监督管理局临床试验默示许可. Retrieved October 11, 2024, from https://mp.weixin.qq.com/s/qM2dy5PhhoosHudMaEeG6Q [9] 北京基因启明生物科技有限公司（以下简称“基因启明”）细胞治疗产品“GKL-006注射液”又一适应症——不可切除局部晚期或转移性的胰腺导管腺癌IND申请获得CDE受理. Retrieved October 11, 2024, from https://mp.weixin.qq.com/s/oFnvEN-xZz-LHusqehyC9g [10] Prime Medicine Announces Strategic Research Collaboration and License Agreement with Bristol Myers Squibb to Develop and Commercialize Multiple Prime Edited Ex Vivo T-Cell Therapies. Retrieved September 30, 2024, from https://investors.primemedicine.com/news-releases/news-release-details/prime-medicine-announces-strategic-research-collaboration-and [11] Resolution Therapeutics Announces £63.5 Million Series B Financing and Chair Appointment. Retrieved October 3, 2024 from https://resolution-tx.com/series-b-new-chair/ [12] Purespring Therapeutics raises £80/$105 million in a Series B financing to transform the treatment of kidney diseases. Retrieved October 10, 2024, from https://www.globenewswire.com/news-release/2024/10/09/2960230/0/en/Purespring-Therapeutics-raises-80-105-million-in-a-Series-B-financing-to-transform-the-treatment-of-kidney-diseases.html [13] Cartherics Raises Over $15 Million in Oversubscribed Private Financing Round to Advance Cell Therapy Programs. Retrieved October 11, 2024, from https://www.businesswire.com/news/home/20240930737118/en/Cartherics-Raises-Over-15-Million-in-Oversubscribed-Private-Financing-Round-to-Advance-Cell-Therapy-Programs [14] Somagenetix AG secures CHF 10 million in Series A financing to advance lentiviral gene therapy for Chronic Granulomatous Disease (CGD). Retrieved October 11, 2024, from https://www.globenewswire.com/news-release/2024/10/08/2959455/0/en/Somagenetix-AG-secures-CHF-10-million-in-Series-A-financing-to-advance-lentiviral-gene-therapy-for-Chronic-Granulomatous-Disease-CGD.html [15] Astellas and AviadoBio Announce Exclusive Option and License Agreement for Gene Therapy AVB-101 Targeting Frontotemporal Dementia and Other Indications. Retrieved October 11, 2024, from https://www.astellas.com/en/news/29501 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

细胞疗法基因疗法免疫疗法临床结果临床3期

2024-10-04

·药明康德

多名患者已停止标准治疗！基因疗法早期临床结果积极

▎药明康德内容团队编辑 Ultragenyx Pharmaceutical公司日前宣布了在研基因疗法UX701在治疗肝豆状核变性（又名Wilson病）的1/2/3期临床试验中的最新结果。试验数据显示，UX701显示出有意义的临床活性，改善了患者的铜代谢。多名获得缓解的患者已经完全停止标准疗法的使用。该公司计划进一步探索UX701的最佳剂量和优化免疫调节治疗方案，增强基因疗法的效果和效率，目标为让大部分接受治疗的患者能够停止使用标准治疗。肝豆状核变性是一种罕见的遗传病，由于ATP7B基因的突变导致ATP7B蛋白的缺失。这是一种运送铜元素的蛋白，它的功能缺失导致铜元素在肝脏和其它组织中积累。随着时间的推移，引起肝脏疾病、精神和/或神经症状，如性格改变、震颤及行走、吞咽或说话困难等，且往往不可逆。 UX701是一款基于腺相关病毒9（AAV9）载体的基因疗法，旨在通过一次静脉输注，让患者稳定表达ATP7B蛋白。在临床前研究中，它能够让铜元素运输和排出正常化。UX701已经获得美国FDA和欧盟监管机构授予的孤儿药资格，它还获得FDA授予的快速通道资格。在这项研究中，目前已有15名患者接受了不同剂量UX701的治疗并且随访时间超过24周。其中6名患者已经完全停止接受标准治疗（包括螯合剂和/或锌疗法）。截至今年8月，第7名患者也已经开始逐步减少标准治疗的使用。在停止标准治疗的患者中，体内铜元素水平稳定在正常的健康水平。安全性方面，UX701耐受性良好，没有意外的治疗伴发不良事件，没有发现显著免疫学安全事件。 Ultragenyx公司表示，在进一步优化UX701的剂量和免疫调节治疗方案后，该公司将开展随机对照研究，评估UX701与安慰剂相比，在治疗肝豆状核变性时的疗效和安全性。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料： [1] Ultragenyx Provides Update on Stage 1 Cohorts in Pivotal Phase 1/2/3 Cyprus2+ Study Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease. Retrieved October 4, 2024, from https://www.globenewswire.com/news-release/2024/10/03/2958032/0/en/Ultragenyx-Provides-Update-on-Stage-1-Cohorts-in-Pivotal-Phase-1-2-3-Cyprus2-Study-Evaluating-UX701-Gene-Therapy-for-the-Treatment-of-Wilson-Disease.html 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

孤儿药基因疗法临床结果快速通道临床研究

2024-10-03

·BioSpace

Ultragenyx Provides Update on Stage 1 Cohorts in Pivotal Phase 1/2/3 Cyprus2+ Study Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease

NOVATO, Calif., Oct. 03, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today reported that the Phase 1/2/3 Cyprus 2+ study of its UX701 gene therapy has demonstrated meaningful clinical activity as well as improvements in copper metabolism in Stage 1. Multiple responders have completely tapered off of standard-of-care treatment with responses seen in all three dose cohorts. The company plans to enroll an additional cohort in Stage 1 at a moderately increased dose and with an optimized immunomodulation regimen to enhance the efficiency and efficacy of the gene therapy, with the objective of having the majority of patients come off of standard-of-care treatment before selecting a dose for the randomized placebo-controlled stage of the study. In Stage 1, 15 patients were enrolled into three sequential dosing cohorts and followed for at least 24 weeks. Six of the patients have completely tapered off of standard-of-care treatment with chelators and/or zinc therapy, and a seventh patient has begun to taper as of the data cut-off date in August. In patients who have tapered off standard-of-care, non-ceruloplasmin bound copper (NCC) has stabilized to normal, healthy levels. In some patients, there were increases in ceruloplasmin-copper activity consistent with improved ATP7b function. From a safety perspective, UX701 has been well tolerated, with no unexpected related treatment emergent adverse events and no significant immunologic safety events as of the data cut-off. The company will be submitting a protocol amendment for the additional cohort at a moderately increased dose and with an optimized immunomodulation regimen to optimize delivery efficiency and efficacy for the AAV vector. “We are encouraged by the clinical activity we’re seeing with UX701 at this interim timepoint, with clear signals of transgene expression and improved trafficking of copper in a subset of patients currently enrolled in the study. These results, in addition to a number of patients tapering off of standard-of-care, give us confidence that this could ultimately be a novel therapy for people living with Wilson disease,” said Eric Crombez, M.D., chief medical officer at Ultragenyx. “A higher dose and optimized immunomodulation should enhance the clinical effect of this gene therapy and the ability to remove current standard-of-care in an even broader set of patients.” U.S. residents can learn more by visiting . Phase 1/2/3 Cyprus 2+ Study Design This study evaluating UX701 for the potential treatment of Wilson disease is designed with three stages. During the first stage (Stage 1), the safety and efficacy of multiple dose levels of UX701 will be evaluated and a dose will be selected for further evaluation in Stage 2. To date, 15 patients have been enrolled into three sequential dosing cohorts to evaluate doses of 5.0 x 10^12 GC/kg, 1.0 x 10^13 GC/kg, and 2.0 x 10^13 GC/kg. A fourth dosing cohort will be added and all patients in Stage 1 will be evaluated over the course of 52 weeks. In Stage 2, a new cohort of patients will be randomized 2:1 to receive the selected dose of UX701 or placebo. The primary safety and efficacy analyses will be conducted at Week 52 of Stage 2. The primary efficacy endpoints are change in 24-hour urinary copper concentration and percent reduction in standard-of-care medication by Week 52. After the initial 52-week study period, all patients will have long-term follow up in Stage 3. About Wilson Disease Wilson disease is a rare inherited disorder caused by mutations in the ATP7B gene, which results in deficient production of ATP7B, a protein that transports copper. Loss of function of this copper-binding protein results in the accumulation of copper in the liver and other tissues, most notably the central nervous system, and failure to properly distribute copper by ceruloplasmin. Patients with Wilson disease experience hepatic, neurologic and/or psychiatric problems. Those with liver disease can experience such symptoms as fatigue, lack of appetite, abdominal pain and jaundice, and can progress to fibrosis, cirrhosis, life-threatening liver failure and death. Wilson disease can be treated by reducing copper absorption or removing excess copper from the body using life-long chelation therapy, but unmet needs exist because some treated patients experience clinical deterioration and severe side effects. Wilson disease affects more than 50,000 people in commercially accessible geographies. About UX701 UX701 is an investigational AAV9 gene therapy designed to deliver stable expression of the ATP7B copper transporter following a single intravenous infusion. It has been shown in preclinical studies to normalize copper trafficking and excretion from the body. UX701 has been granted Orphan Drug Designation in the United States and European Union and Fast Track Designation in the United States. About Ultragenyx Pharmaceutical Inc. Ultragenyx is a biopharmaceutical company committed to bringing novel products to patients for the treatment of serious rare and ultrarare genetic diseases. The company has built a diverse portfolio of approved therapies and product candidates aimed at addressing diseases with high unmet medical need and clear biology for treatment, for which there are typically no approved therapies treating the underlying disease. The company is led by a management team experienced in the development and commercialization of rare disease therapeutics. Ultragenyx’s strategy is predicated upon time- and cost-efficient drug development, with the goal of delivering safe and effective therapies to patients with the utmost urgency. For more information on Ultragenyx, please visit the company’s website at: . Ultragenyx Forward-Looking Statements and Use of Digital Media Except for the historical information contained herein, the matters set forth in this press release, including statements related to Ultragenyx’s expectations and projections regarding its future operating results and financial performance, business plans and objectives for UX701, expectations regarding the tolerability and safety of UX701, and future clinical and regulatory developments for UX701 are forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements involve substantial risks and uncertainties that could cause our clinical development programs, collaboration with third parties, future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements. Such risks and uncertainties include, among others, the uncertainty of clinical drug development and unpredictability and lengthy process for obtaining regulatory approvals, the ability of the company to successfully develop UX701, the company’s ability to achieve its projected development goals in its expected timeframes, risks related to adverse side effects, risks related to reliance on third party partners to conduct certain activities on the company’s behalf , smaller than anticipated market opportunities for the company’s products and product candidates, manufacturing risks, competition from other therapies or products, and other matters that could affect sufficiency of existing cash, cash equivalents and short-term investments to fund operations, the company’s future operating results and financial performance, the timing of clinical trial activities and reporting results from same, and the availability or commercial potential of Ultragenyx’s products and drug candidates. Ultragenyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to the business of Ultragenyx in general, see Ultragenyx’s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) on August 2, 2024, and its subsequent periodic reports filed with the SEC. In addition to its SEC filings, press releases and public conference calls, Ultragenyx uses its investor relations website and social media outlets to publish important information about the company, including information that may be deemed material to investors, and to comply with its disclosure obligations under Regulation FD. Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyx’s Investor Relations website ( ) and LinkedIn website ( ). Contacts Ultragenyx Pharmaceutical Inc. Investors Joshua Higa +1-415-475-6370 ir@ultragenyx.com Media Carolyn Wang +1-415-225-5050 media@ultragenyx.com

孤儿药基因疗法快速通道临床1期免疫疗法

100 项与 Rivunatpagene Miziparvovec 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
肝豆状核变性	临床前	美国	Ultragenyx Pharmaceutical, Inc.	2021-08-14

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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


NCT04884815 (Biospace) 人工标引	临床1/2期	肝豆状核变性	15	UX701	(製廠築壓鬱繭鑰鹹繭夢) = In patients who have tapered off standard-of-care, non-ceruloplasmin bound copper (NCC) has stabilized to normal, healthy levels. In some patients, there were increases in ceruloplasmin-copper activity consistent with improved ATP7b function. 範膚糧鏇選鬱鬱簾淵夢 (觸範繭鹽淵壓鑰壓膚製 ) 更多	积极	2024-10-03