A Phase I/ II, open-label, randomized, 2-arm study, designed to evaluate the safety and explore efficacy of the study drug in development for the treatment of MPS IIIA patients.

开始日期2023-10-04

申办/合作机构

JCR Pharmaceuticals Co., Ltd.

100 项与 Posnafusp alfa 相关的临床结果

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100 项与 Posnafusp alfa 相关的转化医学

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100 项与 Posnafusp alfa 相关的专利（医药）

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项与 Posnafusp alfa 相关的新闻（医药）

2024-11-03

·药明康德

“癌症之王”疾病控制率达80%的共价抑制剂；使淀粉样蛋白几乎全部清除的阿尔茨海默病疗法…… | 一周盘点

▎药明康德内容团队编辑本期看点 1. KRAS G12D共价小分子抑制剂RMC-9805用于经治胰腺导管腺癌（PDAC）患者的早期临床结果亮眼，客观缓解率（ORR）达30%，疾病控制率（DCR）为80%。 2. 潜在“first-in-class”合成致死疗法IDE397在一项1期临床试验中展现积极结果，在甲硫腺苷磷酸化酶（MTAP）缺失的尿路上皮癌（UC）和非小细胞肺癌（NSCLC）患者中的DCR为93%。 3. 罗氏（Roche）用于治疗阿尔茨海默病的在研抗体trontinemab在一项1b/2a期临床试验中，使大多数接受较高剂量的患者大脑中的淀粉样蛋白斑块在28周时几乎全部清除。药明康德内容团队整理 RMC-9805：公布1/1b期临床试验数据 Revolution Medicines公司公布了其小分子抑制剂RMC-9805在经治胰腺导管腺癌患者中的初步积极数据。RMC-9805是一款潜在“first-in-class”的口服KRAS G12D突变选择性共价抑制剂。RMC-9805通过和KRAS G12D以及与伴侣蛋白cyclophilin A蛋白之间形成稳定的高亲和力三复合物，能够破坏KRAS G12D（ON）与RAF激酶等下游效应物的相互作用，抑制KRAS G12D（ON）的下游信号传导。在一项多中心、开放标签、剂量递增和剂量扩展的1/1b期研究RMC-9805-001中，截至2024年9月2日的数据，在推荐的2期剂量（每日一次1200 mg和每日两次600 mg）下，至少接受14周RMC-9805治疗患者的ORR达30%（n=12），DCR为80%（n=32）。此外，RMC-9805显示出良好的安全性特征，并在不同剂量水平上普遍展现良好的耐受性。在每日服用1200 mg RMC-9805的患者中（n=99），超过10%的最常见治疗相关不良事件（TRAEs），包括胃肠道相关毒性（如恶心、腹泻和呕吐）以及轻微皮疹，主要为1级且持续时间有限。 ▲RMC-9805展现初步治疗PDAC的效果（图片来源：参考资料[2]） IDE397：公布1期临床试验数据 IDEAYA Biosciences公司公布了其在研疗法IDE397在MTAP缺失的尿路上皮癌和非小细胞肺癌患者中进行的1期临床试验扩展数据。IDE397是一款潜在“first-in-class”甲硫氨酸腺苷转移酶2α（MAT2A）小分子抑制剂。MTAP缺失的肿瘤因为肿瘤代谢途径的变化对它尤其敏感。MTAP缺失患者群体估计占所有实体瘤的15%，包括大约15%的非小细胞肺癌、28%的食道癌、26%的膀胱癌、和10%的胃癌患者。此次公布的数据显示，在27名可评估患者中，1例患者获得完全缓解（CR），8例患者获得部分缓解（PR）。这9例患者的缓解均已通过RECIST 1.1评估确认，包括4例UC患者（其中1例为CR）和5例NSCLC患者。此外，16例患者疾病稳定。在27名可评估患者中，疾病控制率约为93%。 ▲IDE397的1期临床试验扩展数据（图片来源：IDEAYA Biosciences公司官网） Trontinemab：公布1b/2a期临床试验数据罗氏公布了其在研抗体trontinemab用以治疗轻度至中度阿尔茨海默病（AD）患者的1b/2a期试验积极结果。Trontinemab是一款运用罗氏大脑穿梭（brain shuttle）技术的靶向淀粉样蛋白单克隆抗体。它将靶向淀粉样蛋白的单克隆抗体与可以和转铁蛋白受体相结合的蛋白域融合，可以有效协助抗体穿越血脑屏障，在用药3-6个月后可以快速有效地清除大脑中的淀粉样蛋白沉积。此次公布的结果显示，两种较高剂量（1.8 mg/kg与3.6 mg/kg）的trontinemab在第12周时便导致患者大脑淀粉样蛋白水平迅速且显著地降低。在28周时，大多数接受较高剂量的患者大脑中的淀粉样蛋白斑块几乎全部清除。此外，trontinemab不仅降低了淀粉样蛋白水平，还降低了阿尔茨海默病的其他生物标志物的水平，包括脑脊液当中的总tau蛋白与pTau181等。研究人员认为总体上trontinemab具有良好的安全性，并指出观察到的水肿/渗出相关的淀粉样蛋白相关成像异常（ARIA-E）病例数量非常有限。 ▲在28周时，大部分接受高剂量抗体治疗患者的大脑淀粉样蛋白斑块几乎全部清除（图片来源：参考资料[5]） IDE275（GSK959）：IND申请获得FDA许可 IDEAYA Biosciences公司宣布其候选小分子IDE275（GSK959）向美国FDA递交的IND申请已获批准，将启动1期临床试验。IDE275是一种由IDEAYA与GSK合作发现的潜在“first-in-class”的小分子Werner解旋酶（WRN）抑制剂。在临床前研究中，IDE275在微卫星不稳定性高（MSI-H）生物标志物背景下展示了强烈的选择性合成致死效应，包括在源自结直肠、子宫内膜和胃癌的MSI-H细胞异体移植模型（CDX）和患者异体移植模型（PDX）中，IDE275单药治疗诱导了肿瘤消退。预计IDE275的1期临床试验将于2024年第四季度启动。GSK是IND申请的赞助方，并计划在1期临床试验中将IDE275作为单药以及与PD-1抑制剂联用治疗MSI-H肿瘤患者。 NM32：启动1期临床试验 Numab Therapeutics宣布启动其靶向受体酪氨酸激酶样孤儿受体1（ROR1）和T细胞受体相关抗原CD3的新一代三特异性免疫肿瘤治疗药物NM32的1期临床试验。NM32是潜在“first-in-class”ROR1靶向半衰期增强型T细胞接合器。ROR1是一种肿瘤相关抗原，在实体瘤和血液恶性肿瘤中广泛表达。NM32的分子量较小，与较大的免疫球蛋白G分子（目前CD3接合器的标准形式）相比，能够更有效地渗透肿瘤。NM32的第三个结合部分以血清白蛋白为靶点，延长了半衰期，方便实现两周一次的给药频率。 CNP-106：1b/2a期临床试验完成首例患者给药 COUR Pharmaceuticals公司宣布，其用于治疗全身性重症肌无力（gMG）患者的候选疗法CNP-106的1b/2a期概念验证临床试验已完成首例患者给药。CNP-106是一种使用可生物降解的纳米颗粒封装乙酰胆碱受体（AChR）的产品。gMG主要由体内产生的抗AChR抗体引起。这些抗体会阻断或破坏控制肌肉收缩的重要神经递质乙酰胆碱的神经信号传递给AChR，导致肌肉无法正常收缩。随着病情的发展，gMG可能会引发更严重的健康问题，比如肌无力危象、胸腺肿瘤、甲状腺功能异常以及其他自身免疫性疾病。CNP-106旨在下调或抑制针对AChR的T细胞和抗体水平，从而帮助改善患者的肌肉功能。 BBO-10203：1期临床试验完成首例患者给药 BridgeBio Oncology Therapeutics公司宣布，其用于治疗晚期实体瘤的候选小分子BBO-10203的1期临床试验已完成首例患者给药。BBO-10203是一种潜在“first-in-class”、口服生物可利用的RAS：PI3Kα断路器（breaker）。BBO-10203不直接抑制PI3Kα的催化活性，而是通过选择性地共价结合到PI3Kα的RAS结合域（RBD），阻断RAS和PI3Kα之间的相互作用，从而抑制RAS驱动肿瘤中的PI3Kα-AKT信号传导。这种选择性抑制不受限于PI3Kα和RAS的突变状态。在临床前模型中，即使在个位数纳摩尔浓度下也能完全抑制RAS驱动的pAKT信号。此外，在接受BBO-10203治疗的临床前动物模型中未观察到高血糖现象。BBO-10203有望为患有RAS和PI3Kα驱动的恶性肿瘤患者提供具有临床意义的治疗选择。 ▲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] Revolution Medicines Presents Initial Data from RMC-9805 Monotherapy Study in Patients with Advanced Pancreatic Ductal Adenocarcinoma. Retrieved October 25, 2024 from https://ir.revmed.com/news-releases/news-release-details/revolution-medicines-presents-initial-data-rmc-9805-monotherapy [2] Pancreatic Cancer Update. Retrieved October 25, 2024 from https://ir.revmed.com/static-files/87b6dcb4-94c9-4e79-9d8c-d21f2bb7aaaf [3] IDEAYA Announces Positive Interim Phase 1 Expansion Data of IDE397 in MTAP-Deletion Urothelial and Lung Cancer as Late-Breaker Oral Presentation at EORTC-NCI-AACR 2024. Retrieved October 25, 2024, from https://www.prnewswire.com/news-releases/ideaya-announces-positive-interim-phase-1-expansion-data-of-ide397-in-mtap-deletion-urothelial-and-lung-cancer-as-late-breaker-oral-presentation-at-eortc-nci-aacr-2024-302286733.html [4] IDEAYA Biosciences Announces IND-Clearance for Werner Helicase Development Candidate IDE275 (GSK959) for a Phase 1 Study in MSI-High Solid Tumors. Retrieved October 25, 2024, from https://www.prnewswire.com/news-releases/ideaya-biosciences-announces-ind-clearance-for-werner-helicase-development-candidate-ide275-gsk959-for-a-phase-1-study-in-msi-high-solid-tumors-302287911.html [5] LATEST INTERIM RESULTS FROM THE BRAINSHUTTLE™ AD STUDY, A PHASE IB/IIA STUDY OF TRONTINEMAB IN PEOPLE WITH ALZHEIMER’S DISEASE. Retrieved October 30, 2024 from https://medically.roche.com/global/en/neuroscience/ctad-2024/medical-material/CTAD-2024-presentation-kulic-latest-interim-results-from-brainshuttle-ad-study-pdf.html [6] Numab Therapeutics Announces Initiation of Phase 1 Clinical Study of NM32 Program in Patients with Solid Tumors. Retrieved November 1, 2024, from https://www.globenewswire.com/news-release/2024/10/30/2971606/0/en/Numab-Therapeutics-Announces-Initiation-of-Phase-1-Clinical-Study-of-NM32-Program-in-Patients-with-Solid-Tumors.html [7] COUR Pharmaceuticals Doses First Patient in Phase 1b/2a Clinical Trial Evaluating CNP-106 in Generalized Myasthenia Gravis. Retrieved November 1, 2024, from https://www.globenewswire.com/news-release/2024/10/30/2971607/0/en/COUR-Pharmaceuticals-Doses-First-Patient-in-Phase-1b-2a-Clinical-Trial-Evaluating-CNP-106-in-Generalized-Myasthenia-Gravis.html [8] BridgeBio Oncology Therapeutics (BBOT) Announces First Patient Dosed with First-in-Class BBO-10203, a RAS:PI3Kα Breaker, in the Phase 1 BREAKER-101 Trial for Advanced Solid Tumors. Retrieved November 1, 2024, from https://www.businesswire.com/news/home/20241030409443/en/BridgeBio-Oncology-Therapeutics-BBOT-Announces-First-Patient-Dosed-with-First-in-Class-BBO-10203-a-RASPI3K%CE%B1-Breaker-in-the-Phase-1-BREAKER-101-Trial-for-Advanced-Solid-Tumors/ [9] Thetis Pharmaceuticals Announces Positive Initial Results From Phase 1a Study of TP-317 in Healthy Subjects. Retrieved November 1, 2024, from https://www.businesswire.com/news/home/20241031890209/en/Thetis-Pharmaceuticals-Announces-Positive-Initial-Results-From-Phase-1a-Study-of-TP-317-in-Healthy-Subjects [10] AiCuris Initiates First-in-Human Phase 1 Trial with Antiviral Agent AIC468 to Address BK Virus Infections in Kidney Transplant Recipients. Retrieved November 1, 2024, from https://www.aicuris.com/index.php/fuseaction/download/lrn_file/20241031_aicuris-pr_aic468-fpi_final.pdf [11] Disc Medicine Presents Positive Data from SAD Cohorts of a Phase 1b Trial in Patients with Chronic Kidney Disease (CKD) and Anemia at the 2024 American Society of Nephrology (ASN) Kidney Week. Retrieved November 1, 2024, from https://www.globenewswire.com/news-release/2024/10/25/2969602/0/en/Disc-Medicine-Presents-Positive-Data-from-SAD-Cohorts-of-a-Phase-1b-Trial-in-Patients-with-Chronic-Kidney-Disease-CKD-and-Anemia-at-the-2024-American-Society-of-Nephrology-ASN-Kidn.html [12] Jubilant Therapeutics Inc. announces First Patient Dosing in Global Clinical Trials for JBI-802, a CoREST Inhibitor and JBI-778, a PRMT5 Inhibitor. Retrieved November 1, 2024, from https://www.prnewswire.com/news-releases/jubilant-therapeutics-inc-announces-first-patient-dosing-in-global-clinical-trials-for-jbi-802-a-corest-inhibitor-and-jbi-778-a-prmt5-inhibitor-302287204.html [13] Maze Therapeutics Reports Positive First-in-Human Data from Phase 1 Healthy Volunteer Clinical Trial Evaluating MZE829 as a Potential Treatment for APOL1 Kidney Disease (AKD). Retrieved November 1, 2024, from https://mazetx.com/maze-therapeutics-reports-positive-first-in-human-data-from-phase-1-healthy-volunteer-clinical-trial-evaluating-mze829-as-a-potential-treatment-for-apol1-kidney-disease-akd/ [14] Kymera Therapeutics Announces Dosing of First Participant in Phase 1 Clinical Trial of KT-621, a First-in-Class Oral STAT6 Degrader, for the Treatment of TH2 Immuno-Inflammatory Diseases. Retrieved November 1, 2024, from https://investors.kymeratx.com/news-releases/news-release-details/kymera-therapeutics-announces-dosing-first-participant-phase-1 [15] NAYA Biosciences Announces Initiation of Phase 1/2a Clinical Trial for its GPC3-targeted NK Engager Bispecific Antibody in Patients with Hepatocellular Carcinoma. Retrieved November 1, 2024, from https://www.globenewswire.com/news-release/2024/10/24/2968749/0/en/NAYA-Biosciences-Announces-Initiation-of-Phase-1-2a-Clinical-Trial-for-its-GPC3-targeted-NK-Engager-Bispecific-Antibody-in-Patients-with-Hepatocellular-Carcinoma.html [16] Praxis Bioresearch Obtains FDA Clearance On the IND Application for Its Lead Candidate PRX-P4-003 In Alzheimer’s Disease Apathy. Retrieved November 1, 2024, from https://www.globenewswire.com/news-release/2024/10/29/2970716/0/en/Praxis-Bioresearch-Obtains-FDA-Clearance-On-the-IND-Application-for-Its-Lead-Candidate-PRX-P4-003-In-Alzheimer-s-Disease-Apathy.html [17] Nektar Announces Publication in Nature Communications of Results from Phase 1b Studies of Rezpegaldesleukin in Two Inflammatory Skin Diseases. Retrieved November 1, 2024, from https://www.prnewswire.com/news-releases/nektar-announces-publication-in-nature-communications-of-results-from-phase-1b-studies-of-rezpegaldesleukin-in-two-inflammatory-skin-diseases-302289469.html [18] Aro Biotherapeutics Announces Start of First in Human Study with ABX1100, a Novel First in Class Centyrin-siRNA Conjugate in Development for Pompe Disease. Retrieved November 1, 2024, from https://www.arobiotx.com/aro-bio-announces-start-of-first-in-human-study-with-abx1100 [19] Bitterroot Bio Completes Enrollment in Phase 1 First-in-Human Study of BRB-002 in Healthy Volunteers. Retrieved November 1, 2024, from https://brbio.com/bitterroot-bio-completes-enrollment-in-phase-1-first-in-human-study-of-brb-002-in-healthy-volunteers/ [20] ProMIS Neurosciences Presents Positive Data from PMN310 Phase1a Clinical Trial at the 17th Clinical Trials on Alzheimer’s Disease Conference. Retrieved November 1, 2024, from https://www.globenewswire.com/news-release/2024/10/30/2971622/0/en/ProMIS-Neurosciences-Presents-Positive-Data-from-PMN310-Phase1a-Clinical-Trial-at-the-17th-Clinical-Trials-on-Alzheimer-s-Disease-Conference.html [21] Alterome Therapeutics Announces First Patient Dosed in Phase 1 Study of ALTA2618 in AKT1 E17K-mutant solid tumors (AKTive-001). Retrieved November 1, 2024, from https://www.businesswire.com/news/home/20241030626999/en/Alterome-Therapeutics-Announces-First-Patient-Dosed-in-Phase-1-Study-of-ALTA2618-in-AKT1-E17K-mutant-solid-tumors-AKTive-001 [22] Ajax Therapeutics Announces First Patient Dosed in Phase 1 Clinical Trial Evaluating AJ1-11095, a First-in-Class Type II JAK2 inhibitor, for the Treatment of Myelofibrosis. Retrieved November 1, 2024, from https://www.ajaxtherapeutics.com/ajax-therapeutics/ajax-therapeutics-announces-first-patient-dosed-in-phase-1-clinical-trial-evaluating-aj1-11095-a-first-in-class-type-ii-jak2-inhibitor-for-the-treatment-of-myelofibrosis/ [23] KITHER BIOTECH ANNOUNCES PHASE 1 CLINICAL STUDY OF KIT2014, A NOVEL INHALED PEPTIDE THERAPY FOR CYSTIC FIBROSIS. Retrieved November 1, 2024, from https://www.prnewswire.com/news-releases/kither-biotech-announces-phase-1-clinical-study-of-kit2014-a-novel-inhaled-peptide-therapy-for-cystic-fibrosis-302290900.html [24] Medera Announces Completion of Cohort A and Initiation of Dosing in Cohort B of MUSIC-HFpEF, a Phase 1/2a Clinical Trial Evaluating First-In-Human Gene Therapy SRD-002 for Heart Failure with Preserved Ejection Fraction. Retrieved November 1, 2024, from https://www.prnewswire.com/news-releases/medera-announces-completion-of-cohort-a-and-initiation-of-dosing-in-cohort-b-of-music-hfpef-a-phase-12a-clinical-trial-evaluating-first-in-human-gene-therapy-srd-002-for-heart-failure-with-preserved-ejection-fraction-302285414.html [25] Lexeo Therapeutics Announces Positive Interim Data for LX1001, First-Ever Gene Therapy to Impact the Underlying Genetic Cause of APOE4-Associated Alzheimer’s Disease, at the Clinical Trials on Alzheimer’s Disease (CTAD) Conference. Retrieved November 1, 2024, from https://ir.lexeotx.com/news-releases/news-release-details/lexeo-therapeutics-announces-positive-interim-data-lx1001-first [26] NKGen Biotech Presents Phase 1/2a Troculeucel Data in Alzheimer’s Disease at the 17th Annual Clinical Trials on Alzheimer’s Disease (CTAD) Conference. Retrieved November 1, 2024, from https://www.globenewswire.com/news-release/2024/10/29/2970775/0/en/NKGen-Biotech-Presents-Phase-1-2a-Troculeucel-Data-in-Alzheimer-s-Disease-at-the-17th-Annual-Clinical-Trials-on-Alzheimer-s-Disease-CTAD-Conference.html [27] Regeneration Biomedical Presents Data on First Cohort from an Ongoing Phase I Clinical Trial of Stem Cell Therapy Delivered Directly into the Brains of Patients with Alzheimer’s Disease. Retrieved November 1, 2024, from https://www.globenewswire.com/news-release/2024/10/29/2970530/0/en/Regeneration-Biomedical-Presents-Data-on-First-Cohort-from-an-Ongoing-Phase-I-Clinical-Trial-of-Stem-Cell-Therapy-Delivered-Directly-into-the-Brains-of-Patients-with-Alzheimer-s-Di.html [28] AIMDBIO，TOP1 'FGFR3 ADC' “美国 1 期 IND 批准”. Retrieved November 1, 2024, from http://www.biospectator.com/view/news_view.php?varAtcId=23469 [29] Bexion Pharmaceuticals, Inc. Announces Publication of Phase 1 Data for BXQ-350 in Advanced Solid Tumors. Retrieved November 1, 2024, from https://www.prnewswire.com/news-releases/bexion-pharmaceuticals-inc-announces-publication-of-phase-1-data-for-bxq-350-in-advanced-solid-tumors-302293463.html [30] JCR Pharmaceuticals Initiates First Dosing in Phase I Clinical Trial of JR-441 for Mucopolysaccharidosis Type IIIA. Retrieved November 1, 2024, from https://www.businesswire.com/news/home/20241031198164/en/JCR-Pharmaceuticals-Initiates-First-Dosing-in-Phase-I-Clinical-Trial-of-JR-441-for-Mucopolysaccharidosis-Type-IIIA 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

临床2期临床1期

2024-10-31

·PipelineReview

JCR Pharmaceuticals Initiates First Dosing in Phase I Clinical Trial of JR-441 for Mucopolysaccharidosis Type IIIA

HYOGO, Japan I October 31, 2024 I JCR Pharmaceuticals Co., Ltd. (TSE 4552; “JCR”) announced the initiation of the first patient dosing in Japan in the Phase I clinical trial of JR-441, an investigational enzyme replacement therapy for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA, also known as Sanfilippo syndrome type A). JR-441 is a proprietary recombinant heparan N-sulfatase capable of crossing the blood-brain barrier (BBB). MPS IIIA is a rare genetic disorder characterized by severe central nervous system (CNS) symptoms, for which there is currently no approved treatment. Preclinical studies have demonstrated the potential of JR-441 to address CNS-related symptoms associated with MPS IIIA. This open-label, multicenter, single-arm study aims to assess the safety profile, biological effect, and pharmacokinetic profile of JR-441 in patients aged 1 to under 18 years with MPS IIIA. “MPS IIIA has been one of the most significant challenges in the treatment of CNS symptoms,” said Dr. Kimitoshi Nakamura, Professor of Pediatrics at the Graduate School of Medical Sciences, Kumamoto University, and the Medical Expert of the study. “This novel therapeutic approach represents a new era for managing the condition. We have been eagerly awaiting the opportunity to offer this treatment, and I am hopeful that its effectiveness will be confirmed in the clinical setting, ultimately improving the daily lives of both patients and their families.” JR-441 received Orphan Drug Designation from both the European Commission (EC) and the U.S. Food and Drug Administration (FDA), with a Phase I/II trial underway in Germany, which began in 2023 (JR-441-101, NCT06095388 ). For more details on the Japanese Phase I trial, visit the Clinical Research Submission and Disclosure System (JR-441-JP11, jRCT2071240053 ). About Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A) Mucopolysaccharidosis type IIIA, or Sanfilippo syndrome type A, is an autosomal recessive disease caused by pathogenic mutations in the SGSH gene, encoding a lysosomal enzyme involved in the degradation of heparan sulfate. The accumulation of heparan sulfate in several types of cells of the body, especially in the central nervous system in the brain, results in severe neurological deterioration, cognitive impairment, mild somatic involvement, and shortened lifespan. About JCR Pharmaceuticals Co., Ltd. JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceuticals company that is expanding possibilities for people with rare and genetic diseases worldwide. We continue to build upon our 49-year legacy in Japan while expanding our global footprint into the US, Europe, and Latin America. We improve patients’ lives by applying our scientific expertise and unique technologies to research, develop, and deliver next-generation therapies. Our approved products in Japan include therapies for the treatment of growth disorder, MPS II (Hunter syndrome), Fabry disease, acute graft-versus host disease, and renal anemia. Our investigational products in development worldwide are aimed at treating rare diseases including MPS I (Hurler, Hurler-Scheie and Scheie syndrome), MPS II, MPS IIIA and B (Sanfilippo syndrome type A and B), and more. JCR strives to expand the possibilities for patients while accelerating medical advancement at a global level. Our core values – reliability, confidence, and persistence – benefit all our stakeholders, including employees, partners, and patients. For more information, please visit https://www.jcrpharm.co.jp/en/site/en/ . SOURCE: JCR Pharmaceuticals

临床1期孤儿药

2024-09-03

·Business Wire

JCR Pharmaceuticals Announces Completion of Clinical Trial Notification Process in Japan for Phase I Study of JR-441 for Mucopolysaccharidosis Type IIIA

HYOGO, Japan--( BUSINESS WIRE )-- JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced today the completion of the regulatory review by the Pharmaceuticals and Medical Devices Agency (PMDA) for the clinical trial notification for the Phase I study of JR-441 in individuals with mucopolysaccharidosis type IIIA (MPS IIIA; Sanfilippo syndrome type A). JR-441 is a recombinant form of heparan N-sulfatase that is developed using JCR’s proprietary J-Brain Cargo ® blood-brain barrier (BBB)-penetrating technology. JR-441 addresses a significant unmet medical need by crossing the BBB and targeting the neurological symptoms of MPS IIIA within the central nervous system. "MPS IIIA is a rare and life-threatening lysosomal storage disorder for which there are currently no approved treatment options for individuals living with this devastating disease, and there is a dire need to address the neurological signs and symptoms of MPS IIIA,” said Shin Ashida, Chairman and President of JCR Pharmaceuticals. “We are pleased that the PMDA’s review of the Phase I study plan is complete, and that clinical development program is progressing. We are committed to continuing to advance the JR-441 clinical development program with the goal of meeting the unmet medical needs of this patient community. We look forward to sharing updates on the program when they are available.” The European Commission (EC) and the U.S. Food and Drug Administration (FDA) granted JR-441 orphan drug designation (ODD) for the treatment of MPS IIIA. JR-441 is currently being studied in an ongoing Phase I/II clinical trial in Germany (JR-441-101, NCT06095388 ), apart from this Japanese Phase I study. The impact of this announcement on JCR's consolidated financial results for this fiscal year (ending March 2025) is expected to be minor. About Mucopolysaccharidosis Type IIIA (Sanfilippo Syndrome Type A) Mucopolysaccharidosis type IIIA, or Sanfilippo syndrome type A, is an autosomal recessive disease caused by pathogenic mutations in the SGSH gene, encoding a lysosomal enzyme involved in the degradation of heparan sulfate. The accumulation of heparan sulfate in several types of cells of the body, especially in the central nervous system in the brain, results in severe neurological deterioration, cognitive impairment, mild somatic involvement, and shortened lifespan. About JCR Pharmaceuticals Co., Ltd. JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceuticals company that is expanding possibilities for people with rare and genetic diseases worldwide. We continue to build upon our 49-year legacy in Japan while expanding our global footprint into the US, Europe, and Latin America. We improve patients’ lives by applying our scientific expertise and unique technologies to research, develop, and deliver next-generation therapies. Our approved products in Japan include therapies for the treatment of growth disorder, MPS II (Hunter syndrome), Fabry disease, acute graft-versus host disease, and renal anemia. Our investigational products in development worldwide are aimed at treating rare diseases including MPS I (Hurler, Hurler-Scheie and Scheie syndrome), MPS II, MPS IIIA and B (Sanfilippo syndrome type A and B), and more. JCR strives to expand the possibilities for patients while accelerating medical advancement at a global level. Our core values – reliability, confidence, and persistence – benefit all our stakeholders, including employees, partners, and patients. For more information, please visit https://www.jcrpharm.co.jp/en/site/en/ . Cautionary Statement Regarding Forward-Looking Statements This document contains forward-looking statements that are subject to known and unknown risks and uncertainties, many of which are outside our control. Forward-looking statements often contain words such as “believe,” “estimate,” “anticipate,” “intend,” “plan,” “will,” “would,” “target” and similar references to future periods. All forward-looking statements regarding our plans, outlook, strategy and future business, financial performance and financial condition are based on judgments derived from the information available to us at this time. Factors or events that could cause our actual results to be materially different from those expressed in our forward-looking statements include, but are not limited to, a deterioration of economic conditions, a change in the legal or governmental system, a delay in launching a new product, impact on competitors’ pricing and product strategies, a decline in marketing capabilities relating to our products, manufacturing difficulties or delays, an infringement of our intellectual property rights, an adverse court decision in a significant lawsuit and regulatory actions. This document involves information on pharmaceutical products (including those under development). However, it is not intended for advertising or providing medical advice. Furthermore, it is intended to provide information on our company and businesses and not to solicit investment in securities we issue. Except as required by law, we assume no obligation to update these forward-looking statements publicly or to update the factors that could cause actual results to differ materially, even if new information becomes available in the future.

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适应症	最高研发状态	国家/地区	公司	日期
黏多醣贮积症III型	临床2期	德国	JCR Pharmaceuticals Co., Ltd.	2023-10-04
IIIA型糖原贮积病	临床申请	日本	JCR Pharmaceuticals Co., Ltd.	2024-09-03