Phase I/II, open-label, multicenter, multinational (Japan, Brazil and US),designed to evaluate the safety, pharmacokinetics and explore the efficacy for the treatment of mucopolysaccharidosis type I (MPS I).

开始日期2020-09-01

申办/合作机构

JCR Pharmaceuticals Co., Ltd.

100 项与 Lepunafusp alfa 相关的临床结果

登录后查看更多信息

100 项与 Lepunafusp alfa 相关的转化医学

登录后查看更多信息

100 项与 Lepunafusp alfa 相关的专利（医药）

登录后查看更多信息

项与 Lepunafusp alfa 相关的文献（医药）

2024-03-01·Molecular therapy : the journal of the American Society of Gene Therapy

α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A phase 1/2 trial

Article

作者: Ikeda, Toshiaki ; Takasao, Naoko ; Hirato, Tohru ; Minami, Kohtaro ; Moriuchi, Hiroaki ; Kubo, Toru ; Harmatz, Paul ; Kawashima, Satoshi ; Kira, Ryutaro ; Sato, Yuji ; Hamazaki, Takashi ; Sonoda, Hiroyuki ; Tanizawa, Kazunori ; Martins, Ana Maria ; Schmidt, Mathias ; So, Sairei ; Giugliani, Roberto

Mucopolysaccharidosis type I (MPS I) causes systemic accumulation of glycosaminoglycans due to a genetic deficiency of α-L-iduronidase (IDUA), which results in progressive systemic symptoms affecting multiple organs, including the central nervous system (CNS). Because the blood-brain barrier (BBB) prevents enzymes from reaching the brain, enzyme replacement therapy is effective only against the somatic symptoms. Hematopoietic stem cell transplantation can address the CNS symptoms, but the risk of complications limits its applicability. We have developed a novel genetically modified protein consisting of IDUA fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa; JR-171), which has been shown in nonclinical studies to be distributed to major organs, including the brain, bringing about systemic reductions in heparan sulfate (HS) and dermatan sulfate concentrations. Subsequently, a first-in-human study was conducted to evaluate the safety, pharmacokinetics, and exploratory efficacy of JR-171 in 18 patients with MPS I. No notable safety issues were observed. Plasma drug concentration increased dose dependently and reached its maximum approximately 4 h after the end of drug administration. Decreased HS in the cerebrospinal fluid suggested successful delivery of JR-171 across the BBB, while suppressed urine and serum concentrations of the substrates indicated that its somatic efficacy was comparable to that of laronidase.

2023-06-01·Molecular therapy. Methods & clinical development

Enzyme replacement with transferrin receptor-targeted α-L-iduronidase rescues brain pathology in mucopolysaccharidosis I mice

Article

作者: Tanaka, Satowa ; Minami, Kohtaro ; Inoue, Asuka ; Ito, Jun ; Hirato, Tohru ; Kida, Sachiho ; Yoden, Eiji ; Imakiire, Atsushi ; Sonoda, Hiroyuki ; Yamamoto, Ryuji ; Yoshioka, Aya ; Koshimura, Yuri ; Morimoto, Hideto ; Takahashi, Kenichi ; Mori, Ayaka ; Tanaka, Noboru

Mucopolysaccharidosis I (MPS I), a lysosomal storage disease caused by dysfunction of α-L-iduronidase (IDUA), is characterized by the deposition of dermatan sulfate (DS) and heparan sulfate (HS) throughout the body, which causes several somatic and central nervous symptoms. Although enzyme-replacement therapy (ERT) is currently available to treat MPS I, it does not alleviate central nervous disorders, as it cannot penetrate the blood-brain barrier. Here we evaluate the brain delivery, efficacy, and safety of JR-171, a fusion protein comprising humanized anti-human transferrin receptor antibody Fab and IDUA, using monkeys and MPS I mice. Intravenously administered JR-171 was distributed in major organs, including the brain, and reduced DS and HS concentrations in the central nervous system and peripheral tissues. JR-171 exerted similar effects on peripheral disorders similar to conventional ERT and further reversed brain pathology in MPS I mice. We found that JR-171 improved spatial learning ability, which was seen to deteriorate in the vehicle-treated mice. Further, no safety concerns were noted in repeat-dose toxicity studies in monkeys. This study provides nonclinical evidence that JR-171 might potentially prevent and even improve disease conditions in patients with neuronopathic MPS I without serious safety concerns.

项与 Lepunafusp alfa 相关的新闻（医药）

2025-05-07

·PRNewswire

Mucopolysaccharidosis Type I Market to Witness Upsurge in Growth at a CAGR of 8% During the Forecast Period (2025-2034) | DelveInsight

The future of MPS I treatment is poised for significant advancements, driven by ongoing research and innovative therapies. Emerging therapies, such as stem cell gene therapy, IDUA Gene therapy, and Recombinant DNA, including OTL-203, Lepunafusp alfa (JR-171), RGX-111, and Iduronicrin genleukocel-T (ISP-001), offer novel approaches. These therapies have the potential to reshape the treatment landscape by improving patient outcomes and reducing the limitations of existing treatments. LAS VEGAS, May 7, 2025 /PRNewswire/ -- DelveInsight's Mucopolysaccharidosis Type I Market Insights report includes a comprehensive understanding of current treatment practices, Mucopolysaccharidosis type I emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan]. Key Takeaways from the Mucopolysaccharidosis Type I Market Report According to DelveInsight's analysis, the market size for Mucopolysaccharidosis type I was found to be USD 145 million in the 7MM in 2024. In 2024, the MPS I market size in the US was highest, accounting for 48% of the total market of 7MM. This figure is expected to grow significantly with the introduction of emerging therapies. According to DelveInsight's estimates, in 2024, there were approximately 660 diagnosed prevalent cases of MPS I in the 7MM. Of these, the United States accounted for 36% of the cases. Leading Mucopolysaccharidosis type I companies developing emerging therapies, such as Orchard Therapeutics, Kyowa Kirin, JCR Pharmaceuticals, REGENXBIO, Nippon Shinyaku, IMMUSOFT, and others, are developing novel Mucopolysaccharidosis type I drugs that can be available in the Mucopolysaccharidosis type I market in the coming years. The promising Mucopolysaccharidosis type I therapies in the pipeline include OTL-203, Lepunafusp alfa (JR-171), RGX-111, Iduronicrin genleukocel-T (ISP-001), and others. In March 2025, REGENXBIO Inc. finalized its previously announced strategic partnership with Nippon Shinyaku. Under the agreement, REGENXBIO and Nippon Shinyaku will collaborate to develop and commercialize RGX-111 for Mucopolysaccharidosis I (MPS I), also known as Hurler syndrome, in the United States and Asia. In February 2025, at the 21st Annual WORLDSymposium, Orchard Therapeutics presented updated findings from a proof-of-concept study of OTL-203, an investigational gene therapy for the Hurler subtype of mucopolysaccharidosis type I (MPS-IH), during an encore oral presentation. The presentation summarized key neurological, skeletal, and other clinical outcomes observed in the study. In January 2025, Immusoft announced positive results from the first engineered B Cell (ISP-001) in a human clinical trial, to be presented at the WORLDSymposium. Discover which therapies are expected to grab the major MPS I market share @ Mucopolysaccharidosis Type I Market Report Mucopolysaccharidosis Type I Overview MPS I is a rare inherited lysosomal storage disorder caused by a deficiency in the enzyme alpha-L-iduronidase (IDUA), which is essential for the breakdown of glycosaminoglycans (GAGs) such as dermatan sulfate and heparan sulfate. When these substances accumulate, they lead to progressive damage in cells and tissues, impacting various organ systems, including the skeletal, cardiovascular, respiratory, and nervous systems. The condition presents as a spectrum, with the most severe form being Hurler syndrome and the mildest form known as Scheie syndrome. Diagnosis of MPS I involves testing for elevated GAG levels, enzyme activity assays, and genetic analysis, all of which support early intervention, prognosis determination, and genetic counseling. Early detection is further aided by newborn screening programs. Ongoing disease management relies on comprehensive assessments such as pulmonary function tests, sleep studies, hearing evaluations, eye exams, imaging of the bones, and cognitive testing. Molecular diagnostics and early screening are critical to improving patient outcomes and guiding long-term care. Mucopolysaccharidosis Type I Epidemiology Segmentation The Mucopolysaccharidosis type I epidemiology section provides insights into the historical and current Mucopolysaccharidosis type I patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders. The Mucopolysaccharidosis type I market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into: Diagnosed Prevalence of MPS I Severity-specific Diagnosed Prevalent Cases of MPS I Treated Cases of MPS I Hurler Syndrome Treatment Market The treatment landscape for MPS I (Hurler syndrome) is progressing gradually, with growing insights into both the disease's biology and its management. Traditional therapies like ERT and HSCT remain central, but advances in precision medicine and new pharmacological options are beginning to show promise. Despite these developments, a universal, comprehensive treatment approach remains elusive due to the disease's complexity and variability among patients, requiring a tailored, multidisciplinary strategy. Supportive care plays a critical role and includes various surgical procedures such as adenotonsillectomy, hernia repair, ventriculoperitoneal shunting, heart valve replacement, carpal tunnel release, and spinal decompression. Therapies like physical, occupational, and speech therapy, along with respiratory aids like CPAP, hearing devices, and medications for managing pain and gastrointestinal symptoms, are also essential. Vision issues may require corneal transplants, although surgical interventions in Hurler syndrome can be risky due to anesthetic complications. ALDURAZYME (laronidase), a recombinant version of the human IDUA enzyme produced using Chinese hamster ovary cells, was approved in the U.S., EU4, the UK (in 2003), and Japan (in 2006). It is indicated for patients with Hurler and Hurler-Scheie types of MPS I, and for symptomatic Scheie patients with moderate to severe manifestations. While ALDURAZYME carries a boxed warning due to potential safety concerns, it remains the only approved ERT for MPS I, with no biosimilars available despite the expiration of its patent. To know more about ALDURAZYME mechanism of action and laronidase cost, visit @ ALDURAZYME Patent Expiration Mucopolysaccharidosis Type I Pipeline Therapies and Key Companies OTL-203: Orchard Therapeutics/Kyowa Kirin Lepunafusp alfa (JR-171): JCR Pharmaceuticals RGX-111: REGENXBIO/Nippon Shinyaku Iduronicrin genleukocel-T (ISP-001): IMMUSOFT Discover more about MPS I drugs in development @ Mucopolysaccharidosis I Clinical Trials Market Mucopolysaccharidosis Type I Market Dynamics The MPS I market dynamics are expected to change in the coming years. Current treatments for MPS I, including ERT and hematopoietic stem cell transplantation, have demonstrated effectiveness in managing symptoms and improving the quality of life for patients with severe forms of the disease. Advances in genetic testing and the expansion of newborn screening programs have enabled earlier diagnosis, allowing for timely intervention and significantly improved patient outcomes. Increased awareness of MPS I, coupled with enhanced genetic counseling, has further contributed to earlier detection and better disease management. Additionally, the growing interest in rare diseases has attracted increased funding and support for research, accelerating the development of new and potentially more effective treatments for MPS I. These combined efforts are shaping a more promising future for patients and families affected by this rare lysosomal storage disorder. Furthermore, potential therapies are being investigated for the treatment of MPS I, and it is safe to predict that the treatment space will significantly impact the mucopolysaccharidosis therapeutics market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the MPS I market in the 7MM. However, several factors may impede the growth of the MPS I market. ERTs for MPS I, such as intravenous iduronidase, are lifelong treatments typically administered every week. While ERT has shown effectiveness in alleviating many somatic symptoms, it is unable to cross the blood-brain barrier, rendering it ineffective in addressing the neurological manifestations associated with severe forms of MPS I. This represents a significant limitation in the current therapeutic approach. As an autosomal recessive disorder, MPS I requires both parents to be carriers, which complicates family planning and increases the likelihood of recurrence in future pregnancies. Despite the benefits of available treatments like ALDURAZYME, the financial burden associated with long-term therapy remains a challenge for many patients and families. Additionally, serious adverse reactions—including life-threatening anaphylaxis—have been reported in some patients during or up to three hours after ALDURAZYME infusion, underscoring the need for careful monitoring and consideration of risk-benefit balance in treatment planning. Moreover, MPS I treatment poses a significant economic burden and disrupts patients' overall well-being and QOL. Furthermore, the MPS I market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the MPS I market growth. Scope of the Mucopolysaccharidosis Type I Market Report Therapeutic Assessment: Mucopolysaccharidosis Type I current marketed and emerging therapies Mucopolysaccharidosis Type I Market Dynamics: Key Market Forecast Assumptions of Emerging Mucopolysaccharidosis Type I Drugs and Market Outlook Competitive Intelligence Analysis: SWOT analysis and Market entry strategies Unmet Needs, KOL's views, Analyst's views, Mucopolysaccharidosis Type I Market Access and Reimbursement Download the report to understand which factors are driving MPS I market trends @ Mucopolysaccharidosis Type I Market Trends Table of Contents Related Reports Mucopolysaccharidosis Type I Pipeline Mucopolysaccharidosis Type I Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key MPS I companies, including Orchard Therapeutics, Kyowa Kirin, JCR Pharmaceuticals, REGENXBIO, Nippon Shinyaku, IMMUSOFT, BioMarin Pharmaceutical, Sanofi, among others. Mucopolysaccharidosis Market Mucopolysaccharidosis Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key MPS companies, including Takeda Pharmaceutical, BioMarin Pharmaceuticals, Ultragenyx Pharmaceutical, Sarepta Therapeutics, Sanofi , among others. Mucopolysaccharidosis Type II Market Mucopolysaccharidosis Type II Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key MPS II companies, including Takeda Pharmaceutical Company Limited, BioMarin Pharmaceuticals, Ultragenyx Pharmaceutical, Sarepta Therapeutics, Abeona Therapeutics , among others. Mucopolysaccharidosis Type III Market Mucopolysaccharidosis Type III Market Insights, Epidemiology, and Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key MPS III companies, including Takeda Pharmaceutical Company Limited, BioMarin Pharmaceuticals, Ultragenyx Pharmaceutical, Sarepta Therapeutics, Abeona Therapeutics, Lysogene, Inventiva , among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve . Contact Us Shruti Thakur [email protected] +14699457679 Logo: SOURCE DelveInsight Business Research, LLP WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床1期引进/卖出上市批准临床结果基因疗法

2024-09-05

·Business Wire

JCR Pharmaceuticals’ Data Presentations at SSIEM Annual Symposium 2024 Highlight Investigational Treatments for Lysosomal Storage Disorders

HYOGO, Japan--( BUSINESS WIRE )-- JCR Pharmaceuticals Co., Ltd. (TSE: 4552) made significant contributions at the Society for the Study of Inborn Errors of Metabolism (SSIEM) Annual Symposium 2024, held in Porto, Portugal from September 3-6, 2024. We highlighted our proprietary J-Brain Cargo ® technology through two key presentations focused on advanced therapies for lysosomal storage disorders, demonstrating our pioneering role in medical innovation. "In treating lysosomal storage disorders, it's critical to address the neurological symptoms that often accompany these conditions,” said Shin Ashida, Chairman and President of JCR. “Our focus is on developing therapies that meet these unmet medical needs. Clinical data for JR-171 show its potential to be an effective treatment for both the somatic and neurological symptoms associated with mucopolysaccharidosis I (MPS I). Additionally, the preclinical trials in our GM1 gangliosidosis AAV gene therapy development program have demonstrated the potential of combining our J-Brain Cargo ® technology with gene therapy. We will continue to update on the progress of these programs." JCR showcased the following two presentations: JR-171: Mucopolysaccharidosis Type I (MPS I) Poster The following poster presentation provides a case report for four individuals with MPS I who were treated with JR-171: Neurobehavioural and somatic improvements observed in MPS I patients treated with lepunafusp alfa (JR-171): report of four cases (PO-238) Presenter: Ana Maria Martins, M.D., Ph.D. (Federal University of São Paulo, São Paulo, Brazil) This study reports clinical data from four individuals with MPS I in Brazil who were treated intravenously with JR-171 (2 mg/kg weekly, n=2; 4 mg/kg weekly, n=2), focusing on neurobehavioral changes. These individuals with MPS I experienced marked neurobehavioral and somatic improvements, which resulted in tangible, positive changes in their quality of life. These cases illustrate the positive neuropsychiatric and behavioral changes observed during the Phase 1/2 study of JR-171, as well as the drug’s somatic efficacy, and they demonstrate its potential as an effective treatment for the somatic symptoms and neuronopathy found in individuals with MPS I. Further neurocognitive and developmental evaluation is required to consolidate and establish the efficacy of JR-171 for neuronopathic symptoms. GM1 Gangliosidosis Poster This poster presentation was selected as one of the highest-ranked posters. It highlights preclinical data from an adeno-associated virus (AAV) gene therapy in a mice model of GM1 gangliosidosis: Gene therapy for GM1 gangliosidosis mediated by AAV vector carrying BBB-penetrable enzyme (PO-211) Presenter: Saki Matsushima, Ph.D. (Division of Gene Therapy, Research Center for Medical Sciences, The Jikei University School of Medicine, Tokyo, Japan) This study reports preclinical data that demonstrated efficacy of AAV that expresses anti-transferrin receptor-antibody (anti-TfR antibody) fused β-galactosidase (TβGal). AAV was administered intravenously to the GM1 gangliosidosis mouse models. TβGal expressed in the liver was secreted into the blood and efficiently penetrated the central nervous system (CNS) as well as peripheral organs. Substrates in the CNS decreased in a dose-dependent manner. The data also demonstrated efficacy of TβGal in survival, histology, and behavioral evaluations. This therapeutic approach, which is the combination of AAV and anti-TfR antibody fused biotherapeutics, is expected to be applied for the treatment of several hereditary neurological diseases with CNS involvement. Further evaluation is required. About the Society for the Study of Inborn Errors of Metabolism (SSIEM) Established in 1963, the aim of the society is to foster the study of inherited metabolic disorders and related topics. An international annual symposium is held to promote exchange of ideas between professional leaders in different disciplines who are interested in inborn errors of metabolism. Learn more about SSIEM: https://www.ssiem.org/ . About the J-Brain Cargo ® Platform Technology JCR Pharmaceuticals has developed a proprietary blood-brain barrier-penetrating technology J-Brain Cargo ® , to bring biotherapeutics into the central nervous system. The first drug developed based on this technology is IZCARGO ® (INN: pabinafusp alfa) and was approved in Japan for the treatment of a lysosomal storage disorder. About Mucopolysaccharidosis I (Hurler, Hurler-Scheie, Scheie Syndrome) Mucopolysaccharidosis I (MPS I) is an autosomal recessive lysosomal storage disorders (LSDs) caused by a deficiency of α-L-iduronidase, an enzyme that breaks down glycosaminoglycans (mucopolysaccharides) in the body. The current worldwide prevalence of MPS I is estimated to be approximately 3,000-4,000 (according to JCR internal research). MPS I gives rise to a wide range of somatic and neurological symptoms. A major limitation of current ERT is that it does not address central nervous system (CNS) symptoms because of the enzyme’s inability cross the blood-brain barrier (BBB). MPS I is the only LSD in which hematopoietic stem cell transplantation (HSCT) is part of the standard of case for the severe form of the disease. Significant unmet medical need persists in all forms of MPS I. About GM1 Gangliosidosis GM1 gangliosidosis is an autosomal recessive disease caused by pathogenic mutations in the GLB1 gene which results in dysfunction or lack of production of lysosomal enzyme, β-galactosidase, responsible for the breakdown of GM1 gangliosides and other glycolipids. Accumulation of GM1 gangliosides in multiple tissues including the brain leads to severe neurodegeneration and systemic disease manifestations resulting in premature mortality. GM1 gangliosidosis is classified into three forms (Type I to III) based on the age of onset of clinical symptoms. About JR-171 JR-171 is a recombinant fusion protein of an antibody against the human transferrin receptor and α-L-iduronidase, the enzyme that is missing or malfunctioning in patients with MPS I. By crossing the blood brain-barrier (BBB) through transferrin receptor mediated transcytosis it is expected to be effective against central nervous system (CNS) signs and symptoms of the disease thereby addressing a significant unmet need for the treatment of MPS I. JR-171 previously was granted Fast Track designation by the US Food and Drug Administration (FDA). 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.

快速通道基因疗法

2024-03-19

·BioSpace

JCR Pharmaceuticals Announces Achievement of Milestone Using J-Brain Cargo® Technology for Neurodegenerative Disease in Research Collaboration with Alexion

HYOGO, Japan--(BUSINESS WIRE)-- JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) today announced that it has achieved the first research milestone criterion in a collaboration with Alexion, AstraZeneca Rare Disease (“Alexion”) to develop an undisclosed initial therapeutic molecule that applies JCR’s proprietary J-Brain Cargo®, blood-brain barrier penetration technology, for the treatment of a neurodegenerative disease. (Related release is here). The achievement of a predefined research milestone triggers a milestone payment to JCR. JCR recently strengthened its collaboration with Alexion to address unmet needs by initiating a second research collaboration in December 2023 to apply the same technology to oligonucleotide therapeutics. (Related release is here). The milestone payment has a minor impact on JCR’s consolidated financial results for this fiscal year ending on March 31, 2024. About the J-Brain Cargo® Platform Technology J-Brain Cargo® is a proprietary blood-brain barrier (BBB)-penetrating technology to bring biotherapeutics into the central nervous system. The first drug developed based on this technology and approved in Japan for a lysosomal storage disorder (LSD) is IZGARGO® (INN: pabinafusp alfa) for the treatment of Mucopolysaccharidosis (MPS) type II (Hunter Syndrome). Based on the same platform technology, JR-141(INN: pabinafusp alfa), JR-171 for the treatment of MPS I (INN: lepunafusp alfa) and JR-441 for the treatment of MPS IIIA have advanced into global clinical trials. JCR intends to start clinical trials with four additional programs from its LSD pipeline by FY2028. About JCR Pharmaceuticals Co., Ltd. JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceuticals company that is redefining expectations and 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, Fabry disease, acute graft-versus host disease, MPS II, 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 (Hunter syndrome), MPS III A and B (Sanfilippo syndrome type A and B), and more. JCR strives to expand the possibilities for patients while accelerating medical progress at a global level. Our core values – reliability, confidence, and persistence – benefit all our stakeholders, including employees, partners, and patients. Together we soar. For more information, please visit 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.

上市批准寡核苷酸

100 项与 Lepunafusp alfa 相关的药物交易

登录后查看更多信息

研发状态

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
黏多糖贮积症I型	临床2期	美国	JCR Pharmaceuticals Co., Ltd.	2020-09-01
黏多糖贮积症I型	临床2期	日本	JCR Pharmaceuticals Co., Ltd.	2020-09-01
黏多糖贮积症I型	临床2期	巴西	JCR Pharmaceuticals Co., Ltd.	2020-09-01

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT04227600 (CTgov)	临床1/2期	黏多糖贮积症I型	18	JR-171 (Part1, Dose-escalation)	築醖膚膚廠鹹艱範築網 = 獵鏇構積觸餘顧網願鬱獵餘鹹糧糧襯窪衊繭糧 (繭顧鏇觸壓夢鬱衊願願, 獵襯鏇衊鹽積鬱廠醖鏇 ~ 構膚蓋鹽製夢願襯鑰鹽) 更多	-	2025-03-19
				JR-171 (Part2, 2.0 mg)	築醖膚膚廠鹹艱範築網 = 齋襯製鏇糧獵鹹鹽積獵獵餘鹹糧糧襯窪衊繭糧 (繭顧鏇觸壓夢鬱衊願願, 繭廠夢鏇廠艱艱襯遞製 ~ 淵鑰壓範淵獵廠艱繭願) 更多