An extension of Global Phase III open-label, multicenter designed to evaluate the Long-term safety and efficacy of study drug for the treatment of the MPS II.

开始日期2023-02-22

申办/合作机构

JCR Pharmaceuticals Co., Ltd.

NCT04573023 / Recruiting临床3期

A Phase III Study of JR-141 in Patients with Mucopolysaccharidosis II (STARLIGHT)

A Global Phase III multicenter, randomized, assessor-blinded, active-controlled designed to evaluate safety and efficacy of study drug for the treatment of the MPS II.

开始日期2022-02-14

申办/合作机构

JCR Pharmaceuticals Co., Ltd.

JPRN-jRCT2031210126 / Recruiting临床4期IIT

Post-marketing Clinical Study of IZCARGO in Patients with Mucopolysaccharidosis Type II

开始日期2021-07-27

申办/合作机构-

100 项与 Pabinafusp Alfa 相关的临床结果

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100 项与 Pabinafusp Alfa 相关的转化医学

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100 项与 Pabinafusp Alfa 相关的专利（医药）

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项与 Pabinafusp Alfa 相关的文献（医药）

2023-11-06·Molecular pharmaceutics

Transferrin Receptor-Targeted Iduronate-2-sulfatase Penetrates the Blood-Retinal Barrier and Improves Retinopathy in Mucopolysaccharidosis II Mice

Article

作者: Konaka, Takashi ; Kato, Ryo ; Imakiire, Atsushi ; Masuda, Tomomi ; Minami, Kohtaro ; Suzuki, Hidehiko ; Shirasaka, Ryoji ; Hirato, Tohru ; Yoden, Eiji ; Sonoda, Hiroyuki ; Inoue, Asuka ; Mori, Ayaka ; Morioka, Hiroki ; Morimoto, Hideto

Mucopolysaccharidoses (MPSs) make up a group of lysosomal storage diseases characterized by the aberrant accumulation of glycosaminoglycans throughout the body. Patients with MPSs display various signs and symptoms, such as retinopathy, which is also observed in patients with MPS II. Unfortunately, retinal disorders in MPS II are resistant to conventional intravenous enzyme-replacement therapy because the blood-retinal barrier (BRB) impedes drug penetration. In this study, we show that a fusion protein, designated pabinafusp alfa, consisting of an antihuman transferrin receptor antibody and iduronate-2-sulfatase (IDS), crosses the BRB and reaches the retina in a murine model of MPS II. We found that retinal function, as assessed by electroretinography (ERG) in MPS II mice, deteriorated with age. Early intervention with repeated intravenous treatment of pabinafusp alfa decreased heparan sulfate deposition in the retina, optic nerve, and visual cortex, thus preserving or even improving the ERG response in MPS II mice. Histological analysis further revealed that pabinafusp alfa mitigated the loss of the photoreceptor layer observed in diseased mice. In contrast, recombinant nonfused IDS failed to reach the retina and hardly affected the retinal disease. These results support the hypothesis that transferrin receptor-targeted IDS can penetrate the BRB, thereby ameliorating retinal dysfunction in MPS II.

2022-12-31·mAbs2区 · 医学

Antibodies to watch in 2022

2区 · 医学

Review

作者: Kaplon, Hélène ; Reichert, Janice M. ; Crescioli, Silvia ; Chenoweth, Alicia

In this 13th annual installment of the annual 'Antibodies to Watch' article series, we discuss key events in commercial antibody therapeutics development that occurred in 2021 and forecast events that might occur in 2022. Regulatory review of antibody therapeutics that target the SARS-CoV-2 coronavirus proceeded at an unprecedented pace in 2021, resulting in both emergency use authorizations and full approvals for sotrovimab, regdanvimab, REGEN-COV2, as well as others, in numerous countries. As of November 1, a total of 11 antibody therapeutics had been granted first approvals in either the United States or European Union in 2021 (evinacumab, dostarlimab loncastuximab tesirine, amivantamab, aducanumab, tralokinumab, anifrolumab, bimekizumab, tisotumab vedotin, regdanvimab, REGEN-COV2). The first global approvals of seven products, however, were granted elsewhere, including Japan (pabinafusp alfa), China (disitamab vedotin, penpulimab, zimberelimab), Australia (sotrovimab, REGEN-COV2), or the Republic of Korea (regdanvimab). Globally, at least 27 novel antibody therapeutics are undergoing review by regulatory agencies. First actions by the Food and Drug Administration on the biologics license applications for faricimab, sutimlimab, tebentafusp, relatlimab, sintilimab, ublituximab and tezepelumab are expected in the first quarter of 2022. Finally, our data show that, with antibodies for COVID-19 excluded, the late-stage commercial clinical pipeline of antibody therapeutics grew by over 30% in the past year. Of those in late-stage development, marketing applications for at least 22 may occur by the end of 2022.

2022-06-01·Molecular therapy. Methods & clinical development

Dose-dependent effects of a brain-penetrating iduronate-2-sulfatase on neurobehavioral impairments in mucopolysaccharidosis II mice

Article

作者: Hirato, Tohru ; Morioka, Hiroki ; Yamamoto, Ryuji ; Imakiire, Atsushi ; Minami, Kohtaro ; Sonoda, Hiroyuki ; Morimoto, Hideto

Deposition of heparan sulfate (HS) in the brain of patients with mucopolysaccharidosis II (MPS II) is believed to be the leading cause of neurodegeneration, resulting in several neurological signs and symptoms, including neurocognitive impairment. We recently showed that pabinafusp alfa, a blood-brain-barrier-penetrating fusion protein consisting of iduronate-2-sulfatase and anti-human transferrin receptor antibody, stabilized learning ability by preventing the deposition of HS in the CNS of MPS II mice. We further examined the dose-dependent effect of pabinafusp alfa on neurological function in relation to its HS-reducing efficacy in a mouse model of MPS II. Long-term intravenous treatment with low (0.1 mg/kg), middle (0.5 mg/kg), and high (2.0 mg/kg) doses of the drug dose-dependently decreased HS concentration in the brain and cerebrospinal fluid (CSF). A comparable dose-dependent effect in the prevention of neuronal damage in the CNS, and dose-dependent improvements in neurobehavioral performance tests, such as gait analysis, pole test, Y maze, and Morris water maze, were also observed. Notably, the water maze test performance was inversely correlated with the HS levels in the brain and CSF. This study provides nonclinical evidence substantiating a quantitative dose-dependent relationship between HS reduction in the CNS and neurological improvements in MPS II.

项与 Pabinafusp Alfa 相关的新闻（医药）

2024-12-06

·PipelineReview

MEDIPAL HOLDINGS CORPORATION and JCR Pharmaceuticals Announce Initiation of First Dosing in Phase I/II Clinical Trial of JR-446 for Mucopolysaccharidosis Type IIIB in Japan

– The study aims to evaluate the safety, tolerability, and preliminary efficacy of JR-446 for the treatment of individuals with MPS IIIB, for which there is no approved treatment – TOKYO & HYOGO, Japan I December 05, 2024 I MEDIPAL HOLDINGS CORPORATION (TSE 7459, MEDIPAL) and JCR Pharmaceuticals Co., Ltd. (TSE 4552, JCR) today announced the initiation of the Phase I/II clinical trial of JR-446 in Japan following the dosing of the first individual from the trial. JR-446 is a proprietary blood-brain barrier (BBB)-penetrating α-N-acetylglucosaminidase in development for the treatment of mucopolysaccharidosis type IIIB (Sanfilippo syndrome type B or MPS IIIB). MPS IIIB affects an estimated 500 to 1,000 individuals worldwide, 1 causing severe central nervous system (CNS) symptoms. Despite the dire need, there are currently no approved treatments available for this condition. JR-446, developed using JCR’s proprietary J-Brain Cargo ® technology, has shown promising non-clinical results in addressing the symptoms of this challenging disorder. This Phase I/II clinical trial is an open-label, single-arm, multi-center study that includes individuals under 18 years of age who are diagnosed with MPS IIIB. The study aims to evaluate the safety, tolerability, and exploratory efficacy of JR-446, while also assessing the optimal dosage through the administration of multiple doses. For more details on the trial, visit the Clinical Research Submission and Disclosure System (JR-446-101, jRCT2071240043 ). “We are pleased to be advancing a long-awaited treatment option for MPS IIIB, a condition for which there are no approved therapies,” said Dr. Motomichi Kosuga, Medical Director of the Division of Medical Genetics, National Center for Child Health and Development, and the Medical Expert of the study. “In Japan, MPS IIIB is one of the more frequently observed forms of MPS III, and it primarily affects the CNS, leading to severe symptoms. This trial represents a hopeful step forward in addressing these neurological challenges, and we sincerely hope it will bring meaningful improvements in both the medical outlook and quality of life for patients and their families.” In September 2023, MEDIPAL and JCR entered into a licensing agreement in which MEDIPAL will commercialize JR-446 outside of Japan. In addition, MEDIPAL will support JCR in the clinical development of JR-446 in Japan, including the distribution of investigational drugs, disease awareness, and clinical trial advancement. 2 This collaboration highlights the commitment of MEDIPAL and JCR to pioneer treatments for ultra- rare diseases. By advancing therapies like JR-446, we aim to bring hope to patients and their families, while enhancing corporate value and contributing to a society where everyone can live with physical and mental well-being. About Mucopolysaccharidosis Type IIIB (Sanfilippo Syndrome Type B) Mucopolysaccharidosis type IIIB, or Sanfilippo syndrome type B, is an autosomal recessive disease caused by pathogenic mutations in the NAGLU gene, encoding a lysosomal enzyme involved in the degradation of heparan sulfate. With the accumulation of heparan sulfate in the central nervous system in the brain, individuals with this condition present rapid neurological decline, including sleep disorders, loss of speech, and behavioral changes, which may significantly affect the quality of life of patients and their families. 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 MEDIPAL HOLDINGS CORPORATION MEDIPAL is a holding company which controls, administers and supports the operating activities of companies in which it holds shares in the Prescription Pharmaceutical Wholesale Business; the Cosmetics, Daily Necessities and OTC Pharmaceutical Wholesale Business; and the Animal Health Products and Food Processing Raw Materials Wholesale and Related Business, and conducts business development for the MEDIPAL Group. For more information, visit https://www.medipal.co.jp/english/ . About JCR Pharmaceuticals Co., Ltd. JCR is a global specialty pharmaceuticals company dedicated to advancing treatments for rare and genetic diseases. With nearly 50 years of expertise in Japan, we are expanding to the US, Europe, and Latin America. Our innovative therapies address conditions like growth disorder, MPS II, Fabry disease, acute graft-versus-host disease, and renal anemia. We are also developing treatments for rare diseases like MPS I, MPS II, MPS IIIA and B, and more. Our core values of reliability, confidence, and persistence drive our mission to enhance global medical progress. For more information, visit https://www.jcrpharm.co.jp/en/site/en/ . References SOURCE: JCR Pharmaceuticals

上市批准引进/卖出临床研究

2024-10-23

·BioSpace

JCR Pharmaceuticals Presents Preclinical Gene Therapy Research at the European Society of Gene and Cell Therapy 31st Annual Congress

- Preclinical Data Demonstrate Potential to Deliver an AAV Gene Therapy Using JCR’s Proprietary J-Brain Cargo ® Technology to Treat Central Nervous System Diseases - HYOGO, Japan--(BUSINESS WIRE)-- JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced today that the Company presented preclinical data from its novel adeno-associated virus (AAV) gene therapy research programs in a poster session at the European Society of Gene and Cell Therapy (ESGCT) 31 st Annual Congress, being held October 22-25, 2024, in Rome, Italy. JCR leverages its proprietary J-Brain Cargo ® (JBC) technology for the delivery of adeno-associated virus (AAV) gene therapy to the body and the brain to potentially address the central nervous system (CNS) symptoms by crossing the blood-brain barrier (BBB). “These findings illustrate the potential for applying our proprietary JBC technology as a vehicle to deliver gene therapy across the blood-brain barrier to potentially treat diseases in the central nervous system,” said Hiroyuki Sonoda, Ph.D., Director, Senior Managing Executive Officer, and Executive Director, Research Division at JCR Pharmaceuticals. “This approach has the potential to unlock new therapeutic options for conditions that have historically been challenging to treat. The results from these preclinical studies indicate that the uptake of the gene therapy using our proprietary JBC technology was substantially higher than with AAV9 and a reduction in tropism to the liver, thereby enhancing the safety of AAVs for human-use. We are committed to advancing innovative solutions that address unmet medical needs, and this research marks a crucial step forward.” JCR showcased the following presentation: Incorporation of transferrin receptor binder into AAV enables efficient brain delivery for treatment of genetic CNS diseases (P0133) Presenter: Yuhei Ashida (JCR Pharmaceuticals) The AAV vector leveraging the JBC technology transduced cells in the CNS more efficiently than a conventional AAV9 vector in mice and monkeys. When the modified vector was applied to mouse models of genetic CNS diseases, efficient reduction of toxic substances, histopathological improvements of the brain, improvements of behavioral tests, and greatly improved survival were observed. In addition, a JBC-AAV molecule with additional modifications showed dramatically reduced liver transduction. These results suggest that JCR’s JBC-AAV has the potential to be a promising tool for gene therapy of CNS diseases. Researchers noted the potential advantages of using JBC technology to deliver the AAV gene therapy to the CNS. The JBC-AAV capsid has versatility to fine-tune affinity and uptake. In addition, the capsid has a simple manufacturing scheme and stable production yields. About the European Society of Gene and Cell Therapy (ESGCT) Established in 1992, the European Society of Gene and Cell Therapy (ESGCT) seeks to support scientists and clinicians working in the fields of gene and cell therapy and to promote awareness and understanding of gene and cell therapy and the vast amount of related research in Europe. For more information, please visit . About the J-Brain Cargo ® Platform Technology JCR Pharmaceuticals has developed a proprietary blood-brain barrier (BBB)-penetrating technology, J-Brain Cargo ® , to bring biotherapeutics into the central nervous system (CNS). 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 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 . 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. Contacts Investors & Media: JCR Pharmaceuticals Co., Ltd. Corporate Communications ir-info@jp.jcrpharm.com

上市批准基因疗法

2024-10-16

·BioSpace

JCR Pharmaceuticals to Present at the European Society of Gene and Cell Therapy (ESGCT) 31st Annual Congress

HYOGO, Japan--(BUSINESS WIRE)-- JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) announced today that it will present preclinical data from its proprietary J-Brain Cargo ® -applied adeno-associated virus (AAV) gene therapy preclinical research programs in a poster session at the European Society of Gene and Cell Therapy (ESGCT) 31 st Annual Congress, being held October 22-25, 2024, in Rome, Italy. The poster presentation details are listed below, and the full program can be found on the ESGCT congress website at . Title: Incorporation of transferrin receptor binder into AAV enables efficient brain delivery for treatment of genetic CNS diseases Abstract Number: P0133 Date: October 22, 2024, 7:30-9:00pm CEST Presenter: Yuhei Ashida, Associate Senior Scientist, at JCR Pharmaceuticals About the European Society of Gene and Cell Therapy (ESGCT) Established in 1992, the European Society of Gene and Cell Therapy (ESGCT) seeks to support scientists and clinicians working in the fields of gene and cell therapy and to promote awareness and understanding of gene and cell therapy and the vast amount of related research in Europe. For more information, please visit . About the J-Brain Cargo ® Platform Technology JCR Pharmaceuticals has developed a proprietary blood-brain barrier (BBB)-penetrating technology, J-Brain Cargo ® , to bring biotherapeutics into the central nervous system (CNS). 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 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 . Contacts Investors & Media: JCR Pharmaceuticals Co., Ltd. Corporate Communications ir-info@jp.jcrpharm.com

上市批准基因疗法细胞疗法

100 项与 Pabinafusp Alfa 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11812	-	A16AB09	DB01271

研发状态

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
黏多醣贮积症II型	日本	JCR Pharmaceuticals Co., Ltd.	2021-03-23

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临床结果

适应症

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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


NCT03568175 (Pubmed \| Mol Ther) 人工标引	临床2/3期	黏多醣贮积症II型	28	pabinafusp alfa	鏇鬱簾醖構夢艱築鏇襯(餘製獵鹽憲廠鑰範艱鑰) = 2,124 ± 882.6 ng/mL 鏇齋積壓獵鹹網廠顧鑰 (築艱壓鏇觸築窪襯醖淵 )	积极	2021-02-03
NCT03128593 (Pubmed \| Mol Ther) 人工标引	临床1/2期	黏多醣贮积症II型	14	JR-141	積廠廠窪選醖壓衊鏇蓋(廠衊構顧醖夢淵襯範鑰) = 廠艱艱鏇廠艱憲壓選製獵廠襯鏇鹽艱膚鏇糧簾 (簾構夢艱構選製積構淵 )	积极	2019-02-06