lepunafusp alfa (JR-171) / JCR Pharma - LARVOL DELTA

An Extension Study of JR-171-101 Study in Patients With Mucopolysaccharidosis Type I (MPS I) (clinicaltrials.gov) - P1/2 | N=14 | Completed | Sponsor: JCR Pharmaceuticals Co., Ltd. | Active, not recruiting ➔ Completed

Trial completion • Hurler Syndrome

NEUROBEHAVIOURAL AND SOMATIC IMPROVEMENTS OBSERVED IN MPS I PATIENTS TREATED WITH LEPUNAFUSP ALFA (JR-171): REPORT OF FOUR CASES. (SSIEM 2024) - "Along with the findings on the somatic efficacy of JR-171 in its phase 1/2 study, the observed neurobehavioural changes may suggest potential benefits of the drug on neuronopathy that requires further evaluation."

Clinical • Cardiomyopathy • Cardiovascular

α-L-iduronidase fused with humanized anti-human transferrin receptor antibody (lepunafusp alfa) for mucopolysaccharidosis type I: A Phase 1/2 Trial. (PubMed, Mol Ther) - "Plasma drug concentration increased dose-dependently and reached its maximum approximately 4 hours 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 its somatic efficacy was comparable to that of laronidase."

Journal • P1/2 data • Bone Marrow Transplantation • Hurler Syndrome • Transplantation • IDUA

Enzyme replacement with transferrin receptor-targeted α-L-iduronidase rescues brain pathology in mucopolysaccharidosis I mice. (PubMed, Mol Ther Methods Clin Dev) - "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."

Journal • Preclinical • CNS Disorders • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases

A Study of JR-171 in Patients With Mucopolysaccharidosis I (clinicaltrials.gov) - P1/2 | N=19 | Completed | Sponsor: JCR Pharmaceuticals Co., Ltd. | Active, not recruiting ➔ Completed

Trial completion • Hurler Syndrome

An Extension Study of JR-171-101 Study in Patients With MPS I (clinicaltrials.gov) - P1/2 | N=14 | Active, not recruiting | Sponsor: JCR Pharmaceuticals Co., Ltd. | Recruiting ➔ Active, not recruiting

Enrollment closed • Hurler Syndrome

A Study of JR-171 in Patients With Mucopolysaccharidosis I (clinicaltrials.gov) - P1/2 | N=19 | Active, not recruiting | Sponsor: JCR Pharmaceuticals Co., Ltd. | Recruiting ➔ Active, not recruiting

Enrollment closed • Hurler Syndrome

A Study of JR-171 in Patients With Mucopolysaccharidosis I (clinicaltrials.gov) - P1/2; N=19; Recruiting; Sponsor: JCR Pharmaceuticals Co., Ltd.; Trial completion date: May 2022 ➔ Aug 2022; Trial primary completion date: Mar 2022 ➔ Jun 2022

Clinical • Trial completion date • Trial primary completion date • Hurler Syndrome

An Extension Study of JR-171-101 Study in Patients With MPS I (clinicaltrials.gov) - P1/2; N=15; Recruiting; Sponsor: JCR Pharmaceuticals Co., Ltd.; Not yet recruiting ➔ Recruiting

Clinical • Enrollment open • Hurler Syndrome

A Study of JR-171 in Patients With Mucopolysaccharidosis I (clinicaltrials.gov) - P1/2; N=19; Recruiting; Sponsor: JCR Pharmaceuticals Co., Ltd.; Trial primary completion date: Sep 2021 ➔ Mar 2022

Clinical • Trial primary completion date • Hurler Syndrome

An Extension Study of JR-171-101 Study in Patients With MPS I (clinicaltrials.gov) - P1/2; N=15; Not yet recruiting; Sponsor: JCR Pharmaceuticals Co., Ltd.; Trial completion date: Oct 2026 ➔ Apr 2025; Initiation date: May 2021 ➔ Oct 2021; Trial primary completion date: Oct 2026 ➔ Apr 2025

Clinical • Trial completion date • Trial initiation date • Trial primary completion date • Hurler Syndrome

A Study of JR-171 in Patients With Mucopolysaccharidosis I (clinicaltrials.gov) - P1/2; N=19; Recruiting; Sponsor: JCR Pharmaceuticals Co., Ltd.; Not yet recruiting ➔ Recruiting; Trial completion date: Sep 2021 ➔ May 2022

Clinical • Enrollment open • Trial completion date • Hurler Syndrome

JCR Pharmaceuticals: Announces Completion of the Phase 1/2 Clinical Trial Notification of JR-171 for mucopolysaccharidosis type I (MPS-Phase 1/2 clinical trial is e I) (Market Screener) - "JCR Pharmaceuticals Co., Ltd....announced today the successful completion of the review for the phase 1/2 clinical trial notification of JR-171for MPS I (Hurler syndrome, Hurler-Scheie syndrome, Scheie syndrome) by the Pharmaceuticals and Medical Devices Agency (PMDA)....This study is planned to be conducted in Japan, Brazil, and the United States as the JCR's first global clinical trial. We aim to start this study in September of this year in Japan prior to other countries."

New P1/2 trial • Genetic Disorders • Hurler Syndrome • Lysosomal Storage Diseases

An Extension Study of JR-171-101 Study in Patients With MPS I (clinicaltrials.gov) - P1/2; N=15; Not yet recruiting; Sponsor: JCR Pharmaceuticals Co., Ltd.

Clinical • New P1/2 trial • Gene Therapies • Hurler Syndrome

A Study of JR-171 in Patients With Mucopolysaccharidosis I (clinicaltrials.gov) - P1/2; N=18; Not yet recruiting; Sponsor: JCR Pharmaceuticals Co., Ltd.

Clinical • New P1/2 trial