– JCR will host a webinar on October 4, 2023 to present more details –
HYOGO, Japan–(BUSINESS WIRE)–JCR Pharmaceuticals Co., Ltd. (TSE 4552; Chairman and President: Shin Ashida; “JCR”) today announced key interim 52-week data from its global Phase I/II study Results with JR-171 (INN: lepunafusp alfa) for use in individuals with mucopolysaccharidosis type I (MPS I, also known as Hurler, Hurler-Scheie and Scheie syndrome). JR-171 is a blood-brain barrier (“BBB”) penetrating form of recombinant alpha-L-iduronidase, developed using JCR’s proprietary J-Brain Cargo® technology. There are currently no approved therapies that cross the BBB and address central nervous system (CNS) symptoms in patients with MPS I.
The overall safety data concluded that JR-171 is suitable for long-term treatment of individuals with MPS I. While the duration of treatment and number of enrolled individuals are not intended to demonstrate an effect on central nervous system symptoms, preliminary data suggest that JR-171 may have a beneficial impact on neurological disease burden in symptomatic individuals. Somatic biomarkers remained stable in individuals previously exposed to α-L-iduronidase, whereas somatic biomarkers decreased significantly in treatment-naïve individuals. This suggests that JR-171 provides comparable somatic disease control to somatic enzyme replacement therapy (ERT). Substrate concentrations in the cerebrospinal fluid were significantly reduced in all patients, possibly indicating the effects of reduced substrate in the central nervous system.
“In addition to achieving the primary and secondary endpoints, it is encouraging to observe additional changes in communication, executive function and other important aspects of daily life in individuals treated with JR-171,” said researcher Paul Harmatz, MD, Ph.D. UCSF Benioff Children’s Hospital, Oakland. “Based on these data, JR-171 has the potential to treat somatic and neurological symptoms in MPS I patients, which is ultimately what we need.”
JCR Pharmaceuticals plans to host a webinar on October 4, 2023 to provide further details on the results of the 52-week clinical study. To register for the webinar, please use the following link https://us06web.zoom.us/webinar/register/WN_vLm-1crCQRqIA5gvk0ArkQ.
This matter has no impact on the consolidated business results for the fiscal year ended March 31, 2024.
About JR-171
JR-171 is a recombinant fusion protein of an anti-human transferrin receptor antibody and α-L-iduronidase, an enzyme that is missing or dysfunctional in MPS I patients. Receptor-mediated transcytosis of transferrin across the blood-brain barrier (“BBB”) is expected to be effective against the signs and symptoms of central nervous system (“CNS”) disease, thereby addressing unmet needs in the treatment of MPS I significant needs. JR-171 has previously been granted Fast Track designation by the U.S. Food and Drug Administration and Drug Administration (FDA).
The Phase I/II clinical study is being conducted at multiple centers in Brazil, Japan and the United States and consists of two parts: the first part of the open-label study is a 4-week dose-escalation study I in adult individuals with MPS; Part 2 compared two doses of JR-171 in pediatric and adult patients with MPS I. The primary objectives of the Phase I/II clinical study are to determine the safety of long-term administration of JR-171 in patients with MPS I, the pharmacokinetics of JR-171, study physical disease control based on biomarkers and physical endpoints, and explore the impact of MPS I central Neurological signs and symptoms Early indications of efficacy.
in the worldforumTM value In February 2023, the JCR had proposed that reduced cerebrospinal fluid biomarkers were observed in all patients, and biomarkers in serum and urine were either reduced (in untreated subjects) or stable (in subjects who had undergone enzyme replacement). therapy (“ERT”) after 12 weeks of treatment with JR-171.
No drug-related serious adverse events (SAEs) were reported during the study. Drug-related adverse events (AEs) and the formation of anti-drug antibodies were comparable to those typically observed in somatic ERT. There was no significant difference in the safety profile of JR-171 at any of the dose levels tested.
About J-Brain Cargo® Platform technology
JCR Pharmaceuticals develops proprietary BBB penetration technology J-Brain Cargo®, Introduction of biotherapeutics into the central nervous system. The first drug developed based on this technology and approved in Japan for the treatment of MPS II (mucopolysaccharidosis type II) is IZGARGO® (INN: pabinafusp alfa). Based on the same platform technology, JR-171 is the second project to enter the global clinical stage. JCR plans to begin clinical trials of five additional projects in its LSD pipeline by 2028.
About MPS I (Hurler, Hurler-Scheie, Scheie syndrome)
MPS I is an autosomal recessive lysosomal storage disorder (LSD) caused by a deficiency of alpha-L-iduronidase, a protein that breaks down Enzymes for glycosaminoglycans (mucopolysaccharides). The current global prevalence of MPS I is estimated to be approximately 3,000 (based on JCR internal research). MPS I causes a variety of physical and neurological symptoms. A major limitation of current ERT is that it does not address central nervous system symptoms because the enzyme cannot cross the blood-brain barrier. MPS I is the only LSD that includes hematopoietic stem cell transplantation (“HSCT”) as part of the case criteria for severe disease. Substantial unmet medical needs remain in all forms of MPS I.
About JCR Pharmaceuticals Ltd.
JCR Pharmaceuticals Co., Ltd. (TSE 4552) is a global specialty pharmaceutical company that is redefining expectations and expanding possibilities for patients with rare and genetic diseases worldwide. We continue to build on our 48-year legacy in Japan while expanding our global footprint into the United States, Europe and Latin America. We use our scientific expertise and unique technologies to research, develop and deliver next-generation therapies that improve patients’ lives. Our approved products in Japan include therapies to treat growth disorders, Fabry disease, acute graft-versus-host disease and renal anemia. We develop investigational products globally to treat rare diseases, including MPS I (Hurler, Hurler-Scheie and Scheie syndromes), MPS II (Hunter syndrome), MPS III A and B (types A and B Sanfilippo syndrome), etc. JCR is dedicated to expanding possibilities for patients while accelerating medical advancements around the world. Our core values - reliability, confidence and persistence – benefit all our stakeholders, including employees, partners and patients. We soar together. For more information, please visit https://www.jcrpharm.co.jp/en/site/en/.
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