JR-171 is a blood brain-barrier ( BBB )-penetrating form of recombinant -L-iduronidase that was developed using JCR s proprietary J-Brain Cargo technology. There are no approved therapies that cross the BBB and address the central nervous system (CNS) symptoms for individuals with MPS I.
The overall safety data concluded that JR-171 is suitable for the long-term treatment of individuals with MPS I. Although treatment duration and the number of individuals enrolled were not intended to demonstrate an effect on CNS symptoms, preliminary data suggest that JR-171 may provide a beneficial effect on neurological disease burden in symptomatic individuals. Somatic biomarkers remained stable in individuals previously exposed to -L-iduronidase and were significantly reduced in the one individual who was treatment-na ve. This indicates that JR-171 provides somatic disease control comparable to somatic enzyme replacement therapy (ERT). Concentrations of substrate in the cerebrospinal fluid, which may indicate an effect of substrate reduction in the CNS, were significantly reduced in all patients.
It is encouraging to observe additional changes in communication, executive functioning, and other aspects important in daily life for those individuals treated with JR-171, in addition to achieving the primary and secondary endpoints, said
There is no impact on the consolidated business results for this fiscal year ending on
About JR-171
JR-171 is a recombinant fusion protein of an antibody against the human transferrin receptor and -L-iduronidase, the enzyme that is missing or malfunctioning in subjects 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
The phase I/II clinical study is being conducted at several centers in
At WORLDSympoisumTM 2023 in
There were no drug-related serious adverse events (SAEs) reported during the study. Drug-related adverse events (AEs) and formation of anti-drug antibodies were comparable to profiles typically observed with somatic ERT. There were no gross differences in the safety profile of JR-171 at either dose level tested.
About the J-Brain Cargo Platform Technology
About MPS I (Hurler, Hurler-Scheie, Scheie syndrome)
MPS I is an autosomal recessive lysosomal storage disorders ( LSD ) caused by a deficiency of -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 (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 CNS symptoms because of the enzyme s inability cross the BBB. MPS I is the only LSD in which hematopoietic stem cell transplantation ( HSCT ) is part of the standard of case in the severe form of the disease. Significant unmet medical need persists in all forms of MPS I.
About
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