JCR Pharmaceuticals Co., Ltd. highlighted several programs that rely on J-Brain Cargo®?, a proprietary technology developed by JCR, to deliver medicines across the blood-brain barrier (BBB). The second JR-141 presentation details the global phase III clinical trial study. In addition to the three JR-141 presentations, the WORLD Symposium tm 2024 scientific program featured presentations focusing on: 52-week interim data from the JR-171 phase I/II clinical trial (Hurler syndrome, Hurler-Scheie syndrome or Scheie syndrome); pre-clinical data on an ERT with JR-171 preventing bone deformities in a mouse model of MPS I; JR-441 global clinical trial study design (Sanfilippo syndrome type A).

The following poster presentation provides an overview of the global phase III clinical trial design for JR-141 in the treatment of MPS II: A global phase III study of pabinafusp alfa (JR-141) for neuronopathic mucopolysaccharidosis type II: updated study design (Abstract 154). This abstract provides an overview of the ongoing global phase III clinical trial of pabinafusp Alfa (JR-141) For neuronopathic MPS IIIA. Primary efficacy endpoints are assessed on the basis of HS levels in the CSF and neurocognitive test (BSID-III) results.

A pivotal phase III clinical trial is planned to establish the efficacy and safety of the drug. The following late-breaking abstract discloses patient experiences upon treatment with JR-171: The impact of lepunafusp alfa (JR 171) on the disease burden in MPS I: Patient reported outcomes (Abstract LB-39). Presenter: Ana Maria Martins (Reference Center in Inborn Errors of Metabolism, Federal University of Sao Paulo, Brazil) This late-breaking abstract presents data on patient reported outcomes of individuals treated in an ongoing phase I/II study with lepunafusp alfa, a fusion protein consisting of a transferrin-receptor binding domain and a-L-iduronidase, the enzyme missing or malfunctioning in MPS I. JCR Pharmaceuticals has developed a proprietary BBB-penetrating technology J-Brain Cargo®?., to bring biotherapeutics into the CNS.

The first drug developed based on this technology and approved in Japan for the treatment of MPS II (mucopolysaccharidosis typeII) is IZGARGO®? (INN: pabinafusp alfusp alfa). Based on the same platform technology, JR-171 (INN:lepunafusp alfa) and JR-441 are advancing into global clinical stage.

JCR intends to start clinical trials on four additional programs from the global phase III clinical trial trial study design for JR-141 in a mouse model of M PS I; JR-441 global trial study design for JR-171 in the treatment of MPS I; JR -441 global clinical trial study design for JR- 141 in the treatment of MPSIIIA: A phase I/II clinical trial of JR-141 for treatment of Sanfilippo syndrome type A (MPS IIIA) (Abstract 234): Presenter: Nicole Muschol, M.D. (University Medical Center Hamburg-Eppendorf, International Center for Lysosomal Disorders (ICLD), Hamburg, Germany). This abstract reports the Phase I/II clinical trial study design of JR-441 as a first-in-human, open-label, single-center trial to evaluate the optimal dose, safety/tolerability, pharmacokinetics and exploratory efficacy in individuals with MPS IIIA aged 1 year to 18 years. In several clinical trials with pabinafusp alafusp alfa, JCR obtained evidence of reducing heparan sulfate (HS) concentrations in the CSF, a biomarker that is believed to reflect substrate reduction in the brain and changes in CNS function.

These results were consistent with those obtained in pre-clinical studies. These results were consistent with these results were consistent with those obtain in pre-clinical studies.