Takeda to Commercialize Next-Generation Hunter Syndrome Therapy Through Collaboration with JCR Pharmaceuticals
− JR-141 is a potentially transformative therapy designed to deliver proteins to the brain and peripheral tissues to treat neuronopathic features along with somatic symptoms of Hunter syndrome via intravenous administration
− Takeda to exclusively commercialize JR-141 outside the
− Takeda receives a separate option to obtain an exclusive license to commercialize in the
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Under the terms of the exclusive collaboration and license agreement, Takeda will exclusively commercialize JR-141 outside of
Takeda receives an option under a separate option agreement, which allows Takeda to acquire an exclusive license to commercialize JR-141 in the
“Takeda is committed to continuously improving the way Hunter syndrome is treated. JR-141 introduces a new way to deliver proteins across the blood-brain barrier, overcoming our current challenges to treat the underlying neuronopathic manifestations of Hunter syndrome and help maintain or improve cognitive function in these patients,” said
“JCR is pleased to have reached an agreement with Takeda who is well placed to achieve our common goal of maximizing the impact of JR-141,” said
JR-141 met its primary endpoint in an open-label Phase 2/3 clinical trial in
JR-141 is a recombinant fusion protein of an antibody against the human transferrin receptor and iduronate-2-sulfatase, the enzyme that is missing or malfunctioning in subjects with Hunter syndrome. It is expected to be effective against the neuronopathic manifestations of the disease by crossing the BBB through transferrin receptor mediated transcytosis using J-Brain Cargo®, JCR’s proprietary BBB technology. Uptake into cells is mediated through the transferrin receptor and mannose-6-phosphate receptor. JCR has advanced development activities by establishing the necessary evidence from the molecular design stage to the nonclinical and clinical trial phases. In non-clinical trials, JCR has confirmed both high affinity binding of JR-141 to transferrin receptors, and passage across the BBB into neuronal cells as evidenced by electron microscopy.
In addition, JCR has confirmed that using J-Brain Cargo® technology, enzymes are taken up into various brain tissues. A decrease in substrate accumulation has also been confirmed in an animal model of Hunter syndrome.2,3,4 In several clinical trials with JR-141, JCR obtained evidence of reduction of heparan sulfate concentrations in the CSF, a biomarker for assessing the drug’s effectiveness in reducing disease-causing substrate in the central nervous system, consistent with the results obtained from non-clinical studies. JCR also obtained clinical results that demonstrate positive effects of JR-141 on neurocognition.5,6,7,8
JR-141 was approved by the
About Hunter Syndrome
Hunter syndrome is a severely debilitating, rare lysosomal disease caused by a deficiency of iduronate-2-sulfatase, an enzyme that is needed to break down substances in the body called glycosaminoglycans (GAGs).9 Without this enzyme, GAGs can build up, causing a range of disease-related signs and symptoms.9,10 Roughly two of every three patients with Hunter syndrome are also affected with progressive cognitive decline.11 Hunter syndrome affects 1 in 162,000 total live births, and almost exclusively males.12
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1. Okuyama T, Eto Y, Sakai N, et al. A phase 2/3 trial of pabinafusp alfa, IDS fused with anti-human transferrin receptor antibody, targeting neurodegeneration in MPS-II. Molecular Therapy. 2021;29(2):671-679.
2. Sonoda, et al. A Blood-Brain-Barrier-Penetrating Anti-human Transferrin Receptor Antibody Fusion Protein for Neuronopathic Mucopolysaccharidosis II. Mol Ther. 2018; 26(5): 1366-74.
3. Morimoto, et al. Clearance of heparin sulfate in the brain prevents neurodegeneration and neurocognitive impairment in MPS II mice. Mol. Ther. 2021; https://doi.org/10.1016/j.ymthe.2021.01.027.
4. Yamamoto et al. Nonclinical Safety evaluation of pabinafusp alfa, an anti-human transferrin receptor antibody and iduronate-2-sulfatase fusion protein, for the treatment of neuronopathic mucopolysaccharidosis type II. Mol Genet Metab Rep. 2021; https://doi.org/10.1016/j.ymgmr.2021.100758.
5. Okuyama, et al. Iduronate-2-sulfatase with Anti-human Transferrin Receptor Antibody for Neuropathic Mucopolysaccharidosis II: A Phase 1/2 Trial. Mol Ther. 2020; 27(2): 456-464.
6. Okuyama, et al. A Phase 2/3 Trial of Pabinafusp Alfa, IDS Fused with Anti-Human Transferrin Receptor Antibody, Targeting Neurodegeneration in MPS-II. Mol Ther. 2020; 29(2): 671-679.
7. Giugliani, et al. Iduronate-2-sulfatase fused with anti-human transferrin receptor antibody, pabinafusp alfa, for treatment of neuronopathic and non-neuronopathic mucopolysaccharidosis II: Report of a phase 2 trial in
8. Giugliani, et al. Enzyme Replacement Therapy with Pabinafusp Alfa for Neuronopathic Mucopolysaccharidosis II; an Integrated Analysis of Preclinical and Clinical Data. Preprints 2021; 2021090192.
9. Wraith JE, et al. Mucopolysaccharidosis type II (Hunter syndrome): a clinical review and recommendations for treatment in the era of enzyme replacement therapy. Eur J Pediatr. 2008; 167(3):267-77.
10. Martin R. Recognition and Diagnosis of Mucopolysaccharidosis II (Hunter Syndrome). PEDIATRICS. Volume 121, Number 2,
11. Young I. A clinical and genetic study of Hunter's syndrome. 2 Differences between the mild and severe forms.
12. Meikle PJ, et al. Prevalence of Lysosomal Storage Disorders. JAMA. 1999; 281(3):249-54.
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