Agalsidase Beta BS (agalsidase beta biosimilar) / JCR Pharma, Amicus, GSK, Sumitomo Pharma - LARVOL DELTA

Comparative study on incorporation of three recombinant human α-galactosidase A drugs (agalsidases) into cultured fibroblasts and organs/tissues of Fabry mice. (PubMed, Mol Genet Metab Rep) - "But their affinity for domain 9 of cation-independent mannose 6-phosphate receptor (CI-M6PR), which exists in various cells, was higher in the order: agalsidase beta biosimilar 1 (agalsidase beta BS) > agalsidase beta > agalsidase alfa, which almost coincided with the experimental results regarding the efficiency of their incorporation into cultured fibroblasts derived from a Fabry mouse. On the other hand, no differences in the efficiency of their uptake or reduction of the accumulated substances were observed in the liver, probably due to asialoglycoprotein receptors expressed on the surface of hepatocytes. This information will be useful for making a suitable ERT plan for individual Fabry patients with various backgrounds and for developing new ERT drugs in the future."

Journal • Preclinical • Fabry Disease • Genetic Disorders • ASGR

Pharmacokinetics and pharmacodynamics of JR-051, a biosimilar of agalsidase beta, in healthy adults and patients with Fabry disease: Phase I and II/III clinical studies. (PubMed, Mol Genet Metab) - "Enzyme replacement therapy is currently the standard therapy for the disease, to which two α-galactosidase A formulations have been approved: agalsidase α (Replagal®, Shire) and agalsidase β (Fabrazyme®, Sanofi). In conclusion, these studies demonstrated bioequivalence of JR-051 to agalsidase β in terms of its pharmacokinetics and pharmacodynamics. JR-051 offers a potential new treatment option for patients with Fabry disease."

Clinical • Journal • P1 data • PK/PD data • Fabry Disease • Genetic Disorders • Rare Diseases • Renal Disease

The role of diffusion weighted imaging as supplement to dynamic contrast enhanced breast MRI: Can it help predict malignancy, histologic grade and recurrence? (PubMed, Acad Radiol) - "ADC value is important factor in distinguishing malignancy, differentiating tumors with higher Oncotype score, and PR negativity. Therefore, it can be used as an important tool to assist appropriate treatment selection."

Journal • Breast Cancer • Oncology • Solid Tumor

Non-clinical evaluation of JR-051 as a biosimilar to agalsidase beta for the treatment of Fabry disease. (PubMed, Mol Genet Metab) - "Furthermore, we identified no safety concerns regarding JR-051 in a 13-week evaluation using cynomolgus monkeys. These findings indicate that JR-051 is similar to agalsidase beta in terms of physicochemical and biological properties."

Clinical • Journal