Medipal, JCR dose first patient in global Phase I/II JR-446 trial for MPS IIIB

PUBT · 2d ago
Medipal, JCR dose first patient in global Phase I/II JR-446 trial for MPS IIIB
  • Medipal and JCR Pharmaceuticals dosed the first patient in a global Phase I/II trial of JR-446 for MPS IIIB.
  • Results have not been presented; the study will assess safety, tolerability, and early signs of benefit.
  • Medipal holds commercialization rights for JR-446 outside Japan, shaping potential future revenue if development succeeds.
  • JCR is running the global program as sponsor; a separate Phase I/II study remains ongoing in Japan.
  • The drug received orphan designations in the US, EU, and Japan in 2025, supporting a clearer regulatory path.


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