Product Description
JR-446 is a novel drug developed based on the J-Brain Cargo technology, which has been clinically validated through the approval of IZCARGO for the treatment of MPS II in Japan in 2021. Animal studies have previously confirmed delivery of JR-446 into the Central Nervous System (CNS) and somatic organs and significant reduction of substrate caused by the deficiency in the enzyme that causative for the onset of MPS IIIB. As there is no established standard of care for the treatment of MPS IIIB, JR-446 is believed to have the potential to become a transformative treatment of MPS IIIB. (Sourced from: https://curesanfilippofoundation.org/2024/07/jr-446-enzyme-replacement-mps-iiib-phase-i-ii/)
Mechanisms of Action: NAGLU Inhibitor
Novel Mechanism: Yes
Modality: N/A
Route of Administration: N/A
FDA Designation: None *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: JCR
Company Location:
Company CEO:
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic: Japan
Active Clinical Trial Count: 2
Highest Development Phases
Phase 2: Mucopolysaccharidosis III
Trial |
Phase |
Trial Status |
Disease |
Primary Completion Date |
Probability of Success |
|---|---|---|---|---|---|
| jRCT2071240043 | P2 |
Not yet recruiting |
Mucopolysaccharidosis III |
2029-08-31 |
|
| JR-446-101 | P2 |
Not yet recruiting |
Mucopolysaccharidosis III |
2027-03-31 |