JR-446 Enzyme Replacement | MPS IIIB | Phase I-II | MEDIPAL/JCR

July 1, 2024

Page last updated: July 5, 2024

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Trial Information

Phase I/​II Study of JR-446 in Patients With Mucopolysaccharidosis Type IIIB

“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,” according to JCR.

JCR’s proprietary J-Brain Cargo technology involves the fusion of enzyme to a transferrin receptor targeting antibody. Transferrin receptors are abundant on the surface of blood vessels that supply brain tissue. The combined enzyme-antibody fusion protein can engage with the transferrin receptor to allow the needed enzyme to cross the blood brain barrier and gain entry into the central nervous system.

A graphic representation of the J-brain cargo technology

Study title: An Open-label Phase I/​II Study of JR-446 in Mucopolysaccharidosis Type IIIB

Trial listing: Read this clinical trial’s information on ClinicalTrials.gov, for more details including contact information to reach out to the study sites, full inclusion and exclusion criteria. We encourage you to check the Clinicaltrials.gov link frequently for updates on possible new study sites and changes in enrollment status or criteria.

Study status: Not yet recruiting

Number of participants: 10 patients with Type B

Duration: Up to 4 years

Location: 3 sites – Hiroshima, Okinawa, and Toyoko, Japan

Qualifications to Participate:

Inclusion Criteria:

  • Chronological age of <18 years
  • Confirmed diagnosis of MPS IIIB

Exclusion Criteria:

  • Prior experience to gene therapy or HSCT with successful engraftment
  • Past use of another investigational drug or product in last 4 months or 5 half-lives (whichever is longer) before signing ICF
  • Current participation in a clinical trial or past participation (within 30 days of enrolment into this study) in a study involving invasive procedures
  • Past use of Genistein or Kineret (anakinra) within 4 months before signing ICF
  • Serious drug allergy or hypersensitivity
  • Contraindication for lumbar puncture or MRI
  • History of bleeding disorder or current use of medications that, in the opinion of the investigator, place them at risk of bleeding following lumbar puncture

Trial Updates

July 4, 2024

“An Open-label Phase I/​II Study of JR-446 in Mucopolysaccharidosis Type IIIB” is listed on ClinicalTrials.gov. The listing includes inclusion and exclusion criteria, as well as the locations of the three trials sites and their contact information. Read the listing.

July 1, 2024

MEDIPAL and JCR Pharmaceuticals announced the completion of the regulatory review by Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for the clinical trial notification for the phase I/II study of JR-446, a blood-brain barrier-penetrating α-N-acetylglucosaminidase, for the treatment of mucopolysaccharidosis type IIIB (MPS IIIB; Sanfilippo syndrome type B). The company plans to start the clinical trial in Japan in the coming months (April-September 2024). Read more.

September 2023

MEDIPAL Holdings Corporation and JCR Pharmaceuticals announced that the Board of Directors of both companies have reached agreement on their partnership to develop JR-446, a blood-brain barrier (BBB) penetrating form of α-N-acetylglucosaminidase that was developed using JCR’s proprietary J-Brain Cargo® technology, for the treatment of Mucopolysaccharidosis Type IIIB (MPS IIIB or Sanfilippo syndrome type B).

Under the ex-Japan licensing agreement, MEDIPAL will acquire an exclusive commercialization right of JR-446 with sublicensing rights, including clinical development, manufacturing, and marketing. As regards to the Japan collaboration agreement, MEDIPAL will support RegardingJCR in the clinical development of JR-446 in Japan, including distribution of investigational drugs, disease awareness and conducting clinical trials. Read more.

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