Hunterase (recombinant human iduronate-2-sulfatase) / GC Biopharma, CANbridge Pharma - LARVOL DELTA

Site-specific characterization of mannose-6-phosphate-containing N-glycans on recombinant idursulfase beta for lysosomal targeting in Hunter syndrome therapy. (PubMed, Int J Biol Macromol) - "Idursulfase beta (IDS, Hunterase®), a recombinant human I2S produced in CHO cells, is used clinically in enzyme replacement therapy (ERT)...Phosphorylated N-glycans exhibited site-specifically distribution: Asn221 (one P1, three P2) and Asn255 (three P1, three P2) were fully phosphorylated; Asn90 (one P1) and Asn512 (two P1) showed partial phosphorylation...Furthermore, cellular uptake studies using fluorescent-labeled IDS in human fibroblasts confirmed efficient endocytosis and lysosomal delivery. This study provides the first site-specific characterization of M6P-containing N-glycans in IDS, supporting its therapeutic efficacy in ERT for Hunter syndrome."

Journal • Hunter Syndrome • Lysosomal Storage Diseases • Metabolic Disorders • Pediatrics • Rare Diseases

Efficacy and safety of idursulfase beta in the treatment of mucopolysaccharidosis II: a phase 3, two-part study compared to a historical placebo cohort. (PubMed, Genet Med) - "Idursulfase beta is a safe and effective treatment option in MPS II, addressing crucial somatic ailments presented by patients."

Clinical • Journal • P3 data • Hunter Syndrome

A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ (clinicaltrials.gov) - P1 | N=12 | Active, not recruiting | Sponsor: GC Biopharma Corp | Recruiting ➔ Active, not recruiting | Trial completion date: Jun 2026 ➔ Jun 2027 | Trial primary completion date: Jun 2026 ➔ Jun 2027

Enrollment closed • Trial completion date • Trial primary completion date • Hunter Syndrome

TWO-YEAR SAFETY AND EFFICACY EXTENSION STUDY OF GC1111 IN MPS-II PATIENTS TREATED WITH GC1111 OR IDURSULFASE DURING PHASE 3 STUDY (SSIEM 2024) - "Long-term treatment of GC1111 for 2 years and switched treatment from idursulfase both showed sustained efficacy and tolerable safety. Therefore, GC1111 could be a promising alternative treatment option to idursulfase as ERT for MPS-II."

Clinical • P3 data • Gastroenterology • Gastrointestinal Disorder • Hunter Syndrome • Infectious Disease

A Study of GC1111 in Hunter Syndrom Patients (clinicaltrials.gov) - P3 | N=32 | Completed | Sponsor: Green Cross Corporation | Unknown status ➔ Completed

Trial completion • Hunter Syndrome

A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients With MPS Ⅱ (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: GC Biopharma Corp | Trial completion date: Mar 2025 ➔ Jun 2026 | Trial primary completion date: Mar 2025 ➔ Jun 2026

Trial completion date • Trial primary completion date • Hunter Syndrome

COMBINED ALLOGENEIC BONE MARROW TRANSPLANTATION AND INTRACEREBROVENTRICULAR ENZYME REPLACEMENT THERAPY FOR PATIENTS WITH NEURONOPATHIC MUCOPOLYSACCHARIDOSIS TYPE II (EBMT 2023) - "Recently, IDS-beta (Hunterase®) has been newly approved in Japan as the intracerebroventricular enzyme replacement therapy (ICV-ERT) for the patients with neuronopathic MPS-II...All patients were prepared with the regimen consisting of targeted dose of busulfan (Bu, the median total area under curve was 73.5 mg･h/l [range, 63.5–88.4 mg･h/l]), 200 mg/kg of cyclophosphamide (Cy), and 5 mg/kg of rabbit anti-thymocyte globulin (rATG), and were administered tacrolimus and short-term methotrexate (15 mg/m2 at day 1, 10 mg/m2 at day 3, 6, and 11) for GvHD prophylaxis... Combined allo-BMT with targeted Bu+Cy+ATG and ICV-ERT is suggested to be a promising novel strategy for patients with neuronopathic MPS-II."

Clinical • Acute Graft versus Host Disease • Bone Marrow Transplantation • Graft versus Host Disease • Hunter Syndrome • Immunology • Lysosomal Storage Diseases • Metabolic Disorders • Rare Diseases • Steven-Johnson Syndrome • Transplantation • HLA-DRB1

A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients With MPS Ⅱ (clinicaltrials.gov) - P1 | N=12 | Recruiting | Sponsor: GC Biopharma Corp | Not yet recruiting ➔ Recruiting | Initiation date: Jun 2022 ➔ Sep 2022

Enrollment open • Trial initiation date • Hunter Syndrome

Impact of ERT and follow-up of 17 patients from the same family with a mild form of MPS II. (PubMed, Clinics (Sao Paulo)) - "Here, the authors report the variable progress of the disease with and without ERT in a large Brazilian family with a slowly progressive form of MPS II, harboring the same missense variant in the IDS gene."

Journal • Cardiovascular • Congestive Heart Failure • Heart Failure • Hunter Syndrome • Hurler Syndrome • Musculoskeletal Pain • Transplantation

A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients With MPS Ⅱ (clinicaltrials.gov) - P1 | N=12 | Not yet recruiting | Sponsor: GC Biopharma Corp

New P1 trial • Hunter Syndrome

A Study of GC1111 in Hunter Syndrom Patients (clinicaltrials.gov) - P3; N=34; Recruiting; Sponsor: Green Cross Corporation

Clinical • New P3 trial