Mucopolysaccharidosis II

7
Pipeline Programs
3
Companies
7
Clinical Trials
1 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
1
2
1
3
0
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

3 companies ranked by most advanced pipeline stage

Denali Therapeutics
Denali TherapeuticsCA - South SF
3 programs
1
2
tividenofusp alfaPhase 2/31 trial
tividenofusp alfaPhase 2/31 trial
tividenofusp alfaPhase 1/21 trial
Active Trials
NCT04251026Active Not RecruitingEst. Feb 2031
NCT06075537Enrolling By InvitationEst. Jun 2027
NCT05371613RecruitingEst. Dec 2027
Takeda
TakedaTOKYO, Japan
1 program
1
Iduronate-2-sulfatase enzyme replacement therapyPhase 2/31 trial
Active Trials
NCT00069641Completed96Est. Mar 2005
GC Biopharma
GC BiopharmaKorea - Yongin
3 programs
1
1
1
idursulfase betaPhase 21 trial
GC1111_0.5mg/kgPhase 1/21 trial
GC1123Phase 11 trial
Active Trials
NCT05422482Active Not RecruitingEst. Jun 2027
NCT01301898CompletedEst. Mar 2011
NCT02663024UnknownEst. Jun 2020

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
2031
Denali Therapeuticstividenofusp alfa
Denali Therapeuticstividenofusp alfa
TakedaIduronate-2-sulfatase enzyme replacement therapy
GC Biopharmaidursulfase beta
Denali Therapeuticstividenofusp alfa
GC BiopharmaGC1111_0.5mg/kg
GC BiopharmaGC1123

Clinical Trials (7)

Total enrollment: 96 patients across 7 trials

NCT06075537Denali Therapeuticstividenofusp alfa

An Extension Study of the Long-Term Safety, Tolerability, and Efficacy of Tividenofusp Alfa (DNL310) in Participants With Mucopolysaccharidosis Type II (MPS II) From Study DNLI-E-0002 or Study DNLI-E-0007

Start: Sep 2023Est. completion: Jun 2027
Phase 2/3Enrolling By Invitation
NCT05371613Denali Therapeuticstividenofusp alfa

A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic (nMPS II) or Non-Neuronopathic Mucopolysaccharidosis Type II (nnMPS II)

Start: Jul 2022Est. completion: Dec 2027
Phase 2/3Recruiting
NCT00069641TakedaIduronate-2-sulfatase enzyme replacement therapy

Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)

Start: Sep 2003Est. completion: Mar 200596 patients
Phase 2/3Completed
NCT02663024GC Biopharmaidursulfase beta

Study of Idursulfase-beta (GC1111) in Hunter Syndrome

Start: Dec 2016Est. completion: Jun 2020
Phase 2Unknown
NCT04251026Denali Therapeuticstividenofusp alfa

A Study of Tividenofusp Alfa (DNL310) in Pediatric Participants With Hunter Syndrome

Start: Jul 2020Est. completion: Feb 2031
Phase 1/2Active Not Recruiting
NCT01301898GC BiopharmaGC1111_0.5mg/kg

To Evaluate the Safety and Efficacy of GC1111 (Recombinant Human Iduronate-2-sulfatase) in Hunter Syndrome Patients

Start: May 2010Est. completion: Mar 2011
Phase 1/2Completed
NCT05422482GC BiopharmaGC1123

A Study to Evaluate the Safety, Tolerability, PK and PD of Intracerebroventricular GC1123 in Patients with MPS Ⅱ

Start: Sep 2022Est. completion: Jun 2027
Phase 1Active Not Recruiting

Phase Legend

Preclinical— Lab & animal studies
Phase 1— Safety & dosing
Phase 2— Efficacy testing
Phase 3— Large-scale trials
On Market— Approved & available

Key Insights

1 actively recruiting trials targeting 96 patients
3 companies competing in this space