VivoSeq
Metabolic & Endocrine

Mucopolysaccharidosis

33

Pipeline Programs↗

11

Active Trials↗

15

Companies Involved↗

Mucopolysaccharidosis is a moderately active landscape with 33 programs across 15 companies. The most active players are PTC Therapeutics, Genzyme, a Sanofi Company, JCR Pharmaceuticals Co., Ltd.. 6 therapies are approved with 10 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

55/100

Pipeline Velocity

6/100

Mech. Concentration

14/100

Completion Rate

83/100

Attrition Rate

11/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

P

PTC Therapeutics

1 program

Phase 1G

Genzyme, a Sanofi Company

1 program

Phase 1J

JCR Pharmaceuticals Co., Ltd.

4 programs

Phase 2Ultragenyx logo

Ultragenyx

1 program

Phase 2AstraZeneca logo

AstraZeneca

1 program

Phase 2A

Abeona Therapeutics, Inc

1 program

Phase 2

Pipeline

Programs by Phase

Analysis

Modality Breakdown

67%

Small molecule

20 of 30 classified programs — the dominant modality here.

33%ALL OTHERSProtein / enzyme620%Monoclonal antibody27%Gene therapy27%

Small molecule leads; the ring shows all 30 classified programs, each share of the total. Modality classified for 94% of 35 pipeline programs.

Analysis

Trial Status

44

clinical trials

Completed25 · 57%
Active, not recruiting6 · 14%
Terminated4 · 9%
Unknown status3 · 7%
Recruiting3 · 7%
Withdrawn1 · 2%
Other2 · 5%

Clinical Trials

Trial Activity

44 trials

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)
Recruiting·DDenali Therapeutics Inc.·Phase 2/3·NCT05371613·63 enrolled
An Open-label Phase I/II Study of JR-446 in Mucopolysaccharidosis Type IIIB
Recruiting·JJCR Pharmaceuticals Co., Ltd.·Phase 1/2·NCT06488924·10 enrolled
A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
Recruiting·AAO GENERIUM·Phase 2/3·NCT05208281·32 enrolled
A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis II (STARLIGHT)
Active, not recruiting·JJCR Pharmaceuticals Co., Ltd.·Phase 3·NCT04573023·86 enrolled
Evaluation of Intravenous Laronidase Pharmacokinetics Before and After Hematopoietic Cell Transplantation in Patients With Mucopolysaccharidosis Type IH.
Active, not recruiting·MMasonic Cancer Center, University of Minnesota·N/A·NCT05634512·13 enrolled
An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type II
Active, not recruiting·JJCR Pharmaceuticals Co., Ltd.·Phase 2/3·NCT04348136·27 enrolled
An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II
Active, not recruiting·JJCR Pharmaceuticals Co., Ltd.·Phase 2·NCT03708965·19 enrolled
Phase I/II Study of JR-441 in Patients With Mucopolysaccharidosis Type IIIA
Active, not recruiting·JJCR Pharmaceuticals Co., Ltd.·Phase 1/2·NCT06095388·15 enrolled
Gene Therapy with Modified Autologous Hematopoietic Stem Cells for Patients with Mucopolysaccharidosis Type IIIA
Active, not recruiting·UUniversity of Manchester·Phase 1/2·NCT04201405·5 enrolled
A Global, Open-Label, Phase I/II Trial, to Evaluate Safety, Tolerability, Pharmacodynamic and Preliminary Efficacy of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)
Not yet recruiting·JJCR Pharmaceuticals Co., Ltd.·Preclinical·NCT07640984·12 enrolled
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
Enrolling by invitation·DDenali Therapeutics Inc.·Phase 2/3·NCT06075537·99 enrolled
Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I
Completed·Sanofi logoSanofi·Phase 3·NCT00912925·45 enrolled
Iduronate-2-sulfatase Enzyme Replacement Therapy in Mucopolysaccharidosis II (MPS II)
Completed·Takeda logoTakeda·Phase 2/3·NCT00069641·96 enrolled
Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease
Completed·Sanofi logoSanofi·Phase 3·NCT00258011·3 enrolled
A Study of JR-141 in Patients With Mucopolysaccharidosis II (BR21)
Completed·JJCR Pharmaceuticals Co., Ltd.·Phase 2·NCT03359213·18 enrolled
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Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)