VivoSeq
Other

Angelman Syndrome

15

Pipeline Programs↗

5

Active Trials↗

12

Companies Involved↗

Angelman syndrome is an emerging landscape with 15 programs across 12 companies. The most active players are Orion Corporation, Orion Pharma, Neuren Pharmaceuticals Limited, Merz Pharmaceuticals GmbH. 1 therapy is approved with 4 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

44/100

Pipeline Velocity

6/100

Mech. Concentration

33/100

Completion Rate

92/100

Attrition Rate

6/100

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.

Orion Corporation, Orion Pharma logo

Orion Corporation, Orion Pharma

1 program

Phase 1N

Neuren Pharmaceuticals Limited

1 program

Phase 2M

Merz Pharmaceuticals GmbH

1 program

Phase 2T

Triax

1 program

Phase 2Roche logo

Roche

2 programs

Phase 2Ultragenyx logo

Ultragenyx

1 program

Phase 3

Pipeline

Programs by Phase

Analysis

Modality Breakdown

94%

Small molecule

15 of 16 classified programs — the dominant modality here.

6%ALL OTHERSRNA therapy16%

Small molecule leads; the ring shows all 16 classified programs, each share of the total. Modality classified for 94% of 17 pipeline programs.

Analysis

Trial Status

18

clinical trials

Completed11 · 61%
Recruiting3 · 17%
Active, not recruiting1 · 6%
Terminated1 · 6%
Available1 · 6%
Enrolling by invitation1 · 6%

Clinical Trials

Trial Activity

18 trials

HALOS: A Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of Multiple Ascending Doses of ION582 in Participants With Angelman Syndrome
Recruiting·Ionis Pharmaceuticals logoIonis Pharmaceuticals·Phase 1/2·NCT05127226·70 enrolled
A Phase 2, Open-label, Basket Study Investigating the Safety and Efficacy of GTX-102 in Adult and Pediatric Subjects With Deletion- or Nondeletion-type Angelman Syndrome
Recruiting·Ultragenyx logoUltragenyx·Phase 2·NCT07157254·60 enrolled
Phase 3 Study of the Efficacy and Safety of ION582 in Children and Adults With Angelman Syndrome
Recruiting·Ionis Pharmaceuticals logoIonis Pharmaceuticals·Phase 3·NCT06914609·158 enrolled
Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With Angelman Syndrome (AS)
Active, not recruiting·Ultragenyx logoUltragenyx·Phase 3·NCT06617429·129 enrolled
Long-term Extension of GTX-102 in Angelman Syndrome
Enrolling by invitation·Ultragenyx logoUltragenyx·Phase 3·NCT06415344·255 enrolled
A Study to Investigate the Pharmacokinetics (PK) and Safety and to Provide Proof of Mechanism of Alogabat in Children and Adolescents Aged 5-17 Years With Angelman Syndrome (AS) With Deletion Genotype.
Completed·Roche logoRoche·Phase 2·NCT05630066·48 enrolled
Minocycline in the Treatment of Angelman Syndrome
Completed·UUniversity of South Florida·N/A·NCT01531582·25 enrolled
A Study in Adults and Adolescents With Angelman Syndrome (STARS)
Completed·HHealx AI·Phase 2·NCT02996305·88 enrolled
An Open-Label Study of the Safety, Tolerability, and Pharmacokinetics of Oral NNZ-2591 in Angelman Syndrome
Completed·NNeuren Pharmaceuticals Limited·Phase 2·NCT05011851·17 enrolled
A Study to Investigate the Safety, Tolerability, Pharmacokinetics (PK) and Pharmacodynamics (PD) of RO7248824 in Participants With Angelman Syndrome (AS)
Completed·Roche logoRoche·Phase 1·NCT04428281·74 enrolled
A Trial of Levodopa in Angelman Syndrome
Completed·—·Phase 2/3·NCT01281475·67 enrolled
Dietary Supplements for the Treatment of Angelman Syndrome
Completed·UUniversity of California, San Diego·N/A·NCT00348933·90 enrolled
Study to Evaluate the Efficacy and Safety of Minocycline in Angelman Syndrome
Completed·PPuerta de Hierro University Hospital·Phase 2·NCT02056665·32 enrolled
A Study of OV101 in Individuals With Angelman Syndrome (AS)
Completed·HHealx AI·Phase 3·NCT04106557·104 enrolled
Study on Tolerability of Levodopa/Carbidopa in Children With Angelman Syndrome
Completed·—·Phase 1·NCT00829439·16 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)