VivoSeq
Other

Down Syndrome

39

Pipeline Programs↗

9

Active Trials↗

18

Companies Involved↗

Down syndrome is a moderately active landscape with 39 programs across 18 companies. The most active players are Perha Pharmaceuticals, Ionis Pharmaceuticals, Novartis. 4 therapies are approved with 8 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

57/100

Pipeline Velocity

22/100

Mech. Concentration

9/100

Completion Rate

74/100

Attrition Rate

20/100

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

Perha Pharmaceuticals

1 program

Phase 1Ionis Pharmaceuticals logo

Ionis Pharmaceuticals

1 program

Phase 1Novartis logo

Novartis

1 program

Phase 1M

Metro International Biotech, LLC

1 program

Phase 1AbbVie logo

AbbVie

2 programs

Phase 2V

Validus Pharmaceuticals

1 program

Phase 2

Pipeline

Programs by Phase

Analysis

Modality Breakdown

93%

Small molecule

39 of 42 classified programs — the dominant modality here.

7%ALL OTHERSProtein / enzyme25%Radiopharmaceutical12%

Small molecule leads; the ring shows all 42 classified programs, each share of the total. Modality classified for 98% of 46 pipeline programs.

Analysis

Trial Status

46

clinical trials

Completed26 · 57%
Terminated7 · 15%
Recruiting6 · 13%
Withdrawn2 · 4%
Not yet recruiting2 · 4%
Unknown status2 · 4%
Other1 · 2%

Clinical Trials

Trial Activity

46 trials

Efficacy of Bumetanide to Improve Cognitive Functions in Down Syndrome
Recruiting·—·Phase 2·NCT06465823·64 enrolled
GnRH Therapy on Cognition in Down Syndrome
Recruiting·—·Phase 2/3·NCT04390646·56 enrolled
Evaluating Treatment of ADHD in Children with Down Syndrome
Recruiting·—·Phase 4·NCT04219280·100 enrolled
LEvetiracetam to Prevent Seizures in Symptomatic Alzheimer's Disease in Adults With Down Syndrome
Recruiting·FFundació Institut de Recerca de l'Hospital de la Santa Creu i Sant Pau·Phase 3·NCT07234695·120 enrolled
Natural History of Dysregulation and Aging of the Immune System in People With Trisomy 21 With and Without Thymectomy
Recruiting·National Institute of Allergy and Infectious Diseases (NIAID)·N/A·NCT07416201·700 enrolled
Cholinergic Integrity in Down Syndrome in Association With Aging, Alzheimer's Disease Pathology, and Cognition
Recruiting·VVanderbilt University Medical Center·Phase 2·NCT05231798·30 enrolled
Endotype DIrected Treatment for OSA in Down Syndrome
Not yet recruiting·UUniversity of Arizona·Phase 4·NCT07280468·200 enrolled
AEF0217 in Participants With Down Syndrome
Not yet recruiting·—·Phase 2·NCT07334912·188 enrolled
Integration and Equal Opportunities for People With Down Syndrome: An Innovative Proposal Through Immersive Virtual Reality
Enrolling by invitation·University of Vigo·Preclinical·NCT07668908·20 enrolled
A Double-blind, Placebo-controlled Comparative Study and Open-label Extension Study to Confirm the Efficacy and Safety of E2020 in Subjects With Down Syndrome Having Regression Symptoms and Disabled Activities of Daily Living.
Completed·Eisai logoEisai·Phase 2·NCT02094053·36 enrolled
Efficacy of Rivastigmine in Patients With Down Syndrome
Completed·NNational Taiwan University Hospital·N/A·NCT00748007·14 enrolled
Tofacitinib for Immune Skin Conditions in Down Syndrome
Completed·UUniversity of Colorado, Denver·Phase 2·NCT04246372·47 enrolled
Multicenter Vitamin E Trial in Aging Persons With Down Syndrome
Completed·NNew York State Institute for Basic Research·Phase 3·NCT01594346·349 enrolled
The Effects of Inhalational Anaesthetics in Cognitive Functions in Down Syndrome Patients
Completed·—·Phase 2·NCT02971254·39 enrolled
Investigation of the Safety of Intranasal Glulisine in Down Syndrome
Completed·HHealthPartners Institute·Phase 1·NCT02432716·12 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)