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Other/Down Syndrome

Down Syndrome

18

Pipeline Programs↗

24

Active Trials↗

21

Companies Involved↗

Down syndrome is an emerging landscape with 18 programs across 21 companies. 1 program is in Phase 3.

Intelligence

Pipeline Metrics

Competitive Density

51/100

Pipeline Velocity

43/100

Mech. Concentration

9/100

Completion Rate

75/100

Attrition Rate

15/100

Pipeline

Programs by Phase

Preclinical

No programs

Phase 11

MIB-626

Metro International Biotech, LLC

Phase 216

Tofacitinib

Prism Pharmaceuticals

Ublituximab

TG Therapeutics, Inc.

Asparaginase Erwinia Chrysanthemi

St. Jude Children's Research Hospital

Blinatumomab

Amgen

Calaspargase Pegol

Servier

Phase 31

Levetiracetam

UCB

Approved

No programs

Analysis

Modality Breakdown

65%

Small molecule

11 of 17 classified programs — the dominant modality here.

Small molecule leads; the ring shows all 17 classified programs, each share of the total. Modality classified for 94% of 18 pipeline programs.

Analysis

Trial Status

66

clinical trials

Completed30 · 45%

Recruiting13 · 20%

Not yet recruiting7 · 11%

Terminated7 · 11%

Withdrawn3 · 5%

Active, not recruiting3 · 5%

Other3 · 5%

Clinical Trials

Trial Activity

66 trials

Collection of Whole Blood Specimens From Pregnant Women at Increased Risk of Fetal Chromosomal Abnormality for Use in Development of a Noninvasive Prenatal Test in the Detection of the Relative Quantity of Chromosomal Material in Circulating Cell-Free DNA Extracted From Maternal Plasma

Recruiting·SSequenom, Inc.·N/A·NCT01429389·2,000 enrolled

Modulation of the Immune System in Down Syndrome for Improved Outcomes and Neurodevelopment - 1

Recruiting·UUniversity of Colorado, Denver·Phase 2·NCT07598643·92 enrolled

A Phase 2 Study of Blinatumomab (NSC# 765986) in Combination With Nivolumab (NSC# 748726), a Checkpoint Inhibitor of PD-1, in B-ALL Patients Aged >/= 1 to < 31 Years Old With First Relapse

Recruiting·National Cancer Institute (NCI)·Phase 2·NCT04546399·461 enrolled

Effect of Sensory Integration on Gross Motor Function and Balance in Children With Down Syndrome

Recruiting·—·Preclinical·NCT07785011·40 enrolled

The Neurocognitive Bases of Trust in Intellectual Disability: Affective Evaluation, Trait Attribution, and Epistemic Vigilance

Recruiting·HHospices Civils de Lyon·N/A·NCT07434037·112 enrolled

Adapting the RDAD Intervention for Individuals With Down Syndrome Phase 3- Pilot Test

Recruiting·UUniversity of Kansas Medical Center·Preclinical·NCT07425769·40 enrolled

Brain-directed Treatment to Improve Language in Adolescents and Young Adults With Down Syndrome: the Efficacy of Transcranial Direct Current Stimulation Coupled With Linguistic Training.

Recruiting·—·Preclinical·NCT07044804·36 enrolled

Feasibility and Outcome Measures for Infants With Down Syndrome: Advancing Clinical Trial Readiness for a Harness-based Mobility Intervention

Recruiting·BBoston University Charles River Campus·Preclinical·NCT06219863·6 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

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

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

HomeGrown: A Family-based Lifestyle Intervention to Support Healthy Development of Young Children With Down Syndrome

Active, not recruiting·UUNC Lineberger Comprehensive Cancer Center·Preclinical·NCT07296861·38 enrolled

Effects of Hypoglossal Nerve Stimulation on Cognition and Language in Down Syndrome and Obstructive Sleep Apnea

Active, not recruiting·IInspire Medical Systems, Inc.·Preclinical·NCT04801771·57 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)