VivoSeq
Neurology

Ataxia-Telangiectasia

16

Pipeline Programs↗

3

Active Trials↗

7

Companies Involved↗

Ataxia-telangiectasia is an emerging landscape with 16 programs across 7 companies. The most active players are Novartis, Medexus Pharma, Inc., Quince Therapeutics S.p.A.. 3 therapies are approved with 3 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

44/100

Pipeline Velocity

6/100

Mech. Concentration

25/100

Completion Rate

86/100

Attrition Rate

7/100

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.

Novartis logo

Novartis

1 program

Phase 1M

Medexus Pharma, Inc.

1 program

Phase 2Q

Quince Therapeutics S.p.A.

2 programs

Phase 3Takeda logo

Takeda

1 program

ApprovedEMD Serono logo

EMD Serono

1 program

ApprovedGSK logo

GSK

1 program

Preclinical

Pipeline

Programs by Phase

Analysis

Modality Breakdown

89%

Small molecule

17 of 19 classified programs — the dominant modality here.

11%ALL OTHERSProtein / enzyme15%Vaccine15%

Small molecule leads; the ring shows all 19 classified programs, each share of the total. Modality classified for 100% of 21 pipeline programs.

Analysis

Trial Status

15

clinical trials

Completed6 · 40%
Unknown status4 · 27%
Active, not recruiting2 · 13%
Available1 · 7%
Terminated1 · 7%
Not yet recruiting1 · 7%

Clinical Trials

Trial Activity

15 trials

A Pivotal Study of N-Acetyl-L-Leucine on Ataxia-Telangiectasia (A-T)
Active, not recruiting·IIntraBio Inc·Phase 3·NCT06673056·60 enrolled
NAD Supplementation to Prevent Progressive Neurological Disease in Ataxia Telangiectasia
Active, not recruiting·UUniversity Hospital, Akershus·Phase 2·NCT04870866·13 enrolled
Safety and Efficacy of Mutation-targeted Precision Genetic Therapy for Ataxia-Telangiectasia (A-T)
Not yet recruiting·—·Phase 1/2·NCT07215416·10 enrolled
Amantadine for Improving Neurologic Symptoms in Ataxia-Telangiectasia
Completed·SSheba Medical Center·Phase 4·NCT00950196·30 enrolled
Study for Treatment of Cancer in Children With Ataxia-telangiectasia
Completed·SSt. Jude Children's Research Hospital·N/A·NCT00187057·6 enrolled
Conjugate Pneumococcal Vaccine in Ataxia Telangiectasia (AT)
Completed·IInstitute of Child Health·Phase 3·NCT00656409·30 enrolled
Intra-Erythrocyte Dexamethasone Sodium Phosphate in Ataxia Telangiectasia Patients
Completed·QQuince Therapeutics S.p.A.·Phase 3·NCT02770807·176 enrolled
Baclofen Treatment of Ataxia Telangiectasia
Completed·JJohns Hopkins University·Phase 1·NCT00640003·10 enrolled
Response of Individuals With Ataxia-Telangiectasia to Metformin and Pioglitazone
Completed·NNHS Tayside·Phase 4·NCT02733679·27 enrolled
N-Acetyl-L-Leucine for Ataxia-Telangiectasia (A-T)
Terminated·IIntraBio Inc·Phase 2·NCT03759678·17 enrolled
Ataxia-telangiectasia: Treating Mitochondrial Dysfunction With Nicotinamide Riboside
Unknown status·TThe University of Queensland·N/A·NCT06324877·10 enrolled
MBM-01 (Tempol) for the Treatment of Ataxia Telangiectasia
Unknown status·MMatrix Biomed, Inc.·Phase 2·NCT04887311·20 enrolled
Expanded Access Program (EAP) of Levacetylleucine for Ataxia-Telangiectasia (A-T)
Available·IIntraBio Inc·N/A·NCT07380165
Immunogenicity of Pneumococcal Vaccines in Ataxia-telangiectasia Patients
Unknown status·SSheba Medical Center·N/A·NCT01075438·20 enrolled
Status of Growth Hormone/ Insulin-like Growth Factor-1 (GH/IGF-1) Axis and Growth Failure in Ataxia Telangiectasia (AT)
Unknown status·JJohann Wolfgang Goethe University Hospital·Phase 4·NCT01052623·24 enrolled

Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)