VivoSeq
Neurology

Cerebellar Ataxia

14

Pipeline Programs↗

7

Active Trials↗

12

Companies Involved↗

Cerebellar Ataxia is an emerging landscape with 14 programs across 12 companies. The most active players are CSL Behring, Azurity Pharmaceuticals, Cadent Therapeutics. 2 programs are in Phase 3.

Intelligence

Pipeline Metrics

Competitive Density

44/100

Pipeline Velocity

11/100

Mech. Concentration

33/100

Completion Rate

79/100

Attrition Rate

13/100

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.

CSL Behring logo

CSL Behring

1 program

Phase 1A

Azurity Pharmaceuticals

2 programs

Phase 2C

Cadent Therapeutics

1 program

Phase 2S

Sclnow Biotechnology Co., Ltd.

1 program

Phase 2S

Steminent Biotherapeutics Inc.

1 program

Phase 2Argenx logo

Argenx

1 program

Phase 2

Pipeline

Programs by Phase

Analysis

Modality Breakdown

80%

Small molecule

12 of 15 classified programs — the dominant modality here.

20%ALL OTHERSCell therapy213%Protein / enzyme17%

Small molecule leads; the ring shows all 15 classified programs, each share of the total. Modality classified for 100% of 18 pipeline programs.

Analysis

Trial Status

24

clinical trials

Completed11 · 46%
Recruiting4 · 17%
Unknown status2 · 8%
Terminated2 · 8%
Not yet recruiting2 · 8%
Available1 · 4%
Other2 · 8%

Clinical Trials

Trial Activity

24 trials

A Randomized, Parallel-arm, Double Blind, Placebo-controlled Study to Assess the Efficacy of Fampridine for Patients With Spinocerebellar Ataxia SCA27B Caused by a GAA Expansion in the FGF14 Gene
Recruiting·AAssistance Publique - Hôpitaux de Paris·Phase 3·NCT07185347·70 enrolled
The Benefits of Long-read High-throughput Genomic Sequencing for the Causal Diagnosis of Cerebellar Ataxias
Recruiting·—·N/A·NCT06467175·210 enrolled
Riluzole in Patients With Spinocerebellar Ataxia Type 7
Recruiting·SS. Andrea Hospital·Phase 2/3·NCT03660917·34 enrolled
Retraining Reaching in Cerebellar Ataxia
Recruiting·HHugo W. Moser Research Institute at Kennedy Krieger, Inc.·Preclinical·NCT03879018·18 enrolled
Real-World Data Study of Troriluzole-Treated Patients With Spinocerebellar Ataxia (SCA) Compared to a Matched Natural History Control
Active, not recruiting·BBiohaven Therapeutics Ltd.·N/A·NCT06529146·909 enrolled
Stemchymal® for Polyglutamine Spinocerebellar Ataxia
Not yet recruiting·SSteminent US, Inc.·Phase 2·NCT06397274·20 enrolled
Umbilical Cord Mesenchymal Stem Cells Therapy (19#iSCLife®-SA) for Patients With Spinocerebellar Ataxia
Not yet recruiting·SSclnow Biotechnology Co., Ltd.·Phase 2·NCT03378414·45 enrolled
Effect of Nilotinib in Cerebellar Ataxia Patients
Completed·SSeoul National University Hospital·Phase 2·NCT03932669·70 enrolled
Dalfampridine and Gait in Spinocerebellar Ataxias
Completed·UUniversity of Florida·N/A·NCT01811706·20 enrolled
Efficacy of Riluzole in Hereditary Cerebellar Ataxia
Completed·SS. Andrea Hospital·Phase 2/3·NCT01104649·60 enrolled
Efficacy and Safety Study of Stemchymal® in Polyglutamine Spinocerebellar Ataxia
Completed·SSteminent Biotherapeutics Inc.·Phase 2·NCT02540655·56 enrolled
Could Tai-chi Help Maintain Balance of Spinocerebellar Ataxia Patients
Completed·—·N/A·NCT03687190·21 enrolled
Safety and Efficacy of Intravenous Immune Globulin in Treating Spinocerebellar Ataxia
Completed·UUniversity of South Florida·Phase 2·NCT01350440·5 enrolled
Safety and Tolerability of Lithium in Spinocerebellar Ataxia 2 (SCA2)
Completed·FFederico II University·Phase 2·NCT00998634·20 enrolled
Effect of Riluzole as a Symptomatic Approach in Patients With Chronic Cerebellar Ataxia
Completed·SS. Andrea Hospital·Phase 2·NCT00202397·40 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)