VivoSeq
Neurology

Ataxia

32

Pipeline Programs↗

9

Active Trials↗

29

Companies Involved↗

Ataxia is a moderately active landscape with 32 programs across 29 companies. The most active players are Larimar Therapeutics, Inc., Santhera Pharmaceuticals, Solid Biosciences Inc.. 2 therapies are approved with 8 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

54/100

Pipeline Velocity

15/100

Mech. Concentration

9/100

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.

L

Larimar Therapeutics, Inc.

2 programs

Phase 1S

Santhera Pharmaceuticals

1 program

Phase 1S

Solid Biosciences Inc.

1 program

Phase 1M

Metro International Biotech, LLC

2 programs

Phase 2C

Changchun BCHT Biotechnology Co.

2 programs

Phase 2Biogen logo

Biogen

1 program

Phase 2

Pipeline

Programs by Phase

Analysis

Modality Breakdown

97%

Small molecule

31 of 32 classified programs — the dominant modality here.

3%ALL OTHERSCell therapy13%

Small molecule leads; the ring shows all 32 classified programs, each share of the total. Modality classified for 100% of 35 pipeline programs.

Analysis

Trial Status

51

clinical trials

Completed36 · 71%
Recruiting8 · 16%
Unknown status3 · 6%
Terminated2 · 4%
Withdrawn1 · 2%
Active, not recruiting1 · 2%

Clinical Trials

Trial Activity

51 trials

Acetyl-leucine in Post-stroke Ataxia
Recruiting·KKafrelsheikh University·Phase 3·NCT07275749·200 enrolled
A Study to Learn More About the Effects and Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old (BRAVE)
Recruiting·Biogen logoBiogen·Phase 3·NCT06953583·255 enrolled
An Open-Label Study of CTI-1601 in Subjects With Friedreich's Ataxia
Recruiting·LLarimar Therapeutics, Inc.·Phase 2·NCT06447025·85 enrolled
A Study to Find Out How BIIB141 (Omaveloxolone) is Processed in the Body and to Learn More About Its Safety in Participants With Friedreich's Ataxia Aged 2 to 15 Years Old
Recruiting·Biogen logoBiogen·Phase 1·NCT06054893·35 enrolled
A Study to Learn More About the Long-Term Safety of BIIB141 (Omaveloxolone) in Participants With Friedreich's Ataxia Who Are Prescribed it by Their Own Doctors
Recruiting·Biogen logoBiogen·N/A·NCT06623890·300 enrolled
A Multiple Ascending Dose Study of DT-216P2 in Patients With Friedreich's Ataxia
Recruiting·DDesign Therapeutics, Inc.·Phase 1/2·NCT06874010·20 enrolled
Adaptive Deep Brain Stimulation Targeting the Dentate Nucleus to Treat Spinocerebellar Ataxia
Recruiting·UUniversity of California, San Francisco·Preclinical·NCT07288437·5 enrolled
Adaptive Deep Brain Stimulation Targeting the Interposed Nucleus to Treat Spinocerebellar Ataxia
Recruiting·UUniversity of Florida·Preclinical·NCT07325487·5 enrolled
Natural History of Spinocerebellar Ataxia Type 7 (SCA7)
Active, not recruiting·National Eye Institute (NEI)·N/A·NCT02741440·22 enrolled
Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia
Completed·SSanthera Pharmaceuticals·Phase 3·NCT00537680·70 enrolled
Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients
Completed·SSanthera Pharmaceuticals·Phase 3·NCT00697073·68 enrolled
C-Trelin Orally Disintegrated(OD) Tablet 5mg in Ataxia Due to Spinocerebellar Degeneration
Completed·HHLB Pharmaceutical Co., Ltd.·Phase 4·NCT04107740·160 enrolled
Multiple Ascending Dose Study of CTI-1601 Versus Placebo in Subjects With Friedreich's Ataxia
Completed·LLarimar Therapeutics, Inc.·Phase 1·NCT04519567·27 enrolled
Efficacy of Epoetin Alfa in Patients With Friedreich's Ataxia
Completed·FFederico II University·Phase 2·NCT00631202·10 enrolled
Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone (PROTI)
Completed·SSanthera Pharmaceuticals·Phase 3·NCT01303406·29 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)