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An Open-Label, Multicenter, Extension Study to Evaluate the Long-Term Safety and Efficacy of Dexpramipexole (BIIB050) in Subjects With Amyotrophic Lateral Sclerosis
Source-linked diligence brief with registry provenance, taxonomy normalization and premium analytical context.
Generated
Jun 24, 2026
Report code
NCT01622088-Jun 24, 2026
NCT ID
NCT01622088
Status
TERMINATED
Phase
Phase 3
Sponsor
Knopp Biosciences
Executive brief
Investment-Ready Snapshot
Dexpramipexole (BIIB050) was developed by Knopp Biosciences for the treatment of ALS. The Phase 3 extension study aimed to evaluate long-term safety and efficacy but was terminated due to the failure of the initial Phase 3 study (NCTO1281189) to meet its primary efficacy endpoint. This raises significant concerns regarding the commercial viability of dexpramipexole in the ALS market, which is characterized by high unmet medical need but also intense competition from emerging therapies. The termination may impact investor confidence and future funding opportunities for Knopp Biosciences.
Source & freshness
Provenance
https://clinicaltrials.gov/study/NCT01622088
Indication
Amyotrophic Lateral Sclerosis
Modality
small molecule
Target
Dexpramipexole targets the mitochondrial protein, preventing apoptosis in motor neurons, which is relevant in the context of Amyotrophic Lateral Sclerosis (ALS).
Intervention
Dexpramipexole
Source record
Protocol Description
Detailed source ingestion pending.
Source record
Outcome Measures
Detailed source ingestion pending.
Source record
Eligibility
Detailed source ingestion pending.
AI analysis
Known Results And Readout Context
Detailed source ingestion pending.
IP intelligence
Patent And IP Landscape
Detailed source ingestion pending.
Source record
Contacts
Detailed source ingestion pending.