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NCT04494256 (BIIB105 in Amyotrophic Lateral Sclerosis and Participants With…): BVCT ex-ante prediction vs. reported outcome

On 23 Apr 2024, BioinvestGPT's BVCT predicted technical failure and commercial failure for this trial. The 16 May 2024 readout is classified on the dashboard as True Negative (TN), a correct prediction. Recorded clinical-benefit conclusion: statistically insignificant and commercially insufficient.

Clinical Trial

NCTID
NCT04494256
Acronym
ALSpire
Drug Name
BIIB105
Drug MoA
anti-ATXN2 ASO
Drug Modality
small interfering RNA (siRNA) antisense oligonucleotide
Drug Class
First-In-Class
Therapeutic Area
Nervous System Diseases
Indication
Amyotrophic Lateral Sclerosis and Participants With the ALS Ataxin-2 (ATXN2) Genetic Mutation
Human Patients
99
Sponsor
Biogen / Ionis Pharmaceuticals
Phase
Phase 1 Phase 2

Prediction of , index 1566

Prospective Prediction
Prediction Index
1566
Prediction Date
Commercial Prediction
FAILURE (commercially insufficient additive clinical benefit to SoC regardless of the CAG repeat expansion level in the ATXN2 gene)
Technical Prediction
FAILURE (statistically insignificant additive clinical benefit to SoC regardless of the CAG repeat expansion level in the ATXN2 gene)

Readout of

Prediction Validation
Readout Date
Prediction To Readout
23 days in advance
Readout Data Interpretation
no reduction in ALS biomarkers; discontinued.
Press Release
Prediction Result
Prediction Accuracy
Correct Prediction
Clinical Benefit Conclusion
statistically insignificant and commercially insufficient
Prediction Classification
True Negative (TN)

ClinicalTrials.gov record

ClinicalTrials.gov record, retrieved 2026-09-24. Third-party data.

Official title
A Phase 1/2 Multiple-Ascending-Dose Study With a Long-Term Open-Label Extension to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Effect on Disease Progression of BIIB105 Administered Intrathecally to Adults With Amyotrophic Lateral Sclerosis With or Without Poly-CAG Expansion in the ATXN2 Gene
Phase
Phase 1, Phase 2
Status
Terminated
Enrollment
99
Lead sponsor
Biogen
Conditions
Amyotrophic Lateral Sclerosis
Interventions
DRUG: BIIB105; DRUG: Placebo
Design
Randomized · Sequential · Triple
Primary purpose
Treatment
Start
2020-09-28
Primary completion
2024-08-13
Completion
2024-08-13
Results first posted
2025-10-09
Primary outcomes
  • Part 1: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Treatment-Emergent Serious Adverse Events (TESAEs) (From first dose of the study drug in Part 1 up to end of follow up period in Part 1 (up to Day 260))
  • Part 2: Number of Participants With TEAEs and TESAEs (From first dose of the study in Part 2 up to end of follow up period in Part 2 (up to Day 1184))

NCT04494256 on ClinicalTrials.gov

Return to the dashboardClinicalTrials.gov

How to read these records: evidence · methodology · all trials