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BIIB105
- Development stage
- Discontinued
- Lead developer
- Biogen
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
- Administration
- Intrathecal
01
Overview
BIIB105 is an investigational antisense oligonucleotide (ASO) developed for the treatment of amyotrophic lateral sclerosis (ALS). The drug was designed to reduce the expression of ataxin-2 (ATXN2) protein by binding to its messenger RNA and promoting its degradation. This mechanism aimed to lower ataxin-2 levels in cells, potentially reducing toxic TDP-43 protein aggregates that are implicated in nearly all ALS cases. Despite achieving significant reductions in ATXN2 protein levels during clinical trials, BIIB105 failed to demonstrate clinical benefit on functional outcomes or biomarkers such as neurofilament light chain. As a result, development was discontinued following negative results from a Phase 1/2 trial[1][3][5][7].
- Other names
- ATXN2-ASOATXN-2-ASOATXN 2-ASOAtaxin-2 antisense oligonucleotideAtaxin2 antisense oligonucleotideAtaxin 2 antisense oligonucleotide
02
Targets
ATXN2 (Ataxin-2)
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