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Icerguastat
- Development stage
- Phase 2
- Lead developer
- InFlectis BioScience
- Modality
- Small Molecules
- Administration
- Oral
01
Overview
Icerguastat is an orally available, first-in-class small molecule developed primarily for the treatment of rare neurodegenerative diseases such as Charcot-Marie-Tooth disease and amyotrophic lateral sclerosis. It is a derivative of guanabenz acetate with improved side effect profile. The drug acts as a selective inhibitor of the protein phosphatase 1 regulatory subunit 15A (PPP1R15A), thereby modulating the unfolded protein response (UPR) and reducing endoplasmic reticulum stress. This mechanism has shown potential in reducing muscle degeneration and protein aggregation in preclinical models. Icerguastat has received orphan drug designation for Charcot-Marie-Tooth disease and amyotrophic lateral sclerosis[3][4][5][7].
- Other names
- IFB-088IFB088IFB 088sephin1sephin-1sephin 1
02
Targets
GRIN2B (N-methyl-D-aspartate Receptor Subunit Combination: NR1a/NR2B)PPP1R15A (Protein phosphatase 1 regulatory subunit 15A)
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