Drug intelligence / Profile preview

JRMS-22

Development stage
Preclinical
Lead developer
Neuropeutics
Modality
Small Molecules
Administration
Oral
01

Overview

JRMS-22 is a preclinical small molecule drug developed to prevent and reverse toxic protein aggregation, specifically targeting TDP-43 pathology. TDP-43 (TAR DNA-binding protein 43) mislocalization and aggregation are implicated in the majority of amyotrophic lateral sclerosis (ALS) cases as well as other neurodegenerative diseases. JRMS-22 was discovered by researchers at the University of Toronto and further developed by Neuropeutics Inc, with ongoing collaboration with LifeArc. The compound has demonstrated strong efficacy in reducing TDP-43 aggregation by about 30% after two weeks of treatment in mouse models, showing promise for disease modification in ALS and potentially other disorders characterized by pathological protein aggregation such as frontotemporal dementia, Alzheimer’s disease, Parkinson’s disease, Huntington’s disease, dementia with Lewy bodies, and multiple system atrophy[1][2][5][6][8].

02

Targets

TARDBP (TAR DNA-binding protein 43)

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