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Verdiperstat is a first-in-class, potent, selective, brain-permeant small molecule that acts as an irreversible inhibitor of myeloperoxidase (MPO), an enzyme involved in the generation of reactive oxygen species and implicated in neuroinflammation and oxidative stress. By inhibiting MPO, verdiperstat is hypothesized to reduce microglial activation and neurodegeneration associated with diseases such as multiple system atrophy (MSA) and amyotrophic lateral sclerosis (ALS). Verdiperstat has been investigated primarily for MSA but also for other neurodegenerative conditions. It has received Fast Track and Orphan Drug designations from the FDA and EMA due to unmet medical needs in MSA[3][5][4].
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