The myeloperoxidase (MPO) enzyme is a key driver of oxidative and inflammatory processes. MPO levels are increased in a range of brain disorders, and MPO inhibition is a promising therapeutic strategy for neuroinflammatory and neurodegenerative conditions, including multiple system atrophy (MSA) and amyotrophic lateral sclerosis (ALS). Studies have also linked increased MPO levels with multiple sclerosis and Alzheimer’s disease.
Verdiperstat is an inhibitor of myeloperoxidase (MPO). The company anticipates launching the drug for these two orphan indications in 2023.
ALS, which is sometimes known as Lou Gehrig’s disease in the U.S., has an estimated 30,000 patients in China. The only approved ALS therapy is riluzole, which is used by about one-third of ALS patients. However, riluzole is not disease-modifying, is poorly tolerated, and has a limited survival benefit.
MSA is a rare, rapidly progressing neurodegenerative disease with Parkinson’s-like symptoms affecting about 50,000 patients in China and no disease-modifying therapies.
Other life-cycle opportunities for Verdiperstat include Parkinson’s disease and Alzheimer’s disease.