Verdiperstat is a brain-penetrant, irreversible myeloperoxidase (MPO) inhibitor that is being developed for the treatment of multiple system atrophy (MSA) and amyotrophic lateral sclerosis (ALS).

Other life-cycle opportunities for Verdiperstat include Parkinson’s disease and Alzheimer’s disease.

Multiple System Atrophy (MSA)

Multiple system atrophy is a rare, rapidly progressive, and fatal neurodegenerative disease that leads to death after an average of 6 to 10 years from disease onset. MSA causes Parkinson’s disease-like movement problems (e.g., slow movement, rigid muscles, tremor, and poor balance) as well as problems with involuntary (autonomic) functions, including blood pressure control, bladder function, and digestion. There is no cure for MSA. Only symptomatic therapies are available. MSA is affecting about 50,000 patients in China.

Amyotrophic Lateral Sclerosis (ALS)

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.

Verdiperstat Overview


Click to expend brain-penetrant, irreversible inhibitor of MPO
  • Has the potential to be developed in a number of disease indications associated with oxidative stress, inflammation, and neurodegeneration.
  • Potential to be a best-in-class agent.
  • Licensed from AstraZeneca in September 2018, where it was known as AZD3241.