According to a story from PR Newswire, the biopharmaceutical company Biohaven Pharmaceutical Holding Company, Ltd. has announced that the first patient has signed up for the company’s phase 3 clinical trial testing their investigational product candidate verdiperstat. This drug is being tested as a treatment for the rare disease multiple system atrophy, a progressive, degenerative disorder. Biohaven is focused on the development of new therapies for neuropsychiatric and neurological diseases.
About Multiple System Atrophy (MSA)
Multiple system atrophy, which is also called Shy-Drager syndrome, is a neurodegenerative genetic disorder. It is most characterized by progressive loss of movement ability and voluntary muscle control. Unfortunately, little is known about what causes multiple system atrophy, and researchers have yet to identify any clear risk factors. Men are more likely to be affected. The symptoms are similar to Parkinson’s disease, but drugs that can control Parkinson’s symptoms have little effect. These symptoms include slowed movements, muscle stiffness, tremors, ataxia, and postural instability. Other symptoms including impotence, vocal palsy, urinary tract issues, dry mouth, and reduced ability to sweat may also appear. Treatment is mostly supportive; physical therapy, rehabilitation, and speech therapy are useful for maintaining movement and other capabilities. There is some disparity in the rate of disease progression, but most patients only survive between 6-10 years after their diagnosis. To learn more about multiple system atrophy, click here.
Verdiperstat is an inhibitor of myeloperoxidase, an enzyme that is linked to inflammation and oxidative stress in the brain. Prior trial results suggests that this mechanism of action can have a positive effect on patients with multiple system atrophy. The drug has earned Orphan Drug designation in both the US and EU.
There is a dire need for more effective treatment options for multiple system atrophy. Currently, there are no medications that can slow or halt the progression of the illness, which is ultimately lethal. Biohaven hopes to recruit a total of 250 multiple system atrophy patients for the clinical trial. The period of treatment evaluation with verdiperstat, which will be compared to a placebo, will last 48 weeks.