Krabbe Disease
Polaryx presented key pre-clinical data for PLX-200 their reformulated gemfibrozil drug in a Krabbe mouse model where it was found to reduce astrogliosis and neuro-inflammation, protect myelin, and reduce psychosine accumulation. Importantly, it also restored motor functions and increased lifespan in the Krabbe mice. These are encouraging results as later this year Polaryx will be launching their SOTERIA Phase 2 clinical trial for PLX-200 in patients to assess the safety, tolerability, and clinical activity of the drug across trial sites in the United States as well as in Europe and Asia. This comes after receiving a safe to proceed letter from the Food and Drug Administration (FDA) last year following their submission of an Investigational New Drug application.