HELIOS Phase 2 Clinical Trial

There are currently no approved therapies for the approximately 3,000 people in the U.S., and more around the world, living with Wolfram syndrome. The majority of people with Wolfram syndrome carry mutations in the WFS1 gene, which encodes a protein called wolframin that spans the membrane of the endoplasmic reticulum (ER). Loss of wolframin function leads to ER stress and impaired mitochondrial dynamics, which in turn leads to dysfunction and apoptosis of cells. Because of the clear link between WFS1 mutations and ER stress, Wolfram syndrome is considered a prototypical ER stress disorder.^{1, 5-9}

AMX0035 is hypothesized to reduce neuronal cell death in Wolfram syndrome by simultaneously mitigating ER stress and mitochondrial dysfunction. Preclinical studies have provided evidence that AMX0035 may reduce cell death and improve cellular function.¹⁰

As part of our commitment to explore the full potential of AMX0035 in neurodegenerative diseases, we initiated HELIOS (NCT05676034), a Phase 2 clinical trial designed to evaluate if AMX0035 slows progression of diabetic, visual, and other measures in people with Wolfram syndrome, and to evaluate safety and tolerability.