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Wolfram Syndrome (WS) is an autosomal recessive neurodegenerative disease characterized by juvenile-onset diabetes mellitus, optic nerve atrophy, deafness, and diabetes insipidus. The prognosis of WS is poor, and many people with the disease die prematurely with severe neurological disabilities.1,2

WS is considered a prototypical endoplasmic reticulum (ER) stress disorder. Dysregulation of mitochondrial dynamics and function have also been shown to contribute to disease pathogenesis.2 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.3

As part of our commitment to explore the full potential of AMX0035 in neurodegenerative diseases, we have initiated HELIOS (NCT05676034), a Phase 2 clinical trial in Wolfram Syndrome (WS), which has no known cure.

  1. Urano F. (2016). Wolfram Syndrome: Diagnosis, Management, and Treatment. Current diabetes reports, 16(1), 6.
  2. Pallotta, M.T., Tascini, G., Crispoldi, R. et al. Wolfram syndrome, a rare neurodegenerative disease: from pathogenesis to future treatment perspectives. J Transl Med 17, 238 (2019).
  3. ‌Kitamura, R. A., Maxwell, K. G., Ye, W., Kries, K., Brown, C. M., Augsornworawat, P., Hirsch, Y., Johansson, M. M., Weiden, T., Ekstein, J., Cohen, J., Klee, J., Leslie, K., Simeonov, A., Henderson, M. J., Millman, J. R., & Urano, F. (2022). Multidimensional analysis and therapeutic development using patient iPSC–derived disease models of Wolfram syndrome. JCI Insight, 7(18).

Our commitment

At Amylyx, we are relentlessly driven by a commitment to those living with neurodegenerative diseases. We work collaboratively across everything we do, aspiring to help support and create more moments for the neurodegenerative community.


Our innovative approach to developing transformative therapeutics has far-reaching potential to improve the management of multiple neurodegenerative diseases. Learn more about our growing pipeline.