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Person living with ALS in a motorized chair and their caregiver smiling

Kristina, living with ALS

Amyotrophic lateral sclerosis (ALS) is a relentlessly progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis and eventually, death.1,2 More than 90% of people with ALS have sporadic disease, showing no clear family history.3

We believe that it’s going to take a combination approach, targeting multiple cellular pathways implicated in disease pathogenesis, to find a cure for ALS. For this reason, we are investigating multiple therapies with different or potentially complementary pathways in ALS and other neurodegenerative diseases, in-house and in partnership with leading clinicians and researchers. This includes:

  • Amylyx' development of antisense oligonucleotides (ASOs), including lead ASO AMX0114 aimed at targeting calpain-2 (CAPN2), a gene encoding calcium-dependent proteolytic enzyme and a critical effector of axonal degeneration, which has been implicated in the pathogenesis of ALS and other neurodegenerative diseases. Learn more.
  • Amylyx' ongoing research agreement with Sunnybrook Research Institute (SRI) to expedite the identification of potentially novel drug compounds that inhibit Bax and Bak proteins, regulators of cell death pathways, with high potency, specificity, and appropriate pharmacokinetic properties for development as therapeutics for neurodegenerative diseases, specifically ALS. Learn more.
  • Amylyx' continued commitment to donate clinical trial data to patient registries, such as the PRO-ACT database, to share new information with the research community that could open up new pathways and approaches to developing new treatments. Learn more.
  • In designing our trials, we rely on the input of clinical leaders, advocacy groups, and the neurodegenerative disease community. This collaborative approach keeps people living with neurodegenerative diseases at the center of everything we do and is something we will continue in future trials.
  • PHOENIX (NCT05021536 / EudraCT 2021-000250-26) is a Phase 3, randomized, double-blind, placebo-controlled trial in 69 sites across the U.S. and Europe evaluating the safety and efficacy of AMX0035 in people living with amyotrophic lateral sclerosis (ALS). The trial is fully enrolled at this time, and participants completing the 48-week randomized phase will have the option to enroll in an Open Label Extension (OLE) Phase for up to two years.
  1. Brown, R. H., & Al-Chalabi, A. (2017). Amyotrophic Lateral Sclerosis. New England Journal of Medicine, 377(2), 162–172. https://doi.org/10.1056/nejmra1603471
  2. Al-Chalabi, A., Hardiman, O., Kiernan, M. C., Chiò, A., Rix-Brooks, B., & van den Berg, L. H. (2016). Amyotrophic lateral sclerosis: moving towards a new classification system. The Lancet Neurology, 15(11), 1182–1194. https://doi.org/10.1016/s1474-4422(16)30199-5
  3. ‌Chen, S., Sayana, P., Zhang, X., & Le, W. (2013). Genetics of amyotrophic lateral sclerosis: an update. Molecular Neurodegeneration, 8(1), 28. https://doi.org/10.1186/1750-1326-8-28

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.

Pipeline

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.