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With every step forward, there is hope

Every day represents an opportunity to alter the course of relentlessly progressive neurodegenerative diseases and the people they affect – and a responsibility to improve the lives of people, families, and communities around the world. 

Pipeline

We are committed to exploring the full potential of our existing and exploratory therapies and advancing our pipeline in an effort to support our mission of one day ending the suffering caused by neurodegenerative diseases.

AMX0035 Pipeline Graphic

We are relentless in our pursuit of new therapies and advancing science as part of the fight against neurodegenerative diseases, and we are currently exploring new approaches and compounds in-house and in partnership with leading clinicians and researchers. This includes:

  • Amylyx' development of antisense oligonucleotides (ASOs), including lead ASO AMX0114, which is aimed at targeting calpain-2 (CAPN2), a gene encoding calcium-dependent proteolytic enzyme which has been implicated in the pathogenesis of ALS and other neurodegenerative diseases.

  • Amylyx' ongoing research agreement with Sunnybrook Research Institute (SRI) to expedite the identification of potentially novel drug compounds that inhibit Bax and Bak 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.

Explore information about our ongoing clinical trial:

We are relentless in our pursuit of new therapies and advancing science as part of the fight against neurodegenerative diseases, and we are currently exploring new approaches and compounds in-house and in partnership with leading clinicians and researchers. This includes:

  • Amylyx' development of antisense oligonucleotides (ASOs), including lead ASO AMX0114, which is aimed at targeting calpain-2 (CAPN2), a gene encoding calcium-dependent proteolytic enzyme which has been implicated in the pathogenesis of ALS and other neurodegenerative diseases.

  • Amylyx' ongoing research agreement with Sunnybrook Research Institute (SRI) to expedite the identification of potentially novel drug compounds that inhibit Bax and Bak 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.

Explore information about our ongoing clinical trial:

White logo of PHOENIX

PHOENIX Phase 3 Study

PHOENIX (NCT05021536) is a Phase 3, randomized, placebo-controlled trial in approximately 65 sites across the U.S. and Europe designed to evaluate the safety and efficacy of sodium phenylbutyrate and taurursodiol (also known as ursodoxicoltaurine) in ALS. Recruitment for this trial is closed in the U.S. and ongoing in Europe.

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Be the change

Amylyx employees on stairs wearing medical masks

Be the change

For as long as people have to fight these diseases, we will be relentlessly working towards the development of new therapies. In order to do that, we need your help. If you're just as driven to make an impact, we are looking for people to join our fast-growing team.