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UKEvery 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.
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 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.
- 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.
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 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.
- 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.
Explore information about our ongoing clinical trial:
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.
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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.
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