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Pipeline

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

There is still much to learn about ALS, and R&D is a critical pillar of how we’ll deliver on our mission to one day end the suffering caused by neurodegenerative diseases.

Learn more about our commitment to research in ALS: amylyx.com/ALS

Preclinical
IND-Enabling Studies
Phase 1
Phase 2
Phase 3
Commercial

AMX0035

Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO, also known as Ursodoxicoltaurine)

RELYVRIO® brand name in US ALBRIOZA™ brand name in Canada

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AMX0114

Antisense Oligonucleotide

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Bax and Bak protein inhibitors

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Progressive supranuclear palsy (PSP) is a rare, progressive, and adult-onset neurological disorder that affects walking and balance, eye movement, swallowing, and speech. People living with PSP have a life expectancy of six to eight years after initial diagnosis, and the epidemiology of PSP is similar to that of ALS. PSP is characterized by abnormal tau inclusions and is consequently also known as a tauopathy.

Learn more about our research in PSP: amylyx.com/PSP

Preclinical
IND-Enabling Studies
Phase 1
Phase 2
Phase 3
Commercial

AMX0035

Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO, also known as Ursodoxicoltaurine)

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Hover over Tap Icon Information for more information including regional details.

Wolfram syndrome (WS) is an autosomal recessive neurodegenerative disease characterized by juvenile onset diabetes mellitus, optic nerve atrophy, deafness, and diabetes insipdius. WS is considered a prototypical endoplasmic reticulum (ER) stress disorder. The prognosis of WS is poor, and many people with the disease die prematurely with severe neurological disabilities.

Preclinical
IND-Enabling Studies
Phase 1
Phase 2
Phase 3
Commercial

AMX0035

Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO, also known as Ursodoxicoltaurine) 

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Hover over Tap Icon Information for more information including regional details.

Alzheimer’s Disease is a relentlessly progressive disease affecting the memories, thoughts and behavior of millions of people worldwide. Through our research, we aim to further characterize the biological activity of our candidates to inform potential clinical development.

Preclinical
IND-Enabling Studies
Phase 1
Phase 2
Phase 3
Commercial

AMX0035

Sodium Phenylbutyrate (PB) and Taurursodiol (TURSO, also known as Ursodoxicoltaurine) 

Information icon

Hover over Tap Icon Information for more information including regional details.

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.