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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.

Wolfram syndrome is a rare, genetic, and fatal neurodegenerative disease that progressively impacts multiple organs and systems. Wolfram is considered a prototypical endoplasmic reticulum (ER) stress disorder in which ER stress and mitochondrial dysfunction are driving the underlying disease pathophysiology. The prognosis of Wolfram is poor, and many people with the disease die prematurely.

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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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.

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

AMX0114

Antisense Oligonucleotide

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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.