Amylyx Pharmaceuticals Inc.

Address: 43 Thorndike Street

Sponsor Overview

assignment

Specializations

Amyotrophic Lateral Sclerosis and Motor Neuron Disease

Clinical research activity includes ALS with attention to disease progression, functional decline, and treatment effects in adult patients.

Disease progression in amyotrophic lateral sclerosis
Functional outcomes in motor neuron disease
Therapeutic evaluation of AMX0035

The sponsor’s work in this area centers on neurological conditions marked by progressive loss of motor function and disability.

Progressive Supranuclear Palsy and Neurodegeneration

Research also extends to progressive supranuclear palsy, with interest in clinical measures of symptom burden and neurological deterioration.

PSP clinical assessment
Neurological progression markers
Treatment response in neurodegenerative disease

This reflects involvement in disorders affecting movement, balance, eye control, and cognition.

Pharmacokinetics in Renal Impairment

Studies include participants with renal impairment to examine how treatment exposure changes in the setting of reduced kidney function.

Renal function and drug handling
Medication exposure in kidney impairment
Comparative assessment in healthy participants

These investigations support understanding of treatment use across populations with altered renal clearance.

Pharmacokinetics in Hepatic Impairment

Additional research focuses on hepatic impairment, examining treatment behavior in individuals with compromised liver function alongside healthy volunteers.

Liver function and medication exposure
Hepatic impairment studies
Safety considerations in altered metabolism

This area addresses how hepatic dysfunction may influence therapeutic handling and clinical use.

Healthy Volunteers and Clinical Reference Populations

Trials also involve healthy volunteers as reference participants for comparison with patients and individuals with organ impairment.

Healthy participant comparisons
Reference populations in clinical research
Baseline evaluation of treatment exposure

These studies support interpretation of therapeutic findings across neurological and organ impairment settings.

Trial Name	Country	Status
Evaluation of the Safety and Efficacy of AMX0035 (Phenylbutyrate and Ursodoxicoltaurine) in Patients with Progressive Supranuclear Palsy: A Phase 2b/3 Study	Austria Belgium Bulgaria France Germany Italy Poland Spain Sweden The Netherlands	Not Recruiting
Evaluation of the Safety and Efficacy of AMX0035 (Phenylbutyrate, Ursodoxicoltaurine) in Adult Patients with Amyotrophic Lateral Sclerosis	Belgium France Germany Ireland Italy Poland Portugal Spain Sweden The Netherlands	Not Recruiting
Pharmacokinetic Evaluation of AMX0035 in Subjects with Hepatic Impairment and Healthy Controls	Bulgaria	Not Recruiting
Pharmacokinetic Evaluation of AMX0035 in Subjects with Renal Impairment and Healthy Controls	Bulgaria	Not Recruiting