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Amylyx Pharmaceuticals

Amylyx Pharmaceuticals logo
🇺🇸United States
Ownership
Public
Established
2014-01-01
Employees
384
Market Cap
$145M
Website
http://www.amylyx.com

Clinical Trials

27

Active:16
Completed:5

Trial Phases

3 Phases

Phase 1:15
Phase 2:6
Phase 3:5

Drug Approvals

1

FDA:1

Drug Approvals

RELYVRIO

Approval Date
Jan 2, 2024
FDA

Clinical Trials

Distribution across different clinical trial phases (26 trials with phase data)• Click on a phase to view related trials

Phase 1
15 (57.7%)
Phase 2
6 (23.1%)
Phase 3
5 (19.2%)

Avexitide for Treatment of Post-Bariatric Hypoglycemia

Phase 3
Recruiting
Conditions
Post Bariatric Hypoglycemia
Interventions
Other: Placebo
First Posted Date
2024-12-24
Last Posted Date
2025-07-02
Lead Sponsor
Amylyx Pharmaceuticals Inc.
Target Recruit Count
75
Registration Number
NCT06747468
Locations
🇺🇸

Stanford Health Care - Endocrinology Clinic, Stanford, California, United States

🇺🇸

University of Colorado Health Anschutz Medical Campus, Aurora, Colorado, United States

🇺🇸

East Coast Institute for Research, Jacksonville, Florida, United States

and more 18 locations

AMX0114 in Adult Participants With Amyotrophic Lateral Sclerosis

Phase 1
Recruiting
Conditions
ALS
Interventions
Other: Placebo
First Posted Date
2024-10-30
Last Posted Date
2025-07-08
Lead Sponsor
Amylyx Pharmaceuticals Inc.
Target Recruit Count
48
Registration Number
NCT06665165
Locations
🇺🇸

University of California, San Diego, La Jolla, California, United States

🇺🇸

Orlando Regional Medical Center, Orlando Health Neuroscience Institute, Orlando, Florida, United States

🇺🇸

Georgetown University Hospital Pasquerilla Healthcare Center, Washington, District of Columbia, United States

and more 6 locations

AMX0035 and Progressive Supranuclear Palsy

Phase 2
Active, not recruiting
Conditions
Neurodegenerative Diseases
Atypical Parkinsonism
PSP
Progressive Supranuclear Palsy
Interventions
Other: Placebo
First Posted Date
2023-11-08
Last Posted Date
2024-12-20
Lead Sponsor
Amylyx Pharmaceuticals Inc.
Target Recruit Count
110
Registration Number
NCT06122662
Locations
🇺🇸

Mayo Clinic, Scottsdale, Arizona, United States

🇺🇸

University of Arkansas for Medical Sciences, Little Rock, Arkansas, United States

🇺🇸

Parkinson's & Movement Disorder Institute, Fountain Valley, California, United States

and more 59 locations

AMX0035 in Adult Patients With Wolfram Syndrome

Phase 2
Active, not recruiting
Conditions
Wolfram Syndrome
Interventions
First Posted Date
2023-01-09
Last Posted Date
2025-04-02
Lead Sponsor
Amylyx Pharmaceuticals Inc.
Target Recruit Count
12
Registration Number
NCT05676034
Locations
🇺🇸

Washington University, Saint Louis, Missouri, United States

Extension Study Evaluating The Safety And Tolerability of AMX0035

Phase 3
Completed
Conditions
Amyotrophic Lateral Sclerosis
Interventions
First Posted Date
2022-11-17
Last Posted Date
2025-01-13
Lead Sponsor
Amylyx Pharmaceuticals Inc.
Target Recruit Count
352
Registration Number
NCT05619783
Locations
🇧🇪

University Hospitals Leuven, Leuven, Belgium

🇫🇷

Hospices Civils de Lyon Hôpital Neurologique Pierre Wertheimer Cellule Mutualisée de Recherche Clinique (CMRC), Bron, France

🇫🇷

Hopital Gabriel Montpied Service de Neurologie, Clermont-Ferrand, France

and more 42 locations
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News

FDA Grants Historic Approval to Biogen's Qalsody for Rare Genetic Form of ALS

Biogen's Qalsody (tofersen) receives FDA accelerated approval as the first therapy for SOD1-mutated ALS, marking a significant breakthrough in genetic-specific ALS treatment.

FDA Lifts Clinical Hold on Amylyx's AMX0114 Phase 1 ALS Trial, Paving Way for LUMINA Study

The FDA has lifted the clinical hold on Amylyx Pharmaceuticals' Phase 1 trial of AMX0114, an antisense oligonucleotide for ALS treatment.

Amylyx Pharmaceuticals Appoints Dan Monahan as Chief Commercial Officer to Spearhead Avexitide Launch

Amylyx Pharmaceuticals has appointed Dan Monahan as Chief Commercial Officer to lead the commercialization of their product portfolio.

Pharmaceutical R&D Highlights of 2024: GLP-1s, CRISPR, and More

• GLP-1 receptor agonists like semaglutide and tirzepatide showed potential beyond diabetes and weight loss, including cardiovascular and mental health benefits. • CRISPR gene editing entered the market with Casgevy for sickle cell disease and β-thalassemia, though uptake has been slow due to complex treatment requirements. • Protein-folding algorithms developed by Nobel laureates revolutionized biochemical research and found applications in pharmaceutical drug discovery. • Antibody-drug conjugates (ADCs) saw major investment, with firms focusing on novel linker chemistries to enhance their efficacy and target specificity.

Amylyx Announces Phase 3 LUCIDITY Trial for Avexitide in Post-Bariatric Hypoglycemia

Amylyx Pharmaceuticals initiates Phase 3 LUCIDITY trial for avexitide, a GLP-1 receptor antagonist, targeting post-bariatric hypoglycemia (PBH).

Amylyx Pharmaceuticals Advances Pipeline with Positive Data and Strategic Acquisitions

Amylyx reported positive topline data from its Phase 2 HELIOS trial of AMX0035 in Wolfram syndrome, showing improvement or stabilization across key disease measures.

AMX0035 Shows Sustained Improvements in Wolfram Syndrome Patients in Phase 2 Trial

Amylyx Pharmaceuticals' AMX0035 demonstrated sustained improvements in pancreatic function in adults with Wolfram syndrome in a Phase 2 trial.

Amylyx's AMX0035 Shows Promise in Alzheimer's Disease Biomarker Study

Amylyx Pharmaceuticals published exploratory analyses from the Phase 2 PEGASUS trial showing AMX0035 reduced key Alzheimer's disease biomarkers in cerebrospinal fluid samples.

Amylyx Acquires Phase 3-Ready GLP-1 Receptor Antagonist Avexitide for Hyperinsulinemic Hypoglycemia

Amylyx Pharmaceuticals acquired avexitide from Eiger BioPharmaceuticals for $35.1 million, gaining a first-in-class GLP-1 receptor antagonist with FDA Breakthrough Therapy Designation for post-bariatric hypoglycemia and congenital hyperinsulinism.

Relyvrio Fails Phase 3 Trial, Amylyx Considers Market Withdrawal

Amylyx Pharmaceuticals' Relyvrio failed to demonstrate significant benefit in the Phase 3 PHOENIX trial for ALS, raising concerns about its efficacy.

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