AMX0035

Phase 2Small molecule

AAmylyx Pharmaceuticals Inc.

AMX0035 is one of 71 small molecule programs targeting Wolfram syndrome. It is currently in Phase 2.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Wolfram SyndromeActive, not recruiting

Progressive Supranuclear PalsyActive, not recruiting

Amyotrophic Lateral SclerosisActive, not recruiting

Type 1 Diabetes (T1D)Recruiting

Wolfram SyndromePhase 2

Active, not recruiting

Progressive Supranuclear PalsyPhase 2/Phase 3

Active, not recruiting

Amyotrophic Lateral SclerosisPhase 2/Phase 3

Active, not recruiting

Type 1 Diabetes (T1D)Phase 2

Recruiting

Competition

Competitive Landscape

220 competitors in Amyotrophic Lateral Sclerosis

#DrugCompanyPhaseTrials

Tivicay

GSK

Phase 3

2 2

Radicava

Shionogi

Phase 3

2 3

Simdax

Orion Corporation, Orion Pharma

Phase 3

1 4

Ultomiris

Robitussin

Lithobid

SSolvay Pharmaceuticals

Phase 3

3 7

Minocin

TTriax

Phase 3

2 8

Sodium Valproate

AstraZeneca

Phase 3

1 9

BOTULINUM TOXIN TYPE B

Eisai

Phase 3

1 10

Namenda

AbbVie

Phase 2

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2026

Phase 2Active, not recruiting

Wolfram Syndrome

NCT05676034

Q4 2029

Phase 2/3Active, not recruiting

Progressive Supranuclear Palsy

NCT06122662

Phase 2Recruiting

Type 1 Diabetes (T1D)

NCT07699380

Clinical

Trials by Indication[1]

Amyotrophic Lateral Sclerosis3 trials

A Compassionate Use Protocol of AMX0035 for Treatment of Patients With Amyotrophic Lateral Sclerosis (ALS)

Completed·AAmylyx Pharmaceuticals Inc.·Phase 2/3·NCT04516096·28 enrolled

Phase III Trial of AMX0035 for Amyotrophic Lateral Sclerosis Treatment

Active, not recruiting·AAmylyx Pharmaceuticals Inc.·Phase 3·NCT05021536·664 enrolled

AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS)

Completed·AAmylyx Pharmaceuticals Inc.·Phase 2·NCT03127514·137 enrolled

ALS3 trials

Open Label Extension Study of AMX0035 in Patients With ALS

Completed·AAmylyx Pharmaceuticals Inc.·Phase 2·NCT03488524·90 enrolled

An Intermediate Size Expanded Access Protocol of AMX0035 for ALS

Approved for marketing·AAmylyx Pharmaceuticals Inc.··NCT05286372

Pharmacokinetic and Pharmacodynamic Study of AMX0035 in Patients With ALS

Unknown status·AAmylyx Pharmaceuticals Inc.·Phase 1/2·NCT04987671·14 enrolled

Wolfram syndrome1 trial

AMX0035 in Adult Patients With Wolfram Syndrome

Active, not recruiting·AAmylyx Pharmaceuticals Inc.·Phase 2·NCT05676034·12 enrolled

Alzheimer's Disease1 trial

Study to Assess the Safety and Biological Activity of AMX0035 for the Treatment of Alzheimer's Disease

Completed·AAmylyx Pharmaceuticals Inc.·Phase 2·NCT03533257·95 enrolled

Progressive Supranuclear Palsy1 trial

AMX0035 and Progressive Supranuclear Palsy

Active, not recruiting·AAmylyx Pharmaceuticals Inc.·Phase 2/3·NCT06122662·110 enrolled

2 more indications

Type 1 Diabetes (T1D)1 trial

METabolic MODulation to Enhance Insulin Sensitivity and Mitochondrial Function in Type 1 Diabetes (MetMod-T1D)

Recruiting·UUniversity of Washington·Phase 2·NCT07699380·60 enrolled

Unspecified1 trial

Extension Study Evaluating The Safety And Tolerability of AMX0035

Completed·AAmylyx Pharmaceuticals Inc.·Phase 3·NCT05619783·352 enrolled

Sources

References

[1]
ClinicalTrials.gov — 11 trial records for AMX0035. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — AMX0035 compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for AMX0035. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Amylyx Pharmaceuticals Inc. company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Small molecule[2]
Highest Phase: Phase 2[1]
Company: AAmylyx Pharmaceuticals Inc.

AMX0035

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2026

Q4 2029

Trials by Indication[1]

Amyotrophic Lateral Sclerosis3 trials

ALS3 trials

Wolfram syndrome1 trial

Alzheimer's Disease1 trial

Progressive Supranuclear Palsy1 trial

Type 1 Diabetes (T1D)1 trial

Unspecified1 trial

References

Drug Info

Quick Facts

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