Disease

Amyotrophic lateral sclerosis type 22

Late-stage therapeutic development

Also known as ALS 22, TUBA4A amyotrophic lateral sclerosis, amyotrophic lateral sclerosis 22, amyotrophic lateral sclerosis 22 with or without frontotemporal dementia+2 more

ALS 22, TUBA4A amyotrophic lateral sclerosis, amyotrophic lateral sclerosis 22, amyotrophic lateral sclerosis 22 with or without frontotemporal dementia, amyotrophic lateral sclerosis caused by mutation in TUBA4A, ALS22.

Clinical trials

Associated genes

Related proteins

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Moderate

Key developments

5 clinical trials expected to report results, the earliest in Q2 2027.

Upcoming Milestones

Major developments

Upcoming trial readoutHigh impact

A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Pridopidine in Participants With Amyotrophic Lateral Sclerosis

Results expected Q1 20282026-09-15

Major trial resultsHigh impact

Multicenter, Open-label Extension Study Following the Studies MT-1186-A03 or A04 to Evaluate the Safety of Oral Edaravone in Subjects With Amyotrophic Lateral Sclerosis (ALS)

Results posted2026-05-22

Clinical Milestones7View

Results posted 2

2026-05-22Multicenter, Open-label Extension Study Following the Studies MT-1186-A03 or A04 to Evaluate the Safety of Oral Edaravone in Subjects With Amyotrophic Lateral Sclerosis (ALS)Results posted
2026-01-28HEALEY ALS Platform Trial - Regimen G DNL343Results posted

Results expected 5

2026-09-15A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Pridopidine in Participants With Amyotrophic Lateral SclerosisResults expected Q1 2028
2026-07-21HEALEY ALS Platform TrialResults expected Q3 2027
2026-03-30A Phase 3 Randomized, Placebo-Controlled Trial With a Longitudinal Natural History Run-In and Open-Label Extension to Evaluate BIIB067 Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 MutationResults expected Q3 2027
2026-03-06A Phase 2b/3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, 12 Month Clinical Trial to Evaluate the Efficacy and Safety of MN-166 (Ibudilast) Followed by Open-Label Extension Phase in Subjects With Amyotrophic Lateral SclerosisResults expected Q2 2027
2026-03-04Motor Neurone Disease - Systematic Multi-Arm Adaptive Randomised TrialResults expected Q4 2030

Activity timeline7

2026-09-15ClinicalA Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Pridopidine in Participants With Amyotrophic Lateral SclerosisResults expected Q1 2028
2026-07-21ClinicalHEALEY ALS Platform TrialResults expected Q3 2027
2026-05-22ClinicalMulticenter, Open-label Extension Study Following the Studies MT-1186-A03 or A04 to Evaluate the Safety of Oral Edaravone in Subjects With Amyotrophic Lateral Sclerosis (ALS)Results posted
2026-03-30ClinicalA Phase 3 Randomized, Placebo-Controlled Trial With a Longitudinal Natural History Run-In and Open-Label Extension to Evaluate BIIB067 Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 MutationResults expected Q3 2027
2026-03-06ClinicalA Phase 2b/3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, 12 Month Clinical Trial to Evaluate the Efficacy and Safety of MN-166 (Ibudilast) Followed by Open-Label Extension Phase in Subjects With Amyotrophic Lateral SclerosisResults expected Q2 2027
2026-03-04ClinicalMotor Neurone Disease - Systematic Multi-Arm Adaptive Randomised TrialResults expected Q4 2030
2026-01-28ClinicalHEALEY ALS Platform Trial - Regimen G DNL343Results posted

Clinical trials

16 sponsors · 1 new · 0 completed in the last 12 months (net +1)

The current development programme across all trial phases.

Clinical programme

All trials

Active

Late-stage

Completed

Late-stage studies

Motor Neurone Disease - Systematic Multi-Arm Adaptive Randomised Trial

Phase 2/3 · Recruiting · University of Edinburgh

A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Pridopidine in Participants With Amyotrophic Lateral Sclerosis

Phase 3 · Recruiting · Prilenia

HEALEY ALS Platform Trial

Phase 2/3 · Active, not recruiting · Merit E. Cudkowicz, MD

Randomized Double-blind Controlled Exploratory Clinical Study to Evaluate the Efficacy and Safety of FB1006 in the Treatment of ALS Patients

Phase 4 · Recruiting · Peking University Third Hospital

A Phase 3 Randomized, Placebo-Controlled Trial With a Longitudinal Natural History Run-In and Open-Label Extension to Evaluate BIIB067 Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Mutation

Phase 3 · Active, not recruiting · Biogen

A Phase 2b/3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, 12 Month Clinical Trial to Evaluate the Efficacy and Safety of MN-166 (Ibudilast) Followed by Open-Label Extension Phase in Subjects With Amyotrophic Lateral Sclerosis

Phase 2/3 · Active, not recruiting · MediciNova

Recruiting

Motor Neurone Disease - Systematic Multi-Arm Adaptive Randomised Trial

Phase 2/3 · Recruiting · University of Edinburgh

A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Pridopidine in Participants With Amyotrophic Lateral Sclerosis

Phase 3 · Recruiting · Prilenia

Randomized Double-blind Controlled Exploratory Clinical Study to Evaluate the Efficacy and Safety of FB1006 in the Treatment of ALS Patients

Phase 4 · Recruiting · Peking University Third Hospital

Recently completed

Effects of L-Carnitine in Amyotrophic Lateral Sclerosis Patients With CHCHD10 Mutations

Phase 4 · Completed · ChaodongWang

HEALEY ALS Platform Trial - Regimen G DNL343

Phase 2/3 · Completed · Merit E. Cudkowicz, MD

A Phase IIIb, Open Label Extension Study Evaluating The Safety And Tolerability of AMX0035 Up To 108 Weeks In Adult Participants With Amyotrophic Lateral Sclerosis (ALS) Previously Enrolled In Study A35-004 (PHOENIX)

Phase 3 · Completed · Amylyx Pharmaceuticals Inc.

A Double-blind, Randomized, Multicenter, Placebo-Controlled, Parallel, Phase III Clinical Trial to Evaluate the Efficacy and Safety of Lenzumestrocel(Neuronata-R® Inj.) in Patients With Amyotrophic Lateral Sclerosis

Phase 3 · Completed · Corestemchemon, Inc.

Multicenter, Open-label Extension Study Following the Studies MT-1186-A03 or A04 to Evaluate the Safety of Oral Edaravone in Subjects With Amyotrophic Lateral Sclerosis (ALS)

Phase 3 · Completed · Tanabe Pharma Corporation

Japanese Early-stage Clinical Trial of Ultra-high Dose Methylcobalamin for Amyotrophic Lateral Sclerosis: a Pivotal Phase 3 Randomized Controlled Study

Phase 3 · Completed · Eisai Co., Ltd.

Associated genes

1 match

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

TUBA4A0.70

Disease biology

1 match

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

Tubulin alpha-4A chainvia TUBA4A0.70

Reference

Authoritative identity, definition & identifiers.

Synonyms

References & data sources

Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
Related entities are derived from literature co-mention (studied together) — associative, not causal.