Amyotrophic Lateral Sclerosis

Neurology
62
Pipeline Programs
30
Companies
50
Clinical Trials
5 recruiting
1
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
17
7
21
6
9
2
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

RNA Therapeutic
467%
Small Molecule
233%
+ 66 programs with unclassified modality

On Market (1)

Approved therapies currently available

Biogen
QALSODYApproved
tofersen
Biogen
Antisense Oligonucleotide [EPC]intrathecal2023

Competitive Landscape

39 companies ranked by most advanced pipeline stage

Biogen
BiogenCAMBRIDGE, MA
9 programs
2
1
2
1
TofersenPhase 4RNA Therapeutic1 trial
DexpramipexolePhase 31 trial
TofersenPhase 3RNA Therapeutic1 trial
BIIB105Phase 1/21 trial
BIIB078Phase 12 trials
+4 more programs
Active Trials
NCT04819555Completed1,000Est. May 2023
NCT01556685Completed180
NCT02611674Completed138Est. Aug 2019
+8 more trials
Cytokinetics
CytokineticsSOUTH SAN FRANCISCO, CA
6 programs
3
3
ReldesemtivPhase 31 trial
ReldesemtivPhase 31 trial
TirasemtivPhase 31 trial
CK-2017357Phase 21 trial
CK-2017357Phase 21 trial
+1 more programs
Active Trials
NCT01709149Completed711Est. Mar 2014
NCT01486849Completed27Est. Mar 2012
NCT05442775Terminated71Est. Jun 2023
+2 more trials
E
2 programs
1
methylcobalaminPhase 31 trial
PerampanelN/A1 trial
Active Trials
NCT03020797Terminated12Est. Feb 2023
NCT03548311Completed130Est. Feb 2025
Ionis Pharmaceuticals
2 programs
2
ION363Phase 31 trial
TofersenPhase 3RNA Therapeutic
Active Trials
NCT04768972Active Not Recruiting89Est. Mar 2028
AS
1 program
1
MasitinibPhase 3Small Molecule1 trial
Active Trials
NCT03127267RecruitingEst. Dec 2027
Clene
CleneSALT LAKE CITY, UT
5 programs
3
1
CNM-Au8Phase 2/31 trial
CNM-Au8Phase 21 trial
CNMAu8Phase 21 trial
Gold NanocrystalsPhase 21 trial
CNM-Au8N/A1 trial
Active Trials
NCT05281484Available
NCT04098406CompletedEst. Jul 2021
NCT05299658Active Not RecruitingEst. Aug 2026
+2 more trials
Biohaven
BiohavenNEW HAVEN, CT
3 programs
1
1
Matching PlaceboPhase 2/31 trial
BHV-0223Phase 11 trial
RiluzoleN/ASmall Molecule1 trial
Active Trials
NCT03537807No Longer Available
NCT03520517CompletedEst. Oct 2018
NCT04436510CompletedEst. Dec 2022
Denali Therapeutics
Denali TherapeuticsCA - South SF
3 programs
2
1
DNL343Phase 2/31 trial
DNL343Phase 11 trial
DNL747Phase 1
Active Trials
NCT05006352CompletedEst. Jun 2024
NCT05842941CompletedEst. Oct 2024
Seelos Therapeutics
Seelos TherapeuticsNY - New York
2 programs
1
SLS-005Phase 2/31 trial
TrehaloseN/A1 trial
Active Trials
NCT05597436No Longer Available
NCT05136885Completed161Est. Aug 2023
MediciNova
2 programs
1
1
MN-166Phase 2/31 trial
ibudilastPhase 1/21 trial
Active Trials
NCT02714036Completed35Est. Jun 2020
NCT04057898Active Not Recruiting234Est. Apr 2028
Roche
RocheSTAVANGER NORWAY, Norway
1 program
1
TRO19622Phase 2/31 trial
Active Trials
NCT01285583Completed271Est. Mar 2012
Grifols
GrifolsNEW YORK, NY
2 programs
2
AlbuminPhase 21 trial
Albutein 5%Phase 21 trial
Active Trials
NCT02479802CompletedEst. Jun 2016
NCT02872142CompletedEst. Aug 2019
Novartis
NovartisBASEL, Switzerland
2 programs
2
BLZ945Phase 21 trial
TCH346Phase 21 trial
Active Trials
NCT04066244Terminated28Est. Feb 2024
NCT00036413Completed44Est. Oct 2002
PTC Therapeutics
2 programs
2
EPI-589Phase 21 trial
PTC857Phase 21 trial
Active Trials
NCT02460679Completed19Est. Feb 2018
NCT05349721Terminated336Est. Jan 2025
Alector
1 program
1
AL001Phase 21 trial
Active Trials
NCT05053035TerminatedEst. Oct 2022
Annexon Biosciences
Annexon BiosciencesCA - Brisbane
1 program
1
ANX005Phase 21 trial
Active Trials
NCT04569435CompletedEst. Apr 2024
argenx
argenxBelgium - Zwijnaarde
1 program
1
ARGX-119Phase 21 trial
Active Trials
NCT06441682Active Not Recruiting60Est. Jul 2027
Eledon Pharmaceuticals
1
AT-1501Phase 21 trial
Active Trials
NCT04322149CompletedEst. Mar 2022
Corcept Therapeutics
1
Dazucorilant 300 mgPhase 21 trial
Active Trials
NCT05407324Recruiting279Est. Nov 2027
Apellis Pharmaceuticals
Apellis PharmaceuticalsAustralia - Hawthorn East
1 program
1
PegcetacoplanPhase 21 trial
Active Trials
NCT04579666Terminated249Est. Jul 2023
Revalesio
1 program
1
RNS60Phase 21 trial
Active Trials
NCT02988297Not Yet RecruitingEst. Nov 2028
Teva
TevaIsrael - Petach Tikva
1 program
1
TalampanelPhase 21 trial
Active Trials
NCT00982150Terminated446Est. Jun 2010
Alliance Pharmaceuticals
1 program
1
creatinePhase 22 trials
Active Trials
NCT01257581Completed60Est. Feb 2013
NCT00081250Completed300Est. Jul 2017
Newron Pharmaceuticals
4 programs
2
2
sNN0029Phase 1/21 trial
sNN0029Phase 1/21 trial
sNN0029Phase 11 trial
sNN0029 infusion solutionPhase 11 trial
Active Trials
NCT01999803TerminatedEst. Oct 2015
NCT02269436TerminatedEst. Oct 2015
NCT00800501CompletedEst. Jun 2011
+1 more trials
BrainStorm Cell Therapeutics
2 programs
1
MSC-NTF cells transplantationPhase 1/21 trial
NurOwnN/A1 trial
Active Trials
NCT04681118No Longer Available
NCT01051882Completed12Est. Mar 2013
Design Therapeutics
1 program
1
Intravenous administration of placeboPhase 1/21 trial
Active Trials
NCT02290886CompletedEst. Mar 2022
QT
Q TherapeuticsUT - Salt Lake City
1 program
1
Q-CellsPhase 1/21 trial
Active Trials
NCT02478450UnknownEst. Dec 2025
QurAlis
QurAlisMA - Cambridge
2 programs
2
Multiple ascending doses of QRL-201Phase 11 trial
QRL-101Phase 11 trial
Active Trials
NCT05633459Active Not RecruitingEst. Oct 2026
NCT06714396CompletedEst. Aug 2025
Arrowhead Pharmaceuticals
1 program
1
ARO-SOD1 InjectionPhase 11 trial
Active Trials
NCT05949294Withdrawn0Est. Mar 2025
Genentech
1 program
1
GDC-0134Phase 11 trial
Active Trials
NCT02655614Completed54Est. Mar 2020

+9 more companies

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
2029
2030
BiogenTofersen
BiogenBlood
CytokineticsReldesemtiv
CytokineticsReldesemtiv
Ionis PharmaceuticalsION363
AB ScienceMasitinib
Eisaimethylcobalamin
BiogenTofersen
CytokineticsTirasemtiv
BiogenDexpramipexole
Alliance Pharmaceuticalscreatine
Denali TherapeuticsDNL343
Seelos TherapeuticsSLS-005
CleneCNM-Au8
BiohavenMatching Placebo

Showing 15 of 50 trials with date data

Clinical Trials (50)

Total enrollment: 5,955 patients across 50 trials

A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Chinese Participants With SOD-1 Amyotrophic Lateral Sclerosis (ALS)

Start: Dec 2025Est. completion: Dec 202712 patients
Phase 4Recruiting

MATRIX: Measuring Neutralizing Antibodies in the Patients Treated With Interferon Beta 1a IM, in Mexico and Colombia

Start: Mar 2011180 patients
Phase 4Completed

A Phase 3, Open-Label Extension of COURAGE-ALS (CY 5031)

Start: Aug 2022Est. completion: Jun 202371 patients
Phase 3Terminated

A Study to Evaluate the Efficacy and Safety of Reldesemtiv in Patients With Amyotrophic Lateral Sclerosis (ALS)

Start: Aug 2021Est. completion: Jul 2023489 patients
Phase 3Terminated

FUSION: A Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of ION363 in Amyotrophic Lateral Sclerosis Participants With Fused in Sarcoma Mutations (FUS-ALS)

Start: Jun 2021Est. completion: Mar 202889 patients
Phase 3Active Not Recruiting

Efficacy and Safety of Masitinib Versus Placebo in the Treatment of ALS Patients

Start: Feb 2021Est. completion: Dec 2027
Phase 3Recruiting
NCT03548311Eisaimethylcobalamin

Clinical Trial of Ultra-high Dose Methylcobalamin for ALS

Start: Nov 2017Est. completion: Feb 2025130 patients
Phase 3Completed

An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 (Tofersen) in Adults With Inherited Amyotrophic Lateral Sclerosis (ALS)

Start: Jan 2016Est. completion: Jul 2021176 patients
Phase 3Completed

Ventilatory Investigation of Tirasemtiv and Assessment of Longitudinal Indices After Treatment for a Year

Start: Sep 2015Est. completion: Sep 2017744 patients
Phase 3Completed
NCT01622088BiogenDexpramipexole

Phase 3 Extension Study of Dexpramipexole in ALS

Start: Jun 2012Est. completion: Feb 2013616 patients
Phase 3Terminated

Creatine in Treating Patients With Cancer-Associated Weight Loss

Start: Dec 2004Est. completion: Jul 2017300 patients
Phase 3Completed

HEALEY ALS Platform Trial - Regimen G DNL343

Start: May 2023Est. completion: Oct 2024
Phase 2/3Completed

HEALEY ALS Platform Trial - Regimen E SLS-005 - Trehalose

Start: Feb 2022Est. completion: Aug 2023161 patients
Phase 2/3Completed

HEALEY ALS Platform Trial - Regimen C CNM-Au8

Start: Jul 2020Est. completion: Mar 2023
Phase 2/3Completed
NCT04436510BiohavenMatching Placebo

HEALEY ALS Platform Trial - Regimen B Verdiperstat

Start: Jul 2020Est. completion: Dec 2022
Phase 2/3Completed

Evaluation of MN-166 (Ibudilast) for 12 Months Followed by an Open-label Extension for 6 Months in Patients With ALS

Start: May 2020Est. completion: Apr 2028234 patients
Phase 2/3Active Not Recruiting

Safety Extension Study of TRO19622 in ALS

Start: Oct 2010Est. completion: Mar 2012271 patients
Phase 2/3Completed

Nebulized RNS60 for the Treatment of Amyotrophic Lateral Sclerosis

Start: Oct 2026Est. completion: Nov 2028
Phase 2Not Yet Recruiting

A Safety and Efficacy Study of ARGX-119 in Adult Patients With Amyotrophic Lateral Sclerosis (ALS)

Start: Oct 2024Est. completion: Jul 202760 patients
Phase 2Active Not Recruiting

Dazucorilant in Patients With Amyotrophic Lateral Sclerosis

Start: Nov 2022Est. completion: Nov 2027279 patients
Phase 2Recruiting

Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALS

Start: May 2022Est. completion: Jan 2025336 patients
Phase 2Terminated

An Open-Label Extension for the Phase 2 Study in Early Symptomatic Amyotrophic Lateral Sclerosis Patients on Stable Background Therapy to Assess Bioenergetic Catalysis With CNM-Au8 to Slow Disease Progression in ALS

Start: Nov 2021Est. completion: Aug 2026
Phase 2Active Not Recruiting

A Phase 2 Study to Evaluate AL001 in C9orf72-Associated ALS

Start: Sep 2021Est. completion: Oct 2022
Phase 2Terminated

Study of ANX005 in Adults With Amyotrophic Lateral Sclerosis (ALS)

Start: Jan 2021Est. completion: Apr 2024
Phase 2Completed

Multiple Doses of AT-1501-A201 in Adults With ALS

Start: Oct 2020Est. completion: Mar 2022
Phase 2Completed

MERIDIAN: A Study to Evaluate the Efficacy and Safety of Pegcetacoplan in Adults With Amyotrophic Lateral Sclerosis (ALS)

Start: Sep 2020Est. completion: Jul 2023249 patients
Phase 2Terminated
NCT03843710CleneGold Nanocrystals

31P-MRS Imaging to Assess the Effects of CNM-Au8 on Impaired Neuronal Redox State in Amyotrophic Lateral Sclerosis (REPAIR-ALS)

Start: Mar 2020Est. completion: Mar 2022
Phase 2Withdrawn

Study of Safety and Proof of the Mechanism of BLZ945 in ALS Patients

Start: Dec 2019Est. completion: Feb 202428 patients
Phase 2Terminated

Therapeutic Nanocatalysis to Slow Disease Progression of Amyotrophic Lateral Sclerosis (ALS)

Start: Dec 2019Est. completion: Jul 2021
Phase 2Completed

Efficacy and Safety of Plasma Exchange With Albutein® 5% in Participants With Amyotrophic Lateral Sclerosis

Start: Aug 2016Est. completion: Aug 2019
Phase 2Completed

Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral Sclerosis (ALS)

Start: Jan 2016Est. completion: Feb 201819 patients
Phase 2Completed

Efficacy and Safety of Plasma Exchange With Albumin in Patients With Amyotrophic Lateral Sclerosis

Start: Nov 2014Est. completion: Jun 2016
Phase 2Completed

Study of Safety, Tolerability & Efficacy of CK-2017357 in Amyotrophic Lateral Sclerosis (ALS)

Start: Oct 2012Est. completion: Mar 2014711 patients
Phase 2Completed

Dose Titration Study to Test Safety and Effects of CK-2017357 in Patients With Amyotrophic Lateral Sclerosis (ALS)

Start: Nov 2011Est. completion: Mar 201227 patients
Phase 2Completed

Safety and Efficacy Study of Creatine and Tamoxifen in Volunteers With Amyotrophic Lateral Sclerosis (ALS)

Start: Mar 2011Est. completion: Feb 201360 patients
Phase 2Completed
NCT00982150TevaTalampanel

Extension Study of Talampanel for Amyotrophic Lateral Sclerosis (ALS)

Start: Sep 2009Est. completion: Jun 2010446 patients
Phase 2Terminated

A 12-week, Multicenter, Safety and Dose-ranging Study of 3 Oral Doses of TCH346 in Patients With Amyotrophic Lateral Sclerosis

Start: Jan 2002Est. completion: Oct 200244 patients
Phase 2Completed

Study to Investigate the Safety of the Transplantation (by Injection) of Human Glial Restricted Progenitor Cells (hGRPs; Q-Cells®) Into Subjects With Amyotrophic Lateral Sclerosis (ALS)

Start: Mar 2024Est. completion: Dec 2025
Phase 1/2Unknown

A Study to Assess the Safety, Tolerability, and Effect on Disease Progression of BIIB105 in Participants With Amyotrophic Lateral Sclerosis (ALS) and Participants With the ALS Ataxin-2 (ATXN2) Genetic Mutation

Start: Sep 2020Est. completion: Aug 202499 patients
Phase 1/2Terminated

A Biomarker Study to Evaluate MN-166 in Subjects With Amyotrophic Literal Sclerosis (ALS)

Start: May 2016Est. completion: Jun 202035 patients
Phase 1/2Completed
NCT02290886Design TherapeuticsIntravenous administration of placebo

A Multicenter Phase I/II Clinical Trial to Evaluate Safety of Mesenchymal Stem Cell in Patients With Amyotrophic Sclerosis Lateral

Start: Jul 2014Est. completion: Mar 2022
Phase 1/2Completed
NCT01051882BrainStorm Cell TherapeuticsMSC-NTF cells transplantation

Autologous Cultured Mesenchymal Bone Marrow Stromal Cells Secreting Neurotrophic Factors (MSC-NTF), in ALS Patients.

Start: Jun 2011Est. completion: Mar 201312 patients
Phase 1/2Completed

An Open Label, Safety and Tolerability Continuation Study of Intracerebroventricular Administration of sNN0029 to Patients With Amyotrophic Lateral Sclerosis

Start: Jun 2009Est. completion: Oct 2015
Phase 1/2Terminated

A Safety and Tolerability Study of Intracerebroventricular Administration of sNN0029 to Patients With Amyotrophic Lateral Sclerosis

Start: Dec 2008Est. completion: Jun 2011
Phase 1/2Completed

A Study to Investigate the Safety and Pharmacodynamics of a Single Intrathecal Injection (IT) of INS1202 in Participants With Amyotrophic Lateral Sclerosis (ALS)

Start: Jan 2026Est. completion: Mar 203023 patients
Phase 1Recruiting

A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RAG-17 in Subjects With Amyotrophic Lateral Sclerosis (ALS) With Superoxide Dismutase Type 1 (SOD1) Gene Mutation

Start: Dec 2024Est. completion: Apr 2026
Phase 1Recruiting

A Study Exploring the PK/PD Relationship of QRL-101 in Adults With ALS

Start: Nov 2024Est. completion: Aug 2025
Phase 1Completed

A Study of VRG50635 in Participants With Amyotrophic Lateral Sclerosis (ALS)

Start: Jan 2024Est. completion: Jul 202554 patients
Phase 1Terminated

Study of AROSOD-1 in Adult Participants With Amyotrophic Lateral Sclerosis (ALS)

Start: Jan 2024Est. completion: Mar 20250
Phase 1Withdrawn

Safety and Gut Microbiota Analysis of an Oral Microbiotherapy in Patients With Amyotrophic Lateral Sclerosis

Start: Jun 2023Est. completion: Jul 2024
Phase 1Completed

Related Jobs in Neurology

Phase Legend

PreclinicalLab & animal studies
Phase 1Safety & dosing
Phase 2Efficacy testing
Phase 3Large-scale trials
On MarketApproved & available

Key Insights

9 late-stage (Phase 3) programs — potential near-term approvals
5 actively recruiting trials targeting 5,955 patients
30 companies competing in this space