ALS (Amyotrophic Lateral Sclerosis)

Neurology
2
Pipeline Programs
3
Companies
6
Clinical Trials
2 recruiting
0
Approved Products

Pipeline by Development Stage

Preclinical
Phase 1
Phase 1/2
Phase 2
Phase 2/3
Phase 3
On Market
0
0
1
1
0
0
0
Early DiscoveryClinical DevelopmentMarket

Drug Modality Breakdown

RNA Therapeutic
1100%
+ 3 programs with unclassified modality

Competitive Landscape

3 companies ranked by most advanced pipeline stage

Biogen
BiogenCAMBRIDGE, MA
1 program
1
TofersenPhase 2RNA Therapeutic3 trials
Active Trials
NCT07294144Recruiting30Est. May 2028
NCT03764488Completed8Est. Jul 2021
NCT03070119Completed139Est. Aug 2024
Kadimastem
KadimastemIsrael - Ness Ziona
1 program
1
AstroRxPhase 1/21 trial
Active Trials
NCT03482050Completed16Est. Jun 2020
Cognixion
CognixionCA - Santa Barbara
2 programs
Cognixion + Apple Vision ProN/A1 trial
Cognixion ONEN/A1 trial
Active Trials
NCT07209943Not Yet RecruitingEst. May 2026
NCT06810219RecruitingEst. Oct 2025

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
2027
2028
BiogenTofersen
BiogenTofersen
KadimastemAstroRx
BiogenTofersen
CognixionCognixion + Apple Vision Pro
CognixionCognixion ONE

Clinical Trials (6)

Total enrollment: 193 patients across 6 trials

Long-Term Evaluation of BIIB067 (Tofersen)

Start: Mar 2017Est. completion: Aug 2024139 patients
Phase 3Completed

Tofersen in Non-SOD1 ALS

Start: Dec 2025Est. completion: May 202830 patients
Phase 2Recruiting

A Study to Evaluate Transplantation of Astrocytes Derived From Human Embryonic Stem Cells, in Patients With Amyotrophic Lateral Sclerosis (ALS)

Start: Apr 2018Est. completion: Jun 202016 patients
Phase 1/2Completed

A Study to Evaluate Safety, Tolerability, and Distribution of a Microdose of Radiolabeled BIIB067 (99mTc-MAG3-BIIB067) Co-administered With BIIB067 (Tofersen) to Healthy Adults

Start: Dec 2018Est. completion: Jul 20218 patients
Phase 1Completed
NCT07209943CognixionCognixion + Apple Vision Pro

Augmented Reality BCI Longitudinal Study for Persons With ALS, Stroke, TBI and SCI Utilizing Cognixion + Apple Vision Pro

Start: Oct 2025Est. completion: May 2026
N/ANot Yet Recruiting

Augmented Reality BCI Longitudinal Study for Persons With Late Stage ALS

Start: Jan 2025Est. completion: Oct 2025
N/ARecruiting

Related Jobs in Neurology

Phase Legend

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

Key Insights

2 actively recruiting trials targeting 193 patients
3 companies competing in this space