Facioscapulohumeral Muscular Dystrophy (FSHD)

4
Pipeline Programs
3
Companies
4
Clinical Trials
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
2
0
1
0
Early DiscoveryClinical DevelopmentMarket

Competitive Landscape

2 companies ranked by most advanced pipeline stage

Fulcrum Therapeutics
3 programs
2
1
LosmapimodPhase 31 trial
LosmapimodPhase 21 trial
Losmapimod oral tabletPhase 21 trial
Active Trials
NCT04264442TerminatedEst. Nov 2024
NCT04003974CompletedEst. Jan 2021
NCT05397470TerminatedEst. Nov 2024
aTyr Pharma
aTyr PharmaSAN DIEGO, CA
1 program
1
ATYR1940Phase 1/21 trial
Active Trials
NCT02603562Completed8Est. Dec 2016

Trial Timeline

Clinical trial activity over time

2021
2022
2023
2024
2025
2026
Fulcrum TherapeuticsLosmapimod
Fulcrum TherapeuticsLosmapimod
Fulcrum TherapeuticsLosmapimod oral tablet
aTyr PharmaATYR1940

Clinical Trials (4)

Total enrollment: 8 patients across 4 trials

Efficacy and Safety of Losmapimod in Treating Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) (REACH)

Start: Jun 2022Est. completion: Nov 2024
Phase 3Terminated

Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) With Open-Label Extension (OLE)

Start: Feb 2020Est. completion: Nov 2024
Phase 2Terminated
NCT04003974Fulcrum TherapeuticsLosmapimod oral tablet

Efficacy and Safety of Losmapimod in Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD)

Start: Aug 2019Est. completion: Jan 2021
Phase 2Completed

Evaluate Safety and Biological Activity of ATYR1940 in Participants With Early Onset Facioscapulohumeral Muscular Dystrophy

Start: Mar 2016Est. completion: Dec 20168 patients
Phase 1/2Completed

Phase Legend

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

Key Insights

1 late-stage (Phase 3) programs — potential near-term approvals
3 companies competing in this space