VivoSeq
Neurology

Facioscapulohumeral Muscular Dystrophy

7

Pipeline Programs↗

6

Active Trials↗

9

Companies Involved↗

Facioscapulohumeral Muscular Dystrophy is an early-stage landscape with 7 programs across 9 companies. The most active players are aTyr Pharma, Inc., Avidity Biosciences, Inc., Merck & Co..

Intelligence

Pipeline Metrics

Competitive Density

36/100

Pipeline Velocity

6/100

Mech. Concentration

100/100

Completion Rate

56/100

Attrition Rate

27/100

Competition

Competitive Landscape

Bubble size = clinical trials

Approved
Phase 3
Phase 2
Phase 1
Preclinical

Key Players

Top Companies Companies ranked by the number of pipeline programs they run for this disease.

A

aTyr Pharma, Inc.

1 program

Phase 2A

Avidity Biosciences, Inc.

1 program

Phase 2Merck & Co. logo

Merck & Co.

1 program

Phase 2Roche logo

Roche

1 program

Phase 2Arrowhead Pharmaceuticals logo

Arrowhead Pharmaceuticals

1 program

Phase 2E

Epicrispr Biotechnologies, Inc.

1 program

Phase 2

Pipeline

Programs by Phase

Analysis

Trial Status

15

clinical trials

Completed5 · 33%
Terminated4 · 27%
Recruiting3 · 20%
Active, not recruiting2 · 13%
Enrolling by invitation1 · 7%

Clinical Trials

Trial Activity

15 trials

Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD)
Recruiting·RRestem, LLC.·Phase 1·NCT07086521·16 enrolled
Study of ARO-DUX4 in Adult and Adolescent Patients With Facioscapulohumeral Muscular Dystrophy Type 1
Recruiting·Arrowhead Pharmaceuticals logoArrowhead Pharmaceuticals·Phase 1/2·NCT06131983·60 enrolled
A First-in-human Study of EPI-321 in Facioscapulohumeral Muscular Dystrophy
Recruiting·EEpicrispr Biotechnologies, Inc.·Phase 1/2·NCT06907875·12 enrolled
A Study to Evaluate RO7204239 in Participants With Facioscapulohumeral Muscular Dystrophy
Active, not recruiting·Roche logoRoche·Phase 2·NCT05548556·51 enrolled
Phase 1/2 Study of AOC 1020 in Participants With Facioscapulohumeral Muscular Dystrophy (FSHD)
Active, not recruiting·AAvidity Biosciences, Inc.·Phase 1/2·NCT05747924·90 enrolled
Phase 2 Open-label Extension Study of AOC 1020 in Participants with Facioscapulohumeral Muscular Dystrophy (FSHD)
Enrolling by invitation·AAvidity Biosciences, Inc.·Phase 2·NCT06547216·84 enrolled
Randomized Study of Albuterol in Patients With Facioscapulohumeral Muscular Dystrophy
Completed·OOhio State University·N/A·NCT00004685·90 enrolled
Efficacy and Safety of Losmapimod in Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD)
Completed·FFulcrum Therapeutics·Phase 2·NCT04003974·80 enrolled
Evaluate Safety and Biological Activity of ATYR1940 in Participants With Early Onset Facioscapulohumeral Muscular Dystrophy
Completed·AaTyr Pharma, Inc.·Phase 1/2·NCT02603562·8 enrolled
Evaluate Safety and Biological Activity of ATYR1940 in Participants With Limb Girdle Muscular Dystrophy 2B (LGMD2B) and Facioscapulohumeral Muscular Dystrophy (FSHD)
Completed·AaTyr Pharma, Inc.·Phase 1/2·NCT02579239·18 enrolled
Study to Evaluate the Long-Term Safety, Tolerability, and Biological Activity of ATYR1940 in Participants With Limb Girdle and Facioscapulohumeral Muscular Dystrophy (FSHD)
Completed·AaTyr Pharma, Inc.·Phase 1/2·NCT02836418·8 enrolled
Extension Study to Evaluate the Long-Term Effects of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD) and Charcot-Marie Tooth (CMT) Disease Types 1 and X (CMT1 and CMTX)
Terminated·Merck & Co. logoMerck & Co.·Phase 2·NCT03943290·62 enrolled
Efficacy and Safety of Losmapimod in Treating Subjects With Facioscapulohumeral Muscular Dystrophy (FSHD) With Open-Label Extension (OLE)
Terminated·FFulcrum Therapeutics·Phase 2·NCT04264442·76 enrolled
Efficacy and Safety of Losmapimod in Treating Participants With Facioscapulohumeral Muscular Dystrophy (FSHD) (REACH)
Terminated·FFulcrum Therapeutics·Phase 3·NCT05397470·260 enrolled
Study of ACE-083 in Patients With Facioscapulohumeral Muscular Dystrophy (FSHD)
Terminated·Merck & Co. logoMerck & Co.·Phase 2·NCT02927080·95 enrolled

Data sources: ClinicalTrials.gov (NIH) · OpenFDA (FDA) · ChEMBL (EMBL-EBI) · Open Targets (EBI/Sanger) · MedlinePlus (NIH) · PubMed (NIH) · SEC EDGAR (SEC)