VivoSeq
Rare Disease

Spinal Muscular Atrophy

35

Pipeline Programs↗

25

Active Trials↗

20

Companies Involved↗

Spinal Muscular Atrophy is a moderately active landscape with 35 programs across 20 companies. The most active players are Guangzhou Jiayin Biotech Ltd, Johnson & Johnson, Catalyst Pharmaceuticals, Inc.. 3 therapies are approved with 8 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

58/100

Pipeline Velocity

32/100

Mech. Concentration

10/100

Completion Rate

80/100

Attrition Rate

12/100

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.

G

Guangzhou Jiayin Biotech Ltd

1 program

Phase 1Johnson & Johnson logo

Johnson & Johnson

1 program

Phase 1C

Catalyst Pharmaceuticals, Inc.

1 program

Phase 2L

Lantu Biopharma

1 program

Phase 2N

NMD Pharma A/S

1 program

Phase 2Argenx logo

Argenx

1 program

Phase 2

Pipeline

Programs by Phase

Analysis

Modality Breakdown

80%

Small molecule

24 of 30 classified programs — the dominant modality here.

20%ALL OTHERSRNA therapy310%Gene therapy27%Monoclonal antibody13%

Small molecule leads; the ring shows all 30 classified programs, each share of the total. Modality classified for 94% of 36 pipeline programs.

Analysis

Trial Status

76

clinical trials

Completed37 · 49%
Recruiting11 · 14%
Active, not recruiting10 · 13%
Terminated8 · 11%
Not yet recruiting3 · 4%
Unknown status2 · 3%
Other5 · 7%

Clinical Trials

Trial Activity

76 trials

A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK)
Recruiting·Biogen logoBiogen·Phase 1·NCT06555419·58 enrolled
An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy
Recruiting·LLantu Biopharma·Phase 1/2·NCT06288230·20 enrolled
A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy
Recruiting·SScholar Rock, Inc.·Phase 2·NCT07047144·52 enrolled
A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy
Recruiting·Argenx logoArgenx·Phase 2·NCT07287982·60 enrolled
Real World Clinical Effectiveness & Safety of Vesemnogene Lantuparvovec for Spinal Muscular Atrophy (SMA) in Low-middle Income Countries (LMIC).
Recruiting·LLantu Biopharma·Phase 3·NCT07265232·15 enrolled
MAP THE SMA: a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular Atrophy
Recruiting·FFondazione Policlinico Universitario Agostino Gemelli IRCCS·N/A·NCT05769465·247 enrolled
Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy
Recruiting·NNMD Pharma A/S·Phase 2·NCT05794139·54 enrolled
Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy
Recruiting·HHospices Civils de Lyon·N/A·NCT06321965·60 enrolled
Pilot Study Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies
Recruiting·SSt. Jude Children's Research Hospital·N/A·NCT06532474·24 enrolled
A Phase 1-2, Open-Label, Multicenter Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of GB221 Delivered Into the Cisterna Magna of Pediatric Participants From 2 Weeks to Younger Than 12 Months of Age With Spinal Muscular Atrophy Type 1
Recruiting·GGemma Biotherapeutics·Preclinical·NCT07070999·22 enrolled
SRK-015: Anti-Myostatin Antibody in SMA
Recruiting·Biogen logoBiogen·Phase 2·NCT05272865·180 enrolled
A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND)
Active, not recruiting·Biogen logoBiogen·Phase 3·NCT05067790·45 enrolled
Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen
Active, not recruiting·CClinic for Special Children·Phase 4·NCT05522361·10 enrolled
A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy
Active, not recruiting·Roche logoRoche·Phase 2/3·NCT05115110·259 enrolled
A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy
Active, not recruiting·BBiohaven Pharmaceuticals, Inc.·Phase 3·NCT05337553·269 enrolled
Page 1 of 6

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