VivoSeq
Musculoskeletal

Muscular Atrophy, Spinal

4

Pipeline Programs↗

5

Active Trials↗

2

Companies Involved↗

Muscular Atrophy, Spinal is an early-stage landscape with 4 programs across 2 companies. The most active players are Biogen, Roche. 1 therapy is approved with 1 more in late-stage development.

Intelligence

Pipeline Metrics

Competitive Density

30/100

Pipeline Velocity

6/100

Mech. Concentration

100/100

Completion Rate

100/100

Attrition Rate

0/100

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.

Biogen logo

Biogen

3 programs

Phase 3Roche logo

Roche

1 program

Approved

Pipeline

Programs by Phase

Preclinical
No programs
Phase 12

BIIB115

Biogen

BIIB115-Matching Placebo

Biogen

Phase 2
No programs
Phase 31

Salanersen

Biogen

Approved1

Risdiplam

Roche

Analysis

Modality Breakdown

67%

Small molecule

2 of 3 classified programs — the dominant modality here.

33%ALL OTHERSRNA therapy133%

Small molecule leads; the ring shows all 3 classified programs, each share of the total. Modality classified for 75% of 4 pipeline programs.

Analysis

Trial Status

6

clinical trials

Recruiting3 · 50%
Active, not recruiting2 · 33%
Completed1 · 17%

Clinical Trials

Trial Activity

6 trials

A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy
Recruiting·Roche logoRoche·Approved·NCT05861999·28 enrolled
An Open-Label Study to Assess the Efficacy and Safety of Multiple Doses of Salanersen (BIIB115) Delivered Intrathecally to Treatment-Naïve, Presymptomatic Infants With Genetically Diagnosed Spinal Muscular Atrophy
Recruiting·Biogen logoBiogen·Phase 3·NCT07221669·30 enrolled
A Phase IV Open-Label Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Patients With Spinal Muscular Atrophy After Gene Therapy
Recruiting·Roche logoRoche·Approved·NCT05861986·28 enrolled
An Open-Label Study of Risdiplam in Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy
Active, not recruiting·Roche logoRoche·Phase 2·NCT03779334·26 enrolled
A Randomized, Blinded, Placebo-Controlled, Phase 1 Single Ascending Dose Study in Healthy Adult Male Volunteers and an Open-Label Multiple Ascending Dose Study With Long-Term Extension in Pediatric SMA Participants Previously Treated With Onasemnogene Abeparvovec (Zolgensma™) to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB115
Active, not recruiting·Biogen logoBiogen·Phase 1·NCT05575011·62 enrolled
A Phase II, Open-label Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy
Completed·Roche logoRoche·Phase 2·NCT05808764·11 enrolled

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