Apitegromab

Phase 3Monoclonal antibody

SScholar Rock, Inc.

Apitegromab is one of 14 small molecule programs targeting Spinal Muscular Atrophy. It is currently in Phase 3.

Indications

Total Trials

Active Trials

Competitors

Pipeline

Status by Indication[3][1]

Indication

Spinal Muscular AtrophyActive, not recruiting

Facioscapulohumeral Muscular DystrophyRecruiting

Spinal Muscular AtrophyPhase 3

Active, not recruiting

Facioscapulohumeral Muscular DystrophyPhase 2

Recruiting

Competition

Competitive Landscape

14 competitors in Spinal Muscular Atrophy

#DrugCompanyPhaseTrials

Spinraza

Biogen

Approved

18 2

Evrysdi

Roche

Approved

7 3

Prilosec

DDexcel Pharma Technologies Ltd.

Amondys 45

Onasemnogene Abeparvovec

Novartis

Phase 3

7 6

Salanersen

Biogen

Phase 3

1 7

Apitegromab

SScholar Rock, Inc.

Phase 3

4 8

Taldefgrobep Alfa

BBiohaven Therapeutics Ltd.

Phase 3

1 9

Vesemnogene Lantuparvovec

LLantu Biopharma

Phase 3

2 10

NMD670

NNMD Pharma A/S

Phase 2

Ranked by pipeline maturity and clinical activity. Current drug highlighted in teal.

Timeline

Expected Milestones

Q4 2028

Phase 2Recruiting

Facioscapulohumeral Muscular Dystrophy

NCT07435129

Q1 2029

Phase 2Recruiting

Spinal Muscular Atrophy

NCT07047144

Q2 2029

Phase 3Active, not recruiting

Spinal Muscular Atrophy

NCT05626855

Clinical

Trials by Indication[1]

Spinal Muscular Atrophy4 trials

EAP of Apitegromab for Patients With Spinal Muscular Atrophy

Available·SScholar Rock, Inc.··NCT06877689

Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

Completed·SScholar Rock, Inc.·Phase 3·NCT05156320·188 enrolled

A Phase 2, Double-Blind Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Efficacy, and Safety of Apitegromab in Subjects <2 Years Old With Spinal Muscular Atrophy (SMA)

Recruiting·SScholar Rock, Inc.·Phase 2·NCT07047144·52 enrolled

Show 1 more trial

An Open-Label, Multicenter, Extension Trial to Evaluate the Long-Term Safety and Efficacy of Apitegromab in Patients With Type 2 and Type 3 Spinal Muscular Atrophy Who Completed Previous Investigational Trials of Apitegromab

Active, not recruiting·SScholar Rock, Inc.·Phase 3·NCT05626855·238 enrolled

Overweight1 trial

Efficacy and Safety of Apitegromab for the Treatment of Adults Who Are Overweight or Obese

Completed·SScholar Rock, Inc.·Phase 2·NCT06445075·102 enrolled

Facioscapulohumeral Muscular Dystrophy1 trial

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter, 52-Week Study Evaluating the Efficacy and Safety of Apitegromab in Participants With Facioscapulohumeral Muscular Dystrophy (FORGE)

Recruiting·SScholar Rock, Inc.·Phase 2·NCT07435129·60 enrolled

Sources

References

[1]
ClinicalTrials.gov — 6 trial records for Apitegromab. U.S. National Library of Medicine, National Institutes of Health.
[2]
ChEMBL — Apitegromab compound record. European Bioinformatics Institute (EMBL-EBI).
[3]
Open Targets Platform — Drug–disease associations for Apitegromab. EMBL-EBI & Wellcome Sanger Institute.
[4]
SEC EDGAR — Scholar Rock, Inc. company filings. U.S. Securities and Exchange Commission.

Details

Drug Info

Quick Facts

Modality: Monoclonal antibody[2]
Highest Phase: Phase 3[1]
Company: SScholar Rock, Inc.

Apitegromab

Status by Indication[3][1]

Competitive Landscape

Expected Milestones

Q4 2028

Q1 2029

Q2 2029

Trials by Indication[1]

Spinal Muscular Atrophy4 trials

Overweight1 trial

Facioscapulohumeral Muscular Dystrophy1 trial

References

Drug Info

Quick Facts

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