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apitegromab

apitegromab: developed by 1 company · 5 clinical trials tracked.

apitegromab evidence translation

What we can confirm

What it means

Source-backed records connect apitegromab to SRRK; that link alone does not prove the drug works.

What to watch

Watch trial status, endpoints, and the next source-backed update.
BioSniper AIPage evidence connected

Prepared research question

How could apitegromab → SRRK affect its developer, and what evidence supports the link?

Run cited research

Traceable citations · Unknowns marked

See linked entities and source evidence3 indications · 0 study sponsors

Related indications

SMA; Spinal Muscular Atrophy; Facioscapulohumeral Muscular DystrophyMuscular Atrophy, SpinalNeuromuscular Diseases

Study sponsors

No additional listed-company sponsor is linked.

Development Pipeline (1)
CompanyPhase
Scholar Rock Holding Corp (SRRK)
SMA; Spinal Muscular Atrophy; Facioscapulohumeral Muscular D…
Phase 3
Clinical Trials (5)

Expanded Access Protocol of Apitegromab for Patients With Spinal Muscular Atrophy

No longer available
Expanded AccessScholar Rock, Inc.

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)

NCT07435129·Phase 2·SRRK
Recruiting

Primary endpoint: Assess the efficacy of apitegromab compared with placebo in participants with FSHD

InterventionalRandomizedtriple60 targetStarted 2026-07Completion 2028-121 site · United StatesScholar Rock, Inc.

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)

NCT07047144·Phase 2·SRRK
Recruiting

Primary endpoint: Evaluate the PK of apitegromab in subjects <2 years old with SMA

InterventionalRandomizedquadruple52 targetStarted 2025-09Completion 2029-0325 sites · Belgium, France, Italy, Netherlands…Scholar Rock, Inc.

A Phase 2 Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Apitegromab in Overweight and Obese Adult Subjects

NCT06445075·Phase 2·SRRK
Completed

Primary endpoint: Change from Baseline in total Lean Body Mass (kg) at 24 weeks

InterventionalRandomizedquadruple102 enrolledStarted 2024-05Completion 2025-067 sites · United StatesScholar Rock, Inc.

Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients With Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy

NCT05156320·Phase 3·SRRK
Completed

Primary endpoint: Main Efficacy Population: Change from Baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) total score.

InterventionalRandomizedquadruple188 enrolledStarted 2022-04Completion 2024-1250 sites · Belgium, France, Germany, Italy…Scholar Rock, Inc.

Frequently asked questions

Who is developing apitegromab?

apitegromab is being developed by Scholar Rock Holding Corp.

How many clinical trials involve apitegromab?

BioSniper tracks 5 clinical trials involving apitegromab.