C-HIODlatent growth factors, monoclonal antibodies, TGFβ biology
0-100 chain-logic scale · 15 dimensions · scored on public evidence
Evaluate apitegromab as monotherapy for participants with facioscapulohumeral muscular dystrophy.
Randomized phase 2 clinical study assessing efficacy and safety of apitegromab monotherapy in FSHD.
ClinicalTrials.gov record for a phase 2 FSHD study was supplied with published_at date of 2026-08-01.
Assess SRK-439 in healthy adults, including safety, tolerability, pharmacokinetics, and pharmacodynamics.
Single- and multiple-ascending dose phase 1 study in healthy adults.
Phase 1 healthy-adult SAD/MAD study listed with ClinicalTrials.gov published_at date of 2025-11-21.
Improve motor function and long-term outcomes in patients with later-onset type 2 or type 3 spinal muscular atrophy, including use as adjunctive therapy with approved SMN upregulator therapy and evaluation in younger patients under 2 years old.
Apitegromab is an investigational fully human monoclonal antibody that selectively inhibits myostatin activation; evaluated in TOPAZ, SAPPHIRE, ONYX extension, and a phase 2 pediatric PK/PD, efficacy, safety, and tolerability study.
SAPPHIRE phase 3 trial reported safety and efficacy in nonambulatory type 2 or type 3 SMA; ONYX is evaluating long-term safety and efficacy after prior apitegromab trials; a phase 2 study is evaluating subjects under 2 years old.
The supplied material identifies SAPPHIRE as a phase 3 double-blind randomized placebo-controlled trial and includes a 2026 review on myostatin-pathway inhibition for SMA, but does not provide numerical SAPPHIRE results in the supplied text.
Preserve lean mass in adults with overweight or obesity receiving GLP-1 receptor agonist therapy such as tirzepatide.
Randomized, double-blind, placebo-controlled phase 2 EMBRAZE study of tirzepatide plus apitegromab 10 mg/kg versus tirzepatide plus placebo.
Phase 2 EMBRAZE proof-of-concept results were published in Nature Medicine in 2026.
At week 24, apitegromab produced 1.9 kg less lean mass loss than placebo despite similar total body weight loss, representing 54.9% retention of lean mass relative to placebo; adverse events and serious adverse events were generally balanced between groups.
Overcome immune checkpoint blockade resistance in locally advanced or metastatic solid tumors by targeting latent TGFβ1 in combination with anti-PD-(L)1 therapy.
First-in-human multicenter open-label phase 1 DRAGON trial of SRK-181 alone and in combination with pembrolizumab or other anti-PD-(L)1 therapy, including dose escalation and dose expansion.
Phase 1 DRAGON results were published in Nature Medicine in 2026.
Linavonkibart had a manageable safety profile; no cytokine release syndrome or infusion interruption was observed. In part B, combination therapy with pembrolizumab showed confirmed objective response rates of 20.0% in clear cell renal cell cancer, 18.2% in melanoma, 9.1% in head and neck squamous cell cancer, and 9.1% in urothelial cancer among anti-PD-1-resistant patients.
Discover and develop therapies that selectively target growth factors in their latent form before activation across serious diseases, including neuromuscular disease, immuno-oncology, fibrotic disease, and hematology.
Platform focused on protein growth factors in the TGFβ superfamily, using monoclonal antibodies and latent growth factor biology.
Platform has advanced monoclonal antibodies into clinical development, including apitegromab and linavonkibart/SRK-181.
The supplied material states Scholar Rock has advanced monoclonal antibodies into clinical development using this platform, with clinical programs across SMA, oncology, obesity-related lean mass preservation, and other pipeline areas.
Pipeline-listed Scholar Rock program; the supplied material does not state the target indication or development objective.
No program-specific method is supplied beyond Scholar Rock's broader latent growth factor, monoclonal antibody, and TGFβ superfamily platform description.
Listed on Scholar Rock's pipeline page in the supplied site analysis.
Pipeline-listed Scholar Rock program; the supplied material does not state the target indication or development objective.
No program-specific method is supplied beyond Scholar Rock's broader latent growth factor, monoclonal antibody, and TGFβ superfamily platform description.
Listed on Scholar Rock's pipeline page in the supplied site analysis.