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Apitegromab

Spinal Muscular Atrophy (Types 2 & 3)

Key Facts

Indication
Spinal Muscular Atrophy (Types 2 & 3)
Phase
Phase 3
Status
Active
Company

About Scholar Rock Holding

Scholar Rock is a clinical-stage biopharmaceutical company founded on a paradigm-shifting scientific strategy: targeting the latent, inactive forms of growth factors, particularly within the TGFβ superfamily, to achieve precise, localized therapeutic effects. The company's most advanced asset, apitegromab, has demonstrated promising Phase 3 results in spinal muscular atrophy (SMA) and is positioned as a potential first-in-class muscle-targeted therapy. Supported by a robust proprietary platform and a strong financial position, Scholar Rock's strategy extends beyond neuromuscular diseases into cardiometabolic disorders and immuno-oncology, aiming to build a diversified portfolio of novel biologics.

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About Scholar Rock Holding

Scholar Rock is a clinical-stage biopharmaceutical company founded on a paradigm-shifting scientific strategy: targeting the latent, inactive forms of growth factors, particularly within the TGFβ superfamily, to achieve precise, localized therapeutic effects. The company's most advanced asset, apitegromab, has demonstrated promising Phase 3 results in spinal muscular atrophy (SMA) and is positioned as a potential first-in-class muscle-targeted therapy. Supported by a robust proprietary platform and a strong financial position, Scholar Rock's strategy extends beyond neuromuscular diseases into cardiometabolic disorders and immuno-oncology, aiming to build a diversified portfolio of novel biologics.

View full company profile

Other Spinal Muscular Atrophy (Types 2 & 3) Drugs

DrugCompanyPhase
apitegromab (SRK-015)Scholar RockPhase 3