Scholar Rock Holding

SRRKPhase 3

Scholar Rock is a public biotech company leveraging its unique understanding of protein growth factor activation to develop novel therapeutics. Its core technology platform targets the latent, inactive forms of proteins, aiming for greater specificity and reduced side effects compared to targeting the mature, active forms. The company's most advanced program, apitegromab, has shown promising Phase 2 results in SMA and is advancing into pivotal trials, positioning Scholar Rock as a key player in neuromuscular therapeutics. Beyond SMA, the company is advancing programs in oncology and immuno-oncology.

Market Cap

$5.1B

Employees

100-150

Focus

Biotech

SRRK · Stock Price

USD 44.64+30.37 (+212.82%)

Historical price data

AI Company Overview

Technology Platform

A proprietary platform focused on discovering and developing monoclonal antibodies that selectively target the latent, inactive forms of growth factors, particularly in the TGFβ superfamily, to achieve highly specific inhibition of disease-driving signaling pathways.

Pipeline Snapshot

9 drugs in pipeline, 2 in Phase 3

Drug	Indication	Stage
Apitegromab + Placebo	Spinal Muscular Atrophy	Phase 3
Apitegromab	Spinal Muscular Atrophy	Phase 3
SRK-015	Spinal Muscular Atrophy	Phase 2
Apitegromab + Nusinersen + Risdiplam	Spinal Muscular Atrophy	Phase 2
Apitegromab + Placebo + Tirzepatide	Overweight and Obesity	Phase 2

Funding History

Total raised: $272M

IPO$75MUndisclosedJun 28, 2018

Series C$80MARCH Venture PartnersJun 15, 2018

Series B$70MARCH Venture PartnersJun 15, 2016

Series A$47MARCH Venture PartnersJun 15, 2014

Opportunities

The primary growth opportunity is the potential approval and commercialization of apitegromab as the first muscle-directed therapy for SMA, addressing a large, persistent unmet need in patients on SMN-upregulating therapies.

Success in the SRK-181 oncology program could unlock a major new market in overcoming checkpoint inhibitor resistance.

The broader latent growth factor platform offers a pipeline engine for future programs in fibrosis and immunology.

Risk Factors

Key risks include clinical trial failure of the pivotal Phase 3 SAPPHIRE trial for apitegromab, regulatory hurdles, challenges in commercial adoption as an adjunctive SMA therapy, and intense competition in both neuromuscular and oncology spaces.

The company also faces financial risk as a pre-revenue entity requiring future capital raises.

Competitive Landscape

In SMA, Scholar Rock competes with other muscle-targeting agents from Biohaven and Roche. Its latent myostatin-specific mechanism is a key differentiator. In oncology, it faces competition from other TGFβ inhibitors (Sanofi, etc.); its latent-specific approach aims to improve the therapeutic window. The company's core competitive advantage is its proprietary platform for targeting latent growth factors.

Publications

Pipeline