At Scholar Rock, we value collaborations with innovative partners who will help maximize the global potential of our revolutionary TGFβ platform and our ability to deliver new medicines to patients in need around the globe. Read more to learn about our potential as a partner or collaborator.
“We are committed to building mutually rewarding relationships across the scientific and business communities that help us deliver clinically meaningful therapies to the patients we serve.”
Sam Sabrin
Vice President of Corporate &
Business Development
We at Scholar Rock work to transform patient lives through targeting areas of high unmet medical need
Revolutionary Platform
- Global leader in TGFβ superfamily biology
- Targeting the latent forms of growth factors
- Exquisite selectivity to deliver differentiated therapeutic profiles
Neuromuscular and Beyond
- Rich preclinical pipeline focused on high unmet patient needs
- Phase 3 SAPPHIRE study underway, data readout expected in 2024
- Phase 1 POC DRAGON study underway in immuno-oncology
Positioned for Success
- Compelling proof-of-concept TOPAZ data informed Phase 3 SAPPHIRE study design
- Seasoned leadership team with track record of clinical and commercial success
- Anticipated cash runway into 2025
Strategic Optionality
- Commercial planning underway for apitegromab (SMA) in US and Europe
- Broad platform, including promising early-stage assets, provides opportunities to advance alone or in partnership
We are open to collaborations that maximize the global potential of our revolutionary TGFβ platform
Revolutionary Approach to Regulating TGFβ Superfamily Implicated in Devastating Diseases

TGFβ Superfamily: Highly Sought-After Targets
Recognized by the industry as important targets given their fundamental roles in regulating a variety of cellular processes.
Dysregulation plays a role in devastating diseases that have a high unmet need, including:
- Neuromuscular disorders
- Fibrosis
- Oncology
Scholar Rock’s R&D Platform
Transforming Medical Practice
- Selectively target the latent form of growth factors in the microenvironment of cells and tissues with uniquely designed antibodies
- Overcome the challenges that plague traditional approaches that target the “mature” growth factor, which are difficult to differentiate and lead to unintended negative effects