Our Pipeline
Our pipeline runs on breakthrough science
Scholar Rock is turning scientific discoveries into a pipeline of investigational therapies aimed at diseases where growth factors play a central role.
Our therapeutic approach selectively targets the latent forms of growth factors, which can get to the underlying biology of many serious diseases. We apply this approach to disease-driving targets where it can be most effective.
Our Pipeline
Neuromuscular
Targeting Latent Myostatin
| Preclinical | Phase 1 | Phase 2 | Phase 3 | Commercialization |
Apitegromab (selective anti-latent myostatin antibody)
Spinal Muscular Atrophy
Phase 3
Anticipated Key Milestones:
- Commercial Launch in 2025*
- BLA submission in 2025
Apitegromab in additional rare neuromuscular disorders
Preclinical
SRK-439 (novel anti-latent myostatin antibody)
Rare Neuromuscular Disorders
Preclinical
Anticipated Key Milestones
- File IND in 2H 2025
Undisclosed Program
Neuromuscular Disorders
Preclinical
Immuno-oncology
Targeting Latent TGFβ1
| Preclinical | Phase 1 | Phase 2 | Phase 3 | Commercialization |
SRK-181 (Selective context-independent, anti-latent TGFβ1)
Cancer (locally advanced or metastatic solid tumors)
Phase 1
Anticipated Key Milestones
- Rolling clinical data updates
Fibrotic Disease
| Preclinical | Phase 1 | Phase 2 | Phase 3 | Commercialization |
SRK-373 (Selective context-dependent (LTBP1 & LTBP3) anti-latent TGFβ1)
Fibrosis (multiple fibrotic indications)
Preclinical
Anticipated Key Milestones
- IND-enabling studies
Hematology
Targeting RGMc/HJV
| Preclinical | Phase 1 | Phase 2 | Phase 3 | Commercialization |
SRK-256 (Selective anti-RGMc/HJV)
Iron-restricted Anemia
Preclinical
Anticipated Key Milestones
- IND-enabling studies
TGFβ1 =Transforming Growth Factor Beta 1; LTBP1 = Latent Transforming Growth Factor Beta Binding Protein 1; LTBP3 = Latent Transforming Growth Factor Beta Binding Protein 3; RGM = Repulsive Guidance Molecule; HJV = Hemojuvelin; IND = Investigational New Drug; POC = Proof of Concept
*Contingent upon receipt of regulatory approval
**This site includes information about investigational products and their uses which have not been approved by the U.S. Food and Drug Administration. This information should not be construed as a recommendation for use of any product for unapproved uses.
Partnering
We are exploring partnerships to maximize the potential of our revolutionary approach.
Clinical Trials
We focus on the discovery and development of safe, effective therapies to deliver solutions for patients in need.
Patient Access
As we advance our investigational therapies through clinical development, our goal is to provide access to these therapies at the appropriate time and in the correct manner for patients.
