- Trial Name
- 2 to 21 years old
- To evaluate the efficacy and safety of SRK-015 in patients with later-onset SMA
- Active, not recruiting
- Italy, Spain, the Netherlands, & USA
Apitegromab – Scholar Rock
Apitegromab, or SRK-015, is a muscle-directed therapy, developed by Scholar Rock that aims to reverse or restrict the muscle atrophy and weakness experienced by people living with SMA.
The drug is an antibody that very specifically targets the latent (or inactive) form of an important protein called myostatin. Myostatin is a member of the TGFβ superfamily of growth factors. It is expressed primarily in skeletal muscle cells to inhibit muscle growth and works in concert with other growth factors and hormones to maintain appropriate muscle mass. When injected into the bloodstream, apitegromab selectively binds to latent myostatin which prevents the conversion of myostatin to its active form in muscle tissues, thereby allowing muscle growth and strength.
The European Medicines Agency (EMA) granted Priority Medicines (PRIME) Orphan Medicinal Product Designation to apitegromab for the treatment of SMA and the U.S. Food and Drug Administration (FDA) has granted it Orphan Drug Designation (ODD).
Method of action
Click the links to view the normal process, and process after injection of Apitegromab
Process after injection of Apitegromab
Trials of apitegromab (SRK-015)
- December 2018 - Scholar Rock Announces Positive Opinion by the EMA on Orphan Drug Designation for SRK-015 for the Treatment of SMA
- June 2021 - Apitegromab granted Fast Track Designation in the US for the treatment of SMA
- November 2018 - Scholar Rock Reports Preclinical Data on the Therapeutic Benefit of SRK-015 in Models of SMA
- February 2019 - Scholar Rock Announced Positive Interim Results from Phase 1 Trial of SRK-015 in Healthy Volunteers and Updates on Future Development Plans
- April 2021 - Scholar Rock announces results from TOPAZ phase 2 trial in Type 2 and 3 SMA