Scholar Rock – Apitegromab (SRK 015)

Apitegromab (SRK-015) is a muscle-directed therapy 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 an important protein called myostatin, which restricts muscle mass and strength. Scholar Rock believes that by preventing the activation of myostatin, a clinically meaningful increase in muscle mass and strength will be achieved.

The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) and the European Medicines Agency (EMA) has granted Orphan Medicinal Product Designation, to apitegromab for the treatment of SMA.

Scholar Rock initiated a Phase 2 proof-of-concept clinical trial, TOPAZ, to assess the safety and efficacy of apitegromab in patients with Type 2 and Type 3 SMA. Enrolment was completed in January 2020.

The trial includes three cohorts, each representing a distinct subpopulation of patients:

• Cohort 1: 23 patients,
• Cohort 2: 15 patients,
• Cohort 3: 20 patients

More information

• Clinical trial register
• Scholar Rock

Community letters and press releases

• Apitegromab granted Fast Track Designation in the US for the treatment of SMA – June 2021
• Scholar Rock announces results from TOPAZ phase 2 trial in Type 2 and 3 SMA – April 2021
• Scholar Rock Reports First Quarter 2020 Financial Results and Highlights Business Progress – 7th May 2020
• Scholar Rock Announced Positive Interim Results from Phase 1 Trial of SRK-015 in Healthy Volunteers and Updates on Future Development Plans – 26th February 2019
• Scholar Rock Announces Positive Opinion by the EMA on Orphan Drug Designation for SRK-015 for the Treatment of SMA – 3rd December 2018
• Scholar Rock Reports Preclinical Data on the Therapeutic Benefit of SRK-015 in Models of SMA – 27th November 2018