Scholar Rock Announces Issuance of U.S. Patent Broadly Relevant to Inhibitors of Myostatin Activation

Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced that the United States Patent Office (USPTO) issued U.S. Patent No. 10,981,981 with an expiry of May 2034. This patent broadly covers methods for making inhibitors of myostatin (GDF8) activation based on Scholar Rock’s proprietary platform approach of targeting the precursor forms of growth factors.

“This is another important addition to our patent portfolio protecting our proprietary approach to developing antibodies that bind to the pro and latent forms of myostatin, including apitegromab being developed for the treatment of spinal muscular atrophy,” said Tony Kingsley, President and CEO of Scholar Rock. “Recently announced positive results from the TOPAZ phase 2 trial demonstrate both proof-of-concept for apitegromab in helping improve motor function for patients with Type 2 and Type 3 SMA as well as the therapeutic potential of our scientific platform of targeting the latent forms of growth factors.”

The issued claims are directed to manufacturing methods for developing antibodies that bind selectively to pro/latent myostatin thereby inhibiting myostatin signaling. Scholar Rock’s apitegromab is a selective inhibitor of the activation of latent myostatin and is an investigational product candidate being developed towards an aim of improving motor function in patients with spinal muscular atrophy (SMA). Positive 12-month top-line results from the TOPAZ Phase 2 clinical trial were recently announced, demonstrating the transformative potential of apitegromab as a potent​ial first muscle-directed therapy for SMA.

About Scholar Rock

Scholar Rock is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative medicines for the treatment of serious diseases in which signaling by protein growth factors plays a fundamental role. Scholar Rock is creating a pipeline of novel product candidates with the potential to transform the lives of patients suffering from a wide range of serious diseases, including neuromuscular disorders, cancer, fibrosis and anemia. Scholar Rock’s approach to targeting the molecular mechanisms of growth factor activation enabled it to develop a proprietary platform for the discovery and development of monoclonal antibodies that locally and selectively target these signaling proteins at the cellular level. By developing product candidates that act in the disease microenvironment, the Company intends to avoid the historical challenges associated with inhibiting growth factors for therapeutic effect. Scholar Rock believes its focus on biologically validated growth factors may facilitate a more efficient development path. For more information, please visit www.ScholarRock.com or follow Scholar Rock on Twitter (@ScholarRock) and LinkedIn (https://www.linkedin.com/company/scholar-rock/).