Scholar Rock will present new Apitegromab data including 24-month efficacy and safety data from TOPAZ Phase II at the 2022 Cure SMA Annual Conference

Cambridge, Massachusetts. – (work wireScholar Rock (NASDAQ:SRRK), a clinical-stage biopharmaceutical company focused on treating critical diseases in which protein growth factors play an essential role, announced today that the company will present two on-platform presentations of 24-month data from a phase II trial. Clinical TOPAZ at the 2022 SMA Clinical Research and Care meeting, which is being held in conjunction with Cure SMA’s Annual SMA Conference (June 15-19, 2022).

“We are keen to present new data at this year’s annual SMA conference, in particular the 24-month efficacy and safety results from the TOPAZ trial, as well as the preliminary assessment of the benefits of treating patients with apitegromab regarding effects on quality of life,” said Nagesh Mahanthaba, Ph.D., CEO. The founder of ScholarRock “as social and everyday living activities.” “With this data and insights, we continue to build strong clinical evidence about the potential of apitegromab for treating spinal muscular atrophy, a disease that remains a great unmet need.”

The details of the platform offers are as follows:

Title: Effects of treatment among patients with type II and type III SMA directly reported by patients and caregivers from the Topaz clinical trial.

Presenter: Hemal Shah, PharmD, independent HEOR advisor to Scholar Rock, and CEO of Value Matters, LLC.

Mediator: Thomas Crawford, MD, principal investigator of the Topaz trial and professor of neurology and pediatrics; Johns Hopkins University

Clinical Research Session Thursday, June 16, 4:10pm – 4:30pm PST (Summary #21)

Title: TOPAZ Extension: Efficacy and Safety of Apitegromab for 24 Months in Patients with Late Spinal Muscular Atrophy (Type 2 and Type 3 SMA)

Presenter: Thomas Crawford, MD, Principal Investigator of the Topaz Trial and Professor of Neurology and Pediatrics; Johns Hopkins University.

Mediator: Katherine Klinger, Ph.D., member of the Scientific Advisory Board for Cure SMA and Global Head of Transformational Science, Sanofi SA

Clinical Drug Development Session: Fri, June 17, 11:20am – 11:40am PT (summary #28)

Site: Disneyland Hotel, Anaheim, California

For information on the conference, visit

Conference call / webcast:

Scholar Rock will host a conference call and podcast to discuss 24 months of data from the TOPAZ Phase 2 clinical trial on June 17, 2022 at 8:30 AM ET. To participate in the call, please call 833-519-1308 (domestic) or 800-914-3874 (international) and refer to Conference ID: 6495684. A webcast of the call will also be available in the Investors and Media section of the Scholar Rock website at http:/ / An archived replay of the webcast will be available on the Scholar Rock website at: for approximately 180 days after the presentation.

About Apitegromab

Apitegromab is a selective myostatin activation inhibitor and is a candidate research product for the treatment of patients with spinal muscular atrophy (SMA). Myostatin, a member of the TGFβ superfamily of growth factors, is expressed primarily by skeletal muscle cells, and the absence of its gene is associated with increased muscle mass and strength in many animal species, including humans. Scholar Rock believes that inhibiting myostatin activation with apitegromab may promote a clinically meaningful improvement in motor function in patients with SMA. The US Food and Drug Administration (FDA) has granted fast-track (FTD) and orphan drug (ODD) and rare pediatric disease (RPD) designations, and the European Medicines Agency (EMA) has granted priority drugs (PRIME) and orphan medicinal products designations, to apitegromab for the treatment of SMA. The efficacy and safety of apitegromab has not been demonstrated and apitegromab has not been approved for any use by the FDA or any other regulatory agency.

About Scholar Rock

Scholar Rock is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative drugs to treat critical diseases in which signaling by protein growth factors plays an essential role. Scholar Rock is creating a range of new candidate products with the potential to transform the lives of patients with a wide range of serious illnesses, including neuromuscular disorders, cancer and fibrosis. Scholar Rock’s approach to targeting the molecular mechanisms of growth factor activation has enabled him to develop a special platform for the discovery and development of monoclonal antibodies that locally and selectively target these signal proteins at the cellular level. By developing candidate products that operate in the disease microenvironment, the company intends to avoid the historical challenges associated with inhibiting growth factors for therapeutic effect. ScholarRock believes that its focus on biologically validated growth factors may facilitate a more efficient development path. For more information, please visit or follow Scholar Rock on Twitter (@ScholarRock) and LinkedIn ( Investors and others should note that we communicate with our investors and the public using our corporate website, including, but not limited to, company disclosures, investor presentations, FAQs, Securities and Exchange Commission filings, press releases, public conference call transcripts, and webcast transcripts , as well as on Twitter and LinkedIn. Information we post on our website, Twitter or LinkedIn may be considered material. As a result, we encourage investors, the media, and other interested parties to review the information we publish there on a regular basis. The contents of our website or social media may not be deemed incorporated by reference into any filing under the Securities Exchange Act of 1933, as amended.

Scholar Rock® is a registered trademark of Scholar Rock, Inc.

forward-looking statements

This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including, but not limited to, statements regarding Scholar Rock’s future expectations, plans, and projections, including but not limited to, Scholar Rock’s expectations regarding the growth, strategy, progress, and timing of clinical trials of apitegromab, other candidate products and the choice of indications and timing of development, the ability of any candidate product to perform in humans in a manner consistent with previous non-clinical, preclinical or clinical trial data, and the potential of the producers Candidates and proprietary platform. Use words such as “may,” “may,” “could,” “will,” “should,” “expect,” “plan,” “expect,” “believe,” “estimate,” “project,” “aim” and intent ‘, ‘future’, ‘potential’, ‘going’ and other similar expressions limit such forward-looking statements. All such forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth or contained in these forward-looking statements. These risks and uncertainties include, but are not limited to, that preclinical and clinical data, including results from a phase 2 clinical trial of apitegromab, are not predictive or may be inconsistent with or more appropriate than data from future clinical trials for the same. candidate product, including, but not limited to, the Phase 3 clinical trial of apitegromab in SMA, Scholar Rock’s ability to provide the financial support, resources, and expertise needed to identify and develop candidate products according to the expected schedule, data generated from Scholar Rock’s non-clinical and Preclinical studies and clinical trials, information provided or decisions made by regulatory authorities, competition from third parties that develop products for similar uses, Scholar Rock’s ability to obtain, maintain and protect its intellectual property, Scholar Rock’s reliance on third parties in the development and manufacture of product candidates including Including, but not limited to, the provision of any clinical trials, Scholar Rock’s ability to manage expenses and obtain additional funding when needed to support its business activities, create and maintain strategic business alliances and new business initiatives, and the effects of public health epidemics such as COVID-19 on business operations and expectations, as well as those risks that are more fully discussed in the section titled “Line Factors t” in Scholar Rock’s quarterly report on Form 10-Q for the quarter ending March 31, 2022, as well as discussions about potential risks, uncertainties, and other important factors in Scholar Rock’s subsequent filings with the Securities and Exchange Commission. Any forward-looking statements represent Scholar Rock’s views as of today only and should not be relied upon as representing its views as of any later date. All information in this press release is as of the date of issue, and Scholar Rock undertakes no duty to update such information unless required by law.

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