Scholar Rock to Present Data from TOPAZ Outpatient Cohort Analysis at the 2022 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Scholarly rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on treating serious diseases in which protein growth factors play a fundamental role, today announced that it will present a poster at the 2022 Clinical & Scientific Conference of the Muscular Dystrophy Association (MDA) (March 13 – 16, 2022).

The poster presents data from an exploratory analysis of the outpatient cohort of the Phase 2 TOPAZ trial evaluating apitegromab, a selective inhibitor of latent myostatin activation, in people with spinal muscular atrophy (SMA ) of late onset.

“We are excited to develop and study the therapeutic potential of apitegromab in both ambulatory and non-ambulatory forms of SMA,” said George Nomikos, MD, Ph.D., Senior Vice President of Science medical and clinical services and head of the Muscle Therapeutic Area at Scholar Rock. “The hypothesis-generating information from this analysis identifies factors that may be more likely to be associated with gains in motor function after apitegromab treatment in outpatients with SMA.”

The TOPAZ study evaluated the safety, tolerability, PK/PD, and efficacy of apitegromab in SMA types 2 and 3. This post hoc analysis of 12-month treatment data from the outpatient cohort of Type 3 SMA explored the relationships between the efficacy of apitegromab (pooling data from patients receiving it as monotherapy or in addition to background nusinersen) and factors such as pharmacokinetics and pharmacodynamics (PK/PD), age, body mass index and the presence of scoliosis or joint contractures. The presence or absence of scoliosis and/or joint contractures at baseline were associated with differential responses in the Revised Hammersmith Scale (RHS) score after 12 months of treatment. Patients without scoliosis (n=12) had a mean change of +0.67 points from baseline in the RHS score, while patients with scoliosis (n=11) had a mean change of -1.45 compared to the initial value of the RHS score. Additionally, patients without joint contractures (n=10) had a mean change of +1.10 points from baseline in the RHS score, while patients with joint contractures (n=13) had a mean change mean -1.46 from baseline in RHS score. .

The poster will be available in person and virtually. The details are below:

Title: Apitegromab in SMA: an analysis of PK/PD relationships with efficacy in outpatients from the TOPAZ trial (#72)

Presenter: Dr. Thomas Crawford, principal investigator of the TOPAZ trial and co-director of the Muscular Dystrophy Association Clinic, professor of neurology at Johns Hopkins University

Poster Session Times:

  • Sunday, March 13, 6:00 p.m. – 8:00 p.m. CST

  • Monday, March 14, 10:00 a.m. to 8:00 p.m. CST

  • Tuesday, March 15, 10:00 a.m. to 8:00 p.m. CST

Location: Gaylord Opryland Resort & Convention Center, Nashville, TN, Ryman Hall B1-2

The Scholar Rock booth will be located in the exhibit hall (#206 and #208) and available virtually.

For conference information, visit

About apitegromab

Apitegromab is a selective inhibitor of myostatin activation and is an investigational product candidate for the treatment of patients with spinal muscular atrophy (SMA). Myostatin, a member of the TGFβ growth factor superfamily, is expressed primarily by skeletal muscle cells, and the absence of its gene is associated with increased muscle mass and strength in several animal species, including humans. Scholar Rock believes that inhibiting myostatin activation with apitegromab may promote clinically meaningful improvement in motor function in patients with SMA. The U.S. Food and Drug Administration (FDA) has granted Fast Track (FTD), Orphan Drug (ODD), and Rare Pediatric Disease (RPD) designations, and the European Medicines Agency (EMA) has granted Priority designations Medicines (PRIME) and Orphan Medicinal Product. designations, to apitegromab for the treatment of SMA. The efficacy and safety of apitegromab have not been established, and apitegromab has not been approved for any use by the FDA or any other regulatory agency.

About Scholar Rock

Scholarly rock is a clinical-stage biopharmaceutical company focused on the discovery and development of innovative drugs for the treatment of serious diseases in which protein growth factor signaling plays a fundamental role. Scholar Rock is creating a pipeline of new product candidates that have the potential to transform the lives of patients suffering from a wide range of serious diseases, including neuromuscular disorders, cancer and fibrosis. Scholar Rock’s approach to targeting the molecular mechanisms of growth factor activation has 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 that a focus on biologically validated growth factors can facilitate a more efficient developmental pathway. 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 through our company website., including but not limited to company disclosures, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference call transcripts and transcripts of webcasts, as well as on Twitter and LinkedIn. Information that we post on our website or on Twitter or LinkedIn may be considered material information. Accordingly, we encourage investors, the media and other interested persons to regularly review the information we post there. The content of our website or social media should not be taken as incorporated by reference in any filing under the Securities Act 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 prospects, including, without limitation, Scholar Rock’s expectations regarding its growth, strategy, progress and timing of its clinical trials for apitegromab and other product candidates and the selection of indications and timing of development, ability of any product candidate to work in humans in a manner consistent with data from prior nonclinical, preclinical or clinical trials, and the potential of its product candidates and proprietary platform. The use of words such as “may”, “could”, “could”, “will”, “should”, “expect”, “plan”, “anticipate”, “believe”, “estimate”, “projects”, “intend”, “future”, “potential” or “continue” and other similar expressions are intended to identify such forward-looking statements. All such forward-looking statements are based on management’s current expectations regarding future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those stated or implied. by these forward-looking statements. These risks and uncertainties include, but are not limited to, that preclinical and clinical data, including results from the Phase 2 clinical trial of apitegromab, are not predictive, may be inconsistent with the data generated by future clinical trials of the same or more favorable than these. product candidate, including but not limited to the Phase 3 clinical trial of apitegromab in SMA, the ability of Scholar Rock to provide the financial support, resources and expertise necessary to identify and develop candidate products on time, data generated from non-clinical studies and preclinical studies and clinical trials, information provided or decisions made by regulatory authorities, competition from third parties developing products for similar uses, Scholar Rock’s ability to obtain , maintaining and protecting its intellectual property, Scholar Rock’s reliance on third parties for the development and manufacturing of product candidates, including without limitation to provide any clinical trial, Scholar Rock’s ability to manage expenses and obtain additional funding when needed to support its business activities and establish and maintain strategic business alliances ics and new business initiatives, and the impacts of public health pandemics such as COVID-19 on business operations and expectations, as well as the risks discussed in more detail in the section titled “Risk Factors” of Scholar’s Annual Report Rock on Form 10-K for the fiscal year ended December 31, 2021, as well as discussions of potential risks, uncertainties, and other material factors in Scholar Rock’s subsequent filings with the Securities and Exchange Commission. Any forward-looking statements represent the views of Scholar Rock only as of the current date and should not be relied upon as representing its views as of any subsequent date. All information in this press release is as of the date of publication, and Scholar Rock undertakes no obligation to update this information except as required by law.