Scholar Rock Presents Data Analysis of Multiple Efficacy Endpoints from Phase 2 Apitegromab TOPAZ Trial at European Society for Pediatric Neurology 2022 Congress


CAMBRIDGE, Mass.–(BUSINESS WIRE)–Scholar 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 be presenting on the podium during the 14and Congress of the European Pediatric Neurological Society (EPNS), to be held in Glasgow, UK, from April 28 to May 2, 2022.

The details of the podium presentation are as follows:

Title: Apitegromab in spinal muscular atrophy (SMA): analysis of several efficacy parameters in the TOPAZ trial

  • The presentation will include an analysis of multiple efficacy endpoints, including the Hammersmith Scales and Revised Upper Limb Modulus (RULM) function, from the 12-month Phase 2 TOPAZ trial evaluating apitegromab in patients with SMA types 2 and 3.

Presenter: Dr Thomas Crawford

Session: Parallel Session 15 – Neuromuscular (PA15)

Date hour : May 1, 10:30 a.m. – 12:15 p.m. BST

Location: Gaylord Scottish Event Campus (SEC) in Glasgow, UK

The Scholar Rock booth will be located in the exhibit area, ground floor, Hall 1 & 2 (#08) 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 SMA

Spinal muscular atrophy (SMA) is a rare and often fatal genetic disease that usually manifests in young children. An estimated 30,000 to 35,000 patients have SMA in the United States and Europe. It is characterized by loss of motor neurons, atrophy of voluntary muscles in the limbs and trunk, and progressive muscle weakness. The underlying pathology of SMA is caused by insufficient production of the SMN (survival of motor neuron) protein, essential for motor neuron survival, and is encoded by two genes, SMN1 and SMN2. Although there have been advances in the development of therapies that address the underlying genetic defect of SMA, via SMN-dependent pathways, there is still a high unmet need for therapies that directly address muscle function.

About Scholar Rock

Scholar 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 him 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, 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 company website, including but not limited to company disclosures, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference call and webcast transcripts, and 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.


About Author

Comments are closed.