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

Rhea-AI Summary

Scholar Rock (NASDAQ: SRRK) announced a podium presentation at the 14th European Paediatric Neurology Society (EPNS) Congress in Glasgow, UK, from April 28 - May 2, 2022. The presentation titled Apitegromab in Spinal Muscular Atrophy (SMA) will analyze multiple efficacy endpoints from the 12-month TOPAZ Phase 2 trial on patients with Types 2 and 3 SMA. The data will be presented by Dr. Thomas Crawford on May 1, highlighting apitegromab's potential in improving motor function for SMA patients.

Positive

• Conducting a podium presentation at a significant congress, indicating ongoing clinical engagement.
• Highlighting results from the TOPAZ Phase 2 trial may build investor confidence in apitegromab's efficacy.
• Apitegromab has received multiple designations from regulatory agencies, suggesting a supportive development environment.

Negative

• None.

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- 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 it is delivering a podium presentation at the 14^th European Paediatric Neurology Society (EPNS) Congress, taking place in Glasgow, UK from April 28 - May 2, 2022.

Details of the podium presentation are as follows:

Title: Apitegromab in Spinal Muscular Atrophy (SMA): An Analysis of Multiple Efficacy Endpoints in the TOPAZ Trial

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

Presenter: Dr. Thomas Crawford
Session: Parallel Session 15 – Neuromuscular (PA15)
Date/Time: May 1, 10:30 a.m. – 12:15 p.m. BST
Location: Gaylord Scottish Event Campus (SEC) in Glasgow, United Kingdom

The Scholar Rock booth will be located in the Exhibition Area, Ground Floor, Hall 1 & 2 (#08) and available virtually.

For conference information, visit https://epns-congress.com/

About Apitegromab
Apitegromab is a selective inhibitor of the activation of myostatin and is an investigational product candidate 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 an increase in muscle mass and strength in multiple 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 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 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 disorder that typically manifests in young children. An estimated 30,000 to 35,000 patients are afflicted with SMA in the United States and Europe. It is characterized by the loss of motor neurons, atrophy of the voluntary muscles of 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 the survival of motor neurons, and is encoded by two genes, SMN1 and SMN2. While there has been progress in the development of therapeutics that address the underlying SMA genetic defect, via SMN-dependent pathways, there continues to be a high unmet need for therapeutics that directly address muscle function.

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, and fibrosis. 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/). Investors and others should note that we communicate with our investors and the public using our company website www.scholarrock.com, including, but not limited to, company disclosures, investor presentations and FAQs, Securities and Exchange Commission filings, press releases, public conference call transcripts and webcast transcripts, as well as on Twitter and LinkedIn. The information that we post on our website or on Twitter or LinkedIn could be deemed to be material information. As a result, we encourage investors, the media and others interested to review the information that we post there on a regular basis. The contents of our website or social media shall not be deemed incorporated by reference in any filing under the Securities 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 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 indication selection and development timing, the ability of any product candidate to perform in humans in a manner consistent with earlier nonclinical, preclinical or clinical trial data, and the potential of its product candidates and proprietary platform. The use of words such as “may,” “might,” “could,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “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 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 in or implied by such forward-looking statements. These risks and uncertainties include, without limitation, that preclinical and clinical data, including the results from the Phase 2 clinical trial of apitegromab are not predictive of, may be inconsistent with, or more favorable than, data generated from future clinical trials of the same product candidate, including, without limitation, the Phase 3 clinical trial of apitegromab in SMA, Scholar Rock’s ability to provide the financial support, resources and expertise necessary to identify and develop product candidates on the expected timeline, the data generated from Scholar Rock’s nonclinical and preclinical studies and clinical trials, information provided or decisions made by regulatory authorities, competition from third parties that are developing products for similar uses, Scholar Rock’s ability to obtain, maintain and protect its intellectual property, Scholar Rock’s dependence on third parties for development and manufacture of product candidates including, without limitation, to supply any clinical trials, Scholar Rock’s ability to manage expenses and to obtain additional funding when needed to support its business activities and establish and maintain strategic business alliances and new business initiatives, and the impacts of public health pandemics such as COVID-19 on business operations and expectations, as well as those risks more fully discussed in the section entitled "Risk Factors" in Scholar Rock’s Annual Report on Form 10-K for the year ended December 31, 2021, as well as discussions of 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 only as of today 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 the release, and Scholar Rock undertakes no duty to update this information unless required by law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20220421005002/en/

Scholar Rock:

Investors

Rushmie Nofsinger

Scholar Rock

rnofsinger@scholarrock.com

ir@scholarrock.com

857-259-5573

Media

Ariane Lovell

Finn Partners

ariane.lovell@finnpartners.com

media@scholarrock.com

917-565-2204

Source: Scholar Rock

FAQ

What is the purpose of the TOPAZ trial for apitegromab by Scholar Rock?

The TOPAZ trial aims to evaluate the efficacy of apitegromab in improving motor function in patients with Types 2 and 3 spinal muscular atrophy (SMA).

When will Scholar Rock present results from the TOPAZ trial?

Results from the TOPAZ trial will be presented at the EPNS Congress on May 1, 2022.

What designations has apitegromab received from regulatory agencies?

Apitegromab has received Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA, as well as Priority Medicines and Orphan Medicinal Product designations from the EMA.

What are the expected implications of Scholar Rock's presentation at the EPNS Congress?

The presentation may influence investor sentiment and provide insights into the potential efficacy and market viability of apitegromab for SMA.

What is spinal muscular atrophy (SMA)?

SMA is a rare genetic disorder that leads to the loss of motor neurons and muscle weakness, primarily affecting young children.