Scholar Rock Presents Exploratory Responder Analysis on Efficacy Data from the Apitegromab TOPAZ Phase 2 Trial at the Child Neurology Society Annual Meeting

Rhea-AI Summary

Scholar Rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company, announces a late-breaking poster presentation at the 50th Child Neurology Society Annual Meeting held from September 29 to October 2, 2021, in Boston. The presentation focuses on the efficacy of Apitegromab in patients with later-onset Spinal Muscular Atrophy (SMA) Types 2 and 3, highlighting responder analyses from the Phase 2 TOPAZ study. The poster will be published in the 2021 Annals of Neurology supplement. Apitegromab has received multiple designations from the FDA and EMA, although its efficacy and safety are not yet established.

Positive

• Late-breaking poster presentation on Apitegromab at key neurology conference.
• Focus on SMA, a condition with high unmet medical needs.
• Apitegromab has received Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the FDA.
• Received Priority Medicines and Orphan Medicinal Product designations from the EMA.

Negative

• Efficacy and safety of Apitegromab have not been established.
• Dependence on upcoming Phase 3 trial results for further validation.

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 a late breaker poster presentation as part of the 50^th Child Neurology Society (CNS) Annual Meeting taking place September 29-October 2, 2021 Virtual and Live in Boston, MA.

Details for the Poster at the CNS meeting are as follows:

• Title: Efficacy of Apitegromab in Patients with Later-Onset Spinal Muscular Atrophy (SMA Types 2 and 3): Responder Analysis from the Phase 2 TOPAZ Study (#310)
  • This late breaking poster presentation includes exploratory responder analyses on Hammersmith scale scores, including time to achieve various thresholds of improvement in Hammersmith Functional Motor Scale Expanded (HFMSE) scores.
  • Live poster presentation (#310) will be available on September 30, 2021, from 11:45am- 6:30pm ET during the exhibits and posters open session. In addition, posters will be available during the exhibits and poster review sessions with authors on September 30 from 12:00-1:00pm ET, 5:30-6:30pm and October 1, 2021, from 7:00-8:00am ET.
  • This poster will be published in the 2021 Annals of Neurology supplement or the CNS Annual Meeting Program

Scholar Rock will have a physical booth (#915) at the Hynes Convention Center, Exhibit Halls C & D, as well as a virtual booth at the meeting.

Live Exhibit Hall Times:

• Wednesday, September 29 from 6:00-7:30pm ET
• Thursday, September 30 from 11:30am-6:30pm ET
• Friday, October 1 from 7:00-10:30am ET

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/).

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, SRK-181, and other product candidates and indication selection and development timing, its cash runway, 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,” “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 preclinical and clinical data, including the results from the Phase 2 trial of apitegromab, are not predictive of, are inconsistent with, or more favorable than, data generated from future clinical trials of the same product candidate, including the planned Phase 3 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, the success of Scholar Rock’s current and potential future collaborations, including its collaboration with Gilead, Scholar Rock’s dependence on third parties for development and manufacture of product candidates including 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 Quarterly Report on Form 10-Q for the quarter ended June 30, 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/20210930005108/en/

Scholar Rock:

Investors

Catherine Hu

chu@scholarrock.com

Media

Ariane Lovell

Finn Partners

ariane.lovell@finnpartners.com

917-565-2204

Source: Scholar Rock

FAQ

What was presented at the Child Neurology Society Annual Meeting regarding Scholar Rock?

Scholar Rock presented a late-breaking poster on Apitegromab's efficacy for patients with later-onset Spinal Muscular Atrophy (SMA) Types 2 and 3.

What designations has Apitegromab received from regulatory authorities?

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

What are the results from the Phase 2 TOPAZ study for Apitegromab?

The Phase 2 TOPAZ study includes exploratory responder analyses on Hammersmith scale scores, with ongoing assessments of motor function improvements.

When is the live poster presentation for Scholar Rock?

The live poster presentation will occur on September 30, 2021, from 11:45 am to 6:30 pm ET.

What is the significance of the SMA for Scholar Rock's business?

SMA represents a significant market opportunity due to its high unmet need for effective treatments, which could positively impact Scholar Rock's product offerings.