Scholar Rock to present phase 2 pharmacological data of Apitegromab TOPAZ at the 2021 World Congress of Neurology

CAMBRIDGE, Mass., October 03, 2021– (COMMERCIAL THREAD) –Scholar rock (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious conditions in which protein growth factors play a fundamental role, today announced an electronic poster presentation of the clinical results of the phase trial 2 TOPAZ (NCT03921528) which evaluated apitegromab in patients with spinal muscular atrophy (SMA) type 2 and type 3 to 25e World Congress of Neurology (WCN) which will take place from October 3-7, 2021.

The details of the virtual poster are as follows:

  • Title: Relationship of pharmacokinetics and pharmacodynamics to the efficacy of apitegromab in patients with late spinal muscular atrophy (SMA types 2 and 3): results of the TOPAZ study (poster # EP1150)

  • Time: The virtual e-posters can be viewed throughout the duration of the congress (3-7 October 2021) and will be available on request for 3 months after the conference.

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 the man. Scholar Rock believes that inhibiting myostatin activation with apitegromab may promote clinically meaningful improvement in motor function in patients with ADS. The United States 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 AMS. The efficacy and safety of apitegromab have not been established and apitegromab has not been approved for use by the FDA or any other regulatory body.

About SMA

Spinal muscular atrophy (SMA) is a rare and often fatal genetic disorder that typically occurs in young children. It is estimated that 30,000 to 35,000 patients have ADS 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 motor neuron survival (SMN) protein, essential for motor neuron survival, and is encoded by two genes, SMN1 and SMN2. Although there has been advancement in the development of therapies that treat the underlying genetic defect of SMA, via NMS-dependent pathways, there is still a significant 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 signaling by protein growth factors plays a fundamental role. Scholar Rock is creating a pipeline of new product candidates with the potential to transform the lives of patients with 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 the 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 (

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 the timing of selection and development of indications, its cash flow path, the ability of any product candidate to function in humans in a manner consistent with previous non-clinical results, data from preclinical or clinical trials, and the potential of its product candidates and of its proprietary platform. The use of words such as “may”, “could”, “will”, “should”, “expect”, “plan”, “anticipate”, “believe”, “estimate”, “plan”, “Intend”, “future”, “potential” or “continue” and other similar expressions are intended to identify such forward-looking statements. All of these 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 preclinical and clinical data, including the results of the phase 2 trial of apitegromab, are not predictive, are inconsistent or more favorable than data generated by future clinical trials of the same product candidate. , including the Phase 3 trial of apitegromab in ADS, Scholar Rock’s ability to provide the financial support, resources and expertise to identify and develop product candidates on schedule, data generated from non-clinical and preclinical studies and clinical trials of Scholar Rock, information provided or decisions by regulatory authorities, competition from third parties who develop products for similar uses, the ability of Scholar Rock to obtain, maintain and protect its intellectual property , the success of current and future Scholar Rock collaborations, including its collaboration with Gilead, the addiction of Sc holar Rock on third parties for the development and manufacture of product candidate structure, including to provide clinical trials, Scholar Rock’s ability to manage expenses and obtain additional funding as necessary to support its business operations and establish and maintaining 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 the risks discussed in more detail in the section titled “Risk Factors” in Scholar Rock’s quarterly report on Form 10-Q for the quarter ended June 30, 2021, as well as discussions of the potential risks, uncertainties and other significant factors in Scholar Rock’s subsequent filings with the Securities and Exchange Commission. All forward-looking statements represent the views of Scholar Rock only to date and should not be construed as representing its views at any later date. All information in this press release is as of the date of publication, and Scholar Rock assumes no obligation to update such information, except as required by law.

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Rock Scholar contacts:
Catherine hu
[email protected]

Ariane Lovell
Finnish partners
[email protected]

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