2022-07-01 | NDAQ:SRRK | Press release
Scholar Rock (NASDAQ: SRRK), a Phase 3 clinical-stage biopharmaceutical company focused on treating serious diseases in which protein growth factors play a fundamental role, today announced an e-poster presentation by Thomas Crawford , MD, on the design of the company’s ongoing SAPPHIRE Phase 3 clinical trial in spinal muscular atrophy (SMA) at the 17th International Congress on Neuromuscular Diseases (ICNMD 2022) in Brussels, Belgium (5-9 July 2022). The abstract will be published in the peer-reviewed Journal of Neuromuscular Diseases.
“Recruitment into the ongoing pivotal Phase 3 SAPPHIRE trial, designed to further assess the potential of apitegromab in people with non-ambulatory SMA types 2 and 3, is progressing, and we are encouraged by the enthusiasm and support from the patient and provider communities,” said Nagesh Mahanthappa, Ph.D., Founding CEO and President of Scholar Rock. “We look forward to this upcoming opportunity at ICNMD to share details of the SAPPHIRE trial with fellow physicians around the world who focus on neuromuscular disorders.”
The details of the presentation are as follows:
Title: SAPPHIRE: efficacy and safety of apitegromab in late-onset SMA; Phase 3 trial underway (Abstract #105)
Presenter: Thomas Crawford, MD, professor of neurology and pediatrics; Johns Hopkins University
Session details: Lunch and e-poster session II; Thematic group 04 – SMA: treatment
Date: Thursday, July 7, 2022
Time: 1:00 p.m.-2:00 p.m. CEST
Location: The live event will take place at the ICNMD 2022 Exhibition Hall, Square – Brussels’ Convention Center.
Scholar Rock will have a live booth, #107 and for those attending virtually, the virtual booth will be accessible through the ICNMD Learning Toolkit.
For conference information, visit ICNMD.org.
About the SAPPHIRE Phase 3 trial
SAPPHIRE is an ongoing, randomized, double-blind, placebo-controlled, Phase 3 clinical trial evaluating the safety and efficacy of apitegromab in non-ambulatory patients with SMA type 2 or 3 who are receiving a SMN treatment (nusinersen or risdiplam). Approximately 156 patients aged 2 to 12 years are expected to be recruited into the primary efficacy population. These patients will be randomized 1:1:1 to receive either apitegromab 10 mg/kg, apitegromab 20 mg/kg, or placebo by intravenous (IV) infusion every 4 weeks; for 12 months. An exploratory population of approximately 48 patients between the ages of 13 and 21 will also be evaluated separately. These patients will be randomized 2:1 to receive either apitegromab 20 mg/kg or placebo. In this subpopulation of older adults with SMA, the safety and tolerability of apitegromab will be characterized, and efficacy will also be assessed in an exploratory, non-powered fashion. SAPPHIRE is expected to register 55 sites in the United States and Europe. For more information about SAPPHIRE, visit www.clinicaltrials.gov.
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 l ‘man. Scholar Rock believes that inhibition of myostatin activation with apitegromab may result in clinically significant improvement in motor function in patients with SMA. The U.S. Food and Drug Administration (FDA) has granted Fast Track, Orphan Drug, and Rare Pediatric Disease 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.
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 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 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.
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. 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, the ability of any product candidate to work in humans d in a manner consistent with nonclinical, preclinical or prior clinical trial data, 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,” “project, “Intent,” “future,” “potential,” or “to 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 set forth or implied by such forward-looking statements.These risks and uncertainties include, but are not limited to, the that preclinical and clinical data, including Phase 2 clinical trial results, including extension periods, of apitegromab are not predictive, may be inconsistent with, or more favorable than data generated at from future cli trials of the same product candidate, including but not limited to the Phase 3 clinical trial of apitegromab in SMA, the ability of Scholar Rock’ to provide the necessary financial support, resources and expertise to identify and develop product candidates in a timely manner, 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 developing products for similar uses, Scholar Rock’s ability to obtain, maintain and protect its intellectual property, Scholar Rock’s reliance on parties for the development and manufacture of product candidates, including, without limitation, for providing clinical trials, the ability of Scholar Rock & CloseCurlyQuote; manage expenses and obtain additional funding when needed to support its business operations and establish and maintain strategic business alliances and new business initiatives, and the impacts of public health pandemics such as COVID-19 on operations and business 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 three months ended March 31, 2022, as well as discussions of risks potentials, uncertainties and other important factors in Scholar Rock’s subsequent filings with the Securities and Exchange Commission. All forward-looking statements represent Scholar Rock’s views 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.
1 Dunaway Young, Sally et al. ‘Scoliosis surgery has a significant impact on the motor skills of high-functioning people with spinal muscular atrophy’. Journal of Neuromuscular Diseases. January 1, 2020: 183–192.