Scholar Rock to Showcase Neuromuscular Research Portfolio at 2026 Annual Cure SMA Research and Clinical Care Meeting and 2026 International Research Congress on FSHD
Scholar Rock (NASDAQ:SRRK), a global biopharmaceutical company dedicated to improving the lives of children and adults with spinal muscular atrophy (SMA) and additional rare, severe, and debilitating neuromuscular diseases by applying its world-leading platform in myostatin biology, announced today that it will present new clinical data and research updates at Cure SMA’s Annual SMA Research & Clinical Care Meeting being held June 24-26, 2026, in Orlando, Florida and the FSHD Society’s 2026 International Research Congress on FSHD being held June 25-26, 2026 in Chicago, Illinois.
Presentations will feature updates and analyses related to the Company’s neuromuscular research portfolio, including the Phase 3 SAPPHIRE trial (NCT05156320) and Phase 2 FORGE study (NCT07435129).
The details of presentations at both meetings are as follows:
Cure SMA: 2026 Annual SMA Research and Clinical Care Meeting
Title: Assessing the impact of ‘any point differences’ or ‘stability’ in the Hammersmith Functional Motor Scale-Expanded on the independence of patients with SMA
Presentation type: Poster presentation
Presenter: Stacy Dixon, M.D., Ph.D., University of Colorado Anschutz School of Medicine
Location: Ballroom 7-10, Disney Swan Hotel
Date and time: Thursday, June 25, 4:00-6:00 PM ET
Title: Impact of baseline characteristics on apitegromab efficacy in patients with spinal muscular atrophy
Presentation type: Oral presentation
Presenter: Jing Marantz, M.D., Ph.D., Scholar Rock
Location: Promenade Ballroom, Disney’s Boardwalk Inn
Date and time: Friday, June 26 at 11:40 AM ET
Title: Post hoc analyses from the Phase 3 SAPPHIRE study evaluating apitegromab in patients with nonambulatory type 2 or 3 spinal muscular atrophy
Presentation type: Oral presentation
Presenter: Jena M. Krueger, M.D., Corewell Health Helen DeVos Children’s Hospital, Grand Rapids, Michigan
Location: Promenade Ballroom, Disney’s Boardwalk Inn
Date and time: Friday, June 26 at 12:00 PM ET
FSHD Society: 2026 International Research Congress on FSHD
Title: Trial design for the Phase 2 FORGE study evaluating apitegromab in adults with facioscapulohumeral muscular dystrophy
Presentation type: Poster
Presenter: John Staropoli, M.D., Ph.D., Scholar Rock
Location: United A/B Room, Hyatt Regency O’Hare Chicago
Date and time: Friday, June 26, 12:35-2:00 PM CT
Presentations will be made available in the Publications & Posters section of Scholar Rock’s website following each conference.
For conference information, visit https://www.curesma.org/annual-sma-conference and https://www.fshdsociety.org/event/2026-irc-chicago/.
About Apitegromab
Apitegromab is an investigational fully human monoclonal antibody inhibiting myostatin activation by selectively binding the pro- and latent forms of myostatin in the skeletal muscle. It is the first muscle-targeted treatment candidate in spinal muscular atrophy (SMA) to demonstrate clinical success in a pivotal Phase 3 clinical trial. 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 its highly selective targeting of pro- and latent forms of myostatin with apitegromab may lead to a clinically meaningful 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. Apitegromab has not been approved for any use by the FDA or any other regulatory agency.
About Scholar Rock
Scholar Rock is a late-stage biopharmaceutical company focused on developing and commercializing apitegromab for children and adults with spinal muscular atrophy (SMA) and other rare, severe, and debilitating neuromuscular diseases. As a global leader in myostatin biology, a field focused on proteins that regulate muscle mass, the biopharmaceutical company is named for the visual resemblance of a scholar rock to protein structures. Our commitment to unlock fundamentally different treatment approaches is powered by broad application of a proprietary platform, which has developed novel monoclonal antibodies to modulate protein growth factors with extraordinary selectivity. Scholar Rock works every day to create new possibilities for patients through its highly innovative anti-myostatin program, including opportunities in additional rare neuromuscular diseases. Learn more at ScholarRock.com and follow @ScholarRock on X and on LinkedIn.
Scholar Rock® is a registered trademark of Scholar Rock, Inc.
Availability of Other Information About 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 X (formerly known as Twitter) and LinkedIn. The information that we post on our website or on X 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.
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 and development programs for apitegromab, including its subcutaneous formulation, SRK-439 and its preclinical programs, and indication selection and development timing; the scientific, clinical and therapeutic implications of the data and analyses to be presented at the 2026 Annual Cure SMA Research and Clinical Care Meeting and the 2026 International Research Congress on FSHD, including post hoc analyses from the Phase 3 SAPPHIRE trial, analyses of the Hammersmith Functional Motor Scale-Expanded, and the trial design and conduct of the Phase 2 FORGE study in facioscapulohumeral muscular dystrophy; the potential of apitegromab to provide clinically meaningful benefit in patients with spinal muscular atrophy, FSHD and other rare, severe and debilitating neuromuscular diseases; the therapeutic potential, clinical benefits and safety of any product candidates; 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, whether preclinical and clinical data, including the results from the Phase 3 SAPPHIRE trial, post hoc and subgroup analyses thereof, and any results from ongoing or future clinical trials, including the Phase 2 OPAL clinical trial, the Phase 2 FORGE trial and the Phase 1 clinical trial of SRK-439, will be sufficient to support regulatory approval or further development; that preclinical and clinical data, including the results from the Phase 2 or Phase 3 clinical trial of apitegromab, post hoc analyses, data from any ongoing or future trials of apitegromab or data for SRK-439, are not predictive of, may be inconsistent with, or more favorable than, data generated from future or ongoing clinical trials of the same product candidates; whether observations and analyses presented at scientific congresses will be replicated in, or are predictive of, results from future or ongoing trials, or will be accepted by regulatory authorities, payors or the medical community; Scholar Rock’s ability to manage expenses or provide the financial support, resources and expertise necessary to identify and develop product candidates on the expected timeline; 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; and Scholar Rock's dependence on third parties for development and manufacture of product candidates including, without limitation, to supply any clinical trials, 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 March 31, 2026, 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.
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Investor Contact
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ir@scholarrock.com
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