Overview
Scholar Rock Holding Corp is a biopharmaceutical company developing selective biologic medicines that modulate growth-factor activity, with a focus on diseases involving muscle function. Its lead candidate, apitegromab, is an investigational myostatin inhibitor being developed for spinal muscular atrophy; its pipeline also includes candidates for other muscle-related conditions.[1][2][3][4]
The company’s stated mission is to discover, develop and deliver therapies for people with serious diseases and high unmet medical need. Its development and commercial ambitions span the United States and selected international markets, including Europe. The company reported no product-sales revenue to date in its 2026 second-quarter filing; the reviewed sources did not provide an employee count.[5][6][7] [1][2][3][4][6][7][5]
Products & services
ISEMBYLD™ (apitegromab-mstn; formerly apitegromab) is an FDA-approved biologic for adults and children aged 2 years and older with spinal muscular atrophy (SMA) who are receiving an SMN2-targeted treatment. It is a recombinant monoclonal antibody that binds myostatin proforms, including latent myostatin, to inhibit activation of myostatin, a regulator of muscle mass. The labeled dose is 10 mg/kg by intravenous infusion every four weeks.[8][9][10] The company reported that the U.S. launch was underway following approval.[11]
Apitegromab is also being studied in a Phase 2 trial in SMA patients under age 2 and in the ONYX long-term extension study; a Phase 2 study in facioscapulohumeral muscular dystrophy (FSHD) began in the second quarter of 2026. The company has also reported a Phase 2 proof-of-concept study of apitegromab with tirzepatide, which found preservation of lean mass during tirzepatide-induced weight loss; it was seeking partners for further evaluation of the combination.[12][13]
SRK-439 is an investigational, fully human antibody against pro- and latent myostatin, designed for subcutaneous administration and described by the company as highly potent and selective. Its Phase 1 study in healthy volunteers was underway as of June 30, 2026.[14] Other programs identified for potential partnership include SRK-181, a Phase 2-ready latent TGFβ1 inhibitor for solid tumors resistant to anti-PD-(L)1 therapies; preclinical SRK-373, a selective latent TGFβ1 inhibitor for fibrotic diseases; and preclinical SRK-256, an RGMc/hemojuvelin inhibitor for iron-restricted anemia.[13]
The company reported no product-sales revenue to date in its second-quarter 2026 filing, which preceded ISEMBYLD’s approval; the cited materials do not provide a post-approval product-revenue share or identify separate fee-for-service offerings.[7] [8][12][13][14][7][9][10][11]
Geographic presence
Scholar Rock Holding Corp is headquartered at 301 Binney Street, 3rd Floor, Cambridge, Massachusetts. At year-end 2025, it reported 289 employees, but did not disclose their geographic distribution. Its 2025 filing described an approximately 50-person U.S. customer-facing team and a European commercial build-out beginning in Germany.[15][16]
The company’s Phase 3 SAPPHIRE trial enrolled participants at 48 hospitals across Belgium, France, Germany, Italy, the Netherlands, Poland, Spain, the United Kingdom, and the United States.[17] Scholar Rock reports that it relies on third-party manufacturers and does not identify their locations. Its FY2025 and Q2 2026 filings do not provide revenue by country or region.[18][19] [16][15][17][18][19][15][17]
Business segments
Scholar Rock reports one operating and reportable segment, managed on a consolidated basis. The segment covers research and development of monoclonal antibodies that selectively inhibit growth-factor activation; the company does not report its drug programs, including apitegromab, SRK-181 and SRK-439, as separate segments.[20][21]
This single segment represents all reported operations, but a revenue contribution percentage is not applicable: the company reported no product sales revenue through March 31, 2026. Program-level spending disclosed in the filing is not a separate segment breakdown.[21][22][23] [20][21][22][23]
Company history
Scholar Rock was formed in May 2012, and company materials describe it as founded in October that year. Its scientific founders were Timothy Springer, PhD, and Leonard Zon, MD. An early development milestone was an option-and-license agreement with Janssen Biotech in December 2013.[24][25][26][27]
In May 2018, Scholar Rock began Phase 1 testing of SRK-015, later named apitegromab, and its shares began trading on Nasdaq following the company’s IPO. That December, it entered a three-year TGFβ-signaling research and development collaboration with Gilead. Apitegromab advanced through the Phase 2 TOPAZ study, with positive 12-month results announced in April 2021; the FDA granted the drug Fast Track designation the following month.[28][29][30][31][32][33][34][35]
Scholar Rock advanced apitegromab into the pivotal Phase 3 SAPPHIRE study, reporting positive topline results in October 2024. It submitted a U.S. biologics license application in January 2025, but the FDA issued a Complete Response Letter in September, citing observations at a third-party fill-finish facility. In April 2025, David Hallal became CEO, succeeding Jay Backstrom. The company resubmitted the application in March 2026, and the FDA accepted it for review.[36][37][38][39][40][41] [24][25][26][27][28][29][30][31][32][33][34][35][36][37][38][39][40][41][25][26][40]
Sources
- Scholar Rock Holding Corp — Press release (2026 Q 2)
- scholarrock.com[5]
- Scholar Rock Holding Corp — Full financial report (2026 Q 2)
- www.accessdata.fda.gov[9]
- www.fda.gov[10]
- investors.scholarrock.com[11]
- scholarrock.com[15]
- Scholar Rock Holding Corp — Full financial report (2025 Y)
- clinicaltrials.gov[17]
- Scholar Rock Holding Corp — Full financial report (2026 Q 1)
- investors.scholarrock.com[25]
- investors.scholarrock.com[26]
- Scholar Rock Holding Corp — Full financial report (2018 Y)[27][28]
- Scholar Rock Holding Corp — Full financial report (2020 Y)[33]
- Scholar Rock Holding Corp — Full financial report (2021 Y)[34]
- www.sec.gov[40]
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