Scholar Rock Holding Corp

Scholar Rock Holding Corporation and its subsidiaries (collectively, the “Company”) is 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. As a leader in the biology of the transforming growth factor beta (“TGFβ”) superfamily, the Company’s novel understanding of the molecular mechanisms of growth factor activation enabled the development of a proprietary platform for the discovery and development of monoclonal antibodies that locally and selectively target the precursor, or latent, forms of growth factors. Based on the Company’s innovative, proprietary, and scalable technology platform, the Company is building a world-leading anti-myostatin pipeline.

The Company has one product approved in the United States, ISEMBYLD™ (apitegromab-mstn), for the treatment of SMA. The Company's lead product candidates include a subcutaneous (“SC”) formulation of apitegromab and SRK-439.

Apitegromab is a fully human monoclonal antibody that inhibits myostatin activation by selectively binding the pro- and latent forms of myostatin in skeletal muscle. Myostatin is a catabolic agent that functions as a negative regulator of muscle mass, therefore inhibition of myostatin results in increased muscle mass and strength. On September 11, 2026, the U.S. Food and Drug Administration (“FDA”) approved ISEMBYLD for the treatment of SMA in adults and children two years of age and older who are currently receiving a survival motor neuron 2 (“SMN2”)-targeted treatment. ISEMBYLD is the first and only muscle-targeted treatment approved for patients with SMA currently receiving SMN2-targeted therapy. The Company has initiated the U.S. commercial launch of ISEMBYLD and the Company plans to seek marketing authorizations for apitegromab for use in SMA in other jurisdictions. In October 2026, the Company resubmitted its Marketing Authorisation Application (“MAA”) for apitegromab for the treatment of children and adults with SMA to the European Medicines Agency (“EMA”). In November 2026, the Company submitted a Japanese New Drug Application (“JNDA”) for apitegromab for the treatment of children and adults with SMA to Japan’s Pharmaceuticals and Medical Devices Agency (“PMDA”).

The Company continues to evaluate ISEMBYLD in SMA with its ongoing long-term extension ONYX trial and its Phase 2 OPAL trial in SMA patients under two years of age, which was initiated in the third quarter of 2025. Additionally, the Company is evaluating apitegromab in patients with facioscapulohumeral muscular dystrophy (“FSHD”) with its Phase 2 FORGE trial, which was initiated in the second quarter of 2026.

In addition to the current intravenous (“IV”) formulation, the Company is developing a SC formulation of apitegromab. A Phase 1 study in healthy volunteers has been completed and demonstrated that SC apitegromab has favorable bioavailability and a comparable pharmacodynamic profile relative to IV administered apitegromab. Further development activities are ongoing, including planned EMA regulatory engagements following potential apitegromab approvals in Europe.

The Company’s clinical-stage pipeline also includes SRK-439, a novel, investigational, subcutaneously administered fully human anti-pro/latent myostatin antibody that has high inhibitory potency while maintaining selectivity towards myostatin. SRK-439 is being developed for the treatment of patients with rare, severe, and debilitating neuromuscular diseases. A Phase 1 study of SRK-439 in healthy volunteers is currently underway. The company expects to have topline data by year end 2026 and plans to share data from this study in the first quarter of 2027.

Beyond these clinical-stage product candidates, the Company’s early-stage pipeline includes additional nonclinical programs intended for the treatment of patients with rare, severe, and debilitating neuromuscular diseases. As the Company focuses its strategy on rare neuromuscular diseases, the Company is currently seeking partnerships for its non-neuromuscular disease programs.

The Company was formed in May 2012 and has principal offices in Cambridge, Massachusetts. Since its inception, the Company’s operations have focused on research and development of monoclonal antibodies that selectively inhibit activation of growth factors for therapeutic effect, as well as establishing the Company’s intellectual property portfolio, performing research and development activities and developing commercialization capabilities to support product sales, marketing and distribution activities and following the approval of ISEMBYLD in September 2026, commercializing ISEMBYLD in the U.S. The Company has primarily financed its operations through various equity financings, the exercise of stock options and warrants, as well as research and development collaboration agreements and the Company’s debt facility (Note 13).

Revenue generation activities have historically been limited totwo collaborations, both containing research services and the issuance of a license. Following the FDA approval of ISEMBYLD in September 2026, the Company has commenced commercial activities and begun generating product revenue in the U.S.

The Company is subject to a number of risks similar to those of other life science companies, including, but not limited to, successful commercialization of its approved product, successful discovery and development of its drug candidates including new indications, raising additional capital, development by competitors of new technological innovations, protection of proprietary technology and regulatory approval and market acceptance of the Company’s product and product candidates. The Company anticipates that it will continue to incur significant operating losses for the next several years as it commercializes ISEMBYLD and continues to develop and seek regulatory approval for its new product candidates.

The Company believes that its existing cash, cash equivalents, marketable securities and cash available under the Company’s debt facility as of September 30, 2026 will be sufficient to allow the Company to fund its current operations through at least a period of one year after the date these financial statements are issued.