Nature of the Business	6 Months Ended
Jun. 30, 2025
Nature of the Business
Nature of the Business	1. Nature of the Business Scholar Rock Holding Corporation (the “Company”) is a late-stage biopharmaceutical company focused on developing and commercializing apitegromab for patients with spinal muscular atrophy (“SMA”) and other severe and debilitating neuromuscular diseases. As a global 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. By targeting the signaling proteins at the cellular level and acting in the disease microenvironment, the Company believes that it may avoid the historical dose-limiting safety challenges associated with inhibiting growth factors for therapeutic effect. The Company believes its focus on biologically validated growth factors may facilitate a more efficient development path. The Company’s first product candidate, apitegromab, is a highly selective, fully human, monoclonal antibody, with a unique mechanism of action that results in inhibition of the activation of the growth factor, myostatin, in skeletal muscle. Apitegromab is being developed as a potential first muscle-targeted treatment for SMA. In October 2024, the Company announced positive top-line results from SAPPHIRE, a pivotal Phase 3 clinical trial to evaluate the efficacy and safety of apitegromab in patients with non-ambulatory SMA. The study achieved its primary endpoint. The Company submitted a U.S. Biologics License Application (“BLA”) to the FDA in January 2025, which is now under priority review with a Prescription Drug User Fee Act (“PDUFA”) target action date of September 22, 2025. In March 2025, the Company submitted to the European Medicines Agency (“EMA”) and received validation of its marketing authorisation application (“MAA”) for apitegromab for the treatment of SMA. The FDA granted Fast Track designation, Rare Pediatric Disease designation and Orphan Drug designation to apitegromab for the treatment of SMA in May 2021, August 2020 and March 2018, respectively. The EMA granted Priority Medicines (“PRIME”) designation in March 2021 and the European Commission (“EC”) granted orphan medicinal product designation in December 2018 to apitegromab for the treatment of SMA. If apitegromab is approved on or around its PDUFA action date, the Company expects to initiate a commercial product launch in the fourth quarter of 2025 in the United States, with a commercial launch of apitegromab in Europe anticipated to follow in 2026. The Company continues to develop apitegromab in SMA with its ongoing long-term extension ONYX trial and its plan to initiate the Phase 2 OPAL trial in SMA patients under two years of age in the third quarter of 2025. In October 2023, the Company announced the expansion of its anti-myostatin program with SRK-439, a novel, fully human anti-myostatin monoclonal antibody with high potency and subcutaneous formulation. The Company intends to explore SRK-439’s potential in various, severe debilitating neuromuscular disorders and plans to submit an investigational new drug application in the second half of 2025. The Company’s product candidate, SRK-181, a highly selective inhibitor of the activation of latent TGFβ1, is being developed for the treatment of cancers that are resistant to checkpoint inhibitor therapies, such as anti-PD-1 or anti-PD-L1 antibody therapies (referred to together as anti-PD-(L)1 antibody therapies). SRK-181 has been evaluated in the Company’s Phase 1 DRAGON proof-of-concept clinical trial in patients with locally advanced or metastatic solid tumors that exhibit resistance to anti-PD-(L)1 antibody therapies. The Company completed the Phase 1 DRAGON trial in June 2025. This two-part clinical trial consisted of a dose escalation portion (Part A) and a dose expansion portion evaluating SRK-181 in combination with an approved anti-PD-(L)1 antibody therapy (Part B). Part B included the following cohorts: urothelial carcinoma, cutaneous melanoma, non-small cell lung cancer, clear cell renal cell carcinoma and head and neck squamous cell carcinoma. Additionally, the Company continues to discover and develop a pipeline of product candidates to deliver life-changing therapies for people with serious diseases that have high unmet need, including neuromuscular disorders, cardiometabolic disorders, cancer, and other conditions where growth factor-targeted therapies can play a transformational role. The Company was originally formed in May 2012. Its principal offices are 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 and performing research and development activities. The Company has primarily financed its operations through various equity financings, as well as research and development collaboration agreements and the Company’s debt facility (see Note 11). Revenue generation activities have been limited to two collaborations, both containing research services and the issuance of a license. No revenues have been recorded from the sale of any commercial product. The Company is subject to a number of risks similar to other life science companies, including, but not limited to, successful discovery and development of its drug candidates, raising additional capital, development by its competitors of new technological innovations, protection of proprietary technology and regulatory approval and market acceptance of the Company’s product candidates. The Company anticipates that it will continue to incur significant operating losses for the next several years as it continues to develop its product candidates. The Company believes that its existing cash, cash equivalents, marketable securities, and cash available to the Company at June 30, 2025 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.

Nature of the Business

6 Months Ended

Jun. 30, 2025

1. Nature of the Business

Scholar Rock Holding Corporation (the “Company”) is a late-stage biopharmaceutical company focused on developing and commercializing apitegromab for patients with spinal muscular atrophy (“SMA”) and other severe and debilitating neuromuscular diseases. As a global 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. By targeting the signaling proteins at the cellular level and acting in the disease microenvironment, the Company believes that it may avoid the historical dose-limiting safety challenges associated with inhibiting growth factors for therapeutic effect. The Company believes its focus on biologically validated growth factors may facilitate a more efficient development path.

The Company’s first product candidate, apitegromab, is a highly selective, fully human, monoclonal antibody, with a unique mechanism of action that results in inhibition of the activation of the growth factor, myostatin, in skeletal muscle. Apitegromab is being developed as a potential first muscle-targeted treatment for SMA. In October 2024, the Company announced positive top-line results from SAPPHIRE, a pivotal Phase 3 clinical trial to evaluate the efficacy and safety of apitegromab in patients with non-ambulatory SMA. The study achieved its primary endpoint. The Company submitted a U.S. Biologics License Application (“BLA”) to the FDA in January 2025, which is now under priority review with a Prescription Drug User Fee Act (“PDUFA”) target action date of September 22, 2025. In March 2025, the Company submitted to the European Medicines Agency (“EMA”) and received validation of its marketing authorisation application (“MAA”) for apitegromab for the treatment of SMA. The FDA granted Fast Track designation, Rare Pediatric Disease designation and Orphan Drug designation to apitegromab for the treatment of SMA in May 2021, August 2020 and March 2018, respectively. The EMA granted Priority Medicines (“PRIME”) designation in March 2021 and the European Commission (“EC”) granted orphan medicinal product designation in December 2018 to apitegromab for the treatment of SMA. If apitegromab is approved on or around its PDUFA action date, the Company expects to initiate a commercial product launch in the fourth quarter of 2025 in the United States, with a commercial launch of apitegromab in Europe anticipated to follow in 2026. The Company continues to develop apitegromab in SMA with its ongoing long-term extension ONYX trial and its plan to initiate the Phase 2 OPAL trial in SMA patients under two years of age in the third quarter of 2025.

In October 2023, the Company announced the expansion of its anti-myostatin program with SRK-439, a novel, fully human anti-myostatin monoclonal antibody with high potency and subcutaneous formulation. The Company intends to explore SRK-439’s potential in various, severe debilitating neuromuscular disorders and plans to submit an investigational new drug application in the second half of 2025.

The Company’s product candidate, SRK-181, a highly selective inhibitor of the activation of latent TGFβ1, is being developed for the treatment of cancers that are resistant to checkpoint inhibitor therapies, such as anti-PD-1 or anti-PD-L1 antibody therapies (referred to together as anti-PD-(L)1 antibody therapies). SRK-181 has been evaluated in the Company’s Phase 1 DRAGON proof-of-concept clinical trial in patients with locally advanced or metastatic solid tumors that exhibit resistance to anti-PD-(L)1 antibody therapies. The Company completed the Phase 1 DRAGON trial in June 2025. This two-part clinical trial consisted of a dose escalation portion (Part A) and a dose expansion portion evaluating SRK-181 in combination with an approved anti-PD-(L)1 antibody therapy (Part B). Part B included the following cohorts: urothelial carcinoma, cutaneous melanoma, non-small cell lung cancer, clear cell renal cell carcinoma and head and neck squamous cell carcinoma.

Additionally, the Company continues to discover and develop a pipeline of product candidates to deliver life-changing therapies for people with serious diseases that have high unmet need, including neuromuscular disorders, cardiometabolic disorders, cancer, and other conditions where growth factor-targeted therapies can play a transformational role. The Company was originally formed in May 2012. Its principal offices are 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 and performing research and development activities. The Company has primarily financed its operations through various equity financings, as well as research and development collaboration agreements and the Company’s debt facility (see Note 11).

Revenue generation activities have been limited to two collaborations, both containing research services and the issuance of a license. No revenues have been recorded from the sale of any commercial product.

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

The Company believes that its existing cash, cash equivalents, marketable securities, and cash available to the Company at June 30, 2025 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.