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Here are 1-2 brief analogies for Scholar Rock (SRRK):

• A specialized biotech akin to Sarepta Therapeutics, but with a focus on spinal muscular atrophy and other rare muscle-related disorders.
• An early-stage Vertex Pharmaceuticals, developing novel therapies for serious diseases like neuromuscular disorders and fibrosis.

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• Apitegromab (SRK-015): A muscle-directed antibody in Phase 3 development, designed to inhibit myostatin activation and improve muscle function in patients with Spinal Muscular Atrophy.
• SRK-181: A TGFβ1-targeting antibody in Phase 1 development, aimed at overcoming resistance to checkpoint inhibitors and treating advanced cancers.
• SRK-503: An investigational program in preclinical development, targeting the TGFβ superfamily for potential treatment across a range of fibrotic and other diseases.

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Scholar Rock (SRRK) is a clinical-stage biopharmaceutical company focused on discovering and developing novel medicines. As such, it does not currently have commercialized products generating revenue from direct sales to individuals, hospitals, or pharmacies.

Therefore, Scholar Rock sells primarily to other companies, specifically larger pharmaceutical companies that enter into strategic collaborations or licensing agreements to fund the research, development, and potential commercialization of Scholar Rock's drug candidates.

Based on Scholar Rock's financial disclosures, its major customer and primary source of collaboration revenue is:

• Gilead Sciences, Inc. (GILD)

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David Hallal, Chief Executive Officer & Chairman

David Hallal has served as Chief Executive Officer of Scholar Rock since April 2025, and as Chairman of its Board of Directors since July 2017. He is a biopharmaceutical industry veteran with 30 years of experience in growing and operating successful biotechnology companies. Prior to Scholar Rock, he served as CEO & Chairman of ElevateBio, a cell and gene therapy company. Before ElevateBio, David spent over a decade at Alexion Pharmaceuticals as Chief Executive Officer, Chief Operating Officer, and Chief Commercial Officer, where he was instrumental in transforming the company from a pre-commercial stage in 2006 to over $3 billion in annual revenues by 2016, and transitioning it from a single-product to a multi-product enterprise. He previously held commercial leadership roles at OSI Eyetech, Biogen, and Amgen. He also serves as Chairman of the Board of Directors of ElevateBio and iTeos Therapeutics SA, and as a member of the Board of Directors of Seer Biosciences, Inc.

Vikas Sinha, Chief Financial Officer

Vikas Sinha has served as Chief Financial Officer of Scholar Rock since April 2025. He brings over 30 years of executive leadership experience in the life sciences industry, covering finance, investor relations, business development, strategy, IT, and human resources. Prior to joining Scholar Rock, he co-founded and served as CFO of ElevateBio, where he remains a Board member. He also held the roles of President and CFO at AlloVir Inc. Earlier in his career, Vikas served as CFO at Alexion Pharmaceuticals, playing a pivotal role in the company's growth from a market capitalization of $600 million to over $30 billion, and from no revenue to more than $3 billion annually. He held multiple leadership positions at Bayer AG across the United States, Japan, Germany, and Canada, including Vice President and CFO of Bayer Pharmaceuticals Corporation (U.S.) and Vice President and CFO of Bayer Yakuhin Ltd. (Japan). Vikas currently serves as an independent board member and Audit Committee Chair at Verona Pharma and a board member at Orna Therapeutics.

Akshay Vaishnaw M.D., Ph.D., President, R&D

Akshay Vaishnaw was appointed President of R&D for Scholar Rock in April 2025. He has been a member of Scholar Rock's Board of Directors since 2019. Dr. Vaishnaw previously served as President and President of R&D at Alnylam Pharmaceuticals.

R. Keith Woods, Chief Operating Officer

R. Keith Woods was appointed Chief Operating Officer of Scholar Rock in April 2025. In this role, he focuses on evolving the organization into a fully integrated global enterprise. Prior to joining Scholar Rock, he served as COO of argenx.

Junlin Ho, J.D., General Counsel & Corporate Secretary

Junlin Ho serves as General Counsel & Corporate Secretary for Scholar Rock. She joined Scholar Rock in 2018 and has served as Senior Vice President, Head of Legal and Corporate Secretary since March 2020. As General Counsel, she is responsible for overseeing all legal affairs, including corporate governance, compliance, and intellectual property. She has over 13 years of experience in leading and advising life sciences companies on various legal aspects.

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1. Manufacturing and Regulatory Delays for Apitegromab: The most immediate and significant risk stems from delays in the manufacturing process and subsequent regulatory hurdles for its lead asset, Apitegromab. The company recently received a Complete Response Letter (CRL) from the FDA due to manufacturing site issues, not clinical setbacks, which has delayed the anticipated approval. Furthermore, a manufacturing partner has received an FDA warning letter, further raising questions about the launch timing of Apitegromab. Resolving these issues is critical for the drug's eventual approval and commercialization.
2. Financial Durability and Ongoing Losses: Scholar Rock operates as a clinical-stage biotech without any approved revenue-generating assets, leading to substantial accumulated deficits and continuous net losses. The high costs associated with drug development contribute to these financial pressures. This financial position raises concerns about the company's ability to sustain long-term operations without achieving commercial success or securing additional funding, making financial durability a critical risk.
3. Dependence on Apitegromab's Success and Commercialization Challenges: Scholar Rock's near-term prospects are heavily dependent on the successful development and commercialization of Apitegromab. While Apitegromab has shown positive Phase 3 trial results, there is considerable commercial uncertainty, particularly when competing with established large pharmaceutical companies like Roche and Biogen, which already market standard-of-care treatments for spinal muscular atrophy (SMA). The threat of competitors developing more effective or less costly treatments also poses a risk to Apitegromab's market potential.

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Pliant Therapeutics' PNT2258, an inhibitor of αvβ6 and αvβ1 integrins which activate latent TGFβ, is a clear emerging threat. PNT2258 is in Phase 2 clinical trials for fibrotic diseases, including idiopathic pulmonary fibrosis (IPF), and has reported positive data. This directly competes with Scholar Rock's preclinical SRK-439, which also targets latent TGFβ activation for fibrotic diseases. Pliant's significant lead in clinical development and early positive data for a mechanistically similar approach poses a substantial threat to Scholar Rock's future market positioning in fibrosis.

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Scholar Rock's primary product with a stated addressable market is:

• Apitegromab for Spinal Muscular Atrophy (SMA): Scholar Rock estimates a global market opportunity of greater than $2 billion for apitegromab. The company is preparing for a U.S. commercial launch in the fourth quarter of 2025 and a European rollout in 2026, pending approval.

For other pipeline products such as SRK-181 (oncology) and SRK-439 (obesity/cardiometabolic indications), specific addressable market sizes are not explicitly quantified in the provided information in monetary value or patient numbers. Therefore, for these products, the addressable market size is null.

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Scholar Rock (SRRK) anticipates several key drivers for future revenue growth over the next 2-3 years, primarily centered around its lead drug candidate, apitegromab, and its broader pipeline development.

1. U.S. and European Commercial Launch of Apitegromab for Spinal Muscular Atrophy (SMA): The most significant and immediate driver of future revenue growth is the anticipated commercial launch of apitegromab for the treatment of Spinal Muscular Atrophy. Following the receipt of a Complete Response Letter (CRL) solely related to manufacturing site observations, Scholar Rock expects to resubmit its Biologics License Application (BLA) to the FDA, with approval anticipated by 2026, leading to a U.S. commercial launch. In Europe, the Marketing Authorization Application (MAA) has been validated, with approval expected in 2026, and a subsequent European launch planned to commence with Germany. Analysts project apitegromab has the potential to generate over $2 billion in global revenue in SMA, addressing a critical unmet need for therapies that improve muscle strength in SMA patients.
2. Expansion of Apitegromab into New Indications and Patient Populations: Scholar Rock aims to broaden the market for apitegromab beyond its initial SMA indication by exploring additional patient populations and therapeutic areas. This includes plans to initiate a Phase 2 OPAL clinical trial in Q3 2025 to evaluate apitegromab in SMA patients under two years of age. Furthermore, positive topline data from the Phase 2 EMBRAZE proof-of-concept trial in obesity demonstrated apitegromab's potential to preserve lean mass during weight loss, suggesting a new, large market opportunity in cardiometabolic disorders. The company also intends to initiate clinical development in at least one additional neuromuscular indication by the end of 2025.
3. Advancement of Next-Generation Pipeline Candidate SRK-439: Scholar Rock is progressing SRK-439, a novel subcutaneous anti-myostatin antibody, toward an Investigational New Drug (IND) filing in the second half of 2025. This development could expand the company's therapeutic pipeline in neuromuscular disorders and potentially other indications, offering a next-generation approach to myostatin inhibition.

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Share Issuance

• In November 2020, Scholar Rock completed an underwritten public offering of common stock and pre-funded warrants, resulting in aggregate gross proceeds of approximately $230 million.
• In October 2024, the company commenced a public offering for $275 million of its common stock and pre-funded warrants.
• In October 2024, underwriters fully exercised their option to purchase an additional 1,592,920 shares at $28.25 per share, generating approximately $45 million in additional gross proceeds.

Inbound Investments

• Scholar Rock has access to an additional $50 million under its debt facility in the current year, with a further $50 million accessible post-approval to support the apitegromab launch.
• The company anticipates receiving approximately $16 million from the exercise of outstanding common warrants by year-end 2025.
• Scholar Rock expects to monetize its priority review voucher after drug approval, which is anticipated to generate additional proceeds.

Capital Expenditures

• As of December 31, 2024, Scholar Rock had approximately $437.3 million in cash, cash equivalents, and marketable securities, projected to fund operating and capital expenditure requirements into the fourth quarter of 2026.
• As of March 31, 2025, the company reported $364.4 million in cash, cash equivalents, and marketable securities, expected to fund anticipated operating and capital expenditure requirements into 2027.
• Capital expenditures are primarily focused on commercial readiness and inventory build-out for apitegromab, advancing clinical programs like SRK-439, and developing its technology platform.