Financial Performance - The company reported a net loss of $115.4 million for the six months ended June 30, 2024, with an accumulated deficit of $791.8 million[77]. - The net loss for Q2 2024 was $58.5 million, compared to a net loss of $37.9 million in Q2 2023, reflecting a 54.3% increase[86]. - Total operating expenses for the first half of 2024 were $117.9 million, up 48.2% from $79.6 million in the same period of 2023[93]. - Cash, cash equivalents, and marketable securities decreased by $89.4 million during the first half of 2024, totaling $190.5 million as of June 30, 2024[100]. - For the six months ended June 30, 2024, the company reported a net cash used in operating activities of $99.0 million, compared to $75.6 million for the same period in 2023[108]. - The company incurred net cash provided by financing activities of $6.5 million for the six months ended June 30, 2024, compared to $5.8 million in the same period of 2023[110]. - The company raised approximately $92.4 million from a public offering of 12,408,760 shares at $6.85 per share, which closed on October 16, 2023[102]. - The company has concluded that there is substantial doubt about its ability to continue as a going concern without additional financing[114]. - The company has incurred net losses every year since inception and anticipates continued net losses in the future[135]. - The company may not have sufficient cash, cash equivalents, and marketable securities to fund operations beyond one year without additional external financing[135]. Research and Development - Apitegromab is undergoing a pivotal Phase 3 clinical trial (SAPPHIRE) for SMA, with enrollment completed in 2023 and top-line data expected in Q4 2024[69]. - SRK-181 is in a Phase 1 clinical trial (DRAGON) for cancers resistant to anti-PD-(L)1 therapies, with enrollment completed in December 2023[73]. - The company is advancing SRK-439 towards a potential IND submission in 2025 for cardiometabolic disorders, including obesity[71]. - Research and development expenses are expected to remain substantial as the company continues its clinical trials and product development[81]. - Research and development expenses for Q2 2024 were $42.4 million, up 57.7% from $26.9 million in Q2 2023, primarily due to increased external costs associated with clinical trials[86]. - The external costs for the apitegromab program increased by $8.8 million, mainly due to clinical trial expenses, including the ONYX long-term extension study[88]. - The company anticipates substantial expenses related to the research and development of apitegromab and other product candidates, including costs for clinical trials and commercialization[111]. - The company acknowledges a high failure rate for drugs in clinical trials, which could adversely affect development timelines and regulatory approvals[151]. - Difficulties in patient enrollment for clinical trials may arise due to the small patient population for SMA, estimated at 20,000 in the U.S. and Europe[153]. Regulatory and Compliance - The FDA has granted multiple designations to apitegromab, including Fast Track and Orphan Drug designations[70]. - The company has received Orphan Drug designation from the FDA and the EC for apitegromab for the treatment of SMA[131]. - The regulatory approval process for product candidates is lengthy and unpredictable, with potential for delays in receiving approvals[131]. - The company must comply with extensive regulatory requirements for manufacturing and quality control, which are subject to periodic review and inspections[183]. - The FDA may require a REMS program as a condition of approval, which could entail long-term patient follow-up and additional safety measures[184]. - The company may face significant penalties if it fails to comply with ongoing regulatory obligations after receiving approval for its product candidates[182]. - Regulatory compliance and Good Clinical Practice (GCP) adherence are critical, with potential consequences for trial data reliability if not met[157]. - The company is subject to strict data protection laws, including the EU GDPR, which imposes penalties for non-compliance, potentially up to €20 million or 4% of total worldwide annual turnover for serious offenses[226]. - The UK GDPR, post-Brexit, may lead to additional compliance costs and regulatory challenges, with penalties for non-compliance up to £17.5 million or 4% of worldwide revenue[227]. Commercialization and Market Risks - The company expects to incur significant expenses related to commercialization capabilities if product candidates receive regulatory approval[78]. - The market acceptance of apitegromab and other product candidates is uncertain and depends on factors such as efficacy, clinical data, pricing, and physician willingness to prescribe[188]. - The biopharmaceutical industry is highly competitive, with many companies having greater financial and technical resources, which could impact the commercial opportunity for apitegromab if competitors develop superior products[190]. - Coverage and reimbursement from third-party payors are critical for the success of product candidates like apitegromab and SRK-181[252]. - There is significant uncertainty regarding insurance coverage and reimbursement for newly approved products, impacting potential revenue[255]. - The company anticipates pricing pressures due to increasing governmental efforts to cap healthcare costs and regulate drug pricing[257]. - EU regulations may materially affect the company's ability to market and receive coverage for products in European Member States[258]. Operational Challenges - The company relies on third parties for clinical trials and preclinical studies, which may lead to delays or inability to receive regulatory approval[130]. - The company is dependent on a limited number of third-party manufacturing and supply partners, which may affect the supply of materials[132]. - The company has outsourced significant parts of its IT and business infrastructure to third-party providers, increasing vulnerability to cyber-attacks and potential operational disruptions[211]. - The company relies on third parties for the manufacture of key products, including apitegromab, SRK-181, and SRK-439, which could be adversely affected by disruptions or security breaches[213]. - The company's laboratory operations are concentrated in one location, increasing vulnerability to business interruptions from natural disasters or other events[247]. - Global events, such as the 2022 Russian invasion of Ukraine, could adversely affect clinical trials and overall business operations due to geopolitical instability[248]. Human Resources and Management - The company anticipates challenges in managing growth as it continues to expand its organization and personnel[133]. - Future growth will require hiring additional personnel, which may be challenging in a competitive market for qualified candidates[204]. - The ability to attract and retain qualified management and scientific personnel is critical, and competition for talent may increase costs and hinder recruitment efforts[207]. - Transition in management or loss of key personnel could impair the company's ability to develop its product candidates[208].

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE QUARTERLY PERIOD ENDED March 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION PERIOD FROM _ TO _ COMMISSION FILE NUMBER 001-38501 ______________________________________________ SCHOLAR ROCK HOLDING CORPORATION (Exact name of registrant as sp ...

Exhibit 99.1 Scholar Rock Reports First Quarter 2024 Financial Results and Highlights Business Progress CAMBRIDGE, Mass.--(BUSINESS WIRE)— May 7, 2024-- Scholar Rock (NASDAQ: SRRK), a late-stage biopharmaceutical company focused on advancing innovative treatments for spinal muscular atrophy (SMA), cardiometabolic disorders, and other serious diseases where protein growth factors play a fundamental role, today reported financial results and corporate updates for the first quarter ended March 31, 2024. "Schol ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Fiscal Year Ended December 31, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission File Number: 001-38501 SCHOLAR ROCK HOLDING CORPORATION (Exact name of Registrant as specified in its charter) | Delaware 82-3750435 | | | | --- | --- | --- | | (Sta ...

Scholar Rock Holding Corporation (NASDAQ:SRRK) Q3 2023 Earnings Conference Call November 7, 2023 8:00 AM ET Company Participants Rushmie Nofsinger - Vice President, Investor Relations & Corporate Affairs Jay Backstrom - Chief Executive Officer Mo Qatanani - Head of Research. Ted Myles - Chief Operating Officer & Chief Financial Officer Conference Call Participants Michael Yee - Jefferies Allison Bratzel - Piper Sandler Tessa Romero - JPMorgan Srikripa Devarakonda - Truist Securities Etzer Darout - BMO Capit ...

Ted Myles, MBA Chief Operating Officer & Chief Financial Officer • Developing a novel, highly potent and selective antibody (SRK-439) for cardiometabolic disorders Dose Escalation (3+3) Cohort NSCLC 9 0 10 20 30 40 0 1000 2000 3000 Post Treatment % CD8 Positive Tumor nest size (cell count) Excluded Infiltrated Post-Treatment Pre-Treatment • 10 SD patients, 5 of which remain on study © 2023 Scholar Rock, Inc. All rights reserved. | --- | --- | |--------------------|-------| | | | | | | | | | | | | | | | | | ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE QUARTERLY PERIOD ENDED September 30, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION PERIOD FROM _ TO _ COMMISSION FILE NUMBER 001-38501 ______________________________________________ SCHOLAR ROCK HOLDING CORPORATION (Exact name of registrant a ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE QUARTERLY PERIOD ENDED June 30, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 (I.R.S. Employer Identification No.) 02142 (857) 259 3860 (Registrant's telephone number, including area code) Securities registered pursuant to Section 12(b) of the Exchange Act: FOR ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, D.C. 20549 FORM 10-Q ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE QUARTERLY PERIOD ENDED March 31, 2023 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 301 Binney Street, 3rd Floor Cambridge, Massachusetts (Address of principal executive offices) (Zip Code) (I.R.S. Employer Identification No.) 02142 (857) 259 3860 (Registrant's tele ...

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Fiscal Year Ended December 31, 2022 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Transition Period from to Commission File Number: 001-38501 SCHOLAR ROCK HOLDING CORPORATION (Exact name of Registrant as specified in its charter) | Delaware 82-375043 ...