Core Viewpoint - Scholar Rock's shares surged 362% following the announcement of positive results from the late-stage study of apitegromab for treating spinal muscular atrophy (SMA) [1] Company Overview - Scholar Rock is focused on developing apitegromab, a fully human monoclonal antibody that inhibits myostatin activation, which is crucial for muscle growth regulation [2] - The company has seen an 82.3% increase in shares year-to-date, contrasting with a 2.8% decline in the industry [7] Clinical Study Results - The phase III SAPPHIRE study met its primary endpoint, showing a statistically significant improvement in motor function in SMA patients treated with apitegromab compared to placebo at week 52, measured by the Hammersmith Functional Motor Scale Expanded (HFMSE) [3] - In the main efficacy group (ages 2-12), 30% of patients on apitegromab achieved a 3-point or more improvement in HFMSE, compared to 12.5% in the placebo group [4] - Early motor function gains were observed as soon as eight weeks into treatment, with continued improvement at 52 weeks [5] Safety Profile - Apitegromab was well-tolerated across all age groups, with no significant differences in adverse events between the two dosage strengths (10 mg/kg and 20 mg/kg) [7] - Serious adverse events were not attributed to apitegromab, and no patients discontinued the study due to adverse events [7] Regulatory and Future Plans - Scholar Rock plans to submit regulatory applications for apitegromab in the U.S. and EU in Q1 2025, with existing designations such as Fast Track and Orphan Drug in the U.S. and Priority Medicines in the EU [8] - Detailed data from the SAPPHIRE study is expected to be presented at a medical conference in early 2025 [9] Other Developmental Programs - Scholar Rock is also evaluating apitegromab for cardiometabolic disorders, with a phase II EMBRAZE study completed for preserving lean muscle mass in individuals on GLP-1 receptor agonist therapy [10] - The company is advancing SRK-439, a selective myostatin inhibitor, for treating cardiometabolic disorders [11] - Another candidate, SRK-181, is in a phase I study for advanced cancer treatment [12] Market Impact - Following Scholar Rock's positive data, Biohaven's stock rose 10.8%, as investors anticipate favorable results from its own SMA study [13] - Biohaven is evaluating taldefgrobep alfa (BHV-2000) in a phase III study for SMA, with top-line data expected by the end of 2024 [14]

Group 1 - Scholar Rock Holding Corp. (SRRK) shares increased by 335% following positive Phase 3 trial results for their drug apitegromab, which targets spinal muscular atrophy (SMA) and significantly improves motor function in patients [1] - In the SAPPHIRE study, patients receiving apitegromab experienced an average increase of over 3 points in the Hammersmith Functional Motor Scale Expanded (HFMSE) at week 52, while only 12.5% of placebo patients achieved similar improvements [2] - Patients on apitegromab began to see enhancements in motor function as early as week 8, with these improvements sustained throughout the 52-week study [2] Group 2 - Scholar Rock plans to submit regulatory applications for apitegromab to the U.S. FDA and the European Medicines Agency (EMA) in Q1 2025, potentially providing new treatment options for SMA patients and fostering growth in the biopharmaceutical sector [3]

Core Insights - Scholar Rock Holding Corporation (NASDAQ: SRRK) reported positive results from its phase 3 SAPPHIRE study for apitegromab, aimed at treating spinal muscular atrophy (SMA) [2] Group 1 - The SAPPHIRE study results indicate a significant advancement in the treatment options available for SMA patients [2] - The article highlights the importance of the Biotech Analysis Central service, which provides in-depth analysis of pharmaceutical companies and investment opportunities [2] Group 2 - The author of the article is affiliated with Biotech Analysis Central, which offers a library of over 600 biotech investing articles and a model portfolio of small and mid-cap stocks [2]

Market Overview - U.S. stocks traded lower with the Dow Jones index falling more than 200 points, down 0.49% to 42,146.78, NASDAQ down 0.36% to 18,073.37, and S&P 500 down 0.32% to 5,732.84 [1] - Energy shares increased by 0.5%, while consumer discretionary shares decreased by 1.3% [1] Company News - AstraZeneca entered an exclusive license agreement with CSPC Pharmaceutical Group to develop a novel small-molecule Lp(a) disruptor for dyslipidemia patients [2] - Scholar Rock Holding Corporation's shares surged 310% to $30.44 after positive Phase 3 SAPPHIRE trial results for apitegromab in SMA patients [2] - Arcadium Lithium PLC shares rose 41% to $4.34 following acquisition interest from Rio Tinto [2] - Duckhorn Portfolio Inc shares increased by 102% to $10.88 after better-than-expected Q4 results and an acquisition agreement with Butterfly Equity [2] Declining Stocks - Trevena, Inc. shares dropped 48% to $2.4192 due to a Nasdaq delisting notification [3] - Heritage Insurance Holdings, Inc. shares fell 21% to $9.65 [3] - Universal Insurance Holdings, Inc. shares decreased 18% to $17.12 [3] Commodities - Oil prices increased by 3.4% to $76.99, while gold prices decreased by 0.2% to $2,662.50 [4] - Silver fell 1.1% to $32.025, and copper decreased by 0.5% to $4.5535 [4] Economic Indicators - Eurozone retail sales rose 0.2% month-over-month in August, while the Halifax House Price Index in the UK increased by 4.7% year-over-year in September [5] - Germany's factory orders declined by 5.8% month-over-month in August [5] Asia Pacific Markets - Asian markets closed mixed with Japan's Nikkei 225 gaining 1.80% and Hong Kong's Hang Seng Index gaining 1.60%, while India's BSE Sensex dipped 0.78% [6] Foreign Exchange Reserves - Singapore's foreign exchange reserves fell to SGD 499.7 billion in September from SGD 501.3 billion [7] - Japan's leading economic indicators index fell to 106.7 in August, and the coincident economic indicators index declined to 113.5 [7]

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. | --- | --- | |--------------------|-------| | | | | | | | | | | | | | | | | | ...