Financial Data and Key Metrics Changes - The company ended the quarter with $295 million in cash [28] - The company anticipates receiving approximately $16 million from the exercise of outstanding common warrants by year-end, extending its financial runway into 2027 [29] - The company has an additional $50 million available under its debt facility post-approval to support the upcoming launch [29] Business Line Data and Key Metrics Changes - The company is preparing for the U.S. launch of epitigramab, with a BLA accepted under priority review and a target action date of September 22, 2025 [6][8] - The company plans to expand epitigramab into additional rare, severe, and debilitating neuromuscular diseases [5][10] - The EMBRAZE study met its primary endpoint, showing that epitigramab increased lean mass preservation by over 54% compared to tirzepatide alone [11] Market Data and Key Metrics Changes - Approximately 10,000 patients in the U.S. are currently living with SMA, with about two-thirds having received SMN-targeted therapy [26] - Globally, around 35,000 patients have received SMN-targeted therapies, indicating a significant market opportunity for epitigramab [27] Company Strategy and Development Direction - The company aims to become a global biotech powerhouse by focusing on regulatory approvals for epitigramab, expanding into additional neuromuscular diseases, and disciplined capital allocation [5][31] - The first European launch of epitigramab is planned for Germany in 2026, with ambitions to reach patients across Europe, Asia Pacific, and Latin America [9][13] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ongoing dialogue with the FDA and the collaborative nature of the late cycle meeting, indicating a positive outlook for the BLA review [8][35] - The company is committed to ensuring that no patient with SMA is left behind, emphasizing the urgency of delivering epitigramab to the SMA community [31] Other Important Information - The company is working collaboratively with the FDA and European Medicines Agency to prepare for the commercial launch of epitigramab [6][9] - The company has assembled a highly experienced field team to support the launch of epitigramab [10] Q&A Session Summary Question: Can you tell us more about the specific observations at the two sites? - The company acknowledged that GMP inspections are standard and noted that they were disappointed but not surprised by the observations at two CDMO sites [34][35] Question: What is the current status of the two facilities in terms of product release? - Both facilities continue to operate and manufacture products, with the supply chain having produced over 600 patient years of experience through clinical trials [37][38] Question: How are discussions with FDA regarding the epitigramab review going? - The company reported constructive feedback from the late cycle meeting with the FDA and is working towards the September 22 PDUFA date [44][45] Question: What is the expected enrollment timeline for the OPAL trial? - Enrollment for the OPAL trial is expected to start in Q3 2025, with further guidance on completion timelines to be provided as momentum builds [78] Question: How does the company plan to address the budget impact of dual SMA therapies? - The company is considering the rarity and severity of SMA, along with the compelling clinical benefits of epitigramab, in discussions with payers [70][72]

Apitegromab in SMA - Positive Phase 3 trial data showed a 18 point improvement versus placebo in Hammersmith Functional Motor Scale-Expanded (HFMSE) in SMA patients[23] - 30% of apitegromab patients achieved ≥3-point improvement in HFMSE compared to 125% of placebo patients[23] - FDA accepted BLA under Priority Review with a target action date of September 22, 2025[25] - MAA Validated with anticipated EU approval in 2026[14] - The company is initiating Phase 2 OPAL Trial in Q3 to study apitegromab in patients under 2 years old[14, 27] Pipeline Expansion - EMBRAZE trial achieved primary endpoint with positive Phase 2 proof-of-concept in obesity[14] - Apitegromab combined with tirzepatide resulted in a 549% reduction in lean mass loss versus tirzepatide alone[36] - The company is on track to file IND for SRK-439 in 2H 2025[14, 39, 43] Financial Status - The company had $295 million cash as of June 30, 2025[14]

PART I [Item 1. Financial Statements (Unaudited)](index=8&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) Presents unaudited condensed consolidated financial statements and notes on financial position and performance [Condensed Consolidated Balance Sheets](index=8&type=section&id=Condensed%20Consolidated%20Balance%20Sheets%20as%20of%20June%2030,%202025%20and%20December%2031,%202024) Presents the company's financial position at specific dates, detailing assets, liabilities, and equity | Metric | June 30, 2025 (in thousands) | December 31, 2024 (in thousands) | Change (in thousands) | | :----- | :--------------------------- | :------------------------------- | :-------------------- | | Cash and cash equivalents | $152,925 | $177,878 | $(24,953) | | Marketable securities | $142,088 | $259,400 | $(117,312) | | Total current assets | $319,126 | $451,165 | $(132,039) | | Total assets | $340,045 | $474,922 | $(134,877) | | Total liabilities | $106,752 | $106,288 | $464 | | Total stockholders' equity | $233,293 | $368,634 | $(135,341) | [Condensed Consolidated Statements of Operations and Comprehensive Loss](index=9&type=section&id=Condensed%20Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss%20for%20the%20Three%20and%20Six%20Months%20Ended%20June%2030,%202025%20and%202024) Details the company's revenues, expenses, and net loss over specified three and six-month periods | Metric (in thousands) | 3 Months Ended June 30, 2025 | 3 Months Ended June 30, 2024 | Change ($) | Change (%) | | :-------------------- | :--------------------------- | :--------------------------- | :--------- | :--------- | | Research and development | $62,401 | $42,373 | $20,028 | 47.3% | | General and administrative | $49,708 | $17,125 | $32,583 | 190.3% | | Total operating expenses | $112,109 | $59,498 | $52,611 | 88.4% | | Net loss | $(110,031) | $(58,508) | $(51,523) | 88.1% | | Metric (in thousands) | 6 Months Ended June 30, 2025 | 6 Months Ended June 30, 2024 | Change ($) | Change (%) | | :-------------------- | :--------------------------- | :--------------------------- | :--------- | :--------- | | Research and development | $111,079 | $85,466 | $25,613 | 30.0% | | General and administrative | $78,120 | $32,451 | $45,669 | 140.7% | | Total operating expenses | $189,199 | $117,917 | $71,282 | 60.5% | | Net loss | $(184,754) | $(115,361) | $(69,393) | 60.2% | [Condensed Consolidated Statements of Stockholders' Equity](index=10&type=section&id=Condensed%20Consolidated%20Statements%20of%20Stockholders'%20Equity%20for%20the%20Six%20Months%20Ended%20June%2030,%202025%20and%202024) Tracks changes in the company's equity over the six months, including net loss and equity compensation | Metric (in thousands) | Balance at Dec 31, 2024 | Balance at June 30, 2025 | Change | | :-------------------- | :---------------------- | :----------------------- | :----- | | Total Stockholders' Equity | $368,634 | $233,293 | $(135,341) | | Net loss (6 months) | N/A | $(184,754) | N/A | | Equity-based compensation expense (6 months) | N/A | $37,846 | N/A | | Exercise of stock options (6 months) | N/A | $8,126 | N/A | [Condensed Consolidated Statements of Cash Flows](index=11&type=section&id=Condensed%20Consolidated%20Statements%20of%20Cash%20Flows%20for%20the%20Six%20Months%20Ended%20June%2030,%202025%20and%202024) Summarizes cash inflows and outflows from operating, investing, and financing activities over six months | Metric (in thousands) | 6 Months Ended June 30, 2025 | 6 Months Ended June 30, 2024 | Change | | :-------------------- | :--------------------------- | :--------------------------- | :----- | | Net cash used in operating activities | $(155,616) | $(98,981) | $(56,635) | | Net cash provided by investing activities | $120,020 | $84,006 | $36,014 | | Net cash provided by financing activities | $10,643 | $6,492 | $4,151 | | Net decrease in cash, cash equivalents and restricted cash | $(24,953) | $(8,483) | $(16,470) | [Notes to Condensed Consolidated Financial Statements](index=12&type=section&id=Notes%20to%20Condensed%20Consolidated%20Financial%20Statements) Provides detailed explanations and additional information supporting the consolidated financial statements [1. Nature of the Business](index=12&type=section&id=1.%20Nature%20of%20the%20Business) Describes Scholar Rock's focus as a late-stage biopharmaceutical company and its key product candidates - Scholar Rock is a late-stage biopharmaceutical company focused on developing and commercializing apitegromab for spinal muscular atrophy (SMA) and other neuromuscular diseases, utilizing a proprietary platform targeting the TGFβ superfamily[22](index=22&type=chunk)[79](index=79&type=chunk) - Apitegromab achieved its primary endpoint in the pivotal Phase 3 SAPPHIRE trial for non-ambulatory SMA, is under FDA priority review with a PDUFA target action date of **September 22, 2025**, and received EMA MAA validation. A U.S. commercial launch is expected in **Q4 2025**, with Europe to follow in **2026**, if approved[23](index=23&type=chunk)[82](index=82&type=chunk) - The company is expanding its anti-myostatin program with SRK-439, a novel, fully human anti-myostatin monoclonal antibody, with an investigational new drug (IND) application planned for the **second half of 2025**[24](index=24&type=chunk)[85](index=85&type=chunk) - SRK-181, a highly selective inhibitor of latent TGFβ1 activation for checkpoint inhibitor-resistant cancers, completed its Phase 1 DRAGON trial in **June 2025**, showing encouraging responses[25](index=25&type=chunk)[88](index=88&type=chunk) [2. Summary of Significant Accounting Policies](index=14&type=section&id=2.%20Summary%20of%20Significant%20Accounting%20Policies) Outlines the company's accounting policies and the impact of recent and future accounting pronouncements - The company's significant accounting policies remain materially unchanged from those disclosed in its Annual Report on Form 10-K for the year ended December 31, 2024[31](index=31&type=chunk) - ASU 2023-07 (Segment Reporting) was adopted and did not have a material impact on the condensed consolidated financial position and results of operations[35](index=35&type=chunk) - The company is evaluating the impact of ASU 2023-09 (Income Taxes) and ASU 2024-03 (Expense Disaggregation) on future disclosures, with effective dates in **2025** and **2026/2027**, respectively[36](index=36&type=chunk)[37](index=37&type=chunk) [3. Fair Value of Financial Assets and Liabilities](index=17&type=section&id=3.%20Fair%20Value%20of%20Financial%20Assets%20and%20Liabilities) Details fair value measurements of financial assets, primarily marketable securities and cash equivalents | Asset Category (in thousands) | June 30, 2025 (Total Fair Value) | December 31, 2024 (Total Fair Value) | | :---------------------------- | :------------------------------- | :----------------------------------- | | Money market funds | $132,321 | $97,290 | | U.S. treasury obligations (cash & cash equivalents) | $12,963 | $63,171 | | Marketable securities (U.S. treasury & government agency) | $142,088 | $259,400 | | **Total assets at fair value** | **$287,372** | **$419,861** | - All financial assets measured at fair value on a recurring basis (money market funds, U.S. treasury obligations, and government agency securities) are categorized as **Level 1**, indicating valuation using quoted market prices[38](index=38&type=chunk) [4. Marketable Securities](index=17&type=section&id=4.%20Marketable%20Securities)

[Filing Details](index=1&type=section&id=Filing%20Details) This section provides fundamental information regarding the Form 8-K filing, including registrant details and securities information [General Information](index=1&type=section&id=General%20Information) This section provides the foundational details of the Form 8-K filing, identifying Scholar Rock Holding Corporation as the registrant, the filing date, and its corporate contact information General Information Details | Detail | Information | | :--- | :--- | | **Registrant** | Scholar Rock Holding Corporation | | **Filing Type** | Form 8-K | | **Date of Report** | August 6, 2025 | | **Jurisdiction** | Delaware | | **Commission File Number** | 001-38501 | | **Address** | 301 Binney Street, 3rd Floor, Cambridge, MA 02142 | | **Telephone** | (857) 259-3860 | [Securities Information](index=1&type=section&id=Securities%20Information) This part details the company's registered securities and its status regarding emerging growth company provisions Registered Securities | Title of each class | Trading Symbol(s) | Name of each exchange on which registered | | :--- | :--- | :--- | | Common Stock, par value $0.001 per share | SRRK | The Nasdaq Global Select Market | - The registrant is **not an emerging growth company** as defined in Rule 405 of the Securities Act of 1933 or Rule 12b-2 of the Securities Exchange Act of 1934[3](index=3&type=chunk) [Report Items](index=2&type=section&id=Report%20Items) This section outlines specific disclosures required by Form 8-K, including financial results, Regulation FD disclosures, and a list of exhibits [Item 2.02. Results of Operations and Financial Condition](index=2&type=section&id=Item%202.02.%20Results%20of%20Operations%20and%20Financial%20Condition) Scholar Rock Holding Corporation issued a press release on August 6, 2025, announcing its financial and operating results for the fiscal quarter ended June 30, 2025. This press release is furnished as Exhibit 99.1 and is not considered 'filed' for certain legal purposes - Scholar Rock Holding Corporation issued a press release on **August 6, 2025**, announcing financial and operating results for the fiscal quarter ended **June 30, 2025**[4](index=4&type=chunk) - The press release is furnished as **Exhibit 99.1** to this Report on Form 8-K[4](index=4&type=chunk) - Information in this report and Exhibit 99.1 is furnished, not deemed 'filed' for Section 18 of the Exchange Act, and not incorporated by reference unless expressly stated[5](index=5&type=chunk) [Item 7.01. Regulation FD Disclosure](index=2&type=section&id=Item%207.01.%20Regulation%20FD%20Disclosure) The Company will host a conference call on August 6, 2025, at 8:00 am ET to discuss its Q2 2025 financial and operating results and provide a business update, utilizing presentation slides furnished as Exhibit 99.2. Similar to Item 2.02, this information is furnished, not filed - A conference call is scheduled for **8:00 am ET on August 6, 2025**, to discuss Q2 2025 financial results and provide a business update[6](index=6&type=chunk) - Presentation slides for the conference call are furnished as **Exhibit 99.2** to this Current Report on Form 8-K[6](index=6&type=chunk) - Information in this report and Exhibits 99.1 and 99.2 is furnished, not deemed 'filed' for Section 18 of the Exchange Act, and not incorporated by reference unless expressly stated[7](index=7&type=chunk) [Item 9.01. Financial Statements and Exhibits](index=2&type=section&id=Item%209.01.%20Financial%20Statements%20and%20Exhibits) This section lists all exhibits included with the Form 8-K filing, which comprise the press release, conference call presentation slides, and the interactive data file List of Exhibits | Exhibit No. | Description | | :--- | :--- | | 99.1 | Press Release issued by the Company on August 6, 2025, furnished hereto | | 99.2 | Presentation Slides for August 6, 2025 Conference Call, furnished hereto | | 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document) | [Signature](index=3&type=section&id=Signature) This section confirms the official signing of the Form 8-K report by an authorized corporate officer - The report was signed on behalf of **Scholar Rock Holding Corporation** by **Junlin Ho, General Counsel & Corporate Secretary**, on **August 6, 2025**[10](index=10&type=chunk)[12](index=12&type=chunk)

Business Overview - Scholar Rock's strategy focuses on selectivity in targeting latent growth factors to develop therapeutics across multiple disease areas[12, 15, 17, 19] - The company is advancing a pipeline of product candidates in neuromuscular disorders, cardiometabolic disorders, immuno-oncology, fibrosis, and anemia[20, 23] - Scholar Rock anticipates transformative execution in the next 12-24 months, driven by upcoming milestones and commercialization efforts[12] Apitegromab (Spinal Muscular Atrophy) - Topline readout from the Phase 3 SAPPHIRE trial in SMA is expected in Q4 2024, with a potential SMA launch in Q4 2025 if successful and approved[30] - 48-month data from the TOPAZ clinical trial showed substantial and sustained improvements in motor function outcomes in pooled nonambulatory SMA patients[39, 46, 52] - In the TOPAZ trial, pooled nonambulatory patients showed mean change from baseline in HFMSE of 2.8 at 6 months, 3.6 at 12 months, 4.2 at 24 months, 4.0 at 36 months, and 5.3 at 48 months[40] - In the TOPAZ trial, pooled nonambulatory patients showed mean change from baseline in RULM of 0.6 at 6 months, 1.3 at 12 months, 2.3 at 24 months, 2.4 at 36 months, and 3.6 at 48 months[47] Apitegromab (Obesity) & SRK-439 - A Phase 2 proof-of-concept EMBRAZE study of apitegromab in obesity is enrolling, with topline data expected in Q2 2025[23, 30, 60, 63] - SRK-439, a novel anti-myostatin antibody, is being advanced to IND submission, with preclinical data showing potential for healthier weight loss in combination with GLP-1 receptor agonists[23, 30, 75] - Preclinical data showed that SRK-439 increased lean mass and attenuated regain of fat mass after GLP-1 RA withdrawal in an obesity mouse model[78] - In preclinical studies, SRK-439 demonstrated greater potency than an anti-ActRII antibody at maintaining lean mass during GLP-1 RA-induced weight loss[77, 88]

Apitegromab in SMA - Positive Phase 3 SAPPHIRE data was presented at MDA, showing that 30% of apitegromab patients achieved ≥3-point improvement in HFMSE compared to 12.5% on placebo[19] - Apitegromab demonstrated a 1.8 point improvement (p=0.0192) in HFMSE versus placebo[19] - The FDA has accepted the BLA for Apitegromab under Priority Review, with a PDUFA date of September 22[22] - The company anticipates a commercial launch of Apitegromab in Q3, pending regulatory approval[37] - The company anticipates EU approval in 2026[10] Pipeline Development - The company plans to file the IND application for SRK-439 in Q3[26] - An exploratory Phase 2 EMBRAZE POC study in Obesity readout is on track for June[10] - The company plans to initiate Ph 2 OPAL Trial in Q3 2025 for SMA patients <2 years old[32] Financial Status - The company reported $364.4 million in cash as of March 31, 2025[10]

Core Insights - Scholar Rock Holding's shares increased by 13.7% following positive phase 2 trial results for its weight-loss therapy [1] - The trial demonstrated that the combination of Scholar Rock's apitegromab with Eli Lilly's tirzepatide significantly reduced lean mass loss compared to tirzepatide alone [2] - The therapy is well tolerated and has the potential to support healthier weight loss by preserving lean mass for patients on GLP therapies [3] Market Potential - The GLP-1 drug market, including Eli Lilly's Mounjaro and Novo Nordisk's Ozempic, was valued at $53.5 billion in 2024 and is projected to triple by 2030 [5] - Given the public's preference for fat loss over muscle loss, Scholar Rock's apitegromab could capture a significant share of this expanding market [5]

Core Insights - Scholar Rock's Phase 2 EMBRAZE trial results indicate that apitegromab combined with tirzepatide can significantly preserve lean mass during weight loss, with a notable reduction in lean mass loss compared to tirzepatide alone [1][2][3] Group 1: Trial Results - The trial showed that 30% of the weight loss from tirzepatide was attributed to lean mass loss, while the addition of apitegromab reduced this to 14.6% [3][4] - Patients receiving the apitegromab-tirzepatide combination lost 12.3% of their body weight, compared to 13.4% for those on tirzepatide with placebo [4] - The combination therapy resulted in a higher quality of weight loss, with patients losing 85% fat and 15% lean mass, compared to 70% fat and 30% lean mass in the placebo group [4] Group 2: Market Reaction - Following the positive trial results, Scholar Rock's stock price increased by 18%, reaching $36.77 [6]

Positive Ph2 EMBRAZE Trial Demonstrating Preservation of Lean Mass with Highly Selective Anti-Myostatin Inhibitor During Tirzepatide-Induced Weight Loss June 18, 2025 © 2025 Scholar Rock, Inc. All rights reserved. Today's Speakers David L. Hallal Chief Executive Officer Akshay Vaishnaw, M.D., Ph.D. President, Research & Development © 2025 Scholar Rock, Inc. All rights reserved. 2 Agenda | TOPIC | SPEAKER | | --- | --- | | Scholar Rock's Leading | David L. Hallal | | Anti-Myostatin Platform | Chief Executive ...

Scholar Rock Holding (SRRK) Update / Briefing June 18, 2025 08:00 AM ET Speaker0 Good morning, and welcome to ScholarRock's presentation of top line data from the Phase two EMPRAISE proof of concept trial. At this time, all lines are in listen only mode. Following the presentation, we will conduct a question and answer session. To ask a question, you may press star then 11 on your touch tone phone. To withdraw your question, please press then 11 again. This call is being recorded on Wednesday, 06/18/2025. I ...