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, 2021 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 82-3750435 ...

Deep Insights Advancing Impactful Medicines 40th Annual J.P. Morgan Healthcare Conference January 10-13, 2022 ScholarRock. | --- ...

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, 2021 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 ...

Deep Insights, Impactful Medicines August 2021 ScholarRock. Disclaimers Various statements in this presentation concerning the future expectations, plans and prospects of Scholar Rock, Inc. ("Scholar Rock"), including without limitation, Scholar Rock's expectations regarding its strategy, its product candidate selection and development timing, including timing for the initiation of and reporting results from its clinical trials for its product candidates, its disease indication selection and timing for such ...

PART I - FINANCIAL INFORMATION [Item 1. Financial Statements](index=7&type=section&id=Item%201.%20Financial%20Statements) This section presents the unaudited consolidated financial statements, including balance sheets, statements of operations, and cash flows, reflecting increased net loss and cash usage driven by higher R&D expenses [Consolidated Balance Sheets](index=7&type=section&id=Consolidated%20Balance%20Sheets) As of June 30, 2021, the company's total assets were $332.1 million, a decrease from $388.3 million at year-end 2020, primarily due to a reduction in marketable securities Consolidated Balance Sheet Highlights (in thousands) | Account | June 30, 2021 | December 31, 2020 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $206,919 | $160,358 | | Marketable securities | $75,298 | $180,673 | | Total current assets | $290,366 | $344,404 | | **Total assets** | **$332,051** | **$388,305** | | **Liabilities & Equity** | | | | Total current liabilities | $67,022 | $42,564 | | Total liabilities | $115,395 | $127,535 | | Total stockholders' equity | $216,656 | $260,770 | | **Total liabilities and stockholders' equity** | **$332,051** | **$388,305** | [Consolidated Statements of Operations and Comprehensive Loss](index=8&type=section&id=Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) For the three and six months ended June 30, 2021, revenues were $4.6 million and $9.3 million, respectively, with net losses increasing to $30.7 million and $58.4 million due to higher R&D and G&A expenses Statement of Operations Summary (in thousands, except per share data) | Metric | Q2 2021 | Q2 2020 | 6 Months 2021 | 6 Months 2020 | | :--- | :--- | :--- | :--- | :--- | | Revenue | $4,595 | $3,900 | $9,303 | $8,930 | | Research and development | $25,603 | $16,997 | $48,152 | $33,899 | | General and administrative | $9,265 | $6,365 | $18,631 | $12,187 | | Loss from operations | $(30,273) | $(19,462) | $(57,480) | $(37,156) | | **Net loss** | **$(30,707)** | **$(19,281)** | **$(58,378)** | **$(36,351)** | | Net loss per share | $(0.84) | $(0.65) | $(1.60) | $(1.23) | [Consolidated Statements of Cash Flows](index=10&type=section&id=Consolidated%20Statements%20of%20Cash%20Flows) For the six months ended June 30, 2021, net cash used in operating activities significantly increased to $58.4 million, while investing activities provided $101.6 million, resulting in a $46.6 million increase in cash to $209.4 million Cash Flow Summary for the Six Months Ended June 30 (in thousands) | Cash Flow Activity | 2021 | 2020 | | :--- | :--- | :--- | | Net cash used in operating activities | $(58,357) | $(17,191) | | Net cash provided by investing activities | $101,574 | $95,017 | | Net cash provided by financing activities | $3,344 | $994 | | **Net increase in cash, cash equivalents and restricted cash** | **$46,561** | **$78,820** | | **Cash, cash equivalents and restricted cash, end of period** | **$209,417** | **$117,626** | [Notes to Consolidated Financial Statements](index=11&type=section&id=Notes%20to%20Consolidated%20Financial%20Statements) The notes provide detailed information on the company's business, accounting policies, and financial statement line items, including product candidates, collaboration terms, and debt facility details - The company's lead product candidate, apitegromab, is being developed for Spinal Muscular Atrophy (SMA) and is anticipated to start a **Phase 3 trial** by year-end 2021, while SRK-181 is in a **Phase 1 trial** for cancers resistant to checkpoint inhibitors[21](index=21&type=chunk) - Under the Gilead collaboration, the company recognized **$4.6 million** and **$9.3 million** in revenue for the three and six months ended June 30, 2021, respectively, with **$42.7 million** remaining in deferred revenue as of June 30, 2021[54](index=54&type=chunk) - In October 2020, the company entered into a **$50.0 million** loan agreement, with the first **$25.0 million** tranche funded and a second **$25.0 million** tranche available through December 31, 2021, contingent on clinical trial milestones[48](index=48&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=22&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses business overview, clinical developments, and COVID-19 impact, detailing increased operating expenses, particularly R&D, and the need for additional capital despite current liquidity into 2023 [Overview and COVID-19 Impact](index=22&type=section&id=Overview%20and%20COVID-19%20Impact) The company, a biopharmaceutical firm, is advancing apitegromab (SMA) and SRK-181 (cancer) candidates, while navigating COVID-19 related disruptions to clinical trials and supply chains - **Positive 12-month top-line data** from the TOPAZ **Phase 2 trial** for apitegromab in SMA was announced in April 2021, with a pivotal **Phase 3 trial** expected to start by the end of 2021[66](index=66&type=chunk)[71](index=71&type=chunk) - The DRAGON **Phase 1 trial** for SRK-181 is progressing, with plans to advance to the dose expansion portion (Part B) in mid-2021 and initial data from Part A expected by the end of 2021[73](index=73&type=chunk) - The **COVID-19 pandemic** has caused disruptions, including four patients in the TOPAZ trial missing three doses each due to site access restrictions and slower enrollment in the DRAGON trial[80](index=80&type=chunk)[174](index=174&type=chunk) [Results of Operations](index=34&type=section&id=Results%20of%20Operations) Operating expenses significantly increased, driven by higher R&D costs for apitegromab manufacturing and SRK-181 clinical trials, alongside growth in general and administrative expenses Comparison of Results for the Six Months Ended June 30 (in thousands) | Item | 2021 | 2020 | Change ($) | Change (%) | | :--- | :--- | :--- | :--- | :--- | | Revenue | $9,303 | $8,930 | $373 | 4.2% | | Research and development | $48,152 | $33,899 | $14,253 | 42.0% | | General and administrative | $18,631 | $12,187 | $6,444 | 52.9% | | **Net loss** | **$(58,378)** | **$(36,351)** | **$(22,027)** | **60.6%** | - The **$14.3 million** increase in R&D expense for the first six months of 2021 was driven by a **$7.9 million** increase in apitegromab costs (mainly manufacturing) and a **$9.4 million** increase in internal costs (personnel and new facility), partially offset by a **$3.4 million** decrease in SRK-181 costs[108](index=108&type=chunk)[109](index=109&type=chunk)[113](index=113&type=chunk) [Liquidity and Capital Resources](index=40&type=section&id=Liquidity%20and%20Capital%20Resources) As of June 30, 2021, the company had $282.2 million in cash, cash equivalents, and marketable securities, sufficient to fund operations into 2023, but additional capital will be required to complete clinical development - The company's cash, cash equivalents, and marketable securities totaled **$282.2 million** as of June 30, 2021[117](index=117&type=chunk) - Existing cash is expected to fund operating expenses and capital expenditure requirements into **2023**[130](index=130&type=chunk) - Key funding sources include a November 2020 follow-on offering that raised approximately **$215.9 million** in net proceeds and a **$50.0 million** debt facility, of which **$25.0 million** has been drawn[118](index=118&type=chunk)[119](index=119&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=46&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company is a smaller reporting company and is not required to provide the information for this item - As a **smaller reporting company**, Scholar Rock is not required to provide quantitative and qualitative disclosures about market risk[141](index=141&type=chunk) [Item 4. Controls and Procedures](index=46&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that the company's disclosure controls and procedures were effective as of June 30, 2021, with no material changes in internal control over financial reporting during the period - Management concluded that the company's disclosure controls and procedures were **effective** as of June 30, 2021[143](index=143&type=chunk) - No changes in internal control over financial reporting occurred during the six months ended June 30, 2021, that materially affected, or are reasonably likely to materially affect, internal controls[146](index=146&type=chunk) PART II. OTHER INFORMATION [Item 1. Legal Proceedings](index=49&type=section&id=Item%201.%20Legal%20Proceedings) The company states that it is not party to any material legal proceedings that would be reasonably expected to have a material adverse effect on its business - As of the date of the report, the company is not party to any claim or litigation expected to have a **material adverse effect** on the business[147](index=147&type=chunk) [Item 1A. Risk Factors](index=50&type=section&id=Item%201A.%20Risk%20Factors) This section details numerous risks and uncertainties across product development, manufacturing, operations, intellectual property, financial condition, and common stock - The company identifies several **material risks**, including the **lengthy and expensive process** of product development with an **uncertain outcome**, potential adverse effects from the **COVID-19 pandemic**, **reliance on third parties** for clinical trials and manufacturing, and the **need for additional capital**[150](index=150&type=chunk)[152](index=152&type=chunk)[155](index=155&type=chunk) [Risks Related to Product Development and Regulatory Approval](index=53&type=section&id=Risks%20Related%20to%20Product%20Development%20and%20Regulatory%20Approval) This subsection outlines the significant uncertainties in drug development, including high costs, long timelines, unpredictable regulatory processes, and potential for adverse events or enrollment difficulties - Product development is a **lengthy, expensive process** with an **uncertain outcome**, and the company may experience delays or be unable to complete the development and commercialization of its product candidates[160](index=160&type=chunk) - The **COVID-19 pandemic** has impacted and may continue to impact clinical trials through enrollment delays, site access restrictions, and supply chain disruptions[170](index=170&type=chunk) - The results of preclinical studies and early-stage clinical trials may not predict future results, and interim data may change as more patient data becomes available[179](index=179&type=chunk)[180](index=180&type=chunk) [Risks Related to Manufacturing and Supply](index=79&type=section&id=Risks%20Related%20to%20Manufacturing%20and%20Supply) The company's dependence on third-party contract manufacturers for all preclinical and clinical product supplies creates risks of supply interruption, quality control issues, and scaling challenges - The company relies on **third-party manufacturers** for all clinical trial supplies and does not own its own manufacturing facilities, creating risks of supply interruption or quality issues[243](index=243&type=chunk) - The company relies on a **single source supplier** for the drug substance for both apitegromab and SRK-181, which could create significant disruption if this relationship is impacted[243](index=243&type=chunk) [Risks Related to Our Business and Operations](index=83&type=section&id=Risks%20Related%20to%20Our%20Business%20and%20Operations) This section covers operational risks, including managing organizational growth, retaining key personnel, protecting against cybersecurity threats, ensuring compliance with complex healthcare regulations, and dependence on the Gilead collaboration - The company needs to grow its organization and may experience difficulties managing this growth, including attracting and retaining **qualified personnel**[255](index=255&type=chunk) - Failure to maintain the **Gilead Collaboration Agreement** or failure by Gilead to perform its obligations could negatively impact the business[332](index=332&type=chunk) - The business is subject to **complex healthcare laws and regulations**, including anti-kickback statutes and data privacy laws, non-compliance with which could result in **significant penalties**[279](index=279&type=chunk)[289](index=289&type=chunk) [Risks Related to Intellectual Property](index=117&type=section&id=Risks%20Related%20to%20Intellectual%20Property) The company's success is highly dependent on its ability to obtain and maintain patent, trademark, and trade secret protection, facing risks from application failures, validity challenges, third-party infringement claims, and global protection difficulties - The company's success depends on its ability to obtain and maintain **patent and trade secret protection**, which is a difficult and costly process[348](index=348&type=chunk) - The company depends on **intellectual property licensed from third parties**, and failure to comply with license obligations could result in the loss of significant rights[358](index=358&type=chunk) - Third-party claims of **intellectual property infringement** could prevent or delay product development and lead to costly litigation[374](index=374&type=chunk) [Risks Related to Our Financial Condition and Capital Requirements](index=135&type=section&id=Risks%20Related%20to%20Our%20Financial%20Condition%20and%20Capital%20Requirements) This subsection details the company's history of net losses and the expectation that losses will continue, highlighting the primary risk of needing to raise additional capital to fund operations and complete product development - The company has incurred **net losses** every year since inception and anticipates this will continue as it advances its R&D programs[405](index=405&type=chunk) - The company will require **additional capital** to complete the development and commercialization of its product candidates, and failure to obtain it could force delays or discontinuation of programs[410](index=410&type=chunk) - The ability to use net operating loss carryforwards of **$168.4 million** (federal) and **$170.1 million** (state) may be limited by ownership changes under Section 382 of the tax code[415](index=415&type=chunk) [Risks Related to Our Common Stock](index=141&type=section&id=Risks%20Related%20to%20Our%20Common%20Stock) Risks related to the company's common stock include price volatility, no dividend intention, significant insider ownership concentration, and reduced reporting requirements as an "emerging growth company" and "smaller reporting company" - The trading price of the company's common stock is subject to **high volatility**[419](index=419&type=chunk) - As of June 30, 2021, executive officers, directors, and their affiliates beneficially hold approximately **17.7%** of the outstanding voting stock, allowing them to exert **significant control**[422](index=422&type=chunk) - The company is an **"emerging growth company"** and a **"smaller reporting company,"** which allows for reduced public company reporting requirements[426](index=426&type=chunk)[429](index=429&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=150&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) This section indicates that there were no unregistered sales of equity securities during the reporting period - The company reports **no unregistered sales** of equity securities for the period[446](index=446&type=chunk) [Item 5. Other Information](index=150&type=section&id=Item%205.%20Other%20Information) The company reports that there is no other information to disclose for this item - No information was reported under this item[449](index=449&type=chunk) [Item 6. Exhibits](index=151&type=section&id=Item%206.%20Exhibits) This section provides an index of the exhibits filed with the Form 10-Q, including corporate governance documents, material contracts, and officer certifications

Apitegromab Positioned to be the Potential Next Transformative Therapy for Patients Suffering with SMA KOL Event and Panel Discussion June 15, 2021 & ScholarRock. Disclaimers Various statements in this presentation concerning the future expectations, plans and prospects of Scholar Rock, Inc. ("Scholar Rock"), including without limitation, Scholar Rock's expectations regarding its strategy, its product candidate selection and development timing, including timing for the initiation of and reporting results fr ...

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, 2021 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 ...

Washington, D.C. 20549 FORM 10-K (Mark One) Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the Fiscal Year Ended December 31, 2020 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 82-3750435 ...

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, 2020 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 ...

Deep Insights, Impactful Medicines September 2020 ScholarRock. Disclaimers Various statements in this presentation concerning Scholar Rock's future expectations, plans and prospects, including without limitation, Scholar Rock's expectations regarding its strategy, its product candidate selection and development timing, including timing for the initiation of and reporting results from its clinical trials for its product candidates, its disease indication selection and timing for such selection, the ability o ...