UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ 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: 001-38978 FULCRUM THERAPEUTICS, INC. (Exact Name of Registrant as Specified in its Charter) Emerging growth company ☒ If an emerging ...

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, 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 001-38978 FULCRUM THERAPEUTICS, INC. (Exact name of registrant as specified in its Charter) Delaware 47-4839948 (State or other jurisdict ...

[Filing Information and Forward-Looking Statements](index=1&type=section&id=Filing%20Information%20and%20Forward-Looking%20Statements) This section outlines Fulcrum Therapeutics, Inc.'s Form 10-Q filing details, company status, and cautionary forward-looking statements [Form 10-Q Filing Details](index=1&type=section&id=Form%2010-Q%20Filing%20Details) This section outlines Fulcrum Therapeutics, Inc.'s Form 10-Q filing details, including its non-accelerated, smaller reporting, and emerging growth company status - **Fulcrum Therapeutics, Inc. is filing a Quarterly Report on Form 10-Q for the period ended September 30, 2020**[2](index=2&type=chunk) Registrant Status | Status | Value | | :---------------------- | :-------------------- | | Trading Symbol | FULC | | Exchange | Nasdaq Global Market | | Non-accelerated filer | ☒ | | Smaller reporting company | ☒ | | Emerging growth company | ☒ | - **As of November 4, 2020, 27,462,565 shares of common stock were outstanding**[4](index=4&type=chunk) [Forward-Looking Statements](index=2&type=section&id=Forward-Looking%20Statements) This section provides cautionary statements about forward-looking information, emphasizing risks and uncertainties that could materially alter actual results - **The report contains forward-looking statements regarding operations and financial performance, subject to various risks and uncertainties**[7](index=7&type=chunk) - **Actual outcomes could differ materially due to factors such as ongoing clinical trials for losmapimod (FSHD, COVID-19) and FTX-6058, the impact of the COVID-19 pandemic, and R&D program timing and results**[8](index=8&type=chunk) - **Additional risks include funding capabilities, market acceptance of product candidates, intellectual property position, and the progress of collaborations with Acceleron Pharma Inc. and MyoKardia, Inc**[8](index=8&type=chunk)[11](index=11&type=chunk) [PART I. FINANCIAL INFORMATION](index=5&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) This part presents the unaudited consolidated financial statements, including balance sheets, statements of operations, cash flows, and management's discussion [Item 1. Financial Statements (Unaudited)](index=5&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) This section provides the unaudited consolidated financial statements, encompassing balance sheets, statements of operations, cash flows, and detailed notes [Consolidated Balance Sheets](index=5&type=section&id=Consolidated%20Balance%20Sheets) This section presents the company's financial position, detailing assets, liabilities, and equity at specific points in time Consolidated Balance Sheet Highlights (in thousands) | Metric | September 30, 2020 | December 31, 2019 | | :-------------------------------- | :------------------- | :------------------ | | Cash and cash equivalents | $85,221 | $96,713 | | Marketable securities | $41,804 | — | | Total current assets | $132,112 | $100,083 | | Total assets | $142,215 | $110,439 | | Total current liabilities | $26,455 | $12,140 | | Total liabilities | $37,230 | $23,286 | | Total stockholders' equity | $104,985 | $87,153 | [Consolidated Statements of Operations and Comprehensive Loss](index=6&type=section&id=Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss) This section outlines the company's financial performance over specific periods, including revenue, expenses, and net loss Consolidated Statements of Operations and Comprehensive Loss Highlights (in thousands, except per share data) | Metric | Three Months Ended Sep 30, 2020 | Three Months Ended Sep 30, 2019 | Nine Months Ended Sep 30, 2020 | Nine Months Ended Sep 30, 2019 | | :----------------------------------- | :------------------------------ | :------------------------------ | :----------------------------- | :----------------------------- | | Collaboration revenue | $1,848 | — | $4,598 | — | | Research and development | $15,640 | $13,496 | $42,897 | $58,985 | | General and administrative | $5,312 | $3,510 | $15,525 | $8,742 | | Net loss | $(18,962) | $(16,542) | $(53,099) | $(66,554) | | Net loss per share (basic and diluted) | $(0.70) | $(0.97) | $(2.16) | $(10.33) | [Consolidated Statements of Convertible Preferred Stock and Stockholders' Equity (Deficit)](index=7&type=section&id=Consolidated%20Statements%20of%20Convertible%20Preferred%20Stock%20and%20Stockholders'%20Equity%20(Deficit)) This section details changes in the company's equity, including preferred stock conversions and movements in paid-in capital and accumulated deficit - **Upon IPO completion on July 22, 2019, all 112,500,000 outstanding preferred shares converted into 16,071,418 common shares**[59](index=59&type=chunk) - **Additional paid-in capital increased from $237.9 million at December 31, 2019, to $308.8 million at September 30, 2020**[20](index=20&type=chunk) - **Accumulated deficit increased from $(150.8) million at December 31, 2019, to $(203.9) million at September 30, 2020**[20](index=20&type=chunk) [Consolidated Statements of Cash Flows](index=9&type=section&id=Consolidated%20Statements%20of%20Cash%20Flows) This section summarizes the cash inflows and outflows from operating, investing, and financing activities over specific periods Consolidated Statements of Cash Flows Highlights (in thousands) | Cash Flow Activity | Nine Months Ended Sep 30, 2020 | Nine Months Ended Sep 30, 2019 | | :----------------------------------- | :----------------------------- | :----------------------------- | | Net cash used in operating activities | $(33,829) | $(34,988) | | Net cash used in investing activities | $(42,540) | $(836) | | Net cash provided by financing activities | $64,877 | $64,624 | [Notes to Consolidated Financial Statements](index=10&type=section&id=Notes%20to%20Consolidated%20Financial%20Statements) This section provides detailed explanations and disclosures supporting the consolidated financial statements, covering accounting policies and transactions [Note 1. Nature of the Business and Basis of Presentation](index=10&type=section&id=Note%201.%20Nature%20of%20the%20Business%20and%20Basis%20of%20Presentation) This note describes Fulcrum Therapeutics, Inc.'s focus on rare diseases, operating losses, and expected capital runway into Q2 2022 - **Fulcrum Therapeutics, Inc. focuses on improving the lives of patients with genetically defined rare diseases**[25](index=25&type=chunk) - **The company has incurred recurring losses and negative cash flows from operations since inception, with an accumulated deficit of $203.9 million as of September 30, 2020**[32](index=32&type=chunk) - **Existing cash, cash equivalents, and marketable securities are expected to fund operating expenses and capital expenditure requirements for at least twelve months from the financial statements' issuance date**[33](index=33&type=chunk) [Note 2. Summary of Significant Accounting Policies](index=12&type=section&id=Note%202.%20Summary%20of%20Significant%20Accounting%20Policies) This note details the company's significant accounting policies, noting no material changes during the nine months ended September 30, 2020 - **Consolidated financial statements are prepared in conformity with GAAP**[27](index=27&type=chunk) - **No material changes in significant accounting policies occurred during the nine months ended September 30, 2020, except for marketable securities and recently adopted pronouncements**[35](index=35&type=chunk) - **Recently adopted accounting pronouncements (ASU 2017-08, ASU 2017-11, ASU 2018-18) had no material impact on the consolidated financial statements**[44](index=44&type=chunk)[45](index=45&type=chunk)[46](index=46&type=chunk) [Note 3. Fair Value Measurements](index=14&type=section&id=Note%203.%20Fair%20Value%20Measurements) This note classifies financial assets measured at fair value within Level 1 and Level 2 of the fair value hierarchy, with no transfers between levels Fair Value Measurements at September 30, 2020 (in thousands) | Asset Type | Total | Level 1 | Level 2 | Level 3 | | :---------------------- | :------ | :------ | :------ | :------ | | Money market funds | $85,221 | $85,221 | — | — | | U.S. Treasury securities | $14,998 | — | $14,998 | — | | Corporate bonds | $13,780 | — | $13,780 | — | | Commercial paper | $13,026 | — | $13,026 | — | | **Total** | **$127,025** | **$85,221** | **$41,804** | **—** | - **No transfers between fair value levels occurred during the three and nine months ended September 30, 2020**[50](index=50&type=chunk) [Note 4. Marketable Securities](index=15&type=section&id=Note%204.%20Marketable%20Securities) This note details the company's $41.8 million in marketable securities as of September 30, 2020, all with unrealized gains and maturities under one year Marketable Securities at September 30, 2020 (in thousands) | Security Type | Amortized Cost | Unrealized Gains | Unrealized Losses | Fair Value | | :---------------------- | :------------- | :--------------- | :---------------- | :--------- | | U.S. Treasury securities | $14,996 | $2 | — | $14,998 | | Corporate bonds | $13,751 | $29 | — | $13,780 | | Commercial paper | $13,026 | — | — | $13,026 | | **Total marketable securities** | **$41,773** | **$31** | **—** | **$41,804** | - **The company did not hold any marketable securities as of December 31, 2019**[51](index=51&type=chunk) - **As of September 30, 2020, no securities were in an unrealized loss position, and all marketable securities have a remaining contractual maturity of less than one year**[52](index=52&type=chunk) [Note 5. Property and Equipment, Net](index=15&type=section&id=Note%205.%20Property%20and%20Equipment,%20Net) This note shows a decrease in net property and equipment to $8.4 million as of September 30, 2020, primarily due to accumulated depreciation Property and Equipment, Net (in thousands) | Category | September 30, 2020 | December 31, 2019 | | :----------------------- | :------------------- | :------------------ | | Lab equipment | $6,468 | $5,710 | | Leasehold improvements | $6,210 | $6,210 | | Total property and equipment | $13,844 | $13,152 | | Less: accumulated depreciation | $(5,449) | $(3,947) | | **Property and equipment, net** | **$8,395** | **$9,205** | - **Depreciation expense for the nine months ended September 30, 2020, was $1.6 million, compared to $1.5 million for the same period in 2019**[53](index=53&type=chunk) [Note 6. Additional Balance Sheet Detail](index=15&type=section&id=Note%206.%20Additional%20Balance%20Sheet%20Detail) This note details an increase in prepaid expenses and other current assets to $4.5 million and a rise in accrued expenses to $8.7 million, driven by R&D accruals Prepaid Expenses and Other Current Assets (in thousands) | Category | September 30, 2020 | December 31, 2019 | | :-------------------------------- | :------------------- | :------------------ | | Prepaid expenses | $4,286 | $2,796 | | **Total prepaid expenses and other current assets** | **$4,495** | **$3,370** | Accrued Expenses and Other Current Liabilities (in thousands) | Category | September 30, 2020 | December 31, 2019 | | :-------------------------------- | :------------------- | :------------------ | | External research and development | $5,954 | $2,250 | | Payroll and benefits | $2,255 | $2,239 | | **Total accrued expenses and other current liabilities** | **$8,743** | **$5,496** | [Note 7. Preferred Stock](index=16&type=section&id=Note%207.%20Preferred%20Stock) This note states 5,000,000 shares of undesignated preferred stock were authorized but none issued, as all prior preferred stock converted to common stock post-IPO - **5,000,000 shares of undesignated preferred stock were authorized as of September 30, 2020, and December 31, 2019**[56](index=56&type=chunk) - **No shares of preferred stock were issued or outstanding as of September 30, 2020, and December 31, 2019**[56](index=56&type=chunk) - **Upon IPO completion on July 22, 2019, all 112,500,000 outstanding Preferred Stock shares automatically converted into 16,071,418 common stock shares**[59](index=59&type=chunk) [Note 8. Common Stock](index=16&type=section&id=Note%208.%20Common%20Stock) This note states the company is authorized to issue 200,000,000 common shares, with 27,277,983 outstanding, and details an unused $75.0 million 'at-the-market' offering program - **The company is authorized to issue 200,000,000 shares of common stock**[61](index=61&type=chunk) - **27,277,983 shares of common stock were outstanding as of September 30, 2020**[14](index=14&type=chunk) - **An 'at-the-market' offering program for up to $75.0 million was established on August 11, 2020, with no shares sold through September 30, 2020**[63](index=63&type=chunk) [Note 9. Stock-based Compensation Expense](index=17&type=section&id=Note%209.%20Stock-based%20Compensation%20Expense) This note details stock-based compensation expense of $1.8 million and $5.7 million for the three and nine months ended September 30, 2020, with $16.7 million unrecognized expense remaining Total Stock-based Compensation Expense (in thousands) | Period | 2020 | 2019 | | :----------------------------------- | :----- | :----- | | Three Months Ended September 30, | $1,776 | $1,179 | | Nine Months Ended September 30, | $5,673 | $2,950 | - **As of September 30, 2020, the company had an aggregate of $16.7 million of unrecognized stock-based compensation expense, expected to be recognized over a weighted average period of 2.62 years**[74](index=74&type=chunk) - **Outstanding stock options as of September 30, 2020, totaled 2,868,693 shares with a weighted average exercise price of $11.62**[71](index=71&type=chunk) [Note 10. Collaboration and License Agreements](index=19&type=section&id=Note%2010.%20Collaboration%20and%20License%20Agreements) This note details collaboration and license agreements with Acceleron Pharma Inc. and MyoKardia, Inc., involving upfront payments, milestones, and royalties - **Acceleron Collaboration Agreement (December 2019) included a $10.0 million upfront payment, with eligibility for up to $438.5 million in milestones and tiered royalties**[79](index=79&type=chunk) - **MyoKardia Collaboration Agreement (July 2020) included a $10.0 million upfront payment and $2.5 million prepaid research funding, with eligibility for up to $298.5 million per target in milestones and tiered royalties**[94](index=94&type=chunk) Collaboration Revenue and Deferred Revenue (in thousands) | Collaboration | Revenue (9 Months Ended Sep 30, 2020) | Deferred Revenue (Sep 30, 2020) | | :-------------- | :------------------------------------ | :------------------------------ | | Acceleron | $4,300 | $7,400 | | MyoKardia | $300 | $12,200 | [Note 11. Asset Acquisition](index=24&type=section&id=Note%2011.%20Asset%20Acquisition) This note describes the February 2019 acquisition of an exclusive worldwide license to losmapimod from GSK, with its $25.6 million fair value expensed as in-process R&D - **An exclusive worldwide license to develop and commercialize losmapimod was acquired from GSK in February 2019**[106](index=106&type=chunk) - **12,500,000 shares of Series B Preferred Stock were issued to GSK with an estimated fair value of $25.5 million**[107](index=107&type=chunk) - **The fair value of $25.6 million, inclusive of transaction costs, was recorded as in-process research and development expense in Q1 2019**[109](index=109&type=chunk) [Note 12. Commitments and Contingencies](index=24&type=section&id=Note%2012.%20Commitments%20and%20Contingencies) This note outlines a 10-year operating lease for the corporate headquarters with $20.0 million in minimum payments and confirms no material legal proceedings - **The company has a 10-year operating lease for its corporate headquarters with a total commitment of $25.1 million**[110](index=110&type=chunk) Future Minimum Lease Payments (in thousands) | Year | Amount | | :----- | :----- | | 2020 (remaining) | $579 | | 2021 | $2,354 | | 2022 | $2,424 | | 2023 | $2,497 | | 2024 | $2,572 | | Thereafter | $9,615 | | **Total minimum lease payments** | **$20,041** | - **The company is not currently a party to any material legal proceedings and has not incurred any material costs from indemnification agreements**[114](index=114&type=chunk)[115](index=115&type=chunk) [Note 13. Defined Contribution Plan](index=25&type=section&id=Note%2013.%20Defined%20Contribution%20Plan) This note confirms the company offers a 401(k) Plan to eligible employees but is not required to make, and has not made, any contributions - **The company has a defined contribution savings plan under Section 401(k) of the Internal Revenue Code**[116](index=116&type=chunk) - **The company is not required to make and has not made any contributions to the 401(k) Plan**[116](index=116&type=chunk) [Note 14. Net Loss per Share](index=25&type=section&id=Note%2014.%20Net%20Loss%20per%20Share) This note explains that common stock equivalents were excluded from diluted net loss per share calculations due to their anti-dilutive effect Common Stock Equivalents Excluded from Diluted EPS Calculation | Category | September 30, 2020 | September 30, 2019 | | :----------------------------- | :------------------- | :------------------- | | Outstanding stock options | 2,868,693 | 1,981,457 | | Unvested restricted stock awards | 181,213 | 782,548 | | **Total** | **3,049,906** | **2,764,005** | [Note 15. Related-Party Transactions](index=26&type=section&id=Note%2015.%20Related-Party%20Transactions) This note discloses less than $0.1 million in consulting fees paid to Third Rock Ventures in 2019, with no such expenses recorded in 2020 - **Less than $0.1 million in consulting fees was paid to Third Rock Ventures (TRV) during the nine months ended September 30, 2019**[118](index=118&type=chunk) - **No expenses related to TRV fees were recorded during the three and nine months ended September 30, 2020**[118](index=118&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=27&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) This section offers management's analysis of the company's financial condition and operational results, focusing on its clinical-stage pipeline, R&D efforts, and funding needs [Overview](index=27&type=section&id=Overview) This overview introduces Fulcrum Therapeutics as a clinical-stage biopharmaceutical company focused on rare diseases, highlighting key product candidates and financial performance - **Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on genetically defined rare diseases**[121](index=121&type=chunk) - **Key product candidates include losmapimod for FSHD (Phase 2b/2 open label) and COVID-19 (Phase 3 initiated in Q4 2020), and FTX-6058 for hemoglobinopathies (Phase 1 initiated in Q4 2020)**[121](index=121&type=chunk)[126](index=126&type=chunk)[127](index=127&type=chunk) - **The company incurred net losses of $19.0 million (Q3 2020) and $53.1 million (9 months 2020), with an accumulated deficit of $203.9 million as of September 30, 2020, and expects continued significant operating losses**[129](index=129&type=chunk) [Components of Results of Operations](index=29&type=section&id=Components%20of%20Results%20of%20Operations) This section details the revenue and expense components contributing to the company's operating results, including collaboration revenue, R&D, and general and administrative expenses - **Revenue is solely derived from collaboration agreements with Acceleron and MyoKardia, with no product sales anticipated for several years**[134](index=134&type=chunk)[135](index=135&type=chunk)[139](index=139&type=chunk) - **Research and development expenses are expensed as incurred, encompassing external costs, personnel, IPR&D, and facilities, and are projected to increase significantly**[144](index=144&type=chunk)[145](index=145&type=chunk)[149](index=149&type=chunk)[152](index=152&type=chunk) - **General and administrative expenses are expected to increase due to expanded infrastructure and costs associated with operating as a public company**[154](index=154&type=chunk) [Results of Operations](index=33&type=section&id=Results%20of%20Operations) This section provides a comparative analysis of the company's financial performance for the three and nine months ended September 30, 2020 and 2019 Comparison of Three Months Ended September 30, 2020 and 2019 (in thousands) | Metric | 2020 | 2019 | Change ($) | | :-------------------------- | :----- | :----- | :--------- | | Collaboration revenue | $1,848 | — | $1,848 | | Research and development | $15,640 | $13,496 | $2,144 | | General and administrative | $5,312 | $3,510 | $1,802 | | Net loss | $(18,962) | $(16,542) | $(2,420) | Comparison of Nine Months Ended September 30, 2020 and 2019 (in thousands) | Metric | 2020 | 2019 | Change ($) | | :-------------------------- | :----- | :----- | :--------- | | Collaboration revenue | $4,598 | — | $4,598 | | Research and development | $42,897 | $58,985 | $(16,088) | | General and administrative | $15,525 | $8,742 | $6,783 | | Net loss | $(53,099) | $(66,554) | $13,455 | - **The decrease in R&D expense for the nine-month period was primarily due to a $25.6 million decrease in in-process R&D expenses associated with the GSK agreement in 2019, partially offset by increased manufacturing and clinical activities in 2020**[163](index=163&type=chunk) [Liquidity and Capital Resources](index=35&type=section&id=Liquidity%20and%20Capital%20Resources) This section discusses the company's sources of funding, current cash position, and projected capital runway, along with a summary of cash flow activities - **Operations have been primarily funded through equity offerings (private placement, IPO) and upfront payments from collaboration agreements**[165](index=165&type=chunk) - **As of September 30, 2020, cash, cash equivalents, and marketable securities totaled $127.0 million, expected to fund operations into the second quarter of 2022**[165](index=165&type=chunk)[172](index=172&type=chunk) Cash Flow Summary (in thousands) | Cash Flow Activity | Nine Months Ended Sep 30, 2020 | Nine Months Ended Sep 30, 2019 | | :----------------------------------- | :----------------------------- | :----------------------------- | | Net cash used in operating activities | $(33,829) | $(34,988) | | Net cash used in investing activities | $(42,540) | $(836) | | Net cash provided by financing activities | $64,877 | $64,624 | [Contractual Obligations](index=38&type=section&id=Contractual%20Obligations) This section confirms no material changes to contractual obligations and commitments since the last annual report - **No material changes to contractual obligations and commitments have occurred since the Annual Report on Form 10-K filed on March 5, 2020**[176](index=176&type=chunk) [Off-Balance Sheet Arrangements](index=38&type=section&id=Off-Balance%20Sheet%20Arrangements) This section states that the company had no off-balance sheet arrangements during the reported periods - **The company did not have any off-balance sheet arrangements during the periods presented**[177](index=177&type=chunk) [Critical Accounting Policies and Estimates](index=38&type=section&id=Critical%20Accounting%20Policies%20and%20Estimates) This section highlights the significant judgments and estimates required for financial statement preparation, noting no material changes to critical accounting policies - **The preparation of financial statements requires judgments and estimates, particularly for revenue recognition, accrued expenses, stock-based compensation, and fair value of common/preferred stock**[178](index=178&type=chunk) - **No material changes to critical accounting policies occurred during the three months ended September 30, 2020**[178](index=178&type=chunk) [Recently Issued Accounting Pronouncements](index=38&type=section&id=Recently%20Issued%20Accounting%20Pronouncements) This section refers to Note 2 for a description of recently issued accounting pronouncements - **A description of recently issued accounting pronouncements is disclosed in Note 2 to the consolidated financial statements**[179](index=179&type=chunk) [Emerging Growth Company Status](index=38&type=section&id=Emerging%20Growth%20Company%20Status) This section confirms the company's status as an 'emerging growth company' under the JOBS Act and its election not to opt out of the extended transition period - **The company is an 'emerging growth company' (EGC) under the JOBS Act**[180](index=180&type=chunk) - **The company has elected not to 'opt out' of the extended transition period for complying with new or revised financial accounting standards**[180](index=180&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=38&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) This section details the company's primary market risk as interest rate sensitivity on its $127.0 million cash and marketable securities, with minimal foreign currency exposure - **Primary market risk is interest rate sensitivity, affecting cash equivalents and marketable securities totaling $127.0 million as of September 30, 2020**[181](index=181&type=chunk) - **Minimal exposure to foreign currency exchange rate risk exists, with no hedging currently in place**[183](index=183&type=chunk) - **Inflation did not have a material effect on the business during the three and nine months ended September 30, 2020 and 2019**[184](index=184&type=chunk) [Item 4. Controls and Procedures](index=39&type=section&id=Item%204.%20Controls%20and%20Procedures) This section confirms management's evaluation of disclosure controls and procedures as effective, with no material changes in internal control over financial reporting - **Disclosure controls and procedures were evaluated as effective at the reasonable assurance level as of September 30, 2020**[185](index=185&type=chunk) - **No material change in internal control over financial reporting occurred during the period covered by this Quarterly Report on Form 10-Q**[186](index=186&type=chunk) [PART II. OTHER INFORMATION](index=40&type=section&id=PART%20II.%20OTHER%20INFORMATION) This part provides additional information beyond the financial statements, including risk factors, equity sales, exhibits, and official signatures [Item 1A. Risk Factors](index=40&type=section&id=Item%201A.%20Risk%20Factors) This section details various risks that could materially and adversely affect the company's business, financial condition, and future growth [Risks Related to our Financial Position and Need for Additional Capital](index=40&type=section&id=Risks%20Related%20to%20our%20Financial%20Position%20and%20Need%20for%20Additional%20Capital) This section outlines financial risks, including significant operating losses, the need for additional funding, and the potential impact of the COVID-19 pandemic - **The company has incurred significant operating losses since inception, with an accumulated deficit of $203.9 million as of September 30, 2020, and expects to incur losses for the foreseeable future**[190](index=190&type=chunk) - **Substantial additional funding will be needed to support ongoing operations and growth strategy, with existing capital expected to fund operations only into the second quarter of 2022**[193](index=193&type=chunk)[196](index=196&type=chunk) - **The ongoing COVID-19 pandemic has and may continue to affect the ability to initiate or complete clinical trials, disrupt regulatory activities, and adversely impact business and operations**[203](index=203&type=chunk)[206](index=206&type=chunk) [Risks Related to the Discovery and Development of our Product Candidates](index=45&type=section&id=Risks%20Related%20to%20the%20Discovery%20and%20Development%20of%20our%20Product%20Candidates) This section addresses risks associated with product candidate discovery and development, including high failure rates, trial uncertainties, and regulatory approval challenges - **The company is in early development with only two product candidates in clinical trials (losmapimod and FTX-6058), facing a high risk of failure in the lengthy and expensive development process**[216](index=216&type=chunk)[229](index=229&type=chunk) - **Development of losmapimod for COVID-19 faces uncertainties regarding trial timing, patient enrollment, and potential unexpected safety issues**[218](index=218&type=chunk)[219](index=219&type=chunk)[221](index=221&type=chunk) - **Clinical trials may produce negative or inconclusive results, or new endpoints or methodologies may not be considered clinically meaningful by regulatory authorities, potentially delaying or preventing approval**[231](index=231&type=chunk)[237](index=237&type=chunk) [Risks Related to the Commercialization of our Product Candidates](index=56&type=section&id=Risks%20Related%20to%20the%20Commercialization%20of%20our%20Product%20Candidates) This section covers risks related to commercializing product candidates, including market acceptance, competition, lack of sales infrastructure, and reliance on CMOs - **Even if approved, product candidates may fail to achieve sufficient market acceptance due to competition, pricing, or inadequate third-party coverage and reimbursement**[250](index=250&type=chunk)[265](index=265&type=chunk)[266](index=266&type=chunk) - **The company lacks a sales and marketing infrastructure and faces substantial competition from companies with greater resources and expertise**[252](index=252&type=chunk)[255](index=255&type=chunk)[258](index=258&type=chunk) - **Reliance on contract manufacturing organizations (CMOs) for supply introduces risks of delays, quality issues, and regulatory non-compliance, potentially impacting development and commercialization**[262](index=262&type=chunk)[263](index=263&type=chunk)[264](index=264&type=chunk) [Risks Related to our Dependence on Third Parties](index=61&type=section&id=Risks%20Related%20to%20our%20Dependence%20on%20Third%20Parties) This section highlights risks stemming from reliance on third parties, including CROs, manufacturers, and collaboration partners, which could lead to delays or non-performance - **The company relies heavily on third-party clinical research organizations (CROs) to conduct clinical trials, which reduces control and poses risks of non-performance, delays, or non-compliance with regulatory requirements**[273](index=273&type=chunk)[275](index=275&type=chunk) - **Reliance on third-party manufacturers for product candidates increases the risk of insufficient quantities, unacceptable cost or quality, and potential delays in development or commercialization**[279](index=279&type=chunk)[280](index=280&type=chunk) - **Collaborations with third parties, including Acceleron and MyoKardia, may not be successful, as collaborators have significant discretion and potential for disputes or termination, impacting revenue and development**[284](index=284&type=chunk)[285](index=285&type=chunk)[286](index=286&type=chunk) [Risks Related to our Intellectual Property](index=65&type=section&id=Risks%20Related%20to%20our%20Intellectual%20Property) This section details risks concerning intellectual property, including challenges in obtaining and enforcing patents, key patent expiration, and potential infringement lawsuits - **The ability to obtain, maintain, enforce, and protect patent protection for technology and product candidates is crucial but expensive, time-consuming, and uncertain, with risks of narrow scope or invalidation**[292](index=292&type=chunk)[293](index=293&type=chunk)[294](index=294&type=chunk) - **Losmapimod composition of matter patents licensed from GSK are expected to expire on February 10, 2023, potentially limiting competitive advantage**[296](index=296&type=chunk)[299](index=299&type=chunk) - **The company may face lawsuits alleging infringement of third-party intellectual property rights, leading to substantial litigation expenses, diversion of resources, and potential monetary damages or injunctions**[312](index=312&type=chunk)[315](index=315&type=chunk)[316](index=316&type=chunk) [Risks Related to Regulatory Approval of our Product Candidates and Other Legal Compliance Matters](index=74&type=section&id=Risks%20Related%20to%20Regulatory%20Approval%20of%20our%20Product%20Candidates%20and%20Other%20Legal%20Compliance%20Matters) This section outlines risks related to regulatory approval, including the expensive and uncertain approval process, limitations of expedited pathways, and critical compliance with healthcare and privacy laws - **The marketing approval process is expensive, time-consuming, and uncertain, with no guarantee of obtaining approvals or avoiding post-marketing restrictions**[335](index=335&type=chunk)[337](index=337&type=chunk)[352](index=352&type=chunk) - **Orphan drug designation, Fast Track, Breakthrough Therapy, and accelerated approval pathways do not guarantee faster development or ultimate approval, and exclusivity may be limited**[339](index=339&type=chunk)[341](index=341&type=chunk)[343](index=343&type=chunk)[345](index=345&type=chunk)[346](index=346&type=chunk) - **Compliance with healthcare laws (anti-kickback, false claims, HIPAA), privacy regulations (GDPR, CCPA), and anti-corruption laws (FCPA, Bribery Act) is critical, with potential for significant fines and penalties for violations**[357](index=357&type=chunk)[361](index=361&type=chunk)[363](index=363&type=chunk)[385](index=385&type=chunk)[388](index=388&type=chunk) [Risks Related to Employee Matters and Managing Growth](index=87&type=section&id=Risks%20Related%20to%20Employee%20Matters%20and%20Managing%20Growth) This section discusses risks related to employee matters and managing growth, including the ability to retain key personnel and the challenges of expanding operations - **Future success depends on the ability to retain key executives and attract, retain, and motivate qualified scientific, clinical, and management personnel amidst intense competition**[393](index=393&type=chunk)[394](index=394&type=chunk) - **Expected significant growth in employees and operations, particularly in drug development and regulatory affairs, may lead to difficulties in managing expansion and increased costs**[395](index=395&type=chunk) [Risks Related to our Common Stock](index=88&type=section&id=Risks%20Related%20to%20our%20Common%20Stock) This section addresses risks related to common stock, including significant influence by principal stockholders, stock price volatility, and potential dilution - **Executive officers, directors, and principal stockholders collectively own approximately 63.4% of capital stock, enabling significant influence over company matters**[396](index=396&type=chunk) - **The price of common stock is volatile and can fluctuate substantially due to factors like clinical trial results, regulatory developments, and market conditions**[403](index=403&type=chunk)[405](index=405&type=chunk) - **Sales of a substantial number of shares in the public market, including those from private placements and shelf registration statements, could significantly depress the market price of common stock**[407](index=407&type=chunk)[409](index=409&type=chunk)[410](index=410&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=92&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) This section confirms no unregistered equity sales during Q3 2020 and details the use of $26.5 million from IPO proceeds for clinical development and general corporate purposes - **No unregistered sales of equity securities occurred during the three months ended September 30, 2020**[422](index=422&type=chunk) - **The company received aggregate net proceeds of $63.9 million from its IPO in July 2019**[424](index=424&type=chunk) - **Approximately $26.5 million of the net IPO proceeds were used as of September 30, 2020, to fund clinical development and general corporate purposes, with no material change in planned use**[425](index=425&type=chunk) [Item 6. Exhibits](index=94&type=section&id=Item%206.%20Exhibits) This section lists the exhibits filed with the Form 10-Q, including key collaboration agreements, officer certifications, and XBRL taxonomy documents - **Includes the Collaboration and License Agreement with MyoKardia, Inc. dated July 20, 2020**[429](index=429&type=chunk) - **Includes the First Amendment to the Right of Reference and License Agreement with GlaxoSmithKline entities dated September 23, 2020**[429](index=429&type=chunk) - **Certifications of the Principal Executive Officer and Principal Financial Officer are filed or furnished**[429](index=429&type=chunk) [Signatures](index=95&type=section&id=Signatures) This section confirms the official signing of the report by Robert J. Gould, Ph.D., CEO, and Bryan Stuart, COO and Principal Financial Officer, on November 10, 2020 - **The report is signed by Robert J. Gould, Ph.D., Chief Executive Officer and President**[434](index=434&type=chunk) - **The report is signed by Bryan Stuart, Chief Operating Officer (Principal Financial Officer)**[434](index=434&type=chunk) - **Date of signing: November 10, 2020**[434](index=434&type=chunk)

Company Overview - Fulcrum is a clinical-stage biopharmaceutical company focused on rebalancing gene expression to treat genetically defined diseases[4] - The company utilizes a systematic approach and its FulcrumSeek platform to identify small molecules that can modulate gene expression[4, 5] Pipeline Highlights - **COVID-19 (Losmapimod):** A Phase 3 trial (LOSVID) was initiated in Q3 2020[7, 8, 9] with topline data expected in Q1 2021[8, 17] The trial involves approximately 400 subjects randomized 1:1 to receive either 15mg of losmapimod or a placebo twice daily for 14 days[17] - **FSHD (Losmapimod):** Phase 2b ReDUX4 trial data is expected, with topline data in Q1 2021 and full data in Q2 2021[7, 8, 47] An interim analysis (IA) of the first 29 randomized subjects was conducted in Q3 2020[29] - **Sickle Cell Disease (FTX-6058) & β-Thalassemia:** An IND filing is planned for Q3 2020, with Phase 1 initiation in SCD expected in Q4 2020[7, 8] FTX-6058 has shown potent activity with less than 1 nM concentration and a clean off-target profile[50] Losmapimod for FSHD - The estimated US FSHD population is between 16,000 and 38,000, while the global population is estimated between 300,000 and 780,000[20] - Interim analysis of the ReDUX4 trial showed a 38-fold reduction in DUX4-driven gene expression in the losmapimod arm compared to a 5.4-fold reduction in the placebo arm in highest expressing muscle biopsies[35, 37] FTX-6058 for Hemoglobinopathies - FTX-6058 has demonstrated an absolute 8-18% increase in fetal hemoglobin (HbF) upon treatment, which could reduce mortality risk and recurring events in SCD patients[52] - The company's data indicates that small increases in HbF (1-5%) have the potential to provide clinical benefits to all SCD patients[52] Research and Discovery Collaborations - Fulcrum has research and discovery collaborations with Acceleron and MyoKardia, with potential milestone payments exceeding $800 million, plus upfront payments and R&D reimbursement[55, 56] - Fulcrum received a $10 million upfront payment from Acceleron and a $12.5 million payment at the close of the transaction with MyoKardia, along with reimbursement for relevant research expenses[55]

Fulcrum | --- | --- | --- | --- | --- | --- | |---------------------------------|-------|-------|-------|-------|-------| | | | | | | | | | | | | | | | | | | | | | | | | | | | | | Fulcrum Q2 2020 Conference Call | | | | | | | August 11, 2020 | | | | | | | | | | | | | | | | | | | | Disclaimer and Notice This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including statements regard ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 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: 001-38978 FULCRUM THERAPEUTICS, INC. (Exact Name of Registrant as Specified in its Charter) Delaware 47-4839948 (State or other jurisd ...

Financial Data and Key Metrics Changes - The company entered Q1 2020 with $81.2 million in cash, cash equivalents, and marketable securities, which is expected to support operations into Q3 2021 [29] - Research and development expenses for Q1 2020 were $14.5 million, a decrease from $34.6 million in Q1 2019, primarily due to a one-time cost in the previous year [30] - General and administrative expenses increased to $5.1 million in Q1 2020 from $2.6 million in Q1 2019, attributed to increased personnel costs and public company operating expenses [31] Business Line Data and Key Metrics Changes - The FSHD program received orphan drug designation from both the U.S. FDA and the European Commission, supporting the advancement of losmapimod [9] - The ReDUX4 trial was amended to extend the treatment period from 24 to 48 weeks, allowing for a more robust data set [16][20] - The sickle-cell program, FTX-6058, is progressing towards IND filing, with plans to submit in the second half of 2020 and initiate a Phase 1 trial by year-end [28] Market Data and Key Metrics Changes - The estimated patient population for FSHD in the U.S. is between 16,000 to 38,000, with no approved drugs currently available [12] - The company continues to advance its proprietary product engine, FulcrumSeek, to identify drug targets in genetically defined diseases [23] Company Strategy and Development Direction - The company aims to discover and develop therapeutics for genetically defined diseases, focusing on addressing root causes [6] - The strategy includes increasing fetal hemoglobin levels to develop therapies for sickle-cell disease and beta thalassemia [26] - The company is committed to maintaining high standards in patient safety and data integrity during clinical trials, especially in light of COVID-19 [14][20] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the ability to adapt to challenges posed by COVID-19, emphasizing the commitment to patient safety and data collection [15][20] - The company anticipates reporting interim analysis data from the ReDUX4 trial in Q3 2020 and expects to report top-line data in Q1 2021 [18][32] Other Important Information - The company has made significant progress in its clinical candidate FTX-6058, with robust preclinical data supporting its efficacy [28] - The management highlighted the importance of flexibility in trial protocols to accommodate potential disruptions due to the pandemic [43][54] Q&A Session Summary Question: Concerns about power loss with interim analysis - Management believes the sample size of 80 subjects provides appropriate power for the trial, and the interim analysis will offer initial insights for Phase III planning [35][36] Question: Recruitment status for subjects - Recruitment has been completed, and management is confident they have all necessary subjects [37] Question: Biopsy completion amid pandemic concerns - Management has built flexibility into the protocol to ensure data collection at either 16 or 36 weeks, mitigating risks associated with potential patient dropouts [39] Question: Doubling duration of ReDUX protocol - The extension was made to provide flexibility for data collection over a longer period, accommodating the ongoing pandemic [42][43] Question: Insights from the open-label trial - The open-label trial has been impacted more significantly due to being a single-site study, but valuable learnings have been gained [46] Question: Powering assumptions and study length - Management clarified that the amendment does not impact power assumptions, and patient compliance has been high [50][52] Question: Integrity of biopsy readings at different time points - Studies indicate stability in the DUX4 gene signature, supporting the integrity of readings at both 16 and 36 weeks [53][54]

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2020 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 (I.R.S. Employer Identification No.) (Address of principal executive offices) (Zip Code) Registrant's telephone number, including area code: (617) 651-8851 Securities registered pursuant to S ...

```markdown [PART I](index=5&type=section&id=PART%20I) [Business](index=5&type=section&id=Item%201.%20Business) Fulcrum Therapeutics is a clinical-stage biopharmaceutical company utilizing a proprietary product engine to develop small molecule therapies for genetically defined rare diseases [Overview and Pipeline](index=5&type=section&id=Overview%20and%20Pipeline) Fulcrum is a clinical-stage biopharmaceutical company focused on genetically defined rare diseases, with its lead product losmapimod for FSHD having completed Phase **2b** trial enrollment and FTX-6058 for hemoglobinopathies expected to have an IND submitted in the second half of **2020** - The company's lead product candidate, losmapimod for facioscapulohumeral muscular dystrophy (FSHD), initiated a **Phase 2b** clinical trial in **August 2019**, which completed enrollment in **February 2020**[16](index=16&type=chunk) - The second product candidate, FTX-6058 for hemoglobinopathies (sickle cell disease and ß-thalassemia), is in IND-enabling studies with a planned IND submission in the second half of **2020**[16](index=16&type=chunk)[24](index=24&type=chunk) Fulcrum Therapeutics Pipeline Status | Program (Product Candidate) | Indication | Discovery | Preclinical | Phase 1 | Phase 2 | Phase 3 | Status | | :--- | :--- | :--- | :--- | :--- | :--- | :--- | :--- | | **losmapimod** | FSHD | ✔ | ✔ | ✔ | **✔** | | Completed Ph 2 enrollment | | **FTX-6058** | Sickle Cell Disease | ✔ | **✔** | | | | Submit IND in **2H 2020** | | **FTX-6058** | ß-Thalassemia | ✔ | **✔** | | | | Submit IND in **2H 2020** | | **Discovery Screening** | Duchenne Muscular Dystrophy | **✔** | | | | | Target ID / Validation | | **Discovery Screening** | Friedreich Ataxia | **✔** | | | | | Target ID / Validation | | **Discovery Screening** | Myotonic Dystrophy 1 | **✔** | | | | | Target ID / Validation | | **Discovery Screening** | α-Synucleinopathies | **✔** | | | | | Target ID / Validation | [Our Strategy and Product Engine](index=7&type=section&id=Our%20Strategy%20and%20Product%20Engine) The company's strategy focuses on rapidly developing lead candidates losmapimod and FTX-6058, while continuously expanding its portfolio using a proprietary product engine that integrates patient-derived cell models, libraries, and a database to identify drug targets modulating gene expression - Key strategic components include rapidly developing losmapimod for FSHD, advancing FTX-6058 into clinical trials, and applying the proprietary product engine to grow the portfolio of candidates for genetically defined diseases[30](index=30&type=chunk) - The product engine uses patient-derived tissue-relevant cell models, an annotated small molecule library, and customized CRISPR libraries to identify drug targets[39](index=39&type=chunk)[42](index=42&type=chunk) - FulcrumSeek, a proprietary database, is used with computational biology to generate drug target and biomarker hypotheses by analyzing the effects of perturbagens on human-derived cell systems[48](index=48&type=chunk) - The company focuses on a small molecule approach, citing advantages in biodistribution, tolerability, manufacturing, and patient access compared to other modalities like ASOs or gene therapies[55](index=55&type=chunk) [Our Product Candidate for Facioscapulohumeral Muscular Dystrophy (FSHD)](index=14&type=section&id=Our%20Product%20Candidate%20for%20Facioscapulohumeral%20Muscular%20Dystrophy%20(FSHD)) Fulcrum is developing losmapimod for FSHD, a rare muscular dystrophy caused by aberrant DUX4 gene expression, with a **Phase 2b** trial initiated in **August 2019** and orphan drug designation granted in **January 2020** - FSHD is a rare, progressive muscle wasting disorder with an estimated U.S. patient population of **16,000** to **38,000**, caused by the mis-expression of the DUX4 gene, with no approved therapies[18](index=18&type=chunk)[57](index=57&type=chunk) - Losmapimod, a p38α/ß inhibitor, was identified through Fulcrum's screening process to reduce DUX4 expression and was in-licensed from GSK, which had previously tested it in nearly **3,500** subjects for other indications, providing a substantial safety database[19](index=19&type=chunk)[61](index=61&type=chunk)[62](index=62&type=chunk) - A randomized, double-blind, placebo-controlled **Phase 2b** trial (ReDUX4) was initiated in **August 2019** in **80** patients to evaluate if losmapimod reduces DUX4-driven gene expression in muscle biopsies over a **24**-week period, with enrollment completed in **February 2020**[77](index=77&type=chunk)[113](index=113&type=chunk) - A **Phase 1** trial in healthy volunteers and FSHD patients showed losmapimod was well-tolerated and achieved dose-dependent concentrations in skeletal muscle, supporting the **15** mg twice-daily dose for **Phase 2** trials[111](index=111&type=chunk) [Our Product Candidate for Hemoglobinopathies](index=26&type=section&id=Our%20Product%20Candidate%20for%20Hemoglobinopathies) Fulcrum is developing FTX-6058, a novel small molecule designed to upregulate fetal hemoglobin (HbF) for treating sickle cell disease (SCD) and ß-thalassemia, with an IND submission planned for the second half of **2020** and a **Phase 1** trial in late **2020** - SCD affects approximately **100,000** individuals in the U.S. and is caused by a mutation in the HBB gene, leading to abnormal hemoglobin, where increasing fetal hemoglobin (HbF) is a known compensatory mechanism[127](index=127&type=chunk)[131](index=131&type=chunk)[133](index=133&type=chunk) - FTX-6058 was designed to inhibit a novel drug target identified by Fulcrum's product engine, leading to the elevation of HbF[142](index=142&type=chunk)[143](index=143&type=chunk) - In preclinical studies, FTX-6058 increased HbF levels to approximately **30%** of total hemoglobin in primary human CD34+ cells and showed a greater effect than hydroxyurea[149](index=149&type=chunk) - The company is conducting IND-enabling studies and plans to submit an IND for FTX-6058 in the second half of **2020**, with a **Phase 1** trial in healthy volunteers and SCD patients planned for late **2020**[157](index=157&type=chunk) [Discovery Screening Programs and Collaborations](index=32&type=section&id=Discovery%20Screening%20Programs%20and%20Collaborations) In **2019**, Fulcrum completed four new drug target identification screens and maintains key collaborations with GSK for losmapimod and Acceleron for pulmonary disease, involving potential milestone and royalty payments - The company completed four new drug target identification screens in **2019** for DMD, FA, DM1, and α-synucleinopathies, with identified targets currently under evaluation[160](index=160&type=chunk) - Under the GSK agreement for losmapimod, Fulcrum may be obligated to make milestone payments up to **$37.5 million** for clinical/regulatory events and up to **$60.0 million** for sales milestones, in addition to royalties[172](index=172&type=chunk) - In **December 2019**, Fulcrum entered a collaboration with Acceleron to identify targets for a pulmonary disease, receiving a **$10.0 million** upfront payment, and is eligible for up to **$438.5 million** in various milestone payments plus tiered royalties[176](index=176&type=chunk)[181](index=181&type=chunk) [Intellectual Property, Manufacturing, and Competition](index=35&type=section&id=Intellectual%20Property,%20Manufacturing,%20and%20Competition) Fulcrum's intellectual property strategy combines owned and in-licensed patents, with the losmapimod method-of-use patent expiring in **2038** and reliance on CMOs for clinical supply, facing competition in SCD and ß-thalassemia but none in FSHD - The losmapimod patent portfolio includes in-licensed composition of matter patents from GSK expiring in **2023** and a Fulcrum-owned method-of-use patent for FSHD expiring in **2038**[189](index=189&type=chunk)[190](index=190&type=chunk) - The intellectual property for FTX-6058 includes one owned PCT application; any resulting patents would have a projected expiration in **2039**[191](index=191&type=chunk) - The company does not own manufacturing facilities and relies on third parties, including obtaining losmapimod tablets and API from GSK for current trials and engaging CMOs for future supply[196](index=196&type=chunk) - There are no approved therapies for FSHD, while for SCD and ß-thalassemia, Fulcrum faces competition from existing treatments like hydroxyurea and luspatercept, as well as from companies developing novel small molecules, gene therapies, and gene editing approaches[203](index=203&type=chunk)[205](index=205&type=chunk)[208](index=208&type=chunk) [Government Regulation](index=39&type=section&id=Government%20Regulation) The company's operations are subject to extensive regulation by the FDA and comparable authorities, covering all stages of product development from preclinical studies to post-approval marketing, with potential for expedited pathways and impact from healthcare laws and reforms - Drug development in the U.S. requires completing preclinical testing, submitting an IND to the FDA, conducting three phases of human clinical trials to establish safety and efficacy, and obtaining NDA approval[214](index=214&type=chunk)[229](index=229&type=chunk) - The FDA offers expedited programs such as Fast Track, Breakthrough Therapy, and Accelerated Approval for drugs treating serious conditions with unmet medical needs, which can potentially speed up development and review[245](index=245&type=chunk)[251](index=251&type=chunk) - The company is subject to healthcare fraud and abuse laws, including the federal Anti-Kickback Statute and the False Claims Act, as well as transparency laws like the Physician Payments Sunshine Act[281](index=281&type=chunk)[283](index=283&type=chunk) - Healthcare reform, including the ACA and subsequent legislative actions, impacts drug pricing, coverage, and reimbursement, creating uncertainty and potential downward pressure on prices for any future approved products[288](index=288&type=chunk)[290](index=290&type=chunk) [Risk Factors](index=59&type=page&id=Item%201A.%20Risk%20Factors) The company faces substantial risks, including a history of significant losses and the need for additional capital to fund operations - The company has a history of significant operating losses (**$82.7 million** in **2019**) and expects to incur losses for the next several years, with profitability dependent on successful product development and commercialization[325](index=325&type=chunk) - Substantial additional funding will be required to continue clinical trials and R&D, and failure to raise capital could force delays or elimination of programs[328](index=328&type=chunk) - Clinical drug development is a lengthy, expensive, and uncertain process, where early trial results may not predict future success, and product candidates may fail at any stage[347](index=347&type=chunk) - The company relies on third parties for clinical trials and manufacturing, which introduces risks related to performance, quality, and supply chain continuity[395](index=395&type=chunk)[399](index=399&type=chunk) - Intellectual property risks include the potential inability to obtain and maintain patent protection, the expiration of key patents (losmapimod composition of matter patent in **2023**), and the possibility of infringement lawsuits[416](index=416&type=chunk)[419](index=419&type=chunk)[423](index=423&type=chunk) [Properties](index=103&type=section&id=Item%202.%20Properties) The company's main facility is approximately **28,731** square feet of leased office and laboratory space located in Cambridge, Massachusetts, with the current lease set to expire in **June 2028** - The company's principal facilities consist of approximately **28,731** square feet of leased office and laboratory space in Cambridge, Massachusetts[550](index=550&type=chunk) - The lease for the Cambridge facility expires in **June 2028**[550](index=550&type=chunk) [Legal Proceedings](index=103&type=section&id=Item%203.%20Legal%20Proceedings) As of the reporting date, Fulcrum Therapeutics, Inc. is not a party to any material legal proceedings - The company is not currently a party to any material legal proceedings[551](index=551&type=chunk) [PART II](index=104&type=section&id=PART%20II) [Market for Registrant's Common Equity, Related Stockholder Matters and Issuer Purchases of Equity Securities](index=104&type=section&id=Item%205.%20Market%20for%20Registrant's%20Common%20Equity,%20Related%20Stockholder%20Matters%20and%20Issuer%20Purchases%20of%20Equity%20Securities) Fulcrum's common stock began trading on the Nasdaq Global Market under the symbol "FULC" on **July 18, 2019**, with **74** holders of record as of **February 28, 2020**, and the company has never paid cash dividends - The company's common stock began trading on the Nasdaq Global Market under the symbol "FULC" on **July 18, 2019**[554](index=554&type=chunk) - The company completed its IPO on **July 22, 2019**, selling **4,500,000** shares at **$16.00** per share, resulting in net proceeds of **$63.9 million**[563](index=563&type=chunk)[564](index=564&type=chunk) - As of **December 31, 2019**, none of the net proceeds from the IPO had been used to fund operations[565](index=565&type=chunk) - The company has never declared or paid cash dividends and does not intend to in the foreseeable future[556](index=556&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=106&type=section&id=Item%207.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) The company reported a net loss of **$82.7 million** for **2019**, a significant increase from the **$32.6 million** loss in **2018**, primarily due to a **$45.9 million** rise in R&D expenses, with **$96.7 million** in cash and cash equivalents expected to fund operations into the third quarter of **2021** [Results of Operations](index=110&type=section&id=Results%20of%20Operations) For the year ended **December 31, 2019**, the company's net loss increased to **$82.7 million** from **$32.6 million** in **2018**, primarily due to a surge in research and development expenses to **$71.1 million** from **$25.2 million**, driven by a **$25.6 million** IPR&D expense and **$9.1 million** in higher clinical trial costs Comparison of Results of Operations (**2019** vs. **2018**) | (in thousands) | **2019** | **2018** | Change ($) | | :--- | :--- | :--- | :--- | | Research and development | **$71,072** | **$25,184** | **$45,888** | | General and administrative | **$13,145** | **$8,314** | **$4,831** | | **Total operating expenses** | **$84,217** | **$33,498** | **$50,719** | | Loss from operations | (**$84,217**) | (**$33,498**) | (**$50,719**) | | Other income, net | **$1,540** | **$910** | **$630** | | **Net loss** | (**$82,677**) | (**$32,588**) | (**$50,089**) | - Research and development expenses increased by **$45.9 million**, primarily due to a **$25.6 million** in-process research and development (IPR&D) expense related to the GSK license agreement and a **$9.1 million** increase in external clinical activity costs for losmapimod[597](index=597&type=chunk) - General and administrative expenses increased by **$4.8 million**, mainly from a **$2.5 million** increase in consulting and professional fees and a **$2.3 million** increase in personnel-related costs due to headcount growth[597](index=597&type=chunk) [Liquidity and Capital Resources](index=112&type=section&id=Liquidity%20and%20Capital%20Resources) As of **December 31, 2019**, Fulcrum had **$96.7 million** in cash and cash equivalents, primarily funded by IPO proceeds and collaborations, with existing cash expected to fund operations into the third quarter of **2021** - As of **December 31, 2019**, the company had cash and cash equivalents of **$96.7 million**[600](index=600&type=chunk) Cash Flow Summary (**2019** vs. **2018**) | (in thousands) | **2019** | **2018** | | :--- | :--- | :--- | | Net cash used in operating activities | (**$39,483**) | (**$22,562**) | | Net cash used in investing activities | (**$944**) | (**$8,981**) | | Net cash provided by financing activities | **$64,343** | **$105,025** | - The company estimates that its existing cash and cash equivalents will be sufficient to fund its operating expenses and capital expenditure requirements into the third quarter of **2021**[607](index=607&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=118&type=section&id=Item%207A.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company's primary market risk is interest rate sensitivity on its cash and cash equivalents, with foreign currency exchange rate risk from international vendors, but no material effect from inflation or hedging instruments currently in use - The primary market risk is interest rate sensitivity on cash and cash equivalents of **$96.7 million**, but a **10%** change in rates is not expected to be material[642](index=642&type=chunk) - The company is exposed to foreign currency exchange rate risk due to contracts with vendors outside the U.S., but does not currently hedge this risk[643](index=643&type=chunk) [Financial Statements and Supplementary Data](index=118&type=section&id=Item%208.%20Financial%20Statements%20and%20Supplementary%20Data) This section contains the company's audited consolidated financial statements for the fiscal years ended **December 31, 2019** and **2018**, including the Report of the Independent Registered Public Accounting Firm, Consolidated Balance Sheets, Statements of Operations and Comprehensive Loss, Statements of Convertible Preferred Stock and Stockholders' Equity (Deficit), Statements of Cash Flows, and accompanying Notes Consolidated Balance Sheet Highlights (As of **Dec 31, 2019**) | (In thousands) | Amount | | :--- | :--- | | **Assets** | | | Cash and cash equivalents | **$96,713** | | Total current assets | **$100,083** | | **Total assets** | **$110,439** | | **Liabilities & Equity** | | | Total current liabilities | **$12,140** | | **Total liabilities** | **$23,286** | | Total stockholders' equity | **$87,153** | Consolidated Statement of Operations Highlights (Year Ended **Dec 31, 2019**) | (In thousands) | Amount | | :--- | :--- | | Research and development | **$71,072** | | General and administrative | **$13,145** | | **Total operating expenses** | **$84,217** | | Loss from operations | (**$84,217**) | | **Net loss** | (**$82,677**) | [Controls and Procedures](index=119&type=section&id=Item%209A.%20Controls%20and%20Procedures) Management concluded that the company's disclosure controls and procedures were effective as of **December 31, 2019**, with no material changes reported during the fourth quarter of **2019** - Management concluded that the company's disclosure controls and procedures were effective at a reasonable assurance level as of **December 31, 2019**[648](index=648&type=chunk) - As a newly public company, the annual report does not include a management assessment or auditor attestation on internal control over financial reporting, per SEC transition rules[649](index=649&type=chunk) [PART III](index=120&type=section&id=PART%20III) [Directors, Executive Officers and Corporate Governance](index=120&type=section&id=Item%2010.%20Directors,%20Executive%20Officers%20and%20Corporate%20Governance) Information regarding the company's directors, executive officers, and corporate governance practices is incorporated by reference from the upcoming Definitive Proxy Statement for its **2020** Annual Meeting of Stockholders - Information required for this item will be included in the Definitive Proxy Statement for the **2020** Annual Meeting of Stockholders and is incorporated by reference[654](index=654&type=chunk) [Executive Compensation](index=120&type=section&id=Item%2011.%20Executive%20Compensation) Details concerning executive compensation are incorporated by reference from the company's Definitive Proxy Statement to be filed for its **2020** Annual Meeting of Stockholders - Information required for this item will be included in the Definitive Proxy Statement for the **2020** Annual Meeting of Stockholders and is incorporated by reference[656](index=656&type=chunk) [Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters](index=120&type=section&id=Item%2012.%20Security%20Ownership%20of%20Certain%20Beneficial%20Owners%20and%20Management%20and%20Related%20Stockholder%20Matters) Information regarding security ownership of certain beneficial owners, management, and related stockholder matters is incorporated by reference from the company's Definitive Proxy Statement for its **2020** Annual Meeting of Stockholders - Information required for this item will be included in the Definitive Proxy Statement for the **2020** Annual Meeting of Stockholders and is incorporated by reference[657](index=657&type=chunk) [Certain Relationships and Related Transactions, and Director Independence](index=120&type=section&id=Item%2013.%20Certain%20Relationships%20and%20Related%20Transactions,%20and%20Director%20Independence) Information about certain relationships, related-party transactions, and director independence is incorporated by reference from the company's Definitive Proxy Statement for its **2020** Annual Meeting of Stockholders - Information required for this item will be included in the Definitive Proxy Statement for the **2020** Annual Meeting of Stockholders and is incorporated by reference[658](index=658&type=chunk) [Principal Accounting Fees and Services](index=120&type=section&id=Item%2014.%20Principal%20Accounting%20Fees%20and%20Services) Details regarding principal accounting fees and services are incorporated by reference from the company's Definitive Proxy Statement for its **2020** Annual Meeting of Stockholders - Information required for this item will be included in the Definitive Proxy Statement for the **2020** Annual Meeting of Stockholders and is incorporated by reference[659](index=659&type=chunk) [PART IV](index=121&type=section&id=PART%20IV) [Exhibits and Financial Statement Schedules](index=121&type=section&id=Item%2015.%20Exhibits%20and%20Financial%20Statement%20Schedules) This section lists the consolidated financial statements and exhibits filed as part of the Annual Report on Form **10**-K, with financial statement schedules omitted as not applicable - This section includes the consolidated financial statements and lists all exhibits filed with the Form **10**-K, which are incorporated by reference[662](index=662&type=chunk)[665](index=665&type=chunk) ```

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2019 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: 001-38978 FULCRUM THERAPEUTICS, INC. (Exact Name of Registrant as Specified in its Charter) Delaware 47-4839948 (State or other j ...