Exhibit 99.1 Fulcrum Therapeutics Announces Recent Business Highlights and Financial Results for Fourth Quarter and Full Year 2023 ― On track to report topline data for Phase 3 REACH trial of losmapimod in facioscapulohumeral muscular dystrophy (FSHD) in the fourth quarter of 2024 ― ― Restart of the Phase 1b PIONEER trial of pociredir in sickle cell disease (SCD) underway ― ― Conference call and webcast scheduled for 8:00 a.m. ET today ― CAMBRIDGE, Mass., – February 27, 2024 – Fulcrum Therapeutics, Inc. ® ( ...

[Preliminary Information](index=2&type=section&id=Preliminary%20Information) [Cautionary Note Regarding Forward-Looking Statements](index=2&type=section&id=CAUTIONARY%20NOTE%20REGARDING%20FORWARD-LOOKING%20STATEMENTS) This section cautions against undue reliance on forward-looking statements regarding operations, financial performance, and product development due to inherent risks - Forward-looking statements cover ongoing clinical trials (**losmapimod**, **pociredir**), impact of organizational streamlining, initiation/timing of **R&D programs**, **commercialization plans**, **funding expectations**, potential advantages of **product candidates**, **market acceptance**, **intellectual property**, and **collaborations**[6](index=6&type=chunk)[7](index=7&type=chunk) - Readers are cautioned not to place undue reliance on forward-looking statements, as **actual results may differ materially** due to various **risks and uncertainties**, particularly those detailed in the 'Risk Factors' and 'Management's Discussion and Analysis of Results of Operations' sections[8](index=8&type=chunk)[9](index=9&type=chunk) [Summary Risk Factors](index=3&type=section&id=SUMMARY%20RISK%20FACTORS) This section summarizes principal risks including accumulated losses, funding needs, early development stage, lengthy drug development, adverse events, competition, and third-party reliance Key Financial Risks | Metric | Amount (as of Sep 30, 2023) | Previous (as of Dec 31, 2022) | | :----- | :-------------------------- | :----------------------------- | | Net Loss (9 months) | $72.6 million | $109.9 million (FY2022) | | Accumulated Deficit | $484.9 million | $412.3 million (FY2022) | - The company expects to incur **losses** for several years and will require **substantial additional funding** to support product development programs (**losmapimod**, **pociredir**) and **commercialization efforts**[11](index=11&type=chunk) - Key operational risks include the **early stage of development** with only **two clinical candidates**, the **lengthy and uncertain nature** of clinical drug development (e.g., recent **clinical hold on pociredir**), potential for **serious adverse events**, and **reliance on contract manufacturing organizations (CMOs)** and **third parties for clinical trials**[11](index=11&type=chunk)[12](index=12&type=chunk) - Other risks include **substantial competition**, challenges in **intellectual property protection**, **adverse developments in the financial services industry**, and potential impacts from **pandemics or geopolitical events**[11](index=11&type=chunk)[12](index=12&type=chunk) [PART I. FINANCIAL INFORMATION](index=5&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) [Item 1. Financial Statements](index=5&type=section&id=Item%201.%20Financial%20Statements) This section presents unaudited consolidated financial statements for Q3 2023 and 2022, covering balance sheets, operations, equity, cash flows, and detailed accounting notes [Consolidated Balance Sheets](index=6&type=section&id=Consolidated%20Balance%20Sheets%20as%20of%20September%2030%2C%202023%20and%20December%2031%2C%202022) Consolidated balance sheets show total assets increased to **$278.9 million** (Sep 30, 2023) from **$226.7 million** (Dec 31, 2022), driven by marketable securities and paid-in capital Consolidated Balance Sheet Highlights (in thousands) | Item | Sep 30, 2023 | Dec 31, 2022 | Change ($) | Change (%) | | :-------------------------------- | :----------- | :----------- | :--------- | :--------- | | Total Assets | $278,879 | $226,685 | $52,194 | 23.0% | | Cash and cash equivalents | $30,080 | $35,098 | $(5,018) | -14.3% | | Marketable securities | $227,011 | $167,823 | $59,188 | 35.3% | | Total Liabilities | $23,417 | $27,743 | $(4,326) | -15.6% | | Total Stockholders' Equity | $255,462 | $198,942 | $56,520 | 28.4% | | Accumulated Deficit | $(484,917) | $(412,338) | $(72,579) | 17.6% | - The increase in **total assets** is primarily due to a significant rise in **marketable securities**, while **cash and cash equivalents** saw a decrease[15](index=15&type=chunk) - **Stockholders' equity increased substantially**, mainly driven by **additional paid-in capital** from **common stock issuances**, despite an increase in **accumulated deficit**[15](index=15&type=chunk) [Consolidated Statements of Operations and Comprehensive Loss](index=7&type=section&id=Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss%20for%20the%20three%20and%20nine%20months%20ended%20September%2030%2C%202023%20and%202022) Consolidated statements show a net loss of **$24.0 million** (Q3 2023) and **$72.6 million** (YTD Sep 2023), with nine-month loss decreasing despite lower collaboration revenue and R&D expenses Consolidated Statements of Operations Highlights (in thousands) | Item | 3 Months Ended Sep 30, 2023 | 3 Months Ended Sep 30, 2022 | 9 Months Ended Sep 30, 2023 | 9 Months Ended Sep 30, 2022 | | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | | Collaboration revenue | $759 | $1,183 | $1,934 | $5,657 | | Research and development | $18,238 | $15,366 | $52,802 | $58,216 | | General and administrative | $9,961 | $9,707 | $31,804 | $31,564 | | Total operating expenses | $28,199 | $25,538 | $84,606 | $90,245 | | Loss from operations | $(27,440) | $(24,355) | $(82,672) | $(84,588) | | Other income, net | $3,423 | $617 | $10,093 | $852 | | Net loss | $(24,017) | $(23,738) | $(72,579) | $(83,736) | | Net loss per share (basic & diluted) | $(0.39) | $(0.51) | $(1.19) | $(1.97) | - Collaboration revenue decreased by **35.8%** for the three months and **65.8%** for the nine months ended September 30, 2023, primarily due to the **termination of the Acceleron collaboration agreement**[17](index=17&type=chunk) - Research and development expenses increased by **18.7%** for the three months but decreased by **9.3%** for the nine months ended September 30, 2023, reflecting **shifts in clinical trial activities**[17](index=17&type=chunk) - **Other income, net, significantly increased** for both periods, driven by **higher returns** on **cash, cash equivalents, and marketable securities**[17](index=17&type=chunk) [Consolidated Statements of Stockholders' Equity](index=8&type=section&id=Consolidated%20Statements%20of%20Stockholders%27%20Equity%20for%20the%20three%20and%20nine%20months%20ended%20September%2030%2C%202023%20and%202022) Stockholders' equity increased from **$198.9 million** (Dec 31, 2022) to **$255.5 million** (Sep 30, 2023), driven by common stock offerings and stock-based compensation, despite accumulated losses Stockholders' Equity Changes (in thousands) | Item | Dec 31, 2022 | Sep 30, 2023 | Change ($) | | :-------------------------- | :----------- | :----------- | :--------- | | Common Stock (Amount) | $52 | $62 | $10 | | Additional Paid-In Capital | $612,025 | $741,203 | $129,178 | | Accumulated Deficit | $(412,338) | $(484,917) | $(72,579) | | Total Stockholders' Equity | $198,942 | $255,462 | $56,520 | - The increase in **additional paid-in capital** is largely attributable to **$117.3 million** in net proceeds from a **public offering of common stock** in January 2023[20](index=20&type=chunk) - **Stock-based compensation expense** contributed **$11.4 million** to **additional paid-in capital** for the nine months ended September 30, 2023[20](index=20&type=chunk) [Consolidated Statements of Cash Flows](index=9&type=section&id=Consolidated%20Statements%20of%20Cash%20Flows%20for%20the%20nine%20months%20ended%20September%2030%2C%202023%20and%202022) Cash flows show a net decrease of **$5.0 million** (YTD Sep 2023), a shift from a **$55.5 million** increase (prior year), primarily due to increased net cash used in investing activities Consolidated Cash Flow Highlights (in thousands) | Activity | 9 Months Ended Sep 30, 2023 | 9 Months Ended Sep 30, 2022 | | :------------------------------------------ | :-------------------------- | :-------------------------- | | Net cash used in operating activities | $(67,807) | $(77,354) | | Net cash (used in) provided by investing activities | $(55,046) | $48,775 | | Net cash provided by financing activities | $117,835 | $84,105 | | Net (decrease) increase in cash, cash equivalents, and restricted cash | $(5,018) | $55,526 | | Cash, cash equivalents, and restricted cash, end of period | $31,172 | $92,030 | - **Net cash used in operating activities decreased by $9.6 million**, mainly due to a **lower net loss** and **increased interest income**[23](index=23&type=chunk) - **Net cash used in investing activities increased by $103.8 million**, primarily driven by **net purchases of marketable securities** in 2023 compared to net maturities in 2022[23](index=23&type=chunk) - **Net cash provided by financing activities increased by $33.7 million**, largely from **$117.3 million** in net proceeds from a January 2023 **public offering of common stock**[23](index=23&type=chunk) [Notes to Consolidated Financial Statements](index=10&type=section&id=Notes%20to%20Consolidated%20Financial%20Statements) These notes detail business nature, accounting policies, fair value, cash, marketable securities, property, equity, stock compensation, collaborations, leases, commitments, contingencies, and net loss per share [1. Nature of the Business and Basis of Presentation](index=10&type=section&id=1.%20Nature%20of%20the%20Business%20and%20Basis%20of%20Presentation) Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on rare diseases, with **$484.9 million** accumulated deficit and ongoing need for additional funding - Fulcrum Therapeutics, Inc. was incorporated in Delaware on August 18, 2015, and focuses on developing **small molecules** for **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 $484.9 million** as of September 30, 2023[31](index=31&type=chunk) - In January 2023, the company completed a **public offering**, issuing **9,615,384 shares** of common stock at **$13.00 per share**, generating net proceeds of **$117.3 million**[29](index=29&type=chunk) - Management expects existing cash, cash equivalents, and marketable securities to fund operations for at least **12 months** from the financial statement issuance date, but acknowledges the need for **future financing** through **equity, debt, or collaborations**[32](index=32&type=chunk) [2. Summary of Significant Accounting Policies](index=11&type=section&id=2.%20Summary%20of%20Significant%20Accounting%20Policies) This note outlines significant accounting policies, including consolidation, estimates, and off-balance sheet risk, and details the adoption of new accounting pronouncements with no material impact - The **consolidated financial statements** include Fulcrum Therapeutics, Inc. and its **wholly-owned subsidiary**, Fulcrum Therapeutics Securities Corp., with all **intercompany transactions eliminated**[33](index=33&type=chunk) - Key estimates in financial statement preparation include **revenue recognition**, **accrued expenses**, **stock-based compensation**, and **income taxes**[35](index=35&type=chunk) - The company adopted **ASU No. 2016-13 (Credit Losses)** and **ASU No. 2019-12 (Income Taxes)** effective **January 1, 2023**, with **no material impact** on its financial position or results of operations[37](index=37&type=chunk)[38](index=38&type=chunk) - The company has **no significant off-balance sheet risk** and manages credit risk primarily through **diversified investments** in **cash, cash equivalents, and marketable securities** held at large financial institutions[36](index=36&type=chunk) [3. Fair Value Measurements](index=12&type=section&id=3.%20Fair%20Value%20Measurements) This note details fair value measurements of financial assets, primarily cash equivalents and marketable securities, totaling **$257.1 million** (Sep 30, 2023), mostly classified as Level 2 Fair Value Measurements at September 30, 2023 (in thousands) | Item | Total | Level 1 | Level 2 | Level 3 | | :---------------------- | :------ | :------ | :------ | :------ | | Cash equivalents: | | | | | | Money market funds | $17,336 | $17,336 | — | — | | U.S. Treasury securities | $1,991 | — | $1,991 | — | | Commercial paper | $10,753 | — | $10,753 | — | | Marketable securities: | | | | | | U.S. Treasury securities | $19,516 | — | $19,516 | — | | Government agency securities | $69,690 | — | $69,690 | — | | Commercial paper | $32,552 | — | $32,552 | — | | Corporate bonds | $105,253 | — | $105,253 | — | | **Total** | **$257,091** | **$17,336** | **$239,755** | **—** | - The majority of marketable securities and a portion of cash equivalents are classified as **Level 2**, indicating fair value derived from **observable inputs** other than quoted prices[39](index=39&type=chunk) - There were **no transfers** between fair value levels during the three and nine months ended September 30, 2023[39](index=39&type=chunk) [4. Cash Equivalents and Marketable Securities](index=13&type=section&id=4.%20Cash%20Equivalents%20and%20Marketable%20Securities) This note details cash equivalents and marketable securities, with a total fair value of **$257.1 million** (Sep 30, 2023) and a net accumulated other comprehensive loss of **$886 thousand** from unrealized losses Cash Equivalents and Marketable Securities (in thousands) | Item | Amortized Cost (Sep 30, 2023) | Fair Value (Sep 30, 2023) | Gross Unrealized Losses (Sep 30, 2023) | | :-------------------------- | :---------------------------- | :------------------------ | :------------------------------------- | | Total cash equivalents | $30,084 | $30,080 | $(4) | | Total marketable securities | $227,893 | $227,011 | $(882) | | **Total** | **$257,977** | **$257,091** | **$(886)** | - As of September 30, 2023, the company held **83 debt securities** in an unrealized loss position for less than **12 months** (**$216.6 million** fair value) and **5 debt securities** for greater than **12 months** (**$10.4 million** fair value)[40](index=40&type=chunk) - The company has the **intent and ability to hold** its debt securities until recovery and did not record any **credit-related impairment charges**[41](index=41&type=chunk) [5. Property and Equipment, Net](index=14&type=section&id=5.%20Property%20and%20Equipment%2C%20Net) Property and equipment, net, decreased to **$5.6 million** (Sep 30, 2023) from **$6.9 million** (Dec 31, 2022), primarily due to accumulated depreciation Property and Equipment, Net (in thousands) | Item | Sep 30, 2023 | Dec 31, 2022 | | :-------------------------- | :----------- | :----------- | | Total property and equipment | $17,973 | $17,568 | | Less: accumulated depreciation | $(12,327) | $(10,662) | | **Property and equipment, net** | **$5,646** | **$6,906** | - Depreciation expense was **$0.5 million** for the three months and **$1.7 million** for the nine months ended September 30, 2023[42](index=42&type=chunk) [6. Additional Balance Sheet Detail](index=14&type=section&id=6.%20Additional%20Balance%20Sheet%20Detail) This note details prepaid expenses and other current assets, increasing to **$4.8 million**, and accrued expenses and other current liabilities, decreasing to **$7.8 million** (Sep 30, 2023) Prepaid Expenses and Other Current Assets (in thousands) | Item | Sep 30, 2023 | Dec 31, 2022 | | :-------------------------------- | :----------- | :----------- | | Prepaid expenses | $3,466 | $3,425 | | Prepaid sign-on bonuses subject to vesting provisions | $254 | $92 | | Interest income receivable | $1,099 | $852 | | **Total** | **$4,819** | **$4,369** | Accrued Expenses and Other Current Liabilities (in thousands) | Item | Sep 30, 2023 | Dec 31, 2022 | | :-------------------------- | :----------- | :----------- | | External research and development | $3,107 | $4,700 | | Payroll and benefits | $4,023 | $4,211 | | Professional services | $422 | $535 | | Other | $265 | $105 | | **Total** | **$7,817** | **$9,551** | [7. Preferred Stock](index=14&type=section&id=7.%20Preferred%20Stock) The company authorized **5,000,000** preferred shares, but none were issued or outstanding as of September 30, 2023, and no dividends have been declared since inception - **5,000,000 shares** of preferred stock were authorized, but **none were issued or outstanding** as of September 30, 2023, and December 31, 2022[44](index=44&type=chunk) - **No dividends** have been declared on preferred stock since inception[45](index=45&type=chunk) [8. Common Stock](index=14&type=section&id=8.%20Common%20Stock) This note details common stock, with **200,000,000** shares authorized and **61,822,554** outstanding (Sep 30, 2023), plus shares reserved for employee benefit plans - **200,000,000 shares** of common stock, **$0.001 par value**, were authorized[46](index=46&type=chunk) Common Shares Issued and Outstanding | Date | Shares Issued and Outstanding | | :---------------- | :---------------------------- | | Sep 30, 2023 | 61,822,554 | | Dec 31, 2022 | 52,099,211 | - Common stockholders are not entitled to receive dividends unless declared by the board of directors, and **no dividends** have been declared or paid since inception[47](index=47&type=chunk) Shares Reserved for Future Issuance | Item | Sep 30, 2023 | Dec 31, 2022 | | :------------------------------------------ | :----------- | :----------- | | Shares reserved for exercises of outstanding stock options | 10,108,766 | 6,504,080 | | Shares reserved for vesting of restricted stock units | 78,142 | 76,718 | | Shares reserved for future issuance under the 2019 Stock Incentive Plan | 2,896,782 | 1,941,054 | | Shares reserved for future issuance under the 2019 Employee Stock Purchase Plan | 1,438,938 | 1,061,279 | | Shares reserved for future issuance under the 2022 Inducement Stock Incentive Plan | 1,003,472 | 329,880 | | **Total** | **15,526,100** | **9,913,011** | [9. Stock-based Compensation Expense](index=15&type=section&id=9.%20Stock-based%20Compensation%20Expense) This note details stock-based compensation plans, summarizing stock option and restricted stock unit activity, and total stock-based compensation expense recognized - The **2019 Stock Incentive Plan** and **2022 Inducement Stock Incentive Plan** have seen **increases in shares reserved** for issuance, with **2,896,782** and **1,003,472 shares** available, respectively, as of September 30, 2023[50](index=50&type=chunk)[52](index=52&type=chunk) Stock Option Activity (9 months ended Sep 30, 2023) | Item | Number of Shares | Weighted Average Exercise Price | | :-------------------------- | :--------------- | :------------------------------ | | Outstanding at Dec 31, 2022 | 6,504,080 | $11.99 | | Granted | 5,790,673 | $5.92 | | Exercised | (38,903) | $8.96 | | Cancelled | (2,147,084) | $11.96 | | Outstanding at Sep 30, 2023 | 10,108,766 | $8.53 | | Exercisable at Sep 30, 2023 | 2,890,946 | $12.74 | Stock-based Compensation Expense (in thousands) | Category | 3 Months Ended Sep 30, 2023 | 3 Months Ended Sep 30, 2022 | 9 Months Ended Sep 30, 2023 | 9 Months Ended Sep 30, 2022 | | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | | General and administrative | $2,734 | $2,597 | $8,533 | $7,138 | | Research and development | $1,003 | $756 | $2,820 | $2,941 | | **Total** | **$3,737** | **$3,353** | **$11,353** | **$10,079** | - As of September 30, 2023, the company had **$35.2 million** of unrecognized stock-based compensation expense, to be recognized over a weighted average period of **3.07 years**[58](index=58&type=chunk) [10. Collaboration Agreements](index=17&type=section&id=10.%20Collaboration%20Agreements) This note details collaboration agreements with Acceleron (terminated Oct 2022) and MyoKardia, the latter involving an exclusive worldwide license for cardiomyopathies with upfront, research, milestone, and royalty payments - The **Acceleron Collaboration Agreement**, focused on pulmonary disease targets, terminated effective **October 1, 2022**, resulting in **no collaboration revenue** from this agreement in 2023[61](index=61&type=chunk)[62](index=62&type=chunk) - The **MyoKardia Collaboration Agreement** grants an **exclusive worldwide license** for **genetically defined cardiomyopathies**, including assay screening and research services[64](index=64&type=chunk)[65](index=65&type=chunk) - Under the MyoKardia agreement, the company received a **$10.0 million upfront payment** and **$2.5 million in prepaid research funding**, with potential for up to **$298.5 million** in aggregate **milestone payments** per target and **tiered royalties**[67](index=67&type=chunk) MyoKardia Collaboration Revenue (in thousands) | Period | Collaboration Revenue | | :-------------------------- | :-------------------- | | 3 Months Ended Sep 30, 2023 | $759 | | 3 Months Ended Sep 30, 2022 | $1,183 | | 9 Months Ended Sep 30, 2023 | $1,934 | | 9 Months Ended Sep 30, 2022 | $4,700 | - As of September 30, 2023, deferred revenue from MyoKardia was **$0.3 million**, and unbilled accounts receivable was **$0.6 million**[79](index=79&type=chunk) [11. License Agreements](index=20&type=section&id=11.%20License%20Agreements) This note describes key license agreements: GSK for **losmapimod** (FSHD) and CAMP4 for the **Diamond Blackfan Anemia (DBA)** program, both granting exclusive worldwide rights - Under the **GSK Agreement**, the company has an **exclusive worldwide license** to develop and commercialize **losmapimod**, with potential milestone payments up to **$37.5 million** (including **$7.5 million** achieved) and **tiered royalties** on net sales[80](index=80&type=chunk)[81](index=81&type=chunk) - The **CAMP4 Agreement**, entered in **July 2023**, grants an **exclusive worldwide license** for the **Diamond Blackfan Anemia (DBA) program**, including small molecule compounds and patent rights[84](index=84&type=chunk) - The CAMP4 Agreement includes an undisclosed upfront payment and potential development, regulatory, and sales milestone payments of up to **$35.0 million each**, plus **tiered royalties** on worldwide net sales[85](index=85&type=chunk) [12. Leases](index=22&type=section&id=12.%20Leases) This note details operating lease agreements for corporate headquarters at **26 Landsdowne Street** and office space at **125 Sidney Street**, outlining terms, commitments, and expenses - The lease for **26 Landsdowne Street** (**28,731 sq ft**) commenced **December 2017** and ends **June 30, 2028**, with a total commitment of **$25.1 million** over ten years[89](index=89&type=chunk) Future Minimum Lease Payments (26 Landsdowne Street, in thousands) | Year | Amount | | :--- | :----- | | 2023 | $633 | | 2024 | $2,572 | | 2025 | $2,649 | | 2026 | $2,729 | | 2027 | $2,811 | | Thereafter | $1,426 | | **Total minimum lease payments** | **$12,820** | - Operating lease expense for **26 Landsdowne Street** was approximately **$0.5 million** for the three months and **$1.3 million** for the nine months ended September 30, 2023[89](index=89&type=chunk) - An additional lease for **125 Sidney Street** (**12,196 sq ft**) commenced **November 2021** and ends **March 31, 2024**, with a total commitment of **$1.7 million** over the initial term[91](index=91&type=chunk) [13. Commitments and Contingencies](index=23&type=section&id=13.%20Commitments%20and%20Contingencies) This note addresses commitments and contingencies, including indemnification agreements and a securities class action lawsuit related to a clinical hold on **pociredir** - The company provides **indemnification** to vendors, lessors, business partners, directors, and senior management, with potential **unlimited future payments**, but has **not incurred material costs** to date[93](index=93&type=chunk) - A **class action complaint** (Celano v. Fulcrum Therapeutics, Inc., et al.) was filed on **April 28, 2023**, alleging violations of **Section 10(b)** and **20(a)** of the Exchange Act related to a clinical hold on **pociredir**[94](index=94&type=chunk) - The lawsuit seeks **compensatory damages** for an allegedly inflated stock price between **March 3, 2022**, and **March 8, 2023**, and attorneys' fees and costs. The company intends to **vigorously defend** against this litigation[94](index=94&type=chunk) [14. Defined Contribution Plan](index=23&type=section&id=14.%20Defined%20Contribution%20Plan) This note describes the company's **401(k) defined contribution savings plan**, with contributions of **$0.2 million** (Q3) and **$0.6 million** (YTD Sep) for both 2023 and 2022 - The company has a **401(k) defined contribution savings plan** for eligible employees[96](index=96&type=chunk) 401(k) Plan Contributions (in millions) | Period | 2023 | 2022 | | :-------------------------- | :--- | :--- | | 3 Months Ended Sep 30 | $0.2 | $0.2 | | 9 Months Ended Sep 30 | $0.6 | $0.6 | [15. Net Loss per Share](index=23&type=section&id=15.%20Net%20Loss%20per%20Share) This note provides the calculation of basic and diluted net loss per share, listing anti-dilutive common stock equivalents excluded from the diluted calculation Net Loss Per Share (Basic and Diluted) | Period | 2023 | 2022 | | :-------------------------- | :----- | :----- | | 3 Months Ended Sep 30 | $(0.39) | $(0.51) | | 9 Months Ended Sep 30 | $(1.19) | $(1.97) | Anti-Dilutive Common Stock Equivalents Excluded (in thousands) | Item | 3 Months Ended Sep 30, 2023 | 3 Months Ended Sep 30, 2022 | 9 Months Ended Sep 30, 2023 | 9 Months Ended Sep 30, 2022 | | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | | Outstanding stock options | 10,108,766 | 5,929,065 | 10,108,766 | 5,929,065 | | Unvested restricted stock units | 78,142 | 95,174 | 78,142 | 95,174 | | **Total** | **10,186,908** | **6,024,239** | **10,186,908** | **6,024,239** | [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=24&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) This section discusses financial condition and results, focusing on clinical-stage candidates, operating losses, funding needs, revenue, expenses, and cash flow changes, emphasizing drug development risks [Overview](index=24&type=section&id=Overview) Fulcrum is a clinical-stage biopharmaceutical company developing small molecules for rare diseases, with lead candidates **losmapimod** (FSHD) and **pociredir** (SCD), facing significant losses and requiring additional funding - Fulcrum is a **clinical-stage biopharmaceutical company** focused on genetically defined rare diseases[99](index=99&type=chunk) - **Losmapimod** (FSHD) is in a **Phase 3 clinical trial (REACH)**, with enrollment completed in **September 2023** and topline data expected in **Q4 2024**[99](index=99&type=chunk) - **Pociredir** (SCD) is in a **Phase 1b clinical trial**; a clinical hold was placed in **February 2023** and lifted in **August 2023**. The trial is re-initiating at **12 mg** and **20 mg dose levels**, targeting patients with higher disease severity[100](index=100&type=chunk) - The company has incurred net losses of **$24.0 million** (Q3 2023) and **$72.6 million** (YTD Sep 2023), with an accumulated deficit of **$484.9 million**[103](index=103&type=chunk) - Existing cash, cash equivalents, and marketable securities of **$257.1 million** are expected to fund operations into **2026**, but substantial additional funding will be needed for ongoing R&D and potential commercialization[105](index=105&type=chunk)[107](index=107&type=chunk) [Components of Results of Operations](index=25&type=section&id=Components%20of%20Results%20of%20Operations) This section breaks down revenue and operating expenses, with collaboration revenue from MyoKardia, fluctuating R&D expenses, general and administrative costs, and other income from investments [Revenue](index=25&type=section&id=

[PART I. FINANCIAL INFORMATION](index=1&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) This part presents the company's unaudited financial statements, management's analysis, and disclosures on controls and market risk [Item 1. Financial Statements](index=1&type=section&id=Item%201.%20Financial%20Statements) This section presents the unaudited consolidated financial statements for the periods ended June 30, 2023, and December 31, 2022 [Consolidated Balance Sheets](index=6&type=section&id=Consolidated%20Balance%20Sheets%20as%20of%20June%2030%2C%202023%20and%20December%2031%2C%202022) The balance sheets show a significant increase in total assets and stockholders' equity driven by a recent public offering Key Balance Sheet Metrics | Metric | June 30, 2023 (in thousands) | December 31, 2022 (in thousands) | | :-------------------------------- | :----------------------------- | :------------------------------- | | Total Current Assets | $282,947 | $207,519 | | Total Assets | $300,332 | $226,685 | | Total Current Liabilities | $14,804 | $16,725 | | Total Liabilities | $24,904 | $27,743 | | Total Stockholders' Equity | $275,428 | $198,942 | - Total assets increased by **$73.6 million**, primarily driven by an increase in marketable securities[15](index=15&type=chunk) - Total stockholders' equity increased by **$76.5 million**, largely due to a public offering of common stock in January 2023[15](index=15&type=chunk)[29](index=29&type=chunk) [Consolidated Statements of Operations and Comprehensive Loss](index=7&type=section&id=Consolidated%20Statements%20of%20Operations%20and%20Comprehensive%20Loss%20for%20the%20three%20and%20six%20months%20ended%20June%2030%2C%202023%20and%202022) The company's net loss narrowed year-over-year due to decreased operating expenses, particularly in research and development Key Operational Metrics | Metric (in thousands) | 3 Months Ended Jun 30, 2023 | 3 Months Ended Jun 30, 2022 | 6 Months Ended Jun 30, 2023 | 6 Months Ended Jun 30, 2022 | | :-------------------- | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | | Collaboration Revenue | $880 | $1,882 | $1,175 | $4,474 | | R&D Expenses | $17,849 | $25,019 | $34,564 | $42,850 | | G&A Expenses | $10,323 | $11,098 | $21,843 | $21,857 | | Net Loss | $(23,783) | $(34,070) | $(48,562) | $(59,998) | | Net Loss per Share | $(0.38) | $(0.83) | $(0.80) | $(1.47) | - Net loss decreased by **$10.3 million** for the three months ended June 30, 2023, and by **$11.4 million** for the six months ended June 30, 2023, primarily due to decreased operating expenses and increased other income[17](index=17&type=chunk)[127](index=127&type=chunk)[134](index=134&type=chunk) - Research and development expenses decreased by **$7.2 million** (3 months YoY) and **$8.3 million** (6 months YoY), mainly due to a **$5.0 million** milestone payment in Q2 2022 and reduced FTX-6058 costs due to a clinical hold[129](index=129&type=chunk)[130](index=130&type=chunk)[136](index=136&type=chunk)[138](index=138&type=chunk) [Consolidated Statements of Stockholders' Equity](index=8&type=section&id=Consolidated%20Statements%20of%20Stockholders%27%20Equity%20for%20the%20three%20and%20six%20months%20ended%20June%2030%2C%202023%20and%202022) Stockholders' equity increased significantly due to a public stock offering, while the accumulated deficit continued to grow Key Equity Metrics | Metric (in thousands) | Dec 31, 2022 | Jun 30, 2023 | | :-------------------- | :----------- | :----------- | | Common Stock | $52 | $62 | | Additional Paid-In Capital | $612,025 | $737,466 | | Accumulated Deficit | $(412,338) | $(460,900) | | Total Stockholders' Equity | $198,942 | $275,428 | - Additional paid-in capital increased significantly by **$125.4 million**, primarily from a public offering of common stock in January 2023, which generated net proceeds of **$117.3 million**[20](index=20&type=chunk)[29](index=29&type=chunk) - Accumulated deficit increased to **$460.9 million** as of June 30, 2023, reflecting ongoing net losses[20](index=20&type=chunk)[105](index=105&type=chunk) [Consolidated Statements of Cash Flows](index=9&type=section&id=Consolidated%20Statements%20of%20Cash%20Flows%20for%20the%20six%20months%20ended%20June%2030%2C%202023%20and%202022) Cash flow from financing activities increased substantially due to a stock offering, offsetting cash used in operations and investments Key Cash Flow Metrics | Metric (in thousands) | 6 Months Ended Jun 30, 2023 | 6 Months Ended Jun 30, 2022 | | :-------------------- | :-------------------------- | :-------------------------- | | Net Cash Used in Operating Activities | $(44,901) | $(49,472) | | Net Cash (Used in) Provided by Investing Activities | $(74,066) | $51,054 | | Net Cash Provided by Financing Activities | $117,835 | $3,159 | | Net (Decrease) Increase in Cash, Cash Equivalents, and Restricted Cash | $(1,132) | $4,741 | - Net cash used in operating activities decreased by **$4.6 million**, primarily due to lower external R&D costs related to FTX-6058's clinical hold[143](index=143&type=chunk) - Net cash provided by financing activities significantly increased to **$117.8 million**, driven by **$117.3 million** in net proceeds from a public common stock offering in January 2023[145](index=145&type=chunk) - Investing activities shifted from providing **$51.1 million** in cash in 2022 to using **$74.1 million** in 2023, mainly due to net purchases of marketable securities[144](index=144&type=chunk) [Notes to Consolidated Financial Statements](index=10&type=section&id=Notes%20to%20Consolidated%20Financial%20Statements) These notes detail the company's accounting policies, financial instruments, agreements, and other key financial information [1. Nature of the Business and Basis of Presentation](index=10&type=section&id=1.%20Nature%20of%20the%20Business%20and%20Basis%20of%20Presentation) The company is a clinical-stage biopharmaceutical firm with a history of losses, funded by a recent public offering - Fulcrum Therapeutics, Inc is a clinical-stage biopharmaceutical company focused on genetically-defined rare diseases[25](index=25&type=chunk) - The company has incurred significant losses since inception, with an accumulated deficit of **$460.9 million** as of June 30, 2023, and expects continued losses[11](index=11&type=chunk)[30](index=30&type=chunk) - In January 2023, the company completed a public offering, issuing 9,615,384 shares of common stock at $13.00 per share, generating net proceeds of **$117.3 million**[29](index=29&type=chunk) - Management estimates that existing cash, cash equivalents, and marketable securities will fund operations for at least 12 months from the financial statement issuance date[31](index=31&type=chunk) [2. Summary of Significant Accounting Policies](index=11&type=section&id=2.%20Summary%20of%20Significant%20Accounting%20Policies) No material changes were made to significant accounting policies, and new accounting standards had no material impact - The consolidated financial statements include Fulcrum Therapeutics, Inc and its wholly-owned subsidiary, Fulcrum Therapeutics Securities Corp, with all intercompany transactions eliminated[32](index=32&type=chunk) - No material changes occurred in significant accounting policies during the six months ended June 30, 2023[33](index=33&type=chunk) - The company adopted ASU No 2016-13 (Credit Losses) and ASU No 2019-12 (Income Taxes) effective January 1, 2023, with no material impact on financial position or results[36](index=36&type=chunk)[37](index=37&type=chunk) [3. Fair Value Measurements](index=12&type=section&id=3.%20Fair%20Value%20Measurements) The company's financial assets, primarily cash equivalents and marketable securities, are measured at fair value using Level 2 inputs Fair Value of Financial Assets | Asset Type (in thousands) | June 30, 2023 Total Fair Value | December 31, 2022 Total Fair Value | | :------------------------ | :----------------------------- | :------------------------------- | | Cash Equivalents | $33,966 | $35,098 | | Marketable Securities | $244,198 | $167,823 | | Total | $278,164 | $202,921 | - The majority of financial assets are measured at fair value using **Level 2 inputs** (observable inputs other than quoted prices)[38](index=38&type=chunk) - There were no transfers between fair value levels during the three and six months ended June 30, 2023[38](index=38&type=chunk) [4. Cash Equivalents and Marketable Securities](index=13&type=section&id=4.%20Cash%20Equivalents%20and%20Marketable%20Securities) The company holds a significant portfolio of marketable securities, with a large portion in an unrealized loss position Fair Value of Cash Equivalents and Marketable Securities | Asset Type (in thousands) | June 30, 2023 Fair Value | December 31, 2022 Fair Value | | :------------------------ | :----------------------- | :--------------------------- | | Cash Equivalents | $33,966 | $35,098 | | Marketable Securities | $244,198 | $167,823 | | Total | $278,164 | $202,921 | - As of June 30, 2023, the company held 91 debt securities in an unrealized loss position for less than 12 months, with an aggregate fair value of **$222.2 million**[39](index=39&type=chunk) - The company did not record any credit-related impairments for marketable securities, as it has the intent and ability to hold debt securities until recovery[40](index=40&type=chunk) [5. Property and Equipment, Net](index=14&type=section&id=5.%20Property%20and%20Equipment%2C%20Net) Net property and equipment decreased slightly due to depreciation expense outpacing new acquisitions Property and Equipment Breakdown | Category (in thousands) | June 30, 2023 | December 31, 2022 | | :---------------------- | :------------ | :---------------- | | Lab equipment | $9,475 | $9,057 | | Leasehold improvements | $7,102 | $7,102 | | Total Property and Equipment, Net | $6,161 | $6,906 | - Property and equipment, net, decreased by **$0.7 million** from December 31, 2022, to June 30, 2023[41](index=41&type=chunk) - Depreciation expense for the six months ended June 30, 2023, was **$1.1 million**, a slight decrease from $1.2 million in the prior year period[41](index=41&type=chunk) [6. Additional Balance Sheet Detail](index=14&type=section&id=6.%20Additional%20Balance%20Sheet%20Detail) Accrued expenses decreased primarily due to lower R&D and payroll accruals, while interest income receivable increased Selected Balance Sheet Accounts | Category (in thousands) | June 30, 2023 | December 31, 2022 | | :---------------------- | :------------ | :---------------- | | Prepaid expenses and other current assets | $4,127 | $4,369 | | Accrued expenses and other current liabilities | $7,875 | $9,551 | - Prepaid expenses decreased, while interest income receivable increased significantly from **$852 thousand to $1.4 million**[42](index=42&type=chunk) - Accrued expenses and other current liabilities decreased by **$1.7 million**, primarily due to lower payroll and benefits and external research and development accruals[42](index=42&type=chunk) [7. Preferred Stock](index=14&type=section&id=7.%20Preferred%20Stock) The company has authorized preferred stock but has not issued any shares - As of June 30, 2023, and December 31, 2022, the company had **5,000,000 shares** of undesignated preferred stock authorized, with no shares issued or outstanding[43](index=43&type=chunk) [8. Common Stock](index=14&type=section&id=8.%20Common%20Stock) The number of common shares outstanding increased significantly following a public offering in January 2023 - As of June 30, 2023, **61,822,554 shares** of common stock were issued and outstanding, up from 52,099,211 shares at December 31, 2022[15](index=15&type=chunk) - The increase in outstanding shares is primarily due to the public offering in January 2023[29](index=29&type=chunk) Shares Reserved for Future Issuance | Shares Reserved for Future Issuance | June 30, 2023 | December 31, 2022 | | :---------------------------------- | :------------ | :---------------- | | Outstanding stock options | 10,114,591 | 6,504,080 | | Future issuance under 2019 Stock Incentive Plan | 2,512,307 | 1,941,054 | | Future issuance under 2022 Inducement Stock Incentive Plan | 1,482,845 | 329,880 | [9. Stock-based Compensation Expense](index=15&type=section&id=9.%20Stock-based%20Compensation%20Expense) Stock-based compensation expense increased year-over-year, with a significant amount of unrecognized expense remaining Stock-based Compensation Breakdown | Stock-based Compensation (in thousands) | 3 Months Ended Jun 30, 2023 | 3 Months Ended Jun 30, 2022 | 6 Months Ended Jun 30, 2023 | 6 Months Ended Jun 30, 2022 | | :-------------------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | | General and administrative | $2,391 | $2,076 | $5,799 | $4,541 | | Research and development | $972 | $803 | $1,817 | $2,185 | | Total | $3,363 | $2,879 | $7,616 | $6,726 | - The company has several stock incentive plans (2016, 2019, and 2022 Inducement Plan) and an Employee Stock Purchase Plan (ESPP)[48](index=48&type=chunk)[49](index=49&type=chunk)[51](index=51&type=chunk)[58](index=58&type=chunk) - Total stock-based compensation expense increased by **$0.5 million** for the three months and **$0.9 million** for the six months ended June 30, 2023, compared to the prior year periods[57](index=57&type=chunk) - As of June 30, 2023, unrecognized stock-based compensation expense totaled **$37.8 million**, expected to be recognized over a weighted average period of 3.25 years[57](index=57&type=chunk) [10. Collaboration and License Agreements](index=17&type=section&id=10.%20Collaboration%20and%20License%20Agreements) Collaboration revenue is primarily generated from the MyoKardia agreement, following the termination of the Acceleron agreement - The Acceleron Collaboration Agreement terminated effective October 1, 2022, resulting in no collaboration revenue from this agreement in 2023[60](index=60&type=chunk)[61](index=61&type=chunk) - Under the MyoKardia Collaboration Agreement, the company recognized **$0.9 million** and **$1.2 million** in collaboration revenue for the three and six months ended June 30, 2023, respectively[79](index=79&type=chunk) - MyoKardia made a **$10.0 million** upfront payment and **$2.5 million** prepaid research funding in July 2020, and the company achieved a **$2.5 million** preclinical milestone[66](index=66&type=chunk) - The MyoKardia agreement includes potential milestone payments up to **$298.5 million** per target and tiered royalties on net sales[66](index=66&type=chunk) [11. Right of Reference and License Agreement (GSK)](index=20&type=section&id=11.%20Right%20of%20Reference%20and%20License%20Agreement) The company's agreement with GSK for losmapimod includes significant potential milestone payments and royalties - The company has an exclusive worldwide license from GSK to develop and commercialize losmapimod[80](index=80&type=chunk) - The agreement includes potential milestone payments up to **$37.5 million** (clinical/regulatory) and **$60.0 million** (sales), plus tiered royalties on net sales[81](index=81&type=chunk) - A **$5.0 million** clinical milestone was achieved during Q2 2022[81](index=81&type=chunk) [12. Leases](index=21&type=section&id=12.%20Leases) The company holds operating leases for its corporate headquarters and additional office space with varying expiration dates Lease Liability Summary | Lease Liability (in thousands) | 26 Landsdowne Street | 125 Sidney Street | | :----------------------------- | :------------------- | :---------------- | | Total minimum lease payments | $13,453 | $631 | | Total lease liability | $11,540 | $620 | - The company leases corporate headquarters at 26 Landsdowne Street (expiring June 2028) and office space at 125 Sidney Street (expiring March 2024)[84](index=84&type=chunk)[86](index=86&type=chunk) - Operating lease expense for the six months ended June 30, 2023, was approximately **$0.9 million** for 26 Landsdowne Street and **$0.4 million** for 125 Sidney Street[84](index=84&type=chunk)[86](index=86&type=chunk) [13. Commitments and Contingencies](index=22&type=section&id=13.%20Commitments%20and%20Contingencies) The company is defending against a class action lawsuit related to the clinical hold on its FTX-6058 program - The company is subject to a class action lawsuit (Celano v Fulcrum Therapeutics, Inc, et al) filed in April 2023, alleging securities law violations related to the FTX-6058 clinical hold[89](index=89&type=chunk) - The lawsuit seeks compensatory damages for an allegedly inflated stock price between March 3, 2022, and March 8, 2023, and attorneys' fees[89](index=89&type=chunk) - The company intends to vigorously defend against this litigation and has not incurred material costs related to legal proceedings during the reporting period[89](index=89&type=chunk)[90](index=90&type=chunk) [14. Defined Contribution Plan](index=22&type=section&id=14.%20Defined%20Contribution%20Plan) The company maintains a 401(k) plan for employees with consistent contribution levels year-over-year - The company has a 401(k) Plan for eligible employees[91](index=91&type=chunk) - Contributions to the 401(k) Plan were **$0.2 million** for the three months and **$0.4 million** for the six months ended June 30, 2023, consistent with the prior year[91](index=91&type=chunk) [15. Net Loss per Share](index=22&type=section&id=15.%20Net%20Loss%20per%20Share) All potential common stock equivalents were excluded from diluted net loss per share calculations due to their anti-dilutive effect Anti-Dilutive Securities | Common Stock Equivalents | 3 Months Ended Jun 30, 2023 | 3 Months Ended Jun 30, 2022 | 6 Months Ended Jun 30, 2023 | 6 Months Ended Jun 30, 2022 | | :----------------------- | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | | Outstanding stock options | 10,114,591 | 6,137,740 | 10,114,591 | 6,137,740 | | Unvested restricted stock units | 84,942 | 115,189 | 84,942 | 115,189 | - All common stock equivalents were excluded from diluted net loss per share calculations due to their anti-dilutive effect[92](index=92&type=chunk) [16. Subsequent Events](index=22&type=section&id=16.%20Subsequent%20Events) The company entered into a new exclusive license agreement with CAMP4 Therapeutics after the reporting period - In July 2023, the company entered into a worldwide exclusive license agreement with CAMP4 Therapeutics Corporation for its Diamond Blackfan Anemia (DBA) program[93](index=93&type=chunk)[94](index=94&type=chunk) - Under the CAMP4 agreement, the company will make an undisclosed upfront payment and CAMP4 is eligible for up to **$35.0 million** in development/regulatory milestones and **$35.0 million** in sales milestones, plus tiered royalties[95](index=95&type=chunk) - The company is currently evaluating the accounting treatment for the CAMP4 license agreement[97](index=97&type=chunk) [Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations](index=24&type=section&id=Item%202.%20Management%27s%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) This section analyzes the company's financial condition, results of operations, clinical programs, and future funding needs [Overview](index=24&type=section&id=Overview) The company is advancing its lead candidate losmapimod while its FTX-6058 program remains on clinical hold - Fulcrum Therapeutics is a clinical-stage biopharmaceutical company focused on genetically defined rare diseases, with lead product candidates losmapimod (FSHD) and FTX-6058 (hemoglobinopathies)[100](index=100&type=chunk) - The Phase 3 clinical trial for losmapimod (REACH) is expected to complete enrollment in Q3 2023 and report topline data in Q4 2024[100](index=100&type=chunk) - The FTX-6058 program for SCD is currently on a **full clinical hold** by the FDA as of February 23, 2023, and the IND for beta thalassemia was withdrawn[101](index=101&type=chunk) - The company's proprietary product engine, FulcrumSeek, is used to identify and validate cellular drug targets for genetically defined diseases[103](index=103&type=chunk) - Net losses were **$23.8 million** and **$48.6 million** for the three and six months ended June 30, 2023, respectively, with an accumulated deficit of **$460.9 million**[105](index=105&type=chunk) [Components of Results of Operations](index=25&type=section&id=Components%20of%20Results%20of%20Operations) This section breaks down the key drivers of revenue and operating expenses [Revenue](index=25&type=section&id=Revenue) Collaboration revenue decreased due to the termination of the Acceleron agreement and lower revenue from MyoKardia - The company has not generated product sales revenue and does not expect to for several years[109](index=109&type=chunk) - Collaboration revenue decreased due to the termination of the Acceleron agreement (effective Oct 2022) and lower revenue from the MyoKardia agreement[110](index=110&type=chunk)[113](index=113&type=chunk) Collaboration Revenue Breakdown | Collaboration Revenue (in thousands) | 3 Months Ended Jun 30, 2023 | 3 Months Ended Jun 30, 2022 | 6 Months Ended Jun 30, 2023 | 6 Months Ended Jun 30, 2022 | | :----------------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | | Acceleron | $0 | $100 | $0 | $1,000 | | MyoKardia | $900 | $1,700 | $1,200 | $3,500 | [Operating Expenses](index=27&type=section&id=Operating%20Expenses) Operating expenses are primarily driven by research and development and general and administrative costs [Research and Development Expenses](index=27&type=section&id=Research%20and%20Development%20Expenses) R&D expenses decreased significantly year-over-year, driven by a prior-year milestone payment and the FTX-6058 clinical hold - R&D expenses decreased by **$7.2 million** (3 months YoY) and **$8.3 million** (6 months YoY)[129](index=129&type=chunk)[136](index=136&type=chunk) - The decrease was primarily due to a **$5.0 million** milestone payment to GSK in Q2 2022 and reduced costs for FTX-6058 following its clinical hold[130](index=130&type=chunk)[138](index=138&type=chunk) - R&D expenses are expected to increase significantly in future periods as clinical trials advance and discovery efforts expand[123](index=123&type=chunk) R&D Expense Breakdown | R&D Expenses (in thousands) | 3 Months Ended Jun 30, 2023 | 3 Months Ended Jun 30, 2022 | 6 Months Ended Jun 30, 2023 | 6 Months Ended Jun 30, 2022 | | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | | External R&D | $10,353 | $16,416 | $19,903 | $24,793 | | Employee compensation | $5,037 | $5,311 | $9,844 | $11,531 | | Total R&D Expenses | $17,849 | $25,019 | $34,564 | $42,850 | [General and Administrative Expenses](index=28&type=section&id=General%20and%20Administrative%20Expenses) G&A expenses remained relatively flat year-over-year, with lower professional services costs offsetting higher compensation - G&A expenses decreased by **$0.8 million** (3 months YoY) and remained relatively flat (less than $0.1 million decrease) for the six months YoY[132](index=132&type=chunk)[137](index=137&type=chunk) - The decrease was primarily due to lower professional services costs (consulting, insurance, legal), offset by increased employee compensation and facility costs[132](index=132&type=chunk)[137](index=137&type=chunk) G&A Expense Breakdown | G&A Expenses (in thousands) | 3 Months Ended Jun 30, 2023 | 3 Months Ended Jun 30, 2022 | 6 Months Ended Jun 30, 2023 | 6 Months Ended Jun 30, 2022 | | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | :-------------------------- | | Employee compensation | $5,710 | $5,690 | $12,977 | $11,516 | | Professional services | $3,137 | $4,125 | $5,955 | $8,046 | | Total G&A Expenses | $10,323 | $11,098 | $21,843 | $21,857 | [Other Income, Net](index=29&type=section&id=Other%20Income%2C%20Net) Other income increased substantially due to higher interest rates and a larger cash and investment balance - Other income, net, increased significantly by **$3.3 million** (3 months YoY) and **$6.4 million** (6 months YoY)[133](index=133&type=chunk)[139](index=139&type=chunk) - This increase was primarily due to a higher rate of return on cash, cash equivalents, and marketable securities, coupled with an increased average cash and investment balance[133](index=133&type=chunk)[139](index=139&type=chunk) [Results of Operations](index=29&type=section&id=Results%20of%20Operations) This section provides a comparative analysis of the company's operational results for the three and six-month periods [Comparison of the Three Months ended June 30, 2023 and 2022](index=29&type=section&id=Comparison%20of%20the%20Three%20Months%20ended%20June%2030%2C%202023%20and%202022) Net loss decreased significantly due to lower R&D expenses and higher other income Three-Month Operational Comparison | Metric (in thousands) | Jun 30, 2023 | Jun 30, 2022 | Change ($) | | :-------------------- | :----------- | :----------- | :--------- | | Collaboration revenue | $880 | $1,882 | $(1,002) | | R&D expenses | $17,849 | $25,019 | $(7,170) | | G&A expenses | $10,323 | $11,098 | $(775) | | Net loss | $(23,783) | $(34,070) | $10,287 | - Net loss decreased by **$10.3 million**, primarily driven by a **$7.2 million** reduction in R&D expenses and a **$3.3 million** increase in other income, net[127](index=127&type=chunk)[129](index=129&type=chunk)[133](index=133&type=chunk) [Comparison of the Six Months ended June 30, 2023 and 2022](index=30&type=section&id=Comparison%20of%20the%20Six%20Months%20ended%20June%2030%2C%202023%20and%202022) Net loss narrowed due to reduced R&D spending and higher other income, which offset lower collaboration revenue Six-Month Operational Comparison | Metric (in thousands) | Jun 30, 2023 | Jun 30, 2022 | Change ($) | | :-------------------- | :----------- | :----------- | :--------- | | Collaboration revenue | $1,175 | $4,474 | $(3,299) | | R&D expenses | $34,564 | $42,850 | $(8,286) | | G&A expenses | $21,843 | $21,857 | $(14) | | Net loss | $(48,562) | $(59,998) | $11,436 | - Net loss decreased by **$11.4 million**, primarily due to an **$8.3 million** reduction in R&D expenses and a **$6.4 million** increase in other income, net, partially offset by a **$3.3 million** decrease in collaboration revenue[134](index=134&type=chunk)[136](index=136&type=chunk)[139](index=139&type=chunk) [Liquidity and Capital Resources](index=32&type=section&id=Liquidity%20and%20Capital%20Resources) The company believes its current capital is sufficient to fund operations into mid-2025 but will require future financing - As of June 30, 2023, the company had **$278.2 million** in cash, cash equivalents, and marketable securities[140](index=140&type=chunk) - The company expects existing capital to fund operating expenses and capital expenditure requirements into **mid-2025**[147](index=147&type=chunk) - Future funding requirements depend on clinical trial progress, R&D expansion, regulatory approvals, manufacturing, and potential collaborations[148](index=148&type=chunk) - The company anticipates financing future cash needs through equity offerings, debt financings, collaborations, strategic alliances, and licensing arrangements[150](index=150&type=chunk) [Critical Accounting Policies and Estimates](index=34&type=section&id=Critical%20Accounting%20Policies%20and%20Estimates) No material changes to critical accounting policies were reported during the period - No material changes to critical accounting policies were reported during the three months ended June 30, 2023, from those described in the Annual Report on Form 10-K[152](index=152&type=chunk) [Recently Issued Accounting Pronouncements](index=34&type=section&id=Recently%20Issued%20Accounting%20Pronouncements) Information on recently issued accounting pronouncements is available in the notes to the financial statements - A description of recently issued accounting pronouncements is disclosed in Note 2 to the consolidated financial statements[153](index=153&type=chunk) [Emerging Growth Company Status](index=34&type=section&id=Emerging%20Growth%20Company%20Status) The company utilizes the extended transition period for new accounting standards available to emerging growth companies - The company is an 'emerging growth company' (EGC) and has elected not to opt out of the extended transition period for complying with new or revised accounting standards[154](index=154&type=chunk) [Item 3. Quantitative and Qualitative Disclosures About Market Risk](index=34&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company is exempt from market risk disclosure requirements as a smaller reporting company - The company is a smaller reporting company and is not required to provide disclosures about market risk[155](index=155&type=chunk) [Item 4. Controls and Procedures](index=35&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded that disclosure controls and internal financial reporting controls were effective as of the period end [Evaluation of Disclosure Controls and Procedures](index=35&type=section&id=Evaluation%20of%20Disclosure%20Controls%20and%20Procedures) Disclosure controls and procedures were deemed effective at a reasonable assurance level - Management, including the CEO, concluded that disclosure controls and procedures were effective at the reasonable assurance level as of June 30, 2023[157](index=157&type=chunk) [Changes in Internal Control over Financial Reporting](index=35&type=section&id=Changes%20in%20Internal%20Control%20over%20Financial%20Reporting) No material changes in internal control over financial reporting occurred during the quarter - No material changes in internal control over financial reporting occurred during the quarter ended June 30, 2023[158](index=158&type=chunk) [PART II. OTHER INFORMATION](index=36&type=section&id=PART%20II.%20OTHER%20INFORMATION) This part covers legal proceedings, risk factors, and other required disclosures [Item 1. Legal Proceedings](index=36&type=section&id=Item%201.%20Legal%20Proceedings) The company faces a class action lawsuit alleging securities violations related to the clinical hold on its FTX-6058 program - A class action complaint was filed on April 28, 2023, alleging violations of Section 10(b) and Rule 10b-5 of the Securities Exchange Act of 1934[161](index=161&type=chunk) - The lawsuit is related to the FDA's clinical hold on the IND application for FTX-6058 for sickle cell disease, announced in February 2023[161](index=161&type=chunk) - The plaintiffs seek compensatory damages for an allegedly inflated stock price between March 3, 2022, and March 8, 2023, and attorneys' fees[161](index=161&type=chunk) [Item 1A. Risk Factors](index=36&type=section&id=Item%201A.%20Risk%20Factors) This section details significant risks that could materially affect the company's business, financial condition, and operations [Risks Related to our Financial Position and Need for Additional Capital](index=36&type=section&id=Risks%20Related%20to%20our%20Financial%20Position%20and%20Need%20for%20Additional%20Capital) The company's history of losses and need for substantial future funding pose significant financial risks - The company has incurred significant losses since inception (**$460.9 million** accumulated deficit as of June 30, 2023) and expects to continue incurring losses, potentially never achieving profitability[163](index=163&type=chunk) - Substantial additional funding will be required to support ongoing and planned clinical trials (losmapimod, FTX-6058), R&D, and potential commercialization efforts[167](index=167&type=chunk) - Inability to raise capital when needed could force delays, reductions, or elimination of product development programs or commercialization efforts[167](index=167&type=chunk) - Adverse developments in the financial services industry, such as bank failures, could impair access to funding and adversely affect business operations[189](index=189&type=chunk)[190](index=190&type=chunk) [Risks Related to the Discovery and Development of our Product Candidates](index=41&type=section&id=Risks%20Related%20to%20the%20Discovery%20and%20Development%20of%20our%20Product%20Candidates) The early stage of development and the clinical hold on FTX-6058 create substantial uncertainty for product candidates - The company is early in development with only two clinical-stage candidates (losmapimod, FTX-6058), and **FTX-6058 is currently on clinical hold**, posing significant commercialization risks[191](index=191&type=chunk) - Clinical drug development is lengthy, expensive, and uncertain; preclinical and early clinical trial results may not predict future success, and serious adverse events could halt development[195](index=195&type=chunk)[209](index=209&type=chunk) - The FDA placed a clinical hold on FTX-6058 due to hematological malignancies in nonclinical toxicology studies, which may prevent or delay its clinical development[210](index=210&type=chunk) - Challenges in patient enrollment for clinical trials, especially for rare diseases, could delay or prevent regulatory approvals[205](index=205&type=chunk)[208](index=208&type=chunk) [Risks Related to the Commercialization of our Product Candidates](index=47&type=section&id=Risks%20Related%20to%20the%20Commercialization%20of%20our%20Product%20Candidates) The company faces significant hurdles in market acceptance, competition, manufacturing, and pricing for its potential products - Even if approved, product candidates may fail to achieve market acceptance by physicians, patients, and payors, limiting commercial success[218](index=218&type=chunk) - The company lacks its own sales, marketing, and distribution capabilities and faces risks if unable to establish them or secure effective third-party agreements[220](index=220&type=chunk)[222](index=222&type=chunk) - Substantial competition from larger pharmaceutical and biotechnology companies, with greater resources, poses a significant threat to market share[223](index=223&type=chunk)[227](index=227&type=chunk) - Reliance on contract manufacturing organizations (CMOs) for product supply introduces risks of delays, quality issues, or inability to meet demand[231](index=231&type=chunk)[232](index=232&type=chunk) - Unfavorable pricing regulations, inadequate third-party coverage, or healthcare reform initiatives could harm profitability and market access[241](index=241&type=chunk)[242](index=242&type=chunk) [Risks Related to our Dependence on Third Parties](index=53&type=section&id=Risks%20Related%20to%20our%20Dependence%20on%20Third%20Parties) The company's reliance on CROs and collaboration partners introduces risks related to performance and control - Reliance on third-party CROs to conduct clinical trials reduces control and poses risks of unsatisfactory performance or missed deadlines[250](index=250&type=chunk)[251](index=251&type=chunk)[252](index=252&type=chunk) - Collaborations with third parties, such as MyoKardia, may not be successful, and the company may have limited control over collaborators' efforts and resource allocation[254](index=254&type=chunk)[255](index=255&type=chunk) - Failure to establish or maintain collaborations could force the company to alter development and commercialization plans, potentially requiring additional capital or expertise[262](index=262&type=chunk) [Risks Related to our Intellectual Property](index=55&type=section&id=Risks%20Related%20to%20our%20Intellectual%20Property) Protecting intellectual property is critical and faces challenges from patent prosecution, litigation, and license compliance - Inability to obtain, maintain, enforce, and protect patent protection for technology and product candidates could allow competitors to commercialize similar products[263](index=263&type=chunk)[264](index=264&type=chunk) - Patent prosecution is expensive and complex, with uncertain outcomes regarding issuance, scope, validity, and enforceability[265](index=265&type=chunk)[266](index=266&type=chunk) - Third parties may allege infringement of their intellectual property rights, leading to costly litigation, potential damages, or the need to obtain licenses on unfavorable terms[285](index=285&type=chunk)[290](index=290&type=chunk) - Failure to comply with obligations under intellectual property licenses could result in termination of agreements and loss of critical intellectual property rights[295](index=295&type=chunk) - Inability to protect trade secrets and proprietary information could harm the company's business and competitive position[309](index=309&type=chunk) [Risks Related to Regulatory Approval of our Product Candidates and Other Legal Compliance Matters](index=65&type=section&id=Risks%20Related%20to%20Regulatory%20Approval%20of%20our%20Product%20Candidates%20and%20Other%20Legal%20Compliance%20Matters) The company faces a complex, lengthy, and uncertain regulatory landscape for product approval and post-marketing compliance - The marketing approval process is expensive, time-consuming, and uncertain; delays or failure to obtain approvals would materially impair revenue generation[312](index=312&type=chunk)[316](index=316&type=chunk) - Orphan drug designation or exclusivity may not be obtained or, if granted, may not effectively prevent competition[320](index=320&type=chunk)[323](index=323&type=chunk) - Special designations like fast track or breakthrough therapy do not guarantee faster development, review, or approval[324](index=324&type=chunk)[327](index=327&type=chunk) - Any approved product will be subject to post-marketing restrictions, and non-compliance with regulatory requirements could lead to substantial penalties or market withdrawal[334](index=334&type=chunk)[336](index=336&type=chunk) - Compliance with global privacy and data security requirements (e g, GDPR, CCPA) could result in additional costs, liabilities, or significant fines[342](index=342&type=chunk)[347](index=347&type=chunk) [Risks Related to Employee Matters and Managing Growth](index=73&type=section&id=Risks%20Related%20to%20Employee%20Matters%20and%20Managing%20Growth) Retaining key personnel and managing expected growth are critical to the company's future success - Future success depends on retaining key executives and attracting/retaining qualified personnel, especially given recent executive transitions and a workforce reduction[365](index=365&type=chunk)[366](index=366&type=chunk) - Expected significant growth in employees and operations (drug development, regulatory, sales/marketing) may lead to difficulties in management and could disrupt operations[367](index=367&type=chunk) [Risks Related to our Common Stock](index=74&type=section&id=Risks%20Related%20to%20our%20Common%20Stock) Common stock ownership is concentrated, and its price is subject to high volatility and market conditions - Executive officers, directors, and principal stockholders collectively own approximately **48.9%** of capital stock, enabling them to control or significantly influence matters submitted to stockholders[368](index=368&type=chunk) - Provisions in corporate charter documents and Delaware law could make company acquisition more difficult and prevent attempts to replace management[370](index=370&type=chunk)[371](index=371&type=chunk) - The price of common stock is volatile and can fluctuate substantially due to various factors, including clinical trial results, regulatory developments, and market conditions[373](index=373&type=chunk)[374](index=374&type=chunk) - As an 'emerging growth company,' reduced disclosure requirements may make common stock less attractive to investors, potentially leading to lower trading volume and increased price volatility[378](index=378&type=chunk)[379](index=379&type=chunk) [Item 2. Unregistered Sales of Equity Securities and Use of Proceeds](index=78&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) This item is not applicable for the reporting period - This item is not applicable[387](index=387&type=chunk) [Item 3. Defaults Upon Senior Securities](index=78&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) This item is not applicable for the reporting period - This item is not applicable[388](index=388&type=chunk) [Item 4. Mine Safety Disclosures](index=78&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable for the reporting period - This item is not applicable[389](index=389&type=chunk) [Item 5. Other Information](index=78&type=section&id=Item%205.%20Other%20Information) No other information is reported under this item - No other information is reported[390](index=390&type=chunk) [Item 6. Exhibits](index=79&type=section&id=Item%206.%20Exhibits) This section lists all exhibits filed as part of the Form 10-Q - Exhibits include the Restated Certificate of Incorporation, Amended and Restated Bylaws, First Amendment to Collaboration and License Agreement with MyoKardia, and Employment Agreement with Alex C Sapir[394](index=394&type=chunk) - Certifications of the Principal Executive Officer and Principal Financial Officer are included, as well as Inline XBRL Instance and Taxonomy Extension Documents[394](index=394&type=chunk) [Signatures](index=80&type=section&id=Signatures) The report is duly signed on behalf of the company by its President and Chief Executive Officer - The report was signed by Alex C Sapir, President and Chief Executive Officer (Principal Executive Officer and Principal Financial Officer) on August 3, 2023[398](index=398&type=chunk)

Fulcrum Therapeutics, Inc. (NASDAQ:FULC) Q1 2023 Earnings Conference Call May 15, 2023 8:00 AM ET Company Participants Chris Calabrese - LifeSci Advisors, LLC Robert J. Gould - President and CEO Iain Fraser - Interim Chief Medical Officer Conference Call Participants Edward Tenthoff - Piper Sandler Joseph Schwartz - SVB Securities Unidentified Analyst - Goldman Sachs Matthew Biegler - Oppenheimer Judah Frommer - Crédit Suisse Operator Good morning and welcome to the Fulcrum Therapeutics First Quarter 2023 F ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 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, 2023 | Title of each class | Trading Symbol(s) | Name of each exchange on which registered | | --- | --- | --- | | Common stock, par value $0.001 per share | FULC | The Nasdaq Global Market | or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Fo ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2022 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 FOR THE TRANSITION PERIOD FROM TO Commission File Number 001-38978 FULCRUM THERAPEUTICS, INC. (Exact name of registrant as specified in its Charter) | Delaware | 47-4839948 | | --- | --- | | (St ...

Fulcrum Therapeutics, Inc. (NASDAQ:FULC) Q3 2022 Results Conference Call November 8, 2022 8:00 AM ET Company Participants Stephanie Ascher - Stern Investor Relations Bryan Stuart - President and Chief Executive Officer Esther Rajavelu - CFO Paul Bruno - Senior Vice President, Corporate Development Conference Call Participants Dae Gon Ha - Stifel Judah Frommer - Credit Suisse Matt Biegler - Oppenheimer Joseph Schwartz - SVB Leerink Operator Good morning, and welcome to Fulcrum Therapeutics Third Quarter 2022 ...

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, 2022 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-38978 FULCRUM THERAPEUTICS, INC. (Exact name of registrant as specified in its charter) Delaware 47-4839948 (State or other j ...

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, 2022 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-38978 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 ended Q1 2022 with $195.1 million in cash, cash equivalents, and marketable securities, which is expected to support operations into 2024 [32] - Collaboration revenue for Q1 2022 was $2.6 million, down from $4.8 million in Q1 2021, primarily due to reduced research services [33] - Research and development expenses increased to $17.8 million in Q1 2022 from $16.3 million in Q1 2021, while general and administrative expenses rose to $10.8 million from $5.5 million in the same period [34] - The net loss for Q1 2022 was $28.9 million, compared to a net loss of $17 million in Q1 2021 [35] Business Line Data and Key Metrics Changes - The company is advancing two clinical-stage assets: FTX-6058 for sickle cell disease and losmapimod for FSHD, with significant progress reported in Q1 2022 [9][10] - FTX-6058 is positioned as the only oral HbF inducer in development, with initial data expected to be shared at the European Hematology Association Congress [10][11] - Losmapimod is set to begin dosing patients in the Phase 3 REACH trial, which is the first-ever Phase 3 trial for FSHD [16] Market Data and Key Metrics Changes - Sickle cell disease affects an estimated 100,000 people in the U.S. and millions worldwide, highlighting a significant unmet medical need [26] - The company aims to address the unmet medical need for better treatment options for sickle cell disease through FTX-6058, which has shown potential for broad clinical benefits [27] Company Strategy and Development Direction - The company is focused on treating the root cause of rare genetic diseases and aims to establish itself as a leading rare disease company [23] - The strategic focus includes advancing clinical programs while leveraging the FulcrumSeek product engine to build out the pipeline [22] - The anticipated timing for the initiation of trials in non-sickle cell hemoglobinopathies has been shifted to the second half of the year to prioritize sickle cell disease [15] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the potential of FTX-6058 and losmapimod to provide transformative therapies for patients with significant unmet needs [30] - The company is optimistic about the upcoming data disclosures and the potential for these therapies to become standard of care [50] Other Important Information - The REACH trial for losmapimod is designed to evaluate its efficacy over a 48-week treatment period, with a primary endpoint of reachable workspace [16] - The company plans to nominate its next development candidate by the end of 2022 and submit its fourth IND by the end of Q1 2023 [22] Q&A Session Summary Question: Enrollment update for the first cohort of the 6058 Phase 1b trial - Management indicated that initial data from the first cohort would be shared at the upcoming EHA, with a focus on the primary analysis at one month [44] Question: Expectations for data at EHA - Management emphasized the goal of achieving a 5% to 10% absolute increase in HbF, which would be transformative for patients [50] Question: Enrollment expectations for the REACH trial - Management expressed confidence in a reasonable enrollment timeline for the REACH trial, leveraging previous experience from the ReDUX4 trial [61] Question: Data expectations for 6058 at EHA - Initial data from the 6 mg cohort will be presented, with treatment duration up to three months, aiming to show meaningful increases in HbF [66] Question: Confidence in starting pivotal trials for 6058 - Management stated that robust HbF increases would support transitioning into a registrational trial as early as possible in 2023 [80]