[PART I. FINANCIAL INFORMATION](index=7&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) [Financial Statements](index=7&type=section&id=Item%201.%20Financial%20Statements) This section presents unaudited consolidated financial statements for Q1 2025, reporting a net loss of **$17.7 million** and an accumulated deficit of **$537.1 million** Consolidated Balance Sheet Highlights (Unaudited, in thousands) | Account | March 31, 2025 | December 31, 2024 | | :--- | :--- | :--- | | Cash and cash equivalents | $42,178 | $58,212 | | Marketable securities | $184,425 | $182,809 | | **Total Assets** | **$242,649** | **$260,718** | | Total current liabilities | $8,100 | $11,044 | | **Total Liabilities** | **$14,163** | **$17,684** | | Accumulated deficit | $(537,053) | $(519,398) | | **Total Stockholders' Equity** | **$228,486** | **$243,034** | Consolidated Statements of Operations (Unaudited, in thousands, except per share data) | Metric | Three Months Ended Mar 31, 2025 | Three Months Ended Mar 31, 2024 | | :--- | :--- | :--- | | Research and development | $13,404 | $19,773 | | General and administrative | $6,999 | $10,061 | | **Loss from operations** | **$(20,403)** | **$(29,834)** | | **Net loss** | **$(17,655)** | **$(26,870)** | | Net loss per share, basic and diluted | $(0.28) | $(0.43) | Consolidated Statements of Cash Flows (Unaudited, in thousands) | Cash Flow Activity | Three Months Ended Mar 31, 2025 | Three Months Ended Mar 31, 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | $(15,257) | $(25,284) | | Net cash (used in) provided by investing activities | $(777) | $38,128 | | Net cash provided by financing activities | $0 | $1,651 | - The company expects its existing cash, cash equivalents, and marketable securities to be sufficient to fund operating expenses and capital expenditure requirements for at least 12 months from the date of the financial statements[33](index=33&type=chunk) - The collaboration agreement with Sanofi for losmapimod was terminated for convenience by Sanofi, effective April 17, 2025, following the discontinuation of the drug's development. The company is not entitled to any further payments[66](index=66&type=chunk)[67](index=67&type=chunk) - MyoKardia elected to terminate its collaboration and license agreement for convenience, with the termination becoming effective on June 26, 2025. The company will not be entitled to further milestone or royalty payments[76](index=76&type=chunk)[77](index=77&type=chunk) - In September 2024, the company implemented a restructuring plan to focus on pociredir and DBA, reducing its workforce from 80 to 51 full-time employees[94](index=94&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=31&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses the company's focus on developing pociredir for SCD, with Q1 2025 net loss decreasing to **$17.7 million** due to reduced R&D expenses - The company is a clinical-stage biopharmaceutical company focused on developing small molecules for genetically defined rare diseases, with its lead candidate, pociredir, being developed for Sickle Cell Disease (SCD)[101](index=101&type=chunk) - Clinical trial updates for pociredir: The Phase 1b trial was reinitiated in Q4 2023. The 12 mg dose cohort is fully enrolled, with data expected in early Q3 2025. The 20 mg dose cohort has been initiated, with data expected by the end of 2025[102](index=102&type=chunk) - The company plans to submit an Investigational New Drug (IND) application for Diamond-Blackfan Anemia (DBA) during the fourth quarter of 2025[103](index=103&type=chunk) Comparison of Operating Results (in thousands) | Item | Three Months Ended Mar 31, 2025 | Three Months Ended Mar 31, 2024 | Change ($) | | :--- | :--- | :--- | :--- | | Research and development | $13,404 | $19,773 | $(6,369) | | General and administrative | $6,999 | $10,061 | $(3,062) | | **Total operating expenses** | **$20,403** | **$29,834** | **$(9,431)** | | **Net loss** | **$(17,655)** | **$(26,870)** | **$9,215** | - R&D expenses decreased by **$6.4 million**, primarily due to a **$4.9 million** decrease in external costs from the suspension of the losmapimod program and a **$1.7 million** decrease in employee compensation costs from lower headcount[127](index=127&type=chunk)[129](index=129&type=chunk) - G&A expenses decreased by **$3.1 million**, mainly due to a **$1.4 million** decrease in employee compensation costs and a **$1.1 million** decrease in professional services costs[130](index=130&type=chunk)[135](index=135&type=chunk) - As of March 31, 2025, the company had **$226.6 million** in cash, cash equivalents, and marketable securities, which is expected to fund operations into at least 2027[108](index=108&type=chunk)[131](index=131&type=chunk)[139](index=139&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=41&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) As a smaller reporting company, the company is not required to provide quantitative and qualitative disclosures about market risk - As a smaller reporting company, Fulcrum Therapeutics is not required to provide quantitative and qualitative disclosures about market risk[146](index=146&type=chunk) [Controls and Procedures](index=42&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded disclosure controls and procedures were effective as of March 31, 2025, with no material changes in internal control - The CEO and CFO concluded that the company's disclosure controls and procedures were effective as of March 31, 2025[147](index=147&type=chunk) - No changes in internal control over financial reporting occurred during the quarter that materially affected, or are reasonably likely to materially affect, the company's internal controls[148](index=148&type=chunk) [PART II. OTHER INFORMATION](index=43&type=section&id=PART%20II.%20OTHER%20INFORMATION) [Legal Proceedings](index=43&type=section&id=Item%201.%20Legal%20Proceedings) A securities class action lawsuit related to the pociredir clinical hold was dismissed in full by the court on March 27, 2025 - A securities class action lawsuit filed against the company and its officers, related to the clinical hold on pociredir, was dismissed in full by the court on March 27, 2025[151](index=151&type=chunk)[90](index=90&type=chunk) [Risk Factors](index=43&type=page&id=Item%201A.%20Risk%20Factors) This section details significant risks including financial losses, funding needs, reliance on pociredir, clinical development uncertainties, competition, and intellectual property challenges [Risks Related to Financial Position and Need for Additional Capital](index=43&type=section&id=Risks%20Related%20to%20our%20Financial%20Position%20and%20Need%20for%20Additional%20Capital) The company faces significant historical losses and requires substantial additional funding to advance product candidates, with current cash expected to fund operations into at least 2027 - The company has incurred significant losses since inception, with a net loss of **$17.7 million** for Q1 2025 and an accumulated deficit of **$537.1 million** as of March 31, 2025[153](index=153&type=chunk) - Substantial additional funding is required to continue clinical development of pociredir and other candidates. Failure to raise capital could force delays, reductions, or elimination of programs[156](index=156&type=chunk) - The company's cash, cash equivalents, and marketable securities of **$226.6 million** are expected to fund operations into at least 2027, but this estimate is based on assumptions that could prove wrong[160](index=160&type=chunk) [Risks Related to Discovery and Development of Product Candidates](index=51&type=section&id=Risks%20Related%20to%20the%20Discovery%20and%20Development%20of%20our%20Product%20Candidates) The company is early in development with one clinical candidate, pociredir, facing lengthy, expensive, and uncertain clinical processes and enrollment challenges - The company is early in its development efforts with only one product candidate, pociredir, in active clinical development after suspending the losmapimod program[178](index=178&type=chunk) - Clinical drug development is a lengthy, expensive process with an uncertain outcome. The Phase 3 trial for losmapimod did not meet its primary endpoint, leading to its discontinuation[183](index=183&type=chunk)[185](index=185&type=chunk) - In February 2023, the FDA placed a clinical hold on the IND for pociredir due to hematological malignancies in nonclinical studies. Although the hold was lifted in August 2023, future adverse events could halt or delay development[196](index=196&type=chunk) - The company may experience difficulties enrolling patients in clinical trials for rare diseases, which could delay regulatory approvals. The revised protocol for the pociredir trial has more stringent criteria, making enrollment challenging[190](index=190&type=chunk) [Risks Related to Commercialization of Product Candidates](index=62&type=section&id=Risks%20Related%20to%20the%20Commercialization%20of%20our%20Product%20Candidates) Product candidates may not achieve market acceptance due to substantial competition, lack of sales infrastructure, reliance on CMOs, and small patient populations - The company faces substantial competition from major pharmaceutical companies and recently approved gene therapies for SCD, such as CASGEVY and LYFGENIA[211](index=211&type=chunk)[213](index=213&type=chunk) - The company has no sales, marketing, or distribution infrastructure and establishing these capabilities is expensive and risky[206](index=206&type=chunk)[207](index=207&type=chunk) - The company relies on contract manufacturing organizations (CMOs) for clinical and potential commercial supply, which creates risks related to supply delays, quality issues, and regulatory compliance[218](index=218&type=chunk)[219](index=219&type=chunk) - The addressable patient populations for the company's target rare diseases are small, requiring it to capture a significant market share to achieve profitability[216](index=216&type=chunk) [Risks Related to Dependence on Third Parties](index=68&type=section&id=Risks%20Related%20to%20our%20Dependence%20on%20Third%20Parties) The company relies on third-party CROs and collaborators, which reduces control and introduces risks of performance failures, delays, and potential conflicts of interest - The company relies on third-party contract research organizations (CROs) to conduct its clinical trials, which reduces control over these activities and may lead to delays if these parties do not perform satisfactorily[237](index=237&type=chunk)[239](index=239&type=chunk) - Future collaborations with third parties for development or commercialization are subject to risks, including collaborators not performing as expected, delaying trials, or terminating agreements for convenience[241](index=241&type=chunk)[244](index=244&type=chunk) - If the company cannot establish or maintain successful collaborations, it may have to alter its development plans, which could require additional capital and expertise that may not be available[247](index=247&type=chunk)[249](index=249&type=chunk) [Risks Related to Intellectual Property](index=71&type=section&id=Risks%20Related%20to%20our%20Intellectual%20Property) Success depends on obtaining and enforcing patent protection, an uncertain and expensive process, with risks of litigation, license non-compliance, and trade secret breaches - The company's success depends on its ability to obtain and maintain patent protection for its technology and product candidates, which is an expensive and uncertain process[250](index=250&type=chunk)[251](index=251&type=chunk) - The company may be subject to litigation or other proceedings challenging its patent rights, which could result in invalidation or narrowing of its patents[254](index=254&type=chunk)[268](index=268&type=chunk) - Failure to comply with obligations in license agreements could lead to the loss of important intellectual property rights[282](index=282&type=chunk) - The company relies on trade secrets and confidentiality agreements, which may be breached, and protecting them can be difficult and costly[295](index=295&type=chunk) [Risks Related to Regulatory Approval and Legal Compliance](index=87&type=section&id=Risks%20Related%20to%20Regulatory%20Approval%20of%20our%20Product%20Candidates%20and%20Other%20Legal%20Compliance%20Matters) Marketing approval is expensive and uncertain, with ongoing regulatory oversight and complex healthcare laws posing risks of delays, rejections, and significant penalties - The marketing approval process is expensive, time-consuming, and uncertain, and the company may not obtain approvals for its product candidates[298](index=298&type=chunk)[301](index=301&type=chunk) - Even if orphan drug designation is obtained, it may not prevent competitors from getting approval for different drugs for the same condition or for the same drug if it is proven clinically superior[308](index=308&type=chunk)[311](index=311&type=chunk) - The company's relationships with healthcare providers and payors are subject to fraud and abuse, false claims, and other healthcare laws, with violations potentially leading to criminal sanctions and civil penalties[328](index=328&type=chunk)[330](index=330&type=chunk) - Compliance with global privacy and data security laws (e.g., GDPR, CCPA) is costly and complex, and failure to comply could result in significant fines and penalties[331](index=331&type=chunk)[336](index=336&type=chunk) [Risks Related to Employee Matters and Managing Growth](index=100&type=section&id=Risks%20Related%20to%20Employee%20Matters%20and%20Managing%20Growth) Success depends on retaining key personnel amid intense competition and recent transitions, with future growth requiring significant managerial and financial resources - The company is highly dependent on its key executives and scientific teams and has experienced recent transitions in several leadership roles, including CEO, CFO, and CSO[353](index=353&type=chunk)[354](index=354&type=chunk) - Competition for qualified personnel is intense, and the company's recent workforce reductions may make it a less attractive employer[354](index=354&type=chunk) - Future growth in operations would require significant expansion of managerial, operational, and financial systems, which may be difficult to manage effectively[356](index=356&type=chunk) [Risks Related to Common Stock](index=102&type=section&id=Risks%20Related%20to%20our%20Common%20Stock) The company's stock price is volatile, with significant control by principal stockholders, anti-takeover provisions, and no anticipated cash dividends - As of April 24, 2025, executive officers, directors, and principal stockholders beneficially owned approximately **72.6%** of the company's capital stock, giving them significant control[357](index=357&type=chunk) - The company's stock price has been and is likely to continue to be volatile due to factors such as clinical trial results, regulatory developments, and market conditions[363](index=363&type=chunk) - The company is a "smaller reporting company" and can rely on scaled disclosure requirements, which may make its common stock less attractive to some investors[369](index=369&type=chunk) - The company does not anticipate paying cash dividends in the foreseeable future; capital appreciation will be the sole source of gain for stockholders[374](index=374&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=106&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) This item is not applicable for the reporting period - Not applicable[377](index=377&type=chunk) [Defaults Upon Senior Securities](index=106&type=section&id=Item%203.%20Defaults%20Upon%20Senior%20Securities) This item is not applicable for the reporting period - Not applicable[378](index=378&type=chunk) [Mine Safety Disclosures](index=106&type=section&id=Item%204.%20Mine%20Safety%20Disclosures) This item is not applicable for the reporting period - Not applicable[379](index=379&type=chunk) [Other Information](index=106&type=section&id=Item%205.%20Other%20Information) There is no other information to report for this period - None[380](index=380&type=chunk) [Exhibits](index=108&type=section&id=Item%206.%20Exhibits) This section lists exhibits filed with the Form 10-Q, including CEO and CFO certifications and Inline XBRL documents - Exhibits filed include CEO and CFO certifications pursuant to Sarbanes-Oxley Act Sections 302 and 906[382](index=382&type=chunk) - Inline XBRL documents are included as exhibits for interactive data filing[382](index=382&type=chunk)

Core Insights - Fulcrum Therapeutics has completed enrollment in the 12 mg dose cohort of the Phase 1b PIONEER trial for pociredir in sickle cell disease (SCD) and has initiated the 20 mg dose cohort [1][6] - The company plans to share data from the 12 mg dose cohort in early Q3 2025 and from the 20 mg dose cohort by the end of 2025 [2][5] Recent Business Highlights - The PIONEER trial shows strong engagement with 16 patients enrolled in the 12 mg cohort and adherence rates exceeding 90% [6] - Two abstracts related to pociredir have been accepted for presentation at the 2025 European Hematology Association Congress [6] - The company is advancing its program for inherited aplastic anemias and plans to submit an IND for Diamond-Blackfan anemia in Q4 2025 [6] Financial Results - As of March 31, 2025, Fulcrum had $226.6 million in cash, cash equivalents, and marketable securities, down from $241.0 million at the end of 2024 [5][14] - Research and development expenses for Q1 2025 were $13.4 million, a decrease from $19.8 million in Q1 2024, primarily due to the discontinuation of the losmapimod program [5][15] - General and administrative expenses decreased to $7.0 million in Q1 2025 from $10.1 million in Q1 2024, attributed to reduced employee compensation costs [5][15] - The net loss for Q1 2025 was $17.7 million, compared to a net loss of $26.9 million in Q1 2024 [5][15] Cash Position and Guidance - Fulcrum expects its current cash position to fund operations into at least 2027 [7]

Contact: CAMBRIDGE, Mass., April 24, 2025 (GLOBE NEWSWIRE) -- Fulcrum Therapeutics, Inc. (the "Company") (Nasdaq: FULC), a clinical-stage biopharmaceutical company focused on developing small molecules to improve the lives of patients with genetically defined rare diseases, today announced that its first quarter 2025 financial results will be released on Thursday, May 1, 2025, before the U.S. financial markets open. Management will host a conference call and webcast at 8:00 a.m. ET to discuss the results an ...

Core Viewpoint - Fulcrum Therapeutics, Inc. (FULC) shows significant upside potential with a mean price target of $16.11, indicating a 71% increase from the current trading price of $9.42 [1] Price Target Analysis - The mean estimate consists of nine short-term price targets with a standard deviation of $5.78, suggesting variability in analyst predictions [2] - The lowest estimate is $5, indicating a potential decline of 46.9%, while the highest estimate is $26, suggesting a potential increase of 176% [2] - A low standard deviation indicates strong agreement among analysts regarding the stock's price movement direction [7] Analyst Sentiment - Analysts exhibit growing optimism regarding FULC's earnings prospects, as evidenced by a positive trend in earnings estimate revisions [9] - The Zacks Consensus Estimate for the current year has increased by 61.7% over the past month, with five estimates rising and no negative revisions [10] - FULC holds a Zacks Rank 1 (Strong Buy), placing it in the top 5% of over 4,000 ranked stocks based on earnings estimates [11] Caution on Price Targets - While price targets are commonly referenced by investors, they can often mislead and should not be the sole basis for investment decisions [5][8] - Analysts may set overly optimistic price targets due to business incentives, which can inflate expectations [6]

Core Viewpoint - Fulcrum Therapeutics, Inc. (FULC) shows potential for significant upside, with a mean price target of $13.38 indicating a 69.2% increase from the current price of $7.91 [1] Price Targets and Analyst Consensus - The average price target consists of eight estimates ranging from a low of $5 to a high of $19, with a standard deviation of $5.13, indicating variability in analyst predictions [2] - The lowest estimate suggests a decline of 36.8%, while the highest indicates a potential upside of 140.2% [2] - A low standard deviation signifies greater agreement among analysts regarding price movement, which can be a starting point for further research [8] Earnings Estimates and Analyst Optimism - Analysts are optimistic about FULC's earnings prospects, as indicated by a trend of upward revisions in earnings estimates, which historically correlates with stock price movements [10] - Over the past 30 days, the Zacks Consensus Estimate for the current year has increased by 0.5%, with one estimate moving higher and no negative revisions [11] - FULC holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimate factors, suggesting strong potential for near-term upside [12]

Financial Performance - As of March 31, 2024, Fulcrum Therapeutics reported total assets of $232.6 million, a decrease from $257.7 million as of December 31, 2023, representing a decline of approximately 9.7%[17] - For the three months ended March 31, 2024, Fulcrum reported a net loss of $26.9 million, compared to a net loss of $24.8 million for the same period in 2023, reflecting an increase in loss of approximately 8.5%[19] - Total operating expenses for the first quarter of 2024 were $29.8 million, up from $28.2 million in the same quarter of 2023, marking an increase of about 5.6%[19] - The company had no collaboration revenue for the first quarter of 2024, a decrease from $295,000 in the same period of 2023[19] - As of March 31, 2024, the accumulated deficit stood at $536.5 million, up from $509.7 million at the end of 2023, indicating an increase of approximately 5.3%[17] - Fulcrum's total stockholders' equity decreased to $213.6 million as of March 31, 2024, from $235.2 million at the end of 2023, a decline of about 9.2%[17] - The company expects to incur substantial operating losses and negative operating cash flows for the foreseeable future due to ongoing research and development activities[151] - The net loss for the year ended December 31, 2023, was $97.3 million, and for the three months ended March 31, 2024, it was $26.9 million, with an accumulated deficit of $536.5 million as of March 31, 2024[167] Cash and Liquidity - The company had cash and cash equivalents of $39.9 million as of March 31, 2024, up from $25.6 million at the end of 2023, indicating a significant increase of 56.7%[17] - The Company expects its cash, cash equivalents, and marketable securities to be sufficient to fund operating expenses and capital expenditures for at least 12 months[34] - As of March 31, 2024, total cash equivalents and marketable securities amounted to $213.3 million, a decrease from $236.2 million as of December 31, 2023[40][43] - The company had cash, cash equivalents, and marketable securities totaling $213.3 million as of March 31, 2024, which, along with the expected $80.0 million payment, is projected to fund operations into 2027[175] - The company anticipates continued reliance on additional financing due to the lengthy and uncertain process of product development, which may not yield commercial success for several years[176] - The company may need to raise additional capital through equity offerings, debt financings, or collaboration arrangements, which could dilute existing stockholders' interests[155] Research and Development - Research and development expenses increased by $3.1 million from $16.7 million in Q1 2023 to $19.8 million in Q1 2024, primarily due to increased external research and development costs[140] - The company plans to continue clinical development of losmapimod and pociredir, advance clinical-stage candidates, and seek regulatory approvals for successful candidates[119] - The Phase 3 clinical trial of losmapimod for the treatment of FSHD completed enrollment in September 2023[174] - The FDA imposed a clinical hold on the IND application for pociredir, which was lifted in August 2023[173] - The company is still in the early stages of development for its product candidates and has not completed development of any product candidates[167] - The company has two product candidates in clinical development, with high risks of failure and no completed pivotal clinical trials[201] Collaboration and Revenue - The Company achieved a $2.5 million specified preclinical milestone under the MyoKardia Collaboration Agreement[68] - The Company received a non-refundable upfront payment of $10.0 million from MyoKardia as part of the collaboration agreement[67] - The Company recognized no collaboration revenue associated with the MyoKardia Collaboration Agreement for the three months ended March 31, 2024, compared to $0.3 million for the same period in 2023[81] - The company expects to recognize revenue from the Sanofi Agreement associated with the $80.0 million upfront payment and potential future milestones and royalties[125] - The company has entered into a collaboration and license agreement with Genzyme Corporation (Sanofi) to commercialize losmapimod outside the U.S., sharing global development costs[110] Stock and Equity - The Company has reserved 17,591,700 shares of common stock for future issuance as of March 31, 2024, an increase from 15,388,773 shares as of December 31, 2023[52] - The Company had outstanding stock options totaling 12,289,352 as of March 31, 2024, compared to 6,876,679 for the same period in 2023[99] - The total stock-based compensation expense for the three months ended March 31, 2024, was $3.916 million, compared to $4.253 million for the same period in 2023[62] - The aggregate intrinsic value of stock options outstanding at March 31, 2024, was $35.107 million[58] - The weighted average grant date fair value of stock options granted in the three months ended March 31, 2024, was $6.23 per share[59] Risks and Challenges - The company has incurred significant operating losses since inception and expects to continue incurring losses over the next several years[167] - Clinical trials are lengthy and expensive, with uncertain outcomes, and the company may face delays or additional costs[203][207] - Serious adverse events or unacceptable side effects during product candidate development may necessitate abandonment or limitation of certain candidates[213] - The commercial potential of the pociredir program may be materially and negatively impacted even if regulatory approval is received[215] - The company may need to conduct additional clinical trials or testing, which could increase development costs and delay marketing approvals[207][209] Legal and Regulatory - The company intends to defend against a class action lawsuit alleging misleading statements related to the FDA's clinical hold on pociredir[165] - The FDA placed the IND for pociredir on clinical hold in February 2023 due to concerns over hematological malignancies, which was lifted in August 2023[214] - Regulatory authorities have substantial discretion in the approval process, making it difficult to predict the time and cost of product candidate development[200]

― Sanofi receives exclusive rights to commercialize losmapimod in all territories outside the U.S.; Fulcrum retains full U.S. commercialization rights ― ― Fulcrum will receive an upfront payment of $80.0 million, and is eligible to receive $975.0 million in potential milestones, plus royalties on ex-U.S. product sales; parties will share future global development costs 50:50 ― ― Conference call and webcast scheduled for 8:00 a.m. ET today to discuss the collaboration and other recent corporate developments, ...

[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=

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 ...