4D Molecular Therapeutics(FDMT) - 2023 Q3 - Quarterly Report

PART I. FINANCIAL INFORMATION Item 1. Condensed Unaudited Financial Statements Unaudited condensed financial statements detail increased cash from public offering and license revenue, reducing operating losses Condensed Balance Sheet Highlights (Unaudited) | Account | Sep 30, 2023 ($ in thousands) | Dec 31, 2022 ($ in thousands) | | :--- | :--- | :--- | | Assets | | | | Cash and cash equivalents | 275,678 | 52,351 | | Marketable securities | 43,986 | 166,111 | | Total current assets | 328,243 | 220,511 | | Total assets | 361,613 | 261,846 | | Liabilities & Equity | | | | Total current liabilities | 18,163 | 15,731 | | Total liabilities | 31,679 | 30,509 | | Total stockholders' equity | 329,934 | 231,337 | | Accumulated deficit | (383,044) | (314,490) | Condensed Statements of Operations Highlights (Unaudited) | Metric ($ in thousands) | Three Months Ended Sep 30, 2023 | Three Months Ended Sep 30, 2022 | Nine Months Ended Sep 30, 2023 | Nine Months Ended Sep 30, 2022 | | :--- | :--- | :--- | :--- | :--- | | Collaboration and license revenue | 20,204 | 500 | 20,742 | 1,882 | | Research and development | 25,066 | 18,940 | 71,068 | 58,753 | | General and administrative | 9,112 | 8,055 | 25,889 | 24,441 | | Loss from operations | (13,974) | (26,495) | (76,215) | (81,312) | | Net loss | (10,256) | (25,691) | (68,554) | (80,115) | | Net loss per share, basic and diluted | $(0.24) | $(0.79) | $(1.81) | $(2.48) | Condensed Statements of Cash Flows Highlights (Unaudited, Nine Months Ended) | Cash Flow Activity ($ in thousands) | Nine Months Ended Sep 30, 2023 | Nine Months Ended Sep 30, 2022 | | :--- | :--- | :--- | | Net cash used in operating activities | (51,142) | (64,471) | | Net cash provided by investing activities | 122,399 | 18,729 | | Net cash provided by financing activities | 152,070 | 1,039 | | Net increase (decrease) in cash | 223,327 | (44,703) | | Cash and cash equivalents, end of period | 275,678 | 108,298 | Notes to Unaudited Condensed Financial Statements Notes detail financial position, highlighting public offering, Astellas licensing, and 4D-175 program acquisition - In May 2023, the company completed a public offering, selling 8,625,000 shares at $16.00 per share, resulting in net proceeds of $129.2 million[34] - The company believes its cash, cash equivalents, and marketable securities as of September 30, 2023, are sufficient to fund planned operations for at least one year, despite having an accumulated deficit of $383.0 million and expecting continued operating losses[35] - In July 2023, the company entered into a licensing agreement with Astellas Gene Therapies, Inc. (AGT), receiving an upfront payment of $20 million. The company may receive up to $942.5 million in potential future option fees and milestones[83] - On April 21, 2023, the company acquired worldwide rights to short-form human complement factor H (sCFH) from Aevitas Therapeutics for its 4D-175 program. The agreement includes potential future milestone payments of up to ~$144 million to Aevitas and ~$42 million to the University of Pennsylvania, plus low single-digit royalties[85][86] Collaboration and License Revenue Breakdown (in thousands) | Partner | Three Months Ended Sep 30, 2023 | Nine Months Ended Sep 30, 2023 | | :--- | :--- | :--- | | uniQure | $41 | $566 | | CFF | $155 | $168 | | Astellas | $20,008 | $20,008 | | Total | $20,204 | $20,742 | Item 2. Management's Discussion and Analysis of Financial Condition and Results of Operations Management discusses financial performance, highlighting significant Astellas revenue, increased R&D, and bolstered liquidity - The company is a clinical-stage genetic medicines company with five product candidates in clinical trials: 4D-150 (wet AMD & DME), 4D-710 (cystic fibrosis), 4D-310 (Fabry disease), 4D-125 (XLRP), and 4D-110 (choroideremia)[131] - Recent financing activities include a May 2023 public offering with $129.2 million in net proceeds and a July 2023 licensing agreement with Astellas providing a $20.0 million upfront payment[133][134] - The company believes its existing cash, cash equivalents, and marketable securities will fund planned operations for at least one year from the report's issuance date[170] Comparison of Operating Results (in thousands) | Metric | Three Months Ended Sep 30, 2023 | Three Months Ended Sep 30, 2022 | $ Change | % Change | | :--- | :--- | :--- | :--- | :--- | | Collaboration and license revenue | $20,204 | $500 | $19,704 | 3941% | | Research and development | $25,066 | $18,940 | $6,126 | 32% | | General and administrative | $9,112 | $8,055 | $1,057 | 13% | | Net loss | $(10,256) | $(25,691) | $15,435 | (60)% | - The increase in R&D expenses for the three and nine months ended September 30, 2023 was primarily driven by higher clinical trial costs, mainly for the 4D-150 program, and increased payroll and facilities expenses[158] Item 3. Quantitative and Qualitative Disclosures About Market Risk Market risk disclosure focuses on interest rate sensitivity for cash and marketable securities, with no material impact from rate changes - The company's primary market risk is interest rate sensitivity. As of September 30, 2023, it had $319.7 million in cash, cash equivalents, and marketable securities[194] - Due to the short-to-medium-term maturities of its investments, the company believes an immediate 10% change in interest rates would not have a material effect on the fair value of its holdings[194] - The company does not believe that inflation or interest rate changes have had a significant impact on its results of operations for the periods presented[195] Item 4. Controls and Procedures Management concluded disclosure controls were effective, with no material changes to internal controls during the quarter - Management, including the CEO and CFO, concluded that the company's disclosure controls and procedures were effective at the reasonable assurance level as of September 30, 2023[198] - There were no material changes to the company's internal control over financial reporting during the third quarter of 2023[199] PART II. OTHER INFORMATION Item 1. Legal Proceedings The company reports no material legal proceedings outstanding as of September 30, 2023 - There are no material legal proceedings outstanding at September 30, 2023[102][201] Item 1A. Risk Factors This section outlines significant risks: limited operating history, recurring losses, capital needs, and gene therapy development challenges Risks Related to Operating History, Financial Condition and Capital Requirements Risks include limited operating history, recurring net losses, and the need for substantial additional capital to fund operations - The company is in the early stages of development with a limited operating history and no products approved for sale, making it difficult to evaluate its future success[204][206] - The company has a history of recurring net losses, with an accumulated deficit of $383.0 million as of September 30, 2023, and expects to incur significant net losses for the foreseeable future[207] - Substantial additional capital will be required to finance operations. The company believes its current cash will fund operations for at least one year, but failure to raise more capital when needed could force it to delay, reduce, or eliminate development programs[211][214] Risks Related to Research, Discovery, Development and Commercialization Risks include novel AAV gene therapy development, clinical trial failures, and intense competition from larger pharmaceutical companies - All product candidates are based on novel AAV genetic medicine technology, which has limited regulatory and clinical experience, making the time and cost of development and approval difficult to predict[224] - The company faces substantial competition from large pharmaceutical and biotechnology companies like Regeneron, Roche, Sanofi, Takeda, and Vertex, which have greater financial resources and approved products in the market[277][278] - Clinical trials may be substantially delayed or fail due to numerous factors, including difficulty enrolling patients for rare diseases, manufacturing issues, imposition of clinical holds, and inconclusive results[248][249] - Public perception of gene therapy, potential adverse events in trials, and regulatory scrutiny could negatively impact development, commercialization, and the company's ability to raise capital[233][235] Risks Related to Manufacturing Manufacturing risks stem from gene therapy complexity, reliance on third parties, and challenges in building the company's own facility - Gene therapies are complex and difficult to manufacture, and the company could experience production problems, such as lot failures or recalls, that result in delays to its development programs[287] - The company relies on third parties, such as Catalent, for manufacturing, which reduces control over the process and exposes it to risks of non-performance and non-compliance with cGMP standards[287][297] - The company is building out its own cGMP manufacturing facility, but may face delays in production and cannot guarantee it will produce sufficient quantities to support planned clinical trials in a timely manner[294] - The manufacturing process is vulnerable to contamination and shortages of key raw materials, such as plasmids, which could disrupt production and harm the business[299][300] Risks Related to Regulatory Approval and Other Legal Compliance Matters Extensive regulatory risks include lengthy approval processes, healthcare legislation impact, and compliance with complex laws - The regulatory approval process for product candidates is lengthy, expensive, and unpredictable, and the company may never obtain approval to generate product revenue[302] - The company has received Fast Track designation for 4D-310 and 4D-125, and Orphan Drug designation for 4D-110, 4D-125, and 4D-310, but these designations do not guarantee a faster review or increase the likelihood of approval[316][318] - Enacted and future healthcare legislation, such as the Inflation Reduction Act (IRA), may increase the difficulty and cost of obtaining marketing approval and could negatively affect product pricing[324][328] - Business operations are subject to extensive healthcare laws, including anti-kickback and false claims statutes, which could expose the company to significant penalties if violations occur[342][343] Risks Related to Reliance on Third Parties Reliance on third parties for clinical trials, NHP supply, and collaborations introduces significant operational risks for the company - The company relies on third parties like CROs to conduct clinical trials and research, and any failure by these parties to perform their duties could delay or prevent marketing approval[348][350] - A primary supplier of non-human primates (NHPs), Charles River Laboratories, received a DOJ subpoena regarding its NHP importation from Cambodia. An inability to secure an adequate supply of NHPs could delay preclinical development and increase costs[352] - Future collaborations with third parties for research, development, and commercialization are subject to numerous risks, including collaborators having discretion over resources, potential disputes, and termination of agreements[353][354] Risks Related to Intellectual Property Intellectual property risks include challenges in obtaining and maintaining patent protection, license reliance, and potential infringement litigation - Commercial success depends on obtaining and maintaining patent and trade secret protection, but there is no assurance that patent applications will issue or that issued patents will provide sufficient protection[359] - The company relies on exclusive license agreements with U.C. Berkeley for its 4D-710 and 4D-725 product candidates and with the University of Pennsylvania for its 4D-175 product candidate. Loss of these licenses could seriously harm the business[376] - The company may be subject to litigation or other proceedings related to intellectual property, which could be expensive, time-consuming, and could prevent the sale of its products[456] - Changes in U.S. or foreign patent law, such as the Leahy-Smith Act, could diminish the value of patents and impair the ability to protect product candidates[477][478] Item 2. Unregistered Sales of Equity Securities and Use of Proceeds There were no unregistered sales of equity securities, and no material change in the planned use of IPO proceeds - The company had no unregistered sales of equity securities in the period[485] - There has been no material change in the planned use of proceeds from the company's December 2020 IPO, which generated net proceeds of $204.7 million[486][488] Item 3. Default Upon Senior Securities The company reports no defaults upon senior securities - None[489] Item 4. Mine Safety Disclosures This item is Not applicable to the company's operations - Not applicable[490] Item 5. Other Information The company reports no other information to disclose for this period - None[491] Item 6. Exhibits Exhibits filed with the Quarterly Report on Form 10-Q include the Astellas Gene Therapies License Agreement - A key exhibit filed with this report is the License Agreement between the Registrant and Astellas Gene Therapies, Inc., dated July 5, 2023[493]