Dyne Therapeutics(DYN) - 2024 Q3 - Quarterly Results

Corporate Update and Program Highlights Dyne Therapeutics reported Q3 2024 results, highlighting clinical trial progress and a strong financial position Key Announcements and CEO Commentary Dyne Therapeutics announced Q3 2024 results, highlighting FDA IND clearance for DYNE-101 and registrational cohort enrollment for DYNE-251 - FDA cleared the Investigational New Drug (IND) application for DYNE-101 for myotonic dystrophy type 1 (DM1)[1][2][3] - New clinical data from the DYNE-101 ACHIEVE trial is expected in early January 2025[1][2][4] - Dyne is enrolling patients in a registrational cohort at 20 mg/kg for the DYNE-251 DELIVER trial in Duchenne muscular dystrophy (DMD)[1][2][5] Clinical Program Updates This section details the progress of Dyne's clinical programs, including the ACHIEVE trial for DM1 and the DELIVER trial for DMD ACHIEVE Trial of DYNE-101 in DM1 The FDA cleared the IND for DYNE-101, with the ACHIEVE trial fully enrolled and new data expected in early January 2025 - The U.S. FDA has cleared the Investigational New Drug (IND) application for DYNE-101[3] - The ACHIEVE trial is fully enrolled through the 6.8 mg/kg Q8W cohort, with 56 participants[3] - New data from the ACHIEVE trial, including safety, tolerability, splicing, vHOT, functional measures, and patient-reported outcomes, is anticipated in early January 2025[4] - Dyne continues to pursue expedited approval pathways globally for DYNE-101 utilizing splicing as a surrogate endpoint[5] DELIVER Trial of DYNE-251 in DMD Dyne reported positive efficacy and safety from the DELIVER trial, initiating enrollment for a 20 mg/kg registrational cohort - In September 2024, Dyne reported positive efficacy data and a favorable safety profile from the ongoing Phase 1/2 global DELIVER trial of DYNE-251 in males with DMD mutations amenable to exon 51 skipping[5] - Based on positive data, Dyne has begun enrolling a 20 mg/kg Q4W registrational cohort of 32 participants as part of the DELIVER trial[5] - The Company continues to pursue expedited approval pathways for DYNE-251, including accelerated approval in the U.S. based on dystrophin as a surrogate endpoint[5] Third Quarter 2024 Financial Results This section presents Dyne Therapeutics' financial performance for Q3 2024, including key metrics and balance sheet data Financial Highlights Dyne Therapeutics reported increased R&D and G&A expenses in Q3 2024, leading to a higher net loss, but maintains a strong cash position Third Quarter 2024 Financial Highlights | Metric | Q3 2024 (Millions) | Q3 2023 (Millions) | Change (YoY) | | :----- | :----------------- | :----------------- | :----------- | | Cash, cash equivalents and marketable securities | $723.7 | N/A | N/A | | R&D Expenses | $92.8 | $55.3 | +67.8% | | G&A Expenses | $12.9 | $7.0 | +84.3% | | Net Loss | $(97.1) | $(60.2) | +61.3% | | Net Loss per Share (basic & diluted) | $(0.96) | $(0.99) | -3.0% | - Cash, cash equivalents and marketable securities were $723.7 million as of September 30, 2024, anticipated to fund operations at least into the second half of 2026[7] Condensed Consolidated Statement of Operations (Unaudited) Operating expenses significantly increased in Q3 2024, resulting in a higher net loss, yet net loss per share decreased due to more shares outstanding Condensed Consolidated Statement of Operations (Unaudited) | Metric (in thousands) | Three Months Ended Sep 30, 2024 | Three Months Ended Sep 30, 2023 | Nine Months Ended Sep 30, 2024 | Nine Months Ended Sep 30, 2023 | | :-------------------- | :------------------------------ | :------------------------------ | :----------------------------- | :----------------------------- | | Research and development | $92,800 | $55,251 | $199,601 | $151,918 | | General and administrative | $12,859 | $7,022 | $47,177 | $22,556 | | Total operating expenses | $105,659 | $62,273 | $246,778 | $174,474 | | Loss from operations | $(105,659) | $(62,273) | $(246,778) | $(174,474) | | Other income (expense), net | $8,534 | $2,063 | $18,902 | $5,175 | | Net loss | $(97,125) | $(60,210) | $(227,876) | $(169,299) | | Net loss per share—basic and diluted | $(0.96) | $(0.99) | $(2.49) | $(2.86) | | Weighted average common shares outstanding | 100,882,042 | 61,109,917 | 91,511,621 | 59,107,795 | Condensed Consolidated Balance Sheet Data (Unaudited) Dyne's balance sheet shows a substantial increase in cash and total assets as of September 30, 2024, compared to December 31, 2023 Condensed Consolidated Balance Sheet Data (Unaudited) | Metric (in thousands) | September 30, 2024 | December 31, 2023 | | :-------------------- | :----------------- | :---------------- | | Cash, cash equivalents and marketable securities | $723,674 | $123,100 | | Other assets | $45,170 | $41,982 | | Total assets | $768,844 | $165,082 | | Liabilities | $63,304 | $73,790 | | Stockholders' equity | $705,540 | $91,292 | | Total liabilities and stockholders' equity | $768,844 | $165,082 | Company Information and Disclosures This section provides an overview of Dyne Therapeutics and important disclosures regarding forward-looking statements About Dyne Therapeutics Dyne Therapeutics is a clinical-stage company developing oligonucleotide therapeutics for genetically driven muscle diseases using its FORCE™ platform - Dyne Therapeutics is a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases[9] - The company utilizes its proprietary FORCE™ platform to develop modern oligonucleotide therapeutics designed to overcome limitations in delivery to muscle tissue[9] - Dyne has a broad pipeline for serious muscle diseases, including clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD), and a preclinical program for facioscapulohumeral muscular dystrophy (FSHD)[9] Forward-Looking Statements This section outlines forward-looking statements regarding Dyne's strategy, operations, and financial resources, noting inherent risks and uncertainties - The press release contains forward-looking statements regarding Dyne's strategy, future operations, prospects, plans, objectives, the potential of its FORCE platform, DYNE-101 and DYNE-251, anticipated timelines for clinical trial data, expedited approval pathways, and sufficiency of cash resources[10] - These statements involve substantial risks and uncertainties, including those inherent in product candidate identification and development, clinical trial outcomes, regulatory interpretations, and financial resource sufficiency[10][11] - Dyne disclaims any obligation to update these forward-looking statements, which represent views as of the date of the press release[11] Contacts This section provides contact information for investor relations and media inquiries for Dyne Therapeutics Contact Information Contact details for investor relations and media inquiries for Dyne Therapeutics are provided - Investors can contact Amy Reilly (areilly@dyne-tx.com, 857-341-1203) or Mike Hencke of Kendall Investor Relations (ir@dyne-tx.com)[14] - Media inquiries can be directed to Stacy Nartker (snartker@dyne-tx.com, 781-317-1938)[14]