Core Insights - Dyne Therapeutics, Inc. has revealed new data from its ongoing Phase 1/2 ACHIEVE trial of DYNE-101 for myotonic dystrophy type 1 (DM1) and plans to initiate a global Registrational Expansion Cohort to support U.S. Accelerated Approval submission in H1 2026 [1][4][5] Group 1: Trial Data - Efficacy data from the 6.8 mg/kg Q8W cohort (n=8) showed significant splicing correction at 3 months, linked to improvements in multiple functional endpoints at both 3 and 6 months [2][3] - Analysis of muscle biopsy data indicated a substantial knockdown of DMPK RNA levels, with robust splicing correction at 3 months supporting CASI-22 as a surrogate endpoint for potential U.S. Accelerated Approval [7] Group 2: Safety and Tolerability - Safety data from 56 patients indicated that most treatment-emergent adverse events were mild or moderate, with no serious treatment-emergent adverse events identified [4] - Early and sustained improvement in myotonia was observed, particularly in the 6.8 mg/kg Q8W cohort, with functional measures showing clinical benefits [7] Group 3: Future Plans - Dyne anticipates completing enrollment in the Registrational Expansion Cohort by mid-2025 and aims to submit for U.S. Accelerated Approval in H1 2026 [5]

Company Overview - Dyne Therapeutics is a clinical-stage company focused on developing innovative therapeutics for genetically driven neuromuscular diseases [3] - The company leverages its FORCE™ platform to design targeted therapeutics that address delivery challenges to muscle tissue and the central nervous system [3] - Dyne has a broad pipeline including clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD), as well as a preclinical program for facioscapulohumeral muscular dystrophy (FSHD) [3] Upcoming Event - Dyne Therapeutics' president and CEO John Cox is scheduled to present at the 43rd Annual J P Morgan Healthcare Conference on January 15, 2025 at 10:30 a m PT (1:30 p m ET) [1] - A live webcast of the presentation will be available on the company's website, with a replay accessible for 30 days following the event [2]

WALTHAM, Mass., Jan. 08, 2025 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage neuromuscular disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced it plans to report new clinical data from the Phase 1/2 ACHIEVE clinical trial on January 10, 2025, and to host a webcast at 8:00 a.m. ET. The company intends to issue a press release prior to the start of the event. Investor Conference Call and ...

[Corporate Update and Program Highlights](index=1&type=section&id=Corporate%20Update%20and%20Program%20Highlights) Dyne Therapeutics reported Q3 2024 results, highlighting clinical trial progress and a strong financial position [Key Announcements and CEO Commentary](index=1&type=section&id=Key%20Announcements%20and%20CEO%20Commentary) 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](index=1&type=chunk)[2](index=2&type=chunk)[3](index=3&type=chunk) - New clinical data from the DYNE-101 ACHIEVE trial is expected in early January 2025[1](index=1&type=chunk)[2](index=2&type=chunk)[4](index=4&type=chunk) - Dyne is enrolling patients in a registrational cohort at **20 mg/kg** for the DYNE-251 DELIVER trial in Duchenne muscular dystrophy (DMD)[1](index=1&type=chunk)[2](index=2&type=chunk)[5](index=5&type=chunk) [Clinical Program Updates](index=1&type=section&id=Clinical%20Program%20Updates) 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](index=1&type=section&id=ACHIEVE%20Trial%20of%20DYNE-101%20in%20DM1) 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](index=3&type=chunk) - The ACHIEVE trial is fully enrolled through the **6.8 mg/kg Q8W cohort**, with **56 participants**[3](index=3&type=chunk) - New data from the ACHIEVE trial, including safety, tolerability, splicing, vHOT, functional measures, and patient-reported outcomes, is anticipated in **early January 2025**[4](index=4&type=chunk) - Dyne continues to pursue expedited approval pathways globally for DYNE-101 utilizing splicing as a surrogate endpoint[5](index=5&type=chunk) [DELIVER Trial of DYNE-251 in DMD](index=1&type=section&id=DELIVER%20Trial%20of%20DYNE-251%20in%20DMD) 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](index=5&type=chunk) - 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](index=5&type=chunk) - 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](index=5&type=chunk) [Third Quarter 2024 Financial Results](index=2&type=section&id=Third%20Quarter%202024%20Financial%20Results) This section presents Dyne Therapeutics' financial performance for Q3 2024, including key metrics and balance sheet data [Financial Highlights](index=2&type=section&id=Financial%20Highlights) 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](index=7&type=chunk) [Condensed Consolidated Statement of Operations (Unaudited)](index=3&type=section&id=Condensed%20Consolidated%20Statement%20of%20Operations%20%28Unaudited%29) 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)](index=4&type=section&id=Condensed%20Consolidated%20Balance%20Sheet%20Data%20%28Unaudited%29) 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](index=2&type=section&id=Company%20Information%20and%20Disclosures) This section provides an overview of Dyne Therapeutics and important disclosures regarding forward-looking statements [About Dyne Therapeutics](index=2&type=section&id=About%20Dyne%20Therapeutics) 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](index=9&type=chunk) - The company utilizes its proprietary **FORCE™ platform** to develop modern oligonucleotide therapeutics designed to overcome limitations in delivery to muscle tissue[9](index=9&type=chunk) - 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](index=9&type=chunk) [Forward-Looking Statements](index=2&type=section&id=Forward-Looking%20Statements) 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](index=10&type=chunk) - 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](index=10&type=chunk)[11](index=11&type=chunk) - Dyne disclaims any obligation to update these forward-looking statements, which represent views as of the date of the press release[11](index=11&type=chunk) [Contacts](index=4&type=section&id=Contacts) This section provides contact information for investor relations and media inquiries for Dyne Therapeutics [Contact Information](index=4&type=section&id=Contact%20Information) 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](index=14&type=chunk) - Media inquiries can be directed to **Stacy Nartker (snartker@dyne-tx.com, 781-317-1938)**[14](index=14&type=chunk)

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended September 30, 2024 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: 001-39509 Dyne Therapeutics, Inc. (Exact Name of Registrant as Specified in its Charter) Delawa ...

WALTHAM, Mass., Nov. 07, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced that management is scheduled to present at the following upcoming investor conferences: Guggenheim Inaugural Healthcare Innovation Conference, fireside chat on Wednesday, November 13, 2024 at 4:00 p.m. ET in Boston Stifel 2024 Healthcare Conference, fi ...

- Presentations Will Feature Recent Clinical Data From DYNE-251 and DYNE-101 as Well as Preclinical Data in FSHD and Pompe Disease - WALTHAM, Mass., Oct. 09, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced that previously reported clinical and preclinical data across its pipeline will be featured in poster presentations at ...

NEW YORK, Sept. 10, 2024 (GLOBE NEWSWIRE) -- Bragar Eagel & Squire, P.C., a nationally recognized stockholder rights law firm, is investigating potential claims against Dyne Therapeutics, Inc. ("Dyne" or the "Company") (NASDAQ: DYN) on behalf of Dyne stockholders. Our investigation concerns whether Dyne has violated the federal securities laws and/or engaged in other unlawful business practices. Click here to participate in the action. The investigation focuses on whether the Company issued false and/or mis ...

NEW YORK, Sept. 9, 2024 /PRNewswire/ -- Pomerantz LLP is investigating claims on behalf of investors of Dyne Therapeutics, Inc. ("Dyne" or the "Company") (NASDAQ: DYN). Such investors are advised to contact Danielle Peyton at [email protected] or 646-581-9980, ext. 7980. The investigation concerns whether Dyne and certain of its officers and/or directors have engaged in securities fraud or other unlawful business practices. [Click here for information about joining the class action] On September 3, 2024, Dy ...

NEW YORK, Sept. 6, 2024 /PRNewswire/ -- Attorney Advertising -- Bronstein, Gewirtz & Grossman, LLC is investigating potential claims on behalf of purchasers of Dyne Therapeutics, Inc. ("Dyne" or "the Company") (NASDAQ: DYN). Investors who purchased Dyne securities are encouraged to obtain additional information and assist the investigation by visiting the firm's site: bgandg.com/DYN. Investigation Details On September 3, 2024, Dyne issued a press release "announc[ing] new clinical data from its ongoing Phas ...