WALTHAM, Mass., May 20, 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 positive clinical data from its ongoing Phase 1/2 ACHIEVE trial of DYNE-101 in patients with myotonic dystrophy type 1 (DM1) and its ongoing Phase 1/2 DELIVER trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon ...

WALTHAM, Mass., May 20, 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 positive clinical data from its ongoing Phase 1/2 ACHIEVE trial of DYNE-101 in patients with myotonic dystrophy type 1 (DM1) and its ongoing Phase 1/2 DELIVER trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon ...

Core Viewpoint - Dyne Therapeutics is set to report new efficacy and safety data from its Phase 1/2 ACHIEVE and DELIVER clinical trials on May 20, 2024, indicating an update to its previous guidance for the second half of 2024 [1] Group 1: Clinical Trials Overview - The ACHIEVE trial evaluates DYNE-101 in adult patients with myotonic dystrophy type 1 (DM1), designed as a registrational trial with a 24-week multiple ascending dose (MAD) period, a 24-week open-label extension (OLE), and a 96-week long-term extension (LTE) [6] - Enrollment for the ACHIEVE trial is complete with 56 patients and approximately 500 doses administered, focusing on various dosing cohorts [6] - The DELIVER trial assesses DYNE-251 in males with Duchenne muscular dystrophy (DMD), also structured as a registrational trial with similar phases as ACHIEVE, and has enrolled 48 patients with approximately 480 doses administered [6] Group 2: Upcoming Data Release - Dyne plans to report safety and tolerability data from all enrolled cohorts in both trials, along with pharmacokinetic and pharmacodynamic data [6] - Efficacy data will be shared from specific dosing cohorts in the ACHIEVE trial, including results from the 1.8 mg/kg Q4W cohort (n=16) at 12 months, 3.4 mg/kg Q4W cohort (n=16) at 6 months, and 5.4 mg/kg Q8W cohort (n=8) at 3 months [6] - For the DELIVER trial, data on dystrophin expression will be reported from the 10 mg/kg Q4W cohort (n=8) at 6 months [6] Group 3: Company Background - Dyne Therapeutics focuses on developing innovative therapeutics for genetically driven muscle diseases using its proprietary FORCE™ platform [5] - The company has a broad pipeline targeting serious muscle diseases, including clinical programs for DM1 and DMD, as well as a preclinical program for facioscapulohumeral muscular dystrophy (FSHD) [5]

Core Viewpoint - Dyne Therapeutics is set to report new efficacy and safety data from its Phase 1/2 ACHIEVE and DELIVER clinical trials on May 20, 2024, indicating an update to its previous guidance for the second half of 2024 [1] Group 1: Clinical Trials Overview - The ACHIEVE trial evaluates DYNE-101 in adult patients with myotonic dystrophy type 1 (DM1), designed as a registrational trial with a total of 56 patients enrolled and approximately 500 doses administered to date [6] - The DELIVER trial assesses DYNE-251 in males with Duchenne muscular dystrophy (DMD), also designed as a registrational trial with 48 patients enrolled and approximately 480 doses administered to date [6] Group 2: Upcoming Data Release - Dyne plans to report safety and tolerability data from all enrolled cohorts in both trials, along with pharmacokinetic and pharmacodynamic data [6] - Efficacy data will be shared from various dosing cohorts in the ACHIEVE trial, including results from the 1.8 mg/kg cohort at 12 months, the 3.4 mg/kg cohort at 6 months, and the 5.4 mg/kg cohort at 3 months [6] - For the DELIVER trial, data on dystrophin expression will be reported from the 10 mg/kg cohort at 6 months [6] Group 3: Company Background - Dyne Therapeutics focuses on developing innovative therapeutics for genetically driven muscle diseases using its proprietary FORCE™ platform [5] - The company has a broad pipeline targeting serious muscle diseases, including clinical programs for DM1 and DMD, as well as a preclinical program for facioscapulohumeral muscular dystrophy (FSHD) [5]

- Additional Clinical Data from ACHIEVE Trial of DYNE-101 in DM1 and DELIVER Trial of DYNE-251 in DMD Anticipated in the Second Half of 2024 - WALTHAM, Mass., May 02, 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 reported financial results for the first quarter of 2024 and recent business highlights. "I am excited about the opportun ...

Financial Performance - For the three months ended March 31, 2024, the company reported a net loss of $65.6 million, compared to a net loss of $44.2 million for the same period in 2023[76]. - As of March 31, 2024, the company had an accumulated deficit of $698.2 million[76]. - Total operating expenses for Q1 2024 were $69.2 million, an increase of 52% compared to $45.5 million in Q1 2023[94]. - Net loss for Q1 2024 was $65.6 million, an increase of 48% from a net loss of $44.2 million in Q1 2023[94]. - Research and development expenses rose to $44.5 million in Q1 2024, up from $37.5 million in Q1 2023, reflecting a 19% increase[95]. - General and administrative expenses surged to $24.6 million in Q1 2024, compared to $7.9 million in Q1 2023, marking a 210% increase[97]. - Net cash used in operating activities was $78.5 million in Q1 2024, compared to $46.9 million in Q1 2023[106]. - Interest income increased to $3.0 million in Q1 2024 from $1.5 million in Q1 2023, representing a 100% increase[99]. Clinical Development - The company plans to initiate registrational cohorts for DYNE-101 and DYNE-251 by the end of 2024, following the completion of enrollment in their respective clinical trials[67][72]. - Initial clinical data from the ACHIEVE trial for DYNE-101 showed a favorable safety profile with no serious treatment-emergent adverse events identified[66]. - In the DELIVER trial for DYNE-251, initial data indicated that dystrophin expression levels exceeded those reported in a third-party clinical trial with a 24-fold lower total PMO dose[70]. - The majority of treatment-emergent adverse events in both trials were mild or moderate, with no clinically meaningful changes observed in kidney or liver parameters[66][71]. Cash and Funding - The company believes its existing cash, cash equivalents, and marketable securities will fund its operating expenses through 2025[80]. - Cash, cash equivalents, and marketable securities totaled $453.5 million as of March 31, 2024[101]. - The company expects to fund its operating expenses and capital expenditures through 2025 based on current cash reserves[111]. - The company issued 19,722,500 shares in a follow-on public offering in January 2024, generating net proceeds of $323.9 million[105]. - The company expects to finance cash needs through equity offerings, debt financings, collaborations, and licensing arrangements, which may dilute stockholder ownership[112]. Operational Commitments - The lease agreement for office and laboratory space has a term of 8.5 years, with base rent starting at $0.4 million per month, increasing to $0.5 million per month[115]. - The company has entered into a license agreement with the University of Mons, with payment obligations contingent on achieving specified milestones and generating product sales[114]. - The company has contractual obligations with CROs and CMOs for research and manufacturing services, which are generally cancelable with written notice[113]. Accounting and Compliance - The company is classified as an "emerging growth company" and may take advantage of exemptions from certain reporting requirements until December 31, 2025[121]. - The company has critical accounting policies related to accrued research and development expenses and stock-based compensation, which are expected to be increasingly important as product candidates advance[118]. - There have been no significant changes to critical accounting policies or estimates since the last Annual Report filed on March 5, 2024[119]. - There are no recently issued accounting pronouncements expected to materially impact the financial statements as of March 31, 2024[120]. Risks and Estimates - The company expects to incur significant expenses and increasing operating losses for the foreseeable future as it advances its clinical programs[76]. - The company may face risks associated with substantial capital requirements, including the inability to raise additional funds or enter into favorable agreements[112]. - The company evaluates estimates and assumptions on an ongoing basis, with actual results potentially differing from estimates under different conditions[117].

WALTHAM, Mass., March 13, 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 participate in a fireside chat at the Stifel 2024 Virtual CNS Days on Wednesday, March 20, 2024 at 8:30 a.m. ET. A live webcast will be available in the Investors & Media section of Dyne’s website at https://investors. ...

WALTHAM, Mass., March 13, 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 participate in a fireside chat at the Stifel 2024 Virtual CNS Days on Wednesday, March 20, 2024 at 8:30 a.m. ET. A live webcast will be available in the Investors & Media section of Dyne's website at https://investors.d ...

Pakin Jarerndee/iStock via Getty Images Dyne Therapeutics, Inc. (NASDAQ:DYN) is advancing the use of its FORCE platform to target skeletal muscle disorders. This would be with the use of modern oligonucleotide technology drugs like DYNE-101 and DYNE-251, which are both being advanced for the treatment of patients with myotonic dystrophy type 1 [DM1] and Duchenne Muscular Dystrophy [DMD] with specific exon skipping mutations. The DYNE-101 candidate is being advanced for the treatment of patients with DM1 in ...

- Positive Initial Clinical Data from ACHIEVE Trial in DM1 Patients and DELIVER Trial in DMD Patients Demonstrated Proof-of-Concept, Validating the Promise of the FORCE™ Platform and Targeted Delivery to Muscle - - Strengthened Balance Sheet with $345 Million Public Offering Extending Projected Cash Runway Through 2025 - WALTHAM, Mass., March 05, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (NASDAQ:DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeuti ...