Group 1 - The article promotes a weekly newsletter focused on stocks in the biotech, pharma, and healthcare industries, aimed at both novice and experienced investors [1] - The newsletter provides insights on key trends, catalysts driving valuations, product sales forecasts, and integrated financial statements for major pharmaceutical companies [1] - The author, Edmund Ingham, has over 5 years of experience in the biotech sector and has compiled detailed reports on more than 1,000 companies [1]

Core Viewpoint - Dyne Therapeutics is facing an investigation regarding potential securities fraud and unlawful business practices following a delay in FDA approval for its experimental therapy DYNE-101 for myotonic dystrophy type 1 [1][3]. Group 1: Company Developments - On June 17, 2025, Dyne Therapeutics announced a delay in filing for FDA approval of DYNE-101, which is intended for treating myotonic dystrophy type 1 [3]. - The company revised its protocol for the Phase 1/2 global ACHIEVE trial after a Type C meeting with the FDA, with the new enrollment goal set for the fourth quarter of 2025 and a potential data readout in mid-2026 [3]. - The previous timeline aimed for enrollment completion in mid-2025 and regulatory submission in the first half of 2026, indicating a significant shift in the project timeline [3]. Group 2: Market Reaction - Following the announcement of the delay, Dyne's stock price dropped by $2.96 per share, or 21.42%, closing at $10.86 per share on June 17, 2025 [4].

Summary of Dyne Therapeutics (DYN) Conference Call on June 17, 2025 Company Overview - **Company**: Dyne Therapeutics - **Focus**: Development of DYME-one hundred one for Myotonic Dystrophy Type one (DM1) Key Industry Insights - **Regulatory Progress**: - Dyne Therapeutics received Breakthrough Therapy Designation from the FDA for DYME-one hundred one, indicating significant unmet medical need in DM1 and strong clinical data supporting potential clinical benefits [6][7] - The company is pursuing accelerated approval by revising the protocol for the ongoing registrational expansion cohort of the ACHIEVE trial, with VHOD as the primary endpoint [8][9] Core Points and Arguments - **Clinical Data**: - New long-term clinical data show robust improvement in VHOD as early as three to six months, with strength measured by QMT improving by approximately 10% at six months and 20% at twelve months [11][12] - The updated clinical plan includes increasing the registrational cohort to 60 patients to enhance statistical power [10][18] - Sustained improvements across multiple clinical endpoints were observed, reinforcing the efficacy of DYME-one hundred one [20][21] - **Regulatory Strategy**: - The switch from using CASI-twenty two as a primary endpoint to VHOD is seen as a pragmatic decision based on FDA feedback, allowing for a more straightforward path to accelerated approval [15][19] - The company plans to submit data from the registrational expansion cohort for potential accelerated approval in late 2026 [12][34] - **Safety Profile**: - The safety profile of DYME-one hundred one remains favorable, with no serious treatment-emergent adverse events reported [28][29] Additional Important Insights - **Market Need**: Approximately 40,000 Americans are living with DM1, highlighting the significant unmet need for effective treatments [12] - **Future Plans**: Dyne Therapeutics is preparing for potential launches of DYME-one hundred one and DYNE-two fifty one in 2027, with a focus on leveraging synergies in marketing to healthcare providers [35][36] - **Financial Guidance**: The company has narrowed its cash runway guidance to the fourth quarter of 2026, indicating improved financial projections [37] Conclusion - Dyne Therapeutics is advancing its clinical development of DYME-one hundred one with a clear regulatory strategy aimed at accelerated approval, supported by promising clinical data and a favorable safety profile. The company is also preparing for future product launches, addressing significant unmet needs in the neuromuscular disease space.

Clinical Trial & Regulatory Strategy - Dyne and the FDA have agreed on the next step toward Accelerated Approval, which involves submitting the revised protocol for the Registrational Expansion Cohort of the ACHIEVE trial with vHOT as the primary endpoint[9] - The FDA has granted Breakthrough Therapy Designation for DYNE-101 in DM1 following a Type C Meeting[9] - Data from the Registrational Expansion Cohort are planned for mid-2026 to support a potential U S Accelerated Approval submission in late 2026[9] - The company is planning to initiate a confirmatory Phase 3 clinical trial in Q1 2026[54] DYNE-101 Efficacy Data - New data from the ACHIEVE trial demonstrate robust and sustained functional improvement across multiple measures at 6 and 12 months at the registrational dose of 6 8 mg/kg Q8W[9] - Data support vHOT improvement as an early indicator of clinical benefit with DYNE-101[9, 22, 24, 27, 41, 48] - At 6 months, the DYNE-101 6 8 mg/kg Q8W group showed a -3 0 second change from baseline in vHOT Middle Finger, a -39% change from baseline, compared to a +0 4 second change and +5% change from baseline in the placebo group[30] - At 12 months, the DYNE-101 6 8 mg/kg Q8W group showed a -2 9 second change from baseline in vHOT Middle Finger, a -38% change from baseline[30] - At 6 months, the DYNE-101 6 8 mg/kg Q8W group showed a 10 3 %p change from baseline in Quantitative Muscle Testing (QMT) Total Score, a 20 1% change from baseline, compared to a -2 0 %p change and -3 9% change from baseline in the placebo group[32] - At 12 months, the DYNE-101 6 8 mg/kg Q8W group showed a 5 2 %p change from baseline in Quantitative Muscle Testing (QMT) Total Score, a 10 1% change from baseline[32] - At 6 months, the DYNE-101 6 8 mg/kg Q8W group showed a -1 5 second change from baseline in the 5 Times Sit to Stand Test, a -14 5% change from baseline, compared to a +0 5 second change and +5 2% change from baseline in the placebo group[34] - At 12 months, the DYNE-101 6 8 mg/kg Q8W group showed a -1 2 second change from baseline in the 5 Times Sit to Stand Test, a -12 4% change from baseline[34] - At 6 months, the DYNE-101 6 8 mg/kg Q8W group showed a -0 4 second change from baseline in the 10-Meter Walk/Run Test, a -9 0% change from baseline, compared to a +0 1 second change and +4 3% change from baseline in the placebo group[34] - At 12 months, the DYNE-101 6 8 mg/kg Q8W group showed a -0 3 second change from baseline in the 10-Meter Walk/Run Test, a -7 2% change from baseline[34] - At 6 months, the DYNE-101 6 8 mg/kg Q8W group showed a -12 3 change from baseline in the Myotonic Dystrophy Health Index (MDHI) Total Score, a -46 5% change from baseline, compared to a +1 9 change and +10% change from baseline in the placebo group[37] - At 12 months, the DYNE-101 6 8 mg/kg Q8W group showed a -11 7 change from baseline in the Myotonic Dystrophy Health Index (MDHI) Total Score, a -44% change from baseline[37] Safety Profile - DYNE-101 has demonstrated a continued favorable safety profile[9, 22, 23, 27, 41] - Approximately 1000 doses of the study drug have been administered to date, representing 93 patient-years of follow-up[45] Financial Position - The company has a strong financial position with $677 5 million in cash as of March 31, 2025, with an expected runway into Q4 2026[62]

Core Viewpoint - Dyne Therapeutics has received Breakthrough Therapy Designation from the FDA for DYNE-101, aimed at treating myotonic dystrophy type 1 (DM1), and has outlined a revised plan for U.S. Accelerated Approval based on new long-term functional data [1][2][10]. Group 1: FDA Designations and Approvals - The FDA granted Breakthrough Therapy Designation to DYNE-101 for DM1, which expedites development and review for drugs showing substantial improvement over existing therapies [10]. - Dyne is pursuing U.S. Accelerated Approval for DYNE-101, with a revised protocol submitted to the FDA following a Type C meeting [6][5]. Group 2: Clinical Trial Updates - The ongoing Registrational Expansion Cohort of the ACHIEVE trial will enroll 60 participants, with video hand opening time (vHOT) as the primary endpoint for potential Accelerated Approval [6][5]. - New long-term data from the ACHIEVE trial indicate that DYNE-101 led to a 3.3 seconds improvement in vHOT at 6 months compared to placebo, with sustained improvements observed at 12 months [12]. - The trial's secondary endpoints include various measures of muscle strength and performance, with a reported 20% improvement in strength at 12 months relative to baseline [12]. Group 3: Financial Guidance - As of March 31, 2025, Dyne reported cash, cash equivalents, and marketable securities totaling $677.5 million, expected to fund operations into Q4 2026 [7]. Group 4: Future Plans - Dyne plans to complete enrollment in the Registrational Expansion Cohort by Q4 2025, with data expected in mid-2026 to support a potential U.S. Accelerated Approval submission in late 2026 [6]. - A confirmatory Phase 3 clinical trial is anticipated to begin in Q1 2026 [6].

Core Insights - Dyne Therapeutics plans to provide an update on DYNE-101 for myotonic dystrophy type 1 (DM1) on June 17, 2025, with a webcast scheduled for 8:00 a.m. ET [1] - The company is focused on developing innovative therapeutics for genetically driven neuromuscular diseases, utilizing its FORCE™ platform to enhance delivery to muscle tissue and the central nervous system [3] Company Overview - Dyne Therapeutics is dedicated to discovering and advancing life-transforming therapeutics for individuals with genetically driven neuromuscular diseases [3] - The company has a diverse pipeline that includes clinical programs for DM1 and Duchenne muscular dystrophy (DMD), as well as a preclinical program for facioscapulohumeral muscular dystrophy (FSHD) [3] Investor Engagement - A live webcast of the update will be available on Dyne's website, with a replay accessible for 90 days post-presentation [2] - An accompanying slide presentation will also be provided during the webcast [2]

Core Insights - Dyne Therapeutics announced new preclinical data for DYNE-302, showing potential functional improvement in facioscapulohumeral muscular dystrophy (FSHD) [1][2] - The data will be presented at the 32nd Annual FSHD Society's International Research Congress in Amsterdam on June 12-13, 2025 [1][4] Company Overview - Dyne Therapeutics is focused on developing therapeutics for genetically driven neuromuscular diseases, leveraging its FORCE™ platform [6] - The company has a broad pipeline that includes clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD), as well as preclinical programs for FSHD and Pompe disease [6] Disease Background - FSHD is a rare, progressive genetic disease caused by a mutation in the DUX4 gene, leading to muscle loss and weakness [5] - An estimated 16,000 to 38,000 individuals in the U.S. and approximately 35,000 in Europe are affected by FSHD, with no approved therapies currently available [5] Research Findings - In a mouse model of severe FSHD, a single intravenous dose of DYNE-302 restored the ability to run on a treadmill, indicating potential reversal of muscle damage and inflammation [2][3] - DYNE-302 targets DUX4 mRNA to suppress its expression, aiming to address the underlying cause of muscle damage in FSHD [3] Presentation Details - The oral presentation on DYNE-302's effects will take place on June 13, 2025, at 12:00 p.m. CEST [4] - The presentation will be available on Dyne's website following the session [4]

"Our two lead programs continue to demonstrate compelling and favorable data, including evidence of functional improvement across multiple measures in DM1 and DMD. We are urgently advancing both programs toward potential U.S. Accelerated Approval submissions in 2026 and possible commercial launches in 2027," said John Cox, president and chief executive officer of Dyne. "I am also thrilled to welcome Erick, Vik and Ron to the Dyne leadership team, which now includes a new role for Oxana as chief innovation o ...

Core Insights - Dyne Therapeutics is advancing its lead programs DYNE-101 and DYNE-251 towards potential U.S. Accelerated Approval submissions in 2026, with possible commercial launches in 2027 [2][5] Regulatory Developments - A Type C meeting with the FDA's CDER was held in May 2025 to discuss the regulatory path for DYNE-101 in DM1, with plans for a regulatory update following the receipt of meeting minutes [1][5] - The Registrational Expansion Cohort of the ACHIEVE Trial for DYNE-101 has been initiated, aiming for full enrollment by mid-2025 and data reporting in H1 2026 to support a potential BLA submission [1][5] - The Registrational Expansion Cohort of the DELIVER Trial for DYNE-251 has been fully enrolled, with data expected in late 2025 and a potential BLA submission anticipated in early 2026 [1][5] Financial Performance - As of March 31, 2025, the company reported cash, cash equivalents, and marketable securities totaling $677.5 million, sufficient to fund operations into the second half of 2026 [7] - Research and development expenses for Q1 2025 were $106.4 million, a significant increase from $44.5 million in Q1 2024 [7][8] - General and administrative expenses decreased to $15.9 million in Q1 2025 from $24.6 million in Q1 2024 [8] - The net loss for Q1 2025 was $115.4 million, or $1.05 per share, compared to a net loss of $65.6 million, or $0.81 per share, in Q1 2024 [8][14] Leadership Changes - Dyne has strengthened its leadership team with new appointments, including Erick Lucera as CFO, Vikram Ranade as CBO, and Ranjan Batra as CSO, to support the company's late-stage clinical and commercialization plans [2][5][11]

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 March 31, 2025 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) Delaware 3 ...