Core Insights - Dyne Therapeutics has received Breakthrough Therapy Designation from the FDA for DYNE-251, aimed at treating Duchenne muscular dystrophy (DMD) through exon 51 skipping, with data from the DELIVER trial expected in late 2025 and a potential BLA submission anticipated in early 2026 [1][2][6] Company Overview - Dyne Therapeutics is focused on developing therapies for genetically driven neuromuscular diseases, including DMD and myotonic dystrophy type 1 (DM1) [9] - The company is advancing clinical programs targeting muscle and the central nervous system to address the root causes of these diseases [9] Product Details - DYNE-251 is an investigational therapeutic designed to produce near full-length dystrophin in muscle and the central nervous system, which is crucial for functional improvement in DMD patients [5][8] - The DELIVER trial is a global, randomized, placebo-controlled, double-blind Phase 1/2 clinical trial assessing the safety, tolerability, and efficacy of DYNE-251 in DMD patients with specific mutations [4][5] Clinical Trial Information - The DELIVER trial has completed enrollment of 32 patients in the Registrational Expansion Cohort, with the primary endpoint being the change in dystrophin protein levels at 6 months [6][4] - The trial's multiple ascending dose portion identified a registrational dose of 20 mg/kg of DYNE-251 administered every four weeks [4] Regulatory Designations - DYNE-251 has received multiple designations from the FDA, including Fast Track, Orphan Drug, and Rare Pediatric Disease designations, which facilitate expedited development and review processes [6][7] - The Breakthrough Therapy Designation allows for enhanced FDA support, including senior-level involvement and early communication regarding trial design and regulatory strategy [6][2] Disease Context - Duchenne muscular dystrophy (DMD) is a rare, progressive neuromuscular disorder primarily affecting males, with approximately 12,000 individuals in the U.S. and 16,000 in the EU affected [8] - There is a significant unmet need for new treatment options that can deliver functional improvements for DMD patients [8]

Core Viewpoint - Dyne Therapeutics, Inc. (DYN) shows significant upside potential with a mean price target of $38.21, indicating a 285.6% increase from the current trading price of $9.91 [1] Price Targets and Estimates - The mean estimate consists of 17 short-term price targets with a standard deviation of $14.79, suggesting variability in analyst predictions. The lowest estimate is $9.00 (9.2% decline), while the highest is $66.00 (566% increase) [2] - Analysts' price targets can be misleading, as empirical research indicates they rarely accurately predict stock price movements [7][10] Earnings Estimates - Analysts have shown increasing optimism regarding DYN's earnings, with a strong consensus in revising EPS estimates higher, which correlates with potential stock price increases [11] - Over the last 30 days, the Zacks Consensus Estimate for the current year has risen by 6.1%, with four estimates moving higher and no negative revisions [12] Analyst Rankings - DYN holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimate factors, indicating strong potential for near-term upside [13] Conclusion on Price Movement - While the consensus price target may not be a reliable measure of DYN's potential gains, the implied direction of price movement appears to be a useful guide for investors [14]

[Business Highlights and Clinical Pipeline Update](index=1&type=section&id=Business%20Highlights%20and%20Clinical%20Pipeline%20Update) Dyne advanced DM1 and DMD clinical programs towards 2026 U.S. Accelerated Approval, extending cash runway into Q3 2027 - The company is advancing its DM1 and DMD programs toward potential U.S. Accelerated Approval submissions in **2026**, with possible commercial launches in **2027**[3](index=3&type=chunk) - The company's cash runway is extended into the **third quarter of 2027**, which is expected to fund operations through multiple key milestones, including two data readouts, two potential U.S. Accelerated Approval submissions, and the potential U.S. launch of DYNE-251[3](index=3&type=chunk)[9](index=9&type=chunk) [DYNE-101 in Myotonic Dystrophy Type 1 (DM1)](index=1&type=section&id=DYNE-101%20in%20Myotonic%20Dystrophy%20Type%201%20%28DM1%29) DYNE-101 received FDA Breakthrough Therapy Designation, with ACHIEVE trial enrollment completing Q4 2025 for late 2026 BLA submission - In June 2025, the FDA granted **Breakthrough Therapy Designation** to DYNE-101 for the treatment of DM1[6](index=6&type=chunk) - The company plans to complete enrollment of **60 patients** in the Registrational Expansion Cohort of the ACHIEVE trial in **Q4 2025**[6](index=6&type=chunk) - Data from the ACHIEVE cohort is planned for **mid-2026** to support a potential U.S. Accelerated Approval BLA submission in **late 2026**[6](index=6&type=chunk) [DYNE-251 in Duchenne Muscular Dystrophy (DMD)](index=2&type=section&id=DYNE-251%20in%20Duchenne%20Muscular%20Dystrophy%20%28DMD%29) DELIVER trial for DYNE-251 completed enrollment, with data expected late 2025 for early 2026 U.S. Accelerated Approval BLA submission - Enrollment of **32 patients** in the Registrational Expansion Cohort of the DELIVER trial is complete, with data expected in **late 2025**[10](index=10&type=chunk) - A potential BLA submission for U.S. Accelerated Approval is anticipated in **early 2026** for patients with DMD amenable to exon 51 skipping[10](index=10&type=chunk) [DYNE-302 in Facioscapulohumeral Muscular Dystrophy (FSHD)](index=2&type=section&id=DYNE-302%20in%20Facioscapulohumeral%20Muscular%20Dystrophy%20%28FSHD%29) New preclinical data for DYNE-302 presented in June 2025 demonstrated potential for functional improvement in FSHD - New preclinical data for DYNE-302 was presented in **June 2025**, showing its potential for functional improvement in FSHD[7](index=7&type=chunk) [Financial Condition and Results of Operations](index=2&type=section&id=Financial%20Condition%20and%20Results%20of%20Operations) Dyne strengthened its financial position with new financing, extending cash runway into Q3 2027, despite increased Q2 2025 net loss [Financing Updates and Cash Runway](index=2&type=section&id=Financing%20Updates%20and%20Cash%20Runway) Dyne secured $275 million debt and $230 million from public offering, extending cash runway to Q3 2027 with $683.9 million cash - Entered into a **$275 million** non-dilutive senior secured term loan facility with Hercules Capital, with an initial **$100 million** funded at closing[10](index=10&type=chunk) - Completed a public offering of common stock in July 2025, raising gross proceeds of approximately **$230 million**[10](index=10&type=chunk) - Cash, cash equivalents and marketable securities were **$683.9 million** as of June 30, 2025. The cash runway is expected to extend into the **third quarter of 2027**[12](index=12&type=chunk) [Second Quarter 2025 Financial Results](index=2&type=section&id=Second%20Quarter%202025%20Financial%20Results) Q2 2025 net loss increased to $110.9 million ($0.97/share) due to higher R&D and G&A expenses Q2 2025 vs Q2 2024 Financial Performance | Metric | Q2 2025 (USD) | Q2 2024 (USD) | Change (%) | | :--- | :--- | :--- | :--- | | R&D Expenses | $99.2 million | $62.3 million | +59.2% | | G&A Expenses | $16.6 million | $9.7 million | +71.1% | | Net Loss | $110.9 million | $65.1 million | +70.4% | | Net Loss Per Share | $0.97 | $0.70 | +38.6% | [Condensed Consolidated Financial Statements](index=4&type=section&id=Condensed%20Consolidated%20Financial%20Statements) Condensed financial statements detail Q2 2025 performance and June 30, 2025, financial position, including key operating expenses and net loss Condensed Consolidated Statement of Operations (in thousands, except per share data) | | Three Months Ended June 30, | | :--- | :--- | :--- | | | **2025** | **2024** | | **Operating expenses:** | | | | Research and development | $ 99,236 | $ 62,263 | | General and administrative | $ 16,555 | $ 9,699 | | **Total operating expenses** | **$ 115,791** | **$ 71,962** | | Loss from operations | $ (115,791) | $ (71,962) | | Other (expense) income, net | $ 4,934 | $ 6,860 | | **Net loss** | **$ (110,857)** | **$ (65,102)** | | Net loss per share, basic and diluted | $ (0.97) | $ (0.70) | Condensed Consolidated Balance Sheet Data (in thousands) | | **June 30, 2025** | **December 31, 2024** | | :--- | :--- | :--- | | **Assets** | | | | Cash, cash equivalents and marketable securities | $ 683,925 | $ 642,268 | | Total assets | $ 728,992 | $ 691,234 | | **Liabilities and Stockholders' Equity** | | | | Liabilities | $ 157,547 | $ 61,396 | | Stockholders' equity | $ 571,445 | $ 629,838 | | Total liabilities and stockholders' equity | $ 728,992 | $ 691,234 |

Core Insights - Dyne Therapeutics has extended its cash runway into Q3 2027, supporting multiple potential inflection points including the planned commercial launch of DYNE-251 in early 2027 [1][2] - The Registrational Expansion Cohort of the DELIVER Trial for DYNE-251 in Duchenne Muscular Dystrophy (DMD) is fully enrolled, aiming for U.S. Accelerated Approval submission in early 2026 [1][7] - The ACHIEVE Trial for DYNE-101 in Myotonic Dystrophy Type 1 (DM1) is ongoing, with plans for U.S. Accelerated Approval submission in late 2026 [1][7] Financial Performance - As of June 30, 2025, Dyne reported cash, cash equivalents, and marketable securities totaling $683.9 million, with an additional estimated net proceeds of approximately $215.2 million from a public offering completed in July 2025 [10][12] - Research and Development (R&D) expenses for Q2 2025 were $99.2 million, up from $62.3 million in Q2 2024, while General and Administrative (G&A) expenses increased to $16.6 million from $9.7 million [11][12] - The net loss for Q2 2025 was $110.9 million, or $0.97 per share, compared to a net loss of $65.1 million, or $0.70 per share, in Q2 2024 [12][18] Clinical Development - Dyne is advancing its clinical programs for DM1 and DMD, with two data readouts expected in 2026 and potential commercial launches in 2027 [2][7] - The FDA granted Breakthrough Therapy Designation to DYNE-101 for DM1, and a revised protocol for the ACHIEVE trial has been submitted [7] - Dyne plans to initiate a confirmatory Phase 3 clinical trial for DYNE-101 in Q1 2026 and has completed enrollment of 32 patients in the DELIVER trial [7][9] Financing Updates - In June 2025, Dyne secured a $275 million non-dilutive senior secured term loan facility with Hercules Capital, which includes an initial term loan of $100 million [6][8] - The company completed a public offering of 27,878,788 shares at a price of $8.25 per share, generating gross proceeds of approximately $230 million [8][10] Research Focus - Dyne Therapeutics is focused on developing therapeutics for genetically driven neuromuscular diseases, including DM1, DMD, and preclinical programs for Facioscapulohumeral Muscular Dystrophy (FSHD) and Pompe disease [13]

[PART I. FINANCIAL INFORMATION](index=6&type=section&id=PART%20I.%20FINANCIAL%20INFORMATION) This section presents the company's unaudited financial statements, management's discussion, market risk, and internal controls [Financial Statements (Unaudited)](index=6&type=section&id=Item%201.%20Financial%20Statements%20(Unaudited)) Unaudited financial statements for Q2 2025 highlight increased net loss and operating expenses, a stronger cash position, and a growing accumulated deficit Condensed Consolidated Balance Sheet Highlights (in thousands) | Account | June 30, 2025 | December 31, 2024 | | :--- | :--- | :--- | | **Assets** | | | | Cash and cash equivalents | $485,040 | $435,449 | | Total current assets | $697,950 | $659,279 | | Total assets | $728,992 | $691,234 | | **Liabilities & Equity** | | | | Total current liabilities | $41,472 | $42,258 | | Long-term debt, net | $98,813 | $— | | Total liabilities | $157,547 | $61,396 | | Accumulated deficit | $(1,176,146) | $(949,928) | | Total stockholders' equity | $571,445 | $629,838 | Condensed Consolidated Statements of Operations Highlights (in thousands, except per share data) | Metric | Three Months Ended June 30, 2025 | Three Months Ended June 30, 2024 | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | :--- | :--- | | Research and development | $99,236 | $62,263 | $205,683 | $106,802 | | Total operating expenses | $115,791 | $71,962 | $238,163 | $141,119 | | Net loss | $(110,857) | $(65,102) | $(226,219) | $(130,751) | | Net loss per share | $(0.97) | $(0.70) | $(2.02) | $(1.51) | Condensed Consolidated Statements of Cash Flows Highlights (in thousands) | Cash Flow Activity | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | | :--- | :--- | :--- | | Net cash used in operating activities | $(200,596) | $(133,405) | | Net cash (used in) provided by investing activities | $8,232 | $(168,663) | | Net cash provided by financing activities | $242,015 | $788,250 | - In June 2025, the company entered into a Loan Agreement with Hercules Capital for term loans up to **$275.0 million**, with an initial tranche of **$100.0 million** funded at closing[48](index=48&type=chunk) - During the six months ended June 30, 2025, the company sold 10,660,159 shares of common stock through its at-the-market (ATM) offering program for net proceeds of **$140.6 million**[59](index=59&type=chunk) [Management's Discussion and Analysis of Financial Condition and Results of Operations](index=20&type=section&id=Item%202.%20Management's%20Discussion%20and%20Analysis%20of%20Financial%20Condition%20and%20Results%20of%20Operations) Management discusses clinical progress of FORCE™ platform candidates, increased R&D expenses, and financial runway into Q3 2027 - The company is a clinical-stage entity focused on genetically driven neuromuscular diseases using its proprietary FORCE™ platform, with key programs in DM1, DMD, FSHD, and Pompe disease[80](index=80&type=chunk)[81](index=81&type=chunk) - DYNE-101 (DM1 Program): Received **FDA Breakthrough Therapy Designation** in June 2025. The registrational expansion cohort of the ACHIEVE trial is initiated, with full enrollment planned for Q4 2025, data expected mid-2026, and a potential U.S. Accelerated Approval submission in late 2026[84](index=84&type=chunk)[85](index=85&type=chunk) - DYNE-251 (DMD Program): **Completed enrollment** of the registrational expansion cohort of the DELIVER trial in Q1 2025. Data from this cohort is planned for late 2025, with a potential U.S. Accelerated Approval submission in early 2026[94](index=94&type=chunk) Comparison of Operating Expenses (in thousands) | Expense Category | Six Months Ended June 30, 2025 | Six Months Ended June 30, 2024 | Change | | :--- | :--- | :--- | :--- | | Research and development | $205,683 | $106,802 | $98,881 | | General and administrative | $32,480 | $34,317 | $(1,837) | | **Total operating expenses** | **$238,163** | **$141,119** | **$97,044** | - The **increased R&D expenses** for the six months ended June 30, 2025, was primarily driven by higher manufacturing and clinical trial activity for the DYNE-101 and DYNE-251 programs[129](index=129&type=chunk) - The company believes its existing cash, cash equivalents, marketable securities, proceeds from the June 2025 debt facility, and the July 2025 public offering will **fund operations into the third quarter of 2027**[103](index=103&type=chunk)[146](index=146&type=chunk) [Quantitative and Qualitative Disclosures About Market Risk](index=32&type=section&id=Item%203.%20Quantitative%20and%20Qualitative%20Disclosures%20About%20Market%20Risk) The company reports no material changes in its market risk profile since the prior annual report - There have been **no material changes** in the company's market risk profile during the period ended June 30, 2025[157](index=157&type=chunk) [Controls and Procedures](index=32&type=section&id=Item%204.%20Controls%20and%20Procedures) Management concluded disclosure controls were effective as of June 30, 2025, with no material changes to internal controls - The CEO and CFO concluded that the company's disclosure controls and procedures were **effective** at a reasonable assurance level as of June 30, 2025[160](index=160&type=chunk) - **No material changes** to the internal control over financial reporting occurred during the three months ended June 30, 2025[161](index=161&type=chunk) [PART II. OTHER INFORMATION](index=33&type=section&id=PART%20II.%20OTHER%20INFORMATION) This section covers legal proceedings, risk factors, equity sales, and other required disclosures [Legal Proceedings](index=33&type=section&id=Item%201.%20Legal%20Proceedings) The company reports no legal proceedings to disclose for the period - The company has **no legal proceedings** to report[162](index=162&type=chunk) [Risk Factors](index=33&type=section&id=Item%201A.%20Risk%20Factors) This section details key risks: significant losses, unproven platform, third-party reliance, competition, and regulatory hurdles - The company has a history of significant losses (**$1.2 billion** accumulated deficit as of June 30, 2025) and will need substantial additional funding to continue operations and product development[164](index=164&type=chunk)[170](index=170&type=chunk) - The company's FORCE platform is unproven, and all product candidates are in development. Failure to advance candidates through clinical trials and obtain marketing approval would materially harm the business[15](index=15&type=chunk)[191](index=191&type=chunk)[211](index=211&type=chunk) - The company relies on third parties for manufacturing, research, and clinical testing. Failure of these third parties to perform satisfactorily could delay or derail development programs[230](index=230&type=chunk) - The company faces substantial competition from major pharmaceutical and biotechnology companies developing treatments for the same diseases, including DM1, DMD, and FSHD[264](index=264&type=chunk)[266](index=266&type=chunk)[268](index=268&type=chunk) - The Loan Agreement with Hercules contains restrictive covenants, including a **Minimum Cash Covenant** and a **Minimum Revenue Covenant**, which may limit operating flexibility and could result in default if not met[186](index=186&type=chunk)[187](index=187&type=chunk) - The company's ability to obtain and defend intellectual property for its platform and product candidates is critical and uncertain, and failure to do so would adversely affect its competitive position[293](index=293&type=chunk) [Unregistered Sales of Equity Securities and Use of Proceeds](index=96&type=section&id=Item%202.%20Unregistered%20Sales%20of%20Equity%20Securities%20and%20Use%20of%20Proceeds) The company reports no unregistered sales of equity securities during the period - There were **no unregistered sales** of equity securities in the reported quarter[504](index=504&type=chunk) [Other Information](index=96&type=section&id=Item%205.%20Other%20Information) No directors or officers adopted or terminated trading arrangements during the quarter - **No directors or officers adopted or terminated** a Rule 10b5-1 or non-Rule 10b5-1 trading arrangement during the quarter[507](index=507&type=chunk) [Exhibits](index=97&type=section&id=Item%206.%20Exhibits) This section lists key exhibits filed with the Form 10-Q, including the Loan and Security Agreement and certifications - Key exhibits filed include the **Loan and Security Agreement dated June 27, 2025**, and certifications by the Principal Executive Officer and Principal Financial Officer[509](index=509&type=chunk)

Group 1 - Dyne Therapeutics, Inc. announced a public offering of 24,242,425 shares of common stock at a price of $8.25 per share, aiming for gross proceeds of $200 million before expenses [1] - The offering is expected to close around July 2, 2025, pending customary closing conditions [1] - Dyne has granted underwriters a 30-day option to purchase an additional 3,636,363 shares at the public offering price [1] Group 2 - The offering is conducted under a shelf registration statement filed with the SEC on March 5, 2024, which became effective upon filing [3] - A preliminary prospectus supplement has been filed with the SEC, and a final prospectus will also be filed [3] Group 3 - Dyne Therapeutics focuses on developing therapeutics for genetically driven neuromuscular diseases, including myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD) [5] - The company is also advancing preclinical programs for facioscapulohumeral muscular dystrophy (FSHD) and Pompe disease [5]

Core Insights - Dyne Therapeutics has secured a $275 million non-dilutive senior secured term loan facility with Hercules Capital to advance its clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD) [1][2] Group 1: Financial Details - The loan facility includes an initial $100 million funded upfront, with an additional $175 million tied to clinical, regulatory, and commercial milestones [1][2] - The loan consists of five tranches, allowing Dyne to access up to $115 million based on milestone achievements, and a final tranche of up to $60 million subject to approval [2] Group 2: Strategic Implications - The financing enhances Dyne's ability to advance its DM1 and DMD programs, with potential U.S. Accelerated Approval submissions planned for 2026 [2] - The company is positioned for a potential U.S. launch in DMD in 2027, reflecting confidence in its leadership and pipeline [2][4] Group 3: Company Overview - Dyne Therapeutics focuses on delivering functional improvement for individuals with genetically driven neuromuscular diseases, developing therapeutics targeting muscle and the central nervous system [3] - The company is advancing clinical programs for DM1 and DMD, along with preclinical programs for facioscapulohumeral muscular dystrophy (FSHD) and Pompe disease [3]

Core Viewpoint - DJS Law Group is investigating Dyne Therapeutics for potential violations of securities laws following a significant drop in share price after the company announced revised trial protocols [1][2]. Investigation Details - The investigation centers on whether Dyne Therapeutics made misleading statements or failed to disclose critical information to investors [2]. - Following a June 17, 2025 announcement regarding feedback from the FDA and new efficacy data, Dyne's shares fell by over 19.4% during intraday trading on the same day [2]. Company Background - DJS Law Group specializes in enhancing investor returns through legal advocacy, focusing on securities class actions, corporate governance litigation, and M&A appraisals [4].

Group 1 - Dyne Therapeutics is under investigation by Pomerantz LLP for potential securities fraud or unlawful business practices involving its officers and directors [1] - On June 17, 2025, Dyne announced a delay in filing for FDA approval of its experimental therapy DYNE-101 for myotonic dystrophy type 1, revising its enrollment timeline for the Phase 1/2 global ACHIEVE trial [3] - Following the announcement of the delay, Dyne's stock price dropped by $2.96, or 21.42%, closing at $10.86 per share [4] Group 2 - The revised schedule for the ACHIEVE trial indicates that Dyne now aims to complete enrollment in the fourth quarter of 2025, with data readout expected in mid-2026 and a potential Accelerated Approval submission in late 2026 [3]

Core Viewpoint - Dyne Therapeutics is facing an investigation regarding potential securities fraud or unlawful business practices following a delay in FDA approval for its experimental therapy DYNE-101 for myotonic dystrophy type 1 [1][2]. Group 1: Company Developments - On June 17, 2025, Dyne Therapeutics announced a delay in filing for FDA approval of DYNE-101, which is an experimental therapy for myotonic dystrophy type 1 [2]. - The company revised its protocol for the Phase 1/2 global ACHIEVE trial for DYNE-101 after a Type C meeting with the FDA, with the revised protocol submitted in June [2]. - Dyne now plans to complete enrollment in the Registrational Expansion Cohort by the fourth quarter of 2025, aiming for a data readout in mid-2026 and a potential Accelerated Approval submission in late 2026 [2]. Group 2: Market Reaction - Following the announcement of the delay, Dyne's stock price fell by $2.96 per share, representing a decline of 21.42%, closing at $10.86 per share on June 17, 2025 [3].