BOSTON, Feb. 02, 2026 (GLOBE NEWSWIRE) -- Sparrow Pharmaceuticals, a targeted cardiometabolic therapeutics company, today announced the appointment of Carlo Incerti, M.D., to its Board of Directors, effective January 1, 2026. Dr. Incerti is a highly accomplished executive with a strong background in medicine and over 35 years of strategic experience in the biopharmaceutical industry. “Carlo’s appointment comes at a pivotal moment for Sparrow as we are looking ahead to pivotal trials of clofutriben for type ...

Key Takeaways KROS is advancing KER-065 for DMD, with FDA orphan drug status and a phase II trial planned for early 2026.DYN reported positive data for z-rostudirsen, showing dystrophin gains and functional improvements in DMD.DYN held $791.9M in cash as of Sept. 2025, funding operations into Q3 2027, alongside multiple programs.Keros Therapeutics (KROS) is a clinical-stage biopharmaceutical company developing innovative therapies for patients with disorders caused by abnormal signaling within the transform ...

Analyst’s Disclosure: I/we have a beneficial long position in the shares of DYN either through stock ownership, options, or other derivatives. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article. Seeking Alpha's Disclosure: Past performance is no guarantee of future results. No recommendation or advice is being given as to whether any in ...

Core Insights - Dyne Therapeutics has received Orphan Drug designation in Japan for zeleciment basivarsen (z-basivarsen) to treat myotonic muscular dystrophy type 1 (DM1), highlighting the urgent need for new therapies in this area [1][2] - Z-basivarsen has shown early and sustained improvements in myotonia, muscle strength, and function, with a favorable safety profile, and has also received similar designations in the U.S. and Europe [2][4] - The ACHIEVE trial is a Phase 1/2 clinical trial evaluating z-basivarsen, with a registrational dose of 6.8 mg/kg administered every eight weeks, aiming for potential regulatory submissions [3][4] Company Overview - Dyne Therapeutics focuses on delivering functional improvement for individuals with genetically driven neuromuscular diseases, including DM1 and Duchenne muscular dystrophy (DMD) [6][7] - The company is advancing clinical programs targeting muscle and the central nervous system to address the root causes of these diseases [7] Clinical Trial Details - The ACHIEVE trial is a global, randomized, placebo-controlled, double-blind study assessing the safety, tolerability, and efficacy of z-basivarsen in DM1 patients [3] - The primary endpoint for the registrational expansion cohort is the change in middle finger myotonia measured by video hand opening time (vHOT) at 6 months compared to placebo [3] Disease Context - Myotonic dystrophy type 1 (DM1) is a rare genetic neuromuscular disease affecting approximately 40,000 people in the U.S. and 55,000 in the EU, characterized by high morbidity and early mortality [5] - Symptoms of DM1 can vary widely and include muscle weakness, myotonia, cognitive impairments, and other systemic issues, with no approved disease-modifying treatments currently available [5]

Dyne Therapeutics, Inc. (NASDAQ:DYN) is among the 14 best booming stocks to buy right now, with returns of over 23% in the last three months. Wall Street analysts have a consensus Strong Buy rating on the stock and project a further 120% upside as of the close on January 12. Analysts Project 120% Upside To Dyne Therapeutics, Inc. (DYN) Recent analyst updates include Evercore ISI’s Gavin Clark-Gartner on December 17, trimming the firm’s price target on the stock to $36 from $38, while maintaining an earli ...

Core Insights - Dyne Therapeutics possesses two transformative late-stage assets targeting significant unmet needs in Duchenne Muscular Dystrophy (DMD) and Myotonic Dystrophy Type 1 (DM1) with key milestones approaching in 2026 and 2027 [3] Financial Position - The company has a strong cash position exceeding $1 billion and fully owns its assets, indicating a solid financial foundation for future developments [4] Strategic Outlook - The company plans to submit its first Biologics License Application (BLA) in 2026, complete a second registrational trial in the same year, and launch its first commercial product in the first quarter of 2027, followed by another product launch one year later [3][4] Market Position - Dyne Therapeutics claims to have the best-in-class platform for delivering genetic medicines specifically for neuromuscular diseases, which positions the company favorably within the biotech industry [3]

Dyne Therapeutics Conference Call Summary Company Overview - **Company**: Dyne Therapeutics (NasdaqGS:DYN) - **Industry**: Biotechnology, focusing on neuromuscular diseases Key Points and Arguments Transformative Assets - Dyne Therapeutics has two late-stage assets targeting Duchenne Muscular Dystrophy (DMD) and Myotonic Dystrophy Type 1 (DM1), both of which address significant unmet medical needs [2][3] - The company plans to submit its first Biologics License Application (BLA) in 2026 and launch its first commercial product in Q1 2027, with another product following a year later [2][3] Financial Position - Dyne has a strong cash position exceeding $1 billion, allowing for the development and commercialization of its assets [2] Delivery Platform - The company’s FORCE platform utilizes an antibody fragment to target the transferrin receptor, facilitating the delivery of genetic medicines to muscle and central nervous system (CNS) tissues [3][4] - This platform has shown promising results in non-human primate studies, demonstrating effective delivery of oligonucleotides to critical tissues [5] Clinical Validation - In 2025, Dyne validated its platform in humans, with the first medicine, zurastadersen, showing significant functional improvements in DMD patients [6][12] - The DELIVER trial demonstrated a seven-fold increase in dystrophin levels, a key surrogate marker for DMD, with a p-value of 0.0001, indicating strong statistical significance [11] Functional Improvements - Patients treated with zurastadersen showed improvements in various functional measures, including time to rise and walking speed, compared to placebo [12][14] - The treatment also stabilized lung capacity, which is critical for non-ambulatory patients [14] Market Opportunity - Dyne aims to create a franchise by developing additional exon-skipping therapies for DMD, potentially tripling the market size for the condition [9][18] - The company is also preparing to enter the DM1 market, which is larger and currently lacks approved therapies [20] Regulatory Strategy - Dyne has received breakthrough designation from the FDA for both DMD and DM1, facilitating ongoing dialogue regarding accelerated approval pathways [33][35] - The company is on track to submit data for accelerated approval in Q2 2026 [11][35] Manufacturing and Commercialization - Dyne has established a global supply chain and manufacturing capabilities to support its commercial launch [31] - The company anticipates competitive gross margins similar to other rare disease antibody companies, leveraging efficiencies across its pipeline [32] Future Plans - Dyne plans to initiate a confirmatory Phase 3 study for DMD in Q2 2026, focusing on clinically meaningful endpoints [37][49] - The company is also prioritizing the development of FSHD as the next program in the clinic, alongside ongoing efforts for other DMD exons [39] Additional Important Information - The company has a favorable safety profile from its trials, with most treatment-emergent adverse events being mild or moderate [15][25] - Dyne is actively working to address capacity issues in its clinical trials by increasing the number of enrollment sites [40][41] This summary encapsulates the key points from Dyne Therapeutics' conference call, highlighting the company's strategic direction, clinical advancements, and market potential in the biotechnology sector focused on neuromuscular diseases.

Advancing Clinically Validated Platform Toward Commercialization This presentation contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical fact, contained in this presentation, including statements regarding Dyne's strategy, future operations, prospects and plans, objectives of management, the potential of the FORCE platform and its conjugates, the therapeutic potential of zeleciment basivarsen (z-basivarsen, also known as DYN ...

Financial Position - Dyne Therapeutics expects to report cash, cash equivalents, and marketable securities of approximately $1.1 billion as of December 31, 2025[5] - The estimated cash figure is preliminary and unaudited, subject to completion of the Company's financial closing procedures[6] Investor Communication - A presentation discussing the Company's current operations and future plans was made available to investors on January 12, 2026[8]

WALTHAM, Mass., Jan. 07, 2026 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on delivering functional improvement for people living with genetically driven neuromuscular diseases, today announced that John Cox, president and chief executive officer, is scheduled to present at the 44th Annual J.P. Morgan Healthcare Conference in San Francisco on Wednesday, January 14, 2026 at 5:15 p.m. PT (8:15 p.m. ET). A live webcast will be available in the Investors & Media se ...