dystrophin

- Recently presented data demonstrated sustained functional improvement with DYNE-251 treatment through 18 months - - Data from the fully enrolled DELIVER registrational expansion cohort is planned for late 2025 - April 24, 2025 -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage company focused on advancing life-transforming therapeutics for people living with genetically driven neuromuscular diseases, today announced that the European Commission (EC) has granted orphan drug designation for DYNE-251 for the treatment of Duchenne muscular dystrophy (DMD). DYNE-251 is being evaluated in the Phase 1/2 DELIVER global clinical trial in individuals with DMD who are amenable to exon 51 skipping. Long-term clinical data from the ongoing DELIVER trial of DYNE-251 that demonstrated unprecedented and sustained functional improvement at the selected registrational dose were presented in March at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference. Functional assessments in the DELIVER trial include Stride Velocity 95th Centile (SV95C), an objective digital outcome that is accepted as a primary endpoint for DMD clinical trials in Europe. “Our recent long-term DELIVER trial results demonstrated clinically relevant and sustained functional improvement through 18 months, including as assessed by SV95C, which may support a strong rationale for regulatory approval in Europe,” said Doug Kerr, MD, PhD, chief medical officer of Dyne. “We are pleased that the EC has granted orphan drug designation to DYNE-251, reinforcing our belief that our next-generation exon 51 skipping investigational therapy for DMD may be able to bring clinically meaningful functional improvement to those living with this devastating disease. With full enrollment of the registrational expansion cohort in the DELIVER trial complete, we look forward to sharing data from this cohort in late 2025 and the potential to move forward with our first regulatory submissions in early 2026.” The EC grants orphan drug designation to drugs and biologics intended for the treatment, diagnosis or prevention of rare, life-threatening or chronically debilitating diseases or conditions that affect fewer than five in 10,000 people in the European Union (EU). Orphan designation provides companies with certain benefits, including reduced regulatory fees, clinical protocol assistance, research grants and the potential for up to 10 years of market exclusivity in the EU if approved. DYNE-251 was also granted U.S. Food and Drug Administration (FDA) orphan drug and rare pediatric disease designations in March 2023. Key Milestones for the DELIVER Trial Dyne continues to pursue expedited approval pathways globally for DYNE-251 in patients with DMD who are amenable to exon 51 skipping. Dyne has fully enrolled the registrational expansion cohort of 32 patients as part of the DELIVER trial. Data from this cohort are planned for late 2025. Dyne anticipates filing a Biologics License Application (BLA) submission for US accelerated approval in early 2026. DELIVER is a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial evaluating the safety, tolerability and efficacy of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. The multiple ascending dose (MAD) portion of the study resulted in the selection of a registrational dose and regimen of 20 mg/kg every four weeks. A registrational expansion cohort to support potential regulatory submissions for expediated approvals, including accelerated approval in the U.S., is fully enrolled. The primary endpoint for this cohort is the change from baseline in dystrophin protein levels as measured by Western blot. For more information on the DELIVER trial, visit clinicaltrials.gov (NCT05524883) and euclinicaltrials.eu (2023-510351-31-00). DYNE-251 is an investigational therapeutic being evaluated in the Phase 1/2 global DELIVER clinical trial for people living with DMD who are amenable to exon 51 skipping. DYNE-251 consists of a phosphorodiamidate morpholino oligomer (PMO) conjugated to a fragment antibody (Fab) that binds to the transferrin receptor 1 (TfR1), which is highly expressed on muscle. It is designed to enable targeted muscle tissue delivery and promote exon skipping in the nucleus, allowing muscle cells to create internally shortened, near full-length dystrophin protein, with the goal of stopping or reversing disease progression. DYNE-251 has been granted fast track, orphan drug and rare pediatric disease designations by the U.S. Food and Drug Administration for the treatment of DMD mutations amenable to exon 51 skipping. In addition to DYNE-251, Dyne is building a global DMD franchise and has preclinical programs targeting other exons, including 53, 45 and 44. DMD is a rare disease caused by mutations in the gene that encodes for dystrophin, a protein critical for the normal function of muscle cells. These mutations, the majority of which are deletions, result in the lack of dystrophin protein and progressive loss of muscle function. DMD occurs primarily in males and affects an estimated 12,000 to 15,000 individuals in the U.S. and 25,000 in Europe. Loss of strength and function typically first appears in pre-school age boys and worsens as they age. As the disease progresses, the severity of damage to skeletal and cardiac muscle often results in patients experiencing total loss of ambulation by their early teenage years and includes worsening cardiac and respiratory symptoms and loss of upper body function by the later teens. There is no cure for DMD, and currently approved therapies provide limited benefit. Dyne Therapeutics is discovering and advancing innovative life-transforming therapeutics for people living with genetically driven neuromuscular diseases. Leveraging the modularity of its FORCE™ platform, Dyne is developing targeted therapeutics that deliver to muscle and the central nervous system (CNS). Dyne has a broad pipeline for neuromuscular diseases, including clinical programs for myotonic dystrophy type 1 (DM1) and Duchenne muscular dystrophy (DMD), and preclinical programs for facioscapulohumeral muscular dystrophy (FSHD) and Pompe disease. 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CHARLESTOWN, Mass., April 29, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, will present data from the Phase 1/2 INSPIRE DUCHENNE trial evaluating SGT-003, a next-generation gene therapy product candidate intended for the treatment of Duchenne muscular dystrophy (Duchenne), at the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT), from May 13-17, 2025, in New Orleans, LA. “The data we will present at this year’s ASGCT Annual Meeting reflect Solid’s significant advances in next-generation capsid design and therapeutic approach for Duchenne, including the encouraging initial safety, expression, and biomarker data in the INSPIRE DUCHENNE clinical trial,” said Bo Cumbo, President and CEO of Solid. “We are committed to accelerating safe and effective precision genetic medicines for individuals living with rare neuromuscular and cardiac diseases, and look forward to engaging with the medical and scientific community to advance the field of genetic medicine.” Oral Presentations 1. Title: Initial Experience from the INSPIRE DUCHENNE Phase I/II Study of SGT-003 Microdystrophin Gene Therapy for Duchenne Muscular Dystrophy Session Title: Late-Breaking Abstracts II Date/Time: May 17, 9:15 – 9:30am CT Location/Room: Room 391-392 Presenter: Kevin Flanigan, MD, Advisor Consultant & Researcher, Nationwide Children’s Hospital and investigator in the INSPIRE DUCHENNE clinical trial 2. Title: Insight into the Mechanism of Action of AAV-SLB101, a Novel Muscle-Tropic Capsid for Neuromuscular and Cardiac indications Session Title: AAV Gene Transfer (C): Antibody Evasion, Cardiac & Neuromuscular Targets Date/Time: May 17, 11:30 – 11:45am CT Location/Room: New Orleans Theater A Presenter: Jessica Boehler, PhD, Principal Scientist, Solid Biosciences 3. Title: Potential for AAV-SLB101-Mediated Gene Transfer Treatment in the Context of Natural Seropositivity and After an AAVrh74 Treatment Session Type: Oral presentation Session Title: Modulation of Humoral Immune Responses in AAV Gene Transfer Date/Time: May 16, 1:30 – 3:15pm CT Location/Room: Room 291-292 Presenter: Jessica Boehler, PhD, Principal Scientist, Solid Biosciences Poster Presentations 4. Title: AAV-SLB101, a Novel Muscle-Tropic Capsid, Increases Gene Delivery and Expression Versus AAV9 and AAVrh74 in Mouse Models of DMD and FSHD Muscle Disease Date/Time: May 14, 5:30 – 7:00pm CT Location/Room: Poster Hall I2 Presenter: Prushti Bhavsar, Senior Associate Scientist II, Solid Biosciences 5. Title: Full Length Transgene Quantification Utilizing NanoMosaic Tessie Technology Date/Time: May 13, 6:00 – 7:30pm CT Location/Room: Poster Hall I2 Presenter: Isabella Pajevic, Senior Associate Scientist II, Solid Biosciences 6. Title: Capillary Isoelectric Focusing (cIEF) Platform for Characterization of Charge Variants of Adeno-Associated Virus (AAV) Capsids and Impact on Their Transduction Efficiency Date/Time: May 13, 6:00 – 7:30pm CT Location/Room: Poster Hall I2 Presenter: Brandon Hoyle, Principal Associate Scientist, Solid Biosciences 7. Title: Automation of AAV Capsid ELISA on Tecan Fluent Date/Time: May 14, 5:30 – 7:00pm CT Location/Room: Poster Hall I2 Presenter: Sarah Tong, Associate Scientist, Solid Biosciences Following completion of the conference, presentations will be available on the Scientific Publications & Presentations page of the Our Science section of the Company website, or by clicking here. About Duchenne Duchenne is a genetic muscle-wasting disease predominantly affecting boys, with symptoms usually appearing between three and five years of age. Duchenne is a progressive, irreversible, and ultimately fatal disease that affects approximately one in every 3,500 to 5,000 live male births and has an estimated prevalence of 5,000 to 15,000 cases in the United States alone. About SGT-003 SGT-003 is an investigational gene therapy containing a differentiated microdystrophin construct and a proprietary, next-generation capsid, AAV-SLB101, which was rationally designed to target integrin receptors, and has shown enhanced cardiac and skeletal muscle transduction with decreased liver targeting in nonclinical studies. SGT-003’s microdystrophin construct uniquely includes the R16/17 domains, which localize nNOS to the muscle. Nonclinical studies have shown that nNOS can improve blood flow to the muscle thereby reducing muscle breakdown from ischemia and muscle fatigue. Together, these design features suggest that SGT-003 could be a potential best-in-class investigational gene therapy for the treatment of Duchenne. About INSPIRE DUCHENNE INSPIRE DUCHENNE is a first-in-human, open-label, single-dose, multicenter Phase 1/2 clinical trial to evaluate the safety, tolerability and efficacy of SGT-003 in pediatric participants with a genetically confirmed Duchenne diagnosis with a documented dystrophin gene mutation. INSPIRE DUCHENNE is a multinational trial designed to enroll participants in the United States, Canada, the United Kingdom and Italy. About Solid Biosciences Solid Biosciences is a precision genetic medicine company focused on advancing a portfolio of gene therapy candidates targeting rare neuromuscular and cardiac diseases, including Duchenne muscular dystrophy (Duchenne), Friedreich’s ataxia (FA), catecholaminergic polymorphic ventricular tachycardia (CPVT), TNNT2-mediated dilated cardiomyopathy, BAG3-mediated dilated cardiomyopathy, and additional fatal, genetic cardiac diseases. The Company is also focused on developing innovative libraries of genetic regulators and other enabling technologies with promising potential to significantly impact gene therapy delivery cross-industry. Solid is advancing its diverse pipeline and delivery platform in the pursuit of uniting experts in science, technology, disease management, and care. Patient-focused and founded by those directly impacted by Duchenne, Solid’s mission is to improve the daily lives of patients living with devastating rare diseases. For more information, please visit www.solidbio.com. Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements regarding future expectations, plans and prospects for the Company; the ability to successfully achieve and execute on the Company’s goals, priorities and achieve key clinical milestones; the anticipated benefits of SGT-003; the Company’s SGT-003 clinical program, including planned enrollment and site activations in the INSPIRE DUCHENNE trial, planned regulatory interactions and the potential accelerated approval pathway; and other statements containing the words “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “plan,” “potential,” “predict,” “project,” “should,” “target,” “would,” “working” and similar expressions. Any forward-looking statements are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company’s ability to advance SGT-003, SGT-212, SGT-501, SGT-601, SGT-401 and other preclinical programs and capsid libraries on the timelines expected or at all; obtain and maintain necessary approvals and designations from the FDA and other regulatory authorities; replicate in clinical trials positive results found in preclinical studies and early-stage clinical trials of the Company’s product candidates; replicate preliminary or interim data from early-stage clinicals trials in the final data of such trials; obtain, maintain or protect intellectual property rights related to its product candidates; compete successfully with other companies that are seeking to develop Duchenne, Friedreich’s ataxia and other neuromuscular and cardiac treatments and gene therapies; manage expenses; and raise the substantial additional capital needed, on the timeline necessary, to continue development of SGT-003, SGT-212, SGT-501, SGT-601, SGT-401 and other candidates, achieve its other business objectives and continue as a going concern. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company’s actual results to differ from those contained in the forward-looking statements, see the “Risk Factors” section, as well as discussions of potential risks, uncertainties and other important factors, in the Company’s most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company’s views as of the date hereof and should not be relied upon as representing the Company’s views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. Solid Biosciences Investor Contact: Nicole Anderson Director, Investor Relations and Corporate Communications Solid Biosciences Inc. investors@solidbio.com Media Contact: Glenn Silver FINN Partners glenn.silver@finnpartners.com Source: Solid Biosciences Inc.

Welcome back to Endpoints Weekly! Our team of reporters covered first-quarter earnings results from a handful of companies this week. See below for pharma executives’ comments on tariff exposure, dealmaking and more. We also have an assessment of the industry’s first quarter from Endpoints News’ founding editor John Carroll, who broke down numbers and charts on dealmaking activity, VC investment and more. We’ll be following more Q1 updates next week from Pfizer, Moderna, GSK and others, so stay tuned. And keep an eye out for news from the American Association for Cancer Research Annual Meeting in Chicago, where our reporter Lei Lei Wu is on the ground covering the latest readouts and trends. — Nicole DeFeudis Some of the biggest names in the pharma industry reported their first-quarter earnings this week, including Sanofi, Roche, Merck, Bristol Myers Squibb, Gilead and AbbVie. Tariffs were top of mind for investors , with each company’s executives getting peppered with questions about how President Donald Trump’s trade war would affect the industry. Most opted for a wait-and-see approach , but Roche CEO Thomas Schinecker notably took a proactive stance in warning of a negative impact on dealmaking. Any questions or unknowns about tariffs would make it “more difficult to make financial sense of any M&A deals,” he said Thursday. Roche estimates that four of its medicines make up about 92% of its tariff exposure, though it wouldn’t specify which drugs those are. Here’s a quick rundown of some of the other highlights: FDA Commissioner Marty Makary floated a potential new regulatory pathway for some rare disease treatments. In a SiriusXM interview, Makary highlighted that randomized controlled trials aren’t always feasible for extremely rare diseases. He told Megyn Kelly that drugs could potentially be cleared for use “based on a plausible mechanism” on a “conditional basis,” and the agency would closely monitor patients on the drugs until there’s a clearer signal in the data. He reiterated the pathway in an interview with PBS’ “NewsHour” co-anchor and co-managing editor Amna Nawaz this week. “If you’re treating a condition that only affects 52 kids in the world, we shouldn’t be requiring a randomized controlled trial. We may want to authorize or approve something for that condition based on what I call a plausible mechanism, a plausible scientific mechanism,” Makary said during the interview. Further details remain unclear at this time. The idea echoes work by former CBER Director Peter Marks around accelerated approvals for rare disease gene therapies, as well as a bill from former Sen. Mike Braun (R-IN) and Sen. Kirsten Gillibrand (D-NY) to create a new way for the FDA to grant time-limited provisional approval to rare disease drugs that substantially demonstrate early evidence of safety and efficacy. A Chinese biotech shared preliminary results from the first study to test a CRISPR-based treatment in the brain. Alvin Luk, CEO of HuidaGene Therapeutics, spoke to Endpoints’ Ryan Cross last week about the company’s first clinical trials and its business strategy. What is MECP2 duplication syndrome? It’s a rare neurodevelopmental condition that can cause intellectual disability, movement problems and seizures. HuidaGene gave a low dose of its experimental therapy to a 9-year-old boy with MECP2. Three months later, the boy appeared to have improved eye contact, motor skills and walking ability, and was able to respond to simple directions such as “come to eat” or “go to the bathroom.” The results are still early, but HuidaGene is now considering partnerships that could advance its programs. The company also recently unveiled early results for its experimental CRISPR therapy in Duchenne muscular dystrophy. The DMD and MECP2 therapies were both tested in an investigator-initiated trial, where academic or hospital researchers can move forward with less stringent requirements. But Luk said he plans to take the data to the FDA to get clearance for Phase 1 clinical trials in the US. HuidaGene is working on a broad gene editing toolkit, from Cas12 enzymes for DNA cutting to Cas13 for RNA knockdown to tools for base editing and epigenetic editing. “We can choose which tool is the best for the disease,” Luk said. It was a major factor in his decision to join the company as CEO in 2023. Endpoints’ founding editor John Carroll examined biotech’s first-quarter performance this week, pulling in the numbers and charts from Chris Dokomajilar at DealForma. Carroll notes that despite the XBI falling 27% since Trump won the November election, a massive FDA reorganization and broader HHS layoffs, there are some positive signs under the hood. Most importantly, dealmaking activity remains active, even though IPOs and PIPEs remain cool. Mid- and late-stage assets continue to make attractive takeout options for bigger companies, while licensing deals and other partnerships are holding steady. But a big question remains: Can these trends hold up for the rest of the year as uncertainty lingers from Trump’s tariffs? The number of barely surviving biotechs has reached a crisis level for some investors, and a handful of individuals are looking to do something about it. Nicholas Johnston, a longtime healthcare banker at JP Morgan and Perella Weinberg Partners, recently launched Alis Biosciences, which wants to work with C-suites and boards to make better use of their cash. They estimate over $30 billion in “trapped” capital on the books of an estimated 300 publicly traded biotechs.