SLDB

- Duchenne (SGT-003): First participant dosed in the Phase 3 IMPACT DUCHENNE clinical trial, receipt of positive opinion on the Company’s Pediatric Investigation Plan from the European Medicines Agency and Orphan drug designation from the European Commission mark important advancements in the program’s global development - - Continued progress achieved across the SGT-003 development program, including dosing of 47 participants in Phase 1/2 INSPIRE DUCHENNE clinical trial, and ongoing engagement with the FDA for guidance on a potential accelerated approval pathway for SGT-003 - - Friedreich’s Ataxia (SGT-212): Second participant dosed in the Phase 1b FALCON clinical trial; SGT-212 has been well tolerated with no serious adverse events observed - - CPVT (SGT-501): Company anticipates dosing the first participant in the Phase 1b ARTEMIS clinical trial evaluating SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT) in H2 2026 - - Capital Position: Cash, cash equivalents and available-for-sale securities of $380.7 million at March 31, 2026; the Company’s cash runway is anticipated to extend into H1 2028 - CHARLESTOWN, Mass., May 12, 2026 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today reported financial results for the first quarter ended March 31, 2026, and provided a business update. Bo Cumbo, President and CEO of Solid Biosciences, stated: “The beginning of 2026 has seen Solid make meaningful strides as we continue to execute across our pipeline and platform development efforts. In Duchenne, we dosed the first participant in our Phase 3 IMPACT DUCHENNE clinical trial for SGT-003 and received Orphan drug designation from the European Commission, representing important milestones in the program’s global advancement. We also continue to enroll and dose participants in the Phase 1/2 INSPIRE DUCHENNE clinical trial, which has maintained a consistent and encouraging safety and tolerability profile. “As we progress through the year, we look forward to ongoing engagement with the FDA, focusing on our thoughtful approach to generating rigorous, well-controlled clinical data through our integrated, multi-country, multi-trial development program. We recognize the significant unmet need in D...

- Duchenne (SGT-003): Positive interim Phase 1/2 INSPIRE DUCHENNE data reported at the 2026 MDA Annual Meeting continued to suggest differentiated clinical, safety and tolerability profiles; SGT-003 has been generally well tolerated in the 41 participants dosed as of March 18, 2026 - - Company anticipates dosing the first participant in the Phase 3 IMPACT DUCHENNE trial in April 2026; additional meetings planned with FDA in H1 2026 for guidance on a potential accelerated approval pathway for SGT-003 - - Friedreich’s Ataxia (SGT-212): First participant dosed in the Phase 1b FALCON trial; SGT-212 has been well tolerated with no treatment-related adverse events - - CPVT (SGT-501): Clinical sites activated for Phase 1b ARTEMIS first-in-human clinical trial evaluating SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia (CPVT); first participant dosing expected in Q2 2026 - - Strong Capital Position: Cash, cash equivalents and available-for-sale securities of $187.9 million at December 31, 2025; in March 2026, the Company completed a $240 million private placement; the Company’s cash runway is anticipated to extend into H1 2028 - CHARLESTOWN, Mass., March 19, 2026 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today reported financial results for the fourth quarter and full year ended December 31, 2025, and provided a business update. Bo Cumbo, President and CEO of Solid Biosciences, commented, “We continue to build momentum across our portfolio, highlighted by updated interim INSPIRE DUCHENNE data presented at MDA, positive feedback from our FDA Type C meeting on the Phase 3 IMPACT DUCHENNE trial design for SGT-003, the dosing of our first participant in the FALCON trial for SGT-212 and the completion of an oversubscribed $240 million private placement. Together, these achievements strengthen our operational and financial foundation and position Solid to deliver on the promise of our science and improve the daily lives of people living with devastating rare diseases. “Looking to the year ahead, we expect several important milestones, including continued engagement with the FDA to seek guidance on a potential accelerated approval pathway for SGT-003 for the treatment of Duchenne, dosing the fir...

Solid Biosciences (SLDB) reported updated “positive” interim data from the ongoing Phase 1/2 INSPIRE DUCHENNE clinical trial of SGT-003. The new data, including additional muscle biopsy, serum biomarker, cardiac function, and safety analyses, “add to a growing dataset that suggests the potential of the biological activity of SGT-003 microdystrophin therapy,” the company stated. The interim update was delivered in an oral presentation at the 2026 MDA Annual Meeting on March 11. “SGT-003 has been generally well tolerated in the 40 participants dosed as of March 11, 2026. The safety and tolerability profile observed in the INSPIRE DUCHENNE trial continued to be promising; SGT-003 is administered using a low-burden, steroid-only prophylactic immunomodulation regimen. The trial is being conducted at 15 clinical sites across the US, Canada, Italy and the United Kingdom and participant dosing remains ongoing. Microdystrophin transduction and expression levels, beta-sarcoglycan localization and nNOS activity were evaluated by biopsy in 20 participants (ages 1-10 years) at Day 90 and in 3 participants at Day 360. Results demonstrated robust mean vector copies per nucleus and microdystrophin expression as well as properly localized and restored beta-sarcoglycan-positive fibers and nNOS activity-positive fibers,” the company stated. Bo Cumbo, President and CEO of Solid Biosciences commented, “These data continue to suggest that SGT-003 may be having a disease-relevant treatment effect, with observed robust microdystrophin expression, restoration of key components of the dystrophin-associated protein complex suggesting a direct biologic correlate of dystrophin activity, and improvements across multiple biomarkers of muscle integrity. Together with the promising safety and tolerability profile, these interim results reinforce our confidence in the potential of SGT-003 to meaningfully impact the disease course of Duchenne. Duchenne continues to have an unmet medical need. We are pursuing guidance on a potential accelerated approval pathway for SGT-003 and look forward to continued engagement with the FDA as we work toward that goal. Our focus remains on urgently advancing SGT-003 to provide the Duchenne community with an additional therapeutic choice.” Unlock hedge fund-level data and powerful investing tools for smarter, sharper decisions Stay ahead of the market with th...

Solid Biosciences (SLDB) on Wednesday reported interim results from its phase 1/2 trial of SGT-003,

Sarepta Therapeutics stock jumped after the company unveiled positive top-line results for a study measuring the efficacy of Elevidys, its controversial blockbuster treatment for Duchenne muscular dystrophy. Patients in the study showed “statistically significant, clinically meaningful and durable efficacy across all key motor function measures” three years after treatment, Sarepta said. The mean North Star Ambulatory Assessment score, a 17-item scale that grades functional motor abilities in patients, remained above baseline for the treated group, while the external control group “continued to show the expected age-related decline below their baseline score,” Sarepta said.

- Duchenne (SGT-003): 23 participants have been dosed in the INSPIRE DUCHENNE trial as of October 31, 2025; Solid expects to have dosed 30 participants in total by early 2026, then plans to meet with the FDA to discuss potential registrational pathways in H1 2026 - - SGT-003 has been generally well tolerated using a steroid-only prophylactic immunomodulation regimen; cardiac safety monitoring continued to show reduction in cardiac injury and early signals of cardiac function normalization - - Day 90 biopsy data from 10 treated participants (ages 5-10) showed all participants responded to treatment with mean microdystrophin expression of 58% by western blot, 58% by mass spectrometry, mean microdystrophin positive fibers of 51% by immunofluorescence, and mean beta-sarcoglycan positive fibers of 50% by immunofluorescence - - Strong statistical correlations observed between Day 90 microdystrophin expression levels and key components of the dystrophin-associated protein complex (DAPC), beta-sarcoglycan (r = 0.95) and nNOS (r = 0.95), demonstrated evidence of SGT-003-mediated DAPC restoration and concordant signals of muscle protection via reductions in CK levels (r = -0.78) - - Solid has activated the first clinical trial site and is currently screening participants for IMPACT DUCHENNE, a Phase 3 randomized, double-blind, placebo-controlled ex-U.S. clinical trial of SGT-003 - - FA (SGT-212): Solid has activated the first clinical trial site and is currently screening participants for FALCON, a Phase 1b first-in-human clinical trial evaluating SGT-212 for the treatment of Friedreich’s ataxia - - CPVT (SGT-501): Clinical trial site activation for ARTEMIS, a Phase 1b first-in-human clinical trial evaluating SGT-501 for the treatment of catecholaminergic polymorphic ventricular tachycardia expected in Q4 2025 - - Capsids (AAV-SLB101): Over 30 agreements including licenses executed with corporations, institutions, and academic labs for the use of AAV-SLB101 - - Cash: Company ended Q3 2025 with $236.1 million in cash, cash equivalents and available-for-sale securities; Solid has anticipated cash runway into H1 2027 - CHARLESTOWN, MA, Nov. 03, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today reported financial results for...

- Duchenne (SGT-003): 15 participants dosed in the Phase 1/2 INSPIRE DUCHENNE trial and dosing remains ongoing; On track to discuss regulatory pathways with U.S. FDA in Q4 2025 - - FA (SGT-212): Phase 1b trial initiation expected in Q4 2025 - - CPVT (SGT-501): Phase 1b trial initiation expected in Q4 2025 - - Capsids (AAV-SLB101): Over 25 agreements or licenses with academic labs, institutions and corporations for the use of AAV-SLB101 have been executed - - Cash: Company ended Q2 2025 with $268.1 million in cash, cash equivalents and available-for-sale securities; Solid has anticipated cash runway into H1 2027 - CHARLESTOWN, Mass., Aug. 12, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision, next generation, genetic medicines for neuromuscular and cardiac diseases, today reported financial results for the second quarter ended June 30, 2025, and provided a business update. Bo Cumbo, President and CEO of Solid, commented: “With three clinical-stage programs for neuromuscular and cardiac diseases, Solid now stands at the forefront of innovation in genetic medicine. The strategic expansion of our pipeline beyond Duchenne to include differentiated therapies for both FA and CPVT marks a meaningful stride forward that reflects both our disciplined execution and steadfast dedication to the patient communities we serve. “We continue to receive overwhelming interest in the INSPIRE DUCHENNE trial from families and the clinical community and now have 10 active clinical sites and more than 20 additional participants identified to potentially enter the INSPIRE DUCHENNE trial. As of August 12, 2025, we have dosed 15 participants and SGT-003 continues to be well tolerated, with no treatment emergent SAEs observed, and we continue to recruit and dose patients. We look forward to FDA discussions later this year and will continue to work diligently to advance a new gene therapy option for the Duchenne community,” Mr. Cumbo concluded. Company Updates Neuromuscular Pipeline SGT-003 for Duchenne Muscular Dystrophy (Duchenne) As of August 12, 2025, 15 participants have been dosed in the INSPIRE DUCHENNE trial with participant dosing ongoing across multiple cohorts; SGT-003 continues to be well tolerated with no treatment emergent SAEs observed and an immune suppression regimen that consists of st...

- Duchenne (SGT-003): Participant dosing ongoing in the Phase 1/2 INSPIRE DUCHENNE trial; Solid on track to discuss accelerated pathways with U.S. FDA later in 2025 - - FA (SGT-212): Dosing of first participant anticipated in the second half of 2025 - - CPVT (SGT-501): FDA IND on track for submission first half of 2025 - - Cash: Company ended first quarter 2025 with $306.9 million in cash, cash equivalents, investments and available-for-sale securities; Solid has anticipated cash runway into the first half of 2027 - CHARLESTOWN, Mass., May 15, 2025 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the “Company” or “Solid”), a life sciences company developing precision, next generation, genetic medicines for neuromuscular and cardiac diseases, today reported financial results for the first quarter ended March 31, 2025, and provided a business update. Bo Cumbo, President and CEO of Solid, commented: “The positive initial three patient, 90-day data in the INSPIRE DUCHENNE trial of SGT-003, our next-generation, investigational gene therapy to treat Duchenne muscular dystrophy, were a milestone in the development of a meaningful treatment candidate for this terrible disease. With the initial data demonstrating robust microdystrophin expression and improvements across biomarkers of muscle integrity and, excitingly, encouraging biomarkers of cardiac and liver health, we continue to dose participants as we prepare to engage with the FDA later this year on the potential for accelerated pathways. “To our patient communities, we know that safety is a critical factor in choosing any medical therapy. SGT-003, utilizing our proprietary, rationally designed capsid, AAV-SLB101, contains the lowest dose of any Duchenne gene therapy currently marketed or in clinical development, and to date, has demonstrated an encouraging safety and tolerability profile. We look forward to the 28th Annual Meeting of the American Society of Gene and Cell Therapy (ASGCT) where, on Saturday, we will provide an interim safety update from the INSPIRE DUCHENNE trial during our late-breaking oral presentation. “As an organization, we are intensely focused on driving innovation and advancing the C&GT field with the aspiration to make gene therapies safer, more effective, and truly accessible. These efforts originate with our differentiated and thoughtfully designed neuromuscular and cardiac...