Company Overview - Solid Biosciences is a precision genetic medicine company with approximately 120 employees, primarily focusing on gene therapies [2] - The company has three main drug candidates: one for Duchenne Muscular Dystrophy, another for Friedreich’s Ataxia, and a third for Catecholaminergic Polymorphic Ventricular Tachycardia (CPVT) [2] Product Pipeline - The next program in development targets dilated cardiomyopathies, specifically TNNT2 [3] - Solid Biosciences is building a comprehensive capsid library and platform to enhance gene therapy delivery, focusing on capsids, promoters, dual plasmids, and manufacturing purity [3]

Solid Biosciences (NasdaqGS:SLDB) 2026 Conference February 12, 2026 10:30 AM ET Company ParticipantsBo Cumbo - President and CEOConference Call ParticipantsDebjit Chattopadhyay - Therapeutic AnalystDebjit ChattopadhyayTwenty twenty six Emerging Outlook Biotech Summit. I am Debjit. I'm one of the therapeutic analysts, and my privilege to welcome our next presenting company, Solid Biosciences. Joining from Solid is Bo Cumbo, President and CEO. Thank you so much for your time, Bo.Bo CumboYeah, thank you, Debji ...

Group 1 - The article discusses Solid Biosciences Inc. (SLDB) and its differentiating factors related to SGT-212, highlighting the company's unique position in the biotech sector [2] - The author, Terry Chrisomalis, has extensive experience in the biotech industry and runs a service that provides in-depth analysis of pharmaceutical companies [2] - The Biotech Analysis Central service includes a library of over 600 biotech investing articles and a model portfolio of more than 10 small and mid-cap stocks [2] Group 2 - The article does not contain any stock or derivative positions in the companies mentioned, nor does it plan to initiate any such positions in the near future [3] - The article emphasizes that past performance is not indicative of future results and does not provide specific investment recommendations [4]

Group 1 - The article discusses Solid Biosciences Inc. (SLDB) and its differentiating factors related to SGT-212, highlighting the company's unique position in the biotech sector [2] - The author, Terry Chrisomalis, has extensive experience in the biotech industry and runs a service that provides in-depth analysis of pharmaceutical companies [2] - The Biotech Analysis Central service includes a library of over 600 biotech investing articles and a model portfolio of more than 10 small and mid-cap stocks [2] Group 2 - The article does not contain any stock or derivative positions in the companies mentioned, nor does it plan to initiate any positions in the near future [3] - There is no recommendation or advice provided regarding the suitability of investments for particular investors [4]

Core Insights - Solid Biosciences has aligned with the FDA on the Phase 3 trial design for SGT-003, a potential treatment for Duchenne muscular dystrophy [1][2] - The company anticipates the first participant dosing in the Phase 3 trial, IMPACT DUCHENNE, in Q1 2026 [1] - Solid plans to hold additional meetings with the FDA in the first half of 2026 to discuss a potential accelerated approval pathway for SGT-003 [1][3] Clinical Trials - The Phase 3 trial, IMPACT DUCHENNE, will be a randomized, double-blind, placebo-controlled study targeting ambulant participants aged 7 to less than 12 years [2][8] - The primary endpoint for the trial is the change from baseline in Time to Rise (TTR) velocity evaluated at 18 months, along with other key secondary endpoints [2] - The ongoing Phase 1/2 trial, INSPIRE DUCHENNE, has dosed 36 participants as of February 9, 2026, and SGT-003 has been generally well tolerated [4] Regulatory Strategy - The successful alignment with the FDA on the Phase 3 trial design is seen as a critical step in the company's regulatory strategy for SGT-003 [3] - The company is evaluating the potential to open trial sites in the U.S. due to strong demand from key opinion leaders and patients [3] Product Information - SGT-003 is an investigational gene therapy designed to treat Duchenne muscular dystrophy, featuring a differentiated microdystrophin construct and a proprietary capsid [6] - The therapy aims to enhance cardiac and skeletal muscle transduction while reducing liver targeting, potentially making it a best-in-class option for Duchenne treatment [6] Disease Background - Duchenne muscular dystrophy is a genetic muscle-wasting disease primarily affecting boys, with symptoms typically appearing between ages three and five [5] - The disease has an estimated prevalence of 5,000 to 15,000 cases in the United States, affecting approximately one in every 3,500 to 5,000 live male births [5]

Core Insights - Solid Biosciences Inc. is a life sciences company focused on developing precision genetic medicines for neuromuscular and cardiac diseases [3] - The company will present at the Guggenheim Emerging Outlook: Biotech Summit 2026 on February 12, 2026, at 10:30 AM ET [1] Company Overview - Solid Biosciences is advancing a portfolio of gene therapy candidates targeting rare diseases, including SGT-003 for Duchenne muscular dystrophy, SGT-212 for Friedreich's ataxia, SGT-501 for catecholaminergic polymorphic ventricular tachycardia, and SGT-601 for TNNT2-mediated dilated cardiomyopathy [3] - The company is also developing innovative libraries of genetic regulators and technologies to enhance gene therapy delivery across industries [3] - Founded by individuals affected by Duchenne, the company's mission is to improve the daily lives of patients with rare diseases [3]

Company Overview - Solid Biosciences has successfully dosed 33 patients with no reported drug-induced liver injury, myocarditis, or aHUS, indicating a strong safety profile for their treatments [2] - The company has enrolled its first patient in a double-blind, placebo-controlled study, marking a significant milestone in their clinical development [3] Business Development - Solid Biosciences has established 50 different agreements for their capsids, showcasing their growing reputation as a platform technology company [3] - The company is increasingly recognized for its delivery vehicles, which are being utilized in next-generation therapies, reflecting a positive trend in collaboration and innovation [3]

Summary of Solid Biosciences FY Conference Call Company Overview - **Company**: Solid Biosciences (NasdaqGS:SLDB) - **Event**: 44th Annual J.P. Morgan Healthcare Conference - **Date**: January 13, 2026 - **CEO**: Bo Cumbo Key Points Industry and Company Developments - Solid Biosciences has dosed 33 patients in its phase 1/2 Duchenne muscular dystrophy (DMD) trial called Inspire, reporting no drug-induced liver injury, myocarditis, or AHUS [2][30] - The company has successfully enrolled its first patient in a double-blind placebo-controlled trial and dosed its first patient in Friedreich's ataxia (FA) [2][5] - Solid has established over 50 partnerships for its capsid technology, indicating a growing interest in its platform for next-generation therapies [3][6] Clinical Trials and Pipeline - The Inspire trial aims to enroll 50 to 60 patients, with 33 already dosed and full enrollment expected soon [8][31] - The company is prioritizing FA over CPVT (catecholaminergic polymorphic ventricular tachycardia) due to resource constraints, with plans to dose patients in CPVT this quarter [5][6] - The first patient in the double-blind placebo-controlled trial will be dosed in either Australia or Canada, with European sites also opening [9][32] Safety and Efficacy Data - The safety profile of the therapy appears robust, with 0% drug-induced liver injury and other serious adverse events reported [11][30] - The mean expression of microdystrophin in treated patients is reported at 58%, the highest among current therapies [10][23] - Early cardiac data shows declines in troponin levels and increases in ejection fraction, suggesting potential cardiac benefits [11][28] Capsid Technology - The capsid SLB101 is designed for enhanced binding to muscle tissue, leading to improved distribution and reduced liver targeting [12][19] - The capsid technology is noted for its low viral load (1E14), which is lower than other therapies, potentially leading to fewer side effects [14][40] - The capsid's unique design allows for better muscle perfusion and reduced fibrosis, contributing to the observed clinical benefits [13][19] Market Opportunity - The DMD market is expanding, with approximately 100 boys born with the disease each quarter, indicating a high demand for new therapies [36] - Solid aims to be a leader in the DMD space, with plans for accelerated approval pathways and potential first gene therapy to market in the UK [36][44] Regulatory Strategy - The company is preparing for multiple meetings with the FDA to discuss the phase 3 placebo-controlled trial and the potential for accelerated approval [33][44] - Solid has not reviewed any functional data to maintain the integrity of its discussions with the FDA, ensuring confidence in its regulatory submissions [34][45] Future Milestones - Solid plans to continue dosing patients and gathering data throughout the year, with significant milestones expected that could transform the company [38][39] - The company is focused on ensuring that its therapies are well-positioned for market entry and regulatory approval, with ongoing studies to track long-term outcomes [29][44] Additional Insights - The dual-route administration in FA is a significant milestone, showcasing the company's innovative approach to treatment delivery [37] - The emphasis on safety and efficacy data, along with a strong pipeline, positions Solid Biosciences as a key player in the gene therapy landscape for muscular dystrophies [12][30]

Corporate Presentation January 2026 © 2026 Solid Biosciences Cautionary Note Regarding Forward Looking Statements This presentation 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 key clinical and preclinical milestones; strategies and expectations for the company's SGT ...

Core Insights - Solid Biosciences is advancing its gene therapy programs for neuromuscular and cardiac diseases, with significant progress reported in multiple clinical trials as of January 2026 [1][2][3] Neuromuscular Pipeline - SGT-003 for Duchenne muscular dystrophy has been well tolerated in 33 participants dosed in the Phase 1/2 INSPIRE DUCHENNE trial, with no serious adverse events reported as of January 9, 2026 [3][4] - The company plans to engage with the FDA in the first half of 2026 to discuss the design of a Phase 3 confirmatory trial for SGT-003, aiming for potential accelerated approval [3][4] - The first participant has been enrolled in the Phase 3 IMPACT DUCHENNE trial, with dosing expected in Q1 2026 [1][7] - SGT-212 for Friedreich's ataxia has received FDA Orphan Drug designation and the first participant has been dosed in the Phase 1b FALCON trial, which is the first gene therapy for FA utilizing a dual route of administration [1][7] Cardiac Pipeline - SGT-501 is being evaluated in the ARTEMIS Phase 1b trial for catecholaminergic polymorphic ventricular tachycardia, with participant screening currently underway [1][7] - Initial safety data from the ARTEMIS trial is expected in the second half of 2026 [7] Platform Technologies - Solid Biosciences has executed over 50 agreements for the use of its proprietary next-generation capsid AAV-SLB101, which aims to enhance gene therapy delivery [1][5][7] - AAV-SLB101 has shown promising results in terms of safety and efficacy in the ongoing trials [7]