Core Insights - Solid Biosciences has entered into a non-exclusive worldwide license and collaboration agreement with Andelyn Biosciences for the use of its proprietary capsid, AAV-SLB101, aimed at enhancing gene therapy development [1][2] Group 1: Agreement Details - The agreement allows Andelyn to utilize AAV-SLB101 in conjunction with its AAV Curator® Platform, which optimizes gene therapy manufacturing processes [2] - Financial terms of the agreement have not been disclosed [2] Group 2: AAV-SLB101 Characteristics - AAV-SLB101 is designed for improved skeletal muscle and cardiac tropism while minimizing liver biodistribution [3][5] - The capsid has shown good tolerability in a Phase 1/2 clinical trial involving 23 pediatric participants, with safety data reported as of October 31, 2025 [3] Group 3: Company Statements - The collaboration is expected to accelerate the development of next-generation gene therapies, enhancing safety and efficacy [4] - Andelyn aims to integrate AAV-SLB101 into its platform to provide advanced gene therapy vectors, potentially leading to faster and more cost-effective development [4] Group 4: Company Background - Solid Biosciences focuses on developing precision genetic medicines for rare neuromuscular and cardiac diseases, including SGT-003 for Duchenne muscular dystrophy [6][7] - Andelyn Biosciences specializes in the development and production of viral vectors for gene therapy, with extensive experience in clinical batch production [8]

Core Insights - Solid Biosciences has received an Innovation Passport for its investigational gene therapy SGT-003, aimed at treating Duchenne muscular dystrophy, under the new Innovative Licensing and Access Pathway (ILAP) in the UK [1][2][3] - The ILAP is designed to accelerate the development and access of transformative medicines, facilitating early engagement with regulatory bodies [4] - SGT-003 is currently undergoing clinical trials, including the Phase 1/2 INSPIRE DUCHENNE trial and the Phase 3 IMPACT DUCHENNE trial, with the aim of becoming the first gene therapy for Duchenne in the UK [3][8][9] Company Overview - Solid Biosciences is focused on developing precision genetic medicines for neuromuscular and cardiac diseases, with a portfolio that includes SGT-003 for Duchenne muscular dystrophy [10] - The company aims to improve the lives of patients with rare diseases through innovative gene therapy solutions [10] Clinical Development - SGT-003 is designed with a differentiated microdystrophin construct and a proprietary capsid, AAV-SLB101, which targets integrin receptors and enhances muscle transduction [7] - The ongoing INSPIRE DUCHENNE trial is evaluating the safety and efficacy of SGT-003 in pediatric participants with Duchenne, while the IMPACT DUCHENNE trial is focused on supporting regulatory authorizations outside the US [8][9] Regulatory Framework - The ILAP, launched in 2021, provides a unique end-to-end regulatory access pathway, allowing for early multi-stakeholder engagement and support for clinical development and market access [4] - The Innovation Passport designation enables Solid Biosciences to work closely with the UK's MHRA and other regulatory bodies to expedite the development of SGT-003 [1][3]

Financial Performance - The company reported net losses of $45.8 million and $124.5 million for the three and nine months ended September 30, 2025, respectively, compared to $32.7 million and $82.1 million for the same periods in 2024[108]. - As of September 30, 2025, the company had an accumulated deficit of $908.0 million and expects to incur significant expenses and operating losses for the foreseeable future[108]. - The net loss for the nine months ended September 30, 2025, was $124.5 million, representing a 51.7% increase from a net loss of $82.1 million in the same period of 2024[131]. - Cash used in operating activities was $102.7 million for the nine months ended September 30, 2025, compared to $70.4 million in the same period of 2024[141]. - Other income, net decreased to $5.3 million, down 32.5% from $7.7 million in the previous year, primarily due to changes in the fair value of derivative liabilities[135]. Research and Development - The company has not generated any commercial product revenue to date and does not expect to do so in the foreseeable future[112]. - Research and development expenses for Q3 2025 were $38.9 million, a 42.2% increase from $27.3 million in Q3 2024[125]. - The allocated research and development expenses for SGT-003 increased by 242.8% to $18.1 million in Q3 2025 from $5.3 million in Q3 2024[126]. - Total research and development expenses for the nine months ended September 30, 2025, were $102.2 million, a 55.6% increase from $65.7 million in the same period of 2024[132]. - The total allocated research and development expenses increased by 88.2% to $62.0 million, driven primarily by a $30.1 million increase in costs for SGT-003[132]. Clinical Trials - The FDA has granted Fast Track, Orphan Drug, and Rare Pediatric Disease designations for SGT-003 for the treatment of Duchenne muscular dystrophy[101]. - Interim data from the Phase 1/2 INSPIRE DUCHENNE trial showed a mean microdystrophin expression of 58% and mean microdystrophin positive fibers of 51%[96]. - As of October 31, 2025, 23 participants have been dosed in the INSPIRE DUCHENNE trial, with one treatment-related serious adverse event reported[97]. - The company anticipates that enrollment in the INSPIRE DUCHENNE trial will reach 30 participants by early 2026[98]. - The company activated the first clinical trial site for the Phase 3 IMPACT DUCHENNE trial in October 2025, which aims to support potential ex-U.S. regulatory authorizations[100]. Financial Position - The company had cash, cash equivalents, and available-for-sale securities of $236.1 million as of September 30, 2025, expected to fund operations into the first half of 2027[111]. - The company raised approximately $188.0 million in net proceeds from the February 2025 Offering, selling 35,739,810 shares at $4.03 per share[139]. - The company anticipates needing to raise additional funds to continue operations beyond the first half of 2027[149]. - As of September 30, 2025, the company had cash, cash equivalents, and available-for-sale securities totaling $236.1 million, excluding restricted cash of $1.9 million[140]. - Cash provided by financing activities increased to $189.3 million, up from $115.5 million in the prior year, reflecting successful capital raises[146]. Expenses - Total operating expenses for the nine months ended September 30, 2025, were $129.8 million, a 44.5% increase from $89.8 million in the same period of 2024[131]. - General and administrative expenses for Q3 2025 were $9.2 million, up 17.1% from $7.9 million in Q3 2024[129]. - General and administrative expenses rose to $27.6 million, a 14.1% increase from $24.2 million in the prior year[134]. - Personnel-related expenses in unallocated research and development increased by 43.8% to $9.0 million in Q3 2025 from $6.3 million in Q3 2024[126]. - The company expects general and administrative expenses to increase in the future to support ongoing clinical trials and commercialization efforts[119]. Market and Economic Conditions - Inflation has not had a material effect on the business in the last two years, but future operations may be adversely affected[161]. - An immediate 10% change in market interest rates would not have a material impact on the fair market value of the investment portfolio[160]. - Cash equivalents as of September 30, 2025, consisted of money market funds and treasury bills with maturities of less than 90 days[160]. - Investments as of September 30, 2025, included treasury bills and government bonds with maturities of less than one year[160]. - No material changes to contractual obligations and commitments during the nine months ended September 30, 2025[157].

Clinical Trials and Research - As of October 31, 2025, 23 participants have been dosed in the INSPIRE DUCHENNE trial, with an expectation to dose a total of 30 participants by early 2026[1][6] - The company activated the first clinical trial site for FALCON, a Phase 1b trial for SGT-212, and expects to activate the first site for ARTEMIS, a Phase 1b trial for SGT-501, in Q4 2025[1][4] - The interim data from the INSPIRE DUCHENNE trial suggests a coordinated systemic response to treatment, reinforcing confidence in SGT-003's therapeutic potential[1][3] - Solid activated the first clinical trial site for the Phase 3 IMPACT DUCHENNE trial in October 2025, assessing SGT-003 in pediatric participants outside the U.S.[28] - The company plans to expand the IMPACT DUCHENNE trial into additional countries, having already received regulatory approvals in Canada and Australia[28] - SGT-212, the first investigational gene therapy for Friedreich's Ataxia, began participant screening in October 2025, with the trial designed to evaluate safety and tolerability[29] Financial Performance - The company ended Q3 2025 with $236.1 million in cash, cash equivalents, and available-for-sale securities, with an anticipated cash runway into H1 2027[1][2] - As of September 30, 2025, Solid had $236.1 million in cash, cash equivalents, and available-for-sale securities, an increase from $148.9 million as of December 31, 2024, enabling operational funding into the first half of 2027[35] - Research and Development (R&D) expenses for Q3 2025 were $38.9 million, up from $27.3 million in Q3 2024, primarily due to a $12.8 million increase in costs for SGT-003 related to manufacturing, regulatory, and clinical expenses[39] - General and Administrative (G&A) expenses for Q3 2025 were $9.2 million, compared to $7.9 million in Q3 2024, driven by increased personnel-related costs and legal fees[39] - The net loss for Q3 2025 was $45.8 million, compared to a net loss of $32.7 million in Q3 2024[39] - Total operating expenses for the nine months ended September 30, 2025, were $129.8 million, compared to $89.8 million for the same period in 2024[47] Treatment Outcomes - Day 90 biopsy data from 10 treated participants showed mean microdystrophin expression of 58% by western blot and mass spectrometry, with mean microdystrophin positive fibers at 51%[1][12] - Strong statistical correlations were observed between Day 90 microdystrophin expression levels and key components of the dystrophin-associated protein complex, with a Pearson correlation of 0.95 for both beta-sarcoglycan and nNOS[1][9] - Mean reductions in serum creatine kinase (CK) of 34% at Day 90 and 42% at Day 360 were observed, indicating muscle injury reduction[1][15] - Mean cardiac function trended into normal left ventricular ejection fraction (LVEF) ranges (60-69%) for all SGT-003-treated participants who reached the Day 180 follow-up[1][16] Regulatory and Development Plans - The company plans to meet with the FDA in H1 2026 to discuss potential registrational pathways for SGT-003[1][3] - The company anticipates completing PPQ manufacturing batches for SGT-003 in 2026, aligning with FDA discussions on registrational pathways[27] - Solid has executed over 30 agreements for the use of its proprietary AAV-SLB101 capsid, with more expected by year-end[34] Adverse Events - One treatment-related serious adverse event (SAE) was reported, identified as Grade 3 immune-mediated myositis, which resolved with steroid treatment[1][22]

Core Insights - Solid Biosciences reported positive interim data from the Phase 1/2 INSPIRE DUCHENNE trial for SGT-003, indicating its therapeutic potential for Duchenne muscular dystrophy [1][2][26] - The company plans to meet with the FDA in the first half of 2026 to discuss potential registrational pathways for SGT-003, including accelerated approval options [26][27] - Solid has also activated clinical trial sites for other gene therapy candidates, SGT-212 for Friedreich's ataxia and SGT-501 for catecholaminergic polymorphic ventricular tachycardia [29][31] Clinical Trial Updates - As of October 31, 2025, 23 participants have been dosed in the INSPIRE DUCHENNE trial, with plans to dose a total of 30 participants by early 2026 [1][6] - The trial has shown strong statistical correlations between microdystrophin expression levels and key components of the dystrophin-associated protein complex, indicating effective treatment [7][11] - The first clinical trial site for IMPACT DUCHENNE, a Phase 3 trial for SGT-003, has been activated, with plans to expand into additional countries [28] Safety and Efficacy Data - SGT-003 has been generally well tolerated, with a steroid-only prophylactic immunomodulation regimen showing minimal adverse effects [21][22] - Day 90 biopsy data indicated a mean microdystrophin expression of 58% and significant reductions in biomarkers of muscle injury, such as serum creatine kinase (CK) [11][14] - Cardiac function improvements were observed, with mean reductions in serum cardiac troponin I levels and normalization of left ventricular ejection fraction [17][18] Financial Position - As of September 30, 2025, Solid Biosciences reported $236.1 million in cash and equivalents, providing a cash runway into the first half of 2027 [35] - Research and development expenses for Q3 2025 were $38.9 million, reflecting increased costs associated with SGT-003 [35][36] - The net loss for Q3 2025 was $45.8 million, compared to $32.7 million for the same period in 2024 [44]

© 2025 Solid Biosciences November 2025 SGT-003 INSPIRE DUCHENNE DATA UPDATE 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 th ...

Core Insights - Solid Biosciences Inc. is set to present data from its neuromuscular and cardiac programs at two major conferences in October 2025, showcasing the potential of its innovative pipeline and proprietary capsid, AAV-SLB101 [1][2] Conference Presentations - At the World Muscle Society (WMS) 2025 Annual International Congress, the company will present an oral update on the INSPIRE DUCHENNE trial, focusing on SGT-003, a next-generation microdystrophin gene therapy for Duchenne Muscular Dystrophy [2][3] - A poster presentation at WMS will evaluate muscle integrity biomarkers to assess therapeutic efficacy in Duchenne Muscular Dystrophy [3] - At the European Society of Gene & Cell Therapy (ESGCT) 2025 Annual Congress, the company will present on SGT-003 and its innovative AAV-SLB101 capsid [3][6] Company Overview - Solid Biosciences is focused on developing precision genetic medicines targeting rare neuromuscular and cardiac diseases, with a diverse pipeline including SGT-003 for Duchenne muscular dystrophy, SGT-212 for Friedreich's ataxia, and SGT-501 for catecholaminergic polymorphic ventricular tachycardia [5][7] - The company aims to advance its gene therapy candidates and delivery technologies to significantly impact the field of gene therapy [5][7]

Core Insights - Solid Biosciences Inc. is presenting data from the Phase 1/2 INSPIRE DUCHENNE trial for SGT-003, a gene therapy for Duchenne muscular dystrophy, at the NMSG Annual Meeting in Italy [1][2] - The company emphasizes the favorable profile of SGT-003, particularly its liver tolerability and early biomarker signals, supported by a steroid-only immunomodulation regimen [2] - Solid Biosciences has established over 25 licensing agreements for the use of its next-generation capsid, AAV-SLB101, which is designed for targeted gene therapy [2] Company Overview - Solid Biosciences focuses on developing precision genetic medicines for rare neuromuscular and cardiac diseases, including SGT-003 for Duchenne muscular dystrophy [6][7] - The company aims to improve the lives of patients affected by devastating rare diseases, with a mission rooted in personal experiences with Duchenne [7] Product Details - SGT-003 is an investigational gene therapy featuring a differentiated microdystrophin construct and the proprietary AAV-SLB101 capsid, designed to enhance muscle transduction while minimizing liver targeting [4] - The microdystrophin construct includes R16/17 domains, which may improve blood flow to muscles, potentially reducing muscle breakdown and fatigue [4] Clinical Trial Information - INSPIRE DUCHENNE is a first-in-human, open-label, single-dose, multicenter Phase 1/2 clinical trial assessing the safety, tolerability, and efficacy of SGT-003 in pediatric patients with Duchenne [5] - The trial is multinational, enrolling participants from the United States, Canada, the United Kingdom, and Italy [5]

Core Insights - Solid Biosciences has entered into a non-exclusive worldwide license and collaboration agreement with Kinea Bio for the use of its proprietary capsid, AAV-SLB101, to develop KNA-155, a gene therapy targeting dysferlinopathy [1][2] - Solid will receive an upfront payment and is eligible for additional milestone payments and tiered royalties on net sales [2] - AAV-SLB101 is designed for enhanced muscle tropism and reduced liver biodistribution, showing promising results in preclinical studies and early clinical trials [3][7] Company Overview - Solid Biosciences focuses on developing precision genetic medicines for neuromuscular and cardiac diseases, with a diverse pipeline including therapies for Duchenne muscular dystrophy and other rare diseases [8][9] - The company aims to license AAV-SLB101 broadly to various institutions and companies pursuing treatments for rare diseases, having already established over 25 agreements [7] Collaboration Details - The agreement allows Kinea Bio to utilize AAV-SLB101 as the delivery mechanism for KNA-155, which is currently advancing into IND-enabling preclinical activities [2] - Both companies express confidence in the potential of AAV-SLB101 to deliver safe and effective therapies for patients with dysferlinopathies [6]

Summary of Solid Biosciences Conference Call Company Overview - **Company**: Solid Biosciences (NasdaqGS:SLDB) - **Focus**: Precision genetic medicine, primarily in gene therapy targeting neuromuscular and cardiac diseases, including Duchenne muscular dystrophy (DMD), Friedreich's ataxia, and catecholaminergic polymorphic ventricular tachycardia (CPVT) [2][4] Key Programs - **SGT-003**: Targeting Duchenne muscular dystrophy, currently in clinical trials with 15 patients dosed, showing positive early results [4][8] - **Friedreich's Ataxia Program**: Utilizing dual-route administration to address both CNS and cardiac manifestations [30][31] - **SGT-401**: Focused on CPVT, aiming to regulate calcium signaling to prevent arrhythmias [39][41] - **SGT-601**: Targeting TNNT2 dilated cardiomyopathy, currently in GLP toxicology [2][42] Clinical Data and Efficacy - **DMD Clinical Data**: - 15 patients dosed with SGT-003, showing no serious adverse events (SAEs) and positive biomarkers [12][11] - Achieved a target of over three vector genomes per nucleus, indicating strong expression levels [9][10] - Positive changes in cardiac biomarkers, including ejection fraction and troponin levels [11][12] - **Safety Profile**: - Clean safety profile with only one grade 1 liver enzyme spike reported, managed effectively [15][16] - Focus on maintaining low doses and high full-to-empty capsid ratios to minimize liver-related risks [14][15] Regulatory Strategy - **FDA Engagement**: Plans to meet with the FDA in Q4 to discuss pivotal trial design and potential for accelerated approval [20][21] - **Trial Design**: Proposed multi-country, multi-site, double-blind trial with endpoints including time to rise and cardiac output metrics [25][26] Financial Position - **Current Cash Position**: Approximately $268 million as of Q3, expected to fund operations into the first half of 2027 [43] - **Partnerships**: Open to partnerships but confident in the company's ability to advance independently [43] Industry Context and Innovation - **Gene Therapy Landscape**: Emphasis on advancing gene therapy through innovative capsids, promoters, and manufacturing processes to improve delivery and efficacy [45][46] - **Collaborations**: Currently engaged in 26 collaborations aimed at enhancing gene therapy tools, with a goal of reaching 100 [46][47] Additional Insights - **Market Positioning**: Solid Biosciences aims to elevate the entire gene therapy market by providing advanced tools and technologies to other companies, thereby fostering broader investment in the sector [46][47] This summary encapsulates the key points discussed during the conference call, highlighting Solid Biosciences' strategic focus, clinical advancements, regulatory plans, financial health, and commitment to innovation in gene therapy.