Solid Biosciences Inc. · NASDAQ:SLDB
Solid Biosciences — 41st Annual J.P. Morgan Healthcare Conference (January 2023)
27 slides from Solid Biosciences's conference presentation, Jan 9, 2023
Slide 1: Solid Biosciences 41st Annual J.P. Morgan Healthcare Conference
Solid Biosciences 41st Annual J.P. Morgan Healthcare Conference JANUARY 12, 2023 Exhibit 99.1
Slide 2: Forward Looking Statement
Forward Looking Statement 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 anticipated milestones, business focus and pipeli
Slide 3: 2023 Expected To Be a Year of Transformation and Meaningful Advancements for Solid
2023 Expected To Be a Year of Transformation and Meaningful Advancements for Solid PIPELINE Opportunity to become a leading precision genetic medicines company within neuromuscular and cardiac genetic medicine PROCESS Differentiated CMC expertise, building a robust, scalable manufacturing process ut
Slide 4: 2023: Solid Biosciences At a Glance
2023: Solid Biosciences At a Glance Developing Genetic Medicines for Patients with Rare Neuromuscular and Cardiac Diseases Completed merger between AavantiBio and Solid Biosciences in December 2022 Headquartered in Charlestown, MA Combined pipeline, novel capsid development, and expertise from both
Slide 5: Led By Experienced Team With Deep Expertise in Precision Genetic Medicine
Led By Experienced Team With Deep Expertise in Precision Genetic Medicine Bo Cumbo President and CEO Paul Herzich Chief Technology Officer Roxana Donisa Dreghici, M.D. Head of Clinical Development Ty Howton, J.D. Chief Administrative Officer Carl Morris, Ph.D. Chief Scientific Officer Neuromuscular
Slide 6: Solid is Well-Positioned to Execute on Multiple Programs in the Coming Years
Platform / Genetic Expertise Cardiac Rare Genetic Replacement Therapy (undisclosed #1) Cardiac Rare Genetic Replacement Therapy (undisclosed #2) Neuromuscular DMD uDys Replacement Therapy Anticipated 2H-2023 Neuromuscular Frataxin Replacement Therapy Cardiac BAG3 Replacement Therapy Cardiac Genetic
Slide 7: Next Generation Therapies Start With Delivery Through Innovative Capsids
AAV CARDIAC capsids enhance select cardiac tissue tropism and reduce liver targeting Rational Design approach used to engineer capsid candidates with the goal of improving SKELETAL MUSCLE tropism AAV-SLB101 AAV9 Wild Type AAV-SLB101 AAV9 DMDmdx Human Duchenne Cell Microdystrophin Expression NovCap 1
Slide 8: Combined Company Strengthens Process/Analytical Development & CMC Regulatory Team Capabilities
Combined Company Strengthens Process/Analytical Development & CMC Regulatory Team Capabilities Robust network of CDMO partners along with our internal MS&T expertise and dedicated resources support advancement of Solid’s pre-clinical and early-stage pipeline programs Process optimization and cGMP ma
Slide 9: Transition to Transient Transfection Manufacturing and Use of the AAV-SLB101 Capsid Yielded Additive Improvements
SGT-001-HSV SGT-003-TT Process change (HSV to TT) and AAV-SLB101 capsid combined to increase µDys by 2.3x vs SGT-001 HSV Product with desired quality attributes supported by analytical data with TT process Product demonstrated high levels of in vitro and in vivo transgene expression vs HSV materia
Slide 10: Solid is Well-Positioned for Strategic Pipeline Growth
Solid is Well-Positioned for Strategic Pipeline Growth Leveraging and Integrating Learnings Across Indications Pipeline Strategy Strategic Manufacturing Partnerships and Vector Core Indication Selection Criteria Network of KOL Partnerships Internal Development Capabilities
Slide 11: Indications With High Unmet Need and Significant Market Opportunities
Indications With High Unmet Need and Significant Market Opportunities Notes: In 2020, Solid entered into a collaboration agreement with Ultragenyx for the development of UX810, a next generation Duchenne construct comprised of Solid’s proprietary nNOS microdystrophin and Ultragenyx’s HeLa PCL manufa
Slide 12: Duchenne Muscular Dystrophy and Next Generation SGT-003
Duchenne Muscular Dystrophy and Next Generation SGT-003
Slide 13: Next Generation and Potential For Best-In-Class With SGT-003
Next Generation and Potential For Best-In-Class With SGT-003 Disease Overview Caused by mutations in the dystrophin gene, which leads to the absence of the dystrophin protein Due to progressive and irreversible muscle loss, patients typically lose the ability to walk by their early-teens and succumb
Slide 14: Next-Generation Therapies Utilize Optimized Transgene, Capsid and Manufacturing Process
Next-Generation Therapies Utilize Optimized Transgene, Capsid and Manufacturing Process Capsid Manufacturing Process Transgene nNOS Binding Domain AAV-SLB101 AAV9 Wild Type AAV-SLB101 AAV9 DMDmdx nNOS Microdystrophin uniquely includes the nNOS binding domain, important for prevention of activity-ind
Slide 15: Dose Dependent Expression Localized to Functional Membrane
Observations: Dose Dependent Expression Expression Localized to Functional Membrane All tissues reached 100% expression at all doses by day 29 Quantitative MS analysis will be performed to evaluate full dynamic range of expression Expression Achieved Early (Day 4) and Optimized by Day 29 Quadriceps
Slide 16: SGT-003 with SLB001 Capsid Demonstrated Superior Tropism to AAV9
SGT-003 with SLB001 Capsid Demonstrated Superior Tropism to AAV9 Positive biodistribution and expression data has the potential to translate into better efficacy Quad Heart Liver 5 4 3 2 1 0 Fold Change µDys vg/ug DNA vs. AAV9 (1E14vg/kg, D29) Fold Change µDys Expression vs. AAV9 (1E14vg/kg) AAV9 AA
Slide 17: NHP Data Utilizing AAV-SLB101 Showed Improved Biodistribution in Cardiac and Skeletal Muscle
Increased biodistribution to skeletal & cardiac muscle resulted in increased transgene expression at lower doses* Reduced biodistribution in liver suggests tissue de-targeting and improved safety profile* * Average fold differences calculated from the five skeletal muscle tissues sampled, three card
Slide 18: Friedreich's Ataxia and AVB-202
Friedreich’s Ataxia and AVB-202
Slide 19: AVB-202's dual route of administration is differentiated to treat the primary manifestations
AVB-202’s dual route of administration is differentiated to treat the primary manifestations of morbidity and mortality Disease Overview Monogenic disease caused by loss of frataxin with both neurological and cardiac manifestations affecting muscle control and coordination with possible loss of visi
Slide 20: AVB-202 Rescued Cardiac Function and Extended Survival in Cardiac FA Mouse
AVB-202 Rescued Cardiac Function and Extended Survival in Cardiac FA Mouse Survival Ejection Fraction SDH Activity Robust frataxin expression levels suggest efficacy may be achieved at low doses SDH activity via histochemical stain on heart tissue sections. The percentage of tissue with high stain
Slide 21: NHP Study: Favorable Safety Profile and Utility of Dual Route of Administration
NHP Study: Favorable Safety Profile and Utility of Dual Route of Administration Dose Group IV Dose (vg/kg) IT Dose (vg/brain wt) IV/IT Low 6.0E+12 6.00E+13 IV/IT Mid 1.5E+13 1.50E+14 IV/IT High 3.0E+13 3.00E+14 IV Mid 1.5E+13 - IV High 3.0E+13 - IT Mid - 1.50E+14 IT High - 3.00E+14 Using a dual rout
Slide 22: BAG3 Mediated Dilated Cardiomyopathy
BAG3 Mediated Dilated Cardiomyopathy
Slide 23: Attractive Indication with Clear Mechanistic Rationale, High Unmet Need, and Significant Market Opportunity
Attractive Indication with Clear Mechanistic Rationale, High Unmet Need, and Significant Market Opportunity Key Disease Highlights The BAG3 gene codes for the BCL-2-associated athanogene 3 protein Sufficient levels of functional BAG3 are required for healthy cardiac function BAG3 mutations lead to r
Slide 24: Data Illustrate Superior Cardiac Biodistribution and Transgene Expression Compared to AAV9
Data Illustrate Superior Cardiac Biodistribution and Transgene Expression Compared to AAV9 rh74+cardiac specific promotor showed better BD in the heart over AAV9 with a constitutive promotor. rh74-cardiac specific promotor combination showed increased cardiac expression, and decreased liver expressi
Slide 25: Driving the Future
Driving the Future SOLID
Slide 26: 2023 Anticipated Milestones
Complete SGT-003 GLP tox for next-generation Duchenne therapy 1H 2023 Initiation of Patient Dosing for SGT-003 Late-2023 ` Drug candidate selection and initiation of IND-enabling studies for AVB-202 Report functional data from IGNITE DMD Early-2023 IND Submission for SGT-003 2H 2023 Approximately $2
Slide 27: Thank You
Thank You 41st Annual J.P. Morgan Healthcare Conference JANUARY 12, 2023
Solid Biosciences — 41st Annual J.P. Morgan Healthcare Conference (January 2023) on 6ix