Solid Biosciences Inc. · NASDAQ:SLDB
Solid Biosciences — Investor Presentation (January 2019)
29 slides from Solid Biosciences's investor presentation, Jan 8, 2019
Slide 1: Solid Biosciences
Solid Biosciences Ilan Ganot, Co-Founder, CEO and President January 2019 Exhibit 99.1
Slide 2: Forward-Looking Statements
Forward-Looking Statements This presentation includes “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, which involve a number of risks and uncertainties. These forward-looking statements include all matters that are not historical facts and, wi
Slide 3: Purpose-Built to Solve Duchenne Muscular Dystrophy
Purpose-Built to Solve Duchenne Muscular Dystrophy © 2019 Solid Biosciences 360-degree Approach Address all facets of DMD Differentiated Lead Gene Transfer Preliminary clinical data this quarter Scalable Manufacturing Process Meet clinical and commercial needs
Slide 4: Duchenne Is A Devastating Muscle-Wasting Disease
Duchenne Is A Devastating Muscle-Wasting Disease 10-15,000 cases in the U.S. *Source: Erik Landfeldt, Peter Lindgren, Christopher F. Bell, et al. The burden of Duchenne muscular dystrophy: An international, cross-sectional study. Neurology 2014. Progressive & irreversible Caused by mutations in the
Slide 5: Solid Is Addressing The Full Spectrum Of Duchenne Corrective Therapies
Solid Is Addressing The Full Spectrum Of Duchenne Corrective Therapies Gene therapy to address the genetic cause of DMD Disease understanding Assistive devices Biomarkers and endpoints to improve development Technology to support mobility Disease-modifying therapies Small molecules and biologics to
Slide 6: Solid Pipeline
Solid Pipeline PRECLINICAL PHASE I/II SGT-001 Gene Transfer Anti-LTBP4 Biologic MARKETED Corrective Therapies Disease Modifying Therapies DISCOVERY NAME PHASE III Solid Suit PROTOTYPE DEVELOPMENT TESTING Assistive Devices MARKETED NAME Next-Generation Promoter Next-Generation Vector Next-Generation
Slide 7: Innovation in Gene Transfer Corrective Therapies
Innovation in Gene Transfer Corrective Therapies
Slide 8: Gene Therapy To Address The Genetic Cause Of DMD: Healthy muscle
Gene Therapy To Address The Genetic Cause Of DMD Visual representation only Healthy muscle Muscle Membrane Myofibril Actin Dystrophin Dystrophin protects the muscle from damage and stabilizes critical dystrophin-associated proteins
Slide 9: Gene Therapy To Address The Genetic Cause Of DMD: Dystrophic muscle
Gene Therapy To Address The Genetic Cause Of DMD Visual representation only Dystrophic muscle In DMD, mutations in the dystrophin gene result in the loss of functional dystrophin protein Muscle fibers become unstable, lose the ability to repair and become fibrotic Myofibril Muscle Membrane Actin
Slide 10: Gene Therapy To Address The Genetic Cause Of DMD: Treated muscle
Gene Therapy To Address The Genetic Cause Of DMD Visual representation only Treated muscle Myofibril Microdystrophin Microdystrophin gene transfer encodes for a functional dystrophin protein surrogate designed to replace the missing dystrophin protein Actin Muscle Membrane
Slide 11: Each Component Of SGT-001 Was Carefully Selected
Each Component Of SGT-001 Was Carefully Selected AAV9 Vector Skeletal and cardiac transduction CK8 Promoter Expression is highly targeted SGT-001 microdystrophin Transgene Restore key functions of a complex protein
Slide 12: SGT-001 Microdystrophin Has A Differentiated Composition
SGT-001 Microdystrophin Has A Differentiated Composition Visual representation only Full Length Dystrophin Protein SGT-001 Microdystrophin Protein Exclusive licenses to key patent portfolios covering microdystrophin variants and functional domains (e.g. the nNOS binding domain) SGT-001 selection bas
Slide 13: SGT-001 Promotes Significant Cardiac And Skeletal Muscle Microdystrophin Expression In Mice
SGT-001 Promotes Significant Cardiac And Skeletal Muscle Microdystrophin Expression In Mice Normal Dystrophic 5E13 vg/kg 2E14 vg/kg Heart Skeletal Muscle (Quadriceps) SGT-001 Solid Biosciences data on file.
Slide 14: CK8 Muscle-Specific Promoter Restricts Expression To Muscles
CK8 Muscle-Specific Promoter Restricts Expression To Muscles Solid Biosciences data on file. Three month efficacy study in GRMD canines. Representative only. Non-target Tissue Target Tissue 100% 50% 25% 10% 0% 100% 50% 25% 10% 0% 100% 50% 25% 10% 0% Dystrophin Dystrophin Dystrophin Microdystrophin M
Slide 15: Microdystrophin With nNOS Binding Domain Selected Based On Extensive Comparative Analysis
Microdystrophin With nNOS Binding Domain Selected Based On Extensive Comparative Analysis nNOS Negative nNOS Positive (SGT-001) Vehicle Wild Type J. Chamberlain et al. submitted for publication.
Slide 16: Significant Functional Benefit Demonstrated In Dystrophic Canines
Significant Functional Benefit Demonstrated In Dystrophic Canines Solid Biosciences data on file. Three month efficacy study in GRMD canines. N=3 per group | * p<0.05 statistical significance.
Slide 17: Long-term Durability Observed In Canines
Long-term Durability Observed In Canines 30 Months 1 Month 3 Months 24 Months Hakim et al. American Society of Gene and Cell Therapy (ASGCT) 2018.
Slide 18: IGNITE DMD SGT-001 Clinical Program
IGNITE DMD SGT-001 Clinical Program
Slide 19: SGT-001 Phase I/II Clinical Study Ongoing
SGT-001 Phase I/II Clinical Study Ongoing Ambulatory children and non-ambulatory adolescents aged 4-17 (n=16-32) SGT-001 treatment group Starting at 5E13 vg/kg; plan to escalate dose Observation Matched control group SGT-001 Primary Endpoints: Safety SGT-001 microdystrophin expression Secondary Endp
Slide 20: Early Planning Enabled A Rational Clinical Approach
Early Planning Enabled A Rational Clinical Approach Designed to obtain efficacy data at multiple dose levels Microdystrophin expression, muscle function and dose response Includes randomized control arm for comparative data Multiple patients and their matched controls enrolled Supports evaluation ac
Slide 21: Producing Materials Manufacturing
Producing Materials Manufacturing
Slide 22: Addressing The DMD Gene Therapy Supply Challenge
Addressing The DMD Gene Therapy Supply Challenge HIGH PREVALENCE ( ) ( ) ( ) HIGH AVERAGE PATIENT WEIGHT HIGH DOSES SIGNIFICANT SUPPLY NEEDS Move quickly with a process that scales up to meet the needs of all patients with DMD Solid Manufacturing Strategy © 2019 Solid Biosciences
Slide 23: GMP Manufacturing Process Currently Producing At Significant Volume
Successfully scaled up to 250L in suspension and produced multiple batches Each 250L batch can dose multiple patients Utilizes proven, validated and widely-available standard bioreactors GMP Manufacturing Process Currently Producing At Significant Volume 250 Liter Successful scale up to 250L suspens
Slide 24: Scaling Process To Efficiently Supply Commercial Markets
Continue to optimize process development in Solid labs Maintain low number of bioreactors to support operational efficiencies Create ability to potentially treat 1,000s of patients Scaling Process To Efficiently Supply Commercial Markets 250 Liter Successful scale up to 250L complete Commercial scal
Slide 25: Expanding Pipeline LTBP4 and Next Generation Gene Therapies
Expanding Pipeline LTBP4 and Next Generation Gene Therapies
Slide 26: Anti-LTBP4 Disease-Modifying Therapy To Address Fibrosis
Anti-LTBP4 Disease-Modifying Therapy To Address Fibrosis Age at loss of ambulation (yrs.) 5 10 15 100 80 60 40 20 0 Percent ambulatory Improved Muscle Function Decreased Muscle Fibrosis Positive results from blinded, 24-week efficacy study LTBP4 is a powerful genetic modifier in DMD* mdx/hLTBP4 mice
Slide 27: Internal And Partnered Programs To Build Comprehensive Pipeline For Duchenne
Internal And Partnered Programs To Build Comprehensive Pipeline For Duchenne Augment Muscle Strength Add Functionality to the Microdystrophin Transgene Expand Population and Enable Re-administration Enhance Tissue Expression
Slide 28: Next Generation Screening in Disease-Specific Models Yields Novel Potential AAV Candidates
Next Generation Screening in Disease-Specific Models Yields Novel Potential AAV Candidates Library 1 Library 2 Library 3 Parental capsids AAV9 AAVrh32.33 AAV2 AAV5 AAV8 AAVrh10 AAV6 AAV1 Capsids enriched in quadriceps Capsids decreased in quadriceps Data on file.
Slide 29: Significant Catalysts In 2019
Program Advancement Preliminary data in the first quarter of 2019 Interim analysis in the second half of 2019 Pipeline Progress LTBP4 program toward IND Advance next generation promoters/vectors Support mission with targeted business development Significant Catalysts In 2019 Manufacturing process de
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