Solid Biosciences Inc. · NASDAQ:SLDB

Solid Biosciences — Parent Project Onlus Annual Conference 2019

17 slides from Solid Biosciences's conference presentation, Feb 16, 2019

  1. Slide 1: Microdystrophin Gene Therapy – SGT-001

    Microdystrophin Gene Therapy – SGT-001 Carl Morris, Ph.D., Chief Scientific Officer, Solid Biosciences February 16, 2019

  2. 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

  3. Slide 3: Purpose-Built To Solve Duchenne Muscular Dystrophy

    Purpose-Built To Solve Duchenne Muscular Dystrophy 360-degree Differentiated Lead Scalable Approach Gene Transfer Manufacturing Address all facets Process of DMD Meet clinical and commercial needs © 2019 Solid Biosciences 3

  4. Slide 4: Solid Is Addressing The Full Spectrum Of Duchenne

    Solid Is Addressing The Full Spectrum Of Duchenne CORRECTIVE THERAPIES DISEASE-MODIFYING THERAPIES Gene therapy to address the Small molecules and biologics to genetic cause of DMD address symptoms DISEASE UNDERSTANDING ASSISTIVE DEVICES Biomarkers and endpoints to Technology to support mobility imp

  5. Slide 5: Gene Transfer – SGT-001

    Corrective Therapies Gene Transfer – SGT-001 © 2019 Solid Biosciences 5

  6. Slide 6: What Is Gene Transfer For DMD?

    What Is Gene Transfer For DMD? Gene transfer for DMD is made The combined product is then up of three essential elements: administered to the patient 1 Gene 2 Vector Carries the gene 2 Promoter Controls expression © 2019 Solid Biosciences 6

  7. Slide 7: Each Component Of SGT-001 Was Carefully Selected

    Each Component Of SGT-001 Was Carefully Selected Transgene Restore key functions SGT-001 of a complex protein microdystrophin Promoter Expression is highly targeted CK8 Vector Skeletal and cardiac transduction AAV9 © 2019 Solid Biosciences 7

  8. Slide 8: SGT-001 Microdystrophin Has A Differentiated Composition

    SGT-001 Microdystrophin Has A Differentiated Composition Full Length Dystrophin Protein SGT-001 Microdystrophin Protein • Microdystrophin construct DNA able to fit in AAV9 vector • Contains 5 structural domains and uniquely includes the nNOS binding domain • SGT-001 selection based on more than 30 y

  9. Slide 9: SGT-001 Promotes Significant Cardiac And Skeletal Muscle Microdystrophin Expression in mdx mice

    SGT-001 Promotes Significant Cardiac And Skeletal Muscle Microdystrophin Expression in dystrophic (mdx) mice SGT-001 Normal Dystrophic 5E13 vg/kg 2E14 vg/kg Heart Skeletal Muscle (Quadriceps) Observed at Clinically Relevant Dose Levels © 2019 Solid Biosciences 9

  10. Slide 10: CK8 Muscle-Specific Promoter Restricts Expression To Muscles In Preclinical Studies

    CK8 Muscle-Specific Promoter Restricts Expression To Muscles In Preclinical Studies Target Tissue Non-target Tissue SGT-001 Treated 100% 50% 25% 10% 0% Diaphragm Microdystrophin Dystrophin Dose (vg/kg) SGT-001 Treated 0 1E14 2E14 1E13 100% 50% 25% 10% 0% Liver Microdystrophin Dystrophin Dose (vg/kg)

  11. Slide 11: Dose-escalation - Linking Dose Level To Function In Preclinical Studies

    Dose-escalation - Linking Dose Level To Function In Preclinical Studies Exposure Target Engagement Efficacy / Benefit (How much vector (drug) gets (How much protein is produced?) (What functional changes are observed?) into the tissue?) % Normal (GRMD) 100 50 25 10 0 Dose Level Force Production 1 5

  12. Slide 12: SGT-001 Clinical Program: IGNITE DMD

    SGT-001 Clinical Program IGNITE DMD © 2019 Solid Biosciences 12

  13. Slide 13: SGT-001 Phase I/II Clinical Study Ongoing

    SGT-001 Phase I/II Clinical Study Ongoing 12 months primary endpoint DSMB Continue Cohort 1 Cohort 2* DSMB Observation Starting dose: 5E13 vg/kg Planned dose escalation cohort: Screening, (*Dose – to be determined) Baseline Ambulatory & children, Randomization aged 4-11 yrs Switch to Matched control

  14. Slide 14: IGNITE-DMD – Clinical Trial Update

    IGNITE-DMD – Clinical Trial Update • Dosed the first patient in February 2018 • To date, six (6) patients have been enrolled in IGNITE DMD at University of Florida • Three (3) to the active treatment group (5E13 vg/kg) and, • Three (3) to the delayed treatment control group • Safety profile of all d

  15. Slide 15: 3-month Biopsy Data Summary

    3-month Biopsy Data Summary • All three biopsies showed low levels of microdystrophin protein expression via immunofluorescence • In one patient, microdystrophin was detected via western blot (<5%) and in ~10 percent of fibers via immunofluorescence. • Evidence of co-localization of neuronal nitric

  16. Slide 16: GMP Manufacturing Currently Capable of Supplying Clinical Study

    GMP Manufacturing Currently Capable of Supplying Clinical Study • Successfully scaled up to 250L in suspension and produced multiple batches • SGT-001 material available for planned dose-escalation cohort without delay 2,000 Liter CellSTACK HYPERStack 500+ 250 Liter 50 Liter 25 Liter 2L Liter Commer

  17. Slide 17: Thank You

    SOLID BIOSCIENCES Thank You © 2019 Solid Biosciences 17

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