Sarepta Therapeutics, Inc. · NASDAQ:SRPT

Sarepta Therapeutics — Technical Presentation (January 2021)

16 slides from Sarepta Therapeutics's technical presentation, Jan 7, 2021

  1. Slide 1: Micro-dystrophin SRP-9001-102 Top-line Clinical Data (Part One)

    Micro-dystrophin SRP-9001-102 Top-line Clinical Data (Part One) Doug Ingram President and Chief Executive Officer Louise Rodino-Klapac, Ph.D. Executive Vice President, Chief Scientific Officer January 7, 2021 Exhibit 99.2 SAREPTA THERAPEUTICS SAREPTA THERAPEUTICS, INC.2021 ALL RIGHTS RESERVED. 1

  2. Slide 2: Forward-looking Statements

    Forward-looking Statements This presentation contains "forward-looking statements." Any statements that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believe,” “anticipate,” “plan,” “expect,” “will,” “may,” “intend,” “prepare,” “look,” “potentia

  3. Slide 3: Welcome and Introduction

    Welcome and Introduction Doug Ingram President and CEO SAREPTA THERAPEUTICS SAREPTA THERAPEUTICS, INC.2021 ALL RIGHTS RESERVED. 3

  4. Slide 4: SRP-9001-102 Study Design: Parts 1 and 2

    SRP-9001-102 Study Design: Parts 1 and 2 A randomized, double-blind, placebo-controlled clinical trial to evaluate the safety, efficacy and tolerability of a single dose of SRP-9001 compared to placebo, in boys with DMD aged 4–7 years old; Study is ongoing and remains blinded, functional results for

  5. Slide 5: Micro-dystrophin Protein Expression and Vector Genome Copies per Nucleus

    Micro-dystrophin Protein Expression and Vector Genome Copies per Nucleus Achieved Endpoints (n=20, Week 12) Micro-dystrophin Expression (IF) Vector Genome Copy Number Micro-dystrophin Expression (Western Blot) Percentage of Normal Mean (n=20) 28.1% Intensity (% Normal) Percentage of Dystrophin-posit

  6. Slide 6: Representative Micro-dystrophin Images

    Representative Micro-dystrophin Images Baseline SRP-9001 Merosin (membrane label) Micro-dystrophin Merge Patient had 95% PDPF on treatment NOT APPLICABLE SAREPTA THERAPEUTICS, INC.2021 ALL RIGHTS RESERVED. 6

  7. Slide 7: Functional Results

    Functional Results SAREPTA THERAPEUTICS, INC.2021 ALL RIGHTS RESERVED. 7

  8. Slide 8: NSAA Primary Functional Endpoint: Treated Patients Outperformed Placebo Patients at All Time Points

    NSAA Primary Functional Endpoint: Treated Patients Outperformed Placebo Patients at All Time Points NSAA change from baseline of +1.7 in SRP-9001 treated group vs. +0.9 in placebo group, which is not statistically different (p= 0.37) Separation shown at every timepoint between SRP-9001 and placebo g

  9. Slide 9: NSAA Subgroup Analysis (Ages 4-5): Reached Statistical Significance

    NSAA Subgroup Analysis (Ages 4-5): Reached Statistical Significance In a pre-specified analysis, the 4- to 5-year old group had a statistically significant improvement in NSAA vs. placebo group at week 48 NSAA change from baseline of +4.3 in SRP-9001 treated 4–5-year-olds vs. 1.9 in placebo (p= 0.01

  10. Slide 10: Functional Measures Well Matched at Baseline (4-5 Year Old Group)

    Functional Measures Well Matched at Baseline (4-5 Year Old Group) Characteristic Statistics SRP-9001 Age 4-5 (n=8) Placebo Age 4-5 (n=8) NSAA Mean P-value(vs Placebo) 20.1 0.8318 20.4 100 meter (seconds) Mean P-value(vs Placebo) 58.76 0.7925 59.79 Ascend 4 Steps (seconds) Mean P-value(vs Placebo) 3.

  11. Slide 11: Functional Measures Not Well Matched at Baseline (6-7 Year Old Group)

    Functional Measures Not Well Matched at Baseline (6-7 Year Old Group) Patients in the treated group had significantly lower NSAA scores at baseline Note that an imbalance in NSAA and timed tests exist in the older (6–7-year-olds) between the two groups with the treated group worse than placebo. Char

  12. Slide 12: Safety Summary

    Safety Summary No new safety signals Safe and well tolerated; consistent with previous studies 85% of the treated group had treatment related adverse events vs. 43% in the placebo group The most common treatment related adverse event was vomiting 60% (12/20) in treatment group vs. 19% (4/21) in plac

  13. Slide 13: Next Steps

    Next Steps Continue to advance Part 2 crossover phase; conduct biopsy at week 12 to assess expression and biological markers and longer-term assessments of functional outcomes Enrolled and dosed 11 patients in Study 103 using commercial process material Report biomarker and safety results in Q2 2021

  14. Slide 14: Conclusions

    Conclusions No new safety signals observed Primary biological endpoint (micro-dystrophin expression at 12 weeks post-treatment) achieved Total NSAA score of treated patients vs. placebo demonstrated a positive increase at all post-treatment time points The study did not achieve a statistical signifi

  15. Slide 15: Q&A

    Q&A SAREPTA THERAPEUTICS, INC.2021 ALL RIGHTS RESERVED. 15

  16. Slide 16: Sarepta Therapeutics

    SAREPTA THERAPEUTICS SAREPTA THERAPEUTICS, INC.2021 ALL RIGHTS RESERVED. 16

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