Sarepta Therapeutics, Inc. · NASDAQ:SRPT

Sarepta Therapeutics — Technical Presentation (May 2021)

18 slides from Sarepta Therapeutics's technical presentation, May 18, 2021

  1. Slide 1: Clinical Update: Micro-dystrophin Gene Therapy Study SRP-9001-103

    Doug Ingram President and CEO May 18, 2021 8:30 a.m. ET Louise Rodino-Klapac, Ph.D. Executive Vice President and Chief Scientific Officer Clinical Update: Micro-dystrophin Gene Therapy Study SRP-9001-103: 12-Week Expression and Safety Data Using Commercially Representative Material EXHIBIT 99.2 EXHI

  2. Slide 2: Welcome and Introduction

    Welcome and Introduction Doug Ingram President and CEO Welcome and Introduction Doug Ingram President and CEO SAREPTA THERAPEUTICS, INC. 2021. ALL RIGHTS RESERVED. 2

  3. Slide 3: Forward-Looking Statements

    ©2020 SAREPTA THERAPEUTICS. NOT FOR DISTRIBUTION. COPYING OF THIS MATERIAL BY ANY MEANS WITHOUT SAREPTA’S PRIOR WRITTEN CONSENT IS PROHIBITED. SAREPTA THERAPEUTICS and the SAREPTA Helix Logo are trademarks of Sarepta Therapeutics, Inc. registered in the U.S. Patent and Trademark Office and may be re

  4. Slide 4: Duchenne Muscular Dystrophy (DMD)

    Duchenne Muscular Dystrophy (DMD) DMD affects approximately 1 in 3,500-5,000 males worldwide1 DMD is a rare, fatal neuromuscular genetic disease inherited in an X-linked recessive pattern2 Muscle weakness becomes increasingly noticeable by 3 to 5 years of age, and most patients use a wheelchair by t

  5. Slide 5: SRP-9001 Gene Therapy Construct

    SRP-9001 Gene Therapy Construct VECTOR PROMOTER TRANSGENE Provides systemic delivery to muscle cells, including the heart and skeletal muscle Low level of pre- existing immunity AAVrh74 Designed to generate a functional micro-dystrophin Includes SR2 and SR3 - essential for muscle force3 MICRO-DYSTRO

  6. Slide 6: Micro-dystrophin is a Shortened, Functional Form of Dystrophin

    Micro-dystrophin is a Shortened, Functional Form of Dystrophin Normal Muscle3-8 61 YO Ambulatory Patient9-11 Sarepta Micro-dystrophin4-11 Micro-dystrophin gene therapy is investigational and has not been reviewed or approved by any regulatory authority. 1. Naso MF, et al. BioDrugs. 2017;31(4):317-33

  7. Slide 7: Clinical Results: Micro-dystrophin Study SRP-9001-103

    Clinical Results: Micro-dystrophin Study SRP-9001-103 Expression and Safety Data at 12 Weeks in Patients with Duchenne Louise Rodino-Klapac, Ph.D. Executive Vice President and Chief Scientific Officer Clinical Results: Micro-dystrophin Study SRP-9001-103 Expression and Safety Data at 12 Weeks in Pat

  8. Slide 8: SRP-9001-103 Study

    SRP-9001-103* Study 103 Ongoing phase 1b open-label study using commercially representative material of SRP-9001 Four U.S. sites Boys with Duchenne, ages 4 to <8 20 patients, today’s data from first 11 patients Dose Weight based dosing: 1.33 x 1014 vg/kg *ClinicalTrials.gov Identifier: NCT04626674 B

  9. Slide 9: Questions to Consider When Evaluating Gene Transfer Therapies

    Questions to Consider When Evaluating Gene Transfer Therapies Experiment Vector + transgene Transgene in nucleus Vector Genome Copies/Nucleus Western Blot Immunofluorescence % Positive Fibers (PDPF): % cells with protein Intensity of Fluorescent Signal (IF): How strong is expression in cells with pr

  10. Slide 10: Micro-dystrophin Transduction by Vector Genome Count in First 11 Patients

    Micro-dystrophin Transduction by Vector Genome Count in First 11 Patients Question Is the desired protein made? 2 Is the transgene DNA inside muscle cells? 1 Is the protein at the cell membrane? How much is there? 3 What was the safety and tolerability experience? 4 Based on muscle biopsies from the

  11. Slide 11: Micro-dystrophin Expression by Western Blot at Week 12

    Micro-dystrophin Expression by Western Blot at Week 12 Post-treatment in the First 11 Patients Question Is the desired protein made? 2 Is the transgene DNA inside muscle cells? 1 Is the protein at the cell membrane? How much is there? 3 What was the safety and tolerability experience? 4 Based on mus

  12. Slide 12: Percentage of Dystrophin Positive Fibers and Intensity at Week 12

    Micro-dystrophin Percentage of Dystrophin Positive Fibers and Intensity at Week 12 Post-treatment in the First 11 Patients Question Is the desired protein made? 2 Is the transgene DNA inside muscle cells? 1 Is the protein at the cell membrane? How much is there? 3 What was the safety and tolerabilit

  13. Slide 13: Study 102 Crossover and Study 103 Patients Show Consistent Results

    Study 102 Part 2 Placebo Crossover Patients (Clinical Process Material) and Study 103 Patients (Commercially Representative Process Material) Show Consistent Results Micro-dystrophin Clinical Process Material Study 102 Part 2 Placebo Crossover Patients, Mean (n=11) Micro-dystrophin Commercially Repr

  14. Slide 14: SRP-9001-103 Safety Experience

    SRP-9001-103 Safety Experience Safety was consistent with previous experience with SRP-9001 79 treatment-emergent adverse events in 11 patients Most common adverse event was vomiting Typical onset within first week, mild, and treated with standard antiemetics Increase in liver enzymes were transient

  15. Slide 15: Key Takeaways: SRP-9001 Provides a Differentiated Profile for Duchenne

    Key Takeaways SRP-9001 Provides a Differentiated Profile for Duchenne Confirmed characteristics of commercially representative SRP-9001 Robust transduction – 3.87 mean vector genome copies per nucleus Mean robust expression with proper localization to the sarcolemma membrane Western blot – 55.4% Pos

  16. Slide 16: Next Steps

    3 Next Steps Meet with the FDA, mid-year 2021; and other regulatory agencies Commence Study 301 following FDA meeting Expand Study 103 to include older ambulant and non-ambulant patients 1 2 2 Next Steps 1 2 3 4 Meet with the FDA, mid-year 2021; and other regulatory agencies Commence Study 301 follo

  17. Slide 17: Question and Answer

    Question and Answer Question and Answer SAREPTA THERAPEUTICS, INC. 2021. ALL RIGHTS RESERVED. 17

  18. Slide 18: Dragging tomorrow into today

    #DraggingTomorrowIntoToday Dragging tomorrow into today SAREPTA, SAREPTA THERAPEUTICS, the SAREPTA Helix Logo, VYONDYS, VYONDYS 53, the VYONDYS 53 Logo, EXONDYS, EXONDYS 51, and the EXONDYS 51 Logo are trademarks of Sarepta Therapeutics, Inc. registered in the U.S. Patent and Trademark Office and ma

Sarepta Therapeutics — Technical Presentation (May 2021) on 6ix