Sarepta Therapeutics, Inc. · NASDAQ:SRPT
Sarepta Therapeutics — Technical Presentation (March 2019)
20 slides from Sarepta Therapeutics's technical presentation, Mar 25, 2019
Slide 1: Clinical Update: Micro-dystrophin Study-101
Clinical Update: Micro-dystrophin Study-101 Louise Rodino-Klapac, Ph.D. Senior Vice President, Gene Therapy Sarepta Therapeutics, Inc. Exhibit 99.1
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
Slide 3: Micro-dystrophin Study-101 Clinical Trial Design
Micro-dystrophin Study-101 Clinical Trial Design
Slide 4: Open-Label Trial Design Cohort B
Open-Label Trial Design Cohort B 4 Patients 4-7 years of age Inclusion criteria Confirmed DMD mutation Negative for AAVrh74 antibodies ClinicalTrials.gov Identifier: NCT03375164. Sarepta Therapeutics Data on File. AAVrh74.MHCK7.Micro-dystrophin is investigational and not approved in Argentina.
Slide 5: Cohort B (4-7 Years of Age) Endpoints
Cohort B (4-7 Years of Age) Endpoints Primary endpoint Safety Secondary endpoints Change in micro-dystrophin expression pre- vs post-treatment Decrease in CK 100-meter timed test (100 m) North Star Ambulatory Assessment (NSAA; 10-meter timed test included) Timed up and go (TUG) Ascend and descend 4
Slide 6: Subject Demographics at Baseline
Subject Demographics at Baseline Subject Age (years) CK Levels at Baseline (U/L) 1 5 20,691 2 4 23,414 3 6 34,942 4 4 29,210 ClinicalTrials.gov Identifier: NCT03375164. Sarepta Therapeutics Data on File. AAVrh74.MHCK7.Micro-dystrophin is investigational and not approved in Argentina.
Slide 7: Micro-dystrophin Study-101 Summary: All Patients (n=4)
Micro-dystrophin Study-101 Summary: All Patients (n=4)
Slide 8: Robust Micro-dystrophin Expression in Muscle Fibers at Day 90
Robust Micro-dystrophin Expression in Muscle Fibers from the Gastrocnemius in All 4 Patients at Day 90 Intensity Percentage of Dystrophin-positive Fibers Mean (n=4) 96.0% 81.2% Normal Control Subject 2 Subject 1 Subject 3 Micro-dystrophin Expression (IHC) Subject 4 Post-treatment Pre-treatment Clini
Slide 9: Detection of Micro-dystrophin Expression by Western Blot
Detection of Micro-dystrophin Expression by Western Blot Post-treatment in All 4 Patients at Day 90 Western Quantitation Method Mean Micro-dystrophin Expression (N=4) vs Normal Sarepta 74.3% (not adjusted for fat and fibrotic tissue) Nationwide 95.8% (adjusted for fat and fibrotic tissue) * p-Tyr ph
Slide 10: Summary of Expression Data for all Patients
Summary of Expression Data for all 4 Patients Intensity Percentage of Dystrophin-positive Fibers Mean (n=4) 96.0% 81.2% Sarepta (not adjusted for fat/fibrosis) Nationwide (adjusted for fat/fibrosis) Mean (n=4) 74.3% 95.8% Vector Copies/μg DNA Copies per Nucleus Mean (n=4) >105 3.3 Micro-dystrophin E
Slide 11: Clinical Data Summary for Study-101 (n=4)
Clinical Data Summary for Study-101 (n=4)
Slide 12: Summary of NSAA Data for All 4 Patients
ClinicalTrials.gov Identifier: NCT03375164. Sarepta Therapeutics Data on File. AAVrh74.MHCK7.Micro-dystrophin is investigational and not approved in Argentina. Summary of NSAA Data for All 4 Patients NSAA Change from Baseline to Day 270 Patient Baseline Day 30 Day 60 Day 90 Day 180 Day 270 Change fr
Slide 13: Summary of Clinical Data: Consistent Durable Improvement at 9 Months
* Last timepoint disclosed at World Muscle Society ClinicalTrials.gov Identifier: NCT03375164. Sarepta Therapeutics Data on File. AAVrh74.MHCK7.Micro-dystrophin is investigational and not approved in Argentina. Summary of Clinical Data: Consistent Durable Improvement at 9 Months Change from Baseline
Slide 14: Summary of CK Data for All 4 Patients
ClinicalTrials.gov Identifier: NCT03375164. Sarepta Therapeutics Data on File. AAVrh74.MHCK7.Micro-dystrophin is investigational and not approved in Argentina. Summary of CK Data for All 4 Patients CK Change from Baseline to Day 270 Patient Baseline Day 30 Day 60 Day 90 Day 180 Day 270 1 20691 - 298
Slide 15: CK Significantly Decreases Over Time
ClinicalTrials.gov Identifier: NCT03375164. Sarepta Therapeutics Data on File. AAVrh74.MHCK7.Micro-dystrophin is investigational and not approved in Argentina. CK Significantly Decreases Over Time Mean CK Change from Baseline to Day 270 Baseline 30 DAYS 60 DAYS 90 DAYS 180 DAYS 270 DAYS CK Levels (U
Slide 16: NSAA Significantly Increases Over Time
ClinicalTrials.gov Identifier: NCT03375164. Sarepta Therapeutics Data on File. AAVrh74.MHCK7.Micro-dystrophin is investigational and not approved in Argentina. NSAA Significantly Increases Over Time Mean NSAA Change from Baseline to Day 270 Baseline 30 DAYS 60 DAYS 90 DAYS 180 DAYS 270 DAYS CK Level
Slide 17: Safety (n=4)
Safety (n=4) No serious adverse events in this study 3 Patients had elevated γ‐glutamyl transpeptidase, which resolved with steroid treatment within a week No other clinically significant laboratory findings Subjects had transient nausea generally within the first week coincident with increased ster
Slide 18: Summary
Summary All 4 treated Patients are doing well Biomarkers show large magnitude of effect within 3 months (CK and dystrophin) Initial functional data shows consistent and persistent improvement from Baseline to Day 270 Early results show treated patients performing better than DMD natural history woul
Slide 19: Question and Answer
Question and Answer
Slide 20: Thank You
Thank You o 3 < = < KH Qa lw [aa < YN §
Sarepta Therapeutics — Technical Presentation (March 2019) on 6ix