Sarepta Therapeutics, Inc. · NASDAQ:SRPT
Sarepta Therapeutics — Technical Presentation (January 2026)
23 slides from Sarepta Therapeutics's technical presentation, Jan 26, 2026
Slide 1: SRP-9001-301 Part 1 3-Year Data Topline Results
SRP-9001-301 PART-1 3-Year Data Topline Results Doug Ingram Chief Executive Officer Louise R. Rodino-Klapac, Ph.D. President, R&D and Technical Operations James Richardson, MA (Oxon), BMBCh, MBA, MRCP (Lon) Executive Vice President, Chief Medical Officer DILLON Living with Duchenne January 26, 2026
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: EMBARK (Study 9001-301)
EMBARK (Study 9001-301) Louise Rodino-Klapac, PhD President, R&D and Technical Operations C O N F I D E N T I A L . F O R I N T E R N A L U S E O N L Y. ©SAREPTA THERAPEUTICS, INC. 2026. ALL RIGHTS RESERVED. 3
Slide 4: 3-year Topline Functional Results from Part 1 of EMBARK
3-year Topline Functional Results from Part 1 of EMBARK (Study 9001-301) • On average, ELEVIDYS-treated patients remain above their baseline three years after treatment as measured by NSAA • Statistically significant 73% slowing of disease progression as measured by TTR (time to rise) • Statisticall
Slide 5: Physical Function Typically Peaks Around Age 6-7 in Duchenne
Physical Function Typically Peaks Around Age 6 in Individuals With Duchenne1-3 At the time of their last assessment, mean age of Part 1- treated patients is 9 years old Maximum score Slowed development Limited peak performance Physical function as measured by NSAA* Typically age 6–72 Eventual loss o
Slide 6: Muscle Biopsies of Patients with DMD Show Continued Damage at Early Ages
Muscle Biopsies of Patients with DMD Show Continued Damage at Early Ages Biopsies Showed Fiber Size Variation, and Progressive Loss of Muscle Structure and Integritya,b DMD Quadricep 1 Year DMD Quadricep 3 Years Control Quadricep 4 Years Blue arrows represent necrotic fibers DMD Quadricep 5 Years DM
Slide 7: Potential Outcomes Based on Timing of Therapeutic Intervention
Potential Outcomes Based on Timing of Therapeutic Intervention When Attempting to Stabilize Duchenne Progression Healthy children usually reach peak NSAA score of" 34 after age 41 Interventions Increased score/higher peak2,3 aim to slow or stabilize Stabilization/ underlying slowed decline2,3 pathol
Slide 8: Largest and Most Comprehensive Clinical Program in Duchenne
Largest and Most Comprehensive Clinical Program in Duchenne Clinical trial participants have ranged in age from 2 to 24 years old, with body weights ranging from 12 kg to close to 90 kg STUDY 101 STUDY 102 Ages 4-7, ambulatory Ages 4-7, ambulatory Ages 3+, ambulatory and Ages 4-7, ambulatory Open-La
Slide 9: SRP-9001-301 Study Design
SRP-9001-301 Study Design A completed Phase 3, multinational, double-blind, randomized, placebo-controlled study evaluating the safety and efficacy of ELEVIDYS (delandistrogene moxeparvovec-rokl), in boys with DMD aged 4–7 years old Key functional inclusion criteria: • NSAA score 17-28 (inclusive) a
Slide 10: EMBARK Part 2: Statistically Significant, Clinically Meaningful Differences 2 Years After Treatment
EMBARK Part 2: Statistically Significant, Clinically Meaningful Differences 2 years After Treatment Compared to External Control NSAA TTR* TTR velocity 10MWR time* 10MWR velocity Diff in Means (of change from baseline) and confidence intervals are standardized by dividing by the standard error (SE).
Slide 11: Year 2 Results: Musculoskeletal MRI - Fat Fraction
2 Year Results: Musculoskeletal MRI - Fat Fraction Vastus Lateralis 5 FF (%) Change From BL Part 1 Treated 4 Part 1 Placebo 3 2 1 0 0 52 104 Weeks Post Dosing Placebo N=16 N=14 N=17 N=15 Treated N=15 N=14 N=14 N=16 N=16 N=15 Stabilization in MRI muscle FF in ELEVIDYS treatment group with two years v
Slide 12: Cardiac MRI Shows Patients Remain in Normal Range Over 2 Years
Cardiac MRI Shows Patients Remain in Normal Range Over 2 Years of Follow-up No statistical or clinical differences in these groups Part 1 Treated: Year 1 post- Year 2 post- Crossover Year 1 post- Year 1 post- Baseline treatment treatment Treated: placebo treatment Baseline Subjects (N) 16 16 14 19 1
Slide 13: Results from EMBARK: 3-Year Topline Data, Part 1-treated vs EC
Results from EMBARK: 3-Year Topline Data, Part 1-treated vs EC (Analysis Using LS-Mean by MMRM) James Richardson, MA (Oxon), BMBCh, MBA, MRCP (Lon) Executive Vice President and Chief Medical Officer C O N F I D E N T I A L . F O R I N T E R N A L U S E O N L Y. ©SAREPTA THERAPEUTICS, INC. 2026. ALL
Slide 14: Background on the External Control (EC)
Background on the External Control (EC) • In the absence of a placebo arm after Part 1, the EMBARK study protocol and statistical analysis plan (SAP) are used to create a pre-specified, propensity-weighted external control to contextualize study level results • External control cohorts chosen throug
Slide 15: Baseline Characteristics Balanced for Part 1-treated and EC
Baseline Characteristics Balanced for Part 1-treated and EC Baseline Characteristics (Weighted) Full Analysis Set Disposition Baseline ELEVIDYS External Control Baseline Year 1 Year 2 Year 3 Mean (Min, Max) Mean (Min, Max) Cohort N* N* N* N* Age 5.98 (4.07, 7.87) 6.24 (4.24, 7.99) External Control 1
Slide 16: NSAA Total Score: Change from Baseline
ELEVIDYS-treated patients (n=52) achieved a significantly higher NSAA total score compared to the external control group, with a p-value of 0.0002 Mean age of patients 9 years old at time of last assessment Year 2 Year 3 Key Observations: 2.63 NSAA total score: change from baseline • The functional
Slide 17: TTR Change From Baseline
ELEVIDYS-treated patients demonstrated a highly significant 73% slowing of disease progression on TTR, with a p-value of < 0.0001 Mean age of patients 9 years old at time of last assessment Key Observations: TTRFF Change From Baseline 10 SRP-9001 Group ELEVIDYS (n=52) • The performance gap nearly 8
Slide 18: 10MWR Change From Baseline
ELEVIDYS-treated patients achieved a statistically significant 70% slowing of disease progression on 10MWR, with a p-value of 0.0039 Mean age of patients 9 years old at time of last assessment 5 Key Observations: 10MWR Change From BL SRP-9001 Group ELEVIDYS (n=52) 4 EC Cohort PSW (n=73) EC Group 3.8
Slide 19: Three-year Safety for Part 1 Treated Patients
Three-year Safety for Part 1 Treated patients Year 1 Year 2 Year 3 (EMBARK Y1) (EMBARK Y2)* (EXPEDITION Y1)^ Patients with any SAEs 14 5 4 Patients with treatment- 7 1 0 related SAEs Treatment-related 0 0 0Ŧ deaths • No new safety signals were observed • The safety profile of ELEVIDYS as observed i
Slide 20: Long-term Results from EMBARK Demonstrate Significant and Growing Functional Benefit
Long-term results from EMBARK demonstrate significant and growing functional benefit compared to EC • At the time of their last assessment, mean age of patients treated in EMBARK is 9 years old, corresponding to a nearly universal period of functional decline in Duchenne • On average, ELEVIDYS-treat
Slide 21: Dr. Crystal Proud
Dr. Crystal Proud Chief of Neurology and Director of Neuromuscular Medicine Children’s Hospital of The King’s Daughters ©SAREPTA THERAPEUTICS, INC. 2026. ALL RIGHTS RESERVED. 21
Slide 22: Q&A
Q&A C O N F I D E N T I A L . F O R I N T E R N A L U S E O N L Y. ©SAREPTA THERAPEUTICS, INC. 2026. ALL RIGHTS RESERVED. 22
Slide 23: SRP-9001-301 Part 1 3-Year Data Topline Results
SRP-9001-301 PART-1 3-Year Data Topline Results Doug Ingram Chief Executive Officer Louise R. Rodino-Klapac, Ph.D. President, R&D and Technical Operations James Richardson, MA (Oxon), BMBCh, MBA, MRCP (Lon) Executive Vice President, Chief Medical Officer DILLON Living with Duchenne muscular dystroph
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