Sarepta Therapeutics, Inc. · NASDAQ:SRPT
Sarepta Therapeutics — 44th Annual J.P. Morgan Healthcare Conference (January 2026)
24 slides from Sarepta Therapeutics's conference presentation, Jan 12, 2026
Slide 1: 44th Annual J.P. Morgan Healthcare Conference
DOUG INGRAM MAX, age 10 Chief Executive Officer Dosed with ELEVIDYS Sarepta Therapeutics, Inc. (NASDAQ:SRPT) at age 5 44th Annual J.P. Morgan Healthcare Conference San Francisco, California JANUARY 12, 2026 ©SAREPTA THERAPEUTICS, INC. 2026. ALL RIGHTS RESERVED. 1
Slide 2: Forward-looking statements
Forward-looking statements In order to provide Sarepta’s investors with an understanding of its current results and future prospects, forward-looking statements will be made during this presentation. Any statements that are not statements of historical fact may be deemed to be forward-looking statem
Slide 3: Substantial Duchenne opportunity
Substantial Duchenne opportunity Strong 1 ~80% of the addressable ambulatory population remain untreated with ELEVIDYS; Fundamentals PMOs continue to support revenue trajectory Position Sarepta well into the Future ©SAREPTA THERAPEUTICS, INC. 2026. ALL RIGHTS RESERVED. 3
Slide 4: Strong Fundamentals Position Sarepta well into the Future
Substantial Duchenne opportunity Strong 1 ~80% of the addressable ambulatory population remain untreated with ELEVIDYS; Fundamentals PMOs continue to support revenue trajectory Position Next-generation siRNA pipeline Sarepta well 2 Preclinical data support potential differentiation into the of our p
Slide 5: Strong Fundamentals Position Sarepta well into the Future
Substantial Duchenne opportunity Strong 1 ~80% of the addressable ambulatory population remain untreated with ELEVIDYS; Fundamentals PMOs continue to support revenue trajectory Position Next-generation siRNA pipeline Sarepta well 2 Preclinical data support potential differentiation into the of our p
Slide 6: Preliminary Q4 and Full-year 2025 Results
Total Net Product Revenue Preliminary Q4 2025 $370M 2 Full-year total $1.86B 2 Q4 and Full-year ELEVIDYS Net Product Revenue 2025 Results Q4 2025 $110M2 Full-year total 899M $ 2 Cash and Investments (as of 12/31/25) PMO Net Product Revenue $954M 1, 2 Q4 2025 259M $ 2 1. Includes cash, cash equivalen
Slide 7: ELEVIDYS Today
Strategic approach ELEVIDYS • Focus on HCP segmentation and outreach (e.g., sites of care) Today • • Provide deep dive opportunities around the data and science Expand field force • Enhance understanding of ELEVIDYS 80% among patient and advocacy communities ~ • Cohort 8: Data generated within the n
Slide 8: ELEVIDYS Impact
>1,100 EMBARK Part 1 ELEVIDYS met all key secondary patients endpoints treated Impact All data EMBARK Part 2 achieved confirmed by statistical ELEVIDYS is designed to treat the underlying cause of muscle MRI significance on key endpoints* Duchenne muscular dystrophy, impacting the trajectory of the
Slide 9: ELEVIDYS Impact: EMBARK Part 1 NSAA Total Score
EMBARK Part 2 NSAA* Total Score: ELEVIDYS Baseline to Year 2, blinded study results Crossover date 27 Impact 26 NSAA Total Score 25 24 Part 1 Treated Part 1 Placebo 23 22 0 26 52 78 104 Time in Weeks *North Star Ambulatory Assessment ©SAREPTA THERAPEUTICS, INC. 2026. ALL RIGHTS RESERVED. 9
Slide 10: ELEVIDYS Impact: EMBARK Part 1 NSAA Total Score
EMBARK Part 2 NSAA* Total Score: ELEVIDYS Baseline to Year 2, blinded study results Crossover date 27 Impact 26 NSAA Total Score 25 24 Part 1 Treated Part 1 Placebo 23 22 0 26 52 78 104 Time in Weeks *North Star Ambulatory Assessment ©SAREPTA THERAPEUTICS, INC. 2026. ALL RIGHTS RESERVED. 10
Slide 11: ELEVIDYS Impact: EMBARK NSAA Total Score with Crossover
EMBARK Part 2 NSAA* Total Score: ELEVIDYS Baseline to Year 2, blinded study results Crossover date 27 Impact 26 NSAA Total Score 25 24 Part 1 Treated Part 1 Placebo 23 Crossover Treated 22 0 26 52 78 104 Time in Weeks *North Star Ambulatory Assessment ©SAREPTA THERAPEUTICS, INC. 2026. ALL RIGHTS RES
Slide 12: ELEVIDYS Impact: Two-Year Musculoskeletal MRI Results
Two-Year Results: Musculoskeletal MRI - ELEVIDYS fat fraction (FF) Impact Vastus Lateralis Vastus Lateralis Soleus 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 • Naarding KJ, Reyngoudt H,
Slide 13: PMO Impact
>1,800 10+ PMO patients worldwide ranging from infants to adults in their 30’s years of clinical and commercial Impact experience and over EXONDYS 51, Established safety 2,300+years of life VYONDYS 53 & profile added to patients who have been AMONDYS 45 on therapy Sarepta’s RNA-based PMOs Slowing di
Slide 14: PMO Impact: Real-world evidence
Real-world evidence support Sarepta’s PMO on market PMO therapies 5.4 3-4 3-7 Impact YEAR increase in survival YEAR delay in loss of ambulation YEAR delay in need for nighttime ventilation and significantly EXONDYS 51, slower pulmonary decline VYONDYS 53 & AMONDYS 45 50-90 % reduction in assisted 78
Slide 15: PMO Impact: Mean trend in participants not impacted by COVID
Mean trend in participants not impacted by PMO COVID demonstrates meaningful change Covid Free (n=168) Covid Impacted (n=57) Impact SRP Placebo 4-Step Ascend Velocity (step/second) Active EXONDYS 51, VYONDYS 53 & Placebo AMONDYS 45 ~30 % Covid free W96 LS Mean Diff = 0.11 reduction in disease p = 0.
Slide 16: siRNA Pipeline
DISCOVERY/PRECLINICAL CLINICAL siRNA U.S. patient prevalence αv 6 integrin peptide MUSCLE SRP-1001 Facioscapulohumeral muscular dystrophy, Type 1 (FSHD1) ~16,0001 Pipeline SRP-1003 LUNG Myotonic dystrophy, Type 1 (DM1) ~40,0002 SRP-1002 Idiopathic pulmonary fibrosis (IPF) ~60,0003 CNS SRP-1004 Spin
Slide 17: TRiM Platform: A next-generation approach
A next-generation approach TRiM Delivery Platform Superior tissue targeting siRNA Design Effective and efficient target knock-down, improved durability, specificity, and reduction of off-target effects Safety/Dosing • Favorable safety profile • Potential to dose higher vs other therapies ©SAREPTA TH
Slide 18: Optimal components support best-in-class therapies in FSHD and DM1
Better delivery to muscle with integrin αv6 Optimal Optimal clinical impact αv6 TfR1 components ASO mAb support Higher dosing potential Better muscle penetration (e.g., delivers more Rx to muscle) best-in-class Safety therapies Potency in FSHD and DM1 The tolerability of the αv6-targeting platfor
Slide 19: Our approach to crossing the blood-brain barrier
SYNERGISTIC NEXT-GENERATION APPROACH EXISTING APPROACH Divalent Monovalent Delivery Our Binding Binding to TfR1 with fAb promotes Pharmacokinetics of subcutaneous delivery increases the time window approach to TfR1 with mAb promotes receptor degradation and/or receptor transcytosis and of TfR1-ligan
Slide 20: TfR1-binding designed to optimize CNS delivery in Huntington's disease
TRiM CNS-SC platform demonstrates improved delivery to deep brain in NHP TfR1-binding compared to direct CSF injection designed to CSF delivery via IT injection Systemic delivery via SC injection SRP-1005 in NHP optimize CNS delivery in HTT Protein (Relative to control) 1.0 Huntington’s disease (HD)
Slide 21: Strong Financial Foundation
• Efficient cost structure fueling future Strong profitability Financial – Strategic expense management and financial discipline enables sustainable profit and cash flow generation Foundation • Robust cash position – Base business cash flow positive (excluding Arrowhead transaction and milestone pay
Slide 22: Upcoming Milestones
Upcoming GENE THERAPY RNA siRNA Milestones ELEVIDYS Duchenne EXONDYS 51 Duchenne SRP-1001 FSHD ENDEAVOR Cohort 8 MIS51ON data readout Phase 1/2 preliminary data Numerous value-building complete primary – end of 2026 – 1Q 2026 endpoint data collection milestones expected – 2H 2026 throughout 2026, ac
Slide 23: We know who we're fighting for
We know who we’re fighting for ©SAREPTA THERAPEUTICS, INC. 2026. ALL RIGHTS RESERVED. 23
Slide 24: 44th Annual J.P. Morgan Healthcare Conference
DOUG INGRAM MAX, age 10 Chief Executive Officer Dosed with ELEVIDYS Sarepta Therapeutics, Inc. (NASDAQ:SRPT) at age 5 44th Annual J.P. Morgan Healthcare Conference San Francisco, California JANUARY 12, 2026 ©SAREPTA THERAPEUTICS, INC. 2026. ALL RIGHTS RESERVED. 24
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