Sarepta Therapeutics, Inc. · NASDAQ:SRPT

Sarepta Therapeutics — Q3 2025 Earnings Presentation

42 slides from Sarepta Therapeutics's earnings presentation, Nov 3, 2025

Slide 1: Q3 2025 Earnings Conference Call
November 3, 2025 Q3 2025 Earnings Conference Call Patients can’t wait for the next breakthrough in medical research. So neither will we. Doug Ingram CEO Louise Rodino-Klapac, PhD President, R&D and Technical Operations Patrick E. Moss, PharmD Executive Vice President, Chief Commercial Officer Ryan H
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: Non-GAAP Financial Measures
Non-GAAP Financial Measures This presentation includes both GAAP information and Non-GAAP information. Non-GAAP (loss) income is defined as GAAP net (loss) income excluding interest income, net, depreciation and amortization expense, stock-based compensation expense, restructuring charge, (gain) los
Slide 4: Contents
Contents ESSENCE Topline Results Doug Ingram and Louise Rodino-Klapac, Ph.D. 5-20 Q3 Performance Doug Ingram 21-22 R&D Highlights Dr. Rodino-Klapac 23-29 Commercial Performance Patrick Moss, PharmD 30-31 Financial Results Ryan Wong 32-35 ©SAREPTA THERAPEUTICS, INC. 2025. ALL RIGHTS RESERVED. 4
Slide 6: ESSENCE update
ESSENCE update Confirmatory Trial Completion • Completed ESSENCE confirmatory trial for AMONDYS and VYONDYS which serve ultra-rare populations where disease progression is heterogenous and slow Evidence • Primary endpoint missed statistical significance; however, data demonstrated consistent clinica
Slide 7: VYONDYS and AMONDYS
VYONDYS 53 and AMONYDYS 45 December 2019 February 2021 Approved to treat patients with a confirmed genetic Approved to treat patients with a confirmed genetic mutation that is amenable to exon 53 skipping* mutation that is amenable to exon 45 skipping* *Candidate received accelerated approval in the
Slide 8: ESSENCE (SRP-4045-301): Trial design
ESSENCE* (SRP-4045-301): Trial design Double-blind, placebo-controlled, multi-center study to evaluate the efficacy and safety of casimersen and golodirsen in boys with Duchenne ages 6 to 13 years old; 75 sites across 24 countries Double-Blind Period (96 wk) Open-label Period (+48 wk) Casimersen 30m
Slide 9: Demographics: Cohorts were well-balanced
Demographics: Cohorts were well-balanced Placebo Treated Overall Characteristic Statistics (N=77) (N=148) (N=225) Mean (SD) 9.4 (1.85) 9.4 (2.01) 9.4 (1.95) Age (years) Min, Max 7, 14 6, 14 6, 14 6-8.5 years n (%) 30 (39.0%) 62 (41.9%) 92 (40.9%) > 8.5-13 years n (%) 47 (61.0%) 86 (58.1%) 133 (59.1%
Slide 10: Study results
Study results • Despite numerical superiority, ESSENCE did not meet the primary endpoint of change from BL at W96 in 4-step ascend velocity (4SA) in ITT population – LS mean difference 0.055 steps/second, p-value= 0.309 • COVID impacted study; a post hoc analysis of participants not impacted by COVI
Slide 11: CFB in 4-step Ascend Velocity (step/seconds)
Results of 4-SA Velocity (steps/second) at 96 weeks Primary endpoint indicates a positive trend favoring the treated population 0.0 - Difference Placebo Treated CFB in 4-step Ascend Velocity (step/seconds) Statistics (Treated vs. P-value (N=77) (N=148) Placebo) Active -0.1 - LS Mean -0.3320 -0.2770
Slide 12: Enrollment relative to COVID period
Enrollment relative to COVID period (March 2020 – July 2021) double-blind period COVID Period 43% of COVID impacted participants had consecutively missed doses (on average 8 consecutive doses missed) COVID Period ©SAREPTA THERAPEUTICS, INC. 2025. ALL RIGHTS RESERVED. 12
Slide 13: 4-Step Ascend Velocity (step/second)
Mean trend in participants not impacted by COVID demonstrates meaningful change Covid Free (n=168) Covid Impacted (n=57) 4-Step Ascend Velocity (step/second) Active Placebo Covid free W96 LS Mean Diff = 0.11 p = 0.09 ~30% reduction in disease progression over 2 years on the 4SA ©SAREPTA THERAPEUTICS
Slide 14: 4-SA velocity endpoint significant with prognostic score
The 4-SA velocity endpoint reached statistical significance when the prognostic score was applied >=5th - <=55th Percentile Prognostic score: 0.00 • Composite score to predict future 4-SA velocity based on baseline function – Lower score predicts greater degree of 4-SA velocity decline -0.25 LS-Mean
Slide 15: Safety summary: Strong safety profile
Safety summary: Strong safety profile • Adverse events (AEs) were well tolerated – The incidence of AEs in the treated group (97.3%) was comparable to placebo (98.7%) • Of the 3722 treatment-emergent adverse events (TEAEs), most were mild (88.6%) or moderate (10.3%) in severity and resolved without
Slide 16: Real-world evidence supporting casimersen
Real-world evidence supporting casimersen 15 YEARS Mean age of casimersen-treated patients at first 70% wheelchair claim vs 9.5 to 12.3 years in literature for standard of care1 0 PATIENTS 2.63pp ATTENUATION in FVC%p decline for casimersen- REDUCTION Treated with casimersen have reported LOA up trea
Slide 17: Real-world evidence supporting golodirsen
Real-world evidence supporting golodirsen 3 YEAR DELAY 88% RISK REDUCTION in LOA vs matched external controls (p=0.0016)2 in LOA vs matched external controls over up to 6 years2 90% LOWER Annual rate of tracheostomy 13 YEARS vs matched controls4 70% average age of LOA among patients who lost ambulat
Slide 18: Body of real-world evidence support PMOs
Body of real-world evidence support PMOs 5.4 3-4 YEAR 3-7 YEAR 50-90% 78% 30% YEAR increase delay in delay in need reduction in reduction in risk reduction in in survival loss of for nighttime assisted of reaching ER and ambulation ventilation and ventilation and LVEF<55% and hospital visits signifi
Slide 19: Clinical experience and real-world evidence support PMOs
Clinical experience and real-world evidence support PMOs With a patient adherence rate of more than 90%, this sustained use reflects the clinical value of our PMOs PMO therapies have treated over 1,800 patients worldwide from infants to adults in their 30s Data presented at 2025 WMS showed casimerse
Slide 20: Status and next steps
Status and next steps • Shared initial top line results and analysis with FDA • Plan to schedule meeting with FDA to discuss pathway to traditional approval • Complete the analysis of study results • Share data at future scientific forums and publish in a peer-reviewed journal ©SAREPTA THERAPEUTICS,
Slide 22: Q3 2025 and Recent Highlights
Q3 2025 and Recent Highlights R&D Highlights Financial Highlights Corporate Highlights • ESSENCE: Totality of evidence • Financial strategy executed in Q3 has • Solid ELEVIDYS performance favors risk/benefit profile enhanced our near-term liquidity and despite navigating challenging • ELEVIDYS Label
Slide 25: Safety label updates
Safety label updates Label Supplement • ELEVIDYS remains approved and available for the ambulant patient population • Agreed with FDA to black box warning and removal of non-ambulatory population from the Indication and Usage section of the Prescribing Information Enhanced Immunosuppressive Regimen
Slide 26: Expert Committee presents findings at 2025 World Muscle Society meeting
Expert Committee presents findings at 2025 World Muscle Society (WMS) meeting Committee discussed potential of adding an additional immunosuppression regimen for the non-ambulant population • Committee comprised of global leaders and experts in neuromuscular physicians with ELEVIDYS treatment experi
Slide 27: Results of independent sirolimus study presented at WMS meeting
Results of independent sirolimus study presented at WMS meeting • Study overview – 20 Duchenne patients received ELEVIDYS, first 14 patients received ELEVIDYS with a standard protocol including corticosteroids but no additional immunosuppression; 6 subsequent patients underwent a modified immunosupp
Slide 29: Expected near-term milestones
Expected near-term milestones LGMD2E/R4 FSHD SRP-9003 SRP-1001 • FDA meeting – Q4 2025 • Preliminary data (SAD and MAD study) – Early 2026 DM1 Huntington’s Disease SRP-1003 SRP-1005 • Preliminary data (SAD and MAD study) – • Initiate trial – Year-end 2025 Early 2026 ©SAREPTA THERAPEUTICS, INC. 2025.
Slide 31: Commercial update on performance and outlook
Commercial update on performance and outlook Q3 2025 Performance $ in Millions • ELEVIDYS Demand Demonstrated Resilience Amid Disruptions: $1,495 – Temporary voluntary pause in shipment to ambulatory population resulted in meaningful disruptions – New patient identification efforts delayed – Postpon
Slide 33: Financial highlights
Financial highlights Q3 2025 Financial Results Operating Loss Cash and Total Revenues Investments2 GAAP / Non-GAAP1 $399 million ($103) / ($36) million $865 million Proactive Steps Taken to Strengthen our Balance Sheet and 2025 On Track to Over-Achieve Restructuring Savings • Excluding Arrowhead col
Slide 34: Q3 2025 select financial data
Q3 2025 select financial data Q3 Q3 YoY $ In Millions, except percentages 2025 2024 % Total Product Revenue $370 $430 (14%) Collaboration and Other Revenues $29 $37 Total Revenues $399 $467 Cost of Sales (excludes amortization of in-licensed rights) $151 $92 Combined GAAP R&D and SG&A Expenses $311
Slide 35: FY 2025 updated expense guidance
FY 2025 updated expense guidance Updated Expense Guidance FY 2025 Assumptions As of Nov 3, 2025 Combined Non-GAAP R&D and Includes Arrowhead collaboration transaction costs and SG&A Expenses1 ~ $1,860M milestones Upfront transaction cost ($584M reported in Q1), DM1 Less: Arrowhead collaboration ($88
Slide 38: Condensed Consolidated Statements of Income (Loss)
Condensed Consolidated Statements of Income (Loss) For the Three Months Ended For the Nine Months Ended $ In Thousands, except per share amounts September 30, September 30, 2025 2024 2025 2024 Revenues: Products, net $ 370,043 $ 429,771 $ 1,494,689 $ 1,149,803 Collaboration and other 29,313 37,401 2
Slide 39: Reconciliation of GAAP Reported Net Income to Non-GAAP Net Income
Reconciliation of GAAP Reported Net Income to Non-GAAP Net Income $ In Thousands, except per share amounts Note: Tables may not foot due to rounding ©SAREPTA THERAPEUTICS, INC. 2025. ALL RIGHTS RESERVED. 39
Slide 40: Reconciliation of GAAP to Non-GAAP Tax Rate, Operating Income, and Expenses
Reconciliation of GAAP to Non-GAAP Reported Total Effective Tax Rate, Operating Income, and SG&A and R&D Expenses For the Three Months Ended For the Nine Months Ended $ In Thousands September 30, September 30, 2025 2024 2025 2024 Total effective tax rate, GAAP 7.3 % 1.2 % (1.6) % 14.4 % Less: impact
Slide 41: PMO Revenue Breakdown by Product
PMO Revenue Breakdown by Product Prior Quarter For the Three For the Three Months Ended For the Nine Months Ended Months Ended $ In Thousands September 30, September 30, June 30, 2025 2024 2025 2024 2025 Exondys 51 126,208 140,718 390,019 390,708 126,390 Vyondys 53 32,531 32,170 93,995 97,236 33,478
Slide 42: Q3 2025 Earnings Conference Call
November 3, 2025 Q3 2025 Earnings Conference Call Patients can’t wait for the next breakthrough in medical research. So neither will we. Doug Ingram CEO Louise Rodino-Klapac, PhD President, R&D and Technical Operations Patrick E. Moss, PharmD Executive Vice President, Chief Commercial Officer Ryan H

Sarepta Therapeutics — Q3 2025 Earnings Presentation on 6ix

Sarepta Therapeutics — Q3 2025 Earnings Presentation

Slide 1: Q3 2025 Earnings Conference Call

Slide 2: Forward-looking statements

Slide 3: Non-GAAP Financial Measures

Slide 4: Contents

Slide 5: ESSENCE Topline Readout

Slide 6: ESSENCE update

Slide 7: VYONDYS and AMONDYS

Slide 8: ESSENCE (SRP-4045-301): Trial design

Slide 9: Demographics: Cohorts were well-balanced

Slide 10: Study results

Slide 11: CFB in 4-step Ascend Velocity (step/seconds)

Slide 12: Enrollment relative to COVID period

Slide 13: 4-Step Ascend Velocity (step/second)

Slide 14: 4-SA velocity endpoint significant with prognostic score

Slide 15: Safety summary: Strong safety profile

Slide 16: Real-world evidence supporting casimersen

Slide 17: Real-world evidence supporting golodirsen

Slide 18: Body of real-world evidence support PMOs

Slide 19: Clinical experience and real-world evidence support PMOs

Slide 20: Status and next steps

Slide 21: Q3 Performance

Slide 22: Q3 2025 and Recent Highlights

Slide 23: R&D Highlights

Slide 24: ELEVIDYS Updates

Slide 25: Safety label updates

Slide 26: Expert Committee presents findings at 2025 World Muscle Society meeting

Slide 27: Results of independent sirolimus study presented at WMS meeting

Slide 28: Pipeline Updates

Slide 29: Expected near-term milestones

Slide 30: Commercial Performance

Slide 31: Commercial update on performance and outlook

Slide 32: Financial Results

Slide 33: Financial highlights

Slide 34: Q3 2025 select financial data

Slide 35: FY 2025 updated expense guidance

Slide 36: Q&A

Slide 37: Appendix

Slide 38: Condensed Consolidated Statements of Income (Loss)

Slide 39: Reconciliation of GAAP Reported Net Income to Non-GAAP Net Income

Slide 40: Reconciliation of GAAP to Non-GAAP Tax Rate, Operating Income, and Expenses

Slide 41: PMO Revenue Breakdown by Product

Slide 42: Q3 2025 Earnings Conference Call