Sarepta Therapeutics, Inc. · NASDAQ:SRPT
Sarepta Therapeutics — Corporate Update (July 2025)
39 slides from Sarepta Therapeutics's corporate update, Jul 16, 2025
Slide 1: Patients can't wait for the next breakthrough in medical research
July 16, 2025 Patients can’t wait for the next breakthrough in medical research. So neither will we. Doug Ingram CEO Ian Estepan President and COO Louise Rodino-Klapac, PhD President, R&D and Technical Operations DILLON Living with Duchenne muscular dystrophy ©SAREPTA THERAPEUTICS, INC. 2025. ALL RI
Slide 2: Advancing Sarepta Forward
Advancing Sarepta Forward Doug Ingram CEO ©SAREPTA THERAPEUTICS, INC. 2025. ALL RIGHTS RESERVED. 2
Slide 3: 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 4: Non-GAAP Financial Measures
Non-GAAP Financial Measures This presentation includes both GAAP information and Non-GAAP information. Non-GAAP research and development expenses are defined as GAAP research and development expenses excluding depreciation and amortization expense and stock-based compensation expense. Non-GAAP selli
Slide 5: Renewed strategy grounded in our patient-first, science-driven mission
Renewed strategy grounded in our patient-first, science-driven mission ©SAREPTA THERAPEUTICS, INC. 2025. ALL RIGHTS RESERVED. 5
Slide 6: Today's Focus
Today’s Focus Strategic Restructuring Update Pipeline Focus siRNA • Restructuring to reduce operating Facioscapulohumerol expenses and realign cost structure muscular dystrophy (FSHD) • Designed to meet debt obligations Myotonic Dystrophy • Proactively managing 2027 Type 1 (DM1) Convertible Notes Sp
Slide 7: Organizational Changes
Organizational Changes Ian Estepan Louise Rodino-Klapac, PhD President and Chief President, R&D and Technical Operations Operating Officer Rachael Potter, PhD Patrick Moss, PharmD Ryan Wong Chief Scientific Officer Executive Vice President and Executive Vice President and Chief Commercial Officer Ch
Slide 8: Restructuring Sarepta's Business to Support Long-term Growth
Restructuring Sarepta’s Business to Support Long-term Growth Ian Estepan President and Chief Operating Officer ©SAREPTA THERAPEUTICS, INC. 2025. ALL RIGHTS RESERVED. 8
Slide 9: Preliminary Q2 2025 Total Net Product Revenue and Cash
Preliminary Q2 2025 Total Net Product Revenue $513M1 PMOs $231M1 ELEVIDYS $282M1 Preliminary Q2 2025 Total Cash and Investments2 $850M1 Preliminary Q2 2025 Total Combined R&D and SG&A Expenses GAAP and Non-GAAP3 $338M1 and $294M1, respectively Footnotes 1. Q2 2025 Financials are subject to change up
Slide 10: Duchenne Portfolio of Four Marketed Therapies Expected to Continue Delivering Strong Revenue Stream
Duchenne Portfolio of Four Marketed Therapies Expected to Continue Delivering Strong Revenue Stream Illustrative Minimum Annual In the near-term, our Duchenne franchise is Net Product Revenue expected to continue driving profitability and Opportunity Through 20271 generating significant cash flow, f
Slide 11: Restructured Business Designed to Meet Debt Obligations and Maintain Access to Revolver
Restructured Business Designed to Meet Debt Obligations and Maintain Access to Revolver Strategic Decisions Financial Impact Prioritize Focused investment in our potential Focusing development resources on siRNA best-in-class siRNA platform toward a pipeline while continuing research and durable gro
Slide 12: New Cost Structure Strengthens Financial Position and Pathway to Repay 2027 Notes
New Cost Structure Strengthens Financial Position and Pathway to Repay 2027 Notes Illustrative Average Annual Key considerations guiding revised cost structure: Non-GAAP R&D and SG&A Expenses1 $ in M 1. Alignment with Portfolio Prioritization: Reduced $1,300 -$400 operating expenses to reflect prior
Slide 13: EBITDA Needed to Maintain Revolver Access Expected to be Achievable Under Conservative Revenue Scenario
EBITDA Needed to Maintain Revolver Access Expected to be Achievable Under Conservative Revenue Scenario Maintaining access to revolver requires minimum of $172M trailing 4-qtr EBITDA to meet negative financial covenants Financial Covenants Trailing 4-Qtr EBITDA Required Maximum Secured Net Leverage
Slide 14: The Strength of Our Science and Near-term Pipeline
The Strength of Our Science and Near-term Pipeline Louise Rodino-Klapac, PhD President, R&D and Technical Operations ©SAREPTA THERAPEUTICS, INC. 2025. ALL RIGHTS RESERVED. 14
Slide 15: ELEVIDYS Update
ELEVIDYS Update Label Supplement • ELEVIDYS remains on the market for the ambulant patient population • Working with FDA on label supplement – To ensure the communication of important safety information to prescribers and patients – Expected to include a Black Box warning on the risk of ALI/ALF Enha
Slide 16: Proposed Study Protocol for Immunosuppressive Regimen
Proposed Study Protocol for Immunosuppressive Regimen 6-month study adding sirolimus to standard immunosuppression in non-ambulatory population being proposed to FDA; follows engagement with expert committee Up to 31 Days Part 2 Screening Baseline 12 Weeks Study 305 (out to 6 months) 60 Days N up to
Slide 17: Three Distinct, Proprietary Scientific Platforms
Three Distinct, Proprietary Scientific Platforms RNA GENE THERAPY siRNA Skipping Replacement Knockdown Bypasses an error in the Adds a functional copy Suppresses RNA, allowing the body (a transgene) of a missing or overexpression to skip the mutation malfunctioning gene of mutations ©SAREPTA THERAPE
Slide 18: Sharpened Focus on siRNA Programs Resets Sarepta on Large Unmet Patient Needs
Sharpened Focus on siRNA Programs Resets Sarepta on Large Unmet Patient Needs Generating Leveraging Addressing Advancing Multiple, Sarepta’s Large, Unmet Potential Near-Term Expertise in Patient Needs Best-in-Class Clinical Data Neuromuscular Therapies Readouts Diseases ©SAREPTA THERAPEUTICS, INC. 2
Slide 19: Facioscapulohumeral muscular dystrophy (FSHD)
Facioscapulohumeral muscular dystrophy (FSHD) A rare genetic disease that causes weakness in the skeletal muscles. Progressively spreads from the face into other areas, including scapular girdle, upper limb, pelvic girdle, abdominal and leg muscles.1 • 95% of FSHD cases are linked to deletions of D4
Slide 20: SRP-1001 Reduces DUX4 mRNA in FSHD Patient Cells
SRP-1001 Reduces DUX4 mRNA in FSHD Patient Cells FSHD patient muscle cells express DUX4 in culture and is reduced with SRP-1001 treatment SRP-1001 reduces DUX4 in FSHD myotubes SRP-1001 prevents DUX4-dependent gene activation ©SAREPTA THERAPEUTICS, INC. 2025. ALL RIGHTS RESERVED. 20
Slide 21: SRP-1001 Reduces DUX4 in a FSHD mouse
SRP-1001 Reduces DUX4 in a FSHD mouse FSHD mouse model engineered to induce expression of human DUX4 in skeletal muscle SRP-1001 both prevents and reverses DUX4 mRNA in skeletal SRP-1001 prevents and reverses DUX4-dependent muscle from an FSHD mouse gene activation in skeletal muscle Baseline FSHD F
Slide 22: SRP-1001: Facioscapulohumeral muscular dystrophy (FSHD)
SRP-1001: Facioscapulohumeral muscular dystrophy (FSHD) Proof-of-concept (POC) of DUX4 knockdown and confirmation of registrational dose • Development Status: Phase 1/2 • Design: Placebo-controlled single ascending dose (Part 1) with placebo-controlled multiple ascending dose (Part 2) in FSHD1 • Par
Slide 23: Myotonic dystrophy type 1 (DM1)
Myotonic dystrophy type 1 (DM1) A form of muscular dystrophy that affects muscles and many other organs in the body.1 • Myotonic dystrophy (DM) is the most common form of muscular dystrophy. 2 • There are two types of DM: DM1 is caused by mutations in the DMPK gene and is generally more severe than
Slide 24: SRP-1003 Reduces DMPK in Non-Human Primates (NHP)
SRP-1003 Reduces DMPK in Non-Human Primates (NHP) DMPK mRNA is knocked down to around 80% in NHP skeletal muscle NHP Skeletal Muscle (triceps), IV administration ©SAREPTA THERAPEUTICS, INC. 2025. ALL RIGHTS RESERVED. 24
Slide 25: Reducing DMPK Fixes Splicing Deficit in a DM1 Mouse
Reducing DMPK Fixes Splicing Deficit in a DM1 Mouse DM1 mouse model engineered to induce expression of human DMPK with expanded CUG repeats SRP-1003 reduces pathological DMPK mRNA in the SRP-1003 reverses splicing deficits in DM1 mouse nucleus of a DM1 mouse model (>50% at high dose) model (~60% mis
Slide 26: SRP-1003: Myotonic dystrophy type 1 (DM1)
SRP-1003: Myotonic dystrophy type 1 (DM1) Proof of concept (POC) and confirmation of registrational dose • Development Status: Phase 1/2 • Design: Placebo-controlled single ascending dose (Part 1) with placebo-controlled multiple ascending dose (Part 2) • Participants: Cohorts 1-2 fully enrolled • P
Slide 27: Spinocerebellar ataxia type 2 (SCA2)
Spinocerebellar ataxia type 2 (SCA2) Spinocerebrellar ataxia (SCA) is a group of rare, genetic neurodegenerative disorders leading to severe disability and premature death.1 • In SCA, the nerve fibers carrying messages to and from the brain are affected, resulting in degeneration of the cerebellum (
Slide 28: SRP-1004 Reduces ATXN2 in a Mouse Brain
SRP-1004 Reduces ATXN2 in a Mouse Brain Mouse model engineered to express human ATXN2 SRP-1004 delivered ICV reduces ATXN2 SRP-1004 delivered ICV reduces ATXN2 mRNA 2 weeks after delivery protein 2 weeks after delivery ©SAREPTA THERAPEUTICS, INC. 2025. ALL RIGHTS RESERVED. 28
Slide 29: SRP-1004 Lowers ATXN2 in NHP Brain Regions Important in Disease
SRP-1004 Lowers ATXN2 in NHP Brain Regions Important in Disease >80% reduction in key brain regions (i.e. cerebellum) involved in SCA2 pathology SRP-1004 delivered IT reduces ATXN2 protein 1 month after delivery ©SAREPTA THERAPEUTICS, INC. 2025. ALL RIGHTS RESERVED. 29
Slide 30: SRP-1004: Spinocerebellar ataxia type 2 (SCA2)
SRP-1004: Spinocerebellar ataxia type 2 (SCA2) POC and confirmation of registrational dose • Development Status: Phase 1 • Design: • Randomized placebo-controlled single ascending dose • Multiple ascending dose Part 2 being added • Participants: Cohort 1 fully enrolled • Primary endpoint: Safety • S
Slide 31: Huntington's Disease (HD)
Huntington’s Disease (HD) A fatal genetic disorder that causes brain cells to gradually break down and die. HD deteriorates a person’s physical and mental abilities, and a person with HD is described as having ALS, Parkinson’s and Alzheimer’s disease simultaneously. 1 • HD is caused by a mutation in
Slide 32: SRP-1005 Reduces HTT in Mouse Brain
SRP-1005 Reduces HTT in Mouse Brain HTT siRNA linked to an antibody fragment targeting TfR1 enabling entry into the brain SRP-1005 delivered subcutaneously reduces mouse HTT protein 1 month post delivery Mouse model engineered to express human TfR1 ©SAREPTA THERAPEUTICS, INC. 2025. ALL RIGHTS RESERV
Slide 33: SRP-1005 Reduces HTT in NHP Brain Regions Important to Disease
SRP-1005 Reduces HTT in NHP Brain Regions Important to Disease SRP-1005 binds to NHP TfR1 to access the brain and reduce HTT protein levels SRP-1005 delivered SC (3 weekly doses) reduces HTT protein in key brain regions 43 days post delivery ©SAREPTA THERAPEUTICS, INC. 2025. ALL RIGHTS RESERVED. 33
Slide 34: Bolstering Pipeline with Preclinical Programs and up to 6 Discovery Targets
Bolstering Pipeline with Preclinical Programs and up to 6 Discovery Targets in Muscle (skeletal and cardiac) or CNS PRECLINICAL PROGRAMS DISCOVERY TARGETS • Preclinical CNS leverages TfR1-binding for • Up to 6 targets in muscle (skeletal and optimal CNS delivery cardiac) or CNS • Subcutaneous delive
Slide 35: Expected Near-Term Milestones (2025 / 2026)
Expected Near-Term Milestones (2025 / 2026) FSHD Huntington’s Disease SRP-1001 SRP-1005 • First Participant In (FPI) Cohorts 4 & 5 – Q3 2025 • Clinical Trial Application (CTA) filing – end of 2025 • Preliminary data (SAD Study) – 2H 2025 • Initiate trial – 1H 2026 DM1 LGMD2E/R4 SRP-1003 SRP-9003 •
Slide 36: Q&A
Q&A ©SAREPTA THERAPEUTICS, INC. 2025. ALL RIGHTS RESERVED. 36
Slide 37: Appendix
Appendix ©SAREPTA THERAPEUTICS, INC. 2025. ALL RIGHTS RESERVED. 37
Slide 38: Reconciliation of GAAP to Non-GAAP Total Combined R&D and SG&A Expenses – Preliminary Q2 2025
Reconciliation of GAAP to Non-GAAP Total Combined R&D and SG&A Expenses – Preliminary Q2 2025 $ in Millions Preliminary1 2Q 2025 Total Combined GAAP R&D and SG&A Expenses $338 Stock-based compensation expense ($34) Depreciation and amortization expense ($10) Total Combined Non-GAAP R&D and SG&A Expe
Slide 39: So neither will we.
July 16, 2025 Patients can’t wait for the next breakthrough in medical research. So neither will we. Doug Ingram CEO Ian Estepan President and COO Louise Rodino-Klapac, PhD President, R&D and Technical Operations DILLON Living with Duchenne muscular dystrophy ©SAREPTA THERAPEUTICS, INC. 2025. ALL RI