Sarepta Therapeutics, Inc. · NASDAQ:SRPT
Sarepta Therapeutics — J.P. Morgan 38th Annual Healthcare Conference (January 2020)
36 slides from Sarepta Therapeutics's conference presentation, Jan 13, 2020
Slide 1: 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 2: Some see slow and steady scientific progress. We see a revolution.
Some see slow and steady scientific progress. We see a revolution. DOUG INGRAM President and CEO J.P. Morgan 38th Annual Healthcare Conference January 13, 2020 LIAM Living with MPS IIIA
Slide 3: Making the revolution real
Making the revolution real ©Sarepta therapeutics, inc. 2020. All rights reserved. J.P. Morgan 38th Annual Healthcare Conference ©Sarepta therapeutics, inc. 2020. All rights reserved. J.P. Morgan 38th Annual Healthcare Conference
Slide 4: Broadened reach, expanded alliances, deepened pipeline, strengthened infrastructure
ADVANCED CLINICAL DEVELOPMENT BROADENED REACH EXPANDED ALLIANCES DEEPENED PIPELINE STRENGTHENED INFRASTRUCTURE EVOLVED GENE THERAPY MANUFACTURING
Slide 5: Deepened pipeline: our pipeline in 2018
DEEPENED PIPELINE 0 PROGRAMS 11 PROGRAMS Sarepta-driven programs Sarepta’s partnered programs GENE THERAPY GENE EDITING ENABLING TECHNOLOGIES GENE THERAPY RNA 14 PROGRAMS Our Pipeline in 2018 25 programs in all
Slide 6: Deepened pipeline: our pipeline in 2019
DEEPENED PIPELINE 6 PROGRAMS 14 PROGRAMS Sarepta-driven programs Sarepta’s partnered programs GENE THERAPY GENE EDITING ENABLING TECHNOLOGIES GENE THERAPY RNA 22 PROGRAMS Our Pipeline in 2019 Potential to treat 1.5M patients* *Based on published epidemiology 42 programs in all
Slide 7: Tools and enablers: AAV technology
DEEPENED PIPELINE Tools and Enablers Looking to the Future: Tools and Enablers AAV Technology Collaborations University of Massachusetts Medical School, Dr. Guangping Gao Focused on developing novel human-derived vectors StrideBio Enhance targeting to tissues of interest Expand treatable populations
Slide 8: Gene Editing Innovation Center in Durham, NC
DEEPENED PIPELINE Gene Editing Sarepta establishes Gene Editing Innovation Center (GEIC) in Durham, NC Charlie Gersbach, Ph.D. of Duke University to lead GEIC Pursuing gene editing approaches potentially available for majority of DMD patients Differentiated scientific approach Proprietary dual cut s
Slide 9: Research collaboration with Harvard University
DEEPENED PIPELINE Gene Editing Sarepta establishes multi-year Research collaboration with Harvard University Amy Wagers, Ph.D., Harvard Stem Cell Institute, to lead research project Pursuing in-vivo genome editing approach to treat DMD Unique strategy Fully and precisely restore function of the dyst
Slide 10: The disease: Duchenne muscular dystrophy (DMD)
ADVANCED CLINICAL DEVELOPMENT Gene Therapy The disease Duchenne muscular dystrophy (DMD) On average, every day DMD takes the life of a child in the United States DMD affects approximately 1 in 3,500-5,000 males worldwide1 DMD is a rare, fatal neuromuscular genetic disease inherited in an X-linked re
Slide 11: Differentiated gene therapy for SRP-9001
The science Sarepta’s differentiated gene therapy for SRP-9001 VECTOR PROMOTER TRANSGENE Provides systemic delivery to muscle cells, including the heart and skeletal muscle Low level of pre- existing immunity AAVrh74 Designed to generate a functional micro-dystrophin Includes SR2 and SR3 - essential
Slide 12: SRP-9001 early and nine-month clinical results
EARLY CLINICAL RESULTS MICRO-DYSTROPHIN GENE THERAPY FOR DMD* Expression of micro-dystrophin in muscle fibers1 Expression of micro-dystrophin measured by Western blot**,1 81% 96% 64% Expression of micro-dystrophin measured by signal intensity1 96% Reduction of creatine kinase1 1. Clinical Update: Mi
Slide 13: 2019 progress: SRP-9001 study 102 and study 301
2019 Progress Study 102 Data read out scheduled for early 2021 2019 Progress Study 301 Achieved commercially viable yields Dosed 41 patients Dosed placebo cross-over patients Study is progressing well and uninterrupted On track to commence study mid-2020 The progress SRP-9001 – study 102 and study 3
Slide 14: The disease: Limb-girdle muscular dystrophies (LGMDs)
The disease Limb-girdle muscular dystrophies (LGMDs) *Prevalence estimates range from 0.56 to 5.75 per 100,0001 1. Liewluck T, Milone M, et al. Untangling the complexity of limb-girdle muscular dystrophies. Muscle Nerve. 2018;58(2):167-177. 2. Murphy AP, Straub V. The Classification, Natural History
Slide 15: SRP-9003 gene therapy for LGMD2E
The science SRP-9003 gene therapy for LGMD2E VECTOR PROMOTER TRANSGENE AAVrh74 b-SARCOGLYCAN MHCK7 Designed to transduce skeletal and cardiac muscle with a gene that codes for the full-length, native β-sarcoglycan protein (the lack of which causes LGMD2E) ADVANCED CLINICAL DEVELOPMENT Gene Therapy
Slide 16: LGMD2E demonstrated consistent results
The clinical data LGMD2E demonstrated consistent results PRELIMINARY CLINICAL RESULTS b-SARCOGLYCAN GENE THERAPY FOR LIMB-GIRDLE MUSCULAR DYSTROPHY TYPE 2E*,1,2 Expression of b-sarcoglycan in muscle fibers Expression of b-sarcoglycan measured by Western blot** Reduction of creatine kinase 51% 47% 36
Slide 17: Clinical study underway for SRP-9003 in LGMD2E
The progress Clinical study underway for SRP-9003 in patients with LGMD2E A Single-Center, Open-Label, Systemic Gene Delivery Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9003 Administered by Systemic Infusion in Subjects With LGMD2E (β-Sarcoglycan Deficiency) Read out from high-d
Slide 18: Gene therapy engine at work across LGMD programs
Gene therapy engine at work across LGMD programs LGMD2B LGMD2C LGMD2D LGMD2E LGMD2L ADVANCED CLINICAL DEVELOPMENT Gene Therapy
Slide 19: The disease: Mucopolysaccharidosis Type IIIA (MPS IIIA)
The disease Mucopolysaccharidosis Type IIIA (MPS IIIA) MPS IIIA has a worldwide incidence of up to 1.62 per 100,000 live births1 Signs and symptoms usually become apparent in early childhood and include, developmental delays (e.g., speech problems), challenging behaviors, extreme hyperactivity and p
Slide 20: LYS-SAF302, partnered with Lysogene, for MPS IIIA
The science LYS-SAF302, partnered with Lysogene, a gene therapy for MPS IIIA AAVrh10 SGSH CAG VECTOR PROMOTER TRANSGENE ADVANCED CLINICAL DEVELOPMENT Gene Therapy
Slide 21: LYS-SAF302 in clinical development for MPS IIIA
The progress LYS-SAF302, a gene therapy in clinical development for patients with MPS IIIA Initiated Phase 2/3 study in MPS IIIA patients Completed 2-year natural history study in MPS IIIA (serve as control arm for Phase 2/3) Enrolled 15 patients in Phase 2/3 study Complete dosing (N=20) – H1 2020 2
Slide 22: Proprietary PMO technology
The science: Proprietary PMO Technology Phosphorodiamidate morpholino oligomer (PMO) Technology Specificity: Enhanced affinity for targeting pre-mRNA for precise binding to the selected RNA target Stability: Highly resistant to degradation by enzymes Versatility: Ability to rapidly design and constr
Slide 23: 2019 accomplishments: Proprietary PMO technology
Completed U.S. submission for VYONDYS 53 (Q1 2019) Announced positive dystrophin expression results for casimersen to treat exon 45 amenable patients (Q1 2019) Received a Complete Response Letter (CRL) for VYONDYS 53 (August 2019) Received approval for VYONDYS 53 (December 2019) The fastest CRL reve
Slide 24: EXONDYS revenue since 2016 launch
EXONDYS 51 Revenue Since 2016 Launch* ADVANCED CLINICAL DEVELOPMENT RNA *Candidate received accelerated approval in the U.S., confirmatory studies required $5.4 $154 $301 $365– 375 $400 300 200 100 0 $ Millions 2016 2017 2018 Original 2019 Guidance $370– 380 Revised 2019 Guidance Q4 revenue $100.1M
Slide 25: Casimersen (SRP-4045) and 3 PMOs in 2020
CASIMERSEN (SRP-4045) December 2019: Approved to treat patients with a confirmed genetic mutation that is amenable to exon 53 skipping (8% of DMD population)* January 2020: Initiated rolling NDA submission (8% of DMD population) Poised to have 3 PMOs serving the community in 2020 ADVANCED CLINICAL D
Slide 26: PPMO next-generation technology
Enhances PMO Conjugated peptide greatly increases cell penetration Could potentially lead to more efficient dosing for patients Able to deliver PMOs to unique muscle types (e.g., heart) The science: PPMO Next-Generation Technology Sarcolemma ADVANCED CLINICAL DEVELOPMENT RNA Peptide phosphorodiamida
Slide 27: PPMO: SRP-5051 2019 progress
The science: PPMO Next-Generation Technology Safety and dosing insights in mid-2020 2019 Progress Study for SRP-5051 Initiated MAD study in patients with DMD ADVANCED CLINICAL DEVELOPMENT RNA
Slide 28: Outstanding talent
Outstanding talent We have grown to nearly 800 employees across 4 sites in the U.S. Clinical Development 65 Regulatory Affairs & Quality 47 Technical Operations 46 Research 26 STRENGTHENED INFRASTRUCTURE The number of employees shown reflects headcount added in 2019 and not the total number in the f
Slide 29: Expanded locations and capabilities
Expanded locations and capabilities Columbus, Ohio Gene Therapy Center of Excellence Overview Planned expansion of facility in 2020 All employees have deep AAV expertise Strategic Imperatives Execute on current internal and partnered programs Continue to build an enduring gene therapy engine Advance
Slide 30: Significant progress on gene therapy manufacturing
Significant progress on gene therapy manufacturing Expanded Relationships with Thermo Fisher (Brammer) and Catalent (Paragon) to increase capacity Broad access to capacity in two locations Includes dedicated capacity and additional expansion options Completed single-use SRP-9001 facility at Lexingto
Slide 31: Expanded alliances
University of Massachusetts Medical School Lysogene Columbia University Duke University Genethon Harvard University Institute of Myology Lacerta Therapeutics Murdoch University Nationwide Children’s Hospital Roche StrideBio University of Florida College of Medicine University of Minnesota EXPANDED A
Slide 32: Sarepta and Roche: ex-US strategic alliance
BROADENED REACH Sarepta and Roche: Ex-US Strategic Alliance, SRP-9001 micro-dystrophin gene therapy for DMD Ranked as the largest gene therapy licensing deal and the largest single candidate ex-US licensing deal in history Possibility of success greatly increased Together, we will reach more DMD pat
Slide 33: A new addition to Sarepta's Board
A New Addition to Sarepta’s Board Grew portfolio to 24 approved therapies (total annual revenues, $32B+) Transformed Gilead’s HIV and Hepatitis C portfolios, benefitting patients worldwide John C. Martin, Ph.D. Industry veteran; former Chairman, CEO and President, Gilead Sciences
Slide 34: Upcoming milestones (next 12-18 months)
Upcoming milestones (Next 12-18 Months) SRP-9001 (micro-dystrophin) Gene Therapy Complete commercial process development and analytical development (H1) Gain insights from U.S. Food and Drug Administration on CMC and analytical assays for study 301 (Q2) Commence study 3 (mid-year) Expression and fun
Slide 35: Patients living with Duchenne, MPS IIIA and LGMD2E
Billy LIVING WITH DUCHENNE Lulu LIVING WITH MPS IIIA Connor LIVING WITH DUCHENNE Jamison LIVING WITH LGMD2E
Slide 36: #DragTomorrowIntoToday
#DragTomorrowIntoToday ZYLER Living with Duchenne SAREPTA, SAREPTA THERAPEUTICS, the helix logo, EXONDYS, and EXONDYS 51 are trademarks of Sarepta Therapeutics, Inc. registered in the U.S. Patent and Trademark Office and may be registered in various other jurisdictions. VYONDYS, VYONDYS 53 and DRAG
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