Sarepta Therapeutics, Inc. · NASDAQ:SRPT
Sarepta Therapeutics — 36th Annual J.P. Morgan Healthcare Conference 2018
27 slides from Sarepta Therapeutics's conference presentation, Jan 8, 2018
Slide 1: Precision genetic medicine for rare neuromuscular diseases
Precision genetic medicine for rare neuromuscular diseases 36TH ANNUAL JP MORGAN HEALTHCARE CONFERENCE SAN FRANCISCO, CALIFORNIA JANUARY 8, 2018 NASDAQ: SRPT Jackson Exhibit 99.1
Slide 2: Forward-Looking Statements
2 FORWARD-LOOKING STATEMENTS This presentation contains "forward‐looking statements" within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Statements that are not historical facts or words such as "believes," "anticipates," "plans," "expects,"
Slide 3: Aspiration to be a global top-five rare-disease company
Sarepta Therapeutics aspires to be a global top-five rare-disease focused company, applying its expertise in precision genetic medicine to address a variety of neuromuscular conditions.
Slide 4: All the elements in place for success
All the elements in place for success Multi-platform pipeline Funded for the future Strong foundation Urgent mission
Slide 5: Driven by an urgent mission
Driven by an urgent mission Our goal is to develop life-changing precision genetic medicines to treat 100 percent of individuals with Duchenne muscular dystrophy (DMD) and apply our therapeutic approach to other rare neuromuscular diseases where it is likely to have the most benefit
Slide 6: 2017: Setting the stage for success
2017: Setting the stage for success LAUNCH PARTNERING R&D FINANCIAL EXECUTED SIGNED INITIATED ADDED TOP 5 4 3 >$1B most successful U.S. rare disease launches based on first-year revenue collaborations for next-generation precision genetic therapies clinical studies evaluating potential life-changing
Slide 7: Key milestones/inflection points 2018: A year of transformation
Key milestones/inflection points 2018: A year of transformation Q1 mid-2018 Q3 H2 FDA meeting on golodirsen CHMP decision for EXONDYS (eteplirsen) NCH micro-dystrophin gene therapy readout 48-week data from PhaseOUT DMD (Summit) H2 Safety signals and dosing for SRP-5051 H2 IND for next PPMO candidat
Slide 8: Our path to developing treatments for all eligible individuals with DMD
PROGRAM DISCOVERY PRE-CLINICAL CLINICAL TRIALS COMMERCIAL RNA-TARGETED THERAPIES EXONDYS 51* (eteplirsen) Golodirsen (SRP-4053) Casimersen (SRP-4045) EXON 52 (PMO) OTHER EXON TARGETS** (PMO) SRP-5051 (PPMO) SRP-5053 (PPMO) SRP-5045 (PPMO) SRP-5052 (PPMO) SRP-5044 (PPMO) SRP-5050 (PPMO) UTROPHIN MODU
Slide 9: Development Strategy: Building a Global Franchise in Neuromuscular Diseases
Development Strategy Building a Global Franchise in Neuromuscular diseases Key DMD Other indications DMD PMO DMD Other indications DMD Other indications Direct Platform partnering PPMO DMD Other indications Direct Platform partnering PPMO enhancements (e.g., new peptides, sequence optimization) DMD
Slide 10: What is DMD?
WHAT IS DMD?1-2 AFFECTS 1 IN 3,500-5,000 MALES BORN WORLDWIDE Rare, progressive neuromuscular genetic disease that is 100 percent fatal Average lifespan of mid- to late-20s; typical diagnosis occurs between ages 4-5 Caused by gene mutation that encodes dystrophin, a protein that exists in infinitesi
Slide 11: Experts in RNA-targeted therapies: core technology platform
EXPERTS IN RNA-targeted THERAPIES CORE TECHNOLOGY PLATFORM
Slide 12: Using precision RNA splicing to correct genetic defects
Using PRECISION RNA Splicing To CORRECT Genetic DEFECTS Our RNA platform results in genetic medicine precisely engineered to induce pre-mRNA splicing, which is designed to transform mRNA and produce a truncated but functional protein Sarepta Therapeutics is currently advancing two RNA-targeted techn
Slide 13: PMO programs: several potential near-term catalysts
Strong U.S. launch Managed Access Program opened or opening in 33 countries Marketing Authorization Application (MAA) currently under review in Europe Golodirsen (SRP-4053) Meeting with U.S. FDA in Q1’18 to determine regulatory pathway Casimersen (SRP-4045) Exon-skipping efficiency in pre-clinical m
Slide 14: PPMO: A transformative approach to treating DMD
PPMO: A Transformative Approach To Treating DMD SRP-5051: first PPMO candidate in the clinic Phase 1/2a clinical trial initiated in November 2017 Multi-center, double blind, placebo-controlled, multi-dose efficacy portion of study to initiate by mid-2018 or as soon as a therapeutic dose has been ide
Slide 15: Repeat PPMO doses increased and sustained dystrophin production in vivo
Repeat PPMO Doses Increased and Sustained Dystrophin production In Vivo Quadriceps Diaphragm Heart mdx (DMD) mice were treated with a three monthly IV doses of PPMO @ 40 mg/kg; the single dose PPMO cohort was included as a comparator
Slide 16: PPMO treatment improved muscle function in mdx mice
PPMO Treatment Improved Muscle Function in Mdx Mice mdx (DMD) mice at 7 weeks of age were treated with a single IV dose of saline or PPMO @ 10, 20 40 or 80 mg/kg and WT mice at 7 weeks of age were treated with a single IV dose of saline Mice were tested for grip strength at 8 weeks (1 week post-inje
Slide 17: Potential for up to seven approved therapies in the U.S. by 2022
Potential for up to seven approved therapies in the U.S. by 2022 RNA-Targeted therapy franchise development strategy* Pre-clinical Clinical Sequencing Initial development KEY *timeline not to scale EXONDYS 51 Golodirsen SRP-5051 Rare exon/ platform SRP- 4052 Confirmatory study 50 clinical OLE ESSENC
Slide 18: Next-generation approaches
Next-generation approaches ONGOING GENE THERAPY AND EDITING PROGRAMS
Slide 19: Potential to address all individuals with DMD
potential to address all individuals with DMD Leaders in gene therapy AND EDITING for dmd PARTNER ANNOUNCED MODALITY TYPE STATUS January 2017 Micro-Dystrophin Gene Therapy Research and option agreement June 2017 Micro-Dystrophin Gene Therapy Research and option agreement Manufacturing scale-up under
Slide 20: Micro-dystrophin gene therapy overview
Phase 1/2a clinical trial underway; Interim safety and efficacy Data expected in 2H’18 micro-dystrophin gene therapy overview Uses an adeno-associated virus vector to deliver a shortened version of the dystrophin gene to replace the missing protein in individuals with DMD N H1 R1 R2 R3 H2 R4 R5 R6 R
Slide 21: The promise of micro-dystrophin gene therapy in DMD
GENETHON Data published in Nature communications THE PROMISE OF micro-dystrophin gene therapy IN DMD Study conducted in 12 dogs naturally affected by DMD and treated with Genethon’s micro-dystrophin gene therapy At two-year follow-up, muscle function was significantly restored and clinical symptoms
Slide 22: GALGT2 gene therapy overview
TARGET Potential to restore muscle function despite the absence of the dystrophin protein GALGT2 gene therapy overview Targets the dystroglycan complex to enhance utrophin expression—a largely homologous protein to dystrophin—and preserve muscle function regardless of underlying genetic mutation Ada
Slide 23: Well-Positioned for future success
Well-Positioned for future success
Slide 24: Expansion into other therapeutic areas
Expansion into other therapeutic areas OTHER DISEASES Our Base case REPRESENTS a Significant opportunity GENE THERAPY Potential to address 100 percent of eligible individuals with DMD PPMO A potentially transformative approach to slowing disease progression in DMD Other PMO exon targets being develo
Slide 25: Exondys 51: Strong quarterly growth
Exondys 51: Strong Quarterly growth Key drivers of success: Major DMD centers continue to submit START forms Reimbursement wins in 2017 More eligible individuals getting on therapy Quarterly Revenue (in millions) Strong uptake through first five quarters post-launch *Unaudited
Slide 26: Exondys 51: Strong growth in 2018, FY'18 guidance
FY’18 Guidance $295-305M Exondys 51: STRONG GROWTH in 2018 Anticipate ~100 percent year-over-year revenue growth $154.6M FY’17 Revenue* *Unaudited
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