Sarepta Therapeutics, Inc. · NASDAQ:SRPT
Sarepta Therapeutics — 37th Annual J.P. Morgan Healthcare Conference (January 2019)
26 slides from Sarepta Therapeutics's conference presentation, Jan 7, 2019
Slide 1: 37th Annual J.P. Morgan Healthcare Conference
37th Annual J.P. Morgan Healthcare Conference, San Francisco, CA, January 7, 2019 Doug Ingram President and CEO, Sarepta Therapeutics, Inc. Exhibit 99.1
Slide 2: Forward Looking Statements
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: Our Goals for 2018
OUR GOALS FOR 2018 Build out our pipeline Ensure we have the resources to build for the future Clarify a pathway for our RNA technology Become one of the most important rare disease companies Have another strong year of commercial execution
Slide 4: Expansion of our RNA and gene therapy engine programs
* ** EXPANSION OF OUR RNA AND GENE THERAPY ENGINE 15 PROGRAMS 25 PROGRAMS
Slide 5: Resources and talent
Resources and talent* TALENT 2x PhDs 3x MDs Leading gene therapy team 500+ PROFESSIONALS >$1.1B CASH POSITION *As of December 31, 2018; preliminary and unaudited results
Slide 6: Defined RNA pathway: golodirsen and casimersen
Casimersen 2020 Golodirsen 2019 If successful, we will have 3 RNA-therapies by 2020, serving ~30% of the DMD community. 2016 DEFINED RNA PATHWAY
Slide 7: Execution
execution FY’17 $154.6M 2016 LAUNCH 2018 GROWTH Q4’18* $84.4M FY’18* $301M *Preliminary and unaudited results
Slide 8: Top rare disease companies by market cap
TOP 5 RARE DISEASE COMPANIES* *By Market Cap
Slide 9: We are Sarepta. We will change healthcare
We are Sarepta We will change healthcare
Slide 10: Micro-dystrophin program overview
AAVrh74 AAVrh74 provides broad distribution to all muscle types, including the heart and diaphragm SR2 and SR3 critical for force production MHCK7 promoter enables robust dystrophin expression in heart and skeletal muscle ITR, inverted tandem repeat; SR, spectral-like repeat. MHCK7 PROMOTER INTRON M
Slide 11: Early stage results of micro-dystrophin
Robust expression of micro-dystrophin in muscle fibers Reduction of creatine kinase Improvements across all measured functions*** Well-tolerated * Data from the 4 patients dosed in Study NCT03375164 **NCH Western blot method ***North Star Ambulatory Assessment (NSAA), Time to Rise, 4 Stairs Up, and
Slide 12: The path forward
Current Trial 24 Patients Placebo controlled Met with FDA in Q4 2018 The path forward Confirmatory Trial With Commercial Material Goal to Commence in 2019 Study Goal Functional Benefit of Expression 2 Patients Started Dosing
Slide 13: And our intent is to do this over and over again
And our intent is to do this over and over again
Slide 14: 7,000 rare diseases
7,000 Rare Diseases ~80% are single gene mutations
Slide 15: 7,000 rare diseases impacted globally
7,000 Rare Diseases Impacted Globally 400M Patients And Only 1 FDA Approved GENE THERAPY
Slide 16: Traditional drug development model
10,000s of compounds 250 drug candidates 5 INDs submitted 1 maybe mmm 15 years
Slide 17: Gene therapy can upend this model
Gene therapy can upend this model
Slide 18: Massive opportunity to do good: upending the model
MASSIVE OPPORTUNITY TO DO GOOD AND WE WILL PLAY A ROLE IN upending the model PROBABILITY OF SUCCESS TIMELINES
Slide 19: We engineer solutions
We don’t rely on serendipity We Engineer Solutions PROBABILITY OF SUCCESS
Slide 20: Our manufacturing strategy
Our manufacturing STRATEGY Paragon Brammer Aldevron In-House Capabilities The largest gene therapy capacity around the globe
Slide 21: Disease areas: DMD, LGMD, CMT, MPS, Pompe
Duchenne Muscular Dystrophy Limb-girdle Muscular Dystrophy Charcot-Marie-Tooth Disease Mucopolysaccharidosis Pompe Disease
Slide 22: Programs: most robust precision genetic medicine pipeline
25 Programs The most robust precision genetic medicine pipeline today 40 Programs Goal >1.5M patients with rare disease globally Epidemiology* MASSIVE OPPORTUNITY TO DO GOOD *Based on published epidemiology
Slide 23: We are upending the model
We are upending the model
Slide 24: Because someday is too late
Because someday is too late
Slide 25: Pulling tomorrow into today
Pulling tomorrow into today
Slide 26: Slide 26
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