Sarepta Therapeutics, Inc. · NASDAQ:SRPT
Sarepta Therapeutics — JP Morgan 39th Annual Healthcare Conference 2021
27 slides from Sarepta Therapeutics's conference presentation, Jan 11, 2021
Slide 1: Our mission continues
Our mission continues. DOUGLAS INGRAM President and Chief Executive Officer JP Morgan 39th Annual Healthcare Conference 2021 January 11, 2021 Armed with the most advanced science in genetic medicine, we are in a daily race to rescue lives otherwise stolen by rare disease. At Sarepta, every day is an
Slide 2: 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 3: Micro-dystrophin SRP-9001-102 Top-line Clinical Data (Part 1)
Micro-dystrophin SRP-9001-102 Top-line Clinical Data (Part 1)
Slide 4: SRP-9001-102 Study Design: Parts 1 and 2
SRP-9001-102 Study Design: Parts 1 and 2 A randomized, double-blind, placebo-controlled clinical trial to evaluate the safety, efficacy and tolerability of a single dose of SRP-9001 compared to placebo, in boys with DMD aged 4–7 years old; Study is ongoing and remains blinded, functional results for
Slide 5: Micro-dystrophin Protein Expression and Vector Genome Copies per Nucleus Achieved Endpoints in Part 1
Micro-dystrophin Protein Expression and Vector Genome Copies per Nucleus Achieved Endpoints in Part 1 of Study 102 (n=20, Week 12) Micro-dystrophin Expression (IF) Vector Genome Copy Number Micro-dystrophin Expression (Western Blot)
Slide 6: Study 102 Placebo Crossover Patients Showed Similar Expression Results to Study 101
Study 102 Placebo Crossover Patients Showed Similar Expression Results to Patients in Study 101 Micro-dystrophin Expression (Western Blot) * Does not include 4th patient deemed to be an outlier Micro-dystrophin Protein Expression Results: Study 102 Placebo Crossover Patients (n=11) Vector Genome Cop
Slide 7: NSAA Primary Functional Endpoint: Treated Patients Outperformed Placebo at All Time Points
NSAA Primary Functional Endpoint: Treated Patients Outperformed Placebo Patients at All Time Points NSAA change from baseline of +1.7 in SRP-9001 treated group vs. +0.9 in placebo group, which is not statistically different (p= 0.37) Separation shown at every timepoint between SRP-9001 and placebo g
Slide 8: Functional Measures Well Matched at Baseline (4-5 Year Old Group)
Functional Measures Well Matched at Baseline (4-5 Year Old Group)
Slide 9: Pre-specified NSAA Subgroup Analysis (Ages 4-5): Reached Statistical Significance
Pre-specified NSAA Subgroup Analysis (Ages 4-5): Reached Statistical Significance The 4- to 5-year old group had a statistically significant improvement in NSAA vs. placebo group at week 48 NSAA change from baseline of +4.3 in SRP-9001 treated 4–5-year-olds vs. 1.9 in placebo (p= 0.0172); age was a
Slide 10: Functional Measures Not Well Matched at Baseline (6-7 Year Old Group)
Functional Measures Not Well Matched at Baseline (6-7 Year Old Group) Patients in the treated group had significantly lower NSAA scores at baseline Note that an imbalance in NSAA and timed tests exist in the older (6–7-year-olds) between the two groups with the treated group worse than placebo.
Slide 11: Baseline NSAA Differences Have a Significant Impact on Disease Progression
Mean Baseline NSAA Score for 9001 Treated Patients (6-7 year old group) Mean Baseline NSAA Score for Placebo Patients (6-7 year old group) Age and baseline NSAA are predictors of disease progression Source: Muntoni F, Domingos J, Manzur AY, et al. Categorising trajectories and individual item change
Slide 12: Safety Summary
Safety Summary No new safety signals Safe and well tolerated; consistent with previous studies 85% of the treated group had treatment related adverse events vs. 43% in the placebo group The most common treatment related adverse event was vomiting 60% (12/20) in treatment group vs. 19% (4/21) in plac
Slide 13: Conclusions
Conclusions No new safety signals observed Primary biological endpoint (micro-dystrophin expression at 12 weeks post-treatment) achieved Total NSAA score of treated patients vs. placebo demonstrated a positive increase at all post-treatment time points The study did not achieve a statistical signifi
Slide 14: Next Steps
Next Steps Continue to advance Part 2 crossover phase; conduct biopsy at week 12 to assess expression and biological markers and longer-term assessments of functional outcomes Enrolled and dosed 11 patients in Study 103 using commercial process material Report biomarker and safety results in Q2 2021
Slide 15: Total Estimated Prevalence
1 000. é3 -" 000 Q. TOTAL ESTIMATED PREVALENCE
Slide 16: Estimated global prevalence
globally * Estimated global prevalence
Slide 17: RNA Franchise: PMO and PPMO for Duchenne
RNA Franchise PMO and PPMO for Duchenne
Slide 18: PMO Franchise Revenue and Guidance
PMO Franchise Revenue and Guidance $ $ Millions $301M $381M $456M $537M- 547M Guidance (Unaudited) Q4 revenue $122.6M (Unaudited) $1.9B in cash on 12/31/20 PDUFA DATE: FEBRUARY 2021
Slide 19: Sarepta's 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) Sarepta’s PPMO Next-generation Technology Peptide phosphorodiamidate morpholino oligomer (PPMO) technology
Slide 20: Gene Therapy: LGMD Portfolio
— Gene Therapy } LGMD Portfolio ©SAREPTA THERAPEUTICS, NC 2021 AL RIGHTS RESERVED,
Slide 21: Sarepta's Gene Therapy Pipeline
Sarepta’s Gene Therapy Pipeline SRP-9001 micro-dystrophin GALGT2 Nationwide Children’s Micro-dystrophin Genethon SRP-9003 (LGMD2E β-sarcoglycan) SRP-9004 (LGMD2D α-sarcoglycan) SRP-9005 (LGMD2C γ-sarcoglycan) SRP-6004 (LGMD2B Dysferlin) SRP-9006 (LGMD2L Anoctamin-5) Calpain 3 (LGMD2A) Nationwide Chi
Slide 22: Clinical Results to Date: SRP-9003-101, Cohort 1
Clinical Results to Date: SRP-9003-101 (N=3)*, Cohort 1 (0.5 x 1014 vg/kg)2,3 Mean of >104 vector copies/µg DNA detected 0.6 vector copies/nucleus 36.1% mean β-sarcoglycan protein expression vs normal by western blot Is the transgene DNA inside muscle cells? Is the protein at the cell membrane? How
Slide 23: Clinical Results to Date: SRP-9003-101, Cohort 2
Clinical Results to Date: SRP-9003-101 (N=3)*, Cohort 2 (2.0 x 1014 vg/kg)2,3 Is the transgene DNA inside muscle cells? Is the protein at the cell membrane? How much is there? Is muscle function improved? What was the safety and tolerability experience with SRP-9003? Is the desired protein made? Que
Slide 24: Next Indications: LGMD Types 2C and 2D
Next Indications: LGMD Types 2C and 2D OPPORTUNITY TO GENERATE A STEADY STREAM OF GENE Therapy Candidates in five additional subtypes which together represent more than 70% of all Known LGMDs1 LGMD2C LGMD2B LGMD2D LGMD2A* LGMD2L *This program is in collaboration with Dr. Zarife Sahenk at Nationwide
Slide 25: Sarepta's Pipeline
Sarepta’s Pipeline 27 PROGRAMS 14 PROGRAMS GENE EDITING GENE THERAPY RNA 2 PROGRAMS Potential to treat 1.5M patients* More than 40 programs in all *Based on published epidemiology.
Slide 26: Multi-Platform Genetic Medicine Engine – Upcoming Milestones
Sarepta’s Multi-Platform Genetic Medicine Engine – Upcoming Milestones Gene Therapy Duchenne (SRP-9001) Continue to advance Part 2 crossover phase; conduct biopsy at week 12 to assess expression and biological markers and longer-term assessments of functional outcomes Enrolled and dosed 11 patients
Slide 27: #DraggingTomorrowIntoToday
#DraggingTomorrowIntoToday SAREPTA, SAREPTA THERAPEUTICS, the SAREPTA Helix Logo, VYONDYS, EXONDYS, EXONDYS 51, and the EXONDYS 51 Logo are trademarks of Sarepta Therapeutics, Inc. registered in the U.S. Patent and Trademark Office and may be registered in various other jurisdictions. AMONDYS, AMOND
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