Sarepta Therapeutics, Inc. · NASDAQ:SRPT
Sarepta Therapeutics — J.P. Morgan 40th Annual Healthcare Conference (January 2022)
24 slides from Sarepta Therapeutics's conference presentation, Jan 10, 2022
Slide 1: J.P. Morgan 40th Annual Healthcare Conference
DOUG INGRAM President and CEO J.P. Morgan 40th Annual Healthcare Conference January 10, 2022 Exhibit 99.2
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: Duchenne Muscular Dystrophy (DMD)
Duchenne Muscular Dystrophy (DMD) DMD affects approximately 1 in 3,500-5,000 males worldwide1 DMD is a rare, fatal neuromuscular genetic disease inherited in an X-linked recessive pattern2 Muscle weakness becomes increasingly noticeable by 3 to 5 years of age, and most patients use a wheelchair by t
Slide 4: SRP-9001 Gene Therapy Construct
SRP-9001 Gene Therapy Construct 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 for muscle force3 MICRO-DYSTROPHIN Optimized for desired
Slide 5: SRP-9001-102: Study Design
SRP-9001-102: Study Design Randomization (n=41) Single IV infusion placebo (n=21) Single IV infusion placebo Single IV infusion SRP-9001 Open-label extension period Single IV infusion SRP-9001 (n=20) Part 1: 48 weeks Part 2: 48 weeks Double-blind placebo-controlled Part 3 Randomized, double-blind, p
Slide 6: External Control (EC) Comparison
External Control (EC) Comparison Purpose of Propensity Score Weighting Used with the external comparison group which allowed balancing of relevant covariates such as age, baseline function (NSAA, RFF and 10MWR) Use of a multi-variate approach is more robust than just using age Study SRP-9001-102 Par
Slide 7: SRP-9001-102: Propensity Score Weighting Balanced the Covariates Well
SRP-9001-102: Propensity Score Weighting Balanced the Covariates Well Before Propensity Weighting After Propensity Weighting Parameter SRP-9001 CINRG Pre-Pub Steroid Study Lilly Total Parameter SRP-9001 External Control (N=21) (N=14) (N=83) (N=29) (N=126) (N=20) (N=103) Age Age Mean (SD) 7.23 (1.10)
Slide 8: A One-point Change on the NSAA Shows a Distinct and Observable Difference
A One-point Change on the NSAA Shows a Distinct and Observable Difference Video provided by presenter. Consent given for use. NSAA, NorthStar Ambulatory Assessment. Stand from supine NSAA: 2:1 change Stand from supine NSAA: 0
Slide 9: SRP-9001-102: Significant 2-point Improvement on NSAA in Part 2
SRP-9001-102: Significant 2-point Improvement on NSAA in Patients Receiving SRP-9001 in Part 2 Compared to External Control Group at Week 48 NSAA Total Score p value = 0.0009 2-point Improvement Study week for Part 2 of Study 102 Mean age of patients at time of baseline NSAA, 7.24 years of age Chang
Slide 10: SRP-9001-102: 96 Week Data in 4 to 5-year-old Cohort
Mean NSAA score across all ages (4 to 7-year-olds) treated in Part 1 remained stable out to week 96 SRP-9001-102: 96 Week Data in 4 to 5-year-old Cohort Positive 5-point NSAA improvement from baseline sustained in SRP-9001 treated 4 to 5-year-olds at week 96 Study week Patients increased 5 points fr
Slide 11: SRP-9001-102: Safety Summary
SRP-9001-102: Safety Summary The safety profile of patients treated in Part 2 is consistent with that seen in Part 1 There were no treatment-related serious adverse events, no deaths and no patient study discontinuations due to an adverse event The most common related adverse event in patients treat
Slide 12: Treatment-related Treatment-emergent Adverse Events in Part 2
Treatment-related Treatment-emergent Adverse Events* in Part 2 Safety Population – Part 2 Data Preferred Term SRP-9001 Placebo-crossover patients SRP-9001 treated in Part 1 Total (N=21), n(%) (N=20), n(%) (N=41), n(%) Subjects with any Treatment-Related TEAE 20 ( 95.2) 4 ( 20.0) 24 ( 58.5) Vomiting
Slide 13: SRP-9001-102: Conclusions
SRP-9001-102: Conclusions Patients treated in Part 2 Study 102 improved 2 points vs. predefined external control group Results were highly statistically significant Results are consistent with observed treatment effect and increase our growing body of clinical evidence Observed durable response out
Slide 14: Study 103: 8.6-point NSAA improvement at 3 years
STUDY 101 4 patients 4 to 7-year-olds 8.6-point improvement on NSAA compared to matched natural history group at 3 years; p value (<0.0001)
Slide 15: Study 102 Part 1: 4 to 5-year-olds NSAA improvement
STUDY 101 4 patients 4 to 7-year-olds 8.6-point improvement on NSAA compared to matched natural history group at 3 years; p value (<0.0001) STUDY 102 – Part 1 16 patients 4 to 5-year-olds 2.5-point improvement on NSAA compared to placebo at 1 year; p value (0.0172)
Slide 16: Study 102 Part 1: 6 to 7-year-olds NSAA improvement
STUDY 101 4 patients 4 to 7-year-olds 8.6-point improvement on NSAA compared to matched natural history group at 3 years; p value (<0.0001) STUDY 102 – Part 1 16 patients 4 to 5-year-olds 2.5-point improvement on NSAA compared to placebo at 1 year; p value (0.0172) 1 1SRP-9001 treated 6-7 YO (N=12)
Slide 17: Study 102 Part 2: NSAA improvement
STUDY 101 4 patients 4 to 7-year-olds 8.6-point improvement on NSAA compared to matched natural history group at 3 years; p value (<0.0001) STUDY 102 – Part 1 16 patients 4 to 5-year-olds 2.5-point improvement on NSAA compared to placebo at 1 year; p value (0.0172) STUDY 102 – Part 2 21 patients 5 t
Slide 18: Study 103 Cohort 1: NSAA improvement
STUDY 101 4 patients 4 to 7-year-olds 8.6-point improvement on NSAA compared to matched natural history group at 3 years; p value (<0.0001) STUDY 102 – Part 1 12 patients 6 to 7-year-olds 2.9-point improvement on NSAA compared to matched natural history group at 1 year; p value (0.0129) STUDY 102 –
Slide 19: EMBARK: Only global gene therapy clinical trial underway
STUDY 301 Only global gene therapy clinical trial underway Poised for success based on the clinical evidence generated to date On track to complete enrollment by mid-2022
Slide 20: Commercialized Therapeutics and Pipeline Programs
Commercialized Therapeutics and Pipeline Programs
Slide 21: Robust and Expanding RNA Franchise in Duchenne
Robust and Expanding RNA Franchise in Duchenne $ $ Millions 2019 2020 2021 2022 $381M $456M $612M >$800M (Guidance) (Unaudited) Q4 revenue $178.7M (Unaudited) $2.1B in cash on 12/31/21 21 consecutive quarters of revenue growth; CAGR of 40% FY2017-FY2021; ~30% revenue growth anticipated (2022 vs 2021
Slide 22: Generating a Steady Stream of Personalized Medicine Therapeutics
Generating a Steady Stream of Personalized Medicine Therapeutics Gene Therapy DISCOVERY PRECLINICAL CLINICAL COMMERCIAL RNA Duchenne Muscular Dystrophy (PMO) Gene Editing Duchenne Muscular Dystrophy Limb-girdle Muscular Dystrophy Charcot-Marie-Tooth Mucopolysaccharidosis IIIA Cardiomyopathy Central
Slide 23: 2022 Milestones
2022 Milestones Gene Therapy Duchenne: SRP-9001-301 EMBARK fully enrolled by mid-2022 SRP-9001 additional data at medical conferences Limb-girdle muscular dystrophy: SRP-9003-101 (3-year data for low dose cohort and 2-year data for high dose cohort) Finalize strategy for sarcoglycans (SRP-9003, SRP-
Slide 24: Dragging Tomorrow Into Today
#DraggingTomorrowIntoToday Dragging tomorrow into today SAREPTA, SAREPTA THERAPEUTICS, the SAREPTA Helix Logo, AMONDYS, AMONDYS 45, the AMONDYS 45 Logo, VYONDYS, VYONDYS 53, the VYONDYS 53 Logo, EXONDYS, EXONDYS 51, and the EXONDYS 51 Logo are trademarks of Sarepta Therapeutics, Inc. registered in t
Sarepta Therapeutics — J.P. Morgan 40th Annual Healthcare Conference (January 2022) on 6ix