Sarepta Therapeutics, Inc. · NASDAQ:SRPT
Sarepta Therapeutics — JPMorgan Healthcare Conference 2023
20 slides from Sarepta Therapeutics's conference presentation, Jan 9, 2023
Slide 1: Sarepta Therapeutics JPMorgan Healthcare Conference
Sarepta Therapeutics, Inc. (NASDAQ:SRPT) JPMorgan Healthcare Conference San Francisco, California January 9, 2023 Doug Ingram President and CEO 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: Living with Duchenne muscular dystrophy: A Bellwether Moment
BENJAMIN Living with Duchenne muscular dystrophy A Bellwether Moment…
Slide 4: SRP-9001: Poised to transform the trajectory of Duchenne muscular dystrophy
SRP-9001 Poised to transform the trajectory of Duchenne muscular dystrophy THE OPPORTUNITY
Slide 5: Duchenne is progressive and causes irreversible muscle damage and loss of function
1. Birnkrant et al. Lancet Neurol. 2018 Mar;17(3):251-267; 2. Verma et al. Clin Pediatr (Phila). 2010 Nov;49(11):1011-7; 3. Aartsma-Rus et al. Hum Mutat. 2009 Mar;30(3):293-9; 4. Duchenne UK, Stages of Duchenne https://www.duchenneuk.org/Pages/FAQs/Category/stages-of-duchenne (last accessed August 2
Slide 6: Restoring the dystrophin-associated protein complex (DAPC) restores function
Restoring the dystrophin-associated protein complex (DAPC) restores function
Slide 7: Becker muscular dystrophy shortened dystrophin retains critical elements of dystrophin
Becker muscular dystrophy (BMD) shortened dystrophin protein retains critical elements of dystrophin3-7 NORMAL MUSCLE3-7 61 YO AMBULATORY PATIENT8-9 1. Naso MF, et al. BioDrugs. 2017;31(4):317-334. 2. Chamberlain K, et al. Hum Gene Ther Methods. 2016;27(1):1-12. 3. Gao Q, et al. Compr Physiol. 2015;
Slide 8: BMD shortened dystrophin protein retains critical elements of dystrophin
BMD shortened dystrophin protein retains critical elements of dystrophin3-7 SAREPTA SRP-9001 DYSTROPHIN3-7 61 YO AMBULATORY PATIENT8-9 1. Naso MF, et al. BioDrugs. 2017;31(4):317-334. 2. Chamberlain K, et al. Hum Gene Ther Methods. 2016;27(1):1-12. 3. Gao Q, et al. Compr Physiol. 2015;5(3):1223. 4.
Slide 9: SRP-9001 rationally designed to maximize expression in tissues most affected by Duchenne
SRP-9001 has been rationally designed to maximize expression in tissues most affected by Duchenne1-6 AAVrh74 Viral Vector1-2 MHCK7 Promoter3 SRP-9001 dystrophin Transgene4-6 Affinity for muscle Specific to skeletal and cardiac muscle Assembles DAPC Relatively low level of preexisting immunity and fa
Slide 10: Non-clinical proof-of-concept: dystrophin expression and strength output
Non-clinical proof-of-concept demonstrating a correlation between SRP-9001 dystrophin expression and strength output Skeletal Muscle Diaphragm Heart WT mdx 4.43 x 1013vg/kg 7.00 x 1014vg/kg 1.33 x 1014vg/kg 4.01 x 1014vg/kg Specific Force in TA Wt mdx-LR 1.33e14 4.01e14 vg/kg vg/kg P<0.01 P<0.01
Slide 11: SRP-9001 restores DAPC, reduces CK, improves histopathology
SRP-9001 treatment leads to restoration of DAPC, reduced CK, and improved histopathology Wildtype DMDMDX untreated mice 1.33x1014 vg/kg SRP-9001 SGCB Merged Serum Creatine Kinase Wt mdx 1.33e14 untreated vg/kg P<0.05
Slide 12: SRP-9001 dystrophin expression, transduction, and localization at target dose
SRP-9001 dystrophin expression, transduction, and localization at the dose of 1.33 x 1014 vg/kg Measure Timepoint Study 101 (Early Development Process) (n=4) Study 102 Part 1 & 2 Target Dose a (Early Development Process) (n=29) Study 103 (Intended Commercial Process) (n=20) Mean age (years) at time
Slide 13: Improved benefit over time
Improved benefit over time1 1 year 1 year 1 year 2 year 4 year STUDY 102 Part 2 Integrated Efficacy Analysis at Target Dose STUDY 103 STUDY 102 Part 1 STUDY 101 20 patients 52 patients 20 patients 20 patients 4 patients Strong Expression Reduction in CK Positive Biomarkers 2.4 point3 3.2 point3 Vast
Slide 14: SRP-9001 pathway
SEPT OCT NOV DEC JAN FEB MAR APR MAY JUN SRP-9001 pathway BLA Filed BLA Accepted Priority Review Granted PDUFA Date (May 29, 2023) First Patient Treated SUMMER 2023 2022
Slide 15: Internal gene therapy capabilities complemented by partnerships
Internal gene therapy capabilities complimented by partnerships: Meeting demand to launch SRP-9001 Dedicated Sarepta Facilities and Capabilities Analytical, Process Development & Quality Control Vector & drug product development Non-clinical tox manufacturing Fully equipped AD/QC labs Validated meth
Slide 16: A holistic approach to assessing innovative treatments
A holistic approach to assessing innovative treatments Quality adjusted life-years gained Net costs Adherence- improving factors Reduction in uncertainty Fear of contagion Insurance value Severity of disease Value of hope Real- option value VALUE Productivity Equity Scientific spillover Lakdawalla,
Slide 17: Robust total product revenue for RNA-based PMO franchise in Duchenne
Robust total product revenue for RNA-based PMO franchise in Duchenne $ $ Millions 2019 2020 2021 2023 2022 $381M $456M $612M >$925M (Guidance) ~$2.0B in cash on 12/31/22 (Unaudited) Revenues represent net product revenues from PMO franchise. $843.3M (Unaudited) Q4 revenue $235.5M (Unaudited)
Slide 18: Growth strategy: leveraging current and future opportunities
Growth strategy: Leveraging current and future opportunities as we secure our leadership position in genetic medicine Proprietary technology platforms 3 Deep, advancing pipeline in neuromuscular, cardiac, and neuro to drive future growth 40+ programs 3 late-stage programs in Duchenne and limb-girdle
Slide 19: But most importantly, we are enabling a future
But most importantly, we are enabling a future. ©Sarepta therapeutics, inc. 2023. All rights reserved.
Slide 20: Sarepta Therapeutics JPMorgan Healthcare Conference
Sarepta Therapeutics, Inc. (NASDAQ:SRPT) JPMorgan Healthcare Conference San Francisco, California January 9, 2023 Doug Ingram President and CEO
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