Sarepta Therapeutics, Inc. · NASDAQ:SRPT
Sarepta Therapeutics — Corporate Update (January 2017)
33 slides from Sarepta Therapeutics's corporate update, Jan 10, 2017
Slide 1: RNA-targeted Precision Medicine for Duchenne Muscular Dystrophy
RNA-targeted Precision Medicine for Duchenne Muscular Dystrophy JANUARY 10, 2017 NASDAQ: SRPT 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: What is Duchenne muscular dystrophy?
Affects 1 in 3,500 – 5,000 males born worldwide What is Duchenne muscular dystrophy? Rare progressive neuromuscular genetic disease; fatal with average lifespan of mid to late 20s Diagnosis occurs between ages 4-5 Caused by gene mutation that encodes for dystrophin – a protein that plays a key struc
Slide 4: DMD represents a significant opportunity in key markets around the world
DMD Represents a significant opportunity in key markets around the world 47% of DMD [>12,000 patients] 8 exons Exon 44 Exon 52 Exon 43 Exon 8 Exon 55 Exon 45 Exon 53 Exon 51 29% of DMD [>7,600 patients] 3 exons Exon 45 Exon 53 Exon 51 13% of DMD [>3,400 patients] 1 exon Exon 51 8 exons ≈ half of all
Slide 5: Building value for shareholders in 2017
building value for shareholders in 2017 Exon 45&53 PPMO 4 External Collaborations 3. Accelerate Multiple Treatment Approaches Pursue and secure EMA approval and establish EU footprint Prepare for early access in other jurisdictions 2. Global Expansion EXONDYS 51 launch in US Increase awareness and i
Slide 6: EXONDYS 51 Commercial Update
EXONDYS 51™ Commercial Update
Slide 7: Driving a successful EXONDYS 51 launch: key strategic areas of focus
FIRST Driving prescriptions for all identified/ appropriate Exon 51 skip amenable patients FOURTH Ensuring all DMD patients have genetic tests and are appropriately identified for exon amenability Four Key activities DRIVING A SUCCESSFUL EXONDYS 51 LAUNCH Key Strategic Areas of Focus THIRD Addressin
Slide 8: Commercial execution: U.S. market launch
COMMERCIAL EXECUTION: U.S. MARKET LAUNCH Q4 Net Revenue (unaudited): ~$5.4 Million Genetically Verified Start Forms: Over 250 Since Launch Patient Demographics Current Start Forms: ~60% commercial / 40% Medicaid Age of Patients on Therapy: 9 months - 27 years Average Age of Patients on Therapy: 13 y
Slide 9: Major DMD centers are actively submitting start forms
Exon 51 Patients by DMD Center 95% Top Tier Centers have submitted a START Form 92% Tier 2 Centers have submitted a START Form 60% Tier 3 Centers have submitted a START Form DMD Centers TIER 1 & 2 REPRESENT 80% OF ALL DMD EXON 51 PATIENTS MAJOR DMD CENTERS are actively submitting start forms
Slide 10: Access discussions progressing well
9 National Health Plans Covering >156 Million Lives 22 Regional Accounts Covering 60 Million Lives 5 of the Top Commercial PBMs 3 of the Top Medicaid PBMs 13 of the Top Medicaid States Covering 27 Million Lives Payers actively engaged in learning more about EXONDYS 51 (i.e. >25 AMCP Dossier requests
Slide 11: Majority of plans covering or evaluating coverage case-by-case
Majority of Plans covering or evaluating coverage case-by-case 13% of Covered Lives have a favorable policy and/or covering to USPI 79% of Covered Lives are pending policy decisions, reviewing case-by-case or approved with restrictions 8% of Covered Lives are denying coverage (conversations continue
Slide 12: Patient journey to first infusion
Patient Journey to first infusion Find out about EXONDYS 51 Identify “patient home” site of care and 1:1 KOL to local MD discussion Surgical placement of port Discuss with DMD Expert Schedule appointment for port First EXONDYS 51 infusion Timeline Varies Center-to-Center and Patient-by-Patient Initi
Slide 13: Driving patient identification through genetic testing and interpretation
GENETIC TESTING PPMD partnership (Decode Duchenne support for testing) HCP “Importance of Genetic Testing” Brochure (Field driven) Duchenne.com patient education “knowing your DMD mutation” INTERPRETATION PPMD partnership (Decode Duchenne genetic counselors) MDA supported DMD Chart Review Program Du
Slide 14: Treating more boys with DMD
Treating More boys with dmd
Slide 15: Europe is a large opportunity
E.U. market research and KOL mapping completed Strategic approach to E.U. congress management Larger exon 51 opportunity (patient numbers) than the U.S. EUROPE is a large opportunity IP legal initiatives progressing
Slide 16: European submission (MAA)
EUROPEAN SUBMISSION (MAA) Proposed Indication EXONDYS is indicated for treatment of Duchenne muscular dystrophy (DMD) in adults, adolescents and children aged 4 years and older who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping Clinical Data Eteplirsen vs Untreated Ex
Slide 17: Preparing to rapidly commercialize in EU if approved
Preparing to rapidly commercialize in EU if approved Actively Engaged with EU KOLs Ongoing advisory from top EU DMD experts and significant presence at major conferences World Muscle Society Skip NMD Consortium ICNMD: Symposium Action Duchenne EMA DMD Guidelines CNS Active Clinical Trial Programs in
Slide 18: ESSENCE overview
Overview ~ 99 males to enroll (age 7-13) 96 week, double-blind, placebo controlled Roll over to open-label for 96 weeks Randomized 2:1 (66 active treatment:33 placebo) Conducted at sites in US, Europe, Canada a Global phase three placebo controlled trial for exons 45 &53 currently enrolling Essence
Slide 19: Robust clinical development program
4045-301 (ESSENCE) Exon 45 or Exon 53 96 weeks (plus 96 week extension) ~99 Enrolling 4658-301 (PROMOVI) Exon 51 48 weeks (plus 48 week extension) ~160 ~80 treated ~ 80 untreated Enrolling (Treated & Untreated) 4658-203 Exon 51 96 weeks ~40 ~20 treated ~20 untreated Enrolling (Untreated) ROBUST Clin
Slide 20: Staging a multi-front battle against Duchenne
Staging a multi-front battle against Duchenne
Slide 21: Muscle fibrosis (Catabasis) partnership
Muscle Fibrosis (Catabasis) Exploring benefits of co-administration of exon skipping and NF-KB inhibition Exon Skipping disease modifying backbone to address to underlying cause of DMD Executed four external partnerships expanding our portfolio of DMD treatments Staging a multi-front battle against
Slide 22: Utrophin modulation (Summit)
Exon Skipping disease modifying backbone to address to underlying cause of DMD Executed four external partnerships expanding our portfolio of DMD treatments Staging a multi-front battle against Duchenne Utrophin Modulation (Summit) Potential to be disease-modifying therapy for all underlying mutatio
Slide 23: Gene therapy (Nationwide Children's Hospital)
Exon Skipping disease modifying backbone to address to underlying cause of DMD Executed four external partnerships expanding our portfolio of DMD treatments Staging a multi-front battle against Duchenne Gene Therapy (Nationwide Children’s Hospital) Partnering to advance MicroDystrophin gene therapy
Slide 24: Multi-front battle: external partnerships
Muscle Fibrosis (Catabasis) Exploring benefits of co-administration of exon skipping and NF-KB inhibition Exon Skipping disease modifying backbone to address to underlying cause of DMD Executed four external partnerships expanding our portfolio of DMD treatments Staging a multi-front battle against
Slide 25: Next Generation Pre-clinical data
Next Generation Pre-clinical data
Slide 26: PPMO: next generation antisense platform
PPMO: NEXT GENERATION ANTISENSE PLATFORM TO TARGET MUSCLE AND OTHER ORGANS Proprietary class of PPMO compounds have the potential to provide: Improved delivery in vivo Superior dystrophin production in vivo Tolerability in non-human primates Less frequent dosing Extended patent term A platform techn
Slide 27: Response durability supports less frequent dosing
7 30 60 90 7 30 60 90 PMO PPMO Days Post Single Dose Injection Quadriceps mdx (DMD) mice were treated with a single IV dose of PMO or PPMO @ 40 mg/kg The clinical Western blot method for dystrophin was performed on muscle at 7-90 days post single dose injection Standard Curve (% WT) Standard Curve (
Slide 28: Cell penetrating peptide increases potency vs. PMO
mdx (DMD) mice were treated with a single IV dose of PMO or PPMO (with the clinical cell penetrating peptide) The clinical Western blot method for dystrophin was performed on muscle at 30 days post single dose injection 40 80 120 Dose (mg/kg) 40 80 120 PMO PPMO 40 80 120 Dose (mg/kg) 40 80 120 PMO P
Slide 29: PPMO clinical candidate achieves global delivery in non-human primate
>90% EXON 51 SKIPPING PPMO CLINICAL CANDIDATE ACHIEVES GLOBAL DELIVERY IN NON-HUMAN PRIMATE (NHP) Exon skipping observed in all relevant muscle groups: skeletal, cardiac and smooth muscle NHPs tolerated 4-weekly doses of 20, 40 and 80 mg/kg 20 40 80 20 40 80 20 40 80 20 40 80 Dose (mg/kg) Quadriceps
Slide 30: Accelerating development of PPMO program towards clinical trials
PPMO demonstrates enhanced efficacy vs. PMO Significantly higher dystrophin production Durability of response should support less frequent dosing IND-enabling GLP toxicology studies to begin early this year; target opening IND before year-end 2017 Initial toxicology in mouse and non-human primate in
Slide 31: Comprehensive approach to treating DMD
Up to 7 programs in clinic in 2017 Comprehensive approach to treating DMD Internal External Collaborations Program Discovery Preclinical Phase 1 Phase 2 Phase 3 DMD EXON 53 (SRP-4053) DMD EXON 45 (SRP-4045) PPMO DMD EXON 52 (SRP-4052) UTROPHIN (SUMMIT) MICRODYSTROPHIN GENE THERAPY (NATIONWIDE CHILDR
Slide 32: Sarepta well positioned for success in 2017
Sarepta well positioned for success in 2017 Rapidly advancing pipeline: Exons 45/53 PPMO 4 External collaborations EXPANSION INTO EUROPE AND OTHER REGIONS EARLY US LAUNCH SUCCESS Sarepta will be the global leader in RNA-targeted precision medicine to treat DMD 29
Slide 33: Thank you
Thank you ©2016 Sarepta Therapeutics, Inc. 215 First Street, Cambridge, MA 02142. All rights reserved. Sarepta, Sarepta Therapeutics and the Sarepta logo are registered trademarks of Sarepta Therapeutics, Inc. EXONDYS and EXONDYS 51 are trademarks of Sarepta Therapeutics, Inc.
Sarepta Therapeutics — Corporate Update (January 2017) on 6ix