Sarepta Therapeutics, Inc. · NASDAQ:SRPT
Sarepta Therapeutics — J.P. Morgan Healthcare Conference 2015
37 slides from Sarepta Therapeutics's conference presentation, Jan 12, 2015
Slide 1: Realizing the Potential of RNA-Based Technology
Exhibit 99.1 Exhibit REALIZING JP JANUARY MORGAN 99.115, THE HEALTHCARE 2015 POTENTIAL CONFERENCE OF RNA-BASED TECHNOLOGY
Slide 2: Forward Looking Statements
FORWARD This “potential,” advancing strategic antibiotic-resistant Food potential eteplirsen our for Each and research, product with timelines regulatory impact Commission Any 2forward-looking our product protect uncertainties of presentation additional forward-looking and our product the candidates
Slide 3: Sarepta Corporate Highlights
SAREPTA CORPORATE Sarepta Priority Differentiated 5Significant 2More 3Well-capitalized DMD Chemistries than is remains clinical a10 THERAPEUTICS progress late-stage new tested PMO HIGHLIGHTS trials on with research DMD chemistries made inunderway ~$211 development humans –advancing programs advancin
Slide 4: Duchenne Muscular Dystrophy: Devastating Rare Disease with Significant Unmet Need
DUCHENNE DEVASTATING Affects 25,000- Sarepta’s Follow-on National 4Percent approximately of 30,000 Human lead DMD Exon-Skipping MUSCULAR program patients Genome boys RARE amenable 1(Exon in DISEASE inResearch the Drugs 3,5001 DYSTROPHY 51) U.S. tohave estimated boys skipping and WITH Institute poten
Slide 5: DMD to Becker Muscular Dystrophy Shift Would Be Meaningful
DMD ANY BMD The Typically high Diagnosis Difficulty Patients Loss ECKER B Cardiac Childhood Death 51. Lung Muscular majority CK of SHIFT is COMPARED Ambulation Function can aof readings. between milder Walking Adolesence life Dystrophy often FROM ofthrough patients phenotype Decline run ages TO DUCH
Slide 6: Regulatory and Clinical Update for DMD
REGULATORY DMD 168 All Continued Ongoing NDA Rescoring Agreement Placebo-controlled FDA 168-week Safety Results Dystrophin Patient Meeting 6EMAevaluable Week Feedback communicated still Submission Update encouraged GLOBAL data level from in stability planned clinical Data of decline December to in R
Slide 7: Eteplirsen and Follow-on Exon-Skipping Drugs Clinical Program Status
DMD ETEPLIRSEN Duration Study (weeks) 4658-33 4658-28 4658-201 4658-202 4658-301 4658-204 4658-203 4053-101 4045-301 71. 212 CLINICAL US/EU weeks Treatment Single 12 Exon TBD 28EU 2401 96 48 US EU in nAND Target EU/US US Dose 19 Status extension 12 120 20 36PROGRAM Completed 12 Completed Dosing FOLL
Slide 8: Sarepta's Clinical Pipeline Update
28Sites SAREPTA’S CLINICAL Rare DMD PROMOVI 11 of Disease 18 PIPELINE Eteplirsen SRP-4053 SRP-4045 SRP-4050 SRP-4052 SRP-4055 SRP-4008 Clinical Partially Data 4658-204 4658-203 Enrolling 4053-101 of –274+ 4658-301 PROGRAMS Surgical EXON DMD potentially enrolling 14 39 Hub Development Infusion enroll
Slide 9: PMO Chemistry Safety for Chronic DMD Therapy
PMO not CHRONIC >2080 Given Does Charge Plasma Cleared Sequence-specific 9*Sarepta FOR to doses, neutral half-life through DMD internal activate THE LIFELONG representing PMO boys of the TREATMENT data innate binding 3kidney* to with chemistry on5THERAPY immune file hours* doses ~50 to pre-mRNA pati
Slide 10: Eteplirsen Study Design
ETEPLIRSEN STUDY Study nPlacebo-controlled R 30 Placebo 50 n=4 *Placebo-controlled n=2 24 Muscle Screening/Eligibility Out-of-frame Between Receiving KEY % 6MWT2 Pulmonary 1Safety PK 10 =4 Primary Dystrophin mg/kg/wk weeks n=4 n=2 INCLUSION ENDPOINTS 201: and biopsy: *201: n=4 n=2 the 200 24 n=2 End
Slide 11: Patient Characteristics at Baseline
PATIENT STUDY Cohort mean mITT† PBO/Delayed-Tx* (n=10) ITT 50 (n=12) Total (Min, *11.0) †** to 11The Placebo/delayed-treatment Week mg/kg 6MWT 3012 39.8) (kg) mean Max) Modified-Intent-To-Treat N mg/kg Eteplirsen 24. 9.3 201: Age 4CHARACTERISTICS (cm) baseline 9.1 mean (7.3, 31.5 RANDOMIZED, 4(yrs)
Slide 12: Eteplirsen Study Enrolled More Advanced Patients
ETEPLIRSEN Healthier STUDY Excluded Eteplirsen 9.3 DMD114117 No DMD114876 (DEMAND DMD114044 Phase Ataluren Selected Study *Controlled/prospective 12Per 371sponsor I/II II III screened Yes Baseline for Phase Patients Period (400 201/202 3.2 V) II) III) anclinical STUDY + 7.3 enriched 7.8 IIb out Year
Slide 13: 6-Minute Walk Test and Natural History
SIXpatients NATURAL 6MWT Natural “Those “The related 36MWT, for INCLUDED Included Aldurazyme® Elaprase® Myozyme® Vimizim® Served Note: 1McDonald, 2Mazzone, 4Information 5Muenzer 6Rubin 13 MINUTE sharper Aldurazyme to as above is et history as 6-minute the an the al, et for et who aHISTORY for al Hen
Slide 14: Continuous Eteplirsen Cohort Walking Distance Stable Over Time
LESS LAST =65m* METERS *p BEFORE CONFIRMED PRODUCTION delayed All performer After Note: 14 ?patients 0.017 general 3.2 168 Note: THAN TIME eteplirsen years DYSTROPHIN weeks inshowed Statistical delayed POINT stability 80ofMETERS oftherapy treatment continuous declines group) on analysis the the LOST
Slide 15: 6MWT Maintained at Week 168 in Eteplirsen Patients
PATIENTS PLACEBO-DELAYED Age 9.1 12.4 Mean 9.3 Median 6MWT 254.5m 327.5m 323.0m 399.7m Note: 15 atNote: WK Baseline is 12.5 a MAINTAINED 168 measure Statistical (yrs):ofanalysis total PATIENTS AVERAGE endurance basedDECLINED onand 6MWT modified how DISTANCE well AT Intent-To-Treat SIMILAR the entire
Slide 16: 6MWT Change from Baseline After 168 Weeks
AFTER 6MWT n=10) Short METERS Long Treatment WK After WEEK Baseline DATA Note: 16 36: 168: Term 3.2 Note: BASED CHANGE > 56 ambulation years 65 2.5 Benefit Meters Statistical Meters YEARS of ONtherapy FROM MAXIMUM foranalysis OFthe the BASELINE THERAPY continuous mean based 6MWT age on (WEEK TO etep
Slide 17: Walking Distance by Baseline Population
WALKING POPULATION DATA Eteplirsen Last Dystrophin Baseline/Wk36 The Note: this 17 time study Timepoint Mean BASED point group 6MWT in DISTANCE age: All excludes ON Before Distance (n=10) which 9.8 Subjects <350M MAXIMUM years Confirmation walked two IN >and at 350M POPULATION patients week >350M <3
Slide 18: Function Status at Week 168
Baseline/Wk36 7E SIX *Henricson, 18TEPLIRSEN BOYS AGED et al. TREATED Distance 2013 10-12 <(AVG. BOYS 350MAGE FUNCTION 11.5) AND STATUS SIX AGED AT WEEK 13-15 168 (AVG. (n=12) AGE 13.4) AT WEEK 168 (n=12) NATURAL HISTORY SUBJECT STUDY ON GC-TREATED VS. GC-NAÏVE PATIENTS* (n=347)
Slide 19: Pulmonary Function Respiratory Measures
PULMONARY RESPIRATORY Respiratory Majority MAXIMUM Sensitive FVC Declines SHORT PMO 2’OMe Diaphragm Significant LONG Braverman 19 hasLONG in of been measures increase et MIP respiratory decline INSPIRATORY al; shown STABILITY FUNCTION and FUNCTION Smith of begins of MEP to respiratory dystrophin fai
Slide 20: Stability of Pulmonary Function vs Natural History
STABILITY 9.7 Median 12.5 9.3 Mean NATURAL FVC MIP MEP Age Below Ages *Humbertclaude 20 Wilson Ambulation at & %% 8 8WK 11–Baseline MEP –Predicted Predicted 12.9 11 et 16168 al. HISTORY RESPIRATORY OF 1984 et (yrs): PULMONARY Lost al.equations European SHOWS FUNCTION Journal DECLINES FUNCTION of Ped
Slide 21: Eteplirsen Safety Profile
ETEPLIRSEN SAFETY Well Missed –No 21 Two One Reported Liver-specific PBO/Delayed-Tx Eteplirsen Only No clinically hospitalizations, signs tolerated subject reported treatment-unrelated instances one doses or PROFILE cases instance symptoms cohorts missed significant primarily incidents with SAFETY e
Slide 22: Long-Term Safety of Eteplirsen
LONG-TERM SAFETY ETEPLIRSEN PLACEBO FOR TREATMENT- EMERGENT 24 144 ADVERSE WKS n=4 n=8 6Procedural Vomiting Hypokalaemia Cough Back Fall Headache 4Balance Diarrhoea 3Dermatitis Pyrexia Haematoma Abdominal Nausea Rhinitis Polyuria 2Muscle Musculoskeletal 5Proteinuria† 01Injection Through 3% –1.1% 2.3
Slide 23: Preclinical Safety Profiles
PRECLINICAL NO R ROUTE/DOSE SPECIES DRUG STUDY Bacterial GENOTOXICITY Negative: N/A Mutation Chromosomal induction IV, SAFETY Micronucleus P No Vitals IV: Pulmonary CNS 4C REPEAT 6Eteplirsen Kidney TOXICITY SRP-4045 IV SRP-4053 Immunotoxicity Cynomolgus Reproductive Cardiovascular 9N Complement Toxi
Slide 24: Research Pipeline
5 THERAPEUTICS RESEARCH PIPELINE
Slide 25: 2014 Accomplishments: Building a Fully Integrated Company
2014enrollment SETTING 204 Pompe CTA 301 Myostatin Lupus Initiated Additional DEC JAN IND Sarepta’s labs Manufacturing Small Corporate Cambridge DMD transition TLR Research 25 open Opened sequences ACCOMPLISHMENTS: scale Development research research research successful in research Aresearch toSTRON
Slide 26: What Makes Eteplirsen Different: Sequence, Chemistry, Dose
WHAT SEQUENCE, DMD MORPHOLINO PHOSPHORODIAMIDATE Drug-specific SEQUENCE PHOSPHOROTHIOATE 2’-O-METHYL Charge CHEMISTRY Plasma DOSE Up 26 to 9 50 EXON-SKIPPING neutral negative MAKES half-life mg/kg mg/kg CHEMISTRY optimized subcutaneous intravenous of ETEPLIRSEN OLIGOMER 2-6 hours 19-56sequence days
Slide 27: Differentiated Chemistry and Sequence
DIFFERENTIATED CHEMISTRY, LEIDEN IN CYTOKINE BETWEEN SAREPTA PRO051 EXON-SKIPPING Source: 27 A MDXHeemskerk, Sarepta SEQUENCE RESEARCHERS COMPARISON MOUSE PMOS SCREENING SEQUENCE, Internal ACTIVITY AND et MODEL CHEMISTRY SHOWED al,Data 2’OME 2009 SHOWED DEMONSTRATED STUDIES AND ACROSS CHEMISTRIES UP
Slide 28: Next Generation PMO-Based Chemistries
*28UNIVERSE NUMEROUS POTENTIALLY NEXT PMO-X® SPECIFICITY PMOs STABILITY A are GENERATION Hudziak, PMOplus® charge highly CHEMISTRIES OFENHANCED adaptable resistant neutral etPOSSIBILITIES al.PPMO PMO 1996 which tomolecules degradation CHEMISTRIES VERSATILITY CURRENTLY TISSUE may WITH limit and, TARG
Slide 29: PMO-Based Research Platform
229 PMO-BASED SAREPTA HUMAN Promisingunique SAFETY PMO program: PMOplus: Ongoing Opportunity SAREPTA’S Inhibition Control Alteration of chemistry HAS of dosed mRNA to CHEMISTRY mRNAchemistries evaluation of miRNA mRNA PMO MULTIPLE DATA platform—exon upregulate >3upfunction years PLATFORM in to GENER
Slide 30: Strategic Research Focus in Rare Disease
STRATEGIC SAREPTA THERAPEUTICS 7RESEARCH RGD) Multiple Rare High areas Validated skipping/inclusion IV Sarepta commercialize 30Research dosing Genetic unmet Potential Inoropportunities vitro using Genetically strategic FOCUSING collaborations needs MOVING Disease AREAS data for globally exon single
Slide 31: Sarepta Clinical and Research Programs
10nti SAREPTA CLINICAL Rare DMD Progeria Adult Lupus Marburg Ebola Influenza Infective -A Drug-Resistant 31 NEW Drug-Resistant Diseases Onset Virus Exon & & Becker Progerin Virus RESEARCH Graft Drug-Resistant AVI-7100 IS PROGRAMS AVI-7537 51 53 45 50 44 852 Pompe SRP-4008 MAKING DMD AVI-7288 vs. Ete
Slide 32: Myostatin Inhibition Approach
MYOSTATIN APPROACH SPLICE-ALTERING Inhibition Different Research PMO’s EXON Myostatin AONs RATIONALE Sarepta’s INHIBITION Sarepta PMO SEVERAL MEDICAL Duchenne Muscle Cachexia Open 32 to orProprietary SKIPPING restored IN loss PMO’s technology collaborations PMOplus® MOA ongoing PMO caused of and Exo
Slide 33: Adult Onset Pompe Disease Potential Therapies
ADULT POTENTIAL THERAPIES SAREPTA’S Exon Sarepta’s Approach PMO Adult Has Enzyme Demonstrated 33 potential inclusion or onset PMOplus replacement ONSET technology similar Pompe TECHNOLOGY to –STAND-ALONE partial approach RESTORES provide POMPE tochemistries Disease, DMD has could restoration benefit
Slide 34: Toll-Like Receptor Antagonist Program
TOLL-LIKE SUPPORTS Sarepta Research Provides TLR IP 34 position program signaling has Sarepta program established proprietary PIPELINE RECEPTOR may can hastargets be initiated also beused applicable based been rights APPROACH inin (TLR) multiple inlinked the on to Lupus AONs novel prevention in ANTA
Slide 35: Antibacterial Opportunity: PPMO Restores Antibiotic Susceptibility
OPPORTUNITY Sarepta Six NDM-1 This PPMO’s Broad Opportunities Colistin, PPMO CDC; EMA Harbour Smith 35 programs isand Antibiotic B, commercial ainhibits RESTORES isPPMO’s L, new that Meropenem, etECDC; focused et al.target strategy identified al. for ICAAC bacterial Resistance ICAAC can TO partnersh
Slide 36: Financial Overview
FINANCIAL SHARES RECENT 1.5 TRADING (90 MARKET CASH ~$211 36 (UNAUDITED) million daymillion &average OTHER OUTSTANDING CLOSING CAPITALIZATION shares VOLUME OVERVIEW asvolume) ofdaily INVESTMENTS 12/31/14 PRICE41.3 $14.93 ~$617 million asmillion of 1/7/15
Slide 37: Thank you
THERAPEUTICS
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