UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, DC 20549

FORM 8-K

CURRENT REPORT
Pursuant to Section 13 or 15(d)
of the Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): October 1, 2015

Sarepta Therapeutics, Inc.
(Exact name of registrant as specified in its charter)

 
 	 	 	 	 	
Delaware	 	001-14895	 	93-0797222	
(State or other jurisdiction
of incorporation)	 	(Commission
File Number)	 	(IRS Employer
Identification No.)	
215 First Street
Suite 415
Cambridge, MA 02142
(Address of principal executive offices, including zip code)
(617) 274-4000
(Registrant’s telephone number, including area code)
 
(Former name or former address, if changed since last report)

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):
 
¨	Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)	
 
¨	Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)	
 
¨	Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))	
 
¨	Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))	

 

Item 7.01	Regulation FD Disclosure.	
On October 1, 2015, Sarepta Therapeutics, Inc. (the “Company”) hosted a conference call to provide an update regarding its leading product candidate, eteplirsen. A copy of the presentation is attached as Exhibit 99.1 hereto.
Also on October 1, 2015, the Company issued a press release regarding the conference call described above. A copy of the press release is attached as Exhibit 99.2 hereto.
The information in this report furnished pursuant to Item 7.01, including Exhibits 99.1 and 99.2 attached hereto, shall not be deemed “filed” for the purposes of Section 18 of the Securities and Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section. It may only be incorporated by reference in another filing under the Exchange Act or the Securities Act of 1933, as amended, if such subsequent filing specifically references the information furnished pursuant to Item 7.01 of this report.
 
Item 9.01	Financial Statements and Exhibits.	
(d) Exhibits.

Exhibit
Number	 	Description	
 	 	
99.1	 	Sarepta Therapeutics Investor Update Slide Deck	
 	 	
99.2	 	Press release dated October 1, 2015	

SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

Sarepta Therapeutics, Inc.	
 	 	
By:	 	/s/ Edward Kaye	
 	 	Edward Kaye	
 	 	Interim Chief Executive Officer, Senior
Vice President and Chief Medical Officer	
Date: October 1, 2015

EXHIBIT INDEX

Exhibit
Number	 	Description	
 	 	
99.1	 	Sarepta Therapeutics Investor Update Slide Deck	
 	 	
99.2	 	Press release dated October 1, 2015	

 	
Exhibit 99.1

 
Sarepta Therapeutics Investor Update
October 1, 2015

 	

Presenter
Edward Kaye, MD
SVP, Chief Medical Officer Interim Chief Executive Officer Sarepta Therapeutics Cambridge, Massachusetts, USA
2	 	 	

 	

Forward-Looking Statement
This presentation, contains forward-looking statements. These forward-looking statements generally can be identified by the use of words such as “believes” or belief,” “anticipates,” “plans,” “expects,” “will,” “intends,” “potential,” “possible,” “advance” and similar expressions. These forward-looking statements include statements about the safety and efficacy of eteplirsen, analysis of eteplirsen and control cohort data and their implications, and eteplirsen’s potential as treatment for Duchenne
Muscular Dystrophy, the potential market for our exon skipping product candidates and Sarepta’s mission, commitments and business plans and strategies. Forward-looking statements also include those made during the presentation regarding future business developments and actions and the timing of the same.
Each forward-looking statement contained in this presentation is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statement. Applicable risks and uncertainties include, among others: the results of our ongoing research and development efforts and clinical trials for eteplirsen and our other product candidates may not be positive or consistent with prior results or demonstrate a safe treatment benefit; there may be delays in our projected timelines or our product candidates, chemistries or technologies may never become commercially available for regulatory or other reasons including a negative decision on our NDA for eteplirsen by an advisory committee or the FDA; agency or court decisions with respect to our patents or those of third parties may negatively impact our business; our product candidates and or the use of or application of our chemistries and technology may fail in the research, development or commercialization process for various other reasons including the possibility that we may not be able to comply with all regulatory requests and requirements for the research, development and commercialization of our product candidates; and those risks identified under the heading “Risk Factors” in Sarepta’s Annual Report on Form 10-K for the year ended December 31, 2014 filed with the Securities and Exchange Commission (SEC), Sarepta’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2015 and Sarepta’s other filings with the SEC, which we encourage investors to review at www.sec.gov, for a more detailed discussion on risks and uncertainties relating to our business.
Any of the foregoing risks could materially and adversely affect Sarepta’s business, results of operations and the trading price of Sarepta’s common stock. We caution investors not to place considerable reliance on the forward-looking statements contained in this presentation. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.
3

 	

Panelists
Anne Connolly, MD
Professor, Neurology and Pediatrics Neuromuscular Division Washington University in St Louis St. Louis, Missouri, USA
Jerry R Mendell, MD
The Ohio State University College of Medicine
Nationwide Children’s Hospital
Columbus, Ohio, USA
Eugenio Mercuri, MD, PhD
Professor of Pediatric Neurology Università Cattolica del Sacro Cuore Rome, Italy
4

 	

Panelists (cont’d)
Perry Shieh, MD, PhD
Associate Professor, Department of Neurology Director, Neuromuscular Division
David Geffen School of Medicine, University of California, Los Angeles Los Angeles, California, USA
Steve Wilton, PhD, BSc
Professor
Foundation Chair in Molecular Therapy Centre for Comparative Genomics Murdoch University Perth, Australia
All panel members either advise or consult for Sarepta Therapeutics.
5

 	

Summary of Accomplishments
Confirmed clinical activity of eteplirsen in an intention-to-treat (ITT) analysis of 6-minute walk test (6MWT) for Study 201/202 vs external control
Dystrophin production confirmed by each methodology:
– 4th biopsy
Reverse-transcription–polymerase chain reaction (RT-PCR)
Dystrophin intensity
% dystrophin-positive fibers
Western blot
Expanded clinical program
Expanded safety database
Eteplirsen New Drug Application (NDA) filed for accelerated approval
6

 	

Key Data Included in NDA Filing
ITT analysis of 6MWT or Study 201/202 vs external control
– Intermediate clinical endpoint upon which the eteplirsen NDA filed with FDA
Pulmonary function
4th biopsy
Rescore of % dystrophin-positive fibers
Safety
7

 	

ITT Analysis of 6MWT for Study 201/202 vs External Control
8	 	 	

 	

Establishing an Appropriate External Control Cohort Per FDA Request
Only two Duchenne muscular dystrophy (DMD) registries contained longitudinal 6MWT data up to 36 months and included:
– Genetic mutation data
– Equivalent care standards including steroids
Prospectively defined filters were used to identify external controls from these registries
– Age, steroid use, and mutation type
– External control patients would have been eligible for eteplirsen 201 trial based on baseline characteristics
Leuven Neuromuscular Research
Italian DMD Telethon Center (NMRC), Belgium Eteplirsen N=97 N=89 N=12 (ITT) Clinical 6MWT 6MWT 6MWT, PFT Outcomes
6MWT, 6-minute walk test; NSAA, North Star Ambulation Assessment; PFT, pulmonary function tests.
9

 	

Italian Telethon & Leuven NMRC DMD Natural History
Registries
Investigator-initiated studies, independent of sponsor
Patients treated according to CDC/TREAT-NMD care standards
– Steroid use recorded
Enrolled all patients who met eligibility criteria
– Attending a participating neuromuscular clinic
– Genetically confirmed diagnosis of DMD
– No cognitive impairment that could affect 6MWT performance
6MWT administered by trained physical therapists according to modified American Thoracic Society procedure
Published data in peer-reviewed articles
10

 	

Derivation of External Control Groups by Prospectively Defined Filters
Prospectively defined filters applied to identify
N=186 external controls (age ?7, steroids, mutation
DMD Raw Data
type)
N=116 • Non-biased (recognized institutions, databases Baseline Steroids utilized by established DMD companies)
Baseline & ?1 Post-baseline
6MWT Available • Requested by FDA
N=25 N=91 All Mutations
Age <7 Age ?7
N=41 N=50
Not Amenable to Amenable to Exon Any Exon Exon Skipping Skipping
N=37 N=13
Amenable to Amenable to Exon 51 Other Exon Skipping Exon 51 Skipping
N indicates participants at baseline; some patients did not contribute data through 36 months.
11

 	

Impact of Baseline Age (<7 or ?7) on 6MWT Trends for
Any Genotype
PATIENTS <7 SHOW IMPROVED 6MWT PERFORMANCE WHILE
PATIENTS ?7 SHOW DECLINE
Patients <7 initially improve in walking ability through 24 months and maintain 6MWT above baseline through 36 months
– 54-meter increase observed in the first 24 months
It is challenging to show a benefit in 6MWT in ages <7 as patients are improving due to growth & development where growth outpaces the disease
450 +54 m
(m) N=25 +25 m Age: 6.0 +21 m ***
400
Distance ** *** -31 m
350
N=91 D = 167 m†
6MWT Age: 9.4
300 -93 m p<0.001
Mean **p<0.01; ***p<0.001
250 <7 Years, Any Genotype (N=25)
?7 Years, Any Genotype (N=91) -143 m
200
Baseline Month 12 Month 24 Month 36
†Difference in mean change from baseline.
12

 	

Impact of Baseline Age (<7 or ?7) on 6MWT Trends in Patients Amenable to Exon Skipping
SIMILAR TREND OBSERVED IN EXON SKIP AMENABLE PATIENTS
Patients <7 initially improve in walking ability through 24 months and maintain 6MWT above baseline through 36 months
450 ***
+14 m
N=17 *** 400 Age: 6.0 * +50 m (m) +15 m
350
N=50
Distance Age: 9.7 †
300 -44 m D = 194 m
6MWT p<0.001
250
Mean *p<0.05; ***p<0.001
-111 m
<7 Years, Amenable to Exon Skipping (N=17) 200
?7 Years, Amenable to Exon Skipping (N=50)
-180 m
150
Baseline Month 12 Month 24 Month 36
†Difference in mean change from baseline.
13

 	

Exon 51 Declines More Rapidly Than Other Genotypes
400
300
(m) Distance 200 6MWT Mean 100
External Control—All Mutations, ?7 Years Old
External Control—Any Exon, ?7 Years Old External Control—Exon 51, ?7 Years Old
0
Baseline Month 12 Month 24 Month 36
14

 	

Patient Characteristics at Baseline: Eteplirsen and External Control Groups Were Well Matched
Parameter Pivotal Study 6MWT External Control Groups
Study 201/202 Exon 51 Any Exon Number of patients N=12 N=13 N=50
Age, years
9.4 (1.18) 9.5 (1.45) 9.7 (1.52)
Mean (SD)
6MWT distance, m
363.2 (42.19) 357.6 (66.75) 355.7 (87.28)
Mean (SD)
Deletion mutations 45–50, 48–50, 45–50, 48–50, Skippable represented: 49–50, 50, 52 49–50, 50, 52 mutations Steroid use 100% 100% 100%
15

 	

151-Meter Difference Between Eteplirsen-treated vs Matched External Controls at 3 Years
ITT ANALYSIS, N=12 FOR ETEPLIRSEN-TREATED PATIENTS AT BASELINE, 12, 24, AND 36 MONTHS
External controls were steroid-treated, aged ?7, and amenable to exon 51 skipping
N=12 Age: 9.4
350
(m) N=13 Age: 9.5 **
SEM 300 ±
250
Distance 200 D=151 m†
6MWT p<0.01
150
Mean **p<0.01
100
Eteplirsen-treated, Amenable to Exon 51 Skipping, ?7 (N=12) External Control, Amenable to Exon 51 Skipping, ?7 (N=13)
50
Baseline Month 12 Month 24 Month 36
2 patients in the historical group did not contribute data to the Month 36 timepoint
†Difference in mean change from baseline.
16

 	

Analysis Shows Slower Rate of 6 MWT Decline in
Eteplirsen Treated Patients Compared to Multiple Controls
400
(m) 300
Eteplirsen N=12† Distance N=91†, Ä=43 m‡
200
6MWT N=50†, Ä=79 m‡
Eteplirsen-treated (Exon 51)
Mean 100 N=13†, Ä=151 m‡
External Control—All Mutations, >7 Years Old p<0.01 External Control—Any Exon, >7 Years Old External Control—Exon 51, >7 Years Old 0
0 Month 12 Month 24 Month 36
†Sample size at baseline.
‡Difference at 36 months in mean change from baseline.
17

 	

Individual Patient Data for Eteplirsen (N=12) vs External Control (EC)
DIFFERENCE IN RATE OF DECLINE OBSERVED IN THE MAJORITY OF PATIENTS
External control—Exon 51 (N=13) Eteplirsen-treated (N=12) 500
(m)
400
Distance 300 6MWT 200 Mean 100
0
Baseline Month 12 Month 24 Month 36
Baseline 12 Months 24 Months 36 Months
EC Etep EC Etep EC Etep EC Etep % Walking >300 m 85% 92% 62% 83% 46% 67% 23% 58% % Walking >150 m 100% 100% 92% 83% 77% 83% 46% 75% % Non-ambulant 0% 0% 8% 17% 23% 17% 46% 17%
External control (age ?7, on steroids, amenable to exon 51 skipping)
18

 	

Eteplirsen-treated Patients (N=12) Showed a Lower Rate of Loss of Ambulation Than External Control
6/13 (46%) untreated external controls lost ambulation over 3 years
2/12 (17%; all in year 1) eteplirsen patients lost ambulation over 3 years
50
Eteplirsen-treated—Amenable to Exon 51 Skipping, ?7 (N=12) 46.2%
45 Historical Control—Amenable to Exon 51 Skipping, ?7 (N=13)
40
35
30
Percent 25 23.1%
20
16.7% 16.7% 16.7%
15
10 7.7%
5
2/12 1/13 2/12 3/13 2/12 6/13 0 0–12 0–24 0–36 Total
Months
19

 	

Eteplirsen-treated Cohort Maintains Benefit Through Week 192
10/12 ETEPLIRSEN-TREATED BOYS REMAIN AMBULANT AT WEEK 192 (MEAN AGE 12.9) –
~160 WEEKS SINCE AN ETEPLIRSEN-TREATED BOY LOST AMBULATION
Eteplirsen
0 Week 192
-10 -16% -22% -20
(m) -19% -31%
Baseline -30
-40 -42% -43% from -45% Distance -50
-60 change 6MWT -70 in Eteplirsen-treated, Amenable to Exon 51 Skipping, ?7 (N=12)
Mean -80 Eteplirsen-treated, Excludes Placebo-delayed, ?7 (N=8) -74% -90
External Control, Amenable to Exon 51 Skipping, ?7 (N=11)
-100
Baseline Month 12 Month 24 Month 36 Month 48
N=8 at month 48 as the placebo crossover patients (N=4) began treatment at week 25 (wk 25-192) and have not reached 48 months of treatment as other subjects did (wk 1-192) and have only completed 168 weeks of treatment included above. All patients are included (N=12) above in the eteplirsen values from when they started therapy. No new non-ambulant patients or discontinuations.
20

 	

Pulmonary Function
21

 	

Pulmonary Function: Eteplirsen-treated Patients (N=12) Remain Relatively Stable through Week 192
Age at Baseline (yrs): Age at WK 192 (yrs):
125 Mean 9.3 Mean 12.9
Median 9.7 MEP % Pred (N=12) MIP % Pred (N=12) FVC % Pred (N=12) Median 13.4 101 102 99 98 100 96 95 95
92 92 94 92
92 90 89
87 84 85 93 91
% 82 84 84 88 86 90
77
75 79 81 79
75 78 77 78 76
72 75 74
72 66
50
BL 12 24 36 48 62 74 84 96 120 144 168 192
WEEKS
NATURAL HISTORY SHOWS STEADY DECLINES OVER TIME IN PULMONARY FUNCTION IN DMD PATIENTS
FVC % Predicted MIP % Predicted MEP % Predicted
CINRG 2013 CINRG 2013
MEP, maximum expiratory pressure; MIP, maximum inspiratory pressure; FVC, forced vital capacity; BL, baseline.
*Wilson et al. 1984 equations.
22

 	

Efficacy Summary
SLOWER RATE OF DMD PROGRESSION AT 3 YEARS OBSERVED IN STUDY 201/202 ETEPLIRSEN TREATED PATIENTS AS MEASURED BY MULTIPLE FUNCTIONAL ENDPOINTS
Slowed disease progression at 3 years compared to external controls amenable to exon 51 skipping
– At 3 years 6MWT Ä = 151 m, p< 0.01
– Decrease in proportion losing ambulation (17% vs 46%)
Relative stability in % predicted MIP and MEP over 3 years compared to data from the scientific literature
23

 	

FOURTH BIOPSY
24

 	

Background on Fourth Biopsy
11/12 patients volunteered for surgical biopsy
– All protocols designed in collaboration with FDA
Measurements of mechanism of action
– RT-PCR
Measurements of dystrophin expression
– Percent dystrophin-positive fibers
– Dystrophin signal intensity
– Western blot
Measurements were blinded, randomized, and analyzed by 4 independent pathologists
25

 	

Demonstration of Exon Skipping by RT-PCR and Confirmed by Sequencing
4th Biopsy: All (100%) patients (N=11*) demonstrated exon 51 skipped mRNA product after 180 weeks of treatment was present
In-frame mRNA transcripts as a result of exon 51 skipping confirmed by sequencing in all patients
100% of patients dosed with eteplirsen in completed clinical studies to date demonstrated exon skipping (N=36)
*1 boy opted out of the voluntary surgical biopsy.
26

 	

Dystrophin-positive Fibers Visibly Present in Eteplirsen-treated Patients’ 4th Biopsies Compared to DMD Exon 51 Skip Amenable Control Biopsies
DYSTROPHIN DETECTED IN 10/11 BIOPSIES vs NO DYSTROPHIN-POSITIVE FIBERS IN ANY DMD 51 CONTROLS
Eteplirsen-Treated Week 180 Untreated Exon 51 DMD Controls
PT #1 PT #5 PT #9 DMD #1 DMD #5
DMD #2 DMD #6 PT #2 PT #6 PT #10
DMD #3 DMD #7
PT #3 PT #7 PT #11
DMD #4 DMD #8
PT #4 PT #8
One (1) eteplirsen
DMD #9 subject declined the optional 4th surgical biopsy
27

 	

Patient #11: All 4th Biopsy Images Compared to Exon 51 DMD Skip Amenable Controls at Week 180
SUBJECT 11: EVERY IMAGE OF 4TH BIOPSY SHOWN BELOW TO SHOW MULTIPLE LAYERS OF BIOPSY
Eteplirsen-Treated Week 180: Untreated Every Biopsy Image Patient #11 DMD 51 Controls
PT #11 PT #11 PT #11 PT #11 DMD #1 PT #11 PT #11 PT #11 PT #11 DMD #2 PT #11 PT #11 PT #11 PT #11 DMD #3 PT #11 PT #11 PT #11 PT #11 DMD #4
28

 	

Statistically Significant Increase in Percent Positive Fibers Observed in Treated vs. Control
PROTOCOL REVIEWED BY FDA PRIOR TO EVALUATION OF TISSUE BY
4 BLINDED PATHOLOGISTS
Flagship Nationwide
Increase in Detected Dystrophin Positive Fibers
1453% 641% Treated vs Untreated
60 ***
p<0.001 ***
Treated Untreated
Fibers 50
Positive 40 ***
% ***
(+SD) 30 *** Dystrophin 20 Mean 10
0 Pathol. 1 Pathol. 2 Pathol. 3 Pathol. 4 Flagship Nationwide
29

 	

Mean Fluorescence Intensity Demonstrated a Statistically Significant Increase in Treated vs Controls
Increase in Detected Dystrophin
140% Treated vs Untreated
40 ***p<0.001
(+SD) Intensity 30 Fluorescent 20 Relative 10 Mean
0
Treated Untreated
30

 	

Role of Western Blot
Primary diagnostic tool prior to the introduction of genetic testing
– Absence of band confirmed DMD
– Presence of band confirmed BMD
Nine of 11 eteplirsen-treated patients had an observable dystrophin band
Nine untreated DMD controls amenable to skipping exon 51 used as comparator group
One out of 9 controls had an observable band
DMD, Duchenne muscular dystrophy; BMD, Becker muscular dystrophy.
31

 	

Western Blot Results vs Baseline Patient A
WESTERN BLOT
Patient A Baseline (Untreated) Patient A Week 180 (Treated)
Dystrophin Band (DYS1): Absent Dystrophin Band (DYS1): Present
Consistent 50 µg total protein per lane loaded Exposure time 30 minutes per gel
32

 	

Western Blot Results vs Baseline Patient B
WESTERN BLOT
Patient B Baseline (Untreated) Patient B Week 180 (Treated)
Dystrophin Band (DYS1): Absent Dystrophin Band (DYS1): Present
Consistent 50 µg total protein per lane loaded Exposure time 30 minutes per gel
33

 	

Fourth Biopsy Summary: Following Eteplirsen Treatment, Increased Dystrophin Expression Confirmed by All Quantification Methods
RT-PCR—Exon skipping in 100% of patients and all confirmed by sequencing
Eteplirsen-treated vs Untreated Exon 51-amenable DMD Controls:
Dystrophin Intensity (p<0.001)
– Automated quantification of dystrophin intensity at the sarcolemma using BIOQUANT® software confirmed statistical significant increases
% Dystrophin-positive Fibers (p<0.001)
– 4 blinded pathologists independently scored and confirmed statistically significant increases in dystrophin-positive fibers compared to DMD control biopsies
Western Blot
– Presence of dystrophin protein confirmed in 9 of 11 (82%) of eteplirsen patients at Week 180 vs 1 of 9 (11%) in the DMD control biopsies
34

 	

Rescore of Percent Dystrophin-positive Fibers in Study 201
35

 	

Background on Rescore
Original assessment of dystrophin-positive fibers in Study 4658-201:
Scored by one blinded pathologist
Met primary endpoint
– Statistically significant increase in % dystrophin-positive fibers of eteplirsen-treated patients at Week 24 compared to baseline
Re-assessment of dystrophin-positive fibers in Study 4658-201:
Scored by 3 independent blinded pathologists, plus blinded nationwide pathologist (total 4)
36

 	

3 Independent Pathologists Confirmed Statistically Significant Change in Dystrophin-positive Fibers From Baseline
30 mg/kg: Statistically significant increase of dystrophin-positive fibers from baseline at Week 24, which confirms previously announced result
50 mg/kg: No significant change from baseline at 30
Week 12, which demonstrates a delay in production Placebo **p<0.007 as expected 30 mg/kg positive 50 mg/kg **
Placebo: No significant change from baseline at Weeks—(+SD) 20 12 and 24, which confirms previously announced result
Patient on 30 mg/kg at Week 24
Dystrophin Baseline 10 in from Change 0 Mean Fibers
-10
Week 12 Week 24
Inter-rater reliability (ICC = 0.793) and intra-rater reliability (ICC = 0.944) showed excellent level of concordance among 3 independent pathologists for all treatment groups
37

 	

Safety
38

 	

Eteplirsen Safety Database in the NDA (N=72)
Study & Description Dose (mg/kg) Route Duration of Dosing N
Study 33
0.09, 0.9 IM Single dose 7
Proof-of-concept
Study 28 0.5, 1, 2,
IV† 12 weeks 19
Dose ranging 4, 10, 20
Study 201/202
Double-blind,
30, 50 IV† ~3 years 12 placebo-controlled/ open-label extension
30 IV† 12-24 weeks 12
Studies 301 & 204
Recently initiated 30 IV† <12 weeks 22
ALL ETEPLIRSEN TREATED PATIENTS 72
46 patients (46 patient-years) exposed to ?30 mg/kg proposed clinical dose
†Dose administered once weekly
39

 	

Enhanced Safety Database Submitted in the NDA
NDA submitted with 72 total patients with 46 patient years of experience at ?30mg/kg with >2600 doses provided in the NDA
– 12 patients treated for over 3 years
– 12 patients treated for 3–6 months
– 114 patients to be included in next safety data cut (120-day update)
Most common adverse events were mild and unrelated to study drug similar to 201/202
Adverse drug reactions include flushing, erythema, and mild temperature elevation
No drug-related serious adverse events
– No evidence of drug-related renal, hepatic, coagulation, or severe cutaneous AESIs*
No elevated GGT, glomerular nephritis, hepatocellular injuries
No clinically significant infusion-site reactions with associated ulcers
No thrombocytopenia, intra cranial venous sinus thrombosis, intracranial hypertension
No drug related alopecia
*AESI, adverse event of special interest observed with phosphorothioate anti-sense oligonucleotides
40

 	

Week 192 Safety Update: No Missed Doses Due to Drug-related Adverse Events Through Week 192
Total doses administered >2300 at Week 192
Majority of missed doses due to planned family vacations
No missed doses due to eteplirsen-related adverse events (AEs)
No eteplirsen-related serious AEs
No intermittent dosing needed
No drug holidays, no dose reductions, no discontinuations
No hospitalizations due to drug-related AEs
Most common AEs were mild and unrelated to study drug (201/202)
Adverse drug reactions to Eteplirsen include flushing, erythema, and mild temperature elevation
41

 	

Mean Ejection Fraction Through 192 Weeks (ITT; N=12)
Age at Baseline (yrs): Age at WK 192 (yrs):
Mean 9.3 Ejection Fraction (%) Over 192 Weeks Mean 12.9
Median 9.7 Median 13.4
Mean ± SEM (N=12)
70 60 50 40
30
Week Baseline 1 12 24 36 48 62 74 84 96 120 144 168 192
NATURAL HISTORY SHOWS CARDIAC HEALTH DECREASES OVER TIME AS BOY AGES AND DISEASE PROGRESSES
Mean Change From Baseline to Week 192 No evidence of declining left ventricular ejection fraction at Week 192 of
Mean All Groups 1.49%
eteplirsen treatment
42

 	

Summary of Key Results: Totality of the Data
151-meter advantage in 6MWT of eteplirsen-treated patients compared to Clinical Efficacy external control at 3 yrs (p<0.01)
Pulmonary stability
4th Biopsy (Voluntary: 11 patients) at week 180
– RT-PCR: 100% of patients demonstrated exon skipping, mechanism of action confirmed
Biochemical
– Dystrophin intensity: p<0.001 compared to DMD controls
Efficacy
(Supportive – % dystrophin-positive fibers: p<0.001 vs DMD controls Efficacy) – Western blot: protein production confirmed in 9 of 11 patients
Rescore of Weeks 12 & 24
– 30 mg/kg: Statistically significant increase of dystrophin-positive fibers from baseline at Week 24, which confirms previously announced result p<0.007
Drug continues to remain well tolerated after >2300 doses in 201/202
No injection-site reactions, thrombocytopenia, coagulation, pulmonary Safety embolisms, or renal and hepatic impairment
No hospitalizations due to drug-related adverse events
No decrease in ejection fraction
43

 	

Sarepta’s Vision
OUR MISSION IS TO FIND A TREATMENT FOR EVERY BOY WITH DMD: EVERY MINUTE MATTERS
We are committed to developing therapies for patients with DMD regardless of underlying mutation
– Eteplirsen for exon 51 skipping under FDA review and is the key for PMO exon skipping in DMD
Four clinical trials ongoing, over 100 patients will be receiving eteplirsen once trials fully enroll
– Meeting with EMA and hiring key personnel in 2016 for EU strategy
Exons 53 & 45
– Clinical trials underway in US and EU, will enroll over 100 patients
Our goal is to develop treatments for 8 exons by 2018
– Working collaboratively with the FDA & EMA to determine an approval path in rarer mutations
– Working internally on exons 55, 52, 50, 35, 8, and beginning collaboration on exon-2 duplication
Evaluating approaches beyond exon skipping to bring disease-modifying treatments to all patients with DMD
PMO, phosphorodiamidate morpholino oligomer; EMA, European Medicines Agency.
44

 	

Panel Discussion
45

 	

Thank you
Line open for questions
46

Exhibit 99.2

Sarepta Therapeutics Announces Additional Long-Term Efficacy and Safety Data from
Pivotal Phase IIb Program of Eteplirsen for Treatment of Duchenne Muscular Dystrophy
— Eteplirsen provided a statistically significant 6 minute walk test advantage of 151 meters at three years compared to an external control —
— Fourth muscle biopsy results confirm increased dystrophin production in nearly all eteplirsen-treated patients and exon skipping in 100 percent of patients —
— Eteplirsen safety profile remains consistent with prior results —
Cambridge, Mass.—October 1, 2015—Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-targeted therapeutics, today announced additional clinical efficacy and safety data from the Company’s Phase IIb program of eteplirsen in patients with Duchenne muscular dystrophy (DMD). The data demonstrated that eteplirsen provided a statistically significant advantage of 151 meters in the ability of study participants to walk at three years, compared with external controls. Further, the fourth biopsy data confirmed the mechanism of action of eteplirsen, demonstrating exon skipping in all patients and dystrophin production in nearly all patients. Safety data remained consistent with prior results.
Eteplirsen, Sarepta’s lead drug candidate, is designed to target the underlying cause of DMD by enabling the production of a functional dystrophin protein in patients with mutations amenable to exon 51 skipping. Approximately 13 percent of people with DMD are estimated to have a mutation targeted by eteplirsen/exon 51 skipping.
“We are encouraged by the positive clinical outcomes, such as the statistically significant difference in the 6MWT in eteplirsen-treated patients compared to a control, especially since we see them accompanied by data that continues to demonstrate exon skipping and dystrophin production in most patients,” said Edward Kaye, M.D., Sarepta’s interim chief executive officer and chief medical officer. “We are committed to bringing eteplirsen and our other investigational exon skipping therapies to patients with DMD and will continue to work with all stakeholders to advance these programs as quickly as possible so we can better address the unmet need for treatments in the DMD community.”

Results of Sarepta’s Phase IIb program were included in the New Drug Application (NDA) that Sarepta submitted to the U.S. Food and Drug Administration (FDA) for eteplirsen for the treatment of DMD amenable to exon 51 skipping. The primary clinical endpoint in the NDA was the comparison of the 6MWT ITT analysis of the eteplirsen-treated group compared to an external control with similar inclusion criteria. The FDA granted eteplirsen Priority Review status and assigned a Prescription Drug User Fee Act (PDUFA) action date of February 26, 2016. Previously, the FDA granted Rare Pediatric Disease Designation to eteplirsen, as well as Orphan Drug Designation and Fast Track Status.
New Long-Term Efficacy Data
 
 	•	 	Patients who were treated with eteplirsen experienced a statistically significant 151 meter difference in the 6-minute walk test (6MWT) at three years compared with external DMD controls. The 6MWT is a well-accepted measure of ambulation and clinical function in patients with DMD. (p<0.01).	
 
 	•	 	Eteplirsen-treated patients had a lower rate of loss of ambulation than external DMD controls over three years.	
 
 	•	 	Eteplirsen-treated patients experienced a slower rate of decline through Week 192 than external DMD controls.	
 
 	•	 	Pulmonary function remained relatively stable through approximately four years in eteplirsen-treated patients.	
New results from a fourth biopsy performed on 11 patients demonstrated that exon skipping occurred in 100 percent of patients after 180 weeks of treatment, confirming the mechanism of action of eteplirsen. In addition, biochemical evidence from three quantification methods, analysis of dystrophin positive fibers, dystrophin intensity and Western Blot testing, confirmed that dystrophin was present in most patients following eteplirsen treatment.
Fourth Biopsy Results
 
 	•	 	Confirmed exon skipping in 100% of patients	
 
 	•	 	Percent dystrophin-positive fibers increased (p<0.001) in comparison to untreated controls	
 
 	•	 	Dystrophin intensity increased (p<0.001) in comparison to untreated controls	
 
 	•	 	Western Blot confirmed presence of dystrophin protein in 9 of 11 (82%) of eteplirsen-treated patients at Week 180 vs 1 of 9 (11%) in the DMD control biopsies	
New Long-Term Safety Data
New results from Sarepta’s safety database, which includes approximately 100 patients exposed to eteplirsen, showed that the eteplirsen safety profile remained consistent with prior results. Common adverse drug reactions included flushing, erythema, and mild temperature elevation. No pulmonary embolisms, hospitalizations, injection site reactions or thrombocytopenia have been observed.

Webcast & Conference Call
Sarepta will provide a corporate update and report on recent data from the Phase IIb study of eteplirsen for Duchenne muscular dystrophy via a live webcast and conference call on October 1, 2015 at 7:00 AM EST. The update will be followed by a panel discussion with Duchenne muscular dystrophy experts Anne Connolly, MD; Eugenio Mercuri, MD, PhD; Jerry Mendell, MD; Perry Shieh, MD, PhD; and Steve Wilton, PhD, BSc.
The presentation will be webcast live under the investor relations section of Sarepta’s website at www.sarepta.com and will be archived there for 90 days. Please connect to Sarepta’s website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.
The conference call may be accessed by dialing 877-727-3245 for US domestic callers and 530-379-4673 for international callers. The passcode for the call is 48471076. Please specify to the operator that you would like to join the “Sarepta Corporate Update and Report on Recent Data.”
About the 6-Minute Walk Test (6MWT)
The 6MWT was developed as an integrated assessment of cardiac, respiratory, circulatory, and muscular capacity for use in clinical trials of various cardiac and pulmonary conditions. In recent years, the 6MWT has been adapted to evaluate functional capacity in neuromuscular diseases and has served as the basis for regulatory approval of a number of drugs for rare diseases, with mean changes in the 6MWT ranging from 28 to 44 meters. Additionally, published data from longitudinal natural history studies assessing dystrophinopathy, a disease continuum comprised of DMD and Becker muscular dystrophy, support the utility of the 6MWT as a clinically meaningful endpoint in DMD. These data show that boys with DMD experience a significant decline in walking ability compared to healthy boys over one year, suggesting that slowing the loss of walking ability is a major treatment goal.
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is an X-linked rare degenerative neuromuscular disorder causing severe progressive muscle loss and premature death. One of the most common fatal genetic disorders, DMD affects approximately one in every 3,500 boys born worldwide. A devastating and incurable muscle-wasting disease, DMD is associated with specific errors in the gene that codes for dystrophin, a protein that plays a key structural role in muscle fiber function. Progressive muscle weakness in the lower limbs spreads to the arms, neck and other areas. Eventually, increasing difficulty in breathing due to respiratory muscle dysfunction requires ventilation support, and cardiac dysfunction can lead to heart failure. The condition is universally fatal, and death usually occurs before the age of 30.

About Eteplirsen
Eteplirsen is designed to address the underlying cause of DMD by enabling the production of a functional dystrophin protein. Eteplirsen uses Sarepta’s novel phosphorodiamidate morpholino oligomer (PMO)-based chemistry and proprietary exon-skipping technology to skip exon 51 of the dystrophin gene. This enables the repair of specific genetic mutations that affect approximately 13 percent of people with DMD. By skipping exon 51, eteplirsen may restore the gene’s ability to make a shorter, but still functional, form of dystrophin from messenger RNA (mRNA). Promoting the synthesis of a truncated dystrophin protein is intended to stabilize or significantly slow the disease process and prolong and improve the quality of life for patients with DMD. Eteplirsen has not been approved by the FDA or any regulatory authority for the treatment of DMD.
Data from clinical studies of eteplirsen in DMD patients have demonstrated a broadly favorable safety and tolerability profile and restoration of dystrophin protein expression.
About Sarepta Therapeutics
Sarepta Therapeutics is a biopharmaceutical company focused on the discovery and development of unique RNA-targeted therapeutics for the treatment of rare, infectious and other diseases. The Company is primarily focused on rapidly advancing the development of its potentially disease-modifying DMD drug candidates, including its lead DMD product candidate, eteplirsen, designed to skip exon 51. Sarepta is also developing therapeutics for the treatment of infectious diseases, such as drug-resistant bacteria and other rare human diseases. For more information, please visit us at www.sarepta.com.
Forward Looking Statements
This press release contains “forward-looking statements” within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Any statements contained in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Words such as “believes,” “anticipates,” “plans,” “expects,” “will,” “intends,” “potential,” “possible” and similar expressions are intended to identify forward-looking statements. These forward-looking statements include statements regarding the safety and efficacy of eteplirsen, analysis of eteplirsen and external control data and their implications, eteplirsen’s potential as a treatment for Duchenne Muscular Dystrophy and its potential market size and Sarepta’s commitment to bringing eteplirsen and its other exon skipping investigational therapies to patients with DMD and plans to continue working with all stakeholders to advance these programs as quickly as possible. Forward-looking statements also include those regarding Sarepta’s future business developments and actions and the timing of the same.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Known risk factors include, among others: the results of our ongoing research and development efforts and clinical trials for eteplirsen and our other product candidates may not be positive or consistent with prior results or demonstrate a safe treatment benefit there may be delays in Sarepta’s projected regulatory and development timelines relating to the eteplirsen NDA and plans for commercializing eteplirsen and developing Sarepta’s other product candidates for various reasons including possible limitations of Sarepta’s financial and other resources; Sarepta may not be able to successfully complete its planned commercialization of eteplirsen or continue developing its product candidates as planned for a variety of reasons including due to regulatory, court or agency decisions, such as decisions by the USPTO with respect to patents that cover Sarepta’s product candidates, scale-up of manufacturing may not be successful, and any or all of Sarepta’s product candidates may fail in development or may not receive required regulatory approvals for commercialization (including potentially under an accelerated pathway); and those risks identified under the heading “Risk Factors” in Sarepta’s 2014 Annual Report on Form 10-K or and most recent Quarterly Report on Form 10-Q for the quarter ended June 30, 2015 filed with the Securities and Exchange Commission (SEC) as well as other SEC filings made by the Company which you are encouraged to review.
Any of the foregoing risks could materially and adversely affect Sarepta’s business, results of operations and the trading price of Sarepta’s common stock. For a detailed description of risks and uncertainties Sarepta faces, you are encouraged to review the Company’s filings with the SEC. We caution investors not to place considerable reliance on the forward-looking statements contained in this press release. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.
Internet Posting of Information
We routinely post information that may be important to investors in the ‘For Investors’ section of our website at www.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.
Source: Sarepta Therapeutics, Inc.
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