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REALIZING THE POTENTIAL OF RNA-BASED
JP MORGAN HEALTHCARE CONFERENCE
FORWARD LOOKING STATEMENTS
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 our use of unique PMOs to develop treatments in areas of high unmet need; our
plans to continue advancing eteplirsen towards regulatory approval and moving follow-on exons into human clinical trials; the potential broad application of our chemistries and technolog ies across a variety of disease areas; our strategic research
focus and plans to continue advancing our chemistry platform and technologies, including our To ll-like Receptor Antagonist Program, into additional indications in rare or other diseases and against antibiotic-resistant bacterial infections,
including through collaborations; our capitalization status; the potential market for eteplirsen and follow-on exon skipping drugs; our plans and ability to comply with the U.S. Food and Drug Administration (FDA) requirements to consider a new drug
application (NDA) submission for eteplirsen complete; our plans and ability to successfully initiate and complete additional clinical trials for eteplirsen and other follow-on product candidates in Duchenne Muscular Dystrophy (DMD) and providing
additional data, analysis and other information requested by the FDA and potential timing of the same; the potential of and timing of an NDA submission by us, which will continue to be evaluated based on FDA discussions and as additional data become
available, and a potential filing and acceptance of an NDA for eteplirsen by the FDA on an accelerated or other pathway; our continuing discussions with the EMA regarding a potential approval pathway in Europe; our beliefs regarding the potential of
and safety and efficacy of our product candidates, chemistries and technologies in DMD, rare and infectious diseases and other disease areas; and the timing of and the expected or planned research, development, clinical and regulatory progress for
our product candida tes. Forward-looking statements also include those made during the presentation regarding future business developments and actions and the timing of the same, including our ability to establish and protect intellectual property
rights and potentially commercialize our product candidates without claims of infringement.
forward-looking statement contained in this presentation is subject to risks and uncertainties that could cause actual resu lts to differ materially from those expressed or implied by such statement. Applicable risks and uncertainties include, among
others: we may not have sufficient funds to execute our business plans; 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 reasons;
we may not be able to comply with all regulatory requests and requirements for the research, dev elopment and commercialization of our product candidates; the FDA may determine that substantial additional data is required for accelerated or other
approv al of eteplirsen or that our potential NDA submission for eteplirsen does not qualify for filing, even with additional information; the results of our ongoing research and development efforts and clinical trials may not be positive or
consistent with prior results or demonstrate a treatment benefit ; there may be delays in timelines or we may not make an NDA submission orsuccessfully initiate, conduct or complete clinical trials, or make eteplirsen or any of our product
candidates, chemistries or technologies commercially available for regulatory or other reasons; we may not be able to manufacture sufficient drug supply for our studies or commercialization; agency or court decisions with respect to our patents or
those of third parties may negatively impact our business; and those risks identified under the heading Risk Factors in Sarepta s Quarterly Report on Form 10-Q for the quarter ended September 30, 2014 filed with the Securities
and Exchange Commission (SEC), 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.
SAREPTA THERAPEUTICS
CORPORATE HIGHLIGHTS
Sarepta is a late-stage development company utilizing RNA-based technology with unique phosphorodiamidate
morpholino oligomers (PMOs) to develop treatments for areas of high unmet need
Priority remains on DMD
advancing eteplirsen towards regulatory approval, moving follow-on exons into human clinical trials
Differentiated PMO chemistries with broad application across variety of disease areas
Significant progress made advancing chemistry platform into additional indications in rare diseases and against
antibiotic-resistant bacterial infections
5 DMD clinical trials underway
More than 10 new research programs underway
2 Chemistries tested in humans (PMO & PMOplus )
Well-capitalized with ~$211 million in cash and other investments as of 12/31/2014
DUCHENNE MUSCULAR DYSTROPHY
DISEASE WITH SIGNIFICANT UNMET NEED
Affects approximately 1 in 3,5001 boys worldwide
25,000- 30,000 patients in the U.S. and Europe
Sarepta s lead program (Exon 51) estimated to target ~13% of DMD patients2
Follow-on Exon-Skipping Drugs have potential to treat 60-80% of DMD patients
National Human Genome Research Institute
of DMD boys amenable to skipping each exon listed. Source: Annemieke Aartsma-Rus, et al. Hum Mutat. 2009 Mar;30(3):293-9.
DMD COMPARED TO BECKER MUSCULAR DYSTROPHY (BMD)
ANY SHIFT FROM DUCHENNE TOWARDS BMD WOULD REPRESENT A MEANINGFUL DIFFERENCE IN PATIENT LIFE SPAN AND QUALITY OF LIFE
BMD is a milder phenotype with a more gradual progression1
The majority of patients with BMD remain ambulant significantly longer than patients living with DMD1
Typically between ages 5-10 as development is slow. Some patients are asymptomatic and diagnosed through abnormally
Patients can often run into 20 s and may retain ability to walk through majority
ECKER of life through use of canes. Loss of ambulation typically occurs during 30 s.
B Lung Function Decline Generally mild. Trouble coughing and some difficulty breathing.
Cardiac Function Decline Generally mild, severe cases can lead to mortality in early 20 s.
Childhood Adolesence Early Adulthood
Adulthood Late Adulthood
1. Muscular Dystrophy Association
DMD GLOBAL CLINICAL AND
168 Week Data Released
All evaluable patients remained ambulant
Continued decline across all patients from week 144
Ongoing stability of respiratory function
NDA still planned for mid-2015
will continue to be evaluated based on FDA discussions and as additional data become available
Received on Dystrophin Reassessment Protocol
Rescoring of Dystrophin-Positive Fibers by 3 Independent
Pathologists Underway
FDA Feedback Received on Master Protocol for Exon 53 and Exon 45 Drugs
Agreement to proceed with primary endpoint of 6MWT in combined population
Placebo-controlled study with SRP-4045 and SRP-4053 to begin dosing patients with 30mg/kg in 1H2015
FDA communicated additional data requirements for NDA submission now planned for mid-year 2015
168-week clinical data from study 201/202
Safety data in newly exposed eteplirsen patients (subset with at least 3-month safety data); Dosing expected in 12-24 pts this month
Results from 4th Biopsy are expected for subset of patients in Study 201/202; Scheduling of biopsies underway
Dystrophin Rescore underway
Patient level natural history data being collected
Meeting in December provided preliminary guidance that additional clinical data will be needed for conditional approval
EMA encouraged continued discussions as additional data is compiled from current and new studies
DMD CLINICAL PROGRAM STATUS (US AND EU):
ETEPLIRSEN AND FOLLOW-ON EXON-SKIPPING DRUGS (EXONS 45 AND 53)
Duration Exon Target
Study US/EU n Status DMD Population
4658-33 Single Dose EU 7 Completed Exon 51 10-17 yrs, non-amb
4658-28 12 EU 19 Completed Exon 51 5-15 yrs, amb
4658-201 28 US 12 Completed Exon 51 7-13 yrs, amb
4658-202 2401 US 12 Data through 168 Weeks Exon 51 7-13 yrs, amb
4658-301 48 US 120 Dosing Exon 51 7-16 yrs, amb
4658-204 96 US 20 Dosing Exon 51 7-21 yrs, non-amb
4658-203 96 US 20 Dosing 1Q2015 Exon 51 4-6 yrs, amb
4053-101 48 EU 36 Dosing Jan 2015 Exon 53 6-16 yrs, amb
4045-301 TBD EU/US 90 TBD Exon 45/53 7-16 yrs, amb
1. 212 weeks in extension phase for a total 201/202 duration of 240 weeks.
SAREPTA S EXON SKIPPING
CLINICAL PROGRAMS DISCOVERY PRE-CLINICAL CLINICAL DEVELOPMENT
DMD Eteplirsen (AVI-4658)
Rare Disease DMD SRP-4044
DMD Clinical Development Status
PROMOVI 4658-301 39 Sites
11 of 14 Hub Sites fully initiated