Europe Approves High-Price Gene Therapy – November 2nd, 2012

7:59am EDT

By Ben Hirschler

LONDON (Reuters) – European officials have approved the Western world’s first gene therapy drug from a small Dutch biotech company, in a milestone for the novel medical technology that fixes faulty genes.

The formal clearance from the European Commission paves the way for a launch next summer of the treatment for an ultra rare genetic disease that will cost around 1.2 million euros ($1.6 million) per patient, a new record for pricey modern medicines.

After more than 20 years of experiments and a series of disappointments, the EU approval of Glybera, which treats the genetic disorder lipoprotein lipase deficiency (LPLD), is a significant boost for the gene therapy field.

Joern Aldag, chief executive of Amsterdam-based uniQure, said more such treatments would follow and argued a high price was justified because gene therapy restored natural body function and did not just offer a short-term fix.

“This provides higher benefit to patients than the classical protein replacement strategy and this is why we think we should be fairly and adequately compensated,” he said in a telephone interview on Friday.

Patients with LPLD, which affects no more than one or two people per million, are unable to handle fat particles in their blood and are at risk of acute and potentially fatal inflammation of the pancreas.

The approval follows a positive recommendation from the European Medicines Agency in July.

The privately owned firm is now working with governments on potential pricing strategies, which are likely to vary from country to country, ahead of the commercial roll-out from the second half of 2013.

Aldag said some countries preferred the idea of a one-off payment at the time of treatment but others were interested in an annuity system, which would probably involve charging around 250,000 euros a year for five years.

That kind of annual charge would put Glybera in a similar price range to expensive enzyme replacement therapies for other rare diseases, such as Cerezyme for Gaucher disease from Sanofi’s Genzyme unit.

UniQure is also preparing to apply for regulatory approval for Glybera in the United States, Canada and other markets.

EARLIER SETBACKS

The idea of treating disease by replacing a defective gene with a working copy gained credence in 1990 with the success of the world’s first gene therapy clinical tests against a rare condition called severe combined immunodeficiency (SCID).

People with SCID – also known as “bubble boy disease” – cannot cope with infections and usually die in childhood.

The field then suffered a major setback when an Arizona teenager died in a gene therapy experiment in 1999 and two French boys with SCID developed leukaemia in 2002.

In China, Shenzhen SiBiono GeneTech won approval for a gene therapy drug for head and neck cancer in 2003 but no products have been approved until now in Europe or the United States.

More recently, some large pharmaceutical companies have also been exploring gene therapy. GlaxoSmithKline, for example, signed a deal in 2010 with Italian researchers to develop a SCID therapy. ($1 = 0.7730 euros)

(Reporting by Ben Hirschler; Editing by Helen Massy-Beresford)

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Prosensa Announces Progress on Exon Skipping Compounds for the Treatment of Duchenne Muscular Dystrophy – October 24th, 2012

£10m in milestone payments received from GlaxoSmithKline

Leiden, The Netherlands, 23 October 2012 – Prosensa, the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, has selected clinical candidates for two more compounds for the treatment of Duchenne muscular dystrophy (DMD) and has been granted orphan drug designation for two additional DMD compounds in its pipeline.

 Prosensa identified suitable oligonucleotide candidates for PRO052 and PRO055, designed for the skipping of exons 52 and 55 of the dystrophin gene. PRO052 and PRO055 are currently in pre-clinical development and will be moved to clinical trials as soon as possible.

 Prosensa has also received orphan drug designation by the European Commission for PRO045 and PRO053. These compounds are designed to skip exons 45 and 53 of the dystrophin gene and are expected to enter clinical development within the next 6 months. In parallel, Prosensa and GSK have initiated a large global natural history study in order to generate important data on the progress of DMD that will help to facilitate the development pathways for these compounds.

 Together with drisapersen (PRO051/GSK2402968) and PRO044, already in clinical development, the Prosensa portfolio of six DMD compounds has the potential to treat more than 40% of all DMD patients.

 Hans Schikan, CEO of Prosensa, commented: “Achieving orphan drug status for PRO045 and PRO053 is an important milestone that we believe will allow us to accelerate development of these compounds and initiate clinical trials within the next half year. While GSK has an option to license one compound, PRO045 or PRO053, the other compound will be developed and eventually commercialized by Prosensa in our ambition to become a fully integrated biopharmaceutical player in rare diseases. Together with PRO052 and PRO055, where the same principle applies, Prosensa will be in a position to target rare mutations in specific DMD subpopulations and may provide treatment to more patients with this debilitating disease.”

 Prosensa has the most advanced portfolio of drug candidates for the treatment of DMD, and has also preclinical compounds for Myotonic Dystrophy and Huntington’s Disease. Prosensa’s DMD compounds are based on its proprietary exon-skipping technology that uses antisense oligonucleotides to restore expression of a functional dystrophin protein and to provide potential treatment for patients affected by this progressively debilitating neuromuscular disease.

—ENDS—

 

Notes to editors:

 

About DMD

Duchenne muscular dystrophy (DMD) is a severely debilitating childhood neuromuscular disease that affects 1 in 3,500 live male births. This rare disease is caused by mutations in the dystrophin gene, resulting in the absence or defect of the dystrophin protein. As a result, patients suffer from progressive loss of muscle strength, often rendering them wheelchair-bound before the age of 12. Respiratory and cardiac muscle can also be affected by the disease and most patients die in early adulthood due to respiratory and cardiac failure.

About exon skipping

The dystrophin gene is the largest gene in the body, consisting of 79 exons. Exons are small sequences of genetic code which, via an intermediate step involving RNA, lead to the assembly of sections of protein. In DMD, when certain exons are mutated/deleted, the RNA cannot read past the fault. This prevents the remainder of the exons from being read, resulting in a non-functional dystrophin protein and the severe symptoms of DMD. RNA-based therapeutics, specifically antisense oligonucleotides inducing exon skipping, are currently in development for DMD. These antisense oligonucleotides skip an exon next to a defective exon and thereby correct the reading frame, enabling the production of a novel, functional dystrophin protein. Prosensa’s exon skipping technology originated in Leiden University Medical Center.

About Prosensa

Prosensa is an innovative Dutch biopharmaceutical company focused on the discovery, development and commercialization of RNA-modulating therapeutics correcting gene expression in diseases with significant unmet need, in particular neuromuscular disorders. Prosensa’s current focus is on developing treatments for Duchenne muscular dystrophy (DMD), Myotonic Dystrophy and Huntington’s disease. In 2009 Prosensa entered into a strategic alliance for part of its DMD exon skipping program with GlaxoSmithKline. Prosensa’s lead compound (drisapersen), being developed by GSK, is currently in phase III clinical trials. Prosensa is a privately held biopharmaceutical company, backed by a consortium of Abingworth, Gimv, Idinvest Partners, Life Sciences Partners, MedSciences Capital and New Enterprise Associates. For more information, please visit www.prosensa.com.

 

Prosensa won the 2012 Emerging Star Award at the European Mediscience Awards and was listed as a Fierce 15 Biotech Company.

 

 

For more information please contact:

 

Prosensa enquiries:

Luc Dochez          +31 71 332 2085

Hans Schikan       +31 71 332 2100

Media enquiries:

College Hill Life Sciences

Anastasios Koutsos / Henry Stanley

+44 20 7457 2020

prosensa@collegehill.com

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Let’s Not Stop Until We Cure Duchenne! – October 16th, 2012

There has been so much news about Duchenne in the last month…and it’s good news.  Next month will be the 10th anniversary of our son’s diagnosis of this dreadful disease.  At that time, we searched the internet and found just a few research projects that were in early stages.  Today, we have Glaxo Smith Kline/Prosensa with a drug in a phase III pivotal trial around the world, Sarepta Therapeutics has shown remarkable results in their phase II trial and the Duchenne community is mobilizing to address the FDA with our need for rapid approval for these potential life saving drugs.

Today, Cincinnati Children’s Medical Center has announced what is believed to be the first in the nation to have a device implanted into a Duchenne patient to help his heart pump blood to the body long-term.  Cardiac failure is a leading cause of death in Duchenne…this could be a major advance, although it’s very new and we must be cautious.

CureDuchenne was early in it’s support of exon skipping, and we are actively involved in working with the biotech/pharmaceutical companies and the FDA to get these drugs to all patients as soon as possible.  The news from CCMC today gives us hope that cardiac issues can be treated as we hopefully improve the skeletal muscle strength in Duchenne boys.

Now is the time to double down with our efforts to bring treatments to all of our sons.  Please know that we need everyones help, cooperation and enthusiasm.  Everyone has something to contribute.  Finally, the science is ready for us to be bold, please help us to turn the science into treatments.

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Sarepta Therapeutics Announces Eteplirsen Meets Primary Endpoint of Increased Novel Dystrophin and Achieves Significant Clinical Benefit on 6-Minute Walk Test After 48 Weeks of Treatment in Phase IIb Study in Duchenne Muscular Dystrophy – October 3rd, 2012

Eteplirsen Results in an Increase in Novel Dystrophin to 47% of Normal After 48 Weeks of Treatment; Eteplirsen 50mg/kg Weekly Demonstrates Continued Clinical Benefit of 89.4 Meters in 6-Minute Walk Test Over Placebo/Delayed Treatment Cohort; No Eteplirsen-Related Adverse Events Through Week 48

CAMBRIDGE, MA–(Marketwire – October 03, 2012) – Sarepta Therapeutics (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced that treatment with its lead exon-skipping compound, eteplirsen, met the primary efficacy endpoint, increase in novel dystrophin, and achieved a significant clinical benefit on the primary clinical outcome, the 6-minute walk test (6MWT) over the placebo/delayed treatment cohort in a Phase IIb extension trial in Duchenne muscular dystrophy (DMD) patients.

Eteplirsen administered once weekly at either 30 mg/kg or 50 mg/kg for 48 weeks (n=8) resulted in a statistically significant increase (p≤0.001) in dystrophin-positive fibers to 47.0% of normal. The placebo/delayed treatment cohort, which had received 24 weeks of eteplirsen at either 30 mg/kg or 50 mg/kg following 24 weeks of placebo (n=4), also showed a statistically significant increase in dystrophin-positive fibers to 38.3% of normal (p≤0.009).

“These data represent a significant milestone and a defining moment of progress and hope for patients with DMD and their families, as well as for those of us in the scientific community who have been pursuing potential treatments for this devastating and deadly disease for decades,” said Jerry Mendell, M.D., Director of the Centers for Gene Therapy and Muscular Dystrophy at Nationwide Children’s Hospital and principal investigator of the Phase IIb study. Dr. Mendell added, “By addressing the underlying cause of DMD, eteplirsen has demonstrated unparalleled effects on enabling dystrophin production and slowing the progression of the disease as measured by the 6-minute walk test, with no treatment associated adverse events. While eteplirsen is targeted to DMD patients with a specific genetic mutation, I think the implications for all DMD patients with related genetic mutations are clearly evident.”

Eteplirsen administered once weekly at 50 mg/kg over 48 weeks resulted in an 89.4 meter benefit compared to patients who received placebo for 24 weeks followed by 24 weeks of treatment with eteplirsen in the open-label extension. In the predefined prospective analysis of the study’s intent-to-treat (ITT) population on the primary clinical outcome measure, the change in 6MWT distance from baseline, eteplirsen-treated patients who received 50 mg/kg of the drug weekly (n=4) demonstrated an increase of 21.0 meters in distance walked from baseline (mean=396.0 meters), while patients who received placebo/delayed-eteplirsen treatment (n=4) showed a decline of 68.4 meters from baseline (mean=394.5 meters), for a statistically significant treatment benefit of 89.4 meters over 48 weeks (p=0.016, using ANCOVA for ranked data). There was no statistically significant difference between the cohort of patients who received 30 mg/kg weekly of eteplirsen and the placebo/delayed treatment cohort.

“We are extremely excited about these data, as they demonstrate that longer-term treatment with eteplirsen is translating to continued and unprecedented increases in both dystrophin production and clinical benefit across various subgroups of DMD patients involved in this study,” said Chris Garabedian, President and CEO of Sarepta Therapeutics. “On a broader scale, these results signify the promise and tremendous potential of our RNA-based technology to impact and modulate disease at the genetic level, which may lead to first-ever opportunities to target serious and life-threatening rare conditions at the origin of disease.”

The safety profile of eteplirsen was evaluated across all subjects through 48 weeks and there were no treatment-related adverse events, no serious adverse events, and no discontinuations. Furthermore, no clinically significant treatment-related changes were detected on any safety laboratory parameters, including several biomarkers for renal function.

Summary of Dystrophin: Eteplirsen-Treated Patients in All Dose Groups through Week 48*



----------------------------------------------------------------------------
Treatment Arm                                Mean Change from   p-value
                                             Baseline in %
                                             Dystrophin-
                                             Positive Fibers
----------------------------------------------------------------------------
Eterplirsen (both doses): 48 wks of Tx (n=8) 47.0               ≤0.001
  Eteplirsen 50 mg/kg (n=4)                  41.7               ≤0.008
  Eteplirsen 30 mg/kg (n=4)                  52.1               ≤0.001
Placebo/Delayed Tx: 24 wks of Tx (n=4)       38.3               ≤0.009
  Placebo/50 mg/kg Delayed-Tx (n=2)          42.9               ns
  Placebo/30 mg/kg Delayed-Tx (n=2)          34.2               ns

----------------------------------------------------------------------------

* Values based on Immunofluorescence using anti-dystrophin antibody MANDYS106

Modified Intent-to-Treat (mITT)

The 6MWT results were further analyzed using the mITT population which excluded two patients who were randomized to the 30 mg/kg weekly eteplirsen cohort who showed signs of rapid disease progression within weeks after enrollment and were unable to perform measures of ambulation beyond 24 weeks. This mITT population consisted of 10 patients (4 eteplirsen-treated patients receiving 50 mg/kg weekly, 2 eteplirsen-treated patients receiving 30 mg/kg weekly, and 4 placebo/delayed-treatment patients).

Summary of 6MWT: Eteplirsen versus Placebo/Delayed-Treatment to Week 48*



----------------------------------------------------------------------------
Treatment Arm             Mean Change from   Estimated          p-value
                          Baseline in 6MWT   Treatment Effect
                          (meters)           (Eteplirsen minus
                                             Placebo/Delayed-
                                             Tx)
----------------------------------------------------------------------------
Placebo/Delayed-Tx (n=4)  -60.3
Eteplirsen 50 mg/kg (n=4) +27.1              87.4 m             ≤0.001
Eteplirsen Both Doses     +7.3               67.3 m             ≤0.001
 (n=6)
Eteplirsen 30 mg/kg (n=2) -31.5              28.8 m             ns
----------------------------------------------------------------------------

*Note: Analysis based on Mixed Model Repeated Measures test

Summary of Additional Sub-Group Analyses at Week 48*



----------------------------------------------------------------------------
Subset                    Mean 6MWT Change   Estimated          p-value
                          from Baseline      Treatment Benefit
                          (meters)           (Eteplirsen minus
                                             Placebo/delayed-
                                             Tx)
----------------------------------------------------------------------------

----------------------------------------------------------------------------
Placebo/delayed Tx:       -42.3              58.9 m             ≤0.038
< 9.5 yrs at baseline
(n=2; mean=7.6 yrs)
--------------------------------------------
Eteplirsen:               +16.5
< 9.5 yrs at baseline
(n=3; mean=8.4 yrs)
----------------------------------------------------------------------------
Placebo/delayed Tx:       -63.5              52.1 m             ns
≥9.5 yrs at
 baseline
(n=2; mean=10.1 yrs)
--------------------------------------------
Eteplirsen:               -11.3
≥9.5 yrs at
 baseline
(n=3; mean=10.4 yrs)
----------------------------------------------------------------------------
Placebo/delayed Tx:       -53.5              93.8 m             ≤0.001
Higher 6MWT baseline
(n=2; mean=422m)
--------------------------------------------
Eteplirsen:               +40.3
Higher 6MWT baseline
(n=3; mean=424m)
----------------------------------------------------------------------------
Placebo/delayed Tx:       -65.8              39.6 m             ns
Lower 6MWT baseline
(n=2; mean=367m)
--------------------------------------------
Eteplirsen:               -26.2
Lower 6MWT baseline
(n=3; mean=375m)
----------------------------------------------------------------------------
Placebo/delayed Tx:       -69.0              83.4 m             ≤0.001
Genotype 49-50 deletion
(n=3; age mean=9.2 yrs,
6MWT BL mean=397m)
--------------------------------------------
Eteplirsen:               +14.4
Genotype 49-50 deletion
(n=2; age mean=9.1 yrs,
6MWT BL mean=383m)
----------------------------------------------------------------------------

* Note: Analysis based on Mixed Model Repeated Measures test

An abstract describing the results from this Phase IIb extension study has been accepted as part of the World Muscle Society (WMS) Congress’s Late-Breaking Science program in Perth, Australia during October 9 to October 13, 2012. Principal investigator, Jerry R. Mendell, M.D. of Nationwide Children’s Hospital, will present the data via an oral presentation of the abstract titled, “Results at 48 Weeks of a Phase IIb Extension Study of the Exon-Skipping Drug Eteplirsen in Patients with Duchenne muscular dystrophy (DMD).” Dr. Mendell will present on October 13 at 4:00 p.m. WST UTC +8 hours/4:00 a.m. EDT. Dr. Mendell’s presentation will be posted on the Sarepta website in the “Events & Presentations” section after the session is completed. In addition, Sarepta is sponsoring an educational symposium at WMS chaired by Professor Steve Wilton, PhD, Head of the Molecular Genetic Therapy Group and Director of Translational Research and Development, Australian Neuromuscular Research Institute at the University of Western Australia. Professor Wilton is a long-time collaborator of Sarepta’s whose groundbreaking research has extended the use of antisense oligomers to DMD.

About Study 201 and Study 202 (Phase IIb Eteplirsen Study)

Study 4658-US-201 was conducted at Nationwide Children’s Hospital in Columbus, Ohio. Twelve boys meeting the inclusion criteria being between 7 and 13 years of age with appropriate deletions of the dystrophin gene that confirm eligibility for treatment with an exon-51 skipping drug, received double-blind IV infusions of placebo (n=4), 30 mg/kg of eteplirsen (n=4), or 50 mg/kg of eteplirsen once weekly for 24 weeks (n=4). Muscle biopsies for evaluation of dystrophin were obtained at baseline for all subjects, and after 12 weeks for patients in the 50 mg/kg cohort and after 24 weeks for patients in the 30 mg/kg cohort. Two placebo patients were randomized to the 30 mg/kg cohort and two placebo patients were randomized to the 50 mg/kg cohort. This study design allowed Sarepta to investigate the relationship of dose and duration of eteplirsen treatment on the production of dystrophin over the course of the 24-week study.

Study 4658-US-202 is the extension study to 201 and continues to assess the long-term safety and efficacy of open-label eteplirsen. The four placebo patients were rolled over to open-label eteplirsen at week 24, with six patients on 30 mgs/kg, and six patients on 50 mgs/kg. Third biopsies occurred at 48 weeks in the original study 201 treated patients, and at 24 weeks, the same time point, in the original placebo patients. 6MWT was performed at 32 weeks, 36 weeks, 48 weeks and will continue to be performed every 12 weeks going forward.

About Dystrophin

Dystrophin, a large structural protein, is critical to the stability of myofiber membranes in skeletal, diaphragmatic and cardiac muscle, protecting muscle fibers from contraction-induced damage. Loss of functional dystrophin destabilizes the dystroglycan protein complex, impairing its localization to the muscle membrane, and compromising the integrity of the membrane structure. The absence of functional dystrophin results in muscle membrane breakdown with muscle fibers being replaced by adipose and fibrotic tissue.

About the 6-Minute Walk Test

The 6-minute walk test (6MWT) was developed as an integrated assessment of cardiac, respiratory, circulatory, and muscular capacity (American Thoracic Society 2002) 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 (Rubin 2002, Wraith 2004, Muenzer 2006). 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 (McDonald C, et al, Muscle & Nerve, December 2010) 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 the Statistical Methodology

The Mixed Model Repeated Measures (MMRM) test was used for all statistical analyses of the 6MWT results, including for all subgroups. Analysis of Covariance (ANCOVA) for ranked data was used when the assumptions of normality of the dependent variable (the change in 6MWT distance from baseline) were violated. The inclusion of the two patients with extreme scores due to rapid progression in the ITT population (n=12) resulted in a violation of the normality assumptions of the Change from Baseline in 6MWT data, and thus required the use of ANCOVA for ranked data. The exclusion of these two patients from the mITT population (n=10) resulted in the 6MWT data becoming normally distributed and the MMRM statistics exhibiting much improved residuals and fit statistics as compared to the ITT population. As such, the estimated mean values and their associated p-values for the mITT population were slightly different from those for the ITT population.

Conference Call and Slide Show

The Company will hold a conference call and broadcast a slide show today at 8:00 a.m. EDT (5:00 a.m. PDT) to discuss these results. The audio conference call may be accessed by dialing 866.356.3093 for domestic callers and 617.597.5381 for international callers. The passcode for the call is 93880948. Please specify to the operator that you would like to join the “Sarepta Therapeutics 48-Week Results Call.” To view the slide show while using the audio dial-in please go to the events section of Sarepta’s website at www.sareptatherapeutics.com. The call and slide show will also be webcast live under the events section and will be archived there following the call 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. An audio replay will be available through October 10, 2012 by calling 888.286.8010 or 617.801.6888 and entering access code 67898748.

About Duchenne Muscular Dystrophy and Eteplirsen

Duchenne muscular dystrophy (DMD) is an X-linked rare, degenerative neuromuscular disorder causing severe, progressive muscle loss and a premature death. One of the most common fatal genetic disorders, DMD affects approximately one in every 3,500 boys 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 eventually spreads to the arms, neck and other areas. Eventually, this progresses to complete paralysis and increasing difficulty in breathing due to respiratory muscle dysfunction requiring ventilatory support, as well as cardiac muscle dysfunction leading to heart failure. The condition is terminal, and death usually occurs before the age of 30.

Eteplirsen is Sarepta’s lead drug candidate that is designed to address the underlying cause of DMD by enabling the production of a functional dystrophin protein. Data from clinical studies of eteplirsen in DMD patients have demonstrated a broadly favorable safety and tolerability profile and restoration of dystrophin protein expression. Eteplirsen uses Sarepta’s novel phosphorodiamidate morpholino oligomer (PMO)-based chemistry and proprietary exon-skipping technology to skip exon 51 of the dystrophin gene enabling the repair of specific genetic mutations that affect approximately 13 percent of the total DMD population. By skipping exon 51, eteplirsen may restore the gene’s ability to make a shorter, but still functional, form of dystrophin from messenger RNA, or mRNA. Promoting the synthesis of a truncated dystrophin protein is intended to improve, stabilize or significantly slow the disease process and prolong and improve the quality of life for patients with DMD.

Sarepta is also developing other PMO-based exon-skipping drug candidates intended to treat additional patients with DMD.

About Sarepta Therapeutics

Sarepta Therapeutics is focused on developing first-in-class RNA-based therapeutics to improve and save the lives of people affected by serious and life-threatening rare and infectious diseases. The Company’s diverse pipeline includes its lead program eteplirsen, for Duchenne muscular dystrophy, as well as potential treatments for some of the world’s most lethal infectious diseases. Sarepta aims to build a leading, independent biotech company dedicated to translating its RNA-based science into transformational therapeutics for patients who face significant unmet medical needs. For more information, please visit us at www.sareptatherapeutics.com.

Forward-Looking Statements and Information

In order to provide Sarepta’s investors with an understanding of its current results and future prospects, this press release contains statements that are forward-looking. 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 about the development of eteplirsen and its efficacy, potency and utility in the treatment of DMD and the potential for the creation of novel dystrophin to lead to significant clinical benefit over a longer course of treatment.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Known risk factors include, among others: clinical trials may not demonstrate the safety and efficacy of eteplirsen and/or Sarepta’s antisense-based technology platform; treatment of patients with DMD using eteplirsen over a longer duration may not lead to significant clinical benefit; and any of Sarepta’s drug candidates, including eteplirsen, may fail in development, may not receive required regulatory approvals, or be delayed to a point where they do not become commercially viable.

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 Securities and Exchange Commission. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.
Sarepta Investor and Media Contact:
Erin Cox
425.354.5140
ecox@sareptatherapeutics.com

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VECTTOR Clinical Trial Announcement – September 26th, 2012

Defying Muscular Dystrophy Foundation and the Hope for Gabe Foundation will be sponsoring a double-blind, randomized, placebo-controlled trial of VECTTOR therapy for Duchenne Muscular Dystrophy in Houston, Texas, later this year. This trial will be held in cooperation with Alan Neuromedical Technologies and Dr. Donald A. Rhodes.

What is VECTTOR therapy?

VECTTOR therapy is a form of electrostimulation based upon acupuncture, reflexology, physiology, cellular physiology, and anatomy designed to stimulate the nerves to produce certain vital neuropeptides essential for optimal functioning of the body. VECTTOR therapy is non-invasive and the treatments are not painful. There are no known negative side-effects from VECTTOR therapy. However, the long-term use of electrostimulation has not been studied.

Who is eligible for this study?

The study will include up to 12 individuals with Duchenne Muscular Dystrophy, who are 20 years old or younger and who are non-ambulatory. Further screening criteria may include steroid usage, previous study participation, and prior muscle biopsies. The exclusion criterion includes individuals with a demand type cardiac pacemaker.

What will happen during this study?

Each participant will be randomly assigned either an active or a placebo VECTTOR system. The systems are visually indistinguishable but only the active unit will administer electrostimulation. Each study participant and/or his caretaker will be trained to properly administer the VECTTOR treatments and will be able to do so in their own home. Support staff will be available for questions or concerns throughout the duration of the study.

How long with this study last?

The study will run for six months with a possible six month extension. For the first six months, the participants will be randomly divided into two equal, blinded groups. The first group will receive active VECTTOR treatments; the second group will receive inactive (placebo) treatments. The participants will receive various testing at weeks: 0, 4, 9, 13, and 26. At the end of the six month period, if the testing shows improvement in those participants receiving active treatment; all participants will receive active treatment for the 6 months extension and all testing will be repeated at the end of the second six months.

What will be required of the participants?

Each participant will be required to report to the testing center(s) in or around the greater Houston area at least 6 times, prior to, and throughout the duration of the study. Participants will be expected to administer VECTTOR therapy treatments twice per day for the first month and at least once per day for the remainder of the study. The units are equipped with tracking software to demonstrate compliancy and to collect treatment data. Treatment data will be collected on an SD card installed in the VECTTOR system which will be downloaded, by the participants, to the study coordinator.

Where will the study take place?

All testing will be done in or around the greater Houston area.

Is there payment for participation?

There is no payment for participation, nor reimbursement for travel expenses. However, participants will be provided with all equipment, supplies and testing at no cost to them.

What testing will be performed?

Muscle strength testing and joint range of motion testing will be performed prior to beginning VECTTOR therapy and again at weeks: 4, 9, 13, and 26. If there is an extension phase of the study, the same testing will be repeated at the end of the second six months. The presence of Deep Tendon Reflexes will be assessed prior to beginning treatment and again at weeks: 4, 9, 13, and 26. Provided there is a study extension, this test will be repeated at the end of the second six months. Bone mineral densities will be obtained prior to beginning treatment and again at 26 weeks. Provided there is a study extension, this test will be repeated at the end of the second six months. Physiological sleep studies will be obtained twice before beginning the study. The second study will be used as the baseline test. A follow up study will be obtained at week 26. If there is a study extension, this test will be repeated at the end of the second six months. Echocardiograms will be obtained prior to beginning treatments, and again at week 26. Provided there is a study extension, this test will be repeated at the end of the second six months. Pulmonary function testing will be obtained prior to beginning treatment and again at week 13 and week 26. Provided there is a study extension, this test will be repeated at the end of the second six months.

Eligible participants should contact VECTTORtrial@gmail.com for more information.

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An open letter from Chris Garabedian, president and CEO of Sarepta Therapeutics – August 28th, 2012

August 28, 2012

On behalf of everyone at Sarepta Therapeutics, I wanted to provide you with an update on our clinical program for eteplirsen.

As many of you are aware, on July 24th we released interim data from our ongoing Phase IIb clinical trial of eteplirsen, which provided the first indication that eteplirsen may be having a favorable clinical impact in slowing the progression of the disease for a group of DMD patients with a genetic mutation that is addressable by exon 51 skipping.

Since that time, we have received numerous calls and emails for more information on the program, and especially, about the potential availability of eteplirsen outside of the current clinical trial. We wish to be as transparent as possible about what we are doing to bring eteplirsen to patients who would benefit from the therapy. We know that drug development is a painfully drawn-out process for those who are waiting, and we take seriously our responsibility to act as a true partner to the DMD community as we move forward together.

Since the moment that we saw the interim results last month, we have been working internally and with outside experts to determine the fastest path forward to make this drug more widely available. To do so, our primary focus as a company must be on completing the ongoing clinical trial and demonstrating the safety and efficacy of eteplirsen in a rigorous and convincing way that allows us to initiate discussions with regulators regarding our path forward to secure approval.

In October, we will be able to review and assess the Phase IIb 48-week data. Depending on the results, we will request a meeting with the FDA to determine what next steps will be required. Those discussions will determine what additional clinical trial or trials are needed, what the design of future trials will be, how many patients will participate, and what the makeup of the trial population will be.

At the same time, we must begin to scale up manufacturing of eteplirsen to support future clinical development beyond the current ongoing 12-patient trial. We are currently producing eteplirsen at a scale that is sufficient to meet the needs of the 12 patients in this study. We cannot produce enough eteplirsen to meet needs outside of this trial until we have substantially scaled up our production from current levels.

It is important to stress that eteplirsen is a truly novel type of medicine; the complexity of ramping up production on a larger scale will require a substantial investment of time, money, and expertise in order to demonstrate to regulators that eteplirsen produced at a larger scale has the same characteristics and safety profile as what is currently being used in clinical trials. We have a plan in place to meet this challenge, but we must be thorough and methodical to ensure we are successful.

At the present time, we do not have excess drug supply to make eteplirsen available outside of a clinical trial setting on a compassionate use basis. We can only imagine how difficult this is to hear, but unfortunately we have no other options available to us right now. We must devote our resources to producing enough eteplirsen for our current and future clinical studies so that we can advance this therapy through the regulatory process and, ultimately, secure approval for all DMD patients who would benefit.

We fully understand that for the patients and families impacted by DMD, the pace of drug development can never be fast enough. We pledge to continue to advance eteplirsen through the clinical development process as rapidly and responsibly as possible, and to regularly update the DMD community on our progress to bring this promising therapy to the numerous children and young adults with DMD who so desperately need and deserve a safe and effective treatment option.

Thank you for your consideration.

Sincerely,

Chris Garabedian

President and CEO

Sarepta Therapeutics

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GSK Phase III Exon Skipping Trial Has Completed Recruitment – August 10th, 2012

Study DMD114044: A phase III, randomized, double blind, placebo-controlled clinical study to assess the efficacy and safety of GSK2402968 in subjects with Duchenne muscular dystrophy

The study is expected to complete in July 2013, with study results expected in late 2013.  The results will be presented at the first appropriate medical conference.  Note that GSK2402968 will be referred to by its generic name ‘drisapersen’ in future communications.

Sincerely,

John E. Kraus, MD, PhD                                Padraig Wright, MD, PhD

john.e.kraus@gsk.com                                 padraig.x.wright@gsk.com

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Research Study for Males with Duchenne Muscular Dystrophy – August 8th, 2012

Early Treatment of Cardiomyopathy in DMD

 

Frequently Asked Questions (FAQs) About   This Study

 

What is the purpose of the study?

  • To determine if eplerenone, a type of medication typically used for advanced heart failure patients, in combination with another medicine (ACE-I or ARB) can prevent heart muscle damage in people with DMD.

Who is eligible for the study?

  • If you are a male with DMD, at least 7 years old and:
    • no kidney problems
    • not on eplerenone or other drugs like it
    • can undergo a cardiac MRI scan without sedation and follow breathhold instructions
    • have preserved left ventricular systolic function

How long will this study last?

  • 12 months

  How many study visits are there?

  • After the initial (baseline) screening visit, there are five study visits during a 12-month period.
  • The study visits will happen during months one, three, six, nine and 12.

Where do the study visits take place?

  • The baseline visit will take place either at The Ohio State University (OSU) in Columbus, OH or Cincinnati Children’s in Cincinnati, OH.
  • Blood draw at 1, 3 and 9 months can be done at a local laboratory where you reside.
  • Visits 6 and 12 months are done at the same site where you did your baseline visit.

What will happen during this study?

  • Participants will be randomly assigned to receive either eplerenone (the study drug) or a placebo. A placebo is an inactive substance that looks like the study drug, but contains no medication.
  • Participants will take assigned drug every other day for one month.
  • If after one month, levels of potassium and creatinine (marker of kidney function) are normal, participants will continue to take the drug once daily for the remainder of the study.
  • All participants will have blood drawn at the baseline visit as well as during months 1, 3, 6, 9 and 12.
  • All participants will have a cardiac MRI scan at the baseline, 6 and 12 months.

 Is there payment for participation?

  • There is no payment for participating in the study but you will be reimbursed for travel, food and lodging expenses upon completion of the 6 month visit.
  • The blood draws during months 1, 3, 6 and 9 will be paid for by the study.
  • The cardiac MRI scan at the 6 and 12 month visit will be paid for by the study.
  • The study drug will be provided for free.

Will there be access to the drug once the study has ended?

  • If the results are promising, your physician may choose to prescribe this medication to you or your child after the study has ended.

 Why should I consider participating in this study?

  • While no personal benefit can ever be guaranteed, there are other benefits to participating in a research study. You may be able to:
    • play an active role in your own health care (or that of your child)
    • gain access to new research treatments before they are widely available
    • have access to medical specialists who may not normally be available

and/or

  • help others by contributing to the better understanding and treatment of DMD-associated heart disease.

Where can I learn more about this study?

  • You can learn more about this study at PPMD.com and/or ClinicalTrials.gov.
  •  You may also contact:

Dr. Subha Raman or Beth McCarthy at The Ohio State University, (614) 688-8020 or raman.1@osu.edu

Dr. Kan Hor or Lani Relucio at Cincinnati Children’s, (513)803-1910 or kan.hor@cchmc.org

 

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Sarepta Therapeutics Announces Significant Clinical Benefit With Eteplirsen After 36 Weeks in Phase IIb Study for the Treatment of Duchenne Muscular Dystrophy – July 24th, 2012

Eteplirsen Treatment Results in Benefit of 69 Meters in 6-Minute Walk Test Over Placebo/Delayed Treatment Cohort; No Eteplirsen-Related Adverse Events Through Week 36

CAMBRIDGE, MA–(Marketwire – July 24, 2012) – Sarepta Therapeutics (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, today announced that treatment with its exon-skipping compound, eteplirsen, achieved a significant clinical benefit on the primary clinical outcome, the 6-minute walk test (6MWT), over a placebo/delayed treatment cohort in a Phase IIb trial in Duchenne muscular dystrophy (DMD) patients. Eteplirsen administered once weekly at 50mg/kg over 36 weeks resulted in a 69.4 meter benefit compared to patients who received placebo for 24 weeks followed by 12 weeks of treatment with eteplirsen in the open-label extension. In the predefined prospective analysis of the study’s intent-to-treat (ITT) population on the primary clinical outcome measure, the change in 6MWT distance from baseline, eteplirsen-treated patients who received 50mg/kg of the drug weekly (n=4) demonstrated a decline of 8.7 meters in distance walked from baseline (mean=396.0 meters), while patients who received placebo/delayed-eteplirsen treatment for 36 weeks (n=4) showed a decline of 78.0 meters from baseline (mean=394.5 meters), for a statistically significant treatment benefit of 69.4 meters over 36 weeks (p≤0.019).

“The magnitude of this clinical benefit is an unprecedented treatment effect in DMD. This result represents a major advance in the pursuit of a disease modifying treatment for this severe, progressive and life-threatening disease,” said Jerry Mendell, M.D., Director of the Centers for Gene Therapy and Muscular Dystrophy at Nationwide Children’s Hospital and principal investigator of the Phase IIb study. Dr. Mendell added, “The 6-minute walk test results with eteplirsen, combined with its safety profile to date, make eteplirsen the most promising advance to treat the underlying cause of muscular dystrophy I’ve seen in my more than 30 years in the field.”

The clinical benefit observed in the 50mg/kg treatment cohort compared to placebo was also significant at week 32 with a benefit of 59.9 meters (p≤0.045). The safety profile of eteplirsen was evaluated across all subjects through 36 weeks and there were no treatment-related adverse events, no serious adverse events and no discontinuations. Furthermore, no treatment related changes were detected on any safety laboratory parameters, including several biomarkers for renal function.

“These data suggest that the previously reported levels of dystrophin we observed in muscle biopsies after 24 weeks of treatment are translating to a clinical benefit on the standard measure of ambulation in DMD patients,” said Chris Garabedian, President and CEO of Sarepta Therapeutics. “The magnitude of this 69.4-meter difference after 36 weeks of treatment and the robustness of the statistical analysis is encouraging, especially given the average benefit in the 6-minute walk test for several approved drugs in other diseases has been 30 to 40 meters.”

There was no statistically significant difference between the cohort of patients who received 30mg/kg weekly of eteplirsen and the placebo/delayed treatment cohort.

Modified Intent-to-Treat and Subgroup Analyses

A modified intent-to-treat (mITT) population was evaluated that excluded two patients who were randomized to the 30mg/kg weekly eteplirsen cohort who showed signs of rapid disease progression within weeks after enrollment and were unable to perform measures of ambulation beyond 24 weeks. This mITT population consisting of 10 patients (4 eteplirsen-treated patients receiving 50mg/kg weekly, 2 eteplirsen-treated patients receiving 30mg/kg weekly, and 4 placebo/delayed-treatment patients) was further analyzed.

Summary of 6MWT: Eteplirsen versus Placebo/Delayed-treatment through Week 36*


----------------------------------------------------------------------------
Treatment Arm             Mean 6MWT Change     Estimated Treatment  p-value
                          from Baseline        Effect (Eteplirsen
                          (meters)             minus
                                               Placebo/Delayed-Tx)
----------------------------------------------------------------------------
Placebo/Delayed-Tx (n=4)  -70.9
Eteplirsen 50 mg/kg (n=4) -5.2                 65.8 meters          0.0002
Eteplirsen Both Doses
(n=6)                     -14.6                56.2 meters          0.0004
Eteplirsen 30 mg/kg (n=2) -33.3                37.6 meters          ns
----------------------------------------------------------------------------

*Note: Analysis based on Mixed Model Repeated Measures test

Summary of Additional Sub-Group Analyses at Week 36**


----------------------------------------------------------------------------
Subset                    Mean 6MWT Change     Estimated Treatment  p-value
                          from Baseline        Benefit (Eteplirsen
                          (meters)             minus
                                               Placebo/delayed-Tx)
----------------------------------------------------------------------------

----------------------------------------------------------------------------
Placebo/delayed Tx:       -60.6                63.6 meters          0.0040
 < 9.5 yrs at baseline
(n=2; mean=7.6 yrs)
----------------------------------------------
Eteplirsen:               +3.1
 < 9.5 yrs at baseline
(n=3; mean=8.4 yrs)
----------------------------------------------------------------------------
Placebo/delayed Tx:       -73.4                36.0  meters         ns
≥9.5 yrs at
baseline
(n=2; mean=10.1 yrs)
----------------------------------------------
Eteplirsen:               -37.4
≥9.5 yrs at
baseline
(n=3; mean=10.4 yrs)
----------------------------------------------------------------------------
Placebo/delayed Tx:       -75.5                82.5  meters         0.0001
Higher 6MWT baseline
(n=2; mean=422m)
----------------------------------------------
Eteplirsen:               +7.0
Higher 6MWT baseline
(n=3; mean=424m)
----------------------------------------------------------------------------
Placebo/delayed Tx: Lower -72.1                39.9 meters          ns
6MWT baseline
(n=2; mean=367m)
----------------------------------------------
Eteplirsen:               -32.3
Lower 6MWT baseline
(n=3; mean=375m)
----------------------------------------------------------------------------
Placebo/delayed Tx:       -67.6                68.5 meters          0.0001
Genotype 49-50 deletion
(n=3; age mean=9.2 yrs,
6MWT BL mean=397m)
----------------------------------------------
Eteplirsen:               +0.9
Genotype 49-50 deletion
(n=2; age mean=9.1 yrs,
6MWT BL mean=383m)
----------------------------------------------------------------------------

** Note: Analysis based on Mixed Model Repeated Measures test

About Study 201 and Study 202 (Phase IIb Eteplirsen Study)

Study 4658-US-201 was conducted at Nationwide Children’s Hospital in Columbus, Ohio. Twelve boys meeting the inclusion criteria being between 7 and 13 years of age with appropriate deletions of the dystrophin gene that confirm eligibility for treatment with an exon-51 skipping drug, received double-blind IV infusions of placebo (n=4), 30 mg/kg of eteplirsen (n=4), or 50 mg/kg of eteplirsen once weekly for 24 weeks (n=4). Muscle biopsies for evaluation of dystrophin were obtained at baseline for all subjects, and after 12 weeks for patients in the 50 mg/kg cohort and after 24 weeks for patients in the 30 mg/kg cohort. Two placebo patients were randomized to the 30 mg/kg cohort and two placebo patients were randomized to the 50 mg/kg cohort. This study design allowed Sarepta to investigate the relationship of dose and duration of eteplirsen treatment on the production of dystrophin over the course of the 24-week study.

Study 4658-US-202 is the extension study to 201 and continues to assess the long-term safety and efficacy of open-label eteplirsen. The four placebo patients were rolled over to open-label eteplirsen at week 24, with six patients on 30 mgs/kg, and six patients on 50 mgs/kg. Third biopsies will occur at 48 weeks in the original study 201 treated patients, and at 24 weeks, the same time point, in the original placebo patients. 6MWT was performed at 32 weeks and 36 weeks, and will continue to be performed every 12 weeks going forward.

About the 6-Minute Walk Test

The 6-minute walk test (6MWT) was developed as an integrated assessment of cardiac, respiratory, circulatory, and muscular capacity (American Thoracic Society 2002) 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 (Rubin 2002, Wraith 2004, Muenzer 2006). Additionally, published data from longitudinal natural history studies assessing dystrophinopathy, a disease continuum comprised of Duchenne and Becker muscular dystrophy, support the utility of the 6MWT as a clinically meaningful endpoint (McDonald C, et al, Muscle & Nerve, December 2010) in Duchenne muscular dystrophy (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 the Statistical Methodology

The Mixed Model Repeated Measures (MMRM) test was used for all statistical analyses of the 6MWT results, including for all subgroups. Analysis of Covariance (ANCOVA) for ranked data was used when the assumptions of normality of the dependent variable (the change in 6MWT distance from baseline) were violated. The inclusion of the two patients with extreme scores due to rapid progression in the ITT population (n=12) resulted in a violation of the normality assumptions of the Change from Baseline in 6MWT data, and thus required the use of ANCOVA for ranked data. The exclusion of these two patients from the mITT population (n=10) resulted in the 6MWT data becoming normally distributed and the MMRM statistics exhibiting much improved residuals and fit statistics as compared to the ITT population. As such, the estimated mean values and their associated p-values for the mITT population were slightly different from those for the ITT population.

Conference Call and Slide Show

The Company will hold a conference call and broadcast a slide show today at 8:00 a.m. EDT (5:00 a.m. PDT) to discuss these results. The audio conference call may be accessed by dialing 866.783.2139 for domestic callers and 857.350.1598 for international callers. The passcode for the call is 77518015. Please specify to the operator that you would like to join the “Sarepta Therapeutics 36-Week Clinical Results Call.” To view the slide show while using the audio dial-in please go to the events section of Sarepta’s website at www.sareptatherapeutics.com. The call and slide show will also be webcast live under the events section and will be archived there following the call 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. An audio replay will be available through July 31, 2012 by calling 888.286.8010 or 617.801.6888 and entering access code 44234182.

About Duchenne Muscular Dystrophy and Eteplirsen

Duchenne muscular dystrophy (DMD) is an X-linked rare, degenerative neuromuscular disorder causing severe, progressive muscle loss and a premature death. One of the most common fatal genetic disorders, DMD affects approximately one in every 3,500 boys worldwide. A devastating and incurable muscle-wasting disease, DMD is associated with specific inborn errors in the gene that codes for dystrophin, a protein that plays a key structural role in muscle fiber function. Progressive muscle weakness eventually spreads to the arms, neck and other areas. Eventually, this progresses to complete paralysis and increasing difficulty in breathing due to respiratory muscle dysfunction requiring ventilatory support, as well as cardiac muscle dysfunction leading to heart failure. The condition is terminal, and death usually occurs before the age of 30.

Eteplirsen is Sarepta’s lead drug candidate that is designed to address the underlying cause of DMD by enabling the production of a functional dystrophin protein. Data from clinical studies of eteplirsen in DMD patients have demonstrated a broadly favorable safety and tolerability profile and restoration of dystrophin protein expression. Eteplirsen uses Sarepta’s novel phosphorodiamidate morpholino oligomer (PMO)-based chemistry and proprietary exon-skipping technology to skip exon 51 of the dystrophin gene enabling the repair of specific genetic mutations that affect approximately 13 percent of the total DMD population. By skipping exon 51, eteplirsen may restore the gene’s ability to make a shorter, but still functional, form of dystrophin from messenger RNA, or mRNA. Promoting the synthesis of a truncated dystrophin protein is intended to improve, stabilize or significantly slow the disease process and prolong and improve the quality of life for patients with DMD.

Sarepta is also developing other PMO-based exon-skipping drug candidates intended to treat additional patients with DMD.

About Sarepta Therapeutics

Sarepta Therapeutics — formerly AVI BioPharma — is focused on developing first-in-class RNA-based therapeutics to improve and save the lives of people affected by serious and life-threatening rare and infectious diseases. The Company’s diverse pipeline includes its lead program eteplirsen, for Duchenne muscular dystrophy, as well as potential treatments for some of the world’s most lethal infectious diseases. Sarepta aims to build a leading, independent biotech company dedicated to translating its RNA-based science into transformational therapeutics for patients who face significant unmet medical needs. For more information, please visit us at www.sareptatherapeutics.com.

Forward-Looking Statements and Information

In order to provide Sarepta’s investors with an understanding of its current results and future prospects, this press release contains statements that are forward-looking. 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 about the development of eteplirsen and its efficacy, potency and utility in the treatment of DMD and the potential for the creation of novel dystrophin to lead to significant clinical benefit over a longer course of treatment.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta’s control. Known risk factors include, among others: clinical trials may not demonstrate the safety and efficacy of eteplirsen and/or Sarepta’s antisense-based technology platform; treatment of patients with DMD using eteplirsen over a longer duration may not lead to significant clinical benefit; and any of Sarepta’s drug candidates, including eteplirsen, may fail in development, may not receive required regulatory approvals, or be delayed to a point where they do not become commercially viable.

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 Securities and Exchange Commission. Sarepta does not undertake any obligation to publicly update its forward-looking statements based on events or circumstances after the date hereof.
Sarepta Investor and Media Contact:
Erin Cox
425.354.5140
ecox@sareptatherapeutics.com

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Sarepta Announces Conference Call and Webcast on Tuesday, July 24, 2012, to Discuss 36-Week 6-Minute Walk Test Results From the Phase IIb DMD Study – July 23rd, 2012

CAMBRIDGE, MA–(Marketwire – July 23, 2012) – Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of RNA-based therapeutics, today announced it will hold a conference call at 8:00 a.m. EDT (5:00 a.m. PDT) on Tuesday, July 24, 2012 to discuss 36-week 6-minute walk test results from its Phase IIb study evaluating eteplirsen for the treatment of Duchenne muscular dystrophy (DMD).

The conference call may be accessed by dialing 866.783.2139 for domestic callers and 857.350.1598 for international callers. The passcode for the call is 77518015. Please specify to the operator that you would like to join the “Sarepta Therapeutics 36-Week Clinical Results Call.” The conference call will be webcast live under the events section of Sarepta’s website at www.sareptatherapeutics.com and will be archived there following the call 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.

About Sarepta Therapeutics

Sarepta Therapeutics — formerly AVI BioPharma — is focused on developing first-in-class RNA-based therapeutics to improve and save the lives of people affected by serious and life-threatening rare and infectious diseases. The Company’s diverse pipeline includes its lead program eteplirsen, for Duchenne muscular dystrophy, as well as potential treatments for some of the world’s most lethal infectious diseases. Sarepta aims to build a leading, independent biotech company dedicated to translating its RNA-based science into transformational therapeutics for patients who face significant unmet medical needs. For more information, please visit us at www.sareptatherapeutics.com.
Sarepta Investor and Media Contact:
Erin Cox
425.354.5140
ecox@sareptatherapeutics.com

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