Pipeline Update: Eteplirsen - Sarepta's Q4 & FY12 results, latest developments, and next steps

Sarepta Therapeutics has provided updates on eteplirsen and their next steps with exon skipping in their latest release: Sarepta Therapeutics Announces Fourth Quarter and Full-Year 2012 Financial Results and Recent Corporate Developments.


  • Upcoming meetings with FDA will determine fastest path to pursue eteplirsen approval
  • Additional drugs for Duchenne continue to progress through preclinical development

"Last year was a transformational year for Sarepta as we achieved tremendous progress with our lead DMD product candidate, eteplirsen, which demonstrated safety and efficacy that highlights its potential to be a major advance in the treatment of this disease. We look forward to our upcoming meetings with the FDA to discuss our eteplirsen data and to determine the fastest path toward potential approval, along with our plans to supply the market in the event of an early approval."

-- Chris Garabedian, president and chief executive officer of Sarepta Therapeutics (Source: Sarepta Therapeutics)

2013 Guidance

For 2013, Sarepta anticipates that revenue will be in the $18 to $24 million range and that loss from operations will be in the $85 to $115 million range. The revenue guidance is based on the assumption that Sarepta will continue to receive funding from its current government contracts for Marburg. If Sarepta does not continue to receive this funding, its revenue guidance would change significantly. Additionally, the operating loss guidance is largely based on continuing development and scale up manufacturing for eteplirsen and our follow-on DMD drugs.

Source: Sarepta Therapeutics


Duchenne Program - Recent Developments


  • Announced updated data from Study 202, its open-label, Phase IIb extension study of eteplirsen for the treatment of DMD. Patients treated with eteplirsen for 62 weeks and evaluable on ambulatory measures (modified Intent-to-Treat population) maintained a statistically significant clinical benefit on the primary clinical outcome measure, the 6-minute walk test (6MWT), compared to patients who received placebo for 24 weeks followed by 38 weeks of eteplirsen treatment.
  • Announced a collaboration for the development of an additional exon-skipping drug targeting exon 53, its fourth drug in development, in support of Sarepta's broad-based program for the treatment of DMD. Sarepta's collaboration is with University College London's (UCL) scientist, Professor Francesco Muntoni, MD, the Dubowitz Neuromuscular Centre, the Institute of Child Health and other scientists from the EU and US. The EU Health Innovation-1 2012 Collaborative research grant will support certain IND-enabling activities and clinical proof of concept studies for an exon 53-skipping therapeutic.

Source: Sarepta Therapeutics


Financial Results & More Information

Read Sarepta's press release for more information.


Learn more about eteplirsen

Eteplirsen consists of short pieces of DNA called “antisense oligonucleotides” or “AONs” that are being tested for their ability to convert deletions near Exon 51 in the dystrophin gene from non-functional “out-of-frame” deletions to more functional “in-frame” deletions, such as those typically seen in boys and men with Becker muscular dystrophy. The strategy is commonly called exon-skipping. Although variations on this strategy might ultimately be used to try to correct deletions in many parts of the dystrophin gene, eteplirsen targets the following deletions: 45-50, 47-50, 48-50, 49-50, 50, 52.

Duchenne Research

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