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Sarepta’s hopes high for first Duchenne (DMD) therapy

Authored by James Sheppard

As part of Liftstream’s commitment to rare diseases, in the run up to Rare Disease Day 2013 we will be profiling 10 companies that are leading rare disease research. In the eighth in the series we look at Massachusetts based company Sarepta Therapeutics.

Sarepta, formerly known as AVI BioPharma, is a developing RNA-based therapeutics to treat rare diseases. Sarepta has a particular focus on Duchenne Muscular Dystrophy (DMD) for which they are currently developing 3 separate potential therapies,  the most advanced being Eteplirsen (AVI-4658) which is currently in Phase II clinical trials. Alongside this programme, Sarepta has 5 other development programmes in infectious diseases.

Eteplirsen uses Sarepta’s core PMO chemistry and is intended to skip exon 51 of the Dystrophin gene. By skipping exon 51, Sarepta believes Eteplirsen can restore the gene’s ability to produce a shorter but functional version of Dystrophin. This has the potential to significantly slow the disease process and could prolong the lives of patients with DMD. In October 2012, Sarepta announced the results of a Phase IIb extension trial for Eteplirsen which met its primary endpoint in patients with DMD. The clinical data has allowed Sarepta to advance the programme forward and prepare their primary 48 week dataset for discussion with the FDA. The results led to shares in Sarepta (NASDAQ) shooting up by more than 170% to $40.46 in October 2012. The results from the trial will help to bolster Sarepta’s case for accelerated approval for Eteplirsen.  In addition to Sarepta’s lead asset the company have 2 other programmes for DMD. In November 2012, Sarepta announced a collaboration with University College London, UK, to develop their 2 other programmes in DMD. The goal of the project is to show that targeting other exons will be able to help a wider variety of patients with DMD. At present, Sarepta’s lead asset Eteplirsen, can only help around 13% of the DMD patient population.

Sarepta also have a number of other development programmes for infectious diseases. Of these the Ebola, Marbug Virus and Influenza therapies are currently in Phase I trials. The Ebola programme which was being developed in conjunction with the US Department of Defense, until in October 2012 the Department of Defense announced it would be terminating funding for the programme. Sarepta proceeded to halt the Ebola programme and continue with development in Marburg Virus therapy. Within the Infectious disease portfolio Sarepta recently announced they would be collaborating with the National Institute of Allergy and Infectious Disease on the development of AVI-7100 for Influenza. The collaboration will involve conducting a Phase I clinical trial at the NIH Clinical Center in Bethesda, USA.

Sarepta recently announced that they would be pricing a public offering of $125m common stock to raise funding. The money raised by this offering will be used for the continued development of Eteplirsen and other clinical programmes. 2013 promises to be a pivotal year for Sarepta with a number of key trials planned and reporting.

Liftstream is supporting Rare Disease Day 2013. In profiling this company we hope to bring greater awareness to rare diseases and the companies working in this area. Liftstream is sponsoring the 2013 Orphan Drug Congress and we look forward to having contact with you at these events or by contacting us via Linkedin or Twitter

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