Sarepta Therapeutics, Inc. (SRPT)

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Sarepta Therapeutics Inc (SRPT)

48-Week Results From the Phase IIb DMD Study

October 03, 2012 08:00 am ET


Erin Cox - Manager of Investor

Chris Garabedian - President and Chief Executive Officer


Bill Tanner - Lazard Capital Markets

Chris Marai -Wedbush

Lisa Bayko - JMP Securities

Ed Tenthoff - Piper Jaffray

Kim Lee - ThinkEquity



Welcome to the Sarepta Therapeutics 48-Week Results Conference Call. My name is Jo, and I will be your operator for today. At this time, all participants are in a listen-only mode. We will conduct a question-and-answer session towards the end of this conference. (Operator Instructions). As a reminder, this call is being recorded for replay purposes.

I would like to hand the call over to your host Ms. Erin Cox, Manager of Investor Relations. Please proceed.

Erin Cox

Thank you, Jo, and thank you for joining today's call. Earlier today, we issued a press release detailing 48-week results in our Phase IIb DMD study. The press release and the slides we will be presenting today are available on the news and events section of the investor relations portion of our website at

Joining me on the call today is Chris Garabedian, Sarepta's President and Chief Executive Officer, and Ed Kaye, our Chief Medical Officer.

I would like to note that during this call, we will make a number of statements that are forward-looking including statements about the development and clinical status of Sarepta's product candidates, including eteplirsen and the potential efficacy and clinical results.

These forward-looking statements involve risks and uncertainties many of which are beyond Sarepta's control. Any such risks could materially and adversely affect our business, results of operations and the trading price of Sarepta's common stock. For a detailed description of risks and uncertainties we face, you are encouraged to review the company's filings with the Securities and Exchange Commission.

With that, let me turn the call over to Chris Garabedian, Sarepta's President and Chief Executive Officer. Chris?

Chris Garabedian

Thank you, Erin, and I am very excited to share some important news today about eteplirsen, our drug for the treatment of Duchenne muscular dystrophy. Specifically, I'll be sharing new 48-week data from our Phase IIb extension study that strongly supports the potential for this drug to treat the underlying cause of this devastating genetic disease and to slow its progression.

The data I will share today seems to confirm that treatment with eteplirsen results in robust and consistent levels of dystrophin as a percentage of positive muscle fibers, the essential protein that Duchenne muscular dystrophy patients are lacking and that this novel dystrophin production is translating to a statistically significant clinical benefit for patients.

We believe these results provide tremendous promise to patients with Duchenne muscular dystrophy and we know that the production of dystrophin is a critical protein lacking in the patients that prevents healthy muscle function and growth.

Before I provide an overview of the 48-week results described in a press release this morning, I would like to mention that we are looking forward to sharing more details from this study at World Muscle Society Meeting in Perth, Australia. On October 13th, in a late breaker oral presentation that will be presented by Dr. Jerry Mundell, the principal investigator of our Phase IIb study at the meeting.

We have slides that are available on our website for viewing during the call today. This first slide is meant to provide an overview of the design of Study 201 and Study 202, which is the open label extension study, and the data that we'll describe today has been derived from these studies.

Today, we will be describing dystrophin data from the 48-week biopsy results, and additional 6-minute Walk Test data through 48 weeks. The patients in this study will continue to receive drug and we will continue to capture safety assessments weekly and clinical outcomes including 6-minute walk test data every 12 weeks.

If you refer to the next slide, slide three, I will now provide an overview of the results that were detailed in our press release this morning. The primary efficacy endpoint defined in our protocol and statistical analysis plan of Study 202, which is our long-term safety and efficacy extension study was the increase in dystrophin positive fibers as a percentage of normal compared to baseline.

At 48 weeks, eteplirsen administered once weekly at either 30 mg/kg, or 50 mg/per kg for 48 weeks resulted in a statistically significant mean increase in dystrophin positive fibers of 47% as a percentage of normal across all treated patients with a p-value of 0.001.

There was no significant difference between the 30 mg/kg, and 50 mg/kg arms on the production of dystrophin through 48 weeks but all patients demonstrated high levels of dystrophin positive fibers that ranged from 30% to 60% of normal across these two dose groups.

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 also produced a statistically significant mean increase in dystrophin positive fibers of 38.3% as a percentage of normal with a p-value of less than or equal to 0.009.

The previously reported cohort of patients who were administered eteplirsen once weekly at 30 mg/kg over 24 weeks that resulted in a statistically significant increase in novel dystrophin of 22.5%, which was presented at The American Academy of Neurology Meeting in April. These patients showed an additional increase of approximately 30% additional dystrophin positive fibers between weeks 24 and 48, for a mean of 52.1% dystrophin positive fibers at week 48

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