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Sarepta Therapeutics, Inc. (SRPT)
The 31st Annual J.P. Morgan Healthcare Conference Call
January 9, 2013 7:30 PM ET
Chris Garabedian – President and CEO
Matt Lowe – JP Morgan
Matt Lowe – JP Morgan
Previous Statements by SRPT
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Thank you Matt, thank you JP Morgan for the invitation. I’m really excited to present what will be going on with Sarepta Therapeutics for 2013. I will be making some forward-looking statements so please refer to your SEC filings on Sarepta to understand the risk factors associated with the company.
I wanted to provide a company overview on Sarepta, Sarepta was the highest performing stock in BioTech last year. And we have a lot going on this year that we hope will drive value for investors and really make progress in the company in 2013. We are headquartered in Cambridge, Massachusetts, with about 120 employees. We are very well capitalized we have approximately $187 million in cash as of the end of the year. And our current market evaluation has been around $900 million on the current outstanding shares.
We’ve been focused on building a strong management team as you see here with recent hires at the CFO level, and General Counsel and the VP of Regulatory. These are all individuals who have experience helping build a global biopharmaceutical companies that are independent and continue to create value for shareholders. So, we really are building an organization that understands drug development and commercialization and we expect that this will drive a lot of value for years to come.
We have a pipeline in both the rear disease and infectious disease area, I’m going to be focusing primarily in our Duchenne muscular dystrophy program. And the status of that program and what we have going on in 2013, I will just highlight that we do have two active programs in the infectious disease space that is being funded by our government, both the Department of Defense and the NIH NIID respectively on our Marburg hemorrhagic fever virus program and our Influenza program. We have a lot of compelling proof of concept data that I won’t be talking about here, but that is also proof of concept for our technology and value drivers for the organization.
Our Duchenne muscular dystrophy program is very exciting in terms of what we believe can deliver a disease modifying affects to this population that has this devastating disease. This is a disease that’s characterized by these patients basically inability to produce the essential protein Dystrophin. And because of this lack of Dystrophin they end up on a progressive track toward losing the ability to walk by their pre-teen years and they have a life expectancy into their 20s, are rarely living beyond the age of 30.
The reason for this disease as I mentioned is simply their inability to produce the essential protein dystrophin which is really the shock absorber of the muscles and allows us all to maintain good muscle function into our adult lives and throughout our live.
Our drug Eteplirsen, which is a anti-sense Oligonucleotide with a very different backbone structure than we see with any other RNA therapeutics which we define as morpholinos, they are phosphorodiamidate morpholino oligomers, and we’re able to use our technology to direct alternative splicing or in essence we’re repairing the RNA mutation, the genetic mutation that exists and restoring the translation to produce the protein.
This drug is delivered systemically through IV infusion weekly. It has unique drug like characteristics, it has a short plasma half life, but once it gets uptake into the cell and produces the protein the protein has a longer half life. It’s cleared through the kidney intact, we see no metabolites, so it’s a very hearty compound that’s cleared very efficiently. And we’ve tested now this drug in patients up to 50 mgs per kg for over a year in duration currently, I’ll be talking more about the efficacy and safety profile of this drug.
The mechanism of action is what we describe as Exxon skipping, the Duchenne genotype is defined as a out of frame deletion in the dystrophin gene. This renders them unable to translate to the protein. We know from a natural phenotype that exists called Becker muscular dystrophy which is characterized by an in-frame deletion if we can simply restore translation by turning this out of frame deletion into an in-frame deletion then we can restore the ideas that we can restore the translation of the protein.
We know what the bad actor, this is an example of a patient in our study who has a deletion at 49.50. The bad actor here is Exxon 51. So, if we can silence or hybridize 51 out of the reading frame, we convert a 49.50 deletion to a 49.51 deletion which is in frame and akin to a Becker genotype and what we hope is a phenotype.
We showed this in previous studies with systemic delivery that we’re able to produce the protein in dose escalation studies, looking at 10 to 20 mgs per kg but we didn’t see the high level of dystrophin that we were hoping to see so we did a phase 2b study looking at higher doses over longer duration of treatment to see if we could actually produce dystrophin levels that we believe would be meaningful for this patient population to produce a functional benefit.