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Sarepta Therapeutics Inc. (SRPT)
UBS Global Life Sciences Conference Call
September 20, 2012, 04:00 pm ET
Chris Garabedian - President & CEO
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Thank you Marina and thanks to UBS for the invitation to present here today. I am Chris Garabedian, I am CEO of Sarepta Therapeutics and we have RNA based technology that is being applied in the areas of rare and infectious diseases. I’ll be making some forward-looking statements. Please refer to our SEC filings for risk factors associated with the company.
This is our pipeline. Our lead proprietary program is for the treatment of Duchenne muscular dystrophy. The drug is called Eteplirsen and Duchenne is a genetic base disease. It's very fragmented and our lead program is targeting the most prevalent set of mutations that can be treated with a single drug. We have other exon targets that are in preclinical development now that we believe are highly reproduced to support our lead program.
We also have infectious disease programs that have largely been funded by the Federal Government. We have two active programs against the Ebola and Marburg fever viruses. These are lethal hemorrhagic fever viruses and they are being developed under the animal rule for medical countermeasures for endemic areas as well as against the weaponized threats.
I’ll be talking about our Duchenne program and Duchenne muscular dystrophy is a devastating rare disease that has 100% mortality. It’s a progressive disease that affects about one in 3,500 male births around the globe. These boys typically get diagnosed around the ages of three to five. They end up seeing progressive disease until the pre-teen years where they typically lose ambulation or end up in a wheel chair. They then start to lose their basal muscle function and lose pulmonary and cardiac function. They typically need full time ventilation in their late teens, early 20s and typically pass away in their 20s rarely living beyond the age of 30.
The disease is marked by the boys’ inability to produce the essential protein dystrophin, which is the essential protein to keep the muscle strength and vitality it’s almost a shock absorber of the muscles. And what our technology aims to do is to repair the RNA mutations that occur and fix that so they can restore translation and produce the dystrophin protein.
This is a schema of the dystrophin gene; it’s the largest gene in the human body that produces one of the largest proteins and it manifest itself in two ways; one is a deletion that is in-frame and this leads to a phenotype called Becker muscular dystrophy and this is a much milder muscular dystrophy than Duchenne; often times patients aren’t diagnosed until later in life, often times they live into their adult lives without requiring a wheelchair, sometimes never, sometimes they live a long healthy life.
Duchenne is marked by outer-frame deletion where they cannot translate to produce the protein, whereas Becker’s produces a protein, it’s a functional protein albeit shorter; it’s truncated. So what are technology is aiming to do is to fix the bad actor, the exon that makes it out-of-frame and if we can silence this or hybridize it out of the reading frame or essentially skip over it, we can restore translation and ideally produce the dystrophin protein.
So here is an example of the patient who would be in one of our studies with Eteplirsen that has a deletion of exon 49 and 50. So exon 51 is what is rendering it out-of-frame. And again, with our PMO drug, we can silence or hybridize 51 out of the reading frame and you can see how translation is restored to produce the protein.
We tested this hypothesis initially in an inter-muscular local injection study where we showed good dystrophin production locally, but we did another study following that that was published in The Lancet last year which was the Phase II study conducted in the UK where we showed with systematic delivered therapy once weekly infusions, we were able to see at the highest dose groups tested 10 and 20 mgs/kg weekly we saw every patient show novel dystrophin. We validated this by western blot and RT-PCR and you see an image in the lower right hand corner of one of the pretreatment biopsies looked like from a boy in our study and the post treatment that starts to look like normal muscle tissue.
Well, there were a couple of patients who had a more outsized response generally across this study including dose groups that are listed here, the lower dose groups, we saw low and variable levels of dystrophin produced, and we were really aiming for something that was greater than 10% dystrophin positive fibers and that was based on the literature on animal studies, which show really above 10%, you really start see functional improvement and also the Becker’s phenotype, the studies that have been done in Becker muscular dystrophy show that even levels of 10% to 20% have a very different natural course of the disease and much milder disease even with levels of 10% to 20% dystrophin.