Sarepta Therapeutics, Inc. (SRPT)

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Sarepta Therapeutics (SRPT)

Presentation at Deutsche Bank dbAccess BioFEST

December 4, 2012 7:50 a.m. ET


Chris Garabedian - CFO


Robyn Karnauskas - Deutsche Bank


Robyn Karnauskas

Good morning, everyone. Thanks for joining us so early in the morning. My name is Robyn Karnauskas. I’m the Deutsche Bank biotechnology analyst. And I’m really excited this morning to have Sarepta Therapeutics. And with us we have the president and CEO, Chris Garabedian.

For those of you who are listening on the webcast, my email address is So if you have any questions, I’ll be checking my Blackberry, and I’m happy to read those questions anonymously.

Thank you very much for coming this morning. We really appreciate it. First of all, I was thinking about questions for you all this morning. It’s been a very big year. And the one thing that came to mind was that you have a lot going on for a company that sort of rose to fame this year. You have a lot going on, and I think a lot of investors are very focused not only accelerated approval, but as well as manufacturing, execution, just the company.

And then I thought we’d also talk about just what about this company and the platform technology, and how do we think about this company more longer term? Because everyone’s very focused on accelerated approval, but I’d like to have a better sense of how you view the company longer-term.

With that, maybe we could start off with walking us through, step by step, just first on manufacturing. What do you need to do to get to a place where you have enough product for maybe a confirmatory study, and then under the scenario of what if you’re required to do another pivotal trial, does that vary at all. So, step by step, what needs to be done.

Chris Garabedian

Thanks, Robyn. Thanks to Deutsche Bank for the invitation. It’s a pleasure to be here. On manufacturing, we’ve communicated a lot around timing and when certain activities will take place, and when we’ll be ready for a pivotal trial or confirmatory study.

So, first I’d say anytime you’re scaling up manufacturing, regardless of the compound, there’s complexity, there’s risk, there’s investment in time and resources. So we’re not unique in that regard. That happens with any technology. Morpholinos have not been scaled up before to the degree we’re doing it currently. We have had a lot of experience at small-scale production, both in house and working with contract manufacturers. And we’re very comfortable.

This process has worked very well. It has led to our broader Duchenne program, not just the current Phase IIB study, but obviously the previous studies that we conducted in the U.K. We also have conducted a full preclinical toxicology program. And that’s not an insignificant feat. For example, we completed a nine-month primate study where we went to doses as high as 320 mg/kg. So that’s a lot of drug to produce, even for that one study alone.

So we’re very comfortable with the process, and the purity of the compound, and the stability that we have at this smaller-scale production. We are in the process of scaling up for what we describe as mid-scale production. This is about five to six times what we’ve been doing at a small-scale production process.

So I highlighted the complexity, because I don’t want to suggest that everything is simple, but the drug chemistry itself, and the synthesis is not something that really provides a lot of technical challenges. This is an oligo. It really is a unique type of drug synthesis in that it’s not a biologic. It’s not a small molecule. It requires really a peptide manufacturing process. It’s closer to peptide manufacturing, but it’s not a peptide. So when people make the analogy to [Fuseon], that’s the wrong analogy.

We are an oligo, which means that we need to have each of the subunits. The ACTs and Gs that we produce, need to be linked to our backbone, which we’ve described is also differentiated from other oligos, which typically require linkage to a ribose backbone, a five-sided ribose ring, that’s linked by a phosphorothioate linkage. We have a six-sided morpholine ring at its base, which is linked through a phosphorodiamidate linkage.

These linkages are not difficult, but there’s many. This is a 30 [mer]. So when we talk about steps of the process, depending on who you talk to, they would not consider every one of those subunit linkages a step, because they’re fairly easy interactions to create. But there’s many of them, because we have a 30 [mer].

I just wanted to demystify the idea that this is a real difficult compound or molecule to synthesize. So we’re working contract manufacturers that have experience in the type of peptide scale up that is required for producing our drug, in a larger scale. And that is not as common, so we have a selected group that we’ve identified.

Question-and-Answer Session

Robyn Karnauskas

How many people do large scale oligo…?

Chris Garabedian

There’s a handful. And we’ve had discussions, and we’re engaged with, or are working with, many of those companies. When people ask if a partner would bring a lot to the manufacturing efforts, not really, because the expertise that exists on morpholinos and the process of production exist within our company. That’s really the most experience that’s been done, is in our company. There’s a company that sells reagent morpholinos that also has experience, but in terms of GNP quality to produce a drug for clinical trials, a lot of that knowhow exists within our company.

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