Ian Estepan - Executive Director, Corporate Affairs Douglas Ingram - President & CEO Sandy Mahatme - EVP, CFO & Chief Business Officer Bo Cumbo - SVP & Chief Commercial Officer.

Alethia Young - Credit Suisse Salveen Richter - Goldman Sachs Christopher Marai - Nomura/Instinet Anupam Rama - JP Morgan Brian Skorney - Robert W. Baird & Co.

Joseph Schwartz - Leerink Partners LLC Ritu Baral - Cowen & Company Chad Messer - Needham & Company Dave Lebowitz - Morgan Stanley Tim Lugo - William Blair Matthew Epler - RBC Capital Markets Hartaj Singh - Oppenheimer Gena Wang - Barclays.

Good day, ladies and gentlemen, and welcome to the Sarepta Therapeutics Third Quarter 2017 Earnings Conference Call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session and instructions will follow at that time. [Operator Instructions]. As a reminder, this conference call is being recorded.

I would now like to turn the conference over to Ian Estepan Executive Director of Corporate Affairs. Sir, you may begin..

Thank you, Yukiya, and thank you all for joining today's call. Earlier today we released our financial results for the third quarter of 2017. The press release is available on our website at www.sarepta.com, and our earnings 8-K was filed earlier this afternoon. Joining me on the call today are Doug Ingram, Sandy Mahatme and Bo Cumbo.

After our formal remarks, we will open up the call for Q&A. I'd like to note that during this call, we'll be making a number of forward-looking statements. Please take a moment to review our slide on the webcast, which contains our forward-looking statements.

These forward-looking statements involve risks and uncertainties, many of which are beyond Sarepta's control. Actual results could materially differ from these forward-looking statements as any and such risks can materially and adversely affect the business, results of operation and trading price of Sarepta's common stock.

For a detailed description of applicable risks and uncertainties, we encourage you to review the company's most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K, filed with the Securities and Exchange Commission, as well as the company's other SEC filings.

We plan to file the 10-Q for the third quarter of 2017 by the SEC required filing deadline in November. The company does not undertake any obligation to publicly update its forward-looking statements, including any financial projections provided today based on subsequent events or circumstances.

And with that, let me turn the call over to our CEO, Doug who will provide an overview of our recent progress.

Doug?.

Thank you, Ian. Good afternoon, everyone. We appreciate you joining us for Sarepta Therapeutics third quarter 2017 financial results and corporate update. Four months ago, I joined Sarepta with each passing week, I've become even more excited about what we are doing, the positive impact we can make and what we can become.

I have joined a team that has a noble mission profoundly improving the lives of children suffering from a rare cruel disease. And better still, we at Sarepta have the technology, the pipeline, the resources, the talent and the passion to fulfill that mission. I am proud of the fine work of my team this quarter.

As you all have seen from today's earnings release, the third quarter net revenue for EXONDYS 51 reached $46 million exceeding consensus. Our strong performance places us in a position to once again raise guidance.

As reported today, we are increasing our full year guidance to $150 million to $155 million up from $125 million to $130 million as reported in the previous quarter.

Our performance today reflects the feedback that we have received from scientists, staff leaders of the many physicians of experience treating patients with EXONDYS 51 as well as patients and caregivers who have received this treatment.

We regularly and consistently receive feedback about the positive impact of EXONDYS 51 which in addition to our team's tenacious execution explains our fine performance today. Let's put this performance in context, if we achieve our full year guidance EXONDYS 51 will represent the greatest ultrarare disease full year launch in history.

But let us look across all rare disease launches; if we achieve our guidance for 2017 our performance will place us in the top five most successful rare disease launches of any kind ever rightly sharing that rarefied distinction with biotech stand outs Vortex, Shire, Alexion and Biogen.

Let me now turn to the progress we have made this quarter in advancing our development program. In late September, we reported positive top-line data from our 4053-101 study, which was an important step toward bringing golodirsen to the DMD community and the 8% of patients who could potentially benefit from this therapy.

The 4053-101 results further validate our precision medicine RNA splicing platform and our commitment to scientific excellence to our continuous methodological improvements.

For instance, we believe that the scientific rigor we employed to quantify dystrophin in the study could become the gold standard for the accurate measurement of dystrophin going forward.

As a reminder, the 4053-101 study achieved statistical significance on all primary and secondary biological endpoints in 25 patients with DMD amenable to skipping exon 53.

Golodirsen uses Sarepta's proprietary PMO chemistry to precisely skip exon 53 of the DMD RNA transcript allowing for the production of an internally truncated but functional dystrophin protein. In the study, there was a 100% response rate of exon skipping as measured by RT-PCR.

With all 25 participants in the study exhibiting a statistically significant increase in skipping exon 53 above baseline levels.

Mean dystrophin protein increased to 1.019% of normal compared to a mean baseline of 0.0995% of normal and to remind you that is a P value of better than point 001 as measured by Western blot the primary biological endpoint in this study. And that represented a 10.7%-fold increase over baseline.

This data was also supported by the highly statistically significant increase of dystrophin expression in the sarcolemma membrane as measured by immunohistochemistry.

This is now the second exon skipping therapy to have shown a statistically significant increase and exon skipping, dystrophin production and proper dystrophin localization and it takes us one step further in our journey to treat all of the boys with the DMD who have amenable mutations. We were honored when Dr.

Francesco Muntoni, the principal investigator for the study presented the 4053-101 results at a late breaking posture at the 22nd Annual Congress of the World Muscle Society and we understand that experts in the field were impressed with the rigor of the message and the consistency of the full results.

As a reminder golodirsen along with another one of our drug candidate casimersen are the subject of ESSENCE trial a global randomized, double-blind, placebo-controlled study evaluating patients amenable to skipping exon 45 to exon 53 which together make up approximately 16% of boys suffering from DMD.

Golodirsen recent in vivo results are consistent in vitro models that indicated that it was two times or more efficient in exon skipping versus EXONDYS 51 and it should be noted that casimersen our exon 45 candidate has shown exon skipping efficiency in vitro that is comparable to golodirsen. Turning to another important development from the quarter.

Our marketing authorization application is currently being reviewed by the European Medicines Agency. On our last quarterly conference call, we reported that we received initial feedback from the agency on our application and we requested a six-month clock stop.

We have completed the necessary analyses and have provided the agency with the ADME study results which demonstrated no unexpected findings. The review clock is restarted and we remain on track to receive a response from the EMA's committee for medicinal products for human use the -- on our application in the first half of 2018.

Turning to the rest of our pipeline, let me start with this. The children we serve do not have the luxury of time and that means that we also do not have the luxury of time. We are refining our five-year strategic plan and our 2018 budget.

As we frame our strategy for 2018 and beyond, you will hear me say repeatedly that our plans will be fueled by a sense of urgency to advance our pipeline and bring medicines to market with rapidity and a sense of profound duty to these children who are waiting for therapy.

And as we grow as a fully integrated biopharmaceutical company, our corporate strategy will rest on two fundamental principles. First, for our approved therapies currently EXONDYS 51, we will work doggedly to ensure that eligible patients are able to gain access to and benefit from our DMD therapies.

Second, we will rapidly and diligently advance our pipeline with three goals in mind. First, that the greatest number of DMD patients can benefit from therapy. Second, that we do not rest on our prior success, but continue to advance our therapies to have the greatest impact on the lives of those with DMD.

And third, as we get positive signals that we extend our innovative pipeline into new areas for those who are suffering from genetic diseases can benefit beyond DMD. Let me speak about the PPMO progress. We have spent considerable time this quarter advancing our PPMO platform.

As a reminder PPMO is our next generation platform designed around a proprietary cell penetrating peptide conjugated to our PMO backbone. With the goal of increasing tissue penetration, increasing exon skipping and significantly increasing dystrophin production.

If successful, the PPMO offers the potential for improved efficacy and less frequent dosing for patients. We have previously presented data in the MDX mouse model that indicates that a single dose of PPMO produce significantly more dystrophin protein than a single dose of PPMO in skeletal and cardiac muscle.

In addition to being a promising next generation therapy for DMD, PPMO has the potential to address multiple neuromuscular diseases as well as potentially having utility in a broad range of other diseases.

We are currently reviewing the toxicology from preclinical studies and we remain on track to dose the first patient with a PPMO candidate targeting exon 51 amenable children before the end of this year. At the same time, we are working on an ambitious strategy to advance multiple additional PPMO candidates.

In addition to our internal programs as you know we have three gene therapy collaborations. Two with Nationwide Children's Hospital, and a third with Genethon. Let me first comment on the two programs we have with Nationwide. Significant progress has been made this quarter in both programs and both are scheduled to enter the clinic this year.

The first is our collaboration with Jerry Mendell, M.D. and Louise Rodino-Klapac Ph.D. co-inventors of Nationwide Micro-Dystrophin.

Doctors Mendell and Rodino-Klapac are leaders in neuromuscular gene therapy and the developers of the gene therapy AVXS-101 for spinal muscular atrophy currently licensed to AveXis, which recently received and released positive and promising Phase I results.

As background on the Micro-Dystrophin program in preclinical studies systemic delivery of the construct resulted in high levels of gene expression in skeletal and equally importantly in cardiac muscle. This program is agnostic to genetic mutation and holds the promise of treating a majority of DMD patients.

As we move closer to the clinic, we anticipate the patients will be dosed at levels which are potentially therapeutic. This means that the program should generate not merely safety but also gene expression data. We expect that the first patient in this study will be dosed this quarter. Our second program is a collaboration with Kevin Flanagan M.D.

Nationwide's Director of Gene Therapy and the principal investigator for the Galgt2 program which was developed by researcher Paul Martin Ph.D. This gene therapy program targets the dystroglycan complex to enhance utrophin expression and preserve muscle function.

The Galgt2 approach is also agnostic to genetic mutation and has the potential to treat patients of all ages, disease severities and even to potentially address alternative forms of muscular dystrophy. We expected the first patient in this study will be dosed this quarter. Our third gene therapy partnership is with Genethon.

Genethon recently published data validating its micro Micro-Dystrophin gene therapy approach in an animal model for DMD. The results were featured in an online issue of nature communication.

While early these data highlight the potential for Genethon's Micro-Dystrophin gene therapy program and once again underscore the significance of dystrophin production in the treatment of DMD. Finally, before turning the call over to Sandy for an update on the financials, I would like to officially welcome to Sarepta Dr.

Guriq Basi, our new Chief Scientific Officer. Under Dr. Basi's leadership we will accelerate our pipeline and the pursuit of new innovative approaches to treat DMD and other rare neuromuscular diseases.

With that Sandy?.

Thanks Doug, good afternoon everyone. We are pleased to report that based on continued progress with the launch. We have generated net revenues of $46 million of EXONDYS 51 sales in the third quarter of 2017.

For modeling purposes, it is important to remember that last quarter we reported a change in ordering patterns due to the 4th of July holiday that pulled forward approximately $2 million of revenue from the third quarter into the second quarter. With that in mind, the revenue number for the third quarter would have been approximately $48 million.

As Doug previously mentioned, we have raised our net revenue guidance for the year to a range of $150 million to $155 million. Our ability to forecast is based on commercial trends and our comfort is the trajectory of the launch. This also takes into consideration the holidays and the remaining business days in the quarter.

Our guidance only includes sales generated in the US and does not include any potential sales from our managed access program. We expect very limited revenue from this program late in the fourth quarter and into 2018.

We're quite pleased with how the launch is progressing our Q3 achievements and that our guidance ranks EXONDYS 51 amongst the top rare disease launches of all time. Now moving to the financials. This afternoon's press release provides the details for the third quarter 2017 in both an adjusted or a non-GAAP basis as well as the GAAP basis.

The press release is available on the SEC and company website. The non-GAAP results we will discuss on this call provide a more accurate picture for ongoing operations and the impact of operations on a cash balance and exclude restructuring and stock competition expenses.

Please refer to our press release for a full reconciliation of GAAP and non-GAAP. In the third quarter of 2017, we reported a non-GAAP net loss of $12.4 million or $0.20 per share compared to non-GAAP net loss of $45.9 million or $0.95 per share in the third quarter of 2016.

The decrease is due to the product sales of EXONDYS 51, lower manufacturing expenses due to the capitalization of inventory upon the approval of EXONDYS 51 and offset by increased professional services primarily due to our global expansion. Net revenue for the third quarter of 2017 was $46 million.

No revenue was recognized in the third quarter of 2016. Cost of sales was $3.1 million in the third quarter of 2017 which relates to sales of EXONDYS 51 following the commercial launch in the US.

Included in the cost of sales of approximately $2.3 million of royalty cost of goods sold as a result of the settlement and license agreements we entered into with BioMarin in July of 2017. There was no cost of sales recognized for the same quarter last year.

Prior to the approval of EXONDYS 51 we have spent such manufacturing and material cost as research and development expenses. Adjusted non-GAAP research and development expenses were $32.4 million for the third quarter of 2017 compared to $30.9 million in the third quarter of 2016 an increase of $1.5 million.

The increase was primarily driven by increased patient enrollment in our ongoing clinical trials. A ramp up of preclinical studies for the companies PPMO platform as well as other fallow on exon and increases in professional fees partially offset by lower manufacturing expenses due to capitalization of inventory following the approval of EXONDYS 51.

Adjusted non-GAAP selling, general and administrative expenses were $23.1 million for the third quarter of 2017 compared to $14.8 million in the third quarter of 2016 an increase of $8.3 million due to increased professional services related to our global commercial expansion and the BioMarin settlement as well as compensation and other personnel expenses due to increases in headcount.

We had approximately $618.4 million in cash, cash equivalents restricted cash in investments at the end of this quarter. In addition, we have prepaid approximately $20.3 million towards our 2017 and 2018 manufacturing. We are pleased with the way the business is trending and are well positioned going into 2018.

With that, I'd like to turn the call over to Bo for a commercial update.

Bo?.

Thank you, Sandy. Good afternoon, everyone. We remain confident in our ability to continue the successful launch for EXONDYS 51, as evidenced by the strength of our third quarter earnings and our updated net revenue guidance for the first full-year of launch.

We are very pleased with the progress the team has made to-date and are looking forward to a strong finish to the year. Operationally we performed well during the quarter against our four key activities within the United States.

Driving prescriptions for identified and appropriate Exon 51 skip [ph] patients which has continued to strengthen throughout the launch. Maintaining active dialogue with payers to support coverage and reimbursement decisions for all patients with a minimal mutations to EXONDYS 51.

Addressing procedural barriers to therapy to shorten the timeframe from start to first infusion and ensuring DMD patients have genetic test and are appropriately identified for Exon amenability. Our internal assumptions of the market size have been consistent since launch.

The team continues to ensure our physicians understand the importance of identifying and starving their patients on EXONDYS 51 and we are pleased to hear more physicians doing chart pulls within their own clinics and finding additional patients as we progressed throughout the launch.

Our genetic testing collaborations have also continued to identify more patients who can benefit from EXONDYS 51 and additional Exon skipping products for any potential future launches. Patient demographics have remained fairly consistent throughout the launch. The percent mix of patients on commercial and Medicaid plans are approximately 60:40.

We are pleased that we're seeing adoption across all age groups. The average age of patients currently on therapy is 13 years of age which indicates both ambulatory and non-ambulatory patients are obtaining access to EXONDYS 51. This is consistent with our expectations of how the launch would progress.

From a compliant and persistency perspective, we have previously reported that approximately 40% to 50% of patients opted to have ports placed prior to their first infusion. Based on discussions with physicians and our specialty pharmacy partners, we expect this trend to continue.

We believe cohort placements, home infusions and minimal side-effects reported have all led to higher than anticipated compliance rates. Throughout the first year of launch, we are continuing to see high compliance rates and minimal discontinuations. At the end of the quarter, 100% of Tier 1 and Tier 2 centers have now submitted start forms.

These centers which treat approximately 75% of the DMD population have continued to identify new patients amenable to Exon 51 skipping and submit start forms accordingly.

We are continuing to see an increase in the number of new start forms for Tier 3 sites as well, and now have over 160 individual physicians who have submitted a start form since launch.

Based on ongoing efforts and engagements, we also believe payers have a much better understanding of the disease, the number of patients eligible for treatment under their plan and the patients who would most likely benefit from EXONDYS 51.

We have previously highlighted that educating physicians, healthcare providers and DMD families about the importance of genetic testing would be critical for successful launch.

As a result of increased genetic testing since approval, a greater number of physicians now know which patients have mutations that are minimal to skipping Exon 51 and we expect this trend to continue. Our managed access program continues to expand to include more countries.

And we were beginning to build appropriate commercial operations infrastructure in key regions to support global growth overtime. We've now made offers to select countries managers within key markets across Europe. We have also started building our commercial infrastructure in Latin America and other countries as we look to expand globally.

We benefit our operational preparedness has support a very successful launch in 2017. We are excited about the future at Sarepta and will continue to buildout [indiscernible], commercial infrastructure to prepare for EXONDYS 51 launches in other key markets.

As well as prepare for future approvals with other PMO and PPMO Exon skipping products and our multiple gene therapy programs in DND and beyond. And with that I will turn the call back over to Doug for closing remarks..

Thank you, Bob. We've made great progress this quarter. Looking forward in the coming months we anticipate a flow of important milestones regarding our clinical programs. We are pleased to have a total of seven programs in the clinic by year end 2017 and we anticipate the following milestones in the coming months.

First, we anticipate that both our micro dystrophin and Galgt2 collaborations will enter the clinic this year. We will provide additional color on the design and scope of these trials following initiations. Second, we remain on-track to dose our first patient and our next-generation PPMO program by year end 2017.

We are designing clinical studies to aggressively move this program forward. Third, we anticipate completing enrollment in our ESSENCE study by year end 2017 or early in the first quarter of 2018.

Fourth, and based upon the positive results of our 4053-101 study, we are targeting a meeting with the FDA in the first quarter of 2018 to discuss the [indiscernible] program and the proper path to bring this therapy to the children who are waiting for it.

Fifth, our partner, Summit Therapeutics is on-track to announce 24 week data from their Phase II proof-of-concept clinical trial of azutramide in the first quarter of 2018. And lastly, we anticipate that the review of our application by the CHMP should be complete in the first half of 2018.

We have continued to strengthen our position as the leader in DMD.

With the ongoing commercial success of EXONDYS 51, a deep pipeline like of DMD candidates and platform technology and significant progress towards the realization of our strategic vision upon which we are building a global biopharmaceutical company dedicated to improving the lives of those suffering from devastatic rear neuromuscular diseases.

And with that operator, I would open the call for questions..

[Operator Instructions] And our first question comes from the line of Alethia Young of Credit Suisse. Your line is now open..

Thanks for taking my question. Congrats on all the progress with EXONDYS 51 on the launch. You're going to kill me but I kind to have to -- one, I just -- you know, since you guys we haven't spoken since the adverse effects data base kind of came onto the scene.

I just want to get your perspective on the finding; they were there and then the second question.

I guess, I just wanted up lay out and help us think about that what -- kind of the best case is for this meeting with Exon 53 and what would be a potential more positive case than kind of -- are you talking to the same people that you knew from before when you were working with tippers [ph] and things?.

Thanks for those questions.

So first, let's talk about the FAERS database and the report, just for those make sure we're on the same page, they are -- the FAERS database which is the FDAs database on spontaneous adverse reporting that occurs post-market; they updated their database to provide a user-friendly tool and a few weeks ago some folks, not physicians I think, it was others -- did some searching and then discovered in their searches that there were two children, I believe at the time that had -- three children actually that had -- that were DMD patients and have been on our therapy and that had died.

Here is the obvious issue, the reason we are so passionate about what we do is because this disease is so extraordinarily cool; it will unfortunately kill 100% of these children.

DMD is a horrible and cruel disease, it takes the lives of these children usually in their late teens or very early 20s and certainly before they are 30, almost every child who suffers from DMD is going to die. And these three children unfortunately were among those, they were -- one of them was 27, two of them were 28.

If you -- when DMD takes you, it usually takes you through respiratory or cardiac issues and that's exactly what happened with these kids. So it did not relate to their treatment with EXONDYS 51, it was entirely consistent with the natural history and the course of this disease.

And unfortunately as we treat this disease and as this FAERS database exist, we will see more of children like this and to make that point even more poignantly, we have two children who died waiting to get on therapy while they were going through the preapproval process with FAERS and that's the horrible nature of this disease.

It is a tragedy, it is a tragedy for the families of this children, it is unfortunately also completely to be expected. So that is the -- sort of, what was going on with that.

So the second question as relates to a meeting with the FDA on golodirsen, I think the short answer is, we really can't speculate until we have our meeting with the FDA and the division and it will be at the division level.

I think we're in a good place to have a robust discussion with the FDA, the results of our study with golodirsen not only are statistically significant on all of the measures but also the scientific rigor with which the team approached this study is impressive and I think certainly hopeful we'll impress the division as well and then we'll have to have an open dialogue.

The best case scenario would be that they would find an accelerated pathway with us, perhaps with some additional data they would need or we may join issue and they may see things differently but we won't know exactly what their perspective is until we talk with them and discuss it at a scientific and evidenced base meeting and that's going to be our approach and we'll certainly update folks as we progress..

Great, thanks..

Thank you. Our next question comes from the line of Salveen Richter with Goldman Sachs. Your line is now open..

Thanks for taking my questions.

At this point you're now about a year post launch, just wanted to understand as payers, are -- interacting with physicians; what ongoing clinical assessments are they requiring and how could these evaluations impact reauthorization? And then just secondly, what is the current proportion of DMD patients that have been genotyped? And when we think about growth for EXONDYS 51 in terms of nearly identified patients here, how should we think about that?.

I'll say two broad stroke answers and then Bo please feel free to follow-up and provide more data.

I mean, so the -- particularly as it relates to, I think the kind of the current penetration, the second question; penetration -- the short answer is we are still at launch phase, we made great progress, we have a long way to go, we are not deeply penetrated.

They are short of three groups of children -- there are some children -- the children that are on-therapy, early and launched on therapy. There is a significant queue of patients that are working their way through the process to be on therapy.

And there is a great number of patients that are left to be identified and brought into the Tier 1, 2 and 3 clinics and the genotypes and there is a lot of work to do there. There is a lot of work to continue education in genetic testing and the like as we go forward.

So, we're not deeply penetrating we're doing very well but we've got a lot of runway and a lot of work to do ahead of us.

And then I'll say little bit on the first and the Bo should really add some more about the - on the payer side, the short answer is there apparently with respect to reauthorization oftentimes they've been approaching the treating physicians and asking for granular information from the treating physicians about their experience with EXONDYS 51 and how they see it affecting the patient versus natural history what they would have expected.

To the extent that that is going to be the approach that's being taken these children are going to fare very well in getting reauthorized because as you heard earlier in the script, we have very good adherence and one of the reasons for that is that physicians are reporting a lot of utility with EXONDYS 51 and success with EXONDYS 51 so I think they are going to fight for these children and for these children continue the use of EXONDYS 51 as a therapy.

With that Bo?.

Yes, thanks for the question. I think I'm very pleased with the progress that we've made on the managed care front. And the physicians are actually. I mean the managed care organizations are proactively reaching out to the true traders the KOL's.

And they are - so they are not looking for one individual like a six-minute walk test they're asking really good and thoughtful questions about how the patient is doing. They are also taking a hard look at some of the guidelines that are out there like California, the [ph] CCS guidelines.

Let's take a look at Brooke score, pulmonary function tests and all which is very broadly it's open type of formulary and they're asking questions about PFT for the non-ambulatory patients Brooke scores and just the overall feeling of the patients, I think we've been going through the reauthorization really immediately after launch about six months after launch.

So, we've been going through it. It's been very positive and I think what I'm very pleased about is the managed care plans are thoughtfully reaching out the right people across the country and gathering feedback from multiple KOL's. So, I'm pretty pleased with that.

As far as the genotype question originally at launch we estimated it was roughly 50% percent population was genotype with 40% being non-genotype and a lot of those patients were either very young or they are very old.

And we've made a lot of progress there; I don't have actual percentage for you because we're going to be doing a market research now one-year post approval. But across the board we're seeing genetic testing rates increase, we have a Decode Duchenne program I'm very pleased with the program.

We've had over 700 tests and from those tests we've actually identified 500 DMD patients. And with approximately 13% of those patients being exon 51. And we're also seeing other genetic testing companies have five-fold, six-fold increase from baseline.

So we're going to do the market research, we'll have a better understanding but it's much higher than 60% prelaunch..

Thank you..

Thank you. Our next question comes from the line of Christopher Marai of Nomura. Your line is now open..

Hi, good afternoon guys thanks for taking the questions.

Maybe just on the two that you have enter the clinic in the fourth quarter I was wondering if you could just help us understand the manufacturing process and obviously the dose levels that you'll be entering the clinic with Micro-Dystrophin? Is it correct to assume that those are being produced at nationwide clinicals? And then maybe how should we look at the potential data from that program obviously the dystrophin measure, you know could come rather quickly after the first dose and you sort of hinted that early on the call? And then finally just maybe follow-up a little bit on the parts of the PPMO chemistry and the toxicity profile that you'd be looking for -- you're doing the pretalks now but that's sort of been on focus of investors we talk to.

We would love to hear about what talks you particularly worry about within your - when you see that data and when we will see that data? Thank you..

Great. So, let's walk through them. So first of all yes, the therapy is being manufactured at Nationwide in fact it's been manufactured so we're in good shape to start the clinical program to start dosing. We haven't discussed externally and it isn't fully confirmed that the exact dose that's going to happen.

Although I can tell you that it's intended to be certainly what the experts would imagine would be a therapeutic dose, so we should be able to get good gene expression as well as safety and hopefully good signals on dystrophin.

We haven't fully nailed down or disclosed externally the exact protocol and when biopsies are going to be taken and you are certainly correct that gene expression will happen fairly rapidly in a matter of some months not years and therefore you could envision earlier signals but that's all subject to the protocol and when biopsies are going to be taken.

So, we'll give you a further update on that after the study initiates and we're able to do that. And then on PPMO, so on PPMO we are reviewing the talks and now our goal is to have patience and they will be patients this won't be healthy volunteers.

Patients begin dosing this year we're very hopeful on the safety side obviously the signal that one would look for here really is that kidney issues and that's the issue looking for a signal and we'll have that fully evaluated and we'll certainly, once we have an I&D filed and accepted and we're ready to dose the child we'll press release that and you'll have access to that information when that happens.

And it is our goal to have that this quarter..

Thank you. Our next question comes from the line of Anupam Rama of JP Morgan. Your line is now open..

Hi guys, thanks so much for taking the question. For EXONDYS 51 the age range here that's kind of kicked around between kind of say 13 and 15 years old. You know how is this kind of trending what's assumed in the 2017 guidance and how should we be thinking about this going into 2018? Thanks so much..

Let me, I'm going to say one thing about it and then Bo is going to talk about it. So, as you may have seen that we are about 13 is the average age now.

So, one of the and so our goal of course is to have as a broader coverage as possible as we move forward that's our goal and that means that hopefully if things go well we'll be getting to younger and younger children we'll be enhancing people's ability to get children diagnosed earlier in the like and our goal would be that there would be younger children and the mean age would actually go down over time which would be a signal of very positive answers from our perspective.

But with that said Bo, do you want to make some more comments..

Yes.

I think this is a very positive signal, it shows that what we thought at launch would happen is happening and when you think about -- we break things down into five age groups I mean age groups above 5 years at a time so from a conversion standpoint there is really no difference in conversion percentages across any of the age groups all the way up to like 24, 25 years of age.

So, we're converting ambulatory, non-ambulatory patients at the same rate. But due to the dynamics of this disease unfortunately this cruel disease state there are a lot more boys that are younger the younger age groups. And this is -- and we're also doing a lot of genetic testing.

And so, when you think about the average age of diagnosis being close to five years of age and you're out here putting a lot of emphasis and effort on genetic testing. You're finding a lot of these other kids that are below five and it's just pulling down the age group but not only because we're finding so many more patients.

It's actually a great thing it's what we anticipated pre-launch it's showing up now and we're happy because long term these kids are going to be on the drug for much longer and hopefully generate better efficacy..

Great. Thanks so much for taking the question..

Thank you. Our next question comes from the line of Brian Skorney of Robert W. Baird. Your line is now open..

Hi, good afternoon guys thanks for taking my questions. I guess two, first you mentioned that your efforts are getting to have a better understanding of the benefits of the drug.

Just wondering do you expect to resolve in any formal changes to medical policies as we move towards the fiscal year? And then on the two-gene therapy Micro-Dystrophin programs, could you just review what the differences are between the Nationwide and the Genethon programs and the manufacturing capacities where both of those contracts right now at the centers and if early clinical data justifies that how should we be thinking about the capacity to scale up those gene therapies?.

Yes, okay let me take these in order. So, the first question was sort of the - physicians are getting additional experience with EXONDYS 51 and what does that mean for coverage, so the answer I think is hopefully the body has evidence that support the utility and benefits that come from EXONDYS 51 are increasing.

So, let's first start with there are 100 or more KOL's, thought leaders and physicians who have experience some of them have years of experience with EXONDYS 51 with children and can point with objectivity and with specificity to the benefits that they're receiving that's helpful with payer certainly.

The additional data that we are developing now and we will be in the future, we have non-ambulatory study that's reading out that hopefully will get published in very interesting on EXONDYS 51 it will be very helpful to us forward going forward.

I can't say with certainty that the coverage issues will change immediately, but I think that it should get - there should be an increase in confidence among payers as they see physicians' reactions and you can see it.

I mean the revenue numbers we have right now are important as revenue but they're more important in what they signal what they signal is we're having a very good adherence much better than one would have imagined.

Physicians are fighting to keep these children on drug and children and caregivers are seeing the utility even though they have to get infused every single week and are fighting to stay on drug all because they see the utility and the benefits of this drug and if you couple that with the data that we're developing over time I think there really is a chance to get better policy answers over time.

As it relates to both got Genethon and Nationwide, a couple things one is they are different and they have different promoters and different viral vectors obviously. So Nationwide vector is AAV 74 which is largely homologous to AAV 8. And their promoter - Genethon has AAV2/A.

Both of them have very good manufacturing capabilities from a clinical perspective.

Obviously, if we see positive signals in either one of these programs or with our Galgt2 program, we've got a lot of planning to do from a commercial manufacturing perspective and we certainly will, we should be doing a lot of planning around that and thinking around that we wouldn't pull the trigger on those kinds of issues until we were at the right place.

But certainly, they are well prepared to support us from a clinical perspective no issues there, we're in good shape. If we get to the point where we get positive signals and we need to think about commercial then we're going to have to jump in the gear, but it's premature to do that right now..

Great. Thanks..

Thank you. Our next question comes from Joseph Schwartz of Leerink Partners. Your line is now open..

Thanks very much.

I was wondering if you could (indiscernible) for us with the significant amount of activity that was seen for -- at a percent at the similar stage of development?.

I apologize, we were unable to hear the middle there. So, can you repeat your question..

Yes, sorry about that. I'll speak up. So, I was just wondering if you could draw any comparison that the significant amount of activity that we see by patient advocate for - at a similar stage of development that you find yourself now for golodirsen.

So, could you give us any insight into how you are interacting with the patient advocacy organizations for DMD and do you have any insight into how they may be interacting with the FDA at this juncture for golodirsen?.

Yes, excellent question. But as I understand right now our goal is that of leading with the division on golodirsen and walking through the evidence and do it on evidence based science based perspective. I think it's very valuable at the right stage to ensure that everyone understands the impact of the disease on patients and families.

But I think for the purpose of our meeting with the FDA it's not the appropriate time for that.

So, there are no interactions that I'm aware of between patient advocate groups and the FDA today as it relates to golodirsen and I think really kind of going into our meeting with the FDA, we want to ensure that we keep that focus on data review and evidence based discussion about the path forward..

Okay, great. Thanks.

And when can we expect the earliest - we can expect some clinical data for golodirsen to be recorded?.

Well, we have still with golodirsen, the clinical data would be things like ticking of a watch, very few years away.

We have an ESSENCE trial right now that has exon 45 skipping drug in it and we also have golodirsen exon 53 in it and we are recruiting right now we should be complete with recruiting very soon either at the end of this year or maybe early into next quarter and then we've got couple of years before study would read out there will be some time from now from a ESSENCE trial perspective which is where we would get those results..

Great. Thanks for taking my questions..

Thank you..

Thank you. Our next question comes from the line of Ritu Baral of Cowen & Company. Your line is now open..

Hi guys, thanks for taking the questions. I wanted to ask about some of the other studies that are ongoing. One, have you settled the confirmatory [ph] trial design with FDA? Two, what are your plants at these points for [ph]? And three any plans to run a 101 like study for casimersen, the 45 candidate and then I have a follow-up..

I'm sorry, I apologize for that. I miss the third question..

Casimersen, your 45 candidate was there any plan to run like a 101 like dystrophin study for that candidate and have that read out before others?.

So, we have a safety study for casimersen but that was in non-ambulatory children so, it doesn't have. We weren't taking biopsies so we don't have data for casimersen like we do with golodirsen. Depending on the outcome of our meeting with the FDA we are going to have to review that and decided if that's a meaningful approach.

We can do that post the discussion with the FDA. Going back to the original question on the confirmatory trial. We're still designing trial as we speak, it will likely be probably a combination of a couple of trial that together would be confirmatory.

And I think as you we're working with diligence on that's read out until 2022, but - that done and confirm with the FDA if possible. And [ph] is fully enrolled now and we're going through it..

Got it. And one question actually for Sandy.

Just as you and Bo look at commercial expansion, what should we think about US and Latin America final commercial numbers and impact on SG&A for 2018?.

Yes. So, we are in the process of preparing our long term strategic plan Ritu that would be in Q4, so we'll in a much better place to address it or really next year perhaps at JP Morgan. It'd be too early to speculate on that having said that we've been very in the manner in which we've tackled our SG&A and the growth has been very phased.

We're ensuring that we're using a combination of distributors and booths on the ground, but the team here it's going to be a very phased approach perhaps the EU 5 Brazil and some countries initially and then gradually look at everything else based on the revenue expectations as well as the expenses we might have.

Bo, am I missing anything?.

No, Sandy. I think you've covered it..

But Ritu, to Sandy's point we're taking a very thoughtful and conservative approach and looking at the countries with the biggest opportunity that we can get to it seems possible..

I think our quarter-over-quarter SG&A growth, it's actually even growth, it's actually a contraction to flat indicates that we are taking a very disciplined approach to SG&A. What we're really focused on is uprising research and development and regulatory.

On the platform as relates to Europe and those are the point, we're going to phase - we're going to definitely build, but we're going to phase our build with the way approval happen in Europe.

So, we're going to phase in some regulatory and medical affairs and then as we -and then accessing reimbursement and then we'll phase in additional SG&A as necessary post approval when we get closer to actually launching the drug..

Thanks for taking the questions..

Thank you..

Thank you. And our next question comes from the line of Chad Messer of Needham & Company. Your line is now open..

Great. Thanks for taking my question and congratulations on the progress.

I was wondering if you could comment may be even directionally on start forms and the process from when they are submitted to when a patient gets drug and whether is that getting longer or shorter or has it reached some kind of steady state and then likewise for the numbers of the new start forms that you see? Thanks..

Hi Chad. So, number of start forms we never got into - JP Morgan. But I could tell you that it's been very consistent throughout the launch.

Our team has been working very hard to with all the offices to try to identify and try to identify patients and we've done a really good job of making sure that we find the patients they are appropriate for exon 51 and get them in as soon as possible.

I think from a time to infusion standpoint, you know it really varies and to answer one part of your question. Yes, it's stabilized early on not a quarter and a half ago. And if anything, we are getting faster. But did we - is up to every individual patient takes sort of a different paths.

I've had patients that have got on therapy within three days and I've had others they have taken many months and that could be because of a number of reasons.

One, sometimes there is intellectual issues with the child so that he is not ready for therapy, there is support issues, if there is support problem, then you have to get a -- console so that delays time also it depends on the insurance plan, so it is variable. But overall, it has stabilized if anything it is getting faster.

I am quite pleased with where we are right now and we have multiple patients in the queue..

Thanks..

Thank you. Our next question comes from the line of Matthew Harrison of Morgan Stanley. Your line is now open..

Hello, this is Dave Lebowitz in for Matt. I had a question regarding the, I guess comparing the exon 53 patients versus the exon 45 patients.

How do these patients compare being a typically and how should you look at treating those patients as far as the challenge that's ahead as compared to exon 51 patients?.

Well, I think broadly speaking the assumption is that the phenotype of 53 and exon 45. Boys are the same hence the reason that the FDA permitted us to do a combined study with ESSENCE..

And how they both compare to 51?.

I think the assumption is there the same. There is one significant exon amenable group that is significantly different those are exon 44 amenable children. As we probably mentioned before.

Axon 44 amenable children have a very different gene type these are the children they tend to have a delay and loss of emulation that can be as much as five years on mean difference than the exon 51 children.

But these children and there's a good reason for that those children have low very low level of sporadic background exon skipping which is natural results in that. Even though I will point out that in Western blot it's often difficult to impossible to see the dystrophin production in those exon 44 kids because it's below detectable levels.

And yet they are actually ambulant for an additional five years compared to exon 51. But as it relates to exon 51, exon 53 and exon 45 children they are phenotypically similar to identical..

And I guess one more question on patients coming online have you noticed any specific trends with respect to proximity to infusion center has been primarily patients that are closer and then moving outwards or had there been patients coming from I guess further from the centers as well?.

No, it's been the same since launch. The patients are really all over. They travel to the neuromuscular center, the center of excellence.

They meet with one of the top KOL's in the country and then they if they don't live close to the center they go back home, the KOL teleconferences with the local physician and that's how the patient starts on therapy. It happens really all across country, it's pretty seamless now..

Thanks for taking my questions..

Thank you..

Thank you. Our next question comes from the line of Tim Lugo with William Blair. Your line is now open..

Thanks for taking the question. You mentioned therapeutic levels are expected from your initial doses of the nationwide gene therapy programs. I think in some of the K9 models we've seen some constructs, so 30% to 50% Micro-Dystrophin production. What are your views that this was going to be mimicked in humans.

When do you think we'll know if this is comparable to let's say a Becker's patient with 30% to 50% dystrophin production and I guess what levels of production would you be happy with?.

Well, I suppose given the early nature of this gene therapy we would be happy when solid identifiable dystrophin as measured by Western blot. But to your very good point you've saw the Genethon paper that they're getting significant expression of dystrophin in dog model. So, we don't know.

We'll see, I mean it's very difficult to take the animal models and then to make the assumption that you're going to get identical results in human. So, I want to be very careful about it. It's going to be a lot of differences here obviously in many ways. But the hope is that you get a significant expression of dystrophin in these Children.

Certainly, the hope and that's the reason that with respect to our Micro-Dystrophin program with Nationwide the goal is to go for potentially therapeutic doses there's a strong belief that Nationwide and it's a belief that concur in that we should try as early as possible to confer a therapeutic benefit these children.

Our hope is that we'll get something that would be predicted from the animal models, but we will see that when we see it..

Understood.

And could you maybe talk a little bit about the IP protection around the nationwide programs and the Genethon programs, it looks like there are some filing patents out there covering Micro-Dystrophin?.

Well, there is a lot. We've got, there is great intellectual property surrounding the programs that we have.

There is certainly in gene therapy, generally there is always the potential for a crowded field as related to intellectual property and if we get to the day where we are in a dispute with someone than and arguing about loyalties it'll be a great day for Sarepta and these children..

Understood. Just like the EXONDYS. Thank you..

Thank you. Our next question comes from Matthew Epler of RBC Capital Markets. Your line is now open..

Great. Thanks for taking the question guys.

So, I wanted to ask about the SRP-4053 and specifically any plan to take the current data to EMA now or are you really looking for clarity on what's going to happen with EXONDYS 51 for sharing data from a second drug?.

For golodirsen in Europe. Well, now we are focusing on Europe right now. Definitely specifically for eteplirsen, EXONDYS 51 US and we're going to go through that process and we'll be - thinking going forward.

One needs to understand that what we have today with respect to golodirsen is very solid data from a biological endpoint, great surrogate endpoints, including expression of dystrophin. With that said, there we've got to talk to Europe there is an obvious pathway in Europe without some additional discussions regarding using surrogate endpoints alone.

So, we'll have to really think that issue through before we approach the agency in Europe about that.

So, I would say for as it stands right now, our goal is as relates to Europe work with the EMA on our file for golodirsen and we'll get a response on that file in the first half of 2018 and as it relates to the FDA to have a good evidence based discussion with the FDA in the first quarter of 2018 and talk to the FDA in a collaborative way about that path forward for golodirsen in United States..

Alright, understood thank you..

Thank you..

Thank you. And our next question comes from the line of Lisa Bayko of JMP Securities. Your line is now open..

Hi, this is John [ph] on for Lisa. Thanks for taking the questions and congrats. Just a couple short ones from me.

You mentioned that average age is 13 years, I was wondering what's the average weight and what's the dropout rates you are seeing is there anything characteristic of a patient that would drop out whether it would be court status of AH or ambulatory status anything you can comment there?.

So, two things. And then Bo you either correct me or add to it. Okay. So, first of all with respect of the weight, we haven't provided weight. We're using revenue as our - surrogate for this, we have been providing ages and we'll continue to do that going forward.

As relates to drop out rates, the assumption at launch was we would probably good drop-out rates you would expect as low as 20%, as high as 30%. We'll looking at getting well over 90%. So, it's very difficult to talk about trends, because we are seeing almost no dropouts at all..

John, there was something, I know that there has been some reports out there that had a drop-out rate listed as higher and yes, I can tell you absolutely is in the single digits and that has not contended that is stabilized all throughout the launch. So, we are very very pleased..

Great. And one last one for me.

Do you know how many state Medicaid plans have formal coverage policies in place and how long does that typically take to get a coverage policy?.

I don't think we have the data in front of us on the number of state Medicaid plans..

We don't go into. A lot of the plans, lot of the states actually have it was just case by case. I mean most of the states do a case by case for these types of patients.

What I can tell you though is that we definitely made effort to target the largest states and the three largest states, the Medicaid states have the broadest policies and then we leveraged these policies with the other state.

California really starting the trend with the CCS guidelines, looking at Brooke, Scale and looking at pulmonary function and then that transferred over to Florida and then that transferred over to Texas and now we've used this across the country. So, many times when states do not have a plan, a policy in place they go case by case.

They look at big states that have a lot of resources like California, Florida, and Texas and they adopt these types of plans..

Got it. That's helpful. Thank you and congrats again on the quarter..

Thank you..

Thank you. Our next question comes from the line of Hartaj Singh of Oppenheimer. Your line is now open..

Yes, thank you. Just had one question then just a very quick follow-up. The one question is Doubt, Bo, Sandy I mean you are going to have the PPMO 51 candidate risk benefit profile looks good assuming it looks good next year. You're going to have potentially a fast following to exon 51 in the clinic.

Just how do you feel about that from a strategic planning perspective? And then I just have a very quick follow up after that. Thank you..

Yes, it would be very good, well hopefully it would be a fast follower if it goes well. Our goal is to continuously improve EXONDYS 51 is conferring significant benefit, physicians are seeing a lot of utility. The issue with our PMO's is clear, this is what's wonderful about the PMO's.

The PMO's are an amazing precision medicine, when the therapy gets into the cell and by the way 100% response rate neighbors study with a look at it as far as getting in the cell.

Gets in the cell, it gets to the light to the pre-messenger RNA, it does precision editing at the RNA level it creates the right messenger RNA, it creates dystrophin, the dystrophin gets to the localized in the right place. Really very elegant.

The issue for the PMO is that they are neutral and so they don't penetrate the cell as well as one would like and therefore the amount of benefit you get is limited by that. It is benefiting these kids, it's not fully transforming these kids.

EXONDYS 51 is wonderful but kids aren't going to envision that they can live 60 or 70 years old and that's our mission. So, the good news with PPMO is that PPMO shows positive signals and it's a fast follower then that's just a wonderful answer for children and for the company, we're going to pursue that aggressively..

Great. And then just a quick follow-up for Sandy. I mean we've got JP Morgan coming up, I assume that Sarepta is going to be there, on the fourth quarter you said you're going to give, you are going to think about next year.

Then are going to follow a strategy similar to this year which is sort of think about all you know what you can do next year sort of conservatively and then maybe as you get more visibility in 2018 keep on upgrading guidance. I mean is that sort of a game plan that you think is sort of has potential? And again, thank you..

Thanks for the question Hartaj. It's a little too early to speculate. We had three very good quarters as we've seen we have updated the guidance as we got more visibility and the trajectory of the launch. And as we get more information, perhaps at JP Morgan we'll be able to provide more guidance. But at this point it's too early to speculate..

Thank you. And our next question comes from the line of Gena Wang of Barclays. Your line is now open..

Thank you for taking my question. The first one is regarding EXONDYS 51 launch in the US. 40% to 50% of patients opted have quit.

Just wondering what is the breakdown of these patients on Medicaid and commercial care?.

Yes, actually Gena. Thanks for the question. But I don't have the actual breakdown from a port standpoint from commercial to Medicaid. But I can tell you it's been pretty universal right from the beginning from right at the start, the ports came out of left field and it was all kids. It was lot of kids.

So, I am assuming the mix is going to be the same, because really, it's about the disease progression with the child and whether they can find the veins or not has been uniform across the spectrum of ages. So, I don't have the breakdown for Medicaid, but you can assume it's roughly split..

I see. Okay and just a very quick question regarding the European filing.

Can you remind us the data that were included in the submission package and for the ex-US launch, which countries you can launch with FDA approval and which countries you will have to depend on the European approval?.

Well, there is two things. First, the application itself. So, the application is a fulsome robust discussion of all the clinical data that we have around EXONDYS 51. So, it is not merely an example of biological data, its functional data. We have obviously dystrophin production is important.

We have good backgrounders on things like exon 44 amenable children alike then we go into clinical data. We've got information on the 6-minute walk, time to loss of ambulation, we have some recent data that we have yet to publish, but we are working on that. But some good data on pulmonary function test it will support the approval process.

It is a very robust discussion of clinical data generally.

As relates to launch countries they're really in Europe in particular there are really aren't any countries that one could commercially launched a drug without an approval so it's a really commercially launch the drug will be depended upon a positive response from EMA and then working throughout the reimbursement issues in individual countries.

In the interim, we do have a Managed Access. The Managed Access Program is broadly across the world we've got European countries, we've got Middle East, North Africa, South America, Asia. But that's not a commercial launch, although there is we do sell the drug in that situation.

It's not a commercial launch for name patient basis and we've rolled this out in two waves over the last couple of months..

Thank you very much..

Thank you..

Thank you. I'm showing no further questions at this time. I would like to turn the conference back over to Doug Ingraham, President and Chief Executive Officer for closing remarks..

Thank you everyone for joining today's call. We look forward to updating you on our ongoing progress in the company -- coming months and with that I would ask you all have a good evening..

Ladies and gentlemen, thank you for your participation in today's conference this concludes the program you may now disconnect. Everyone have a great day..