Sarepta Therapeutics' (SRPT) CEO Ed Kaye on Sarepta Corporate Update on Accelerated Approval of EXONDYS 51TM Conference Call (Transcript)

| About: Sarepta Therapeutics, (SRPT)

Sarepta Therapeutics, Inc. (NASDAQ:SRPT)

Sarepta Corporate Update on Accelerated Approval of EXONDYS 51TM Conference Call

September 19, 2016 4:00 PM ET

Executives

Ed Kaye - Senior Vice President, Chief Medical Officer and Interim Chief Executive Officer

Sandy Mahatme - Senior Vice President and Chief Financial Officer

Bo Cumbo - Vice President, Global Commercial Development

Analysts

Tim Lugo - William Blair

Chad Messer - Needham & Company

Michelle Gilson - Oppenheimer & Company

Brian Skorney - Robert W. Baird & Co.

Christopher Murray - Nomura Securities

Stephen Brozak - WBB Securities, LLC

Lisa Baker - JMP Securities

Ritu Baral - Cowen and Company

Edwin Doerffel - Wedbush Securities

Debjit Chattopadhyay - Janney Montgomery Scott LLC

Ted Tenthoff - Piper Jaffray

Gena Wang - Jefferies LLC

Joseph Schwartz - Leerink Partners LLC

Ed Kaye

[Call Starts Abruptly] Successfully achieved in Professor Steve Wilton’s lab. So we could not be happier for today’s approval, which allows EXONDYS 51 and exon skipping drug should be available to patients in the United States. This extraordinary community has supported, encouraged, and inspired our work on creating therapies to treat the underlying cause of Duchenne muscular dystrophy.

I would like to acknowledge of all the people living with DMD, and in particular, the 12 boys who participated in our 201, 202 trial, who allowed us to study them for five years and also providing a novel treatment for others, who have been combating this crippling disease.

I was speaking to patients as well as caregivers, physicians, and investigators. This approval is a direct result of your commitment and resolve. We could not be prouder of this achievement and we thank you.

As many of you know, DMD is a pediatric X-linked recessive neuromuscular disease, caused by mutations in the DMD gene, which prevents the reduction of functional dystrophin protein. Dystrophin plays a vital role in the structure, function, and preservation of muscle cells. The progressive loss of muscle function culminates in respiratory and cardiac complications that result in premature death.

Eteplirsen is designed to bind to Exon 51 of dystrophin pre-messenger RNA, resulting in exclusion of this exon during messenger RNA processing in patients with genetic mutations that are amenable to exon 51 skipping. Exon skipping is intended to allow for production of an internally truncated dystrophin protein. EXONDYS 51 is indicated for the treatment of Duchenne muscular dystrophy and patients who have a confirmed mutation of the DMD gene that is amenable to Exon 51 skipping.

This indication is approved under accelerated approval, based on an increase in dystrophin and skeletal muscle observed in some patients treated with EXONDYS 51. Accelerated approval is based on a benefit observed on a surrogate endpoint, that is reasonably likely to predict a clinical benefit, even though a clinical benefit has not yet been established.

Continued approval for this indication may be contingent upon verification of a clinical benefit in confirmatory trials. The recommend – recommended dose of EXONDYS 51 is 30 milligrams per kilogram administered once weekly has a 35 to 60-minute intravenous infusion.

EXONDYS 51 is the only duchenne dystrophin therapy approved in the United States. Importantly, EXONDYS 51 has been approved with a broad label. It does not have any restrictions on age, disease severity, and has no contraindications. Every patient amenable to skipping Exon 51 is a potential candidate for treatment with EXONDYS 51.

On June 6, we announced that the FDA requested that Sarepta provide dystrophin data, as measured by western blot, from biopsy samples from the PROMOVI study. In an evaluation of 12 patients treated with EXONDYS 51, past showed evidence of production of truncated dystrophin protein in western blot after 48 weeks.

The average dystrophin protein level was 0.16% of normal before treatment and 0.44% of normal after 40 weeks of treatment with EXONDYS 51. These results were highly statistically significant with a corresponding P-value of less than 0.008. We are ready to launch EXONDYS 51 to be approximately 13% of DMD patients, who may benefit from Exon 51 skipping.

What is exciting as today’s milestone is, we are far from completing our mission of providing potential treatments for the vast majority of patients with DMD. Our work has just begun. Around the world, there are many patients living with DMD, who do not have a medicine that treats the underlying cause of their disease. As a next step in providing potential therapies to patients with DMD, we plan on filing our MAA for eteplirsen in Europe by the end of the year.

I would now like to return the call over to Sandy Mahatme. Sandy?

Sandy Mahatme

Thanks, Ed. I would like to echo Ed’s sentiments about the significant achievement and the sense of pride we feel in being able to deliver this treatment to amenable patients in the Duchenne community. We are appreciative of the support we received. This drug would not have been approved without a deep understanding of the disease.

As we commenced for EXONDYS 51 launch in the U.S. it is important to understand that DMD is an extremely rare and life threatening disease. In the U.S., an orphan drug is classified as a disease, sorry, and often disease is classified as a disease that impacts fewer than 200,000 patients. An ultra-rare disease affects an even smaller population, and EXONDYS 51 will be treating such an ultra rare population.

We are committed to helping appropriate patients access EXONDYS 51 and there are many factors that contributed to setting the price of EXONDYS 51, including the time and the cost to develop the medicine, the number of patients, the patient’s ability to access the medicine, as well as our commitment to reinvest in research and development of new medicines, such as, follow-on treatments to help other patients who have deletions that are not amenable to skipping Exon 51.

We also considered the novelty and the attributes of the therapy and believe there is a fundamental benefit of treating the underlying cause of DMD in indicated patients rather than just treating the symptoms of the disease. In the U.S., we expect the net annual cost of EXONDYS 51 therapy for patients with DMD to be approximately $300,000. This pricing takes into account the weight-based dosing in EXONDYS 51 label and the typical expected patient age and weight over time.

These estimates are based on our clinical experience in trials, the natural history of the disease, and the expectation that patients will be diagnosed earlier in the course of the disease and be able to go on therapy now at a younger age than patients in our clinical trials. This average cost also accounts for expected compliance rates and mandatory government discounts.

EXONDYS 51 will be available in both a 2ML and 10 ML vial. The 2ML vial will cost $1,600 and the 10ML vial will cost $8,000. Along with the approval of EXONDYS 51, we are pleased to have received a rare pediatric disease, priority review voucher or PRV. This voucher is an incentive to encourage the development of new drugs in biologics for the prevention and treatment of rare pediatric disorders. We can use this voucher to receive priority review on one of our own future market applications that would otherwise be a standard review or we can transfer the voucher to another company as a source of non-dilutive financing.

In order to support a strategic initiative, we intend to offset some of the costs associated with the continued build out of our clinical development pipeline, our manufacturing scale up, and entry into European markets by exploring a sale of the PRV. We will not be providing any financial guidance on today’s call, nor we will be providing any guidance on market assumptions, gross to net, or sales potential.

We will continue to evaluate the possibility of providing guidance over the next few quarters once we gain comfort through the ramp of the launch, market dynamics, and key matrixes.

I would now like to turn the call over to Sarepta’s Head of Commercial, Bo Cumbo to talk about a commercial preparedness.

Bo Cumbo

Thanks, Sandy, and good afternoon, everyone. I’m pleased to be here today to discuss our plans to bring EXONDYS 51 to eligible patients who have equally waiting its approval. We have three key priorities in ensuring the successful launch of EXONDYS 51.

First is, educating physicians and other healthcare professionals about the importance of genetic testing. Now, that there’s a treatment approved for specific deletions, it is imperative that all patients with DMD know the mutations. Personalized medicine will be successful only with the Duchenne community support of initiatives for genetic testing in both the clinical trial and therapeutic settings.

Second is, supporting access for eligible patients. We have therapeutic specialists and account managers around the country, who will begin educating healthcare providers and payers about EXONDYS 51 immediately.

So physicians are informed and prepared to discuss and initiate treatment with their patients, and that patient – payers are aware of the value of EXONDYS 51, who bring their patient population and offer appropriate coverage. We have a very experienced national accounts team that will be calling on payers across the country. As is typical for most rare disease launches, we anticipate to take 30 to 90 days for patients to get coverage. So we expect a lag of this duration before any sales are recognized.

Third is, our establishment of our support program designed to help patients seeking access to EXONDYS 51 SareptAssist. Sarepta’s commitment to patients and families impacted by Duchenne muscular dystrophy extends beyond treatment. SareptAssist consist of an integrated offering of support for patients and providers, including access to dedicated case managers to help patients and their providers understand the insurance process, including claims processing and financial assistance options.

SareptAssist also provides assistance to appropriate patients, including those who are uninsured, underinsured, or who need financial assistance to pay for therapy. For more information on how to apply for any of these types of assistance, or authorized distributors, especially pharmacies can be found at www.SareptAssist.com or by calling 1-888 Sarepta, that’s 1888-727-3782 between 8:30 AM and 6:30 PM Eastern.

Finally, we are ready. Commercially, our team is in place and our supply chain is ready to provide product to patients.

And with that, I’ll turn the call back over to Ed.

Ed Kaye

Thanks, Bo. As I stated earlier, our goal is to treat as many patients amenable to exon skipping as possible. The first step in achieving this goal is the initiation of our ESSENCE Study for patients amenable to skipping Exon 45 and Exon 53.

ESSENCE, a placebo-controlled study of Exon 45 and Exon 53 is now recruiting patients. Based on feedback from the Advisory Committee Meeting and the FDA Draft Guidance, we believe the trials for exon skipping drugs may need to be of longer duration. For this reason, the patients in ESSENCE – in the ESSENCE Study will be studied for up to two years.

We will continue to work with the agency on endpoints and interim analyses that could potentially end the trial earlier if clear efficacy is achieved. We hope this study will establish dystrophin as a surrogate endpoint and will help validate other endpoints besides just kind of walk test that can be used to measure efficacy in a shorter time period.

We hope new endpoints will benefit the entire Duchenne community and be used by other treatment modalities besides exon skipping. If the results of the ESSENCE Study are positive, we hope the information learned will speed and shape the development for the follow-on and rare exons. Our goal is that, today’s approval of EXONDYS 51 will be the first of many to come.

And with that, operator, we can now open the call to questions.

Question-and-Answer Session

Operator

[Operator Instructions] Our first question comes from the line of Tim Lugo with William Blair. Your line is open.

Tim Lugo

Thanks for taking my question. And I just want to pass along my congratulations to the whole team and the patient groups. I know everyone had to move now for the approval.

Ed Kaye

Thanks, Tim.

Tim Lugo

No problem. And I guess to off, how many patients in the U.S. do you believe know their genotypes and have a confirmed mutation for an Exon 15 amenable therapy? And it sounds like your supply chain is ready to launch, maybe can you give us some more color around the manufacturing side, I know that was an area of an improvement sometime ago?

Ed Kaye

Yes, I think, there has been a great deal of effort that we put into the manufacturing. So we have enough supply right now on hand to really supply patients for over a year. And we are developing and continuing to develop our processes, so that we will also be ready for a European approval and rest of world.

So many of the concerns, I think, that had been expressed earlier because of the delays that we’ve had in getting this drug approved, we’ve been able to overcome. So we feel very confident that we’ll be able to supply it. And in regards to how many patients that have been genotype. I’m going to – and Bo Cumbo and his group have been looking at this very carefully.

So, Bo, can you comment on that?

Bo Cumbo

Hi, Tim, yes. Thank you very much for the question. As with other rare diseases, it’s very hard to pin down epidemiology. And it’s a little too early to pinpoint and really get to a specific number of what percentage of the population has been genotype or how many patients are amenable to Exon 51.

But with that said, it is one of our primary focus. We actually have representatives out there, working with the physicians, discussing the importance of genetic testing, and we have many other initiatives around the country working with other groups on exactly what you just brought up about genetic testing and making sure that we do identify everyone that can go on drug.

Ed Kaye

Yes, and say, we’ve been working very closely with Pat Furlong and her group at PPMD and the code Duchenne. So it’s a program that allow support for genetic testing for those people whose insurance won’t pay for it if this problems with reimbursement.

So I think it’s been a very good program. And hopefully, it allows people despite any insurance issues to have their genetic code determine, and then they can see if they potentially could be a candidate for Exon 51 skipping.

Tim Lugo

Okay. Thanks for the question.

Operator

Thank you. Our next question comes from the line of Chad Messer with Needham & Company. Your line is open.

Chad Messer

Great. Thanks for taking my question, and I let me add my congratulations. I think the right outcome came about and obviously to every one of the companies dedication was required for that. I was wondering if you could just talk a little bit about some of the post-marketing requirements that are in the letter from the FDA. They seem to be interested in you guys getting some data on different doses of both the eteplirsen and and the other exon skipping drugs. What can you tell us about how you’re thinking about those studies?

Ed Kaye

Yes. And I think, obviously, we’re still in the preliminary stage as we haven’t had any direct discussions with the FDA, specifically about the protocol, and we will be working with them. I think the emphasis in our discussions have been that they would like to look at higher doses and to look at the comparison. And obviously, these would also be launch studies.

We do have a fairly way in helping to develop the protocols would be looking at novel endpoints. So I think, we’re firmly committed to working with the FDA to make sure that we get them the clinical information that they need to eventually give us a long-term approval. But I think in a lot of those details, we’ll still be working out as far as that’s concerned. And then really the protocols will be finalized probably in mid-2017.

Chad Messer

Great, thanks. And I guess just to be clear, we should not think of PROMOVI and ESSENCE as sufficient efficacy studies for full approval?

Ed Kaye

Well, I think, it’s really the entire collection of data. It’s the whole post-marketing requirements that will be included. And obviously, it will take us sometime to get all that information. So I think, we will certainly get that for the FDA. But again, this is a – this is going to be a long process.

Chad Messer

All right, great. Thanks.

Operator

Our next question comes from the line of Michelle Gilson with Oppenheimer. Your line is open.

Michelle Gilson

Hi, guys, congratulations. I was just wondering, is there for SRP-4045 and SRP-4053, is there a path towards an NDA based on dystrophin for those follow-on exon skipping molecules, as well? And I know the post-marketing requirements included a study for them as well.

Ed Kaye

Yes. So I think, that’s a very good question. Obviously, the way the study was set up, it was not only to measure dystrophin, but to have a clinical endpoint. And certainly, it’s something that could be discussed and that would have – we have not had specific discussions about the approval for 45 and 53. But that’s certainly a possibility for discussion.

Michelle Gilson

Okay, great. And then what is the average weight assumption that you guys have for your pricing?

Ed Kaye

So the average weight that we’re basing it on is 25 kilos. And that was within the range of what we were seeing in our clinical studies. But the reason why we’re focused on 25 is that, we expect with greater awareness and earlier diagnosis and especially with the advent of new more on screening. But long-term, this would be the weight that we would expect overall.

Michelle Gilson

Okay And then do you have a ballpark number on number of patients you guys have already identified?

Ed Kaye

No, not yet, and we’re not giving guidance on that. But obviously, Bo and his team have been working very hard to try to identify patients. And that’s something that will give a little guidance on what we have from our numbers.

Michelle Gilson

Okay, great. Thank you so much and congratulations again.

Ed Kaye

Thanks, Michelle.

Operator

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

Brian Skorney

Hey, good afternoon, guys. And I want to express my congratulations too on the approval. I guess, the first question I have is in terms of the ongoing clinical studies with eteplirsen, PROMOVI, and non-ambulatory study, the younger kids, are those going to be continued to be run under in R&D expense, or is there a plan to convert those over to commercial drug?

Ed Kaye

Yes. So I – so when we – it’s really a mixture. I think one of the things we’re looking at is, of course, the – PROMOVI study is also important for our European filing and we’re – we want to make sure that we have the information. The younger patients from the 203, there’s some very important MRI data that we hope to get out. I think in some of the older boys that were on drug actually those boys will be transferred over to commercial drug just because that was really a safety study.

So, I think, what we’re looking at is for those boys, where we don’t need the clinical information for regulatory purposes, they can be – they will be transferred over to commercial therapy.

Brian Skorney

Got it. And then really quick, I don’t know I missed if you gave an update in terms of the discussions with MAA, and at what point you think you’d be in a position to submit MAA there?

Ed Kaye

Yes. So we are shooting for end of year submission at the MAA that will be dependent on, obviously, we’re having discussions, we’ll have a discussion with the raptor. We’ll make sure that we have all the requirements. But internally, our goal is to submit this by the end of the year.

Brian Skorney

Great. Thanks a lot and congrats again.

Ed Kaye

All right. Thank you.

Operator

Our next question comes the line of no more Christopher Murray with Nomura Securities. Your line is open.

Christopher Murray

Hi, guys. Thanks for taking the questions and congratulations. Maybe a follow-up on some excellent questions by Michelle Gilson. I was just wondering, how far you with respect to enrolment in PROMOVI? How many patients maybe have enrolled? Could you provide an update?

And then secondly, any plans to submit for approval of Exon 53 based on the 48-patient trial? And when do we see data on that, again? Thank you.

Ed Kaye

Okay. So if we look at that PROMOVI, we’re continuing to enroll on that. And we expect that, we will be fully enrolled by the end of the year for our treatment end. We’re a little behind and the non-treatment end has always been more challenging when and we’ll decide what would be an adequate number of patients. But we’ll – but it’s continuing and and we expect enrolment by the end of this year.

In regards to the study that you’re talking about the 4053-101, which is done in Europe, which is I assume you’re talking about for Exon 53. So we have 25 patients that are currently in under treatment for that and they will be in by sometime in first quarter. They should be completed the 48-week. We have 14 boys who are currently in the untreated group for that.

So sometime by the time we review the biopsies, we should have some information for that hopefully in the first-half of next year as far as for dystrophin.

Christopher Murray

Great. Congrats again.

Ed Kaye

Thanks, Chris.

Operator

Our next question comes from the line of Steve Brozak with WBB. Your line is open.

Stephen Brozak

Hi, obviously, congratulations. This is a real testament to everything your entire team has done. I’ll just ask one quick question. Given that you probably treated more patients, given the pool that you’re talking about than most, how is your process going to change in terms of what’s the difference going to be between what you’re doing and the operations post-approval that you’ve got more insight than a lot of folks do?

Ed Kaye

Are you referring specifically to manufacturing, Steve?

Stephen Brozak

Manufacturing everything across the board?

Ed Kaye

Well, one of the things that I think we’ve been able to do is been really able to assemble a very sophisticated and experienced team. So from the standpoint of the commercial organization, medical affairs under Dana Martin, all of our patient case managers and reimbursement specialists. That’s been in place for sometime and really we’ve been ready to go for six months, if we based on the first PDUFA date of end of February.

So that’s been there. That group is ready to go. What we’re really focusing on also is kind of beefing up our research and development group, because we’re not only interested in obviously what we’re doing now for 51 and 45 and 53, in but we’re very committed to the follow-on sequences for the other exon skipping drugs. In our next-generation chemistry, the PPMO is progressing quite well. So, we really have a platform within a platform.

And so, I think, the focus will be is on making sure that we execute on and focus on DMD and make sure that we’re really committed to get everything we need for all, not only for the first three drugs, but for all the following exons and the for the next generation of exon skipping drugs.

Stephen Brozak

Great. So the takeaway is you’ve been ready for six months.

Sandy Mahatme

That’s right.

Stephen Brozak

That’s something that everyone…

Sandy Mahatme

We are ready to go.

Stephen Brozak

Well congrats. I am delighted to see that you are ready to go, and that you’re going.

Operator

Thank you. Our next question comes from the line of Lisa Baker with JMP Securities. Your line is open.

Lisa Baker

Hi, my congratulations as well and thanks for taking the question. Can you talk a little bit more about just your preparedness with for reimbursement and how long you might think it might take to get patients on therapy and what sort of support networks you will have for that, for that patient et cetera.

Sandy Mahatme

Yes Lisa, I’m going to let Bo answer that one because he’s been actually working very hard on this for the last six months.

Bo Cumbo

Hi, Lisa. Thanks for the question. I think as with other rare diseases and we’ve taken a look at many rare diseases and I also have a very experienced national accounts team that has launched many drugs within, not only rare disease, but other infused products. And we believe that the average is going to take anywhere from 30 days to 90 days for the payers to actually reimburse and that’s really depending on the plan, whether it is commercial plan or maybe some other Medicaid state. So, the average overall is 30 days to 90 days, so as start forms come in downstairs to our case managers they will start the process and then we can expect reimbursement and patients can sort of expecting to be infused somewhere in that range of 30 days to 90 days depending on the plan.

Lisa Baker

Okay. And can you talk about how you will be distributing the drugs?

Bo Cumbo

Yes we have a very close network. We have the distribution already in place. Whether it is going to be distribution to the hospitals, if it’s infused at the hospital location or if it’s infused, if it is a provider or the family wants to have their infusion at home. We have that capability as well when we set the distribution to infuse at home. We’re using very tight closed specialty distributors and special day infused pharmacies that have done many, many rare disease launches in the past and are working with all the other rare disease companies.

Lisa Baker

And you’re a patient assistance, you know what I’m talking about is like for reimbursement and whatnot is that something you will have in-house or are you outsourcing that function?

Bo Cumbo

We felt that it was very important when we were building the model is that we stay as close to the families and the provider as possible, so we built our hub in-house and these are rare disease expert case managers that came from Shire, Biogen, Genzyme et cetera. So, we built the model in-house, we have an individual that heads up the patient support programs that came – he built Biogen, Shire, Vertex et cetera and he is a full-time employee here. So, we feel we have one of the best patient support hubs and services out there in the industry.

Lisa Baker

Okay that’s great, thank you. And then just I noticed on your post approval commitments with exploring on once-daily dose, can you maybe talk about what kind of safety information you have on those that frequent and sort of drug supply to execute that trial?

Bo Cumbo

So Lisa we – that was just a suggestion, an example, but the actual protocol has not been delineated by the FDA, so basically we have commented that we will compare the 30 mg per milligram per week to a higher dose, but the details have to be worked exactly where that’s going to look like.

Lisa Baker

Okay and then just final question, can you remind us how many reps you’ll have just for modeling sizing purposes, thank you.

Bo Cumbo

Yes, we are really not going to talk about exactly how many reps, I mean there is still very interesting market and competitive market and landscape, but I can tell you that it’s very similar to other rare disease companies with actinology at the same time size.

Lisa Baker

All right. Thanks a lot.

Operator

Our next question comes from the line of Ritu Baral with Cowen and Company. Your line is open.

Ritu Baral

Hi guys congratulations. I think today we will all remember for some time. I wanted to ask about the post approval study as well, I noticed that the endpoint is the Northstar, I wanted to know what conversations that you’ve had with the regulators either FDA or EMA on acceptance of that endpoint, if that’s going to be part of a ESSENCE and what would constitute success or failure of that Phase 4?

Bo Cumbo

So again the clinical endpoints and again just want to remind everyone that the details of the protocol haven’t been completely worked out and that is obviously that something that will take months to do.

We do have a Nortstar that is in all of our studies and all of our trials, but what would constitute success, especially over two years, you know we still have two walk out some of those details, but again we are looking in a number of different endpoints in the younger boys, we’re looking at the 100 meter walk/run to see how well that correlates, we are looking at the 10 meter walk/run, so obviously we are continuing to collect a lot of data. As we get more and more data we will compile it and then we will have discussions with the agency to make sure we have a trial that will meet their needs. So, we will have further details once we get closer to actually finalizing the protocol.

Ritu Baral

Is there is any sense that the agency is moving away from 6 minute walk for any reason?

Bo Cumbo

Not that we have for specifically, I think there is an interest in exploring other endpoints. Obviously, the six minute walk test does not work for a younger population and there is interest and things like the Bayley developmental scale, if you’re talking about boys or younger than four years of age, so there’s a lot of exploration to other type of endpoints and I think one of the things that will be interesting is how well these endpoints correlate and things like dystrophin. So, I think we are in the early phases of looking at some of these endpoints and as we go through, we will obviously want to make sure that we have the most sensitive and the most robust endpoint that will demonstrate clinical efficacy.

Ritu Baral

Got it. One quick question actually from a client over email, are patient’s going to be able to receive half of vial either of the 10 ml or the 2 ml, it kind of seems like half of vial might be unused or partial vial might be unused depending on the weight of the patient to get the correct dose?

Bo Cumbo

Hi Ritu, this is Bo. No, this is why we have 10 and a 2 ml vial to make sure that wastage is minimized to at least one ml.

Sandy Mahatme

And typically Ritu and this is what I’ve done and as far as clinical practice in using some of the IV infusions, it’s typically we will round up or round down depending on the vial, it is not like you’re going to be wasting obviously drug. And so the physicians are pretty good about making sure they get a dose and you can make it – you round it up or down to make sure you get the right dose without wasting valuable drug.

Ritu Baral

Got it. Last question, thanks for your patience. What is the strategy in Europe for approaching BioMarin about in the IP situation there?

Sandy Mahatme

Well you know if we look at the IP in Europe, obviously we are still kind of waiting for the appeal of the opposition decision. We do know that there is a backlog in Europe right now as far as the appeals cases, so we don’t have a specific time. However, our expectation is by the time we are done with our application for the MAA that will very likely have a decision at that time. We’re going ahead with our MAA application. We’re not waiting for working on the IP, and obviously we will look at a number of different potential solutions, but we think the first solution could very well be legal solution to this.

Ritu Baral

Got it. Ed, Sandy, and Bo congratulations and Ian thanks for putting up with us the whole time.

Bo Cumbo

Thank you.

Sandy Mahatme

Thanks Ritu.

Operator

Our next question comes from the line of Heather Behanna with Wedbush Securities. Your line is open.

Edwin Doerffel

Hi this is Edwin, I’m on the call for Heather. Congratulations on the consolidated approval. I just wanted to ask about COGS, what your COGS are when you find to recognize them? Thank you.

Sandy Mahatme

Ed you want to talk?

Ed Kaye

Sure. Hi, Edwin thanks for the call, thanks for the question. We have multiple quarters where we will be selling zero cost or low cost products. So once our low cost period is over we anticipate that the COGS is a percentage of revenue should be somewhere in the low teens.

Edwin Doerffel

Okay thank you and lastly if you could just provide a little bit of more color on your manufacturing and how do you plan to keep pace with supply in the market? Thank you.

Sandy Mahatme

Yes, I think as Ed mentioned before, we have sufficient supply and capacity to supply the US market for over a year, and we have various initiatives underway to expand capacity to supply both the U.S., as well as XDS market both from a clinical perspective, as well as a commercial perspective.

So, we have various manufacturers in the U.S. from a sub-unit and API fill and finish perspective and we’re looking at backup manufacturers as well, which we will be bringing them on stream gradually as we get a better read in Europe. And simultaneously, we will expand from what we have right now, which is midscale manufacturing capacity to a larger scale. But again the key part here is, it’s going to be a very phased-in approach depending upon the epidemiology, as well as the feedback from Europe in terms of the EMEA submission.

Edwin Doerffel

Thank you so much and congrats once again.

Sandy Mahatme

Thank you.

Ed Kaye

Thank you.

Operator

Thank you. [Operator Instructions] Our next question comes from the line of Debjit Chattopadhyay with Janney. Your line is open.

Debjit Chattopadhyay

Hi good afternoon guys and thanks for taking my question. One quick one on the European strategy, the patients who were and rolled in the PROMOVI study not just the 13 patients from which we have the dystrophin data, can the overall patients who have completed 48 weeks that data be used to augment the European filing?

Sandy Mahatme

We have not had that specific discussion with the raptor, but we are looking at – things like that to see if we can augment the natural history. Obviously there is a lot of natural history that will be available. So that’s something that we will have a discussion about.

Debjit Chattopadhyay

And in terms of alternative dosing schedule that the FDA is asking you to look at, does it have to be a daily dosing schedule or could it be a 50 mix per kig [ph] weekly as opposed to some sort of a daily schedule? Thanks so much and congratulations

Sandy Mahatme

No, it doesn’t have to be on our daily schedule and I think the detail about what an alternative dose should be for a comparative group, we have to work out those details.

Debjit Chattopadhyay

Thank you so much.

Operator

Thank you. Our next question is comes from the line of Ted Tenthoff with Piper Jaffray. Your line is open.

Ted Tenthoff

Hi, thank you very much and my congratulations too on the approval. I’m sorry, it took a little bit of time to dial into the conference call, could you repeat what the average price assumptions are and what is your expectation for gross to net discount?

Sandy Mahatme

So Ted it’s Sandy. We indicated previously that we are not providing any financial guidance on the call, including gross to net. In terms of the drug pricing, we had indicated that it’s $300,000 per year for 25 kg kid on average.

Ted Tenthoff

Excellent, thanks for that and most sincere congratulations to a very great day for Duchenne boys.

Sandy Mahatme

Thank you, Ted.

Operator

Thank you our next question comes from the line of Gena Wang with Jefferies. Your line is open.

Gena Wang

Thank you for taking my questions and I wanted to add my congratulations as well. So, I have a question regarding the post marketing clinical trials, so just wondering I think, quite a few being already asked, just wondering, it seems like FDA wanted you to conduct two year randomized double-blind control trial of a top lesson in Exon 51 skipping, so one dose would be 30 mg per kid as approved dose and the other dose, I think many people already asked could be significantly high exposure, so what would be considered approvable or like considered as a positive in this situation? Would that be like the high exposure, those has to show better north star results versus current approved dose?

Sandy Mahatme

Well again. I think the details obviously need to be worked out and what would be considered different, I think the issue really comes in is that really the purpose of that study is, does a higher dose give a better outcome and so obviously this is something that we could have to work out the details with the FDA. And certainly it’s – there will be – a lot of negotiations have to occur and our protocol has to be finalized by the second quarter of 2017. So, we will have many discussions with them and want to make sure that we’re providing them with a study that will give them the information that they need.

Gena Wang

I see. I will just have a quick follow-up regarding the is ESSENCE study or Exon 45 in the 53, but we are left right now with one dose and compared to the placebo and it seems FDA also want you to have a higher dose, would you also incorporate like similar dosing or higher exposure dose into the ESSENCE trial?

Sandy Mahatme

Yes so, again that’s a point of discussion that will occur. The 45 and 53 also have a higher efficiency in regards to exon skipping, so we’d have to determine what that higher exposure would be. So those are some of the details that we haven’t worked out as yet.

Gena Wang

Okay, thank you.

Operator

Our next question comes from the line of Joseph Schwartz with Leerink Partners. Your line is open.

Joseph Schwartz

Thanks a lot and congrats as well. I was wondering if you could talk a little bit about your pricing assumptions and how much of the $300,000 year price that you expect the average patient to consume, do you expect to carry over into additional exons, in other words are you anticipating already similar price level and the additional exon because I think many of us had expected a higher price? Thanks.

Sandy Mahatme

Well I think Joe there is, obviously an important question. And I think what we’re trying to do is be as thoughtful as possible, obviously we are in the middle of the range of the rare disease population for these orphan indications and we did take into consideration how much R&D was spent prior to this time, obviously we have a large number of post marketing requirements that we are doing now and we will continue to do and we are committed to continuing to do research in the follow-on exons where we are committed to looking at our next-generation chemistry and so we’ve really included all of this in our pricing considerations and given the sensitivity to pricing we’ve tried to be very what we think is reasonable given all of the costs for this and even the innovation that goes into manufacturing.

So, it’s – we will be, the focus is really trying to make sure that all the patients can gain access to it and that’s why some of the programs to help patients go through this for the reimbursement to make it as easy as possible, and then really investing to make sure that we’re going to capture all of the boys not just the 51 [indiscernible] boys, but boys who could benefit the potentially up to 80% of boys who could be available to exon skipping. So that really is of focus and that went into considerations of the pricing.

Joseph Schwartz

Thanks for taking my question.

Operator

Thank you and we have a follow-up from the line of Tim Lugo with William Blair. Your line is open.

Tim Lugo

Hi thanks. Just one quick follow-up. You mentioned the prior year view voucher in your line intro comments, are you looking to monetize that soon or just what are your plans around that?

Ed Kaye

Yes, Sandy do you want to comment.

Sandy Mahatme

Hi, Tim. So, yes as I said in my prepared remarks we do intend to sell the product we give vouchers as a source of non-diluted financing and we’ve already reached out to a bunch of potential buyers, so that’s something we are very actively pursuing right now.

Tim Lugo

Okay. And maybe if I could sneak one more and sorry about that, but what are the average weight of patients in your non-ambulatory study?

Sandy Mahatme

I don’t have that offhand, but obviously these are older boys. We have some boys that are up to 21, so the weights are obviously – significantly higher than that, but right now I actually – offhand I don’t have those numbers in front of me.

Tim Lugo

All right. Thanks for the questions and congratulations.

Operator

Thank you. And I’m showing no further questions at this time. I’d like to turn the call back to Dr. Edward Kaye for closing remarks.

Ed Kaye

Well, I just want to really thank everybody for their support over, really a number of years and all the ups and downs of Sarepta and I think if we think back, this disease was described over 150 years ago and people have been looking to try to come up with some type of therapy and also this last Labor Day marks 50 years since the Jerry Lewis MDA Telethon tried to get research funds for Duchenne muscular dystrophy and I think it’s really a commitment on the part of all the efficacy groups and the patients and the positions physicians and really helped work with us to get this done, and this has been 50 years in the making. And I think it’s a great day for the boys with Duchenne and we’re very happy to be part of it. So thank you everyone for participating in this phone call.

Operator

Ladies and gentlemen, thank you for participating in today’s conference. This does conclude the program and you may all disconnect. Everyone have a wonderful day.

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