Ian Estepan - Executive Director, Corporate Affairs Edward Kaye - President, Chief Executive Officer and Chief Medical Officer Sandy Mahatme - Senior Vice President and Chief Financial Officer Bo Cumbo - Vice President, Global Commercial Development.

Ritu Baral - Cowen & Company Hartaj Singh - Oppenheimer Simos Simeonidis - RBC Capital Markets Tim Lugo - William Blair Debjit Chattopadhyay - Janney Capital Markets Steve Brozak - WBB Liisa Bayko - JMP Securities Edward Tenthoff - Piper Jaffray.

Welcome to the Sarepta Therapeutics Fourth Quarter 2016 Earnings Conference Call. My name is Andrew and I will be facilitating the audio portion of today's interactive broadcast. [Operator Instructions]. At this time I would like to turn the call over to Ian Estepan, Executive Director of Corporate Affairs. You have the floor, sir..

Thank you, Andrew and thank you all for joining today's call. Earlier today we released our financial results for the yearend and fourth quarter of 2016. The press release is available on our website at www.sarepta.com and our earnings 8K was filed earlier this afternoon. We filed our 10K for 2016 with the SEC after market close today.

Joining me on the call are Edward Kaye, Sarepta's Chief Executive Officer, Sandy Mahatme, Sarepta's Chief Financial Officer and Bo Cumbo, Sarepta's Senior Vice President, Head of Commercial.

I would like to note that during this call we will be making a number of forward-looking statements about our future business plans, strategy, financial performances and projections, priorities and product candidate development plans including statements about EXONDYS 51 mechanism of action, potential benefits, market size, anticipated changes in conversion rates, first quarter and yearend revenue projections for EXONDYS 51, our beliefs regarding physicians commitment to assist patients navigating the reimbursement landscape, payers understanding related to DMD, EXONDYS 51 eligible patients and patients most likely to benefit, our plans to continue with payers on coverage for access for EXONDYS 51 and our expectation that our efforts will translate into more [indiscernible] on-therapy and position us well for the rest of the year.

Our goal to expand into Europe and DMD population in Europe, our proposed indication expected timelines for and expected EMA evaluation process relating to our MAA submission, our planned activities in support of a potential launch of EXONDYS 51 in Europe including future resolution of intellectual property actions in the EU, our planned named patient program and related timelines, our development and advancement plans for follow-on EXONDYS 51 and clinical trials including ESSENCE, PROMOVI and studies [Technical Difficulty] as well as results we hope to see in expected time lives.

Our timelines and plans relating the post marketing commitments and requirements, the potential of timelines for and our plans in connection with third party partnerships and collaborations, the potential of and planned studies for our PPM platform, the expected timelines for our expected PRV sale and use of proceeds, our expectation regarding the patient journey as more patients get infused with [indiscernible], our beliefs on the impact of genetic testing increases on finding eligible patients for EXONDYS 51 and enrolment in ongoing clinical trials.

Our belief that we are well positioned for future growth and the company aiming to introduce innovative trial design and corporate numbers of patients that will allow for faster regulatory approval and goals of helping as many DMD patients as possible.

These forward-looking statements involve risks and uncertainty, any of which are beyond Sarepta's control. Actual results could materially differ from these forward-looking statements as any and such risk can materially and adversely affect the business, results of operations and trading prices throughout this common stock.

For a detailed description of applicable risks and uncertainties, I urge to review our company's annual report on 10K filed with the Securities and Exchange Commission today as well as the company's other SEC filings.

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. With that, let me turn the call over to Ed for of the corporate and clinical updates.

Ed?.

Thanks, Ian. Good afternoon, everyone. Thank you for joining us today for our financial and corporate update for the fourth quarter of 2016. Today we will provide some high level remarks on the launch of EXONDYS 51 and an update on our clinical programs, our regulatory progress as well as some recent corporate developments.

Sandy will provide an update on our financials for the fourth quarter of 2016 and offer revenue projections for the first quarter and full year of 2017 based on the information that we have available to-date. Bo will review the progress of the launch of EXONDYS 51.

I am pleased that our earnings call falls on this particular day because it coincides with Rare Disease Day. We thank the entire Duchenne community who have tirelessly worked to improve outcomes for patients diagnosed with Duchenne muscular dystrophin. We stand with rare disease community on this quest.

We aim to introduce innovative trial designs that incorporate smaller numbers of patients that will allow for faster regulatory approvals. Our goal remains to help as many DMD patients as possible.

As many of you know DMD is a fatal paediatric [indiscernible] muscular disease caused by mutations in the DMD gene which prevents the translation of a 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. EXONDYS 51 is the first treatment for DMD approved in the U.S. and targets dystrophin deficiency the underlying cause of Duchenne.

EXONDYS 51 is designed to bind to Exon 51 in dystrophin pre-messenger RNA, resulting in the exclusion or skipping of this Exon, and therefore restoring the reading frame and allowing for the production of an internally truncated dystrophin protein.

Approximately 13%^of boys with DMD are amenable to skipping Exon 51 meaning EXONDYS 51 has the potential to treat 13% of patients with DMD. It's important to note that other prescribed therapies for DMD such as steroids are meant to address symptoms of the disease not impact the underlying cause. Although we are still at an early stage of the launch.

We are seeing trends emerge that bolster our confidence in the commercial prospects for EXONDYS 51. Strong patient demand and physician interest has continued into the first quarter. Patient demand has been demonstrated by a steady flow of [indiscernible] from the end of Q4 to the present time.

Physician interest has continued resulting in prescriptions at every top tier center carrying for DMD boys. In addition recent feedback leads us to believe that physicians are committed to assisting patients navigate the reimbursement landscape.

Many of these physicians have been prescribing EXONDYS 51 since launch and along the way have gained important experience and tools to support reimbursement efforts. These efforts along with a normal course of payer determinations has helped to increase conversion rates with February being the highest to-date.

We anticipate conversion rates to continue to accelerate. Based on our activities to-date we 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.

Overall we are quite pleased with the continued progress of the launch, but our work is not done. There remains payers who are not currently providing the coverage for those patients in need of a therapy.

We will work and we will continue to work with these insurers towards a goal of providing these boys the therapy that they have waited so patiently to access. Our goal of treating more patients with DMD starts by expanding into Europe and other regions, in fact more boys are affected by DMD in Europe than in the United States.

On December 19th we announced that our marketing authorization application was validated by the European Medicines Association. The CHMP typically takes approximately 12 to 15 months to review an application. Our rapporteur is Spain and co- rapporteur is Sweden.

Importantly the proposed indication for EXONDYS 51 in the MAA includes all patients aged four or older. We have submitted a robust package of clinical dystrophin and safety data to support the review of the therapy. We believe clinical data will be central in the evaluation of our MAA.

The data we submitted includes four years, six minute walk test data and loss of ambulation data from our 201, 202 study compared to an external control. We have also submitted preliminary inflection data compared to natural history data.

Our safety data base is now comprised of over 150 boys treated, 81 of whom have greater than one year of treatment. Dystrophin data from multiple studies will be provided in support of the drugs mechanism of action. We look forward to answering any questions the CHMP may have over the coming months to help facilitate the review of this application.

We have initiated some key activities in support of the potential launch of EXONDYS, we are close to hiring a general manager in the region and are scaling up manufacturing. We have a number of pending intellectual property legal actions in Europe which are designed to facilitate access to the European market.

We are looking forward to the future resolution of those efforts either through our legal efforts or businesses discussions. We are also looking to expand a number of patients treated in our geographical presence by creating a name patient program. We expect this program will begin in a limited capacity in late 2017.

Now let's shift our focus through our pipeline a follow-on exon programs. Our clinical teams are continuing to work diligently to support the development and advancement of the follow on Exons in our current clinical trials.

On September 28, we announced that the first patient was dosed in the ESSENCE trial, ESSENCE is a randomized double blind placebo control study for patients amenable to either Exon 45 or Exon 53 skipping. We plan to enrol approximately 99 patients in total including at least 30 patients in each of the Exon 53 and also Exon 45 amenable treated arms.

Patients have completed 96 weeks in this study and they may roll over into an open label extension arm in which all patients will receive active treatment. We look forward to the first European site in this global studies to be activated this quarter.

ESSENCE is enrolling well in the United States and we expect the trial to be fully enrolled by the end of this year. We are pleased to announce that the treated arm of PROMOVI our Phase 3 open label study in patients minimal to skipping Exon 51, as completed enrolment.

As a reminder PROMOVI is a 96 week study enrolling patients aged 7 to 13, who can walk between 30 to 450 meters as measured by the six minute walk test. We expect data from this trial on 2019.

Study 4053101 our European Phase 2 study of patients amenable to skipping Exon 53 is also completed enrolment and we expect dystrophin data from this study in 2017. We believe that our clinical candidate that skip Exon 45 and 53 have better Exon skipping efficiency than the [indiscernible] based on invitro testing.

We are eager to see if increased Exon skipping efficiency will lead to the detection of larger quantities of measureable dystrophin. We are working with the FDA on the protocols for dystrophin measurement and quantification. Once the protocol has been finalized we will be able to provide more specific timing of the data read out.

We will be measuring Exon skipping and dystrophin production via RTPCR, immunofluorescent signal intensity, percent dystrophin positive fibers and finally western block. This comprehensive approach is the most robust and thorough way to evaluate dystrophin production.

Continued approval of EXONDYS 51 maybe contingent upon verification of a clinical benefit and confirmatory trials providing clinical data is one of the many goals of our trials and post marketing requirements. The final protocols for the post-marketing commitments are due in the second quarter of 2017.

We are in active discussions with the agency, define trial designs that can potentially yield meaningful data to support access and to move the science forward. Finally before I turn the call over to Sandy Mahatme and Bo Cumbo for financial and commercial updates I would also like to provide an update on our corporate partnerships.

We aim to explore novel approaches for the treatment of Duchenne muscular dystrophin with complimentary technologies to make the most meaningful impact on this devastating disease. Last quarter we entered into an exclusive license agreement for European Summits Utrophin Modulator pipeline for the treatment of DMD.

Utrophin up regulation is not mutation specific and therefore has the potential to help all boys with DMD. Phase out DMD is a Phase 2 clinical trial evaluating [indiscernible] in patients with DMD and it aims to establish proof of concept for this Utrophin modulator.

The 48 week open label trial is expected to enrol up to 40 patients ranging in age from their 5th to their 10th birthdays besides in the United Kingdom and the U.S. Patient enrol is ongoing and data from the first group of 24 week biopsies are expected in the second or third quarter of 2017.

In January we announced two partnerships with nationwide Children's hospital. The first partnership will evaluate the potential of a microdystrophin gene therapy program that utilizes the AAV vector. The second partnership lead by Dr. Kevin Flanigan focuses on [indiscernible] therapy.

This approach attempts to stabilize muscle protein by changing the sugar structure on the protein. This pathway is exciting to us because it has the potential to treat all patients suffering from DMD regardless of their mutation and potentially other dystrophies [ph] that effect the [indiscernible] complex.

We expect both of these programs with nationwide to potentially move into the clinic as early as this year. Moving on to collaborations in September we announced a joint research collaboration with Catabasis to explore our combination drug treatment approach for DMD.

In a designated DMD mouse model increased dystrophin protein expression was observed with an Exon skipping modality in combination with an exit. The company believe that these results warrant for the research. We plan on presenting the data at an upcoming scientific meeting.

We believe our partnerships collaborations and our own developmental pipeline represent the most comprehensive approach to treat DMD. Now I would like to turn the call over to Sandy for an update on our financials for the fourth quarter of 2016.

Sandy?.

Thanks, Ed. Good afternoon. Last week we announced that we entered into an agreement to sell our rare paediatric disease priority review voucher or PRV to Gilead for $125 million.

The sale of this non-core asset will provide an important source of non-diluted capital to support the rapid advancement of our follow-on Exon skipping candidates and next RNA targeted [indiscernible] platform.

Now returning our focus to EXONDYS 51 at this stage of the launch, there are too many variables in flux for us to provide detailed forward-looking guidance for the year. However, as Ed mentioned previously, and Bo will elaborate on, recent trends have provided some visibility into launch trajectory.

Based on data and trends from January and February, we expect Q1 net revenues of approximately 13 million to 15 million which is in line with the consensus numbers provided by FactSet and Bloomberg. We have said that we expect conversions to continue to accelerate.

For this reason based on information we have to-date, we anticipate net revenues for the year will exceed $80 million. We're quite pleased with how the launch is progressing and believe that we are well positioned for future growth.

This afternoon's earnings release provided details for the fourth quarter and full year of 2016 in both an adjusted or a non-GAAP basis as well as GAAP basis. The press release is available on SEC and company websites.

The non-GAAP results we will discuss on this call provide a more accurate picture of ongoing operations and the impact of operations on our cash balance and they exclude restructuring, collaboration payments, as well as stock compensation expenses. Please refer to our press release for a full reconciliation of GAAP and non-GAAP.

In the fourth quarter of 2016, we reported an adjusted our non-GAAP net loss of $38.7 million or up $0.71 cents per share compared to a non-GAAP net loss of $58.3 million or $1.30 per share in the fourth quarter of last year.

Revenue for the fourth quarter of 2016 was $5.4 million, an increase from revenue of $1.3 million for the fourth quarter of 2015 primarily due to sales of EXONDYS 51 following the September 2016 commercial.

Adjusted research and development expenses were $27.8 million for the fourth quarter of 2016 compared to $38.6 million in the fourth quarter of 2015, a decrease of $10.8 million. Adjusted G&A expenses were 16.1 million for the fourth quarter of 2016 compared to $20.7 million in the fourth quarter of 2015 which is a decrease of $4.6 million.

For the full year 2016, we reported an adjusted our non-GAAP net loss of $192 million or $3.94 compared to non-GAAP net loss of $187.9 million or $4.44 per share for the prior year.

The incremental loss of 4 million was primarily driven by increased costs for ongoing clinical trials, partially offset by lower manufacturing expenses because of the capitalization of inventory which occurred upon the approval of EXONDYS 51.

Revenue for the full year 2016 was $5.4 million, an increase from $1.3 million in the prior year primarily due to the EXONDYS 51 launch. Revenue from research contracts and other grants decreased by $1.3 million in 2016 as all of the work related to government grants was completed.

Adjusted research and development expenses were $136 million for both the full year 2016 as well as the prior year. Adjusted G&A expenses were $60.7 million for the full year of 2016 compared to $53.3 million for the prior year which is an increase of $7.4 million.

We expect non-GAAP search and development expenses to grow very modestly compared to 2016, keeping in mind the pre-approval EXONDYS 51 manufacturing costs were included in R&D.

Non-GAAP selling general and administrative expenses is expected to increase by between $20 million to $24 million compared to 2016 due to medical affairs programs and commercial activities. We had approximately $329.3 million in cash and investments at the end of the year which is prior to the sale of the PRV.

In addition, we have prepaid approximately 22.3 million towards the 2017 manufacturing expenses. The uptick in cash burn in Q4 is primarily related to the $40 million upfront payment to Summit Therapeutics. With that I would like to turn the call over to Bo for an update on the U.S. launch of EXONDYS 51.

Bo?.

Thank you, Sandy. Good afternoon, everyone. We continue to be encouraged by the early stages of the EXONDYS 51 launch. We have previously outlined four key initiatives to support a successful launch. Today I'll provide an update on these initiatives.

The first initiative is to work diligently with physicians to identify eligible patients and initiative start forms. At the JPMorgan conference we announced that we had received over 250 start forms in the first full quarter of launch. As expected, there is initial volumes of start forms in the first couple of months of launch.

After this [indiscernible] subscribers have continued to submit start forms for their eligible patients. This has resulted in a steady flow of patients coming into the system week over week. We have seen some physicians submit multiple start forms after they have successfully dosed a patients with EXONDYS 51.

All of the top tiered DMD submitters have now submitted a start form. The second key initiative is to work with payers to facilitate broad access to EXONDYS 51 for eligible patients. Our national account team and medical team have now met with payers across the country that represent over 200 million covered lives.

This is the first time payers have been introduced to DMD and the impact this fatal disease has on patients and their families. As part of these meetings, we shared data on the number of patients allowing plans to better understand the limited impact on their overall budgets.

We also discussed the scientific standards of accelerated approval and the importance of providing access to all eligible patients. We feel that we've made considerable progress with payer discussion since the beginning of the year.

Plans representing over $60 million covered lives have recently established a coverage policy that provides access to patients. We also are aware that some state Medicaid review boards are reaching out to local Duchenne physician experts to help inform policy coverage decisions.

We understand these interactions have led to favorable policies and help to facilitate patients obtaining reimbursement. These recent observations lead us to believe that conversion rates will continue to accelerate.

Third, we are addressing procedural steps such as identifying site of care and hospital formulary approvals to speed the time to treatment. As more patients are infused, we expect patients to have less bearable and more efficient patient journey. We still see about 40% to 50% of patients getting ports prior to infusion.

Although it delays the time to first infusion, we believe this will lead to higher compliance and persistent rates over time. The fourth and final initiative we have undertaken to drive a successful launch focuses on educating physicians, healthcare providers and DMD families about the importance of genetic testing.

Now that there is an approved treatment, it's very important that all patients with DMD know their mutation and know whether the mutation is amenable to EXONDYS 51. We have continued to see an increase of genetic testing over the past few months and increased recognition of the importance of genetic testing.

We believe this will aid in finding eligible patients for EXONDYS 51 and also help patients enrol in ongoing clinical trials. In conclusion, we have made strong progress this quarter and continue to work with those payers with restrictive policies.

We expect these efforts to translate into more boys on therapy and positioning us well for the rest of the year. And with that I'll turn the call back over to Ed..

Thanks, Bo. Before we open the call to questions I would like to conclude by talking about the potential of our PPMO platform which is our next generation RNA targeted therapy.

A specifically designed self-penetrating peptide is added onto the PMO backbone with a goal of increasing tissue penetration leading to greater Exon skipping efficiency and dystrophin production which we hope will lead to better efficacy as well as less frequent dosing for patients.

After many attempts by many other companies, this is the first time a PPMO, RNA target of therapeutic has demonstrated this level of efficacy and tolerability in the non-human primate. We observed widespread and dose dependent Exon skipping in all of the muscles that are involved in the pathology of DMD that is skeletal, cardiac and smooth muscle.

We plan to conduct further toxicological studies in the first half of 2017. Successful completion of these studies could support moving PPMO into the clinic which the end of the year.

Based on the preclinical data to-date we believe that PPMO can potentially broaden our ability to treat various neuromuscular diseases due to its ability to penetrate and attack muscle membrane. We will look to partner indications outside of the neuromuscular space. And with that, operator, we can open up the call to questions..

[Operator Instructions] We will be taking our first question from the line of Salveen Richter from Goldman Sachs. Your line is now open..

This is actually Kerry on the line.

So just starting off regarding the EXONDYS 51 launch, could you provide some color on the mix of ambulatory versus non-ambulatory patients basis starting on the drugs, and what trends are you seeing in the average weight of these patients and just finally how should we think about the rate of new start forms being added as we go forward in the rest of the year.

Thank you..

Yes. I think, you know, certainly your questions are very valid about looking at the percentage of ambulatory. Unfortunately because it's so early in the launch and it's been very, very fluid as far as the population going in, what we can say is we expect, you know, because of the pent up demand, we expect more early in the launch older boys.

But we reasonably have good guidance on the way at this point.

So I think we will know and Bo, any other comments?.

Yes, we really don't have information on ambulatory versus non-ambulatory but we provided information at JPMorgan conference that the average age was '13 and that's held pretty steady..

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

You mentioned a couple times, Ed and Bo that you have prescribers at every top tier center.

How many top tier centers are you guys counting? Can you give us an idea of how your marketing strategy sort of segments centers and can you talk about the number of discreet prescribing physicians and its trends?.

I won't go into details of how many centers. I'll tell you that the top centers, we tier the top two centers which make up roughly 80% of the entire DMD market. Tier 1 which makes up 50% of the entire DMD market, every single center has provided a start form.

Of tier 2 which makes up the other 30% of the top 80% of the market, every center with the exception of one center has sent in a start form. So when you look at the overall market of 80% of all the centers that treat DMD, the largest centers across the country, all but one have start form and all of our top tier centers have start forms..

And trends on number of discreet prescribing physicians?.

We provided some information at JP Morgan. Really it's held steady. There's really not a lot of DMD prescribing physicians. I believe at JPMorgan we said greater than 100 physicians have prescribed. It might be one or two additional physicians because now we have 100% of the Tier 1s and we're up to 96% of the Tier 2 centers.

It's still greater than a hundred. It's pretty much the majority of the KOLs across the country are prescribing..

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

This is Nina on for Brian. I just wanted to ask some questions about the gene therapy program.

So first, if you could just talk a little bit more about the manufacturing capabilities of nationwide in terms of being able to manufacturer the vector and then if you could also just talk a little bit about the differences in the promoter used at nationwide versus the bamboo and solid program..

Sure. I think the reason why we were so interested in nationwide is that they have already done a couple of human gene therapy trials. They are currently doing it for SMA.

They've also done it for [indiscernible] they have and recently really increased their GMV facilities and their ability to produce vector including really having a pretty good control of the number of [indiscernible] that are produced. So because they've demonstrated really nice track record, we feel comfortable that they could supply it.

The other thing that we had liked about the program and as you probably know most of the [indiscernible] are fairly similar what they differ on is the promoter and we like the promoter that currently Dr.

Madala [ph] is using because it also addresses the cardiac and it appears to address is better than the current promoter that are planning to into the clinic in the not too distant future. It is an AVA like it's a RH-72 capsid [ph] so very similar to other AVAs.

Overall I think it was because of the promoter and also the track record of nationwide to be able to produce the vector that gave us some confident that this was a very reasonable program to support..

Our next question comes from the line of [indiscernible] from JPMorgan. Your line is now open..

Quick one for me, in the opening comments you talked about the DMD genotyping and uptick coming to market.

How are you thinking about that curve over the next 12 to 18 months and in what timeframe do you think you could get all the patients sort of in a geneo-type?.

We realized early on that we needed to really focus on genetic testing and importance of genetic testing and as we discussed last year, we did our first wave of market research before approval, actually in 2015, early 2016 and we noticed that genetic testing was hovering right around 60% across the universe and we needed to focus on that.

So we put a lot of emphasis on it. Had materials also worked with PPMD, [indiscernible] and we have made a lot of progress. Actually we announced at JPMorgan that Decode Duchenne had a sever fold increase.

We also saw another genetic testing company that had a five-fold increase and earlier today I think PPMD announced that we just hit our 500th application for Decode Duchenne which is great to see. So the efforts are making an impact.

We also just completed our second wave of market research looking at genetic testing whether all the initiatives we put in place are making an impact and our early research suggests that yes, we've seen a 19% increase in genetic testing since wave 1 market research to wave 2.

So we feel that we're going to wait a full year into the launch and really take a hard look, a real deep dive on genetic testing across the US, but our early market research on wave 2 is suggesting that we are absolutely making an impact and the goal is to be able to find not only boys eligible for EXONDYS 51 but this will also enhance enrolment in our clinical trials.

So we feel very good that we're making a big impact across the country..

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

Just one question, I think at JPMorgan you mentioned that from the time of patient start to when you actually start, you know, putting the revenues on counting revenues, there's about a 60 to 90-daytime frame and you're hoping to accelerate that.

Can you give any color and sort of thoughts, you know, how that is progressing now and how you sort of see that changing over the next few months and into 2018? Thank you..

Yes, we gave early initial guidance of 30 to 90 days and feel over the course of the launch, over the next couple quarters that's going to be in line. Obviously you'll have plans that have a more restrictive process that patients could see over the 90 days. We do see wide variability between approvals.

We've seen some as early as three days and others that have been much longer. We do feel quarter over quarter this is going to the conversion time is going to increase and that's really going with the access that we're seeing right now in the market..

Our next question comes from the line of Simos Simeonidis from RBC Capital Markets. Your line is open..

Ed, in your discussions with CHMP, can you tell us whether there is a sense of urgency on their part, do they understand the severity an unmet need of the disease similar to what happened with the ESDA. And the reason I'm asking this is I know you mentioned that there's a 12 to 15 month timeframe during which a decision could be made.

Do you get a sense that the rapporteur and core rapporteur are working at a pace or rate that could help the company get to a decision perhaps on the early side of that, and secondly, the other question I was going to ask is in your projections for the over 80 million for the year, does that assume that you get all 250 patients that you talked about at JPMorgan more or less? Can you talk about that? Thank you..

Sure. So focusing on Europe, I think there does certainly seem to be an urgency. There has been a number of meetings between patient groups and physicians specifically on DMD. We're fortunate to have one of the CHMP members is a paediatric neuromuscular expert who has been very much involved in this whole process.

I think he's also served as someone who's helped inform and educate his colleagues at the CHMP.

If we look at some of the recent - some of the meetings that recently came out specifically focusing on end points and recall that our end point is the six-minute walk test up to four years of data compared to the external control, one of the things that was recently a couple of weeks ago was stated that by the experts at this meeting was that despite all of the problems and flaws in the six-minute walk test, it still remains the most sensitive test for DMD in boys over 7 years of age and it was helpful to have members of the CHMP hear that kind of recommendation from the experts in the field.

As you know, there is no priority review that occurs in Europe. So the minimal time is 12 months. You know, it's possible it could be earlier but typically it's 12 months and there can be some delays related to the clock stop when questions are asked. So if you look at typical MAA applications, the average is about 15 months.

So unfortunately it doesn't have the same urgency that can happen with priority review in the US but I think there is a sense that the patients are in need. I think the urgency that comes about with the approval of EXONDYS 51 in the United States has led to that urgency. I think so far the discussions we've had with CHMP have been very helpful.

So we look forward to answering their questions and then just finally in regards to your question about how many of these boys we expect to be able to roll over into reimbursement, of those 250 start forms and our expectation is that the majority of them would be rolled over and would be on reimbursed therapy and I think a lot of what we're doing is reaching out to these insurance carriers that are slow to write a policy.

What we're seeing is that there's a lot of information that needs to be given, especially concerning the clinical data if they ask and so I think there's always going to be some delays but I think overall what we're seeing is our expectation is that most of these boys are going to be on reimbursed therapy by the end of the year..

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

For the 80 million number mentioned by you, Sandy, does that include any name patients there [Technical Difficulty] other geographies I believe you said that you have a name patient program up and running sometime in Q4.

And can you maybe compare having a name patient program versus some of the other launches where we've seen name patient sales early on.

Could you just maybe give us some ideas about how meaningful those sales usually are to launches?.

So currently more than $80 million number does not include any EAP sales or ATU sales or name patient program sales nor do they include any sales that we will do through third party distributors, ourselves in other markets that are not subject to any IP issues and where we have freedom to operate.

So there could be some upside there as well but at this point it's too early for us to be guiding on those. We expect to roll out our EAP program probably in Q4 so we might be able to get some pickup from there.

In terms of what the potential upside could be in those markets, again it's too early to guide but if you look at PTC as example, you could use that as a proxy to come up with some numbers..

Our next question comes from the line Matthew Harrison from Morgan Stanley. Your line is open..

This is Vikram on for Matthew. So just a quick one from our side. Could you provide any update as to when we could see dystrophin production data from the EXONDYS 53 studies, the western block data? Thanks..

Sure. So we are doing very complete analyses and so what we have is our muscle map program which is a computerized read to give - present us from positive fibers and also the intensity and then we have new improved RTPCR methodology and then probably the most time we have spent is in our western block methodology.

What we have done is we wanted to make sure that all of these methods would be really appreciated by the FDA and that we wouldn't have any concerns when the data comes out. So we have sent all the protocols and this was also part of our confirmatory studies to make sure they were in agreement to the protocols that we would be doing.

So we submitted those to the FDA. We're waiting to get feedback from them and once we have their feedback, you know, then we're ready to initiate these dystrophin studies and present this from positive fibers.

So I think given the challenges we have with not knowing exactly when we'll have feedback, you know, we're guiding that we will have this data in 2017 but it's a little hard to be more specific..

Our next question is from the line of Debjit Chattopadhyay from Janney Capital Markets. Your line is open..

Firstly is the addressable market closer to 1400 or 1800 and the second being are payers are dragging the feet, are they using the 300K in the line in the sand and finally the progress and timing of the patient reported outcome measures as a gage for clinical impact and how payers are likely to utilize that information..

So Deb, we missed your second question unless the guys get heard. I'm sorry..

I mean so it's been priced roughly about 300,000 based on 55 rate. So I am just wondering if the payers who are dragging their feet on reimbursement, do they want to use that as a kind of line in the sand knowing some of the older boys could be significantly higher than 300K..

And I can start from what we have seen, we have not seen any line in the sands specifically over [indiscernible] size of the child, that has not been an issue. Obviously there are some payers that have restricted the reimbursement to boys who qualify for the trial.

So the ambulatory boys and I think what we're seeing, though, is that when we ask discussions specifically going over, you know, the fact that we have data in boys down to four years of age and boys of up to 21 years of age, we're working to try to have broader really reimbursement and many of the plans have talked with our really our key opinion leaders and when the key opinion leaders are recommending typically what we've seen and this was true for California Children's Medicaid, the recommendation that the key opinion leaders made in California was boys four years of age and up, you know, basically, you know, even non-ambulatory but it was the two issues where if they had a FAC of less than 30 which meant they were likely on a ventilator or if they had a [indiscernible] score of 6 which meant that they had no movement of their upper extremities, those were not recommended.

So short of that, it was a relatively broad inclusion of boys that could be expected to be treated.

And I think, you know, I think just overall, I think what we're seeing is it does, you know, take some effort and fortunately I think we have very experienced medical affairs and account managers, national account managers who are working with the payers and that is moving things over to try to get, more and more of these patients approved.

Finally, the first patient reported outcome that we expect is for the non-ambulatory because that's probably the most pressing need that we need to get out there. That has gone through and we have worked with the patient groups but also with companies that really develop these non-ambulatory patient-reported outcomes.

We also have a patient-reported outcome that we've placed in our essence study which is from the ambulatory boys, that data will be later.

So we would expect that we will be getting and we will be doing this patient reported outcomes on older boys who are non-ambulatory if they agree and that kind of data we're willing to give and we have spoken to the payers about getting that data.

We also are working on getting a registry up and running to try to get as much data collected and again we said we would be willing to share this data and HIPAA-compliant manner with the insurance companies going forward..

And my first question basically in terms of the true addressable market opportunity is that 1400 or 1800?.

Actually we haven't changed our epidemiology of 3500, 5000 we're still evaluating the market making sure that when we do give guidance that we give you an accurate figure of what we really feel is out there. We're still to be quite honest with you, you know, we work with size, we work with the different sides out there and talk about genetic testing.

Some of the sites, some of the KOLs think that they have two or three patients, then they go back in, they pull all their charts, they're working with their staff and they find three or four more.

Obviously, we need to get a really good handle, a few more months under us with the launch before we think we can give you, a full line in the sand of where we believe our epidemiology numbers are. Going to back to what Ed was talking about sort of a line in the sand, he's absolutely correct.

They are not drawing line on the sand, they look at data, they look at publications, they try to get to know the disease and really understand what is dystrophin, what is accelerated approval, how does dystrophin actually help these boys? I can give you an example of a managed care plan that was on the West Coast, a pretty large plan that put a policy in place earlier this year that was very restricted.

It was ambulatory only, it was greater than age 7. It had biopsies in there, we went in, was working with them. MSL team went in, talked to them sort of about our clinical trials and about our data.

They are reviewing the policy, they're actually removing the current policy and putting in a new one in place that will go down less than 7, it's ambulatory, non-ambulatory and they remove the biopsies, same thing happened with the very large Medicaid plan, recently where they put their policy out there, it was going to be restricted to ambulatory only.

It was also age 7 or greater very similar to our clinical trials but this Medicaid pulled in some of the KOLs in the local market, put them actually up during the Medicaid review board and had an open conversation.

The end result came out, and this plan just happened in the last two weeks, the end result came out that they removed the age all the way down to age 4. It's ambulatory and non-ambulatory only.

I mean ambulatory and non-ambulatory so it provides access to all and we're seeing this across the board and actually when you start looking at all the payers, if you take the start forms that I have in the system downstairs and you take a look at who are the top players, the Top 10 players in the country, that represents over 138 million lives and we have access, the children have access to over 100 million lives.

This gives you a really good base to work from over the next couple of quarters of driving pull-through and driving patients on therapies. So feel very good right now of where we're at. We've got work to do with some of the plans but this was the same it would be with any launch. It's just magnified because it's a rare disease..

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

Most of the questions have been asked and answered but one of the items that you were just talking about and going all the way back to orphan drug releases, [indiscernible] through Genzyme, started to find a lot of unexpected positive outcomes. You're obviously genuinely prepared for feedback.

Can you detail in as much specificity as possible how you would start to look and engage and relay the information that you start to see from the KOLs, the patients and coming back to you on things that frankly you wouldn't expect that are positive and how quickly you think you might be able to absorb it and start to make decision based on that?.

This is a something that we have been thinking about and I think and you're right. This was true for Cerezyme and also [indiscernible] and Myozyme.

We did see unexpected outcomes that weren't captured in the clinical trials and I think our approach has been to really listen to the families and the physicians and try to understand what is happening to these boys.

So what we're hearing but we haven't been able to substantiate is that there are actual changes and improvements that have been seen in boys who are non-ambulatory and sometimes happening within months.

And things such as being able to raise their hands over their head which they couldn't before, being able to open up bottles, being able to transfer out of a wheelchair.

So again our expectations for the non-ambulatory were that if we could just slow down the progression of the disease that would be great and I think but now what we're seeing is that there may be some improvements you know quality of life activities that are really important and that's what we're trying to capture in these patient reported outcomes so that we actually know and we're getting the feedback, I will say the mothers are probably the most informative because they observe these boys.

They have very good ideas. And we're also getting it from the caregivers, the physicians, the nurses, the physical therapists who are taking care of these boys on a regular basis.

So that's the kind of data we are certainly trying to incorporate and make sure that we can include this when we talk about - but we have to really substantiate it and get hard numbers, make sure that we understand what is happening. But I think that's a lot of the focus and really that we're trying to do..

Our next question comes from the line of Liisa Bayko from JMP Securities. Your line is open..

Can you maybe just give us an early read on compliance, how that's going and any discontinuations, anything like that..

Yes, so obviously, you know, a lot of the voice of course has been on the drug for a short period of time. We know compliance related to the clinical trials was quite high. I mean higher than certainly I had ever seen before and I think a lot of that is true for an intravenous therapy especially for a disease that is fatal.

So far what we have seen is a very good compliance and the same is true certainly on the commercial. I think what we have seen is that these boys, if they have difficulty with venous access, they do need a port. So we're making sure that they have access to a port because as you know these boys are on chronic steroids.

They have a lot of subcutaneous fat. It's very difficult to try to start the IVs. So that seems to make, that also makes a difference in compliance.

But so far I think, we've really seen a very good track record as far as compliance and given the nature of the disease, we expect that would continue and remember, for clinical trials, we have boys that are over five years of age, you know, two of who were non-ambulatory and that he have remained on the therapy even after the trial has stopped.

So I think the expectation and compliance will be quite high..

Okay. And then what is a fair gross to net assumption to make for this year..

Again it's a little early with a few months into the launch. We'll get a better idea about of gross-to-net in some of the subsequent quarters and we will be able to guide at that point..

Our next question comes from the line of Edward Tenthoff from Piper Jaffray. Your line is open..

Two quick questions if I may.

Firstly, with respect to clinical supply on hand that's already been billed through R&D 2016, how much supply do you guys have on hand? In other words how long should we be anticipating sort of gross margins in 90 range and where do you think they ultimately come out?.

Yes, so over 2016 and late 2015, we have built up a good bit of inventory both for clinical as well as commercial and as a result, the gross margins from a commercial standpoint should be significantly higher it had been projected earlier on.

So what we guided on some of our prior calls was that our cost of goods for commercial will be in the low teens and that we should have inventory in-hand to supply us for at least the next year or so at close to zero cost of goods. Subsequently, we will see our cost of goods in the low teens or so.

In addition to obviously the commercial inventory, we'll have enough goods on hand all of our clinical trials for all our trials for 53, 45 and 51 as well as PPMO..

I'm sorry, you broke up there for just one sec. So it will be low, i.e.

low single digits so you worked through that inventory and how long will that be?.

That should last us approximately a year or so. Had zero or close to zero low cost of goods and in subsequently it should be in the low teens. We'll see that some point in early 2018..

We have a follow-up question from the line of Simos Simeonidis from RBC Capital Markets. Your line is back open..

The stock is down 15% after market and I'm just going to ask this.

I was wondering whether your guidance that you gave for the year for over 80 million, how confident are you about this? Could this be a case where you're guiding kind of low and hoping that this is going to be a good year that you can beat easily? And perhaps another way to ask is how do you get to 80 million from '15? Because obviously the market didn't like what just happened in the past hour.

Thank you..

Yes I mean obviously this has been a difficult concept for us to kind of talk about because we're really, you know, we're only two months into the year. It's challenging to try to figure out.

I think what we've tried to be is, you know, really give reasonable expectations and we did say we expected to exceed 80, but we didn't want to guide into something that we don't have enough data right now to be able to give, you know, really hard figures.

We expect that we'll continue to update as we go farther and farther down the launch and then I think we'll allow us to get closer hopefully to really sound numbers.

But I think what we're trying to do is, you know, just allow people to know where we are at this particular point, two months into the quarter but to stay that overall I think we are feeling very confident..

Thank you. That's all the time that we have for questions today. So I would like to turn the call back over to management for closing comments..

Thank you, everyone for joining today's call. We believe we are building the strong foundation and has all of the elements in place for a successful launch and look forward to updating you on our progress in the next quarterly call. As always but especially on rare disease days we are thinking of those suffering from a rare disease.

Together we hope to make an impact on your lives. Thank you very much..

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