Earnings Labs

Sarepta Therapeutics, Inc. (SRPT)

Q4 2016 Earnings Call· Tue, Feb 28, 2017

$21.12

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Transcript

Operator

Operator

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.

Ian Estepan

Analyst

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…

Edward Kaye

Analyst

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.…

Sandy Mahatme

Analyst

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…

Bo Cumbo

Analyst

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…

Edward Kaye

Analyst

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

Operator

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

Unidentified Analyst

Analyst

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.

Edward Kaye

Analyst

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?

Bo Cumbo

Analyst

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.

Operator

Operator

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

Ritu Baral

Analyst

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?

Edward Kaye

Analyst

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.

Ritu Baral

Analyst

And trends on number of discreet prescribing physicians?

Edward Kaye

Analyst

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.

Operator

Operator

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

Unidentified Analyst

Analyst

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.

Edward Kaye

Analyst

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.

Operator

Operator

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

Unidentified Analyst

Analyst

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?

Bo Cumbo

Analyst

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.

Operator

Operator

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

Hartaj Singh

Analyst

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.

Bo Cumbo

Analyst

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.

Operator

Operator

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

Simos Simeonidis

Analyst

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.

Edward Kaye

Analyst

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.

Operator

Operator

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

Tim Lugo

Analyst

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?

Sandy Mahatme

Analyst

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.

Operator

Operator

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

Unidentified Analyst

Analyst

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.

Edward Kaye

Analyst

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.

Operator

Operator

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

Debjit Chattopadhyay

Analyst

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.

Edward Kaye

Analyst

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

Debjit Chattopadhyay

Analyst

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.

Edward Kaye

Analyst

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.

Debjit Chattopadhyay

Analyst

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

Bo Cumbo

Analyst

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,…

Operator

Operator

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

Steve Brozak

Analyst

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?

Edward Kaye

Analyst

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.

Operator

Operator

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

Liisa Bayko

Analyst

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

Edward Kaye

Analyst

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.

Liisa Bayko

Analyst

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

Sandy Mahatme

Analyst

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.

Operator

Operator

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

Edward Tenthoff

Analyst

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?

Edward Kaye

Analyst

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.

Edward Tenthoff

Analyst

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?

Edward Kaye

Analyst

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.

Operator

Operator

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

Simos Simeonidis

Analyst

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.

Edward Kaye

Analyst

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.

Operator

Operator

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.

Edward Kaye

Analyst

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.

Operator

Operator

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.