Emily Hill – Director, Investor Relations Stuart Peltz – Chief Executive Officer Shane Kovacs – Executive Vice President, Chief Financial Officer and Head of Corporate Development Mark Rothera – Chief Commercial Officer.

Simos Simeonidis – RBC Capital Markets Alethia Young – Deutsche Bank Geoff Meacham – Barclays Tazeen Ahmad – Bank of America Chris Marai – Oppenheimer Anupam Rama – JP Morgan Jeremiah Shepard – Credit Suisse Kumar Raja – Citi Ritu Baral – Cowen and Company Debjit Chattopadhyay – Roth Capital Partners Heather Behanna – Wedbush Securities.

Good day, ladies and gentlemen and welcome to PTC Therapeutics Q1 2015 Earnings Call. At this time, all participants are in a listen-only mode. Later, we will have a question-and-answer session and instructions will follow at that time. [Operator Instructions] As a remainder this conference call is being recorded.

I’d now like to turn the conference call over to Emily Hill. Please proceed..

Thank you. Welcome to today’s conference call to discuss first quarter 2015 earnings results and the commercial launch of Translarna. Before we start, let me remind you that today’s call will include forward-looking statements based on current expectations.

These include statements about our future expectations, regarding clinical developments, regulatory, and commercialization timelines, the potential success of our products and product candidates, addressable patient populations and financial projections.

Actual results may differ materially from those indicated by these statements as a result of the variety of risks and uncertainties including those discussed under the headings. Special notes regarding forward-looking statements and Risk Factors in our most recent Form 10-K which is available from the SEC or our website.

Such statements represent our judgment as of today and PTC undertakes no obligation to publicly update any forward-looking statements except as required by law. With that, let me pass the call over to Stuart..

Thanks, Emily. Good afternoon. Thank you for being on the call today, we’re off to a strong start this year. We’ve had several significant accomplishments since our last call two months ago. We continue to see growth in our launch of Translarna the first treatment for Duchenne muscular dystrophy.

We have updated the share on our SMA program, which was recently highlighted at the American Academy of Neurology. Additionally, we recently moved our cancer stem cell program, targeting BMI1, into the clinic. I will discuss each of these points during today’s call. Let’s cover Translarna first.

We are proud to have launched the first approved treatment of nonsense mutation Duchenne muscular dystrophy to patients with this devastating disorder. Translarna targets the underlying cause of the disease. As a reminder, Translarna was approved by the European Medicines Agency last August.

We remain committed to bringing Translarna to patients as quickly as possible. We assembled the team with substantial experience, commercializing product in the ultra orphan-disease space.

We have submitted pricing and reimbursement dossiers in key European countries the rollout through the remainder of 2015, will be staggered on the country-by-county basis in Europe and outside of the EU.

I’m extremely pleased with the continued ramp up we are seeing in our commercial launch for Translarna, as we remain highly focused on bringing this new therapy to DMD patients across Europe, as well as numerous other countries around the world.

As of April 30, we now have 82 DMD patients, on commercial Translarna therapy through either reimbursed early access or commercial sales, almost doubled the number of patients we reported at the end of February, a little more then two months ago.

We have been shipping products to patients throughout Europe including Germany, Spain, France, Italy, Greece and most recently Austria. Outside of Europe, we are shipping products to Turkey, Israel and Columbia. Feedback from the field so far remains positive. We are please to have seen such a strong reception and uptick in the last nine weeks.

As a remainder, during our plan 2015 quarterly call, we intend to continue providing you with the number of patients on commercial Translarna therapy. Beyond 2015, we will evaluate the metrics that would be most useful. We have also begun building out our U.S. commercial infrastructure in anticipation of a Translarna launch in the first half of 2016.

We recently hired Eric Pauwels, as head of commercial operations for the Americas. Eric brings a wealth of commercial experience having launched multiple ultra orphan disease products in the U.S. and internationally. We are pleased to have him on our team.

As a remainder, we began submitting our rolling NDA to the FDA for Translarna for the treatment of nonsense mutation DMD in December of last year.

This will allow the FDA to review the majority of our materials ahead of our commission of the data from our confirmatory Phase 3 ACT DMD trial from which top line results are expected in the fourth quarter of this year.

While we are very excited about the current commercial opportunity for Translarna and DMD, we are equally focused on bringing Translarna to patients in other indications with high unmet medical needs as well. As you know, Translarna is also advancing in nonsense mutation cystic fibrosis.

We initiated the second Phase 3 trial with Translarna ACT CF in June of 2014. Enrollment is on-track to be completed by the end of the year. We anticipate results from ACT CF will be available in the second half of 2016. Modeled after our successful DMD experience, we intend to apply for early approval in Europe once ACT CF is well enrolled.

We anticipate submitting this application in the second half of 2015. Because Translarna is already approved in Europe, the CHMP review process maybe completed in as little of three months. This can mean a potential Translarna launch in CF across Europe in 2016.

Given Translarna’s mechanism of action it has the potential to address a wide array of genetic disorders caused by a nonsense mutation. On average an 11% of every monogenic disorders is caused by a nonsense mutation. There are now over 30 publications demonstrating Translarna’s activity in a number of preclinical nonsense mutation disease models.

We have been reviewing this publication as part of our life cycle management strategy with aims to realize Translarna’s application as a precision based medicine with a potential to treat patients across an array of genetic disorders.

We are pleased to announce that the next indication we will investigate with Translarna is nonsense mutation aniridia. One of the 30 publications that I referenced earlier highlighted Translarna’s ability to promote nonsense mutation readthrough in an aniridia mouse model.

Aniridia the genetic disorder caused by mutations in the PAX6 gene, which occurs in about one of 60,000 births. Congenital aniridia is a rare devastating disorder of the eye, resulting from disruption in the development of multiple parts of the eye, including the iris, cornea, lens, retina and optic nerve.

From new and important pre-clinical results used in Translarna in the nonsense mutation PAX6 aniridia mouse model we reported yesterday May 3 at the Association for Research in Vision and Ophthalmology conference.

In this model, translarna inhibited disease progression reverting congenital ocular malformations in the cornea, lens and retina and restoring the electrical responses of the retina measured using the electroretinography. We plan to initiate the Phase 2 proof-of-concept study with translarna, in aniridia before the end of the year.

This year we look forward to advancing other translarna clinical program as we investigated additional indications. As a reminder, we are initiating the Phase 2 proof-of-concept study in tranlarna in MPS 1.

Translarna is a small molecule which can cross the blood brain barrier, existing treatment for MPS 1 do not address the central mucous system, manifestations of this disorder which can be quite severe. We expect to have data from the Phase 2 study in MPS 1 by the end of the year.

We also continue to evaluate additional indications for translarna, we use multiple metrics to prioritize indications including those with high unmet medical needs, clinical endpoint for study design and established animal model.

It is our goal to maximize translarna’s potential as a product and a pipeline and bring translarna to as many patients as possible, who suffer from rare and neglected disorders. Let me now shift to the SMA program, which is a partnership with Roche and the SMA foundation.

SMA is a genetic neuromuscular disease caused by missing or defective SMN1 gene which result in reduced levels of the SMN protein. So some molecule SMN2 gene predominantly generates a shortened messenger RNA due to alternative splicing and only produces small amount of the SMN protein.

Insufficient levels of SMN protein are responsible for the loss of motor neurons within the spinal cord. We need the muscle atrophy and death in instance and path of in its most severe form. It is estimated that this devastating disease affects one in every 11,000 newborns there are no marked therapy for SMA.

RG7800 is an orally available small molecule, being investigated for its ability to modify the splicing of the SMN2 RNA towards the production of full length messenger RNA.

As you may recall, pre-clinical studies in the animal models of SMA demonstrated that the full length SMN mRNA let to an increase in the SMN protein with significant benefits on survival and motor function. A tremendous amount of effort in resources has been put into the SMA program by all three collaboration partners.

Recently, result to a percentage from the Phase 1 study in healthy volunteers at the emerging science session, at the annual meeting of the American Academy of Neurology. These results showed a dose depended effect of RG7800 on SMN2 splicing demonstrating proof of mechanism.

These data are meaningful because both SMA patients and healthy volunteers shared the same SMN2 gene. After single dose, there was approximately an 80% increase in the full length mRNA production.

As a reminder, our previously published data in animal models of SMA demonstrated restoration of full motor function and survival at SMN protein levels below those in healthy animals. Based on the Phase 1 results, a Phase 2 multi-dose study MOONFISH was initiated last November in SMA patients.

This study is enrolling up to 64 patients with one daily oral dosing for 12 weeks. The primary objective of this trial is to investigate the safety and tolerability of RG7800 while also measuring pharmacokinetics, SMN protein production and changes in SMN2 mRNA levels. Exploratory end-point also includes muscle and nerve function measurements.

As you know, as part of every drug development programs, you also perform preclinical and safety and toxicology works to support the chronic use in patients. There are recent data from RG7800 on this topic. In the first cohort of movement dosing has been successfully completed.

RG7800 was well-tolerated and preliminary review of the blinded data indicates substantial increases in SMN2 full length messenger RNA. These data are inline with the observations in healthy volunteer Phase 1 study. Recently, long-term monthly data have shown findings in the eye at drug concentrations above those explored in patients’ to-date.

We will now adverse – observed in the clinic as a precautionary measured we have temporarily suspended dosing of additional patients, while we further evaluate this results. Let me summarize, the safety tolerability and pharmacodynamic data observed with RG7800 so far in the clinic are encouraging.

The preclinical findings in the eye have not been observed in human and activities are ongoing the fully evaluate this findings and confirm our next steps for MOONFISH. As you know, learning about safety and dose optimization is a normal part of drug development. Let me turn next to our cancer stem cell program targeting BMI1.

As we have discussed in the past, our compound PTC596 reduced the level of the BMI1 protein in preclinical model. BMI1 is up regulated in the tumor stem cell population and is an important component of the complex that allows cancer cells to become resistant to chemotherapy.

I’m very proud to have a fourth internally discussed the program at PTC entering clinical development. PTC596 recently enter into a Phase 1 study in advanced cancer patients with solid tumor. We’re excited about this program and we will keep you aware of the progress as we move forward. Finally, let me briefly discuss our anti-biotic program.

We have developed the platform that has identified new and novel chemical entities which target multi-drug resistant bacteria. The first compound from this platform is an antibiotic. PTC672 has entered IND-enabling studies for treatment of drug resistant gonorrhea. This is a new and novel chemical scaffold.

With the unique target that is highly selective for the gonorrhea pathogens. It is great science and reflects the strong research platform that is driving that PTC. We look forward to sharing more with you about these programs throughout this year. With that, let me turn it over to Shane.

Shane?.

Thanks, Stu. We finished the quarter with $280 million of cash and marketable securities on our balance sheet and no debt. We believe that this cash position should be sufficient to fund our operations through late 2017. Here are the financial highlights from the 2015 first quarter.

Beginning this quarter, we’re now booking translarna revenue from our commercial and reimbursed early access sales ex-U.S. on an invoice basis.

As you may recall, for the first few months of our launch in 2014, we like many companies during a launch for their first product had only been booking translarna revenue on a cash basis when payment was received.

We have now established a pattern of collectability from numerous countries where we are shipping translarna, which allows us to begin recognizing revenue upon product shipment and invoice to the customer.

As a result, in an addition to recording revenue for product shipped in the first quarter of this year, we’re now booking it throughout this quarter for revenue that we had differed in 2014, under the cash accounting methodology.

We are pleased to refer that in the first quarter of 2015, we recorded $5.1 million in commercial Translarna product sales. $3.7 million of this was from sales occurring in the first quarter and the remaining $1.4 million which from sales previously differed in 2014, that we are now recognizing.

Total revenues for the quarter of 2015, were approximately $7.5 million which compared to the first quarter of 2014 of approximately $9.2 million. In addition to our commercial product sales, we recorded $2.4 million in collaboration in grant revenues this quarter.

The $9.2 million recorded in Q1 of 2014, had included one time milestone payments from Roche of $7.5 million, for the phase one initiation in the SMA program. R&D expense for the first quarter of 2015, were approximately $27.9 million which included $4.7 million in non-cash, stock-based compensation expense.

And this compared to $15.9 million for the same period in 2014, including $1.9 million in non-cash, stock-based compensation expense. R&D expenses increased year-over-year as a result of increased clinical development in supply chain activities in support of the Translarna launch as well as in conjunction with our expanding clinical stage pipeline.

SG&A expenses were $17.6 million for the first quarter, including $5.1 million in non-cash stock-based compensation expense, compared to $7.5 million for the same period in 2014, including $1.8 million in non-cash stock-based compensation expense.

SG&A expenses have increased year-over-year as the result of the growth we are experiencing associated with commercial activities in support of the launch of Translarna across Europe and other regions.

Our organization is growing across many roles, both for the European launch activities as know as in preparation for a U.S launch in the first half of 2016. Overall the company reported a net loss of approximately $37.9 for the first quarter of 2015, compared to approximately $14.1 million for the same period of 2014.

We currently have 33.9 million shares issued and outstanding which includes $0.4 million shares in restricted stock grants. Joining us for our Q&A is our Chief Commercial Officer, Mark Rothera. Operator, let us take the first call. Please.

[Operator Instructions] And our first question will come from the line of Simos Simeonidis with RBC Capital Markets. Please go ahead..

Hi guys, thank you for taking the question.

I’ll start with the Shane, congrats on the progress on the adding I guess, I think 40 patients between the end of February and the end of April 42 to 82, I was wondering Shane whether you can help us kind of give an idea of – on the price per patient or revenue per patient I should say, or the $3.7 million in actual revenue for Q1 roughly how many patients that corresponds to?.

Yes, thanks for the question, Simos. I think we are happy to announce the 82 patients that we had on reimbursed commercial or the access therapy as of April 30, obviously that doesn’t necessarily correspond exactly to the revenue that we’ve reported for the first quarter which is ending March 31.

And I think our guidance remains the same in terms of at least how we model our revenues internally is it better price of US$300,000 on a net per patient per year basis..

So that’s the number we should still be looking at that hasn’t changed in anyway?.

Yes, no that has not changed it is consistent..

Okay, great.

I’ll ask one more and jump back in the queue, this one first to – on the SMA – on the SMA and rather knew that you disclosed today, can you first of all tell us what this high finding is and maybe talk about the order of magnitude higher than the doses that you had seen, that is was observed in preclinical versus the doses that you have tested in humans, basically how much higher was it in animals, in monkeys you actually disclosed that was in primates versus the doses that has been tested in humans?.

Yes, thanks for the question, it was – it was an exposure that we are far higher than what we had in humans, we haven’t actually disclosed the precise dose and its important to remember really that this dose – that the concentration that patients were dosed that we’re below this.

And I think the other important point is really that it’s a non-clinical finding in the eye and that I think really from the point of view of what we saw was at those doses which we are below that we are able to see splicing and as we said it was substantial splicing comparable to what we saw in the Phase 1 study.

So we felt a pretty good that we’re seeing on target mechanism of action. And then really what we’re doing now is just evaluating this eye finding. So we better understand it..

And Stu, was this something that was communicated to the FDA or EMA and was the decision made at the request or that behalf of the agencies or where you in Roche just acted on your own to suspend dosing..

Yes, so no patients were affected or really in the abundance of caution. We decided to suspend the patients on the next cohort, no patients where above their exposure range where we saw this. And so we will be its part of the process. We will be reporting at the non-clinical findings to the regulatory authorities as it’s typical in these processes..

All right. Thank you very much for taking the questions..

Sure..

Our next question comes from the line of Alethia Young with Deutsche Bank. Your line is now open, please proceed..

Hey guys, thanks for taking my questions. A couple, one that is follow-up a little bit more on the SMN. Do you have any like the color on maybe the timeline any additional work needed? And then, does this affect the infant study as well for you and that the first part question..

Yes, I think that’s thanks for this question, it’s a good question. As you know, we just recently learn this. So that it’s really too early right now to give you any detail on the timeline in such. We didn’t know of the cohort 1, our type 1 patients were dosed. And then obviously these are influence and so in the abundance of cautious.

As we go ahead and understand this finding and evaluated we will come back and then give you more clarity on the timeline..

And then on kind of DMD and the world orphan being what's happened. I know you guys have some presentations there and I’m a lot of people go and get there but maybe kind of highlight if there were any relevant takeaways pertained to maybe findings and takes around the ongoing DMD trial design there..

Sure. Yes, so we had actually we had a couple of our folks both Mark Rothera and then Young, both going to that meeting and actually presenting our experiences, it’s a consequence of being really the pioneers of this. So maybe I will ask Mark, you want to give a little bit about what you are found at the meeting..

Yes, I think the main goal of that presentation was just to share with the broader community and illustrate what it is to bring a new drug, which is a new chemical entity with the novel mechanism of actions, which is also the first pioneer root to market for a disease like Duchene Muscular Dystrophy.

The kind of learnings that you have along the way and to illustrate that story and also demonstrate that sort of based on that learning how that helps really define the ACT DMD Phase 3 trial, confirmatory trial that we’re currently undertaking. So it was more about a general tool to that audience..

Okay, thanks guys..

Thank you..

Our next question comes from the line of Geoff Meacham with Barclays. Your line is now open. Please proceed with your question..

Good afternoon guys and thanks for taking the question. I got a couple, one on Translarna, I don’t know if you can, I know it’s early in the launch but maybe give us some segment data, for example, like may be geographic breakout and again what you’ve seen so far it’s early but the persistent rates would be kind of helpful.

So just maybe, just more detail on your commercial experience thus far and I have a couple of follow-ups?.

Okay, thanks. Well clearly we are very pleased with the positive reception that Translarna is getting and I think it’s too outlined earlier it’s now being a reimbursed and shipped to nine countries, so we are really pleased to see that.

With respect to compliance, when you think about this kind of high unmet medical need, very good and strong compliance for a aggressive dementia, but I think its still too early and premature to give you long-term guidance where that would be, but too very happy at this point..

Gotcha, okay.

And then for SMA, I wasn’t clear if there is really a mechanistic basis for what you guys have seen in the eye, have you picked up any sort of toxicity in any other assays that you guys have done or is this relatively new finding and then just wanted you guys to talk a little bit about may be what you can disclose as the next steps as you interact with U.S., European regulators? Thanks..

Yes, sure, thanks. Thanks Geoff on that, clearly it’s a as expected when you do development it’s a process of doing short-term, medium-term and then longer-term safety studies and you gain information and this is just part of the development process and you gain and understand what you need to monitor and what issues might arise.

So this was a finding from a more long-term safety study. And again in the monkey model where we start in the eye exposures that were above where we were in patients and so I think what we need to do is try and figure out if that we can what’s the potential mechanism is trying to work on that. And understand where the best therapeutic window.

At the end of the day, I think, what we only always try and do with these things because something we’ve been doing now for years in the company and working with our collaborators just to try and you just this in standard drug development is to identify what an appropriate dose is within the therapeutic window. So I think that’s what the goal.

I think what we’re trying to do is understand what the safety finding is get – see if you could better understand the mechanism and then understand what the appropriate dosing could be..

Okay, thanks, guys..

Our next question comes from the line of Tazeen Ahmad with Bank of America. Your line is now open, please proceed..

Hi guys, thanks for taking my questions.

For Translarna, can you just give us a little bit of color you did have impressive growth in patients on therapy from your February update, but in March and April that you see acceleration in the number of patients in April relative to March or was it kind of evenly distributed? And also can you give us some color on the profile or the patients themselves in terms of age and may be what their baseline workability is? And then finally, a doctor is being selective and the types of nonsense mutation patients that they are putting on drug are you finding that they’re just prescribing at any of their patients around the nonsense mutation form of DMD..

So I think you’ve asked a number of questions. So I think that in the first quarter, you did see some pent up demand from countries that have been waiting to come online. And so as a bonus affect I think.

And probably too early to give you sort of direction on precisely how that goes going forward, bearing in mind that for Europe, you are talking about the addition of multiple launches overtime with multiple countries under that one regulatory approval. But clearly we’re very pleased with the way its going and stay tuned with regards to the future.

Our focus is the current label, which is patient age five years and over, who are ambulatory and working very hard to make sure that they are identified and they are going on to therapy rapidly and that’s what physicians are doing when they have found these patients.

Once the ACT DMD trial comes [ph] out we’ll be looking to seek a broader label in Europe and in the U.S. and on that basis enable more patients to get treated..

Okay. And then just a follow-up for SMN, I think, in the past you had indicated that might have been possible for the MOONFISH study to read out of in the early part of 2015, but given what you just told us is that less likely now.

Do you think it could be well into 2016 before we see the data?.

Yes I think, thanks for that, it’s really just soon to give you any real information on the exact development timeline. Of course we are encouraged by the preliminary clinical data that we saw so far. So I think the best we could say is stay tuned then we’ll update you as we understand it further..

Okay, thanks guys..

Our next question comes from Chris Marai with Oppenheimer. Your line is now open, please receive the question..

Hey. Good morning, guys, thanks for taking the questions. I was just wondering with respect to aniridia, if you could help us understand your choice of selection for this to be sort of the next indication for Translarna.

Is there some commercial connection between aniridia indication from your salesforce perspective and then some synergies you could realize there? Or is this a potential opportunity to have, sort of reformulated therapy for the disorder.

And then finally on that note, would you be looking at reformulation for specific eye drops or a different dosing locality? Thanks.

Yes things for the question. I mean the way when we think about what are the indications that we want to move forward in on Translarna is multiple things that we think about for instance, the high unmet medical need, the patients known in terms of the nonsense mutation either some pre-clinical data, are there some end points, that one can view.

And so I think obviously that then will go to a valuable commercial perspective if you can show that and I think aniridia is, I think you’ve heard or was eluded to earlier on in this call, is that obviously some very impressive preclinical animal results demonstrating Translarna’s activity in this.

And so I think it was really based on that, I think, what we are working on now is that the clinical plan which we think would be in a sense of probably around the year study where we would looking at both structure and function within the eye to look to at changes within that.

So I think a) it fits all the important criteria of high met medical need, something that we can measure and be able to move forward on..

Okay and then with respect to the SMA program and the eye findings, I’m just wondering with respect to, I guess, how long the folks have been dosed? Is this a finding that is in some way a result of drug accumulation in patients? Or is it too early to tell? And what those sort of accumulations are those doses tested be related to the amount of drug on board somebody who had potentially been dosed over a longer period of time.

Thanks..

Yes, thanks. I think that’s a good point. There were actually no findings of this sort in patients that we observed and these were at exposures in monkeys at higher concentrations, than we’ve ever given to patients. So I think this is not an issue in terms of that.

We’re obviously exploring what the – we would and I say – we and by were maybe collaboration and trying to understand some of the questions that you are asking, but no patient itself were put an any risk.

Obviously, I think, one point is, and this is something you do an animal model as you do, you increase levels of dosing to identify those things that you want to monitor and this is one of the findings that we found. And obviously, I think, the point and that was done in the long-term safety toxicology study.

So we’re going to be looking that and I think, the important point here is, I think, is that we and Roche and the SMA foundation a highly committed to this and obviously, this is such a clearly high unmet medical need that really our goal is to move forward understand what happens and so that we can decide what is the appropriate dose and we’re in the correct therapeutic window.

So that’s we’re at right now..

Great, thanks for taking the questions..

Our next question comes from the line of Anupam Rama with JP Morgan. Your line is now open. Please proceed with your question..

Hey guys, just a quick question on PTC596. The clinicaltrials.gov indicates just patients in advanced solid tumors I’m just wondering if you could give more specifically which solid tumors you will be focused on..

Yes, yes, thanks for that.

Yes, obviously this is its our BMI program, which is just to remind everybody its an important molecule to that’s in a sense an important regulator of cancer stem cell growth and reduction of this would lead not only to inhibition of tumor growth or reduce the number of cancer stem cell, and there was – we’re very proud that it was recently added into – entered into the clinic.

And it really this is a predominantly a safety and PK study and its really all commerce. So that’s what we’re doing right now. So we’ll have a pretty good idea of where the safety and where the pharmacokinetics or PK is..

Great, thanks for taking my question..

Our next question comes from the line of Jason Kantor with Credit Suisse. Your line is now open. Please proceed with your question..

Hey, good afternoon. This is Jeremiah in for Jason. I know you’ve looked at RG7800, quite expensively in animal models and particularly in the two different mouse models.

And I was wondering did you see any of this previously in the mouse model? And also when you did some pretty extensive preclinical work with the same mice, you did a transcript analysis and look for any changes in splicing.

Were any changes in splicing consistent in transcripts that are important for eye development or eye maintenance?.

Yes, no, that’s a good question. We did not see these findings in any mouse model or even rat model for that matter. So this was a monkey finding that was observed again at a exposure concentration that were above those that we’re seeing. And obviously I think the compound itself was shown to be pretty selective a small number of things.

So we’ll also be looking at that as well as just looking at the overall affects to try and figure out of what this is due to. But I think – I think to put it in perspective a little bit, this is standard development process where you can get a finding and you get stop and you take a look to find out what the issues are.

So you know I think that’s just where we are right now..

Okay.

And then one more follow up as you know with the expansion of MOONFISH that was put on clinical trials.gov, obviously it’s posted April 17 and I’m sure there is some negotiate in your ataluren babies that are seven months or young, you know, was this finding available to the FDA during the negotiations or is this something that’s kind of happened very recently?.

This is a recent finding. So this was just started – this was something what that we just recently uncovered. So it wasn’t part of those negotiation..

Okay. And on last question. I noticed that you’ve started [indiscernible] DMD registry recently. Maybe could you just talk about this a little more like you know where the geographic regions you guys are focusing on, does it include the U.S.

and also like sort of is this part of your larger preclinical or pre-commercialization efforts in the U.S.?.

Yes, so yes – this is – so I think registries and I can have Mark talk a little bit more about this. This is one of the standard things that one does in an especially of – in the areas that’s an ultra-orphan drug, when you’re the first in every type of drug that was approved.

And so ultimately, there are three other questions and you want to follow-up to understand long-term benefits. And that’s one of the role of what our registry does for us. It allows us to collect data in the commercial setting. And that’s what we plan to do.

And so I think we have been working in Europe to be able to get that, so that we will be collecting data around Europe but also at the appropriate time we will be doing some in the U.S.

I think, Mark do you have any?.

Just emphasize, this is going to be a global initiative that we are working closely with the community treat an MD and other umbrella organizations because we’re also helping the whole DMD feels really elaborate a disease area registry which also incorporate product registry information.

And I think as Stu said I think this is extremely help for in a very rare disease. It never produced long-term outcome data. I know it is useful when you’re looking at it from a life cycle perspective..

Great, thanks for taking the questions..

Thank you..

Our next question comes from the line of Yaron Weber with Citi. Your line is now open. Please proceed with your question..

Hi. This is Kumar Raja in for Yaron. Thank you for taking my question. For the U.S. launch, how far are you in the preparation and what kind of preparations have you done there? And also for SMN you have presented data on SMN protein levels in plasma.

So are you guys planning to look at SMN levels in the spinal fluid?.

Great. So I’ll pass the first part on and then we’ll talk about the SMA..

So thank you, yes. The U.S. is clearly very large – is our largest potential market for Translarna and we’ve started to build the leadership team and the team that will put all the elements in place for successful launch next year.

You probably saw that we’ve hired Eric Paul as the Head of Commercial Operations for the Americas and he brings great deal of experience in commercializing ultra-orphan both in the United States and internationally and we’re delighted to have him onboard.

And he is now building our leadership team in the United States, so that we can put all the groundwork in place between now and the end of this year for the launch next year..

Yes, and then the question in terms of the SMA, I mean what we’ve found especially in our preclinical study, where we look – and we’d look not only in the plasma but muscle, brain, spinal cord, so we’re able to show that the plasma was indicative of what was occurring in all the tissue type.

And I think this is an important point that we were able to show increased levels of SMA protein in all of the tissues that we’re investigating. So I think in a way, we have the advantage with an orally available small molecule to be able to look at the effects of what happens in plasma.

And then be able to correlate that with some of the functions of that would be associated with the expectations of improving neuromuscular junctions and muscle integrity..

Do you think that would be acceptable by the regulatory authorities?.

I think that it’s too early to tell right now. We need to gather all the data and then figure out – you just understand that better before we can make any claims about that..

Okay, great. Thank you so much..

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

Hi guys, thanks for taking the questions. First question is on the Translarna launch, what are your current thoughts on timing of formal commercial launches in the rest for the you top five, UK, France or Italy.

And what a transition to a formal launch make any difference to the dollars booked on revenues?.

So thanks for the question. You’re quite right to underline that sort of guiding fact, so launches is the pricing and reimbursement agreement with the authorities. As you know that we’re very committed to bring Translarna patients as rapidly as possible. And so, we’re very pleased in countries like Italy and France.

We’re able to obtain the ATU cohort, which essentially allows physicians to prescribe the drug today on a reimbursed basis for those forward in the label.

So the difference will be that when we finish pricing reimbursement formal negotiations, we will also be able to promote, right now we’re not in the promotional phase, however it’s clear that doctors are interested and willing to be prescribing Translarna now for patients.

You mentioned the UK, the UK you probably noted that the decision making process of funding of highly specialized in orphan drug therapies is being redesigned. And last December that process is put on hold until the redesign is complete.

And while, we see this as still being in transition we’re expecting the NHS England to make a decision and to fund Translarna with that decision by the end of this quarter. So we’re watching that carefully and we’re expecting a decision that coming couple of months..

Got it. And a quick question on SMN. I sort of think any of us would be this dose as if your efficacy data didn’t looks as good as it does.

Stu, can you just comment on whether the eye finding that you saw was in the front of the eye or back of the eye and is there any test gene it could be related to the chemistry of the compound?.

Yes, I think that – thanks for that. And just – I think it’s a little too early to say disclose. I think we need to go through and understand it and then give you a better feel for that at another time. So I think that’s really what we can say right now.

And I think, just to put it again in perspective, I think when you think about the drug development every drug has a therapeutic window and our job is to – any job within developing a - our drug has identified in active dose within that window and that’s just part of the process that’s going on now..

And my last question is on the CF program, how have – can you give us more detail or characterization of your discussions with CHMP on potential conditional approval in CF and Europe.

And can you compare the discussions and negotiations with what happened during your Duchene’s review as well as published in the summary of approval, for instance has there been a scientific advisory counselor committee called on the drug?.

Yes, it’s a good question. We’ve had a scientific advisory workshop committee where we felt that there are – it was a positive discussion to be able to move forward to submit for an approval. In this case there is a consequence of having the approval as a consequence of the Duchene Muscular Dystrophy, as you know there is a – it’s a variation.

So the actual timeline within that process is actually is short. And I think it will be as little as a three-month process. And so what – and therefore what our plans are to – as a trial fully enrolls that we or gets relatively well enrolled that we then submit the MAA as a consequence of that.

So we are looking to do that sometime in the fourth quarter to be able to do that. And so I think, stay tuned. I think we are pretty well experienced in being able to do this. So we are excited to move this forward..

Great, thanks for taking the questions..

Our next question comes from the line of Debjit Chattopadhyay with Roth Capital Partners. Your line is now open..

Hey, thanks for taking my questions. Just wondering in terms of for the SMN program, the data that was presented at the AAN, the efficacy was primarily the 90 milligram dose.

So projecting this forward where do you think the right dose will be for the program to restart?.

So I think what we presented at the AAN was that there was a dose dependent increase in the concentration of SMA splicing. And so, I think the goal then was to go from there into the MOONFISH. And then to look at the dose to look at have multiple [focuses] to find the best and appropriate dose.

I think what we reported here today already is that we had dose to first cohort where we saw – what we said was substantial splicing as a consequence of that. So we feel good about that in terms of showing that the drug demonstrated activity.

And is inline with the changes of what we saw in the Phase 1 studies which was up to 80% on an average within that. We’re saying that within this. We’re seeing that or more. So we feel pretty good about the demonstration of the splicing effect.

So now that the job is just to figure out understand a bit more of the safety finding and then to find to be able to – like any other drug program is then to define, can you find the efficacious dose within the therapeutic window.

And therefore, I think its important to remember that the exposure was below – in patients was below what we saw in the exposure that caused the finding in monkeys..

And Stu, how long was the monkey study, I mean in terms of the cumulative exposure of the drug, how long did it take for the eye symptoms to manifest?.

Yes, this was a longer-term study. It was a nine-month study..

So you should be presumed that the first cohort that you treated in the MOONFISH study was below the 90 milligram dose and it was one of the lower doses and if you are seeing substantial splicing, – worse case scenario you can avoid going high doses?.

Yes, I think what we’re saying is that the exposure was below that. And therefore what we need to do like we do in any other drug development program is to define what the best exposure is within the therapeutic window. I think that’s where we’re at right now..

And regarding Translarna, outside of ex-Germany in the Western European countries, are you seeing any pricing pushback that is maybe dragging formal reimbursement discussions?.

So I think I’ll passes onto Mark, but I think in general, I think what everyone will say is that obviously this is the pricing reimbursement is always something that you go back and forth with the payers. But I think we’re moving quite rapidly. And so Mark, maybe you want to….

Sure, Stu, well. So we are shipping Translarna on a reimbursed basis into countries outside the EU on a named patient basis, so for example into Columbia. And essentially those prices reflect European commercial prices. So that is the way the system works and we’re being reimbursed to that level..

And one more question regarding the rapid switch from the cash basis to our shipped basis, within just a first quarter, as you are still rolling out in other European countries.

I’m just – curious what drove that rapid change over?.

Yes, thanks Debjit.

I think as Mark spoke to we’re now shipping to patients and in nine countries and that’s expanding with time and we’ve got now those up a nice track record of receiving payment, which is given us the confidence that we were able to now switch our revenue recognition to booking revenue on shipment and invoice as opposed to waiting for cash payment or getting paid pretty consistently now for a decent period of time..

Well, thank you and good luck..

Thank you..

Thanks again..

Thank you..

The next question comes from the line of Heather Behanna with Wedbush Securities. Your line is now open. Please proceed with your question..

Hey guys, thanks for taking the question. Most of my questions have been answered.

Just I’m curious about the Translarna launch, when we think outside of Europe, where most of patients are concentrated in center of excellence and places like South America, if you could just give us a little bit of color, where patients were found and if it’s concentrated or if the market is a bit more fragmented?.

Okay, thanks for the question. I think it’s actually a mixed picture. You have some very large senses. So for example, in Brazil, there are some senses, which are extremely large and very well organized. But there are also a lot of patients out in other parts of Brazil in very small and less sophisticated senses.

So part of our job is to work with the community to ensure appropriate genotyping and identification of patients is underway. Certainly, we refer to in Europe there is some countries which have extremely well organized, reference sense of networks like France. So we get the whole spectrum depending on which country in the world we’re looking at..

Great. Thanks for the color..

You are welcome..

Thank you. And with no further questions in the queue, I would like to turn the call back over to the speakers for closing remarks..

Yes, thank you. And thank you all for joining the call. As you know, it’s been our priority to get these therapies as quickly as possible to patients, obviously reflects high unmet medical need.

We’re pleased with Translarna in the fact that today, we’ve got an 82 patients and the uptick seems good and that we look forward to updating you in the future throughout the year. So thank you for joining the call..

Ladies and gentlemen, thank you for your participation in today’s conference. This does conclude the program and you may now disconnect. Have a good day everyone..