Emily Hill - Director, Investor Relations Stuart Peltz - Chief Executive Officer Shane Kovacs - EVP and Chief Financial Officer Mark Rothera - Chief Commercial Officer.

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

Thank you for holding for PTC's Second Quarter 2015 Conferece Call. At this time, all participants are in a listen-only mode. Following the formal remarks, PTC Management will open the lines for question-and-answer period.

Please advise that the call is being taped at the company's request and will be archived on the company's webcast until August 13 which is two weeks from today. PTC's current investor presentation slide deck is available at the same website location. At this time, I’d now like to turn the call over to Emily Hill of PTC..

Thank you. Welcome to today’s conference call to discuss second quarter 2015 earnings results. The commercial launch of Translarna and other corporate update. 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 a 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-Q 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. And good afternoon. Thank you for being on the call. There is a lot to talk about today. I'll give you a quick overview of where we are and then going to the detail. Let's start with Translarna.

After over 17 years of developing Translarna which is now approved in Europe, it is exciting to be rapidly approaching the completion of our ACT DMD trial. This is the large study of DMD ever conducted. We anticipate ACT DMD will have top line data available in the fourth quarter of this year.

We look forward to a potential US approval and a commercial launch in the first half of 2016. The commercial launch for Translarna for nonsense mutation DMD is going quite well. As of June 30th, Translarna generated $11.2 million in product sales revenue year-to-date.

There are now 106 commercial patients on therapy and we are shipping Translarna to 12 countries including our first shipments to Brazil via reimburse main patient program. We continued working closely with health authorities to bring Translarna to patients as quickly as possible. Now let me turn to our efforts in cystic fibrosis.

ACT CF, our Phase 3 trial with Translarna is on track to complete enrollment by the end of this year. We are now preparing an application to add nonsense mutation CF to our label in Europe.

This application will be based on results from our previous Phase 3 trial including new analysis of our data which will provide us with increased confidence in the positive benefits of Translarna for nonsense mutation CF patient. I'll provide more detail on this analysis in the few moment.

I am also very excited about our SMA which is collaboration with Roche and SMA Foundation.

As you may recall proof of mechanism based on increased efficiency of SMA to RNA slicing was demonstrated in both Phase 1 and Phase 2 study, dosing in the MOONFISH trial was temporally put on hold in May based on a preclinical I finding seen in the long-term in monkey. We've learned more about this finding.

Studies conducted so far combined with addition of preclinical data expected in this fall could allow dosing in MOONFISH to resume in the first quarter of 2016. I'll address this program later on today's call. Let me take you through more details of what I just outlined.

In terms of the commercial activities for Translarna Duchenne muscular dystrophy, we are extremely proud to have launched the first approved treatment for nonsense mutation DMD to patients with this live threatening disorder.

Translarna reach through nonsense mutation to produce full length proteins and therefore addresses the underlying cause of the disease. Translarna was approved by the European Medicine Agency for the treatment of nonsense mutation DMD in ambulatory patients aged 5 and over last August and the commercial launch began at the end of last year.

Translarna commercialization outside of the US is a staggered country by country reimbursement process. In Germany, we are very pleased to have received a G-BA rating of three out of the scale six. This is important as it acknowledges Translarna as having convincing evidence of a clinically meaningful added benefit to patients.

We are proud to receive this rating as it is recognition of the importance Translarna for nonsense mutation DMD patient. We are also working through Market Access in the UK. The UK is in the process of putting in place a mechanism for budgeting also orphan drug.

As part of the process NHS England has decided to include input from NICE resulting in a delay of the funding decision. We thanked the patient groups and physicians for their ongoing support and we will continue working through this process to bring Translarna to DMD patient. Translarna is now directly commercially available in four EU countries.

We also have Translarna reimburse in eight countries through reimbursed early access program in both Europe and Latin America. We are now shipping product to patients in Germany, Spain, France, Italy, Greece, Austria, Denmark and Norway. Outside of Europe, we are shipping products to patients in Turkey, Israel, Columbia and most recently Brazil.

Brazil is one of the largest markets in the world. And it is worth noting the unique process they have for reimburse early access. For patients with high unmet medical need, physicians can request access for drugs to treatment they believe provide benefit to patient.

On a case by case basis, the Brazilian court can authorize reimburse access to this drug. So far we are aware of about 18 patients who have gone through the individual approval process and are in various stages of gaining access to Translarna. We recently began shipping Translarna to the first of these patients through reimbursed early access.

As part of our commitment to patients, in the second quarter we also initiated a sibling program for brothers of patients involved in our DMD trial. For families with more than one child suffering from nonsense mutation DMD having only one child enrolled in our clinical trial with access to Translarna can cause great stress.

We work to find a solution to assure those siblings who are not eligible to participate in the trial can still have access. Because of this program, brothers in both Europe and the US are now receiving Translarna. As of July 28, we now have a 106 DMD patients receiving Translarna in reimbursed program.

This can be either through reimbursed early access or direct commercial sale. We will continue to provide patient numbers for the remainder of 2015 fiscal year and will then assess what metric to report to the investment and community in 2016.

In addition to the 106 patients are commercially reimbursed Translarna, we currently having approximately 375 patients in our clinical trial and extension program. This is the total of approximately 500 DMD patients currently on therapy.

As Translarna become commercially available, we expect to transition a significant portion of these patients to commercial therapy. We are also preparing for a US launch in the first half of 2016.

As a reminder, we began submitting and enrolling new drug application to the FDA for Translarna for the treatment of nonsense mutation DMD in December of last year. This has given the FDA an opportunity to review the majority of our material ahead of our submission of date from a confirmatory Phase 3 at DMD trial.

Top line results are expected in the fourth quarter of this year. As you know, Translarna is also rapidly advancing the nonsense mutation cystic fibrosis. Nonsense mutation CF is a most severe form of the disease for which there are currently no other treatments available or in development.

ACT CF our second Phase 3 trial is on track for enrollment to complete by yearend. We anticipate results will be available in the second half of 2016. We are in the process of finalizing an application to add CF to our Translarna label in Europe and are on track to submit this application to the EMA later this year.

In conjunction with our preparation for the submission, we've been in discussion with experts and held numerous advisory board meeting. Natural history data indicate that while older patients tend to have a more gradual decline in pulmonary function, patients under 18 years age experience more rapid rate of decline.

In light of this data, PTC performed additional analysis. As a reminder, in our initial Phase 3 trial our post-hoc analysis of non-TOBI patients overall showed an absolute change in SEV1 of 3.5% and a relative change of 5.7% plus a decrease in exacerbation of 41% versus placebo.

Our new analysis demonstrate that the subgroup of non-TOBI patients under 18 years of age experience the robust treatment response including an improvement of SEV1 above base line. A 5.4% absolute benefit and an 8.4% relevant benefit in SEV1 versus placebo.

Importantly, the non-TOBI patients under 18 had a decrease in exacerbation of 60% versus placebo. We believe this analysis is an important new finding and plan to include in our CF submission to the EMA. As Translarna is already approved in Europe for DMD, the CHMP's initial review of the CF submission maybe completed in as little as three months.

This convenes a European launch of Translarna in nonsense mutation CF in the first half of 2016. We are preparing for the potential that in the near-term we could be simultaneously expanding Translarna's commercial access for DMD outside the US while launching for DMD in the US and launching for CF in Europe.

We have to continue develop our global footprint to be ready for these commercialization opportunities. We now have a presence in over 30 countries across Europe, North America, South America and Asia Pacific region. While this is all quite exciting, it is just the beginning.

There are many disorders with high unmet medical need where no existing treatment is available. The value for Translarna lies in this potential to benefit patients across the vast array of genetic disorder caused by nonsense mutation. Our strategy is now to rapidly expand the clinical development of Translarna across multiple genetic disorders.

On average 11% of every monogenetic disorder is caused by a nonsense mutation. There are now 30 publication demonstrating Translarna's activity in many preclinical nonsense mutation disease model in different organ system. We are currently pursuing four initial indications in distinct area.

A neuromuscular disorder DMD, pulmonary disease CAT, lysosomal storage disorder MPS1 and an eye disease aniridia. Translarna truly is a pipeline within a product.

We are studying our site to deliver on our commitment to address rare and neglected disorders by pursuing the strategy for Translarna's pipeline expansion which we are calling 10 by 20; by 2020 we plan to investigate at least 10 nonsense mutation indications in the clinic in order to maximize Translarna potential as both our product and a pipeline.

Let me now shift to the SMA program. SMA is a genetic neuromuscular disease caused by missing or defective SMN1 gene which results in reduced levels of the SMN protein.

In sufficient level of SMN protein are responsible for the loss of motor neurons within the spinal cord leading to the muscle atrophy and death in instance and path of in its most severe form. SMA is the leading genetic cause of death in instant. There are no marketed therapies for SMA.

RG7800 is an orally available small molecule that has been investigated for its ability to modify the splicing of the SMN2 transcript for the production of full length mRNA. A tremendous amount of effort in resources has been put into this SMA program by all three collaboration partners.

Data from our Phase 1 study in healthy volunteers represented at the annual meet of the American Academy of Neurology in April. These results showed a dose depended effect or RG7800 on SMN2 splicing demonstrating proof of mechanism. After a single dose there was approximately an 80% increase in full length SMN2 mRNA production.

Based on the Phase 1 results, a Phase 2 multi-dose study MOONFISH was initiated last November in SMA patients. The primary objective of this trial is to investigate the safety and tolerability of RG7800 while with pharmacokinetics and SMN biomarkers as secondary measure. Dosing of the first cohort of MOONFISH was successfully completed in April.

RG7800 was well tolerated and preliminary review of the blinded data indicates substantial increases in SMN2 full length mRNA. These data are in line with the results from the healthy volunteer study and represent the second independent study showing proof of mechanism.

We announced on our last earnings call that the dosing of MOONFISH was temporally suspended as we evaluate a non clinical eye finding seen in monkey after nine months of dosing and exposure levels above those dosed in the clinic.

I want to remind you that the safety tolerability and pharmacodynamic data observe with RG7800 in the clinic so far are encouraging and these preclinical findings in the eye have not been observed in human. We've been working to understand this eye finding including performing additional preclinical study.

The results of this study combined with the final study that will complete this fall could allow MOONFISH to resume dosing in the first quarter of 2016. We are very excited about the SMA program. Previous and new publications have highlighted the importance of having SMN and protein both in tissues within the CNS and peripherally.

These results from this publications are significant as a emphasize the importance of a drug that can distribute both in the CNS and in the peripheral tissue.

Our orally available small molecule have demonstrated broad this tissue distribution in addition to crossing the blood brain barrier which we anticipate would be advantageous in the treatment of patients with SMA. Let me now turn to our cancer stem cell program targeting BMI1.

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. As you may recall, our compound PTC596 reduced the levels of the BMI protein in preclinical model.

I’m very proud to have a fourth internally discovered program at PTC entering clinical development. PTC596 entered into a Phase 1 study in the second quarter in advanced cancer patients with solid tumor. We are excited about this program and we will keep you aware of the progress as we move forward.

Furthermore, we have a number of interesting research programs to continue to progress. In addition to Translarna, we have nonsense suppression platform that is growing. We have a follow on program underway and are identifying the number of promising compound to help maintain and extend our franchise in nonsense suppression of disorders.

With that let me turn it over to Shane.

Shane?.

Thanks. As Stuart mentioned earlier, Translarna sales continue on a strong trajectory and we've now generated year-to-date June 30 sale of $11.2 million.

In the second quarter, we recorded $6.2 million in Translarna net product sales representing approximately 69% sequential growth over the $3.7 million of normalized sales in the first quarter of this year which excludes $1.4 million of deferred revenue booked in Q1 from 2014. As a reminder, in 2014, we had been recognizing revenue on a cash basis.

Total revenues for the second quarter of 2015 were approximately $6.8 million compared to the prior period of $1.7 million. In addition to our commercial product sales, we recorded $0.6 million in collaboration and grant revenues in the second quarter of 2015.

Research and development expenses in the second quarter were $28.2 million compared to $18.3 million for the same period in 2014. R&D expenses have increased year-over-year as a result of additional cost associated with our expanding clinical stage pipeline.

SG&A expenses were $17.2 million for the second quarter of 2015 which compared $8.7million in the same period last year. SG&A expenses have increased year-over-year as the result of the growth we are experiencing associated with our commercial activities in support of the launch of Translarna across Europe and other regions.

Our organization is growing across many roles, both for the ex US launch activities as well as in preparation for a potential US launch in the first half of next year. We reported a net loss of approximately $38.4 million for the second quarter of 2015, compared to approximately $25.1 million for the same period of 2014.

Additionally, we finished the quarter with $255 million in cash and marketable securities on our balance sheet and no debt. We currently have 34.2 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, can we please take the first question..

[Operator Instructions] And our first question comes from Anupam Rama from JP Morgan Your line is now open. Please go ahead..

Hi, guys. Thanks very much for taking the questions. Just wondering just two quick ones just if you could talk to a little bit about the sequential patient adds either in the commercials fails countries or the reimbursed access countries as well -- and if there is any difference there.

And then if you could give us a sense of how mapping in Europe is going to for nonsense mutation DMD patients to the specific centers excellence. If you give us an update there. Thanks. .

Yes, thanks for call Anupam. Maybe I'll have Mark point to take question. .

Okay. So thanks for the questions. So clearly we are very happy with the way the launch is progressing as we saw we delivered $11.2 million in sales. And year-to-date and as you said 69% growth Q2 over Q1 from a revenue perspective.

As we are going through the launch process I think you quite right the outline that what we are doing is we are overlaying sort of sequential launches from countries and early access countries. All at slightly different right through of uptake and system. So it can be a little bit lumpy.

It is not a linear adds but what's really great is that we now have 12 countries up and running. And as I do said we have a footprint now that gets us to over to 30 countries around the world. And just illustrate our lumpiness just in the last two days since we declared the 106, we've already another four patients they gone on drug.

So that's bring us to total of 110. So it is a very dynamic situation. You also mentioned patient identification. And this remains a very high priority for us as an organization. This is a very rare disease. We are of course the first to launch a drug in this area. And we've been focusing on mapping patients to census in Europe.

So for example in the big five EU we are around 40% of the expected prevalence now map. And we continue to create awareness around the need for genotyping and extending our presence into more centers if you like some of the smaller centers where DMD patients are. So we continue to grow the number of map and identify patients in Europe day to day..

Thank you. And our next question comes from Tazeen Ahmad from Bank of America. Your line is now open. Please go ahead. .

Hi, good evening, guys. Thanks for taking my questions.

The first one to when do you think doctors will be in a position to talk about the efficacy that they are seeing with Translarna in the commercial setting? I know that their view is that because it is a safe drug everybody will try to prescribe it to their nonsense mutation patients but based on now about what -- what you know about the drug and the natural history of the disease how long do you think it would take to really tease out what kind of effects it's having?.

Yes, thanks for the question. Obviously I think we talked to patients throughout the period and so I think there has been an anecdotal discussion about the efficacy. So I think we feel pretty good. We put a lot of indications of what they are doing and so I think we feel pretty good about it.

Also I think that goes along with this is high compliance, also I think which is also indicative and helpful that gives you pretty good idea that people are staying within their trial as well.

So it has been very strong in the early stages than -- and I think this is really quite telling and then I think while we hear considerable amount anecdotal information I think the other thing that we have said up in place basically commercial sale, the registry which over time will collect data and really the purpose there is while we have demonstrated that Translarna changes the course of the disorder, we'd like to get long term data over time and I think the best way to do this is to registry.

So I think we feel -- I feel pretty bullish about that, really good and this along with the registry will be very helpful. .

Okay. Are there any other details that you can share about the SMA program? Thanks for the update.

What gives Roche the confidence that they can restart the trial or enrolling the trial again in 2016?.

Yes, thanks. That's an important, so SMA so as you remember I think the couple of important points here right.

This is an orally valuable drug that distribute quite well throughout the body so doesn't go off tissue and we showed then both in animal model both the periphery as well as in CNS, we have been able to see substantial SMA changes than in the Phase 1 as well as in Phase 2.

We saw slightly changes so that -- which really important here as we know that the drug affects us having activity so that's really important and those we didn't see any alteration in the clinic. This is really a preclinical finding and what was really important about -- and as we said we put that on hold while we understood the preclinical finding.

And we obviously had to -- we learned a lot about obviously the importance of the monitor ability and so I think that was one thing that gave us confidence that there is a strong path forward.

I think that was the key point in there and so along with the preclinical work that's ongoing as well as some data that expected I think that in itself would allow us to have the potential to start in the first quarter of next year.

And maybe the next point I think would be -- the other thing I also want to mention that this program has been -- is incredibly important obviously to us and Roche as well as the SMA patient and to the community as a whole and so this has been a very strong program. And so we are all very excited about the RG7800 moving forward.

It was obviously a large amount of chemical optimization. And we've always had a strong program to have additional molecules to move forward. And that's always something that we have in the background as well. So I think who gives us a lot of confidence is that we have strong program with RG7800 as well as additional other compound. .

Okay. And then maybe one last question. Maybe this is for Shane.

Do you have any idea of when you receive the input from NICE in order to be able to launch in UK?.

Yes. Maybe I'll give you a little bit color here. So just to give everyone the little background. NHS England has delayed the overall process and therefore they have asked for specialize view. So maybe I have Mark, you want to give a little more detail on the overall progress and where we are at..

Sure. Of course the context is the 12 countries already made decision to reimburse Translarna including eight in Europe. Between big countries like Germany and France and Italy and Spain. And as you know when NHS England has been putting in place a new mechanism to evaluate funding of highly specialized an orphan drug over the last six months.

We had expected the NHS England is going to make an independent decision on the funding in June. However, we learned till late June that they decided to wait for NICE's input, the National Institute of Clinical Excellent.

And after the NHS does rely on NICE recommendation for determining funding decisions, so we are expecting NICE will produce draft guidance on Translarna in October with a final decision in February. So we are now highly engaged in a NICE process and we will continue to work to help patients to get access to this treatment as quickly as we can. .

Thank you. And your next question comes from Geoff Meacham from Barclays. Your line is now open. Please go ahead. .

Hey, guys. Afternoon and thanks for taking the question. Stuart why don't you talk a little bit about the additional indication for Translarna, the 10 indications.

Have you guys looked into this from the standpoint of being able to do a trial with a number of different indications assuming that they are smaller disease market for things like using surrogate end points or biomarkers or do you feel like one by one you have to tackle them as you did with DMD and CF and down the road MPS and aniridia..

Thanks. That's really this question I think so what we've done thus far right as you said is CF and DMD we've done independently and obviously when you think about Translarna is a new chemical entity and DMD was a new indication and in the therapeutic area with a new mechanism of act so we started out one by one.

And we are as we said after CF and DMD and already MPS and then we are looking at number of others.

But I think you actually have a very good point and there is -- if you think about some metabolic cycle diseases where various mutation can lead that in a sense in the same cycle and that you may look at certain metabolic diseases and be able to sense -- in specially with small numbers you put them together and use in the sense of similar output.

So you have a very good point that's one of the things that we are considering. You can do that with some of the licenses on storage disorder as well as some of the eye diseases. So I think in some ways we are looking at doing some one by one somewhere doing thinking about it exactly the way you placed it.

And then others we may do some physician investigator trial as well and some of those like so I think we are going to take a multi prong approach. So really in the way I think about this is -- as we got the first European approval and the process of completing this trial and then having Translarna.

In a sense we are changing for right early on you usually quite protective of the molecule now we are in this position now to really try to expand it to bring to as many patients as possible so great value for them and also our investors. So I think and I think we are really at the right time to really get this going right now.

And that's what we are going to be pretty aggressive about. .

Okay. And just a follow up on earlier question just on commercial. In some of the countries that you launched -- for the longest period, is there any trend with regard to time to get reimbursement for that process to complete. I was just curious how you guys can optimize that even further. Thanks. .

So thank you for that question. What tends to happen is that you have really trends according to each different country system. So that in a country where reimbursement is a relatively efficient process thing goes very quickly.

There are some countries where it case by case, we mentioned Brazil which is third or fourth largest market in the world when it comes to treatment of this nature. Those are case by case, applications from physicians and patients have to be adjudicated in the court case by case.

And so that can take some time although we are very pleased to have learned just recently that already the court approved 18 cases in Brazil and rest of those patients. So again it is not really so much in one country specifically there is variation but you see variation between different countries. .

Thank you. And your next question comes from Heather Behanna from Wedbush Securities. Your line is now open. Please go ahead. .

Hi, thank you. First I just had a question on the cystic fibrosis data, the new data. I was wondering if you could just give us some color on how large the sub group is patients under 18 who were not on TOBI and if we might see those data at NACFC or at another conference coming up soon..

Yes.

I think we are looking to present and I think part of this is -- actually if you go back and think through our -- the understanding the natural history and I think it has been a lot of work in CF and really trying to understand this better and from learnings from the DMD and how important it is to look at particular group of which you would expect to see larger difference and certainly the patient who are under 18 where they have higher SEV1 they tend to see greater decline and so you might see better effects of that, that was what we have been talking about some of the key aspects.

And so we did then and the good news is that the groups were well balanced so that was the stratification factor during less than 18 so that and may directly well balance of the group so well balance and those about in that group about 25 patients or so per group.

We feel pretty good about this data that this is actually helping giving a stronger view of thing improvement and it is actually improvement of our baseline. So we will be presenting that at conference in the near future. .

Great. And then also I just had a question on the SMA program you have been alluding to a study that will be reading out in the fall, is that -- can you give us any color if something that you are working on monitoring, have potential toxicity rises or if you can give us any color on whether or not it is reversible or any other information..

Yes, sure. So we have been doing, obviously the importance as we said about monitor ability and then also as part of usually preclinical studies there is a recovery portion that you can monitor. So we are just wanted to complete the recovery portion of that. So that's what we will be doing..

Thank you. And your next question comes from Christopher Marai from Oppenheimer. Your line is now open. Please go ahead. .

Hi, good afternoon, thanks for taking the questions. Just another update I guess on cystic fibrosis. Thanks for the new data but I was wondering if you also comment on rate of enrollment in that trial. And when do you think that will be complete and perhaps why it might be slower than I guess as original anticipated.

And it is just holding up the cystic fibrosis conditional approval filing for Translarna in the EU..

No. Yes, thanks for the question. I think that's a good question. We area actually we are happy with the CF enrollment. I think what we have been saying it will be completed by the end of the year. And as such that contract and that's what we are doing. We are now preparing the filing so that we will get that so I think we feel pretty good about this.

So I think yes so I think so also they are fine and pretty well here and we are excited to be doing this. .

Okay then with respect to the exclusion of Tobramycin, is that causing any issues by your estimation?.

Yes, no, just to remind everyone Tobramycin conformed at launch activity and so we are not really finding this to be a real issue. The physicians can move patients to other inhaled antibiotic and so we haven't' really found this to be a problem.

And so I think that view is reflected by just that we are pretty well on track in terms of what we thought enrollment would be. The only thing that needs to be if anyone was on inhaled Tobramycin they are needed to be wash out period prior to the trial but beyond that I don't think we've really seen any real issue. .

Okay, great. And then I am wondering if you could just comment perhaps on what you are seeing from the expansion trial for Translarna and DMD. I know you are running central for Phase 2 as well as of course new enrollment the extent study from the Phase 3.

And also with respect to that are you aware of this six minute walk that did -- is that extension study enrollment and does that give you further confidence in the read out for the Phase 3 trial. Thanks..

Yes. So the extension trial is ongoing both in the current trial as well as the extension trial have been going on for the previous trials as well. So now there was a bit where data of where we stop the trial and the restarted. And in the case of the US obviously we were just doing safety, so we are gathering that those data over time.

We are very encouraged that the majority of basically all the Translarna patients have stayed on therapy and we have patients sum up of the four year and been very well tolerated. So we are in the process of collecting or have collecting all the safety and efficacy. There is not a lot of data there.

You have to actually present in an appropriate time wise we would plan to. So once we have the complete data set. So that program is ongoing. .

Thank you. And your next question comes from Ritu Baral from Cowen and Company. Your line is now open. Please go ahead. .

Hi, guys. Thanks for taking the question. The first question is on how you're going to release the DMD data in Q4 as you've guided, obviously all the permanent 0.6 minute walk data, but do you anticipate having some of the other key secondary end points at the time of top line data release.

And if so, would do you consider as those most important secondary endpoints, whether it be North Star or some of the function test including rise from floor.

Any thoughts on that right now?.

Yes, no, thanks for the point cycle question. I think yes as we said we plan to release the data sometime in the fourth quarter of this year. I think our goal will be to certainly tell you about the primary top line data. The most important of those data are those -- what must be key secondary as the time function as well.

And then the North Star another are more tertiary endpoint. So and clearly the six minute walk test and depending on how quickly release the data, how much we will know but I think we are planning to as soon as we get the data. We are excited about this and once we complete that analysis and we will put a report out there for you..

Got it. And I get back to the SMA update that you gave us. You mentioned additional preclinical studies that you guys were going to start.

Can you tell us a little bit more about those? And what physiology those focused on?.

Yes we were -- as we said we were -- this was an eye find and we wanted to just understand some ongoing study to understand more about metabolism, the normal thing that one would do. And then looking at making sure and obviously looking at how do you monitor and things like that were important. And then -- so that sort of thing that we would be doing.

So that we can then go and say yes we understand the safety issues and potential safety issues that could be, how to monitor and what it looks like after recovery. And so those are things that are ongoing right now.

So I think at the end of the day I think we as part of development program we feel we are learning it about this and this data will be all put together in the fall so that or completed so that we then based on this data we have this could allow MOONFISH to restart in the fourth quarter 2016 -- first quarter sorry first quarter.

Are the new studies just in monkeys or they are across multiple species?.

So right now -- this is the recovery obviously was that the monkey portion. Other than that just as constrains of partnership, the precise details I am not able to disclose right now..

Got it. And back to this new CF analysis that you presented.

Was that something that was asked for by the EMA or something that PTC thought was important? I am just trying to get to if there are may be implications for any potential label in the EU?.

No. This is something that we were thinking about as well as, when you think about clarification factor and the understanding of the natural history that really this been -- considering the amount of work that has been done in CF and natural history to keep looking at more carefully.

It looks like really that there is this where the rate of decline occurs more rapidly in the younger patients and then in terms of pulmonary function you see a plateau of certain age in terms of -- or slowing of the rate of law.

And so that -- so it was really that sort of analysis and talking with key opinion leaders, experts in the field that based this really important and learning that we said, went back and looked at this to take a look at it, it really was is really to be able to see 8.4% relative increase was really above baseline, and the 60% reduction in the exacerbation in this group help really give and really excited the experts in the field and gave us confidence, continued confidence of the data.

So this is going to be part of our submission. .

So it was more a PTC and KOL driven analysis?.

Yes. .

Okay. And last question really quickly.

Are you still on track for MPS1 biomarker data by the end of the year?.

I think so. What we were saying is that we've announced that the trial is ongoing and that we are going to have some data, our plan really is to have some data about the end of the year..

Thank you. And your next question comes Simos Simeonidis from RBC Capital Markets. Your line is now open. Please go ahead. .

Thanks for taking the question. This one for Shane, Shane Just to make sure comparing apples to apples, in terms of net revenues for Translarna. The $6.2 million for this quarter, should be compared to the $3.7 million for Q1, meaning there's no deferred revenue including the $6.2 million. .

Yes, that's right, Simos. If you recall in 2014 we were recording revenue on a cash receipt basis where we booked actually I think about $0.7 million of revenue in 2014 and had deferred receipt that had not receipt cash payment into 2015.

That was then recognized in the first quarter when we had enough of track record of end receipt that we felt comfortable changing our accounting to record revenue on shipment and change of ownership and therefore the first quarter we recorded $5.1 million of Translarna product sales which was comprised as you point out of $3.7 million of really Q1 demand but the recognition of the $1.4 million from the 2014..

Okay, great. And then as you are going into other countries and geographies, in terms of price per patient, should we still be thinking of the $300,000 per patient per year number..

Yes. I think we are very still very consistent in terms of our own internal thinking about how we think about the price per patient per year are still being $300,000. .

Perfect, last one. This is probably for Mark. Mark, if you can help me understand maybe the progression of the launch.

In the three iteration of the numbers of patients you have given, tell me if this is being an unfair comparison what I am going to say, the first number I saw was 42 patients roughly for the first two months of the year, it was February 21st. Then had another 40 for the next two months for the year by April 30th.

And now for the next two months it was down to 24 patients. Is it just it is going to be kind of lumpy as you get maybe just one more country for example instead of three or four countries quarter-to-quarter versus this being a slowdown and number of patients you are getting on..

Yes. I think this in a very early stage of launch.

What you are seeing is that lumpiness due to the fact that you get boluses of patients for example coming on as you get say a major market that opens up and you get a bolus and then so what you are doing if you are sort of overlaying this very different market with different situations, so it is not going to be an steady state growth scenario yet.

It is going to take us a while before we have the large enough massive countries and we have long enough trajectory of sales to start getting some more predictable pace of patient add. But as I say I think the thing that is very encouraging is that we are getting great feedback from the market where we've already launched.

We are already in 12 countries now and because our footprint has grown now 30, we expect to see more countries come on stream and increase number of patients' accrual in existing countries. .

And then actually and to Mark's point really, if you think about like we had the cut off date like July 28 but in the last couple of days we just got four patients. So it goes and it is unpredictable when they come up. So I mean just shows you from 106 to 110 in two days.

So I think at the end of the day we are pretty happy about how it is progressing..

Thank you. And your next question comes from Robyn Karnauskas from Deutsche Bank. Your line is now open. Please go ahead. .

Hey guys this is Ale on for Robyn. Thanks for taking our questions. So in terms of DMD what kind of penetration levels look like in some of the countries that have been reimbursing the longest such as Germany.

And then also what are your thoughts on the DMD panel and the fall and the way to PTC and with your enrolling submission do you think you will be at the panel..

Mark, why don't you do the first part?.

So well Ale sharing with you overall patient numbers on retrospective basis. We are actually not going into details on a country by country basis. So I am not in a position to share with you specifics of our market share in a country.

But I think you can see from the overall numbers how we are like growing and how that supporting patients in more and more country..

And then in terms of your question and the potential panel, I think right now I think what's been reported that there is planning to be advisor committee in the November timeframe from the neurology panel so it is not obviously interesting to think that they want-- obviously we could welcome the opportunity to participate in the DMD panel and it is pioneer in this space.

We've obviously been very deeply invested into development of therapies for DMD patient. We have had numerous conversations with the FDA and I think they have even been able to -- they told us that we could tell everyone that this is really of their highest priority and that we need to tell that to everybody.

And so what we are doing is working closely with them in terms of what's the fastest route to get and allowing the patients as quickly as possible and as we go through this process we will keep you posted on what -- how we agree and what's the best way to move forward in this..

Thank you. [Operator Instructions] And your next question comes from Debjit Chattopadhyay from Roth Capital Partners. Your line is now open. Please go ahead. .

Hey, good afternoon, everybody. And thanks for the questions. Just wondering on the CF data. Is it fair to assume that a bulk of the benefit is being driven by the younger patients and how does that reflect on the ongoing ACT CF study in terms of enrollment.

Are you specifically enrolling in X amount younger patients or it's too late to change enrollment criteria?.

Good question. So I think what we've done is to -- that there was -- if you think about it there is less than 18 years old what about 8.3% improvement above compared to placebo and then the less 18 there was about 4.3% relative improvement to placebo. So you see a bigger effect.

And I think obviously the criteria for entry into the trial is basically similar -- the current trial is similar to what was previous and so we expect to have a fair number of patients that will be less than 18 years of age and that what will do obviously is within the plan, is also makes this an important stratification and part of an analysis.

As well as looking at the overall we will look at this group as well. But I think at the end of the day we expect to see a statistics with significant improvement in pulmonary function reduction that's the basis that would be just larger in this particular group. So we feel pretty good about it..

Since Translarna is largely a weight based drug and I'm just trying to understand what the average age of the patients are on the commercial drug right now. Just to get a sense of what the physicians are thinking.

Are they putting the younger patients on the drug first or they are putting the older patients?.

Thanks for that. That's a good question I think right now -- what we have on label is five years and greater that our ambulatory, so they are predominantly including patients that are five years and older. So it is a spread of that is obviously are capable of being ambulatory so we get a spread of that..

You'd mentioned in 106 patients and then four more recently added. And there was 18 patient number from Brazil.

Just wondering, does that 106, including 18 patients or Brazil shipment was just one or two patients and it's really not I think indicated in the 106 or 110 numbers yet?.

Yes. I think what I was giving you as an example was the 18 that we know that the court have approved but we've only just initiated in the first one or two patients. So there is a lot of works still to do to ensure that we are supplying those other patients that the court has approved. .

Great. One last question on the SMA program. So when you expect or restarted, would you think you're going with the highest dose, which I believe the 90 milligrams or do you think you would need to go with lower dose to avoid eye toxicity, if any.

And in terms of impact on the SMN2 copy number with the lower dose versus the higher dose? Thank you so much for the questions. .

Yes, thanks for the question. Yes, it says we move forward on and make the decision, to move forward and initiate dose well, so we'll probably give more color to that at that point. So right now we are not disclosing much more than that. Thanks for the question. .

Thank you. I am not showing any further questions at this time. I would now like to turn the call back to Stuart Peltz for any closing remarks..

Yes, thanks. I want to thank you all for joining the call this afternoon. And in closing we are very pleased with the progress that we are making to drive the growth in 2015 and beyond. We are quite encouraged by the early results of our Translarna launch and products sales revenues that we've generated.

And we are excited about our growth prospects both in DMD and CF. and look forward to advancing the solid pipeline we have built, that we hope will bring value to patients with rare and neglected disorder. So thank you for the time -- taking the time to be here this afternoon..

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