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

Ritu Baral - Cowen Christopher Marai - Oppenheimer Simos Simeonidis - RBC Capital Markets Alethia Young - Deutsche Bank Kumar Raja - Citi Jeremiah Shepard - Credit Suisse Tazeen Ahmad - Bank of America Eric Joseph - J.P. Morgan Debjit Chattopadhyay - ROTH Capital Partners Heather Behanna - Wedbush Securities.

Thank you for holding for PTC’s Fourth Quarter and Year End 2014 Conference Call. At this time, all participants are in a listen-only mode. Following the formal remarks, PTC’s management will open the lines for a question-and-answer session.

Please be advised that this call is being taped at the company’s request and will be archived on the company’s website until March 14th, 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 like to turn the call over to Emily Hill of PTC..

Thank you. Welcome to today’s conference call to discuss fourth quarter and full year 2014 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 a variety of risks and uncertainties including those discussed under the heading Special Notes Regarding Forward-Looking Statements and Risk Factors in our most recent form 10-Q which are available from the SEC or on 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..

Good morning. Thanks for joining our call. I am delighted to be here today to review our performance and financial for the fourth quarter and 2014 year end. Last year was transformative for us, with a number of notable accomplishments. We are now a growing commercial stage biopharma company in the orphan space.

Developing and delivering groundbreaking small molecules that target RNA-based processes. We are proud to have launched the first approved treatment for Duchenne muscular dystrophy to patients with this devastating disorder. Translarna was approved by the European Medicines Agency or EMA last August.

We have been working with patients and efficacy groups throughout the development of Translarna and understand their urgent need for treatment. We are working hard to bring Translarna to patients as quickly as possible. We assembled the teams with substantial experience, commercializing product in the ultra orphan space.

We have now submitted pricing and reimbursement dossiers in key European countries. And in December of 2014, only four month after approval, we launched our first commercial program in Germany and we look forward to continuing our commercial rollout through 2015.

We also finished 2014, with reimbursed, early access and named patient program authorized in Spain, France, Italy, Turkey, Israel and Columbia. This year, we will also pursue reimbursed earlier access program outside the EU, including Brazil, Argentina and Australia. We are still in the early days of the launch.

We are pleased that feedback from both physicians and patients has been positive. In December, we also submitted a rolling NDA to the FDA for Translarna approval.

This will allow the FDA to review the majority of our materials ahead of the data from our confirmatory Phase 3 ACT DMD trial, from which topline results are expected in the fourth quarter of this year. We began building out our U.S commercial infrastructure in anticipation of potential Translarna launch in the first half of 2016.

Furthermore, Japan is also an important market for us. It is the second largest pharmaceutical market and we have made progress establishing the PTC’s footprint in Japan and we are working with regulatory authorities to determine the most experience regulatory path towards approval in Japan.

As you might recall, we have worldwide rights to Translarna and we intend to bring this important therapy to patients globally. We estimate there is an addressable market of about 7000 patients with massive mutation DMD worldwide and the commercial prospect for Translarna in DMD alone is over a $1 billion peak sales opportunity.

The rollout in Translarna will be staggered on the country by country basis in Europe and outside of the EU, where EMA approval is referenced. During the 2015 quarterly earnings calls we intent to provide you with the number of patients on Translarna therapy. Beyond 2015, we will evaluate the metrics that will be most useful.

I am pleased to share with you now that as of February 25th we had 42 DMD patients on commercial Translarna therapy through either reimbursed early access program or commercial sales. We anticipate the ramp up to continue throughout the year.

While we are very excited about the current commercial opportunity for Translarna and DMD, we are equally focused on bringing additional innovative therapies to patients. We achieved major milestones in 2014, with moving our pipeline forward and advancing new therapies into clinical development.

In cystic fibrosis, we initiated a second Phase 3 trial with Translarna, ACT CF. Enrollment is on track to be completed by the end of this year. We anticipate a data 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 this will occur in the second half of 2015, because Translarna is already approved in Europe, the CHMP review process could be three months. In recent years, the understanding of CF has evolved. The CF community now recognizes the disease comprised of multiple subtypes with differing levels of severity.

We believe that the recent data from the TRAFFIC and TRANSPORT trial has put expectations around the outcome of these different classes into new perspective. We feel confident that the FEV1 response we saw in non-TOBI patients in our previous Phase 3 data is clinically meaningful it would be a basis for U.S.

approval if replicated in our current trial, especially given that non-TOBI patient CF is one of the most severe forms of the disease. Because of Translarna’s mechanism of action, it has the potential to address a wide array of genetic disorder.

We are therefore developing Translarna in other indications to fully capture its potential as a product in the pipeline. This year we look forward to advancing another program into the clinic if Translarna moves into MPS I patients. Translarna is a small molecule which can cross the blood brain barrier.

Existing treatments do not address the CNS manifestation of MPS I, which can be quite severe. Well setting up sites and identifying patients for our open-label MPS I trial, we received tremendous enthusiasm from both physicians and patients for the potential to address this unmet medical need.

Based on this feedback, we have decided to amend our trial design to include patients currently on enzyme replacement therapy oppose transplant treatment, we do not fully address CNS issue. We continue to expect the data from this trial will be available towards the end of this year.

I will now shift to the SMA program, which is a partnership with Roche and the SMA Foundation. This program has moved into the clinic in 2014. As you may recall, as published in the journal Science we demonstrated that small molecule could be high selective in modifying splicing of the SMN2 transcript.

A Phase 1 study in healthy volunteers was completed in the spring of 2014 with impressive results. The result showed a dose dependent effect on SMN2 splicing, demonstrating proof of mechanism. These Phase 1 results will be presented in the first half of this year.

Based on Phase 1 results, a Phase 2 trial MOONFISH was initiated in November in SMA patient. This study will enroll approximately 50 patients with once daily oral dosing for 12 weeks. Now the primarily objective of this trial is focused on safety and tolerability, SMN protein production and changes in RNA level are also be monitored.

Exploratory end-point also includes muscle and nerve function measurements. We are also very excited about this program, both for the promise it brings to SMA patients and the validation of our splicing platform.

In November of 2014, we hosted our second Annual Science Day, during which we went into detail on additional programs advancing in our pipeline. For Translarna, which we’ve mentioned is a product in a pipeline.

In addition to MPS I, we discussed potential mix indications for which we plan to initiate at least one additional proof-of-concept trial later this year. In oncology, our cancer stem cell program, PTC596 will begin a Phase 1 study in advanced cancer patients with solid tumors in the first half of this year.

Lastly, given increasing antibiotics resistant, we are excited to have a novel antibiotic, PTC672, entering ANDA enabling studies for Gonorrhea. We look forward to sharing more with you about these programs throughout this year.

Before turning the call over to Shane who will walk you through the financial metrics for the year, I'd like to highlight one. PTC completed two very successful follow-on offerings in 2014, raising net proceeds of approximately $236 million. This contributed to our strong cash position of $350 million at the end of the year.

These funds will allow us to independently commercialize Translarna worldwide to investigate as a new indication and to continue to advance the exciting programs in our pipeline. With that, let me turn it over to Shane.

Shane?.

Thanks, Stu. 2014 was a key year in our corporate developments. We are now a well financed, commercial growth entity. We finished the quarter and year ending December 31, 2014 with over $315 million of cash and marketable securities on our balance sheet and no debt.

As Stu mentioned in 2014, we completed two successful financings through public offerings of common stock where we secured an additional $236 million of net cash proceeds. We believe that this cash position should be sufficient to fund our operations through late 2017.

Here are the financial results from the 2014 fourth quarter and full year, as well as financial guidance for 2015. We are now booking Translarna revenue from our commercial and reimbursed early access sales.

It is important to note that like many companies when beginning their first commercial sales, we are only recognizing revenue on a cash basis when payment is received, as opposed to when product is shipped and invoiced. In 2014, we had shift in invoice for $2.5 million in commercial Translarna product orders.

However, we’ve received payments of $0.7 million that was therefore recognized as revenue and reflected on our income statement. The $1.7 million of Translarna invoices that remain outstanding as of December 31st were booked as deferred revenue on our balance sheet.

We are working to determine the appropriate time to change our recognition of Translarna revenue. This will largely hinge upon our establishing a pattern of collectibility such that revenue will be recognized upon shipment, assuming all other revenue recognition criteria are met.

Total revenues for the fourth quarter of 2014 were approximately $12.7 million, compared to total revenue in the fourth quarter of 2013 of approximately $4.4 million. The increase was primarily due to the $10 million SMA milestone we received from Roche and recognized in the fourth quarter.

Total revenue for 2014 was $25.2 million versus $34.7 million in 2013. In total, we received $17.5 million in milestone payments from Roche during the year. So the overall decrease in revenue for the year was due to a reduction in the recognition of non-cash deferred revenue, compared to the same period in 2013.

Research and development expenses accelerated in the fourth quarter of 2014 in conjunction with our expanding clinical program, as well as in support of the launch of Translarna.

R&D expenses in the quarter were $26.9 million, including $3.2 million in non-cash stock-based compensation expense, compared to $15 million for the same period in 2013, including $1.6 million in non-cash stock-based compensation expense. R&D expense for the full year 2014 was $79.8 million, compared with $54.9 million for 2013.

As the year progressed, our expenses increased as a result of increased clinical development activity such as the initiation of ACT CF and manufacturing of Translarna product for both clinical and commercial supply, as well as activities in support of our launch where we have grown our regulatory, quality and CMC supply chain functions.

SG&A expenses were $18 million for the fourth quarter of 2014, including $3.5 million in non-cash stock-based compensation expense, compared with $7.5 million for the same period in 2013, including $1.7 million of non-cash stock-based compensation expense. And total SG&A in 2014 was $44.8 million versus $25.2 million in 2013.

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

In support of our commercial launch activities, we have added to our organization across many roles, including commercial, marketing, medical affairs and medical information, as well as financed, legal, IT and facilities costs associated with establishing our international infrastructure.

The company reported a net loss of approximately $27.3 million for the fourth quarter 2014, compared to approximately $17.9 million for the same period of 2013. The net loss for the full year 2014 was $93.8 million, compared with $51.6 million for the same period in 2013.

Looking forward, we anticipate operating expenses for the full year 2015 to be between $160 million and $170 million, excluding expected non-cash stock-based compensation expense of approximately $30 million, for total operating expenses of approximately $190 million to $200 million.

We currently have 33.6 million shares issued and outstanding, which includes 0.7 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?.

Thank you, sir. [Operator Instructions] Our first question comes from Ritu Baral of Cowen. Your line is now opened..

Hi, guys. Thanks for taking the questions.

Do you guys have expanded access programs in other geographies that are not reimbursed and if so, how do enrollment in those compared to the overall number, the 42 number that you gave us today?.

Hi. Ritu, thanks for the question. I will let, Mark talk little bit about that..

So, thank you for the question. We are focusing our Expanded Access Program on countries, which have a recognized reimbursed early access program. And as we’ve mentioned previously that includes countries in Latin America, other countries in the European, Middle East and Africa region and we are now looking into the Asia-Pacific region.

In addition though we've been evaluating a compassionate used program for additional countries where reimbursement is not possible today and we will be coming back to you in the future with more details around that..

Great.

Any breakout that you can give us on the 42 patients either between reimbursed expanded access or German patients, any color you can give us on the breakdown?.

At this time, we are not actually splitting out patient numbers by different geographies. But suffice it to say that those numbers do include a combination of both commercial drug into Germany and sales into early access markets..

Great. Thanks. And last question, I will hop back into the queue.

Moving to the MPS I protocol amendment, how do you think that may change either statistics on endpoints or how you evaluate a potential clinical benefit in these patients, especially for post-transplant patients who may have some impact I guess on cognitive function?.

Yeah. Hi. That’s a good question. Especially, if you think about the ERT treated patients, which don’t pass the -- which doesn’t pass the blood brain barrier that you will be able to see higher levels of GAG in the CFS. So we think clearly, we will be able to see reductions there in that and clearly for patients that this is for proof-of-concept.

And so we think we’ve talked to key investigators in the CEEZAD. Any change in the CFS would be actually promising. And then with more innovative approaches measuring GAG levels, I think we will be able to look not only at the CFS but GAG levels in plasma and urine as well..

Great. That’s helpful. Thanks for taking the questions..

Thank you. Our next question comes from Christopher Marai of Oppenheimer. Your line is now opened..

Hi. Good morning, guys and congratulations on the quarter and the initial launch. Stu, I think my first question is really regarding your comments that you are looking at the Japanese market. I was wondering now how if you want to potentially address that.

Are you looking at regional partnership? Or how should be your strategy in Japan, particularly in DMD, where obviously there are patients? Thank you..

Hey, Chris.

Hey, Mark, why don’t you…?.

Okay. Hi, Chris. I am sure you are well aware that your Japan is the second largest pharmaceutical market in the world, and it’s one that is very friendly towards orphan drugs. So we are intending to build up our own infrastructure at PTC in Japan.

And we’re very pleased by the progress we're making in terms of engagement with regulators and with key investigators. And right now, we’re working on is the most expedient regulatory path that we can undertake in agreement with the regulators to enable access for Japanese patients as quickly as possible..

Great.

And is there any timeline on access for those patients to provide at this time?.

Yes. We have been -- we are working on that right now. I think one of the interesting things is, we are already on the Japan’s DMD, a drug lag list.

As you might know that drug lag list is for getting for those diseases that are for very high unmet medical need and that the key physicians, who are trying to figure out what are the -- what drugs need to come into Japan have put actually Translarna already on that list. And I think that really does highlight the importance of Translarna.

And knowing that they are already very well aware of it and made it up a priority to try and share rapid access to patients. So we think that’s exciting for us. We are looking forward to working closely with the regulators to expedite the approval of Translarna so that patients can receive that in Japan..

Sounds great. And then one last question here on the U.K. Obviously, there is some noise there and there is a bit of political, what have you going on about reimbursement of drugs. And I was just wondering where has that put you in terms of your estimates for potential sales into the U.K. market and any update on that front? Thanks..

Okay, Chris. So I think you are aware that the National Health Service in England has been changing its decision-making process around funding for ultra-orphan and specialized drugs. You heard that last December. But with the latest information we have, we’re still expecting a decision on funding by late second quarter this year.

So I think things are tracking in line with our expectations. You probably also saw some that we see as very meaningful that Duchenne muscular dystrophy patients in that families were in London quite recently demonstrating outside of parliament to demand Translarna’s access immediately.

And even the Prime Minister came to meet with these patients and their families. And out on the floor of parliament, he emphasized how important it is that patients get access to orphan drugs, like Translarna quickly..

Great. Thanks. That’s really helpful. And I appreciate the question..

Thank you..

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

Good morning. Thank you very much for taking the questions and congratulations on everything that’s happened this year and this quarter. Maybe I will start with the quick question on for Shane.

Shane, is it possible to tell us how many patients these 0.6 million come from? Were they all from Germany? And can you maybe touch on COGS?.

Yes. So just quickly we -- our sales are predominantly through Europe through the combination of both commercial and early access sales. And I think as Mark alluded to earlier, we are not really breaking out either patient numbers or revenue numbers in terms of early access versus commercial.

In terms of the cost of goods sold, I think consistent with our conservatism around revenue recognition, and I think we’ve previously disclosed that we’ve also taken a more conservative position in terms of cost of goods where we have been expensing through the R&D line all Translarna supply both clinical as well as commercial up until through probably the rest of this year..

Okay. Great. A question for Mark.

Mark, can you maybe help us understand the level of awareness and excitement? I think in the previous question you alluded to that, but how much different countries know that the drug is available and how quickly do you think that once the specific country for example would be selling the drug, you could see people -- you see patients go on board?.

So we’ve got really great feedback coming from physicians and patient organizations. And as you can imagine, the Duchenne muscular dystrophy community is a very connected community. So there is generally a lot of awareness out there.

As to access, well, you know, I think you will appreciate that in Europe whilst you have one approval for 31 countries, each country has their own access mechanism that takes different time frames. And so if you like launching in Europe actually is a scaling up of whole sequence of different launches over those 31 countries over time.

So really our focus has been to engage with the payers in different countries to initiate those access conversations..

Last question, Mark, for you also.

Where are you with building out your team? Are you at 100%, halfway there in terms of building out everything in terms of labor structure and personnel?.

Thanks for the question. A great deal of our focus as you can imagine over the 12 months has been on the European launch. So we’re at good 80%. The infrastructure is kind of 80% built, as I said, in 31 countries in Europe. So essentially there is still some gap. So we are going to be fitting over the coming months.

A lot of our attention now is focused into prelaunch work and preparation for U.S. launch next year as well as early access program support in Latin America. And we mentioned earlier, beginning preparations in Japan as well..

Thank you very much..

Thank you. Our next question comes from Alethia Young of Deutsche Bank. Your line is now open..

Hey, great. Thanks for taking my question and congrats guys on the progress. Just one simple question to start off with.

When you look at the 42, like, how does that match up versus your expectations where we stand now, like if you think big picture?.

Hi, Alethia, thanks for the question. Hey, Mark, why don’t you….

Well, you are asking for a relative measure here, we really feel that launch is on track. We are very pleased. You think about the fact that thanks to our efforts to put in place early access program.

We’ve actually enabled patients to go on reimbursed therapies six to 12 months before they might have got drug through the conventional commercial only route. So I think we are in a great place and building some good momentum..

Okay. And then just go into your CF Translarna kind of rolling filing here in the second half of the year. I mean, do you think that the groundwork you’ve laid over DMD would make this progress a little bit easier? I know you know that that might take around three months.

But I just wanted to know what incremental needs to be provided? Is it just the data, or is it something more around ataluren that they will be looking for this time?.

Hi. Thanks. So clearly what we are trying to do is just follow the playbook that we did for Duchenne muscular dystrophy, knowing that there is a variation as a consequence of having the approval. And it will be based on the current data that we have now. So it’s really just setting it up from there..

Okay.

And then just talking about 596, can you talk about what the mechanism is there with the cancer stem cell?.

Sure. So the mechanism is the target is the BMI program and it causes the reduction of the BMI protein levels.

And just to remind everyone, it’s an important cancer stem cell that regulates many of the important regulatory pathways, such that by reducing that you reduced the level of -- it not only inhibits tumor growth, but reduces or brings to low levels those cells that can be, in a sense stem cell like to either metastasize or grow. So it’s novel target.

And so that -- so we are targeting it by reducing the levels of BMI-1 protein production..

Okay.

And then just one last on Translarna, going back to the price, have pricing and reimbursement kind of from what you know as far as you move forward kind of remained in line with what you saw early on in the launch?.

Hey, Mark, why don’t you take that?.

So I am sure you are aware that in the ultra-orphan space, there is always negotiation around access and our job is to explain Translarna’s value, the high unmet medical need and this process is not always easy, but I believe we have some of the best people in the field working with us.

And given that Translarna is really an innovative therapy and the first to be approved for Duchenne muscular dystrophy, and it’s been developed for very small patient population with the very high unmet medical need that we are in good place, we have good prospects. So those negotiations really are taking place over the coming months..

Hey, great. Thanks..

Thank you. Our next question comes from Yaron Werber of Citi. Your line is now open..

Hi. This is Kumar Raja in for Yaron. Thank you for taking my question. Out of these 42 patients, can you tell us how many are from earlier clinical trials versus the non-clinical trial patients? And also, can you provide us update on what patient identification efforts are? And I have a one more follow-up question on SMA..

Okay. Thanks for the question. In terms of the number of patients who are on previous clinical trial, we really those patients -- we didn’t really have any patients, who were previously on clinical trial, so there is no real overlap there. In terms of the second half of the question, I’ll pass it over to Mark..

So you’re quite right to mention patient identification in genotyping, we’re doing a lot of work to find patients and map them to treatment center and it’s really a key priority for us. Our goal is to get the vast majority of eligible patients from drug as quickly as possible.

And now that there is a treatment available, you can understand that there is a higher incentive outlet for people to genotype in the community. However, it is rapidly evolving and the numbers of genotype do vary from country to country. And we’re putting in place things to support genotyping country by country.

So for example, we’re making sure that we know where all DMD patients are being genotyped currently, but we're also looking at finding centers, which are maybe smaller centers that are less obvious to ensure the patients in those centers that also getting genotype.

So really it’s sort of mix to max strategy where we do whatever we can to help an efficient country to make it the more efficient and ones that are less efficient to put more in place to make them become more efficient..

Thanks. And for the MOONFISH Phase 2 data, looks like the time move slightly from like late'15 to now in '16.

Have you guys made any changes there in this trial for this delay?.

Yeah. Thanks. So just to remind everybody, the MOONFISH is the SMA trial in patients. And I think what we’ve always said is that it will take about a year, so I think we’re on track for that. So this is just going to be about 50 patients that will be recruited for this trial. So I think we feel good about this completing in a timely manner..

Okay. Great. Thanks..

Thank you. Our next question comes from Jason Kantor of Credit Suisse. Your line is now open..

Hey, good morning. This is Jeremiah filling in for Jason. Thank you for taking the questions.

I mean, you touched on the recognition that Translarna is getting in Japan, but do you expect that the current development program is efficient for Japanese approval? Or do you think you might need another study particularly with maybe the majority of Japanese patients?.

Yes. Hi, Jeremiah.

So Mark, why don’t you talk a little bit about our plans?.

Okay. So as we mentioned earlier, I think it's great news that the drug is already on the drug lag list. So that means that the authority see this is a high unmet need and in a way want to work with us to find an expedited path to get the drug to patients in Japan.

We are still working on the precise nature of the kind of study that we may have to do to enable that to happen. But again, the framework for those discussions is to try and find the solution as quickly as possible..

So we anticipate I think just to follow up a little bit that, we think there need to be some PK work done, but we don’t necessarily anticipate it to be that onerous, but we’ll be working with the regulatory authority to define the most expeditious path..

I know it’s still early in the launch, but how quickly do you expect to get paid for invoice sales? Should the -- would the deferred amount be recognized as a one quarter lag or maybe potentially longer?.

Hey, Jeremiah. Thanks for the question. Obviously, we’re still early in the launch. We just launch in Germany about two months ago. And so our experience in terms of getting payment is still relatively nascent.

So our expectations so far, we’re getting paid on what we think it to be a pretty timely manner, but we’ll see how experience goes throughout the year and as we launch from country to country to country..

Just one last question in terms of SMA. You mentioned that we should see that Phase 1 data in healthy patients in the first half of this year, if I heard it correctly.

But also when might we see the natural history in the biomarker data for the SMA program as well?.

Yes. Clearly, there is a natural history of program that’s been ongoing. And so I think as we as a group collective, there will be appropriate venue. I don’t think we need to find exactly when that will be presented at this time..

Okay. Thanks for taking my questions..

Thank you. Our next question comes from Tazeen Ahmad of Bank of America. Your line is now open..

Hi, guys. Thanks for taking my question. Can you give us a little bit more granularity on what the ex-U.S. DMD populations are maybe by of region? I know EU is a little bit higher than what it is in the U.S.

But can you potentially give us estimates of addressable populations in South America and Japan?.

Hey, Tazeen. Mark, don’t you sort of go through that a little bit..

So our estimates in the DMD population about 10% to 15% have nonsense mutation, we typically use 13% for planning purposes. And if you look outside of Europe for example where there are 2,500 nonsense mutation DMD patients, our estimates are around 2,100 for North America.

Big countries in Latin America like Brazil, we’d estimate around a 1,000, couple of 100 in Argentina and Columbia. And then out in Japan, probably a minimum of 600, possibly a slightly higher incidence nonsense mutation DMD in Japan, Turkey, for example, another important country with around 400 nonsense mutation patients.

So we’ve seen in the countries we’re currently covering and intending to cover around 7,000 nonsense mutation DMD patients..

Okay. Thanks. That’s very helpful.

Can I ask how many of -- in your current Phase 3 trial, how many of the patients are ex-U.S.?.

Yes. We have a good mix between U.S. and Europe and we don't really -- we haven’t really defined the cut. There’s about 230 patients in the trial now. It’s the largest DMD trial ever done, but we haven’t yet cut out how many are in Europe and U.S..

Okay. And then one question on pricing, I think you said in the past that you expect the average ex-U.S. price to be around $300,000.

But in terms of the countries you’re choosing to launch at initially, could we assume that the price that you’d be able to get in those countries might be a little bit higher than that?.

So we’re not -- we don’t really talk about country by country pricing. And we’ll continue to guide overall our planning assumption is a net $300,000 per patients. Take into account numerous factors, like weight -- it’s a weight-based drug, exchange rates potential, discounts in some areas, et cetera.

So our blended number from a planning perspective is $300,000 net..

Okay. And then last question is in terms of guidance you expect going forward, it was really helpful to hear the number of patients that are on drug.

But going forward, how are you thinking about of sort of guiding the street on how the launch is going?.

Yeah. I think -- thanks, Tazeen. I think as we talked about, there is still a lot of variables that are happening. This is the first launch ever into this community.

And so at least at this point, we won’t be giving any forward-looking guidance in terms of either revenues or patients but we intend to report each quarter where we are in terms of actual patient numbers. So, hopefully that will be helpful as you track our launch..

Okay. Great. Thank you..

Thank you. Our next question comes from Anupam Rama of J.P. Morgan. Your line is now opened..

Hey, guys. This is Eric in for Anupam this morning. Most of my questions have already been answered but I thought, maybe I’d circle back to MPS I.

Maybe -- perhaps, I was just wondering if you could offer a little more color on the feedback you’ve been getting from physicians about Translarna's potential on MPS I? And whether there is anything in your pre-clinical data that points to it that as a potential there on top of ERT and whether or not in your proof-of-concept study you are now considering a more of a randomize type of the line there? Thanks..

Yeah. Thanks for question, Eric. When you think about MPS I, clearly the CNS, cardiac and bone are clear issues and that’s why there is still a significant unmet medical need.

And we’ve done -- and I think we’ve talked about this previously in our study where we saw a reduction in GAG level, not only in plasma but also in brain and various other tissues and that’s not surprising to us considering that. We know that Translarna is a small molecule that can pass the blood brain barrier.

So, we think that there’s a real advantage considering that patient. The fewer patients that are either not ERT, or had bone marrow transplant that they would be able to help those patients, even though they’re on either ERT or bone marrow transplant, to be able to help where there’s still real issues there.

So, we think that’s a real advantage to be able to do that. And that should help I think, unlock the full potential of Translarna and be valuable in addressing the major unmet medical needs in this patient population..

Okay. Thanks..

Thank you. Our next question comes from Debjit Chattopadhyay of ROTH Capital Partners. Your line is now opened..

Hey. Good morning and thanks for the questions here. Just quickly on Japan first.

Would need to keep these studies in healthy volunteers, or would that be a DMD patients and does that similar kind of framework apply to the two major Latin American countries?.

Hey Debjit. How are you? Thanks for the question. Yeah, we are currently in discussions with the Japanese regulatory authorities. So, I think it’s a little too early to actually define precisely the regulatory there. So as we go through the discussions, I think we’ll give more color, as we are more assure on where we are at.

In terms of Latin America, we don’t think there will be any other criteria that’s needed there..

And do you think the Japanese approval could potentially come ahead of the ACT DMD Phase 3 readout, or do you think they are going to potentially hold up and wait for the data readout?.

Yeah. I think right now as we’ve really initiated the regulatory discussions and talking with the investigators. I think it’s too early to give timelines in terms of where we are at right now in terms of when that will happen..

And on -- there have been about 700 to 800 patients in Translarna clinical studies to date, right. And most of these patients have rolled over to some sort of a maintenance program or compassionate use program.

How are you following the outcomes of these patients over the long-term?.

Yeah. So a couple points there. Clearly in completion of both the Phase 2a and the Phase 2b studies, well, we terminated it after the 2b that we then started extension studies So we’ve been collecting a predominantly safety data but in Europe some efficacy either once or twice a year, so we are accumulating that.

And so we will be accumulating that over time. And then at the appropriate time, obviously present something from there. In addition, we will be in the post-marketing setting. We are setting up registries in which we’ll continue to collect longitudinal data for patients who are getting either expaned access or commercial drug.

So, we can get additional information on the long-term benefits of Translarna. And then in the current studies where there is an extension study where we’ll continue to get additional data of that..

So at -- what I am trying to get at is, at this point do you have any sort of fuel as to -- especially say in DMD, how these patients are tracking with respect to natural history studies?.

Yeah. Because it’s somewhat complicated where they went on and off and the minimal datasets we have. And as we collect it, I think at the appropriate time we’ll be able to present that at a venue. I think we’ve encouraged that virtually all of the DMD Translarna patients to date have stayed on therapy and some even up to four years.

So it’s very well tolerated and at the appropriate time, we’ll be presenting some of that data..

And do you guys plan to update on country-by -country approvals or you will just update patient numbers at the end of every quarter?.

So as we said earlier, we will be providing updates on patient numbers on reimbursed therapy on these calls and no doubt, there will be over time visibility into countries, where those launches have taken place..

And if you expect to hear back some nice by the middle of the second quarter, would that still be in time to be on the schedule for 2015 reimbursement or does this push it out to 2016?.

The current expectation is that a decision would be made by the end of second quarter for funding in the second half of this year..

Great. Thanks so much and good luck..

Thank you..

Thank you..

Thank you. [Operator Instructions] Our next question comes from Heather Behanna of Wedbush Securities. Your line is now opened..

Hey, guys. Congrats on the progress and thanks for taking the questions. Just the question a little bit on Latin American sort of a follow-up on the patient identification.

When we think the Latin American opportunity, our most of the patient identified or treated in concentrated centers or is it more of a fragmented system compared to some countries in Europe?.

So what we have found so far is that in countries like Brazil, there are a number of very important centers with relatively large numbers of patients. But then beyond there is a sort of more fragmented picture.

So as we establish ourselves in Brazil, we’ll be initially focus more on supporting the big centers, but in time expanding into new geographies as well..

And to follow-up on that, I think we are working hard not only on working with them, but also genotyping just making sure that the patients are all genotyped..

Great. Thanks. That’s helpful.

And then a quick question for Shane, just with -- would you guys being domiciled in Ireland? If you could give any color on just how we should tax rate moving forward into Ireland?.

Yeah. I mean, if you think about what we've done with our -- setting up our international headquarters in London. I think many other companies, sorry, in Dublin -- many other companies have had similar tax structures. I think ultimately it recognize the blended tax rate in the low teens.

I think we are still little early in terms of giving any guidance on where we would ultimately land..

Okay. Fair enough. Thank you..

Thank you. And our final question comes from Christopher Marai of Oppenheimer. Your line is now opened..

Hi, guys, and thanks for taking the follow-up. I was just wondering on the MPS disorders, obviously, there’s other nonsense mutations affected patient beyond MPS 1, wondering how you are looking at those.

Number two, how are you measuring urine GAGs, just remind us, I remember some conversations that you may be looking at a different sort of methodology to test out during this trial and I think it was may be BioMarin who had a sort of proprietary method here? And finally, how do you think about tissue distribution and activity dependent concentration -- activity dependence on dose with respect to MPS? Obviously, you want to cross blood brain barrier and I think you guys do, but also there are many tissues that are unaffected by ERT? But obviously, we would want to see activity across the board in those for the ultimate benefit of patients, you may be comment on that? Thanks..

Sure. Thanks. Okay. So lets -- we’ll break that down to the other lysosomal storage disorders, you are actually quite right. There are other ones that one could easily measure and look for reduction and that there are nonsense mutation patients.

And clearly, what we are doing is thinking about what other indications of that one could actually do in terms of proof-of-concept. And so we are actively thinking hard about that as well and I think we’ll talk about that as we make decisions on that. In terms of the GAGs, there are now, again you are quite right there a nice discussion.

I think BioMarin have the nice method or has developed a nice method in terms of being the able to more accurately monitor GAG levels. There are -- that ones that are similar to that as well and we’ll be using, I think, some more updated methods for measuring GAG levels both in the CSF, as well as in blood and urinary GAGs level.

So and I think that sort of goes to your point in terms of tissue distribution the fact that we’ll be taking measurement both in the urine, as well as blood, as well as in the CSF, will give us an opportunity to see some of that see differences and distributions to the effect in different tissue types.

So, again, because Translarna is an orally bioavailable molecule that distributes well throughout, the tissues were encouraged by the possibility of being able to go to tissues where ERT may not be able to go to..

Great. And just a quick one on Emi1, do you have any plans to remind me to go into hematological malignancies? That’s all. Thanks..

Yeah. That’s a good question. Obviously, we are starting out with the typical Phase 1 oncology study that looks at PK and safety and then examines tumor response. But we are also excited -- and that’s going to be a escalating dose.

And but we are still -- we are excited about hema mal as you move forward -- and as we move forward and we hope to rapidly move as we get more closely to the appropriate dose to looking those indications as well. So that’s in our game plan as well..

Great. Thanks. Congrats on the progress..

Thanks. Thanks..

Thank you. And that does conclude our question-and-answer session portion. I’d now like to turn the call back to Mr. Stuart Peltz for any closing remarks..

Thank you and thanks for being on the call. And I think as you’ve heard we are in an excitement place where we are commercializing Translarna and developing our robust pipeline in that. We look forward to updating you over the year. Thank you for your interest and for joining our call today. Thanks again..

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