Good day, ladies and gentlemen, and welcome to the PTC Therapeutics First Quarter Financial Results Conference Call. At this time, all participants are in a listen-only mode. Later, there will be a question-and-answer session and instructions will follow at that time. [Operator Instructions] As a reminder, this conference call is being recorded. I would now like to turn the conference over to Jane Baj. Ma'am you may begin.

Thank you. Welcome to PTC’s call to discuss first quarter financial results, our plans for the Emflaza launch and other business updates. Before we start, let me remind you that today’s call will include forward-looking statements based on current expectations. Forward-looking statements include all statements, other than those of historical facts including statements concerning financial guidance, our expectations with respect to the future commercial availability of and access to Emflaza including timing of the commercial launch, annual pricings and patient insurance mix, the anticipated benefits of the Emflaza acquisition, our future expectations regarding other clinical, regulatory and commercialization matters including with respect to potential outcome and anticipated timelines, addressable patient populations for Translarna and Emflaza and the potential success of Translarna for the treatment of nmDMD and Emflaza for the treatment of DMD. Actual results may differ materially from expressed or implied by forward-looking statements as a result of a variety of risks and uncertainties including those related to our ability to finalize preparations for commercial launch of Emflaza and effect such launch including resolution of outstanding regulatory, distribution channel and commercialization matters, our ability to secure adequate pricing, coverage and reimbursement terms with third party payers for our product in a timely matter, whether and to what extent third party payers impose additional requirements before improving Emflaza prescription reimbursement, our ability to integrate Emflaza into our business and realize the anticipated benefits of the acquisition, changes in laws and regulations, our ability to satisfy our obligations under the terms of the credit and security agreement with mid cap financial trust and those risks discussed under the heading Special Note Regarding Forward-Looking Statements and Risk Factors in our first quarter Form 10-Q which is available from the SEC and 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. We will disclose certain non-GAAP information during this call. Information regarding our use of GAAP and non-GAAP financial measures and a reconciliation of GAAP to non-GAAP is available in today’s earnings release. With that, let me hand the call over to our CEO, Stuart Peltz.

Good morning. Thank you for joining the call to review what has been a busy and eventful first quarter at PTC. We are very happy to report strong progress on Translarna net sales globally, totaling $26.4 million dollars for Q1 which represents a 40% increase over first quarter 2016 sales. We are pleased with the continuous progress of the Translarna business since launching in 2014. We are also very excited to have completed the acquisition of Emflaza and have been rapidly preparing to launch this product and make it available to patients. The need for broad availability of Emflaza in the United States has been clear to us, and the strong demand to date with over 900 prescriptions and start forms submitted by physicians gives us even more confidence in the launch. On today's call, we will share details of the Emflaza launch. Because patient access is key to us, we have concentrated the effort on having programs available to help eligible patients access Emflaza regardless of their financial or insurance status and as well as minimize or eliminate their out-of-pocket expenses. We will then speak to our growing ex-U.S. Translarna Duchenne business followed by regulatory clinical program updates including SMA. We will wrap up with the financial results for the first quarter. Let's begin with an update on Emflaza. Since founding PTC nearly 20 years ago, we've been focused on addressing the unmet medical need in Duchenne muscular dystrophy, a rare type of a genetic disorder that leads to progressive muscle weakness. It has been our mission to fundamentally change the lives of patients with Duchenne. After years of research and development, it was a tremendous milestone for the Duchenne community and for PTC to receive marketing authorization from the EMA in 2014 and launched Translarna for Nonsense Mutation…

Thanks, Stu. As Stu mentioned earlier in the call, Emflaza is a great strategic fit on many levels. Beyond addressing a significant unmet need, we believe Emflaza leverages our existing commercial platform. It diversifies PTC’s business enabling revenue generation through two products and expands our global commercial geographic footprint to include the United States. We are looking forward to introducing Emflaza to the U.S. market and are finalizing preparations with the goal of launching in the coming weeks. Let me start with the current demand we are seeing. Of the estimated 9000 Duchenne patients five years and above who are eligible for Emflaza, approximately 10% or over 900 prescriptions and START forms have already been submitted to our hub services center which we call EMFLAZACares. We will work to enable these patients to initiate treatment with Emflaza as quickly as possible once the launch is underway. Our hub services teams will help each patient navigate the insurance coverage process which may take a few weeks to few months to conclude depending on the path. As is typical for a rare disease, we would expect payers to require prior authorizations to be in place and in some cases to require additional steps before Emflaza can be made available on a reimbursed basis. The varying timelines to put insurance coverage in place impacts the speed at which we can recognize revenue. While we have not yet launched our initial 2017 guidance for Emflaza sales, it is between $5 million and $10 million. As Stu mentioned, our ultimate goal is to help all eligible patients get access to Emflaza irrespective of insurance status or ability to pay. In terms of insurance mix, we estimate that half the patients will be covered by commercial payers and the other half by public payers, mainly Medicaid.…

Thanks Mark. Let me now touch on a few other important points. Our commitment has been and continues to be to provide access to Translarna to all patients who may benefit. Since 2005 over 400 nonsense mutation DMD boys and young men have participated in our clinical trials and the vast majority have chosen to remain on Translarna. We believe this long term compliance speaks to the benefits Translarna is providing to patients in the United States and around the world. We believe our data to-date and feedback from physicians and patients supports a positive benefit risk profile. We continue to interact with the FDA as our application is being reviewed. As you know we filed our NDA for Translarna over protests with the FDA and were given a PDUFA date of October 24. The PDUFA date is the target date for the FDA to complete its review. Let me know switch to another one of our programs which is based on our small molecule splicing platform. This technology has been new to discover potential new therapies for spinal muscular atrophy or SMA a rare genetic neuromuscular disorder that generally manifests early in life and is the leading genetic cause of death in infants and toddlers. We have a robust program in collaboration with Roche and the SMA foundation around oral SMN2 splicing modifiers. We believe that an oral therapy which has exposure throughout the body which includes both muscle and nerve tissues and has the potential to provide considerable advantages for SMA patients. The lead compound RG7916 is currently being studied in two clinical trials in SMA patients and each is expected to transition into the pivotal stage once the initial dose escalation portion is complete. The SUNFISH trial is in Type 2, 3 SMA patients and the FIREFISH trial is in Type 1 SMA patients. Data from the first portion of the trials are expected to be presented at a scientific conference later this year and early next year. Initiation of the pivotal stage of both studies is expected in the second half of 2017. Start of the pivotal stage from either study will trigger a single $20 million milestone payment to PTC from Roche. Before I turn the call over to Shane, I wanted to say a few words. As you know, Shane will be returning to investment banking at RBC to help lead its healthcare team and will be departing PTC later this month. Shane served with passion and dedication as our Executive Vice President, CFO and Head of Corporate Development for the past four years. Shane leaves PTC well positioned to continue our commercial growth and on track to deliver our plans for further development and innovation. On behalf of all of us at PTC, I thank Shane for all his hard work and dedication and wish him well in his new role. With that, let me turn it over to Shane to review our financial results for the first quarter.

Thanks Stuart. The last four years have been incredible learning experience for me and it's been a privilege to be a part of this growing team. PTC has accomplished an incredible amount over these short few years and I truly believe the company is well positioned to continue on a strong growth trajectory while pursuing novel and innovative science focused in the rare disease space. I would now like to review our financial highlights for the first quarter of 2017. As mentioned earlier in the call, Translarna net sales were $26.4 million for the first quarter of 2017 representing 40% growth versus the same quarter in 2016. We reported a net loss of approximately $29 million for the first quarter of 2017 which declined over $12 million from a $41 million loss for the same period last year. Our operating losses are anticipated to continue declining as we leverage our existing infrastructure against a growing revenue base now with two commercial products. GAAP R&D expenses were approximately $27 million for the first quarter of 2017 compared to approximately $31 million for the same period in 2016. Non-GAAP R&D expenses in the first quarter were approximately $23 million compared to $26 million in the same period last year representing a year-over-year decline of over $3 million. R&D expenses have declined as a result of reduced clinical trial expenses associated with the winding down of our ACT DMD and ACT CS extension trials. GAAP SG&A expenses were approximately $26 million for the first quarter of 2017 which were relatively flat compared to the same period last year. Non-GAAP SG&A expenses were approximately $21 million for the first quarter of 2017 compared to approximately $20 million for the same period in 2016 or an increase of approximately $1 million. SG&A expenses are expected…

Thank you. [Operator Instructions] Our first question comes from Tazeen Ahmad with Bank of America. You may begin.

Hi guys, this is Peter Stapor on for Tazeen. Congratulations on a nice quarter. Shane wish you the best in your next role. So few questions here. First what gives you confidence in higher Translarna guidance for the year?

Hi, this is Mark Rothera. I just wanted to say that guidance that we've given is based on our visibility of the demand that we’re seeing already in the 25 or so countries that we’re selling in around the world, and as you can tell we're already halfway through Q2, so we're very confident in raising that guidance.

Got it, okay. And also on Translarna for the U.S. approvals, are you expecting an adcom?

Yes, so well, obviously that's up to the FDA. We certainly believe that it deserves an adcom because it is a new chemical entity in the new area, and certainly it's important to do this and we think that we as well as the community deserves it.

Got it. Okay and last question on the SMA program, I was curious your thoughts on the recent AAN data from AveXis and Biogen and where do you think the PTC drug would fit into the SMA market?

Sure. Obviously we think that's promising, we think that the PTC-Roche collaboration that drug we think has an advantage of the – it’s orally bioavailable drug that hits, that actually distributes to all tissues and therefore goes to not only to the central nervous system but also to muscle and other tissues and organs that are affected, and so that, so we think it’s high, so we think that that is truly important and so therefore it gets all tissues. And on the other point also, we will be having I just also want to make a point that we will be having data coming up this quarter, this year I should say, as well as next year that we will both measure both the RNA levels as well as proteins that we will be presenting at conferences.

Thank you.

Thank you. Our next question comes from Alethia Young with Credit Suisse. You may begin.

Hi guys, thanks for taking my question and Shane I wish you the best. I’m sure we will see each other again. On the pricing of the $35,000, I mean I know you gave us a little bit of commentary but I'm just curious like how you think about kind of that pricing in the U.S. and have you gotten pushback from kind of payers as you've tested it, since it is much cheaper in Europe? And then my second question is on Translarna, can you maybe discuss the countries where you're seeing kind of the biggest contribution and maybe ones there are still opportunities so that we can help kind of frame where growth could go? Thanks.

Sure. Thanks Alethia. So I think, so from the point, I think the way we thought about this is that we wanted to make sure that we had sustainable pricing that balance the needs, getting them to all eligible patients from the United States, and that population is 9,000 patients. And prior to this approval, only a minority of patients were able to get that, so that was really important, so it allows us, the pricing was based on knowing that also while maintaining an adequate infrastructure and also to have programs that includes continued investment into the Duchenne community as well as to provide a financial return to our shareholders. So those were the things that really went into our thought into defining the price. Mark, do you have any other?

I will just add that we believe it's an important therapy that slows the progression of disease and as Stuart alluded to, we have some data that we’re looking forward to bringing out into a professional scientific meeting in the near term that speaks to the importance of this treatment and many patients haven't had access; and so, we’re committed bringing that to patient irrespective of insurance status or ability to pay.

And the second part in terms of.

I think you had a question about Translarna is that correct?

Yes it was, you know kind of, you are in 25 countries but kind of where are the ones there that are largest contributors at this point, where are ones where there's opportunity for you to grow the kind of frame where we could see growth go? Thanks.

Right, so to remind you, we've built a footprint of 47 countries, we’re in just over 25, so whilst we don’t go into specific countries, we've made a lot of progress in Western Europe. We're expanding into Central and Eastern Europe. We’re expanding into the Middle East. We're also making good progress in Latin America. So there's a lot of growth opportunities still ahead in the years to come. We are also doing a lot of work on patient identification. So even where we have a country where we're in we continue to grow by helping the communities better identify suitable patients.

And then just, just a follow up back to the pricing thing. So have you guys kind of vetted this price like with payers already or is there something that you will kind of do later, I'm sure you've vetted it with the community?

Yes, we've spent a lot of time since we announced that we were going to acquire the drug, engaging the different stakeholders including the payers and we've made a lot of good progress. We think the patients are going to get coverage. I think the typical things that we'll see a sort of typical of a rare disease drug of this nature that there will be prior authorizations to ensure that the right patients are getting the drug, and in some cases they may be a step edits through an alternative first, where medical necessity forms will be required to switch them to Deflazacort and Emflaza. So but overall we're seeing good progress and we're looking forward to continuing sharing with the payers the high unmet need, why this is an important therapy including the data that I referred to earlier.

Great, thanks.

Thank you. Our next question comes from Ritu Baral with Cowen. You may begin.

Good morning guys. Thanks for taking the question. One I'd like to begin a little bit further to the commercial infrastructure Mark that you mention in your prepared remarks, you mentioned hub services team for Emflaza care case managers and field force assuming you guys have MSLs as well, what is the total head count that's in place right now and then how do you think of time to sell, you mentioned a few months to a few weeks, what do you anticipate that time to sell being say in Q2 and Q3 of this year versus this time next year? And then I have a follow up as well.

So just on the commercial side we have a team of about 50 or half which are out in the field. You know they're very specialized team that know how to deal with rare disease drugs of this nature. They engage with all the different stakeholders, the payers, patient organizations, the physicians. In addition to that we also have the hub services organization that is supporting us that in Emflaza care. So we have a whole team set up there, ready to go to help patients and as you saw they're already 900 waiting focused, so there is prescription to be filled, so they will if you like hold the hands of patients through this system which can be a bit scary from a patient perspective getting reimbursement can feel a little bit scary at times from a patient perspective. So they are there to really help facilitate that process. Did I answer your question, are there any other aspects?

Yes, the case managers, are they part of the hub services team or are they part of that 50-50% commercial force.

The hub services team in addition to the 50 that I mentioned.

Got it and then, go ahead.

Yes, I think you mentioned the time to fill, well clearly we haven't launched yet. I mean I think what we're trying to do is give you some guidance on the fact that some payers it'll be a relatively straightforward process could just take a week or two others is going to be maybe a longer process that might take a few months. I think it’s a little early to give you kind of a specific guidance, but an average patient.

Got it and my last question has to do with the weight that you gave, the price that you gave was for a 25 kilogram patient does this reflect I’m assuming this means you're going to price on a weight basis and does this reflect what do you think the average patient will be, do you expect given the profile of the drug, heavier patients at first or younger patients at first, how do you think about that?

So we've gone with the typical patient because we think that limited to the paediatric conditions. We think this is going to be the most common way that which the drug you are going to be prescribed. And given our commitments to work with the community to help with earlier diagnosis and access to treatment even earlier we think that in the long run that represents a good weight to anchor this price on and yes some patients will be heavier and some will be lighter, but that I think is a good guide post.

Got it. Thanks for taking the questions.

Thank you. Our next question comes from Geoff Meacham with Barclays. You may begin.

Hi guys. This is Evan on for Geoff. Congrats on the quarter and the progress. So, going to Emflaza, what has the payer feedback been than thus far with the price. With access I know you said that there is, some will be, some payers will be easier to work with, some will be more difficult? And then also how do we think about the Medicaid, because I know probably a lot of these patients could be covered by Medicaid due to their disability in the United States and would have access will be there for those patients?

Yes, I mean we've already put a lot of work into engaging with payers and I think this is going to be a constant dialogue because remember this is a rare disease, they are not that familiar with and I think it is incredibly important that they understand, this is a very severe and high unmet medical need and it is very important to bring a drug of this nature which has proven efficacy to help slow down disease progression. So, in our experience today it's just a question of time rather than is on the whole this is about going through certain steps like a prior authorization or a switching from prednisone, a medical necessity form might be required. So it just depends on each individual patient's circumstances. As I said it’s more about time rather than whether it will happen or not. And on the Medicaid side, I mean yes, I think it's important to recognize that that's around 50% of patients are likely to be covered by Medicaid in the long run and those patients who are not on the whole able to access the Deflazacort and Emflaza and so we’re very committed to help those patients through the reimbursement process and we have the programs in place like open to copay support for patients of this nature so they have little or no out of pocket cost.

And have you engaged with states to start getting coverage on Emflaza?

Yes we have.

Okay.

Yes we have, and also I think a lot of work has been done prior to the acquisition as well.

Okay and how has the DMD community received this whole, your acquisition and now you are basically your commercial launch plans and I guess what do you plan to discuss with them later today?

Yes, so I think you know we've been talking with them, I think the other point is that we've, we've been in this community for now almost two decades working with the community to bring new therapies to patients. So, I think, at the end of the day they know that we've been committed to this to the Duchenne community and that so, they know us and I hope and I trust that they trust us.

Okay, thanks for taking the questions. I appreciate it.

Thank you. Our next question comes from Joel Beatty with Citi. You may begin.

Hi good morning and thanks for taking the questions. My first one is have you had interactions with the FDA on your Translarna NDA since the filing of a protest?

Yes, we have, as we said I think in the last call that we began with the interaction, so we've begun that and so we won’t be talking much about those interactions because of the normal things that occur through the NDA and we won’t be disclosing things beyond that.

Okay. And then my last question is in cases when peers require a step at it before using Emflaza what if a patient wants to use Emflaza as first line therapy, how would you address the access to the drug in that case? Thanks.

Yes, I think the key point there is the docs that need to make the case for the switch and using a medical necessity form and there are a variety of reasons they might determine as why it’s important to make that switch. So I think with that in place, it is entirely possible for a patient to be switched.

Okay, thank you.

Thank you. Our next question comes from Matthew Eckler with RBC Capital Markets. You may begin.

Hey guys, good morning. Thanks for taking the questions, so could you tell us what you're assuming as the average per milligram cost for Emflaza?

Well I think probably the most relevant anchor point we've given you today is what we think the price will be for the typical patient which is 35,000 net per patient per year for the 25 kilo boy. I struggle to do the math here live is to how to translate that in a mill price.

So that maybe a different question here, when you guys have modeled out the launch opportunity for Emflaza, what do you see peak sales opportunity in the U.S. and then two really when do you expect this acquisition to be accretive?

Hi Matt, it’s Shane here from PTC. So in terms of the launch and peak sales we have not sort of, we're still nascent in terms we haven't even launched the drug yet, so I'm not sure we’re in a position to give you what sort of peak sales opportunity is. Clearly, we talk about the overall market which is up to about 9,000 potential patients five years and older in the United States and ultimately we would our goal is to ensure access for all of those who are in need of it. And what we gave on the guidance when we actually completed the acquisition was that we did anticipate that this transaction would be accretive to cash flow in 2018.

Okay, thank you.

Thank you, I'm showing no further questions. At this time, I would like to turn the call back over to Stuart Peltz for closing remarks.

Thank you. So to wrap up, we're pleased that the landscape of therapies for Duchenne boys has been positively changing over the last few years and I'm proud that PTC is bringing two of the three approved products to Duchenne patients. This is the mission we've been working towards over the last two decades. We believe that introduction of Emflaza, the first treatment for all eligible patients five years and over with Duchenne muscular dystrophy, regardless of the mutation type will serve as a catalyst to both raise disease awareness and improve access to the current standard of care. Thank you all for joining the call.

Ladies and gentlemen, this concludes today's conference. Thank you for your participation and have a wonderful day.