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

Alethia Young – Credit Suisse Anupam Rama – JPMorgan Evan Seigerman – Barclays Joel Beatty – Citi Ritu Baral – Cowen and Company.

Good day, ladies and gentlemen, and welcome to the PTC Therapeutics Fourth Quarter 2016 Earnings Conference Call. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session and instructions will be given at that time. As a reminder, this conference is being recorded.

I would now like to turn the floor over to Emily Hill. Please go ahead..

Thank you. Welcome to PTC’s call to discuss 2016 earnings, 2017 guidance, our recently announced planned acquisition of Emflaza from Marathon Pharmaceuticals 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 planned acquisition of all rights to Emflaza from Marathon Pharmaceuticals including with respect to matters of timing, financial impact and anticipated benefits to us and our integration and commercial plans and our future expectations regarding our other clinical regulatory and commercialization timelines and potential outcomes, addressable patient populations and the potential success of Translarna for the treatment of nmDMD and of our product candidate.

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 planned acquisition and confirmation thereof including satisfaction of conditions to closing the acquisition in the anticipated timeframe or at all; our ability to realize the anticipated benefits of the acquisition, including the possibility that the expected benefits from the planned acquisition will not be realized or will not be realized within the expected time period; business disruption being greater than expected; changes in laws and regulations and those risks discussed under headings Special Note Regarding Forward-Looking Statements and Risk Factors in our most recent Form 10-Q, which is available from the SEC or our website and our 2016 Form 10-K, which will be available from the SEC or on our website this morning.

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 please hand the call over to our CEO, Stuart Peltz..

Good morning. Thank you for joining us on the call today to review what we accomplished in 2016 and our developments in 2017. Today, we reported Translarna net sales of $25.1 million dollars for Q4 and $81.4 million for the full-year 2016, representing a 142% increase over full year 2015 sales.

We have a strong and growing business outside of the United States based on the expanding access to Translarna for nonsense mutation Duchenne muscular dystrophy patients around the world as Translarna was the first ever approved therapy in DMD, it the foundation of our business as we continue to establish our opposition as a global leader in DMD.

Let me first start with the big news that we entered into an agreement with Marathon for the acquisition of Emflaza. We’ll then speak to our growing global Translarna DMD business and provide a few regulatory and clinical program updates as well as the financial results for the fourth quarter and year-end 2016.

We are very excited to announce the planned expansion of our business with the agreement to acquire Emflaza. As we will be discussing on the call we believe Emflaza is the best-in-class anti-inflammatory disease modifying therapy for Duchenne muscular dystrophy.

We believe from both published data and data from our own clinical trial that Emflaza has improved efficacy and a desirable safety profile over existing steroid based treatment.

For nearly 20 years, we've been working to address the unmet medical needs in Duchenne muscular dystrophy, a rare childhood genetic disorder that leads to progressive muscle weakness followed by the loss of one's ability to walk, a loss of the use of one’s arms, the need for ventilation and death at far too early an age.

Despite evidence demonstrating that steroids are beneficial to DMD patients, they continue to be underutilized. Furthermore, prior to the FDA approval access to Emflaza has been limited to a small number of patients. We aim to close this gap with the launch.

Since 1998, we have been driven by advancing the knowledge and discovery treatment that can alter the course of DMD and fundamentally change the lives of patients, who previously had no hold. Since founding the company, we have made it a point to always consider the patient's point of view and to be an active committed partner to the DMD community.

Emflaza aligns with our mission to bring important therapies to patients with rear diseases with limited or no treatment options and we look forward to working with the DMD community to bring Emflaza to all eligible patients in the United States.

DMD treatment guidelines recommend steroids as a fundamental component of the standard of care as they reduce inflammation, which is critical to maintaining muscle integrity and delaying disease progression.

The Emflaza safety and efficacy result support that this is a disease modifying therapy for DMD patients due to its ability to slow the progression of the disease. Emflaza is the only approved therapy for all of DMD of patients five years and older regardless of their genetic mutation.

Given the ability of Emflaza to maintain motor function with a favorable safety profile, we will work with healthcare professionals, the DMD community and the public to ensure they understand the benefits of this newly available therapy, which in turn we’ll hope make it available for all eligible DMD patients in the United States as part of their standard of care.

As pioneers in the DMD community, we have developed substantial relationships with patients, physicians and other stakeholders in our efforts to bring best-in-class therapies to DMD patients. We are committed to make this important therapy available to all eligible patients in the U.S. and to study its long-term benefits.

Now let me hand the call over to Mark Rothera to discuss the commercial opportunities of Emflaza in the U.S.

Mark?.

Thanks, Stu. The planned acquisition of Emflaza is a really great strategic fit for PTC on many levels. Beyond addressing the significant unmet need, we believe Emflaza leverages our existing clinical and commercial platform.

This acquisition will diversify PTC’s business, enabling revenue generation through two products and expanding our commercial geographic footprint to increase the United States. We believe we are uniquely positioned to leverage our existing global commercial expertise in DMD and our U.S. leadership team to enable a successful launch of Emflaza.

Importantly, the near-term U.S. revenue is expected to be generated by Emflaza would also enable us to continue our deep investment in R&D in the Duchenne muscular dystrophy arena. Furthermore, an Emflaza launch would enable PTC to establish a complete U.S.

medical sales and marketing footprint ahead of a potential Translarna launch if we were able to obtain marketing approval. We're excited to introduce Emflaza to the U.S. market following closing of the planned acquisition.

We understand the importance of access and affordability to the community and we plan to reexamine the price Emflaza and work with key stakeholders to ensure patient access is a priority. We are finalizing our commercialization plans and intend to share more after the close of the transactions.

Let me wrap up by saying, we have a shared goal of improving the diagnosis and treatment of patients. This is expected to start with Emflaza and we hope will be complemented by a future Translarna launch in the United States. The U.S. DMD community needs a strong voice and partner advocating for improved standards of care.

We believe we are well-placed to undertake this mission. I'll now pass the call to our CFO, Shane Kovacs, to review the financial aspects of the transaction..

Thanks Mark. Let me start by turning to the slides on our website to review the details of the planned Emflaza acquisition. Last night we entered into an agreement to acquire all rights to Emflaza on a cash and debt-free basis from Marathon Pharmaceuticals via an asset purchase agreement.

We anticipate that the transaction will close subject to Hart Scott Rodino clearance and other closing conditions in the second quarter of this year.

The upfront consideration to be paid at closing includes approximately $75 million in cash and approximately $65 million of value in PTC common stock, or up 6.9 million shares, with any shortfall to be made whole with additional cash consideration, for total upfront consideration of $140 million.

The total number of shares to be delivered to Marathon at close will be calculated based on the 15-day volume weighted-average price ending on the third trading day immediately prior to close.

Beginning in 2018, Marathon will also be entitled to payments based on Emflaza net sales, which we expect will range from the low-to-mid 20s percentage on a blended average basis based on Emflaza net sales, as well as a single $50 million sales-based milestone. While we have strong U.S.

commercial leadership already in place, as part of the planned transaction PTC anticipates retaining certain key members of the Marathon Team who are expected to play a critical role in the launch. As Mark mentioned earlier, we believe there are approximately 9,000 addressable DMD patients five years and older in the United States.

Until recently, only a small fraction of these patients had been able to access Emflaza. More importantly, Marathon began an early access program in the U.S., and has been expanding access to the drug through those clinical sites that participated in the program.

It is our goal to enable access for all those patients who may benefit, and we believe that demand for access is high. Our highest priority is to enable continued access for patients currently using Emflaza, as well as access for new patients. Transitioning these patients through the reimbursement process can take time.

As we work to close the acquisition and prepare for the commercial launch of Emflaza, we will be better positioned to provide additional information. As mentioned earlier by both Stuart and Mark, we firmly believe in the strategic rational for this transaction.

From a financial perspective, this transaction will clearly diversify our business with a second commercial product and a new and very important geography, the United States. Furthermore, it will allow PTC to establish its U.S. commercial footprint, which can then be further leveraged for a potential future launch of Translarna in the United States.

Emflaza's FDA approval provides it with seven years of orphan drug exclusivity, and with the potential for an additional six months of exclusivity upon completing a pediatric study in patients under five years of age.

While the transaction requires incremental investment both to complete additional post-marketing studies, as well as the investment in U.S.-based sales and marketing infrastructure, we believe the expected future free cash flow will support PTC's ongoing research and development efforts as we strive to discover and develop new treatments.

We therefore anticipate an increase in our operating expenses in 2017 to support the launch activities as well as to begin the post-marketing commitments, while sales are expected to begin later this year. As we progress into 2018 and beyond, we anticipate this transaction will then become accretive, both to earnings and cash flow.

I'll pass the call back to Stu now. .

In summary, we're very excited to have the opportunity to bring this critical disease modifying therapy to DMD patients in the United States, helping to improve standard of care for this rare, fatal, genetic childhood disease. Let's switch gears and discuss Translarna's commercialization outside of the United States.

Our global launch of Translarna is tracking well, and the feedback we receive from physicians and patients about Translarna remains very positive. We have seen good growth year-over-year across Europe, Latin America, and the Middle East. Let me hand the call back over to Mark for further details on our commercial efforts.

Mark?.

Thanks, Stu. We built a strong sustainable and growing Translarna business globally. As you know, Translarna was approved by the European Medicines Agency in 2014.

Translarna is now available to patients in countries through commercial sales or reimbursed early access programs in Europe, Latin America, and the Middle East, representing a sustainable and growing ex-U.S. business. Today we reported net sales for 2016 of $81.4 million, representing a 142% increase versus 2015.

Our revenue guidance for Translarna net sales in 2017 is $105 million to $125 million, and we are confident we can meet this guidance. To remind you, we estimate that there are over 7,000 addressable Nonsense Mutation DMD patients worldwide, with 85% of these patients being ex-U.S.

Our goal is to make Translarna available to Nonsense Mutation DMD patients around the world as promptly as possible, since every day counts for them. Our strategy is to achieve this through both rapid geographic commercial expansion and increasing penetration of markets where reimbursed Translarna is already available.

Increasing penetration is achieved both through the conversion of known Nonsense Mutation DMD patients onto drug and the continued focus on patient identification through DMD awareness programs, as well as genetic testing activities. In addition, we continue to be pleased with the remarkably high level of compliance to Translarna therapy.

Let me now address these points in more detail. We have a global footprint in 47 countries and are selling Translarna in over 25 of them. We will continue to add new countries where Translarna will be introduced during the course of this year.

Growth is also being driven by increasing penetration in the countries where we're currently selling, in particular through the continued effort to identify patients.

Remember, we are leading this effort worldwide on the back of the first ever launch in Duchenne, and we are working with the DMD community to increase awareness of symptoms and ensure genotyping capacity and capability is rapidly available.

We are pleased that patient compliance to therapy in the commercial setting remains very high, estimated at over 90%, underlining the benefit patients are feeling. In 2016 we successfully navigated pricing reimbursement discussions in many key European countries at price levels that are sustainable to our organization.

In 2017 there remains select key countries where we expect final pricing and reimbursement to conclude by the end of the year. We expect a pricing and reimbursement decision from the French authorities in the fourth quarter of this year, where patients are currently receiving Translarna through a reimbursed early access program.

In Spain, we are looking to secure reimbursement on a national level in a challenging economic environment for orphan drugs. At the same time we have been selling through a reimbursed early access program on a regional basis in Spain for over two years, and we expect this system will continue.

Our goal is to bring Translarna to patients as quickly as possible. As part of our commitment to the DMD community, Translarna has been made available on a cost-free basis to hundreds of clinical trial participations, some for over 10 years.

Moreover, as the first Company to launch a therapy for DMD globally, we also feel a responsibility to help improve the standard of care generally, including increasing disease awareness to enable earlier diagnosis, enhancing access to multi-disciplinary specialty centers, improving steroid use and physiotherapy.

It’s very important to diagnose patients as early as possible. In some countries patients still aren’t diagnosed until seven or eight years of age, which is very late in the course of the disease. We’re investing in increased disease awareness with a goal of earlier diagnosis.

We are also investing in novel genotyping programs to make sure DMD patients know their mutation type to identify the appropriate therapy. Finally, we plan to seek expansion of the European label to include patients two to four years old, based on our ongoing pediatric study.

This would potentially increase the addressable patient population by an estimated 20%, and importantly allow treatment to preserve muscle function to be initiated even earlier, giving the possibility of better long-term outcome. Let me now turn the call back to Stu..

Thanks, Mark. Let me now focus on our efforts in the U.S. We have now filed our NDA for Translarna over protest 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. The file-over-protest mechanism allows us to have the FDA review our NDA after receiving a refuse to file in 2016.

We have led the way in pioneering the DMD development path, including the characterization of its natural history and its clinical trial designs.

Over 400 Nonsense Mutation DMD boys and young men have participated in our clinical trial dating back to 2005, and the vast majority have chosen to remain on Translarna, foregoing participation in other studies with alternative investigative therapies.

We believe this long-term compliance speaks to the benefit Translarna is providing to patients in the U.S. and around the world. We believe our data to date and feedback from physicians and patients support a positive benefit/risk profile of Translarna, as is confirmed by our EMA marketing authorization renewal.

With respect to our EMA marketing authorization, we have committed to conducting a longer term trial to confirm the benefits of Translarna in DMD. Translarna is also in development in proof-of-concept studies for aniridia and PS1, Dravet and CDKL5.

As a mechanism-based therapy, Translarna has the potential to of benefit a number of rare disease indications. Success in any given indication depends on a number of factors, including the protein levels necessary for clinical benefit and the sensitivity and variability of the endpoint.

We recently reported on results that did not demonstrate clinical benefit in cystic fibrosis. We are disappointed by this outcome for patients and other stakeholders, but we also understand this is part of drug development. While the CF program comes to an end, we continue to investigate the benefit of Translarna in other indication.

I would now like to focus on another program in our clinical pipeline. As we have discussed before, we have developed a platform technology to identify molecules that modulate splicing. This technology has been used to discover potential new therapeutics for Spinal Muscular Atrophy, or SMA.

SMA is a rare genetic neuromuscular disorder that is caused by a missing or defective SM1 gene, which results in reduced levels of SMN protein. The disease 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, small molecule SMN-2 splicing modifiers as way to address the disease. An oral therapy, which has exposure to both muscle and nerve tissues, has the potential to provide considerable advantages for patients.

We recently announced developments regarding the oral SMN-2 splicing modifier, RG7916. Phase 1 results were highlighted by Roche at the Child’s Neurology Society Meeting in Vancouver showing dose dependent increases in full-length SMN MRNA.

This data supported moving into patients, and the program recently advanced into a Phase 2 study in SMA type 2/3 patients. The study, named SUNFISH, is a two-part study investigating the safety, tolerability and efficacy of RG7916. The first part is a dose escalation study to evaluate safety and tolerability.

After dose selection, the study will transition into a pivotal efficacy segment. A similarly designed two-part study called FIREFISH in type 1 SMA patients is also enrolling. Initiation of the pivotal second part of both studies is expected midyear and will trigger a single $20 million milestone payment to PTC from Roche.

To wrap up, we are working diligently to bring therapies to DMD patients in the United States. Completing the acquisition and launching Emflaza will with the first step. We also look forward to the FDA completing its review of Translarna for Nonsense Mutation DMD this year.

Meanwhile, Translarna’s commercial presence continues to expand around the world and we continue to deliver strong commercial results. With that, let me turn it over to Shane to review our financial results for the fourth quarter and year-end.

Shane?.

Thanks, Stuart. I’d now like to review our financial highlights for the fourth quarter and full year 2016. As mentioned earlier in the call, Translarna net product sales were $25.1 million in the fourth quarter of 2016. This represents a 98% increase versus the same quarter in 2015.

For the full year 2016 we generated $81.4 million in Translarna net sales, which represents a 142% increase over the $33.7 million in Translarna sales generated in 2015.

We reported a net loss of approximately $27 million for the fourth quarter of 2016, which declined by approximately $24 million from approximately $51 million for the same period in 2015. Full year net loss for 2016 was approximately $142 million versus approximately $170 million in 2015, a decline of approximately $28 million.

Our operating losses are anticipated to continue to decline as we leverage our existing infrastructure against a growing revenue base. Non-GAAP R&D expenses for the fourth quarter were approximately $22 million compared to $31 million in the same period in 2015, representing a year-over-year decline of approximately $9 million.

R&D expenses have declined as a result of reduced expenses associated with the completion of our ACT DMD clinical trial. For the full-year 2016, non-GAAP R&D expenses were approximately $100 million versus approximately $106 million in the prior year.

Non-GAAP SG&A expenses were approximately $20 million for the fourth quarter of 20116 compared to approximately $20 million for the same period in 2015, a decline of approximately $2 million. SG&A expenses declined as a result of restructuring taken in early 2016.

Non-GAAP SG&A expenses increased sequentially versus approximately $19 million in the third quarter of 2016, or by approximately $1 million. For the full-year 2016, non-GAAP SG&A expenses increased to approximately $77 million versus approximately $64 million in 2015.

SG&A expenses are expected to increase as we expand our global commercial infrastructure. Our net cash burn for the fourth quarter was approximately $17 million, which represents a monthly burn rate of approximately $6 million. We ended the year in a strong cash position, with nearly $232 million in cash and marketable securities on our balance sheet.

We would like to take the time today to reiterate the 2017 Translarna net sales guidance we gave earlier this year of between $105 million and $125 million. Given this morning’s announcement regarding the acquisition of Emflaza, we will be revisiting our operating expense guidance and year-end balance sheet cash expectations on our Q1 earnings call.

We will now turn the call over to the operator to start Q&A..

Thank you. [Operator Instructions] Our first question comes from the line of Alethia Young from Credit Suisse..

Hey guys, thanks for taking my questions.

One, I wanted to ask about with this deal, maybe how you’re thinking about, like how much sales force you would need to add maybe in the United States? And then also what do you think kind of the peak, or kind of the frame the sales opportunities for us, maybe more on a little more of numerical fashion in thinking about this? And then also just, can you give us some general thoughts on what you think the right price is for Emflaza? Thanks..

Sure. There’s a couple questions in there. Let me get Mark to talk a little about the commercial..

Sure. Hi, Alethia. This is a, if you like, classic orphan drug launch that we’re facing in the United States.

So you need a very focused, not a particularly large organization that’s highly skilled and understands what it takes to bring drugs for a very rare disease of this nature to about 9,000 patients in the United States who have DMD that are five years and older and that would be eligible for Emflaza.

What I’m very pleased about is that we have an extremely experienced U.S. leadership team in place that has many, many orphan drug launches under their belt. And we’re also looking forward to supplementing that with key members from the Marathon team who have been involved in the execution of the launch to ensure continuity..

And as to the second question in terms of pricing, I think first of all to put in the context, we think that first and foremost we clearly believe that Emflaza is the best anti-inflammatory steroid which is disease modifying and that we’re committed to enable patient access in the U.S.

We appreciate that pricing is receiving a lot of attention and we believe that a change needs to be made. So let me pass back to Mark for just a little bit more clarity..

Sure, Stu. As Stu said, look, we’re re-examining the current price of Emflaza. The key is to ensure that all DMD patients have access to the best standard of care.

We believe Emflaza is an important new therapy that’s fundamental for standard of care because it addresses inflammation, which is key to preserving muscle function and ultimately slowing disease progression. So ensuring access to the drug is a priority, and until recently only a small fraction of patients had access.

We’re committed, then, to work with advocacy groups, physicians, payers and others to fully understand their interests and needs. Fundamentally we believe a change does need to be made. However, it’s premature to speculate exactly on what that price level is going to be at this time.

When the deal is concluded, we look forward to sharing our commercialization plans with you..

Thank you. And our next question comes from the line of Anupam Rama from JPMorgan..

Hey, guys. Thanks so much for taking the question. Maybe one for Shane. Know you mentioned broadly that the deal could be accretive but you didn’t define a timeframe on how we should be thinking about that. I know that you’re still working through the expenses and the pricing here, but what’s your base case assumption here for the timeframe? Thanks..

Hi, Anupam. Thanks for the question. I think clearly on the call and as Mark just alluded to, we will be building out a focused commercial team and so there will be incremental operating expenses with respect to that this year.

Clearly additionally there are post-marketing commitments that we will have to execute over the coming years with respect to the approval. That being said, therefore we’ll think about updating our guidance in terms of operating expenses probably on our first-quarter call as we close the launch and kind of finalize our plans.

But we do anticipate that going into 2018 that this deal should become accretive, both in terms of cash flow and earnings..

Thanks so much for taking the questions..

Thank you. And our next question comes from the line of Geoffrey Meacham from Barclays..

Hi, guys. This is Evan on for Geoff. Thanks for taking the questions, and congrats on the acquisition. Can you help me better understand some of the commercial rational with deflazacort, especially there’s been some controversy in the news recently suggesting that patients previously had been able to import the drug into the United States.

Just help me better understand the commercial rational to actually having it officially available and approved in the United States and how it fits broader within your U.S. strategy..

Sure. Thanks for the question. And so the way we’re thinking about this really is that up until now there’s been really no approved steroid therapy for Duchenne Muscular Dystrophy in the United States.

We know obviously it’s a fatal genetic disorder, and that really Emflaza is the best-in-class steroid and patients should have access to it as a standard of care.

And so, while there has been some availability from that point of view, really prior to this approval there is really less than 10% of the patients that were able to get Emflaza, so really it wasn’t really available to patients. This now allows, with the FDA approval, allows Emflaza to become available to those patients.

And I think really it’s been our mission over the years to bring the best-in-class therapies to patients with rare neglected disease and Duchenne Muscular Dystrophy. I think it allows – I think it’s a very strong strategic fit with our mission to bring new and important therapies to Duchenne Muscular Dystrophy patients. It helps us establish a U.S.

commercial presence for the potential launch of Translarna. It will bring in resources and revenues to continue on our innovative and research and development engine. I think it helps us – we believe we’ve established global leadership in Duchenne Muscular Dystrophy. This helps cement that point of view.

So from a strategic perspective we think it’s a very strong strategic fit and is very – fits right into what our mission is, is to bring drugs of – the best-in-class drugs to patients where there’s limited or no treatment options..

And then also I know Marathon had previously disclosed potential pricing for deflazacort.

Are you sticking with that, revisiting that? What are your thoughts there?.

Yes. I think – as I think Mark had talked on the last, answering to the last call, that we’re – obviously we’re re-examining the current price of Emflaza to ensure that all DMD patients have access to it. I think what we’ve said, we believe that a change needs to be had.

But at this time, since we’ve just signed the transaction, it’s really too premature to speculate on the price level..

Okay, great. Then just one more.

Now that you have a PDUFA date in October, do you expect that FDA will host the panel? What are any other thoughts on the regulatory process there?.

Yes. Thanks for that. We’re pleased that we’ve received the acknowledgement of our filing and that the PDUFA date is October 24. We’re obviously working hard ourselves to prepare ourselves to be ready for the Advisory Committee. As of that, as we’re under review, we’re in the position now really not to comment.

We don’t know when and if that will occur, and so we’re not able to comment on the details of the subsequent things that are going on within the Agency. We’ll notify you, obviously, when an Advisory Committee, if and when it’s scheduled..

Okay, great. Thanks for taking the questions. Really appreciate it..

Thank you..

Thank you. And our next question comes from the line of Joel Beatty from Citi..

Hi. Good morning, and thanks for taking the questions. Could you tell us a little bit more about how deflazacort compares to other glucocorticoids like prednisone for DMD, both on efficacy and any safety findings..

Sure. So we think that now really Emflaza is the first and really only treatment that’s been approved in the United States for all patients five years and older, regardless of the genetic mutation.

I think that the results from both – the results that are currently published as well as results from our double-blind placebo-controlled trials really show that there is an improved efficacy and a more desirable safety profile over other existing steroids. We’re very confident that is the case, and you’ll be hearing more about that.

There’s publications that support that statement as well..

Okay. Then maybe one more question.

Could you share any experience or feedback from how deflazacort has been used in other countries where it’s been available for a longer period of time?.

So I think the – Mark, why don’t you talk a little about that?.

Yes, I think the real need here in the U.S. is that there has been very little access to deflazacort, and it’s only very recently that there has been, since the approval the opportunity to start supplying patients with Emflaza. I think externally in other countries there is a lot more access.

I think patients have already been benefiting from many more years to access to Emflaza. What we want to do is bring the best standard of care to patients in the United States..

Okay. Thank you..

Thank you. And our next question comes from the line of Ritu Baral from Cowen and Company..

Hi, guys. Thanks for taking the question. Can you talk to how many patients are currently on deflazacort in the expanded access program currently? I’m sure you have gone through in very painful detail the deflazacort clinical data.

Who do you see as the right patient? Is it all 9,000 patients? Is it a particular subset who could most benefit from the potential weight gain advantage on the safety side? Also, can you talk a little more about your post-marketing requirements as you understand them right now?.

Yes, sure. So first let me say that as we think that a steroid regiment is part of the standard of care and that all patients should be on steroids, we think it’s clear that it has benefit for patients and they should be on it.

I think that the results show that Emflaza is the best of the steroid compound class, and therefore we think all patients should be on Emflaza as the direct compound of choice. We think all eligible patients five years and greater should be on Emflaza. As to the expanded access program and such, it’s still growing.

I think Mark, let me pass it on to you to comment further..

Yes. I think it’s probably a little premature to give you all the details around that. We will certainly look forward to giving you more precision once we’ve actually completed this deal. But I think it’s been very helpful initiative to enable patients already to start accessing the drug.

And we’re very committed to ensuring that those that are in that program will continue with uninterrupted supply as we move into the launch phase.

But as I say, I think in terms of providing guidance on patient numbers and other factors like that, as Shane said, we have a Q1 earnings call and we’ll look forward to providing more details in the future..

And then on the question that you had on additional studies, there are certain post-marketing commitments that were requested by the FDA that we would be obligated to conduct or discuss the conduction to do those as part of the post-marketing commitment.

They’re mainly associated with both safety in PK, and most importantly there’s a pediatric study that we’ll be doing that will allow for an additional six-month extension. We may also look into longer-term benefit of Emflaza using a registry. That’s a possibility as well. So those are the types of things that we’re thinking about..

Understood. Helpful. Thanks, guys..

Thank you. And that concludes our question-and-answer session for today. I’d like to turn the conference back over to Stuart Peltz for any closing comments..

Well. Thank you. I think in close, our vision is to fundamentally change the lives of patients living with Duchenne Muscular Dystrophy where previously there was no hope, and has given our work great purpose. Today it is deeply meaningful to us to be the Company that is bringing Emflaza to Duchenne Muscular Dystrophy patients and families in the U.S.

The launch of Emflaza in the U.S. will mark a fundamental change in the multidisciplinary treatment of Duchenne Muscular Dystrophy to improve the standard of care and change the course of the disease. We’ll work hard with the community to advance knowledge, raise disease awareness, promote diagnosis and early intervention.

This advancement, combined with our U.S. regulatory developments for Translarna, marks significant progress in our pursuit of bringing best-in-class therapies to DMD patients, their families and the DMD community.

It will enable us to build a greater and accelerated presence in the U.S., one with a clear path towards revenue generation, and importantly it will accelerate the pursuit of the goals we share with the DMD community. It is with special thanks to this community that we’re so excited about the opportunity ahead. Thank you for joining the call..

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