PTC Therapeutics Inc (PTCT) 2016 Q1 法說會逐字稿

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 we'll conduct a question and answer session and instructions will be given at that time. If anyone should require operator assistance, please press * and then zero on your touchtone telephone. As a reminder, this conference call is being recorded. I'll now introduce your host for today's conference, Jane Baj, please go ahead.

Thank you. Welcome to our conference call. Today we will discuss PTC's first quarter 2016 financial results and provide a corporate update. Before we start, let me remind you that today's call will include forward-looking statements based on current expectations.

These include statements about our future expectations regarding clinical development, regulatory and commercialization timelines, and potential outcomes, including statements related to our ability to resolve the matter set forth in the Refuse to File Letter we have received from the FDA in connection with our new drug application for Translarna for the treatment of Duchenne muscular dystrophy, and our ability to obtain and maintain marketing authorizations for Translarna, including in Europe, the potential success of our product and product candidates, adjustable 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 the timing and outcome of interactions PTC has with the FDA with respect to Translarna, including whether we are required to perform additional clinical and non-clinical studies at significant cost, which if successful may enable FDA review of an NDA whether the EMA determines that the risk/benefit balance of Translarna supports continuation of our marketing authorization in the EEA, the outcome of pricing and reimbursement negotiations in those countries in which we are authorized to sell Translarna, and the price at which we are able to sell Translarna. And those risks discussed under the heading Special Note Regarding Forward-looking Statements and Risk Factors in our 2015 Form 10-K and our most recent Form 10-Q, which will be available from the FCC on our website later today. Such statements represent our judgement as of today and PTC undertakes no obligation to publicly update any forward-looking statements except as required by law.

Information regarding our use of GAAP and non-GAAP financial measures, and a reconciliation of GAAP to non-GAAP is available in today's financial results press release.

With that, let me pass the call over to Stuart.

Thank you. Good afternoon. Thank you for joining us on the call. We are happy to report on the progress we've made in the first quarter across our regulatory, commercial and clinical fronts. Important first quarter updates include recent discussions with regulatory authorities in both the United States and Europe, progress on our Translarna commercialization efforts, development in our clinical pipelines, and financial results for the quarter.

Let me start with the work we're doing to make Translarna available to all patients globally who may benefit, including patients in the United States. We have begun a dialogue with the FDA to discuss and clarify the matters described in the recent Refuse to File Letter. We anticipate that this process may require multiple interactions before we come to a conclusion. Given the sensitivity around these discussions, as well as their iterative nature, we will refrain from any public comments, including responding to questions today as they might adversely affect the overall integrity of the process. We intend to provide an update once we have clarified our regulatory strategy in the United States. We believe that this approach is in the best interests of our patients and their families, our investors, and the company.

At the start of the year, we also submitted the ACT DMD Phase 3 results to the EMA in support of the marketing authorization for Translarna for the treatment of nonsense mutation DMD, and separately submitted the request for annual renewal of our conditional marketing authorization, which involves an assessment of Translarna's benefit/risk profile.

As I'm sure you are aware, the process with the CHMP is also in iterative one. As part of this process, the CHMP has sent us questions related to these submissions, These are primarily focused on an assessment of efficacy in light of the ACT DMD data. They also include clarification questions related to observed changes in blood pressure and lipids in Translarna treated patients considering that boys with DMD already have compromised cardiac function. I would note that our product label already requires monitoring for blood pressure and lipids.

CHMP plans to hold a scientific advisory group, or SAG meeting, to review whether the results of the ACT DMD study support the benefit/risk profile of Translarna. The SAG allows our team, as well as outside experts in the DMD field, to provide their view on the clinical results from our Translarna study. We believe that the totality of our data across two large placebo-controlled trials demonstrates that Translarna preserves muscle function in DMD boys, and in our view, supports a positive benefit/risk assessment. We anticipate the CHMP will issue its opinion on these submissions in mid-2016.

Many people have asked us about the potential outcomes of the CHMP process for DMD. We believe that there are three potential scenarios. The first is we receive full approval so that Translarna is then subject to the standard review after five years versus the current annual review. The second is that we receive an extension of our current conditional approval. The Agency can impose additional conditions, such as providing longer term data collected in a post-marketing setting. And third, the EMA could pull the marketing authorization for Translarna. We are obviously devoting significant regulatory efforts to achieving either of the first two positive scenarios as they enable continued access to Translarna outside of the US.

We are actively pursuing regulatory approvals around the world. In Canada, we plan to submit a new drug submission to Health Canada incorporating the results of the company's Phase 3 ACT DMD study later this year with a decision expected in 2017. We are also pursuing Japan, one of the largest pharmaceutical markets in the world, and a market favorable to orphan drugs. In consultation with the Japanese regulators, we have recently initiated a Phase 1 study to assess tolerability and pharmacokinetics of Translarna in Japanese healthy volunteers. This study is the first step towards bringing Translarna to DMD patients in Japan.

Now switching gears to our commercialization efforts for Translarna. Our global launch of Translarna is tracking well, and the feedback we receive from physicians and patients about Translarna remains positive. We have seen good growth across Europe, Latin America and the Middle East, and we have generated significant sequential quarter over quarter revenue growth. In particular, this quarter we generated almost $19 in sales revenue, which is nearly a 50% increase over the fourth quarter of last year. Revenue from reimbursed early access programs in Latin America is growing, and we received a significant order from Brazil in the first quarter. There is significant unmet medical need in Latin America with a large number of nonsense mutation DMD patients, and we are working closely with regional DMD communities to bring Translarna to these patients.

We believe that there may be as many as 7,000 nonsense mutation DMD patients globally in markets where reimbursed therapy is possible. Our experience to date has shown that approximately 40% of the total nonsense mutation DMD patient population are on label. We have found that country specific prevalence and estimates vary depending on male birth rates, incidence of both DMD and nonsense mutation, and local standards of care. While we continue to refine our patient model, we are not in a position to provide regional or country specific breakouts.

In Europe, market access discussions continue on a country by country basis. We know that every day counts for families facing Duchenne muscular dystrophy. We continue to work closely with all stakeholders to bring Translarna to patients around the world as quickly as possible. On April 15th, NICE issued draft guidance recommending Translarna subject to a final Managed Access Agreement with NHS England. NICE recently extended the timeframe for finalization of their guidance. I want to thank the UK DMD community, NICE, and NHS England for all the ongoing support they are showing us as we work together to conclude this process.

I would also like to provide an update for Germany. As we discussed on our last call, we experienced a real disconnect between the German Health Technology Group, GBA, which gave Translarna a favorable rating of three, meaning Translarna demonstrated a quantifiable benefit, and the GKV, the insurance consortium, which was not willing to fund Translarna at a sustainable price. The GKV has recently been communicating they are much less willing to fund orphan drugs at prices considered sustainable by the industry.

As a result of our negotiations with GKV, we officially delisted Translarna from the German pharmacy ordering system as of April 1st. I am pleased to report that the first commercial orders into Germany have already been successfully fulfilled via a pathway that allows patients with high unmet medical need to access treatment when no other options are available. We appreciate all the efforts of the German physicians and payers to expedite this process so patients have uninterrupted access to Translarna.

We continue to work with local authorities across Europe, including in countries where we already have reimbursed early access programs, to finalize reimbursement and pricing agreements. Across the industry, there is significant pressure on pricing for pharmaceuticals, and orphan drug pricing is also drawing attention.

As we have communicated to payers, Translarna represents an innovative new medicine for fatal genetic disorders with no other treatments addressing the underlying cause. PTC has invested over 18 years of research and development for a therapy that targets a very small patient population. We are working hard to negotiate access for Translarna for DMD patients at a price that reflects the value we are delivering to patients and ensures the long-term success of our business. To date we have successfully concluded pricing and reimbursement discussions in six European countries at prices we consider to be sustainable.

In Latin America, there has been significant interest in Translarna from patients and physicians. Currently we have reimbursed early access programs in Columbia, Argentina, Peru and Brazil, which we believe will continue to grow in 2016. By working with investigators and advocacy groups to facilitate genotyping for patients, we have had success identifying nonsense mutation DMD patients in the region.

Let me now turn to our program for Translarna in nonsense mutation cystic fibrosis, which continues to progress. As discussed previously, we completed enrollment of our Phase 3 ACT CF trial in November 2015 with approximately 280 patients. The trial remains on track with topline data expected in 2017.

We submitted a type 2 variation to our marketing authorization in Europe to request approval for Translarna for nonsense mutation cystic fibrosis during the third quarter of 2015. Translarna is the only treatment in development to address the underlying cause of nonsense mutation cystic fibrosis, which is considered the most difficult to treat population. The submission was based on post hoc analysis of the results from our previous Phase 3 study.

Based on recent interactions with regulators, there is substantial risk that the results from our confirmatory ACT CF trial will be required for approval. We filed for early EMA approval because we believe that Translarna has demonstrated favorable benefit/risk, and consistent with our mission we wanted to bring Translarna to patients and their families as rapidly as possible. Furthermore, as with DMD, it affords us an opportunity to educate and discuss with the CHMP the benefits of Translarna for patients afflicted by nonsense mutation cystic fibrosis. We anticipate that the CHMP will issue its opinion in mid-2016.

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 therapies for spinal muscular atrophy, or SMA. SMA is a rare genetic neuromuscular disorder that is caused by a missing or defective SMN1 gene, which results in reduced levels of SMN protein. The SMN protein is critical to the health and survival of the nerve cells in the spinal cord. 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 SMN2 splicing modifiers as a way to address the disease. An oral therapy which has exposure to both muscle and nerve tissue has the potential to provide considerable advantage.

As we discussed on our last call, concurrent with the development of our lead compound, RG7800, a robust research program regarding SMN2 splicing modifiers continue to advance and RG7916 was also identified as a development candidate. We recently completed a Phase 1 study in healthy volunteers with this compound. Preliminary results indicate that RG7916 increased the production of full length SMN2 mRNA and was well tolerated.

Because healthy individuals have both the SMN1 and SMN2 gene, we are in the unique position that changes in splicing can be measured in the blood of healthy volunteers. This allows us to rapidly monitor both the exposure and the activity of RG7916 in an early study. Results of this Phase 1 study support the continued development of RG7916. Together with our collaborators, we are planning to begin a clinical study in SMA patients later this year. Once we have data on SMA patients for RG7916, we will be able to compare the results for both compounds to determine the best one to move forward in subsequent clinical development.

To wrap up, while we are working to address the RTF Letter, we have delivered strong commercial results and are actively preparing for upcoming regulatory interactions, and have continued to advance our clinical pipeline. With that, let me turn it over to Shane to talk about our financial results for the first quarter. Shane?

Thanks, Stuart. We're happy to report a strong start to the first quarter of 2016 with Translarna net product sales of $18.9 million, which represents a 49% quarter over quarter increase versus the $12.7 million we reported in the fourth quarter of 2015.

Contributing to this growth, as Stu mentioned, was a large order from Brazil. Brazil represents a significant market opportunity for us with a population nearly two-thirds the size of the United States. Our team has made significant progress working with local key opinion leaders to help identify DMD boys with the nonsense mutation. In Brazil, orders for certain pharmaceuticals are fulfilled in larger bulk orders on a semi-annual basis. As a result, the timing of these orders is expected to have an impact on our quarterly reported revenues, and may result in our second quarter net sales being lower than those for the first quarter that we're reporting today.

First quarter revenues were negatively impacted by a lower net sales price in Germany as a result of the mandatory discount imposed by the GKV prior to delisting. This lower price in Germany is expected to tail into our second quarter revenues during which period patients were transitioned to imported therapy.

Overall, we continue to have confidence in our ability to achieve our prior revenue guidance of $65 million to $85 million in Translarna net product sales for 2016. This guidance assumes current exchange rates and the continued rollout on a country by country basis for Translarna outside of the US. As we progress throughout this year, we anticipate updating this guidance when and if appropriate.

As a result of the Refuse to File Letter we received from the US FDA, we announced a workforce reduction as part of a plan to optimally manage expenses. This resulted in an approximate 18% reduction in the size of our workforce, primarily affecting our employees in the US. This will result in a one-time charge of approximately $2.6 million of which $1.9 million was recognized in the first quarter. We remain focused on executing our clinical development plan and continuing to invest in our commercial growth outside of the US where we are making progress expanding access to Translarna for nonsense mutation DMD patients.

Non-GAAP R&D expenses in the first quarter were $27.1 million, an increase of approximately $3.8 million compared to the same period in 2015. Non-GAAP R&D expenses decreased sequentially versus the fourth quarter of 2015 by $4.3 million from $31.4 million. Non-GAAP SG&A expenses were $21.3 million for the first quarter of 2016 compared to $12.5 million for the same period in 2015. Non-GAAP SG&A expenses decreased sequentially versus the fourth quarter of 2015 by $0.4 million from $21.7 million.

We reported a net loss of approximately $41.2 million for the first quarter of 2016 compared to approximately $37.9 million for the same period of 2015. Net loss decreased sequentially versus the fourth quarter of 2015 by $9.7 million from $50.9 million. Our net cash burn for the first quarter was approximately $40 million, which is typically larger due to the timing of payout of annual cash bonuses to employees which otherwise accrue throughout the year. We ended the quarter with $299 million in cash and marketable securities on our balance sheet, which puts us in a healthy capital position.

In addition to reaffirming our revenue guidance for the year, today we are also providing guidance on operating expenses. We currently anticipate non-GAAP operating expenses to be between $185 million and $195 million, excluding approximately $40 million in non-cash stock-based compensation expense, for total operating expenses of approximately $225 million to $235 million for 2016. As a result, PTC expects to finish 2016 with approximately $200 million in cash and cash equivalents.

Joining for the Q&A is my colleague Mark Rothera, our Chief Commercial Officer. Operator, can you now open the call for questions?

Thank you. (Operator instructions) Our first question comes from Christopher Marai of Oppenheimer. Your line is open.

Good afternoon, thanks for taking the questions, and congrats on the quarter. I was just wondering if you could further elaborate on sales in Germany. I was wondering, you know are those sales prior to the importation pathway in newly identified patients or are those old patients who previously received drug and now have been converted? And then could you perhaps comment, I guess this is for Mark, on patient finding efforts in Germany now that you're using that importation pathway? And I have a follow up, thanks.

So hi, Chris, this is Mark. Thanks for the question. So I think the first priority for patients in Germany is that those that are on existing treatment get successfully transferred onto the foreign importation process. So we're very pleased to see that that is already happening and it's very fast. If you think about the fact that we delisted on the 1st of April, already numerous patients have already shifted onto foreign importation. So that's the first priority, and this will happen, you know we expect in the coming month or so. Beyond that, as new patients get identified, they will then be able to also access Translarna through this process.

But just to be clear, Chris -- its' Shane speaking. For the first quarter sales, none of those sales in the first quarter we're reporting today reflect the [reimportation] into Germany.

Right, they are all discounted prior to delisting.

Got it. And then how far along are you with respect to converting those Q1 delisted patients into importation pathway patients? Is there a percentage you could give roughly?

Thank you very much for asking, but I think you'll appreciate that we really don't go down to patient type of level detail. But, you know, you may recall that we were suggesting it could take a couple of months to undertake this process, and we're really pleased that in less than a month we've already seen you know numerous patients already switched onto this. And I guess more importantly, you've seen it today, we've reiterated our revenue guidance at $65 million to $85 million this year.

Got it. And then just with respect to the ongoing NICE discussions, I mean it sounds like they were delayed slightly. Could you just confirm what we've learned, which is that the addition time required with NICE is not about a price debate but it's rather about other aspects, such as like five-year follow up and other higher requirements that they had of you? Thanks.

So, you will recall that we got a positive recommendation for funding from NICE back in April. And you're right, they've recently just extended the periods for public consultation, followed about eight weeks before we're in -- by which they've agreed to publish at that point the final guidance. So it's in this period that NHS England and PTC are just working to finalize the elements of the Managed Access Agreement. And this is a high priority for both parties. You know, we've had multiple productive discussions and we have a draft agreement, and we're just looking forward to finalizing that as soon as possible. There are many different elements in a Managed Access Agreement.

Okay. And just to be clear, it's not regarding a price debate, correct?

This is about finalizing the draft agreement. And as I say, there are multiple different points that were open that needed just to be closed. It's a general point that, you know, this whole agreement has many different chapters, it just needs to be finalized, the Is dotted and the Ts crossed.

Understood. Thanks so much.

Thank you. Our next question comes from Geoff Meacham of Barclays. Your line is open.

Hey, guys. This is Evan Siegerman on for Geoff. Thanks for taking my questions. So the first one I have is about the current Phase 3 in CF. You know, I know you had mentioned something about this before, but how much of a role does this have in EMA's decision regarding conditional approval? And from the commentary, it appears that you will most likely need this, but could you give us some understanding of why EMA would potentially conditionally approve without this data?

So let me just remind you that we -- you know obviously that CF is actually a variation on the conditional approval. So it's actually a full approval on the -- it's a variation -- the submission is really based on the full approval from our previous Phase 3 trial. So it's really just based on recent interactions with the regulators that -- recent interactions with the regulators that really basically that we submitted the basis of that and that we're testing whether it's sufficient enough to demonstrate benefit for patients. So I think what we're doing is that they're linked, and so in a sense the CF variation really is dependent on the market authorization for DMD. So that's really the connection between the two of them.

Okay. And, you know, switching to DMD, could you remind us what the criteria would be for EMA to pull the drug? And are there any precedents for where -- you know to pull a drug from conditional approval where there's no safety signal?

Yes, so let me just remind you, as I said in the prepared remarks, there's three -- there are three ways in which we can get this drug, which is full approval, conditional approval, or pull the drug. And you know obviously we're working hard to ensure that we get one of the two there. There are a small number of cases of conditional approval and that each one is different. So there's not a lot of data points to draw any conclusions from them. There's not very many that have been pulled. And our focus is really with the CHMP to make sure we discuss with them the efficacy of Translarna and the belief that we have that the results support the benefit/risk for the patient.

Okay. And then one more follow up regarding your negotiation with UK NICE and NHS. Is this delay at all related to the, you know the decision from EMA regarding conditional approval becoming full approval or being pulled from the market? Are they waiting on that to make a final decision?

No, I think what -- they are two independent agencies working on -- you know have different roles and functions, so they're independent as a consequence of that.

Okay, so there's no connection whatsoever between the two of them in your view?

No, one's about pricing and reimbursement and one's about approval.

Okay.

And maybe just to build on that. You know, bear in mind that we're now commercialized in many, many countries where, you know again, these have been completely dissociated processes. So, you know we've already shown you that we have the drug reimbursed and available in numerous countries.

Okay, great. Thank you so much for taking my questions.

Thank you.

Our next question comes from Anumpan Rama of JPMorgan. Your line is open.

Hi, this is [Uko] on the call for Anumpan Rama this evening. Thanks for taking my questions. Would you provide some progress on commercial uptake of Translarna globally, such as number of patient adds and countries that now have access to Translarna?

So just to remind you, we have established a commercial, global commercial footprint that covers 46 countries. And, you know, we reported previously, you know 23, 24 countries where we're already reimbursed and patients are gaining access. So the drivers of growth remain that not only do we continue to expand the number of countries where access is possible, but in countries where reimbursement is already available, we continue to have patients have access and expand the number of patients. And all of that really adds up to say that we're reiterating our guidance of $65 million to $85 million for this year.

Okay, great. Thank you.

Thank you. Our next question comes from Heather Behanna of Wedbush Securities. Your line is open.

Great, thanks, and congrats on the quarter. Just a couple of questions. One is just when will we start seeing cost of goods for Translarna?

Hi, Heather, it's Shane.

Hey.

So once we -- we've had discussions with our auditors about capitalizing inventory, and I think once we confirm approval with the EMA and ultimately a path forward in the US, that we would begin -- that we would have certainty with respect to our operating expenses going to produce Translarna product for commercial sale. At that point we would begin capitalizing those expenses as inventory and then expensing in accordance with sales as cost of goods. But up until now, we have been expensing those production costs in the operating expenses.

Got it. That's helpful. And then just switching gears for a second. On the SMA program, if you could just give us any color on how you've been monitoring patients for the potential for any ocular toxicities for the new compound and sort of how that will weigh into your decision process once you get data in SMA patients?

Yes, so let me just remind you on the, you know we've chosen RG7916 is moving forward in healthy --has moved forward in healthy volunteers, that was picked I should remind you, as we were going on in having a robust discovery program while we still had RG7800, and that was then moved forward because it was more [potent] and efficacious.

And it showed that to be the case in a Phase 1 trial in that it had a -- we believe that RG7916 has the potential to have a wider safety window as compared to 7800. And as I said in the prepared remarks, we're going to move forward with it in SMA patients, and once we have the SMN protein data and SMA RNA level in patients for RG7916 we'll be able to compare the activity in both compounds and prepare the best path forward.

Along with that, we'll be looking at -- obviously we'll be watching for -- monitoring for any eye issues and that will then also be having -- undertaking longer term toxicity studies to explore the safety and tolerability of the compound at higher exposures and at longer duration of treatment than has been investigated both in the healthy volunteers and subsequent SMA patients. And so once that's all through, we look forward to sharing those results with you once they're final.

Great. So just a quick follow up. Were you able in the healthy volunteers to see that what you saw pre-clinically as far as (inaudible) is being recapitulated that the results are you know, -- that the splicing levels were higher than what you saw for 7800?

Yes, so I think what we're -- you know what we're able to disclose really right now is that we completed the Phase 1, single dose Phase 1 study, and we're able to show that the SMN RNA was showing very nice splicing. And as we said, it was efficacious, so we're excited about that. And obviously we're moving that forward and then we'll be able to compare 7916 to 7800 once that data is complete.

Okay, thanks.

Thank you. Our next question comes from Alethia Young of Credit Suisse. Your line is open.

Hey, guys, this is Ellie on for Alethia. Thanks for taking my questions. My first question is just if you can just characterize a little bit how large the order was from Brazil. And then, you know, what the gross rate of sales would have been without this order from Brazil.

Hi, thanks for the question, Ellie. As you may expect, we don't break out any specific country revenue numbers. However, these large orders from Brazil are expected to have an impact on our quarterly reported revenues. And as a result, I think as we disclosed during the quarter, it may result in our second quarter net sales being lower than those that we're reporting today.

Okay, that's helpful. And then just in terms of your guidance, I was just curious kind of you know what pricing assumption you have baked in there. If you could just comment a little bit on that.

Yes, I think as we talked about in prior calls now, (inaudible) second year of launch as we mature with this commercial expansion we have moved away -- or we have moved towards giving revenue guidance which integrates many factors, including price, exchange rates, patients numbers, compliance and average weights of patients. So really there's a number of factors that go into that overall guidance that we've given.

Great. Thanks.

Thank you. Our next question comes from Gena Wang of Jefferies. Your line is open.

Thank you for taking my questions. So I know you mentioned that you won't give us specific numbers, patient numbers from each country, but just wondering, could you provide total number of patients currently on drug? I know last time you provided it was 206, and I wonder if this number has increased since last time.

So thank you for the question. You know, we did mention actually at the previous call that we're moving away from providing specific patient numbers, but to move towards revenue guidance for the first time. So that's where we are today, but clearly the growth that you have seen quarter on quarter is driven primarily by patient growth. You know, that's the main driver. And I mention that's partly due to new geography and partly to increased penetration of countries where we're already present. And Brazil has been a tremendous collaboration between PTC and the Ministry of Health where there's a common goal really, to look after the patients with a very high unmet need of this nature.

Thank you. Maybe just one follow-up question regarding cystic fibrosis label extension. Could you remind us at which stage in the process are you at right now?

So yes, so with the cystic fibrosis, so as you may recall, we have an ongoing cystic fibrosis trial that the results will come out in early 2017. And we also filed a variation and we're in the middle of that now. We anticipate that that will be completed mid-year this year, mid-year of this year, and so we're in the midst of that process now.

Sorry, maybe I did not make it clear. Just wondering, you know, have you submit, you know which stage are you at? Have you submit the response to the EMA or which stage are you at? I just wanted to have a little bit more clarity.

Yes, so you know so the process is one in where -- the process is one in where you have questions that they're asking, respond to those questions. We've done that. We're anticipating that there might be another round of questions. So we're in the middle of those process. We're waiting for a response on that. So that's where we are right now. We anticipate that the process then would conclude mid-year sometime.

Okay. Thank you.

Thank you. I'm not showing any further questions in queue at this time. I'd like to turn the call back over to management for any further remarks.

Yes. Well thank you. Thank you for joining the call today. All of us in the rare disease community are working hard to develop treatments for patients who are without options today. when you're trying to get a new drug to the market in an uncharted therapeutic area, it's not surprising to encounter development and regulatory challenges. The way you succeed is to persevere. We continue to make progress, and we remain firmly committed to bring Translarna to patients globally. Thank you for joining us today.

Ladies and gentlemen, thank you for participating in today's conference. This concludes today's program. You may now disconnect. Everyone have a wonderful day.