PTC Therapeutics, Inc. (PTCT) Q4 2015 Earnings Report, Transcript and Summary

Good day, ladies and gentlemen, and welcome to the PTC Therapeutics Fourth Quarter and Year-end 2015 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 follow at that time. [Operator Instructions] As a remainder, this conference call may be recorded. I would now like to turn the conference over to Emily Hill. Please go ahead.

Thank you. Welcome to our conference call. Today, we will discuss PTC's fourth quarter and full year 2015 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 developments, regulatory and commercialization timelines and potential outcomes including statements related to our ability to work with the FDA to resolve matters set forth in the Refuse to File letter we received in connection with our New Drug Application for the Translarna for the treatment of Duchenne muscular dystrophy and our ability to obtain and maintain marketing authorization for Translarna, the potential success of our products and product candidates, addressable patient populations and financial projections. Actual results may differ materially from those indicated by these statements as a result of a variety of risks and uncertainties including the timing and outcome of future interactions PTC has with the FDA with respect to Translarna, including whether we are required to perform additional clinical and non-clinical trials at significant costs which if successful may enable FDA review of an NDA submission; whether the EMA or other regulators agree with our interpretation of the results of ACT DMD or our other clinical trials; the outcome of pricing and reimbursement negotiations in those countries in which w are authorized to sell Translarna; and those risks discussed under heading 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 2015 Form 10-K which will be available from the SEC on our website later today. Such statements represent our judgment as of today and PTC undertakes no obligation to publicly update forward-looking statements except as required by law. Information regarding our use of GAAP and non-GAAP financial measures and the 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.

Good afternoon. Thank you for being on the call. I'm going to start by addressing our recent Refuse to File from the FDA. Receiving this letter was shocking and deeply disappointing. As you know, we received the letter from the FDA on the evening of February 22. We have spent the last few days analyzing the letter’s content and assessing our best path forward with both our internal regulatory team as well as outside experts. The letter stated that our NDA was not sufficiently complete to permit a substantive review. There were two areas that served as the basis for the RFT. First, in the view of the FDA, both the Phase 2b and Phase 3 ACT DMD trials were negative and do not provide substantial evidence of effectiveness. The FDA also characterized certain of our adjustments to the ACT DMD study as post hoc and therefore not supportive of effectiveness. Second, the FDA noted that the NDA does not contain adequate information regarding the abuse potential of Translarna. We are surprised by this letter given the FDA’s recent guidelines on flexibility for DMD drug development and the willingness to review NDAs of other DMD therapies that missed the primary endpoints. With regard to evidence of effectiveness, let me remind you that we have now completed two large double blind, placebo controlled trials in over more 400 nonsense mutation DMD patients. In a rare devastating progressive disorder, we have demonstrated clinically meaningful benefit in one year studies across both primary and secondary endpoints. We believe the totality of the data is supportive of the effectiveness and demonstrates a strong safety profile. Indeed, it was on the basis of this positive benefit risk profile that the European Medicine Agency approved Translarna in August 2014. With respect to evaluating the potential for abuse, this is an FDA requirement for new molecules that cross the blood-brain barrier. We believe based on the preclinical and clinical work we have done, we should be able to address the FDA’s concern that demonstrates that Translarna has not shown central nervous system effects associated with abuse potential. PTC is engaging in a dialog with the FDA to discuss and clarify the matter set forth in the letter and to determine the best path forward. Translarna deserves a full and fair hearing in front of the FDA including an advisory committee with representatives of the DMD community. We believe in the strength of our data and what it represents to patients. Our goal remains to obtain regulatory approval for Translarna in DMD in the United States. While we fully appreciate the gravity of the FDA letter, it should not overshadow a number of key milestones that we successfully achieved in 2015. We continue to build a leading fully integrated biotech company that discovers, develops and commercializes novel therapies for rare and neglected diseases. We've been working closely with all stakeholders to bring Translarna to patients around the world as quickly as possible. To that end, since the beginning of this year, we're pleased that patients in Argentina, Czech Republic, Singapore, Hungary and Portugal now have reimbursed access to Translarna. In total, we now have commercial sales in 23 countries and developed a global footprint in over 46 countries. We're supplying Translarna commercially to an increasing number of patients across Europe, Latin America, Asia Pacific and other regions. Of the estimated 5,000 nonsense mutation DMD ex-US patients, we have already identified over 1,000. We continue to support genotyping and patient identification efforts worldwide. Our goal for 2016 is to expand access to Translarna to at least 35 countries. As we told you at this time last year, for 2016, we are transitioning to providing Translarna revenue guidance in order for the investor community to track our progress. As Shane will discuss in more detail, our Translarna ex-US DMD revenue guidance for 2016 will be $65 million to $85 million. As we continue to expand Translarna globally, we are working through market access on a country-by-country basis. As everyone is probably aware, payers in Europe have become increasingly sensitive to pricing and reimbursement in healthcare in recent years. We have been working hard with the local reimbursement authorities to ensure sustainable access to Translarna. We recently met with NHS England where we made great progress outlining a managed access agreement. Overall, we feel we have been in very constructive dialogs with NHS England and NICE. These discussions which included other stakeholders culminated in a draft managed access agreement. Subject to NICE approval, this would allow for sustainable reimbursed patient access in England. In Germany, we’ve had multiple discussions with the reimbursement body over the course of the last several months to come to agreement on prices and reimbursement. Recently, these discussions transitioned into an arbitration process which did not lead to an acceptable agreement. As a result, if necessary, PTC could choose to delist Translarna from the German pharmacy ordering system. Under these circumstances, patients and healthcare professionals have the opportunity to access Translarna through a reimbursed importation pathway possible under German law, thus minimizing any access issues for German patients while maintaining a sustainable price. In addition to our strong commercial progress to-date, we are actively pursuing regulatory approvals around the world. We believe the results across two large placebo-controlled trials in DMD demonstrate Translarna's activity and combined with its favorable safety profile support a strong regulatory package demonstrating Translarna's efficacy. Of important note, although we did not achieve statistical significance, Translarna is the only DMD therapy that have shown clinically meaningful benefit across both its primary as well as its secondary endpoints in one year clinical studies. This has been an important point of emphasis with key opinion leaders and regulators. Over the course of 2015, Translarna was reviewed and approved by regulators in South Korea and Israel. We're also currently under regulatory review in Canada where we received an expedited review. In Europe, we have submitted the ACT DMD results and have requested on that basis that regulators remove the condition to the existing marketing authorization. As you are aware, Translarna has the potential to be an important therapy for approximately 10% to 15% of patients suffering from many genetic disorders who have nonsense mutation. In 2015, we announced our 10/20 strategy to investigate at least 10 indications beyond DMD and CF with Translarna by 2020. We currently have four of these indications under development including MPS1, aniridia and two genetically defined epilepsy CDKL5 in Dravet. Let me now turn to our program for Translarna in nonsense mutation cystic fibrosis. We experienced overwhelmingly strong demand from physicians and patients who wanted to participate in our Phase 3 ACT CF trial. The trial completed enrollment in November 2015 with approximately 280 patients. The 48-week trial will complete by the end of this year with top line data expected in early 2017. In September of 2015, we submitted an application to nonsense mutation cystic fibrosis through the existing label for Translarna in Europe. This application was based on results from our previous Phase 3 trial. We believe that the results are compelling and that it’s important to make Translarna available to nonsense mutation cystic fibrosis patients. We are working through the process for a potential CF approval in Europe. As expected, as part of the process, we received questions from the European regulators and we're currently in the process of responding to them. We anticipate a decision on potential approval for CF in Europe in mid 2016 and our team is preparing for a potential launch for CF in that region. Let me now shift to a program in our splicing platform technology. Our first program using this technology addresses the potential new therapeutic for spinal muscular atrophy or SMA. SMA is a genetic neuromuscular disease caused by a missing or defective SMN1 gene which results in reduced levels of SMN protein. PTC, together with Roche and the SMA Foundation, have a robust program around oral, small molecule SMN through splicing modifiers as a way to address the disease. A SMA treatment with exposure to muscle and nerve tissues may have considerable advantages for patients. While the clinical trials of RG7800 are on hold, an additional compound RG7916 was also chosen as a development candidate. We recently began a Phase 1 study of RG7916 in healthy volunteers. As we described previously, because healthy individuals have both the SMN1 and SMN2 genes, there is an advantage in that changes in splicing could be measured in the blood of healthy volunteers. Results from this study will be used to evaluate which is the best compound to move forward in subsequent clinical development. Let me now turn to our cancer stem cell program targeting BMI1. BMI1 is up regulated in the tumor stem cell population and is an important component of the complex that allows cancer cells to become resistant to chemotherapy. As you may recall, our compound PTC596 reduced the level of the BMI protein in preclinical oncology model. I'm very proud to have a fourth internally discovered program as PTC entering clinical development. PTC596 began a Phase 1 study in the second quarter of 2015 in advanced cancer patients with solid tumor. We are excited to share data from this program later this year. As you can see, we are poised to accomplish many commercial regulatory and clinical milestones in 2016. With that let me turn it over to Shane to talk about our financial results for the quarter and 2015. Shane?

Thanks, Stu. We are happy to report a landmark first year launch outside of US with Translarna net product sales of $33.7 million. In the fourth quarter, we recorded $12.7 million in net product sales representing approximately 30% sequential growth over the $9.8 million we recorded in the third quarter of 2015 and versus $0.6 million in the prior year period. Non-GAAP R&D expenses in the fourth quarter were $31.4 million compared to $23.7 million for the same period in 2014. R&D expenses increased year-over-year as a result of additional costs associated with our expansion of our clinical development activities, including late stage studies in both DMD and CF. Non-GAAP SG&A expenses were $21.7 million for the fourth quarter of 2015 compared to $14.5 million for the same period in 2014. SG&A expenses have increased year-over-year as a result of the commercial launch of Translarna. PTC has grown its capabilities, both domestically as well as internationally to support our current and planned global launch activities. We reported a net loss of approximately $51 million for the fourth quarter of 2015, compared to approximately $27 million for the same period of 2014. Our net cash burn for the fourth quarter was approximately $33 million and we finished 2015 with nearly $339 million in cash and marketable securities on our balance sheet. We currently have 34.3 million shares issued and outstanding, which includes 0.3 million of unvested restricted stock grants. In terms of guidance, and as Stu mentioned earlier, we are now in a position to provide revenue guidance for 2016. Based on our internal assumptions, with respect to the continued commercial expansion of Translarna for DMD outside of the US, we currently anticipate net sales to be between $65 million and $85 million 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. With respect to operating expenses for the year, given the Refuse to File letter we recently received, we are re-evaluating our 2016 operating budget and are therefore not in a position to provide operating expense guidance at this point. We anticipate providing expense guidance on our future call. Joining the Q&A are my colleagues Bob Spiegel, CMO, Mark Rothera ,our CCO and Mark Boulding, our Executive Vice President and CLO, who oversees our regulatory function.

[Operator Instructions] Our first question comes from the line of Geoff Meacham with Barclays. Your line is now open.

Hi, guys. This is Carter on for Jeff. Thanks for taking the questions. I guess first on the RTF and the FDA’s characterization of some of the analysis of this post hoc, I am trying to reconcile that with past comments that the stat plan [ph] was submitted to the FDA earlier in 2015. So I guess, specifically between that time stat plan went in and the sNDA or the NDA went in, was there any interaction with the FDA on the stat plan over this period and were there any changes the stat plan from your side over that period. And then second question, just real quickly, on the 2016 guidance, what are the assume surrounding pricing in Germany? Thank you.

Great, thanks Carter for the questions. So maybe back to first review, some of the – just some of the history, so that let me just review a little bit on, we received the letter from the FDA on Monday night, and obviously as I said in the call, that we were very surprised to have received this letter given the FDA’s recent guidelines on flexibility for DMD drug development as well as their willingness to review NDAs of other DMD therapies that have missed primary endpoints. And let me remind you that we completed two large double-blind, placebo-controlled trials in over 400 nmDMD, Duchenne muscular dystrophy patients in the rare demonstrated progressive disorder, and we believe we have demonstrated clinically meaningful benefit in one year studies across, both primary and secondary endpoint. And that obviously we believe that the totality of the data is supported by the effectiveness and demonstrates a very strong safety profile. And we’re really on the basis of the benefit risk profile that the European Medicines Agency approved Translarna in August 2014. Overall on the sub-group analysis, the FDA characterized, first of all that the FDA characterized our NDA as not supportive of effectiveness. This would include the sub-group analysis. So I think in terms of the history, it’s important to remember that we included the sub-group’s most statistical analysis plan, which we submitted in the spring of 2015. At that time, the FDA commented on the statistical analysis plan that had no comments on our subgroup. We submitted the final statistical analysis plan to the FDA before unwinding activity study. However, in the RTF letter, the FDA characterized that PTC proposed a post hoc adjustment of ACT DMD that eliminates data from a majority of enrolled patients. So we need further discussion with the FDA to understand the current perspective on our sub-group analysis. We believe that FDA’s perspective in the RTF letter may be that although we pre-specified the sub-groups, relying on the sub-group as the main analysis is considered as a post hoc adjustment and we will be talking to them further on this point. In terms of the German pricing and reimbursement, let me ask Mark Rothera, maybe to comment a bit on that.

Sure, Stu. Well, as you’re aware, in the past we reported patient numbers and we gave pricing guidance. Now, as Shane mentioned, when moving to revenue guidance, which integrates both pricing expectations, patient numbers, average waits of patients and exchange rate. And so the question you’re asking is integrated certainly into our thinking on the revenue forecast. Specifically, as you all are aware, the GKV took a very stuff stance on pricing in Germany and our expectation is that unless there is a change on that we will or the patients will be able to import Translarna from an alternative country in a pathway that’s available by law, that’s available both to existing and new patients and allow them to receive ongoing treatment at a sustainable price level.

Thank you.

Thank you. Our next question comes from the line of Anupam Rama with JP Morgan. Your line is now open.

Hi guys, this is Eric in for Anupam, thanks for taking the question. Just a couple of questions on the EU. To what extent are your ongoing PNR discussions contingent on a CHMP opinion on ACT DMD. And just with respect to Translarna availability in Germany going forward, to what extent the GKV’s decision affects your ability to market to certain physicians? And how does reimbursement outside the GKV system represent a hurdle to patients and physicians? Thanks.

Thanks for that, Eric. I will just start on and then I will pass it on to Mark. Clearly, we have a lot of pricing and reimbursement discussions going on that are based on our Phase 2b data and we’re in the midst of that, and I will have Mark talk about that as well as in terms of the German -- as Mark alluded to and I think will talk a bit about, part of this is a clear path to allow patients to maintain access of the drug at what we think is a more stable and sustainable price and he can talk about all that. So maybe I will pass then on to you Mark.

As you’re aware we are already selling Translarna in 23 countries and there are six countries where we have already been through the pricing and reimbursement negotiations and other commercial price. So there isn’t a contingency around the CHMP decision that’s expected here. With regard to Germany, there is a process I mentioned that enabled patients to have access to essential medicines that are not available on the German market. The drug is approved in Germany, it can be promoted in Germany, it could be prescribed in Germany, but by choosing to delist, what we are avoiding is a list price at a level that we believe is unsustainable and we are enabling patients to request authorization of the drug through importation. And just to remind you, the GBA, which is the health technology assessment study in Germany already opined on the value of Translarna and gave the drug a rating of 3 on a scale of 1 to 6, where 1 is the highest and 6 is the lowest. About 50% of orphan drugs that go into the Germany HTA process get a 4 or a non-quantifiable benefit, whereas Translarna was given a 3 or a quantifiable benefit. So when it comes to patients requesting access to the drug, not only is it a drug that the insurers will see has been given a 3 from a medical value perspective, but it’s the only approved drug for what is a very life-threatening condition. I guess, the final thing to say around this is that the GKV appeared to have a taken a tough stance. It seems contextual. I don’t know if you saw that press release of the 21st of January, but they are really pushing hard on both on pricing and the GBA value assessment at this time, and I think we are caught up in that situation.

Thank you. Our next question comes from the line of Ritu Baral with Cowen. Your line is open.

I have three questions; one is very, very quick, just as a follow-up. In the guidance that you have put out today, assuming the same $300,000 Pan-European average drop price that you have stated previously?

So as I mentioned earlier, the guidance on revenue do not specifically give a view on a particular price, it’s an integrated view on pricing in different jurisdictions, patient numbers await exchange rates. So we're not going into specific pricing on a country by country basis. But to be clear, we stand by on confidence in the $65 million to $85 million revenue range that we've quoted in our revenue guidance ex-US.

Is there a chance that full-year 2015 revenues will need to be restated because of the refund you might have due to Germany because of the arbitration process?

Hi, Ritu its Shane. It’s a great question. Under the German system, when you’re approved to launch as we did in December of 2014, your first 12 months are pricing of where the company sets and thereafter which would be for us as of December of 2015 you're obligated to accept the price that you're settled on or the one that we would have just received through German arbitration. So for the month of December, the one month in 2015, any sales for that month will be at the lower price and we've made that adjustment in our gross to net what we reported today.

And do you have a specific timeline for speaking to FDA on the RTF set, have you had any phone conversations with them or do you have a meeting scheduled yet?

Obviously our -- the next step for us are really is obviously our goal is to ultimately get it approved so that the US patients can benefit from it. At this point, we have requested a call with the FDA to clarify and understand better the points they made in their RTF letter. And then obviously there is multiple options and the path that we choose will be determined really through subsequent discussions with the FDA and what we’ll do is update you when we have more clarity.

Can you just take one more in there? Did the abuse potential come up in your summer 2015 letter or I’m sorry meeting before the ACT DMD data came out?

Did what, I’m sorry say that again?

The abuse potential qualifier in the RTF?

Come up previously, no. First of all it's really our belief that ataluren doesn't have any abuse potential did not come up previously and that’s based both on the structure of ataluren, the lack of new signal from animal studies as well as our clinical studies that we’ve conducted in different indications. In the context of their preliminary review, the FDA may not have appreciated all of the information we provided on this topic in our NDA. So our plan is to provide a single document that gathers together the relevant information from the NDA and we do believe that this would be sufficient to satisfy the FDA.

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

I've got a couple, one is, just if you could give us a little bit more color on what's going on with NICE in the UK. And then the second question is, if you could talk a little bit about if there is still any lag in revenue or what exactly is the gross to net adjustment or what factors planned to that?

So, maybe I’ll just give a little bit of color and then I can talk. In general, we've been working hard obviously with the local reimbursement authorities to ensure sustainable access to Translarna and that overall we feel we've had very constructive dialog with both NHS England and NICE. So, I think that's where we’re at, so I think that we’re waiting, we’ve had these NICE discussions we've elaborated a managed access program with them that we’re waiting for NICE to approve. And so that's where we are right now with that. In terms of the revenue may be Shane do you want to?

I mean, for us we recognize obviously our net product sales revenue in minor adjustments from a gross to net Heather like for instance in Germany there always is a mandatory 7% discount, which we’d always been reporting in our gross to net. And in addition, as you may know in certain countries or territories we have chosen to work with certain third-party distributors and those distributor margins are factored into our gross to net pricing, so any sales that we report are the net sales for PTC.

And then just a quick follow-up on NICE, I know that there was a meeting on the 17th of February discussing ataluren. So I was just curious if that was the full dataset including Phase 3 and if we should probably expect something published from that meeting at some time in the next few months?

I think there was some discussion that we had that which was following up NHS England meeting. And so I think it was a very good and constructive dialog that we had at the NICE meeting and so we will then wait for their final decision that will come out of that.

Thank you. Our next question comes from the line of Christopher Marai with Oppenheimer. Your line is open.

First I’d like to start with maybe the pricing discussions in Germany; obviously we've heard some numbers thrown around. I'm wondering if you could further elaborate on a discount that was requested in the arbitration. And then two, I know you don’t want to list the drug in Germany at that price, and Germany is a reference point for many other countries. Is it possible that other countries could look at those arbitration documents, are they going to be public and then push for a price in order to what the German authorities have requested? And I've got a follow-up.

So, maybe Mark wanted to talk a little bit about the pricing discussions that [indiscernible].

So, hi Chris. You're quite right and just to be very open; I mean the request was for a 57% discount on top of the 7% mandatory rebate. So clearly that would take us into an unsustainable and unrealistic price level for Translarna. And as we mentioned earlier, the plan B that we’d already understood was available to patients is to have the drug imported from abroad at a more sustainable price level. Indeed even in the arbitration process, the GKV admitted that that was available to these patients and it’s a tried and tested route for medications that are urgently required for patients.

So and just to add on that as Mark, is that each country does have its own process and just a reminder that to date, we've established an agreed commercial price at six countries now. And that really we think importantly by not establishing this unacceptable reference price, we are seeking to protect the value of Translarna as a consequence of that, so that's why we did this or will be doing this.

And then, I guess the next question will be focusing on EMA and the conditional approval removal, I was wondering if you could elaborate perhaps on what exactly was required for that and any discussions they had with you. As part of the conditional approval needed to run the trial in terms of removing it, was there any clarity that you guys have received that the trail needs to be successful or support the benefit et cetera, could you perhaps elaborate on what was actually required there I think?

Really, actually when you look at actually what they asked for really the condition was completion of the ACT DMD trial. Now that being said obviously they are reviewing the ACT DMD data and we expect to have a decision by mid-year on the applications to remove the conditions from a market in our authorization. And let me – I know everyone is thinking about RTF, but what I can remind you is the last time we received an RTF from the FDA for our NDA was based on the Phase 2b data that the EMA approved Translarna based on those same results.

Just maybe one last one here for Mark and Shane perhaps, just may be break out the number of patients on drug in the EU and the rest of the world I guess it would be not EU, not US, and then maybe break out the current number of patients that you have receiving drug in Germany for us? Thank you.

So Chris thanks for the question. I think you heard on the call earlier that we estimate there are about 5,000 nonsense mutation DMD patients ex-US. We've already identified about 1,000 of those patients, we’ve given you a revenue guidance that gives you a sense of the revenues that we’re going to achieve this year and that represents not only pricing assumptions but patient growth. We’re not breaking this down at this time by region just to be clear.

Not even Germany?

Not by country, no.

Got it, okay, I tried. Thanks.

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

So the first one may be for the refuse to file letter, just wondering did the FDA ask about the biomarker data.

Yes. So in terms of the discipline data I think it's important that no mention of the dystrophin or biomarker measurement was stated in the RTF and that we are very confident that the FDA did not have any issue with the fact that we were not measuring dystrophin and ACT DMD. We had discussed our trial design with the agency multiple times and I think this is very consistent with what the FDA said at both their dystrophin workshop last June and the advisory board and briefing books recently from the other DMD companies.

So I have a few questions regarding the European market. So, I think Mark you mentioned that there are six countries that secured reimbursement. Just wondering, I think I have the name Germany, Denmark, Norway, Austria, and Sweden just wondering what the sixth country?

We've actually had reimbursement in Slovakia, Hungary and the Czech Republic just to be clear in addition to the countries you mentioned. So in total --

Maybe it's worth just --

So Austria, Denmark, Norway, Slovakia, Hungary, and the Czech Republic.

I see, okay. And as for the $33.7 million in 2015, wonder if you could tell us how much was generated from actual commercial launch which means through the reimbursement system and how much were from patient name programs?

We don’t give that breakdown. It’s actually an interesting combination of both, because as you know, there are mechanisms that are allowed to reimburse early access as well as full commercial launch, but actually the distinction between those two becomes quite blurred and at the end of day, we are selling Translarna in 23 countries now.

Okay. So maybe last question, so for Germany if you delist Translarna, can patients only get drugs from the countries that had commercial launch or can they also get drugs from countries with the patient name program?

Either it’s probably possible, but it’s most likely, because the authorities will look to -- or the pharmacists will look to source where it’s easiest to get the drug, they will probably go to places where there is already good commercial availabilities from a supply chain point of view.

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

Hi, guys. Thank you for taking the questions. Regarding the RTF, what is your plan forward given what’s in the letter, do you plan to go through an appeal process or how do you plan to go about, I guess, convincing the FDA regarding the effectiveness of the drug?

Yeah. Thanks, Simos. So we’ve already put in a request for a call with the FDA. There is a clear path, as you mentioned, to escalate this decision within the agency. We’re also entitled to a Type A median, which is granted within 30 days. So, our goal is to -- with the FDA is to discuss and agree upon a path forward for the best possible outcomes and following these discussions, we’ll be in a better position to update you on what that path is.

Okay. And then in Europe, can you remind us, I don’t know if it has been disclosed in the past or not, but does the CHMP that’s reviewing the CF, is it the same, the same repertoires that are reviewing the conditional approval in DMD.

Yeah. The repertoires that review the DMD are the same repertoires who review CF.

So the, okay, all right. Thank you very much.

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

Hey, guys. This is [indiscernible] for Alethia. Thanks for taking the question. So just in terms of Latin America, since you guys were talking about the large number of patients in this region, could you discuss sort of how pricing is at here and how much this price depends on the list price in Europe?

Sure. Mark wants to talk a little bit about that?

So. Hi, yes. We’re obviously delighted now that Translarna is available on a reimbursed basis in Argentina as well as Brazil and Columbia and Peru. The pricing there is actually set in dollars and I obviously don’t go into specific prices as we said before, prices are integrated into our revenue guidance that includes the overall net pricing as well as patient numbers, waits, et cetera. So I’m not in a position to give you specifics around those countries.

Okay. That’s helpful. Thanks. And then just a quick follow-up, in terms of sort of how the delisting would potentially work in Germany. Can you maybe sort of help us think about how this would work particularly with some maybe examples of drugs that delisted from the German pharmacy ordering system, but then continue to say towards a higher list price in other European countries or maybe if there are situations we can love to where the commercial prices may have differed significantly, say between different European countries? Thanks.

So I understand that the law change in 2011 that allows for this foreign drug importation and that there aren’t 100s of these examples, but for example, I can give you a few today that for epilepsy, GSK’s Trobalt and Eisai's Fycompa are examples of products that were delisted and then actively imported. I mean not only was it communicated that the drugs were available through company communication, but also interestingly the insurers themselves communicated that the drugs were available through foreign importation. So this is a tested route. I think what is also I think very important to think about is that Translarna is for a very high unmet medical need. There are no alternatives and the drug has been given a good benefit rating by the G-BA. So I’m sure the insurers when they look at this, will take a favorable view.

Thank you. Our next question comes from the line of Ritu Baral with Cowen. Your line is open.

Hi, guys. Thanks for taking the follow-up. Stuart, now that you’ve had a few days to think about the RTF letter, if you start talking to FDA and the worst case scenario unfolds, what is your commitment, what is PTC’s commitment to potentially doing another, yet another study for Translarna in DMD or what are your thoughts at this point on the worst case scenario?

Yeah. So we’ve been obviously doing a lot of thinking about this over the last few days and really our goal is to get Translarna approved in the United States as it is in Europe and other places around the world. In terms of when you think about our commitment, I could tell you we’ve been -- I founded this company about 18 years ago and gone through a number of ups and downs of this process. So I think in terms of our commitment, it’s been very strong. Ultimately though, we have to discuss all our options with the agency as we move forward. And so until we have more dialog, it’s probably premature to predict what the next steps are or whether we need additional trials. I will point out also, we’ve already done two large well controlled placebo controlled trials that define the very robust dataset and so we’re going to be talking to them about those results and how we could move forward on that.

And then on the subpopulation analysis that you mentioned that you have proposed in the plan, but that was not commented upon, was there any subpopulation, any six minute walk cut that you presented at the time of top line data that was commented on, on when you submitted this test plan or was none of it essentially commented on?

I think what was -- when you think about, what we said was we characterized this that the FDA characterized that the results in the NDA are not supportive of effectiveness and this would include the subgroup analysis. So I think what we need to do is to look -- is what we want to do is to go forward with the FDA to discuss this with them further.

Okay. And last question, the expanded access program and the named patient programs that you have ongoing currently, is there any time limit on any of these or expiration date on any of the programs that you have in place right now?

There aren’t any specific deadlines that are sort of date deadlines, but what you will see is that in countries in Europe for example where the early access program is possible, those will transition once we’ve been through the pricing reimbursement discussions into full commercial sales. So it’s a sort of an evolutionary process. In countries outside of Europe, like for example Singapore or we just recently talked about Argentina, we still have to at some point file for local registration and until that happens, it will continue to be on a name patient basis.

So but it sounds like in Europe, you can’t sustain the business just on a named patient program going forward in perpetuity at some point, the price discussions will be held and…

Yes. The system is designed to transition from early access through to full commercial by the pricing reimbursement discussions. So that’s the normal process.

And is this a 2 to 3 year process from this date as far as you can get a handle on right now?

It just depends on every country system. They’re all different. So some happen faster and some can take longer. It just depends on the timelines and the process for each country.

Thank you. That does conclude our Q&A session. I would like to turn the call back to Stuart Peltz for closing remarks.

Well, I just want to -- we all understand that these are frustrating times for all our constituents, not only for our investors, but for the US boys and young men and their families who are suffering from Duchenne muscular dystrophy. For them, every day counts and every function loss cannot be regained. That’s why I work so hard to bring Translarna to the patients and I will focus our energy to resolve the process with the FDA and bring this therapy to the US and also mutation DMD patients. Thank you for joining the call.

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