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Edited Transcript of PTCT earnings conference call or presentation 28-Feb-19 9:30pm GMT

Q4 2018 PTC Therapeutics Inc Earnings Call

South Plainfield Mar 16, 2019 (Thomson StreetEvents) -- Edited Transcript of PTC Therapeutics Inc earnings conference call or presentation Thursday, February 28, 2019 at 9:30:00pm GMT

TEXT version of Transcript

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Corporate Participants

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* Christine Utter

PTC Therapeutics, Inc. - Principal Financial Officer & Treasurer

* Emily Hill

PTC Therapeutics, Inc. - Executive Director of IR

* Marcio Souza

PTC Therapeutics, Inc. - COO

* Stuart W. Peltz

PTC Therapeutics, Inc. - Co-Founder, CEO & Executive Director

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Conference Call Participants

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* Alethia Rene Young

Cantor Fitzgerald & Co., Research Division - Head of Healthcare Research

* Brian Corey Abrahams

RBC Capital Markets, LLC, Research Division - Senior Analyst

* Eric William Joseph

JP Morgan Chase & Co, Research Division - Analyst

* Gena Wang

Barclays Bank PLC, Research Division - Research Analyst

* Joel Lawrence Beatty

Citigroup Inc, Research Division - VP & Analyst

* Mark William Connolly

Crédit Suisse AG, Research Division - Research Analyst

* Raju Yashaswi Prasad

William Blair & Company L.L.C., Research Division - Senior Research Analyst

* Ritu Subhalaksmi Baral

Cowen and Company, LLC, Research Division - MD and Senior Biotechnology Analyst

* Tazeen Ahmad

BofA Merrill Lynch, Research Division - VP

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Presentation

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Operator [1]

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Good day, ladies and gentlemen, and welcome to the PTC Therapeutics Fourth Quarter and Full Year 2018 Financial Results Conference Call. (Operator Instructions) As a reminder, this call is being recorded.

It is now my pleasure to introduce Head of Investor Relations, Ms. Emily Hill. Please go ahead.

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Emily Hill, PTC Therapeutics, Inc. - Executive Director of IR [2]

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Hello, good afternoon, and thank you for joining us to discuss our 2018 fourth quarter and full year corporate updates and financial results. Joining me on today's call is our CEO, Stuart Peltz; our Chief Operating Officer, Marcio Souza; and our Principal Financial Officer, Christine Utter.

Before we start, let me remind you that today's call will include forward-looking statements based on current expectations. Please take a moment to review our slides from our current corporate presentation, which contains our forward-looking statements. Our actual results could materially differ from these forward-looking statements as any and such risks can materially and adversely affect our business and results of operations.

For a detailed description of applicable risks and uncertainties, we encourage you to review the company's most recent annual report on Form 10-K filed with the Securities and Exchange Commission, as well as the company's other SEC filings.

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 pass the call over to our CEO, Stuart Peltz.

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Stuart W. Peltz, PTC Therapeutics, Inc. - Co-Founder, CEO & Executive Director [3]

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Thanks, Emily, and thank you for joining us on today's call. 2018 was a transformative year for PTC. Going into the year, we set out several ambitious goals. I'm proud to say that we achieved those goals. We met our DMD revenue guidance, expanded the use of Emflaza, achieved a label expansion for Translarna and advanced our oncology pipeline with 2 potential products now in the clinic.

Importantly, we also advanced our SMA program. Following a productive discussion with the FDA, we and our partners, Roche and the SMA Foundation, feel confident to submit the results of FIREFISH and SUNFISH in an NDA later this year. We are very proud that the SMA candidate resulted from PTC's internal splicing program. We have been utilizing this platform to identify other compounds that selectively and specifically modulate splicing. We have a number of other splicing programs that are solely owned by PTC.

Besides achieving these goals, we also strengthened PTC with 2 business development deals, adding a CNS gene therapy platform with multiple programs, and in-licensing 2 products that we plan to commercialize in Latin America. You may recall that in early 2018, we presented a 3-year vision for the company to become a fully integrated orphan drug company. Execution is at the core of everything we do at PTC, and we are proud to have accomplished this 3-year plan in just 1 year.

Now, looking forward, we have defined what we want to deliver over the next 3 to 5 years. We described these longer-term goals at the beginning of this year, and I'd like to review them with you today. On the commercial front, in 2018 we had $263 million in revenue from the DMD franchise. Over the next few years, we anticipate 3 product launches - risdiplam, TEGSEDI and AADC gene therapy, as well as expanding Emflaza's label to include DMD patients 2 to 5 years old. We also have potential for a U.S. approval of Translarna in 2020 based on the outcome of a dystrophin study that started at the end of last year.

The combined potential revenue of these products that are currently in our pipeline is anticipated to grow in excess of $1.5 billion in potential revenues by 2023. We will continue to invest and expand our discovery clinical-stage pipeline. We plan to accelerate 20 programs over the next 5 years through internal and external innovation sources across small molecule and gene therapy.

In addition to outlining our 5-year vision, we have also recently provided updates on key programs that we'll cover in more detail later in the call, including patient identification efforts ahead of our planned BLA this year for the AADC deficiency, a planned NDA submission this year for SMA and the anticipated launch of TEGSEDI in Latin America.

We are proud that PTC has positioned itself to be able to become a leading orphan drug company with diversified pipeline of innovative programs and products. We are leaders in DMD, have built a solid revenue base and have strong talent with committed and motivated employees. We ended 2018 with a cash balance of $227 million and recently completed an equity offering with net proceeds of approximately $225 million. We are in a very strong cash position to execute on our strategic vision.

I'll now pass the call to Marcio to review our clinical and commercial progress.

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Marcio Souza, PTC Therapeutics, Inc. - COO [4]

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Thanks, Stu. We have a vision of building a portfolio of multiple products to serve patients with rare disease with the potential 2023 revenue in combined excess of $1.5 billion, as Stu just discussed.

Our expertise in all aspects of our business, from patient finding to (inaudible) access, is a key driver of our long-term growth. We have spent considerable time and efforts to build cross-functional teams that can rapidly increase disease awareness, identify the patients and bring differentiated therapies to the patients that need them. This includes a state-of-the-art patient service, which is a key component of rare disease treatments.

As Stu also mentioned, our DMD franchise had an exceptional 2018, reporting a combined revenue of $263 million. This includes sales of Translarna outside of the U.S., which is now in over 40 countries, and sales of Emflaza in the United States.

In 2018, the EMA approved our label expansion for Translarna to treat nonsense mutation DMD patients aged 2 to 5. Importantly, this label expansion allows patients in all the countries that recognize the EMA approval to gain access to therapy at a younger age, which we believe would maximize the benefits to them.

Additionally, there is an equal and very (inaudible) unmet needs to treat nonambulatory patients. With that in mind, late last year, we have submitted to the EMA a label expansion application for the treatment of nonambulatory patients. We are in the process of answering some questions we got from the EMA, and we expect to finalize the regulatory process later this year. We believe Translarna has the potential to benefit all patients regardless of where they are in the disease progression.

In the United States, we have begun our dystrophin study, which will lead to an expected resubmission of our Translarna NDA for accelerated approval later next year. Our DMD franchise also includes Emflaza, which is approved for all patients, ambulatory and nonambulatory, age 5 and older in the United States. We continue to work hard to establish Emflaza as the standard of care in the U.S.

At the end of last year, we received a request from the FDA to file for pediatric label expansion for Emflaza. The FDA informed us that we have sufficient data to file the sNDA and that a study would not be required to demonstrate safety and efficacy in this population. We have submitted the sNDA for Emflaza for patients 2 to 5 years old at the beginning of this year, and the action date we received from the agency is July 4. Our commercial team is diligently preparing for the launch in this expanded indication later this year.

Based on the expected continued growth of both Translarna and Emflaza, we have given 2019 DMD franchise revenue guidance of $285 million to $305 million. We are proud to have established this strong commercial footprint globally, which has made us the partner of choice for some very unique business development opportunities.

Last year, we in-licensed Latin America commercial rights to 2 products from Akcea, TEGSEDI and Waylivra. To be successful in the region, it's essential to have the right process and the right team in place. Leveraging our Latin America infrastructure, we have completed all necessary additional hiring to support these products. Importantly, we have also established the necessary process to support patients, including an exclusive partnership with a nursing support team.

On the regulatory front, we have filed TEGSEDI with Anvisa and we expect approval later this year. We are eager to bring TEGSEDI to the Latin America market, where the polyneuropathic form of hATTR is the most prevalent, with approximately 6,000 patients in that region. We're especially proud of this progress in a competitive market, as this one is expected to be. The speed and execution are critical for success, and we believe we are ahead of the game there.

I would now like to highlight our CNS gene therapy platform. As you may recall, our strategy in pursuing CNS-specific gene therapy includes some key advantages. The first is the ability to target the specific area where the disease process is occurring, maximizing the benefit/risk. Secondly, because cell turnover in the brain is so low, there is the potential for durable effects and the potential to use a small amount of the vector, which we call microdosing, which reduces the amount of factoring burden for the process. Lastly, the combination of the target approach with this small dose reduces the potential for immunogenicity, reducing the overall risk for the programs.

Our gene therapy products are rapidly advancing. With AADC, we have started precommercial efforts ahead of the anticipated filing of the BLA later this year. As we said before, we have already identified approximately 100 AADC-deficiency patients in the United States and Europe through early key opinion leader interactions. We continue to identify new patients steadily, week after week, where we're now moving to a more focused screening program at at-risk populations.

For instance, we have identified a cohort of approximately 100,000 apparently misdiagnosed patients in cerebral palsy clinics in the United States. Those patients present with normal MRIs. We expect to have the screening programs in cerebral palsy implemented this quarter. We plan to share an update on the screening programs as well as on the patient identification later this year.

Beyond the AADC, we plan to submit an IND by the end of this year for the next gene therapy program in the pipeline to treat Friedreich's ataxia. FA is a devastating disease with no real underlying treatments at this point. We have all the elements in place to start the clinical trial in patients following the IND submission. Next, the most advanced program is our gene therapy candidate for Angelman syndrome, which we plan to file an IND next year.

As we have said, we are developing a robust pipeline of gene therapy candidates, and to that end, we intend to declare new candidates later this year. In order to support this growth in the gene therapy pipeline, we are also in the process of securing in-house gene therapy manufacturing. Lastly, we continue to advance our oncology programs with several patients dosed in our DIPG trial and having great progress in our AML trial with PTC299. We expect both trials to complete enrollment before the end of the year.

I will now turn the call back to Stu.

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Stuart W. Peltz, PTC Therapeutics, Inc. - Co-Founder, CEO & Executive Director [5]

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Thanks, Marcio. The progress that's been made across our clinical and commercial fronts is impressive. I'd now like to highlight our splicing programs and the area of innovation in which we are proud to be both the pioneers and leading the field.

An emerging area of therapeutic focus is the ability to modulate splicing with a small molecule, in which our technology produced the most advanced compound, risdiplam. We expect this compound, which is partnered with Roche and the SMA Foundation, to be potentially approved next year for SMA types 1, 2 and 3.

This is great news for patients with SMA, but it has a broader implication for PTC as a powerful validation of our splicing platform technology. It is now clear that we can identify selective and specific compounds that modulate splicing to treat disease. A small molecule also has the advantages of broad tissue distribution, as well as ease of administration.

We are applying our splicing platform to other diseases with high unmet medical need and have programs across the R&D spectrum. These include familial dysautonomia and Huntington's disease. I'm happy to report that at the end of 2018, we declared the development candidate for familial dysautonomia, and we expect to enter the clinic this year.

I'd now like to turn the call over to Christine Utter, our Principal Financial Officer. Christine?

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Christine Utter, PTC Therapeutics, Inc. - Principal Financial Officer & Treasurer [6]

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Thanks, Stu. Earlier today, we issued a press release summarizing the details of our financial results for the fourth quarter and full year of 2018, and I refer you to the release for full details. I'll start with a few comments on our financial performance and our guidance for 2019.

Starting with our top line results, we reported $263 million in combined revenue across our DMD franchise for the full year of 2018, compared to $174.1 million in 2017. Translarna net product revenues were $171 million for 2018. This compares to $145.2 million in 2017, representing an 18% year-over-year increase in Translarna revenue. For Emflaza, we reported net product revenues of approximately $92 million for 2018, a revenue increase of over $60 million from 2017.

We are proud to provide our expectations with respect to continued commercial growth. Our 2019 DMD franchise revenue guidance is $285 million to $305 million. As Stu mentioned, we have also outlined the potential combined revenues for products in our existing pipeline in excess of $1.5 billion by 2023.

Non-GAAP R&D expenses were $155.9 million for 2018, excluding $16.1 million in noncash stock-based compensation expense, compared to $102 million for 2017, excluding $15.5 million in noncash stock-based compensation expense. This increase in R&D expense reflects costs associated with advancing our gene therapy platform and increased investment in our research programs, as well as advancement of our clinical pipeline.

Non-GAAP SG&A expenses were $136.4 million for 2018, excluding $17.2 million in noncash stock-based compensation expense, compared to $106.2 million in 2017, excluding $15.1 million in noncash stock-based compensation expense, reflecting continued investment in commercial activities to support our DMD franchise.

I would also like to reiterate our non-GAAP R&D and SG&A expenses guidance for full year 2019 of $360 million to $370 million, excluding noncash stock-based compensation expense of approximately $35 million. This expense guidance reflects advancing gene therapy and supporting our commercial products.

Net loss for full year 2018 was $128.1 million, compared to a net loss of $79 million for 2017. Cash, cash equivalents and marketable securities totaled approximately $227 million at December 31, 2018, compared to $191 million at December 31, 2017. I would also like to point out we recently completed a public equity offering resulting in combined net proceeds of approximately $225 million.

I will now hand the call over to the operator to start our question-and-answer session. Operator?

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Questions and Answers

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Operator [1]

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(Operator Instructions) And our first question comes from the line of Ritu Baral with Cowen.

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Ritu Subhalaksmi Baral, Cowen and Company, LLC, Research Division - MD and Senior Biotechnology Analyst [2]

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I have 1 question and 1 follow-up. The first question is on risdiplam and the submission. What indication or label are you going to be submitting for? I know the 2 trials span type 1 and type 2, but do you anticipate filing for a certain age range, a certain weight range? And then my follow-up question is on the patient identification activities for AADC. You mentioned that there was a pool of about -- a pool of 100,000 patients misdiagnosed with cerebral palsy with normal MRIs. Can you say, like, how many centers that's across and whether this is some sort of opt-in program that the screening will use, or will you just sort of go through the centers and go through blood spots? Thanks for taking the questions.

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Stuart W. Peltz, PTC Therapeutics, Inc. - Co-Founder, CEO & Executive Director [3]

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Hey, thanks, Ritu. Yes, so in terms of the SMA, I think the plan is really, and it was based on our conversation with the FDA, was to file based on Part 1 of both type 1, 2 and 3. So really, to get a broad label within that. As for AADC, maybe let -- Marcio, you want to?

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Marcio Souza, PTC Therapeutics, Inc. - COO [4]

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Yes, yes, absolutely. And maybe just -- hey, Ritu. Just to finish on the -- on what Stu started, right, for -- that's no expected restriction on the age or weight of those patients. We feel that we have a very comprehensive program, several cohorts that examine that relationship. Roche did a really good job here on executing that trial. And that's why we are reinforcing what our belief is, that this is the most competitive SMA program globally. With a move to the AADC effort of patient identification, there are basically 2 layers of clinics in the United States, and outside of the U.S. there's a lot of, like, different dynamics. In the U.S., there are, like, 65 or so main centers for cerebral palsy, and then our expanded cohorts, that is up to 200 give and taken.

The way we're doing this is through a multipronged approach, so one is, we're offering 4 centers, if they want to conduct the screening as a "research project," so that's basically a protocol-driven, we're going to go for the IRBs, like contract with them, so on and so forth. And we have a very good uptake there. There is a number of the centers that really wants to do it that way and go for their entire population. For the ones that either don't have the capacity right now or they don't have, like, the structure to run as a research, we have what we call a known protocol; we're providing them the ability to screen their patients. And we have those centers opt in there as well.

Currently, we're looking into, like, the sample of the whole bloods, but we are validating and finishing the steps of validation for dried blood spot, so moving to that process, it's going to be faster as well, more productive throughout the year. But all -- pretty much all have been on board on the -- what we call Tier 1. That's the majority of the patients are in. So, should start screening patients in the next couple days, so in Q1. And expect to report the progress, like, later in the year, in a more consolidated fashion. Does that help?

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Ritu Subhalaksmi Baral, Cowen and Company, LLC, Research Division - MD and Senior Biotechnology Analyst [5]

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Yes, it does. And for the screening, is this something that's sort of done in the background anonymously, or is it something that you need sort of individual family, individual patient -- parent, I guess, approval to run the assay?

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Marcio Souza, PTC Therapeutics, Inc. - COO [6]

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Yes. No, that's a really great question. So for the one that is done with the protocol -- so this is part of, like, a "research" program, right? So it's -- there is a consent form, and they're going to go through that, and so on. For the other one, it's basically a tool that's being offered to the families. We are looking to this marker; in the U.S., we can do, like, a 3-OMD, so it's a precursor of dopamine. That's very simple and when that's elevated, we go and you genotype these patients. Outside of the U.S. is a little bit more country by country. Most of them we're just providing the test and they're going through and screening the patients they have, so it's more a conversation with the family. As you can imagine, and every day we learn more about this as well, these families really want to know if it is not cerebral palsy, if that's something that can be done, so we're seeing, like, very motivated families seeking this and having positive conversations. So we don't see, like, any barrier there. The barriers were more on putting the process in place, and now that we have that, it should go relatively quickly.

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Operator [7]

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And our next question comes from the line of Eric Joseph with JPMorgan.

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Eric William Joseph, JP Morgan Chase & Co, Research Division - Analyst [8]

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Just a couple from us. The first on guidance for 2019. I just wanted to unpack DMD sales a little bit. It's -- the top end of the range still looks as though you're tracking under the run rate for fourth quarter, so I know that you have talked a little bit about the expectation to see -- in seeing lumpy demand over the course of the year, but is there anything else that's implicated in the -- in current guidance? Anything in the -- with respect to pricing? And if there are any expectations around pricing headwinds, where might they be coming from? And then I have a follow-up.

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Stuart W. Peltz, PTC Therapeutics, Inc. - Co-Founder, CEO & Executive Director [9]

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Yes, great. I think -- thanks, Eric. I'll start and then pass it on, and I think -- see, I think we're adding on patients both for Emflaza and Translarna that continue to grow. We do plan on some approvals in certain countries that can have transient aspects on the dynamics of the market, but I'll pass it on to Marcio to sort of go into what their thinking is on that.

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Marcio Souza, PTC Therapeutics, Inc. - COO [10]

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Sure thing, Stu. And hey, Eric. So a couple dynamics here, and I think they are important for everyone to understand. So one is this, Stu just mentioned. We are seeing patient growth, or net patient growth, in both products in all geographies. So the premise of the business and the base is quite positive. There are -- the way we're looking into this, right, is to reach our goal, like a 2023 goal that we presented earlier this year, is the dynamic of patients over time increasing staying on therapy. And to do that, in some of the countries this year, we believe we're going to have to make some adjustments in terms of the pricing/volume mix. Some -- so there is a little uncertainty, I would say, on that, that we are accounting for in the guidance. Specifically as it comes to Translarna, since some of these markets became quite big, but there is still a lot of potential.

So when you're looking into the amount of patients that are being treated versus the potential, it doesn't make sense for us to just, like, be stubborn and not try to gather as many patients, because at the end of the day that's why you're doing business, is to treat these patients. So there might be a little bit of a negative impact on the price mix throughout the year, which is going to correct once we get those patients all on drug and then the other countries started. So that's one that might impact.

I think it's equally important to talk about what is not in the guidance, right? So we don't have the 225 for Emflaza. You might have noticed on the prepared remarks that we talked about the July 4 approval by the FDA. We received the notification by them that that's the action date. We hope it's an approval. And then with that, we're going to be executing the launch. While patients between 2 and 5 are not necessarily fully diagnosed yet, there is a pool of patients there, and we're going to be adding those, and depending on how fast we can, that might have a significant upside for the guidance. And the nonambulatory in Europe is not included either here, since we are still in regulatory conversations. So we tried to be, as we always are, like, quite responsible in the way we guide, considering all the market dynamics.

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Eric William Joseph, JP Morgan Chase & Co, Research Division - Analyst [11]

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Got it. And so just a follow-up on Emflaza, if I could. We can see that the trial design is up on clinicaltrials.gov. Just wondering how we should be thinking about recruitment timelines; also, the level of input and discussion had with FDA in its design. And I'm also curious to know whether there are opportunities for -- to expand the exclusivity runway here, potentially through an additional orphan indication. Thanks.

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Marcio Souza, PTC Therapeutics, Inc. - COO [12]

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Sure. And are you referring to the limb-girdle (multiple speakers)?

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Eric William Joseph, JP Morgan Chase & Co, Research Division - Analyst [13]

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Sorry, the limb-girdle Phase 3 study, yes.

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Marcio Souza, PTC Therapeutics, Inc. - COO [14]

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Yes, yes, I thought so, because 33, that was the 225, we actually removed because the FDA told us we don't need to conduct that anymore. So we gave -- we got a lot of inputs from key opinion leaders here from the biggest center in the U.S. that reads [2i]. We're fairly confident with some of the natural history they gave to us. That was what drove the design. Obviously we filed that with the FDA. And the recruitment we expect to be relatively fast, because there is absolutely nothing approved or in development for that specific subtype of limb-girdle, which is the most prevalent type in Caucasians, at least with the not so vast and expanded literature that is available there. So we're going to provide a little bit more updates later in the year on how it's going, but we feel really good about that.

On the expansion of the runway in terms of the product, there's a number of things we are doing. Some of them have to do with -- for some populations, it's not that convenient to deliver. Maybe some other formulations would be more appropriate here. There is a number of elements that we're not quite ready to discuss publicly, but we should be at some point, and you might see some things coming up at clinicaltrials.gov as well, in the near future in terms of how we are approaching that. Right now we are -- the base case is the runway that is present for the products, but we are, as I just said, we are looking for a number of ways to potentially expand that.

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Operator [15]

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And our next question comes from the line of Gena Wang with Barclays.

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Gena Wang, Barclays Bank PLC, Research Division - Research Analyst [16]

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First one is, just wondering, maybe follow Eric's question, what is the average pricing now for Translarna? Previously our understanding was, like, $300,000.

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Marcio Souza, PTC Therapeutics, Inc. - COO [17]

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So -- hey, Gena, it's Marcio. So the price has been quite stable for Translarna, right? We haven't guided to any specific price. But one thing that we took a lot of care of is, we don't want, like, countries to be (inaudible) importing or patients to be treated differently in one country or another, so we kept the price fairly stable globally. We have some, like, large markets, like, entering now, with the potential to expand dramatically, so that's put a little bit of a negotiation dynamic on the table. And it's the 5th year of the product as well, so we are looking into here, where are the patients that are not being treated? So quite positive, the dynamic so far, especially for an international product that is not in the U.S. We're not talking -- when I'm talking about price mix, we're not talking about crazy erosion of value. I just wanted to make that clear. But we believe there are some responsible adjustments that can be done in order to expand, just proportionally, the number of patients on drug.

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Gena Wang, Barclays Bank PLC, Research Division - Research Analyst [18]

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Okay, so -- but just wondering, are we talking about, like, 20%, 30% discount, or are we talking about 50% discount?

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Marcio Souza, PTC Therapeutics, Inc. - COO [19]

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No.

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Gena Wang, Barclays Bank PLC, Research Division - Research Analyst [20]

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In the new territory?

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Marcio Souza, PTC Therapeutics, Inc. - COO [21]

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Yes, yes. I think what you're talking about is, depending on the size of the opportunity on some of those markets, and the willingness of the payer, the governments to negotiate longer-term contracts, that we would be willing to give discounts like we have in the U.S. for CMS, for example. That's the kind of thing we are talking about.

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Gena Wang, Barclays Bank PLC, Research Division - Research Analyst [22]

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Okay. And then another quick question regarding risdiplam. Just wondering, would early data of pivotal portions from both SUNFISH and FIREFISH be part of FDA final decision, since both trials already complete enrollment now, and should have initial data by the time FDA would take a look and then make a decision?

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Stuart W. Peltz, PTC Therapeutics, Inc. - Co-Founder, CEO & Executive Director [23]

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Yes. So no, the plan, though, is really -- is based on Part 1 of the -- both SUNFISH and FIREFISH trial to make that sufficient for -- we thought that we had very good discussions and that this would be sufficient for approval. So that's the game plan of what we're planning on.

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Gena Wang, Barclays Bank PLC, Research Division - Research Analyst [24]

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Okay. And then very quick last question, regarding the Latin America commercial preparation, just wondering, how much investment in terms of dollars you would need to set up a sales force and to be commercial-ready for -- in both TEGSEDI and Waylivra?

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Marcio Souza, PTC Therapeutics, Inc. - COO [25]

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Yes, no, of course. So first thing is just to say, we hired everyone we had to hire in Latin America. Kudos to Eric and his team, who were, like, very ready to do this, executed in a matter of weeks. So we don't actually expect to add anyone else to support TEGSEDI. We obviously don't have anyone dedicated to Waylivra since the product's not approved elsewhere, so we're not putting any efforts right now. There are basically 3 major components, right? The commercial, medical and then the patient supports. And we see them kind of equally. Like, in this phase right now, it's mostly unsolicited requests, so our medical affairs folks are in the fields. We have neurologists on staff. We have, like, great healthcare professionals on our medical staff that are giving that support. Equally important is the patient monitoring, both to help them maximize the standard of care, the testing, get familiar with the testing program we're going to have later, but then understands this connection with PTC, and that's in place as well. It's a third party. In our view, it's the best provider of this kind of service in Latin America, and it's an exclusive contract with us. And then the third part is, we did not create a special field force for TEGSEDI. We're actually expanding what I normally call solve for ZIP Codes, meaning we are reducing the areas of coverage. Brazil's a very large country. Argentina is a large country. We're reducing the area of coverage so we have more people doing both products, because what is seen is, there is about 50% to 70% overlap between the locations and the key opinion leaders and treaters with Translarna and TEGSEDI, so it's highly complementary, so.

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Stuart W. Peltz, PTC Therapeutics, Inc. - Co-Founder, CEO & Executive Director [26]

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And so I think the good point there, as well, is that these are already ongoing efforts.

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Marcio Souza, PTC Therapeutics, Inc. - COO [27]

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Correct. All of it.

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Stuart W. Peltz, PTC Therapeutics, Inc. - Co-Founder, CEO & Executive Director [28]

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That all this is -- we've already started working, and so physicians are aware of this, and we've also submitted for approval already, so -- which we anticipate will be this year. So there's already a lot of effort that's been ongoing to get ready for this.

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Operator [29]

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

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Joel Lawrence Beatty, Citigroup Inc, Research Division - VP & Analyst [30]

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The first one is on TEGSEDI and kind of a follow-up question of what was just asked. And what do you see as the reasonable expectations for the shape of the launch curve there, in terms of the key steps that need to be taken towards ramping up sales?

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Marcio Souza, PTC Therapeutics, Inc. - COO [31]

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Sure. Hey, Joel. So the beginning of the launch, or the beginning even prelaunch, we expect to have a number of patients as we -- to be perfectly honest, we are seeing these already, requesting TEGSEDI for the different channels, right? The more they hear about it, the more they hear, the fact that actually PTC is involved, since we have, thankfully, a very positive reputation with the physicians who are starting to get requests, and this is being treated as (inaudible) patient requests and going through the process, this takes several months. So it's going to be fairly slow in the first few months. What we expect is, right after the approval, and I'm not going to answer too much on the strategy here because this is a potentially competitive market, but to actually accelerate real quick, we're entering next year with a potential substantial gain of market share and then growing on the following years. So it's low, I would say, on the next 12 to 18 months, and then it's starting to have, like, a hockey stick type of -- so it's an unusual launch, if you were to think about major geographies like the U.S. and Europe, but fairly usual for rare disease in the geographies in Latin America.

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Stuart W. Peltz, PTC Therapeutics, Inc. - Co-Founder, CEO & Executive Director [32]

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We do think there's a lot of advantages here as a consequence of just the properties of TEGSEDI, that it's injectable, that there'll be -- the ease of, in a sense, of patient pickup as a consequence of that, and we're out there now.

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Marcio Souza, PTC Therapeutics, Inc. - COO [33]

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Exactly.

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Joel Lawrence Beatty, Citigroup Inc, Research Division - VP & Analyst [34]

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Okay. And then maybe one other question on Translarna. You have the new Phase 3 trial that initiated late last year. Could you discuss the design of that trial and what needs to be shown to support an approval in the U.S.?

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Stuart W. Peltz, PTC Therapeutics, Inc. - Co-Founder, CEO & Executive Director [35]

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Marcio, why don't you...

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Marcio Souza, PTC Therapeutics, Inc. - COO [36]

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Sure, of course. So that -- we call that study 44 -- 45, sorry. I just got the number wrong there. So it's basically -- it's an open-label study with 20 patients aged 4 to 7. We're taking a biopsy at baseline. It's a single center in California and another at 9 months. We're not going to read any of this before all the samples are collected, right? So that becomes the block control in terming the trial. We're using 2 methodologies which were discussed and agreed with the FDA to measure dystrophin. So the expectation is that we would see a statistical difference between the baseline and the 9 months in terms of increase of the amount of protein as measured by an ELISA-type method that's called ECL and a secondary measure that is ICH, is a more defined ICH, actually, on (inaudible), where we would see the localization in the sarcolemma, as you expect to. So that's the trial we're enrolling right now. We have a couple of patients on the trial. We, like, have a good interest for that. We expect to enroll, like, this year, in the next several months, with a potential resubmission. Obviously we expect the trial to be positive. We hope it's going to be positive, and we're going to resubmit to the FDA under the old NDA early next year.

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Operator [37]

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And our next question comes from the line of Tazeen Ahmad with Bank of America.

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Tazeen Ahmad, BofA Merrill Lynch, Research Division - VP [38]

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Maybe one about risdiplam. I think, Stu, in your current deck that's posted today, as well as the deck that was available back in January, you talk about $1.5 billion in potential revenues to the company by 2023. And in that pie chart, you talk about roughly $200 million in royalties to PTC on risdiplam. I was wondering if you could give us some color on how you came up with that number, vis-a-vis either priced or market penetration into the SMA market.

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Stuart W. Peltz, PTC Therapeutics, Inc. - Co-Founder, CEO & Executive Director [39]

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Yes. So we looked at what we thought would be, in terms of in excess of $2 billion, where -- and it was based on that, and I think you could see in the previous -- in a previous press release, we'd put out precisely what was the royalties where the -- for example, it was in the excess of 14% above $1 billion, in excess 16% -- so 16% above $2 billion. And so it was based on that sort of mix, that we looked at the numbers to be around $200 million.

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Tazeen Ahmad, BofA Merrill Lynch, Research Division - VP [40]

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And based on early doctor checks that we have done, it certainly seems that physicians would be excited for an oral. Could this number be conservative if the switch rate potentially from Spinraza is faster?

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Stuart W. Peltz, PTC Therapeutics, Inc. - Co-Founder, CEO & Executive Director [41]

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It's -- that's funny you said that. We've had meetings where sometimes people think that that is conservative as what we -- what it can be. So yes, I think -- we thought we'd start out where we are today and then discuss and grow it as we -- as time goes on.

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Marcio Souza, PTC Therapeutics, Inc. - COO [42]

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It may be -- Stu, if I may jump in here, Tazeen. So a couple of dynamics that I believe are important as well, right? So there is obviously debate on drug price internationally. We try to take, like, a relative conservative stance on that. So that's one point of, I would say, upside on the model. In general, we know the type 3s, for example, are largely unserved right now. Our own internal checks, and it seems like yours as well, show that physicians and patients would be very excited to go from, like, the teens percent that is being treated right now with the current treatment available to a vast majority of the patients with risdiplam. That market is not competitive. We expect to really dominate that. Similarly, in type 2, and then type 3s, type 1s are probably going to be the most competitive of all at the time of launch.

When you're looking to an oral molecule that is durable in effect, it's been safe to date, the -- it's obviously very easy to administer, then it comes to access. The beauty of this model is, everyone knows globally how to get access to small molecules for chronic treatment. So this is a plug-and-play. And to that point, you're right, we have a starting point here for $200 million, but we're not going to stop there, and I'm sure Roche, by all the conversations we have with them, are not going to stop there either, trying to maximize this value.

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Stuart W. Peltz, PTC Therapeutics, Inc. - Co-Founder, CEO & Executive Director [43]

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Yes. A similar product that really came out of our splicing platform, and that, when we think about it from the competitive nature of this, this really can be really competitive globally because of its -- not only because of its ease, the broad distribution, but as Marcio said, how -- people understand how to get access to the drugs like this.

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Tazeen Ahmad, BofA Merrill Lynch, Research Division - VP [44]

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Okay, thanks. And then, maybe, Marcio, on AADC, you have talked about finding about 100 patients or so in the U.S. and EU. Over what period of time has that taken you?

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Marcio Souza, PTC Therapeutics, Inc. - COO [45]

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Yes. So that took about, I would say, 10 weeks for us. I'm going to say, though, the efforts, like -- we built the team to do that, right? So that took a few weeks. Like, we have a very clear strategy on countries that are a priority, and no one's going to be surprised: the U.S., Germany for example, like a few countries in Europe. Like, these are our priority right now, so we're putting extra efforts there. Interesting thing is, like, we were just in a congress, and went to, like, a symposium on AADC. Right after that, we got, like, some new docs that we didn't really have relationships with, and we got a number of patients that they had genotyped before in a couple labs in Europe that are -- so we are seeing this, I would say, every week in different geographies, both in our core geographies that we are reporting here, but in noncore geographies as well.

You might have seen this -- I know you do a lot of really diligent work on this disease -- is the number of mutations reported at the time we did the acquisition was about 50. Now it's more than 80. So we're seeing, like, a huge effort there, and it's diversifying the pool. So I believe that teases the patients who are not there, there's the founder mutations, pretty much dead by now. You can't have 80 founding mutations. Well, theoretically you can, but then it would be in 80 different pools, and in fact, that would be a good thing for us, but that's not the -- that's not what is happening. So the last 10 weeks or so, the screening program's barely started, so we expect to see a good acceleration throughout the year as well.

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Stuart W. Peltz, PTC Therapeutics, Inc. - Co-Founder, CEO & Executive Director [46]

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And I think that's a really good point to -- well, not that there wasn't effort to find the patients, but it really was finding those that people already -- actually already knew was there, that we're able to find them. And it hasn't taken, yet, the efforts of looking for the unidentified patients yet. So that's just started. So it wasn't that -- in a sense, I know it's always tough to find patients, but relatively speaking, we're able to find them because they've already been genotyped. Now there's a whole bunch that will be in these -- that will be in those clinics that we'll be able to find, who are just misdiagnosed, so that's the next step.

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Tazeen Ahmad, BofA Merrill Lynch, Research Division - VP [47]

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Okay, so it seems like since the acquisition, you have made quite a bit of progress, so in terms of your target, 5,000 to 6,000 patients, potentially, how long do you think it'll take you to get to that, or close to that number?

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Marcio Souza, PTC Therapeutics, Inc. - COO [48]

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Great question, right? And you can imagine that I'm being quite pushy with my team and working very hard here to get there. Like, we see this is going to be a little bit of a classic rare disease launch. We're really focusing on the U.S. and some key geographies, as I mentioned. We believe it's going to be a very successful launch. We did put, on the pie that you were mentioning before, a substantial number of patients to be treated in 2023. But even by then, I don't expect to have, like, all the patients identified. What we expect to see is about, for example, 100 new patients per year in the U.S. And as we move towards, like, 2022, 2023, that's -- the incident patient population is going to be more obvious, and you're going to start to get those patients who are normally "lost." I think it's a great day to discuss this, right? It's Rare Disease Day. Rare diseases are so underdiagnosed globally, and there is so much push now to include genotyping panels and so on. So in the space of, I would say, 5 to 7 years, you would expect to have a very good number -- percent of those patients, and the majority of the incident patients. So it's going to be a substantial market, the way we see it.

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Stuart W. Peltz, PTC Therapeutics, Inc. - Co-Founder, CEO & Executive Director [49]

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And we're working hard to make sure we've got a strong launch upon approval so the patients are lined up and ready to . . .

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Marcio Souza, PTC Therapeutics, Inc. - COO [50]

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To roll, yes.

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Stuart W. Peltz, PTC Therapeutics, Inc. - Co-Founder, CEO & Executive Director [51]

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To roll.

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Tazeen Ahmad, BofA Merrill Lynch, Research Division - VP [52]

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Okay. And if I might squeeze a last question in really quickly, on your oncology portfolio, you are doing a study in AML with PTC299; can you just give us an idea of what type of patients that's going to be in? Within AML?

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Marcio Souza, PTC Therapeutics, Inc. - COO [53]

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Yes, yes, of course. So 299, right, DHODH inhibitor, I think that the body of literature is showing that that's a key pathway for hematologic malignancies in general, but in specific AML, it's been growing. We have some good preclinical and clinical data showing that the mechanism is being activated. It's a 3-plus-3 design with 3 cohorts in refractory relapsed patients. So we are really getting very severe patients. We are starting, as we normally do in trials like this, right, with a dose that's relatively small and subtherapeutic, moving on to true therapeutic-level dose. The -- it is a relatively small -- sorry, slow design, in a sense, so we expect to enroll during the year all cohorts, and to move into an expansion early next year. Depending on what we are seeing, because this is open-label, we are seeing the hematological data for all those patients, we're being able to learn as well the kind of patient we would want on the expansion to maximize the probability of success there. Did that help?

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Operator [54]

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Our next question comes from the line of Alethia Young with Cantor Fitzgerald.

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Alethia Rene Young, Cantor Fitzgerald & Co., Research Division - Head of Healthcare Research [55]

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I guess 2, since I'm going to follow the trend line here. One, can you just talk a little bit more specifically about, with the AADC gene therapy, what's kind of left to work through in the manufacturing? And I mean, do you view that as, like, a big hurdle as far as getting to the finish line with the FDA, or is it just kind of business as usual? And then the second question, as far as SMA, risdiplam readouts over the year, I mean, are we looking at basically every medical meeting we'll have some sort of update on the Part 1 of the study? And just wanted to get -- kind of get a characterization of, like, what data we should expect over the year. Thanks.

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Stuart W. Peltz, PTC Therapeutics, Inc. - Co-Founder, CEO & Executive Director [56]

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Yes. I think if -- thanks, Alethia. For in terms of the second question, first, yes, we'll -- obviously the next meeting is AAN, and we'll be having a full presentation of the results then, so we're excited to be there for that, and we'll do it at the upcoming meetings that continue on after that as well. So we'll continue to update you on that as well. In terms of the second question -- AADC manufacturing, why don't you?

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Marcio Souza, PTC Therapeutics, Inc. - COO [57]

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Yes, yes, let me give -- so I'll -- hey, Alethia. Business as usual and manufacturing for gene therapy, I don't think, belong on the same sentence these days, right? So I'm going to -- I'm not going to be cocky there and say that it is. We take extremely seriously all the feedback we got from the FDA. We've been pleased and blessed for having the interactions we had with them and get the guidance on the type C meeting we had late last year. We're following all these steps. [MBL], that is our CDMO, has been great to work with, giving a lot of attention to everything. And right now, all these steps that we have to move to get to the BLA later this year are occurring. And hopefully, and [Neal] and his team are working extremely hard to get there -- everything is going to continue in place. We'll continue to expand. Part of what is a consideration for us here is obviously we want to have a successful launch, so we have to have several commercial batches manufactured to that commercial launch, so we're looking to that capacity. We're looking to continue to add the material. And I know we discussed this in one of our calls, but just for the broader group's view, we had 2 manufacturing CDMOs before, both in the United States, that did a clinical trial product. So we are able to use that process at MBL on the commercial, and now we are in the late stage of implementing the methods and everything else the FDA discussed with us for a BLA later this year, as I mentioned.

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Stuart W. Peltz, PTC Therapeutics, Inc. - Co-Founder, CEO & Executive Director [58]

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Yes, and the -- from a -- just to remind everybody as well, it's one of the advantages of what we're doing, is it's [stereotactic] surgery, so the level that we need -- and this is one of the reasons, when we went with Agilis, is that the requirement of manufacturing is much smaller. We're around the 2x10(11) viral particles total, so it's -- well, like Marcio said, you never take it for granted. They're different -- in terms of what other programs are, it's a different level of what you need for -- to manufacture.

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Operator [59]

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And our next question comes from the line of Raju Prasad with William Blair.

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Raju Yashaswi Prasad, William Blair & Company L.L.C., Research Division - Senior Research Analyst [60]

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Just a quick clarification on something you previously said, Stu. The NDA submission is only going to be based on Part 1 of SUNFISH and FIREFISH? And I guess just that first. Then I have a follow-up.

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Stuart W. Peltz, PTC Therapeutics, Inc. - Co-Founder, CEO & Executive Director [61]

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Yes, that's right. That -- it was based on our interactions with the FDA where they saw the results of both SUNFISH and FIREFISH, that we're submitting based on that.

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Raju Yashaswi Prasad, William Blair & Company L.L.C., Research Division - Senior Research Analyst [62]

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Okay. And I guess, was there any amount of time that they wanted to see those patients out before you could submit, or is it just when the CMC package and the rest of the package is put together that you can submit it?

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Stuart W. Peltz, PTC Therapeutics, Inc. - Co-Founder, CEO & Executive Director [63]

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Yes. When we're putting -- we're going to -- our plan is to put together the package and have it submitted this year, later this year. So it's really based on that, that -- and most of this is just getting ready to be able submit the NDA. So that's what that's more about, not about it longer-term, necessarily.

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Raju Yashaswi Prasad, William Blair & Company L.L.C., Research Division - Senior Research Analyst [64]

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Okay. I guess what would -- if the Part 2 of the study didn't show 5 patients, what would the -- were there any discussions there, or how is Part 2 going to factor into kind of regulatory discussions moving forward?

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Marcio Souza, PTC Therapeutics, Inc. - COO [65]

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So, Raju, basically, the conversation with the FDA was centered under, like, in -- and some of the data they've seen has not been public, because this was at the different time points than the one we showed at WMS. And based on that, I believe that that's the interpretation of the feedback we got from them, is that they're looking to proportions, right? We always use absolute numbers, but the proper way to look into this is like, for Part 2, FIREFISH, there was 12.5% of the babies sitting. What we are looking to right now, if you were to apply the proportion to Part 1, that's about 2.5 babies, right? So 3 babies would be to that threshold. When they look into this, they're like, look, that's robust data, and the (inaudible) and a few other things. With the SUNFISH, it's similar, right? We're seeing patients that normally decline on the MFM, 32 base, on the natural history, increasing.

So the package, and we never got definitive answers before a full review from any regulatory agency, but based on the briefing book data they received, they're like, this looks sufficient for the file. The -- both trials completed 1 year or more of exposure, right now for Part 1, so it meets the normal, I would say, noncodified in the statutes, but normally used threshold of 1 year of exposure, 100 patients, a year of exposure, things like that. So Roche feels very confident. We feel very confident with the package. And it's now more procedural documents that have to be put in place for an upcoming submission.

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Raju Yashaswi Prasad, William Blair & Company L.L.C., Research Division - Senior Research Analyst [66]

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Great, that's helpful. And then just a clarification question on the milestone payments. I think it says $400 million in the press release. Approximately what percent of those are regulatory and -- in milestone versus set milestone payments?

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Marcio Souza, PTC Therapeutics, Inc. - COO [67]

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So we posted some slides today in the -- our SEC filings, in our slides. There's a very detailed schedule of all the payments. But maybe, Christine, you want to, like, go over it?

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Christine Utter, PTC Therapeutics, Inc. - Principal Financial Officer & Treasurer [68]

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Yes. So there's about $87 million related to regulatory milestones, and the remaining are sales-based milestones. And as Marcio said, all the details, as well as the tiered royalties, are also in those schedules.

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Raju Yashaswi Prasad, William Blair & Company L.L.C., Research Division - Senior Research Analyst [69]

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Great. And then one question on AADC. Any color you can provide on the U.S. versus ex-U. S. split between those 100 patients?

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Marcio Souza, PTC Therapeutics, Inc. - COO [70]

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Sure, happy to. So it's about half and half right now, like, give and taken. So the -- as I mentioned before, right, we're putting effort on both regions. The -- in Europe it's relatively easy. If physicians request the kits, they pass this, so we're probably going to see a little bit of an uptake in Europe on the next couple of months, potentially. I don't know because we haven't run, but potentially faster than the U.S. But it's equal right now. We expect that this is pandemic, so don't expect to have a concentration in one region or another. Obviously putting a lot of emphasis to get all the screening up and running in the U.S., because the U.S. is our first launch -- it's the most important pharmaceutical market in the world, you want it to be very successful here.

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Raju Yashaswi Prasad, William Blair & Company L.L.C., Research Division - Senior Research Analyst [71]

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Great. And then just quick last one. What's the age range between those patients?

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Marcio Souza, PTC Therapeutics, Inc. - COO [72]

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They are -- right now, the information we have, we don't age for all of them, but they are within the age that were exposed to the treatment before.

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Operator [73]

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And our next question comes from the line of Brian Abrahams with RBC Capital Markets.

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Brian Corey Abrahams, RBC Capital Markets, LLC, Research Division - Senior Analyst [74]

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My first question is on the AADC program. Among the patients you identified, can you talk about what proportion we should be thinking about as being eligible for treatment with the gene therapy? Would all of them be eligible, or just a small part?

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Stuart W. Peltz, PTC Therapeutics, Inc. - Co-Founder, CEO & Executive Director [75]

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Yes. Yes, no, thanks, Brian, for the question. What we've seen is that -- and what we anticipate is that all patients would be eligible for the gene therapy treatment. We've seen -- actually, and it's interesting, regardless of age, we've seen improvement in all the patients thus far.

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Brian Corey Abrahams, RBC Capital Markets, LLC, Research Division - Senior Analyst [76]

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Got it. And then bigger picture, just a little bit more about your plans to build out the in-house gene therapy franchise, with respect to what type of manufacturing platform you plan to use? What's the progress been like to date? Any specific timeline goals or milestones we should be thinking about? And potential associated costs there, as you build out that emerging franchise?

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Stuart W. Peltz, PTC Therapeutics, Inc. - Co-Founder, CEO & Executive Director [77]

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Yes. Yes, thanks for that. I think on -- one point is, we're going to have our own internal gene therapy group building out the vectors and things like that as well. And then I think as we talked about in terms of -- we thought that it's important to control the manufacturing ourselves, and we've been working hard to be able to bring it in-house, and I think probably within the next 60, 90 days, we'll have probably more to talk about then.

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Brian Corey Abrahams, RBC Capital Markets, LLC, Research Division - Senior Analyst [78]

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Okay, fair enough. And then last one from me. Curious your latest views on use of capital for continued external business development versus investing in the internal pipeline and other capabilities like manufacturing. Where do you stand? What's your latest thoughts?

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Stuart W. Peltz, PTC Therapeutics, Inc. - Co-Founder, CEO & Executive Director [79]

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So I'll start, and then you add to -- have Christine -- our view is, obviously, we're, as we've talked about in the vision of what we'll be doing, we'll be expanding both our research and clinical programs internally, and so we said we'll be -- as we said in our vision of what we would be. And so we're doing that as well, but we've always said we'll continue to look externally as well. Obviously you can't do everything in-house, and so we'll continue the search and try and see if there's other things that are a compatible fit for us. In terms of capital, maybe, Christine, you want to?

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Christine Utter, PTC Therapeutics, Inc. - Principal Financial Officer & Treasurer [80]

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Yes. Just to remind you, we had $227 million in cash at year-end. We recently completed the equity offering of the pipeline, $225 million. So with our solid revenue base and our diversified pipeline, we'll continue to invest both internally and also continue to look externally, what fits our strategic vision.

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Operator [81]

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And our next question comes from the line of Martin Auster with Crédit Suisse.

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Mark William Connolly, Crédit Suisse AG, Research Division - Research Analyst [82]

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This is Mark on for Marty. Thanks for taking my questions. I guess looking at risdiplam, I have a couple of questions on that particular product. When should we expect to see functional data for the JEWELFISH study? And can you outline what specific data you may provide? And similarly, when should we expect to see initial RAINBOWFISH results? And what do you expect the initial data out-take to entail? Thank you.

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Stuart W. Peltz, PTC Therapeutics, Inc. - Co-Founder, CEO & Executive Director [83]

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I don't know if we've -- so the -- I guess a couple of points, is the -- one is the JEWELFISH has been expanded to include patients that are treated with gene therapy. And so while that's relatively new, we would probably have data at the Cure SMA meeting, is probably when we would be talking more about that.

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Marcio Souza, PTC Therapeutics, Inc. - COO [84]

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Yes. And maybe, if I may, Stu, so JEWELFISH is a safety trial at its core. Obviously there are some measures there. We are looking into the PK and the exposure, protein levels. We showed some of that data at WMS, which was extremely positive, in my view, when you're seeing patients switching from other therapies and having an increase in sustainable levels of SMN protein. So that's going to continue. Cure SMA is the next expected, because the cohort was just expanded to include other type of treatments. RAINBOWFISH is only beginning of enrollment, so not quite ready to give, like, an estimate on when that's going to be reading out, but as you can imagine, there's a lot of interest with physicians being more and more confident with risdiplam on treating for symptomatic patients with it.

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Operator [85]

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Thank you. And that concludes our question-and-answer portion. I would now like to turn the call back over to CEO, Mr. Stuart Peltz, for closing remarks.

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Stuart W. Peltz, PTC Therapeutics, Inc. - Co-Founder, CEO & Executive Director [86]

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Thank you. So as -- and thank you all for joining the call today, and as you know, today is Rare Disease Day, and I think it's an important reminder of what drives us here at PTC, where we're focused on rare disease, really, every day.

And as you've seen, after a transformative 2018, we're now really now executing on our vision to be a leading company with a diversified portfolio on the scientific cutting edge to treat rare genetic disorders. With an expected BLA for gene therapy treatment of AADC and a planned NDA for spinal muscular atrophy treatment, both this year, we'll be advancing towards this goal. We look forward to delivering on the 5-year plan to bring innovative products that make a significant difference in patients' lives and enable us to achieve the potential $1.5-billion revenues by 2023.

Thank you all for joining the call today.

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Operator [87]

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