PTC Therapeutics Inc

NASDAQ:PTCT

Earnings Call Transcript 2018-Q2

[Operator]

Good day, ladies and gentlemen, and welcome to the PTC's Second Quarter Financial Results and Corporate Update. At this time, all participants are in a listen-only mode. Later, we will conduct a question-and-answer session and instructions will be given at that time. [Operator Instructions] As a reminder, this call is being recorded.

I would now like to turn the conference over to Emily Hill, Head of Investor Relations. Ma'am you may begin.

[Emily Hill, Head of Investor Relations]

Hello, good afternoon, and thank you for joining us to discuss our 2018 second quarter corporate update 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 slide on our simultaneous presentation which contains our forward-looking statements. Our actual results could materially from those 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 recently quarterly report on Form 10-Q and 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 CEO, Stuart Peltz.

[Stu Peltz, Chief Executive Officer]

Thanks, Emily. And thanks for joining us on this afternoon. Joining me on the call today are Marcio and Christine. Marcio will provide the commercial and clinical development update. Christine will end the call with a review of our financials.

The first half of 2018 has clearly been transformative for PTC. As I outlined at our 20th Anniversary Analyst Day we have a strategy to build out PTC as a rare disease leading biotech company where we provided a three to five-year plan. I hope you can see we have aggressively been executing on that strategy. I'm incredibly proud of my team. Taking the outline of the strategic vision and aggressively executively on it, in the first half of this year.

We continue to successfully grow our Duchenne franchise. In event our splicing franchising and niche oncology pipeline. And most recently we've announced two important transactions that strengthened our pipeline with late stage and commercial asset. Let me discuss each of those now. Most recently we have announced an important agreement with Akcea which allows us to commercialize two rare diseases drug in Latin America. Tegsedi and Waylivra. Tegsedi has been approved by the European Commission for treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). The approval by the European Commission is important as is critical in Latin America. The polyneuropathic form of (hATTR) which Tegsedi has indicated for occurred more frequently in individuals of Portuguese ancestry because Latin America and in particular Brazil contains a large portion of polyneuropathic (hATTR) patient. The region is strategically important one for Tegsedi.

Tegsedi is also under review by the United States FDA with a PDUFA of October 6. The second product Waylivra is under regulatory review in the United States and Europe for the treatment of people with familial chylomicronemia syndrome (FCS). With a PDUFA coming up later this month on August 30. This collaboration reflects a strategic initiative to leverage PTC's global infrastructure and our successes in bringing innovative drugs the patients in Latin America. We're eager to bring these important therapies to patients in these regions as quickly as possible.

The next transaction I'll review the Agilis acquisition. Puts us on the forefront of the gene therapy. On July 19, when we announced our agreement to acquire Agilis Biotherapeutic. The Agilis gene therapy platform for treating orphan monogenic CNS disorders is an ideal fit with our strategy of building out a leading rare disease biotech company. This transaction places us in a leading position in the gene therapy space for rare diseases including a late stage CNS gene therapy program. We planned to file a BLA in 2019 for the lead program for the treatment of AADC deficiency. We're very excited by the lead asset as well as the technology behind the platform.

I'll ask Marcio to elaborate more on these transactions later in the call. Our DMD franchise was strengthened in the first half of this year on several fronts. The EMA's approved a label expansion for Translarna in nonsense mutation Duchenne muscular dystrophy patients ages two to five years olds. DMD is a degenerative disease. Treating patients earlier allows for better preservation of muscle function. Importantly, this label expansions allows patients in countries that recognize EMA approval to gain access to the therapy at a younger age.

Our DMD franchise also includes in Emflaza, for all United DMD patients over age five. We're working hard to establish Emflaza as the standard-of-care in the United States. Recent DMD guidelines reinforce this effort. The guidelines state that Emflaza should be initiated for all DMD patients as early as possible. Our commercial execution has led to strong year-over-year revenue growth. In the second quarter 2018, our revenue for the DMD franchise totaled approximately $68 million. Our guidance for full year 2018 remains $260 million to $295 million.

Beyond the current growth opportunity for Translarna outside the United States we're also looking to expand and bring Translarna to US patients. We plan to initiate a dystrophin study this year to submit to the FDA next year for potential United States accelerated approval of Translarna. Let me now turn to our internal sciences research programs and our pipeline. I'll start with our internally developed splicing platform. We have an oral small molecule as a treatment for SMA advancing in the clinic. This program is currently in pivotal studies, in partnership with Roche and the SMA Foundation. The ongoing pivotal studies include FIREFISH, for Type 1 SMA infants and SUNFISH for Type 2 and 3 SMA patients.

Encouraging interim data from the dose finding portions of both studies were recently presented at the Cure, SMA meeting in Dallas. During the clinical presentation of the FIREFISH dose finding study a video was shown of three babies sitting independently. I'll provide more details on both studies later in the call. The success in our SMA program have validated our splicing platform which was expand and to include Huntington's disease and Familial Dysautonomia. These are progressing and are currently in lead optimization stages. We expect both programs to advance into the clinic by 2020.

Let me now pass the call over to Marcio to update you on our clinical and commercial efforts. Marcio?

[Marcio Souza, Chief Operating Officer]

As Stu just mentioned, we continued for advanced toward our objective for the DMD franchise in 2018 increasing the use of Translarna globally and transforming Emflaza into standard-of-care in the United States. We are pleased with EMA Translarna label expansion to include patients aged two to five years old. The launch process has now started and I'm optimistic about the additional patients to retrieve Translarna in this expanded patient population.

We have been working very hard to help the community [ph] in the age of diagnose for DMD. Before launch of Translarna four years ago the average age if diagnose was around six to eight years old depending on the country. In most geographies where we operate, the age has been reduced by at least six months and as much as two years. We continue to invest in improving disease awareness, patient identified and general splicing to further advance these agenda. We're committed to continue to drive all the diagnose efforts to improve patients outcome.

We have a similar commitment to improve patient outcomes with Emflaza. We're marching forward to establish Emflaza as a standard of care for DMD patients in the US. I'm happy to report the first wave of the transitioning the plaza cord [ph] experience patients to commercial therapy is complete. We're now expanding the Emflaza us to the segment of patients who have been previously or are currently on prednisone. The second phase has been lower than expected. Conversion on the payer side is happening, but has been more time consuming than we previously anticipated mostly due to the doctor's office burden of paper work and processing.

We have a number of key initiatives underway to ensure Emflaza up stakes continuous and accelerated. This includes improving administrative supports and continuing to remove barriers with payers. Based on increased information around Emflaza's benefit. Two important publications demonstrating the effect of Emflaza with prednisone have recently been published. We've just announced an important publication in [indiscernible] comparing the efficacy end phase of the plazacore [ph] in prednisone from the placebo arm of XNG [ph] study.

The results demonstrated a clinically differentiated benefit of the plazacore [ph] over prednisone in the slowing disease progression as measured using physical function endpoints and the time to delayed of plaza [ph]. Reaching naïve patients is yet another critical step in our long-term strategy. In the United States about half of the DMD patients are still not currently seated with corticosteroid treatment. The current DMD treatment guidelines recommends starting Emflaza treatment upon diagnosis. We have a long-term strategy to penetrate the segment of untreated DMD patients as well as to expand the label to address the youngest patients aged two to five.

Our highly differentiated DMD franchise has been growing year-over-year. In the first half of 2018 the combined franchise reported revenue growth of 67% versus the same periods in the prior year. We're reiterating our expected guidance for the full year of 2018 of $260 million to $295 million. The global commercial Translarna business and Emflaza positions has diversified our product portfolio revenue and geographic footprint. Positioning PTC as an outstanding rare disease BD partner. We have now demonstrated our ability to integrate and rapidly launch products for rare disease.

In line with our strategy we have chosen from formative transactions in the past month. Most recently we announced collaboration effect Akcea to commercialize two rare disease drugs in Latin America. Tegsedi and Waylivra. For our commercialization efforts with Translarna we have established a strong presence in key countries including Brazil, Argentina, Chile and Colombia. Our patient identification efforts have resulted in strong year-over-year growth in these countries. Tegsedi has received marketing authorization approval from European Commission for the treatment of stage 1 or 2 polyneuropathy in adult patients with (ATTR). ATTR is a devastating disease with limited treatment options.

As Stu said, Latin America and in particular Brazil contains many such patients, so the region is strategically important one for Tegsedi. We anticipate Brazil to be one of the largest polyneuropathy ATTR markets worldwide. In Latin America, we understand to be more than 6,000 patients eligible for treatment. Our team is working hard with the Akcea, Ionis teams to execute on a robust regulatory and go-to-market strategies for the region.

We are uniquely positioned from a competitive standpoint to execute in Latin America. The second product Waylivra is under regulatory review in the US and Europe for the treatment of FCS. Waylivra has the potential to be the first and only therapy indicated for FCS. We expect the market opportunity for Waylivra to have the potential to be similar to one of Translarna in Latin America. FCS has been starting to [ph] misdiagnosed and we expect patient identification to be the gaining factor for launch. FCS is caused by impaired function of the enzyme lipoprotein lipase and is characterized by a risk of unpredictable and potentially fatal acute pancreatitis. We are excited with the opportunity to bring this life changing drugs to patients.

Let me now turn to the Agilis acquisition we recently announced. Earlier last month, we announced the planned acquisition of Agilis Biotherapeutics. The acquisition bring us four genetherapy programs at various stages of development all focused on trading CNS disorders. Starting with late stage program in AADC deficiency for which we plan to submit a BLA, in 2019. This is caused by programs [ph] including Friedreich's ataxia and Angelman syndrome as well as other cognitive disorders. This acquisition as current genetherapy platform and it strengthen our pipeline adding both late and early stage gene therapy programs, which we are eager to bring to patients.

Many of these gene therapy programs will benefit from the advantage of targeted micro-dosing. Directly to specific structures in the CNS. Micro-dosing of the vital factors bring many advantages because that allows for small scale manufacturing, reduced risk of menogenisty [ph] while maximizing efficacy in the CNS. This transaction aligned with our strategic priorities bring in late stage therapies for rare disease and leveraging our global infrastructure.

We are confident that we're well equipped to drive value with both strong commercial and clinical capabilities and a continued focus on transforming wise of patients with rare disorders. I would now hand the call back to Stu. Stuart?

[Stu Peltz, Chief Executive Officer]

Thanks Marcio. I'd like to share more detail on data recently generated in our SMA program. Most of you are familiar with a program which is based on our small molecule splicing platform. This technology has been used to discovery potential therapies for spinal muscle atrophy or SMA. A rare genetic neuro muscular disorder that generally manifests early in life and is the leading genetic cause of death in infants and toddlers.

We have a robust program in collaboration with Roche and the SMA Foundation around oral SMA to splicing modifiers. We believe that an oral systemic therapy provides a competitive advantage because of its broad tissue distribution and ease of administration. Earlier clinical data has been shown that RG7916, drives SMN2 splicing towards a complete restoration of full length SMN2 messenger RNA. This program is currently in pivotal stages with two registrational studies. FIREFISH for Type 1 infants and SUNFISH for Type 2 and 3 patients.

The JEWELFISH study while not registrational is also enrolling patients from other splicing therapies. Data from JEWELFISH and from the dose finding portion of FIREFISH and SUNFISH were presented at the June at the Cure SMA meeting in Dallas demonstrating sustained benefit and safety and tolerability. Data from the 21 babies in the dose finding portion of FIREFISH highlighted continued survival. During the presentation the investigator of the study showed a video of these babies demonstrating improved motor function including head control, rolling from supine in sitting [ph]. Three babies were depicted sitting independently.

The first baby received their first dose 6.8 months of age and was depicted sitting at week 35. The second baby received their first dose at four months of age and was depicted sitting at week 26. The third baby was first dosed at five months and to - sitting at week 26. Notably the last baby was also depicted riding at Dragon Ride-on toy across the hospital. Previously published national history data indicate that the meeting age of the of densely [indiscernible] for SMA type 1 events to be between eight and 10.5 months.

Of the 20 months baby presented already, 13 have surpassed the national history expectation of 10.5 months survival. It was also noted no babies have required a tracheostomy or permanent ventilation since study initiation and no baby have lost the ability to swallow. The meeting age of the first dose was 6.7 months and babies have received RG7916 for a duration of up to 24 months. As of June presentation, RG7916 has been well tolerated at all dose levels and there have been no drug related safety findings even through withdrawal.

Two tests were previously reported to disease progression and were determined not to be drug related. The FIREFISH dose finding study is now complete and all babies have been transitioned to the therapeutic dose for an extension trial. Ongoing data presentation from this trial are expected throughout 2018 including additional survival data shopping [ph] 10 score and other motor milestone. We look forward to share with you these developing data with you. The next anticipated medical meeting presentation is at the World Muscle Society Congress in early October. Data on additional sitting SMA babies will be shared at this point.

Based on reports of an increasing number of sitting babies. We and the collaborators believe there's a potential to file an NDA in 2019. Recruitment is ongoing globally for the pivotal registrational portion of the FIREFISH and SUNFISH studies and enrollment is expected to complete this year. The SUNFISH is a blinded study with a primary endpoint of motor function at 12 months. The primary endpoint of the open label FIREFISH study is a percentage of instance for sitting without support at 12-month of treatment as discussed by the Bayley Infant Scale.

The SMA program is not only progressing towards and oral and systemic treatment for SMA patient. It also validates that our splicing platform technology can identify selective compounds that modulate pre-mRNA splicing. We are now applying our expertise to other challenging diseases with high unmet medical needs, and have internal preclinical programs. These include programs in Huntington's Disease and Familial Dysautonomia. We expect both programs to enter the clinic by 2020. We are very excited about our splicing programs and cover them in-depth at our Analyst Day. And I refer you to those slides on our website for full details.

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

[Christine Utter, Principal Financial Officer and Treasurer]

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

Starting with our top line results, we reported strong performance across our DMD franchise. As Stu mentioned, our results for second quarter of 2018 were total net product revenue of $68.2 million compared to $47.9 million in the second quarter of 2017. Based on where we stand today, we are reiterating our 2018 revenue guidance of $260 million to $295 million. Translarna net product revenues were $47.8 million for the second quarter of 2018, representing 4% growth over the prior period. Mostly as a result of increased penetration in existing geographies. The 4% growth rate reflects the uneven ordering patterns from Latin America which made quarter-over-quarter comparison last representative of underlying growth.

Our Translarna guidance contemplates this lumpiness and reflects the total patient demand for the year. Emflaza reported net product revenues of $20.3 million in the second quarter of 2018. Non-GAAP R&D expenses were $28.7 million for the second quarter of 2018 excluding $3.9 million in non-cash stock-based compensation expense compared to $26.9 million for the same period in 2017 excluding $3.9 million in non-cash stock-based compensation expense. This increased an R&D expense to reflect increased investment in our research program and the advancement of our clinical pipeline.

Non-GAAP SG&A expenses were $29.4 million for the second quarter of 2018 excluding $4.1 million and non-cash stock-based compensation expense compared to $24.9 million for the same period in 2017 excluding $4 million in non-cash stock-based compensation expense. Reflecting continued investment in commercial activities to support our DMD franchise.

Net loss for the second quarter of 2018 was $9.5 million compared to a net loss of $17.5 million for the same period in 2017. This decline in net loss reflects our growing revenue base as we continue to leverage our successful international corporate infrastructure. Cash, cash equivalents and marketable securities totaled approximately $296 million at June 30, 2018 compared to $191 million at yearend 2017.

We are proud of the transactions we just announced in the last month. As we close on these transactions and move forward with integration. We will provide update to our expected spend for the year. I will now hand the call over to the operator to start our question-and-answer session. Operator?

[Operator]

[Operator Instructions] and our first question comes from Brian Abrahams with RBC Capital Markets.

[Unidentified Analyst]

This is actually Rick on the line for Brian. Thanks for taking our questions and congrats on all the progress. First I just wanted to follow-up Stu on your comments about the potential for accelerated approval of RG7916. Based on the data emerging out of FIREFISH, what is your current understanding on what the bar for the potential for accelerated approval is? Is there a threshold number of babies achieving the sitting up in a supine milestone, you think that would be supportive of the current rate approval? Or are there any other motor milestones you think could play into accelerated approval?

[Stu Peltz, Chief Executive Officer]

Thanks for the question and we think that's an important question and maybe as you might remember what we reported at the Cure SMA meeting where we saw a number of babies be able to or have development milestones that included being able to raise the head, being able to turn over as well as being able to sitting. Where sitting is the endpoint for the trial and in that, in the part 1 of that where there were 21 babies within that trial. We had reported at the Cure SMA meeting in June that there were three babies that were able to be sitting, so we thought that was actually really quite important. Overtime now, what we're saying is that there are as a consequence of more time passing that there are additional babies sitting since that time on top of the three that we had and so we wanted at this point and probably you've heard from before, we're –Roche has disclosed last week that there is potential filing in 2019 and I think, that just reflects the potential to file for that - reflects that the increased number of babies sitting that would probably be presented at the World Muscle Meeting at that time. But what we wanted to do here was really reflect that the increasing number of sitting [indiscernible] since that is the endpoint that we're seeing increased numbers.

And I think we've always said before that five out of 40 would be statistically significant in the part 2 of the trial, seeing additional sitters in the part, in this part just gives us more confidence for potential. So I think it's obviously going to require additional conversation with regulators so that they look at both safety and efficacy for potential filing, but just the fact that we continue to see such exciting results make us think about how to move business rapidly as possibly for patients.

[Unidentified Analyst]

Okay, that's great. That's really helpful. I have a follow-up, so assuming positive results from the FIREFISH study and type 1 patients. What kind of implications would that have for a potential expedited approval pathway in type 2 and type 3 SMA patients? I know since they all have a same underlying genetic defect but the progression of disease in type 1 SMA is much quicker. Is there the potential for some sort of submission package where you could maybe combine the achievement of motor milestone data from type 1 patients with biomarker and safety data from type 2 and 3 patients?

[Stu Peltz, Chief Executive Officer]

Yes I think that's again a really good question that would require additional conversations with the regulators to see how they would think about as in terms of knowing what you see. In type 1 and extrapolating to type 2 others, which is not uncommon in the orphan disease space. So it's certainly a conversation we would have and we have an ongoing SUNFISH trial that certainly would then be continuing on as well. Did you?

[Marcio Souza, Chief Operating Officer]

Maybe just to add a little bit right. So SUNFISH our trial for type 2 and 3 as we mentioned before we expect this trials to be enrolled relatively assume by the end of the year. So we see that both part 1 in SUNFISH where we collect like safe data and the sensing dose and now this people tell phase for SUNFISH are going to be fundamental. So while they are expecting of the disease they fundamental different in terms of how they progress but the program is so comprehensive and there is so much data in terms of like the number of patients that being, the exposure, our undertaking of the PK and PG relationships and so on that. We believe it's by far the one development most advance in terms of, just comprehensive and large it is. So that gives us confidence, that if you were to find up that forward with the regulators you file for type 1, we could follow shortly thereafter with type 2, 3 already been altogether. So we stay tuned and we're going to keep you guys updated.

[Unidentified Analyst]

Great. That's it from me. Thanks for taking the question.

[Operator]

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

[Unidentified Analyst]

This is Sean calling in for Joel. Congrats on such busy and [indiscernible] quarter, so I've got a few questions. Kind of had follow-up on SMA. I think we're going to see it kind of your first, I think a few data World Muscle Society for SUNFISH. Can you just review what are the most endpoints that we should be looking for us?

[Stu Peltz, Chief Executive Officer]

Yes thanks for that. So SUNFISH that's for part 1 where we've in the past showed both the RNA as well as protein levels and overtime preferably we're measuring MFM and others as well. So it's obviously it's open label there, but we'll be looking at the MFM scores and things like that as well.

[Unidentified Analyst]

Excellent and then I just have two follow-up questions on hATTR program. Can you just briefly describe any work you've done to give us confidence and the estimate 6,000 patient population and if any out there?

[Stu Peltz, Chief Executive Officer]

Sure. Yes I think, there's been a lot of prevalence and due diligence in look at this. Marcio, why don't you go through a little bit on, what we've done?

[Marcio Souza, Chief Operating Officer]

Absolutely like as you might know when we mentioned like during the press release last week right in the collaboration, so let me first say how excited we are, we think that Akcea and Ionis are incredible partners like their cooperation so far has been tremendous and there was a lot of data. They have collected previously during the due diligence in the last several months, we managed to follow the key opinion leaders in the country. And we look into both primary research and secondary research but also we have a network of labs that we've been working on in Latin America that collects genetic information the immunological information. So we rely on all of that and considering just how large the ancestry, background in terms of like [indiscernible] and mutation in Brazil alone for example it is, we feel very confident on the number that we provided is about 6,000 patients in the region. It is mostly concentrated in the South of South America, so Brazil and Argentina for example are the two most important countries in that region, but we see the potential across the region just if I slide the different genetic background in the other countries.

[Stu Peltz, Chief Executive Officer]

But we feel good about the notion that well we think there's around 10,000 to 15,000 patients with the polyneuropathy that really this area gives between a third and half of those patients and we feel pretty comfortable that this is a really important valuable area for the drug.

[Unidentified Analyst]

Thank you for that and I caught event and just with a brief follow-up. Can you talk a little bit on your strategy to best identify these patients considering it is such a heterogeneous disease?

[Marcio Souza, Chief Operating Officer]

Yes, absolutely. So we're doing a lot of things already and part of the joint development and diligence Akcea Group, right we exchanged a lot of ideas. I'm going to updating all of you like in future quarters in terms of what we're doing. But one of the key things is really mapping this patients to have the clinical diagnose but not the genetic diagnose to the sites. We know where these patients are concentrated right now. We believe that there are areas outside of the main centers that are being historically treating HDTR our patients where there is large boost, we're focusing on those large boosts having the team already presence in all these countries make a huge difference. Right we have medical directors and MSLs and like a field team that is really like going to all these accounts that is a fairly large overlap in terms of neurologist with the accounts that we're being already developing relationships or have relationships for in terms for Translarna so it's kind of a perfect fit. We're going to be giving updates, now in a sense is a competitive space and we believe that we have a competitive advantage here.

So we're not going to be sharing as much as we shared before in relations to Translarna since we believe we are ahead at least few years ahead of the competitors here and we want to make sure ,we remain so.

[Unidentified Analyst]

Excellent. Thank you so much guys. Appreciated.

[Operator]

Thank you. Our next question comes from Gena Wang with Barclays.

[Gena Wang, Barclays]

I'm just wondering, I think in the press release you also comment the five-year net revenue growth rate 15% compound annual growth rate in 2022. So that translate into I think roughly $350 million in 2022, just wondering what are the assumptions for this $350 million in 2022 and do you think that are you being looked at conservative in terms of that assumption?

[Marcio Souza, Chief Operating Officer]

Gena, thank you so much for the question. The way we look into guidance and I mentioned this before right in our future guidance as well. It's always in terms of like the midpoint or so or what we're expecting, so there's a number of initiatives that we're doing to get that 15%. One; we just wanted to highlighted we just got the future five label rectified by the C so that alone in total of the population we're talking about a very large number of patients. I mentioned in my prepared remarks as you probably saw, a lot of those patients are not identified yet, but I'm extremely happy with the progress that was done in all the countries operating in terms of identify this patients general type in reducing. So a lot of our assumptions are both in identification and conversion, but they don't defer much in terms of what we're seeing right now in terms of the trajectories. There are few territories that we're going to be adding and increasing the penetration we mentioned that before specifically like Latin America where we just discussed recently our presence there, we intensified the presence. We feel the deal with Akcea are very synergistic on that regards, it's going to be put more people in the ground, more efforts it's going to help with Translarna as well and the Middle East vision as well.

So its multi-prong, in a sense approach but patient identification, expansion of the label and this new geographies are going to be key drivers here. The interesting thing that I believe sometimes get lost is even on the key EUC countries where we launched way back 40 years ago. We're still seeing fairly robust growth in the number of patients and identification and so on, what gives us extra confidence on this, long-term.

[Stu Peltz, Chief Executive Officer]

And then, I think your point the good one is, there's potential upsides we have depending on things like we're working now for the non-ambulatory. If you may remember the pulmonary function data which was really quite strong and so, we're in the process of trying to get a label expansion of that and could be considerable upside that could be 40% upside in terms of that. So yes there's potential for greater growth as well.

[Gena Wang, Barclays]

Okay and then just one another also revenue related question Emflaza seems pretty flat. I think you also commented. It seems like the conversion of new patient to the drug happening a little bit slow, just wondering if you could provide. I don't know how much detail you can give but if you can just give a sense how this quarter compared to the last quarter in terms of the new patient growth?

[Stu Peltz, Chief Executive Officer]

Sure. And Marcio, why don't you give a little color on thinking on?

[Marcio Souza, Chief Operating Officer]

Of course, so there's two drivers here. Or may I say three drivers here that are impacting this quarter. So one is, the gross to net is still fairly similar to what we're seeing. Last quarter we mentioned was a little bit higher and we're expecting before, so we have a little bit more on the gross and net adjustments. We're seeing growth in patients both on the prescriptions and on the conversions quarter-over-quarter. But what else happening is, when we launched this last year. We mentioned about our expectations for the conversion between the time we get the prescription in house, to the time the patient gets expense commercial product , to be between three and six months.

In the first quarter, in the second quarter after launch we mentioned as well that we're seeing more on the lower end of that so around the three to four-ish months and now we're seeing on the other ends, more on the six-ish months. So what's happening is, within our expected conversion timelines but it's skewing in a sense towards the six months conversion. So what we're doing right now in a very focused way, as you go patient-by-patient basically and help them reduce that. So we have hundreds of patients right now but which we have prescriptions that we're working with them, we're working with their providers. There is something new for the neuromuscular accounts where now they have multiple products and they never had it. Where they have to manage all those products and some of them are Part D, some of them are Part B so it's a little bit confusing for those accounts, so we're seeing they need a little bit more support than not and we're intensifying that, we're making sure that everything is in place to bring that average now, to where we would like to be and driving. We're seeing some movement already on the last several weeks. We expect that to improve and to get to the end of the year and beyond on where we wanted to be where [indiscernible] pretty much where we were before.

[Stu Peltz, Chief Executive Officer]

And the other thing that's probably worth noting as well is, there's recently been two publications that have been really quite useful that are demonstrating the superiority of Emflaza over prednisone. One was the synergy national history study that showed just how much better Emflaza was over prednisone and then the recent publication of our placebo data of comparing Emflaza to prednisone again showing improvements as well and that's been very helpful in having the position to understand the differences between steroids and then the payers as well. So it's a combination of all of that we're working hard to make sure that it pushes through the large number of prescriptions we already have in the funnel, we're trying to get it through that funnel.

[Gena Wang, Barclays]

Yes, that's very helpful. Thank you.

[Operator]

Thank you. Our next question comes from Tazeen Ahmad with Bank of America.

[Tazeen Ahmad, Bank of America]

Stu I just wanted to get your thoughts about your partnership with Akcea. You are very good at doing ex-US launch as you've demonstrated that very clearly. I just wanted to get a sense about how you're thinking about this particular opportunity with ATTR patients given. I guess unlike some of your over launches it's likely that at some point in time there will be more than one drug option available for this community of people.

[Stu Peltz, Chief Executive Officer]

Sure. Maybe I'll touch on this. I could pass on to Marcio as well. I think this is where we're really excited about this obviously. Its unique and that it's polyneuropathic form of it in that. It's in a way almost I mean some of the positions there knowing more is the Portuguese disease because it really has a unique and for that particular form of it and the fact that it's actually injectable versus infusion is a really major advantage in this area in which there is not a lot of infusion support there as well. So there's not only it's the drug [indiscernible] but the route of administration and the simplicity of it, is going to be a major advantage in that group as well and so that's the work we've done and the due diligence and looking at the deal really suggest that's having it's - the physicians there have looked at the efficacy and really believed that the efficacies of this drug is really quite good and similar, the comparable but the similarities around of, how it's administered gives this a major advantage.

[Marcio Souza, Chief Operating Officer]

And just to complement a little bit of what Stu is saying right. The one key thing here that we look we're exploring several deals and this came to mind, is our ability to compete and interesting on how we did, the team is just going around and understanding how many competitive situation each one of us like myself included all down to my team like [indiscernible] has seen in Latin America and all the way to the ground in all of us like from top to bottom have being on one or more competitive situations on the ground and being very successful on that. So that was a key components. You're absolutely right. We expect this to be competitive. We believe we'll have an advantage for multiple reasons, time is one, the acceptance of the physicians, the interest of the patients for an FC [ph] drug versus having to be on a hospital for example are using steroids before the administration. But very importantly it comes down to people being able to compete on the grounds and we have this thing that's highly motivated to make sure that we're going to make it happen for the TTR patients and make this drug successful.

[Stu Peltz, Chief Executive Officer]

And there's probably one that you should on touch on, that's I think important Marcio that it's a complex system in Brazil requiring license and a little [ph] labs that we've spent the last 12 to 18 months building. You may want to talk a little bit about that as well.

[Marcio Souza, Chief Operating Officer]

Parts of what we believe is the competitive advantage here is just how established we are there. It takes very long time as Stu just mentioned to just be ready. Get all the proper license, make sure we have the right to relationships in place, that everything from compliance perspective as well established considering all the environment Latin America and we checked all the box, we run through several due diligence, we forward theme in our systems and we're very confident we can deliver on that as far as patients. Because at the end of the day that's why we're doing this. Right there are thousands of patients there that needs the drug now and you're going to make sure to provide to them.

[Tazeen Ahmad, Bank of America]

Okay thanks for all that color and I'm sorry if you addressed this earlier, but similar to how you talked about, how long you think it will take for you to address your estimated adjustable gene therapy population. How long do you think it could take you to reach this estimated success and population that you're going for?

[Stu Peltz, Chief Executive Officer]

Yes that really not going to happen overnight and that are differently from the [indiscernible] deficiency that I believe that's the one you're referring to where that is very few patients identified and we're really doing the ground up in terms of the awareness. The biggest different here with hATTR is like, [indiscernible]. If you go to Latin America, specifically Brazil and Argentina have three data patients, have seen a patient have lot of patient or had someone in the family. So it's a little bit different in terms of the awareness. It's mostly making sure they have the correct referral network, they general type being and so on so forth, so we expect that to be faster.

Now at the same time there are much more patients, so logistically and the conversations with the governments and so on are little bit difference than for AADC deficiency, so we don't see one necessarily taking precedent over the other, we're going to put the best effort on both of them, but it is a multi-year process not few months.

[Stu Peltz, Chief Executive Officer]

Yes, the AADC that was more finding those deficiency because and we'll go to cerebral palsy, clinics, severe epilepsy clinics to trying and find a large, where we - is where we think a lot of those patients will be. In this case, as Marcio said is, it's the awareness in these countries is really quite high, it's not about genotype being and getting them onto drug. So we think they're different as with all orphan disease as much as effect it take some time to get to peak.

[Tazeen Ahmad, Bank of America]

Okay, thank you for that color.

[Operator]

Thank you. Our next question comes from Raju Prasad with William Blair.

[Raju Prasad, William Blair]

Can you provide maybe some color on how you expect the ramp for some of the younger patients in the EU on Translarna? Is it something where you've got several commercial sites already available and 20 countries and maybe there will be a little bit of bullish of commercial patients early on and then, as you continue to identify patients kind of slower ramp or is it something where it will take some time to develop and hit peak?

[Marcio Souza, Chief Operating Officer]

Raju thanks for the question. So this is going to take probably a little bit of time we don't see necessarily [indiscernible] off patients pretty much anywhere right. It is as I mentioned before that is very few patients until recently identified on this. We made a lot of progress. I'm very happy with the progress that was made, but now is really getting patients and as I said, there are several rules and some of them are individual patients. Requests that are happening already, all the accounts [indiscernible] have to submit [indiscernible] and we're going through that. But my joint of the countries, we had to wait until the green light for the certification to submit the dossiers. So now we're in that phase where someone's going to be granted faster than others. So it's going to be staggered in a sense launch which we accounted for when we're projecting our CAGR and getting to that point where can maximize it.

There is lot of enthusiasm from the patients and the physicians perspective and that's what gives us confidence that you're going to penetrate this relatively fast, but just the way this system is in Europe where is our priority markets right for this. It takes some time to get this through the system.

[Stu Peltz, Chief Executive Officer]

But - actually let me just also I think kudos to the team because one of the things that Marcio talked about is changing the time of diagnoses which requires not only they do a lot of hard work on disease awareness and genotyping, but to change it in average six months and then some case much as two years is a tremendous efforts in the four or so years that we've doing that. And so that really then got as a consequence, well it's not where we wanted to be, so then are starting to taking that they're picking up earlier and so, we think that's the efforts are sort of paying off on all the hard work that's been done for the four years, so there will be patients - actually we've even known patients or physicians who've decided that when they find younger patients put them on earlier because I think the general notion is, that when they're younger they've more muscle therefore there's more to help them and we think that's going to bear out through as well.

[Raju Prasad, William Blair]

Great and any update on kind of FDA high level discussions with the FDA, on the dystrophin study. I noticed there was, are you guys playing and trying to get favorable study design. But anything you can kind of comment there?

[Stu Peltz, Chief Executive Officer]

Yes I think, what we comment what we've said in the past is that. We anticipate starting the dystrophin study by the end of this year. We'll be completed by the end of next year, where it can then be filed for accelerated approval and I think that we're on track to do that.

[Raju Prasad, William Blair]

Great and then just the last one. Can you just comment on the types of hospitals or specialty centers that you would expect AADC patients in the US to show up at, is there any overlap with some of the other rare diseases that obviously you guys are looking at like SMA or anything of that nature that you can leverage?

[Stu Peltz, Chief Executive Officer]

Sure and Marcio, do you want to talk little about that before?

[Marcio Souza, Chief Operating Officer]

Yes of course, so there are couple of targets that we have met and the Agilis team has done a tremendous job on [indiscernible] before like we're happy with everything they've done and established relationships with key opinion leaders. So the key focus I would say for the next few months is going to be on this cerebral palsy clinics not only in the United States but globally in general by the patient journey mapping that we did and by the work that Agilis did, it seems to be that that's where the most patients are going to be concentrated. So we're going to focus there, but also patients with severe epilepsy or with diagnosed of severe epilepsy, they actually don't have epileptic crisis, these patients are misdiagnosed and you're going to be looking there. I think the biggest overlap that you're starting to see is with some of the efforts that BioMarin is doing to diagnose the patterns, patients. So we've been hearing on some physicians that they're doing like a tremendous job on the identification there and that's starting to raise the awareness of disease and genetic base of some of those CNS disease, so that's helping. There's a little bit of spillover effort there. So nothing specifically there. We've done before they're essentially not SMA very little in terms of the differential diagnose on the neuromuscular that is outside of the United States a good overlap on the treating physicians where in several countries you don't' see the neuromuscular expert taking care of the ENG [ph] but more a general neurologist or pediatric neurologist and then you're going to have a little bit of more overlap. But in the US it's probably the CB clinics and some of the epilepsy centers.

[Raju Prasad, William Blair]

Great thank you.

[Operator]

Thank you. Our next question comes from Martin Elster [ph] with Credit Suisse.

[Unidentified Analyst]

I guess a couple follow ups from some of the earlier questions on RG7916. The part 2 of the FIREFISH study, is your expectation that the data will be reported to the public at a press release or conference once the required number of infant sitting up is achieved or will run till some completion of that study that's kind of time dependent? And then on SUNFISH, I had a question regarding Spinraza the functional benefit of Spinraza seem to kind of separate out from placebo around the nine month endpoint. I was wondering your perspectives about how 7916 functions and it's differences versus Spinraza and benefiting the fact. Do you think that's a reasonable timeframe to see separation. With this drug do you think that there is anything about it that should make us expect something different? Thanks.

[Stu Peltz, Chief Executive Officer]

Martin, thanks for the question. The first part of the question 7916 part 2 of FIREFISH I think that's a good question. And I think a lot will depend on how we think about into the first part of the, obviously the second part of the FIREFISH is a pivotal study so we'll obviously think high before when we talk about. But it is an open label study, so be able to be seen. So we'll have to talking with our partners about what's the best way to start presenting that and putting that in line with what's happening in the first part and how we move forward on that. On the SUNFISH side, I think that's a very interesting question, and it's just going to work quieter sometime, to see I think you even see from some of the earlier parts of FIREFISH data that even some of the patients that are older within the study, you're still seeing improvements faster in the chop and 10 from the patients on RG7916.

In a way that's in line how we taught that might be especially when we look at the pre-clinical data where we were able to show previously that we can actually dose in the animal models much later even in the [indiscernible] and be able to recover that. So we think it's a high effective molecule and I think only time will tell us, how well that - if that will bear out in the type 2 and 3. But we're quite hopeful that will be the case.

[Unidentified Analyst]

Thanks for your thoughts and I appreciate you taking the questions.

[Operator]

Thank you. And I'm not showing any further questions in the queue. I would like to turn the call to Stuart Pelts for his final remarks.

[Stu Peltz, Chief Executive Officer]

Thank you all for joining. Obviously what our vision is to continue to build a fully integrated rare disease biotech company that really leverages the deep scientific expertise then world class commercial capabilities. The programs will obtain through these transactions along with our ongoing DMD splicing and niche oncology franchises. Really does provide I think a number of value creation opportunities over the next two years. So with that, let me again just thank you all for being on the call.

[Operator]

And ladies and gentlemen, thank you for participating in today's program. This concludes the conference and you may all disconnect. Have a wonderful day.