Good day ladies and gentlemen and thank you for your patience. You've joined the PTC Therapeutics 2018 First Quarter And Corporate Update And Financial Results Conference Call. At this time, all participants are in a listen only mode. Later, we will conduct a question and answer session and instructions will be given at that time. As a reminder, this conference may be recorded.
I would now like to turn the call over to your host, Head of Investor Relations, Ms. Emily Hill. Ma'am, you may begin.
Thank you. Hello, good afternoon, and thank you for joining us to discuss our 2018 first quarter 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 I hand the call over to Stewart, I would like to remind you that today we will be making forward looking statements. These statements include all statements other than those of historical facts, including statements concerning financial guidance, our expectations with respect the future commercial availability of and access to Emflaza and Translarna, the timing and outcome of any future resubmission of an NDA for Translarna to the FDA, our future expectations regarding other clinical, regulatory and commercialization matters, including respect potential outcomes and anticipated timelines and anticipated timelines of our SMA collaboration with Roche and the SMA Foundation addressable patient populations for Translarna and Emflaza and the potential success of Translarna for the treatment of NMDMD and Emflaza for the treatment of DMD.
Actual results may differ materially from expressed or implied by forward looking statements as a result of a variety of risk and uncertainties including those related to our commercialization of returns with 3rd party payers for our products in a timely manner, whether and to what extent 3rd party payers impose additional requirements for approving Impa's prescription reimbursement, changes in laws and regulations, our ability to resolve the matters set forth from the denial of our appeals complete response letter we received from the FDA in connection with our NDA for Translarna for the treatment of NMDMD and those risks discussed under the headings forward looking statements in our Form 10 Q for the quarter ended March 31, 2018, and risk factors in our Form 10 Q for the quarter ended March 31, 2018, and in our Form 10 K for the year ended 2017, which is available from the SEC and on our website. Such statements represent our judgment as of today, and PTC undertakes no obligation to publicly update any forward looking statements, except as required by law. We will disclose certain non GAAP information during this call. Information regarding our use of GAAP and non GAAP financial measures and a reconciliation of GAAP to non GAAP available in today's earnings release.
With that, let me pass the call over to Stu.
Thanks, Emily, and thanks for joining us this afternoon. Joining me on the call today are our Chief Operating Officer, Marcio Souza and our Principal Financial Officer, Christine Utter. Marcia will provide the commercial and internal clinical development updates. Christine will end the call with a thorough review of our first quarter results and our strong financial outlook. We've had a busy first quarter in 2018.
As we recently highlighted at our 20th anniversary Analyst Day, we've had several developing programs across the Duchenne franchise splicing platform and niche oncology pipeline. Our DMD franchise is growing with Translarna available in many countries outside the United States for nonsense mutation, Duchenne muscular dystrophy and with Emflaza here in the U. S. For all DMD patients over 5. Over the past 2 decades, we have worked to bring therapies to patients with rare disorders beginning with Duchenne.
We are now expanding on that experience to bring differentiated therapies to patients with high unmet medical need Our vision is to continue to build and world class commercial capabilities. We are now in the position of having 2 commercial products and we were built an effective global commercial operation. We seek to leverage our commercial platform by continuing to deliver new and differentiated therapies in rare disorders. Our commercial success with both Translarna and Plaza is a reflection of our understanding of the unmet need for Duchenne patients. We work diligently to ensure patients have access to therapies that make a difference to them.
Our commercial execution has led to strong year over year revenue growth. In the first quarter of 2018, our revenue for the Duchenne franchise totaled $56,000,000. Our guidance for full year 2018 is between 2 $60,000,000 $295,000,000. We have also set a 5 year goal for the compounded growth of Translarna revenue of 15%. Beyond the current growth opportunity for Translarna outside the United States, we are also looking to expand and bring Translarna to U.
S. Patients. To that end, we the FDA has informed us for an application towards accelerated approval. We expect to initiate such a study by the end of the year. Let me now turn to our internal science 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 or spinal muscular atrophy advancing in the clinic. This program is currently in pivotal studies in partnership with Roche and the SNA 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 annual meeting of the American Academy of Neurology in April.
I'll provide more details has generated Huntington's disease and feels familial dysautonomia programs. These are progressing and are currently in the lead optimization stages. We expect both programs We are focused on rare oncology indications where we can leverage our RNA biology expertise in addressing high unmet medical need. There are currently 2 advanced candidates, PTC596, which is currently in the clinic and PTC 299 which we anticipate will reenter the clinic later this year. Let me now pass the ball over to Marcio who will update you on our clinical and commercial efforts.
Marcio?
Hey, thanks too. 2018 is off to a strong start for our GMD franchise with 2 out of the 3 approved GMD therapies worldwide. And Plaza approved in the U. S. For all GMD patients 5 years older.
And we are pleased with the progress to date and look forward to further expansion of Emflaza use. Since learn is available outside of the U. S. For nonsense mutations in DMD patients, As you know, the label for both therapies currently cover patients 5 years older. And it's well understood concepts that treating earlier patients with DMD would allow for muscle preservation and therefore better patient's outcome.
Aligned with our focus on superior care for patients, we're investing heavily in programs to lower the average age of diagnosed worldwide. Currently, patients in most countries have an average delay of diagnose after presentation of symptoms of more than 40 years. We're investing in genotyping and disease state awareness campaigns to improve the standard of care for DMD patients globally. Complementary to that efforts, we're also working to spends the labels of both Emflaza and Translarna to patients aged as young as the age of two. Our application for our label expansion for Translarna is under review with European regulatory authorities, and we expect a CHMP decision by midyear.
We're also working to finalize a pediatric study for Emflaza in the United States. Now, we'll focus on our commercial performance and expectations beginning with Emflaza. We launched Emflaza in the U. S. In the middle of May last year.
The initial strategic rationale for Emflaza acquisition remains the same as we are marching forward to establish Emflaza as the standard of care for GMG patients in the U. S. We are pleased by the Lancet publication in November of last year of the Synergy 10 years natural history study showing a clear benefit of Emflaza. This was further reinforced by the updated GMD standard of care guidelines, recommending Emflaza initiation at the time of diagnosis. We have completed our 1st initiative of transitioning the Flazacore experience patients to commercial therapy.
And we are now expanding Emflaza use to the segment of patients who have previously used or are currently on prednisone. We have a number of key initiatives underway to ensure that Emflaza is established as a standard of care in these patients. Additional publications are expected, which will reinforce the benefit of Emflaza over prednisone. Emfa's education programs and conference presentations also allow us to share the data supporting the benefits for DMD patients of Emflaza. Because of this data, we understand the needs to bring Emflaza to all DMD patients.
As early as possible. We are proud that through an optimized special pharmacy distribution, and the high touch patient support programs, we have achieved broad access with low out of pocket costs for patients. Reaching naive patients is another critical step in our long term strategy. It is known that in the U. S, about 50% of the DMD patients are still not currently treated with any corticosteroid therapy.
We believe this is a result of the historical view of the risk benefit of a less safe and less effective drug that predates the Emflaza data publications and our launch efforts. This is now reflected in the current DMD treatment guidelines. We have a long term strategy to penetrate to address the youngest patients aged 2 to 5. Now switching gears to Translarna. As Stu said, we remain committed to bringing Translarna to the U.
S. Patients and at the same time, we continue to expand the adoption globally. We're pleased the FDA has recommended a path forward towards the cellular approval. And our intention is to quantify dystrophin in Translarna treated patients in 2 parallel cohorts. One cohort in patients who have been treated with Translarna for at least 1 year and another one in naive patients.
We are currently focused on validating the methods for dystrophin quantification and we intend to initiate this study by the end of the year. With expected readouts during 2019. Outside of the U. S, we first launched Translarna in 2014, We are confirming our expectation for a 15% composite growth through end year 2022. We're excited about Translarna's growth outlook.
The main driver for future growth will continue to be new patient initiation. On Translarna globally. During Q1, we saw growth in patients uptake in all geographies. It's not uncommon in ultra orphan disorders to continue to identify patients once the treatment is available, and we continue to have success in patient densification in all geographies we operate. Patients and physicians have expressed the benefit of treatment from Translarna and compliance as previously reported has remained very high over 90%.
I'd like to point out as we have before that there is a significant variability in the ordering patterns in certain Latin America countries. And while we do not guide revenues on a quarterly basis, our annual Translarna revenue guidance of 170 to $185,000,000 takes into consideration expected variability of this quarterly ordering patterns and reflects the underlying patient demands. Have diversified our product portfolio, revenue and geographic footprints, positioning P2C as an outstanding rare disease business development partner. We have now demonstrated the ability to integrate our products and successfully launch in a challenging therapeutic area. As Stu mentioned earlier, one of the goals for our future growth is to be opportunistic in in licensing and acquiring assets to build our pipeline while all to invest in our internal developments.
Let me now provide a short update on the development programs. As you've described before, we appreciate the perspective of the DMD community on the importance of treating patients as early as possible. In order to preserve muscle function. We plan to conduct pediatric study for Emflaza as requested by the FDA. This study upon completion would provide us an additional 6 months of market exclusivity.
We intend to initiate this study in 2018. Additionally, we have completed APK study of Translarna in children aged 2 to 5. Data from this study was the base of our submission for label expansion to the EMA, which is currently under review. Both of these efforts align well with the intent of the DMD guidelines to begin treatment 41 is started enrolling patients in the third quarter of last year. As a reminder, the conduct of this study is a specific obligation of our EMA approval and the FDA has stated this could serve as a confirmatory study in connection with a potential accelerated approval in the West.
We expect this study to be fully enrolled in 2018 submits our specific obligation with DMA. At our Analyst Day in April, I spoke about the strategy As Stu mentioned, we are focused on rare oncology indications. We have 2 lead assets PTC 299 and PTC 596. 596, our candidates for solid tumor, was designed to have properties to allow blood brain barrier penetration That contributes to tumor resistance. This program is currently focused on addressing rare solid tumors with high unmet needs.
We aim to start new clinical trials in 2 solid tumor indications this year. The other development candidates we're very excited about is PTC299. 299 is a potent DHOG inhibitor. Which is in development for hematological malignancies. We have prior clinical experience with 299 in solid tumors.
Which demonstrate good dose linearity and PD response, while generally well tolerated 2 serious events of drug induced liver injury occur resulting in a clinical holds and the discontinuation of the program in solid tumors. Based on the response we've seen in clinic, we perform extensive work to better understand the mechanism of action for identify patient populations with tumor types, which are sensitive to D8 or G8 for our clinical path forward. We found PTC299 demonstrate broader and more potent activity against leukemic cells. Then against solid tumors, which we expected will enable us to dose at much lower levels in the clinic. We plan to move PTC299 back into the clinic in hematological tumors in the third quarter of this year.
We also have a fast follower the ATOG inhibitor currently in late stage chemical optimization. We're confident that we are well equipped to drive value with both strong commercial and clinical capabilities, and a continued focus on transforming lives of patients with rare disorders. With that, I'll hand the call back to Stuart.
Thanks Marcio. I'd like to share more detail on data recently generated in our SMA program. Most of you are familiar with the program, which is based on our small molecule splicing platform. This technology has been used to discover potential new therapies for spinal muscular atrophy or SMA, a rare genetic neuromuscular 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 SMN2 splicing modifiers.
We believe that an oral systemic therapy provides a competitive advantage because of its broad tissue distribution and ease of administration. Earlier data has been shown that RG-seven thousand nine hundred and sixteen drive SMN2 splicing towards a complete restoration of full length SMN2 Messenger RNA. This program is currently in pivotal stages with 2 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 JUULFITION from the dose finding portion of Firefish and Sunfish were presented at the annual meeting of the Academy of Neurology in April. Data from the dose finding portion of the Firefish study included SNN protein level increases of up to 6.5 fold with a median increase of 3.2 fold. Data from the 21 babies enrolled was presented at the AAN and highlighted continued survival. Previously published natural history data indicate that the median age of event free survival for SMA Type I infants to be between 8 10.5 months. Of the 21 babies presented, 9 have to pass the natural history expectations of 10.5 months survival.
It was also noted no babies have required a tracheostomy or permanent ventilation since study initiation and no baby has lost the ability to swallow. The median age of first dose was 6.7 months and babies have received RG-seventy nine sixteen for a duration of up to 14.8 months. As of the April presentation, RG7916, has been well tolerated at all It was previously reported in January that 2 babies died due to disease progression and the deaths were determined not to be drug related. The Firefish dose finding study is now complete and all babies are being transitioned to the therapeutic dose for an extension trial. Ongoing data presentation from this trial is expected throughout 2018, including more survival data chapping ten scores and other motor milestones.
We look forward to sharing these developing data with you. As you may have heard from several key opinion leaders at our Analyst Day in April, now that survival data has been presented in SMA Babies, the bar has been raised. Physicians and families look forward now to treatment therapies that allow SMA patients to achieve developmental milestones and have broader function. Recruitment is ongoing globally for the pivotal part of Firefish study. The primary endpoint of this registrational study is the percentage of infants who are sitting without support at 12 months of treatment as assessed by the Bailey Income Scale.
Presentations on the SUNFISH and JEWELFISH studies also supported safety and tolerability of RG-seventy nine sixteen. Data from the dose finding portion of SUNFISH was also presented at AAI, showing that S and N protein levels increases of approximately 2.5 fold were sustained for up to 35 weeks. In October 2017, Sunfish enters the pivotal stage and is currently enrolling. At that time, all SUNFISS patients from dose finding study were transitioned to the therapeutic dose and have been maintained with no dropouts or serious adverse events. Lastly, preclinical data were also presented demonstrating that RG-seven thousand nine hundred and sixteen increases FMM protein levels in several tissues, including brain, muscle and blood.
These increases were proportional and correlated. These results demonstrate the relevance of SNN protein level increases in blood as The SMA program is not only progressing towards an oral and systemic treatment for SMA patients. It also validates that our splicing platform technology can identify selective compounds that modulate pre mRNA splicing. We are now applying
our
These include programs in Huntington's disease and familial dysautonomia. We expect both programs to enter the clinic by 2020. We're very excited about our splicing programs and covered 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 Kristine Otter, our Principal Financial Officer.
Let me pass it over to Christine.
Thanks, Stu. Earlier today, we issued a press release summarizing the details of our financial results for the first quarter of 2018, and I refer you to that release for full details. I'll start with a few we reported strong performance across our DMD franchise. As Sue mentioned, our results for first quarter of 2018 were as expected with reported total net product revenue of approximately $56,000,000. Based on where we stand today, we are reiterating our 2018 guidance of $260,000,000 to $295,000,000.
Translarna net product sales were $36,800,000 for the first quarter of 2018, representing 39% growth over the prior period, driven by both expansion into new territories, and increased penetration in existing geographies. In plasma net product sales reflects success of the launch with reported revenue of $19,200,000 in the first quarter of 2018 having just launched in May 2017. Total revenues for the first quarter compared to $26,500,000 in of Translarna and a successful U. S. Emflaza launch.
Non GAAP R&D expenses were $27,600,000 for the first quarter of 2018, excluding $3,700,000 in non cash stock based compensation expense compared to $22,900,000 for the same period in 2017, excluding $4,500,000 in non cash stock based compensation expense. Reflects the increased investment in were $29,000,000 for the first quarter of 2018, excluding $4,000,000 in non cash stock based compensation expense compared to $20,900,000 for the same period in 2017, excluding $4,600,000 in non cash stock based compensation expense, reflecting continued investment in our commercial activities for our DMD franchise. Net loss for the first quarter of 2018 was $19,300,000 compared to a net loss of $29,100,000 for the same period in 2017. This decline in net loss reflects International Corporate Infrastructure. Cash, patch of equivalents, and marketable securities totaled approximately $178,300,000 at March 31, 2018, compared to $191,200,000 atyearend2017.
In April, we successfully completed public offering of 4,600,000 shares of common stock, raising $117,900,000 in the public market. We intend to use the net proceeds from this offering primarily to transact in potential business development opportunities and to fund and accelerate our research and development efforts, including our programs for alternative slicing and our niche oncology pipeline. I will now
Thank to prevent any background noise Our first question comes from the line of Brian Abrahams of RBC Capital Markets. Your question please.
Hi there. Thanks for taking my questions. First question on Translarna, commercially, Can you tell us a little bit more about whether you saw any inventory effects, seasonality the impact of ordering patterns in the first quarter. And where you are quarter over quarter with respect to patient demand and volume and persistence and compliance?
Hey, Brian, thanks for the question. Let me give Marcio to
Hey, Brian, thank you very much for the question. So we did see, as you probably recall, right, Q4 was a very strong quarter for us last year. So we had a lot of demand for both patients and some orders that came very late on quarters. So we had to some extent a carryover that is not what I would call in events or building because you really don't see like patients caring inventory or like payers carrying inventory for a long time for Translarna, but that is some effect of that as we transition from the end of the year to this year. The important thing that we're seeing as we look into and what makes gives me comfort and optimism towards the end of the year is we saw like patients being added in every region, as I mentioned, a few minutes ago, and the compliance is still remaining pretty high.
We are not seen drop offs like to the later part of your question, historically, we've seen more than 90%. So 90 of compliance and persistence. And we are still seeing exactly that same range in all regions. We're adding patients There is always a little bit of delay here and there at the very beginning of the year. So I'm not sure if I was going to call that much as, like a seasonality effects as it is like just holidays and things like that that impacts, but nothing major that we are seeing in terms of the dynamics of the patients.
I hope that answers the question.
Yes, that's very helpful. And then, on the U. S. Approval geared, studies and investigations for Translarna. Can you maybe help us understand a little bit better what the comparison would be for the patients who are on drug for at least a year in terms of, dystrophin levels?
Would that be compared to baseline levels for the naive group? Or do you have would you be using sort of historical data from other sources? Maybe help us understand how that data would be, would all fit together, the naive versus the experienced patients?
Yes, we would use sort of a combined control, that would be untreated patients. So that would what we're looking for obviously is to represent what a baseline would look like, which I think and there is some precedent for this. And in which how Sarepta had done this previously. So we would use that as the baseline and compare the treatment to pay of patients who've been on for some time to that control.
Got it. And one more for me, if you don't mind, With respect to the SMA program in 7916, what would you be looking for in terms of the milestone and functional data as we kind of see data evolve over the course of this year at the more therapeutic doses and with longer follow-up. Is there any specific goal? And I'm also curious with respect to whether there's any specific symptoms like respiratory symptoms, for instance, that you might be particularly focused on to help us understand whether there's improved brainstem penetration versus available therapies? And I'll hop back in the queue.
Yes, thanks for that. Obviously, we're pretty excited about RG7916 and we think it has the potential to be best in class because of its systemic nature of it. And you can and I think there's a lot of data now that we've shown, pre clinically how it passes the blood brain barrier and that the levels of protein in the blood represents in a sense about a one to one that we've seen, not only in rodents, but also in non human primates as well. So we're pretty confident of that as well. And just to remind everyone, we saw up to a 6.5 fold increase in the babies that were in the type 1 babies.
And we talked a bit about the survival and the how we're passing the the milestones on that in terms of the 8.5 months and how 16 babies are have surpassed 8 and a half monthlies. So we feel pretty good about that. And over the coming months, obviously, we're going to look at all the motor milestones milestones as well, hopefully giving about CHOP INTAN, babysitting, all that will come as it comes through and it gets formally looked at in the as the data improves. So that's where we're at. That's what we hope to be able to talk about, at least describe as the year goes on.
Thank you. Our next question comes from the line of Gena Wang of Barclays. Your line is open.
Thank you very much for taking my questions. And apologies if some already discussed that I missed the beginning of the call. Just wanted to ask regarding, I think, of this week, I mean, this quarter, any inventory impact stocking, destocking for both Translarna and Insuaza?
Sure. Marcia, Yes. Hey, Jenna. Yeah, we're seeing, I mentioned this a little bit before, but we did see, a little bit of, I wouldn't call inventory build as I mentioned in terms of patients getting shipments late in Q4. So obviously they use of that shipments go through Q1.
So just that's the natural use that we see in some geographies. They end up getting specifically for Translarna, right? They start getting for like more than a month or so. So there is a little bit of spillover throughout the year, but it's not really inventory as much in a strict sense, but, they use patterns that we see throughout the year. So that impacts a little bit at the very beginning.
With Emflaza specifically, that isn't much of that dynamic. What do we see is really the use and the authorizations being happening pretty much throughout the month at the beginning of the month. It's harder to ship as well close to the holidays in the United States. So we try to get that all done as early as possible. So not as much even for this small amounts that are described for Translarna, we don't see that for, happening for Orin Plaza.
Okay. And then any more color regarding, I don't know if you comment earlier regarding the number of patients beyond the initial bridging programs. Like new patients for this quarter for INFLASA?
Sure. So we stopped disclosing specific patient numbers in Q3 last year, but just to give you a little bit of color on that, right? So we're seeing growth in patients like we're pretty happy with what we are seeing in terms of activities from fields, the programs we put in place, we move to this what we call the 2nd phase of the launch now. So pretty much the bridging that you mentioned before and the patients who were previously experienced with the FASA cord, they've been transitioned. So what we are seeing more and more now is in the 2nd phase, as I described, these patients who are being treated with like prednisone, prednisolone or like any other forms that there's a lot of stuff, a lot of ways this patient is being treated.
It's starting treatments, it's staying on that treatment. And we are, we are pretty happy with the trajectory that we are seeing right now, but obviously the work does not stop. And we're like looking every day on how to make this better and how to get more patients on what we believe ultimately is best for them. It's the only approved treatment in the U. S.
And the best standard of care. Okay.
Thank you. And then just one quick question regarding SMA program. Can you just remind us, like, in terms of a rough timeline that you or and or Roche will present the SMA functional data? And also if you can just remind us in terms of onset of the drug, like how quickly once you start to dose the patient how quickly you will start to see the protein level increase and then that could translate into the clinical benefit?
Yes, sure. Yes, thanks for that. And just to remind as we were talking about a little bit ago, that we have the SUNFISH type 1 study, the Part 1 where we had where we talked about previously where we had 6 of the 21 babies, we talked about previously 2 had had passed that were non drug related and that 16 babies already are, aligned with the key milestone of 18.5 months. So just to remind everyone that the median age of enrollment was about 6.7 months and they've been on drug for up to 14.8 months. So, what we're going to be doing is going to be presenting data throughout this year.
And obviously, we're excited about that and stay tuned. We'll be talking about We'll be going from not just survival data, but going to then we'll be talking about the motor function milestones and and measurements like CHOP INTAN, put out babysitting, other things, other talk about all the important milestones that we'll be measuring throughout the year. And we intend to have such discussions during as we see them and decide that we need to present that, describe them or to have them at scientific meetings. So that's our plan. So we plan to have hopefully a significant number of discussions with you over the year.
Okay. And then just quick question regarding the, how quick onset the drug, once you start to dose the patient, yeah.
Yes. Yes. So, I mean, obviously, it's an orally bioavailable systemic drug that gets into all tissues. We saw in blood that occurred actually quite rapidly, where you start seeing a production of the protein. You give it for a day, you get RNA may And then from there, you get protein relatively quickly.
So you can measure quite rapidly.
Okay. So that happened usually in days, right? You will be able to see? Okay.
That's correct.
Okay. Thank you very much.
Our next question comes from the line of Raju Prasad of William Blair. Your line is open.
Thanks for taking the question and sorry if I'm going to address this. Hop on the call a little late. But as far as the dystrophin study for the U. S, have you met with the FDA yet? Are you have any kind of incremental color on the type of data that you'll have to submit and potentially from what what parts of the body or what muscle types?
Sure. Hey, Marcio wants to take that.
Yes, and thanks for the question. So we've been talking into the FDA, well, I'll be very happy with the openness that the agents has had with us in terms of being even a little bit more informal, some of the communications and keeping the door open for back and forth that we might require with them as we mentioned late last year that we would be talking to them during Q1. And we do regularly talk. What we are focusing right now really with our understanding of some of these conversations is that the key is really for us to pick the methods continue collaborating with them and understand like what the decision wants to do in terms of the qualification. So we are running few tracks in parallel based on that, we have some softwood jitting house and some collaborations.
We are doing externally with private labs as well on the quantification. That's the rate limiting. As I mentioned before, and we discussed at the Analyst events on April 17, we expect to start this trial in the U. S. Before the end of the year.
We see no issues whatsoever right now in March towards that date. And it's really a parallel track between the clinical initiation in the methodology to be, validated.
Great. And then just a quick question. In the press release, for the part 2 of the FIREFISH study, the endpoint is the proportion of patients sitting without support after 12 months of RG7 916 treatment, does that assume that the patient's median age is going to be around 6 months? I thought it was kind of an 18 month end point for other companies?
So, no, right. So it's the number of patients sitting at the 12 months and unassisted for 5 seconds.
Oh, okay. Got it. And that's agnostic to the age they are when they're enrolled in the study. So if it's one or 2 month old, it would be when they were 13 or 14 months?
That's correct. That it's I think it's important for us to remind here, right? So the if you look into part 1, these patients were fairly old and they had to be symptomatic at 3 months, no later than 3 months. What is a little bit different when you look into other data sets out there These are like the what we call like type 1 business are the most severe type 1 patients. We are seeing like the main age sorry, the median age on the last cut that we showed to you guys was 6.7 months.
So independently of all of that and we discussed this before, we're very confident on how this study is going and we're looking forward to show that So it's illustrative of to answer our questions directly, to the dates they are enrolled is 12 months after enrollment.
And maybe just a little color to that. We are part of the reason we chose that they had to be being diagnosed by 3 months because it is an open label study. So that really sort of defines them as a truly type 1.
Great. Thanks. Welcome.
I was just going to put one point And is the outcome of that is we're looking for a 5 or greater for out of 40 would be statistically significant.
Thank you. Our next question comes from the line of Joel Beatty of Citi. Your line is open.
Hi, thanks for taking the questions. First one is on the SMA program. Could you share any data to date on the differences between those levels that you saw in part 1 of the study including any information you might be able to provide on how the eventual dose that was chosen for the pivotal part of the study was chosen. As well as any implications from the dose finding study in terms of the effects you might expect to see in the first patients that were enrolled compared to the patients that were enrolled later in the study?
Yes, sure. I think it's obviously the first part was really a dose finding study. And really the doses we're looking both at variables such as age and weight as part of that. And so that once we completed that portion, we're now moving them up to what we think is the efficacious dose and following from all those from there. So I think that's the that's sort of the point where everyone is on the equal playing field in the sense of having the same dose is now occurring.
So, they all working on the same dose. And we were trying we were picking the dose really based on, we had the advantage of having a PD marker that lets us look at the level of S and M protein. So you really could actually get to define how well you're doing by that dose. So that was the key part of this.
Got it. And then one other question on the 15% growth this year from Translarna, Could you give any color on is this expected kind of across the board or what particular regions or groups do you expect to contribute to that growth post?
Sure. So the 15 percent composite growth until 2022, right, that we gave as a guidance The expectation there in the base is really driven by patient accrual. So we expect to continue to to accrue patients globally. What we are seeing right now, what our projections show, what the programs are put in place are supporting, is that this growth is going to continue to come for all geographies that we operate. Like beginning of the year, like, of the last couple of months, We're seeing this materialize in all the geographies.
We're happy with the progress on patient identification. We're happy with the progress in terms of genotyping. And as we expand, obviously, in the later years, the remaining patients and the more percent wise growth is going to come for the regions that are less penetrated like the Middle East and Africa, CEE and Latin America. But in general, all the regions are growing. They're all included in the potential.
I think it's worth to mention as well as I talked in the remarks a few minutes ago, we are in the last stage of the conversations with DMEA for the labor expansion for the 2 to 5. And that's going to add more patients to the pool, going to be able to treat them globally And we hopefully are going to be resolving this in the next couple of months with EMA. I'm going to be able to launch the product later this year beginning of next year and start to accrue patients.
Great. Thank you.
Thank you.
Thank you. And at this time, I'd like to turn the call back over to Stuart Peltz, for any closing remarks, sir.
Hey, thank you. And, thank you all for joining the call. And, as you can see, we're pretty excited about 2018. And we think this is going to be a really important year for us with several milestones that think are going to potentially drive value creation. And we'll continue to grow the Duchenne franchise.
We're going to present additional data from our SMA program. And we'll be sharing more on the oncology pipeline. In addition, it's our goal to do at least one transaction in business development this year to add something else on. So I think there's a lot of value generation to occur. And I thank you for joining the call today.
Thank you sir. Ladies and gentlemen, this concludes today's conference. Thank you for your participation and have a wonderful day.