Good day, ladies and gentlemen, and welcome to the Vertex Pharmaceutical Second Quarter 2018 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, today's program may be recorded. I would now like to introduce your host for today's program, Michael Partridge, Vertex Pharmaceuticals Inc.
Please go ahead.
Thank you, and welcome to
the Vertex 2nd quarter 2018 conference call. This is Michael Partridge, Senior Vice President of Investor Relations for Vertex. Tonight, we will review our financial results and our continued progress to develop new medicines for all people with cystic fibrosis. Doctor. Jeff Lyden, Chairman and CEO Phoenix Smith, Chief Operating Officer, and will provide prepared remarks this evening.
Stuart Arnold, Chief Commercial Officer and Doctor. Reshma Kewal Remani, Chief Medical Officer, and join us for Q And A. We recommend that you access the webcast slides as you listen to this call. These slides are available for download on our website. This conference call is being recorded and a replay will be available on our website starting later tonight.
We will make forward looking statements on this call. These statements are subject to the risks and uncertainties discussed in detail in today's press release and our filings with the Securities And Exchange Commission. These statements, including without limitation, those regarding Vertex's market estimate medicines, the ongoing development and potential commercialization of any triple combination regimen, for cystic fibrosis, Vertex's other programs and Vertex's future financial performance are based on management's current assumptions. Apture outcomes and events could differ materially. I will now turn the call over to Doctor.
Jeff White. Thanks, Michael. Good evening, everyone. In the first half
of twenty eighteen, Ertex continued
to make tremendous progress across our business, especially in the area of cystic fibrosis. Today, we are treating more patients with ICF medicines than ever before
and delivering important clinical benefits
to thousands of people around the world. First to the launch of SYMDEKO in the U. S. The demand for this medicine has been strong across a wide range of eligible patients. Including those who previously discontinued or never and similar to prior launches in the U.
S. For KALYDECO and ORKAMBI. And feedback from patients and physicians has been highly positive. Treating more patients is driving significant revenue growth. And on the basis of the rapid uptake of SYMDEKO in the first half of twenty eighteen, we are raising our total revenue guidance for 2018, which Ian will review in a moment.
2nd, we are moving towards achieving
our goal to treat CF patients at younger and younger ages. So we may help deliver transformative benefits early in life and slow or prevent the progression of disease. This progress is exemplified by the pending approvals for KALYDECO in Children's youngest 1 year of age and for Acadian Children ages 2 to 5 years where we expect decisions from the FDA this summer. We're also evaluating SYMDEKO in Children ages 6 to 11 and expect data from this study later this year. 3rd, our 2 triple combination regimens that contain a next generation corrector are proceeding rapidly through Phase III development.
We expect to complete enrollment of our phase 3 study for both the X659 and the X445 triple combination regimens in the second half of this year. Based on an anticipated completion of enrollment for both programs, we expect to submit a new drug application no later in mid-twenty 19. The VX-six fifty nine VX-four forty five programs have moved exceptionally fast advancing from first synthesis of the molecules all the way to late stage development in a little over 2 years. I look forward to updating you on the continued progress for these programs in the coming months. Beyond CF, we continue to invest to discover and develop medicine in other serious diseases.
In our pain program, we have generated phase 2 data for selective NaV1.8 inhibitor VX-one hundred and fifty, representing the first proof of concept for NaV1.8 inhibition in the treatment of both acute pain and chronic inflammatory pain. These days have provided important and cleared clinical validation for this We expect to have Phase 2 results for BS-one hundred and fifty and a third type of pain or path of pain in early 2019. We're no longer progressing VX-one hundred and twenty eight, our first follow on NaV1.8 inhibitor based on PK and tolerability findings from a phase 1 study. We believe there is significant potential in the treatment of pain with an NaV1.8 inhibitor, and we continue to invest in research and development efforts to advance VX-one hundred and fifty in the clinic and to move additional NaV1.8 inhibitors into development. With our partner CRISPR Therapeutics, we're advancing CTX001 as the first gene editing treatment for both sickle cell disease and beta thalassemia using CRISPR Cas9 technology.
In beta thalassemia, we obtained approval in the UK for a clinical trial application or CPA for CTX001 earlier this year and recently obtained a CTF approval in Canada. We remain on track to initiate the first study of CTX001 betathalassemia later this year. In sickle cell disease, we also recently obtained CPA approvals in Canada and the UK. And we continue to work with the U. S.
FDA to address the agency's questions regarding the IND for CTX001 was submitted earlier this year. We also continue to make significant strides with our internal research efforts, We have comped out the late stage preclinical development for alpha-one antitrypsin deficiency or AAT and focal segmental glomerulosclerosis or FSGS. These early stage programs demonstrated a strong fit with our business model and research strategy, and we aim to develop transformative medicines for serious diseases in specialty markets to create the greatest value for both patients and shareholders. We choose diseases with well understood value where we can use or create early clinical markers to support the potential for transformative benefit and enable rapid development timelines. With both AAT and FSGS, we have the ability to design early stage clinical studies that may provide initial proof of concept data 2019 or 2020 to inform further development.
This is very analogous to how we successfully advanced our CF portfolio. In summary, we've made tremendous progress across our business in the first half of the year. In CF, it's remarkable that it was just 1 year ago when we announced first phase 2 data for a triple combination regimen. By this time, next summer, we may have submitted for FDA approval, one of these further defining the path toward treating up to 90% of all people with this devastating disease. With our pipeline, we're advancing multiple new medicines of fundamentally change the treatment of other serious diseases in the future.
Revenues, which will drive sustainable long term earning and operating margin growth and enable continued investment in the discovery of new future medicines. I'll now turn the call over to Ian.
Thanks, Jeff, and good evening to everyone. I'm pleased to review our second quarter 2018 financial results. Which are highlighted by the launch of SYMDEKO in the U. S. And our upward revision for 2018 full year financial guidance for total CF revenues.
Revenues first. Total CF product revenues of $750,000,000 in the second quarter of 2018 represents a 46% increase compared to the $514,000,000 we recorded in the second quarter of 2017. We continue to see significant revenue growth as we increase the number of patients treated with our medicines globally. 2nd quarter included $186,000,000 in revenues from the launch of SYMDEKO in the U. S, which is the primary driver of the rapid growth in total CF revenues.
Demand for SYMDEKO is strong, and we are seeing favorable early trend in persistence and compliance and are receiving positive feedback from patients and physicians. 5 months into launch in the U. S, public and private insurance plans, representing 95% of covered lives are processing claims for SYMDEKO and nearly all state Medicaid programs are providing coverage for SYMDEKO. The demand has been particularly strong among the F508delhoppers, I guess, patients initiating treatment for the fullest time and also in patients who discontinued oral county coming back to initiate therapy for SYMDEKO. We're also seeing patients switching from old Camry to SYMDEKO as evidenced by Cambridge revenues of $236,000,000 in the U.
S. For the 2nd quarter compared to $282,000,000 for the first quarter of this year. Based on the launch to date and our expectation for continued growth in SYMDEKO revenues as more patients initiate and remain on treatment over the coming months, We today revised our guidance for total CF product revenues to $2,900,000,000 to $3,000,000,000
from a prior range
of $2,650,000,000 to $2,800,000,000. The midpoint of this new range represents approximately 36% growth over 2017. Our guidance does not assume completion of new reimbursement agreements outside of the U. S. During 2018.
With reimbursement outside the U. S, we remain focused on providing broad access to current and future medicines by establishing long term reimbursement agreements. The latest example of this portfolio type of reimbursement agreement was announced with Sweden in June and provides immediate access to ORKAMBI as a framework for rapid access to future medicines. We established similar agreements in Ireland and other countries
and are engaged in ongoing discussions
with additional countries regarding long term portfolio arrangements. Now to expenses. Our second quarter 2019 non GAAP combined R and D and SG and A expenses were $388,000,000, compared to $333,000,000 in the second quarter of 2017. This increase was primarily due to the advancement of the portfolio of triple combination regimens for CF and investment to support the treatment of patients with our medicines globally. Our guidance for combined non GAAP R and D and SG and A expense of $1,500,000,000 to $1,550,000,000 is unchanged.
The key investment drivers continue to be the execution fiscal studies for 2 triple combination regimens, supply chain investment for triple combination regimens, and incremental investment to support the launch of SYMDEKO. Non GAAP net income for the second quarter of 2018 was $244,000,000, with an EPS of $0.94, compared to non GAAP net income of $99,000,000 and an EPS of $0.39 for the second quarter of 2017. The increase in non GAAP net income and EPS was largely driven by the strong growth in total CF product revenues. We ended the quarter with approximately $2,800,000,000 in cash, cash equivalents and marketable securities compared to two 0.1 at the beginning of this year. The financial profile of our business is strong.
We continue to see significant growth in revenues, expanding operating margins and increasing earnings, and we expect these trends to continue as we expand access to our medicines globally and increase the number of patients eligible for and treated with our medicines. We also continued to invest in internal R and D for CF and other diseases and in external innovation through business development to create future medicines that will continue to drive growth. I look forward to updating you as the year progresses. And with that, I'll open the line to questions.
Our first question comes from the line of Phil De Du from Cowen And Company. Your question, please.
Afternoon. Thanks for taking my question. Questions on the SYMDEKO launch. Congratulations on the number. It's really impressive, but curious to get a little bit more color on the trends there.
So I guess what were the U. S. Sales work can be in the quarter and how did they compare to SYMDEKO? And given that they're probably not too far off, what is your sense of Cymbigo's penetration of its U. S.
Opportunity given that the bottom end of the guidance can kind of be hit by flat quarters in H2? Seems like you'd suggest it's largely penetrated, but I'm curious to get a little bit more information
Sure. Hi, Phil. It's Stewart. I'll try to answer all those. So for ORKAMBI in the U.
S. We recorded net revenues of $236,000,000 for the quarter compared to the $186,000,000 that Iain referenced for SYMDEKO. In terms of how the launch is going, as Jeff said in his prepared remarks, we've actually seen strong uptake across the 3 different patient populations that we were anticipating that we'd see, demand in. That's those who have Tridor can be previously and discontinued. Those who have, never been exposed to a CFTR modulator, either ORKAMBI or KALYDECO, And we saw a fair amount of transitions from ORKAMBI to SYMDEKO, and that's the reason why you see the sequential decline for ORKAMBI from Q1 to Q2.
In terms of, for the balance of the year, whilst the launch is off to a strong start, we do continue to expect that there is a further growth opportunity for SYMDEKO because they're not yet, as you might expect, for 4 or so months into the launch, we're not yet fully penetrated into those patient populations that were either naive or seeing patients discontinue.
Thank you. Our next question comes from the line of Robin Karnasquez from TD. Your question, please.
Hi, guys. Thanks. Question. So the question I wrote a little bit about thinking about the company more long term now that the 6 fibrosis is playing out and some of your competitors are dropping off. We have to speak about capital location, business development, particularly, where at least in, you know, your pain and your, and is your and CRISPR are more early stage or phase 2.
How do you how do you think about how you might grow the business, earlier using using some capital?
Hey, Robin, this is Jeff. I'll maybe take the 1st long term strategic part of it and then I'll turn it over to Ian. To talk a little more specifically about business development. So as we look at the business and we've talked about this a little bit before, mission 1 is to complete the journey at CF. And obviously, that really involves bringing the best triple ledger in the patients as quickly as possible.
We're well on track
to do that. The good news
today is that those trials are enrolling very rapidly. And if and when we do that and we have a high level of confidence, we will, we see growth coming from the CF franchise for a number of years going forward. So I think that's really the first important point. And both top line and bottom line growth, that's significant. Then obviously, we're focusing a lot of attention on what comes after CF, and we divide that into 2 parts.
Our internal pipeline, we're we believe we have a very unique scientific innovation engine that now generated multiple breakthrough medicines And therefore, we're confident that we're going to do that again in some of the diseases we're talking about like AAT, sickle cell, pain, and FSGS. And the good news about those is that I think we'll have a lot more visibility to them earlier than many people expect because the early stage clinical trials, like in CF give you a pretty good sense of where you are after 20 patients, 50 patients etcetera. So in 2019, 2020, we expect to have a lot more visibility to the success of those programs. That's the internal part. Obviously, we're also accumulating a lot of capital and that gives us a lot of opportunity to invest externally, more and more each quarter as you're seeing.
And so we are seeing our VBFs ramp up pretty significantly as I said before, are we going to go out and buy short term revenue? No, we really don't need to, but we certainly want to supplement our pipeline and invest in the kinds of innovation. And maybe I'll turn it over to Ian who can review our strategy with you there. Sure. First of all,
as Jeff reviews, our broader strategy, as we look out side of the company, everything we look at is absolutely consistent to how we think about the company inside. We have three areas that we're focused on our side of the company obviously, first is cystic fibrosis. We should look at everything in cystic fibrosis and see if it's complementary to our approaches to affecting the underlying cause of the disease. So we do look at everything that moves, and that includes different modalities and therapies for cystic fibrosis. Second area is really platforms in early stage technologies.
And you've seen us complete a couple of deals in the last couple of years. That have been very important for us in terms of getting us into new areas of science and new modalities. And in particular, Jeff updated you on our our Krista Therapeutics collaboration on the call this evening where we've made really nice progress in beta thalassemia and sickle cell. And then the 3rd area that we continue to look at is kind of more opportunistic in terms of products and medicines that would be consistent our overall disease strategies, going from the underlying cause of biology through to the disease itself and how those diseases may fit inside vertex and be consistent what we're working on inside Vertex. We're very active.
We're more active than we've ever been. We're able to do that because today, we do have capital that we can apply outside the company. So we look forward to advancing that, that efforts within our business and advising you when we closed certain transactions. Thank you.
Our next question comes from the line of Harrison from Morgan Stanley. Your question please.
Thank you for taking the questions. This is Jeff Hung in for Matthew. I guess first, for SYMDEKO, are you seeing patients come back the market. Can you comment on persistence and compliance compared to RPM?
Yes, Geoff, this is Stuart here. So yes, we are seeing patients who had previously been initiated on or can
be discontinued,
we are seeing a large number of those patients be reinitiated on a CFTR modulator in this case, SYMDEKO, and it really is that growth in PACE to have been treated with a CFTR modulator right, those who had discontinued or those who were naive to therapy had never been treated with ORKAMBI that's driving the growth in revenues that you saw in the 2nd quarter. And it's that adding new patients, which is underlying the increase in our revenue guidance that we gave tonight to $2,900,000,000 to $3,000,000,000.
Great. Thanks. And then can you comment on if you view the UK pricing dispute as isolated or potentially broadening out to the rest
of Europe, the price of
the media is obviously very low compared to your existing prices.
Yes. So I mean,
I'll just start by reminding you and others on the call that, we've been successful in a large number of countries across Europe in securing pricing and reimbursement agreements, be it Germany, Italy, Ireland, the Netherlands, And as a result of that, I'm thrilled to say that thousands of patients have access to ORKAMBI today, But obviously, our goal is to ensure that all eligible patients in all countries, have access to ORKAMBI and indeed, are our future med And so we are absolutely focused on securing reimbursement in those markets where we don't yet have access for patients. There's a number of significant markets. Obviously, defined by the number of patients there, places like Australia and the UK, there are particularly large numbers of patients there. And so obviously, we're very focused on those as we are in all countries where we don't yet have access. And we know those patients have been waiting too long and we're not going to stop until we get access for those people.
Great. Thanks. And maybe one last one, on the CRISPR IND, that you have written comments from the FDA about the ID hold, can you comment on the difference between what the UK and Canada regulator asked and what the FDA is asking? And any comments about what you need to be received in the U. S?
Thank you.
Yes, we're obviously in those discussions with the FDA to answer the questions. As you, as you know, this is, likely going to be the 1st gene editing trial outside of cancer to be approved for human trials. And so I think the FDA is being appropriately cautious and conservative. We're in the process of answering their questions. We obviously don't comment on that while we're those discussions.
But as soon as we've answered them, I have a clear plan forward. We'll let you know.
Thank you. Our next question comes from the line of Karen Flynn from Goldman Sachs. Your question, please.
Hi. Thank you.
This is Gavin on for Terrence. Congrats on the quarter. Maybe just one on the triple combo. Can you give us any update Is there upside to completing enrollment before the end of the year and potentially starting those or getting a readout before mid 2019? Yes, this is Jeff.
So, what we've told you today is really what we know obviously early
on in the enrollment
of the 4 45 trial. So it's just too early to give you any more specifics than that. But I think the good news is that we're going to be able to complete enrollment of both of those trials early than we thought by the end of the year. And we're very confident that will allow us to to make a choice of the best regimen and submit, that application with the FDA by mid-twenty 19.
Our next question comes from the line of Michael Yee from Jefferies. Your question please.
Hi. This is Andrew on for Mike. Actually, a a pit follow-up on the last question. Would you consider disclosing the data for both triples at the same time, or would they be staggered 1 by 1, or would they be disclosed once you submit the NDA? Thanks.
Hey, Andrew, it's Ian. At this point in time, really too early for us to figure out how we're going to disclose this. As Jeff made, just previously in the comments, we've just announced that we expect to complete enrollment in the second half of this year. We are comfortable based on that, though, saying that we expect to filed an NDA in the U. S.
By no later than mid next year of mid-twenty 19. To start talking about how we would disclose data and how it rolls out and whether these studies are close enough together to do it both together, just a little too early. We do anticipate we don't open an issue to you and to other investors. And as the year progresses and we talk to you more, will give you guidance on how we think about it when we have greater visibility.
Thanks. And just a quick follow-up on the concert molecule, Have you completed the dose ranging studies? What else needs to happen basically?
Hi, there. This is Reshma. As you know, VX-five sixty one, which is a molecule being licensed from Concert is an important part of our portfolio. As we drive towards getting a medicine for 90% of patients with CF with the once a day pill. We are wrapping up our, study design and discussing that with the FDA.
And as soon as we finish all that up, we're going to be starting the trials.
Thanks again.
Thank you. Our next question comes from the line of Laura Chico from Raymond James. Your question, please?
Yes. Hi. This is actually, Timur, Ivanko Filora. So I guess the first question we have is about seasonality for SYMDEKO. Looks like you've had a great lunch.
And the summer is halfway through in the US. And wondering if you can opine on the potential for any seasonal headwinds that might impact US looking ahead. And how should we be thinking about this dynamic in 2H18?
Yes. So this is Stuart. So yes, it is not untypical to see a seasonal dip in compliance as people go on their vacation and their normal kind of routine, if I can call it, that is disruptive. We've been focused on that for a number of years now. It certainly was a a big focus of ours.
Last year is, again, this year. And so we're going to be doing everything we can, working with providers and directly with patients who are applicable to try and maintain their compliance with their physician's instructions. So exactly how that's going to play out. Obviously, you know, it's too early for us to, to say right now, but it's a phenomenon we're familiar with, the one we're certainly, focused on very closely. We'll be doing that again this year.
Okay. Thanks. And maybe a bigger question on, bigger picture question on reimbursement. Do you think do you think the reimbursement landscape has changed or how the, companies will continue to be rewarded for innovation has changed recently, or has it always been a tough situation like it's been recently? Thank you.
Yes, this is Jeff. So, Ken, when we talk about reimbursement, we always separate the U. S. And Europe. And countries outside, yes, they're very, very different.
In the U. S, We believe that innovation is rewarded, and that's really based on our own experience and those of others of breakthrough drugs like our 3 CF drugs, that had very broad and very rapid coverage from all of the major payers, including the government payers. And we don't see that changing, over the coming years. In Europe, obviously, it's a very different environment, single payer environment. And again, over the last 2 to 3 years, at least, it's been a difficult environment.
I think for all companies, including the innovative companies. And one of the things we're pleased with as Stuart said is that we've been able to fairly rapidly secure reimbursement in a large number of European countries, and we're moving our discussions along into several countries that remain fast. The UK and Australia being primary examples. It's always a bit of a difficult discussion and we're we understand that those countries look at the following the price per patient at the total budget impact. But so far, we're pleased with what we're seeing, and we think that innovative transformational breakthrough growth will always be at the upper end of the reimbursement and price envelope.
And those new categories we're developing both in CF and beyond.
Thank you. Our next question comes from the line of Geoffrey Porges from Leerink. Your question, please.
Could you give us the U. S. Sales of KALYDECO? 2nd, could you tell us the channel inventory contribution to this detector revenue number? And then, on the triple combination, we suggested that later than mid-twenty 19, put it the early 2019, given the fact you're fully enrolled already.
And regardless of when you file it, should we assume the same sort of accelerated review schedule that the FDA has given you for your previous combinations? Thanks.
So, Jeff, this is Stewart. On the Caladico U. S. Sales recorded net revenues quarter were $161,000,000.
And in terms
of channel inventory, there was no, meaningful channel build at all in our Q2 numbers, all of the growth we saw there was driven by organic patient growth, more patients going on to our medicines.
Sorry, this is Jeff. Just to talk about the timeline. It's just too early to give you much more than we've given today, which is what we know that, that we're now very confident based on what we've seen with the 659 enrollment in the very beginning of the 4 45 enrollment that both trials will complete. You may, one statement, Jeff, I just want to make sure I do to clarify, which is you said that we've completed enrollment, we actually have not completed enrollment of the 659 trial yet. So you want to be be accurate about that.
But we do think both trials will complete or both programs will complete enrollment by the end of the year. If you do the math on that end, that would allow us to compare the regimen's filing MDA in the U. S. By midyear. You asked about how the FDA will deal with these.
Obviously, don't speak for the FDA and I can't, and it's going to depend on the data. So if the data is as promising as our phase 2 data, we're obviously going to push very hard. I think the FDA is very interested in moving as quickly as possible, taking all appropriate precautions, but I think we're going to have to see the data. Thanks very much. Thank
you. Our next question comes from the line of Brian Abrahams from RBC Capital Markets.
Hi, thanks for taking my questions and congrats on the strong quarter. Just want to go back to reimbursement internationally and just sort of wondering what your expectations are and sort of next steps for some of the key ex U. S. Countries like UK and France? I guess I'm curious is there just a fundamental difference in reviews on the value of disease modifying medicines or do you believe that with additional negotiations or with additional data, and perhaps triple combo data, you can sort of get get past this.
And this is resolvable. Thanks.
Yes. So, Brian, thanks for the question. So, in terms of specifically where we are in the UK and Australia, the two countries that you reference in the UK. Obviously, we recently received a counter offer from the NHS. We and many in the CF community believe that offers significantly undervalues, both our current and our future medicines.
And so we're not able to, set that offer. And in terms of next steps, we're looking to meet, with those of the head of the NHS and indeed the Minister of Powell to try and progress those discussions, because as I said, we're certainly not going to give up on fighting for access for those patients in the UK who's been waiting too long. In Australia, ORKAMBI, both the 6 to 11 indication and the 12 plus indication were reviewed by the Pharmaceutical Benefits Advisory Committee feedback in Australia, which is the body that reduced both clinical effectiveness and cost effectiveness. We're yet to receive their formal minutes from that meeting. And when we do, which will be in the next few weeks, then that will help us determine what the path forward there is.
What I would say is that, as I referenced in the answer to an earlier question, we have successfully secured pricing and reimbursement in a number of other countries in Europe, where now thousands of patients are able to access ORKAMBI. And importantly, we were able there to strike pricing reimbursement agreements, which we believe give us a fair price for the level of clinical benefit and innovation that ORKAMBI delivers. And we need that, that fair return to enable us to continue the journey in CF and other diseases, as Jeff was describing in his prepared remarks. So we are going to continue to fight for access in the UK and Australia to get access for those patients who've been away taking too long, but we can only
do that at a price which fairly reflects the
value of the medicines and the benefit they bring for patients.
Operator, we will take 2 more questions.
Certainly. Our next question comes from the line of Geoff Meacham from Barclays. Your question, please.
Hi. This is Olivia Bray on for, Jeff. Thanks for taking the questions. Just a question on the triples. Are you expecting 659 or 4 for 5 to yield by a result?
In one population over the other. And maybe as a follow-up, have you given any more thought as to what your strategy and objective is in triple combinations beyond these 2? Thanks.
Yes, this is Jeff. So let me to reach those. Just to take you back to the phase 2, Dave, you'll remember that 4, 4, 4, 5, and 6, 5, 9, in fact, all four of our triple combinations were remarkably similar. In fact, the consistency and the similarity of the data was quite striking. That's really the only data we have to make assumptions about phase 3.
That's why we're doing the phase 2 studies. But based on that data, we would expect 659 and 445 from an efficacy standpoint to be quite similar. Beyond these two, your next part of the question, I've said before, we continue to develop additional next generation correctors and it's actually fairly remarkable. We have many of them, and they continue to improve over time. And so that we're left with a sort of a good problem, which is which of these do we
take into the clinic? We obviously can't take
them all because we have 20 or 30 of them point that are better, which of them will be taken about how much better do they have to be. Our goal is to get everyone to carrier levels. And as you know, even with 659 or 445 are quite close in some populations. And so you should expect to see 1 or more of them come into the clinic we're just trying to choose which one and put a threshold on for how much better it needs to be before we bring them in. But then moving forward pretty quickly, so I think you can expect to see additional compounds entering the clock.
Tulary Casono from JP Morgan.
It's Carmen on for Corey. Thanks for squeezing us in here. Most of my questions have been answered, but Just one more. You've recently seen some data from some of your competitors and we're lucky to see more in the coming months. What are your latest thoughts on competitive positioning of your franchise and what continues to give you confidence in maintaining a leadership position?
Thanks.
Yes, thanks for the question. It's obviously an important one. As you know, we don't comment on individual competitors. You'll have to ask them about their programs and we've seen some of those results recently. I would comment on where we think we are an second part of the question, what gives us so much confidence?
We're really pleased with where we are. As I said in my prepared remarks, it's actually I've been in fitness industry for 35, 40 years, and I don't think I've ever seen 2 molecules progress from synthesis into well into phase 3 or completion of phase 3 in 2 to 2.5 years. It's been a really remarkably accelerated journey for these 2 triple combinations. And so we feel better and better about our competitive position both based on the data, based on the speed at which we're moving. As I said, well, we should be in a position to file an NDA in the U.
S. And also an application shortly thereafter for the best of these triple regimens that can provide therapy that up to 90% of patients at very high levels of efficacy. We'll get with confidence. It's really our 20 years of understanding of the biology of this disease, the ability that we've run our HPD assay, several 100,000 times and they predict the human results virtually perfectly quantitatively. That's held up with the triples as well.
And we're making better and better triples as we go. And so we're actually getting very close to our goal of getting the carrier levels. And as you know, when your carrier levels, carriers don't get the disease. So our goal is very simple. Get a medicine of triple medicine to patients that provides carrier levels for everyone, get it to them as young as possible,
And
we believe they won't develop cystic fibrosis as we know that is used for that.
And we're well down that road.
Great. Thanks.
Thank you. This does conclude the question and answer session of today's program. I'd like to hand the program back to Michael Cartridge for any further remarks.
Thank you very much. Everybody, for joining us
this evening. The Investor Relations team will
be here. If you have
any additional questions, we do have any talking. Thanks.
Thank you, ladies and gentlemen, for your participation in today's conference. This does conclude the program. You may now disconnect. Good day.