Insmed Incorporated (INSM) Earnings Call Transcript & Summary

September 5, 2025

US Health Care Biotechnology Company Conference Presentations 35 min

Earnings Call Speaker Segments

Tiago Fauth

Analysts
#1

All right. Thanks, everyone, for joining us. I'm Tiago Fauth, biotech analyst here at Wells Fargo. We're joined today by Insmed for a fireside chat. We have Sara here. Thanks so much for joining us. Appreciate it.

Sara Bonstein

Executives
#2

Thanks for having us. I really appreciate it.

Tiago Fauth

Analysts
#3

I'd like to just open up with just some broad intro remarks. We have a lot of detailed questions that we usually get from investors that we want to walk through. But just overall, it's a very different setup for the company. A lot of moving parts. So perhaps what is the current state of affairs, and then we'll dive in.

Sara Bonstein

Executives
#4

Yes, sure. I'd be happy to provide a brief overview. And again, thanks for having Insmed here at this conference. So one takeaway that I'd ask everyone to kind of walk away from today's discussion is while we have been able to show tremendous growth from a value perspective as well as hope for patients over the last 12, 18 months or so, the potential that we have from here, we think could be just as great, if not greater, than what we've been able to show. And let's kind of walk through why I have conviction in saying that. So we have 3 programs that are in clinical/commercial stage. Actually, we have 4, but we'll focus on 3 right now. ARIKAYCE. ARIKAYCE is our kind of our diesel engine that has been our commercial product for now 7-plus years. And in each of these programs, you'll see this unique opportunity where we have the current indication or area of focus, but we also have the ability to grow that. And ARIKAYCE, while it's continued to be successful year-over-year, double-digit growth, we have the potential to have that label expansion, and that data will read out first half of next year, and we can kind of dive into that and have the ability to be a $1 billion-plus program. As you think about brensocatib, now we can call it BRINSUPRI on a commercial basis, obviously, showed tremendous success in both the WILLOW and ASPEN programs, both of those being published in the New England Journal of Medicine and now most recently, having FDA approval for brensocatib now under the commercial name of BRINSUPRI. This is the first-in-class, first-in-disease drug to now non-CFBE or bronchiectasis patients now have the opportunity to go into their physician's office, get this diagnosis and actually have something available to treat them. So that's absolutely tremendous. We said $5 billion peak sales opportunity for BRINSUPRI and NCFB. But what I think is just as exciting is the opportunity that we have in some of the other indications like chronic rhinosinusitis without nasal polyps and HS. And those data readouts will be forthcoming, and we believe can be very significant value inflection points from company perspective and obviously, potential huge impact on patient assuming success. And then our third program, treprostinil palmitil inhalation powder, again, a program we developed in our own research lab similar to ARIKAYCE. We put out positive PH-ILD data last year. And then most recently, back in June, we put out positive Phase II data in PAH. And so that gives us a ton of conviction in that program. That's what we call our sort of 3 for 3, the successful ARISE data in ARIKAYCE, the successful ASPEN data in brensocatib and the successful data in ILD and PH. And again, we have the ability to be 3 for 3, unlike many in this industry. So we are humbled by that, that we've been able to produce that, but then also have the ability to multiplex across various indications and continue that value creation to hopefully be able to come out to you guys all in the coming quarters with additional inflection points. So happy to dig into any of that. We do have a lot of cash also. I wouldn't be a CFO unless I mentioned it. So thank you to all of you that have supported us through this journey to allow us to have about $1.9 billion in cash as of last reported to help fund our business.

Tiago Fauth

Analysts
#5

Perfect. And I know there's a lot of focus on the BRINSUPRI launch, but perhaps since we had news that are relevant to you guys this week, let's start with TPIP. So again, early this week, you had United reporting positive TETON data in IPF that could unlock another large commercial opportunity. So can you just recap how you guys are framing peak opportunity for TPIP previously? And what has changed since, I guess, you had a couple of data sets. You had your competitor with a positive data set in a large indication. So what's the current state of affairs?

Sara Bonstein

Executives
#6

Yes. Yes. TPIP, we're obviously really excited about this program. We believe what you hear from the medical community that this could be the prostanoid of choice, obviously, assuming success in our Phase III programs that will be up and running very shortly. So let's take each in turn. What we've said previously is we believe TPIP, this was before, obviously, the data that we put out most recently with PAH in June and before the United readout. What we said in PAH and PH-ILD, we believe that could be a $2 billion peak sales opportunity. As you reflect on the strength of the PAH data, 35% PVR, 35-meter 6-minute walk, those were sort of unprecedented type results. We obviously know we owe you sort of a refresh on those numbers, and that did not include the potential for IPF with now having the first of the TETON data is what we have committed to is we would look to move quickly into a Phase III program. And again, that $2 billion peak sales number does not include IPF and does include, I would say, a little bit of a different TPP for PAH.

Tiago Fauth

Analysts
#7

More conservative product...

Sara Bonstein

Executives
#8

Yes. So some free options in that number for sure.

Tiago Fauth

Analysts
#9

Got it. And thinking about next steps and gating steps, everyone is super excited. So how quickly can you actually get into clinic? I know it's not that easy as an operator. So for the 3 indications, again, I guess you just flipped the data cut. So I'm assuming you guys are already in the drawing board, but what about for PAH, ILD and PAH? Any upcoming regulatory interactions that need to happen? Is it more about just finalizing trial design? Regulatory, like what's the lineup here?

Sara Bonstein

Executives
#10

Yes. So let's go through each. There's a lot of indications there. We'll start with ILD. So what we've committed to is to start that Phase III program by the end of the year. I'm happy to say we remain on track to start that program by the end of the year. So excited to kick that off and provide more options for patients in that clinical setting. For PAH, we've shared that we have our FDA meeting as planned as we normally do after a Phase II data readout in October. So again, we'll share more data and feedback once we have that interaction. But our expectation is we look to start that study in 2026. And then IPF, we were obviously wanted to see the TETON data, but we have been thinking internally what that could look like, and we'll be putting that in here, in motion and going through all the operational steps. We won't -- I won't be here committing to a specific time line on IPF from a Phase III perspective, but we would look to move directly into Phase III and do that as soon as possible.

Tiago Fauth

Analysts
#11

Got it. And we get some pushback just in terms of enrollment dynamics, right, especially for PAH, where polypharmacy is kind of standard of care, and there's a lot more moving parts, I guess. But you guys had success with an uptick in enrollment on the PAH trial post publishing or at least disclosing blinded data, right? So any concerns from a feasibility perspective across any of these indications? Again, the standard of care is slightly different. The current penetration for therapeutics is different. So what are some considerations from an execution perspective?

Sara Bonstein

Executives
#12

Yes. Yes. So it's absolutely fair. We were not known as a PAH company, right? We have very strong and established relationships in the NTM market as well as the bronchiectasis market with the pulmonologists and ID docs. We have spent a lot of time over the last handful of years establishing those relationships. And part of the reason that we put out the blended blinded data, which I know everybody loves last year, was to arm conversations with the medical community. And what you saw in sort of the uptick last year in enrollment, once we put out that blended blinded data, you saw a significant uptick in enrollment in the PAH program. And now with the strong PAH Phase IIb data in hand, we believe that we are becoming part of the conversation and a very important part of the conversation on the medical side of potential opportunity for patients. What physicians say is the more drug they can get into the patient, the better if you look at the sheath models and all the past data and the prodrug formulation of TPIP, we believe, provides that opportunity for patients, and we're eager to get those Phase III started.

Tiago Fauth

Analysts
#13

Got it. Perfect. Let's move on to BRINSUPRI. Again, what is the #1 question that you guys are getting right now as you're heading into the launch, right? I'm assuming we're not going to be able to talk about any metrics. For us, it has been mostly around access, right? And you guys have been very proactively outlining your strategy. What are some of the steps that you guys have taken to ensure that seamless launch and broad access, I guess?

Sara Bonstein

Executives
#14

Yes. So we did invest early in this potential launch, which has now turned into a launch. And again, thank you to the investment community for giving us that latitude to be able to invest early. And so we invested early on a couple of fronts. One on -- as you think about the medical affairs strategy, we invested early in our medical science liaisons and our medical outcome liaisons to help with that disease state awareness. And we felt that, that was critical knowing it was a first-in-class and first-in-disease drug to get that education out there. We also invested early in our field force expansion. So we had an existing field force that called on pulmonologists and ID docs for ARIKAYCE in the NTM space. We expanded that back in October of last year. They were in field. So gosh, almost 10 months before launch. And we added about 120 reps, and that gave us the ability to call on every pulmonologist in the United States as well as the appropriate ID docs and some other kind of specialties to cover this patient population to establish those relationships to do the appropriate and compliant disease state awareness. So that investment, we think, was absolutely critical for when we got that approval on August 12 from FDA. And then third, and I think gets to the heart of your question on that market access side. So we can have the best trial results and have approval from FDA and have a clean label. And if you don't have access for patient, you're not going to be able to have the impact on patient, which is why we do what we do at Insmed is to have an impact on patient. And so we invested very early in some in-house expertise on market access and building out that team. And then we also invested in our 2 sort of field-facing market access functions, field access managers and case managers. And those are the folks that help on the white glove "service" for the patient side as well as the support on the back office side to help through the medical exception process, which we know very, very well from ARIKAYCE and help that paper process. And then on the payer side, the conversations that we had was on this frictionless launch. How can we make it as straightforward for the treating physician from administrative perspective? And so the conversations that we had were what is the appropriate targeted rebating strategy that we could put into place to allow for this frictionless launch, which would mean physician attestation versus getting sort of bogged down with tons of paperwork and medical records and all those good things. We felt like having access on day 1 on the initial script as well as the reauthorization. This is a chronic therapy that they will take potentially for the rest of their lives. Having that sort of all worked through was going to be most impactful for patient, for access and that would then result in the most positive uptick from a revenue and value creation perspective.

Tiago Fauth

Analysts
#15

So if you were to break down like payer mix and again, some of those considerations, so it seems like at least the paperwork is not going to be as cumbersome. It may not require the -- it may not require the super extensive medical history. So I guess what are some of the gating steps then for a large uptake, right? Because you hear on both extremes, you hear, hey, this could be huge. There is definitely an unmet need. I've heard from physicians that there's a lot of excitement. What are the gating steps on why this actually could have, again, a super strong I&I launch, but I've heard some fairly bullish near-term metrics. Is it more about capacity, physician education, the more community center? Again, we usually talk to specialists, there's a bias there. What are some of those pushes and pulls?

Sara Bonstein

Executives
#16

So a couple of things there. A couple of pushes and pulls to kind of call out. If you think about how many appointments there are at a pulmonologist's office, right? So a pulmonologist sees bronchiectasis patients as well as folks with a lot of other similar type of conditions, asthma, COPD and so on and so forth. And so there's only so many appointments in a day. I would anticipate a patient will need to see their pulmonologists for the majority of scripts that are written. So that is one sort of getting through the funnel. Obviously, once the script is written, it needs to go to the insurance provider and needs to kind of go through the channel. We have tried to make that as seamless as possible. I will just take this opportunity to comment on some of the information we've shared previously on third quarter. So obviously, approval on August 12 from FDA. If you look at ARIKAYCE as an analog, it took about 4 weeks to get through the channel. We were ready on day 1 with our reps in field promoting on drug as similarly as we were with BRINSUPRI, but it does take time for a specialty drug to get through the channel. So that is something just to be very mindful of. I won't speak specifically on the nuances that obviously have occurred since August 12. But what we have said previously is you could expect a handful of weeks of revenue in Q3. And so just be mindful of that. Q4 will really be the first full quarter of being able to see revenue and performance from this product. The other just some shining lights that I'll kind of share is we did have a disease state awareness website. We had about 1 million unique sort of hits on that site. We had about close to 70,000 self-identified individuals that said, I am a patient that has bronchiectasis. So you can see an active community, an active patient population. But again, it takes time to get patients funneled through their pulmonologists and scripts in hand, drug sent to their house, all that good stuff.

Tiago Fauth

Analysts
#17

Got it. So again, Q3 results, we're going to hear on the earnings call late October, early November. And then you generally preannounce revenues. I think is it fair to assume as the last several years in January at a conference, we might hear about it.

Sara Bonstein

Executives
#18

So a couple of things. What we've committed to from a metrics perspective is during this launch period before we provide formal guidance, we would provide number of new patient starts and cumulative number of prescribers on a quarterly basis for BRINSUPRI. I think it's -- if you look at our past practice, not committing to anything, but if you look at our past practice, we have historically preannounced revenue for ARIKAYCE at a health care conference that happens in January. And so if you sort of follow that cadence, you can assume that the first real kind of meaningful quarter of revenue for BRINSUPRI could come at that point. And we will provide those specific metrics that we talked about, cumulative number of prescribers and new patient starts through the launch period once we provide full year revenue guidance, which I would not anticipate in '26, not committing to when we would provide it, but would not anticipate that to happen in '26. Once we do provide that, then we let the revenue numbers kind of stand for themselves.

Tiago Fauth

Analysts
#19

Got it. Just a follow-up on the access side of things because a lot of investors are really honed in on the 2 exacerbations or more. How does that actually impact that initial 250,000 patients that everyone estimates that would fit that criteria. So Again, how formulaic has been when you talk to payers about the specifics around exacerbations and documentation, again, you mentioned mostly physician attestation. So I guess, could you actually see potentially broader adoption than originally the Street is modeling? I'm just trying to think about some of the puts and takes on the access side. It does not feel like it's an overly burdensome process to actually get this reimbursed.

Sara Bonstein

Executives
#20

Yes, yes. So a couple of thoughts. If you look at the claims data, so there was already an ICD-10 code established for this disease indication, which was really helpful because there was claims data. There's about 500,000 diagnosed patients in the U.S. with NCFB. Based on the data, we believe and based on some published literature, we believe about half of them have had 2 or more exacerbations in the prior 12 months. To be clear, the label that was provided by FDA is a very broad label. The label allows physicians to choose physician discretion on which doses, is it 10 and 25? So the medical community was very happy with the broad label. It does not preclude that it needs to be 2 or more or anything like that. It is a broad label. In the clinical section, it references the inclusion/exclusion criteria from our Phase III program, which was 2 or more exacerbations in the prior 12 months. So the conversations with payers are really around this is what you studied in a clinical setting. This is what we will support from a reimbursement perspective. The importance of that physician at a station is really critical and the disease state awareness and speak up early and often campaign that we did throughout the prelaunch period was also very critical. So physicians had a better line of sight to when their patients were exacerbating. The typical patient demographic here is elderly population, typically more female. They're typically the people that like to take care of everyone else and not need to be taken care of themselves. They aren't always the ones that speak up and say, "Hey, there's something going on, they kind of brushed under the rug." So that's why sort of some of that pre-disease state awareness work was very, very important. So we're making a physician attestation. That's up to the physician and the patient as to sort of that process I will just kind of note the safety profile, obviously, here is attractive, and it is a small molecule. So it is not overly onerous from a patient perspective.

Tiago Fauth

Analysts
#21

And that actually segue into my next question, which is related to peak sales opportunity and again, long-term compliance and adherence to therapy, right? So questions that we get are related to, again, if the sell side is modeling this to be a $5 billion to $6 billion opportunity in bronchiectasis, that is one of the most successful single indications I&I launches ever. And a few folks are skeptical around that, given that a lot of drugs are launched in I&I markets, they usually do hit blockbuster potential, but perhaps not as high as the Street is currently modeling for BRINSUPRI and bronchiectasis. Competition, I think, is the biggest factor in trying to reconcile those 2 ideas. But again, what are some factors that make BRINSUPRI actually that special and those lofty metrics achievable?

Sara Bonstein

Executives
#22

Yes. So we put out some metrics, some launch metrics on what really, really great launches look like, and you saw those just -- it's obviously not formal guidance. And then I know folks have studied what really good I&I type launches look like. A couple, I think, unique factors with BRINSUPRI. There is nothing available for these patients today. So today, when patients go into their doctor's office, they explain their symptoms, the doctor may or may not even give them a CT scan. A CT scan is a definitive way to diagnose for this condition, but they may not even give them a CT scan because they're not going to be able to treat them until August 12 any differently because there was nothing approved. And so I think that goes to another sort of layer here on that undiagnosed or misdiagnosed patient population. But let's focus on the diagnosed patient population for right now. If you think about from a patient perspective, they were told, I have this condition, but I can't do anything for it. So continue with your airway clearances, you're going to have to go antibiotic, you might have to get hospitalized. So now patients have the first and only available medicine that can reduce exacerbations. And the other, I think, really important piece that you saw in the data is depending on the dose and the statistical powering and all that kind of stuff, some quality of life. And so patients feel better when they're on this. And so a patient that is that has bronchiectasis and exacerbates, they sort of -- they put their life on hold. They don't go to family events. They don't plan things. Their exacerbation can last. It's not just an hour. It can last days, it can last weeks. So it really is life altering for these patients. And so between the way of administration, nothing else available, the quality of life, they feel better, reducing exacerbations, exacerbations create permanent lung damage for these patients. And then that kind of comes into this vicious sort of cycle of they had permanent lung damage, their lungs, they're more susceptible to having a future exacerbation. If they could stop that or they can halt that or alter that course, they're going to want to do that.

Tiago Fauth

Analysts
#23

Got it. Fair enough. Let's move on to some additional indications you guys are pursuing there. So again, chronic rhinosinusitis without nasal polyps, data by year-end, right? So mechanistic, there's some rationale there. There are very few comps, I guess, tons of failures in that space. Why did you guys decide to pursue that? What success look like? Let's kind of unpack that opportunity.

Sara Bonstein

Executives
#24

Yes. Yes. CRS without nasal polyps, this is one of the things I'm most excited about. There's a lot to be excited about, but it's one that I'm super excited about. So let's take a step back and kind of walk through the trial, the design, what we're trying to do there. And so if you think about chronic rhinosinusitis without nasal polyps from a -- not a scientist, obviously. So you can view it very similar to its bronchiectasis in the lung. This is very similar, but in the nasal capacity. And so what we're studying here, it's a thorough, very Willow-like kind of Phase II. It was designed for 270 patients. We overenrolled at 288 patients. It's a 3-arm study, 1:1:1 randomized placebo, 10 milligram and 40 milligram. So we studied a higher dose, and it's 24 weeks in duration. And what we're looking here for is change in total sinus symptom score. So what is that? What does that mean? There's 3 components of the total sinus symptom score. There is pain or pressure in your face. There's nasal congestion and there's discharge. Those are the 3 components that you're studying. And each of the individual components are on a scale from 0 to 3. So it's a total scale of 0 to 9. And patients have to have a 5 or greater coming into the trial. They can put on a course of steroids to make sure they're kind of stable on steroids, then they check them again, make sure they're still at a 5. So these are more the severe end of patient. And then what we're looking for, for success would be a 1-point placebo-adjusted change. is what we would look for. And the way we measure that is the last 28 days of the study, every day, they give us their score, we average it. And then we see do we have -- what is the change and we would look for a greater than 1 change placebo adjusted or 1 or greater.

Tiago Fauth

Analysts
#25

And again, in the past, you guys have said that you are seeing, at least on a blended blinded basis, something that is exceeding that 1 point, right? What's the latest in terms of the blended blinded data that you can share? And perhaps, again, how do you actually assess that, right? So again, for PAH and the blended blinded data, you had a lot of comps not as many comps on this indication. We were able to find only 2, 3 studies that could be relevant here. So how should we interpret the data that you know so far and how that's trending?

Sara Bonstein

Executives
#26

Yes. Yes. So a couple of things there. So we look at blended blinded data internally because if you don't see any change on a blended blinded basis, that should be a red flag, right? And so we look to see, are we seeing something? We don't know where that's coming from. We don't know if that's coming from placebo patient, drug patient, but we need to see some level of change. And so last time that we've spoken about this, we've said we've seen a north of a 2-point change. Again, that's not determining to say that those are drug patients, but we are seeing a north of 2-point change. So that gives us encouragement that -- we are seeing change. Now we obviously need to unblind it and see is that change coming from those that are on drug. The other piece I would just comment on is there is an approved nasal steroid. They saw, I want to say, about 0.9 -- 0.7 to 0.9 depending on which dose change, placebo-adjusted change. So that helps to hopefully put it in frame of reference. And this is a significant patient population. If you look just again in the U.S., CRS without nasal polyps, there's almost 30 million patients. I think it's 29 million patients in the U.S. that have CRS without nasal polyps. Now about 3 million of those are steroid nonresponders and 200,000 patients in the U.S. alone each year get surgery from this condition. So it's definitely an underserved patient population. And we're just as eager as you all are to turn over that data, and we remain confident that we'll be able to have that data in-house before end of the year.

Tiago Fauth

Analysts
#27

Got it. Pivoting to hidradenitis suppurativa. Again, a lot more investor skepticism there, understandably so, given the check of history in the indication. You built in an interim analysis on your trial. So can you talk about next steps? Again, we know it's neutrophil driven. There's definitely a neutrophilia component of the disease. Hard to know exactly how the biology is going to play out, but what is the setup there?

Sara Bonstein

Executives
#28

Yes, yes. So we were slightly more cautious on this indication, while we believe in it. And as you said, obviously, neutrophil driven. We don't have a lot of good animal models. And so we wanted to be very thoughtful as we design the program, both from a patient perspective as well as a resource allocation perspective. So this is a little bit of a smaller study than the BiRCh study that we just spoke about for CRS. This study we call CEDAR. It's about 200 patients, 204 patients, similar in design, 1:1:1 randomized, placebo, 10 and 40. It is a 16-week study. We sized it and powered at 0.1 because we wanted to be able to have it be a little bit smaller and get information a little bit sooner. And we also built in a futility analysis at week 16. While patients will go on for a total of 52 weeks, at week 16, there is a cut of data after 100 patients get to week 16, there will be an independent board that will unblind the data. We will not see it. It will be an independent board that unblinds the data, and they will determine should we proceed or not proceed. There will be no p-value. There will -- it will just be a signal of efficacy. And so they will just tell us yes or no. So that's all we will get to be fully, fully clear, and we'll look to share that information first quarter of next year. Once we share that information, then we'll provide guidance, assuming that is go to move forward on the timing for the full 204-patient study. But yes, eager to see that. That one is the one I'm a little more still excited about, but cautiously optimistic as we enter that.

Tiago Fauth

Analysts
#29

I think that's fair. And again, in terms of patient numbers, both for CRS, you guys have mentioned in the past, this could be bronchiectasis like to an extent. I think even for HS, most folks think that a new therapeutic could get substantial revenue opportunity. So what is the revenue opportunity across some of these indications? It's starting to add up.

Sara Bonstein

Executives
#30

Yes, starting to add up for sure. Yes. So all we've committed to externally is the $5 billion peak sales for NCFB. What we have said is for CRS without nasal polyps, we believe that could be just as large, if not larger, from a revenue opportunity perspective. More to come on that. We know we owe you some education on the TAM and the market opportunity there, but that could be very, very significant. HS on its own is also significant, hundreds of thousands of patients in the U.S. alone for HS. So not willing to commit to a peak sales number for either of those today, but they are very significant. And as you sort of stack up ARIKAYCE, the label expansion, brensocatib, TPIP, some of the early-stage research, get into some big numbers.

Tiago Fauth

Analysts
#31

Fair enough. And again, you have been talking about DPP1 inhibition as kind of a new class, right? And we're starting to get some more questions on the follow-on molecules. Some of that is life cycle management of brenso, but also you've alluded to maybe exploring much larger indications. So what's the rationale there? What's the product profile? When should we start to see some progress towards that? What are some of the gating steps?

Sara Bonstein

Executives
#32

Yes, yes. That's -- like CRS, the follow-on that work that our research team has done is also something that is really excites me and not something we've talked about as much externally because of everything else that we have going on. But hold on for 2026, you'll hear more about that. But what I can say is on the heels of the WILLOW data that we put out in very, very early February of 2020. It was actually my first day in the office. It was a really cool first day. But on the heels of putting out that WILLOW data, we double down on the mechanism. We really believed in and still believe in the DPP1 mechanism and our research lab has done a ton of work. So a big shout out and kudos to all of our great scientists back in New Jersey and around the globe that have done this work. And so we've developed over 800 different follow-on molecules, and we'll look to move forward the first of those into IND candidate very soon. So you'll hear more about that in 2026 and more significant market opportunities potentially, maybe something like an RA and those types of -- and all follow-on DPP-1s would be fully owned by us.

Tiago Fauth

Analysts
#33

Perfect. ARIKAYCE, again, I feel back because now it's always the last item on the list, right? But again, at least when we hear from physicians we talk to, on the low end, they think their revenues could double. And on the high end, we hear even as much as 5x the refractory revenue opportunity. Yes. Can you just talk about the frontline expansion, expectations for that? We have data upcoming. There was a lot of skepticism you'd be able to show a clear benefit in PRO. I think that's kind of gone, right? Like the data has been pretty compelling so far. So I know it doesn't get as much airtime, but again, not an immaterial part of the business.

Sara Bonstein

Executives
#34

Yes. Yes. I appreciate you calling that out because it has been really our mainstay and has helped us have a lot of the success along with the support from the investment community that we've had with the other programs. So ENCORE, that readout will be first half of next year, and that's not something to overlook. What we were able to show in the ARISE program, which was the first of the 2 label expansion programs is we were able to show a meaningful difference on both the patient-reported outcome tool. So this is an 8 question utilizing the quality of life bronchiectasis questionnaire, 8 questions, and we are powered for ENCORE to show a 4-point difference between groups, and we showed north of that in the ARISE program, also if you think about culture conversion. So FDA cares about PRO, field function survive. We're also looking at culture conversion. The Japanese regulatory authorities are focused there as well as the patients and payers and physicians in the U.S. And what we were able to show in ARISE was north of a 16 percentage change there. And so really excited to be able to unblind and see the data in ENCORE first half of next year. You'll get both the PRO data, you'll get the culture conversion data. And from a patient number perspective, 12,000 to 17,000 patients in the U.S. alone with refractory NTM. That's about 100,000 all of NTM. So we've set a $1 billion peak sales opportunity for ARIKAYCE. We obviously are a mainstay in that community, the only approved product and the only with a high recommendation for use in international treatment guidelines for refractory. So more to come there.

Tiago Fauth

Analysts
#35

Fantastic. I know we only have about a minute left, but always the fourth pillar. We don't get to talk that much about it. But just again, news flow is probably going to be a little slower than some of these other programs. But overall, what are some of the next steps for the fourth pillar?

Sara Bonstein

Executives
#36

Yes. So we did announce that we dosed our first patient in DMD. So that was, again, a humbling experience and really eager to see what that program could potentially do for those boys and those families. We will also look to move forward additional programs in our gene therapy, ALS would be the next one that would move forward. And then we obviously have the follow-on DPP1. So you'll hear a lot more next year on the early stage sort of fourth pillar, but yet another value inflection creation point for this story. And 2026, hold on to your hats because '24 and '25 are really big important years, but '26 is going to be a great one as well.

Tiago Fauth

Analysts
#37

Awesome. Fantastic.

Sara Bonstein

Executives
#38

Thank you. Thank you all.

Tiago Fauth

Analysts
#39

Thank you so much, Sara. Appreciate it.

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