Insmed Incorporated (INSM) Earnings Call Transcript & Summary

March 3, 2025

NASDAQ US Health Care Biotechnology conference_presentation 35 min

Earnings Call Speaker Segments

Lyla Youssef

analyst
#1

Thanks, everyone, for joining us this morning for the Insmed fireside chat at the TD Cowen Healthcare Conference. Thanks, everyone, for joining us again this year. Nice and bright and early. We have the CEO of Insmed, Will Lewis, with us this morning as my [indiscernible].

William Lewis

executive
#2

I'll take this opportunity to say congratulations on your [indiscernible]

Lyla Youssef

analyst
#3

Thank you. Thank you, Will. And I appreciate -- well, as long as you bring it up, I'd like to thank all the clients here for their votes for the TD Cowen team and look forward to continued excellent relationship.

Lyla Youssef

analyst
#4

That was Will's way of avoiding me asking how the FDA review is going for brenso. That was a nice way to try to distract me. Okay. So how is the FDA review going? I think there's probably sort of less concern amongst investors on the data and maybe more concern now on the process and internal FDA flow and capacity and how you guys think about your meetings going into your August 12 PDUFA?

William Lewis

executive
#5

Yes. For purposes of this conference and for anyone who's listening, the one message I would leave you with is that the FDA review process is going extremely well. Everything is going as expected or ahead of schedule, which is remarkable given the backdrop you were just describing. And I think it's worth spending an extra moment on that backdrop. Everybody has a concern about whether or not different government agencies may have people leaving or cut or what have you. Remind everybody a couple of things. The first is that the PDUFA fees that we pay cover the cost and the salaries of those who are doing the NDA review process. So to the degree anyone in any part of any of these agencies is insulated, we are to the greatest degree possible. The second thing I'd observe is that the staffers themselves, we haven't seen big turnover in our area. And these are very dedicated people. And what's really interesting to them as employees, if we can step into their mind for a moment, is to be a part of novel medicines that are going to make a big difference where heretofore nothing has happened. And that describes to a T, what is going on with brensocatib in the potential approval process for bronchiectasis. There is nothing approved to treat this condition, and this is a novel mechanism of action with an entirely new approach to inhibiting the inflammatory cascade that brings upon these patients, their pulmonary exacerbations. Those are essentially like a small heart attack for the lung. The permanent damage and the ability to reduce the numbers of those to prevent the onset of those, to delay the first arrival of those and as we have shown statistically significantly in our ASPEN study is all an extremely exciting turn of events for this area. When the WILLOW study came out, the Phase II data at the American Thoracic Society, this was during the COVID era. So this is done over a webcast, but there was a physician who described this as the holy grail of pulmonary medicine. And today, we sit in a very enviable position, I think, with the review of that data post the ASPEN and WILLOW trial now going at a very appropriate pace. We're very excited about what we're seeing. The PDUFA date has been set for August 12.

Lyla Youssef

analyst
#6

No Ad Comm.

William Lewis

executive
#7

no Ad Comm, priority review. All those things have fallen into place. So we're pretty excited.

Lyla Youssef

analyst
#8

How should we be thinking about the label language from a perspective of easing insurance coverage?

William Lewis

executive
#9

Yes. So I think...

Lyla Youssef

analyst
#10

And especially on like whether exacerbations or frequent exacerbations will be included in indications.

William Lewis

executive
#11

So our expectation, although we obviously don't know at this point, is that the label will not include a reference to prior exacerbations or exacerbations as the hurdle, if you will, for who's in or who's out. This is for the treatment of bronchiectasis. We'll see whether that reads through. But in any event, what we also want people to understand is we fully expect the market access world to limit the use of this drug to patients who have met the entry criteria of our Phase III study was those who had experienced 2 or more exacerbations in the last 12 months. As a consequence, a lot of our effort over the last 2 years through our medical science liaisons and our interactions with physicians in these academic settings has been to emphasize the importance of looking for exacerbations in your patients, documenting those events in the medical records and also approaching the patient community and encouraging them to speak up, if they experience exacerbations, to their physician. A lot of this hasn't really happened to the greatest degree it could have over the last decade or so because there's nothing approved to treat the condition. So the ability to document that you're having these exacerbations or that they're increasing or decreasing is really irrelevant in a world where the physician has nothing they can do about it. What brensocatib promises, if it's approved, is the first-ever medicine that would have an impact there. So documenting that, speaking up about that, those are really important activities that we've been trying to encourage for both the patients and their physicians.

Lyla Youssef

analyst
#12

So the AV team in the back [indiscernible] up here because I will not stop.

William Lewis

executive
#13

[indiscernible]

Lyla Youssef

analyst
#14

Going back to that conversation, how -- the flip side of it, how do payers perceive the value proposition for brenso? How are they viewing the cost savings around managing exacerbations?

William Lewis

executive
#15

Yes. So we know that exacerbations vary in their severity. Some result, in every case, in a modification of medication and intervention by the physician. They can result in hospitalization. We saw a trend toward improvement in that in the WILLOW study and the ASPEN study. That was very encouraging. But I think the right way to think about this is not as a swap out of value. You're not going to get dollar-for-dollar improvement by treating this condition versus leaving it alone. But it is the first-ever approved medicine. The vast majority of these patients are Medicare, something in the order of 60%. So this will have to be covered by Medicare as the first and only medicine approved to treat this condition. But what was really exciting about the ASPEN data from our point of view is not just the reduction in percent -- percent reduction in exacerbations, which was a clinically relevant level, nor was it the delay on the onset of the first exacerbation or the percentage of patients who went exacerbation free for a year. All of those were statistically significant. But the surprising data at the 25-milligram dose was the preservation of lung function that was not expected. And both from the market access point of view and the physician perspective, particularly among the experts.

Lyla Youssef

analyst
#16

Because the payers are picking up on that.

William Lewis

executive
#17

The payers are picking up on that as a very important aspect. And so we think that's a pretty exciting thing to be able to show a statistically significant level.

Lyla Youssef

analyst
#18

So pulling that together, what does that mean for your expectations for prior authorizations? Are chest CTs going to be required for diagnosis just because they can be a gating factor in securing a CT?

William Lewis

executive
#19

Yes. We framed out the addressable market at 0.5 million patients in the U.S. Roughly half of those we assume, based on our data and analysis, have had 2 or more exacerbations. But all of those patients are definitively diagnosed with bronchiectasis, which includes a CT scan.

Lyla Youssef

analyst
#20

So they're sitting there with the paperwork.

William Lewis

executive
#21

They're sitting there with the paperwork ready to go. So what we have to do at this point is to go and find the additional patients who may also be bronchiectatic but have not yet had that CT scan or that definitive diagnosis from the pulmonologist. We think that population could be much larger than the existing prevalent population that is already diagnosed and ready to go today. To be clear, our estimates contemplate only the diagnosed population of patients in the U.S., Europe and Japan.

Lyla Youssef

analyst
#22

Do you think there'll be prior auths past that CT diagnosis?

William Lewis

executive
#23

So I think there will be a prior authorization that will require a definitive diagnosis of bronchiectasis, which does include a CT scan. But we are working with the -- in our work right now with the market access world to try to ensure that the attestation or the check the box, if you will, that the patient has bronchiectasis and meets the entry criteria of the study, 2 or more exacerbations that, that can be done by the physician quickly without providing all the documentation you...

Lyla Youssef

analyst
#24

Not like an exacerbation document to treat exacerbation.

William Lewis

executive
#25

So if the physician can attest to the exacerbation, that's the ideal prior authorization. We're very comfortable with that. We think it's entirely appropriate. It's consistent with what we've seen in the Phase III study. It serves the patients' best interest, which is our North Star. But as we progress beyond that, we think how can the market access world work to preserve its own financial margins. And one of the ways they do that is that they're going to begin to bring resistance potentially to reauthorization of medicines across the board.

Lyla Youssef

analyst
#26

That was the next question about reauthorization requirements.

William Lewis

executive
#27

So what we're going to do there is ensure that as a part of our contracting process out of the gate that there's a clear path for reauthorization. There's a clear path for initial use and the combination of those 2 things are something that [indiscernible] has paid for out of the gate to ensure that both the authorization and the reauthorization are smooth. We want as our objective, the phrase we use is a frictionless launch. So the ability to access the medicine for the appropriate patient and for that patient to continue to see benefit over time.

Lyla Youssef

analyst
#28

So what do you think the reauthorization requirements will be? Like will there be a requirement for improved something like less exacerbations or steady lung function or stability of disease or whatever, what will the reauthorization likely need to attest to?

William Lewis

executive
#29

So our experience to date is that none of that will be part of the reauthorization. The reauthorization is simply going to be a physician attesting the fact that the patient should receive -- continue to receive the medicine because in their judgment, the patient is benefiting from this.

Lyla Youssef

analyst
#30

And as you speak to payers, you're going in with that price point that you have discussed with the investment community or that's more [indiscernible]

William Lewis

executive
#31

And we've actually narrowed it down quite a bit to the point where we're able to go in and discuss the target product profile with the data from ASPEN and indicate what our intentions are. And we are, I would represent today, very confident in the outcome of that being positive. This is an exciting medicine. Even for the market access world, there is nothing approved to treat this condition, and to have a low burden medicine with a safety profile comparable to placebo means that from a market access point of view, yes, you're preserving lung function at the 25-milligram dose according to the data, but you also have a low treatment burden with a low AE profile. The worst thing that happens in the market access world is that they pay for a medicine that the patient stops using because either of an adverse event or it's too burdensome. And we know that from ARIKAYCE, right, our approved drug for the treatment of NTM is an inhaled antibiotic. It creates cough. It has -- it can cause people to lose their voice. There's real challenges there. We had to work through those, and we've done so very successfully. We're still growing at double digits after 7 years of launch. So that's something we learned. It's the same call point. This is a totally different experience. This is a once-a-day pill that they take with an adverse event profile from the data that shows it comparable to placebo. That's actually a medicine the market access world can get excited about.

Lyla Youssef

analyst
#32

So let's talk about the logistics and the launch a little bit. And you mentioned 500,000 patients that are [indiscernible] CT, but beyond that, there are patients nondiagnosed. Out of the gates, are you focusing just on that 500,000? Or will there be some incremental sales effort and marketing effort for driving increased form of diagnosis right away?

William Lewis

executive
#33

So that -- the short answer to that is yes, there's some effort in that regard at the pulmonologist, and this comes in various forms. One is what I referred to earlier, we're going to be coming up on the American Thoracic Society meeting here. For the last 2 years, we have been educating physicians and patients about the importance of identifying their exacerbations, documenting them, and that includes patients who are in the pulmonologist's office that are experiencing exacerbations but may not have a definitive diagnosis of bronchiectasis. Some insight on how many of these patients there are can be gleaned from the success of the Verona launch, where they are targeting the top 2 deciles of pulmonologists and have had a pretty successful launch because the exacerbation patients for COPD are already resident in the pulmonologist's office. That means that these patients we're describing, if we think about the funnel and step back for a minute, there are 20 million people in the U.S. that have COPD. Of that, it's estimated somewhere between 20% and 40% of those patients probably also have bronchiectasis. We know today from the roles that the -- the medical claims forms that there are 0.5 million patients definitively diagnosed with bronchiectasis. Somewhere between that 0.5 million number and that implied number of 4 million to 8 million patients is a group of patients who may be comorbid with COPD or asthma and bronchiectasis are experiencing exacerbations and with a definitive CT scan and the diagnosis by a pulmonologist, they would be on label and eligible for treatment. So we think that 500,000 number is just the beginning. We don't know how much bigger it is. But certainly, the background data here is extremely encouraging that, that number could grow.

Lyla Youssef

analyst
#34

What's your current plan for the number of the sales force that will be detailing brenso and call points upon approval like are you going to have tiering of pulmonologists? What's the variables in determining who's a priority call amongst pulmonologists? Is it going to be a center of excellence-driven initial launch? Or are you going straight to the community?

William Lewis

executive
#35

So here's what I'm going to take a moment to brag on the commercial team at Insmed. If we rewind the clock to the ARIKAYCE launch, which was for refractory MAC lung disease, the Street estimated that we were going to do $40 million to $60 million in our first year of launch. We did $130 million, and that was with a strategy that targeted both centers of excellence as well as community-level physicians. To do that in that market, we had about 70-odd therapeutic specialists. We have since added an additional 120 in the U.S. So we come close to 200 therapeutic specialists. So let me describe the caliber of these people. Many of them are the ones that participated in the launch of ARIKAYCE, and we know how successful that was. That was a top 10 rare disease and non-oncology launch in history for an inhaled antibiotic with a 30% dropout rate. These people are exceptional. The 120 we've added, we had over 7,000 resumes applying for those 120 jobs. So we have brought together what I consider to be the best in the business in targeting this launch. In parallel to this, out of recognition of the importance of this launch in this disease state, the COPD Foundation has launched a campaign to identify centers of excellence in treating NTM and bronchiectasis, and they intend to identify roughly 200 of these centers over the coming years that would be specialized in the treatment of these conditions. That is going to help bring standards of care for the best treatment for these patients as the only approved medicines for those 2 conditions, it doesn't get any better than that for us in terms of laying the groundwork. Our strategy with these roughly 200 therapeutic specialists is to be able to call on every single pulmonologist in the United States, and that is how we're going to go about this. We certainly looked here. We haven't defined what that would be in terms of who's going to get what kind of effort. But it's fair to say the centers of excellence will certainly get plenty of attention, but so too will the community-level physicians. And that, in our judgment, is the key to a sustained and successful launch.

Lyla Youssef

analyst
#36

Can you -- amongst those community pulmonologists, are there codes or proxy Rxs that you can use to identify those potential high prescribers? Like can you go after their use of COPD meds or geography going into sort of the retirement belt, et cetera. How are you thinking about ROI there?

William Lewis

executive
#37

Yes. So the first thing we have done is put our therapeutic specialists fully trained out in the field early. So they were all trained and deployed as of October 1 of last year. And what they've been doing during this time is disease state awareness about bronchiectasis, while in parallel, they're targeting the promotion of ARIKAYCE appropriately for refractory MAC lung disease patients. So we have an ongoing dialogue already about potential demand for this drug from those interactions in a highly compliant way, we are understanding what physicians see on the landscape and how this medicine in our minds may be able to be helpful to the appropriate patients. That gives us a lot of insights. In addition, there are ICD-10 codes that are already in existence for the treatment of bronchiectasis. So we can look at those data and understand where patients may be. The excitement and enthusiasm around this drug cannot be overstated at the World Bronchiectasis Meeting in Scotland last year. One of the key opinion leader physicians stood up in front of the room after our data was released and described it as our vertex moment. This is the arrival of the first and impactful medicine for the treatment of the disease that has heretofore been untouched. So the -- we know we've talked to 90% of physicians, and those physicians have told us -- or pardon me, let me be very clear. We talked to all the physicians. 90% of them have said they intend to treat their patients who have 2 or more exacerbations. And in fact, they intend to call them into the office. We've talked to more than 90% of covered lives, and we know that the market access world is going to be supportive of this medicine. We have more than 41,000 people who have gone to our website and downloaded information brochures and registered so that the day this medicine is approved, we can send out an e-mail directing people to their physicians, 41,000 of them, to tell them to go and ask about getting the prescription. So the background for this is about as positive as it could be.

Lyla Youssef

analyst
#38

I remember that presentation by Chalmers. I think I used the word messianic in my note.

William Lewis

executive
#39

Messianic, yes.

Lyla Youssef

analyst
#40

He would have gotten a standing innovation, but we couldn't get out of the chairs.

William Lewis

executive
#41

[indiscernible]

Lyla Youssef

analyst
#42

Yes. Europe and Japan time lines for approval and commercial expansion and what you see of the -- like the attention there.

William Lewis

executive
#43

Yes. So if you look at the history of drugs and revenue generation associated with the successful treatment of appropriate patients, what you see is typically about 80% of the revenue is generated by the United States and the balance comes from outside the U.S. It varies to a degree, but our expectation is that we'll probably end up somewhere in that neighborhood with the vast majority of that coming from the U.S. Having said that, we have certainly seen significant demand in both Europe and Japan.

Lyla Youssef

analyst
#44

There a lot of smokers in there.

William Lewis

executive
#45

Yes, there are, as we all know when we visit. But -- and we've seen our own experience with ARIKAYCE, right? So we're already in dialogue with all of these physicians, talking to them and understanding what they see on the landscape in terms of bronchiectasis. I would say that we will be cautious about Europe. Europe is a region of the world that struggles more now than ever with identifying an approval and reimbursement of novel medicines. And so we want to be responsive to the patient demand, but also cautious about our deployment of capital in support of that. Japan is a different story. We see north of 20% of our revenue right now coming from Japan. And so I think we're going to learn a lot from the U.S. launch, but we're also in dialogue with the physicians in both those regions now. I remind everybody, we already have commercial infrastructure in Europe and Japan that is our own. And so it will be our expectation that we will expand to meet the demand that we perceive in those regions in a proportionate way. Those launches will come next year, that's our expectation. And the approval path for those, I think, is pretty straightforward.

Lyla Youssef

analyst
#46

All right. We're going to turn now to the upcoming TPIP data, which is going to be pretty important for the stock even at these levels, CRPH data and the PVR change. Is 25% placebo adjusted still the right number that defines success in your mind? Can you talk about bookends around it?

William Lewis

executive
#47

Yes. So to be really clear, 25% is not what I would consider success, 25% PVR reduction is what I would consider a home run. If you look at the range of PVR percent reductions in prostanoids as a class, they range from the low double digits to sort of just about 20%. So we've always said that if we could get to the high end of that or above it even, that would be a very solid win. If we could get as high as 25% in that area, that would be a home run and that would put us clearly the best-in-class prostanoid out there. To remind everybody, treprostinil palmitil inhalation powder, or TPIP, is a dry powder formulation of treprostinil with a 16 carbon chain appended to it with an ester bond. That dry powder inert form of the molecule is then breathed in and the ester bond is cleaved off by esterases in the lung. The practical result of that is you're getting a slow release of the active moiety of treprostinil directly delivered to the lung. We think that the inactive form of the drug reduces the side effects as you breathe it in. That's particularly relevant for PH-ILD, but it's also relevant for PAH. We put out a PH-ILD set last year that was very compelling. It was lost a little bit in the shadow of the brensocatib ASPEN data, which is okay. But this year, the PAH data will be at the front end of our next series of events. So TPIP will have PAH Phase II data from 100 patients randomized 2:1 treatment versus placebo. And our expectation is with the primary endpoint of pulmonary vascular resistance percent reduction, we should be best-in-class relative to every other prostanoid. That's an extremely exciting outcome if we can achieve it. Anything close to that will still be exciting because this is the only once-a-day treatment that is available that gives 24 hours of coverage. And let me just make 2 observations about the data set you're going to see. The first is that pulmonary vascular resistance percent reduction in our measurement is taken 24 hours after the drug is administered at trough compared to our competitors who measure it right after administration. That's a very significant issue because they have to repeat the administration over and over again to keep that level. And the truth is that shortly after it's administered, it drops off dramatically. We're looking at it 24 hours later. So that's an extremely high hurdle and not a perfect apples-to-apples comparison for them. It puts us in a major disadvantage. But we think we're going to do well despite that. And I think that, that's a very significant issue here with this drug. The other is keep a close eye on what the actual percent reduction is because the max tolerated dose we permit in this Phase II study is 640 micrograms. Now that's more than 60% greater than what you get with Tyvaso in any form. But what I will also tell you is that we have now, in our open-label extension study, gone to double that level, 1,280 micrograms. That is unprecedented in terms of the amount of treprostinil we're able to administer. And the suggestion for that did not come from the company. It came from the Scientific Advisory Board that was reviewing the data from Phase II and said, keep going. So we now have data that will come out at 640, but we know we can go in some patients as high as 1,280, and that portends even greater efficacy, we believe, than what you're going to see with these Phase II data that are coming out.

Lyla Youssef

analyst
#48

How should we be thinking about secondary endpoints? So 6-minute walk, do they need to go lockstep? Does PVR and 6-minute walk need to go lockstep, which biologically they should in order to sort of have that cohesive argument for activity? Like if you show a PVR of 20% -- or PVR reduction of 20%, what's the 6-minute walk that you want to see to be supportive?

William Lewis

executive
#49

Yes. So we -- it's an interesting question. There's a lot of variability around 6-minute walk. Everyone has seen that in these data sets across all kinds of molecules. And right now, there are roughly 12 or 14 or so approved medicines to treat PAH. That's why we think it's going to be so important for us to present as a target product profile as the only once-a-day treatment that would ensure a PVR reduction of roughly 20%. We'll see what it ends up being. We know that the approval criteria is your 6-minute walk benefit. That's what the FDA looks for because the assumption is by reducing pulmonary vascular resistance, you increase exercise tolerability as measured by 6-minute walk. So 6-minute walk is administered. It's notoriously variable. What's interesting is that in our PH-ILD study last year, we saw a 30-meter improvement, which is quite dramatic compared to drugs that have been approved with as low as 15% -- 15-meter improvement. So what do we need to see? That would -- precedent would suggest 15. I'll be disappointed if it's not 20, but it's highly variable. So it's something you have to be cautious about. If we see a pulmonary vascular resistance reduction of, let's just say, 20% and a 20-meter improvement in 6-minute walk, and we have the only once-a-day approved therapy that covers patients overnight, and we know we can go up 100% more in amount from there, that is a very winning product profile.

Lyla Youssef

analyst
#50

So if you have a 6-minute walk that suggests 20-ish or so and at correspondingly higher PVR. Let's fast forward Phase III data based on 6-minute walk, you show something like a 25-meter difference, which I think we discussed last night of sort of the approvals for a hospital. So you have this superior PVR data, but like equivalent 6-minute walk. In pulmonologists' minds, is that a superior drug?

William Lewis

executive
#51

Yes. Hands down. And it's not just because of that...

Lyla Youssef

analyst
#52

So PVR is more meaningful for pulmonologists than it necessarily is to...

William Lewis

executive
#53

And 6-minute walk is just notoriously variable, right? People have a hard time interpreting what that really means to have a 6-minute walk benefit. I'll give you a great example. We've seen in our study in blended blinded data, PVR reductions in excess of 60%, 65%, right, dramatic reductions. We've seen correlations between that and 6-minute walk improvement. I saw a piece of data the other day that showed me a dramatic PVR reduction, but 6-minute walk went completely the wrong way. And I want to leave it to the inquiry to find out what was going on. It turns out the patient had a hip replacement. So this is the kind of thing that can happen in clinical study. You have to dig into the data to know what's really going on. And so I would just say 6-minute walk is a necessary measure to hit. We'll power the study appropriately to ensure that we do. But I also believe that the PVR measurements we're taking, which are 24 hours after administration, if they get into the 20s, that is a clear win. And we'd be very excited to be able to show that because it would be entirely consistent with what we showed last year in the PH-ILD data. 39 patients randomized 3:1. We saw a 30-meter improvement in 6-minute walk, and we saw an improvement in time to clinical worsening, which is about as good a measure as you could hope for with a benign safety profile, particularly important for PH-ILD patients. One of the things these patients do is they struggle if they have cough. And if you're bringing in the active moiety, that induces cough. So we'll see what the data show. I would say we're excited about the possibility that we could have a clear best-in-class profile with this compound. And I think right now, it's fair to say that most of the Street does not give us much, if any, credit for this program. If the data come out as we suggest, I think it's going to be [indiscernible] We're getting a little more attention now than I think people are anticipating the readout. Let's remember that sotatercept which showed a 33.9% PVR reduction after Phase II, which is the best data they had, right after that Phase II data was produced, they were purchased for $11.5 billion by Merck. So the value creation surrounding these Phase II data can't be overstated.

Lyla Youssef

analyst
#54

So how should we -- assuming Phase II success, right, with the 6-minute, how should we be thinking about what you want to take into Phase III, balancing strength and tolerability? Where is the unmet need on that sort of -- on that sensitivity analysis based on what patients want, what doctors want, how do you see it?

William Lewis

executive
#55

Yes, it's interesting. We went to the physicians when we were designing this molecule and said, would you prefer to have a molecule that is more -- is tolerated better or a molecule that pushes the dose despite adverse events? And they universally said the latter. They want to push the dose because this is a fatal disease and their experience teaches them that the more treprostinil you can deliver, the more effective it is for patients, the more benefit they'll receive, particularly when it's administered by inhalation. So that sets us up to want to push the dose as high as possible. We're going to be setting the max tolerated dose of Phase III at the 1,280-microgram level because we've seen in our last blended blinded data report, north of -- well, just about 80% of patients got to the max tolerated dose of 640 micrograms in just 5 weeks. In the real world, they'd have much longer to titrate up because of the study and the artificial need to show impact over a sustained period of time. We had to cut off titration after 5 weeks. But my expectation is we'll go to 1,280. We'll give patients much longer to get there. And as a consequence, we're going to see patients going much higher than 640.

Lyla Youssef

analyst
#56

Well, we have 1 minute to talk about brenso for chronic -- for CRS. That data is coming out. Remind us about what that primary endpoint is, what the powering is and what a meaningful change in that data is.

William Lewis

executive
#57

So CRS without nasal polyps is an addressable market that is at least as big as bronchiectasis, if not bigger. We are targeting patients who are nonresponsive to steroid therapy, the only approved medicine to treat this condition and those who are undergoing or have undergone surgery. We are looking at a primary endpoint of what's called STSS or sinus total symptom score, and we are 80% powered to show a 0.97 point change on that score. Patients come in on that measurement between 0 and 9, 9 being the most severe. They have to be at least a 5 on stable background steroid therapy, which means they're highly symptomatic. If we can show that roughly 1 point improvement, that is clinically meaningful. We do know that the only other approved medicine, this inhaled steroid, showed a benefit of about 0.7 to 0.9 point improvement on the STSS score. We are 90% powered to show about a 1.14 point change. So the variability around that point measurement is not that great. We should be able to demonstrate a clear benefit. I'm super excited about what that represents. It is a gigantic market with nothing novel that has been approved to treat that really ever. And just to put it in perspective, we think there are several hundred thousand patients incidents every year coming in that need surgery or have had surgery, and this is a medicine that would be able to have an impact there. If it proves out, let's just think about what this means. We have the TPIP PAH Phase II data the middle of this year. We have the launch in bronchiectasis in the U.S. in roughly the third quarter of this year. We have the CRS without nasal polyps readout, which is going to validate a market as big as bronchiectasis at the end of this year. At the beginning of next year, we have the ENCORE data, which is the expansion by about fivefold of the ARIKAYCE market. We have, after that, the readout in hidradenitis suppurativa, first 100 patients looking at that indication. And we have 3 different gene therapies targeting DMD, ALS and Stargardt disease, all of which will be in the clinic and showing patient data within the next 12 to 18 months. This is an unbelievably busy time for Insmed. And as dramatic as last year's impact was, we think the next 12 to 18 months are going to take us to a whole new level.

Lyla Youssef

analyst
#58

Great. With that, we are out of time. Will, thank you so much. Thanks, everybody, for joining us.

William Lewis

executive
#59

Thanks, everyone.

For developers and AI pipelines

Programmatic access to Insmed Incorporated earnings transcripts and 32,000+ others is available through the EarningsCalls.dev REST API. Plans from $24.99/month — full transcripts, speaker segments, full-text search, and the recently-added /api/v1/transcripts/recent polling endpoint for ETL pipelines.