Insmed at Goldman Sachs: Strategic Milestones and Financial Strength

On Wednesday, 11 June 2025, Insmed Inc. (NASDAQ:INSM) presented at the Goldman Sachs 46th Annual Global Healthcare Conference, outlining its strategic direction amid recent data releases and future milestones. The discussion, led by CEO Karen and Will from Insmed, showcased the company’s robust financial health and positive results from late-stage programs. While the overall tone was optimistic, challenges in launching new therapies were also acknowledged.

Key Takeaways

• Insmed has a strong cash position of $1.2 billion, supporting its clinical programs.
• Positive Phase II data for TPIP in pulmonary arterial hypertension (PAH) positions it as a potential cornerstone therapy.
• Brensocatib’s PDUFA date is set for August 12, with launch preparations in full swing.
• The company is targeting over $400 million in revenue from ARIKAYCE this year.
• Future milestones include regulatory approvals and expanded market opportunities for key therapies.

Financial Results

• Insmed holds approximately $1.2 billion in cash, providing a solid foundation for its operations and clinical trials.
• ARIKAYCE is projected to generate between $405 million and $425 million in revenue this year.

Operational Updates

Brensocatib:

• PDUFA date on August 12, with launch preparations underway.
• 53,000 patients have expressed interest in brensocatib.
• Market access will focus on patients with two or more exacerbations in the past year.

TPIP:

• Phase II data showed a 35% reduction in pulmonary vascular resistance, surpassing expectations.
• NT proBNP levels dropped by 60% in the TPIP arm, indicating strong efficacy.
• The Phase III design includes a higher dosage based on physician recommendations.

ARIKAYCE:

• The ENCORE study readout is expected early next year, potentially expanding its label to all MAC NTM patients.

Future Outlook

• Brensocatib’s launch will focus initially on bronchiectasis, with potential expansion into CRS without nasal polyps and hidradenitis suppurativa.
• Positive data in CRS could present a larger opportunity than bronchiectasis.
• Insmed anticipates significant regulatory, clinical, and revenue milestones in the next 12 to 18 months.
• The company continues to explore additional molecules for asymmetric returns.

Q&A Highlights

• The launch of brensocatib may face challenges in the back-office fulfillment process.
• A one-point improvement in the CRS trial would be considered a positive outcome.
• The team is prepared to address any issues that arise during the launch.

For a detailed understanding of Insmed’s strategic plans and data insights, refer to the full conference call transcript.

Full transcript - Goldman Sachs 46th Annual Global Healthcare Conference:

Karen, CEO, Insmed: Karen, CEO of Insmed. Will, thanks so much for joining us. It’s been an incredible last twenty four hours, but even more broadly, the last year has seen an incredible transformation for Insmed as a story. Last night, you referenced on Fast Money, this is just the beginning. As you sit here now and you look forward, what can we expect from Insmed?

Will, Insmed: Well, of all, I appreciate the opportunity to be here. And yes, I really do believe that this is just the beginning. It’s kind of a fascinating time for Insmed because people approach and they say, Oh my goodness, your data has been good for brensocatib, good data for, TPIP. ARIKAYCE is still hitting on all cylinders. How did this come about?

And I always like to say it’s one step at a time and then all at once, thirteen years to get to this point. But I emphasize all that because this really was a deliberate construction. We wanted to get to a place where when we flipped the data card on brensocatib for bronchiectasis, we had after that a series of events that would continue almost like a flywheel to drive value as we bring drugs forward that we know are going to make a difference in the lives of patients. And so if we just take a step back and think about that, our three late stage programs have all hit. So we’re three for three, which is a very rare thing to accomplish, as everyone in this room appreciates, in biotech generally, but particularly in this kind of a market environment.

So what does that look like from here? Each one of those has the opportunity to address additional populations. In the case of ARIKAYCE, we have ENCORE running that will read out beginning of next year, and that will hopefully afford us the opportunity to go to all MAC NTM patients. In the case of brensocatib, our PDUFA date is August 12. We’re on track there.

That continues to look good, and that would be the launch in bronchiectasis, followed by data in CRS without nasal polyps. And let me just highlight for everybody, that indication is every bit in our mind as big, if not bigger than bronchiectasis. So that Phase II readout in the fourth quarter of this year is going to be a very important event for us. Beyond that, we have a readout in hidradenitis suppurativa, a Phase II trial using brensocatib to address that condition. And the 100 patients for that will read out in the first part of next year.

Beyond that, we have obviously TPIP, the excellent data that came out yesterday in PAH. We had the good data from last year in PAHLD. And so that drug now moves expeditiously into Phase III trials for both PAH and PHILD. Behind all of that, we also have other compounds that are coming forward into the clinic this year and next, including, famously, our pillar with several gene therapies, but also successor DPP1 molecules and other mechanisms of action that are novel that are related to DPP1 inhibition, which we have unlocked over the course of the last several years as we’ve done our scientific work around that mechanism of action. So collectively, we think we’re bringing forward, individual DPP1 molecules that are successors to brensocatib to address, market opportunities like rheumatoid arthritis, COPD, potentially IBD.

So some very big indications will be percolating up in the course of the next several years. So we’re super excited about all of that. And happily, thanks to the excellent work of our Chief Financial Officer, Sarah Bonstein, we are in a situation where we have roughly $1,200,000,000 as of the last reported quarter. And so we’re in a very strong position financially to pursue all of these opportunities. So it is indeed a very bright day at Insmed right now.

But as I said yesterday, and I’ll continue to say, this is just the beginning.

Karen, CEO, Insmed: Great. That’s great overview. Maybe we can dig into TPIP since most recent. Many have probably seen the data, but maybe provide some high level thoughts here on the data that you were able to see from TPIP and put into context for us how that compares to other prostanoids as well as other agents that are currently available in the field?

Will, Insmed: Sure. So the PAH market, as everyone is well aware, is a very competitive market. There are roughly a dozen medicines already approved to treat this condition. It is a fatal condition with a mortality rate of about fifty percent in five years. From that perspective, there’s a clear unmet medical need here.

What we were able to bring forward yesterday was, to the best of our knowledge, at least looking at the primary endpoint of pulmonary vascular resistance reduction, placebo controlled, the best data ever produced in a controlled clinical trial setting, addressing that particular primary endpoint for these patients. And this is, at least arguably, one of the better proxies for the pathophysiology of this disease. Reducing pulmonary vascular resistance is a direct measure of how hard the right heart has to work to make up for the constriction of the vasculature, which is the hallmark of this disease. And the challenge of that is historically being able to accomplish vasodilation in a way that is sustained. Most of the drugs in the prostanoid class that accomplish that do it in a transient way, and so they have to be dosed multiple times a day, or they’re given parenterally, which, does afford constant exposure, but then it has systemic, side effects and also challenges with regard to the amount you’re actually able to get to the patient.

So collectively, there are gaps in the treatment paradigm. And I think what we have done yesterday through our chemistry is really introduce a molecule that accomplishes the original intention behind prostanoid therapy, vasodilation that is sustained over a twenty four hour timeframe with one single dose, and accomplishing two heavily pretreated patients of different functional classes, PVR reduction at thirty five percent, which is really quite a remarkable accomplishment. And I would just add to that two other thoughts. One, the consistency of the data. In the secondary endpoint of six minute walk, 35.5 meters, p values that are comfortably very, very low.

We were highly statistically significant on the PVR reduction. We also saw NT proBNP levels drop 60%, which is just remarkable. And some of the other exploratory endpoints we had were also favorable to the TPIP arm. So that would be my point, the consistency. The is the safety.

These were no side effects that were unexpected. We saw a very clean, from our perspective, safety profile. You do see side effects with this drug. That’s expected. And indeed, the physician tends to push the patient’s dose to the highest tolerable because of the severity of the disease and the interest in seeing the benefit of this drug, pulled through.

So all that said, the data yesterday couldn’t have been better from our point of view, and it, really is an exciting day for patients with this condition.

Karen, CEO, Insmed: As you think about the profile that has now emerged on the back of this data, where do you see TPIP fitting into the treatment landscape?

Will, Insmed: Well, here we’d have to take direction from the treating community because what we found when we went out with our target product profile, which was similar to but more modest than what we saw yesterday, was their belief and expression that what we would see is, this being used as the cornerstone of therapy in the prostanoid class, to which other therapies would or could be added, therapies like sotatercept, which are very effective drugs and perhaps in as a complement to TPIP, could be very effective in getting these patients almost to a point of normalcy. There’s really an opportunity here to think about these patients receiving a clinical benefit that is heretofore unprecedented as a byproduct of the strength and safety of our data and some of the other medicines that are out there. But we absolutely have said from day one, we wanted this not just to be a convenience play, we wanted it to be a clinical efficacy play. And we were able to produce that data yesterday. And that opens the door to the use of this drug, as expressed to us by the treating community, as the new cornerstone of therapy for PAH patients.

Karen, CEO, Insmed: Maybe to underscore that point, prior to the data release, 20% placebo adjusted PVR reduction had been what physicians had mentioned to you would be clinically meaningful. That extra 15% that you were able to capture here, just speak a little bit more about how much more meaningful that can be.

Will, Insmed: I mean, think it is to use the words of some of the physicians we heard, this is this data is unheard of. It was not expected to be this efficacious. It certainly was not expected to see that coupled with, a low p value advance on the six minute walk measure. That’s notoriously difficult, to capture. I think that extra PVR benefit is is nothing short of remarkable.

And that’s what we’ve heard from the treating community. I think even from the investment community and as difficult a market as we all inhabit right now, we haven’t heard anything but compliments. So I think we’re about as excited by these data as we could be. And the most important thing to realize is that we look at some of these outlier patients, who are getting north of 50% or 60% PVR reductions within these data sets. And we think that, that possibility may be available for patients through combination therapies and that opens a brand new day for patients with this disease.

I will also add two important points. Number one, we measured at, I wouldn’t call it trough levels, but low levels twenty four hours after administration of the drug, roughly. So that is a remarkable finding because it means that all of these efficacy data we’re talking about are twenty four hours after administration. So our assertion that this is a twenty four hour coverage is now established by the clinical data. The is that we went to six forty micrograms as a max available dose to patients in this study.

However, in the open label extension, these same treating physicians told us we should consider going to twelve eighty, and indeed, we have. So now there is the possibility that as strong as these data are, there may yet be more efficacy to obtain for patients that are appropriate should physicians want to go even higher. And that is going to be a feature and a hallmark of our Phase III design. We will allow patients to go, up to a max of twelve eighty, not six forty, and that may, bring forward even greater efficacy than what we’ve already seen.

Karen, CEO, Insmed: Maybe given the breadth of the data from the Phase IIb study, the consistency of the signal seen here, is there any potential for this data set to serve as a regulatory submission package?

Will, Insmed: So people always ask, they go right to this, well, your stat sig and you hit on all the data points, and I love that idea. I think we just have to be very cautious about this and see it very objectively. On the one hand, these data are remarkably consistent. The findings are very compelling. There’s no question about that.

On the other hand, we only have about 67 patients in this study that received study drug. So from a safety point of view, that database is not as extensive as one would normally want to go to the FDA to have that kind of discussion. And at the same time, this underlying moiety is treprostinil. So there is a world where one could say, well, there’s enough understanding of the safety profile of this drug so as they might not feel uncomfortable by that more limited safety database. I think the short answer is we’re going to have the discussion with FDA, hear what their perspective is.

But for purposes of the investment community, I would just counsel you to assume that we’re going be doing a full Phase III program for both PAH and PHILD. These data are very exciting, and, I’m caught up in it as much as anybody. But, but I think we have to go and have a sober discussion with the FDA and see what their thoughts are.

Karen, CEO, Insmed: Early thoughts on what a Phase III could look like here?

Will, Insmed: So we haven’t put out a lot of detail on that. I think the basic principle would be we want to, as we always do, replicate what we saw in Phase two and Phase three. So not a lot of change, not a lot of novelty. Some people ask about different approaches to Phase three. I think what we’ve shown here is that on the approvability measure of six minute walk, we can clearly have an impact even in heavily pretreated patients.

And consequently, I think that’s the way we want to focus our trial, to be well powered to accomplish an improvement in six minute walk because we know that’s a reliable, approvable endpoint. And between the PH ILD last year, where we saw a six minute walk improvement and again this year in the PAH study, I think we’ve got enough data to arm ourselves with an estimate of what we would hope to accomplish for approval in a Phase III program.

Karen, CEO, Insmed: Great. And maybe one last question here on TPIP. Previous estimates were for $2,000,000,000 plus in 2,000,000,000 plus in peak sales for TPIP. How do yesterday’s data change that?

Will, Insmed: Well, so I think the market has to evolve along a number of different parameters before we can sort of really give a better view on that. What I would observe is that when sotatercept was acquired for $11,500,000,000 after it put out Phase II data for which I think got to a PVR reduction of about 33.9%, That was acquired at a time when estimates were about $1,500,000,000 for peak sales of that drug. Today, they sit at around $8,000,000,000 So that is a remarkable ambition for that drug and class. I’m not here to suggest that we’re going to move to that number, but I do I do hear that and I I think it speaks to the enormous unmet medical need in this space. And I think what Merck has certainly said is that this is a drug that is likely to extend the lives of patients.

So you can see both an expansion of the pie and a divvying up of it as as it were will based on the strength of the various compounds and data that they can produce. From that perspective, we think this is a very exciting market opportunity. The unmet medical need here is very severe, and so we’re anxious to be able to contribute.

Karen, CEO, Insmed: Great. Maybe switching over to Brenzakatib. Imminent maybe not imminent, but very soon. Feels like that.

Will, Insmed: August 12 is right around the corner.

Karen, CEO, Insmed: Maybe just to confirm here, how have your or maybe characterize for us the interactions you’ve had with the agency recently? Does everything appear to be on track ahead of August 12?

Will, Insmed: Yes. Bottom line, everything is on track. We’re in a good place on or ahead of schedule with pretty much everything we need to do. The dialogue with the FDA has been consistent and productive. I think, one of the things that we often get asked is what’s your experience with the agency?

Our experience is if there’s been no disruption at the agency. Things have gone very well. And we haven’t seen big turnover in the team or that sort of thing. So, from that perspective, I think we’re in a very good place, and we feel like August 12 is a pretty reliable, target deadline. We’re ready.

I think that’s the other important message, Whether it comes on August 12, or earlier or or after, whenever it comes, we’re going to be ready. We think it’s going come on August 12, and that’s what we’re prepared for. But we’ve been practicing on the margin to to move earlier should we need to. And I would just say that, from my perspective, the team has just done exceptional work getting ready. Just to remind everybody, we added all of our therapeutic specialists and had them out in the field as of October one of last year.

So we have been betting on success here. And so they’ve been out in the market promoting ARIKAYCE appropriately and also doing disease state awareness about bronchiectasis. So we know the physicians, we know where the patients are. We’re just waiting for the green light. And I’ve spent time with the leadership team of the therapeutic specialists.

I can tell you they’re ready to go. Their plans are in place. And we have absolutely a world class team. I always like to throw out this statistic. We went out to hire 120 therapeutic specialists, and we had over 7,000 resumes.

So when I say we have the best of the best when it comes to a commercial team, we really were able to pick, the the folks that we wanted for this for this opportunity.

Karen, CEO, Insmed: With your sales force, having been out in the field doing disease state awareness, as you mentioned here, many of these physicians, I’m sure, have been exposed to the ASPEN data, to brensocatib’s profile. What is the feedback that your team is hearing as to the interest and the receptivity of physicians to prescribe this?

Will, Insmed: I would say it’s universally positive. The physicians and the patients have been desperate for a therapy to treat these patients for a very long time. I’ve been brought back to the World Bronchiectasis Conference in Scotland when we put our data out just a little over a year ago. And someone stood up in the room, who was a physician, and said, this is our Vertex moment. We finally have a medicine.

And I think they were referring to the potential of this medicine to really shape and change the lives of patients with this disease. As a ever approved therapy, assuming we get that regulatory approval as we expect, that brings with it a different set of challenges. These are challenges with which we are very familiar because we had the same profile when we launched ARIKAYCE as the ever approved therapy for refractory MAC lung disease. We were conditionally approved for that, And we had a great deal of success with that launch. I’ll remind everybody that The Street thought we were going to do about 40,000,000 to $60,000,000 in our year, and we ended up doing a little over 130,000,000 So the opportunity here is significant.

I think we want to be cautious about how fast we run away with potential until we see what we encounter in the marketplace. But clearly, there is an unmet medical need. Physicians are anxious to use this drug, and I think patients are as well. We’ve got now 50,000 plus people that have signed up. I’m getting the sign.

How many is it?

Karen, CEO, Insmed: 53,000.

Will, Insmed: 53,000, just for the latest number, have signed up on our website as patients wanting information about the availability of this drug. So day one, that’s 53,000 people we’re going to be targeting to make aware that this medicine is now available. When you think about the background of the addressable market here, we’ve said it’s about roughly five hundred thousand, five hundred and fifty thousand patients today diagnosed with bronchiectasis, about half of whom have two or more exacerbations. That means that we’ve got somewhere between ten percent and twenty percent of the addressable market theoretically have already signed up on our website. That’s a very encouraging sign.

Karen, CEO, Insmed: What are you hearing in terms of the patient profile based off of your market research? The patient profile, though, would be the to come on to therapy.

Will, Insmed: Yes. I think, look, clearly, patients who are experiencing exacerbations, two or more in the last twelve months are the target patients we’re going to go after. To be specific, we don’t think the label will necessarily restrict that, but we think market access will, and we’re comfortable with that. We think that was the entry criteria for our study. It’s appropriate to limit it, from the market access point of view to where we know we have the clinical data that demonstrates safety and efficacy.

And with that in mind, we think it’s those two or more exacerbating patients. We’ve taken note as we’ve learned more about this patient community that there are a number of patients out there who are seeing their pulmonologist five, six times a year. So these are patients that are clearly symptomatic, struggling with their condition and the possibility that they’re going to be able to have access to a medicine like this, I think we’re going to see rapid uptake. And our hope is that we’ll be able to facilitate that for physicians who find appropriate patients.

Karen, CEO, Insmed: And remind us here the overlap with patients who may have COPD, asthma. What is the overlap of bronchiectasis with these other comorbidities?

Will, Insmed: It’s really the story behind the addressable market here, and that is that there are roughly twenty million people in The U. S. With COPD. The literature would suggest somewhere between four percent and sixty percent of those, it’s a wide range, have bronchiectasis as well. So that’s a massive potential number.

So somewhere in there is a group of patients who have COPD and are very likely bronchiectatic as well that may be on max dose Lava Lama’s and still experiencing exacerbations. The success of the VORONA launch speaks to those symptomatic patients needing additional support. And importantly, the success of that launch teaches us that these patients are already resident with the pulmonologist. They’re not with primary care. And so they are accessible day one.

To be diagnosed with bronchiectasis, you have to have a CT scan and a pulmonologist has to review your symptoms and and designate as such. And then we’re looking for patients who have two or more documented exacerbations in the last twelve months. So I think you’re going to see patients who are comorbid with COPD and asthma that are going to be coming into the top of the funnel here in a large way. As we think about what that looked like in our clinical trials, we had roughly fifteen percent to twenty percent of patients who were comorbid with COPD and asthma. And importantly, the clinical data showed the drug was just as effective in those folks.

So I think, there’s the identified patients day one, and then there are those who are comorbid who are also going to be, folks that we’re going to be targeting once they’re diagnosed and they have those exacerbations documented.

Karen, CEO, Insmed: Maybe speak a little bit about the work you’ve done on the payer side. You’ve talked about contracting for access. Maybe talk about the strategy behind that and why you feel that is the right approach.

Will, Insmed: Well, certainly, with the ever approved therapy, we would have the ability to say, this is it, you have to cover it and have that kind of a posture or dialogue. We think the better way to go is to contract, modest contracting discounts to ensure not just access, which we think we’re going to get pretty uniformly, but more the consistency of the prior authorization and to ensure that, that process is smooth. Also reauthorization. Those are important elements to ensure that the launch starts with momentum and continues that momentum. So that’s the strategy behind contracting for access.

We think that’s going to work very effectively. We’re having very specific conversations now, I would describe those as extremely productive. And so as a consequence, we feel like this should be what we term internally a frictionless launch so that a patient gets diagnosed, gets a prescription written by the physician, and then that access back end runs smoothly. That’s really what we’re after.

Karen, CEO, Insmed: The goal of a frictionless launch, what could kind of be a hurdle to that? Is it the diagnosis of patients? Is it the ability of patients to go see their physician to actually come on to treatment? Is it the payer access, coverage there? What would be the one component you would say is the most difficult?

Will, Insmed: It’s interesting. I just take a step back. I’ve spent the last several months calling chief commercial officers of other public companies that have had successful launches and asking them what their experience has been with their launch. And almost to a person, they said that the base case scenario they had was wildly off. It was either 3x higher or 3x lower what they thought it was going to be.

And that’s a byproduct of a number of different factors. In our case, I don’t think it’s diagnosis or access. I think the biggest, challenge will be the back office fulfillment of, the authorization process of a specialty pharmaceutical. And that is something that some offices are more comfortable with than others. But we’re certainly resourced to try to support that in a compliant and appropriate way, and that’s where we’re going to be concentrating.

I will share and or confess, depending on your point of view, that the last presentation we gave to our Board of Directors included a slide that said the three things that went wrong with the launch, and it was three blank lines. We don’t know what’s going to go wrong with the launch, but something will. It always does. And of course, the mindset we need to have is one of responsiveness and flexibility to spring into action to be ready for whatever those challenges may be that present themselves. I think this team is ready.

I think about some of the other launches that have occurred recently, and we take note that the preparation for those launches really was constrained to a six month or even a twelve month preparatory time frame. We’ve been ready for launch since ATS, which is in May of every year, two years ago. We started preparing with disease state awareness, thinking about how to make this launch successful. And through the excellent work of the commercial team and the medical team, we’re ready. And I think we have the right team.

It’s absolutely top shelf. And their preparation has been to none.

Karen, CEO, Insmed: So obviously, bronchiectasis, super exciting here, but that’s just scratching the surface in terms of other neutrophil mediated diseases you could go after. You’re currently studying them now in CRS without nasal polyps as well as HS. Speak a little bit more about the opportunity that you see across both those indications and help frame expectations ahead of the data sets that are coming.

Will, Insmed: Yes. So I want to be really clear about this. What we are trying to do strategically at the company is access novel MOAs that have the ability to create that asymmetric return. And they do that by having material impact on disease states where there’s a clear unmet medical need. In the case of DPP1, this begins to look like not just our molecule addressing a couple of different diseases, but the entire mechanism of DPP1 inhibition.

And that’s why after we had our Phase two data that looked so good in WILLOW, we kicked off a very aggressive effort to create additional molecules, additional DPP1 molecules to target other diseases. We think we’ve unlocked something that is akin to CFTR modulation in terms of its impact on the potential target populations. This could continue to yield benefit for different disease states, and that’s where we’re putting some of our energy. So yes, brensocatib and bronchiectasis had stellar data, but we’re looking at CRS without nasal polyps. Just to put that in perspective, there’s nothing approved to treat that other than a generic steroid, which we are allowed we are allowing patients to be on in our Phase II trial.

There are currently thirty two million people in The United States with CRS without nasal polyps. The incidence rate of surgery and need for treatment for the severe population is four hundred thousand patients a year right now. That’s what we estimate. So that is almost as big as the bronchiectasis market diagnosed today, hitting every single year. So if these data are good at the end of the year, and we’re hoping that they will be, we have an enormous opportunity, much bigger in my mind than what we’re facing with bronchiectasis.

So we are just beginning. Beyond that, we have hidradenitis suppurativa, which is a Phase II study we’re running. The background there, neutrophil mediated for sure, a little bit more opaque in terms of whether or not we’re going to be able to have effect, and that’s a difficult to treat disease. So we’ve taken the conservative approach to look at the 100 patients coming through that trial. We’re going to unblind that data to a panel of experts who will then give us a thumbs up or a thumbs down.

We won’t get any information from it, but we’ll learn whether or not that trial should continue or if it’s futile. And we’ll have that information in the part of next year. So those two disease indications for brensocatib will create value in just the next twelve months. In parallel, these other molecules I was describing that are coming forward to address COPD, rheumatoid arthritis, potentially irritable bowel disease. These are all, neutrophil mediated where we think the DPP1 may have an efficacy role to play.

And if that’s the case, we’re unlocking a real enormous amount of value just around DPP-one, and that has a major amount of focus at the company. In parallel, we’re also doing ARIKAYCE, we’re also doing TPIP, and we’re also doing this tremendous pipeline that we haven’t spent a lot of time talking about, but which is going to start reading out clinical data next year. So we’ve had a wonderful run here for the last twelve months, but it is truly just beginning. And when we look at something like CRS without nasal polyps, once again, my favorite phrase, blended blinded data, always be cautious. But what we’ve seen and what we’ve talked about is that there is enough of a move in the symptom score we’re looking at as a primary endpoint to give us conviction that if this drug is working as we expect, we’re going to see that very clearly in this data set.

More specifically, temporally, that is aligned with the timing of when you would expect the onset of this drug, we are seeing clear symptom relief among patients in the study. Now we don’t know whether they’re on drug or not, and that’s kind of an important point, but that’s a blinded study. And when we flip it over the card to find out, we will know. But it certainly gives us a lot of comfort to see that there is symptom improvement. We’re hopeful that it is a byproduct of the drug.

And if that’s the case, we’re unlocking an enormous opportunity in this fourth quarter of this year.

Karen, CEO, Insmed: What is a good result?

Will, Insmed: Well, so what you’ve seen historically with an inhaled generic steroid is about just under a one point difference improvement on what’s called the total symptom score, which is a score that goes from about zero to nine, and it looks at different measures of symptoms for these patients. And we’re hoping for basically a one point improvement. If we can see that, we know that’s a path to approval based on precedent, and it gives us real encouragement that what we’re seeing in the blended blinded data is actually quite a bit more substantial than that.

Karen, CEO, Insmed: That’s a one point improvement over the one point that current standard of care has? Or that’s

Will, Insmed: a Yes. It would be we’re looking for a one point improvement for patients that are heavily pretreated on stable background therapy. So these are steroid nonresponders. They’re patients who’ve had, surgery or multiple surgeries or are eligible for them. So these are the severe end of the spectrum, relatively speaking.

On the zero to nine scale, they have to come in on that stable background therapy with a score of five or higher. So these are highly symptomatic patients. And if we can show a one point improvement for that patient profile, we know we’ve got a winner.

Karen, CEO, Insmed: Great. And then just what does data look what does good data look like for HS?

Will, Insmed: So HS, I would just say that what we’re looking for there is the hurdle is a thumbs up that this trial should continue, and that will be at the beginning part of next year. Once again, we’re not going actually see the data that they’re going to look at, but we’re going to know whether or not the drug is having an effect. And if it is, then clearly a win would be them telling us to continue to run the study.

Karen, CEO, Insmed: Great. And I’d be remiss if we don’t talk about ARIKAYCE at all, your base business here. But maybe just speak a little bit about your confidence on the growth trajectory from here.

Will, Insmed: Yes. So again, we’re targeting just over $400,000,000 in revenue for this year. I can’t remember the specific range. I’m going get in trouble for it. So what is it?

$4.00 5 to $4.25, and we are we’re still good with that. So what I want to just say to people is that’s just the beginning because we expect ENCORE to work. Remember that we did the ARRISE study, that read out favorably. And we think that since ENCORE was recruited at the same time, it is in effect the exact same study, just run for a longer period of time, that it should work and that would give us access to all MAC NTM patients once the regulatory process is completed. So that means by the end of next year, presuming that the data is good in the beginning part of next year, we’re going be in a position where we are filed and waiting for the FDA’s response.

And that would allow us to have another launch right on the heels of the brensocatib launch in bronchiectasis. So you’re going to see a series of regulatory, clinical and revenue generating milestones from this company in the next twelve to eighteen months that are going to be hard to match in this ecosystem.

Karen, CEO, Insmed: And just to remind us, the relative opportunity for frontline versus refractory where you’re currently approved.

Will, Insmed: So between The U. S, Europe and Japan, it’s roughly 30,000 addressable patients that we target, with the majority of those in Japan and The U. S. If you think about all MAC NTM, it increases to several 100,000. So it is a little over 100,000 in The U.

S, a little over one hundred thousand in Japan and tens of thousands in Europe. So it’s an extraordinarily interesting opportunity. Again, nothing else approved to treat this condition. And right now, we’re currently the only medicine that is strongly recommended for use in the international guidelines for refractory MAC, and we would hope to see that pull through to all MAC NTM. So look forward to ARIKAYCE ENCORE data, HS data, CRS data, the launch of brensocatib and bronchiectasis, multiple gene therapies, successor DPP1s and another novel mechanism of action that we’re working on somewhat secretly within the company that we think is a successor to DPP1 that would have a role to play in bronchiectasis in the future.

Karen, CEO, Insmed: Great. Well, that, Will, thank you so much. Lots of things to look forward to here.

Will, Insmed: Thank you very much.

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