Insmed Incorporated (INSM) Earnings Call Transcript & Summary

Good morning everyone. Welcome to Morgan Stanley Global Healthcare Conference. I'm Jeff Hung, one of the biotech analysts. For important disclosures, please see the Morgan Stanley research disclosure website at www.morganstanley.com/researchdisclosures. If you have any questions, please reach out to your Morgan Stanley sales representative. For this session, we have Insmed with Chief Commercial Officer, Drayton Wise. Welcome.

Thank you, Jeff.

So maybe let's start for those who may not be as familiar with Insmed, can you provide a brief introduction?

Yes, I'd be happy to. And thank you for the invitation. It's an honor to be up here representing the team at Insmed. For those of you who don't know the story closely, Insmed has completely transformed over the past four months. And I think that's probably going to be the understatement of the day. We're a global biopharmaceutical company, and our focus is on serious and rare diseases where we can be first or best-in-class and to make the most meaningful impact on patients. When I joined the company over 10 years ago, we had 50 to 60 employees in the company. Today, we have north of 1,000. We're operating in the U.S., in Europe and Japan. We're doing clinical trials all over the world. And we've organized the company around four pillars. So first pillar would be ARIKAYCE, second pillar would be brensocatib, third pillar, TPIP, fourth pillar, early-stage research. And we've organized the company very deliberately to have data readouts with each of these programs over time, over a condensed period of time, nearly at the same time. In the past year, we've had data readouts from our first three pillars. We've had ARISE data in ARIKAYCE. We've had ILD data in TPIP. And of course, we've had the ASPEN data that I expect to spend some time talking about today. And then in the near future and over the course of the next year or more, we're going to be looking at data readouts additionally from CRS with HS and with PAH. So we've got a lot going on in the company. With ARIKAYCE briefly, we launched the product in 2018. That was a product in the U.S. We had to build their own infrastructure capabilities. It was our first launch, our team, and we delivered a top 10 rare disease launch in the first full year of launch. And what excites me about the brensocatib launch, which is upcoming in the middle of next year, the core team, the core architects of that team are the core architects of the brensocatib launch. And the synergies of these two products in bronchiectasis or NTM are just, it's hard to state how perfect the overlap is with the NTM and the bronchiectasis. Staying with the ARIKAYCE for a moment, we had the ARISE readout, which I think exceeded a lot of physicians' and the community's expectations at the end of last year. And it gives us a good read-through for what we can expect with ENCORE, which will be the confirmatory trial and what will be the base case for what we believe our approval will be when we said that we'll have data readout in early '26 with that trial. With the ENCORE study and with the potential expanded label, we think that that's going to increase the addressable patient population by 3x to 5x. And we believe that program will be a $1 billion program on its own and all MAC. Shifting gears to bronchiectasis and the second pillar with brensocatib. Brensocatib has the chance to be one of those really special products in the industry. We think it has the chance to be one of the best pulmonary launches, if not one of the best launches. And we've said clearly that we believe just the bronchiectasis indication alone could generate over $5 billion in U.S. sales. That's again in one indication. It doesn't even include the follow-on indications where we're currently in Phase II with CRS without nasal polyps, and we're going to be opening sites and initiating our HS trial, the dermatology trial later this year. So we believe that product really could be a pipeline and a product and have the opportunity to help a lot of patients. With TPIP, I think we've all heard Will say that, that's a sleeper inside the company. We believe that's a $2 billion opportunity. And the reason to believe that is, we believe that the prodrug, treprostinil, wherever treprostinil works, ours should work better. And we've had some encouraging data readout this year, and we're looking forward to more later in next year. And then we've got the 30 different preclinical programs in our pillar 4. So there's a lot going on in the company. It's an exciting time. I think we've been able to deliver double-digit ARIKAYCE growth year-over-year, planned for the bronchiectasis launch for a couple of reasons. Regardless of your role and responsibility at Insmed, when you come to work, you have one thing in mind, how do you make impact on a patient and our culture is something special. When you walk into the walls, when you talk with people we interact with they get it. They see that there's something different about Insmed in terms of how we operate as a team and have each other's back. And I think that's been the secret sauce to make us continue to deliver double-digit growth year after year, 6 to 7 years into launch with ARIKAYCE. And I believe that's what's going to set us up for success with brochiectasis. Probably a little bit longer of an overview than you wanted.

Yes, no problem. Look, that's great. Very helpful. So maybe let's start with ARIKAYCE, and can you just talk about the launch dynamics across the different geographies. You've touched a little bit upon that, but are there aspects that lead to particularly strong growth in the U.S. and Japan versus Europe?

Yes. I continue to be amazed with this product and the impact it's having on patients and the fact that we're in year 6, and we're continuing to grow double-digit growth in the U.S. Our growth is strong in Japan and Europe. They're a little behind us in terms of how long they've been on the market. But the U.S., the team has just been relentlessly focused on finding new patients, new appropriate patients and helping them with on therapy. Some of the tools that we've used to help identify new patients is we continue to invest in different data sources and improve our capabilities, whether it's advanced analytics. We, of course, have a Google partnership to look at artificial intelligence, AI, machine learning to see how we continue to find the right patient at the right time. And the team continues to be focused on each day going out and helping those patients. So I give the commercial team in the U.S. an awful lot of credit and just continue to be amazed that we're growing like that in the U.S. In Japan, we've had fantastic growth in Japan, starting really in about the second quarter of last year, you saw an inflection point. That coincided with a new leadership team. We brought in some restrictions being lifted from the COVID pandemic. And what we're seeing is continued growth, continued excitement. Every time we call on an account, we're having good interactions to the point where we've decided to add a handful more therapeutic specialists because we think the opportunity is much more than we're tapping into. So we're excited about what Japan has ahead of us. And then Europe continues to deliver. It's a smaller market for us. We have tremendous team there, and they continue to deliver double-digit growth year-over-year as well.

And then so you kind of teased about ARISE and ENCORE. So maybe can you just briefly touch upon the results from ARISE, and why those results help to increase your conviction on positive results for ENCORE?

Yes. So I was with some of the top experts when the ARISE -- shortly after the ARISE data came out. And to a person, they all said, how impressed they were with the data, and a couple said that exceeded their -- at a conference far exceeded their expectations. But when you think about what was the purpose for ARISE, it delivered exactly what we needed it to. It validated the patient-reported outcome, it allowed us to work with the FDA to now have a primary endpoint for ENCORE. We have a clear understanding of what we need to deliver with the primary endpoint there. And then the culture conversion rate, I mean, 80% culture conversion. When you play that forward and, let's say, ENCORE produces similar results, which we expect it to, for us to be able to go into a physician and a payer and say, why would you wait? Why would you not start this patient on ARIKAYCE on day 1 with the background therapy and give them the best chance to convert, that only converts sooner but durable culture conversion and that's what that data shows. And that's what we believe we'll be able to do with, again, the physicians, the payers and the patients is, why would you start with anything else? This is going to give you the best chance to get rid of your bug. And we do think the blinded data has suggested in ENCORE that we should. I hope and expect to see similar results to what we saw in ARISE. It gives me conviction there, just keep waiting for it.

And with potential expansion into frontline MAC, like can you just talk about how you would think about the sales growth trajectory across geographies for that versus what you've seen already? Would you expect it to be kind of a similar trend? Or are there differences that you would anticipate?

As I mentioned, we believe that with all MAC, it's a $1 billion opportunity globally. And how do you get comfortable with that? We think that the addressable market will grow by 3x to 5x. We've guided this year to $340 million to $360 million. So that it -- conservatively, you can get to $1 billion estimate there with the product. The growth, I would expect primarily to come with all MAC from the U.S. and Japan. I think the payer dynamics in Europe are going to make it a bit challenging. I don't think we'll see a huge impact in Europe over the front line, but I do think you'll see significant growth in the U.S. and Japan.

Okay. Great. Maybe shifting to brensocatib. Can you just give us a brief overview of bronchiectasis and kind of what are the current treatments? How does it affect patients' lung function?

Bronchiectasis could be a devastating disease, progressive lung function disease, where you have this dilation of the airways permanently. And that can lead to -- it can be caused by inflammation, multiple infections, and what the patient experiences from that is chronic cough, fatigue, increased mucus production. And just the simple things we take for granted in our daily lives become really tough for these patients. And it's tough for them to plan because they never know when the exacerbation is going to come. So we spend a lot of our time talking to patients and talking about the impact of their disease. We have them come to our office. And we're actually building like an emerging center so we can have -- put our commercial and medical teams through so they can understand truly what it means to be a bronchiectasis patient. But they talk about the fear of the exacerbation. So once you start having the exacerbations, as you get an exacerbation, you then become more frequently exacerbated, and then what that causes is the decline in lung function. So the goal of treatment is to reduce the exacerbations and hopefully preserve lung function. And that's exciting with what we saw out of our brensocatib out of the ASPEN data, which is encouraging.

And speaking of ASPEN data, can you just walk us through the results and how they position brenso for market uptake?

Yes. We were thrilled that both doses hit the primary endpoint of reduction of exacerbation, both did around approximately 20% and that -- again, that is the goal of treatment, is to reduce the exacerbations. Where we are also encouraged is in the 25-milligram dose, you saw a preservation of lung function. One of the top experts in the world at Dundee at the World Bronchiectasis, he called the disease modification potentially. And that's -- it's a bold claim, but when you think about being able to preserve lung function over placebo over time not only 52 weeks, but presumably later, it could be a real game-changer for patients. And that's what we're excited to continue to look through the population data as well as the quality of life because for the patient, they, of course, want to remove the fear of exacerbating, but they also want to feel better. And for the first time, you've seen a product that now in its clinical trials has shown quality of life that shifted. But to just drive point home, really clear, there are no approved therapies in bronchiectasis. The experts are treating with airway clearance techniques, they are -- when an acute exacerbation happens, they'll do antibiotics. And a lot of these exacerbations will end up in hospitalizations. But we believe, if approved, brensocatib will be the standard of care and the foundation of therapy for these patients.

And maybe if you can talk about your thoughts on how the ASPEN data sets -- how those compare to the BI Phase II AIRLEAF data that were recently released?

It's a tough question to answer because the data -- they put out very limited data. And I believe the reason they did it the way they did was because World Bronchiectasis is there, it was in Dundee, Scotland with Dr. James Chalmers. And it felt like they rushed the data. So we don't really understand what the data is, Jeff. We're going to learn more next Tuesday at the European Respiratory Society. I think they're going to release a little bit more of their data, but it's really hard to compare not knowing what their data is and you certainly can't compare a registrational Phase III trial with a Phase II trial that -- I mean our ASPEN data has 1,700 patients in it. To quote James Chalmers at World Bronchiectasis, it's an unfair question. It's comparing his quote elephants and dogs.

And so you mentioned about the two doses from Aspen the 10 and 25. How are you thinking about dosing and titration and any considerations for whether there's a preferred dose to move forward with?

Again, we were thrilled that both doses hit the primary endpoint. And I think that what we've heard from our physicians is not only as we talked to and them and engaged with them in person, whether it's at World Bronchiectasis or multiple conferences since then, whether it's in market research, what we've heard from the payers is exacerbations are the goal of treatment. You want to reduce the exacerbations. And fortunately, both doses significantly hit the primary endpoint and reduce exacerbations for around 20% with each. We're extremely encouraged as the payers, as the physicians and as the patients are on the other benefits as well. The preservation of lung function, the FVC1, the quality of life scores, the BEST scores. So while we're going to continue to analyze the subpopulation data continue to discuss this internally, we're going to put our best foot forward with whatever dose we think is the right dose. But ultimately, it's going to be an FDA's decision.

Okay. And you expect to file the NDA in the fourth quarter. What remains outstanding ahead of the filing?

As you can imagine, it's a massive data set. And the team is spending nights and weekends back in Bridgewater, continuing to work on the data, analyze the data, write the clinical study reports and just continue to clean and put our best foot forward. We're going to put the highest quality package we can together for this registration so that we can get this drug in the hands of patients who need it middle of next year. And I'd say that our time lines are on track or slightly ahead of track of where we thought they would be.

Great. You've estimated about 1 million diagnosed non-CF bronchiectasis patients at launch. But given the vicious cycle can draw in patients with comorbid COPD and asthma, how are you thinking about like potential label around the bronchiactisis diagnosis? Would it specify primary versus secondary diagnosis?

Our base case assumption at launch, especially in the U.S., will be the indication statement for patients with bronchiectasis. So it's going to be a broad label is the expectation based on our discussions. And that means as long as the patient has a diagnosis of J47 code that says they have non-CF bronchiectasis, they will be on label for the product. So that's our base case going into it. Though I certainly expect the payers to go down to the clinical trial section of the package insert and say, for patients who have two or more exacerbations, but that's part of the education campaign we're doing now with not only physicians but patients. So with the physicians, we're saying what we heard a lot from physicians is, why would I diagnose them? Why would I give them a J code if there's no approved therapy. And for the patients, why would I call my doctor if they have no tool to provide for me. So what we're trying to do is educate them on the importance of getting those things documented now so that by the time we come to launch that we'll be able to have what we expect to be a really simple prior authorization, a simple checklist that basically says, if the patient has been diagnosed with bronchiectasis through a CT scan, are they symptomatic, have they had symptoms in the last -- 2 exacerbations in the last 12 months? If so, they can get approved for the drug. So a lot of that education campaigns are going on. But to answer your question specifically, regardless of the comorbidity, whether they have COPD, asthma, PCD, alpha-1, as long as they have bronchiectasis diagnosis, they will be on label.

And you talked about the education that's ongoing, but can you just talk about the broader launch strategy and the commercial launch activities and what steps might be gated until approval?

This is my favorite question because about 4 months ago in all of the investor meetings and analyst meetings, I couldn't get anyone to ask me any questions about commercial. It's all about ASPEN. So the world sort of woke up to the commercial opportunity a few months ago. But at Insmed, we've been planning for potential success in ASPEN and a launch preparation for years. So the disease state education, as I mentioned, we started doing that formally last year at ATS, where we were engaging physicians on the importance of the role of inflammation and neutrophils and why it's critical to understand that inflammatory component. And with patients, we've been educating them on the importance of speaking up when they flare up and recognizing what a flare is. Flare is an exacerbation in patient language. And we have found and it far exceeded my expectations that this patient group is highly engaged. Like as of now, we have over 30,000 patients who've taken a high-value action, whether it's to sign up for a CRM program, opt in for more information. And we believe that number is just going to continue now based on the ASPEN data being out there. So we really are excited about the launch preparations. With payers and access, again, we expanded that team significantly. Back in the end of last year, they've been in the payer offices. We've covered over 90% of the payer lines at the moment in terms of education, the burden of the disease, the importance. And there was a lack of knowledge from the insurance companies around bronchiectasis. And they appreciated us being and educating this and putting it on their radar. And I think they've all -- we've had many of them invite us back in for clinical presentations, and particularly once we are able to do the preapproval exchange information with, the payer will be in the offices doing that, sharing our clinical data well in advance of launch. The field teams, the customer-facing teams, our medical science liaisons, we nearly doubled that team. They're trained, have been in the field. We just added 120 additional sales reps or therapeutic specialists in the U.S., taking us up to 184. And one of the things I like to say there is any dollar that we invest in bronchiectasis will help ARIKAYCE and vice versa. So you think about now, we have 184 territories out there as opposed to 64 promoting ARIKAYCE in the first position starting October 1, and then disease state awareness with bronchiectasis. So the plan is coming together really well. Our medical affairs team has been generating data. I think last I checked, we've had over 75 publications in either bronchiectasis or brensocatib. We're engaging with the professional societies. We're engaging with the patient advocacy groups appropriately. And I think we have a really tight plan for successful launch year.

Great. And then on pricing, you recently guided to a potential wholesale price between that of FASENRA and OFEV. What factors would bring you closer to either the low or the high end?

So the guidance we gave was $40,000 to $96,000, and that's our range. And I wanted to be clear that's the range. It's not the beginning of the range with $40,000. And we're going through now. We're able to meet with in market research, we're able to meet with payers and share product X, which is blinded data for brensocatib and do the payer research. And what I'm happy to say now a month or 2 removed -- or a few months removed from commercial day, having seen the clinical profile, we are square in that range. So I feel very confident in that range. And as you can imagine, we joke around the office, but Will and -- I don't know if we talk about anything else other than access and pricing because this is probably the most important strategic decision we will make. The factors that go in are value for money, what do we believe the value to the overall health care system. What do we think that the value that we need to generate for our stakeholders are, but the most important decision we will factor in, is how do we ensure patients have access on day one. That's the most important factor we can have is because we want this product in as many patient's hands as possible.

Great. Maybe moving to TPIP. Can you just remind us the goal of the program and how TPIP could be differentiated treatment for PH-ILD and PAH?

Yes. In my mind, there are ultimately three goals for this program. Number one, Tyvaso is an important product. It's impacted and helped a lot of patients. We believe because our product is a prodrug of treprostinil that we Tyvaso, anywhere it worked, we should work better. One of the limitations of Tyvaso is the 4x a day dosing, where with TPIP, we're going to be able to dose once a day. So clearly there's a convenience factor, but make no mistake, the program isn't about bringing a more convenient Tyvaso out. It's about bringing a product out that could have more impact. So the other two areas that are goals of the program is, what we've seen in our clinical data, we can get to significantly higher doses than what's currently out there even with the 4x a day dosing. We were able to get -- to add 60% more treprostinil than the 4x a day dosing. And with a safety profile, that seemed better, more tolerable. So if you add all those together, a onetime a day product where you can get to significantly higher doses with a potentially better safety profile, one would have to assume the outcomes are going to be there. And what gives us hope about that is the blended/blinded PDR data that we've put out, we showed nearly a 20% reduction in PVR blended/blinded again, so we didn't know if it was a placebo patient or an old drug patient, so we blended them. And what we were encouraged about is the blended data was as good as we've seen in any of the just drug; arm in terms of PVR reductions with Tyvaso in the past. So we think we've got a really good product here. It's a great profile, and we're looking forward to seeing the data.

And either clinically or commercially, are there any considerations that investors should take into account when thinking through PH-ILD and PAH?

Yes. I think -- so ILD is a bigger market. I think there are approximately 130,000 -- 135,000 patients out there with ILD versus around 90,000 patients with PAH. The other big thing I think about commercially is, ILD is just less competitive. There's Tyvaso. And PAH is getting to be a more competitive market. But again, with our clinical profile or at least what we anticipate our data to show based on the blended/blinded data we've had, we think this product can be the standard prostacyclin for both PAH and ILD, and the pricing is still going to be the same.

So for PH-ILD, can you just talk through the Phase II results and what gives you confidence for proceeding to Phase III? But then, are there any changes that are planned for the Phase III that could balance the disease severity?

The ILD data was encouraging for a couple of reasons. I mean the goal of the study was a safety study, so to make sure that we have a safe product here. And we saw that we did. In fact, we had a safety profile that was comparable to placebo and potentially in some areas, even maybe a little bit better. And then that's how the study was powered. A small study, right, randomized 3:1. But what we also saw, we were not powered to was some encouraging efficacy data that we need to figure out. So to answer your question around what will Phase III look like, we haven't gotten there yet. The team is working very hard to design that study now and discuss with the different regulatory authorities.

And you talked about the PAH data. At what point do you expect to have enough data to unblind? And what would you be most focused on for evaluating whether proceed to Phase III?

So what will be most focused on the PDR reductions and the safety profile, and then we'll unblind that data, I believe, in the second half of next year is what we've said.

Great. Maybe in the last couple of minutes, one question on the earlier stage programs. You're starting to trough for DMD this year. And can you just remind us how your program is differentiated from other gene therapies, particularly on the ability to package microdystrophin versus full-length dystrophin?

Yes. I'm going to remind you, Jeff, I'm the commercial guy, but it's -- my understanding, we're talking with the team and understanding the program as I do, it really comes down to delivery. It's the intrathecal delivery as opposed to the systemic delivery. And with the specific delivery, you can get higher loads into the liver, which could obviously lead to bigger problems with the current DMD therapies. And with our load, the intrathecal delivery, at least what we saw in the preclinical data from our R&D data last year is that we could get to higher doses with less impact on the liver.

Okay. In the last minute, any other questions that you get regularly on the commercial side that we didn't talk about or any other aspects that we might have missed?

With brensocatib, I think a lot of the questions I've been getting is around access and how do we ensure day 1 access. And again, that's where we spend most of our time thinking right now and planning for because what we want to ensure is that the payers are educated on the disease, on bronchiectasis, the impact of bronchiectasis on our clinical profile and why they need to support it. But we also got to make sure that we continue to build out the team to support the whole ecosystem. So one of the areas that we're also proud of is our patient support program. We ended up as it should have been, it was a long debate as we were prepared for the launch of ARIKAYCE, do we move our patient support program in-house or do we just rent services out. And we decided we wanted the people who are interacting with the patients on a day-to-day basis to be Insmed employees. And that was probably one of the best decisions we made for the ARIKAYCE launch because what we hear played back from our patients, patient adverse groups, the physicians who treat them, is our ARIKAYCE program and our patient support program is second to none, and the trust they built, the empathy they have when they speak to the patients and just the ability to help patients navigate the system when the system can be really scary for an elderly patient. So we're looking to build out that team as well. We're going to go from 10 ARIKAYCE coordinators to potentially 40 or 50. We're going to -- we call field access managers, those people who are out. Some companies call them field reimbursement managers, they're helping. The offices navigate through the system and understand what it's needed to get a product approved. So when I say we're going to build out the ecosystem to help support the entire community for day 1 access, we will, because I'm confident the physicians are going to write, what they're telling us in market research is they have 80% or higher intent to prescribe this product. They want to use this product in appropriate patients. The payers, I believe, will provide access to the product and we will make sure we have every resource there to help patients get the product. And I really am excited about the upcoming launch. I truly believe it has the chance to be one of the most special launches in anyone's career.

Great. Well, let's leave it there. Thanks so much for your time.

Awesome. Thanks.