Insmed Incorporated ($INSM)

Thanks, everyone, for being here. We're really happy to have Insmed for our next fireside chat, represented by the CFO. Sara, Thanks for being here.

Thanks so much for having us. Really appreciate it.

So I think we'll jump right into the commercial questions. I'll start maybe just high level. You guys had what I think objectively, at least in my opinion, was a pretty strong quarter. I guess coming out of that, big picture, how do you feel about the guidance you've given, the trajectory for the rest of the year and maybe the key drivers there and then we can sort of dive into each one.

Yes, sure, sure. So maybe just to start off, we had a tremendous quarter, 44% growth. If you look at really any and all of the analogs out there, we've exceeded that from Q4 to Q1. And I really kind of hang my hat on, how did we do that and it's really all around operational excellence. And that started years and years and years ago from the way we designed the trial to the execution of the trial to the interactions with the FDA, to the disease state awareness that we did compliantly with both our medical and commercial teams on educating both physicians and potentially patients on what bronchiectasis is, what to look for to speak up, be heard when you have an exacerbation that then translates to the great work from our commercial effectiveness team, on how do you forecast this product. If you talk to most sort of CEOs, CFOs and if you give them truth serum and you'd to say, how did you do with your initial forecasting. They probably will vastly off one way or the other. It's astonishing how great our team has been doing on that front. And then pricing reimbursement and access. So we all know in this industry, you can develop the most impactful products. And if you don't have a pathway to pricing reimbursement and access, patients are not going to get the products. And we are continuously hitting it out of the park on all of those dynamics, which I'm sure we'll go through. So this launch could not be going better. It is now positioned to us with the revenue guide that we've given of at least $1 billion this year to be a top 25 launch of all time in our sector. So biotech can do it. Biotech mid-cap companies can launch a very meaningful blockbuster, multi-blockbuster, mega blockbuster, whatever language you want to use products and we couldn't be more proud of the impact we're having on this patient population.

Got it. Maybe we can start with the payers. I guess, heading into the quarter, there was some concern that there could be some seasonality. I guess, ultimately, was there less of that than expected? And how much of that was due to the work you've done with the payers? And I guess, what do we expect for the year going forward? .

Yes. So we're obviously early days in launch. So we've just gone through really 2.5 quarters. We had our [ stub ] period in Q3, first full quarter in Q4 and first Q1 most recently. So still learning around seasonality, vacations, Q1 dynamics, all of those good things, but specifically on the payers. So a couple of things to kind of peel on that onion. So first, you have are patients able to get it approved, right? And what we're able to see is over 90% of payers are approving this if they're going through -- if the patients are going through specialty pharmacy. So absolutely remarkable. Then refill rates, industry average for a monthly prescription is about every 37 days would be industry average for refills. We're hitting that out of the park and exceeding that. If you look at our inLighten program, so that's our patient support program and that's an ability to provide additional sort of like [indiscernible] service to patients. Industry average is sub-50% enrollments. We're north of 80% enrollment for inLighten. So all of these are just the foundational elements on why this uptick from a revenue perspective has been able to show such tremendous growth and why we are getting such great support by the payer community.

Got it. That's helpful. You mentioned the refill rate, and I think this ties also into the compliance question that we've been hearing a lot. So maybe you can address that head on. I guess as you think about benchmarks for compliance, you've talked about statins, why is statin the right comparison? And then talked in broad strokes about doing better than that? I mean can you maybe put a finer point on what that means.

Yes. So big lesson learned. This is an area where I think we kind of stubbed our toe with education, and we own that absolutely. So when we were talking about discontinuation rates and really kind of opening up our playbook of launch excellence. We were providing all of those additional metrics and guide to say things are either at or exceeding our internal expectations and things are going really, really well. So with discontinuations, we were pointing to statins because we thought that was a really high bar. And so we were trying to give ourselves a really high bar. And we were saying we're doing even better than what we can perceived as a very, very high bar. I think the disconnect was the education around clinical trial setting versus real world setting. And I'll give you some other sort of benchmarks that we can kind of hang our hat on. If you look at things like asthma or COPD, their clinical trial discontinuation is around low -- high single to mid-teens. In real world after a year, they are about 40%, mid-40s. If you look at IPF PPF, those are fatal conditions. In the clinical trial setting, sort of mid-teens is the discontinuation in real-world setting, it's in the mid-30 percentiles. So you can see the fact that we were pointing to statins and we were doing even better was to say, we're doing the best-in-class here. There is no issue. Let me be 1,000% clear, there is absolutely no issue with discontinuation. The issue was we didn't appropriately educate ahead of time. This product is being well received by payers. This product is being well received by physicians. This product is being well received by patients. And the other thing I would just comment on specifically on this topic is as the patient had discontinued for one reason or the other, obviously, that is standard in our industry. If they then have another exacerbation and they go back to their pulmonologists, what is the pulmonologist going to say? There is one approved product for this condition that has a very favorable safety profile. You may want to try BRINSUPRI again and give it an adequate amount of time to see if you're starting to feel better. So we couldn't be -- we think that team is kicking out of the park, doing a great job and no issue at all on that topic.

I'm going to ask a few more on this topic just to get a -- but I guess as you think about -- as you talk to your physicians and you maybe talk to some patients, I guess, how do those that do stop taking the drug, is it because they feel that there's some adverse event? Is it because maybe they're not feeling the improvement? I guess what's happening on the patient side there?

Yes. Yes. So Importantly, we do survey work on both the physician and the payer side. I mean on the physician and the patient side every month. And we sort of switch up who we're getting that feedback from to make sure it's a very broad and diverse group. And universally, the feedback has been very consistent on no issue on the payer side and very positive patient experience. And you'll even see that. I know a lot of folks in this room and probably listening on the phone, read all social media posts and all that kind of stuff. So you see that very consistent theme on positive patient experience, especially, again, reading some of the social post. Those patients that are on the 25 milligram, which is very aligned with what you saw in the clinical trial on some of the sort of secondary end points on feeling better on the PRO, preservation of lung function, all those good things. I would remind you that this drug just mechanistically takes a little bit of time to reach full effect. So you have 2 to 4 weeks to reach full pharmacodynamic effects. If you look at the KM curves, it's usually -- there is that time point. So physicians where we can continue to lean in is that education around if it's a KOL physician that was either in the trial or out of the medical conferences and know this mechanism inside it out or if it's more of a trialist, more of a community level, we're obviously targeting both and more of the education on giving your patients adequate time on therapy. We are not seeing. And again, early days in all of this, but we are not seeing any sort of underlying concern or anything. We are continuing to execute really well. Patients are staying on therapy and the potential for the continued growth on both the chronic use of this therapy and the increased patient population here is going to bode us really well for -- we've said $5 billion peak in revenue. We feel really confident in that number, and there's a couple more sort of layers of that onion to continue to grow that.

Last 1 on this, I promise.

No, I know it's a hot topic, and it's important that we address it. .

You've laid out that you're clearly doing at least as well as some important benchmarks. But I guess, you never want to just do good. I guess, how do you do even better? I mean, are there things that you can do to just not just be better than the industry benchmarks, but to really knock it out of the park like you've been doing in some of the other areas?

Yes. So I will say we're also knocking out of the park in this area as well. We are best in class. We believe we are best-in-class in sort of continuation rate, and I hang my hat on why are we able to do that? It's because of the great field teams that we have to support patients. We have our inLighten program. We have our field access managers, those are folks that can provide white glove service on both the patient and the back office side. I think companies do sometimes stub their toe and invest in the more traditional sort of sales rep commercial role, but then forget about the patient experience role. And we learned specifically with ARIKAYCE, the importance of that and carried that through with BRINSUPRI and provided this inLighten program with over 80% enrolled in inLighten. That sort of patient buzz and that -- those social media discussions has continued to help with the excitement of that program. Industry average is sub-50%. So patients are seeing the value in enrolling in those inLighten programs, the field access managers, helping the back office staff on sort of the paper walk -- paperwork. We know the medical exception process. We've had ARIKAYCE in the market for 8-plus years here and had very favorable payer dynamics with ARIKAYCE and we're continuing that with BRINSUPRI. And I believe we're hitting out of the park on all of those metrics.

To touch on the new patient start dynamic as well. I guess the company presented some data on sort of organic or versus patients that had been maybe part of a bolus. I guess how do you think about how that's going to change over the course of the year? I mean you've talked about seeing patient growth. So I guess, how confident are you in sort of that breakdown that you gave between patients and any leading metrics that suggest that you actually are seeing more patients going in.

Yes. No, it's a great question. And this is a little bit of an art versus a science because how can you really discern we call them ready and waiting patients, right? But how do you know? And so we feel confident in the numbers that we've been able to put out based on our internal models based on sort of the list that physicians had created and as you just sort of look at the dynamics. And so with any new launch, especially a first in disease, first-in-class, you're going to have a condition that people were diagnosed maybe 6 months ago or maybe 6 years ago or maybe 20 years ago, and they have never been able to really have an approved product until now for this. So you're willing to have those ready and waiting patients. And it took a little bit of time to see them. You didn't see them all in the subperiod of Q3 because the large institutions needed to get all of their sort of ducks in a row. So you saw sort of that spike in Q4 and to a lesser extent in Q1. And so we tried to articulate what those numbers were. What we're now able to discern is that true organic growth in Q1 was around 6,300 patients. And if you look at really successful launches, we'll take [indiscernible] as an example, they were able to sustain steady new patient adds at this point in the launch. And that's what we're aspiring to do. So sort of the -- not giving quarterly guidance, of course, we obviously have our annual $1 billion plus for 2026. But what we're saying is having steady organic growth at that 6,300 would be what would drive us towards the $1 billion plus. We will obviously always continue to look to see how we can do better for the patients that we serve, but you should assume the 6,300 number.

Yes. Another key aspect of, I guess, the bronchiectasis market is and how many patients may be misdiagnosed or maybe not have had the full workup. So I guess, how do you think about the point at which in the launch that some of these patients with COPD or asthma might be returning, getting more proper diagnosis and anything you can do to pull that forward.

Yes, so the TAM. This is a really, really important piece as you're just thinking about sort of the overall health and opportunity for this brand. So we had the benefit of -- although there was nothing approved in this disease setting before BRINSUPRI got approval last year that there was a reimbursement code. So there were patients that were diagnosed. There was claims data, we were able to analyze. And so what that told us is 500,000 diagnosed patients with non-cystic fibrosis bronchiectasis, and about half of them had 2-or-more exacerbations, which while the label is broad, our inclusion/exclusion criteria for the trial was 2 or more. And what we anticipate would be where the payers would sit. That's 2, 3 years old, I think it's about a 3-year old data. TAMs naturally grow for a lot of reasons. They naturally grow because our population increases. And I think sort of everyone has that check the box, but there's a lot more layers to that onion. As you're thinking about a first in disease mechanism and all the disease state awareness and education that we did years in advance, that helps to continue to grow awareness and grow patients coming into the physician's office to ask for a high res scan, which is the definitive diagnosis here and/or physicians getting their patients scanned and/or when they get them scanned asking to check for bronchiectasis, additional sort of layers of this onion are while this is very impactful condition for patients from a quality of life perspective and lung function perspective, it is not a fatal condition. So you have new incidents adding on top of prevalence. So you have sort of that compounding factor, which you don't have in some other disease states. And then I think -- so that's sort of base TAM. And so a lot of ways for that 500,000 to grow, and we know we need to update you on that. But then sort of the secondary launch that we talk about are all these potentially undiagnosed patients as you look at the COPD and/or asthma market. In COPD and/or asthma, it's north of 30 million patients in the U.S. You could take a whole bunch of subsets of that and how many of those or actively exacerbating patients -- how many of those are actively within the pulmonologists community, but we believe that there could be literature is all over the place, 4% to 60%. So it's not really helpful, but we believe that there could be a cohort of additional patients that are diagnosed today with COPD and/or asthma that may be underdiagnosed and also have bronchiectasis. And you could see folks like the ATS group that we provided a sponsorship, but they are doing a retrospective real-world analysis with 7 institutions to say, let me go back and look at the scans and see are these patients actually in our sort of in our community already and are just underdiagnosed that now we have a potential ability to help these patients. Another example is the [ Tie Pennington ] campaign that we kicked off about 1.5 weeks, 2 weeks ago. His mom suffers from bronchiectasis and he's been the primary caregiver. So just that education. As you think about who this patient is, they are typically female, they are typically elderly and they're typically the ones that are used to taking care of everybody else. So the education here on bringing out those patients, why we started early and while we're continuing this, it's not going to be the light switch. It's not going to be overnight. We believe we can potentially start seeing add-ins of the top of the funnel for these underdiagnosed patients probably next year. But most importantly is they would be on label. We don't need to go run another long, expensive Phase III program. They would be on label once they have their definitive diagnosis of noncystic fibrosis bronchiectasis.

It doesn't take a lot of imagination at this point to see how you can reach your '26 guide or sort of the peak guide and you painted a positive picture as much as I'd love you guys to announce some change in guidance at the healthy conference this is not going to happen. But I guess what would you need to see or what are the key things that you're tracking just given how well everything is going that may inform any changes to that in the future..

So I'll remind everyone that we provided revenue guidance very early. So the typical sort of cadence for biotech would be they would launch in the second half of the year that whole next year, they would not provide any guidance. And then in the beginning of the following year, they would provide guidance. We had such bullishness in our stub period in our first full -- first full quarter and the confidence in our commercial effectiveness team to forecast this product to be able to provide a guide this year because it's so early in the launch, we provided a floor. We said at least $1 billion and that gets us to top 25 launch of all times. That is something we should all be very much celebrating. And I know we're celebrating that internally, the fact that we can have that much impact on patient lives this early in the launch. We are a conservative company in nature. So the fact that we have provided guidance was already sort of going out on the limb. We need more than 1 quarter of actuals before we would think about updating guidance. I'll remind you, we just finished calendar Q1 from a revenue perspective. So we'll continue to track the progress here. The launch is going very, very well. I just would like to comment just briefly on the peak sales both the $1 billion and the $5 billion, and we can comfortably get there with U.S.-only diagnosed patients today. So as you think about sort of that TAM and the additional sort of layers of the onion that we just talked through and what that could mean for '27, '28 and beyond, it could be really impactful as we think about what this product could mean from a peak sales perspective. .

Internationally on the strategy, you've been pretty clear about how you're thinking about Europe. But I wanted to touch on Japan specifically, given that there is a fair amount of patients there as well, and you've had commercial success with ARIKAYCE, I guess how are you thinking about BRINSUPRI Japan?

Yes, yes. So it's a great call out. Japan has shown tremendous contribution from ARIKAYCE, and we've been able to continue to have great impact on patient lives in Japan for ARIKAYCE. We believe we have the potential to do the same with BRINSUPRI. We're obviously going through that process now from a regulatory perspective, we'll have the appropriate pricing conversation, but we do believe we have a path to be impactful for patients in Japan. The one piece I would comment on is you should not expect any revenue contribution from Japan in 2026, that would be a 2027.

8Got it. We've got just about 3 minutes left. You've got a lot of things going on at the company.

BRINSUPRI, lot to talk about. Yes, so much. Yes.

Maybe just sticking with ARIKAYCE. I just mentioned it, recently had some Phase III trial success. How are you thinking about the ability of that trial to grow the opportunity?

Yes. So the ENCORE data, huge success we were able to put that out earlier this year. And now we have the work to do with the regulatory authorities for both here in the U.S. and in Japan for that label expansion. I'll remind you that diagnosed patients today in the U.S. about 12,000 to 17,000, full NCM is about 100, similar in Japan, a little bit bigger, 15 to 18 refractory, but 125,000 in Japan for all NTM. What [ ENCORE ] showed us is that at the 6-month mark. And the reason I'm saying -month mark, is that is we can then bring the compare that to the refractory trial that we ran. At the 6-month mark, 80% of patients culture converted. For the refractory, it was 30%. So we were able to show that treating early appears to show significant benefit for these patients to be able to clear their sputum. And the other thing we saw in ENCORE is a lower discontinuation rate. So again, just treating early lower burden of disease allow patients to be able to tolerate ARIKAYCE. So work is now underway. Obviously, with the regulatory authorities, in parallel with the payers, being included in the international treatment guidelines would obviously be something that will be important here. So we can't control that, but looking forward to that potentially being updated, we have set $1 billion-plus peak sales here. It's just we need to think through sort of the duration of use for the earlier line setting and all of those good things, but we believe we have another blockbuster on our hands.

Heard you guys that you're a conservative company, but I think the TAM is potentially expanding by at least sixfold year with the frontline label if you get it. and you guys already have a sales force in place. I guess why can't it be more? Or maybe is the shape of the ramp going to be more pulled forward? I guess how do we think about what that actually looks like when you can launch.

Yes. So more work to do now that we have the ENCORE data ENHANZE, I think understanding if we are included in the treatment guidelines, what they say on duration of use, and what sort of that initial uptake will do. But we have tremendous confidence in it just some more work. And once we've done that work, we'll come back to you all...

Got 1 minute left. So I mean, we just touch on TPIP, you guys that are potentially exploring 4 studies, unique molecule. I guess how is the operationalization of those 4 studies progressing?

TPIP probably the most underlooked program in our companies. You're thinking about 4 very meaningful indications, all either in Phase III or progressing to Phase III very soon. We believe that we have the potential to be the best-in-class prostanoid therapy available for patients if we can reproduce what we saw in Phase II. So huge opportunity there. Operationally, the team is kicking it out of the park. We have 2 Phase IIIs underway right now in PH-ILD and then PPF and IPF will initiate either latter part of this year, early next year. Other things just to kind of draw a circle around is you will get open-label extension, 1-year look on the PH program, third quarter of this year. and again, looking to show sustained best-in-class pro-BNP functional class safety of those fun things. So more to come on that program.

Fortunately, we're out of time. We certainly could have gone another 25 minutes, but thank you so much for being here.

Thank you so much.