Thanks everyone for being here. We're really happy to have Insmed for our next fireside chat, represented by their CFO, Sara. Thanks for being here.
Thanks so much for having us, really appreciate it.
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?
Yeah, sure. 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 hang my hat on how did we do that, and it was really all around operational excellence. That started 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 CEOs, CFOs, and if you give them truth serum and you say, "How did you do with your initial forecasting?" They probably were vastly off one way or the other. It's astonishing how great our team has been doing on that front. Then pricing, reimbursement, and access. We all know in this industry you can develop the most impactful product, and if you don't have a pathway to pricing, reimbursement, and access, patients are not going to get the products. We are continuously hitting it out of the park on all of those dynamics, which I'm sure we'll go through. This launch could not be going better. It has now positioned 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.
Biotech can do it, biotech mid-cap companies can launch 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 one 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? 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?
We're obviously early days in launch. We've just gone through really 2.5 quarter . We had our stub period in Q3, first full quarter in Q4, and first Q1 most recently. Still learning around seasonality, vacations, Q1 dynamics, all of those good things. Specifically on the payers. A couple of things to peel on that onion. First you have are patients able to get it approved, right? What we're able to see is over 90% of payers are approving this if the patients are going through specialty pharmacies. Absolutely remarkable. 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, that's our patient support program, that's an ability to provide additional white glove service to patients. Industry average is sub 50% enrollments. We're north of 80% enrollment for inLighten. 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 we can address that head-on. I guess as you think about benchmarks for compliance, you've talked about statins. I guess why is statin the right comparison?
Then talked in broad strokes about doing better than that. Can you maybe put a finer point on what that means?
Yeah. Big lesson learned. This is an area where I think we stubbed our toe with education, and we own that absolutely. When we were talking about discontinuation rates and really 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 well. With discontinuations, we were pointing to statins because we thought that was a really high bar, we were trying to give ourselves a really high bar, and we were saying we're doing even better than what we perceived as a 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 benchmarks that we can hang our hat on.
If you look at things like asthma or COPD, their clinical trial discontinuation is around high single to mid-teens. In real world, after a year, they are about a 40%, mid-40%. If you look at IPF, PPF, those are fatal conditions. In the clinical trial setting, mid-teens is the discontinuation. In real world setting, it's in the mid-30 percentiles. 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.
The other thing I would just comment on, specifically on this topic is, if a 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 pulmonologist, 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. 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 because.
Yeah
I get it.
Yeah.
I guess as you think about, as you talk to your physicians and you maybe talk to some patients, I guess out of 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?
Importantly, we do survey work on both the physician and the payer side, I mean, on the physician and the patient side every month. We sort of switch up who we're getting that feedback from to make sure it's a very broad and diverse group. Universally, the feedback has been very consistent on no issue on the payer side and very positive patient experience. You'll even see that, I know a lot of folks in this room and probably listening on the phone read all the social media posts and all that kind of stuff.
You see that very consistent theme on positive patient experience, especially, again, reading some of the social posts, 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 endpoints 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. You have two to four we eks to reach full pharmacodynamic effect. If you look at the Kaplan-Meier curves, there is that time point.
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 at all the medical conferences and know this mechanism inside and 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 one 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.
good. I guess, how do you do even better? Are there things that you can do to 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?
Yeah. I will say we're also knocking it 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. 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 those social media discussions has continued to help with the excitement of that program. Industry average is sub 50%. Patients are seeing the value in enrolling in those inLighten programs, the field access managers helping the back office staff on sort of the paperwork. We know the medical exception process. We've had ARIKAYCE on the market for 8+ years here and had very favorable payer dynamics with ARIKAYCE, and we're continuing that with BRINSUPRI. I believe we're hitting it out of the park on all of those metrics.
I wanted to touch on the new patient start dynamic.
as well. I guess the company presented some data on sort of organic 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. 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 coming in?
Yeah. 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? How do you know? 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. With any new launch, especially a first-in-disease, first-in-class, you're going to have a condition that people were diagnosed maybe six months ago or maybe six years ago, or maybe 20 years ago, and they have never been able to really have an approved product until now for this. You're going to have those ready and waiting patients. It took a little bit of time to see them.
You didn't see them all in the stub period of Q3 because the large institutions needed to get all of their ducks in a row. You saw that spike in Q4 and to a lesser extent in Q1. 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. If you look at really successful launches, we'll take REVERE 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. Sort of, not giving quarterly guidance, of course. We obviously have our annual of $1+ billion for 2026, but what we're saying is it's having steady organic growth at that 6,300 would be what would drive us towards the $1+ billion .
We will obviously always continue to look to see how we can do better for patients that we serve. You should assume the 6,300 number.
Yeah. Another key aspect of, I guess, the bronchiectasis market is how many patients may be misdiagnosed or maybe not have had the full workup. 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 diagnoses, and anything you can do to pull that forward?
Yeah. The TAM, this is a really, really important piece as you're just thinking about the overall health and opportunity for this brand. 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. There were patients that were diagnosed. There was claims data we were able to analyze. What that told us is 500,000 diagnosed patients with non-cystic fibrosis bronchiectasis, and about half of them had two or more exacerbations, which while the label is broad, our inclusion/exclusion criteria for the trial was two or more and that's what we anticipate would be where the payers would sit. That's two, three years old. I think it's about three-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-resolution CT 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 layers of this onion are, while this is a very impactful condition for patients from a quality of life perspective and lung function perspective, it is not a fatal condition. You have new incidence adding on top of prevalence. You have that compounding factor which you don't have in some other disease states.
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. 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, I don't know, 30 million patients in the U.S. You could take a whole bunch of subsets of that and how many of those are actively exacerbating patients, how many of those are actively within the pulmonologist community, but we believe that there could be Literature's all over the place, 4%-60%, so it's not really helpful. 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.
You could see folks like the ATS group that we provided a sponsorship, but they are doing a retrospective real-world analysis with seven institutions to say, "Let me go back and look at the scans and see are these patients actually in our community already and are just underdiagnosed, and now we have a potential ability to help these patients." Another example is the Ty Pennington campaign that we kicked off about 1.5 week , two weeks ago. His mom suffers from bronchiectasis, and he's been the primary caregiver. Just that education. As you think about who this patient is, they're typically female, they're typically elderly, and they're typically the ones that are used to taking care of everybody else.
The education here, bringing out those patients, why we started early and why we're continuing this, it's not going to be a 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. 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 non-cystic fibrosis bronchiectasis.
It doesn't take a lot of imagination at this point to see how you can reach your 2026 guide or sort of the peak guide, and you've painted a pretty positive picture. As much as I'd love you guys to announce a change in guidance at the healthcare conference, it's not going to happen, I guess, what would you need to see? What are the key things that you're tracking, just given how well everything's going that may inform any changes to that in the future?
Yeah. I'll remind everyone that we provided revenue guidance very early. The typical sort of cadence for a biotech would be they would launch in the second half of a 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 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. That gets us to top 25 launch of all times.
That is something we should all be very much celebrating. 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, the fact that we even provided guidance was already sort of going out on a limb. We need more than one quarter of actuals before we would think about updating guidance. I'll remind you, we just finished calendar Q1 from a revenue perspective. 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. We can comfortably get there with U.S.-only diagnosed patients today.
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 2027, 2028 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's a fair amount of patients there as well, and you've had commercial success with ARIKAYCE. I guess, how are you thinking about BRINSUPRI in Japan?
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 conversations, 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.
Got it. We've got just about three minutes left. You've got a lot of other things going on at the company.
Yeah. Passionate about BRINSUPRI, a lot to talk about. Yeah.
So am I.
Yeah.
Maybe just sticking with ARIKAYCE as 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?
Yeah. The ENCORE data, a 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 to 17,000. Full NTM 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 six-month mark, and the reason I'm saying six-month mark is that is we can then compare that to the refractory trial that we ran. At the six-month mark, 80% of patients culture converted. For the refractory, it was 30%.
We were able to show that treating early appears to show significant benefit for these patients to be able to clear their sputum. The other thing we saw in ENCORE is a lower discontinuation rate. Again, just treating early, lower burden of disease allow patients to be able to tolerate ARIKAYCE. 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. We can't control that, but looking forward to that potentially being updated. We've said $1+ billion 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.
I heard you guys that you're a conservative company, but I think the TAM is potentially expanding by at least sixfold here with the frontline label if you get it. 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?
Yeah. More work to do now that we have the ENCORE data in hand. 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. We have tremendous confidence in it, just some more work, and once we've done that work, we'll come back to you all and educate you.
Got 1 minute left. Maybe we just touch on TPIP.
Yeah.
You guys are potentially exploring four studies, unique molecule. I guess, how's the operationalization of those four studies progressing?
Yeah. TPIP, probably the most underlooked program in our company, as you're thinking about four 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. Huge opportunity there. Operationally, the team is kicking it out of the park. We have two phase IIIs underway right now in PH-ILD and PAH, PPF and IPF will initiate either latter part of this year or early next year. Other thing just to kind of draw a circle around is you will get a open-label extension, one-year look on the PAH program, third quarter of this year. Again, looking to show sustained best-in-class pro BNP, functional class, safety, all those fun things.
More to come on that program.
Unfortunately, we're out of time. We certainly could have gone another 25 minutes.
Yeah.
Thank you so much for being here.
Thank you so much. Thank you so much.