BridgeBio Pharma, Inc. (BBIO) Earnings Call Transcript & Summary

June 9, 2025

NASDAQ US Health Care Biotechnology conference_presentation 35 min

Earnings Call Speaker Segments

Kyuwon Choi

analyst
#1

Good morning, everyone. I'm Paul Choi, and I cover the biotechnology sector here at the firm. It's our pleasure to have BridgeBio here for our next panel and Neil, CEO, join us for Q&A. What we'll do is I'll let Neil kick it off with a little bit of overview and just kind of what's topical with BridgeBio, and then we'll dive into questions. If you have questions along the way, please raise your hand, and we'll try and get a mic to you if possible. So with that, Neil, I'll turn it over to you.

Neil Kumar

executive
#2

Yes. Thanks, Paul, for having me, and thanks to Sonia, Ben, Lyla and others for having us back. I noticed you sat us between our 2 competitors in ATTR cardiomyopathy. Next year, maybe BioMarin and Ascendis, I guess the good news is we're the smallest in any of those groups, so lots of room for growth. Yes, 2025 is obviously an extraordinarily important year for BridgeBio. Really 4 things that we're focused on. First and foremost is the launch of Attruby, which we'll have an opportunity to talk about into the ATTR cardiomyopathy market. That's really an existential thing for us because we need to prove that we can be the best owner of a commercial asset in the context of a competitive space with great scientific and medical data. If we can do that, the second priority is really to continue to build on that growth aspect with our 3 Phase IIIs and $1 billion-plus marketplaces reading out late this year or early next year. It's ADH1, LGMD2I and achondroplasia. So hopefully, we can talk about those as well. The third is probably an underestimated piece but has been an area of focus for us, which is the continued prosecution of expansion indications around our late-stage medicines. We've talked about ADH1 as it sort of extends into hypoparathyroidism. I think an obvious one is hypochon for achondroplastic franchise, and a smaller one is Fukuyama syndrome for LGMD2I franchise. But those are amongst the highest ROI opportunities we see in the present portfolio. And then the fourth is continued management of our burn profile. We obviously deconglomerated last year, spinning out our oncology franchise and spinning out our early-stage Gondola franchise, 2 exciting readouts there later this year, almost 16 assets in the pipeline. But for Bridge, we'd like to keep that OpEx minus stock-based comp at around $200 million per quarter. And we view ourselves as a very lean operation, less than $20 million to IND, $50 million a year, usually on Phase IIIs or so. But obviously, the breadth of the work that we do leads to reasonable amounts of spend. And so we really want to manage that carefully. The vast majority of our spend right now is on commercial. We think that's appropriate. So those are kind of the 4 categories we're focused on. Happy to take it from there in any way.

Kyuwon Choi

analyst
#3

Okay. Great. Maybe we'll start with the Attruby launch since it's probably top of mind with investors. Your early metrics look really strong. It's going pretty well. Maybe you can tell for us what's resonated, I guess, in the marketplace so far. And with the competitive space continuing to evolve quite rapidly as recently as this past quarter here, just how you're thinking about the launch trajectory perhaps and the cadence over the remainder of 2025.

Neil Kumar

executive
#4

Yes, great question. I think it really starts with the science and the medicine for us, and maybe I'll spend a second on what's been happening because there's so much data in the space. But we can't really play games as a small sponsor around contracting or American taxpayers footing the bill for a buy-and-bill or something like that. We have to stay laser-focused on what's best for patients at the lowest cost, which our drug is obviously in this space. And from the scientific and clinical perspective, it really starts with our 3-42-50 messaging. So as early as 3 months, you see effect of this product on the salient endpoint, which is cardiovascular hospitalization and all-cause mortality. That's the earliest time point that we've seen in this space as a point estimate. The 42% relative risk reduction against that same endpoint is, again, the highest in terms of relative risk reduction that we see at 30 months in this space. And then the 50% reduction in hospitalization is quite important, suggesting that people can live longer and live better lives. And all of that, we believe, is underpinned by the fact that we have the most potent small molecule stabilizer in this space. We believe small molecule stabilizers will be frontline, and we continue to see that in the marketplace as we can talk about. We've shown in 404 preclinical assays that we are the most potent stabilizer. We sort of cheekily put up an e-mail from Jeff Kelly, who invented tafamidis, to one of our founders, suggesting that he also believes that acoramidis is the most potent stabilizer. But I think most importantly, we see it in the serum TTR data, which is clinically relevant to a lot of folks looking at patients with ATTR cardiomyopathy. So what's exciting really recently is 2 categories of data. The first is allowing us to connect the dots between ever-better stabilization and outcomes. I can remember talking to you even years ago when we were Eidos about, well, how much more risk reduction does an additional 5% stabilization really buy you? And we just released a seminal paper in JACC. It's starting to get quite a bit of excitement. Mat Maurer was the lead author on it, tying serum TTR levels and elevation in serum TTR to decreasing probability of mortality. And what's really profound are really 2 things in that. One is when we look at our database, there is no better predictor of downstream mortality other than eGFR characterization at baseline than change in serum TTR levels at day 28. That's remarkable. It's not NT-pro. It's not some quality-of-life thing. It's not even hospitalization. It is change in the day 28 serum TTR level. So that's one. And second, what is remarkable about our finding is that every mg per deciliter increase in serum TTR leads to approximately a 5% decrease in probability of mortality. So if you think about it, the average wild-type patient in our trial might have 23, 24 mg per deciliter at baseline, and they're going up to 33, 34, 9 to 10 or so change. So really profound decreases in probability of mortality associated with Attruby. And I'd say the second important point there is, recall, when folks were dropped in on tafamidis, but then moved on to our open-label extension, we were able to see what kind of elevation they would get in serum TTR levels, and it was a little over 3, so a little bit over a 15% reduction in probability associated with mortality at a p-value of less than 0.002. So really remarkable outcomes. And I think as people learn more about that, they're going to get more and more excited about the benefits of an ever-more potent stabilizer. One last thing is as in any commercial setting, it's important to start thinking about patients not as some monoculture but rather different pockets of patients and where can Attruby be the best drug because it's not going to be the best drug for all patients. So the first thing we started looking at was Afib or the cardiac arrhythmic population. It's almost half of the population with ATTR cardiomyopathy. And here, what you can see from the clinical data is that small molecule stabilizers, but not knockdowns for some strange reason, at least as far as we can see from the AE tables, have an impact. And what we were able to publish just recently is a 43% reduction in cardiovascular hospitalization in that Afib population associated with treatment with Attruby. So that's important. That's one subpopulation. And finally, another subpopulation is the sickest of patients, those variant patients. And what we've shown is a 59% risk reduction in ACM and CVH, which I think is the most profound relative risk reduction in the space, again, and the only statistically significant relative risk reduction. So we're going to use that data to go out to the clinical community and educate them on, hey, do you have a patient with Afib, the V122I patient population, which has been dramatically underserved, how do we continue to push there and those types of things. So that's really the science of medicine. That is what underpins our launch.

Kyuwon Choi

analyst
#5

Maybe just as a quick follow-up is, are those subpopulations you can eventually pursue on label, whether it's Afib or the variant or any plans to do that?

Neil Kumar

executive
#6

No, plans right now to expand the label.

Kyuwon Choi

analyst
#7

Medical education? Yes.

Neil Kumar

executive
#8

Yes, just mostly by medical education and publishing, yes.

Kyuwon Choi

analyst
#9

Okay. Great. You talked a little bit about different patient populations, but I was just curious, as you think about how the marketplace is evolving here and just what is easiest for a community physician versus an academic or a heart failure center specialist physician to sort of appreciate? And how is that mix of prescribers perhaps changed over time? Do you see the market eventually getting to a general cardiologist community? Or just maybe just how you think about the strength and weaknesses of your product and your franchise in each of those settings?

Neil Kumar

executive
#10

Yes. Great question. I'd say at the broadest level, the market continues to go across all of those segments. I mean I'll give you some example. You heard Albert up here talking about his franchise, but we've seen incredible growth in our franchise continue even with new market entrants. So the growth in May was similar to the growth that we had in April, similar to the growth that we had in March. And that, to me, suggests an ever-growing community of cardiologists that are prescribing. Certainly, we see that in our data. It also suggests, obviously, an ever broadening awareness and so that we're finding patients. Look, we haven't seen physicians that only use one product or another by and large. I mean there's a few outliers. And I think that the call point has been growing outside of the COEs, but within COEs, certainly, we have a lot of Attruby scripts within the "community" or high-volume heart failure practices. There's quite a bit of prescribing. It hasn't gotten to what you might call the general cardiologist as much yet. But just as a reminder, there's 10,000 of these high-volume heart failure practices out there. And so I think the opportunity is ripe in all of those settings for Attruby. We haven't seen one where we're not meaningfully selling, especially in that front line. So obviously, with the entrance of knockdowns, we're going to lose some switch patients. So how has the brand continued to grow so aggressively? I think a large part is that we're taking increasing share in the front line.

Kyuwon Choi

analyst
#11

Okay. Great. You launched with a very unique patient access program, including patients who are in your clinical trial getting free drug for a lifetime as well there are other different patient access schemes and so. But you've also said on your first commercial stage earnings call that utilization of these programs was somewhat lower than expected. So has this held true in this quarter? And just maybe how do you think about use of patient access programs on the forward here?

Neil Kumar

executive
#12

Yes. Thus far, it's been lower than we expected from the get-go, I think. We do have a lot of new-to-market edits. I mean from an access perspective, a new drug is always going to be the most disadvantaged. And certainly, we're going to be the most disadvantaged when we started as compared to brands that have either been on in polyneuropathy or on in the marketplace for years. So I expect that, that will dramatically improve over time. And -- but I do expect some of the very generous access programs that we put in place to be used, especially as we push more and more into the variant population, which has traditionally been underserved. Again, our hope being that anyone who needs Attruby can get Attruby as expeditiously as possible.

Kyuwon Choi

analyst
#13

Okay. Great. Maybe just as a follow-up to that, you've talked about Medicare access in your last update being at parity versus tafamidis. Maybe just what other insurance coverage or commercial coverage have you -- progress have you made this quarter? And just sort of where are we in terms of marking to market in terms of insurance coverage?

Neil Kumar

executive
#14

Yes. I mean, I think the -- a lot of those numbers are driven by the fact that Pfizer's franchise was mostly all formulary as well. The new-to-market edits were obviously unique to us. And so we continue to make progress with locking in contracts with the major 5 insurers as associated with Medicare, and that's going to be a utilization game over time. I think it's important for us to continue to communicate that we do have all these scientific benefits at the lowest cost and as we communicate that both to folks in the U.S. and in Europe, where the launch has been extraordinarily strong, I think, in part because of that and in part because of the hospitalization reduction, that we'll continue to have a privileged access situation.

Kyuwon Choi

analyst
#15

Okay. Great. I want to ask a -- and switch to a topic maybe that probably comes up with you a lot as well and which is -- comes up a lot in our conversations, which is just sort of the intellectual property landscape with regard to stabilizer here. And given that specifically tafamidis and acoramidis have similar mechanisms of action, I guess what is your latest thought on the Pfizer IP and just sort of how you see that potentially evolving over the next few years? And then what steps are you thinking about as a management team for BridgeBio should a generic tafamidis enter either at the end of this decade or at some point in the 2030s?

Neil Kumar

executive
#16

Yes. So I won't comment on Pfizer's IP position. I think people know our position on it, but Albert is sticking to the talk track, and I think they will continue to do so, consistent with what they've done with other brands. I think the mechanism of action being similar, yes, you're right. There's small molecule stabilizer, but one is highly much more potent than the other and has a different mechanism of binding. And generally, I think its outperformance as compared to taf in terms of its potency is going to be the anchor point as to how we get around any generic sort of entry in the future. I mean, look, we got real-world evidence from Maurer and Masri suggesting discrepant survival rates over time. We're going to continue to do switching studies in and around serum TTR and some other key biomarkers. So I think as people get more and more evidence that Attruby is the right drug for some of their patients, it's not going to be as challenged by a generic entry. I'd also say this. I mean, I think we're basically generic as compared to AMVUTTRA. I mean there's a $500,000 drug in the marketplace right now, and it's being prescribed just as well to -- any generic that would come into this space would have the same patient co-pay as we do or as VYNDA would. And that's about $2,000, whereas they would lack patient access programs, they would lack obviously, the ongoing research et cetera, et cetera. So I think that the dynamic of the generic entry here is a little bit different as well. And you don't see that many physicians reaching for a VYNDA 20 right now. It is just, I think, broadly being better and better described that ever more stabilization is going to be better for patients. So it's kind of how we think about it. I don't think genericization whenever it occurs. I think it's a little later than maybe my colleagues do who were on this stage before. But whenever it occurs, I don't think it's really going to meaningfully affect Attruby if we do our work correctly.

Kyuwon Choi

analyst
#17

Okay. Great. Maybe just switching gears to the pipeline and thinking about your commercial programs as well as your clinical stage programs. Over time, I'm sure you're probably thinking about international markets as well, too. And given that some of your competitors' revenues are significantly ex U.S., for instance, in the achondroplasia space, about 3/4 of BioMarin's VOXZOGO revenues come from ex U.S. markets, how are you thinking about potential most-favored-nation exposure in terms of pricing for your partner drugs, such as BEYONTTRA ex U.S. or future commercialized drugs that are ex U.S. as well?

Neil Kumar

executive
#18

Yes. So I'd say it's too early to tell precisely what impact MFN is going to have on any sponsor, and my crystal ball is as foggy as anyone else's. We have been in touch with folks within the administration. And a lot would have to go wrong for MFN to meaningfully affect us. It'd have to go to Part D. It have to be applied to orphan products. It had to be applied to biotech firms. And just as a reminder, net pricing is not all that different for orphan products between the U.S. and EU, especially where there's no standard of care established like you do see in cardiomyopathy, achondroplasia and the like. So I think MFN for our category of drugs is going to be a little less meaningful for all of those reasons, but we'll have to wait and find out.

Kyuwon Choi

analyst
#19

Okay. Maybe now turning to the pipeline. And let's talk about the CALIBRATE study for Encaleret in ADH1. Can you maybe just give us a status on how that program is enrolling and just sort of what the latest thought on timing for top line results could be here? And then subsequent to that, how quickly would you turn around an NDA filing for that?

Neil Kumar

executive
#20

Yes. Great question. Yes, so we continue to guide to late this year for the readout of that trial. It's fully enrolled. Actually, it's already 90% of people have enrolled to the open-label extension. So we're closing in here on the final pieces of the trial. Very, very low dropout rates. So I think Encaleret is turning out to be a magical little medicine for a wide variety of reasons. Look, we're prepared to turn around the NDA. We did it pretty fast for Attruby. I think we could do it even faster for Encaleret. That's the challenge to the team. And we've been doing things like batch data collecting, all of the different site quality inspections, et cetera, et cetera, to make it even faster this time around. And I expect priority review for this drug as well. So hopefully, commercial launch not too far out with respect to ADH1.

Kyuwon Choi

analyst
#21

Okay. Great. Maybe just briefly on the ADH1 market, how is that sort of recognized and/or diagnosed in the endocrinology community? And just how familiar are physicians with it versus, let's say, HPT, hypopara? And just sort of how does it fit in the sort of population and treatment landscape currently?

Neil Kumar

executive
#22

Yes, great question. I mean, I think we've commented on the few thousand patients that are already identified with ADH1. There is an ICD-10 code that you can look at that, I think, understrips the number of patients that we see in academic medical centers. One thing that's interesting is if you look at the nonsurgical hypopara community, which it's about 20% to 25% ADH1 or otherwise patients that haven't been identified with calcium-sensing receptor, gain-of-function mutations, but we find that it's about 1/4 of the population similar to HFpEF having a certain percent that is ATTR cardiomyopathy. A vast majority of those patients are seen in the community. And we know [indiscernible] are. And so a big part of what we're doing today in terms of medical education and a big part of the commercial launch will be going out to those practices and talking to those physicians about, hey, look, if you think that ADH1 might be afoot here, there are genetic tests that you can quickly determine whether or not Encaleret will be the right drug for you. So that will be a big part of the push.

Kyuwon Choi

analyst
#23

Great. Earlier, you touched on life cycle management, and you're already obviously thinking about that for Encaleret in terms of expansion to hypothyroidism. This is a market that, I think, has captured investors' attention quite a bit, given the recent success of Ascendis. But you said there's also some high amount of unmedical need there. How can you -- how do you think about what need will be out there for you post a potential hypoparathyroidism clinical program and approval for Encaleret?

Neil Kumar

executive
#24

Yes. Well, look, I think the approved product, obviously does remarkable things for patients. It makes sense if you don't have PTH to put PTH back. But it's important to remember what PTH is doing in the normal human body, which is it has a diurnal regulation going from 10 to 60 or so over the course of the day. And what the medicine today is doing is it comes in with effectively a uniform PTH concentration. And that's why you see people step up in dose over time. And ultimately, they get to the point where about 20% of patients can't continue to dose up for bone resorption issues and others that you're well familiar with. So there's at least 20% of the community that is not well served by the current drug. And I've never seen one drug serve everyone, so that's not a knock on that drug. Second is a daily injection, and we have a daily oral -- once-daily oral. Look, we've been on this stage talking a lot about efficacy and safety, and that's what's going to drive everything. But being in the commercial marketplace, you can see little changes in -- convenience can change compliance rates and the enthusiasm for patients to be on it. And for many patients with a 30% injection site reaction level, they'd rather take a once-daily oral. And then the final thing is I always come back to efficacy. What you want to do for this condition is you want to normalize serum and urine calcium. And I'm really pleased today to say our 10th patient from our small preclinical -- clinical study in HP came in. We have an 80% responder rate on both serum and urine calcium. So that point estimate, albeit small ends, is by far and away the highest that we've seen in the space. So you get a single daily oral, incredibly safe. Yes, it's an orthogonal mechanism to PTH, but in a PTH independent way, it's able to rectify the thing that's gone wrong for these patients. And we think that will be quite attractive for many of the patients in the HP community.

Kyuwon Choi

analyst
#25

Great. Maybe just as a follow-up to that, one of the key things that patients look for in the HP community is reduction of pill burden. A fair number of them are large amounts of calcium and calcitriol and so forth and vitamin D. Just how do you think about that metric and that sort of convenience down the road being a factor in shaping preference shares in the marketplace?

Neil Kumar

executive
#26

Yes, we'll have to see how that -- the 1,25-dihydroxyvitamin D regimen, how we change that over time will be certainly something we're looking at in Phase III, not something we've measured to date. Serum calcium, obviously, would -- that's an obvious thing that we would get around and that would go down. You don't take standard of care in the context of Encaleret even in the ADH1 trial.

Kyuwon Choi

analyst
#27

Okay. Great. Maybe -- are there any other aspects of life cycle management for Encaleret that you would potentially want to call out at this point? Or is it just something you're still keeping close to the vest here?

Neil Kumar

executive
#28

No. I think those 2 are the main ones, ADH1 being the salient marketplace and NHP being the follow-on that there aren't a lot of other conditions that are associated with gain-of-function in calcium-sensing receptor.

Kyuwon Choi

analyst
#29

Okay. Great. I want to turn maybe to ribitol, which I think is a program that's maybe a little underloved among investors here. And just first, people are obviously following other dystrophies very closely like DMD in the marketplace here. But maybe just sort of what is the opportunity maybe to start here with the Limb-Girdle and just how are patients identified?

Neil Kumar

executive
#30

Yes. So this is a well-diagnosed community, some already 7,000 patients between the U.S. and EU. You can see that from how fast our clinical trial enrolled. You can see that from the wide variety of now 3 natural history studies that have been done in the space. So it's a big market, bigger than exon 51, I would say, but not as big as DMD broadly, obviously. And the opportunity there is to be first and best in class. I think we haven't seen an agent yet really move the needle for this otherwise devastating condition, and that's what we hope to do with the readout here later this year.

Kyuwon Choi

analyst
#31

In terms of the metrics in Limb-Girdle, I guess, what are the key ones that you would highlight for us? I think in the DMD space, people are familiar with dystrophin or microdystrophin production, time to walk and/or North Star assessments. Just sort of how do treaters in the Limb-Girdle's 2I space think about what they want to see in terms of patient outcomes?

Neil Kumar

executive
#32

Yes, very similar. Accepting this is a different path of mechanism, so you could substitute dystrophin expression, if you will, for glycosylation of the alpha-dystroglycan complex. And here, what we've agreed with, with the FDA and what the clinical community agrees with as well is that any rise in ADG glycosylation would be meaningful to patients. 5% to 10% on an absolute basis is able to create something remarkable for -- in the mouse model. And you can see that in the natural history as well going from homozygous to heterozygous patients. So that's really the aim, the core aim over this 12-month study. Obviously, we have a confirmatory trial that's going out to look at North Star and some of those associated endpoints. I don't imagine that those will change meaningfully in 12 months. You can see that from the natural history. But we'll be looking at everything. We'll be looking at ambulation, to your point. We'll be looking at lung function. And our hope is that we're able to -- similar to our Phase II, show some improvements that are discrepant with natural history there in addition to profound biochemical improvements both on ADG as well as CK. And hopefully, all of that lines up in the same way it did in our Phase II.

Kyuwon Choi

analyst
#33

When we're talking about Encaleret earlier in ADH1, it's clearly a biomarker-driven disease where you focus on calcium, here in terms of dystrophies, I think the agency has expressed a view that -- or at least that seems to be evolving in real time that they're amenable to surrogate endpoints like biomarkers or dystrophin and so forth, similar here for Limb-Girdle, but you also talked about functional outcomes down the road in confirmatory trial. Maybe just at a high level, Neil, as you sort of watch the agency's commentary here on surrogate endpoints as well as confirmatory trials, how does your ribitol program, I guess, line up with this potentially?

Neil Kumar

executive
#34

Yes. We've been very impressed to date with our discussions with the agency. They haven't really meaningfully changed from the last group in and around what people call surrogate endpoints. And especially when it's a causal piece of the condition, serum calcium and urine calcium, obviously being the core drivers effectively of ADH1. So that's not really an accelerated approval. That's a full approval. But in the case of LGMD2I, it's what you can measure. It's a devastating condition, and we know it to be on the causal pathway. So I think there's a lot of enthusiasm for those types of conditions. We're seeing it with Canavan disease. We're seeing it with LGMD2I. Obviously, there's no need to talk about it with our achondroplastic franchise. But yes, no, continued enthusiasm there. And my hope is that sponsors continue to do the right thing in running confirmatory trials like we are. And then it all comes together for patients in a really nice way.

Kyuwon Choi

analyst
#35

Great. And maybe just to close it out on Limb-Girdle, you've talked about the 7,000 patients that have been identified in sort of the major geographies here. As you think about it, what does that translate to in terms of a potential market opportunity?

Neil Kumar

executive
#36

Yes, we think it's well over $1 billion. Obviously, the pricing in this space, you can look at the comps, is fairly reasonable. And so like that's how we think about the primary indication. The secondary that I talked about, Fukuyama is mostly -- most of the patients are in Japan. And so we'll have to see what the price point is there and just how big that marketplace is, but it stands to reason that the molecule should work in that condition, which is an upstream mutation in the same FKRP-associated signaling pathway.

Kyuwon Choi

analyst
#37

Okay. Great. Maybe turning to achondroplasia and your infigratinib program. That's also a data set that could come potentially end of this year, maybe early next year. The marketplace continues to be very attractive given the commercial success of BioMarin's VOXZOGO. But obviously, you first have to complete your pivotal trial here. And so just maybe to start, what will you share in terms of top line metrics, either at year-end or early next year? And just maybe help us contextualize how you think about infigratinib potentially performing versus some of the commercial or clinical-stage agents out there?

Neil Kumar

executive
#38

Yes. Great question. I mean, I think we'll share everything that we've been traditionally sharing baseline characteristics; responder rate; obviously, change from baseline in AHV, which is the primary endpoint; absolute AHV proportionality. All of those things, I think you can expect us to have a fulsome data report upon top line. How does it compare to the CMPs? I've been outspoken on this. I think it's obviously a much more efficacious agent. To start targeting the well-described condition at its source, I think, has its benefits. Obviously, it did in terms of the Phase II with higher-point estimates on change from baseline, but more importantly, I think the impact on proportionality and others. And obviously, it's a once-daily oral. And again, I just -- I'm seeing the impact of convenience across the board. I think it's -- I don't think we need to even post a better point estimate in the Phase III. I think we will, by the way, but I don't think we need to, to get a majority market share here. Why? Because the totality of the evidence suggests that this is a better product, more efficacious product, a safer product and then ultimately, one that is much more convenient for the community that we're serving here.

Kyuwon Choi

analyst
#39

Do you plan to present the data at a medical meeting prior to your NDA filing? Or is that just something you'll do in parallel at some point?

Neil Kumar

executive
#40

I'd imagine that we would do that. I don't know exactly what the medical meeting time line would be. But yes, New England Journal paper and a medical meeting would be the way to go if it's successful.

Kyuwon Choi

analyst
#41

Great. Maybe just touching on something you said a minute ago, which is just oral administration in the pediatric community, pediatric patient population, I guess. Can you maybe just share with us what your survey work shows versus the injectables, whether it's a daily or a weekly here?

Neil Kumar

executive
#42

Yes. Because I mean, just going back to my prior point, too, people do understand that -- so for instance, our trial goes all the way down to 3 years of age, right? You're taking a hit on the point estimate on change from baseline in AHP the younger you go. All the rest of the trials that have been generated for approval are 5 and up. So this kind of like just like what is the point estimate, what is this, what is that, I think it's going to be less important to clinicians than the totality of the evidence and the convenience. And I think with an oral, especially in sachet form, for young patients going all the way down to 3, our survey work suggests that you take a majority share, again, even if your point estimates are roughly similar to the 1.5 placebo adjusted that you see from the other drugs. But again, I think it will be higher. And I think you will see impacts on other parts of the disease pathology that you don't see targeting downstream with the CMP.

Kyuwon Choi

analyst
#43

Okay. Great. Maybe one more on infigratinib, which is, I think, a question you probably get as well, which is just thinking about the regulatory framework for your potential filing as well as potentially completion of the BioMarin confirmatory trial and just what time lines there might look like and just sort of what your interaction on the agency has been with that regard.

Neil Kumar

executive
#44

Yes. Unchanged again. We had BTD, which obviously was I thought quite compelling because they were able to look at the totality of other evidence and suggest that this is a breakthrough. We've heard nothing from the agency that we wouldn't be able to get approval based on the trial design that we have now.

Kyuwon Choi

analyst
#45

Okay. Great. In our last few minutes, I want to talk maybe a little bit more about what happens or what BridgeBio looks like over the coming years. You have 3 Phase III programs that are going to finish this year and potentially could translate to commercial stage starting next year. And then you've talked a little bit about life cycle management for these assets as well, but -- as well as managing cash burn on the forward here. And just I guess, how much do you think about the shape of the company over the next few years looking like as you moderate your late-stage program and transition more towards -- back to your historical development program and some clinical-stage programs, just -- and the transition to profitability. Can you maybe comment a little bit on that?

Neil Kumar

executive
#46

Yes. I mean we're in serious growth mode right now. And our hope is that these Phase IIIs reading out later this year provide us with that growth engine. So we sort of couple demonstrated excellence with Attruby. Hopefully, that continues with these new growth modes. And I think that should continue to be the engine of growth for some time for BridgeBio. I don't anticipate a whole lot of early-stage research ongoing, especially with the deconglomeratization that we did last year. But we'll obviously have a lot of ideas internally should we need to re-energize that growth engine. Right now, the focus really is continued execution, continue to try to capture the value for some of these programs that we think are a bit unrepresented amongst investors. And hopefully, with strong launches against LGMD2I, ADH1 and in the achondroplastic setting, we could be off to the races here in the next couple of years.

Kyuwon Choi

analyst
#47

So if I'm hearing you correctly, obviously, potentially multiple product launches starting next year, maybe '27. And just as you think about the expansion in your sales force, when does that process sort of start? Should we think about baking that as sort of an ongoing growth investment for the next couple of years?

Neil Kumar

executive
#48

Yes, that's why I sort of referenced that $200 million per quarter. Actually, like modeling it out over the next 36 months, I think that's going to be fairly accurate with some error bar around it. Why is that? Because I think R&D expense will moderate as we get through some of these Phase IIIs and -- but that will be then paired with increased commercial expense. I should say the commercial expense associated with ATTR cardiomyopathy is vastly different than even that associated with achondroplasia. And ADH1 and LGMD2I, we're the only game in town. So that's a very, very different type of launch. You can look at -- I think we use the Ultragenyx KKC launch as a good comp, but there are others that the expense can be far lower. And we have a shared infrastructure now around market access, medical affairs, LDN, hub management, patient access. All of those things can be applied across brands. So it will just be -- what's bespoke to each opportunity will be a very small marketing team, a pretty small sales team for those remaining of the indications and the sales force, obviously.

Kyuwon Choi

analyst
#49

Great. You've obviously partnered out acoramidis with Bayer in Germany and Alexion, AstraZeneca in Japan. Are you thinking about those other 3 properties, just -- agents, excuse me, doing those by yourselves given what -- how you've characterized them both in the U.S. and abroad?

Neil Kumar

executive
#50

I think we can do them abroad. I think obviously, there's a shared -- there's some shared overlap in call point between ADH1 and achondroplasia. And I think we're building out rest of world actually for Attruby. And we've seen that maybe it's not maybe as complicated or expensive as we give it credit for. First, lots of companies have spent way too much [indiscernible]. And so we certainly weren't in the position to raise the capital to do so for the Attruby launch at relatively reasonable cost, but that might be different for ADH1 and achondroplasia. And so we'd like to maintain the opportunity to do it. If a better owner comes along at a price that's higher than NPV, we would also look at that, too.

Kyuwon Choi

analyst
#51

Okay. Great. We're out of time here. So thank you very much, Neil.

Neil Kumar

executive
#52

Thank you. Appreciate it.

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