BridgeBio Pharma, Inc. (BBIO) September 2026 Conference Transcript

Well, good afternoon, everyone. Thanks for joining us here after the one session. Get right back into the fireside discussions. So my name is Derek Archula. I'm one of the biotech analysts here at Wells Fargo. Really excited to have next here Bridge Bio from the company. We have Chinmay Shukla, Chief Business Officer. Congratulations on that, as well as Anna Wade, COO of the Neuromuscular Division. So thank you guys for joining us.

Derek, thank you for hosting us, and thanks to the investors for joining us. We're excited to speak to you today and excited for all our conversations.

Well, Chime, maybe just to kind of level set us with the business and where you guys are, we've got a pretty solid growing base business with the Truby, a couple, three launches upcoming. So maybe just give us a state of the business before we dig in.

Yeah, it's a great question. So state of the business today is if you look at our post-phase three drugs, right, that's in ADH1, limb girdle, ATTRCM, obviously, and achondroplasia. We have $8 billion of de-risk peak year sales potential. Total, if you look in the entire pipeline, we have $10 billion of risk-adjusted sales potential. So if you feel great about that, I think if you look at a Truby second quarter, global revenues were annualizing to over a billion dollars. U.S. revenues were $222 million. dollars. We've been growing very nicely at about, you know, $30 million, $25 to $30 million quarter over quarter. We expect to keep growing at that $25 to $30 million quarter over quarter cadence for a Truby. I think all the clinical updates on a Truby have been extremely positive. We can get into that more. And so that gives me a lot of confidence that in 27, as we launch these next three drugs, that's going to add a lot of growth. And then eventually, you know, Second half, 27, once these clinical messages take hold in the community, Truby is also going to start to accelerate. So we feel very good about where we are. And the last thing which I'll say, which is important in the long-term perspective of the business, is for us, as we start to have all these revenues and generate cash flow, we think about where can we invest it. And if you look at the genetic disease landscape, Innovation has never been higher. Unmet need has never been higher. And so, yes, of course, we have to grow into all of that, and we have to do it in the right way. But we feel like we have the opportunity to help a lot of patients, and that, I think, will accrue to the benefit of our shareholders.

Well, maybe let's start with Atrubi. You provided some good context there in terms of growth and things. But we just came out from ESC. We had kind of the cardio transform data and all that stuff. So I guess what's your view in terms of how the market continues to evolve and the role of stabilizers?

Yeah, so there's been a lot of clinical updates in the TTR space. And so let me sort of summarize where I think we are at right now and where we see this market growing. So first of all, stabilizers post-cardio transform, it's very clear that today as well as in the future, almost all new frontline ATTRCM patients will start on a stabilizer. Today that number is north of 80 percent. I don't think that that's going to change given what we saw in CardioTransform. I also think that combination therapy is not really effective and it's not going to be a big part of this market going forward. And so that's the second thing which we learned. The third thing which we learned is the two knockdowns are very similar to each other, and that obviously feeds into why stabilizers are frontline, but if you look at the placebo-adjusted knockdowns, they're the same. If you look at the hazard ratio on monotherapy, they're the same. And then the last thing, which I think ESC told us, which I think is a little lost sometimes from investors, is there is meaningful room to help beyond Vendamax. And so obviously today, a ton of patients, majority of frontline patients start on Vendamax, which makes sense given it's the established product. But we think a Truby as the next gen, second generation differentiated stabilizer will take more and more share and more patients will start on that drug because simply put, it's a better drug. And so I think that we feel very good about where we are, whether it's because if you look at subpopulations like AFib variant, whether you look at unique kidney protective effects of a Truby. And now what you're starting to see, which is really interesting, is real world evidence start to come to the front, right? And so you've had one publication, we've heard talks about there being more independent real world studies which are coming out. So we have to see what happens at HFSA and AHA. But I think all of that will mean that stabilizers will continue to be the frontline therapy of choice. And then over time, we do expect a Truby to keep taking more and more share and become the preferred stabilizer.

So what, you know, you talked about some of these data sets that are coming, you know, whether they're, you know, yours or, you know, externally, like, what other evidence do you want to generate, you know, between now and maybe the, the, to famous, you know, patent expiry to really drive home what you're talking about in terms of that differentiation?

Yeah, so all of our work on this front stems from what physicians tell us is most convincing to them. And what we have learned is, number one, subgroup data, right? So that's why this AFib data really drove AFib and variant subgroup data, which showed that a TRUBI confers a very large benefit amongst the AFib population and has the highest point estimate in the variants of population. Actually, it has a statistic benefit on variants, which is really remarkable if you think about it, given the low number of patients in that segment in the trials, right? I think that that is convincing. I think we've now built on that with this very unique nephroprotective effect that a TRUBI has. That is still in its early days right now. I don't think that that message has fully gone out to the community. We just published a paper on it a couple of months ago. I think it's going to take six to nine months for people to fully appreciate just how profound that renal protective effect is that a TRUBI confers. I think the third thing which we've heard from physicians is real world evidence. and so of course the most convincing real-world evidence is when it's not published by us so far there have been I think three studies to my understanding one we've been involved with the other two we haven't been involved with even in studies where we are not involved you are seeing that acramidus outperforms trafamidus in real-world settings we hear that there is going to be a big publication at HFSA on real-world evidence which I think will further move move the field in terms of understanding how to use these drugs in the real world setting. So those are the kinds of things which I think are going to be important. I think our job right now is to educate physicians on all these benefits which have already been generated. I think the other factor which we don't need to educate physicians on, but which maybe we need to do a better job educating Wall Street on, is things like channel dynamics, which mean that But even in 2031, when Windermax does eventually go genetic, because this is a channel controlled by specialty pharmacies, institutional specialty pharmacies, as opposed to payers, we do think that volume for a Truby is going to remain high. And I actually think if you look at drugs like this, and we updated our corporate decks, we have some examples there now. If you look at these kinds of situations, you always see that the second gen differentiated the product continues to grow after the first-gen product has a loss of exclusivity. And I also know that some of your own work suggests that, so I think you can also help us with that.

Well, I was going to say also, when you think about the dynamics, and some of this came out at ESC in terms of we're treating these patients earlier. And I always feel like there's this, maybe it's a wrong way to think about it, but after 2031 generic tabamis comes and then it just truly goes off a cliff. but the fact of the matter is if you're putting patients on 2029 2030 they're probably going to be on drug for four to five years and the payers are not really going to make you switch a patient that's doing well right so like you have that maybe at least giving you that kind of revenue tail and then it's up to you in terms of like again talking about those channel dynamics in terms of just even keeping the volume increasing maybe slower but you're still growing i agree with you yeah see we've done our homework so um but i guess well you've done a lot of work on this So I know you're up to date on that. So I guess when we think about TTR in general, I guess, were there any other kind of takeaways from ESC in terms of where you think the field's going just from an overall perspective in terms of the diagnosis rate and the growth because you've got a lot of tailwinds just in that sense as well.

Yeah. So that's a very good point. And actually, if I think about the two biggest tailwinds for a Truby, one of them is the fact that the frontline market continues to grow. So before we launched in 2024, we were saying that there are about 8,000 to 12,000 new patients, frontline patients who start NETTRCM therapy every year in the U.S. I think about six to nine months ago, we updated that to say that there are, call it, 12,000 to 16,000 patients. And I think now what we are seeing is that there are at least 16,000 patients. So if you see, you know, you've gone from 8K to 16K over a period of two years, and it's still growing, right? And our share in that market is also growing. So that is a big tailwind. Nothing at ESC, you know, everything at ESC reinforced that. The more awareness about ATTRCM, the more patience people find with this disease. So I actually feel every time we have these big conferences where there's a lot of focus on this disease, it helps a lot with diagnosis. It helps a lot with awareness. So that is definitely a big tailwind for us. And I actually think that that tailwind coupled with the differentiation and, you know, slow gains and share are the two reasons why, despite the fact that we have some headwinds, such as the second line pool normalizing, such as Pfizer rebating, we are able to keep growing consistently. And that is why, you know, if you saw last quarter, we had nice growth quarter over quarter. And if you look next few quarters, we expect that to keep going.

Gotcha. You know, and not to put you on the spot, but in terms of, like, you know, silencer uses in the front line, I mean, I guess, you know, El Nile will say they're growing. You guys are saying you're growing. So is Pfizer not growing? Like, or is everyone growing? Like, how are the share dynamics going? And I guess, you know, where do you guys think you'll be? Because I think one of the things that you guys had said in the past was, like, your assumption was it was going to be a four-player market and what your share was going to be. So, you know, will you update some of those estimates?

Yeah, great. I'll take that in the reverse order. Yeah, we have said before that we expect at peak at Truby to get 30% to 40% share. We're doing more market research now, and we will update it. I will say that that is at peak. I do think, as I mentioned, it's going to take a year or so for these messages to truly get understood by the community. So I do think for the next few quarters, we will keep growing by that 25 to 30 million quarter-over-quarter sales absolute number that we have said before. I do think we will update that 30 to 40% peak share number. Does it go 35 to 45, 35 to 40? You know, wait to see that. I will not create that much news right now. The second thing which I would say is, how are the frontline dynamics evolving? This is a stabilizer first market, right? So more than 80% of frontline patients, by our calculation, start on a stabilizer. That number is not really changing. And really what's changing is a majority of patients today start on Vindomax. Little by little, I think we're making inroads such that we are getting a little more share, right? So if you think about it, you know, let's try to make it very rough. If more than 80% are on a stabilizer, that means less than 20% are on a knockdown. Most of that is obviously going to Mvutra. of the remaining 80%. If I say half of the stabilizer patients, more than that are on Windermax, that means 55% or so, 60% are starting on Windermax. We have said our own share is north of 25% and growing slowly. And so that is how that is evolving.

Excellent. Maybe we shift gears to the pipeline because there's so much to talk about, but I think it truly seems like growing-based business, durable, and, you know, more to come in terms of some data on differentiation. It seems like that's the message. Okay, awesome. So, I don't know, it's like kind of dealer's choice what we should start with. We should start with limb girdle. Yeah, let's start with limb girdle. Yeah, let's do that. Because, I mean, this is probably one where maybe the unmet need is the highest. But, you know, Anna, maybe you want to kind of just lay the foundation in terms of, like, the indication and basically, you know, the therapy that you guys will be bringing to the market.

Yeah, absolutely. So BBP418 is being developed for limb-girdle muscular dystrophy type 2-iron-9. There are no currently approved therapies in the space. And as you said, the OMET need is incredibly high. We're super excited about the potential that BBP418 has in this indication. So we read out our interim analysis from our phase 3 trial last year. In that study, we not only saw differences from placebo that was statistically significant and met all of our primary endpoints, but the differences observed were actually on treatment. There were improvements from baseline, which is pretty much unprecedented in the muscular dystrophy field, and then there were declines on placebo that were consistent with the natural history. Overall, this patient population is around 7,000 patients in the U.S. and EU. We estimate currently in the U.S. is about 2,000 to 2,500 patients. So I guess what's a launch in this indication look like? um so yeah it's basically a very concentrated call point so around 150 to 160 MDA centers in the US where the vast majority of these patients that are genetically diagnosed are currently treated so it's a very targeted like capital efficient launch the we're very focused right now on finding patients so we've mentioned that we've identified over 500 patients currently and as I said most of these patients are currently at these concentrated call points so field force is currently our educating physicians payers and yeah we're anticipating that adoption will be very strong based on market research so far yeah I was gonna say so like in terms of like the doctors and their the education process like how long do you think that'll take and is it you know because this is a indication where there's really no therapies is this going to be where you know basically every can is or every you know per patient is a candidate essentially or they're gonna be some you know kind of narrowing of the actual true candidates that would actually be best to respond on the drug yeah great question so what we're hearing right now is you're absolutely right so most patients are going to be considered a candidate for therapy here and adoption is very high across all patients the The main sort of drivers of that are obviously that currently there are no available therapies these patients are anticipating that they have to climb year on year and we actually saw in the interim analysis that were benefits as early as three months on the ambulatory measures which is really driving urgency to treat and I think currently what would might that there's a lot of obviously patient need that will be driven by very strong patient advocacy groups to use that are doing a lot of the education on the patient side and then in terms of physicians as I mentioned we already have the field force out there I think we're not anticipating a bolus though just because I think the dynamics in these centers are that you're not getting the patients currently go into centers about once every year or twice a year so that might mean that sort of patients calling up physicians might have some there might be some delay in them getting into the centers I mean I guess when you think about these patients in their you know kind of journey to diagnosis like how long does that take and you know what's what's kind of your method of even just finding these patients?

Are there good, you know, ICD-9, ICD-10 codes to locate these patients, or is this still kind of like a lot more kind of digging in work that you need to do?

It's a combination. So basically right now there is a dedicated ICD-10 code, which is great. It went into effect last October though, so it's still relatively new. We are seeing uptake already, and it is continuing to see new patients come in every month. We also have data from genetic testing databases where we've been able to identify patients as while, but we're anticipating that that genetic diagnosis will continue to increase. Based on market research now, it seems like the standard of care in these specialist centers is that people are getting genetically diagnosed as standard of care, so it's really about patients getting to those centers, which is probably the delay in diagnosis, but once they're there, they are getting diagnosed efficiently with genetic testing.

Gosh, now this is a different type of launch than I would say at Truby, like more rarities, more focused, so I guess, you know, are there any key learnings that you can take from kind of the Atrubi experience and, you know, kind of apply it to the BPP419, 418?

Yeah, I think that there are two different archetypes of launches, right? There's launch in the competitive market, which is Atrubi and Infogratnib, and then there's launch in a market which you're building, which is 418 and ADH1 in Caleret, right? I think that some of the similarities, you know, there are some differences such as marketing and things like that, but there are some similarities such as how do you design a process which quickly gets patients on drug, right? So not the kind of payer side of access but more the patient and physician process side of access. I'll let Anna comment on that because one thing which obviously at Truby we have white glove service really working closely with the physicians offices as well as the patients to get them on drug and that is going to be key here also.

So maybe Anna I'll let you comment on how you guys are thinking about it and learnings from Truby yeah so I mean we're out there right now talking to payers the pair research has been incredibly positive as well like they're very receptive to the to the data that we've seen as we mentioned this is like they're not used to seeing placebo controlled data in the neuromuscular space and then also seeing these improvements from baseline and the functional measures is pretty differentiating so we're not anticipating sort of big coverage hurdles for access to reimbursement we are planning on offering a similar suite of services to what was mentioned for a true be for getting patients on drug at launch I guess it's a good you know pricing analog for little yeah so we the pay has always referred to the exon skippers here is the the most relevant pricing analog and I think as I mentioned that the main difference here is that we're seeing functional benefits and a placebo-controlled setting which they haven't already seen in that case so I think you could think that about those is like an analogous sort of the anchor and then we might think about premium pricing on that.

The three launches you know discuss the other two but do you think limb girdle has the the potential to be the fastest ramping or do you think there's kind of like you know some I don't know friction points just because it's a new indication it's never had a therapy approved there maybe I'll start and then I'll ask you to build on it so I think that we think all these three launches are going to be really good strong launches as Anna mentioned we don't expect there to be a bolus or anything like that in any of these launches look there are going to be new to market edits these are markets which we are building. There's going to be diagnosis which we are educating people on. All of that, especially for limb girdle and ADH1 is true, but nothing specific that I would call out as a pain point. It's all things that our team has diligently mapped. Actually, these guys have done a great job of mapping each step of the patient journey as well as each step of the physician journey and making sure that at every point where there is potential for friction, we have a plan for how to overcome it uh and so you know i think that it's they're all going to go really well we feel good about them uh and then i think your question was if if this one will be faster than some of the other ones and and on that one i'm going to ask you guys to do some you don't want to pick your favorite child i am told i am told that that's not nice yes exactly um that's fair All right, well, maybe moving on, let's maybe talk about incalorate and ADH1.

So a smaller population, you guys have been doing a lot of work in identifying patients. So maybe just talk about the setup there for that launch.

Yeah, so very excited about it, right? Because these patients who have ADH1, first of all, the prevalence is probably about 12,000. So there are a ton of patients who are suffering with this disease. currently we've only genetically confirmed and identified about 500 patients but we know that in the claims data there is already about 2200 patients so we know that there are more patients that we can go after and try and convert so huge unmet need their drug basically normalizes the patient right because your blood and serum blood and urine calcium levels become normal pth levels become normal. That is a profound effect. So we're very excited about that. I think that the call point is concentrated, but not as concentrated as limb girdle, right? I think here you're going after a slightly broader pool of endocrinologists, but what's very interesting is that there is extremely high overlap with hyperparathyroidism. Actually, if you have non-surgical hyperparathyroidism, and the guidelines recommend that you get tested for ADH1. What that means is we have a group of physicians who we can identify and target at launch and really try to, A, drive diagnosis there, B, drive awareness there, and then also try and convert some of their patients who are ADH1 patients onto our drug. In terms of, I think, how we expect the launch to go, very similar to limb girdle, I think that it should be a strong, steady launch, building on top of each other. It is an autosomal dominant disease. So one thing which we have seen is once we find a few people in the family who have the disease, we can do sponsored family testing and find a lot more people who have the disease. So that's actually a pretty interesting kind of difference between these two diseases. And I definitely do think that, you know, pricing here is going to be less than limb girdle, but still extremely strong. I think the comps that we've given on the low end are Yorby Path, which is about 300K, and then on the high end, UV Well, which is about 500K. I do think it's possible we're closer to the high end than the low end, given the drug has significant benefits. And that's how we think about it. And of course, what's very exciting about N-calorie is, and I know you're probably going to want to talk about this, is there is a huge expansion indication and hypoparathyroidism right behind it. We're very excited that we've kicked off a phase three trial there. We expect that trial to read out pretty shortly here by the end of 27, definitely by early 28. And that, you know, we have a beachhead with ADH1 and then we have a giant expansion opportunity with HP. And it could be the first oral agent approved in that condition, which I think patients will really wait for and want to be on.

Gotcha. And then I guess maybe just talk to the strength of the data and what you believe mechanistically for kind of the expanded opportunity and just general hypopara versus ADH1 and what gets you confident in running that phase three.

Yeah, I think the first thing which gets us confident is our phase two data set, right? So we did a small study, a small phase two study, 10 patients. These are some of the sickest HP patients. They didn't have the parathyroid gland. and completely independent of PTH, we saw normalization of blood and urine calcium in a majority of those patients. That's really profound benefit, especially normalization of urine calcium, which is a key unmet need area. I think second, and I'm gonna be a bit quick here because I know we're running out of time, but we expand, Neil actually expanded upon this on our earnings call, but mechanistically, there is a strong rationale for a agent acting on the calcium sensing receptor in the kidney to have an effect on MHP. And so I would really encourage you guys to listen to that. And I think he goes into a good amount of detail, which I can't cover here due to lack of time. So that's just the papers which are coming out. Some of that is very new, they've come out this year, has also really encouraged us to go after it. And then the third thing really is, we derive a lot of our confidence from experts in the field, like Dr. Mike Collins at NIH, as well as other folks who really understand these endocrinology diseases. And I think that that QL excitement is the third thing which has made us excited about starting a phase three here and helping a lot of patients.

Got it. So yeah, maybe let's move to Infragratinib just so we can get through it. But this one, excellent data, phase three data, and achondroplasia, first oral agent in that indication as well. And with the launch of UV well, we're definitely learning that the market is getting larger. And there's, you know, patients that were not really being, you know, they didn't want to go on Voxoga. So I guess now a couple, you know, months now into the UVL launch, where are you guys kind of thinking about, or, you know, are you more excited about the infrarotinum upcoming launch and ultimately the opportunity there for an oral agent?

We are extremely excited about the infrarotinum launch. We have been for a long time. What is remarkable is that we have a best-in-class drug, oral, best-in-class estimate on efficacy. but what is even more exciting and we presented at SP and we had a press release on this earlier today is we are moving beyond height now right the benefits are not just on height and yes we talked about proportionality which is a big deal in this community with our top line and now at SP we talked about sleep apnea ear infections the fact that infrarotinib is able to show clinically significant large benefits on these things which are on these measures beyond height I think is means that this will be able to grow the market significantly if you look in Europe about three-quarters or so of patients of kids are on some echondroplastic option in America maybe a fourth hour and really that difference is moving beyond height and having an oral option and that I think is the opportunity for infragragnib and I think that is why we are very excited to be able to, you know, we've partnered with the community, learned so much from them along our development journey, and now we are excited to serve them in the commercial realm, you know, assuming that the FDA approves the drug.

Gotcha. I mean, is this mostly, like, new start market? Do you think you'll switch patients? Like, how do you think, you know, you'll get your foothold?

And as you said, I think your data on body proportionality and that is going to be, you know, pretty meaningful for docs and for the patients so you know what do you kind of in envision you know early in the launch yeah so we are going to be pulling from three segments of patient of children the first segment of families and children that we're going to be pulling from our children who are on Voxoco or Ueval today you know they are on it because there's no other option they understand that moving beyond height is what they're looking for they don't want to take injections and so an oral option which has benefits, significant benefits beyond height, would be appealing to them, right? And that's going to be a key market for us, but of course that's a smaller portion of the children in the U.S. The second group of children are children who have tried Voxogo, and for whatever reason, either because there was no benefit beyond height or because they could not take the injections, they've decided to go off of Voxogo. We know that those children and those families want something. We suspect what they want is an oral option which moves beyond height and we think that we'll be able to offer it to them and that's the second group. The third group of families and children that we are hoping to help are folks who have never tried Voxovo UV well. Some of them are needle phobic, some of them really want to be on an agent which helps on things like proportionality and not just height and so that's going to be the third sort of segment that we're going to be targeting and i think we're going to see uptake in all three segments and and i think that what we are most excited about is growing the market so segments two and three but i do think segment one is also going to be important gotcha maybe with the last two minutes here you know chief business officer like as you think about you know the kind of overall business a truby pipeline launches like you know so we're going to have these launches there's going to be a 2027 event.

That's going to be exciting. But what's kind of next, right? That's what we're always worried about. It's like, what's kind of next in the pipeline? Where are you guys going to be kind of focusing, you know, your efforts? And as you said, kind of, where are you going to take your resources and deploy the capital?

Yeah, that's a great question. And I'm not surprised that Wall Street's asking what's next. So here's how we think about it, right? I think our first priority is funding and accelerating the Truby launch as well as the launches in Limb Girdle, Acorn, and ADH1. Best and highest use of our capital. I think that's what shareholders will reward us probably for over the next 12 months. Beyond that, I think that we really want to invest in our pipeline, whether, and, you know, the main idea there is our Hypopara trial. And so that I also think is on investors' radars and something that we're prioritizing. Beyond that, if we have excess capital, which we do right now, we're in the lucky position of having excess capital, having our losses come down over time, we always think about what's the right way to deploy capital. It could be through share buybacks, but it could also be at the right time. And our preferred path is to do it through organic growth. So we want to start new R&D programs, like I mentioned to you, and I think you are pretty close to this. You've covered a lot of genetic disease companies. There is profound innovation happening in our space and the opportunity to help patients is immense so we have a sister company called gondola bio where we own about a 12 13 stake in it at the right time maybe it's sooner maybe it's later but i i hope it's sooner we would like to integrate these companies use the cash flows and the capital which we have to accelerate that and to really make not just four medicines but to make 40 medicines and i think that that's the shape of company which investors, patients, and the community can be very excited about, and that's what we're aiming to build.

Jimmie, I think we'll leave it there. Anna, thank you so much.

Thank you.