BridgeBio Pharma, Inc. (BBIO) May 12, 2026 Conference Transcript

Welcome to the afternoon session on this, our first day of the annual healthcare conference in Toasty, Las Vegas. My name is Jason Zemanski, I'm one of the SMIDCAP analysts here at B of A. Very pleased to have joined me, the team from Bridge Bio. I have Tom Tremarki, CFO and President, and Chinmay Shukla, Vice President, Strategic Finance. Gentlemen, thank you so much for joining us.

Thank you for having us. It's great to be back in sunny Las Vegas here with you.

Excellent. So we'll end with maybe two or three minutes. So if you have a question from the audience, we'll pass around a microphone. In the meantime, maybe let's start with a little bit of background for those newer to the story. Can you just give us an overview of BridgeBio and a Truby?

Yeah, sure. I can start and maybe pass over to my fill in some blanks. So BridgeBio is a biotech company that was founded just over 10 years ago now. And we were created to do two things. One was to deliver as many meaningful medicines as we can to patients in the shortest possible timeframe. And two was to really be focused on things like NPV and cost of capital so we could make our business sustainable so we could be around for generations and generations to leave a big impact on the world and hopefully deliver a lot of medicines to patients that desperately need them. So one decade in, three approved products, including a Truby, which is our main commercial product today, just reported our first quarter where we did $180.6 million in U.S. revenue, so annualizing about $720 million in the U.S., clearly on track to break blockbuster status globally this year. and right behind that is a pipeline of products that are post-phase three now. Actually, the news of the morning is that our second of three NDAs was just submitted. So we'll have three launches in the coming months. First, limb girdle muscular dystrophy type 2i, that's BPP418. Second would be incalurate for DH1 and chronic hypoparathyroidism. And third would be infagratinib for achondroplasia, hypochondroplasia. Now, just to give you a high-level view of what this all looks like from a financial picture, we see a Truby clearly as a multi-billion dollar product. We think it's about $4 billion in peak sales here in the U.S., rapidly expanding market that we think can reach $20 billion. And then I think that next three products together can do at least $4 billion in aggregate. So we see $8 billion plus in peak year sales here, de-risked and in striking distance in the portfolio.

Yeah, and just to build on what Tom said, so beyond the $8 billion, we have expansion indications, hypoparathyroidism, and calarid is being investigated there. That probably adds another couple billion dollars to our peak year cells. And then obviously we have the hypochondroplasia trial with infagradinib. So I think that 10 years in, I think the company has de-risked $8 billion of peak year cells and probably will de-risk about $10 billion in a couple of years.

Great. Thanks for the answer. Just to dive right in, you reiterated guidance today and last week during earnings that a Truby could be a $4 billion drug. So maybe can you discuss some of the assumptions here, be it in terms of market growth, the competitive landscape, or the potential of your depleter program?

Yeah, happy to expand upon all of those questions, Jason. So let's first start with the size of the market. So TRCM is a market which is already very large today, and it's growing significantly every quarter. So I think we believe that it's a $15 to $20 billion market. We are already at north of 25% share in treatment-naive patients. We think we'll be at 30 to 40% share at peak. This is all based on volume, of course, and we think all of that, you know, translates to about a $4 billion or so peak year sales. estimate for the U.S. Now, beyond that, I would say, you know, we don't count our depleter program in any PKR cells estimate, just because it's a little too early. It's still preclinical. We're very excited about the program. I think that it's very synergistic with the stabilizer, which a Truby is, right? I think the stabilizer makes sure that you don't have any toxic monomer which is being formed and being deposited in the heart. The depleter goes and actually clears the toxic monomer already deposited in the heart and elsewhere so we think actually the two of them can work well together of course more to be seen on that front and we're excited to you know likely have it in our in the clinic next year or early 28.

Got it so again you bring up a good point I guess what would make a prescriber opt for a stabilizer like a Truby versus a silencer and we may have two on the market in short order.

Yeah, I think it comes down to three things, right? I think one is the efficacy. If you think about what is causing this disease, it's the dissociation of the tetramer into toxic monomer. So the more you can reduce the toxic monomer from forming, the better you're going to do on efficacy. And actually this experiment has now been proven out with all these trials having read out. A Truby is the only near complete product on the market. It's a near complete stabilizer. So that obviously is translated into really strong efficacy, whether it's, you know, if you look at the sort of when we disclosed our top line data, you know, 50% reduction in CVH at 30 months, immediate increase in serum TTR. What's most profound is separation on hard outcomes like ACM and CVH as early as one month. So that I think is the first big driver, right? Efficacy. I think physicians want to first look for efficacy because this is a deadly disease. The second thing becomes safety. And again, I think given Vindemax's long history in this category, I think physicians are very comfortable with the safety profile of stabilizers. Truby also has a very, very benign safety profile. And so I think that that's also been very helpful. And then the third thing really becomes convenience, right? I think for many physicians, for many patients, it's much more convenient to take an oral pill. it's safe you can take it every day you don't have to drive into the doctor's office as opposed to having to schedule injections which you know which come with a lot of complication as well as injection site reactions and also by the way even with the injection you still have to take vitamin a every day so you don't really get rid of the pill burden so those are the three axes that i think is the reason why physicians prefer stabilizers in the first line setting and then increasingly what we're seeing, and obviously, again, I would just say, you know, we've already said that we have more than 25% share in treatment-naive patients. So, you can really see a lot of physicians are choosing a Truby over any other product in this category.

Maybe if we could dive a little bit deeper on the efficacy front. You recently indicated you're going to present more data at ESC, obviously, without giving away too much. Can you frame sort of what we're seeing thus far and how that compares to not only the other silencers, but, you know, you're dealing with a gorilla in the room in terms of the other stabilizer.

Yeah, so there are really three things that I would probably, you know, put these ongoing research efforts into, right? The overall goal is to continue to establish a TRUBI as a differentiated, unique molecule in the ATTRCM landscape. And the first thing that obviously we've come out with on that front, which we actually came out with last year, is the importance of serum TTR, right? And we, again, reinforced it in a recent presentation. But we have shown that moving patients from Vendamax to a TruBee causes their serum TTR to increase. You don't see that effect when they move from a TruBee to Vendamax. And then what's most important is we've also seen that every mg per deciliter increase in serum TTR is correlated with about a 5% decrease in all-cause mortality. So a TRUBI increases serum TTR by about 3 mg per deciliter in the publications that we have shown. So that translates to about a 15%. Anyhow, that's overwind max. So that translates into about a 15% or so benefit on ECM. And that story is, I think, the first part of the story which has resonated. I think the second part of the story which we got out last year and we continue to push on is really strong data and key subgroups. So the two subgroups which we've talked about a lot are the AFib patients. About half of these patients have AFib. So the fact that we showed really strong data in that patient population showing reduction in AFib-related events also has been quite profound and has actually been a contributor to the second wave of acceleration that we've seen. And then the third thing, which I think we're expanding upon right now is really the unique renal protective effect of a TRUBI as well as, interestingly, a bunch of real-world evidence. So both real-world evidence from independent groups as well as real-world evidence published by Bridge, which kind of compares a TRUBI and Rindemax on various outcomes such as cardiovascular hospitalizations and other items. So in all of these things, we've seen a Truby show clear and consistent benefit, and it's not really one piece of data. It's the totality of the data, which when physicians look at it, you know, gives them confidence to, you know, use a Truby in a lot of their patients.

Let's pivot to the near-term sort of growth outlook. What should investors be thinking about as they update their models heading into 2Q the rest of the year? What dynamics, either tailwinds or headwinds, might the street be overlooking?

Yes, I would say the tailwind would be simply that we continue to drive growth in the frontline setting. So we've, from day one in this launch, been focused on gaining share and positioning a Truby as the first-line go-to agent of choice. Maximal potency, near-complete stabilization in a disease driven by destabilization of a tetramer. So we've grown share up to north of 25% through today, and I think we'll continue to do that into 2Q while the market expands. Now, at the same time, we've seen roughly a splitting of second line overall with the knockdowns. And interestingly, this is a more recent trend. While combination use is a relatively small piece of the market, our share there has grown three times in the last quarter. So increasingly, that tells us that physicians are believing that Attribute is the best stabilizer. And so when they want to reach for a combination, they're putting the best stabilizer on, plus whatever knockdown they have available to them.

What about the potential for further acceleration? I know one of the hallmarks of your patient assistance program is 30 days free drug. You know, as those patients start to become paying customers, what kind of boost, you know, and how do we model that moving forward quarter to quarter?

Yeah, so as we discussed early on in the launch, this really is more of a timing from demand to revenue issue where when you first receive a script, if it's a fill using the first month free, it's simply one month delay until you start getting a paid script that translates into net sales. And so that was a much bigger impact on quarterly numbers early in the launch than it is today. The program is still very popular, although in any given period, fills that are new starts are a smaller and smaller proportion of total volume. So less impactful today than it was a year ago. You know, next quarter, the following quarters, it will be even that much less impactful.

What gets you more into some of the prescriber offices? Is there a friction point at this point? Is there something unsettled? Same with community prescribers. I mean, how do you reach those who either aren't treating ATTR patients or alternatively are reaching for the silencer first?

Yeah, that's a very good question, Jason. I think it comes down to two or three things, right? The first is, you know, it takes time, right? It takes time for us to go visit everyone to make sure that the systems are wired and to get the physicians comfortable with using a Truby. What's interesting is we have noticed that once a prescriber starts using a Truby, they're actually quite likely to keep using it and use it in more and more of their patients. So we've only been out there for about a year or so. So I definitely think that's the first angle. I think the second angle is it takes a little bit of time also for the data that we generate to really be out in the community. for example you know the serum ttr data as well as the afib data which i talked about a few minutes ago we generated that kind of q1 q2 of last year and really the acceleration that we saw in the psfs which which which we were we used to report until february was really in q4 and really in q1 of this year and so that's the six to nine month lag and that's just because you know you have to go to a conference present it make sure that physicians actually know the information and then as our salespeople always remind us we have to tell the physician seven times before they retain that information and and you know you start to drive some of the changes in behavior which are very important here so that certainly is also an element and so we do expect that as we get more real world evidence data as we get more of our kidney protection work out there you know we'll be able to talk about it with physicians and as there's more awareness eventually it will lead to an increase and further increase, I would say, in treatment-naive patients as well as switch patients being on or should be.

Maybe one on the, probably one of the biggest overhangs until recently, the IP case going on with one of your competitors. You know, when you were discussing this in last week's call, you indicated this added confidence to that $4 billion peak potential number. I guess, can you A, talk a little bit about when you expect sort of, you know, to reach that peak market share? And then B, in terms of the payer response once a stabilizer becomes generic, what's the impact there? And again, it gives you confidence that it's really not going to be as big a hit as I think some of the more concerned investors were.

Yeah, happy to touch on both of these points, Jason. So in terms of peak sales and when you reach time to peak and all of that the way i always think about it is we already know that we have north of 25 percent treatment naive patients today our goal we have said is 30 to 40 percent there so in terms of patient volume we've made a lot of progress already it usually takes a few years for that to translate into peak year sales and and and that's kind of why you know on our call we mentioned given the fact that we now have seven years or so for a truebie to truly be on the market with only a branded stabilizer competitor, we feel like we will be in a very good spot and kind of reach peak by that time, maybe with some room to spare. So that's on the first piece. And that's one of the big reasons why, you know, we feel very confident about the error bars on our valuation, as we say, as having narrowed quite a bit. The second thing, which I would say in terms of the payer response, we don't really see this category as being, you know, highly managed. The first thing is, you know, a Truby is a differentiated product. And, you know, you can see that in terms of despite having an incumbent on the market within just a year, more than a quarter of new patients are started on a Truby. That tells you that we've been able to overcome a lot of resistance from physicians, which really is because they do view the product as a differentiated and unique product, which is right for their patients. So that I think is the first part, which gives us confidence on the payer side and then on the second side what i would say is if you map just the ecosystem dynamics given this is party and a lot of volume flows through specialty pharmacies usually you don't see any difference to the patient cost as well as to you know there's no real incentive that we would see negative incentives so you know that's on that piece and again what i would say is you know it's fine to talk about it on theoretical basis which we're happy to talk about. But if you just look at analogs, such as the prostate cancer market with Zytica or Gleevec, you can see that when part D, you know, first to market, part D molecules go generic, the second to market, definitely the second to market, more differentiated molecules don't see any really hit to their sales. It's really the growth rate maybe slows down a little bit.

But as we were just discussing previously, we think we'll be, you know, at peak by then. excellent well you have a rich pipeline so i'm going to let's switch to that jackie from the team yeah uh so let's pivot to bbp 418 so from a commercial standpoint can you discuss the efforts to identify the roughly 7 000 patients and is this likely the biggest challenge yeah great question so uh just just to to start with a little bit of background so bbp 418 is a oral small molecule for a disease called limb girdle muscular dystrophy type 2i.

We think there's about 7,000 patients between the U.S. and Europe, and probably another 2,000 that are actionable with this molecule in Japan. So fairly large, you know, in context of other types of genetic diseases. And you're spot on. I think much of our effort today in developing this market and shaping the market is focused on creating awareness that there's a drug coming so that, you know, there's some urgency to get in. And if you're not genotype, get in your physician's office and get a genotype test because that will make you eligible for therapy when we're approved. And two, I think just driving awareness of the remarkable data that we've delivered from the phase three study that read out last fall. I think it's worth taking a moment to rehash a bit of that. So this was a placebo-controlled randomized phase three study in patients with limb girl 2i. We read out what was meant to be a 12-month interim readout, mainly focused on a biopsy-based biomarker that was meant to demonstrate that this biomarker is a surrogate. And what we saw, surprisingly, was statistical significance on every endpoint we looked at, including all of the functional endpoints, including what was meant to be the full approval endpoint, the North Star test, as well as walk and other function-based endpoints. all at 12 months. Not only that, but patients who received 418 actually improved from prior to starting therapy. So really profound effect here and a data set that is really unique in context of what's been shown to date in any type of muscular dystrophy. So we were at the major conference in this space, MDA, in March. I think it was one of the most important presentations at that conference. There was a hugely positive response from the KOL community and a huge amount of inbound, either from patients to their prescriber or directly to us through advocacy groups on the back of that. So we've got great, great momentum, great product, but there's still work to do over the next couple of months to continue expanding our pool of diagnosed eligible patients and driving awareness and urgency to act when we do get that FDA approval.

Yeah, you bring up a great point. I think the Fortify study, you were meant to have an additional 24 months of analysis in order to evaluate the North Star, this metric that tracked function, you know, absent that data or as that data is still evolving ahead of, you know, a likely submission, your submission and then potential approval, you know, is that going to affect things at all? Do you think some docs are waiting for the kind of the final read through or, you know, is it, you know, potential that we might see even better data as there's greater exposure and kind of maintenance of some of the function?

Yeah, great question. So I don't think physicians will be waiting to see longer-term data here. I think these data are very convincing and really unlike anything they've seen in caring for certainly limb girdle patients, but patients of all types of neuromuscular conditions, quite frankly. And so I think the fact that we were able to see such a robust and rapidly occurring effect, not on just one endpoint, but all of the endpoints, right? We had FEC, we had Northstar, we had a walk-based endpoint, as well as all of the biomarkers like CK and the biopsy-based endpoint. I think it is as convincing as a data set can be. So I think there'll be, I think physicians will want to use this drug as soon as they are able to have access to it. Now, there is one question around whether we would receive accelerated versus full approval, and that has some implications on what happens to the ongoing three-year endpoint here. And personally, I don't see it as ethical to continue the study given the results to date, but unfortunately, it's not up to me. And so, but we'll be waiting for some feedback from FDA on this, but our base case is that the study will continue and that will get approved on an accelerated basis and complete the study.

Great. Let's pivot to Encalaret, ADH1. You announced this morning that you had submitted the NDA for FDA approval. I guess one of the biggest inbounds we're getting here is what gives you certainty that the market for ADH1 is 3,000 to 5,000?

Yeah. So as we think about Encalaret, and especially in ADH1, they're really two factors which give us confidence on the billion-dollar estimate which we've put out for PKR cells. On the prevalence side, I would say, you know, we've already identified almost 2,000 claims analysis patients. And as we drive further diagnosis, whether that's through screening, you know, we sponsor genetic tests for patients. And the guidelines say that if you have non-surgical hypopyra, you should get tested. And almost a fourth of the patients are positive. So that's been a big source of new patients, which we have found. Another thing is, given we have claims, we've been able to leverage a lot of machine learning and artificial intelligence tools to further identify patients. And then the last thing is we've been doing sort of sponsored testing at family events because this is an autosomal dominant disease. And so if you find one person in the family who has it, it's quite likely that there are other people in the family who have it. And so I think that that's been another source. And of course, all these sources will just accelerate as we get on the market and people actually feel better and are benefiting from the drug because then they have even more incentive to, for example, tell their family members that, hey, not only might you have this, but there's this wonderful drug which can probably help you a lot. So that's kind of on the prevalence side, but I do think that, you know, we have about 2,000 patients in claims today, and as we keep up with our efforts, that number should only go up in terms of patients who we've diagnosed who can be on therapy, ultimately probably reaching close to that 12,000 number, which is the prevalence. In terms of the price, though, because that's the other big component here, right, and I think that, you know, just given the high unmet need in this patient population, and given the complications here, as well as just given the profound data that Encalirate has generated with more than three-fourths of the patients normalizing their disease, that's pretty remarkable. I mean, you don't really see data where disease is not just stabilized, it's, you've actually reversed it, right? So you've kind of normalized the whole thing. So I think that, I think that that means that we're set up well for really premium pricing here. And so, you know, normally in these settings, what we tell people is Chris Vita is probably on the low end and Exondus is probably on the high end. But you multiply, you know, it's a pretty good range for pricing. You multiply the patients you already have today with that kind of price point, gets you very close to that billion-dollar number.

Yeah, I would say for both Limgirdle and ADH1, we're more and more confident every day that these can both be billion-dollar-plus products for us here. When you consider the strength of the data we reported, plus everything we're learning out in this market between response from advocacy, response from KOLs, and just the patient counting that we're able to do now that we're out there in a more of an organized way developing this market, I think our confidence is growing.

You bring up a good point. As you expand into the chronic hypoparathyroidism indication, you know, the number of patients there are about 200,000, about tenfold more than what you see for ADH1. So in terms of pricing, I mean, both indications you've indicated could be a billion-dollar opportunity.

Does that mean that you'll maintain sort of that premium pricing and look towards maybe those who are underserved by the um analogs or you know is there something else in play here yeah so of course the billion dollars we say for incalorate is just for adh1 does not really include the hp opportunity at all so just want to make sure that we start there because it's important second in terms of pricing the way we think about it is we're going to first launch an adh1 that's the community we have an opportunity to serve here at the start so we're going to price it kind of thinking about that community in the future you know my hope is we certainly get the opportunity to serve the HP community also and at that point we can you know look at the pricing but but we also have to look at the data which we've generated before we think about pricing and then the other thing which I'd say is look it is an oral option which you know convenience is a big factor and it also has some unique benefits in HP in terms of normalizing both blood and urine calcium so premium pricing is not unjustified in any way, but let's first look at the data and then decide. For now, I would say let's just think about ADH1 for the pricing side.

One quick one on infragratinib. Obviously, the data Propel-3, very strong, but if you talk to most physicians, height is sort of a less of a factor than the medical sequela of the disease. At the same time, there are some concerns about the MOA just given, you know, the history of the molecule. So do you think that prescribers are going to wait and see what both, you know, the kind of the longer term endpoints and the longer term safety are going to look like before feeling comfortable here? Or do you think that there's kind of strong adoption points just given the convenience of the pill?

Yes, I think really it's three legs on the stool that make this a really exciting product. One, first and foremost is the efficacy we've seen. So category leading effects on the primary endpoint, which is change for baseline and annualized high velocity, which is not the most important health issue for these children, but it is a marker of potency here. Second, we've seen for the first time in this space, a statistically significant effect on proportionality, which suggests in a timeframe that's relevant to these children and their parents, they should expect to get some benefit on functionality physical function due to the disproportionate short stature of this condition um so that's efficacy second um the convenience of this product when you compare it to a daily injection or even a weekly injection is a huge deal here these are children that will be treated from the time they're toddlers at first then eventually we'll get all the way down to infants um through when their growth plates close which can be as late as 18 years of age um they really want something that's more convenient easy to give less conspicuous is something that matters also it makes them feel sometimes we've heard from the community that the fact that you have to have your pen with you and always have an injection if you're at summer camp or something it makes you feel different which is not always something children want want to feel um and third is safety i mean as far as i can tell from reviewing all the data And Infragratinib in the large phase three and all of the phase two data today, it's got the best safety profile. So it's kind of like, what more do you want? Clean safety, great efficacy, best form factor on route administration.

Maybe we have time to squeeze in one more, and naturally it'll be complex and difficult to answer. But you've had an amazing run of success in terms of clinical output. You know, that said, you're looking at three non-overlapping launches. is how do you manage these at the same level of execution that you've hit with a Truby?

Yeah, so first I would start by saying we're privileged to have tremendous leaders across the board here, Matt Outen that leads our commercial team, but also the CEOs that sit on top of each of the affiliates where we're about to launch these products are really remarkable leaders and operators and colleagues. So I think that's crucial here. So great team. Second, yes, they are in distinct conditions, but we do benefit from some of the more general commercial infrastructure that we get spread across where we've already demonstrated excellence. So access, for example, or hub services, these kind of things, one team that gets deployed across products. So there's some efficiency and synergy there. and then I think we benefit from having a very close and dedicated relationship with each one of these prescriber patient communities advocacy communities at the level of each asset so we already have a strong foundation to build upon and so from here the the really the lift is just bringing on sales forces for each one of those products which are relatively small and manageable and, of course, field medical. We're in the process of doing that right now. Tom, Chinmay, thank you so much for joining us. Thank you, Jason. Thank you all for joining us.