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

um great welcome back everyone this afternoon session of our second day the 2026 learning partners global healthcare conference this is uh my money ferruhar senior analyst drank medicines um welcome everyone to miami and i am very pleased to be hosting chinmay shukla sbp of strategic finance uh from bridge bio uh chumai how are you doing i'm doing great money thank you for inviting us to the conference for hosting us and thank you to the broader layering team as well as all the investors for the interest we are deeply grateful and happy to answer any questions you guys might have great let's dive in some of this will be a little bit repetitive previous meetings we've had before of course but let's try and get into a little more depth so i'll start with where we are on ttr obviously the launch has been accelerating in absolute terms with new patient ads an experience a little bit different than some of your competitors, although this market is complicated, so apples and oranges comparisons are always dangerous. Talk to us a little bit about where we are in terms of the evolution of price and volume growth respectively in terms of bringing in earlier patients, and do they have a different severity mix, do they have a different payer mix, et cetera. Like how do we think about how that market evolves as we go into a quite mature multiplayer market?

Yeah, happy to talk about that. So I think the way I would think about this is our patient growth has accelerated. So just to remind everyone, we went from about 60 patients a week from when we launched at our update last year, about a year ago exactly, to now having more than 150 patients a week as of our last update. So there has been substantial acceleration and actually what we saw is two waves of acceleration, right? First start launch accelerated from call it 60 to 100 and then it's accelerated again to this higher number. That's pretty rare in genetic disease launches and we've been studying as to why we've been able to accomplish this. It comes down to a few factors, right? I think the first thing is the clinical data. So it starts with the fact that we are the first and only near-complete stabilizer on the market for this disease. It's a devastating disease. Physicians and patients have really wanted a potent molecule like a Truby and have reacted well to it. And beyond the clinical data we generated on our label, we've done more work to identify the power of a Truby. I will talk to you about the AFib data, which we've talked about before, as well as the variant data. and all of that has resonated very strongly with the physician community. The second thing which has caused this acceleration is just more patients are being found. That is a function of awareness of this disease going up. It's also helped a little bit by some of the initiatives we have undertaken like using AI for diagnosis. The third thing which has happened is that the prescriber base has broadened. So we've always talked about academic centers, we've talked about community physicians. I think that there is an important nuance which gets mixed, which gets missed here, and that is the high-volume heart failure clinics. And I know we've talked about this a little bit before with Humana, and I will say you've been early on this bandwagon, but really now what we are seeing is these high-volume heart failure clinics, you know, especially with the AFib data, which is, you know, half of these patients have some component of AFib, they're getting much more confident at keeping the patient in their practice as opposed to sending them to an AMC, and that's been very powerful for a Truby. All of this has meant that our patient volume has gone up quite a lot, as you guys have noticed. Primarily, the strength is driven in treatment-naive patients. As we've discussed before, that is the segment of the market which we are focused on, where we continue to win very strongly. We have north of 25% treatment-naive share just a year into our launch, and at launch, we had said that our goal is to be north of 30 percent so we feel very good about where we are there finally on the net price dynamic net pricing for a true b has been very very stable um you know we have not seen really any changes in our discontinuation rates compliance um you know paid drug all of that our growth to net has been um you know in that 30 to 40 range which we have guided to as our long-term growth to net you know it can move around a little bit in there i think in the first quarter maybe it's going to take up a little bit but I still think it's not going to be very noticeable and it's going to be well within the range that we have given out to people.

So let's talk a little bit about the European experience. You guys don't sell buyout to yourselves, that's through your partner buyer. Talk about where they are in terms of sequence of launches. You're being a little more of a complex country by country kind of hand-to-hand combat launch and talk to us a little bit about where they are into new patient share in the countries they're launched and timing that we should think about for incremental new launches.

Yeah so that's a great question and we're very excited about the potential for Acramatis not just in the US but globally because we do think that this safe oral small molecule stabilizer can be the backbone of therapy for all patients worldwide. We've been very grateful for our partnership with Bayer but I think that we've been very pleased with how that's gone so far. We expected it would go very well, and it has. Specifically, since it's their information, I'm gonna be a little more careful about it, just to be a good partner to bear. But I think what they've said before and what we've said before is it starts at Germany. That's the first country that you launch in in Europe. And in Germany right now, they have north of 50% treatment share. Remember I said here in the U.S. we are at north of 25%. Part of that is because in Germany there is a single payer system, so you don't have some of the access challenges that you seem to have in the U.S. You also have systems which can actually cross-trial, compare between all these different data sets. And when you do that, it's very clear that a Truby offers the most benefit and it's also priced in the most competitive way. So that's where they are at in Germany. I think beyond that, the next launch will be, I think in Denmark and then from there they're going to go on to broader countries like Spain Italy France so on and so forth and I think that it's been disclosed that they've won the bid in Denmark so it's going very very well we're very excited about the momentum there we get you know our royalties on beyond our sales start at 30 percent and go up to mid to high 30s so that's to be a pretty significant uh sort of stream of income for us and uh watching them launch in europe has also taught us on how we should think about our launches for the next three drugs which we're going to have in house so so it's been a great partnership and we're really grateful for

it let's talk a little bit about uh combination therapy i'm going to move out of ttr momentarily i promise um how do you think about this market evolving whenever tefaminis becomes generic and how that will affect combination therapy and your positioning versus competitors?

It's a great question, Mani. So maybe I'll just use this opportunity to touch very briefly on our thoughts about TAF IP. And it is a little odd to comment about someone else's IP, and I want to be upfront about it. It's not something that we really want to do, but I know that that's been a topic of conversation amongst investors. So I know that there are, you know, we've mapped three potential outcomes one being pfizer is able to prevail second being pfizer settles the third being there is a generic entry in 29 we think that there is a very high likelihood that pfizer in some way prevails here whether it's by winning outright or by a settlement which pushes it out into the 2030s but regardless of whenever there is a generic right i think the question becomes you know if you look at any other category where a first to market went and generic, which we think here will be in the 2030s, potentially 2035, and there was a second-to-market, more potent drug available, the sales of that second drug have continued to grow across all sort of analogs that we have studied. Specifically, to answer your question on combination therapy, I think that there are a few things to consider here. One is that there has been no benefit shown for combination therapy, and that's just what the data says. Actually, that's also what the guidelines in the U.S. say. And U.S. guidelines tend to be conservative, but even they say that there's no benefit shown with combination therapy. You see that also in the clinical sort of commercial experience that we are having right now. There's not a lot of combination news going on out there. I will admit there are some people, there are a small group of physicians who really want to try and use it, but it's not really a big portion of this market today. I think finally, in terms of evolution of this marketplace, I would say that ultimately our goal here is to establish a Truby as the most potent stabilizer. And regardless of whether people want to try a combination with a knockdown, which I don't think makes a ton of sense, or a depleter, which I do think makes a ton of sense, we think that having the best background therapy, which is the best stabilizer, as the starting therapy, and then you can add things on if you need it, is the way to go. And that is where I think the field is going, and that is where a Truby is positioned.

Let's talk about the depleter. you have brought in a depleter of your own as part of strategic expansion, life cycle extension, however you want to think about it. When you look to the NeurImmune, Alexion, AstraZeneca, sort of nested Russian dolls ownership of that asset, when you look to that data set, how is that going to inform your own strategic plan for your depleter?

That's a great question. So we are looking to see the data that reads out. I think it's going to, it might influence how we design our trial. But before we get into all of that, maybe let me take a step back and explain to you as to why we are so excited about our depleter program and why did we decide to start one in-house as opposed to buy one of the existing ones, which I know a couple of them were being shopped around. So it really comes down to the fact that this is a mass action disease where the tetramer is dissociating into monomers. the monomers can then clump in your heart and that can cause cardiomyopathy the therapy which we have in a truby is a very very powerful drug but all it does is it stabilizes the tetramer so it doesn't really directly affect the monomers already deposited there are some natural clearing mechanisms through which the heart can remodel itself but you know a truby is not doing anything to significantly accelerate that a depleter could theoretically go in and start to remove some of these monomers, and that could be an interesting way to almost reverse the disease in a way. And that is why we think it is very synergistic with a stabilizer, and that is why we've been very excited about it. The other reason why we, you know, so then we went out and looked at depletors, and we felt like all of them had some issues which we felt we could do better at, and that is where Dr. Richard Scheller comes in. He's our chairman of R&D. We've worked with him for a very long time he is an antibody expert as is widely known and when we sat down with him and we thought about what would a good molecule here look like we realized we could design one on our own and that's why we started our in-house depleter program it's going to be in the clinic probably at the end of next year or early in 28 and I think as these other trials read out we'll think about the patient selection as well as where exactly do we take this and how do we trial it based on that data so it's a little too early to give more specifics than that but that's how we are thinking about it and that's why we're very excited about this opportunity so let's slide from TTR down the pipeline chart

to a contraplasia a fairly mature market but when they're still growing you've got a couple of agents in there although both are different mechanisms of action than you. Talk to us about your strategy for U.S. launch, switch versus new patients, and then we'll move on to OUS after that.

Yeah, happy to talk about that, and thanks for the question on Infragratinib. So, you know, before we talk about the commercial launch, it is important to be grounded in the data and the clinical profile, because that guides a lot of what happens commercially. So just to very quickly recap what we have seen, Infragratinib targets the disease at its source. And like you mentioned, it's a, you know, it's a differentiated and I would argue a much better and direct way of addressing achondroplasia as well as hypochondroplasia and other targets. These are all FGFR3 gain of function mutation caused conditions. And so targeting FGFR3 directly is very helpful in that context. And that's what we do. So we target both the JAK-STAT as well as the MAF-K pathway, whereas some of our competitors only target one of the pathways. So that mechanistic superiority has then translated into significant clinical differentiation, right? So whether we talk about height where we have the best point estimate at 2.1 centimeter per year, but beyond that height is not the most important thing here for the community. It is proportionality which is what matters. We're the only agent that in a clinical placebo-controlled clinical trial at 12 months was able to show a statistic effect on proportionality And I think people are really underestimating the impact of that data set and what that means for the community. Beyond that, the drug was remarkably safe, right? We had less than 4% Hyperfos, and we didn't really have any other major AEs that anyone was worried about. And then when I take that whole clinical data and I put it together with the fact that this is an oral agent, that really means a ton of good things for the commercial opportunity. So that brings me to directly answering your question, right? so a profile like this should help us get a lot of switches because there are people you know taking 3,000 injections is just not that I think for children and the families who are going through this it is much better for them to have an overall option available so we're proud to have that hopefully be available next year to them and I think that that's going to drive a lot of switching and in the naive section I would say there are two further segments of the market right one is whatever is the incident population which you know we'll have a fair shot at but the other segment of the market which people are not really paying a ton of attention to I would argue is the expansion of the market by bringing online children and families who currently don't take any sort of an intervention because either because you know they don't want to take an injection which which can be very painful and I know that now there's some talk in the community of actually having to monitor for hypotension on the competitors which is a pretty major issue with children as well as excessive headiness the other issue is proportionality right like you want to see effects beyond height it's not a cosmetic thing it is a there's a situation where we can actually affect the well-being of the child that is where this proportionality data will help us expand the market and i'll leave you with this tidbit you know we've analyzed about five or six hundred launches and i'm going to publish a paper on it channel sounds very nerdy but one of the things that we are now able to do is look at cases where you had you know not just cherry pick but look at all cases where you had injections in the market and then an oral came on and when we analyze a bunch of those analogs what we see is five years after the entry of an oral option the market expands by about threefold and that is the real opportunity here on on bringing online new patients who are new children who were not before being you know having this option as well as getting switches.

From there, let's go out to look at the global opportunity. Obviously for achondroplasia, OUS is a larger part of the market versus US than in many other indications. This is common in a lot of rare diseases. Talk to us about how your approach to launching is in different geographies and other places where you would be more likely a distro, a partner, etc. How do you make those decisions?

Yeah, so we are currently going through that at our company. We have an excellent head of international that we've hired is used to work formerly at Alexion this is very experienced individual that we're very proud to have on our team now and and he and his group are now working through how to you know map the global launches for the next three products that we have we are we have retained worldwide rights to all of them that's kind of important in this climate I would say and I think what I would also point out is I think the opportunity for something like acondroplasia is probably ultimately going to be similar in the U.S. and Europe for Infragratinib, for an oral option, right? Just because I think there are more people who are needle phobic in the U.S. than Europe. And so in Europe, the injections, you know, don't have some of the issues that they have in the U.S. So I think it's going to be a very standard launch. I think that we are probably, the regulatory filings are probably going to be three to four months behind the U.S.

The launch is going to be three to four months behind the U.S. we're going to start with germany and from there we're going to cascade out to other countries our plans are still being finalized on that point right now so so more to come as we further solidify it but hopefully this gives you the picture for the next 12 to 18 months i want to talk a little bit about how to think about the achondroplasia market in terms of timing of your launch versus timing of your hypochondroplasia data and how your medical science liaison strategy plays out to get those same physicians to treat those same patients to get leverage on that data set?

Yeah, that's a great question. So, I think that let's start with, maybe I'll first map out the ACON timelines and then and I'll overlay the HypoCon timelines, and then we'll try to weave the two in together, right? So on Acorn, we expect to file our NDA in the second half of this year. It has breakthrough resignation, so we think that there's a good chance that it gets priority review. Ultimately, of course, we have to have the dialogue with the agency, but I think that that sets us up well for a launch in the first half of next year for Infragratinib in Acorn Replasia, and then from there, you know, we'll continue to go forward. On the HypoCon side, you know, we kicked off the trial last year for our phase two, phase three, our trial for HypoCon and using Infragratinib. Actually, the enrollment of the trial was extremely quick. It was much faster than what we had modeled internally. And we expect to get some phase two data out second half of this year. It is also important to note that our top line and feed data was so strong for ACON that we have now accelerated the HYPOCON trial, and so it's now like going full speed ahead with its phase three trial portion. The way all this plays out is we'll be able to go talk about the further expansion into HYPOCON, you know, in a compliant way with our MSLs, with the KOLs, and there's going to be a steady cadence of data first, you know, this year, more at conferences next year. Remember, we are also going to keep publishing on our ACON data like we have done with a Truby. And then ultimately, I think that that trial probably reads out in 28 or so. And that's kind of how that's going to go out.

Okay. So talking about how to think about the margin profile of the company. Obviously, the balance sheet issue has become markedly less acute. There's a lot more comfort as you've addressed cost of capital, refinancing, et cetera. Obviously, the revenue growth has helped. Not the most brilliant, not the most innovative thing I've ever said. Growing revenue is good. But let's talk a little about what happens to the margin profile as you roll each of these assets on. As you roll on Akon, as you enter ADH1, et cetera, limb girdle. How should we think about modeling gross margin and how much of that drops to operating margin from an operating leverage perspective?

Yeah, so I'm going to maybe not get into super specific guidance right now, just because as much as I like creating news, I don't like creating news that much. But I think, let me try to paint a picture of how the company is going to shape up because it's very interesting and it's something that even experienced people have a hard time truly grasping because one, I don't think that, I don't know of any other company who's launched four products in such quick succession all in huge markets with three in one year potentially. so it's very unusual position to be in it's a privileged position to be in and we hope we can capitalize on that to serve patients I think the way I think about it is these are all small molecules they're all very easy to manufacture so you know our cogs expense is not that much it's it's you know very low and I know that while I'll not give guidance I know that cell side models are that 95% or so and that kind of intuitively makes some sense to me again and I wanna emphasize that's not our guidance, that's how it seems to me Wall Street is modeling it. Beyond that, I think most of these assets don't really have a heavy royalty burden. They've sold a small royalty on a Truby of 5% to Blueall and CBP, although that hits below the line. And then we have a royalty out to Novartis and Inserm on Enfy. Otherwise, they're all de minimis royalties on ADH1 and Limgirdle. As I then think about going from gross profit to operating income, you really have to think about R&D expense and SG&A expense, right? And what I would say is that our R&D expense is, you know, flattish from 24, from 25 going into 26. It's probably going to drop a little bit for the existing portfolio starting 27, 28. The reason for that is, you know, we've been very disciplined on our burn and our finance team has done a remarkable job of working with the business and identifying opportunities to optimize costs. I think that we have multiple trials ongoing right now with pediatric ADH1, hypopara, hypocon, and we're also on OALYs for all the phase three trials which we've just read out over the last three, four months. So there is R&D expense, but we are working very hard to maintain a flat or a flat spend there. Our SG&A expense is going to go up. It is interesting to note that if I look at the total spend of the company, operating spend, cash spend of the company, we were at about a hundred million before we read out Akramedis phase three. To launch Akramedis we went up to around 200 million of total spend and I think with these next three launches we're gonna be somewhere around 300 million maybe a little higher maybe you know somewhere around there by the end of 26 going into 27 so you are getting a lot of lift here where you're doing three more launches for the price of your first launch and so I do think that that bodes very well for long-term margins and those are one of the points that we were trying to make at our earnings call that just the ability of this engine to generate cash which we can then reinvest in R&D at higher hours to serve patients and to serve shareholders I think is is quite underappreciated in my personal view so let's talk a little bit

about these launches we talked about a con I was about TTR ongoing let's talk about when we're going to see the transition to commercial for limb girdle how we should think about you know about pricing launch velocity obviously it's a little bit different than TTR you don't have a you don't have a big pharma building it up for three years ahead of you ahead of time but the The market's kind of yours in this patient population. So walk me through how we should think about that.

Yeah, so let me talk to you a little bit about it. And again, just to briefly recap the data, right, we hit stat-sig on not just the biomarker, but also functional endpoints like FVC, 100-meter time test, as well as NSAID, which is super important. We're going to have a plenary talk at MDA, I think today or tomorrow, and so I would encourage all of you guys to check that out because I'll have some more data there. I think this kind of profile where you're actually not just stabilizing a disease but reversing a disease means that we should be able to get value in terms of pricing as well as I think that patients are going to be excited to be on this drug. So I know we've publicly said that, you know, there are somewhere around 2,000 patients in the U.S. who have limb girdle. You know, maybe half of them have already been identified. So that's going to be the target at launch for us. And then from there, we are finding more patients. I think that we are looking at misdiagnosis done, currently hiding in Becker's, as well as some of the other limb girdle dystrophies. And that's kind of where we're finding some more patients, too. So it's a very exciting opportunity ahead of us there, and it's a billion-dollar-plus molecule.

Sliding down the pipeline chart further, if you don't like this you should probably just stop having so many positive pivotals i like this very much uh we go to adh1 uh talk obviously a different size market different maturity etc talk about where we are in terms of timing towards pivot towards filing and approval and that model bill that's a little bit different yeah so adh1 is going to be a true who, you know, it's significantly underdiagnosed.

In many ways, it reminds me of the TTR market when Windermax was launched. We've done a good job of identifying almost a couple thousand patients in the ADH1 market, but we know that the prevalence in the U.S. is about 12,000, so we have a long ways to go. Again, the data there has been very helpful. I know I probably sound a little nerdy talking about data on every question, but the clinical data does guide the commercial opportunity, and here we have a molecule which basically cures the disease in more than three-fourths of the patients, right? It normalizes blood and serum calcium both. And so that's helped us. I think we're doing a lot of patient finding through algorithms, but also through just old-school family mapping, as well as testing programs, because, you know, a sixth of the hypopara patients probably have ADH1. So we're trying to drive awareness that, hey, if you have hypopara And if it's non-surgical, you should probably screen the patient for ADH1 and test them. It's an easy-to-use kit. And that's also helping us find a ton of patients. And we disclosed more than 1,700 patients in claims databases over the last two years.

That's helpful. And can you remind us to give a sense of approximately what the margin profile of that asset is? Because it doesn't have the same loyalty burden as the other.

Yeah, I think that, again, you know, normally these kinds of molecules end up having 70, 80 percent margin just because, you know, they're not that expensive to launch. And I think that that is the profile after we have done a ton of investment and work. So I want to be very clear about it. It's not on the first day we've invested throughout this period. But, yeah, that's kind of the number which I would say there.

Great. That's helpful. We're winding down to the end of our time here.

Thank you so much. looking forward to seeing more data and a couple more launches from you guys soon yeah no thank you money for hosting us and we're excited about the opportunity ahead of us to serve patients and and i think that's going to translate to significant shareholder returns and beyond that i know in our pipeline i just want to flag we also own an equity stake in gondola bio which is an extremely exciting uh early stage r d engine um which you know over time we can think about integrating again but that's a discussion for another day assuming our cost of capital improves thank you thank you