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

Yes, I was going to say like in terms of like the doctors and the education process, like how long do you think that will take? And is it -- because this is an indication where the therapies, is this going to be where basically every can is -- or every patient is a candidate essentially? Or are there going to be some kind of narrowing of the actual true candidates that would actually be best to respond on the drug?

Yes. 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 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 3 months on the ambulatory measures, which is really driving urgency to treat. I think currently, what might there's a lot of, obviously, patient need that will be driven by very strong patient advocacy groups to 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 the 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 on 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 kind of the journey to diagnosis, like how long does that take? And what's kind of your method of even just finding these patients? Are they good 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 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 well. And what 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 [indiscernible] testing.

Got you. Now this is a different type of launch than I would say it would be like more rarities more focused. So I guess are there any key learnings that you can take from kind of the Attruby experience and kind of apply it to the BBP-418?

Yes. I think that there are 2 different architects of launches, right? There is a launch in the competitive market, which is Attruby and infigratinib, and then there's launch in a market which you are building, which is 418 and [indiscernible], right? I think that some of the similarities, 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. Sure, I'll let Anna comment on that because 1 thing which obviously Attruby we have white club 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 learning from Attruby.

Yes. So I mean, we're out there right now to talking to payers. Payer research has been incredibly positive as well, like they're very receptive to the data that we've seen. As we mentioned, this is like the notes to seeing placebo-controlled data in the neuromuscular space and then also seeing these improvements from baseline in the functional measures is pretty differentiating. So we're not anticipating sort of big coverage huddles for access reinvestment. We are planning on offering a similar suite of services to what was mentioned for Attruby for getting patients on drug at launch.

I guess it's a good pricing analog for [indiscernible].

Yes. So we -- the pay as always referred to the [indiscernible] is here is the nice relevant pricing analog. I think, as I mentioned, the main difference here is that we're seeing functional benefits in a polebo-controled setting, which they haven't really seen in that case. So I think you would think that about those as like an analog as sort of the anchor and then we might think about premium pricing on that.

And the 3 launches discussed the other 2, but do you think [indiscernible] has the potential to be the fastest ramping? Or do you think there's kind of like some, I don't know, friction points just because it's a new indication that'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 3 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 [indiscernible] ADH1 is true. But nothing specific that I would call out as a pain point. It's all things that are team has diligently -- 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. And so I think that it's -- they're all going to go really well. We feel good about them. And then I think your question was if this 1 will be faster than some of the other ones. And on that one, I'm going to ask you guys to do some work.

You don't want to pick your favorite child?

I am told that that's not my...

You can't do that on...

That's fair. All right. Well, maybe moving on -- so let's maybe talk about Encaleret in H1. 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.

Yes. 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 have only genetically confirmed and identified about 500 patients. But we know that in the claims data, there is already about 20 to 100 patients. So we know that there are more patients that we can go after and try and convert. So huge unmet need. The drug basically normalizes the patient, right? Because your [indiscernible] burden 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 [indiscernible] 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 hypoparathyroidism. Actually, if you have nonsurgical hypoparathyroidism, 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 where ADH1 patients onto our drug. In terms of, I think, how we expect the launch to go, very similar to [indiscernible], 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 their disease. So that's actually a pretty interesting kind of difference between these 2 diseases. And I definitely do think that pricing here is going to be less than [indiscernible], but still extremely strong. I think the comps that we've given on the lower end are [indiscernible], which is about $300,000, and then on the high end, Uwe, which is about $500,000. I do think it's possible we are closer to the high end than the low end given the drug is has significant benefits. And that's how we think about it. And of course, what's very exciting about Encaleret is -- and I know you probably going to want to talk about this is there is a huge expansion indication in hypoparathyroidism right behind it. We're very excited that we have kicked off a Phase III trial there. We expect that trial to read out pretty shortly here by the end of '27. [indiscernible] 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 a profound benefit, especially normalization of urine calcium, which is a key unmet need area. I think second -- and I'm going to be a bit quick here because I know we're running out of time, but we expand -- Neil actually expanded upon this in our -- on our earnings call. But mechanistically, there is a strong rationale for an agent acting on the cash-in sensing receptor in the kidney to have an effect on in each. 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 talking like [indiscernible] as well as other folks who really understand these endocrinology diseases. And I think that, that KOL excitement is the third thing, which has made us excited about starting a Phase III here and helping a lot of patients.

Got it. So yes, maybe let's move to infigratinib just so we can get through it. But this is 1 excellent data, Phase III data in achondroplasia first oral agent in that indication as well. And with the launch of UV, we're definitely learning that the market is getting larger. And there's patients that were not really being -- they didn't want to go on box. So I guess -- now a couple of months now into the UV well launch, where are you guys kind of thinking about -- or are you more excited about the infragratinib upcoming launch and ultimately the opportunity there for an oral agent?

We are extremely excited about the infagratinib 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 infigratinib was 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 3/4 or so of patients of kids are on some [indiscernible] option in America, maybe a 1/4 are. And really that difference is moving beyond height and having an oral option. And that, I think, is the opportunity for infigratinib, and I think that is why we are very excited to be able to -- 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 round assuming the FDA approves the drug.

Got you. I mean do you -- is this mostly like new start market? Do you think you'll switch patients? Like how do you think you'll get your foothold. And as you said, I think your data on body proportionality and that is going to be pretty meaningful for docs and for the patients. So what do you kind of envision early in the launch?

Yes. So we are going to be pulling from 3 segments of children. The first segment of families and children that we're going to be pulling from are children who are on [indiscernible] today. 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 [indiscernible]. 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 [indiscernible]. 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 [indiscernible] some of them are needle for [indiscernible], 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 3 segments. And I think that what we are most excited about is growing the market, so segments 2 and 3. But I do think segment 1 is also going to be important.

Got you. Maybe with the last [indiscernible] here, Chief Business Officer. Like as you think about the kind of overall business, Attruby, pipeline launches, like -- so we're going to have these launches is going to be 2027 event. That's going to be exciting. But what's kind of next, right? That's overall we is like what's kind of next in the pipeline? Where are you guys going to be kind of focusing your efforts and as you said, kind of where are you going to take your resources and deploy the capital?

Yes, that's a great question. And I'm not surprised that Wall Street is asking what's next. So here's how we think about it, right? I think our first priority is funding and accelerating the Attruby launch as well as the launches in [indiscernible] and ADH. 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 -- the main idea there is our hypopara trial. And so that I also think is on investors' radars and something that you'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 with events. So we have a sister company called GondolaBio, where we own about a 12%, 13% stake at the right time, maybe it's sooner, maybe it's later, but 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 4 medicines, but to make 40 medicines. And I think that, that's the shape of company with investors patients and the community can be very excited about, and that's what we are aiming to build.

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

Thank you.