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

Welcome, everyone. I'm Josh Schimmer from the Cantor Biotech Equity Research team. I'm very pleased to introduce from BridgeBio. We have Chinmay Shukla immediately to my right, SVP of Investor Relations and Strategic Finance. Next to Chinmay, we have Anna Wade, Chief Operating Officer of BridgeBio Neuromuscular and beside Anna, we have Julie Miller Everett, not Everett Miller, we got that, Chief Operating Officer of Bridge Bio Skeletal Dysplasias. So great to have the team with us. Chin, maybe you want to kick things off, give us a quick snapshot of where BridgeBio is today? And where the company is headed.

Yes. Happy to do that. First, Josh, thank you for hosting us, and thank you to all the investors for joining us here and as well as on our one-on-ones. So BridgeBio today is at an inflection point, we have $8 billion in post Phase III care sales assets. The first of that is at, which is launch, which we think is a $4 billion drug Clearly, it was already annualizing to over $1 billion in 2Q. So we're building on our strength there. Right after that, we have 3 launches upcoming very shortly now. I think the first one, Limb-girdle is going to be in November. And then we expect to launch in achondroplasia as well as ADH1 sort of early next year. So that's where we are at. All those 4 indications put together are $8 billion in peak cells. What's very exciting is that right behind that, we have an expansion indication in hypoparathyroidism which is probably worth another couple of billion dollars, especially when you put it with the deplete antibody, which is in early stage in ATTR-CM. And then that's not it. I know, Josh, you've always liked for us to think more holistically and think more broadly about genetic diseases and helping patients who are suffering with these diseases. So we have a sister company, Gondola Bio, where they have a late-stage EPP program as well as 16 or 17 very early state assets, which are all very exciting. So there is a lot happening. We're trying to do a lot for patients. And my hope is that over time, that value is going to accrue to shareholders also.

All right. Well, there's a lot to get through, so we'll see how much we can get through in the next 27 or so minutes. Why don't we start with the Attruby remarkable drug, remarkable progress and yet still seems like a long road ahead to achieve what that drug should achieve. Why don't we begin with kind of some of the takeaways after the cardio transform data presentation and how you think that these incremental pieces of information help position Attruby in the competitive TTR landscape.

Yes. Happy to do that. I think that cardio transform was, I would say, the last big data event, which has now really clarified a lot of the picture for us ATTR-CM. So where are we today? I think the first thing to realize is that stabilizer therapies. There are 2 main types of therapies, stabilizers lockdowns, stabilizer therapy, which is the type of therapy that Trub is they are going to be the treatment of choice for frontline ATTR-CM patients. If a new patient comes in, which is a majority -- vast majority of patients today, they are going to be put on a stabilizer therapy. The knockdown therapies take about 1.5 years to work, whereas a Attruby takes about a month to work. And then you also have to take into account that combination therapy, which some people had hopes for was proven decisively to be inefficacious in cardio transform. I think the second thing which we learned is that the 2 knockdown agents are more similar to other than different if you look at the placebo-adjusted knockdowns and the Gilmore all paper, you saw that the 2 knockdown percentage is adjusted for placebo were very similar. And then that translated to monotherapy hazard ratios for the 2 knockdown agents to also be about 0.67 and 0.7, respectively. So very, very similar. And then the third thing, which I would say is we were very encouraged to see that clearly, there is room to do more beyond Windex which is the first-generation old stabilizer, which is where -- or Attruby, which is our second-generation new stabilizer has a unique differentiated edge. And so we do think that having that near complete stabilizing and a true, it confers a very unique kidney protective effect. We also see the fact that it has much stronger effects on hospitalization as well as time to separation, hard outcomes separating as early as one month. So we think that, that is where Attruby is positioned. Today, we have more than 25% share in frontline patients. We have said our goal is to be a 30% to 40% peak. We're doing market research to refine that. And my expectation is that, that 30% number will probably move up on the bottom end range at least. And then the last thing, which I would say is in our experience, it takes a little bit of time for all this data to really get out there in the field. We've noticed that it takes physicians model visits explaining the data carefully these and data sets. And as we do all of that, I think it takes about a year or so for these amazing data sets for Trub to translate into script growth and then ultimately, revenue growth. So near term, we expect about a $25 million to $30 million quarter-over-quarter sales increase for the next 3 or 4 quarters, which should then start to accelerate as you get into, call it, late '27 early.

Okay. And given the claims data analysis you've been able to generate, it seems to be really differentiating versus to females and resonating I'm surprised your goal is that 30% to 40% range not 2x that or more.

Yes. So that's a great question. So we think that Attruby is the best drug for ATTR-CM patients. And we do think that everyone should be on Attruby. We actually in Europe, more than 50% of new patients are on Attruby, especially in Germany, which is where the drug was first launched in the auto by our partner, Bayer. So why is it that I'm saying that our goal in America is 30% to 40%? Well, I think there are a few reasons for that. The first reason is I don't think most investors believe 30% to 40%. So let's start there, so we can sort of walk them through it. over time. Second of all, I think that there are some unique differences in our health care systems such as the buy and bill economics and things like that to impact prescribing on the edges in a world, it wouldn't. And I know the government is thinking about that, but it does right now. And so we have to be realistic about that. But what I would say is, we have built a very strong foundation of evidence now that Attruby is a differentiated stabilizer. It's not just another random, it's a much better stabilizer. And that started with the label. As you remember very well, Josh, we're the only agent in the field, which has the webinar complete on our label. Beyond that, what I would say is we've then built upon that to say that, that near complete stabilization is correlated with a decrease in all-cause mortality, then we've built upon that with crop analysis, whether it's the AFib patients or the variant patients. And yes, now we are expanding upon that with a unique kidney protective effect, which Attruby has as well as the same data, which you are saying, although the claims data is maturing. So my expectation is we'll see more independent studies come out at HSA we are always excited when an independent person looks at a Trub versus Vendorax because we feel so good about our profile.

What should we be looking for at HFSA in terms of additional claims analysis that could either bolster the case for Attruby or not?

Yes. So if you look thus far, right, we've done analysis from our own Phase III trials, whether that's looking at a Trub versus Vendomax in the trial, and we've also looked at subgroup analysis. I think some people have also done some comparisons of Trub versus Vyndamax on serum TTR in real-world settings. I think what the field is looking for is clinical worsening outcomes. So what do I mean by that? Things like diuretic intensification, hospitalizations. Those are hard outcomes. It takes a little bit of time for this data to mature, but I think we're almost 2 years into our launch. So I know that there are some systems between looking into this. And my expectation is that they will publish what they -- people publish what they found on a Attruby versus Windomax. And really, that clinical worsening outcome should drive additional behavior with cardiologists, though I will caveat the fact that it takes 6 to 9 months for all of that data to get in the field and then start to get reflected just because we have to go and educate people on it.

And what's your confidence then when it's all said and done with all the claims data analysis at the time that generic TAF enters that you have built such a strong case for Attruby's differentiation that generic TAF is not going to be a problem.

So well, first, I'll answer that by saying our confidence is extremely high. That's the simple answer. And let me build upon that. I think that we already have built a solid foundation of evidence. I think that the earliest that avenovaxgenatic could enter is mid-2031. So we have another 4 or 5 years before that, and I've been saying it takes about 9 months, maybe 12 months to have all of these -- to have this material educated to the physician. So we do significant time -- the same thing, which I would say, which is a little under appreciated by investors is if you look at the channel dynamics here, it's really a channel which is dominated by specialty pharmacies and institutional specialty pharmacies, and the stakeholder dynamics there are such that branded drugs are preferred more. And then the last thing, which I would say we've updated our corporate deck on of this material. If you look at any single -- any pharmaceutical category when a first-to-market esport molecule goes generic, the second more potent molecule, the sales keep going up. And I think the best example of that, which, interestingly, investors have inbounded in to us and told us to talk about it is Gleevec as well as the next generation per which came in at biogeneric.

Yes. Okay. Excellent. And why don't we come to you? CEO of BridgeBio neuromuscular, that's going to be BBP-418. Is there any other programs within neuromuscular at this point? Or is that yet to come?

That's yet to come, but it's definitely something we intend to continue to evaluate and Jim might speak to some of the programs within assist company Gondola later which you got some in.

Okay. Got it. So for 418, I expect you've had your mid-cycle review now with the FDA. How are you feeling about approval prospects and/or need for ADCOM or other considerations?

Yes. So we're feeling really good about our approval prospects. So just to take a step back, our PDUFA is on November 27 of this year. In our data set that we announced last October, we saw clinical benefit on functional outcomes. So we basically saw improvements in pulmonary function as well as ambulatory measures. And that data as well as the full data set that the agency has seen it really is supportive of an approval in November, and they're also still considering traditional approval is the most appropriate path, which would not have any sort of further commitments to do a sort of confirmatory study.

Okay. So I think the estimate is around 4,000 patients in the U.S. total with LimbGirdle 2 or 9 with 500 identified. Is that correct? And maybe talk a little bit about the cadence of patient identication and what the addressable market you expect.

Yes, absolutely. So we estimate there are about actually 7,000 in the U.S. and Europe and within the U.S., it's about 2,000 to 2,500 of which we currently have identified around 500 patients. where we know where they are and they are difficultly confirmed. We are continuing to see growth there as we ingest more testing data, we have people out in the field, and they are sort of profiling accounts, and we continue to sort of add patients on a sort of monthly basis through that as well as we actually got a unique ICD-10 code that came in, in October of last year. So it's still not particularly mature, but we are seeing the current use of it, and it is growing on a month-by-month basis. well. So we're continuing to see sort of momentum there with the increase in patients we've identified today.

And do you expect all 500 patients identified or good candidates for winning.

Yes. So we do believe that the majority of patients are really good candidates for therapy, and that's driven by a few things. So first off, you look at the clinical trial inclusion criteria from both the Phase II and the Phase III, that it's very broad. So it includes both genotypes. It includes ambulatory and nonambulatory patients. a variety of different functions. I think the only sort of potential narrowing of that population is just based on the age. So basically, we were studying 12 and up in the Phase III trial. And also, there's a body weight limit basically based on our current dosing. So we dose 9 grams twice a day for 30-kilogram patients and then 12 grams twice a day 50-kilogram patients and above. So we wouldn't anticipate that there's lowest body weight, youngest individuals right now would be candidates for therapy, but pretty much everyone else should be. The HCP market research we've been doing has shown really strong adoption across all of those different patient segments that I mentioned.

Okay. Are there useful pricing benchmarks you have in mind?

The most useful pricing analogs that payers always referred to are the exon skippers and obvious, it's a very similar disease. It's sort of similar prevalence as well. And I think the main sort of thing to consider when you're looking at those analogs is that those analogs really were just based on biomarker data and didn't have functional outcomes. So when we have been doing payer research. The fact that we have these functional benefits that are seen in our data set means that we potentially could price at a premium to those.

How should we think about ex U.S. commercial plans, so that may actually be a good opportunity to talk about the MFN agreement that Bridge recently forged with the White House.

Yes. Maybe I'll give a few general comments and then I might ask Julie to talk about it a little bit more because obviously, Infigratis at the cutting edge of ex U.S. for Bridge right now. So our base case plan is to commercialize globally with the next 3 products, whether that's 418 and calorie or infigratinib. Really, that comes down to a few different things. One is we are in an MFN world right now, and we're very happy with our agreement with the government and partnering with the government, which I can touch on in a minute. But you never know if you look 10, 15 years out, this is now part of the biotech business, and so we have to think very long term and strategically about these transactions. I think the second thing is, these are going to be significant more focused launches ex U.S. also because the patients are congregated at a small number of sites of care. We already have presence in these countries through clinical trial sites as well as deep relationships with KOLs. And then I think about the fact that while these things are capitally efficient launches, we also now are in a better capital position. We had about $1.7 billion of cash on our balance sheet as of July 1 pro forma. So that makes me feel better about our ability to actually invest properly in these geographies and get the right value out of them. Look, having said that, if someone comes along and makes an offer which fits our better on the hypothesis, phone is always open and we're always happy to engage. But our base case plan, given all of these considerations is to commercialize ex U.S. on our own, we have made good progress already. We're being lean about staffing ex-U.S., but we've already made a good progress on the regulatory front and some senior international hires. Maybe I'll Julie comment on it for a minute.