Oh, well, thank you so much, everybody. Thank you for joining us, Thomas and Julie from BridgeBio. It is really nice to have you here. We will kick it off first with you guys presenting some slides and giving an overview before we start with the Q&A.
Great. Thank you so much for having us, Jeffrey, and thanks to the whole Bernstein team. Thomas Trimarchi, President and CFO of BridgeBio, joined by Julie Miller Everett, our Chief Operating Officer of our Skeletal Dysplasias Business . Many of you will know Julie from her critical role in the Attruby launch, and we have just moved her over to help with the infigratinib launch, where she brings more than a decade of commercial experience, particularly in the ultra-rare and skeletal dysplasia space. So really privileged to have her with me here today. Maybe I will start just by saying a few words and setting some context for hopefully a great dialogue here with you today, Jeffrey.
BridgeBio is a company that at the highest level, we are here in this world to help as many patients living with genetic disease as we possibly can in the shortest amount of time that we can get that done. We do that through creating first or best-in-class medicines, and putting them out into the marketplace.
Today, the business late-stage portfolio is incredibly unique, I would say. If you have studied the industry, you are certainly looking at it today as I know you are. First, we have Attruby, which is our best-in-class, near complete stabilizer for ATTR cardiomyopathy. We are about six quarters into that launch. The importance of that launch to our business cannot be overstated. So at once, I think that has proven us as a capable commercial organization, and, I think that is widely regarded as one of the better biotech launches we have seen in recent years. Annualizing north of $1 billion today globally, on track to be on a full year basis, blockbuster status globally by the end of this year. Really, that is owed to a few things. First, this is a large and expanding marketplace. We are seeing something like 15,000, 16,000 patients come to the bottom of the funnel every year.
Really attractive from a patient needs standpoint. I think we have clearly a differentiated and best-in-class stabilizer, which are post some of the recent data sets, I would say solidified as the backbone of first-line therapy. Really exciting molecule there. Actually, this morning, we had some incredibly important news come out. We published the first of its kind real-world data set comparing Attruby directly to tafamidis-
That is great.
where we have seen about a third improvement in the composite of including all-cause mortality and CV hospitalization. Really important data set that has not really yet been disseminated-
Yeah
into the marketplace and impacted prescribing behaviors. That will take place over the coming year. So really proves us out as a competent commercial organization and also I think gives us the confidence to launch-
Yeah
The next three products, not only in the U.S. but globally. So, I think the truly unique thing about BridgeBio today is that still early days of one product launch and another three coming in the next year. So we've got Limb-Girdle Muscular Dystrophy Type 2I/R9, which is first in class molecule for a high unmet need form of muscular dystrophy. There we've got a product that can actually remarkably improve function on several fronts. One, patients are able to breathe better than when they started therapy. They're able to walk better, and they're able to actually, through measure of general symptom scores like the North Star Ambulatory Assessment, restore overall function and in a statistically significant way against placebo. So really exciting molecule there that's going to do a lot of good for patients. This is a clear billion-dollar opportunity.
ADH1 in encaleret is another small molecule that's read out phase III in the last year or so. Again, basically functionally cured about 76% of the patients in our phase III. So, really important data set that's going to change a lot of lives in this marketplace. And there again, we've got a billion-dollar opportunity, no competition, looking forward to building that market. Finally, achondroplasia, which I hope we have plenty of time to speak about given we've got Julie with us. This is today a billion-dollar marketplace where we're going to come in with a clearly distinct, differentiated, and unique molecule.
First and only oral in the space and a pivotal data set that really is giving the market exactly what it's been asking for many years, which is really a profound effect on height, but more importantly, a compelling effect on the health of these children, whether it's proportionality, or some of the things we've been able to demonstrate recently, including an effect on sleep apnea and ear infection. So overall, big picture, acoramidis we see is on track to deliver $4 billion-plus in peak year sales, and then you've got another $4 billion, I would say, de-risked and about to see uptake into the marketplace next year, with expansion available
Yeah
to us beyond that. There are some interesting opportunities, including hypochondroplasia and other skeletal dysplasias, as well as post-surgical hypoparathyroidism. That is the opportunity in the next 18 months, I would say.
Beyond that, our hope is that we can create many new meaningful medicines for patients beyond the four that I talked about and have a durable business that can grow for many decades. Beyond the four products that hopefully we will spend most of the time on today, we have had a very prolific, productive discovery engine that has
Yeah
continued to put molecules in the clinic. I think when the time is right, we can access some of that pipeline growth. It is housed today in our sister company called GondolaBio, which is private. With that long-winded, perhaps, context setting, maybe we can get right into the Q&A.
It was a great context setting, and yes, we will definitely touch on a lot of these drugs as we are doing our conversation and excited to hear more. Maybe even taking just a step back, just as an introduction, I would love to hear what both of you think is the DNA of the company, what makes BridgeBio tick, and why are you so successful?
Thank you for saying that. I would say two things in my mind are different and special about this company. First, this comes back to the patient impact mission, we really try and work on diseases where there is both a tremendous unmet need out in the community, either there is no available therapy today or existing therapies are inadequate. We try to work on diseases where we can ask, how do we drive a large magnitude of impact for patients? For us, that usually brings us to conditions where we can take a complicated biological problem and convert it to something much more like an engineering issue, where we can kind of connect all the dots between genetics and what we need to do to the target and how we affect it to translate to symptoms and outcomes for patients.
When you do that, I think you can, A, drive probability of technical success even beyond the all-comer kind of genetic disease levels, and I think we have demonstrated for the first 10 years that we can do that. B, it lets you really have a clear thesis on what your therapeutic hypothesis is, so that when it is not working or it is no longer intact, you can kill it and reallocate resources elsewhere. So that is, I think, one thing in terms of the operating system that is unique and has allowed us to be successful. Two, from a business standpoint, I think we catch a little bit of flak from time to time from our investors for spending too much time talking about NPV, but it actually does matter if you want to create a business that is going to be durable for generations to come.
You really have to be focused on, am I investing in a way that is going to allow me to be sustainable from an economic standpoint? I think, clearly, if you look at the opportunity for us to transition our P&L over the next 18 months, I think that clearly has benefited us to date. So those are two things that pop into the top of my mind, but Julie.
Well said. I would just add one more, which is that we have truly differentiated clinical science and outcomes.
Yeah.
When you look at the two pillars that we're building as an organization right now for commercial execution, we have hyper-competitive markets like amyloidosis and achondroplasia, where we have best-in-class products that are truly differentiated if you follow the science and the literature.
Yeah.
Then we have Pillar II, which is these traditional rare disease markets where we're building a market based on clinically defined conditions, well-articulated science, high unmet need. I think that's really what differentiates us, is having a scientific but also commercial organization that's able to develop drugs and then execute across both pillars.
That's excellent. Maybe kicking it off, as you gave a really nice overview that highlighted some of your four current key drugs in the near term and on market now. Love to maybe just start with Attruby.
Sure.
Give us an overview, any additional information you want to share beyond your nice introduction up front and how important it is for patients and for the company.
Yeah, absolutely. Attruby, as I mentioned, that's annualizing today at north of a billion, just six quarters into launch. It's a really interesting moment for this product where we see a large and continuously expanding market. As I mentioned, 15,000, 16,000 new patients a year right now today is our estimate. Just two years ago, that was more like 8,000. Last year it was something like 10,000 to 12,000 was our estimate. We continue to see strong momentum for the market overall. At the same time, we continue to grow our share of the market. I think we're really entering, I would say, a second chapter of the Attruby launch.
The first 6 quarters were really about getting out there, getting into the centers of excellence, establishing ourselves as the new player, the differentiated player, and really grabbing market share with our initial marketing message that were really anchored around the primary analysis from our phase III study, so 342/50. Three-month separation, 42% reduction in hospitalizations and mortality, and a 50% reduction in hospitalizations, which by the way, given a shifting patient population, most events that occur in this population today are hospitalizations, so really important that we have strong data there. I think that has gotten us where we are today, which is about 25% share of new patient starts and growing.
Really the next leg up of growth is going to come from a bunch of new data sets that we've just put out into the market or have been shared with the marketplace through competitive updates. On our end, we've seen two really important new pieces of data come out very recently. First, we've published an effect that shows Attruby actually can have a kidney protective effect in these patients, right? Actually the mechanism is something we're still working on, and I think we're going to have dedicated studies to actually demonstrate this in a prospective way. I think that could explain why we see separation on outcomes as early as one month or within weeks of treatment. That's really important.
I think that is just starting to be disseminated into the marketplace, and we could see an effect on prescribing behaviors there in the year to come. Second, as I mentioned this morning, real-world data set. One of the big questions we've had from prescribers that are more on the fence in terms of preference for Attruby versus anything else has been, "Okay, I can see scientifically that you've got a much more potent molecule, but it's very difficult for me to compare across clinical studies.
Yeah.
They were run in different eras. The population's been shifting rapidly. How do I really know that it's going to deliver a differential outcome for patients? Actually, sometimes they ask the patient which medicine they want to be on, which is not great, if you ask me. I think the real-world data set we've published this morning is going to be very impactful in answering that question of, does more stabilization truly matter in terms of outcomes? So, again, what we've seen there is a 34% improvement on the composite of diuretic intensification, CV hospitalizations, and all-cause mortality for acoramidis as compared to tafamidis. Again, that effect is seen in weeks, not months or years. So hopefully, we can create some urgency to start using acoramidis in new patients or even switch more of your tafamidis patients onto acoramidis.
There's going to be more to come, right? I think there is When you see a study coming from a sponsor, there's sometimes appropriate skepticism as to the methodology there. But there's an independent real-world data set coming out in, what is it? Two weeks or three weeks at HFSA, which hopefully can provide additional validation of what we've published this morning in our study. So, I see chapter two of this launch being driven by some of these newer data sets and leading to really a re-acceleration of growth as we look into the second half of next year.
That's great. More data, especially real-world data sets, are very helpful, so it's great to see. Thomas, speaking of recent data, a bit of a different mechanism, but CARDIO-TTRansform was presented at ESC last month. Just curious what you think of that data set and how it could be impacting the stabilizer class. I realize it's a silencer, but it was a very impactful presentation last month at ESC.
Yeah. Julie, feel free to chime in here, but I think the, a little feedback here, sorry. I think the most important takeaway from that data set was this whole concept of combining a stabilizer and a knockdown is really without any merit. We have seen no treatment effect of adding a knockdown on top of even a weak stabilizer. For the prescribers that were thinking that might be a good idea scientifically and could lead to a good outcome for patients, I think we have just too much evidence today to continue with that. You saw from multiple major KOLs at the podium say this is practice-changing, meaning that is no longer an approach that we should be thinking about for our patients. That really suggests that frontline backbone of standard of care ought to be a stabilizer.
As we just discussed, all of the data sets coming out here continue to suggest that acoramidis ought to be your treatment of choice and where you start. It is going to take some time. It is going to take some effort. It will not happen without a lot of fighting and repetition and educating the marketplace, but we are optimistic that the data is all on our side, and we can continue growing share over the next year and beyond.
Yeah.
Yeah, well said. The only thing I would add to that is, I think one of the other things it clarified is this concept of how do you define efficacy in cardiac amyloidosis? Mechanistically speaking, we discuss a lot this concept of the TTR tetramer, and ultimately what is disease-causing is the toxic monomer. When CARDIO-TTRansform read out, a lot of folks started digging into what is what we call mean max efficacy. When you look at the stabilizers, you have tafamidis. It is about a 60% stabilizer, meaning 40% monomer still forming disease. You have acoramidis, that is a greater than 90% stabilizer, so less than 10% toxic monomer disease forming. Then when you looked at the knockdowns, it is the inverse, right? It is like how much do you knock down and then toxic monomer remaining.
We saw that with eplontersen as well as AMVUTTRA, they were in the 60s to 80s, right? Scientifically speaking, I think a lot of dots were connected on an outcomes basis that prior to CARDIO-TTRansform were more of a theoretical basis.
Wow.
It is nice for us now to see that narrative evolve. Final point is that even with tafamidis, more as that background therapy in CARDIO-TTRansform and still seeing no benefit. Now doctors are also, to Thomas's point, taking into account what we have seen with the stabilization data, acoramidis being a more complete stabilizer. You now see the real-world evidence. You see the kidney protection data, and it just takes it to the next level that even with tafamidis, the trial was not successful. With acoramidis as the backbone of care, I think it is even further differentiated from the knockdowns.
I would just say two more things that struck me as-
We like this topic.
Really important from ESC. One, you have continued to see event rates go down with the shifting population, better care. That is really important because I think we can say almost definitively now a head-to-head study is not possible. So that puts increased importance on real-world data sets like we just reported this morning in terms of providing evidence for treatment decisions. Two, really, I think related, it means that most of the events happening today are hospitalizations.
Yeah.
It makes it that much more important to have really, really compelling hospitalization data around your molecule, and we have market-leading data there as well. 50% reduction in hospitalizations in just 30 months. I think we are positioned extremely well to continue growing this brand over the next years to come. It is not going to happen overnight.
Yeah.
But with I think the right team, the right resourcing, and a little bit of luck, I think this can be a massive product for us and a huge impact for patients.
Absolutely. Well, just asking a sales question, how should we be thinking about the quarter by quarter dynamics for Attruby sales?
Yeah. I think for the next several quarters, I would say till we get into really the end of 2027, we would be thinking about, kind of a $25 million to $30 million quarter on quarter absolute delta. That is driven by a few things really. First would be the dynamic around second line, the tafamidis switch. We saw a pretty significant kind of bolus pool of patients early in our launch that were coming off of tafamidis onto new products, ours and Wainua, the knockdown. Which makes sense, right? This was a market where there was one player in town for a long time, that was tafamidis. It was a huge step up in terms of care for patients, but clearly inadequate, right, in terms of the level of efficacy and stabilization.
We knew there was a big pool of patients that were progressing and worsening, but had nothing to switch to, had just been hanging on to tafamidis. We saw early in our launch, I would say, a larger than expected contribution from tafamidis switchers. Certainly, what we can ascertain from dynamics around the knockdowns is they were benefiting significantly from switch early on in the launch too. I think at this point we've kind of worked through that initial pool and that's normalized. Second line is growing at, I would say, steady state now, which is lower than it was a year ago. That's one thing that's, I think, influencing the near term quarter-on-quarter dynamics. Two is related, which is duration related to patient mix.
As I said, early in the launch, contribution from second line was bigger than we would've expected, and that has shifted towards a greater and greater frontline contribution over every quarter since launch to the point where today new starts volume vast majority is treatment naive frontline share. But we're dealing with duration from the cohort that came on drug six quarters ago. That's another thing that I think you should be mindful of as you think about quarter-on-quarter dynamics near term. Then I think, come late 2027, all of these new data sets that we've been talking about should begin to get reflected in treatment behavior and share, and we'll start to see a re-acceleration of sales growth at that point.
That's great. You sort of highlighted the Attruby launch, great data that's just being released just now, your real world studies, and now maybe looking even beyond that to life cycle management. Is there anything you would like to highlight or you're thinking through when you want to speak to what the potential life cycle management of the drug could be?
I'll start. Julie, chime in. I think there's at least three important studies that we're thinking about right now. One is the ACT-EARLY study , which is the first of its kind. This is a primary prevention study in variant patients where based on high risk of progression from being asymptomatic to having heart failure based on family history and various other factors. You were randomizing patients to placebo or Attruby, and the goal there would be to prevent patients from progressing to heart failure. This is analogous to what we've seen in the statin class with some of those seminal studies there. Practice changing, basically, disease prevention is always the goal if you can figure out a way to do it.
That's key, and I think that also has the effect today of helping drive the narrative around early diagnosis, early intervention, even for patients that do show up to the clinic with heart failure, don't wait.
Yeah.
ACT-EARLY. Two, we've just kicked off a cardiac imaging study. The goal there is going to be basically to elaborate and validate a signal that we saw in our phase III study in a substudy that we had with cardiac imaging. We want to see actual cardiac MRI-based disease reversal. We think it's possible based on the signal we've seen and based on the potency of Attruby. But I think if we can show that in a prospective study, dedicated study, that again would have the opportunity to be practice changing.
Third is we talked about we're looking for ways to elaborate on and again validate this renal protective effect that we've seen from our phase III in a dedicated prospective study that really just allows physicians to understand this effect and understand that it is distinct and unique from other approaches that are out there today, and really can explain some of the compelling data we've seen on the early separation of outcomes, whether compared to placebo or again in a real world setting compared to tafamidis where you see separation within weeks.
Well before moving on to the next drug, any last comments on Attruby you feel like we haven't had a chance to discuss yet? Anything else?
No, I would just say based on new evidence that's emerging across the category, including real world evidence, seamless execution at the commercial level, differentiated outcomes in terms of science and commercial delivery, we feel more confident than ever in our ability to execute on $4 billion peak or more.
I would say one last life cycle thing that I didn't mention is we should have a once daily form coming-
Yeah
into the clinic in the next year, 18 months. Which I think there's a minority of prescribers and patients that are choosing tafamidis due to once daily form. But we want to be able to provide a competitive offering on that front. It has the, I would say, additional business benefit of giving us a whole new 20-year patent that should protect exclusivity for many, many years to come.
Well, great. Then, you highlighted three other great drugs that are in the process of making their way to patients. Maybe start with the first one, BBP-418.
Yep.
Maybe give us an overview of that drug and maybe dive a little deeper beyond what you gave in your intro.
Yeah. So BBP-418 will be the first-ever therapy for a disease called Limb-Girdle Muscular Dystrophy Type 2I/R9 . This is, I would say, most phenotypically similar to Becker Muscular Dystrophy, where really bad disease where patients experience loss of ambulation, loss of physical function. Around a third experience loss of pulmonary function, cardiac issues. So really bad progressive disease that is like Chinese water torture is what our patients tell us it's like, where every day there's something you can do less well than you could do the day before, and you're just constantly every day losing function. So really terrible condition. Nothing available today. Significant unmet need in terms of prevalence. There's around 2,000, 2,500 patients we believe in the U.S. today that have this condition, and another 5,000 or so in Europe, and another 2,000 in Japan with a related condition called Fukuyama Muscular Dystrophy.
BBP-418 is basically targeting the disease directly at its source. So basically, the genetics of 2I are basically loss of function, partial loss of function, an enzyme called FKRP, which is glycosylating or adding a sugar moiety to a structural protein called alpha-dystroglycan. If you're not adding the sugar, it doesn't function properly. If you destroy FKRP, not getting that sugar, not getting proper muscle function, and then you get all the symptoms and phenotypes that we see in these patients. So what our drug does is basically provides high levels of a key substrate upstream of FKRP and drives additional flux through the residual enzyme activity that these patients do have, to restore that aDG glycosylation. So, what we saw in our phase III was basically our initial approach was to go for a surrogate based, biopsy based biomarker kind of approval, accelerated approval.
And we developed this very elegant biomarker assay that actually is able to measure the amount of glycosylation that you're putting on alpha-DG. And we had a phase III that read out one year endpoint, and we thought we were going to be able to see an improvement in that biopsy based biomarker and file with the FDA and try and convince them that this was reasonably likely to predict a future clinical benefit. What we were frankly blown away to see at 12 months was a profound and consistent impact on every single endpoint we measured in the study. What was to be the confirmatory functional endpoint, which is the North Star composite, that we saw a stat sig benefit, an improvement from baseline FVC. We saw a stat sig benefit improvement from baseline. 10-meter walk test, we saw a stat sig benefit improvement from baseline.
Basically, everywhere you look, the patients are getting better, and we've seen a highly stat sig benefit as compared to placebo. Really compelling data set. I think really unique even broadly amongst neuromuscular clinical development where you just don't see that many well-controlled, well-designed, placebo-controlled trials deliver this kind of benefit. We're really excited to get this out into the marketplace and to hopefully change a lot of lives.
That's great. Assuming positive outcome with the FDA, what is your view on the potential for the drug's launch and its value proposition?
Yeah. Just with the prevalence numbers I just walked you through, this is a clear billion-dollar-plus opportunity, in the U.S. alone even. I think there are analogs in similar markets with much weaker datasets to suggest price point could be very compelling here. You think about the exon skippers for DMD around the $700,000 price point. I think we're going to have a lot of flexibility to price well. With the FDA, yeah, it's been a very constructive dialogue, so expect we'll get approved on or before our November 27, 2026 PDUFA date. The question there really is, will we get full or accelerated approval? I think, hopeful that we could get a full approval here. Then I think around uptake, so where do we stand today in terms of market building, market development?
Today we have around 500 of the 2,000, 2,500 patients that we think exist in the U.S. identified, confirmed with the correct genotype, and basically mapped to a prescriber. So that's our initial pool of patients that we're going to be targeting early days of launch. The way to think about the cadence there is we ought to be able to work through that initial pool of patients within the first three years of launch. So maybe 100 year one, another 200 year two, and then perhaps 300 year three. At the same time, we'll be growing that pool through all of our marketing efforts out there in the field with our medical affairs and sales force to drive awareness, genotyping, referrals into the centers of excellence where genotyping is standard of care, and really to drive hopefully broad uptake over the several years following launch.
That's great. Well, before we switch over to BBP-305, any last comments, questions, comments on BBP-418 ?
No, exciting product. I think first of its kind in the muscle dystrophy space, so really excited to get out there and make big patient impact.
Perfect. Well then switching gears to BBP-305, maybe you could once again give us a nice overview where it stands.
Yeah
and outlook.
Yeah. This is encaleret, which is a calcium-sensing receptor. First indication is a form of genetic hypoparathyroidism, where the disease is caused by an activating mutation in the calcium-sensing receptor. As we typically like to do, there's a genetic defect, and we like to hit it directly with a small molecule. This is actually the first indication of a broad set of indications we'll be going after with this molecule, including postsurgical or hypoparathyroidism broadly. But for ADH1, this is, again, a high unmet need where patients. Actually, there's a case report that just came out of England, I think, following a family where two of the children in the family actually died in their 30s from some of the terrible symptoms, heart failure, and just general tetany and seizures. It is a very serious condition.
If you talk to specialists that treat this, that treat hypoparathyroidism, their ADH1 patients are usually their most difficult to manage, where they see very low serum calcium driving all the symptoms. They try to manage it with supplements, which just makes the elevated urinary calcium that much worse, and it's basically creating a ticking timebomb down the path of renal destruction. The need here was for a therapy that could basically fix the aberrant signaling in the calcium-sensing receptor and hopefully alleviate the low blood calcium and the high urinary calcium. What we saw in phase III was, in six months of therapy, we were able to completely withdraw conventional therapy, which is supplements and calcium supplements and vitamin D, and put more than 75% of our patient population into the normal range on both of those aspects, urine and blood calcium. Incredibly compelling.
That's a functional cure for more than three-quarters of our study population. More than 90% had parathyroid hormone into the normal range almost instantly. Really profound effect in this population, which is incredibly exciting for this population that desperately needs care. Excited to get out there in the marketplace. Again, this is similar to Limb-Girdle 2I. This is a condition where there's nothing really available. There's no sponsor out there marketing, driving awareness, so that's on us to do.
We're out there today with field medical and sales leadership working on driving urgency to test. Here, it's a dominant condition, meaning families that have this condition have about 50% chance of passing it on. Family testing has actually been incredibly helpful here. We actually have a mobile testing lab van that we bring to advocacy and family events, and actually it's been surprisingly productive to date. We'll keep driving that along with more conventional, I would say, market development tactics. That product has a May 8 PDUFA date, so under FDA review. I think the overlooked aspect of encaleret is actually the expansion opportunity into a broader suite of hypoparathyroidism conditions, including the biggest bucket, which is postsurgical hypoparathyroidism, where we've just kicked off a phase III pivotal study. Those are first patient there in the next couple of weeks.
That could add, I would say, a couple billion in opportunity to this product. We will enroll this study next year and hopefully read it out late next year to early 2028. So large expansion opportunity on the horizon there.
That is great. Last but not least, the last drug you highlighted earlier, infigratinib. I would love to hear your overview there, what you are thinking in terms of long-term perspective of that drug.
Yeah, absolutely. Similar to what you heard Thomas say about our other molecules, infigratinib also directly targets the mechanism of this condition at its source, the genetic mutation for FGFR3, whereas some of the other products in the market today are more indirect treatment mechanisms. Scientifically speaking, we feel very good about mechanism. As many of you know about the achondroplasia space, there are two treatments available today, CNP analog products that have really shaped the market and provided an option where nothing was available today. What we feel very encouraged about, starting with the U.S., when you look at the market, severely under-penetrated. It is only about 20% to 30% of families have opted into treatment today, and that is due to some very, very good reasons.
The first is that the only currently available products are injections, whether that be a daily injection or a weekly injection, very, very difficult and limiting for families. Second is that this community has been very vocal that they want to see benefits beyond height. We want to see benefits on complications, functionality, comorbidities of the condition. Infigratinib is the first product to show statistical significant benefit within 52 weeks on benefits beyond height, so specifically proportionality. Not only did we demonstrate that largest annualized height velocity across the category, but it is these benefits beyond height that are really meaningful for physicians and the community. Just two weeks ago at ESPE, we also released new data. Previously, we had shared in the primary data readout as well as our New England Journal manuscript, the height velocity and the proportionality data.
We just also included data on otitis media, which ear infections, as well as sleep apnea. So two common and difficult complications of this condition. Also within 52 weeks, infigratinib demonstrated benefit on both of those outcomes, the only product to do so. As we are preparing for launch here in the U.S., there's really three distinct categories for source of business. The first is that large portion of the market that has not opted for treatment today that we believe an oral therapy that demonstrates benefit beyond height can unlock. The second source of business is those who are on an injectable therapy but might be seeking more in terms of convenience and efficacy that's being left on the table. And the third category of source of business is those who were previously treated and have discontinued. Our evidence points resonate well across all three categories.
Go-to-market in the U.S., we have our medical team sized and deployed, facilitating scientific exchange today. We're building out our commercial infrastructure to really call on where we know patients are being managed and families are seeking care today, as well as where some families who are not seeking care today are interacting with mostly their primary care. So we have a team that's ready to educate and inform the community so that they are ready to make an informed choice about therapy. Ex-U.S., it's a very different environment, so ex-U.S. treatment penetration is much higher. It's only about 20% to 30% in the U.S. Outside of the U.S., it's more 70% to 80%. So we've created a global international commercial organization that has developed a bespoke go-to-market model based on country.
We've looked at all the primary markets across Europe, Latin America, Asia Pacific, and we've been able to design a commercial infrastructure ranging on one end of the spectrum from a direct presence, the other end of the spectrum to more the distributor or partner market strategy. Each unique market, we've been able to design our commercial infrastructure to maximize rate of speed, uptake, as well as price potential. It's all about demonstrating value in these markets, and we feel we have the clinical evidence to do so, as well as the medical team and the commercial team to support the launch.
That's excellent, and a really nice overview of the key programs in your company. In the last minute here, any last things you'd like to share about BridgeBio before we wrap it up?
Yeah, no, I think 2027 really is going to be a transformative year for the company, where we're going to go from one product, R&D excellence in the last year to four products launched globally. I think when the time is right, there's a huge amount of pipeline we could bring back in to develop a very unique and generational integrated biopharmaceutical company that can do a lot of good in this world. So, I think it's a tremendously exciting 18 months to come and hopefully beyond that as well for many, many years.
Excellent. Well, thank you both for your time today. It was great to discuss the company together.
Thank you.
Thank you.