BridgeBio Pharma, Inc. — Call Transcript 2026

Okay. Well, we're going to get started with our next session. I'm Andrew Tsai, Senior Biotech Analyst at Jefferies. Thanks for joining me today. It's my pleasure to have the BridgeBio team with me. To my direct left is Tom Trimarchi, CFO, and to his left, Ananth Sridhar, CEO of Endocrine Disorders. Welcome, both of you. Thanks for having us. It's great to be back in New York, sun's shining. Yes Knicks are in the finals, and we're at Jefferies. What more can you ask for? Great. Those in the audience less familiar with the BridgeBio story or revisiting it, would you mind just briefly giving us an introduction about it, where you are with all your programs? Congratulations on all the success, by the way, milestones over the next 6-12 months would be very helpful. Yeah, of course. BridgeBio Pharma is a company that's about 10 years old now. We were really built to deliver patient impact at scale, specifically focusing on patients with genetic diseases. In the first decade, I think we've been remarkably successful in pioneering a fairly new and innovative R&D model that's yielded the commercial and late-stage portfolio we have in front of us today, which includes Attruby, which is about a year into the launch, and it's really still ramping. We're annualizing about $720 million in the sales in the U.S., and also have seen strong uptake ex-U.S. with our partner Bayer. That's, I think, the first leg of the stool or chair. Right behind that, we've got three products that are now on the other side of really remarkable phase III data sets that we're focused on submitting to the FDA and global regulatory authorities and will be launching starting at the end of this year. That's one in limb-girdle muscular dystrophy Type 2I/R9 (BBP-418). We see this as a huge unmet need. It'll be first drug to that market, clear $1 billion TAM for us. Right behind that, we have encaleret for hypoparathyroidism, with our first indication being a genetic subtype called ADH1. Ananth is here with me, hopefully we can spend a good amount of time on that. Third will be infigratinib. That's a small molecule oral FGFR3 inhibitor for FGFR3-related skeletal dysplasias, starting with achondroplasia with an expansion into hypochondroplasia. That's a multibillion-dollar opportunity for us, clearly. Lot to be excited about. Yeah, I think the next 12 months are going to be focused on execution, where we can keep rolling on Attruby, keep seeing strong uptake, importantly in the first line treatment-naive setting. We'll be really putting our heads down and focusing on getting our next three drugs approved on time, hopefully with the right label, so that we can go and deliver these medicines to patients. Fantastic. Maybe starting with Attruby, it's doing very well. You're capturing, like you mentioned, a lot of first-line market share. I think it was above 25%. That's right. most recently. That's right. The question is, how are you exactly doing that? Because you aren't first to market per se, yet you're doing this. There's no head-to-head study been done against Pfizer's compounds. What's resonating with doctors and so forth? Yeah. It's a great question. I would say going back to the design of the molecule, we made Attruby specifically to be an ultra-potent kind of next gen TTR stabilizer. Remember, this is a disease where your TTR tetramer is unstable, falls apart, makes amyloid, goes to your heart, ultimately kills you, right? We engineered this compound to be maximally potent against stabilization, right? We're a 90%-plus stabilizer, near complete stabilizer per our label. That is the foundational scientific differentiation of this compound. What we've seen that yield in the clinic is distinct and differentiating data on basically everything we can measure. Going to our primary analysis from our phase III study where we saw market leading efficacy, our key marketing messages are 3-42-50. That's three months time to separation, a 42% reduction in overall survival events, and 50% reduction in hospitalization events. Really compelling data as physicians look across the space at what else is out there. I think importantly, the time to separation is particularly clearly distinct from the other options. We've also shown more recently, if you look at not time to first event, if you look at recurrent events, we're actually seeing a separation almost immediately at one month. That's really meaningful to physicians, and so that's the foundation of differentiation. We get asked by investors a lot about how do you plan to differentiate? Well, if you're reaching for Attruby, you already believe it's differentiated. 25% and growing in the first line setting, one in four patients are getting this because their physician believes it is the best drug for them and it's differentiated. I think we have to continue driving that message home, and we do that in various ways, right? We have a constant stream of new data that surrounds this core message 3-42-50. Lot of data in subpopulations that are pretty compelling, whether it's AFib or the variant patients. I think looking forward, some of the new data in the real world evidence domain is going to be really important and impactful. That's starting to come out right now. We'll have more to publish on that front as well. To give physicians a little bit more of a contemporaneous apples to apples comparison against tafamidis, which I think is going to stack up favorably as the clinical trial data have as well. Great. That makes a lot of sense. Q1, you did about $180 million, fifth quarter, a full launch. That's up from $145 million. When I look at the trajectory, it's linear. It's quite amazing to see it's not decelerating. First, it's kind of a narrow-minded question, but Q2, how does volume look? The weekly patient adds, is it consistent? Second part of that would be, Q1, there's technically seasonality, and yet you were able to manage to get sales to grow linearly. Wouldn't Q2 accelerate on gross net improvement, for instance? Yeah, that's a great question. Maybe let me start on the second part of the question, and I'll weave into my answer, hitting the first part as well. The point about seasonality in Q1, I understand that basically you're saying if there's a structural headwind in the funnel in Q1, shouldn't that be a big boost as you look sequentially to Q2? We didn't see any seasonality. We didn't see anything in the funnel that suggested an unusually poor conversion from demand to sales. That was pretty consistent with what we've seen in the quarters leading up to it. I wouldn't expect that headwind to tailwind dynamic Q1 to Q2. What we are seeing, which we're happy to see, is a steady and consistent improvement in that average weekly new starts number. I think that's a product of two things. One, our ability to continue capturing share in the frontline setting, two, a market that continues to expand. On the first part, we talked about the reasons why, right? I think increasingly physicians are seeing Attruby as the right option, they're getting more and more familiar with how to use it, how to get it, all of that, we've made it very easy to do all of that. That's driving share. The market is continuing to expand. This time last year, I'd have told you we think there's probably 2,000 to 3,000 treatment-naive patients coming to the bottom of the funnel every quarter. That looks more like 3,000 to 4,000 now, we don't see any signs of slowing on that front. I think that's how I'd put it. Understood. Momentum seems strong. You'll generate some more real-world evidence to further solidify your positioning. I think at one point, correct me if I'm wrong, you've kind of mentioned. "Wall Street's never wrong.". I could be wrong, but you mentioned maybe we get 30% peak market share. You're already at 25%, why couldn't this be 50%? Is this just a very conservative statement from you guys? Look, we're a bunch of former scientists or current scientists at this company, we're very data-oriented. The 30%-40% comes from market research we've done. Before the launch, that was coming from a survey of around 200 prescribers, and we've refreshed that along the way and continue to think 30%-40% share of frontline at peak is the right number for now. Obviously, we hope to push that higher, but we're still in the early days of launch, and I think that feels like the right range given where we are now. When we're in that range, hopefully soon, above 30%, maybe that's time to think, "Okay, where could this go? What's the upside here? Okay. Maybe two more, it's more investor-related questions before we move on to the pipeline. I just have to ask, there's generic Vyndaqel that could be available 2028. I think it's one filer. Generic Vyndamax, I think there's three that Pfizer settled for mid-2031. The question is, if generics can enter, why is there not a price vortex? Why is there no volume change affecting Attruby at the end of the day? Yeah. I think it comes back to the way this product is positioned and viewed increasingly as the best-in-class differentiated stabilizer and the right drug to start your patients on. I think the good news is we have almost six years to continue hammering that message and growing that belief in the marketplace before there's a generic tafamidis. Our hope is that when that eventually does happen in mid-2031, but really the theoretical pressure would come in 2032, that by then, it is very clear to patients, prescribers, payers that the best outcome is going to be had by starting a patient, hopefully early and on Attruby. There's going to be a health economic argument there too. It's better for the system to put a patient on the drug that's going to stop their disease and to prevent them from going to the hospital and to make them feel better and live longer. That's the most important thing we will do. No change in strategy as a result of the dynamics around the other products in the class. I think we're very confident that this is possible, right? To continue growing our brand through emergence of a generic in class. The reason for that is because if you look at analogs where something similar has happened, it's almost always the case that if you have a differentiated product and you're able to communicate that to the market, that you can continue accelerating growth through emergence of a generic. We've seen this in spaces like the androgen receptor inhibitors with Xtandi and Zytiga. We've seen it in PAH. We've seen it in a lot of different categories. Thank you. The other line of investor questioning, I'm pretty sure you'll respond in the same way, but I'm just curious. Ionis has their CARDIO-TTRansform data coming up in second half 2026. It is my understanding a proportion of patients could be on combo with tafamidis plus Ionis. If that succeeded, how should we think about that? Because there are no Ionis plus Attruby patients in that study. my understanding. Investors feel a little bit, they don't know how to think about it. What's your view about that? Yeah. First, this is a large, well-powered study in our minds. We think it's going to be successful, and we think it'll probably hit in all of the important subgroups. Just in terms of what we're planning for, that's clearly the scenario. What do we think would be the impact of that on the marketplace? Well, if that increases the view that combination therapy is interesting, particularly in second line, where we're seeing it mainly today, although there are some prescribers that are able to start patients on a stabilizer and a knockdown today. If that becomes a more consensus view from prescribers, we'll continue to communicate to them that they should put their patient on the best stabilizer. They already increasingly are reaching for Attruby with that in mind. Really, we're talking about instead of Attruby monotherapy and Attruby plus a knockdown, and if the doctor thinks that's appropriate for the patient, I think that's fine with us. Interestingly, we said this on our last earnings call, we've seen, I think, about a tripling of our share in the combination setting over the last several months, to the point where in patients that are receiving combination therapy, we're nearly at parity with tafamidis as the stabilizer in that combination. I think continue to hammer the differentiation messages that serve us well in monotherapy frontline will also help us if the market trends towards more combination use as well. Just to add on that, implicit in some of Tom's comments is if that is where the field trends with the data, it's important to recall that the way the paradigm and the treatment regimen is studied in that approach is stabilizer first, then knockdown added on. Like Tom is suggesting, really capturing frontline share remains our priority and will continue to be that case regardless of the outcome of the study. Yep. Exactly. Regardless, congratulations on the strong execution. More to come. Shifting gears, Ananth, to you then on ADH1 and encaleret. I think the NDA, remind me, was it submitted in May? That's right. Okay. You should get some kind of acceptance July. Well, I guess for both of you, anyway, do you expect an AdCom for this program? We would not, no. An AdCom would be in a case where the risk-benefit profile is ambiguous. I don't think there's a debate on that in the data set we've generated from our phase III. My understanding, there are no drugs approved for this indication, ADH1. Remind us the prevalence, and in the U.S., what % of those patients are diagnosed, identified, ready to go by the time you launch? It's a great question. If we look at the general population genetics databases, UK Biobank, Geisinger, TOPMed, All of Us, NHLBI, they all concentrate and triangulate to about a 1 in 25,000 estimate for the carrier frequency of gain-of-function calcium-sensing receptor variants. That approximates to about 10,000 to 12,000 patients in the U.S. As of the latest cut of the claims data, there's about 2,000 patients that have been claimed as ADH1 patients as of the end of 2025. That would approximate about one in five, one in six have been diagnosed. There's certainly a reasonable population to launch into. Again, like you mentioned, there's no other approved agents for these patients today, with a reasonable opportunity to grow that with increased disease awareness. One anecdote that we saw from the data from 2025 alone in the ICD claims, is about 70 new patients are being claimed a month on average. It's a fairly linear trend, at least, that we saw last year, and we'll continue to monitor how that evolves this year. Have you, at Bridge, found all these patients with the ICD-10 codes, to be clear? Do you know where they are? Yes. When we have access to these ICD-10 data sets, we are able to see institutions down to the NPI level of where patients are being claimed. Oh, go ahead. I would just say, the work between now and the early innings of launch is to get out there, confirm that they're ADH1, make sure they've had the right genetic confirmation so that they're actionable early on in the launch. Our team is in the field, our medical team is in the field, our sales leadership, including Ananth, are out in the field doing that, obviously in a compliant way today. We'll continue doing that over the next year plus. Okay. For a prevalence size of this in the 12,000 or so U.S. patients, what kind of pricing power do you think you have? Especially given the level of robust phase III data that you've generated. How should we think about the bookends? Sure. That's a great question. In our emerging and maturing conversations with payers in the U.S., what we've heard pretty consistently is that the payers are going to evaluate this condition in terms of its prevalence, like you're suggesting. Single high digit thousands is the price point that payers would manage the drug to label. Wherever that would index us is products like Crysvita, Skyclarys, Truseltiq. Those are products that have price points in those rare prevalent conditions that would bracket probably the pricing opportunity that we see as reasonable to payers. I see. Even within the 2,000 diagnosed patient pool, that's a big number on that kind of price point. Okay. Should you be approved, let's call it January of 2027, when do you exactly launch specifically? Why wouldn't there be a bolus, or are you expecting a bolus? We would be prepared and intend to launch soon after PDUFA date, similar to our dynamics with the Attruby. We've developed the muscle power and the infrastructure and the systems to support that cadence of launch, and certainly something we intend to continue. In terms of just the rate of adoption, there's going to be identified patients that we work through the data as well as our field interactions to drive diagnoses like we've discussed. Paired with just an understanding that the visit frequency doesn't suggest that everyone shows up to their doctor's office on day one. Typically, the guidelines recommend about a Q3 assessment schedule for these patients with their specialists. That translates more into every four or five months, every six months, sometimes, depending on people's lives. There is going to be a natural cadence to an initial launch adoption curve that depends on visit frequency as well as specialist interactions. Understood. As we think about, the beauty here is each of your products have life cycle management opportunities. You're starting a hypo-PTH as a phase III study in the summer- That's right. of 2026. Remind us how long it took you to do the ADH1 study from start to finish, and could it be a similar timeframe for hypo-PTH? Yeah. That's a great segue. We are intending to launch our RECLAIM-HP phase III study of encaleret and chronic hypoparathyroidism this summer, so in the coming months. In terms of the cadence of the study, we're planning for a six-month study analogous to our ADH1 program, but we think the enrollment opportunity is far more rapid. Relative to ADH1, there's precedent set here in terms of disease identification. The prevalent population is far more emergent. We believe that there is an opportunity to have a rapid enrollment curve with a six-month study. Life cycle management is really exciting here because with a successful study, we could have a potential indication expansion into a broader population soon after a PDUFA date. At the end of the day, for hypo-PTH, is it your guys' thinking that you might show superior efficacy relative to the competitor? Yeah. One of our design tenets that we're excited about is that we want to elevate the assessment of urine calcium, 24-hour urine calcium, and the primary endpoint to assess the response rate to encaleret. Why we think this is really important is it is an important surrogate for renal health, renal outcomes that really does play a long-term effect in these patients' lives. It's something that's currently not reflected on labels for replacement hormone and something that we think can not only differentiate encaleret from an evidence perspective, but also in terms of its use, in terms of driving urine calcium down. As of today, replacement hormone probably normalizes urine calcium about 60% of participants in their phase III. In our phase II sentinel study, we saw 80% of individuals not only normalize urine calcium but also blood calcium. There is an opportunity to demonstrate differentiated benefit on both metrics, and we're excited to evaluate our drug on that basis. I think in the post-surgical population, if we can replicate or even come close to what we've seen already in phase II, that'd be differentiating alone. It'd be an oral option in what is going to be a very large market where there's definitely a need for multiple types of products. On top of that, I think the non-surgical population is one where we could really be viewed as the best treatment option. There's a big unmet need there, right? These are patients that I don't really know that PTH replacement makes a lot of sense, so basically, untapped market opportunity for us to own. On top of, by then, ADH1 hopefully will be in the early days, but well ramped up and in key part of clinical practice. Okay, great. Moving on to LGMD, which actually could be approved later this year. Before I forget, all three of these programs, my understanding, you get a PRV voucher if they're approved, or? We will get one for limb-girdle. We don't expect to get one for encaleret, just given we have a pediatric study ongoing, but its first approval will be in adults. For infigratinib, we may get one as well. Okay, great. LGMD2I, again, another strong data set. I think you've mentioned the patient prevalence is around 7,000 in the U.S. and E.U. What % of that 7,000 is actually in the U.S., and by the time you're approved, what kind of line of sight do you have in terms of number of patients you've found? Yeah, good question. About 7,000 U.S. and Europe. There is a bias towards Europe due to a founder mutation in Northern Europe, but we see about 2,000-3,000 here in the U.S. based on genetic prevalence estimates. Most of those are going to be symptomatic and in need of therapy. It's a muscular dystrophy. It's evident that you have an issue, and actually, it's a really terrible condition where although it's not as rapid deterioration as something like DMD, it is a steady kind of torturous loss of function every day. Huge urgency to treat here, I think, is what we'll see. I think the thing that we need to do for the next couple of years is drive genetic diagnosis to be at a more complete level. Right now, most of these patients know they have a type of muscular dystrophy. Many of them know they have a limb-girdle muscular dystrophy. Until now, there's not really been a need to. Genotype- Determine that it's a 2I versus any of the many other types of limb-girdle muscular dystrophy. I would say the plus here is fairly concentrated site of care in the U.S. At least there's around 166 MDA centers, where about half the patient receive care. In those centers, testing is much more prevalent already. We need to drive that to be at a more complete level, and we also need to drive testing in other sites of care. Understood. Here, the patient prevalence seems like it's even more rare than ADH1. Going back to pricing power, is this kind of a DMD exon skipping type of pricing, which is like $1 million? I wouldn't put an exact number on it, we see here the bookends are something like Crysvita to the exon skippers. Certainly, that's the right range to think about. I think there's a tremendous value proposition here with this product for the system, for patients certainly, and for providers. If you think about what we've delivered here, I think differentiated amongst all neuromuscular data sets, honestly, where we've seen in a well-controlled study, an amazing effect, consistent effect on all of the important clinical endpoints that we measured. Importantly, and I think some people miss this, for patients that randomized to BBP-418 in our study, the mean actually increased across all these measures, or improved across all these measures, meaning the patients are doing better on drug than they were doing off drug, whereas you typically think about in muscular dystrophies, how can I just stop the decline or slow the decline? We actually gave back function, both mobility type measures, lung function. Really amazing outcome. We're hopeful that in our discussions with payers that we'll be able to price this well. Right. The same compound is going after 2U, 2M, Fukuyama, I think. Are those next trials going to be phase IIIs, straight to phase IIIs? 2U, 2M, as well as expansion into a broader pediatric population will happen starting now. Basically, those will be label-enabling studies. Fukuyama, really interesting Japan-centric opportunity. There's about 2,000 patients in Japan. That will need probably a little bit more work with the regulators to determine the exact registration path. We're working on that right now as well. Okay, great. The last couple minutes, achondroplasia. I think, is the NDA filing in Q3 of this year or- Second half, yeah. Second half. I guess you're unique here relative to the competitors, not only being oral, but with great data, but I think you're the only one at breakthrough designation. Are you expecting a priority review, and is there going to be ADCOM? I would not expect an AdCom, although we'll know more once we've submitted our NDA and heard from FDA at day 60. We may be eligible for a priority review voucher, that's interesting as well in terms of liquidity. I think you're right. This molecule is very clearly distinct amongst other options for various reasons. One, we know that this market wants a safe, convenient oral option. We've heard that time and time again, going back to the origination of this program at BridgeBio. On top of that, I think we've got the data to say we have a unique proposition in terms of efficacy as well. Primary endpoint result, obviously very strong, importantly, I think we've demonstrated first of its kind data against proportionality, which I think is immediately impactful in terms of daily function for these children, and will yield increasing benefits over time. I think importantly, I think that gives hope to the community that this drug actually will be able to finally give something other than just height. We're looking forward to elaborating on that hope and hopefully delivering against it. Should this be approved and on launch, the low-hanging fruit is it switches from the competitors or naive patients? Yeah. Actually, this is kind of an interesting market where there's an incumbent, but they're dramatically under-penetrated in the U.S. in particular. Our job is to both drive volume off of injectables, because I think there's going to be a lot of demand to get on an oral if you've already gotten on therapy. Also, we have to expand this market, and we know that the desire to start with an oral is certainly a reason that we are seeing under-penetration in the U.S. We really have to drive both of those. Okay, great. We'll wait on price, but unless you wanted to make. Here, it's a little bit easier, right? There's two comps out there that are, I think Voxzogo is around $400 and change, and the weekly injectable is average around $500. It's easier comps. Understood. That's a good guidepost on the low end, in my opinion. Okay. Very good. Well, thank you so much for taking the time. Thanks for all the updates, and thank you everyone for listening. Thanks, Andrew. Thank you, Andrew.

Speaker 2: Okay. Well, we're going to get started with our next session. I'm Andrew Tsai, Senior Biotech Analyst at Jefferies. Thanks for joining me today. It's my pleasure to have the BridgeBio team with me. To my direct left is Tom Trimarchi, CFO, and to his left, Ananth Sridhar, CEO of Endocrine Disorders. Welcome, both of you. Okay. okay Well, we're going to get started with our next session. well we're going to get started with our next session I'm Andrew Tsai, Senior Biotech Analyst at Jefferies. i'm andrew tsai senior biotech analyst at jefferies Thanks for joining me today. thanks for joining me today It's my pleasure to have the BridgeBio team with me. it's my pleasure to have the bridgebio team with me To my direct left is Tom Trimarchi, CFO, and to his left, Ananth Sridhar, CEO of Endocrine Disorders. to my direct left is tom trimarchi cfo and to his left ananth sridhar ceo of endocrine disorders Welcome, both of you. welcome both of you

Speaker 3: Thanks for having us. It's great to be back in New York, sun's shining. Thanks for having us. thanks for having us It's great to be back in New York, sun's shining. it's great to be back in new york sun's shining

Speaker 2: Yes Yes yes

Speaker 3: Knicks are in the finals, and we're at Jefferies. What more can you ask for? Knicks are in the finals, and we're at Jefferies. knicks are in the finals and we're at jefferies What more can you ask for? what more can you ask for

Speaker 2: Great. Those in the audience less familiar with the BridgeBio story or revisiting it, would you mind just briefly giving us an introduction about it, where you are with all your programs? Congratulations on all the success, by the way, milestones over the next 6-12 months would be very helpful. Great. great Those in the audience less familiar with the BridgeBio story or revisiting it, would you mind just briefly giving us an introduction about it, where you are with all your programs? those in the audience less familiar with the bridgebio story or revisiting it would you mind just briefly giving us an introduction about it where you are with all your programs Congratulations on all the success, by the way, milestones over the next 6-12 months would be very helpful. congratulations on all the success by the way milestones over the next 6-12 months would be very helpful

Speaker 3: Yeah, of course. BridgeBio Pharma is a company that's about 10 years old now. We were really built to deliver patient impact at scale, specifically focusing on patients with genetic diseases. In the first decade, I think we've been remarkably successful in pioneering a fairly new and innovative R&D model that's yielded the commercial and late-stage portfolio we have in front of us today, which includes Attruby, which is about a year into the launch, and it's really still ramping. We're annualizing about $720 million in the sales in the U.S., and also have seen strong uptake ex-U.S. with our partner Bayer. That's, I think, the first leg of the stool or chair. Yeah, of course. yeah of course BridgeBio Pharma is a company that's about 10 years old now. bridgebio pharma is a company that's about 10 years old now We were really built to deliver patient impact at scale, specifically focusing on patients with genetic diseases. we were really built to deliver patient impact at scale specifically focusing on patients with genetic diseases In the first decade, I think we've been remarkably successful in pioneering a fairly new and innovative R&D model that's yielded the commercial and late-stage portfolio we have in front of us today, which includes Attruby, which is about a year into the launch, and it's really still ramping. in the first decade i think we've been remarkably successful in pioneering a fairly new and innovative r&d model that's yielded the commercial and late-stage portfolio we have in front of us today which includes attruby which is about a year into the launch and it's really still ramping We're annualizing about $720 million in the sales in the U.S., and also have seen strong uptake ex-U.S. with our partner Bayer . we're annualizing about $720 million in the sales in the u.s and also have seen strong uptake ex-u.s with our partner bayer That's, I think, the first leg of the stool or chair. that's i think the first leg of the stool or chair Right behind that, we've got three products that are now on the other side of really remarkable phase III data sets that we're focused on submitting to the FDA and global regulatory authorities and will be launching starting at the end of this year. That's one in limb-girdle muscular dystrophy Type 2I/R9 (BBP-418). We see this as a huge unmet need. It'll be first drug to that market, clear $1 billion TAM for us. Right behind that, we have encaleret for hypoparathyroidism, with our first indication being a genetic subtype called ADH1. Ananth is here with me, hopefully we can spend a good amount of time on that. Right behind that, we've got three products that are now on the other side of really remarkable phase III data sets that we're focused on submitting to the FDA and global regulatory authorities and will be launching starting at the end of this year. right behind that we've got three products that are now on the other side of really remarkable phase iii data sets that we're focused on submitting to the fda and global regulatory authorities and will be launching starting at the end of this year That's one in limb-girdle muscular dystrophy Type 2I/R9 ( BBP-418). that's one in limb-girdle muscular dystrophy type 2i/r9 ( bbp-418) We see this as a huge unmet need. we see this as a huge unmet need It'll be first drug to that market, clear $1 billion TAM for us. it'll be first drug to that market clear $1 billion tam for us Right behind that, we have encaleret for hypoparathyroidism, with our first indication being a genetic subtype called ADH1. right behind that we have encaleret for hypoparathyroidism with our first indication being a genetic subtype called adh1 Ananth is here with me, hopefully we can spend a good amount of time on that. ananth is here with me hopefully we can spend a good amount of time on that Third will be infigratinib. That's a small molecule oral FGFR3 inhibitor for FGFR3-related skeletal dysplasias, starting with achondroplasia with an expansion into hypochondroplasia. That's a multibillion-dollar opportunity for us, clearly. Lot to be excited about. Yeah, I think the next 12 months are going to be focused on execution, where we can keep rolling on Attruby, keep seeing strong uptake, importantly in the first line treatment-naive setting. We'll be really putting our heads down and focusing on getting our next three drugs approved on time, hopefully with the right label, so that we can go and deliver these medicines to patients. Third will be infigratinib. third will be infigratinib That's a small molecule oral FGFR3 inhibitor for FGFR3-related skeletal dysplasias, starting with achondroplasia with an expansion into hypochondroplasia. that's a small molecule oral fgfr3 inhibitor for fgfr3-related skeletal dysplasias starting with achondroplasia with an expansion into hypochondroplasia That's a multibillion-dollar opportunity for us, clearly. that's a multibillion-dollar opportunity for us clearly Lot to be excited about. lot to be excited about Yeah, I think the next 12 months are going to be focused on execution, where we can keep rolling on Attruby, keep seeing strong uptake, importantly in the first line treatment-naive setting. yeah i think the next 12 months are going to be focused on execution where we can keep rolling on attruby keep seeing strong uptake importantly in the first line treatment-naive setting We'll be really putting our heads down and focusing on getting our next three drugs approved on time, hopefully with the right label, so that we can go and deliver these medicines to patients. we'll be really putting our heads down and focusing on getting our next three drugs approved on time hopefully with the right label so that we can go and deliver these medicines to patients

Speaker 2: Fantastic. Maybe starting with Attruby, it's doing very well. You're capturing, like you mentioned, a lot of first-line market share. I think it was above 25%. Fantastic. fantastic Maybe starting with Attruby, it's doing very well. maybe starting with attruby it's doing very well You're capturing, like you mentioned, a lot of first-line market share. you're capturing like you mentioned a lot of first-line market share I think it was above 25%. i think it was above 25%

Speaker 3: That's right. That's right. that's right

Speaker 2: most recently. most recently. most recently

Speaker 3: That's right. That's right. that's right

Speaker 2: The question is, how are you exactly doing that? Because you aren't first to market per se, yet you're doing this. There's no head-to-head study been done against Pfizer's compounds. What's resonating with doctors and so forth? The question is, how are you exactly doing that? the question is how are you exactly doing that Because you aren't first to market per se, yet you're doing this. because you aren't first to market per se yet you're doing this There's no head-to-head study been done against Pfizer's compounds. there's no head-to-head study been done against pfizer's compounds What's resonating with doctors and so forth? what's resonating with doctors and so forth

Speaker 3: Yeah. It's a great question. I would say going back to the design of the molecule, we made Attruby specifically to be an ultra-potent kind of next gen TTR stabilizer. Remember, this is a disease where your TTR tetramer is unstable, falls apart, makes amyloid, goes to your heart, ultimately kills you, right? We engineered this compound to be maximally potent against stabilization, right? We're a 90%-plus stabilizer, near complete stabilizer per our label. That is the foundational scientific differentiation of this compound. What we've seen that yield in the clinic is distinct and differentiating data on basically everything we can measure. Going to our primary analysis from our phase III study where we saw market leading efficacy, our key marketing messages are 3-42-50. Yeah. yeah It's a great question. it's a great question I would say going back to the design of the molecule, we made Attruby specifically to be an ultra-potent kind of next gen TTR stabilizer. i would say going back to the design of the molecule we made attruby specifically to be an ultra-potent kind of next gen ttr stabilizer Remember, this is a disease where your TTR tetramer is unstable, falls apart, makes amyloid, goes to your heart, ultimately kills you, right? remember this is a disease where your ttr tetramer is unstable falls apart makes amyloid goes to your heart ultimately kills you right We engineered this compound to be maximally potent against stabilization, right? we engineered this compound to be maximally potent against stabilization right We're a 90%-plus stabilizer, near complete stabilizer per our label. we're a 90%-plus stabilizer near complete stabilizer per our label That is the foundational scientific differentiation of this compound. that is the foundational scientific differentiation of this compound What we've seen that yield in the clinic is distinct and differentiating data on basically everything we can measure. what we've seen that yield in the clinic is distinct and differentiating data on basically everything we can measure Going to our primary analysis from our phase III study where we saw market leading efficacy, our key marketing messages are 3-42-50. going to our primary analysis from our phase iii study where we saw market leading efficacy our key marketing messages are 3-42-50 That's three months time to separation, a 42% reduction in overall survival events, and 50% reduction in hospitalization events. Really compelling data as physicians look across the space at what else is out there. I think importantly, the time to separation is particularly clearly distinct from the other options. We've also shown more recently, if you look at not time to first event, if you look at recurrent events, we're actually seeing a separation almost immediately at one month. That's three months time to separation, a 42% reduction in overall survival events, and 50% reduction in hospitalization events. that's three months time to separation a 42% reduction in overall survival events and 50% reduction in hospitalization events Really compelling data as physicians look across the space at what else is out there. really compelling data as physicians look across the space at what else is out there I think importantly, the time to separation is particularly clearly distinct from the other options. i think importantly the time to separation is particularly clearly distinct from the other options We've also shown more recently, if you look at not time to first event, if you look at recurrent events, we're actually seeing a separation almost immediately at one month. we've also shown more recently if you look at not time to first event if you look at recurrent events we're actually seeing a separation almost immediately at one month That's really meaningful to physicians, and so that's the foundation of differentiation. We get asked by investors a lot about how do you plan to differentiate? Well, if you're reaching for Attruby, you already believe it's differentiated. 25% and growing in the first line setting, one in four patients are getting this because their physician believes it is the best drug for them and it's differentiated. That's really meaningful to physicians, and so that's the foundation of differentiation. that's really meaningful to physicians and so that's the foundation of differentiation We get asked by investors a lot about how do you plan to differentiate? we get asked by investors a lot about how do you plan to differentiate Well, if you're reaching for Attruby, you already believe it's differentiated. 25% and growing in the first line setting, one in four patients are getting this because their physician believes it is the best drug for them and it's differentiated. well if you're reaching for attruby you already believe it's differentiated 25% and growing in the first line setting one in four patients are getting this because their physician believes it is the best drug for them and it's differentiated I think we have to continue driving that message home, and we do that in various ways, right? We have a constant stream of new data that surrounds this core message 3-42-50. Lot of data in subpopulations that are pretty compelling, whether it's AFib or the variant patients. I think looking forward, some of the new data in the real world evidence domain is going to be really important and impactful. That's starting to come out right now. We'll have more to publish on that front as well. To give physicians a little bit more of a contemporaneous apples to apples comparison against tafamidis, which I think is going to stack up favorably as the clinical trial data have as well. I think we have to continue driving that message home, and we do that in various ways, right? i think we have to continue driving that message home and we do that in various ways right We have a constant stream of new data that surrounds this core message 3-42-50. we have a constant stream of new data that surrounds this core message 3-42-50 Lot of data in subpopulations that are pretty compelling, whether it's AFib or the variant patients. lot of data in subpopulations that are pretty compelling whether it's afib or the variant patients I think looking forward, some of the new data in the real world evidence domain is going to be really important and impactful. i think looking forward some of the new data in the real world evidence domain is going to be really important and impactful That's starting to come out right now. that's starting to come out right now We'll have more to publish on that front as well. we'll have more to publish on that front as well To give physicians a little bit more of a contemporaneous apples to apples comparison against tafamidis, which I think is going to stack up favorably as the clinical trial data have as well. to give physicians a little bit more of a contemporaneous apples to apples comparison against tafamidis which i think is going to stack up favorably as the clinical trial data have as well

Speaker 2: Great. That makes a lot of sense. Q1, you did about $180 million, fifth quarter, a full launch. That's up from $145 million. When I look at the trajectory, it's linear. It's quite amazing to see it's not decelerating. First, it's kind of a narrow-minded question, but Q2, how does volume look? The weekly patient adds, is it consistent? Second part of that would be, Q1, there's technically seasonality, and yet you were able to manage to get sales to grow linearly. Wouldn't Q2 accelerate on gross net improvement, for instance? Great. great That makes a lot of sense. that makes a lot of sense Q1, you did about $180 million, fifth quarter, a full launch. q1 you did about $180 million fifth quarter a full launch That's up from $145 million. that's up from $145 million When I look at the trajectory, it's linear. when i look at the trajectory it's linear It's quite amazing to see it's not decelerating. it's quite amazing to see it's not decelerating First, it's kind of a narrow-minded question, but Q2, how does volume look? first it's kind of a narrow-minded question but q2 how does volume look The weekly patient adds, is it consistent? the weekly patient adds is it consistent Second part of that would be, Q1, there's technically seasonality, and yet you were able to manage to get sales to grow linearly. second part of that would be q1 there's technically seasonality and yet you were able to manage to get sales to grow linearly Wouldn't Q2 accelerate on gross net improvement, for instance? wouldn't q2 accelerate on gross net improvement for instance

Speaker 3: Yeah, that's a great question. Maybe let me start on the second part of the question, and I'll weave into my answer, hitting the first part as well. The point about seasonality in Q1, I understand that basically you're saying if there's a structural headwind in the funnel in Q1, shouldn't that be a big boost as you look sequentially to Q2? We didn't see any seasonality. We didn't see anything in the funnel that suggested an unusually poor conversion from demand to sales. That was pretty consistent with what we've seen in the quarters leading up to it. I wouldn't expect that headwind to tailwind dynamic Q1 to Q2. What we are seeing, which we're happy to see, is a steady and consistent improvement in that average weekly new starts number. I think that's a product of two things. Yeah, that's a great question. yeah that's a great question Maybe let me start on the second part of the question, and I'll weave into my answer, hitting the first part as well. maybe let me start on the second part of the question and i'll weave into my answer hitting the first part as well The point about seasonality in Q1, I understand that basically you're saying if there's a structural headwind in the funnel in Q1, shouldn't that be a big boost as you look sequentially to Q2? the point about seasonality in q1 i understand that basically you're saying if there's a structural headwind in the funnel in q1 shouldn't that be a big boost as you look sequentially to q2 We didn't see any seasonality. we didn't see any seasonality We didn't see anything in the funnel that suggested an unusually poor conversion from demand to sales. we didn't see anything in the funnel that suggested an unusually poor conversion from demand to sales That was pretty consistent with what we've seen in the quarters leading up to it. that was pretty consistent with what we've seen in the quarters leading up to it I wouldn't expect that headwind to tailwind dynamic Q1 to Q2. i wouldn't expect that headwind to tailwind dynamic q1 to q2 What we are seeing, which we're happy to see, is a steady and consistent improvement in that average weekly new starts number. what we are seeing which we're happy to see is a steady and consistent improvement in that average weekly new starts number I think that's a product of two things. i think that's a product of two things One, our ability to continue capturing share in the frontline setting, two, a market that continues to expand. On the first part, we talked about the reasons why, right? I think increasingly physicians are seeing Attruby as the right option, they're getting more and more familiar with how to use it, how to get it, all of that, we've made it very easy to do all of that. That's driving share. The market is continuing to expand. This time last year, I'd have told you we think there's probably 2,000 to 3,000 treatment-naive patients coming to the bottom of the funnel every quarter. That looks more like 3,000 to 4,000 now, we don't see any signs of slowing on that front. I think that's how I'd put it. One, our ability to continue capturing share in the frontline setting, two, a market that continues to expand. one our ability to continue capturing share in the frontline setting two a market that continues to expand On the first part, we talked about the reasons why, right? on the first part we talked about the reasons why right I think increasingly physicians are seeing Attruby as the right option, they're getting more and more familiar with how to use it, how to get it, all of that, we've made it very easy to do all of that. i think increasingly physicians are seeing attruby as the right option they're getting more and more familiar with how to use it how to get it all of that we've made it very easy to do all of that That's driving share. that's driving share The market is continuing to expand. the market is continuing to expand This time last year, I'd have told you we think there's probably 2,000 to 3,000 treatment-naive patients coming to the bottom of the funnel every quarter. this time last year i'd have told you we think there's probably 2,000 to 3,000 treatment-naive patients coming to the bottom of the funnel every quarter That looks more like 3,000 to 4,000 now, we don't see any signs of slowing on that front. that looks more like 3,000 to 4,000 now we don't see any signs of slowing on that front I think that's how I'd put it. i think that's how i'd put it

Speaker 2: Understood. Momentum seems strong. You'll generate some more real-world evidence to further solidify your positioning. I think at one point, correct me if I'm wrong, you've kind of mentioned. "Wall Street's never wrong.". I could be wrong, but you mentioned maybe we get 30% peak market share. You're already at 25%, why couldn't this be 50%? Is this just a very conservative statement from you guys? Understood. understood Momentum seems strong. momentum seems strong You'll generate some more real-world evidence to further solidify your positioning. you'll generate some more real-world evidence to further solidify your positioning I think at one point, correct me if I'm wrong, you've kind of mentioned. "Wall Street's never wrong.". i think at one point correct me if i'm wrong you've kind of mentioned "wall street's never wrong." I could be wrong, but you mentioned maybe we get 30% peak market share. i could be wrong but you mentioned maybe we get 30% peak market share You're already at 25%, why couldn't this be 50%? you're already at 25% why couldn't this be 50% Is this just a very conservative statement from you guys? is this just a very conservative statement from you guys

Speaker 3: Look, we're a bunch of former scientists or current scientists at this company, we're very data-oriented. The 30%-40% comes from market research we've done. Before the launch, that was coming from a survey of around 200 prescribers, and we've refreshed that along the way and continue to think 30%-40% share of frontline at peak is the right number for now. Obviously, we hope to push that higher, but we're still in the early days of launch, and I think that feels like the right range given where we are now. When we're in that range, hopefully soon, above 30%, maybe that's time to think, "Okay, where could this go? What's the upside here? Look, we're a bunch of former scientists or current scientists at this company, we're very data-oriented. look we're a bunch of former scientists or current scientists at this company we're very data-oriented The 30%-40% comes from market research we've done. the 30%-40% comes from market research we've done Before the launch, that was coming from a survey of around 200 prescribers, and we've refreshed that along the way and continue to think 30%-40% share of frontline at peak is the right number for now. before the launch that was coming from a survey of around 200 prescribers and we've refreshed that along the way and continue to think 30%-40% share of frontline at peak is the right number for now Obviously, we hope to push that higher, but we're still in the early days of launch, and I think that feels like the right range given where we are now. obviously we hope to push that higher but we're still in the early days of launch and i think that feels like the right range given where we are now When we're in that range, hopefully soon, above 30%, maybe that's time to think, "Okay, where could this go? when we're in that range hopefully soon above 30% maybe that's time to think "okay where could this go What's the upside here? what's the upside here

Speaker 2: Okay. Maybe two more, it's more investor-related questions before we move on to the pipeline. I just have to ask, there's generic Vyndaqel that could be available 2028. I think it's one filer. Generic Vyndamax, I think there's three that Pfizer settled for mid-2031. The question is, if generics can enter, why is there not a price vortex? Why is there no volume change affecting Attruby at the end of the day? Okay. okay Maybe two more, it's more investor-related questions before we move on to the pipeline. maybe two more it's more investor-related questions before we move on to the pipeline I just have to ask, there's generic Vyndaqel that could be available 2028. i just have to ask there's generic vyndaqel that could be available 2028 I think it's one filer. i think it's one filer Generic Vyndamax, I think there's three that Pfizer settled for mid-2031. generic vyndamax i think there's three that pfizer settled for mid-2031 The question is, if generics can enter, why is there not a price vortex? the question is if generics can enter why is there not a price vortex Why is there no volume change affecting Attruby at the end of the day? why is there no volume change affecting attruby at the end of the day

Speaker 3: Yeah. I think it comes back to the way this product is positioned and viewed increasingly as the best-in-class differentiated stabilizer and the right drug to start your patients on. I think the good news is we have almost six years to continue hammering that message and growing that belief in the marketplace before there's a generic tafamidis. Our hope is that when that eventually does happen in mid-2031, but really the theoretical pressure would come in 2032, that by then, it is very clear to patients, prescribers, payers that the best outcome is going to be had by starting a patient, hopefully early and on Attruby. There's going to be a health economic argument there too. Yeah. yeah I think it comes back to the way this product is positioned and viewed increasingly as the best-in-class differentiated stabilizer and the right drug to start your patients on. i think it comes back to the way this product is positioned and viewed increasingly as the best-in-class differentiated stabilizer and the right drug to start your patients on I think the good news is we have almost six years to continue hammering that message and growing that belief in the marketplace before there's a generic tafamidis. i think the good news is we have almost six years to continue hammering that message and growing that belief in the marketplace before there's a generic tafamidis Our hope is that when that eventually does happen in mid-2031, but really the theoretical pressure would come in 2032, that by then, it is very clear to patients, prescribers, payers that the best outcome is going to be had by starting a patient, hopefully early and on Attruby. our hope is that when that eventually does happen in mid-2031 but really the theoretical pressure would come in 2032 that by then it is very clear to patients prescribers payers that the best outcome is going to be had by starting a patient hopefully early and on attruby There's going to be a health economic argument there too. there's going to be a health economic argument there too It's better for the system to put a patient on the drug that's going to stop their disease and to prevent them from going to the hospital and to make them feel better and live longer. That's the most important thing we will do. No change in strategy as a result of the dynamics around the other products in the class. I think we're very confident that this is possible, right? To continue growing our brand through emergence of a generic in class. It's better for the system to put a patient on the drug that's going to stop their disease and to prevent them from going to the hospital and to make them feel better and live longer. it's better for the system to put a patient on the drug that's going to stop their disease and to prevent them from going to the hospital and to make them feel better and live longer That's the most important thing we will do. that's the most important thing we will do No change in strategy as a result of the dynamics around the other products in the class. no change in strategy as a result of the dynamics around the other products in the class I think we're very confident that this is possible, right? i think we're very confident that this is possible right To continue growing our brand through emergence of a generic in class. to continue growing our brand through emergence of a generic in class The reason for that is because if you look at analogs where something similar has happened, it's almost always the case that if you have a differentiated product and you're able to communicate that to the market, that you can continue accelerating growth through emergence of a generic. We've seen this in spaces like the androgen receptor inhibitors with Xtandi and Zytiga. We've seen it in PAH. We've seen it in a lot of different categories. The reason for that is because if you look at analogs where something similar has happened, it's almost always the case that if you have a differentiated product and you're able to communicate that to the market, that you can continue accelerating growth through emergence of a generic. the reason for that is because if you look at analogs where something similar has happened it's almost always the case that if you have a differentiated product and you're able to communicate that to the market that you can continue accelerating growth through emergence of a generic We've seen this in spaces like the androgen receptor inhibitors with Xtandi and Zytiga. we've seen this in spaces like the androgen receptor inhibitors with xtandi and zytiga We've seen it in PAH. we've seen it in pah We've seen it in a lot of different categories. we've seen it in a lot of different categories

Speaker 2: Thank you. The other line of investor questioning, I'm pretty sure you'll respond in the same way, but I'm just curious. Ionis has their CARDIO-TTRansform data coming up in second half 2026. It is my understanding a proportion of patients could be on combo with tafamidis plus Ionis. If that succeeded, how should we think about that? Because there are no Ionis plus Attruby patients in that study. my understanding. Investors feel a little bit, they don't know how to think about it. What's your view about that? Thank you. thank you The other line of investor questioning, I'm pretty sure you'll respond in the same way, but I'm just curious. the other line of investor questioning i'm pretty sure you'll respond in the same way but i'm just curious Ionis has their CARDIO-TTRansform data coming up in second half 2026. ionis has their cardio-ttransform data coming up in second half 2026 It is my understanding a proportion of patients could be on combo with tafamidis plus Ionis. it is my understanding a proportion of patients could be on combo with tafamidis plus ionis If that succeeded, how should we think about that? if that succeeded how should we think about that Because there are no Ionis plus Attruby patients in that study. my understanding. because there are no ionis plus attruby patients in that study my understanding Investors feel a little bit, they don't know how to think about it. investors feel a little bit they don't know how to think about it What's your view about that? what's your view about that

Speaker 3: Yeah. First, this is a large, well-powered study in our minds. We think it's going to be successful, and we think it'll probably hit in all of the important subgroups. Just in terms of what we're planning for, that's clearly the scenario. What do we think would be the impact of that on the marketplace? Well, if that increases the view that combination therapy is interesting, particularly in second line, where we're seeing it mainly today, although there are some prescribers that are able to start patients on a stabilizer and a knockdown today. If that becomes a more consensus view from prescribers, we'll continue to communicate to them that they should put their patient on the best stabilizer. They already increasingly are reaching for Attruby with that in mind. Yeah. yeah First, this is a large, well-powered study in our minds. first this is a large well-powered study in our minds We think it's going to be successful, and we think it'll probably hit in all of the important subgroups. we think it's going to be successful and we think it'll probably hit in all of the important subgroups Just in terms of what we're planning for, that's clearly the scenario. just in terms of what we're planning for that's clearly the scenario What do we think would be the impact of that on the marketplace? what do we think would be the impact of that on the marketplace Well, if that increases the view that combination therapy is interesting, particularly in second line, where we're seeing it mainly today, although there are some prescribers that are able to start patients on a stabilizer and a knockdown today. well if that increases the view that combination therapy is interesting particularly in second line where we're seeing it mainly today although there are some prescribers that are able to start patients on a stabilizer and a knockdown today If that becomes a more consensus view from prescribers, we'll continue to communicate to them that they should put their patient on the best stabilizer. if that becomes a more consensus view from prescribers we'll continue to communicate to them that they should put their patient on the best stabilizer They already increasingly are reaching for Attruby with that in mind. they already increasingly are reaching for attruby with that in mind Really, we're talking about instead of Attruby monotherapy and Attruby plus a knockdown, and if the doctor thinks that's appropriate for the patient, I think that's fine with us. Interestingly, we said this on our last earnings call, we've seen, I think, about a tripling of our share in the combination setting over the last several months, to the point where in patients that are receiving combination therapy, we're nearly at parity with tafamidis as the stabilizer in that combination. I think continue to hammer the differentiation messages that serve us well in monotherapy frontline will also help us if the market trends towards more combination use as well. Really, we're talking about instead of Attruby monotherapy and Attruby plus a knockdown, and if the doctor thinks that's appropriate for the patient, I think that's fine with us. really we're talking about instead of attruby monotherapy and attruby plus a knockdown and if the doctor thinks that's appropriate for the patient i think that's fine with us Interestingly, we said this on our last earnings call, we've seen, I think, about a tripling of our share in the combination setting over the last several months, to the point where in patients that are receiving combination therapy, we're nearly at parity with tafamidis as the stabilizer in that combination. interestingly we said this on our last earnings call we've seen i think about a tripling of our share in the combination setting over the last several months to the point where in patients that are receiving combination therapy we're nearly at parity with tafamidis as the stabilizer in that combination I think continue to hammer the differentiation messages that serve us well in monotherapy frontline will also help us if the market trends towards more combination use as well. i think continue to hammer the differentiation messages that serve us well in monotherapy frontline will also help us if the market trends towards more combination use as well

Speaker 1: Just to add on that, implicit in some of Tom's comments is if that is where the field trends with the data, it's important to recall that the way the paradigm and the treatment regimen is studied in that approach is stabilizer first, then knockdown added on. Like Tom is suggesting, really capturing frontline share remains our priority and will continue to be that case regardless of the outcome of the study. Just to add on that, implicit in some of Tom's comments is if that is where the field trends with the data, it's important to recall that the way the paradigm and the treatment regimen is studied in that approach is stabilizer first, then knockdown added on. just to add on that implicit in some of tom's comments is if that is where the field trends with the data it's important to recall that the way the paradigm and the treatment regimen is studied in that approach is stabilizer first then knockdown added on Like Tom is suggesting, really capturing frontline share remains our priority and will continue to be that case regardless of the outcome of the study. like tom is suggesting really capturing frontline share remains our priority and will continue to be that case regardless of the outcome of the study

Speaker 3: Yep. Exactly. Yep. yep Exactly. exactly

Speaker 2: Regardless, congratulations on the strong execution. More to come. Shifting gears, Ananth, to you then on ADH1 and encaleret. I think the NDA, remind me, was it submitted in May? Regardless, congratulations on the strong execution. regardless congratulations on the strong execution More to come. more to come Shifting gears, Ananth, to you then on ADH1 and encaleret. shifting gears ananth to you then on adh1 and encaleret I think the NDA, remind me, was it submitted in May? i think the nda remind me was it submitted in may

Speaker 1: That's right. That's right. that's right

Speaker 2: Okay. You should get some kind of acceptance July. Well, I guess for both of you, anyway, do you expect an AdCom for this program? Okay. okay You should get some kind of acceptance July. you should get some kind of acceptance july Well, I guess for both of you, anyway, do you expect an AdCom for this program? well i guess for both of you anyway do you expect an adcom for this program

Speaker 1: We would not, no. An AdCom would be in a case where the risk-benefit profile is ambiguous. I don't think there's a debate on that in the data set we've generated from our phase III. We would not, no. we would not no An AdCom would be in a case where the risk-benefit profile is ambiguous. an adcom would be in a case where the risk-benefit profile is ambiguous I don't think there's a debate on that in the data set we've generated from our phase III. i don't think there's a debate on that in the data set we've generated from our phase iii

Speaker 2: My understanding, there are no drugs approved for this indication, ADH1. Remind us the prevalence, and in the U.S., what % of those patients are diagnosed, identified, ready to go by the time you launch? My understanding, there are no drugs approved for this indication, ADH1. my understanding there are no drugs approved for this indication adh1 Remind us the prevalence, and in the U.S., what % of those patients are diagnosed, identified, ready to go by the time you launch? remind us the prevalence and in the u.s what % of those patients are diagnosed identified ready to go by the time you launch

Speaker 1: It's a great question. If we look at the general population genetics databases, UK Biobank, Geisinger, TOPMed, All of Us, NHLBI, they all concentrate and triangulate to about a 1 in 25,000 estimate for the carrier frequency of gain-of-function calcium-sensing receptor variants. That approximates to about 10,000 to 12,000 patients in the U.S. As of the latest cut of the claims data, there's about 2,000 patients that have been claimed as ADH1 patients as of the end of 2025. That would approximate about one in five, one in six have been diagnosed. There's certainly a reasonable population to launch into. Again, like you mentioned, there's no other approved agents for these patients today, with a reasonable opportunity to grow that with increased disease awareness. It's a great question. it's a great question If we look at the general population genetics databases, UK Biobank, Geisinger, TOPMed, All of Us, NHLBI, they all concentrate and triangulate to about a 1 in 25,000 estimate for the carrier frequency of gain-of-function calcium-sensing receptor variants. if we look at the general population genetics databases uk biobank geisinger topmed all of us nhlbi they all concentrate and triangulate to about a 1 in 25,000 estimate for the carrier frequency of gain-of-function calcium-sensing receptor variants That approximates to about 10,000 to 12,000 patients in the U.S. that approximates to about 10,000 to 12,000 patients in the u.s As of the latest cut of the claims data, there's about 2,000 patients that have been claimed as ADH1 patients as of the end of 2025. as of the latest cut of the claims data there's about 2,000 patients that have been claimed as adh1 patients as of the end of 2025 That would approximate about one in five, one in six have been diagnosed. that would approximate about one in five one in six have been diagnosed There's certainly a reasonable population to launch into. there's certainly a reasonable population to launch into Again, like you mentioned, there's no other approved agents for these patients today, with a reasonable opportunity to grow that with increased disease awareness. again like you mentioned there's no other approved agents for these patients today with a reasonable opportunity to grow that with increased disease awareness One anecdote that we saw from the data from 2025 alone in the ICD claims, is about 70 new patients are being claimed a month on average. It's a fairly linear trend, at least, that we saw last year, and we'll continue to monitor how that evolves this year. One anecdote that we saw from the data from 2025 alone in the ICD claims, is about 70 new patients are being claimed a month on average. one anecdote that we saw from the data from 2025 alone in the icd claims is about 70 new patients are being claimed a month on average It's a fairly linear trend, at least, that we saw last year, and we'll continue to monitor how that evolves this year. it's a fairly linear trend at least that we saw last year and we'll continue to monitor how that evolves this year

Speaker 2: Have you, at Bridge, found all these patients with the ICD-10 codes, to be clear? Do you know where they are? Have you, at Bridge, found all these patients with the ICD-10 codes, to be clear? have you at bridge found all these patients with the icd-10 codes to be clear Do you know where they are? do you know where they are

Speaker 1: Yes. When we have access to these ICD-10 data sets, we are able to see institutions down to the NPI level of where patients are being claimed. Yes. yes When we have access to these ICD-10 data sets, we are able to see institutions down to the NPI level of where patients are being claimed. when we have access to these icd-10 data sets we are able to see institutions down to the npi level of where patients are being claimed

Speaker 2: Oh, go ahead. Oh, go ahead. oh go ahead

Speaker 3: I would just say, the work between now and the early innings of launch is to get out there, confirm that they're ADH1, make sure they've had the right genetic confirmation so that they're actionable early on in the launch. Our team is in the field, our medical team is in the field, our sales leadership, including Ananth, are out in the field doing that, obviously in a compliant way today. We'll continue doing that over the next year plus. I would just say, the work between now and the early innings of launch is to get out there, confirm that they're ADH1, make sure they've had the right genetic confirmation so that they're actionable early on in the launch. i would just say the work between now and the early innings of launch is to get out there confirm that they're adh1 make sure they've had the right genetic confirmation so that they're actionable early on in the launch Our team is in the field, our medical team is in the field, our sales leadership, including Ananth, are out in the field doing that, obviously in a compliant way today. our team is in the field our medical team is in the field our sales leadership including ananth are out in the field doing that obviously in a compliant way today We'll continue doing that over the next year plus. we'll continue doing that over the next year plus

Speaker 2: Okay. For a prevalence size of this in the 12,000 or so U.S. patients, what kind of pricing power do you think you have? Especially given the level of robust phase III data that you've generated. How should we think about the bookends? Okay. okay For a prevalence size of this in the 12,000 or so U.S. patients, what kind of pricing power do you think you have? for a prevalence size of this in the 12,000 or so u.s patients what kind of pricing power do you think you have Especially given the level of robust phase III data that you've generated. especially given the level of robust phase iii data that you've generated How should we think about the bookends? how should we think about the bookends

Speaker 1: Sure. That's a great question. In our emerging and maturing conversations with payers in the U.S., what we've heard pretty consistently is that the payers are going to evaluate this condition in terms of its prevalence, like you're suggesting. Single high digit thousands is the price point that payers would manage the drug to label. Wherever that would index us is products like Crysvita, Skyclarys, Truseltiq. Those are products that have price points in those rare prevalent conditions that would bracket probably the pricing opportunity that we see as reasonable to payers. Sure. sure That's a great question. that's a great question In our emerging and maturing conversations with payers in the U.S., what we've heard pretty consistently is that the payers are going to evaluate this condition in terms of its prevalence, like you're suggesting. in our emerging and maturing conversations with payers in the u.s what we've heard pretty consistently is that the payers are going to evaluate this condition in terms of its prevalence like you're suggesting Single high digit thousands is the price point that payers would manage the drug to label. single high digit thousands is the price point that payers would manage the drug to label Wherever that would index us is products like Crysvita, Skyclarys, Truseltiq. wherever that would index us is products like crysvita skyclarys truseltiq Those are products that have price points in those rare prevalent conditions that would bracket probably the pricing opportunity that we see as reasonable to payers. those are products that have price points in those rare prevalent conditions that would bracket probably the pricing opportunity that we see as reasonable to payers

Speaker 2: I see. Even within the 2,000 diagnosed patient pool, that's a big number on that kind of price point. Okay. I see. i see Even within the 2,000 diagnosed patient pool, that's a big number on that kind of price point. even within the 2,000 diagnosed patient pool that's a big number on that kind of price point Okay. okay Should you be approved, let's call it January of 2027, when do you exactly launch specifically? Why wouldn't there be a bolus, or are you expecting a bolus? Should you be approved, let's call it January of 2027, when do you exactly launch specifically? should you be approved let's call it january of 2027 when do you exactly launch specifically Why wouldn't there be a bolus, or are you expecting a bolus? why wouldn't there be a bolus or are you expecting a bolus

Speaker 1: We would be prepared and intend to launch soon after PDUFA date, similar to our dynamics with the Attruby. We've developed the muscle power and the infrastructure and the systems to support that cadence of launch, and certainly something we intend to continue. In terms of just the rate of adoption, there's going to be identified patients that we work through the data as well as our field interactions to drive diagnoses like we've discussed. Paired with just an understanding that the visit frequency doesn't suggest that everyone shows up to their doctor's office on day one. We would be prepared and intend to launch soon after PDUFA date, similar to our dynamics with the Attruby. we would be prepared and intend to launch soon after pdufa date similar to our dynamics with the attruby We've developed the muscle power and the infrastructure and the systems to support that cadence of launch, and certainly something we intend to continue. we've developed the muscle power and the infrastructure and the systems to support that cadence of launch and certainly something we intend to continue In terms of just the rate of adoption, there's going to be identified patients that we work through the data as well as our field interactions to drive diagnoses like we've discussed. in terms of just the rate of adoption there's going to be identified patients that we work through the data as well as our field interactions to drive diagnoses like we've discussed Paired with just an understanding that the visit frequency doesn't suggest that everyone shows up to their doctor's office on day one. paired with just an understanding that the visit frequency doesn't suggest that everyone shows up to their doctor's office on day one Typically, the guidelines recommend about a Q3 assessment schedule for these patients with their specialists. That translates more into every four or five months, every six months, sometimes, depending on people's lives. There is going to be a natural cadence to an initial launch adoption curve that depends on visit frequency as well as specialist interactions. Typically, the guidelines recommend about a Q3 assessment schedule for these patients with their specialists. typically the guidelines recommend about a q3 assessment schedule for these patients with their specialists That translates more into every four or five months, every six months, sometimes, depending on people's lives. that translates more into every four or five months every six months sometimes depending on people's lives There is going to be a natural cadence to an initial launch adoption curve that depends on visit frequency as well as specialist interactions. there is going to be a natural cadence to an initial launch adoption curve that depends on visit frequency as well as specialist interactions

Speaker 2: Understood. As we think about, the beauty here is each of your products have life cycle management opportunities. You're starting a hypo-PTH as a phase III study in the summer- Understood. understood As we think about, the beauty here is each of your products have life cycle management opportunities. as we think about the beauty here is each of your products have life cycle management opportunities You're starting a hypo-PTH as a phase III study in the summer- you're starting a hypo-pth as a phase iii study in the summer-

Speaker 1: That's right. That's right. that's right

Speaker 2: of 2026. Remind us how long it took you to do the ADH1 study from start to finish, and could it be a similar timeframe for hypo-PTH? of 2026. of 2026 Remind us how long it took you to do the ADH1 study from start to finish, and could it be a similar timeframe for hypo-PTH? remind us how long it took you to do the adh1 study from start to finish and could it be a similar timeframe for hypo-pth

Speaker 1: Yeah. That's a great segue. We are intending to launch our RECLAIM-HP phase III study of encaleret and chronic hypoparathyroidism this summer, so in the coming months. In terms of the cadence of the study, we're planning for a six-month study analogous to our ADH1 program, but we think the enrollment opportunity is far more rapid. Relative to ADH1, there's precedent set here in terms of disease identification. The prevalent population is far more emergent. We believe that there is an opportunity to have a rapid enrollment curve with a six-month study. Life cycle management is really exciting here because with a successful study, we could have a potential indication expansion into a broader population soon after a PDUFA date. Yeah. yeah That's a great segue. that's a great segue We are intending to launch our RECLAIM-HP phase III study of encaleret and chronic hypoparathyroidism this summer, so in the coming months. we are intending to launch our reclaim-hp phase iii study of encaleret and chronic hypoparathyroidism this summer so in the coming months In terms of the cadence of the study, we're planning for a six-month study analogous to our ADH1 program, but we think the enrollment opportunity is far more rapid. in terms of the cadence of the study we're planning for a six-month study analogous to our adh1 program but we think the enrollment opportunity is far more rapid Relative to ADH1, there's precedent set here in terms of disease identification. relative to adh1 there's precedent set here in terms of disease identification The prevalent population is far more emergent. the prevalent population is far more emergent We believe that there is an opportunity to have a rapid enrollment curve with a six-month study. we believe that there is an opportunity to have a rapid enrollment curve with a six-month study Life cycle management is really exciting here because with a successful study, we could have a potential indication expansion into a broader population soon after a PDUFA date. life cycle management is really exciting here because with a successful study we could have a potential indication expansion into a broader population soon after a pdufa date

Speaker 2: At the end of the day, for hypo-PTH, is it your guys' thinking that you might show superior efficacy relative to the competitor? At the end of the day, for hypo-PTH, is it your guys' thinking that you might show superior efficacy relative to the competitor? at the end of the day for hypo-pth is it your guys' thinking that you might show superior efficacy relative to the competitor

Speaker 1: Yeah. One of our design tenets that we're excited about is that we want to elevate the assessment of urine calcium, 24-hour urine calcium, and the primary endpoint to assess the response rate to encaleret. Why we think this is really important is it is an important surrogate for renal health, renal outcomes that really does play a long-term effect in these patients' lives. It's something that's currently not reflected on labels for replacement hormone and something that we think can not only differentiate encaleret from an evidence perspective, but also in terms of its use, in terms of driving urine calcium down. As of today, replacement hormone probably normalizes urine calcium about 60% of participants in their phase III. In our phase II sentinel study, we saw 80% of individuals not only normalize urine calcium but also blood calcium. Yeah. yeah One of our design tenets that we're excited about is that we want to elevate the assessment of urine calcium, 24-hour urine calcium, and the primary endpoint to assess the response rate to encaleret. one of our design tenets that we're excited about is that we want to elevate the assessment of urine calcium 24-hour urine calcium and the primary endpoint to assess the response rate to encaleret Why we think this is really important is it is an important surrogate for renal health, renal outcomes that really does play a long-term effect in these patients' lives. why we think this is really important is it is an important surrogate for renal health renal outcomes that really does play a long-term effect in these patients' lives It's something that's currently not reflected on labels for replacement hormone and something that we think can not only differentiate encaleret from an evidence perspective, but also in terms of its use, in terms of driving urine calcium down. it's something that's currently not reflected on labels for replacement hormone and something that we think can not only differentiate encaleret from an evidence perspective but also in terms of its use in terms of driving urine calcium down As of today, replacement hormone probably normalizes urine calcium about 60% of participants in their phase III. as of today replacement hormone probably normalizes urine calcium about 60% of participants in their phase iii In our phase II sentinel study, we saw 80% of individuals not only normalize urine calcium but also blood calcium. in our phase ii sentinel study we saw 80% of individuals not only normalize urine calcium but also blood calcium There is an opportunity to demonstrate differentiated benefit on both metrics, and we're excited to evaluate our drug on that basis. There is an opportunity to demonstrate differentiated benefit on both metrics, and we're excited to evaluate our drug on that basis. there is an opportunity to demonstrate differentiated benefit on both metrics and we're excited to evaluate our drug on that basis

Speaker 3: I think in the post-surgical population, if we can replicate or even come close to what we've seen already in phase II, that'd be differentiating alone. It'd be an oral option in what is going to be a very large market where there's definitely a need for multiple types of products. On top of that, I think the non-surgical population is one where we could really be viewed as the best treatment option. There's a big unmet need there, right? These are patients that I don't really know that PTH replacement makes a lot of sense, so basically, untapped market opportunity for us to own. On top of, by then, ADH1 hopefully will be in the early days, but well ramped up and in key part of clinical practice. I think in the post-surgical population, if we can replicate or even come close to what we've seen already in phase II, that'd be differentiating alone. i think in the post-surgical population if we can replicate or even come close to what we've seen already in phase ii that'd be differentiating alone It'd be an oral option in what is going to be a very large market where there's definitely a need for multiple types of products. it'd be an oral option in what is going to be a very large market where there's definitely a need for multiple types of products On top of that, I think the non-surgical population is one where we could really be viewed as the best treatment option. on top of that i think the non-surgical population is one where we could really be viewed as the best treatment option There's a big unmet need there, right? there's a big unmet need there right These are patients that I don't really know that PTH replacement makes a lot of sense, so basically, untapped market opportunity for us to own. these are patients that i don't really know that pth replacement makes a lot of sense so basically untapped market opportunity for us to own On top of, by then, ADH1 hopefully will be in the early days, but well ramped up and in key part of clinical practice. on top of by then adh1 hopefully will be in the early days but well ramped up and in key part of clinical practice

Speaker 2: Okay, great. Moving on to LGMD, which actually could be approved later this year. Before I forget, all three of these programs, my understanding, you get a PRV voucher if they're approved, or? Okay, great. okay great Moving on to LGMD, which actually could be approved later this year. moving on to lgmd which actually could be approved later this year Before I forget, all three of these programs, my understanding, you get a PRV voucher if they're approved, or? before i forget all three of these programs my understanding you get a prv voucher if they're approved or

Speaker 3: We will get one for limb-girdle. We don't expect to get one for encaleret, just given we have a pediatric study ongoing, but its first approval will be in adults. For infigratinib, we may get one as well. We will get one for limb-girdle. we will get one for limb-girdle We don't expect to get one for encaleret, just given we have a pediatric study ongoing, but its first approval will be in adults. we don't expect to get one for encaleret just given we have a pediatric study ongoing but its first approval will be in adults For infigratinib, we may get one as well. for infigratinib we may get one as well

Speaker 2: Okay, great. LGMD2I, again, another strong data set. I think you've mentioned the patient prevalence is around 7,000 in the U.S. and E.U. What % of that 7,000 is actually in the U.S., and by the time you're approved, what kind of line of sight do you have in terms of number of patients you've found? Okay, great. okay great LGMD2I, again, another strong data set. lgmd2i again another strong data set I think you've mentioned the patient prevalence is around 7,000 in the U.S. and E.U. i think you've mentioned the patient prevalence is around 7,000 in the u.s and e.u What % of that 7,000 is actually in the U.S., and by the time you're approved, what kind of line of sight do you have in terms of number of patients you've found? what % of that 7,000 is actually in the u.s and by the time you're approved what kind of line of sight do you have in terms of number of patients you've found

Speaker 3: Yeah, good question. About 7,000 U.S. and Europe. There is a bias towards Europe due to a founder mutation in Northern Europe, but we see about 2,000-3,000 here in the U.S. based on genetic prevalence estimates. Most of those are going to be symptomatic and in need of therapy. It's a muscular dystrophy. It's evident that you have an issue, and actually, it's a really terrible condition where although it's not as rapid deterioration as something like DMD, it is a steady kind of torturous loss of function every day. Huge urgency to treat here, I think, is what we'll see. Yeah, good question. yeah good question About 7,000 U.S. and Europe. about 7,000 u.s and europe There is a bias towards Europe due to a founder mutation in Northern Europe, but we see about 2,000-3,000 here in the U.S. based on genetic prevalence estimates. there is a bias towards europe due to a founder mutation in northern europe but we see about 2,000-3,000 here in the u.s based on genetic prevalence estimates Most of those are going to be symptomatic and in need of therapy. most of those are going to be symptomatic and in need of therapy It's a muscular dystrophy. it's a muscular dystrophy It's evident that you have an issue, and actually, it's a really terrible condition where although it's not as rapid deterioration as something like DMD, it is a steady kind of torturous loss of function every day. it's evident that you have an issue and actually it's a really terrible condition where although it's not as rapid deterioration as something like dmd it is a steady kind of torturous loss of function every day Huge urgency to treat here, I think, is what we'll see. huge urgency to treat here i think is what we'll see I think the thing that we need to do for the next couple of years is drive genetic diagnosis to be at a more complete level. Right now, most of these patients know they have a type of muscular dystrophy. Many of them know they have a limb-girdle muscular dystrophy. Until now, there's not really been a need to. I think the thing that we need to do for the next couple of years is drive genetic diagnosis to be at a more complete level. i think the thing that we need to do for the next couple of years is drive genetic diagnosis to be at a more complete level Right now, most of these patients know they have a type of muscular dystrophy. right now most of these patients know they have a type of muscular dystrophy Many of them know they have a limb-girdle muscular dystrophy. many of them know they have a limb-girdle muscular dystrophy Until now, there's not really been a need to. until now there's not really been a need to

Speaker 2: Genotype- Genotype- genotype-

Speaker 3: Determine that it's a 2I versus any of the many other types of limb-girdle muscular dystrophy. I would say the plus here is fairly concentrated site of care in the U.S. At least there's around 166 MDA centers, where about half the patient receive care. In those centers, testing is much more prevalent already. We need to drive that to be at a more complete level, and we also need to drive testing in other sites of care. Determine that it's a 2I versus any of the many other types of limb-girdle muscular dystrophy. determine that it's a 2i versus any of the many other types of limb-girdle muscular dystrophy I would say the plus here is fairly concentrated site of care in the U.S. i would say the plus here is fairly concentrated site of care in the u.s At least there's around 166 MDA centers, where about half the patient receive care. at least there's around 166 mda centers where about half the patient receive care In those centers, testing is much more prevalent already. in those centers testing is much more prevalent already We need to drive that to be at a more complete level, and we also need to drive testing in other sites of care. we need to drive that to be at a more complete level and we also need to drive testing in other sites of care

Speaker 2: Understood. Here, the patient prevalence seems like it's even more rare than ADH1. Going back to pricing power, is this kind of a DMD exon skipping type of pricing, which is like $1 million? Understood. understood Here, the patient prevalence seems like it's even more rare than ADH1. here the patient prevalence seems like it's even more rare than adh1 Going back to pricing power, is this kind of a DMD exon skipping type of pricing, which is like $1 million? going back to pricing power is this kind of a dmd exon skipping type of pricing which is like $1 million

Speaker 3: I wouldn't put an exact number on it, we see here the bookends are something like Crysvita to the exon skippers. Certainly, that's the right range to think about. I think there's a tremendous value proposition here with this product for the system, for patients certainly, and for providers. If you think about what we've delivered here, I think differentiated amongst all neuromuscular data sets, honestly, where we've seen in a well-controlled study, an amazing effect, consistent effect on all of the important clinical endpoints that we measured. I wouldn't put an exact number on it, we see here the bookends are something like Crysvita to the exon skippers. i wouldn't put an exact number on it we see here the bookends are something like crysvita to the exon skippers Certainly, that's the right range to think about. certainly that's the right range to think about I think there's a tremendous value proposition here with this product for the system, for patients certainly, and for providers. i think there's a tremendous value proposition here with this product for the system for patients certainly and for providers If you think about what we've delivered here, I think differentiated amongst all neuromuscular data sets, honestly, where we've seen in a well-controlled study, an amazing effect, consistent effect on all of the important clinical endpoints that we measured. if you think about what we've delivered here i think differentiated amongst all neuromuscular data sets honestly where we've seen in a well-controlled study an amazing effect consistent effect on all of the important clinical endpoints that we measured Importantly, and I think some people miss this, for patients that randomized to BBP-418 in our study, the mean actually increased across all these measures, or improved across all these measures, meaning the patients are doing better on drug than they were doing off drug, whereas you typically think about in muscular dystrophies, how can I just stop the decline or slow the decline? We actually gave back function, both mobility type measures, lung function. Really amazing outcome. We're hopeful that in our discussions with payers that we'll be able to price this well. Importantly, and I think some people miss this, for patients that randomized to BBP-418 in our study, the mean actually increased across all these measures, or improved across all these measures, meaning the patients are doing better on drug than they were doing off drug, whereas you typically think about in muscular dystrophies, how can I just stop the decline or slow the decline? importantly and i think some people miss this for patients that randomized to bbp-418 in our study the mean actually increased across all these measures or improved across all these measures meaning the patients are doing better on drug than they were doing off drug whereas you typically think about in muscular dystrophies how can i just stop the decline or slow the decline We actually gave back function, both mobility type measures, lung function. we actually gave back function both mobility type measures lung function Really amazing outcome. really amazing outcome We're hopeful that in our discussions with payers that we'll be able to price this well. we're hopeful that in our discussions with payers that we'll be able to price this well

Speaker 2: Right. The same compound is going after 2U, 2M, Fukuyama, I think. Are those next trials going to be phase IIIs, straight to phase IIIs? Right. right The same compound is going after 2U, 2M, Fukuyama, I think. the same compound is going after 2u 2m fukuyama i think Are those next trials going to be phase IIIs, straight to phase IIIs? are those next trials going to be phase iiis straight to phase iiis

Speaker 3: 2U, 2M, as well as expansion into a broader pediatric population will happen starting now. Basically, those will be label-enabling studies. Fukuyama, really interesting Japan-centric opportunity. There's about 2,000 patients in Japan. That will need probably a little bit more work with the regulators to determine the exact registration path. We're working on that right now as well. 2U, 2M, as well as expansion into a broader pediatric population will happen starting now. 2u 2m as well as expansion into a broader pediatric population will happen starting now Basically, those will be label-enabling studies. basically those will be label-enabling studies Fukuyama, really interesting Japan-centric opportunity. fukuyama really interesting japan-centric opportunity There's about 2,000 patients in Japan. there's about 2,000 patients in japan That will need probably a little bit more work with the regulators to determine the exact registration path. that will need probably a little bit more work with the regulators to determine the exact registration path We're working on that right now as well. we're working on that right now as well

Speaker 2: Okay, great. The last couple minutes, achondroplasia. I think, is the NDA filing in Q3 of this year or- Okay, great. okay great The last couple minutes, achondroplasia. the last couple minutes achondroplasia I think, is the NDA filing in Q3 of this year or- i think is the nda filing in q3 of this year or-

Speaker 3: Second half, yeah. Second half, yeah. second half yeah

Speaker 2: Second half. I guess you're unique here relative to the competitors, not only being oral, but with great data, but I think you're the only one at breakthrough designation. Are you expecting a priority review, and is there going to be ADCOM? Second half. second half I guess you're unique here relative to the competitors, not only being oral, but with great data, but I think you're the only one at breakthrough designation. i guess you're unique here relative to the competitors not only being oral but with great data but i think you're the only one at breakthrough designation Are you expecting a priority review, and is there going to be ADCOM? are you expecting a priority review and is there going to be adcom

Speaker 3: I would not expect an AdCom, although we'll know more once we've submitted our NDA and heard from FDA at day 60. We may be eligible for a priority review voucher, that's interesting as well in terms of liquidity. I think you're right. This molecule is very clearly distinct amongst other options for various reasons. One, we know that this market wants a safe, convenient oral option. We've heard that time and time again, going back to the origination of this program at BridgeBio. On top of that, I think we've got the data to say we have a unique proposition in terms of efficacy as well. Primary endpoint result, obviously very strong, importantly, I think we've demonstrated first of its kind data against proportionality, which I think is immediately impactful in terms of daily function for these children, and will yield increasing benefits over time. I would not expect an AdCom, although we'll know more once we've submitted our NDA and heard from FDA at day 60. i would not expect an adcom although we'll know more once we've submitted our nda and heard from fda at day 60 We may be eligible for a priority review voucher, that's interesting as well in terms of liquidity. we may be eligible for a priority review voucher that's interesting as well in terms of liquidity I think you're right. i think you're right This molecule is very clearly distinct amongst other options for various reasons. this molecule is very clearly distinct amongst other options for various reasons One, we know that this market wants a safe, convenient oral option. one we know that this market wants a safe convenient oral option We've heard that time and time again, going back to the origination of this program at BridgeBio. we've heard that time and time again going back to the origination of this program at bridgebio On top of that, I think we've got the data to say we have a unique proposition in terms of efficacy as well. on top of that i think we've got the data to say we have a unique proposition in terms of efficacy as well Primary endpoint result, obviously very strong, importantly, I think we've demonstrated first of its kind data against proportionality, which I think is immediately impactful in terms of daily function for these children, and will yield increasing benefits over time. primary endpoint result obviously very strong importantly i think we've demonstrated first of its kind data against proportionality which i think is immediately impactful in terms of daily function for these children and will yield increasing benefits over time I think importantly, I think that gives hope to the community that this drug actually will be able to finally give something other than just height. We're looking forward to elaborating on that hope and hopefully delivering against it. I think importantly, I think that gives hope to the community that this drug actually will be able to finally give something other than just height. i think importantly i think that gives hope to the community that this drug actually will be able to finally give something other than just height We're looking forward to elaborating on that hope and hopefully delivering against it. we're looking forward to elaborating on that hope and hopefully delivering against it

Speaker 2: Should this be approved and on launch, the low-hanging fruit is it switches from the competitors or naive patients? Should this be approved and on launch, the low-hanging fruit is it switches from the competitors or naive patients? should this be approved and on launch the low-hanging fruit is it switches from the competitors or naive patients

Speaker 3: Yeah. Actually, this is kind of an interesting market where there's an incumbent, but they're dramatically under-penetrated in the U.S. in particular. Our job is to both drive volume off of injectables, because I think there's going to be a lot of demand to get on an oral if you've already gotten on therapy. Also, we have to expand this market, and we know that the desire to start with an oral is certainly a reason that we are seeing under-penetration in the U.S. We really have to drive both of those. Yeah. yeah Actually, this is kind of an interesting market where there's an incumbent, but they're dramatically under-penetrated in the U.S. in particular. actually this is kind of an interesting market where there's an incumbent but they're dramatically under-penetrated in the u.s in particular Our job is to both drive volume off of injectables, because I think there's going to be a lot of demand to get on an oral if you've already gotten on therapy. our job is to both drive volume off of injectables because i think there's going to be a lot of demand to get on an oral if you've already gotten on therapy Also, we have to expand this market, and we know that the desire to start with an oral is certainly a reason that we are seeing under-penetration in the U.S. also we have to expand this market and we know that the desire to start with an oral is certainly a reason that we are seeing under-penetration in the u.s We really have to drive both of those. we really have to drive both of those

Speaker 2: Okay, great. We'll wait on price, but unless you wanted to make. Okay, great. okay great We'll wait on price, but unless you wanted to make. we'll wait on price but unless you wanted to make

Speaker 3: Here, it's a little bit easier, right? There's two comps out there that are, I think Voxzogo is around $400 and change, and the weekly injectable is average around $500. It's easier comps. Here, it's a little bit easier, right? here it's a little bit easier right There's two comps out there that are, I think Voxzogo is around $400 and change, and the weekly injectable is average around $500. there's two comps out there that are i think voxzogo is around $400 and change and the weekly injectable is average around $500 It's easier comps. it's easier comps

Speaker 2: Understood. That's a good guidepost on the low end, in my opinion. Okay. Very good. Well, thank you so much for taking the time. Thanks for all the updates, and thank you everyone for listening. Understood. understood That's a good guidepost on the low end, in my opinion. that's a good guidepost on the low end in my opinion Okay. okay Very good. very good Well, thank you so much for taking the time. well thank you so much for taking the time Thanks for all the updates, and thank you everyone for listening. thanks for all the updates and thank you everyone for listening

Speaker 1: Thanks, Andrew. Thanks, Andrew. thanks andrew

Speaker 3: Thank you, Andrew. Thank you, Andrew. thank you andrew