Bridgebio Pharma (NASDAQ:BBIO) Q2 2026 Earnings Call

0:00 Good afternoon. I will be your conference operator today. All lines have been placed on mute to prevent any background noise. After the company's remarks, there will be a question and answer session. Before we begin, I would like to remind everyone that today's call may contain forward looking statements within the meaning of the federal securities laws, including, but not limited to, statements about BridgeBio's future operating and financial performance, business plans and prospects and strategy. These statements are based on current expectations and assumptions that are subject to risks and uncertainties, which could cause actual results to differ materially from those expressed or implied in these forward looking statements.

0:46 For a discussion of these risks and uncertainties, please refer to the disclosure in today's earnings release and BridgeBio's periodic reports and SEC filings. All statements made here are based on information available to BridgeBio as of today, and the company undertakes no obligation to update any forward looking statements made during this call, except as required by law. With that completed, BridgeBio, you may begin your conference. Good afternoon, everyone, and thank you for joining BridgeBio Pharma's second quarter twenty twenty six earnings call. I'm Chinmay Shukla, senior vice president, strategic finance. With me today are Neil Kumar, our CEO, who'll walk through our commercial pipeline and business updates Matt Outen, our chief commercial officer, who'll provide additional detail on our TruBio and our launch readiness and Tom Tremarkey, our president and CFO, who will review our financial results.

1:41 During today's call, we will cover another quarter of consistent growth for Atruby, along with new data reinforcing its clinical differentiation, including the first evidence of direct kidney protection in ATTR Centimeters. We will then turn to the pipeline, where this quarter, all three of our late stage programs moved from data into being with the FDA,

2:06 with our first PDUFA date now set for November 27. And we will review our financial position, including the billion dollar preferred equity financing completed on July 1 and how it supports the three launches ahead of us. Following our prepared remarks, we will open the call for questions. For the Q and A session, we will be joined by Anant Shreedhar, Christine Hsu, and Justin To, who lead our programs with EncalaRate, DBP-four eighteen, and Infograten, respectively. With that, I'll turn it over to Neil. Thanks, Chinmay, and thanks, everyone, for joining today. As always, these calls are where we communicate relevant aspects of our business to investors, and so we welcome your questions and feedback. In sessions past, we've had occasion to marry comments on the portfolio with comments regarding financing and strategy.

2:58 Today, however, I want to focus entirely on the portfolio itself and the progress being made across research, development and commercial. I'm going to do so because I believe, as I hope you might appreciate at the end of my somewhat lengthy comments, that this is an important transition point for BridgeBio, one in which if we continue executing at a high level, sets us up well for delivering substantial returns for patients and investors alike. Put more simply, it feels like we're at t=zero in BridgeBio's next chapter. I don't say this glibly, but rather due to the following and overlapping advances. First, as we will discuss, the combination of learnings from CardioTransform,

3:37 our own unique kidney protective data and extraordinary real world evidentiary results come together to provide the basis of what I'm calling Launch 2.0 for Atruvi. I believe we will start to see significant commercial fruit from this in the six to nine month range and beyond judging from analogs. We think the market is shaping up to be a stabilizer first market with a constrained number of competitors and one in which we have increasing numbers of proof points that our near complete stabilizer is superior to Pfizer's partial stabilizer. Second, all three NDAs for LGMD2I, ADH1 and achondroplasia have been submitted, with LGMD2I and ADH1 garnering priority review and are hoping that achondroplasia might too. Our commercial readiness work is on track, even ahead of what we were able to do with ATTR cardiomyopathy, given our relatively lean resourcing at the time, to deliver strong launches.

4:33 Third, our chronic hypoparathyroid Phase III, which we believe is overlooked, has commenced and will read out in the next eighteen months, with potential to provide a differentiated efficacy and safety profile, as we will discuss, in addition to being the only oral in the space. Finally, we anticipate novel trials in areas like Turner and hypochondroplasia for ifragatinib, a new trial in a to be disclosed high proteinuria orphan kidney disease for achoramidis and the advancement of a potentially best in class TTR antibody into the clinic in the coming twelve to eighteen months. All of this activity together provides the substrate for well over $10,000,000,000 in risk adjusted revenue, with $8,000,000,000 of that being post Phase III today. In addition, our interest in earlier but still advanced genetic medicine R and D within our gondola pipeline continue to bear fruit, so this is a company with no dearth of pragmatic ideas that can drive a the continued flux of important medicines on a risk adjusted basis for the next decade or more to come.

5:34 I'll begin my portfolio comments with Atruby. First and most importantly, we observed continued commercial momentum this quarter with Atruvi being the fastest growing brand in the space at 23%, and this growth does not account for the impacts of Cardio Transform, our kidney data and most of the real world evidence data to date since that occurred after the quarter end. We've always said that the most important thing commercially and medically in this whole space is diagnosing new patients. To that end, we were heartened to see the substantial overall market growth of 19% this quarter, representing a 51% increase year on year and substantially outstripping the market growth observed in the last three quarters. Consistent with these numbers is the growth in frontline patients where stabilizers have dominated share, a trend that we think will strengthen as we learn more from Cardio Transform's important results.

6:21 Indeed, we observed a slight downtick in numbers of second line patients in the second quarter. We believe our share in frontline has grown some two to three percentage points, although it's hard to tell precisely given some of the inventory dynamics from our competitor Pfizer. Our gross to net also remains within the 30% to 40% that we have indicated previously. Going forward, we expect that the first line market will continue to grow, and we intend to continue growing our share in it, which should translate into continued steady sales growth. Atruby's strongest tailwind, however, is its continually growing clinical differentiation story, driven for the most part by the expanding body of real world evidence as well as the now documented renal protective effect. In July of this year, we published insert heart failure on acrombinis driving the first ever early and sustained direct kidney protective effects in ATTR cardiomyopathy, including chronic eGFR slope improvement and urinary albumin to creatinine ratio reduction.

7:22 The upshot of this is that Etrube may protect the heart and the kidney simultaneously in ATTR patients, a hemodynamically mediated effect which we do not observe with other ATTR cardiomyopathy medicines, either knockdowns or other stabilizers. Critically, as pointed out in the paper, the dynamics of this effect mirror the early separation uniquely observed with Atruby in terms of clinical outcomes, helping to explain this early impact. Furthermore, and intriguingly, the magnitude of the acute dip in eGFR on Atruevi is actually important and suggestive of downstream benefit. More specifically, comparing acaramidis versus placebo subgroups with acute eGFR dips greater than or equal to the median of 4.89 mlm1.73 m2 favored acaramidis for all cause mortality or cardiovascular related hospitalization with a whopping hazard ratio of 0.42 with an associated p value of 0.006, and cardiovascular related hospitalization alone with a similarly impressive hazard ratio of 0.34 with an associated p value of 0.002.

8:36 Intriguingly, within the placebo arm, eGFR dips portended worse outcomes. So something initially thought to be a crutch has now been shown to be an important differentiator for our product. The observed effect compares favorably to what we see in other kidney protective cardiac treatments like SGLT2 inhibitors. In a recently held meeting of nephrologists and cardiologists, one KOL explained to me, It looks like you have a kidney drug here. Building on that, as referred to above, we intend to further interrogate this signal by conducting clinical studies in an orphan kidney indication. More information on that in the weeks to come. Meanwhile, the generation of real world evidence continues apace. When one looks at analogs in the cardiovascular space where double blind head to heads were not immediately possible, real world evidence sets the bedrock of ultimate commercial outperformance.

9:27 The most storied of these analogs is likely the Eliquis Xarelto marketplace. Calling back to last quarter, there was an independent propensity score matched analysis presented at SCAI and since published, which continues to resonate with physicians. That analysis associated Atruvio with a thirty seven percent reduction in composite cardiovascular events and a thirty four percent reduction in hospitalizations at six months relative to tafamidis, with an effect deepening at nine months. Remarkably, there was no observed clinical outcome that did not favor Etrube versus Vindamax in all measures except for dizziness and syncope reached statistical significance of less than zero point zero one with an n just shy of six hundred patients.

10:13 Building on this data, we have our own now soon to be published and available online today preprint analysis that parenthetically has been downloaded more than 400 times now, showing again a Truvia outperformance as compared to Vindamax. Importantly, in this study, a thirty four percent reduction in diuretic intensification, heart failure hospitalization and mortality was observed, again statistically significantly, and separation is again observed as early as thirty days and continues to improve over time. These types of analyses are what the community has been asking for. Importantly, a large scale independent EHR based analysis will be coming at HFSA. Our hope is Atrube continues to perform well there and that then these several RWE studies will form the basis for decision making and guideline updates.

11:04 The growing body of research supporting Attribute's clinical differentiation will take place alongside evidence from other studies in this rapidly evolving field of ATTR cardiomyopathy. Last month, as you all know, the top line results for CardioTransform studying oplontirsen in ATTR cardiomyopathy read out and the study did not meet its primary efficacy endpoint with no benefit observed with combination therapy. At this point, we mostly want to acknowledge that this is a blow to the patients who participated in the trial and their families and the investigators, and we feel for them as part of the ATTR cardiomyopathy community. The case for combination therapy seems today null from a trial data perspective. Given the similar degrees of knockdown between oplontrosyn and vutrisiran, we'll be interested to see how the knockdown performs in two settings: number one, does the monotherapy relative risk reduction continue to underperform what we observed from Etruevia thirty months and two, does monotherapy knockdown actually not outperform a partial stabilizer in Tafamidis as we actually observed in HELIOS B?

12:08 Recall, of course, that in addition to the real world evidence I just cited, everywhere we looked in our ATTRIBUTE trial, Akeramidis outperformed Tafamidis. The conclusions of this important study run by AstraZeneca and Ionis, we believe will likely reinforce the case for stabilizers first. And if the monotherapy benefit again lags in time, as was observed with vutrisiran and in magnitude of effect as compared with Atruvi, we believe this begins to make an even stronger case for using Atruvi first in the second line setting. Now I'd like to discuss the three pipeline programs that have moved into regulatory review this quarter and which we are preparing to launch. For BPP-four eighteen, our LGMD2I program, the FDA accepted our NDA on May 27 with priority review.

12:55 The PDUFA date is 11/27/2026, and there is no advisory committee planned. We continue to have positive interactions with the agency. This is in line to be the next approval in our portfolio, and it will be the first approved therapy for LGMD2I, a devastating condition affecting a little more than one thousand patients in The U. S. Alone with significant unmet need. There's really no displacing credible competition in this space with gene therapy really the only other pipeline approach, and it suffers from safety and efficacy issues coupled with the fact that too much FKRP is toxic, so dosing might well be an issue. I'll remind everyone as well that the data generated by our program are easily the most profound ever in the LGMD space and perhaps the broader muscular dystrophy space, given that biochemical improvements tied strongly to functional and statistically significant improvements in ambulation, breathing, and other outcomes, and that the drug promoted improvements as opposed to the ever worsening observations on placebo.

13:56 From a clinical perspective, our goals are number one, to educate broadly on already established data, and two, to reinforce our observations in the non ambulatory and severe patient population that may initially be reluctant to try anything. Recall, we observed remarkably consistent benefit in our trial across ages, degree of severity, and the homozygous and compound heterozygous populations. Building on that, we'll be analyzing whether our established functional impacts also marry with some cardiovascular benefit, which affects many patients on the severe end of the spectrum. Our plan is to cut that data and present the results at World Muscle Society in late September, early October, so we are hopeful for a good outcome for the patients we serve there. As we prepare for launch, our neuromuscular commercial and medical field teams are hired, trained and in the field. And MarketAxess is engaging with payers in a pre approval information exchange.

14:51 There are approximately five hundred genetically confirmed patients today in The United States, with many who remain unidentified and misclassified within the broader LGMD or bactrimuscular dystrophy space. Our goal is to find every patient who can benefit and be ready the moment we're able to reach them. Turning to ALKALERIT for EH1, the FDA accepted our NDA on July 22 with a PDUFA target action date of 05/08/2027, and no advisory committee planned. At the end of July, the agency granted priority review, and we have announced that today. We have also submitted our MAA to the EMA on July 27, and it is under review. Incalorate would be the first therapy approved for ADH1 in both The United States and EU, and we are excited to serve this patient population.

15:42 Speaking of that population, our patient finding efforts continue, and more than two thousand two hundred patients have been identified in the ICD-ten claims between October 2023 and June 2026. That is an increase of about three hundred since the first quarter, and it's been driven by genetic testing, awareness education, use of the ICD-ten code, and BridgeBio supported family testing events. We have also completed enrollment in the first of four cohorts in our pediatric ADH-one study and are preparing to open cohort two. But ADH-one approval is the beginning of the Caloran's potential, not the end. Chronic hyperparathyroidism affects some two hundred thousand patients in The U. And EU, a blockbuster opportunity in and of itself, where as discussed last quarter, we see a real appetite for an oral option that corrects both hypocalcemia and hypercalciuria.

16:34 I want to spend a minute on this opportunity because I think it's been overlooked significantly by investors. First, there may be a belief that PTH replacement is the beginning and end of the game here, with advances around dosing, for instance, going from daily to weekly, being the only salient dynamic for patients. But that overlooks a couple of key facts. First, the benefits of existing therapy do not importantly extend to normalization of urine calcium, with some 40% of patients not normalizing and some fifty percent of CHP patients actually being hypercalcuretic. Two, there's a well documented decrease in efficacy of PTH replacement over time, suggesting that other approaches may be important here. Third, perhaps most importantly, there is a need for a drug that spares the impact of PTH mediated bone issues, especially considering that in a recent survey of one hundred and sixty patients, forty eight percent of them had osteoporosis or osteopenia.

17:30 And fourth, that many individuals would prefer an oral medicine. I think some may have discounted this opportunity based on likely probability of technical success. That I believe is a mistake. First, the pathomechanism here is well described. Recall first that the hypocalcuria in chronic HP arises from three independent contributors: one, loss of calcium reabsorption at the distal nephron, that's PTH driven. Second, decreased calcium reabsorption in the thick ascending limb, that's calcium sensing receptor driven. And third, obviously exacerbation by conventional therapy. Analogous to PTH activity in the kidney to mediate reabsorption of calcium, Incalorate's action on the calcium sensing receptor has been shown to increase paracellular reabsorption of calcium in the thick ascending limb by reducing Claudine 14 expression, which in turn decreases the amount that integrates into the Claudine sixteen nineteen complex, which acts as a calciuria promoting pore blocking component.

18:32 This mechanistic rationale helps to explain the observation from our proof of concept Phase II, where eighty percent of postsurgical hyperthyroid patients administered within CALARITE achieved both normal blood and urine calcium within five days. Okay, so we understand how negative allosteric modulation of the calcium shunting receptor can mechanistically raise serum and lower urine calcium, even in a wild type setting. But for those of you who don't want to bet on mechanism, recall also there's clinical evidence in the wild type setting that exists for these drugs, namely the extensive data from the legacy clinical development program of CALLAY in osteoporosis participants expressing wild type calcium sensitive receptor, like the chronic hyperparathyroidism population that we intend to study in the RECLAIM HP trial.

19:20 And recall that in that osteoporosis study, the drug demonstrated dose proportional increases in serum calcium at daily doses of fifteen milligrams or above. So we believe, given the endpoints of serum and urine calcium normalization, with all that we've seen and know and the stability of those endpoints statistically, that we have a high probability of technical success trial on our hands. Secondly, investors may believe that the opportunity is not near term, but this is a relatively quick trial given the aforementioned endpoints and the rapidity of onset of our drug. And as mentioned in our press release, we have already activated our first sites for the RECLAIM trial, our global Phase III, and have begun screening with FPI imminent and a trial readout expected in the next eighteen months.

20:09 Okay. Finally, I'll come to ifragatinib, our oral treatment for achondroplasia, where we presented our Phase III PROPEL III results at the International Conference on Children's Bone Health on June 30, and simultaneously published them in the New England Journal of Medicine, the only achondroplasia program with Phase III results in the New England Journal. Following that publication, I'm excited to announce we submitted our NDA, and we are targeting an M and A submission in Q4 of this year. We hope to see NDA acceptance and ideally priority review in Q4 of twenty twenty six, with approval following in mid-twenty twenty seven. Approval would make infogrutinib the first FGFR3 targeted oral therapeutic for achondroplasia. And on top of its oral dosing advantage, it remains the only therapy with efficacy measures beyond annualized high velocity demonstrated in a placebo controlled setting at fifty two weeks, including proportionality.

21:04 Adding to this, we demonstrated a clean functional differentiator in our Phase III results with a statistically significant 0.37 standard deviation improvement on arm span with a p value of less than 0.0001. This is the first ever placebo controlled arm span benefit in an achondroplasia trial. We look forward to presenting more data in the second half of this year and continuing to build Imfibratinib's scientific story through the pre approval period. On the commercial front, our regional sales directors and medical affairs personnel are on boarded and the field medical team is fully built. Our RSDs are building teams for meaningful share of voice in a market where two competitors are already present and where we see a real gap, especially in The U. S, between kids confirmed to have achondroplasia and those on treatment.

21:56 We continue to think our peak achievable share in this space is above sixty five percent. Finally, I also want to make mention of the critical work occurring off our balance sheet at Gondola Bio, where BridgeBio shareholders retain exposure via our ownership stake and ongoing operational support. Our program in EPP announced positive Phase 2a data in June, and following a productive EOP2 meeting with the agency, we will be initiating a Phase 2bthree study in Q3 of this year. Critically, given the 80 plus percent magnitude of PP9 reduction coupled with the quick onset of action and SAFE profile, the agency suggested that the IIb could form the basis of registration if PP9 lowering was met statistically and other functional trends lined up with it from the point estimate standpoint.

22:44 Meanwhile, the rest of the pipeline continues to progress with some 17 programs and indications including ADPKD, alpha-one antitrypsin, neurofibromatosis type one, and CMT1A. In total, the activity has potential to yield five additional INDs by the end of this year, with some eight clinical proof of concept readouts to come in the twenty twenty seven-twenty twenty eight timeframe. Of course, despite all of this, we continue to stay focused on delivering our important medicines to patients in the commercial setting. And for more information on that, I'll pass it over to Matt. Thanks, Neil. Q2 was another strong quarter that demonstrated consistent growth in the treatment naive segment for as physicians are increasingly starting and keeping patients on Atruvio.

23:31 Net product revenue was $222,400,000 marking another quarter of $35,000,000 or more of sequential sales increase. I want to spend a moment on the composition of that growth, because that is the part that matters most for how we think about the franchise from here. The engine is the first line. Our first line share stepped up again in Q2 on a first line market that held roughly steady quarter over quarter, and new patient starts were consistent with the first quarter. That is the durable driver of this franchise, and it is what we are building against.

24:13 The second line, or switch segment, is behaving differently, and I want to be clear about it. The forced vindicule switching that inflated that pool in the fourth and first quarters has now largely been worked through. At roughly eighteen months post launch, the switch opportunity is settling into a lower and more normalized steady state. What changed there is the size of the pool, not our performance within it. So the shape of our growth is evolving. Continued first line strength partially offset by a smaller switch market. That is the mix we would expect going forward, and it is the mix we are planning around. Neil covered the clinical differentiation data, so I want to speak to what it is doing commercially, because this was a meaningful quarter on that front.

25:05 The endpoints Neil walked through are the ones practicing cardiologists manage week to week, such as hospitalizations, diuretic escalation, and kidney function. And because much of that work was conducted independently of us, it carries the credibility with physicians and with payers that sponsor generated data does not. We expect additional independent, real world work to read out over the balance of the year. On Cardio Transform, the outcome was disappointing for patients who had hoped combination therapy would improve on stabilizer monotherapy. What it did do is reinforce stabilization as the first line standard of care. As the only near complete stabilizer available, we believe Atruvi is well positioned in that setting.

25:54 That said, the first line remains competitive, and we expect it to stay that way. Our job is to keep earning share on the strength of the data quarter by quarter. Neil noted last quarter that we expected Acaramatis to reach blockbuster status in 2026, and we remain on track for that. To be precise about what sits inside of that number, we are referring to worldwide sales of Acaramatis, which includes BBIONTRA sales recorded by our partners outside of The United States. It is not a forecast for The US Atruvi net product revenue. For the balance of my time, I want to focus on the three approvals ahead of us. The Yatruvi launch gave us much of the infrastructure any future launch requires, and we have been hard at work making sure each of these goes as well as that one did.

26:45 These would be the fourth, fifth, and sixth launches in BridgeBio's history. Let me take them in expected order of approval. First, BVP-four18. LGMD2IR9 has never had an approved therapy. Approval would mark the first for LGMD2IR9 and the first for any form of limb girdle muscular dystrophy. We have submitted a brand name and have conditional acceptance of a proposed proprietary name from the FDA, which we will announce at approval. Our field medical team, sales leadership, and sales team are hired and in field. More than ninety five percent of the sales team has prior neurology experience, with an average of nine years in rare disease.

27:33 These patients are diagnosed and managed by neurologists and neuromuscular specialists working with a multidisciplinary team, so our target universe is concentrated, roughly 700 institutions and 5,300 target specialists, with priority reach against approximately 150 parent MDA centers. Ahead of any approval, the team is focused on disease state education and genetic testing awareness, and we continue to build a scalable patient identification engine that has already identified eligible patients. We are also engaged with payers through preapproval information exchange so they understand the value story ahead of the decision, and we will bring the same patient support programs that have supported our prior launches.

28:21 Second, Incalirate in ADH-one. At the end of July, the FDA granted priority review for Incalirate. The PDUFA target action date is 05/08/2006, and no advisory committee meetings currently planned. We have built an equally strong field team here, with nearly ninety percent bringing rare disease experience. ADH1 is a genetically distinct condition driven by gain of function mutations in the calcium sensor receptor, which causes low serum calcium, low or inappropriately normal PTH, and a more pronounced increase in urine calcium than hypoparathyroidism generally. Incalorate is designed to target that receptor directly, with the potential to address both serum and urine calcium.

29:08 If approved, it would be the first therapy specifically indicated for adult and adolescent patients with ADH1. As with BVP-four eighteen, we are engaged early with payers so that the clinical rationale is well understood before a decision. Third, infigratinib and achondroplasia. We have submitted the NDA, and we anticipate approval in mid-twenty twenty seven. Unlike the other two launches, infigratinib enters the market where competitors are already established. We have delivered against that kind of setup before. What we hear consistently from families, from our HCP and community steering committees, and from market research is that there is real anticipation for an oral option. And awareness of infrogonib is high.

29:55 The ability to give this medicine as a small, once daily capsule is about considerably more than convenience. Aversion to injections is one of the primary barriers keeping families from starting treatment at all, one of the leading reasons they discontinue, and a persistent burden on daily routines and family dynamics. Infogratinib can be swallowed or the capsule can be twisted open and sprinkled over food. No refrigeration, no reconstitution, no working out how to travel with it, no injection site reactions, and no shots. Families and physicians also see the differentiation as more than the capsule. They consistently point to the efficacy in the PROPEL three program and in particular, the proportionality data in the pre specified three to eight year old subgroup.

30:45 Operationally, our commercial infrastructure continues to build, and we are being deliberate here because this community is unique and requires a different kind of support when families are weighing whether to start therapy. Our partnership with the achondroplasia community over the past seven years is underpinning how we are approaching this launch. In short, we are on track across all three programs. With that, I'll turn the call over to Tom. Thank you, Matt. Good afternoon, everyone. I'll now walk through our financial results for the second quarter of twenty twenty six. Our commentary will focus on GAAP financials unless otherwise noted. Total revenues for the second quarter of twenty twenty six were $243,700,000 compared to $110,600,000 for the same period in 2025.

31:31 The $133,100,000 increase was primarily driven by a $150,900,000 increase in Attribi net product revenue. Attribi net product revenue in the quarter was $222,400,000 compared to $71,500,000 in the same period last year. Royalty revenue increased to $15,400,000 compared to $1,600,000 in the same period last year, primarily earned from net product sales of BEYONDRA in The EU and Japan. License and services revenue was $5,800,000 compared to $37,400,000 in the same period last year, which included a one time $30,000,000 regulatory milestone recognized under the Alexion agreement following pricing approval in Japan.

32:15 Total operating expenses for the second quarter of twenty twenty six were $335,700,000 compared to $241,200,000 for the same period last year. A $94,500,000 increase reflects deliberate and disciplined investment in Atruby and preparations for three upcoming launches. It was primarily driven by scale up of sales, marketing, medical affairs, and pre commercial product supply related activities. Turning to the operating line. In the second quarter, we recorded a $107,100,000 loss from operations compared to a $134,300,000 loss in the same period last year, an improvement of $27,200,000 or approximately 20% year over year. Now on to the balance sheet. As of 06/30/2026, our cash, cash equivalents and marketable securities were $720,200,000 Subsequent to the quarter end, on 07/01/2026, we closed a $1,000,000,000 preferred equity investment led by sixth Street, with participation from Healthcare Royalty, putting our cash balance at approximately $1,700,000,000 as of 07/01/2026.

33:23 We believe our current cash position provides us with a significant runway to fund our operating activities, execute on three potential launches over the next twelve months, and continue to invest in Atruvio's commercial growth, all while maintaining the financial discipline we have demonstrated to date. With that, I'll turn the call back over to Chinwei. Thank you, Neil, Matt, and Tom. Operator, please open the line for questions now. Thank you. We will now begin the question and answer session. We'll go first to Tyler Van Buren at TD Cowen.

34:12 Hey guys, good evening and congratulations on another strong quarter. It's great to see the more than $35,000,000 in sequential U. S. Revenue that Atruvi added again this quarter. But as the release specifically calls out Atrube growth led by the treatment naive segment as physicians increasingly start and keep patients on Atrube, can you discuss what is driving that consistency in the first line?

34:34 And perhaps most importantly, given competitive developments, why those drivers are durable. Perhaps you could also layer that in with expectations for the potential impact that the cardio transform failure and upcoming data ESC could have on a TrueBio's treatment naive share as well. Thanks, Tyler. I'm going to pass it on to Matt to comment on some of the commercial dynamics. And then I'll pass it on to Neil if he wants to add things on cardio transform expectations at ESC. Okay. Thanks for the question, Tyler. I think there's two interesting components here. There's the reason that Atruvio has done so well to date, namely how quickly Atruvio separates from placebo along with the incredible reduction in hospitalization rates.

35:19 And then there's the new data that Neil discussed today. The performance you've seen to date has been rooted in the clinical differentiation story. Now we can add to that with compelling insights from the real world evidence, kidney data, and Cardio Transform. And this is going to add on to the earlier messaging and continue to push share forward in the future. And I'll let Neil add on with the CardioTransform folks. Yeah, thanks, Tyler. I guess I'd just say, we have to see what the data looks like. But by and large, I would expect that Stabilizer Frontline will do nothing but gain from the cardio transformed data set. So just be a

35:55 larger pool. And in that pool, I think, to Matt's point, we'll continue to differentiate. And I think we're gonna see the the fruit, as I as I mentioned in my comments, of the real world evidence, kidney differentiation, appear really by kinda like half a year, from now or so. If you if you look at analogs, it generally takes about six to nine months to pull through, some of this data. Obviously, also dependent on on what HFSA looks like in terms of the independent RWE analysis. But if everything continues to to go the way of a true be I mean, you know, as you well know, it's sort of like

36:29 as you start to connect all the dots from biochemistry to serum TTR, I mean, big per deciliter is a five percent decrease in mortality risk in thirty months to all of the real world evidence against both survival. At least we'll see that at HFSA. And we saw some hints of that with the MOZRE and independent more data around the time that we launched, and hospitalization and ODI, as we mentioned today. I think all of

36:57 that comes together to say we have a superior stabilizer, and that's really the message we we gotta continue to hit. I mean, my expectation would be that we really hit a a positive second derivative here and continue to grow pretty aggressively in the frontline over the coming twelve to eighteen months, but let's see. We'll go next to Corey Kazimov at Evercore ISI. Hey. Good afternoon. Thanks for taking my question. Perhaps not surprisingly, I also want to ask a question regarding cardio transform, missing the primary endpoint. So, you know, at this point, we obviously know there was substantial background stabilizer use and putting the silencer on top of it didn't improve outcomes. So I know you touched on on some of this in your prepared remarks, but in your view, does this not only cement stabilizers as kind of the first line backbone here in future treatments?

37:52 But also, do you have any feedback at this point your KOLs and payer discussions as to how prescribing and reimbursement of any combination therapy may evolve from here? Thank you.

38:05 Yeah. Thanks for the question. Maybe I'll start, and, Matt, you can add add on. You know, I'd say it's a little early for us to to get feedback from payers. On the KOL side, for sure, we've been hearing, I think, a bit of surprise, honestly. There are folks that can be convinced with biochemistry and biophysics, but I think a large trial like this convinces a lot of folks and might be changing folks' minds. So I do think stabilizer will be an increasingly large part. They already are a large part, but an increasingly large part of the front line. And I think that's where the real action will be in this category. I'd say the three things that we're looking for with regard to cardio transform, I do think oplonderson and Vud have a very similar knockdown profile. We have to look at pharmacokinetics and see whether aplondrocin is slightly superior to Boot, because Boot obviously took a long time to get to its mean max knockdown. But that will be the first thing that will be intriguing to look at.

39:04 And then within the context of the clinical data, first and foremost, what's the thirty month data look like? Is anyone getting to three forty, two fifty? And to Matt's point, how quickly are folks separating in terms of effect? Because I think if you look at the totality of evidence, my suspicion will be that not only do you get the magnitude of relative risk reduction that basically Atruvio will look superior at thirty months, but if there's no early separation, it really starts to suggest that you ought to be using ATRUVY in that switch setting, just given both its magnitude of benefit and the early onset now well described by this kidney data that

39:45 we put out and we'll continue to elaborate on. I think the second super intriguing point will be to see whether or not monotherapy knockdown actually outperforms a partial stabilizer. People sometimes I know you and I chatted about this, but people sometimes forget that in Helios Phe and that would tell us that all Jack Baker that Boo didn't significantly outperform TAP, which was a bit of a head scratcher to me based on the toxic monomer hypothesis until you look at the pharmacokinetics. Then here again, if an opt out doesn't outperform a partial stabilizer, recall we've had a stabilizer that outperformed TAP in every single part of the ATTRIVUE trial that we looked at in all major RWE studies. And so again, it starts to establish, I think, a true view as a superior efficacious agent as compared to both knockdowns and the partial stabilizer of Pfizer. So this will be the second big thing we're looking for.

40:41 Jonathan, anything else you'd add? No. That's well said. I think we're interested in seeing the full data set at ESC. But certainly, the results don't appear to support combination therapy, which just then reinforces stabilization as the backbone of therapy. And again, your comments, I think, on the partial stabilizer versus a near complete stabilizer, that's kind of where we are. And I don't think anything we see at ESD is going to change that based on the initial results that were posted.

41:11 We'll go next to Ally Merle at Barclays. Guys. Thanks for taking the question, and congrats on all the progress. So the Pfizer release cited net price erosion from new payer contracts, while your gross to net has remained stable within the range you guided to. Given it's really launched at a list price below tafamidis, do you see any need to respond on price? Or is clinical differentiation carrying access and share on its own? Thanks. Yeah, thanks, Ali. It's an important question. I think we'd like clinical differentiation to continue to carry the day here. There's no way that we could respond and meet Pfizer's rebates if they're going to be aggressive in that channel, Nor do I think we need to. I think we've had productive discussions with our partners all all the way through, basically, the channel.

42:02 They understand what we're trying to accomplish in terms of clinical differentiation, in terms of the added reduction in hospitalizations. Here's where the real world evidence really comes in handy. Mean, a thirty five percent or thirty four percent in an independent study reduction in hospitalizations as compared to TAP, that's super meaningful. These are patients that are quite sick, quite expensive. And there's some untoward things that can also happen when you favor one brand over the other. So I think long term, these brands will be a parity, generally, in terms of access. And then I think clinical differentiation will be where we win. So we do not intend to chase anyone down the rabbit hole of trying to play near term price dynamic games. Our next question comes from Salim Syed at Mizuho.

42:50 Great. Congrats on the quarter, guys, and thanks for the question. Just one from us on this heart failure publication data on the kidney protection. So obviously, the better stabilizer, everybody knows that all the real world curves show that also attruby is better than TAF. Just wondering how this adds into sort of that thinking here. Like when you guys are talking to physicians, how important is this kidney protection? How meaningful is it in terms of how they're prescribing a stabilizer or choosing a stabilizer? And if we kind of like drag that forward a little bit, with the list price already being below TAF, what does this eventually mean for the true B as the market evolves and when tafamidis goes generic? Thanks so much.

43:38 Yeah. Salim, thanks for the question. I'm going to let Matt handle all the kidney data that's being received by KOLs, and then I'll also go back on the generic question. But Matt, why don't you talk about differentiation and the kidney data? Yeah. I mean, I think first thing to note, this is new. So up to this point, it's been about the 340,002 and $50,000 as Neil mentioned. It's about early separation and not only how fast a TruBio works, but how well it works, how many people it keeps out of the hospital, how soon you see the curves separate. So that's what's led us through Q2. I think in terms of the kidney data, it's very important to physicians. And you're going to see that impact moving forward, which I think is that to me is probably one of the most exciting things about the call today because the kidney data hasn't been out. It's brand new. So you're going to see that impact now as we move forward over the next couple of quarters.

44:35 Yeah. And just to build on that, I know we've discussed this, so I'll be brief on this. But we do expect the brand to keep growing even after Winamax goes to genetics in mid-twenty twenty one in The US. And really, there are five reasons for it, right? I think the first is, Atruvius clinically differentiated, you've heard a lot about that on the call today, that is driving the strength and treatment naive for us, and I think it's going to keep driving strength there. The second, which I think is less understood by folks, is that stakeholder economics in this market, especially DSPs, they don't largely support a preference for generics. I think you can also see that Pfizer has been successful in defining other franchises. And I think that there's a potential for some upside because if if Pfizer stops promoting post ROE, that would increase relative share of voice for a Truvy. So I think if you look at all of this and you look at all the analysis on analog, which I know you and and and Bennett have have done a detail on that, I think that we expect that even as opposed to market, less potent stabilizers goes generic, a near complete stabilizer, our true view is gonna keep growing.

45:43 So can I just build on one point that Matt made? Because I think the kidney data is super fresh. So we're gonna have to see in the next six to nine months how it changes prescribing behavior. But first and foremost, I think it's important because the actual mechanism of turning down toxic monomer, you wouldn't expect to pick up impact as early as twenty eight days or or one month. So here now, you have a viable mechanism by which you have this early onset of efficacy. And as I mentioned in my remarks, I hinted at, it was previously considered a harmful piece of our label. But I think now what you see is the greater that diminution early, the better off you are later in terms of cardiovascular hospitalization and death. So that's also a profound suggestion here that I think will be very important on a go forward basis. And you've got to remember, patients, close to fifty percent of patients who they see on cardiomyopathy have some sort of kidney involvement. So this is an important this protective signature is going to be an important piece, we believe, of the emerging story here, and potentially an interesting piece of the story if we can move Atruvio into novel indications.

46:58 We'll move to our next question from Andrew Sy at Jefferies. Hey, congrats on the solid execution. Thanks for taking my question. So I think this was a quarter where all three of your pipeline programs moved from the clinic into the regulatory phases. You got LGMD submitted within five months of the top line, two prior reviews, no AdCom's planned. So it seems like your relationship with the FDA is quite healthy, but maybe talk to us in detail what your regulatory engagement has been like and how you're feeling about the review timelines from here. I'd also be curious about your ex US interactions too. Thank you. Sure. Happy to take that. I mean, think, first and foremost, as you probably know, in the rare disease setting, kind of the gold standard is the ability to run an RCT, a solid RCT with a placebo arm. And we've been able to do that across all three indications here and demonstrate profound functional benefits.

48:00 I think one of the senior administrators at the agency once said that we're kind of the poster child of what one tries to do, at least in the rare disease setting. It's not obviously always going to be the case. For instance, in Canavan disease, we may not be able to run an analogous trial. But certainly for these three data sets with the p values where they are, with the safety where it is, I mean, people forget that with these small molecules, we've been able to provide an exquisite safety profile. So the risk benefit is pretty straightforward as well. So, yeah, based on all of that, we've we've had productive discussions with the agency to date, and, you know, we look forward to getting to engage them on that front. And similarly, I would say, in Europe as well, there hasn't been a a dichotomy between the tenor of our conversations there yet.

48:49 Our next question comes from Anupam Rama at JPM. Hey, guys. Thanks so much for taking the question. I'm just thinking a little bit about the November 27 PDUFA for four eighteen limb girdle muscular dystrophy. Sounds like you guys have made a lot of progress here on the field team, the neuromuscular field team being hired, trained, deployed. Can you walk us through what the near term focus here to be ready on your launch readiness? And then how you're going about identifying more patients heading into PDUFA to go beyond that, I think, 500 patients you talked about being identified today? Thanks so much. Time's milk. We're going to pass on to Christine because alcohol doesn't go to launch.

49:35 Hi, Advan. Just as a reminder, this is an opportunity where we think it's a $1,000,000,000 peak sales opportunity. We think there's 7,000 patients in The US and EU, with 2,000 to 3,000 in The US. In The US, in terms of launch readiness, we do benefit from having a concentrated prescriber base, with the majority of patients treated at about 150 NDA centers. So as we mentioned, we do have a dedicated sales force that's been fully hired and trained. They're in the field now, really focused on disease awareness and site profiling before the launch. Our MSLs are also fully trained. They've been in the field for over a month. They're also focused on disease awareness and increasing the awareness of genetic testing, and that's been key for driving increasing patient ID and genetic testing.

50:26 On the patient side of things, we have identified the over fifteen hundred patients who are genetically confirmed. That's actually grown over the course of the year, and we would expect it to actually continue growing. We have seen that genetic testing rates have also increased over the past nine months, and that key to increasing the number of patients that are identified, including the fact that we now have dedicated sales force as well as MSLs in the field driving awareness. There's also a new dedicated ICD-ten code specific for LGMD2IR9, and that's also going to help with tracking patients and just greater visibility as we commercialize BCP418. On the payer side of things, this is an area of strength for this launch where we can really maximize access and price.

51:19 The market research with payers has been consistently positive. They've been quite receptive to the strength of our data and the unmet need on the patient side. They view the closest priced analog as the exon skipping D and D drugs as a comparable patient population for them. And I guess the ones having here, they even acknowledge that we have much stronger data because we actually have the functional data. It's not just based on biomarkers. And so that's an area of strength for us at this launch. We'll go next to John Boyle at William Blair. Hi, team. Congrats on the strong quarter, and thanks for taking our question. So I wanted to ask on Incalarette.

52:06 Now that you have priority review, the MAA is submitted and diagnoses are increasing with each month with the ICD 10 code. Wondering if you could walk us through the launch setup into the May 2027 PDUFA date. And as a follow-up with RECLAIM HP now screening, hoping you could walk us through how you view the size of that opportunity and how you're viewing it as the next leg of growth for the franchise. Thanks. Thanks, John. Really appreciate your questions. I'm gonna pass it on to Anant to talk about EncalaRate. Sure. John, thanks for the great question. The setup in advance of our PDUFA date for IMPALAYRID and ADH1, as we shared today, we see about over 2,200 patients uniquely coded under the dedicated ICD-ten code, which is E20.810 for autosomal dominant hypocalcemia.

53:01 What we see is about seventy patients per month have been diagnosed and coded according to that code in the claims databases. And it's suggestive of what we would have anticipated, which is the availability of promising and positive clinical data driving awareness and suspicion to test for ADH1 in the clinic. And between now and PDUFA, as one might expect, we're investing further in raising disease data awareness. And we have our medical team meeting with institutions and providers, amplifying disease state awareness efforts, and growing familiarity with our evidence. And between now and PDUFA as well, we will continue to engage with our payer audience.

53:52 To date, the interactions have been quite positive. The anticipation for a new and first modality directly targeted to treat ADH1 has been quite well received amongst the payer audience, and we anticipate constructive dialogue as we approach the PDUFA more closely. And then to your second question regarding RECLAIM, it's a really exciting update today as we shared that screening activities have started for that Phase III study. We anticipate to deliver top line results from that study in about eighteen months or so. And it might be a great opportunity for us to grow the clinical utility of Calorit into the broader chronic hypoparathyroid population. We see around two hundred thousand individuals in The US and Europe to be afflicted with chronic hypoparathyroidism.

54:43 If we're successful in this indication, we see another blockbuster opportunity for us to grow into. We'll move to our next question from Derek Archila at Wells Fargo. Hey, good afternoon. Thanks for taking the questions. Just a quick one. So I know in the past you had mentioned, like, 30% to 40% peak share for a true be assumed a four player market with combo use expanding. So I guess, how does the failure of Cardio Transform raise that feeling? And just curious if you plan to update that assumption anytime soon. Thanks. Yeah. Thank you for the question. We're conducting market research, and I think that we'll probably kick it off more after the Cardio Transform results come out more fully at ESC. And so at that point, we can more formally talk about what we expect to speak share.

55:33 I think as Neil mentioned in his prepared remarks, we do think that the case for combo therapy scientifically is quite dead now, and so I think that that does benefit, and I think the stabilizer should also remain time as we've discussed. So we expect those things to be positive, but we don't have new market research to share at this point. It would be a bit preliminary to do it before the medical conference has happened and physicians have had a chance to digest all of it. Really appreciate your question. Our next question comes from Luca Izzi at RBC. Oh, great. Yeah. Thanks so much for taking my question. Congrats on the progress. Maybe on achondroplasia, you know, obviously, BioMarin last week mentioned that a hundred patients have switched from Bugzogo to UBioL or less than ten percent of all the Bugzogo patients. You know, they're obviously arguing that ten percent such a low number to suggest that the market is very sticky and the patients are loyal to Bugzogo.

56:30 And, you So just wondering what's your comment on that? What's your view on that number as we think about the launch of Imprepnu potentially next year? Thanks so much. Thanks, Luca. Appreciate the question. I'm going to pass it on to Justin to talk about the Empaglutin program. Yeah. Thanks so much for the question. Yeah. I think we've been really pleased with what we've heard the last few weeks from both BBIORA and Zendesk. I think there's a lot of favorable tailwinds for our upcoming launch. You know, on the BBIORAN side of things, they continue to increase the treatment rate and build the market globally, really enlarging the pie for everyone across all markets. Recall it's easier to get switch than to get a patient who had never been on treatment before. I think that's been really great to see their launch continue to accelerate there. Now based on the recent Ascendis numbers, it really validates two of our key assumptions for launch. The first is that there's really not that much brand stickiness in this space.

57:21 Families want their kids to switch to Motion and Be an option. And when we're on the market, not only will we have the Motion and an option, but by far the most efficacious. And so Ascendis having a strong launch here is good for us if families and agencies think about switch and think about new options. And ever since Ascendis has approved, we've noticed a huge uptick in outreach from the agency. Now the second key assumption that Ascendis' launch validates is that having a more convenient option will expand the market. I think Ascendis is seeing a good chunk of their treatment naive scripts from families who never went on boxed over or you haven't just used some of the math based on environment and Ascendis' remarks. And we know from multiple analogs from prior launches that the availability of the first oral tends to expand the market by two to three times. So we think in totality, some of the numbers we're seeing for both Byron and Ascended in their remarks is going to be pretend well for our launch.

58:14 Next, we'll go to Jason Zemanski at Bank of America. Good afternoon. Congrats on the nice quarter, and thanks for squeezing us in. BEYONDRA royalties just reached $15,000,000 for the quarter. It looked like they're starting to scale quickly. So as in CALILRET for 2018 and now in filgotinib moved towards their respective European decisions, How are you weighing potential partner strip structures like the BEYONDRA agreement versus commercializing independently ex US? And I mean, is there anything you can extrapolate from your experiences about maximizing value abroad? Thanks. Hey, Jason. It's great to hear from you, and thank you for the question. Our framework for any partnership decision always remains we want to do what is going to be best for patients and shareholders alike, And we want to put the asset in the hands of the person that that is the best owner.

59:08 I think that, we think for these next three launches, we feel very confident about being able to commercialize them globally on our own. I think we have learned a lot from the Atruby launch, and I think we're excited to grow our footprint internationally. Because I think actually serving those countries and KOLs is going to help us improve our drug drug development engine too. So that's how we are thinking about it. Obviously, with the fact that we have about 1,700,000,000 of cash on our balance sheet, we're very well capitalized to to fund those launches. And and I think that the footprint is also going to be light as as as we've discussed before. So I think that's how we are thinking about it today. But, you know, we're always open to interesting suggestions and ideas, and and all we always evaluate is what is best for our shareholders and and patients that we wish to serve.

59:58 Well, also, it's important to your control price globally in an MFN world, so that's what we intend to do. And next, we'll move to Danielle Brill at Truist Securities. Hi, guys. Good afternoon. Thanks for the question, and congrats on the really strong execution this quarter. So it looks like operating loss improved roughly 20% year over year despite the added investment required to support potentially three new launches over the next twelve months. So as a TrueBio continues to scale and the portfolio transitions to a multiproduct commercial business, how should investors think about incremental margins and operating leverage from here? What are the key milestones that ultimately drive Bridge to profitability and sustainable cash flow generation?

1:00:49 Thank you. Hey, Danielle. Thanks for the question. So I would say with another quarter behind us, we are increasingly confident in the evolution of the P and L towards a point where we'll start to see breakeven profitability and ultimately cash generation in relative near term. Just to give you a sense for how we think of this. So we look year on year, we're seeing an improvement on the operating line, which has been pretty consistent year on year for the last few quarters. Quarter on quarter though, we're pretty much stable. We expect to be stable on the operating lines in the next several quarters before that starts to improve again towards the end the year into next year. To break that down a bit further, you've got two pieces really driving this. One is a true view which is in basically I would say margin expansion mode where OpEx is relatively stable, but we're seeing obviously sales growth continue to improve the margin.

1:01:40 That's pretty much offsetting the investment we're making into the upcoming launches. So we're scaling up all the activities around field medical and marketing, as well as expensing pre commercial inventory right now. So as we get towards steady state on those activities towards the end of next year, we'll start to see, again, a trend toward improving the operating line and ultimate breakeven on the horizon as we move into 2027. And that concludes our q and a session. I will now turn the conference back over to Chin Mai for closing remarks. Thank you, everyone, for joining us for our second quarter earnings call today. We appreciate your interest and we look forward to seeing many of you at our commercial day in New York on October 8, where we will go deeper on commercial readiness and launch strategy across our three upcoming launches.

1:02:35 Thank you. And this concludes today's conference call. Thank you for your participation. You may now disconnect.