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Presentation

BridgeBio Pharma, Inc. misses on earnings expectations. Reported EPS is $-0.84 EPS, expectations were $-0.7.

Operator: 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. And if you would like to withdraw your question, press 1 again. Thank you. 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 law, including but not limited to statements about BridgeBio Pharma, Inc.’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 risk and uncertainties, which could cause actual results to differ materially from those expressed or implied in these forward looking statements.

For a discussion of these risks and uncertainties, please refer to the disclosure in today’s earnings release and BridgeBio Pharma, Inc.’s periodic reports and SEC filings. All statements made here are based on information available to BridgeBio Pharma, Inc. 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 Pharma, Inc., you may begin your conference.

Chinmay Shukla: Good afternoon, everyone, and thank you for joining BridgeBio Pharma, Inc.’s first quarter 2026 earnings call. I am Chinmay Shukla, Senior Vice President, Strategic Finance. With me are Neil Kumar, our CEO, Matthew Outten, our Chief Commercial Officer, and Thomas Trimarchi, our President and Chief Financial Officer. On today’s call, Neil will walk through our commercial pipeline and business updates, with Matt providing additional commercial detail and Tom covering financials. Following our prepared remarks, we will open the call for questions. For the Q&A session, we will be joined by Ananth Sridhar, Anna Wade, and Justin To who will lead our program with BCP-418, and encaleret, respectively. With that, I will turn it over to Neil.

Neil Kumar: Thanks, Chinmay, and thanks, everyone, for joining us today. As always, this is a forum in which we communicate salient aspects of our business that are of interest to investors, and we welcome your questions and feedback along the way. I want to spend the bulk of my time today talking about three things. The first is the Atruvio franchise, and I want to talk about our continued commercial momentum there and how we think about clinical differentiation. Importantly, these two things, plus the economics associated with the Part D orphan drug channel, underpin our confidence that Atruvio will continue to grow even past 2032. The second thing I want to discuss is launch readiness across the three exciting first-in-class or best-in-class brands we have in ADH1, LGMD2I, and achondroplasia.

Although there are no major near-term clinical catalysts for any of these brands, there is a tremendous amount of activity going toward ensuring expeditious and high-quality approvals and launches. In its history, BridgeBio Pharma, Inc. has demonstrated across now three approved products, with hopefully three more to come, the ability to take on post–Phase III regulatory submission activities at very high quality, and we intend to build on this tradition with these new brands. I will end my comments addressing the current gap between our intrinsic value and where our shares trade today. I have heard from investors in the past that too much NPV talk is not what people tune into these calls to hear. At this point, it is my responsibility to discuss matters related to capturing the value that investors who have been in our stock for a long time have helped create.

Our focus at BridgeBio Pharma, Inc. has always been on long-term value creation, and on reliably being able to take in money to do more work over time. And by the way, our activities across the greater bio ecosystem show that there are many more of these R&D opportunities out there. But this model is reliant on capturing the value of the work for investors, which is why today I will be discussing a share buyback program that will commence immediately. Let me begin my comments by talking about Atruvio. As many of you have read in the press release, we had $180.6 million in U.S. Atruvio net product revenue this quarter, which represents 24% growth from the last quarter and 392% growth year over year and is consistent with the brand globally becoming a blockbuster in 2026.

Our focus continues to be on winning in the frontline. We believe a 95% stabilizer that preserves the native tetramer is not only the optimal solution, but even against combinations is the only solution one should start with, as it provides the highest degree of management of TTR monomer deposition, provides impact more quickly, is consistent with the pharmacokinetics of TTR stabilization, and ultimately achieves all of this in a cost-efficient and easy-to-access manner. Parenthetically, our data suggests a tripling in combo use with Atruvio with the various knockdowns, suggesting that the message that one should reach for a better stabilizer, even in combination, is resonating. As it relates to the frontline, our major competition continues to be Pfizer, and our best understanding of our share is that it has grown from the NBRx share I quoted at the JPM talk of 25% even furthermore, but still remains behind what Pfizer has been able to accomplish in the frontline.

We believe that in this quarter, total new patient starts in the category were in excess of 6,100 new patients. We believe that for the first time, we are convincingly the second brand by volume in the space. There is more work to be done, but all of these trends continue to be in the right direction for us. So how do we pour some gasoline on these growing sales? The obvious way to do that in our mind is through clinical differentiation. We began to see reasonable inclines in the second derivative of our growth as the serum TTR story began to evolve in the marketplace, with multiple papers suggesting that higher levels of serum TTR are associated with lower levels of mortality at 30 months. As a reminder, every mg per deciliter of incremental increase in serum TTR seems to correspond to a 5% decrease in mortality risk at 30 months, meaning a more potent stabilizer should lead to better outcomes for the patients that we serve.

We do not hear from many physicians quibbling with the fact that Atruvio is a near-complete stabilizer. Building on that, we are now beginning to explore and are confident in the outperformance of acoramidis versus tafamidis in the real-world setting. There were only really two real-world evidence studies reported to date, survival studies done in Colombia and by Dr. Mazri, that showed outperformance of acoramidis; both those were comparing our trial data and not, at that point, classic real-world data because we did not have enough time in the market to demonstrate anything in the classic real-world evidence setting. That is now changing. At ACC, an independent real-world evidence study presented by the Valley Health System of Nevada revealed statistically significant outcome improvements associated with acoramidis as compared to tafamidis.

Building on this, we have a study in medRxiv that we will publish shortly in a major journal showing that Atruvio reduces diuretic intensification by 43% as compared to tafamidis. We intend to continue studying and publishing on differentiation in the real-world setting and are glad to see the cardiology community doing so independently. Interestingly, one of the benefits of acoramidis that was identified in the independent real-world evidence study was a lower incidence of acute kidney injury. As I mentioned in my J.P. Morgan talk, we are driving toward what we believe will be a seminal publication with potential impact for patients, physicians, and even on our label, as it discusses an observed rapid hemodynamically mediated renal protective effect, which is unique to Atruvio as opposed to the other stabilizers and knockdowns in the space.

We continue to present and publish on acoramidis at major medical meetings as well. At ACC recently, we presented long-term efficacy and safety data from our Phase III open-label extension showing sustained clinical benefit from acoramidis at month 54, including a remarkable statistically significant risk reduction of 45% in all-cause mortality with a p-value of less than 0.0001, and a 49% reduction in cardiovascular mortality, again with a similar p-value, versus placebo. I would like to turn to the rest of the pipeline now. On LGMD2I, as I alluded to in my earlier comments, our team was able to go from top-line data to NDA submission in 155 days, consistent with our ethos that every minute counts and the fast pace that we have previously set with regard to our TTR regulatory submissions.

We continue to work closely with the agency and foreign regulators to bring this medicine as expeditiously as possible to the patients who need it. I had the opportunity to attend the top-line results presentation recently in Orlando at the MDA meeting. It was a trip I will not soon forget. I was struck by the excitement our data generated not only within the LGMD2I community, but more broadly, given the striking results associated with BCP-418, suggesting that functional improvement is possible targeting well-described conditions at their source. Given the already ~500 genetically confirmed patients in the United States today, the highly engaged patient community, and physician education being conducted by the team, all of this augurs well for a positive launch dynamic.

Moving to ADH1, I will be leaving from here to a very similar gathering—top-line presentation of our CALIBRATE Phase III data—at the European Congress of Endocrinology in just a matter of days. Here again, we will be looking to drive excitement into the broader physician community and to educate the patient community and establish a base of data that, together with our publication of our data, can ensure market-building exercises continue with high fidelity. Importantly, BridgeBio Pharma, Inc. has been supporting via grant family genetic testing events in the United States that continue to identify new patients with relatively high yield. Although we will be launching at a time when the majority of patients with ADH1 have not been identified yet, the combination of genetic testing, ICD-10 codes, and broad disease awareness education, plus our belief that we will be able to find ever more patients in need of this compelling drug product, supports the opportunity.

Furthermore, our Phase III in chronic hypoparathyroidism will be commencing this summer and is bolstered by recent published work that illuminates the central role the calcium-sensing receptor in the kidney plays in regulating calcium metabolism. Finally, moving to achondroplasia, the results from this trial came after LGMD2I and ADH1, but I suspect given the strength of the results, prominence of the condition, and the remarkable KOLs we are privileged to partner with, that the Phase III manuscript will be forthcoming in a major medical publication and we anticipate presenting the full PROPEL 3 data set at a medical conference in 2026. Early commercial research here suggests unaided awareness in excess of 40% of the prescribing physician community, which for those of you who have commercialized brands know is a very high starting point and certainly higher than we have seen before in our own portfolio.

Finally, I want to touch on the share repurchase program that we announced today. To do so, I would like to go back to BridgeBio Pharma, Inc.’s founding principles. The company was built on two things: to help as many patients as possible, and to establish a corporate and financial model that creates and captures value in predictable, responsible ways. That value capture has always been part of the mission. We talk about NPV, and while we anchor to intrinsic value, we try to make the right economic decision at every port. The reason for that is because if we capture value, more capital flows into drug development over time, and more patients get served, by us and others employing our decentralized, diversified model. Unfortunately, at this moment, we have not adequately captured value for the investors we serve, given the large disconnect between our NPV per share and our firm’s intrinsic value.

Even with the revision of Invitae’s entry from 2035 to mid-2031 or early 2032, our intrinsic value remains markedly higher than where our shares trade today for investors. To that end, the board has authorized a $500 million share repurchase program. These repurchases should allow our shareholders to concentrate their ownership in a portfolio whose risk profile has fundamentally improved. Of note, we have employed this technique in the past some six times. In aggregate, even accounting for the pre–Part A buyback, we have driven substantial returns for investors with our share repurchases. While we have this lever, we still believe in putting capital into our launches and advancing our clinical trials. Repurchases are additive and opportunistic, not substitutive, and are a direct result of our strong balance sheet.

On the balance sheet point, we will always size deployment such that we preserve full flexibility to finance every critical program and activity in our portfolio. Plenty of liquidity and the ability to easily service our liabilities is a requirement before we deploy dollars into the buyback. With that, I will turn the call over to Matt to talk more about our commercial efforts.

Matthew Outten: Thank you, Neil, and good afternoon, everyone. Q1 was another strong quarter for Atruvio, delivering an impressive 24% increase in net sales from Q4 and a 392% year-over-year increase from Q1 2025. Growth was driven by our existing and expanded sales teams accelerating new patient starts and first-line share gains. There are several factors which contributed to these results. Market momentum has remained strong. New-to-brand market share hit its fastest quarter-over-quarter growth since Q1 2025, and first-line patients have increased each and every month of the launch. Fill rates, app rates, gross-to-net, compliance, and persistency all continue to remain in line with expectations. Insurance reauthorization dynamics have been a topic of industry discussion this quarter; I want to address them directly.

Atruvio did not experience reauthorization disruptions for two reasons. Part D, as in David, is a continuous plan-based model which avoids the annual renewal friction of Part B, as in boy. That structural advantage matters. In addition, in 2025, the average copay for Atruvio patients was only $190 for the entire year, with many patients paying $0 out of pocket as well. Our field teams executed with exceptional discipline to keep patients on therapy without interruption. We hire exceptional people, and those people make sure that any patient who wants a near-complete stabilizer can get Atruvio and, importantly, can stay on Atruvio. Turning to our pipeline, we are encouraged by early indicators across our three anticipated near-term launches on LGMD2I R9, ADH1, and achondroplasia.

In LGMD2I R9, we are entering a disease area where no approved therapy exists. We have onboarded a commercial leadership team and have set up a specialized patient identification and field reimbursement infrastructure. Our goal is simple: find every patient who can benefit and be ready to serve them from day one of approval. In ADH1, our claims analysis has already identified nearly 2,000 in the U.S., and that number continues to grow. We have built a dedicated sales leadership team and patient infrastructure tailored to this community. In CALIBRATE, we will be the first medication to target the disease mechanism correctly and it is orally administered. Physician excitement is high, and we are ready to move immediately at approval. In achondroplasia, we are preparing for a global launch with a truly differentiated clinical profile.

Infigratinib is the first medication to demonstrate statistically significant improvement in body proportionality, not just improvements in average height velocity, and the only oral option in the category. For families seeking an alternative to injectables, or returning to treatment after a negative experience, the clinical profile and route of administration of infigratinib is very compelling. To summarize, Q1 continues to reflect a durable growth trajectory for Atruvio and proof of the commercial capability we have built at BridgeBio Pharma, Inc.—an organization that knows how to launch, scale, and build franchises. We remain focused on execution for patients, for families, for prescribers, and for long-term value creation. I will now turn the call over to Tom.

Thomas Trimarchi: Thank you, Matt, and good afternoon, everyone. I will now walk through our financial results for 2026. Our commentary will focus on GAAP financials unless otherwise noted. Total revenues for 2026 were $194.5 million compared to $116.6 million for the same period in 2025. The $77.9 million increase was primarily driven by a $143.9 million increase in Atruvio net product revenue. Atruvio net product revenue in the quarter was $180.6 million compared to $36.7 million in the same period last year. Royalty revenue increased by $9.3 million to $9.5 million, primarily earned from net product sales of acoramidis in Europe and Japan. License and services revenue was $0.4 million compared to $79.7 million for the same period last year.

The decline reflects recognition of a one-time $75 million regulatory milestone from the prior year. Total operating expenses for 2026 were $290.5 million compared to $218.4 million in the same period last year. The $72.1 million increase was due to deliberate and disciplined investment in Atruvio and preparations for three upcoming launches. SG&A expenses were $163.9 million, an increase of $57.5 million compared to the same period last year, reflecting measured investment in our commercial capabilities. R&D expenses were $126.6 million, an increase of $15.2 million, driven by investments in medical affairs and CMC in support of our next three launches. On the operating line, in the first quarter, we recorded a $106 million operating loss.

For the last five quarters, our loss from operations has narrowed by more than 50%, reflecting OpEx discipline and strong execution on Atruvio. Looking at the quarterly trend, if we back out one-time milestone payments, we have seen improvement in the operating line every quarter since the Atruvio launch. Looking ahead, we expect the trend in loss from operations to flatten over the next two quarters as we ramp up launch readiness activities for the next few products, and continue narrowing toward the end of this year as we transition to a P&L breakeven, followed by cash flow positivity, which we expect to be sustainable from that point on. Turning to the balance sheet, we ended the first quarter with $940.2 million in cash, cash equivalents and marketable securities compared to $587.5 million at the end of last year.

We believe our current cash position provides us with significant runway to fund our operating activities, advance our three late-stage programs to approval and launch, and continue to invest in Atruvio’s growth, all while maintaining financial discipline. With that, I will turn the call back over to Chinmay.

Chinmay Shukla: Thank you, Neil, Matt, and Tom. Operator, please open the line for questions now.

Operator: Thank you. Ladies and gentlemen, we will now begin the question and answer session. At this time, I would like to remind everyone, in order to ask a question, please press star followed by the number one on your telephone keypad. And if you would like to withdraw your question, press star 1 again. In the interest of time, we kindly ask everyone to limit themselves to one question only. We will pause for a moment to compile the Q&A roster. Thank you. Our first question comes from the line of Tyler Van Buren with TD Cowen. Please go ahead.

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Tyler Van Buren: This is Sam on for Tyler. Thanks very much for taking our questions, and congrats on another strong quarter. I was just wondering, can you talk about what is driving the continued IQVIA acceleration? And specifically, what you are seeing in those treatment-naive patients? Thank you very much.

Matthew Outten: We are definitely excited about the continued performance of Atruvio. I think the acceleration you are referring to is being driven by a few things. The first is physicians’ desire to use the only near-complete stabilizer on the market. Stabilization is the backbone of therapy in ATTR cardiomyopathy, and near-complete stabilization, along with Atruvio’s speed in showing separation from placebo, is attractive to patients and HCPs. They want something that is going to work quickly, and Atruvio has shown that it can do that. And recently, as you heard from Neil in his original remarks, the real-world evidence has backed all of these points up for both the treatment-naive patients and the switch patients, and that is helping to drive our share upwards.

Neil Kumar: Maybe the only thing I would add there is the serum TTR story. You saw a bevy of papers both from us, but then importantly from others, with the numbers that I put forth—each mg/dL increase associating with pretty remarkable decreases in mortality downstream. As a reminder, when we put patients on acoramidis following administration of tafamidis, coming out of our Phase III trial, you saw a 3.4 mg/dL increase and every patient increased their serum TTR level. So it does not really matter how you measure it—you are getting increases in serum TTR. The question outstanding was never whether acoramidis is a better stabilizer; I think most people can understand that even if they cannot understand a specific in vitro assay.

The bigger question was how much more is that buying in terms of downstream results? The serum TTR work was just the first part of that. I think you can see a lot more of that in the coming 12 to 24 months, really just because we have enough patients now with enough duration that we can start to ask and answer those questions.

Operator: Our next question comes from the line of Mani Foroohar with Leerink Partners. Please go ahead.

Matthew Outten: Hey, Manny. Thanks for the question. So the clarity on where the IP sits is clearly a meaningful positive for BridgeBio Pharma, Inc. and Atruvio. We now have at least six years of runway before generics, which is more than enough time to reach peak share, and obviously this all materially reduces any tail risk to the NPV of the program. I do not think the resolution really changes our commercial strategy. We have always been focused on establishing Atruvio as the treatment of choice in ATTR cardiomyopathy, given its differentiated profile, and we are executing against that. You heard both Neil and me talk about the value of the literature that we are producing as well as all the commercial activities we are undertaking to really reinforce that differentiation and drive share.

We have shared our belief that Atruvio will be a $4 billion drug; we said that last year in our Q1 2025 earnings call. I do not think we have ever been more confident in that estimate. If anything, there might be some room for potential upside. As Neil mentioned in his prepared remarks, Atruvio is likely to keep going even after 2032 given the ecosystem and channel dynamics that exist here with Part D. That is what is driving our confidence.

Neil Kumar: Maybe I will add to that. I personally did think—and I said it—that it would be 2035, so obviously I was wrong on that. That is a little bit of a discount, but not material, as I mentioned in my comments. The bigger thing is, all of these differentiating studies that we are running are really starting to resonate with clinicians, in addition to the fact that access programs are superior. I guess what your question really relates to is: will we run a double-blind head-to-head? We still might; we reserve the option to do that. Certainly, we would be excited to do that in the context of either TTR levels or NT-proBNP. But how we size a hospitalization study is difficult if you look at the number of events. We are going to have a strong look at the placebo arm of the upcoming APOLLO-type trial to really understand what those event rates look like in the context of clinical trials to see if there are some double-blind head-to-head opportunities.

But there is nothing obvious right now, so let us continue doing the real-world evidence studies, which I think are the best characterization of the differential competitive dynamic.

Operator: Our next question comes from the line of Biren Amin with Piper Sandler. Please go ahead.

Biren Amin: Can you walk us through the board’s decision to authorize the $500 million share repurchase program and how you are thinking about balancing that against your investment in new launches and pipeline? And what, if any, considerations are there for additional business strategic initiatives with this share repurchase program now being announced? Thanks.

Neil Kumar: I can take that. Right now, the focus is on focused execution against the pipeline that we have. We have ample growth that has not been valued in the context of this company today—the LGMD2I launch that I referred to, the ADH1 launch, the achondroplasia launch even in its totality. We are projecting market share numbers well in excess of what typically a third mover gets in that space. Then you think about the consequential additional indications, both in terms of hypochondroplasia and, importantly, in terms of chronic hypoparathyroidism that we are kicking off, and then we have the Canavan program. That constellation of activities is more than enough to drive long-term growth for any company, and that is what we are focused on.

Can we fully finance all of that comfortably? We feel like we can. Therefore, beyond that, what do we do with excess capital? We think the best relative return way that we can deploy capital right now is into our own shares, given the disconnect between intrinsic value and where we are trading. That is what the discussion came down to. I know the normal way that a biotech would grow is to say, who cares if share price is low, let us go ahead and dilute everyone and just keep going after science that we believe in. That is not a reliable, sustainable long-term model in my belief, nor is it one that we intend to employ here at BridgeBio Pharma, Inc.

Operator: Our next question comes from the line of Cory Kasimov with Evercore ISI. Please go ahead.

Cory Kasimov: I want to follow up on the real-world evidence that was referenced in both the press release and the prepared remarks that reinforces Atruvio differentiation versus tafamidis. Can you unpack what this real-world data is showing and how it compares with what was demonstrated in the clinical trial setting? Is anything different now than it was, or just more of it? Thank you.

Neil Kumar: It is pretty different because recall that we had a significant left shift in our clinical trial. Our placebo outperformed the on-drug arm of ATTR-ACT. So there is no way for us to actually, apples to apples, go across things like diuretic intensification. And by the way, even the use of diuresis and renin-angiotensin control meds and SGLT2i use in this population—all of that has changed pretty markedly. It almost made it impossible, excepting the in-trial comparisons we can make between tafamidis and acoramidis with all the available caveats there—where acoramidis has outperformed tafamidis in all aspects, which you did not see in HELIOS-B. I think real-world evidence is the right way to do this within systems or across a constellation of systems that we know have a lot of integrity in terms of clinical studies.

Here, you are seeing things like what we mentioned in terms of diuretic intensification. We certainly did not look at downstream kidney effects like the Nevada system did, but it is all resonant with the advantages that we think Atruvio has versus tafamidis in terms of mortality and hospitalization. It is nice to see it actually play out in the real world.

Operator: Our next question comes from the line of Salim Syed with Mizuho. Please go ahead.

Salim Syed: Congrats on another great quarter. Just one from us maybe on infigratinib and PROPEL 3. Since you have had that read, I am sure you have done some market share work or at least spoken to additional folks in the achondroplasia community, both on the clinician side and family side. What has the feedback been, and how has additional feedback informed your expectations for the commercial opportunity? I believe you said previously you think about achondroplasia as being a $2.5 billion TAM and maybe hypochondroplasia the same. Just curious if you have any other color to offer on the commercial opportunity. Thank you.

Matthew Outten: Thanks for the question. The feedback from clinicians has been overwhelmingly positive. HCPs are telling us that they are constantly being asked by families when the oral is coming—not only by families on treatment today, but more importantly, those that stayed on the sidelines, which as a reminder makes up about 70% to 80% of the U.S. market. The consistent best-in-class profile is continuing to resonate with clinicians. They understand that AHI is best in class, height score is best in class, we have the most attractive safety profile, and, importantly, proportionality. The proportionality data point is the one that is resonating most with clinicians because this is the only product that has a statistically significant result in proportionality in a 3- to 8-year-old population.

This demonstrates how directly targeting FGFR3 impacts more than just height. On that note, we will be releasing more data on how infigratinib is benefiting more than just height in medical conferences later this year, some of which has never been seen before in a 52-week placebo-controlled trial. Ultimately, all this reinforces our belief that we will have a big market share—actually, more than 65% market share—as validated by our market research.

Operator: Our next question comes from the line of Eliana Merle with Barclays. Please go ahead.

Eliana Merle: Thanks for taking the question—two from me. First, on limb-girdle, you submitted the LGMD NDA very quickly—from our math, about 155 days from top line—which is very fast compared to average. Can you walk us through where you stand on launch readiness and how you are preparing to get this drug into the hands of the limb-girdle community from day one? And then a second question on the ATTR space: how are you thinking about what we will see from CardioTransform specifically, and what hazard ratio do you think could be competitive?

Anna Wade: Thanks for the question, Eliana. We are really excited. We have our commercial and sales leadership onboard, and we are getting ready to have the sales reps hired later this year. We also now have our medical leadership in place as well as a seasoned MSL team with neuromuscular and rare disease experience. In Q1, our major catalyst was the NDA conference that Neil mentioned in March, where Dr. Kathy Matthews, a leading KOL in the field, presented our Phase III interim analysis data. We got incredibly positive feedback at the meeting about the compelling data package. Since MDA, we have heard about significant patient outreach to neuromuscular centers, and internally, we have received many inbound inquiries from both patients and physicians.

Our focus right now is driving awareness of the Phase III data, as well as emphasizing the importance of genetic diagnosis leveraging sponsored testing programs that are currently available, so that on day one of launch, we are ready to get patients on therapy.

Neil Kumar: Let me address your second question on CardioTransform. We agree it is a super well-powered trial. Against the primary it should be good, and even in the subpopulations, taking the same point estimate from 0.05 or less, they are pretty well powered. I actually expect that they will hit on almost everything they are interrogating. Then it comes back to how to cross-trial compare and how to understand this knockdown technology. Part of that will be how much knockdown they get and whether they are able to improve on the PK profile because I think the reason that vutrisiran performed similarly to eplontersen in HELIOS-B has to do with the timing it takes to get to mean mass knockdown; it took a lot longer than I would have expected.

We will see if that is the case with their drug. Overall, honestly, CardioTransform has more to do with the commercial dynamics with Alnylam than it does with us, especially given the combo data that I just told you about. I think people are going to reach for stabilizers first line anyway. Number one. Number two, I think when they are failing stabilizers, they are going to want a better stabilizer onboard in combination. So if that trial does hit, I do not see it having a meaningful effect for us. From a biochemical standpoint, you always want to preserve the native tetramer. We are seeing more and more information about that. I am surprised people have not been looking at the publicly available FAERS database and what things look like when you are knocking things down versus actually stabilizing, which is consistent with the 225,000-plus patient studies out there suggesting that higher levels of serum TTR are better for you.

I understand that in a short trial those signals are not necessarily resolvable, but over the longer period of time, stabilizers will continue to be frontline and, in combination, will have a pretty good stay there too. On your hazard ratio question, we think the bar is relative risk reduction of 42% and risk reduction of 50% on hospitalization, which I suspect—if this study is consistent with the rest of the modern studies—will be where a vast majority of the events lie. So that 50% reduction in hospitalization is what I will be looking for.

Operator: Our next question comes from the line of Anupam Rama with J.P. Morgan. Go ahead.

Anupam Rama: Congrats on the quarter. Quick one here. I know the NDA is on track for the first half of this year, and the press release highlighted nearly 2,000 now identifiable patients with an ICD-10 code. Can you give us an update about further patient identification efforts and how this sets up how we should all be thinking about the initial launch curve? Thanks so much.

Neil Kumar: Good to hear from you. The foundation of everything we are doing around patient identification is really awareness—awareness of the disease as an important distinct subset within hypoparathyroidism, and then, of course, awareness of our drug encaleret and the wonderful effect we can have for these patients. A major catalyst for awareness will be the upcoming presentation at ECE next week, followed by U.S. presentations later in the year, then hopefully a very high-impact publication as well. In the background, there are some important tactics and strategies. First, as you mentioned, the ICD-10 code is a huge advantage. Many rare diseases do not have an ICD-10 code; we likely will have one that has been in place for a couple of years already, so there is a good amount of data.

That lets us take our analytics capabilities, put them on top of this, and really deploy our field-based medical and commercial leadership in a more surgical way to go to offices, spread awareness, and also make sure the patients they think they have are appropriately diagnosed with sponsored genetic tests or other commercially available genetic tests. Third, and this has been a bigger driver of identification than I would have thought, is family tracing. It makes sense when you consider this is a dominant condition, so there is a 50% chance of passing it on. When we find one person with this condition, if they look and go to a family event, we find out that many of their brothers, cousins, aunts, uncles may be affected. That has been a valuable source of patient identification as well.

We will continue all these efforts and accelerate them as we approach launch.

Operator: Our next question comes from the line of Derek Archulo with Wells Fargo. Please go ahead.

Derek Archulo: Congrats on the progress. Just to follow up on infigratinib: some recent commentary and some of the early KPIs from the Voxzogo launch seem positive and maybe early validation of this market expansion thesis. How do you think about infigratinib’s profile versus the injectables, and how could this further accelerate the market expansion potential? Thanks.

Matthew Outten: Thanks, Derek. Most physicians and families are excited about the total package of infigratinib—not just one thing. It starts with a 2.1 cm change from baseline in annualized height velocity, the largest effect shown across any of the three Phase III programs, which was remarkably consistent across the age groups. Then, as I mentioned earlier, the only statistically proven proportionality benefit—a demonstration of the importance of directly engaging FGFR3. Then you have a differentiated safety profile—no injection site reaction, no symptomatic hypertension, no hypertrichosis. I think the safety differentiation is further playing out given these increasing and concerning signals of SCFE and femur fractures, which are both looking like potential CNP class effects.

On top of this, we are a daily oral. Based on historical benchmarks, we know that when an oral enters a market with only injectables, it expands the market on average by 3 to 4 times at year five after launch. Ultimately, families will have a choice of either 365 injections a year, 52 injections a year, or zero—and I know which one they prefer.

Operator: Our next question comes from the line of Paul Choi with Goldman Sachs. Please go ahead.

Paul Choi: Hi, thank you and good afternoon, and thanks for taking the question. Sticking with infigratinib, I want to ask with regard to the PROPEL infant and toddler study in patients who are newborns or up to two years old. Can you comment on your updated thoughts on enrollment timing and when that might potentially be completed in the wake of your positive results from the PROPEL study, and how you think about timing for that potentially being completed and added to the label? Thank you.

Matthew Outten: Thanks for the question, Paul. There is a lot of excitement from sites following the efficacy in the Phase III PROPEL 3 data. What we know from the field shows that the earlier you intervene, the more likely you impact central outcomes. We will provide an update on timing later on once we have more clarity as this program progresses.

Operator: Our next question comes from the line of Analyst with Jefferies. Please go ahead.

Analyst: Hey. Thanks for all the updates. Congrats on execution. I have a bigger-picture question on your broader pipeline. Now that you have succeeded across four major programs all the way through Phase III, as investors think about the sustainability of your R&D engine, can you talk about how you are currently thinking about the next wave of development beyond your portfolio, how open you are to adding more to the pipeline in the near term, and what indication areas you could be interested in? Thank you.

Neil Kumar: Thanks for the question. Right now, our comments and actions have always been consistent with being very focused on the opportunity in front of us. It is not often that a biotech will launch three different products in three different indication settings alongside a pretty competitive market at the same time, and that is going to take, certainly against our lean backbone, all of the focus that we have. I also mentioned earlier that we have significant additional opportunities associated with every single one of our drug products, including some that we have not talked about vis-à-vis Atruvio. There is some pretty interesting stuff to do. We have an internal pipeline—programs in ADPKD, eliminating polyketide monomers, and the complement antibody program in ATTR cardiomyopathy.

Those are programs that are very capital efficient. We also have backup programs against all of our current pipeline programs so that we can do what is right for the patient and physician community to continue to serve them as long as possible. All of those things together represent the menu of activities that we are interested in the near term. Obviously, we are students of the genetic disease space. BridgeBio Pharma, Inc. has a significant stake in another company called Bonum Therapeutics, which is one of our sister companies and has 17 programs ranging from Phase II all the way back to the preclinical setting—small molecules, ASOs, antibodies, all targeting well-described genetic conditions at their source. We are happy for that to be an off-balance-sheet R&D exercise for now, as is BridgeBio Oncology Therapeutics, and really to focus on what we need to focus on here: continued prosecution of our pipeline programs, delivery of these important medicines to patients, and ultimately the capturing of the value that we have created for the investors that have backed us for many years.

Operator: Our next question comes from the line of Danielle Brill Bongero with Truist. Please go ahead.

Danielle Brill Bongero: Congrats on the great quarter. Neil, I believe you mentioned in your prepared remarks that there were 6,100 new patient starts across the class in the quarter. If I recall correctly, this represents a meaningful step up from prior quarters where I think it was more in the 4,000 new patients range. What is driving this step up, and moving forward, how should we think about the size of the quarterly patient pool that you are actively competing for? Thank you.

Neil Kumar: I think this market continues to grow to somewhere between 5,000 and 6,000 a quarter in terms of new patient starts or new-to-brand patients. I do not think we had 4,500 last quarter; I think it was above 5,000. A little bit of this math has to do with us guessing for our competitors what the inventory holdback was or what the inventory buys were and things of that nature—so we can never get it fully right. We can get our own patient numbers fully right. But that does suggest continued growth. Will it continue to grow? I think so. There are 250,000 patients with a cardiomyopathy at the low end in the U.S., and I think we are doing a better job of three things. One is making sure that the algorithms are in the EMR so that people look for these patients.

There is the Tracer AI stuff that we have been doing and other algorithms that individual health care systems have been putting forth to get people to think, “Ah, maybe this is a TTR-CM patient.” Second is driving genetic testing into variant-heavy populations; that has been helpful as well. Third, and probably the best, is broad physician awareness and education through speaker bureaus, really getting out into the academic practices and the community practices to educate them more. All of that is positive. On the negative side, we have heard quite a bit about this PYP shortage—the technetium scan is one of the ways to get a definitive diagnosis—so we have to keep an eye on that. We heard about that before in 2025, I think in the second or third quarter; the market continued to grow.

That is resolvable. There are three major suppliers, and I expect in the years going forward we should not see much more of this supply chain iteration around PYP availability. Long story short, I believe you should see superlinear growth in identification, given the number of patients that we believe have a cardiomyopathy coupled with the number of sponsors in the playing field and the availability of reasonable testing. I would expect that positive trend to continue, with some error bars quarter to quarter.

Operator: Our next question comes from the line of Jason Zemansky with Bank of America. Please go ahead.

Jason Zemansky: Good afternoon. Congrats on the great progress, and thanks for taking our question. Maybe one more on encaleret, if we may. As you look beyond the ADH1 opportunity to the broader chronic hypoparathyroidism opportunity, how are you thinking about encaleret’s positioning relative to the parathyroid hormone replacement therapies? Is there a particularly attractive subgroup to target, or are you looking at the broader opportunity as a whole? And maybe talk about some of the pricing implications of pursuing a market that maybe looks a little bit like 25,000 patients in the U.S. and EU versus 200,000? Thanks.

Matthew Outten: Hey, Jason. Great to hear from you, and thank you for the question. We look forward to seeing you next week at the conference. In chronic hypoparathyroidism, when we did our market research, three things drove our excitement. First, this will be the first oral option available in the chronic setting. The ability to give patients freedom from injection-site reactions and all the pain that comes with it resonates very well. Second, if you look at the current options available, you do see an effect in terms of blood calcium normalization, but you do not really see an effect on urine calcium normalization. With encaleret, we have a very unique profile where we could normalize potentially both blood and urine calcium, and we saw that in our, albeit small, Phase II trial, where around 80% of the patients normalized both blood and urine calcium—these are patients who are very sick and did not have the parathyroid gland or any amount of the hormone.

Third, there is a potential safety risk in terms of bone resorption from giving PTH at high levels for the whole day. We could completely avoid that. The ADH1 readout significantly de-risks and highlights the therapeutic profile for encaleret. Those three things—oral administration, urine calcium normalization, and potential benefit on safety—are why we think we can compete and get a reasonable share even in the chronic hypoparathyroidism market, obviously assuming the trial works.

Operator: Our next question comes from the line of Analyst with Morgan Stanley. Please go ahead.

Analyst: This is Morgan on for Sean. Thanks for taking our question. Can you remind us specifically for infigratinib in achondroplasia what kind of, if any, commercial preparations are taking place from BridgeBio Pharma, Inc.? Thank you.

Matthew Outten: Thanks for the question. We have built strong commercial and medical leadership, bringing on both sides of the business with experience in second and third launches with superiority data, especially with launch and global experience as well. Ultimately, we are making sure we get the word out not just to leading geneticists and ASCs, but also to the broader community of pediatric endocrinologists who are excited about having an oral option, especially for families who are not seen at super-specialized centers of care and are more interested in something that is easier for families to administer. There is a lot of education going on that front. Also remember, we have a lot of the teams in place from the Atruvio launch that can help with future launches across indications—market access with the payers, specialty pharmacies—these are the same individuals.

We have relationships with all of those people and are able to launch quickly as a result, and get access and coverage.

Operator: Our next question comes from the line of John Boyle with William Blair. Please go ahead.

John Boyle: Hi, team. Thanks so much for taking our question and congrats on a strong quarter. Patient advocacy groups for achondroplasia seem to have a pretty big voice in the indication. Have you had interactions with them and can you speak to how the infigratinib profile is resonating there?

Matthew Outten: Thanks for the question. That has been a core tenet of how we have developed since the very beginning. We have been working alongside advocacy groups both in the United States and internationally, making sure that their input and voice are implemented in our development program and how we think about endpoints. For us, being able to target FGFR3 directly addresses their concerns of being able to look at not just height outcomes, but health outcomes as well, which is something we expect to play out in the longer term and in our clinical extension program. They have been wonderful partners with us, and we anticipate that persisting through commercialization and launch as well.

Operator: Thank you. Ladies and gentlemen, that concludes our Q&A session. I will now turn the call back over to the BridgeBio Pharma, Inc. team for closing remarks.

Chinmay Shukla: Thank you, everyone, for your questions today. We really appreciate your interest and look forward to updating you again next quarter.

Operator: Ladies and gentlemen, that concludes our conference call. You may now disconnect your lines. Have a pleasant day.

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