Q1 2026 BridgeBio Pharma Inc Earnings Call

May 7, 2026

[Analyst]: Well, there's something, it's hard to explain. My baby-

Speaker #1: But my baby.

Speaker #2: 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.

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. If you would like 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 one 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'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.

Speaker #2: If you would like 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 one again.

Speaker #2: 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.

Operator: 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'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.

Speaker #2: 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 risk and uncertainties, which could cause actual results to differ materially from those expressed or implied in this forward-looking statement.

Speaker #2: For a discussion of this risk and uncertainties, please refer to the disclosure in today's earnings release and BridgeBio's periodic reports and SEC filing. 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.

Operator: 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.

Speaker #3: Good afternoon, everyone, and thank you for joining BridgeBio Pharma's first quarter 2026 earnings call. I am Chinmay Shukla, Senior Vice President, Strategic Finance. With me are Neil Kumar, our CEO; Matt Outten, our Chief Commercial Officer; and Tom Trimarchi, our President and Chief Financial Officer.

Chinmay Shukla: Good afternoon, everyone, thank you for joining BridgeBio Pharma's Q1 2026 Earnings Call. I am Chinmay Shukla, Senior Vice President, Strategic Finance. With me are Neil Kumar, our CEO; Matt Outten, our Chief Commercial Officer; and Tom 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 programs with encaleret, BBP-418, and infigratinib respectively. With that, I'll turn it over to Neil.

Speaker #3: 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.

Chinmay Shukla: 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 programs with encaleret, BBP-418, and infigratinib respectively. With that, I'll turn it over to Neil.

Speaker #3: For the Q&A session, we will be joined by Anant Sridhar; Anna Wade; and Justin Doe, who will lead our programs with. And Calyrex, BBP 418, and Integratnet, respectively.

Speaker #3: With that, I'll turn it over to Neil.

Speaker #4: 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.

Neil Kumar: Thanks, Chinmay. 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 ATTRUBY 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 R&D orphan drug channel, underpin our confidence that ATTRUBY will continue to grow even past 2032. The second thing I want to discuss is launch readiness against the three exciting first-in-class or best-in-class brands we have in ADH1, LGMD2I, and achondroplasia.

Speaker #4: I want to spend the bulk of my time today talking about three things: the first is the Attubi franchise, and I want to talk about our continued commercial momentum there, and how we think about clinical differentiation.

Neil Kumar: Importantly, these two things, plus the economics associated with the R&D orphan drug channel, underpin our confidence that ATTRUBY will continue to grow even past 2032. The second thing I want to discuss is launch readiness against the three exciting first-in-class or best-in-class brands we have in ADH1, LGMD2I, and achondroplasia.

Speaker #4: Importantly, these two things, plus the orphan drug channel, underpin our confidence that Attubi will continue to grow even past 2032. The second thing I want to discuss is launch readiness against the three exciting first-in-class or best-in-class brands we have in ADH1, LGMBQI, and ACONDRPLAGIA.

Speaker #4: 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.

Neil Kumar: 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 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'll end my comments by addressing the current gap between our intrinsic value and where our shares trade today. I've 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.

Speaker #4: approved products with hopefully three more to come. The ability to take on post-phase three regulatory submission activities at very high quality and we intend to build on this tradition with BridgeBio has demonstrated across now three these new brands.

Speaker #4: I'll end my comments by addressing the current gap between our intrinsic value and where our shares trade today. I've heard from investors in the past that too much MPV talk is not what people tune into these calls to hear, but 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.

Neil Kumar: I'll end my comments by addressing the current gap between our intrinsic value and where our shares trade today. I've 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.

Speaker #4: Our focus at BridgeBio has always been on long-term value creation, and on reliably being able to take in money to do more work over time.

Neil Kumar: Our focus at BridgeBio has always been on long-term value creation and on reliably being able to take in money to do more work over time. By the way, our activities across the BridgeBio ecosystem show that there are many more of these R&D opportunities out there. 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 Attruby.

Speaker #4: And by the way, our activities across the BridgeBio 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'll be discussing a share buyback program that will commence immediately.

Speaker #4: Let me begin my comments by talking about Attubi. As many of you have read in the press release, we've had 186 oh, we've had 180.6 million dollars of Attubi sales in US Attubi net product revenue this quarter, which represents a 24% growth from the last quarter and a 392% growth year on year, and is consistent with the brand globally becoming a blockbuster in 2026.

Neil Kumar: As many of you have read in the press release, we've had $180.6 million of ATTRUBY sales in US ATTRUBY net product revenue this quarter, which represents a 24% growth from the last quarter and a 392% growth year-on-year and is consistent with the brand globally becoming a blockbuster in 2026.

Speaker #4: Our focus continues to be on winning in the front line, where 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 PTR monomer deposition, provides impact more quickly, and is consistent, which is consistent with the pharmacokinetics of PTR stabilization, and ultimately achieves all of this in a cost-efficient and easy-to-access manner.

Neil Kumar: Our focus continues to be on winning in the front line, where 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 monitor deposition, provides impact more quickly, which 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 ATTRUBY with the various knockdowns, suggesting that the message that one should reach for a better stabilizer, even in combination, is resonating.

Speaker #4: Parenthetically, our data suggests a tripling in combo use with Attubi with the various knockdowns, suggesting that the message that one should reach for a better stabilizer, even in combination, is resonating.

Speaker #4: As it relates to the front line, our major competition continues to be Pfizer, and our best understanding of our share is that it has grown from the MBRX share I quoted at the JPM talk with 25% even furthermore, but still remains behind what Pfizer has been able to accomplish in the front line.

Neil Kumar: As it relates to the front line, our major competition continues to be Pfizer, and our best understanding of our share is that it has grown from the MBRF share I quoted at the JPM talk of 25% even furthermore, but still remains behind what Pfizer has been able to accomplish in the front line. We believe that in this quarter, total new patient starts in the category were in excess of 6,100 new patients, and we believe that for the first time we are convincingly the second brand by volume in this space. There's more work to be done, but all of these trends continue to be in the right direction for us. How do we pour some gasoline on these growing sales? The obvious way to do that in our mind is through clinical differentiation.

Speaker #4: We believe that in this quarter, total new patient starts in the category where an excess of 6,100 new patients, and we believe that for the first time, we are convincingly the second brand by volume in the space.

Speaker #4: There's 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?

Speaker #4: 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, the serum TTR story began to evolve in the marketplace with multiple papers suggesting that higher levels of serum TTR associated with lower levels of mortality at 30 months.

Neil Kumar: 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 associated with lower levels of mortality at 30 months. As a reminder, every mg per deciliter of incremental increase in serum TTR seems to lead to a 5% decrease in mortality risk at 30 months, meaning a more potent stabilizer. we do not hear from many physicians quibbling with the fact that ATTRUBY is a more potent stabilizer, it is, should lead to better outcomes for the patients that we serve. Building on that, we are now beginning to explore and are confident in the outperformance of acoramidis versus tafamidis in the real-world setting.

Speaker #4: As a reminder, every MIG per deciliter of incremental increase in serum TTR seems to lead to a 5% decrease in mortality risk at 30 months, meaning a more potent stabilizer and we do not hear from many physicians quibbling with the fact that Attubi is a more potent stabilizer.

Speaker #4: It is. Should lead to better outcomes for the patients that we serve. Building on that, we are now beginning to explore and are confident in the outperformance of Akkeramidus versus Difamidus in the real-world setting.

Speaker #4: There were only really two real-world evidence studies reported to date: survival studies done in Colombia and by Dr. Mosry that showed outperformance of Akkeramidus in both, but those were comparing our trial data and not—at that point—we didn't have enough time to be in the market long enough to demonstrate anything in the classic real-world evidence setting.

Neil Kumar: There were only really two real-world evidence studies reported to date, survival studies done in Colombia and by Dr. Masri that showed outperformance of acoramidis both. Those were comparing our trial data and not at that point. We didn't have enough time at that point to be in the market long enough to demonstrate anything in the classic real-world evidence setting. That is now changing. At FCAI, 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 ATTRUBY reduces diuretic intensification by 43% as compared to tafamidis. We intend to continue studying and publishing on ATTRUBY's differentiation in the real-world setting and are glad to see the cardiology community doing so independently.

Speaker #4: That is now changing. At SCAI, an independent real-world evidence study presented by the Valley Health System of Nevada revealed statistically significant outcome improvements associated with Akkeramidus as compared to Difamidus.

Speaker #4: Building on this, we have a study in MedRxiv that we will publish shortly in a major journal, showing that Attubi reduces diuretic intensification by 43% as compared to Difamidus.

Speaker #4: We intend to continue studying and publishing on Attubi's differentiation in the real-world setting and are glad to see the cardiology community doing so independently.

Speaker #4: Interestingly, one of the benefits of Akkeramidus that was identified in the independent real-world evidence study was a lower incidence of acute kidney injury. As I mentioned in my JPMorgan talk, we are driving toward what we believe will be a seminal publication.

Neil Kumar: 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 JP 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 ATTRUBY 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.

Speaker #4: 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 Attubi as opposed to the other stabilizers and knockdowns in the space.

Speaker #4: We continue to present and publish on Akkeramidus and major medical meetings as well at ACC recently. We presented long-term efficacy and safety data from our phase three open-label extension showing sustained clinical benefit from Akkeramidus at month 54, including a remarkable statistically significant risk reduction of 45% in all-cause mortality with a p-value of less than 0.001 and a 49% reduction in cardiovascular mortality again with a similar p-value versus placebo to Difamidus or to Akkeramidus.

Neil Kumar: 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 to tafamidis or to acoramidis. All right. I'd 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.

Speaker #4: All right. I'd 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.

Speaker #4: 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.

Neil Kumar: 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 a top-line result presentation recently in Orlando at the NDA meeting. It was a trip I won't soon forget. I was struck by the excitement that our data generated, not only within the LGMD2I community, but more broadly, given the striking results associated with BBP-418 suggest that functional improvement is possible when targeting well-described conditions at their source. Given the already 500 or so genetically confirmed patients in the United States to date, the highly engaged patient community and physician education being conducted by the team, all of this augurs well for a positive launch dynamic.

Speaker #4: I had the opportunity to attend the top-line result presentation recently in Orlando at the NDA meeting. It was a trip I won't soon forget.

Speaker #4: I was struck by the excitement of our that our data generated not only within the LGMD2i community, but more broadly given the striking results associated with BBP-418 suggests that functional improvement is possible when targeting well-described conditions at their source.

Speaker #4: Given the already 500 or so genetically confirmed patients in the United States to date, the highly engaged patient community and physician education being conducted by the team, all of this augurs well for a positive launch dynamic.

Speaker #4: Moving to ADH1, I'll be leaving from here to a very similar gathering of top-line presentation of our Calibrate phase three data at the European Congress of Endocrinology in just a matter of days.

Neil Kumar: Moving to ADH1, I'll be leaving from here to a very similar gathering, a top-line presentation of our CALIBRATE phase III data at the European Congress of Endocrinology in just a matter of days. Here again, we'll 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 phase III data, can ensure market building exercises can continue with high fidelity.

Speaker #4: Here again, we'll 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 Phase 3 data, can ensure market-building exercises can continue with high fidelity.

Neil Kumar: Importantly, BridgeBio 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 are launching at a time when a vast majority of patients with ADH1 have not been identified yet, the combo of genetic testing, ICD-10 codes, and broad disease awareness education helps buttress our belief that we will be able to find ever more patients in need of this compelling drug product. Furthermore, our phase III in chronic hyperparathyroidism will be commencing this summer, and it's bolstered by recent published work that illuminates the central role that the calcium-sensing receptor in the kidney plays in regulating calcium metabolism.

Speaker #4: Importantly, BridgeBio has been supporting via grant family genetic testing events in the United States that continue to identify new patients with relatively high yield.

Speaker #4: Although we have been we are launching at a time when a vast majority of patients with ADH1 have not been identified yet, the combo of genetic testing, ICD-10 codes, and broad disease awareness education helps buttress our belief that we will be able to find ever more patients in need of this compelling drug product.

Speaker #4: Furthermore, our phase three and chronic hyperparathyroidism will be commencing this summer, and it's bolstered by recent published work that illuminates the central role that the calcium-sensing receptor in the kidney plays in regulating calcium metabolism.

Speaker #4: Finally, moving to acondroplasia, 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're privileged to partner with that the phase three manuscript will be forthcoming in a major medical publication and we anticipate presenting the full propelled three data set at a medical conference in the second half of 2026.

Neil Kumar: Finally, moving to achondroplasia, the results from this trial came after LGMD2I and ADH1, I suspect given the strength of the results, prominence of the condition, and the remarkable KOLs we're 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 dataset at a medical conference in H2 2026. Early commercial research here suggests unaided awareness in excess of 40% amidst the prescribing physician community, which for those of you who are commercial experts know is a very high starting point and certainly higher than what we've seen before in our own portfolio. Finally, I want to touch on the share repurchase program that we announced today. To do so, I'd like to go back to BridgeBio's founding principles.

Speaker #4: Early commercial research here suggests unaided awareness in excess of 40% of our prescribing physician community, which, for those of you who are commercial experts, is a very high starting point and certainly higher than we've seen before in our own portfolio.

Speaker #4: Finally, I want to touch on the share we purchased program that we announced today. To do so, I'd like to go back to BridgeBio's founding principles.

Speaker #4: 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.

Neil Kumar: 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. While we talk about NPV, while we anchor to intrinsic value, while we try to make the right economic decision at every fork. 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.

Speaker #4: That value capture has always been part of the mission, why we talk about MPV, why we anchor to intrinsic value, why we try to make the right economic decision at every fork.

Speaker #4: 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.

Speaker #4: Unfortunately, at this moment, we have not adequately captured value for the investors we serve given the large disconnect between our MPV per share and our firm's intrinsic value.

Speaker #4: Even with the revision of Vindamax's entry from 2035 to mid-2031 or early 2032, our intrinsic value remains markedly higher than what we've been able to get the shares to trade to today for investors.

Neil Kumar: Even with the revision of VYNDAMAX's entry from 2035 to mid-2031 or early 2032, our intrinsic value remains markedly higher than what we've been able to get the shares to trade to 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.

Speaker #4: 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.

Speaker #4: Of note, we have employed this technique in the past some six times, some six times. In aggregate, even accounting for the pre-part A buyback, we have driven substantial returns for investors, with our share repurchases.

Speaker #4: 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.

Speaker #4: And our direct results of our strong balance sheet. On the balance sheet point, we will always size deployment such that we preserve full flexibility to finance every credible program and activity in our portfolio.

Neil Kumar: On the balance sheet point, we will always size deployments such that we preserve full flexibility to finance every credible 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. Okay. With that, I will turn the call over to Matt to talk more about our commercial efforts.

Speaker #4: Plenty of liquidity and the ability to easily service our liabilities is a requirement before we deploy dollars into the buyback. Okay. With that, I'll turn the call over to Matt to talk more about our commercial efforts.

Speaker #4: Thank you, Neil. And good afternoon, everyone. Q1 was another strong quarter for Atrubi, delivering an impressive 24% increase in net sales from Q4 and a $392% year-over-year increase from Q1 2025.

Matt Outten: Thank you, Neil. Good afternoon, everyone. Q1 was another strong quarter for Attruby, 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. Bill rates, cap rates, gross to net, compliance, and persistency all continue to remain in line with expectations. Insurance reauthorization dynamics has been a topic of industry discussion this quarter, and I want to address them directly. Attruby did not experience reauthorization disruptions for two reasons.

Speaker #4: Growth was driven by our existing and expanded sales teams accelerating new patients' starts and first-line share gains. There are several factors which contributed to these results.

Speaker #4: 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.

Speaker #4: Bill rates, cap rates, gross-to-net compliance, and persistency all continue to remain in line with expectations. Insurance authors reauthorization dynamics have been a topic of industry discussion this quarter, and I want to address them directly.

Speaker #4: Atrubi did not experience reauthorization disruptions for two reasons. Part D's and David is a continuous plan-based model which avoids the annual renewal friction of Part B, as in boy.

Matt Outten: 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 co-pay for Attruby patients was only $190 for the entire year, making many patients pay only $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 Attruby and, importantly, can stay on Attruby. Turning to our pipeline, we are encouraged by early indicators across our 3 anticipated near-term launches in LGMD 2 R9, ADH1, and achondroplasia. In LGMD 2 R9, we are entering a disease area where no approved therapy exists.

Speaker #4: That structural advantage matters. In addition, in 2025, the average copay for Atrubi patients was only $190 for the entire year. Making many patients pay only $0 out of pocket as well.

Speaker #4: 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 Atrubi and, importantly, can stay on Atrubi.

Speaker #4: Turning to our pipeline, we are encouraged by early indicators across our three anticipated near-term launches and LGMD2i R9, ADH1, and acondroplasia. In LGMD2i R9, we are entering a disease area where no approved therapy exists.

Speaker #4: We've 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.

Matt Outten: We've 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 patients in the US, and that number continues to grow. We've built a dedicated sales leadership team and patient infrastructure tailored to this community. Encaleret would be the first medication to target the disease mechanism directly, and it's orally administered. Physician excitement is high, and we're 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.

Speaker #4: In ADH1, our claims analysis has already identified nearly 2,000 patients in the US, and that number continues to grow. We've built a dedicated sales leadership team and patient infrastructure tailored to this community.

Speaker #4: In calorate, would be the first medication to target the disease mechanism directly and its orally administrated. Physician excitement is high, and we're ready to move immediately at approval.

Speaker #4: In acondroplasia, we are preparing for a global launch with a truly differentiated clinical profile. Infragotinib is the first medication to demonstrate statistically significant improvement in body proportionality.

Speaker #4: 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 Infragotinib is very compelling.

Matt Outten: For families seeking an alternative to injectables or returning to treatment after a negative experience, the clinical profile and route of administration of acoramidis is very compelling. To summarize, Q1 continues to reflect a durable growth trajectory for ATTRUBY and proof of the commercial capability we've built at BridgeBio, 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'll now turn the call over to Tom.

Speaker #4: To summarize, Q1 continues to reflect a durable growth trajectory for Atrubi and proof of the commercial capability we've built at BridgeBio. An organization that knows how to launch, scale, and build franchises.

Speaker #4: We remain focused on execution for patients, for families, for prescribers, and for long-term value creation. I'll now turn the call over to Tom.

Speaker #1: Thank you, Matt, and good afternoon, everyone. I'll now walk through our financial results for the first quarter of 2026. Our commentary will focus on gap financials, less otherwise noted.

Tom Trimarchi: Thank you, Matt, and good afternoon, everyone. I'll now walk through our financial results for Q1 2026. Our commentary will focus on GAAP financials, unless otherwise noted. Total revenues for Q1 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 Attruby net product revenue. Attruby net product revenue in the quarter was $180.6 million compared to $36.7 million for the same period last year. Royalty revenue increased by $9.3 million to $9.5 million, primarily earned from net product sales of BEYONTTRA in Europe and Japan.

Speaker #1: Total revenues for the first quarter of 2026 were $194.5 million, compared to $116.6 million for the same period of 2025. The $77.9 million increase was primarily driven by a $143.9 million increase in Atrubi net product revenue.

Speaker #1: Atrubi net product revenue in a 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 Beyondra in Europe and Japan.

Speaker #1: License and services revenue was $4.4 million compared to 79.7 million for the same period last year. The decline reflects the recognition of one-time $75 million regulatory milestone from the prior year.

Tom Trimarchi: License and services revenue was $4.4 million compared to $79.7 million for the same period last year. The decline reflects the recognition of one-time $75 million regulatory milestone from the prior year. Total operating expenses for Q1 2026 were $290.5 million compared to $218.4 million for the same period last year. The $72.1 million increase reflects deliberate and disciplined investment in ATTRUBY 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 activities.

Speaker #1: Total operating expenses for the first quarter of 2026 were $290.5 million, compared to $218.4 million for the same period last year. The $72.1 million increase reflects deliberate and disciplined investment in Atrubi.

Speaker #1: 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 activities.

Speaker #1: 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.

Tom Trimarchi: 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 3 launches. Turning to the operating line. In Q1, we recorded a $106 million operating loss. For the last 5 quarters, our loss from operations has narrowed by more than 50% due to OpEx discipline paired with strong execution on ATTRUBY. Looking at the quarterly trend, if we back out one-time milestone payments, we've seen an improvement in the operating line every quarter since ATTRUBY launched.

Tom Trimarchi: 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 3 launches. Turning to the operating line. In Q1, we recorded a $106 million operating loss. For the last 5 quarters, our loss from operations has narrowed by more than 50% due to OpEx discipline paired with strong execution on ATTRUBY. Looking at the quarterly trend, if we back out one-time milestone payments, we have seen an improvement in the operating line every quarter since ATTRUBY launched.

Speaker #1: Turning to the operating line. In the first quarter, we recorded a $106 million operating loss. Over the last five quarters, our loss from operations has narrowed by more than 50% due to OPEX discipline, paired with strong execution on Atrubi.

Speaker #1: Looking at the quarterly trend, if we back out one-time milestone payments, we've seen an improvement in the operating line every quarter since Atrubi launched.

Speaker #1: 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 three products.

Tom Trimarchi: Looking ahead, we expect the trend in loss from operations to flatten over the next 2 quarters as we ramp up launch readiness activities for the next 3 products and continue narrowing toward the end of this year into 2027 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 Q1 with $940.2 million in 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 3 late-stage programs towards approval and launch, and continue to invest in ATTRUBY's commercial growth, all while maintaining financial discipline we've demonstrated to date.

Speaker #1: And continue narrowing toward the end of this year in 2027 as we transition toward P&L break-even, followed by cash flow, positivity, which we expect to be sustainable from that point on.

Speaker #1: 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.

Speaker #1: We believe our current cash position provides us with significant runway to fund our operating activities. Advance our three late-stage programs towards approval of launch and continue to invest in Atrubi's commercial growth.

Speaker #1: All while maintaining financial discipline we've demonstrated to date. With that, I'll turn the call back over to Jim.

Tom Trimarchi: With that, I'll turn the call back over to Chinmayi.

Speaker #2: Thank you, Neil, Matt, and Tom. Operators, please open the line for questions now.

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

Speaker #3: Thank you. Ladies and gentlemen, we will now begin the question-and-answer session. At this time, I would like to remind everyone that in order to ask a question, please press star, followed by the number one on your telephone keypad.

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 1 on your telephone keypad. 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 1 question only. We'll 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.

Speaker #3: And if you would like to enjoy your question, press star one again. In the interest of time, we kindly ask everyone to limit themselves to one question only.

Speaker #3: We'll 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 Calvin.

Speaker #3: Please go ahead.

[Analyst] (TD Cowen): Hello, this is Sam on for Tyler. Thanks very much for taking our questions, congrats on another strong quarter. I was just wondering, can you guys talk about what's driving the continued ATTRUBY acceleration and specifically what you're seeing in those treatment-naive patients? Thank you very much.

Speaker #4: Hello. 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 guys talk about what's driving the continued Atrubi acceleration?

Speaker #4: And specifically, what you're seeing in those treatment-naive patients? Thank you very much.

Speaker #5: Hey, this is Matt. I'll take that one. And thanks for the question. We're definitely excited about the continued performance of Atrubi. I think the acceleration you're referring to is being driven by a few things.

Matt Outten: Hey, this is Matt. I'll take that one, thanks for the question. We're definitely excited about the continued performance of ATTRUBY. I think the acceleration you're 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-CM, and near complete stabilization, along with ATTRUBY speed in showing separation from placebo, is attractive to patients and HCPs. They want something that's going to work quickly. ATTRUBY has shown that it can do that. 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. That's helping to drive our share upwards.

Speaker #5: The first is physicians' desire to use the only near-complete stabilizer on the market. Stabilization is the backbone of therapy and ACTRCM. And near-complete stabilization, along with Atrubi's speed and showing separation from placebo, is attractive to patients and HCPs.

Speaker #5: They want something that's going to work quickly. And Atrubi has shown that it can do that. And recently, as you heard from Neil and his original remarks, the real-world evidence has backed all of these points up for both the treatment-naive patients and the switch patients.

Speaker #5: And that's helping to drive our share upwards.

Speaker #6: Yeah, maybe the only thing I'd add there is the serum TPR story I think you saw a bevy of papers, both from us, but then importantly, from others.

Tom Trimarchi: Yeah. Maybe the only thing I'd add there is, the serum TTR story, I think, you know, you saw a bevy of papers both from us, but then importantly from others, suggesting the numbers that I put forth, the mg/dL increase associating with pretty remarkable decreases in mortality downstream. You know, obviously, 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 everyone increased their serum TTR levels. It doesn't really matter how you measure it, you're just getting increases in serum TTR. The question outstanding was never, I don't know if, Matt, if you'd agree with this, whether or not acoramidis is a better stabilizer.

Speaker #6: Suggesting the numbers that I put forth that make for deciliter increase associating with pretty remarkable decreases in mortality downstream. And obviously, as a reminder, when we put patients on apparameters following administration of tafamidus, coming out of our phase three trial, you saw a 3.4 mg/dL increase.

Speaker #6: And everyone increased. Their serum TPR level. So it doesn't really matter how you measure it. You're just getting increases in serum TPR. The question outstanding was never out of note, Matt, if you'd agree with this, whether or not apparameters is a better stabilizer.

Speaker #6: I think most people can understand that even if they can't understand a specific in vitro assay. And the bigger question was how much more is that buying me in terms of downstream results and the serum TPR work was just the first part of that.

Tom Trimarchi: I think most people can understand that even if they can't understand a specific in vitro assay. The bigger question was, how much more is that buying me in terms of downstream results? The serum TTR work was just the first part of that. I think you're gonna 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.

Speaker #6: 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.

Speaker #3: Our next question comes from the line of Manny Furuar with Lyric Partners. Please go ahead.

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

Speaker #7: Hey, guys. Thanks for the question to wrap on results.

Mani Foroohar: Hey, guys. Thanks for the questions. Congrats on the results. In the aftermath of the tafamidis IP evolution and some clarity on genericization of taf, not acoramidis, walk us through operationally how you think about the development and commercial strategy for acoramidis into 2031, 2032 and beyond?

Speaker #5: In the aftermath of the tafamidus IP evolution, and some clarity on genericization, of TAF, not of apparameters, walk us through operationally how you think about the development and commercial strategy for apparameters.

Speaker #5: Into 2031-32 and beyond.

Chinmay Shukla: Yeah. Yeah. Hey, Mani. Thanks for the question. The clarity on Renaly mics IP is clearly a meaningful positive for BridgeBio and Attruby. You know, we now have at least 6 years of runway before genetics, which is more than enough time to reach peak share. Obviously, this all materially reduces any tail risk we would have to the NPV of the program. I don't think the resolution really changes our commercial strategy. We've always been focused on establishing Attruby as the treatment of choice in ATTR-CM, given its differentiated profile, and we're executing against that. I think you heard both Neil Kumar and Matt Outten talk about the bevy of literature that we are producing, as well as all the commercial activities which we are undertaking to really reinforce that differentiation and drive share.

Speaker #2: Yeah. Yeah. Hey, Manny. Thanks for the question. So the clarity on randomized ideas is clearly a meaningful positive for Bridge and Atrubi. We now have at least six years of runway before genetics, which is more than enough time to reach peak share.

Speaker #2: And obviously, this all materially reduces any tail risk we would have to the NPV of the program. I don't think the resolution really changes our commercial strategy.

Speaker #2: We've always been focused on establishing Atrubi as the treatment of choice in ACTRCM, given its differentiated profile. And we're executing against that. I think you heard both Neil and Matt talk about the bevy of literature that we are producing as well as all the commercial activities which we are undertaking to really reinforce that differentiation and drive share.

Speaker #2: I guess what I'll say at the end is just we've shared our beliefs that Atrubi will be a $4 billion drug. Last year, in our Q1 '25 earnings call, I don't think we've ever been more confident in that estimate.

Chinmay Shukla: I guess what I'll say at the end is just, you know, we've shared our belief that Attruby will be a $4 billion drug last year in our Q1 2025 earnings call. I don't think we've ever been more confident in that estimate. I actually think, if anything, there might be some room for potential upside. I think as Neil Kumar mentioned in his prepared remarks, Attruby is likely to keep growing even after 2032, given the ecosystem and channel dynamics that exist here with Part D. That's kind of what's driving our confidence. Yeah, I think it's a good thing for us. We've reduced the error bars on our valuation.

Speaker #2: I actually think if anything, there might be some room for potential upside. I think as Neil mentioned in his prepared remarks, Atrubi is likely to keep growing even after 2032, given the ecosystem and channel dynamics that exist here with Part B.

Speaker #2: And so that's kind of what's driving our confidence. So yeah, I think it's a good thing for us. We've reduced the error bars on our valuation.

Speaker #5: Yeah. And maybe I'll add to that. I mean, I personally did think, Manny, and we talked about it, that it would be 2035. So obviously, I was wrong on that.

Neil Kumar: Yeah. Maybe I'll add to that. I mean, you know, I personally did think, Mani, and we talked about it, that it would be 2035. Obviously I was wrong on that. That, that is a little bit of a discount, but not material, as I mentioned in my comments. The biggest thing is like, you know, all of these differentiating studies that we're running, it is really starting to resonate with clinicians, in addition to the fact that the access programs are superior. You know, I guess what your question is really alluding to is like, will we run a double-blind head-to-head? We still might. We reserve the option to do that. Certainly, we've been excited to do that in the context of either TTR levels or NT-proBNP.

Speaker #5: That is a little bit of a discount. But not a material, as I mentioned in my comments. And the bigger thing is all of these differentiating studies that we're running, it is really starting to resonate.

Speaker #5: With clinicians, in addition to the fact that the access programs are superior. So I guess what your question is really alluding to is will we run a double-blind head-to-head?

Speaker #5: We still might. We reserve the option to do that. Certainly, we've been excited to do that in the context of either TPR levels or NT-proBNP.

Neil Kumar: How we size like a hospitalization study is difficult if you look at the number of events. We're gonna have a strong look at the placebo arm of the upcoming opontin 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. You know, there's nothing obvious right now, so we'll just continue doing the roll of evidence studies, which I think are the best, honestly, characterization of sort of differential competitive dynamics.

Speaker #5: But how we size a hospitalization study is difficult if you look at the number of events. We're going to have a strong look at the placebo arm of the upcoming Apondrison 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.

Speaker #5: But there's nothing obvious right now. So let's continue doing the real-world evidence studies, which I think are the best, honestly, characterization of sort of differential competitive dynamics.

Speaker #3: Our next question comes from the line of Karen. I'm in with Piper Sandler. Please go ahead.

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

Speaker #8: Yeah. Hi, guys. Thanks for taking my questions. Can you walk us through the board's decision to authorize the $500 million share repurchase program and how you're thinking about balancing that against your investment in new launches and pipeline?

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

Speaker #8: And I guess 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 off. I mean, I think right now the focus is on focused execution against the pipeline that we have right now. You know, we've got, in discussions with investors, ample growth that has not been valued in the context of this company to date. I mean, the LGMD2I launch that I referred to, the ADH1 launch, the Acon launch even in its totality. I think, you know, we're projecting market share numbers well in excess of what typically a third mover gets in that space. You think about the consequential additional indications, both in terms of hypocon and importantly in terms of chronic hyperthyroidism that we're kicking off. We've got the Canavan program.

Speaker #5: Yeah, I can take that off. I mean, I think right now the focus is on focused execution against the pipeline that we have right now.

Speaker #5: We've got, in discussions with investors, ample growth that has not been valued in the context of this company to date. I mean, the LGMD2i launch that I referred to, the ADH1 launch, the ACOM launch even is fatality, I think.

Speaker #5: We're projecting market share numbers well in excess of what typically a third mover gets. In that space. And then you think about the consequential additional indications, both in terms of hypocon and importantly, in terms of chronic hyperparathyroidism that we're kicking off.

Speaker #5: And then we've got the Canavan program. So that constellation of activities is more than enough to drive long-term growth for any company. And that's kind of what we're focused on.

Neil Kumar: Like, that constellation of activities is more than enough to drive long-term growth for any company, and that's kind of what we're focused on. Can we fully finance all of that comfortably? We feel like we can. Therefore, beyond that, what ought we do with excess capital? We think the best, relative, you know, in terms of relative return, way that we deploy capital right now is into our own shares, just given the disconnect between intrinsic value and where we're trading. That's really what the discussion came down to. I know the normal way that a biotech would grow is say, Well, who cares? Share price is low.

Speaker #5: Can we fully finance all of that comfortably? And we feel like we can. And therefore, beyond that, what ought we do with excess capital?

Speaker #5: And we think the best, relative in terms of relative return, way that we can deploy capital right now is into our own shares, just given the disconnect between intrinsic value and where we're trading.

Speaker #5: So that's really what the discussion came down to. It's hard to I know the normal way that a biotech would grow is say, "Well, who cares?

Speaker #5: Share price is low. Let's go ahead and dilute everyone, and just keep going after science that we believe in. But that is not a reliable, sustainable, long-term model in my belief.

Neil Kumar: Let's 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, and nor is it one that we intend to employ here at BridgeBio.

Speaker #5: And nor is it one that we intend to employ here at BridgeBio.

Speaker #3: Our next question comes from the line of Corey Kasimo with Evercore ISI.

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

Cory Kasimov: Hey, good afternoon, guys. Thanks for taking the question. Great to see all the ongoing progress. I wanna follow up on this real world evidence that was referenced in both the press release and the prepared comments that reinforces ATTRUBY differentiation versus tafamidis. Can you kind of unpack, you know, what this real world data that you're seeing, 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.

Speaker #8: Hey, good afternoon, guys. Thanks for taking the question. Great to see all the ongoing progress. So I want to follow up on this real-world evidence that was referenced in both the press release and the prepared comments that reinforces Atrubi's differentiation versus TAFT.

Speaker #8: Can you kind of unpack what this real-world data that you're seeing and how it compares with what was demonstrated in the clinical trial setting?

Speaker #8: Is anything different now than it was, or just more of it? Thank you.

Speaker #5: Yeah. It's pretty different because recall that we had a significant left shift in our clinical trial. Our placebo outperformed the on-drug arm of ATTRACT.

Neil Kumar: Yes. It's pretty different because recall that we had a significant left shift in our clinical trial. Like, there was, you know, our placebo outperformed the on-drug arm of the trial. There, there's kinda no way for us to actually apples to apples go across, like, diuretic intensive case. Oh, by the way.

Speaker #5: So the kind of no way for us to actually apples to apples go across diuretic intensification. Oh, and by the way, even the use of diuresis and rhythm control meds and SGLT2i use in this population, all of that stuff has changed pretty markedly.

Tom Trimarchi: Even the use of diuresis and Renin-control meds and, you know, SGLT2i use in this population, all of that stuff has changed pretty markedly. It almost made it impossible, excepting the in-trial comparisons we could make between tafamidis and acoramidis with all of the available caveats there, where, and just as a reminder, acoramidis 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're seeing things like, you know, what we've mentioned in terms of diuretic intensification. We certainly didn't look at, you know, downstream kidney effects like the, you know, the NAVADA system did.

Speaker #5: So it almost made it impossible, excepting the in-trial comparisons we can make between Tefamidus and Acramidus with all of the available caveats there, where, I mean, this is a reminder, Acramidus outperformed Tefamidus in all aspects, which you did not see in Helios B.

Speaker #5: But 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.

Speaker #5: And here, you're seeing things like what we've mentioned in terms of diuretic intensification, which certainly didn't look at downstream kidney effects like the Nevada system did.

Tom Trimarchi: It's all resonant, I suppose, with the advantages that we think ATTRUBY has versus tafamidis in terms of mortality and hospitalization. It's just nice to see it actually play out in the real world.

Speaker #5: But it's all resonant, I suppose. With the advantages that we think acoramidis has versus tafamidis in terms of mortality—and hospitalization—it's just nice to see it actually play out in the real world.

Speaker #3: Our next question comes from the line of Salim Sayed with Mizuho. Please go ahead.

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

Speaker #9: Hey, guys. Thanks for the question and congrats on another great quarter. Just one from us maybe on Integratinib and Propel 3. So since you guys have had that read, I'm sure you must have done some market share work or at least spoken to additional folks in the ACOM community both on the clinician side and family side.

Salim Syed: Hey, guys. Thanks for the question, and congrats on another great quarter. Just one from us maybe on infigratinib and PROPEL 3. You know, since you guys have had that read, I'm sure you must 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. Curious what the feedback has been there. How has the additional feedback informed your expectations for the commercial opportunity? I believe you said previously you sort of think about achondroplasia as being a two and a half billion dollar TAM and maybe hypocon the same. Curious if you have any other color there to offer on the commercial opportunity. Thank you.

Speaker #9: Just curious what the feedback has been there? How has the additional feedback informed your expectations for the commercial opportunity? I believe you said previously you sort of think about ACON as being a two-and-a-half-billion-dollar TAM and maybe hypocon the same.

Speaker #9: Just curious if you have any other collar there to offer in the commercial opportunity. Thank you.

Speaker #5: Yeah. Thanks for the question, Salim. I think the feedback from the clinician and caregivers may have been overwhelmingly positive. ACPs are telling us that they're constantly being asked by families when the oral is coming.

Tom Trimarchi: Yeah. Thanks for the question, Salim. I think the feedback from the clinician and caregiver community has been overwhelmingly positive. You know, HCPs are telling us that they're constantly being asked by families when the oral is coming. You know, both from families who are on treatment today, but more importantly, those that have stayed on the sidelines, which as a reminder makes up about 70% to 80% of the US market. You know, the consistent best-in-class profile is continuing to resonate with these clinicians. You know, they understand that the AHB is best in class, the IC score is best in class. We have the most attractive safety profile and importantly, on proportionality, right?

Speaker #5: Both from families who are on treatment today, but more importantly, those who have stayed on the sidelines, which does a reminder, makes up about 70 to 80 percent of the US market.

Speaker #5: The consistent best-in-class profile is continuing to resonate with clinicians. They understand that AHB is best in class. The high T-score is best in class.

Speaker #5: We have the most attractive safety profile. And importantly, unproportionality, right? The proportionality data point is the one that's resonating most with clinicians because this is the only product that has a stat-fixed result in proportionality in a three to eight age group population.

Tom Trimarchi: The proportionality data point is the one that's resonating most with clinicians because this is the only product that has a stat sig result in proportionality in a 3 to 8 age group population. Which is a demonstration of how directly targeting FGFR3 impacts more than just height here in skeletal health. On that note, we will be releasing more data on how infigratinib is benefiting more than just height in some medical conferences later this year, some of which has never been seen before in a 52-week placebo-controlled trial. Stay tuned there. Ultimately, all this is to show that it really reinforces our belief that we will have a peak market share, potentially more than 65% market share, as validated by our current market research here.

Speaker #5: And this, which is a demonstration of how directly targeting FGFR3 impacts more than just height here is about health. And on that note, we will be releasing more data on how Integratinib is benefiting more than just height in some medical conferences later this year.

Speaker #5: Some of which has never been seen before in a 52-week placebo-controlled trial, so stay tuned there. Ultimately, all of this is to show that it really reinforces our belief that we will have a peak market share potentially north of 60% to 80% market share as valued by our market research here.

Speaker #3: Our next question comes from the line of Elimer with Barclays. Please go ahead.

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

Speaker #10: Hey, guys. Thanks for taking the question. Two from me. First, on Lim Gardel. So you submitted the Lim Gardel NDA very quickly from our mouth, about 150 days from top line, which is very fast compared to average.

Eliana Merle: Hey, guys. Thanks for taking my question. Two from me. First on limb-girdle. You submitted the limb-girdle NDA very quickly, from our math, about 150 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're preparing to get this drug into the hands of the limb-girdle community from day 1? A second question on the ATTR space. How are you thinking about what we will see from CARDIO-TTRansform, specifically trial stat sig, and, you know, the trial is very well-powered, but what's the hazard ratio that you think could be competitive, and how are you thinking about that?

Speaker #10: Can you walk us through where you stand on launch readiness and how you're preparing to get this drug into the hands of the Lim Gardel community from day one?

Speaker #10: And then a second question on the ATTR. Space. How are you thinking about what we will see from cardiotransform, specifically? Trial stat fig and the trial is very well powered.

Speaker #10: But what's the hazard ratio that you think could be competitive? And how are you thinking about that?

Tom Trimarchi: Hey, Eliana. This is Tom, good to hear from you. I'm gonna pass the limb-girdle one to Anna Wade here and then the CARDIO-TTRansform to Neil. First I want to say I'm really proud of our team for the quick turnaround on the NDA, and you can expect that level of efficiency from the next two as well. Over to Anna and then to Neil, the CARDIO-TTRansform answer.

Speaker #5: Hey, Ellie. This is Tom. Good to hear from you. So I'm going to pass the Lim Gardel one to Anna Wade here, and then the cardiotransform to Neil.

Speaker #5: But first, I want to say I'm really proud of our team for the quick turnaround on the NDA and you can expect that level of efficiency from the next two as well.

Speaker #5: But over to Anna and then to Neil with the cardiotransform.

Speaker #10: Thanks for the question, Ellie. And thanks for the kind word, Tom. So yeah, we're really excited. We have a commercial and sales leadership on board.

Anna Wade: Thanks for the question, Ellie, and thanks for the kind words, Tom. Yeah, we're really excited. We have our commercial and sales leadership on board. We're 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, as Neil mentioned, in March, where Dr. Katherine Mathews, a leading KOL in the field, presented our phase III interim analysis data, and we had incredibly positive feedback at the meeting about the compelling data package. Since NDA, we've heard about significant patient outreach to neuromuscular centers, and internally, we have received many inbound inquiries from both patients and physicians.

Speaker #10: We're getting ready to have the sales rep 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.

Speaker #10: In Q1, our major catalyst was the NDA conference, as Neil mentioned in March, where Dr. Kathy Matthews, a leading KOL in the field, presented our Phase 3 interim analysis data.

Speaker #10: And we had incredibly positive feedback at the meeting about the compelling data package. Since NDA, we've heard about significant patient outreach to neuromuscular centers and internally, we have received many inbound inquiries from both patients and physicians.

Speaker #10: Our focus right now is driving awareness of the Phase 3 data as well as emphasizing the importance of genetic diagnoses, leveraging responsive testing programs that are currently available so that day one of launch, we can be ready to get patients onto therapy.

Anna Wade: Our focus right now is driving awareness of the Phase III data, as well as emphasizing the importance of genetic diagnoses, leveraging responsive testing programs that are currently available, so that day one of launch we can be ready to get patients onto therapy.

Speaker #3: Our next question comes from.

Operator: Our next question comes.

Tom Trimarchi: Well, let me just Cardio. I think let me address her second question, which I think was on the CARDIO-TTRansform. We agree with you. It's a super well-powered trial. I mean, obviously against the primary, it looks good. Even in the subpopulation, like if you take the same point estimate from HELIOS-B on combo and you just take the Z-score and then you say how many more patients would you need, it's like, you know, it's 2 and a half times more to hit a P value of 0.05 or less, and they're pretty well powered for that. I actually expect that they'll hit on almost everything that they're interrogating. It comes back to, yeah, how do I cross-trial compare?

Speaker #5: I think let me address her second question, which I think was on cardiotransform. So yeah, we agree with you. It's a super well-powered trial.

Speaker #5: I mean, obviously, against the primary, it looks good. And even in the subpopulations, like if you take the same point estimate from Helios B on combo, and you just take the Z-score, and then you say how many more patients would you need?

Speaker #5: It's like two and a half times more to hit a p-value of 0.05 or less. And they're pretty well powered for that. So I actually expect that they'll hit on almost everything that they're interrogating.

Speaker #5: And so then it comes back to, yeah, how do I cross-trial, compare? How do I understand this? Knockdown technology. Part of that will be how much knockdown they get and are they able to improve on the PK profile?

Tom Trimarchi: You know, how do I understand this knockdown technology? Part of that will be how much knockdown they get and are they able to improve on the PK profile? Because, you know, the reason that, you know, performs similarly to AMVUTTRA or vutrisiran in the HELIOS-B trial, has to do with the timing it takes for vutrisiran to get to mean max knockdown. You know, it takes a lot longer than I would have expected. We'll see if that's the case with their drug. I think overall, honestly, CARDIO-TTRansform 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 mean, I think people are gonna reach for stabilizers first line anyway, number one.

Speaker #5: Because I think the reason that TAF performs similarly to InVitra or Boot in the Helios B trial has to do with the timing it takes for Boot to get to mean max knockdown.

Speaker #5: So it takes a lot longer than I would have expected. So we'll see if that's the case with their drug. I think overall, honestly, cardiotransform has more to do with the commercial dynamics with all myeloma than it does with us, especially given the combo data that I just told you about.

Speaker #5: I mean, I think people are going to reach for stabilizers first line anyway, number one. Number two, I think when they're failing stabilizers, they're going to want a better stabilizer on board in combination.

Tom Trimarchi: Number two, I think when they're failing stabilizers, they're gonna want a better stabilizer on board in combination. If that combo arm does it, I don't see it having a meaningful, super meaningful effect for us. Again, I mean, you know, I think from a biochemical standpoint, you always want to preserve the native tetramer. We're seeing more and more information about that. You know, I'm surprised people haven't been looking at the publicly available FAERS database and what do things look like when you're knocking things down versus actually stabilizing and all consistent with the 225,000 plus patient studies that we've seen out there suggesting that higher levels of CRCPR are better for you. I understand that in a short trial, those signals aren't necessarily resolvable.

Speaker #5: So if that combo arm does hit, I don't see it having a meaningful, super meaningful effect. For us. Again, I mean, I think from a biochemical standpoint, you always want to preserve the native tetramer.

Speaker #5: We're seeing more and more information about that. I'm surprised people haven't been looking at the publicly available FAIRS database and what do things look like when you're knocking things down versus actually stabilizing and all consistent with the two 25,000-plus patient studies that we've seen out there suggesting that higher levels of serum TPR are better for you.

Speaker #5: But I understand that in a short trial, those signals aren't necessarily resolvable. But I think over the longer period of time, stabilizers will continue to be frontline and then in combination, I think we'll have a pretty good say there too.

Tom Trimarchi: I think over the longer period of time, stabilizers will continue to be frontline and then in combination, I think we'll have a pretty good stay there too. That's kinda That's how we're thinking about it. We do think CARDIO-TTRansform will be positive just given the patient numbers. On your hazard ratio question, I mean, obviously, 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, are gonna be a vast majority of the events will be hospitalization, not mortality. I think that bar, 50% reduction in hospitalization is kinda what I'll be looking for.

Speaker #5: But that's how we're thinking about it. We do think cardiotransform will be positive just given the patient numbers. On your hazard ratio question, I mean, obviously, we think the bar is relative risk reduction of 42%.

Speaker #5: And risk reduction of 50% on hospitalization, which I suspect if this study is consistent with the rest of the modern studies, are going to be a vast majority of the events will be hospitalization, not mortality.

Speaker #5: So I think that that bar, 50% reduction in hospitalization, is kind of what I'll be looking for.

Speaker #3: Our next question comes from the line of Anupam Rama with JPMorgan. Please go ahead.

Operator: Our next question comes from the line of Anupam Rama with JP Morgan. Please go ahead.

Speaker #11: Hey, guys. Thanks so much for taking the question and congrats on the quarter. Quick encounter one here. Know the NDA is on track here for the first half of this year.

Anupam Rama: Hey, guys. Thanks so much for taking the question, and congrats on the quarter. Quick on acoramidis here. Know the NDA is on track here for H1 of this year. Then the press release highlighted nearly 2,000 now identified, identifiable patients with 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.

Speaker #11: And then the press release highlighted nearly 2,000 now identified identifiable patients with 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?

Speaker #11: Thanks so much.

Ananth Sridhar: Daniel, good to hear from you. Yeah. I would say that the foundation of everything that we're doing around patient identification is really awareness. Awareness of the disease as an important distinct subset within hypopara, and then of course, awareness of our drug Caloryx and the wonderful effect it can have for these patients. I think a major catalyst for awareness is gonna be the upcoming presentation at ECE next week, followed by US presentations later in the year and then hopefully a very high impact publication as well. In the background, I think there's some important tactics and strategies that we continue to employ. First, as you mentioned, the ICD-10 code is a huge advantage to us here. Many rare diseases don't have an ICD-10 code.

Speaker #5: Thank you. Good to hear from you. Yeah. So I would say that the foundation of everything that we're doing around patient identification is really awareness.

Speaker #5: Awareness of the disease as an important distinct subset within hypothyroid. And then, of course, awareness of our drug and calorie and the wonderful effect it can have for these patients.

Speaker #5: I think a major catalyst for awareness is going to be the upcoming presentation that you see next week. Followed by US presentations later in the year and hopefully a very high-impact publication as well.

Speaker #5: But in the background, I think there's some important tactics and strategies that we continue to employ. First, as you mentioned, the ICD-10 code is a huge advantage to us here.

Speaker #5: Many rare diseases don't have an ICD-10 code. We're lucky to have one that's been in place for a couple of years already. So there's a good amount of data in that.

Tom Trimarchi: We're lucky to have one that's been in place for a couple of years already, so there's a good amount of data in that. 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 into the offices, spread awareness, and also make sure the patients they think they have are appropriately diagnosed with other sponsor genetic tests or other commercially available genetic tests. Then third, and I think this has been a bigger driver of identification than I would have thought is family tracing. Makes sense when you consider this is a dominant condition, so there's 50% chance of passing on.

Speaker #5: 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 into the offices, spread awareness, and also make sure that the patients they think they have are appropriately diagnosed with our sponsor genetic tests or other commercially available genetic tests.

Speaker #5: And then third, and I think this is been a bigger driver of identification than I would have thought, is just family tracing. Makes sense when you consider this is a dominant condition.

Speaker #5: So there's 50% chance of passing it on. So when we tend to find one person with this condition, if they look and go to a family event, we find out that many of them have brothers, cousins, aunts, and uncles also have the condition.

Tom Trimarchi: When we tend to find one person with this condition, if they look and go to a family event, we find out that many of them, you know, brothers, cousins, aunts and uncles also have the condition. That's been a real valuable source of patient identification as well. We'll continue all these efforts and accelerate them as we approach launch.

Speaker #5: So that's been a real valuable source of patient identification as well. So we'll continue all these efforts and accelerate them as we approach launch.

Speaker #3: Our next question comes from the line of Derek Archullo with Wells Fargo. Please go ahead.

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

Derek Archila: Hey there. Thanks for taking my questions, and congrats on the progress. Just a follow-up on Infigratinib. You know, some recent commentary and some of the early KPIs from the ubelel launch seem positive and maybe, you know, early validation of kind of this market expansion thesis. Just curious how you think about Infigratinib's profile versus the injectables and how this could further accelerate, you know, this market expansion potential. Thanks.

Speaker #12: Hey there. Thanks for taking my questions and congrats on the progress. Just a follow-up on the progranulin. Some recent commentary and some of the early KPIs from the UBWell launch—early validation of, kind of, this market expansion thesis.

Speaker #12: So just curious how you think about infragranulin's profile versus the injectables and how this could further accelerate this market expansion potential. Thanks.

Speaker #5: Yeah. No, thanks, Derek. Yeah, I think ultimately physicians and families are excited about the total package of infragranulin, right? Not just one thing. It starts with a 2.1-centimeter change from baseline HP.

Tom Trimarchi: Yeah. No, thanks, Derek. You know, I think ultimately physicians and families are excited about the total package of infigratinib, right? Not just one thing. It starts with a 2.1 centimeter change from baseline HB. You know, the largest effects shown across any of the three phase IIIs, which was remarkable, weight consistent across the age groups. Of course, as mentioned earlier, the only stat sig improvement on proportionality, a demonstration of the importance of directly engaging FGFR3. You have a differentiated safety profile, right? With no injection site reactions, no symptomatic hypertension, no hypertriglycerides. I think the safety differentiation is further playing out given the increasing and concerning signals of SKIFD and femur fractures, which are both looking like potential CMV collapse effects. You know, the bow on top of this for us is being the daily oral.

Speaker #5: The largest effects shown across any of the three phase threes, which was remarkable weight consistent across the age groups. Then, of course, as I mentioned earlier, the only statistic improvement on personality is demonstration of the importance of directly engaging FGFR3.

Speaker #5: And then you have a differentiated safety profile, right? With no injection site reactions, no symptomatic hypertension, no hypertrichosis, and I think the safety differentiation is further playing out given the increasing and concerning signals of skiffy and femur fractures which are both looking like potential CMV collapse effects.

Speaker #5: The bone on top of this for us is being the 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 three to four times at year five after launch.

Tom Trimarchi: Based on historical benchmarks, we know that when oral enters a market with only injectables, it expands the market on average by 3 to 4 times at year 5 after launch. Ultimately, the families here will have a choice of either 365 injections a year, 52 injections a year or 0, and I know which one most families would prefer.

Speaker #5: So ultimately, the families here will have a choice of either 365 injections a year, 52 injections a year, or zero. I know which one those families would prefer.

Speaker #3: Our next question comes from the line of Paul Choi with Goldman Sachs. Please go ahead.

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

Speaker #13: Hi. Thank you. And good afternoon. And thanks for taking the question. Sticking with the infragranulin, I want to ask with regard to the PROPEL infant and toddler study in patients who are newborns or up to two years old.

Paul Choi: Hi, thank you and good afternoon, and thanks for taking the question. Sticking with the infigratinib, I want to ask with regard to the.

Paul Choi: PROPEL Infant & Toddler study in patients who are newborns or up to 2 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? just, you know, how you think about timing for that potentially being completed and being added to the label. Thank you.

Speaker #13: 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?

Speaker #13: And just how you think about timing for that potentially being completed and being added to the label? Thank you.

Speaker #5: Yeah. Thanks for the question, Paul. Yeah, definitely. I think there's a lot of excitement from sites and from families after seeing the phase three PROPEL3 data, right?

Neil Kumar: Thanks for the question, Paul. Definitely, I think there's a lot of excitement from sites and from families after seeing the phase III PROPEL 3 data, right? Just kind of what we know from the field shows that the earlier you intervene, the more likely your impact likely to impact clinical outcomes. I think we've seen definitely a burst of excitement there. You know, we'll provide an update on timing probably later on once we have some more clarity once this program progresses.

Speaker #5: Just kind of what we know from the field shows that the earlier intervening, the more likely your impact likely to impact clinical outcomes. I think we've seen definitely a burst of excitement there.

Speaker #5: So we'll provide an update on timing probably later on once we have some more clarity once this program progresses.

Speaker #3: Our next question comes from the line of Andrew Tsai with Jefferies. Please go ahead.

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

Speaker #14: Hey, thanks for all the updates. Congrats on the execution. I have a bigger picture question for you guys on your broader pipeline. Now that you have succeeded across four major programs, all the way through phase three, as investors think about the sustainability of your R&D engine, maybe talk about how you're currently thinking about the next wave of development beyond your portfolio and how much you're open to adding more to the pipeline in the near term and what indication areas you could be interested in.

Andrew Talliot: Hey, thanks for all the updates. Congrats on the execution. I have a bigger picture question for you guys 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, maybe talk about how you're currently thinking about the next wave of development beyond your portfolio and how much you're open to adding more to the pipeline in the near term and what indication areas you could be interested in. Thank you.

Andrew Tsai: Hey, thanks for all the updates. Congrats on the execution. I have a bigger picture question for you guys 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, maybe talk about how you're currently thinking about the next wave of development beyond your portfolio and how much you're open to adding more to the pipeline in the near term and what indication areas you could be interested in. Thank you.

Speaker #14: Thank you.

Speaker #5: Yeah. Thanks for the question. I think right now, as I think a lot of our comments and actions have been consistent with, we're very focused on executing the opportunity in front of us.

Neil Kumar: Yeah, thanks for the question. I think right now, as I think, you know, a lot of our comments and actions have always been consistent with, we're very focused on executing the opportunity in front of us. It's 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's gonna take, certainly against our lean backbone, all of the focus that we have. You know, I also mentioned earlier that we have pretty significant additional opportunities associated with every single one of our drug products, including some that we haven't talked about vis-à-vis ATTRUBY. I actually think that there's some pretty interesting stuff to do.

Speaker #5: It's not often that a biotech will launch three different products in three different indication settings alongside a pretty competitive market at the same time.

Speaker #5: And that's going to take certainly against our lean backbone all of the focus that we have. I also mentioned earlier that we have pretty significant additional opportunities associated with every single one of our drug products including some that we haven't talked about vis-à-vis Atruvi.

Speaker #5: So I actually think that there's some pretty interesting stuff to do. We have an internal pipeline obviously prosecuting programs in ADPKD, LMNA, Dilated Cardiomyopathy, and the depleter antibody program in ATTR cardiomyopathy.

Neil Kumar: We have an internal pipeline, obviously prosecuting programs in ADPKD, LMNA, nodal cardiomyopathy, and the depleter antibody program in ATTR cardiomyopathy. That's additionally, you know, programs that are very, very capitally efficient, but programs that we have an eye on to date. And then we've got backup programs against all of our current pipeline programs so that we can do what's right for the patient and physician community to continue to serve them as long as possible. All of those things put together, I would say, represent the menu of activities that we're interested in in the near term. Obviously, we're students of the genetic disease space.

Speaker #5: So that's additionally programs that are very, very capital efficient, but programs that we have an eye on today. And then we've got backup programs against all of our current pipeline programs so that we can do what's right for the patient and physician community to continue to serve them as long as possible.

Speaker #5: So all of those things put together, I would say represent the menu of activities that we're interested in in the near term. Obviously, we're students of the genetic disease space.

Neil Kumar: BridgeBio has a significant stake in another company called GondolaBio, which is really kind of one of our sister companies, and that has 17 programs that I think we've gone through ranging from phase II, all the way back to the preclinical setting. It's a very, very exciting slate of small molecules, ASOs, antibodies, all targeting well-described genetic conditions at their source.

Speaker #5: French Bio has a significant stake in another company called Gondola Bio which is really kind of one of our sister companies. And that has 17 programs that I think we've gone through ranging from phase two all the way back to the preclinical setting.

Speaker #5: And it's a very, very exciting slate of small molecules ASOs, antibodies, all targeting well-described genetic conditions at their source. And so I would say that we're happy for that to be sort of 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.

Neil Kumar: We're happy for that to be sort of an off-balance sheet R&D exercise for now, as is BridgeBio Oncology Therapeutics, and to really focus on what we need to focus on here, which is 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.

Speaker #5: Which is 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 packed us for many years.

Speaker #3: Our next question comes from the line of Daniel Breel with Truist. Please go ahead.

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

Speaker #15: Hey, guys. Thanks for the question and 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.

Danielle Brill: Hey, guys. Thanks for the question and 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 pretty meaningful step up from prior quarters where I think it was more in the 4,000 new patients range. Just curious what's driving the step up here, moving forward, how should we think about the size of the quarterly patient pool that you're actively competing for? Thank you.

Speaker #15: If I recall correctly, this represents a pretty meaningful step up from prior quarters where I think it was more in the 4,000 new patients range.

Speaker #15: Just curious what's driving this step up here and moving forward, how should we think about the size of the quarterly patient pool that you're actively competing for?

Speaker #15: Thank you.

Speaker #5: Yeah, sure. I mean, I think this market continues to grow. Somewhere between I think our internal numbers are somewhere between 5,000 and 6,000 in that range.

Neil Kumar: Yeah, sure. I mean, I think this market continues to grow, somewhere between like, you know, I think our internal numbers are somewhere between 5,000 and 6,000, you know, in that range, a Q in terms of these new patient starts, or new patients to brand. Actually, I don't think we have 4,500 for the last Q. I think it was above 5,000. A little bit of this math has to do with like us guessing for our competitors what the inventory holdback was or what the inventory buys were and things of that nature. Can never get it fully right. We can get obviously our own patient numbers fully right. That does suggest, yeah, continued growth.

Speaker #5: A quarter in terms of these new patient starts or new patients to brand. So actually, I don't think we have 4,500 for the last quarter.

Speaker #5: I think it was above 5,000. But 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.

Speaker #5: So you can never get it fully right. We can get obviously our own patient numbers fully right. But that does suggest, yeah, continued growth.

Speaker #5: I mean, will it continue to grow? I think so. Obviously, there are 250,000 patients with ATTR cardiomyopathy at the low end in the US. And I think we're doing a better job of three things.

Neil Kumar: I mean, will it continue to grow? I think so. Obviously, there's 250,000 patients with ATTR cardiomyopathy at the low end in the US. I think we're doing a better job of three things. One is making sure that the algorithms are in EMR so that people think to look for these patients. You know, there's all the tracer AI stuff that we've been doing, other algorithms that individual healthcare systems have been putting forth to just get people to think, maybe this is a ATTR patient. That's one. Second is driving genetic testing into variant-heavy populations. That's been helpful as well.

Speaker #5: One is making sure that the algorithms are in easy MRs so that people think or look for these patients. There's all the tracer AI stuff that we've been doing other algorithms that individual healthcare systems have been putting forth to just get people to think, maybe this is a TDR patient." So that's one.

Speaker #5: Second is driving genetic testing into variant heavy populations that's been helpful as well. And the third and probably the best is just broad physician awareness and education through speaker bureaus, really getting out into the captive practices and the community practices to educate them more.

Neil Kumar: Certainly probably the best is just broad physician awareness and education through speaker bureaus, really getting out into the captivated practices and the community practices to educate them more. All of that's positive. I'd say on the negative side, we've heard quite a bit about this PYP shortage, the technetium scan, one of the ways that you can we can do the scans to get a definitive diagnosis alongside the ELISA assay.

Speaker #5: So all of that's positive. I'd say on the negative side, we've heard quite a bit about this PYP shortage that technician scan one of the one of the ways that you can do the scans to get a definitive diagnosis alongside the ELISA assay.

Speaker #5: So that we have to keep an eye on. We've heard about that before. Like in 2025, we heard about that, I think, in the second or third quarter.

Tom Trimarchi: That we have to keep an eye on. You know, we've heard about that before, like in 2025, we heard about that, I think, in Q2 or Q3. You know, one of the mid-quarters and the market continued to grow. We'll keep an eye on that. That's resolvable. There's 3 major suppliers of that. I think I expect in years going forward, we shouldn't see much more of this sort of, this supply chain iteration around PYP availability. Yeah, I guess long story short, I believe that I think Evercore put out a nice analysis of this 3 years ago or so.

Speaker #5: So one of the mid-quarters and the market continues to grow. But we'll have to keep an eye on that. That's resolvable. There's three major suppliers of that.

Speaker #5: And then I think I expect in the years going forward, we shouldn't see much more of this sort of this supply chain iteration around PYP availability.

Speaker #5: But yeah, I guess, long story short, I believe that you should see—I think Evercore put out a nice analysis of this three years ago or so.

Tom Trimarchi: It was like you should see super linear growth in identification given the number of patients that we believe having geographic autopsy, coupled with the number of sponsors in the playing field and, you know, the availability of reasonable testing. I would expect that trend to that positive trend to continue with some, you know, with some error of ours quarter to quarter.

Speaker #5: It was like, "You should see super linear growth in identification given the number of patients that we believe have ATTR cardiomyopathy coupled with the number of sponsors in the playing field and the availability of reasonable testing." I would expect that trend to that positive trend to continue.

Speaker #5: With some error bars, quarter to quarter.

Speaker #3: Our next question comes from the line of Jason Zemanski with Bank of America. Please go ahead.

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

Speaker #16: Good afternoon. Congrats on the great progress and thanks for taking our question. Maybe one more on in Colorado if we may. As you look beyond the ADH1 opportunity to sort of the broader chronic hypoparathyroidism opportunity, how are you thinking about in Colorado's positioning?

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

Speaker #16: I guess relative to the parathyroid hormone replacement therapies? Is there a particularly attractive subgroup to target or are you looking at sort of the broader opportunity as a whole?

Speaker #16: And then maybe if you could talk about some of the pricing implications of pursuing a market that maybe looks a little bit like 25,000 patients in the US and EU to versus 200,000.

Speaker #16: Thanks.

Speaker #5: Yeah. Hey, Jason. It's great to hear from you and thank you for the question. We look forward to seeing you next week at the conference.

Chinmay Shukla: Hey, Jason. It's great to hear from you, and thank you for the question. We look forward to seeing you next week at the conference. In terms of encaleret and chronic hypoparathyroidism, when we did our market research, it's really three things which drove our excitement about the opportunity. The first is that this will be the first oral option available in this chronic setting. The ability to give patients freedom from injection site reactions and all the pain that comes with it is something that resonates very well, I would say. The second thing is, if you look at the current options available, you lose an effect in terms of blood calcium normalization, but you don't really see that effect on urine calcium normalization.

Speaker #5: So in terms of in Colorado and chronic hypoparathyroidism, when we did our market research, it's really three things which drove our excitement about the opportunity.

Speaker #5: The first is that this would be the first oral option available in the chronic setting. And so the ability to give patients freedom from injection site reactions and all the pain that comes with it is something that resonates very well, I would say.

Speaker #5: The second thing is if you look at the current options available, you do see an effect in terms of blood calcium. Normalization, but you don't really see that effect on urine calcium normalization.

Speaker #5: And I think within calories, 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 two trial where we had around 80% of the patients normalizing both blood and urine calcium.

Chinmay Shukla: I think with encaleret, we have a very unique profile where we could normalize potentially both blood and urine calcium. We saw that in our healthy small phase II trial where we had, you know, around 80% of the patients normalizing both blood and urine calcium. These were patients who were very sick and did not have the parathyroid gland or any amount of the hormone. Really the third thing is that there is a potential safety risk in terms of bone resorption from giving PTH at high levels for the whole day. I think we could completely avoid that. I think actually the ADH1 readout significantly de-risks that ox profile for encaleret.

Speaker #5: And these were patients who were very sick and did not have the parathyroid gland or any amount of the hormone. And then really the third thing is that there is a potential safety risk in terms of bone resorption from giving TTH at high levels for the whole day.

Speaker #5: And so I think we could completely avoid that. And I think actually the ADH1 readout significantly de-risked the toxic profile for calories. And so I think that those three things: oral, urine calcium normalization, and potential benefit on safety is really why we think we can compete and get a reasonable share even in the chronic hypopara market.

Chinmay Shukla: I think that those three things, oral urine calcium normalization and potential benefit on safety is really why we think we can compete and get a reasonable share even in the chronic hypopara market, obviously, assuming that the trials work.

Speaker #5: Obviously, assuming that the trials work.

Speaker #3: Our next question comes from the line of Shannon LeMan an with Morgan Stanley. Please go ahead.

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

[Analyst] (Morgan Stanley): Hi, this is Morgan on for Sean. Thanks for taking our question. Can you just remind us specifically for infigratinib and achondroplasia, what kind of, if any, commercial preparations are taking place from BridgeBio? Thank you.

Speaker #17: Hi, this is Morgan on for Sean. Thanks for taking our question. Can you just remind us specifically, for infigratinib and achondroplasia, what kind of, if any, commercial preparations are taking place from BridgeBio?

Speaker #17: Thank you.

Speaker #5: Oh, thanks for the question. I think we've done a lot doing both strong commercial and medical leadership here, right? Bringing on for both sides of the business leadership has experience in both second and third market with security data, especially with experience kind of launching an oral.

Tom Trimarchi: Thanks for the question. I think we've done a lot to really build strong commercial and medical leadership here, right? Bringing on for both sides of the business, leadership that has experience in both second and third market with security data, especially with experience kind of launching oral. We've also brought a lot of experience with certain groups with skeletal dysplasia experience as well. Ultimately, I think right now we're trying to make sure that we get the word out, not just to the leading geneticists and AMCs, but also to the broader kind of community pediatric endocrinologists who are really excited about having an oral option, especially for families who have, you know, if they're not seeing kind of these super specialized centers of care, are more interested in having something that's easier for families to administer as well.

Speaker #5: We've also brought a lot of experience from groups with skills displayed to experience as well. So ultimately, I think right now we're trying to make sure that we get the word out, not just to the leading geneticists and AMCs, but also to the broader kind of community pediatric endocrinologists who are really excited about having an oral option, especially for families who have if they're not seeing kind of these super specialized centers of care, more interested in having something that's easier for family to administer as well.

Speaker #5: So a lot of information going on on that front.

Tom Trimarchi: A lot of exploration going on in that front.

Speaker #18: Yeah. I think to remember, we have a lot of the teams in place from the Atrubi launch that can also help with the future launches for all of the indications.

Matt Outten: Yeah. I think to remember, we have a lot of the teams in place from the ATTRUBY launch that can also help with the future launches for all of the indications. Think about market access with the payers, the pharmacies. These are all the same individuals. We have relationships with all of those people, are able to launch quickly, I think, as a result of that and get access and coverage.

Speaker #18: Think about market access with the payers, the pharmacies. These are all the same individuals and we have relationships with all of those people. And are able to launch quickly, I think, as a result of that and get access and coverage.

Speaker #3: Our next question comes from the line of John Boyle with William Blair. Please go ahead.

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. Wondering if you've had any interactions with them, and if you could speak to how the infigratinib profile is resonating there.

Speaker #19: Hi, team. Thanks so much for taking our question and congrats on a strong quarter. So patient advocacy groups for a chondroplasia seem to have a pretty big voice in the indication.

Speaker #19: So wondering if you've had any interactions with them and if you could speak to how the Invegradinib profile is resonating there.

Speaker #5: Yeah. Again, thanks for the question. I think that's just been a core tenet of how we've been developing drugs since the very beginning. We've been working alongside advocacy groups, both in the United States and internationally, on making sure that their input and voice is implemented in our development program and how we think about influence.

Tom Trimarchi: Thanks, thanks for the question. I think that's just been a core tenet of how we've been developing the drug since the very beginning. You know, we've been working alongside advocacy groups, both in the United States and internationally, on making sure that their input and voice is implemented in our development program and how we think about endpoints. You know, 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, kind of post-label extension program. I think they've been really wonderful partners with us, and we anticipate that persisting through commercialization and launch as well.

Speaker #5: 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 are hoping legal extension program.

Speaker #5: And I think they've been really wonderful partners with us and we anticipate that persisting through commercialization and launch as well.

Speaker #3: Thank you. And ladies and gentlemen, that concludes our Q&A session. Now we'll now turn the call back over to the BridgeBio team for closing remarks.

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

Speaker #20: Thank you, everyone, for your questions today. We really appreciate your interest and look forward to updating you again next quarter.

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.