Q1 2025 BridgeBio Pharma Inc Earnings Call
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Operator: And I know no one can swing the blues like Glenn Williams can Well, I sat down to hold our singing As they were taking down the tent The stars above the barren trees Was his only aria And charcoal gypsy maidens Can strut their feathers well Good afternoon, I will be your conference operator today. All lines have been placed on me 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.
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Speaker Change: 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.
Speaker Change: I'd like to ask a question. Please press star followed by the number one on your telephone keypad.
Operator: If you would like to withdraw your question, please press star 1 again. Thank you.
Speaker Change: If you would like to withdraw your question. Please 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 laws, including but not limited to statements about <unk> future operating and financial performance business plans and prospects and strap.
Operator: Before we begin, I would like to remind everyone that today's call may contain forward-looking statements within the meaning of the federal securities laws, including but not limited to statements about Bridgebio's future operating and financial performance, business plans and prospects and strategies. 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 Change: <unk>.
Speaker Change: These statements are based on current expectations and assumptions that are subject to risks and uncertainties.
Speaker Change: Which could cause actual results to differ materially from those expressed or implied in these forward looking statements.
Operator: For a discussion of these risks and uncertainties, please refer to the Disclosure Interface earnings release and Bridgebio's periodic reports and FEC 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.
Speaker Change: For a discussion of these risks and uncertainties. Please refer to the disclosure in today's earnings release, Enbridge Bios periodic reports and SEC filings.
Speaker Change: Statements made here are based on information available to <unk> as of today and the company undertakes no obligation to update any forward looking statements made during this call except as required by law.
Operator: With that completed, Bridgebio, you may begin your conference.
Speaker Change: <unk> completed bench bio you may begin your conference.
Chinmay Shukla: Good afternoon, and thank you for joining Bridgebio's Q1 2025 Earnings Conference Call. This is Chinmay Shukla, VP of Strategic Finance at Bridgebio. Today, we will discuss our financial results for the first quarter of 2025 and provide an overview of how we measure success in our business.
Speaker Change: Good afternoon, and thank you for joining goodbye.
Speaker Change: Q1, 'twenty earnings conference call.
Speaker Change: This is Jed my surplus VP of strategic finance had been smile.
Speaker Change: So David will discuss our financial results for the fourth quarter of 2025 and <unk>.
Speaker Change: Provide an overview of how we measure success in our business will provide insight into the early results from the launch of a <unk> as well as provide an update on our pipeline, which has three phase II readouts expected in the next year, including our efforts in expansion indications such as in chronic hyperparathyroidism today's call will feature Neel Kumar.
Chinmay Shukla: We'll provide insight into the early results from the launch of Atrubi, as well as provide an update on our pipeline, which has three phase three readouts expected in the next year, including our efforts in expansion indications, such as in chronic hypoparathyroidism.
Chinmay Shukla: Today's call will feature Neil Kumar, Chief Executive Officer, Matt Oughton, Chief Commercial Officer, and Tom Tremarkey, President and Chief Financial Officer.
Matt Outten: Chief Executive Officer, Matt Outten, Chief commercial officer, and Tom to Marquee, President and Chief Financial Officer.
Chinmay Shukla: For the Q&A portion of the call, Anant Sridhar, Chief Operating Officer of Bridgebio CardioRenal, and Justin To, Chief Executive Officer of Bridgebio Schedule Displasias, will also join. Following the remarks by our team, we will open up the call for Q&A.
Speaker Change: For the Q&A portion of the call and on SEDAR, Chief operating officer, Rich myocardial email and Justin <unk> Chief Executive Officer of Bridge Biocatalyst Michels will also join following the remarks by our team we will open up the call for Q&A.
Nisha: With that, I'll turn the call over to Nisha.
Speaker Change: With that I'll turn the call over to Neil.
Nisha: Thanks to everyone on the line for the time and welcome to our first earnings call. We're grateful to open up another dimension of our dialogue with our investor partners. You've collectively allowed us to deliver for the patients we serve, and we look forward to your feedback on how to continually improve our discussion in this setting.
Neil: Thanks to everyone on the line for the time and welcome to our first earnings call were grateful to open up another dimension of our dialogue with our Investor partners.
Neil: Collectively allowed us to deliver for the patients we serve and we look forward to your feedback on how to continually improve our discussion in this setting.
Nisha: Today, we know the star of the show is the Atruvi launch, and the good news is that the brand is delivering for patients and the business. 36.7 million in revenue this past quarter suggests that clinicians and patients are resonating with our differentiated clinical efficacy, safety, and accessibility. We continue to educate on our therapeutic impact, which comes as early as three months with a 42% relative risk reduction on cardiovascular hospitalization and mortality at 30 months, including a 50% reduction in cardiovascular hospitalization at that same time point. All these best-in-class point estimates are available at the lowest price point in the marketplace.
Neil: Today, we know the star of the show is the <unk> launch and the good news is that the brand is delivering for patients and the business $36 $7 million in revenue. This past quarter suggest that clinicians and patients are resonating with our differentiated clinical efficacy safety and accessibility.
Neil: We continue to educate on our therapeutic impact which comes as early as three months with a 42% relative risk reduction on cardiovascular hospitalization and mortality at 30 months, including a 50% reduction in cardiovascular hospitalization at the same time point.
Neil: All these best in class point estimates are available at the lowest price point in the marketplace.
Nisha: There's much to like there.
Nisha: And we're aware that there's much work yet to be done. You'll hear about some of that work today as it pertains both to commercial tactics and further development work.
Neil: As much to like there and we're aware that there is much work yet to be done youll hear about some of that work today as it pertains both the commercial tactics and further development work.
Nisha: We add to a truly good news the continued progress across our pipeline of three additional blockbuster products. Our trials in limb girdle muscular dystrophy type 2i, achondoplasia, and ADH1 remain on track with low dropout rates, frequent data collection, and positive site audits, all in preparation for an efficient NDA submission should the data be positive. Furthermore, key additional markets are being opened up. Our hypochondroplasia trial enrolled with incredible speed, and we now have first patient in there. Our work within Calorit in the hyperparathyroid setting positions it well for a registrational trial, which if successful, could pave the way for the first oral compelling solution in that space.
Neil: We add to attribute good news the continued progress across our pipeline of three additional blockbuster products our trials in limb girdle muscular dystrophy type <unk>, a contemplation and ADH one remain on track with low dropout rates frequent data collection and positive site audits all in preparation for an efficient NDA submission should the data.
Neil: Positive.
Neil: More key additional markets are being opened up our hypo contemplating a trial enrolled with incredible speed and we now have first patient in there our work within calories in the hypothyroidism setting positions it well for a registrational trial, which if successful could pave the way for the first oral compelling solution in that space.
Nisha: Before we move to discussion into further specifics regarding the business, and since this is our first time hosting this type of call, I wanted to briefly address how we measure the performance of our business on an ongoing basis. It's nice that this quarter was a success, but there will be harder quarters, I am sure, and through it all, you should expect for us to communicate consistently and in terms of net gains or losses in NPV. For instance, in this last quarter, our NPV increased by 9% given changes in the following variables. Number one, the time factor of money, especially given the late stage of our portfolio.
Neil: Before we move to a discussion into further specifics regarding the business and since this is our first time hosting this type of call.
Neil: Wanted to briefly address how we measure the performance of our business on an ongoing basis.
Neil: Nice that this quarter was a success, but there will be harder quarters, I am sure and through it all you should expect for us to communicate consistently and in terms of net gains or losses in NPV.
Neil: Instance, in this last quarter, our MTV increased by 9% given changes in the following variables number one the time factor of money, especially given the late stage of our portfolio number two a higher than anticipated revenue driven by a faster uptake of a treaty than we anticipated and likely a slightly higher peak year share by volume.
Nisha: Number two, a higher and anticipated revenue driven by a faster uptake of a true V than we anticipated, and likely a slightly higher peak year share by volume than our market research had projected. Third, the success of our small study in HP and other sponsors setbacks in the space of oral HP medicines. Fourth, the early enrollment of our run-in of our hypochondroplasia study, moving our timelines in for that program. And fifth, a slight decrease in our modeled cost of capital, given our recent convertible offering. There were MPV drags, most prominently including tariffs. However, these have a close to negligible effect at less than 1% of our overall MPV.
Neil: Then our market research had projected.
Neil: Third the success of our small study and HP and other sponsors setbacks in the space of orally <unk> medicines.
Neil: The early enrollment of our run hand of our Hypo contemplated study moving our timelines in for that program.
Neil: And fifth a slight decrease in our model and cost of capital given our recent convertible offering.
Neil: So we're MPV drags most prominently including tariffs. However, these have a close to negligible effect at less than 1% of our overall NPV.
Nisha: Stepping back from MPV, and as a reminder, our overall objective here at Bridgebio is simple. To maximize the positive change we can have in terms of quality-adjusted life years for the patients that we serve as quickly as possible. We do so by creating as many meaningful medicines as possible, as quickly as possible, within three constraints. The first is that each program must have beautiful science, which in our vernacular needs a high POTS, driven by understanding a mechanism of disease optimally paired with a therapeutic mechanism of action that targets a well-described genetic condition at its source.
Neil: Stepping back from NPV and as a reminder, our overall objective here a bridge viale a symbol to maximize the positive change we can have in terms of quality adjusted life years for the patients that we serve as quickly as possible, we do sell by creating as many meaningful medicines as possible as quickly as possible within three constraints the FERC.
Neil: Is that each program must have beautiful science, which in our vernacular means a high driven.
Neil: Driven by understanding the mechanism of disease optimally paired with a therapeutic mechanism of action that targets a well described genetic condition added source. These types of programs historically have a three to four times higher probability of Facebook to SaaS, then all comer drug R&D efforts over time and many programs there's elevated probably.
Nisha: These types of programs historically have a three to four times higher probability of technical success than all-comer drug R&D efforts. Over time and many programs, this elevated probability of technical success moves us from being a speculative lottery ticket to being something akin to an engineering company. The second constraint is that we strive for each medicine that we make to be first-in-class or best-in-class. Determining best-in-class can be tricky in the absence of double-blind head-to-heads, but we approach this using a straightforward Bayesian approach. For instance, in TTR, if faced with a decision as to what drug to use, a logical person would first interrogate the salient endpoint, death and hospitalization, at a common time point.
Neil: Ability of technical success moves us from being a speculative lottery ticket to being something akin to an engineering company.
Neil: The second constraint is that we strive for each medicine that we make to be first in class or best in class.
Neil: Determining best in class can be tricky in the absence of double blind head to heads, but we approach. This using a straightforward Bayesian approach for instance in TCR, if faced with decision as to what drug to use a logical person would first interrogate the surrogate endpoint death and hospitalization at a common time point.
Nisha: In this case, at 30 months, a relative risk reduction of 42% is a better point estimate as compared with any of the other products in this marketplace. You would then ask, how quickly does each drug take action? And you would find that the three-month beginning of separation between placebo and active is the earliest point estimate of timing of impact in this field. So logically, Atrevi is, at worst, no worse than other medicines. You would then look at safety. Here you would find little difference between small molecules, but would observe that they do not have the safety signals some knockdowns have.
Neil: In this case at 30 months of relative risk reduction of 42% is a better point estimate as compared with any of the other products. In this marketplace. You would then ask how quickly does each drug take action and you would find that the three months beginning of separation between placebo and active is the earliest point estimate of timing of <unk>.
Neil: Packed in this field so logically what truly is at worst no worse than other medicines.
Neil: You would then look at safety.
You will find little difference between small molecules, but would observe that they do not have the safety signals some knockdowns half.
Nisha: Vitamin A deficit, which by the way, turns knockdowns into a once daily pill regimen, injection site reactions, the absence of impact on AFib, whereas both small molecules had meaningful reductions there. The imbalance of cardiac SAEs is observed in the IMPATRA study, and the as of yet unexplained imbalance in death on the review surround trial. You couple that with the overall finding that across multiple studies, TTR is a cross-species conserved protein, and that ever higher levels of TTR lead to ever longer lives and less disease over time. So, logically, small-molecule stabilization from these data is the safest approach.
Neil: M&A deficit, which by the way it turns knockdowns into a once daily pill regimen injection site reactions.
Neil: Absent of impact on Afib, whereas both small molecules had meaningful reductions there the imbalance of cardiac assays as observed in the <unk> study and the as of yet unexplained imbalance in deaths on the reviews ran trial.
Neil: You couple that with the overall finding that across multiple studies PCR is a cross species conserve protein and that ever higher levels of GTR.
Neil: Lead to ever longer lives and less disease over time.
Neil: So logically small molecule stabilization from these data is the safest approach finally, the logician might look at price only to discover that truly has the lowest price point.
Nisha: Finally, the logician might look at price only to discover that Atruvi has the lowest price point. We believe, given all of that, the choice is clear. We apply similar reasoning in other competitive spaces like a contemplation.
Neil: We believe given all of that the choice is clear.
Neil: We apply similar reasoning in other competitive spaces like achondroplasia.
Nisha: Our final constraint is that each program we work on should be NPV-positive to ensure firm-level sustainability. We use all available levers during the R&D stage to optimize NPV, including time, cost, cost of capital, and probability of technical success. The better our model is at generating economic value from projects, the further we can push into markets where others cannot extract adequate value, in addition to being a better owner of obviously NPV-positive projects. The fact that we will spend well under $100 million per program to get each one of our potential blockbusters from the preclinical stage through proof of concept sets us up well for future growth that is economically attractive.
Neil: Our final constraint is that each program, we work on should be NPV positive to ensure firm level sustainability, we use all available levers during the R&D stage to optimize NPV, including time cost cost of capital and probability of technical success. The better our model is it generating economic value from projects. The further we.
Neil: Can push into markets, where others cannot extract adequate value. In addition to being a better owner of obviously NPV positive projects.
Neil: The fact that we will spend well under $100 million per program to get each one of our potential blockbusters from the preclinical stage through proof of concept.
Neil: US up well for future growth that is economically attractive.
Nisha: Okay, so that's our overall objective function with its attendant constraints. And you heard the first and most important frame that we use to think about the business, which is MPV.
Neil: Okay. So thats, our overall objective function with its attendant constraints.
Neil: And you heard the first and most important frame that we used to think about the business, which is the NPV.
Nisha: There are other frameworks that we also use to look at the business, understanding the business by staging capability, research, development, and commercial, understanding the business in the context of the overall ecosystem, and understanding the business program by program. Using the bi-stage framework first, and as I mentioned earlier, we are pleased with the ongoing progress of our commercial launch and the building of a sustainable competitive advantage there, which means, of course, that our group is able to extract more profit from the asset than another would be able to. I've also touched already on our ongoing development wins.
Neil: There are other frameworks that we also used to look at the business understanding the business by staging capability research development and commercial understanding the business in the context of the overall ecosystem.
Neil: And understanding the business program by program.
Neil: Using the by stage framework first and as I mentioned earlier, we are pleased with the ongoing progress of our commercial launch and the building of a sustainable competitive advantage, there, which means of course that our group is able to extract more profit from the asset then another would be able to.
Neil: I've also touched already on our ongoing development wins on discovery, our bread and butter, we continued to advance programs in genetic dilated cardiomyopathy and ADP JV. We also hold significant stakes in <unk> oncology therapeutics and gondola bio.
Nisha: On discovery, our bread and butter, we continue to advance programs in genetic dilated cardiomyopathy and ADPKD. We also hold significant stakes in Bridgebio Oncology Therapeutics and Gondola Bio. In the latter, we expect phase two data in our EPP program later this year, and we expect to generate up to six development candidates in 2025. From an ecosystem framing, the substrate for making genetic medicines remains incredible. We see that in the progress Gondola is making, and more broadly, with the depth of genetic disease starting points being produced and accessible at low price points.
Neil: In the latter we expect phase II data in our <unk> program later this year and we expect to generate up to six development candidates in 2025.
Neil: From an ecosystem framing the substrate for making genetic medicines remains incredible we see that in the progress gondola is making and more broadly with the depth of genetic disease, starting points being produced and accessible at low price points.
Nisha: Finally, at a program level, Matt will have more to say about a commercial launch of a Truvy. Our goal there, as a reminder, is $4.3 billion in peak year sales, or about 30% of a $15 billion marketplace. That gets delivered against approximately $380 million of spend on the brand per year. Almost 25% of current spend is on further research and development activities, like ACT Early, which we think could continue to improve our medicine's positioning and its ability to help patients in later years. The product of this spend also includes salient results like our variant data, where we obtained a hazard ratio of 0.41 with a p-value of less than 0.02 in the sickest of patients.
Speaker Change: Finally at a program level, Matt will have more to say about our commercial launch of a truly our goal. There is a reminder is $4 $3 billion in peak year sales or about 30% of our $15 billion marketplace.
Speaker Change: It gets delivered against approximately $380 million of spend on the brand per year almost 25% of current spend is on further research and development activities Like Act early which we think can continue to improve our medicines positioning and its ability to help patients in later years.
Speaker Change: Probably the spend also includes salient results like our variant data, where we obtained a hazard ratio of zero point 401, with a P value of less than 0.0 to in the sickest of patients and new work. We are doing regarding afib, which is an emerging marker of disease progression. The first result of which we plan to.
Nisha: And new work we are doing regarding AFib, which is an emerging marker of disease progression, the first result of which we plan to present at ESC later this year.
Speaker Change: Present at ESC later this year.
Nisha: For Infogradinib, a first-in-class oral FGFR3 inhibitor in development for both achondroplasia and hypochondroplasia, the Pivotal Propel 3 Phase 3 is fully enrolled, and we expect last participant, last visit by the end of this year.
Speaker Change: For <unk>, a first in class oral <unk> inhibitor in development for both achondroplasia and hypo contemplation. The pivotal propel three phase III is fully enrolled and we expect last participant last visit by the end of this year.
Nisha: We have also reached regulatory alignment with the FDA on our clinical development plan for infogratinib in children with achondroplasia from ages 0 to 3, and we expect to initiate clinical development in this important age range by the end of the year. Excitingly, we have also enrolled the run-in for our Phase 2 hypochondroplasia trial well ahead of benchmark.
Speaker Change: We have also reached regulatory alignment with the FDA on our clinical development plan for <unk> in children with Achondroplasia from ages zero to three and we expect to initiate clinical development in this important age range by the end of the year Excitingly. We've also enrolled the run in for our phase two hypo contemplation trial.
Speaker Change: Well ahead of benchmarks.
Nisha: For Encalerate, a negative allosteric modulator of the calcium-sensing receptor, our Phase III Calibrate study is fully enrolled. We expect last patient, last visit, and top-line results in the second half of this year.
Speaker Change: For and calibrate a negative allosteric modulator of the calcium sensing receptor are phase III calibrate study is fully enrolled we expect last patient last visit and topline results in the second half of this year in parallel and calories is also being studied in hyperparathyroidism and we announced today positive POC data front.
Nisha: In parallel, Encalerate is also being studied in hyperparathyroidism, and we announced today positive POC data for Encalerate in this key expansion indication. Preliminary evidence in the ongoing POC study has demonstrated that 78% of the first nine study participants were able to achieve normal blood and urine calcium levels within five days of Encalerate administration.
Speaker Change: <unk> in this key expansion indication.
Speaker Change: So any evidence in the ongoing POC study has demonstrated that 78% of the first nine study participants were able to achieve normal blood and urine calcium levels within five days of in calories administration.
Nisha: Moving to BBP418, an oral first-in-class disease-modifying therapy in Phase 3 development for treatment of individuals living with limb-girdle muscular dystrophy type 2i. We have fully enrolled Fortify, a Phase 3 registrational placebo-controlled study, evaluating the safety and efficacy of BBP418. The study includes a planned interim analysis to 12 months, focused on assessing a surrogate endpoint biomarker, glycosylated alpha-distriglycan, to support accelerated approval in the United States. We anticipate top-line readout from that Phase 3 interim analysis second half of this year as well.
Speaker Change: Moving to <unk> $4 eight an oral first in class disease modifying therapy in phase III development for treatment of individuals living with limb girdle muscular dystrophy type <unk>, we have fully enrolled fortify our phase III registrational placebo controlled study evaluating the safety and efficacy of <unk>.
Speaker Change: <unk> hundred one eight the study includes a planned interim analysis at 12 months focused on assessing a surrogate endpoint biomarker glad cost related Alpha district ligand to support accelerated approval in the United States, We anticipate topline readout from that phase III interim analysis second half of this year as well.
Nisha: Finally, BBP812 is an AAV9 gene therapy and development for cannabis disease, an ultra rare neurodegenerative disease that usually leads to death in the first two decades of life. Here, we are pursuing an accelerated approval approach using a single, seamless, registrational trial to bring this potential therapy to children with cannabis as quickly as possible. A meeting this month with the FDA reinforced the validity of our approach, which centers around the use of urine NAA in tandem with other clinical measures and the suggested BLA filing that we have laid out by end of 2026. While this is not a very prevalent disease, we believe it's a great example of our model, targeting disease at its source and operating legally to enable us to go after a well-validated condition in an NPV-positive manner.
Speaker Change: Finally, <unk> <unk> is an AAV non gene therapy in development for Canavan disease in ultra rare neurodegenerative disease that usually leads to death in the first two decades of life here.
Speaker Change: Here, we are pursuing an accelerated approval approach using a single seamless registrational trial to bring this potential therapy to children with Canada and as quickly as possible.
Speaker Change: Our meeting this month with the FDA reinforced the validity of our approach which centers around the use of urine NAA in tandem with other clinical measures and as suggested BLA filing that we have laid out by end of 2026.
Speaker Change: While this is not a very prevalent disease. We believe it is a great example of our model targeting disease at its source in operating legally to enable us to go after a well validated condition and NPV positive manner.
Nisha: Okay, that's the final framing.
Speaker Change: Okay. That's the final framing to wrap up my comments <unk> is executing well and importantly possessed of a tremendously strong foundation for the future number one an unmatched collection of late stage generic disease businesses with favorable economic prospects number two a cadre of outstanding managers that are dedicated to this.
Nisha: To wrap up my comments, Bridgebio is executing well and importantly possessed of a tremendously strong foundation for the future. Number one, an unmatched collection of late-stage genetic disease businesses with favorable economic prospects. Number two, a cadre of outstanding managers that are dedicated to their specific assets business and to Bridgebio. Number three, a diversity of stakes, including wholly owned assets and significant stakes in gondola bio and Bridgebio oncology therapeutics. Number four, first choice ranking with many academics when seeking a partner to discover and develop new medicines. And number five, a culture that is distinctive for most biotechs I have seen, and that is decentralized, independent thinking and lives the value that every minute counts for the patients we serve.
Speaker Change: Civic asset business and to bridge bile number three a diversity of stakes, including wholly owned assets and significant stakes in gondola bio Enbridge bio oncology therapeutics number four first choice ranking with many academics, one seeking a partner to discover and develop new medicines and number five a culture that is.
Speaker Change: Linked in for most biotechs I've seen and that is decentralized independent thinking and live the value that every minute counts for the patients we serve.
Nisha: We're excited to work with you, our investors, to continue to build this company and to keep you up to date on our progress for patients.
Speaker Change: We're excited to work with you our investors to continue to build this company and to keep you up to date on our progress for patients with that I'll hand, it over to Matt to walk through attributes commercial performance in more detail.
Matt Oughton: With that, I'll hand it over to Matt to walk through a TruBee's commercial performance in more detail. Thanks, Neil. To add to your comments, the launch of a Truby is off to a strong and encouraging start. Let me walk through some of the highlights in terms of what we are seeing out in the marketplace and what we think is driving the rapid momentum we've built in just a few short months. The first highlight is the early uptake across all major prescriber and patient segments. As shared in our press release, 2,072 unique patients have received a prescription for a TRUBY through April 25th, and 756 unique health care providers have written at least one prescription.
Matt Outten: Thanks, Neil to add to your comment we will launch of a truly is off to a strong and encouraging start let me walk through some of the highlights in terms of what we're seeing out in the marketplace and what we think is driving a rapid momentum. We've built in just a few short months the <unk>.
Matt Outten: First highlight is the early uptake across all major prescriber and patient segments.
Matt Outten: Third in our press release 2072 unique patients have received a prescription for <unk> through April 2000, and 756 unique healthcare providers have written at least one prescription. This early adoption spans all physician segments, including large academic centers regional amyloid clinics.
Matt Oughton: This early adoption spans all physician segments, including large academic centers, regional amyloid clinics, high volume heart failure specialists, and community cardiologists. Importantly, we're seeing it should be used across the full spectrum of patients, wild-type invariant, newly diagnosed, as well as switches from partial stabilized. The fact that these different patient types are all gaining timely access and that prescribers are already writing for multiple patients, gives us confidence that we're building a strong foundation for a true.
High volume heart failure specialists and community cardiologists.
Matt Outten: Importantly, we're seeing attributing used across the full spectrum of patients wild type invariant.
Matt Outten: <unk> as well as switches from partial stabilizers. The fact that these different patient types are all gaining the timing access and that prescribers are already writing for multiple patients gives us confidence that we're building a strong foundation for a truly.
Matt Oughton: So let's discuss what is driving the uptake. It comes down to strong clinical endpoints paired with our transparent patient-first support program. Starting with the clinical data, Atruvi is the only therapy for ATTRCM that has demonstrated a separation from placebo in as early as three months. No other medicine has demonstrated that, and it's not just about efficacy. Atruvi also positively impacts KCCQ scores, which directly correlate with better quality of life for patients. That's resonating deeply with physicians who want to make a meaningful impact on their patients' daily lives and patients who want to keep their functional activity levels.
Matt Outten: So let's discuss what is driving the uptake it comes down to strong clinical endpoints paired with our transparent patient support programs.
Matt Outten: Starting with the clinical data are truly the only therapy for <unk> that has demonstrated a separation from placebo and as early as three months no. Other medicine has demonstrated that and it's not just about efficacy attribute also positively impacts Casey CQ scores, which directly correlate with better quality of life.
Matt Outten: For patients that is resonating deeply with physicians, who want to make a meaningful impact on their patients' daily lives and patients who want to keep their functional activity levels.
Matt Oughton: Second, let's look at hospitalization rates. Atruvi is the only ATTR-CM therapy to demonstrate 50% relative risk reduction in cardiovascular hospitalization rates. This includes a statistically significant reduction in compositive mortality and hospitalization in the variant ATTR-CM population, a subgroup widely regarded as among the most difficult to treat. This was highlighted in the recent variant subgroup data presented at ACC.
Matt Outten: Second lets look at hospitalization rates are truly the only AT&T RCM therapy to demonstrate 50% relative risk reduction in cardiovascular hospitalization rates. This includes a statistically significant reduction in composite of mortality and hospitalization in the varying ATT Dr.
Matt Outten: <unk> population a subgroup widely regarded as among the most difficult to treat this was highlighted in the recent various subgroup data presented at ACC.
Matt Oughton: And third, affordability. Atruvi is the most cost-effective therapy in ATTRCM available. 10% less expensive than tefamidus, and 50% less expensive than nutriceran. It's not just about the WAC price, though. Atruvi has a free trial program for all patients, regardless of insurance status, and lifetime free drugs for patients who participated in our pivotal trials. That matters to physicians, patients, and payers, especially when coupled with the three-month onset of treatment effect and 50% reduction in cardiovascular hospitalization at 30 months. No other ATTRCM therapy checks all of these boxes.
Matt Outten: And third affordability, a truly is the most cost effective therapy, and ATT RCM available, 10% less expensive <unk> and 50% less expensive than interests are and it's not just about the WAC price, though a truly has a free trial program for all patients regardless of insurance status.
Matt Outten: And lifetime free drug for patients who participated in our pivotal trials that matters to physicians patients and payers, especially when coupled with the three months onset of treatment effect and 50% reduction in cardiovascular hospitalization at 30 months.
Matt Outten: No other <unk> therapy checks all of these boxes.
Matt Oughton: Bridgebio has a commercial model that is different, and it's working. A tribute will be followed by three additional commercial launches in 2026 and 27. What really differentiates Bridgebio, though, is how we bring products to market. There are no convoluted value-based contracts, no vague promises of future rebates contingent on volume thresholds. What we offer is simple, transparent, and built around the people who matter most, patients and their care teams. And we're hearing from the field that this clarity, paired with strong clinical data, is helping accelerate adoption. And once an HCP decides to prescribe Atruvi, we make it easy to get the medication to patients.
Matt Outten: <unk> bio has a commercial model that is different and it's working attribute will be followed by three additional commercial launches in 2026 and 27.
Matt Outten: What really differentiates bridge bio, though is how we bring products to market. There are no convoluted value based contracts no vague promises of future rebates contingent on volume thresholds. While we also a simple transparent and built around the people who matter most patients and their care teams and we're hearing from the field that this.
Matt Outten: Clarity paired with strong clinical data is helping accelerate adoption.
Matt Outten: And once in HCP decides to prescribe attribute we make it easy to get the medication to patients patient can receive attribute within 48 hours, we have two world class specialty pharmacies and the attribute network and also allow physicians to fill prescriptions in their own in house pharmacies.
Matt Oughton: Patients can receive Atruvi within 48 hours. We have two world-class specialty pharmacies in the Atruvi network, and also allow physicians to fill prescriptions in their own in-house pharmacies.
Matt Oughton: All of this investment is paid off. We are gaining share in the crucial first line setting and our conversion to paid rate and time to paid prescription is well ahead of industry benchmark.
Matt Outten: All of this investment has paid off we are gaining share in the crucial first line setting and our conversion to paid rate and time to paid prescription is well ahead of industry benchmarks.
Matt Oughton: Looking towards the future, our focus heading into the rest of the year is as follows. continue marching towards our long term goal of 30 to 40% share of the ATTR CM market. and increased share in the critical first line setting, which is a leading indicator of launch success. While these early signs are encouraging, and they reflect not just the strength of the product, but the preparation and the commitment of the broader Bridgebio team, we are also excited about what's ahead and remain focused on reaching as many patients as possible, as quickly as possible.
Matt Outten: Looking towards the future our focus heading into the rest of the year is as follows continue marching towards our long term goal of 30% to 40% share of the ATR cm market.
Matt Outten: And increased share in the critical first line setting, which is a leading indicator of launch success.
Matt Outten: While these early signs are encouraging and they reflect not just the strength of the product, but the preparation and the commitment of the broader bridge bio team. We are also excited about what's ahead and remain focused on reaching as many patients as possible as quickly as possible.
Tom Tremarkey: With that, I'll turn it over to Tom for a review of the financial. Thank you, Matt, and good afternoon, everyone. Q1 2025 marked Bridgebio's first full quarter of net product revenue from the Attribute US commercial launch, a major milestone for the company and a significant step forward in our evolution into a fully integrated genetic medicine business. I'll now walk through the financial highlights for the first quarter of 2025. Please note that our commentary on today's call will focus on gap financial measures and what's otherwise indicated. Total revenues were $116.6 million for Q1 2025 and consists of a truly net product revenue and licensed services revenue.
Tom: With that I'll turn it over to Tom for a review of the financials.
Tom: Thank you, Matt and good afternoon, everyone Q1, 2025, <unk> first full quarter of net product revenue from the attributes U S. Commercial launch a major milestone for the company and a significant step forward in our evolution into a fully integrated genetic medicine business.
Tom: Now I'll walk through the financial highlights for the first quarter of 2025. Please.
Tom: Please note that our commentary on todays call will focus on GAAP financial measures unless otherwise indicated.
Tom: Total revenues were $116 6 million for Q1, 2025 and consist of attributing net product revenue and license and services revenue.
Tom Tremarkey: Truly net product revenue was $36.7 million driven by strong demand across all major prescribers and patient segments. License and Services Revenue was $79.9 million in the quarter, primarily driven by the recognition of the $75 million Rego Tour milestone related to Biantra's EU approval. Also in Q1, we received Biantra approval in Japan, for which we expect to recognize a $30 million milestone in the second quarter. Total operating expenses for the first quarter of 2025 were $218.4 million compared to $210.2 million in the same period last year. This increase reflects our continued investment in the Atruvi brand and our advancing weight-based pipeline.
Tom: Contributing net product revenue was $36 $7 million driven by strong demand across all major prescribers for patient segments.
Tom: License and services revenue was $79 $9 million in the quarter, primarily driven by the recognition of the $75 million regulatory milestones related to be honest EU approval also in Q1, we received <unk> approval in Japan for which we expect to recognize a $30 million milestone in the second quarter.
Tom: Total operating expenses for the first quarter of 2025 were $218 4 million compared to $210 $2 million in the same period last year. This increase reflects our continued investment in the truly brand and our advancing late stage pipeline, including in our total operating expenses was $29 4 million of stock based compensation expense compared to.
Tom Tremarkey: Included in our total operating expenses was $29.4 million in stock-based compensation expense, compared to $28.9 million in the first quarter of 2020. Looking forward, we expect only modest growth in quarterly operating expenses for the remainder of the year with an offset total cash burn provided by a Truby Sales in the US and Biantra XUS. R&D expense for the first quarter of 2025 was $111.4 million, compared to $141 million in the same period last year. This decrease was largely due to the strategic carve out of our ecology business and early stage research programs, allowing us to focus resources on the attribute launch and late stage pipeline.
Tom: $28 9 million in the first quarter of 2024.
Tom: Looking forward, we expect only modest growth in quarterly operating expenses for the remainder of the year with an offset of total cash burn provided by HIV sales in the U S can be entre ex U S.
Tom: <unk> expense for the first quarter of 2025 was $111 4 million compared to $141 million in the same period last year. This decrease was largely due to the strategic carve out of our oncology business and early stage research programs, allowing us to focus resources on the attribute launched in late stage pipeline SG.
Tom Tremarkey: SG&A expense for the first quarter of 2025 was $106.4 million, compared to $65.8 million in the same period last year. This increase was driven by the full-scale commercial rollout of a Truby, including field team deployment, payer engagement, and patient support infrastructure. Restructuring expense for the first quarter of 2025 was $0.6 million compared to $3.4 million in the same period last year. We ended the quarter with $540.6 million in cash and cash equivalents, which does not include $105 million in regulatory milestone payments anticipated in Q2 for ex-US approvals of Biantra. We believe we are well-financed to support the continued execution of the Atribute launch and deliver on key milestones from the pipeline this year.
Tom: SG&A expense for the first quarter of 2025 was $106 4 million compared to $65 8 million in the same period last year.
Tom: This increase was driven by the full scale commercial rollout of a truly including field team deployment payer engagement and patient support infrastructure.
Tom: Restructuring expense for the first quarter of 2025 was <unk> $6 million compared to $344 million in the same period last year.
Tom: We ended the quarter with $546 million in cash and cash equivalents, which does not include $105 million in regulatory milestone payments anticipated in Q2 for ex U S approvals of beringer.
We believe we are well financed to support the continued execution of the attribute launch and deliver on key milestones from the pipeline. This year, we look forward to sharing additional commercial updates throughout the year with the top line data from our three phase III programs over the next year with 88, one <unk> in the second half of 2025 and achondroplasia in early 2026 with that.
Tom Tremarkey: We look forward to sharing additional commercial updates throughout the year and the top-line data from our three phase three programs over the next year with ADH1 and LGMD2i in the second half of 2025 and Acondysplasia in early 2026.
Tom Tremarkey: With that, I'll turn the call back to Jim. Thank you, Neil, Matt, and Tom.
Chile: I'll turn the call back to Chile.
Chile: Thank you, Neil Matt and Tom I'll now hand, it back to the moderator to open the line for questions I would like to request that analysts to limit themselves to one question. Each so that more people got a chance to ask their questions.
Operator: I'll now hand it back to the moderator to open the line for questions. I would like to request our analysts to limit themselves to one question each so that more people get a chance to ask their questions. At this time, we'd like to remind everyone in order to ask a question, please press the star button followed by the number one on your telephone keypad. We'll pause just for a moment to compile the Q&A roster.
Speaker Change: At this time I would like to remind everyone in order to ask a question. Please press the start button.
Speaker Change: Followed by the number one on your telephone keypad will cost us for a moment to compile the Q&A roster.
Salim Syed: Your first question comes from the line of Salim Syed of Nuzuho. Please go ahead. Great. Congrats on the quarter, guys. And congrats also on your first earnings call. Maybe just one for Neil or Matt here. Obviously, the quarter came in healthier than I think people had thought going into the print. And I appreciate the commentary that you provided, Matt, on some of the dynamics there. But any sort of granularity you could provide on the tailwind and what you really deem that's working well for you here? Yeah, maybe I'll kick it off. Thanks, Salim.
Speaker Change: Your first question comes from the line of Salim Sayed of Mizuho. Please go ahead.
Speaker Change: Great Congrats on the quarter guys and congrats also on your first earnings call.
Speaker Change: Maybe just one for Neil or Matt here, obviously the quarter came in.
Speaker Change: And then I think people had thought.
Speaker Change: Going into the print and I appreciate it.
Speaker Change: The commentary that you provided.
Speaker Change: On some of the dynamics, there, but any sort of granularity you can provide on the tailwind and what you're really deem that's working well for you here. Thank you.
Speaker Change: Yes, maybe I'll kick it off thanks Aleem.
Neil Kumar: You know, it's probably too early for some of the higher cost commercial tactics that we've invested in to show ROI right now. So hearteningly, I think a lot of the demand we're seeing to date really is a product number one of the differentiated clinical efficacy we have as a reminder, and you know, this the earliest separation and point estimate that we've seen in the field at three months, 42% relative risk reduction against ACM and CVH at 30 months, again, the best point estimate we've seen at 30 months, coupled with that 50% reduction in hospitalization, which turns out to be a hugely meaningful measure for patients who both want to live longer, but also live healthier.
Speaker Change: It's probably too early for some of the higher cost commercial tactics that we've invested in to show ROI right now so heartening Lee I think a lot of the demand. We're seeing to date really is a product number one of the differentiated clinical efficacy. We have as a reminder, and you know this the earliest separation as a point estimate that we've seen in the field.
Speaker Change: Three months, 42% relative risk reduction against ACM and CVA should 30 months again, the best point estimate we've seen at 30 months, coupled with that 50% reduction in hospitalization, which turns out to be a hugely meaningful.
Speaker Change: Measure for patients, who both want to live longer but also lead healthier. So I'd say that's point number one point number two as you saw as well from the Pfizer call. This morning.
Neil Kumar: So I'd say that's point number one. Point number two, as you saw, as well from the Pfizer call this morning, continued market growth, right? We're one fifth diagnosed in this space, some 50,000 or so patients diagnosed, we think there's 250 to 300,000 in the US alone. And so physician education, coupled with there being a variety of therapeutic interventions now available, I think is driving that growth. So that I think is another tailwind. And then the third is the access programs that Matt talked about, team, I think has done a terrific job of ensuring that patients when they are prescribed, we can get the medicine and stay as long as they need to.
Speaker Change: <unk> market growth rate or one fifth diagnosed in this space some 50000 or so patients diagnosed we think there is 250 to 200000 in the U S alone and so physician education.
Speaker Change: With there being a variety of therapeutic intervention now available I think is driving that growth. So that I think is another.
Speaker Change: <unk> and then the third is the access programs that Matt talked about the team I think has done a terrific job of ensuring that patients when they are prescribed which we can get the medicine and stay on the medicine.
Speaker Change: As long as they need to so those would be the three salient drivers the final thing.
Neil Kumar: So those would be the three salient drivers.
Neil Kumar: You know, I'd say the final thing is a little bit of what a physician described to us the other day as karma. Obviously we're the only sponsor in this space that gave free drug for life for their trial participants. Unfortunately, others decided not to. We're also the only sponsor in the space running a primary prevention study, which I think many physicians view as the ultimate in trying to catch patients in this mass action condition as early as possible to do as much as we can for their condition. So, you know, those types of things, I think over a long period of time will stand us in good stead as a sponsor here in a competitive space.
Speaker Change: As a little bit of what a physician describe to us the other day as Karma, obviously were the only sponsor in this space that gave free drug for life for their trial participants. Unfortunately, others decided not to we're also the only sponsor in the space running a primary prevention study, which I think many physicians view as the ultimate and trying to catch patients in this math.
Speaker Change: Action condition as early as possible to do as much as we can for their condition. So those types of things I think over a long period of time will stand us in good stead as a sponsor here in a competitive space I don't know, Matt if you'd add anything.
Neil Kumar: Well, I think in this the people we've spent a lot of time putting the team together both internal and field to make it as easy as we can for both prescribers and for patients and I think you're seeing the good results. Great. Thanks, guys. Congrats again.
Speaker Change: Thank goodness the people we've spent a lot of time, putting the team together both internally.
Speaker Change: Yes.
Speaker Change: To make it as easy as we can for both prescribers and for patients and I think you're seeing the good results of that.
Speaker Change: Great. Thanks, guys Congrats again.
Speaker Change: Yeah.
Speaker Change: Thanks Lynn.
Tyler Van Buren: Your next question comes from the line of Tyler Van Buren of TD Kallen. Please go ahead. Hey guys, my congrats on the Stellar Atruvi result as well and the ongoing progress of the pipeline. So again, the 37 million of Atruvi sales far exceeded expectations. So when you say that the paid conversion rate is well ahead, can you help quantify that? Was the time to pay quicker than a month for some patients? And how much stocking was there in the Yeah.
Speaker Change: Your next question comes from the line of Tyler Van Buren of TD Cowen. Please go ahead.
Speaker Change: Hey, guys. Congrats on a stellar jewelry result, as well and the ongoing progress with the pipeline. So again, the $37 million of attribute sales far exceeded expectations. So would you say that.
Speaker Change: Paid conversion rate as well ahead can you help quantify that was the time to paid quicker than a month for some patients.
Speaker Change: And how much stocking was there in the quarter.
Matt Oughton: Hey, Tyler. Great to hear from you. And thanks for the question.
Speaker Change: Yeah, Hey, Tyler great to hear from you and thanks for the question I am going to pass it onto back to comment more on conversion. Yes. Thanks for the question, we're really happy with how conversion is tracking and Thats for both free trial to paid and also a commercial prescriptions to paid.
Matt Oughton: I'm going to pass it on to Matt to comment more on conversion. Yeah. Thanks for the question. You know, we're really happy with how conversion is tracking and that's for both free trial to pay and also commercial prescriptions to pay. Everything regarding conversion is consistent or better than historical launches that we've studied.
Speaker Change: Everything regarding conversion is consistent or better than historical launches that we've studied I will just point out our free trial acts like a normal prescription.
Matt Oughton: I will just point out a free trial acts like a normal prescription. A HCP writes it, the pharmacy processes it, and the patient gets it. And we designed our network specifically with conversion in mind. And that's what makes the access so easy.
Speaker Change: <unk> rights at the pharmacy processes, it and the patient gets it and we designed our network specifically with conversion in mind and that's what makes the access so easy.
Matt Oughton: Our goal remains the same 30 to 40 percent peak share. It takes about three to six years usually to hit. We're hoping we can do that a little bit faster.
Speaker Change: Our goal remains the same 30% to 40% peak share.
Speaker Change: It takes about three to six years, usually the hit we're hoping we can do that a little bit faster.
Tom Tremarkey: In regards to your channel and inventory question, I mentioned we do have a limited distribution network. That allows for very frequent ordering and it allows our customers, our distributors, to have just-in-time inventory. So there's no need for them to really hold large quantities. And so typically I would say that this small group of distributors holds about one to two weeks of inventory.
Speaker Change: In regards to your channel and inventory question I mentioned, we do have a limited distribution network that allows for very frequent ordering and it allows our customers our distributors to have just in time inventory. So there is no need for them to really hold large quantities.
Speaker Change: So typically I would say that this small group of distributors holds about one to two weeks of inventory.
Tom Tremarkey: Hey, Tyler. This is Tom. Let me just put a finer point on the inventory question. As I mentioned in the prepared remarks, the sales in the core are primarily driven by demand, so we've seen only a minor impact. on Total Sales in the Quarter. So really demanding.
Speaker Change: This is Tom let me just put a finer point on the inventory question as I mentioned in the prepared remarks, the sales in the quarter, primarily driven by demand. So we've seen only a minor impact of inventory on total sales in the quarter, So really demand driven here.
Tyler Van Buren: Thanks, Tyler. I know you had another question. happy to answer more about this. Well, that's great. Thank you.
Speaker Change: Yes, Thanks, Tyler I know you had another question so.
Happy to answer more about this our audio next question.
Speaker Change: That's great. Thank you.
Manny Forouhar: Your next question comes from the line of Manny Forouhar of Fleer & Partners. Please go ahead. Hey guys, congrats on the quarter. Clearly blew out expectations, despite having shown pretty good scripts repeatedly. I want to dive in on the script numbers. I'm looking at the incremental scripts on a weekly basis, through that first update on January 10th, was in the 60s per week. And then through the update gave on an incremental basis on February 17th, that was running north of 100 scripts a week. And again, running through from 12 and four weeks into launch and 22 weeks into launch, i.e.
Speaker Change: Your next question comes from the line of Manny for <unk> Leerink partners. Please go ahead.
Manny: Hey, guys congrats on the quarter.
Clearly you blew out expectation.
Speaker Change: Despite having some pretty good scripts repeatedly I wanted to dive in on the script numbers I'm looking at the incremental scripts on a weekly basis.
Speaker Change: That first update on January 10th, but then the <unk> per week and then through the update you gave on an incremental basis on February 17th that was running north of a 100 scripts a week and again running through from 244 weeks interruption 'twenty two weeks into launch from February 17th.
Manny Forouhar: From February 17th to April 25th, you're still running at about 110, 109 scripts per week, as reported. I know it's just math, and there's a lot of nuance that goes into script recording.
Speaker Change: April 25th Youre still running at about 110 109 scripts per week.
Speaker Change: As reported.
Speaker Change: I know, it's just math.
Speaker Change: And there's a lot of nuance that goes into the script recordings.
Manny Forouhar: Could you give us a sense of what the sort of character and velocity of new of new patient scripts you're seeing right now? And so what is the what is the kind of direction of travel that's there?
Speaker Change: Give us a sense of what the tariffs are in velocity of news from new patient scripts that you're seeing right now and sort of what is the what is the kind of direction of travel there, but I have a follow up question.
Manny Forouhar: And I have a follow up.
Manny Forouhar: Great, I can take that.
Speaker Change: Great I can take that thank you for the question I mean, I think starting out the thesis really again it remains unchanged CPR levels go up with the <unk> and it's the only near complete stabilizer on the market when we sort of take that thesis and then dive into okay, well, what's happening and then how do we think thats going to impact things going forward.
Manny Forouhar: Thank you for the question. I mean, I think starting out the thesis really, again, it remains TTL levels go up with the Trudy and it's the only near complete stabilizer on the When we sort of take that thesis and then dive into, okay, well, what's happening and then how do we think that's going to impact things going forward? The month over month growth rate in the Treatment 90 section has been very strong, and that's been across all segments. So this is in the large centers. This is in the community. It's across all types of patients, variant, wild type.
The month over month growth rate in the treatment naive section has been very strong and that's been across all segments. So this is in the large centers. This is in the community class across all types of patients Varian wildfire.
Manny Forouhar: There hasn't been that sort of a, you know, one area or another that's done better. We've sort of seen this across the board. And so then when we look at sort of the two groups, we have the Treatment 90. That's our focus, and that will continue, we believe, to grow over time as it has Then you have the switch patients, of course, when we launched, we had 100% of the switch share because we were the only option you could switch to. As you now with new entrants in the market, that's going to evolve over time, because there are now more choices.
Speaker Change: It hasnt been in sort of a.
Speaker Change: One area or another that has done better than we sort of seems across across the board and so then when we look at sort of the two groups. We have the treatment naive patients. That's our focus and that will continue we believe to grow over time as it has been then you have the switch patients of course, when we launched we had 100 per.
Speaker Change: A scent of the switch share because we were the only option you could switch to.
Speaker Change: As you know with new entrants in the market that's going to evolve over time, because they are now more choices.
Manny Forouhar: And so I think that the data and how we report will evolve over time as well, based on that. But that's kind of where we are and how we see it.
Speaker Change: So I think that the data and how we report will evolve over time as well based on that.
Speaker Change: That's kind of where we are and how we see it moving forward.
Manny Forouhar: Great, that's helpful.
Speaker Change: Great that's helpful.
Manny Forouhar: And I have sort of a non PTR question. to everyone's surprise. When you think about limb girdle data coming later on this year, which obviously we've been discussing a number of calls. This is not the first time you guys have brought it up. We talked about it at my conference a couple years running about the file ability. of that data set, or whether a later data set in the same study will be required. Do any of the changes of FDA...
Speaker Change: And I have sort of a non GTR question.
Speaker Change: So everyone's surprise.
Speaker Change: Do you think about limb girdle data coming later on this year.
Speaker Change: Which obviously we've been discussing a number of calls disrupt first time you guys have brought it up we talked about at my conference a couple of years running.
Speaker Change: The file ability.
Speaker Change: Of that dataset or whether a later data set from the same study will be required.
Speaker Change: Do any of the changes of FCA.
Speaker Change: Concerned you are on your ability to file on a biomarker or do you think you have to show a distinct level a clear clinical improvement on a non biomarkers about strong store base.
Neil Kumar: Thank you for listening. Where are we in terms of the fileability on that biomarker for Yeah, I can, I can take that. Mani, you know, we haven't met with the agency of late on the LGMT2I program, but we have had some close to 10 meetings now over the course of the last month, as it pertains to either pipeline at Gondola or pipeline here at Bridge, probably the latest meeting was in and around the Canavan program. And I have seen nothing but I would say a positive inclination toward trying to get first in class medicines that target pathomechanism, like our drug for LGMT2I onto the marketplace as quickly as possible to benefit the children that are suffering from these conditions.
Speaker Change: Where are we in terms of the file ability on a biomarker for limb girdle.
Speaker Change: Yes, I can.
Speaker Change: Can take that.
Speaker Change: <unk>.
Speaker Change: We haven't met with the agency of late on the <unk> program, but we have had some close to 10 meetings now over the course of the last month.
Speaker Change: As it pertains to other pipeline, a gondola or pipeline here a bridge probably the latest meeting was in and around the <unk> program and I have seen nothing but.
Speaker Change: Yeah.
Speaker Change: I would say a positive inclination towards trying to get first in class medicines that target patent mechanism like our drug for LG <unk> onto the marketplace as quickly as possible to benefit the children that are suffering from these conditions and so I don't think the rather clear guidance that we've gotten from the <unk>.
Neil Kumar: And, and so I don't think the rather clear guidance that we've gotten from the agency to date in and around around glycosylation of ADG is going to change. I really don't. You know, I have been on the record saying, I think it's going to be a problem if we go the right way and hit our primary endpoint on ADG, but we go the wrong way on all clinical measures. Obviously, I don't think that's going to be the case based on what we observed in phase two, which was albeit an open label study against natural history where we saw improvement in measures of ambulation and other clinical outcomes.
Speaker Change: To date in and around file ability around.
Speaker Change: Constellation of Atg is going to change.
Speaker Change: I really don't.
Speaker Change: I have been on the record, saying I think it's going to be a problem. If we go the right way and hit our primary endpoint on atg, but we go the wrong way on all clinical measures, obviously, I don't think thats going to be the case based on what we observed in phase two which was albeit an open label study against natural history, what we saw improvement in.
Speaker Change: Measures of ambulation.
Neil Kumar: So, so I do believe these things will move toward the positive, or at least a few will move toward the positive. Obviously, modified NorStar, NorStar, which is historically been the gold standard in these conditions, it's all noise over the course of the first 12 months. So whether the point estimate goes one way or the other, I think natural history has made that very clear. And that is why we're running the confirmatory trial. This is effectively a nested trial design that So I mean, you know, long story short, yeah, we continue to believe that this is going to be an approvable endpoint and in terms of Professor Leigh.
Speaker Change: Clinical outcomes. So so I do believe these things will move.
Speaker Change: Towards the positive or at least a few will move towards the positive.
Speaker Change: Obviously modify Northstar Northstar, which has historically been the gold standard in these conditions. It's all noise over the course of the first 12 months. So whether the point estimate goes one way or the other I think natural history has made that very clear and that is why we're running the confirmatory trial. This is effectively a nest and trial design that goes on for another two and a half years.
Speaker Change: Post the.
Speaker Change: Post the readout later this year, so I mean.
Speaker Change: Long story short, yes, we continue to believe that this isn't going to be.
Speaker Change: Approvable endpoint and in terms of atg like oscillation increase.
Manny Forouhar: Awesome. Thanks, guys.
Speaker Change: Awesome. Thanks, guys.
Thanks, Mike.
Tyler Van Buren: Your next question comes from the line of Tyler Van Buren of TD Calum. Please go ahead. Hey, guys. Yeah, I just had a second question I meant to ask, but just the ADH-1 and Limberl Phase III's reading out there in the second half of the year clearly have a high probability of success.
Speaker Change: Your next question comes from the line of Tyler Van Buren of TD Cowen. Please go ahead.
Speaker Change: Okay.
Speaker Change: Hey, guys. Yeah, I just had a second question I meant to ask but just the 80 H, one and limb girdle phase III reading out during the second half of the year clearly have a high probability of success. So can you it would be great to hear you elaborate on what investors should be excited about and more importantly focus on those opportunities beyond the ongoing <unk> launch what you think the magnitude.
Neil Kumar: So can you, it'd be great to hear you elaborate on why investors should be excited about, and more importantly, focus on those opportunities beyond the ongoing Atrubi launch. What do you think the magnitude of those opportunities could be? Yeah, good question, Tyler. You know, as I was suggesting, we continue to be heartened by both the clinical operational positioning of LGMD2I, ADH1, and our achondroplasia trials, as well as the potential to help a broad swath of patients. And I think from an investor standpoint, one has to consider the sheer size of these marketplaces. LGMD2I, as we've discussed, some 7 to 8,000 patients between the US and EU, that's a substantial market size as compared to many of the other muscular dystrophy programs that you see out there at the price points that are attended in the space.
Those opportunities could be.
Speaker Change: Okay.
Speaker Change: Yes, good question Tyler.
Speaker Change: Suggesting we continue to be heartened by both the clinical operational.
Speaker Change: Positioning of <unk>.
Speaker Change: H, one and our achondroplasia trials.
Speaker Change: As well as the potential to help a broad swath of patients and I think from an investor standpoint, one has to consider this year size.
Speaker Change: These marketplaces <unk> as we've discussed some seven to 8000 patients just in the U S and EU, that's a substantial market sizes compared to many of the other muscular dystrophy programs that.
Speaker Change: That you see out there at the price points that are attendant in this space and with 88 one.
Neil Kumar: And with ADH1, a rather much more prevalent condition, maybe 10 to 12,000 patients in the United States alone, as suggested by statistical genetics methodology, some 4,000 or so already identified to date, that's going to be really a program associated with the market build and finding new patients. And we've already shown that we can do that in some of the sequencing efforts we've undertaken within the non-surgical hypopara community, where we're reliably finding something like 20 to 25% of patients actually harbor the gain-of-function mutations in the calcium-sensing receptor. impact on parameters that this community cares a great deal about, like proportionality and the like.
Rather much more prevalent condition, maybe 10 to 12000 patients in the United States alone.
Speaker Change: As suggested by state.
Speaker Change: Statistical genetics methodologies on 4000, or so already identified to date, that's going to be really a.
Program associated with the market build and finding new patients.
Speaker Change: And we've already shown that we can do that in some of the sequencing efforts we've undertaken within the non surgical hyper para community, where we are reliably finding something like 20% to 25% of patients actually harbor the gain of function mutations in the calcium sensing receptor. So I think two very exciting commercial opportunities two very exciting opportunities for first in <unk>.
Speaker Change: <unk> medicines for patients there and then I don't think I need to belabor, the achondroplasia hypothesis, where we feel.
We have an exciting oral best in class potentially best in class option that can provide differential safety differential efficacy because it targets as well described condition at its source.
Speaker Change: Driving differential both changes in growth and then also importantly impact on parameters that this community cares a great deal about like proportionality and alike.
Neil Kumar: And you know the size of that market from the Voxelgo launch that's ongoing.
Speaker Change: And you know the size of that market from from the box over launch that's ongoing so I think all three very exciting opportunities.
Neil Kumar: I think all three are very exciting.
Biren Amin: Thank you. Your next question comes from the line of Biren Amin of Equity Research Healthcare. Please go ahead. Yeah, hi. Thanks for taking my questions.
Speaker Change: Thank you.
Speaker Change: Your next question comes from the line of <unk> Amin of equity Research healthcare. Please go ahead.
Amin: Yeah, Hi, Thanks for taking my questions congrats on the quarter.
Biren Amin: Congrats on the quarter. And you know, really good launch out of the gate. I guess my question is, what's resonating with healthcare professionals as it relates to the Truby launch? And what are the biggest hurdles that you're seeing for adoption? And then maybe a second question, what was the gross net for the quarter and any impact that you've seen from the Part D redesign?
Amin: And really quick launch out of the gate I guess my question is what's resonating with health care professionals as it relates to the <unk> launch and what are the biggest hurdles that you are seeing for adoption and then maybe a second question what was the gross to net for the quarter and any impact that you've seen from the part D redesign.
Matt Oughton: Hey Viren, thanks for the question. I'm going to pass it on to Matt to discuss the HCPPs and then I'll throw it to Tom to discuss the... Yeah, thanks. And I guess there's sort of two parts. The feedback from the physician segments has been positive, as we've discussed. Our messaging around, we call it the 342.50, but that ability to separate as early as three months and getting results around the hospitalization and the all-cause mortality that we've described. Physicians want to see a drug work really fast, and they want to be able to tie it to hard outcomes.
Randy: Hey, Randy Thanks for the question.
Randy: I am going to pass it on to macro discuss THC Bbs and then offered to Tom to discuss the growth from that base.
Speaker Change: Yes, Thanks, and I guess, there's sort of two parts to this.
Speaker Change: The feedback from the physicians segments has been positive as we've discussed.
Speaker Change: Our messaging around we call it the $3 $42 50, but that ability to separate as early as three months and getting results around the hospitalization and the all cause mortality that.
Speaker Change: That we've described.
Speaker Change: Physicians want to see a drug work really fast and they want to be able to tie it to heart heart outcomes of course patients due to this work gives people confidence and so those messages have been resonating extremely well and I think thats resulted in positioning <unk>, but then also then.
Matt Oughton: Of course, patients do too. So this is what gives people confidence. And so those messages have been resonating extremely well. And I think that's resulted in physicians prying a tribute, but then also then repeating it, right? Those are the reasons that you sort of try a medication, and then when you see how well people are doing on it, then that encourages you to try it again. And so we've seen a lot of repeat prescribers across a lot of the different segments as well, and I don't think our expectations were otherwise or are going to change anytime soon.
Speaker Change: Repeating it right that those are the reasons that you sort of try a medication and then when you see how well people are doing on it than that encourage you to try it again, so we're seeing a lot of repeat prescribers across a lot of the different segments as well and I don't think our expectations, where otherwise are going to change anytime soon we have very.
Neil Kumar: We have very strong data package, and we also have very strong programs to help patients get on the medication once that prescription.
Speaker Change: Strong data package and we also have very strong programs to help patients get on the medication works that prescriptions have been written.
Tom Tremarkey: and then I'll pass it to Neil to add on. Yeah, maybe I'll just elaborate on that for a second before I throw it over to Tommy. You know, I think the ever-increasing availability of data, like most recently, Biren, I think we've talked about the variant data, you know, the fact that you could achieve a hazard ratio of 0.41 with statistical significance in that relatively small subpopulation and, as someone mentioned earlier, the sickest of subpopulations continues to reinforce that differential levels of stabilization can lead to ever-better outcomes. And we're starting to find that that's resonating. We've got a bevy of serum TTR-associated literature coming out suggesting that ever-higher levels of serum TTR correlate with ever-lower levels of downstream hospitalization and mortality.
Neil: And then ill pass it to Neil to add yes, maybe.
Neil: Maybe I will just elaborate on that for us that can provide third over time I think the ever increasing.
Neil: Availability of data like most recently.
Neil: <unk>.
Neil: We've talked about the various data.
Neil: That you could achieve a hazard ratio of 0.41 with statistical significance in that relatively small subpopulation and.
Someone mentioned earlier the sickest of subpopulations continues to reinforce that differential levels of stabilization can lead to ever better outcomes and we are starting to find that thats resonating. We've got a bevy of serum GTR associated literature coming out, suggesting that ever higher levels of serum GTR correlate whatever low.
Speaker Change: Your level of downstream hospitalization and mortality the connecting the dots between ever better stabilization and all of these downstream outcomes. I think is what's starting to resonate with the physician community on top of what Matt suggested the backbone, which is $3 40 to 50.
Neil Kumar: The connecting of the dots between ever-better stabilization and all of these downstream outcomes I think is what's starting to resonate with the physician community on top of what Matt suggested, the backbone, which is the 340.
Tom Tremarkey: Tony want to talk about it you, Sir. Group. So as we said before, the way to think about this is the floor is going to be the mandatory IRA rebate of 20%. And on top of that. You know, something like you'd see in other categories where contracting is not really happening. I would say, you know, given where we are in the launch early innings, we should expect some variability here quarter quarter this quarter, gross net trended slightly favorable versus what we were expecting. We also saw a slightly lower use of our first month free program versus what we were expecting.
Speaker Change: Tom you want to talk about again, yes sure on gross to net specifically so as we've said before the way to think about this as the floor is going to be the mandatory IRA a rebate.
Speaker Change: <unk> of 20% on top of that Youre going to add.
Speaker Change: Something something like you'd see in other categories, where contract is not really happening that's what we're seeing here I would say.
Speaker Change: Given where we are in the launch early innings, we should expect some variability quarter to quarter this quarter.
Net trended slightly favorable versus what we were expecting.
Speaker Change: We also saw slightly lower use of our first month free program versus what we were expecting and we saw slightly lower use of our patient assistance program and what we're expecting.
Tom Tremarkey: And we saw slightly lower use for Great, thank you. Just to add that, you know, the results are reflective of strong, strong underlying demand for Truby and the execution of our commercial team. And as Matt mentioned, not a lot of inventory or anything like that.
Speaker Change: These three things together actually.
Speaker Change: <unk> and costs.
Speaker Change: Slightly better net revenue per unit and what we were expecting but we expect all this to normalize throughout the course of the year.
Speaker Change: Great. Thank you Mr <unk>.
Speaker Change: The results are reflective of strong underlying demand for <unk>.
Speaker Change: The execution of our commercial team and as Mac mentioned, not a lot of inventory or anything like that.
Tom Tremarkey: Thanks, perfect.
Speaker Change: Thanks perfect.
Speaker Change: Okay.
Cory Kasimov: Your next question comes from the line of Cory Kasimov of Evercore. Please go ahead. Hey, good afternoon, guys. Thanks for taking the question. So it's great to see that Ruby's off to such an impressive start. And you kind of alluded to this in a prior response. But now that you're on the market for for over a quarter, how are you thinking about new patient starts for the category going forward? I know previously you've talked about numbers like two to 3000 restarts per quarter, but that's already looking pretty conservative. So any kind of new color there?
Speaker Change: Next question comes from the line of Cory <unk> from Evercore. Please go ahead.
Cory: Hey, good afternoon, guys. Thanks for taking my question.
It was great to see our resolve to such an impressive start and you kind of alluded to this in a prior response, but now that you're on the market for over a quarter. How are you thinking about new patient starts for the category going forward I know previously you've talked about numbers like two to 3000 restarts per quarter, but that's already looking pretty conservative so any.
Cory: Color there would be appreciated thank you.
Matt Oughton: Yeah, hey, Cory, thanks for the question. I think you're kind of thinking about it, like we're thinking about it, it does seem to keep going up every quarter. And if you look historically, you know, that's no different. I think it is ramping a bit faster. But that also makes sense, because you're getting new products in the market. So the more companies that launch a product into a space, that creates a higher share of voice. And I think that gets people educated either to go get screened, or for physicians to think to themselves, I mean, if you're a cardiologist, and you have test patients, and you're not thinking to yourself, hey, some of these must be ATTRCN patients, all of these companies are discussing it, educating, talking about it, it gets people looking.
Cory: Yes, Hey, Corey Thanks for the question I think you're kind of thinking about it like we're thinking about it it does seem to keep going up every quarter and if you look historically.
Cory: No different I think it is ramping a bit faster, but that also makes sense, because youre getting new products in the market. So the more companies that launch a product into a space that creates a higher share of voice and I think that gets people educated either to go get screened or for physicians to think for themselves I mean of euro cardiac.
Cory: <unk> and you have <unk> patients and you are not thinking to yourself hey, some of these must be <unk> patients. All of these companies are discussing it educating talking about it it gets people looking and if you look you find it.
Matt Oughton: And if you look, you find that it's, it's not that uncommon. You know, most cardiologists have some patients that need to be treated. So I think that, you know, we don't see that stopping anytime soon. The market could easily be a $20 billion market, and we're not there yet. So there's still a lot of patients who are out there who have not been diagnosed. But I think you're going to see continued high numbers of people being diagnosed now that that interest is just continuing to grow.
Cory: Not that uncommon.
Cory: Most cardiologists have some patients that need to be treated so I think that.
Cory: We don't see that stopping anytime soon the market could easily be a $20 billion market and we're not there yet so there's still a lot of patients who are out there who have not been diagnosed but I think youre going to see continued client members.
Speaker Change: Of people being diagnosed now that that interest is just continuing to grow and Corey just to add one last thing a lot of the new diagnosis is coming from the high volume heart failure clinics that Max mentioned in his remarks.
Matt Oughton: Yeah, and Corey, just to add one last thing, you know, a lot of the new diagnosis is coming from the high volume heart failure clinics that Matt mentioned in his remarks, and obviously an oral small-molecule stabilizer is a great fit for that. Absolutely, it's very helpful.
Cory: Obviously, an oral small molecule stabilizer as a great fit for those patients.
Cory: Absolutely that's very helpful. Thank you.
Greg Harrison: Your next question comes from the line of Greg Harrison of Scotiabank. Please go ahead. Hey, good afternoon, guys. Congrats on the huge start to the launch. And thanks for taking our questions.
Greg Harrison: Your next question comes from the line of Greg Harrison of Scotiabank. Please go ahead.
Greg Harrison: Hey, good afternoon, guys. Congrats on the huge start to the launch and thanks for taking our questions.
Matt Oughton: So in our initial modeling conversations with you guys, our takeaway was that your expectation was for initial uptake to come, you know, primarily or almost exclusively from newly diagnosed patients. But to get to the revenue number you've reported, you have to get most of them by our math. So assuming that's not the case, you know, is this a function of much larger market growth than you expected, or are you getting a large percentage or maybe even most of your patients as tofamidiz switches? And if that's the case how do you expect this trend to evolve from here?
Speaker Change: So in our initial modeling conversations with you guys or takeaway was that your expectation was for the initial uptake to come.
Greg Harrison: Primarily or almost exclusively from newly diagnosed patients.
Greg Harrison: To get to the revenue number you reported you have to get most of that by our math.
Greg Harrison: So assuming that's not the case.
Greg Harrison: This.
Greg Harrison: A function of.
Greg Harrison: Much larger market growth there.
Greg Harrison: Than you expected or.
Greg Harrison: Are you getting a large percentage or maybe even most of your patients as to fabulous switches.
Greg Harrison: That's the case, how do you expect this.
Greg Harrison: Just trying to evolve from here.
Matt Oughton: Hey, Greg, thank you so much for the question. So I think as we have said consistently, our focus at launch has been the treatment naive market. Very early on, we got a few more switch patients than we expected, but that started to normalize. Focus continues to be new treatment naive patients. And I think Matt mentioned in his remarks that there we are seeing consistent monthly growth, gradually. In terms of what's driving the number, I think it's sort of all of the above, right? I think it's a little bit of the market growth, obviously strong demand and strong conversion.
Speaker Change: Hey, Greg. Thank you so much for the question. So I think as we've said consistently our focus at launch has been the treatment naive market.
Speaker Change: Early on we got a few more switch patients than we expected, but that start to normalize.
Speaker Change: Our focus continues to renew are treatment naive patients.
Speaker Change: Matt mentioned in his remarks that there we are seeing consistent monthly growth gradually.
Speaker Change: In terms of what's driving that number I think it sort of all of the above right I think thats.
Greg Harrison: Little bit of the market growth you know, obviously strong demand and strong conversion, maybe I'll turn it on to Matt and he can talk a little bit more about the factors driving conversion and the market size.
Matt Oughton: Maybe I'll throw it on to Matt and he can talk a little bit more about the factors driving conversion and the market. Yeah, no, I think you, I think you hit it well, Chinmay. I don't know that I would add that much. You know, it's a little tricky when you try to figure out who a switch patient is. You know, they can, they look like a new patient most of the time. So I think it's a little, it's a little careful trying to determine who's a switch and who's new. Our focus is on the newly diagnosed.
Matt Outten: Yes, no I think I think you hit it well Jim.
Speaker Change: I don't know that I would add that much.
Speaker Change: It's a little tricky when you try to figure out who is switch patient is.
Speaker Change: They can they look like a new patient most of the time. So I think it's a little easier a little careful trying to determine who the switch and who knew our focus is on the newly diagnosed we do see switch patients coming in and sometimes we can tell that their switch patients other times it is not clear.
Matt Oughton: We do see switch patients come in and sometimes we can tell that they're a switch patient. Other times it's not clear.
Matt Oughton: But I think, as you think about the rest of 25, moving into 26, I think the market continues to grow. It's been growing double digits now for quite a long time. And this past quarter, you know, 1 of the best we've seen. I would not expect that to disappear anytime soon. There will be switch patients who want to true be, and there will be a lot of newly diagnosed patients that want to true be for all the reasons we've discussed, whether it's the data itself, the programs we offer. I think we're very optimistic about all of that.
Speaker Change: But I think as you think about the rest of 'twenty five moving into 2006 I think the market continues to grow it's been growing double digits now for quite a long time and this past quarter. One of the best we've seen I would not expect that to disappear anytime soon there will be switch patients who wanted truly and there will be a lot of newly diagnosed.
Speaker Change: Those patients that wanted to review for all the reasons, we've discussed whether it's debt to.
Speaker Change: The data itself the programs we offer.
Speaker Change: I think we are very optimistic about all of that.
Greg Harrison: Great, thanks again.
Speaker Change: Okay.
Speaker Change: Great. Thanks again.
Paul Choi: Question comes from the line of Paul Choi, Goldman Sachs. Please go ahead. and congratulations on knocking it out of the park in your first quarter here on the lawn.
Speaker Change: Your next question comes from the line of Paul Choi Goldman Sachs. Please go ahead.
Speaker Change: Okay.
Speaker Change: Congratulations on that knocking it out of the park in your first quarter here on the launch.
Tom Tremarkey: I want to just shift gears for a minute, maybe just ask a policy question, which is to think about as a truly growth in scale over the coming years here, or this year and next year, as well as these start to get sales from Europe, from Bayer and on Biantra and AstraZeneca in Japan, could you maybe just help us think through where your IP is domiciled and just sort of the tariff implications and just sort of the royalty streams that you'll be getting from your ex-US partners and just sort of how that ultimately affects your margin and your sort of profitability profile?
Speaker Change: I wanted to just shift gears for a minute and maybe just ask a policy question glitches to think about.
Speaker Change: As a true re growth in scale over that over the coming years here or this year and next year.
Speaker Change: As well as you start to get sales from Europe from a buyer and on the Entre and Astrazeneca in Japan could you maybe just help us think through.
Speaker Change: Where your IP is domiciled I'm, just sort of a tariff implications I'm just sort of the royalty streams that youll be getting from from your ex U S partners and just sort of how that ultimately affects your margin story.
Speaker Change: Sort of profitability profile any color there or directional guidance would be helpful. Thanks.
Tom Tremarkey: Any color there or directional guidance would be helpful. Thanks.
Tom Tremarkey: Hey, Paul, thanks for the question. This is Tom. Yeah, so as Neil indicated in his prepared remarks, we're fortunate to have very little impact. From any of the tariff discussions that are ongoing. We've done a pretty deep dive on this. As you know, the pharmaceuticals have been exempt from the reciprocal tariffs. But we've also looked at the IEPA and what's been said in public around the potential 232 tariff. the industry. When we put all that together, based on our supply chain, we see very, very minor impact. That's because Attribute is made here in the USA.
Speaker Change: Okay.
Tom: Paul Thanks for the question this is Tom.
Speaker Change: So as Neil indicated in his prepared remarks.
Speaker Change: We are fortunate to have very little impact from from any of the tariff discussions are ongoing we've done a pretty deep dive on this as you know the pharmaceutical has been exempt from the reciprocal tariffs.
Speaker Change: But we will also look at <unk> and what's been said in public around the potential to 32 tariffs affecting the industry when.
Speaker Change: When we put all that together based on our supply chain, we see very very minor and diagnosed because attributes made here in the USA and then we're of course, an American company and we have all our IP domiciled here in the U S. So overall very minor impact to the business.
Tom Tremarkey: And then we're of course, an American company and we have all our IP dominant. overall very minor impact to the business.
Tom Tremarkey: I'm not sure I understood the question on on the royalties and how that relates. But, you know, that's off the top line revenue number. So obviously, Money coming to us is before any any implication on tariffs that would be on their end, but maybe it didn't. No, that makes sense.
Speaker Change: I'm not sure I understood. The question on on the royalties and how that relates but.
Speaker Change: The top line revenue number so obviously we're getting.
Speaker Change: Money coming to us as before any any implication on tariffs that would be on their own but maybe I didn't understand the question.
Speaker Change: No.
Speaker Change: Makes sense.
Ross: Ross doses.
Speaker Change: Okay.
Anupam Rama: Your next question comes from the line of Anupam Rama of J.P. Morgan. Please go ahead.
Speaker Change: Your next question comes from the line of Anil Com Rama of Jpmorgan. Please go ahead.
Neil Kumar: Hey, guys, thanks so much for taking the question and congrats on the quota with a Truby. I wanted to just pop in with a very quick pipeline question. So you shared some data today on the chronic hypoparathyroidism indication. So what gets you excited about this opportunity, particularly from, you know, you talked about Neil from an NPV perspective, how does that all fit in? Thanks so much.
Speaker Change: Hey, guys. Thanks, so much for taking my question and congrats on the quarter with a truly I wanted to just pop in with a very quick pipeline question. So you shared some data today on the chronic hyperparathyroidism indication. So what gets you excited about this opportunity, particularly from you talked about Neil from an NPV perspective, how does that all fit in.
Speaker Change: Thanks, so much.
Anupam Rama: Hey Anupam, thanks for the question. We're going to drive it to Neil to talk about the NPV and then Anupam can talk more about the data and our exciting plans for the phase. Yeah, I mean, from an MPV standpoint, Noop, I think you know that this is an exciting extension population, quite a bit larger than ADH1 itself with actually a pretty reasonable price point as well, given where Ascend has ended up pricing their medicine. So there's a variety of different ways for us to take advantage of the opportunity now that many of the oral competitors have fallen by the wayside.
Speaker Change: Hey, Bob Thanks for the question, we're going to drive it to Neil to talk about the MPD and then announce and talk more about the data and our exciting plans for the phase III in HP.
Speaker Change: Yes, I mean from an NPV standpoint, new but I think you know that this is an exciting extension population quite a bit larger than 80, H. One itself was actually a pretty reasonable price point as well given where our center senator pricing their medicine, so and there is a variety of different ways for us to take advantage of the opportunity now that many of the.
Speaker Change: The oral competitors have fallen by the wayside.
Anupam Rama: I'm going to actually pass it over to Anup to talk a little bit about the exciting data that we just Hey Anupam, great question. Thanks for your interest in this program. So, as you mentioned, we reported data from the first nine participants of post-surgical hypoparathyroidism that were treated with Encalerate. Normalized blood and urine calcium concomitant. Transcripts provided by Transcription Outsourcing, LLC. Transcription Outsourcing, LLC. from the library. The President of Incalerate.
Speaker Change: I'm going to actually pass it over to <unk> to talk a little bit about the exciting data that we just posted.
Speaker Change: Hey, Great question. Thanks for your interest in this program. So as you mentioned, we reported data from the first nine participants with post surgical hyperparathyroidism were treated with <unk>.
Speaker Change: Normalized blood and urine calcium concomitant Wei and <unk> 78 of these participants and serves to be.
Speaker Change: Important oral option for these patients seeking to resolve calcium homeostasis. So we think that this could be a treatment changing.
Speaker Change: A paradigm changing treatment and we will advance development towards registration for this program in terms of the NPV or the market opportunity like Neil mentioned this is about this by 7% to eight times larger marketplace than the aviation market loans. So it can grow and expand there.
Speaker Change: The presence of <unk>.
Speaker Change: As we continue to investigate molecule.
Anupam Rama: Thanks so much, guys.
Speaker Change: Thanks, so much guys.
Allie Merle: Your next question comes from the line of Allie Merle of UBS, please go ahead. Hey guys, thanks for taking the question and congrats on the quarter. I'm curious what you're seeing so far in terms of commercial trends since Ambutra's approval. You have a few weeks of experience now with this so far. So, I mean, I guess specifically, was the rate of new unique patient prescriptions consistent with what you saw in February and March? Did you see an increase by an April and associate with a broader prescriber base? Or have you maybe seen a dip in the rate of new unique patient starts just with the launch of a competitor?
Speaker Change: Your next question comes from the line of Elie Merrill of UBS. Please go ahead.
Elie Merrill: Hey, guys. Thanks for taking the question and congrats on that.
Elie Merrill: So what youre seeing so far in terms of commercial trends stents antitrust approval.
Elie Merrill: And experience know that so far.
Elie Merrill: So I mean, I guess, specifically it was the right.
Elie Merrill: Patient prescriptions consistent with what you saw in February and March.
Elie Merrill: <unk> is an important difference.
Elie Merrill: Farmer base or have you maybe seen in cap rate.
Speaker Change: Since with the launch of a competitor and any sort of high level commentary on how you're thinking about positioning are there certain segments, where you see potential greater uptake.
Matt Oughton: And any sort of high-level commentary on how you're thinking about positioning? Are there certain segments where you see potential greater uptake or advantages in terms of your use?
Elie Merrill: Or.
Elie Merrill: Awesome.
Elie Merrill: Yes.
Matt Oughton: Sir, this is Matt. Thank you. For the second half of your question, the audio was a little garbled, so let me answer the first one and then. The second one. address that as well, your first one regarding Ambutra. You know, I, there's definitely a place for lots of choices in this market. And I think as a company, we're obviously terms of where Ambutra is going to be placed, you know, they seem to be earmarked for mixed phenotype at the moment. That's probably 10% of the overall market. you know, their variant data wasn't that big, and they're very expensive.
Elie Merrill: Sure This is Matt.
Elie Merrill: Thank you for the second half of your question.
Elie Merrill: Audio was a little garbled. So let me answer the first one and then if you want to repeat the second one I'm happy to address that as well your first one regarding <unk>.
Elie Merrill: <unk>.
Elie Merrill: There's definitely a place for lots of choices in this market and I think as a company. We're obviously happy when patients have additional choices in terms of where <unk> is going to be placed.
Elie Merrill: They seem to be earmarked for mixed phenotype at the moment, that's probably 10% of the overall market.
Elie Merrill: Theyre variant data wasn't stat, Sig and they are very expensive. So it's twice as expensive as a truly right now.
Matt Oughton: So it's twice as expensive as a true be right. and they have to compete obviously with our 340-250 which has been for the Got it. And then just maybe this was a part of the question that broke off. Let me know if you can't hear me. But just in terms of the rate of unique patient prescriptions, I guess, have you seen any change in that since the approval of Infutra? So, you know, for instance, if you saw a dip in the rate of new, unique patient prescriptions in April or say now that you have a broader prescriber base, you've seen a similar rate in new patient prescriptions or even higher.
Elie Merrill: And they have to compete obviously with our $342 50, which has been.
Elie Merrill: Pretty successful so I don't I don't know, where thats going to land for them I think they have to figure out their pricing.
Elie Merrill: If youre going to charge doubled you need to have better results and I think you can ask them to talk about that I'm sure. They will have opinions on that but.
Elie Merrill: Right now with <unk>.
Elie Merrill: Early but we're not seeing a lot of uptake outside of the mixed phenotype.
Speaker Change: Got it and then just maybe I missed the part of the question that broke off may not be you can't hear me, but just in terms of the rate of unique patient prescription.
Elie Merrill: Have you seen any change in that.
Speaker Change: Since the approval options.
Speaker Change: And you saw that in the rate of new unique.
Speaker Change: Patient prescriptions in April or ethane that you have a broader prescriber base, you've seen a similar rate in new patient prescriptions or even higher.
Matt Oughton: Thanks. Yeah, so the April data, I probably wouldn't be able to comment on. I mean, you know, they were sort of out two weeks in Q1. And now, you know, we picked up some data, we see some of the same stuff that you guys see. The trouble is, you know, even, you know, it's buy and bill. And so, you know, they're trying to convince a community physician to put out over $100,000 per injection. You know, the vitamin A part is cheap. So, you know, from a cost perspective, that's fine. You still have to take that every day.
Speaker Change: Yes, so the April data I, probably wouldn't be able to comment on I mean, they were sort of out two weeks in Q1 and now we've picked up some data we see some of the same stuff that you guys see.
Speaker Change: The trouble is.
Speaker Change: It's buy and bill and so.
Speaker Change: They are trying to convince the community physicians to put out over $100000 per injection.
Speaker Change: The vitamin a par cheap so from a cost perspective that signed is the uptake that everyday with that out of pocket for the doctor. They have the cash flow that so they have to put that money out and then they have to wait to get reimbursed.
Matt Oughton: But that out of pocket for the doctor, they have to cash flow that. So they have to put that money out, and then they have to wait to get reimbursed. Well, if you start stacking up a lot of patients, that's a lot of money. And it gets pretty expensive. So we haven't seen it yet. It doesn't necessarily mean it is or isn't happening. I think you'd have to ask them. Maybe they can do what we did and throw out some early numbers on what the first month of launch . Got it. Thanks. No, thank you, I appreciate the question.
Speaker Change: You start stacking up a lot of patients that's a lot of money and it gets pretty expensive. So we haven't seen it yet it doesn't necessarily mean, it is or isn't happening.
Speaker Change: I think you'd have to ask them, maybe they can do what we did in neuro ophthalmology numbers on what the first month of launch looks like.
Speaker Change: Okay got it thanks.
Speaker Change: Okay.
Speaker Change: No. Thank you.
Speaker Change: Comes from the line of Jason Szymanski with Bank of America. Please go ahead.
Jason Zemansky: Great.
Jason Zemansky: Good afternoon. Congratulations on the quarter and thanks for taking our questions. I wanted to return to the question of segment growth for Atrubi, if I may. It sounds like you expect the contribution from switches to slow at some point, but what does that look like over the near term? I mean, do you have a sense of how quickly you may be going through a bolus of, I don't know, refractory patients versus those, as you mentioned, you know, are looking for a Hey, Jason, thanks for the question. I think that high level, I can say a couple of things and then Matt's going to add more details.
Jason Szymanski: Great. Good afternoon, congratulations on the quarter and thanks for taking our questions I wanted to return to the question of segment growth for <unk>. If I may it sounds like you expect the contribution from switches to slow at some point, but what does that look like over the near term I mean do you have a sense of how quickly you may be going through a bolus.
Jason Szymanski: I don't know refractory patients versus those as you mentioned are looking for a stronger stabilizer because they believe.
Jason Szymanski: And the better outcomes over time.
Jason Szymanski: Hey, Jason Thanks for the question.
Jason Szymanski: That.
Jason Szymanski: A high level I can I can say a couple of things and then Matt will add more details back.
Matt Oughton: But as we've said all along, you know, we're not seeing any bolus or anything like that. I do think that the focus of our launch is Treatment Naive and there we're seeing consistent monthly share growth and we expect that to continue. We had 100% share in the switch population and obviously that's going to evolve now that there's a competitor. So that's sort of how we're thinking about it. Obviously, it's very early to say and we look forward to seeing how the next quarter. I'll just add, I mean, I agree, we didn't see Ebola. We don't spend a lot of time sort of thinking about it from that perspective.
Jason Szymanski: As we have said all along we're not seeing any bullets or anything like that I do think that the focus of our launches treatment naive and there we're seeing consistent monthly share growth and we expect that to continue we had 100% sure in the switch population and obviously that's going to evolve now that there is a <unk>.
Jason Szymanski: So that's sort of how we're thinking about it obviously, it's very early to say and we look forward to.
Jason Szymanski: Seeing how the next quarter goes.
Jason Szymanski: Yes, I'll just add I mean, I agree we didn't see a bolus I mentioned earlier, it's a little tricky to absolutely know if someone has a switch or a newly diagnosed we don't spend a lot of time sort of thinking about it from that perspective is patients want to get a truly we try to make sure. They can get it for <unk>.
Matt Oughton: If patients want to get a Truvy, we try to make sure they can get it for whoever they are newly diagnosed or switch. In terms of what that looks like over time, I think that's hard to predict. You can take the number of patients onto famitids, you can add on a progression rate of whatever you think is correct and then kind of model that out over time. I think that's probably the best approach you have.
Jason Szymanski: Whoever they are newly diagnosed or switch in terms of what that looks like over time, I think thats hard to predict you can take the number of patients on to <unk> you can add on a progression rate of whatever you think is correct and then kind of model that out over time, I think thats, probably the best approach of adding.
Operator: There are no further questions at this time. And that concludes our Q&A for today. I will now hand this call back over to the company. Please go ahead. Thanks everyone for your questions today. We appreciate your interest in Bridgebio and look forward to updating you again next quarter. This concludes our conference for today. We thank you for participating and ask that you please disconnect your line.
Speaker Change: There are no further questions at this time and that concludes our Q&A for today I will now hand, the call back over to the company. Please go ahead.
Speaker Change: Thanks, everyone for your questions today, we appreciate your interest in <unk> and look forward to updating you again next quarter.
Speaker Change: This concludes our conference for today, we thank you for participating and ask that you. Please disconnect your lines.
Speaker Change: [music].