Earnings Call Transcript - BBIO Q1 FY2025

Operator

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. 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 bridge bios, future operating and financial performance, business plans, and prospects and strategy. These statements are based on current expectations and assumptions that are subject to risks and uncertainties, which could cause actual results to differ materially from those expressed or implied in these forward-looking statements. For a discussion of these risks and uncertainties, please refer to the disclosure in today's earnings release and BridgeBio's periodic reports and SEC filings. All statements made here are based on information available to BridgeBio's as of today, and the company undertakes no obligation to update any forward-looking statements made during this call, except as required by law. With that completed, BridgeBio, you may begin your conference.

Chinmay Shukla, Other

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. We'll provide insight into the early results from the launch of a Truby 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. Today's call will feature Neil Kumar, Chief Executive Officer, Matt Outen, Chief Commercial Officer, and Tom Trimarchy, President and Chief Financial Officer. For the Q&A portion of the call, Anant Sridhar, Chief Operating Officer of Bridge Bio Cardio Reno, and Justin Toh, Chief Executive Officer of Bridge Bio Schedule Dysplasias, will also join. Following the remarks by our team, we will open up the call for Q&A. With that, I'll turn the call over to Neil.

Neil Kumar, CEO

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. Today, we know the star of the show is the Atrubi 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 and cardiovascular hospitalization at that same time point. All these best-in-class point estimates are available at the lowest price point in the marketplace. There is much to like there. 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. We add to a Truby's good news the continued progress across our pipeline of three additional blockbuster products. Our trials in limb-girdle muscular dystrophy type 2I, achondroplasia, 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 with incalorate 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. Before we move to the 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. Number two, a higher and anticipated revenue driven by a faster uptake of a Truby than we anticipated and likely a slightly higher peak year share by volume than market research had projected. Third, the success of our small study in HP, and other sponsor 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 NPV. Stepping back from NPV and as a reminder, our overall objective here at Bridge Bio 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 means 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. 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, in 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 decision as to what drug to use, a logical person would first interrogate the salient endpoint, death and hospitalization, at a common time point. 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, Atruvi 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, 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 as observed in the NPATRO study, and the as of yet unexplained imbalance in death on the review Saran 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. Finally, the logician might look at price only to discover that a Truby has the lowest price point we believe given all of that the choice is clear we apply similar reasoning in other competitive spaces like achondroplasia our final constraint is that each program we work on should be mpv positive to ensure firm level sustainability we use all available levers during the r d stage to optimize mpv 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 MPV-positive projects. The fact that we will spend well under $100 million per program to get each one of our potential blockbusters from the pre-clinical stage through proof of concept sets us up well for future growth that is economically attractive. 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. There are other frameworks that we also use to look at the business, understanding the business by stage and capability, research, development, and commercial, understanding the business in the context of the overall ecosystem, and understanding the business program by program. 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 than another would be able to. I've also touched already on our ongoing development wins. On Discovery, our bread and butter, we continue to advance programs in genetic dilated cardiomyopathy and ADPKD. We also hold significant stakes in bridge bio-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 finally at a program level matt will have more to say about a commercial launch of a truby our goal there as a reminder is 4.3 billion dollars in peak year sales or about 30 percent of a 15 billion dollar marketplace that gets delivered against approximately 380 million dollars 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 thickest of patients, and new work we are doing regarding AFib, which is an emerging marker of disease progression, the first results of which we plan to present at ESC later this year. For infagradinib, 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. We have also reached regulatory alignment with the FDA on our clinical development plan for infagratinib 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 two hypochondroplasia trial well ahead of benchmarks. For incalorate, a negative allosteric modulator of the calcium sensing receptor, our phase three calibrate study is fully enrolled. We expect last patient, last visit, and top-line results in the second half of this year. In parallel, incalorate is also being studied in hyperparathyroidism, and we announced today positive POC data for incalorate in this key expansion indication. Polymerary 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 incalorate administration. Moving to BBP418, an oral first-in-class disease modifying therapy in Phase III development for treatment of individuals living with limb girdle muscular dystrophy type III, we have fully enrolled Fortify, a Phase III registrational placebo-controlled study evaluating the safety and efficacy of BVP 418. The study includes a planned interim analysis of 12 months focused on assessing a surrogate endpoint biomarker, glycosylated alpha-distroglycan, to support accelerated approval in the United States. We anticipate top-line readout from that Phase III interim analysis second half of this here as well. Finally, BBP812 is an AAV9 gene therapy in development for Canavan 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 Canavan 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 the disease at its source and operating leanly to enable us to go after a well-validated condition in an NPV-positive manner. Okay, that's the final framing. 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 Bridge Bio. Number three, a diversity of stakes, including wholly owned assets and significant stakes in Gondola Bio and Bridge Bio 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. 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 Atrube's commercial performance in more detail. Thanks, Neil. To add to your comments,

Matt Outten, CFO

the launch of Atrube 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 TRUBI through April 25th, and 756 unique health care providers have written at least one prescription. 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 a TRUBI used across the full spectrum of patients, wild-type invariant, newly diagnosed, as well as switches from partial stabilizers. 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 Atruvi. So, let's discuss what is driving the uptake. It comes down to strong clinical endpoints paired with our transparent patient-first support programs. 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. 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 composite of 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. And third, affordability. Atrubi is the most cost-effective therapy in ATTRCM available, 10% less expensive than tefamidus and 50% less expensive than nutricerine. It's not just about the WAC price, though. Atrubi has a free trial program for all patients, regardless of insurance status, 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-month onset of treatment effect and 50% reduction in cardiovascular hospitalization at 30 months. No other ATTR-CM therapy checks all of these boxes. BridgeBio has a commercial model that is different and it's working. A Truby will be followed by three additional commercial launches in 2026 and 27. 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. 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 Atrubi, we make it easy to get the medication to patients. Patients can receive Atrubi within 48 hours. We have two world-class specialty pharmacies in the Atrubi network and also allow physicians to fill prescriptions in their own in-house pharmacies. 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 benchmarks. 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 percent share of the ATTR CM market, and increase 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 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. With that, I'll turn it over to Tom for a review of the financials. Thank you, Matt, and good afternoon,

Tom Trimarchi, CFO

everyone. Q1 2025 marked Bridge Bio'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 unless otherwise indicated total revenues were 116.6 million dollars for q1 2025 and consists of a truly net product revenue and license and services revenue. A Truvi 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 a $75 million regulatory milestone related to Biantra's EU approval. Also in Q1, we received Biantra approval from 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 Attruby brand and our advancing late-stage pipeline. Included in our total operating expenses was $29.4 million of stock-based compensation expense compared to $28.9 million in the first quarter of 2024. Looking forward, we expect only modest growth in quarterly operating expenses for the remainder of the year, with an offset to total cash burn provided by Atribi sales in the U.S. and Biantra ex-US. 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 oncology business and early-stage research programs, allowing us to focus resources on the Atribi launch and late-stage pipeline. 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-U.S. approvals of BIANTRA. We believe we are well-financed to support the continued execution of the Truby launch and deliver on key milestones from the pipeline this year. 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 and achondroplasia in early 2026. With that, I'll turn the call back to Chimlay.

Chinmay Shukla, Other

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 our analysts to limit themselves to one question each so that more people get a chance to ask their questions.

Operator

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 this for a moment to compile the Q&A roster. Your first question comes from the line of Salim Syed of Nezuho. Please go ahead.

Salim Syed, Analyst — Mizuho

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. I appreciate the commentary that you provided, Matt, on some of the dynamics there. But any sort of granularity you could provide in the tailwind and what you really deem that's working well for you here.

Neil Kumar, CEO

Yeah, maybe I'll kick it off. Thanks, Saleem. 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. So I'd say that's point number one. And 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 250,000 to 300,000 in the U.S. 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. The team, I think, has done a terrific job of ensuring that patients, when they are prescribed Truvy, can get the medicine and stay on the medicine as long as they need to. So those would be the three salient drivers. 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 conditions. 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. I don't

Matt Outten, CFO

know, Matt, if you'd have anything. Well, I think to miss the people, we've spent a lot of time putting the team together, both internally and on the field, 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.

Salim Syed, Analyst — Mizuho

Great. Thanks, guys. Congrats, again.

Operator

Thanks, Wayne. Your next question comes from the line of Tyler Van Buren of TD Callen. Please go ahead.

Tyler Van Buren, Analyst — TD Cowan

Hey, guys. My congrats on the stellar attribute result as well and the ongoing progress of the pipeline. So again, the $37 million of the Truvi 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 quarter?

Chinmay Shukla, Other

Yeah. Hey, Tyler, great to hear from you. And thanks for the question. I'm going to pass down to Matt to comment more on conversion. Yeah, thanks for the question. You know,

Matt Outten, CFO

really happy with how conversion is tracking and that's for both free trial to paid and also commercial prescriptions to paid everything regarding conversion is consistent or better than historical launches that we've studied 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 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 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 hey Tyler this is Tom let me just put

Tom Trimarchi, CFO

a finer point on the inventory question as I mentioned the prepared marks the the sales in the quarter are primarily driven by demand so we've seen only a minor impacted inventory on total sales in the quarter. So really demand-driven here.

Chinmay Shukla, Other

Yeah, thanks, Tyler. I know you had another question. So happy to answer more about this or go to your next question.

Tyler Van Buren, Analyst — TD Cowan

No, that's great. Thank you.

Operator

Your next question comes from the line of Manu for Ruhar of Learink Partners. Please go ahead.

Speaker 5

Hey, guys. Congrats on the quarter. Clearly blew out expectations despite having shown pretty good scripts repeatedly. And 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 you gave on an incremental basis on february 17th that was running north of 100 scripts a week and again running through from 12 and 44 weeks into launch and 22 weeks into launch i.e from february 17th to april 25th you're still running at about 110, 109 scripts per week as reported. I know that's just math, and there's a lot of nuance that goes into script recording. Could you give us a sense of what the character and velocity of new patient scripts you're seeing right now, and sort of what is the kind of direction of travel that's there? And I have a follow-up question.

Matt Outten, CFO

I can take that. Thank you for the question. I mean, I think starting out, the thesis really, again, it remains unchanged. TTR levels go up with the Trudy, 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 that's going to impact things going forward, the month-over-month growth rate in the treatment-na-use section has been very strong, and that's been across all segments. So this is in the large centers. This is in the community. That's across all types of patients. variant, wild type. 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 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% of the switch share because we were the only option you could switch to. As now with new entrants in the market, that's going to evolve over time because there are now more choices. 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 moving forward.

Speaker 5

Great. That's helpful. 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 on 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 of years running, about the filability of that data set or whether a later data set from the same study will be required. Do any of the changes at FDA concern you around your ability to file on a biomarker? Do you think you have to show a distinct level of clear clinical improvement on a non-biomarker slash functional basis? Where are we in terms of the filability on that biomarker for limb girdle?

Neil Kumar, CEO

Yeah, 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 a 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 LGMD2I 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 agency to date in and around fallibility, 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 2, which was albeit an open-label study against natural history where we saw improvements in measures of ambulation and other clinical outcomes. So, I do believe these things will move toward the positive, or at least a few will move toward the positive. Obviously, modified North Star, North Star, which is historically 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 goes on for another two and a half years post the readout later this year. So I mean, you know, long story short, you know, we continue to believe that this is going to be an approvable endpoint in terms of ADG glycosylation increase.

Operator

Awesome. Thanks, guys.

Tyler Van Buren, Analyst — TD Cowan

Thanks, Mike.

Operator

Your next question comes from the line of Tyler Van Buren of TD Cowan. Please go ahead.

Tyler Van Buren, Analyst — TD Cowan

Yeah, I just had a second question that I meant to ask. But just the ADH1 and Limb Girl Phase 3s reading out during the second half of the year clearly have a high probability of success. So it would be great to hear you elaborate on why investors should be excited about and, more importantly, focus on those opportunities beyond the ongoing Truby launch, what you think the magnitude of those opportunities could be.

Neil Kumar, CEO

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 7,000 to 8,000 patients between the U.S. and E.U. So 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 attendant in the space. And with ADH1, a rather much more prevalent condition, maybe 10,000 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. So I think two very exciting commercial opportunities, two very exciting opportunities for first-in-class 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 could provide differential safety, differential efficacy, because it targets this well-described condition at its source, driving differential both changes in growth and then also importantly, impact on parameters that this community cares a great deal about, like proportionality and the like. And you know the size of that market from the VoxEvo launch that's ongoing. So

Operator

I think all three very exciting opportunities. Your next question comes from the line of Buren Amin of Equity Research Healthcare. Please go ahead.

Speaker 10

Yeah, hi. Thanks for taking my questions. 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 to net for the quarter and any impact that you've seen from the Part D redesign?

Chinmay Shukla, Other

Hey, Viren, thanks for the question. I'm going to pass it on to Matt to discuss the HCP piece, and then also to Tom to discuss the gross to net piece.

Matt Outten, CFO

Yeah, thanks. And I guess there's sort of two parts to this. You know, the feedback from the physician segments has been positive, as we've discussed. Our messaging around, you know, 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 hard outcomes 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 trying a Truby but then also then repeating it right those are the reasons that you sort of try a medication and then you when you see how well people are doing on it then that encourages you to try it again so we've seen a lot of repeat prescribers across a lot of the different segments as well and i don't think your expectations were otherwise or are going to change anytime soon we have very strong data package and we also have very strong programs to help patients get on the medication once that prescription has been written and then i'll pass it to neil to add on yeah maybe i'll

Neil Kumar, CEO

I'll just elaborate on that for a second before I throw it over to Tom. You know, I think the ever-increasing availability of data, like most recently, Barron, 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 thickest 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. 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 what matt suggested the backbone which is the 342 50. tom you want to talk about chicken yeah

Tom Trimarchi, CFO

sure and gross to net specifically so um yeah as we've said before the way to think about this is the floor is going to be the mandatory um ira uh rebate of 20 percent and on top of that you're going to add um you know something something like you'd see in other con uh categories for contracting is not really happening so that's what we're seeing here i would say um you know given where we are in the launch early innings we should expect some variability here quarter a 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, and we saw slightly lower use of our patient assistance program than what we were expecting. These three things together actually converged and caused slightly better net revenue per unit than what we were expecting, but we expect all of this to normalize throughout the course of the

Chinmay Shukla, Other

year. Great, thank you. Just to add that, you know, the results are reflective of 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. Thanks, perfect. Next question comes from the

Operator

line of Corey Kasimov of Evercore. Please go ahead. Hey, good afternoon, guys. Thanks for

Cory Kasimov, Analyst — Evercore

take in 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 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 2,000 to 3,000 restarts per quarter, but that's already looking pretty conservative. So any kind of new color there would be appreciated. Thank you.

Matt Outten, CFO

Yeah. 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, 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 HFPEF patients, and you're not thinking to yourself, hey, some of these must be ATTRCM patients. All of these companies are discussing it, educating, talking about it. It gets people looking. And if you look, you find it. It's not that uncommon. Most cardiologists have some patients that need to be treated. So I think that 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.

Chinmay Shukla, Other

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 monocular stabilizer is a great fit for those patients.

Cory Kasimov, Analyst — Evercore

Absolutely. It's very helpful. Thank you.

Operator

Your next question comes from the line of Greg Harrison of Scotiabank. Please go ahead.

Greg Harrison, Analyst — Scotiabank

Hey, good afternoon, guys. Congrats on the huge start to the launch and thanks for taking our questions. 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 diagnose 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 to families switches and if that's the case how do you expect this trend to evolve from here

Chinmay Shukla, Other

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, you know, 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, you know, obviously strong demand and strong conversion. Maybe I'll throw it on to Matt and he can talk a little bit more about the factors, you know, driving conversion and the market size. Yeah, no, I think

Matt Outten, CFO

you did it well, Chinma. 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. We do see switch patients come in and sometimes we can tell that they're a switch patient. Other times it's not clear. 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 in double digits now for quite a long time, and this past quarter, you know, one of the best we've seen. I would not expect that to disappear anytime soon. There will be switched patients who want a Truby, and there will be a lot of newly diagnosed patients that want a Truby for all the reasons we've discussed, whether it's the data itself, the programs we offer. You know, I think we're very optimistic about all of that. Great. Thanks again. Your next question comes from the

Operator

on Paul Charles, Goldman Sachs. Please go ahead. And congratulations on knocking it out of the

Paul Charles, Analyst — Goldman Sachs

park in your first quarter here on the launch. 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 you start to get sales from Europe, from Bayer and on Biantra and AstraZeneca in Japan. Could you maybe just help us think through, you know, where your IP is still most out 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, you know, sort of profitability profile. Any color there or directional guidance would be helpful.

Tom Trimarchi, CFO

Hey, Paul, thanks for the question. This is Tom. Yeah, so as Neil indicated in his repair marks, 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 pharmaceutical has been exempt from the reciprocal tariffs, but we've also looked at the IEPA or IEPA and what's been said in public around the potential 232 tariffs affecting the industry. When we put all that together based on our supply chain, we see very, very minor impact. That's because a Truby is made here in the USA. And then we're, of course, an American company and we have all our IP domiciles here in the U.S. So overall, very minor impact to the business. I'm not sure I understood the question on the royalties and how that relates, but that's off the top line revenue number. So obviously, we're getting money coming to us as before. Any implication on tariffs that would

Paul Charles, Analyst — Goldman Sachs

be on their end? But maybe I didn't understand the question. No, that makes sense on the ex-U.S.

Operator

business. Your next question comes from the line of Anupam Rama of J.P. Morgan. Please go ahead.

Anupam Rama, Analyst — J.P. Morgan

Hey, guys. Thanks so much for taking the question, and congrats on the quarter 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.

Chinmay Shukla, Other

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 three in HP.

Neil Kumar, CEO

Yeah, I mean, from an NPV 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 Ascendus ended up pricing their medicines. So there's a variety of different ways for us take advantage of the opportunity now that many of the oral competitors have fallen by the wayside. I'm going to actually pass it over to Anant to talk a little bit about the exciting

Anant Sridhar, COO

data that we just posted. Hey, Anantam, 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 incalerate. It normalized blood and urine calcium concomitantly in 78 of these participants and serves to be the important oral option for these patients seeking to resolve calcium homeostasis. So we think that this could be a treatment-changing or paradigm-changing treatment, and we will advance development towards registration for this program. And in terms of the MPV or the market opportunity, like Neil mentioned, this is about a seven to eight times larger marketplace than the ADH1 market alone, so it could grow and expand the presence of incalerate as we continue to

Operator

investigate the molecule. Your next question comes from the line of Allie Murrow of UBS. Please go ahead. Hey, guys. Thanks for taking the question,

Allie Murrow, Analyst — UBS

and congrats on the quarter. I'm just curious what you're seeing so far in terms of commercial trends since Amzutra'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 this is the 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? 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

Matt Outten, CFO

advantages in terms of your use? Sure. This is Matt. Thank you. So the second half of your question, the 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 Ambutra. You know, 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 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 stat fig, and they're very expensive. So it's twice as expensive as a Truby right now. And they have to compete, obviously, with our $342.50, which has been pretty successful. So I don't know where that's going to land for them. I think they have to figure out their pricing. You know, if you're going to charge double, you need to have better results. And, you know, I think you can ask them to talk about that. I'm sure that they'll have opinions on that. But right now, it's early, but we're not seeing a lot of uptake outside of the mixed phenotype.

Allie Murrow, Analyst — UBS

Got it. And then just maybe this was the 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 Ambutra? So, you know, for instance, if you saw a dip in the rate of new unique patient prescriptions in April, or let's say now that you have a broader prescriber base, you've seen a similar rate in new patient prescriptions or even higher. Thanks.

Matt Outten, CFO

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've 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's cheap. So, you know, from a cost perspective, that's fine. You still have to take that every day. 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 uh it gets pretty expensive so i we haven't seen it yet it doesn't necessarily mean it is or isn't happening um you know i think you'd have to ask them uh maybe they can do what we did and throw out some early numbers on what the first month of launch

Allie Murrow, Analyst — UBS

looks like. Got it. Thanks. No, thank you. Appreciate your question.

Operator

It comes from the line of Jason Szymanski of Bank of America. Please go ahead.

Jason Szymanski, Analyst — Bank of America

Great. 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 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 stronger stabilizer because they believe in the better outcomes over time?

Chinmay Shukla, Other

Hey, Jason, thanks for the question. I think that high level, I can see a couple of things, and then Matt's going to add more details. 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,

Matt Outten, CFO

and we look forward to seeing how the next quarter goes. Yeah, 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 is a switch or a newly diagnosed. We don't spend a lot of time sort of thinking about it from that perspective. If patients want to get a Truby, we try to make sure they can get it for, you know, 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 on safamidus. You can add on a progression rate of whatever you think is correct and then kind of model that out over time i think

Operator

that's probably the best approach of any 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

Chinmay Shukla, Other

thanks everyone for your questions today we appreciate your interest in bridge bio and look forward to updating you again next quarter this concludes our conference for

Operator

today we thank you for participating and ask that you please disconnect your lines You