Welcome to day two of our London Health Care Conference. I'm Andrew Tsai, Senior Biotech Analyst at Jefferies, and it's my pleasure to have the Bridge Bio team with me to my direct right, Thomas Tremarchi, CFO, and to his right, Matt Outen, Chief Operating Officer. Commercial Officer. Commercial Officer, I'm sorry. Well, welcome, both of you.
Thanks, Andy.
So maybe for people in the audience less familiar with the story, would you mind?
See what's coming after that. But I think we've been off to a very good start. The end of this month actually represents one year since launch. You guys have seen our first three-quarter results. So we've been very happy with the progress. I think we're really focusing on a lot of the same things that we started out doing. And that's been working. So I think we'll see where the end is. but I think we feel very comfortable with 30% to 40% in an ever-growing and expanding market.
And there has never been a head-to-head study done within the ATTRCM class, so it remains debated, at least on my side in the investor community. So in the meantime, however, you're generating some real-world data that may help you make your case that a TrueBees differentiate. So what exactly are you guys gathering and accumulating in the real world to convince more doctors to prescribe more and payers to give more access, for instance?
Yeah, I think there's two parts to the story. I'll start on the first part and then if Tom wants to add anything. I think our label for Truby is very strong. So we're the only drug that has near complete stabilization in the label. We were able to separate from placebo as early as three months. Again, not head to head, but no one's been able to show anything faster than that. And also our reduction in hospitalizations at 50%, also the best that's been seen to date. So I think between those items, doctors have been impressed, patients have been impressed. And I think that's a big reason why you're seeing physicians wanting to choose a Truby and patients asking for it when they go into the office. We've also set up a number of what we consider best-in-class patient assistance programs, extremely generous. And, you know, we get drug to patient within just a few days, which I also think is a big differentiator compared to what others are doing in the market.
I would just say that we'll get more specific in terms of data and evidence that we're generating. Our medical team has got a very broad evidence generation plan that they're executing on. And so you'll see us start to communicate a bit more through the course of 2026 on real-world evidence-type data sets as well as investigator-sponsored studies. And, of course, we're also running one company-sponsored study right now in primary prevention, and we'll be adding an imaging study on top of that starting next year as well. So very active on the evidence-generation side, which I think will allow us to, in absent a head-to-head study, continue to elaborate on our category-leading profile.
And as we think about 2026, What would you say are the key drivers to volume, sales? So far, you know, sales have grown linearly. It's not slowing down. What does it take for sales to accelerate even faster?
Yeah, it's a good question. I think there's kind of two things. It's like, one, the market continues to grow just by the number of patients that are being diagnosed, and I think we need to continue to encourage that. So, it's talking to doctors, educating patients, making sure that people are looking for this disease. Once you start looking for it, it's a rare disease, but it is somewhat common, and it still continues to be underdiagnosed. So, I think if you look historically at how the market's been growing every quarter, I don't think that slows down in 2026. In fact, I think the fact that there are now three companies out with an approved product in ATTRCM, I think that actually helps to accelerate the finding of patients. And, again, it's sort of like, you know, once you get someone to understand, it's not difficult to find a patient. PYP scans can get them everywhere. They're not invasive. We're not biopsying the heart anymore. I think that drives quite a bit. And then in terms of what we have to do as an organization, again, I think our data speaks for itself. Very strong label. And if you look at the conference presence and what's being said from the podium, even as recently as last weekend at AHA, there's a lot of really positive things being said about a Truby. And I don't see any of that changing in 26.
I must ask, then, is it your internal expectation, 2026, we see an acceleration then?
So I'm going to let you answer that.
I mean, so you've seen already in the trailing two quarters an uptick in the number of new patients that we're able to add to the brand period on period. So I would expect that trend to continue. And again, I think the main driver of this is the fact that we're consistently gaining share in the first-line treatment-naive setting and the fact that the overall pie continues to expand.
And speaking of first-line share, I believe my understanding is buyers launching in the EU in certain countries, and your first-line share already is close to 50%. So here in the U.S., I think it's just in the 20% ranges. Why the disconnect a little bit?
Yeah, so bears off to an amazing start. I think we've been obviously really happy with their performance in Germany. I think there's a few reasons for that. Um, but you know, it's, it's, it's somewhat complicated in a sense, uh, from a reimbursement perspective, right? In the U S getting a prescription is one thing, and then you have to actually go through your insurance where in Germany, uh, you don't, if you get the prescription and once the drug is covered and reimbursed, it's paid for. Uh, so I think there are some factors that you don't find, uh, in Germany that you do find in the U S healthcare. Um, there's also three products being used in ATTRCM right now in Germany, you're really only seeing two so it's just less of a pie being being split but i think it's a great market when you when it's just based on the science we're at over 50 or whatever bear has um has released on that but so i think our expectation is we'll continue to grow and uh our hope is that
bear continues to grow as well yeah i would just say they've been a fantastic partner obviously are executing extremely well in this launch and i think the fact that they're um either currently or soon-to-be market leader in Germany demonstrates the potential of this brand when you remove some of the strange market forces that we have here in the U.S. So we're really excited to see that. And, of course, we also retain 30% escalating tier of royalty, so we own a significant part of those economics. So very, very good for all of us.
And in the meantime, as we think about Q4 trends, long story short, as I feel like October, the patient ads accelerated relative to your prior update in August in terms of the weekly patient ads, but I'm curious how are November trends shaking out? Do you want me to
take that? Yeah, I mean, so we haven't obviously said very much. We keep our numbers kind of quiet until we release them at the end of the quarter. I guess what I would say, though, is that we're growing, right? We've shown that quarter over quarter. We don't anticipate anything stopping with a big focus on the newly diagnosed patients. And I think we're continuing to focus on that and expecting growth to continue as well.
Now, I just added, I mean, we've said this before, but weekly numbers are volatile. They bounce around, but we're seeing a consistent trend upward in kind of the periodic ads to the brand, which is exactly the right direction. And then I think as we think about fourth quarter, I do have to caveat all this by saying There's three fewer selling weeks usually in the fourth quarter to the holiday. So just keep that in mind as you're trying to think about what your number should be at in 4Q.
Such as Thanksgiving, New Year's, Christmas.
Thanksgiving, Christmas, New Year's. It tends to slow down, yeah.
And come January, there's another major broker conference. Do you plan to share an update on then, or should we stay tuned for Q4 EPS later in February, March?
Yeah, the January event you speak of. I mean, we're thinking about what we want to have as our main message there. And, you know, there's a bunch of different ideas, but so stay tuned.
Okay, understood. And you mentioned you're working on the – you're doing other studies in the meantime, like the ACT Early Pre-Symptomatic Trial. When does that data read out, and are there other clinical studies you're evaluating to bolster a TRUBI's differentiation?
Yeah, so there's no specific timeline guidance on the readout. That's mainly because it's an event-driven study and we're early on in enrollment, so it's a bit premature to put a firm timeline on that. But as soon as we're in a position to, we certainly will. It's a very interesting study, I think, that is consistent with our scientific and research leadership in this field, where we're enrolling variant patients that have a high risk based on family history and asking if we put them on a Truby before they have any symptoms of heart failure, can we actually prevent onset of disease? And so there'll be a Truby versus placebo, and that'll play out over the course of, I would say, the next couple of years. We also are, I believe next year, we will be kicking off a cardiac imaging study to elaborate on the sub-study that we actually had in the attribute phase three where we showed actual reversal of amyloid burden in the heart as well as improvement in cardiac function. So we'll be looking to elaborate on that through a larger prospective multi-center study and something we're very excited about.
And then maybe one last question before we turn to the pipeline. We're approaching one year into the launch of a TrueBeat. Does it make sense to provide guidance? What's your philosophy on that?
Let me turn that around on you. Do you think we should give guidance? I mean, we're thinking about it. You know, look, I think the... Just don't say I'm back. I mean, look, we've evolved our relationship with the street in terms of the commentary on quarters. And I think right now, you know, it could be a good opportunity to start providing guidance. But at the same time, we've been given the wisdom from others that you should wait as long as you can. But I'm curious what you think we should do.
I think it could make sense to think about it to help investors. We can do a vote. But my take is it's appropriate. The timing is okay. Well, we'll look into that. So moving to the pipeline, you have LGMD successful, ADH1 successful, Echondro hopefully successful too. Is it your intention to market these three drugs yourselves in both the U.S. and ex-U.S.?
Yeah, I'm going to take that.
Yes. So I think there's several things that are happening. We are building out our XUS capabilities. We do have an office in Switzerland right now, and the plan is to market these drugs ourselves. And then, of course, we have the great partnership with Bayer in Europe and Alexion in Japan, but then we would also be marketing Acroamidus in the rest of the, you know, not every country, but obviously then certain countries will take a Truby, what's known as Biantra, into those countries as well. So it's quite a lot going on, actually, over the next 18 months for us with potentially three new drug launches plus the Biantra launches in those other countries. So if you know people looking for a job, we're hiring.
Okay, very good. So maybe LGMD first filing early 2026. But you'll have a data update. I think you mentioned MDA conference in 2026. um how much more what can we expect you to share in that next analysis yeah so just taking a step
back um this is a first in class therapy for a type of muscular dystrophy called lgmd2ir9 we've read out our interim top line data just a couple weeks ago where we showed success on the primary endpoint with a very small p-value p less than 0.0001 that was the agreed upon, you know, primary endpoint for an accelerated approval path with the FDA. At the same time in the interim, we showed really a profound improvement on two functional endpoints that we've disclosed so far, FEC or forced vital capacity, which suggests the patients can actually breathe better after taking our drug versus where they were at baseline, and also a significant improvement on 100-meter time tests, again, suggesting better ability to ambulate versus pre-therapy. So really, I think unique and profound data in the context of many muscular dystrophy trials that we've looked at. So yeah, we plan to publish, present the data at MDA, publish in the journal following that. I think what you can expect at MDA is a deeper analysis on the primary endpoint and key secondaries that we've shown the top line, but you'll have the context of baseline, change over time, things like that that you'll typically see in a podium presentation. And then I think you should look for us to keep the drumbeat of presence at the muscular muscle meetings and follow up with a journal article as well.
And remind me how many patients are there in the U.S. versus ex-U.S.?
So there's about 7,000 in U.S. and Europe. Two to 3,000 will be in the U.S. The rest are in Europe. and we see this as a clear billion dollar plus opportunity, no competition, none really in the pipeline, anywhere close. So with these data, I think we're tremendously excited about the
potential to change patients' lives. And let's just say you file, you will be filing early 2026. This could be approved by early 2027. On day one, how many patients will you have line of sight to
in the u.s yeah so so this is a fairly well mobilized and diagnosed population um we know that because of the pace of enrollment that's sometimes kind of like a biomarker for how how how big is the population in the wild um this enrolled uh about eight months ahead of schedule 20 over enrolled we've also run a natural history that's got about 150 participants there's a another u.s natural history study so there there are hundreds if not over a thousand patients that are kind of counted in clinical trial or actual observational studies and databases. So there's a big pool of patients there. The biggest thing that we're going to be trying to drive out into the community in the next year is really just awareness and testing. Most limb girdle patients have a diagnosis of limb girdle. It's just knowing that you have two eye versus another type, that that's something we need to work on. And there's also some belief that there may be some misdiagnosis within the Becker population, but that's also going to be a priority for us.
And how does this interim finding, I mean, you're not just seeing stabilization procedure, you're seeing improvement in FEC. How does this instill confidence in the final primary endpoint, I think, out to 36 months for this phase three study, which is North Star, I believe.
Yeah, it's North Star. So, I mean, we were not expecting to be able to observe a statistically significant change in any of the functional endpoints at 12 months. That's the whole reason for the regulatory strategy leading up to the top-line readout, which was to demonstrate that alpha-DG, our biopsy-based marker, is reasonably likely to predict subsequent actual clinical benefit. Now, we've seen actual clinical benefit at 12 months in a subset of the patients, which really is just an upside surprise in terms of the effect size, which we were not anticipating. So I think it's very de-risking for the longer-term endpoint, and our team is working tirelessly on getting in front of the agency to talk about kind of next steps.
Okay. And the same compound is going after another LGMD subtype maybe next year. And should we expect a similar trial design as 2I? Or is it 2I that you're pursuing?
There's 2M and 2U are smaller indications with the same kind of same pathomechanism. Then there's also a condition called Fukuyama that has the same kind of defect in the alpha dysrhyglycine complex where this medicine could be used as well. That's about 1,000, thousand patients in Japan, which we'll also be looking to run an expansion trial there.
Okay. Thank you. Shifting gears to ADH1, also successful, filing an NDA early 2026. So how many patients? What's the peak sales potential? How are you thinking about price?
Yeah. So this is Sincalorit, which is our calcium-sensitizing receptor inhibitor for hypoparathyroidism. The first indication is a genetic form of hypoparathyroidism called ADH1, which arises from an activating mutation in the calcium sensitive receptor. So mutation turns it on, our molecule turns it down. Now, what we've seen to date in the phase three were actually remarkable results where these patients who are normally hypocalcemic have to take a ton of calcium supplements to manage their blood calcium to anywhere near the normal range as a consequence, and ends up getting dumped out in the urine, and you're basically destroying the kidney over time. So no effective therapy today. What we showed in our phase three was that 76% of patients could completely come off of their standard of care supplements, go on in calorite and normalize both blood and urine calcium at the same time. We've also seen 91% of patients have normalization of serum PTH levels. So basically, you're taking calorite, you stop taking supplements, and your disease is functionally fixed by looking at these lab values. Really exciting, exciting data. It'll be really important for the field. In terms of epi and market opportunity here, we see in the genetic databases, actually we published a paper on this recently, there's around 12,000 carriers of the ADH1 mutation in the U.S., similar number likely in Europe. We don't know how many of those we can get a correct diagnosis or how many of those are actually going to be symptomatic. What we know today is there's somewhere between 3,000 to 4,000 that are diagnosed or at least suspected to have ADH1 that are symptomatic and will be eligible for therapy. Similar to limb girdle 2i, over the next year, we're going to be deploying our field medical force to drive awareness, to get physicians using the genetic tests so that we can have confirmed diagnosis of ADH1.
And what's the most logical venue next to share the detailed data?
Could be endo or perhaps a publication early next year.
And separately, you're expanding into PTH by starting a phase three next year in 2026. Any color around the timelines to the data readout? Could it be faster than the time you took to enroll and complete ADH1, for instance?
Yeah, certainly the enrollment times could be slightly quicker there, although I would just call out that it only took us about 18 months to enroll the ADH1 study. that's a smaller patient population, and it's one where, especially when we started that study, awareness of the disease versus just idiopathic hypoparathyroidism as a kind of lumping indication was lower than it is today. But yeah, there's something like four or five times as many patients with chronic hypoparabroadly versus ADH1, so we do expect that could roll quicker. Need to get some regulatory feedback from the agency on the phase three design, and then as soon as we can we'll be kicking off the phase three um so we see adh1 is a clear billion dollar plus opportunity where no competition or any coming behind us uh and there's never been any effective therapy to date so first best in class we should own that market and then i think um chronic hypopera is is somewhere between a three to five billion dollar or maybe even larger global opportunity um it will be more complicated because there's competition but i think there's room for multiple segments and multiple winners. Okay. And then that last couple of minutes,
achondroplasia, again, data early 2026. Talk about why you think this compound could be differentiated versus competitors. Yeah. So I would say it's differentiated
in two ways. One, it's the only agent out there that treats the disease directly at the source. so it targets FGFR3 directly. The proof therapies that are ahead of us are targeting CNP, which is kind of downstream. So we're hitting the genetic driver directly, and our goal is just to normalize the aberrant signaling that causes the condition. We've seen in phase two that when you normalize FGFR3 signaling in these children, you can have a profound impact on height, so an increase in their height velocity that is highly meaningful to these children and their families, as well as some early, but I think exciting data on proportionality, which would be a game changer if we could replicate that in a larger study. So in terms of what we're looking for in the phase three, I think, sorry, I forgot to mention, the other differentiating factor that is super critical here in this category is it's an oral small molecule that's given once a day. So this a condition where you're going to be treating children from infancy through closure of their growth plates when they're somewhere between 15 and 18 years old. Therapies ahead of us are injectables, either daily injection or weekly injection. We know that that is a significant burden for these families because we spend a lot of time with them and they tell us. And it's a significant barrier to initiating therapy and it is also a reason for discontinuation. So I think that is going to be game-changing if we can come to market with an orable therapy. So what we're looking for in the phase three is essentially on the efficacy side, we would like to see primary endpoint data. So the primary endpoint is change from baseline AHV versus placebo. We'd like to see that in line with what's been shown from the CNPs to date. If we can provide that with a manageable safety profile, meaning safety consistent with phase two to date, which is little but some low-grade hyperphosphatemia or phosphorus elevation and no other problematic FGFR-related issues like ocular tox, if we can show that, you know, with an oral ROA, we think that has potential to really lead this market.
And then proportionality, cherry on top kind of thing.
I wouldn't expect that in the top-line readout or the 12-month endpoint. I think it's a bit early. It might take longer for that to evolve.
Thank you so much for the update. Thank you, everyone, for listening. That's all the time we have.
Thanks, Andy.