Ultragenyx at BofA Conference: Strategic Moves in Rare Diseases

On Tuesday, 13 May 2025, Ultragenyx (NASDAQ:RARE) presented at the BofA Securities 2025 Healthcare Conference, outlining its strategic direction with both optimism and caution. The company aims for GAAP profitability by 2027, driven by its robust pipeline and commercial programs. However, potential regulatory challenges and competition remain on the horizon.

Key Takeaways

• Ultragenyx targets GAAP profitability by 2027 through its four commercial programs.
• The company is advancing clinical trials for Osteogenesis Imperfecta and Angelman syndrome, with commercialization expected in 2026 and data in 2025, respectively.
• Revenue for 2025 is projected to range between $640 million and $670 million.
• Pricing strategies for new treatments, such as Sanfilippo syndrome, are set between $2 million and $4 million.
• The company is focused on global manufacturing and FDA interactions remain on schedule.

Financial Results

• Projected 2025 revenue: $640 million to $670 million.
• Full-year GAAP profitability anticipated in 2027.
• Ex-US sales, especially in Latin America with Crysvita, are expanding.

Operational Updates

• FDA interactions for the Sanfilippo program are progressing on track.
• Global manufacturing capabilities include a US-based gene therapy facility.

• Ongoing clinical trials:

- Osteogenesis Imperfecta: Phase 3 interim analysis midyear, final analysis by year-end.

- Sanfilippo syndrome: BLA under review, PDUFA date set for August 18.

- Gene therapy: BLA filing expected midyear.

- Wilson disease: Phase 3 enrollment by year-end.

Future Outlook

• Strategic focus on expanding existing commercial programs and launching pipeline assets.
• Cetrusumab for OI expected to commercialize in 2026, with a faster ramp-up anticipated due to established physician relationships.
• Monitoring potential impacts from tariffs, executive orders, and market competition.

Q&A Highlights

• Uncertainty surrounds the impact of the "most favored nation" executive order on pricing.
• The company sees room for multiple players in the Angelman market if data is compelling.
• Interim analyses for OI are driven by patient needs and a desire for expedited market entry.

For further details, readers are encouraged to refer to the full transcript.

Full transcript - BofA Securities 2025 Healthcare Conference:

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: good morning. Welcome to the first session of the Bank of America Healthcare Conference. I’m Tazeen Ahmad, I’m one of the senior Smith Biotech Analysts here at the firm. It’s my pleasure to have with me our first presenting company, it’s Ultragenyx. Presenting for Ultragenyx this morning is CFO Howard Horn.

Howard, thanks for making the trip down from San Francisco this morning.

Howard Horn, CFO, Ultragenyx: Thank you for having me.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: And I’ll also just mention that Josh Higa is in the audience as well from IR in case anybody has any questions. So Howard, maybe we can just do a quick general overview of the company and we can then move to I never thought I’d say this, but the macro factors affecting SMID biotech these days.

Howard Horn, CFO, Ultragenyx: Alright. Yeah. So starting off, Ultragenyx is what we think of as a next generation rare disease company, and we’re in a unique situation where we’re headed towards full year GAAP profitability in 2027. That’s driven by growth of our current four commercial programs, and we also have three upcoming near term launches that are gonna transform the company. Those launches are for 01/2011, ’4 zero ’1, ’1 hundred ’40 ’3.

We’ll talk about those, I know, today. Each of them has a PRV associated with them, and that’s, you know, even before we talk about the Angelman program. So there’s a lot of exciting stuff coming up for us. Specifically for 2025, I’ll highlight five things. I’ll do it quickly.

But top line this year, six forty to six seventy million in total. We will have data from our OI program this year. We are currently under review for our 111 gene therapy program with a PDUFA date of August 18. We are about to file midyear our four zero one gene therapy program, so BLA will go in midyear. And we are enrolling our one zero two phase three trial that’ll happen before the end of the year, and we should update it next year.

So a lot of activity in the back half of this year.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: Okay. So we’ll get to those catalysts in a second, but I wanted to take a couple of minutes to talk about items that you might have already addressed most of these on previous interactions with investors and also on your recent earnings call. But maybe let’s start on the most recent update was the executive order that was announced yesterday as it relates to most favored nation. Can you just talk about how it could potentially impact Rare?

Howard Horn, CFO, Ultragenyx: Yeah. That that is actually the toughest of the new sort of things coming out of DC. Candidly, I don’t know. I don’t know that I know enough. And by the way, I really appreciated the note you put out yesterday.

I think details are still to be revealed. I think the markets spoke yesterday by saying they didn’t think it was gonna be a big impact for pharma and biotech, but my candid answer is I don’t know yet.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: So can you talk about, for your current business for your commercial products, what is the payer mix?

Howard Horn, CFO, Ultragenyx: We haven’t really stated that yet, so can’t give you an answer.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: Yeah. So I think as time moves on, people will probably probe you a little bit about that just because of the Medicaid component. And can you just also remind us about what your ex US sales percentage?

Howard Horn, CFO, Ultragenyx: Yeah. So ex US sales is a growing part, Latin America in particular with Crysvita for us. But we, you know, we we have a healthy growing business across all of our regions.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: And so if something needed to be done, would you make a choice between choosing to be in a particular country versus wanting to maintain price or maybe I’m going too far ahead? You know, in general, some companies have said that they have a very narrow price range for their products. Would in the world of rare disease, that be a safe assumption to make about Ultragenyx as well?

Howard Horn, CFO, Ultragenyx: Yeah. Unfortunately, it’s too soon to to think that all through. I think I’d I’d pivot back to our mission as a company, which is to help rare disease patients, you know, globally. And that’s, you know, that’s where we know we are today.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: Okay. And then can you talk about interactions with FDA? How have they been going over the last, I guess, now two months? Any changes that you’ve noticed?

Howard Horn, CFO, Ultragenyx: Yeah. So this specifically related to our one eleven Sanfilippo program. We don’t usually comment on details of regulatory interactions, but given all the changes at the agency we did and have and what we’ve shared is that the one eleven process is ongoing exactly the way you would have laid it out or it has been laid out. So we had our mid cycle review on time, and we know that the inspections of our manufacturing facilities and our clinical sites have been scheduled and ongoing on time. So everything is on track.

I think in a in a more macro statement, we have a long history of working well with the agency, and I do expect that to continue.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: Okay. And then lastly, any impact that you’re expecting from general tariffs?

Howard Horn, CFO, Ultragenyx: Yeah. That’s another interesting one. Still clearly evolving. We, like other companies, are running scenarios, analyzing, trying to learn, you know, what might be the final decisions there. Ultimately, we do have a global supply chain, like others.

But when when we look at the current landscape, I really don’t think there’s a material impact on any of our programs, including Crysvita.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: And have you been public about where your manufacturing takes place?

Howard Horn, CFO, Ultragenyx: We have manufacturing all over, but I would note that we have a US factory for gene therapy that’s right outside of Boston. Okay.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: Alright. So we’ll follow-up as other events happen to ask those questions perhaps again next quarter. So maybe let’s move on to osteogenesis. So for OI, can you just give us a quick overview of the program? And people have been talking about the first interim that happened, the second one that’s coming and the potential for a third and last.

And so for those who may not be as familiar, can you just explain to us what those mean?

Howard Horn, CFO, Ultragenyx: Yeah. So so Elia is a bone disease, where, you know, you can break bones even rolling over in bed. So it’s a pretty it can be pretty debilitating. We are in our phase three trial now. And I think what you’re mentioning is we had a couple of interim analysis in that phase three trial, one of which was at the cusp of the year, interim analysis one.

We’re heading into interim analysis two midyear, and then there’s a final analysis if we need to go there at the end of the year. In general, whether it’s at the midterm or the IA two or the final analysis, we feel really great about the program. And regardless of what time point we get the data, I don’t think that impacts the ultimate commercial opportunity. But we’re often asked, you know, how do you feel about IA two? What, you know, what is at the core of why you think it could work?

Mhmm. And I think, basically, looking back at the phase two data, we’re very encouraged by that. That’s fundamentally why we’re excited. And if you think about IA one, that was at a time point where we had seven months of treatment at minimum for all patients. And so if you think about needing a few months for bone mineral density to improve and for the drug to really have an effect, there’s only a couple of months of therapeutic window there where you can see a separation between the placebo arm and the active arm.

And so if you fast forward to this, you know, this midyear time point for IA two, that’s twelve months minimum for all patients. So you’ve added at least another five months to that handful of months before, so more than a % of time. So that gives us some hope that we could hit at IA two.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: So what was the reasoning behind having you know, why not wait till the end to do the final analysis? What made the company confident that interim reads at these specific time points? Because this is a matter of time. Right? You collect more events, and that increases the chances of the study working.

But why did the interims make sense?

Howard Horn, CFO, Ultragenyx: Well, our our phase two data gave us a hint that it could make sense. Right? I one, the initial one would have been an early hit, but not impossible. IA two is sort of more consistent with maybe the data we’ve seen in phase two and IA three or sorry, the final analysis, we’d get there. But ultimately, the reason we chose to do it is kind of twofold.

One is for patients. Right? The earlier we can get them off of placebo, the better. And then also a financial one where months do matter for net present value. So our thought was if we could get it to the market sooner, that would be better.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: So let’s talk about the second interim. Emil had given some color about potential timing as it relates to data lock. Can you just give us a summary of that?

Howard Horn, CFO, Ultragenyx: Yeah. I’ll I’ll go with the midyear answer. There’s not there’s not much more I can sharpen that point.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: But has data lock happened?

Howard Horn, CFO, Ultragenyx: So we did say it at the we we did say at the quarterly call that data lock has happened

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: Yeah.

Howard Horn, CFO, Ultragenyx: And that it’ll take some number of months to get that data ready. And then we will you know, whether we hit or not, we will tell the street.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: And so, I guess in terms of the the study itself, if you do have to go to a third and final read, what would give the company confidence that that would be the sufficient amount of time that would be needed? Because people have been trying to debate what potential issues there could be with study design, study design in general. But in this case, for this study, what could make the second interim not have collected enough events? And then what gives you confidence that the third and final would be sufficient in terms of time?

Howard Horn, CFO, Ultragenyx: Yeah. So fundamentally, we’re looking for these two lines to separate. Right? And I think should we not hit at IA two, I think the fundamental my hypothesis hypothesis is that the fundamental reasoning would be that you just you had some more variability in the placebo group, and they didn’t have enough fractures to show separation. Recall the p value for interim analysis two is point o one, So it’s a reasonably stringent threshold, but what we’ve seen in data in the past, I don’t think it’s an irrational one to choose.

But then in the final analysis, the, I think the p value is point o four or thereabouts. So that’s a that’s a much, you know, better bar, if you will, to hit. And I think with those additional months, the chances that those lines separate is good.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: So what is the market opportunity for OI?

Howard Horn, CFO, Ultragenyx: Yeah. So we think it’s bigger than Crysvita. And so in particular, maybe to frame it, 60,000 patients in the world, about a quarter of those in The US. So 60,000 patients for OI, fifty thousand for XLH, and maybe 15,000 in The US versus 12,000 for XLH. And what we’re hearing from physicians is that they have fifty percent more OI patients in their clinics.

These are the KOLs. Now that doesn’t mean that there’s fifty percent more patients. It just means that with the severity of the disease, these patients are coming into the the key centers. So I think that’s good news for us because, one, we already know these docs from our XLH experience, and, two, that makes those patients more accessible. So I think together, that could mean that we have a faster ramp up than we saw with Crysvita.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: So you talk about the total number of patients, but do you think that all of them would be needing treatment beyond what they have now? Can you just remind us of what patients have available to them now?

Howard Horn, CFO, Ultragenyx: Yeah, so patients are often on bisphosphonates, although that’s not, you know, an approved medicine here in The US and for most countries around the world. And our impression is that that’s not satisfactory for them. And so they are whether they’re type one, type three, type four, I think the majority of those patients, certainly all the threes and fours, and at least most of the type ones would be looking for treatment.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: Yeah. And when we go to the the type of patients, so there seems to be a heterogeneity in terms of number of fractures in a way a patient can have. And so how did you account for that during enrollment of the study? I think a question that we’ve gotten is how do you get comfortable with what the baseline fracture rate of patients is when they’re enrolled when they were enrolled into the study?

Howard Horn, CFO, Ultragenyx: Yeah. The sorry. Forgive me. Just reminding myself of the actual numbers. So the phase two had about seventy percent type ones, and the phase three is about fifty fifty percent type ones, versus threes and fours.

And that threes and fours have typically a higher they’re more severe to be disease and higher fracture rate. So our theory is that there may be a higher fracture rate in the phase three, but the inclusion criteria were the same between the phase two and three. And our stratification of the phase three was actually done on age and fractures, so not by type, but it’s pretty correlated, as I was just mentioning, between number of fractures that you’d see at baseline by type.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: And is there any concern that due to behavioral changes that naturally happen during a clinical trial, people are much more diligent in some cases? Is there any kind of concern that maybe their behavior pattern would be such that you don’t collect enough fractures in that time period?

Howard Horn, CFO, Ultragenyx: I smile because we’ve heard both sides of that, that patients sometimes are less active sometimes in a trial. But in this case, some of these patients are very inactive. So even going to the clinic is more activity than they’re used to, and that could risk fractures. Also, if patients are feeling better and they get more active, and we’ve heard of examples of this, they might receive fractures that way. But ultimately, what we’ve seen is these fragility fractures, which are the ones we’re trying to stop or change, they really fall away.

The phase two data shows that they fall away in the first few months after treatment.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: Now what about the safety profile of the drug? Can you talk us through cetrusumab’s profile thus far?

Howard Horn, CFO, Ultragenyx: Yeah. I think I think I’d say it’s a classic antibody. Know, antibodies are are safe and pretty safe, at least. And I think ultimately that’s encouraging for us because we wanna use it as a chronic therapy. We wanna have in home dosing as an option too.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: And how would that work? So let’s say that the study is positive. When you say in in home option as well, would that be right at the beginning, or would that have to evolve over time?

Howard Horn, CFO, Ultragenyx: So this is something we have experience with with Crysvita. Mhmm. And, you know, we we would try to implement it as soon as we could.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: Okay. So assuming that, let’s say, either this coming interim or the final interim is positive, maybe let’s just think about timing of the final interim. Would that happen at year end or after?

Howard Horn, CFO, Ultragenyx: Yeah. We’ve said in the fourth quarter, so before year end.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: And would so if the results happen in the fourth quarter, would would we be made known what the results are in the fourth quarter? Would there be a period like you just described about data log happening and, you know, waiting to reveal that data until you have a little bit more details.

Howard Horn, CFO, Ultragenyx: Yeah. I think we haven’t set that expectation yet. Maybe the one that is more near term is for phase two or sorry, for interim analysis too. Whether we hit or not, we will tell the street. Sure.

And, you know, the there’s two trials running in parallel here. Right? So there’s Orbit and there’s COSMIC. We will, you know, we will take a look at that second trial if we hit on the first, so we’d spend some alpha and look at it. If we don’t hit on that orbit, we would wait on COSMIC and look at those together at the next time point.

But this summer is pretty much all we’ve talked about in terms of exact timing.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: Okay. So can you potentially give us a range of when this could become commercial just based on success at the second interim versus success a few months later at the final read?

Howard Horn, CFO, Ultragenyx: Yeah. We’d we’d be commercializing in 2026.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: And under either scenario? Ideally, yes. Right. So think Aimle has been talking about confidence that the drug is active, which we would agree with, and that the timing of whether it stopped at the second interim or third interim is not potentially as meaningful as some investors might think. Is it because of the reason you described?

Because doesn’t it really change the time when the product would become commercial?

Howard Horn, CFO, Ultragenyx: Well, I think there will be a series of months in between the two, but I think ultimately maybe Amos’ comment is related to the fact that we think the strength of the data and how profound it is in terms of impacting fractures Yeah. Doesn’t really depend on whether it’s interim analysis two or the final analysis.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: And then can you talked about the benefits that that have been seen on seen with the effect on bone mineral density. Why is that important?

Howard Horn, CFO, Ultragenyx: Yeah. So I think that what we saw in the phase two was pretty profound. And I think the question from the community was, would that translate over to reduced fractures? And that’s precisely what we saw. Right?

We saw fragility fractures waning after the first few months. That’s what we’re hoping to see in phase three. And I think you know, what we’re trying to prevent is the fragility fractures. We’re trying to prevent the pain that these patients experience, morphometric vertebral fractures, which can often, you know, change the skeleton forever. So these are the things we’re trying to avoid.

So transformative therapy is the goal.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: This is also thinking ahead, but what would a label look like? Would it would it be the goal for every OI patient to be eligible to take this therapy?

Howard Horn, CFO, Ultragenyx: I think that’s our hope.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: And how is the competitive landscape shaping?

Howard Horn, CFO, Ultragenyx: So right now, bisphosphonates, we’ve talked about. I think, you know, with the with the second trial we’re running, we’re hopefully gonna show that we are substantially better, and the space is one that we can, you know, be the leading therapy

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: Based on your experience with Crysvita, do you have a sense on how many of the doctors you as a company already have had touch points with versus ones that you would have to build out relationships with?

Howard Horn, CFO, Ultragenyx: Yeah. We think it’s about a 90% overlap between XLH Docs and Docs for OI. So that’s really good news for us. We we have a very good relationship with these folks.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: So then in terms of the investment that would need to be made for the commercial launch of this product, Is it going to be meaningful?

Howard Horn, CFO, Ultragenyx: Yeah. So it’s very rare. So I was a former athlete. It’s very rare that you get to run this play the second time around, but with all the hindsight and knowledge from the first time around. So we feel we feel like that’s a privilege.

We do still have some of our Crysvita sales force and some of our patient find organization in place. So the build on that is sort of you know, it’s it’s modest. Mhmm. So there’s not a lot to be invested there.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: Okay. And then can you talk about your partner, Moreau, the type of interactions that you have? Maybe just describe them to us and and let us know the the nature of the relationship, Moreau, has European rights. And so maybe can you talk about why they were the right partner for this particular program?

Howard Horn, CFO, Ultragenyx: Yeah. So they they are a terrific partner. We the relationship works as follows, that we run global clinical and global regulatory. Mhmm. They run commercial in Europe, and they pay to us a mid teens fixed royalty.

And then we would run commercial everywhere else around the globe and pay them a tiered mid teens royalty plus the milestones.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: And how do you overall think about the European market opportunity versus the The US opportunity number of patients wise?

Howard Horn, CFO, Ultragenyx: It is certainly it’s certainly smaller. I think it’s a good market. We’ve characterized it as one that, you know, we we have a base of operations there ourselves. Mhmm. So I think I think it will be, of our markets, maybe not as fast growing as The US and Latin America, but certainly important contributor.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: And so do you think that that cetrusumab could be the the biggest product for the company over the next several years?

Howard Horn, CFO, Ultragenyx: For sure.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: Okay. So with that in mind, let’s move on to another indication, another program that has gotten a lot of attention, and that’s for Angelman. Can you just remind us on where you are in development there?

Howard Horn, CFO, Ultragenyx: Yeah. So we’re enrolling our phase three now, and we have said that we expect to complete that enrollment this year and have data next.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: And how is that how are you expecting enrollment to go? Because you are competing with another company. Yeah.

Howard Horn, CFO, Ultragenyx: So we have sites open globally, so North America, Asia, Europe. And to date, we’re we’re on our plan. We feel really good about it. We haven’t heard about a lot of competition for enrollment, so it’s not as though people are waiting for other trials. So it’s it’s been going to plan.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: Are these enrollment centers concentrated?

Howard Horn, CFO, Ultragenyx: No. They’re they’re all around.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: And can you just talk to us about, geographically, where your sites are located?

Howard Horn, CFO, Ultragenyx: Yeah. So US, Canada, multiple places in Europe, Japan.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: And is the profile of the Angelman patient any different geography wise?

Howard Horn, CFO, Ultragenyx: I don’t think so. Okay.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: Now can you talk to the endpoint that you’ve chosen for phase three? Because at the time that Bayley for cognition was something that people were trying to understand a little bit better.

Howard Horn, CFO, Ultragenyx: Yeah. So we, as you know, we had maybe five to choose from. We thought of cognition, or I think of it, as sort of a predicate to some other developmental areas. Also, saw a good movement on it with the Bayley score in our phase two. So we for those reasons, we chose it as our primary.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: And what would be is there I guess, what would be the concern of using that versus any of the others that you looked at? And and, you know, the flip side is what gave you confidence that that is the right one to look at?

Howard Horn, CFO, Ultragenyx: Yeah. So we had choices, as I mentioned. And in fact, we have a a key secondary endpoint called the MDRI Yep. Which takes five of these things and puts them together Yeah. Which I guess we’ll talk about in a minute perhaps.

But ultimately, as I’ve mentioned, cognition rose to the top because we did see significant movement in it, and and the agency preferred that we choose one.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: Is there any subset subjectivity involved in these endpoints?

Howard Horn, CFO, Ultragenyx: To some degree. But when you when you think about natural history and you think about the possibility of a placebo effect, you don’t you don’t see much more than maybe a point of movement in that regard. And we’re showing, you know, the minimal difference that we think is important is five points, and we’re showing, you know, increasing trajectory well beyond that.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: So you talked a second ago about this NDRI scale. Can you talk about the components that go into it? And, you know, how do doctors think about that versus Bayley?

Howard Horn, CFO, Ultragenyx: Yeah. So this is a very heterogeneous disease. Right? And so some patients will have issues with sleep or behavior or cognition or gross motor. I think I hit them all, but we have five of them in there.

And the idea is that the NDRI lets you see all of that together at once and to see where you’re making gains and to not have to just choose one of them. And what we’ve heard is that it’s a simplifying way to take all this complex data and all the heterogeneity and look at it in one place. Mhmm. And so from physicians and from patients, they’ve said it’s been a very helpful tool to deal with all of that information.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: And so what is your expectation for when the study would read out?

Howard Horn, CFO, Ultragenyx: We have said back half of next year, I think, because it’s a forty eight week study and if we were rolling in the back half of this year.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: And so what would be good data for that study?

Howard Horn, CFO, Ultragenyx: I think that we keep showing that we’ve improved on these five domains.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: So simply just being stat sig on on your primary as well as looking at at MDRIs?

Howard Horn, CFO, Ultragenyx: Yeah. I I think, you know, we don’t need MDRI to hit to to to win here, but I think that’ll be a helpful extra piece of information because ultimately, your primary matters. But when you’re prescribing, I think they wanna see the totality of the data, and I think that’s where MBRI helps with that story for patients and docs.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: And how big is how big is this opportunity relative to OI?

Howard Horn, CFO, Ultragenyx: It is it is about the same size in terms of patients. Josh, remind me of the total number we’ve talked about. Yeah. About 60.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: And in terms of the investment you would have to make for Angelman, would that be something that you would need to do from scratch, or would you be able to leverage what you already have?

Howard Horn, CFO, Ultragenyx: That would be that would be new.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: And how big are you thinking an investment you would need for that

Howard Horn, CFO, Ultragenyx: would be? Haven’t gotten there yet. But in rare diseases, we can typically be very efficient.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: Okay. So between these two products, you know, this could potentially, as I said as you said a second ago, OY could be the company’s biggest product. But if Angelman is also successful, how are you thinking about, you know, the need for future investment in r and d as well as how to manage expenses for general SG and A launches?

Howard Horn, CFO, Ultragenyx: Yeah. So I guess I started off by talking about our pathway to profitability. We’re committed to that full year profitability, GAAP profitability in 2027. And, you know, we we don’t need any we don’t need all of these for your near term launches to work to have that be our case. I think what that will tell us, though, is the slope of our our EPS and, therefore, how much we would have to reinvest.

We have we have multiple I and d ready assets internally, so we’re excited to bring those forward when we can. Also, company was built sort of bringing things in from the outside. That’s part of our DNA. Yeah. And we would look to be able to do that too.

But I think, ultimately, the amount of investment we would put after this generation of programs will depend on sort of the the slope of their success.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: Yeah. So I guess one difference between OI and Angelman might be that you might have OI, you know, to yourself, but with Angelman, you might have competition. And do you think that the Angelman market, just based on the numbers you gave us just now, is big enough to support two players?

Howard Horn, CFO, Ultragenyx: So it’s certainly big enough to support one really good one. Mhmm. But whether whether it can support more, I think, really, is gonna depend on what the data looks like. And so, could it support you? Too sure.

But I think how that dynamic will play out will be really told by the data.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: Okay. And then, maybe let’s move on to Wilson disease. Mhmm. Can you talk to us about where that’s in development currently?

Howard Horn, CFO, Ultragenyx: Yeah. So we’re enrolling a fourth cohort for Wilson, and we expect to have that enrolled before the end of the year, but it’s in phase two in development.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: And same question as I’ve been asking, how big is this indication?

Howard Horn, CFO, Ultragenyx: So it’s also about fifty thousand patients globally, maybe a quarter of those in The US.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: And, you know, I guess, what is the current protocol in terms of standard of care for these patients?

Howard Horn, CFO, Ultragenyx: So it’s chelators and zinc, and our aspiration is to be able to have the majority of patients come off.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: How how successful are chelators and zinc with these patients?

Howard Horn, CFO, Ultragenyx: I I think not so successful. I think when you look at the livers of patients who are deemed to be under control, there’s still some trouble there.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: We’ve talked about this question about tech transfer in the past, but just can you just remind us for this particular program, what is the plan for that?

Howard Horn, CFO, Ultragenyx: Yeah. So this one is in our manufacturing plant in Bedford already. I think the tech transfer story was around 04/2001 where we had that external lead initially and then took a little bit of a time delay. Yeah. But now that it’s in our plant, I think that’s a much better situation.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: So I guess for anything that would come out of that that facility going forward, it would be pretty much leveraging off of what you’ve already done That’s the plan. In terms of tech transfer. Yeah. Okay. One question that I’ve gotten is what kind of cogs to expect on these types of products?

Howard Horn, CFO, Ultragenyx: Yeah. We have not been super specific about that, but I think the advantage of having our own plant and the methodology that we use in the plant means that we can reduce these COGS much lower than you’d probably expect. And so I think there’s a healthy margin to be had.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: Okay. And then just in terms of pricing, Emil has always talked about responsibly pricing products, and that’s part of the makeup of Ultragenyx as a company. Some have asked me about when you have a product where current standard of care can involve things that are clearly inferior but also much cheaper. Does that, in your mind, raise the bar of what needs to be shown in order for a product to be commercially successful just in general, not related to any of these particular programs?

Howard Horn, CFO, Ultragenyx: Yeah. Maybe you’re thinking of like four zero one where you have very cheap alternatives with cornstarch. I ultimately, like using that one as an example, I think the value proposition there is that that disease has such urgency and it has life and death implications that patients would see the value in the gene therapy, the one time gene therapy. It’s been described to me that having that disease is like living potentially with a gun to your head that could go off if you miss dose of your cornstarch slurry. And while it is inexpensive, I think it is a challenging way to exist.

And so our hope is that the value of the gene therapy would bring, you know, priced responsibly would be would be well well better than what they’re currently being served by.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: Okay. And then the last product I wanted to talk to you about really quickly is the market opportunity for Sanfilippo syndrome. Can you just tell us what your market data shows on that and what the competitive landscape is?

Howard Horn, CFO, Ultragenyx: Sure. Three thousand to five thousand patients globally, about a quarter of those in The US. No approved therapies today. And our thought is that with that being a very lethal disease, that the price point could be in the 2 to 4,000,000 range.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: And how are you thinking about competitive landscape as it relates to potentially Denali?

Howard Horn, CFO, Ultragenyx: We’re tracking it, but we we know we’re ahead, and we’re excited to get this thing to the market ideally before the end of the year.

Tazeen Ahmad, Senior Biotech Analyst, Bank of America: Okay. Perfect. With that, we’re out of time. So thanks for joining us this morning. Thanks everybody for sitting in.

Howard Horn, CFO, Ultragenyx: Thank you. Great to start the day with you.

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