BioCryst Pharmaceuticals, Inc. (BCRX) Earnings Call Transcript & Summary
January 12, 2026
Earnings Call Speaker Segments
Jessica Fye
AnalystsGreat. Welcome. Good morning, everyone. My name is Jess Fye. I'm a biotech analyst at JPMorgan. We're so glad to have you here at the 44th Annual JPMorgan Healthcare Conference. We're continuing the conference with BioCryst. And first, you're going to hear a presentation from the management team, then we're going to go into some Q&A. [Operator Instructions] But with that, let me pass it over to the company's CEO, Charlie Gayer.
Charles Gayer
ExecutivesThank you. Good morning. Thanks, Jess. Thanks to the JPMorgan team for inviting us here this year. I'm here with Bob Ghias, our CFO; and Dr. Bill Sheridan, our Chief Development Officer. And this is a really exciting time to be taking over this year as CEO of BioCryst because if you've been following BioCryst over the years, I think you'll see that it's not the same BioCryst that you've seen before. We have some really exciting things going on. Before I get started, my team and I will be making some forward-looking statements. Those statements have risks. You can read about them on Slide 2 or our website. And then we also will be making -- using some non-GAAP financial measures in this as we talk about last year's preliminary results and guidance for this year. 2025 was a really transformative year for BioCryst. We accomplished a lot. First and foremost, ORLADEYO growth 2025 we saw the most new patient prescriptions in the United States since the first year of the launch. This was in year 5, which you don't normally see in a rare disease launch. We made great progress on paid rate improvement in the United States for ORLADEYO, ending Q1 at 84% and the year at 81%, and this compares to our long-term goal that I'll touch on more later of 85%. We sold our European business. The business was going great, but as one product in Europe, it was not profitable for us. And we sold it to Neopharmed Gentili for $250 million in cash upfront, and that helped us not only streamline our business, but pivot directly into a business development strategy where we turned around and got into the proposed acquisition of Astria Therapeutics that we expect to close in the next couple of weeks. And that gives us another late-stage rare disease HAE asset in Navenibart that we're really excited about. We also achieved profitability for the first time in BioCryst history. And now that we've done that, we don't intend to go back. We expect to be profitable from here on out. And then finally, right at the end of the year in December, we got approval for ORLADEYO for kids, allowing us to bring oral preventative therapy for HAE kids down to as young as age 2, which we think will be transformative for the HAE community. So a really big year for BioCryst. Preliminary results for the year, $601 million in ORLADEYO revenue, beating our latest guidance of $590 million to $600 million that we announced after Q3. And what's really important here for everyone to look at is the next line, $563 million in our ongoing business. So this excludes the European business. So this is the baseline to measure our progress going forward, ended the year with great cash position, nearly $340 million. And that sets us up for 2026, where we are anticipating ORLADEYO revenue of $625 million to $645 million. Total revenue, once you include RAPIVAB of 635 to 660. And then on our base business prior to the Astria acquisition, because of the removal of our European business and other efficiencies, our base operating expenses on a non-GAAP basis will be $380 million to $390 million and then adding $70 million to $80 million once we closed the Austria deal to continue the operations there, completing the -- moving towards completion of the Navenibart clinical trial. So a strong year for prescriptions, 1,600 patients on therapy in the U.S. despite the competition in the HAE space. So this sets us up to have 3 very strong pillars for our growth strategy. Starting with ORLADEYO, a commercial product that is on track for $1 billion at peak that we expect to hit in 2029. We're getting towards a greater than 80% contribution margin on that business. Once we add Navenibart, this is going to be north of 90% contribution margin on the commercialization. And then very importantly, a really long runway with ORLADEYO. We have composition of matter protection out to May of 2040. So as we hit peak in 2029, we have over a decade of significant cash generation on that business. BioCryst in the past was all about our internal R&D, and we'll continue to do that in a very disciplined way with allocation of capital there. But we have some programs that we're really excited about, led by our Netherton syndrome program that I'll talk a little bit more about. This is a high need rare disease, and we have the potential to be the first truly transformative drug in a condition where there is no therapy for these patients right now. And then external opportunities with all the cash generation from ORLADEYO, eventually from Navenibart, we will continue to look for late-stage rare disease assets, where we can plug that into our commercialization engine and really leverage the infrastructure that we built and the skills that we have in commercialization to drive future value in rare disease. So again, this starts with ORLADEYO, a molecule that we developed ourselves was first approved in 2020 is now entering its sixth year of launch and still growing. As you can see from the right side of the slide, about 50% of the patients who have tried ORLADEYO in the U.S. are still on therapy after 5 years, and the growth continues. And the reason they're still on therapy is because ORLADEYO is a drug that either works fantastically well for you or it doesn't, in which case you move on. And we knew this going back to our Phase III pivotal trial, APeX-2, when we looked at the long-term 2-year data from this trial. And the patients who stayed on ORLADEYO 150 milligrams had a 91% reduction in attacks versus their baseline. And so in the real world, that's what physicians and patients are discovering is you should expect great efficacy from ORLADEYO. And if you don't get it, that's okay. You have many other options that you can try. And what this translates to is when a patient starts, patients start on ORLADEYO, 60% of them make it to a year. And then after a year because they're doing well, very few drop off therapy. So ORLADEYO is not only the most differentiated product as the only oral prophylaxis product in the market. It's also highly competitive on an efficacy perspective. And so what this sets up for the next several years in ORLADEYO is we believe 15% roughly year-over-year CAGR in revenue growth out to 2029, where we expect to hit $1 billion. And if you look at the chart on the right, this is very achievable. All we need to do to get -- we've got 1,600 -- a little more than 1,600 patients today, at net average of 150 net patients per year over 4 years, gets us to where in the United States gets us to where we need to go. A little bit of improvement in our paid rate going from 81% at the end of last year to 85%, and we continue to make progress year-over-year in this, modest price increases and then contribution from our businesses in Japan, Canada, and the rest of the world. This is a very achievable path to $1 billion. And then that sets up what we expect to be coming in a couple of weeks with the acquisition of Astria. We're really pleased with this deal. We were purchasing Astria for $700 million in total enterprise value. And it starts with the strategic fit. We have shown that we're really good at commercializing differentiated products in the HAE space. And this Navenibart fits right into that strategy, right into our core area of expertise. Navenibart is a very differentiated product. It has the potential to be in every 3 to 6-month injectable dose. And when patients and doctors hear about that 3 to 6-month profile, they get excited. This is something that makes them think about switching from existing injectable products. And it's very simple for them to understand because kallikrein inhibition is a mechanism that they already know well. So you put these 2 things together in 1 team. As ORLADEYO hits peak, we won't need to spend as much marketing dollars in ORLADEYO. But it's the exact same team that we'll be commercializing. So we don't need more people to commercialize Navenibart. And so that just strengthens our cash flow position and sets up the opportunity to do more in the rare disease space. So what's the opportunity for these 2 products, where you've got the only oral prophy product right now in ORLADEYO and then you're adding in a differentiated injectable product. What we see is that the U.S. HAE population on prophylaxis has been growing steadily at about 3% to 5% a year, and we expect that to continue as more patients are diagnosed. And as more patients continue to move over to prophylaxis. So ORLADEYO has at least another 2 years where it is the only oral prophy product in the market. So we'll continue to grow with ORLADEYO. And then as I explained patients are already doing well on one pill once a day, why would they switch to anything else. Then from an injectable population, there's about 5,000 patients and growing who are on some type of injectable prophy and Navenibart comes in there with the most patient-friendly -- every 3- to 6-month dosing profile. And so the majority of the opportunity there is to switch patients from other prophy. So 2 very complementary prophylaxis products that meet the needs of the market. And we're going to do this with a team that has great rare disease commercialization experience has been doing it in a very dedicated and differentiated way using a sole-source specialty pharmacy that allows us to generate evidence, evidence that we use to help our commercialization efforts on a day-to-day basis and then evidence that helps us build more into the clinical literature and teaches physicians, in this case, about ORLADEYO in the future about Navenibart and in the future about other products, perhaps like 17725. So as I said, Navenibart is highly differentiated, early proof-of-concept data from Phase Ib shows that it's got a very competitive efficacy profile regardless of whether it's the 3 or the 6-month dosing. Very importantly, it's a small injection, 3 months will be a 2-milliliter injection every 6 months, we'll be double that in an auto injectors. And in the Phase Ib, no injection site pain, which is something that a lot of patients experience on the market leader today, lanadelumab. And so as I mentioned, you put these 2 together, we're addressing both segments of the market. There is a growing preference for oral prophylaxis and we're addressing that with ORLADEYO. In fact, we're building that preference. But for those patients already doing well on an injectable, they're sometimes reluctance to switch to anything else unless the benefit is very clear to them. And that's where we think that Navenibart is going to step in and be very clear, you can go from every 2 weeks or every 4 weeks to every 3 months or every 6 months, and that's going to be the value proposition without giving up any efficacy. So you put these 2 products together in 1 portfolio, and this is what it could look like. You've got ORLADEYO growing at mid-teens revenue CAGR through 2029. And hitting peak in 2029 is we do expect additional competition. But that's right when we'll have the first full year of Navenibart revenue kicking in. So $1 billion late this decade and then Navenibart keeps us growing at double digits, mid-double -- or mid-teens revenue into the 2030s. And all of this is on a very steady base of operating expense. So we expect our OpEx over this time to grow at about 4% or 5%, mid-single digits. And what do you get for that? You get the team that's commercializing both of these products. Again, we don't need to grow it because we have the expertise. We have enough sales reps. We have the marketers. We have the marketing dollars. We just need to redeploy the focus between ORLADEYO and Navenibart. You get the G&A function that supports all of this. And you get the finance team, you get the HR team, pharmacovigilance, all of that is built into this. And then you also get our R&D. And so drugs like 17725, the cost of developing that and taking it forward is included in this chart. What is not included is the potential revenue from something like 17725. And so that becomes upside. So we're going to be really disciplined on our operating expenses, growing revenue from HAE and beyond. And then it gives us the ability with -- we see $1 billion in cash in 2029 to redeploy and look for other late-stage assets that our team can commercialize. So let's turn quickly to our pipeline. And the real thing to focus on here is BCX-17725 for Netherton syndrome. It's about to enter initial proof of concept trial in Netherton patients. We have other undisclosed targets that we're excited about, and we hope to bring forward in the future. But right now, let's focus on 17725. Netherton syndrome is a really horrible disease. You can see from the picture of the kid on this page. It's a genetic disorder that causes uncontrolled skin turnover and not only does that obviously affect the skin, but for patients, this leads to increased infections, increase of atopic conditions, asthma, food allergies. What we hear from patients is patients with Netherton spend their whole day thinking about how they're going to deal with this disease. And there are no approved therapies for this condition right now. We've done initial work showing that there are about 1,600 patients looking in claims data in the U.S. I'll tell you about some additional work that we're doing that suggests it could be even larger. So it's a classic rare disease, no treatments available right now. And our therapy is going to be replacing the missing protein and controlling KLK5 that is the uncontrolled bad actor in this case. And we have the potential to be the first targeted therapy in the first systemic targeted therapy. So as I'm sure everyone is familiar in rare disease, you often see this where the prevalence, the incidence goes up once there are therapies and it makes sense because doctors don't tend to look for things that they can't treat. And we think -- we've seen this in HAE. We have people on our team who launched Cinryze back in the day, and they thought there were 2,000 or 3,000 HAE patients. Now we know there are over 10,000 in the U.S. And we think the same thing will happen in Nethertons. We're in the middle of doing -- we do a lot of really deep analytical work. That's a part of our commercial strategy and success. And we're doing that work in Nethertons, and we're using natural language processing models right now digging into EMR data, and it's suggesting that the population could be 3,000-plus in the U.S. alone for this high need disease. So more to come on that, but we're excited about the opportunity. 17725 inhibits KLK5, which, again, because of the natural genetic defect the control of KLK5 is what's missing. That is at the top of a cascade of proteases and ultimately, cytokines that cause the skin to turnover, cause patients to have itch, cause redness and many other problems. We believe that inhibiting KLK5 really significantly, and that's what 17725 does can control this cascade and has the potential to restore normal skin turnover and all everything downstream from there. So where are we in this program right now? We're through healthy volunteers. And our healthy volunteer work showed 2 important things. Number one, this drug appears to be safe in multi doses. So that's super important. And then number two, you can see from this slide, the drug is getting to the epidermis. That's where it needs to go. So you look at the right side here, we used a phosphorescence tag on the antibody. And you can see not only does 17725 flood the dermis, which is great, but it gets into that outer layer, which is the epidermis. That's where KLK5 acts. That's where the drug needs to be. So this doesn't prove everything, but it was an important derisking of the program, and we'll see how the drug behaves in patients. So we're done with -- we've got a Phase I study that studied healthy volunteers and now it's moving into patients. We're finished with a healthy volunteers. We're moving into both Parts 3 and 4 of this Part 3 is expected to be 1 to 3 patients and it's a 1-month dosing period, 3 doses over 1 month. And then Part 4 is dosing for 3 months, every 2 weeks in up to a dozen patients. That's what you really want to focus on. We are highly confident, by the end of this year, we will have up to about a dozen patients who have gone through 3 months with follow-up, and we'll be looking to see not only the safety and exposure. Is it getting to the skin, but importantly, is it changing the clinical picture? Is it improving skin turnover? Is itch going away for patients. And that's the information we're looking for to design our Phase III program, go to the regulators next year and begin a pivotal study for this high need disease. So upcoming milestones, key milestones in about 2 weeks from today, we should be through the Astria acquisition. All that remains is a shareholder vote from the Astria shareholders. Then by the end of next year, and it could be sooner, depending on the size of effect that we see, 17725 proof-of-concept in Nethertons. And then early next year, Netherton's top line -- sorry, Navenibart topline result for HAE, which is obviously a critical stage towards moving towards filing and approval. This is on top of quarterly revenue growth in both ages 12 and up and the pediatric indication and more and more cash generation for the company. So again, I'm excited for what we have going on at BioCryst right now. It's going to be a big year, and we have a lot of opportunity in front of us. So with that, I will turn it over to Jess and...
Jessica Fye
AnalystsGreat. Thank you. And as a reminder, if you have a question in the room, just raise your hand. And if you have a question on the webcast, you can send me them on the iPad here. So great, so we've had some competitive entrants in HAE prophylaxis recently. Can you just expand a little bit on how you've seen that landscape evolve over the past year and just kind of what you're seeing with the new entrants.
Charles Gayer
ExecutivesSure. Yes, HAE for a rare disease is very competitive. I think there's 10 or 11 products approved right now, which is fantastic for patients. The most recent approvals have been an oral acute therapy and then 2 injectable prophy therapies. And so as I was describing, we have the most differentiated prophy therapy as the only oral prophy on the market. And for many patients, that's what they're looking for. And so that's a segment that we really own. Overall, the market keeps going more and more towards preventing attacks with prophylaxis, which is the right thing to do. So it's probably around 80% of patients in the U.S. are on prophylaxis, and we see that continuing to grow. And then having the opportunity to treat the occasional breakthrough attack with an oral acute therapy is a great opportunity for patients. So we see that as a potential benefit for patients if they can be on ORLADEYO plus an oral acute is great. But the oral -- or sorry, the injectable prophy products moving from every 2 weeks to once a month is a nice to have for patients, but it's not changing the trajectory for ORLADEYO because of our differentiation. So we've seen all through 2025, even as new -- new products came on, continued strong demand for ORLADEYO.
Jessica Fye
AnalystsAnd with the European ORLADEYO rights divested, can you also just talk through in a little more detail how ORLADEYO still gets to $1 billion peak sales without that region. So like how much of that growth is coming from market expansion versus market share?
Charles Gayer
ExecutivesSure. The -- several years ago, we saw -- we announced that we saw ORLADEYO getting to $1 billion. That was with a 12 and up indication. As Jeff was suggesting, that also included our European business. So when we sold off our European business, we looked at our current U.S. business. We also looked at the upcoming opportunity in treating kids down to age 2. And based on the pace of growth in the U.S. the addition of the pediatric population, which is both underdiagnosed and undertreated from a prophy standpoint and then modest growth from other regions. We see ourselves still getting to $1 billion because of that product differentiation, capsules for 12 and up, oral pellets for kids in a market where they only have injectable opportunities right now. You put all that together. So we'll get most of that $1 billion in the U.S. and then contributions from Japan, Canada and other regions. Yes, the question was about Avoralstat, which is I didn't mention this, but it's on our pipeline slide, which is Avoralstat is being developed for diabetic macular edema. We're in the middle of a Phase I proof-of-concept study in DME patients. And we decided in the second half of last year, to finish this Phase I study, but that this is not a rare disease. So this is not core to our strategy. So what we're going to do is finish that program, and we're going to be looking for partners to spin it out to out-license it. We think that having a kallikrein inhibitor dosed via suprachoroidal injection. So at the back of the eye has the potential to be there for many months to inhibit kallikrein is a great opportunity for patients. But we're going to finish that Phase I, see what we have. We're making good progress on it and then see if we can out-license that this year.
Jessica Fye
AnalystsSo for the ORLADEYO business, you talked about by 2029 when you reach a steady state, the franchise just might not require the same level of resourcing. So how should investors think about the SG&A associated with ORLADEYO as it matures? And how should we think about kind of terminal operating margins with that franchise?
Charles Gayer
ExecutivesSure. Maybe I'll start and then Babar can add some comments to that, which is we have a small sales force. We have 40 rare disease specialists covering the market. And we think they cover the HAE space better than any other team. They are super excited about launching Navenibart, and they can do ORLADEYO and Navenibart at the same time. As ORLADEYO reaches peak the market is going to be very familiar with it. We don't need to spend as much promoting that. And so maybe that takes a second position to Navenibart. We devote more of our efforts to Navenibart our marketing dollars to Navenibart. So basically, same pool of money, same great team just shifting the priorities around, so very similar OpEx. Babar, is there anything you want to add?
Babar Ghias
ExecutivesYes. And I think we disclosed this in our quarterly reports that at this point in time, we are not spending more dollars to buy revenues. The only expansion that you see in our S&M expenses primarily, let's say, you're selling more, you're going to pay the specialty pharmacy fees more. So at this point in time, I think the expense structure for the base business is steady state. But even further, I think the overall business is reaching a point of equilibrium in the sense that as programs advance or basically, we are also very disciplined in terms of cutting programs that do not make the mark. We expect that the R&D expenses to remain fairly standard and going forward as well.
Jessica Fye
AnalystsAnd then I guess just bigger picture, you've laid out like very detailed long-term projections. What's the rationale behind kind of like why you're providing such kind of specific long-term guidance for folks?
Charles Gayer
ExecutivesWe -- we have -- let me start with the revenue and then we can talk about the OpEx, too. So we have launch to date, a big part of our success has been our ability to do deep market research and really predict what patients and doctors want. And we do very detailed models that predicts the future in this space. It's been very predictive of where ORLADEYO has gone launched to date. Basically, 5, 6 years ago, we predicted where we are -- where we were in 2025. We're doing the same thing for Navenibart. So we want to help investors see what we see from that standpoint. And then from the operating expense, we really want to show how disciplined we are around our capital allocation right now and how we're balancing between those 3 priorities of driving the commercial growth, devoting what we should devote but not going overboard on the internal R&D and then allowing for cash generation for business development as the opportunities come up.
Jessica Fye
AnalystsSo then maybe related to kind of the R&D outlook, if the Navenibart Phase III is going to be kind of ongoing in '26 and wrapping up the data in '27. What's the right way to think about kind of the near medium-term R&D trajectory?
Charles Gayer
ExecutivesYou want to handle that one, Babar?
Babar Ghias
ExecutivesYes. So I think on the Navenibart side, there is going to be an incredible amount of synergies from the Astria acquisition since we are not going to have the G&A overhead. So we are guiding to this morning that upon closing of the transaction, we'll have about $70 million to $80 million of additional burn. But that will essentially go down because that trial is in its Phase III enrollment. So we are preparing for -- we're doing the enrollment, we're preparing for pivotal CMC activities. So going forward, that expense will come down. But naturally, if 17725 is successful, then essentially, we'll see some pivotal trial. But having said that, and this goes back to the point in terms of strategy, any rare disease trial that you're looking at, you're not looking at several hundred million dollars of expense. So year-on-year, you'll see some expenses go up. Others go down as trials complete, but we're not looking at several hundred million dollars of R&D expense by nature of our business in terms of focus on rare diseases.
Jessica Fye
AnalystsSo you talked about the [indiscernible] trial, and I think you talked about Part 4 data by the end of '26. What about Part 3? Do we get that kind of in between there?
Charles Gayer
ExecutivesSo I'll start on that and then Bill can add some color. Both Part II and Part 4 are open label. Part 3 is only 3 doses over a month. Part 4 is 6 doses over 3 months and then follow up. And the other key thing here is that Part 3 is not a gate to part for. It's really about when the sites are -- have the IRBs approved to go into Part 4. And so some patients will go straight into Part 4, and we may just have a couple of patients in Part 3. So what we're looking for is when the outcomes in patients are really significant. When we see clear signs of clinical improvement that allow us to declare that we have a drug that can move on to a Phase III pivotal trial. Bill, anything else to add?
William Sheridan
ExecutivesSure. What matters for us is seeing a big treatment effect in enough patients to give us the confidence to invest in a pivotal study. So 12 weeks of opportunity of dosing and with follow-up gives us more of an opportunity to see that. We're measuring, as you would expect, symptoms of pain, itch, redness, well-being, general health and if the drug gets into the skin in sufficient quantity, I'm confident it will work. We've got the right target. So a very exciting program. I can't wait to see the data. But you should focus on having sufficient numbers of patients through 12 weeks of dosing to give a realistic estimate of treatment effect size.
Charles Gayer
ExecutivesAnd the one thing I'd add is we're seeing a lot of enthusiasm from -- there are not that many KOLs yet who focus on this disease, but the global KOLs, the U.S., Europe, Australia who see this disease are very enthusiastic about this, the profile of this product and helping us complete this trial.
Jessica Fye
AnalystsAnd you talked about a big treatment effect and then you kind of outlined a number of clinical metrics. So have we moved beyond looking at biomarkers and now that we're in patients?
William Sheridan
ExecutivesThis disease pathology is all -- is starting the epidermis. So there isn't really a plasma biomarker of use for estimating pharmacodynamic effect like there would be in a disease like HAE, where you could use kallikrein inhibition as a tool. So this area lacks that. So we really have to focus on clinical outcomes. So it's all about the clinical outcomes here.
Jessica Fye
AnalystsAnd then is the idea that you would go straight into some kind of pivotal study? Or is there kind of like a Phase II step in between?
William Sheridan
ExecutivesThe ideal progression here is to see dramatic improvement in the skin and go straight into a pivotal. And given that there are no approved therapies, we're anticipating regulatory engagement, both with the FDA and with international regulators to enable that to happen once we have the data from this study.
Jessica Fye
AnalystsCan you give us a bit of a framework maybe some epidemiology stats on just how to think about the opportunity in Netherton syndrome?
Charles Gayer
ExecutivesSure, there's first caution, I would say is don't just look at the literature because like any really rare disease at this stage in the game, you look at the literature, you'll see numbers of case reports. And so what we've been doing is going deeper than that. There's no ICD-10 code for this disease. So we've done some work in a year or so ago looking at mentions of bamboo hair in claims data. Bamboo hair is a feature that kind of is what it sounds like. You look at hair, particularly under a microscope. It looks like bamboo that is distinct to Netherton patients. And we found about 1,600 U.S. patients with mentions of bamboo hair. But we weren't satisfied with just that. So we're going deeper. As I mentioned, we're looking at EMR. We're using NLM technology to look at EMR data, to look at other features of the disease, other diagnosis patterns. And we think that this is now likely a 3,000-patient plus market. At some point in the future, there'll probably be an ICD-10 code. But right now, it's really critical for us to do everything we can to determine the size, understand the size of this market, and we think it's a lot bigger than what you'll see in the literature.
Jessica Fye
AnalystsAnd it sounds like highly symptomatic. So is this like a difficult diagnosis? Or are these patients -- I would think they would become well identified. How do we kind of reconcile that with the idea that they may be underdiagnosed.
Charles Gayer
ExecutivesThe simple story is what I said earlier, which is doctors don't look for things that they can't solve. And there's no diagnosis code, so they can't peg it there. Netherton syndrome is particularly acute and life-threatening for kids, for infants because at young ages, children's skin turns over a lot faster. Kids are growing and maintaining a skin barriers even that much more important. So it can lead to more diagnosis there, but some of these kids are missed. Then as patients get older, it becomes more of the chronic stage, which is still a really difficult disease. But as I mentioned, patients spend their whole day operating around this disease. They typically apply creams, over-the-counter creams across their whole body multiple times a day. They have other special things that they learn to deal with. But what we hear from patients is because their doctors have nothing to offer them, they get frustrated, and they stop going to ask anymore. So they kind of disappear into the woodwork of the health care system. And we think that having a targeted therapy is going to help them come back out. And we've seen this. We heard the story in HAE, where patients would get misdiagnosed. They kind of bumble along on their own for the years, but now that there's therapy. They're with good doctors who know what they're doing, and it's completely changed in the last 15, 20 years.
Jessica Fye
AnalystsSo coming back around to Avoralstat for DME, now that that's kind of in the bucket of kind of potential licensing opportunity, should we expect to hear about the initial data or only kind of when that deal materializes?
Charles Gayer
ExecutivesMost likely when a deal materializes. Let me tell you where we are right now. We've got -- in the Phase I study, we've got 3 cohorts. We've completed the first cohort at a 100-microgram dose. That was by design, is really a safety dose. So we do not expect to see much efficacy from that. The next cohort goes to 250 micrograms and the third cohort goes to 500 micrograms. We just opened the IND in the U.S. So we're starting to enroll U.S. studies. Previously, it was just in Australia. And so we're confident that towards the middle of this year, we'll have a lot more data there. But you're likely to see the data more when we announce a deal.
Jessica Fye
AnalystsGreat. So we're about out of time, so we'll leave it there. Thank you.
Charles Gayer
ExecutivesGreat. Thanks, Jess.
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