BioCryst at TD Cowen Conference: Strategic Growth Insights

On Wednesday, 05 March 2025, BioCryst Pharmaceuticals Inc (NASDAQ: BCRX) presented at the TD Cowen 45th Annual Healthcare Conference, offering a detailed look into its strategic advancements and financial outlook. The company shared optimistic updates, including an upward revision in sales guidance, while also addressing challenges such as leadership transitions.

Key Takeaways

• BioCryst raised its 2025 sales guidance for Orladeo to $535 million-$550 million.
• The company aims for $1 billion in peak sales for Orladeo, driven by strong market growth.
• An external search is underway for a new CFO following Anthony’s departure.
• Positive pediatric study results for Orladeo could influence broader treatment adoption.
• BioCryst is focused on achieving profitability and substantial cash flow generation.

Financial Results

• Orladeo Global Sales Guidance (2025): Increased to $535 million-$550 million.
• Peak Sales Estimate: Targeting $1 billion in global peak sales.
• Ex-U.S. Contribution: 12% of total sales, with expected growth of 1-2% annually.
• 2025 Revenue Guidance: $535 million to $550 million.
• 2025 OpEx Guidance: $425 million to $435 million.
• Revenue CAGR (2025-2027): 20%.
• Expense CAGR (2025-2027): 5%.
• Projected 2027 Revenue for Orladeo: $750 million.
• Projected 2027 OpEx: $450 million.
• Projected 2027 Operating Profit: $300 million.
• Projected 2027 Cash Flow: $200 million.
• Medicare Paid Rate: Aiming for 80% by year-end, up from 55% last year.
• Gross to Net Impact: Co-pay assistance and reauthorization processes affect revenue.

Operational Updates

• New Patient Starts: Consistent with the first year of launch, indicating strong growth.
• Free Drug to Pay Drug Conversion: Improved to 80% by end of the previous year.
• Pediatric NDA Submission: Planned for this year, based on positive pediatric study data.
• BCX1775 (Netherton Syndrome): Phase 1 study underway, patient enrollment planned.
• DME Program: Clinical trials to begin this year, focusing on plasma kallikrein inhibitor.
• Prescriber Growth: Adding 60 new prescribers quarterly.
• Source of Business: 52% from switches, 17% treatment-naive.
• Retention Rate: 60% stay on therapy after a year, with minimal attrition.
• Market Growth: Estimated at 11,000, with Pro fee growing from 7,300 to 9,300.
• Pediatric Data: Median age of onset swelling at two years.
• Other Prescribers: Continuing steady growth among 1,200 prescribers.

Future Outlook

• Orladeo Peak Sales: Confident in reaching $1 billion.
• Ex-U.S. Growth: Expecting steady growth in international markets.
• Pediatric Market Impact: Anticipating a "halo effect" from pediatric data.
• BCX1775 (Netherton Syndrome): Aiming for patient data this year.
• DME Program: Initial patient data expected this year, with vision data next year.
• Profitability Focus: Committed to profitability and cash flow generation.
• Competitive Landscape: Confident in Orladeo’s competitive position despite new injectables.

Q&A Highlights

• Anthony’s Transition: Transition period ends on April 9.
• CFO Search Confidence: Confident in finding a suitable replacement.
• IRA Impact: Charities remain operational.
• First Quarter Guidance: Expected to be flat to down.
• Patient Demand: Strong feedback and satisfaction reported.
• Tolerance Improvements: Significant improvements noted.

Readers are encouraged to refer to the full transcript for a more detailed account of the conference call.

Full transcript - TD Cowen 45th Annual Healthcare Conference:

Stacy Ku, Biotech Analyst, TD Cowan: Alright. Good morning. Thanks so much for joining us at TD Cowan’s health care conference. My name is Stacy Ku, one of the biotech analysts, and I’m here with my colleague, Vish Shah. We’d love to welcome John Stonehouse, CEO of BioCryst, Helen Thackeray, Chief of R and D for Fireside Chat and, of course, we have Nick Wilder in in the in the audience.

So thanks so much, all of you, for joining today.

John Stonehouse, CEO, BioCryst: Thanks for having us.

Stacy Ku, Biotech Analyst, TD Cowan: So ahead of our q and a discussion, John, do you want to just very briefly share any color on the announcement, that we saw yesterday morning for Anthony?

John Stonehouse, CEO, BioCryst: Yeah. Before I do that, Helen and I are going to be making some forward looking statements. They have risks. You can find our risk factors on our website. Yeah.

You know, it’s sad to see Anthony go, but we’re super grateful that he gave us a transition to April 9. He’s built a fantastic team. He added a ton of value to the company through a lot of the debt and royalty financings. And as a result, the company is in the strongest financial position we’ve ever been in. And so I think the profile of our company now to attract a really good CFO is is fantastic.

And the search has started. We’re looking externally, and and, I’m real confident we’ll find somebody great.

Stacy Ku, Biotech Analyst, TD Cowan: Okay. Well well, wonderful. Obviously, the fundamentals have been incredibly solid. John, Oradeo is obviously now entering the fifth year of launch. And what struck us, when we were kind of preparing for for this chat is company is now guiding 25 revenues that are above the original peak sales estimate that you all initially issued.

So clearly, well on your way, the billion in in overall global peak sales. To be backwards looking for just a moment, you know, walk through the different drivers of growth, that, as we look at Oral A Deal’s performance in ’24, which actually grew on a year over year basis. And then, of course, as we look to ’25, what gives you conviction in your guidance of global Oral A Deal sales, which you recently increased, to $535,000,000 to $550,000,000, up from $515,000,000 to $535,000,000 just a few months after kind of months and a half after giving initial guidance for the year.

John Stonehouse, CEO, BioCryst: Alright. So if I forget some of these questions, you guys just remind me of Absolutely. All right. So let’s start with your statement. It is pretty wild to think that the original guidance was 500 and then 500 plus and then the emphasis on the plus.

But yes, I mean, we’re extremely confident in the billion dollar peak sales. And so let’s explain what we’re seeing in 2024. I think the first piece is the underlying growth, new patient starts was the same as it was in the very first year launch. That never happens in a rare disease launch four years out. And so why is that?

I think one of the things, the strategy that we had is to put out data to show that when Orlodeo works, it really works like injectable therapies. Right? Ninety eight high eighties, ’90 percent reduction in attacks. A lot of attack free months. So people are controlled.

This myth of people will sacrifice efficacy for convenience is just not true. It’s a myth. And so we put out conference after conference after conference going through this last weekend, Quad AI, and Helen can talk about it, you know, more and more really large real world studies, four fifty patients in type one, type two, three fifty patients in C1 inhibitor. And so what docs are seeing from that data is it works. It really works.

And so that’s caused them to try it, and then they have their own experience in their practice, and they see it work in their practice. All of that has led to an increased confidence in the drug. And

Helen Thackeray, Chief of R and D, BioCryst: one of

John Stonehouse, CEO, BioCryst: the things we’re talking about in one of the investor meetings earlier this morning is this, there’s two things that cause an attack, low C1 inhibitor and trigger. And stress is a trigger. When you’re controlled on an oral once a day, you forget you’re sick. And so everything just gets better, and people, you know, have this ease of life that is less worrisome about their disease or their therapy. And and so that’s what’s causing and the other piece that we made progress on the commercial side was improving the free drug to pay drug.

We’re now in, you know, a couple of years ago, we were in the low 70s, high 60s. It’s 60% of our business and now we’ve moved to 80 by the end of last year. So that was a big improvement. And then to answer your question about ’25, we put out guidance at the beginning of the year, and we said what wasn’t in that guidance was what would happen with the IRA and the paid rate on Medicare. And what we’ve seen now, and that’s why we increased it last week, is that it’s snapping back a lot faster.

And and we believe that we could be at 80% paid by the end of the year with Medicare, which makes up about 20%. We were at 55 at at the end of last year.

Stacy Ku, Biotech Analyst, TD Cowan: Wonderful progress. And, like, and kind of a quick comment on on something that you brought up. The cable feedback is is I think it’s begrudgingly become more and more positive, and a lot of it is coming from the patient demand and the patient satisfaction. And if patient’s coming back and saying, I’m feeling better. This is the drug I want.

Then clinicians are actually more willing to prescribe it to others because they are hearing that real world evidence of those that are that respond very well to Orla Deo. Let’s let’s drill down a little bit into kind of your your other comment for ’25, for the IRA Medicare redesign. So are you seeing kind of in q one a a lot of HEE patients that are Medicare successfully signing up and converting over to paid drug? Are we kind of still in the knife fight of Q1 right now? How are you thinking about charity support, the transition?

And I know you’ve talked about a lot of different factors.

John Stonehouse, CEO, BioCryst: Yeah. I mean, honestly, the piece that we see that gives us the confidence is that the paid rate goes up and it’s gone up in, you know, what, the first two months of the year. And so, and it’s gone up a lot. So we thought this was going to be a gradual kind of three year process. And what happened is it snapped back.

And I think, you know, we can speculate of why, but the two main charities that provide co pay assistance for HAE patients are open for business, and and they weren’t. The last two years, they they had waiting lists, and so that’s changed. So we feel that IRA had contributed that because it’s a lower total amount. So money can go further.

Stacy Ku, Biotech Analyst, TD Cowan: Okay. Understood. Understood. And then kind of last near term discussion on ’25, maybe talk about the ex U. S.

Contribution really briefly. And again, another really short term question. You all talked about Q1 being kind of flat or maybe a touch below or, you know, somewhat in line with what we saw in Q4. Last year, you all gave very detailed quarter over quarter commentary. Is that gonna be a similar pattern this year?

John Stonehouse, CEO, BioCryst: Yeah. I think we did, and I’ll I’ll do it again here. But let me, well, let let me go to that. Because of all the effects of co pay assistance on the commercial side, right, and deductibles having to pay that off upfront, and so that hits the gross to net. You know, 60% of our business is commercial and a lot of those plans have reauthorization at the beginning of the year.

So there’s some period, where patients are on free drug. We’d like it to be as short a period as possible to get them on to paid, but the offices have to go through the reauthorization. So it could be one month, two months, three months, four months. We try to push it as short as we can with the offices. And so it’s inevitable.

And when you have more patients on therapy, you’re going to have more of a hit on co pay assistance. You’re going to have more patients that have to go from prior auth back to paid. And so yeah, to be really clear, our guidance in the first quarter is flat to down. Overall, we’re going to have a very strong year, but our guidance is flat to down. And then on the ex U.

S, your first question, it’s just steady, nice steady growth. It’s the fourth quarter is always a little bit larger in terms of percentage because of some distribution arrangements that we have, but and I think it was around 13%, fourteen %. But if you look at the full year of what contribution ex U. S. Had, it’s about 12, and it’ll grow a percent or two each year, roughly.

Stacy Ku, Biotech Analyst, TD Cowan: Okay. Understood. So you all were just at QuadAI. Just maybe talk about the patient demand. Is it you all going after new clinicians?

Are you all trying to kind of have clinicians write more? What is the dynamic now as we’re kind of thinking about a more mature launch?

John Stonehouse, CEO, BioCryst: Yeah. I’ll let Helen, because she was there, talk about QuadAI because it it sounded like it was a lot of excitement. And then I’ll come back to what are we seeing in terms of prescribing.

Helen Thackeray, Chief of R and D, BioCryst: Yeah. So Quad AI, we had some great data come out and that drove some of the conversations. So we had these two large datasets, real world evidence, looking at types one and two HEE and then C1 normal HEE. That drives a lot of the discussions around how well patients are doing. It’s over 800 patients worth of data.

So it really drives the discussion with physicians around, this is a drug that’s working for patients who are taking it, doing well, they’re doing really well. And so we get to the physician conversation. We also have, the interest of patients. So I think to your question, I think it’s both. I think there’s new patients and there’s the increasing prescribing by physicians.

And we’re seeing and hearing things that are very consistent with that as we talk with physicians about their excitement at Quad dot ai. Okay.

John Stonehouse, CEO, BioCryst: So source of business, 52% is coming from switches, and this has been pretty consistent. It probably will change because on demand will change over time. We believe that on demand or prophy will go to 85%. It’s probably more in the 75% to 80%. Percent.

32% switches from on demand and then 17%, treatment naive patients. And then on the doc side, there’s 500 of the top prescribers that make up 50% of the prescribing for HAE. We’re at about 80%, but we’re continuing to chip away at that 20% and then get them to go broader because they have larger practices. And then on the other 1,200, we’re continuing steady growth. We’re adding 60 new prescribers every quarter.

So that tells you something. Okay.

Stacy Ku, Biotech Analyst, TD Cowan: That’s I mean, that’s incredible if the 60 prescribers each write one prescription. That is very meaningful ads. Okay. Wonderful. Helen, you kind of alluded to this a little bit, but the size of the market is actually rapidly evolving, rapid at least in the last few years.

You last spoke about the number of patients that are diagnosed and treated is actually closer to eight thousand five hundred with around seven thousand two hundred prophylaxis patients. Correct me if I have the math wrong, which should grow to over nine thousand. So as we think about your opportunity and and maybe even the growth of prophylaxis treatment, how are you thinking about within your estimates and expectations the growth of the market and put that in context of your billion dollar big sales?

John Stonehouse, CEO, BioCryst: Yeah. Let me start with just correcting you slightly. So the market we believe now is around 11,000. And we see pro fee around 7,300, I think it’s 7,500 last year, it’ll grow to above 9,300 and so in 02/1933. So steady, steady growth and that’s not surprising.

It’s about 5% growth per year. And that’s not surprising in a rare disease, even one that’s established and has so many therapies because normal C1 inhibitors, another really interesting population that we’ve produced, as Helen said, a really robust data set that shows market decreases in attack rates from baseline. And that’s a population, I think, in general that physicians had trouble with and therapies weren’t as effective. And it’s a third of our business. And so that’s lumped into that number now and this contributing to some of the growth.

Stacy Ku, Biotech Analyst, TD Cowan: Okay. Wonderful. Helen, can we talk a little bit more about the pediatric study results? Apex P, clearly,

Helen Thackeray, Chief of R and D, BioCryst: I believe you had all said submitting the pediatric NDA this year? Yes. Yes. So we at QuadAI, we had our pediatric dataset presented in a late breaking abstract, this is last Sunday. And it’s that dataset that is the basis of the sort of the information that we’ll be putting into for the NDA, which will be this year.

We learned a couple of things in that data set. So first, we were assessing the safety, tolerability, exposure levels of oral granules of Orlodeo in the pediatric population. So we gathered the data that we need to be able to submit and talk about dosing with the agency. Secondly, we saw a clear impact on that population, patients who are doing very well, the pediatric patients. This is a large data set.

It’s twenty nine patients of whom twenty five are continuing therapy at the end of the timeframe. So great continuation rates and very good improvement in attack rate. Thirdly, we learned with that that the pediatric population, in fact, has very severe disease starting at very young ages. And that seems to be a surprise, in the field that the conventional wisdom is that this is a disease that really is seen more starting in adolescence. What we saw was a median age of onset of swelling at two.

So there’s a very high need in the youngest patients. There’s also very severe disease with hospitalizations in the youngest patients. And so we’re very pleased to have an oral granule formulation that may be coming for those patients to be using as prophylaxis.

John Stonehouse, CEO, BioCryst: And this halo effect, I think, is going to be really interesting to see because we see it with adolescents, you know, 14, 15 year old kid in a family of HAE patients does really well on Orla Deo and the parent starts to think, hey, maybe I should try it. And so we think we’re going to get that with the pediatric population as well. And none of the pediatric numbers are in any of our guidance or in our billion dollars. So that’s a plus.

Stacy Ku, Biotech Analyst, TD Cowan: That’s going to be one of my questions. And it’s interesting you talk about the 25 patients that stayed in the study. It could be interpreted two different ways. Either the tolerability is incredibly, kind of better than expected, I would say, or these clinicians are getting a lot of experience with Orla Deo and being able to guide kind of patients through that initial few months of trial and error and and then realizing how to recognize

John Stonehouse, CEO, BioCryst: the Orla But the rate of AEs was different though, wasn’t it, Helen? Yeah. So Yeah. So it’s not just the dropouts, it’s the rate of

Stacy Ku, Biotech Analyst, TD Cowan: GI The actual GI

Helen Thackeray, Chief of R and D, BioCryst: experience and the tolerability.

Stacy Ku, Biotech Analyst, TD Cowan: So the tolerability significantly improved.

Helen Thackeray, Chief of R and D, BioCryst: We think tolerability is very good with oral granules. Wonderful. Great. And just one last point. I did have a conversation at AAAAI, with Raffy Tachian, who’s an expert and a pediatrician, expert in HEE.

He’s at UCLA. He was able to give some color to the the, how physicians in the field look at the pediatric population, how they look at the overall population, and the changes that are that are, seen with oral prophylaxis.

Stacy Ku, Biotech Analyst, TD Cowan: So that was that was

Helen Thackeray, Chief of R and D, BioCryst: a, really interesting conversation, and I point you to the, that’s available on our website as well. Yep.

Stacy Ku, Biotech Analyst, TD Cowan: That’s a that’s a great transition to kind of the overall CAEL perspectives on, the need for oral agents in HAE, especially for for prophylaxis, just given kind of the quantity of treatment. What are your overall views, in terms of the industrial market, the oral market? Those are kind of transition and are well controlled on orals. How should we think about, maybe the moat that you all have, when it comes to Orla Deo? And, of course, I know last fireside chat, we spoke ad nauseam about the compliance rate and retention rate of of patients on Orla Deo.

But it’s something that continues to come up, and I we do think it’s very underappreciated.

John Stonehouse, CEO, BioCryst: Yeah. You know, the the retention rate now is, you know, we got four years of it and it’s solid, right? It’s sixty percent stay on therapy at a year and we lose very little after that. And so, and the number one reason for somebody going off is, you know, unfortunately with some, the drug doesn’t work, right? But when it works, it works as well as injectable therapy.

So, in terms of the evolution of the marketplace, there’s always going to be a need for rescue therapy because not a single one of these drugs, even the ones under investigation are perfect. And everybody has breakthrough attacks. And remember, what causes a attack is low C1 inhibitor and a trigger and stress is a trigger. So sometimes life gets in the way and you have an attack even when you’re on a really good therapy. So, but but clearly things are moving to prophylaxis.

I mean, we’re hearing that around the world now, and and it was less so in Europe and and in Asia and in Latin America. And the exciting thing is that the worldwide guidelines now are very clear. They’re contributions from physicians all around the world. And it’s, you know, the recommendation is you should be offered prophylactic therapy because that just makes complete sense because why would you suffer from an attack and treat it versus prevent it? So I see that continuing to move more and more towards prophylaxis.

You know, there’ll be competitive, injectables coming out this year that will create more noise around that. And we think, you know, if you’re good, if you’re ready to switch, of course, you would try Orlodeo first to see if it works for your patient.

Stacy Ku, Biotech Analyst, TD Cowan: Our KOLs do tell us that additional injectable entrants are very unlikely to impact the oral prophylaxis options in HAE. But maybe as we think about that switch market, you kind of alluded to the injectable entrance, maybe agitating some more of these, patients that have been on, you know, twice weekly injections or or even every two or every month injections. So just maybe talk about your views on what the other entrants are going to do in more detail.

John Stonehouse, CEO, BioCryst: Yes. We put out some pretty extensive market research that we’ve talked to you about, and it’s in our slide deck. And it’s the most robust market research that I’ve seen in this space in terms of numbers and then methodology, doing both conjoined preference share and then putting it in a market simulation and doing Monte Carlo analysis on 6,000 simulations. I think so it might be worth a little bit of time to just talk about the dynamic we see in the market. In general, the number one obstacle that we get when somebody is on prophylactic therapy is, if they’re controlled, why would I change?

Like, you know, if it isn’t broke, don’t fix it. And so getting a physician to understand that there’s very little downside to trying Orla DAO and they could get that same control on a once daily capsule is our job. And because patients don’t see doctors more than once or twice a year, your opportunity to influence that is very infrequent. So that’s why you see this kind of steady trajectory versus a hockey stick. It’s only going to get harder for the next people that come into the market, right?

It was easier for TechSiro, it was harder for us, it’s going to be harder for the next one and even harder for each one after that. And so when you look at the output of our market research, you see that slow chipping away of the new entrants. It’s not a hockey stick for any one of them. And it’s taking largely away from Taxaro. But not so surprisingly, at the end of ’twenty three or ’thirty three, excuse me, we you look at the market leaders in terms of patient share and it’s Taxaro and Arliteo.

And why is that? Because it works. And and so you have to have something really disruptive to get people to say, yeah, I’m ready to switch. And and oral therapy is disruptive. And, you know, we’ll have years of opportunity to keep chipping away at that.

Stacy Ku, Biotech Analyst, TD Cowan: So Yep. And and, of course, you have durability to 2,039. And and, again, to your point, you are doing a lot of different work to make sure Oladeo remains relevant, switch studies. I think it’s all, within the competitive landscape, really, really compelling. So kind of hit on the the the peak sales opportunity for for Oladeo.

So we do wanna make sure we leave enough time for pipeline. So we as we are heading into kind of the second half of this year, we’ve get been getting a lot more high level questions on BCX one seven seven two five. So, Helen, if you could talk a little bit more about this asset and what are you looking for specifically to move forward? And we’re obviously then move into the kind of the market opportunity.

Helen Thackeray, Chief of R and D, BioCryst: Yes. So this is our next drug in the clinic, where our goal is to have a drug that is comes to market as a success behind Orla Deo. And so we have several opportunities to do that. BCX1775 is a fusion protein and a KLK5 inhibitor. And our goal with that is to deliver a functional cure for Nethersen syndrome, which is a genetically driven disease.

It has a missing protein function. That missing protein function is a regulator of KLK5 and our product is intended to deliver replacement of that missing regulation of KLK five. Netherson syndrome is a severe disease, is an ultra rare disease. It is genetic, And it’s a it’s a skin condition in which the pathology is disruptive peeling of the skin, disruptive separation of the skin. So the skin is not normal, and it is thin, it scales and peels, and it is highly inflamed.

So there’s both skin peeling and inflammatory components. Those stem from KLK5 activity being unregulated. And, what we’re going to be doing with our program is moving forward, we’re now in healthy volunteers, moving to patients to assess seventeen seventy five’s ability to get into the skin and to have activity in suppressing KLK5. And then finally, this year, to look at the skin healing as a result of suppression of KLK5 and suppression of that both the cascade of skin separation and then the associated inflammatory cascade. And our goal is to have data in patients this year showing, all those penetrations of the skin activity on target activity in the skin and then finally healing of the skin as a result of this functional replacement of the missing protein.

Stacy Ku, Biotech Analyst, TD Cowan: Okay. You all had maybe previously discussed starting to dose patients by q three. And the healthy volunteer in patient study, is that gonna happen around the same time as you’re getting, collecting information from healthy volunteers? Or are you going to finish healthy volunteers before then moving on to the study?

Helen Thackeray, Chief of R and D, BioCryst: So this is all one study. And that’s one of the things about an ultra rare disease is you want to get into patients as soon as you can. For us, that means in the same Phase one study. So we’re currently dosing in healthy volunteers, dose escalating through a standard approach. And our plan is to open sites to enroll patients in that same study this year and have that first experience in patients.

We would then go to a larger study once we have information around the dose, so that’ll be next year, in which we’re assessing a larger number of patients with durability of effects to look for long term healing.

John Stonehouse, CEO, BioCryst: And Helen, you might want to just talk about the difference in KLK5 and what you see in healthy versus what you see in patients that will give us information about do we see activity or not?

Helen Thackeray, Chief of R and D, BioCryst: Yes. So there is a difference in what we can gain with this in healthy volunteers versus patients. KLK5 is our target, of course. It is normally regulated in healthy skin. And so it doesn’t bind to the drug.

We wouldn’t expect it to bind to the drug. We wouldn’t expect to see skin levels or skin activity because there’s no abnormal activity of KOK5. However, in patients, abnormal KOK5 activity is the target. And so that’s our opportunity to, in the course of the same study, assess the drug’s activity as well as clinical outcomes.

Stacy Ku, Biotech Analyst, TD Cowan: I know it’s still early days, but, are these patients you know, I know you had it’s around sixteen hundred. Are they essentially diagnosed and available to be enrolled in a clinical trial like this? Is it gonna take time to kind of go out and find these patients? I know you all are doing a lot of work around kind of trying to

Helen Thackeray, Chief of R and D, BioCryst: understand the true prevalence of the of nethertins as well. So we certainly have patients identified and available for clinical trials at this point. However, this is a disease where there’s no targeted therapy available. It’s a disease for which a genetic test is necessary to get diagnosis, and it’s also a disease that’s treated with standard of care for a larger sort of category of conditions that are ichthyosis conditions or scaly, very severe skin disease. We think that there are about we’ve estimated about sixteen hundred patients in The U.

S. Based on looking at a term, which is specific to Netherland syndrome, and that’s bamboo hair. It’s how the hair looks under the microscope. But that we think that significantly under recognizes the actual population with Netherland syndrome. And as a targeted therapy becomes into trials and then becomes available, we would expect physicians to be doing testing on patients who have ichthyosis to see if they have the subcategory of Nothergent syndrome when we may see, enlargement of that under patient populations identified.

This is not uncommon for rare disease. It’s certainly what happened with HAE. At the advent of targeted therapies available for HAE, the population started to be understood to be much larger than originally. I think there are 2,000 patients originally, and now we’re at 11,000 patients. And I would expect something similar to happen with Netterton syndrome once a targeted therapy is available that makes it important to then diagnose in this patient why do they have hysteosis.

Okay.

Stacy Ku, Biotech Analyst, TD Cowan: And you all are trying to go for every two week dosing or better?

Helen Thackeray, Chief of R and D, BioCryst: Yes. Or better, yes. So this is a potent therapy. It has very high affinity, which means once it’s stuck to the target, it doesn’t come off. And we expect it to be in the skin for the duration of the KLK5, that level of skin being there.

So we expect to dose, we’re targeting every two weeks. We will have to see in the patient data whether that’s actually the the range or whether it’s something longer as skin turnover takes place. But this would be a subcutaneous dose, and it would be about every two weeks to start.

Stacy Ku, Biotech Analyst, TD Cowan: Okay. And we’re also expecting some updates for your additional pipeline program in DME. Very briefly, do you want to just discuss the vorasat, what you’ve seen so far that gives you a lot of excitement and maybe the cadence of updates?

Helen Thackeray, Chief of R and D, BioCryst: Yeah. So this is our next program after Nathalie Syndrome, and it will be in the clinic this year. It’s a program that will go will be dosing patients initially rather than needing to go through healthy volunteer studies. But it’s a program with a plasma clicline inhibitor that has unique properties that make it appropriate to instill in a depot like suspension in the suprachoroidal space in the eye. We I’m excited about this because we now have some evidence starting to build to show that this could be really this could be a differentiated mechanism of action.

We have a drug that could be delivering sufficient exposure to the right place in the retina to get activity and enough to have improvement in clinical outcomes with plasma chalocline inhibitor where that’s been elusive in trials so far, and we think that’s probably a result of having inadequate exposure in the right place. So we will be in patients this year, our very first few patients will be looking for the outcomes that will tell us if we in fact have a therapy to take forward. We’ll be looking for the edema and changing the edema this year. And then next year, we’ll be looking for improvement in vision.

Stacy Ku, Biotech Analyst, TD Cowan: Okay. In the last few moments, obviously, you are advancing the pipeline, but also being conscientious stewards of your capital. So So maybe just remind us your guidance on the next few years in terms of growth of OpEx spend versus Orla Deo sales.

John Stonehouse, CEO, BioCryst: Yes. So the guidance for this year, revenue, as I said before, is five thirty five to five fifty. OpEx is four twenty five to four thirty five. And then we also gave the compound annual growth rate 25 through 27. So on revenue, it’s 20%.

On, expense, it’s 5%. And so what that gets you to, if you do the math, is $7.50 in revenue for Orlodeo, four fifty in OpEx. And so operating profit of 300 below the line, it’s around 100 ish with the interest in royalties. And so that’s $200,000,000 in cash flow. So we’re really excited about moving towards profitability, and we’re going at a really good clip.

Stacy Ku, Biotech Analyst, TD Cowan: Wonderful. Well, as always, pleasure to have you all.

John Stonehouse, CEO, BioCryst: Yeah. Great to see you. Thank you.

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